Vericel Receives FDA Fast Track Designation for Ixmyelocel-T, an Investigational Product for the Treatment of Patients with A...
February 21 2017 - 7:30AM
Vericel Corporation (NASDAQ:VCEL), a leading developer of
autologous expanded cell therapies for the treatment of patients
with serious diseases and conditions, today announced that the U.S.
Food and Drug Administration (FDA) has designated the investigation
of ixmyelocel‑T for reduction in the risk of death and
cardiovascular hospitalization in patients with chronic advanced
heart failure due to ischemic dilated cardiomyopathy as a Fast
Track Development Program.
“Receiving Fast Track designation highlights both the unmet
medical need for improved therapies to treat advanced heart failure
due to dilated cardiomyopathy and the significance of the results
from the ixmyelocel-T Phase 2b ixCELL-DCM clinical study” said Nick
Colangelo, president and CEO of Vericel. "We believe that achieving
important regulatory milestones such as Fast Track designation
enhances the value of ixmyelocel-T and our efforts to partner the
further development of this program."
The Fast Track program is an expedited drug development and
review program for new drugs or biologics intended to treat serious
or life-threatening conditions that demonstrate the potential to
address an unmet medical need. The purpose of the program is
to get important new drugs to the patient earlier. For more
information on Fast Track, visit the FDA website
(http://www.fda.gov/forpatients/approvals/fast/ucm405399.htm).
About Advanced Heart Failure Dilated
cardiomyopathy (DCM), a progressive disease of the heart, is a
leading cause of heart failure and heart transplantation. DCM
is characterized by weakening of the heart muscle and enlargement
of the heart chambers, leading to systolic abnormalities
(difficulty of the left ventricle to pump blood). Heart
enlargement and poor function generally lead to progressive heart
failure with further decline in the ability of the heart to pump
blood efficiently throughout the body. There is no cure for
heart failure and there are limited treatment options in the
advanced, refractory stage of the disease. Pharmacological
interventions are typically introduced in earlier stages of heart
failure and maximized as the condition progresses, with more
invasive and aggressive interventions reserved for patients in
later stages. By the time a patient progresses to the
advanced stage of heart failure, they are being treated with
multiple drugs with limited success for the treatment of persistent
and severe symptoms, may have an implantable
cardioverter‑defibrillator (ICD) or cardiac resynchronization
therapy, and have few remaining treatment options (Yancy 2013).
About Ixmyelocel-TIxmyelocel-T is an
investigational autologous expanded multicellular therapy
manufactured from the patient's own bone marrow using Vericel's
proprietary, highly automated, fully closed cell-processing system.
This process selectively expands the population of mesenchymal
stromal cells and alternatively activated macrophages, which are
responsible for production of anti-inflammatory and pro-angiogenic
factors known to be important for repair of damaged tissue.
Ixmyelocel-T has been designated as an orphan drug by the U.S. Food
and Drug Administration for use in the treatment of DCM.
About the ixCELL-DCM Trial The ixCELL-DCM
clinical trial was a multicenter, randomized, double-blind,
placebo-controlled Phase 2b study designed to assess the efficacy,
safety and tolerability of ixmyelocel-T compared to placebo when
administered via transendocardial catheter-based injections to
participants with end-stage heart failure due to ischemic DCM, who
have no reasonable revascularization options (either surgical or
percutaneous interventional) likely to provide clinical benefit.
All participants were on maximized pharmacological heart failure
treatment and had an automatic implantable cardiac defibrillator or
cardiac resynchronization therapy. The primary endpoint of the
ixCELL-DCM clinical trial is the number of all-cause deaths,
cardiovascular hospital admissions, and unplanned outpatient and
emergency department visits to treat acute decompensated heart
failure over the 12 months following administration of ixmyelocel-T
compared to placebo. Primary endpoint results were presented in a
late-breaking clinical trial session at the American College of
Cardiology's (ACC) 65th Annual Scientific Session. The ixCELL-DCM
trial met its primary endpoint with a 37% reduction in the
composite endpoint, primarily driven by a reduction in all cause
deaths and cardiovascular hospitalizations. In addition, this
study showed internal consistency (ie, repeatability) in observable
or "hard" efficacy endpoints of survival and cardiovascular
hospitalizations (total number and time to events), reduction in
ventricular arrhythmias, and safety results including major cardiac
adverse events (MACE), serious adverse events (SAEs), deaths, and
intravenous pharmacological treatment for heart failure.
Because the trial met the primary endpoint, patients who
received placebo or were randomized to ixmyelocel‑T in the
double-blind portion of the trial but did not receive ixmyelocel-T,
have been offered the option to receive treatment with
ixmyelocel‑T.
About Vericel Corporation Vericel develops,
manufactures, and markets autologous expanded cell therapies for
the treatment of patients with serious diseases and
conditions. The company markets three cell therapy products
in the United States. Vericel is marketing MACI®
(autologous cultured chondrocytes on porcine collagen membrane), an
autologous cellularized scaffold product indicated for the repair
of symptomatic, single or multiple full-thickness cartilage defects
of the knee with or without bone involvement in adults, which has
recently been approved by the FDA. Carticel® (autologous
cultured chondrocytes) is an autologous chondrocyte implant for the
treatment of cartilage defects in the knee in patients who have had
an inadequate response to a prior arthroscopic or other surgical
repair procedure. Epicel® (cultured epidermal autografts) is
a permanent skin replacement for the treatment of patients with
deep dermal or full thickness burns greater than or equal to 30% of
total body surface area. Vericel is also developing
ixmyelocel‑T, an autologous multicellular therapy intended to treat
advanced heart failure due to ischemic dilated cardiomyopathy
(DCM). For more information, please visit the company's
website at www.vcel.com.
Epicel®, Carticel®, and MACI® are registered trademarks of
Vericel Corporation. © 2017 Vericel Corporation. All
rights reserved.
This document contains forward-looking statements, including,
without limitation, statements concerning anticipated progress,
objectives and expectations regarding the commercial potential of
ixmyelocel-T and our other products, clinical activity timing, and
objectives and expectations regarding our company described herein,
all of which involve certain risks and uncertainties. These
statements are often, but are not always, made through the use of
words or phrases such as "anticipates," "intends," "estimates,"
"plans," "expects," "we believe," "we intend," and similar words or
phrases, or future or conditional verbs such as "will," "would,"
"should," "potential," "can continue," "could," "may," or similar
expressions. Actual results may differ significantly from the
expectations contained in the forward-looking statements. Among the
factors that may result in differences are the inherent
uncertainties associated with competitive developments, clinical
trial and product development activities, regulatory approval
requirements, estimating the commercial potential of our products
and product candidates, market demand for our products, product
performance and our ability to supply or meet customer demand for
our products. These and other significant factors are discussed in
greater detail in Vericel's Annual Report on Form 10-K for the year
ended December 31, 2015, filed with the Securities and Exchange
Commission ("SEC") on March 14, 2016, Quarterly Reports on Form
10-Q and other filings with the SEC. These forward-looking
statements reflect management's current views and Vericel does not
undertake to update any of these forward-looking statements to
reflect a change in its views or events or circumstances that occur
after the date of this release except as required by law.
References
Yancy CW, Jessup M, Bozkurt B, Butler J, Casey DE Jr, Drazner
MH, et al. 2013 ACCF/AHA guideline for the management of heart
failure: A report of the American College of Cardiology
Foundation/American Heart Association task force on practice
guidelines. Circulation. 2013 Oct 15;128:e240‑e327.
CONTACT:
Chad Rubin
The Trout Group crubin@troutgroup.com
(646) 378-2947
or
Lee Stern
The Trout Group lstern@troutgroup.com
(646) 378-2922
Vericel (NASDAQ:VCEL)
Historical Stock Chart
From Mar 2024 to Apr 2024
Vericel (NASDAQ:VCEL)
Historical Stock Chart
From Apr 2023 to Apr 2024