CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company (“Catabasis”), and Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage developer of innovative RNA-targeted therapeutics (“Sarepta”), today announced a joint research collaboration to explore a combination drug treatment approach for Duchenne muscular dystrophy (DMD). The two companies will contribute their respective expertise to study an exon skipping treatment developed by Sarepta, together with an oral NF-kB inhibition treatment developed by Catabasis in a mouse model of DMD.
“We are excited to work with Sarepta on this joint research collaboration, which to our knowledge is the first time two companies are testing a combination of investigational therapies to treat Duchenne. Although we believe edasalonexent (CAT-1004) has the potential to be a disease-modifying monotherapy, we think there is benefit to exploring innovative ways to make the most meaningful difference in this devastating disease”, said Jill C. Milne, Ph.D., chief executive officer of Catabasis. “In addition to our continued development of edasalonexent, we are pleased to take the first step via this collaboration to determine if edasalonexent may be complementary to an exon-skipping treatment strategy in the treatment of DMD using a preclinical model.”
“We recognize the extreme unmet medical need in DMD and are committed to determining the best treatment strategies for patients affected by Duchenne,” said Edward Kaye, M.D., Sarepta’s chief executive officer. “We believe exon skipping has the potential to target the underlying genetic cause of the disease by restoring the mRNA reading frame to produce dystrophin in skeletal muscle. We are pleased to initiate activities with Catabasis to evaluate a potential combination treatment approach of exon-skipping and NF-kB inhibition in DMD.”
NF-kB inhibition and exon-skipping represent two novel investigational treatment strategies in Duchenne, each with the potential for disease-modifying effects when used as monotherapy. The objective of the joint research is to study the safety and efficacy of combining these two treatment strategies using a mouse model of DMD, including evaluating the potential for additional or synergistic benefits.
About Catabasis
At Catabasis Pharmaceuticals, our mission is
to bring hope and life-changing therapies to patients and their
families. Our SMART (Safely Metabolized And Rationally Targeted) linker
drug discovery platform enables us to engineer molecules that
simultaneously modulate multiple targets in a disease. We are applying
our SMART linker platform to build an internal pipeline of product
candidates for rare diseases and plan to pursue partnerships to develop
additional product candidates. For more information on the Company's
drug discovery platform and pipeline of drug candidates, please visit www.catabasis.com.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is
an oral small molecule that has the potential to be a disease-modifying
therapy for all patients affected by Duchenne muscular dystrophy (DMD or
Duchenne), regardless of the underlying mutation. Edasalonexent inhibits
NF-kB, a protein that is activated in Duchenne and drives inflammation
and fibrosis, muscle degeneration and suppresses muscle regeneration. In
animal models of DMD, edasalonexent inhibited NF-kB, reduced muscle
degeneration and improved muscle regeneration and function, and
beneficial effects were observed in skeletal, diaphragm and cardiac
muscle. The FDA has granted orphan drug, fast track and rare pediatric
disease designations and the European Commission has granted orphan
medicinal product designation to edasalonexent for the treatment of DMD.
We have previously reported safety, tolerability and reduction in NF-kB
activity in Phase 1 trials in adults. We are currently conducting the
MoveDMD® trial of edasalonexent in 4-7 year-old boys affected
by Duchenne. From Part A of the MoveDMD trial, we have reported that
edasalonexent was generally well tolerated with no safety signals
observed and we observed successful NF-kB target
engagement. Pharmacokinetic results demonstrated edasalonexent plasma
exposure levels consistent with those previously observed in adults at
which inhibition of NF-kB was observed.
About Sarepta Therapeutics
Sarepta Therapeutics is a
commercial-stage biopharmaceutical company focused on the discovery and
development of unique RNA-targeted therapeutics for the treatment of
rare neuromuscular diseases. The Company is primarily focused on rapidly
advancing the development of its potentially disease-modifying DMD drug
candidates, including EXONDYS 51, designed to skip exon 51 and approved
under the accelerated approval pathway. For more information, please
visit us at www.sarepta.com.
About Duchenne Muscular Dystrophy
DMD is an X-linked rare
degenerative neuromuscular disorder causing severe progressive muscle
loss and premature death. One of the most common fatal genetic
disorders, DMD affects approximately one in every 3,500-5,000 males
worldwide. A devastating and incurable muscle-wasting disease, DMD is
associated with specific errors in the gene that codes for dystrophin, a
protein that plays a key structural role in muscle fiber function.
Progressive muscle weakness in the lower limbs spreads to the arms, neck
and other areas. Eventually, increasing difficulty in breathing due to
respiratory muscle dysfunction requires ventilation support, and cardiac
dysfunction can lead to heart failure. The condition is universally
fatal, and death usually occurs before the age of 30.
Forward-Looking Statements
Any statements in this press
release about future expectations, plans and prospects for Catabasis
and/or Sarepta, including statements about future clinical trial plans
and other statements containing the words “believes,” “anticipates,”
“plans,” “expects,” “may” and similar expressions, constitute
forward-looking statements within the meaning of the Private Securities
Litigation Reform Act of 1995. These forward-looking statements include
statements regarding the joint research collaboration plans of Sarepta
and Catabasis to explore a combination drug treatment approach for DMD
and the potential benefit of the products being researched in DMD.
Actual results may differ materially from those indicated by such
forward-looking statements as a result of various important factors,
including: uncertainties inherent in the initiation and completion of
preclinical studies and clinical trials and clinical development of
Catabasis’ or Sarepta’s product candidates; availability and timing of
results from preclinical studies and clinical trials; whether interim
results from a clinical trial will be predictive of the final results of
the trial or the results of future trials; expectations for regulatory
approvals to conduct trials or to market products; availability of
funding sufficient for Catabasis’ or Sarepta’s foreseeable and
unforeseeable operating expenses and capital expenditure requirements;
other matters that could affect the availability or commercial potential
of Catabasis’ or Sarepta’s product candidates; and general economic and
market conditions and other factors discussed in the “Risk Factors”
section of each of Catabasis’ and Sarepta’s Quarterly Report on Form
10-Q for the period ended June 30, 2016, which are each on file with the
Securities and Exchange Commission, and in other filings that Catabasis
or Sarepta may make with the Securities and Exchange Commission in the
future. In addition, the forward-looking statements included in this
press release represent Catabasis’ and Sarepta’s views as of the date of
this press release. Each of Catabasis and Sarepta anticipates that
subsequent events and developments will cause their respective views to
change. However, while either Catabasis or Sarepta may elect to update
these forward-looking statements at some point in the future, each
company specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as representing the
views of either Catabasis or Sarepta as of any date subsequent to the
date of this release.