Aug 13, 2026 earnings call — Tinlarebant NDA acceptance for Stargardt disease
FDA accepted tinlarebant NDA with priority review and set PDUFA date of 02/12/2027.
EPS $-0.70 (est $-0.73)
Key quotes
"We are very pleased to announce that the FDA has accepted our new drug application for tinlarebant with priority review. And establishing a PDUFA date of 02/12/2027."
"At ASRS, we presented new secondary endpoint data demonstrating subjects treated with tinlarebant showed a halt to a slightly decreased QAF values, decreased by approximately 2%."
"And we are extremely well funded into the future with a cash runway to commercialize tinlarebant following a potential regulatory approval and to continue to advance our pipelines."
Analyst Q&A 6
Dragon 2 study and pediatric plans
Q (Judah Frommer · Morgan Stanley): Analyst asked if Dragon 2 could help the U.S. filing or regulatory process and if there are plans to study tinlarebant in children under 12.
A: Management said Dragon 2 is essentially a Japan study for the PMDA and they do not believe it will contribute to the U.S. NDA. They are starting a pediatric (PIP) study in London for ages 3 to 11 to inform regulation for patients younger than 12.
"As for the Dragon 2, I think at this stage, it is still pretty much, a Japan study for the PMDA." — Yu-Hsin Lin (CEO·CHAIR)
Royalty payment, Europe filing, patient numbers
Q (Marc Goodman · Leerink): Analyst asked how much the one-time royalty payment was, when they will file in Europe, and whether they have claims database patient numbers for the U.S.
A: Management said the payment is related to Phase 3 completion but Columbia asked them to keep the amount confidential. They will disclose patient numbers at a virtual commercial day in September. They are focused on the FDA PDUFA date of February 12 and expect the European filing to come after U.S. approval, aligning messages with other regulators.
"So right now, we are focused on, the FDA with the PDUFA date in February 12." — Yu-Hsin Lin (CEO·CHAIR)
Manufacturing site and AdCom likelihood
Q (Tazeen Ahmad · BofA): Analyst asked where manufacturing is done and whether the site completed an FDA inspection, and asked about the chance and timing of an FDA Advisory Committee (AdCom).
A: Management said they have both U.S. and ex-U.S. CDMOs but will not name them; they are large, well-known firms. They do not believe an AdCom is planned now but said the FDA could decide that later and they will update when they have more details.
"So right now, we do not believe there is a AdCom being planned, but that does not mean that, you know, further down the line, the FDA would want to use an AdCom." — Yu-Hsin Lin (CEO·CHAIR)
Priority review voucher and GA interim timing
Q (Steven Seedhouse · Cantor): Analyst asked if they expect a priority review voucher and whether they would auction it, and asked timing for the geographic atrophy (GA) trial interim readout.
A: Management expects to receive a priority review voucher if approved because of the rare pediatric disease designation, but they have not decided whether to sell or use it. For the GA interim, they said the busiest FDA interaction will be December and January 2027 and expect the GA interim analysis to be in Q1 next year, probably after February, with FDA approval the top priority.
"Well, yeah, we do expect that if we receive approval, we should get the priority review voucher just because we do have, the rare pediatric disease designation." — Hao-Yuan Chuang (CFO)
Label age range and data across ages
Q (Yi Chen · H. C. Wainwright): Analyst asked whether the FDA had said the label will include patients over age 12.
A: Management said there has been no label discussion yet during review, though they believe the data support a broader label. Management added that lesion growth is similar across age groups and, given the shared ABCA4 cause, they see no reason the label would not include older patients, but they will not comment on label specifics while the NDA is under review.
"Given that the underlying cause of the disease namely ABCA4 dysfunction, is exactly the same, I would see no reason why the label would not include patients older than 20." — Hendrik Scholl
Patient dosing compliance and Japan timing
Q (Nathan L. Mata · H. C. Wainwright): Analyst asked what percent of patients were compliant after 24 months and when the Japan submission would occur.
A: Management said dosing compliance in the Stargardt study was in excess of 90%. They said the Japan submission will be concurrent with the U.S. process and, given the Sakigake Designation, will probably be around 3 months after FDA approval.
"So given the Sakigake Designation, it will probably be around 3 months after FDA approval." — Yu-Hsin Lin (CEO·CHAIR)