Krystal Biotech Announces FDA Clearance of Investigational New Drug Application for KB408 for the Treatment of Type 1 Alpha-1 Antitrypsin Deficiency
• Orphan Drug Designation Granted to KB408
PITTSBURGH, Sept. 21, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development, and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug Application (IND) for KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD).
KB408 is a modified, replication-defective, non-integrating HSV-1-derived vector carrying two full-length copies of the serpin family A member 1 (SERPINA1) gene to enable expression of alpha-1 antitrypsin (AAT). KB408 is formulated for inhaled delivery to the respiratory cells of the lungs via nebulization.
“We are excited to advance KB408, our investigational gene therapy for patients with alpha-1 antitrypsin deficiency, into the clinic in our Serpentine-1 study,” said Hubert Chen, M.D., Senior Vice President of Clinical Development at Krystal Biotech. Dr. Chen continued, “This IND acceptance represents an important milestone for us as we work to address a serious lung disease with limited treatment options, and also allows us to demonstrate the potential of our platform to deliver genes repeatedly to epithelial cells of the lung.”
On August 15th, the Company submitted an IND application to request FDA authorization to initiate a Phase 1 clinical trial of KB408. At the end of the 30-day review period, the Company received notification from the FDA that the IND has been cleared. The Company anticipates dosing the first patient in a Phase 1 clinical trial in Q1 2024. On September 5th, the FDA granted orphan-drug designation for KB408 for the treatment of AATD.
The Phase 1 clinical trial is a Phase 1, open-label, single dose escalation study in adult patients with AATD with a PI*ZZ genotype. Three planned dose levels of KB408 will be evaluated with three patients in each cohort to evaluate the safety, tolerability, and efficacy of KB408. Details about the Phase 1 study can be found at www.clinicaltrials.gov under NCT identifier: NCT06049082.
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