Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — Investor Presentation (February 2023)
57 slides from Lisata Therapeutics's investor presentation, Feb 6, 2023
Slide 1: Corporate Presentation – Targeted Therapy Delivered
C o p y r i g h t © 2 0 2 3 L i s a t a T h e r a p e u t i c s , I n c . A l l r i g h t s r e s e r v e d . LISATA EUTICSTHERAP Targeted Therapy Delivered David J. Mazzo, Ph.D. Chief Executive Officer Corporate Presentation | February 6, 2023 Nasdaq: LSTA www.lisata.com EXHIBIT 99.1
Slide 2: Forward-looking Statements Notice
Forward-looking Statements Notice 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, incl
Slide 3: Lisata Therapeutics overview
LISATA EUTICSTHERAP Nasdaq-listed clinical stage therapeutics development company with a novel solid tumor targeting and penetration technology to improve the efficacy of anti-cancer drugs 3 Lisata Therapeutics
Slide 4: Investment rationale
Investment rationale 4 * As of 12/31/2022; unaudited Proprietary field-leading technology in underserved global indications backed by a strong IP portfolio $69.2 million cash and investments*- no debt; Highly capital efficient development plans funded through critical milestones Multiple projected p
Slide 5: Why Oncology? Improved cancer treatment is a global need
Why Oncology? - Improved cancer treatment is a global need 5 Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, or nearly one in six deaths1 – World Health Organization 1 www.who.int/news-room/fact-sheets/detail/cancer
Slide 6: Opportunity: solid tumors are a large & growing treatment market
Opportunity: solid tumors are a large & growing treatment market 1 CA A Cancer J Clinicians, Volume: 72, Issue: 1, Pages: 7-33, First published: 12 January 2022, DOI: (10.3322/caac.21708) 6 It is estimated that more than 1.9 million new cases of cancer will be diagnosed in 2022 In the U.S. alone, so
Slide 7: Challenge: intratumoral drug exposure insufficient for ideal response
Challenge: intratumoral drug exposure insufficient for ideal response Clinical response to many anti-cancer drugs is suboptimal Tumor targeting and intratumoral penetration are inadequate • Tumor stroma acts as an effective barrier to anti-cancer agent penetration • Tumor microenvironment immuno
Slide 8: Solution: Lisata's CendR Platform
Solution: Lisata’s CendR Platform® Converts tumor stroma from barrier to conduit for penetration of anti-cancer treatments • Combination with many existing & emerging anti-cancer drugs possible in multiple indications • Mechanism effective with co-administered or tethered anti-cancer therapies • C
Slide 9: LSTA1: CendR Platform lead development candidate
LSTA1: CendR Platform® lead development candidate Multiple Phase 1b to 2b studies in metastatic pancreatic ductal adenocarcinoma (mPDAC) combined with standards-of-care (SoC) chemotherapy [i.e., (gemcitabine + nab-paclitaxel) or FOLFIRINOX] • Granted Fast Track and Orphan Drug Designations by the
Slide 10: Broad applicability: noteworthy existing partnerships and beyond
Broad applicability: noteworthy existing partnerships and beyond 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/costs in licensed territories Potential f
Slide 11: LSTA1 Mechanism of Action and Early Clinical Results
LSTA1 [formerly known as CEND-1] Well-characterized Mechanism of Action with Compelling Early Clinical Results
Slide 12: LSTA1: mechanism of action step one
αvβ3/β5 integrin LSTA1 (Furin & others) Protease Cleavage CendR1 Peptide Fragment Tumor Vascular Endothelial Cell Cell Nucleus 12 LSTA1: 9 amino acid cyclic peptide; high binding specificity and affinity to αvβ3/β5 integrins that are selectively expressed on: • Tumor vascular endothelium • Cancer-
Slide 13: LSTA1: mechanism of action step two
LSTA1: mechanism of action step two Neuropilin-1 CendR Peptide Fragment Co-administered or tethered anti-cancer drugs CendR Transport Pathway Tumor Vascular Endothelial Cell Nucleus Gap junction opening 13 The CendR fragment then binds with high affinity to an adjacent receptor, neuropilin-1, acti
Slide 14: LSTA1 selectively and efficiently facilitates intratumoral penetration
LSTA1 selectively and efficiently facilitates intratumoral penetration Whole body imagining of mice with pancreatic ductal adenocarcinoma (arrow) dosed with Fluorescent Quantum Dots (FQDs) with and without LSTA1 1 Braun et al., Nature Mater. 2014. 2 Liu, Braun et al., Nature Comm. 2017. 14 Etching
Slide 15: Increased tumor penetration enhances antitumor activity across modalities
1 Hurtado de Mendoza et al, Nature Comms, 2021. 2 Liu X et al., J Clin Invest, 2017. 15 Lung cancer + gemcitabine + LSTA1 Breast cancer + nanoparticle Abraxane + LSTA1 GI cancer + adoptive cell therapy + LSTA1PDAC + irinotecan nanoparticles + LSTA1 Orthotopically transplanted KPC PDAC tumors CEND-1
Slide 16: LSTA1 Phase 1b results reinforce promise of improving SoC efficacy
LSTA1 Phase 1b results reinforce promise of improving SoC efficacy N= # of study participants Median Overall Survival Median Progression-Free Survival Objective Response Rate Complete Response Partial Response Stable Disease Progressive Disease Disease Control Rate 16 weeks CA19-9 >20% drop N=171 6.
Slide 17: LSTA1 Clinical Development Portfolio
LSTA1 [formerly known as CEND-1] Clinical Development Portfolio Fast Track and Orphan Drug designated (PDAC) - USA
Slide 18: LSTA1 capital efficient development plan (1)
Development Partner(s) [Development Venue] Indication and Trial Product/Comparator Stage of Development Lisata/AGITG [Australia/New Zealand/Ireland] First-line mPDAC; Gemcitabine/nab-paclitaxel with LSTA1 or placebo Phase 2b (ASCEND) Lisata [United States] Various Solid Tumors; SoC with LSTA1 or pla
Slide 19: LSTA1 capital efficient development plan (2)
Development Partner(s) [Development Venue] Indication and Trial Product/Comparator Stage of Development WARPNINE/Lisata [Australia] Locally advanced resectable PDAC; Durvalumab/gemcitabine/nab-paclitaxel + LSTA1 Phase 1b/2a (iLSTA) WARPNINE/Lisata [Australia] Locally advanced resectable Gastroesopha
Slide 20: Tissue-Penetrating Nanocomplex (TPN) Platform
Tissue-Penetrating Nanocomplex (TPN) Platform™ Applying the CendR Platform® to nucleic acid-based drugs
Slide 21: Anticancer applications of nucleic acid-based therapeutics
Anticancer applications of nucleic acid-based therapeutics Tumor stroma serves as primary impediment to effective delivery of antisense oligonucleotides (ASO) and small interfering RNS (siRNA) drugs • >95% of ASO and siRNA drugs sequestered in endosomes • Passive targeting (i.e., lipid nanoparticles
Slide 22: TPN Platform: applying CendR technology to nucleic acid delivery
TPN Platform™ : applying CendR technology to nucleic acid delivery 2222 CendR peptides provide tumor and/or immune cell targeting with optimized tumor penetration Technologies to evade endosome sequestration Simpler synthesis vs. biologics such as virus-like particles, Ab-conjugates or exosome
Slide 23: CD34+ Cell Therapy Platform Technology
CD34+ Cell Therapy Platform Technology
Slide 24: CD34+ cell therapy current clinical trials
2424 Sponsor [Development Venue] Indication and Trial Product/Comparator Stage of Development Lisata [Japan] Critical Limb Ischemia & Buerger's Disease; HONEDRA® (LSTA12) Registration Eligible Lisata [United States] Diabetic Kidney Disease; LSTA201 Phase 1b – Proof of Concept CD34+ cell therapy curr
Slide 25: HONEDRA: 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 26: HONEDRA: autologous CD34+ cell therapy
HONEDRA®: autologous CD34+ cell therapy 2626 1 Reinecke H., European Heart Journal, 2015 Apr 14;36(15):932-8 2 Kinoshita et al, Atherosclerosis 224 (2012) 440-445 CLI is arterial obstruction impeding blood flow in the lower extremities with severe rest pain and non-healing ulcers Buerger’s disea
Slide 27: HONEDRA: registration eligible trial completed
HONEDRA®: autologous CD34+ cell therapy Registration eligible clinical trial completed • CLI and BD 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) Development Status
Slide 28: LSTA201 Diabetic Kidney Disease
LSTA201 (formerly known as CLBS201) Diabetic Kidney Disease
Slide 29: LSTA201 in diabetic kidney disease (DKD)
LSTA201 in diabetic kidney disease (DKD) CKD stages are determined by estimated glomerular filtration rate (eGFR), 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 microcirculat
Slide 30: LSTA201 clinical trial completed
LSTA201 in diabetic kidney disease Clinical trial completed • CKD patients can tolerate mobilization, donation and administration of CD34+ cell therapy • LSTA201 did not demonstrate consistent improvement kidney function in all subjects Development Status 3030 • Further clinical study required to
Slide 31: A wealth of anticipated key milestones
2023 2024 ASCEND [AUS, NZ, IRE] First-line mPDAC Basket Trial [USA] Various Solid Tumors CENDIFOX [USA] Pancreatic, Colon & Appendiceal cancers MORPHEUS [Multi-national] First-line mPDAC Qilu: Phase 1b/2a Trial [CHN] First-line mPDAC Qilu: Phase 2b Trial [CHN] First-line mPDAC iLSTA [AUS] Resectable
Slide 32: Lisata Therapeutics: financial summary
Lisata Therapeutics: financial summary $69.2M Cash & Investments (12/31/2022)* $0 Debt 1H2025 (through key development milestones) Projected Cash Runway Common Shares Outstanding (12/31/2022): 7.9 million shares Options Outstanding (12/31/2022): Exercise Price: $0.02 - $4.22 = 1,127,000 shares Exerc
Slide 33: Investment rationale
Investment rationale 33 * As of 12/31/2022; unaudited Proprietary field-leading technology in underserved global indications backed by a strong IP portfolio $69.2 million cash and investments*- no debt; Highly capital efficient development plans funded through critical milestones Multiple projected
Slide 34: Investor Relations Contact
C o p y r i g h t © 2 0 2 3 L i s a t a T h e r a p e u t i c s , I n c . A l l r i g h t s r e s e r v e d . LISATA EUTICSTHERAP Targeted Therapy Delivered Investor Relations Contact: John D. Menditto VP, IR & Corporate Communications o: (908) 842-0084 | e: jmenditto@lisata.com Nasdaq: LSTA | www.l
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Slide 36: Development partners and strategic rationale (1)
Development Partner(s) [Development Venue] Indication and Trial Product/Comparator Stage of Development Strategic Rationale Lisata/AGITG [Australia/New Zealand/Ireland] First-line mPDAC; Gemcitabine/nab-paclitaxel with LSTA1 or placebo Phase 2b (ASCEND) Corroborate Phase 1b results in a placebo- con
Slide 37: Development partners and strategic rationale (2)
Development Partner(s) [Development Venue] Indication and Trial Product/Comparator Stage of Development Strategic Rationale WARPNINE/Lisata [Australia] Locally advanced resectable PDAC; Durvalumab/gemcitabine/nab-paclitaxel + LSTA1 Phase 1b/2a (iLSTA) Assess LSTA1 safety and effectiveness in combina
Slide 38: CD34+ cell therapy trials and strategic rationale
3838 Sponsor [Development Venue] Indication and Trial Product/Comparator Stage of Development Strategic Rationale Lisata [Japan] Critical Limb Ischemia & Buerger's Disease; HONEDRA® (LSTA12) Registration Eligible Assess safety and efficacy of LSTA12 in a controlled trial vs. SoC alone in the context
Slide 39: ASCEND: Phase 2b randomized trial in mPDAC
ASCEND: Phase 2b, blinded, randomized trial in mPDAC Sponsor/Partner Australasian Gastro-Intestinal Trials Group (AGITG) in collaboration with the NHMRC Clinical Trials Centre at the University of Sydney Lisata funded (LSTA eligible for ~43% rebate on all qualified R&D expenses in AUS) Objective
Slide 40: ASCEND trial design
R INTERVENTION Arm (N=60) • Nab-paclitaxel 125 mg/m2 IV • LSTA1 3.2 mg/kg IV • Gemcitabine 1000 mg/m2 IV Dose on days 1, 8, 15 every 28 days CONTROL Arm (N=30) • Nab-paclitaxel 125 mg/m2 IV • Matching LSTA1 Placebo IV • Gemcitabine 1000 mg/m2 IV Dose on days 1, 8, 15 every 28 days INTERVENTION Arm (
Slide 41: Phase 1b/2a open-label trial in mPDAC in China
Sponsor/Partner Qilu Pharmaceutical (funds all development in China) Objective Evaluate safety, pharmacokinetics and preliminary efficacy of LSTA1 added to SoC in Chinese patients with mPDAC Design Phase 1b/2a open-label study in advanced mPDAC patients of Chinese ethnicity testing SoC chemoth
Slide 42: Qilu Phase 1b/2a trial design
7 Day Safety Evaluation LSTA1 1.6 mg/kg + nab-pac* + gem Days 1, 8, and 15 every 28 days Confirm Eligibility Informed Consent Extension Stage mPDAC 1.6 mg/kg LSTA1 Day 1 Phase 1b/2a study evaluating the safety, pharmacokinetics, and preliminary efficacy of LSTA1 for injection in Chinese patients wit
Slide 43: CENDIFOX trial overview
Sponsor/Partner University of Kansas Medical Center (Investigator initiated trial in U.S.) KUCC funded; Lisata provides LSTA1 Objective Evaluate the safety and therapeutic effect of LSTA1 in combination with neoadjuvant FOLFIRINOX-based therapies and an EGFR inhibitor for the treatment of panc
Slide 44: CENDIFOX trial design
Surgery COHORT 1 Resectable and borderline resectable PDAC Key Objectives: • Pathological response • Immune response pre- & post- treatment • PFS, OS FOLFIRINOX X 3 Cycles (± Panitumumab if RAS/BRAF wildtype - Cohorts 2, 3) Tissue immune profiling Biopsy if archival tissue not available COHORT 2 Col
Slide 45: Basket: Phase 2 PoC trial in various cancers
Sponsor/Partner Lisata (U.S.) Objective Evaluate the preliminary efficacy, safety and tolerability of LSTA1 in combination with standards of care in subjects with advanced solid tumors Design Phase 2 randomized, double-blind, placebo-controlled, proof-of-concept trial in 2nd line head and neck
Slide 46: Basket trial design
Dosed on Days 1, 8 every 21 days X 8 cycles Dosed every 21 days Dosed every 21 days Disease Progression Response rates Safety LSTA1 + paclitaxel Confirm Eligibility Informed Consent 2nd line Esophageal SCC (after failure on first line IO) Survival Analysis 72-hour run-in without SoC 72-hour run-in w
Slide 47: Phase 2b trial in mPDAC in China
Sponsor/Partner Qilu Pharmaceutical (funds all development in China) Objective Further evaluate safety and therapeutic efficacy of LSTA1 when added to SoC in Chinese patients with mPDAC Design Phase 2b, double-blind, placebo-controlled, randomized study evaluating LSTA1 + SoC (Qilu-produced na
Slide 48: Qilu Phase 2b trial design
Days 1, 8, 15 and every 28 days Disease Progression Response rates PFS Safety Gemcitabine + Qilu produced nab-paclitaxel + LSTA1 3.2 mg/kg Confirm Eligibility Informed Consent 1:1 Survival AnalysismPDAC R Gemcitabine + Qilu produced nab-paclitaxel + placebo N=TBD N=TBD Phase 2b, double-blind, placeb
Slide 49: MORPHEUS: Phase 1b/2 mPDAC umbrella trial
MORPHEUS: Phase 1b/2 mPDAC Umbrella trial Sponsor/Partner Roche/Lisata co-funding trial; Roche operationalizing trial Roche supplying atezolizumab Objective Evaluate safety and effectiveness of LSTA1 in combination with gemcitabine and nab- paclitaxel ± atezolizumab in mPDAC Design Phase 1b/
Slide 50: MORPHEUS trial design
MORPHEUS: Phase 1b/2 mPDAC Umbrella trial Eligibility • Patients with mPDAC • No prior systemic therapy for PDAC Gemcitabine + Nab-paclitaxel + LSTA1 + atezolizumab1:1 R Gemcitabine + Nab-paclitaxel + LSTA1 Biopsy Biopsy Screening Stage 1 Stage 2 Preliminary Phase Expansion Phase Disease Progression
Slide 51: iLSTA: Phase 1b/2a trial in locally advanced PDAC
iLSTA: Phase 1b/2a trial in locally advanced PDAC with chemo & IO Sponsor/Partner WARPNINE, Inc. (registered charity in Australia) is funding trial Lisata providing study drug Objective Evaluate safety and therapeutic effect of LSTA1 in combination with IO & Chemo in locally advanced PDAC; det
Slide 52: iLSTA trial design
iLSTA: Phase 1b/2a trial in locally advanced PDAC with chemo & IO Cohort 2 Gemcitabine + nab-paclitaxel + LSTA1 N=5 Cohort 1 Gemcitabine + nab-paclitaxel N=5 Cohort 3 Durvalumab + Gemcitabine + nab-paclitaxel + LSTA1 N=10-20 12 weeks 8 weeks tumor burden assessments until 24 months or recurrence 12
Slide 53: iGoLSTA: Phase 1b/2a trial in locally advanced GEAC
Sponsor/Partner WARPNINE, Inc. (registered charity in Australia) is funding trial Lisata providing study drug Objective Evaluate safety and therapeutic effect of LSTA1 in combination with IO & Chemo in locally advanced GE AdenoCa; determine if inoperable tumors can become operable Design Pha
Slide 54: iGoLSTA trial design
Cohort 2 FOLFIRINOX + LSTA1 N=5 Cohort 1 FOLFIRINOX N=5 Cohort 3 Nivolumab + FOLFIRINOX + LSTA1 N=10-20 12 weeks 8 weeks tumor burden assessments until 24 months or recurrence 12 month Follow-up Primary Endpoint 24 months Follow-up completion Biopsy Screening Biopsy Week 12 Tumor burden assessment a
Slide 55: Phase 2a trial of LSTA1 with SOC in first-line GBM
Sponsor/Partner Tartu University Hospital (Investigator initiated trial in Estonia) Lisata providing study drug and funding trial Objective Evaluate safety, tolerability, and therapeutic effect of LSTA1 in combination with standard- of-care (temozolomide) in patients with previously untreated
Slide 56: GBM trial design
Days 1, 2, 3, 4, 5 and every 28 days for 6 cycles Disease Progression Response rates Safety Temodar® + LSTA1Confirm Eligibility Informed Consent 1:1 Survival Analysis 72-hour Run-in without SoC Newly Diagnosed GBM R Temodar® + LSTA1 matching placebo N=20 N=20 Phase 2a proof-of-concept double-blind,
Slide 57: Phase 1b/2a open-label trial in mPDAC in Japan
Sponsor/Partner Tsukuba Clinical Research & Development Organization (T-CReDO) Lisata providing study drug; AMED Grant funding trial in Japan Objective Evaluate safety, pharmacokinetics and preliminary efficacy of various doses of LSTA1 added to SoC in Japanese patients with mPDAC Design Pha
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