Alnylam Pharmaceuticals Inc. said the 164-patient Helios-A phase III study with next-generation RNAi drug vutrisiran hit its primary endpoint as well as both secondary goals in the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. The primary endpoint was change from baseline in the modified Neuropathy Impairment Score at nine months as compared to historical placebo data from the Apollo phase III study of Alnylam’s Onpattro, cleared by the FDA for ATTR polyneuropathy in August 2018.
HONG KONG – With the sale of a group of noncore assets to a little-known Chinese company, Takeda Pharmaceutical Co. Ltd. took another step in a multibillion-dollar string of divestitures that is part of a plan to cut debt and focus on five key business areas. Takeda announced on Dec. 21 the sale of a portfolio of noncore prescription drugs marketed in China to Hasten Biopharmaceutic Co. Ltd. (China).
PERTH, Australia – Clinuvel Pharmaceuticals Ltd. announced two new pharmaceutical indications for its lead product, Scenesse (afamelanotide). “The company has until now focused on one drug and has slowly gathered evidence and is now in a position to add other products to its pipeline,” Clinuvel CEO Philippe Wolgen told BioWorld.
Miragen Therapeutics Inc. gained ground lost on Wall Street earlier this month and then some, with shares (NASDAQ:MGEN) closing at $1.26, up 74 cents or 142% after the company disclosed plans to take over Viridian Therapeutics Inc., conducting at the same time a private placement that will raise $91 million.
Intellia Therapeutics Inc. is looking to disrupt the transthyretin (TTR) amyloidosis (ATTR) market with NTLA-2001, its CRISPR-based treatment designed to be a potential cure for the disease. The drug, which is delivered via a lipid nanoparticle, edits the patient's DNA in vivo to create a stop codon and eliminate the expression of TTR, the protein that aggregates in ATTR patients' nervous systems and hearts, disrupting their functions.
CEO Eric Dube said Retrophin Inc. will “share more [about clinical development plans] once the deal has closed” in the fourth quarter of this year and Orphan Technologies Ltd. belongs to his firm, which has seen only animal data so far with OT-58, an enzyme replacement therapy (ERT) for classical homocystinuria (HCU).
One of 19 children dosed with an experimental gene therapy for Sanfilippo syndrome has died following the treatment, given as part of a phase II/III trial of the medicine run by its developer, Paris-based Lysogene SA. The trial has been on clinical hold since June, but the immediate cause of the death is unknown, the company said.
The regulatory path for Saniona AB’s Tesomet for treating two rare eating disorders, Prader-Willi syndrome (PWS) and hypothalamic obesity, continues to be a winding one with surprises along the way. The newest twist is pre-IND feedback from the FDA that knocked the stock down 10.5% on Oct. 9.
BEIJING – Canbridge Pharmaceuticals Inc. last week won marketing approval in China for its first rare disease drug, the mucopolysaccharidosis II therapy Hunterase (idursulfase beta injection).
PERTH, Australia – With the launch of its lead product, Scenesse (afamelanotide), in the U.S. and Europe, Melbourne-based Clinuvel Pharmaceuticals Ltd. is expanding its global footprint and widening its pipeline as it opens a new R&D center in Singapore and builds up its franchise in photo medicine.
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