Some research on another orphan drug candidate which was the target of an acquisition several months ago...
In April this year, US biopharma Sucampo acquired the small US startup biotech, Vtesse, in a deal valued at US$200 million.
Vtesse’s sole drug candidate, VTS-270, is a potential therapy for the rare genetic condition Niemann-Pick Type C1 disease, which is a progressive and deadly genetic disease that affects 2,000 to 3,000 people globally, mostly children. There are no approved treatments for the disease which causes an abnormal build up of cholesterol in the brain, liver, and spleen and leads to developmental and neurological problems like ataxia.
Under the terms of the deal, shareholders of Vtesse, a privately owned company, received $170 million in cash and 2,782,678 shares of Sucampo, valued at the time at US$11. If the drug reaches market, Vtesse shareholders will also receive royalties on sales. Furthermore, they will be eligible for a share from any sales proceeds if Sucampo is successful in getting and selling a Priority Review Voucher. The deal came as Vtesse completed enrolment for a pivotal Phase 3 trial in 51 patients.
Results of a previous Phase 1/2a trial in 14 patients were released in March 2014, two months following the announcement that VTS-270 had received Breakthrough Therapy designation from the FDA. More detailed results of the trial were published in The Lancet this week. The open-label, dose-escalation Phase 1/2a trial studied safety, tolerability and clinical efficacy of intrathecal administration (injection into the spinal canal) of VTS-270 after 12 months and 18 months of treatment. The study demonstrated clinically meaningful reduction in signs and symptoms of disease progression as measured by the NPC Clinical Severity Scale. Compared with the historical data, half of the patients in this study saw an improvement or no worsening in the neurological severity score. Serious adverse events were limited to an acceleration of the hearing loss which is part of the natural progression of this disease.
The NPC Clinical Severity Scale (or NPC-SS) measures changes in domains such as eye movement, gait, speech, swallowing, fine motor skills, cognition, hearing, memory, and presence and severity of seizures, compared with a natural history cohort. In each category, patients can score zero to five points, with zero indicating normal function and five indicating severe disability or loss of that category of function. The scale was developed in recent years to characterize and quantify disease progression after recognition that the absence of a universally accepted clinical outcome measure was an impediment to the design of a therapeutic trial for Niemann-Pick. The NPC-SS is being used as the primary outcome measure in Sucampo’s current Phase 3 trial in which VTS-270 will be administered as a once-fortnightly injection for 12 months. Results are expected in mid-2018.
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