Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — Investor Presentation (September 2022)
35 slides from Lisata Therapeutics's investor presentation, Sep 22, 2022
Slide 1: Targeted Therapy Delivered
Targeted Therapy Delivered S e p te mb e r 2 2 , 2 0 22 | N a s d a q : L STA | w w w. l i s a ta . co m David J. Mazzo, Ph.D., Chief Executive Officer Exhibit 99.1
Slide 2: Forward-looking Statements
Forward-looking Statements This presentation contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in
Slide 3: Investment highlights
Investment highlights Stable finances: ~$76 million cash & investments as of 9/15/22; no debt Proprietary field-leading technology in underserved global indications backed by a strong IP portfolio Platform technology “validated” by strong existing partnerships with potential for many others Multiple
Slide 4: Proprietary platform technologies
Proprietary platform technologies 4 CendR Platform™ - a targeted tissue penetration technology to enhance drug delivery to solid tumors Converts tumor stroma from barrier to conduit for efficient delivery of chemo-, targeted and immunotherapies • Delivery accomplished via co-administration or by t
Slide 5: Clinical development pipeline with broad therapeutic reach
Clinical development pipeline with broad therapeutic reach 5 LSTA1 (formerly known as CEND-1), advancing in a variety of difficult-to-treat solid tumor applications Ongoing multiple studies in first-line, metastatic pancreatic ductal adenocarcinoma (mPDAC) in combination with standard-of-care (SoC
Slide 6: Therapeutic potential attracts strategic partners
Therapeutic potential attracts strategic partners Strategic partnership in China with Qilu Pharmaceutical Exclusive rights to LSTA1 in China, Taiwan, Hong Kong and Macau Qilu assumes all development and commercialization responsibilities in the licensed territories Potential for up to $225 mil
Slide 7: Development programs and next milestones table
Sponsor/Funding Partner Trial Products Indication Development Stage Next Development Milestone CendR Platform™ Programs Lisata (Global) Gemcitabine/nab-paclitaxel ± LSTA1 First-Line Metastatic Pancreatic Ductal Adenocarcinoma (mPDAC) AGITG (Australia/New Zealand) Gemcitabine/nab-paclitaxel ± LSTA1 Q
Slide 8: LSTA1 (formerly known as CEND-1) CendR Platform
LSTA1 (formerly known as CEND-1) CendR Platform™
Slide 9: LSTA1 mechanism of action (part 1)
LSTA1 mechanism of action (part 1) or αvβ3/β5 integrin LSTA1 Protease Peptide Cleaved CendR Peptide Fragment Tumor Vascular Endothelial Cell Nucleus Cargo Co- admin Drug 9 LSTA1 - 9 amino acid cyclic peptide; high binding specificity and affinity to αvβ3/β5 integrins that are selectively expressed
Slide 10: LSTA1 mechanism of action (part 2)
LSTA1 mechanism of action (part 2) Neuropilin-1 receptor CendR Peptide Fragment Co-administered anti-cancer therapy CendR Pathway Tumor Vascular Endothelial Cell Nucleus Gap junction opening 10 CendR fragment binds to Neuropilin-1 receptor, activating the CendR transport pathway • CendR peptide an
Slide 11: LSTA1 selectively and efficiently facilitates intratumoral delivery
LSTA1 selectively and efficiently facilitates intratumoral delivery Tumor-Targeting Penetration: In vivo Imaging pancreatic ductal adenocarcinoma (PDAC) with LSTA1 + Fluorescent Quantum Dots (FQDs) 1 Braun et al., Nature Mater. 2014. 2 Liu, Braun et al., Nature Comm. 2017. 11 FQD + LSTA1 followed
Slide 12: Increased tumor penetration enhances activity across treatment modalities
1 Hurtado de Mendoza et al, Nature Comms, 2021. 2 Liu X et al., J Clin Invest, 2017. 12 Lung cancer + gemcitabine Breast cancer + nanoparticle Abraxane GI cancer + adoptive cell therapyPancreatic ductal adenocarcinoma Orthotopically transplanted KPC PDAC tumors CEND-1 + irinotecan nanoparticles (i.v
Slide 13: Treatment of solid tumors represents a large unmet clinical need
Treatment of solid tumors represents a large unmet clinical need Estimated New Cancer Cases and Deaths in the United States, 20221 1 CA A Cancer J Clinicians, Volume: 72, Issue: 1, Pages: 7-33, First published: 12 January 2022, DOI: (10.3322/caac.21708) It is estimated that more than 1.9 million new
Slide 14: Phase 1b clinical results of LSTA1
Compelling Phase 1 clinical results of LSTA1 14 Phase 1b: 31 subjects enrolled, 29 evaluable first-line, mPDAC patients from 3 sites in Australia [gemcitabine + nab-paclitaxel) with and without LSTA1 LSTA1 well-tolerated, no dose-limiting toxicities; safety with LSTA1 consistent with SoC alone
Slide 15: Ongoing & Planned LSTA1 Clinical Trials
Ongoing & Planned LSTA1 Clinical Trials
Slide 16: ASCEND: Phase 2b randomized, double-blind trial in Aus and NZ
ASCEND: Phase 2b randomized, double-blind trial in Aus and NZ Sponsor/Partner Australasian Gastro-Intestinal Trials Group (AGITG) in collaboration with the NHMRC Clinical Trial Centre at the University of Sydney AGITG co-funded Design Phase 2b randomized, double-blind study in mPDAC Study Size
Slide 17: LSTA1 Phase 1b/2 trial in China
LSTA1 Phase 1b/2 trial in China Sponsor/Partner QILU Pharmaceutical (provides all funding) Design Phase 1b/2 open-label study in advanced mPDAC patients Study Size 50 subjects (~15 sites; Chinese population) Endpoints Primary: AEs, SAEs, Objective Response Rate, Duration Response Rate, Disea
Slide 18: CENDIFOX: Phase 1b/2 trial in U.S.
CENDIFOX: Phase 1b/2 trial in U.S. Sponsor/Partner University of Kansas Medical Center (Investigator initiated trial) Design Phase 1b/2 open-label study in pancreatic, colon and appendiceal cancers Study Size 50 subjects Endpoints Primary: Drug Safety Secondary: Overall Survival, Disease-f
Slide 19: Planned LSTA1 clinical trials
Planned LSTA1 clinical trials PHASE 2/3 ADAPTIVE TRIAL IN mPDAC PHASE 2A BASKET TRIAL IN MULTIPLE TUMOR TYPES Sponsor Lisata Lisata Design Phase 2/3, adaptive, double-blind, placebo- controlled, randomized trial in mPDAC (Global) - pending FDA agreement Phase 2, double-blind, placebo-control
Slide 20: Tissue-Penetrating Nanoparticle (TPN) Platform
Tissue- Penetrating Nanoparticle (TPN) Platform™
Slide 21: TPN Platform for nucleic acid medicine delivery in solid tumors
TPN Platform™ for nucleic acid medicine delivery in solid tumors 21 RNA-based drugs have not been successful in the treatment of cancer despite advancement of candidates to multiple “undruggable” high-interest anticancer targets Early antisense oligonucleotide (ASO) and small interfering RNA (si
Slide 22: TPN Platform addresses nucleic acid tumor delivery challenges
TPN Platform™ addresses nucleic acid tumor delivery challenges 22 Self-Assembly Varying components enables control of particle size and other properties Tumor-Penetrating NanocomplexsiRNA pictured, also demonstrated with single stranded antisense and microRNAs CendR targeting peptide Endosome escape
Slide 23: CD34+ Cell Therapy Platform Technology
CD34+ Cell Therapy Platform Technology
Slide 24: XOWNA (LSTA16) Coronary Microvascular Dysfunction (USA)
XOWNA® [LSTA16 (formerly known as CLBS16)] Coronary Microvascular Dysfunction (USA)
Slide 25: XOWNA development status
XOWNA® development status 2525 1 Marinescu MA, et al. JACC Cardiovasc Imaging. 2015;8:210-220 Coronary Microvascular Dysfunction (CMD) represents a large unmet medical need • Deficient heart microvasculature without large vessel obstructive disease causing frequent, severe angina • Not treatable b
Slide 26: HONEDRA (LSTA12) Critical Limb Ischemia (Japan)
HONEDRA® [LSTA12 (formerly known as CLBS12)] Critical Limb Ischemia (Japan) SAKIGAKE designated – Japan Orphan Drug designated (Buerger’s disease) - USA Advanced Therapeutic Medicinal Product (ATMP) designated – EU
Slide 27: Indication: critical limb ischemia (CLI)
Indication: critical limb ischemia (CLI) Severe arterial obstruction impeding blood flow in the lower extremities • Includes severe rest pain and non-healing ulcers Buerger’s disease (BD: inflammation in small and medium arteries) is a form of CLI associated with a history of heavy smoking (orph
Slide 28: HONEDRA registration-eligible study in Japan
HONEDRA® registration-eligible study in Japan Primary Endpoint Time to continuous CLI-free (2 consecutive monthly visits, adjudicated independently) Target Study Size 35 subjects; recruited across 12 centers in Japan • 30 with no-option CLI (ASO) + 5 with BD; all Rutherford category 4 or 5 Dose
Slide 29: HONEDRA development next steps
HONEDRA® development next steps Combined CLI and BD interim data suggest trend toward efficacy and acceptable safety • HONEDRA® was safe and well tolerated • Treatment group reached CLI-free status faster than SoC group (primary endpoint) Consultation process with the Pharmaceuticals & Medical D
Slide 30: LSTA201 Diabetic Kidney Disease
LSTA201 (formerly known as CLBS201) Diabetic Kidney Disease
Slide 31: LSTA201 in diabetic kidney disease (DKD)
LSTA201 in diabetic kidney disease (DKD) The stages of CKD are determined by GFR rate, an indication of how well the kidneys are filtering blood1 CKD is often associated with progressive microvasculature damage and loss2,3 Preclinical studies show that microcirculation replenishment improves k
Slide 32: LSTA201: Phase 1b open-label, proof-of-concept study in U.S.
LSTA201: Phase 1b open-label, proof-of-concept study in U.S. Endpoints Change in eGFR compared to baseline, assessed at 6 months Change in Urine albumin-to-creatinine ratio (UACR) and urine protein-to-creatinine ratio (UPCR) from baseline to 3 and 6 months Study Size 6 patients (1 sentinel - u
Slide 33: Anticipated milestones
Anticipated milestones Oncology Programs Ischemic Disease Programs 3333 2022 2023 2024 HONEDRA® PMDA clinical pre-consultation 2Q22 LSTA201 topline data expected 1Q23 HONEDRA® PMDA non-clinical consultation 2023 HONEDRA® PMDA formal clinical consultation 2Q23 HONEDRA® pre-JNDA pre-consultation 3Q23
Slide 34: Investment highlights
Investment highlights Stable finances: ~$76 million cash & investments as of 9/15/22; no debt Proprietary field-leading technology in underserved global indications backed by a strong IP portfolio Platform technology “validated” by strong existing partnerships with potential for many others Multiple
Slide 35: Investor Relations Contact
S e p te mb e r 2 2 , 2 0 22 | N a s d a q : L STA | w w w. l i s a ta . co m Investor Relations Contact: John D. Menditto VP, IR & Corporate Communications o: (908) 842-0084 | e: jmenditto@caladrius.com
Lisata Therapeutics — Investor Presentation (September 2022) on 6ix