- Topline results from the Phase 1 study showed a favorable safety and tolerability profile with no drug-related severe or serious adverse events.
- Topline data from the multiple ascending dose cohorts of 40, 80 and 120 mg/kg demonstrated the average level (“Cavg“) of functional alpha-1 antitrypsin (“AAT”) achieved by INBRX-101 was 40.4 micromolar (“µM”) over the 21-day dosing interval following the third 80 mg/kg dose.
- Functional AAT levels collected from 65 healthy individuals with the MM genotype revealed a 5th/95th percentile range of 23 to 57 µM and a median of 38 µM.
SAN DIEGO, CA, USA I May 16, 2022 I Inhibrx Inc. (Nasdaq: INBX), a biotechnology company with four clinical programs in development and an emerging pre-clinical pipeline, today announced topline results from a Phase 1 clinical trial evaluating the safety, pharmacokinetics (“PK”) and pharmacodynamics (“PD”) of INBRX-101, an optimized recombinant human AAT-Fc fusion protein, in patients with alpha-1 antitrypsin deficiency (“AATD”).
Data from this multi-country Phase 1 study are from 31 patients with AATD: 26 with the ZZ genotype, 3 with the SZ genotype and 2 with the MZ genotype of the SERPINA1 gene, the underlying cause of AATD. Treatment was well tolerated with no severe or serious adverse events related to the study drug. Drug-related adverse events were predominantly mild and those few that were moderate in severity were all transient and reversible, with minimal or no symptomatic care. No safety-related or PK/PD-related signs of neutralizing anti-drug antibodies were observed.
Dose-related increases in maximal and total INBRX-101 exposure occurred across the entirety of the single and multiple ascending dose ranges.
Data from the multiple ascending dose cohorts of INBRX-101 at 40, 80 and 120 mg/kg IV every three weeks showed the expected accumulation of functional AAT levels. Based on PK modeling, accumulation is expected to continue following subsequent doses and reach a steady-state after a total of approximately five to six consecutive doses, administered every three weeks.
The current standard of care, plasma-derived AAT, dosed once weekly at 60 mg/kg, achieves a Cavg of functional AAT of 17.8 µM over the weekly dosing interval as calculated from steady-state area under the curve (“AUC”) values reported in Stocks et al. BMC Clinical Pharmacology 2010, 10:13. INBRX-101 achieved a mean Cavg of functional AAT of 40.4 µM over the 21-day dosing interval following the third 80 mg/kg dose.
To date, bronchoalveolar lavage fluid samples have been processed from two 80 mg/kg multiple ascending dose cohort individuals and confirm the presence of INBRX-101 in the lung fluid.
Additionally, functional AAT levels were measured in plasma samples from 65 normal MM genotype individuals. This analysis revealed the 5th and 95th percentiles of functional AAT levels in the normal MM genotype individuals were 23 and 57 µM, respectively, with a median of 38 µM.
“We believe this data demonstrates the potential of INBRX-101 to change the paradigm of treatment of AAT deficiency by maintaining patients in the normal range of functional AAT while reducing infusions from 52 annually to as few as 12 annually. We look forward to working with regulators, clinicians and patients to expedite this therapy to AAT deficient patients as rapidly as possible,” said Mark Lappe, CEO of Inhibrx.
The Company will host a live webcast presentation today, May 16th, at 1:30 p.m. PT to further discuss the results.
About INBRX-101 and AATD
INBRX-101 is a precisely engineered recombinant human AAT-Fc fusion protein designed to safely achieve and maintain levels of AAT found in healthy individuals with the potential for once-monthly dosing.
AATD is an inherited orphan disease affecting an estimated 100,000 patients in the United States. AATD is characterized by deficient levels of the AAT protein, which causes loss of lung tissue and function and decreased life expectancy. Plasma-derived AAT is the current standard of care and does not maintain patients in the normal AAT range, requires frequent and inconvenient once-weekly IV dosing, and relies on plasma collection practices that might not be sustainable.
About Inhibrx, Inc.
Inhibrx is a clinical-stage biotechnology company focused on developing a broad pipeline of novel biologic therapeutic candidates in oncology and orphan diseases. Inhibrx utilizes diverse methods of protein engineering to address the specific requirements of complex target and disease biology, including its proprietary sdAb platform. Inhibrx has collaborations with 2seventy bio (formerly bluebird bio), Bristol-Myers Squibb and Chiesi. For more information, please visit www.inhibrx.com.
SOURCE: Inhibrx
Post Views: 147
- Topline results from the Phase 1 study showed a favorable safety and tolerability profile with no drug-related severe or serious adverse events.
- Topline data from the multiple ascending dose cohorts of 40, 80 and 120 mg/kg demonstrated the average level (“Cavg“) of functional alpha-1 antitrypsin (“AAT”) achieved by INBRX-101 was 40.4 micromolar (“µM”) over the 21-day dosing interval following the third 80 mg/kg dose.
- Functional AAT levels collected from 65 healthy individuals with the MM genotype revealed a 5th/95th percentile range of 23 to 57 µM and a median of 38 µM.
SAN DIEGO, CA, USA I May 16, 2022 I Inhibrx Inc. (Nasdaq: INBX), a biotechnology company with four clinical programs in development and an emerging pre-clinical pipeline, today announced topline results from a Phase 1 clinical trial evaluating the safety, pharmacokinetics (“PK”) and pharmacodynamics (“PD”) of INBRX-101, an optimized recombinant human AAT-Fc fusion protein, in patients with alpha-1 antitrypsin deficiency (“AATD”).
Data from this multi-country Phase 1 study are from 31 patients with AATD: 26 with the ZZ genotype, 3 with the SZ genotype and 2 with the MZ genotype of the SERPINA1 gene, the underlying cause of AATD. Treatment was well tolerated with no severe or serious adverse events related to the study drug. Drug-related adverse events were predominantly mild and those few that were moderate in severity were all transient and reversible, with minimal or no symptomatic care. No safety-related or PK/PD-related signs of neutralizing anti-drug antibodies were observed.
Dose-related increases in maximal and total INBRX-101 exposure occurred across the entirety of the single and multiple ascending dose ranges.
Data from the multiple ascending dose cohorts of INBRX-101 at 40, 80 and 120 mg/kg IV every three weeks showed the expected accumulation of functional AAT levels. Based on PK modeling, accumulation is expected to continue following subsequent doses and reach a steady-state after a total of approximately five to six consecutive doses, administered every three weeks.
The current standard of care, plasma-derived AAT, dosed once weekly at 60 mg/kg, achieves a Cavg of functional AAT of 17.8 µM over the weekly dosing interval as calculated from steady-state area under the curve (“AUC”) values reported in Stocks et al. BMC Clinical Pharmacology 2010, 10:13. INBRX-101 achieved a mean Cavg of functional AAT of 40.4 µM over the 21-day dosing interval following the third 80 mg/kg dose.
To date, bronchoalveolar lavage fluid samples have been processed from two 80 mg/kg multiple ascending dose cohort individuals and confirm the presence of INBRX-101 in the lung fluid.
Additionally, functional AAT levels were measured in plasma samples from 65 normal MM genotype individuals. This analysis revealed the 5th and 95th percentiles of functional AAT levels in the normal MM genotype individuals were 23 and 57 µM, respectively, with a median of 38 µM.
“We believe this data demonstrates the potential of INBRX-101 to change the paradigm of treatment of AAT deficiency by maintaining patients in the normal range of functional AAT while reducing infusions from 52 annually to as few as 12 annually. We look forward to working with regulators, clinicians and patients to expedite this therapy to AAT deficient patients as rapidly as possible,” said Mark Lappe, CEO of Inhibrx.
The Company will host a live webcast presentation today, May 16th, at 1:30 p.m. PT to further discuss the results.
About INBRX-101 and AATD
INBRX-101 is a precisely engineered recombinant human AAT-Fc fusion protein designed to safely achieve and maintain levels of AAT found in healthy individuals with the potential for once-monthly dosing.
AATD is an inherited orphan disease affecting an estimated 100,000 patients in the United States. AATD is characterized by deficient levels of the AAT protein, which causes loss of lung tissue and function and decreased life expectancy. Plasma-derived AAT is the current standard of care and does not maintain patients in the normal AAT range, requires frequent and inconvenient once-weekly IV dosing, and relies on plasma collection practices that might not be sustainable.
About Inhibrx, Inc.
Inhibrx is a clinical-stage biotechnology company focused on developing a broad pipeline of novel biologic therapeutic candidates in oncology and orphan diseases. Inhibrx utilizes diverse methods of protein engineering to address the specific requirements of complex target and disease biology, including its proprietary sdAb platform. Inhibrx has collaborations with 2seventy bio (formerly bluebird bio), Bristol-Myers Squibb and Chiesi. For more information, please visit www.inhibrx.com.
SOURCE: Inhibrx
Post Views: 147
