Avalo Therapeutics, Inc. · NASDAQ:AVTX
Avalo Therapeutics — Cerecor — Investor Presentation (July 2019)
31 slides from Avalo Therapeutics's investor presentation, Jul 10, 2019
Slide 1: Cerecor Corporate Highlights July 2019
Exhibit 99.1 Cerecor Corporate Highlights July | 2019
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation may include forward-looking statements made pursuant to the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts. Such forward-looking statements are subject to significant risks and unc
Slide 3: Cerecor is an integrated biopharmaceutical company
Cerecor is an integrated biopharmaceutical company developing innovative therapies at the cutting edge of science. Our pipeline filled with forward-thinking ideas propels us forward. We are driven to change the lives of patients with rare orphan diseases in pediatrics and neurology. Driven by Scienc
Slide 4: Cerecor Today
Cerecor Today Focused on Advancing R&D, while Optimizing Commercial Capabilities • Emerging clinical & early-stage pipeline Innovative • Three 505(b)(2) programs with expedited path to NDA Pipeline • Focus on orphan, neurological & pediatric diseases Commercial • Building a commercial capability Foo
Slide 5: Overview
Overview 1 Management Team 2 Historical Milestones 3 Pediatric Rare Disease & Neurology Pipeline 4 Commercial Pediatric Portfolio 5 Strategic Growth Plans and Outlook 6 Financial Highlights 5|
Slide 6: Management Team
Management Team Extensive experience in development and commercialization 30+ years industry experience 8+ years industry experience • President and CEO of Chelsea Therapeutics • Chairman, President at Ichorion Therapeutics Dr. Simon • Chief Business and Strategy Officer, Patrick • SVP, Business Dev
Slide 7: Historical Milestones
Historical Milestones Management Change 2013 2014 2015 2016 2017 2018 2019 CERC-301 Patent Granted CERC-301 Cerecor IPO Acquisition Acquisition to 2035 CERC-406 October 15, 2015 TRx Pharma Avadel (AVDL) (Zylera) Pediatric assets Acquired worldwide CERC-800s rights of two NMDA 3 Orphan Drug receptors
Slide 8: Cerecor Evolution
Cerecor Evolution Neurological Disorders Pediatric Franchise Pediatric Rare Diseases Innovative Approaches FDA-Approved 505(b)(2) Assets & to CNS Diseases Products Platform Chemistry • CERC-301 • Poly-Vi-Flor® | Tri-Vi-Flor® • CERC-801 • CERC-406 • Karbinal® ER • CERC-802 • CERC-501 • AcipHex® Sprin
Slide 9: Emerging Clinical & Early-Stage Pipeline
Emerging Clinical & Early-Stage Pipeline Mechanism of Program Target Indication Development Stage Action D-Galactose CERC-801 PGM1 Deficiency Phase 1 505(b)(2) replacement D-Mannose CERC-802 MPI Deficiency replacement IND-Enabling 505(b)(2) L-Fucose SLC35C1-CDG CERC-803 replacement (CDG-IIc) IND-Ena
Slide 10: Market Potential
Market Potential World-Wide Estimated Patient Populations 290M Ultra Rare Larger CNS 185M Indications Pediatric Orphan Diseases OH >65 Develop Internally & Early Clinical (Ph l/2) Commercialize Independently 100M Proof-of-Concept 40M DM OH with Optionality 1.9M 2.9M SCI OH 1.5M 500 500 nOH 250 50 50
Slide 11: R&D Milestones
R&D Milestones Multiple value generating inflection points over next 12 to 18 months Program Target Indication Upcoming Milestone CERC-801* PGM1 Deficiency FDA Discussion - 2H19 CERC-802* MPI Deficiency IND Acceptance – 2H19 Disorders Metabolic CERC-803* SLC35C1-CDG (CDG-IIc) IND Filing - 2020 MOA S
Slide 12: Pediatric Rare Disease Portfolio
Pediatric Rare Disease Portfolio
Slide 13: Congenital Disorders of Glycosylation (CDGs)
Congenital Disorders of Glycosylation (CDGs) Orphan diseases with an estimated prevalence of 1 in 100,000 • Genetic diseases that result in impaired glycoprotein production and function • Glycoproteins are critical for cell structure and function, particularly for circulating proteins and enzymes su
Slide 14: CERC-800s Substrate replacement therapies for CDGs
CERC-800s Substrate replacement therapies for CDGs Monogenic disorders resulting in glycosylation defects with broad clinical spectrum, including life-threatening complications Multi-system disease manifestation in PGM1 Deficiency CERC-801 D-Galactose leads to significant improvement in key clinical
Slide 15: CERC-800s Substrate replacement therapies for CDGs
CERC-800s Substrate replacement therapies for CDGs Oral, small molecule, naturally occurring monosaccharides used as standards-of-care for CDGs • Safe • Efficacious • Rapid Onset of Action D-Galactose D-Mannose L-Fucose Eligibility CERC-801 CERC-802 CERC-803 505(b)(2) NDA Pathway ✓ ✓ ✓ NCE 5-yrs Exc
Slide 16: The Value of a Pediatric Review Voucher
The Value of a Pediatric Review Voucher PRV Sales to Date with Purchase Amount 400 The Mean purchase price over the past 5 years has been $140M $350 350 The Median purchase price over the past 5 years has been $125M 300 $245 250 $200 200 150 $125 $125 $130 $125 $130 $110 $105 100 $80 $81 $80 $68 50
Slide 17: CERC-800s Retrospective chart reviews and registry data
CERC-800s Substrate replacement therapies for CDGs Retrospective chart reviews & registry data have been successfully used to minimize or obviate prospective clinical studies Developer Therapeutic (Indication) Pivotal Study Strategy Retrospective case series summary (13/23 Carbaglu patients with com
Slide 18: CERC-913 ProTide Nucleotide for DGUOK Deficiency
CERC-913 ProTide Nucleotide for Deoxyguanosine Kinase (DGUOK) Deficiency Overcome key limitations of direct substrate replacement: Stability, Permeability & Kinase Bypass Phenotypic Rescue CERC-913 Attributes • Proof-of-concept in patient-derived & animal-based disease models • ProTide similar to ad
Slide 19: Neurology Disorders
Neurology Disorders
Slide 20: CERC-301 NR2B selective NMDA receptor antagonist for nOH
CERC-301 NR2B selective NMDA receptor antagonist for nOH Autonomic nervous system fails to regulate vasoconstriction due to underlying neurological disease • Rapid and significant (20mm Hg) drop in blood pressure caused by postural change • Increased risk of falls or injury, leading to decreased qua
Slide 21: CERC-301 Phase 1 SAD in PD patients with nOH
CERC-301 NR2B selective NMDA receptor antagonist for nOH Opportunity to rapidly demonstrate proof-of-concept in patients Phase 1 SAD in PD patients with nOH Visit 1 Visit 2 Visit 3 Visit 4 Visit 5a Arm 1 Enrollment • 12 active centers in US pbo 8 mg 12 mg 16 mg 20 mg (n = 5) • N = 20 (8, 12, 16 & 20
Slide 22: CERC-301 Positive Phase 1 results
CERC-301 NR2B selective NMDA receptor antagonist for nOH Positive Phase l results depict a rapid, robust and sustain increase in Systolic Blood Pressure in Parkinson’s Patients with nOH • Top-line results demonstrate rapid, sustained and clinically Orthostatic Standing Test – 20 mg vs PBO; Pre-dose
Slide 23: CERC-406 COMT inhibitor for Parkinson's Disease
CERC-406 COMT inhibitor for Parkinson’s Disease Next generation, CNS-penetrant COMT inhibitor to enhance efficacy and minimize toxicity seen with 1st generation therapies • As PD progresses the therapeutic window of L-Dopa becomes smaller, increasing the on/off periods Extended ‘on’ Time A Typical D
Slide 24: CERC-406 COMT inhibition biomarker
CERC-406 COMT inhibitor for Parkinson’s Disease COMT inhibition provides an in vivo biomarker for target engagement by measuring DOPAC and HVA • COMT regulates dopamine (levodopa) breakdown via the conversion of dihydroxyphenyl-acetic acid (DOPAC) to homovanillic acid (HVA) 1st Gen COMT Drawbacks /
Slide 25: CERC-611 AMPA receptor antagonist for partial onset seizures
CERC-611 TARP-ɣ8 dependent AMPA receptor antagonist for partial onset seizures Phase 1-ready candidate with therapeutic potential for partial onset seizures in patients with epilepsy Significant Unmet Need Unique Mechanism of Action • Epilepsy affects over 65 million • AMPA receptors mediate fast pa
Slide 26: Commercial Pediatric Portfolio
Commercial Pediatric Portfolio
Slide 27: Building Commercial Capabilities in Pediatrics
Building Commercial Capabilities in Pediatrics Eight commercial products promoted across 42 U.S. Territories Fully-integrated commercial team with revenues reinvested into operations and R&D Capability-Building: Sales, Operations, Regulatory, Marketing and Training 27|
Slide 28: Why Pediatrics?
Why Pediatrics? Pediatrics Represents a Focused, Defined and Specific Patient Population Treated by One Specialty Segment Our Existing Pediatric Product Portfolio May be Used in Up To 75% of the Top 25 Pediatric Diagnosis Codes Top 25 Pediatric Codes 2013 AAP Pediatric Coding Newsletter1 1. Routine
Slide 29: Financials
Financials Net Product Sales Last 4 QTR Trend ($ Millions) FY19 Net Sales Guidance = $20 to $22 Million Cash Trend Analysis ($ Millions) 29|
Slide 30: 2019 Growth Plans
2019 Growth Plans 1 2 3 Accelerate Business Advance Build Commercial Development Pipeline Excellence Activity CERC-801 Grow Market Share Acquire/in-license commercial-ready or CERC-802 Expand Commercial marketed asset(s) Footprint CERC-803 Acquire/in-license CERC-301 complimentary pipeline assets CE
Slide 31: Driven by Science, Inspired by Hope
Driven by Science NASDAQ:CERC www.cerecor.com Inspired by Hope