KB407 / Krystal Biotech - LARVOL DELTA

CORAL-1: A Study Assessing KB407 for the Treatment of Cystic Fibrosis (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Krystal Biotech, Inc. | Trial completion date: Jul 2025 ➔ Jan 2026 | Trial primary completion date: Jun 2025 ➔ Dec 2025

Trial completion date • Trial primary completion date • Cystic Fibrosis • Genetic Disorders • Immunology • 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

CORAL-1: A Study Assessing KB407 for the Treatment of Cystic Fibrosis (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Krystal Biotech, Inc. | Trial completion date: Jul 2024 ➔ Jul 2025 | Trial primary completion date: Jul 2024 ➔ Jun 2025

Trial completion date • Trial primary completion date • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Krystal Biotech Announces Initial Clinical Update for Rare Respiratory Disease Programs… (GlobeNewswire) - P1 | N=12 | NCT05504837 | Sponsor: Krystal Biotech, Inc. | "KB407 for the treatment of cystic fibrosis (CF): As of the December 6, 2024 data cut-off, a total of five (5) patients had been enrolled in CORAL-1. Three patients received a single 109 PFU KB407 dose in Cohort 1 and three patients, including one roll-over from Cohort 1, received two daily 109 PFU KB407 doses in Cohort 2. All but one patient was on background modulator therapy....The initial focus of Cohorts 1 and 2 was safety of single and repeat inhaled administration of KB407 in patients with CF. As observed with KB408, KB407 has been well tolerated in all patients dosed to date....The Company expects to report data from Cohort 3 in 1H 2025..."

P1 data • Cystic Fibrosis

Krystal Biotech Announces Third Quarter 2024 Financial Results and Provides Business Updates (GlobeNewswire) - "KB407 for the treatment of cystic fibrosis (CF); The Company recently activated two additional clinical sites for its KB407 Phase 1 CORAL-1 study and expects to report interim molecular data from the study in 1H 2025."

P1 data • Cystic Fibrosis

Krystal Biotech Announces Second Quarter 2024 Financial Results and Provides Business Updates (Krystal Biotech Press Release) - "KB407 for the treatment of cystic fibrosis (CF)...the Company cleared Cohort 2 of the Phase 1 CORAL-1 study. CORAL-1 is a multi-center, dose escalation study evaluating KB407 in patients with CF, regardless of their underlying genotype. The Company expects to initiate the third and final cohort in 2H 2024."

Trial status • Cystic Fibrosis

Krystal Biotech Receives European Commission Orphan Designation for KB407 for the Treatment of Cystic Fibrosis (GlobeNewswire) - “Krystal Biotech, Inc...announced today that the European Commission has granted orphan designation for KB407 for the treatment of Cystic Fibrosis (CF). KB407 is an investigational, redosable gene therapy designed to correct the underlying cause of cystic fibrosis by delivering two copies of the CFTR gene directly to the airway epithelial cells when delivered via a nebulizer.”

Orphan drug • Cystic Fibrosis • Genetic Disorders

Evaluation of KB407, An HSV-1-Based Gene Therapy Vector for the Treatment of Cystic Fibrosis, in Healthy and Patient-Derived Airway Cells Including an Apical-out Diseased Airway Organoid Model (ATS 2024) - "KB407 is able to transduce and deliver full-length CFTR to clinically relevant airway epithelial cell populations in vitro, including patient-derived apical-out organoids, supporting future clinical development of KB407 as an inhaled therapeutic for patients with CF."

Clinical • Gene therapy • Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Herpes Simplex • Immunology • Pulmonary Disease • Respiratory Diseases • CFTR

Krystal Biotech Announces Fourth Quarter and Full Year 2022 Financial Results and Operational Progress (GlobeNewswire) - "The Company is working closely with the Therapeutics Development Network of the Cystic Fibrosis Foundation to validate its clinical protocol and plans to initiate a Phase 1 dose ranging clinical (CORAL-1) trial in the US in the first half of 2023 to assess safety and preliminary efficacy....The Company is presently screening patients for enrollment in a Phase 1 clinical trial in Australia to study safety and determine frequency of dosing application."

Trial status • Cystic Fibrosis • Genetic Disorders

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

Krystal Biotech Announces Third Quarter 2023 Financial Results and Provides Business Update (Krystal Biotech Press Release) - "Cohort 1 of the Phase 1 (CORAL-1) study has been enrolled and completed....We are working to initiate Cohort 2 of the CORAL-1 study following safety review by the Data Monitoring Committee. The CORAL-1 study is a multi-center, dose-escalation trial of KB407 in patients with CF, regardless of their underlying genotype. The Company anticipates announcing data from the Phase 1 study in 2024."

P1 data • Trial status • Cystic Fibrosis

A Study of Inhaled KB407 for the Treatment of Cystic Fibrosis (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: Krystal Biotech, Inc. | N=13 ➔ 0 | Recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

A Study Assessing KB407 for the Treatment of Cystic Fibrosis (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Krystal Biotech, Inc. | N=20 ➔ 12 | Trial completion date: Mar 2024 ➔ Jul 2024 | Trial primary completion date: Mar 2024 ➔ Jul 2024

Enrollment change • Trial completion date • Trial primary completion date • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Krystal Biotech Announces First Patient Dosed in Phase 1 Clinical Trial of KB407 for the Treatment of Cystic Fibrosis (GlobeNewswire) - "Krystal Biotech, Inc...announced today that the first patient has been dosed at the Cystic Fibrosis Institute of Chicago in the Company’s Phase 1 CORAL-1/US study evaluating KB407, an engineered HSV-1-based, aerosol-delivered, mutation agnostic, genetic medicine for the treatment of patients with cystic fibrosis (CF)...'We look forward to continued enrollment into the Phase 1 study with anticipated data in 2024.'"

P1 data • Trial status • Cystic Fibrosis • Genetic Disorders

A Study Assessing KB407 for the Treatment of Cystic Fibrosis (clinicaltrials.gov) - P1 | N=20 | Recruiting | Sponsor: Krystal Biotech, Inc. | Not yet recruiting ➔ Recruiting | Initiation date: Nov 2022 ➔ Jun 2023

Enrollment open • Trial initiation date • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Respiratory cell-type affinity and absolute CFTR expression in the primate airway upon nebulization of KB407 (NACFC 2022) - "Taken together, these data indicate that KB407 capablytransduces multiple cell types that contribute to native CFTR function inprimate airways and expresses the transgene at physiologically relevantlevels, providing further support for the potential of KB407 as an inhalablegene therapy for the treatment of CF."

Cystic Fibrosis • Fibrosis • Genetic Disorders • Herpes Simplex • Immunology • Pulmonary Disease • Respiratory Diseases • CFTR • MUC5AC

Krystal Biotech Announces Third Quarter 2022 Financial Results and Operational Highlights (GlobeNewswire) - "The Company continues to screen patients for enrollment in the Phase 1 clinical study of inhaled KB407 (CORAL-1/AU study) and plans to initiate dosing in the fourth quarter of 2022."

Trial status • Cystic Fibrosis • Genetic Disorders

A Study Assessing KB407 for the Treatment of Cystic Fibrosis (clinicaltrials.gov) - P1 | N=20 | Not yet recruiting | Sponsor: Krystal Biotech, Inc.

New P1 trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Krystal Biotech Announces Second Quarter 2022 Financial Results and Reports Updates on Operational Progress (Krystal Biotech Press Release) - "KB407 for the treatment of Cystic Fibrosis ('CF'): The Company has begun screening patients for enrollment in the Phase 1 clinical study of inhaled KB407 (CORAL-1/AU study)..."

Enrollment status • Cystic Fibrosis • Genetic Disorders

Krystal Biotech Receives FDA Acceptance of KB407 IND Application for Cystic Fibrosis Clinical Trial (GlobeNewswire) - "Krystal Biotech... today announced that the United States Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application to evaluate KB407 in a clinical trial for cystic fibrosis (CF)....The Company anticipates initiating the clinical trial in 2H 2022. The Phase 1 clinical trial will utilize nebulized administration to deliver KB407 in up to 20 adults with CF....The primary endpoint of the trial will be the safety and tolerability of nebulized KB407."

IND • New P1 trial • Cystic Fibrosis • Genetic Disorders

A Study of Inhaled KB407 for the Treatment of Cystic Fibrosis (clinicaltrials.gov) - P1 | N=13 | Recruiting | Sponsor: Krystal Biotech, Inc. | Not yet recruiting ➔ Recruiting | Trial completion date: May 2022 ➔ Oct 2024 | Initiation date: Oct 2021 ➔ Mar 2022 | Trial primary completion date: Mar 2022 ➔ Jul 2024

Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Krystal Biotech Announces First Quarter 2022 Financial Results and Reports Updates on Operational Progress (GlobeNewswire) - "Krystal Biotech, Inc...today reported financial results and key operational progress updates for the first quarter ending March 31, 2022....Phase 1 clinical trial of inhaled KB407 in patients with CF in Australia is expected to start in 2Q 2022....Anticipate filing an investigational new drug ('IND') application and initiating a Phase 1 trial clinical program in the U.S. in 2H 2022."

IND • New P1 trial • Trial status • Cystic Fibrosis • Genetic Disorders

Krystal Biotech Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Update on Operational Progress (GlobeNewswire) - "Phase 1 trial of KB407 in cystic fibrosis patients in Australia expected to begin in 1H 2022; IND filing and...Company plans to expand the Phase 1 trial clinical program to the U.S. in 2H 2022."

IND • Trial status • Cystic Fibrosis • Genetic Disorders

A Study of Inhaled KB407 for the Treatment of Cystic Fibrosis (clinicaltrials.gov) - P1; N=13; Not yet recruiting; Sponsor: Krystal Biotech, Inc.

New P1 trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

[VIRTUAL] Nonclinical pharmacology of nebulized KB407 for the treatment of cystic fibrosis (NACFC-I 2021) - "These data support repeated application of KB407 as a broadly applicable gene therapy for the treatment of CF."

Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Ophthalmology • Pulmonary Disease • Respiratory Diseases