Roche Holding AG’s Genentech Inc. unit received U.S. FDA approval on June 20 of Piasky (crovalimab) to treat adults and children 13 and older with paroxysmal nocturnal hemoglobinuria and a body weight of at least 40 kg (88 pounds).
Following a day of deals that collectively bring a potential $3.75 billion into three biopharma companies – namely Ascidian Therapeutics Inc., Belharra Therapeutics Inc. and Mabcare Therapeutics – researchers will be busy discovering new therapies for neurological and immunological diseases, and advancing globally a candidate for solid tumors.
Becoming the first pneumococcal conjugate vaccine specifically designed for adults 18 and older, Merck & Co. Inc.’s 21-valent candidate, Capvaxive (V-116), gained U.S. FDA approval on its June 17 PDUFA date. The Rahway, N.J.-based company expects to take significant market share based on positive phase III findings from the Stride-3 trial. Analysts have estimated the product could reach $2 billion in annual global sales.
Following a phase II/III failure, Stamford, Conn.-based Cara Therapeutics Inc. discontinued its only pipeline program, that of oral difelikefalin for moderate to severe pruritis in adults with notalgia paresthetica, placing the company’s future in question.
Focused on oral therapies for obesity, diabetes and rare diseases, Boston-based Syntis Bio Inc., which raised $15.5 million through seed funding last year, emerged from stealth to advance its synthetic tissue-lining technology and a pipeline of candidates.
Focused on oral therapies for obesity, diabetes and rare diseases, Boston-based Syntis Bio Inc., which raised $15.5 million through seed funding last year, emerged from stealth to advance its synthetic tissue-lining technology and a pipeline of candidates.
Two years after signing a $1.6 billion partnership with Marengo Therapeutics Inc., Paris-based Ipsen SA is back at it again, this time teaming up for two additional assets that target “cold” tumors in a deal that could bring privately held Marengo $1.2 billion. “We do something really novel and innovative and I think it’s very important to continue to validate it,” Marengo CEO Zhen Su told BioWorld, adding that with the newest deal, “we see validation all around this … It’s a success on success.”
Washington-based Vanda Pharmaceuticals Inc. has faced a challenging few years involving a federal lawsuit against the U.S. FDA, patent infringement litigation, increasing generic competition and dwindling sales, as well as a complete response letter nixing plans to expand its melatonin receptor agonist Hetlioz (tasimelteon) into insomnia, yet it has recently received a higher, unsolicited acquisition offer of $466 million from a second company, Cycle Pharmaceuticals Ltd.
Annexon Inc.’s stock (NASDAQ:ANNX) climbed 31% June 4 following news that a single infusion of C1q protein complex inhibitor ANX-005 at the 30-mg/kg dose delivered a statistically significant 2.4-fold improvement in Guillain-Barré syndrome patients. Shares closed at $5.99, up $1.41.
Moderna Inc. gained U.S. FDA approval on May 31, nearly three weeks past its original PDUFA date, for its respiratory syncytial virus (RSV) vaccine Mresvia (mRNA-1345), which had both breakthrough therapy and fast track designations in the U.S.
