Priavoid A life without dementia Company Presentation, BIO, June, 2017 Prof. Dr. Dieter Willbold (co-founder and future Chairman of the Supervisory Board)
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
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
Alzheimer’s Disease Registered drugs address only symptoms and have severe side e ff ects Generic Name Brand Name Approved Side E ff ects Nausea, vomiting, loss of appetite and increased Donepezil Aricept All stages frequency of bowel movements Nausea, vomiting, loss of appetite and increased Galantamine Razadyne Mild to moderate AD frequency of bowel movements Memantine Namenda Moderate to severe AD Headache, constipation, confusion and dizziness Mild to moderate AD Nausea, vomiting, loss of appetite and increased Rivastigmine Exelon frequency of bowel movements Moderate to severe AD Headache, diarrhea, dizziness, loss of appetite, Memantine + Namzaric Donepezil vomiting, nausea, and bruising 4
Intervention Strategies Up to now, no disease modifying drug has been approved Monoclonal an(bodies binding to A β beta and gamma secretase inhibitors APP A β monomer A β oligomer A β fibril (toxic) 5
Intervention Strategies Our strategy is to directly eliminate cytotoxic A β oligomers PRI-002 (all-D-enan(omeric pep(de) APP A β monomer A β oligomer A β fibril (toxic) 6
Primary Pharmacodynamics - in vitro QIAD: Quantitative determination of interference with A β aggregate size distribution PRI-002 eliminates toxic A β oligomers A β oligomer (toxic) QIAD assay ( QIAD assay (Brener Brener et al., Scienti et al., Scienti fj fj c Reports, 2015) c Reports, 2015) 7
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
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 9
Development Plan Phase II Clinical Trial of PRI-002 Step Activity 2015 2016 2017 2018 2019 2020 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
Intellectual Property Rights Ten patent families fj led in EU, US, JP, CN. The most important ones have been fj led 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 § 11
Unique Selling Propositions ➔ New mechanism of action § Speci fj c elimination of toxic A β oligomers ➔ New drug substance class § Synthetic all- D -peptide ➔ Oral drug administration § Protease-resistant 12
All- D -Peptides Our platform for the generation of CNS drugs ➔ All- D -peptides combine the stability of small molecules with the selectivity of Chemical Microarray proteins and antibodies. Modifica;on Mirror- Ra;onal Image Phage Design Display All- D - Pep(des Pre-clinical studies and Clinical Trials 13
The Drug Pipeline All- D -Peptide compounds and development status Compound Screening/ IND Phase I Phase II Indica(on Target POC Animal Design Package* Clinical Trial Clinical Trial Alzheimer´s A β PRI-002 Inflamma;on PRI-003 ALS ALS SOD1 in progress Tauopathies Tau in progress Hun(ngton´s PolyQ in progress α -Synuclein in progress Parkinson´s *Toxicology | Safety | Pharmacokinetics 14
The Founders Expertise in Life Science, Health Care, and Corporate Development Dieter Willbold Dagmar Jürgens Knut Adermann Antje Willuweit Ralph Zahn Gunther Kauselmann Director of Quality Management Chairman of the Director Clinical Director CMC Director Preclinical Managing Director and Supervisory Board Development Research Regulatory Compliance 15
A life without dementia Thank you for your attention! We are here to fj nd investors and/or partners to carry out a Clinical Phase II study 16
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