Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a late-stage
biopharmaceutical company aimed at developing and commercializing
therapies for the treatment of rare genetic disorders of obesity,
announced today that results from two pivotal Phase 3 studies
evaluating setmelanotide in proopiomelanocortin (POMC) deficiency
obesity and leptin receptor (LEPR) deficiency obesity were
published in The Lancet Diabetes & Endocrinology. As previously
reported, data from the studies demonstrate that treatment with
setmelanotide, the company’s melanocortin-4 receptor (MC4R)
agonist, led to statistically significant and clinically meaningful
reductions of weight and hunger.
“Results from Rhythm’s pivotal Phase 3 studies,
which are the largest studies to date in POMC and LEPR deficiency
obesities, provide evidence regarding the safety and efficacy of
setmelanotide and we believe they validate its potential long-term
use as a novel treatment for severe obesity and hyperphagia,” said
co-author Peter Kühnen, M.D., Institute for Experimental Pediatric
Endocrinology, Charité Universitätsmedizin Berlin, Germany. “It is
important to recognize the signs of these rare genetic disorders
because we may soon have a targeted treatment option available for
the first time for obesity disorders caused by impairments of the
MC4R pathway.”
Rhythm initially reported positive topline data
from the Phase 3 studies in August 2019 and subsequently presented
updated data in a late-breaking research forum during the 37th
Annual Meeting of The Obesity Society at ObesityWeek® 2019.
Eight of 10 participants with POMC deficiency
obesity (80%; P<0.0001 compared with historical data) and five
of 11 participants with LEPR deficiency obesity (45%; P=0.0001
compared with historical data) achieved at least 10 percent weight
loss at approximately one year. The mean percent change in “most
hunger” score in participants aged 12 years and older was -27.1
percent (n=7; P=0.0005) in POMC deficiency obesity and -43.7
percent (n=7; P<0.0001) in LEPR deficiency obesity. Consistent
with prior clinical experience, setmelanotide was generally
well-tolerated in both trials. The most common adverse events were
injection site reaction, skin hyperpigmentation, and nausea.
“These results are significant because, as we know from natural
history data, individuals living with POMC or LEPR deficiency
obesity consistently experience substantial weight gain each year
beginning in early childhood, and we would not expect any of these
patients to be able to achieve 10 percent weight loss over the
course of a year without continued treatment,” said co-author
Karine Clément, professor of nutrition at Pitié-Salpêtrière
hospital and Sorbonne University in Paris. “These data and the
significant unmet need to address the obesity and hyperphagia
caused by rare genetic disorders of obesity underscore the
importance of testing for genetic variants that may impair MC4R
activation and lead to severe obesity.”
In May 2020, Rhythm announced that the U.S. Food
and Drug Administration (FDA) accepted the company's New Drug
Application (NDA) for setmelanotide for the treatment of POMC
deficiency obesity and LEPR deficiency obesity, granted Priority
Review of the NDA and assigned a Prescription Drug User Fee Act
(PDUFA) goal date of November 27, 2020. In July 2020, the Company
announced the submission of its Marketing Authorization Application
(MAA) to the European Medicines Agency (EMA) for the treatment of
POMC deficiency obesity and LEPR deficiency obesity.
“We are grateful to the authors and the
investigators involved in our pivotal Phase 3 clinical trials for
their continued partnership in advancing setmelanotide to address
significant unmet needs facing people with rare genetic disorders
of obesity,” Murray Stewart, M.D., Chief Medical Officer of Rhythm,
said.
The article is available online here:
http://www.thelancet.com/journals/landia/article/PIIS2213-8587(20)30364-8/fulltext.To
request a copy of the article, email medinfo@rhythmtx.com.
About
SetmelanotideSetmelanotide is an investigational,
melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key
biological pathway that independently regulates hunger, caloric
intake, and energy expenditure. Variants in genes may impair the
function of the MC4R pathway, potentially leading to hyperphagia
and early-onset, severe obesity. Rhythm is currently developing
setmelanotide as a targeted therapy to potentially restore the
function of an impaired MC4R pathway and, in so doing, potentially
reduce hunger and weight in patients with rare genetic disorders of
obesity. Currently, no pharmacologic therapies exist to treat these
conditions.
The FDA has granted Breakthrough Therapy designation to
setmelanotide for the treatment of obesity associated with genetic
defects upstream of the MC4R pathway, which includes POMC
deficiency obesity, LEPR deficiency obesity, Bardet-Biedl Syndrome
(BBS) and Alström syndrome. The EMA has also granted PRIority
MEdicines (PRIME) designation for setmelanotide for the treatment
of obesity and the control of hunger associated with deficiency
disorders of the MC4R pathway. Both the FDA and EMA have granted
orphan drug status to setmelanotide for POMC and LEPR deficiency
obesities. Rhythm is also evaluating setmelanotide for reduction in
hunger and body weight in a pivotal Phase 3 trial in people living
with BBS and Alström syndromes, with topline data from this trial
expected in the fourth quarter of 2020 or early in the first
quarter of 2021.
About Rhythm PharmaceuticalsRhythm is a
late-stage biopharmaceutical company focused on the development and
commercialization of therapies for the treatment of rare genetic
disorders of obesity. Rhythm is leveraging the Rhythm Engine --
comprised of its Phase 2 basket study, TEMPO Registry, GO-ID
genotyping study and Uncovering Rare Obesity program -- to improve
the understanding, diagnosis and potentially the treatment of rare
genetic disorders of obesity. For healthcare professionals, visit
www.UNcommonObesity.com for more information. For patients and
caregivers, visit www.LEADforRareObesity.com for more information.
The company is based in Boston, MA.
Forward-Looking Statements This
press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
All statements contained in this press release that do not relate
to matters of historical fact should be considered forward-looking
statements, including without limitation statements regarding our
business strategy and plans, including regarding commercialization
of setmelanotide; the potential, safety, efficacy, and regulatory
and clinical progress of setmelanotide, including anticipated
timing of data readouts and our expectations surrounding potential
regulatory approvals and timing thereof. Statements using words
such as “expect”, “anticipate”, “believe”, “may”, “will” and
similar terms are also forward-looking statements. These statements
are neither promises nor guarantees, but involve known and unknown
risks, uncertainties and other important factors that may cause our
actual results, performance or achievements to be materially
different from any future results, performance or achievements
expressed or implied by the forward-looking statements, including,
but not limited to, the impact of our management transition, our
ability to enroll patients in clinical trials, the design and
outcome of clinical trials, the impact of competition, the ability
to achieve or obtain necessary regulatory approvals, risks
associated with data analysis and reporting, our liquidity and
expenses, the impact of the COVID-19 pandemic on our business and
operations, including our preclinical studies, clinical trials and
commercialization prospects, and general economic conditions, and
other important factors discussed under the caption “Risk Factors”
in our Quarterly Report on Form 10-Q for the quarterly period
ended June 30, 2020 and our other filings with
the Securities and Exchange Commission. Except as required by
law, we undertake no obligations to make any revisions to the
forward-looking statements contained in this release or to update
them to reflect events or circumstances occurring after the date of
this release, whether as a result of new information, future
developments or otherwise.
Corporate Contact:David ConnollyHead of
Investor Relations and Corporate CommunicationsRhythm
Pharmaceuticals, Inc.857-264-4280dconnolly@rhythmtx.com
Investor Contact:Hannah DeresiewiczStern
Investor Relations,
Inc.212-362-1200hannah.deresiewicz@sternir.com
Media Contact:Adam Daley Berry & Company
Public Relations212-253-8881adaley@berrypr.com
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