Small molecule drug shows promise in rare disease

The CBL-0201DD Phase II study by Caliway Biopharmaceuticals evaluated the first-in-class small-molecule drug for the treatment of Dercum’s disease, a rare disorder characterised by painful lipoma growth on the torso, which has shown significant reductions in lipomas size and achieving complete clearance. There are currently no approved or effective medications for this condition, making the Phase II study a promising potential treatment for the disorder.

Twelve participant were selected to analyse the safety and efficacy of CBL-514 injections. The primary objective revealed that following CBL-514 treatments, the dimensions of painful lipomas in the high-dose group reduced by 51.3% and 54.7% at 4 and 8 weeks. In contrast, the dimensions of painful lipomas in the low-dose group were reduced by 40% and 34.7% at 4 and 8 weeks post-CBL-514 treatments.

The secondary objective showed that compared to the control group there was a 90.3% reduction in the dimensions of painful lipomas in the high-dose group after CBL-514 treatment. From the patients with Dercum’s disease who received the high-dose, 64.5% exhibited either complete clearance or recorded lipoma dimensions reducing by more than half the size after CBL-514 treatments when compared to baseline.

Caliway revealed that the global market for Dercum’s disease treatment reached $11.3 billion in 2021 and anticipates significant growth with a compound annual growth rate (CAGR) of 6.76%. The prediction is that the global Dercum disease treatment market will reach $19.95 billion by 2030. This is promising in ensuring that treatment for Dercum patients can surpass the testing stages and become approved, accessible, and on the market.

Detailed efficacy and safety results from the Dercum’s disease CBL-0201DD study will be published in the Journal of Rare Diseases. Currently, Caliway is planning for the CBL-0202DD Phase IIB study IND application to further investigate CBL-514.