RARE Daily

Rhythm Enters Licensing Deal with RareStone to Commercialize Rare Obesity Drug in China

December 6, 2021

Rhythm Pharmaceuticals, a company focused on treatments for rare genetic diseases of obesity, and China-based rare disease company RareStone (formerly Citrine Medicine) have entered an exclusive licensing agreement for the development and commercialization of Imcivree (setmelanotide) in China, including mainland China, Hong Kong and Macau.

Photo: David Meeker, chair, president and CEO of Rhythm

This licensing agreement marks the first expansion of Rhythm’s pipeline into Asia and is designed to accelerate patient access to Imcivree (setmelanotide) where there remains significant unmet need to address the severe, early-onset obesity and hyperphagia that characterize both acquired and genetic diseases of the melanocortin-4 receptor (MC4R) 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 China in Rhythm’s global EMANATE trial, a phase 3, randomized, double-blind, placebo-controlled trial to evaluate setmelanotide in five independent sub-studies 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.

“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 China,” said David Meeker, chair, president and CEO of Rhythm.

RareStone was founded in 2019 with funding from leading health care investors, including Eight Roads, F-Prime Capital, Vivo Capital, Quan Capital, 3H Health Investment and WU Capital. The Shanghai-based company is focused on building an ecosystem to support patients and families living with rare diseases in Greater China and has dedicated itself to improving the lives of patients with rare and intractable diseases by making diagnosis and essential treatments available and accessible to those who need them.

“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 MC4R pathway,” said Shawn Xiang, CEO of RareStone. “Rhythm’s precision medicine, Imcivree (setmelanotide), approved by FDA and authorized by the European Commission and Great Britain’s Medicines and Healthcare Products Regulatory Agency, has transformed the treatment paradigm for rare genetic diseases of obesity. 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.”

According to the terms of the licensing agreement, RareStone will make an upfront payment to Rhythm of $7 million and issue $5 million in equity to Rhythm. Rhythm will be eligible to receive development and commercialization milestones of up to $63.5 million, as well as tiered royalty payments on annual net sales of Imcivree.

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 and 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.

Author: Rare Daily Staff

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