Direktnyheter • May 19
Belite Bio Advances Tinlarebant With Swiss Orphan Status and U S FDA Filing Progress Swissmedic granted orphan drug status to Belite Bio’s lead candidate tinlarebant for treating Stargardt disease in Switzerland.
Tinlarebant is reported as the first therapeutic candidate to show clinical efficacy in Stargardt disease by significantly reducing retinal lesion growth in the Phase 3 DRAGON trial.
Belite Bio has started a rolling New Drug Application submission with the U.S. FDA, with completion targeted by the second quarter of 2026.
The combination of orphan status in Switzerland and reported Phase 3 efficacy in Stargardt disease highlights regulatory and clinical milestones around tinlarebant that could shape expectations for the company’s lead program.
Investors may want to focus on the timing, scope and outcomes of the rolling U.S. NDA process, since regulatory feedback and any additional data disclosures can influence both perceived approval risk and the potential addressable market in a rare disease setting. Tillkännagivande • May 14
Belite Bio, Inc to Report Q1, 2026 Results on May 20, 2026 Belite Bio, Inc announced that they will report Q1, 2026 results on May 20, 2026 Tillkännagivande • Apr 24
Belite Bio Initiates Rolling Submission of New Drug Application to the U.S. Food and Drug Administration for Tinlarebant for the Treatment of Stargardt Disease Belite Bio Inc. initiated a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for tinlarebant, an investigational novel oral therapy for the treatment of Stargardt disease type 1 (STGD1), a rare, inherited retinal disorder caused by mutations in the ABCA4 gene. Tinlarebant has previously been granted Breakthrough Therapy Designation (BTD) by the FDA for STGD1, and the FDA has previously granted Belite approval for the rolling submission of the NDA. The Company expects to complete the NDA rolling submission in the second quarter of 2026. Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in geographic atrophy (GA), or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease Designation in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1. The Company has completed a Phase 3 trial (DRAGON) in adolescent STGD1 subjects, and the drug is currently being evaluated in a Phase 2/3 trial (DRAGON II) in adolescent STGD1 subjects and a Phase 3 trial (PHOENIX) in subjects with GA. Breakeven Date Change • Mar 05
Forecast breakeven date pushed back to 2028 The 7 analysts covering Belite Bio previously expected the company to break even in 2027. New consensus forecast suggests losses will reduce by 7.5% per year to 2027. The company is expected to make a profit of US$164.2m in 2028. Average annual earnings growth of 61% is required to achieve expected profit on schedule. Price Target Changed • Mar 03
Price target increased by 10% to US$212 Up from US$192, the current price target is an average from 8 analysts. New target price is 26% above last closing price of US$168. Stock is up 181% over the past year. The company is forecast to post a net loss per share of US$2.77 next year compared to a net loss per share of US$2.31 last year. Breakeven Date Change • Mar 02
Forecast breakeven date moved forward to 2027 The 7 analysts covering Belite Bio previously expected the company to break even in 2028. New consensus forecast suggests losses will reduce by 39% to 2026. The company is expected to make a profit of US$46.9m in 2027. Average annual earnings growth of 58% is required to achieve expected profit on schedule. Tillkännagivande • Jan 28
Belite Bio, Inc Completes Enrollment in the DRAGON II Clinical Trial of Tinlarebant for Stargardt Disease (STGD1) Belite Bio Inc. announced the completion of enrollment of 60 subjects, including 15 Japanese subjects, in the Phase 2/3 DRAGON II clinical trial evaluating tinlarebant for the treatment of Stargardt disease type 1 (STGD1). DRAGON II clinical trial is a global, 24-month, randomized, double-masked, placebo-controlled study designed to evaluate the efficacy, safety, and tolerability of tinlarebant in adolescent patients with STGD1. The trial had a targeted enrollment of 60 adolescent subjects aged 12 to 20 years old across Japan, the United States, and the United Kingdom, with participants randomized 1:1 to receive either tinlarebant or placebo. Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in geographic atrophy (GA), or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease Designation in the U.S., Orphan Drug Designation in the U. S., Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1. Breakeven Date Change • Dec 31
Forecast to breakeven in 2028 The 6 analysts covering Belite Bio expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$118.6m in 2028. Average annual earnings growth of 59% is required to achieve expected profit on schedule. New Risk • Dec 12
New minor risk - Shareholder dilution The company's shareholders have been diluted in the past year. Increase in shares outstanding: 21% This is considered a minor risk. Shareholder dilution occurs when there is an increase in the number of shares on issue that is not proportionally distributed between all shareholders. Often due to the company raising equity capital or some options being converted into stock. All else being equal, if there are more shares outstanding then each existing share will be entitled to a lower proportion of the company's total earnings, thus reducing earnings per share (EPS). While dilution might not always result in lower EPS (like if the company is using the capital to fund an EPS accretive acquisition) in a lot cases it does, along with lower dividends per share and less voting power at shareholder meetings. Currently, the following risks have been identified for the company: Major Risk Revenue is less than US$1m. Minor Risk Shareholders have been diluted in the past year (21% increase in shares outstanding). Breakeven Date Change • Nov 21
Forecast to breakeven in 2027 The 6 analysts covering Belite Bio expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$9.18m in 2027. Average annual earnings growth of 76% is required to achieve expected profit on schedule. Tillkännagivande • Nov 10
Belite Bio, Inc has completed a Follow-on Equity Offering in the amount of $15.00005 million. Belite Bio, Inc has completed a Follow-on Equity Offering in the amount of $15.00005 million.
Security Name: American Depository Shares
Security Type: Depositary Receipt (Common Stock)
Securities Offered: 230,770
Price\Range: $65
Discount Per Security: $4.225
Security Name: Warrants
Security Type: Equity Warrant
Securities Offered: 230,770
Transaction Features: Registered Direct Offering Price Target Changed • Nov 06
Price target increased by 13% to US$106 Up from US$93.75, the current price target is an average from 4 analysts. New target price is approximately in line with last closing price of US$112. Stock is up 36% over the past year. The company is forecast to post a net loss per share of US$1.92 next year compared to a net loss per share of US$1.18 last year. Tillkännagivande • Nov 04
Belite Bio Inc. Announces UK’s Medicines and Healthcare Products Regulatory Agency Agrees to Conditional Marketing Authorization Application Based on Interim Analysis Results for Treatment of Stargardt Disease with Tinlarebant Belite Bio Inc. announced that United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) has agreed to accept a Conditional Marketing Authorization application for Tinlarebant for the treatment of Stargardt disease based on the interim analysis results from the Phase 3 DRAGON trial. MHRA’s response is based on the interim analysis results which fulfil the criteria for a Conditional Marketing Authorization application. The Company remains on track to report final topline data from the Phase 3 DRAGON trial in the fourth quarter of 2025. These results are expected to be submitted to the MHRA for full Marketing Authorization Application. The pivotal Phase 3 DRAGON trial is a randomized, double-masked, placebo-controlled, global study designed to evaluate the safety and efficacy of Tinlarebant in adolescent patients with Stargardt disease. The trial enrolled 104 subjects across 11 jurisdictions, including the U.S., United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia, with a 2:1 randomization (Tinlarebant:placebo). The primary efficacy endpoint is the growth rate of atrophic lesions, alongside the assessment of safety and tolerability. Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in geographic atrophy (GA), or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Breakthrough Therapy Designation, Fast Track Designation and Rare Pediatric Disease designation in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1. Tillkännagivande • Oct 16
Belite Bio, Inc. Announces China NMPA Agrees to New Drug Application with Priority Review Based on Interim Analysis Results for the Treatment of Stargardt Disease with Tinlarebant Belite Bio Inc. announced that the Center for Drug Evaluation of China's National Medical Products Administration ("NMPA") has agreed to accept the New Drug Application (NDA) with priority review for Tinlarebant for the treatment of Stargardt disease based on the interim analysis results from the Phase 3 DRAGON trial. NMPA's response is based on the interim analysis results showing statistical significance in the primary endpoint of the Phase 3 DRAGON trial. The Company remains on track to report final topline data from the Phase 3 DRAGON trial in the fourth quarter of 2025. These results are expected to be submitted to the NMPA as part of the NDA that is currently under preparation in accordance with China CDE's guidance. The pivotal Phase 3 DRAGON trial is a randomized, double-masked, placebo-controlled, global study designed to evaluate the safety and efficacy of Tinlarebant in adolescent patients with Stargardt disease. The trial enrolled 104 subjects across 11 jurisdictions, including the U.S., United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia, with a 2:1 randomization (Tinlarebant:placebo). The primary efficacy endpoint is the growth rate of atrophic lesions, alongside the assessment of safety and tolerability. Belite's lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of bisretinoid toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. Tillkännagivande • Sep 13
Belite Bio Inc. Announces Completion of DRAGON, a 2-Year, Phase 3 Trial of Oral Tinlarebant in the Treatment of Stargardt Disease Belite Bio Inc. announced the completion of the last subject visit in the Phase 3 DRAGON clinical trial evaluating Tinlarebant for the treatment of Stargardt disease type 1 (STGD1). The DRAGON trial enrolled 104 adolescent subjects across 11 jurisdictions, including the United States, United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia, with a 2:1 randomization (Tinlarebant:placebo). A total of 94 subjects completed the study, with the last study visit conducted on September 11, 2025. The primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability of Tinlarebant will also be assessed. Belite Bio expects to report top-line results from the DRAGON trial in Fourth Quarter 2025 and plans to file New Drug Applications in 1H 2026. About Tinlarebant (a/k/a LBS-008) Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in geographic atrophy (GA), or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. Tillkännagivande • Sep 08
Belite Bio, Inc announced that it expects to receive $124.999936 million in funding from RA Capital Management, L.P., Eventide Asset Management, LLC, Marshall Wace LLP, RTW Investments, LP, Soleus Capital LLC, Vestal Point Capital, LP Belite Bio, Inc announced a private placement and entered into a purchase agreement to issue 1,953,124 Ordinary shares, par value $0.0001 per share, at an issue price of $64 per ordinary share and warrants to purchase 1,953,124 ordinary shares for aggregate gross proceeds of $124,999,936 on September 8, 2025. Each Warrant will be immediately exercisable, expire two years from the date of issuance and have an exercise price of $76.80 per ordinary share. The securities issued under the Securities Purchase Agreements have not been registered under the Securities Act of 1933, as amended in reliance on the exemption from registration provided by Section 4(a)(2) of the Securities Act and/or Rule 506 of Regulation D promulgated thereunder, or under any state securities laws. The transaction is expected to close on September 9, 2025. The transaction will include participation from RA Capital Management as the lead investor and from Eventide Asset Management, Marshall Wace, RTW Investments, Soleus Capital and Vestal Point Capital. In connection with the offering, the Company shall pay the reasonable fees and expenses of counsel for the lead investor to the transactions contemplated hereunder, in an amount not to exceed $150,000. Tillkännagivande • Aug 08
Belite Bio, Inc has filed a Follow-on Equity Offering in the amount of $15.00005 million. Belite Bio, Inc has filed a Follow-on Equity Offering in the amount of $15.00005 million.
Security Name: American Depository Shares
Security Type: Depositary Receipt (Common Stock)
Securities Offered: 230,770
Price\Range: $65
Security Name: Warrants
Security Type: Equity Warrant
Securities Offered: 230,770
Transaction Features: Registered Direct Offering Buy Or Sell Opportunity • Jul 30
Now 20% overvalued after recent price rise Over the last 90 days, the stock has risen 4.7% to US$64.81. The fair value is estimated to be US$53.84, however this is not to be taken as a sell recommendation but rather should be used as a guide only. Price Target Changed • Jul 08
Price target increased by 10% to US$93.75 Up from US$85.00, the current price target is an average from 4 analysts. New target price is 56% above last closing price of US$60.12. Stock is up 22% over the past year. The company is forecast to post a net loss per share of US$1.72 next year compared to a net loss per share of US$1.18 last year. Tillkännagivande • Jul 02
Belite Bio, Inc. Announces Completion of Enrollment in the Pivotal Global Phase 3 Phase 3 PHOENIX Trial Evaluating Oral Tinlarebant in Geographic Atrophy Belite Bio Inc. announced the completion of enrollment in the PHOENIX trial, a global, 24-month Phase 3 pivotal trial evaluating the safety and tolerability of Tinlarebant and its potential to reduce atrophic lesion growth rate in patients diagnosed with geographic atrophy (GA) in dry age-related macular degeneration (AMD). The PHOENIX study is a 24-month, randomized, double-masked, placebo-controlled, multicenter, pivotal Phase 3 trial. The study is ongoing across sites in the United States, the United Kingdom, France, Czech Republic, Switzerland, China, Taiwan, and Australia. Tillkännagivande • May 21
Belite Bio, Inc. Announces FDA Granting of Breakthrough Therapy Designation for Tinlarebant for the Treatment of Stargardt Disease Belite Bio Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Tinlarebant for the treatment of Stargardt disease (STGD1) based on the previously reported interim data from the ongoing Phase 3 DRAGON trial. There are currently no approved treatments for STGD1. The FDA grants Breakthrough Therapy Designation to expedite the development and regulatory review of drugs that are intended for serious or life-threatening conditions. The designation is based on preliminary clinical evidence indicating that a drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Tinlarebant is an orally administered, once-a-day tablet intended as an early intervention for maintaining the health and integrity of retinal tissues in Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) patients. Currently, there are no FDA approved treatments for STGD1 and no approved orally administered treatments for GA. Therefore, if approved, Tinlarebant would be a novel oral therapeutic addressing an unmet medical need in both STGD1 and GA. Belite Bio's advancement of, and anticipated future activities on preclinical studies, clinical development, regulatory milestones, and commercialization of its product candidates; and any other statements containing the words "expect", "hope", "indicate", "look forward to", and similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to Belite Bio's ability to demonstrate the safety and efficacy o its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; the timing of potential submission with FDA; the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio's drug candidates; the potential efficacy of Tinlarebant, as well as those risks more fully discussed in the "Risk Factors" section in Belite Bio's filings with the U.S. Securities and Exchange Commission. Price Target Changed • Mar 26
Price target increased by 7.3% to US$85.00 Up from US$79.25, the current price target is an average from 4 analysts. New target price is 30% above last closing price of US$65.21. Stock is up 63% over the past year. The company is forecast to post a net loss per share of US$1.47 next year compared to a net loss per share of US$1.18 last year. Breakeven Date Change • Mar 21
No longer forecast to breakeven The 4 analysts covering Belite Bio no longer expect the company to break even during the foreseeable future. The company was expected to make a profit of US$23.0m in 2027. New consensus forecast suggests the company will make a loss of US$7.90m in 2027. Tillkännagivande • Mar 20
Belite Bio, Inc, Annual General Meeting, Apr 15, 2025 Belite Bio, Inc, Annual General Meeting, Apr 15, 2025. Location: 12750 high bluff drive suite 475, california 92130, san diego, United States Breakeven Date Change • Mar 18
No longer forecast to breakeven The 4 analysts covering Belite Bio no longer expect the company to break even during the foreseeable future. The company was expected to make a profit of US$18.1m in 2027. New consensus forecast suggests the company will make a loss of US$609.3k in 2027. New Risk • Mar 06
New minor risk - Shareholder dilution The company's shareholders have been diluted in the past year. Increase in shares outstanding: 15% This is considered a minor risk. Shareholder dilution occurs when there is an increase in the number of shares on issue that is not proportionally distributed between all shareholders. Often due to the company raising equity capital or some options being converted into stock. All else being equal, if there are more shares outstanding then each existing share will be entitled to a lower proportion of the company's total earnings, thus reducing earnings per share (EPS). While dilution might not always result in lower EPS (like if the company is using the capital to fund an EPS accretive acquisition) in a lot cases it does, along with lower dividends per share and less voting power at shareholder meetings. Currently, the following risks have been identified for the company: Major Risk Revenue is less than US$1m. Minor Risk Shareholders have been diluted in the past year (15% increase in shares outstanding). Tillkännagivande • Mar 01
Belite Bio, Inc. Announces Interim Analysis Results from the Pivotal Global Phase 3 DRAGON Trial of Tinlarebant in Adolescent Stargardt Disease Subjects Belite Bio Inc. announced that following a pre-specified Interim Analysis of the pivotal global Phase 3 "DRAGON" trial data of Tinlarebant in adolescent Stargardt disease patients, the Data Safety Monitoring Board (DSMB) has recommended the trial proceed without any modifications. The Interim Analysis was performed when all subjects completed the one-year assessment. The study design for the DRAGON trial included an adaptive sample size re-estimation that would determine the need for an increase in sample size in order to enhance power, based on a treatment effect observed at the Interim Analysis. The recommendation by the DSMB that the trial should proceed without modifications indicates that a sample size increase is not warranted. In addition, the DSMB recommended to submit the data for further regulatory review for drug approval. According to the DSMB, Tinlarebant is well-tolerated and the safety profile remains consistent with previously observed data and the mechanism of action for Tinlarebant. New Risk • Feb 27
New minor risk - Profitability The company is currently unprofitable and not forecast to become profitable over the next 3 years. Trailing 12-month net loss: US$33m Forecast net loss in 3 years: US$1.7m This is considered a minor risk. Companies that are not profitable are more likely to be burning through cash and less likely to be well established. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. Without profits, the company is under pressure to grow significantly while potentially having to reduce costs and possibly needing to take on debt or raise capital to remain afloat. Currently, the following risks have been identified for the company: Major Risk Revenue is less than US$1m. Minor Risk Currently unprofitable and not forecast to become profitable over next 3 years (US$1.7m net loss in 3 years). Tillkännagivande • Feb 07
Belite Bio, Inc has filed a Follow-on Equity Offering. Belite Bio, Inc has filed a Follow-on Equity Offering.
Security Name: American Depositary Shares
Security Type: Depositary Receipt (Common Stock)
Securities Offered: 258,309
Price\Range: $58.07
Discount Per Security: $3.77455
Security Name: Warrants
Security Type: Equity Warrant
Securities Offered: 258,309
Transaction Features: Registered Direct Offering Breakeven Date Change • Dec 31
Forecast to breakeven in 2027 The 5 analysts covering Belite Bio expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$18.1m in 2027. Average annual earnings growth of 58% is required to achieve expected profit on schedule. New Risk • Dec 06
New minor risk - Share price stability The company's share price has been volatile over the past 3 months. It is more volatile than 75% of American stocks, typically moving 10% a week. This is considered a minor risk. Share price volatility indicates the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. It also increases the risk of potential losses in the short term as the stock tends to have larger drops in price more frequently than other stocks. Currently, the following risks have been identified for the company: Major Risk Revenue is less than US$1m. Minor Risks Share price has been volatile over the past 3 months (10% average weekly change). Shareholders have been diluted in the past year (12% increase in shares outstanding). Price Target Changed • Nov 13
Price target increased by 18% to US$66.75 Up from US$56.50, the current price target is an average from 4 analysts. New target price is 19% below last closing price of US$82.23. Stock is up 122% over the past year. The company is forecast to post a net loss per share of US$1.18 next year compared to a net loss per share of US$1.19 last year. Recent Insider Transactions Derivative • Oct 06
Key Executive notifies of intention to sell stock Yu-Hsin Lin intends to sell 40k shares in the next 90 days after lodging an Intent To Sell Form on the 3rd of October. If the sale is conducted around the recent share price of US$48.00, it would amount to US$1.9m. Since December 2023, Yu-Hsin's direct individual holding has decreased from 533.93k shares to 531.73k. There have been no trades via on-market transactions or options from company insiders in the last 12 months. Recent Insider Transactions Derivative • Sep 06
Director notifies of intention to sell stock Wan-Shan Chen intends to sell 7k shares in the next 90 days after lodging an Intent To Sell Form on the 4th of September. If the sale is conducted around the recent share price of US$48.10, it would amount to US$320k. As of today, Wan-Shan currently holds no shares directly (This sale likely refers to shares that have not yet been received). There have been no trades via on-market transactions or options from company insiders in the last 12 months. Tillkännagivande • Sep 02
Belite Bio Announces Appointment of Hendrik P. N. Scholl as Chief Medical Officer Belite Bio Inc. announced that its board of directors has appointed Hendrik P. N. Scholl, MD, MA, as the Chief Medical Officer of the Company, effective immediately. Dr. Scholl is the foremost globally recognized authority on Stargardt disease and age-related macular degeneration (AMD), bringing decades of expertise in treating retinal diseases, including the two key indications targeted by Belite Bio's lead drug candidate, Tinlarebant. Dr. Scholl served as the founding and scientific co-director of the Institute of Molecular and Clinical Ophthalmology Basel (IOB) and Professor of Ophthalmology at the University of Basel, where he also led the Department of Ophthalmology as its Chairman. He currently serves as President of the European Vision Institute as well as Chairman of the largest clinical research network in ophthalmology in Europe, EVICR.net, and its Expert Committee on Retinal Dystrophies. He is also the Founder and President of the Swiss Association for Research in Vision and Ophthalmology (ARVO-SWISS). Dr. Scholl’s distinguished career in academia includes leadership positions at several key academic institutions. Recently, he served as Professor of Ophthalmology and Endowed Chair at the Wilmer Eye Institute of Johns Hopkins University Medical School. At the Johns Hopkins Hospital, he was the Head of the Retinal Degeneration Clinic and the Director of the Visual Neurophysiology Service. For the Wilmer Eye Institute, he also served as the Co-director of the Johns Hopkins Center for Stem Cells and Ophthalmic Regenerative Medicine. Dr. Scholl is the coordinating principal investigator of the largest natural history study of Stargardt disease (ProgStar Study), which enrolled 365 subjects. Throughout his career, he has participated in over 10 clinical studies both in Stargardt disease and AMD, authored over 280 articles and reviews in peer-reviewed journals, and received numerous prestigious awards, including the European Vision Award, the President’s Award of the American Society of Retinal Specialists, the W. Richard Green Award and the Paul Henkind Memorial Award of the Macula Society, the Swiss Alfred-Vogt Award, and the Kupfer award of ARVO. He holds an honorary doctorate from Semmelweis University in Budapest, Hungary, and is Adjunct Professor at the Medical University of Vienna, Austria. Over the course of his 25 years of experience, Dr. Scholl has led and participated in numerous boards and advisory committees. He currently serves on the Scientific Advisory Board of Pro Retina Deutschland, Foundation Fighting Blindness, Erasmus University Medical Center, AIBILI, and the Institut de la Vision (Paris); on the Investment Advisory Board of Droia NV; and the Data Safety Monitoring Board of Roche Holding AG and ViGeneron GmbH. Dr. Scholl graduated from the University of Tübingen, Germany with a Doctor of Medicine (Dr. med.) and Master of Arts. He completed his specialist training at the University Eye Hospital in Tübingen before being awarded a fellowship from the German Research Foundation (DFG) at Moorfields Eye Hospital and Institute of Ophthalmology in London, and a DFG Heisenberg Professorship for Macular Diseases at the Eye Clinic of the University of Bonn, Germany. Recent Insider Transactions Derivative • Aug 16
Key Executive notifies of intention to sell stock Yu-Hsin Lin intends to sell 100k shares in the next 90 days after lodging an Intent To Sell Form on the 14th of August. If the sale is conducted around the recent share price of US$49.32, it would amount to US$4.9m. Since December 2023, Yu-Hsin's direct individual holding has decreased from 533.93k shares to 531.73k. There have been no trades via on-market transactions or options from company insiders in the last 12 months. Recent Insider Transactions Derivative • Jun 02
CFO & Director notifies of intention to sell stock Hao-Yuan Chuang intends to sell 50k shares in the next 90 days after lodging an Intent To Sell Form on the 30th of May. If the sale is conducted around the recent share price of US$46.69, it would amount to US$2.3m. Since March 2024, Hao-Yuan has owned 312.21k shares directly. There have been no trades via on-market transactions or options from company insiders in the last 12 months. Tillkännagivande • May 08
Belite Bio, Inc. Presents Additional Analysis from Phase 2 Study of Tinlarebant in Stargardt Disease at the ARVO Annual Meeting Belite Bio Inc. announced additional findings from the 24-month Phase 2 study of Tinlarebant in adolescent Stargardt disease (STGD1) at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting. Tinlarebant is Belite Bio's orally administered tablet intended to slow disease progression in patients affected with STGD1 and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD). Genetic profiling was performed on the 13 adolescent STGD1 patients enrolled in a Phase 2 study of Tinlarebant. Eleven of these subjects (85%) harbored severe pathogenic/likely pathogenic ABCA4 variants. Despite these severe variants, 42% of Tinlarebant-treated subjects (5 out of 12) did not develop incident atrophic (definitely decreased autofluorescence, DDAF) retinal lesions and no change in questionably decreased autofluorescence (QDAF) was observed during the 24-month treatment period. Incident atrophic lesions appeared in seven subjects at different timepoints over 24 months; four of these subjects developed DDAF lesions after month 12. The mean DDAF lesion growth at month 24 was 0.51 mm2 with a range of variation of 0.4 mm2, a growth rate that is significantly lower than what has been observed in natural history studies. Importantly, six STGD1 patients who had a mean bilateral best corrected visual acuity (BCVA) loss of 10 letters per year prior to enrollment in the Phase 2 study showed a mean BCVA loss equivalent to 1.9 letters per year during the 24-month treatment period. Analysis of genotype-phenotype relationships revealed that sibling subjects with identical ABCA4 mutations had different rates of lesion growth and BCVA loss indicating that identical genotypes do not necessarily predict an identical course of disease. Notably, retinal imaging data from the Phase 2 study was reanalyzed using a novel lesion size quantification method that utilizes a mathematical classification of lesions to reduce subjective reader bias and provide enhanced accuracy and superior precision compared to the traditional method of DDAF lesion quantification. This analysis revealed DDAF lesions within the macula in 12 eyes of eight subjects at baseline. Analysis of change in atrophic lesion area within the macula of these eyes over 24 months showed a halt in lesion growth into the macula after 16 months. This finding is consistent with the observed stabilization of visual acuity. Tillkännagivande • Apr 26
Belite Bio, Inc has filed a Follow-on Equity Offering in the amount of $24.999964 million. Belite Bio, Inc has filed a Follow-on Equity Offering in the amount of $24.999964 million.
Security Name: American Depositary Shares
Security Type: Depositary Receipt (Common Stock)
Securities Offered: 651,380
Price\Range: $38.38
Transaction Features: Registered Direct Offering Recent Insider Transactions Derivative • Mar 25
Director notifies of intention to sell stock Wan-Shan Chen intends to sell 10k shares in the next 90 days after lodging an Intent To Sell Form on the 22nd of March. If the sale is conducted around the recent share price of US$43.77, it would amount to US$438k. As of today, Wan-Shan currently holds no shares directly (This sale likely refers to shares that have not yet been received). There have been no trades via on-market transactions or options from company insiders in the last 12 months. Tillkännagivande • Mar 22
Belite Bio, Inc Announces PMDA Submission of Tinlarebant for Stargardt Disease Clinical Trial in Japan Belite Bio Inc. announced its submission to the Pharmaceuticals and Medical Devices Agency (PMDA) to initiate a clinical trial of Tinlarebant in adolescent STGD1 in Japan (“DRAGON II”). The DRAGON II trial is a combination of Phase 1b open-label study to evaluate the pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese adolescent STGD1 subjects and a Phase 2/3, global, multicenter, double-masked, placebo-controlled, randomized study designed to evaluate the efficacy, safety and tolerability of Tinlarebant in adolescent STGD1 subjects. Approximately 60 subjects, aged 12 to 20 years old, including approximately 10 Japanese subjects, are targeted for enrollment in the Phase 2/3 portion of the trial with a 1:1 randomization (tinlarebant:placebo). The data from Japanese subjects is intended to facilitate future NDA applications in Japan. Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in GA, or advanced Dry AMD. Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., and Orphan Drug Designation in the U.S., Europe, and Japan for the treatment of STGD1. STGD1 is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by mutations in a retina-specific gene (ABCA4), which results in progressive accumulation of bisretinoids leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging systems have helped ophthalmologists identify and monitor disease progression. Currently, there are no FDA approved treatments for STGD1. Importantly, STGD1 and GA, or advanced Dry AMD, share a similar pathophysiology, which is characterized by the excessive accumulation of bisretinoids, retinal cell death, and progressive loss of vision. Vision loss occurs slowly, despite peripheral expansion of “dead retina,” until the disease reaches the center of the eye (the macula). Therefore, Belite Bio is evaluating safety and efficacy of Tinlarebant in GA patients in a 2-year Phase 3 study (PHOENIX). Dry AMD is a leading cause of vision loss in older adults. Geographic Atrophy, or GA, is the advanced stage of AMD. Currently, there are no FDA approved orally administered treatments for GA and no FDA approved therapies for the other stages of Dry AMD other than GA. There are an estimated 20 million AMD patients in the U.S. and over 196 million patients worldwide with an estimated global direct healthcare cost of USD 255 billion. Recent Insider Transactions Derivative • Mar 17
Chief Scientific Officer notifies of intention to sell stock Nathan Mata intends to sell 6k shares in the next 90 days after lodging an Intent To Sell Form on the 14th of March. If the sale is conducted around the recent share price of US$38.30, it would amount to US$230k. As of today, Nathan currently holds no shares directly (This sale likely refers to shares that have not yet been received). There have been no trades via on-market transactions or options from company insiders in the last 12 months. Recent Insider Transactions Derivative • Dec 31
CFO & Director notifies of intention to sell stock Hao-Yuan Chuang intends to sell 100k shares in the next 90 days after lodging an Intent To Sell Form on the 28th of December. If the sale is conducted around the recent share price of US$45.70, it would amount to US$4.6m. Since March 2023, Hao-Yuan has owned 512.21k shares directly. There have been no trades via on-market transactions or options from company insiders in the last 12 months. Recent Insider Transactions Derivative • Dec 07
Director notifies of intention to sell stock Wan-Shan Chen intends to sell 6k shares in the next 90 days after lodging an Intent To Sell Form on the 28th of November. If the sale is conducted around the recent share price of US$37.48, it would amount to US$225k. As of today, Wan-Shan currently holds no shares directly (This sale likely refers to shares that have not yet been received). There have been no trades via on-market transactions or options from company insiders in the last 12 months. Recent Insider Transactions Derivative • Nov 19
Key Executive notifies of intention to sell stock Yu-Hsin Lin intends to sell 100k shares in the next 90 days after lodging an Intent To Sell Form on the 16th of November. If the sale is conducted around the recent share price of US$37.02, it would amount to US$3.7m. Since December 2022, Yu-Hsin has owned 731.73k shares directly. There have been no trades via on-market transactions or options from company insiders in the last 12 months. Recent Insider Transactions Derivative • Sep 03
Key Executive notifies of intention to sell stock Yu-Hsin Lin intends to sell 50k shares in the next 90 days after lodging an Intent To Sell Form on the 31st of August. If the sale is conducted around the recent share price of US$32.00, it would amount to US$1.6m. Since December 2022, Yu-Hsin has owned 731.73k shares directly. There have been no trades via on-market transactions or options from company insiders in the last 12 months. New Risk • Aug 14
New minor risk - Shareholder dilution The company's shareholders have been diluted in the past year. Increase in shares outstanding: 9.5% This is considered a minor risk. Shareholder dilution occurs when there is an increase in the number of shares on issue that is not proportionally distributed between all shareholders. Often due to the company raising equity capital or some options being converted into stock. All else being equal, if there are more shares outstanding then each existing share will be entitled to a lower proportion of the company's total earnings, thus reducing earnings per share (EPS). While dilution might not always result in lower EPS (like if the company is using the capital to fund an EPS accretive acquisition) in a lot cases it does, along with lower dividends per share and less voting power at shareholder meetings. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (16% average weekly change). Revenue is less than US$1m. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$13m net loss in 3 years). Shareholders have been diluted in the past year (9.5% increase in shares outstanding). New Risk • Jul 29
New major risk - Share price stability The company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of American stocks, typically moving 16% a week. This is considered a major risk. Share price volatility increases the risk of potential losses in the short-term as the stock tends to have larger drops in price more frequently than other stocks. It may also indicate the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (16% average weekly change). Revenue is less than US$1m. Minor Risk Currently unprofitable and not forecast to become profitable over next 3 years (US$18m net loss in 3 years). Price Target Changed • Jul 28
Price target decreased by 21% to US$45.67 Down from US$57.50, the current price target is an average from 3 analysts. New target price is 89% above last closing price of US$24.10. Stock is down 28% over the past year. The company is forecast to post a net loss per share of US$1.05 next year compared to a net loss per share of US$0.63 last year. Tillkännagivande • Jul 28
Belite Bio, Inc. Doses First Subject in Pivotal Phase 3 Phoenix Trial Evaluating Oral Tinlarebant for Ga Belite Bio Inc. announced that, in its pivotal Phase 3 "PHOENIX" trial for subjects with GA, the first subject has been dosed at the Retina Associates of Southern California, a clinical trial site in the United States. Phase 3 PHOENIX Trial Outline 2-yeaprprospective, randomized (2:1, active:placebo, n430 subjects), double-masked, placebo-conolled trial dedesigned to assess the efficacy and safety of daily oral Tinlarebant. Enrollment of GA subjects across multiple centers globally has begun in the third quarter 2023. The primary endpoint will be based upon the slowing of DDAF lesion growth rate from baseline to month 24, compared to placebo. An interim analysis of efficacy and safety is expected to be conducted at the mid-point of the trial. Tillkännagivande • Jul 25
Belite Bio, Inc. Completes Enrollment in Pivotal Global Phase 3 Dragon Trial Evaluating Oral Tinlarebant for Stargardt Disease Belite Bio Inc. announced that enrollment of its pivotal global Phase 3 "DRAGON" trial for patients with Stargardt Disease (STGD1) has completed. Tinlarebant is an oral, once daily retinol binding protein 4 (RBP4) antagonist designed to lower levels of ocular vitamin-A based toxins implicated in STGD1. Professor Michel Michaelides, the Principal Investigator and the Chief Investigator of the DRAGON trial from Moorfields Eye Hospital in the United Kingdom, one of the top recruiting sites globally, added that, “It has been a pleasure and privilege to be one of the recruiting sites for this promising Phase 3 DRAGON trial. The sustained slowing of disease progression demonstrated in the 18-month Phase 2 trial for patients with STGD1 is highly encouraging, and further reinforces company's belief in the transformative potential of Tinlarebant to be the first FDA-approved treatment option for STGD1.”The pivotal Phase 3 DRAGON trial is a randomized, double-masked, placebo-controlled, global and multi-center study, designed to evaluate the safety and efficacy of Tinlarebant in adolescent STGD1 patients. The DRAGON trial has global sites in 11 countries, including the U.S., the United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia. 90 patients have been enrolled in this study with a 2:1 randomization (active:placebo). The primary efficacy endpoint is slowing of lesion growth rate, along with the assessment of safety and tolerability. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease Designation in the U.S., and Orphan Drug Designation in both the U.S. and Europe for STGD1. Topline data for Tinlarebant in the Phase 2 STGD1 study is expected in the fourth quarter of 2023 and the interim data for the Phase 3 DRAGON trial is expected by mid-2024. In addition, Belite plans to have the first patient enrolled in the 2-year Phase 3 study (PHOENIX) of Tinlarebant in Geographic Atrophy (GA) in the third quarter of 2023. Tillkännagivande • May 11
Belite Bio, Inc to Report Q1, 2023 Results on May 10, 2023 Belite Bio, Inc announced that they will report Q1, 2023 results on May 10, 2023 Tillkännagivande • Nov 19
Belite Bio, Inc Finalizes Phase 3 Clinical Trial Plans for Advanced Dry AMD Treatment with Tinlarebant (LBS-008) Belite Bio Inc. announced that, following discussions with the FDA, it has finalized the study design for its planned Phase 3 clinical trial to evaluate efficacy and safety of Tinlarebant (a/k/a LBS-008) in patients with geographic atrophy (GA) associated with Dry AMD. Tinlarebant is a novel oral therapy intended as an early intervention to prevent the accumulation of vitamin A-based toxins (bisretinoids) that cause STGD1 and contribute to pathogenesis in Dry AMD. Bisretinoids are formed as by-products of vitamin A in the visual cycle. Tinlarebant works by reducing the level of serum RBP4, the carrier protein which transports the retinol to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids to preserve the health of retinal tissues. STGD1 is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by a dysfunctional retina-specific protein (ABCA4) which causes an early, aberrant accumulation of cytotoxic byproducts of vitamin A in the retina leading to retinal cell death and progressive loss of vision. There are currently no approved treatments available for STGD1. Dry AMD is a leading cause of vision loss in the U.S. Dry AMD has a heterogenous etiology but shows a pathophysiology that is similar to that of STGD1 in which the excessive accumulation of cytotoxic bisretinoids leads to retinal cell death and loss of vision. There are an estimated 11 million patients with dry AMD in the U.S. and over 196 million patients worldwide with an estimated global direct healthcare cost of USD 255 billion. An advanced vision-threatening form of dry AMD is Geographic Atrophy (GA), which is characterized by progressive atrophy of the retinal pigment epithelium and underlying choriocapillaris, followed by loss of photoreceptors within the retina, and ongoing visual impairment. Over 8 million people are affected worldwide with GA, which is approximately 20% of all individuals with AMD. The incidence of GA is expected to rise as the age-burden of developed countries is increasing. There are currently no approved treatments available for Dry AMD. Board Change • Nov 16
Less than half of directors are independent Following the recent departure of a director, there are only 3 independent directors on the board. The company's board is composed of: 3 independent directors. 4 non-independent directors. Independent Director Ita Lu was the last independent director to join the board, commencing their role in 2022. The company's minority of independent directors is a risk according to the Simply Wall St Risk Model. Tillkännagivande • Oct 03
Belite Bio, Inc Presents 12-Month Interim Results of LBS-008 Phase 1b/2 Study in Adolescent Stargardt Disease At AAO Annual Meeting 2022 Belite Bio Inc. presented one-year data from their ongoing two-year Phase 2 clinical study of Tinlarebant in STGD1 as part of the oral presentation series at the Annual Meeting of the American Academy of Ophthalmology (AAO) held during September 30 - October 3, 2022 at McCormick Place, Chicago. To date, all 13 patients have completed one-year of treatment in the ongoing two-year Phase 2 study of Tinlarebant. The results for safety and tolerability assessments, and retinal imaging data have been collected for the evaluation of disease progression. Images from spectral-domain optical coherence tomography imaging, an imaging modality that permits visualization of the retinal anatomy, have shown a stabilization of retinal thickness in many subjects. Fundus autofluorescence imaging shows no autofluorescence expansion (QDAF) in 7 of 13 (53.8%) subjects, and 12 of 13 (92.3%) subjects also show no atrophic lesion (DDAF) after one year of treatment. More importantly, 9 of 13 (69.2%) subjects show a stabilization or improvement in visual acuity throughout the one-year treatment period. According to the international prospective study of STGD1 (the ProgStar Study), childhood-onset patients with no atrophic lesion (DDAF) at baseline experienced an average lesion growth rate of 0.66 mm(2) for the left eyes and 0.74 mm(2) for the right eyes at one year. In addition, the Prospective Cohort Study of Childhood-Onset Stargardt Disease by Georgiou et al. reported an average atrophic lesion growth rate (DDAF) of 0.69 mm(2) /year for children. Belite Bio's one-year data from ongoing Phase 2 study showed an average lesion growth rate of 0.03 mm(2) /year, demonstrating a promising trend toward halting or slowing the disease progression in the study cohort. Belite Bio expects the next data readout in its Phase 2 STGD1 study to occur during the second quarter of 2023 when all subjects will complete 18 months of treatment. Tillkännagivande • Sep 18
Belite Bio Receives Approval of Lbs-008 Phase 3 Clinical Trial in China Belite Bio Inc. announced the approval from the National Medical Products Administration (NMPA) of China to initiate the Phase 3 clinical trial of LBS-008 in adolescent STGD1 in China. Belite Bio's DRAGON trial is a 2-year Phase 2 trial and a 2-year Phase 3 trial of LBS-008 in adolescent STGD1 subjects which is currently underway. The Phase 2 trial has enrolled a total of 13 subjects at clinical sites in Australia and Taiwan. Preliminary data from the Phase 2 trial at the first 6-month interval shows that 8 of the 13 patients (or 61.5%) recorded a gain in best-corrected visual acuity (BCVA) in at least one eye, including 2 patients who recorded a BCVA gain in both eyes. In addition, there were no atrophic lesions in any of the 13 subjects at study start and only 1 subject showed evidence of a retinal lesion (0.3mm(2) in size) at 6-months. Belite expects the next data readout of this Phase 2 trial to occur in the last quarter of 2022 when all subjects have completed 12 months of treatment. The DRAGON trial is a phase 3, randomized, double-masked, placebo-controlled, global and multi-center study, designed to evaluate the safety and efficacy of LBS-008 in adolescent STGD1 patients. To date, the Company has commenced the DRAGON trial in the U.S., the United Kingdom, Germany, Belgium, Switzerland, Hong Kong, Taiwan, and Australia. The accumulation of toxic bisretinoids have also been implicated in the progression of Dry AMD, a disease with a huge unmet need, which primarily affects the elderly and shows a pathophysiology that is similar to that of STGD1. This finding has led to the sponsorship and endorsement of LBS-008 by the NIH Blueprint program as a promising first-in-class oral medication to slow or halt the progression of Dry AMD. Belite believes that LBS-008 has the potential to be an effective early intervention treatment to maintain the health of retinal tissues in Dry AMD. Belite plans to initiate a Phase 2/3 clinical trial for Dry AMD in the fourth quarter of 2022. About LBS-008 LBS-008 is a novel oral therapy intended as an early intervention to prevent the buildup of toxins in the eye that cause STGD1 and contribute to Dry AMD. These toxins are by-products of vitamin A in the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. LBS-008 works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for transport of retinol into the eye. By modulating the amount of retinol entering the eye, LBS-008 reduces the formation of vitamin A-based toxins which have been implicated in STGD1 and Dry AMD in order to maintain the health of retinal tissues. LBS-008 has been granted Fast Track Designation, Rare Pediatric Disease Designation in the U.S., and Orphan Drug Designation in the U.S. and Europe for the treatment of STGD1. Stargardt Disease STGD1 is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by a dysfunctional retina-specific gene (ABCA4) which results in massive accumulation of toxic vitamin A byproducts (known as 'bisretinoids') in the retina leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging have helped ophthalmologists identify and monitor disease progression. STGD1 and Dry AMD share a similar pathophysiology characterized by excessive accumulation of cytotoxic bisretinoids, retinal cell death, and loss of vision. Vision loss occurs slowly, despite peripheral expansion of 'dead retina', until the disease reaches the center of the eye (the macula). Dry Age-related Macular Degeneration Dry AMD is a leading cause of vision loss in the U.S. There are no approved treatments available for Dry AMD. There are an estimated 11 million Dry AMD patients in the U.S. and over 196 million patients worldwide with an estimated global direct healthcare cost of US$255 billion. Tillkännagivande • Aug 23
Belite Bio Initiates Pivotal Phase 3 Clinical Trial of Lbs-008 in Stargardt Disease in the U.S Belite Bio Inc. announced that it has commenced enrollment for the U.S. Phase 3 clinical trial of LBS-008 in patients with Stargardt Disease (STGD1), a progressively blinding disease with no approved treatment. LBS-008 is an orally-available, small molecule retinol binding protein 4 (RBP4) antagonist that selectively reduces the delivery of vitamin A (retinol) to the eye, leading to a reduction of toxic vitamin A byproducts (bisretinoids) that have been implicated in the onset and progression of STGD1. Sponsored by the NIH Blueprint program to treat non-neovascular age-related macular degeneration (Dry AMD), LBS-008 is also endorsed by NIH as a promising first-in-class oral medication to slow or halt the progression of Dry AMD, a disease which primarily affects the elderly and shares a similar pathophysiology as STGD1. There are currently no approved treatments for STGD1 or Dry AMD by the U.S. Food and Drug Administration (FDA). Belite Bio has completed multiple randomized, double-blind, placebo-controlled, Phase 1 trials of LBS-008 in healthy adult subjects, including a single ascending dose, or SAD trial in 40 subjects in the U.S., and a SAD trial in 39 subjects and a multiple ascending dose, or MAD, trial in 32 healthy adult subjects in Australia. These trials were conducted to confirm the safety, toxicity, PK, and PD of LBS-008 on a range of SAD (10-50 mg in the U.S.; 25-400 mg in Australia) /MAD (5-25 mg in Australia) levels in healthy adult subjects in fasted /fed conditions. All dose levels were well tolerated. Belite Bio is currently conducting a 2-year Phase 2 trial and a 2-year Phase 3 (DRAGON) trial of LBS-008 in adolescent STGD1 subjects. The Phase 2 trial has enrolled a total of 13 subjects at clinical sites in Australia and Taiwan. Preliminary data from the Phase 2 trial at the first 6-month interval shows that 8 of the 13 patients (or 61.5%) recorded a gain in best-corrected visual acuity (BCVA) in at least one eye, including 2 patients who recorded a BCVA gain in both eyes. In addition, there were no atrophic lesions in any of the 13 subjects at study start and only 1 subject showed evidence of a retinal lesion (~0.3mm2 in size) at 6 months. Belite Bio expects the next data readout of this Phase 2 trial to occur in the last quarter of 2022 when all subjects have completed 12 months of treatment. The Phase 3 (DRAGON) trial, a randomized, double-masked, placebo-controlled, global and multi-center study, is designed to evaluate the safety and efficacy of LBS-008 in adolescent STGD1 patients. To date, the Company has commenced the Phase 3 clinical trial in the U.S., the United Kingdom, Germany, Belgium, Switzerland, Hong Kong, Taiwan, and Australia. Approximately 60 patients are targeted for enrollment in this study with a 2:1 randomization (active:placebo). Additionally, because the accumulation of toxic bisretinoids has also been implicated in the progression of Dry AMD, Belite Bio believes that LBS-008 has the potential to be effective for the treatment of Dry AMD as well. Belite Bio plans to initiate a Phase 2/3 clinical trial for Dry AMD in the 4th quarter of 2022. Tillkännagivande • Aug 02
Belite Bio, Inc to Report Q2, 2022 Results on Aug 10, 2022 Belite Bio, Inc announced that they will report Q2, 2022 results on Aug 10, 2022 Tillkännagivande • Jul 20
Belite Bio, Inc Submits Investigational New Drug Application to FDA for Approval to Proceed with LBS-008 Phase 3 Clinical Trial for the Treatment of Stargardt Disease Belite Bio Inc. announced the submission of an Investigational New Drug application to the U.S. Food and Drug Administration (FDA) to support Phase 3 development of LBS-008, the Company’s orally administered treatment for Stargardt Disease (STGD1), a rare genetic eye disease that causes progressive vision loss in children and adults. LBS-008 is a small molecule retinol binding protein 4 (RBP4) antagonist that selectively reduces the delivery of vitamin A (retinol) to the eye leading to a reduction of toxic vitamin A byproducts (bisretinoids) that have been implicated in the onset and progression of STGD1. Sponsored by the NIH Blueprint program to treat non-neovascular age-related macular degeneration (dry AMD), LBS-008 is also endorsed by NIH as a promising first-in-class oral medication intended to slow or halt the progression of dry AMD, a disease which primarily affects the elderly and shares a similar pathophysiology as STGD1. There are currently no FDA approved treatments for STGD1 or dry AMD. Belite Bio is currently conducting a 2-year Phase 2 trial and a 2-year Phase 3 trial of LBS-008 in adolescent STGD1 subjects. The Phase 2 trial, which has enrolled 13 subjects at clinical sites in Australia and Taiwan, was initiated immediately following the successful completion of a Phase 1b dose-finding trial in the same adolescent STGD1 patients. The preliminary data from the Phase 2 trial at the first 6-month interval shows that 8 of the 13 patients (or 61.5%) recorded a gain in BCVA in at least one eye, including 2 patients who recorded a gain in BCVA in both eyes. And 12 of the 13 patients had no DDAF lesion measured at the start of Phase 2 and at 6-months, only 1 of the 13 patients had a DDAF lesion growth of 0.3mm2 in both eyes during the 6-month period. LBS-008 is a novel oral therapy that prevents the accumulation of toxic byproducts of vitamin A which are known to cause STGD1 and contribute to dry AMD. These toxins are byproducts of the visual cycle, which is dependent on the supply of vitamin A to the eye. LBS-008 works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), a carrier protein that transports retinol to the eye. By modulating the amount of retinol entering the eye, LBS-008 reduces the formation of toxins which have been implicated in STGD1 and dry AMD. LBS-008 has been granted fast track designation, rare pediatric disease designation (RPD) in the U.S., and orphan drug designation (ODD) in the U.S. and Europe. Tillkännagivande • May 06
Belite Bio, Inc Presents Interim Results of LBS-008 Phase 1b/2 Study in Adolescent STGD1 at ARVO Annual Meeting 2022 Belite Bio Inc. (the ‘Company’) presented interim results of a Phase 1b/2 study of LBS-008 in adolescent STGD1 at The Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2022. LBS-008 is Belite Bio’s lead asset being developed for the treatment of STGD1 and Dry AMD. John Grigg, the study’s principal investigator and Head Specialty of Ophthalmology at the University of Sydney and Consultant Ophthalmologist at the Sydney Children’s Hospitals Network Westmead and Sydney Eye Hospital provided a presentation of the study and data. Study Background - STGD1 is the most common inherited macular dystrophy (causing blurring or loss of central vision). To date there are no treatments. This Phase 1b/2 study examined the safety and tolerability of LBS-008, a retinol binding protein 4 (RPB4) antagonist, in adolescent STGD1 subjects. Preclinical studies have shown that RBP4 inhibition slows disease progression and prevents retinal degeneration in a STGD1 animal model. Disease - Childhood-onset STGD1 is characterized by accumulation of bisretinoids which cause progressive retinal atrophy leading to rapid visual loss. Because bisretinoid toxins are derived from circulating vitamin A (retinol), reduction of retinol delivery to the eye, via antagonism of RPB4, has been explored as a means to slow disease progression in STGD1. LBS-008 is an orally administered, potent and specific non-retinoid antagonist of RPB4 and has been developed in order to determine whether reduction of circulating RPB4-retinol is a safe and effective treatment approach for STGD1 subjects. Trial Design - The Phase 1b/2 study is a multicenter, single arm, open-label study followed by a 2-year extension to evaluate safety, tolerability and efficacy of LBS-008. Thirteen subjects aged 12-18 years will receive oral LBS-008 (5 mg) daily over a 2-year treatment period. One (1/13) subject is dosing at 2mg a day. Treatment emergent adverse events (TEAEs), PK/PD, and visual function outcomes will be evaluated. Results - All 13 subjects have received at least 7 months of treatment and have completed the scheduled assessments at the first 6-month interval. The preliminary safety results of the Phase 1b/2 study show that the only drug-related adverse events reported were: delayed dark adaptation (DDA), reported by 9 of 13 subjects (or 69.2%) of which, 6 subjects having recovered; xanthopsia/chromatopsia was reported by 9 subjects (or 69.2%), with 3 subjects having recovered; and night vision impairment, reported by 1 patient (or 7.7%). All AEs were graded as mild. No severe AEs were reported and no AEs required discontinuation of treatment. No clinically significant findings in relation to vital signs, physical exams, cardiac health, or organ functions. The majority of subjects (8 of 13, 61.5%) recorded a gain in BCVA (ETDRS score) in at least one eye indicating stabilization of visual acuity, including 2 subjects with visual improvement in both eyes. Retinal imaging by fundus autofluorescence photography showed a trend for preventing or slowing expansion of autofluorescence. Areas of questionably decreased autofluorescence were either unchanged or reduced in one or both eyes in 8 of 13 subjects. There was no conversion to definitely decreased autofluorescence (atrophic retina) in 12 of 13 subjects. 6 subjects showed an improvement (narrowing) of the Ellipsoid Zone defect width in at least one eye, including 3 subjects showing improvements in both eyes. Where quantitative autofluorescence was available qAF was correlated with improvements in VA. Conclusions - LBS-008 (5 mg p.o., daily) effectively reduces RBP4 levels by 80-90%. Safe and well tolerated at the 7-month time point. A trend for preventing or slowing expansion of autofluorescence. A trend for improvement of the EZ defect width. Visual acuity has stabilized in a majority of subjects. The Company expects the next near-term data readout in its STGD1 Phase 2 trial to occur in the last quarter of 2022 when all subjects have completed 12 months of treatment. LBS-008 - LBS-008 is a novel oral therapy that prevents the buildup of toxins in the eye that cause STGD1 and contribute to dry AMD. These toxins are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. LBS-008 works by reducing and maintaining the levels of serum retinol binding protein 4 (RBP4), a carrier protein that transports retinol to the eye. By modulating the amount of retinol entering the visual cycle, LBS-008 reduces the formation of the toxins which have been implicated in progression of STGD1 and dry AMD in order to maintain the health of retinal tissues. LBS-008 has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of STGD1. Dry Age-related Macular Degeneration - Dry AMD is a leading cause of vision loss in the U.S. for which no approved treatments are available. There are an estimated 11 million dry AMD patients in the U.S. and over 196 million patients worldwide with an estimated global direct healthcare cost of $255 billion. Tillkännagivande • May 05
Belite Bio Receives FDA Fast Track Designation For LBS-008 Belite Bio Inc. announced that LBS-008, an orally administered tablet, has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of STGD1. This decision was based upon FDA’s review of non-clinical data and preliminary clinical data from studies of LBS-008.The Company expects the next near-term data readout in its STGD1 Phase 2 trial to occur in the last quarter of this year when all subjects have completed 12 months of treatment. Board Change • Apr 28
No independent directors Following the recent departure of a director, there are no independent directors on the board. The company's board is composed of: No independent directors. 4 non-independent directors. CFO & Director Hao-Yuan Chuang was the last director to join the board, commencing their role in 2021. The company's lack of independent directors is a risk according to the Simply Wall St Risk Model. 