Hansa Biopharma Ab · OMXSTO:HNSA
Hansa Biopharma Ab — Q4 2023 Earnings Presentation
104 slides from Hansa Biopharma Ab's earnings presentation, Feb 2, 2024
Slide 1: Investor Road Show Year-end Report/Q4 2023
Investor Road Show Year-end Report/Q4 2023 Lund, February 2, 2024
Slide 2: Forward-looking statements
Forward-looking statements This presentation may contain certain forward-looking statements and forecasts based on our current expectations and beliefs regarding future events and are subject to significant uncertainties and risks since they relate to events and depend on circumstances that will occ
Slide 3: Table of contents
Table of contents 1. Q4 2023 Business Update 4 2. Company overview 17 3. Imlifidase in kidney transplantations 34 4. Completed and ongoing studies 43 5. Our enzyme technology 56 6. Clinical development programs 65 7. Next generation enzymes 74 8. Gene therapy 78 9. ESG Overview 95 10. Capital Market
Slide 4: Business update Q4'2023
Business update Q4’2023 4
Slide 5: Hansa enters 2024 in a strong position to execute on key priorities
Hansa enters 2024 in a strong position to successfully execute on our key priorities 1 Q4: Strong commercial performance 2 Pipeline: Encouraging read-outs across several indications ü Strong revenue generation in Q4 2023 ü AMR: Full data from AMR phase 2 study • SEK 43m in Idefirix product sales • G
Slide 6: Key strategic priorities
Key strategic priorities Commercialize Idefirix® in Advance our ongoing Expand our IgG-cleaving first indication and markets clinical programs enzyme technology 1 2 3 • Successfully launch • Achieve approval/ • Expand IgG-cleaving Idefirix® in Europe usage of imlifidase in enzyme technology • Secure
Slide 7: Continued progress against our key launch metrics
Continued progress against our key launch metrics led by in-market growth Market Market Patient Transplant Center Development Access Identification Readiness & Use 7 14 28 ~50 - Medical guidelines 9 6 25 implemented on a national Market access secured in Post Approval Study ~50 clinics are Idefirix
Slide 8: Potential to disrupt transplantation care in the U.S. with imlifidase
Potential to disrupt transplantation care in the U.S. with imlifidase ConfIdeS phase 3 trial will further advance potential for imlifidase to address unmet need in desensitization U.S. ConfIdeS ConfIdeS sites cover more than 20% of total transplantation volumes in the U.S1 Phase 3 463 UW Seattle 41
Slide 9: Commercial partnership with NewBridge Pharmaceuticals expands market to MENA
Commercial partnership with NewBridge Pharmaceuticals expands market to Middle East & North Africa (MENA) The MENA region will be the third region outside Europe where Idefirix is commercialized aimed at enabling kidney transplantation in highly sensitized kidney transplant patients GCC Key centers
Slide 10: Advancing HNSA-5487 – a next-gen enzyme for repeat dosing
Advancing HNSA 5487 – a high potential next-gen enzyme for repeat dosing Broaden the IgG free window Rapidly cleaves IgG and could potentially create a HNSA-5487 longer IgG-low period Engineered for lower immunogenicity Address unmet need in autoimmune disease Short and long-term Powerful 5487 IgG c
Slide 11: Potential indication landscape for HNSA-5487
Potential indication landscape for HNSA-5487 and reasons to believe First in Human Study Results Long interval Short interval redosing redosing ü Administration was safe and well in combination with humoral inhibitor tolerated AAV Gene therapy Longer-term oncolytic virus ü PD showed a fast and compl
Slide 12: Strong momentum across the pipeline in areas of high unmet need
Strong momentum across the pipeline in areas of high unmet need Phase 1 Phase 2 Phase 3 HNSA-5487 (Lead from NiceR) Antibody Mediated Rejection (AMR) US ConfIdeS Study in kidney • Encouraging first read-out • Primary endpoint met • 104 patients enrolled; Completed • Ongoing collection of Completed •
Slide 13: Strong revenue generation in Q4 2023
Strong revenue generation in Q4 2023, including SEK 43m in Idefirix product sales Product sales improved +113% vs Q4 2022 and +163% vs Q3 2023; Growth driven by uptake in U.K., Spain, Germany Revenue (Q/Q) Q4'22 Q1'23 Q2'23 Q3'23 Q4'23 50.4 Product sales SEKm 36.7 30.8 24.2 43.3 22.8 20.3 29.6 14.3
Slide 14: Continued investments in commercialization and R&D activities
Continued investments in commercialization and R&D activities SG&A expenses (Q/Q) R&D expenses (Q/Q) Operating loss (Q/Q) Q4'22 Q1'23 Q2'23 Q3'23 Q4'23 Q4'22 Q1'23 Q2'23 Q3'23 Q4'23 Q4'22 Q1'23 Q2'23 Q3'23 Q4'23 -146 -84 -92 -93 -96 -182 -176 -103 -106 -108 -202 -112 -115 -228 SEKm SEKm SEKm -129 SG
Slide 15: Operations financed into 2025
With current cash position and projected burn-rate, Hansa’s operations are financed into 2025 Operating cash flow (Q/Q) Cash & short-term investments (Q/Q) Number of employees (Q/Q) Q4'22 Q1'23 Q2'23 Q3'23 Q4'23 1,496 168 168 -110 -183 -173 -207 -193 1,287 SEKm 162 1,103 Employees 159 SEKm 908 732 O
Slide 16: 2023 achievements and upcoming milestones 2024/25
2023 achievements and upcoming milestones 2024/25 2023 2024 2025 Q4 2023 HNSA-5487 (Lead NiceR candidate): GBS Phase 2: - U.S. ConfIdeS (Kidney tx) Phase 3: ✓ - Outcome of comparative efficacy analysis High-level data readout from Phase 1 BLA submission Long-term follow-up (Kidney tx): Genethon Crig
Slide 17: Company overview
Company overview 17
Slide 18: Hansa Biopharma today
Hansa Biopharma today A successful track record and a promising future… A validated technology Broad clinical pipeline § Commercial stage biotech company § Imlifidase being investigated in seven § Approval in kidney transplantation (EU) ongoing clinical programs in § Market Access in 14 European mar
Slide 19: We are building a global leader in rare diseases
We are building a global leader in rare diseases Today Tomorrow We are launching our first commercially We envision a world where patients approved product for enablement of kidney with rare immunologic transplantation in Europe* diseases and conditions can lead long and healthy lives 19 * European
Slide 20: Hansa Biopharma's history
Hansa Biopharma’s history The EU Commission grants Provisional approval conditional approval for Idefirix® in received in Australia; highly sensitized kidney transplant Marks first time Idefirix has patients in Europe been approved in both Market access living and deceased IPO 2. Clinical developmen
Slide 21: Key milestones achieved during the last 12 months
Key milestones achieved during the last 12 months Collaboration with Genethon Provisional approval for to evaluate imlifidase as pre- Idefirix® in Australia in Pricing and treatment to gene therapy both living and reimbursement granted GNT-0003 in Crigler-Najjar deceased donors' for Idefirix® for us
Slide 22: Imlifidase
Imlifidase a novel approach to eliminate pathogenic IgG Origins from a bacteria A unique IgG antibody-cleaving enzyme Inactivates IgG in 2-6 hours Streptococcus pyogenes § Species of Gram-positive, § Interacts with Fc-part of IgG with extremely high specificity § Rapid onset of action that inactivat
Slide 23: Our Business model
Our Business model Leveraging our technology platform to develop new therapies targeting rare diseases with unmet medical need across a range of indications Growth engine Value chain Commercialization Leveraging our We are controlling Build-up of Indications Multiple proprietary antibody the full va
Slide 24: Potential indication universe
Gene therapy pre-treatment Potential indication universe (partnership opportunities) Crigler- Transplantation and post transplantation … … Najjar (Own commercial infrastructure in EU/US) Limb- Pompe Autoimmune Girdle … Lung Heart (LGMD) disease indications Duchenne Chronic AMR First generation antib
Slide 25: Key strategic priorities
Key strategic priorities Commercialize Idefirix® in Advance our ongoing Expand our IgG-cleaving first indication and markets clinical programs enzyme technology 1 2 3 • Successfully launch • Achieve approval/ • Expand our IgG- Idefirix® in Europe usage of imlifidase in cleaving enzyme follow-on indi
Slide 26: Our culture is driven by people passionate about making changes
Our culture is driven by people passionate about making changes Purpose Diverse and Skilled and Motivated driven culture international experienced team workforce For fourth consecutive year Hansa is certified as a Helping patients with rare diseases serves ~45% >50% Great Place to Work® with 100% pa
Slide 27: Experienced Board and Executive Committee
Experienced Board and Executive Committee Extensive experience from the global healthcare industry Executive Committee Board of Directors Søren Tulstrup Hitto Kaufmann Peter Nicklin Hilary Malone President & CEO (2018) CSO (2023) +30 years in the Healthcare sector +20 years in R&D Board Member (2021
Slide 28: Hansa Biopharma's Governance Structure
Hansa Biopharma’s Governance Structure Risk Executive management Committee Compliance Board of Directors Audit Remuneration Scientific U.S. Committee Committee Committee Committee Nomination External auditors General Meeting Committee Electing / Appointing Reporting / Informing 28
Slide 29: Strong technology protection through patents and orphan drug designations
Strong technology protection through patents and orphan drug designations Patent coverage out to 2035 in key markets Orphan drug designation § Our lead product, imlifidase, is protected by six patent § Orphan drug designation is granted to drugs intended for rare diseases families including both gra
Slide 30: Hansa Biopharma is financed into 2025
Hansa Biopharma is financed into 2025 Financing to support the continued development of Hansa’s antibody-cleaving enzyme technology platform and commercial preparations toward regulatory approval in the U.S. Raised Financing Raised SEK 1.1bn SEK SEK 400m (2020) ~750m (Dec, 2022) (July 2022) 1,600 Ca
Slide 31: Mid-term financial priorities
Mid-term financial priorities Our key financial priorities over the coming years will be focused on ensuring a successful European launch of Idefirix®, while targeting mid-term product profitability With the recent financing Hansa is fully financed into 2025 We expect to use our current cash positio
Slide 32: Strategic plan to build U.S. presence
Strategic plan to build U.S. presence ahead of potential regulatory approval and commercial launch of imlifidase in the US Three shots on goal to enter important US market Critical functions to be established • Small and agile team with deep clinical and U.S. marketplace expertise • Comprehensive fu
Slide 33: An exciting journey ahead!
An exciting journey ahead! Our future Hansa Biopharma is a recognized global leader in rare diseases across multiple broad therapeutic areas with several market leading products and Key milestones to be achieved a highly valuable pipeline of late- • Expand Idefirix® label in transplantation and in o
Slide 34: Imlifidase in kidney transplantation
Imlifidase in kidney transplantation 34
Slide 35: Long-term market uptake of Idefirix depends on early experiences
The long-term market uptake of Idefirix is highly dependent on successful early experiences in patients For decades, medical practice (SoC) in transplantation Idefirix addresses the limitations of these other has been predicated on compatibility as modalities modalities and is the first and only app
Slide 36: Idefirix is the first and only approved drug in Europe for desensitization
Idefirix® is the first and only approved drug in Europe for desensitization of highly sensitized kidney transplant patients Inability to match or effectively desensitize patients remains a barrier for transplantation in highly sensitized patients. Between 80,000 and 100,000 kidney transplant patient
Slide 37: Unique market position requires consideration of sales and transplant cycle
The unique market position of Idefirix® requires consideration of both the sales- and the transplant cycle Sales and transplant cycle adds complexity and time to patient treatment Excellence revolves around four Sales Cycle Transplant Cycle strategic themes Price and reimbursement; Desensitization W
Slide 38: Encouraging patient outcome in new markets
Encouraging patient outcome in new markets following imlifidase-enabled kidney transplantations First living donor 51-year-old highly 43-year-old highly transplantation in sensitized male patient sensitized female kidney Australia enabled by transplanted at the transplant patient was imlifidase was
Slide 39: Only 1 in 4 patients are offered access to a lifesaving transplantation
Only 1 in 4 patients are offered access to a lifesaving transplantation Up to 15% of patients are highly sensitized ~50,000 transplantations done annually in Europe and the U.S. Breakdown of the kidney transplant waitlist in U.S. and EU U.S. annual transplantations Total YoY 2022 20446 5864 26,310 3
Slide 40: Market Access obtained in 14 markets
Market Access obtained in 14 markets covering markets with 3/4 of transplant volumes in Europe Positive reimbursement decision received in Slovenia as of February 1, 2024 Health Technology Assessments (HTA) dossiers submitted Reimbursed Early Access Program 3,192 transplants1 Pricing & reimbursement
Slide 41: Commercial sales uptake
Scaling Idefirix® globally as we transform the desensitization treatment landscape and advance a new way of transplanting patients 1 Build the foundation for Idefirix® 2 Expanding internationally 3 Potential label expansion ü ✓ Commercialize in early-launch countries • Leverage experience to scale I
Slide 42: Eurotransplant Desensitization Program
Eurotransplant Desensitization Program set to transform desensitization across eight European membership countries First patient treated in the new Eurotransplant Desensitization Program; Second wave of patients identified for treatment through the Program Eurotransplant zone 8 member countries Euro
Slide 43: Completed and ongoing studies in kidney transplantation
Completed and ongoing studies in kidney transplantation 43
Slide 44: Long term data confirms sustained benefit at five years
Long term data confirms sustained benefit at five years in graft survival and overall patient survival Stable long-term outcomes on graft Results is a continuation of the 3-year data survival and patient survival • After 5 years graft survival (death censored) was 82%, in line with outcomes seen at
Slide 45: Potential to disrupt transplantation care in the U.S. with imlifidase
Potential to disrupt transplantation care in the U.S. with imlifidase ConfIdeS phase 3 trial will further advance potential for imlifidase to address unmet need in desensitization Involved ConfIdeS sites cover more than 20% of U.S. ConfIdeS total transplantation volumes in the U.S1 Phase 3 463 UW Se
Slide 46: Imlifidase may complement the US Kidney Allocation System
Imlifidase may complement the US Kidney Allocation System, as thousands of patients are still unlikely to find a match KAS was revised in the Factors impacting the KAS score1 U.S. in 2014 to increase § Waiting time equity of transplantation. § Age § Transplantation history However, thousands of § Se
Slide 47: U.S. ConfIdeS study
U.S. ConfIdeS study: Potential to disrupt transplantation care in the U.S. with imlifidase U.S. trial design 64 highly sensitized kidney patients with the highest unmet medical need Timeline § Patients with a cPRA score of ≥99.9% will be enrolled § First patients enrolled at Columbia University, NYC
Slide 48: Idefirix receives provisional approval in Australia
Idefirix receives provisional approval in Australia First market to approve use in transplants from both living and deceased donors Transplant statistics First living donor transplantation Kidney transplantation landscape in Australia ~15,200 patients suffer First living donor from ESRD and need tra
Slide 49: First patient treated in post-authorization efficacy study
First patient treated in post-authorization efficacy study (PAES) of Idefirix® (imlifidase) in highly sensitized kidney transplant patients The study will provide further important insights regarding Idefirix® desensitization treatment of highly sensitized kidney transplant patients An open-label Ph
Slide 50: Study 01 results
The 01 study results Study 01 Data showed complete removal of Phase 1 IgG and a good tolerability profile Efficacy ü Rapid degradation of IgG in serum in subjects dosed with 0.12 and 0.24 mg/kg imlifidase. CLINICALTRIALS.GOV ID Imlifidase had full effect within 6 hours. The entire IgG pool was conve
Slide 51: Study 02 results
Study 02 The 02 study results Phase 2 Data showed that 1-2 doses of imlifidase at 0.25 mg/kg BW resulted in HLA antibody levels acceptable for transplantation1 CLINICALTRIALS.GOV ID NCT02224820 ü Imlifidase is well tolerated in patients with chronic kidney disease SUBJECTS ü Efficacy results strongl
Slide 52: Study 03 proved safety and efficacy
The 03 study proved safety and efficacy Study 03 HLA antibodies at acceptable levels; enabling transplantation in all patients Phase 2 Analysis of IgG in patient serum Analysis of complement binding HLA CLINICALTRIALS.GOV ID before and after imlifidase treatment antibodies before and after imlifidas
Slide 53: Study 04 results
Study 04 The 04 study results Phase 2 Study proved safety and efficacy with Cedar Sinai’s standard protocol (rituximab and IVIg) CLINICALTRIALS.GOV ID Cedar’s desensitization protocol in NCT024226684 Graft function (eGFR) post six months combination with imlifidase SUBJECTS 17 Patients (US) DOSES/FO
Slide 54: Study 06 results
The 06 study results Study 06 Study showed proved safety and efficacy in making highly Phase 2 sensitized patients eligible for kidney transplantation Graft function (eGFR) post imlifidase DSA level pre-dose and post imlifidase CLINICALTRIALS.GOV ID NCT02790437 SUBJECTS 18 Patients (US+Sweden+France
Slide 55: Completed studies with imlifidase in transplantation
Completed studies with imlifidase in transplantation SUBJECTS/ STATUS/ STUDY STUDY DESIGN PRIMARY ENDPOINT SECONDARY ENDPOINTS COUNTRY PUBLICATION • Randomized placebo-controlled dose- • Efficacy in IgG cleavage, the Study 01 29 subjects Complete Phase 1 escalation study with 29 (20 active plus 9 •
Slide 56: Our antibody cleaving enzyme technology
Our antibody cleaving enzyme technology 56
Slide 57: Broad clinical pipeline in transplantation, autoimmune diseases, and gene therapy
Broad clinical pipeline in transplantation, autoimmune diseases, and gene therapy Research/ Marketing Project Indication Preclinical Phase 1 Phase 2 Phase 3 Authorization Marketed Partner Next Anticipated Milestone EU: Additional agreements around EU: Kidney transplantation in highly sensitized reim
Slide 58: Development of IgG-modulating technologies
Development of IgG-modulating technologies Mechanisms can be both complementary and competing Imlifidase – IgG-cleaving enzyme PLEX, plasmapheresis, B-cell depleting mAbs Deactivates IgG within 2-6 F(ab’)2 Lowering antibody levels hours through enzymatic immunoadsorption through B-cell elimination c
Slide 59: Imlifidase mode of action
Imlifidase mode of action Novel approach to effectively eliminate pathogenic IgG Proven mechanism of action Inactivation of IgG in human serum • Interacts with Fc-part of IgG with extremely high specificity • Rapid onset of action that takes down IgG below detectable • Cleaves IgG at the hinge regio
Slide 60: Our unique antibody cleaving enzyme technology
Our unique antibody cleaving enzyme technology may have relevance across a range of indications Targeting rare IgG mediated diseases Auto-immune diseases Transplantation Anti-GBM disease paves the way for Shaping a new standard for desensitization will development in other autoimmune diseases help e
Slide 61: The technology platform is the primary basis for achieving our vision
The technology platform is the primary basis for achieving our vision IgG levels after imlifidase treatment in in highly sensitized patients Targeting rare IgG mediated diseases and conditions – First 30 days IgG Key opportunities: • Expanding into new indications • Reduce immune response to IgG-cle
Slide 62: Our IgG antibody-cleaving enzyme, imlifidase
Our IgG antibody-cleaving enzyme, imlifidase Origins from Streptococcus pyogenes Imlifidase consists of 311 amino acids • Cysteine protease derived from an Immunoglobulin G (IgG)- degrading enzyme of Streptococcus pyogenes • Contains only one cysteine - no disulfide bridges • Monomeric protein with
Slide 63: Imlifidase is a lyophilized product formulation
Imlifidase is a lyophilized product formulation Shelf life of 18 months at 2-8o Celsius storage; Ongoing stability studies indicate a shelf life of at least 24 months. Imlifidase will be infused in 15 minutes § The product for commercial supply will be a lyophilized (cold chain) powder concentrate (
Slide 64: Manufacturing process
Manufacturing process Hansa has close collaborations with highly experienced European based third party CMOs Drug substance production process (API) Drug product production process (upscaling) Northway Biotech Baxter Fermentation/ Protein purification Protein purification cont. Filling Thawing of dr
Slide 65: Clinical development programs
Clinical development programs 65
Slide 66: Autoimmune attacks
Autoimmune attacks A result of when the body’s immune system by mistake damages its own tissue Blood Brain Autoimmune hemolytic anemia, Multiple sclerosis, Immune thrombocytopenia Neuromyelitis optica Over 100 different Thyroid GI tract Hashimoto’s disease, Crohn’s disease types of Graves’ disease A
Slide 67: Hansa's enzyme technology in autoimmune diseases
Hansa’s antibody cleaving enzyme technology may have relevance in several autoimmune diseases where IgG plays an important role in the pathogenesis Rapidly progressive Neurological Skin Blood ~1 000* patients13 glomerulonephritis disorders disorders disorders AHA ~1 000 patients*1 <1 000 patients*8
Slide 68: Anti-GBM, a rare acute autoimmune disease
Anti-GBM, a rare acute autoimmune disease Results from Phase 2 study of imlifidase in anti-GBM Incidence Standard of Care disease published in Journal of American Society of § Plasma Exchange 1.6 § Cyclophosphamide (CYC) Nephrology (JASN)3 § Glucocorticoids 10 out of 15 patients were dialysis indepe
Slide 69: New pivotal phase 3 trial with imlifidase in anti-GBM
New pivotal phase 3 trial with imlifidase in 50 anti-GBM patients to evaluate kidney function after six months Illustrative Study Design Untreated § Open-label, controlled, randomized, multi-center phase 3 trial evaluating renal function in patients with severe anti- Start of PLEX (Multiple rounds)
Slide 70: Phase 2 trial in Guillain-Barré Syndrome
Imlifidase demonstrated positive safety, tolerability, and early efficacy outcomes in phase 2 trial in Guillain-Barré Syndrome (GBS) Today’s standard of care, IVIg or PLEX Potential with imlifidase Incidence Standard of Care Illustrative Illustrative § Intravenous immune 1-2 globulin (IVIG) or Paral
Slide 71: New investigator-initiated phase 2 study in ANCA-associated vasculitis
New investigator-initiated phase 2 study in ANCA-associated vasculitis - a group of autoimmune diseases characterized by inflammation of blood vessels with very few treatment options today Incidence Standard of Care Vasculitis means The investigator-initiated trial ~3 in 100,000 annually across EU/U
Slide 72: Long term graft survival is challenged by AMR episodes
Long term graft survival is challenged by antibody mediated rejection (AMR) episodes following kidney transplantation Incidence Standard of Care Potential with imlifidase vs. PLEX in AMR Illustrative Acute AMR episodes occur in § Intravenous immune globulin (IVIg) or 5-7% of annual kidney transplant
Slide 73: Imlifidase met primary endpoint in phase 2 trial in AMR
Imlifidase met primary endpoint in phase 2 trial in patients with AMR episodes following kidney transplantation Remaining donor specific antibody levels, within 5 days Primary endpoint was the maximum reduction in DSA level at any time point during the 5 days following the start of treatment § Patie
Slide 74: Next generation enzymes
Next generation enzymes 74
Slide 75: Encouraging first-in-human data of HNSA-5487
Encouraging data from the first-in-human trial of HNSA-5487 as we continue to explore the potential of our next-gen enzyme for repeat dosing An enzyme with lower immunogenicity could potentially enable repeat dosing for innovative treatment approaches in a broad range of indications benefiting patie
Slide 76: Short interval redosing with HNSA-5487
Short interval redosing with HNSA-5487 could potentially prolong the IgG-low period Short interval 5487 redosing Enabling treatments Short-term treatment Hypothetical model through IgG-low period in autoimmune diseases 1st dose Repeat dosing of HNSA-5487 can HNSA-5487 has potential to more HNSA-5487
Slide 77: Long interval redosing with HNSA-5487
Long interval redosing with HNSA-5487 in combination with humoral inhibitor in relapsing autoimmune diseases and chronic AMR HNSA-5487 rapidly cleaves Long interval 5487 repeat dosing IgG - chronic humoral Hypothetical model inhibition adds duration of effect § HNSA-5487 rapidly HNSA- Humoral Humora
Slide 78: Gene Therapy
78 Gene Therapy 3% HANSAT BIOPHARMA
Slide 79: Exploring opportunities in gene therapy
Exploring opportunities in gene therapy Exploring the opportunities in systemic administration of gene therapy for our unique antibody cleaving enzyme platform to potentially enable gene therapy treatment in NAb+ patients A Encouraging Significant Partnership revolutionary pre-clinical unmet need st
Slide 80: Tropism and target tissue
Tropism and target tissue AAV subtypes targets different tissues AAV 4 & 8 Brain (local target) ~1x1012 vg AAV 1, 2 & 5 Eye (local target) ~1x1011 vg AAV 3, 7 & 8 AAV 6, 7, rh74 Liver (systemic) ~1x1014 vg Muscle (systemic) ~1x1015 vg Target tissues Dose of gene therapy (vg) 80
Slide 81: Pre-existing antibodies towards AAVs limit gene therapy
Pre-existing antibodies towards AAVs are a limiting factor in Gene Therapy and excludes patients from clinical trials AAVs, the delivery system of Gene Therapy Prevalence of NAbs in AAVs AAV 1 CNS, Eye, Skeletal muscle Up to 70% § Wildtype Adeno Associated Viruses (AAV) belong to the family of parvo
Slide 82: Imlifidase may potentially eliminate NAbs in gene therapy
Neutralizing antibodies (Nabs) are immunological barriers in gene therapy; imlifidase may potentially eliminate Nabs Between approximately 5%-70%1,2 of patients considered for gene therapy treatment carry neutralizing anti-AAV antibodies forming a barrier for treatment eligibility 1 Antibodies preve
Slide 83: Systemic gene therapy is an emerging opportunity
Systemic gene therapy is an emerging opportunity with a focus on the potential to correct diseases Preclinical programs with Sarepta, AskBio and Genethon Ongoing clinical study with Sarepta causing genes in rare monogenic diseases AAV8 etc Potential gene therapy indications NAbs: up to ~60% (current
Slide 84: Global exclusive agreements with three partners in gene therapy
Global exclusive agreements with three partners in gene therapy To develop and promote imlifidase as pre-treatment ahead of gene therapy in select indications Partner Access to key resources Indication exclusivity Collaborative research, development and commercialization § World leader within gene D
Slide 85: Global and exclusive agreement with Sarepta Therapeutics
Global and exclusive agreement with Sarepta Therapeutics to develop and promote imlifidase as pre-treatment ahead of gene therapy in select indications Indication exclusivity: Hansa’s key resources Sarepta’s key resources • Duchenne Muscular • Imlifidase validated with positive clinical efficacy • W
Slide 86: Imlifidase pre-treatment enhances transduction and expression in NHPs
Imlifidase pre-treatment decreases pre-existing antibodies and enhances transduction and transgene expression in NHPs Transduction† Expression in Skeletal Muscle‡ 1.5 ns vg/nucleus normalized to Cohort 1 * 1.0 eGFP expression (%Area) 50 (a.u.) 40 0.5 30 20 10 0.0 0 Cohort 3: Cohort 4: Cohort 5: Coho
Slide 87: Duchenne muscular dystrophy is progressive and causes irreversible muscle damage
Duchenne muscular dystrophy (DMD) is progressive and causes irreversible muscle damage and loss of function Incidences DMD signs at early age, with most patients using a wheelchair by age 12 1 in 3,500 to 5,000 male births worldwide ~14% have pre-existing IgG antibodies to rh74 0 1 2 3 5 10 12 15 +2
Slide 88: Sarepta's EMBARK data
Sarepta’s EMBARK (SRP-9001-301) data fails on meeting primary endpoint, but secondary endpoints favors ELEVIDYS Data from EMBARK will be used to file for extended label beyond 4–5-year-olds Study design Sarepta believes data support label expansion following results and discussions with FDA Phase 3,
Slide 89: Limb-girdle muscular dystrophy
Limb-girdle muscular dystrophy (LGMD) is a group of diseases that cause weakness and wasting of the muscles Incidences Girdle Muscles SRP-9003 β-sarcoglycan (SGCB) gene therapy for treatment of LGMD2E 1.63 per 100,000 individuals; Initiation of VOYAGENE over 30 subtypes exist, and both Arm and gende
Slide 90: Collaboration with AskBio in Pompe disease
Collaboration with AskBio to evaluate imlifidase in gene therapy targeting Pompe disease Feasibility program to evaluate imlifidase as pre-treatment ahead of gene therapy in Pompe disease for patients with pre-existing neutralizing antibodies (NAbs) to adeno-associated virus (AAV) Fully owned subsid
Slide 91: Pompe disease
Pompe disease, an ultra-rare disease is caused by the deficiency of an enzyme called alpha-glucosidase (GAA) Incidences Late Onset Pompe Disease An ultra-rare indication impacting 1 in 40,000 births or ~200 cases per year across US and Europe Respiratory § Respiratory failure § Diaphragm weakness, s
Slide 92: Collaboration with Genethon in Crigler-Najjar Syndrome
Collaboration with Genethon to evaluate imlifidase in gene therapy targeting Crigler-Najjar Syndrome Hansa’s key resources and deliverables Genethon’s key resources and deliverables + § Imlifidase validated with positive clinical efficacy and safety data as § A pioneer in the discovery and developme
Slide 93: Crigler-Najjar syndrome
Crigler-Najjar (CN) syndrome is an ultra-rare hereditary disorder caused by deficiency in UGT1A1 (liver enzyme) Incidences Build-up of free bilirubin in serum and tissue can become toxic in the brain An ultra-rare indication impacting 1 0.6-1 per 1,000,000 newborns around the world 1,2 Bilirubin is
Slide 94: Feasibility program with Genethon's gene therapy
Feasibility program to evaluate imlifidase as pre-treatment to Genethon’s gene therapy in patients with severe Crigler-Najjar syndrome Study design and timeline Evaluation of safety and efficacy of “GNT0003” in seropositive § Study expected in a small patient population to AAV8 patients pre-treated
Slide 95: ESG Overview
ESG Overview 3% HAN Sar BIOPHARMA
Slide 96: Committed to driving our business forward sustainably
At Hansa we are committed to driving our business forward in a sustainable way guided by three strategic ESG principles Healthy people Address unmet medical need and ensure equitable access to care Healthy business Make a difference by operating an ethical, transparent and responsible business and c
Slide 97: Formalising our ESG approach
Formalising our ESG approach At Hansa, we have always strived to achieve sustainable business practices. We are now formalizing our approach to sustainability and ESG issues, starting with identifying our key material focus areas. ESG context Stakeholder dialogue Key material aspects Our ESG approac
Slide 98: Our key ESG material aspects
Our key ESG material aspects Environment Social Governance Climate & waste impacts of Unmet needs and Putting patients first Third-party risks Safety, efficacy and ethics production and logistics equity in health In the biopharma industry, We diligently select new To build a successful Hansa’s envir
Slide 99: UN Sustainable Development Goals
UN Sustainable Development Goals The Sustainable Development Goals (SDGs) were adopted by all UN Member States in 2015 as a universal call to action to end poverty, protect the planet and ensure that all people enjoy peace and prosperity by 2030. They have since become a gold standard for sustainabi
Slide 100: Capital Markets
Capital Markets 3% HAN Sar BIOPHARMA 100
Slide 101: Ownership in Hansa Biopharma
Ownership in Hansa Biopharma Top 10 shareholders as per December 31, 2023 Classification of ownership as per June 30, 2023 Ownership by region Ownership by type Name No. of shares Ownership Redmile Group, LLC 9,653,214 18.3% 22% Institution s & funds 27% Nexttobe AB 2,155,379 4.1% Sweden Oth er inc
Slide 102: Company collected consensus
Company collected consensus Consensus is based on a collection of analyst estimates pre-Q4 2023 report (Dec 2023) Patient uptake, EU Revenue, SEKm Price Target, SEK WACC Q4’23e FY’23e FY’24e FY’25e Q4’23e FY’23e FY’24e FY’25e Average 80 12% 14 38 74 124 44 134 229 381 Median 90 13% 12 37 65 106 39 1
Slide 103: Contact our Investor Relations and Corporate Affairs team
Contact our Investor Relations and Corporate Affairs team Contact Calendar and events Feb 6, 2024 Aktiespararna, Falkenberg Klaus Sindahl Feb 8, 2024 Frankfurt MidCap Seminar, Frankfurt VP, Head of Investor Relations Feb 14, 2024 Redeye Cell Therapy & Growth Day, Stockholm Mobile: +46 (0) 709-298 26
Slide 104: HANSAT
MZ HANSAT BIOPHARMA