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 several data presentations showing that
setmelanotide therapy resulted in sustained and deepened weight
loss in patients with severe obesity caused by rare MC4R pathway
diseases during The Obesity Society’s Annual Meeting at
ObesityWeek®.
Rhythm and its collaborators delivered a total of six
presentations at the conference in Dallas from October 14 to 17.
Presentation highlights include the following results from Rhythm’s
open-label, long-term extension studies evaluating setmelanotide in
individuals with hypothalamic obesity, Bardet-Biedl syndrome (BBS)
or obesity due to POMC or LEPR deficiency, as of a data cutoff date
of June 13, 2023:
- 25.5% reduction in mean body mass index (BMI) from baseline in
patients with hypothalamic obesity (n=12) at one year;
- 16.0% reduction in mean BMI in adult patients with Bardet-Biedl
syndrome (BBS) (n=9) at three years;
- 0.7 reduction in mean BMI Z score in pediatric patients younger
than 18 with BBS (n=15) at three years;
- 20.3% reduction in mean BMI in adult patients with obesity due
to POMC or LEPR deficiency (n=11) at four years; and
- 1.2 reduction in mean BMI Z score in pediatric patients younger
than 18 due to POMC or LEPR deficiency at four years.
“We are excited to highlight the data showing meaningful,
long-term benefits of setmelanotide in patients living with
hyperphagia and severe obesity caused by rare MC4R pathway diseases
at ObesityWeek,” said David Meeker, M.D., Chair, President and
Chief Executive Officer of Rhythm. “We believe the one-year BMI
reduction of more than 25% in patients with hypothalamic obesity –
where individuals trended towards normal body weight – is
particularly encouraging as it shows continued improvement over our
previously reported 16-week and six-month data. The hypothalamic
obesity data affirm our confidence as we advance our pivotal, Phase
3 trial and work to bring setmelanotide to patients and families
facing this challenging disease for which there are no approved,
effective therapies.”
Hypothalamic Obesity Data at One YearTwelve
patients who enrolled in Rhythm’s open-label, 16-week Phase 2 trial
and who also enrolled in the long-term extension trial and reached
one year or more on setmelanotide were included in the one-year
data analysis. In addition to the BMI and BMI Z score reductions
provided above, highlights from the late-breaking poster, “Weight
Reduction in Patients With Hypothalamic Obesity Treated with
Setmelanotide for 12 Months” by lead author Christian L. Roth,
M.D., Seattle Children’s Research Institute, include:
- Mean change of -1.1 in BMI Z score from baseline in pediatric
patients (n=11) at one year on therapy;
- Three of 11 pediatric patients achieved normal weight at one
year, as defined by the U.S. National Institutes of Health (NIH)
and World Health Organization (WHO) (>5th to <85th BMI
percentile);
- Eleven of 12 patients (91.7%) improved by one or more weight
classes based on BMI or BMI percentile as defined by the NIH and
WHO; and
- Body composition changes were favorable, with larger percent
decreases in total fat mass compared with lean muscle mass.
There were no serious adverse events (AE), no AEs that led to
study discontinuation during the trial, and no new safety concerns
were observed.
Bardet-Biedl Syndrome Data at Three Years Three
years of treatment with setmelanotide showed sustained, meaningful
benefit in weight-related measures in patients with BBS, as
reported in a poster entitled, “3-Year Setmelanotide Weight
Outcomes in Patients with Bardet-Biedl Syndrome and Obesity” by
lead author Jack Yanovski, M.D., Ph.D., Eunice Kennedy Shriver
National Institute of Child Health and Human Development, National
Institutes of Health.
POMC and LEPR Deficiency Data at Four
YearsLong-term treatment with setmelanotide demonstrated
sustained weight-related efficacy in pediatric and adult patients
with obesity due to POMC or LEPR deficiency, as reported in the
oral presentation, “Four-Year Setmelanotide Weight Outcomes of
Patients with POMC and LEPR Deficiency Obesity” by lead author
Wendy K. Chung, M.D., Ph.D., Division of Molecular Genetics,
Department of Pediatrics, Columbia University, New York, NY.
Rhythm announced additional presentations including:
- In an oral presentation, “Impact of Setmelanotide on Metabolic
Syndrome Risk in Patients with POMC and LEPR Deficiency” by lead
author Martin Wabitsch, M.D., Ph.D., Department of Pediatrics and
Adolescent Medicine, University of Ulm in Germany, data showed
intervention with setmelanotide may reduce the risk of future
metabolic syndrome, cardiovascular disease (CVD) and type 2
diabetes mellitus (T2DM) in patients with obesity due to POMC or
LEPR deficiency obesity.
- In a poster titled, “Cardiac, Renal, and Endocrine/Diabetes
Mellitus Outcomes in Children with Bardet-Biedl Syndrome” by lead
author Jeremy Pomeroy, Ph.D., M.S., of the Marshfield Clinic
Research Institute, researchers showed that the severity of obesity
was associated with increased prevalence of cardiac,
endocrine/diabetes, and renal outcomes early in life based on an
analysis of 318 pediatric patients with BBS enrolled in the
Clinical Registry Investigating BBS (CRIBBS). Researchers concluded
that timely diagnosis and early implementation of hyperphagia and
weight management strategies in pediatric patients with BBS may
reduce the risk and burden associated with these
comorbidities.
- In a poster titled, “Impact of Setmelanotide on Metabolic
Syndrome Risk in Patients with Bardet-Biedl Syndrome” by lead
author Andrea Haqq, M.D., M.H.S., Division of Pediatric
Endocrinology at the University of Alberta, data showed that
treatment response with one year of setmelanotide was associated
with reductions in MetS-Z-BMI score in pediatric patients with BBS,
which are associated with reduced risk of metabolic syndrome, CVD
and T2DM.
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, including with respect to our Phase 3 clinical trial
evaluating setmelanotide in hypothalamic obesity, the open-label,
long-term extension studies evaluating setmelanotide in individuals
with hypothalamic obesity, BBS or obesity due to POMC or LEPR
deficiencies, the potential benefits of setmelanotide for patients,
including those with BBS and hypothalamic obesity, 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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