Rare Disease Report

BMS Sells 2 of Its Orphan Drugs For $470 million To Treat PSP and DMD

APRIL 14, 2017
James Radke
Bristol-Myers Squibb (BMS) has turned a $175 million orphan drug investment into a $470 million payday.  In 2014, BMS bought iPierian and its 2 orphan drugs for $175 million. BMS is now selling those 2 drugs – BMS-986168 in development to treat progressive supranuclear palsy (PSP) and BMS-9866089 in development to treat Duchenne muscular dystrophy (DMD) – to Biogen for $300 million and Roche for $170 million, respectively.
 
Not bad for a 3 year investment.
 
Under the terms of the agreement, Biogen will pay to BMS an upfront payment of $300 million for BMS-986168 with potential milestone payments of up to $410 million. Biogen also will assume all remaining obligations to the former stockholders of iPierian, Inc. related to BMS’ acquisition of the company in 2014. Further, Roche will pay to BMS an upfront payment of $170 million for BMS-9866089 with potential milestone payments of up to $205 million.
 
BMS will also receive tiered double-digit royalties if either drug is approved and commercialized.

BMS-986168 and Progressive Supranuclear Palsy (PSP)

BMS-986168 is a monoclonal antibody designed to bind to and decrease levels of extracellular Tau (eTau) protein. A phase 1 study has been completed.
 
Progressive supranuclear palsy (PSP) is a progressive neurodegenerative disorder affecting approximately 20,000 individuals in the United States. The average onset of PSP symptoms typically begins after age 60. The most common features of PSP are loss of balance leading to unexplained falls, blurred vision, problems controlling eye movement and slurred speech.
 
Since early signs of PSP can mimic other neurological conditions such as Parkinson’s disease, a PSP is often delayed.
 
Currently, there are no approved treatments for PSP.
 
BMS-98168 may also be of benefit to patients with Alzheimer’s disease and the addition of the drug to Biogen’s pipeline for neurodegenerative disease could bring great value to the company.

BMS-986089 and Duchenne Muscular Dystrophy (DMD)

BMS-986089 is a novel fusion protein designed to suppress myostatin, a negative regulator of muscle growth. A phase 1 /2 study is underway.
 
DMD is caused by lack of a functional dystrophin protein, a protein that helps keep muscle cells intact. Patients with progressive muscle disorder experience symptoms in early childhood, losing the ability to walk as early as age 10. These patients, mostly boys, experience life-threatening heart and lung complications in their late teens and twenties.

There are many subsets of the Duchenne population based on the type of mutation found in the dystrophin gene.

Currently, the only approved drugs for DMD are Sarepta’s Exondys 51 (eteplirsen), that treats approximately 13% of those in the DMD population with a specific mutation of the dystrophin gene susceptible to exon 51 skipping, and PTC Therapeutics’ glucocorticoid  Emflaza (deflazacort). BMS-986089 would theoretically be effective in all DMD patients.
 


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