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 it has entered into a
global licensing agreement with LG Chem, Ltd. ("LG Chem"), a
leading global company headquartered in South Korea that
specializes in life sciences as one of its core businesses, for
LB54640, an investigational oral small molecule melanocortin-4
receptor (MC4R) agonist now in Phase 2 clinical trials.
“We are excited to build upon the strong chemistry and early
translational work performed by LG Chem, a highly regarded company
with deep and broad experience and expertise in the pharmaceutical
business. The development of an effective oral therapy for treating
MC4R pathway diseases has been a major goal for the industry and
the early data from LG Chem suggest they have identified a specific
therapy for the MC4R without hyperpigmentation or cardiovascular
side effects,” said David Meeker, MD, Chair, Chief Executive
Officer and President of Rhythm Pharmaceuticals. “We believe
Rhythm’s deep developmental experience and global commercial
presence uniquely positions us to move this molecule forward with
the goal of offering a full portfolio of treatment options to
patients struggling with hyperphagia and severe obesity and
ensuring they get the treatment that is right for them.”
In a Phase 1 trial in healthy overweight adults, LB54640
demonstrated dose-dependent weight reduction. LB54640 also
demonstrated a favorable safety profile in the trial, with no
changes in blood pressure or heart rate observed and no
hyperpigmentation observed. In addition, LB54640 has received
orphan drug designation from the U.S. Food and Drug Administration
for leptin receptor (LEPR) deficiency and
proopiomelanocortin(POMC).
Dr. Jeewoong Son, President of LG Chem Life Sciences Company,
said, “Rhythm has pioneered the development and global
commercialization of the first and best-in-class therapy for
patients with hyperphagia and severe obesity associated with
diseases like Bardet-Biedl syndrome (BBS), hypothalamic obesity and
certain genetically-defined pathway diseases. We believe Rhythm is
the ideal partner because of its extensive genetic obesity
database, global network of clinical trial sites and investigators,
a track record of regulatory successes and - most importantly -
because Rhythm values patients and addresses their urgent needs for
care.”
Under the terms of the agreement, Rhythm will assume sponsorship
of two Phase 2 studies designed to evaluate weight loss efficacy,
safety, tolerability and pharmacokinetics of LB54640. The SIGNAL
trial is a randomized, placebo-controlled, double-blind study
designed to enroll and evaluate approximately 28 patients with
acquired hypothalamic obesity. Participants will receive one of
three doses of LB54640 by oral administration once daily for up to
52 weeks, and the primary endpoint of the study is the change from
baseline in body mass index after 14 weeks of treatment. The
open-label, single-arm, 52-week ROUTE trial is designed to enroll
five patients with POMC, LEPR, or proprotein convertase
subtilisin/kexin type 1 (PCSK1) deficiency obesity. Participants
will receive LB54640 by oral administration once daily for up to 52
weeks, and the primary endpoint of the study is the change from
baseline in body mass index after 14 weeks of treatment.
Under the terms of the agreement, Rhythm has agreed to pay LG
Chem $40 million in cash and $20 million in Rhythm equity at
closing and an additional $40 million in cash 18 months after
closing.
Also under the terms of the agreement, Rhythm has agreed to pay
LG Chem up to $205 million upon achieving certain regulatory and
sales milestones, as well as royalties.
About LG ChemLG Chem is a leading global
chemical company with a diversified business portfolio in the key
areas of petrochemicals, advanced materials, and life sciences. The
company manufactures a wide range of products from high-value added
petrochemicals to renewable plastics, specializing in cutting-edge
electronic and battery materials, as well as drugs and vaccines to
deliver differentiated solutions for its customers. LG Chem Life
Sciences develops, manufactures, and globally commercializes
pharmaceutical products, with a focus on Oncology, Immunology, and
Metabolic diseases. Our mission is to transform people’s lives
through inspiring science and leading innovation. For more
information, please visit www.lgchem.com.
About Rhythm PharmaceuticalsRhythm 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 for setmelanotide in other rare diseases, as well as RM-718
and a preclinical suite of small molecules 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.
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-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
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 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.
Rhythm Corporate
Contact:David ConnollyExecutive Director, Investor
Relations and Corporate CommunicationsRhythm Pharmaceuticals,
Inc.857-264-4280dconnolly@rhythmtx.com
Rhythm Media
Contact:Adam DaleyBerry & Company Public
Relations212-253-8881adaley@berrypr.com
LG Chem Media
Contact:Son Junil / Kim Junamlgchempr@lgchem.com
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