Krystal Biotech’s ‘Promising’ Cystic Fibrosis Drug Scores Orphan Designation

Gene therapy company Krystal Biotech (KRYS) has announced that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to KB407, currently in pre-clinical development for the treatment of cystic fibrosis (“CF”).

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Shares in Krystal rose 5% in Monday’s after-hours trading following the announcement.

KB407 is an inhaled, repeat-dose gene therapy product currently in the preclinical phase with plans to file an investigational new drug (IND) application in 2021.

“We are pleased to receive Orphan Drug Designation for KB407 to treat cystic fibrosis as this is an important step forward in our efforts to address the continued unmet need in this devastating disease,” said Suma Krishnan, founder and COO of Krystal.

She added: “We are excited by the results of the in vitro data thus far, as presented at ASGCT earlier this year, and we look forward to sharing in vivo animal data later this year.”

The FDA’s Office of Orphan Drug Products grants Orphan Drug Designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the US.

Orphan Drug Designation may allow Krystal Biotech to be eligible for a seven-year period of US marketing exclusivity upon approval of KB407, tax credits for certain clinical research costs and a waiver of the Prescription Drug User Fee Act (PDUFA) filing fees.

Shares in Krystal are currently trading down 19% year-to-date, but the Street has a bullish Strong Buy consensus on the stock’s outlook. That’s with a $68 average analyst price target, indicating upside potential of over 50%.

William Blair analyst Raju Prasad has a buy rating on Krystal Biotech, noting that Krystal continues to execute on its stated goals. He believes that Krystal remains undervalued at current levels given several catalysts in data presentations from multiple clinical programs anticipated in the next 12 months.

Meanwhile HC Wainwright analyst Joseph Pantginis noted that “promising” preclinical results on KB407 were presented at the American Society of Gene & Cell Therapy in May 2020.

“The data demonstrate in vitro, a profile of transduction efficiency and functionality of the target gene consistent with its predecessors KB103 and KB105 and provides the scientific rationale for progressing the development of this asset in CF” he concluded. (See KRYS stock analysis on TipRanks).