Biogen bets $45M on Ionis' phase 1 Alzheimer's med

Biogen biologics plant in Denmark
Biogen's bet on Ionis comes after the company resurrected aducanumab, the amyloid-aimed drug that was deemed to fail two phase 3 studies. (Biogen)

Looks like Biogen is going all in on Alzheimer’s disease—the Big Biotech is picking up a tau-targeting treatment from Ionis Pharmaceuticals for $45 million upfront. 

Currently in phase 1, the antisense treatment, IONIS-MAPTRx, is designed to cut down on the production of tau proteins in the central nervous system. Formally known as MAPT (microtubule-associated protein tau), these proteins can form tangles in the brain and are thought to cause neurodegenerative diseases like Alzheimer’s and some types of dementia. However, Alzheimer’s research has focused on tau tangles and amyloid plaques but has not seen a new treatment approved in more than 15 years. Many treatments have shown promise through phase 2 before crashing and burning in phase 3. 

That said, Ionis—and Biogen—think antisense treatment has a better chance. 

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RELATED: Biogen resurrects aducanumab, arguing expanded data set supports FDA filing in Alzheimer's 

“In preclinical studies, MAPT-targeted antisense treatment demonstrated prevention and reversal of pathology,” Ionis said in a statement.  

In addition to the $45 million licensing fee, Ionis could rake in up to $155 million in milestone payments, as well as royalties. 

Under the deal, Ionis will complete the phase 1 study of IONIS-MAPTRx in patients with mild Alzheimer’s disease as well as a yearlong extension study. Biogen will pick up any studies that need to come after those along with responsibility for regulatory filings and commercialization, according to the statement. 

RELATED: 2019’s top 15 clinical trial flops (and a flip-flop) | Flip-flop: Aducanumab 

The move comes after Biogen resurrected aducanumab, the amyloid-aimed drug that a data committee earlier this year deemed likely to fail two phase 3 studies in mild Alzheimer’s. At the time, the company canned both studies and called off one that would have tested aducanumab in Alzheimer’s prevention. 

Then, Biogen announced in October that, actually, they’d gotten it wrong. The futility analysis was incorrect, and not only was the program back on track but the interim data were strong enough to allow it to ask the FDA to approve the drug. 

And that’s not all. The company revealed last week that gosuranemab, which it licensed from Bristol-Myers Squibb, failed a midstage test in progressive supranuclear palsy. Though it’s axing work in that brain disorder, the phase 2 study is going full steam ahead, testing the drug in—you guessed it—Alzheimer's. Biogen says this makes sense “given differences in disease pathology.” 

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