Purespring Therapeutics announced today that the European Medicines Agency (EMA) has granted orphan drug designation to PS-002, the company's lead program for the treatment of IgA nephropathy (IgAN). This designation provides significant regulatory incentives to support the development of treatments for rare conditions affecting fewer than 5 in 10,000 people in the European Union.
The London-based company, focused on transforming kidney disease treatment, is now advancing PS-002 toward Phase I/II clinical trials following strong preclinical results presented at the American Society of Nephrology (ASN) Kidney Week 2024.
Understanding IgA Nephropathy and the Unmet Need
IgA nephropathy is a common chronic autoimmune kidney disease primarily affecting young adults. The condition occurs when immunoglobulin A (IgA) proteins become trapped in the kidney's glomeruli, causing inflammation, damage, and progressive scarring throughout the kidney.
Currently, there are no approved therapeutic options to halt disease progression or cure IgAN. Approximately one-third of high-risk patients lose kidney function within five years, ultimately requiring dialysis or kidney transplantation.
"By developing a novel approach to treat IgAN, we believe we can offer hope to patients across Europe and globally who currently have limited therapeutic options, furthering our mission to transform the lives of people suffering from kidney diseases," said Haseeb Ahmad, Purespring's Chief Executive Officer.
Pioneering Podocyte-Targeted Gene Therapy
Purespring's innovative approach makes it the first company to successfully treat kidney disease models by directly targeting podocytes, specialized cells implicated in approximately 60% of renal diseases. The company utilizes a proprietary adeno-associated viral (AAV) gene therapy platform to deliver therapeutic genes specifically to these cells.
The EMA orphan designation provides Purespring with several advantages, including market exclusivity, protocol assistance for study design and scientific evaluation, and regulatory fee reductions. These benefits will support the company's efforts to advance PS-002 through clinical development.
"Orphan drug designation will significantly support Purespring's goal of progressing PS-002 through clinical development and, more broadly, with bringing solutions for kidney disease indications to patients," Ahmad commented. "We're delighted that the EMA has recognised the promise of this potential breakthrough medicine and we look forward to working with them on the development pathway."
Robust Financial Backing and Pipeline Development
Purespring's development efforts are supported by an oversubscribed £80 million ($105 million) Series B financing round completed in October 2024. The company has raised a total of £115 million ($149 million) to date from leading biotech investors including Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and the British Business Bank.
Beyond PS-002 for IgAN, Purespring is developing additional gene therapy programs targeting both rare and common kidney diseases. The pipeline includes treatments for diseases caused by mutations in the NPHS2 gene and other monogenic glomerular kidney diseases.
Scientific Foundation and Leadership
Purespring was founded on the groundbreaking work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol. The company's platform approach enables streamlined gene therapy development for both acquired and genetic renal diseases, potentially offering treatments that could halt, reverse, or even cure various kidney conditions.
The EMA orphan drug designation represents a significant milestone in Purespring's mission to address the substantial unmet needs in kidney disease treatment, particularly for conditions like IgAN where therapeutic options remain severely limited.
As PS-002 progresses toward clinical trials, the company's innovative podocyte-targeting approach could potentially transform the treatment landscape for IgAN patients and establish a new paradigm for addressing kidney diseases more broadly.
