Pair of deals boosts Biogen’s early-stage CNS pipeline


Biogen has signed a brace of collaborations to expand its central nervous system (CNS) pipeline, adding new candidates for Alzheimer’s, multiple sclerosis and other neurological diseases.

The first of the new deals is a $415 million collaboration with protein degradation specialist C4 Therapeutics, focusing on Alzheimer’s, Parkinson’s and other neurological diseases, while Biogen is also paying Skyhawk Therapeutics $74 million upfront to license therapeutic candidates for MS, spinal muscular atrophy (SMA) and other diseases developed using its RNA platform, plus undisclosed milestones.

The two deals continue Biogen’s tight focus on neurology – first pursued in 2016 when it spun off its haemophilia business into Bioverativ (since acquired by Shire) – but contribute to an ongoing effort to reduce the company’s reliance on a portfolio of fast-maturing MS therapies headed by Tecfidera (dimethyl fumarate) which has started to see sales flatten.

Biogen currently has five potential Alzheimer’s treatments and one Parkinson’s treatment in development, and analysts have become concerned that its focus on tough-to-treat diseases make its pipeline very high risk despite other projects in stroke, MS, lupus and other diseases.

C4T’s platform focuses on the use of small-molecule drugs – called degronimids – that can target, degrade and clear proteins through the ubiquitin/proteasome pathway, and could potentially target proteins that so far have proved to be “undruggable.”

Skyhawk’s approach is to develop small-molecule drugs that can correct RNA mis-splicing leading to loss of RNA expression by exon-skipping. It is another way to go after hard-to-target disease mechanisms and according to Skyhawk could have particular utility in diseases caused by the loss of function of key proteins that are hard to tackle with antisense drugs or gene therapies.

So far, neither companies’ programmes have progressed into the clinic, but that hasn’t stopped them signing up big-name partners. Skyhawk signed a five-year strategic partnership with Celgene last year – also in the neurological disease area – which netted an upfront fee of $60 million and a $40 million equity investment. It’s not yet clear whether that could be affected by Celgene’s $74 billion merger with Bristol-Myers Squibb announced last week.

Meanwhile, last week C4T announced another new collaboration alongside its Biogen alliance, revealing it has signed an expanded deal with Roche on new cancer treatments that could now be worth up to $900 million. The targets and indications in the programme haven’t been revealed.

Roche and C4T  have been working together since 2016, but have now firmed up that initial arrangement – which was valued at $750 million – and added to the total deal value. Under the terms of the deal C4T will carry out discovery and development work through to early clinical development, with Roche taking an option on the right to continue development thereafter.