Orphazyme agrees to sell itself, and its 'pipeline-in-a-product,' to KemPharm
Two months ago, Orphazyme enlisted the help of the Danish courts to help oversee a restructuring in the wake of trial failures, C-suite shakeups and regulatory rejections. The move was expected to help the company find a way to either sell itself or its assets, attempting to keep the research going on what it once called its “pipeline-in-a-product.”
On Sunday, the resolution of the court-run efforts came into view.
KemPharm has agreed to acquire Orphazyme and all assets and operations, the companies announced Sunday evening, including the once-hyped drug candidate arimoclomol. KemPharm will pay $12.8 million in cash for the transaction and assume liabilities equal to about $5.2 million. The deal is expected to close before June 1.
In addition, the Florida-based biotech intends to keep on the “majority” of Orphazyme’s remaining employees, though the exact number is not clear. Endpoints News has reached out for comment and will update this story accordingly.
Investor reaction to the news was muted, with KemPharm shares $KMPH down roughly 3% in early Monday trading.
The acquisition effectively marks the end of Orphazyme’s story, which began with high hopes for its sole drug as it pitched arimoclomol as a potential treatment for a wide range of CNS diseases. Though the biotech had submitted its NDA for Niemann-Pick disease type C, Orphazyme had also tested it — or planned to test it — in ALS, Gaucher disease and inclusion body myositis.
Orphazyme’s June 2021 CRL apparently proved too tough to overcome on its own, however. The rejection had been just one setback in a string of bad news and followed multiple pivotal trial misses and its CEO’s abrupt resignation in late 2020. The chief executive’s replacement also departed in February for Leo Pharma.
As the drug heads over to KemPharm, it’s evident from the company’s announcement it believes it can change arimoclomol’s fortunes. CEO Travis Mickle said in a statement that it has “carefully evaluated” the CRL and other FDA correspondence and intends to resubmit the NDA in Niemann-Pick disease type C “as early as” the first quarter of 2023.
“We believe the efficacy signal for arimoclomol in NPC is convincing and that there is a viable regulatory path that could enable a successful NDA resubmission,” Mickle added.
KemPharm is keeping details on that potential pathway close to the vest for now, but may have more answers in a Monday morning investor event. The biotech cited its past experience working through CRLs as a reason it feels it can get arimoclomol across the finish line, alluding to struggles with an abuse-deterrent opioid for acute pain.