Sorrento Therapeutics, Inc. · OTC:SRNE
Sorrento Therapeutics — Investor Presentation (February 2014)
36 slides from Sorrento Therapeutics's investor presentation, February 2014
Slide 1: Sorrento Therapeutics: Next-Generation Cancer Therapeutics
Sorrento Therapeutics Next-Generation Cancer Therapeutics February 2014
Slide 2: Safe Harbor Statement
Safe Harbor Statement NASDAQ: SRNE This presentation contains "forward-looking statements" as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA), including statements regarding expectations, beliefs or intentions regarding our business, technologies and products
Slide 3: Management Team
Management Team Henry Ji, Ph.D. Inventor of G-MAB® Technology Board of Directors President, CEO & President & CEO of Stratagene Genomics Director VP of CombiMatrix and Stratagene William S. Marth - Chairman Albany Molecular (President and CEO) Vuong Trieu, Ph.D. Founder and CEO of IgDraSol Co-invent
Slide 4: Investment Highlights
Investment Highlights Late-Stage Cancer Drug Cynviloq Product launch expected in 1H 2016 Addresses multi-billion dollar paclitaxel market Abbreviated regulatory pathway (“bioequivalence”) for approval RTX Intractable Cancer Pain Treatment Ongoing Phase 1/2 study Orphan drug status received Three pot
Slide 5: Next-Generation Cancer Therapeutics
Next-Generation Cancer Therapeutics G-MAB High-diversity human Ab library Lead mAb programs include PD-L1, PD-1, and CCR2 TUMOR Cynviloq Bioequivalence regulatory pathway Efficacy demonstrated NA, EU and Australia rights ADC: Antibody Drug Conjugate G-MAB targets toxin to cancer cell Proprietary tox
Slide 6: Multiple Commercialization & Partnership Opportunities
Multiple Commercialization & Partnership Opportunities INDICATION Metastatic Breast Cancer Non-Small Cell Lung Cancer } 505(b)(2) Bioequivalence 1H 2014 1H 2015 Cynviloq Pancreatic Cancer (BE* or sNDA) Bladder Cancer (sNDA) Ovarian Cancer (sNDA) PHASE 1 PHASE 2 RTX Intractable Cancer Pain In progres
Slide 7: Lead Oncology Product Opportunity: Cynviloq
Lead Oncology Product Opportunity Cynviloq 7
Slide 8: Cynviloq: Next Generation Paclitaxel Therapy
Cynviloq: Next Generation Paclitaxel Therapy Maximum Peak Generation Formulation Tolerated Dose Product Sales 1st Cremophor EL Taxol® excipient: 175 mg/m2 ~ $1.6B (WW in 2000) paclitaxel Polyoxyethylated castor oil 2nd Biological polymer: 260 mg/m2 Est. >$1.7B* (US) Abraxane Mean size Donor-derived
Slide 9: Cynviloq Represents a High Value Proposition
Cynviloq Represents a High Value Proposition 1. FDA concurred – July 2013 a. Available data support pursuing 505(b)(2) regulatory pathway b. Bioequivalence (BE) study sufficient for approval of indications in Abraxane label (MBC and NSCLC) 2. Efficacy demonstrated in Phase 2 and Phase 3 studies a. A
Slide 10: Clinical Efficacy & Safety Summary
Clinical Efficacy & Safety Summary Total number of patients across all trials: 1,260 Phase 1: Trials established higher MTD in US - Dana Farber Cancer Inst, Russia, & S. Korea (total n=80) >300 mg/m2 (q3w) vs. 175 mg/m2 (Taxol; weekly) and 275 mg/m2 (Abraxane; q3w) Phase 2: Completed trials in MBC,
Slide 11: Equivalent PK in Mice
Equivalent PK in Mice Abraxane Cynviloq AUCinf Drug HL (h) T max (h) Vz (mL/kg) Cl (mL/h/kg) (h*ng/mL) Abraxane 2.99 0.08 61561.33 2103.71 487.32 Cynviloq 2.83 0.08 58151.31 2103.58 515.90 Data on file IV bolus at 30 mg/kg; n = 3 11
Slide 12: Comparable PK in Humans
Comparable PK in Humans Data from 2 separate studies (3 h infusion, 135 mg/m2 dose, n=3) Abraxane data from: Nuhad K. Ibrahim, Phase I and Pharmacokinetic Study of ABI-007, a Cremophor-free, Protein-stabilized, Nanoparticle Formulation of Paclitaxel. Clinical Cancer Research 2002;8:1038-44 12
Slide 13: Simulated PK Parameters Supportive of BE
Simulated PK Parameters Supportive of BE Comparison of mean non-compartmental PK parameters of Cynviloq (T) and Abraxane (260 mg/m2) at 30 min infusion time: Cmax Ratio AUCinf Ratio (ng/mL) Cmax(T)/Cmax(R) (ng*h/mL) AUCinf(T)/AUCinf(R) Cynviloq 19486 22198 (Simulated PK) 99.6% 109.2% Abraxane 19556
Slide 14: Bioequivalence = Efficient Pathway to Market
Bioequivalence = Efficient Pathway to Market BE registration study in breast cancer patients (2014) - Duration = 12 months (including patient recruitment) - Direct trial cost ~$5M Key Parameters*: Abraxane Cynviloq Dose: 260 mg/m2 Cycle 1 (n = 50) (n = 50) Infusion time: 30 min Duration: 3 weeks + C
Slide 15: Potential Cynviloq Advantages
Potential Cynviloq Advantages Cynviloq Cynviloq Abraxane Taxol Advantage Maximum Tolerated Dose Potential for >300 260 175 (mg/m2) higher efficacy Rapid reconstitution: Convenience for busy no foaming concerns ● ● practices and pharmacies No donor-derived human serum albumin (HSA) ● ● No viral / pri
Slide 16: Cynviloq Market Opportunity
Cynviloq Market Opportunity ~70,000 patients treated with paclitaxel-based regimen in 1st line 100,000 # of Patients Treated in 1st line (US Only; 2012) 90,000 Other regimens ~55,000 patients with 80,000 PAC+ treated paclitaxel as 1st line therapy in MBC, NSCLC & OC 70,000 60,000 50,000 15,903 PC pa
Slide 17: Potential to Expand Label Indications – 2nd Line Bladder Cancer
Potential to Expand Label Indications – Example: 2nd Line Bladder Cancer Cynviloq Best Supportive Care Phase 2 (Korea)* Phase 2 (Japan)**/ Phase 3 (EU)*** 260-300 mg/m2 q3w n=23** / n=108*** n=34# ORR 21% - / 0%*** PFS 2.7 M - / 1.5 M*** OS 6.5 M 2.3 M** / 4.3 M*** Summary: High unmet need - no FDA-
Slide 18: Next Steps for Cynviloq
Next Steps for Cynviloq BE study initiation: 1Q 2014 NDA filing: 2015 Product launch (MBC and NSCLC): 2016 sNDA planning for label expansion into pancreatic, bladder, and ovarian cancers BE NDA FDA LAUNCH Study Filing Approval 2014 2015 2016 2016 18
Slide 19: Clinical Stage Pain Management Asset: RTX
Clinical Stage Pain Management Asset RTX 19
Slide 20: Resiniferatoxin (RTX): Non-opiate Pain Treatment
Resiniferatoxin (RTX): Non-opiate Pain Treatment Single injection of RTX for treatment of intractable pain Produces broad, permanent analgesia No effect on normal sensation, acute pain perception or muscle function Ultrapotent agonist of TRPV1 receptors causing neuronal apoptosis TRPV1 up-regulated
Slide 21: Two Injection Sites = Two Products for Human Use
Two Injection Sites = Two Products for Human Use Intraganglionic Intrathecal (injection into (injection into nerve body) spinal column) 21
Slide 22: Single Injection Addresses Cancer Pain in Dogs
Single Injection Addresses Cancer Pain in Dogs Single intrathecal injection n=18 Permanent ablation of nerves Observational Pain Score VAS (0 to 100 mm) Attractive therapeutic effect: ALL dogs responded (p < 0.0001 for all time points) Personality & mood visibly improved No opioid-associated side ef
Slide 23: Next Steps for RTX Development
Next Steps for RTX Development Intractable cancer pain clinical Phase 1/2 trial (intrathecal injection); n~40 patients Phase 1/2 trial for osteosarcoma (intraganglionic injection); n~15 patients (under NIH CRADA) Filing for MUMS designation for osteosarcoma in dogs ~3 years for clinical development
Slide 24: Immunotherapy Programs: G-MAB & ADC
Immunotherapy Programs G-MAB & ADC 24
Slide 25: G-MAB: Library of Therapeutic Antibodies
G-MAB: Library of Therapeutic Antibodies Proprietary technology: Very high library diversity: RNA amplification used for 2.1 x 1016 distinct antibodies library generation Freedom-To-Operate Fully human antibodies No stacking royalties High successful screening hit rate Most Difficult Targets: G Prot
Slide 26: Anti-PD-L1 mAbs Exhibit Potent Activity
Anti-PD-L1 mAbs Exhibit Potent Activity Immune Modulation* Tumor Mouse Model** *** Tumor Growth Inhibition (%) T Cell Activation (%) IFN-γ (pg/mL) IL-2 (pg/mL) Day Competitor mAb Sorrento mAb * mAbs @ 0.05 mg/mL ** xenograft model using H1975 human NSCLC cells; % inhibition relative to control mAb t
Slide 27: Potent Antibody against Difficult GPCR Target
Potent Antibody against Difficult GPCR Target* 3.5 Cell Binding mAb (EC50 – nM) Sorrento 0.17 3 Competitor 21 2.5 Disease Score** 2 Untreated PBS Sorrento D1 mAb therapeutic 1.5 1 0.5 0 13 14 15 16 17 18 19 20 21 22 23 24 25 26 27 28 29 30 31 32 33 34 35 36 37 38 Days After Disease Induction * Sorre
Slide 28: Antibody Drug Conjugates (ADCs)
Antibody Drug Conjugates (ADCs) Key Components: 1. Target-specific internalizing antibody 2. Potent cytotoxic prodrugs 3. Linker and conjugation chemistries Drug released in CANCER CELL 28
Slide 29: K-Lock Conjugation Enables Homogeneous ADCs
K-Lock Conjugation Enables Homogeneous ADCs Current industry standard chemistry 2 Proprietary 1 K-Lock chemistry 3 0 purified 2 Sorrento’s homogenous ADC No need for: non-natural amino acids genetic re-engineering enzymatic posttranslational modification 29
Slide 30: C-Lock Conjugation Stabilizes ADCs
C-Lock Conjugation Stabilizes ADCs Maleimide conjugation Destabilizes antibody structure Current industry Reduced target specificity standard Altered PK profile Drug-antibody linkage not stable Off-target drug effects C-lock conjugation Enhances ADC stability Sorrento’s Prolongs PK profile proprieta
Slide 31: Proprietary Toxin Potency Higher than DM1
Proprietary Toxin Potency Higher than DM1 SKBR3 (Breast Cancer Cell Line) Sorrento toxin 1 (K-lock) >15x higher Sorrento toxin 2 (K-lock) potency DM1 (conventional) MMAE (C-lock) MMAE (conventional) cleavable MC-vc-PAB linker 31
Slide 32: Proprietary ADC Screening and Optimization Panels
Proprietary ADC Screening and Optimization Panels Fast track to IND Identification of optimal combination of linker, conjugation chemistry and drug payload essential for efficient and expedited development from target to candidates 32
Slide 33: Positioned to Become Oncology Leader
Positioned to Become Oncology Leader Cynviloq RTX G-MAB & ADC 33
Slide 34: Company Comparables
Company Comparables Company Small Molecule Antibody Targeted Mkt Cap* Oncology Drug Platform Drug Delivery Puma: PBYI MBC (Phase 3) ~$3.2B Pre-revenue Clovis: CLVS NSCLC, MBC (Phase 1) ~$2.4B Pre-revenue MorphoSys: MOR.DE Antibody ~$2.2B Pre-revenue Library CAT: Acquired (2006) Antibody ~$1.4B Pre-r
Slide 35: Investment Highlights
Investment Highlights Late-Stage Cancer Drug Cynviloq Product launch expected in 1H 2016 Addresses multi-billion dollar paclitaxel market Abbreviated regulatory pathway (“bioequivalence”) for approval RTX Intractable Cancer Pain Treatment Ongoing Phase 1/2 study Orphan drug status received Three pot
Slide 36: Sorrento Therapeutics
Sorrento Therapeutics Next-Generation Cancer Therapeutics Contact: Henry Ji President and CEO hji@sorrentotherapeutics.com (858) 668-6923
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