Belite Bio(BLTE) - 2023 Q4 - Annual Report

Introduction & Corporate Update The CEO highlighted 2023 milestones, including Phase 3 trial initiations and positive Phase 2 data, with further updates planned for 2024 CEO's Statement & 2023 Highlights The CEO highlighted 2023 as a productive year, marked by the initiation of the Phase 3 GA trial, completion of enrollment for the Phase 3 Stargardt disease trial, and positive 24-month data from the Phase 2 Stargardt trial - The CEO emphasized key 2023 milestones: initiating the Phase 3 GA trial, completing enrollment for the Phase 3 Stargardt disease trial, and obtaining positive 24-month data from the Phase 2 Stargardt trial[26] - The company plans to share additional analysis from its Phase 2 Stargardt trial at the ARVO annual meeting in May and expects one-year interim data from the Phase 3 DRAGON trial later in 2024[26] Clinical Development Update Updates on Tinlarebant's clinical progress include ongoing Phase 3 trials for Stargardt Disease and Geographic Atrophy, building on positive Phase 2 data Tinlarebant (LBS-008) Overview Tinlarebant is an oral, once-daily RBP4 antagonist designed to slow disease progression in Stargardt Disease (STGD1) and Geographic Atrophy (GA) - Tinlarebant is an orally administered tablet intended to slow disease progression in patients with Stargardt Disease (STGD1) and Geographic Atrophy (GA)[11] - Its mechanism involves acting as a retinol binding protein 4 (RBP4) antagonist to decrease the delivery of vitamin A (retinol) to the eye, reducing the accumulation of toxic byproducts[35] - Tinlarebant has received Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug Designations (ODD) from the FDA for STGD1[3] Stargardt Disease (STGD1) Program The pivotal global Phase 3 "DRAGON" trial has completed enrollment with 104 adolescent subjects, with interim data expected in Q4 2024 - The pivotal global Phase 3 "DRAGON" trial has completed enrollment with 104 subjects across 11 countries, with interim data expected in Q4 2024[2][25][27] - Data from a 24-month Phase 2 trial showed a sustained lower atrophic lesion growth in Tinlarebant-treated subjects compared to a similar control group (p<0.001)[25][20] - In the Phase 2 trial, 42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesions during the 24-month treatment period[34][36] Geographic Atrophy (GA) Program The company has initiated the pivotal global Phase 3 "PHOENIX" trial for Tinlarebant in GA subjects - The pivotal global Phase 3 "PHOENIX" trial in GA subjects has been initiated, with the first patient dosed in Q3 2023[11][4] - The trial is a 24-month, randomized (2:1 active:placebo), double-masked, placebo-controlled study targeting approximately 430 subjects[13] - As of the report date, the PHOENIX trial has enrolled 56 subjects and received approval to initiate in eight countries[28][11] Financial Results The company reported a strong cash position at year-end 2023, despite an increased net loss driven by higher R&D expenses for clinical trials Financial Highlights Belite Bio ended 2023 with a strong cash position of $88.2 million, more than double the $42.1 million at year-end 2022, bolstered by financing activities - Cash position increased to $88.2 million as of December 31, 2023, compared to $42.1 million on December 31, 2022[37][22] - The company raised $22 million from the exercise of warrants and $29 million from its ATM Offering as of December 31, 2023[29][5] Net Loss Summary | Period | Net Loss | Net Loss per Share | | :--- | :--- | :--- | | Full Year 2023 | $31.6 million | ($1.19) | | Full Year 2022 | $12.6 million | ($0.63) | | Q4 2023 | $7.0 million | ($0.25) | | Q4 2022 | $6.8 million | ($0.27) | Expense Analysis For the full year 2023, R&D expenses surged to $24.8 million from $8.9 million in 2022, primarily due to costs associated with the DRAGON and PHOENIX Phase 3 trials - Full-year 2023 R&D expenses were $24.8 million, a significant increase from $8.9 million in 2022, primarily due to expenses for the DRAGON and PHOENIX trials[29] - Full-year 2023 G&A expenses were $6.8 million, up from $4.0 million in 2022, mainly due to increased share-based compensation and professional service fees[21] - For Q4 2023, R&D expenses were $4.9 million (vs. $5.2 million in Q4 2022), and G&A expenses were $2.1 million (vs. $1.5 million in Q4 2022)[29][21] Consolidated Financial Statements The consolidated financial statements show a significant increase in total assets and shareholders' equity, alongside higher operating expenses and net loss for 2023 Consolidated Statements of Operations and Comprehensive Loss The company's total operating expenses for FY 2023 were $31.67 million, a sharp increase from $12.82 million in FY 2022, leading to a net loss of $31.63 million Full Year Operations (Audited, in thousands of USD) | | Year Ended Dec 31, 2023 | Year Ended Dec 31, 2022 | | :--- | :--- | :--- | | Research and development | 24,844 | 8,869 | | General and administrative | 6,824 | 3,952 | | Total operating expenses | 31,668 | 12,821 | | Loss from operations | (31,668) | (12,821) | | Net loss | (31,632) | (12,648) | Fourth Quarter Operations (Unaudited, in thousands of USD) | | Three Months Ended Dec 31, 2023 | Three Months Ended Dec 31, 2022 | | :--- | :--- | :--- | | Research and development | 4,862 | 5,226 | | General and administrative | 2,093 | 1,495 | | Total operating expenses | 6,955 | 6,721 | | Net loss | (6,991) | (6,783) | Consolidated Balance Sheets As of December 31, 2023, Belite Bio's total assets grew to $94.6 million from $44.3 million in the prior year, driven by a significant increase in cash to $88.2 million Assets (in thousands of USD) | | Dec 31, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | Cash | 88,157 | 42,089 | | Total current assets | 89,940 | 42,807 | | TOTAL ASSETS | 94,642 | 44,273 | Liabilities and Shareholders' Equity (in thousands of USD) | | Dec 31, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | Total current liabilities | 3,633 | 2,104 | | TOTAL LIABILITIES | 4,211 | 2,772 | | Total shareholders' equity | 90,431 | 41,501 | | TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | 94,642 | 44,273 | Other Information The company announced a webcast to discuss results and provided important cautions regarding forward-looking statements and associated risks Webcast and Contact Information The company announced it would host a webcast on March 12, 2024, to discuss financial results and provide a business update - A webcast was scheduled for March 12, 2024, at 4:30 p.m. Eastern Time to discuss financial results and provide a business update[16][30] - Contact information for Media and Investor Relations is provided in the report[24] Forward-Looking Statements This press release includes forward-looking statements concerning clinical data, development timelines, regulatory milestones, and the potential efficacy of Tinlarebant - The report contains forward-looking statements regarding the company's expectations for clinical development, regulatory milestones, and commercialization of its product candidates[17] - Key risks that could cause actual results to differ include the ability to demonstrate safety and efficacy, clinical results not supporting further development, and the timing of regulatory decisions[17]