Inhibrx's Experimental Drug For Inherited Disease Capable For Accelerated FDA Approval

 

• Based on discussions with the FDA, Inhibrx Inc INBX has the potential to pursue accelerated approval for INBRX-101 for emphysema due to alpha-1 antitrypsin deficiency (AATD) using functional alpha-1 antitrypsin (AAT) serum levels as the surrogate endpoint. 
• Inhibrx also detected INBRX-101 in the bronchoalveolar lavage fluid (BALF) samples from all AATD patients tested in the Phase 1 study.
• Inhibrx plans to initiate in Q1 of 2023 a potential registration-enabling trial using functional AAT as a surrogate endpoint.
• Based on data from the completed Phase 1 study, INBRX-101 is predicted to maintain patients above the lower threshold of the normal range and achieve an average level of functional AAT.
• The FDA also requested additional data on the correlation between functional AAT levels and the clinical benefit of AATD to further support serum AAT levels as a surrogate endpoint that is reasonably likely to predict clinical benefit.

https://www.benzinga.com/general/biotech/22/10/29136708/inhibrxs-experimental-drug-for-inherited-disease-capable-for-accelerated-fda-approval