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.
Agios Pharmaceuticals Inc.’s fourth-quarter and full-year 2023 financial report in mid-February highlighted further prospects with Pyrukynd (mitapivat), the pyruvate kinase activator for hemolytic anemia in adults with pyruvate kinase deficiency (PKD).
Cerevel Therapeutics Inc.’s positive results from the long-shot pivotal phase III Tempo-3 trial with tavapadon – the first D1/D5 receptor partial agonist being studied as a once-daily treatment for Parkinson’s disease – added gravy to the $8.7 billion takeover by Abbvie Inc., disclosed late last year. The buyout’s centerpiece was the late-stage asset emraclidine, a positive allosteric modulator of the muscarinic M4 receptor, touted as a potential best-in-class, next-generation antipsychotic for schizophrenia, which strikes more than 5 million people in the G7 (U.S., France, Germany, Italy, Spain, U.K. and Japan).
After Sage Therapeutics Inc. reported a phase II failure with oral dalzanemdor, also known as SAGE-718, in mild cognitive impairment related to Parkinson’s disease (PD), Wall Street’s eyes turned to ongoing mid-stage efforts with the same N-methyl-D-aspartate receptor-positive allosteric modulator in Huntington’s disease and Alzheimer’s disease.
With an IND filing in systemic lupus erythematosus expected during the third quarter of 2024, Cullinan Therapeutics Inc. changed its name from Cullinan Oncology Inc. and detailed its plans to target autoimmune diseases with CLN-978, a CD19xCD3 T-cell engager.
Wall Street may not have responded as positively as Ultragenyx Pharmaceutical Inc. would have liked after the firm unveiled new data from the phase I/II study with GTX-102 for the treatment of Angelman syndrome (AS). Patients in expansion cohorts A & B treated with a set dose and regimen of the intrathecally delivered antisense oligonucleotide (ASO) showed rapid and clinically meaningful improvement across multiple domains.
As Novartis AG’s approved prostate cancer therapy, Pluvicto (177Lu-PSMA-617), continues on a growth trajectory, the firm signed a licensing deal with Arvinas Inc. potentially valued at north of $1 billion for global development and commercialization of ARV-766, the latter’s second-generation proteolysis targeting chimera (PROTAC) androgen receptor degrader targeting the same disease.
Stable disease in about half the patients tested wasn’t enough for Wall Street, and shares of Vincerx Pharma Inc. (NASDAQ:VINC) nosedived by $3.72, or 78%, to close April 9 at $1.06 on the disclosure of preliminary phase I data with small-molecule drug conjugate VIP-236 in metastatic solid tumors.
Mixed opinions from the U.S. FDA’s Oncology Drugs Advisory Committee last month didn’t stop the agency from green-lighting an expanded label for Abecma (idecabtagene vicleucel) to include adults with relapsed or refractory multiple myeloma (r/r MM) after two or more prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody.
