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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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
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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.
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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
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Intervention Strategies Up to now, no disease modifying drug has been approved
Monoclonalan(bodies
bindingtoAβ
Aβ oligomer(toxic)
Aβ fibrilAβmonomerAPP
betaandgammasecretaseinhibitors
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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)
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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)
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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
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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
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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
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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
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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
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The Drug Pipeline All-D-Peptide compounds and development status
*Toxicology | Safety | Pharmacokinetics
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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
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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