SOUTH PLAINFIELD,
N.J., June 29, 2023 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced today that the MIT-E
trial of vatiquinone for the treatment of mitochondrial disease
associated seizures (MDAS) failed to achieve its primary endpoint
of reduction in observable motor seizures. The study showed
evidence of treatment effect in reducing seizure frequency in the
overall study population and in the largest subgroup of children
with Leigh syndrome, in whom benefit was also observed in the key
secondary endpoints of occurrence of status epilepticus and
disease-related hospitalizations.
"We are incredibly grateful to the patients, their families and
our investigators who participated in this important study," said
Dr. Matthew Klein, Chief Executive
Officer of PTC Therapeutics, Inc. "While we are disappointed with
the results of the study, we hope that there are learnings that can
benefit the development of other therapies for patients with
mitochondrial disease, who remain without approved treatments for
this highly morbid and fatal set of diseases."
The MIT-E study is a double-blind, placebo-controlled study of
vatiquinone in pediatric patients with genetically confirmed
mitochondrial disease and associated refractory seizures. The study
enrolled 68 children at study sites worldwide. The study included a
24-week placebo-controlled phase followed by a 48-week open-label
phase. The primary endpoint was the reduction in observable motor
seizures during the 24 weeks placebo-controlled phase. All subjects
who completed the placebo-controlled phase were eligible to
participate in the open-label phase of the study.
While the MIT-E study results do not support the advancement of
vatiquinone for the treatment of MDAS, PTC continues to plan to
discuss the results of the MOVE-FA trial of vatiquinone for the
treatment of Friedreich ataxia with regulatory authorities based on
the important signals of clinical benefit on key subscales of the
mFARS, such as upright stability.
About Vatiquinone
Vatiquinone is an investigational
new drug that targets 15-lipoxygenase, a regulator of key energetic
and oxidative stress pathways. Vatiquinone has been evaluated in a
number of clinical studies and has demonstrated an impact on
mortality risk and a number of neurological and neuromuscular
disease symptoms.1,2,3 The treatment also has
extensive safety data, with the longest duration of exposure being
over 10 years.
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
Forward-Looking Statements:
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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 with
respect to 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
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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
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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.
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.
References:
- Hinman A, et al. PloS one. 2018;13:e0201369.
- PTC. EPI-743 Pre-Clinical Data Deck.
- Kahn-Kirby AH, et al. PloS one. 2019;14:e0214250.
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SOURCE PTC Therapeutics, Inc.