Krystal Biotech expects results from three clinical programs between February and the first half of 2027
Key Facts
On November 2, 2026, Krystal Biotech updated timelines for three clinical programs, expecting preliminary results for KB803 after the final patient visit in February 2027, top-line results for KB801 during 2027, and interim results for KB407 in the first half of that year. The update reflects different paths for each program, from replacing withdrawals in the KB803 trial to slower patient recruitment in KB801. KB407’s ongoing study is still testing repeat doses ahead of the expected interim results. The company did not specify a marketing date for these programs in its disclosure. Meanwhile, the company reported commercial revenue of $127.3 million from VYJUVEK in the third quarter and provided updates on other programs awaiting readouts in 2027.
In the registered IOLITE trial of KB803, the company added 3 patients, including a one-year-old child, to replace 2 patients who withdrew for reasons unrelated to safety. It expects the final patient visit in the trial to take place in February 2027, with preliminary results to be announced shortly afterward. The company said the additional enrollment was intended to address the effect of the two withdrawals on the trial’s progress. This timeline places the program’s readout after patient visits are complete, rather than treating results as available as soon as the additional enrollment ends. The disclosure did not specify a subsequent date for taking a regulatory step based on the results.
Enrollment remains open in the EMERALD-1 trial of KB801, and the company has activated 22 sites in the United States. It said recruitment is progressing more slowly than expected, attributing this to narrow eligibility criteria and the difficulty of recruiting patients for a placebo-controlled trial in an area where an approved treatment is available. The trial aims to enroll about 60 patients, and the company expects to complete enrollment and announce top-line results in 2027. The readout timeline is therefore tied to completing recruitment of the target number, as the activated sites continue trying to recruit participants. The company provided no more specific timing within next year in its update.
In the KB407 cystic fibrosis study, 3 patients were receiving doses at the time of the update, and the company expects to enroll 3 additional patients and begin dosing them before the end of 2026. The open-label, single-arm study is testing repeat doses over 24 weeks in patients who cannot use, do not tolerate or do not benefit from modulator therapies. Interim results are expected in the first half of 2027. The company said earlier interim results showed delivery and expression of normal CFTR protein in the lungs of patients treated with KB407. In the third quarter, the company was selected for the U.S. Food and Drug Administration’s Complex Innovative Trial Design meeting program for the program, and it aims to start the pivotal trial in 2027 after aligning with the agency on the design and statistical analysis.
VYJUVEK commercial revenue provides a source of funding for development, with global net product revenue of $127.3 million in the third quarter of 2026, compared with $97.8 million in the same period of 2025. The company said global revenue for the treatment increased 30% year over year, with a gross margin of 94% during the quarter. Cash, cash equivalents and investments totaled $1.2 billion on September 30, 2026. The chief executive said continued growth in VYJUVEK revenue supports profitability and funding for progress across the development pipeline. The board authorized a share repurchase program of up to $400 million, without obligating the company to buy any amount or setting an expiration date for the program.
The updates covered other programs in addition to the three main timelines. The company continues to enroll patients in cohort 2B of the SERPENTINE-1 study of KB408 and expects interim results in 2027. Enrollment of 5 patients in the HALITE-1 study of KB111 is complete, and dosing is ongoing; the company expects interim results before the end of 2026. For the KB707 program, the company presented results from an expansion cohort in the KYANITE-1 study at the American Society of Clinical Oncology’s annual meeting in May 2026; treatment in combination with pembrolizumab recorded an objective response rate of 31% and a disease control rate of 75%. The company expects to enroll about 70 patients in a global cohort evaluating KB707 with chemotherapy and begin dosing with the selected regimen by mid-2027.
On the commercial front, the company said pricing negotiations had been completed in Germany and Italy, and it expects to launch VYJUVEK in Italy in early 2027, while negotiations are ongoing in other European countries and the United Kingdom. In November 2025, the company said treatment coverage approvals in the United States had exceeded 615, following the FDA’s label expansion in September to include patients with dystrophic epidermolysis bullosa from birth. The stock closed at $332.29 on October 30, 2026. In premarket trading on November 2, it fell 3.70% after the third-quarter results were announced, despite earnings and revenue exceeding analysts’ estimates. Adjusted earnings per share were $2.05, compared with a consensus estimate of $1.84, while revenue was $127.34 million, against an estimate of $126.77 million.