Belite Bio Receives Sakigake (Pioneer Drug) Designation of Tinlarebant for Stargardt Disease in Japan
Belite Bio has received the Sakigake (Pioneer Drug) designation from Japan's MHLW for its lead drug, Tinlarebant, aimed at treating Stargardt Disease (STGD1). Tinlarebant, an oral tablet, has shown promise in a 24-month Phase 2 trial by slowing the progression of atrophic lesions in adolescent STGD1 patients. 42% of participants with known ABCA4 mutations didn't develop new lesions, and no change in existing lesions was observed.
The global Phase 3 trial for adolescent STGD1, named 'DRAGON,' has completed enrollment, with interim results expected in Q4 2024. Additionally, the DRAGON II trial has started, and a global Phase 3 trial for Geographic Atrophy (GA) in Dry AMD, termed 'PHOENIX,' is ongoing. Tinlarebant has also received Orphan Drug Designation in Japan for STGD1 treatment.
- Sakigake designation for Tinlarebant in Japan accelerates drug approval for STGD1.
- 24-month Phase 2 trial shows Tinlarebant slows disease progression in adolescent STGD1 patients.
- 42% of Phase 2 participants with ABCA4 mutations saw no new lesion development.
- Global Phase 3 trial 'DRAGON' for STGD1 completed enrollment with interim results expected in Q4 2024.
- Initiation of DRAGON II trial for adolescent STGD1 patients.
- Tinlarebant granted Orphan Drug Designation in Japan for STGD1.
- No current Phase 3 trial results available, increasing uncertainty for investors.
- Long wait until Q4 2024 for interim Phase 3 results may affect investor confidence.
- Potential dependency on favorable clinical results to maintain stock performance.
Insights
The Sakigake Designation granted to Tinlarebant is a significant milestone for Belite Bio. This designation, akin to the FDA’s Breakthrough Therapy designation in the U.S., underscores the potential efficacy of Tinlarebant in treating Stargardt Disease (STGD1). The data from the 24-month Phase 2 trial is promising, showing a substantial reduction in atrophic lesion growth in adolescent subjects. Specifically, 42% of subjects with known pathogenic ABCA4 mutations showed no new atrophic lesions during the trial period. This is a critical indicator of Tinlarebant's potential long-term benefits in halting disease progression. However, it’s important for investors to note that while these results are promising, they come from a relatively small sample size. The completion of the pivotal global Phase 3 trial and its subsequent data will be important in verifying these findings on a larger scale. If Phase 3 results are consistent with Phase 2, Tinlarebant could become a leading treatment for STGD1, providing significant benefits to patients and stakeholders.
The Sakigake designation not only accelerates the regulatory review process in Japan but also positions Belite Bio favorably in the Japanese market, potentially ahead of competitors. The designation helps the company to benefit from prioritized consultations and reviews, which can shorten the time to market. Given that the Japanese market is known for its stringent regulatory environment, this approval also serves as a strong endorsement of Tinlarebant's clinical potential. Additionally, the completion of enrollment for the pivotal Phase 3 trial and the ongoing global trials indicate that Belite Bio is strategically poised to capture a significant market share in the retinal disease segment. Investors should also consider the potential for expanded indications, such as Geographic Atrophy in advanced Dry Age-related Macular Degeneration (Dry AMD), which could considerably broaden the drug’s market potential. While regulatory and market risks always exist, the strategic steps taken so far appear to be well-calculated and the Sakigake designation is a strong positive indicator for the company’s future prospects.
From a financial perspective, the Sakigake designation for Tinlarebant in Japan could significantly enhance Belite Bio’s valuation by reducing the time to potential revenue generation in this key market. This designation often leads to quicker market entry and, hence, faster monetization opportunities. The completion of the global Phase 3 trial enrollment and the robust Phase 2 results provide a solid foundation for investor confidence. However, investors should carefully watch the upcoming interim data expected in 4Q 2024. Positive interim results could act as a strong catalyst for the stock, while any negative surprises could lead to volatility. Moreover, the Orphan Drug Designation, which provides benefits like market exclusivity and tax credits, further strengthens the potential financial upside. It's important for investors to remain aware of the inherent risks and uncertainties in biopharmaceutical development, but the strategic gains from these designations could elevate Belite Bio’s market position considerably.
- Tinlarebant is Belite Bio’s orally administered tablet intended to slow disease progression in patients affected with Stargardt Disease (STGD1) and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD)
- Data from a 24-month Phase 2 trial in adolescent STGD1 subjects showed a sustained lower atrophic lesion growth in Tinlarebant-treated subjects compared to ProgStar participants possessing similar baseline characteristics (aged ≤18 years) (p<0.001)
- In the Phase 2 trial, 5 of 12 subjects (
42% ) with known pathogenic ABCA4 mutations, no incident atrophic (DDAF) lesions were formed during the 24-month treatment period and no change in autofluorescent (QDAF) lesions was observed - Enrollment of a pivotal global Phase 3 trial of Tinlarebant in adolescent STGD1 subjects (“DRAGON”) has been completed with interim data expected in 4Q 2024
- Initiated DRAGON II trial of Tinlarebant in adolescent STGD1 patients
- Tinlarebant has been granted Orphan Drug Designation in Japan for the treatment of STGD1
- A global Phase 3 trial in GA (“PHOENIX”) is ongoing
SAN DIEGO, June 12, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio” or the “Company”), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announces that its lead pipeline, Tinlarebant, has been granted Sakigake Designation by the Ministry of Health, Labour and Welfare in Japan (MHLW) for the treatment of STGD1.
Sakigake designation was established by MHLW to accelerate drug approval process in Japan for innovative drugs with prominent effectiveness targeting serious diseases, in order to make them available to patients in Japan ahead of the rest of the world, by providing (a) prioritized consultation, (b) pre-application consultation, (c) prioritized review, (d) assignment of a review partner, and (e) extension of re-examination period.
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 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., Orphan Drug Designation in the U.S. Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1.
Stargardt Disease (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).
GA in advanced Dry Age-related Macular Degeneration (Dry AMD)
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 US
About Belite Bio
Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting retinal degenerative eye diseases which have significant unmet medical needs such as (i) atrophic age-related macular degeneration (AMD), commonly known as Geographic Atrophy (GA) in advanced dry AMD, and (ii) autosomal recessive Stargardt disease type 1, or STGD1, in addition to specific metabolic diseases. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook or visit us at www.belitebio.com.
Important Cautions Regarding Forward Looking Statements
This press release contains forward-looking statements about future expectations and plans, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the potential implications of clinical data for patients, and Belite Bio’s advancement of, and anticipated preclinical activities, clinical development, regulatory milestones, and commercialization of its product candidates, and any other statements containing the words “expect”, “hope” 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 of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; 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. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.
Media and Investor Relations Contact:
Jennifer Wu /ir@belitebio.com
Julie Fallon / belite@argotpartners.com
FAQ
What designation did Tinlarebant receive from Japan's MHLW?
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