How TolaSure Gel Targets Keratin Mutations in Epidermolysis Bullosa Simplex

Karen McGuire, PhD, chief executive officer and co-founder of BioMendics, recently spoke with Dermatology Times from the debra Care Conference in Los Angeles, California, about epidermolysis bullosa simplex (EBS) and the development of TolaSure Gel. McGuire explained what fast track status¹ means for the investigational treatment and detailed the drug's mechanism of action in EBS. She also distinguished TolaSure Gel from rapamycin-based approaches previously used in the clinic for EBS.

What Fast Track Designation Means for TolaSure Gel

Fast Track designation recognizes an unmet medical need in EBS, McGuire said, though it does not represent approval and does not always guarantee a shorter development timeline. The status provides a more efficient regulatory pathway, allowing more frequent interactions with the FDA as BioMendics advances its clinical manufacturing programs and addresses regulatory questions. McGuire noted the FDA's review of the fast track data package does not indicate the agency has concluded TolaSure Gel is effective, but does show support for continued expedited development.

"Fast track really shows the seriousness of EBS and the urgency for developing new treatments for patients," McGuire said.

For rare disease programs like EBS, McGuire said additional FDA interactions help confirm the company is asking the right questions and generating the right data. She said the interactions also help ensure the program moves as efficiently as possible toward the pivotal trial needed for approval.

How TolaSure Gel Targets Keratin Mutations in EBS

TolaSure Gel was designed to address the underlying biology of EBS rather than manage the consequences of blistering, McGuire said. The gel targets autosomal dominant mutations in keratin 5 and keratin 14, which produce misfolded keratins inside keratinocytes. Through inhibition of the mechanistic target of rapamycin (mTOR) and activation of autophagy, McGuire said TolaSure Gel strengthens intermediate filament networks and reduces skin blistering.

"What we're able to target with TolaSure is really these mutated, misfolded keratins that are being produced inside these keratinocytes, and we do this through inhibition of mTOR, the turning on of autophagy," McGuire said.

TolaSure Gel Versus Rapamycin-Based mTOR Inhibitors

TolaSure Gel is not a new version of sirolimus or rapamycin, which have previously been used in the clinic for EBS, McGuire said. She described TolaSure as a brand new small molecule chemical entity designed to overcome rapamycin's limitations, including poor skin penetration and immunosuppressive effects. Though both are associated with mTOR inhibition, McGuire said TolaSure and rapamycin are fundamentally different molecules with different pharmacologic properties.

"These are brand new small molecule chemical entities, specifically designed to also overcome some of rapamycin's problems, like skin penetration and some of the immunosuppressive effects of that drug," McGuire said.

TolaSure Gel remains an investigational drug, McGuire said, with safety and efficacy still under evaluation in the ongoing TAMES-02 phase 2 clinical study in EBS.²

References

1. BioMendics receives FDA Fast Track Designation for BM-3103 (TolaSure Gel) for epidermolysis bullosa simplex. News release. Business Wire. June 23, 2026. Accessed August 5, 2026. https://www.businesswire.com/news/home/20260623529098/en/BioMendics-Receives-FDA-Fast-Track-Designation-for-BM-3103-TolaSure-Gel-for-Epidermolysis-Bullosa-Simplex
2. About clinical trials. BioMendics. Accessed August 5, 2026. https://biomendics.com/index.php/patients-caregivers/about-clinical-trials