• Tinlarebant is Belite Bio’s orally administered pill meant to sluggish illness development in sufferers affected with Stargardt Illness (STGD1) and Geographic Atrophy (GA) in superior Dry Age-related Macular Degeneration (Dry AMD (NASDAQ:))
• Knowledge from a 24-month Part 2 trial in adolescent STGD1 topics confirmed a sustained decrease atrophic lesion progress in Tinlarebant-treated topics in comparison with ProgStar individuals possessing related baseline traits (aged ‰¤18 years) (p
• Within the Part 2 trial, 5 of 12 topics (42%) with recognized pathogenic ABCA4 mutations, no incident atrophic (DDAF) lesions had been fashioned in the course of the 24-month therapy interval and no change in autofluorescent (QDAF) lesions was noticed
• Enrollment of a pivotal international Part 3 trial of Tinlarebant in adolescent STGD1 topics (DRAGON) has been accomplished with interim knowledge anticipated in 4Q 2024
• Initiated DRAGON II trial of Tinlarebant in adolescent STGD1 sufferers
• Tinlarebant has been granted Orphan Drug Designation in Japan for the therapy of STGD1
• A worldwide Part 3 trial in GA (PHOENIX) is ongoing

SAN DIEGO, June 12, 2024 (GLOBE NEWSWIRE) — Belite Bio, Inc (NASDAQ: BLTE) (Belite Bio or the Firm), a clinical-stage biopharmaceutical drug growth firm centered on advancing novel therapeutics concentrating on degenerative retinal illnesses which have important unmet medical wants, in the present day proclaims that its lead pipeline, Tinlarebant, has been granted Sakigake Designation by the Ministry of Well being, Labour and Welfare in Japan (MHLW) for the therapy of STGD1.

Sakigake designation was established by MHLW to speed up drug approval course of in Japan for progressive medicine with outstanding effectiveness concentrating on critical illnesses, with the intention to make them accessible to sufferers in Japan forward of the remainder of the world, by offering (a) prioritized session, (b) pre-application session, (c) prioritized assessment, (d) task of a assessment associate, and (e) extension of re-examination interval.

About Tinlarebant (a/okay/a LBS-008)

Tinlarebant is a novel oral remedy that’s meant to scale back the buildup of vitamin A-based toxins (generally known as bisretinoids) that trigger retinal illness in STGD1 and likewise contribute to illness development in GA, or superior Dry AMD. Bisretinoids are by-products of the visible cycle, which depends on the provision of vitamin A (retinol) to the attention. Tinlarebant works by decreasing and sustaining ranges of serum retinol binding protein 4 (RBP4), the only service protein for retinol transport from the liver to the attention. By modulating the quantity of retinol getting into the attention, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Quick Observe Designation and Uncommon Pediatric Illness designation within the U.S., Orphan Drug Designation within the U.S. Europe, and Japan, and Sakigake Designation in Japan for the therapy of STGD1.

Stargardt Illness (STGD1)

STGD1 is the most typical inherited retinal dystrophy (inflicting blurring or lack of central imaginative and prescient) in each adults and kids. The illness is brought on by mutations in a retina-specific gene (ABCA4), which ends up in progressive accumulation of bisretinoids resulting in retinal cell loss of life and progressive lack of central imaginative and prescient. The fluorescent properties of bisretinoids and the event of retinal imaging techniques have helped ophthalmologists determine and monitor illness development. At the moment, there are not any FDA permitted therapies for STGD1.

Importantly, STGD1 and GA, or superior Dry AMD, share the same pathophysiology, which is characterised by the extreme accumulation of bisretinoids, retinal cell loss of life, and progressive lack of imaginative and prescient. Imaginative and prescient loss happens slowly, regardless of peripheral enlargement of lifeless retina, till the illness reaches the middle of the attention (the macula). Due to this fact, Belite Bio is evaluating security and efficacy of Tinlarebant in GA sufferers in a 2-year Part 3 research (PHOENIX).

GA in superior Dry Age-related Macular Degeneration (Dry AMD)

Dry AMD is a number one reason behind imaginative and prescient loss in older adults. Geographic Atrophy, or GA, is the superior stage of AMD. At the moment, there are not any FDA permitted orally administered therapies for GA and no FDA permitted therapies for the opposite phases of Dry AMD apart from GA. There are an estimated 20 million AMD sufferers within the U.S. and over 196 million sufferers worldwide with an estimated international direct healthcare price of US$255 billion.

About Belite Bio

Belite Bio is a clinical-stage biopharmaceutical drug growth firm centered on advancing novel therapeutics concentrating on retinal degenerative eye illnesses which have important unmet medical wants reminiscent of (i) atrophic age-related macular degeneration (AMD), generally generally known as Geographic Atrophy (GA) in superior dry AMD, and (ii) autosomal recessive Stargardt illness sort 1, or STGD1, along with particular metabolic illnesses. For extra data, observe us on Twitter, Instagram, LinkedIn, Fb (NASDAQ:) or go to us at www.belitebio.com.

Vital Cautions Concerning Ahead Trying Statements
This press release accommodates forward-looking statements about future expectations and plans, in addition to different statements relating to issues that aren’t historic details. These statements embrace however will not be restricted to statements relating to the potential implications of medical knowledge for sufferers, and Belite Bio’s development of, and anticipated preclinical actions, medical growth, regulatory milestones, and commercialization of its product candidates, and another statements containing the phrases anticipate, hope and related expressions. Precise outcomes could differ materially from these indicated within the forward-looking statements on account of numerous necessary elements, together with however not restricted to Belite Bio’s capability to display the protection and efficacy of its drug candidates; the medical outcomes for its drug candidates, which can not assist additional growth or regulatory approval; the timing to finish related medical trials and/or to obtain the interim/ultimate knowledge of such medical trials; the content material and timing of selections made by the related regulatory authorities relating to regulatory approval of Belite Bio’s drug candidates; the potential efficacy of Tinlarebant, in addition to these dangers extra absolutely mentioned within the Danger Elements part in Belite Bio’s filings with the U.S. Securities and Alternate Fee. All forward-looking statements are primarily based on data at the moment accessible to Belite Bio, and Belite Bio undertakes no obligation to publicly replace or revise any forward-looking statements, whether or not on account of new data, future occasions or in any other case, besides as could also be required by regulation.

Media and Investor Relations Contact:
Jennifer Wu /ir@belitebio.com
Julie Fallon / belite@argotpartners.com

Supply: Belite Bio, Inc