An Open-label, Phase 2 Trial to Evaluate the Efficacy and Safety of a Single Intravenous Administration of GNT0003 (an Adeno-associated Viral (AAV) Vector Expressing the UGT1A1 Transgene) Following Imlifidase Pre-treatment in Adult Participants With Severe Crigler-Najjar Syndrome (CNS) Requiring Daily Phototherapy and Presenting Pre-existing Anti-AAV8 Antibodies

Clinical trial rationale:
CNS is an ultra-rare (<1/1 million newborns), autosomal recessive disorder of bilirubin conjugation caused by mutation in the gene coding for uridine 5'-diphosphate glucuronosyltransferase (UGT1A1), that causes the accumulation of neurotoxic unconjugated bilirubin (UCB).
Reduction of UCB is managed with phenobarbital in mild CNS, and daily phototherapy in severe CNS.
There is no authorized curative medical treatment for CNS. Liver transplantation is currently the only curative treatment for severe CNS.
GNT0003 is a genetically modified recombinant (r) viral vector composed of the AAV8 viral capsid carrying the UGT1A1 transgene which aims to correct the dysfunction of the mutated gene by achieving durable expression of a functional copy of the affected gene.
Imlifidase (IgG-degrading enzyme) has demonstrated its efficacy in highly sensitized adult kidney transplant patients.
To give participants with pre-existing anti-AAV8 antibodies access to gene therapy treatments, this trial aims to demonstrate the safety and efficacy of GNT0003 following imlifidase pre-treatment in adult participants with severe CNS requiring daily phototherapy and presenting with pre-existing anti-AAV8 antibodies.
Primary objective: to assess efficacy of a single intravenous administration of GNT0003 following imlifidase pre-treatment in participants with severe CNS requiring phototherapy and pre-existing AAV8 antibodies
Secondary objective: to collect data on safety and tolerability of GNT0003 and imlifidase, efficacy of imlifidase, pharmacokinetic and pharmacodynamic profile of GNT0003, and Quality of Life.
The trial will include 3 parts:
* A baseline period for at least 3 months
* A treatment period
* A follow-up period:
* Initial post-treatment follow-up over 48 weeks
* Long-term follow-up for 4 additional years
This trial will be conducted in accordance with the International Conference on Harmonization Guideline for Good Clinical Practice and the Declaration of Helsinki. Participants must be consented using the approved Informed Consent Form before any procedures specified in the protocol are performed.

Start Date08 Nov 2024

Sponsor / Collaborator

Genethon

[+1]

NCT06461546 / Not yet recruitingPhase 2IIT

Imlifidase in Living Donor Renal Transplantation Highly Sensitized Recipients (LIVEDES)

This is a Phase II, pilot, prospective, unicentric trial, to evaluate Imlifidase could improve the transplantability of the highly sensitized patients with good outcomes respect to survival and functionality of the graft.

Start Date01 Nov 2024

Sponsor / Collaborator

Fundació Clinic per a la Recerca Biomedica

NCT06241950 / Enrolling by invitationPhase 1

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association With Imlifidase in Subjects With Duchenne Muscular Dystrophy With Pre-existing Antibodies to rAAVrh74

This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.

Start Date29 Jan 2024

Sponsor / Collaborator

Sarepta Therapeutics, Inc.

[+1]

100 Clinical Results associated with Imlifidase

100 Translational Medicine associated with Imlifidase

100 Patents (Medical) associated with Imlifidase

247

Literatures (Medical) associated with Imlifidase

01 Nov 2024·DRUG AND CHEMICAL TOXICOLOGY

Effects of idebenone and coenzyme Q10 on NLRP3/caspase-1/IL-1β pathway regulation on ethanol-induced hepatotoxicity in rats

Article

Author: Halici, Zekai ; Bahador Zirh, Elham ; Palabiyik-Yucelik, Saziye Sezin ; Yoladi, Fatma Betül ; Baydar, Terken

Chronic and excessive alcohol consumption leads to liver toxicity. There is a need to investigate effective therapeutic strategies to alleviate alcohol-induced liver injury, which remains the leading cause of liver-related morbidity and mortality worldwide. Therefore here, we looked into and evaluated how ethanol-induced hepatotoxicity was affected by coenzyme Q10 (CoQ10) and its analog, idebenone (IDE), on the NLRP3/caspase-1/IL-1 pathway. Hepatotoxicity induced in rats through the oral administration of gradually increasing dosages of ethanol (from 2 to 6 g/kg/day) over 30 days and the effect of CoQ10 (10 or 20 mg/kg) and IDE (50 or 100 mg/kg) were evaluated. Serum hepatotoxicity markers (ALT, AST, GGT, ALP, and TBIL), tissue oxidative stress markers and the mRNA expressions of IL-1β, IL-18, TGF-β, NF-κB, NLRP3, and caspase-1 were evaluated. Masson's trichrome staining was also used to visualize fibrosis in the liver tissue. The results indicated that ethanol exposure led to hepatotoxicity as well as considerable NLRP3/caspase-1/IL-1β pathway activation. Moreover, CoQ10 or IDE treatment reduced measured parameters in a dosage-dependent manner. Thus, by inhibiting the NLRP3/caspase-1/IL-1 pathway, CoQ10 and IDE can prevent the hepatotoxicity caused by ethanol, although CoQ10 is more effective than IDE. This study will provide insight into new therapeutic avenues that take advantage of the anti-inflammatory and antioxidant properties of CoQ10 and IDE in ethanol-induced liver diseases.

01 Nov 2024·INTERNATIONAL JOURNAL OF PHARMACEUTICS

Randomly methylated β-cyclodextrin improves water – solubility, cellular protection and mucosa permeability of idebenone

Article

Author: Celesti, Consuelo ; Bulzomí, Maria ; Paterniti, Irene ; De Gaetano, Federica ; Vincenzo Giofrè, Salvatore ; Anna Ventura, Cinzia ; Mannino, Deborah ; Iraci, Nunzio ; Esposito, Emanuela

Neurodegenerative diseases such as Alzheimer's are very common today. Idebenone (IDE) is a potent antioxidant with good potential for restoring cerebral efficiency in cases of these and other medical conditions, but a serious drawback for the clinical use of IDE in neurological disorders lies in its scarce water solubility, which greatly inhibits its bioavailability. In this work, we prepared the inclusion complex of IDE with randomly methylated β-cyclodextrin (RAMEB), resulting in improved water solubility of the included drug; then its in vitro biological activity and ex vivo permeability was evalutated. The solid complex was characterized through FT-IR spectroscopy, Thermogravimetric analysis (TGA) and Differential Scanning Calorimetry (DSC). A 78-fold improvement of the solubility of IDE in water resulted, together with a strong 1:1 host-guest interaction (association constant of 12630 M^-1), and dissolution of the complex within 15 min, all evidenced during the in-solution studies. Biological in vitro studies were then performed on differentiated human neuroblastoma cells (SH-SY5Y) subjected to oxidative stress. Pretreatment with IDE/RAMEB positively affected cell viability, promoted the nuclear translocation of Nrf2, and increased the levels of GSH as well as those of the endogenous antioxidant enzymes Mn-SOD and HO-1. Lastly, the complexation significantly improved the permeation of IDE through isolated rat nasal mucosa.

01 Nov 2024·CURRENT MEDICINAL CHEMISTRY

Idebenone Attenuates Diabetic Retinopathy by Modulating Autophagy Via Targeting Akt Signaling

Article

Author: Yu, Zhenqian ; Liu, Gang

Introduction::

Diabetic Retinopathy (DR) is a common microvascular issue caused by diabetes. Idebenone (IDE) is a coenzyme Q10 analog and antioxidant that has been utilized in the treatment of neurodegenerative diseases.

Method::

Our goal was to investigate how IDE might treat diabetic retinopathy. An in vivo DR model was established by injecting a single dose of streptozotocin (STZ). Rats were treated with IDE, and their vascular function was measured by ultrasound. The retina structure was checked by haematoxylin and eosin (HE) staining. The expression of biomarkers of autophagy and apoptosis was measured by western blotting assay. The retina endothelial cell line RF/6A was stimulated with high glucose (HG) and treated with IDE. Cell proliferation and apoptosis were assessed using the Edu assay, TUNEL assay, and flow cytometry, respectively.

Result::

Reduced peak systolic velocity (PSV), mean velocity (MV), end-diastolic velocity (EDV), and increased pulsatility index (PI) and resistance index (RI) were observed in diabetic rats; however, these traits were reversed by IDE therapy. IDE alleviated the STZ-induced disordered retina structure. The IDE administration suppressed DR-induced apoptosis and autophagy both in vivo and in vitro. IDE suppressed the activation of Phosphatidylinositol 3 kinase (PI3K) signaling. Activation of PI3K abolished the IDE-alleviated retina damage and cell death.

Conclusion::

IDE regulated the autophagy of retina cells to alleviate diabetic retinopathy via regulating the PI3K signaling pathway.

News (Medical) associated with Imlifidase

05 Dec 2024

·www.prnewswire.com

Hansa Biopharma Completes Enrolment in Global Pivotal Phase 3 Trial of imlifidase in Anti-Glomerular Basement Membrane Disease

Data from the trial is expected to be shared in 2025 LUND, Sweden, Dec. 5, 2024 /PRNewswire/ -- Hansa Biopharma, "Hansa" (Nasdaq Stockholm: HNSA) today announced it has completed the enrolment of patients in the GOOD-IDES-02 trial, a global pivotal Phase 3 trial in anti-glomerular basement membrane (anti-GBM) disease. Anti-GBM is a rare, severe autoimmune condition affecting around 1.6 people per million annually.1 Imlifidase has been granted orphan drug designation for the treatment of anti-GBM disease by both the U.S. FDA and the European Medicines Agency (EMA). Enrolment completion was originally planned for 2025. Søren Tulstrup, President and CEO, Hansa Biopharma said, "We are very pleased with the fast enrolment of all 50 patients in the GOOD-IDES-02 trial. The completion of patient enrolment in this pivotal Phase 3 trial is an important milestone in our efforts to investigate the potential role for imlifidase to address the high unmet need in anti-GBM. The majority of patients with anti-GBM today lose kidney function and two-thirds experience kidney failure requiring long-term dialysis. We look forward to sharing further updates on the outcome of the study and potential path forward in 2025." Anti-GBM is Hansa Biopharma's most advanced program in the autoimmune space. GOOD-IDES-02 is an open label, multi-center Phase 3 trial involving over 40 centers across the US, UK, and EU. A total of 50 patients have been enrolled in the trial. In the trial, 25 patients were randomized to receive imlifidase in combination with standard of care (SoC), consisting of a combination of immunosuppressives, glucocorticoids, and plasma exchange, and 25 patients received only SoC. The primary objective of the study is to assess the superior effect on kidney function of imlifidase in combination with SoC versus SoC alone in the treatment of patients affected by severe anti-GBM disease. The performance of the treatment is assessed at 6 months through the evaluation of renal function as measured by estimated glomerular filtration rate (eGFR) and need of dialysis. In addition, the safety profile and efficacy on pulmonary symptoms and health related quality of life aspects are evaluated. More information about the trial is available at ClinicalTrials.gov under NCT05679401. Contacts for more information: Evan Ballantyne, Chief Financial Officer [email protected] Stephanie Kenney, VP Global Corporate Affairs [email protected] Notes to editors About anti-GBM disease Anti-glomerular basement membrane (anti-GBM) disease, also known as Goodpasture disease, is a rare, severe autoimmune condition affecting around 1.6 people per million annually1 with majority of patients losing their kidney function.2,3 In anti-GBM disease, the immune system mistakenly develops antibodies against an antigen intrinsic to the glomerular basement membrane, resulting in an acute immune attack of the kidneys and, in around half of the patients, also the lungs. Approximately two thirds of anti-GBM patients will experience kidney failure and require long-term dialysis while awaiting potential kidney transplantation.4 Some patients may also experience bleeding from the lungs. In one out of six patients, anti-GBM disease can become fatal during the acute phase. Imlifidase has been granted orphan drug designation for the treatment of anti-GBM disease by both the U.S. FDA and the European Medicinal Agency (EMA). About imlifidase Imlifidase is a unique antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets IgG and inhibits IgG-mediated immune response.5 It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their activity within hours after administration. Imlifidase has conditional marketing approval 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.5 About imlifidase 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.6-8 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). 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. Hansa Biopharma has developed a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa Biopharma has a rich and expanding research and development program based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy and cancer. 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. ©2024 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. Canney M, et al. Spatial and Temporal Clustering of Anti-Glomerular Basement Membrane Disease. Clin J Am Soc Nephrol. 2016 Aug 8;11(8):1392-1399. doi: 10.2215/CJN.13591215. 2. McAdoo SP, Pusey CD. Anti-Glomerular Basement Membrane Disease. Clin J Am Soc Nephrol. 2017 Jul 7;12(7):1162-1172. doi: 10.2215/CJN.01380217 3. Kluth DC, Rees AJ. Anti-glomerular basement membrane disease. J Am Soc Nephrol. 1999 Nov;10(11):2446-53. doi: 10.1681/ASN.V10112446. 4. Hellmark T, Segelmark M. Diagnosis and classification of Goodpasture's disease (anti-GBM). J Autoimmun. 2014 Feb-Mar;48-49:108-12. doi: 10.1016/j.jaut.2014.01.024. 5. European Medicines Agency. Idefirix® summary of product characteristics. Available at: . 6. Angum F, et al. The Prevalence of Autoimmune Disorders in Women: A Narrative Review. Cureus. 2020 May 13;12(5):e8094. doi: 10.7759/cureus.8094. 7. Wang L, et al. Human autoimmune diseases: a comprehensive update. J Intern Med. 2015 Oct;278(4):369-95. doi: 10.1111/joim.12395. 8. 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 This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Orphan DrugClinical Result

03 Dec 2024

·www.prnewswire.com

Genethon and Hansa Biopharma announce initiation of a Phase 2 trial of imlifidase as a pre-treatment to GNT-0003 in severe Crigler-Najjar syndrome

The trial will be conducted in patients with pre-existing anti-AAV antibodies which limit use of gene therapy treatment. LUND, Sweden and EVRY, France, Dec. 3, 2024 /PRNewswire/ -- Hansa Biopharma, "Hansa" (Nasdaq Stockholm: HNSA) and Genethon, a pioneer and a leader in gene therapy research and development for rare genetic diseases, today announced initiation of GNT-018-IDES, a Phase 2 trial in patients with Crigler-Najjar syndrome with pre-existing antibodies against adeno-associated virus (AAV) vectors. The trial will evaluate the efficacy and safety of a single intravenous administration of Genethon's gene therapy GNT-0003 following pre-treatment with imlifidase, Hansa's first-in-class immunoglobulin G (IgG) antibody cleaving enzyme therapy, in patients with severe Crigler-Najjar syndrome and pre-formed antibodies to AAV serotype 8 (AAV8). Søren Tulstrup, President and CEO, Hansa Biopharma said, "We know that anti-AAV antibodies prevent up to 1 in 3 people from benefitting from gene therapies using AAV-vectors.1-4 That's why our collaboration with Genethon and the initiation of the Phase 2 clinical trial in Crigler-Najjar syndrome is so important. This collaboration with Genethon is the second of our three partnerships with leading gene therapy companies to have reached the clinical stage, marking an important milestone in our efforts to enable a much larger group of patients to benefit from potentially lifesaving gene therapies." Antibodies against AAV vectors remain a major challenge, as their presence in patients excludes them from entering clinical studies with potentially curative gene therapy treatments and from access to currently marketed and future gene therapies. Frédéric Revah, CEO, Genethon added: "This new clinical trial reflects Genethon's commitment in pursuing innovative strategies to ensure and broaden access to gene therapies for patients suffering from rare diseases. Patients with pre-existing neutralizing antibodies against AAV vectors cannot today benefit from gene therapy. The initiation of this clinical trial and the collaboration with Hansa Biopharma is a crucial step for Genethon and highlights several years of pioneering research to understand and control the immune response to AAV in order to make gene therapy more effective and to increase the number of patients able to access it." GNT-018-IDES, sponsored by Genethon, is a single arm Phase 2 trial with a total of three patients aged ≥18 years with Crigler-Najjar syndrome and pre-formed anti-AVV8 antibodies and requiring phototherapy. Once screened, patients will undergo a three-month observational period before being dosed with imlifidase followed by GNT-0003. Genethon and Hansa expect to communicate data from the trial in 2025. GNT-0003 is currently being evaluated in a pivotal clinical trial following the positive results of the phase 1-2 dose escalation study showing safety and efficacy of GNT-0003, and was granted PRIME priority drug status from the EMA. If successful, GNT-0003 would be the first gene therapy treatment for Crigler-Najjar syndrome. Contacts for more information: HANSA BIOPHARMA Evan Ballantyne, Chief Financial Officer [email protected] Stephanie Kenney, VP Global Corporate Affairs [email protected] GENETHON Stéphanie Bardon [email protected] +33 (0) 6.45.15.95.87 Notes to editors About imlifidase Imlifidase is a unique antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets IgG and inhibits IgG-mediated immune response.5 It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their activity within hours after administration. Imlifidase has conditional marketing approval 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.5 About Crigler-Najjar syndrome Crigler-Najjar syndrome is a rare genetic liver disease characterized by abnormally high levels of bilirubin in the blood (hyperbilirubinemia), which leads to irreversible neurological damage manifested as muscle weakness, lethargy, deafness, mental retardation, and eye movement paralysis. This accumulation of bilirubin is caused by a deficiency of the UGT1A1 enzyme, responsible for transforming bilirubin into a substance that can be eliminated by the body. It can result in significant neurological damage and death if not treated quickly. At present, patients must undergo phototherapy for up to 12 hours a day to keep their bilirubin levels below the toxicity threshold. Crigler-Najjar syndrome is an ultra-rare disease affecting less than one case per one million people per year.6 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.1-4,7 In some patients the immune system carries antibodies that counteract the gene therapy treatment preventing its success.1-4,8-10 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.9 Currently, it is estimated that anti-AAV antibodies on average prevent 1 in 3 people from benefiting from gene therapy treatments.1-4,8 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. Hansa Biopharma has developed a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa Biopharma has a rich and expanding research and development program based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy and cancer. 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. About Genethon A pioneer in the discovery and development of gene therapies for rare diseases, Généthon is a non-profit organization created by the AFM-Téléthon. The first gene therapy to treat spinal muscular atrophy, incorporating technologies developed at Genethon, is marketed worldwide. With over 200 scientists and professionals, Genethon pursues its goal of developing innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen products from Genethon's R&D or collaborations are in clinical trials for diseases of the liver, blood, immune system, muscles and eyes. A further seven products could enter clinical trials in the next five years. To find out more ©2024 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 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. 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. 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. 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. European Medicines Agency. Idefirix® summary of product characteristics. Available at: . . Last accessed: 29 November 2024 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. 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. 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. 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 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

Phase 2Gene Therapy

17 Oct 2024

·www.prnewswire.com

Hansa Biopharma Reports Third Quarter and Interim January - September 2024 Financial Results

Q3 2024 marks highest ever in-market quarterly sales performance and fourth consecutive quarter of strong sales performance LUND, Sweden, Oct. 17, 2024 /PRNewswire/ -- Hansa Biopharma today announced its interim report for January-September and third quarter 2024 financial results. Søren Tulstrup, President and CEO, Hansa Biopharma said, "The third quarter of 2024 marks the highest ever IDEFIRIX® sales performance to date, resulting in four consecutive quarters of strong sales performance. The launch of IDEFIRIX in Europe continues to advance with adoption in international organ allocation systems and an increase in new and repeat utilization in leading transplant clinics. Equally, we see strong momentum in our clinical development programs in autoimmune, gene therapy and transplantation. Enrollment in the Phase 3 GOOD-IDES-02 study in anti-GBM has reached 86%. In the Post Authorization Efficacy and Safety (PAES) study in kidney transplant enrolment has reached 78%. Finally, just after quarter end, we communicated positive results from the 12-month analysis of NICE-01, demonstrating that HNSA-5487 can robustly and very rapidly reduce IgG levels, has clear redosing potential, and a favorable safety and tolerability profile. Initial clinical development will focus on neuromyelitis optica (NMO), myelin oligodendrocyte glycoprotein antibody disease (MOGAD), and myasthenia gravis (MG)." Financial Performance The Company recorded total revenue of 78.4 MSEK. Of this, 69.5 MSEK is attributed to IDEFIRIX sales, marking the highest ever quarterly in-market sales. This excludes the impact of a 29.7 MSEK provision related to potential price adjustments from cumulative sales since the launch of IDEFIRIX in Europe in 2020. Of the total provision only 4.9 MSEK was related to Q3 sales. Net of the provision, Q3 2024 IDEFIRIX sales were 39.8 MSEK and total year to date IDEFIRIX sales were 114.5 MSEK. Previously, the Company recorded a provision of 19.9 MSEK in Q2 2024. Of this, 2.0 MSEK was related to sales in Q2 2024. Of the total provision taken in Q2 and Q3 2024, 42.7 MSEK relates to previous periods since launch in 2020. Pipeline Progress Progress across the pipeline included the strong momentum in enrollment in the Phase 3 GOOD-IDES-02 study in anti-GBM (86%), and in the Post Authorization Efficacy and Safety (PAES) study in kidney transplantation (78%). Further efficacy data from the 15-HMedIdes-09 Phase 2 trial in Guillain-Barré Syndrome (GBS) is scheduled to read out by the end of 2024. The ConfIdeS trial continues to progress following completed randomization in May and data from the study is expected to provide the basis for a Biologics License Application (BLA) with the US Food and Drug Administration (FDA) in the second half of 2025. On 7 October 2024, Hansa announced positive results from a 12-month follow up analysis from the NICE-01 trial of HNSA-5487, assessing IgG recovery, immunogenicity and redosing potential. Financial Summary Conference Call Details Hansa Biopharma will host a telephone conference today Thursday, October 17, 2024, at 14:00 CET / 8:00 am ET. The event will be hosted by Søren Tulstrup, President and CEO, Evan Ballantyne, CFO, and Hitto Kaufmann, CR&DO. The call will include a review of the interim results and a business and pipeline update. It will be held in English. Slides used in the presentation will be live on the company website during the call under Events & Presentations 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): 1-877-270-2148 Participant International Dial In: 1-412-902-6510 *Please ask to be joined into the Hansa Biopharma call Join the webcast here: For the full financial calendar and list of events in 2024 please visit This is information that Hansa Biopharma AB is obliged to make public pursuant to the Securities Markets Act. For more information: Evan Ballantyne, Chief Financial Officer [email protected] Stephanie Kenney, VP Global Corporate Affairs [email protected] Notes to editors 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. Hansa Biopharma has developed a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa Biopharma has a rich and expanding research and development program based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy and cancer. 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. ©2024 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: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Financial StatementClinical ResultGene Therapy

100 Deals associated with Imlifidase

External Link

KEGG	Wiki	ATC	Drug Bank
D11470	Imlifidase	L04AA41	DB15258

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Renal transplant rejection	EU	Hansa Biopharma AB	25 Aug 2020
Renal transplant rejection	IS	Hansa Biopharma AB	25 Aug 2020
Renal transplant rejection	LI	Hansa Biopharma AB	25 Aug 2020
Renal transplant rejection	NO	Hansa Biopharma AB	25 Aug 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Anti-Glomerular Basement Membrane Disease	Phase 3	US	Hansa Biopharma AB	22 Dec 2022
Anti-Glomerular Basement Membrane Disease	Phase 3	AT	Hansa Biopharma AB	22 Dec 2022
Anti-Glomerular Basement Membrane Disease	Phase 3	BE	Hansa Biopharma AB	22 Dec 2022
Anti-Glomerular Basement Membrane Disease	Phase 3	CZ	Hansa Biopharma AB	22 Dec 2022
Anti-Glomerular Basement Membrane Disease	Phase 3	DK	Hansa Biopharma AB	22 Dec 2022
Anti-Glomerular Basement Membrane Disease	Phase 3	FR	Hansa Biopharma AB	22 Dec 2022
Anti-Glomerular Basement Membrane Disease	Phase 3	DE	Hansa Biopharma AB	22 Dec 2022
Anti-Glomerular Basement Membrane Disease	Phase 3	IE	Hansa Biopharma AB	22 Dec 2022
Anti-Glomerular Basement Membrane Disease	Phase 3	IT	Hansa Biopharma AB	22 Dec 2022
Anti-Glomerular Basement Membrane Disease	Phase 3	NL	Hansa Biopharma AB	22 Dec 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04711850 (EudraCT number: 2018-000022-66, 2020-004777-49, Pubmed \| Clin Transplant)	Not Applicable	Renal transplant rejection DSA	-	Imlifidase	pmuqqossqs(csmptwmncl) = klyvdotvkw lajonuwmla (bwyobwmjdj ) View more	Negative	01 Jul 2024
				Imlifidase (Plasmapheresis)	pmuqqossqs(csmptwmncl) = ctrskeybxy lajonuwmla (bwyobwmjdj ) View more
NCT03897205 (16-HMedIdes-12, CTgov)	Phase 2	Renal transplant rejection	30	Imlifidase (Imlifidase)	xjwfbmtppi(uxsrklpqsz) = imsruzkhgq vvifvkozrx (tmullvznuy, wdybiwucbz - qebngberye) View more	-	28 Feb 2024
				Plasma Exchange (Plasma Exchange)	xjwfbmtppi(uxsrklpqsz) = wxvbdnqlwv vvifvkozrx (tmullvznuy, setfccvhkf - hcxwdwdyno) View more
NCT03897205 (16-HMedIdes-12, PRNewswire) Manual	Phase 2	AMR Syndrome	30	imlifidase	izrrjrsmic(icrwiotsdw) = iytspwyauv dintdzrwta (zdwipriori ) View more	Positive	14 Dec 2023
				standard of care	izrrjrsmic(icrwiotsdw) = lytyhmfoin dintdzrwta (zdwipriori ) View more
NCT03943589 (15-HMedIdeS-09, Biospace) Manual	Phase 2	Guillain-Barre Syndrome	-	imlifidase+ intravenous immunoglobulin	edluzozysg(mlemqynqpm) = Imlifidase was safe and well tolerated yfhmvphukc (ziwdfqhzjr ) View more	Positive	07 Dec 2023
NCT03897205 (Biospace) Manual	Phase 2	Graft Rejection	30	Imlifidase	nntgyvbycn(gxnzyjrkvr) = With regard to safety, imlifidase was well tolerated, and no safety signals were encountered during the study pmysdohpki (ntrmsfnxzj )	Positive	28 Nov 2022
				plasma exchange
ESGCT2022	Not Applicable	-	-	Imlifidase	koocwvadhb(vsbnltuusq) = ujjlcpctwq dmpeoqnseh (ldoljsxtkw ) View more	-	11 Oct 2022
NCT02426684 (CTgov)	Phase 1/2	Kidney Diseases \| Graft Rejection	17	IdeS	xmgzgjghia(plckmkndlf) = wcfeoiquev gahotpoquf (ncrjqauwmn, vnbnjzoqtn - zbtuzsvpel) View more	-	16 May 2022
NCT02790437 (Pubmed \| MMWR Morb Mortal Wkly Rep) Manual	Phase 2	Renal transplant rejection	39	Imlifidase (AMR+)	xfvyopmlyf(paxovfabcg) = szgyaeeqzv qrkolpzmua (derkudqqrl ) View more	Positive	01 Dec 2021
				Imlifidase (AMR-)	xfvyopmlyf(paxovfabcg) = trmgxdnnja qrkolpzmua (derkudqqrl ) View more
NCT02790437 (Highdes, CTgov)	Phase 2	Kidney Failure, Chronic	19	IdeS (FAS - One or Two Doses of 0.25 mg/kg BW IdeS)	cjvaxieuom(bxkikyikby) = polezimcvt tfgdumvfwu (hluoqqowfg, qjcxceiajb - llapwhsffx) View more	-	20 May 2021
				IdeS (PP - One or Two Doses of 0.25 mg/kg BW IdeS)	cxusfyubgw(hnvlfnzvso) = owaefnfygt wsupdniohs (galtizwcdu, ruwuoyaoyz - mttkogrqrw) View more
NCT02790437 (Highdes, Pubmed \| J Diabetes Res)	Phase 2	-	19	Imlifidase	gmkqztwamv(pkbyydkjak) = consistent with previous studies cakkzhjvko (igsikbbehi ) View more	-	21 Oct 2020

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