belzupacap sarotalocan (AU-011) / Aura Biosci - LARVOL DELTA

SUO 2025: Trial in Progress: Safety and Efficacy of Belzupacap Sarotalocan (AU-011), a Virus-like Drug Conjugate, in Participants with Non-Muscle Invasive Bladder Cancer (UroToday)

Trial status • Bladder Cancer

AU-011-102: A Phase 1, Open-label Trial of Belzupacap Sarotalocan (AU-011) in Bladder Cancer (clinicaltrials.gov) - P1 | N=55 | Recruiting | Sponsor: Aura Biosciences | Trial primary completion date: Dec 2026 ➔ Mar 2027

Trial primary completion date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

CoMpass: A Phase 3 Randomized, Masked, Controlled Trial to Evaluate Efficacy and Safety of Belzupacap Sarotalocan (AU-011) Treatment Compared to Sham Control in Subjects With Primary Indeterminate Lesions or Small Choroidal Melanoma (clinicaltrials.gov) - P3 | N=100 | Recruiting | Sponsor: Aura Biosciences | Trial completion date: Mar 2027 ➔ Aug 2028 | Trial primary completion date: Mar 2026 ➔ Nov 2027

Trial completion date • Trial primary completion date • Choroidal Melanomas • Eye Cancer • Melanoma • Ocular Melanoma • Oncology • Solid Tumor • Uveal Melanoma

Additional Phase 1 Biological Activity Data Presented at the 45th Congress of the Société Internationale d’Urologie (SIU) (GlobeNewswire) - "Multiplex immune fluorescence data from the Phase 1 trial of bel-sar demonstrating robust induction of adaptive immune memory in patients with non-muscle invasive bladder cancer (NMIBC) were presented....These multiplex immune fluorescence data (n=5 patients from the Phase 1 trial) further characterize the previously announced results from the Company’s completed Phase 1 clinical trial of bel-sar in patients with NMIBC....In treated lesions, natural killer cell density increased up to 40x, CD4+ cytolytic T cell density increased up to 7x, and CD4+ and CD8+ memory T cells were observed after bel-sar treatment."

P1 data • Bladder Cancer

Ongoing Phase 3 CoMpass Trial (GlobeNewswire) - "...The Company currently expects that it can complete CoMpass enrollment in 2026 and provide topline data readout for the 15-month primary endpoint in the fourth quarter of 2027."

Enrollment status • P3 data: top line • Choroidal Melanomas • Uveal Melanoma

Ongoing Phase 1b/2 Trial (GlobeNewswire) - "This trial will evaluate additional doses and cycles of bel-sar in approximately 26 intermediate and high-risk NMIBC patients. Patients will be monitored for response assessments and recurrence at 3, 6, 9, and 12 months. This trial is actively enrolling and remains on track, with initial three-month clinical data expected in mid-2026."

P1/2 data • Trial status • Bladder Cancer

Trial in Progress: Safety and Efficacy of Belzupacap Sarotalocan (AU-011), a Virus-Like Drug Conjugate, in Participants with Non-Muscle Invasive Bladder Cancer (NMIBC) (SUO 2025) - P1 | "The trial is actively enrolling at multiple sites, with participants to be distributed across immune-ablative and neoadjuvant treatment cohorts. "

Clinical • Bladder Cancer • Choroidal Melanomas • Eye Cancer • Genito-urinary Cancer • Melanoma • Ocular Melanoma • Oncology • Solid Tumor • Urothelial Cancer • CD4 • CD8

Metastases to the Choroid (GlobeNewswire) - "The Company has dosed the first patient in an ongoing Phase 2 clinical trial in metastases to the choroid. A protocol amendment has now been implemented to expand the entry criteria to include all metastases arising from different solid tumors to facilitate enrollment into the study and broaden the proof of concept for bel-sar in this indication....With a four-week efficacy endpoint, the Company expects early proof of concept data from this trial in 2026."

P2 data • Trial status • Solid Tumor

Cancers of the Ocular Surface (GlobeNewswire) - "The Company is planning to initiate a Phase 1 proof-of-concept trial to assess safety, feasibility and tumor response through histopathologic evaluation at a 2–4-week time point. Development activities for this program remain on track, with early proof of concept data from Australian clinical sites expected in 2026."

Clinical data • New P1 trial • Eye Cancer

SYMP-27: Safety and Efficacy of AU-011, a Virus-Like Drug Conjugate, in Participants with NMIBC (SIU 2025) - No abstract available

Clinical • Bladder Cancer • Oncology

NMIBC: A Phase 1, Open-label Trial of Belzupacap Sarotalocan (AU-011) in Bladder Cancer (clinicaltrials.gov) - P1 | N=55 | Recruiting | Sponsor: Aura Biosciences | N=21 ➔ 55 | Trial completion date: Jul 2024 ➔ Apr 2027 | Trial primary completion date: Jun 2024 ➔ Dec 2026

Enrollment change • Trial completion date • Trial primary completion date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

AU-011-203: Suprachoroidal Administration in Subjects With Metastases to the Choroid (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Aura Biosciences | Not yet recruiting ➔ Recruiting

Enrollment open • Eye Cancer • Ocular Melanoma • Oncology

Clearside Biomedical Announces Multiple Presentations to be Featured at the Clinical Trials at the Summit Meeting (GlobeNewswire) - "Clearside Biomedical, Inc...announced today that the use of its SCS delivery platform will be featured in multiple presentations at the Clinical Trials at the Summit (CTS) Meeting on June 21, 2025 in Las Vegas, Nevada."

Clinical data • Age-related Macular Degeneration • Choroidal Melanomas • Diabetic Retinopathy

Beyond the injection: delivery systems reshaping retinal disease management. (PubMed, Expert Opin Pharmacother) - "Specific innovations discussed include the ranibizumab port delivery system, EYP-1901 (Duravyu, vorolanib implant), KSI-301 (tarcocimab tedromer), KSI-501, OTX-TKI (Axpaxli, axitinib implant), 4D-150, revakinagene taroretcel-lwey (Encelto, NT-501, encapsulated cell therapy), Xipere (triamcinolone acetonide injectable suspension), AU-011 (belzupacap sarotalocan targeted delivery), ABBV-RGX-314, elamipretide, and OCS-01 (high concentration dexamethasone)...Challenges include overcoming the blood-retinal barrier, surgical complications with implantable devices, and ensuring patient adherence. Advances in smart delivery systems, drug formulations, and predictive models, alongside interdisciplinary collaboration, will be crucial in achieving personalized, effective, and sustainable retinal therapies."

Journal • Review • Age-related Macular Degeneration • Diabetic Macular Edema • Diabetic Retinopathy • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders

Aura Biosciences Reports First Quarter 2025 Financial Results and Business Highlights (GlobeNewswire) - "First Patient Enrolled in Multi-Dose Phase 1b/2 Trial of Bel-sar in Non-Muscle-Invasive Bladder Cancer (NMIBC); Initial Data at 3 Months Expected by Year-End 2025...Update on Ongoing Phase 3 CoMpass Trial: CoMpass is the first registration-enabling study in early-stage choroidal melanoma...Given the momentum in the study globally, the Company believes study enrollment may be completed as early as the end of 2025....Research and development expenses increased to $23.3 million for the three months ended March 31, 2025 from $17.1 million for the three months ended March 31, 2024, primarily due to ongoing clinical and contract research organization costs associated with the progression of the Company’s Phase 3 trial of bel-sar in early-stage choroidal melanoma and manufacturing and development costs for bel-sar."

Commercial • P1/2 data • Trial status • Bladder Cancer • Choroidal Melanomas

Patent Application Filed for New Formulation of Bel-sar for Use in Bladder Cancer (GlobeNewswire) - "The Company has filed a patent application for a new formulation of bel-sar for use in urologic oncology. This new formulation is designed to enable convenient in-office urologist procedures with enhanced storage and handling at refrigerator temperatures, as well as an adjusted volume and concentration....The Company has filed a patent application with the U.S. Patent and Trademark Office covering the new formulation, which if issued, would provide patent coverage for this formulation into 2046."

Patent • Bladder Cancer

Aura Biosciences Reports Fourth Quarter and Full Year 2024 Financial Results and Business Highlights (GlobeNewswire) - "Update on Ongoing Phase 3 CoMpass Trial: CoMpass is the first registration-enabling study in early-stage choroidal melanoma....The CoMpass trial is actively enrolling globally. To identify appropriate patients to meet the enrichment strategy of documented growth, the Company has enabled a pre-screening ‘run in’ period. Globally, since June 2024, investigators have registered over 175 patients in pre-screening as having met initial enrollment criteria for the study. The acceleration in pre-screening is driven by increasing momentum in the United States and European Union."

Trial status • Choroidal Melanomas

Additional Ocular Oncology Indications:…Metastases to the Choroid (GlobeNewswire) - "The Company has initiated a Phase 2 clinical trial in metastases to the choroid and has sites activated with patients in prescreening. Metastases to the choroid is an indication with high unmet medical need and no approved therapies. The Company expects initial data from this trial in 2025."

P2 data • Trial status • Eye Cancer

Positive Data from Phase 1 Trial of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) Presented at the 40th Annual European Association of Urology Congress (GlobeNewswire) - P1 | N=21 | NCT05483868 | Sponsor: Aura Biosciences | "Clinical Complete Responses Observed with Single Low-Dose of Bel-sar in Patients with Intermediate and High-Risk NMIBC with Robust Cell-Mediated Immunity and Urothelial Field Effect; Favorable Safety Profile; Only Grade 1 Drug-Related Adverse Events Reported in Less Than 10% of Patients; Data Supports Potential for a Paradigm Shifting Approach in the Front-Line Treatment of patients with NMIBC....We look forward to our virtual urologic oncology investor event, where we will outline the Phase 1b/2 trial to further evaluate bel-sar’s clinical activity and future development plans."

New P1/2 trial • P1 data • Bladder Cancer • Urothelial Cancer

Virtual urologic oncology investor event on Monday, March 24, 2025, at 4:30 pm Eastern Time (GlobeNewswire) - "Aura will host a virtual urologic oncology investor event featuring...additional data from the ongoing Phase 1 trial in NMIBC, a multi-center, open-label clinical trial designed as a window of opportunity study to assess the safety and feasibility of local administration of bel-sar as a monotherapy prior to transurethral resection of bladder tumor (TURBT), the standard of care procedure....At the event, Aura will also provide a bladder cancer program update, including the planned Phase 1b/2 trial expansion and future development plans."

New P1/2 trial • P1 data • Bladder Cancer

Aura Biosciences Announces Additional Data from Ongoing Phase 1 Trial in Non-Muscle Invasive Bladder Cancer to be Presented as a Late-Breaking Abstract at the 40th Annual European Association of Urology Congress (GlobeNewswire) - "Aura Biosciences, Inc...announced that additional Phase 1 data evaluating bel-sar (AU-011) for the treatment of patients with non-muscle invasive bladder cancer (NMIBC) will be presented at the 40th Annual European Association of Urology (EAU) Congress being held March 21-24, 2025, in Madrid, Spain. Aura will also participate in the EAU Research Forum during the Congress."

P1 data • Bladder Cancer

Targeting ocular malignancies using a novel light-activated virus-like drug conjugate. (PubMed, Adv Ophthalmol Pract Res) - "A newly-developed virus-like drug conjugate is a promising nanoparticle with a defined target: the novel virus-like particle-photosensitizer conjugate Belzupacap sarotalocan (Bel-sar, previous name AU-011)...Its photodynamic therapy effectively treats pigmented and non-pigmented tumors while inducing immunogenic cell death through DAMP exposure, triggering local and systemic immune responses. Combining Bel-sar PDT with immunotherapy improves efficacy in preclinical models, warranting further clinical investigation."

Journal • Review • Eye Cancer • Melanoma • Ocular Melanoma • Oncology • Solid Tumor • Uveal Melanoma

Oncolytic Viruses and Immunotherapy for the Treatment of Uveal Melanoma and Retinoblastoma: The Current Landscape and Novel Advances. (PubMed, Biomedicines) - "The most recent advances in preclinical- and clinical-phase studies are further summarized. The recent developments in virus-like drug conjugates (i.e., AU011), oncolytic viruses for metastatic UM, and targeted immunotherapies have shown great results in clinical trials for the future clinical application of these novel technologies in the treatment algorithm of certain intraocular tumors."

Journal • Review • Eye Cancer • Immune Modulation • Immunology • Melanoma • Ocular Melanoma • Oncology • Retinal Disorders • Retinoblastoma • Solid Tumor • Uveal Melanoma

Aura Biosciences Reports Third Quarter 2024 Financial Results and Business Highlights (GlobeNewswire) - "The Company is initiating clinical development for bel-sar as a potential treatment for metastases to the choroid, an indication with high unmet medical need and no approved therapies. The Company aims to enroll the first patients in a Phase 2 trial in 2024....The Company plans to continue development of bel-sar in bladder cancer with an initial focus on low-grade, intermediate risk NMIBC patients, through a planned trial expansion to test additional doses and treatment regimens with the opportunity to assess early durability of response at 3 months. In parallel, the Company is planning regulatory discussions on the design of the next trial with the goal of expediting clinical development in this patient population."

Clinical protocol • New P2 trial • Trial status • Bladder Cancer • Choroidal Melanomas • Eye Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Multiple Clinical Complete Responses Demonstrated Following Single Low Dose Administration of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) in Ongoing Phase 1 Trial (GlobeNewswire) - P1 | N=21 | NCT05483868 | Sponsor: Aura Biosciences | "Aura Biosciences...announced positive early data from an ongoing Phase 1 clinical trial of bel-sar (AU-011) in patients with NMIBC. To date, the trial includes 13 patients, with the primary endpoints of evaluating the safety and feasibility of local administration of bel-sar alone (n=5) and bel-sar with light activation (n=8)....10 of 13 study participants had low grade disease, approximating the 70% incidence of this patient population among all NMIBC patients. The other 3 study participants had high grade disease. In patients receiving bel-sar with light activation (n=8), 4 out of 5 patients with low grade disease demonstrated a clinical complete response with no tumor cells remaining on histopathological evaluation. 2 out of 3 patients with high grade disease demonstrated visual tumor shrinkage observed on cystoscopy."

P1 data • Bladder Cancer