Viking Therapeutics, Inc. · NASDAQ:VKTX
Viking Therapeutics — Biotech Showcase 2016
28 slides from Viking Therapeutics's conference presentation, Jan 11, 2016
Slide 1: Corporate Presentation, Biotech Showcase, January 2016
Corporate Presentation Biotech Showcase, January 2016 EXHIBIT 99.1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains statements about our future expectations, plans and prospects that constitute forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from
Slide 3: Introduction
Introduction Viking is a San Diego-based biotech company developing novel drugs for metabolic disorders Programs licensed from Ligand Pharmaceuticals Incorporated Our clinical-stage programs are targeting: Large, underserved markets Each with global product market opportunities exceeding $1B annuall
Slide 4: Investment Highlights
Investment Highlights Focused on first-in-class or best-in-class drugs for metabolic disorders Lead clinical programs have demonstrated preliminary efficacy in humans VK5211: In Phase 2 development for hip fracture Non-steroidal selective androgen receptor modulator (SARM) Promising efficacy in bone
Slide 5: Pipeline Overview and Development Plans
Preclinical Phase 1 Phase 2 Phase 3 Development Plans VK5211: SARM Hip Fracture Complete the ongoing trial in 2016 and report topline data in 2H16 VK2809: TRb Hypercholesterolemia, NASH Complete the Phase 2 trial in 2016 and report topline data in 4Q16/1H17 VK0214: TRb X-ALD Complete in vivo POC stu
Slide 6: Pipeline Catalysts and News Flow
Pipeline Catalysts and News Flow Key Upcoming Milestones Phase 2 hip fracture results expected 2H16 Phase 2 hypercholesterolemia/fatty liver results expected 4Q16/1H17 X-ALD in vivo POC data results expected mid-2016 4Q15 1Q16 2Q16 3Q16 4Q16 1Q17 2Q17 VK5211: Hip Fracture VK2809: Hypercholesterolemi
Slide 7: Clinical Programs: VK5211 SARM, Hip Fracture
Clinical Programs VK5211: Selective Androgen Receptor Modulator Hip Fracture
Slide 8: VK5211: SARM Program for Hip Fracture
VK5211: SARM Program for Hip Fracture Novel Selective Androgen Receptor Modulator (SARM), for hip fracture VK5211 is a potentially best-in-class small molecule SARM Promising efficacy signals Phase 1: Significant improvements in lean body mass Animals: Positive effects on bone Target indication: Reh
Slide 9: VK5211: Potential Therapeutic Benefits of SARMs
VK5211: Potential Therapeutic Benefits of SARMs Goal: Retain beneficial properties of natural androgens with fewer side effects relative to anabolic steroids Muscle Mass Muscle Strength Bone Formation Bone Strength Bone Resorption Cognition Libido Energy MUSCLE BONE CNS
Slide 10: VK5211: Hip Fracture an Ideal Market for SARMs
HF associated with high mortality, loss of function; increased risk of 2nd fracture 20% - 30% 1-yr mortality; 50% lose walking independence; 20% require long-term care Post-fracture: Patients experience accelerated losses of bone mineral density (BMD), lean body mass (LBM) Rates of decline greatly e
Slide 11: VK5211: Preclinical and Clinical Summary
VK5211: Preclinical and Clinical Summary Excellent preclinical profile Low nM Ki for androgen receptor, high tissue selectivity Primates: Substantial increases in lean body mass at 12 weeks Rodents: Rapid increases in bone mineral density, stimulation of bone formation Three Phase 1 studies successf
Slide 12: VK5211: Significant Weight Gain Observed in Primates
VK5211: Significant Weight Gain Observed in Primates Robust, progressive effects observed in both sexes; >70% of gains retained after 4 week recovery period. Progressive Weight Gains Through 13 Weeks Significant Increases Observed in Both Sexes Week 0 Week 4 Week 8 Week 13 Week of VK5211 Administrat
Slide 13: VK5211: Clinical Highlights, Rapid Effect on LBM
VK5211: Clinical Highlights, Rapid Effect on LBM Phase 1 multiple ascending dose study, healthy subjects (n=76) Statistically significant improvement in LBM after 21 days Positive trends in strength and performance measurements Androgenic effect on total T, SHBG Shown to be safe, well-tolerated, no
Slide 14: VK5211: Ongoing Phase 2 Trial, Hip Fracture
VK5211: Ongoing Phase 2 Trial, Hip Fracture 12-week Phase 2 trial in hip fracture patients initiated 4Q15 Primary endpoint: Change in lean body mass Exploratory endpoints: Change in BMD, functional status, ADL, QOL Data expected in 2H16 Discuss results with FDA Primary and exploratory endpoints prov
Slide 15: Clinical Programs: VK2809 Selective Thyroid Receptor-β Agonist
Clinical Programs VK2809: Selective Thyroid Receptor-β Agonist Lipid disorders
Slide 16: Thyroid-β Agonist Program
Thyroid-b Agonist Program Proprietary platform for small molecule thyroid mimetics Highly tissue and receptor selective Differentiated pro-drug technology, excellent oral bioavailability Unique chemical scaffolds, expected wider safety window vs. other approaches Biological profiles suggest potentia
Slide 17: Thyroid Receptor Overview
Thyroid Receptor Overview Nuclear hormone receptors Two major subtypes Thyroid beta receptor: Liver, brain; modulates cholesterol, triglyceride levels Thyroid alpha receptor: Cardiac tissue, modulates heart rate, contraction Therapeutic goal, lipid setting: b-receptor selectivity; minimize alpha-eff
Slide 18: VK2809: Preclinical Summary, Hypercholesterolemia
VK2809: Preclinical Summary, Hypercholesterolemia Lead TRb program: Liver-targeted pro-drug of potent TRb agonist Ki = 2.2nM, b:a selectivity 16:1 Excellent in vivo safety profile (Proc. Natl. Acad. Sci., 104(39), 15490-15495, (2007)) Potent cholesterol reduction observed in five species Rabbits Cyn
Slide 19: VK2809: Promise in NAFLD and NASH
Multi-Hit Hypothesis: VK2809: Promise in NAFLD and NASH Proportion of patients with improvement or resolution in: Patients1 Mean BMI reduction Complete resolution of NASH Steatosis Steatohepatitis Fibrosis 766 19% - 42% 70% 92% 81% 66% Cirrhosis HCC Liver failure Healthy liver Meta analysis: Clin. G
Slide 20: VK2809: Potent Reduction of Liver Fat in Preclinical Models
VK2809: Potent Reduction of Liver Fat in Preclinical Models Novel liver-targeted mechanism: Releases drug selectively in liver tissue Rapid reduction of fat content observed in models of steatosis Reduction in liver triglycerides reflected in reduced liver weights Monkeys: 18% - 29% reduction in liv
Slide 21: VK2809: 14-Day Phase 1b Highlights
VK2809: 14-Day Phase 1b Highlights Placebo-controlled trial (n=56), mild hypercholesterolemia Enrollment criterion: LDL ≥ 100 mg/dL Results: clinically, statistically significant reductions in LDL Up to 79% reduction in triglycerides Major component of liver fat Shown to be safe and well-tolerated,
Slide 22: VK2809: Phase 2 Trial, Hypercholesterolemia, Fatty Liver
VK2809: Phase 2 Trial, Hypercholesterolemia, Fatty Liver Phase 2 trial, hypercholesterolemia patients with fatty liver disease Multi-arm, dose ranging, ~20 patients per arm Primary endpoint: DLDL-C Exploratory: Changes in liver fat, triglycerides, inflammatory markers, other Potential topline data 4
Slide 23: TRb Agonists in X-Linked Adrenoleukodystrophy, X-ALD
TRb Agonists in X-Linked Adrenoleukodystrophy, X-ALD Orphan neurodegenerative disorder X-linked: Carried by females, primarily manifesting in males EU, approximately 12,000 patients; U.S. 8,000 patients No cure, no approved therapy Most severe form: Cerebral ALD Rapidly progressive inflammatory demy
Slide 24: TRb: X-Linked Adrenoleukodystrophy
TRb: X-Linked Adrenoleukodystrophy Caused by mutation in gene for the ATP-Binding Cassette transporter D1 (ABCD1) Peroxisomal transporter of very long chain fatty acids (VLCFA) Graphic adapted from http://www.x-ald.nl/origin-and-metabolism-of-vlcfa/. ABCD1: Normal function to transport VLCFA into pe
Slide 25: TRb: Strong Rationale for Benefit in X-ALD
TRb: Strong Rationale for Benefit in X-ALD Alternative VLCFA transporters ABCD2,-3 are induced by thyroid hormones Functional overlap with ABCD1; mechanistically compensate for ABCD1 deficiency Over-expression of ABCD2 corrects VLCFA elevation in vitro and in vivo Thyroid receptor-b isoform primaril
Slide 26: TRb: X-Linked Adrenoleukodystrophy, Plans
TRb: X-Linked Adrenoleukodystrophy, Plans Current status Evaluation of VK0214 ongoing, encouraging in vitro results In vivo model in progress POC results expected 2016 IND-enabling work 2H16
Slide 27: Investment Highlights
Investment Highlights Focused on first-in-class or best-in-class drugs for metabolic disorders Lead clinical programs have demonstrated preliminary efficacy in humans VK5211: In Phase 2 development for hip fracture Non-steroidal selective androgen receptor modulator (SARM) Promising efficacy in bone
Slide 28: Biotech Showcase, January 2016
Corporate Presentation Biotech Showcase, January 2016