Outlining the Clinical Development Strategy of TolaSure for EB Simplex

“That pivotal trial is really ultimately our goal...We're really trying to get this on the market as quickly as we can for patients,” Karen McGuire, PhD, CEO and co-founder of BioMendics, said in the second part of this conversation with Dermatology Times.

McGuire outlined the design and objectives of the ongoing TAMES-02 clinical trial evaluating TolaSure in patients with epidermolysis bullosa simplex (EBS), as well as the company’s broader clinical development strategy. Building on encouraging results from the TAMES-01 study, TAMES-02 is designed as a randomized, placebo-controlled phase 2 trial with blister surface area selected as the primary efficacy endpoint.

Unlike its predecessor, which relied on internal controls, TAMES-02 enrolls patients into 2 parallel groups receiving either active treatment or placebo. Importantly, the study incorporates a crossover design, allowing patients initially assigned to placebo to transition to active therapy after 2 months. This approach ensures that all participants have access to the investigational drug while preserving the rigor needed to assess treatment effect. Investigators aim to replicate the rapid and substantial reductions in blister burden observed in TAMES-01, reinforcing blistering as the key driver of disease severity in EBS.

The trial plans to enroll 40 patients, with an interim analysis scheduled after approximately 20 participants have completed treatment. If outcomes remain consistent with earlier findings, BioMendics intends to pursue breakthrough therapy designation and, pending regulatory alignment, advance directly into a pivotal phase 3 program. TolaSure has already received orphan drug and rare pediatric disease designations.

Beyond EBS, McGuire highlighted plans to expand TolaSure’s application to other rare keratinopathies. These include epidermolytic ichthyosis, driven by keratin 1 and keratin 10 mutations, and pachyonychia congenita, associated with keratin 6 and keratin 16 variants. Preclinical and early clinical work suggest that TolaSure’s autophagy-mediated mechanism of action is consistent across these disorders, supporting its potential as a platform therapy for multiple inherited diseases of keratin dysfunction.

TAMES-02 is actively recruiting at major centers of excellence, including Stanford University and Northwestern University, under the leadership of Joyce Teng, MD, PhD, and Amy Paller, MD. To minimize barriers to participation, BioMendics covers all travel-related expenses for enrolled patients. The company is also seeking engagement from dermatologists who care for larger EBS populations and may be interested in participating in future phase 3 studies.

To learn more, visit the recruitment page.