BOSTON, Jan. 04, 2024 (GLOBE NEWSWIRE) — 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 provided a business update and announced it has completed screening for enrollment in the pivotal, Phase 3 clinical trial evaluating setmelanotide in patients with acquired hypothalamic obesity.
“2023 was a truly transformational year for Rhythm, as we made excellent progress with the execution of our IMCIVREE® (setmelanotide) commercial plan while also advancing important development efforts to expand the number of patients living with hyperphagia and severe obesity that could potentially benefit from this therapy,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We believe the rapid over enrollment in less than one year and execution of our Phase 3 hypothalamic obesity trial is a strong indicator of the unmet need for an effective therapy for patients who have no approved therapeutic options and the community’s excitement around setmelanotide’s potential to make a meaningful difference in their lives.”
Rhythm also announced today that the federal health government in Spain has approved reimbursement for IMCIVREE® (setmelanotide) for the treatment of obesity and hunger control related to Bardet-Biedl syndrome (BBS) or biallelic proopiomelanocortin (POMC). ), a subtilisin protein. /kexin convertase type 1 (PCSK1) or biallelic leptin receptor (LEPR) deficiency.
“We are pleased that the Ministry of Health in Spain has recognized the treatment needs of patients with hyperphagia and early-onset obesity of BBS and POMC/LEPR deficiencies and IMCIVREE as the first and only precision medicine for patients with these diseases,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm. “We are excited to achieve access in this important market and deliver IMCIVREE to patients and families.”
BBS and POMC deficiencies, PCSK1, and LEPR are rare diseases with characteristic symptoms of hyperphagia, pathological starvation leading to abnormal foraging behaviors, and severe, early-onset obesity. BBS is also linked to cognitive impairment, polydactyly, kidney dysfunction, hypogonadism, and visual impairment. POMC deficiency or LEPR are ultra-rare diseases with very few identified patients. With multiple symptoms as components of a syndrome, patients with BBS, still underdiagnosed, are more easily identified. Rhythm estimates the prevalence of BBS at around 700 people in Spain with around one hundred patients identified.
About Rhythm Pharmaceuticals Rhythm is a commercial-stage biopharmaceutical corporation 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 FDA for chronic weight management in adult and pediatric patients aged 6 years and older with chronic obesity. monogenic or syndromic due to proopiomelanocortin. (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency was demonstrated by genetic testing or in patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). The European Commission (EC) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) have made melanotide legal for the treatment of obesity and hunger control related to genetically shown BBS or biallelic POMC in genetically shown loss of function , adding PCSK1. Array LEPR deficiency. or biallelic deficiency in adults and young people aged 6 years and older. Additionally, Rhythm is advancing an extensive clinical progression program for setmelanotide in other rare diseases, as well as a preclinical set of small molecules for the treatment of congenital hyperinsulinism. Rhythm is based in Boston, MA.
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 through an FDA-approved control demonstrating POMC variants. PCSK1 or LEPR matrix that are interpreted as pathogenic, probably pathogenic or of doubtful importance (VUS) or BBS.
In the European Union, setmelanotide is indicated for the treatment of obesity and hunger control related to genetically proven Bardet-Biedl syndrome (BBS) or genetically proven biolelanocortin biopiomelanocortin (POMC), adding PCSK1, a biallelic leptin deficiency. LEPR receptor deficiency (LEPR) in adults and youth 6 years and older.
In the United States and Europe, setmelanotide will need to be prescribed and supervised by a doctor who is an expert in obesity with an underlying genetic etiology.
WARNINGS AND PRECAUTIONS
Skin monitoring: Setmelanotide would possibly cause widespread accumulation of skin pigmentation and darkening of pre-existing nevi due to its pharmacological effect. Full-body skin examinations need to be performed annually to monitor for new and pre-existing pigmented skin lesions before and remedies with setmelanatide.
Heart rate and blood pressure monitoring: Heart rate and blood pressure will be monitored as a component of popular clinical practice on a medical scale (at least at one and both 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 that lasts longer than four hours deserve to be treated to seek emergency medical attention for a possible priapism remedy.
Paediatric population: The prescribing physician should periodically assess the reaction to treatment with setmelanotide. In developing children, the effect of weight loss on growth and maturation should be assessed. The prescribing physician should monitor growth (height and weight) using age- and sex-appropriate expansion charts.
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 medication contains less than 1 mmol of sodium (23 mg) depending on the dose, which is necessarily “sodium-free. “
The most common side effects were hyperpigmentation (51%), injection reaction (39%), nausea (33%) and headache (26%).
Pregnancy There is no knowledge about the use of setmelanotide in pregnant women. Animal studies do not imply direct adverse effects with respect to reproductive toxicity. However, management of setmelanotide in pregnant rabbits resulted in a minimization of maternal food intake, resulting in embryo-fetal effects. As a precautionary measure, pregnancy should not be started with setmelanotide or when trying to become pregnant, as weight loss during pregnancy could harm the fetus. If a patient taking setmelanotide has reached a solid weight and becomes pregnant, attention should be paid to treatment with setmelanotide as there is no evidence of teratogenicity in non-clinical knowledge. If a patient who is taking setmelanotide and continues to lose weight becomes pregnant, treatment with setmelanotide should be discontinued or the dose decreased. reduce while tracking recommended weight gain during pregnancy. The attending physician should closely monitor the pregnancy weight in a patient taking setmelanatide.
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.
For SUSPECTED ADVERSE REACTIONS, please contact Rhythm Pharmaceuticals at 1 (833) 789-6337. Refer to the summary of product characteristics in ANNEX V for a list of European national adverse reaction systems.
Forward-Looking Statements This press release includes 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 past facts deserve to be considered forward-looking statements. , adding, without limitation, statements relating to the prospective, safety, efficacy and regulatory and clinical progress of setmelanotide, adding the expected timeline for the initiation of clinical trials and the publication of knowledge from clinical trials and our related expectations with the possible regulatory submissions, approvals and schedule thereof, adding RM -718 and setmelanotide, our advertising strategy and plans, adding what is related to the advertising of setmelanotide in the United States and Europe, the application of genetic tests and the prospective of relevant expansion, as well as expectations related to possible market opportunities. for our product candidates. Statements that use words such as “expect,” “anticipate,” “believe,” “may,” “will,” “target” and similar terms are also forward-looking statements. These statements are subject to dangers and uncertainties, including, but not limited to, dangers related to our liquidity and expenses, our ability to enroll patients in clinical trials, the design of the trials and their results. clinical trials, the ability to discharge mandatory regulatory approvals, relevant pitfalls with research and knowledge reporting, inability to identify and expand other product candidates, unfavorable pricing regulations, third-party reimbursement practices or care reform initiatives medical, dangers relevant to the legislation and regulations governing our foreign countries. operations and pricing of any relevant compliance program. Array the effect of competition, dangers similar to liskill’s product claims, the inability to maintain our collaborations, or the failure of those collaborations, our dependence on third parties, dangers similar to intellectual property, our ability to hire and retain personnel mandatory, the effect of the COVID-19 pandemic and general economic situations on our business and operations, adding our preclinical studies, clinical trials and advertising, clients are unable to realize the expected benefits of our acquisition of Q for the quarter ended on September 30, 2023 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no legal responsibility to revise or update any forward-looking statements contained in this release to reflect occasions or events arising after the date of this release, whether as a result of new information or long-term developments. . in another way.
Corporate Contact:David ConnollyExecutive Director, Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, [email protected]
Media Contact: Adam DaleyBerry