- Results from the APHENITY trial in PKU and
long-term extension study including Phe tolerance to be highlighted
-
SOUTH
PLAINFIELD, N.J., Aug. 28,
2023 /PRNewswire/ -- PTC Therapeutics, Inc.
(NASDAQ: PTCT) today announced that results from the APHENITY
clinical trial and long-term extension study will be presented at
the Society for the Study of Inborn Errors of Metabolism (SSIEM)
Annual Symposium 2023 which is being held from Aug. 29 to Sept. 1 in Jerusalem, Israel.
Topline results of the APHENITY trial will be presented in an
oral presentation, "Phase 3 APHENITY Study: Oral Sepiapterin for
the Treatment of Phenylketonuria." The presentation will take place
on Thursday, Aug. 31 during the
Parallel Session: Phenylketonuria, beginning at 10:45 IST.
The poster "B284: Phase 3 APHENITY Long-Term Study Design:
Sepiapterin for Treatment of Phenylketonuria," will also be
presented on Wednesday, Aug. 30
during the poster walk at 18:35 IST.
The poster contains an updated data cut of the ongoing APHENITY
long-term extension study. Phe tolerance results continued to
demonstrate that sepiapterin treated patients maintain mean blood
Phe control (>360 µmol/L) while significantly increasing protein
intake, consistent with previous results.
PTC will also be sponsoring a symposium, "Advancing the
Treatment of PKU: Results of the APHENITY Trial," which will be
hosted on Aug. 31 at 12:30 IST in Dulcin Hall. The symposium will
bring together leading international experts to discuss the latest
results from the APHENITY trial, highlight the unique features of
sepiapterin, and share findings from the dietary Phe tolerance
assessment in the open-label extension study.
In addition, PTC will share ongoing research for aromatic
l-amino acid decarboxylase (AADC) deficiency during an oral
presentation, poster and sponsored symposium.
More information about the SSIEM Annual Symposium can be found
at ssiem.org.
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
percent 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 percent 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.
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; 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.
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:
|
1.
|
de Groot MJ, Hoeksma M,
Blau N, et al. Mol Genet Metab 2010;99:S86–S89.
|
2.
|
Phenylketonuria (PKU).
Available at:
https://www.mayoclinic.org/diseases-conditions/phenylketonuria/symptoms-causes/syc-20376302.
Accessed October 2021.
|
3.
|
Blau N, van Spronsen
FJ, Levy HL. Lancet 2010;376:1417–1427.
|
4.
|
Al Hafid N,
Christodoulou J. Transl Pediatr 2015;4(4):304–317.
|
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SOURCE PTC Therapeutics, Inc.