“The update shared this week by Inhibrx on their Recombinant Alpha-1-antitrypsin (INBRX-101) is exciting news for the Alpha-1 Community. The potential for a once a month dosing of Alpha-1-antitrypsin could be a very important advance in therapies for Alpha-1-antitrypsin deficient individuals. We are excited for the positive progress in their research and look forward to the completion of Inhibrx’s development of this novel treatment for Alphas,” stated Mark Brantly, MD, Scientific Director, Alpha-1 Foundation.
Interim functional PK data from this multi-country multi-center Phase 1 study are from 21 patients with AATD, all with the ZZ mutation of the SERPINA1 gene, the underlying cause of AATD. Interim safety data are from 24 patients with AATD. There were no drug-related severe or serious adverse events at doses up to and including 120 mg/kg single dose and 80mg/kg multi-dose. Drug-related adverse events were predominantly mild with a few moderate events and all were transient and reversible. No signs of neutralizing anti-drug antibodies have been seen.
Dose-related increases in maximal and total INBRX-101 exposure occurred across the entirety of the tested single ascending dose range of 10 to 120 mg/kg.
Data from the first multiple ascending dose cohort of INBRX-101 at 40 mg/kg IV every three weeks showed the expected accumulation of functional alpha-1 antitrypsin, or AAT, levels with observed trough levels exceeding the goal of the current standard augmentation therapy with plasma-derived AAT.
Functional AAT and bronchoalveolar lavage data from patients to be administered three INBRX-101 doses of 80 or 120 mg/kg IV every three weeks are expected in the first half of 2022.
“These initial data demonstrate the potential of INBRX-101 to significantly improve treatment for patients with AATD by maintaining them in the normal range of functional AAT and reducing infusions from 52 annually to possibly as few as 12 annually,” said Mark Lappe, CEO of Inhibrx.
The Company will host a live webcast presentation today at 5:30 a.m. PT to further discuss the results.
Source: Inhibrx
