Pharmalink has stopped a Phase IIb trial of its primary IgA nephropathy treatment early after it met its primary endpoint in a planned interim analysis. The success makes Pharmalink a rare example of a private biotech with an unpartnered, Phase III-ready asset, characteristics that inevitably lead to talk of IPOs and deals.
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| Pharmalink CEO Johan Häggblad |
A Data and Safety Monitoring Board (DSMB) found both doses of Nefecon--an oral, modified-release form of the corticosteroid budesonide--were associated with a significant drop in the urine protein creatinine ratio (UPCR) compared to placebo. With the DSMB calculating a p-value of 0.0066 on an intention-to-treat basis, the data were deemed strong enough to warrant stopping the 150-person trial. The treatment phase is due to stop at the end of April, after which Pharmalink will follow up with the participants for three months before publishing final data in the third quarter.
The speedy resolution is one of several ways in which the trial has exceeded expectations. Stockholm, Sweden-based Pharmalink set out aiming to enroll 90 patients, only for strong uptake to enable it to increase its target to 150 people. The performance suggests there is demand for treatments for primary IgA nephropathy, a disease caused by the buildup of immunoglobulin A in the kidneys. "No current therapies address the root cause of the disease. A new medicine ... would be very welcome news indeed," Uppsala University Hospital Professor Dr. Bengt Fellström said in a statement.
Pharmalink is now advancing its plans to bring such a medicine to patients, starting with preparations for a Phase III trial. Data from the Phase IIb trial of Nefecon--which is designated an orphan drug by FDA--will feed into the design and dosing of the next study. Pharmalink must also consider how to pay for the trial. The company last tapped investors in 2013, at which time it raised SEK 96 million ($11 million) in a Series C round. Nordic investors Investinor and Industrifonden co-led the round, which was designed to finance the Phase IIb Nefecon trial and late-stage work on Busulipo.
Nefecon is now a considerably more attractive asset than it was back in 2013. Pharmalink calculates there are 200,000 people with primary IgA nephropathy in major markets. And the disease can place significant burdens on healthcare systems. As many as two-fifths of patients develop renal failure and many go on to need renal transplants or long-term dialysis.
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