#USA #Epidermolysis Bullosa #Paradigm Therapeutics #Zorblisa #Mt Pleasant

Paradigm Therapeutics Secures Investment for Innovative Skin Therapy

Paradigm Therapeutics, Inc., a clinical-stage biopharmaceutical company, has recently announced a significant financial boost in the form of a $12.5 million investment from Eshelman Ventures, LLC. This financing aims to propel the development of SD-101, also known as Zorblisa™. Zorblisa™ is a pioneering topical therapy designed to treat the extensive skin surface of patients suffering from Epidermolysis Bullosa (EB), a rare and debilitating genetic disorder.

Investment Details and Strategic Advisory

The announcement comes alongside the appointment of Dr. Eshelman as a strategic advisor to Paradigm Therapeutics. The funding is earmarked for completing critical pre-commercialization activities and fulfilling requirements for a New Drug Application (NDA) anticipated in late 2025. Robert Ryan, Ph.D., CEO of Paradigm Therapeutics, expressed enthusiasm about the prospects this funding brings in advancing Zorblisa™ towards regulatory approval.

“We are excited about the opportunity to complete the development of SD-101,” Dr. Ryan stated, emphasizing the pressing need for comprehensive skin therapies for all patients suffering from Epidermolysis Bullosa. The company is fervently dedicated to creating a new therapeutic alternative for this patient demographic that currently faces limited treatment options.

Understanding Epidermolysis Bullosa

Epidermolysis Bullosa is a rare, genetic skin disorder characterized by extreme fragility of the skin, leading to the formation of blisters and open wounds, often from birth. Patients experience chronic pain and require extensive care as traditional treatment options do not adequately address the systemic issues related to the skin's fragility. The chronic nature of EB can lead to disfigurement and in severe cases, fatal complications.

Zorblisa™ has been developed in response to this dire medical need, offering hope for improved management of skin wounds across all subtypes of EB. Treatment with Zorblisa™ aims to alleviate the debilitating symptoms associated with the disease and provide a foundation for enhanced quality of life for patients.

Clinical Trials and Efficacy

Clinical trials for Zorblisa™ (SD-101) have demonstrated significant promise. Conducted across Phase II and Phase III stages, these trials included a substantial sample of 217 patients diagnosed with various forms of EB, including simple, recessive dystrophic areolar, and junctional EB. Initial results indicate that Zorblisa™ effectively promotes wound closure and significantly reduces the total skin surface area affected by blisters and injuries.

Zorblisa™ has distinguished itself as the first EB treatment to receive the FDA's Innovative Therapy Designation due to its groundbreaking approach. Furthermore, it has been recognized by both the FDA and the European Commission as an orphan drug, opening pathways for accelerated review processes which can ultimately bring this treatment to the market much sooner.

Moving Forward

Dr. Eshelman remarked on the critical nature of this investment, noting, “As an investor and strategic advisor, I am fully committed to collaborating with the Paradigm team to ensure this innovative therapy reaches the patients who urgently need it.” He highlighted the broader goal of addressing the unmet medical needs of EB patients and enhancing their quality of life through transformative medical solutions.

Paradigm Therapeutics remains focused on delivering Zorblisa™ to a global audience, intensifying efforts to partner with organizations that can aid in its swift rollout post-approval. The urgency of treating EB’s challenging symptoms drives their mission, with a dedicated team striving to implement significant changes in treatment dynamics for the disease.

In conclusion, the successful development and impending commercialization of SD-101 (Zorblisa™) stand to redefine standards of care for individuals battling the hardships of Epidermolysis Bullosa, setting a precedent for innovative therapeutic approaches in rare disease management.