Novo Nordisk A/S sent shock waves across the cannabinoid receptor 1 (CB1) space after unveiling Sept. 20 phase IIa data with monlunabant, a small-molecule oral inverse agonist, formerly INV-202, brought aboard via the $1 billion-plus buyout of Inversago Pharmaceuticals Inc. last summer. The trial investigated the efficacy and safety of a once-daily 10-mg, 20-mg, and 50-mg dose of monlunabant vs. placebo on body weight after 16 weeks in 243 people with obesity and metabolic syndrome. Subjects were equally randomized among the four treatment arms. From a baseline of 110.1 kg, all doses of the drug achieved a statistically significant weight loss compared to placebo; those given the 10-mg dose saw their weight drop by 7.1 kg compared to a reduction of 0.7 kg with placebo. Limited additional upside was seen at higher doses, the Bagsvaerd, Denmark-based firm said. Other CB1/weight-loss players’ stocks were hurt more than Novo’s by the report. Shares of Sky Bioscience Inc. (NASDAQ:SKYE) fell almost 59%, and those of Corbus Pharmaceuticals Holdings Inc. (NASDAQ:CRBP) tumbled by 102%.

Biohaven refuses to back down in phase III of rare disease

A little more than a year after the U.S. FDA refused to review the NDA for Biohaven Ltd.’s ultra rare disease treatment, new and positive phase III data has changed the treatment’s momentum. The results showed that troriluzole in treating spinocerebellar ataxia, an ultra-rare neurodegenerative disorder, hit its primary endpoint with statistically significant improvements after both one and two years of treating patients. The original phase III did not reach statistical significance in the overall patient population using troriluzole, a prodrug form of a glutamate modulating agent. The company’s stock (NASDAQ:BHVN) was up 12% at midday, with shares going for $45.37 each.

ECTRIMS 2024: Driving a fast CAR on the MS road and beyond

Chimeric antigen receptor (CAR) T-cell therapy moved the needle at the 2024 ECTRIMS (European Committee for Treatment and Research in Multiple Sclerosis) congress and will continue to do so – not just in multiple sclerosis (MS), but in other autoimmune diseases as well. On Sept. 18^th, Dominic Borie from Kyverna Therapeutics Inc. headed the list of presenters of a sponsored satellite symposium discussing the potential of CAR T-cell therapy in the management of MS and other neurological autoimmune disorders such as Stiff Person Syndrome and myasthenia gravis. Borie depicted the journey so far for KYV-101, Kyverna's autologous anti-CD19 CAR T-cell therapy.

EMA committee recommends Pfizer’s Hympavzi for hemophilia

For once, the EMA appears to have pipped the U.S. FDA to the post, with Pfizer Inc.’s hemophilia A and B therapy Hympavzi (marstacimab) recommended for approval in Europe on Sept. 20, while the U.S. PDUFA date is set for the fourth quarter of the year. Hympavzi targets tissue factor pathway inhibitor (TFPI), a naturally occurring anticoagulation protein that prevents the formation of blood clots. Inhibiting the protein restores the balance between bleeding tendency and blood clot formation. The once-per-week treatment, administered subcutaneously as a standard dose with a pre-filled, auto-injector pen, will be the first TFPI to be approved for the treatment of hemophilia A and B.

PhRMA challenge to US price negotiations lives again

The U.S. Court of Appeals for the Fifth Circuit revived the Pharmaceutical Research and Manufacturers of America’s (PhRMA) constitutional challenge to the Medicare price negotiations, sending it back to a federal court in Texas for a do-over. In its split opinion rejecting the government’s arguments on standing and jurisdiction, the appellate court made it clear Friday that the National Infusion Center Association, which was joined in the suit by PhRMA and the Global Colon Cancer Association, has standing to challenge the negotiations on behalf of its members. In addition, the Fifth Circuit said, the lower court has the jurisdiction to hear the case without it first being channeled through the Department of Health and Human Services.

Hong Kong’s role grows as China moves to up, reform bio sector

Both the biotech industry and Hong Kong have become strategic points for China as the People’s Republic of China works to lay a biotech “belt and road” through Asia to expand global influence. Leung Chun-ying, vice chairperson of the Chinese People’s Political Consultative Conference, recently highlighted the Chinese Communist Party’s central committee’s firm “resolution” to prioritize the sector while underscoring the “need for a scientific revolution.” “The media … may say that China is ‘rolling back’, but that is not true,” Leung said at Bio Hong Kong 2024 Sept. 11.

Respiratory vaccine firm Vicebio raises $100M in series B

Vicebio Ltd. is funded to take two of its molecular clamp respiratory infection vaccines through phase II development, after raising $100 million in a series B. The round will finance the company to advance VXB-241, a bivalent vaccine against respiratory syncytial virus (RSV) and metapneumovirus (MPV); and VXB-251, a trivalent formulation designed to protect against RSV, MPV and parainfluenza virus, to the point where there is sufficient data on both to decide whether to proceed with expensive phase III trials.

Drug manufacturers next on US FTC’s rebate hit list

When the U.S. FTC filed suit Friday against the country’s three largest pharmacy benefit managers over their alleged use of rebates to artificially inflate U.S. insulin prices, it also put the three big insulin makers, and other drug manufacturers, on notice that they could be next. The FTC’s Bureau of Competition “remains deeply troubled by the role drug manufacturers play in driving up prices of life-saving medications like insulin,” said Rahul Rao, the bureau’s deputy director. He added that the bureau “reserves the right to recommend naming drug manufacturers as defendants in any future enforcement actions over similar conduct.”

Mpox and cancer therapies among August’s 22 FDA approvals

August saw the U.S. FDA approve 22 drugs, an increase from July's 17 but fewer than the 28 approved in June. This brings the 2024 monthly average to just over 19 approvals, outpacing last year's average of 16 per month, as well as the 12.5 per month seen in 2022 and 17 per month in both 2021 and 2020.

Also in the news

Acadia, Amplia, Apellis, Ascendis, Axcelead, Biocon, Biosyent, Biotech, Broken String, Cosmo, Fortress, Impact, Innovative, Ipsen, Janssen, Johnson & Johnson, Merck, Neuraptive, Novo Nordisk, Passage, Pfizer, Quralis, Rejuveron, Samsung Bioepis, Sanofi, Sensorion, Tectonic, Zenas