After several delays, the interleukin-15 superagonist Anktiva (nogapendekin alfa inbakicept) from Immunitybio Inc has been approved by the U.S. FDA for treating bladder cancer, specifically Bacillus Calmette-Guérin-unresponsive non-muscle-invasive bladder cancer with carcinoma in situ, with or without papillary tumors. Anktiva will be on the market by mid-May, the company said. The approval was announced on April 22, the day before its PDUFA date. In May 2023, the FDA issued a complete response letter regarding the drug’s BLA, which had been accepted in July 2022, halting development for months. The company’s stock (NASDAQ:IBRX) surged 11% at midday to $5.49 per share.

CAR T pipelines bloom to treat world’s largest cancer population

As of Jan. 31, 2024, there were more than 300 CAR T trials registered in China, surpassing the U.S. and becoming the country with the most CAR T therapy clinical trials. Among them, CD19 is the most frequently studied target, according to BioWorld and Cortellis. The rapid evolution of CAR T-cell therapies in China has escalated over the past decade from the start of the first clinical trials in 2013 to the country becoming an established host for CAR T-cell-related trials by 2017, according to Yongxian Hu and researchers from Zhejiang University in Hangzhou, China. Chinese cell therapy companies – backed by $2.37 billion in funding in 2021 – have since significantly increased basic research and trial output for CAR Ts, which was welcomed by large patient demand.

Abeona plunges on CRL for pz-cel in RDEB

Shares of Abeona Therapeutics Inc. (NASDAQ:ABEO) were trading at $3.93, down $3.43, or 46% on a regulatory update for prademagene zamikeracel (pz-cel). The Cleveland-based firm said the U.S. FDA issued a complete response letter (CRL) related to pz-cel’s BLA for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The CRL follows the completion of Abeona’s late-cycle review meeting with the agency in March 2024. Requested is more information related to chemistry, manufacturing and controls (CMC), but no new trials for the cell-based graft therapy. RDEB is a rare connective tissue disorder caused by a defect in the COL7A1 gene that results in the inability to produce type VII collagen. Abeona expects to submit the CMC information by the end of the third quarter of this year.

Biopharma deal and M&A values continue fluctuations in Q1

In the initial quarter of 2024, biopharma deal value totaled $43.99 billion, surpassing the values of two quarters last year but falling short of two others. Biopharma M&As, meanwhile, reached $50.06 billion, marking a decrease from the fourth quarter last year’s $84.03 billion but exceeding the values of the other three quarters.

Vertex taps Treefrog’s stem cell tech for up to $780M

Vertex Pharmaceuticals Inc. has tapped into Treefrog Therapeutics SA’s high-throughput stem cell manufacturing technology in a deal worth potentially up to $780 million to help it advance its type 1 diabetes programs, including phase I/II asset VX-880. Bordeaux, France-based Treefrog’s technology, C-stem, enables exponential growth of induced pluripotent stem cells (iPSCs) cells in 3D by mimicking the natural microenvironment, and is capable of growing up to 15 billion cells in a single batch, in one week. The company said the method reduces manufacturing expenses for iPSC-derived cell therapies, while improving safety and efficiency.

Sanofi’s BTK inhibitor hits phase III endpoint in thrombocytopenia

An oral Bruton’s tyrosine kinase inhibitor that Sanofi SA acquired in 2020 through its $3.68 billion buyout of Principia Biopharma Inc. is headed toward regulatory filings in the U.S and EU by the end of the year, following phase III data in immune thrombocytopenia (ITP). Dosed at 400 mg twice daily, rilzabrutinib achieved its primary endpoint of durable platelet response, and showed a consistent safety profile, in adults with persistent or chronic ITP, a condition for which the drug has both fast track and orphan drug designations in the U.S. The pivotal trial, Luna 3, enrolled patients who were refractory to prior therapy.

Samsung Bioepis, Celltrion make biosimilar inroads in Europe, US

Samsung Bioepis Co. Ltd. and Celltrion Inc. are making headway in Europe and U.S. with respective follow-on biologic products, with Samsung Bioepis the latest to gain EMA approval for Pyzchiva, a Stelara (ustekinumab, Janssen Pharmaceuticals Inc.) biosimilar, on April 23. With Pyzchiva’s approval, Samsung Bioepis has seven biosimilar products launched in Europe, including SB-4 (etanercept, Enbrel), SB-2 (infliximab, Remicade), SB-5 (adalimumab, Humira), SB-3 (trastuzumab, Herceptin), SB-8 (bevacizumab, Avastin), SB-11 (ranibizumab, Lucentis) and SB-12 (eculizumab, Soliris). Incheon-based Celltrion is also gaining biosimilar ground. In Europe, Celltrion’s follow-on biologics of Remicade (infliximab), known as Remsima and Remsima SC, claimed a 74% market share in the EU5 countries of Germany, France, Spain, Italy and the U.K. in the third quarter of 2023. The EU5 are known to account for 60% of Europe’s autoimmune disease market.

Brain Trust Bio’s drug delivery method to be tested in Australia

Brain Trust Bio Inc. (BTB) will soon begin phase I trials in Australia of its IT-Riluzole delivered to the brain via a continuous intrathecal drug delivery method in people with amyotrophic lateral sclerosis. The company’s concept is to take known drugs that work and make them better by delivering them exclusively to where patients need them most, BTB co-founder and CEO Chen Benkler told BioWorld.

BioWorld Insider Podcast: Mark McKenna storms back with Mirador and eyes the development landscape

In one of the biggest financings of the year so far, former Prometheus Biosciences Inc. CEO Mark McKenna helped raise $400 million to launch a new company, Mirador Therapeutics Inc. He didn’t sit on the sidelines for long after Merck & Co. Inc. bought Prometheus for $10.8 billion in 2023. He recruited key Prometheus executives to focus on Mirador’s genetic approach to drug discovery and precision medicine. McKenna said there was too much left undone to just hang back. In this BioWorld Insider episode, he talks about the new company and the multi-billion-dollar drugs that he believes provide sub-optimal efficacy compared to the tailor-made therapies he wants to develop. He also has deep insights into drug pricing and why the investment market is so tough on companies that don’t have A-plus science and teams.

Also in the news

Acadia, Actinogen, Anthos, Aslan, Benevolentai, Biohaven, Bioxcel, Brise, Calluna, Cellectis, Centessa, CN, Daré, Enlivex, Evexta, Genetic Technologies, Hepion, Ideaya, Immedica, Incyte, Janssen, Krystal, Kynos, Lantern, LSL, Maia, Medigene, Nexus, Oncimmune, Palisade, Paxmedica, Pfizer, Pharmala, Profluent, Proqr, Red Arrow, Red Light Holland, Sandoz, Seamless, Sillajen, Sygnature Discovery, Tarsus, Tharimmune, Tiziana, Vanda, Wave, Xbrane