Paradigm Therapeutics, Inc. has secured a $12.5 million investment from Eshelman Ventures, LLC to advance SD-101 (Zorblisa), a novel topical therapy developed for treating Epidermolysis Bullosa (EB). The funding will support completion of all activities required for a planned New Drug Application (NDA) submission in the second half of 2025, as well as preparations for U.S. commercialization.
As part of the agreement, Dr. Eshelman, founder of Eshelman Ventures, will join Paradigm as a strategic advisor. The investment marks a significant milestone in bringing the first topical therapy designed to treat wounds and lesions across the entire skin surface for all EB subtypes to market.
"We are excited about the opportunity to complete the development activities for SD-101 (Zorblisa) and to move a path forward towards registration of this therapy for patients with all EB subtypes," said Robert Ryan, Ph.D., Chief Executive Officer of Paradigm Therapeutics. "There is a tremendous need for novel whole skin surface treatment options for treating the debilitating skin effects across all patients with Epidermolysis Bullosa."
Understanding Epidermolysis Bullosa
Epidermolysis Bullosa is a rare genetic disorder characterized by extremely fragile skin that blisters and tears easily, often beginning at birth. The condition can be chronic, potentially disfiguring, and in some cases fatal. Currently, there are no approved therapies specifically developed to treat the entire skin surface across all EB subtypes.
The disease manifests in several genetic and symptomatic variations, but all forms share the common symptom of skin that blisters and tears, sometimes from minimal friction or trauma. This creates significant challenges for patients, particularly children, who must cope with painful wounds throughout their lives.
SD-101 (Zorblisa): A Breakthrough Approach
SD-101 is a topical cream that has shown potential to improve the severe skin disruptions experienced by patients across all EB subtypes. The therapy has completed both Phase II and Phase III clinical trials, which included a total of 217 patients with Simplex, Recessive Dystrophic (RDEB), or Junctional EB.
In these studies, SD-101 was applied topically over the entire body daily in both adults and children as young as 21 days old for a three-month period. The primary outcome measurement was assessment of target wound closure, with secondary measurements including reduction in body surface area (BSA) coverage of blisters and wounds.
"Epidermolysis Bullosa is a devastating disease," Dr. Ryan emphasized. "We believe the clinical data show beneficial effects of the use of SD-101 across the range of EB patients, and strongly support progression towards registration. Results from the completed Phase II and Phase III trials indicate that SD-101 demonstrated a broad benefit with whole body treatment of the lesions and wounds on the entire skin surface in pediatric and adult patients across all EB patients, who have limited whole body treatment alternatives."
The clinical data also demonstrated that SD-101 maintains a favorable safety profile when used long-term, an important consideration for a chronic condition requiring ongoing treatment.
Regulatory Status and Future Plans
SD-101 has received several important regulatory designations that highlight its potential significance for EB patients:
- First EB treatment to receive the FDA's Breakthrough Therapy designation
- Rare Pediatric Disease designation from the U.S. Food and Drug Administration
- Orphan Drug designation from both the FDA and the European Commission
Upon approval, Paradigm will be eligible to receive a Priority Review Voucher (PRV), which could provide additional value to the company.
"Paradigm's goal is to provide this therapy to patients on a global basis and is working in parallel with potential partners to work towards this goal as quickly as possible," Dr. Ryan stated.
Strategic Partnership with Eshelman Ventures
The investment from Eshelman Ventures represents more than just financial support. Dr. Eshelman's appointment as a strategic advisor brings valuable pharmaceutical development and commercialization expertise to Paradigm.
"Eshelman Ventures is honored to invest in Paradigm Therapeutics and play a pivotal role in accelerating the development of SD-101 as a transformative treatment for Epidermolysis Bullosa," said Dr. Eshelman. "As an investor and strategic advisor, I am deeply committed to working closely with this exceptional team as we work tirelessly to bring this innovative therapy to patients in need."
Dr. Eshelman further emphasized the urgency driving this partnership: "The urgency of addressing the challenges faced by EB patients is what drives us; our goal is to provide an improved quality of life in this area of unmet need. Together, we will strive to revolutionize treatment options and make a meaningful impact on the lives of those affected by this debilitating disease."
Practical Advantages of SD-101
One of the notable advantages of SD-101 is its practical formulation. The topical cream requires no special handling and can be stored at room temperature, making it potentially more accessible for patients and caregivers managing this challenging condition.
With the new funding secured, Paradigm Therapeutics is positioned to complete the final steps needed for regulatory submission and potential approval of what could be a significant advancement in the treatment landscape for Epidermolysis Bullosa patients worldwide.
