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 new data from the Company’s long-term extension (LTE)
trial, which show continued body mass index (BMI) and weight
reductions in patients with Bardet-Biedl syndrome (BBS) or POMC or
LEPR deficiency obesity (biallelic) receiving between 18 months and
three years of setmelanotide therapy. Rhythm and its collaborators
delivered these data in poster presentations at the Endocrine
Society Annual Meeting & Expo (ENDO), being held June 11-14,
2022 in Atlanta.
“These presentations build on our strong pivotal trial data and
further illustrate setmelanotide’s ability to safely deliver
sustained, clinically meaningful weight loss for individuals living
with severe obesity caused by BBS, or by POMC or LEPR deficiency,”
said David Meeker, M.D., Chair, President and Chief Executive
Officer of Rhythm. “Responses to our precision therapy are shown to
be durable, with both adult and pediatric patients experiencing
continued reductions in body weight-related measures throughout the
long-term extension. This supports our belief in setmelanotide as a
chronic therapy across our approved or registration-stage
indications. As we approach the June 16 PDUFA goal date for BBS, we
are eager to deliver setmelanotide to the waiting community, and we
look forward to offering patients and their families a safe,
effective medicine that can provide lasting benefit.”
Bardet-Biedl SyndromeA total of 42 patients
with BBS who were treated with setmelanotide in Rhythm’s Phase 2 or
Phase 3 trial continued into the LTE. As of the data cutoff, 30 and
19 of these patients had received at least 18 and 24 months of
setmelanotide therapy, respectively. The data were presented in a
poster, “Long-term Efficacy of Setmelanotide in Patients with
Bardet-Biedl Syndrome,” by Jesús Argente, M.D., Ph.D., Universidad
Autónoma de Madrid in Spain. Highlights include:
- Mean (SD) percent change in BMI across all patients was −9.5%
(10.5%; n=30) and −14.3% (11.6%; n=19) at 18 and 24 months,
respectively;
- Patients 18 years old or older achieved a percent change in
weight of −8.6% (10.3%; n=15) and −14.9% (10.4%; n=6) at 18 and 24
months of treatment, respectively;
- Six of 15 patients (40%) and five of six patients (83.3%)
achieved ≥10% weight reduction at 18 and 24 months of treatment,
respectively;
- Patients younger than 18 years old achieved a mean (SD) change
in BMI Z score of −0.83 (0.50; n=13) and −0.72 (0.54; n=12) at 18
and 24 months of treatment, respectively;
- All patients younger than 18 years of age had a BMI Z score
reduction of ≥0.2 points. Twelve of 13 patients (92.3%) and 9 of 12
patients (75%) younger than 18 years of age achieved BMI Z score
reductions of ≥0.3 points at 18 and 24 months of treatment,
respectively; and
- As of the data cutoff, a total of 38 patients were receiving
ongoing treatment in the LTE trial; four patients discontinued
treatment, one due to an adverse event that was not related to
treatment.
POMC or LEPR deficiency obesity (biallelic) A
total of 24 patients with POMC or LEPR deficiency obesity who were
treated with setmelanotide in Rhythm’s Phase 2 or Phase 3 trial
continued into the LTE. As of the data cutoff, 21 and 15 of these
patients had received at least 24 or 36 months of setmelanotide
therapy, respectively. These data were presented in a poster
entitled, “Long-term Efficacy of Setmelanotide in Patients With
POMC or LEPR Deficiency Obesity,” by Karine Clément, M.D., Ph.D.,
Professor of Nutrition at Pitié-Salpêtrière Hospital, Sorbonne
Université and Head of the INSERM Nutriomics Laboratory in Paris.
Highlights include:
- Across all patients, mean (SD) percent change in BMI was −16.7%
(16.0%; n=21) and −17.5% (20.5%; n=15) after 24 and 36 months of
treatment, respectively.
- Patients 18 years old or older achieved a mean (SD) percent
change in body weight of −16.7% (16.2%; n=10) and −13.5% (15.9%;
n=8) after 24 and 36 months of treatment, respectively.
- Seven of 10 patients (70%) and five of eight patients (62.5%)
achieved ≥10% weight reduction after 24 and 36 months of treatment,
respectively.
- Patients younger than 18 years old achieved a mean (SD) change
in BMI Z score of −0.94 (0.95; n=10) and −0.73 (1.41; n=4) after 24
and 36 months, respectively.
- Eight of 10 patients (80%) and two of four patients (50%)
younger than 18 years old achieved BMI Z score reductions of ≥0.3
points at 24 and 36 months of treatment, respectively.
- As of the data cutoff, a total of 24 patients were receiving
ongoing treatment in the LTE trial; three patients discontinued
treatment, none due to adverse events.
Consistent with prior clinical observations, setmelanotide was
generally well tolerated in the LTE trial across both indications
and no new safety signals were observed.
The data presented from each LTE trial cohort were all as of a
cutoff date of Oct. 29, 2021.
All Rhythm’s presentations from ENDO will be available on the
Publications and Presentations section of its website:
https://www.rhythmtx.com/publications/.
About Rhythm PharmaceuticalsRhythm is a
commercial-stage biopharmaceutical company committed to
transforming the treatment paradigm for people living with rare
genetic diseases of obesity. Rhythm’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 in July and September 2021,
respectively, by the European Commission (EC) and Great
Britain’s Medicines & Healthcare Products Regulatory
Agency (MHRA) 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 supplemental New Drug Application (sNDA) to
the FDA, which was accepted for filing in November
2021 and is currently assigned a Prescription Drug User Fee
Act (PDUFA) goal date of June 16, 2022, for the treatment of
obesity and control of hunger in adult and pediatric patients six
years of age and older with Bardet-Biedl Syndrome (BBS) or Alström
syndrome. 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 also is
under review. 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.
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 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).
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.
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, EU SmPC
and MHRA SmPC 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, including the
anticipated timing for initiation of clinical trials and release of
clinical trial data and our expectations surrounding potential
regulatory submissions, approvals and timing thereof, our business
strategy and plans 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, 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 quarter ended March 31, 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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