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 six abstracts have been accepted for
presentation – three oral presentations and three posters - at The
Obesity Society’s Annual Meeting at ObesityWeek® to be held October
14-17 in Dallas, TX.
The following abstracts were accepted as late-breakers for
presentation:
- “Weight Reduction in Patients With Hypothalamic Obesity Treated
with Setmelanotide for 12 Months” will be presented on October 17
from 11:45 a.m. to 1:15 p.m. CT in Exhibit Hall D. The lead author
on this research is Christian L. Roth, M.D., Seattle Children’s
Research Institute, Seattle, WA.
- “3-Year Setmelanotide Weight Outcomes in Patients with
Bardet-Biedl Syndrome and Obesity” will be presented on October 17
from 11:45 a.m. to 1:15 p.m. CT in Exhibit Hall D. The lead author
on this research is Jack Yanovski, M.D., Ph.D., Eunice Kennedy
Shriver National Institute of Child Health and Human Development,
NIH, Bethesda, MD.
- “4-Year Setmelanotide Weight Outcomes of Patients with POMC and
LEPR Deficiency Obesity” will be featured in an oral presentation
on October 17 from 10:30 to 10:45 a.m. CT in D174. The lead author
on this research is Wendy K. Chung, M.D., Ph.D., Division of
Molecular Genetics, Department of Pediatrics, Columbia University,
New York, NY.
- “Cardiac, Renal, and Endocrine/Diabetes Mellitus Outcomes in
Children with Bardet-Biedl Syndrome,” will be presented on October
15 from 11:45 a.m. to 1:15 p.m. CT in Exhibit Hall D. The lead
author on this research is Jeremy Pomeroy, Ph.D., M.S., Marshfield
Clinic Research Institute.
Rhythm will also present two additional oral presentations:
- “Impact of Setmelanotide on Metabolic Syndrome Risk in Patients
with POMC and LEPR Deficiency” will be presented on October 16 from
8:15 to 8:30 a.m. CT in Ballroom C. This research was led by Martin
Wabitsch, M.D., Ph.D., Department of Pediatrics and Adolescent
Medicine, University of Ulm in Germany.
- “Impact of Setmelanotide on Metabolic Syndrome Risk in Patients
with Bardet-Biedl Syndrome” will be presented on October 16 from
8:30 to 8:45 a.m. CT in Ballroom C. The lead author is Andrea Haqq,
M.D., M.H.S., Division of Pediatric Endocrinology at the University
of Alberta.
ObesityWeek® will be held at the Kay Bailey Hutchison Convention
Center in Dallas. Rhythm will post these data presentations on the
Company’s website on the “Publications and Presentations” page
following the conference.
In addition, the Company will host an investor conference call
and webcast to discuss these data presentations on Wednesday,
October 18, 2023, at 8:00 a.m. ET. This conference call will be
accessible under “Events & Presentations” in the Investor
Relations section of the Company’s website at www.rhythmtx.com. A
replay will be available on the Rhythm website for 30 days
following the presentation.
About Rhythm Pharmaceuticals Rhythm is a
commercial-stage biopharmaceutical company committed to
transforming the lives of patients and their families living with
hyperphagia and severe obesity caused by rare melanocortin-4
receptor (MC4R) diseases. Rhythm’s lead asset, IMCIVREE
(setmelanotide) is approved by the U.S. Food and Drug
Administration (FDA) and authorized by the European Commission (EC)
and the UK’s Medicines & Healthcare Products Regulatory
Agency (MHRA) for use in accordance with product labeling.
Additionally, Rhythm is advancing a broad clinical development
program for setmelanotide in other rare MC4R pathway diseases, as
well as a preclinical suite of investigational candidates 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 andi 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) and BBS.
In the European Union, setmelanotide is indicated for the
treatment of obesity and the control of hunger associated with
genetically confirmed Bardet-Biedl syndrome (BBS) or genetically
confirmed loss-of-function biallelic proopiomelanocortin (POMC),
including PCSK1, deficiency or biallelic leptin receptor (LEPR)
deficiency in adults and children 6 years of age and
above.
In Canada, setmelanotide is indicated for the treatment of
obesity due to Bardet-Biedl syndrome (BBS) or genetically-confirmed
biallelic pro-opiomelanocortin (POMC), proprotein convertase
subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR)
deficiency due to variants interpreted as pathogenic, likely
pathogenic, or of uncertain significance in adults and children 6
years of age and above. 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.
In Europe, Setmelanotide should be prescribed and supervised by
a physician with expertise in obesity with underlying genetic
etiology. WARNINGS AND
PRECAUTIONS Skin
Monitoring: Setmelanotide may lead to generalized
increased skin pigmentation and darkening of pre-existing naevi
because of its pharmacologic effect. Full body skin examinations
should be conducted annually to monitor pre-existing and new skin
pigmentary lesions before and during treatment with
setmelanotide.
Heart Rate and Blood Pressure Monitoring: 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.
Prolonged Penile Erection: Spontaneous penile
erections have been reported in clinical trials with setmelanotide.
Patients who have a penile erection lasting longer than 4 hours
should be instructed to seek emergency medical attention for
potential treatment of
priapism. Depression: In
clinical trials, depression has been reported in patients treated
with setmelanotide. Patients with depression should be monitored at
each medical visit during treatment with setmelanotide.
Consideration should be given to discontinuing setmelanotide if
patients experience suicidal thoughts or
behaviors. 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. The prescribing
physician should monitor growth (height and weight) using age- and
sex-appropriate growth curves.
Excipients: This medicinal product contains 10
mg benzyl alcohol in each ml. Benzyl alcohol may cause allergic
reactions. Patients who are pregnant or breastfeeding should be
advised of the potential risk from the excipient benzyl alcohol,
which might accumulate over time and cause metabolic acidosis. This
medicinal product should be used with caution in patients with
hepatic or renal impairment, because of the potential risk from the
excipient benzyl alcohol which might accumulate over time and cause
metabolic acidosis.
Sodium: This medicinal product contains less
than 1 mmol sodium (23 mg) per dose, that is to say essentially
“sodium-free.” ADVERSE
REACTIONS The most frequent adverse reactions
are hyperpigmentation (51%), injection site reaction (39%), nausea
(33%), and headache (26%). USE IN
SPECIFIC POPULATIONS
Pregnancy There are no data from the use
of setmelanotide in pregnant women. Animal studies do not indicate
direct harmful effects with respect to reproductive toxicity.
However, administration of setmelanotide to pregnant rabbits
resulted in decreased maternal food consumption leading to
embryo-fetal effects. As a precautionary measure, setmelanotide
should not be started during pregnancy or while attempting to get
pregnant as weight loss during pregnancy may result in fetal harm.
If a patient who is taking setmelanotide has reached a stable
weight and becomes pregnant, consideration should be given to
maintaining setmelanotide treatment as there was no proof of
teratogenicity in the nonclinical data. If a patient who is taking
setmelanotide and still losing weight gets pregnant, setmelanotide
should either be discontinued, or the dose reduced while monitoring
for the recommended weight gain during pregnancy. The treating
physician should carefully monitor weight during pregnancy in a
patient taking setmelanotide.
Breast-feeding It is unknown whether
setmelanotide is excreted in human milk. A nonclinical study showed
that setmelanotide is excreted in the milk of nursing rats. No
quantifiable setmelanotide concentrations were detected in plasma
from nursing pups. A risk to the newborn/infant cannot be excluded.
A decision must be made whether to discontinue breastfeeding or to
discontinue/abstain from setmelanotide therapy taking into account
the benefit of breastfeeding for the child and the benefit of
therapy for the mother.
Fertility No human data on the effect of
setmelanotide on fertility are available. Animal studies did not
indicate harmful effects with respect to fertility.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm
Pharmaceuticals at +1 (833) 789-6337. See Summary of Product
Characteristics’ APPENDIX V for a list of European national
reporting systems to communicate adverse
reactions. 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 setmelanotide and our other preclinical
investigational candidates, and our participation in upcoming
events and presentations Statements using words 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, risks
relating to our liquidity and expenses, our ability to enroll
patients in clinical trials, the design and outcome of clinical
trials, the ability to achieve necessary regulatory approvals,
risks associated with data analysis and reporting, failure to
identify and develop additional product candidates, unfavorable
pricing regulations, third-party reimbursement practices or
healthcare reform initiatives, risks associated with the laws and
regulations governing our international operations and the costs of
any related compliance programs, our ability to obtain or maintain
orphan drug designations for setmelanotide or to obtain or maintain
exclusivity in any use, the impact of competition, risks relating
to product liability lawsuits, inability to maintain our
collaborations, or the failure of these collaborations, our
reliance on third parties, risks relating to intellectual property,
our ability to hire and retain necessary personnel, the impact of
the COVID-19 pandemic and 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 our
Quarterly Report on Form 10-Q for the three months ended June 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 Connolly Executive Director,
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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