February 12, 2024
By Jeanne Philpott
The US Food and Drug Administration (FDA) has granted an orphan drug designation (ODD) to US-based biotech Disc Medicine’s DISC-3405 to treat rare blood cancer polycythemia vera (PV).
Disc gained exclusive rights to develop and market the anti-TMPRSS6 (Transmembrane Serine Protease 6) humanized antibody in the US and other territories when it teamed up with Mabwell Therapeutics in a $412.5m licensing agreement in January 2023.
In October 2023, DISC initiated an ongoing Phase I clinical trial (NCT06050915) for DISC-3405, previously named MWTX-003 with data from the study now expected in H1 2024. The orphan drug designation comes after the drug had previously received fast-track designation from the FDA.
The 64-patient Phase I study is divided into groups receiving either a single or multiple doses of DISC-3405, or a placebo. In the single ascending dose (SAD) phase, two subjects serve as sentinels: one receives DISC-3405, and the other placebo. Additional subjects in the cohort are dosed at least 24 hours after the last sentinel dosing, following approval from the principal investigator. Subsequent multiple ascending dose (MAD) cohorts are enrolled only after a sufficient safety observation period for the SAD cohort, removing the need for sentinels in MAD cohorts.