Purespring's PS-002 receives EMA orphan drug status for primary IgA nephropathy treatment

Purespring Therapeutics, a forward-thinking company dedicated to revolutionizing kidney disease treatments, announced a significant milestone with its lead program, PS-002, receiving orphan drug designation from the European Medicines Agency (EMA) for IgA nephropathy (IgAN). This designation is a notable achievement as it provides critical incentives to drug developers targeting rare conditions, including market exclusivity, guidance on study design, and reductions in regulatory fees. The European Commission grants this status based on a favorable opinion from the EMA Committee for Orphan Medicinal Products.

IgAN is a prevalent autoimmune kidney disorder often affecting young adults. It occurs when immunoglobulin A (IgA) deposits accumulate in the kidney's glomeruli, causing inflammation and damage that can lead to scarring. Unfortunately, there is currently no cure or approved treatment to halt the disease's progression. A significant number of patients with high-risk IgAN face a decline in kidney function, eventually necessitating dialysis or a kidney transplant within five years.

Purespring’s Chief Executive Officer, Haseeb Ahmad, emphasized the importance of the orphan drug designation in advancing PS-002 through the necessary clinical development stages. This status not only aids in the progression of their lead program but also aligns with Purespring’s overarching mission to deliver innovative solutions for kidney diseases to patients. The company is eager to collaborate with the EMA to navigate the development pathway of PS-002, believing that this novel approach offers renewed hope to patients, particularly those with limited treatment options.

Building on a robust foundation supported by an oversubscribed £80 million ($105 million) Series B financing completed in October 2024, Purespring is actively moving PS-002 towards Phase I/II clinical trials for IgAN. The company is also working on additional gene therapy initiatives aimed at both rare and common kidney diseases. Notably, Purespring is the first to successfully address kidney disease models by targeting the podocyte, a specialized cell involved in about 60% of kidney ailments, using its proprietary adeno-associated viral (AAV) gene therapy platform.

At the American Society of Nephrology (ASN) Kidney Week 2024, Purespring showcased the potential of PS-002 as a pioneering modality in treating IgAN. The company, founded on the groundbreaking work of Professor Moin Saleem from the University of Bristol, focuses on therapies that target the podocyte. This approach enables efficient development of gene therapies for various renal diseases, offering potential not just to halt progression, but also to reverse or cure both rare and common kidney conditions.

Purespring's pipeline includes programs targeting IgAN and other kidney diseases mediated by the complement system, alongside treatments for disorders caused by mutations in the NPHS2 gene and other monogenic glomerular kidney diseases. Based in London, the Purespring team combines deep expertise in podocyte biology and kidney diseases with significant experience in gene therapy. This is underpinned by a culture of diversity, creativity, and a commitment to delivery.

Support from leading biotech investors such as Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and the British Business Bank has been instrumental to Purespring’s progress. The company has successfully raised £115 million ($149 million) to date, reflecting robust investor confidence and commitment to its mission of transforming kidney disease treatment.