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 the presentation of new data supporting the potential for
setmelanotide to treat the early-onset obesity, hyperphagia and
metabolic impairment associated with Bardet-Biedl syndrome (BBS).
In addition, the Company announced cumulative safety data from
across the setmelanotide clinical development program. These data
were presented at the Pediatric Endocrine Society (PES) 2022
Virtual Annual Meeting, held April 28 – May 1.
The Company and its collaborators delivered one oral
presentation and three e-poster presentations, including:
- New data from the 52-week Phase 3 trial evaluating
setmelanotide in patients with BBS, which demonstrate that
setmelanotide improved body weight measures, as well as total HDL
and LDL cholesterol and triglyceride levels, with no changes in
blood pressure or heart rate;
- New data from the 52-week Phase 3 trial specific to adolescents
and children with BBS, which show that setmelanotide treatment was
associated with clinically meaningful reductions in body mass index
(BMI), BMI-Z scores, and percent of the 95th BMI percentile
(%BMI95);
- New results from a study based on in-depth interviews conducted
with BBS patients and caregivers enrolled in either Rhythm’s Phase
2 Basket Trial or Phase 3 BBS pivotal trial, which suggest that
setmelanotide treatment reduced patients’ hyperphagia, body weight,
and obsessive focus on food, resulting in substantially improved
general health and emotional well-being; and
- Cumulative safety data from across 561 participants treated in
Rhythm’s 16 Phase 1, 2, or 3 clinical trials of setmelanotide in
healthy volunteers and people with obesity due to rare genetic
variants, which demonstrate that setmelanotide was generally well
tolerated.
“The new data presented at PES reinforce setmelanotide’s
potential to be a safe and effective precision therapy, which can
deliver meaningful benefit to people living with BBS, reducing
hyperphagia, weight, and comorbid factors of the metabolic
syndrome, while also improving emotional well-being,” said Linda
Shapiro, M.D., Ph.D., Chief Medical Officer of Rhythm. “As we
prepare for a potential U.S. approval in BBS later this quarter, we
are encouraged by these new data and look forward to sharing them
with the health care community, as we continue efforts to educate
about BBS, the patient and caregiver experience, and the need for
treatment.”
New Data on the Impact of Setmelanotide Treatment on
Lipid Parameters and Vital Signs from the Phase 3 Trial in
BBSAndrea Haqq, M.D., M.H.S., Professor, Department of
Pediatrics, Division of Pediatric Endocrinology/Metabolism at the
University of Alberta, delivered an oral presentation entitled,
“Impact of Setmelanotide Treatment on Lipid Parameters and Vital
Signs in Patients with Bardet-Biedl Syndrome in a Phase 3
Trial.”
“Due to their early-onset, severe obesity and hyperphagia,
people living with BBS are at a high risk of comorbid metabolic
syndrome,” said Dr. Haqq. “The data presented at PES suggest for
the first time that the benefits of setmelanotide treatment go
beyond weight and hunger reduction, affecting lipid parameters that
are known to play important roles in increasing the likelihood that
patients develop ischemic heart disease, stroke, and chronic kidney
disease. These results highlight the potential for setmelanotide to
address the major health ramifications associated with rare genetic
diseases of obesity, and further support its advancement as a
precision therapy for BBS.”
The pivotal Phase 3 trial included 31 response-evaluable
patients aged 7 years old or older with BBS. Patients’ fasting
lipid profiles were assessed before study initiation and after 52
weeks of therapy; weight and vital signs were assessed throughout
the trial. As previously disclosed, data from this Phase 3 trial
show that treatment with setmelanotide was associated with
significant reductions in body weight and hunger.
This presentation included new data specific to patients’ lipid
parameters and vital signs. Following one year of setmelanotide
treatment, the mean percent changes in total cholesterol, HDL
cholesterol, LDL cholesterol and triglycerides were -6.1%, 5.3%,
-7.8% and -9.6%, respectively. No systematic changes in blood
pressure or heart rate were identified.
New Data for Setmelanotide in Pediatric and Adolescent
Patients with BBS and Severe Obesity Robert Haws, M.D.,
Marshfield Clinic Research Institute, presented an e-poster
entitled, “Setmelanotide Treatment in Pediatric and Adolescent
Patients with Bardet-Biedl Syndrome and Severe Obesity,” which
provided additional data specific to adolescents and children
enrolled in Rhythm’s Phase 3 clinical trial. As previously
disclosed, patients younger than 18 years of age with BBS (n=16)
had a mean reduction in BMI-Z score of 0.8.
This presentation included new data specific to patients’ BMI
and %BMI95, which show that pediatric patients with BBS had a mean
reduction in BMI of -3.36kg/m2 (-9.50%) and a mean change in %BMI95
of -17.3.
New Data Detailing Patient- and Caregiver-reported
Experience of Hyperphagia from Phase 3 Trial in BBSIn an
e-poster entitled, “Patient and Caregiver-Reported Experiences of
Hyperphagia in Bardet-Biedl Syndrome Before and During
Setmelanotide Treatment,” Claire Ervin, M.P.H., Senior Director,
Patient-Centered Outcomes Assessment at RTI Health Solutions,
presented qualitative data from 19 interviews conducted with BBS
patients and caregivers who participated in Rhythm’s Phase 2 Basket
Trial and Phase 3 BBS pivotal trial.
Prior to setmelanotide treatment, all participants described
their hunger, or their child’s hunger, as all-consuming, and noted
a negative impact on daily life, including difficulties with
concentration, decrements in emotional and physical well-being and
impaired relationships. All participants experienced or observed
notable reductions in hunger during treatment with setmelanotide.
This led to a decrease in food-seeking behaviors, weight loss, and
better health. All participants reported improvements in how they
felt – physically and/or emotionally – and generally reported high
levels of satisfaction with treatment.
Clinical Safety Summary of Setmelanotide in Healthy
Volunteers with Obesity and Patients with Rare Genetic Diseases of
Obesity Jesús Argente, M.D., Ph.D., Professor in the
Department of Pediatrics and Pediatric Endocrinology, Universidad
Autónoma de Madrid in Spain, presented an e-poster entitled,
“Clinical Safety Summary of Setmelanotide in Healthy Volunteers
with Obesity and Patients with Rare Genetic Diseases of Obesity,”
which detailed safety data from across 16 Phase 1, 2 or 3 clinical
trials of setmelanotide.
As of March 8, 2021, 561 people, including 228 otherwise healthy
volunteers with obesity and 308 patients with rare genetic diseases
of obesity, had received at least one dose of setmelanotide, with
24% of patients receiving treatment for at least six months and 17%
of patients for one year. Data suggest that setmelanotide was
generally well tolerated, with a mostly consistent safety profile
across age, sex, ethnicity, race, and clinical diagnosis.
All Rhythm’s presentations from PES 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, 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, 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 Annual Report on
Form 10-K for the year ended December 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 ConnollyExecutive Director, 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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