Mediar Therapeutics Inc. emerged from stealth, unveiling $105 million in investment and bold ambitions to develop new ways of tackling fibrosis. Mediar is challenging what CEO Rahul Ballal called “the fundamental dogma” of fibrosis therapy: “You have to address fibrosis at its initiation, when inflammation is rampant.” Mediar, in contrast, is focused on developing therapies that either disrupt or reverse the fibrotic process, particularly when it progresses from a moderate to a severe state. Crucially, its approach is biomarker-based. “All of our targets can be measured in the blood and, more importantly, correlate with disease progression and, in some cases, with outcomes,” Ballal told BioWorld.

Bellicum stock rocked by CRS in GoCAR-T phase I/II; quits, mulling options

Shares of Bellicum Pharmaceuticals Inc. (NASDAQ:BLCM) were trading midday at 45 cents, down 41 cents, or 47%, as Wall Street reacted to news that the phase I/II study with GoCAR-T will be discontinued. The study was designed to test GoCAR-T cell candidates BPX-602 and BPX-603 when combined with the activating agent rimiducid in heavily pretreated cancer patients. Bellicum chose to abandon the experiment after the patient most recently treated with BPX-601 for metastatic castration-resistant prostate cancer turned up serious immune-mediated adverse events, including grade 4 cytokine release syndrome: the second dose-limiting toxicity to arise in this cohort of dose escalation. A review of strategic alternatives is underway, Bellicum said.

Further manufacturing delays cast doubt on Lumevoq despite sustained phase III efficacy

Patients with Leber hereditary optic neuropathy who received bilateral injections of Gensight Biologics SA’s Lumevoq (lenadogene nolparvovec) are continuing to see statistically significant visual improvements three years into the phase III REFLECT trial, but the missed primary endpoint at 1.5 years, along with a series of manufacturing mishaps, have left the gene therapy’s approval prospects uncertain.

NMPA accepts enfortumab vedotin application from Astellas

The NMPA accepted a BLA from Astellas Pharma Inc. for enfortumab vedotin, which is designed for the treatment of patients with locally advanced or metastatic urothelial cancer who previously received treatment with a PD-1/L1 inhibitor and platinum-based chemotherapy.

US RX pricing controls begin to kick in

Despite the new Medicare inflation rebate, the U.S. price of 27 Part B drugs subject to the rebate provision in the Inflation Reduction Act saw price hikes exceeding the rate of inflation. The manufacturers of those drugs will be billed for the rebates in 2025, but Medicare beneficiaries should see a drop in their coinsurance for those drugs, for the next quarter at least. How much relief they realize will depend on the type of supplemental coverage they have and the dose and treatment protocol they’re on, Medicare Director Meena Seshamani said at a White House media briefing. Meanwhile, Novo Nordisk Inc. reported that it’s lowering the U.S. list prices of several insulin products by up to 75% beginning Jan. 1, 2024. Following a similar move by Eli Lilly and Co., Novo Nordisk’s announcement puts more pressure on Sanofi SA, the other major insulin provider in the U.S., to follow suit.

What’s next for Paxlovid

Pfizer Inc.’s Paxlovid has become such a part of the COVID-19 treatment scene in the U.S. that it’s easy to forget it’s only authorized for emergency use. That could soon change. The FDA is convening its Antimicrobial Drugs Advisory Committee March 16 to consider Pfizer’s new drug application for the co-packaged nirmatrelvir and ritonavir treatment.

Also in the news

AB Science, Acesion, Akeso, Annji, Aridis, Astellas, Atea, Avenue, Biohaven, Cantargia, Defence, Ellipses, Endo, Eureka, Exscientia, Gibson, Gradalis, Hutchmed, Immutep, Innoviva, Macrogenics, Medivir, Mirum, Myosin, Novan, Novo Nordisk, Onkosxcel, Orano, Protai, Proxygen, Puma, Reneo, Sapience, Servier, Synaffix, Takeda, Telix, Truebinding, Tscan, Xortx