The bad news in March 2022 from Takeda Pharmaceutical Co. Ltd. regarding Natpara, its recombinant human parathyroid hormone for hypoparathyroidism (HPT), served to generate more interest in the already bubbling space, where a handful of players large and small own prospects at various clinical stages.
Structure Therapeutics Inc. raised $33 million in a financing round to speed up clinical trials of its lead assets targeting chronic diseases and to improve its technology platform. Previously known as Shouti Inc., the company also has rebranded itself as Structure to “reflect its foundation in structural biology and computational design.”
The U.S. FDA has placed a clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating AT-845 following a serious adverse event of peripheral sensory neuropathy in one of the trial participants. AT-845 is an adeno-associated virus (AAV) gene replacement therapy being studied in adults with late-onset Pompe disease.
Abliva AB has gone above its market capitalization to raise the money it needs to start a phase II/III trial of KL-1333 for the treatment of primary mitochondrial disease.
Arrowhead Pharmaceuticals Inc. and Vivo Capital LLC have launched a joint venture named Visirna Therapeutics for RNA interference therapeutics in the greater China market. Arrowhead is the majority shareholder of the new entity, while Vivo invested $60 million in the new entity.
Sanofi SA’s enzyme replacement therapy, Xenpozyme (olipudase alfa), has been approved for use in Japan, making it the world’s first and only approved therapy to treat acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick type B disease. Sanofi’s executive vice president and global head of R&D, John Reed, hailed it as a “watershed moment” that was the culmination of 20 years of research.
I-Mab Biopharma Co. Ltd. formed a partnership with Hubei Jumpcan Pharmaceutical Co. Ltd. in a deal worth up to ¥2.016 billion (US$315.2 million) to develop, manufacture and commercialize recombinant human growth hormone (rhGH) eftansomatropin alfa (TJ-101) in mainland China.
In its first big pharma deal since it was founded around a cell programming technology in 2009, Immusoft Corp. signed Takeda Pharmaceutical Co. Ltd. to a research collaboration and license option targeting rare inherited metabolic disorders. The agreement brings an undisclosed up-front fee and research funding to Immusoft, which is also eligible to earn more than $900 million if all options are exercised and all milestones hit.
Scohia Pharma Inc. has secured an agreement with Huadong Medicine Co. Ltd., enabling the former to tap the greater China market for SCO-094, its GLP-1R and GIPR dual agonist.
HONG KONG – Canbridge Pharmaceuticals Inc. signed a collaboration and licensing agreement that could be worth $591 million, gaining global rights to develop, manufacture and commercialize gene therapy candidates from Logicbio Therapeutics Inc. for the treatment of Fabry and Pompe diseases. The candidates are based on Logicbio’s adeno-associated virus (AAV) sL65, the first produced from its Saavy capsid development platform.
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