Rockwell Medical, Inc. · NASDAQ:RMTI
Rockwell Medical — Investor Day (September 2020)
64 slides from Rockwell Medical's investor day presentation, Sep 24, 2020
Slide 1: New Indications Opportunities for Ferric Pyrophosphate Citrate (FPC)
NEW INDICATIONS OPPORTUNITIES FOR FERRIC PYROPHOSPHATE CITRATE (FPC) Rockwell Medical, Inc. September 24, 2020 www.RockwellMed.com
Slide 2: Our Company Vision
OUR COMPANY VISION Transforming Iron Deficiency and Anemia Management Our vision is to establish a new standard of care for patients suffering with iron deficiency and iron deficiency anemia. Iron deficiency anemia afflicts a subset of the two billion people worldwide who are nutritionally iron defi
Slide 3: Call Participants
CALL PARTICIPANTS Rockwell Medical Russell Ellison, M.D., M.Sc. President and Chief Executive Officer, Rockwell Medical, Inc. Marc Hoffman, M.D. Chief Medical Officer, Rockwell Medical, Inc. Timothy Chole Vice President of Marketing,Tim Chole Rockwell Medical, Inc. Vice President of Marketing Key Op
Slide 4: Forward Looking Statements
FORWARD LOOKING STATEMENTS Certain statements in this presentation may constitute “forward-looking statements” within the meaning of the federal securities laws, including, but not limited to, potential new indication opportunities for Ferric Pyrophosphate Citrate (“FPC”), the timing for the commerc
Slide 5: Rockwell Medical Is Repositioned for Growth
ROCKWELL MEDICAL IS REPOSITIONED FOR GROWTH Transforming Iron Deficiency and Anemia Management 1 2 3 4 5 6 Commercial-stage Solid base business Triferic®* - one of the Ferric pyrophosphate Global FPC market Experienced Team company with an • #2 supplier of dialysis most innovative citrate (FPC) plat
Slide 6: FPC New Indications Opportunity Assessment
FPC NEW INDICATIONS OPPORTUNITY ASSESSMENT Objective and Process to Date OBJECTIVE Expand the use of our ferric pyrophosphate citrate (or FPC) molecule for patients through the development of FPC to treat medical conditions with unmet clinical needs outside of the dialysis setting. PROCESS • Ideatio
Slide 7: Agenda
AGENDA Introduction & Overview of FPC Russell Ellison, M.D., M.Sc. CEO, Rockwell Medical, Inc. FPC New Indication Opportunity: Iron Deficiency Anemia in Home Infusion Patients • Introduction Russell Ellison, M.D., M.Sc. CEO, Rockwell Medical, Inc. • EXPERT PERSPECTIVE: Home Infusion Therapy Connie S
Slide 8: Ferric Pyrophosphate Citrate (FPC): Overview
FERRIC PYROPHOSPHATE CITRATE (FPC): OVERVIEW First and only FDA-approved treatment indicated for the replacement of iron as maintenance therapy in adult patients with hemodialysis-dependent chronic kidney disease (HDD-CKD) (Triferic®) • Donates iron directly and completely to transferrin, bypassing
Slide 9: FPC Is a True 2nd Generation Parenteral Iron
FPC IS A TRUE 2ND GENERATION PARENTERAL IRON • Water-soluble iron salt administered via slow infusion • No complex carbohydrate shell; tightly bound to pyrophosphate and citrate1 • Donates 100% bioavailable iron to transferrin immediately once in the blood • Utilizes the body’s natural iron transpor
Slide 10: FPC Donates Bioavailable Iron Directly and Completely to Transferrin
FPC DONATES BIOAVAILABLE IRON DIRECTLY AND COMPLETELY TO TRANSFERRIN Negligible NTBI Even at the Highest Dose of FPC Administered to Healthy Volunteers 10 mg FPC Non-Transferrin Bound Iron 4 h 260 10 mg FPC Transferrin Bound Iron 4 h 10 mg FPC Total Serum Iron (BL Corr) 4 h 210 TBI nearly equals tot
Slide 11: Features and Benefits of FPC vs. IV Iron Oxide Nanoparticles
FEATURES AND BENEFITS OF FPC VS. IV IRON OXIDE NANOPARTICLES (IONP) Macromolecular IV iron is sequestered within the reticuloendothelial system FPC • FPC bypasses the reticuloendothelial system Muscle, and donates iron directly to transferrin RES other In the inflamed patient, increased hepcidin blo
Slide 12: Excellent Clinical Safety Profile of FPC (Triferic) in Hemodialysis Patients
EXCELLENT CLINICAL SAFETY PROFILE OF FPC (TRIFERIC®) DEMONSTRATED IN HEMODIALYSIS PATIENTS Clinical Development Program in the U.S.1,2,3 • No deaths during or attributed to Triferic (>100,000 Doses) • No anaphylaxis • No iron overload (serum ferritin did not increase) • Adverse event rate similar to
Slide 13: FPC Currently Marketed in Renal, Development Stage for New Indications
FPC CURRENTLY MARKETED IN RENAL, AT DEVELOPMENT STAGE FOR NEW INDICATIONS Triferic for ESKD Patients… FPC for Additional Indications… Three approved formulations Phase II ready Indicated for iron replacement and maintenance of hemoglobin in adult patients undergoing hemodialysis Priority 1 For treat
Slide 14: FPC New Indication Opportunity: Iron Deficiency Anemia in Home Infusion
FPC NEW INDICATION OPPORTUNITY: IRON DEFICIENCY ANEMIA IN HOME INFUSION Top Priority 14
Slide 15: Connie Sullivan, President and CEO, NHIA
CONNIE SULLIVAN, B.S. PHARM. President and CEO, National Home Infusion Association (NHIA) ❖ Home infusion industry leader and patient advocate ❖ Over 25 years experience in home infusion clinical practice, operations, and executive leadership ❖ Expert consultant - United States Pharmacopeia panel de
Slide 16: Home Infusion Therapy
HOME INFUSION THERAPY What is home infusion therapy? Specialized services that allow patients to receive intravenous medications at home. Providers are specialized, closed-door pharmacies with expertise in sterile compounding and clinical management of IV therapies. Therapy is supported by multi-dis
Slide 17: Home Infusion Therapy: Benefits
HOME INFUSION THERAPY What are the benefits of home infusion? Allows patients with diseases and disorders requiring multiple on-going infusions of medications to receive them in the comfort of their own home. Benefits include1,2: • Increased quality of life (QOL) • Shorter hospital/skilled nursing f
Slide 18: Commercial Payers and CMS Provide Reimbursement for Home Infusion Therapies
COMMERCIAL PAYERS AND CMS PROVIDE REIMBURSEMENT FOR HOME INFUSION THERAPIES The majority of home infusion therapy patients are covered by commercial insurance. Payers are increasingly motivated to reimburse home infusions to reduce in-office visits, hospitalizations, and save costs. Commercial Payer
Slide 19: The Home and Specialty Infusion Market Is Growing
THE HOME AND SPECIALTY INFUSION MARKET IS GROWING The home and specialty infusion marketplace is experiencing explosive Unique growth and provides a favorable reimbursement opportunity for Therapy Patients suitable drugs.1 (annually) Anti-infectives 1,441,520 Comparison of Home Infusion Parenteral N
Slide 20: Home Infusion Is Expected to Continue a Growth Trend
HOME INFUSION IS EXPECTED TO CONTINUE A GROWTH TREND IN THE FUTURE • Continued industry growth is expected, driven by… • High rates of patient demand, and satisfaction with services • Site of care optimization programs driven by commercial payers • Legislation to expand coverage for Medicare benefic
Slide 21: Iron Deficiency Anemia Is a Recognized Issue for Total Parenteral Nutrition Patients
IRON DEFICIENCY ANEMIA IS A RECOGNIZED ISSUE FOR TOTAL PARENTERAL NUTRITION PATIENTS1 IDA is a common complication occurring in 40-55% of patients on long-term PN. A majority of patients suffer from conditions/diseases that put them at a high risk for developing IDA. • Patients with IDA can exhibit
Slide 22: Management of Iron Deficiency Anemia in Home Infusion Is a Broken Process
MANAGEMENT OF IRON DEFICIENCY ANEMIA IN HOME INFUSION THERAPY PATIENTS IS A BROKEN PROCESS Many patients on home infusion therapy suffer from diseases with which iron deficiency and anemia are commonly Anemia is untreated until associated, but current treatment patterns are inadequate. symptoms beco
Slide 23: Proactive Management of IDA in Hemodialysis – A Model for Home Infusion?
PROACTIVE MANAGEMENT OF IDA IN HEMODIALYSIS – A MODEL APPROACH FOR HOME INFUSION? Established Guidelines for therapeutic targets Protocols (iron measures, hemoglobin / hematocrit targets, etc.) Iron deficiency is proactively Regular lab testing of iron measures and H&H Monitoring adjustment of iron
Slide 24: In Summary
IN SUMMARY ❖ Home infusion therapy is a rapidly growing area of medicine with over 3.2 million patients served every year ❖ Growth trend is likely to continue driven by cost savings vs. office-based or hospital care, an improving reimbursement landscape, and emerging standards resulting from the glo
Slide 25: Significant Market Opportunity for FPC in Home Parenteral Nutrition Population
A SIGNIFICANT MARKET OPPORTUNITY IDENTIFIED FOR FPC IN THE HOME PARENTERAL NUTRITION PATIENT POPULATION Many patient groups requiring home infusion therapies suffer from diseases that are associated with an incidence of iron deficiency and anemia. For home parenteral nutrition patients specifically,
Slide 26: Additional Home Infusion Patients May Be Candidates for FPC
ADDITIONAL HOME INFUSION THERAPY PATIENTS WITH IRON DEFICIENCY ANEMIA MAY BE CANDIDATES FOR FPC The following home infusion therapies are provided to patients for the treatment of diseases that may be associated with a risk of iron deficiency anemia. Applicability of FPC will depend on length of the
Slide 27: Iron Management for Home Infusion with FPC vs. Traditional IV Iron
IRON MANAGEMENT FOR HOME INFUSION WITH FPC VS. TRADITIONAL IV IRON THERAPY APPROACH IRON REPLETION THERAPY IRON MAINTENANCE THERAPY Traditional IV Iron* FPC Traditional IV Iron* FPC (proposed) (proposed) 200 – 500mg per dose 15 – 30 min infusion 2 – 5 doses (office visits) Physician Office Indicatio
Slide 28: A Market Access Pathway Is in Place for FPC as Home Infusion Therapy
A MARKET ACCESS PATHWAY IS IN PLACE FOR FPC AS A HOME INFUSION THERAPY • FPC for home infusion will likely be eligible for favorable reimbursement • Drugs reimbursed via buy and bill process for home infusion providers • Coverage available under the Medicare Part B DME POS* benefit (narrow pathway)
Slide 29: FPC Is Uniquely Suited for Use as a Home Infusion Therapy
FERRIC PYROPHOSPHATE CITRATE IS UNIQUELY SUITED FOR USE AS A HOME INFUSION THERAPY Home infusion clinicians are hesitant to recommend IV FPC has been demonstrated to have a safety profile similar iron supplementation due to the potential for severe to placebo in prospective randomized clinical trial
Slide 30: Clinical Development Strategy: Unique NDA
FPC HOME INFUSION OPPORTUNITY: CLINICAL DEVELOPMENT STRATEGY: UNIQUE NDA • Leverage existing NDA • Preclinical Tox √ • Preclinical Pharmacology √ • Drug Substance √ • Long Term Safety √ • New contents needed for NDA • Drug Product Section New IP • New Package (new dosage not a rate limiting step, e.
Slide 31: Planned Regulatory Approach
FPC HOME INFUSION OPPORTUNITY: PLANNED REGULATORY APPROACH • Type C meeting with FDA to discuss our clinical development plan (EOY 2020 - early Q1 2021) • Establish efficacy and safety in two well-defined patient populations to minimize variability of clinical study . . . • Uncomplicate clinical ass
Slide 32: Building on Precedent
FPC HOME INFUSION OPPORTUNITY: BUILDING ON PRECEDENT Precedents for this strategy are evident in the labels of other iron products, e.g. Feraheme® (ferumoxytol) 1, Injectafer® (ferric carboxymaltose) 2 and Monoferric® (ferric derisomaltose)3 All are indicated for the treatment of iron deficiency ane
Slide 33: Outline of Phase II and Phase III Clinical Development
FPC HOME INFUSION OPPORTUNITY: OUTLINE OF PHASE II AND PHASE III CLINICAL DEVELOPMENT 1) Utilize a Phase II observational study to validate feasibility assumptions and fully characterize treatment setting • Determine iron deficits, by indication • Establish practice treatment patterns and dosage and
Slide 34: Potential Phase III Home Infusion Study Design
POTENTIAL PHASE III HOME INFUSION STUDY DESIGN* Methods and Cohort Treatment phase** Long Term Follow-up (LTF) 9 month Rx + pts randomized to FPC Maintenance dose 7- 9 month LTF group receiving 10mg FPC/week 2mg/hr. 12°/12° Randomized 2:1 Placebo/SoC placebo-controlled, double-blind CRUISE-like endp
Slide 35: FPC Home Infusion Opportunity: In Summary
FPC HOME INFUSION OPPORTUNITY: IN SUMMARY ❖ Home infusion therapy is an area of medicine experiencing explosive growth – a trend that will likely continue in this decade ❖ Iron deficiency anemia (IDA) is a common co-morbidity in many sub-groups of patients receiving home infusion therapy, particular
Slide 36: FPC New Indication Concept: Acute Heart Failure
FPC NEW INDICATION CONCEPT: ACUTE HEART FAILURE Under Consideration 36
Slide 37: Heart Failure
HEART FAILURE (OR HF) Inability of the Heart to Meet the Body’s Metabolic Demands Approximately 4.9 million cases in the United States today • Over 400,000 new cases per year • The most common cause of hospitalization in people over 65 years • Increasing numbers of HF patients due to survival from h
Slide 38: Heart Failure (continued)
HEART FAILURE (OR HF) Inability of the Heart to Meet the Body’s Metabolic Demands Mechanism • HFrEF: HF with reduced ejection fraction (50% of cases) • HFpEF: HF with preserved ejection fraction (50% of cases) Etiology • Ischemic (approx. 2/3 of HFrEF) • Non-ischemic Onset • Acute decompensation (AD
Slide 39: Heart Failure Is Increasing and Iron Deficiency Is a Common Co-morbidity
HEART FAILURE IS INCREASING IN THE U.S. AND IRON DEFICIENCY IS A COMMON CO-MORBIDITY Heart failure in the U.S. is a large and growing patient population. Over a million patients are hospitalized each year with acute decompensated heart failure. Iron deficiency a common co-morbidity in all forms of H
Slide 40: Acute Heart Failure Opportunity: Market Access & Reimbursement
FPC ACUTE HEART FAILURE OPPORTUNITY: MARKET ACCESS & REIMBURSEMENT The majority of heart failure patients admitted to hospital are insured by Medicare / Medicaid through a well- established universal approach usually resulting in a bundled reimbursement structure Payer Source of Inpatient Heart Fail
Slide 41: Significant Market Opportunity for FPC in Iron Deficiency in Hospitalized AHF
A SIGNIFICANT MARKET OPPORTUNITY MAY EXIST FOR FPC AS A TREATMENT OF IRON DEFICIENCY IN HOSPITALIZED AHF The AHF opportunity for FPC is associated with a large patient population with a high incidence of iron deficiency.1 FPC has theoretical acute clinical advantages (<30 days) vs. traditional IV ir
Slide 42: Inder Anand, M.D., FRCP, D. Phil.
INDER ANAND, M.D., FRCP, D. PHIL. ❖ Emeritus Professor of Medicine, University of Minnesota Medical School ❖ Former Director, Heart Failure Program, VA Medical Center Minneapolis MN ❖ Former Senior Advisor, NHLBI, NIH ❖ Founder member of the Heart Failure Society of America (HFSA) ❖ One of the Found
Slide 43: Heart Failure, Anemia and Iron Deficiency
HEART FAILURE, ANEMIA AND IRON DEFICIENCY • Anemia and Iron deficiency (ID) are common comorbidities in heart failure (HF) & often coexist. Together or independently they are associated with worse symptoms, increased hospitalizations and mortality. • Treating anemia in HF with erythropoiesis-stimula
Slide 44: Absolute or Relative Iron Deficiency Is an Independent Risk Factor in Heart Failure
Absolute or Relative Iron Deficiency is an Independent Risk Factor in Heart Failure 546 patients with stable HF; LVEF 26 ± 7%. Overall, absolute or relative ID was present in 37% patients; 57% in those with anemia, 32% in those without anemia. ID was associated with death or Heart Tx independent of
Slide 45: In HF Iron Deficiency May Be a More Important Prognostic Marker Than Anemia
IN HF IRON DEFICIENCY MAY BE A MORE IMPORTANT PROGNOSTIC MARKER THAN ANEMIA Iron deficiency but not anemia remained an independent predictor of mortality (HR] 1.42, 95% CI 1.14-1.77, p = .002) International pooled cohort, 1,506 HF patients with and without ID and anemia 45 Klip et al. Am Heart J 201
Slide 46: Mechanisms of the Association of ID with Adverse Outcomes
MECHANISMS OF THE ASSOCIATION OF ID WITH ADVERSE OUTCOMES - MULTIFACTORIAL • Iron is an essential component of all energy producing mitochondrial enzymes. • ID reduces cardiac and skeletal muscle iron content causing both structural and several functional mitochondrial abnormalities, including decre
Slide 47: Does Oral Iron Replacement Therapy Help?
DOES ORAL IRON REPLACEMENT THERAPY HELP? • Oral iron supplementation is standard therapy for ID; readily available, inexpensive & effectively raises serum iron levels non-HF patients. • Because HF is an inflammatory state, elevated levels of cytokines and the peptide hepcidin prevent iron absorption
Slide 48: IRONOUT-HF: Oral Iron Not Effective
IRONOUT-HF: ORAL IRON NOT EFFECTIVE • Phase 2 RCT, 225 patients with HFrEF, NYHA class II to IV HF, median LVEF, 25% • Anemic or non-anemic patients Hb; 9-15 g/dL (men) or 9-13.5 g/dL (women) with ID • Randomized: oral iron polysaccharide 150 mg BD/placebo. 48 Lewis, GD et al.JAMA. 2017;317(19):1958
Slide 49: Studies with IV Iron in Heart Failure
STUDIES WITH IV IRON IN HEART FAILURE Efficacy of iron sucrose or FCM investigated in 5 RCT in Outpatients • ↑ Exercise Capacity (Peak VO₂ ; 6MWT) • Improved QoL scores • Improved NYHA class • ↓ NT - proBNP level • Because of small size, outcomes such as death and hospitalization for HF could not be
Slide 50: Meta-Analysis of RCT Comparing IV Ferric Carboxymaltose and Placebo in HFrEF
META-ANALYSIS OF 4 RCT COMPARING IV FERRIC CARBOXYMALTOSE AND PLACEBO IN HFREF Individual patient data from 4 RCTs comparing FCM with placebo in 839 Individualpatients patient with HFrEF data from and 4 ID, RCTs 504 comparingrandomized FCM to FCMwith placebo and 335 in to 839 placebo. patients with
Slide 51: How Does IV Iron Improve Outcomes in Patients with HF and Iron Deficiency?
HOW DOES IV IRON IMPROVE OUTCOMES IN PATIENTS WITH HF AND IRON DEFICIENCY INDEPENDENT OF ANEMIA? • Does IV iron restore iron stores in the body and particularly in the heart and skeletal muscles and how rapid is this effect? • Are these effects associated with improvement in the cardiac and skeletal
Slide 52: IV Iron Restores Skeletal & Myocardial Iron Stores
IV IRON RESTORES SKELETAL & MYOCARDIAL IRON STORES T2* and T1 Mapping Cardiac Magnetic Resonance Imaging Randomized chronic stable patients with HFrEF and ID to placebo (n=26) or IV FCM (n=27 ) T2* and T1 cardiac magnetic resonance imaging to assess myocardial iron at at baseline, 7 and 30 days afte
Slide 53: IV Iron Rapidly Restores Skeletal and Myocardial Iron Stores
IV IRON RAPIDLY RESTORES SKELETAL AND MYOCARDIAL IRON STORES T2* AND T1 MAPPING CARDIAC MAGNETIC RESONANCE IMAGING THE MYOCARDIAL-IRON TRIAL 53 Nunez et al. JAHA. 2020;9: e014254 ▪Nunez et al. JAHA. 2020;9: e014254.
Slide 54: Iron Repletion Is Associated with Improvement in Muscle Mitochondrial Energetics
IRON REPLETION IS ASSOCIATED WITH IMPROVEMENT IN MUSCLE MITOCHONDRIAL ENERGETICS • FERRIC-HF II tested the hypothesis that IV iron in HF enhances skeletal muscle energetics as reflected by shorter PCr recovery half-time (PCr t1/2) on 31P MRS. • 40 patients (50% anemic) HFrEF, NYHF class ≥II and ID w
Slide 55: How Do We Explain These Findings and the Next Steps?
HOW DO WE EXPLAIN THESE FINDINGS AND THE NEXT STEPS? • The IV iron studies show that although IV iron rapidly entered the myocardial cells and improved the skeletal muscle mitochondrial function, the functional changes were modest with no appreciable improvement in cardiac function. • Taken together
Slide 56: Clinical Hypothesis
CLINICAL HYPOTHESIS • Increasing data suggest that IV iron in ambulatory patients with heart failure with reduced ejection fraction (HFrEF) improves symptoms, exercise capacity, but long-term effects on hospitalizations, mortality and safety are unknown • Re-uptake and response of cardiac tissues li
Slide 57: Rationale for IV FPC vs. IV Iron Oxide Nanoparticles in Hospitalized AHF Patient
RATIONALE FOR IV FPC VS. IV IRON OXIDE NANOPARTICLES IN THE HOSPITALIZED AHF PATIENT IONPs are delivered in very large, bolus doses ranging from 100-750mg/dose • These large doses are known to increase inflammation and increase hepcidin which paradoxically prevents the release of iron to the tissue
Slide 58: FPC for Acute Heart Failure: Clinical Development Strategy
FPC FOR ACUTE HEART FAILURE: CLINICAL DEVELOPMENT STRATEGY • Target indication: treatment of Iron Deficiency (with or without anemia) in adult in-patients with acute heart failure (HFrEF) • Over the past 20 years, there has been considerable progress in the treatment of chronic heart failure • Heart
Slide 59: Potential Proof of Concept Study Design
POTENTIAL PROOF OF CONCEPT STUDY DESIGN Baseline MRI: Follow-up MRI: iron stores/energetics iron stores/energetics ventricular function ventricular function Strain Strain Echocardiography Strain Echocardiography Echocardiography Biomarkers Biomarkers Biomarkers BiomarkersDay 1 Day 2 Day 3 Control: S
Slide 60: What Questions Would the PoC Study Answer?
FPC FOR ACUTE HEART FAILURE: What questions would the PoC Study answer? Feel & Function → Clinical Benefit • Improved LV Function demonstrated by ECHO as measured by global longitudinal strain (GLS) • Improvement in energetics measured from Phosphocreatine/Creatine kinase (CK) aka Creatine phosphoki
Slide 61: FPC Acute Heart Failure Opportunity: In Summary
FPC ACUTE HEART FAILURE OPPORTUNITY: IN SUMMARY ❖ Over a million patients are hospitalized each year with acute decompensated heart failure. ❖ Iron deficiency a common co-morbidity in all forms of HF (50-70%) representing a significant unmet need. ❖ A significant body of evidence exists to support t
Slide 62: Development Plan Near Term Milestones
DEVELOPMENT PLAN NEAR TERM MILESTONES * Opportunity for multiple data readouts and Type C Investor Day Request Type C Meeting milestone reports Sep 24 with FDA ACUTE HEART FAILURE Type C Type C Meeting End of Phase II Request with FDA HOME INFUSION Meeting with FDA HOME INFUSION 2020 1 H 2 H 2021 1
Slide 63: Q&A
Rockwell Medical Russell Ellison, M.D., M.Sc. President and Chief Executive Officer, Rockwell Medical, Inc. Marc Hoffman, M.D. Chief Medical Officer, Rockwell Medical, Inc. Timothy Chole Q&A Vice President of Marketing,Tim Chole Rockwell Medical, Inc. Vice President of Marketing Key Opinion Leaders
Slide 64: Important Safety Information
IMPORTANT SAFETY INFORMATION Warnings and Precautions ◦ Serious hypersensitivity reactions, including anaphylactic-type reactions, some of which have been life-threatening and fatal, have been reported in patients receiving parenteral iron products. Patients may present with shock, clinically signif