Kamada Ltd. · NASDAQ:KMDA
Kamada — Investor Presentation (March 2017)
48 slides from Kamada's investor presentation, March 2017
Slide 1: Kamada Investor Presentation
KAMADA INVESTOR PRESENTATION NASDAQ & TASE: KMDA March 2017 |
Slide 2: Forward Looking Statement
Forward Looking Statement This presentation is not intended to provide This presentation includes certain non-GAAP financial investment or medical advice. It should be noted that information, which is not intended to be considered in some products under development described herein isolation or as a
Slide 3: Kamada - Company Profile (KMDA)
Kamada - Company Profile (KMDA) Growing Biopharma company generating revenue and profits from 10 proprietary products Commercial Leader in Alpha-1 Antitrypsin (“AAT”) products globally, both commercialized and in development, and specific immunoglobulins Stage Biotech Glassia®, for AAT deficie
Slide 4: Kamada Investment Highlights
Kamada Investment Highlights • Globally Positioned Biopharmaceutical Company focused on Orphan Diseases and Plasma-Derived Protein Therapeutics • $100M of revenues expected in 2017 • Flagship Product Glassia® Approved for Alpha-1 Antitrypsin (AAT) Deficiency Disease o Unique and Differentiated Produ
Slide 5: Diversified Product Portfolio with Extended Global Reach
Diversified Product Portfolio with Extended Global Reach Diverse Portfolio of Predominantly Plasma-Derived Protein Therapeutics Global Presence with Exposure to Emerging Markets Respiratory Glassia® Alpha-1 Antitrypsin (human) Proprietary Products KamRAB™ Anti-rabies immunoglobulin (human) Segment I
Slide 6: AAT Deficiency: Relatively Common, Potentially Lethal, Often Undiagnosed
AAT Deficiency: Relatively Common, Potentially Lethal, Often Undiagnosed Genetic/Hereditary condition causing decreased levels of AAT in blood and tissues Affects at least 100,000 people in the US and a similar number in Europe Z Predisposes to lung and liver diseases AAT deficiency-associated lun
Slide 7: Glassia is a Differentiated Product
Glassia® is a Differentiated Product Key Product Advantages AATD (IV) Product Sales w/o Milestone Revenues (in $M) ● Glassia® is the first and only liquid, ready-to- use, IV plasma-derived AAT product No reconstitution required Reduces treatment time Reduces risk of contamination and infection
Slide 8: Growth of Glassia Driven by Strategic Partnership with Baxalta
Growth of Glassia® Driven by Strategic Partnership with Baxalta (part of Shire) • Commencement: Sales to Baxalta started in September 2010 • Agreements: Distribution, technology license, and supply of fraction IV • Product: AAT IV (Glassia®), including all future AAT IV indications in the territorie
Slide 9: Significant Opportunity to Expand the AATD Market
Significant Opportunity to Expand the AATD Market ● Patients suffering from AAT Deficiency AATD Prevalence: ~200,000 (“AATD”) remain under-identified and under- Yet Fewer than 5% of Potential Patients in treated the U.S. and Europe are Treated ● Only ~6% of cases treated in the U.S. and ~2% in EU ~
Slide 10: AAT Serves as An Exciting Potential Therapy for Multiple Indications
AAT Serves as An Exciting Potential Therapy for Multiple Indications AAT is a safe plasma-derived protein with known & newly discovered therapeutic roles Anti- Immune- Tissue Antimicrobial Inflammatory modulatory Protective 10 |
Slide 11: High Value Pipeline Focused on Orphan Indications
High Value Pipeline Focused on Orphan Indications Product Indication Phase I Phase II Phase III Market Partners Intravenous AAT Deficiency FDA Approved (2010) U.S.: AAT POC Study Completed D1-AAT (IV) Type 1 Diabetes* Double Blind, Ph II U.S.: In Process Phase I/II In Process G1-AAT (IV) GVHD* U.S.:
Slide 12: Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency (AATD)
Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency (AATD) 12 |
Slide 13: Most effective mode of treatment for reaching primary site of injury
Most effective mode of treatment for reaching primary site of injury Benefits of Inhaled AAT 5x ELF levels 2-5 fold than IV Improved Quality Of Life (QOL) ELF = Epithelial Lining Fluid 13 |
Slide 14: Inhalation Enables Delivery of AAT 5x Higher Than Intravenous
Inhalation Enables Delivery of AAT 5x Higher Than Intravenous Inhaled AAT ~ 50% of the dosage reaches airway tree and alveoli Alveoli Emphysema • DLCO • CT densitometry Airway Obstruction Intravenous AAT ~ 2% of the dosage • FEV1/SVC reaches alveoli and airway tree • FEV1 14 | Inhaled AAT Phase II/I
Slide 15: Inhaled AAT for AATD: Completed Pivotal Phase II/III Trials in Europe and Phase II in the U.S.
Inhaled AAT for AATD: Completed Pivotal Phase II/III Trials in Europe and Phase II in the U.S. EU US Phase II / III trial - Completed Phase II - Completed Description ● Over 160 AATD subjects, majority are treatment- ● Randomized; Sample size of 36 subjects naïve ● Double blind, placebo controlled,
Slide 16: Inhaled AAT Phase II/III Trial: Summary of Results
Inhaled AAT Phase II/III Trial: Summary of Results Results demonstrate: 1. Primary and secondary endpoints did not demonstrate statistical significant difference. 2. Efficacy in lung function (statistically significant) 3. Change in the nature of exacerbations - Reduction in number of Type 1- exacer
Slide 17: Inhaled AAT Phase II/III Trial Results: Spirometry Measures (MMRM)
Inhaled AAT Phase II/III Trial Results*: Spirometry Measures (MMRM**) Least Squares Means (SEM) Least Squares Means (Changes at Week 50 from (SEM) method: Lung Baseline) P-Value** Overall treatment effect P-Value** (Changes at (Overall Function Week 50) Effect) AAT Placebo AAT Placebo (N= 84) (N= 81
Slide 18: Inhaled AAT slowed FEV1 deterioration better than former IV trials
Inhaled AAT slowed FEV1 deterioration better than former IV trials IV studies Inhaled Seesholm 1997 AATD reg 1998 Dirksen 1999 Wenecker 2001 Chapman 2005 KAMADA INH 0 -10 -20 -30 -40 -50 -60 -70 -80 13.4 ml/y diff 50 ml/y diff -90 Chapman KR, Stockley RA, COPD:6:177–184 AAT Placebo 18 | Inhaled AAT
Slide 19: Inhaled AAT Phase II Topline Result: ELF AAT Antigenic Level & Inhibitory Capacity Increased
Inhaled AAT Phase II Topline Result: ELF AAT Antigenic Level & Inhibitory Capacity Increased Significantly * • Inhaled AAT is the most effective mode of treatment for reaching the primary sites of potential lung injury, and restoring AAT inhibitory capacity • Trial demonstrated strong safety profile
Slide 20: Inhaled AAT: Moving Forward
Inhaled AAT: Moving Forward EMA: EU Front FDA: U.S. Front ● MAA submitted (centralized ● Approach FDA with results to obtain procedure) March 2016 guidance on the clinical/ regulatory pathway for licensing the IH AAT by ● Day 120 comments received, Kamada in the U.S. response expected early 2017 ● E
Slide 21: Immune-Modulatory Indications
Immune-Modulatory Indications 21 |
Slide 22: Mechanistic Evidence - Alpha1-Antitrypsin, a Therapeutic Approach
Mechanistic Evidence - Alpha1-Antitrypsin, a Therapeutic Approach AAT Immunomodulation AAT promotes a tolerance-inducing profile Anti-inflammatory Protect Cells from Injury Protects cells from IL-1/IFN- Induces the production of induced injury and reduces the anti-inflammatory mediators levels of
Slide 23: AAT to Treat Graft Versus Host Disease
AAT to Treat Graft Versus Host Disease 23 |
Slide 24: Graft Versus Host Disease (GVHD): A Major Complication in Stem Cell Transplantation
Graft Versus Host Disease (GVHD): A Major Complication in Stem Cell Transplantation Deadly side effects 20% 50% 70% of deaths are caused are non responsive mortality in patients by GVHD to steroids with grade III/IV GVHD complications Searching for an effective treatment • Standard of care prophylax
Slide 25: Proof-of-Concept Study with AAT (IV) for Graft-Versus-Host Disease
Proof-of-Concept Study with AAT (IV) for Graft-Versus-Host Disease Phase I/II study open label of Dose: 4 dose groups - 15 day regimen. Doses given 24 patients with steroid-resistant on days: 1, 3, 5, 7, 9, 11, 13 and 15 GVHD following allogeneic bone- Primary End Points: % of patients at each dosin
Slide 26: AAT May Exert a Protective Effect on the Bowel Mucosa in Gut GVHD
AAT May Exert a Protective Effect on the Bowel Mucosa in Gut GVHD Study results have indicated that AAT may exert healing of the bowel mucosa in gut GVHD slowing/stopping the disease progression and re-modulation of the immune attack Administration of AAT as salvage Preliminary results are therapy f
Slide 27: Images from Phase I/II Clinical Study Interim Report
Images from Phase I/II Clinical Study Interim Report Before After 8 doses of AAT Duodenits Suspect severe Moderate mucosal denudement upper and lower GVHD and edema noted throughout the duodenum 27 |
Slide 28: Initiating Phase II/III Study with AAT (IV) for GVHD in Collaboration with Shire
Initiating Phase II/III Study with AAT (IV) for GVhD in Collaboration with Shire Pivotal phase II/III study, Study objective: to evaluate the safety and efficacy of randomized, two-part, multi- AAT (IV) as an add-on biopharma-co-therapy to standard-of-care steroid treatment as the first-line center,
Slide 29: Advancing the GvHD Opportunity
Advancing the GvHD Opportunity US EU (in collaboration with Shire): (independent): Initiated Pivotal phase II/III study, Positive Scientific Advice from EMA randomized, two-part, multi- for the proposed development center, placebo controlled, with program. Received guidance for the AAT (IV) for the
Slide 30: AAT to Treat Lung Transplant Rejection
AAT to Treat Lung Transplant Rejection 30 |
Slide 31: Lung Transplant Rejection - Attractive Opportunity To Deliver A Differentiated Therapy
Lung Transplant Rejection - Attractive Opportunity To Deliver A Differentiated Therapy Lungs have the highest rate of rejection 100 among transplanted solid organs Adult Lung Transplants Recipients Survival Rate* o ~33% will experience acute rejection within 80 the first year o ~50% will develop chr
Slide 32: On Going Phase II Study With AAT IV For Prevention Of Lung Transplant Rejection
On Going Phase II Study With AAT IV For Prevention Of Lung Transplant Rejection Phase II: Prospective, open label, Study objective: To assess the safety of AAT IV standard of care (SOC) administration and the effect on rate and severity of controlled, randomized, parallel acute and chronic lung reje
Slide 33: AAT to Treat Newly Diagnosed Type-1 Diabetes
AAT to Treat Newly Diagnosed Type-1 Diabetes 33 |
Slide 34: AAT (IV) is a Promising Potential Treatment for Newly Diagnosed Type-1 Diabetes Patients
AAT (IV) is a Promising Potential Treatment for Newly Diagnosed Type ‐1 Diabetes Patients Market Opportunity Drug Impact Benefits Type-1 Diabetes Studies have shown Preservation of beta cells occurs when the immune that AAT protects correlates with reduced risk system attacks and destroys beta cell
Slide 35: Newly Diagnosed Type-1 Diabetes Clinical Trial Ongoing
Newly Diagnosed Type‐1 Diabetes Clinical Trial Ongoing Phase II: Double-Blind, Study objective: To evaluate the efficacy and safety of human, Alpha-1 Antitrypsin (AAT) in the treatment of Randomized, Placebo- new onset Type 1 Diabetes Controlled, Multicenter Study Design: Two doses, placebo controll
Slide 36: KamRAB: Human Rabies Immune Globulin
KamRAB: Human Rabies Immune Globulin Kamada’s human rabies immune globulin is a post-exposure prophylaxis (PEP) for rabies U.S. Opportunity: Strategic agreement with Kedrion S.p.A for the clinical development and marketing of KamRAB in U.S. ● U.S. pivotal Phase II/III clinical trial met primary endp
Slide 37: Financials
KAMADA High Quality Pharmaceuticals 37
Slide 38: Compelling Investment Driven by Multiple Pillars of Growth
Compelling Investment Driven by Multiple Pillars of Growth Additional Unencumbered The Kamada New Pipeline Products Pillars Geographies Inhaled AAT G1-AAT (IV): GVHD Existing Anchor Potential to sell phase I/II in process Products for AATD in existing and new ($0.5-1B)* Europe & US products into
Slide 39: Strong Financial Profile with Revenue Growth and Expanding Profitability
Strong Financial Profile with Revenue Growth and Expanding Profitability ● Stable, profit generating revenue stream from marketed products ● Revenue Guidance: $100 M in 2017 ● Strategic partnership model results in efficient operating expenses o Baxalta/Shire purchase obligations provide predictable
Slide 40: Sustained Revenues and Gross Profits are Funding R&D
Sustained Revenues and Gross Profits are Funding R&D $M FY2014 FY2015 FY2016 % change Proprietary 44 43 56 30% Products Distribution 27 27 21 (22%) Total Revenues 71 70 77 10% Gross Profit 15 16 22 37% R&D (16) (17) (16) S&M and G&A (10) (11) (11) Net Loss (13) (11) (7) (36%) Adjusted (5) (6) (1) EB
Slide 41: Future Milestones and Value Creation
Future Milestones and Value Creation Milestone Date FDA guidance for Inhaled AAT for AATD regulatory path 1H-2017 Response to EMA comments for Inhaled AAT for AATD 1H-2017 Approved IND for registration trial of Inhaled AAT for AATD in US 2H-2017 2017 Approved CTA for registration GvHD trial in EU 2H
Slide 42: Kamada Investment Highlights
Kamada Investment Highlights Globally Positioned Biopharmaceutical Company focused on Orphan Diseases and Plasma-Derived Protein Therapeutics • $100 M of revenues expected by 2017 • Flagship Product Glassia® Approved for Alpha-1 Antitrypsin (AAT) Deficiency Disorder o Unique and Differentiated Produ
Slide 43: Thank You
THANK YOU www.kamada.com 43 |
Slide 44: Next Generation AAT Inhaled
Next Generation AAT Inhaled • Inhaled directly to the Lungs • Clinical trial in Europe completed • Registration file submitted to EMA • Expecting mid 2017 approval “ The study results demonstrated primarily that the overall treatment effect on lung functions, is of significant clinical value. This s
Slide 45: Fewer symptoms in first EX - AAT vs. Placebo
Fewer symptoms in first EX - AAT vs. Placebo Less Type I (3 symptoms) and more type II (2 symptoms) AAT Placebo Type/Category P Value N=85 N=83 Type I 16 (18.8%) 26 (31.3%) 0.0614 Type II 23 (27.1%) 12 (14.5%) 0.0444 Type III 34 (40.0%) 33 (39.8%) 0.9746 None 12 (14.1%) 12 (14.5%) 0.9498 45 | Invest
Slide 46: Improved daily score
Improved daily score AAT patients tended to have better AAT patients tended to have better Dyspnea score Well-Being score Dyspnea 4 Week Moving Average Graphs Well Being 4 Week Moving Average Graphs AAT Placebo 46 | Inhaled AAT Phase II/III
Slide 47: In the Words of the Key Opinion Leaders
In the Words of the Key Opinion Leaders “ EU Phase 2/3: “ The study results demonstrated primarily that the overall treatment effect on lung functions, is of significant clinical value. This study is the first study ever that is indicative of inhaled AAT’s ability to potentially reduce lung inflamma
Slide 48: Clinical Development for Newly Diagnosed Type-1 Diabetes: New Exciting Prospects
Clinical Development for Newly Diagnosed Type‐1 Diabetes: New Exciting Prospects Phase I/II Open Label Study to evaluate the safety, tolerability and efficacy of AAT on beta cell preservation and glycemic control on newly diagnosed T1D pediatric patients (N=24) End-of-study slope analysis of C-pepti