Latest news with #PurespringTherapeutics
Yahoo
6 days ago
- Business
- Yahoo
Purespring Therapeutics receives UK CTA approval for Phase I/II clinical trial of PS-002 in patients with primary IgA nephropathy (IgAN)
First patient in Phase I/II clinical trial expected to be enrolled in Q4 2025 UK Clinical Trial Application (CTA) approval shortly follows U.S. IND clearance and granting of European Medicine Agency (EMA) orphan drug designation, enabling readiness for initiation across sites in both the U.S. and Europe PS-002, Purespring's lead precision nephrology programme, targets the complement pathway known to be a driver of IgA nephropathy and is supported by a wealth of preclinical data London – 5 August 2025 - Purespring Therapeutics, a precision nephrology company focused on transforming the treatment of kidney diseases, today announces that its UK Clinical Trial Application (CTA) for a planned Phase I/II study of PS-002, Purespring's lead programme, in patients with IgA nephropathy (IgAN) has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA), the NHS Health Research Authority (HRA) and Research Ethics Committee (REC). 'The CTA approval for our Phase I/II clinical trial of PS-002 represents another key milestone as we complete our transition to a clinical-stage precision nephrology company,' said Haseeb Ahmad, Purespring's Chief Executive Officer. 'Building on the recent FDA IND clearance and EMA orphan drug designation, this further validates the potential of our podocyte-targeting approach to go beyond symptom management and directly target kidney disease at its source. Looking ahead, we are committed to working closely with regulators and sites across the U.S. and Europe with the view to expand the therapeutic options available for people living with IgAN.' PS-002 was developed to target the underlying cause of many kidney diseases by modulating complement activation in the kidney via precision targeting of podocytes. The programme is initially focused on the treatment of IgA nephropathy (IgAN), a rare and chronic autoimmune kidney disease that primarily affects young adults. In IgAN, aberrant immunoglobin A (IgA) protein becomes trapped in the kidney's filters, known as the glomeruli, causing complement activation, inflammation, damage and scarring. A significant proportion of affected patients will go on to develop kidney failure despite currently available therapies. The Phase I/II clinical trial, which is expected to enroll its first patient in Q4 2025, will evaluate local administration of PS-002 to treat IgAN. In the Phase 1 part of the Phase I/II study, the main read-outs will be safety parameters, which, together with efficacy biomarkers, will be leveraged to select a dose for the Phase 2 part of the study. This second phase will be used to further define the safety profile and provide early markers of efficacy. Enabled by this latest regulatory approval and the recent U.S. IND clearance, as announced in July 2025, the Phase I/II study will recruit patients across the U.S. and Europe. For further information, contact: Purespring: Peter Mulcahycontact@ (0)20 3855 6324LinkedIn ICR Healthcare Amber Fennell, Sarah Elton-Farrpurespring@ Notes to Editors About Purespring Purespring is developing therapies to halt or prevent kidney disease, one of humankind's most poorly treated disease areas. Founded on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, Purespring is the first company to successfully treat kidney disease by targeting the podocyte, a specialised cell that is implicated in the majority of renal disease. Purespring's platform approach enables streamlined gene therapy development for both acquired and genetic renal diseases, offering the potential to halt, reverse and even cure both rare and common kidney diseases. The Company currently has a pipeline of programmes in development including the lead asset for treatment of IgA Nephropathy (IgAN) and other complement mediated kidney disease. The Company also has programmes for disease caused by mutations in the gene NPHS2, as well as other monogenic glomerular kidney diseases. Based in London, the Purespring team combines world-leading expertise in podocyte biology and kidney disease with a wealth of experience in gene therapies, anchored in a culture of diversity, creativity and delivery. Purespring is backed by leading biotech investors, including Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and the British Business Bank and has raised £115m ($149m) to date. For more information please visit: and follow us on in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
6 days ago
- Business
- Yahoo
Purespring Therapeutics receives UK CTA approval for Phase I/II clinical trial of PS-002 in patients with primary IgA nephropathy (IgAN)
First patient in Phase I/II clinical trial expected to be enrolled in Q4 2025 UK Clinical Trial Application (CTA) approval shortly follows U.S. IND clearance and granting of European Medicine Agency (EMA) orphan drug designation, enabling readiness for initiation across sites in both the U.S. and Europe PS-002, Purespring's lead precision nephrology programme, targets the complement pathway known to be a driver of IgA nephropathy and is supported by a wealth of preclinical data London – 5 August 2025 - Purespring Therapeutics, a precision nephrology company focused on transforming the treatment of kidney diseases, today announces that its UK Clinical Trial Application (CTA) for a planned Phase I/II study of PS-002, Purespring's lead programme, in patients with IgA nephropathy (IgAN) has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA), the NHS Health Research Authority (HRA) and Research Ethics Committee (REC). 'The CTA approval for our Phase I/II clinical trial of PS-002 represents another key milestone as we complete our transition to a clinical-stage precision nephrology company,' said Haseeb Ahmad, Purespring's Chief Executive Officer. 'Building on the recent FDA IND clearance and EMA orphan drug designation, this further validates the potential of our podocyte-targeting approach to go beyond symptom management and directly target kidney disease at its source. Looking ahead, we are committed to working closely with regulators and sites across the U.S. and Europe with the view to expand the therapeutic options available for people living with IgAN.' PS-002 was developed to target the underlying cause of many kidney diseases by modulating complement activation in the kidney via precision targeting of podocytes. The programme is initially focused on the treatment of IgA nephropathy (IgAN), a rare and chronic autoimmune kidney disease that primarily affects young adults. In IgAN, aberrant immunoglobin A (IgA) protein becomes trapped in the kidney's filters, known as the glomeruli, causing complement activation, inflammation, damage and scarring. A significant proportion of affected patients will go on to develop kidney failure despite currently available therapies. The Phase I/II clinical trial, which is expected to enroll its first patient in Q4 2025, will evaluate local administration of PS-002 to treat IgAN. In the Phase 1 part of the Phase I/II study, the main read-outs will be safety parameters, which, together with efficacy biomarkers, will be leveraged to select a dose for the Phase 2 part of the study. This second phase will be used to further define the safety profile and provide early markers of efficacy. Enabled by this latest regulatory approval and the recent U.S. IND clearance, as announced in July 2025, the Phase I/II study will recruit patients across the U.S. and Europe. For further information, contact: Purespring: Peter Mulcahycontact@ (0)20 3855 6324LinkedIn ICR Healthcare Amber Fennell, Sarah Elton-Farrpurespring@ Notes to Editors About Purespring Purespring is developing therapies to halt or prevent kidney disease, one of humankind's most poorly treated disease areas. Founded on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, Purespring is the first company to successfully treat kidney disease by targeting the podocyte, a specialised cell that is implicated in the majority of renal disease. Purespring's platform approach enables streamlined gene therapy development for both acquired and genetic renal diseases, offering the potential to halt, reverse and even cure both rare and common kidney diseases. The Company currently has a pipeline of programmes in development including the lead asset for treatment of IgA Nephropathy (IgAN) and other complement mediated kidney disease. The Company also has programmes for disease caused by mutations in the gene NPHS2, as well as other monogenic glomerular kidney diseases. Based in London, the Purespring team combines world-leading expertise in podocyte biology and kidney disease with a wealth of experience in gene therapies, anchored in a culture of diversity, creativity and delivery. Purespring is backed by leading biotech investors, including Syncona Limited, Sofinnova Partners, Gilde Healthcare, Forbion, and the British Business Bank and has raised £115m ($149m) to date. For more information please visit: and follow us on in to access your portfolio
Yahoo
28-04-2025
- Health
- Yahoo
Purespring Therapeutics granted European Medicines Agency (EMA) orphan drug designation for PS-002 for the treatment of patients with primary IgA nephropathy (IgAN)
Purespring Therapeutics EMA designation provides orphan status to medicines being developed for rare conditions that are intended for the diagnosis, prevention or treatment of diseases affecting fewer than 5 in 10,000 people in the European Union Purespring has previously demonstrated the potential of PS-002, the Company's lead programme, as an important novel targeted treatment for primary IgAN PS-002 is progressing towards a Phase I/II clinical trial, supported by a strong preclinical data package London – 28 April 2025 - Purespring Therapeutics, a pioneering company focused on transforming the treatment of kidney diseases, today announces that the European Medicines Agency (EMA) has granted orphan drug designation to PS-002, Purespring's lead programme, in IgA nephropathy (IgAN). Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion adopted by the EMA Committee for Orphan Medicinal Products. Orphan designation provides incentives to developers of medicines for limited patient populations, including market exclusivity, protocol assistance (guidance on study design and scientific evaluation) and regulatory fee reductions. IgAN is a common, chronic autoimmune kidney disease that primarily affects young adults. In patients with the disease, a protein called immunoglobulin A (IgA) becomes trapped in the filters of the kidney, known as glomeruli, causing inflammation, damage and scarring to the whole kidney. There are currently no approved therapeutic options to stop disease progression, nor a cure, and about one third of high risk IgAN patients will go on to lose kidney function within five years and require a kidney transplant or dialysis. Haseeb Ahmad, Purespring's Chief Executive Officer, commented: '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. 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. '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.' Supported by an oversubscribed £80 million / $105 million Series B financing, which completed in October 2024, Purespring is progressing PS-002 towards a Phase I/II clinical trial for IgAN, with additional gene therapy programmes in development for the treatment of both rare and common kidney diseases.
