This was a two day conference with an interesting and diverse programme focusing on rare diseases and orphan drugs, with plenary sessions, networking sessions, and parallel sessions on specific topics such as clinical development, gene therapies, rare disease advocacy groups, and very useful roundtable discussions to addressing a particular question or challenge about clinical studies on rare diseases. Overall, there was a focus on cell and gene therapy.
This is a great opportunity to reinforce our connections and partnerships with pharmaceutical companies already working on ataxia, such as Biomarin and Reata. For that meeting, we also made new contacts with companies who have an interest in ataxia, such as Jupiter Orphan, and we have engaged with them after this conference. Another opportunity was to speak to companies dealing with early access to medications for patients with a rare condition and who can support pharmaceutical companies in extending clinical studies with an open label use of the orphan drug being tested. This is called ‘extended access’ to drugs in the US and ‘compassionate use’ in Europe. After the conference Ataxia UK, then spoke to a company called Wep Clinical, who has offices in both the US and the UK, to explore ways in which we could partner with them.
There was another event running in parallel called ‘Pitch and Partners’ where industry representatives were presenting some aspect of their research programme and updates on clinical studies. Of interest to the ataxias was a presentation by Biohaven on their study using a modified form of Riluzole; the molecule is called Trigriluzole. After a small study (8 weeks), they reported results showing not significant benefit for patients with SCAs. I was able to ask questions about their future plans; Biohaven have decided to extend the study to 52 weeks and focus on SCAs for now. They might also look into other forms of ataxias in the future.
