Phototoxicity and liver damage are significant challenges for individuals with Erythropoietic Protoporphyria (EPP), a rare genetic disorder caused by the accumulation of protoporphyrin IX (PPIX). At ASH 2024, Bhavik Shah and his team presented findings on PORT-77, a novel ABCG2 inhibitor that may help manage these effects.
Key findings:
PORT-77 inhibits ABCG2, reducing PPIX accumulation in the blood, skin, and liver.
Mice treated with PORT-77 showed reduced light-induced skin damage.
Long-term use was associated with lower liver PPIX levels and improved liver health.
Currently in a phase 1 study, assessing safety and pharmacokinetics.
These results suggest that PORT-77 may offer a potential therapeutic approach for patients with EPP.
Read more: https://lnkd.in/db3svATN
Thanks to Bhavik Shah and the research team, including Edmond Huang, Yvonne Pak, Chris Henry, Sunita Babbar, Athiwat Hutchaleelaha, Robert Zamboni, Xiaochao Ma, Pete Schmidt, Morgan Paull, and Uma Sinha for their contributions.
