BioMendics, a clinical-stage biopharmaceutical company focused on rare skin diseases, has launched the pivotal TAMES-02 clinical trial to evaluate TolaSure, a first-in-class topical therapy for patients with generalized intermediate to severe Epidermolysis Bullosa Simplex (EB Simplex). The multicenter, randomized, double-blind, placebo-controlled study represents a significant advancement in addressing a rare inherited skin disorder that affects an estimated 30,000 to 50,000 individuals worldwide.
Addressing an Unmet Medical Need
EB Simplex is a rare, inherited blistering disorder characterized by skin fragility and recurrent, painful blister formation, particularly on the hands and feet. The condition causes painful blistering due to fragile skin, leaving patients and families facing a lifelong battle without disease-modifying options. Current treatment has been limited to supportive wound care and infection control, creating a significant therapeutic gap for affected patients.
TolaSure aims to change this reality by targeting the mutant keratin proteins responsible for structural instability in the skin's epidermis. By reinforcing fragile keratinocytes, the therapy offers a novel mechanism that directly addresses the root cause of blister formation rather than merely managing symptoms.
"Our mission is to bring meaningful therapies to patients living with rare diseases like EB Simplex—conditions that have been overlooked for too long," said Dr. Karen M. McGuire, CEO and Founder of BioMendics. "With TolaSure, we're targeting the root cause of blistering by reinforcing fragile keratinocytes. The TAMES-02 trial is a critical step toward proving safety and efficacy."
Trial Design and Academic Partnerships
TAMES-02 is being conducted in partnership with Stanford University School of Medicine and Northwestern University Feinberg School of Medicine, both globally recognized leaders in dermatology research. The trial will evaluate TolaSure in patients aged 4 and older and includes a comprehensive study design with multiple phases.
The study consists of a 2-month double-blind, placebo-controlled phase, followed by a 2-month open-label phase where all participants receive TolaSure, and a 6-month follow-up period. Importantly, the trial allows patients to treat plantar (foot) blistering—a major source of pain and disability for those living with EB Simplex.
Dr. Joyce Teng, Principal Investigator at Stanford University, emphasized the collaborative nature of the research: "Our early studies with TolaSure showed meaningful reduction in blistering, which is incredibly encouraging for the EB Simplex community. Clinical research in rare skin diseases requires collaboration with companies like BioMendics and patient advocacy groups like debra of America to accelerate progress toward transformative therapies."
Building on Previous Success
The TAMES-02 trial builds on the success of the earlier TAMES-01 study, which demonstrated TolaSure's safety and efficacy in adult patients. The early data showed meaningful reduction in blistering, providing the foundation for this expanded clinical program.
Dr. Amy Paller, Principal Investigator at Northwestern University, added: "TAMES-02 is a vital next step. The collaboration with BioMendics and Stanford enables us to rigorously evaluate this therapy and, hopefully, bring real progress to patients who have had limited options for far too long."
Ensuring Broad Access
Recognizing the geographic and logistical barriers facing many rare disease patients, BioMendics has implemented strategies to ensure broad trial access. The company is covering travel costs to bring patients from across the United States and internationally to participate in the study.
"We're really excited to be able to cover travel cost to bring patients in from all over the United States and internationally," said Dr. Aleesha McCormick, Senior clinical research scientist at BioMendics. "EB Simplex patients don't all live close to an Academic Medical Center like Stanford and Northwestern and our goal is to help as many patients be able to participate in the study as possible."
The study design also allows recruitment of slightly less severe patients who may not have experienced sufficient disease flares to be eligible for other clinical studies, potentially expanding access to earlier intervention in the disease course.
Future Therapeutic Potential
Beyond EB Simplex, BioMendics sees strong potential for TolaSure in other rare keratin disorders such as pachyonychia congenita and epidermolytic ichthyosis. If successful, the company plans to seek strategic partnerships to help accelerate development and deliver this treatment to patients as quickly as possible.
The TAMES-02 trial reflects BioMendics' ongoing commitment to developing innovative, targeted treatments for rare skin disorders and is currently open for patient enrollment. As the trial progresses, the dermatology community will be monitoring results that could signal the arrival of the first disease-modifying treatment for EB Simplex, offering renewed hope for patients long underserved by traditional dermatologic therapies.
