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RareStone to seek marketing authorization for IMCIVREE to treat obesity due to biallelic POMC, PCSK1 and LEPR deficiencies and Bardet-Biedl and Alström syndromes in mainland China, Hong Kong and Macau –
BOSTON, December 2, 2021 -- RareStone Group, a rare disease focused company aiming to establish the first rare disease ecosystem in China, Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced an exclusive licensing agreement for the development and commercialization of IMCIVREE
® (setmelanotide) in Greater China, including mainland China, Hong Kong and Macau. This licensing agreement is designed to accelerate patient access to IMCIVREE where there remains significant unmet need to address the severe, early-onset obesity and hyperphagia that characterize genetic and acquired diseases of the POMC-melanocortin pathway.
According to the terms of the agreement, RareStone will seek local approvals to commercialize IMCIVREE for the treatment of obesity and hyperphagia due to biallelic proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency, as well as Bardet-Biedl and Alström syndromes. Additionally, RareStone will fund efforts to identify and enroll patients from Greater China in Rhythm’s global EMANATE trial, a Phase 3, randomized, double-blind, placebo-controlled trial (with five independent sub-studies) to evaluate setmelanotide in patients with obesity due to a heterozygous variant of POMC/PCSK1 or LEPR; certain variants of the SRC1 gene, certain variants of the SH2B1 gene, or PCSK1 N221D deletions within the MC4R pathway.
“There is a significant need in China for a therapeutic option to treat patients with early-onset, severe obesity and hyperphagia caused by variants in genes of the POMC-melanocortin pathway,” said Shawn Xiang, Ph.D., CEO of RareStone. “A recent, non-interventional genotyping study in China showed that 2.6% of 4,000 patients with obesity had a genetic variant with known relevance to obesity, and we look forward to building on that study with several key providers of genetic testing in China. In addition, we are eager to deliver the proven clinical benefit of IMCIVREE to patients in China and plan to pursue local approvals rapidly in five initial indications, while supporting Rhythm’s ongoing clinical development efforts more broadly.”
“RareStone, a company committed to treating rare diseases, is well-positioned to leverage its network of hospitals and key opinion leaders, deep regulatory experience and community-building infrastructure to advance IMCIVREE through clinical development and regulatory approvals in Greater China,” said David Meeker, M.D., Chair, Chief Executive Officer and President of Rhythm. “We are thrilled to enter into this agreement, which substantially accelerates our ability to address the needs of patients living in Greater China and potentially make IMCIVREE available to many more patients with rare genetic diseases of obesity.”
About RareStone Group
RareStone Group is dedicated to building the first rare disease ecosystem in China to improve the lives of patients and families impacted by rare diseases with comprehensive and sustainable solutions. RareStone Group consists of two business subsidiaries: Citrine Medicine and Zircon Health. RareStone Group was founded by Eight Roads Ventures, F-Prime Capital, and Vivo Capital in 2019 and closed a USD 80 million Series A financing in July 2020.
Citrine Medicine is a pharmaceutical company aiming to provide effective and affordable treatment solutions to rare disease patients in China. Citrine Medicine is building a comprehensive rare disease product platform through fast licensing and co-development to support and accelerate scalable commercialization. Citrine Medicine currently focuses on four therapeutic areas: neuroscience, endocrine & metabolic diseases, liver & kidney diseases, and rare pediatric oncology. Citrine currently has six assets in the pipeline. Among these, three assets are licensed from global partners and are now in the pre-NDA stage in China (WAKIX® indicated for narcolepsy, Alkindi® indicated for pediatric CAH, and Efmody® indicated for adult CAH); another three assets are in-house developed assets targeting LSD related diseases and are currently in IND-enabling stage in China.
Zircon Health is a Health technology company aiming to build the largest patient-centered open ecological service platform in China with the help of cutting-edge technology. Zircon Health will leverage Internet digital tools, big data, and artificial intelligence technologies to improve the awareness of rare diseases, promote disease screening and diagnosis, carry out full-cycle disease management, and explore various payment innovations, providing comprehensive and one-stop health service solutions for patients and families affected by rare diseases. Zircon Health will focus on directly engaging and empowering patients and patient organizations to help the rare disease community in China improve access to knowledge, access to care, access to payment, and access to non-treatment solutions.
For more information, visit
www.rarestonegroup.com
Corporate Contact: communications@rarestonegroup.com
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. Rhythm’s precision medicine, IMCIVREE (setmelanotide), was approved in November 2020 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 obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing and in July and September 2021, respectively, by the European Commission (EC) and Great Britain’s Medicines & Healthcare Products Regulatory Agency (MHRA) for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE is the first-ever FDA-approved and EC- and MHRA-authorized therapy for patients with these rare genetic diseases of obesity. The Company submitted a supplemental New Drug Application (sNDA) to the FDA, which was accepted for filing in September November 2021 and assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 16, 2022. Rhythm also submitted a Type II variation application to the European Medicines Agency in October 2021 seeking regulatory approval and authorization for setmelanotide to treat obesity and control of hunger in adult and pediatric patients 6 years of age and older with BBS or Alström syndrome in the European Union. Additionally, Rhythm, along with its partners, is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine, the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.
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, including the anticipated timing for initiation of clinical trials and release of clinical trial data and our expectations surrounding potential regulatory submissions, approvals and timing thereof, our business strategy and plans, including regarding commercialization of setmelanotide, our participation in upcoming events and presentations, and the sufficiency of our cash, cash equivalents and short-term investments to fund our operations. 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, the impact of our management transition, 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 liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, 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 quarter ended September 30, 2021 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.