Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage
biopharmaceutical company committed to transforming the care of
people living with rare genetic diseases of obesity, today
announced a collaborative research agreement with the Clinical
Registry Investigating Bardet-Biedl Syndrome (CRIBBS).
“This collaborative research agreement with Rhythm and CRIBBS
paves the way for a comprehensive population study that we believe
will deepen our understanding of the needs of patients and families
living with Bardet-Biedl syndrome (BBS) and the real-world impact
severe obesity and hyperphagia, or insatiable hunger, has on them,”
said Robert Haws, M.D., of the Marshfield Clinic Research
Institute.
More than 600 individuals living with BBS are enrolled in
CRIBBS, with more than 75 percent of them in the United States. The
registry is designed to advance the understanding of BBS and its
impact on patients and families, improve the care of people living
with the disease, and augment scientific and clinical research.
CRIBBS collects de-identified patient information through medical
records, patient and caregiver interviews and annual surveys. It is
supported by the BBS Foundation and Family Association, Bright
Light to BBS, and Marshfield Clinic Research Institute.
Under the collaboration, health outcomes researchers will review
data collected through registry questionnaires to quantify and
describe unmet medical needs of adults and children with BBS and
the impact severe obesity and hyperphagia has on them.
Specifically, researchers will analyze natural history of treatment
patterns and weight changes over time, as well as specific health
outcomes and quality of life measures. The study also will examine
the impact of hyperphagia on quality of life and developmental
delays.
“With an improved understanding of the impacts of severe obesity
and hyperphagia on long-term health and quality of life, we expect
to be able to develop more tailored solutions to identify, properly
diagnosis and treat people living with BBS and, we hope, improve
our collective ability to address the needs and challenges facing
this community,” said Murray Stewart, M.D., Chief Medical Officer
of Rhythm.
BBS is an ultra-rare genetic disease that affects multiple organ
systems. Clinical features of BBS may include hyperphagia,
early-onset, severe obesity, cognitive impairment, polydactyly,
renal dysfunction, hypogonadism and visual impairment. There is
great variability in presentation and severity of these symptoms
across individuals with BBS. In the United States, Rhythm estimates
that BBS affects approximately 1,500 to 2,500 people.
About Rhythm
PharmaceuticalsRhythm is a commercial-stage
biopharmaceutical company committed to transforming the treatment
paradigm for people living with rare genetic diseases of obesity.
The Company’s precision medicine, IMCIVREE (setmelanotide), was
approved in November 2020 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 obesity due to
POMC, PCSK1 or LEPR deficiency confirmed by genetic testing and by
the European Commission (EC) in July 2021 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-authorized therapy for these rare genetic
diseases of obesity. Rhythm is advancing a broad clinical
development program for setmelanotide in other rare genetic
diseases of obesity. The Company is leveraging the Rhythm Engine
and the largest known obesity DNA database - now with approximately
37,500 sequencing samples - to improve the understanding, diagnosis
and care of people living with severe obesity due to certain
genetic deficiencies. The company is based in Boston, MA.
IMCIVREE™ (setmelanotide)
IndicationIMCIVREE is indicated for chronic weight
management in adult and pediatric patients 6 years of age and older
with obesity due to proopiomelanocortin (POMC), proprotein
convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor
(LEPR) deficiency. The condition must be confirmed by genetic
testing demonstrating variants in POMC, PCSK1,
or LEPR genes that are interpreted as pathogenic, likely
pathogenic, or of uncertain significance (VUS).
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, including obesity associated with other genetic
syndromes and general (polygenic) obesity.
Important Safety
Information
WARNINGS AND
PRECAUTIONS
Disturbance in Sexual
Arousal: Sexual adverse reactions may occur in
patients treated with IMCIVREE. Spontaneous penile erections in
males and sexual adverse reactions in females occurred in clinical
studies with IMCIVREE. Instruct patients who have an erection
lasting longer than 4 hours to seek emergency medical
attention.
Depression and Suicidal
Ideation: Some drugs that target the central nervous
system, such as IMCIVREE, may cause depression or suicidal
ideation. Monitor patients for new onset or worsening of
depression. Consider discontinuing IMCIVREE if patients experience
suicidal thoughts or behaviors.
Skin Pigmentation and
Darkening of Pre-Existing Nevi: IMCIVREE may cause
generalized increased skin pigmentation and darkening of
pre-existing nevi due to its pharmacologic effect. This effect is
reversible upon discontinuation of the drug. Perform a full body
skin examination prior to initiation and periodically during
treatment with IMCIVREE to monitor pre-existing and new skin
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.
ADVERSE REACTIONS
- The most common adverse reactions (incidence ≥23%) were
injection site reactions, skin hyperpigmentation, nausea, headache,
diarrhea, abdominal pain, back pain, fatigue, vomiting, depression,
upper respiratory tract infection, and spontaneous penile
erection.
USE IN SPECIFIC
POPULATIONSDiscontinue IMCIVREE when pregnancy is
recognized unless the benefits of therapy outweigh the potential
risks to the fetus.
Treatment with IMCIVREE is not
recommended for use while 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 Full Prescribing
Information for IMCIVREE.
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, anticipated
benefits of and activities under our research collaboration with
CRIBBS, and our business strategy and plans. 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, the impact of our management transition, 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 quarterly period ended March 31,
2021 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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