Sucampo buys rare disease firm Vtesse

US biotech Sucampo is to buy privately-held rare disease specialist Vtesse for $200 million, gaining rights to a drug for the rare genetic disorder Niemann-Pick Disease Type C1 (NPC-1).

Sucampo will pay up front, having funded the acquisition through issuing common stock and with its own cash reserves without external financing.

US-based Vtesse has one drug, VTS-270, which is in a phase 2b/3 pivotal trial for NPC-1, a genetic lysosome storage disease that causes early death in the vast majority of cases.

Neurological symptoms, often beginning in childhood, include dementia, difficulties walking and tremor, progressing to the point where the patient is completely bedridden prior to eventual death.

There is no US-approved treatment for NPC-1, which occurs once in every 100,000-150,000 births with around 2,000-3,000 cases estimated globally.

Results of a fully-enrolled global pivotal clinical trial are expected in mid-2018, with a launch possible in 2019 if the drug is approved.

VTS-270 is a well-characterised mixture of 2-hydroxypropyl-ß-cyclodextrins (HPßCD) with a specific compositional fingerprint that distinguishes it from other HPßCD mixtures.

It is administered by an intrathecal infusion to directly address the neurological manifestations of the disease.

Vtesse has only been in business for around two years after spinning out of incubator Cydan.

The acquisition comes as Sucampo attempts to rebuild its pipeline after it scrapped development of gastro drug cobiprostone in non-erosive reflux disease (NERD) and or symptomatic gastroesophageal reflux disease (sGERD).

Sucampo will instead develop cobiprostone for oral mucositis – mouth ulcers commonly associated with cancer chemotherapy.

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