Krystal Biotech Receives FDA Acceptance of KB407 IND Application for Cystic Fibrosis Clinical Trial


News and research before you hear about it on CNBC and others. Claim your one week free trial for StreetInsider Premium here.

  • KB407 is an investigational, redistributable gene therapy candidate designed to treat all patients with cystic fibrosis, regardless of the underlying genetic mutation

PITTSBURGH, Aug. 01, 2022 (GLOBE NEWSWIRE) — Crystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in redistributable gene therapy, today announced that the United States Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for evaluating KB407 in a clinical trial for cystic fibrosis (CF).

KB407 is a modified HSV-1 vector carrying two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to respiratory cells in the lungs. By inducing normal full-length CFTR protein expression in the lungs, treatment with KB407 has the potential to restore the flow of ions and water into and out of lung cells to correct pulmonary manifestations of the disease in patients, regardless of their underlying genetic mutation.

Cystic fibrosis is a genetic disease that affects people of all racial and ethnic groups by causing mucus to build up in the lungs leading to persistent lung infections and progressive lung disease. Sources including the Cystic Fibrosis Foundation say there are nearly 40,000 children and adults living with cystic fibrosis in the United States and approximately 105,000 people diagnosed with cystic fibrosis in 94 countries.

“We are excited to advance KB407, our investigational gene therapy for patients with cystic fibrosis, into the clinic. It is designed to address the root cause of disease, regardless of a patient’s mutation by instructing the body to produce its own functional protein,” said Hubert Chen, MD, senior vice president of clinical development at Krystal Biotech. Dr. Chen continued, “Furthermore, this IND acceptance represents an important milestone for us as it allows us to demonstrate the power of our platform to deliver genes, beyond skin cells, to respiratory cells. .

On July 1, 2022, the Company submitted an IND application to initiate the Phase 1 clinical trial of KB407. At the end of the 30-day review period, the Company received notification that the FDA accepted the IND allowing the start of the Phase 1 clinical trial. The Company plans to launch the clinical trial in 2H 2022.

Phase 1 Trial OverviewThe Phase 1 clinical trial will use nebulized delivery to administer KB407 to up to 20 adults with cystic fibrosis. The study will recruit 3 cohorts sequentially to evaluate increasing doses of KB407. The primary endpoint of the trial will be the safety and tolerability of nebulized KB407. Changes in lung function from baseline will be assessed by forced expiratory volume in one second.

About KB407KB407 is an investigational, redistributable gene therapy designed to correct the underlying cause of cystic fibrosis by delivering two copies of the CFTR gene directly to airway epithelial cells when delivered via a nebulizer. By inducing normal full-length CFTR protein expression in the lungs, treatment with KB407 has the potential to restore the flow of ions and water into and out of lung cells to correct pulmonary manifestations of the disease in patients, regardless of their underlying genetic mutation.

About Cystic FibrosisCystic fibrosis is caused by genetic mutations that result in a dysfunctional or absent CFTR protein and is the most common fatal inherited disease in the United States. Lack of functional CFTR in the secretory epithelial cells of the airways causes accumulation of dehydrated mucus in the lungs, pancreas and other organs. This buildup of mucus in the lungs leads to loss of lung function and eventually respiratory failure. According to the US Cystic Fibrosis Foundation, the median age at death for patients with cystic fibrosis in the United States was 34.1 years in 2020.

Although CFTR modulators are effective in patients with certain CFTR mutations, patients may still experience pulmonary symptoms that require treatment. Importantly, approximately 10-15% of CF patients have genetic mutations that are not expected to respond to current therapies and currently have no disease-modifying treatment options available, representing an unmet need. satisfied important.

About Krystal Biotech, Inc.Krystal Biotech, Inc. (NASDAQ:KRYS) is a pivotal-stage gene therapy company leveraging its proprietary redosable gene therapy platform and in-house manufacturing capabilities to develop life-changing drugs for patients with of serious diseases, including rare diseases of the skin, lung and other regions. For more information, please visit http://www.krystalbio.comand follow @KrystalBiotech on LinkedIn and Twitter.

Forward-looking statements All statements in this press release regarding Krystal Biotech, Inc.’s future expectations, plans and prospects, including statements regarding the clinical utility of KB407 and the anticipated timing of the KB407 Phase 1 clinical trial program in the United States, and other statements containing the words “anticipate”, “believe”, “estimate”, “expect”, “intend”, “may”, “plan”, “predict “, “project”, “target”, “potential”, “likely”, “will”, “could”, “should”, “continue” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation. Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements due to various important factors, including: the availability or commercial potential of product candidates, including KB407, the sufficiency of cash resources and the need for additional funding and t or other important factor as set forth under “Risk Factors” in the Company’s monthly and quarterly reports filed with the United States Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this release. The Company expects subsequent events and developments to cause its views to evolve. However, while the Company may choose to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be taken to represent the views of the Company as of any date subsequent to the date of this release.

CONTACTS:Investors and media:Meg DodgeKrystal Biotech[email protected]Source: Krystal Biotech, Inc.

main logo

Source: Krystal Biotech, Inc.


Comments are closed.