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 two new publications detailing
the burden of hyperphagia and obesity for adult caregivers,
families and patients living with Bardet-Biedl Syndrome (BBS) based
on results of The CAREgiver Burden in BBS (CARE-BBS) study. The
research is published in the peer-reviewed journal, The Orphanet
Journal of Rare Diseases.
“BBS is characterized by hyperphagia – an
insatiable hunger that results in extreme food seeking behaviors –
and early-onset, severe obesity, both of which have broad negative
impacts on the lives of patients living with BBS and their
caregivers,” said Prof. Andrea M. Haqq, M.D., Department of
Pediatrics, Faculty of Medicine & Dentistry, University of
Alberta. “These results demonstrate that hyperphagia and severe
obesity associated with BBS negatively affect performance in
school, work and social relationships. Importantly, the findings
from the CARE-BBS study were similar across countries,
demonstrating the universality of hyperphagia and underscoring the
need for therapies that can effectively treat hyperphagia in order
to alleviate the extensive clinical and nonclinical impacts of the
disease.”
“Burden of Hyperphagia and Obesity in Bardet-Biedl
Syndrome: a Multi-country Survey,” includes results from a
cross-sectional survey of 242 adult caregivers of patients with BBS
across the United States, the United Kingdom, Canada and Germany.
Caregivers of patients with BBS reported that hyperphagia has broad
impacts, well beyond contributing to obesity. Caregivers observed
hyperphagic behaviors throughout the day, most frequently
negotiating for food (90%) and waking up and asking or looking for
food during the night (88%). Hyperphagia had at least a moderate
negative impact on most patients’ mood/emotions (56%), sleep (54%),
school (57%), leisure (62%), and familial relationships (51%).
“Caregiver Burden in Bardet-Biedl Syndrome:
Findings from the CARE-BBS Study,” quantifies caregiver burden
associated with obesity and hyperphagia. The findings show that
patients’ hyperphagia had a moderate-to-severe impact on caregiver
mood (56.6%), sleep (46.6%), and relationships (48.0%),
contributing to a high level of personal strain and work
productivity loss for caregivers in the workforce, as well as a
meaningful family impact.
“We continue to learn more about the full extent of
hyperphagia’s negative impact on BBS patients, their families and
caregivers,” said David Meeker, M.D., Chair, President and Chief
Executive Officer of Rhythm. “These results provide valuable
insights into the physical, social and economic burdens of
hyperphagia for both patients and their families and show the need
for therapies designed to alleviate hyperphagia and, as a result,
improve 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) is approved by
the U.S. Food and Drug Administration (FDA) and
authorized by the European Commission (EC) and the
UK’s Medicines & Healthcare Products Regulatory
Agency (MHRA) for use in accordance with product labeling.
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
proopiomelanocortin (POMC), including PCSK1, deficiency or
biallelic leptin receptor (LEPR) deficiency in adults and children
6 years of age and above.
In Canada, setmelanotide is indicated for the
treatment of obesity due to Bardet-Biedl syndrome (BBS) or
genetically-confirmed biallelic pro-opiomelanocortin (POMC),
proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin
receptor (LEPR) deficiency due to variants interpreted as
pathogenic, likely pathogenic, or of uncertain significance in
adults and children 6 years of age and above.
Limitations of UseSetmelanotide 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.
In Europe, Setmelanotide should be prescribed
and supervised by a physician with expertise in obesity with
underlying genetic etiology.
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 REACTIONS
The 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-fetal 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 Statements
This 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 for patients with
BBS or POMC, PCSK1 and LEPR deficiencies, and the potential
positive impacts of setmelanotide therapy for caregivers of
patients with BBS. 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, risks
relating to our liquidity and expenses, our ability to enroll
patients in clinical trials, the design and outcome of clinical
trials, the ability to achieve necessary regulatory approvals,
risks associated with data analysis and reporting, failure to
identify and develop additional product candidates, unfavorable
pricing regulations, third-party reimbursement practices or
healthcare reform initiatives, risks associated with the laws and
regulations governing our international operations and the costs of
any related compliance programs, the impact of competition, risks
relating to product liability lawsuits, inability to maintain our
collaborations, or the failure of these collaborations, our
reliance on third parties, risks relating to intellectual property,
our ability to hire and retain necessary personnel, 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 the other
important factors discussed under the caption “Risk Factors” in our
Quarterly Report on Form 10-Q for the three months ended March 31,
2023 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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