Wall Street took in stride mixed FDA briefing documents with regard to the upcoming adcom review of Bluebird Bio Inc.’s two gene therapy prospects, and shares of the company (NASDAQ:BLUE) closed at $3.61, up 63 cents, or 21%.
Shares of Aeglea Biotherapeutics Inc. tumbled 51% June 2 on news that it received a refusal to file (RTF) letter from the U.S. FDA regarding the BLA for pegzilarginase for the treatment of arginase 1 deficiency, with the agency requesting additional efficacy data.
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.
Swanbio Therapeutics Inc. closed a $56 million series B round to take its lead gene therapy program, SBT-101, into clinical development later this year. The candidate, comprised of an adeno-associated virus type 9 vector encoding the ABCD1 peroxisomal ATP-binding cassette transporter, is in development for adrenomyeloneuropathy (AMN), an inherited disease that affects the central nervous system.
It was a patient-reported outcome, one that could actually be seen in the mirror, that alerted researchers they might be on track in their phase I/II study of cystinosis. The patient noticed that for the first time in his life his hair had become darker, like his brother’s. It was all because the rare disease inhibiting the pigment in his body was being impacted by the treatment. “It’s a secondary issue, but I find it fascinating,” Avrobio Inc.’s CEO, Geoff MacKay, told BioWorld. “When you run trials like this, you stumble upon some fascinating results.”
Omass Therapeutics Ltd. has raised £75.5 million (US$94.6 million) in a series B round, as it continues to advance five small-molecule programs against previously intractable membrane-bound targets to the clinic.
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.
Drug delivery opportunities are at the heart of Halozyme Therapeutics Inc.’s nearly $1 billion acquisition of Antares Pharma Inc. The deal brings Halozyme an autoinjector platform to complement its Enhanze drug delivery technology, which is designed to reduce hours-long treatments to minutes.
The fortunes of Protalix Biotherapeutics Inc. improved dramatically with phase III results from the Fabry disease study called Balance, and a resubmission of the BLA for pegunigalsidase alfa (PRX–102) is planned for the second half of this year. In February, an MAA for PRX-102 was submitted to the EMA.
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.
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