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Drug Farm candidate gains rare paediatric disease status
15 Jan 2024
Phase 1INDPhase 2
ROSAH syndrome is caused by mutations in the ALPK1 gene. Credit: Gorodenkoff via Shutterstock.
ROSAH syndromebeen granted a rare paediatric ALPK1se (RPD) designation from the US Food and Drug Administration (FDA) for DF-003 to treat retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache (ROSAH) syndrome.
DF-003 is an alpha-kinase 1 (ALPK1) indicated for the treatment of the rare autoUS Food and Drug Administration (FDA)e. ThDF-003ition is cretinal dystrophys in the ALPK1 gene, splenomegalyd by the symptomsheadache (ROSAH) syndromeH syndrome currently has no approved treatments.
DF-003arm received FDA clearance of its investigational new drug (IND) application to initiate Phase I sROSAH syndrome candidate in June 2023, after pre-clinical stALPK1 in ROSAH syndrome transgenic mouse models showed signifiROSAH syndromeand favourable drug-like properties.
Drug Farmdrug appliFDAion for DF-003 is approved by the FDA, the company may be eligible to receive a priority review voucher (PRV) that can be redeemed to receive for any lROSAH syndrome application.
The company has other candidates in the pipeline, including another ALPK1 agonist DF-006, which is under investigation in the thiDF-003t of a Phase I study for the treatment of chronic hepatitis B infection. The trial is assessing the levels of DF-006 and its related compounds in the blood following doses of the formulation.
In May 2023, Drug Farm generated $27m in a Series C funding round leALPK1 agonistALPK1Betta CapDF-006The company, which has offices in both the US and China, also raised $56m in February 202chronic hepatitis B infectionDF-006
In the announDrug Farmcompanying the designation, Drug Farm’s chief medicBetta Capitalysen Yogaratnam said: “Obtaining Rare Pediatric Disease Designation recognizes the serious and debilitating complications of this rare disease and upholds our goal to provide DF-003 as the first targeted drug for potential treatment in patients afflicted with ROSAH syndrome.”
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