Avalo Therapeutics, Inc. · NASDAQ:AVTX
Avalo Therapeutics — Cerecor — Investor Presentation (March 2019)
29 slides from Avalo Therapeutics's investor presentation, Mar 20, 2019
Slide 1: Cerecor Corporate Highlights March 2019
Exhibit 99.1 Cerecor Corporate Highlights March | 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: Integrated biopharmaceutical company for rare orphan diseases
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 Neurology & Pediatric Rare Disease 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 20+ years industry experience 25+ years industry experience • Chairman and CEO, Sucampo • President and COO of Zylera Pharmaceuticals Matthew V. Peter S. Pharmaceuticals • Executive Director, Victory Pharma Phillips Greenleaf
Slide 7: Historical Milestones
Historical Milestones Management Change 2013 2014 2015 2016 2017 2018 CERC-301 Cerecor IPO Acquisition Acquisition CERC-406 October 15, 2015 TRx Pharma Avadel (AVDL) (Zylera) Pediatric assets Acquired worldwide rights of two NMDA receptors from Merck CERC-501 CERC-611 CERC-501 Acquisition Acquired w
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-Flor̺ | Tri-Vi-Flor̺ • CERC-801 • CERC-406 • Karbinalဆ ER • CERC-802 • CERC-501 • AcipHex̺ 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 replacement Phase 1 505(b)(2) D-Mannose CERC-802 MPI Deficiency replacement IND-Enabling 505(b)(2) L-Fucose CERC-803 CDG-IIc replacement IND-Enabling 505(b)(2
Slide 10: Market Potential
Market Potential World-Wide Estimated Patient Populations Develop Internally & Early Clinical (Ph1/2) Commercialize Independently Proof-of-Concept with Optionality 40M Larger CNS Indications 1.9M Ultra Rare Pediatric Orphan Diseases 700k 500 500 250 50 CERC-801 CERC-802 CERC-803 CERC-913 CERC-301 CE
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 Phase I Data 1H19 CERC-802* MPI Deficiency IND Filing 1H19 CERC-803* CDG-IIc IND Filing 2020 Disorders Metabolic CERC-913 DGUOK Deficiency IND F
Slide 12: 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 13: 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 14: CERC-800s Naturally occurring monosaccharides and eligibility
CERC-800s Substrate replacement therapies for CDGs Oral, small molecule, naturally occurring monosaccharides used as standards-of-care for CDGs OH • Safe HO O HO O • Efficacious HO OH HO OH • Rapid Onset of Action OH OH D-GalactoseD-Mannose L-Fucose Eligibility CERC-801 CERC-802 CERC-803 ض ض ض b)(2)
Slide 15: 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 16: 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 17: 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 1 in 5 to decrea
Slide 18: CERC-301 Phase 1 study in Parkinson's patients with nOH
CERC-301 NR2B selective NMDA receptor antagonist for nOH Initiated Phase 1 study of CERC-301 in Parkinson’s patients diagnosed with nOH CERC-301 Clinical Database • Established safety profile in ~400 patients and healthy volunteers • Multiple studies demonstrate rapid, durable & dose-dependent incre
Slide 19: CERC-301 Phase 1 proof-of-concept study design
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 20: 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 21: 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 22: 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 23: Overview
Overview 1 Management Team 2 Historical Milestones 3 Neurology & Pediatric Rare Disease Pipeline 4 Commercial Pediatric Portfolio 5 Strategic Growth Plans and Outlook 6 Financial Highlights 23|
Slide 24: 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 24|
Slide 25: 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 26: Net Product Sales
Net Product Sales Net Product Sales Last 4 QTR Trend ($ Millions) $6 $5 $4 $3 $2 $1 $0 Q1 2018 Q2 2018 Q3 2018 Q4 2018 FY19 Net Sales Guidance = $20 to $22 Million 26|
Slide 27: Balance Sheet
Balance Sheet Cash Trend Analysis ($ Millions) 20,000 17,500 15,000 12,500 10,000 7,500 5,000 2,500 0 Q1 2018 Q2 2018 Q3 2018 Q4 2018 Mar 10 2019* Total Assets Trend Analysis ($ Millions) 80,000 70,000 60,000 50,000 40,000 30,000 20,000 10,000 0 Q1 2018 Q2 2018 Q3 2018 Q4 2018 Mar 10 2019* 27| *Esti
Slide 28: 2019 Growth Plans
2019 Growth Plans 1 2 3 Accelerate Business Advance Build Commercial Development Pipeline Excellence Activity CERC-301 Grow Market Share Acquire/in-license commercial-ready or CERC-406 Expand Commercial marketed asset(s) Footprint CERC-611 Acquire/in-license CERC-801 complimentary pipeline assets CE
Slide 29: Driven by Science, Inspired by Hope
Driven by Science NASDAQ:CERC www.cerecor.com Inspired by Hope