An interesting development. There were two Phase 1/2...

An interesting development.

There were two Phase 1/2 announcements made by Biogen/Ionis.

Firstly, Phase 1/2 results of an Ionis antisense therapy for the neurodegenerative disease, ALS, indicated that the drug was safe but unlikely to be effective. Biogen had an option to license the program but chose not to exercise its right and both parties agreed to terminate the program.

Following Phase 1/2 results, Biogen also opted not to exercise its option over ION-582, an Ionis antisense therapy for Angelman syndrome. It didn’t say why. Walking away from ION-582 means that Biogen will no longer have any neurodevelopmental drugs in its extensive CNS pipeline. As for potential acquisitions, Biogen CEO, Chris Viehbacher has said this week that Biogen won’t be doing any large deals this year, such as its US$7.3bn acquisition of Reata last year. But he said he could be interested in 2025.

Ionis is happy enough with the ION-582 results that it has opted to continue with development itself. The drug was found to be safe and tolerable. It was reported that roughly two thirds of the 51 participants in the open label study demonstrated improvement in cognition, communication and motor skills exceeding changes seen in a natural history study over a 6 month period. Further details of the study results won’t be released until July. In the meantime, the Company is planning to meet with the FDA to discuss its plans for a pivotal trial.

Last June, Roche announced that it would abandon further development of rugonersen, its antisense therapy for Angelman syndrome, following release of Phase 1 data. Again, the drug was considered to be tolerable and safe but Roche said it didn’t meet its “strict criteria” for advancement. Reading between the lines, the efficacy signals weren’t strong enough.

There is a third antisense therapy in development for Angelman syndrome – Ultragenyx’s GT-102. Its Phase 1/2 trial commenced in early 2020. Phase 1/2 data was positive, with improvements seen in cognition, communication, behaviour and sleep following 6 months of treatment. After positive communications with the FDA, Ultragenyx is hoping to commence a pivotal 100-120 placebo-controlled trial later this year.


Ionis announces positive topline results from Phase 1/2a trial of ION582 for Angelman syndrome | Ionis Pharmaceuticals, Inc.

Biogen walks away from Ionis-partnered ALS, Angelman prospects (fiercebiotech.com)

Roche drops antisense drug after seeing early efficacy data (fiercebiotech.com)

Events & presentations—Ultragenyx Pharmaceutical Inc.

Biogen CEO Looks for Small Deals While Alzheimer’s Drug Ramps Up (msn.com)