Intralesional BO-112 met its main endpoint in the phase 2b SPOTLIGHT-204 trial, with 61% of patients with resectable basal cell carcinoma responding.
Intralesional BO-112, an investigational double-stranded RNA (dsRNA) nanoparticle immunotherapy, met its primary composite endpoint of visual and pathological response at Week 24 in the phase 2b SPOTLIGHT-204 trial in resectable primary basal cell carcinoma (BCC), according to topline data from Highlight Therapeutics presented as a late-breaking abstract at the
Surgical excision, including Mohs micrographic surgery, remains the recommended approach for most patients with BCC, though procedures on the head, neck, and face can leave lasting scarring or functional deficits. SPOTLIGHT-204 tested whether a short course of in-office injections before planned excision could clear lesions both visually and histologically.
Highlight Therapeutics described SPOTLIGHT-204 as the largest cohort of high-risk head and neck BCC treated with an intralesional agent to date.¹ M. Teresa Fernández-Figueras, MD, PhD, presented these new data during the EADV congress's late-breaking news session on September 30, 2026.²
SPOTLIGHT-204 (NCT06422936) is a multicenter, single-arm study enrolling 50 patients with resectable primary BCC: 40 with high-risk disease, including 36 with head and neck lesions, and 10 with low-risk disease. Participants received 3 once-per-week intralesional BO-112 injections, followed by complete surgical excision at the 24-week mark. Median age was noted as 75 years (range, 34-90 years), and 48% of subjects were reported to be female.¹
A central review committee assessed the primary endpoint, a composite of visual and pathological response (grade 3 or better) across all treated lesions. In the modified intention-to-treat population of 46 patients with 54 evaluable lesions, 61% (28/46) reached this end point.¹ The company described strong agreement between visual and histologic assessments.
Response rates held across both risk strata, at 58% (21/36) among high-risk patients and 70% (7/10) among those deemed low-risk.¹
Complete visual and complete pathological response, each scored as grade 1, were observed in 56% (18/32) of high-risk individuals with head and neck lesions. Among those with facial lesions, the complete response rate reached 62% (16/26).¹
Josep Malvehy, MD, PhD, director of the skin cancer program at Hospital Clínic of Barcelona, pointed to this population as the 1 with the greatest unmet need for nonsurgical options in the company's announcement. He noted these patients are among the most difficult to manage surgically and often express the most concern about treatment-related changes in appearance and function. Malvehy further described the results as supporting BO-112 as a well-tolerated, nonsurgical option for this group.¹
No serious adverse events, grade 3 or higher treatment-related adverse events (TRAEs), or treatment discontinuations were reported. Most TRAEs were mild, with 91% classified as grade 1 and 88% resolving within 3 days. The trial also met its key secondary end points, which included safety, tolerability, and response assessed separately by investigators and by central review.¹
BO-112 is designed to engage innate antiviral sensing pathways and induce immunogenic tumor cell death, with subsequent development of tumor-specific adaptive immunity. The company positions the agent as a way to convert immunologically "cold" tumors into immune-visible ones. It describes BO-112 as a first-in-class, multitarget agent activating both innate and adaptive immunity.¹
Highlight Therapeutics plans to advance BO-112 into late-stage development for localized primary BCC. The agent has not been approved by any regulatory authority.¹
The company also cautioned these topline figures reflect a preliminary analysis as of the data cutoff and could change after full analysis. The release did not specify the design or timing of a registrational trial.¹
Editor's Note: This summary has been edited for grammar and clarity using artificial intelligence tools.
References
