Priavoid. A life without dementia. Company Presentation, BIO, June, Prof. Dr. Dieter Willbold

Transcription

1 Priavoid A life without dementia Company Presentation, BIO, June, 2017 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 2

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. 3

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 4

5 Intervention Strategies Up to now, no disease modifying drug has been approved Monoclonal an(bodies binding to Aβ beta and gamma secretase inhibitors APP Aβ monomer Aβ oligomer (toxic) Aβ fibril 5

6 Intervention Strategies Our strategy is to directly eliminate cytotoxic Aβ oligomers PRI-002 (all-d-enan(omeric pep(de) APP Aβ monomer Aβ oligomer (toxic) Aβ fibril 6

7 Primary Pharmacodynamics - in vitro QIAD: Quantitative determination of interference with Aβ aggregate size distribution PRI-002 eliminates toxic Aβ oligomers Aβ oligomer (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) 8

9 Pharmacokinetics and Toxicology Overview of studies Study type Pharmacokinetic studies In rodents In non-rodents Immunogenicity ADA studies in rodents ADA studies in non-rodents T cells Routine parameters Routine parameters Metabolization Free fraction Stability in plasma, SGF, SIF and microsomes Toxicology 10-day repeated-dose toxicity study Dose-range-finding study 4-week repeated dose toxicity in rodents, GLP 4-week repeated dose toxicity in non-rodents, GLP Genotoxicity AMES, GLP Micronucleus Test system / administration in vivo, rats, i.v., p.o. in vivo, cynomolgus, i.v., p.o. surface plasmon resonance surface plasmon resonance in vitro rats cynomolgus blood, human cells in vitro in vivo, rat, p.o. in vivo, cynomolgus, p.o. in vivo, rat, p.o. in vivo, cynomolgus, p.o. in vitro, salmonella typhimurium reverse mutation assay in vitro, CHO-K1 9

10 Development Plan Phase II Clinical Trial of PRI-002 Step Activity Q1 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 10

11 Intellectual Property Rights Ten patent families filed in EU, US, JP, CN. The most important ones have been filed in 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 A2 Filed: April 5, 2013 DE, EP, CN, JP, US Method for treating blood, blood products and organs Publication number: WO A3 Filed: April 5, 2013 DE, EP, CN, JP, US Novel D-enantiomeric peptides derived from D3 and use thereof Publication number: WO A2 Filed: September 13, 2013 DE, EP, CN, JP, US 11

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 12

13 All-D-Peptides Our platform for the generation of CNS drugs Microarray Chemical Modifica;on All-D-peptides combine the stability of small molecules with the selectivity of proteins and antibodies. Mirror- Image Phage Display All-D- Pep(des Ra;onal Design Pre-clinical studies and Clinical Trials 13

14 The Drug Pipeline All-D-Peptide compounds and development status Indica(on Target Compound Screening/ Design POC Animal IND Package* Phase I Clinical Trial Phase II Clinical Trial Alzheimer s Aβ PRI-002 ALS Inflamma;on PRI-003 ALS SOD1 in progress Tauopathies Tau in progress Hun(ngton s PolyQ in progress Parkinson s α-synuclein in progress *Toxicology Safety Pharmacokinetics 14

15 The Founders Expertise in Life Science, Health Care, and Corporate Development Dieter Willbold Dagmar Jürgens Knut Adermann Antje Willuweit Ralph Zahn Gunther Kauselmann Chairman of the Supervisory Board Director Clinical Development Director CMC Director Preclinical Research Managing Director Director of Quality Management and Regulatory Compliance 15

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 16