- Translarna CHMP opinion re-examination
request to be submitted -
SOUTH PLAINFIELD,
N.J., Sept. 28, 2023 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced today further strategic
prioritization and associated workforce reduction. The portfolio
prioritization continues the process initiated in May 2023 as the company continues to focus its
resources on its differentiated, high potential R&D programs
and on support of the robust global commercial infrastructure. The
company also confirmed today its plans to submit the re-examination
request for the CHMP opinion on Translarna.
"We remain confident that we have the data to address the
concerns raised by CHMP in its negative opinion," said Matthew B. Klein, M.D., Chief Executive Officer,
PTC Therapeutics. "The totality of evidence collected in three
placebo-controlled trials and in the STRIDE registry provide clear
evidence of Translarna's benefit. In addition, the patient and
physician communities strongly believe in the benefits of
Translarna and have shared their motivation to support the efforts
to maintain authorization for the only approved Duchenne muscular
dystrophy therapy in Europe."
The workforce reduction impacted approximately 25% of the
organization, and primarily included employees working on
early-stage research programs, employees at the gene therapy
manufacturing facility located in Hopewell, NJ and associated SG&A
functions. Implementation of these cost saving measures is expected
to result in an approximate 20% reduction in annualized operating
expenses compared to 2023 OPEX guidance.
"I would like to acknowledge the tremendous contribution of the
PTC team members affected by the reduction in workforce," Klein
continued. "The workforce and OPEX reductions continue the efforts
we began in May to focus resources on key R&D programs and our
commercial enterprise. PTC has a number of valuable assets that
form the foundation of a promising future including the sepiapterin
PKU program, the PTC518 Huntington disease program and the Evrysdi
collaboration and royalty revenue."
About PTC Therapeutics, Inc.
PTC is a global
biopharmaceutical company focused on the discovery, development and
commercialization of clinically differentiated medicines that
provide benefits to patients with rare disorders. PTC's ability to
innovate to identify new therapies and to globally commercialize
products is the foundation that drives investment in a robust and
diversified pipeline of transformative medicines. PTC's mission is
to provide access to best-in-class treatments for patients who have
little to no treatment options. PTC's strategy is to leverage its
strong scientific and clinical expertise and global commercial
infrastructure to bring therapies to patients. PTC believes this
allows it to maximize value for all its stakeholders. To learn more
about PTC, please visit us at www.ptcbio.com and follow us on
Facebook, Instagram, LinkedIn and Twitter at @PTCBio.
For More
Information:
Investors:
Kylie
O'Keefe
+1 (908) 300-0691
kokeefe@ptcbio.com
Media:
Jeanine
Clemente
+1 (908) 912-9406
jclemente@ptcbio.com
Acronyms:
CHMP: Committee for Medicinal Products for
Human Use
EMA: European Medicines Agency
OPEX: Operating Expenses
PKU: Phenylketonuria
SG&A: Selling, General and Administrative
Forward-Looking Statement
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. All statements contained in this press release, other than
statements of historic fact, are forward-looking statements,
including statements regarding the extent, timing and financial
aspects of the strategic prioritization and reduction in workforce;
PTC's plans for interactions with the European Medicines Agency
(EMA); the outcome of any re-examination process; the clinical
utility and potential advantages of Translarna (ataluren); the
future expectations, plans and prospects for PTC, including with
respect to the expected timing of clinical trials and studies,
availability of data, regulatory submissions and responses and
other matters, future strategy, future operations, future financial
position, future revenues, projected costs; and the objectives of
management. Other forward-looking statements may be identified by
the words, "guidance", "plan," "anticipate," "believe," "estimate,"
"expect," "intend," "may," "target," "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: the outcome of pricing,
coverage and reimbursement negotiations with third party payors for
PTC's products or product candidates that PTC commercializes or may
commercialize in the future; the timing of and actual expenses
incurred in connection with the strategic prioritization and
reduction in workforce, which may be in different periods and may
be materially higher than we estimate; the savings that may result
from the strategic prioritization and reduction in workforce, which
may be materially less than we expect; PTC's ability to maintain
its marketing authorization of Translarna for the treatment of
nmDMD in Brazil, Russia, the European Economic Area (EEA) and
other regions, including whether the European Medicines Agency
(EMA) determines in the re-examination process that the
benefit-risk balance of Translarna authorization supports renewal
of such authorization or its conversion to a full approval; PTC's
ability to complete Study 041, a randomized, 18-month,
placebo-controlled clinical trial of Translarna for the treatment
of nmDMD followed by an 18-month open-label extension, which is a
specific obligation to continued marketing authorization in the
EEA; PTC's ability to utilize results from Study 041 to support a
marketing approval for Translarna for the treatment of nmDMD in
the United States and a conversion
to a standard marketing authorization in the EEA; whether
investigators agree with PTC's interpretation of the results of
clinical trials and the totality of clinical data from our trials
in Translarna; significant business effects, including the effects
of industry, market, economic, political or regulatory conditions;
changes in tax and other laws, regulations, rates and policies; the
eligible patient base and commercial potential of PTC's products
and product candidates; PTC's scientific approach and general
development progress; the sufficiency of PTC's cash resources and
its ability to obtain adequate financing in the future for its
foreseeable and unforeseeable operating expenses and capital
expenditures; and the factors discussed in the "Risk Factors"
section of PTC's most recent Annual Report on Form 10 -K, 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 any
product will receive or maintain regulatory approval in any
territory, or prove to be commercially successful, including
Translarna.
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.