Krystal Biotech (KRYS) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Technology and product overview

• Utilizes a modified viral vector for gene delivery, with the lead product approved for dystrophic epidermolysis bullosa (DEB) and launched in the U.S. about 12-13 months ago.

• European launch expected in the first half of next year, and Japan launch anticipated late next year or early 2026.

• Pipeline includes four clinical-stage programs targeting different tissues and formulations.

Launch experience and patient adoption

• Over 98% of DEB patients receive treatment at home, highlighting the importance of convenience.

• Patient and physician experiences have been positive, with strong drug performance and ongoing launch success.

• As of July, there were 400 reimbursement approvals, with logistical steps required before dosing.

• Achieving 60% market share in two years is a key target, with ongoing efforts to identify and educate patients and physicians.

• Barriers include lack of genetic information and the need for education about gene therapy, but reluctance to start therapy is low.

Patient identification and outreach

• Focus remains on the 1,200 identified U.S. patients, with broader efforts to find new patients planned for next year.

• Strategies will include family tree analysis, genetic testing encouragement, and claims data review.

• Incremental outreach is ongoing, but major expansion will follow after reaching current market share goals.