- Elamipretide in Phase 3 development for
primary mitochondrial myopathy -
- If option is exercised, Alexion & Stealth
will co-develop and co-promote subcutaneous elamipretide in U.S.
& Alexion will receive exclusive ex-U.S. license -
- Stealth to receive initial payment of $30
million for equity investment, option fee and development funding,
with potential for additional option-related &
milestone-dependent payments if option is exercised -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Stealth
BioTherapeutics Corp (NASDAQ:MITO) today announced an agreement for
an option to co-develop and commercialize elamipretide for
mitochondrial diseases. Currently being evaluated in a Phase 3
study in people with primary mitochondrial myopathy (PMM) - a
genetic mitochondrial disease - elamipretide is a novel, potential
first-in-class therapy that targets mitochondrial dysfunction.
There are currently no therapies approved to treat PMM, which is
characterized by debilitating skeletal muscle weakness, chronic
fatigue and exercise intolerance. Under the terms of the agreement,
Alexion will have the opportunity to exercise the option following
the delivery of results from the Phase 3 study currently underway
in PMM.
This press release features multimedia. View
the full release here:
https://www.businesswire.com/news/home/20191010005160/en/
“Mitochondria play a critical role in normal organ function and,
when dysfunctional, can have devastating consequences on multiple
organ systems, leading to many serious diseases, such as primary
mitochondrial myopathy,” said John Orloff, M.D., Executive Vice
President and Head of Research & Development at Alexion. “Given
our strong existing relationships with neuromuscular specialists –
who play a critical role in treating PMM – we believe this is an
exciting potential opportunity to further expand our rare neurology
portfolio and look forward to the possibility of working with
Stealth to realize the promise of elamipretide for patients.”
“Our mission is to deliver therapies to patients suffering from
devastating mitochondrial diseases, and this partnership will
enhance our ability to rapidly deliver on that mission,” said
Stealth Chief Executive Officer Reenie McCarthy. “Together with
Alexion, which is widely recognized for its demonstrated ability to
bring important new therapies for rare diseases to children and
adults in need, we believe we can achieve synergies of execution
that will both expedite and increase patient access following
achievement of key upcoming clinical and regulatory milestones for
our PMM and Barth syndrome programs.”
Under the terms of the agreement, Alexion will receive an
exclusive option to partner with Stealth in the development of
subcutaneous elamipretide based on final results from the Phase 3
study currently underway in PMM. If Alexion chooses to exercise the
option, the companies will co-develop subcutaneous elamipretide in
the U.S. for PMM and Barth syndrome, as well as Leber’s hereditary
optic neuropathy (LHON), which is currently in earlier stage
clinical development. Upon commercialization, the agreement would
provide for a 50-50 co-promote between the two companies in the
U.S. and Alexion would receive exclusive rights to develop and
commercialize subcutaneous elamipretide outside the U.S. Alexion
will make initial payments to Stealth totaling $30 million,
including an option fee, an equity investment and development
funding. If the option is exercised, the agreement provides for
additional payments, including an option exercise fee, an
additional equity investment, development funding and potential
regulatory and commercial milestone payments. Stealth’s other
pipeline assets, including SBT-272, are not included in the
option.
Stealth will host a conference call and webcast at 8:30 a.m. ET
today to review this partnership and the potential opportunity for
elamipretide. To access the call please dial 866-939-3921
(domestic) and 678-302-3550 (international) and provide the
passcode 49106405. A live audio webcast of the call will be
available on the Investors & News section of Stealth’s website
at https://investor.stealthbt.com/. The archived webcast will be
available approximately two hours after the conference call and
will be available for 30 days following the call.
About Primary Mitochondrial Myopathy Primary
mitochondrial myopathy (PMM) affects patients afflicted with a
heterogenous group of genetic disorders whose disease predominantly
impairs skeletal muscle function, with many patients suffering from
lifelong functional impairment. PMM is among the most common forms
of mitochondrial disease; patients must have a genetically
confirmed diagnosis of primary mitochondrial disease plus a
predominantly myopathic clinical presentation to have PMM. Signs,
symptoms and disease severity vary significantly among patients,
but the most common symptoms include progressively debilitating
skeletal muscle weakness, chronic fatigue and exercise intolerance.
As a result, the diagnostic process can be challenging, requiring a
multi-disciplinary approach, most frequently involving
neuromuscular specialists and geneticists. There are currently no
approved treatments for PMM, and standard-of-care is supportive,
typically focusing on the specific symptoms experienced by each
individual patient.
About Elamipretide Elamipretide, a mitochondria-targeted
therapeutic, is in clinical development for a variety of diseases
caused by mitochondrial dysfunction. Elamipretide targets the inner
mitochondrial membrane where it associates with cardiolipin – the
signature phospholipid of the inner mitochondrial membrane, which
plays a role in many mitochondrial processes, including respiration
and energy conversion. This elamipretide-cardiolipin association
has been shown to normalize the structure of the inner
mitochondrial membrane, thereby improving mitochondrial function.
In preclinical and clinical studies, elamipretide was shown to
increase mitochondrial respiration, improve the electron transport
chain function and ATP production, and reduce formation of
pathogenic reactive oxygen species levels. Functional benefit is
believed to be achieved through improvement of ATP production and
interruption and potential reversal of damaging oxidative
stress.
Elamipretide is being investigated in late-stage clinical
studies in primary mitochondrial myopathy (PMM) and Barth syndrome
as well as in earlier stage clinical studies in Leber’s hereditary
optic neuropathy (LHON) and geographic atrophy associated with dry
age-related macular degeneration (GA). Elamipretide has received
Fast Track and Orphan Drug designations for PMM, Barth syndrome and
LHON, as well as Fast Track designation for GA.
About Alexion Alexion is a global biopharmaceutical
company focused on serving patients and families affected by rare
diseases through the discovery, development and commercialization
of life-changing therapies. As the global leader in complement
biology and inhibition for more than 20 years, Alexion has
developed and commercializes two approved complement inhibitors to
treat patients with paroxysmal nocturnal hemoglobinuria (PNH) as
well as the first and only approved complement inhibitor to treat
atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine
receptor (AchR) antibody-positive generalized myasthenia gravis
(gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion
also has two highly innovative enzyme replacement therapies for
patients with life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency
(LAL-D). In addition, the company is developing several
mid-to-late-stage therapies, including a second complement
inhibitor, a copper-binding agent for Wilson disease and an
anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G
(IgG)-mediated diseases as well as several early-stage therapies,
including one for light chain (AL) amyloidosis and a second
anti-FcRn therapy. Alexion focuses its research efforts on novel
molecules and targets in the complement cascade and its development
efforts on the core therapeutic areas of hematology, nephrology,
neurology, and metabolic disorders. Alexion has been named to the
Forbes’ list of the World’s Most Innovative Companies seven years
in a row and is headquartered in Boston, Massachusetts’ Innovation
District. The company also has offices around the globe and serves
patients in more than 50 countries. This press release and further
information about Alexion can be found at: www.Alexion.com.
[ALXN-G]
About Stealth Stealth is a clinical-stage biotechnology
company focused on improving the lives of patients suffering from
diseases involving mitochondrial dysfunction through the discovery,
development and commercialization of novel mitochondrial medicines.
Mitochondria, found in nearly every cell in the body, are the
body's main source of energy production and are critical for normal
organ function. Dysfunctional mitochondria characterize a number of
rare genetic diseases, collectively known as primary mitochondrial
diseases, and are also involved in many common age-related
diseases. Stealth believes its lead product candidate,
elamipretide, has the potential to treat both rare genetic and
common age-related mitochondrial diseases. Stealth is studying
elamipretide in the following primary mitochondrial diseases:
primary mitochondrial myopathy, Barth syndrome and Leber's
hereditary optic neuropathy. Stealth is also studying elamipretide
in geographic atrophy associated with dry age-related macular
degeneration. Stealth’s other pipeline candidates include SBT-272,
being evaluated for rare neurodegenerative disease indications, and
SBT-20 and SBT-259, being evaluated for rare peripheral
neuropathies. Stealth has optimized its discovery platform to
identify novel mitochondria-targeted compounds, which may be
nominated as therapeutic product candidates or utilized as
scaffolds to deliver other compounds to mitochondria. Stealth has
assembled a highly experienced management team, board of directors
and group of scientific advisors to achieve its mission of leading
mitochondrial medicine.
Forward-Looking Statements This press release includes
forward-looking statements, including statements related to the
therapeutic benefits of elamipretide; the potential of elamipretide
to target mitochondrial dysfunction (and to serve as a potential
first-in-class therapy that targets mitochondrial dysfunction); the
potential of elamipretide as a treatment for PMM and other
indications; the agreement is a potential opportunity to further
expand Alexion’s rare neurology portfolio and offers the
possibility of working with Stealth to realize the promise of
elamipretide for patients; Stealth has optimized its discovery
platform to identify novel mitochondria-targeted compounds, which
may be nominated as therapeutic product candidates or utilized as
scaffolds to deliver other compounds to mitochondria; Alexion’s
ability to use its relationship with neuromuscular specialists to
bring elamipretide to patients suffering with PMM and other
indications; the potential benefits of the collaboration, including
the realization of synergies between Alexion and Stealth to
accelerate the clinical development and commercial launch and
patient access to elamipretide for children and adults; and the
achievement of key upcoming clinical and regulatory milestones for
Stealth’s PMM and Barth syndrome programs. Such forward-looking
statements are subject to risks and uncertainties that could cause
actual results to differ materially from those expressed or implied
in such statements, including the process by which an early stage
product such as elamipretide could potentially lead to an approved
product for the treatment of PMM is long and subject to highly
significant risks, including for example, decisions of regulatory
authorities regarding the adequacy of our research, marketing
approval or material limitations on the marketing of our products,
delays, interruptions or failures in manufacture and supply,
failure to satisfactorily address matters raised by the U.S. Food
and Drug Administration and other regulatory agencies, the
possibility that results of clinical trials are not predictive of
safety and efficacy results in broader patient populations, the
possibility that clinical trials could be delayed, the risk that
anticipated regulatory filings are delayed, the risk that estimates
regarding the number of patients with PMM are inaccurate; the
collaboration may not achieve the anticipated benefits for Alexion,
Stealth and for patients; the anticipated benefits of elamipretide
may not be realized even if approved for sale; patients, physicians
and payers may not accept elamipretide as a treatment for the
indications being pursued; the discovery platform under development
may not achieve the anticipated benefits due to technological
challenges or otherwise; the anticipated contributions of the
parties to the agreement (including the expected synergies) may not
come to fruition due to technical and commercial limitations and
other factors; and a variety of other risks set forth from time to
time in Alexion's filings with the SEC, including but not limited
to the risks discussed in Alexion's Quarterly Report on Form 10-Q
for the period ended June 30, 2019 and in Alexion’s other filings
with the SEC, and in Stealth’s filings with the SEC, including but
not limited to the risks discussed in Stealth’s Annual Report on
Form 20-F for the period ended December 31, 2018. Alexion and
Stealth disclaim any obligation to update any of these
forward-looking statements to reflect events or circumstances after
the date hereof, except when a duty arises under law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20191010005160/en/
Alexion: Media Megan Goulart, 857-338-8634 Senior
Director, Corporate Communications Investors Susan
Altschuller, Ph.D., 857-338-8788 Vice President, Investor Relations
Stealth: Media Lindsey Colegrove, dna Communications
212-537-8771, Media@StealthBT.com Investors Lauren Stival,
Stern Investor Relations 212-362-1200, IR@StealthBT.com
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