BELITE BIO, INC Q4 FY2024 earnings call

• Tinlarebant is a first-in-class oral therapy for Stargardt disease and geographic atrophy. Phase 3 DRAGON trial for Stargardt had interim analysis from DSMB recommending proceeding without sample size change and to submit data for regulatory review. DRAGON 2 trial for Stargardt has enrolled 11 of 60 subjects. - GA Phase 3 PHOENIX trial has enrolled over 400 subjects and is expected to increase enrollment to 500. - Safety profile of Tinlarebant in DRAGON 1 trial was good with low withdrawal rates, stabilized visual acuity, and transient ocular adverse events. - Financials: 2024 R&D, G&A, and net loss increased compared to 2023, but cash runway remained strong.

Belite Bio does not have traditional product segments. Instead, its performance is centered around the clinical trial progress of Tinlarebant. In 2024, R&D expenses were $29.9 million (up from $28.8 million in 2023) due to increased royalty payments and share-based compensation. G&A expenses were $10.1 million (up from $6.8 million in 2023) primarily from increased share-based compensation. Net loss was $36.1 million (up from $31.6 million in 2023). Cash position at year-end 2024 was $31.7 million in cash and $113.5 million in investments.

• Phase 3 DRAGON trial completion for Stargardt is on track for end of 2024. - GA Phase 3 PHOENIX trial enrollment expected to close by Q2 2024 with increased sample size to 500. - Plan to submit data for regulatory review as recommended by DSMB for Tinlarebant's approval.

• Uncertainty in regulatory agencies' alignment with DSMB's recommendation for drug approval. - Potential challenges in payer reimbursement if the label for Tinlarebant is broader than the currently enrolled patient groups.

Q: Regarding DSMB's recent recommendation for Stargardt trial, what are the potential outcomes from interactions with regulatory authorities?

A: Tom Lin stated they believe it's a positive outcome and will follow DSMB's recommendation to request regulatory review, noting regulatory agencies may align with DSMB's recommendation but if not, they'll carry on with the study.

Q: What drove the decision to increase the sample size to 500 for GA's PHOENIX trial?

A: Tom Lin said the GA study is progressing smoothly, and enrolling more subjects boosts success chances while remaining feasible within the timeline.

Q: About Stargardt disease, will the future prescription label be restricted to enrolled patients?

A: Hendrik Scholl stated the label would not be restricted, as there's no reason not to prescribe to adult patients with Stargardt disease.

Q: Have any warrants attached to the $15 million capital raise been exercised?

A: Tom Lin responded that no warrants have been exercised yet.

Q: What kind of difference in lesion growth between placebo and treatment is the DRAGON study powered for?

A: Nathan Mata said the DRAGON study is powered for a 40% treatment effect with 80% power to detect that effect at the second year.