Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced that the U.S.
Food and Drug Administration (FDA) has placed a clinical hold on
SRP-5051 (vesleteplirsen), the Company’s next-generation
peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) to
treat patients with Duchenne muscular dystrophy who are amenable to
exon 51 skipping. The hold in Part B of Study 5051-201, also known
as MOMENTUM, follows a serious adverse event of hypomagnesemia. FDA
is requesting information on all cases of hypomagnesemia, including
a small number of non-serious grade 2 cases, and to assess the
adequacy of the risk mitigation and safety monitoring plan. In the
next few days we will respond to the Agency with this information
and proposed changes to the monitoring plan.
“Patient safety is
always our top priority. The hypomagnesemia was identified through
lab tests conducted as part of the monitoring outlined in the
protocol of the MOMENTUM study and is similar to previously
observed cases of hypomagnesemia in clinical trials of SRP-5051.
The hypomagnesemia was transient and patients’ magnesium levels
returned to normal following additional supplementation,” said
Louise Rodino-Klapac, Ph.D., executive vice president and chief
scientific officer, Sarepta Therapeutics. “Globally, we have
enrolled approximately half of the planned patients in Part B of
MOMENTUM. The study is ongoing, and we remain on track to complete
enrollment by the end of the year. We will work to share
information with FDA with the goal of resuming screening and dosing
in the U.S. as quickly as possible.”
Sarepta will host an
investor conference call on Thurs., June 23, 2022 at 4:15 pm
Eastern time, to discuss this update. The presentation will be
webcast live under the investor relations section of Sarepta's
website
at https://investorrelations.sarepta.com/events-presentations and
archived there following the call for one year. Please connect to
Sarepta's website several minutes prior to the start of the
broadcast to ensure adequate time for any software download that
may be necessary. The conference call may be accessed by dialing
(844) 534-7313 for domestic callers and (574) 990-1451 for
international callers. The passcode for the call is 6055778. Please
specify to the operator that you would like to join the "SRP-5051
Program Update."
About SRP-5051
(vesleteplirsen) SRP-5051 is an investigational agent
using Sarepta’s PPMO chemistry and exon-skipping technology to skip
exon 51 of the dystrophin gene. SRP-5051 is designed to bind to
exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon
during mRNA processing in patients with genetic mutations that are
amenable to exon 51 skipping. Exon skipping is intended to allow
for production of an internally shortened functional dystrophin
protein. PPMO is Sarepta’s next-generation chemistry platform
designed around a proprietary cell-penetrating peptide conjugated
to the PMO backbone, with the goal of increasing tissue
penetration, increasing exon skipping, and significantly increasing
dystrophin production. Around 13% of DMD patients have mutations
that make them amenable to skipping exon 51. If successful, the
PPMO offers the potential for improved efficacy and less frequent
dosing for patients.
About MOMENTUM
(Study SRP-5051-201) MOMENTUM is Phase 2, multi-arm,
ascending dose study of SRP-5051, infused monthly and will assess
dystrophin protein levels in skeletal muscle tissue following
SRP-5051 treatment. The study will enroll up to 60 participants,
both ambulant and non-ambulant, between the ages of 7 to 21 at
sites in the U.S., Canada, and the European Union. The study will
also assess safety and tolerability.
In 2021, the Company
announced results from Part A of MOMENTUM showing that after 12
weeks, 30 mg/kg of SRP-5051 dosed monthly resulted in 18 times the
exon skipping and eight times the dystrophin production as
eteplirsen, dosed weekly for 24 weeks. Reversible hypomagnesemia
was identified in patients taking SRP-5051. The protocol for Part B
of MOMENTUM includes magnesium supplementation and monitoring of
magnesium levels.
More information can
be found on www.clinicaltrials.gov.
About Sarepta
TherapeuticsSarepta is on an urgent mission: engineer
precision genetic medicine for rare diseases that devastate lives
and cut futures short. We hold leadership positions in Duchenne
muscular dystrophy (DMD) and limb-girdle muscular dystrophies
(LGMDs), and we currently have more than 40 programs in various
stages of development. Our vast pipeline is driven by our
multi-platform Precision Genetic Medicine Engine in gene therapy,
RNA, and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Internet
Posting of InformationWe routinely post information that
may be important to investors in the 'For Investors' section of our
website at www.sarepta.com. We encourage investors and
potential investors to consult our website regularly for important
information about us.
Forward-Looking StatementsThis press release
contains "forward-looking statements." Any statements contained in
this press release that are not statements of historical fact may
be deemed to be forward-looking statements. Words such as
"believes," "anticipates," "plans," "expects," "will," "intends,"
"potential," "possible" and similar expressions are intended to
identify forward-looking statements. These forward-looking
statements include statements regarding the potential benefits of
PPMO and SRP-5051; our belief that the hypomagnesemia identified in
MOMENTUM is similar to previously observed cases in clinical trials
of SRP-5051; our approach to monitoring and managing
hypomagnesemia; and our expected timelines, plans, and milestones,
including completing enrollment of Part B of MOMENTUM by the end of
the year, our plan to respond to the Agency with the requested
information and proposed changes to the monitoring plan in the next
few days, and resuming screening and dosing in the U.S. as quickly
as possible.
These forward-looking
statements involve risks and uncertainties, many of which are
beyond our control. Known risk factors include, among others:
success in preclinical and clinical trials, especially if based on
a small patient sample, does not ensure that later clinical trials
will be successful, and may not be consistent with the final data
set and analysis thereof or result in a safe or effective treatment
benefit; different methodologies, assumptions and applications we
utilize to assess particular safety or efficacy parameters may
yield different statistical results, and even if we believe the
data collected from clinical trials of our product candidates are
positive, these data may not be sufficient to support approval by
the FDA or foreign regulatory authorities; we may not be able to
execute on our business plans and goals, including meeting our
expected or planned regulatory milestones and timelines, clinical
development plans, and bringing our product candidates to market,
due to a variety of reasons, some of which may be outside of our
control, including possible limitations of company financial and
other resources, manufacturing limitations that may not be
anticipated or resolved for in a timely manner, regulatory, court
or agency decisions, such as decisions by the United States Patent
and Trademark Office with respect to patents that cover our product
candidates and the ongoing COVID-19 pandemic; and those risks
identified under the heading “Risk Factors” in Sarepta’s most
recent Annual Report on Form 10-K for the year ended December 31,
2021, and most recent Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission (SEC) as well as other SEC
filings made by the Company, which you are encouraged to
review.
Any of the foregoing
risks could materially and adversely affect the Company’s business,
results of operations and the trading price of Sarepta’s common
stock. For a detailed description of risks and uncertainties
Sarepta faces, you are encouraged to review the SEC filings made by
Sarepta. We caution investors not to place considerable reliance on
the forward-looking statements contained in this press release.
Sarepta does not undertake any obligation to publicly update its
forward-looking statements based on events or circumstances after
the date hereof.
Source: Sarepta Therapeutics, Inc.
Investor Contact: Ian Estepan,
617-274-4052iestepan@sarepta.com
Media Contact: Tracy Sorrentino,
617-301-8566tsorrentino@sarepta.com
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