- Highly statistically significant and
clinically meaningful results -
- 63% mean blood Phe reduction in primary
analysis population (p<0.0001) -
- Conference call and webcast to be held at
8:00 AM EDT -
SOUTH
PLAINFIELD, N.J., May 17, 2023
/PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today
announced that the primary endpoint was achieved in the APHENITY,
Phase 3 registration-directed clinical trial of sepiapterin in
adult and pediatric patients with phenylketonuria (PKU).
"The positive results from the APHENITY trial bring us one step
closer to providing a therapy that could deliver meaningful benefit
to PKU patients," said Matthew B.
Klein, M.D., Chief Executive Officer, PTC Therapeutics. "The
Phe reductions observed in the placebo-controlled portion of the
study are consistent with, and, in some cases, exceed the magnitude
of Phe reductions recorded in the open label portion of the study.
We look forward to meeting with regulatory authorities to discuss
the path to approval."
The placebo-controlled portion of the study included 98 patients
in the primary analysis population. The mean percent Phe reduction
in sepiapterin treated patients was 63%. In the subset of classical
PKU patients, the mean percent Phe reduction was 69%. Minimal
reductions in Phe levels were observed in the placebo treated
patients resulting in a highly statistically significant
sepiapterin treatment benefit (p<0.0001). Sepiapterin was
generally well tolerated with no serious adverse events.
Today's Conference Call and Webcast
PTC will hold a conference call at 8 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 APHENITY
APHENITY was a global
double-blind, placebo-controlled, registration-directed study which
enrolled 156 children and adults with PKU.
Participants were randomized to receive sepiapterin or
placebo for six weeks with the primary endpoint being reduction in
blood phenylalanine levels. The trial consisted of two
parts. Part 1 was a run-in phase, during which all screened
subjects received sepiapterin for two weeks. Only
those subjects who demonstrated a reduction in phenylalanine
levels of 15% or more from baseline in Part 1 were randomized
to receive either sepiapterin or placebo in Part 2 of
the clinical trial. The primary analysis population consists of
those who had greater than 30% reduction in phenylalanine
levels from baseline during Part 1 of the trial. The primary
outcome measure is the reduction of blood phenylalanine
levels from baseline compared to Weeks 5 and 6 in patients
from Part 2 of the clinical trial. All patients are eligible to
enroll in an open label long term clinical trial designed to
further evaluate the long-term safety and durable effect of
sepiapterin.
About Sepiapterin
Sepiapterin (formerly PTC923) is an
oral formulation of synthetic sepiapterin, a precursor to
intracellular tetrahydrobiopterin, which is a critical enzymatic
cofactor involved in the metabolism and synthesis of numerous
metabolic products. Sepiapterin is a more bioavailable precursor
than exogenously administered synthetic BH4 and has the potential
to treat the broad range of PKU patients.
About Phenylketonuria
Phenylketonuria (PKU) is a rare,
inherited metabolic disease, which affects the brain.1
It is caused by a defect in the gene that helps create the enzyme
needed to break down phenylalanine.1 If left untreated
or poorly managed, phenylalanine – an essential amino acid found in
all proteins and most foods – can build up to harmful levels in the
body. This causes severe and irreversible disabilities, such as
permanent intellectual disability, seizures, delayed development,
memory loss, and behavioral and emotional problems.1
Newborns with phenylketonuria initially don't have any symptoms,
but symptoms are usually progressive, and damage caused by toxic
levels of phenylalanine in the first few years of life is
irreversible.2,3 Diagnosis of phenylketonuria usually
takes place during newborn screening programs.4 There
are an estimated 58,000 people with phenylketonuria globally.
About PTC Therapeutics, Inc.
PTC is a
science-driven, 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, 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; expectations with respect to potential
regulatory submissions and commercialization of sepiapterin for
phenylketonuria, or PKU, and potential development and regulatory
milestone payments that PTC may be obligated to make with regards
to sepiapterin, expectations with respect to the COVID-19 pandemic
and related response measures and their effects on PTC's business,
operations, clinical trials, regulatory submissions and approvals,
and PTC's collaborators, contract research organizations, suppliers
and manufacturers; 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
sepiapterin for PKU; 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
sepiapterin.
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 presentation except
as required by law.
References:
- de Groot MJ, Hoeksma M, Blau N, et al. Mol Genet Metab
2010;99:S86–S89.
- Phenylketonuria (PKU). Available at:
https://www.mayoclinic.org/diseases-conditions/phenylketonuria/symptoms-causes/syc-20376302.
Accessed October 2021.
- Blau N, van Spronsen FJ, Levy HL. Lancet
2010;376:1417–1427.
- Al Hafid N, Christodoulou J. Transl Pediatr
2015;4(4):304–317.
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SOURCE PTC Therapeutics, Inc.