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
presented the first-ever data on the health related quality of life
(HRQOL) and experience of patients with obesity due to POMC or LEPR
deficiency and updated results from the Uncovering Rare Obesity®
(URO) genetic testing program at the Obesity Medicine Association’s
Overcoming Obesity 2021 Conference and its Digital Experience (DX)
Oct. 14-23.
The Company and its collaborators delivered four poster
presentations, including:
- New HRQOL data from post-hoc analyses of Phase 3 trials
evaluating setmelanotide in patients with POMC or LEPR deficiency
obesity that showed setmelanotide treatment led to sustained,
clinically meaningful HRQOL improvements in a majority of
patients;
- New results from a study based on in-depth patient interviews
conducted in patients with POMC and LEPR deficiency obesity
enrolled in Rhythm’s pivotal Phase 3 trials, which highlighted that
the reduced hunger and improved satiety resulting from
setmelanotide treatment substantially and meaningfully changed
patients’ lives; and
- Two presentations detailing updated results from Rhythm’s URO
genetic testing of approximately 8,500 people in the United States
with early-onset, severe obesity project that:
- 64.5% of individuals who had genetic sequencing performed may
carry variants associated with rare genetic diseases of obesity,
including 54.6% with variants in the melanocortin-4 receptor (MC4R)
pathway that may qualify them for enrollment in Rhythm’s EMANATE or
DAYBREAK trials or for treatment with IMCIVREE; and
- 1.96% of individuals who had genetic sequencing performed may
carry biallelic variants in one of 22 Bardet-Biedl Syndrome
(BBS)-associated genes or the ALMS1 gene, of which up to 0.34%
carried variants considered pathogenic or likely pathogenic.
“These new data contribute to the growing body of evidence that
supports setmelanotide’s potential to deliver clinically meaningful
weight loss and clinically meaningful improvements in patient
reported hyperphagia, as well as HRQOL, reinforcing the value of
setmelanotide’s potential for the treatment of rare genetic
diseases of obesity of the MC4R pathway,” said Linda Shapiro, M.D.,
Ph.D., Chief Medical Officer of Rhythm. “As we prepare to initiate
our next wave of clinical trials, we are encouraged by these new
results from the URO genetic testing program, which suggest the
potential prevalence of genetic variants among people living with
early-onset, severe obesity. These results reinforce the importance
of genetic testing for clinical decision making in individuals with
early-onset, severe obesity and hyperphagia and support our plans
to initiate the Phase 3 EMANATE and Phase 2 DAYBREAK trials later
this year, which will expand our clinical development of
setmelanotide into patients with variants in any of 36 genes that
may impair the MC4R pathway.”
New HRQOL Data and Patient-reported Experience of Hunger
from Phase 3 Trials in POMC and LEPR Deficiency
ObesitiesInvestigators presented two posters with data
generated from post-hoc analyses of the Company’s Phase 3 trials in
POMC and LEPR deficiency obesities.
In a presentation entitled, “Quality of Life in POMC or LEPR
Deficiency: Setmelanotide Phase 3 Trials,” Peter Kühnen, M.D.,
Charité - Universitätsmedizin Berlin, Corporate Member of Freie
Universität Berlin und Humboldt-Universität zu Berlin, Institute
for Experimental Pediatric Endocrinology presented data on the
HRQOL burden improvements before and after treatment with
setmelanotide.1 Highlights include:
- Setmelanotide resulted in clinically meaningful improvement in
HRQOL in eight of 13 (73%) of patients after 52 weeks of treatment,
with improvements in HRQOL that were 2 to 3 times larger than the
relevant meaningful threshold; and
- Meaningful HRQOL improvements were observed as early as Week 5
on therapy, and these improvements were maintained throughout the
study as they mirrored clinically meaningful hunger reductions and
weight loss in these patients.
Martin Wabitsch, M.D. Division of Pediatric Endocrinology and
Diabetes, Center for Rare Endocrine Diseases, Department of
Pediatrics and Adolescent Medicine, University of Ulm, Ulm,
Germany, presented qualitative data from a series of patient
interviews in a poster entitled, “Patient Experience of Hunger in
POMC or LEPR Deficiency.” Patients reported that hyperphagia and
the constant inability to feel satiety negatively affects their
HRQOL, while reduced hunger and improved satiety achieved on
setmelanotide therapy substantially and meaningfully changed their
lives, profoundly improving their ability to function at school or
work. Additionally, these patients reported that discontinuing
treatment with setmelanotide would be devastating.
“The results of these studies underscore the tremendous burden
of POMC and LEPR deficiency obesity, better characterizing the
experience of people living with these conditions and highlighting
the significant impact of severe obesity and insatiable hunger on
their daily lives,” said Dr. Kühnen. “Importantly, these data
suggest setmelanotide can deliver clinically meaningful HRQOL
benefits, reducing the significant burden of disease by providing
patients with improvements in their feelings of insatiable hunger,
in addition to substantial weight loss. These results further
reinforce the value of setmelanotide as the first U.S., EU and
UK-approved precision medicine for chronic weight management in
POMC and LEPR deficiency obesities.”
Updated Data from the URO Testing ProgramIda
Moeller, ScD, ScM, MMSc, Director of Biomedical Informatics at
Rhythm, presented, “Variants in Obesity-related Genes in a
Population with Early-onset Obesity.” Rhythm’s URO testing program
is designed to expand access to genetic testing for patients with
suspected rare genetic diseases of obesity in the U.S. As of July
12, 2021, Rhythm had collected genetic sequences from approximately
8,500 individuals with early-onset, severe obesity, including 788
individuals who had genetic sequencing performed on the Company’s
updated genetic panel with 79 genes and the full chromosomal region
16p11.2.
Based on an integrated yield weighted by the number of
individuals sequenced for each gene, utilizing the data from the
two panels (the original panel and the updated expanded panel),
Rhythm projects that 64.5% of individuals sequenced may carry
actionable variants linked to rare genetic diseases of obesity.2
Rhythm also estimates that 54.6% of individuals sequenced carry
variants in the MC4R pathway that may qualify them for enrollment
in Rhythm’s EMANATE or DAYBREAK trials or commercial treatment with
IMCIVREE.
Additionally, Dr. Robert Haws, M.D., Marshfield Clinical
Research Institute, presented, “Frequency of BBS and ALMS1 Variants
in a Cohort With Early-onset Obesity.” Based on updated URO
results, the Company projects that 0.34% of individuals with
early-onset, severe obesity may carry pathogenic or likely
pathogenic variants in genes known to be associated with BBS or
Alstrom syndrome (or 0.24%, excluding patient samples from a
leading BBS clinic where higher frequency of BBS-related variants
would expected). Including patients with variants of unknown
significance (VUS), Rhythm estimates that 1.96% of individuals with
early-onset, severe obesity may carry biallelic variants in one of
22 known BBS-associated genes or ALMS1.3
Also at the Overcoming Obesity 2021 Conference, Rhythm and its
collaborators presented three additional abstracts as posters, all
of which detailed previously reported clinical data:
- “Efficacy and Safety of the Melanocortin-4 Receptor Agonist
Setmelanotide in Obesity Due to Bardet-Biedl Syndrome: a Phase 3
Trial,” as presented by Dr. Robert Haws, M.D., Marshfield Clinical
Research Institute;
- “Setmelanotide in POMC, PCSK1, or LEPR Heterozygous Deficiency
Obesity (Phase 2),” as presented by Sadaf Farooqi, Ph.D.,
Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge
Biomedical Research Centre, University of Cambridge;
- “Trial of a Once-Weekly Setmelanotide Formulation in Patients
with Obesity,” as presented by Annette Valles-Sukkar, Director
Clinical Operations, Rhythm Pharmaceuticals.
All Rhythm’s presentations from Obesity Medicine Association’s
Overcoming Obesity 2021 Conference and its Digital Experience will
be available on the Publication 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.
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
_______________________________1 Assessments were taken for
patients in Rhythm’s Phase 3 trials who experienced weight loss of
at least five kilograms (or at least five percent of baseline body
weight for the patients who weighed less than 100 kg). QOL was
assessed using the Impact of Weight on Quality of Life-Lite
(IWQOL-Lite) scale for patients 18 years or older, and using the
Pediatric Quality of Life Inventory (PedsQL) for children and
adolescents aged 8 to 12 and 13 to 17 years, respectively.2 The
64.5% figure represents a weighted yield from 8,599 URO samples
collected as of July 12, 2021. Prior to May 2021, Rhythm’s URO
panel tested for variants in 40 obesity-related genes, including 11
genes eligible for the DAYBREAK or EMANATE trials; data for those
11 genes is available in all 8,599 samples. Rhythm launched URO
2.1/3.0 in early May 2021, which now sequences 79 obesity-related
genes and the 16p11.2 chromosomal region, including 25 additional
DAYBREAK/EMANATE genes. Data on all 79 genes (including all 36
DAYBREAK/EMANATE genes) was available for 788 patients, and then
used to calculate a weighted yield across the total study
population.3 Data on the frequency of BBS and ALMS1 variants were
collected from URO samples as of July 5, 2021, which included a
smaller sample size of 8,459 sequenced individuals.
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