A readout of phase III data of buntanetap in Parkinson’s disease is up next as Annovis Bio Inc. tries to recover from the market’s reaction to mixed phase II/III results in Alzheimer’s disease. The company’s stock (NYSE:ANVS) dropped 60% when the data were released on April 29 and continued downward on April 30, with shares closing 23.5% lower at $5.57 each. The shares’ 12-month peak was on Dec. 27 at $19.54 each.

Newron Pharmaceuticals SpA added heft to the data supporting evenamide, reporting positive top-line results from a phase II/III placebo-controlled trial of the glutamate inhibitor in patients with chronic schizophrenia who were receiving a second-generation antipsychotic, but having an inadequate response to that treatment.

In March 2024, BioWorld reported on 261 phase I-III clinical trials updates, showing a 10.6% increase from February's count of 236 and up from January’s 252. However, March’s tally is an 18.2% decline from March 2023’s 319 updates. The average monthly count of phase I-III updates in the first three months of 2024 stood at roughly 277, compared to 305 for all of 2023.

Gilead Sciences Inc. is aiming to capitalize on the early August 2022 buyout of privately held U.K. biotech Mirobio Ltd. with the advancement of PD-1 agonist GS-0151 into phase Ib trials for rheumatoid arthritis, a decision that Leerink analyst David Risinger hailed as positive for others at work with the intriguing mechanism. Paying $405 million for Oxford-based Mirobio, Gilead took ownership of the firm’s checkpoint agonists to treat autoimmune diseases.

Acrivon Therapeutics Inc.’s $130 million financing disclosed April 9 hiked confidence in then-pending data with ACR-368 (prexasertib), the selective small-molecule inhibitor that targets checkpoint kinase 1 (CHK1) and CHK2. Undergoing tests in a potentially registrational phase II trial across multiple tumor types, ACR-368 also raised the stakes for Boundless Bio Inc., which is developing CHK1 inhibitor BBI-355.

About a year after the U.S. FDA cleared Krystal Biotech Inc.’s topical gene therapy, Vyjuvek (beremagene geperpavec), as the first drug for recessive or dominant dystrophic epidermolysis bullosa, competitor Abeona Therapeutics Inc. disclosed a complete response letter (CRL) from the FDA related to its cell-based graft therapy, prademagene zamikeracel.

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.

Genfleet Therapeutics Inc. has entered the KRAS G12C inhibitor race in the U.S. as it gears up to begin phase III trials of GFH-925 (IBI-351) in patients with refractory metastatic colorectal cancer (CRC) following FDA clearance. GFH-925 was the first KRAS G12C inhibitor to receive breakthrough therapy designation from China's National Medical Products Administration for previously treated advanced CRC.