Carmine Therapeutics Inc. said Tuesday that Takeda Pharmaceutical Co. Ltd. has engaged it in a research collaboration aimed at the discovery, development and commercialization of new nonviral gene therapies for two rare disease targets.
Viiv Healthcare Ltd., late Thursday, won FDA approval for Rukobia (fostemsavir), a gp120-directed attachment inhibitor for the treatment of adults with multidrug-resistant HIV-1 infection failing their current antiretroviral regimen due to resistance, intolerance or safety considerations. The drug was reviewed under FDA's fast track and breakthrough therapy status programs.
The failure of an experimental refractory chronic cough drug to yield statistically significant reductions in placebo-adjusted cough frequency sent shares of its developer, Bellus Health Inc. (NASDAQ:BLU), down 71.7% to $3.40 on July 7.
Shares of Sweden-based Hansa Biopharma AB (NASDAQ:HNSA) jumped 32.7% to SEK53.90 (US$5.81) on July 2 as Sarepta Therapeutics Inc. became the first partner to license its lead asset, imlifidase, to enable gene therapies to treat muscular dystrophy patients harboring neutralizing antibodies to the adeno-associated virus (AAV) vectors the medicines employ.
CSL Behring has licensed global rights to Uniqure NV's late-stage hemophilia B gene therapy candidate, AMT-061, for $450 million up front, plus up to $1.6 billion in potential milestone payments and royalties. Uniqure positioned the deal as a boon for its broader gene therapies pipeline, anchored by a Huntington's disease (HD) program. But the move appeared to sour investors hoping for a rumored near-term M&A takeout, sending company shares (NASDAQ:QURE) falling 21.8% to $49.22 on June 25.
Carmine Therapeutics Inc. said Tuesday that Takeda Pharmaceutical Co. Ltd. has engaged it in a research collaboration aimed at the discovery, development and commercialization of new nonviral gene therapies for two rare disease targets. Terms of the deal included an up-front payment of undisclosed value for Carmine, plus research funding, and more than $900 million in potential milestone payments.
Shares of Kiniksa Pharmaceuticals Ltd. hit a 52-week high on June 29 as pivotal phase III results showed that Arcalyst (rilonacept), a medicine it licensed from Regeneron Pharmaceuticals Inc., led to a 96% reduction in risk of recurrent pericarditis events among 61 people not helped by standard-of-care treatments.
Artiva Biotherapeutics Inc., of San Diego, has secured $78 million in series A financing to support its development of off-the-shelf universal NK cells for use in combination with monoclonal antibody therapy and tumor-targeting CAR-NK cell therapies.
Prevailing after an initial complete response letter from the FDA, Chiasma Inc. on June 26 has finally secured U.S. approval for Mycapssa, the first oral formulation of the acromegaly medicine octreotide to get a regulatory green light. Octreotide has until now only been available by injection. The drug was approved for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide, both somatostatin analogues (SSAs).
Prevailing after an initial complete response letter from the FDA, Chiasma Inc. on Friday has finally secured U.S. approval for Mycapssa, the first oral formulation of the acromegaly medicine octreotide to get a regulatory green light.
