Tinlarebant

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Belite Bio to Participate in Three Upcoming Investor Conferences
Globenewswire· 2025-08-27 12:00
Company Overview - Belite Bio, Inc is a clinical-stage drug development company focused on novel therapeutics for degenerative retinal diseases with significant unmet medical needs, including Stargardt disease type 1 and Geographic Atrophy in advanced dry age-related macular degeneration [2] - The lead candidate, Tinlarebant, is an oral therapy aimed at reducing toxin accumulation in the eye, currently undergoing Phase 3 and Phase 2/3 studies [2] Upcoming Events - The executive management team will participate in three investor conferences: - Cantor Global Healthcare Conference on September 5, 2025, at 8:00 am ET - Morgan Stanley 23rd Annual Global Healthcare Conference on September 8, 2025, at 5:35 pm ET - H.C. Wainwright 27th Annual Global Investment Conference on September 9, 2025, at 8:00 am ET [3] - Webcasts of the presentations will be available on the investor relations section of the Belite Bio website, with replays archived for 90 days [1]
Belite Bio(BLTE) - 2025 Q2 - Earnings Call Presentation
2025-08-11 20:30
Financial Performance - Total operating expenses increased to $17596 thousand in Q2 2025 from $10471 thousand in Q2 2024[13] - Research and Development (R&D) expenses increased to $11049 thousand in Q2 2025 from $9078 thousand in Q2 2024[13] - General and Administrative (G&A) expenses increased significantly to $6547 thousand in Q2 2025 from $1393 thousand in Q2 2024[13] - Net loss increased to $16320 thousand in Q2 2025 from $9494 thousand in Q2 2024[13] - The company had $1492 million in cash, liquidity fund, time deposits and U S treasury bills[13] - The company raised approximately $15 million in gross proceeds in a registered direct offering on August 8, 2025[13] Pipeline and Clinical Trials - Belite Bio is conducting a Phase 3, 2-year global trial ("PHOENIX" Study) for Stargardt Disease (STGD1) with completed enrollment of 529 subjects[10] - A Phase 3, 2-year global trial ("DRAGON" Study) is ongoing for Tinlarebant with 104 subjects aged 12-20, with completion expected in Q4 2025[10] - A Phase 2/3, 2-year global trial ("DRAGON II" Study) is ongoing for Tinlarebant with 60 subjects aged 12-20[10] Tinlarebant - Tinlarebant is a novel, once-daily oral tablet designed to reduce retinol delivery to the eye by binding to serum retinol binding protein 4 (RBP4)[11] - Belite Bio believes early intervention targeting emerging retinal pathology is the best approach to slow disease progression in STGD1 & GA[11]
Belite Bio Announces Completion of Enrollment in the Pivotal Global Phase 3 PHOENIX Trial Evaluating Oral Tinlarebant in Geographic Atrophy
Globenewswire· 2025-07-02 07:00
Core Insights - Belite Bio has completed enrollment in the PHOENIX trial, a pivotal Phase 3 study evaluating Tinlarebant for geographic atrophy in dry age-related macular degeneration [1][2][3] Company Overview - Belite Bio is a clinical-stage biopharmaceutical company focused on developing therapeutics for degenerative retinal diseases, including Stargardt disease type 1 and geographic atrophy [5] - The company's lead candidate, Tinlarebant, is an oral therapy aimed at reducing retinal toxin accumulation and is currently in multiple clinical trials [5] Clinical Trial Details - The PHOENIX trial is a 24-month, randomized, double-masked, placebo-controlled, multicenter study conducted across several countries, including the United States, the United Kingdom, France, Czech Republic, Switzerland, China, Taiwan, and Australia [3] - The trial aims to evaluate the efficacy of Tinlarebant in slowing the growth of atrophic lesions, a key marker of disease progression in geographic atrophy [2][3] Market Need - Geographic atrophy is a progressive condition that severely impacts vision and quality of life, with no approved oral treatments currently available [2][4] - The completion of enrollment in the PHOENIX trial reflects a significant unmet medical need for patients suffering from this condition [2][4] Regulatory Designations - Tinlarebant has received several designations, including Rare Pediatric Disease designation, Fast Track Designation, and Breakthrough Therapy Designation in the United States, as well as Orphan Drug Designation in multiple regions [4]
Belite Bio Announces FDA Granting of Breakthrough Therapy Designation for Tinlarebant for the Treatment of Stargardt Disease
GlobeNewswire News Room· 2025-05-21 07:00
Core Points - Belite Bio, Inc. has received Breakthrough Therapy Designation from the FDA for Tinlarebant, aimed at treating Stargardt disease (STGD1), which currently has no approved therapies [1][2] - The designation is based on interim data from the Phase 3 DRAGON trial, indicating substantial improvement over existing therapies and a favorable safety profile [2][6] - The DRAGON trial is a global study involving 104 subjects across 11 jurisdictions, designed to evaluate the safety and efficacy of Tinlarebant [5][6] Company Overview - Belite Bio is focused on developing novel therapeutics for degenerative retinal diseases, including STGD1 and Geographic Atrophy (GA) [8] - Tinlarebant is an orally administered tablet intended for early intervention in maintaining retinal health, with no current FDA-approved treatments for STGD1 or GA [7][8] - The company is also conducting a Phase 2/3 study (DRAGON II) and a Phase 3 study (PHOENIX) for other related conditions [8] Clinical Trial Insights - The Phase 3 DRAGON trial is randomized, double-masked, and placebo-controlled, with a primary efficacy endpoint focused on the growth rate of atrophic lesions [5] - The trial's completion is expected by Q4 2025, including a three-month follow-up period [6] - Tinlarebant has previously received multiple designations, including Fast Track and Rare Pediatric Disease Designations in the U.S. [6]
Belite Bio Reports First Quarter 2025 Financial Results and Provides Corporate Update
Globenewswire· 2025-05-14 01:00
Core Viewpoint - Belite Bio, Inc is advancing its clinical development of Tinlarebant, targeting degenerative retinal diseases, with positive interim results from the Phase 3 DRAGON trial and plans for trial completion by the end of 2025 [2][4]. Clinical Highlights - Tinlarebant is an oral, once-daily RBP4 antagonist that reduces RBP4 levels in the blood, decreasing vitamin A delivery to the eye, which is crucial for normal vision but can lead to toxicity in certain retinal diseases [3]. - The independent Data Safety Monitoring Board (DSMB) recommended the continuation of the DRAGON trial without modifications, with completion expected in Q4 2025 [4]. - The ongoing Phase 3 trial (PHOENIX) for Tinlarebant in geographic atrophy (GA) patients has enrolled 464 out of the targeted 500 subjects [4][14]. Financial Highlights - As of March 31, 2025, the company reported current assets of $157.4 million, including cash and liquidity funds [7]. - Research and development expenses for Q1 2025 were $9.4 million, up from $6.8 million in Q1 2024, primarily due to increased share-based compensation and clinical trial expenses [8]. - General and administrative expenses rose to $6.1 million in Q1 2025 from $1.6 million in Q1 2024, attributed to share-based compensation [9]. - The company reported a net loss of $14.3 million for Q1 2025, compared to a net loss of $7.9 million in the same period of 2024 [11][17]. Corporate Highlights - In February 2025, Belite Bio raised $15 million in gross proceeds through a registered direct offering, with potential additional proceeds from warrant exercises [6]. - The company is focused on maintaining strong execution across its late-stage clinical programs to deliver new treatment options for degenerative retinal diseases [2]. Upcoming Milestones - A conference call and webcast is scheduled for May 14, 2025, to discuss financial results and provide a business update [12]. - The primary efficacy endpoint for the DRAGON trial is the growth rate of atrophic lesions, with safety and tolerability also being assessed [5][14].
Belite Bio, Inc to Present at the dbVIC - Deutsche Bank ADR Virtual Investor Conference May 15th
GlobeNewswire News Room· 2025-05-12 12:35
Company Overview - Belite Bio, Inc is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs, including Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD) [4] - The lead candidate, Tinlarebant, is an oral therapy aimed at reducing toxin accumulation in the eye and is currently undergoing multiple clinical trials, including a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) for STGD1, as well as a Phase 3 study (PHOENIX) for GA [4][7] Recent Developments - Dr. Nathan L. Mata, the Chief Scientific Officer, will present at the dbVIC - Deutsche Bank American Depositary Receipt (ADR) Virtual Investor Conference on May 15, 2025, providing an opportunity for investors to engage with the company [1] - The DRAGON trial, a pivotal global Phase 3 trial for Tinlarebant in adolescent STGD1 subjects, is expected to be completed by Q4 2025, with an independent DSMB recommending submission of interim analysis data for regulatory review [7] - The ongoing PHOENIX trial for Tinlarebant in GA subjects has enrolled over 460 participants [7] Event Details - The virtual investor conference will take place on May 15, 2025, at 12:30 pm ET, allowing real-time interaction between investors and the company [2] - Participation in the conference is free of charge, and an archived webcast will be available for those unable to attend live [3][2]
Belite Bio to Participate in Four Upcoming Investor Conferences
Globenewswire· 2025-05-12 12:00
Core Insights - Belite Bio, Inc is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs [2] - The company is actively participating in four upcoming investor conferences to present its developments and engage with investors [1][3] Company Overview - Belite Bio targets diseases such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) associated with advanced dry age-related macular degeneration (AMD) [2] - The lead candidate, Tinlarebant, is an oral therapy aimed at reducing toxin accumulation in the eye and is currently undergoing multiple clinical trials, including a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) for STGD1, as well as a Phase 3 study (PHOENIX) for GA [2] Upcoming Events - The company will present at the Deutsche Bank Depositary Receipts Virtual Investor Conference on May 15, 2025, at 12:30 pm ET [3] - A fireside chat is scheduled for May 20, 2025, at the H.C. Wainwright 3rd Annual BioConnect Investor Conference at NASDAQ [3] - Another fireside chat will take place on May 27, 2025, at the Stifel 2025 Ophthalmology Forum [3] - The Benchmark's Healthcare House Call One-on-One Conference will feature a fireside chat on May 29, 2025, at 12:45 pm ET [3]
Belite Bio to Host Webcast on May 14, 2025, to Discuss First Quarter 2025 Financial Results
Globenewswire· 2025-05-07 12:00
Core Insights - Belite Bio, Inc is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for degenerative retinal diseases with significant unmet medical needs [3] Group 1: Company Overview - Belite Bio is advancing treatments for conditions such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD) [3] - The company's lead candidate, Tinlarebant, is an oral therapy aimed at reducing toxin accumulation in the eye and is currently undergoing multiple clinical trials, including a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) for STGD1, as well as a Phase 3 study (PHOENIX) for GA [3] Group 2: Upcoming Events - Belite Bio will host a webcast on May 14, 2025, at 4:30 p.m. Eastern Time to discuss its financial results and provide a business update for the first quarter ended March 31, 2025 [1][2] - The webcast can be accessed via a provided link and will be available for replay for approximately 90 days after the event [2]
Belite Bio Announces Poster Presentations at the Association for Research in Vision and Ophthalmology 2025 Annual Meeting
Globenewswire· 2025-04-29 12:00
Core Insights - Belite Bio, Inc. is focused on developing novel therapeutics for degenerative retinal diseases, specifically targeting Stargardt disease and geographic atrophy, which have significant unmet medical needs [1][5] Group 1: Presentations at ARVO Annual Meeting - The company will present two posters at the ARVO Annual Meeting from May 4-8, 2025, in Salt Lake City, Utah [1] - The first presentation will cover baseline characteristics of adolescent subjects in the Phase 3 DRAGON trial of Tinlarebant [2] - The second presentation will discuss pharmacokinetic and pharmacodynamic properties of Tinlarebant in adolescent Japanese patients [2] Group 2: Tinlarebant Overview - Tinlarebant is an oral therapy designed to reduce the accumulation of vitamin A-based toxins (bisretinoids) that lead to retinal diseases [3] - The drug works by modulating serum retinol binding protein 4 (RBP4) levels, thereby reducing bisretinoid formation [3] - Tinlarebant has received multiple designations, including Fast Track and Orphan Drug Designation in the U.S., and Sakigake Designation in Japan for treating STGD1 [3] Group 3: Stargardt Disease (STGD1) - STGD1 is the most common inherited macular dystrophy, leading to central vision loss due to mutations in the ABCA4 gene [4] - There are currently no FDA-approved treatments for STGD1, highlighting the need for effective therapies [4] - The disease progression is monitored through the accumulation of bisretinoids, which also affects patients with geographic atrophy [4] Group 4: Company Background - Belite Bio is a clinical-stage biopharmaceutical company dedicated to developing therapies for degenerative retinal diseases and specific metabolic diseases [5] - The lead candidate, Tinlarebant, is being evaluated in multiple Phase 3 studies, including DRAGON and PHOENIX, targeting both STGD1 and geographic atrophy [5]
Belite Bio(BLTE) - 2024 Q4 - Earnings Call Transcript
2025-03-17 22:40
Financial Data and Key Metrics Changes - In 2024, the company reported R&D expenses of $29.9 million, an increase from $28.8 million in 2023, primarily due to higher royalty payments and increased share-based compensation [32] - General and administrative (G&A) expenses rose to $10.1 million in 2024 from $6.8 million in 2023, driven by increased share-based compensation [33] - The net loss for 2024 was $36.1 million, compared to $31.6 million in 2023 [33] - Cash and investments at the end of 2024 totaled $145.2 million, up from $88.2 million at the end of 2023 [33] Business Line Data and Key Metrics Changes - The company is advancing its lead product, Tinlarebant, in Phase 3 trials for Stargardt disease and geographic atrophy, with significant progress reported in both trials [4][10] - The Phase 3 DRAGON trial for Stargardt disease has maintained a sample size of 104 subjects after an interim analysis recommended proceeding without modifications [8][20] - The PHOENIX trial for geographic atrophy has enrolled over 400 subjects, with plans to increase enrollment from 450 to 500 subjects [10][30] Market Data and Key Metrics Changes - Tinlarebant has received multiple designations, including Rare Pediatric Disease and Fast Track in the U.S., and Pioneer Drug designation in Japan, indicating a significant unmet medical need [6] - The company is uniquely positioned with ongoing global Phase 3 trials, which may lead to the first oral treatment for degenerative retinal diseases [11] Company Strategy and Development Direction - The company aims to position Tinlarebant as a first-in-class oral therapy for Stargardt disease and geographic atrophy, focusing on early intervention to slow disease progression [4][5] - The strategy includes expanding patient enrollment in clinical trials to enhance the chances of regulatory approval and market success [40] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the regulatory review process following the DSMB's recommendation for drug approval based on interim results [38] - The company plans to expedite a second Phase 3 trial for geographic atrophy if positive signals are observed in the ongoing PHOENIX trial [44] Other Important Information - The company raised $15 million in gross proceeds through a registered direct offering in February 2025, ensuring a full year's cash runway [34] - The dropout rate in the PHOENIX trial is approximately 20%, which is considered manageable given the elderly population involved [52] Q&A Session Summary Question: Potential outcomes from regulatory interactions regarding Stargardt disease - Management believes that regulatory agencies will likely align with the DSMB's positive recommendation for drug approval, but if not, they will continue with the study [38] Question: Reason for increasing sample size in the GA trial - The decision to increase the sample size to 500 patients was driven by smooth progress in the GA study and the desire to enhance the chances of success [40] Question: Update on discontinuation rates in the GA trial - The dropout rate for the PHOENIX trial is approximately 20%, which is lower than historical rates for similar studies [52] Question: Approval expectations for Stargardt disease patients - Management indicated that if efficacy is demonstrated in adolescents, it should facilitate approval for adults as well [56] Question: Current status of regulatory submissions - The company is working with multiple CROs for regulatory submissions, and the process is currently underway [78] Question: Expected treatment effect in the DRAGON study - The DRAGON study is powered for a 40% treatment effect with 80% power to detect that effect at the second year [84]