Lisbon, April 23, 2025 (Lusa) - Portuguese pharmaceutical company BIAL has agreed to be a founding member of the biotech company ‘Orphan Therapeutics Accelerator’ (OTXL), focused on obtaining and restarting the development of promising ‘shelved’ clinical-stage drugs for ultra-rare diseases and providing a path to commercialisation.

The agreement with the biotech will streamline research into rare diseases and, as a founding member, Bial will play a key role in the selection, final stages of development and commercialisation of therapeutic programmes, which will be announced in the coming months, it said in a statement.

This is one of the partnerships that OTXL is developing with global biotechs dedicated to rare diseases, pharmaceutical companies, CROs (Specialised Clinical Research Companies), CMDOs (Specialised Pharmaceutical Manufacturing Companies) and providers of Artificial Intelligence-based platforms.

The aim of the partnerships is to resume the development of promising therapies that were in the clinical trials phase but were discontinued by the companies due to financial and market challenges.

"Our aim in supporting this ambitious project is to make sure that some of these treatments can be investigated again, namely in clinical trials, so that they have a significant impact for patients, as well as a substantial value for those who invest in their development," explained the head of Rare Diseases at BIAL, Smitha Jagadish.

As a non-profit organisation, OTXL aims to work with regulators, investors and other stakeholders to change the research model in rare diseases and better serve patients and families, said OTXL founder and CEO Craig Martin.

More than 400 million people, half of them children, are affected globally by rare diseases, most of which are genetic, progressive and chronic diseases.

There are currently more than 11,000 diagnosed rare diseases of which 95% are untreatable.

VP/AYLS // AYLS

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