DUBLIN – The EMA has rejected Biogen Inc.’s application for European Union approval of Aduhelm (aducanumab), its controversial Alzheimer’s disease drug. Its human medicines committee (CHMP) issued a negative opinion on Biogen’s dossier during its December meeting this week, stating that the data from the key studies submitted in support of the application “were conflicting and did not show overall that Aduhelm was effective at treating adults with early stage Alzheimer’s disease.” What’s more, CHMP noted, the amyloid beta-targeting antibody “could potentially cause harm,” as evidenced by abnormalities in brain scan images from some patients that may be evidence of bleeding or swelling. It also questioned whether these abnormalities could be “properly monitored and managed in clinical practice.”

Genfit signs potential $541M pact with Ipsen for elafibranor in PBC

Genfit SA sealed a global development and commercialization deal with Ipsen Pharma SA for elafibranor in primary biliary cholangitis (PBC). Banking €120 million (US$135.2 million) up front, Genfit could collect as much as €360 million in milestone payments, plus tiered double-digit royalties of up to 20%. Elafibranor, a first-in-class PPAR alpha and delta agonist, is expected to yield top-line phase III data from the PBC study called Elative in early 2023. Genfit also disclosed the acquisition of rights to an earlier-stage asset, GNS-561, from Genoscience Pharma SA. The autophagy/PPT1 inhibitor has completed a phase Ib study in cholangiocarcinoma. Terms include clinical and regulatory milestone payments plus tiered royalties for Genoscience, with Genfit taking a €3 million equity stake in the firm. Shares of Genfit (NASDAQ:GNFT) were trading midday at $4.86, up $1.54, or 46%.

Awaiting NASH data, Northsea pads coffers with $80M series C

LONDON – Metabolic diseases specialist Northsea Therapeutics BV closed a $80 million series C to fund phase II development in two further indications, as it awaits the final results from the phase IIb trial of its lead product, icosabutate, in the treatment of nonalcoholic steatohepatitis (NASH), which are due at the start of 2023. As the phase IIb results land, Northsea will launch phase II development of the second program, SEFA-1024 in the treatment of dyslipidemia, and a phase II study for the orphan indication of intestinal failure associated liver disease. All three products are structurally engineered fatty acids that are designed to overcome the inherent limitations of naturally occurring counterparts.

Bellerophon posts strong phase II data in pulmonary hypertension

Top-line data from Bellerophon Therapeutics Inc.’s phase II study of its inhaled nitric oxide technology, Inopulse, showed decreases in mean pulmonary arterial pressure and pulmonary vascular resistance in treating pulmonary hypertension associated with sarcoidosis. The dose-escalation, proof-of-concept study was conducted to see if Inopulse could show a hemodynamic benefit in the indication. The Warren, N.J.-based company’s stock (NASDAQ:BLPH) leapt 27% upward on the news at midday. Inopulse is also in a phase III study for treating fibrotic interstitial lung disease. Enrollment began in November for treating patients at low, intermediate, and high risk of associated pulmonary hypertension.

Qilu in-licenses HBV-focused RNAi therapy from Arbutus in $300M deal

Qilu Pharmaceutical Co. Ltd. licensed rights to AB-729, an RNAi therapeutic from Arbutus Biopharma Corp., in a deal worth up to $300 million. Qilu obtained rights to develop and commercialize the phase II asset for the treatment of hepatitis B (HBV) in mainland China, Hong Kong, Macau and Taiwan. Under terms of the agreement, Qilu will pay Arbutus $40 million up front, as well as up to $245 million in development, regulatory and sales milestones. In addition, Qilu will make a $15 million equity investment in Arbutus at a price of $4.19 per share.

Best year on record for IPOs, VC rounds; biopharma raises $116B in 2021

With a few weeks left in the year, the biopharma industry has raised a total of $116.18 billion through 1,506 transactions, both of which are second only to 2020.

Also in the news

20n Bio, Abeona, Addex, Advaxis, Alzamend Neuro, Amylyx, Antengene, Astellas, Basilea, Bellerophon, Bellus Health, Biogen, Biolinerx, Biomarin, Cassiopea, Cosmo, Erasca, GBT, Glycomine, GSK, Hyloris, Iktos, Karyopharm, Kineta, Lipidor, Lyell Immunopharma, Medicago, Medicenna, Merck, Mindmed, Miromatrix, Mynd Life Sciences, Navidea, Obseva, Paratek, Passage, Pfizer, Providence, Purdue, RDIF, Ridgeback, Seagen, Senhwa, Skyline, Specific Biologics, Taysha Gene Therapies, UCB, Vaneltix, Vaxthera, Vera, Vincerx, Xalud