Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy
12 Giugno 2024 - 10:30PM
Business Wire
Pfizer Inc. (NYSE: PFE) today announced that CIFFREO, a Phase 3
global, multicenter, randomized, double-blind, placebo-controlled
study evaluating the investigational mini-dystrophin gene therapy,
fordadistrogene movaparvovec, in ambulatory patients with Duchenne
muscular dystrophy (DMD) did not meet its primary endpoint of
improvement in motor function among boys 4 to 7 years of age
treated with the gene therapy compared to placebo. The primary
endpoint in the final analysis was assessed by change in the North
Star Ambulatory Assessment (NSAA) at one year after treatment. Key
secondary endpoints, including 10-meter run/walk velocity and time
to rise from floor velocity, also did not show a significant
difference between participants treated with fordadistrogene
movaparvovec and placebo.
The overall safety profile of fordadistrogene movaparvovec in
the CIFFREO trial was manageable, with mostly mild to moderate
adverse events, and treatment-related serious adverse events
generally responding to clinical management.
“We are extremely disappointed that these results did not
demonstrate the relative improvement in motor function that we had
hoped. We plan to share more detailed results from the study at
upcoming medical and patient advocacy meetings, with the goal of
ensuring that learnings from this trial can help improve future
clinical research and development of treatment options that can
improve care for boys living with Duchenne muscular dystrophy,”
said Dan Levy, MD, PhD, Development Head for Duchenne muscular
dystrophy, Pfizer. “We are grateful for the boys, their families,
advocates, and the investigators who have participated in this
research and the continuing effort to advance treatment options for
this debilitating disease.”
Pfizer will continue to closely monitor all participants
enrolled in the study and is evaluating appropriate next steps for
the program.
About the Fordadistrogene Movaparvovec Clinical
Program
CIFFREO is a Phase 3 global, multi-center, randomized,
double-blind, placebo-controlled study to assess the safety and
efficacy of fordadistrogene movaparvovec investigational gene
therapy in ambulatory male participants, aged 4 to 7 years, with a
genetic diagnosis of DMD who are on a stable daily regimen of
glucocorticoids. The primary endpoint of the study is a change from
baseline to one year in the North Star Ambulatory Assessment (NSAA)
total score. For more information, visit ciffreoduchennetrial.com
or clinicaltrials.gov.
The CIFFREO study is currently on a dosing pause due to a fatal
serious adverse event in the Phase 2 DAYLIGHT trial (NCT05429372).
DAYLIGHT is a study that is evaluating the safety and tolerability
of fordadistrogene movaparvovec in participants 2 years to 3 years
of age with DMD. Pfizer is actively working to gather additional
information on the event to understand the potential cause.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a serious genetic disease
characterized by progressive muscle degeneration and weakness.
Symptoms usually manifest in early childhood between the ages of 3
and 5. The disease primarily affects boys. Muscle weakness can
begin as early as age 3, first affecting the muscles of the hips,
pelvic area, thighs, and shoulders, and later the skeletal
(voluntary) muscles in the arms, legs, and trunk. By their early
teens, patients typically lose their ability to walk and the heart
and respiratory muscles are also affected, ultimately resulting in
premature death. DMD is the most common form of muscular dystrophy
worldwide with an incidence of 1 in every 5,000 live male
births.1
About Pfizer: Breakthroughs That Change Patients’
Lives
At Pfizer, we apply science and our global resources to bring
therapies to people that extend and significantly improve their
lives. We strive to set the standard for quality, safety and value
in the discovery, development and manufacture of health care
products, including innovative medicines and vaccines. Every day,
Pfizer colleagues work across developed and emerging markets to
advance wellness, prevention, treatments and cures that challenge
the most feared diseases of our time. Consistent with our
responsibility as one of the world's premier innovative
biopharmaceutical companies, we collaborate with health care
providers, governments and local communities to support and expand
access to reliable, affordable health care around the world. For
175 years, we have worked to make a difference for all who rely on
us. We routinely post information that may be important to
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more, please visit us on www.Pfizer.com and follow us on X at
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Disclosure Notice
The information contained in this release is as of June 12,
2024. Pfizer assumes no obligation to update forward-looking
statements contained in this release as the result of new
information or future events or developments.
This release contains forward-looking information about
fordadistrogene movaparvovec, an investigational mini-dystrophin
gene therapy for Duchenne muscular dystrophy, topline results from
the Phase 3 CIFFREO study and plans to share more detailed results
from the study at upcoming medical and patient advocacy meetings
that involve substantial risks and uncertainties that could cause
actual results to differ materially from those expressed or implied
by such statements. Risks and uncertainties include, among other
things, the uncertainties inherent in research and development;
uncertainties related to further analysis of data from the Phase 3
CIFFREO study, including the sharing of more detailed results from
the study at upcoming medical and patient advocacy meetings;
uncertainties regarding the commercial success of Pfizer’s gene
therapy portfolio; uncertainties regarding the impact of COVID-19
on Pfizer’s business, operations and financial results; and
competitive developments.
A further description of risks and uncertainties can be found in
Pfizer’s Annual Report on Form 10-K for the fiscal year ended
December 31, 2023, and in its subsequent reports on Form 10-Q,
including in the sections thereof captioned “Risk Factors” and
“Forward-Looking Information and Factors That May Affect Future
Results”, as well as in its subsequent reports on Form 8-K, all of
which are filed with the U.S. Securities and Exchange Commission
and available at www.sec.gov and www.pfizer.com.
1 Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S,
Trifirò G. Global epidemiology of Duchenne muscular dystrophy: an
updated systematic review and meta-analysis. Orphanet J Rare Dis.
2020;15(1):141.
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