Priavoid A life without dementia - Company Presentation, BIO, June, 2017 Prof. Dr. Dieter Willbold
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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
2Our 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.
3Alzheimer’s Disease
Registered drugs address only symptoms and have severe side effects
Generic Name Brand Name Approved Side Effects
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
Memantine + Moderate to severe AD Headache, diarrhea, dizziness, loss of appetite,
Namzaric
Donepezil vomiting, nausea, and bruising
4Intervention 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 Aβ fibril
(toxic)
5Intervention Strategies
Our strategy is to directly eliminate cytotoxic Aβ oligomers
PRI-002
(all-D-enan(omeric pep(de)
APP Aβ monomer Aβ oligomer Aβ fibril
(toxic)
6Primary 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)
7Primary 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)
8Pharmacokinetics 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
9Development 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
10Intellectual 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
11Unique 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
12All-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
13The Drug Pipeline
All-D-Peptide compounds and development status
Screening/ IND Phase I Phase II
Indica(on Target Compound POC Animal
Design Package* Clinical Trial 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
14The 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 Director Clinical Director CMC Director Preclinical Managing Director Director of Quality Management
Supervisory Board Development Research and
Regulatory Compliance
15A 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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