Hansa Biopharma Completes Randomization in Pivotal Phase 3 US ConfIdeS Trial

Rhea-AI Summary

Hansa Biopharma (HNSA) has completed the randomization phase in its pivotal Phase 3 US ConfIdeS trial. This trial evaluates the efficacy of imlifidase in kidney transplantation for highly sensitized patients. The trial involves 64 patients and is conducted across 24 US sites. The primary endpoint is kidney graft function at 12 months, measured by eGFR. Data from this trial will support a Biologic License Application (BLA) to the FDA, expected in H2 2025. Imlifidase, already conditionally approved in Europe under IDEFIRIX® for similar indications, is also being explored for autoimmune diseases and as a pre-treatment in gene therapy.

Positive

• Completion of randomization in Phase 3 trial indicating progress.
• Trial includes 64 highly sensitized kidney transplant patients.
• 24 US sites involved, enhancing trial credibility.
• Primary endpoint focused on kidney graft function at 12 months, a significant measure.
• Supports upcoming Biologic License Application (BLA) to FDA, targeted for H2 2025.
• Imlifidase has conditional marketing approval in Europe, showcasing prior success.
• Potential expansion of imlifidase use in autoimmune diseases and gene therapy.

Negative

• Data submission for FDA approval only expected in the second half of 2025, indicating a potential delay in market entry.
• Outcome and effectiveness of imlifidase in the trial remain uncertain until data is available.
• No immediate impact on revenue or profitability reported from this trial phase.

Medical Research Analyst

The completion of randomization in Hansa Biopharma's ConfIdeS trial marks a significant milestone for the company and the medical field. This Phase 3 trial of imlifidase targets a highly specific patient group – those highly sensitized for kidney transplantation. If successful, this could address a major unmet need, potentially improving the lives of many who struggle with finding compatible donors due to their high levels of antibodies.

From a clinical standpoint, the primary endpoint of kidney graft function at 12 months (measured by eGFR) is critical. eGFR is a widely accepted measure of kidney function and is used to determine how well the kidneys are filtering waste from the blood. A successful outcome here would strengthen imlifidase’s profile, supporting its BLA submission. Considering imlifidase already has conditional approval in Europe, positive results in this trial could significantly bolster its case for full approval in the US.

For retail investors, the potential for accelerated approval means a quicker path to market, which could enhance Hansa’s revenues sooner than later. However, it's essential to recognize the inherent risks in clinical trials; outcomes are not guaranteed and any negative results could impact stock prices adversely.

Financial Analyst

Hansa Biopharma’s announcement is significant from a financial perspective. Completion of patient randomization in the Phase 3 trial brings the company one step closer to potentially filing for a Biologic License Application under the FDA's accelerated approval pathway. This pathway can expedite the availability of treatments that fill an unmet medical need, which can translate to faster revenue generation.

Investors should note that successful trial outcomes and subsequent FDA approval could open up the US market, significantly impacting Hansa’s revenue streams. The US market for kidney transplantation is considerable and the introduction of imlifidase could disrupt current treatment protocols, positioning Hansa as a leader in this niche.

However, investors should also be aware of the financial risks associated with clinical trials. The company may experience increased operational costs associated with the trial and any delays or negative trial outcomes could adversely affect stock performance. Additionally, competition in the biopharmaceutical sector is fierce and Hansa will need to demonstrate clear benefits of imlifidase over existing treatments.

Market Research Analyst

From a market perspective, the successful completion of randomization in Hansa Biopharma’s ConfIdeS trial could position the company strongly within the biopharmaceutical landscape. The specific targeting of highly sensitized kidney transplant patients indicates a focused approach to address a significant unmet need, which could differentiate Hansa in the eyes of healthcare providers and payers.

The market for desensitization treatments in kidney transplantation is not only substantial but also under-served. If imlifidase proves effective, Hansa could secure a first-mover advantage in the US, potentially leading to rapid adoption and market penetration. Additionally, the mention of imlifidase’s potential applications in autoimmune conditions and gene therapy pre-treatment highlights the drug’s versatility, which could further enhance market interest and investor confidence.

However, market entry is not without challenges. The company will need to navigate pricing strategies, reimbursement landscapes and build awareness among clinicians. Educational initiatives and robust clinical data dissemination will be important to drive adoption.

LUND, Sweden, May 31, 2024 /PRNewswire/ -- Hansa Biopharma AB, ("Hansa" or the "Company") (NASDAQ Stockholm: HNSA), today announced recruitment and randomization in its US ConfIdeS trial is complete. ConfIdeS is a pivotal Phase 3 open label, randomized, controlled trial of imlifidase in kidney transplantation. Data from the trial is expected to support a Biologic License Application (BLA) submission under the accelerated approval pathway to the US Food and Drug Administration (FDA) in the second half of 2025.

Søren Tulstrup, President and CEO, Hansa Biopharma said, "The randomization of 64 patients in the ConfIdeS trial is an important step in bringing imlifidase to the US to help address the significant unmet need faced by highly sensitized kidney transplant patients. Our hope is that the ConfIdeS trial will help further validate the role imlifidase may play as a desensitization treatment that enables HLA-incompatible kidney transplantation. We look forward to sharing the data from the ConfIdeS trial in due course."

The ConfIdeS trial is evaluating kidney function in 64 highly sensitized (cPRA ≥99.9%) kidney transplant patients with positive crossmatch against a deceased donor, comparing desensitization using imlifidase with standard of care. A total of 24 US sites are participating in the trial and its primary endpoint is kidney graft function at 12 months, measured by eGFR (estimated Glomerular Filtration Rate).

Imlifidase has been granted conditional marketing approval in Europe 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. Imlifidase is also being studied in autoimmune conditions including anti-glomerular basement membrane (anti-GBM) disease and Guillain-Barré syndrome (GBS) and as a pre-treatment to gene therapy in rare disease patients with pre-existing antibodies.

This is information that Hansa Biopharma AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the contact person set out below, at 13:30 CEST on 31 May 2024.

Contacts for more information:

Evan Ballantyne, Chief Financial Officer
IR@hansabiopharma.com

Stephanie Kenney, VP Global Corporate Affairs
media@hansabiopharma.com

Notes to editors

About highly sensitized patients

Highly sensitized 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.¹ 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.^2,3 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.^2,3 Across the U.S. and Europe, highly sensitized patients comprise around 10-15% of the total of patients on transplant waiting lists.^4,5

About IDEFIRIX^® (imlifidase)

Imlifidase is an antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets and cleaves immunoglobulin G (IgG) antibodies and inhibits IgG-mediated immune response.⁶ 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. The use of IDEFIRIX should be reserved for patients who are unlikely to be transplanted under the available kidney allocation system, including prioritization programs for highly sensitized patients.⁶ IDEFIRIX was reviewed as part of the European Medicines Agency's (EMA) Priority Medicines (PRIME) program, which supports medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options.⁶

Imlifidase is an effective strategy for desensitization of transplant patients with donor-specific anti-HLA (Human Leukocyte Antigens) antibodies (DSAs).⁷ Highly sensitized patients have high levels of these preformed antibodies that can bind to the donor organ and damage the transplant.⁸ 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.7,9-11 Hansa is collecting further clinical evidence and will submit additional efficacy and safety data based on one observational follow-up study and one post-approval efficacy study.

Full product information can be accessed via the initial Summary of Product Characteristics found here.

About kidney failure

Kidney disease can progress to kidney failure or End-Stage Renal Disease (ESRD), identified when a patient's kidney function is less than 15%.¹² ESRD poses a significant health burden, affecting nearly 2.5 million patients worldwide.¹² 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 on transplant waiting lists across the U.S., European Union and United Kingdom.¹³

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 www.hansabiopharma.com and follow us on LinkedIn.

©2024 Hansa Biopharma. Hansa Biopharma, the beacon logo, and IDEFIRIX are trademarks of Hansa Biopharma AB, Lund, Sweden. All rights reserved.

References        

1. 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.        
2. 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.     
3. 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/       
4. OPTN/SRTR 2022 Annual Data Report. HHS/HRSA; 2024. Accessed [May 2024]. http://srtr.transplant.hrsa.gov/annual_reports/Default.aspx
5. SRTR Database and individual assessments of allocation systems    
6. European Medicines Agency. Idefirix^® summary of product characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/idefirix-epar-product-information_en.pdf.
7. Jordan SC, et al. IgG Endopeptidase in Highly Sensitized Patients Undergoing Transplantation. N Engl J Med  2017;377:442-453. DOI: 10.1056/NEJMoa16125        
8. Manook M, et al.  Post-listing survival for highly sensitised patients on the UK kidney transplant waiting list: a matched cohort analysis. Lancet. 2017 Feb 18;389(10070):727-734. doi: 10.1016/S0140-6736(16)31595-1.      
9. 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.   
10. 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.   
11. 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    
12. NIH (2018). What is kidney failure? Available at: https://www.niddk.nih.gov/health-information/kidney-disease/kidney-failure/what-is-kidney-failure.
13. Newsletter Transplant 2022. International figures on donation and transplantation. Available at: Newsletter Transplant – latest edition | Freepub (edqm.eu) [Accessed May 2024].

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SOURCE Hansa Biopharma AB

FAQ

What is the Hansa Biopharma ConfIdeS trial?

The ConfIdeS trial is a pivotal Phase 3 study evaluating imlifidase in kidney transplantation for highly sensitized patients.

How many patients are involved in the ConfIdeS trial?

The ConfIdeS trial involves 64 highly sensitized kidney transplant patients.

When is the data from the ConfIdeS trial expected?

Data from the ConfIdeS trial is expected in the second half of 2025.

What is the primary endpoint of the ConfIdeS trial?

The primary endpoint is kidney graft function at 12 months, measured by eGFR.

What is imlifidase?

Imlifidase is a treatment used for desensitization in kidney transplant patients, conditionally approved in Europe under the name IDEFIRIX®.

Which company is conducting the ConfIdeS trial?

Hansa Biopharma (HNSA) is conducting the ConfIdeS trial.

How many US sites are participating in the ConfIdeS trial?

A total of 24 US sites are participating in the ConfIdeS trial.

What conditions other than kidney transplant is imlifidase being studied for?

Imlifidase is also being studied for autoimmune conditions and as a pre-treatment in gene therapy.