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 first patients have been dosed
in the Company’s Phase 1 clinical trial of RM-718, an
investigational, weekly melanocortin-4 receptor (MC4R)-specific
agonist designed to be MC1R-sparing and to potentially avoid
hyperpigmentation.
“We are excited to begin clinical development of RM-718, our
next-generation MC4R agonist,” said David Meeker, M.D., Chair,
President and Chief Executive Officer of Rhythm. “Based on
encouraging preclinical data, we believe RM-718 has the potential
to reduce hyperphagia – a pathological hunger that leads to
abnormal food-seeking behavior – and treat severe obesity, which
are the two hallmark symptoms of rare MC4R pathway diseases. In
addition, RM-718 was designed to provide a weekly dosing regimen
and to avoid hyperpigmentation.”
This Phase 1 trial is a three-part study to evaluate safety,
tolerability and pharmacokinetics (PK). The study consists of Part
A: single ascending doses (SAD) of RM-718 in healthy participants
18 to 55 years old with obesity; Part B: multiple ascending doses
(MAD) of RM-718 in healthy participants 18 to 55 years old with
obesity; and Part C: MAD of RM-718 in patients 12 to 65 years old
with hypothalamic obesity. Cohorts in Parts A and B are
double-blind, placebo-controlled, and randomized 2:1. Study
participants will receive one weekly dose of either RM-718 or
placebo in Part A, four weekly doses of either RM-718 or placebo in
Part B, and four weekly doses of open-label RM-718 in Part C.
RM-718 or placebo doses are administered weekly via subcutaneous
injection. The anticipated enrollment for this study is 96
participants.
About RM-718RM-718 is an investigational,
weekly, MC4R-specific agonist that has demonstrated the potential
to reduce body weight and hunger in preclinical
studies. RM-718 is designed to be highly selective and
MC1Rsparing and thereby not cause hyperpigmentation.
About Rhythm PharmaceuticalsRhythm 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 design or progress, trial enrollment, potential benefits
and adverse effects, and potential regulatory submissions,
approvals and timing thereof of RM-718. Statements using word 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 quarter 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 ConnollyHead of 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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