NEW YORK--(BUSINESS WIRE)--PIF Partners announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its proprietary investigational therapeutic 101-PGC-005 (‘005) for the treatment of systemic juvenile idiopathic arthritis (sJIA) flares. ‘005 is a Type IA prodrug of dexamethasone that targets CD206+ macrophages. Currently, ‘005 is in Phase 3 clinical trials in 9 centers in India for the treatment of ARDS induced by COVID-19, registered under CTRI/2024/01/061531.
“We are thrilled the FDA has recognized the potential of ‘005 to address the significant unmet medical needs of children suffering from sJIA,” said Alec Goldberg, CEO of PIF Partners. “This designation is a pivotal milestone that reinforces our commitment to developing innovative therapies for rare pediatric inflammatory diseases. By targeting activated macrophages with the most powerful anti-inflammatory compounds, we enhance efficacy and reduce toxicity. Critically, we have demonstrated no suppression of the HPA system, thus supplementing rather than replacing the natural anti-inflammatory response. We are eager to advance our clinical program and expand our research into other orphan diseases characterized by systemic inflammation.”
The FDA awards RPDDs to promote the development of new drugs and biologics for serious or life-threatening diseases that primarily affect children under 18 years old and impact fewer than 200,000 people in the United States. This designation makes PIF Partners eligible to receive a Priority Review Voucher (PRV) upon approval of ‘005. A PRV can be redeemed to obtain priority review for a subsequent marketing application, accelerating the review process from the standard 10 months to six months.
About sJIA
sJIA is a rare and severe autoinflammatory disease affecting children under 16. Unlike other forms of juvenile arthritis, sJIA is characterized by systemic symptoms such as high spiking fevers, rash, enlargement of the liver and spleen, and inflammation of the lining of the heart and lungs (serositis), in addition to persistent joint inflammation. sJIA accounts for approximately 10-20% of all cases of juvenile idiopathic arthritis.
The disease poses significant challenges, including chronic pain, growth retardation, joint damage, and potential life-threatening complications like macrophage activation syndrome (MAS). Current treatment options are limited and often involve long-term use of corticosteroids and immunosuppressive drugs, which can have serious side effects. There is a pressing need for new therapies that can effectively manage both the systemic and joint-related symptoms with improved safety profiles.
About PIF Partners
PIF Partners is a holding company with exclusive rights to ‘005 and other therapeutics using the underlying macrophage targeting platform. The company is seeking a partner to rapidly progress the development and commercialization of novel therapies for systemic juvenile idiopathic arthritis (sJIA) and other rare diseases characterized by systemic inflammation. Focused on addressing critical gaps in treatment, the company is advancing a pipeline of exclusively licensed therapeutic candidates targeting CD206+ macrophages. For more information, please visit pifpartners.com.
