- Ablynx NV stock leaped on Monday after the company released positive topline data from a late-stage study that tested its lead candidate in patients with a rare blood disorder.
- The HERCULES study assessed Ablynx's caplacizumab as a treatment for acquired thrombotic thrombocytopenic purpura (aTTP), a disease that causes blood platelets to stick together, thereby inducing clots. All study participants received standard of care therapy, but those on caplacizumab took significantly less time for their blood platelet counts to reach normal levels — the study's primary endpoint — than those on placebo.
- The drug also met key secondary endpoints. The experimental arm, for instance, showed a 74% relative reduction in the percentage of patients who experienced at least one major thromboembolic event, saw their aTTP return or died from the disease during the testing period.
Ablynx has a well-stocked pipeline, yet few of its assets are in the more advanced stages of clinical study. The company's two most prominent candidates are caplacizumab and the rheumatoid arthritis drug vobarilizumab, though the former appears to have a little better of an outlook. Last year, AbbVie exited a co-development partnership on vobarilizumab, leaving Ablynx to finance expensive late-stage testing solo.
While the Beligum-based drugmaker remained confident it had enough cash to keep developing vobarilizumab, CEO Edwin Moses said he and his team were looking for a potential partner for commercialization.
Bringing caplacizumab to market, however, could be a boon to the company's revenues and overall cash position. The rare disease landscape is growing at a rapid clip, and market research firms anticipate specialty and orphan drugs to take up bigger portions of drug spend in the coming years.
Ablynx intends to tack on the HERCULES data to the Marketing Authorization Application it submitted to European regulators in February. The company also plans to submit a Biologics License Application with the Food and Drug Administration in the first half of 2018. Should caplacizumab make it through those hurdles, it would be the first drug specifically approved for aTTP.
On an Oct. 2 investor call, Moses said his company intends to have 100 people working on the commercialization of this lead product across the U.S. and Europe. Ablynx plans to hire through contract sales organizations, and further establish its U.S. operations — in part by setting up a subsidiary and bringing on medical science liaisons in the 12 months before caplacizumab's stateside commercialization, which is predicted for 2019.
"We will now proceed with that very quickly," Moses said.
Ablynx shares opened at €14 ($16.45) apiece Monday, up 12% from Friday's close. They climbed higher later in the morning, reaching a peak of €16.30 ($19.15) per share.