- Dose-dependent lowering of blood
Huntingtin (HTT) protein levels at 12 weeks -
-
Favorable tolerability profile with no treatment-related serious
adverse events or NfL spikes -
- Conference call and webcast to be held
June 21st at
8:00 am EDT -
SOUTH
PLAINFIELD, N.J., June 21,
2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:
PTCT) today shared interim data from the 12-week portion of the
PIVOT-HD Phase 2 study of PTC518 in Huntington's disease (HD)
patients. The study demonstrated dose-dependent lowering of
Huntingtin (HTT) protein levels in peripheral blood cells, reaching
a mean 30% reduction in mutant HTT levels at the 10mg dose level.
In addition, PTC518 exposure in the cerebrospinal fluid (CSF) was
consistent with or higher than plasma unbound drug levels.
PTC518 treatment was also demonstrated to be well tolerated,
with no treatment-related serious adverse events, no reports of
peripheral neuropathy or dose-limiting toxicities. Furthermore,
there were no CSF neurofilament light chain protein (NfL)
treatment-related spikes, with an overall trend towards lowering of
CSF NfL levels following 12 weeks of PTC518 treatment.
"We are very pleased with the encouraging data from the PIVOT-HD
interim analysis demonstrating dose-dependent HTT lowering, desired
CSF exposure and a favorable tolerability profile without evidence
of treatment-related serious adverse events or CSF NfL spikes,"
said Dr. Matthew Klein, Chief
Executive Officer, PTC Therapeutics, Inc.
PIVOT-HD is a global, placebo-controlled study and consists of
two parts: an initial 12-week placebo-controlled phase focused on
PTC518 pharmacology and pharmacodynamic effect, followed by a
9-month placebo-controlled portion. The study will initially
include two dose levels, 5 milligrams and 10 milligrams, with the
ability to include a third dose level of up to 20 milligrams.
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.
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.
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SOURCE PTC Therapeutics, Inc.