A Phase 1/2 Trial of Direct Administration of AdVEGF-All6A+, a Replication Deficient Adenovirus Vector Expressing a cDNA/Genomic Hybrid of Human Vascular Endothelial Growth Factor, to the Ischemic Myocardium of Subjects with Angina Pectoris Secondary to Coronary Artery Disease that is Refractory to Drug Therapy and Unsuitable for Revascularization

Start Date20 Jan 2022

Sponsor / Collaborator

Xylocor Therapeutics, Inc.

NCT01757223 / WithdrawnPhase 1/2IIT

Phase I/II Study, Administration of AdVEGF-All6A+, a Replication Deficient Adenovirus Vector Expressing a cDNA/Genomic Hybrid of Human Vascular Endothelial Growth Factor to the Ischemic Myocardium...

The proposed Phase I/II clinical trial will be used to determine the safety and toxicity of direct administration of the vector AdVEGF-All6A+ to the ischemic myocardium and to generate preliminary evidence regarding whether direct administration of AdVEGF-All6A+ to the ischemic myocardium will induce growth of collateral blood vessels and improve cardiac function. This is a three-part, multinational/multi-center, placebo controlled study.

Start Date01 Dec 2020

Sponsor / Collaborator

Weill Medical College of Cornell University

NCT04125732 / CompletedPhase 1/2

A Phase 1/2 Trial of Direct Administration of AdVEGF-All6A+, a Replication Deficient Adenovirus Vector Expressing a cDNA/Genomic Hybrid of Human VEGF, to the Ischemic Myocardium of Subjects With Angina Pectoris

The primary purpose of this trial is to determine the safety of XC001 (AdVEGFXC1) in patients who suffer from angina caused by coronary artery disease and have no other treatment options. Subjects in this study will receive one of four intramyocardial doses of XC001 that expresses human vascular endothelial growth factor (VEGF) which induces therapeutic angiogenesis (revascularization).

Start Date24 Jan 2020

Sponsor / Collaborator

Xylocor Therapeutics, Inc.

100 Clinical Results associated with Encoberminogene Rezmadenovec

100 Translational Medicine associated with Encoberminogene Rezmadenovec

100 Patents (Medical) associated with Encoberminogene Rezmadenovec

Literatures (Medical) associated with Encoberminogene Rezmadenovec

01 Jan 2024·Gene therapy

Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases

Article

Author: Dittrich, Howard C ; Gianchetti, Albert ; Thorne, Barb ; Stewart, Duncan J ; Manza, Linda L ; Reinhardt, Rickey R ; Byrnes, Dawn

Adenovirus-mediated gene therapy holds promise for the treatment of cardiovascular diseases such as refractory angina. However, potential concerns around immunogenicity and vector dissemination from the target injected tissue require evaluation. This study was undertaken to evaluate the safety and biodistribution of XC001, a replication-deficient adenovirus serotype 5 vector expressing multiple isoforms of human vascular endothelial growth factor (VEGF), following direct administration into normal rat myocardium. Animals received the buffer formulation or increasing doses of XC001 (1 × 10⁷, 2.5 × 10⁸ or 2.5 × 10⁹ viral particles). Based on in-life parameters (general health, body weights, clinical pathology, serum cardiac troponin I, plasma VEGF, and gross necropsy), there were no findings of clinical concern. On Day 8, intramyocardial administration of XC001 was associated with dose-related, left ventricular myocardial inflammation at injection sites, resolving by Day 30. XC001 DNA was not detected in blood at any time but was present at Day 8 around the site of injection and to a much lesser extent in the spleen, liver, and lungs, persisting at low levels in the heart and spleen until at least Day 91. These findings demonstrate that intramyocardial injection of XC001 is supported for use in human studies.

01 Aug 2023·Circulation. Cardiovascular interventions

EXACT Trial: Results of the Phase 1 Dose-Escalation Study.

Article

Author: Sun, Benjamin C ; Henry, Timothy D ; Dittrich, Howard C ; Latter, David A ; Crystal, Ronald G ; Tarka, Elizabeth A ; Byrnes, Dawn ; Ohman, E Magnus ; Povsic, Thomas J ; Arnaoutakis, George J ; Peterson, Mark W ; Williams, Adam R ; Rosengart, Todd K ; Traverse, Jay H ; Mokadam, Nahush A ; DiCarli, Marcelo F ; Answini, Geoffrey A ; Boudoulas, Konstantinos D ; Anderson, R David ; Pepine, Carl J

BACKGROUND:

New therapies are needed for patients with refractory angina. Encoberminogene rezmadenovec (XC001), a novel adenoviral-5 vector coding for all 3 major isoforms of VEGF (vascular endothelial growth factor), demonstrated enhanced local angiogenesis in preclinical models; however, the maximal tolerated dose and safety of direct epicardial administration remain unknown.

METHODS:

In the phase 1 portion of this multicenter, open-label, single-arm, dose-escalation study, patients with refractory angina received increasing doses of encoberminogene rezmadenovec (1×10⁹, 1×10¹⁰, 4×10¹⁰, and 1×10¹¹ viral particles) to evaluate its safety, tolerability, and preliminary efficacy. Patients had class II to IV angina on maximally tolerated medical therapy, demonstrable ischemia on stress testing, and were angina-limited on exercise treadmill testing. Patients underwent minithoracotomy with epicardial delivery of 15 0.1-mL injections of encoberminogene rezmadenovec. The primary outcome was safety via adverse event monitoring over 6 months. Efficacy assessments included difference from baseline to months 3, 6 (primary), and 12 in total exercise duration, myocardial perfusion deficit using positron emission tomography, angina class, angina frequency, and quality of life.

RESULTS:

From June 2, 2020 to June 25, 2021, 12 patients were enrolled into 4 dosing cohorts with 1×10¹¹ viral particle as the highest planned dose. Seventeen serious adverse events were reported in 7 patients; none were related to study drug. Six serious adverse events in 4 patients were related to the thoracotomy, 3 non-serious adverse events were possibly related to study drug. The 2 lowest doses did not demonstrate improvements in total exercise duration, myocardial perfusion deficit, or angina frequency; however, there appeared to be improvements in all parameters with the 2 higher doses.

CONCLUSIONS:

Epicardial delivery of encoberminogene rezmadenovec via minithoracotomy is feasible, and up to 1×10¹¹ viral particle appears well tolerated. A dose response was observed across 4 dosing cohorts in total exercise duration, myocardial perfusion deficit, and angina class. The highest dose (1×10¹¹ viral particle) was carried forward into phase 2.

REGISTRATION:

URL: https://www.

CLINICALTRIALS:

gov; Unique identifier: NCT04125732.

01 Nov 2021·American heart journalQ2 · MEDICINE

Epicardial delivery of XC001 gene therapy for refractory angina coronary treatment (The EXACT Trial): Rationale, design, and clinical considerations

Q2 · MEDICINE

Article

Author: Henry, Timothy D ; Pepine, Carl J ; Answini, Geoffrey A ; Ohman, E Magnus ; Crystal, Ronald G ; Mokadam, Nahush A ; Dittrich, Howard C ; Rosengart, Todd K ; Traverse, Jay H ; Povsic, Thomas J ; Reinhardt, Rickey R

BACKGROUND:

Patients with refractory angina (RA) have poor quality of life and new therapies are needed. XC001 is a novel adenoviral vector expressing multiple isoforms of vascular endothelial growth factor (VEGF) promoting an enhanced local angiogenic effect.

METHODS:

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) trial is a 6-month (with 6-month extension) phase 1/2, first-in-human, multicenter, open-label, single-arm, dose-escalation study to evaluate the safety, tolerability, and preliminary efficacy of XC001 in patients with RA. The trial will enroll 33 patients in an initial (n = 12) ascending dose-escalation phase (1 × 10⁹, 1 × 10¹⁰, 4 × 10¹⁰, and 1 × 10¹¹ viral particles), followed by phase 2 (n = 21) assessing the highest tolerated dose. Patients must have stable Canadian Cardiovascular Society (CCS) class II-IV angina on maximally tolerated medical therapy without options for conventional revascularization, demonstrable ischemia on stress testing, and angina limiting exercise tolerance. XC001 will be delivered directly to ischemic myocardium via surgical transthoracic epicardial access. The primary outcome is safety via adverse event monitoring through 6 months. Efficacy assessments include difference from baseline to month 6 in time to 1 mm of ST segment depression, time to angina, and total exercise duration; myocardial blood flow at rest, and stress and coronary flow reserve by positron emission tomography; quality of life; CCS functional class; and angina frequency.

CONCLUSIONS:

The EXACT trial will determine whether direct intramyocardial administration of XC001 in patients with RA is safe and evaluate its effect on exercise tolerance, myocardial perfusion, angina and physical activity, informing future clinical investigation.

CLINICAL TRIAL REGISTRATION:

NCT04125732.

News (Medical) associated with Encoberminogene Rezmadenovec

02 May 2024

·www.biospace.com

XyloCor Therapeutics Positive EXACT Phase 2 Data for Lead Candidate XC001 Simultaneously Presented at Society for Cardiovascular Angiography & Interventions (SCAI) 2024 Scientific Sessions

– 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.--(BUSINESS WIRE)-- 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 at . The Circulation: Cardiovascular Interventions full article titled “Angiogenic Gene Therapy for Refractory Angina: Results of the EXACT Phase 2 Trial” is available at . 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 .

Clinical ResultGene TherapyPhase 2License out/in

25 Aug 2023

·www.biospace.com

XyloCor Therapeutics Presents Phase 2 Data Highlighting Safety and Efficacy of XC001 at the European Society of Cardiology (ESC) Congress 2023

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 pro > 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.--(BUSINESS WIRE)-- 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 pro 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 . View source version on businesswire.com: Contacts Corporate and Investor Relations: A. Brian Davis, XyloCor Therapeutics, Inc. brian.davis@xylocor.com 610-541-2056 Media: Mike Beyer Sam Brown Inc. Healthcare Communications mikebeyer@sambrown.com 312-961-2502 Source: XyloCor Therapeutics, Inc. View this news release online at:

Clinical ResultGene TherapyLicense out/inPhase 2

03 Aug 2023

·www.biospace.com

Results from XyloCor Therapeutics’ Phase 1 Portion of EXACT Trial of XC001 for Cardiovascular Disease Published in Circulation: Cardiovascular Interventions

- 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.--(BUSINESS WIRE)-- 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 1×1011 viral particles for future clinical research of XC001. The Circulation: Cardiovascular Interventions full article is available at 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 .

Phase 1Gene TherapyLicense out/inClinical Result

100 Deals associated with Encoberminogene Rezmadenovec

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Indication	Highest Phase	Country/Location	Organization	Date
Angina Pectoris	Phase 2	US	-	21 Jul 2021
Coronary Artery Disease	Phase 2	US	Xylocor Therapeutics, Inc.	24 Jan 2020
Ischemia	Phase 2	US	Xylocor Therapeutics, Inc.	24 Jan 2020
Refractory angina	Phase 2	US	Xylocor Therapeutics, Inc.	24 Jan 2020

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EXACT (ESC2023)	Phase 2	Refractory angina	-	XC001 gene therapy	flceyquejt(goxgkikoxo) = htonqncpsc nykccnixtk (pfclssustl, 31.3 - 139.7)	Positive	25 Aug 2023
phase 1/2 clinical trial (ASGCT2021)	Phase 3	Refractory angina VEGF	-	XC001	rkedcwqhws(eduxvchcek) = vector-derived VEGF secreted by the cardiomyocytes was capable of stimulating a VEGF-responsive cell line similarly to control recombinant VEGF protein ffkaiuskan (vcraxbrqic ) View more	-	27 Apr 2021
phase 1/2 clinical trial (ASGCT2021)	Phase 3	Refractory angina VEGF	-	XC001		-	27 Apr 2021

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