Vericel Corporation · NASDAQ:VCEL
Vericel — Aastrom Biosciences — Investor Presentation (April 2013)
25 slides from Vericel's investor presentation, Apr 23, 2013
Slide 1: Aastrom Biosciences: The Future of Cell Therapy
Aastrom Biosciences The Future of Cell Therapy April 2013
Slide 2: Safe Harbor
Safe Harbor This presentation contains forward-looking statements, including, without limitation, statements concerning product-development objectives, clinical trial strategies, clinical trial timing and expected results, market data, potential market opportunities, market development plans, antici
Slide 3: Aastrom Biosciences Highlights
Leading Autologous Cell Therapy Platform Highly Differentiated Product Positive Clinical Data in Areas of Large Unmet Need Developing patient-specific multicellular therapies Automated cGMP manufacturing system Expandable and highly scalable Ixmyelocel-T is a unique multicellular therapy Key effecto
Slide 4: Ixmyelocel-T is a unique multicellular therapy
Ixmyelocel-T is a unique multicellular therapy; key effector cells are M2-like macrophages and MSCs 4
Slide 5: Ixmyelocel-T has multiple biological activities that promote repair & regeneration
Ixmyelocel-T has multiple biological activities that promote repair & regeneration of ischemic tissue 5
Slide 6: Therapeutic platform provides efficient cell collection, production and delivery
Aastrom’s therapeutic platform provides for efficient cell collection, production and delivery 6
Slide 7: GMP manufacturing platform is highly automated and expandable, with low COGS
Ixmyelocel-T GMP manufacturing platform is highly automated and expandable, with low COGS Automated, Fully-Closed GMP Manufacturing System Single-use disposable bioreactor cassette Application key Incubator Processor System manager Highly Scalable Modular Expansion Enables COGS < 10% at commercial s
Slide 8: Final product is convenient and easy to use
Ixmyelocel-T final product is convenient and easy to use Manufactured to finished product specifications Streamlined logistics Shipped via Fedex overnight in a qualified container Ready to use when received No freezing or thawing No refrigeration No reconstitution 72-hour shelf life Easy to administ
Slide 9: Multicellular platform offers potential for multiple therapeutic applications
Aastrom’s unique multicellular platform offers potential for multiple therapeutic applications 9 Bone Cardiac Liver Pancreas Kidney Joints Bone Marrow Neural Vascular Muscle Lung Autoimmune Disease Atherosclerosis Ixmyelocel-T Potential Therapeutic Applications
Slide 10: Current focus is ixmyelocel-T for advanced heart failure due to ischemic DCM
Current focus is on developing ixmyelocel-T to treat advanced heart failure due to ischemic DCM 10
Slide 11: Heart failure represents a significant unmet medical need
ADD AHA CHF Forecast Heart failure represents a significant unmet medical need and growing public health problem 11 CRT $25,000-$50,000 initial LVAD2 $150,000 initial, $225,000 1st year Transplantation3 $200,000 initial, $1M 1st year Source: Heidenreich et al. Circulation 2013;123:933-944 Sources: 1
Slide 12: Ischemic DCM is a leading cause of heart failure and heart transplantation
Ischemic DCM is a leading cause of heart failure and heart transplantation 12 Description Disease of the heart muscle characterized by a weakened and enlarged chambers, reduced wall thickness, and inability to sufficiently pump blood 3rd most common cause of heart failure Leading cause of heart tran
Slide 13: A majority of refractory advanced heart failure patients have DCM
Heart Failure 5.5 Million Patients Class III/IV NYHA 1.8 Million Patients Refractory to Rx Therapy 265,000 Patients Sources: Heidenreich et al. Circulation 2013;123:933-944; Health Research International; America Heart Association Key DCM Assumptions: Prevalence Rate - 36/100,000 adults, Incidence R
Slide 14: Refractory ischemic DCM market is a significant commercial opportunity
14 The refractory ischemic DCM market represents a significant commercial opportunity for ixmyelocel-T Key Assumptions: 48 months to peak sales, price = $40,000, 3% annual price increase, no “off label” use Year Sources: Health Research International, America Heart Association Key DCM Assumptions: R
Slide 15: Ixmyelocel-T reduced tissue damage in a murine model of heart failure
Ixmyelocel-T significantly and reproducibly reduced tissue damage in a murine model of heart failure Naïve Mouse Control Vehicle ixmyelocel-T Naïve Mouse Control Vehicle ixmyelocel-T N=6 N=11 N=5 N=9 N=7 N=6 15 Study conducted at Medigenix, LLC; data presented at the 18th Annual International Societ
Slide 16: Trend towards reducing apoptosis in the infarct border zone
Ixmyelocel-T demonstrated a trend towards reducing apoptosis in the infarct border zone Mean +/- SEM 16 Study conducted at Medigenix, LLC; data presented at the 18th Annual International Society for Cellular Therapy Meeting, June 2012. Murine Non-Acute Infarct Model N=2 N=3 N=3 Control Vehicle Ixmye
Slide 17: Treatment increased plasma nitrates
Ixmyelocel-T treatment increased plasma nitrates, which may contribute to observed protective effects *** *** P < 0.001 vs. Vehicle 17 n=3 n=5 n=7 n=9 Study conducted at Medigenix, LLC; data presented at the 18th Annual International Society for Cellular Therapy Meeting, June 2012. Murine Non-Acute
Slide 18: Phase 2a DCM clinical objectives and study parameters
Phase 2a DCM clinical objectives focused on safety, optimizing delivery and patient selection Phase 2a Clinical Objectives Demonstrate ixmyelocel-T safety in DCM patients Evaluate delivery approaches Gain clinical insight into patient selection Phase 2a Study Parameters: Two 12-month randomized open
Slide 19: Phase 2a safety results: well-tolerated in DCM patients
Phase 2a safety results demonstrated that ixmyelocel-T was well-tolerated in DCM patients Adverse Events Per Patient Days 0-5 Days 6+ Ixmyelocel-T Control Ixmyelocel-T Control IMPACT-DCM 6.71* N/A# 8.21 7.87 CATHETER-DCM 0.93* N/A# 5.93 7.83 *Only ixmyelocel-T subjects underwent mini-thoracotomy, th
Slide 20: Lower percentage of MACE events in ischemic DCM groups
20 Subjects treated with ixmyelocel-T in the ischemic DCM groups had a lower percentage of MACE events *Combined IMPACT-DCM and CATHETER-DCM data in the ischemic DCM groups.
Slide 21: Consistent positive trends in efficacy parameters in ischemic DCM groups
21 Consistent positive trends were observed in several efficacy parameters in the ischemic DCM groups* *Combined IMPACT-DCM and CATHETER-DCM data in the ischemic DCM groups
Slide 22: Phase 2b ixCELL-DCM Study Design
Phase 2b ixCELL-DCM Study Design Objectives To evaluate the efficacy, safety and tolerability of ixmyelocel-T compared to placebo in patients with heart failure due to ischemic DCM Patients Males and females, age 30-85 Diagnosis of ischemic DCM according to WHO criteria Not a candidate for reasonabl
Slide 23: ixCELL-DCM Phase 2b study execution is highly achievable
ixCELL-DCM Phase 2b study execution is highly achievable Well-defined patient population at a well-defined point in disease progression Advanced heart failure patients refractory to medical therapy Strong coordination between heart failure specialists and interventional cardiologists Existing relati
Slide 24: Ischemic DCM indication provides a path to commercialization
The ischemic DCM indication provides a compelling path to commercialization for ixmyelocel-T 24 2014 2015 2013 Study Results FDA end of Phase 2 meeting Complete Patient Enrollment First Patient Enrolled Next Steps: Complete patient enrollment Hold FDA end of Phase 2 meeting Complete Phase 3 study de
Slide 25: Aastrom Biosciences Highlights
Leading Autologous Cell Therapy Platform Highly Differentiated Product Positive Clinical Data in Areas of Large Unmet Need Developing patient-specific multicellular therapies Automated cGMP manufacturing system Expandable and highly scalable Ixmyelocel-T is a unique multicellular therapy Key effecto
Aastrom Biosciences — Investor Presentation (April 2013) on 6ix