Biogen has licensed an investigational Alzheimer’s treatment from longstanding collaborator Ionis, fronting $45m with the potential of an additional $155m payout.
The drug – IONIS-MAPTRx – is an antisense therapy designed to reduce production of microtubule-associated protein tau (MAPT) or tau in the central nervous system.
This protein is thought to contribute to or cause a number of neurodegenerative diseases, including Alzheimer’s disease and some forms of frontotemporal degeneration. It is one of two proteins commonly associated with Alzheimer’s, the other being beta-amyloid.
In pre-clinical research, IONIS-MAPTRx demonstrated prevention of the disease and reversal of pathology associated with tau.
“The licensing of IONIS-MAPTRx, currently in a phase 1 clinical study in mild Alzheimer’s disease patients, is an important milestone for the programme,” said Brett Monia, Ionis’ chief operating officer.
“It brings us another step closer to potentially delivering a therapy to patients who have few or no effective treatment options,” he added.
A lot of the pharma industry’s efforts in Alzheimer’s have been directed at the beta-amyloid plaques seen in the brains of patients – however, the attempt to bring candidates targeting beta-amyloid to market have all failed so far.
The only promising beta-amyloid-targeting candidate is Biogen’s aducanumab – the pharma industry was shocked when Biogen decided to revive the drug after originally abandoning it in phase 3.
However, according to Biogen, analysis of patients treated at the higher-dose were promising enough to warrant an application to the FDA.
In a much-anticipated presentation delivered earlier this month, Biogen unveiled a fresh look at the ENGAGE and EMERGE studies of aducanumab.
EMERGE gave a positive, if modest, readout for the high-dose arm on cognition scores, but ENGAGE didn’t show clinical efficacy for the drug and the consensus among experts is that the dataset doesn’t meet the FDA’s usual criteria for approval.
Alzheimer’s is notoriously lacking in treatment options however, so the mixed data could be enough to get a green light from the FDA.
Biogen and Ionis have already successfully brought a treatment to market – Spinraza (nusinersen), a treatment for amyotrophic lateral sclerosis (ALS). It also became the first drug in the US to be approved for spinal muscular atrophy in 2017.