AesRx is developing two orphan drugs. Aes-103 for sickle cell disease is a potential blockbuster. Aes-210 for pouchitis is in Phase 2
Aes-103 is a novel, orally bioavailable small molecule drug for sickle cell disease (SCD). AesRx is developing Aes-103 in collaboration with the NIH. A Phase 1 trial is complete and a Phase 1/2a trial in SCD patients is expected to begin at the NIH in 2Q12. There are 75,000-100,000 SCD patients in the US and 40,000 in the EU. The FDA has designated Aes-103 as an Orphan Drug. JP Morgan estimates the market for a SCD drug to be $4+ billion.