LUND, Sweden, Jan. 7, 2025 /PRNewswire/ -- Hansa Biopharma AB, "Hansa" or the "Company" (Nasdaq Stockholm: HNSA), today announced that management will attend the 43rd Annual J.P. Morgan Healthcare Conference. If you are interested in a meeting, please contact Hansa Biopharma at ir@hansabiopharma.com.
The Company has achieved several key milestones in the last 12-months across its three key therapeutic areas: Autoimmune, Gene Therapy and Transplantation.
In Autoimmune, the company announced positive data from the 15-HMedIdeS-09 Phase 2 trial in Guillain Barre Syndrome (GBS) and indirect treatment comparison to the International Guillain-Barré Syndrome Outcome Study (IGOS) demonstrating the potential of imlifidase, the Company's first-generation IgG cleaving molecule, to address a significant unmet need in GBS. Additionally, the GOOD-IDES-02 (Phase 3 trial in anti-GBM) completed enrolment and positive results from the NICE-01 Phase 1 trial and additional 12-month analysis for HNSA-5487, the Company's second-generation IgG cleaving molecule, demonstrated rapid and robust IgG reduction and redosing potential.
In Gene Therapy, the Company initiated two trials with gene therapy partners: SRP-9001-104 Phase 1b trial in Duchenne Muscular Dystrophy (DMD) with Sarepta Therapeutics, Inc. (Nasdaq: SRPT) and GNT-018-IDES Phase 2 trial in Crigler-Najjar Syndrome with Genethon. In both trials, imlifidase is being evaluated as a pre-treatment to gene therapy in patients with anti-AAV antibodies.
In Transplantation, randomization of the ConfIdeS (pivotal US Phase 3 trial in kidney transplantation) trial was completed. The Company had four consecutive quarters of strong IDEFIRIX (imlifidase) sales across Europe with Q3 2024 being the highest ever quarterly IDEFIRIX sales performance (69.5 MSEK/$6.4M), underscoring the important role that IDEFIRIX can play in desensitization in kidney transplantation.
In 2025, the Company has several key milestones:
Data read out of the pivotal US Phase 3 ConfIdeS trial for imlifidase and submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) in 2H 2025
Data read out of Sarepta Therapeutics' Phase 1b trial SRP-9001-104 in DMD (2H 2025)
Data read out of the GOOD-IDES-02 Phase 3 trial in anti-GBM
HNSA-5487 development pathway alignment with regulatory agencies in neuro-autoimmune diseases (1H 2025) with an initial focus in myasthenia gravis (MG)
Hansa is developing novel immunomodulating biologic therapies based on its proprietary, first in class IgG cleaving platform and is focused on IgG-driven immune mediated diseases. The Company has two IgG cleaving compounds including imlifidase, a first generation, first in class, single dose therapy with proven efficacy and safety and HNSA-5487, a second-generation IgG cleaving molecule with redosing potential. Imlifidase is conditionally approved in the EU for desensitization in kidney transplantation, with late-stage trials in autoimmune diseases where IgG is a driver of disease, and as a pre-treatment to gene therapy in patients with anti-AAV antibodies.
Imlifidase is a unique antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets IgG and inhibits IgG-mediated immune response.1 It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their activity within hours after administration. Imlifidase is conditionally approved in Europe and is marketed under the trade name IDEFIRIX® for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor.1 Full product information can be accessed via the initial Summary of Product Characteristics found here.
About HNSA-5487
HNSA-5487 is Hansa Biopharma's second-generation IgG-cleaving enzyme with the potential to prolong the IgG-low window and redosing potential. In the NICE-01 Phase 1b trial, HNSA-5487 demonstrated rapid and highly robust reduction of IgG levels by more than 95 percent within a few hours post treatment. In a 12-month follow up analysis IgG levels returned to normal range six months after initial dosing. This confirms that HNSA-5487 mirrors the extremely high efficacy of imlifidase, the Company's first-generation IgG-cleaving enzyme, in reducing total IgG levels. No serious adverse events were observed and as previously communicated HNSA-5487 is safe and well tolerated.
About Hansa and Kidney Transplantation
Kidney disease can progress to kidney failure or End-Stage Renal Disease (ESRD), identified when a patient's kidney function is less than 15%.2 ESRD poses a significant health burden, affecting nearly 2.5 million patients worldwide.2 A kidney transplant is the treatment of choice for suitable patients with ESRD because it offers improved survival and quality of life benefits, and is cost savings compared to long-term dialysis. There are approximately 170,000 kidney patients in the U.S. and Europe waiting for a new kidney.3
Highly sensitized kidney transplant patients have pre-formed antibodies called donor specific antibodies (DSAs) with a broad reactivity against human leukocyte antigens (HLAs), which can cause tissue damage and potentially transplant rejection.4 The presence of DSAs means that highly sensitized patients tend to have limited or no access to transplant, as finding a compatible donor organ can be particularly challenging.5,6 The complexity of their immunological profile means that highly sensitized patients spend longer time than average on transplant waiting lists, with evidence showing that this longer time waiting for a suitable donor relates to an increased mortality risk.7,8 Across the U.S. and Europe, highly sensitized patients comprise around 10-15% of the total of patients on transplant waiting lists.9,10
Imlifidase is a promising new strategy for desensitization of transplant patients with donor-specific anti-HLA (Human Leukocyte Antigens) antibodies (DSAs).11 Highly sensitized patients have high levels of these preformed antibodies that can bind to the donor organ and damage the transplant.4 Once they are inactivated with imlifidase, there is a window of opportunity for the transplant to take place. By the time the body starts to synthesize new IgG, the patient will be receiving post-transplant immunosuppressive therapy to reduce the risk of organ rejection.
The efficacy and safety of imlifidase as a pre-transplant treatment to reduce donor-specific IgG was studied in four Phase 2 open-label, single-arm, six-month clinical trials.10-13 Hansa is collecting further clinical evidence and will submit additional efficacy and safety data based on one observational follow-up study and one post authorization efficacy study (PAES).
About Hansa and Autoimmune Diseases
Autoimmune diseases form a group of serious diseases caused by the immune system attacking the body. In many autoimmune diseases the immune system mistakenly recognizes the body's own proteins, as foreign and mounts an immune response, creating antibodies to attack the body's own cells and tissues.14-16 Pathogenic IgG can contribute to a broad spectrum of autoimmune diseases.
Hansa Biopharma is exploring how imlifidase and HNSA-5487 may be able to prevent or slow the progression of these diseases and their debilitating, life-threatening symptoms. Imlifidase is currently being studied in the following autoimmune diseases: anti-glomerular basement membrane (anti-GBM) disease and Guillain-Barré Syndrome (GBS). HNSA-5487 is moving quickly into the clinical phase focusing on patients with myasthenia gravis (MG) and potentially other neuro-autoimmune diseases.
About Hansa and Gene Therapy
Imlifidase is currently being evaluated as a pre-treatment to gene therapy in areas of high unmet need. Many gene therapies are based on the use of Adeno Associated Viruses (AAV) vectors.17-19 In some patients the immune system carries antibodies that counteract the gene therapy treatment preventing its success.18-24 Pre-treatment with imlifidase prior to AAV-based gene therapy treatment has the potential to inactivate antibodies and thereby enable gene therapy in patients with pre-existing antibodies to AAV-based gene therapies.23 It is estimated that anti-AAV antibodies on average prevent 1 in 3 people from benefiting from gene therapy treatments.18-21
About Hansa Biopharma
Hansa Biopharma 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 second-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 www.hansabiopharma.com and follow us on LinkedIn.
3. Newsletter Transplant 2022. International figures on donation and transplantation. Available at: Newsletter Transplant - latest edition I Freepub (edgm.eu) Accessed: May 2024.
4. Eurostam Report (A Europe-wide strategy to enhance transplantation of highly sensitized patients on the basis of acceptable HLA mismatches.) Available at https://cordis.europa.eu/project/id/305385/reporting.
5. Redfield RR, et al. The mode of sensitization and its influence on allograft outcomes in highly sensitized kidney transplant recipients. Nephrol Dial Transplant. 2016 Oct;31(10):1746-53. doi: 10.1093/ndt/gfw099.
6. Lonze BE, et al. IdeS (Imlifidase): A Novel Agent That Cleaves Human IgG and Permits Successful Kidney Transplantation Across High-strength Donor-specific Antibody. Ann Surg. 2018 Sep;268(3):488-496. doi: 10.1097/
7. Alelign T, Ahmed MM, Bobosha K, Tadesse Y, Howe R, Petros B. Kidney Transplantation: The Challenge of Human Leukocyte Antigen and Its Therapeutic Strategies. J Immunol Res. 2018 Mar 5;2018:5986740. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5859822/
8. Heidt S, et al. Highly Sensitized Patients are Well Serves by Recieving a Compatible Organ Offer Based on Acceptable Mismatches. Front Immunol. 2021;12:687254. Available at: https://pubmed.ncbi.nlm.nih.gov/34248971/
9. Organ Procurement and Transplantation Network (OPTN) and Scientific Registry of Transplant Recipients (SRTR). OPTN/SRTR 2022 Annual Data Report. U.S. Department of Health and Human Services, Health Resources and Services Administration; 2024. Accessed [June 2024].
10. Jordan SC, et al. Imlifidase Desensitization in Crossmatch-positive, Highly Sensitized Kidney Transplant Recipients: Results of an International Phase 2 Trial (Highdes). Transplantation. 2021 Aug 1;105(8):1808-1817. doi: 10.1097/TP.0000000000003496.
11. Jordan SC, et al. IgG Endopeptidase in Highly Sensitized Patients Undergoing Transplantation. N Engl J Med 2017;377:442-453. DOI: 10.1056/NEJMoa16125
12. Winstedt L, et al. Complete Removal of Extracellular IgG Antibodies in a Randomized Dose-Escalation Phase I Study with the Bacterial Enzyme IdeS--A Novel Therapeutic Opportunity. PLoS One. 2015 Jul 15;10(7):e0132011. doi: 10.1371/journal.pone.0132011. PMID: 26177518; PMCID: PMC4503742.
13. Lorant T, et al. Safety, immunogenicity, pharmacokinetics, and efficacy of degradation of anti-HLA antibodies by IdeS (imlifidase) in chronic kidney disease patients. Am J Transplant. 2018 Nov;18(11):2752-2762. doi: 10.1111/ajt.14733.
14. Angum F, et al. The Prevalence of Autoimmune Disorders in Women: A Narrative Review. Cureus. 2020 May. 12(5):e8094. doi: 10.7759/cureus.8094.
15. Wang L, et al. Human autoimmune diseases: a comprehensive update. J Intern Med. 2015 Oct;278(4):369-95. doi: 10.1111/joim.12395.
16. Ma H, Murphy C, Loscher CE and O'Kennedy R (2022) Autoantibodies – enemies, and/or potential allies? Front. Immunol. 13:953726. doi: 10.3389/fimmu.2022.953726
17. Lundstrom K. Viral Vectors in Gene Therapy: Where Do We Stand in 2023? Viruses. 2023 Mar 7;15(3):698. doi: 10.3390/v15030698. PMID: 36992407; PMCID: PMC10059137.
18. Boutin S, et al. Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum Gene Ther. 2010 Jun;21(6):704-12. doi: 10.1089/hum.2009.182. PMID: 20095819.
19. Calcedo R, Wilson JM. Humoral Immune Response to AAV. Front Immunol. 2013 Oct 18;4:341. doi: 10.3389/fimmu.2013.00341. PMID: 24151496; PMCID: PMC3799231.
20. Veron P, Leborgne C, Monteilhet V, Boutin S, Martin S, Moullier P, Masurier C. Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors. J Immunol. 2012 Jun 15;188(12):6418-24. doi: 10.4049/jimmunol.1200620. Epub 2012 May 16. PMID: 22593612.
21. Kruzik A, et al. Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors. Mol Ther Methods Clin Dev. 2019 Jun 7;14:126-133. doi: 10.1016/j.omtm.2019.05.014. PMID: 31338384; PMCID: PMC6629972.
22. Falese L, et al. Strategy to detect pre-existing immunity to AAV gene therapy. Gene Ther. 2017 Dec;24(12):768-778. doi: 10.1038/gt.2017.95. Epub 2017 Nov 6. PMID: 29106404; PMCID: PMC5746592.
23. Leborgne C, et al. IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies. Nat Med. 2020 Jul;26(7):1096-1101. doi: 10.1038/s41591-020-0911-7. Epub 2020 Jun 1. PMID: 32483358.
24. Au H.K, et al. (2022) Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings. Front. Med. 8:809118. doi: 10.3389/fmed.2021.809118
