– Sarepta obtains exclusive worldwide licenses to four
clinical-stage and three preclinical-stage programs in muscle,
central nervous system, and rare pulmonary disorders, including
potential best-in-class siRNA-based treatments for DM1 and
FSHD
– Additionally, Arrowhead and Sarepta have entered into a
discovery partnership pursuant to which Sarepta will nominate, and
Arrowhead will deliver, IND-ready constructs for six targets across
skeletal muscle, cardiac, and CNS
– Investigational treatments leverage Arrowhead’s leading
Targeted RNAi Molecule (TRiMTM) platform, capable of deep and
durable target-gene knockdown
– Upon closing, Arrowhead to receive $500 million in an
upfront payment and $325 million equity investment at a 35%
premium, plus additional future milestone payments and
royalties
– Separately, Sarepta’s Board of Directors has approved a
share repurchase authorization of up to $500 million
– Sarepta to host an investor conference call on Tuesday,
Nov. 26, 2024, at 8:30 a.m. ET
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in
precision genetic medicine for rare diseases, today announced an
exclusive global licensing and collaboration agreement with
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR). Sarepta will obtain
exclusive global rights to multiple clinical, preclinical, and
discovery-stage programs for rare, genetic diseases of the muscle,
central nervous system (CNS), and the lungs.
The agreement will add meaningfully to Sarepta’s mid- and
early-stage pipeline, complementing the Company’s existing
leadership in Duchenne muscular dystrophy and limb-girdle muscular
dystrophies and gene therapy, while adding new indications and
expanding into adjacent therapeutic areas. In addition, Doug
Ingram, president and chief executive officer, Sarepta, will be
appointed to Arrowhead’s Board of Directors.
The clinical-stage programs covered under the agreement
include:
- ARO-DUX4: designed to reduce the production of human
double homeobox 4 (DUX4) protein in skeletal muscle; currently in a
Phase 1/2 clinical study for the treatment of facioscapulohumeral
muscular dystrophy (FSHD)
- ARO-DM1: designed to target and suppress myotonic
dystrophy protein kinase (DMPK) in skeletal muscle; Phase 1/2
clinical study for myotonic dystrophy type 1 (DM1)
- ARO-MMP7: designed to reduce expression of matrix
metalloproteinase 7 (MMP7) in pulmonary epithelial cells; Phase 1/2
clinical study for idiopathic pulmonary fibrosis (IPF)
- ARO-ATXN2: designed to target the ataxin-2 protein
(ATXN2) in the CNS; expected to begin Phase 1/2 clinical study for
spinocerebellar ataxia 2 (SCA2) by the end of 2024
The clinical programs use Arrowhead’s proprietary Targeted RNAi
Molecule (TRiMTM) platform, which is designed to deliver siRNA to
multiple tissue and cell types throughout the body to initiate the
RNA interference mechanism and induce rapid and durable knockdown
of target genes.
The preclinical programs covered under the agreement will
leverage Arrowhead’s TRiM CNS delivery platform designed for
subcutaneous administration and include:
- ARO-ATXN1: designed to target the ataxin-1 protein
(ATXN1) for SCA1
- ARO-ATXN3: designed to target the ataxin-3 protein
(ATXN3) for SCA3
- ARO-HTT: designed to target huntingtin (HTT), a gene
linked to Huntington's disease
Additionally, Sarepta and Arrowhead have entered into a
discovery collaboration for up to six additional muscle, cardiac,
and/or CNS targets, using Arrowhead’s novel delivery technologies.
As part of the collaboration, Sarepta has an exclusive license to
Arrowhead’s technology to develop therapeutics against a broad
range of skeletal muscle gene targets.
“With the launch of Elevidys going exceedingly well, this broad
siRNA collaboration with Arrowhead provides a synergistic platform
to complement Sarepta’s gene therapy and gene editing engine.
Through a strategic deployment of capital, we are able to access
Arrowhead’s leading RNAi platform and will work to rapidly advance
new treatments for devastating genetic diseases where there is
significant unmet need. The agreement affords multiple potential
blockbuster opportunities, serves our strategic priorities for the
remainder of the decade and beyond, and diversifies our business
model across one-time therapies and chronic treatments allowing for
long-term growth and success. Given the strength of our performance
and ability to generate substantial cash to invest in our business
over the next several years, Sarepta’s Board of Directors has
approved a $500 million share repurchase program as part of our
overall capital allocation strategy,” said Mr. Ingram. “We look
forward to embarking on this partnership with Arrowhead, having
been impressed with their scientific capabilities in developing a
potentially best-in-class approach to siRNA and the quality of the
team they have built. Over the course of the next 12-18 months, we
expect to share multiple data readouts from across our
pipeline.”
“We welcome the Sarepta team as new Arrowhead collaboration
partners who bring a wealth of clinical, regulatory, and commercial
expertise in key areas outside of our cardiometabolic focus. We see
our TRiM platform as a broad and elegant solution for delivery of
siRNA to multiple cell types throughout the body. We also have a
very efficient drug discovery engine that continues to generate
many promising programs, and we have great confidence in Sarepta’s
ability to take the next steps to advance and commercialize
multiple Arrowhead-discovered drug candidates, which we believe
have the potential to be best-in-class,” said Chris Anzalone,
Ph.D., president and CEO at Arrowhead. “At the close of this
agreement, Doug Ingram will be appointed to the Arrowhead board of
directors. He has led Sarepta as they advanced multiple
investigational medicines through the clinical and regulatory
process, built a commercial organization from the ground up,
launched multiple drugs, and moved the company toward
profitability. His experience and guidance will be valuable as
Arrowhead seeks the same transition.”
“Robust and compelling early data from Arrowhead’s
differentiated siRNA approach platform suggests potentially
best-in-class treatments that will profoundly improve the lives of
those with rare, genetic diseases,” said Louise Rodino-Klapac,
Ph.D., chief scientific officer and head of research and
development, Sarepta. “The targeted ligand approach, combined with
Arrowhead’s clinically validated siRNA chemistry, suggests the
potential for deep and durable knockdown of proteins that are
over-expressed in these conditions. Arrowhead’s innovative approach
to cross the blood brain barrier with subcutaneous dosing
represents a potential paradigm shift for the CNS preclinical and
discovery programs as part of this collaboration.”
Summary of Financial Terms
Under the terms of the agreement, Sarepta will pay Arrowhead an
upfront payment of $500 million and an equity investment of $325
million in Arrowhead common stock, priced at a 35% premium to the
30-day volume weighted average price prior to the announcement of
the agreement. Arrowhead will also receive $250 million to be paid
in annual installments of $50 million over five years, in addition
to being eligible for future milestone payments and royalties.
Arrowhead will be responsible for Phase 1/2 trials currently
underway, and clinical stage programs will transition to Sarepta no
later than the completion of current trials. Preclinical assets
will transition to Sarepta upon completion of IND-enabling
activities by Arrowhead.
The deal will be funded with cash on hand and Sarepta does not
plan to raise additional capital via the debt or equity capital
markets. The transaction is expected to close early 2025, subject
to the expiration or termination of the waiting period under the
Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other
customary conditions.
Ropes & Gray LLP is serving as legal advisor to Sarepta.
Share Repurchase Program
Sarepta’s Board of Directors has also approved a share
repurchase program of up to $500 million of the Company’s
outstanding common stock over the next 18 months.
The timing of any stock repurchases will be based upon the
continuing analysis of market, financial, and other factors by the
Company’s management. Repurchases under the authorized stock
repurchase program may be made using a variety of methods, which
may include, but are not limited to, open market purchases,
privately negotiated transactions, accelerated share repurchase
agreements or purchases pursuant to a Rule 10b5-1 plan under the
Securities Exchange Act of 1934, as amended. The authorized stock
repurchase program may be suspended, delayed or discontinued at any
time.
Sarepta Investor Call Details
At 8:30 a.m. ET on Nov. 26, 2024, Sarepta will host a conference
call and webcast to discuss this agreement.
The event will be webcast live under the investor relations
section of Sarepta’s website at
https://investorrelations.sarepta.com/events-presentations and
following the event a replay will be archived there for one year.
Interested parties participating by phone will need to register
using this online form. After registering for dial-in details, all
phone participants will receive an auto-generated e-mail containing
a link to the dial-in number along with a personal PIN number to
use to access the event by phone.
About Sarepta Therapeutics
Sarepta 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 LinkedIn, X, Instagram and Facebook.
Sarepta Forward-Looking Statements
This 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 but are not limited to
statements regarding the closing of the transaction and the equity
investment; Sarepta’s payment of regulatory and sales milestones,
and royalty payments to Arrowhead pursuant to the agreement; the
expected targets of the clinical and pre-clinical programs licensed
pursuant to the agreement; ongoing development of therapeutics
against a broad range of skeletal muscle gene targets by Sarepta
and Arrowhead; Arrowhead’s potentially best-in-class approach to
siRNA; the expected timing of future data readouts; and statements
about the amount and timing of repurchases under Sarepta’s share
purchase program and the methods to execute such repurchases.
These forward-looking statements involve risks and
uncertainties, many of which are beyond Sarepta's control. Known
risk factors include, among others, market conditions, the expected
benefits and opportunities related to the collaboration agreement
may not be realized or may take longer to realize than expected due
to a variety of reasons, including any inability of the parties to
perform their commitments and obligations under the agreement,
challenges and uncertainties inherent in product research and
development and manufacturing limitations; success in preclinical
testing and early clinical trials, especially if based on a small
patient sample, does not ensure that later clinical trials will be
successful, and early results from a clinical trial do not
necessarily predict final results; Sarepta may not be able to
execute on its business plans, including meeting its expected or
planned regulatory milestones and timelines, research and clinical
development plans, for various reasons, some of which may be
outside of Sarepta’s control, including possible limitations of
company financial and other resources; and those risks identified
under the heading “Risk Factors” in Sarepta’s most recent Quarterly
Report on Form 10-Q for the quarter ended September 30, 2024, 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
Sarepta’s business, results of operations and the trading price of
Sarepta’s common stock. 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, except as required by law.
Source: Sarepta Therapeutics, Inc.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20241126242387/en/
Investor Contact: Ian Estepan, 617-274-4052
iestepan@sarepta.com
Media Contact: Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com
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