Eli Lilly and Sangamo Therapeutics have entered into a licensing agreement worth over $1.4bn to deliver genomic medicines for central nervous system (CNS) diseases.

The deal gives Lilly an exclusive worldwide licence to use Sangamo’s neurotropic adeno-associated virus capsid for one initial target, and the company also has the option to add another four.

CNS diseases are a group of conditions that affect the brain or spinal cord. They include neurodegenerative diseases such as Alzheimer’s disease and multiple sclerosis, as well as infections and tumours.

Sangamo’s STAC-BBB has already demonstrated potent blood-brain barrier penetration and neuronal transduction in nonhuman primates, according to the genomic medicine company.

The agreement will see Sangamo complete a technology transfer related to the STAC-BBB capsid. Lilly will then take on responsibility for all research, preclinical and clinical development, regulatory interactions, manufacturing and global commercialisation of any products resulting from the collaboration.

Sangamo will receive $18m upfront and will be eligible for up to $1.4bn in additional licensed target fees and milestone payments across the five potential neurology disease targets, as well as tiered royalties on future net sales.

Roche’s Genentech unit and Repertoire Immune Medicines have entered into a partnership worth $765m to discover and develop T cell-targeted immune medicines for an undisclosed autoimmune disease.

The collaboration and licence agreement centres around Repertoire’s DECODE platform, which will be used to generate target discoveries that Genentech will further optimise and translate into new drugs.

Autoimmune diseases, including rheumatoid arthritis and type 1 diabetes, affect more than 50 million people in the US. They occur when the immune system is unable to distinguish between healthy cells and invading pathogens.

Repertoire’s platform is designed to map the immune synapse, offering a comprehensive overview of the interactions between T-cell receptors and their cognate antigenic epitopes.

Under the terms of the agreement, Repertoire will lead target discovery activities using its platform, while Genentech will take on responsibility for preclinical and clinical development, as well as global commercialisation of therapies utilising the target discovery work.

In exchange, the Flagship Pioneering-founded biotech will be eligible to receive $35m upfront and up to $730m in development, regulatory and commercial milestones, plus tiered royalties.

Biogen and Eisai’s Alzheimer’s disease (AD) drug Leqembi (lecanemab) has been approved by the European Commission (EC).

The decision makes Leqembi the first therapy authorised in the EU that targets an underlying cause of the neurodegenerative disorder, according to the companies.

The EC has approved the drug to treat adults with a clinical diagnosis of mild cognitive impairment and mild dementia due to AD who have one or no copies of the ApoE4 gene with confirmed amyloid pathology.

Administered intravenously every two weeks, Leqembi works by binding to and reducing clumps of amyloid beta protein that form plaques in the brain.

The approval was supported by results from the late-stage Clarity AD trial, in which treatment with Leqembi in the EU indicated population reduced clinical decline on the global cognitive and functional scale, CDR-SB, by 31% at 18 months compared to placebo.

Leqembi was also associated with a 33% reduction in decline compared to placebo at 18 months on the AD Cooperative Study-Activities of Daily Living Scale for Mild Cognitive Impairment, which measures information provided by people caring for AD patients.

Pfizer’s Hympavzi (marstacimab) has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA) to treat haemophilia A or B in adult and paediatric patients.

The drug has been authorised by the regulator to prevent or reduce bleeding episodes in patients ages 12 years and older who weigh at least 35kg and have severe haemophilia A without factor VIII inhibitors or severe haemophilia B without factor IX inhibitors.

More than 11,800 people were living with haemophilia A or B in the UK in 2023/2024, including 2,700 with severe disease.

Pfizer’s Hympavzi, given as a once-weekly subcutaneous injection using a pre-filled pen, works by reducing the amount of TFPI, a naturally occurring protein that prevents blood from clotting too much. This promotes the formation of thrombin, an enzyme critical in blood clotting.

The MHRA’s decision on the drug was based on results from the phase 3 BASIS trial, in which the annualised bleeding rate for treated bleeds was 7.85 for routine factor-based prophylaxis during a six-month observation period and 5.08 for Hympavzi prophylaxis during a 12-month active treatment period.