Fortress Biotech, Inc. · NASDAQ:FBIO
Fortress Biotech — Investor Presentation (February 2017)
15 slides from Fortress Biotech's investor presentation, Feb 28, 2017
Slide 1: Corporate Overview
Corporate Overview March , 2017 CAELUM BIOSCIENCES
Slide 2: Forward Looking Statements
Forward Looking Statements 2 Statements in this presentation that are not descriptions of historical facts are forward - looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995 . We have attempted to identify forward - looking sta
Slide 3: About Us
About Us 3 Caelum Biosciences, a Fortress Biotech Company, is a clinical stage biotechnology company developing treatments for rare and life - threatening conditions . Caelum’s lead asset, CAEL - 101 , is a novel antibody in Phase 1 b clinical trials that is being developed for patients with AL Amyl
Slide 4: Opportunity Overview in AL Amyloidosis
CAEL - 101 Opportunity Overview in AL Amyloidosis AL Amyloidosis effects major organs leading to a high mortality CAEL - 101 well - tolerated and sustained organ response in Phase 1 Preparing for Phase 2 Broad protection through regulatory exclusivity and IP Pioneering antibody developed to specific
Slide 5: Amyloid Fibrils Formation in Tissue
Amyloid Fibrils Formation in Tissue 5 Misfolded protein AL Amyloidosis patients Misfolded proteins collect together Collection of misfolded protein creates Amyloid fibrils Plasma Cells Plasma Cells Produce Proteins Normal Protein cell Bone marrow produce plasma cell Bone Amyloid Fibrils deposit in t
Slide 6: AL Amyloidosis Impact on Organs & Tissue
AL Amyloidosis Impact on Organs & Tissue 60% - 80% of patients - leads to end - stage kidney disease 65% - 75% of patients - leads to heart failure and high mortality 20% - 45% of patients - peripheral neuropathy leading to pain, numbness, and weakness 6 5% - 35 % - Other o rgans
Slide 7: Survival Post Diagnosis
Survival Post Diagnosis * Surviving Percentage Months Follow Up *1997 - 2006 0 12 24 36 48 60 100 40 60 80 20 0 Kumar et al, Mayo Clinic Proc, 2011 86(1):12 - 8. 1 Year Mortality - 47% Median Mortality - 1.5 Years AL Amyloidosis Mortality Remains High 7 5 Year Mortality - 72%
Slide 8: Summary
CAEL - 101 Summary 8 Pioneering antibody that lead to new treatment discovery κ Bence Jones protein isolated and used to develop CAEL - 101 Dissolves human AL λ and κ in preclinical studies Interim Phase 1 data of 21 Patients, CAEL - 101 is well - tolerated and safe showing no dose limiting toxicity
Slide 9: Specificity of Antibody Binding
CAEL - 101 binds to Liver and Bone Amyloid Fibrils Wall, JS et al. Blood. 2010 Sep 30;116(13):2241 - 4. Specificity of Antibody Binding 9 Radiolabeled CAEL - 101
Slide 10: Amyloidomas Dissolved in Treated Mice
Day - 1 Day - 14 Human Amyloid deposits injected in Mice Untreated Mice CAEL - 101 Treated Mice Wall, JS et al, Tijdschr Nucl Geneeskd. 2011 Dec;33(4):807 - 814 and Wall, JS et al, Blood 2010;116:2241 - 2244 Day - 14 Day - 1 A myloidomas Dissolved in CAEL - 101 Treated Mice With Human AL λ and κ 10
Slide 11: Phase 1a/1b Organ Response Rates
Renal Response Phase 1a & 1b (n=8) Cardiac Response Phase 1a & 1b (n=8) Overall Responders Best Organ Response Phase 1a Phase 1b 12 Weeks 4 Weekly Doses 8 Weeks Single Dose 63% Responders 70% Responders 5/8 Patients 7/10 Patients Responder Stable Progressior >30% and >300 pg /ml increase in NT - pro
Slide 12: Marked and Sustained Cardiac Response After a Single Dose
Marked and Sustained Cardiac Response After a Single Dose PATIENT 3 PROFILE □ Refractory λ AL Amyloidosis □ Baseline NT - proBNP approx. 13,000 ng/L □ Previous treatments: 1 □ Best Hematologic response to chemotherapy: VGPR □ No Organ response to chemotherapy □ Persistently elevated NT - proBNP □ NY
Slide 13: Marked and Sustained Renal Response After a Single Dose
PATIENT 7 PROFILE □ Refractory λ AL Amyloidosis □ Baseline 24 - hr urine protein in mg/24hr: approx. 10,000 □ Previous treatments: 6 □ No Organ response to chemotherapy □ Persistence of significant proteinuria Organ response to CAEL - 101 □ 24 hour urine protein in ng/24 hr : approx. 3,000 Marked an
Slide 14: Highlights
CAEL - 101 Highlights 14 Experienced Leadership Team Phase 2 to commence in 2018 Organ activity independent of light chain sub - type Marked and sustained responses even after a single dose No dose limiting toxicity Promising Phase 1a/1b data Pioneering antibody
Slide 15: Key Leadership
Lindsay Rosenwald, MD Executive Chairman Michael Spector Chief Executive Officer Key Leadership 15 • Prolific biotechnology entrepreneur • Chairman and CEO of Fortress Biotech • 20+ years founding and capitalizing numerous public and private biotechnology and life sciences companies • 25+ years of l
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