Alpine Immune Sciences, Inc.

Otsuka Pharmaceutical’s 2018 purchase of Visterra seems to be paying off after its anti-APRIL antibody passed a registrational test in a rare kidney disease. The IgA nephropathy (IgAN) antibody, named sibeprenlimab, hit the trial’s primary endpoint by achieving a “statistically significant and clinically meaningful” reduction in 24-hour urine protein-to-creatine ratio versus placebo at nine months, according to a Tuesday release . The study enrolled 530 people with IgAN who were taking standard of care. Otsuka said it plans to review the interim data with the FDA with an eye to accelerated approval. Bank of America Securities analysts said sibeprenlimab could become a first-in-class biologic for IgAN with approval possible as soon as the second half of next year. The Phase 3 trial is ongoing and expected to end in 2026. The company is tracking the study’s secondary endpoints like change in kidney function over 24 months. The Japanese company added sibeprenlimab to its pipeline as part of its $430 million buyout of Visterra in 2018. The monoclonal antibody works by blocking APRIL, a cytokine that plays a key role in B cell dysregulation and the overproduction of galactose-deficient IgA1. The APRIL target has been at the heart of several major deals, including Vertex’s $4.9 billion acquisition of Alpine Immune Sciences and its dual BAFF/APRIL antagonist povetacicept, which is set to enter Phase 3 development this year. Last year, Novartis spent $3.2 billion on Chinook Therapeutics and its pipeline of kidney drugs, including anti-APRIL antibody zigakibart. Otsuka has projected its drug could reach more than 100 billion Japanese Yen ($663 million) in peak sales. The BofA analysts’ expectations were even higher at around 505 billion Japanese Yen ($3.3 billion) in 2035. Jefferies analysts said cross-trial comparisons suggest sibeprenlimab has best-in-class potential, as well as more convenient dosing. IgAN affects around 390,000 people in the US, EU and Japan, the analysts added. It is the leading cause of kidney disease in young people.

An experimental medicine Otsuka Pharmaceutical acquired in a buyout six years ago has succeeded in a Phase 3 trial in a rare kidney disease, the company said Tuesday.The drug, called sibeprenlimab, met its main goal in a study in IgA nephropathy, or IgAN, a chronic condition that can lead to kidney failure. Upon a pre-specified, interim analysis after nine months of treatment, the company said sibeprenlimab therapy was associated with a statistically significant and clinically meaningful reduction, versus placebo, in the amount of protein in participants urine a marker of kidney health.Otsuka didnt provide specifics, making the magnitude of the drugs effects unclear. It also said, without details, that the drugs safety profile was consistent with previously reported data. But the company intends to discuss with the Food and Drug Administration the possibility of an accelerated approval filing while the study continues. The trial is expected to wrap up in early 2026.For Otsuka, the results are a measure of validation for its bet on Visterra, a Waltham, Massachusetts-based biotechnology startup and sibeprenlimabs developer. Visterra was founded in 2008 around a type of antibody drug discovery technology that originated at MIT. The company initially used that technology to develop drugs for infectious diseases before broadening its therapeutic scope, and tried, unsuccessfully, to go public in 2017.Otsuka stepped in a year later and acquired the company for $430 million in upfront cash. Visterra has since operated as a wholly owned subsidiary of Otsuka and has still been run by its longtime CEO, Brian Pereira. The company now has a pipeline of drugs mostly for immune diseases and led by sibeprenlimab, which, following Tuesdays study results, could become the first treatment of its kind for IgAN.Some estimates hold that upwards of 130,000 people in the U.S. have IgAN, a progressive autoimmune condition that damages the kidneys. While several therapies are available, there remains room for improvement, as patients with the disease still see their kidney function decline.The need for better therapies, along with a growing understanding of the diseases underlying biology, has made IgAN a focus of an increasing number of drug companies. Since 2023, Novartis, Biogenand Vertex Pharmaceuticals have all acquired biotechs with IgAN drugs in testing. Travere Therapeutics and Calliditas Therapeutics have recently launched IgAN medicines. Others, including Q32 Bio and Vera Therapeutics, are advancing would-be therapies as well.These emerging medicines are more commonly aimed at immune-mediated drivers of the disease, analysts at the investment bank Piper Sandler wrote in a recent report. Sibeprenlimab targets a protein dubbed APRIL thats implicated in the progression of IgAN. The drug Vertex acquired in a buyout of Alpine Immune Sciences aims at APRIL and a second protein called BAFF. Felzartamab, which Biogen acquired in its acquisition of HI-Bio, blocks the protein CD38.Otsukas trial results suggest that by targeting APRIL, we could provide a new therapeutic strategy for people living with this progressive kidney disease,“ said John Kraus, the executive vice president and chief medical officer of Otsukas pharmaceutical development and commercialization division, in a statement. '

Purespring Therapeutics has reeled in £80 million ($105 million) to test two gene therapies in humans. The Sofinnova Partners-led Series B, disclosed Wednesday morning, will carry the London startup into an open-label Phase 1/2 next year for a kidney disease that has attracted a lot of attention in recent years from regulators and pharmaceutical acquirers. The startup’s lead gene therapy, PS-002, will be investigated for IgA nephropathy, or IgAN. The autoimmune and kidney disease, in which too much IgA protein stays in the kidneys, can lead to end-stage renal disease. Purespring anticipates it could snag an accelerated approval from its upcoming Phase 1/2, CEO Julian Hanak told Endpoints News . Travere , Novartis and Calliditas have received FDA nods in the past few years for their oral medicines. During that approval process, Purespring would plan to launch a confirmatory Phase 3 to get full approval, Hanak said. To fund that, Purespring would have a few options: raise a crossover round, go through an IPO and/or secure a commercialization partnership with a pharma company, the CEO said. Purespring’s experimental gene therapy goes after a particular cell type known as the podocyte, which is key to a kidney’s ability to operate. Judo Bio, a new siRNA startup that emerged earlier this week with $100 million, also plans to target podocytes to directly treat renal diseases. Purespring’s candidate “actually could work in conjunction and in complementation with some of the new therapies coming on the market,” Hanak said. “It’ll be a one-and-done therapy because it’s gene therapy and because podocytes are totally differentiated, non-dividing cells. Once the gene therapy is in them, we’ll get persistence of expression.” There’s been a wave of M&A in the IgAN field, with Novartis , Vertex and Biogen each dishing out more than $1 billion to buy clinical-stage drug developers Chinook, Alpine Immune Sciences and Human Immunology Biosciences, respectively, in the past 15 months. “I think the industry has woken up to the fact that this is a huge unmet medical need,” Hanak said. He initially joined Purespring as chief development officer in 2021, coming from Biogen, where he served as CMC leader for the gene therapy work acquired from Nightstar . Purespring could go into “any other complement-mediated kidney disease affecting podocytes, so lupus nephritis, ANCA vasculitis and so on,” Hanak said, but the initial focus is on IgAN and an undisclosed second indication that will enter the clinic before the Series B runs out in a few years. The startup could also ink partnerships over the coming two years, the CEO said. “Companies have been approaching us about access to our platform to deliver their genes to the kidney,” he added. It has an internal process development manufacturing setup, Hanak said, noting the company also works with Charles River for plasmid DNA and with Columbus, OH-based Andelyn Biosciences for vector supplies. “We have to get the cost of goods for the product down, which we’ve been able to do,” Hanak said. “Our dose is very low, 50-to-100-fold lower than a traditional, systemic gene therapy because of our platform.” Purespring derived from the work of University of Bristol professor Moin Saleem, who works in pediatric renal medicine. The company launched with a £45 million Series A from Syncona in November 2020. Syncona returned for the Series B alongside Forbion. The respective UK and Dutch investors have paired up with other gene therapy companies before, including a $170 million round for ophthalmology-focused Beacon Therapeutics in July. Other Purespring investors include Gilde Healthcare and British Patient Capital. The company has 46 employees and doesn’t plan to rapidly hire. “We have all the juice we need to deliver,” Hanak said.