Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global
commercial-stage biopharmaceutical company focused on transforming
the lives of patients and their families living with rare
neuroendocrine diseases, today announced that it will host a live
conference call and webcast at 8:00 a.m. ET on Thursday, February
22, 2024 to report its fourth quarter and full year 2023 financial
results and provide a corporate update.
To access the live conference call, participants may register
here. While not required, it is recommended that participants join
the call ten minutes prior to the scheduled start.
Also today, Rhythm announced that David Meeker, M.D., Chair,
President and Chief Executive Officer, will participate in fireside
chats at two upcoming conferences:
- Guggenheim’s 6th Annual Biotechnology Conference on Wednesday,
February 7, 2024 at 1:30 p.m. ET in New York, NY.
- and TD Cowen 44th Annual Health Care Conference on Tuesday,
March 5, 2024 at 2:50 p.m. ET in Boston, MA.
Live webcasts of both the earnings conference call and the
fireside chats will be available under "Events and Presentations"
in the Investor Relations section of the Rhythm Pharmaceuticals
website at http://ir.rhythmtx.com/. The archived webcast of the
conference call will be available on Rhythm’s website approximately
two hours after it concludes and will be available for 30 days
following the call. A replay of the Guggenheim and Cowen webcasts
will also be available on the Rhythm website for 30 days following
the presentations.
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed
to transforming the lives of patients and their families living
with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE®
(setmelanotide), an MC4R agonist designed to treat hyperphagia and
severe obesity, 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 that includes investigational MC4R agonists LB54640 and
RM-718, as well as a suite of preclinical candidates for the
treatment of congenital hyperinsulinism. Rhythm’s headquarters is
in Boston, MA.
Setmelanotide Indication
In 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.
Limitations of Use
In 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 REACTIONS
The most frequent adverse reactions are hyperpigmentation (51%),
injection site reaction (39%), nausea (33%), and headache
(26%).
USE IN SPECIFIC POPULATIONS
Pregnancy
There 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-feeding
It 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.
Fertility
No 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 and LB54640, the potential
timing, payments due, and benefits of the global licensing
agreement for LB54540, including with respect to the consummation
of the transaction, expectations regarding the design, enrollment,
or outcome of clinical trials of LB54640, the outcome of the Phase
2 trial of setmelanotide in hypothalamic obesity, the ability to
reach any net sales or revenue milestones, and obtaining regulatory
approvals in connection with the global licensing agreement.
Statements using word such as “expect”, “anticipate”, “believe”,
“may”, “will”, "aim" 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, failure to realize the anticipated
benefits of our acquisition of Xinvento B.V. or significant
integration difficulties related to the acquisition, and the other
important factors discussed under the caption “Risk Factors” in our
Quarterly Report on Form 10-Q for the quarter ended September 30,
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 Connolly
Executive Director, Investor Relations and Corporate
Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com
Media Contact:
Adam Daley
Berry & Company Public Relations
212-253-8881
adaley@berrypr.com
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