Flexion Therapeutics, Inc. (Nasdaq:FLXN) today announced clearance
of the company's Investigational New Drug (IND) application for
FX201 in knee osteoarthritis (OA), a painful, chronic and
progressive disease that affects more than 15 million adults living
in the United States. Flexion has initiated a Phase 1 multicenter,
open-label, single ascending dose study and expects to treat the
first patient by the end of this year.
"Disease progression in osteoarthritis involves multiple tissues
in the joint, and in preclinical models of osteoarthritis,
intra-articular injection of FX201 has demonstrated improvements in
bone, cartilage and synovium as well as symptomatic benefit,” said
Neil Bodick, MD, PhD, Chief Scientific Officer at Flexion. “We
believe a single injection of FX201 will persistently provide
therapeutic concentrations of IL-1Ra, a potent anti-inflammatory
protein, with the potential to both provide long-term pain relief
and modify disease progression.”
FX201 is a helper-dependent non-integrating adenovirus
containing the human interleukin-1 receptor antagonist (IL-1Ra)
gene under the control of an inflammation sensitive promoter.
Preclinical data show that gene expression persists for at least
one year, and IL-1Ra expression increases in response to
inflammation. Nonclinical safety and efficacy data submitted in the
IND application demonstrated that a single administration of FX201
was well-tolerated, had no significant biodistribution outside the
target tissues, and pharmacology studies with the rat, mouse, and
horse orthologues showed symptomatic improvement and delay in
disease progression. Results showing dose-dependent decreases in
the severity of cartilage and bone lesions following anterior
cruciate ligament transection in rats will be presented at the
American College of Rheumatology (ACR) annual meeting on Tuesday,
November 12th at 9:00 a.m. ET (Abstract: 1974).
The Phase 1, multicenter, open-label, dose-escalation trial will
evaluate the safety and tolerability of FX201 in male and female
patients, 30-80 years of age, with painful OA of the knee. The
study is expected to enroll up to 24 patients across six sites in
the U.S.
“We expect this study to provide data characterizing dosing,
safety, and tolerability as well as initial insight into clinical
activity including effects on pain, function, and structural
progression in OA,” said Javad Parvizi, MS, MD, FRC, orthopedic
surgeon at the Rothman Institute and principal investigator for the
study. “If successful, FX201 could be transformative in the
treatment of knee OA.”
Prior to initiating the FX201 clinical program, Flexion formed a
Scientific Advisory Board (SAB) of world-renowned gene therapy
experts to provide on-going technical and strategic guidance as the
company advances the clinical development of FX201. The
appointments to Flexion's Gene Therapy SAB include:
- Robert Kotin, PhD is the Scientific founder of Generation Bio.
Previously he was the Vice President of production at Voyager
Therapeutics. He is currently on the faculty at University of
Massachusetts Medical School.
- Philip Reilly, MD, JD is a physician geneticist who helped
found Bluebird bio and Voyager Therapeutics, two gene therapy
companies that are publicly traded.
- Gregory Robinson, PhD is currently Chief Scientific Officer at
Akouos. Previously, he served as the Chief Scientific Officer at
Nightstar Therapeutics and Agilis Biotherapeutics.
- J. Fraser Wright, PhD is a co-founder of Spark Therapeutics,
Inc. where he served as Chief Technology Officer. He is currently
on the faculty at Stanford University.
Dr. Wright commented, “In the development of a treatment for a
prevalent, non-monogenic musculoskeletal disease, FX201 has the
potential to open a new domain for gene therapies.”
As part of Flexion’s ongoing commitment to expand scientific
understanding of OA, Flexion co-sponsored a study to evaluate the
potential of machine learning to measure and predict disease
progression and its effects on bone shape. At ACR, Dr. Bodick and
co-authors will present the results from the study which used
machine learning to evaluate magnetic resonance imaging (MRI) data
from the Osteoarthritis Initiative to develop a three-dimensional
OA bone shape model of disease progression. The study looked at
47,858 knee MRIs (9,433 knees; 5,031 without OA), taken at various
time points over an eight year period, with the aim of establishing
a relationship between structure and clinical outcomes. The
findings will be presented on Tuesday, November 12th at 9:00 am ET
(Abstract: 2196).
About FX201FX201 is a clinical stage, locally
administered, gene therapy product candidate designed to stimulate
the production of an anti-inflammatory protein, interleukin-1
receptor antagonist (IL-1Ra), whenever inflammation is present
within the joint. Inflammation is a known cause of pain, and
chronic inflammation is thought to play a major role in the
progression of OA. By persistently suppressing inflammation,
Flexion believes FX201 holds the potential to both reduce OA pain
and modify the disease.
About Osteoarthritis (OA) of the Knee OA, also
known as degenerative joint disease, affects more than 30 million
adults living in the U.S. and accounts for more than $185
billion in annual expenditures. In 2016, more than 15 million
Americans were diagnosed with OA of the knee and the average
age of physician-diagnosed knee OA has fallen by 16
years, from 72 in the 1990s to 56 in the 2010s. The prevalence of
OA is expected to continue to increase as a result of aging,
obesity and sports injuries. Each year, more than 15 million
Americans are treated for OA-related knee pain.
About Flexion Therapeutics Flexion
Therapeutics (Nasdaq:FLXN) is a biopharmaceutical company
focused on the development and commercialization of novel, local
therapies for the treatment of patients with musculoskeletal
conditions, beginning with osteoarthritis, a common form of
arthritis. The company's core values are focus, ingenuity,
tenacity, transparency and fun. For the past three years,
Flexion has been named one of the Best Places to Work by
the Boston Business Journal, and Flexion was also recognized
as a Top Place to Work
in Massachusetts by The Boston Globe in 2017
and 2018. Visit flexiontherapeutics.com.
Forward-Looking Statements Statements in this
press release regarding matters that are not historical facts,
including, but not limited to, statements relating to the future of
Flexion; our plans to develop and commercialize FX201, including
the expected timing of the Phase 1 clinical trial and data
therefrom; the potential therapeutic and other benefits of FX201;
and the expected future prevalence of OA, are forward-looking
statements. These forward-looking statements are based on
management's expectations and assumptions as of the date of this
press release and are subject to numerous risks and uncertainties,
which could cause actual results to differ materially from those
expressed or implied by such statements. These risks and
uncertainties include, without limitation, risks associated with
developing FX201; the fact that the results of prior preclinical
studies may not predict results of subsequent preclinical or
clinical studies; our reliance on third parties to manufacture
FX201; the risk that we may not be able to maintain and enforce our
intellectual property, including licensed intellectual property
related to FX201; the risk the license agreement with Baylor could
be terminated early if we do not comply with our obligations;
competition from alternative therapies; regulatory developments and
safety issues, including difficulties in obtaining and maintaining
regulatory approvals to conduct clinical trials and market FX201;
risks related to key employees, markets, economic conditions,
health care reform, prices and reimbursement rates; and other risks
and uncertainties described in our filings with the Securities
and Exchange Commission (SEC), including under the heading
"Risk Factors" in our Quarterly Report on Form 10-Q for the quarter
ended June 30, 2019. The forward-looking statements in this
press release speak only as of the date of this press release, and
we undertake no obligation to update or revise any of the
statements. We caution investors not to place considerable reliance
on the forward-looking statements contained in this press
release.
Contact:
Scott Young Vice President, Corporate Communications &
Investor Relations Flexion Therapeutics, Inc. T: 781-305-7194
syoung@flexiontherapeutics.com
Julie Downs Senior Manager, Corporate Communications &
Investor Relations Flexion Therapeutics, Inc. T: 781-305-7137
jdowns@flexiontherapeutics.com
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