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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.
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. Despite the market reception, analyst reaction was largely supportive of the move, embracing CSL as a natural fit for the program and, in the words of SVB Leerink analyst Joseph Schwartz, appreciating the transfer of "any potential remaining risk in hemophilia B to CSL Behring as QURE rings the cash register."
Yumanity Therapeutics Inc. said that Merck & Co. Inc. is making a big bet on its nascent neurodegenerative disease pipeline, licensing exclusive rights to programs addressing amyotrophic lateral sclerosis (ALS) and a rare type of dementia in a deal with up to $500 million in potential milestone payments. The collaboration also includes an up-front payment, money toward a series C financing and potential royalties on net sales of the licensed programs, though the value of those elements wasn't disclosed.
A new U.K. trial evaluating drugs for hospitalized patients in the early stages of COVID-19 has elected to test Evelo Biosciences Inc.'s anti-inflammatory monoclonal microbial, EDP-1815, after an earlier trial found it led to favorable anti-inflammatory activity in psoriasis patients.
A second attempt by Nabriva Therapeutics plc to win U.S. approval for the antibiotic Contepo (fosfomycin) has been undone by COVID-19. Manufacturing issues stymied the original NDA. This time, restrictions of FDA staff travel to Europe sunk the venture, leading to a new complete response letter (CRL) for the candidate as a potential treatment of complicated urinary tract infections (cUTI), including acute pyelonephritis.
