- Study met primary endpoint of change from baseline in body
weight for ponsegromab compared to placebo across all ponsegromab
doses tested, reaching 5.6% mean increase at the highest dose
evaluated at 12 weeks; ponsegromab was generally considered safe
and well-tolerated at all dose levelsi
- At the highest dose evaluated, improvements were seen from
baseline in appetite and cachexia symptoms, physical activity, and
muscle massi
- Based on positive Phase 2 results, registration-enabling
studies will start in 2025
Pfizer Inc. (NYSE: PFE) today announced its Phase 2 study of
ponsegromab, a monoclonal antibody directed against growth
differentiation factor-15 (GDF-15), met its primary endpoint of
change from baseline in body weight compared to placebo in people
with cancer cachexia and elevated levels of GDF-15. Cachexia is a
common, life-threatening wasting condition that can significantly
impact quality of life in affected patients with cancer.ii,iii
Results will be presented today as a late-breaking Proffered Paper
Presentation (LBA82) at the European Society for Medical Oncology
(ESMO) 2024 Congress and have simultaneously been published in The
New England Journal of Medicine (NEJM).
“Cachexia is a common condition in cancer patients, associated
with weight loss, functional decline, and ultimately poor outcomes.
Despite the number of people suffering from cachexia, there are no
available options for us to help treat patients,” said Jeffrey
Crawford, M.D., George Barth Geller Professor for Research at Duke
Cancer Institute, and principal investigator. “This study showed us
those who received ponsegromab had improvement in body weight,
muscle mass, quality of life, and physical function. These findings
offer hope that a breakthrough targeted treatment is potentially on
the horizon for our patients.”
The Phase 2 study included 187 participants with non-small cell
lung cancer, pancreatic cancer, or colorectal cancer. Ponsegromab
demonstrated significant and robust increases in body weight after
12 weeks across all doses: 2.02% (95% confidence interval (CI),
-0.97 to 5.01%) in the 100 mg treatment group, 3.48% (95% CI, 0.54
to 6.42%) in the 200 mg group, and 5.61% (95% CI, 2.56 to 8.67%) in
the 400 mg group, compared to placebo. Improvements across multiple
domains of the cachexia phenotype were observed in the 400 mg group
including in measures of appetite and cachexia symptoms, physical
activity and skeletal muscle index. No clinically significant
adverse trends were noted with ponsegromab administration.
Treatment related adverse events occurred in 8.9% of patients
taking placebo and 7.7% of patients taking ponsegromab.i
“Discovered and developed in-house at Pfizer, ponsegromab
represents our ability to translate deep scientific expertise into
patient benefit,” said Charlotte Allerton, Head of Discovery and
Early Development, Pfizer. “These results provide strong evidence
that we have unlocked a mechanism to interrupt a critical driver of
cachexia, GDF-15, which has the potential to impact patients with
cancer cachexia and other life-threatening conditions. We look
forward to advancing this program as part of our broader
cardiometabolic portfolio to address weight management across the
spectrum of patient need.”
Based on these positive results, Pfizer is discussing late-stage
development plans with regulators with the goal of starting
registration-enabling studies in 2025. Ponsegromab is also being
investigated in a Phase 2 study in patients with heart failure (HF)
and elevated serum GDF-15 concentrations (NCT05492500).
About the Phase 2 Trial in Cancer Cachexia The primary
objective of the Phase 2 study (NCT05546476) is to assess the
effect of ponsegromab on body weight in patients with cancer
(non-small cell lung cancer, pancreatic cancer or colorectal
cancer), cachexia and elevated serum GDF-15 concentrations
(>1500 pg/mL). Secondary and
exploratory endpoints objectives included measures such as: Change
from baseline in appetite and cachexia symptoms, digital measures
of physical activity, and changes in lumbar skeletal muscle index
(LSMI). Patients (n=187) received either ponsegromab (100 mg, 200
mg or 400 mg) or placebo once every four weeks subcutaneously for
12 weeks.
About Cachexia Cachexia is a complex, disabling, and
life-threatening metabolic condition that is estimated to affect
about 9 million people worldwide.ii Symptoms such as weight and
muscle loss can reduce patients’ ability to tolerate treatment for
their underlying chronic diseases, such as cancer and heart
failure, and can severely impact quality of life.ii,iii In cancer,
cachexia can diminish the efficacy of cancer treatments and is
thought to contribute to decreased survival rates and may cause up
to 30% of cancer-related deaths.iv Despite its severity, there are
no FDA-approved treatments for cachexia.iii
About Ponsegromab Ponsegromab is an investigational
monoclonal antibody designed to treat cachexia by targeting GDF-15.
Prior Phase 1b data in participants with cancer cachexia
demonstrated proof-of-mechanism for ponsegromab with robust
suppression of unbound circulating GDF15 levels observed. The
results showed encouraging signals of efficacy that included
increases in body weight and encouraging improvements in patient
reported outcomes.
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
investors on our website at www.Pfizer.com. In addition, to learn
more, please visit us on www.Pfizer.com and follow us on X at
@Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook
at Facebook.com/Pfizer.
Disclosure Notice The information contained in this
release is as of September 14, 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
ponsegromab, an investigational monoclonal antibody designed to
treat cachexia, including its potential benefits and late-stage
development planning, and Pfizer’s cardiometabolic portfolio, 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,
including the ability to meet anticipated clinical endpoints,
commencement and/or completion dates for our clinical trials,
regulatory submission dates, regulatory approval dates and/or
launch dates, as well as the possibility of unfavorable new
clinical data and further analyses of existing clinical data,
including results from the Phase 2 study of ponsegromab in patients
with heart failure; the risk that clinical trial data are subject
to differing interpretations and assessments by regulatory
authorities; whether regulatory authorities will be satisfied with
the design of and results from our clinical studies; whether and
when drug applications may be filed in any jurisdictions for any
potential indication for ponsegromab or any other cardiometabolic
product candidates; whether and when any such applications that may
be filed for ponsegromab or any other such product candidates may
be approved by regulatory authorities, which will depend on myriad
factors, including making a determination as to whether the
product's benefits outweigh its known risks and determination of
the product's efficacy and, if approved, whether ponsegromab or any
such other product candidates will be commercially successful;
decisions by regulatory authorities impacting labeling,
manufacturing processes, safety and/or other matters that could
affect the availability or commercial potential of ponsegromab or
any such other product candidates; uncertainties regarding the
impact of COVID-19 on our 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.
_________________________ i Groarke J, et al. Ponsegromab for
the Treatment of Cancer Cachexia. The New England Journal of
Medicine. https://doi.org/10.1056/NEJMoa2409515. Accessed September
14, 2024. ii Cleveland Clinic. Cachexia (Wasting Syndrome).
Cachexia (Wasting Syndrome): Symptoms & Treatment
(clevelandclinic.org). Accessed September 3, 2024. iii Lisa Martin,
Michael B. Sawyer, Cancer Cachexia: Emerging Preclinical Evidence
and the Pathway Forward to Clinical Trials, JNCI: Journal of the
National Cancer Institute, Volume 107, Issue 12, December 2015,
https://doi.org/10.1093/jnci/djv322 iv National Cancer Institute.
Cancer Cachexia: After Years of No Advances, Progress Looks
Possible. Treating Cancer Cachexia: Progress Looks Possible - NCI.
Accessed September 3, 2024.
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