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 the publication of interview-based
patient and caregiver reported experiences of hunger and quality of
life with setmelanotide treatment in Bardet-Biedl syndrome (BBS).
The research is published in the peer-reviewed journal Advances in
Therapy.
“Hyperphagia and early-onset, severe obesity are hallmark
characteristics of BBS that place a considerable burden on patients
and their families,” said Prof. Andrea M. Haqq, M.D., Department of
Pediatrics, Faculty of Medicine & Dentistry, University of
Alberta. “Our results show that hyperphagia – a pathological,
all-consuming hunger leading to an obsessive focus on food –
negatively affects the lives of patients and families, posing
difficulties with concentration, emotional and physical
manifestation, and impaired relationships. Our analyses also
demonstrate that setmelanotide therapy resulted in meaningful
improvements for these patients, such as weight loss and a decrease
in obsessive focus on food and food-seeking behaviors. Further,
these interviews exemplify the usefulness of patient experience
data as recommended by the U.S. Food and Drug Administration for
patient-centered drug development.”
The qualitative study publication, titled, “Interview-Based
Patient- and Caregiver-Reported Experiences of Hunger and Improved
Quality of Life With Setmelanotide Treatment in Bardet-Biedl
Syndrome,” includes patients with BBS or their caregivers who
participated in Phase 2 and 3 clinical trials of setmelanotide. A
total of 19 interviews were conducted with patients (n=8) and
caregivers (n=11) to explore patient experience and caregiver
observations of hyperphagia before and during setmelanotide
treatment. Before setmelanotide treatment, most patients (n=7;
87.5%) and caregivers (n=10; 90.9%) experienced negative effects
directly related to hyperphagia. In addition, most participants (15
of 19 overall [78.9%]; 5 of 8 patients [62.5%] and 10 of 11
caregivers [90.9%]) described a lack of control with eating.
All participants reported substantial improvements in
hyperphagia and satiety as well as weight loss after initiating
setmelanotide treatment, including improved focus and concentration
related to reductions in obsessive behaviors associated with food.
All caregivers noted the ability to give their child greater
autonomy around food choices and consumption and a reduced need to
monitor their child’s food intake. Approximately half of patients
and caregivers noted that improvements in hunger had benefited
family dynamics.
“Until now, the impact of hyperphagia on the lives of these
patients and their families has not been fully understood. This
study confirms the substantial burden and provides additional
insights into how treatment with setmelanotide facilitated
improvements in hyperphagia and promoted beneficial changes in
patient and caregiver experiences,” said David Meeker, M.D., Chair,
President and Chief Executive Officer of Rhythm.
Rhythm also announced the publication of a study, titled,
“Health State Utilities Associated With Hyperphagia: Data for Use
in Cost-Utility Models,” that evaluated the assessment of the
substantial impact of severe hyperphagia on patients’ quality of
life. This study is the first to estimate the impact of hyperphagia
on health state utilities independently of any specific underlying
indication. These data can be incorporated into cost-utility models
conducted to assess the value of treatments for rare MC4R pathway
diseases. Data published in the open-access journal Obesity Science
and Practice show that increasing severity of hyperphagia is
associated with profound impacts on quality of life that are
comparable to other severe health states, such as stroke and
progressive metastatic cancers, which similarly have a broad impact
on many aspects of quality of life.
About Bardet-Biedl SyndromeBardet-Biedl
syndrome (BBS) is a heterogenous rare genetic syndromic disease
that presents with a variety of symptoms that evolve over time
including visual impairments, renal disease, polydactyly, genital
abnormalities, cognitive impairment, and hyperphagia and
early-onset, severe obesity arising from impairment of the
hypothalamic MC4R pathway. In the United States, BBS affects
approximately 4,000 to 5,000 individuals with similar prevalence in
Europe.
About Rhythm PharmaceuticalsRhythm 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), an MC4R agonist designed to treat hyperphagia and
severe obesity caused by rare MC4R pathway diseases, is approved 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 monogenic or syndromic obesity due to pro-opiomelanocortin
(POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or
leptin receptor (LEPR) deficiency confirmed by genetic testing, or
patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS).
Both the European Commission (EC) and the UK’s Medicines &
Healthcare Products Regulatory Agency (MHRA) have authorized
setmelanotide for the treatment of obesity and the control of
hunger associated with genetically confirmed BBS or genetically
confirmed loss-of-function biallelic POMC, including PCSK1,
deficiency or biallelic LEPR deficiency in adults and children 6
years of age and above. 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 IndicationIn the United
States, setmelanotide is indicated for chronic weight management in
adult and 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)
or 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 pro-opiomelanocortin (POMC),
including PCSK1, deficiency or biallelic leptin receptor (LEPR)
deficiency in adults and children 6 years of age and above.
Limitations of UseIn the United
States and Europe, Setmelanotide should be prescribed and
supervised by a physician with expertise in obesity with underlying
genetic etiology.
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.
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 REACTIONSThe most frequent adverse
reactions are hyperpigmentation (51%), injection site reaction
(39%), nausea (33%), and headache (26%).
USE IN SPECIFIC POPULATIONS
PregnancyThere 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-foetal 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-feedingIt 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.
FertilityNo 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
StatementsThis press release contains forward-looking
statements within the meaning of the U.S. 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,
the potential benefits of setmelanotide for patients, including
those with BBS, and our business strategy and plans, including
regarding commercialization of setmelanotide. 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 and general
economic conditions 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 quarter ended December 31,
2022 and our other filings with the U.S. 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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