Ac Immune Sa · NASDAQ:ACIU
Ac Immune Sa — KOL Breakfast NYC July 2019
25 slides from Ac Immune Sa's conference presentation, Jul 11, 2019
Slide 1: ACI-24 anti-Abeta vaccine for prevention and therapy in Alzheimer's disease and Down syndrome
www.acimmune.com © 2019 AC Immune. Not to be used or reproduced without permission. ACI - 24 a nti - Abeta vaccine for prevention and therapy in Alzheimer’s disease and Down syndrome NASDAQ: ACIU | KOL Breakfast NYC | July 2019 Version 07 July 2019
Slide 2: Disclaimer
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 2 Disclaimer This presentation may contain statements that constitute “forward - looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the
Slide 3: AC Immune's dual technology platforms
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 3 AC Immune’s dual technology platforms SupraAntigen TM Vaccines and antibodies specific to disease causing conformations Morphomer TM Conformation - sensitive small molecules (1) Mode of
Slide 4: ACI-24 structure
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 4 ACI - 24 structure Strong antibody response against pathological human Abeta without activation of Abeta - specific T - cells Liposomal anchor (Palmitic chains) Liposomal bilayer (Choles
Slide 5: Key preclinical results: preferential binding to Abeta oligomers and plaques
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 5 Key preclinical results: preferential binding to Abeta oligomers and plaques ACI - 24: proof - of - concept Binding of ACI - 24 immunized mouse sera to Abeta monomers, oligomers and fibe
Slide 6: Key preclinical results: memory restoration in AD and DS mouse models
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 6 Key preclinical results: memory restoration in AD and DS mouse models ACI - 24: proof - of - concept (1 ) Object recognition test ; (2) APPxPS - 1 model; (3) TS65Dn model Ref.: Muhs et a
Slide 7: ACI-24: summary of preclinical studies
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 7 01 Non - clinical and toxicology studies for AD and DS indications ACI - 24: summary of preclinical studies ACI - 24 in non - clinical studies: ▪ Strong and consistent anti - Abeta IgG r
Slide 8: Phase 1/2 Study of ACI-24 in Mild to Moderate Alzheimer's Disease
ACI - 24 - 0701 study: A Phase 1/2 Double - Blind, Randomized, Placebo - Controlled, Adaptive Design Study of the Safety, Tolerability, Immunogenicity and Efficacy of ACI - 24 in Patients with Mild to Moderate Alzheimer’s Disease
Slide 9: Study overview in AD
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 9 Study overview ACI - 24 - 0701 study in AD Study population ▪ Participants with mild to moderate probable Alzheimer’s disease (NINCDS - ADRDA 1 criteria) ▪ MMSE 2 : 18‒28; age >40 and <9
Slide 10: Clinical study design in AD
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast | July 2019 10 Clinical study design ACI - 24 - 0701 study in AD Cohort 1 (10 µg ACI - 24 or placebo) Cohort 2 ( 100 µg ACI - 24 or placebo) Cohort 3 (300 µg ACI - 24 or placebo) Cohort 4 ( 1000 µg ACI -
Slide 11: Demographics and baseline characteristics
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 11 ACI - 24 - 0701 study in AD Demographics and baseline characteristics Characteristic ACI - 24 Placebo Dose 10 µg 100 µg 300 µg 1000 µg - Number of patients 9 9 9 9 12 Age , mean (SD 1 )
Slide 12: Overall summary of AEs – safety analysis set
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 12 ACI - 24 - 0701 study in AD Overall summary of AEs 1 – safety a nalysis set Characteristic ACI - 24 Placebo Dose (# of patients) 10µg (9) 100µg (9) 300µg (9) 1000µg (9) - (12) Treatment
Slide 13: Immunogenicity study results in AD
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 13 Anti - Abeta IgG No IgG response in patients receiving placebo Dose - related late - onset IgG response No immune response observed at 2 lowest doses (Cohorts 1 and 2) Dose dependent im
Slide 14: Abeta PET exploratory analysis in AD
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 14 Abeta PET 1 exploratory analysis ACI - 24 - 0701 study in AD ▪ A dose dependent 3 trend in reduction of brain amyloid accumulation observed in cohorts 3 and 4 at week 52 Change in compo
Slide 15: Exploratory analysis on cognition
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 15 ACI - 24 - 0701 study in AD Exploratory analysis on cognition ▪ A positive trend on cognition measured by MMSE ▪ Observed during the treatment period for the highest dose versus placebo
Slide 16: Key learnings in AD
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 16 ACI - 24 - 0701 study in AD Key learnings ▪ Safe in AD participants with no study treatment - related SAEs 1 ▪ Dose - related anti - Abeta IgG responses observed ▪ A potential dose - de
Slide 17: ACI-24-1301 study in Down syndrome – currently ongoing
ACI - 24 - 1301 study: Phase 1 b Multi - Center, Double - Blind, Randomized, Placebo - Controlled Dose Escalation Study of the Safety, Tolerability and Immunogenicity of ACI - 24 in Adults with Down Syndrome – currently ongoing
Slide 18: Study overview – ACI-24-1301 study in DS
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 18 Study overview – Study currently ongoing ACI - 24 - 1301 study in DS Study population ▪ Male or female participants with DS age ≥25 to ≤45 years ▪ Cytogenetic diagnosis of either Trisom
Slide 19: Clinical study design – ACI-24-1301 study in DS
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 19 Clinical study design ACI - 24 - 1301 study in DS Cohort 1 (300 µg ACI - 24 or placebo) Cohort 2 ( 1000 µg ACI - 24 or placebo) Note: Option to expand one of the above ACI - 24 doses an
Slide 20: Demographics and baseline characteristics
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 20 Demographics and baseline characteristics ACI - 24 - 1301 study in DS Characteristic ACI - 24 Placebo Dose 300 µg 1000 µg - Number of subjects 6 6 4 Age , mean (SD 1 ) (min; max) 33.5 (
Slide 21: Overall summary of AEs – interim data
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 21 Overall summary of AEs 1 – Safety Analysis Set – interim data as of June 26, 2019 ACI - 24 - 1301 study in DS Characteristic ACI - 24 Placebo Dose of study treatment (# of patients) 300
Slide 22: Preliminary immunogenicity results – interim data
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 22 Anti - Abeta IgG responses Observed a boosting effect with time (MSD 1 ) Consistent response in majority of patients at highest dose (MSD 1 ) Early onset response, first increase in tit
Slide 23: ACI-24-1301 study in DS key learnings – interim data
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 23 ACI - 24 - 1301study in DS Key learnings – interim data as of June 26, 2019 ▪ Safe in DS subjects with no SAEs 1 and no early withdrawal at any dose to date. No indication of CNS inflam
Slide 24: AC Immune: pioneers in anti-Abeta and anti-Tau vaccines
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 24 AC Immune: pioneers in anti - Abeta and anti - Tau vaccines 2008 ACI - 24 - 0701 study Dose escalation in mild to moderate AD 2013 ACI - 35 - 1201 study Dose escalation and exploration
Slide 25: We shape the future of neurodegeneration
© 2019 AC Immune. Not to be used or reproduced without permission. NASDAQ: ACIU | KOL Breakfast NYC | July 2019 25 We shape the future of neurodegeneration by discovering and developing breakthrough therapies through pioneering science and precision medicine AC Immune