On January 17, 2025 Stamford reported positive results for its ASN-002-003 multi-lesional clinical trial (NCT 04416516. Protocol No ASN-002-003) evaluating SP-002, an Adenovirus-5 replication deficient vector encoding human Interferon- g in combination with vismodegib (a Hedgehog Pathway Inhibitor) in subjects presenting with mul/ple BCCs (Press release, Stamford Pharmaceuticals, JAN 17, 2025, View Source [SID1234649770]). The collected demographic data revealed subjects that were presenting with multiple BCCs had a history of multiple BCCs (and oLen a family history of multiple BCCs), thus representing a high-burden disease pa/ent group. 1 Three cohorts (cohorts 1, 2, 6) were evaluated in ASN-002-003. 2 Cohort 1 (1 target lesion) and Cohort 2 lesions (up to 3 target lesions)2 were both treated with 1.0e11vp/lesion and 4-weeks of vismodegib. 3 Cohort 6 (up to 3 target lesions) were treated with 1.5e11vp/lesion and 4-weeks of vismodegib. Cohort 1, 2 and 6 achieved a complete histological clearance rate (CHC) of 75%, 53% and 48% in the Intent to treat (ITT) population (46 lesions evaluated). A Histopathologic criteria4 predic/ve of poor response to SP-002 was first iden/fied in the ASN-002-001 (NCT02550678. Protocol No ASN-002-001) clinical study and was also evaluated in the current study (ASN-002-003). In ASN-002-003, all lesions lacking these histopathologic features predictive of poor response (n=23) across Cohorts 1, 2 and 6, achieved complete histological clearance, resul/ng in a CHC rate of 100%.

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The combination of SP-002 plus vismodegib was consistent with safety profiles of the individual agents with no new safety signals observed. Adverse events were generally mild to moderate in severity, no grade 4 or 5 events were observed. The observed safety profile for eligible subjects treated with weekly injections for local reactions was as follows: No Adverse Events (AEs): 15.2%; Grade 1 AEs: 34.8%; Grade 2 AEs: 47.8%; and Grade 3 AEs: 2.2%. Local AEs included swelling, erythema and ulceration/scabbing at the injection site.

Based on the study data, all participants experienced at least one treatment-emergent adverse event (TEAE), with 95.2% repor/ng events potentially related to vismodegib. Common systemic adverse events included muscle spasms (52.4%), nausea (19.0%), and fa?gue (14.3%), consistent with the known safety profile of the drug. While most AEs were mild to moderate, they were carefully monitored to ensure participant safety and provide insights into vismodegib’s tolerability.

"We are very pleased with these positive results from the Phase 2 study demonstrating the safety and efficacy of our gene therapy for patients with multiple nodular and superficial BCCs. We are particularly excited that the histopathologic criteria, which would allow responsive patients to be identified at screening and used for patient selection. This histopathologic criterion was first noted in the ASN-002-001 clinical study and has now been successfully reproduced in ASN-002-003. We believe this enables us to develop a useful treatment option for patients with nodular BCC and addresses the important need for a non-surgical option for those with BCCs in the H-zone or for those who are not ideal candidates for surgery." Dr Clement Leong (PhD), CEO of Stamford Pharmaceuticals.

"We are excited to have a novel approach that seeks to address the significant unmet needs in the treatment of basal cell carcinoma, particularly for patients managing multiple lesions. This innovative solution may hold promise for tumors located in the H-zone or other high-risk areas of the body, where effective and targeted non-surgical care options are critically important." Dr Sherrif F. Ibrahim, (MD, PhD), Rochester Dermatologic Surgery, P.C. "These results highlight the potential of this product to substantially enhance clearance rates compared to existing non-surgical treatment options for nodular BCC." Professor John Lear, MB ChB, MD, FRCP (UK), Mid Cheshire Hospitals NHS foundation trust, UK.

Analyses of the full Phase 2 dataset are ongoing and additional biomarker/translational research findings are to be presented at upcoming medical conferences. SP-002 has received Orphan Drug designations from the U.S. Food and Drug Administration (FDA). Stamford plans to discuss these findings with regulatory authorities in the coming months to prepare for a pivotal late-stage clinical study.