Mirati Therapeutics Inc.’s cancer treatment Krazati (adagrasib) is having a tough time outside the U.S. The EMA’s Committee for Medicinal Products for Human Use (CHMP) came out with a negative opinion on an MAA for treating KRAS G12C-mutated advanced non-small-cell lung cancer (NSCLC). The ruling came with a flurry of positive CHMP opinions for companies, including Abbvie Inc., Beigene Ltd. and the Janssen Pharmaceutical Cos., plus a mixed result for Ipsen SA after a stumble earlier this week.
Despite imperfect data and the use of post hoc analyses to overcome a failed endpoint, the U.S. FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 10-4 June 28 that Ipsen SA’s ultra-rare bone disease drug, palovarotene, demonstrated efficacy in treating patients with fibrodysplasia ossificans progressiva (FOP).
After nearly 10 years in clinical development and just a few months after an EMA rejection, Ipsen SA will be making its case June 28 before a U.S. FDA advisory committee for its ultra-rare bone disease drug, palovarotene.
Where European regulatory decisions were concerned, there was good news and bad news for pharma today as Sanofi SA and Regeneron Pharmaceuticals Inc. got the go-ahead for expanded approval of Dupixent (dupilumab) in pediatric atopic dermatitis patients whilst Ipsen SA’s ultra-rare bone disease drug palovarotene was left off the shelf as efficacy data failed to impress.
Parties to Astrazeneca plc’s potential $1.8 billion takeover of Cincor Pharma Inc. are keeping mum about contingent value rights included in the deal for baxdrostat, the phase III-bound aldosterone synthase targeter in the works to treat hypertension and chronic kidney disease. Another acquisition that captured headlines as the J.P. Morgan Healthcare Conference launched in San Francisco: the $1.4 billion-plus agreement whereby Chiesi Farmaceutici S.p.A. will take ownership of Amryt Pharma plc. And there was more, as Ipsen SA pledged $952 million to make Albireo Pharma Inc. its own.
As Marengo Therapeutics Inc. gears up to start human trials with its lead compound, the company nailed down a deal with Ipsen SA worth $45 million up front and as much as $1.59 billion-plus in milestone rewards to advance a pair of candidates from the Selective T Cell Activation Repertoire (STAR) platform into the clinic.
Ipsen SA’s $247 million buyout of Epizyme Inc., and the recent decision by Nordic Nanovector SA to dump its phase IIb program with CD-37-targeted Betalutin (177Lu lilotomab satetraxetan), served to highlight the hot space of relapsed/refractory follicular lymphoma, where bispecifics have been showing particular promise.
Shares in Ipsen SA edged higher June 29 after the firm announced it had refiled its palovarotene NDA with the U.S. FDA for the ultra-rare disease fibrodysplasia ossificans progressiva. The regulator granted a six-month priority review for the drug, which was once written off and had its FDA filing pulled in 2021 after officials asked for further analyses and data.
After its $247 million acquisition of Epizyme Inc., Ipsen SA will continue to shop for M&A prospects, said Ipsen CEO David Loew, noting that “the [company’s] firepower has not been absorbed in a significant fashion by this deal,” and the firm might undertake a larger transaction next time in oncology, rare diseases, or neurology.
Fresh off licensing a potential Parkinson’s disease therapy last month, Ipsen SA is again looking to build out its neurodegenerative disease portfolio, this time in Huntington’s disease and Angelman syndrome. An exclusive options deal with Exicure Inc. could bring it two new spherical nucleic acid (SNA) programs for the indications while delivering up to $1 billion for Exicure, plus potential royalties, on top of a $20 million up-front payment. The deal, Exicure's second major SNA collaboration after a hair-loss disorders deal with Abbvie Inc.’s Allergan, sent Exicure shares (NASDAQ:XCUR) up 34.1% to $1.81 on Aug. 2.
