UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
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Item 7.01. Regulation FD Disclosure.
A copy of a slide presentation that Lisata Therapeutics, Inc. (the “Company”) will use at investor and industry conferences and presentations is attached to this Current Report as Exhibit 99.1 and is incorporated herein solely for purposes of this Item 7.01 disclosure.
The information in this Item 7.01, including Exhibit 99.1 attached hereto, is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, or otherwise subject to the liabilities of that Section. The information in this Item 7.01, including Exhibit 99.1 attached hereto, shall not be incorporated by reference into any registration statement or other document pursuant to the Securities Act of 1933, except as otherwise expressly stated in such filing.
Item 8.01 Other Events.
As previously disclosed, in December 2020, the Company commenced enrollment in its Phase 2b FREEDOM Trial of XOWNA®, a double-blind, randomized, placebo-controlled clinical trial designed to further evaluate the efficacy and safety of intracoronary artery delivery of autologous CD34+ cells in subjects with Coronary Microvascular Dysfunction (CMD) and without obstructive coronary artery disease and was expected to complete enrollment in approximately 12 months. While early enrollment proceeded to plan with the first patient treated in January 2021, the COVID-19 pandemic resulted in insurmountable enrollment rate challenges and population heterogenicity. As a result, in May 2022, the Company announced that enrollment in the FREEDOM Trial had been suspended and that it intended to conduct an interim analysis of the data from not less than the first 20 patients enrolled using the 6-month follow-up data to evaluate the efficacy and safety of XOWNA® in subjects with CMD. Following the analysis of results of the FREEDOM Trial subjects completing 6-month follow-up along with Key Opinion Leaders’ input, the Company’s board of directors determined that execution of a redesigned FREEDOM-like trial would be the appropriate next step, but the cost of such a trial would be prohibitively expensive to undergo alone without a strategic partner. Accordingly, the Company’s board of directors concluded that XOWNA® development will only be continued if a strategic partner that can contribute the necessary capital for a redesigned trial is identified and secured. There can be no assurance that we will be able to identify such a partner and enter into an agreement with such partner on acceptable terms or at all.
Item 9.01 Exhibits.
| Exhibit No. | Description | ||||
| Lisata Therapeutics, Inc. Corporate Presentation, September 22, 2022 | |||||
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
CALADRIUS BIOSCIENCES, INC.
By: /s/ David J. Mazzo
Name: David J. Mazzo, PhD
Title: Chief Executive Officer
Dated: September 22, 2022
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
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 this communication regarding strategy, future operations, future financial position, future revenue, projected expenses, prospects, plans and objectives of management are forward-looking statements. In addition, when or if used in this communication, the words “may,” “could,” “should,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” “plan,” “predict” and similar expressions and their variants, as they relate to Lisata or its management, may identify forward-looking statements. Examples of forward-looking statements include, but are not limited to, statements relating to the long-term success of Lisata’s recently completed merger (the “Merger”) with Cend Therapeutics, Inc. (“Cend”), including the ongoing integration of Cend’s operations; Lisata’s continued listing on the Nasdaq Capital Market; expectations regarding the capitalization, resources and ownership structure of Lisata; the approach Lisata is taking to discover and develop novel therapeutics; the adequacy of Lisata’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; and the difficulty in predicting the time and cost of development of Lisata’s product candidates. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ongoing COVID-19 pandemic on Lisata’s business, the safety and efficacy of Lisata’s product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in Lisata’s clinical programs, Lisata’s ability to finance its operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of Lisata’s scientific studies, Lisata’s ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in Lisata’s markets, the ability of Lisata to protect its intellectual property rights; unexpected costs, charges or expenses resulting from the Merger; potential adverse reactions or changes to business relationships resulting from the completion of the Merger; potential underperformance of Lisata’s business following the Merger as compared to management’s initial expectations; and legislative, regulatory, political and economic developments. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in Lisata’s Annual Report on Form 10-K filed with the SEC on March 22, 2022, and in the proxy statement/prospectus filed by Lisata with the Securities and Exchange Commission relating to the Merger. Except as required by applicable law, Lisata undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. 2
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 potential value creating data and business development events projected in the next 12-24 months Seasoned management with domain expertise along with big pharma and emerging pharma experience Nasdaq-listed with a focused mid-late-stage clinical development pipeline and a promising preclinical platform │ NOVEL INTRATUMORAL DELIVERY TECHNOLOGY TO IMPROVE THERAPEUTIC EFFICACY OF SoC* DRUGS │ │ EXISTING CAPITAL EXPECTED TO FUND ANTICIPATED MILESTONES │ EXISTING STRATEGIC PARTNERSHIPS │ 33 *SoC = standard-of-care
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 tethering Selectively depletes intratumoral immunosuppressive cells Combination with many existing chemo- and immuno-therapeutics possible in a variety of indications Tumor-Penetrating Nanocomplex (TPN) Platform™ - broad potential for delivery of nucleic acid-based therapies Designed to address challenges to ASO and siRNA delivery posed by stroma barrier and endosome sequestration Clinical development candidate identification expected in 2023 CD34+ Cell Therapy Platform - designed to address diseases and conditions caused by ischemia CD34+ cells repeatedly demonstrated vascular repair in multiple organs and have been clinically studied in a variety of ischemic diseases by numerous investigators across many sites and countries • Consistent results of rigorous clinical studies comprising >1,000 patients published in peer reviewed journals1-4 • Single treatments elicited durable therapeutic effects • Treatment generally well-tolerated 1 Povsic, T. et al. JACC Cardiovasc Interv, 2016, 9 (15) 1576-1585 2 Losordo, D.W. et al. Circ Cardiovasc Interv, 2012; 5:821–830 3 Velagapudi P, et al, Cardiovas Revasc Med, 2018, 20(3):215-219 4 Henry T.D., et al, European Heart Jour 2018, 2208–2216 4
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) chemotherapy Basket trial initiation planned in 2023 expanding development to other solid tumors and additional anti-cancer drug combinations, including immunotherapies Granted Fast Track as well as Orphan Drug Designation by the U.S. FDA in PDAC CD34+ autologous cell therapy development programs advancing to next development milestone XOWNA® development will continue if a partner is identified that can contribute the necessary capital HONEDRA® (SAKIGAKE designated) advancing through Japanese regulatory process toward JNDA CLBS201 proof-of-concept (PoC) results expected in 1Q23 No additional capital outlay necessary to reach identified milestones 5
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 million to Lisata for milestones and tiered double-digit royalties on potential sales $10 million payment due to Lisata for proceeding to Phase 3 in mPDAC in China Clinical development collaboration with Roche in mPDAC LSTA1 tested in combination with atezolizumab in mPDAC as part of Morpheus trial 6 Additional partnership opportunities for broad applications of LSTA1 and the CendR Platform™ Ongoing discussions support goal to partner CD34+ programs 6
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 Qilu (China) Gemcitabine/nab-paclitaxel ± LSTA1 Roche/Lisata (Multi-national) LSTA1 + nab-paclitaxel + gemcitabine ± atezolizumab KUCC - IIT (U.S.) LSTA1 + FOLFIRINOX + panitumumab* Pancreatic, Colon and Appendiceal Cancers Lisata (U.S.) LSTA1 in combination with SoC Various Solid Tumors Lisata (U.S.) TPN development candidate CD34+ Platform Programs Lisata (U.S.) XOWNA® (LSTA16) Coronary Microvascular Dysfunction Lisata (Japan) HONEDRA® (LSTA12) Critical Limb Ischemia and Buerger’s Disease Lisata (U.S.) LSTA201 Diabetic Kidney Disease FDA feedback 4Q22 Trial initiation planned 1Q/2Q23 Enrollment completion target 4Q23 Data expected 2024 Preliminary data expected 2H23 Trial initiation target 1Q23 Enrollment completion target 4Q23 Data expected 2024 Trial initiation planned 1Q/2Q23 Development candidate ID target 2023 Phase 1 planned for 2024 Partner sought to advance development PMDA consultation underway Data expected 1Q23 Phase 2/3 adaptive Phase 2b (ASCEND) Phase 1b/2 Phase 1b/2 Phase 1b/2 (CENDIFOX) Phase 2a Phase 2b (FREEDOM) Phase 2 Phase 1b – PoC Robust portfolio of development candidates 7 *Panitumumab may be added for colorectal or appendiceal patients without Ras mutation 7 Preclinical
LSTA1 (formerly known as CEND-1) CendR Platform™
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 on: • Tumor vascular endothelium • Cancer-associated fibroblasts, a major component of tumor stroma • Intratumoral immunosuppressive cells Once bound to αvβ3/β5 integrins, LSTA1 is cleaved by proteases that are up-regulated in tumors, releasing a C-end Rule (CendR) linear peptide fragment 9
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 and co-administered/tethered drugs penetrate the stroma and tumor, providing greater access to their target(s) • Activating the CendR pathway opens intratumoral gap junctions enhancing extravasation of immune cells into tumors 10
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 by etching solution • FQDs show selective tumor penetration in the presence of LSTA1 +etch* +etch* +LSTA1 FQD alone following etching solution to quench fluorescence in circulation 11
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. co-admin) Pancreatic ductal adenocarcinoma KPC mice genetically engineered to develop PDAC CEND-1 + gemcitabine (i.v. co-admin) Increased tumor penetration enhances activity across treatment modalities 12 Breast cancer + antibody (Herceptin®)
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 cases of cancer will be diagnosed in 2022 In the U.S. alone, over 90% of new cancer cases are solid tumors An estimated 609,360 people will die from cancer in 2022, corresponding to ~1,700 deaths per day 13 Pancreatic cancer is one of the deadliest cancers in the U.S. with a five-year survival rate of only 11%, representing a high unmet medical need
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 Favorable LSTA1 pharmacokinetic profile with median T1/2 ~2 hours Unprecedented improvement of SoC anti-tumor activity 1,2 • Overall Response Rate (PR+CR=ORR) 59% (vs. 23%) including Complete Response • Disease Control Rate at 16 weeks 79.3% (vs. 48%) • CA19-9 circulating tumor biomarker reductions in 96% of patients (vs. 61%) • Median Progression-Free Survival 9.7 months (vs. 5.5 months) • Median Overall Survival 13.2 months (vs. 8.5 months) 1 Dean A, et al., The Lancet Gastroenterology & Hepatology, 2022. 2 Von Hoff D, et al., New England Journal of Medicine, 2013. 14
Ongoing & Planned LSTA1 Clinical Trials
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 125 subjects (~40 sites in Australia and New Zealand) Endpoints Primary: Progression Free Survival Secondary: AEs, SAEs, Overall Survival, Objective Tumor Response Rate Control/Comparator SoC chemotherapy (gemcitabine/nab-paclitaxel) with LSTA1 or placebo Objective Evaluate the effect of adding LSTA1, compared to placebo, to SoC chemotherapy in patients with untreated mPDAC Timing Enrollment completion target 4Q23 Data expected 2024 1616
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, Disease Control Rate, Overall Survival, and Progression Free Survival Secondary: Pharmacokinetic parameters Control/Comparator SoC chemotherapy (gemcitabine/Qilu-produced nab-paclitaxel) with and without LSTA1 Objective Evaluate safety, pharmacokinetics and preliminary efficacy of LSTA1 added to SoC in Chinese patients with mPDAC Timing Preliminary data expected 1H23; full data expected 2024 1717
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-free Survival, Overall Response Rate, RO Resection Rate, Pathological Response Rate Control/Comparator SoC chemotherapy (neoadjuvant FOLFIRINOX-based therapies with LSTA1 or placebo Objective Evaluate the safety of LSTA1 in combination with neoadjuvant FOLFIRINOX-based therapies for the treatment of pancreatic, colon, and appendiceal cancers Timing Enrollment completion target 4Q23 Data expected 2024 1818
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-controlled trial in multiple advanced solid tumor types (U.S.) Study Size N=389 N=120 (depending on number of arms in the "basket") Endpoints Primary: OS Secondary: Safety, ORR, PFS Primary: OS Secondary: Safety, ORR, PFS Control/Comparator Placebo; in combination with SoC chemo (gem/nab-paclitaxel) Placebo; in combination with tumor-type specific SoC chemo Objective Evaluate the efficacy and safety of LSTA1 in subjects with previously untreated mPDAC (next step in development toward U.S. registration) Evaluate the preliminary efficacy, safety and tolerability of LSTA1 in combination with standards of care in subjects with advanced solid tumors Timing FDA feedback: 4Q22 Trial initiation target: 1Q/2Q23 Trial initiation target: 1Q/2Q23 19
Tissue- Penetrating Nanoparticle (TPN) Platform™
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 (siRNA) anticancer programs failed to translate preclinical efficacy to clinical success • >95% of ASO and siRNA drugs sequestered in endosomes • Tumor stroma serves as primary impediment to effective delivery • High doses to drive intratumoral concentration resulted in on- and off-target side effects, including, but not limited to, clotting factors and renal toxicities Passive targeting (i.e., lipid nanoparticles) appears ineffective Non-targeted cell-/tissue-penetrating moieties can disrupt unintended tissues Moieties to target tumor increase bulk and may exacerbate problem of transiting stroma Targeted approach to transit tumor stroma may enable effective solid tumor treatment 21 DELIVERY ISSUES LIMIT ANTICANCER APPLICATIONS OF RNA-BASED THERAPEUTICS
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 moiety 22 Peptides provide tumor and/or immune cell targeting Unique CendR pathway activation to penetrate stroma and deliver efficacious drug concentrations to all layers of solid tumors Technologies to evade endosome sequestration Targeted tissue penetration drives dose- and toxicity-sparing potency Ease of synthesis vs. biologics such as virus-like particles, Ab-conjugates or exosomes
CD34+ Cell Therapy Platform Technology
XOWNA® [LSTA16 (formerly known as CLBS16)] Coronary Microvascular Dysfunction (USA)
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 by stents/bypass; responds poorly or not at all to available pharmacotherapies • U.S. CMD population potentially treatable by XOWNA® ranges from ~415,000 to ~1.6 million patients1 • Compelling Phase 2a (published ESCaPE-CMD trial) results show the potential of XOWNA® to significantly improve symptoms of CMD • Phase 2b (FREEDOM) trial impacted directly and indirectly by COVID pandemic resulted in insurmountable enrollment rate challenges and population heterogenicity; trial enrollment suspended in May 2022 after ~1/3 of the intended subjects enrolled Summary Analysis of results of FREEDOM Trial subjects completing 6-month follow-up along with KOL input suggests that execution of a redesigned FREEDOM-like trial is an appropriate next step • Cost of such trial is financially challenging in a “go-it-alone” strategy XOWNA® development will continue if a partner is identified that can contribute the necessary capital Next Steps
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
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 (orphan population) Patients with no-option CLI have persistent symptoms even after bypass surgery, angioplasty, stenting and available pharmacotherapy CLI has been categorized as Rutherford Classification Stages1 • Stages: 1-3 (mild to severe claudication); 4 (rest pain); 5 (minor tissue loss); 6 (major tissue loss) • CLI patients are at high risk of amputation and death with increasing Rutherford score Multi-million-dollar opportunity with an increasing prevalence of arteriosclerosis obliterans (ASO) and CLI in Japan Positive previously published Phase 2 results in Japan3,4 2727 1 Reinecke H., European Heart Journal, 2015 Apr 14;36(15):932-8 3 Kinoshita et al, Atherosclerosis 224 (2012) 440-445 4 Losordo, D.W. et al, Circulation 2012; 5(6):821-830
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 Up to 106 cells/kg of HONEDRA® (LSTA12) Control/Comparator SoC: wound care plus drugs approved in Japan • Including antimicrobials, antiplatelets, anticoagulants and vasodilators Mode of Administration Intramuscular, 20 injections in affected lower limb in a single treatment Objective Demonstrate a trend toward efficacy and acceptable safety to qualify for consideration of early conditional approval under Japan’s Regenerative Medicine Development Guidelines 2828
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 Devices Agency (PDMA) is underway in support of the planned filing of a Japan New Drug Application 2929
LSTA201 (formerly known as CLBS201) Diabetic Kidney Disease
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 kidney function CD34+ cells are promoters of new capillary growth, improving the microvasculature Therapies currently available and/or expected to be available over the next 5–10 years will slow the progression of CKD/DKD A regenerative DKD therapy (i.e., one that reverses disease course) could represent a medical and pharmacoeconomic breakthrough Development Rationale To demonstrate that CD34+ cell mobilization, donation, and administration can be tolerated by patients with CKD and type 2 diabetes To demonstrate that regeneration of the kidney microcirculation using CD34+ cell therapy improves kidney function Clinical Strategy 1 2020 Dallas Nephrology Associates. 2 Chade AR. (2017) Small Vessels, Big Role: Renal Microcirculation and Progression of Renal Injury. Hypertension; 69(4):551-563. 3 Zuk, Anna & Bonventre, Joseph. (2016). Annual Review of Medicine. 67. 293-307. 10.1146/annurev-med-050214-013407. 3131
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 - unilateral inj., 1 sentinel - bilateral inj., 4 bilateral inj. patients) Dose 1 x 106 – 300 x 106 cells administered as a one-time infusion Patient Population Stage 3b DKD Design Open-label, proof-of-concept Phase 1b study Mode of Administration Intra-arterial injection into one or both renal arteries Timing Top-line data target for all subjects: 1Q23 3232
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 Ph1b/2 study (Roche; multi-national) of LSTA1 + atezolizumab in mPDAC target initiation 4Q22 Ph1b/2 study (Qilu; China) of LSTA1 in mPDAC preliminary data expected 1H23 Ph2a Basket study of LSTA1 in solid tumors target initiation 1Q/2Q23 TPN development candidate 2023 Ph2/3 adaptive study of LSTA1 in mPDAC target initiation 1Q/2Q23 Ph1b/2 study (Qilu; China) of LSTA1 in mPDAC final data expected 2024 Ph1b/2 study (CENDIFOX; U.S.) of LSTA1 enrollment completion target 4Q23 Ph2b study (ASCEND; Australia) of LSTA1 enrollment completion target 2024 Ph2b study (ASCEND; Australia) of LSTA1 data expected 2024 Ph1b/2 study (CENDIFOX; U.S.) of LSTA1 data expected 2024
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 potential value creating data and business development events projected in the next 12-24 months Seasoned management with domain expertise along with big pharma and emerging pharma experience Nasdaq-listed with a focused mid-late-stage clinical development pipeline and a promising preclinical platform │ NOVEL INTRATUMORAL DELIVERY TECHNOLOGY TO IMPROVE THERAPEUTIC EFFICACY OF SoC* DRUGS │ │ EXISTING CAPITAL EXPECTED TO FUND ANTICIPATED MILESTONES │ EXISTING STRATEGIC PARTNERSHIPS │ 3434 *SoC = standard-of-care
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