Imlifidase

Imlifidase successfully met primary endpoint in pivotal US Phase 3 ConfIdeS trial in kidney transplantation. First clinical data supports imlifidase ability to substantially reduce anti-AAV antibodies to allow administration of gene therapy. LUND, Sweden, Oct. 30, 2025 /PRNewswire/ -- Hansa Biopharma AB, "Hansa" (Nasdaq Stockholm: HNSA), today announced its interim report for January-September 2025. Renée Aguiar-Lucander, CEO, Hansa Biopharma said, "A key milestone in Q3 was the highly statistically significant outcome of the ConfIdeS trial, where imlifidase met its primary endpoint of eGFR at 12 months in kidney transplantation. This marks the first time imlifidase has been evaluated in a randomized and controlled trial, and we are thrilled with the results. The data clearly demonstrate a statistically significant (p < 0.0001) and clinically meaningful benefit for the highly sensitized patients in the imlifidase arm, underscoring the urgent need for effective desensitization therapies. These findings offer real hope to patients who currently face years on dialysis and limited transplant options. Based on the strength of the data, we are targeting a Biologics License Application (BLA) submission to the FDA under the accelerated approval pathway before year-end 2025, and we intend to request Priority Review. Two gene therapy trials, one sponsored by Sarepta and the other sponsored by Genethon, the latter reported as a subsequent event, support the ability of imlifidase to substantially reduce anti-AAV antibodies to allow administration of gene therapy, which is very exciting." Financial Performance Year to date IDEFIRIX product sales totalled 143.6 MSEK and represented an increase of 29.1 MSEK or 25.4% compared to the same period a year ago (114.5 MSEK). Year to date product sales represent approximately 102% of total full-year product sales achieved in 2024. Product sales in the third quarter of 2025 totalled 30.1 MSEK, compared to 39.8 MSEK for the same period in 2024. Lower sales numbers in the third quarter are largely driven by fewer than expected kidney transplants during the summer period, the pause in the German Eurotransplant Prioritized Program and reimbursement challenges in Catalunya, one of the largest regions in Spain. Several corporate initiatives will be rolled out during Q4 to capture the existing market potential and support enhanced growth in 2026 and beyond Pipeline Progress U.S. ConfIdeS trial: Imlifidase successfully achieved its primary endpoint in the U.S. Phase 3 pivotal ConfIdeS study in kidney transplantation. At 12 months, mean eGFR was 51.5 mL/min/1.73m2 in the imlifidase arm compared to 19.3 mL/min/1.73m2 in the control arm demonstrating a statistically significant and clinically meaningful difference of 32.2 mL/min/1.73m2 (p<0.0001). Imlifidase was generally well tolerated with a safety profile consistent with previous clinical trial experience. Hansa plans to file a BLA submission with the FDA before year-end 2025. SRP-9001-104 in Duchenne muscular dystrophy (DMD): Topline results were reported for three patients with DMD treated with imlifidase prior to administration of Sarepta's ELEVIDYS in the trial. After a single dose of imlifidase, patients experienced a rapid reduction of IgG antibodies, to levels ≥95% less than baseline. In addition, pre-existing anti-AAV antibodies were reduced below a titre of 1:400, enabling treatment with ELEVIDYS. No new safety signals were observed in the trial. These first clinical data support the ability of imlifidase to substantially reduce anti-AAV antibodies to allow administration of gene therapies. Financial Summary Conference Call Details Hansa Biopharma will host a telephone conference today Thursday, 30 October 2025, at 13:00 CET / 8:00 am EDT. The event will be hosted by Renée Aguiar-Lucander, CEO, Richard Philipson, CMO, Evan Ballantyne, CFO, and Maria Törnsén COO and President U.S. The call will be held in English. Slides used in the presentation will be live on the company website during the call under Financial reports | Hansa Biopharma and will also be made available online after the call. To participate in the telephone conference, please use the dial-in details provided below: Participant Dial In (Toll Free): 1-833-821-3542 Participant International Dial In: 1-412-652-1248 *Please ask to be joined into the Hansa Biopharma call Join the webcast here: Webcast | Hansa Biopharma Third Quarter Earnings Contacts for more information: Evan Ballantyne, Chief Financial Officer [email protected] Kerstin Falck, VP Global Corporate Affairs [email protected] Notes to editors About Hansa Biopharma Hansa Biopharma AB is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life-altering treatments for patients with rare immunological conditions. The company has a rich and expanding research and development program based on its proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in autoimmune diseases, gene therapy and transplantation. The company's portfolio includes imlifidase, a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients and HNSA-5487, a next-generation IgG cleaving molecule with redosing potential. Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The company is listed on Nasdaq Stockholm under the ticker HNSA. Find out more at and follow us on LinkedIn. ©2025 Hansa Biopharma AB. Hansa Biopharma, the beacon logo, IDEFIRIX, and IDEFIRIX flower logo are trademarks of Hansa Biopharma AB, Lund, Sweden. All rights reserved. This information was brought to you by Cision The following files are available for download: SOURCE Hansa Biopharma AB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SEVILLE, SPAIN — Gene therapies are offering new hope to some patients with rare disease, but not everyone. A third or more of patients are often turned away because they’ve already been infected with natural versions of the viruses used to shuttle therapeutic genes into cells. Antibodies developed from those prior viral exposures would render the gene therapy ineffective, and potentially unsafe. But a new study from the French research institute Genethon and the Swedish company Hansa Biopharma suggests a way to overcome that restriction. Jeremy Do Cao, a pediatrician at Antoine-Béclère Hospital in Paris, tested a new approach in a 22-year-old woman who was previously ineligible to participate in a clinical trial of Genethon’s gene therapy for her ultra-rare disease, Crigler–Najjar syndrome. That disease is caused by mutations that break an enzyme essential for metabolizing bilirubin. Genethon’s gene therapy, called GNT003, uses the viral vector AAV8 to deliver the genetic instructions for making the bilirubin-busting enzyme. The patient was excluded from the original trial because she had antibodies to AAV8, but she was included in a new study that used a bacteria-derived enzyme called imlifidase, marketed as Idefirix by Hansa, to temporarily clear out those antibodies, Do Cao told Endpoints News . After receiving that imlifidase pretreatment, the patient got Genethon’s gene therapy, which appeared to at least partially work by temporarily lowering her bilirubin levels. Do Cao presented the results on Friday at the annual meeting of the European Society of Gene and Cell Therapy in Spain. “This is the first time we have seen a patient treated with AAV vectors that already had pre-exposure to AAVs,” Giuseppe Ronzitti, a researcher at Genethon who was part of the study, told Endpoints. Ronzitti admits there are still many unanswered questions about the approach, including whether it will work for all varieties of AAV vectors and any kind of disease. He said that a small subset of people have very high levels of anti-AAV antibodies, and it is unclear if imlifidase pretreatment will work for these patients. It is also unknown if the strategy could enable redosing of AAV gene therapies, which is currently impossible since patients acquire immunity to the virus after treatment. “The important thing is to continue this clinical testing,” Ronzitti said. There are plans to enroll two more patients in the Crigler–Najjar syndrome study, he added. If bilirubin levels are left unchecked in people with Crigler–Najjar syndrome, the molecule can cause irreversible brain damage. Blue light phototherapy can turn bilirubin into a form that is more readily excreted from the body, but the process is time-consuming, akin to lying in a tanning bed for half a day. “Imagine sleeping under them for 12 hours every night of your life to be able to function normally and not have any neurological impairment,” Do Cao said in his presentation. That is what his patient had to do, and a primary goal of the new study was to see if the gene therapy would eliminate the need for phototherapy, he added. The patient was given an infusion of imlifidase, along with an allergy medicine and a steroid, two days before receiving the gene therapy infusion. Antibodies targeting AAV8 plummeted briefly before jumping back four days later as the imlifidase wore off. Her bilirubin levels also dropped within a week, suggesting that the viral vector successfully delivered the therapeutic gene to the liver. Bilirubin levels remained lower for about three months as the patient continued undergoing daily phototherapy. But when she reduced and then stopped phototherapy, her bilirubin levels shot back up to the baseline levels she previously maintained. That allowed her to go on her honeymoon free from the blue lights, Do Cao said. Nicola Brunetti-Pierri, a pediatrician and geneticist from Telethon Institute of Genetics and Medicine in Italy who wasn’t involved in the study, told Endpoints that the rise in bilirubin levels suggests that some expression of the gene therapy diminished over time. But he said it doesn’t necessarily mean that the anti-AAV8 antibodies — which came back at much higher levels than baseline — are responsible. “It may be dependent on other issues,” Brunetti-Pierri said. “It is a single patient, so it’s hard to draw conclusions. But the first results are in a way promising.” The imlifidase treatment seemed safe, and the temporary depletion of antibodies didn’t cause an infection, although the patient also received antibiotics as a precaution. The patient experienced two moderate and two mild episodes of liver enzyme elevations that are sometimes seen after gene therapy infusion and can suggest liver damage and inflammation. There were no severe adverse events. Hansa originally developed imlifidase as a way to clear antibodies prior to kidney transplantation and has a conditional approval for the drug in the EU. The company also has agreements with AskBio, Genethon and Sarepta to test the enzyme as a pretreatment in gene therapy. “Our intention is to prove that we can remove antibodies prior to different gene therapies and extend the label for imlifidase with those different applications,” Lena Winstedt, vice president, head of scientific affairs at Hansa, told Endpoints. “We’re pretty sure that if it works with one vector, it would work with another vector.” Paris-based Vivet Therapeutics is working on its own version of imlifidase, dubbed VTX-PID. On Thursday at the conference, Vivet CEO Jean-Philippe Combal presented the results of a Phase 1 study in healthy volunteers showing that his company’s drug could reduce anti-AAV3B antibodies and provide a window of two to five days for a potential gene therapy administration. But Vivet’s drug didn’t lower antibody levels far enough in patients who had high levels of antibodies — specifically an antibody titer of 1/100. That was the same antibody titer that the patient in the Genethon and Hansa study had. Both Combal and Ronzitti independently told Endpoints that antibody titers are measured differently in different labs and it is hard to compare across studies. “There exists no validated, standardized test that works for every capsid, every AAV,” Ronzitti said. “That’s where the complication lies.”