Amylyx Pharmaceuticals to Announce Plans for an Open Label Extension (OLE) Phase for Global PHOENIX Trial of AMX0035 in ALS at the 17th International Congress on Neuromuscular Diseases
July 06 2022 - 9:00AM
Business Wire
Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the
“Company”) today announced a poster on the ongoing international
Phase 3 PHOENIX trial (study A35-004, NCT05021536) of AMX0035
(sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as
ursodoxicoltaurine]) in people living with amyotrophic lateral
sclerosis (ALS) will be presented on Saturday, July 9, at the 17th
International Congress on Neuromuscular Diseases (ICNMD 2022) in
Brussels, Belgium. The poster will include an overview of the
PHOENIX trial design and methodology, as well as an update on a
planned open-label extension (OLE) phase for PHOENIX.
“The Phase 3 PHOENIX trial is enrolling a larger and broader
group of people living with ALS than were enrolled in the Phase 2
CENTAUR trial, helping us to generate additional data on potential
treatment effects of AMX0035,” said Leonard H. van den Berg, M.D.,
Ph.D., Professor of Neurology at UMC Utrecht in the Netherlands and
Chairman of the Treatment Research Initiative to Cure ALS
(TRICALS), a large European trial network dedicated to finding
effective treatments for ALS. “We look forward to sharing our plans
for the OLE phase at ICNMD.”
Presentation Details:
Title: International Phase 3 Trial Evaluating Sodium
Phenylbutyrate and Taurursodiol in Amyotrophic Lateral Sclerosis
(PHOENIX) Poster Number: 11 Date:
Saturday, July 9, 2022 Time: 12:45-2:15 p.m. CEST
About AMX0035
AMX0035 (sodium phenylbutyrate and taurursodiol) is an oral
fixed-dose medication approved with conditions as ALBRIOZA™ to
treat amyotrophic lateral sclerosis (ALS) in Canada and with
marketing applications pending in the United States and European
Union. The combination of sodium phenylbutyrate and taurursodiol
may reduce neuronal cell death, hypothesized to occur by
simultaneously mitigating endoplasmic reticulum (ER) stress and
mitochondrial dysfunction. AMX0035 is also being explored for the
potential treatment of other neurodegenerative diseases.
About the PHOENIX Trial
The Phase 3 PHOENIX clinical trial (NCT05021536) is a 48-week,
randomized placebo-controlled global clinical trial further
evaluating the safety and efficacy of AMX0035 (sodium
phenylbutyrate and taurursodiol) for the treatment of ALS. The
primary efficacy outcome of the trial will be a composite measure
of survival and Amyotrophic Lateral Sclerosis Functional Rating
Scale-Revised (ALSFRS-R) total score progression over 48 weeks and
survival and tolerability over 48 weeks. Secondary endpoints
include change in slow vital capacity (SVC), measured both at home
using a self-administered spirometer to support virtual data
collection and at clinic sites using standard spirometry, quality
of life patient-reported outcome assessments, ventilation-free
survival rates and other measures. More information on the PHOENIX
trial can be found at www.clinicaltrials.gov and
eudract.ema.europa.eu.
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 long-term follow-up
phase. The trial met its primary efficacy endpoint of reducing
functional decline as measured by the Amyotrophic Lateral Sclerosis
Functional Rating Scale – Revised (ALSFRS-R).
Overall, reported rates of adverse events and discontinuations
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. 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 Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and
creating more moments for the neurodegenerative 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.com and follow
us on LinkedIn and 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 potential regulatory approval of AMX0035 and the potential of
AMX0035 or other future therapeutic candidates as a treatment for
ALS and other neurodegenerative diseases. Any forward-looking
statements in this statement 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: the success,
cost, and timing of Amylyx’ program development activities, Amylyx’
ability to execute on its strategy, regulatory developments,
expectations regarding the timing of regulatory review of AMX0035,
Amylyx’ ability to fund operations, and the impact that the ongoing
COVID-19 pandemic will have on Amylyx’ operations, as well as those
risks and uncertainties set forth in Amylyx’ United States
Securities and Exchange Commission (SEC) filings, including Amylyx’
Annual Report on Form 10-K for the year ended December 31, 2021,
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. 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/20220706005028/en/
Media Becky Gohsler Finn Partners (646) 307-6307
amylyxmediateam@amylyx.com Investors Lindsey Allen Amylyx
Pharmaceuticals, Inc. (857) 320-6244 Investors@amylyx.com
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