– The Extroducer® Infusion Catheter System® enables local delivery of XC001 to the heart without the need for surgery –

– XC001 has achieved positive Phase 1/2 results in the EXACT Trial validating its transformative potential for treatment of refractory angina in patients who have exhausted available treatment options and have a debilitating quality‑of‑life –

Wayne, PA & Tullinge, Sweden, July 10, 2024 — XyloCor Therapeutics, Inc., (“XyloCor”), a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, and SmartWise, a unit of SmartCella Holding AB (“SmartCella”), have entered into a licensing agreement under which XyloCor has rights to the Extroducer® Infusion Catheter System®, a first‑in‑class endovascular device designed to deliver advanced therapies directly into the heart [and hard‑to‑reach tissues]. XyloCor plans to deploy the Extroducer to support catheter‑based endocardial delivery of its lead gene therapy candidate, XC001 (encoberminogene rezmadenovec), in future clinical studies and commercial use.

“This agreement with SmartCella will enable XyloCor to build upon its robust foundation of efficacy and safety data for XC001 by offering the potential for improved safety and ease of delivery without surgery via this novel catheter,” said Al Gianchetti, President and CEO of XyloCor. “Teaming up with SmartCella will help in our effort to optimize patient safety and tolerability while maintaining accurate delivery of XC001 to target areas in the heart for patients with refractory angina. It also opens up the potential to develop XC001 earlier in the coronary artery disease progression for even larger patient groups.”

XC001 is designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple isoforms of vascular endothelial growth factor (VEGF). With the use of the Extroducer catheter, XyloCor can offer patients a better delivery option for local administration of XC001 directly to the heart, that is less invasive and eliminates potential risks associated with surgical administration.

“We welcome the Extroducer delivery of XC001 as it offers a more efficient method for gene therapy administration for patients with refractory angina,” said Timothy D. Henry MD, Interventional Cardiologist and Director of the Lindner Center, The Christ Hospital, Cincinnati, Ohio. “Preclinical models provide strong evidence that this approach will maintain, or even improve the efficacy when compared to surgical delivery and it should lower the risk of complications that may arise from surgical administration. I am looking forward to initiating the Phase 2b trial of XC001 in patients with refractory angina using this innovative administration approach”

The recently published EXACT Phase 1/2 trial assessed the use of one‑time gene therapy with XC001 as a new therapeutic approach in refractory angina – a debilitating and chronic condition that impacts over one million people in the United States and is growing in prevalence. In the EXACT trial, 42 patients with class II‑IV angina were treated with XC001 directly administered to the heart following minimally invasive surgical access. The results demonstrated that treatment with XC001 can be safely administered and achieve durable clinical improvements of exercise duration, and angina frequency, due to a decrease in ischemic burden, as measured by Positron Emission Tomography (PET) imaging. Notably, six months after treatment 43% of patients had no chest pain with ordinary activities and 58% reported no angina episodes at 12‑month clinical follow up. XC001 was well tolerated in the patient population and there were no serious adverse events related to the drug. The Phase 2b trial will be a randomized double‑blinded study assessing the safety and efficacy of XC001 administered via the Extroducer® Delivery Catheter in coronary artery disease patients with refractory angina.

“The collaboration underscores the transformative potential of the Extroducer in delivering XC001 therapy for patients with refractory angina. A great example of a powerful combination of delivery system and drug therapy representing a substantial advancement in treatment options. The collaboration with such a distinguished partner as XyloCor marks a significant milestone for our global expansion efforts and will also enable us to further explore and harness the future capabilities of the Extroducer, ultimately expanding the benefits to a greater number of patients in need,“ said Niklas Prager, CEO of SmartCella.

Terms of the agreement include a global license to XyloCor for use of the Extroducer for the administration of XC001 and provide for SmartCella to supply catheters to XyloCor in clinical trials and commercial use in exchange for an upfront payment, clinical, regulatory and commercial milestones and a royalty on sales. Total deal value amounts to approximately USD 130 million and mid‑single digit royalties.

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one‑time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

About Extroducer® Infusion Catheter System

The Extroducer® Infusion Catheter System is a first‑in‑class endovascular delivery device which enables direct‑to‑tissue drug delivery. The Extroducer® addresses a significant unmet need in the field of novel therapies, enabling targeted delivery of a wide range of modalities for solid tumor treatment, genetic disorders and tissue repair, to name but a few. Using standard equipment and routine interventional radiology approaches, the Extroducer provides access to hard‑to‑reach tissues by safely penetrating the vessel wall and delivering payload directly to the target location. Smartwise received U.S. Food and Drug Administration (FDA) clearance under 510(k) for the Extroducer® delivery catheter in June 2022.

About XyloCor

XyloCor Therapeutics, Inc. is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

About SmartCella

SmartCella, founded in 2014, is an innovative biotechnology company based in Stockholm, Sweden. SmartCella’s vision is to combine first‑in‑class delivery platforms with cutting‑edge cell and mRNA therapies to unleash the full potential of targeted therapies. The company has three main business units, Smartwise, SmartCella Solutions and ProCella. For more information, visit www.smartcella.com.

Contacts

Corporate and Investor Relations:

A. Brian Davis, XyloCor Therapeutics
brian.davis@xylocor.com
610-541-2056

Media Contact:

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

SmartCella Contact:

Niklas Prager, CEO
+46 768 117744;
niklas.prager@smartcella.com
Source: XyloCor Therapeutics, Inc.

click here to download the PDF

– Positive Phase 2 EXACT Trial results validate transformative potential of novel gene therapy XC001 for treatment of refractory angina and support continued clinical development –

– Novel therapeutic approach aims to fill significant unmet medical need for patients with refractory angina who have exhausted available treatment options and have a debilitating quality‑of‑life –

Wayne, PA, May 2, 2024 — XyloCor Therapeutics, Inc., a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today presented final results from the Phase 2 portion of its Phase 1/2 clinical trial (EXACT) of its lead gene therapy candidate XC001 (encoberminogene rezmadenovec) for refractory angina at the Society for Cardiovascular Angiography & Interventions (SCAI) 2024 Scientific Sessions, May 2‑4, 2024 in Long Beach, CA. These encouraging results supporting XC001’s safety and efficacy potential are being simultaneously published in Circulation: Cardiovascular Interventions.

The EXACT trial assessed the use of one‑time gene therapy with XC001 as a new therapeutic approach in refractory angina – a debilitating and chronic condition that impacts over one million people in the United States and is growing in prevalence. XC001 is designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple vascular endothelial growth factor (VEGF) isoforms. In the Phase 2 portion of the EXACT trial, 32 patients with class II‑IV angina were dosed with the maximal dose of XC001 through minimally‑invasive transepicardial delivery (direct administration to the heart).

“The results of the EXACT trial suggest that angiogenic gene therapy with XC001 has the potential to improve cardiovascular outcomes for refractory angina patients without revascularization or other treatment options,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and Kenta Nakamura, M.D., Assistant Professor at the University of Washington, lead authors of the EXACT study results. “There is significant need for novel therapies for this serious and disabling condition and we hope that the clinically meaningful evidence emerging from the EXACT trial is the catalyst for continued development that will further validate the potential of this innovative gene therapy for patients.”

The Phase 2 results validated the transformative disease modifying potential of XC001 to reduce ischemia and improve the quality‑of‑life for cardiac patients who have no treatment options. The results demonstrated that treatment with XC001 can be safely administered and achieve durable clinical improvements including: increases in exercise duration, decrease in ischemic burden as measured by Positron Emission Tomography (PET) imaging, and a reduction in angina frequency. Notably, 93% of patients in the trial entered the trial with chest paint so severe that it markedly limited daily activities and six months after treatment 43% of patients had no chest pain with ordinary activities. VEGF gene therapy with XC001 was well tolerated in the patient population and there were no serious adverse events related to the drug or unexpected serious adverse events related to XC001 administration.

“The results from the EXACT trial represent a promising moment for people with refractory angina and the cardiovascular community as we drive forward toward our goal to deliver a long overdue new treatment option,” said Al Gianchetti, President and CEO of XyloCor. “We are preparing our next clinical trial to advance the development of XC001 and further unlock its transformative medical potential for patients and their families.”

Details regarding the SCAI 2024 scientific session is as follows:

Title: VEGF Gene Therapy Improves Exercise Time, Ischemia, and Symptoms in Patients with Refractory Angina: Results of the Phase II EXACT Trial

Lead Presenter: Kenta Nakamura, M.D., Associate Professor at the University of Washington

Date and Time: Thursday, May 2, 2024; 9:31‑9:38 AM PT

Location: Long Beach Convention Center, 104A, First Level

An additional press release from SCAI on the Phase 2 EXACT Trial results and poster session is available here.

The Circulation: Cardiovascular Interventions full article titled “Angiogenic Gene Therapy for Refractory Angina: Results of the EXACT Phase 2 Trial” is available here.

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one‑time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 multicenter, open‑label, single‑arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial in which additional subjects were enrolled at the highest tolerated dose (1 x 10¹¹ vp, the highest tested dose). In the EXACT trial, this investigational gene therapy was administered directly to the heart muscle through a mini‑thoracotomy by a cardiac surgeon.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

About XyloCor

XyloCor Therapeutics, Inc. is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Corporate and Investor Relations:

A. Brian Davis, XyloCor Therapeutics
brian.davis@xylocor.com
610-541-2056

Media Contact:

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– Positive Phase 2 EXACT Trial results at 6‑months underscore significant potential of investigational therapy in refractory angina –

– Six‑month data have since been sustained out to 12‑months supporting durability of XC001 safety and efficacy profile –

– XC001 targets unmet medical need among patients with refractory angina who have a debilitating quality‑of‑life burden and no available treatment options –

Wayne, PA, August 25, 2023 — XyloCor Therapeutics, Inc., a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today presented results from the Phase 2 portion of its Phase 1/2 clinical trial (EXACT) of its lead gene therapy candidate XC001 (encoberminogene rezmadenovec) for refractory angina at the European Society of Cardiology (ESC) Congress 2023. During the ESC Congress poster session, Dr. Thomas Povsic, Principal Investigator of the EXACT trial, presented the primary six‑month outcome data from the recently completed EXACT trial, which demonstrated that treatment with XC001 resulted in improvements across all key efficacy endpoints. The findings underscore its strong potential as a novel therapeutic approach for the treatment of this disabling condition.

XC001 is a one‑time gene therapy designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple vascular endothelial growth factor (VEGF) isoforms. In the Phase 2 portion of the EXACT trial, 32 patients with class II‑IV angina were dosed with the maximal dose of XC001 through transepicardial delivery (direct administration to the heart). XC001 met all of its safety and exploratory objectives and showed potential transformative benefits for the patient population. Among the notable topline results presented at the ESC Congress 2023 included:

• VEGF gene therapy with XC001 administered via minimally invasive transepicardial delivery was generally well tolerated.
• There were no serious adverse events related to the drug or unexpected serious adverse events related to XC001 administration.
• Patients demonstrated improvements in key efficacy measures most notably total exercise time, time to the development of ST‑depression (an objective measure of ischemia), angina frequency, and reduction in ischemic burden as measured by Positron Emission Tomography (PET) imaging.
• The six‑month results showed that XC001 achieved a clinically meaningful biologic effect, warranting further study in larger randomized clinical trials.

“Refractory angina is a debilitating and chronic condition that is growing in prevalence and these patients have exhausted all treatment options,” said Thomas Povsic,  M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “The six‑month results from the EXACT trial – which have now been sustained out to 12 months – demonstrate that gene therapy with XC001 has the potential to be safely administered while improving quality of life for these cardiac patients.”

“The clinical research results presented at ESC Congress 2023 highlight XyloCor’s continuing efforts to transform the treatment paradigm in refractory angina through the promise of one‑time gene therapy,” said Al Gianchetti, President and CEO of XyloCor. “We are excited to share data that provides evidence for angiogenesis and a promising efficacy and tolerability profile for XC001. These results strongly support our continued development of this novel therapeutic approach.”

Further background and results presented in the ESC Congress 2023 poster session titled “Angiogenic gene therapy for refractory angina: Results of the Epicardial delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) Phase 2 Trial” can be found here.

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one‑time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 multicenter, open‑label, single‑arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial in which additional subjects were enrolled at the highest tolerated dose (1 x 10¹¹ vp, the highest tested dose). In the EXACT trial, this investigational gene therapy is administered directly to the heart muscle through a mini‑thoracotomy by a cardiac surgeon.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

About XyloCor

XyloCor Therapeutics, Inc. is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Corporate and Investor Relations:

A. Brian Davis, XyloCor Therapeutics
brian.davis@xylocor.com
610-541-2056

Media Contact:

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– Findings from the Phase 1 dose escalation portion of the EXACT trial of XC001 in refractory angina provided the dose selection and safety justification for the recently completed Phase 1/2 study –

– XC001 is a one‑time gene therapy candidate designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple VEGF isoforms –

– XyloCor is moving forward with urgency to address potential of XC001 as transformative therapy for patients with ischemic heart disease with significant unmet need –

Wayne, PA, August 3, 2023 — XyloCor Therapeutics, Inc., a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, announced today that Circulation: Cardiovascular Interventions has published results from the Phase 1 portion of its Phase 1/2 clinical trial (EXACT) of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in patients with refractory angina. The findings from the Phase 1 dose escalation study, previously reported at the American Association for Thoracic Surgery (AATS) and the American Society of Gene and Cell Therapy (ASGCT) in May 2022, revealed that XC001 is well tolerated at all dose levels and provided justification to proceed to Phase 2 with the highest dose tested.

“The results from the Phase 1 study provided the mechanistic underpinning that was the catalyst for the successful completion of the Phase 2 EXACT trial,” said Thomas Povsic, M.D., Ph.D., lead author of the journal article, Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “Patients with refractory angina are highly symptomatic and have an exceedingly poor quality of life. With a robust body of positive and sustained safety and efficacy out to 12 months from the EXACT trial, we believe that XC001 has the potential to fill the significant unmet need for this patient population who currently lack treatment options.”

“We are thrilled with the publication of these EXACT trial results in Circulation: Cardiovascular Interventions, a highly‑regarded and influential international journal for cardiovascular research,” said Howard Dittrich, Chief Medical Officer of XyloCor. “We would like to acknowledge all of the authors for their contributions in highlighting the promise of XC001 and thank patients and their families for their participation in the EXACT trial. Our team is singularly focused on continuing to unlock the transformative potential of XC001 for improving outcomes in cardiovascular disease.”

The Phase 1 portion of the Phase 1/2 EXACT study was a first‑in‑human, multicenter, open‑label, single‑arm, dose‑escalation study to evaluate the safety, tolerability, and preliminary efficacy of increasing doses of XC001, and to establish the best dose to carry forward for additional study in Phase 2. Twelve patients were enrolled into four dosing cohorts. Notably, the study demonstrated that adenoviral vector doses higher than those used in previous studies were well tolerated and more robust efficacy was demonstrated at the higher doses. This established a dose of 1x10¹¹ viral particles for future clinical research of XC001.

The Circulation: Cardiovascular Interventions full article is available at https://www.ahajournals.org/doi/abs/10.1161/CIRCINTERVENTIONS.123.012997

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one‑time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 multicenter, open‑label, single‑arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial in which additional subjects were enrolled at the highest tolerated dose (1 x 10¹¹ vp, the highest tested dose). The investigational gene therapy is administered directly to the heart muscle through a mini‑thoracotomy by a cardiac surgeon.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

About XyloCor

XyloCor Therapeutics, Inc. is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Corporate and Investor Relations:

A. Brian Davis, XyloCor Therapeutics
brian.davis@xylocor.com
610-541-2056

Media Contact:

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– XC001 demonstrated durable improvements across multiple efficacy measures 12 months after treatment, underscoring its scientifically‑sound approach to achieve biological effect and improve angina symptoms –

– Patients showed continued improvements in exercise capacity and reductions in episodes of chest pain that were sustained to 12 months –

– Robust body of mechanistic evidence from EXACT trial highlights significant potential of XC001 in cardiovascular disease –

Wayne, PA, July 18, 2023 — XyloCor Therapeutics, a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today reported positive 12‑month data from the Phase 2 portion of its Phase 1/2 clinical trial (EXACT) designed to assess the safety and provide preliminary evidence of efficacy of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in patients with refractory angina. At the 12‑month mark in the extension period of the trial, XC001 demonstrated durable improvements across multiple efficacy measures, including continued improvement in total exercise duration and reductions in ischemic burden and ischemic symptoms. Earlier this year, XyloCor reported positive results from the primary study period for the Phase 2 portion EXACT trial at six months. New results at 12 months highlight significant, clinically‑meaningful impacts that are now sustained out to 12 months, pointing to the potential of XC001 as a novel therapeutic approach for the significant unmet medical need in refractory angina.

“The durability and, in the case of exercise time, continued improvements observed at 12 months signals a sustainable activity which is an exciting step forward in the advancement of gene therapy for cardiovascular disease,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “These 12‑month data build upon the positive results achieved at the 3- and 6‑month marks of the trial. In total, the outcomes of the EXACT study form a robust body of mechanistic evidence to propel the next stage of XC001’s development, suggesting that a single treatment may have long‑term benefit.”

XC001 is a one‑time gene therapy candidate designed to reduce ischemic burden by creating new blood vessels in the heart. The six‑month primary study period in the Phase 2 portion of the EXACT trial was followed by a month 12 follow up period. At 12 months, patients demonstrated sustained and continued increases in total exercise duration (TED) over baseline, representing a significant and clinically meaningful change. In addition, there was a sustained and robust decrease in episodes of chest pain (angina) and nitroglycerin use. Cardiac imaging at 12 months provided additional evidence of the potential mechanism of action to achieve a biological effect, confirmed by a sustained reduction in ischemic burden observed over time.

“With the 12‑month results from our EXACT trial, XyloCor continues to take a lead role in fulfilling the promise of gene therapy for people with cardiovascular disease,” said Al Gianchetti, President and CEO of XyloCor. “These results further enhance our confidence that we are on the right path for transforming outcomes in cardiovascular disease.”

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one‑time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 multicenter, open‑label, single‑arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial in which additional subjects were enrolled at the highest tolerated dose (1 x 10¹¹ vp, the highest tested dose). The investigational gene therapy is administered directly to the heart muscle through a mini‑thoracotomy by a cardiac surgeon.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

About XyloCor

XyloCor Therapeutics is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Corporate and Investor Relations

A. Brian Davis, XyloCor Therapeutics
brian.davis@xylocor.com
610-541-2056

Media Contact

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– No serious adverse events related to drug product were reported –

– Patients demonstrated improvements in exercise capacity and reductions in episodes of chest pain –

– Cardiac imaging results provide mechanistic evidence supporting the therapeutic potential of XC001 in cardiovascular disease –

Wayne, PA, January 26, 2023 — XyloCor Therapeutics, a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced completion of the Phase 2 portion of its Phase 1/2 clinical trial (EXACT) designed to assess the safety and preliminary evidence of efficacy of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in patients with refractory angina. EXACT met both safety and efficacy objectives. There were no safety issues related to drug product or unexpected serious adverse events related to XC001 administration. Six‑month data from 28 patients in the Phase 2 portion of the study showed improvements in several key efficacy measures, including reduction in ischemic burden.

“We are excited to see EXACT completing its 6‑month endpoint. The trial met all of its safety and exploratory objectives, showing intriguing benefits in these needy patients across a variety of objective and subjective measures,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “The strong range of mechanistic evidence demonstrate that administration of XC001 is a scientifically‑sound approach for achieving a biological effect that has the potential to improve patients’ quality of life.”

XC001 is a one‑time gene therapy designed to reduce ischemic burden by creating new blood vessels in the heart. In the Phase 2 portion of the EXACT trial, evidence of the drug’s mechanism of action was demonstrated by the reduction of ischemic burden measured by cardiac positron emission tomography (PET) imaging. The reduction in ischemic burden was accompanied by an improvement in total exercise duration, an important measure of exercise capacity. Prior to treatment, almost all subjects had marked limitations on ordinary physical activity. Six months after treatment, nearly half of all subjects were able to conduct ordinary physical activity without causing angina. The data from the Phase 2 EXACT study are potentially meaningful for patients with refractory angina, which includes more than one million people in the United States, who have no treatment options.

“We are excited to share this positive topline data from the Phase 2 portion of the EXACT trial, reinforcing our confidence in XC001 as a novel therapeutic approach with the potential to address the significant unmet medical needs of people with refractory angina,” said Al Gianchetti, President and CEO of XyloCor. “We now look forward to pursuing key upcoming milestones in XC001’s continued development, including finalizing our pivotal trial design through our ongoing discussions with the FDA and other regulatory authorities.”

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one‑time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

About the EXACT Study

The recently completed Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 multicenter, open‑label, single‑arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial in which additional subjects were enrolled at the highest tolerated dose (1 x 10¹¹ vp, the highest tested dose). The investigational gene therapy is administered directly to the heart muscle through a mini‑thoracotomy by a cardiac surgeon.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

About XyloCor

XyloCor Therapeutics is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Corporate and Investor Relations

A. Brian Davis, XyloCor Therapeutics
brian.davis@xylocor.com
610-541-2056

Media Contact

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– Positive Phase 1 results reported at the American Association for Thoracic Surgery (AATS) and the American Society of Gene and Cell Therapy (ASGCT) revealed XC001 is well tolerated at all dose levels –

– Phase I data support XC001 therapeutic effect and potential dose response –

– Topline Phase 2 data readout expected in February 2023 with interim results in the second half of this year –

Wayne, PA, June 28, 2022 — XyloCor Therapeutics, a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced it has achieved enrollment of target number of subjects in the Phase 2 portion of its ongoing Phase 1/2 clinical trial (EXACT) for refractory angina. Topline results from the Phase 2 study are expected in February 2023 with interim results in the second half of this year.

“Achievement of this important milestone in the Phase 2 portion of the study is a testament to the clinical need in this patient population and I am eager to see the Phase 2 results as they emerge,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “Patients with refractory angina have no treatment options, and the results from the Phase 1 portion of the EXACT trial suggest a dose response and therapeutic potential which is encouraging for the development of XC001 as a treatment to improve these patients’ quality of life. We are very excited to see this more definitive evaluation of the safety and efficacy of this approach.”

“We are pleased to announce this important milestone in enrollment for our Phase 2 study especially during this unprecedented and challenging time,” said Al Gianchetti, President and CEO of XyloCor. “An estimated one million people suffer from refractory angina in the United States, and we are encouraged that XC001 may address the high unmet need in this patient group. XyloCor also plans to study XC001 in other patient groups as well, including as adjunctive therapy in patients undergoing bypass surgery.”

Individuals with refractory angina experience pressure or intense pain in the chest due to insufficient blood flow to the heart muscle. These symptoms can severely impact quality of life and may worsen comorbidities.

XyloCor’s lead investigational drug, XC001 (encoberminogene rezmadenovec) is a locally administered, single‑dose gene therapy currently in development as a novel approach to treating patients with refractory angina who have no other medical and surgical options. The treatment strategy is to use local administration to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects. XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial is a Phase 1/2 multicenter, open‑label, single‑arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial with target enrollment of 27 additional subjects at the highest tolerated dose (1 x 10¹¹ vps, the highest tested dose). The investigational gene therapy is administered directly to the heart muscle through a mini‑thoracotomy by an experienced cardiac surgeon at top cardiovascular research sites across the United States.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

About XyloCor

XyloCor Therapeutics is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Corporate and Investor Relations

A. Brian Davis, XyloCor Therapeutics
brian.davis@xylocor.com
610-541-2056

Media Contact

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– Data from the Phase 1 dose‑escalation portion of the Phase 1/2 EXACT study demonstrate XC001 was well‑tolerated at all dose levels tested; highest dose level evaluated selected for ongoing Phase 2 portion of the study –

– Preliminary efficacy data highlight XC001 potential for patients with refractory angina with no other treatment options –

– Treatment strategy is to use local administration to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects –

– Completion of Phase 2 enrollment is expected by the end of May 2022 –

Wayne, PA, May 18, 2022 — XyloCor Therapeutics, a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced presentation of initial clinical data from the Phase 1 portion of its ongoing Phase 1/2 clinical trial (EXACT) for refractory angina at the American Association for Thoracic Surgery (AATS) Annual Meeting on May 15, 2022, and at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting on May 18, 2022.

XC001: Locally administered, single‑dose gene therapy candidate to address the unmet need in refractory angina

XyloCor’s lead investigational drug, XC001 (encoberminogene rezmadenovec), is under development as a novel approach to treating patients with refractory angina who have exhausted other medical and surgical options. This investigational gene therapy is designed to activate naturally occurring biological pathways by creating new vessels to improve blood flow to areas of the heart not receiving adequate blood supply. This restored blood supply could potentially improve patients’ quality of life by enabling them to resume physical activities and it could reduce episodes of chest pain associated with refractory angina.

In the Phase 1 portion of the EXACT study, 12 subjects with Canadian Cardiovascular Society (CCS) angina class 2‑4 without revascularization options were divided into four escalating dose groups with three subjects each. Each subject received 15 epicardial injections of XC001 at one of the four dosage levels. Safety, efficacy and tolerability evaluations were measured as adverse events (AEs), serious adverse events (SAEs) and change from baseline from three to six months post‑treatment in exercise capacity, ischemic burden by positron emission tomography (PET) imaging, and patient‑reported symptomatology.

No drug‑related SAEs, bleeding complications or ventricular arrhythmias were observed in this Phase 1 dose‑escalation study. Over a six‑month follow up there were a total of 17 SAEs in seven subjects. Eleven SAEs were related to the underlying disease process or other causes. The other six SAEs which occurred in four subjects were judged to be related to the administration procedure, with none of those being unexpected nor resulting in patient death.

“The administration of XC001 appears to have been well‑tolerated at all tested doses,” said Nahush Mokadam, M.D., presenting author at AATS, Division Director, Cardiac Surgery, The Ohio State University Wexner Medical Center and Associate Director of the Heart and Vascular Center and site Principal Investigator for this study. “Objective criteria, including results from exercise tolerance tests and PET scans, suggest therapeutic potential.”

Although the Phase 1 portion of the study was primarily focused on safety and Phase 2 dose selection, initial clinical efficacy data appeared promising. Notably, the data showed positive trends in total exercise duration and reductions in patient symptoms and ischemic burden. Although patient numbers are small, preliminary data suggest that response may be correlated to administered dose.

“The preliminary efficacy evaluation suggests a dose response which is encouraging for the development of XC001 as a therapeutic strategy,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “We anticipate that the Phase 2 expansion portion of this study, which is testing the highest and most efficacious dose from Phase I, will complete enrollment this month. We are incredibly excited by the potential for this investigational therapy to improve the quality of life for these cardiac patients.”

“In many other gene therapy trials, safety concerns arose due to systemic administration of high viral particle loads,” added Dr. Povsic. “In contrast, because we can inject XC001 directly into the heart, we can dramatically reduce overall viral particle loads and systemic exposure while increasing efficacy.”

EXACT Phase 1/2 Study Data Presentations at AATS and ASGCT

Lead author, Dr. Mokadam presented three‑month data from the Phase 1 study in the presentation, Dose Escalation Study of Encoberminogene Rezmadenovec (Adenoviral Vector with Multiple Isoforms of Vascular Endothelial Growth Factor) in Refractory Angina: Phase 1 Results at the AATS Annual Meeting.

Dr. Povsic will present six‑month data from the Phase 1 study in the presentation, Preliminary Safety, Tolerability and Efficacy of Direct Epicardial Administration of Encoberminogene Rezmadenovec to Ischemic Myocardium in Patients with Refractory Angina: Six Month Phase 1 Data at the ASGCT 25th Annual Meeting.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial is a Phase 1/2 multicenter, open‑label, single‑arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, to be followed by an expansion phase of the trial with 27 additional subjects at the highest tolerated dose. The trial is designed to assess the preliminary safety and efficacy of XC001. The investigational gene therapy is administered directly to the heart muscle through a mini‑thoracotomy by an experienced cardiac surgeon. The EXACT trial is being conducted at top cardiovascular research sites across the United States.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain. An estimated one million people suffer from refractory angina in the United States.

About XyloCor

XyloCor Therapeutics is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Media Contact

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– Elizabeth Tarka, M.D. appointed Chief Medical Officer –

– A. Brian Davis named Chief Financial Officer –

Wayne, PA, January 18, 2022 — XyloCor Therapeutics, a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced that it has appointed Elizabeth Tarka, M.D. as Chief Medical Officer and A. Brian Davis as Chief Financial Officer. These experienced pharmaceutical industry executives will enhance the company’s clinical development, operational, and financial capabilities and drive its ongoing growth.

“I am delighted to welcome Liz and Brian to the leadership team at XyloCor,” said Al Gianchetti, President and Chief Executive Officer. “Liz is a proven R&D leader who brings a strong track record managing late‑stage clinical development programs with particular expertise in cardiovascular medicine. Brian will leverage his extensive management and finance experience in both private and public biotech companies to accelerate Xylocor’s strategic and corporate objectives. Their collective experience will drive XyloCor toward our objectives as we focus on advancing our pipeline of transformative gene therapies intended to improve the lives of people with cardiovascular disease.”

Dr. Tarka is a cardiologist with over 20 years of experience in the pharmaceutical and biotechnology industry. She has dedicated her career to the development of innovative therapies that improve human health. Dr. Tarka’s experience includes leadership roles across all phases of late‑stage clinical development and a track record of effectively partnering with stakeholders to enable the successful execution of clinical trials. She joins the company from Idera Pharmaceuticals where she served as CMO. Before that, Dr. Tarka was Vice President, Clinical Development at Complexa, Inc., a clinical stage biopharmaceutical company focused on life‑threatening fibrosis and inflammatory diseases. Earlier in her career, she served as Clinical Program Leader for Xarelto® (rivaroxaban) at Janssen Pharmaceuticals, where she was responsible for the design, implementation, and medical oversight for large multinational trials. Prior to her tenure at Janssen, Dr. Tarka worked at GlaxoSmithKline in the Metabolic Pathways and Cardiovascular Therapeutic Area. She has been on the faculty and had numerous major teaching and clinical responsibilities at the University of Pennsylvania and affiliated hospitals. She is trained in Cardiology and Internal Medicine and has published in a number of peer‑reviewed journals. Dr. Tarka earned a BA in Biochemistry and an MD from the University of Pennsylvania where she also completed her residency and fellowship training.

Mr. Davis joins XyloCor with a proven background as a seasoned financial executive, including over 15 years of experience as a CFO for publicly traded, commercial- and clinical‑stage biopharmaceutical companies, and nearly 30 years as a financial professional in the life sciences industry. He has extensive expertise in fundraising, financial strategy, negotiating strategic transactions involving acquisition and disposition of commercial and clinical‑stage assets, shareholder relations, and SEC accounting, reporting, and compliance. Mr. Davis has raised over $600 million in public and private equity financings, including leading an initial public offering, and nearly $200 million in debt financings. Most recently, he was CFO at Verrica Pharmaceuticals, where he held managerial responsibility for executing equity and debt financings, analyst and shareholder relations, financial aspects of commercial launch preparation, business development, finance, accounting, tax, and treasury. Before that, Mr. Davis had similar duties as CFO at Strongbridge Biopharma plc, Tengion, Inc., and Neose Technologies, Inc. Mr. Davis is a Certified Public Accountant. He earned an MBA from The Wharton School, University of Pennsylvania and an undergraduate degree in Accounting from Trenton State College.

About XyloCor

XyloCor Therapeutics is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The company’s lead product candidate, XC001, is currently being investigated in a Phase 2 clinical trial for patients with refractory angina for which there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, MD, and Todd Rosengart, MD, has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Corporate and Investor Relations:

A. Brian Davis
XyloCor Therapeutics
brian.davis@xylocor.com
610-541-2056

Media Contact

Mike Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– Independent Data Monitoring Committee authorized proceeding to Phase 2 at highest dose level tested following review of clinical safety data from the Phase 1 dose escalation –

– Phase 2 clinical data readouts on safety and efficacy of XC001 anticipated in 2022 –

– Company plans to commence study startup of XC001 as an adjunct to CABG in 2H21, and clinical studies in additional cardiovascular indications are under discussion –

– Progress highlights potential of gene therapy in cardiovascular disease –

Wayne, PA, July 27, 2021 — XyloCor Therapeutics, a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced the completion of the Phase 1 dose‑escalation component of its Phase 1/2 clinical trial (EXACT) of XC001 (encoberminogene rezmadenovec), its lead investigational gene therapy candidate for patients with refractory angina who have no further treatment options.

Following a review of clinical data from the Phase 1 dose escalation component of the study, the Independent Data Monitoring Committee (IDMC) authorized proceeding to the Phase 2 component of the study at the highest dose tested. Since the IDMC authorization, three patients have been dosed in the Phase 2 expansion cohort. This progress highlights the potential for gene therapy to go beyond rare diseases and to address larger patient populations with significant unmet needs, such as chronic cardiovascular diseases.

XyloCor also confirms that it plans to submit an additional Phase 2 clinical study to the U.S. Food and Drug Administration (FDA) for XC001 as adjunctive therapy to coronary artery bypass grafting (CABG) in 2H21. The company also plans other clinical studies in additional cardiovascular indications, including heart failure caused by ischemic heart disease and as adjunctive therapy to percutaneous coronary intervention.

“Patients with refractory angina are forced to live with the ongoing burden of a disease that limits their activities on a daily basis due to chest pain,” said Thomas Povsic, M.D., Ph.D., Duke University cardiologist and National Principal Investigator for the EXACT study. “With a unique mechanism of action that restores blood flow to the heart via the creation of new blood vessels, XC001 represents a novel therapeutic approach for patients who have exhausted other medical and surgical options. It is very exciting to now move forward with exploring XC001’s potential in the Phase 2 portion of EXACT as a one‑time therapy for patients with refractory angina.”

“In our mission to deliver safe and effective gene therapies that transform the lives of people with cardiovascular disease, we are excited to achieve this important milestone and advance into the Phase 2 portion of our study,” said Al Gianchetti, President and Chief Executive Officer of XyloCor Therapeutics. “XC001 has enormous potential to significantly improve the lives of patients with refractory angina. We are grateful for the support of patients and their families, as well as the EXACT trial investigators as we continue to study the safety and efficacy of XC001 and look forward to reporting results in 2022.”

About XC001

XC001 (encoberminogene rezmadenovec) is a novel, investigational gene therapy designed to stimulate the growth of new blood vessels in the heart, in order to bypass diseased vessels and improve coronary blood flow. XC001 delivers the gene for vascular endothelial growth factor (VEGF), a naturally occurring protein, in targeted myocardial cells, thus stimulating the creation of new blood vessels via a process called angiogenesis. XC001 employs a proprietary multi‑isoform VEGF expression cassette that has been optimized to maximize expression of VEGF. XC001 has been granted Fast Track designation by the FDA for study in refractory angina. XyloCor commenced the EXACT Trial, a Phase 1/2 study of XC001 in chronic refractory angina, in 2020.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial is a Phase 1/2 multicenter, open‑label, single‑arm, dose‑escalation trial. 12 subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, to be followed by an expansion phase of the trial with 21 additional subjects at the highest tolerated dose. The trial is designed to assess the preliminary safety and efficacy of XC001. The investigational gene therapy is administered directly to the heart muscle through a mini‑thoracotomy by an experienced cardiac surgeon. The EXACT Trial is being conducted at top cardiovascular research sites across the United States.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain. An estimated one million people suffer from refractory angina in the United States.

About XyloCor

XyloCor Therapeutics is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co-founded by Ronald Crystal, MD, and Todd Rosengart, MD, has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Media Contact

Mike Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– Company closes additional $22.6 million in new financing –

– Proceeds will fund initiation of new clinical trial for lead gene therapy candidate XC001 as adjunctive therapy for patients undergoing coronary artery bypass graft surgery –

Malvern, PA, March 22, 2021 — XyloCor Therapeutics, a private clinical‑stage biopharmaceutical company focused on the development of gene therapy for the significant unmet patient needs in advanced coronary artery disease, today announced the closing of an additional $22.6 million financing. Fountain Healthcare Partners led the oversubscribed financing joined by new investors Longwood Fund and Lumira Ventures. All existing institutional investors including Sofinnova Investments and LSP (Life Sciences Partners) participated in the financing. The additional financing builds upon XyloCor’s 2018 Series A financing round, bringing total investment in the company to $41.9 million to date. XyloCor’s lead product candidate, XC001, is an investigational gene therapy currently being studied in a Phase 1/2 clinical trial (EXACT) for patients with refractory angina, a chronic condition for which there are no treatment options.

The financing will enable XyloCor to expand its clinical development program for XC001, including the initiation of a new trial of XC001 as a potential adjunctive therapy to augment the effectiveness of coronary artery bypass graft surgery (CABG). XyloCor is at the forefront of scientific research and clinical study in the application of gene therapy to address vast unmet treatment needs in large patient populations with cardiovascular disease. In both its initial potential indication in refractory angina, and as an adjunctive therapy for patients undergoing CABG, XC001 represents a novel therapeutic approach.

“We greatly appreciate the recognition by Fountain Healthcare Partners, Longwood Fund, and Lumira Ventures of the value we have created since our initial funding and in XC001’s enormous potential for improving the lives of patients with advanced coronary disease,” said Al Gianchetti, president and chief executive officer of XyloCor Therapeutics. “With the support of our investors, we can build on the progress we have made since our initial funding to pursue, with a sense of urgency, new clinical indications where XC001 has promise for addressing unmet medical needs.”

“XyloCor has created significant value with XC001 with the progress the team has achieved on clinical and CMC milestones. Based on our experience, excellence on both fronts is critical to success in the gene therapy field,” said Aidan King, managing partner and co‑founder, Fountain Healthcare Partners, who also joined XyloCor’s board of directors. “We are gratified that this additional capital accelerates XC001’s development and expands its potential impact to the significant unmet need among CABG patients who are at high risk for incomplete revascularization.”

Board of Directors Additions

Joining Mr. King as a member of the XyloCor board of directors is Daniel Hétu, M.D., managing director, Lumira Ventures, and Perry Nisen, M.D., Ph.D., executive partner, Sofinnova Investments. Alan Colowick, M.D., MPH, will now serve an independent board member of XyloCor.

About XC001

XC001 is an investigational gene therapy designed to promote the growth of new blood vessels in the heart, with these new blood vessels bypassing diseased blood vessels and improving blood flow in the heart. XC001 deposits the gene for vascular endothelial growth factor (VEGF) in targeted heart cells. VEGF is a naturally occurring protein and it is believed that XC001 enables the heart cells to produce more VEGF, thus stimulating the creation of new blood vessels, a process called angiogenesis. XC001 has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for study in refractory angina. An Investigational New Drug (IND) application for XC001 is open with the FDA. XyloCor commenced the EXACT Trial, a Phase 1/2 study of XC001 in chronic refractory angina in 2020.

The EXACT Trial

The EXACT clinical trial is a Phase 1/2 multicenter, open-label, single arm, dose escalation trial. Approximately 12 subjects (n=3 per cohort) who have refractory angina will be enrolled into 4 ascending dose groups, followed by an expansion phase of the trial with 21 additional subjects at the highest tolerated dose. The trial is designed to assess the safety and efficacy of XC001. The investigational gene therapy will be administered directly to the muscle tissue of the heart by an experienced cardiac surgeon. The EXACT Trial was initiated in 2020 and is ongoing at top cardiovascular research sites across the United States.

About Chronic Refractory Angina

Chronic angina pectoris occurs when the heart muscle does not receive as much oxygen as it needs for the amount of work it is performing, and this often results in chest pain. This is usually due to coronary artery disease. Patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and CABG have refractory angina and currently have no treatment options. These patients often become sedentary because of their symptoms, which in turn can exacerbate comorbidities and severely impact quality of life causing further deterioration of their health status. An estimated one million people suffer from refractory angina in the United States.

About Coronary Artery Bypass Graft Surgery (CABG)

CABG is a procedure used to treat coronary artery disease — the narrowing or blockage of the blood vessels that supply oxygen and nutrients to the heart muscle. During CABG, a healthy artery or vein from the body is connected, or grafted, to the blocked coronary artery. The grafted artery or vein bypasses the blocked portion of the coronary artery. This creates a new passage, and oxygen-rich blood is routed around the blockage to the heart muscle. Approximately 500,000 CABG procedures are performed annually in the United States, in which an estimated one-third of patients are at risk for incomplete coronary revascularization, often resulting in persistent angina. An adjunctive treatment to CABG, such as gene therapy with XC001, may reduce the incidence of incomplete revascularization.

About XyloCor

XyloCor Therapeutics is a biopharmaceutical company focused on the development of novel gene therapy for unmet needs in advanced coronary artery disease. In the United States, coronary artery disease is a leading cause of death and disability. The company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor also has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, MD, and Todd Rosengart, MD, has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Media Contact

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

Malvern, PA, July 21, 2020 — XyloCor Therapeutics today announced it has successfully dosed the first two patients in the EXACT Trial, a Phase 1/2 dose escalation trial evaluating the safety, tolerability and efficacy of its lead candidate XC001 in patients with refractory angina. The trial will enroll patients who are not responding to medication and are unsuitable for coronary artery bypass graft or percutaneous coronary intervention. XC001 is an investigational novel gene therapy designed to activate naturally occurring biological pathways to improve blood flow to areas of the heart not receiving adequate blood supply. One patient was dosed at The Christ Hospital Health Network and a second at Minneapolis Heart Institute Foundation.

“People with refractory angina are forced to make many sacrifices in their lives as chest pain limits their ability to perform ordinary physical activities,” said Al Gianchetti, President and Chief Executive Officer of XyloCor Therapeutics. “Dosing the first patients in this clinical trial is an important milestone in XyloCor’s efforts to advance an innovative treatment that could reduce chest pain and enable people to resume the normal daily activities that improve their overall quality of life.”

“XC001 has the potential as a one‑time gene therapy that will relieve chest pain by restoring blood flow to the heart,” said Rickey Reinhardt, MD, PhD, Chief Medical Officer of XyloCor Therapeutics. “The EXACT trial will provide us with vital data on the safety and efficacy of XC001 and we believe it will confirm evidence seen in previous pre‑clinical and clinical programs with this mechanism of action.”

“Therapeutic treatments for refractory angina are limited and thus results in poor health including frequent angina with an extremely diminished quality of life. There is a tremendous need to explore gene therapy as a viable treatment option for advanced coronary artery disease, especially for patients who have exhausted all other medication and surgical options,” concluded Mr. Gianchetti.

About the EXACT Trial

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) is a Phase 1/2 first‑in‑human, multicenter, open‑label, single arm dose escalation trial evaluating the safety, tolerability and efficacy of XC001 at six months in patients who suffer from chronic angina caused by coronary artery disease with no other treatment options. The trial will enroll 12 patients (n=3 per cohort) who will receive one of four ascending intramyocardial doses of XC001, followed by an expansion cohort of 17 patients of the highest tolerated dose. Secondary endpoints at six months include efficacy measures of improvement in exercise capacity, reduction in angina episodes, improvement in coronary blood flow and improvement in quality of life.

More information about the EXACT trial is available at https://clinicaltrials.gov/ct2/show/NCT04125732.

About XyloCor

XyloCor Therapeutics is a biopharmaceutical company focused on the development of novel gene therapy for unmet needs in advanced coronary artery disease. In the United States, coronary artery disease is a leading cause of death and disability. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor also has a secondary product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. Co‑founded by Ronald Crystal, MD, and Todd Rosengart, MD, XyloCor has an exclusive license agreement with Cornell University for the worldwide rights to develop, manufacture and commercialize XC001. For more information, visit www.xylocor.com.

Media Contact

Mike Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

Philadelphia, PA, June 9, 2020 — XyloCor Therapeutics, a private clinical‑stage biopharmaceutical company focused on the development of gene therapy for unmet needs in cardiovascular disease, strengthens its leadership team with the appointment of Alexander Gaidamaka, PhD, as Senior Vice President of Technology, Manufacturing & Quality. Dr. Gaidamaka brings a wealth of product development, regulatory and manufacturing experience across multiple technology platforms and GMP quality design and operations from his tenures with Sanofi Group, AmpliPhi Biosciences, Personalized OncoTherapeutics Inc. and several other companies.

“At this critical time in our growth, we are excited to have Alexander join our team. His broad therapeutics experience from pre‑clinical and clinical development through manufacturing processes will be invaluable to both our clinical and pre‑clinical gene therapy programs,” said Al Gianchetti, President and Chief Executive Officer of XyloCor Therapeutics. “Quality manufacturing is imperative when developing gene therapies, and his insights and skillset, developed from experience both in the US and globally, will bring XyloCor Therapeutics one step closer to helping patients with refractory angina who currently have no treatment options.”

With more than 25 years of experience in the pharmaceutical industry, Dr. Gaidamaka previously served as Vice President of Chemistry, Manufacturing & Controls for AmpliPhi Biosciences where he led and defined CMC strategy and application, implemented GMP production of clinical trial material and completed several IND submissions to the U.S. Food and Drug Administration for clinical trials and compassionate use. As Chief Executive Officer and Chief Scientific Officer at Personalized OncoTherapeutics, he developed a scalable technology concept for a personalized therapeutic cancer vaccine platform and conducted early research studies within cross-functional international projects. As Director of Biologics Development at Merial (Sanofi) and Senior Manager of QC Immunochemistry at Sanofi Pasteur, he executed on a broad spectrum of tasks from development to product approval, manufacturing and all aspects of GMP quality systems.

Dr. Gaidamaka began his career in pharmaceuticals by developing a series of generic and brand medicines when serving as the Head of the Department of Immunopharmacology & Allergology at State Scientific Center of Drugs, Ukraine. He then founded Biopharm LLC (Ukraine) and served as Chief Executive Officer. He also served as a subject matter expert for biologics at Ukrainian Pharmacopeia Committee. He received his PhD in immunology and microbiology and a Doctor of Veterinary Medicine from Kharkov Veterinary Academy. He is also an author of 42 publications and holds two patents.

About XyloCor

XyloCor Therapeutics is a biopharmaceutical company focused on the development of novel gene therapy for unmet needs in advanced coronary artery disease. In the United States, coronary artery disease is a leading cause of death and disability. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor also has a secondary product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. Co‑founded by Ronald Crystal, MD, and Todd Rosengart, MD, XyloCor has a licensing agreement with Weill Cornell Medicine for the worldwide rights to develop, manufacture and commercialize XC001. For more information, visit www.xylocor.com.

Media Contact

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– Recognized industry leader to head clinical development programs –

Philadelphia, PA, August 7, 2019 — XyloCor Therapeutics, a private clinical‑stage biopharmaceutical company focused on the development of gene therapy for unmet needs in cardiovascular disease, strengthens its leadership team with the appointment of Rickey Reinhardt, MD, PhD, as Chief Medical Officer. Dr. Reinhardt formerly served as Chief Medical Officer for Comet Therapeutics where he led the strategy and execution of clinical development programs and provided oversight of regulatory affairs.

“Dr. Reinhardt’s broad and deep experience as a scientist and clinical drug developer will be invaluable to XyloCor as we advance our novel gene therapy programs,” said Al Gianchetti, President and Chief Executive Officer of XyloCor. “His proven success in driving drug development from discovery through phase 4 in areas such as oncology, cardiometabolics and rare diseases, including several clinical-stage gene therapy programs, will be a tremendous asset to our company.”

With more than 22 years of experience in the pharmaceutical industry, Dr. Reinhardt will focus on clinical development of XyloCor’s pipeline and its lead candidate, XC001, for the treatment of refractory angina. During his tenure at Comet Therapeutics, Dr. Reinhardt led the company’s clinical development efforts focused on inborn errors of metabolism. Previously, he served as Vice President of Clinical Research and Development at REGENXBIO where he led the cross‑functional team in charge of driving multiple gene therapy programs for the treatment of rare metabolic genetic and ophthalmology diseases.

Prior to joining REGENXBIO, Dr. Reinhardt served as Vice President and Head of Clinical and Alternative Development Programs at GlaxoSmithKline where he directed the development and successful global approvals of Tanzeum/Eperzan for the treatment of diabetes. While at GSK, he also led the European approval of the first ex‑vivo gene therapy for the treatment of severe combined immunodeficiency, Strimvellis, and expanded the label for Nucala to include the treatment of eosinophilic granulomatosis with polyangiitis (EGPA). Dr. Reinhardt has also held senior positions at Covance and Novo Nordisk. At Novo Nordisk, he spent 11 years in the Clinical Pharmacology, Clinical Research and Medical Affairs departments in which several biologics for the treatment of diabetes and obesity were brought to commercialization.

“I am excited to join XyloCor to lead the clinical development team as we work to uncover the potential of our pipeline,” said Dr. Reinhardt. ”XyloCor is poised to be a leader in cardiovascular gene therapy, and I am proud to be part of our effort to bring new advances in the treatment of coronary artery disease.”

Dr. Reinhardt began his career in pharmaceuticals at Merck in the clinical pharmacology group where he contributed to the development and approval of EMEND®. Prior to entering the industry, he spent 11 years as a bench scientist and in academia, which included serving as Clinical Assistant Professor of Medicine in the Division of Endocrinology, Metabolism and Nutrition at Robert Wood Johnson Medical School of Rutgers University, and as a Senior Medical Staff Fellow at the National Institutes of Health (NIH).

He received both his medical degree and PhD from Louisiana State University and holds a BS in biochemistry from the University of California - Los Angeles. He completed his endocrinology fellowship at the NIH and his residency in internal medicine at George Washington Hospital in Washington, DC. Dr. Reinhardt has published more than 80 papers, abstracts, reviews and book chapters. He holds board certifications in both internal medicine and endocrinology.

About XyloCor

XyloCor Therapeutics is a biopharmaceutical company focused on the development of novel gene therapy for unmet needs in advanced coronary artery disease. In the United States, coronary artery disease is a leading cause of death and disability. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor also has a secondary product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. Co‑founded by Ronald Crystal, MD, and Todd Rosengart, MD, XyloCor has a licensing agreement with Weill Cornell Medicine for the worldwide rights to develop, manufacture and commercialize XC001. For more information, visit www.xylocor.com.

Media Contact

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

– Novel gene therapy for patients with treatment-resistant angina who have no remaining treatment options is ready to enter clinical development –

– Pharmaceutical industry veteran and former GSK executive Al Gianchetti leads company as CEO –

Philadelphia, PA, December 6, 2018 — XyloCor Therapeutics, a private clinical‑stage biopharmaceutical company focused on the development of gene therapy for unmet needs in cardiovascular disease, today announced the close of a $17 million Series A financing which was co‑led by Sofinnova Ventures and LSP (Life Sciences Partners). Proceeds from the financing will be used to advance the development of lead product candidate XC001, a cardiovascular angiogenic gene therapy, and expand the company’s business and operational structure. XyloCor is led by pharmaceutical industry veteran and former GlaxoSmithKline (GSK) executive, Al Gianchetti as Chief Executive Officer and has expanded its Board of Directors to include a number of leading industry experts.

“XyloCor was created to develop a proprietary gene therapy for patients with refractory angina who have no remaining treatment options. This significant raise enables the research and development for our lead investigational product XC001, which has the potential to provide new hope for patients who suffer from this chronic and debilitating disease,” said Al Gianchetti, President and Chief Executive Officer of XyloCor. “We are grateful for the support and confidence demonstrated by our investors who bring highly valued contributions to XyloCor beyond the financial resources. Sofinnova and LSP are leaders in helping companies bring innovative therapeutics to patients who need them, and both have extensive experience in cardiovascular disease.”

The U.S. Food and Drug Administration (FDA) recently granted Fast Track designation to the company’s lead investigational product XC001, for clinical studies as a one‑time treatment for the improvement of exercise tolerance in patients with chronic angina that is refractory to standard medical therapy and not amenable to conventional revascularization procedures including coronary artery bypass surgery, percutaneous coronary intervention and stents. An Investigational New Drug (IND) application for XC001 is open with the FDA and XyloCor intends to commence clinical trials in 2019. XyloCor also has a secondary product, XC002, in discovery stage being investigated for the regeneration of cardiac tissue in patients with cardiac damage from heart attacks.

Chronic angina pectoris or chest pain occurs when the heart muscle does not receive as much oxygen as it needs for the amount of work it is performing. This is usually due to coronary artery disease. Patients with chronic angina who become resistant to current standard of care, whether medical or surgical, are considered refractory and often become sedentary because of their symptoms, which in turn can exacerbate comorbidities causing further deterioration of their health status.

“Gene therapy is an exciting and newly emerging area of medicine. With a robust scientific foundation and an experienced team, XyloCor is poised to address serious unmet medical needs in cardiovascular disease,” said co‑founder and advisor, Ronald Crystal, MD, the Bruce Webster Professor of Internal Medicine and Chairman, Department of Genetic Medicine, Weill Cornell Medicine and Director of its Belfer Gene Therapy Core Facility.

“XC001 has been designed to stimulate the formation of new coronary blood vessels to serve areas of the heart that are not receiving adequate blood supply, which may allow patients to increase their daily activities and improve their quality of life,” said co‑founder and board member, Todd Rosengart, MD, Professor and DeBakey-Bard Chair of the Michael E. DeBakey Department of Surgery, Baylor College of Medicine.

XyloCor is being led by Al Gianchetti who has more than 25 years of drug development and commercialization experience. He has served in a number of executive-level management positions at GSK and leadership roles with small biotech and specialty pharma companies. Mr. Gianchetti has extensive experience in both commercial strategy/operations and R&D, including involvement in Phase 1 through 3 clinical programs and several product launches.

XyloCor Expands and Formalizes Board of Directors

In conjunction with the financing, XyloCor has expanded its Board of Directors. New board members include Fouad Azzam, PhD, MBA, General Partner, LSP and Alan Colowick, MD, MPH, Partner, Sofinnova. They will serve alongside existing Board members Glenn Batchelder, Executive Chairman; Todd Rosengart, MD, Founder and Advisor; and Al Gianchetti, XyloCor President and CEO. Ronald Crystal, MD, Founder and Advisor and John de Koning, PhD, Partner, LSP will participate as board observers.

About Sofinnova

Founded in 1974, Sofinnova specializes in clinical and late preclinical investments in biopharmaceutical products. Their goal is to actively partner with entrepreneurs across all stages of company development. The firm seeks to build world class companies that aspire to dramatically improve the current state of medical care and the lives of patients through bringing innovative products to market. For more information, visit www.sofinnova.com.

About LSP

LSP (Life Sciences Partners) is an independent European investment firm, providing financing for private and public life sciences companies. LSP’s mission is to connect investors to inventors, focusing on unmet medical needs. Since the late 1980s, the LSP team has invested in about 100 innovative enterprises, many of which have grown to become leaders of the global life sciences industry. With over € 2 billion ($2.3 billion) of investment capital raised to date and offices in Amsterdam, Munich and Boston, LSP is Europe’s leading life sciences investor. LSP also invests in public companies through its Euronext-listed LSP Life Sciences Fund (Bloomberg: LSP NA). XyloCor Therapeutics is the second investment of LSP’s latest flagship fund, LSP 6. For more information, please visit www.lspvc.com.

About XyloCor

XyloCor Therapeutics is a biopharmaceutical company focused on the development of novel gene therapy for unmet needs in advanced coronary artery disease. In the United States, coronary artery disease is a leading cause of death and disability. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor also has a secondary product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. Co‑founded by Ronald Crystal, MD, and Todd Rosengart, MD, XyloCor has a licensing agreement with Weill Cornell Medicine for the worldwide rights to develop, manufacture and commercialize XC001. For more information, visit www.xylocor.com.

Media Contact

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

click here to download the PDF

Newtown Square, PA, May 8, 2017 — XyloCor Therapeutics Inc., a privately held biotech company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead product candidate XC001 (AdVEGF‑All6A+), a cardiovascular angiogenic gene therapy. XC001 is a one‑time treatment being investigated for improving exercise tolerance in patients who have chronic angina that is refractory to standard medical therapy and not amenable to conventional revascularization procedures such as coronary artery bypass surgery and percutaneous coronary intervention and stents.

“Achieving Fast Track status validates the need for XC001, which has the potential to be a unique treatment for this serious condition with high unmet need — chronic, refractory angina,” said Al Gianchetti, President and Chief Executive Officer of XyloCor. “This designation is supported by strong scientific evidence for XC001 and clinical validation of this mechanism of action in refractory angina. This important designation is intended to contribute to an expedited development and regulatory review process, which can get the drug sooner to patients who can benefit from it.”

The FDA Fast Track designation is designed to facilitate the development and expedite the review of new drugs and vaccines intended to treat or prevent serious conditions and that demonstrate the potential to address an unmet medical need.

XC001 is a novel gene therapy that promotes angiogenesis, the formation of new vessels that can provide arterial blood flow to myocardial regions with inadequate blood supply. Enhancing myocardial blood flow with therapeutic angiogenesis is intended to relieve myocardial ischemia, improve regional and global left ventricular performance, alleviate angina symptoms and disability and potentially improve prognosis.

“There are many patients in the United States with refractory angina and there are no available treatment options,” said Magnus Ohman, Professor of Medicine, The Kent and Siri Rawson Director, Duke Program for Advanced Coronary Disease, Duke University School of Medicine. “These patients have significant limitations in terms of their daily activities because of the chest pain associated with their ischemic disease and XC001 could be an important new option for them.”

An IND for XC001 is open with the FDA and XyloCor intends to commence clinical trials upon funding.

About XyloCor

XyloCor Therapeutics is a private biopharmaceutical company developing novel gene therapy for people with unmet medical need from advanced coronary artery disease. XyloCor is focused on developing its lead product, XC001, for patients with refractory angina with no treatment options and its secondary product, XC002, for patients with cardiac tissue damage from heart attacks. XyloCor was founded by Dr. Ronald Crystal and Dr. Todd Rosengart, who both sit on XyloCor’s advisory board. Dr. Crystal is the Bruce Webster Professor and Chairman, Department of Genetic Medicine, Weill Cornell Medicine and Director of the Belfer Gene Therapy Core Facility. Dr. Rosengart is Professor and Chairman, DeBakey Bard Chair of Surgery, Michael E. DeBakey Department of Surgery, Baylor College of Medicine. XyloCor has a licensing agreement with Cornell University granting the company worldwide rights to develop, manufacture and commercialize XC001. With a strong scientific foundation, compelling preclinical and clinical evidence and an experienced team, XyloCor is poised for success and to help patients lead better, healthier lives. For more information, visit www.xylocor.com.

Company Contact: Al Gianchetti, President and Chief Executive Officer, info@xylocor.com

Media Contact: Mike Beyer, Sam Brown Inc., mikebeyer@sambrown.com, 312-961-2502