- FDA lifts PTC518 partial
clinical hold based on PIVOT-HD data -
- Conference call and webcast to be held
June 20th at
8:00 am EDT -
WARREN,
N.J., June 20, 2024 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) today shared interim results from
the Phase 2 PIVOT-HD study of PTC518 in Huntington's disease (HD)
patients. At Month 12, PTC518 treatment resulted in dose-dependent
lowering of mutant huntingtin (mHTT) protein in the blood and
cerebrospinal fluid (CSF) in the interim cohort of patients. In
addition, favorable trends were demonstrated on several relevant HD
clinical assessments including Total Motor Score (TMS) and
Composite Unified Huntington's Disease Rating Scale (cUHDRS).
Furthermore, following 12 months of treatment, PTC518 continues to
be safe and well tolerated.
"The evidence of both CNS biomarker and early clinical effects
at Month 12 along with the continued favorable tolerability profile
supports the promise of PTC518 to address the need for an effective
and safe disease-modifying therapy for patients living with
Huntington's disease," said Dr. Matthew B.
Klein, Chief Executive Officer, PTC Therapeutics, Inc. "With
these data in hand, we look forward to the next steps in the PTC518
development program."
At Month 12, durability of dose-dependent mHTT lowering in the
blood was demonstrated with lowering of 22% and 43% for 5mg and
10mg dose levels, respectively. In the CSF, dose dependent mHTT
lowering was also demonstrated with lowering of 21% and 43%, for
5mg and 10mg dose levels, respectively. In addition, at Month 12,
PTC518 treatment resulted in a notable slowing in progression of
motor symptoms as assessed by the TMS (2.0 points worsening for 5mg
and 1.3 points worsening for 10mg vs. 4.9 points worsening for
placebo).
In addition, PTC announced that the FDA has lifted the partial
clinical hold on the program based on review of the PIVOT-HD
data.
Today's Conference Call and Webcast
PTC will hold a conference call at 8:00 am
EDT today to discuss this news. To access the call by phone,
please click here to register and you will be provided with dial-in
details. To avoid delays, we recommend participants dial in to the
conference call 15 minutes prior to the start of the call. The
webcast conference call can be accessed on the Investor section of
the PTC website at https://ir.ptcbio.com/events-presentations. A
replay of the call will be available approximately two hours after
completion of the call and will be archived on the company's
website for 30 days following the call.
About PTC518
PTC is developing a potential treatment
for Huntington's disease based on our splicing platform technology.
PTC518, a small molecule that can be taken orally, reduces the
production of the mutated Huntingtin protein that leads to
injury and death of the neuron, which results in disease
progression. The orally bioavailable small molecule penetrates the
blood brain barrier, is selective, titratable, and not effluxed –
which are key differentiation properties.
About Huntington's Disease
Huntington's disease (HD)
is a rare, hereditary, genetic disorder of the central nervous
system.1 It is caused by a defective gene. This gene produces a
protein, called Huntingtin, which is involved in the functioning of
the nerve cells in the brain (neurons). When the gene is defective,
it produces an abnormal (or mutated) Huntingtin protein that is
toxic and causes neuron damage and neuron death.2 HD usually
presents in people who are in their 30s or 40s. Symptoms can
present earlier in life, and this is called the Juvenile HD.2,3
There are also cases of infantile HD, when symptoms develop in
children who are younger than 10 years old.2 While symptoms vary
from person to person, the disease primarily affects the brain and
results in abnormal movements, difficulties with speech, swallowing
and walking, as well as a number of other symptoms including
behavioral, cognitive and motor symptoms. 4,5 While there are
therapies approved for specific disease symptoms, currently, there
is no cure for HD and there are no approved drugs that delay the
onset or slow disease progression.
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 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 with respect to
the future expectations, plans and prospects for PTC, PTC's
strategy, including with respect to the expected timing of clinical
trials and studies, availability of data, regulatory submissions
and responses and other matters, 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; 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.
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.
Acronyms:
CNS: Central Nervous System
CSF: Cerebrospinal Fluid
FDA: U.S. Food and Drug Administration
References:
- World Health Organization, 2020. 8A01.10 Huntington disease.
Available at:
https://icd.who.int/browse11/l-m/en#/http://id.who.int/icd/entity/2132180242
Accessed October 2021.
- Gatto EM, González Rojas N, Persi G, et al. Clin Parkinsonism
Rel Disord 2020;3:100056.
- Tabrizi SJ, Flower MD, Ross
CA, et al. Nat Rev Neurol 2020;16(10):529–546.
- Roos RAC. Orphanet J Rare Dis 2010;5:40.
- Kirkwood SC, Su JL, Conneally P, et al. Arch Neurol
2001;58(2):273–278.
View original
content:https://www.prnewswire.com/news-releases/interim-pivot-hd-results-demonstrate-evidence-of-favorable-cns-biomarker-and-clinical-effects-at-month-12-in-huntingtons-disease-patients-302177663.html
SOURCE PTC Therapeutics, Inc.