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 May 2024·Circulation-Cardiovascular Interventions

Angiogenic Gene Therapy for Refractory Angina: Results of the EXACT Phase 2 Trial

Article

Author: Latter, David A. ; Dittrich, Howard C. ; Rosengart, Todd K. ; Peterson, Mark W. ; Burke, Christopher R. ; Bakaeen, Faisal G. ; Chaitman, Bernard R. ; Povsic, Thomas J. ; Sun, Benjamin C. ; Traverse, Jay H. ; Ohman, E. Magnus ; Koch, Gary G. ; Pepine, Carl J. ; Nakamura, Kenta ; DiCarli, Marcelo F. ; Kowalewski, Elaine ; Answini, Geoffrey A. ; Byrnes, Dawn G. ; Henry, Timothy D. ; Williams, Adam R. ; Crystal, Ronald G. ; Mokadam, Nahush A.

BACKGROUND::

XC001 is a novel adenoviral-5 vector designed to express multiple isoforms of VEGF (vascular endothelial growth factor) and more safely and potently induce angiogenesis. The EXACT trial (Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment) assessed the safety and preliminary efficacy of XC001 in patients with no option refractory angina.

METHODS::

In this single-arm, multicenter, open-label trial, 32 patients with no option refractory angina received a single treatment of XC001 (1×10 11 viral particles) via transepicardial delivery.

RESULTS::

There were no severe adverse events attributed to the study drug. Twenty expected severe adverse events in 13 patients were related to the surgical procedure. Total exercise duration increased from a mean±SD of 359.9±105.55 seconds at baseline to 448.2±168.45 (3 months), 449.2±175.9 (6 months), and 477.6±174.7 (12 months; +88.3 [95% CI, 37.1–139.5], +84.5 [95% CI, 34.1–134.9], and +115.5 [95% CI, 59.1–171.9]). Total myocardial perfusion deficit on positron emission tomography imaging decreased by 10.2% (95% CI, −3.1% to 23.5%), 14.3% (95% CI, 2.8%–25.7%), and 10.2% (95% CI, −0.8% to −21.2%). Angina frequency decreased from a mean±SD 12.2±12.5 episodes to 5.2±7.2 (3 months), 5.1±7.8 (6 months), and 2.7±4.8 (12 months), with an average decrease of 7.7 (95% CI, 4.1–11.3), 6.6 (95% CI, 3.5–9.7), and 8.8 (4.6–13.0) episodes at 3, 6, and 12 months. Angina class improved in 81% of participants at 6 months.

CONCLUSIONS::

XC001 administered via transepicardial delivery is safe and generally well tolerated. Exploratory improvements in total exercise duration, ischemic burden, and subjective measures support a biologic effect sustained to 12 months, warranting further investigation.

REGISTRATION::

URL: https://www.clinicaltrials.gov ; Unique identifier: NCT04125732.

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 ; Anderson, R David ; Boudoulas, Konstantinos D ; 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 (1×10 9 , 1×10 10 , 4×10 10 , and 1×10 11 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 1×10 11 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 1×10 11 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 (1×10 11 viral particle) was carried forward into phase 2.

REGISTRATION::

URL: https://www.clinicaltrials.gov ; Unique identifier: NCT04125732.

News (Medical) associated with Encoberminogene Rezmadenovec

10 Jul 2024

·www.prnewswire.com

XyloCor and SmartCella enter into license agreement for use of the Extroducer to administer novel gene therapy XC001 to the heart

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. and TULLINGE, Sweden, July 10, 2024 /PRNewswire/ -- 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 . 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 . SmartCella Contact: Niklas Prager, CEO, +46 768 117744;[email protected] Nina Nornholm, Head of Corporate Communication +46 708 550356, [email protected] Corporate and Investor Relations: A. Brian Davis, XyloCor Therapeutics, Inc. [email protected] 610-541-2056 Media Contact: Mike Beyer Sam Brown Inc. Healthcare Communications, [email protected] 312-961-2502 This information was brought to you by Cision . The following files are available for download:

License out/inPhase 2Gene TherapyClinical Result

10 Jul 2024

·www.biospace.com

XyloCor Therapeutics and SmartCella Enter into License Agreement for Use of the Extroducer Infusion Catheter System to Administer Novel Gene Therapy XC001 to the Heart

- 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--(BUSINESS WIRE)-- 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 . 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 .

License out/inGene TherapyPhase 2Clinical Result

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

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Indication	Highest Phase	Country/Location	Organization	Date
Angina Pectoris	Phase 2	US	-	21 Jul 2021
Coronary Artery Disease	Phase 2	-	Weill Medical College of Cornell University	01 Dec 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	voyjijctzy(vwyjifpdns) = wowpywsrql vfnnbnapci (cixjbiruty, 31.3 - 139.7)	Positive	25 Aug 2023
phase 1/2 clinical trial (ASGCT2021)	Phase 3	Refractory angina VEGF	-	XC001	cgdxqtubky(leslwaivvy) = vector-derived VEGF secreted by the cardiomyocytes was capable of stimulating a VEGF-responsive cell line similarly to control recombinant VEGF protein kdgzmtwpwu (dpijgarffz ) View more	-	27 Apr 2021
phase 1/2 clinical trial (ASGCT2021)	Phase 3	Refractory angina VEGF	-	XC001 (Human Cardiomyocyte Cell Lines)		-	27 Apr 2021

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