Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM

Mirum Pharmaceuticals — Technical Presentation (2017)

15 slides from Mirum Pharmaceuticals's technical presentation, 2017

  1. Slide 1: Long-term phase 2 study of maralixibat in children with Alagille syndrome: IMAGINE results

    A long-term phase 2 safety and efficacy study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with Alagille syndrome: preliminary results from the IMAGINE study Richard J Thompson King's College London, London, UK Study funded by Shire Development LLC

  2. Slide 2: Co-authors

    Co-authors ▪ Alastair Baker,1 Deirdre A Kelly,2 Joan Gu,3 Thomas Jaecklin,4 Patricia McClean,5 Binita M Kamath,6 Benjamin L Shneider7 1. Paediatric Liver Centre, King’s College Hospital, London, UK 2. Liver Unit, Birmingham Children's Hospital, Birmingham, UK 3. Shire, Lexington, MA, USA 4. Shire, Z

  3. Slide 3: Disclosures

    Disclosures ▪ Note to authors: AASLD will add a disclosures slide based on the information you provided for the abstract submission

  4. Slide 4: Maralixibat: a potential treatment for children with Alagille syndrome

    Maralixibat: a potential treatment for children with Alagille syndrome ▪ Alagille syndrome (ALGS) is a genetic disorder affecting multiple organs including the liver1,2 – ALGS manifests in infancy or childhood – Bile duct abnormalities lead to cholestasis and often end-stage liver disease and early

  5. Slide 5: Study design: IMAGO and the IMAGINE extension

    Study design: IMAGO and the IMAGINE extension IMAGO (LUM001-302): IMAGINE (LUM001-303): long-term extension study 13-week randomized double-blind Double-blind period placebo-controlled study Dose Dose Stable dosing escalation optimization Maralixibat 280 µg/kg/day Maralixibat 140 µg/kg/day Optimizat

  6. Slide 6: Inclusion/exclusion criteria

    Inclusion/exclusion criteria Key inclusion criteria Key exclusion criteria ▪ Completed IMAGO to week 13 ▪ Maralixibat-related AE in IMAGO that led to discontinuation ▪ IMAGO inclusion criteria – Diagnosis of ALGS ▪ Non-adherent in IMAGO – Male or female aged 1−18 years ▪ IMAGO exclusion criteria – s

  7. Slide 7: Reported outcomes and analyses

    Reported outcomes and analyses Outcomes Analyses ▪ Primary efficacy: change in sBA levels ▪ Initial ad hoc analysis of data up to from baseline week 96 – Baseline was IMAGO week 0 – Study database not yet locked (IMAGINE week –13) ▪ No inferential statistical hypothesis ▪ Pruritus assessments testin

  8. Slide 8: Disposition, demographics and disease characteristics

    Disposition, demographics and disease characteristics Enrolled participant Disposition to week 96 characteristics (N = 19) N Characteristic Value Completed IMAGO 19 Age, years, Maralixibat 14 5.0 (1, 17) median (range) Placebo 5 Sex Entered IMAGINE and Male, n (%) 10 (52.6) 19 received maralixibat R

  9. Slide 9: Analysis of treatment response

    Analysis of treatment response IMAGINE study week ID 4 2 4 8 12 24 48 84 96 (21.1%) A ✓ ✓ ✓ ✓ ✓ ✓ ✓ B ✓ ✓ ✓ C ✓ ✓ ✓ ✓ ✓ ✓ ✓ 15 D ✓ ✓ ✓ ✓ DNC (78.9%) ≥ 70% decrease in sBA and ✓ > 1.0-point improvement in ItchRO(Obs)a Responder Non-responder (Required at ≥ 2 of last 3 visits for response) aFrom IMAGO

  10. Slide 10: IMAGINE sBA and ItchRO(Obs) results overall and in previous IMAGO treatment subgroups

    IMAGINE sBA and ItchRO(Obs) results overall and in previous IMAGO treatment subgroups sBA ItchRO(Obs) n = 14 13 13 13 13 12 11 9 4 4 14 14 13 12 12 4 4 n= 5 5 5 5 5 5 3 3 2 2 5 5 5 5 5 2 1 n = 19 18 18 18 18 17 14 12 6 6 19 19 18 17 17 6 5

  11. Slide 11: IMAGINE CSS results overall and in previous IMAGO treatment subgroups

    IMAGINE CSS results overall and in previous IMAGO treatment subgroups n = 14 14 13 13 12 11 11 12 4 4 n= 5 5 5 5 5 5 4 4 2 2 n = 19 19 18 18 17 16 15 16 6 6

  12. Slide 12: Potentially maralixibat-related treatment-emergent AEs (TEAEs)

    Potentially maralixibat-related treatment-emergent AEs (TEAEs) TEAE Participants, n (%) TEAE Participants, n (%) Any 13 (68.4) Gastrointestinal 11 (57.9) Leading to discontinuation 1 (5.3) Abdominal pain 9 (47.4)a Diarrhoea 6 (31.6) Maximum severity Abnormal faeces 1 (5.3)a Mild 9 (47.4) Flatulence

  13. Slide 13: Summary

    Summary ▪ sBA levels and pruritus scores improved with maralixibat treatment in the study population – These effects were maintained for up to 96 weeks in this snapshot ▪ 4/19 participants were classified as responders to maralixibat ▪ Improvement in parent-rated ItchRO scores appeared greatest in p

  14. Slide 14: Conclusions

    Conclusions ▪ These results suggest that maralixibat provided long-term pruritus relief and reduced sBA levels in a subset of children with ALGS – Factors determining response in certain patients remain unclear ▪ Mild or moderate gastrointestinal AEs were consistent with the mode of action of marali

  15. Slide 15: Acknowledgements

    Acknowledgements ▪ We thank the participants, caregivers, investigators and co-ordinators involved in the study

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