Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage
biopharmaceutical company focused on transforming the lives of
patients and their families living with rare neuroendocrine
diseases, today announced that the Company delivered three
presentations – one oral and two posters – at The Pediatric
Endocrine Society’s (PES) Annual Meeting held May 2-5, 2024 in
Chicago, IL.
In the oral presentation, Rhythm showcased data that was
previously announced on December 6, 2023, from its 52-week, Phase 3
pediatrics trial in patients between the ages of 2 and younger than
6 years (N=12) with Bardet-Biedl syndrome (BBS) or obesity due to
proopiomelanocortin (POMC) or leptin receptor (LEPR) deficiency.
Treatment with setmelanotide achieved the primary endpoint with a
3.04 mean reduction in BMI-Z score (a measure of body mass index
deviations from what is considered normal) and 18.4 percent mean
reduction in body mass index (BMI).
“The hyperphagia and severe obesity of rare, genetically-caused
melanocortin-4 receptor (MC4R) pathway diseases can present at a
young age and have a significant impact on patients and their
families,” said presenting author Megan Kelsey, M.D., Children’s
Hospital Colorado. “These data demonstrate setmelanotide’s
potential to achieve clinically significant weight loss in children
ages 2 to younger than 6 years old, potentially allowing us to
treat patients earlier in life.”
Rhythm remains on track to complete submission of a
supplementary New Drug Application (sNDA) to the U.S. Food and Drug
Administration (FDA) seeking a label expansion to treat pediatric
patients between the ages of 2 and younger than 6 years in approved
indications in the first half of 2024.
Rhythm also presented two additional poster presentations at PES
2024:
- “Weight Reduction in Pediatric
Patients With Hypothalamic Obesity Treated With a Therapy for 12
Months”
- “Efficacy in Weight Reduction and
Safety in Pediatric Age Groups With Rare Melanocortin-4 Receptor
Pathway–Related Obesity Treated With a Therapy for 12 Months”
About Rhythm Pharmaceuticals Rhythm is a
commercial-stage biopharmaceutical company committed to
transforming the lives of patients and their families living with
rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE®
(setmelanotide), an MC4R agonist designed to treat hyperphagia and
severe obesity, is approved by the U.S. Food and Drug
Administration (FDA) for chronic weight management in adult and
pediatric patients 6 years of age and older with monogenic or
syndromic obesity due to pro-opiomelanocortin (POMC), proprotein
convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor
(LEPR) deficiency confirmed by genetic testing, or patients with a
clinical diagnosis of Bardet-Biedl syndrome (BBS). Both the
European Commission (EC) and the UK’s Medicines & Healthcare
Products Regulatory Agency (MHRA) have authorized setmelanotide for
the treatment of obesity and the control of hunger associated with
genetically confirmed BBS or genetically confirmed loss-of-function
biallelic POMC, including PCSK1, deficiency or biallelic LEPR
deficiency in adults and children 6 years of age and above.
Additionally, Rhythm is advancing a broad clinical development
program for setmelanotide in other rare diseases, as well as
investigational MC4R agonists LB54640 and RM-718, and a preclinical
suite of small molecules for the treatment of congenital
hyperinsulinism. Rhythm’s headquarters is in Boston, MA.
Setmelanotide IndicationIn the United States,
setmelanotide is indicated for chronic weight management in adult
and pediatric patients 6 years of age and older with monogenic or
syndromic obesity due to POMC, PCSK1 or LEPR deficiency as
determined by an FDA-approved test demonstrating variants in POMC,
PCSK1 or LEPR genes that are interpreted as pathogenic, likely
pathogenic, or of uncertain significance (VUS) or BBS.
In the European Union, setmelanotide is indicated for the
treatment of obesity and the control of hunger associated with
genetically confirmed BBS or loss-of-function biallelic POMC,
including PCSK1, deficiency or biallelic LEPR deficiency in adults
and children 6 years of age and above. In Europe, setmelanotide
should be prescribed and supervised by a physician with expertise
in obesity with underlying genetic etiology.
Limitations of Use Setmelanotide is not
indicated for the treatment of patients with the following
conditions as setmelanotide would not be expected to be
effective:
- Obesity due to suspected POMC, PCSK1 or LEPR deficiency with
POMC, PCSK1 or LEPR variants classified as benign or likely
benign.
- Other types of obesity not related to POMC, PCSK1 or LEPR
deficiency, or BBS, including obesity associated with other genetic
syndromes and general (polygenic) obesity.
Contraindication Prior serious
hypersensitivity to setmelanotide or any of the excipients in
IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis)
have been reported.
WARNINGS AND PRECAUTIONS
Skin Pigmentation and Darkening of Pre-Existing
Nevi: Generalized increased skin pigmentation and
darkening of pre-existing nevi have occurred because of its
pharmacologic effect. Full body skin examinations prior to
initiation and periodically during treatment should be conducted to
monitor pre-existing and new pigmentary lesions.
Heart rate and blood pressure monitoring: In
Europe, heart rate and blood pressure should be monitored as part
of standard clinical practice at each medical visit (at least every
6 months) for patients treated with setmelanotide.
Disturbance in Sexual Arousal: Spontaneous
penile erections in males and sexual adverse reactions in females
have occurred. Patients who have an erection lasting longer than 4
hours should seek emergency medical attention.
Depression and Suicidal Ideation: Depression
and suicidal ideation have occurred. Patients should be monitored
for new onset or worsening depression or suicidal thoughts or
behaviors. Consideration should be given to discontinuing
setmelanotide if patients experience suicidal thoughts or
behaviors, or clinically significant or persistent depression
symptoms occur.
Hypersensitivity Reactions: Serious
hypersensitivity reactions (e.g., anaphylaxis) have been reported.
If suspected, advise patients to promptly seek medical attention
and discontinue setmelanotide.
Pediatric Population: The prescribing physician
should periodically assess response to setmelanotide therapy. In
growing children, the impact of weight loss on growth and
maturation should be evaluated. In Europe, the prescribing
physician should monitor growth (height and weight) using age- and
sex-appropriate growth curves.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol
Preservative in Neonates and Low Birth Weight Infants:
Setmelanotide is not approved for use in neonates or infants.
Serious and fatal adverse reactions including “gasping syndrome”
can occur in neonates and low birth weight infants treated with
benzyl alcohol-preserved drugs.
ADVERSE REACTIONS Most common
adverse reactions (incidence ≥20%) included skin hyperpigmentation,
injection site reactions, nausea, headache, diarrhea, abdominal
pain, vomiting, depression, and spontaneous penile
erection.
USE IN SPECIFIC POPULATIONS
Lactation: Not recommended when
breastfeeding. To report SUSPECTED ADVERSE REACTIONS, contact
Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at
1-800-FDA-1088 or www.fda.gov/medwatch. See section 4.8 of the
Summary of Product Characteristics for information on
reporting suspected adverse reactions in Europe.
Please see the full Prescribing Information for
additional Important Safety
Information. 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 our products or product candidates, including
setmelanotide to treat BBS in patients between the ages of 2 and
younger than 6 years, the anticipated timing and expectations
surrounding potential regulatory submissions and label expansion,
our participation in upcoming events and presentations, and the
date, time, and content thereof. 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,
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 ability to
successfully commercialize setmelanotide, our liquidity and
expenses, our ability to retain our key employees and consultants,
and to attract, retain and motivate qualified personnel, and
general economic conditions, and the other important factors
discussed under the caption “Risk Factors” in our Annual Report
on Form 10-K for the year ended December 31, 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
Connolly Head of Investor Relations and Corporate
Communications Rhythm Pharmaceuticals,
Inc. 857-264-4280 dconnolly@rhythmtx.com
Media Contact: Adam Daley Berry &
Company Public Relations 212-253-8881
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