KB408 / Krystal Biotech - LARVOL DELTA

Serpentine-1: A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: Krystal Biotech, Inc. | Trial completion date: May 2026 ➔ Dec 2026 | Trial primary completion date: Jun 2025 ➔ Dec 2025

Trial completion date • Trial primary completion date • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Pulmonary Disease • Respiratory Diseases

Krystal Biotech Announces First Quarter 2025 Financial and Operating Results (GlobeNewswire) - "$88.2 million in 1Q VYJUVEK revenue and $429.4 million since launch in Q3 2023...VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)...The Company continues to expect a decision by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) on its Japan New Drug Application (JNDA) in 2H 2025...The Company continues to enroll Cohort 3 of CORAL-1, the Company’s multi-center, dose escalation study evaluating KB407 in patients with CF...The Company received full sanctioning of the study protocol by the Cystic Fibrosis Foundation Therapeutic Development Network in January and remains on track for an interim molecular data readout for Cohort 3 patients in mid-2025...The Company continues to enroll in Cohort 2 and is working to enroll in Cohort 3 of SERPENTINE-1....The Company expects to report molecular results for the additional Cohort 2 and 3 patients later this year."

Enrollment status • Japan approval • P1 data • Sales • Alpha-1 Antitrypsin Deficiency • Cystic Fibrosis • Dermatology • Genetic Disorders

Murine Toxicology Study of Repeat-Dose Inhaled KB408, an HSV-1-Based Vector for the Treatment of Alpha-1 Antitrypsin Deficiency (ATS 2024) - "KB408 showed no adverse effects and limited biodistribution when delivered via inhalation to mice, supporting the initiation of a first-in-human Phase 1 single ascending dose study."

Preclinical • Alpha-1 Antitrypsin Deficiency • Gene Therapies • Genetic Disorders • Hepatology • Herpes Simplex • Ophthalmology • Pulmonary Disease • Respiratory Diseases • ELANE • SERPINA1

Krystal Biotech to Present at the American Thoracic Society 2024 International Conference (GlobeNewswire) - "Krystal Biotech...announced today that the Company will be presenting new preclinical data on respiratory genetic medicine candidates KB407 and KB408 at the American Thoracic Society (ATS) 2024 International Conference being held from May 17-22, 2024 in San Diego, California....Both KB407 and KB408 are being evaluated in ongoing Phase 1 studies....as well as an overview of KB408 IND-enabling studies conducted to support initiation of the ongoing Phase 1 SERPENTINE-1 study in patients with AATD."

Clinical data • Preclinical • Trial status • Alpha-1 Antitrypsin Deficiency • Cystic Fibrosis

Nonclinical pharmacology of KB408, an HSV‐1‐based vector designed for the treatment of alpha‐1 antitrypsin deficiency, in the SERPINA1 knockout mouse model (ESGCT 2023) - "KB408 was also found to be well tolerated in the airways of both healthy and SERPINA1 knock-out immunocompetent mice. Taken together, these observations support the development of KB408 as a novel gene therapy for the treatment of AATD-related lung disease."

Preclinical • Alpha-1 Antitrypsin Deficiency • Gene Therapies • Genetic Disorders • Hepatology • Herpes Simplex • Pulmonary Disease • Respiratory Diseases • ELANE • SERPINA1

Serpentine-1: A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Krystal Biotech, Inc. | Trial completion date: Jun 2025 ➔ May 2026

Trial completion date • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases

Serpentine-1: A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Krystal Biotech, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases

Serpentine-1: A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Krystal Biotech, Inc.

New P1 trial • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases • ELANE

Krystal Biotech Announces FDA Clearance of Investigational New Drug Application for KB408 for the Treatment of Type 1 Alpha-1 Antitrypsin Deficiency (GlobeNewswire) - "Krystal Biotech, Inc...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)....The Company anticipates dosing the first patient in a Phase 1 clinical trial in Q1 2024."

IND • New P1 trial • Alpha-1 Antitrypsin Deficiency • Genetic Disorders

Krystal Biotech Announces Orphan Drug Designation Granted to KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency (GlobeNewswire) - "Krystal Biotech, Inc...announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD)."

Orphan drug • Alpha-1 Antitrypsin Deficiency • Genetic Disorders