Orphan drug designation granted to PT-112

Phosplatin Therapeutics have announced that their small molecule conjugate of pyrophosphate and platinum, PT-112, has been granted orphan drug designation by the US Food and Drug Administration (FDA). This status ensures financial incentives for companies developing drugs for rare, yet life-threatening conditions.

The drug is now being tested in phase I/II clinical trials as a single agent for the treatment of Relapsed and Refractory Multiple Myeloma (RRMM). The mechanism of action is thought to rely on damage associated molecular patters (DAMPs) that lead to T-cell activation, in particular in the bone marrow, making it a good candidate for MM. Pre-clinical data was presented at both ASCO and ASH in 2017, and early dosing studies that have tested the drug with solid tumors indicate a good overall tolerance. The agent is expected to lead to lower toxicity and less resistance than other chemotherapeutics.