- PHOENIX Phase 3 trial topline results on track for mid-2024
and will inform regulatory next steps in the EU - Final decision
from the European Commission expected by the end of 2023
Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the
“Company”) today announced that the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency
(EMA) confirmed its initial negative opinion on the Marketing
Authorisation Application (MAA) for AMX0035 (sodium phenylbutyrate
and ursodoxicoltaurine [also known as taurursodiol]), under the
trade name ALBRIOZA®, for the treatment of amyotrophic lateral
sclerosis (ALS) in the European Union (EU). The decision follows
the conclusion of the CHMP’s formal re-examination procedure of an
initial negative opinion adopted in June 2023.
ALBRIOZA was approved with conditions by Health Canada in June
2022 and granted a full approval by the U.S. Food and Drug
Administration (FDA) under the trade name RELYVRIO® in September
2022.
The European Organization for Professionals and Patients with
ALS (EUpALS) Patients and Carers Expert Board said in a statement,
“We are disappointed to learn of this outcome, as it is a further
setback for the more than 30,000 people living with ALS and their
loved ones in Europe who have not seen therapeutic progress for
this devastating disease in over 25 years.”
“We share the frustration felt by the European ALS community,
who has no time to wait for new, safe, and effective treatment
options,” said Stéphanie Hoffmann-Gendebien, General Manager and
Head of EMEA at Amylyx. “We remain committed to exploring all
potential paths forward in support of the Company’s goal of getting
AMX0035 to people living with ALS in the EU as quickly as
possible.”
“AMX0035 is the first and only drug to show an effect on both
function and survival in the same trial. Since the medication’s
approval with conditions in Canada and full approval in the U.S.,
thousands of people have been prescribed AMX0035 in North America.
ALS has no geographical boundaries, and we are working with urgency
toward providing timely, broad, and sustainable access to AMX0035
for eligible people living with ALS who may benefit,” said Joshua
Cohen and Justin Klee, Co-CEOs of Amylyx.
Amylyx continues to focus on the completion of the PHOENIX Phase
3 clinical trial, which was initiated prior to its Marketing
Authorisation Application submission and will provide additional
data on the efficacy and safety profile of ALBRIOZA in people
living with ALS. If PHOENIX is supportive, Amylyx plans to seek
approval in the EU as quickly as possible. Topline results are
anticipated in mid-2024.
PHOENIX is a 48-week, randomized placebo-controlled global Phase
3 clinical trial further evaluating the safety and efficacy of
AMX0035 for the treatment of ALS. The study enrolled 664
participants living with ALS across 69 sites in either Europe or
the U.S., the majority of which are members of the NEALS or TRICALS
consortia. The design of PHOENIX was informed by the results of the
Phase 2 CENTAUR clinical trial of AMX0035, which met its
prespecified primary outcome and demonstrated a statistically
significant benefit in function as well as an observed benefit on
survival in a longer-term post hoc analysis. Overall, reported
rates of adverse events and discontinuations in CENTAUR were
similar between AMX0035 and placebo groups during the 24-week
randomized phase; however, gastrointestinal events occurred with
greater frequency (≥2%) in the AMX0035 group. More information on
the PHOENIX trial can be found at
https://classic.clinicaltrials.gov/ct2/show/NCT05021536 or
https://www.clinicaltrialsregister.eu/ctr-search/search, EudraCT
Number: 2021-000250-26.
The CHMP opinion will be forwarded to the European Commission,
who will adopt the final decision on this application. A decision
is anticipated by the end of 2023.
About RELYVRIO®/ ALBRIOZA™ / ALBRIOZA® / AMX0035
RELYVRIO®, an oral, fixed-dose combination of sodium
phenylbutyrate and taurursodiol (known as ursodoxicoltaurine
outside of the U.S.), is approved to treat amyotrophic lateral
sclerosis (ALS) in adults in the U.S. and approved with conditions
as ALBRIOZA™ for the treatment of ALS in Canada. AMX0035 is being
studied for the potential treatment of other neurodegenerative
diseases, and Amylyx is exploring its treatment in other
populations and regions. The formulation of RELYVRIO, ALBRIOZA, and
AMX0035 are identical.
About the CENTAUR Trial
CENTAUR was a multicenter Phase 2 clinical trial in 137
participants with ALS encompassing a 6-month randomized
placebo-controlled phase and an open-label extension (OLE)
long-term follow-up phase. The trial met its primary efficacy
endpoint.
Detailed safety and functional efficacy data from CENTAUR were
published in the New England Journal of Medicine. Data from
additional analyses from the CENTAUR trial were published in Muscle
& Nerve in 2020 and 2022, and the Journal of Neurology,
Neurosurgery, and Psychiatry in 2022.
The CENTAUR trial was funded, in part, by the ALS ACT grant and
the ALS Ice Bucket Challenge, and was supported by The ALS
Association, ALS Finding a Cure (a program of The Leandro P.
Rizzuto Foundation), the Northeast ALS Consortium, and the Sean M.
Healey & AMG Center for ALS at Mass General.
About ALS
ALS is a relentlessly progressive and fatal neurodegenerative
disorder caused by motor neuron death in the brain and spinal cord.
Motor neuron loss in ALS leads to deteriorating muscle function,
the inability to move and speak, respiratory paralysis and
eventually, death. More than 90% of people with ALS have sporadic
disease, showing no clear family history. ALS affects approximately
29,000 people in the U.S. and more than 30,000 people are estimated
to be living with ALS in Europe (European Union and United
Kingdom). People living with ALS have a median survival of
approximately two years from diagnosis.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and
creating more moments for the neurodegenerative disease community
through the discovery and development of innovative new treatments.
Amylyx is headquartered in Cambridge, Massachusetts and has
operations in Canada and EMEA. For more information, visit
amylyx.eu and follow us on LinkedIn and X, formerly known as
Twitter. For investors, please visit investors.amylyx.com.
Forward-Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995, as amended. Because such statements are subject to risks
and uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. Such
statements include, but are not limited to, statements regarding
the timing and results of the global Phase 3 PHOENIX trial; the
potential approval of AMX0035 for the treatment of ALS in countries
other than the United States and Canada; the plans to seek approval
in the future in Europe; the potential of AMX0035 as a treatment
for ALS and the Company’s plans to explore the use of AMX0035 for
other neurodegenerative diseases; and expectations regarding our
longer-term strategy. Any forward-looking statements in this press
release are based on management’s current expectations of future
events and are subject to a number of risks and uncertainties that
could cause actual results to differ materially and adversely from
those set forth in or implied by such forward-looking statements.
Risks that contribute to the uncertain nature of the
forward-looking statements include: Amylyx’ ability to fund
operations, the success, cost, and timing of Amylyx’ program
development activities, Amylyx’ ability to execute on its
commercial and regulatory strategy, regulatory developments,
expectations regarding the outcome of the re-examination of AMX0035
for the treatment of ALS in the EU, and Amylyx’ reliance on third
parties, including to conduct clinical trials and manufacture
products, and the effect of global economic uncertainty and
financial market volatility caused by economic effects of rising
inflation and interest rates, the COVID-19 pandemic, geopolitical
instability, changes in international trade relationships and
military conflicts, as well as the risks and uncertainties set
forth in Amylyx’ United States Securities and Exchange Commission
(SEC) filings, including Amylyx’ Quarterly Report on Form 10-Q for
the quarter ended June 30, 2023, and subsequent filings with the
SEC. All forward-looking statements contained in this press release
speak only as of the date on which they were made. Subject to any
obligations under applicable law, Amylyx undertakes no obligation
to update such statements to reflect events that occur or
circumstances that exist after the date on which they were
made.
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version on businesswire.com: https://www.businesswire.com/news/home/20231012969323/en/
Media Amylyx Media Team (857) 799-7274
amylyxmediateam@amylyx.com Investors Lindsey Allen Amylyx
Pharmaceuticals, Inc. (857) 320-6244 Investors@amylyx.com
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