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Krystal Biotech, Inc. KRYS announced initial clinical data updates on its two inhaled genetic medicine pipeline candidates, KB407 and KB408, which are being developed in early-stage studies for treating rare respiratory diseases.
Shares of the company were down 7.3% on Dec. 12 following the announcement of the news.
KB407 is being developed in a phase I study for the treatment of cystic fibrosis (CF), while KB408 is being developed in a phase I study for treating alpha-1 antitrypsin deficiency (AATD).
The latest clinical updates include molecular data as well as safety and tolerability data for both KB407 and KB408.
Year to date, shares of Krystal Biotech have rallied 39.8% against the industry’s decline of 8.7%.
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More on KRYS' Development of KB407
The open-label, phase I CORAL-1 study is evaluating KB407 for treating adult patients with CF. The company recently activated two additional clinical sites for its KB407 phase I CORAL-1 study.
Per the company, the CORAL-1 study is designed to include up to three dose escalation cohorts evaluating either one, two, or four daily administrations of 109 PFU of KB407 via inhalation.
Based on the preclinical data submitted to date by Krystal Biotech, the Cystic Fibrosis Foundation (CFF) Therapeutic Development Network (TDN) Clinical Research Executive Committee has granted conditional sanctioning of the KB407 phase I CORAL-1 study protocol. It is subject to review by the data monitoring committee charter, if required, to match CFF TDN standards.
As of the data cut-off date of Dec 6, 2024, a total of five patients from Cohort 1 and Cohort 2 have been enrolled in the CORAL-1 study. Treatment with KB407 was well tolerated in all patients and no serious adverse events were observed.
KRYS plans to report data from Cohort 3 of the CORAL-1 study in the first half of 2025.
More on KRYS' Development of KB408
The open-label, phase I SERPENTINE-1 study is investigating KB408 for treating adult patients with AATD. In September, Krystal Biotech amended the protocol for its phase I SERPENTINE-1 study on KB408 to include mandatory bronchoscopies in this cohort to measure alpha-1 antitrypsin expression.
Per the company, the SERPENTINE-1 study is designed to include up to three dose escalation cohorts evaluating single administrations of 109, 1010 and 1011 PFU of KB408 via inhalation.
As of the data cut-off date of Dec 6, 2024, a total of seven patients from Cohort 1 and Cohort 2 have been enrolled in the SERPENTINE-1 study.
Clear evidence of SERPINA1 gene delivery and AAT expression following treatment with KB408 was observed in AATD patients and no serious adverse events were reported.