XyloCor Therapeutics Positive EXACT Phase 2 Data for Lead Candidate XC001 Simultaneously Presented at Society for Cardiovascular Angiography & Interventions (SCAI) 2024 Scientific Sessions and Published in Circulation: Cardiovascular Interventions
May 02 2024 - 1:27PM
Business Wire
– 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
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
https://scai.org/gene-therapy-treatment-increasing-bodys-signal-new-blood-vessel-growth-shows-promise.
The Circulation: Cardiovascular Interventions full article
titled “Angiogenic Gene Therapy for Refractory Angina: Results of
the EXACT Phase 2 Trial” is available at
https://www.ahajournals.org/doi/epdf/10.1161/CIRCINTERVENTIONS.124.014054.
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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240501532555/en/
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