SOUTH PLAINFIELD, N.J.,
Oct. 4, 2016 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today announced that the
Proceedings of the National Academy of Sciences (PNAS) has
published new results further validating Translarna's™ (ataluren)
mechanism of action to promote readthrough of premature stop codons
resulting from nonsense mutations in genetic disorders. The results
reported in PNAS establish ataluren's ability to alter the protein
production process at premature stop codons and to promote the
insertion of specific amino acids and restore the production of a
full-length functional protein.
"These new results help us better understand ataluren's
mechanism of action as well as confirm its protein restoration
effect in genetic disorders," said Stuart
W. Peltz, Ph.D., co-founder and Chief Executive Officer of
PTC Therapeutics. "These results further support our clinical
findings demonstrating the production of full-length functional
protein in nonsense mutation Duchenne muscular dystrophy and cystic
fibrosis. Given this mechanism, ataluren offers the potential for a
new therapeutic approach for multiple nonsense mutation genetic
disorders by targeting the underlying cause of the disease."
The results published by PTC Therapeutics, Dr. Allan Jacobson and his team at the University of Massachusetts Medical School, and Dr.
David Bedwell and his team at the
University of Alabama, demonstrate
ataluren treatment produces a protein that is similar to the
protein from cells that do not have a nonsense mutation. The
findings were verified in multiple nonsense mutation models. In
addition, there have been almost 40 publications to date, many by
independent investigators, demonstrating the clinical activity of
Translarna across a spectrum of rare diseases.
"These data provide new insight on ataluren's effect on protein
production and validates that it targets the source of nonsense
mutation genetic disorders," said Allan
Jacobson, Ph.D., co-founder and Board member of PTC
Therapeutics and the Gerald L. Haidak, MD and Zelda S. Haidak professor of cell biology and
chair of microbiology and physiological systems at University of Massachusetts Medical School.
"Therapeutic nonsense suppression is a potentially powerful
approach for the treatment of the large number of genetic disorders
caused by nonsense mutations."
About Translarna™ (ataluren)
Translarna, discovered
and developed by PTC Therapeutics, Inc., is a protein restoration
therapy designed to enable the formation of a functioning protein
in patients with genetic disorders caused by a nonsense mutation. A
nonsense mutation is an alteration in the genetic code that
prematurely halts the synthesis of an essential protein. The
resulting disorder is determined by which protein cannot be
expressed in its entirety and is no longer functional, such as
dystrophin in Duchenne muscular dystrophy. Translarna is licensed
in the European Economic Area for the treatment of nonsense
mutation Duchenne muscular dystrophy in ambulatory patients aged
five years and older. Translarna is an investigational new drug in
the United States. The development
of Translarna has been supported by grants from Cystic Fibrosis
Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic
Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office
of Orphan Products Development; National Center for Research
Resources; National Heart, Lung, and Blood Institute; and Parent
Project Muscular Dystrophy.
About PTC Therapeutics
PTC is a global
biopharmaceutical company focused on the discovery, development and
commercialization of orally administered, proprietary small
molecule drugs targeting an area of RNA biology we refer to as
post-transcriptional control. Post-transcriptional control
processes are the regulatory events that occur in cells during and
after a messenger RNA, or mRNA, molecule is copied from DNA through
the transcription process. PTC's internally discovered pipeline
addresses multiple therapeutic areas, including rare disorders and
oncology. PTC has discovered all of its compounds currently under
development using its proprietary technologies. PTC plans to
continue to develop these compounds both on its own and through
selective collaboration arrangements with leading pharmaceutical
and biotechnology companies. For more information on the company,
please visit our website www.ptcbio.com.
For More Information:
Investors:
Emily
Hill
+ 1 (908) 912-9327
ehill@ptcbio.com
Media:
Jane Baj
+1 (908) 912-9167
jbaj@ptcbio.com
Forward Looking Statements:
All statements, other than those of historical fact, contained
in this press release, are forward-looking statements, including
statements regarding the future expectations, plans and prospects
for PTC; the clinical utility and potential advantages of
Translarna (ataluren); and the objectives of management.
Other forward-looking statements may be identified by the words
"potential," "plan," "target," "anticipate," "believe," "estimate,"
"expect," "intend," "may," "project," "possible," "potential,"
"will," "would," "could," "should," "continue," and similar
expressions.
PTC's actual results, performance or achievements could differ
materially from those expressed or implied by forward-looking
statements it makes as a result of a variety of risks and
uncertainties, including those related to: PTC's ability to
maintain its marketing authorization of Translarna for the
treatment of nmDMD in the European Economic Area (EEA), including
whether the European Medicines Agency (EMA) determines that the
benefit-risk balance of Translarna supports renewal of PTC's
marketing authorization in the EEA; the nature and scope of any new
nonsense mutation Duchenne muscular dystrophy (nmDMD) trial that
PTC may design with the input of the EMA and PTC's ability to
enroll, fund and conduct such trial; the outcome of future
interactions PTC has with the FDA with respect to Translarna for
the treatment of nmDMD, including whether PTC is required to
perform additional clinical and non-clinical trials at significant
cost and whether such trials, if successful, may enable FDA review
of a New Drug Application submission by PTC; the EMA's
determinations with respect to PTC's variation submission which
seeks to add Translarna for the treatment of nonsense mutation
cystic fibrosis to PTC's marketing authorization in the EEA; the
scope of regulatory approvals or authorizations for Translarna (if
any), including labeling and other matters that could affect the
availability or commercial potential of Translarna; the outcome of
ongoing or future clinical trials or studies, including ACT CF and
the Phase 2 study of Translarna for nmDMD in pediatric patients;
the eligible patient base and commercial potential of Translarna
and PTC's other product candidates; PTC's ability to commercialize
and commercially manufacture Translarna in general and specifically
as a treatment for nmDMD, including its ability to establish and
maintain arrangements with manufacturers, suppliers, distributors
and production and collaboration partners on favorable terms; the
outcome of pricing and reimbursement negotiations in those
territories in which PTC is authorized to sell Translarna;
expectations for regulatory approvals, including PTC's ability to
make regulatory submissions in a timely manner (or at all), the
period during which the outcome of regulatory reviews will become
available, adverse decisions by regulatory authorities, other delay
or deceleration of the regulatory process, and PTC's ability to
meet existing or future regulatory standards with respect to
Translarna; PTC's ability to fulfill any additional obligations,
including with respect to further trials or studies relating to
cost-effectiveness, obtaining licenses or satisfying requirements
for labor and business practices, in the territories in which it
may obtain regulatory approval, including the United States, EEA and other territories;
the initiation, conduct and availability of data from clinical
trials and studies; PTC's scientific approach and general
development progress; the sufficiency of PTC's cash resources and
PTC's ability to obtain adequate financing in the future for PTC's
foreseeable and unforeseeable operating expenses and capital
expenditures; and the factors discussed in the "Risk Factors"
section of PTC's most recent Quarterly Report on Form 10-Q as well
as any updates to these risk factors filed from time to time in
PTC's other filings with the SEC. You are urged to carefully
consider all such factors.
As with any pharmaceutical under development, there are
significant risks in the development, regulatory approval and
commercialization of new products. There are no guarantees that
Translarna will receive full regulatory approval in any territory
or maintain its current marketing authorization in the EEA, or
prove to be commercially successful in general, or specifically
with respect to the treatment of nmDMD.
The forward-looking statements contained herein represent PTC's
views only as of the date of this press release and PTC does not
undertake or plan to update or revise any such forward-looking
statements to reflect actual results or changes in plans,
prospects, assumptions, estimates or projections, or other
circumstances occurring after the date of this press release except
as required by law.
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SOURCE PTC Therapeutics, Inc.