ABI-009 is a clinical phase, ‘best-in-class’, targeted mTOR inhibitor with demonstrated safety and evidence of efficacy. A Registration enabling trial (endpoints pre-negotiated with the FDA) is ongoing in Advanced Malignant PEComa (a rare aggressive form of sarcoma). ABI-009 has Orphan Designation, Fast Track Designation and the registration trial completion and NDA filing is targeted for 2019.
ABI-009 has a unique and advantageous clinical and pharmacological profile including efficient CNS penetration. Unlike other drugs in its class, ABI-009 is being developed in highly select populations in diseases driven by mTOR activations in oncology, cardiovascular and CNS diseases with an eye towards de-risked and capital efficient path to registration.