IgA protease

TOKYO, July 18, 2024 /PRNewswire/ -- Renalys Pharma, Inc. (Headquarters: Chuo-ku, Tokyo; "Renalys Pharma") announced today that the first person was dosed in its registrational Phase III clinical trial of sparsentan for the treatment of IgA nephropathy in Japan. In April this year Renalys submitted an Investigational New Drug (IND) Application for sparsentan to the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan. The registrational Phase III study is a multicenter, open-label, single arm study in Japanese patients with IgA nephropathy, and is planned to confirm the efficacy and safety of sparsentan in approximately 30 Japanese patients. Results from the urine protein/creatinine ratio (UP/C) endpoint in the study are expected in the second half of 2025 to support a submission for approval to PMDA. Sparsentan was developed by Travere Therapeutics, Inc. ("Travere"). Renalys Pharma has an exclusive license for development and commercialization of sparsentan in Japan, South Korea, Taiwan, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, the Philippines, Singapore, Thailand, and Vietnam. In 2023, Travere received FDA accelerated approval for sparsentan (U.S. brand name: FILSPARI®) to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression*. In addition, Travere submitted a supplemental New Drug Application to FDA in March 2024 which was granted as Priority Review and a PDUFA target action date of September 5, 2024, to convert the existing U.S. accelerated approval to full approval based on 2-year confirmatory results from the Phase III PROTECT Study, the only head-to-head study in IgA nephropathy versus an active comparator. In the PROTECT Study, FILSPARI® demonstrated a significant reduction in proteinuria, preservation of kidney function and a well-tolerated safety profile compared with active control irbesartan. Renalys Pharma is committed to advancing its business to contribute to improving the lives of patients in Japan and Asia through the development of innovative therapies that can improve the management of kidney disease, first and foremost by delivering sparsentan as soon as possible. *FILSPARI® (sparsentan) U.S. Indication: FILSPARI is an endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g. About IgA Nephropathy The management of patients with chronic kidney disease (CKD) and end-stage renal disease (ESRD) has become a social issue that places a heavy burden on the Japanese healthcare system. IgA nephropathy is widely known as one of the main causes of kidney failure and is considered to develop when abnormal IgA proteins are deposited in kidney tissue, inhibiting kidney function and causing inflammation. IgA nephropathy is a rare intractable disease (designated as an intractable disease 66) with many unresolved mechanisms of onset and severity. Currently, there is a lack of treatment for IgA nephropathy and an unmet need for such treatment in Japan. About Renalys Pharma, Inc. Renalys Pharma, a privately held late-stage clinical biopharmaceutical company based in Japan, is committed to the development of multiple innovative therapeutics targeting unmet needs in the management of renal disease for Japanese and Asian patients. Founded by Catalys Pacific and SR One in 2023, the company aims to address the growing problem of "drug loss" by catalyzing access to new treatments for kidney disease patients in Japan and other Asian regions. Company Name: Renalys Pharma, Inc. Address: 3-11, Nihonbashi-honcho 2-chome, Chuo-ku, Tokyo, Japan Representative: BT Slingsby, MD, PhD, MPH Established: April 2023 URL: [Disclaimer] Information concerning pharmaceutical products (compound under development) contained herein is not intended as advertising or as medical advice, but intended for disclosure of management information. SOURCE Renalys Pharma, Inc.

TOKYO, April 7, 2024 /PRNewswire/ -- Renalys Pharma, Inc. (Headquarters: Chuo-ku, Tokyo; "Renalys Pharma") announced that it has submitted an Investigational New Drug (IND) Application for sparsentan to the Pharmaceuticals and Medical Devices Agency (PMDA) for a Phase III clinical trial in Japan. The Phase III study is a multicenter, open-label, single arm study in Japanese patients with IgA nephropathy, and is planned to confirm the efficacy and safety of sparsentan in approximately 30 Japanese patients. First Patient In (FPI) is expected in the second half of 2024. Sparsentan was developed by Travere Therapeutics, Inc. ("Travere") and Renalys Pharma has an exclusive license for development and commercialization of sparsentan in Japan, South Korea, Taiwan, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, the Philippines, Singapore, Thailand, and Vietnam. In 2023, Travere received FDA accelerated approval for sparsentan (U.S. brand name: FILSPARI®) to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression*. In addition, Travere submitted a supplemental New Drug Application to FDA in March 2024, for conversion of the existing U.S. accelerated approval to full approval based on 2-year confirmatory results from the Phase III PROTECT Study, the only head-to-head study in IgA nephropathy versus an active comparator. In the PROTECT Study, FILSPARI® demonstrated a significant reduction in proteinuria, preservation of kidney function and a well-tolerated safety profile compared with active control irbesartan. Renalys Pharma is committed to advancing its business to contribute to improving the lives of patients in Japan and Asia through the development of innovative therapies that can improve the management of kidney disease, first and foremost by delivering sparsentan as soon as possible. About IgA Nephropathy The management of patients with chronic kidney disease (CKD) and end-stage renal disease (ESRD) has become a social issue that places a heavy burden on the Japanese healthcare system. IgA nephropathy is widely known as one of the main causes of kidney failure, and is considered to develop when abnormal IgA proteins are deposited in kidney tissue, inhibiting kidney function and causing inflammation. IgA nephropathy is a rare intractable disease (designated as an intractable disease 66) with many unresolved mechanisms of onset and severity. Currently, there is a lack of treatment for IgA nephropathy and an unmet need for such treatment in Japan. About Renalys Pharma, Inc. Renalys Pharma is a private, late-stage clinical biopharmaceutical company in Japan committed to the development of multiple innovative therapeutics that address unmet needs in the management of renal disease for Japanese and Asian patients. The company was established by Catalys Pacific and SR One to address the growing problem of "drug loss" by catalyzing an access to new treatments for kidney disease patients in Japan and other Asian territories. Company Name: Renalys Pharma, Inc. Address: 3-11, Nihonbashi-honcho 2-chome, Chuo-ku, Tokyo, Japan Representative: BT Slingsby, MD, PhD, MPH Established: April 2023 URL: [Disclaimer] Information concerning pharmaceutical products (compound under development) contained herein is not intended as advertising or as medical advice, but intended for disclosure of management information. SOURCE Renalys Pharma, Inc.

Selecta Biosciences "remains committed" to SEL-212, which Selecta still hopes to submit to the FDA for approval as a chronic refractory gout treatment next year. Three months after laying off a quarter of its workforce, Selecta Biosciences is now halting investment in all but one of its non-partnered programs to eke out enough cash into 2027. The company “remains committed” to its Swedish Orphan Biovitrum-partnered asset SEL-212, which Selecta still hopes to submit to the FDA for approval as a chronic refractory gout treatment next year. The drug is a formulation of the immunogenic enzyme pegadricase and biodegradable nanoparticles encapsulating the immunomodulator rapamycin that the biotech calls ImmTOR. It recently hit the primary endpoint in a pair of phase 3 trials that suggest potential to take on Horizon Therapeutics’ gout drug Krystexxa. The rest of Selecta’s suite of candidates won’t be so lucky, with the biotech announcing today that development will now be paused for a combination ImmTOR-IL program, which had been on track to begin preclinical studies later this year to set up the drug for entering the clinic. Selecta had been hoping to explore the drug’s potential in a variety of autoimmune indications, starting with the liver. “The company is currently assessing ways to support the development of this program through potential partnerships,” Selecta said in the release. Analysts had held out hope that Selecta would stick with the ImmTOR-IL program, with Mizuho’s Uy Ear suggesting back in May that prioritizing “ImmTOR-IL for T-cell mediated autoimmune liver diseases would provide multiple shots on goal.” However, Selecta will continue working on Xork, an IgG protease candidate that Astellas licensed in January to develop with the Japanese pharma’s gene theapy for Pompe disease. “Our actions today will allow us to preserve capital and maintain our stockholders’ interest in [SEL-212] without the dilution that would have been required to support the development of our pipeline assets over the long term,” CEO Carsten Brunn, Ph.D., said in the release. “While we believe that our pipeline programs represent great potential, we intend to pursue partnership opportunities to advance the balance of our portfolio and maximize their value.” The company had already sidelined SEL-302 in May, with Selecta reiterating today that it’s hoping to find a partner for the asset. The AAV gene therapy is in a phase 1/2 trial in combination with ImmTOR for the treatment of an inherited disorder called methylmalonic acidemia. At the time, the biotech also made the decision to lay off 25% of its workforce as part of cost-cutting measure to keep cash flowing into the second half of 2025. Selecta ended June with $115 million in cash and equivalents, which—combined with its latest pipeline refocus and money coming in from Sobi—it now expects to last through to 2027.