FDA U-turn gives uniQure a renewed path to accelerated approval in Huntington's disease
uniQure has confirmed it will submit a marketing application for AMT-130, its gene therapy for Huntington's disease, after the FDA agreed during a recent meeting that three years of data from the therapy's key trial would be sufficient to support approval. The company expects to file between July and September.
The decision follows a reversal from the agency's position earlier this year, when regulators indicated that available data would not support an accelerated approval pathway. UniQure firsts findings demonstrated that among patients given the higher dose, signs of disease progression had slowed by 75% after three years. However, FDA staff subsequently indicated the results weren't adequate to support approval, a shift the company described as a "key shift from prior communications," and later "strongly recommended" uniQure conduct an additional trial using a sham-surgery control group.
Beyond Huntington's disease, the FDA's willingness to reconsider its position highlights the growing role that biomarkers may play in future gene therapy approvals. As developers increasingly target rare and slowly progressing diseases where traditional clinical endpoints can take years to mature, regulatory flexibility around surrogate markers could help accelerate patient access while reducing development timelines and costs.
Read more via Biopharma Dive and FirstWord Pharma
Be Biopharma discontinues lead haemophilia B cell therapy programme
Be Biopharma has terminated development of BE-101, its lead engineered B-cell therapy for haemophilia B. The programme was being evaluated as a potentially transformative approach designed to provide sustained production of Factor IX through engineered B cells. However, early clinical data failed to demonstrate the level of therapeutic activity required to justify continued development.
The decision will result in the closure of the programme's ongoing clinical trial and marks a significant setback for the company. The setback is also indicative of the increasingly disciplined capital environment facing biotech companies. Investors and management teams alike are placing greater emphasis on clear clinical signals and capital efficiency, forcing companies to make difficult portfolio decisions earlier in development.
Read more via BioSpace
Anocca doses first patients with precision TCR-T therapy for pancreatic cancer
Swedish cell therapy company Anocca has dosed the first patients in a clinical trial evaluating its precision TCR-T cell therapy for pancreatic cancer. The therapy targets mutant KRAS, one of the most common oncogenic drivers in solid tumours and a mutation found in the vast majority of pancreatic cancers. The programme uses Anocca's precision-engineered T-cell receptor platform to identify and attack tumour cells carrying specific KRAS mutations.
Pancreatic cancer remains one of the deadliest forms of cancer, with limited treatment options and poor long-term survival rates. The initiation of patient dosing therefore represents an important milestone for both the company and the broader TCR-T field.
Anocca's programme reflects growing industry momentum around TCR-based approaches, which offer the ability to target intracellular cancer antigens that are inaccessible to conventional CAR-T therapies. As more TCR-T programmes enter the clinic, the field is moving closer to answering one of the most important questions in cell therapy, can engineered T cells achieve meaningful success in solid tumours at scale.
Read more via PharmaTimes
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