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 that David Meeker, M.D., Chair, Chief
Executive Officer and President, will participate in a fireside
chat at the Canaccord Genuity 42nd Annual Growth Conference on
Wednesday, August 10, 2022 at 12:30 p.m. ET in Boston, MA.
A live audio webcast of the presentation will be available under
“Events & Presentations” in the Investor Relations section of
the Company’s website at www.rhythmtx.com. A replay of the webcast
will be available on the Rhythm website for 30 days following the
presentation.
About Rhythm PharmaceuticalsRhythm is a
commercial-stage biopharmaceutical company committed transforming
the lives of patients and their families living with hyperphagia
and severe obesity caused by rare melanocortin-4 receptor (MC4R)
pathway diseases. Rhythm’s precision medicine, IMCIVREE
(setmelanotide), 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 POMC, PCSK1 or LEPR deficiency confirmed
by genetic testing, or patients with a clinical diagnosis of
Bardet-Biedl syndrome (BBS). The European Commission (EC) and Great
Britain’s Medicines & Healthcare Products Regulatory Agency
(MHRA) have authorized IMCIVREE for the treatment of obesity and
the control of hunger associated with genetically confirmed
loss-of-function biallelic POMC, including PCSK1, deficiency or
biallelic LEPR deficiency in adults and children 6 years of age and
above. IMCIVREE is the first-ever FDA-approved and EC- and
MHRA-authorized therapy for patients with these rare genetic
diseases of obesity. The Company submitted a Type II variation
application to the European Medicines Agency seeking regulatory
approval and authorization for setmelanotide to treat obesity and
control of hunger in adult and pediatric patients 6 years of age
and older with BBS in the European Union. Additionally, Rhythm is
advancing a broad clinical development program for setmelanotide in
other rare genetic diseases of obesity and is leveraging the Rhythm
Engine and the largest known obesity DNA database -- now with
approximately 45,000 sequencing samples -- to improve the
understanding, diagnosis and care of people living with severe
obesity due to certain genetic deficiencies. Rhythm’s headquarters
is in Boston, MA.
About SetmelanotideSetmelanotide is a
melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key
biological pathway that regulates hunger, caloric intake and energy
expenditure. Variants in genes may impair the function of the MC4R
pathway, potentially leading to hyperphagia and early-onset, severe
obesity. Rhythm is developing setmelanotide as a targeted therapy
to potentially restore the function of an impaired MC4R pathway
and, in so doing, potentially reduce hunger and weight in patients
with rare genetic diseases of obesity.
In the EU and Great Britain, IMCIVREE is indicated for the
treatment of obesity and the control of hunger associated with
genetically confirmed loss-of-function biallelic POMC, including
PCSK1, deficiency or biallelic LEPR deficiency in adults and
children 6 years of age and above. IMCIVREE should be prescribed
and supervised by a physician with expertise in obesity with
underlying genetic etiology.
Rhythm’s Type II variation application to the European Medicines
Agency (EMA) for the treatment of obesity and control of
hyperphagia in adult and pediatric patients 6 years of age and
older with BBS is under review. The Company is also continuing to
advance the most comprehensive clinical research program ever
initiated in MC4R pathway diseases, including the pivotal Phase 3
EMANATE clinical trial evaluating setmelanotide in four independent
sub-studies in patients with obesity due to POMC insufficiency
caused by heterozygous variants in the POMC or PCSK1 genes, LEPR
insufficiency caused by heterozygous variants in the LEPR gene,
SRC1 deficiency caused by a variant in the NCOA1 gene, and SH2B1
deficiency caused by a variant in the SH2B1 gene or 16p11.2
deletion encompassing the SH2B1 gene. The Phase 2 DAYBREAK trial is
evaluating setmelanotide in patients with severe obesity and
hyperphagia caused by rare variants associated with 10 prioritized
MC4R-relevant genes. Rhythm has also initiated a Phase 3 pediatric
trial and a Phase 3 trial evaluating a weekly formulation of
setmelanotide.
IMCIVREE®
(setmelanotide) IndicationIn the United
States, IMCIVREE is indicated 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 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)
- Bardet-Biedl syndrome (BBS)
Limitations of UseIMCIVREE is not indicated for
the treatment of patients with the following conditions as IMCIVREE
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
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Spontaneous
penile erections in males and sexual adverse reactions in females
have occurred. Inform patients that these events may occur and
instruct patients who have an erection lasting longer than 4 hours
to seek emergency medical attention.
Depression and Suicidal Ideation: Depression
and suicidal ideation have occurred. Monitor patients for new onset
or worsening depression or suicidal thoughts or behaviors. Consider
discontinuing IMCIVREE if patients experience suicidal thoughts or
behaviors, or clinically significant or persistent depression
symptoms occur.
Skin Pigmentation and Darkening of Pre-existing
Nevi: Generalized increased skin pigmentation and
darkening of pre-existing nevi have occurred. Perform a full body
skin examination prior to initiation and periodically during
treatment to monitor pre-existing and new pigmentary lesions.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol
Preservative in Neonates and Low Birth Weight Infants:
IMCIVREE 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
- The 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 POPULATIONSTreatment with
IMCIVREE is not recommended when breastfeeding. Discontinue
IMCIVREE when pregnancy is recognized unless the benefits of
therapy outweigh the potential risks to the fetus.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm
Pharmaceuticals at 833-789-6337 or FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
Please see the full Prescribing Information for
additional Important Safety Information.
Forward-Looking
StatementsThis 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, our expectations
surrounding potential regulatory submissions, approvals and timing
thereof, our business strategy and plans, including regarding
commercialization of setmelanotide, and our participation in
upcoming events and presentations. 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 liquidity and
expenses, the impact of the COVID-19 pandemic on our business and
operations, including our preclinical studies, clinical trials and
commercialization prospects, 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 quarter
ended June 30, 2022 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
Investor
Contact:Hannah DeresiewiczStern Investor Relations,
Inc.212-362-1200hannah.deresiewicz@sternir.com
Media Contact:Adam
DaleyBerry & Company Public
Relations212-253-8881adaley@berrypr.com
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