Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — Investor Presentation (March 2023)
51 slides from Lisata Therapeutics's investor presentation, Mar 30, 2023
Slide 1: Targeted Therapy Delivered – Corporate Presentation
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 . Targeted Therapy Delivered David J. Mazzo, Ph.D. Chief Executive Officer Corporate Presentation | March 30, 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 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 potential va
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: tumor targeting & intratumoral penetration are inadequate
Tumor targeting and intratumoral penetration are inadequate • Tumor stroma acts as an effective barrier to anti-cancer agent penetration • Tumor microenvironment immunosuppressive cells can lead to tumor resistance and/or metastases • Continued or escalating dosing of non-targeted anti-cancer ther
Slide 8: Solution: Lisata's CendR Platform technology
Solution: Lisata’s CendR Platform® technology 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 th
Slide 9: LSTA1: lead clinical development candidate of the CendR Platform
LSTA1: lead clinical development candidate of the CendR Platform® 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 Desi
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: Well-characterized Mechanism of Action with Compelling 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: Steps 1 & 2
αvβ3/β5 integrin LSTA1 (Furin & others) Protease Cleavage CendR 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 upregulated on: • Tumor vascular endothelium • Tumor cells Once
Slide 13: LSTA1 Mechanism of Action: Step 3
LSTA1 Mechanism of Action: Step 3 Neuropilin-1 CendR Peptide Fragment Co-administered or tethered anti-cancer drugs CendR Transport Pathway Tumor Vascular Endothelial Cell Cell Nucleus Gap junction opening 13 The CendR fragment then binds with high affinity and selectivity to an adjacent receptor,
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 treatment 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 Clinical Development Portfolio Fast Track and Orphan Drug designated (PDAC) - USA
Slide 18: LSTA1 clinical development portfolio (1 of 2)
Development Partner(s) [Development Venue] Indication and Trial Product/Comparator Stage of Development (Status) Lisata/AGITG [Australia/New Zealand/Ireland] First-line mPDAC; Gemcitabine/nab-paclitaxel with LSTA1 or placebo Phase 2b (ASCEND) (Enrolling) Lisata [United States] Various Solid Tumors;
Slide 19: LSTA1 clinical development portfolio (2 of 2)
Development Partner(s) [Development Venue] Indication and Trial Product/Comparator Stage of Development (Status) Tartu University/Lisata [Estonia] First-line Glioblastoma Multiforme; Temozolomide ± LSTA1 Phase 2a (Pending Initiation) UCSD/Columbia University/Lisata [United States] Peritoneal Carcino
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 CD34+ cell therapy current clinical trials Legacy development programs provide potential value upside with
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 clinical 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: A wealth of anticipated key milestones
2023 2024 ASCEND [AUS, NZ, IRE] First-line mPDAC BOLSTER Trial [USA] Various Solid Tumors (Basket Trial) CENDIFOX [USA] Pancreatic, Colon & Appendiceal cancers MORPHEUS [Multi-national] First-line mPDAC Qilu: Phase 1b/2a Trial [CHN] First-line mPDAC Qilu: Phase 2 Trial [CHN] First-line mPDAC iLSTA [
Slide 29: Lisata Therapeutics: financial summary
Lisata Therapeutics: financial summary $69.2M Cash & Investments (12/31/2022) $0 Debt 1H2025 (Funding through key development milestones) Projected Cash Runway Through Common Shares Outstanding (12/31/2022): 7.9 million shares Options Outstanding (12/31/2022): Exercise Price: $0.02 - $4.22 = 1,127,0
Slide 30: Investment rationale
Investment rationale 30 * As of 12/31/2022 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 potential v
Slide 31: 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 . Targeted Therapy Delivered Investor Relations Contact: John D. Menditto VP, IR & Corporate Communications o: (908) 842-0084 | e: jmenditto@lisata.com Nasdaq: LSTA | www.lisata.com
Slide 32: Zz o = ® 2. 2. «a,
Zz o = ® 2. 2. «a,
Slide 33: Development partners, trials and strategic rationale (1 of 3)
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 34: Development partners, trials and strategic rationale (2 of 3)
Development Partner(s) [Development Venue] Indication and Trial Product/Comparator Stage of Development Strategic Rationale Tartu University/Lisata [Estonia] First-line Glioblastoma Multiforme; Temozolomide ± LSTA1 Phase 2a Assess LSTA1 safety and effectiveness in additional tumor type (GBM) a in pl
Slide 35: Development partners, trials and strategic rationale (3 of 3)
3535 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 36: ASCEND: Phase 2b, blinded, 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 37: 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 38: 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 39: Phase 1b/2a open-label trial in mPDAC in China: 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 40: 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 41: 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 42: BOLSTER: Phase 2 blinded, randomized 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 43: Basket: Phase 2 blinded, randomized PoC 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 44: Phase 2 blinded, placebo-controlled 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 45: Phase 2 trial in mPDAC in China: 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 46: iLSTA: Phase 1b/2a trial in locally advanced PDAC with chemo & IO
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 47: 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 48: 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 49: 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 50: 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 51: Phase 2a 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,
Lisata Therapeutics — Investor Presentation (March 2023) on 6ix