{"success":true,"data":{"pressRelease":{"id":"137349","rtpr_id":"nGNX7ZkFj7-20260908","ticker":"BLTE","exchange":"NASDAQ","all_tickers":["BLTE"],"title":"Belite Bio Announces Submission of New Drug Application to the Ministry of Health, Labour, and Welfare in Japan under the Sakigake Designation System for Tinlarebant for the Treatment of Stargardt Disease Type 1","author":"Globe Newswire","published_at":"2026-09-08T10:00:00.262Z","article_body":"* U.S. Food and Drug Administration accepted the New Drug Application with\nPriority Review for tinlarebant for the treatment of Stargardt disease type 1;\nPrescription Drug User Fee Act target action date of February 12, 2027\n* If approved, tinlarebant would be the first-ever approved treatment for\nStargardt Disease Type 1 and could become the first Sakigake pharmaceutical\nproduct for ophthalmology disease in Japan\nSAN DIEGO, Sept. 08, 2026 (GLOBE NEWSWIRE) -- - Belite Bio\n(https://www.globenewswire.com/Tracker?data=5cThJhGKrc--_dIuAqT5jnLq08iVy0A2EoLi4P_pyBcRW4VuPR6e4McmP990F04QM3jOcfeY-cvXfn6s2KVgmKhLT1zUEPLwguwxSDLK-jwWN6n8wANOfwBSsvc3aEsM8uBdZm6-pLkwQYDpRyq7IISw8FdhWbvU2kYkB14FoY09vPfxBAqfhWhKTDUKFqzaqi5jMKMgE1UQc3loLctLty7SAKpMJJikxsDIbU8mUqE=),\nInc (NASDAQ: BLTE) (“Belite Bio(®)” or the “Company”), a\nclinical-stage drug development company focused on advancing novel\ntherapeutics targeting degenerative retinal diseases that have significant\nunmet medical needs, today announced the submission of a New Drug Application\n(NDA) to the Ministry of Health, Labour, and Welfare (MHLW) in Japan for\ntinlarebant in Stargardt disease type 1 (STGD1). Tinlarebant is an\ninvestigational, once-daily oral therapy for the treatment of STGD1, a rare,\ninherited retinal disease caused by mutations in the ABCA4 gene that leads to\nprogressive and irreversible vision loss. STGD1 affects an estimated 9,500\npeople in Japan alone, and there are currently no approved treatment options\nfor the disease.\n\nTinlarebant has been designated a SAKIGAKE pharmaceutical product for\naccelerated review and the NDA in Japan was filed side-by-side with the United\nStates NDA to enable simultaneous early access for patients in both regions.\nTinlarebant will be reviewed under the Sakigake Designation System - Japan’s\nexpedited regulatory pathway for innovative medical products. If approved, it\ncould become the first Sakigake pharmaceutical product for ophthalmology\ndisease in Japan.\n\n“The NDA submission in Japan under Sakigake designation reflects the\nsignificant unmet need for people living with STGD1 and is backed by our\nrobust data showing tinlarebant’s ability to slow the growth rate of retinal\nlesions compared to placebo. This is another important milestone as we\ncontinue to make rapid progress against our goal to bring tinlarebant to STGD1\npatients around the world,” said Dr. Tom Lin, Chairman and Chief Executive\nOfficer of Belite Bio. “I'd like to sincerely thank everyone in Japan who\nparticipated in or helped facilitate our clinical trials, as well as our\nentire team who helped make this submission possible. We look forward to\nworking closely with the MHLW as they review our application.”\n\nSakigake designation was established by MHLW to accelerate the drug approval\nprocess in Japan for innovative drugs with prominent effectiveness targeting\nserious diseases, in order to make them available to patients in Japan ahead\nof the rest of the world, by providing (a) prioritized consultation, (b)\npre-application consultation, (c) prioritized review, (d) assignment of a\nreview partner, and (e) extension of re-examination period.\n\n“Although Stargardt disease was first described nearly 120 years ago, no\napproved treatment has been established to date. We view our NDA in Japan as\nan important step toward delivering the first therapeutic option in the\ncountry for people living with Stargardt disease,” said Kaz Tsunaba,\nPresident of Belite Bio Japan. “We extend our sincere gratitude to the\npatients, their families, and the healthcare professionals who have supported\nthe development of this therapy. As tinlarebant could become the first\nophthalmology pharmaceutical product to receive regulatory approval under the\nSAKIGAKE designation, we remain committed to advancing all necessary\npreparations to ensure that it can be delivered to patients in Japan as early\nas possible.”\n\nAbout Tinlarebant (a/k/a LBS-008)\n\nTinlarebant is a novel oral therapy that is intended to reduce the\naccumulation of vitamin A-based toxins (known as bisretinoids) that cause\nretinal disease in Stargardt disease type 1 (STGD1) and also contribute to\ndisease progression in geographic atrophy (GA), or advanced dry age-related\nmacular degeneration (AMD). Bisretinoids are by-products of the visual cycle,\nwhich is dependent on the supply of vitamin A (retinol) to the eye.\nTinlarebant works by reducing and maintaining levels of serum retinol binding\nprotein 4 (RBP4), the sole carrier protein for retinol transport from the\nliver to the eye. By modulating the amount of retinol entering the eye,\ntinlarebant reduces the formation of bisretinoids. Tinlarebant has been\ngranted Breakthrough Therapy Designation, Fast Track Designation, and Rare\nPediatric Disease Designation in the U.S., Orphan Drug Designation in the\nU.S., Europe, Japan, and Switzerland, and Sakigake Designation in Japan for\nthe treatment of STGD1.\n\nAbout Belite Bio\n\nBelite Bio is a clinical-stage drug development company focused on advancing\nnovel therapeutics targeting degenerative retinal diseases that have\nsignificant unmet medical needs, such as Stargardt disease type 1 (STGD1) and\ngeographic atrophy (GA) in advanced dry age-related macular degeneration\n(AMD), in addition to specific metabolic diseases. Belite Bio’s lead\ncandidate, tinlarebant, is an oral therapy intended to reduce the accumulation\nof bisretinoid toxins in the eye. The Company has completed a Phase 3 trial\n(DRAGON) in adolescent and adult subjects with STGD1, which met its primary\nendpoint, and the drug is currently being evaluated in a Phase 2/3 trial\n(DRAGON II) in adolescent and adult subjects with STGD1 and a Phase 3 trial\n(PHOENIX) in subjects with GA. For more information, follow us on X,\nInstagram, LinkedIn, and Facebook, or visit us at www.belitebio.com.\n\nImportant Cautions Regarding Forward Looking Statements\n\nThis press release contains forward-looking statements within the meaning of\nSection 27A of the Securities Act of 1933, as amended, and Section 21E of the\nSecurities Exchange Act of 1934, as amended, including statements made\npursuant to the safe harbor provisions of the Private Securities Litigation\nReform Act of 1995. These forward-looking statements relate to future\nexpectations, plans and prospects, as well as other statements regarding\nmatters that are not historical facts. These statements include but are not\nlimited to statements regarding Belite Bio’s advancement of regulatory\nreview process, the ability and efficacy of tinlarebant to treat STGD1 and GA,\nthe potential approval of tinlarebant as the first therapy in the U.S. and\nJapan for people living with STGD1, Belite’s ability to successfully launch\nand market tinlarebant after its potential approval, as well as any other\nstatements regarding matters that are not historical facts, and any other\nstatements containing the words “may”, “will”, “expect”,\n“believe”, “target”, “plan”, “intend”, “continue”,\n“hope”, “potential”, “anticipate”, “estimate”, “look\nforward”, and other similar expressions. Actual results may differ\nmaterially from those indicated in the forward-looking statements as a result\nof various important factors related to Belite Bio’s business, including but\nnot limited to Belite Bio’s ability to demonstrate the safety and efficacy\nof its drug candidates; the clinical results for its drug candidates, which\nmay not support further development or regulatory approval; the timing to\ncomplete any ancillary clinical trials and/or to receive the interim/final\ndata of such clinical trials; the timing to communicate with and submit trial\ndata to regulatory authorities for drug approval in various jurisdictions; the\ncontent and timing of decisions made by the relevant regulatory authorities\nregarding regulatory approval of Belite Bio’s drug candidates; Belite\nBio’s ability to successfully commercialize tinlarebant, if approved,\nincluding its ability to build out commercial infrastructure, achieve market\nacceptance, and execute a timely product launch, as well as those risks more\nfully discussed in the “Risk Factors” section in Belite Bio’s filings\nwith the U.S. Securities and Exchange Commission. All forward-looking\nstatements are based on information currently available to Belite Bio, and\nBelite Bio undertakes no obligation to publicly update or revise any\nforward-looking statements, whether as a result of new information, future\nevents or otherwise, except as may be required by law.\n\nMedia and Investor Relations Contact:\nir@belitebio.com\n\n(https://www.globenewswire.com/NewsRoom/AttachmentNg/7f86ac70-6347-41c6-a622-e7000c42ad35)\n\n\n\nGlobeNewswire, Inc. 2026","article_body_html":"","raw_payload":{"data":{"id":"nGNX7ZkFj7-20260908","title":"Belite Bio Announces Submission of New Drug Application to the Ministry of Health, Labour, and Welfare in Japan under the Sakigake Designation System for Tinlarebant for the Treatment of Stargardt Disease Type 1","author":"Globe Newswire","ticker":"BLTE","created":"2026-09-08T10:00:00.262Z","tickers":["BLTE"],"exchange":"NASDAQ","article_body":"* U.S. Food and Drug Administration accepted the New Drug Application with\nPriority Review for tinlarebant for the treatment of Stargardt disease type 1;\nPrescription Drug User Fee Act target action date of February 12, 2027\n* If approved, tinlarebant would be the first-ever approved treatment for\nStargardt Disease Type 1 and could become the first Sakigake pharmaceutical\nproduct for ophthalmology disease in Japan\nSAN DIEGO, Sept. 08, 2026 (GLOBE NEWSWIRE) -- - Belite Bio\n(https://www.globenewswire.com/Tracker?data=5cThJhGKrc--_dIuAqT5jnLq08iVy0A2EoLi4P_pyBcRW4VuPR6e4McmP990F04QM3jOcfeY-cvXfn6s2KVgmKhLT1zUEPLwguwxSDLK-jwWN6n8wANOfwBSsvc3aEsM8uBdZm6-pLkwQYDpRyq7IISw8FdhWbvU2kYkB14FoY09vPfxBAqfhWhKTDUKFqzaqi5jMKMgE1UQc3loLctLty7SAKpMJJikxsDIbU8mUqE=),\nInc (NASDAQ: BLTE) (“Belite Bio(®)” or the “Company”), a\nclinical-stage drug development company focused on advancing novel\ntherapeutics targeting degenerative retinal diseases that have significant\nunmet medical needs, today announced the submission of a New Drug Application\n(NDA) to the Ministry of Health, Labour, and Welfare (MHLW) in Japan for\ntinlarebant in Stargardt disease type 1 (STGD1). Tinlarebant is an\ninvestigational, once-daily oral therapy for the treatment of STGD1, a rare,\ninherited retinal disease caused by mutations in the ABCA4 gene that leads to\nprogressive and irreversible vision loss. STGD1 affects an estimated 9,500\npeople in Japan alone, and there are currently no approved treatment options\nfor the disease.\n\nTinlarebant has been designated a SAKIGAKE pharmaceutical product for\naccelerated review and the NDA in Japan was filed side-by-side with the United\nStates NDA to enable simultaneous early access for patients in both regions.\nTinlarebant will be reviewed under the Sakigake Designation System - Japan’s\nexpedited regulatory pathway for innovative medical products. If approved, it\ncould become the first Sakigake pharmaceutical product for ophthalmology\ndisease in Japan.\n\n“The NDA submission in Japan under Sakigake designation reflects the\nsignificant unmet need for people living with STGD1 and is backed by our\nrobust data showing tinlarebant’s ability to slow the growth rate of retinal\nlesions compared to placebo. This is another important milestone as we\ncontinue to make rapid progress against our goal to bring tinlarebant to STGD1\npatients around the world,” said Dr. Tom Lin, Chairman and Chief Executive\nOfficer of Belite Bio. “I'd like to sincerely thank everyone in Japan who\nparticipated in or helped facilitate our clinical trials, as well as our\nentire team who helped make this submission possible. We look forward to\nworking closely with the MHLW as they review our application.”\n\nSakigake designation was established by MHLW to accelerate the drug approval\nprocess in Japan for innovative drugs with prominent effectiveness targeting\nserious diseases, in order to make them available to patients in Japan ahead\nof the rest of the world, by providing (a) prioritized consultation, (b)\npre-application consultation, (c) prioritized review, (d) assignment of a\nreview partner, and (e) extension of re-examination period.\n\n“Although Stargardt disease was first described nearly 120 years ago, no\napproved treatment has been established to date. We view our NDA in Japan as\nan important step toward delivering the first therapeutic option in the\ncountry for people living with Stargardt disease,” said Kaz Tsunaba,\nPresident of Belite Bio Japan. “We extend our sincere gratitude to the\npatients, their families, and the healthcare professionals who have supported\nthe development of this therapy. As tinlarebant could become the first\nophthalmology pharmaceutical product to receive regulatory approval under the\nSAKIGAKE designation, we remain committed to advancing all necessary\npreparations to ensure that it can be delivered to patients in Japan as early\nas possible.”\n\nAbout Tinlarebant (a/k/a LBS-008)\n\nTinlarebant is a novel oral therapy that is intended to reduce the\naccumulation of vitamin A-based toxins (known as bisretinoids) that cause\nretinal disease in Stargardt disease type 1 (STGD1) and also contribute to\ndisease progression in geographic atrophy (GA), or advanced dry age-related\nmacular degeneration (AMD). Bisretinoids are by-products of the visual cycle,\nwhich is dependent on the supply of vitamin A (retinol) to the eye.\nTinlarebant works by reducing and maintaining levels of serum retinol binding\nprotein 4 (RBP4), the sole carrier protein for retinol transport from the\nliver to the eye. By modulating the amount of retinol entering the eye,\ntinlarebant reduces the formation of bisretinoids. Tinlarebant has been\ngranted Breakthrough Therapy Designation, Fast Track Designation, and Rare\nPediatric Disease Designation in the U.S., Orphan Drug Designation in the\nU.S., Europe, Japan, and Switzerland, and Sakigake Designation in Japan for\nthe treatment of STGD1.\n\nAbout Belite Bio\n\nBelite Bio is a clinical-stage drug development company focused on advancing\nnovel therapeutics targeting degenerative retinal diseases that have\nsignificant unmet medical needs, such as Stargardt disease type 1 (STGD1) and\ngeographic atrophy (GA) in advanced dry age-related macular degeneration\n(AMD), in addition to specific metabolic diseases. Belite Bio’s lead\ncandidate, tinlarebant, is an oral therapy intended to reduce the accumulation\nof bisretinoid toxins in the eye. The Company has completed a Phase 3 trial\n(DRAGON) in adolescent and adult subjects with STGD1, which met its primary\nendpoint, and the drug is currently being evaluated in a Phase 2/3 trial\n(DRAGON II) in adolescent and adult subjects with STGD1 and a Phase 3 trial\n(PHOENIX) in subjects with GA. For more information, follow us on X,\nInstagram, LinkedIn, and Facebook, or visit us at www.belitebio.com.\n\nImportant Cautions Regarding Forward Looking Statements\n\nThis press release contains forward-looking statements within the meaning of\nSection 27A of the Securities Act of 1933, as amended, and Section 21E of the\nSecurities Exchange Act of 1934, as amended, including statements made\npursuant to the safe harbor provisions of the Private Securities Litigation\nReform Act of 1995. These forward-looking statements relate to future\nexpectations, plans and prospects, as well as other statements regarding\nmatters that are not historical facts. These statements include but are not\nlimited to statements regarding Belite Bio’s advancement of regulatory\nreview process, the ability and efficacy of tinlarebant to treat STGD1 and GA,\nthe potential approval of tinlarebant as the first therapy in the U.S. and\nJapan for people living with STGD1, Belite’s ability to successfully launch\nand market tinlarebant after its potential approval, as well as any other\nstatements regarding matters that are not historical facts, and any other\nstatements containing the words “may”, “will”, “expect”,\n“believe”, “target”, “plan”, “intend”, “continue”,\n“hope”, “potential”, “anticipate”, “estimate”, “look\nforward”, and other similar expressions. Actual results may differ\nmaterially from those indicated in the forward-looking statements as a result\nof various important factors related to Belite Bio’s business, including but\nnot limited to Belite Bio’s ability to demonstrate the safety and efficacy\nof its drug candidates; the clinical results for its drug candidates, which\nmay not support further development or regulatory approval; the timing to\ncomplete any ancillary clinical trials and/or to receive the interim/final\ndata of such clinical trials; the timing to communicate with and submit trial\ndata to regulatory authorities for drug approval in various jurisdictions; the\ncontent and timing of decisions made by the relevant regulatory authorities\nregarding regulatory approval of Belite Bio’s drug candidates; Belite\nBio’s ability to successfully commercialize tinlarebant, if approved,\nincluding its ability to build out commercial infrastructure, achieve market\nacceptance, and execute a timely product launch, as well as those risks more\nfully discussed in the “Risk Factors” section in Belite Bio’s filings\nwith the U.S. Securities and Exchange Commission. All forward-looking\nstatements are based on information currently available to Belite Bio, and\nBelite Bio undertakes no obligation to publicly update or revise any\nforward-looking statements, whether as a result of new information, future\nevents or otherwise, except as may be required by law.\n\nMedia and Investor Relations Contact:\nir@belitebio.com\n\n(https://www.globenewswire.com/NewsRoom/AttachmentNg/7f86ac70-6347-41c6-a622-e7000c42ad35)\n\n\n\nGlobeNewswire, Inc. 2026"},"type":"article","timestamp":"2026-09-08T10:00:00.314347082Z","server_sent_at_ms":1788861600314},"received_at":"2026-09-08T10:00:00.476Z","source_url":null},"analysis":{"id":"126224","press_release_id":"137349","analysis_json":{"industry":{"label":"Biotechnology","sector":"Health Care"},"redFlags":["Approval not assured — FDA PDUFA action not until February 12, 2027 and no MHLW review timeline specified","Clinical-stage company with no approved products; commercial success contingent on regulatory outcomes in both jurisdictions"],"eventType":"regulatory","narrative":"Belite Bio has submitted a New Drug Application to Japan's Ministry of Health, Labour, and Welfare for tinlarebant in Stargardt disease type 1, with the application reviewed under Japan's expedited Sakigake Designation System.\n\nThe Japanese filing was made side-by-side with the U.S. NDA, which the FDA has accepted with Priority Review and a PDUFA target action date of February 12, 2027.\n\nIf approved, tinlarebant would be the first-ever approved treatment for STGD1 — a rare inherited retinal disease affecting an estimated 9,500 people in Japan with no current treatment options — and could become the first Sakigake-designated ophthalmology product in Japan.\n\nThe filing is backed by the completed Phase 3 DRAGON trial, which met its primary endpoint by slowing retinal lesion growth, while Phase 3 (PHOENIX) testing in geographic atrophy continues.","sentiment":"bullish","agentHooks":{"shouldPost":true,"suggestedAngle":"Potential first-ever Stargardt treatment advances on dual U.S.-Japan expedited regulatory tracks, with a February 2027 PDUFA catalyst ahead."},"keyFigures":{"drugName":"tinlarebant (LBS-008)","phaseOfTrial":"Phase 3","customDimensions":{"us_pathway":"FDA Priority Review with Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations","regulatory_pathway":"Sakigake Designation System (Japan)","stgd1_patients_japan":9500,"pdufa_target_action_date":"February 12, 2027"}},"quotedText":"The NDA submission in Japan under Sakigake designation reflects the significant unmet need for people living with STGD1 and is backed by our robust data showing tinlarebant’s ability to slow the growth rate of retinal lesions compared to placebo.","namedEntities":{"people":[{"name":"Dr. Tom Lin","role":"Chairman and CEO of Belite Bio"},{"name":"Kaz Tsunaba","role":"President of Belite Bio Japan"}],"products":["tinlarebant","LBS-008"],"companies":[{"name":"Belite Bio, Inc.","ticker":"BLTE","relationship":"filer"},{"name":"Ministry of Health, Labour, and Welfare (MHLW)","relationship":"regulator"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"}],"dollarAmounts":[]},"materialImpact":{"score":3,"reasoning":"NDA submission to Japan's MHLW under the expedited Sakigake pathway, filed side-by-side with a U.S. NDA already accepted with Priority Review (PDUFA date February 12, 2027). A meaningful de-risking milestone toward a potential first-ever STGD1 approval, but still a filing step rather than a binary approval event."},"tickerRelevance":{"others":[],"primary":"BLTE"},"globalImportance":32,"audienceRelevance":30,"eventTypeSecondary":[],"importanceComponents":{"tickerTier":"clinical-stage small/mid-cap biotech","eventGravity":"NDA submission under expedited Sakigake pathway, potential first-in-class indication","sectorWeight":"biotech regulatory milestones carry outsized equity sensitivity","pdufaCatalyst":"2027-02-12","firstInClassPotential":true}},"event_type":"regulatory","event_type_secondary":null,"sentiment":"bullish","material_impact_score":3,"narrative":"Belite Bio has submitted a New Drug Application to Japan's Ministry of Health, Labour, and Welfare for tinlarebant in Stargardt disease type 1, with the application reviewed under Japan's expedited Sakigake Designation System.\n\nThe Japanese filing was made side-by-side with the U.S. NDA, which the FDA has accepted with Priority Review and a PDUFA target action date of February 12, 2027.\n\nIf approved, tinlarebant would be the first-ever approved treatment for STGD1 — a rare inherited retinal disease affecting an estimated 9,500 people in Japan with no current treatment options — and could become the first Sakigake-designated ophthalmology product in Japan.\n\nThe filing is backed by the completed Phase 3 DRAGON trial, which met its primary endpoint by slowing retinal lesion growth, while Phase 3 (PHOENIX) testing in geographic atrophy continues.","key_figures":{"drugName":"tinlarebant (LBS-008)","phaseOfTrial":"Phase 3","customDimensions":{"us_pathway":"FDA Priority Review with Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations","regulatory_pathway":"Sakigake Designation System (Japan)","stgd1_patients_japan":9500,"pdufa_target_action_date":"February 12, 2027"}},"named_entities":{"people":[{"name":"Dr. Tom Lin","role":"Chairman and CEO of Belite Bio"},{"name":"Kaz Tsunaba","role":"President of Belite Bio Japan"}],"products":["tinlarebant","LBS-008"],"companies":[{"name":"Belite Bio, Inc.","ticker":"BLTE","relationship":"filer"},{"name":"Ministry of Health, Labour, and Welfare (MHLW)","relationship":"regulator"},{"name":"U.S. Food and Drug Administration","relationship":"regulator"}],"dollarAmounts":[]},"model_name":"glm-4.7","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-09-08T10:02:15.705Z","global_importance":32,"audience_relevance":30,"importance_components":{"tickerTier":"clinical-stage small/mid-cap biotech","eventGravity":"NDA submission under expedited Sakigake pathway, potential first-in-class indication","sectorWeight":"biotech regulatory milestones carry outsized equity sensitivity","pdufaCatalyst":"2027-02-12","firstInClassPotential":true}},"durationMs":63506,"modelName":"glm-4.7"}}