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 2 clinical trial evaluating LB54640, an
investigational oral melanocortin-4 receptor (MC4R) agonist, in
hypothalamic obesity.
“Based on LG Chem’s significant preclinical work and encouraging
results from a Phase 1 trial, we believe LB54640 has the potential
to be an effective oral therapy for treating MC4R pathway diseases
without hyperpigmentation,” said David Meeker, M.D., Chair, Chief
Executive Officer and President of Rhythm Pharmaceuticals. “We look
forward to advancing this molecule with the goal of offering a full
portfolio of treatment options to patients struggling with
hyperphagia and severe obesity and ensuring they get the treatment
that is right for them.”
The Phase 2 trial is a randomized, placebo-controlled,
double-blind study to assess the effect of LB54640 on safety,
weight reduction, hunger, and quality of life in patients 12 years
of age and older (N=28) with hypothalamic obesity. Patients will
take an oral daily dose of either LB54640 (low, middle, or high
dose) or placebo for 14 weeks. The primary endpoint of the study is
the change from baseline in body mass index after 14 weeks of
treatment, and patients may continue on therapy for up to 52
weeks.
Rhythm entered into a global licensing agreement with LG Chem,
Ltd. ("LG Chem") for LB54640 in January 2024. In a Phase 1 trial in
healthy overweight adults, LB54640 demonstrated dose-dependent
weight reduction and a favorable safety profile with no changes in
blood pressure or heart rate observed and no hyperpigmentation
observed.
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 Indication In 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 LB54640, the potential timing, payments due,
and benefits of the global licensing agreement for LB54540,
expectations regarding the design, enrollment, or outcome of
clinical trials of LB54640, the ability to reach any net sales or
revenue milestones, and obtaining regulatory approvals in
connection with the global licensing agreement; and the timing of
any of the foregoing. Statements using words such as “expect”,
“anticipate”, “believe”, “may”, “will”, “aim” 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 any of the following: 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, data analysis and reporting,
failure to identify and develop additional product candidates, the
impact of competition, product liability lawsuits, inability to
maintain our collaborations, or the failure of these
collaborations, our reliance on third parties, intellectual
property, 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 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 Quarterly Report
on Form 10-Q for the three months ended March 31, 2024 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 adaley@berrypr.com
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