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.
Surrozen Inc., a startup developing regenerative medicines leveraging tissue-specific Wnt modulation, has raised $50 million in series C financing. Proceeds from the round will be used to advance the company’s top two antibodies to the clinic, one for the treatment of severe liver disease and a second for moderate to severe inflammatory bowel disease (IBD).
Four years after the EMA first approved Novartis AG's Ilaris (canakinumab) for the treatment of adult-onset Still’s disease (AOSD), the FDA has followed suit, giving it a green light in the indication following a priority review. The rare rheumatic condition usually affects younger adults and can occur as infrequently as once, but also intermittently or chronically.
C4 Therapeutics Inc., a company focused on destroying disease-causing proteins via degradation, has closed a $170 million financing intended to help move four cancer candidates into the clinic by the end of 2022.
Shares of Humanigen Inc. (NASDAQ:HGEN) climbed 30.2% to $5.31 on Monday after the company disclosed that, when treated with the company's anti-GM-CSF antibody, lenzilumab, 12 hospitalized patients with severe or critical pneumonia as a result of COVID-19 showed "rapid clinical improvement."
Just 80 days after first issuing an emergency use authorization (EUA) for oral formulations of chloroquine phosphate (CQ) and hydroxychloroquine sulfate (HCQ) in treating COVID-19, the FDA is revoking that authorization in light of clinical data and scientific literature that raised questions about whether benefits of the treatment outweigh the risks.
Threads of hope and caution stitched together a wide-ranging appraisal of COVID-19 vaccines at June 9's BIO Digital plenary, "A Way Forward." The good news, delivered by one of the world's most sober participants in the pandemic response, mercifully came first.
