Paradigm Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing treatments for rare diseases, has announced the enrollment of the first patient in its Phase 3 clinical trial, SD-007. The study will evaluate SD-101 (Zorblesia™), an investigational topical cream designed to treat chronic wounds and skin lesions associated with epidermolysis bullosa (EB).
The trial marks an important milestone in the clinical development of SD-101 as the company continues to investigate potential treatment options for patients living with this rare genetic disorder.
Phase 3 SD-007 Trial to Evaluate SD-101's Efficacy and Safety
The SD-007 study is a multicenter, prospective, randomized, double-blind, placebo-controlled clinical trial designed to assess the efficacy and safety of SD-101 in patients with three types of epidermolysis bullosa:
-
EB simplex
-
Recessive dystrophic epidermolysis bullosa
-
Junctional epidermolysis bullosa
The study will evaluate several clinical outcomes, including the closure of selected chronic wounds, changes in the total body surface area affected by wounds and lesions, and improvements in itching.
Approximately 80 patients aged 28 days to 12 years are expected to participate in the trial. Recruitment will take place at 17 clinical centers across 14 countries in North America, South America, Europe, Asia, and Australia.
According to the study protocol, participants will apply SD-101 cream or the study comparator daily to their entire body for two months. Patients who complete SD-007 may also be eligible to participate in a subsequent study, SD-008, providing an opportunity for continued treatment.
Additional information about the clinical trial is available on the company's website at www.paradigmtherapeutics.com.
Paradigm Therapeutics Highlights Unmet Treatment Needs in EB
Robert Ryan, Ph.D., president and CEO of Paradigm Therapeutics, highlighted the considerable challenges faced by patients with epidermolysis bullosa, including skin blisters, chronic wounds, persistent pain, itching, infections, and scarring.
He noted that existing treatment options have limitations and that additional therapies are needed to address the widespread skin involvement associated with the condition.
According to Ryan, SD-101 is being developed as a potential alternative approach that targets sores and lesions across the skin surface in patients with EB simplex, recessive dystrophic EB, and junctional EB.
He also emphasized that findings from previously completed Phase II and Phase III clinical trials support further investigation of daily whole-body application. The ongoing development program will continue to evaluate the treatment's potential to promote targeted wound closure and reduce the total area of skin affected by lesions.
Paradigm Therapeutics aims to complete the development program and advance SD-101 toward potential regulatory approval. The company is also working with potential partners to support future development and global availability.
Eshelman Ventures Supports SD-101 Development
Dr. Fred Eshelman of Eshelman Ventures, LLC, expressed support for Paradigm Therapeutics and its efforts to advance SD-101 as a potential treatment for epidermolysis bullosa.
He highlighted the daily challenges experienced by patients and their families and emphasized the need for additional treatment options. Eshelman Ventures is investing in Paradigm Therapeutics to support the continued development of the investigational therapy.
Clinical Development Program for SD-101
SD-101 (Zorblesia™) is an investigational topical cream developed for daily application to the skin. The formulation can be stored at room temperature and does not require specialized clinical handling, which may help facilitate its use by patients and caregivers.
The treatment has previously been evaluated in completed Phase II and Phase III clinical trials involving a total of 217 pediatric and adult patients with EB simplex, recessive dystrophic EB, or junctional EB, including very young infants.
In these studies, participants applied SD-101 daily to their entire body for three months. The clinical development program assessed wound closure, changes in the extent of skin involvement, and treatment safety.
The ongoing SD-007 trial will further investigate the efficacy and safety of SD-101. Eligible participants who complete the study may be able to continue treatment through the subsequent SD-008 study.
SD-101 Receives Rare Pediatric Disease and Orphan Drug Designations
SD-101 has received Rare Pediatric Disease and Orphan Drug designations. These designations are intended to support the development of treatments for rare or serious diseases and do not constitute marketing authorization or guarantee regulatory approval.
Following regulatory approval, Paradigm Therapeutics will be eligible for a Priority Review Order (PRO), according to the company.
The company has selected Kapadi, a clinical research organization (CRO), to support the management of the global Phase 3 study.
Understanding Epidermolysis Bullosa
Epidermolysis bullosa is a group of rare genetic disorders characterized by fragile skin that blisters and tears easily following friction or minor trauma. Symptoms often appear at birth or during early childhood and may continue throughout a person's life.
Depending on the subtype and severity, the condition can cause:
-
Recurrent skin blisters and open wounds
-
Persistent pain and itching
-
Skin infections and scarring
-
Difficulties with mobility and daily activities
-
A substantial impact on patients' quality of life and that of their families
Severe forms of EB can lead to serious complications and may be life-threatening.
Although treatment options have advanced, significant unmet medical needs remain, particularly for therapies that address widespread skin wounds and lesions across different EB subtypes.
About Paradigm Therapeutics, Inc.
Paradigm Therapeutics, Inc. is a privately held, clinical-stage biopharmaceutical company focused on developing innovative therapies for rare diseases with significant unmet medical needs.
Its lead development program, SD-101 (Zorblesia™), is an investigational topical treatment designed to address the skin manifestations of epidermolysis bullosa. The company is continuing clinical development to further evaluate the treatment's efficacy and safety.
About Kapadi
Kapadi is a global clinical research organization specializing in clinical development in oncology and cell and gene therapy. With operations across North America, Europe, Asia, and Australia, the company supports biopharmaceutical sponsors in managing complex clinical programs through scientific expertise, regulatory capabilities, and technology-enabled operations.
About Eshelman Ventures, LLC
Eshelman Ventures, LLC is an investment fund founded by Dr. Fred Eshelman. It focuses on innovative companies in healthcare, life sciences, and biopharmaceuticals that aim to transform patient care and address significant unmet medical needs.
The enrollment of the first patient in Paradigm Therapeutics' Phase 3 SD-007 trial represents a milestone in the clinical development of SD-101 for epidermolysis bullosa. The study will further evaluate the investigational cream's safety and efficacy in pediatric patients with multiple EB subtypes. Its findings may help clarify the treatment's potential role in managing chronic wounds and skin lesions, although regulatory approval and clinical benefit remain to be established.
