Rhythm Pharmaceuticals and Axovia Therapeutics Announce Joint Research Collaboration in Bardet-Biedl Syndrome
Rhythm Pharmaceuticals (RYTM) and Axovia Therapeutics announced a joint research collaboration to advance the understanding of Bardet-Biedl syndrome (BBS). The partnership combines Rhythm's expertise in melanocortin-4 receptor pathway research and their commercially available drug for treating hyperphagia and severe obesity in BBS patients with Axovia's focus on gene therapy addressing loss of sight and obesity in BBS patients. The collaboration aims to pool knowledge from their respective screening efforts to improve patient care.
Rhythm Pharmaceuticals (RYTM) e Axovia Therapeutics hanno annunciato una collaborazione di ricerca congiunta per approfondire la comprensione della sindrome di Bardet-Biedl (BBS). La partnership combina l'expertise di Rhythm nella ricerca sul percorso del recettore melanocortin-4 e il loro farmaco disponibile in commercio per il trattamento della iperfagia e dell'obesità severa nei pazienti affetti da BBS con il focus di Axovia sulla terapia genica per affrontare la perdita della vista e l'obesità nei pazienti con BBS. L'obiettivo della collaborazione è mettere insieme le conoscenze derivanti dai rispettivi sforzi di screening per migliorare la cura dei pazienti.
Rhythm Pharmaceuticals (RYTM) y Axovia Therapeutics anunciaron una colaboración en investigación conjunta para avanzar en la comprensión del síndrome de Bardet-Biedl (BBS). La asociación combina la experiencia de Rhythm en la investigación de la vía del receptor melanocortina-4 y su medicamento disponible comercialmente para tratar la hiperfagia y la obesidad severa en pacientes con BBS, con el enfoque de Axovia en la terapia génica que aborda la pérdida de visión y la obesidad en pacientes con BBS. La colaboración tiene como objetivo unir conocimientos de sus respectivos esfuerzos de cribado para mejorar la atención al paciente.
리듬 제약 (RYTM)와 악소비아 치료제가 바르데-비들 증후군 (BBS)에 대한 이해를 높이기 위해 공동 연구 협력을 발표했습니다. 이 파트너십은 리듬 제약의 멜라노코르틴-4 수용체 경로 연구에 대한 전문성과 BBS 환자의 과식증 및 심각한 비만 치료를 위한 상용 약물을 Axovia의 유전자 요법에 대한 집중과 결합합니다. 이 협력의 목표는 각자의 스크리닝 노력에서 얻은 지식을 모아 환자 치료를 개선하는 것입니다.
Rhythm Pharmaceuticals (RYTM) et Axovia Therapeutics ont annoncé une collaboration de recherche conjointe pour faire avancer la compréhension du syndrome de Bardet-Biedl (BBS). Ce partenariat combine l'expertise de Rhythm dans la recherche sur la voie du récepteur mélanocortine-4 et leur médicament disponible commercialement pour traiter l'hyperphagie et l'obésité sévère chez les patients atteints de BBS, avec le focus d'Axovia sur la thérapie génique pour traiter la perte de vision et l'obésité chez les patients atteints de BBS. L'objectif de cette collaboration est de regrouper les connaissances issues de leurs efforts de dépistage respectifs pour améliorer les soins aux patients.
Rhythm Pharmaceuticals (RYTM) und Axovia Therapeutics haben eine gemeinsame Forschungszusammenarbeit angekündigt, um das Verständnis des Bardet-Biedl-Syndroms (BBS) voranzubringen. Die Partnerschaft kombiniert Rhythms Fachwissen auf dem Gebiet der Forschung zum Melanocortin-4-Rezeptorweg und deren kommerziell verfügbarem Medikament zur Behandlung von Hyperphagie und schwerer Fettleibigkeit bei BBS-Patienten mit Axovias Fokus auf Gentherapie zur Bekämpfung von Sehverlust und Fettleibigkeit bei BBS-Patienten. Ziel der Zusammenarbeit ist es, das Wissen aus ihren jeweiligen Screening-Bemühungen zu bündeln, um die Patientenversorgung zu verbessern.
- Strategic partnership combining complementary expertise in BBS research
- Potential to expand understanding and treatment options for BBS patients
- Collaboration with leading BBS expert Prof. Phil Beales
- None.
Insights
This research collaboration between Rhythm Pharmaceuticals and Axovia Therapeutics represents a strategic partnership in the rare disease space, specifically targeting Bardet-Biedl syndrome (BBS). The partnership combines Rhythm's expertise in melanocortin-4 receptor pathway treatments with Axovia's gene therapy approach. Rhythm's current BBS treatment for hyperphagia and obesity complements Axovia's focus on vision loss, potentially leading to more comprehensive treatment options.
The collaboration's focus on disease understanding and epidemiology could expand the identified patient population, potentially increasing the market opportunity for both companies' treatments. However, this is primarily a research-stage collaboration without immediate revenue implications or specific financial commitments disclosed.
BOSTON and LONDON, Oct. 24, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM) and Axovia Therapeutics Ltd. today announced a joint research collaboration designed to advance the understanding of Bardet-Biedl syndrome (BBS).
“We believe there is a significant need and opportunity with the growing awareness of BBS to better understand the disease and its epidemiology,” said Prof. Phil Beales, Axovia Therapeutics’ Chief Executive Officer and Co-Founder. “At Axovia, we are utilizing gene therapy to address the loss of sight and obesity experienced by patients with BBS, and we are pleased to be collaborating with Rhythm, a global company that has pioneered research in the melanocortin-4 receptor pathway and developed the first and only commercially available drug to help treat hyperphagia and severe obesity in patients living with BBS. Rhythm and Axovia Therapeutics are natural collaborators in such an effort.”
“Prof. Beales is a leading expert on BBS and was involved in creating the original diagnostic criteria for patients living with this disease,” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. “We are excited to work together with Axovia Therapeutics to advance our shared understanding of the disease and pool the knowledge we have gained from our respective screening efforts with the goal of further improving the lives of patients and their families living with BBS.”
About Bardet-Biedl syndrome (BBS)
BBS is a rare autosomal recessive ciliopathy that presents with a variety of signs and symptoms that evolve over time including visual impairment, renal disease, polydactyly, genital abnormalities, cognitive impairment, 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 Axovia Therapeutics
Axovia Therapeutics is leading the development of therapies that address the genetic causes of blindness and obesity which are driven by ciliopathies. Ciliopathies are a group of more than 50 inherited genetic diseases linked to more than 950 genes that impact the function of cilia which are critical for protein transport and cellular signaling. The company is positioned to initiate clinical studies for its lead program for Bardet-Biedl Syndrome (BBS), AXV-101, in mid-2025 based on robust preclinical data, scaled manufacturing and established patient registries. The initial subretinal study is designed to halt photoreceptor cell death and retinal degeneration and the CNS delivery program, which will begin in 2026, will seek to address hyperphagia and obesity. AXV-101 has achieved U.S. Food and Drug Administration Orphan Drug Designation and Rare Pediatric Disease Designation. Axovia was formed following decades of work on ciliopathies at University College London by co-founders Professor Phil Beales and Dr. Victor Hernandez.
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients 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. The EC has also authorized setmelanotide for control of hunger and treatment of obesity in children as young as 2 years old, living with BBS or POMC, PCSK1, or LEPR deficiency. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists LB54640 and RM-718, and a preclinical suite of small molecules 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 BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. In Europe, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
Limitations of Use
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.
Contraindication
Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported.
WARNINGS AND PRECAUTIONS
Skin Pigmentation and Darkening of Pre-Existing Nevi: Generalized increased skin pigmentation and darkening of pre-existing nevi have occurred because of its pharmacologic effect. Full body skin examinations prior to initiation and periodically during treatment should be conducted to monitor pre-existing and new pigmentary lesions.
Heart rate and blood pressure monitoring: In Europe, 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.
Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Patients who have an erection lasting longer than 4 hours should seek emergency medical attention.
Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Patients should be monitored for new onset or worsening depression or suicidal thoughts or behaviors. Consideration should be given to discontinuing setmelanotide if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur.
Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue setmelanotide.
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. In Europe, the prescribing physician should monitor growth (height and weight) using age- and sex-appropriate growth curves.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: Setmelanotide is not approved for use in neonates or infants. Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and low birth weight infants treated with benzyl alcohol-preserved drugs.
ADVERSE REACTIONS
Most common adverse reactions (incidence ≥
USE IN SPECIFIC POPULATIONS
Lactation: Not recommended when breastfeeding.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe.
Please see the full U.S. Prescribing Information and EU Summary of Product Characteristics 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, potential regulatory submissions, approvals and timing thereof of setmelanotide and our other product candidates, and our business strategy and plans, the potential benefits of a joint research collaboration between Rhythm and Axovia Therapeutics, and the potential of Rhythm’s and Axovia Therapeutics’ drug candidates to provide treatment options for patients with BBS. Statements using words 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 ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the three months ended June 30, 2024 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 Contacts:
Corporate:
David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com
Media:
Sheryl Seapy
Real Chemistry
(949) 903-4750
sseapy@realchemistry.com
Axovia Contact:
Professor Phil Beales
Chief Executive Officer
investors@axovia.com
FAQ
What is the purpose of Rhythm Pharmaceuticals (RYTM) and Axovia Therapeutics' collaboration?
What treatments do Rhythm Pharmaceuticals (RYTM) and Axovia Therapeutics offer for BBS?
What is the focus of Axovia Therapeutics' research in BBS treatment?
