Bristol-Myers Squibb in Deals with Biogen, Roche to License Potential Therapies for Progressive Supranuclear Palsy, Duchenne

José Lopes, PhD avatar

by José Lopes, PhD |

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Duchenne MD protein

New York-based Bristol-Myers Squibb announced two separate agreements with Biogen of Cambridge, Mass., and Switzerland’s Roche to license compounds that aim to treat progressive supranuclear palsy (PSP) and Duchenne muscular dystrophy (DMD).

Tau protein contributes to the stability of basic cellular structures known as microtubules to transport molecules. Abnormal addition of phosphate groups to tau is a hallmark of several diseases, including Alzheimer’s disease, frontotemporal dementia and PSP.

Although predominantly intracellular, extracellular Tau (eTau) has been observed in preclinical studies. Increasing evidence has shown that eTau can form aggregates that cause intracellular aggregation and cell death.

The agreement with Biogen involves the compound BMS-986168, an antibody engineered to bind to and decrease the levels of eTau protein. It calls for Biogen to pay Bristol-Myers Squibb $300 million up front, with potential milestone payments of up to $410 million.

Currently, a multi-center Phase 1 trial – CN002-003 (NCT02460094) – evaluating safety, tolerability and pharmacology of BMS-986168 in patients with PSP is undergoing. Phase 2 study (NCT03068468), not yet recruiting patients, should take place in 2019. Beyond PSP, this compound shows potential for future development in other neurodegenerative diseases, including Alzheimer’s.

In a similar deal, Roche will pay Bristo-Myers Squibb $170 million up front, with potential milestone payments of up to $205 million for compound BMS-986089 — a fusion protein designed to suppress the protein myostatin, a negative regulator of muscle growth. The use of myostatin inhibitors for treating muscle wasting disorders is an increasingly popular strategy aiming to increase muscle strength and function.

These agreements are subject to regulatory clearance and are expected to close sometime in the next few months.

BMS-986089 is currently being investigated in a Phase 1/2 clinical trial (NCT02515669) evaluating its safety and tolerability in boys with DMD. A Phase 2/3 study (NCT03039686), not yet recruiting patients, is expected to be finalized in 2020.

“Licensing these assets to Biogen and Roche will enable Bristol-Myers Squibb to prioritize the other promising opportunities for asset development that have advanced across our diversified portfolio,” Mike Burgess, MD, PhD, head of cardiovascular, fibrosis and immunoscience development at Bristol-Myers Squibb, said in a press release. “We recognize the significant unmet medical needs for patients with PSP and with DMD, and are pleased to put the future development of these compounds into the hands of Biogen and Roche, who both have strong capabilities, focus and leadership in neurodegenerative and rare diseases.”