Autolus Therapeutics plc has been granted FDA approval for Aucatzyl (obecabtagene autoleucel) for the treatment of acute lymphoblastic leukemia (ALL) in adults, becoming the first marketed CAR T therapy that does not have a risk evaluation and mitigation strategy attached to its label. The approval was based on results of the Felix clinical trial in relapsing/remitting ALL, which showed a strong safety profile compared to existing CD19 CAR T-cell therapies. The conduct of the trial was dogged by the COVID-19 pandemic, but of the 65 patients from an initial dosed cohort of 95 for whom efficacy could be evaluated, 63% achieved overall complete remission.

Abbvie’s emraclidine falls flat in two phase II schizophrenia trials

Abbvie Inc.’s much-hyped emraclidine, the centerpiece of its $8.7 billion buyout of Cerevel Therapeutics Inc., failed to hit its endpoints in two phase II trials in schizophrenia, sending company shares (NYSE:ABBV) down more than 11% in morning trading. The company said it plans to review data to determine the next steps, but it’s unlikely the positive allosteric modulator of the muscarinic M4 receptor will live up to its potential as a next-generation antipsychotic. Benefiting from the news, however, was Bristol Myers Squibb Co., which gained U.S. FDA approval in September 2024 for Cobenfy (xanomeline-trospium), a dual M1/M4 muscarinic agonist, with shares (NYSE:BMY) jumping 11% this morning.

Enterobiotix’s oral microbiota shows promise in liver cirrhosis

Microbiome specialist Enterobiotix Ltd. has reported positive results for the phase Ib trial of its EBX-102 fecal microbiota therapy in patients with stable liver cirrhosis. The placebo-controlled study involved first-in-human dosing with the company’s encapsulated oral formulation of microbiota derived from the stools of healthy donors. After initial dosing, patients were followed up for 12 weeks to assess changes in a range of clinical and blood biomarkers. At the higher dose of EBX-102 there were significant shifts in stool bacterial microbiome composition and bacterial metabolites compared to placebo, as assessed by RNA sequencing.

Rapt scales back after zelnecirnon clinical hold

Rapt Therapeutics Inc. has decided to shut down its zelnecirnon (RPT-193) program in asthma and atopic dermatitis (AD), causing the company’s stock (NASDAQ:RAPT) to sharply decline at midday. Zelnecirnon, an oral small-molecule CC chemokine receptor 4 antagonist, was in two placebo-controlled phase II studies when, in February, the U.S. FDA placed the clinical trials on a clinical hold after a serious adverse event of liver injury was found in one patient in the AD study. Rapt said that, based on recent feedback from the FDA, it would halt the zelnecirnon development program. The shares were trading 44% lower at midday, at about $1.61 each.

Next Generation Gene pioneers gene therapy approach with China data

Gene therapy faces complexities in delivering treatments due to persistent safety concerns and daunting immune responses, but Next Generation Gene Therapeutics Inc. (NGGT) has found a way around this issue using dual-functional vectors to simultaneously remove harmful, mutated genes and replace them with normal, healthy genes to restore cellular function. NGGT co-founder and CEO Lixin Jiang told BioWorld that the company employs a unique strategy of initiating investigator-initiated trials (IITs) in China to reduce development risks and costs. One of the biggest issues he sees in drug development is reliance on animal data. In China, companies have another option for cell and gene therapies to test a drug in IITs before submitting an IND for formal phase I trials. Regulators have been quite flexible in allowing companies to run trials with fewer patients, he said.

Alteogen draws Daiichi in $300M pact for subcutaneous Enhertu

Alteogen Inc. scored another deal for its human recombinant enzyme, ALT-B4, on Nov. 8, reeling in Daiichi Sankyo Co. Ltd. in a potential $300 million deal including $20 million up front and up to $280 million in milestone payments, along with royalties based on net sales. Under the deal terms, Toyko-based Daiichi gained exclusive global rights to use ALT-B4 to develop and commercialize a subcutaneous version of Enhertu (trastuzumab deruxtecan), an HER2-directed antibody-drug conjugate (ADC) jointly developed with Astrazeneca plc and U.S. FDA approved for HER2-positive breast cancer patients in 2019. Like many ADCs, Enhertu is administered intravenously, and comes in a 100-mg lyophilized powder in a single-dose vial.

Duchembio files for IPO to grow radiopharma business

Radiopharmaceutical biotech and contract development and organization firm Duchembio Co. Ltd. filed a securities report to South Korea’s Financial Services Commission Nov. 11, kickstarting the IPO process to list on the Korea Exchange. Duchembio said it would offer about 1.43 million shares on the tech-heavy Kosdaq board at a price band of ₩12,300 (US$8.82) to ₩14,100 per share, raising up to ₩20.16 billion (US$14.3 million) with the IPO.

Also in the news

Akari, Allogene, Altimmune, Amgen, Apimeds, Ascletis, Aulos, Aummune, Climb, Disc Medicine, Dizal, Epibone, Harbour, Indaptus, Johnson & Johnson, Kineta, Longeveron, Moderna, Nektar, Neurotech, Nimbus, Ocugen, Paratek, Peak, Radella, Rubedo, Systimmune, Travere