Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global
commercial-stage biopharmaceutical company focused on transforming
the lives of patients and their families living with rare
neuroendocrine diseases, today announced plans to add a cohort of
patients with hypothalamic obesity in Japan to its ongoing global
Phase 3 clinical trial of setmelanotide, with dosing expected to
begin in the third quarter of 2024.
“Following constructive discussions with Japan’s Pharmaceuticals
and Medical Devices Agency (PMDA), we are pleased to have developed
a clear and efficient plan to support the potential approval of
setmelanotide for hypothalamic obesity in Japan,” said David
Meeker, M.D., Chair, President and Chief Executive Officer of
Rhythm. “With data showing a higher per-capita incidence and
prevalence of patients with hypothalamic obesity in Japan compared
to the United States and Europe and no approved therapeutic
options, there is a major unmet need for a therapy to treat the
rapid onset of severe obesity and hyperphagia of hypothalamic
obesity.”
Rhythm estimates that there are approximately 5,000 to 8,000
patients in Japan with hypothalamic obesity, a rare form of obesity
that occurs following damage to the hypothalamic region of the
brain, which includes the melanocortin-4 receptor (MC4R) pathway
and is responsible for controlling physiological functions such as
hunger and weight regulation. The condition most frequently follows
the growth and surgical removal or other treatment of
craniopharyngioma, astrocytoma, or other rare brain tumors. These
individuals often experience rapid weight gain, a reduction in
energy expenditure, and an increase in hunger leading to severe
obesity within six to 12 months following the hypothalamic
lesions.
The Company and Japan’s PMDA agreed to add a cohort of 12
Japanese patients to the ongoing trial and, pending completion of
the trial, to use these data as part of the Company’s registration
package to seek approval from Japan’s Ministry of Health, Labor and
Welfare. In addition to efficacy data, Rhythm will collect and
submit pharmacokinetics (PK) data from Japanese patients,
expediting the typical pathway of collecting such data from an
earlier-stage trial in Japanese subjects.
Rhythm also announced today that it completed enrollment in the
pivotal, 120-patient cohort in its global Phase 3 trial of
setmelanotide in hypothalamic obesity with patients, aged 4 years
or older with hypothalamic obesity randomized 2:1 to setmelanotide
therapy or placebo for a total of 60 weeks, including up to eight
weeks for dose titration. As agreed to with both the United States
Food and Drug Administration (FDA) and the European Medicines
Agency (EMA), Rhythm’s regulatory submissions would be based on
data from this cohort. The additional 12-patient Japanese cohort
will not affect timing for regulatory submissions in the United
States or Europe. The Company remains on track to obtain top-line
study results in the first half of 2025.
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 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 design or progress, potential regulatory submissions,
approvals and timing thereof, including our Phase 3 trial of
setmelanotide for patients with hypothalamic obesity in Japan, the
United States or in Europe, the potential benefits of setmelanotide
for patients with hypothalamic obesity, and our business strategy
and plans, including regarding commercialization of IMCIVREE.
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 Quarterly Report on Form 10-Q for the three months
ended September 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 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
Rhythm Pharmaceuticals (NASDAQ:RYTM)
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Rhythm Pharmaceuticals (NASDAQ:RYTM)
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