Autolus Therapeutics Plc

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorisation to Autolus Therapeutics’ Aucatzyl (obecabtagene autoleucel) to treat acute lymphoblastic leukaemia (ALL). The CAR T-cell therapy has been specifically authorised for use in adults with relapsed or refractory (R/R) B-cell precursor ALL. Approximately 765 new cases of ALL, an aggressive blood cancer, are diagnosed in the UK every year. Up to 50% of patients will relapse following frontline treatment, and survival rates remain very poor in adults with R/R disease. Administered as an intravenous infusion, Aucatzyl works by taking patients’ T cells and transforming them into CD19-targeting CAR T cells. When transferred back into the body, these modified cells can recognise and destroy the cancer cells. The MHRA’s decision on the therapy was supported by data from the open-label FELIX trial, in which 52 of the 94 R/R B-cell ALL patients who were given at least one infusion of Aucatzyl showed complete remission of the disease with an 81% probability of overall survival at 12 months. Julian Beach, MHRA interim executive director, healthcare quality and access, said: “We are committed to making innovative treatment options, like CAR T-cell therapy, available to patients as quickly as possible, ensuring our approval is underpinned by robust evidence of efficacy alongside the highest standards of safety. “We are assured that the appropriate regulatory standards for the approval of this medicine have been met.” In line with its conditional marketing authorisation pathway, the MHRA will review new efficacy and safety information for Aucatzyl at least once every year. The therapy is now being evaluated by the National Institute for Health and Care Excellence (NICE) for potential use on the NHS. FELIX lead investigator, Claire Roddie, University College London Cancer Institute, said: “Having treated a number of patients with Aucatzyl as part of the FELIX clinical trial, I am delighted that we have moved closer to eligible R/R B-cell ALL patients being able to access Aucatzyl. “We now look forward to NICE completing its assessment of the medicine to potentially make it an option for eligible patients on the NHS.”

WATERTOWN, Mass., April 15, 2025 (GLOBE NEWSWIRE) -- Remix Therapeutics (Remix), a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address the underlying drivers of disease, today announced the appointment of life science executive, Linda C. Bain, to its Board of Directors. Ms. Bain is a Venture Partner at Atlas Venture and currently serves as a Board Advisor to Remix Therapeutics. “Linda brings more than 20 years of financial, operational and executive leadership success at innovative life science companies,” said Matt Patterson, Chairman of Remix Therapeutics Board of Directors. “Her substantial knowledge and experience successfully managing organizations through significant periods of growth will be invaluable to support Remix as it advances its innovative clinical-stage and discovery programs.” “Having partnered with Linda in her role as Board Advisor to Remix, we are excited to have her join our Board of Directors,” said Pete Smith, PhD, Co-Founder and Chief Executive Officer of Remix Therapeutics. “Linda’s impressive track record of leading financial and operational strategy for both private and publicly held biopharmaceutical companies will be beneficial to Remix’s evolution and achieving our goals.” Prior to Atlas Venture, Ms. Bain served as Chief Operating Officer (COO) and Chief Financial Officer (CFO) of Mariana Oncology and oversaw the company’s acquisition by Novartis in 2024 for $1B upfront and up to $750M in potential milestone payments. She has raised nearly $1B in capital for public and private life science companies over the past 10+ years. Prior to Mariana Oncology, Ms. Bain served as CFO of Codiak BioSciences, CFO of Avalanche Biotechnologies, and Vice President of Finance, Business Operations, and Treasurer at bluebird bio. She previously held senior roles at Genzyme, Fidelity Investments, AstraZeneca and Deloitte & Touche. She is currently on the Board of Directors of Arvinas, Autolus Therapeutics and Hemab Therapeutics.“I am thrilled with the opportunity to contribute my skills and experience to a Company at the forefront of tackling disease drivers with a novel approach, modulating RNA processing with small molecules,” said Ms. Bain. “I look forward to joining the Remix Board of Directors and working with the team to best position the Company to deliver on this important mission and advancing new options for patients in great need of new treatments.” About Remix TherapeuticsRemix Therapeutics is a clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and address disease drivers at their origin. Remix's REMaster™ technology platform leverages cutting-edge data science, biomolecular sciences and chemistry approaches to identify orally administered compounds that modulate gene expression. Remix's innovative therapeutic approach led to the discovery of REM-422, a first-in-class RNA processing modulator in oncology, now being evaluated in Phase 1 clinical studies to treat acute myeloid leukemia (AML), high-risk myelodysplastic syndrome (HR-MDS) and adenoid cystic carcinoma (ACC). For more information visit www.remixtx.com. Contacts: Media Contact:Lisa BuffingtonBuffington Commslbuffington@remixtx.com Investor Contact:Will O'Connor Precision AQWill.OConnor@precisionaq.com

+Plus, news about Autolus, MacroGenics and BlissBio: Akeso bispecific looks promising in cervical cancer: The China-based biotech said Friday that all patients in a Phase 3 cervical cancer trial responded to treatment with a bispecific antibody called cadonilimab. The addition of the PD-1xCTLA-4 bispecific on top of chemoradiotherapy in the COMPASSION-18 study generated complete and partial response rates of 84.8% and 15.2%, respectively. Median PFS was not reached, but the 12-month PFS rate was 74.9%. Cadonilimab is already approved in China for patients with recurrent or metastatic cervical cancer who have not responded to platinum-based chemotherapy. — Elizabeth Cairns Pulmatrix to divest assets ahead of merger: The biotech will seek buyers for three of its pipeline programs as well as its iSPERSE platform technology. Pulmatrix in November agreed to a reverse merger with Cullgen, which was expected to close this month but is now set to close in the first half of 2025. — Max Gelman BioNTech’s option for Autolus CAR-T fizzles out: The German biotech allowed its option to lapse for AUTO1/22, a CAR-T cell therapy in Phase 1 trials for childhood acute lymphoblastic leukemia. Back in February 2024 , BioNTech paid $50 million in cash and bought $200 million of Autolus’ shares in a deal that granted it access to AUTO1/22. The deal also covered AUTO6NG, a programmed T-cell therapy in early trials in neuroblastoma, and Aucatzyl, which has since been approved for an aggressive form of leukemia. BioNTech declined to exercise its time-limited option to AUTO1/22 because of a pipeline prioritization , and it lapsed Feb. 8. — Elizabeth Cairns MacroGenics shelves prostate cancer ADC: The company had to stop dosing prostate cancer patients in a Phase 2 trial of its antibody-drug conjugate, called vobramitamab duocarmazine, in July . On Thursday , it said it would shut down all development and seek a partner for the product, which targets the tumor antigen B7-H3 and delivers a cytotoxic duocarmycin payload. MacroGenics said the results of the trial do not justify any more investment; in the patients treated before dosing was halted, mature median radiographic progression-free survival was 9.5 months with the lower dose and 10 months with the higher. — Elizabeth Cairns BlissBio to proceed with ADC on its own: Eisai had signed an option deal in 2023 to advance a BlissBio program called BB-1701, but last month said in its third-quarter earnings that it would no longer do so. Instead, BlissBio will develop BB-1701 on its own. Eisai had been partnered with liquid biopsy company Angle on development plans, and now Angle is planning to work with BlissBio. — Max Gelman