Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage
biopharmaceutical company focused on transforming the lives of
patients and their families living with hyperphagia and severe
obesity caused by rare melanocortin-4 receptor (MC4R) pathway
diseases, today announced four oral presentations at the 61st
Annual European Society for Paediatric Endocrinology (ESPE) Meeting
being held September 21-23, 2023 in The Hague, Netherlands.
“We are excited to deliver multiple presentations at ESPE 2023
including statistics that deepen the understanding of the genetics
of obesity from our Rare Obesity Advanced Diagnosis™ (ROAD) testing
program as we learn more about the importance of genetics in
patients with early-onset, severe obesity,” said David Meeker,
M.D., Chair, President and Chief Executive Officer of Rhythm.
Impact of Setmelanotide on Metabolic Syndrome
RiskAndrea Haqq, M.D., Division of Pediatric
Endocrinology, University of Alberta in Canada, and Jesús Argente
M.D., Ph.D., Department of Pediatrics and Pediatric Endocrinology,
Universidad Autónoma de Madrid in Spain, delivered an oral
presentation on the effects of setmelanotide on the metabolic
syndrome severity score based on body mass index (MetS-Z-BMI) score
in pediatric patients with Bardet-Biedl syndrome (BBS).
In an oral presentation titled, “Impact of Setmelanotide on
Metabolic Syndrome Risk in Pediatric Patients with POMC and LEPR
Deficiency,” researchers led by Martin Wabitsch, M.D., Ph.D.,
Department of Pediatrics and Adolescent Medicine, University of Ulm
in Germany, presented new findings regarding setmelanotide
treatment in pediatric patients with pro-opiomelanocortin (POMC) or
leptin receptor (LEPR) deficiency.
Analyses from ROAD genetic testing programIn an
oral presentation titled, “Frequency of MC4R Pathway Variants in a
European Cohort of Individuals with Early-Onset Severe Obesity,”
researchers led by Anthony Goldstone, M.D., Ph.D., Department of
Brain Sciences, Imperial College London, assessed a large
European-based cohort of individuals with early-onset severe
obesity, finding that 20.4% of individuals sequenced carried a
variant in one or more of 11 specific MC4R pathway-related genes,
including 5.0% of individuals with variants classified as
pathogenic, likely pathogenic or suspected pathogenic.
“Frequency of Obesity-Related Gene Variants in a European
Population with Early-Onset, Severe Obesity,” as presented orally
by Jesús Argente M.D., Ph.D., Department of Pediatrics and
Pediatric Endocrinology, Universidad Autónoma de Madrid in Spain,
shows that among individuals with early-onset, severe obesity,
approximately 31.4% carried variants believed to be associated with
obesity.
In addition, Rhythm is hosting a satellite symposium at ESPE
2023, titled, “Hyperphagia and early-onset, severe obesity: The
role of precision medicine in the treatment of leptin
melanocortin-4 receptor (MC4R) pathway diseases.”
About Rhythm Pharmaceuticals Rhythm
is a commercial-stage biopharmaceutical company committed to
transforming the lives of patients and their families living with
hyperphagia and severe obesity caused by rare melanocortin-4
receptor (MC4R) diseases. Rhythm’s product, IMCIVREE
(setmelanotide) is approved by the U.S. Food and Drug
Administration (FDA) and authorized by the European Medicines
Agency (and the UK’s Medicines & Healthcare Products
Regulatory Agency (MHRA) for use in accordance with product
labeling. Additionally, Rhythm is advancing a broad clinical
development program for setmelanotide in other rare MC4R pathway
diseases, as well as a preclinical suite of investigational
candidates for the treatment of congenital hyperinsulinism.
Rhythm’s headquarters is 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 the
potential, safety, efficacy, and regulatory and clinical progress
of setmelanotide, the potential benefits of setmelanotide for
patients with cardiovascular diseases and type 2 diabetes mellitus,
as well as severe obesity due to certain genetic deficiencies,
expectations regarding the prevalence of patients living with MC4R
pathway-related gene variants, and our participation in upcoming
events and presentations. Statements using words such as
“expect”, “anticipate”, “believe”, “may”, “will” and similar terms
are also forward-looking statements. Such statements are subject to
numerous risks and uncertainties, including, but not limited to,
risks relating to our liquidity and expenses, our ability to enroll
patients in clinical trials, the design and outcome of clinical
trials, the ability to achieve necessary regulatory approvals,
risks associated with data analysis and reporting, failure to
identify and develop additional product candidates, unfavorable
pricing regulations, third-party reimbursement practices or
healthcare reform initiatives, risks associated with the laws and
regulations governing our international operations and the costs of
any related compliance programs, our ability to obtain or maintain
orphan drug designations for setmelanotide or to obtain or maintain
exclusivity in any use, the impact of competition, risks relating
to product liability lawsuits, inability to maintain our
collaborations, or the failure of these collaborations, our
reliance on third parties, risks relating to intellectual property,
our ability to hire and retain necessary personnel, the impact of
the COVID-19 pandemic and general economic conditions on our
business and operations, including our preclinical studies,
clinical trials and commercialization prospects, and the other
important factors discussed under the caption “Risk Factors” in our
Quarterly Report on Form 10-Q for the three months ended June 30,
2023 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 ConnollyExecutive
Director, Investor Relations and Corporate CommunicationsRhythm
Pharmaceuticals, Inc.857-264-4280dconnolly@rhythmtx.com
Media Contact:Adam DaleyBerry & Company
Public Relations212-253-8881adaley@berrypr.com
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