The US Food and Drug Administration (FDA) has granted clearance to Krystal Biotech’s investigational new drug application (IND) for KB408 to treat alpha-1 antitrypsin deficiency (AATD), a rare genetic disease.
The company submitted the application in August this year seeking approval for the initiation of Phase I clinical trial of KB408.
The regulator, after the 30-day review of the application, notified the company that its IND has been cleared.
Krystal Biotech clinical development senior vice president Hubert Chen said: “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.
“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.”
Krystal Biotech plans dosing the first participant in the trial in the first quarter of next year.
The single dose escalation, open-label study intends to assess the pharmacodynamics and safety of KB408 in AATD adults with a PI*ZZ genotype.
In the study, the efficacy, safety, and tolerability of three dosages of KB408 will be assessed in each cohort of three patients.
KB408 is an inhaled formulation of the company’s replication-defective, non-integrating herpes simplex virus type 1 (HSV-1) derived vector, which delivers functional full-length human serpin family A member 1 (SERPINA1) gene to the airways of the AATD patients.
Via nebulisation, the formulation can be delivered to the respiratory cells of patients.
This month, the FDA also granted orphan-drug designation to KB408 to treat AATD.