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Presentation Priavoid 10JUL2017 AAIC Willbold very short...

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Company Presentation, BIO, June, 2017 Priavoid A life without dementia Prof. Dr. Dieter Willbold (co-founder and future Chairman of the Supervisory Board)
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  • Company Presentation, BIO, June, 2017

    Priavoid A life without dementia

    Prof. Dr. Dieter Willbold (co-founder and future Chairman of the Supervisory Board)

  • 2

    The Company At a glance

    Established: probably in July 2017

    Location: Jülich, Germany (60 km from Düsseldorf)

    Parent Organizations: Research Center Jülich (FZJ) and Heinrich Heine University Düsseldorf (HHU)

    Pre-seed Funding Sources: FZJ, Helmholtz Association, HHU, Volkswagen Foundation

  • 3

    Our Vision A life without dementia

    ➔ We want to stop Alzheimer’s disease using a new treatment strategy that employs a new class of orally available compounds.

    ➔  In parallel, this new strategy is also applied to other neurodegenerative diseases.

  • 4

    Alzheimer’s Disease Registered drugs address only symptoms and have severe side effects

    Generic Name Brand Name Approved Side Effects

    Donepezil Aricept  All stages Nausea, vomiting, loss of appetite and increased frequency of bowel movements

    Galantamine Razadyne  Mild to moderate AD Nausea, vomiting, loss of appetite and increased frequency of bowel movements

    Memantine Namenda Moderate to severe AD Headache, constipation, confusion and dizziness

    Rivastigmine Exelon Mild to moderate AD

    Nausea, vomiting, loss of appetite and increased frequency of bowel movements

    Memantine + Donepezil Namzaric

    Moderate to severe AD

    Headache, diarrhea, dizziness, loss of appetite, vomiting, nausea, and bruising

  • 5

    Intervention Strategies Up to now, no disease modifying drug has been approved

    Monoclonalan(bodies

    bindingtoAβ

    Aβ oligomer(toxic)

    Aβ fibrilAβmonomerAPP

    betaandgammasecretaseinhibitors

  • 6

    Intervention Strategies Our strategy is to directly eliminate cytotoxic Aβ oligomers

    PRI-002(all-D-enan(omericpep(de)

    Aβ oligomer(toxic)

    Aβ fibrilAβmonomerAPP

  • Primary Pharmacodynamics - in vitro QIAD: Quantitative determination of interference with Aβ aggregate size distribution PRI-002 eliminates toxic Aβ oligomers

    Aβoligomer(toxic)

    QIAD assay (Brener et al., Scientific Reports, 2015)

    7

  • 8

    Primary Pharmacodynamics Most important results

    §  Eliminates oligomers (QIAD)

    §  Improves spatial learning and cognition after oral treatment

    (Morris water maze test, two mouse models)

    §  Deceleration of neurodegenerative progression

    (SHIRPA test, third mouse model)

  • 9

    Pharmacokinetics and Toxicology Overview of studies

    Study type Test system / administration Pharmacokinetic studies

    In rodents in vivo, rats, i.v., p.o. In non-rodents in vivo, cynomolgus, i.v., p.o.

    Immunogenicity ADA studies in rodents surface plasmon resonance ADA studies in non-rodents surface plasmon resonance T cells in vitro Routine parameters rats Routine parameters cynomolgus

    Metabolization Free fraction blood, human cells Stability in plasma, SGF, SIF and microsomes in vitro

    Toxicology 10-day repeated-dose toxicity study in vivo, rat, p.o. Dose-range-finding study in vivo, cynomolgus, p.o. 4-week repeated dose toxicity in rodents, GLP in vivo, rat, p.o. 4-week repeated dose toxicity in non-rodents, GLP in vivo, cynomolgus, p.o.

    Genotoxicity AMES, GLP in vitro, salmonella typhimurium reverse mutation assay Micronucleus in vitro, CHO-K1

  • 10

    Development Plan Phase II Clinical Trial of PRI-002

    Step Activity 2015 2016 2017 2018 2019 2020Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4

    1 Bionalytical method development and validation (GLP)

    2 API manufacture development (supply for tox/safety)

    3 GLP tox studies in rat & cynomolgus (4 weeks)

    4 PK in rat & cynomolgus

    5 Safety pharmacology (GLP)

    6 API manufacture phase I (GMP, kg-scale)

    7 Clinical trial application phase I (SAD)

    8 SAD clinical phase I trial

    9 MAD clinical phase I trial

    10 API manufacture for 6/9-months tox rat & cyno (GMP)

    11 6/9-months tox in 2 species supporting phase II approval

    12 Formulation development for phase II

    13 API manufacture phase II

    14 IMP manufacture for clinical supply phase II

    15 Clinical phase II trial

    accomplished ongoing planned

  • 11

    Intellectual Property Rights Ten patent families filed in EU, US, JP, CN. The most important ones have been filed in 2013. Most of them are either granted or about to be granted by the respective agencies.

    Polymers containing multivalent amyloid-beta-binding D-peptides and their use §  Publication number: WO 2013150127 A2 §  Filed: April 5, 2013 §  DE, EP, CN, JP, US

    Method for treating blood, blood products and organs §  Publication number: WO 2013150126 A3 §  Filed: April 5, 2013 §  DE, EP, CN, JP, US

    Novel D-enantiomeric peptides derived from D3 and use thereof §  Publication number: WO 2014041115 A2 §  Filed: September 13, 2013 §  DE, EP, CN, JP, US

  • 12

    Unique Selling Propositions

    ➔  New mechanism of action §  Specific elimination of toxic Aβ oligomers

    ➔  New drug substance class §  Synthetic all-D-peptide

    ➔  Oral drug administration §  Protease-resistant

  • 13

    All-D-Peptides Our platform for the generation of CNS drugs

    All-D-Pep(des

    Mirror-ImagePhage

    Display

    Microarray ChemicalModifica;on

    Ra;onalDesign

    Pre-clinicalstudiesandClinicalTrials

    ➔  All-D-peptides combine the stability of small molecules with the selectivity of proteins and antibodies.

  • Indica(on Target Compound Screening/Design POCAnimalIND

    Package*PhaseI

    ClinicalTrialPhaseII

    ClinicalTrial

    Alzheimer´s Aβ PRI-002

    ALS Inflamma;on PRI-003

    ALS SOD1 inprogress

    Tauopathies Tau inprogress

    Hun(ngton´s PolyQ inprogress

    Parkinson´s α-Synuclein inprogress

    14

    The Drug Pipeline All-D-Peptide compounds and development status

    *Toxicology | Safety | Pharmacokinetics

  • 15

    The Founders Expertise in Life Science, Health Care, and Corporate Development

    Knut Adermann Director CMC

    Dagmar Jürgens Director Clinical Development

    Dieter Willbold Chairman of the Supervisory Board

    Antje Willuweit Director Preclinical Research

    Ralph Zahn Managing Director

    Gunther Kauselmann Director of Quality Management and Regulatory Compliance

  • 16

    A life without dementia

    Thank you for your attention! We are here to find investors and/or partners to carry out a Clinical Phase II study


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