Medivir Ab · OMXSTO:MVIR
Medivir Ab — Investor Presentation (November 2024)
32 slides from Medivir Ab's investor presentation, November 2024
Slide 1: Improving life for advanced liver cancer (HCC) patients
Placeholder agenda Improving Improvinglife forfor life advanced advanced liver liver cancer (HCC) patients cancer (HCC) patients Fostrox – Displacing The first oral, chemotherapy liver-targeted with the treatment first oral, liver-activated forofadvanced inhibitor HCC DNA replication
Slide 2: Important notice
Important notice You must read the following before continuing. The following applies to this document and the information provided in this presentation by Medivir AB (publ) (the “Company”) or any person on behalf of the Company and any other material distributed or statements made in connection wit
Slide 3: Fostrox: The first oral, liver-targeted treatment tailored for HCC
Fostrox (fostroxacitabine bralpamide) The first oral, liver-targeted treatment tailored for HCC Oral, liver-activated small molecule inducing DNA 10.9 months time to progression, damage in tumor cells, sparing healthy liver cells3 substantially better than SoC1,2 12 Unique, liver-targeted No DNA dam
Slide 4: Medivir management team
Medivir management team CEO CMO CFO CSO Jens Lindberg Dr. Pia Baumann Magnus Christensen Fredrik Öberg • > 25 years of experience from • MD PhD with a specialist degree in • > 25 years of experience in finance. • PhD in Medical Science & Adjunct pharmaceutical industry with focus on medical & radiat
Slide 5: First-to-market opportunity in 2nd line HCC market valued >$2.5bn
First-to-market opportunity in 2nd line HCC market valued >$2.5bn Slide 5
Slide 6: 2nd line HCC – a large and growing commercial opportunity
2nd line HCC – a large and growing commercial opportunity with significant need for new treatment options3 $4.0 bn Growth driven by: HCC to increase +122% in the US and +82% in China2 by 2030, caused by fatty liver disease +115% $2.8 bn With improved 1L treatment, more patients will be fit enoug
Slide 7: Absence of effective treatment options in 2nd line HCC
Absence of effective treatment options in 2nd line HCC Treatment algorithm – major Competitive landscape in 2nd line HCC highlights lack of novel mechanisms in need for new 2nd line options development with fostrox + Lenvima at the forefront 1st line treatment “We are becoming greedy, IO combinati
Slide 8: Fostrox – tailored for the specific needs of HCC
Fostrox – tailored for the specific needs of HCC Slide 8
Slide 9: Targeted treatment approach critical in liver cancer (HCC)
Targeted treatment approach critical in liver cancer (HCC) ~80% of patients have impaired liver function due to underlying liver disease1,2 Key objective – target cancer Tumor growth cells, minimize primarily occurs effects on healthy locally in the liver1 cells Critical to achieve selective targeti
Slide 10: Fostrox – tailored for HCC to achieve targeted DNA damage in liver tumor cells
Fostrox – tailored for HCC to achieve targeted DNA damage in liver tumor cells with minimal impact on healthy liver cells The same, proven & highly successful approach used in HCV to Combining prodrug & payload enables oral administration maximize liver-targeting2 & targeted (>100-fold) liver exposu
Slide 11: Fostrox MoA – targeted DNA damage in liver tumor cells
Fostrox MoA – tailored for HCC to achieve targeted DNA damage in liver tumor cells with minimal impact on healthy liver cells Inactive Prodrug cytotoxic Fostrox taken as oral tablet Prodrug transports inactive payload via first-pass metabolism to the liver1 Rapidly activated by liver enzymes Trapped
Slide 12: 100-fold higher liver targeting vs IV administration & selective DNA damage
100-fold higher liver targeting vs IV administration & selective DNA damage in tumor cells enabling highly targeted mechanism >100-fold higher liver targeting with Liver tumor cells divide significantly Fostrox induces DNA damage in tumor fostrox than iv troxacitabine in rats more often than non-tum
Slide 13: Fostrox + Lenvima shows promise of improved outcomes in 2L HCC
Fostrox + Lenvima shows promise of improved outcomes in 2L HCC Slide 13
Slide 14: Fostrox Clinical Development Program
Fostrox Clinical Development Program; monotherapy PoC established, focus on combination approach in 2L HCC Future – Phase 1a Mono Phase 1b Mono Phase 1b Combo Phase 2a Combo Phase 2b Combo Single patient Fostrox + Lenvima Fostrox + Lenvima Fostrox + Lenvima 3+3 dose Intrapatient dose dose escalation
Slide 15: Fostrox + Lenvima phase 1b/2a study design
Fostrox + Lenvima phase 1b/2a study design Fostrox + LEN Treatment until PD Fostrox + LEN • Adv/inop HCC 30 mg RP2D – 30 mg (n=15) Primary Endpoint: • 2L/3L (n=3) Safety • ≥ 18 y • ECOG ≤ 1 Fostrox + LEN* Key Secondary • Child-Pugh A 20 mg Endpoints: ORR, DCR, (n=3) TTP/PFS Patients were enrolled at
Slide 16: Global phase 1b/2a study with fostrox + Lenvima (TKI)
Global phase 1b/2a study with fostrox + Lenvima (TKI) UK 3 centres Key study features Korea 6 centres Fostrox + Lenvima in second and third line advanced HCC Spain 15 sites in South Korea, Spain and UK 6 centres Rapid recruitment speed, phase 2a dose expansion fully recruited in <5 months Me
Slide 17: Patient characteristics reflecting generous inclusion criteria
Patient characteristics reflecting generous inclusion criteria Patient characteristics1 N = 21 Mean age (range) 62 yrs (42 - 82) Gender, Female / Male (%) 24 / 76 ECOG Performance status 0/1 (%) 71 / 29 Child-Pugh A (%) 100 Viral/Non-viral (%) 76* / 24 Extra hepatic lesion(s) Y/N (%) 67 / 33 AFP ≥4
Slide 18: Median TTP 10.9 months, improved efficacy vs Lenvima alone
Median TTP 10.9 months, indicating substantially improved efficacy compared with Lenvima alone1 Median time to progression (TTP) with fostrox + LEN – investigator review, RECISTv1.1 Fostrox + LEN (n =21)1 Median TTP, mo 10.9 95% CI (4.1 – 18.1) • Median time to progression 10.9 months ORR 24% • Medi
Slide 19: Median TTP 10.9 months, longer than Lenvima monotherapy and other 2L HCC treatments
Median time to progression (TTP) 10.9 months, remarkably longer than Lenvima monotherapy and other 2L HCC treatments Median TTP (Kaplan-Meier) with fostrox + Lenvima Median TTP/PFS vs previous studies in 2L HCC ~3.5-4 months Lenvima after IO combo: Fostrox + LEN (n =21)1 Kobayashi et al. 2023 (n=12)
Slide 20: Best % change in target lesion
More than 75% of patients experiencing tumor shrinkage1 Best percentage change in target lesion size related to treatment response in first line 30% 20% Disease control on prior therapy Best % change in target lesion 16% Primary refractory on prior therapy 10% Median duration of response 7.0 11%
Slide 21: Fostrox + Lenvima data signals superiority compared with Lenvima monotherapy or IO combos
Fostrox + Lenvima data signals superiority compared with Lenvima monotherapy or IO combo treatments in 2nd line HCC Lenvima in 2L Lenvima in 2L Keytruda + TKI Fostrox + HCC1 – Korea HCC2 – Japan in 2L HCC3 Lenvima4 “The response rate of 24% is higher than historical Median data of Lenvima alone in 2
Slide 22: Fostrox + Lenvima shows encouraging tolerability
Fostrox + Lenvima shows encouraging tolerability enabling patients to remain on treatment long-term Slide 22
Slide 23: Absolute neutrophil and platelet counts were stable over treatment
Absolute neutrophile and platelet counts were stable over the course of treatment, enabling long-term use1 Longitudinal neutrophil & platelet counts, at all time points measured over first 10 months of treatment 8 000 Neutrophils Platelets 350 000 7 000 300 000 6 000 250 000 neutrophils per microlit
Slide 24: Liver targeting supports encouraging tolerability profile
Liver targeting properties supports encouraging tolerability profile, enabling patients to remain on treatment long-term Treatment Treatment Fostrox LEN emergent AEs emergent AEs Treatment- Treatment- Adverse Events* any grade Grade ≥ 3 related Grade ≥ 3 related Grade ≥ 3 No of pts (%) No of pts (%)
Slide 25: Stable liver function during treatment with fostrox + Lenvima
Stable liver function during treatment with fostrox + Lenvima – no deterioration in liver enzymes or change in ALBI score ALT change over duration of AST change over duration of ALBI score change over duration of treatment treatment treatment 200 -1,50 ALBI Score 80 -2,00 150 60 -2,50 AST (U/L) ALT
Slide 26: Randomized phase 2b to enable breakthrough designation & accelerated approval
Randomized phase 2b to enable breakthrough designation & accelerated approval Slide 26
Slide 27: Phase 2b; dose optimization run in & adaptive design
Phase 2b; dose optimization run in & adaptive design to enable breakthrough therapy designation & accelerated approval filing FPI Q2 2025 2025 2026 Study Read Out H1 2027 Part 1 Dose select Part 2 Inclusion criteria Fostrox 30 mg + DSMB: Fostrox xx mg + DSMB: Primary endpoint: • Adv/inop HCC Lenvima
Slide 28: Coordinating PI and US PI for fostrox + Lenvima phase 2b study
Coordinating PI and US PI for fostrox + Lenvima phase 2b study among the most well renowned global HCC thought leaders Dr. Maria Reig – Coordinating PI Dr. Anthony B. El-Khoueiry, MD – US PI • Head of the BCLC (Barcelona Clinic for Liver Cancer), who • Associate Director for Clinical Research at USC
Slide 29: Highly distinguished steering committee
Highly distinguished steering committee Phase 2b fostrox + Lenvima vs Lenvima Dr. Maria Reig – Coordinating PI Dr. Anthony B. El-Khoueiry, MD – US PI • Head of the BCLC and Liver Oncology Unit at Hospital Clinic of • Associate Director for Clinical Research at USC Norris and Barcelona in Spain. Asso
Slide 30: Fostrox – The first oral, liver-targeted treatment tailored for HCC
Fostrox – The first oral, liver-targeted treatment tailored for HCC Oral, liver-activated small molecule inducing DNA 10.9 months time to progression, damage in tumor cells, sparing healthy liver cells3 substantially better than SoC1,2 12 Unique, liver-targeted No DNA damage in DNA damage in Fostrox
Slide 31: Board with extensive early & late stage drug development experience
Board with extensive early & late stage drug development experience Chairman of the Board – Dr. Uli Hacksell Dr. Lennart Hansson, Ph D Genetics Member since 2018, Chairman since 2021 Member since 2018 Over 30 years pharma & biotech experience, Extensive experience of pharmaceutical & inc
Slide 32: Thank You!
Thank You! Slide 32