AbbVie and Xilio Therapeutics have announced a new partnership aimed at developing tumour-activated, antibody-based immunotherapies, with the deal worth more than $2.1bn.

The collaboration and option-to-licence agreement will include masked T-cell engagers and will combine AbbVie’s capabilities in oncology with Xilio’s proprietary tumour-activation technology.

Xilio outlined that it is currently using its platform to advance a pipeline of clinical and preclinical immunotherapies. This includes masked multi-specific molecules that are “designed to achieve tumour-selective activation by leveraging masking and other unique components that are optimised for the specific target,” it said.

The biotech will receive $52m in total upfront payments from AbbVie, including a $10m equity investment, and will be eligible to receive up to approximately $2.1bn in total payments for option-related fees and milestones, as well as tiered royalties.

Theodora Ross, vice president, early oncology research and development at AbbVie, said the company is “committed to expanding [its] research and development efforts in oncology.

“This includes investigation of novel immunotherapy approaches that aim to generate improved next-generation cancer treatments for patients in need,” she said.

Biogen and Stoke Therapeutics have announced a partnership worth $550m to advance a disease-modifying treatment for Dravet syndrome.

Up to 38,000 people in the US, UK, EU and Japan are estimated to be living with Dravet syndrome, a genetic developmental and epileptic encephalopathy characterised by recurrent seizures and significant cognitive and behavioural impairments.

Stoke’s zorevunersen is an investigational antisense oligonucleotide that targets the SCN1A gene, the underlying cause of most cases of Dravet syndrome, to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and improve neurodevelopment, cognition and behaviour.

Stoke, which recently announced plans to initiate a global phase 3 registrational study of the drug, will continue to lead zorevunersen’s global development and will retain exclusive development and commercialisation rights in the US, Canada and Mexico, while Biogen will gain exclusive commercialisation rights in the rest of the world.

Under the terms of the agreement, Biogen will contribute to 30% of the external clinical development costs and Stoke will receive an upfront payment of $165m, up to $385m in development and commercial milestone payments, and tiered royalties in Biogen’s territory.

Sobi’s Sanofi-partnered efanesoctocog alfa has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat haemophilia A.

The drug has been authorised under the brand name Altuvoct to treat and prevent bleeding in patients aged two years and older with severe or moderate cases of the genetic bleeding disorder.

Haemophilia is a rare condition resulting from insufficient levels of functioning factor VIII, a protein that is essential for blood clotting.

Among the clinical evidence supporting the MHRA’s decision on the drug were results from the late-stage XTEND-1 study of 159 patients aged 12 years and older with severe haemophilia A.

Weekly injections of Altuvoct led to 65% of patients reporting zero overall bleeding episodes over the course of the year-long study, while the remaining 35% saw significant reductions in bleeding episodes.

Sanofi and Sobi collaborate on efanesoctocog alfa in the US, where it is marketed as Altuviiio, while Sobi has final development and commercialisation rights in Europe, North Africa, Russia and most Middle Eastern markets, with Sanofi holding these rights in North America and all other regions worldwide.

Roche has announced that a tablet formulation of its spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) has been approved by the US Food and Drug Administration (FDA).

Evrysdi was originally authorised by the regulator in 2020 to treat the progressive neuromuscular disease in patients aged two months and older, before being granted expanded approval in 2022 for infants aged under two months.

Until now, the drug has only been available as an oral solution, administered daily at home by mouth or feeding tube. It is hoped that the tablets, which can be stored at room temperature and either swallowed whole or dispersed in water, will provide more freedom and independence for patients.

SMA affects approximately one in every 10,000 babies worldwide and can result in patients’ physical strength and ability to walk, eat or breathe being significantly diminished or lost.

The FDA’s latest decision on Evrysdi, which makes it the first and only tablet approved for SMA, was based on results from a bioequivalence study demonstrating that the 5mg tablet and original oral solution provided comparable exposure to the drug.