Biogen and Skyhawk Partner to Develop Small Molecule Therapies for Neurological Diseases

Biogen and Skyhawk Therapeutics have created a strategic partnership that will allow both companies to use Skyhawk’s SkySTAR technology platform for the discovery of new small molecule treatments for neurological diseases, including multiple sclerosis and spinal muscular atrophy.

Under the terms of the agreement, Biogen will be given an exclusive license to the worldwide intellectual property rights associated with the therapeutic candidates. The company will have the option to license any resulting therapy from this collaboration, and will be responsible for their development and potential commercialization.

Skyhawk was paid $74 million upfront by Biogen, and is eligible to receive further milestone payments and royalties. One portion of the $74 million payment will be used to future research services; the rest of the payment will be allotted in the first quarter of 2019 for further research and development.

The STAR technology — which stands for “small molecule therapies for alternative splicing in RNA” — was developed to correct genetic defects at the mRNA level.

“Skyhawk’s platform offers a powerful approach to target neurological conditions using selective RNA-modulating small molecules, creating exciting possibilities for potential new therapies,” Michael Ehlers, MD, PhD, executive vice president of research and development at Biogen, said in a press release.

DNA is used as a template to make a molecule called mRNA, which, in turn, is used as a template to make proteins. Many neurological diseases develop due to a process called exon skipping, which causes important regions within the mRNA molecule to be “skipped” from the final mRNA sequence during a process called mRNA splicing.

Skyhawk’s SkySTAR technology platform is designed to help develop small molecules that can target specific binding regions on the mRNA molecule of interest. Specific small molecules that are most likely to succeed are tested for efficacy using patient cells — a method that can quickly inform of the molecule’s ability to reverse irregular mRNA splicing and treat the disease.

“Biogen is a leading neuro-focused biopharmaceutical company with a compelling history of drug development across a range of challenging neurological conditions,” said Bill Haney, co-founder and chief executive officer of Skyhawk. “We look forward to working with their team with the goal of potentially enhancing the treatment options we could bring to the neuroscience community.”