BEIJING – Rare disease specialist Canbridge Pharmaceuticals Inc., of Beijing, said it is collaborating with the Horae Gene Therapy Center at the University of Massachusetts (UMass) Medical School to conduct gene therapy research with a focus on neuromuscular conditions. The move is expected to add new assets to the company’s portfolio.
Soleno Therapeutics Inc.’s phase III DESTINY PWS (C601) trial evaluating once-daily diazoxide choline controlled-release tablets for treating patients with Prader-Willi syndrome (PWS) missed its primary endpoint of change from baseline in hyperphagia, or insatiable hunger, which is the disease’s predominant syndrome.
Passed up for acquisition by former partner Retrophin Inc., Censa Pharmaceuticals Inc. has found a new home in rare disease specialist PTC Therapeutics Inc., which has agreed to pay $10 million up front for the opportunity to develop CNSA-001 (sepiapterin), a candidate for orphan metabolic diseases, starting with phenylketonuria (PKU). The proposed transaction also includes up to 850,000 shares of PTC common stock (NASDAQ:PTCT), valued around $40 million, plus additional rewards for achieving development, regulatory and commercial milestones. Shares of PTC closed at $46.91 on May 7, down $1.31.
9 Meters Biopharma Inc., of Raleigh, N.C., is newly born from the merger of Innovate Biopharmaceuticals Inc. and privately held RDD Pharma Ltd., of New York and Tel Aviv, Israel. About $22 million in new financing, led by Orbimed Advisors Ltd., and the signing of another merger into the new company of Richmond, Calif.-based Naia Rare Diseases Inc., which develops GLP-1 to treat short bowel syndrome, completes 9 Meters’ new path.
San Francisco-based RDMD Inc. netted $14 million in series A money fueling an approach designed to generate clinical evidence that will speed drug development in rare diseases.
LONDON – Evox Therapeutics Ltd. has validated its exosome drug delivery technologies in a $882 million deal with Takeda Pharmaceutical Co. Ltd., in which the partners will develop protein replacement and messengerRNA (mRNA) therapies in five rare disease indications.
LONDON – Evox Therapeutics Ltd. has validated its exosome drug delivery technologies in a $882 million deal with Takeda Pharmaceutical Co. Ltd., in which the partners will develop protein replacement and messengerRNA (mRNA) therapies in five rare disease indications. As an indicator of the potential therapeutic power of exosome delivery, the lead program in the collaboration rests on the ability of exosomes to cross the blood-brain barrier and deliver a correct copy of the NPC1 gene, to treat the inherited neurodegenerative disorder, Niemann-Pick type C.
The Rare Disease Day, which takes place at the end of February each year, is designed to focus global attention on the need for therapies to treat patients suffering from devastating rare diseases. The most recent event represented the 13th year it has been held. Over that period, research and development in the area has come a long way, and there are now 420 companies around the world that are active in developing regenerative medicines and advanced therapies for the treatment of rare diseases, according to a new report released by the Alliance for Regenerative Medicine (ARM).
Palvella Therapeutics Inc. has completed enrollment of its phase II/III trial for treating adults with pachyonychia congenita, a rare disease that eventually leads patients to a life of chronic pain and severe problems with walking.
HONG KONG – Beijing-headquartered Canbridge Pharmaceuticals Inc. has completed a $98 million series D financing round to target rare genetic diseases in China, and is considering an IPO after several rounds of financing to further pursue its goal.
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