Tinlarebant

DSMB Endorses Continuation of Phase 3 DRAGON Trial for Tinlarebant in Stargardt Disease

3 months ago

• Independent Data Safety Monitoring Board recommends proceeding with Belite Bio's Phase 3 DRAGON trial of Tinlarebant for Stargardt disease without modifications, maintaining the original 104-subject sample size. • Interim analysis reveals Tinlarebant demonstrates favorable safety profile with visual acuity stabilization in most subjects, showing less than three-letter score changes under both standard and low luminance conditions. • The pivotal global trial, spanning 11 jurisdictions, is expected to complete by Q4 2025, evaluating the first potential treatment for Stargardt disease with multiple regulatory designations worldwide.

Belite Bio Secures $15 Million in Direct Offering to Advance Retinal Disease Therapeutics

4 months ago

• Belite Bio has entered into a securities purchase agreement for $15 million through a registered direct offering of ADSs and warrants at $58.07 per unit, with potential for additional $15 million from warrant exercises. • The clinical-stage biopharmaceutical company focuses on developing treatments for degenerative retinal diseases, including Stargardt disease and Geographic Atrophy in advanced dry AMD. • The offering, expected to close by February 7, 2025, will provide working capital for advancing the company's novel therapeutic pipeline targeting significant unmet medical needs.

Belite Bio Secures $15 Million Through Direct Offering to Advance Tinlarebant Development

4 months ago

• Belite Bio has announced a registered direct offering to raise $15 million in funding, strengthening its financial position for drug development initiatives. • The company's lead candidate Tinlarebant is advancing through clinical development, with the funding expected to support ongoing trials and regulatory activities. • The offering's completion remains subject to market conditions and customary closing requirements, reflecting standard securities transaction procedures.

Dry AMD Therapeutic Pipeline Shows Promise with 70+ Companies Advancing Novel Therapies

4 months ago

• The dry age-related macular degeneration (AMD) therapeutic landscape is robust, with over 70 companies developing more than 80 therapies. • Key companies like Alkeus Pharmaceuticals and Roche are evaluating new drugs, aiming to improve the treatment options for dry AMD. • Recent clinical trials show promise, with Ocugen reporting positive data for OCU410 and Eyestem Research achieving a milestone in cell therapy for geographic atrophy. • Several therapies, including gene therapies and complement inhibitors, are in various clinical trial phases, addressing the unmet needs in dry AMD treatment.

Lin BioScience Receives U.S. FDA Fast Track Designation For LBS-007

5 months ago

Lin BioScience's investigational drug LBS-007 has been granted Fast Track Designation by the U.S. FDA for treating acute myeloid leukemia, highlighting its potential to address unmet medical needs in acute leukemia treatment. The company is conducting a Phase 1/2 trial in patients with relapsed or resistant acute leukemias across the US, Australia, and Taiwan.

Belite Bio's Tinlarebant Phase III DRAGON Trial Approaches Critical Milestone for Stargardt Disease Treatment

5 months ago

Belite Bio's Phase III DRAGON trial investigating Tinlarebant, a novel oral treatment for Stargardt Disease (STGD1), is set to release interim analysis data in early 2025. The study represents a potential breakthrough for STGD1 patients, as there are currently no approved treatments for this inherited retinal dystrophy.

Gilead's Trodelvy Receives FDA Breakthrough Therapy Designation for SCLC; Seladelpar Recommended for EU Approval

5 months ago

• The FDA granted Breakthrough Therapy Designation to Gilead's Trodelvy for extensive-stage small cell lung cancer (ES-SCLC) after platinum-based chemotherapy, based on promising results from the TROPiCS-03 study. • Gilead will collaborate with Terray Therapeutics to discover and develop novel, small molecule therapies across multiple targets, leveraging Terray's tNova platform. • The European Medicines Agency's CHMP recommended approval of Gilead's seladelpar for primary biliary cholangitis (PBC) in combination with UDCA or as monotherapy.

LBS-007 Receives FDA Fast Track Designation for Acute Myeloid Leukemia Treatment

6 months ago

• Lin BioScience's LBS-007 has been granted Fast Track Designation by the FDA for acute myeloid leukemia (AML) treatment, expediting its development. • LBS-007, a novel cell cycle inhibitor, is currently in Phase 1/2 trials for relapsed or resistant acute leukemias in the US, Australia, and Taiwan. • The Fast Track designation aims to address the unmet medical need in AML, offering potential for more frequent FDA interactions and accelerated approval pathways. • LBS-007 has demonstrated a promising safety profile in Phase 1 dose escalation, with no significant adverse effects observed, according to Lin BioScience.

Belite Bio Advances Tinlarebant Trials for Stargardt Disease and Geographic Atrophy

6 months ago

• Belite Bio dosed the first patient in the Phase 2/3 DRAGON II trial of Tinlarebant for Stargardt disease, building on positive Phase 1b results. • The pivotal Phase 3 PHOENIX trial of Tinlarebant for geographic atrophy continues to enroll rapidly, with over 280 subjects currently participating. • Interim analysis from the pivotal Phase 3 DRAGON trial of Tinlarebant in adolescent Stargardt disease subjects is expected by early 2025. • Hendrik P.N. Scholl, MD, MA, a leading expert in retinal diseases, has been appointed as Belite Bio's Chief Medical Officer.

Belite Bio's Tinlarebant Shows Progress in Stargardt Disease and Geographic Atrophy Trials

6 months ago

• Belite Bio dosed the first patient in the Phase 2/3 DRAGON II trial of Tinlarebant for Stargardt disease (STGD1). • The global Phase 3 PHOENIX trial of Tinlarebant for geographic atrophy (GA) has enrolled over 280 subjects. • Interim analysis from the pivotal Phase 3 DRAGON trial in adolescent STGD1 subjects is expected by early 2025. • Hendrik P.N. Scholl, MD, MA, a leading ophthalmology expert, has been appointed as Chief Medical Officer.

AAO 2024: Emerging Data in Retinal Therapies for Geographic Atrophy, Stargardt Disease, and Retinitis Pigmentosa

7 months ago

• Alkeus Pharmaceuticals will present results from clinical studies of oral gildeuretinol for geographic atrophy secondary to age-related macular degeneration (SAGA) and Stargardt disease (TEASE). • Annexon will share new analyses of ANX007 from the Phase 2 ARCHER trial in geographic atrophy, highlighting clinical results and anatomical changes. • Nanoscope Therapeutics is set to share topline data from the RESTORE trial of MCO-010 therapy in patients with severe vision loss due to retinitis pigmentosa (RP).

Belite Bio Presents Promising Phase II Data on Tinlarebant for Adolescent Stargardt Disease

7 months ago

• Belite Bio will present Phase II trial data of Tinlarebant for adolescent Stargardt disease at the AAO 2024 meeting, showcasing its potential as a novel therapy. • The study evaluated Tinlarebant, an oral therapy designed to reduce vitamin A-based toxins, in young patients with Stargardt disease over two years. • Results showed stabilized visual acuity and halted lesion growth in treated subjects, suggesting a potential benefit in slowing disease progression. • Tinlarebant was well-tolerated, with mild adverse events and no discontinuations, supporting its continued evaluation in Phase 3 trials.

Belite Bio Doses First Patient in Phase 2/3 DRAGON II Trial of Tinlarebant for Stargardt Disease

8 months ago

• Belite Bio has dosed the first patient in the Phase 2/3 DRAGON II trial, evaluating Tinlarebant for Stargardt disease (STGD1) treatment. • The DRAGON II trial will assess Tinlarebant's efficacy, safety, and tolerability in approximately 60 adolescent STGD1 subjects across multiple sites. • Tinlarebant, an oral therapy, aims to reduce vitamin A-based toxins that contribute to retinal disease in STGD1 and advanced Dry AMD. • The trial includes sites in the U.S., U.K., and Japan, with data from Japanese subjects intended to support future NDA applications in Japan.

Drug Development Updates