BIAL signs agreement with biotech to speed up research into rare diseases
BIAL, a 100-year-old biopharmaceutical company with a strong commitment to innovation, will be a founding member of the Orphan Therapeutics Accelerator (OTXL), a non-profit biotech established in June 2024 with the aim of addressing the challenges of researching and commercialising therapies for rare diseases, which lead to many promising treatments being abandoned or shelved.
This agreement is part of a set of partnerships that OTXL is developing with global biotechs focused on rare diseases, CROs (Contract Research Organisations), CMDOs (Manufacturing and Development Organisations) and providers of Artificial Intelligence-based platforms, with a view to restoring development of promising therapies that were in clinical trials but were discontinued by the companies working on them due to financial and market challenges.
BIAL, as one of the founding members, will play a key role in the selection, final stages of development and commercialisation of a number of therapeutic programmes, which should be announced in the coming months.
Through strategic funding from BIAL and other partners, OTXL plans to start work in the near term on two to three therapeutic programmes targeting ultra-rare diseases in great need of treatment, then expand to others, utilizing a portion of revenues from commercialising the initial programs. This partnership reinforces BIAL's commitment to working with other pharmaceutical and biotech companies, academia, R&D centres of excellence and patient.
“There is a lot of promising science sitting on the sidelines that could be benefitting children and others facing rare diseases,” said Smitha Jagadish, Head of Rare Diseases of BIAL. “Often these treatments have delivered meaningful results for patients in clinical trials but research in the field of rare diseases cannot always be approached from a traditional perspective. Our aim in supporting this bold effort is to get more of these treatments back into clinical trials so they may deliver meaningful impact for patients as well as substantial value to those who invest in their development.”
“Many of us involved in this venture have been working in rare diseases for decades,” stated Craig Martin, Founder and CEO of the OTXL. “We are focused on taking advantage of opportunities as a non-profit to work with regulators, payors, investors and other stakeholders to completely shift the model to work better for patients and families.”
Rare diseases are mostly genetic, progressive and chronic and affect more than 300 million people globally, half of them children. There are currently around 11,000 rare diseases, 95 per cent of which are untreatable. Although advances in genomic medicine, including cell and gene therapies, have opened up new possibilities, access to effective therapies is still a significant challenge.
About BIAL: BIAL is a 100-year-old innovation-orientated biopharmaceutical company that seeks to improve the lives of people around the world. As a company strongly committed to therapeutic innovation, BIAL has established an ambitious R&D programme, consistently investing more than 20% of its annual revenues in this area. The company's main areas of focus are neurosciences and rare diseases. In Europe, BIAL has a production unit and an R&D unit in Portugal (headquarters) and subsidiaries in several European countries - Spain, Germany, the UK, Italy and Switzerland. In addition, BIAL is present in the USA and in some emerging markets. The company's internationalisation strategy has also involved establishing partnerships and licensing agreements, collaborating with well-established partners to bring its healthcare solutions to everyone who needs them. BIAL products are present in more than 50 countries, fulfilling its purpose of making a real difference in the lives of people living with severe illnesses around the world. For more information about BIAL, please visit: www.bial.com
About the Orphan Therapeutics Accelerator: The Orphan Therapeutics Accelerator (OTXL) is a patient-centered non-profit biotech focused on obtaining, reinitiating development and commercialising promising “shelved” clinical-stage treatments for ultra-rare conditions. When an OTXL-developed treatment is approved and generates revenue, a portion is returned to the Orphan Therapeutics Accelerator to fund additional programs. For more information, please visit: orphantxl.com
