Santhera, reeling from DMD blow, lands cystic fibrosis drug

Human neutrophil elastase (Nevit Dilmen/CC BY-SA 3.0)

Santhera Pharmaceuticals has licensed a clinical-phase cystic fibrosis asset from Polyphor. The Swiss biotech penned a CHF 127.5 million ($138 million) deal for the human neutrophil elastase inhibitor to expand its clinical pipeline.

Polyphor, another Swiss biotech, will pocket CHF 6.5 million upfront, with up to CHF 121 million more to follow if the drug—POL6014—passes a series of milestones. Santhera said the payments are tied to “development, regulatory and particularly sales milestones,” implying the deal is backloaded and it will only pay the bulk of the amount if POL6014 comes to market.

POL6014 is some way from that point. Polyphor has taken the drug through a phase 1b supported by cash from Cystic Fibrosis Foundation Therapeutics, the former nonprofit drug discovery wing of the Cystic Fibrosis Foundation.


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Polyphor was gearing up for a multiple ascending dose study when Santhera formalized its interest in the asset. Santhera will now take charge of that trial, which is due to get underway in the second half of 2018. Beyond that, Santhera is planning to talk to regulators in the U.S. and Europe about a clinical trial program spanning cystic fibrosis and other indications.

RELATED: Small cap Santhera slides on DMD setback

The rationale for developing POL6014 centers on the role neutrophil elastase plays in the disease. The enzyme is produced by the neutrophils that proliferate in patients with cystic fibrosis, leading to lung tissue damage and other negative outcomes. POL6014 is designed to inhibit the enzyme. 

Santhera and Polyphor think that delivering POL6014 to the lung, while keeping systemic exposure low, will mitigate the progressive lung damage at the root of the deterioration of cystic fibrosis patients. The mechanism also lends itself to the treatment of other diseases in which neutrophil elastase plays a role, including non-cystic fibrosis bronchiectasis and alpha-1 antitrypsin deficiency.

Acquiring the global rights to POL6014 moves Santhera into a new field. The biotech has focused to date on mitochondrial diseases, including Duchenne muscular dystrophy (DMD). But that strategy took a hit last year when the European drug review board rejected a filing to expand use of Raxone to DMD patients. Santhera appealed but was rejected once again last month.

Shares in the biotech are down more than 60% over the past year.

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