Swedish Orphan Biovitrum Ab · OMXSTO:SOBI
Swedish Orphan Biovitrum Ab — Investor Day (July 2023)
38 slides from Swedish Orphan Biovitrum Ab's investor day presentation, Jul 19, 2023
Slide 1: Sobi KOL investor event: The journey for Vonjo
Sobi KOL investor event The journey for Vonjo® 19 July 2023
Slide 2: Forward looking statement
Forward looking statement This presentation may not be published, distributed or transmitted in the United States, Canada, Australia, Japan or any other jurisdiction in which the distribution, release or publication would be unlawful. This presentation does not constitute an offer of securities for
Slide 3: Agenda and presenters
Agenda and presenters Guido Oelkers, Chief Executive Officer Opening remarks Swedish Orphan Biovitrum Overview of Myelofibrosis John Mascarenhas, Professor of Medicine and Vonjo Icahn School of Medicine at Mount Sinai Emerging Insights & Sarah Buckley, Medicine Development Lead Future Potential of P
Slide 4: Vonjo is part of our journey to build the leading rare haematology portfolio
Vonjo is part of our journey to build the leading rare haematology portfolio Leading products Only oral TPO-RA First in class C3 Safe and First and only in Haemophilia A with no food inhibitor and only efficacious CD-19 JAK1-sparing and B restrictions and sub-cutaneous ADC in severe inhibitor that c
Slide 5: Well-prepared CTI integration capturing Vonjo's value
Well-prepared CTI integration capturing Vonjo’s value Positive and smooth first two weeks focused on seamless continuation of Vonjo operations Life-cycle management planning initiated for geographic expansion and extended indications Thoughtful strategy to maximize Vonjo’s 1+1=3 potential and synerg
Slide 6: Agenda and presenters
Agenda and presenters Guido Oelkers, Chief Executive Officer Opening remarks Swedish Orphan Biovitrum Overview of Myelofibrosis John Mascarenhas, Professor of Medicine and Vonjo Icahn School of Medicine at Mount Sinai Emerging Insights & Sarah Buckley, Medicine Development Lead Future Potential of P
Slide 7: Myelofibrosis is a blood cancer with splenomegaly and abnormal blood counts
Myelofibrosis is a blood cancer with clinical features including splenomegaly and abnormal blood counts Abnormal haematopoiesis: driven by mutations affecting the JAK/STAT pathway1 Decreased Decreased Blood Blood Count Count Splenomegaly: resulting from bone marrow fibrosis and extramedullary hemato
Slide 8: Symptoms and complications impact QoL and prognosis of myelofibrosis patients
Symptoms and associated disease complications impact QoL and prognosis of myelofibrosis patients Symptoms Complications Symptoms of MF may go unrecognized in early disease, but with progression they can lead to reduced QoL, functional status, and activities of daily living1-3 20% risk of leukemic tr
Slide 9: Myelofibrosis is characterised by activated JAK-STAT signalling and inflammation
Myelofibrosis is characterised by activated JAK-STAT signalling and inflammation Different pathways involved in myelofibrosis Myddosome/IRAK1 JAK-STAT Pathway1-5 Pathway5-6 JAK1 JAK2 IRAK1 STAT STAT NFκB STAT3 Haematopoiesis Proinflammatory cytokines, Proinflammatory cytokines (dysregulated in MPNs)
Slide 10: Proliferative and cytopenic MF are clinically and biologically different
Proliferative and cytopenic MF are clinically and biologically different and present across a spectrum The Phenotypic Spectrum of Myelofibrosis1,2 Proliferative Myelofibrosis Cytopenic Myelofibrosis Lab values Lower blood counts, increased Normal or elevated blood counts (clinical presentation)1,2 c
Slide 11: Cytopenias are a common feature of MF progression and JAK1/2 inhibitor side effect
Cytopenias are a common feature of MF progression and a frequent side effect of JAK1/2 inhibitors Thrombocytopenia Prevalence of moderate-to-severe Patients with ~70% thrombocytopeniaa in patients with thrombo- ~45% cytopenia (%) MF rises over time1-3✝ ~25% Time from diagnosis At diagnosis 1-year po
Slide 12: Patients with severe cytopenias have significantly shorter overall survival
Myelofibrosis patients with severe cytopenias have significantly shorter overall survival Thrombocytopenia Anaemia OS in patients with MF by platelet count1 OS in patients with MF by degree of anaemia2 Worst survival in cytopenic myelofibrosis patients1,2 Patients frequently have more than one cytop
Slide 13: Symptom burden is also worse in patients with thrombocytopenia
Symptom burden is also worse in myelofibrosis patients with thrombocytopenia Symptom burden in myelofibrosis patients with thrombocytopenia1 10.0 No or mild thrombocytopeniaa Symptom Severity (MPN-SAF score) 9.0 Moderate-to-severe thrombocytopeniab 8.0 7.0 6.0 * 5.0 † † * † 4.0 * † * * * 3.0 2.0 * 1
Slide 14: Pacritinib is the only approved JAK inhibitor studied in platelets <50 x 10^9/L
Pacritinib is the only approved JAK inhibitor studied in MF patients with platelet counts <50 x 109/L at baseline Pacritinib Pacritinib is indicated in the US for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythaemia vera or post-essential thrombocythemia) m
Slide 15: Pacritinib is a JAK1-sparing JAK2 inhibitor acting on multiple pathways
Pacritinib is a JAK1-sparing JAK2 inhibitor that acts on multiple pathways involved in myelofibrosis Myddosome/ BMP/ACVR1 JAK-STAT Pathway IRAK Pathway Pathway* JAK1 JAK2 IRAK1 ACVR1 STAT STAT NFκB STAT3 SMAD Not inhibited Inhibited by pacritinib * Published data on file. This pathway is not current
Slide 16: Pacritinib received accelerated FDA approval in February 2022 based on PERSIST-2
Pacritinib received accelerated FDA approval in February 2022 based on data from PERSIST-2 PERSIST-2 study design1 Patient Population Randomization Pacritinib 400 mg QD* n=104 Co-Primary Endpoints†: • PMF, PET-MF, PPV-MF • 1:1:1 • ≥35% spleen volume • DIPSS intermediate- or • N = 311 reduction at We
Slide 17: Baseline demographics
Baseline demographics Characteristics PERSIST-2 patient demographics (ITT population; N=311)1 Median age 68 years (range 32 to 91) Gender 55% male, 45% female Ethnicity 86% Caucasian, 14% non-Caucasian 55 x 109/L Median baseline platelet count • 45% of patients had Grade ≥3 thrombocytopenia (platele
Slide 18: Approval based on spleen volume reduction in severely thrombocytopenic MF patients
Pacritinib approval was based on spleen volume reduction in severely thrombocytopenic MF patients Percentage of patients with platelet counts <50 x 109/L achieving Median percent change from baseline in spleen volume at Week 24 in ≥35% SVR from baseline to Week 24 (ITT Efficacy Population)1 patients
Slide 19: Pacritinib was observed to be generally well tolerated
Pacritinib was observed to be generally well tolerated Key safety overview from PERSIST-21 Most common adverse events of any grade occurring in ≥20% of patients (N=106) treated with PAC 200mg BID were diarrhoea (45%), thrombocytopenia (32%), nausea (30%), anaemia (23%) and peripheral oedema (19%) •
Slide 20: Post hoc risk-adjusted safety analysis showed favourable safety profile
A post hoc risk-adjusted safety analysis showed favourable safety profile for pacritinib 200 mg BID Events per 100 patient-years are calculated as 100 times the number of patients with an event divided by the cumulative time on treatment for each patient until the first adverse event for patients wi
Slide 21: The confirmatory Phase 3 PACIFICA trial is ongoing
The confirmatory Phase 3 PACIFICA trial is ongoing PACIFICA Study Design1-3 Pacritinib Key Eligibility Criteria Co-primary endpoints 2:1 Randomization 200 mg BID • PMF, PET-MF, PPV-MF at 24 weeks: N=399 • SVR ≥35% • DIPSS Intermediate- or high- risk disease Stratification at Physician’s Choice • Red
Slide 22: NCCN guidelines updated to include pacritinib as first and second line treatment
The NCCN guidelines have been recently updated to include pacritinib as a first and second line treatment Update since previous version No / limited symptom Watch and wait burden Clinical Trial Lower-risk Continue treatment, Response disease Clinical Trial monitor for progression Symptom burden Ruxo
Slide 23: Agenda and presenters
Agenda and presenters Guido Oelkers, Chief Executive Officer Opening remarks Swedish Orphan Biovitrum Overview of Myelofibrosis John Mascarenhas, Professor of Medicine and Vonjo Icahn School of Medicine at Mount Sinai Emerging Insights & Sarah Buckley, Medicine Development Lead Future Potential of P
Slide 24: Additional insights emerging on the benefit of pacritinib
Additional insights have been emerging on the benefit of pacritinib for myelofibrosis patients ACVR1 inhibition and Consistency of Risk-adjusted safetya anaemia benefita,c responsea Comparison of Pacritinib mTSS analysis of PERSIST-2 landmark pacritinib vs. before allogeneic PERSIST-2a OS analysisa
Slide 25: mTSS: Retrospective analysis shows PERSIST-2 would have met primary endpoint
mTSS: A retrospective analysis based on mTSS shows PERSIST-2 would have met the primary endpoint mTSS Reduced by ≥50%1,2 (ITT Efficacy Populationa, Week 24) 40 35 35% PERSIST-2 included tiredness as part of the Percentage of Patients 30 31% total symptom score (TSS) endpoint. 25 20 A retrospective a
Slide 26: PAC vs. RUX: Pacritinib showed larger response compared to ruxolitinib
PAC vs. RUX: Pacritinib 200 mg BID showed larger response compared to ruxolitinib in PERSIST-2 Retrospective Analysis - Efficacy endpoints1 (ITT Efficacy Populationa, Week 24) b Patients treated with pacritinib versus ruxolitinib achieved higher rates of spleen volume reduction and total symptom sco
Slide 27: ACVR1: Multiple pathways in myelofibrosis can contribute to anaemia
ACVR1: Multiple pathways in myelofibrosis can contribute to anaemia ACVR1 is a receptor kinase that controls the expression of the peptide hormone hepcidin Inhibition of ACVR1 reduces production of hepcidin, which leads to increased iron availability for erythropoiesis In parallel, inhibition of IRA
Slide 28: ACVR1: Pacritinib is a potent ACVR1 inhibitor in vitro
ACVR1: Pacritinib is a potent ACVR1 inhibitor in vitro, with predicted 24-hour on-target inhibition Pacritinib Concentration-Time Curve1 Momelotinib Concentration-Time Curve1 Pacritinib is a highly potent, 24-hour inhibitor of ACVR1 Limitation: These data are not included in the Pacritinib Prescribi
Slide 29: Anaemia: Pacritinib increases transfusion independence
Anaemia: Pacritinib increases transfusion independence (PERSIST-2 retrospective analysis) TI conversion rate (Gale criteria*)1 Rate of Transfusion Independence1 Among patients requiring RBC transfusion at baseline Over 12-week interval through Week 24 (Gale criteria) PAC 200 mg BID BAT P-value N=41
Slide 30: Consistency of response regardless of baseline cytopenias
Consistency of response: Pacritinib has similar efficacy regardless of baseline cytopenias Retrospective Analysis Spleen Reduction Over Time by Subgroups1 TSS Reduction Over Time by Subgroups1 Evaluable population (PERSIST-1 and PERSIST-2) Evaluable population (PERSIST-1 and PERSIST-2) • The depth o
Slide 31: Consistency of response: Haemoglobin stability/improvement with pacritinib
Consistency of response: Haemoglobin stability/ improvement with pacritinib Median Change in Haemoglobin Over Time by Baseline Haemoglobin Subgroups1 Evaluable population (PERSIST-1 and PERSIST-2); retrospective analysis 50 40 Haemoglobin (g/L) (IQR) 30 Median Change in 20 Median haemoglobin 10 0 re
Slide 32: The future potential of pacritinib
The future potential of pacritinib We are committed to exploring the potential of pacritinib to benefit patients in myelofibrosis and beyond 32
Slide 33: Pacritinib could address unmet needs in additional myelofibrosis populations
Pacritinib has the potential to address unmet needs in additional patient populations in myelofibrosis Illustration of myelofibrosis patient populations Approx. 20k myelofibrosis patients in the US Approx. 20k myelofibrosis patients in the US Anaemia Pre- Lower-risk Disease Pacritinib inclusion in N
Slide 34: Pacritinib could be beneficial in other indications based on mechanism of action
Treatment with pacritinib could be beneficial in other indications based on its mechanism of action Immunology, rheumatology, Malignant haematology e.g., AML, CMML, MDS, dermatology Waldenström macroglobulinemia Inflammation e.g., GvHD, SLE, VEXAS syndrome Kinase-driven Cytopenia hyperproliferation
Slide 35: Pacritinib is well positioned for further exploration in myelofibrosis and beyond
Pacritinib is well positioned for further exploration of its benefits in myelofibrosis and beyond Additional data generation into the benefit of Real world pacritinib in different patient populations to insights understand its potential and enable wider patient access Strong scientific rationale for
Slide 36: Agenda and presenters
Agenda and presenters Guido Oelkers, Chief Executive Officer Opening remarks Swedish Orphan Biovitrum Overview of Myelofibrosis John Mascarenhas, Professor of Medicine and Vonjo Icahn School of Medicine at Mount Sinai Emerging Insights & Sarah Buckley, Medicine Development Lead Future Potential of P
Slide 37: Combined leadership in haematology
Combined leadership in haematology Vonjo has a Severe thrombocytopenic differentiated profile in MF represents an unmet myelofibrosis clinical need Complementary Expectation: highly haematology reach and accretive to Sobi’s expertise revenue and margins 37
Slide 38: Thank you
Thank you Sobi is a trademark of Swedish Orphan Biovitrum AB (publ). © 2022 Swedish Orphan Biovitrum AB (publ) – All rights reserved Swedish Orphan Biovitrum AB (publ) SE-112 76 Stockholm • Sweden www.sobi.com
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