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Krystal Biotech Secures FDA Authorization for Investigational New Drug Application (IND) of KB408 to Address Type 1 Alpha-1 Antitrypsin Deficiency

Friday, September 22, 2023

Krystal Biotech, Inc. (NASDAQ: KRYS), a biotechnology firm specializing in genetic medicines for diseases with significant unmet medical needs, has obtained FDA clearance for its Investigational New Drug Application (IND) for KB408, designed to address alpha-1 antitrypsin deficiency (AATD).

KB408 is a modified, non-integrating HSV-1-derived vector carrying two full-length copies of the SERPINA1 gene, enabling the expression of alpha-1 antitrypsin (AAT). This formulation is designed for delivery to lung respiratory cells through nebulization.

Hubert Chen, M.D., Senior Vice President of Clinical Development at Krystal Biotech, expressed enthusiasm for advancing KB408 into clinical trials, emphasizing its potential to tackle a severe lung disease for which treatment options are limited.

The IND application for a Phase 1 clinical trial of KB408 was submitted on August 15th and received FDA clearance at the end of the 30-day review period. The company anticipates administering the first patient in the Phase 1 clinical trial in Q1 2024. KB408 also received orphan-drug designation from the FDA on September 5th for AATD treatment.

The Phase 1 clinical trial, known as the Serpentine-1 study, is an open-label, single-dose escalation study involving adult patients with AATD carrying a PI*ZZ genotype. It will assess the safety, tolerability, and efficacy of KB408 across three planned dose levels, with three patients in each cohort. 

KB408 is an inhaled (nebulized) formulation of Krystal Biotech's innovative replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, encoding the human alpha-1 antitrypsin protein for AATD treatment.

Alpha-1 Antitrypsin Deficiency (AATD) is a rare genetic disorder resulting from mutations in the SERPINA1 gene. These mutations reduce AAT protein levels and functionality. In individuals with the PI*ZZ genotype, the most common form of AATD, these mutations lead to the accumulation of misfolded AAT protein in the liver and low AAT protein levels in the blood. This deficiency allows unchecked inflammation and lung damage, eventually causing life-threatening pulmonary impairment and severe respiratory insufficiency. Current disease management includes weekly infusions of plasma-derived AAT augmentation therapy, with the clinical benefits yet to be fully established. There is currently no cure for AATD.

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