NASHVILLE, Tenn., Feb. 4, 2025
/PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq:
CPIX), a specialty pharmaceutical company with development
efforts focused on new products for rare diseases, today announced
positive top-line results from its Phase 2 FIGHT DMD trial. The
study evaluated ifetroban, a novel oral therapy for Duchenne
muscular dystrophy (DMD) heart disease – the leading cause of death
in DMD patients. It marks a breakthrough for these patients, as
it's the first successful Phase 2 study specifically targeting the
cardiac complications of their condition.
DMD is a rare and incurable pediatric disease caused by
mutations in the gene encoding dystrophin, a protein critical for
muscle function, including the heart. Patients with DMD slowly lose
muscle function, resulting in the inability to walk, difficulty
breathing and heart failure. While current treatments can help
manage some DMD symptoms, there are no approved therapies
specifically targeting DMD-related heart disease, highlighting a
critical unmet medical need.
A previous study conducted at Vanderbilt
University Medical Center demonstrated that ifetroban is
protective against cardiomyopathy in several preclinical models of
muscular dystrophy. The results of that study were published in the
Journal of the American Heart Association (West 2019). Based
on those promising results, Cumberland Pharmaceuticals became the
first recipient of an FDA Office of Orphan Products Development
clinical trial grant for DMD, funding the development of this Phase
2 clinical trial.
"These results represent a significant milestone in DMD
cardiomyopathy," said Larry W.
Markham, MD, Professor of Pediatrics and Medicine,
Indiana University School of Medicine,
Division Chief of Pediatric Cardiology at Riley Children's Hospital
and Principal Investigator of the FIGHT DMD trial. "We are seeing
evidence that there is an opportunity to potentially alter the
course of heart disease in DMD patients. The improvement in cardiac
function observed with ifetroban, particularly in the high-dose
group, offers hope for these patients and their families."
The FIGHT DMD trial (NCT03340675), is a 12-month, double-blind,
randomized, placebo-controlled study evaluating ifetroban, an oral
thromboxane receptor antagonist. The trial enrolled 41 DMD patients
who received either low dose ifetroban (150 mg per day), high dose
ifetroban (300 mg per day), or placebo. The study's primary
endpoint was an improvement in the heart's left ventricular
ejection fractions (LVEF).
Key findings include:
- High dose ifetroban treatment resulted in an overall 3.3%
improvement in LVEF.
- The high dose ifetroban group showed an increase in 1.8% in
LVEF, while the study placebo group showed an expected decline in
LVEF of 1.5%
- When compared with propensity matched natural history controls,
the difference was even more pronounced, with the high dose
treatment providing a significant 5.4% overall improvement in
LVEF, as the control patients experienced a 3.6% decline in
LVEF
- Both doses of ifetroban were well-tolerated, with no
serious drug-related events.
"The cardiac imaging data from this trial is compelling,"
noted Jonathan Soslow, MD, Professor
of Pediatrics, Vanderbilt University,
pediatric cardiologist and cardiac imaging expert. "The
preservation and even improvement in cardiac function seen with
ifetroban treatment stands in stark contrast to the expected
decline we typically observe in untreated DMD patients."
"This trial represents hope for our Duchenne community," said
Pat Furlong, Founding President and
CEO of Parent Project Muscular Dystrophy. "Heart disease remains
one of the most devastating aspects of Duchenne, and these results
suggest we may finally have a therapeutic option that could make a
meaningful difference in the lives of patients and
families."
"These impressive results represent a pivotal moment for
Cumberland Pharmaceuticals and, more importantly, for the DMD
community," said A.J. Kazimi, Chief Executive Officer of Cumberland
Pharmaceuticals. "As the first company to receive FDA Orphan
Products Development funding for a DMD clinical trial, we're
honored to be advancing a potential breakthrough therapy for
DMD-related heart disease. These results validate our commitment to
developing innovative treatments for rare diseases and underscore
the importance of collaborative partnerships between industry,
academia, and regulatory agencies in addressing critical unmet
medical needs."
Ifetroban is a once-daily oral medication that works by blocking
the thromboxane receptor, which plays a key role in inflammation
and fibrosis. The drug has received both Orphan Drug
Designation and Rare Pediatric Disease Designation from
the FDA, highlighting its potential significance in treating this
devastating condition. If approved, ifetroban would be the first
therapy specifically indicated for DMD-related heart disease.
Cumberland has secured a
growing portfolio of patents with claims associated with the
product for this DMD indication. Next steps include further data
analysis and completion of a full study report in preparation for
an end of Phase 2 meeting with the FDA to determine next steps
associated with the product's development and
commercialization.
More information regarding the FIGHT DMD trial can be found
here: www.fightdmdtrial.com
References:
Soslow JH, Xu M, Slaughter JC, et al. Cardiovascular Measures of
All-Cause Mortality in Duchenne Muscular Dystrophy. Circ Heart
Fail. 2023 Aug;16(8):e010040. doi:
10.1161/CIRCHEARTFAILURE.122.010040. Epub 2023 Jun 8. PMID: 37288563; PMCID: PMC10524475.
West JD, Galindo CL, Kim K, et al. Antagonism of the
thromboxane-prostanoid receptor as a potential therapy for
cardiomyopathy of muscular dystrophy. J Am Heart Assoc.
2019;8(21):e011902. doi: 10.1161/JAHA.118.011902.
About Cumberland Pharmaceuticals
Cumberland Pharmaceuticals Inc. is the largest
biopharmaceutical company founded and headquartered in Tennessee and is focused on providing unique
products that improve the quality of patient care. The company
develops, acquires, and commercializes products for the hospital
acute care, gastroenterology and oncology market
segments.
The company's portfolio of FDA-approved brands includes:
- Acetadote® (acetylcysteine)
injection, for the treatment of acetaminophen poisoning;
- Caldolor® (ibuprofen) injection,
for the treatment of pain and fever;
- Kristalose® (lactulose) oral, a
prescription laxative, for the treatment of constipation;
- Sancuso® (granisetron) transdermal,
for the prevention of nausea and vomiting in patients receiving
certain types of chemotherapy treatment;
- Vaprisol® (conivaptan) injection,
to raise serum sodium levels in hospitalized patients with
euvolemic and hypervolemic hyponatremia; and
- Vibativ® (telavancin) injection,
for the treatment of certain serious bacterial infections including
hospital-acquired and ventilator-associated bacterial pneumonia, as
well as complicated skin and skin structure
infections.
In addition to this Duchenne muscular dystrophy program, the
company also has Phase 2 clinical studies underway evaluating its
ifetroban product candidate in patients with Systemic Sclerosis and
Idiopathic Pulmonary Fibrosis.
For more information on Cumberland's approved products, including full
prescribing information, please visit the individual product
websites, which can be found on the company's website:
www.cumberlandpharma.com.
Forward-Looking Statements
This press release contains forward-looking statements, which
are subject to certain risks and reflect Cumberland's current views on future events
based on what it believes are reasonable assumptions. No assurance
can be given that these events will occur. Forward-looking
statements include, among other things, statements regarding the
company's intent, belief or expectations, and can be identified by
the use of terminology such as "may," "will," "expect," "believe,"
"intend," "plan," "estimate," "should," "seek," "anticipate," "look
forward" and other comparable terms or the negative thereof. As
with any business, all phases of Cumberland's operations are subject to factors
outside of its control, and any one or combination of these factors
could materially affect Cumberland's operation results. These factors
include macroeconomic conditions, including rising interest rates
and inflation, competition, an inability of manufacturers to
produce Cumberland's products on a
timely basis, failure of manufacturers to comply with regulations
applicable to pharmaceutical manufacturers, natural disasters,
public health epidemics, maintaining an effective sales and
marketing infrastructure, and other events beyond the company's
control as more fully discussed in its most recent annual report on
Form 10-K as filed with the U.S. Securities and Exchange Commission
("SEC"), as well as the company's other filings with the SEC from
time to time. There can be no assurance that results anticipated by
the company will be realized or that they will have the expected
effects. Readers are cautioned not to place undue reliance on
forward-looking statements, which speak only as of the date hereof.
The company does not undertake any obligation to publicly revise
these statements to reflect events after the date hereof.
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SOURCE Cumberland Pharmaceuticals Inc.
