Avalo Therapeutics, Inc. · NASDAQ:AVTX
Avalo Therapeutics — Cerecor — Investor Presentation (June 2020)
56 slides from Avalo Therapeutics's investor presentation, Jun 1, 2020
Slide 1: Patient Inspired Science
Exhibit 99.1 Patient Inspired Science Establishing a leading, pediatric rare and orphan disease-focused biopharmaceutical company to deliver impactful new medicines to patients June | 2020 Corporate Highlights | Focus on COVID-19 ARDS
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: Highlights
Highlights • Recent Merger of Cerecor and Aevi has created a rich pipeline of novel, 1st in class assets all with proven mechanistic rationale • Only known anti-LIGHT mAb in the clinic, offers potential to treat cytokine storm-induced COVID-19 ARDS in the near-term and broader ARDS indication beyond
Slide 4: Clinical-Stage Pipeline
Clinical-Stage Pipeline Core Research & Development Stage Therapeutic Mechanism Development Program Lead Indication Area of Action Areas Preclinical Phase 1 Phase 2 Phase 3 Upcoming Milestone Initial Data Anti-LIGHT mAb ARDS CERC-002 4Q 2020 Initial Data Immunology Inflammation Anti-LIGHT mAb Crohn’
Slide 5: CERC-007 Anti-IL-18 mAb for Multiple Myeloma & AOSD
CERC-007 Phase 2-ready, anti-IL-18 monoclonal antibody for Multiple Myeloma & Adult Onset Still’s Disease
Slide 6: Phase II-Ready Asset for Multiple Myeloma and AOSD
Phase II-Ready Asset for Multiple Myeloma (MM) and Adult Onset Still’s Disease (AOSD) First-in-class, only fully human anti-IL-18 mAb with potential to address multiple tumor types and inflammatory conditions Strong • Elevated IL-18 is correlated with poor survival in MM patients and disease Scienti
Slide 7: Strong Potential in Multiple Myeloma
Strong Potential in Multiple Myeloma Elevated IL-18 levels correlate with poor survival in multiple myeloma patients IL-18 Levels Are Elevated in Many MM Patients and Correlate with Poor Survival (n=69) (n=76) • Patients with high IL-18 have significantly worse median survival (42 months vs. >84 mon
Slide 8: Additional Targets within IL-18-Mediated Autoimmune Disorders
Additional Targets within IL-18-Mediated Autoimmune Disorders IL-18 levels correlate with AOSD severity Elevated Serum IL-18 Levels in AOSD Patients IL-18bp Response Rates 1000000 80 100000 10000 60 * 18 (pg/ml) 1000 40 100 Serum IL - 10 Active AOSD in AOSD in Comparison Comparison 20 AOSD Partial R
Slide 9: First-in-Class Anti-IL-18 High Affinity Monoclonal Antibody
First-in-Class Anti-IL-18 High Affinity Monoclonal Antibody Data from phase 1 study demonstrated favorable PK and safety profile • Potent and durable IL-18 inhibition – Evaluated in phase 1 SAD for COPD (n = 31) – IV doses of 10, 30, 100, 300 or 1000 mg – Well-tolerated • Phase 2-ready asset – 13-we
Slide 10: Program Update as of June 2020
Program Update as of June 2020 Anticipate proof-of-concept trial initiation Q4 2020 in both MM and AOSD • Pre-IND meeting with the FDA completed, concurrence on high-level design • CRO under contract (PRA); sites selected; study start anticipated early 4Q 2020 Multiple Myeloma • Classic 3+3 dose esc
Slide 11: CERC-006 Dual mTORC 1/2 Inhibitor for Complex Lymphatic Malformations
CERC-006 Phase 2-ready, Dual mTORC 1/2 small molecule inhibitor for Complex Lymphatic Malformations
Slide 12: Phase II-Ready Asset for Complex Lymphatic Malformations
Phase II-Ready Asset for Complex Lymphatic Malformations Potential first-in-class potent inhibitor of mTORC1/mTORC2 High Unmet • Orphan disease(s) with combined US prevalence of 30 to 60k associated with Need high mortality rates of 20% to 50% over 3 to 7 years Demonstrated • Off label use of siroli
Slide 13: Complex Lymphatic Malformations Are Potentially Life-threatening Congenital Diseases
Complex Lymphatic Malformations Are a Family of Potentially Life-threatening Congenital Diseases • Neoplastic lesions caused by mutations in Lymph Valve PI3K/AKT/mTOR pathway • Leads to local proliferation of lymphatic endothelial cells and perturbation of lymph flow – Fluid accumulation in limbs, a
Slide 14: High Potency, Second Generation, Dual Inhibitor of mTORC1/2
High Potency, Second Generation, Dual Inhibitor of mTORC1/2 Potential for improved efficacy and tolerability • Phase 2-ready asset – 4-week nonclinical tox studies completed – Previously studied in Phase 1 MAD (n = 128) – Development discontinued upon determination that target efficacious doses were
Slide 15: Program Update as of June 2020
Program Update as of June 2020 Anticipate proof-of-concept trial initiation Q1 2021 • Pre-IND meeting with the FDA completed, concurrence on inclusion criteria and high-level design Complex Lymphatic • Key sites identified and KOLs engaged, study start anticipated 1Q 2021 Malformations • Orphan Drug
Slide 16: CERC-800s Monosaccharide therapy for CDGs
CERC-800s Monosaccharide therapy for Congenital Disorders of Glycosylation (CDGs)
Slide 17: Treatment for Congenital Disorders of Glycosylation
Treatment for Congenital Disorders of Glycosylation (CDGs) Monosaccharide therapy for PGM1-CDG, MPI-CDG and LADII High Patient • Ultra-rare Orphan diseases with an estimated 1,000 to 1,500 patients Unmet Need world-wide; no approved therapies to date Demonstrated • Data from the literature shows cli
Slide 18: Pharmaceutical Grade Treatments for CDGs
Pharmaceutical Grade Treatments for CDGs Opportunity to be the first FDA approved drugs for CDGs • Established therapeutic POC OH OH HO O • GMP manufacturing and FDA HO O HO O approval will ensure quality and consistency HO OH HO OH HO OH OH OH OH • Potential for reimbursement D-Galactose D-Mannose
Slide 19: Program Update as of June 2020
Program Update as of June 2020 All three programs on track • Natural history data is being provided by NIH funded Frontiers in Congenital Disorders of Glycosylation Consortium (FCDGC) Project in June 2020 • Pivotal trial will be run in collaboration with the Frontiers Project in 4Q 2020; CERC-801 tr
Slide 20: CERC-002 Anti-LIGHT mAb for COVID-19 ARDS
CERC-002 Anti-LIGHT monoclonal antibody in clinical studies for COVID-19 ARDS
Slide 21: The Impact of Cytokine Storm Induced COVID-19 ARDS
The Impact of Cytokine Storm Induced COVID-19 ARDS The outbreak of Coronavirus Disease 2019 (COVID-19) has created a global health crisis The viral infection triggers a hyperactive Approximately 1,500* people in immune response leading to cytokine the United States die each day storm and Acute Respi
Slide 22: Hyperactive Immune Response Leads to Cytokine Storm
Hyperactive Immune Response Leads to Cytokine Storm Cytokine storm induced ARDS is a major driver of poor COVID-19 outcomes Protective Immune Response Hyperactive Immune Response Immune cells arrive at the site of Excessive cytokines lead to over-recruitment of infection but do not overwhelm immune
Slide 23: LIGHT as a Key Driver of Inflammatory Response in ARDS
LIGHT is Potentially a Key Driver of the Inflammatory Response in Cytokine Storm in ARDS LIGHT Releases Inflammatory Cytokines and Activates Both T Cells and B Cells FasL LIGHT TL1A HVEM-mediated LTβR-mediated Signaling Pathways Signaling Pathways • Highly expressed in neutrophils and macrophages an
Slide 24: LIGHT Significantly Elevated in Hospitalized COVID-19 Patients
LIGHT is Significantly Elevated in Hospitalized COVID-19 Patients LIGHT Levels in Hospitalized LIGHT Levels in Both Non-Ventilated COVID-19 Patients and Ventilated Patients P < 0.0001 P < 0.0001 P < 0.0001 Free LIGHT levels are significantly elevated in serum of hospitalized patients with COVID-19,
Slide 25: Elevated LIGHT Levels Linked to Mortality in Ventilated Patients
Elevated LIGHT Levels Are Linked to Mortality in Ventilated Patients Elevated LIGHT Levels Associated with Key Implications Increased Mortality in Ventilated Patients • In ventilated patients, LIGHT levels were higher in those patients that eventually died than in those patients that recovered. This
Slide 26: Elevated LIGHT Most Strongly Linked to Mortality in Patients 60+
Elevated LIGHT is Most Strongly Linked to Mortality in Patients 60+ Association Between Elevated LIGHT and Key Implications Mortality Strongest in Patients Over 60 P = 0.021 • In patients over 60, LIGHT levels were significantly higher in those that eventually died than in those patients that recove
Slide 27: Cytokine Storm Drives ARDS Across Etiologies
Cytokine Storm Drives ARDS Across Etiologies Patients may progress rapidly and often require invasive mechanical ventilation Pre-ARDS Disease Course ARDS Progression Mild ARDS Moderate ARDS Severe ARDS COVID-19 infection COVID-19 ARDS All etiologies of ARDS have high unmet need, with COVID-19 infect
Slide 28: Potential Beyond COVID-19 ARDS
Potential Beyond COVID-19 ARDS CERC-002 may be applicable to the broader ARDS population CERC-002 COVID-19 ARDS High Morbidity and Mortality Broader ARDS Patients High ICU / Ventilation Cost Patients ($90 – $125 K per patient) • Viral / Bacterial Infections • Trauma Rationale for LIGHT as a Target •
Slide 29: COVID-19 and Broader ARDS Target Populations
COVID-19 and Broader ARDS Target Populations COVID-19 ARDS provides a potential path to treat a larger patient population in broader ARDS U.S. COVID-19 Related ARDS Patients U.S. Broader ARDS Patients Excluding COVID-19 416 427 392 404 380 367 340 354 19 ARDS Incidence 19 ARDS - COVID Diagnosed Broa
Slide 30: CERC-002: A Novel First-in-Class Anti-LIGHT mAb
CERC-002: A Novel First-in-Class Anti-LIGHT mAb The only known clinical stage anti-LIGHT antibody Free LIGHT Assay Developed in Collaboration with Myriad RBM Enables a biomarker / precision VH VH medicine development approach NH NH 2 2 CH Positive Toxicology Profile VL VL 8-week monkey toxicology st
Slide 31: FDA Clearance to Initiate Clinical Trial in COVID-19 ARDS
Clearance from the FDA to Initiate Clinical Trial in Cytokine Storm-Induced COVID-19 ARDS with CERC-002 Clear path for clinical development in COVID-19 ARDS, creating a path for development in generalized ARDS Planned Development Path for CERC-002 1H 2H 1H 2H 1H 2H 2020 2020 2021 2021 2022 2022 Q2 Q
Slide 32: CERC-002 Treatment of Cytokine Storm-Induced COVID-19 ARDS
CERC-002 Treatment of Cytokine Storm-Induced COVID-19 ARDS Primary Endpoint: Respiratory Failure and Mortality Over 28 Days Proof-of-Concept Trial Design Randomized, Double-blind, Placebo-controlled, Multi-Center, Proof-of-Concept Clinical Trial of CERC-002 in Adults with COVID-19 ARDS Inclusion Cri
Slide 33: Physician Perspectives on CERC-002
Physician Perspectives on CERC-002 Front line physicians1 (n=14) with significant experience in COVID-19 and ARDS broadly viewed CERC-002’s mechanism as novel with potential to fill an urgent unmet need Urgent Medical “There are very few options for ARDS patients so I would Need definitely want to u
Slide 34: Highlights Through 2022
Highlights Through 2022 Multiple catalysts and 3 potential PRV awards from first-in-class medicines for diseases with no approved treatment options CERC-801 CERC-801 TOP LINE DATA Potential NDA APPROVAL CERC-803 CERC-803 TOP LINE DATA Potential NDA APPROVAL Initial Data from CERC-802 CERC-802 CDG FI
Slide 35: www.cerecor.com
NASDAQ:CERC www.cerecor.com
Slide 36: Select Board and Management Team Members
Select Board and Management Team Members Michael F. Cola Chief Executive Officer Garry A. Neil, MD Chief Scientific Officer Sol J. Barer, PhD Chairman 36|
Slide 37: References: CERC-002
References: CERC-002 • LIGHT: Lymphotoxin-like, exhibits Inducible expression, and competes with HSV Glycoprotein D for HVEM, a receptor expressed by T lymphocytes; encoded by TNFSF14 (Tumor Necrosis Factor Superfamily 14). • Bellani G, Laffey JG, Pham T, et al. Epidemiology, Patterns of Care, and M
Slide 38: References: CERC-006, CERC-007, CERC-800s
References: CERC-006, CERC-007, CERC-800s • Adams DM, Trenor CC 3rd, Hammill AM, et al. Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies. Pediatrics. 2016;137(2):e20153257. • Bhagwat SV, Gokhale PC, Crew AP, et al. Preclinical characterization of OSI-027, a potent
Slide 39: Appendix
Appendix
Slide 40: Key Financial Information
Key Financial Information (CERECOR
Slide 41: Financial & Investor Information
Financial & Investor Information Key financial highlights NASDAQ:CERC The following data is as of April 30, 2020 • Outstanding shares – 59.6M • Average Daily Volume – 136K • Cash – $13M 41|
Slide 42: CERC-002 Anti-LIGHT mAb for Pediatric Crohn's Disease
CERC-002 Anti-LIGHT monoclonal antibody in clinical studies for Pediatric Crohn’s Disease
Slide 43: Elevated Free LIGHT Levels Detected in Pediatric Crohn's Disease Patients
Elevated Free LIGHT Levels Detected in Pediatric Crohn’s Disease Patients Using Cerecor’s Proprietary Free LIGHT Assay Plasma LIGHT levels are significantly elevated in Crohn’s Disease patients (n=238) over healthy individuals (n=9) and are displayed as • Cerecor has developed a proprietary and box-
Slide 44: CERC-006 Dual mTORC 1/2 Inhibitor for Complex Lymphatic Malformations
CERC-006 Phase II-ready, Dual mTORC 1/2 small molecule inhibitor for Complex Lymphatic Malformations
Slide 45: Off-label Use of mTOR Inhibitor Sirolimus in LM
Off-label Use of mTOR Inhibitor Sirolimus in LM Open-label clinical studies support efficacy, however use is limited by tolerability issues and lack of FDA approval • Phase II trial enrolled patients with complicated vascular anomalies1 – Study enrolled patients with different subtypes of LM not con
Slide 46: CERC-006 Treatment of Patients with Complex Lymphatic Malformations
CERC-006 Treatment of Patients with Complex Lymphatic Malformations Dual mTOR inhibitor to reduce ALK and PI3K expression and optimize dose for safety Proposed Phase 1b Study A Multicenter, Phase 1b Study of CERC-006 in Patients with Complex Lymphatic Malformations Inclusion Criteria Patients with C
Slide 47: CERC-007 Anti-IL-18 mAb for Multiple Myeloma
CERC-007 Phase 2-ready, anti-IL-18 monoclonal antibody for Multiple Myeloma
Slide 48: Current and Future Myeloma Market
Current and Future Myeloma Market Enduring unmet need is expected, given the lack of curative therapies; the U.S. MM market is estimated to increase to ~$15 B over the next five years, though Without a Curative Therapy, MM Physicians Multiple myeloma is a Large Seek Improvements in MM Regimens and G
Slide 49: CERC-007 Treatment of Patients with Resistant and Refractory Multiple Myeloma
CERC-007 Treatment of Patients with Resistant and Refractory Multiple Myeloma Initiating Trial in Multiple Myeloma as a Single Agent with Plans for Combination Proposed Dose Escalation and Expansion Trial Design A Multicenter, Open-Label, Dose-Escalation Phase 1b Study of CERC-007 in Subjects with R
Slide 50: CERC-007 Anti-IL-18 mAb for Adult Onset Still's Disease
CERC-007 Phase 2-ready, anti-IL-18 monoclonal antibody for Adult Onset Still’s Disease
Slide 51: CERC-007 Treatment of Patients with Adult Onset Still Disease
CERC-007 Treatment of Patients with Adult Onset Still Disease Possible Best-in-class and first-in-class anti-IL-18 mAb Proposed Proof-of-Concept Trial Design A Multicenter, Phase 1b Study of CERC-007 in Subjects with Active Adult Onset Stills Disease Inclusion Criteria Patients with active AOSD as m
Slide 52: CERC-800s Monosaccharide therapies for CDGs
CERC-800s Monosaccharide therapies for Congenital Disorders of Glycosylation (CDGs)
Slide 53: CERC-801 Treatment of PGM1-CDG
CERC-801 Treatment of PGM1-CDG Study to be Performed in Partnership with NIH Consortium Proposed Pivotal Trial Design A Phase 3, Open Label, Efficacy, Safety, and Tolerability Study of CERC-801 in patients with Phosphoglucomutase-1 deficiency related congenital disorders of glycosylation (PGM-1) Inc
Slide 54: CERC-802 Treatment of MPI-CDG
CERC-802 Treatment of MPI-CDG High Risk of Thrombotic Events Permits Maintenance of Key Serum Biomarkers Proposed Pivotal Trial Design A Phase 3, Open Label, Efficacy, Safety, and Tolerability Study of CERC-802 in patients with Mannose phosphate isomerase deficiency related congenital disorders of g
Slide 55: CERC-803 Treatment of LAD-II
CERC-803 Treatment of LAD-II Fucose Glycosylation is Key to Function of White Blood Cells Proposed Pivotal Trial Design A Phase 3, Open Label, Efficacy, Safety, and Tolerability Study of CERC-803 in patients with Lymphocyte Adhesion Disorder Type II (LAD-II) Inclusion Criteria Patients with known MP
Slide 56: www.cerecor.com
NASDAQ:CERC www.cerecor.com