Avalo Therapeutics, Inc. · NASDAQ:AVTX

Avalo Therapeutics — Cerecor — Investor Presentation (July 2021)

51 slides from Avalo Therapeutics's investor presentation, Jul 26, 2021

  1. Slide 1: Establishing a leading rare and orphan disease-focused biopharmaceutical company

    July | 2021 Establishing a leading, rare and orphan disease-focused biopharmaceutical company to deliver impactful new medicines to patients Patient-Inspired Science Exhibit 99.1

  2. Slide 2: Forward-Looking Statements

    2 | 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

  3. Slide 3: Pipeline Highlights

    3 | *Orphan Drug Designation, Rare Pediatric Disease Designation; Eligibility for Priority Review Voucher Upon Approval. mAb, monocolonal antibody. Pipeline Highlights  Cerecor has a rich pipeline of six novel, first-in-class assets in eight clinical development programs across immunology, oncology

  4. Slide 4: Clinical-Stage Pipeline

    4 | Clinical-Stage Pipeline Core Research & Development Areas Therapeutic Area Program Mechanism of Action Lead Indication Development Stage Anticipated Milestone Preclin Phase 1 Phase 2 Pivotal Trial Immunology Inflammation CERC-002‡ Anti-LIGHT mAb COVID-19 ARDS Received FTD* CERC-002 Anti-LIGHT mA

  5. Slide 5: CERC-002

    Anti-LIGHT monoclonal antibody in clinical studies for Crohn’s disease and COVID-19 ARDS CERC-002

  6. Slide 6: CERC-002: A Novel First-in-Class Anti-LIGHT mAb

    6 | CERC-002: A Novel First-in-Class Anti-LIGHT (TNFSF14) mAb In-licensed From Kyowa Kirin Co., Worldwide Exclusive Rights* for All Indications (2021) (2021) *Kyowa Kirin has an option to retain the rights in Japan.  Novel, first-in-class fully human subcutaneous (SQ) monoclonal antibody (mAb)  On

  7. Slide 7: CERC-002: Crohn's Disease

    * * oe FS 8 . * ee CERC-002: Crohn’s Disease, (ceREcoR

  8. Slide 8: Executive Summary: CERC-002 Demonstrates Potential Proof-of-Concept

    8 | Executive Summary: CERC-002 Demonstrates Potential Proof-of-Concept in Initial Low-Dose Cohort  Open-label proof-of-concept study in patients with moderate to severe Crohn’s disease who previously failed 3 or more lines of biologic therapies, including anti-TNFα*  Clinically meaningful mucosal

  9. Slide 9: Inflammatory Bowel Disease Overview

    9 | 1. Centers for Disease Control and Prevention. Inflammatory bowel disease. https://www.cdc.gov/ibd/features/IBD-more-chronic-diseases.html. Accessed July 17, 2021. 2. Atreya R et al. Front Med (Lausanne). 2020;7:517. 3. Knowles SR et al. Inflamm Bowel Dis. 2018;24(4):742-751. 4. Byron C et al. J

  10. Slide 10: Substantial Opportunity Remains in the Treatment of IBD

    10 | 1. Atreya R et al. Front Med (Lausanne). 2020;7:517. 2. Clearview Healthcare Partners. Crohn’s disease and ulcerative colitis. Initial disease landscape overview. May 2021. Substantial Opportunity Remains in the Treatment of IBD  Majority of patients are designated moderate/severe and treated

  11. Slide 11: LIGHT Is a Key Driver of Inflammation

    11 | LIGHT Is a Key Driver of Inflammation LIGHT Inflammation HVEM-Mediated Signaling Pathways LTβR-Mediated Signaling Pathways  LIGHT (TNFSF14) is a pro-inflammatory cytokine and a co-stimulator of T cells and Th1 cytokines, including interferon (IFN)-γ1  LIGHT is expressed on activated T cells,

  12. Slide 12: Multiple Lines of Evidence Support Importance of LIGHT in IBD

    12 | 1. Data on file, Cerecor, Inc. 2. Moraes L et al. Inflamm Bowel Dis. 2020;26(6):874-884. 3. Cohavy O et al. J Immunol. 2005;174(2):646-653. 4. Wang J et al. J Immunol. 2005;174(12):8173-8182. 5. Shaikh RB et al. J Immunol. 2001;167(11):6330-6337. 6. Jungbeck M et al. Immunology. 2009;128(3):451

  13. Slide 13: Elevated LIGHT Levels Detected in Pediatric Crohn's Disease

    13 | Data displayed as box-and-whiskers graph. *Determined by Mann-Whitney U test. Plasma samples from CHOP Biobank; controls are matched for age and gender. Source: Cardinale C et al. Manuscript in preparation. Elevated LIGHT Levels Detected in Pediatric Crohn’s Disease  Approximately 83% of pedia

  14. Slide 14: CERC-002 Crohn's Disease Proof-of-Concept Trial Design

    14 | Screening (Visit 1) Open-Label Treatment Period (Visits 2-10) (8 weeks) Safety Follow-Up (Visit 11) CERC-002, SQ injections every 14 days (N=8, 4 subjects in each cohort; Cohort 1: 1mg/kg; Cohort 2: 3mg/kg) Telephone Visit Eligibility & Washout Week: -10 0 1 2 3 4 5 6-8 127 8 Visit #: 1 2 3 4 5

  15. Slide 15: Phase 1b Initial Data (Cohort 1): Patient-Level Data

    15 | CERC-002 Crohn’s Disease Proof-of-Concept: Phase 1b Initial Data (Cohort 1) Patient-Level Data Disease severity according to SES-CD score1: Remission: 0-2 Mild: 3-6 Moderate: 7-15 Severe: >15 Subject # Age (yrs) Prior Therapy / # of Prior Lines SES-CD LIGHT (pg/mL) Response Baseline 8 Weeks Bas

  16. Slide 16: Preliminary Efficacy Results (Patient-Level Data)

    16 | Preliminary Efficacy Results (Patient-Level Data) SES-CD ScoresPlasma LIGHT Levels  Clinically meaningful mucosal healing, determined by colonoscopy, in 3 of 4 subjects (SES-CD) SE S- CD S co re 25 Baseline (Day 0) Week 8 20 10 5 0 15 Pl as m a LI G HT L ev el s ( pg /m L) 500 Baseline (Day 0)

  17. Slide 17: Independent Preliminary Safety Data Results

    17 | 1. Perlin DS et al. CERC-002, a human anti-LIGHT mAb reduces respiratory failure and death in hospitalized COVID-19 ARDS patients (https://medrxiv.org/cgi/content/short/2021.04.03.21254748v1). Independent Preliminary Safety Data Results – CERC-002, SQ Injection (1mg/kg)  No serious adverse eve

  18. Slide 18: CERC-002: COVID-19 ARDS

    ie = gt ee - a4 CERC- 002: COVID-AS ARDS (ceREcoR

  19. Slide 19: Phase 2 Trial Met Primary Endpoint in COVID-19 ARDS

    19 | Phase 2 Clinical Trial Met Primary Endpoint in Patients Hospitalized with COVID-19 ARDS Executive Summary: Final Data Analysis  CERC-002 significantly reduced respiratory failure and mortality in Phase 2 clinical trial in patients hospitalized with COVID-19 acute respiratory distress syndrome

  20. Slide 20: LIGHT Is a Central Driver of COVID-19-Related Cytokine Storm

    20 | 1. Perlin DS et al. mSphere. 2020;5(4):e00699-20. 2. Arunachalam PS et al. Science. 2020;369(6508):1210-1220. LIGHT Is a Central Driver of COVID-19–Related Cytokine Storm Clinical Trial Initiated After Compelling Biomarker Study Completed June 2020  In patients ≥60 years, LIGHT levels were sig

  21. Slide 21: Cytokine Storm Drives ARDS Across Etiologies

    21 | Cytokine Storm Drives ARDS Across Etiologies Reducing LIGHT levels may limit the proportion of patients requiring invasive mechanical ventilation, which drives high cost of treatment and low quality of life in ARDS ARDS ProgressionPre-ARDS Disease Course All ARDS patients are candidates for int

  22. Slide 22: CERC-002 Treatment of Cytokine Storm-Induced COVID-19 ARDS

    22 | PaO2, partial pressure of oxygen, FiO2, fraction of inspired oxygen. CERC-002 Treatment of Cytokine Storm-Induced COVID-19 ARDS 1:1 Randomization CERC-002 (16 mg/kg [max. 1200 mg]) on Day 1 by SQ injection + Standard of Care at the site Placebo-matched SQ injection + Standard of Care at the sit

  23. Slide 23: Patient Disposition

    23 | ITT, intent-to-treat; SoC, standard of care. Source: Data on file, Cerecor, Inc. Patient Disposition Screening: Patients With COVID-19–Associated Pneumonia and Mild to Moderate ARDS 1:1 Randomization to SoC + CERC-002 or Placebo (n=83) Patients Administered CERC-002 or Placebo (n=82) Patients W

  24. Slide 24: Patient Demographics

    24 | *Calculated from patients dosed (n=40 for CERC-002, n=42 for placebo). Source: Data on file, Cerecor, Inc.; Publication under review. Patient Demographics Characteristic CERC-002 (n=41) Placebo (n=42) Age (yrs), mean (SD) 59.2 (14.5) 58.1 (14.2) Age Group <60 yrs, n (%) ≥60 yrs, n (%) 20 (48.8)

  25. Slide 25: A Single Dose of CERC-002 Reduced LIGHT Levels Dramatically and Rapidly

    25 | Source: Perlin DS et al. CERC-002, a human anti-LIGHT mAb reduces respiratory failure and death in hospitalized COVID-19 ARDS patients (https://medrxiv.org/cgi/content/short/2021.04.03.21254748v1). A Single Dose of CERC-002 Reduced LIGHT Levels Dramatically and Rapidly Rapid and significant red

  26. Slide 26: CERC-002 Significantly Reduced Respiratory Failure and Mortality

    26 | *Prespecified analysis. Source: Data on file, Cerecor, Inc. CERC-002 Significantly Reduced Respiratory Failure and Mortality Efficacy was highest in patients aged ≥60* years (n=34, P=0.042), the population most vulnerable to severe complications and death with COVID-19 infection Pa tie nt s( %

  27. Slide 27: A Single Dose of CERC-002 Reduced Mortality by ~50%

    27 | Source: Data on file, Cerecor, Inc. A Single Dose of CERC-002 Reduced Mortality by ~50% CERC-002 Placebo 28-day Mortality 7.7% 14.3% 60-day Mortality 10.8% 22.5%  A trend in ~50% reduction in mortality was observed at both the 28-day and 60-day timepoints  Efficacy observed is on top of corti

  28. Slide 28: Safety and Tolerability

    28 | Source: Data on file, Cerecor, Inc. Safety and Tolerability  CERC-002 was well-tolerated at a single dose of 16 mg/kg  No serious adverse events (SAEs) attributable to CERC-002  Majority of adverse events (AEs) judged to be mild or moderate  No evidence of increased infections or AEs relate

  29. Slide 29: COVID-19 ARDS Provides Potential Path to Broader ARDS Population

    29 | CAGR, compound annual growth rate. Sources: Eworuke E et al. J Crit Care. 2018;47:192-197; Bellani G et al. JAMA. 2016;315(8):788-800; Rubenfeld GD et al. N Engl J Med. 2005;353(16):1685-1693; UpToDate; Physician Interviews; ClearView Analysis. COVID-19 ARDS Provides Potential Path to Treat a L

  30. Slide 30: Next Steps CERC-002 Clinical Program

    30 | *TNFα, tumor necrosis factor alpha. Next Steps CERC-002 Clinical Program  Crohn’s Disease ‒ Further evaluation of cohort data, including tissue LIGHT levels ‒ Cohort 2 (3 mg/kg dose) fully enrolled; complete data anticipated in 2H21  Ulcerative Colitis ‒ Expand clinical study to patients with

  31. Slide 31: CERC-007

    Phase 1b anti-IL-18 monoclonal antibody for Multiple Myeloma and Still’s Disease (AOSD and sJIA) CERC-007

  32. Slide 32: Data From Phase 1 Study Demonstrated Favorable PK and Safety Profile

    32 | *COPD, chronic obstructive pulmonary disease; IV, intravenous; SAD, single ascending dose. Source: Data on file, AstraZeneca. Data From Phase 1 Study Demonstrated Favorable PK and Safety Profile First-in-Class Anti-IL-18 High-Affinity Monoclonal Antibody  In-licensed from Medimmune/AstraZeneca

  33. Slide 33: Multiple Myeloma: Second Most Common Blood Cancer Globally

    33 | 1. NCI SEER. Cancer stat facts: myeloma. https://seer.cancer.gov/statfacts/html/mulmy.html. Accessed July 22, 2021. 2. Palumbo A et al. N Engl J Med. 2011;364(11):1046-1060. 3. ClearView Analysis 2020. Multiple Myeloma: Second Most Common Blood Cancer Globally Disease OverviewMultiple Myeloma (

  34. Slide 34: Strong Potential in Multiple Myeloma

    34 | *HR, hazard ratio; **IL-18, interleukin 18. Source: Nakamura K et al. Cancer Cell. 2018;33(4):634-648.e5 Strong Potential in Multiple Myeloma  Patients with high IL-18 have significantly worse median survival (42 mo vs >84 mo; P=0.0026; HR*, 1.84)  Reducing IL-18** levels prolongs survival in

  35. Slide 35: CERC-007 Treatment of Resistant and Refractory Multiple Myeloma

    35 | *IMID, immunomodulatory drug; **RP2D, recommended phase 2 dose; ***SPEP, serum protein electrophoresis; ****IL-18, interleukin 18. CERC-007 Treatment of Resistant and Refractory Multiple Myeloma Initiating Trial in Multiple Myeloma as a Single Agent With Plans for Combination CERC-007: Expansio

  36. Slide 36: Adult-Onset Still's Disease (AOSD) Overview

    36 | *IL-18, interleukin 18; **CRP, C-reactive protein; ***NSAID, nonsteroidal anti-inflammatory drug. 1. ClearView Healthcare Partners Analysis, May 2017. 2. Gerfaud-Valentin M et al. Autoimmun Rev. 2014;13(7):708-722. 3. Kudela H et al. BMC Rheumatol. 2019;3:4. Adult-Onset Still’s Disease (AOSD) O

  37. Slide 37: Systemic Juvenile Idiopathic Arthritis (sJIA) Overview

    37 | *IL, interleukin; **NSAID, nonsteroidal anti-inflammatory drug; ***DMARD, disease-modifying anti-rheumatic drug; ****CRP, C-reactive protein. 1. ClearView Healthcare Partners Analysis, May 2017. 2. Kudela H et al. BMC Rheumatol. 2019;3:4. Systemic Juvenile Idiopathic Arthritis (sJIA) Overview 

  38. Slide 38: Proof-of-Concept Clinical Data

    38 | Subcutaneous administration of 80 mg or 160 mg 3 times weekly. Response defined as an improvement of joint count (both Swollen Joint Count [SJC] and Tender Joint Count [TJC] according to a 44-joint assessment) by ≥20% from baseline values, and a 70% decrease of CRP levels compared with baseline

  39. Slide 39: CERC-007 Treatment of Adult-Onset Still's Disease

    39 | CERC-007 7 mg/kg (max 500 mg) q4wks (n=6) *IL-18, interleukin-18; **CRP, C Reactive Protein; ***DAS, Disease Activity Score; ****ESR erythrocyte sedimentation rate CERC-007 Treatment of Adult-Onset Still’s Disease Potential Best-in-Class and First-in-Class Anti-IL-18* mAb 12 weeks AOSD initial

  40. Slide 40: CERC-006

    Phase 2-ready, dual mTORC 1/2 small molecule inhibitor for Complex Lymphatic Malformations CERC-006

  41. Slide 41: Potential for Improved Efficacy and Tolerability

    41 | *MAD, multiple ascending dose; **MTD, maximum tolerated dose; ***mTOR, mammalian target of rapamycin; ****ATP, adenosine triphosphate; *****IC, half maximal inhibitory concentration. 1. Mateo J et al. Br J Cancer. 2016;114(8):889-896. 2. Bhagwat SV et al. Mol Cancer Ther. 2011; 10(8):1394-1406.

  42. Slide 42: Complex Lymphatic Malformations

    42 | Source: Figure adapted from Brouillard P et al. J Clin Invest. 2014;124(3):898-904. Complex Lymphatic Malformations: Family of Potentially Life-Threatening Congenital Diseases  Neoplastic lesions caused by mutations in PI3K/AKT/mTOR pathway  Leads to local proliferation of lymphatic endotheli

  43. Slide 43: Off-Label Use of mTOR Inhibitor Sirolimus in Lymphatic Malformations

    43 | Source: Adams DM et al. Pediatrics. 2016;137(2):e20153257. Open-Label Clinical Studies Support Efficacy; Use Is Limited by Tolerability Issues and Lack of FDA Approval Off-Label Use of mTOR Inhibitor Sirolimus in Lymphatic Malformations  Phase 2 trial enrolled patients with complicated vascula

  44. Slide 44: CERC-006 Treatment of Complex Lymphatic Malformations

    44 | *Pi3K, phosphatidylinositol 3-kinase; *AKT, protein kinase B. CERC-006 Treatment of Complex Lymphatic Malformations Dual mTOR Inhibitor to Modulate PI3K* and AKT** Activity CERC-006 two dose groups: 0.5 mg and 1mg twice daily 4-week treatment Initial data anticipated 3Q 2021 Multicenter, Phase

  45. Slide 45: CERC-800s

    Monosaccharide therapy for Congenital Disorders of Glycosylation (CDGs) CERC-800s

  46. Slide 46: Congenital Disorders of Glycosylation and Substrate Supplementation Therapy

    46 | 1. Wong et al. Genet Med. 2017;19(11):1226-1235. 2. Harms et al. Acta Paediatr. 2002;91(10):1065-1072. 3. Marquardt et al. Blood. 1991;94(12):3976-3985. Impaired Glycoprotein Production and Function Can Simply Be Restored With Substrate Supplementation Therapy Congenital Disorders of Glycosylat

  47. Slide 47: Pharmaceutical Grade Treatments for CDGs

    47 | D-MannoseD-Galactose L-Fucose OHO OH OH HO OH OHO OH OH HO OH CERC-801 CERC-802 CERC-803 Accelerated pathway ✓ ✓ ✓ FDA ODD** ✓ ✓ ✓ Priority Review Voucher* ✓ ✓ ✓ Pivotal data anticipated 1Q 2022 2H 2021 2H 2021 *All three CERC-800 compounds granted Rare Pediatric Disease Designation prior to Se

  48. Slide 48: Key Financial Information

    . a Key Financial Information (cérecor a

  49. Slide 49: Financial & Investor Information

    49 | Financial & Investor Information Key Financial Highlights The following data is as of March 31, 2021  Outstanding common shares – 89.1M  Fully diluted shares – 114.2M  Average daily trading volume – 1M  Cash – $38.3M NASDAQ: CERC

  50. Slide 50: Select Management Team Members

    50 | Select Management Team Members Proven Track Record in Drug Development and Commercialization Michael Cola Chief Executive Officer • Former President of Specialty Pharmaceuticals, Shire plc • Senior management, AstraMerck and AstraZeneca plc Garry Neil, MD Chief Scientific Officer • Former Corpo

  51. Slide 51: www.cerecor.com

    NASDAQ: CERC www.cerecor.com

Cerecor — Investor Presentation (July 2021) on 6ix