gildeuretinol (ALK-001) / Tarsus Pharma - LARVOL DELTA

TEASE-EXT: Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE) (clinicaltrials.gov) - P2 | N=200 | Active, not recruiting | Sponsor: Alkeus Pharmaceuticals, Inc. | Trial completion date: Dec 2026 ➔ Dec 2029 | Trial primary completion date: Dec 2026 ➔ Dec 2028

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

TEASE: Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease (clinicaltrials.gov) - P2 | N=160 | Active, not recruiting | Sponsor: Alkeus Pharmaceuticals, Inc. | Enrolling by invitation ➔ Active, not recruiting | Trial completion date: Nov 2026 ➔ Nov 2028 | Trial primary completion date: Jun 2026 ➔ Jun 2028

Enrollment closed • Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Safety and Effects of Gildeuretinol Acetate on Retinal Atrophic Lesions in Stargardt Disease: The TEASE-1 Randomized Clinical Trial. (PubMed, JAMA Ophthalmol) - P2 | "Further research is needed to determine the clinical relevance of this end point. ClinicalTrials.gov Identifier: NCT02402660."

Clinical • Journal • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Tarsus Pharmaceuticals Completes Acquisition of Alkeus Pharmaceuticals, Expanding Leadership in Eye Care (GlobeNewswire) - "With the completion of the acquisition, Tarsus adds worldwide rights to ALK-001, a new, investigational, late-stage molecular entity designed to reduce the accumulation of toxic dimers while preserving the normal visual cycle. Tarsus believes ALK-001 represents a potential blockbuster opportunity in one of the largest inherited retinal diseases. ALK-001...is currently being evaluated in the global NORTHSTAR Phase 3 trial, with topline data anticipated in 2029."

M&A • P3 data: top line • Inherited Retinal Dystrophy

Alkeus Pharmaceuticals Announces Japan Ministry of Health, Labor and Welfare (MHLW) Has Granted Orphan Drug Designation to Gildeuretinol for Stargardt Disease (GlobeNewswire) - "Gildeuretinol is currently being evaluated in the global Phase 3 NORTHSTAR Study."

Orphan drug • Ophthalmology

Gildeuretinol Acetate vs Placebo for Geographic Atrophy Secondary to Age-Related Macular Degeneration: The SAGA Randomized Clinical Trial. (PubMed, Ophthalmol Retina) - "The prespecified primary endpoint was not met. Prespecified piecewise and secondary functional/patient-reported analyses favored gildeuretinol with nominal P values; these exploratory findings support further evaluation in adequately powered trials."

Clinical • Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Baseline characteristics from the Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE) clinical program (ARVO 2026) - "Based in Cambridge, Mass., Alkeus is developing therapies for serious diseases of the eye with high unmet need. Alkeus' breakthrough-designated lead candidate, gildeuretinol acetate (ALK-001), currently is being evaluated in clinical trials for the treatment of Stargardt disease."

Clinical • Diabetic Retinopathy • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

NORTHSTAR: Study of ALK-001 on the Progression of Stargardt Disease (clinicaltrials.gov) - P3 | N=230 | Recruiting | Sponsor: Alkeus Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Nutritional supplements: current evidence for retinitis pigmentosa and Stargardt disease. (PubMed, Curr Opin Ophthalmol) - "Most nutritional supplements studied to date have not demonstrated durable or clinically meaningful benefit in IRDs. Vitamin A supplementation in retinitis pigmentosa is no longer supported by current evidence, while NAC remains a promising metabolic therapy under investigation. High-quality, genotype-informed clinical trials with clinically relevant endpoints are needed before nutritional interventions can be incorporated into IRD management guidelines."

Journal • Review • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

NORTHSTAR: Study of ALK-001 on the Progression of Stargardt Disease (clinicaltrials.gov) - P3 | N=230 | Not yet recruiting | Sponsor: Alkeus Pharmaceuticals, Inc.

New P3 trial • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

SAGA: Phase 2/3 Study of ALK-001 in Geographic Atrophy (clinicaltrials.gov) - P2/3 | N=200 | Completed | Sponsor: Alkeus Pharmaceuticals, Inc. | Phase classification: P3 ➔ P2/3

Phase classification • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Real-World Outcomes in Anaplastic Lymphoma Kinase-Positive Advanced Non-Small Cell Lung Cancer in Argentina: A Multicenter Retrospective Study (GAOT-ALK001). (PubMed, JCO Glob Oncol) - "This real-world study describes outcomes and treatment patterns among patients with ALKp in Argentina. It highlights disparities in access to optimal therapies and reinforces the need for equitable access to new-generation ALK inhibitors to improve clinical outcomes."

Journal • Real-world evidence • Retrospective data • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • ALK

Stargardt's Disease: Molecular Pathogenesis and Current Therapeutic Landscape. (PubMed, Int J Mol Sci) - "Small-molecule therapies target vitamin A dimerization (e.g., ALK-001), inhibit lipofuscin accumulation (e.g., soraprazan), or modulate the visual cycle (e.g., emixustat hydrochloride). Gene therapy trials explore ABCA4 supplementation including strategies like RNA exon editing (ACDN-01) and bioengineered ambient light-activated OPSIN...Trials like DRAGON (Phase 3, tinlarebant), STARLIGHT (phase 2, bioengineered OPSIN) show promise, but optimizing efficacy remains challenging. With the key problem of establishing genotype-phenotype correlations, the future of STGD1 therapy may rely on approaches targeting oxidative stress, lipid metabolism, inflammation, complement regulation, and genetic repair."

Journal • Review • Age-related Macular Degeneration • Gene Therapies • Inflammation • Inherited Retinal Dystrophy • Macular Degeneration • Metabolic Disorders • Ophthalmology • Retinal Disorders • ALK

Data from the TEASE-1 study of gildereutinol for Stargardt disease (GlobeNewswire) - P2 | N=160 | TEASE-1 (NCT02402660) | Sponsor: Alkeus Pharmaceuticals, Inc. | "The study showed daily oral gildeuretinol significantly slowed the growth of atrophic retinal lesions in Stargardt disease by a 21.6% reduction versus the untreated group (p<0.001). This treatment effect was maintained across all pre-specified sensitivity analyses. Gildeuretinol was well tolerated and demonstrated a consistent safety profile across all studies to date."

P2 data • Ophthalmology

Alkeus Pharmaceuticals Presents Gildeuretinol Data During the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting May 4-8, 2025 (GlobeNewswire) - P2/3 | N=220 | SAGA (NCT03845582) | Sponsor: Alkeus Pharmaceuticals, Inc.| "Alkeus Pharmaceuticals, Inc...today announced additional data from its Phase 2 clinical study (SAGA) in geographic atrophy (GA) secondary to age-related macular degeneration (AMD) in which patients treated with oral gildeuretinol had a slower decline in VFQ-25 (vision-related quality of life) and FRI (functional reading independence) index scores compared to placebo at month 24....In SAGA, a placebo-controlled, double masked Phase 2 study of gildeuretinol in GA secondary to AMD, gildeuretinol showed a clinically meaningful trend in slowing GA growth rate of 13.4% from 0 to 24 months (p=0.075), the study’s primary endpoint. In a sensitivity analysis, gildeuretinol showed a statistically significant reduction in the GA lesion growth rate of 15.3% vs. placebo from 6 to 24 months (p=0.047)....The majority of adverse events were mild or moderate."

P2 data • Age-related Macular Degeneration

TEASE-EXT: Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE) (clinicaltrials.gov) - P2 | N=200 | Active, not recruiting | Sponsor: Alkeus Pharmaceuticals, Inc. | Enrolling by invitation ➔ Active, not recruiting | N=140 ➔ 200

Enrollment change • Enrollment closed • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

TEASE: Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease (clinicaltrials.gov) - P2 | N=160 | Enrolling by invitation | Sponsor: Alkeus Pharmaceuticals, Inc. | Trial primary completion date: Dec 2024 ➔ Jun 2026 | Recruiting ➔ Enrolling by invitation | Trial completion date: Mar 2025 ➔ Nov 2026

Enrollment status • Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Alkeus Pharmaceuticals Announces Presentations of Gildeuretinol Data During the Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting May 4-8, 2025 (GlobeNewswire) - "Alkeus Pharmaceuticals, Inc...today announced that results from its clinical studies of investigational oral gildeuretinol for the treatment of Stargardt disease (TEASE) and geographic atrophy secondary to age-related macular degeneration (SAGA) will be presented at the 2025 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) being held May 4-8 in Salt Lake City."

Clinical data • Age-related Macular Degeneration

Oral Gildeuretinol Treatment in Geographic Atrophy Secondary to AMD in the SAGA Trial: Safety, Efficacy and Patient-reported Outcomes (ARVO 2025) - P3 | "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Patient reported outcomes • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Safety and Efficacy of Oral Gildeuretinol for Stargardt Disease: TEASE-1 Trial (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Alkeus Pharmaceuticals Announces Two Presentations of Oral Gildeuretinol Data During the 48th Annual Meeting of the Macula Society Being Held February 12-15, 2025 (GlobeNewswire) - "Alkeus Pharmaceuticals...announced that results from its clinical studies of investigational oral gildeuretinol for the treatment of Stargardt disease (TEASE) and geographic atrophy secondary to age-related macular degeneration (SAGA) will be presented at the 48th Annual Meeting of the Macula Society being held February 12-15 in Charlotte Harbor, Fla."

P2 data • P3 data • Age-related Macular Degeneration • Inherited Retinal Dystrophy

Alkeus Pharmaceuticals Announces New Positive Interim TEASE-3 Study Results Showing Gildeuretinol Prevented Disease Progression in Early-Stage Stargardt Patients (GlobeNewswire) - P2 | N=140 | TEASE (NCT02402660) | Sponsor: Alkeus Pharmaceuticals, Inc. | "Two additional TEASE-3 participants who recently completed 24 months of therapy showed no disease progression, consistent with prior results.A total of five patients have completed the 24-month study. Following the study, these patients have remained on treatment and continue to show no disease progression, with the longest duration of therapy to date being more than seven years.TEASE-3 is the first clinical trial in early-stage Stargardt patients to show no disease progression over multiple years, including preservation of visual acuity.These data will be presented January 14 at 4:30 p.m. PST during the J.P. Morgan Healthcare Conference...'We plan to submit an NDA to the U.S. Food and Drug Administration for gildeuretinol as a treatment for Stargardt disease as soon as possible in 2025 based on compelling data generated to date from our multi-study clinical program.'"

FDA filing • P2 data • Ophthalmology

Alkeus Pharmaceuticals Receives FDA Rare Pediatric Disease and Fast Track Designations for Gildeuretinol as a Treatment for Stargardt Disease (GlobeNewswire) - "Alkeus Pharmaceuticals, Inc...announced that gildeuretinol (ALK-001), an investigational oral therapy, has received Rare Pediatric Disease and Fast Track designations from the U.S. Food and Drug Administration (FDA) for the treatment of Stargardt disease."

Fast track • Ophthalmology

SAGA: Phase 3 Study of ALK-001 in Geographic Atrophy (clinicaltrials.gov) - P3 | N=200 | Completed | Sponsor: Alkeus Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed | N=300 ➔ 200 | Trial completion date: Apr 2024 ➔ Jun 2024

Enrollment change • Trial completion • Trial completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Alkeus Pharmaceuticals Announces Results from the SAGA Study of Oral Gildeuretinol in Patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration (GlobeNewswire) - P3 | N=300 | SAGA (NCT03845582) | Sponsor: Alkeus Pharmaceuticals, Inc. | "Alkeus Pharmaceuticals, Inc. today announced that the SAGA study of oral gildeuretinol acetate (ALK-001) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) demonstrated a reduction of 0.25 sqmm/year vs. placebo (p=0.07) in the primary endpoint of GA lesion growth rate at 24 months. Gildeuretinol demonstrated a statistically significant reduction in the loss of low luminance visual acuity (LLVA) at 24 months (p=0.03). Gildeuretinol showed a favorable safety profile consistent with other studies of gildeuretinol in Stargardt disease. The topline results will be presented as a late breaker at the 128th Annual Meeting of the American Academy of Ophthalmology (AAO) during Retina Subspecialty Day on Friday, October 18, in Chicago."

P3 data • Age-related Macular Degeneration • Ophthalmology