Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage
biopharmaceutical company committed to transforming the care of
people living with rare genetic diseases of obesity, this week
presented the first-ever data on the health-related quality of life
(HRQOL) and experience of patients with Bardet-Biedl syndrome (BBS)
who were treated in its Phase 3 trial of setmelanotide at The
Obesity Society’s ObesityWeek®, a virtual conference that runs from
Nov. 1 to 5.
Also at ObesityWeek®, the company shared an oral presentation
detailing efficacy and safety data from its Phase 3 trial of
setmelanotide in BBS and Alström syndrome, as well as poster
presentations with new hunger reduction data from the SRC1 and
SH2B1 genetic deficiency cohorts in its exploratory Phase 2 Basket
Trial. Additional presentations include new data on utilization of
the Uncovering Rare Obesity® (URO) genetic testing program, an
analysis of the frequency of melanocortin-4 receptor (MC4R) pathway
variants in U.S. patients with severe obesity, and a review of
single minded-1 (SIM1) missense variants associated with severe
obesity.
“We are excited to share multiple presentations at ObesityWeek,
which demonstrate continued progress toward our goal of
transforming the care of people living with rare genetic diseases
of obesity,” said Linda Shapiro, M.D., Ph.D., Chief Medical Officer
of Rhythm. “The new BBS HRQOL data build on the safety and efficacy
results we first announced last year, further validating our belief
in setmelanotide’s potential as the first-ever therapy to address
the hyperphagia and severe obesity that affect the lives of
patients and families living with BBS. In addition, the new data
from the cohorts of patients with SH2B1 and SRC1 genetic
deficiencies in our Phase 2 Basket Trial demonstrated
setmelanotide’s ability to reduce hunger in people living with
these rare genetic diseases of obesity in the trial, further
reinforcing our confidence in the DAYBREAK and EMANATE trials we
are launching soon to expand our clinical evaluation into a total
of 36 genes with strong or very strong relevance to the MC4R
pathway.”
Setmelanotide in BBS: HRQOL and Efficacy and Safety
DataElizabeth Forsythe, Ph.D., Great Ormond Street
Institute of Child Health, Faculty of Population Health Sciences,
University College London, presented a poster entitled, “Quality of
Life in Patients with Bardet-Biedl Syndrome in a Setmelanotide
Phase 3 Trial.” This research was conducted as a post-hoc analysis
of Rhythm’s Phase 3 study, using the self-reported Pediatric
Quality of Life Inventory (PedsQL) or the Impact of Weight on
Quality of Life Questionnaire-Lite (IWQOL-Lite), both of which are
100-point scales, with zero being the worst and 100 being the best.
Highlights include:
- 85% of patients reported clinically meaningful improvements in
their HRQOL status after one year of setmelanotide therapy, or
preserved their non-impaired HRQOL status;
- For adult patients, changes in their IWQOL score were
clinically meaningful with a mean increase of 12 points after one
year on setmelanotide therapy from 74.9 at baseline;
- In pediatric patients, changes in their PedsQL score were
clinically meaningful with a mean increase of 11.2 after one year
on setmelanotide therapy from 67.2 at baseline;
- For the subset of patients without cognitive impairment,
clinically meaningful improvements in outcomes such as body mass
index and hunger mirrored their improvements in HRQOL;
- HRQOL improvements were sustained over the 52-week trial
period.
Robert Haws, M.D., Clinical Research Center at the Marshfield
Clinic Research Institute, delivered an oral presentation entitled,
“Efficacy and Safety of Open-Label Setmelanotide in Bardet-Biedl
Syndrome: A Phase 3 Trial.” Data from this trial showed that 52
weeks of setmelanotide treatment was associated with clinically
significant reduction in BMI in patients with BBS:
- Patients 18 years old or older (n=15) achieved a mean reduction
in BMI of 9.1% from baseline 46.4 kg/m2; and
- Patients younger than 18 years (n=16) achieved a mean reduction
in BMI of 9.5% from baseline 37.4 kg/m2.
Setmelanotide Therapy Achieved Hunger Reductions in
SH2B1 and SRC1 Deficiency ObesitiesRhythm also presented
new hunger reduction data from its exploratory Phase 2 Basket Trial
evaluating setmelanotide in patients with obesity due to variants
of the SRC1 gene or the SH2B1 gene.
Jesús Argente, M.D., Ph.D., Professor in the Department of
Pediatrics and Pediatric Endocrinology, Universidad Autónoma de
Madrid in Spain, presented a poster entitled, “Effects of
Setmelanotide on Obesity, Hunger, and Safety in SH2B1 Deficiency: A
Phase 2 Trial;” and Sadaf Farooqi, M.D., Ph.D., Wellcome-MRC
Institute of Metabolic Science and NIHR Cambridge Biomedical
Research Centre, University of Cambridge, presented, “Effects of
Setmelanotide on Obesity, Hunger, and Safety in SRC1
Insufficiency.”
In addition to confirming that patients with SRC1 deficiency or
SH2B1 deficiency experience severe obesity beginning at a young
age, data from these cohorts demonstrated that three months of
setmelanotide therapy resulted in reductions in most hunger scores
in patients 12 years old and older, regardless of whether the
patients met the study definition of weight responder.1
At the 59th Annual European Society for Paediatric Endocrinology
(ESPE) Meeting in September 2021, Rhythm presented topline analyses
from the Phase 2 Basket Trial that showed three months of
setmelanotide therapy achieved clinically meaningful weight loss or
BMI-Z reduction in 30% (9 of 30) of patients with SRC1 deficiency
and in 43% (15 of 35) of patients with SH2B1 deficiency.
Additional Rhythm poster presentations at ObesityWeek
include:
- Ida Moeller, ScD, ScM, MMSc, Director of Biomedical Informatics
at Rhythm, presented, “Frequency of MC4R pathway variants in a
large US cohort of patients with severe obesity;”
- Jill Garrison, Ph.D., Associate Director, Medical Affairs, at
Rhythm, presented, “Uncovering Rare Obesity Genetic Testing
Program: Overview and Health Care Provider Utilization;” and
- Megan Vogel, Ph.D., Scientist, Pre-Clinical Biology at Rhythm,
presented, “Biochemical Characterization of Single Minded-1
Missense Variants Associated with Severe Obesity.”
All Rhythm’s presentations from ObesityWeek will be available on
the Publication and Presentations section of its
website: https://www.rhythmtx.com/publications/
1A responder was defined as having ≥5% weight loss in those ≥18
years old or ≥0.15 reduction in BMI Z score in those <18 years
old.
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 in September 2021 and submitted a Type II variation
application to the European Medicines Agency in October 2021
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 or Alström syndrome in both the
United States and European Union. 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 37,500 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, 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,
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 June 30, 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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