(AGENPARL) – Fri 01 August 2025 9.15 PM CEST / 1-Aug-2025 / Hansa Biopharma AB (STO:HNSA)
Lund, Sweden, 1 August 2025. Hansa Biopharma AB, “Hansa” (Nasdaq Stockholm:
HNSA), today announced topline results from three patients with Duchenne
muscular dystrophy (DMD) treated with Hansa’s imlifidase prior to receiving
Sarepta’s ELEVIDYS (delandistrogene moxeparvovec-rokl) in the SRP-9001-104
trial.
After one dose of imlifidase, three patients experienced a rapid reduction of
IgG antibodies, to levels ≥95% less than baseline. In addition, in these three
patients pre-existing anti-AAV antibodies were reduced below a titre of 1:400,
which enabled treatment with ELEVIDYS. The safety profile of imlifidase was in
keeping with prior experience and the trial did not generate any new safety
signals. Twelve weeks after administration of the gene therapy, patients in the
trial demonstrated evidence of AAV-mediated transduction and expression of micro
-dystrophin, however with levels lower than seen in other trials with ELEVIDYS.
Based on these outcomes, Hansa and Sarepta will discuss appropriate next steps
for the program.
Renée Aguiar-Lucander, CEO, Hansa Biopharma said, “These are the first results
from a clinical trial, assessing imlifidase’s potential for patients with high
levels of anti-AAV antibodies to access approved gene therapies. We are
encouraged that imlifidase was able to substantially reduce both IgG antibodies
and pre-existing anti-AAV-antibodies, to enable patients to be treated with gene
therapy.  We also look forward to reporting data from another ongoing gene
therapy collaboration later this year, to continue to collect evidence of the
potential benefits of imlifidase in gene therapy.”
This is information that Hansa Biopharma AB (publ) is obliged to make public
pursuant to the EU Market Abuse Regulation. The information was submitted for
publication, through the agency of the contact person set out below, at 21:15
CEST on 2025-08-01.
— ENDS —
Contacts for more information:
Evan Ballantyne, Chief Financial Officer
Notes to editors
About Duchenne Muscular Dystrophy (DMD)
DMD is a rare and fatal genetic disease caused by a mutation in the DMD gene
encoding for the protein dystrophin. It is an irreversible, progressive disease
that causes the muscles in the body to become weak and damaged over time,
leaving most patients in a wheelchair by the age of 12. It is currently
estimated that DMD affects one in 3,500 to 5,000 males born worldwide.[1,2]
Approximately 14% of DMD patients have pre-existing IgG antibodies to the rh74
vector.[3] Many gene therapies are based on the use of Adeno Associated Viruses
(AAV) vectors. In some patients the immune system carries antibodies called anti
-AAV antibodies that counteract the gene therapy treatment preventing its
success.
About SRP-9001-104
SRP-9001-104 is an open-label trial of ambulatory male patients with DMD between
four and nine years of age, and with pre-existing antibodies to ELEVIDYS. Up to
six patients will be treated with imlifidase followed by ELEVIDYS. All patients
included in the trial are currently ineligible to receive ELEVIDYS due to the
presence of antibodies targeting AAVrh74, the vector used to deliver Sarepta’s
gene therapy treatment.
About imlifidase 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.[4-7] In some patients the immune system
carries antibodies that counteract the gene therapy treatment preventing its
success.[ 5][-11] 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.[10] Currently, it is estimated that NAbs on average prevent 1 in 3
people from benefiting from gene therapy treatments.[ 5-11]
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 http://www.hansabiopharma.com and follow us on
LinkedIn (https://www.linkedin.com/company/hansa-medical-ab/).
ELEVIDYS is a trademark of Sarepta Therapeutics Inc
©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.
References
1. Emery AE. Neuromuscul Disord. 1991;1(1):19-29. doi: 10.1016/0960
2. Stark AE. Ann Transl Med. 2015 Nov;3(19):287. doi: 10.3978/j.issn.2305
3. Goedeker NL, et al. Evaluation of rAAVrh74 gene therapy vector
seroprevalence by measurement of total binding antibodies in patients with
Duchenne muscular dystrophy. Ther Adv Neurol Disord. 2023 Jan
4. Lundstrom K. Viral Vectors in Gene Therapy: Where Do We Stand in 2023?
5. 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
6. Calcedo R, Wilson JM. Humoral Immune Response to AAV. Front Immunol. 2013
7. 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
8. 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
9. 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
10. 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:
11. 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
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Hansa Biopharma AB, Scheelevägen 22, Lund, 22362 Sweden
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