Avalo Therapeutics, Inc. · NASDAQ:AVTX
Avalo Therapeutics — Cerecor — Investor Presentation (January 2019)
33 slides from Avalo Therapeutics's investor presentation, Jan 18, 2019
Slide 1: Forward-Looking Statements
Cerecor Inc. 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
Slide 2: Driven by Science Inspired by Hope
Driven by Science Inspired by Hope 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 pediatr
Slide 3: Cerecor Today
Cerecor Today Building a Robust Biotech Pipeline of Therapies in Neurology, Pediatric and Orphan Rare Diseases While Developing Commercial Capabilities with our Revenue Producing Pediatric Franchise Focused on Research and Development While Building Our Commercial Capabilities
Slide 4: Corporate Highlights
Corporate Highlights Emerging clinical & early-stage pipeline Focus on orphan, neurological & pediatric indications Innovative Pipeline Building a commercial capability Pediatric franchise generating positive cash flow Commercial Footprint Fully-integrated commercial and R&D organization New managem
Slide 5: Overview
Overview Management Team 1 Historical Milestones 2 Neurology & Pediatric Rare Disease Pipeline 3 Commercial Pediatric Portfolio 4 Strategic Growth Plans and Outlook 5 Financial Highlights 6
Slide 6: Management Team
Management Team 20+ years industry experience Chairman and CEO, Sucampo Pharmaceuticals CEO, Histogenics Corporation President, MedImmune Ventures Manager, Centocor Biotech (Johnson & Johnson) 20+ years industry experience V.P. Global CMC & Development, Sucampo Pharmaceuticals CSO, Pharming Group Sr
Slide 7: Historical Milestones
Historical Milestones 2017 2013 2016 2015 2014 CERC-301 CERC-406 Acquired worldwide rights of two NMDA receptors from Merck CERC-611 Acquired worldwide rights from Eli Lilly CERC-501 Sold to Janssen CERC-501 Acquired worldwide rights from Eli Lilly 2018 CERC-301 Enters Phase 1 study for nOH Acquisit
Slide 8: Cerecor Evolution
Cerecor Evolution FDA-Approved Products Poly-Vi-Flor® Tri-Vi-Flor® Karbinal™ ER AcipHex® Sprinkle™ Cefaclor Flexichamber™ Millipred® Veripred® Ulesfia® Innovative Approaches to CNS Diseases CERC-301 CERC-406 CERC-501 CERC-611 505(b)(2) Assets & Platform Chemistry CERC-801 CERC-802 CERC-803 CERC-913
Slide 9: Emerging Clinical & Early-Stage Pipeline
Program Mechanism of Action Target Indication Lead Opt IND-Enabling Phase 1 Neurology Division NCE CERC-301 GluN2B selective, NMDA Receptor antagonist Neurogenic Orthostatic Hypotension (nOH) CERC-406 CNS-targeted, selective COMT inhibitor (2nd Gen) Motoric and non-motoric symptoms of Parkinson’s CE
Slide 10: Market Potential
Market Potential World-Wide Estimated Number of Patients 50 250 500 700k 1.9M 500 nOH Parkinson’s Disease CDG PGM1 Deficiency DGUOK Deficiency CDG MPI Deficiency CDG-IIc Ultra Rare Pediatric Orphan Diseases Epilepsy 40M CERC-301 CERC-406 CERC-611 CERC-801 CERC-802 CERC-803 CERC-913
Slide 11: R&D Milestones
*505(b)(2) Pathway R&D Milestones Multiple value generating inflection points over next 6 to 12 months Neurological Disorders CERC-301 Neurogenic Orthostatic Hypotension Phase I Data 1H19 CERC-406 Adjunct for Parkinson’s Disease IND Filing 1H20 CERC-611 Partial onset seizures in epilepsy In Process
Slide 12: Neurogenic Orthostatic Hypotension (nOH)
Neurogenic Orthostatic Hypotension (nOH) CERC-301 NR2B selective NMDA receptor antagonist for nOH Initiated Phase 1 study of CERC-301 in Parkinson’s patients diagnosed with neurogenic Orthostatic Hypotension (nOH) Caused by a failure of the autonomic system to regulate BP upon postural change, incre
Slide 13: CERC-301 NR2B selective NMDA receptor antagonist for nOH
CERC-301 NR2B selective NMDA receptor antagonist for nOH Multiple exposures in >375 subjects, providing robust safety data & blood pressure effect Dose-dependent increase in SBP Long-term clinical benefit Rapid onset of action Superior safety profile than midodrine Use as stand-alone or adjunctive w
Slide 14: CERC-301 Phase 1 SAD in PD patients with nOH
a Randomized 4:1 CERC-301 NR2B selective NMDA receptor antagonist for nOH Opportunity to rapidly demonstrate proof-of-concept in patients Visit 1 Visit 2 Visit 3 Visit 4 Visit 5a Arm 1 (n = 5) pbo 8 mg 12 mg 16 mg 20 mg Arm 2 (n = 5) 8 mg pbo 12 mg 16 mg 20 mg Arm 3 (n = 5) 8 mg 12 mg pbo 16 mg 20 m
Slide 15: CERC-406 COMT inhibitor for Parkinson's Disease
2nd generation, CNS-selective COMT inhibitor to enhance efficacy and minimize toxicity seen with 1st generation therapies CERC-406 COMT inhibitor for Parkinson’s Disease Dopamine “replacement” therapy can effectively control PD symptoms, but over time patients may begin to experience “on/off” episod
Slide 16: CERC-406 Our Lead Candidate
CERC-406 COMT inhibitor for Parkinson’s Disease – Our Lead Candidate Biomarkers for translational proof-of-concept with promising drug properties High specificity for COMT expressed in the CNS Good oral bioavailability & brain penetration Acceptable PK profile in rat & dog Low potential for drug to
Slide 17: 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 Epilepsy affects over 65 million patients worldwide 30%-40% of patients refractory; high degree of poly-pharmacy comm
Slide 18: Ichorion Acquisition
Ichorion Acquisition All stock ($26M) transaction announced on September 25th, 2018 Strategic, Clinical & Commercial Fit to Accelerate Transformation Private biopharma focused on therapies for ultra-rare pediatric diseases White-space opportunity that compliments Cerecor’s existing commercial footpr
Slide 19: CERC-800s Substrate replacement therapies for CDGs
D-Mannose D-Galactose L-Fucose Eligibility CERC-801 CERC-802 CERC-803 505(b)(2) NDA Pathway Ö Ö Ö NCE 5-yrs Exclusivity Ö Ö Ö ODD 7-yrs Exclusivity Ö Ö Ö Priority Review Voucher Ö Ö Ö EMA ODD 10-yrs Exclusivity Ö Ö Ö CERC-800s Substrate replacement therapies for CDGs Oral, small molecule, naturally
Slide 20: 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 CERC-801 Multi-system disease manifestation in PGM1-CDG D-Galactose leads to significant improvement in key clinical sympto
Slide 21: CERC-800s development framework leveraging 505(b)(2) pathway
Secure use rights for KOL clinical datasets Primary data strengthen regulatory submissions CERC-800s Substrate replacement therapies for CDGs CERC-800s development framework leveraging 505(b)(2) pathway Leverage published literature to demonstrate efficacy & safety Clinical & nonclinical data availa
Slide 22: CERC-800s Regulatory precedents
Developer Therapeutic (Indication) Pivotal Study Strategy Carbaglu (NAGS Deficiency) Retrospective case series summary (13/23 patients with complete data) & 3 patients treated prospectively (2010) Cholbam (Bile Acid Disorders) Case report form from retrospective chart review of patients in open-labe
Slide 23: CERC-913 ProTide for DGUOK Deficiency
Phenotypic Rescue Proof-of-concept in patient-derived & animal-based disease models ProTide similar to advanced clinical candidates & approved drugs Metabolite ID & PK profile in dog support translational PKPD CERC-913 Attributes CERC-913 ProTide Nucleotide for Deoxyguanosine Kinase (DGUOK) Deficien
Slide 24: Overview
Overview Management Team 1 Historical Milestones 2 Neurology & Pediatric Rare Disease Pipeline 3 Commercial Pediatric Portfolio 4 Strategic Growth Plans and Outlook 5 Financial Highlights 6
Slide 25: Building Commercial Capabilities in Pediatrics
~50% of the Overall Market Potential Covered with 32 Territories Sales Force Expansion Underway to Increase SOV Building Commercial Capabilities in Pediatrics 1 Chief Commercial Officer 1 EVP Marketing & Investor Relations 1 Director of Sales 3 Regional Sales Managers 1 Director of Sales Training an
Slide 26: Pediatric Franchise with Eight Product Lines
Pediatric Franchise with Eight Product Lines Zylera Pharmaceuticals Avadel Pediatric Assets
Slide 27: Why Pediatrics?
ICD-10-CM codes are displayed as 24 code categories that include the 25 diagnoses from the International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM) list (2 otitis media codes were included in ICD-9-CM). 1. AAP pediatric coding newsletter coding.aap.org August 2013 W
Slide 28: Net Product Sales
Net Product Sales Net Product Sales Last 4 QTR Trend (in $000’s) FY18 Net Sales Guidance = $18 to $20 million 0 500 1,000 1,500 2,000 2,500 3,000 3,500 4,000 4,500 5,000 Q4 2017 Q1 2018 Q2 2018 Q3 2018
Slide 29: Balance Sheet
Balance Sheet Cash Trend Analysis (in $000’s) Total Assets Trend Analysis (in $000’s) 0 1,000 2,000 3,000 4,000 5,000 6,000 7,000 8,000 Q4 2017 Q1 2018 Q2 2018 Q3 2018 0 10,000 20,000 30,000 40,000 50,000 60,000 70,000 Q4 2017 Q1 2018 Q2 2018 Q3 2018
Slide 30: 2019 Growth Plans
2019 Growth Plans Build Commercial Excellence Accelerate Business Development Activity Advance Pipeline Grow Market Share Expand Commercial Footprint Acquire/in-license commercial-ready or marketed asset(s) Acquire/in-license complimentary pipeline assets CERC-301 CERC-406 CERC-611 CERC-801 CERC-802
Slide 31: www.cerecor.com
NASDAQ:CERC www.cerecor.com
Slide 32: Driven by Science Inspired by Hope
Driven by Science Inspired by Hope
Slide 33: Cerecor
( CERECOR