JMP Healthcare Conference June 2018
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1 NASDAQ: GNMX JMP Healthcare Conference June , Aevi Genomic Medicine
2 Forward-Looking Statement This presentation includes certain estimates and other forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, including statements with respect to anticipated operating and financial performance, clinical results, potential partnerships, licensing opportunities and other statements of expectation. Words such as expects, anticipates, intends, plans, believes, assumes, seeks, estimates, should and variations of these words and similar expressions, are intended to identify these forward-looking statements. While we believe these statements are accurate, forward-looking statements are inherently uncertain and we cannot assure you that these expectations will occur and our actual results may be significantly different. These statements by the Company and its management are based on estimates, projections, beliefs and assumptions of management and are not guarantees of future performance. Important factors that could cause actual results to differ from those in the forward-looking statements include the factors described in the Company s filings with the U.S. Securities and Exchange Commission. The Company disclaims any obligation to update or revise any forward-looking statement based on the occurrence of future events, the receipt of new information, or otherwise. 2018, Aevi Genomic Medicine 2
3 Management Team Michael F. Cola Chief Executive Officer Garry A. Neil, MD Chief Scientific Officer Brian D. Piper Chief Financial Officer Sol J. Barer, PhD Chairman 2018, Aevi Genomic Medicine 3
4 Investment Highlights Aevi Genomic Medicine (NASDAQ:GNMX) is focused on unlocking the potential of genomic medicine Exclusive option to the genomic discoveries with The Center for Applied Genomics (CAG) at Children s Hospital of Philadelphia (CHOP) Lead asset (AEVI-001) is a first-in-class mglur neuromodulator in Phase 2b clinical trials in a genetic subset of patients with ADHD Second asset (AEVI-002) is a first-in-class monoclonal antibody in a Phase I/II trial for severe pediatric-onset Crohn s Disease 2018, Aevi Genomic Medicine 4
5 ADHD Symptoms in Children 2018, Aevi Genomic Medicine 5
6 ADHD Comorbidities Stahl & Mignon, , Aevi Genomic Medicine 6
7 Breakthrough CHOP Discovery: Novel underlying genetic association with >20% of ADHD ADHD has long been known to be highly heritable (70%) but gene associations have been inconsistent and difficult to replicate ~10 fold increased frequency of rare recurrent mutations in mglur gene network in ADHD 1,2 Mutations also present in several related neuropsychiatric disorders The genetic finding parallels recent imaging studies 3 >20 % of ADHD patients have an underlying mglur network CNV mutation 1. ADHD (n=3,506) vs. normal controls (n=13,327). 2. Elia, et al. (2012) Nat Genet, 44(1): Gallo and Posner (2016) Lancet Psychiatry, 3(6): , Aevi Genomic Medicine 7
8 Expression levels of key proteins affected by mglur network CNVs may play a role in etiology of disease and response to AEVI weeks 3-5 weeks 6-8 weeks 1-2 weeks 6-7 weeks Key Takeaways Cells from patients with known CNVs and ADHD will be used to develop stem cells and differentiated to neuronal cells Derive from CAG Biobank: CNV Containing PBMCs from ADHD Children PBMCs from typically developing children Peripheral Blood Mononuclear Cells (PBMC) recovery and culture IPS cell generation using Sendai reprogramming method 3 best clones selected for characterization IPS cell characterization by marker staining (OCT4 and TRA 1-60) 2 clones selected and frozen Differentiate IPS cells into neurons Examine expression for all clones +/- AEVI- 001 Follow-on experiments As expression is one of our hypotheses, these cells will be assessed for their expression of different gene products Assess these expression levels with and without AEVI-001 to determine how our medicine may affect expression levels PBMC: Peripheral Blood Mononuclear Cells IPS Cells: Induced Pluripotent Stem Cells 2018, Aevi Genomic Medicine 8
9 GRM5 Expression Analysis using Patient Derived Samples Key Takeaways Cells derived from ADHD patients from the CHOP biobank were reprogrammed to IPS Cells and differentiated into neurons mrna was extracted from cells cultured with and without AEVI-002 and GRM5 expression was assessed Patient showed lower GRM5 expression levels compared to controls and AEVI-001 did not alter expression levels up to 30 ug/ml AEVI-001 would appear to function through an alternative mechanism other than normalizing expression in affected patients Aevi is continuing to develop IPS cells from patient derived samples to assess the functional characteristics of these cells with AEVI-001 to elucidate mechanism of action and additional selection criteria for patients that may benefit from therapy 2018, Aevi Genomic Medicine 9
10 Effect of mglur Mutations on Anatomy and Brain Function in ADHD Conducted by Dr. Jonathan Posner at Columbia University 2018, Aevi Genomic Medicine
11 Effect of mglur Mutations on Anatomy and Brain Function in ADHD Patients Studied 61 youth with and without ADHD (Genotyped at CHOP) 9 ADHD with mutations in MGluR system (ADHD + mglur) 26 ADHD without mutations in MGluR system (ADHD - mglur) 26 healthy controls (HC) Samples matched on: Age: = 12.32, Sex, Race/ethnicity Methodology Structural (T1) MRI Voxel-based morphology (VBM) Size & shape of brain regions Resting-state functional MRI Functional connectivity Brain regions with synchronized neural activity Focused on connectivity within the default mode network (DMN), a neural network highly implicated in ADHD and attentional symptoms. 2018, Aevi Genomic Medicine 11
12 Structural MRI reduced parietal volumes Right Parietal Cortex Left Parietal Cortex p<0.05 with permutation testing ADHD youth with mglur mutations show decreased grey matter volumes in left and right parietal cortex 2018, Aevi Genomic Medicine 12
13 Resting State fmri: mglur mutations vs mglur- ADHD Reduced Default Mode Network (DMN) connectivity Right Parietal Cortex Left Parietal Cortex ADHD youth with mglur mutations show decreased DMN connectivity with left and right parietal cortex 2018, Aevi Genomic Medicine 13
14 Study-001 Phenotype/Genotype: Odds Ratio of Phenotype in 9-gene subset Vs. CNV negative ADHD patients In 9-gene subset mglur+ ADHD patients symptoms associated with emotional dysregulation were most prevalent including: Disruptive behavior Anger control Inappropriate sounds & movement Emotional dysregulation often has significant life consequences 1 1 Shaw, et al. (2014) Am J Psychiatry, 171(3): Disruptive behavior Inappropriate movements Anger control Homework Completing work Inappropriate sounds Absenteeism Unhappy at school Disobedience Inconsistent performance Copying from board Immaturity Math Risk taking Test taking Distractibility Hyperactivity Peer relationships Reading Spelling Self-esteem Motor skills Poor health Retaining information Speech Writing Attention Motivation p=0.01 p=0.004 p=0.01 p=0.04 p<0.001 Data presented at 63rd Annual American Academy of Child and Adolescent Psychiatry Meetings (AACAP) in Oct Odds Ratio, 95% Confidence Interval 2018, Aevi Genomic Medicine 14
15 Structural Summary Compared to ADHD mglur-, ADHD mglur+ have reduced volumes in the bilateral parietal cortices, as well as reduced connectivity between parietal cortices and posterior cingulate cortex Findings suggest genotypic effects on the structure and function of the default mode network, a neural network highly implicated in ADHD and attentional symptoms We will further evaluate whether the anatomical and functional changes are related to phenotypic changes seen in the 001 study We also plan a small trial to examine the effect of AEVI-001 on functional activity 2018, Aevi Genomic Medicine 15
16 AEVI-001 in ADHD 2018, Aevi Genomic Medicine
17 TRxs (Millions) ADHD Market is Large and Growing Total ADHD Sales in US = $11 billion 70 MM TRxs with a 5-year CAGR of 5.7% 92% Stimulants; 8% Non-Stimulants Growth vs. Prior Year 14.8% 16.1% 6.5% 0.2% 7.2% 11.8% 9.1% 12.5% 10.5% 4.6% 6.5% 4.9% 5.8% 5.6% Source: IMS, NPA, TRx 2018, Aevi Genomic Medicine 17
18 Current ADHD Treatments focus on Monoamine Neurotransmitter Pathways ADHD medications are thought to improve symptoms by increasing catecholamine neurotransmission primarily through Dopamine or Norepinephrine 2018, Aevi Genomic Medicine 18
19 Current ADHD Pharmacotherapy Aimed at Monoamines has Limitations The mainstay of treatment has been stimulants Stimulants have safety and tolerability issues: Cardiovascular risk Substance abuse Anxiety Appetite suppression Growth retardation Sleep disturbances Mood lability Non-stimulant options developed to address concerns regarding: Tolerability Safety/abuse Compliance concerns Have lower efficacy than stimulants with other serious safety concerns All currently available ADHD medications impact monoamine neurotransmitters including dopamine, norepinephrine, and serotonin 2018, Aevi Genomic Medicine 19
20 Glutamate Transmission in ADHD 2018, Aevi Genomic Medicine
21 Glutamate Amino acid neurotransmitter Most abundant excitatory neurotransmitter in mammals 1-3 Found throughout brain highly concentrated in hippocampus and amygdala 1 Plays key role in many brain functions including plasticity, learning, memory and nearly every major excitatory function of the human brain classes of receptor: ionotropic (iglur) and metabotropic (mglur) 1-3 Excessive glutamate is toxic by exciting neurons to death 3 Need right concentration at the right place and the right time for health Blood brain barrier shields brain from glutamate in the blood 3 Implicated in a number of diseases: Autism 4, Anxiety 5, ADHD, and Schizophrenia 6 1. Anwyl (2009) Neuropharmacology;56(4): Ohgi et al. (2015) Curr Mol Med;15(3): Zhou, Y. & Danbolt, N.C. J Neural Transm (2014) 121: Hadley et al. (2014) Nature Communications; 5: Swanson et al. (2005) Nat Rev Drug Discov; 4(2): Moghaddam, and Javitt, (2012) Neuropsychopharmacology Reviews, 37(1): , Aevi Genomic Medicine 21
22 Recent Evidence Supports the Role of Glutamate in ADHD and Other Disorders Neuroimaging studies suggest that glutamate levels are abnormal in children with ADHD 1 Increased glutamate in anterior cingulate cortex of children with ADHD 1. Spencer et al. (2014) J Clin Psychiatry;75(11): Moore, C.M. et al. (2006) Am. J. Psychiatry 163(2): , Aevi Genomic Medicine 22
23 AEVI-001: A Novel MOA for ADHD Oral, non-monoamine, non-stimulant CNS penetrant Well-characterized pharmacology Excellent preclinical and clinical safety profile O H N O N Shown to ameliorate cognitive impairment in animal behavioral models 1,2,3 Prior human exposure in vascular dementia: Originator: Nippon Shinyaku Developed through phase 3 Excellent safety profile Not sufficiently effective in dementia at doses tested (up to 300 mg per day) 1. Ogasawara et al. (1999) Pharmacol Biochem Behav, 64(1): Nakagawa et al. (1988) Jpn J Pharmacol, 46(Suppl.): Abst O Nakagawa et al. (1990) Jpn J Pharmacol, 52(Suppl. I): Abst P Oka et al. (1997) Naunyn Schmiedebergs Arch. Pharmacol, 356(2): Hirouchi et al. (2000) Eur J Pharmacol, 387(1): , Aevi Genomic Medicine 23
24 AEVI-001: First-in-class mglur Neuromodulator Indirect modulator of glutamatergic signaling Effects through multiple receptor pathways pre and post-synaptic 1. Oka et al. (1997) Naunyn Schmiedebergs Arch. Pharmacol, 356(2): Hirouchi et al. (2000) Eur J Pharmacol, 387(1): , Aevi Genomic Medicine 24
25 A Novel Approach to ADHD Precision Medicine rather than empirical therapy Therapy: novel mechanism of action Genomics: biomarker predictive of response Imaging: showing functional effect of the therapy Targeting specific comorbidities using genomic biomarker 2018, Aevi Genomic Medicine 25
26 SAGA Trial Results 2018, Aevi Genomic Medicine
27 SAGA Trial Design in mglur+ Adolescents Multicenter (24 sites) Inclusion criteria: Adolescents (12-17 years of age) ADHD-RS-5 28 Positive for mglur mutation Primary endpoint: ADHD-RS-5 Key secondary endpoint: CGI-I 1:1 randomization Placebo-controlled Dose optimization: mg BID *Elia et al. (2012) Nat Genet, 44(1): , Aevi Genomic Medicine 27
28 Phase II SAGA Study: Responder Analysis Post-hoc analysis of patients responding to AEVI-001 revealed a 9-gene subset predictive of response to AEVI-001 accounts for approximately 10% of pediatric ADHD patients. 1 Novel discovery: prevalence of CNTN4 mutations in ADHD Represents approximately 5% of pediatric ADHD All CNTN4 mutation+ patients on AEVI-001 achieved clinical response More severe ADHD phenotype with emotional dysregulation features CNTN4 mutation association with neurodevelopmental diseases supports study in ASD Potential to launch with a more concentrated but predictive genotype and with a first-ever biomarker 1 Data on file and Elia, J. et al, Glutamatergic network gene mutations in children and adolescents with ADHD. Poster presented at: 6th World Congress on ADHD; April 21, 2017; Vancouver, Canada 2018, Aevi Genomic Medicine 28
29 Change in ADHD-RS Score from Baseline Change in ADHD-RS Score from Baseline Comparative Analysis of 9-Gene Subset from the SAGA Trial Change in ADHD Symptoms 0 SAGA Change in ADHD-RS (N=96) 0 SAGA 9-Gene Subset Change in ADHD-RS (n=42) Placebo AEVI-001 Placebo AEVI n=24-10 n=50 p=ns -10 Δ = 11.7 p = n=46-15 n=18-20 Baseline Week 1 (100 mg BID) Week 2 (200 mg BID) Week 3 (400 mg BID) Week 4 (Optimized Dose) Week 5 (Maintenance Dose) Week 6 (Maintenance Dose) -20 Baseline Week 1 (100 mg BID) Week 2 (200 mg BID) Week 3 (400 mg BID) Week 4 (Optimized Dose) Week 5 (Maintenance Dose) Week 6 (Maintenance Dose) ADHD-RS: parent reported scale on hyperactivity and inattention, 18 questions; scored , Aevi Genomic Medicine 29
30 Percentage of Total ADHD Frequency of Responder Genes to AEVI-001 Total set of 9 genes represents approximately 10% of the pediatric ADHD population 1 Through our validation process we decided to drop Neuro Gene E (<.5% of ADHD patients) which didn t validate on the newer version of the Omni 2.5 gene chip CNTN4 Neuro Gene A 9-Gene Responder Subset in SAGA Trial (n=42) GRM Gene A GRM Gene B Neuro Gene B Neuro Gene C Neuro Gene D Neuro Gene E Neuro Gene F 1 Data on file and Elia, J. et al, Glutamatergic network gene mutations in children and adolescents with ADHD. Poster presented at: 6 th World Congress on ADHD; April 21, 2017; Vancouver, Canada 2018, Aevi Genomic Medicine 30
31 AEVI (ASCEND) 2 Part (A,B) Sequential Adaptive Confirmatory Efficacy Trial in mglur+ ADHD Multi-center (20-25 US sites) Inclusion criteria: 6-17 years of age ADHD-RS-5 28 Positive for 8 gene subset, including CNTN4 (Part A) - or - non-mutated (Part B) Primary endpoint: ADHD-RS-5 Key secondary endpoint: CGI-I 1:1 randomization Placebo-controlled Dose optimization: mg BID Screening Period (up to 4 weeks inclusive of washout, if applicable) Dose-optimization Period (4 weeks) Dose Maintenance Period (2 weeks) Visit #: /ET Dose: 100 mg BID, 200 mg BID, or 400 mg BID, Dose optimization based on response assessment Maintenance on optimal dose End of treatment: Day 42 Follow-up Call (1 week) Week: , Aevi Genomic Medicine 31
32 9-Gene Subset Responders: ADHD Total Addressable Market Overall US ADHD Market US market sales: $11 billion 1 Stimulants dominate market: ~90 % of sales 1 ~6M pediatric (ages 6-17) patients and ~10M adult patients in US 2,3 8-Gene Subset ADHD Market (~10% of ADHD patients) 4 ~600K pediatric patients and ~1M adult patients $2B - $3B market size based upon current brand pricing Additional lifecycle opportunities where mglur mutation are found: ASD, Pediatric Anxiety, Tourette s, Bipolar, etc. 1.IMS NSP, (Visser S et al Journal of American Academy of Child and Adolescent Psychiatry 2014, 53(1):34-46) 3. (Kessler R et al American Journal of Psychiatry 2006, 163(4):716-23) 4.Data on file and Elia, J. et al, Glutamatergic network gene mutations in children and adolescents with ADHD. Poster presented at: 6 th World Congress on ADHD; April 21, , Aevi Genomic Medicine 32
33 Fasoracetam Co-crystal 2018, Aevi Genomic Medicine
34 Pharmaceutical Co-Crystals pharmaceutical Co-crystals offer the possibility of exploring different pharmaceutical properties and enhancing the final drug product 2,3 Schultheiss N., Newman A. Crystal Growth & Design 2009, 9: , Aevi Genomic Medicine 34
35 Co-crystal Pharmaceuticals: Recent FDA Guidance Regulatory Classification of Pharmaceutical Co-Crystals Guidance for Industry 1 Regulatory Classification of Pharmaceutical Co-Crystals Guidance for Industry was issued February Farxiga (dapagliflozin propanediol) Entresto (sacubitril/valsartan) 2018, Aevi Genomic Medicine 35
36 Aevi-004 Co-crystal: Advantages AEVI-004, a co-crystal of fasoracetam, has enhanced physical properties: Stability Higher Melting point AEVI-004 was engineered to maintain: Solubility Permeability Bioavailability Maintained Bolla & Nangia, Chem. Comm. 54: , , Aevi Genomic Medicine 36
37 FDA feedback on AEVI-004 FDA provisionally agreed that AEVI-004 is a co-crystal and therefore NCE eligible. FDA provisionally agreed existing tox path studies are acceptable to support AEVI-004 with minimal preclinical bridging studies Switching to AEVI-004 for Phase 3 should have minimal impact on program cost and timing 2018, Aevi Genomic Medicine 37
38 ADHD AEVI-001 Regulatory Exclusivity Hatch-Waxman Data Exclusivity 5 years for NCE EMA Exclusivity (NCE) 10 years Aevi-001 Additional Test and Treat Patent Term Exclusivity = 20 years Aevi-004 Co-crystal Composition of Matter Patent Term Exclusivity = 20 years Provisional Patents Filed 2019 Note: Hatch-Waxman exclusivity starts upon approval of NDA by FDA 2018, Aevi Genomic Medicine 38
39 AEVI-001: Next Steps On track to complete ASCEND (8-Gene Mutation) by Q Part B of ASCEND in mglur- ADHD to complete after Part A results will allow comparison of response rates and safety in ADHD patients with and without the mutation Hold End of Phase II meeting with FDA to determine requirements for Phase 3 program post completion of ASCEND Genetic biomarker development continuing in parallel to support Phase 3 and launch 2018, Aevi Genomic Medicine 39
40 Aevi Genomic Medicine Pipeline Compound Indication Preclinical Phase 1 Phase 2 Status AEVI-001 AEVI-002* (anti-light mab) AEVI-005* mglur+ Genetic Subset in ADHD (pediatric, age 6-17) mglur+ Genetic Subset in ASD Severe Pediatric Onset Crohn s Disease Undisclosed pediatric rare disease Top-Line Data Q Plan to Initiate Q Initial Data Year-end 2018 Initiating in vitro POC work * Partnered with KHK 2018, Aevi Genomic Medicine 40
41 Financial Update $26M cash and cash equivalents at 3/31/2018 Current cash resources estimated to fund operations into early 2019, including through the receipt of: Top-line data from AEVI-001 in the ASCEND trial in mglur+ Genetic Subset ADHD (Q4 2018) Initial data from the signal-finding trial of AEVI-002 in Severe Pediatric Onset Crohn s Disease (year-end 2018) 2018, Aevi Genomic Medicine 41
42 Summary ADHD is a large, growing market that remains unsatisfied AEVI-001 is a novel non-stimulant with a unique MOA Potentially the first ADHD medication with a genetic biomarker to identify patients who could most benefit from the product Projected to report results of ASCEND POC study in patients with 8-gene mutation by Q Additional potential for AEVI-001 in other psychiatric conditions with 8-gene mutation Aevi has sufficient funding into early , Aevi Genomic Medicine 42
43 Thank you 2018, Aevi Genomic Medicine 43
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