UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 OR 15(d) of The Securities Exchange Act of 1934
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
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Item 2.02 Results of Operations and Financial Condition.
The information in Item 7.01 is incorporated by reference.
Item 7.01 Regulation FD Disclosure.
On August 12, 2024, Lisata Therapeutics, Inc. (the "Company") issued a press release in connection with its financial results for the second quarter ended June 30, 2024. A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated into this Item 7.01 by reference.
A copy of a slide presentation that the Company will use at investor and industry conferences and presentations is attached to this Current Report as Exhibit 99.2 and is incorporated herein solely for purposes of this Item 7.01 disclosure.
The information in this Item 7.01, including Exhibits 99.1 and 99.2 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 Exhibits 99.1 and 99.2 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 9.01. Financial Statement and Exhibits.
| Exhibit No. | Description | ||||
| Press Release, dated August 12, 2024 | |||||
| Lisata Therapeutics, Inc. Corporate Presentation, August 12, 2024 | |||||
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.
LISATA THERAPEUTICS, INC.
By: /s/ David J. Mazzo
Name: David J. Mazzo, PhD
Title: President & Chief Executive Officer
Dated: August 12, 2024
Exhibit 99.1
Lisata Therapeutics Reports Second Quarter 2024 Financial Results and Provides Business Update
Phase 2b ASCEND trial top-line data remains on track to be reported in fourth quarter of 2024
Available cash projected to fund current operations into early 2026 and all active studies through to data
Conference call scheduled for today at 4:30 p.m. Eastern Time
BASKING RIDGE, NJ (August 12, 2024) – Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, provided a business update and reported financial results for the second quarter ended June 30, 2024.
“The second quarter generated strong momentum for Lisata as we continued to advance multiple ongoing and planned clinical studies centered around our novel investigational product, certepetide,” stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Lisata. “We have a lot to look forward to with multiple key data readouts projected over the next 18 months, including topline results from the Phase 2b ASCEND trial. These results have transformative potential for the Company as we plan to explore conditional approvals with various regulatory agencies and/or to design an optimized Phase 3 program in metastatic pancreatic ductal adenocarcinoma, an aggressive, often fatal, form of pancreatic cancer. In just the first half of 2024, certepetide has received U.S. FDA Orphan Drug and Rare Pediatric Disease designation in osteosarcoma, and a waiver for evaluating certepetide in a pediatric population with pancreatic cancer in Europe (EMA). These agency recognitions further validate and support our excitement and the broad therapeutic potential of this innovative therapy.”
Dr. Mazzo added, “Our continued prudent, strategic financial management allows us to reaffirm our projection that available cash will fund current operations into early 2026, providing the necessary capital for all planned trials through to completion.”
Development Portfolio Highlights
Certepetide as a treatment for solid tumors in combination with other anti-cancer agents
Certepetide (formerly LSTA1) is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to penetrate solid tumors more effectively. Certepetide actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor. Certepetide has also been shown to modify the tumor microenvironment, diminishing its immunosuppressive nature and inhibiting the metastatic cascade. Along with our collaborators, we have amassed significant non-clinical data demonstrating enhanced delivery of various existing and emerging anti-cancer therapies, including immunotherapies and RNA-based therapeutics. To date, certepetide has also demonstrated favorable safety, tolerability, and clinical activity in completed and ongoing clinical trials designed to test its ability to enhance the effectiveness of standard-of-care (“SoC”) chemotherapy for pancreatic cancer. Lisata is exploring the potential of certepetide to enable a variety of treatment modalities to treat a range of solid tumors more effectively. Certepetide has been awarded Fast Track designation (U.S.) and Orphan Drug Designation for pancreatic cancer (U.S. and E.U.) as well as Orphan Drug Designation for glioma (U.S.) and osteosarcoma (U.S.). Additionally, certepetide has received Rare Pediatric Disease Designation for osteosarcoma (U.S.). Currently, certepetide is the subject of multiple ongoing or planned Phase 2a and 2b clinical studies being conducted globally in a variety of solid tumor types in combination with a variety of anti-cancer regimens, including:
•ASCEND: Phase 2b double-blind, randomized, placebo-controlled clinical trial evaluating two dosing regimens of certepetide in combination with SoC chemotherapy (gemcitabine/nab-paclitaxel) in patients with metastatic pancreatic ductal adenocarcinoma (“mPDAC”). Cohort A of the study receives a single dose of 3.2
mg/kg certepetide essentially simultaneously with SoC, while Cohort B is identical to Cohort A, but with a second dose of 3.2mg/kg of certepetide given four hours after the first. The trial is being conducted at 25 sites in Australia and New Zealand led by the Australasian Gastro-Intestinal Trials Group in collaboration with the University of Sydney and with the National Health and Medical Research Council Clinical Trial Centre at the University of Sydney as the Coordinating Centre. The conclusion of a planned interim futility analysis in 2023 by the Independent Data Safety Monitoring Committee was that the conditions for futility were not met and that the study should proceed to completion. With trial enrollment completed in the fourth quarter of 2023, Lisata expects topline data from the 95 patients assigned to Cohort A of the study to be reported in the fourth quarter of 2024 and the complete data set of all 158 patients from the study to be available by mid-2025.
•BOLSTER: Phase 2a double-blind, placebo-controlled, multi-center, randomized trial in the U.S. evaluating certepetide in combination with SoC in first- and second-line cholangiocarcinoma (“CCA”). The Company achieved complete enrollment in first-line CCA nearly six months ahead of plan, accelerating anticipated topline data readout to mid-2025. Based on this rapid enrollment rate and the pressing need to improve treatment outcomes in patients that have progressed after first-line CCA treatment, a second cohort has been added to the BOLSTER trial evaluating subjects in second-line CCA. Lisata expects to enroll the first patient by the fourth quarter of 2024.
•CENDIFOX: Phase 1b/2a open-label trial in the U.S. of certepetide in combination with neoadjuvant FOLFIRINOX based therapies in pancreatic, colon and appendiceal cancers. The trial has completed enrollment in the pancreatic cohort and expects to complete enrollment in the remaining two cohorts by the end of 2024.
•Qilu Pharmaceutical, the licensee of certepetide in the Greater China territory, is currently evaluating certepetide in combination with gemcitabine and nab-paclitaxel as a treatment for mPDAC. During the 2023 ASCO Annual Meeting, Qilu Pharmaceutical presented an abstract sharing preliminary data from the study which corroborated previously reported findings from the Phase 1b/2a trial of certepetide plus gemcitabine and nab-paclitaxel conducted in Australia in patients with mPDAC. As previously reported, Qilu has begun treating patients in their Phase 2 placebo-controlled trial in mPDAC.
•iLSTA: Phase 1b/2a randomized, single-blind, single-center, safety and pharmacodynamic trial in Australia evaluating certepetide in combination with the checkpoint inhibitor, durvalumab, plus SoC gemcitabine and nab-paclitaxel chemotherapy versus SoC alone in patients with locally advanced non-resectable PDAC. Enrollment completion is expected in the second half of 2024.
•A Lisata-funded Phase 2a, double-blind, placebo-controlled, randomized, proof-of-concept study evaluating certepetide in combination with SoC temozolomide versus temozolomide alone in patients with newly diagnosed GBM is being conducted across multiple sites in Estonia and Latvia and is targeted to enroll 30 patients with a randomization of 2:1 in favor of the certepetide treatment group.
•FORTIFIDE: Phase 1b/2a, double-blind, placebo-controlled, three-arm, randomized study in the U.S. to evaluate the safety, tolerability, and efficacy of a 4-hour continuous infusion of certepetide in combination with SoC in subjects with second-line mPDAC who have progressed on FOLFIRINOX. As part of this study, Lisata has engaged Haystack Oncology to use its MRD™ technology to measure circulating tumor DNA levels at multiple timepoints in patients throughout the study as an exploratory endpoint for analyzing the early therapeutic effect of certepetide. The Company expects to enroll the first patient in the study by the first half of 2025.
Second Quarter 2024 Financial Highlights
For the three months ended June 30, 2024, operating expenses totaled $5.5 million, compared to $6.9 million for the three months ended June 30, 2023, representing a decrease of $1.4 million or 19.7%.
Research and development expenses were approximately $2.6 million for the three months ended June 30, 2024, compared to $3.2 million for the three months ended June 30, 2023, representing a decrease of $0.6 million or 17.7%. This was primarily due to a reduction in expenses associated with the Phase 2b ASCEND trial which completed enrollment in the prior year, lower spend on chemistry, manufacturing and control (“CMC”) related expenses and
lower equity expense partially offset by an increase in expenses associated with our enrollment activities in the current year for our BOLSTER trial.
General and administrative expenses were approximately $2.9 million for the three months ended June 30, 2024, compared to $3.7 million for the three months ended June 30, 2023, representing a decrease of $0.8 million or 21.3%. This was primarily due to one-off related severance costs in the prior year associated with the elimination of the Chief Business Officer position on May 1, 2023, a reduction in equity expense and a decrease in directors and officers insurance premiums in the current year.
Benefit from income taxes was $0.0 million for the three months ended June 30, 2024, compared to $2.3 million for the three months ended June 30, 2023. In April 2023, we received net proceeds of $2.2 million from the sale of tax benefits to a qualified and approved buyer pursuant to the New Jersey Economic Development Authority’s Technology Business Tax Certificate Transfer Program.
Overall, net losses were $5.0 million for the three months ended June 30, 2024, compared to $4.0 million for the three months ended June 30, 2023.
Balance Sheet Highlights
As of June 30, 2024, Lisata had cash, cash equivalents, and marketable securities of approximately $38.3 million. Based on its current expected capital needs, the Company believes that its projected capital will fund its current proposed operations into early 2026, encompassing anticipated data milestones from all its ongoing and planned clinical trials.
Conference Call Information
Lisata will hold a live conference call today, August 12, 2024, at 4:30 p.m. Eastern Time to discuss financial results, provide a business update and answer questions.
Those wishing to participate must register for the conference call by way of the following link: CLICK HERE TO REGISTER. Registered participants will receive an email containing conference call details with dial-in options. To avoid delays, we encourage participants to dial into the conference call 15 minutes ahead of the scheduled start time.
A live webcast of the call will also be accessible under the Investors & News section of Lisata’s website and will be available for replay beginning two hours after the conclusion of the call for 12 months.
About Lisata Therapeutics
Lisata Therapeutics is a clinical-stage pharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for the treatment of advanced solid tumors and other major diseases. Lisata’s product candidate, certepetide (formerly LSTA1), is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to selectively target and penetrate solid tumors more effectively. Lisata has already established noteworthy commercial and R&D partnerships based on its CendR Platform® technology. The Company expects to announce numerous milestones over the next two years and believes that its projected capital will fund operations into early 2026, encompassing anticipated data milestones from its ongoing and planned clinical trials. For more information on the Company, please visit www.lisata.com.
Forward-Looking Statements
This communication 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 the Company’s clinical development programs 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, the potential efficacy of certepetide as a treatment for patients with metastatic pancreatic ductal adenocarcinoma and other solid tumors; statements relating to 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: results observed from a single patient case study are not necessarily indicative of final results and one or more of the clinical outcomes may materially change following more comprehensive reviews of the data and as more patient data becomes available, including the risk that unconfirmed responses may not ultimately result in confirmed responses to treatment after follow-up evaluations; the risk that product candidates that appeared promising in early research and clinical trials do not demonstrate safety and/or efficacy in larger-scale or later clinical trials; 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; 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 February 29, 2024, and in other documents filed by Lisata with the Securities and Exchange Commission. 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.
Contact:
Investors:
Lisata Therapeutics, Inc.
John Menditto
Vice President, Investor Relations and Corporate Communications
Phone: 908-842-0084
Email: jmenditto@lisata.com
Media:
ICR Westwicke
Elizabeth Coleman
Senior Associate
Phone: 203-682-4783
Email: elizabeth.coleman@westwicke.com
- Tables to Follow -
Lisata Therapeutics, Inc.
Selected Financial Data
(in thousands, except per share data)
Selected Financial Data
(in thousands, except per share data)
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||||||||||||
| (unaudited) | (unaudited) | (unaudited) | (unaudited) | |||||||||||||||||||||||
| Statement of Operations Data: | ||||||||||||||||||||||||||
| Research and development | $ | 2,601 | $ | 3,162 | $ | 5,842 | $ | 6,341 | ||||||||||||||||||
| General and administrative | 2,922 | 3,713 | 6,282 | 7,378 | ||||||||||||||||||||||
| Total operating expenses | 5,523 | 6,875 | 12,124 | 13,719 | ||||||||||||||||||||||
| Operating loss | (5,523) | (6,875) | (12,124) | (13,719) | ||||||||||||||||||||||
| Investment income, net | 493 | 668 | 1,082 | 1,338 | ||||||||||||||||||||||
| Other expense, net | (14) | (150) | (201) | (163) | ||||||||||||||||||||||
| Net loss before benefit from income taxes and noncontrolling interests | (5,044) | (6,357) | (11,243) | (12,544) | ||||||||||||||||||||||
| Benefit from income taxes | — | (2,330) | (798) | (2,330) | ||||||||||||||||||||||
| Net loss | (5,044) | (4,027) | (10,445) | (10,214) | ||||||||||||||||||||||
| Less - net income attributable to noncontrolling interests | — | — | — | — | ||||||||||||||||||||||
| Net loss attributable to Lisata Therapeutics, Inc. common stockholders | $ | (5,044) | $ | (4,027) | $ | (10,445) | $ | (10,214) | ||||||||||||||||||
| Basic and diluted loss per share attributable to Lisata Therapeutics, Inc. common stockholders | $ | (0.61) | $ | (0.50) | $ | (1.26) | $ | (1.28) | ||||||||||||||||||
| Weighted average common shares outstanding | 8,308 | 8,021 | 8,301 | 8,004 | ||||||||||||||||||||||
| June 30, 2024 | December 31, 2023 | |||||||||||||||||||||||||
| (unaudited) | ||||||||||||||||||||||||||
| Balance Sheet Data: | ||||||||||||||||||||||||||
| Cash, cash equivalents and marketable securities | $38,262 | $50,535 | ||||||||||||||||||||||||
| Total assets | 42,571 | 54,694 | ||||||||||||||||||||||||
| Total liabilities | 4,576 | 6,800 | ||||||||||||||||||||||||
| Total equity | 37,995 | 47,894 | ||||||||||||||||||||||||
# # #
© L i s a t a T h e r a p e u t i c s , I n c . 2 0 2 4 . 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. President and Chief Executive Officer www.lisata.com Corporate Presentation | August 12, 2024 Nasdaq: LSTA Exhibit 99.2
2 Forward-looking statements advisory 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”, “target” 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 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, develop and commercialize 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 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 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 February 29, 2024, and in other documents filed by Lisata with the Securities and Exchange Commission. 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. Disclosure
Lisata at a Glance Company Overview 3
Company Overview 4 Lisata Therapeutics (Nasdaq: LSTA) Projected cash runway into early 2026, funding all development programs through data A clinical stage therapeutics company rapidly developing a novel solid tumor targeting and penetration technology with TME* modifying properties to improve the efficacy of anti-cancer drugs Seasoned management with successful international drug development experience and expertise Platform technology “validated” by existing partnerships with potential for many others Multiple product and business milestones projected over the next 18 months Proprietary field- leading technology in underserved global indications *TME = Tumor Microenvironment
Seasoned leadership with proven track record in drug approvals worldwide 5 Kristen K. Buck, MD Executive Vice President of R&D and Chief Medical Officer Dr. Buck is a board certified and licensed physician with >20 years of strategic global drug development, drug/device safety/epidemiology, FDA, and clinical practice experience. Gregory Berkin Chief Information Officer and Data Protection Officer James Nisco SVP of Finance and Treasury and Chief Accounting Officer Tariq Imam VP of BD and Operations and Corporate Counsel John Menditto VP of Investor Relations and Corporate Communications Bill Sietsema, PhD VP of Global Regulatory Affairs Ryan Quick VP of Chemistry, Manufacturing and Controls Detailed bios can be found at www.lisata.com David J. Mazzo, PhD President and Chief Executive Officer, Member of the Board of Directors With >40 years of experience, Dr. Mazzo is a global pharmaceutical executive noted for his strategic prowess and his vast experience developing and launching new products across all therapeutical areas. Company Overview
Accomplished, industry veteran, independent board directors Detailed bios can be found at www.lisata.com 6 Gregory B. Brown, MD Chairman Biopharma executive with expertise in evaluating scientific, technical, clinical, and medical products as well as in healthcare systems and payor/reimbursement dynamics. Steven M. Klosk Director Pharma executive with 25+ years of experience in the pharmaceutical CDMO industry and proven leadership across all stages of product development. Cynthia L. Flowers Director Biopharma executive with extensive experience in leading and managing companies during transformative years of growth, particularly when it comes to commercialization of products in many therapeutic areas including oncology. Mohammad Azab, MD, MSc, MBA Director Pharma executive with 30+ years of experience in clinical research, business management, and global development, bringing multiple drugs to market across oncology and other therapeutic areas. Heidi Henson Director Senior executive with over two decades of financial operations experience with both public and private companies. Company Overview
Therapeutic Focus and Rationale Problem, Solution and Approach 7
Improved solid tumor treatment remains a vital, growing global need Therapeutic Focus and Rationale 8 2022 20 million new cases2 9.7 million deaths 2050 35 million new cases2 18.5 million deaths 77% In 2023, in the U.S. alone, of ~2 million newly diagnosed cancer cases, >90% were solid tumors1 1 https://seer.cancer.gov/statfacts/html/common.html; data retrieved November 2, 2023. 2 https://gco.iarc.who.int/tomorrow/en/dataviz/tables?mode=population&years=2050&types=1&populations=903_904_905_908_909_935_900; data retrieved Feb 12, 2024. Examples of solid tumors include cancers of the lung, breast, pancreas, liver, bile duct, kidneys, ovaries, brain, colon, prostate, esophagus, and head & neck projected
9 Therapeutic Focus and Rationale Current solid tumor treatments & patient outcomes are suboptimal Diagram: Abizanda-Campo, S. et al, Microsyst Nanoeng 9, 154 (2023) Tumor stroma acts as a physical barrier to anti-cancer agents An immunosuppressive tumor microenvironment (TME) contributes to tumor resistance and/or metastases Prolonged or escalated dosing of non-targeted anti-cancer therapies generally leads to intolerable off-target side effects A challenging tumor microenvironment complicates “targeting” and “penetration”
Maximizing solid tumor treatment success Step 1 Employ a tumor selective RGD motif Optimize Tumor Targeting OBJECTIVE: APPROACH: Step 2 Employ Internalizing RGD (iRGD) peptide to trigger CendR active transport mechanism Enhance Tumor Penetration OBJECTIVE: APPROACH: Step 3 Employ a specific iRGD that reduces immunosuppressive T cells, recruits cytotoxic T cells and inhibits metastases Modify the TME OBJECTIVE: APPROACH: A rational drug development approach to overcoming the obstacles to achieving optimized outcomes for patients with solid tumors Certepetide now in mid- to late-stage clinical development for solid tumor treatments based on a large body of preclinical evidence Result 10 Therapeutic Focus and Rationale
Certepetide promises optimized solid tumor treatment outcomes Certepetide converts tumor stroma from a barrier to a conduit for anti-cancer drugs 1 Sugahara, et al. Mol Cancer Ther; 14(1) January 2015; Hamilton, et al., J MolMed. April 2015; and Miyamura, et al., bioRxiv. May 2023. 11 Therapeutic Focus and Rationale Certepetide combats resistance and metastases1 Selectively depletes immunosuppressive T cells while enhancing the concentration of cytotoxic T cells Inhibits the metastatic cascade Effective with co-administered or molecularly bound (tethered) anti-cancer therapies Co-administration presents an initial streamlined development path to registration Tethering creates a new chemical entity providing new compound patent protection Certepetide is agnostic to the modality of the companion anti-cancer therapy
Certepetide development strategy is composed of two main pillars Therapeutic Focus and Rationale 12 Pursue rapid global registration in pancreatic ductal adenocarcinoma (mPDAC), initially combined with gemcitabine/nab-paclitaxel standard-of-care (SoC) Phase 2b 100% enrolled By 2030, pancreatic cancer is predicted to become the second most common cause of cancer mortality1 Today, only 3% of people diagnosed with pancreatic cancer will survive for 5 years Current life expectancy at the time of diagnosis is just 4.6 months Focus on Pancreatic Cancer & Other Advanced Solid Tumors 1 Europe Is Facing a Pancreatic Cancer Emergency - Medscape - January 25, 2024. Demonstrate certepetide effectiveness when combined with a variety of SoC regimens (e.g., chemotherapy, immunotherapy, etc.) in a variety of solid tumors Multiple Phase 1b/2a studies underway
Partnerships Noteworthy existing relationships and potential for many more 13
Existing partnerships support certepetide’s promise and broad applicability Strategic commercial partnership in China with Qilu Pharmaceutical Qilu granted exclusive rights to certepetide in China, Taiwan, Hong Kong and Macau Qilu assumes all development and commercialization responsibilities/costs in licensed territories • Strategy and activities under the auspices of a Joint Steering Committee with Lisata executives Lisata collected $15 million in milestones to date Potential for additional $221 million in milestones plus royalties on sales to Lisata R&D alliances contribute resources with minimal commercial interest in certepetide Australasian Gastro-Intestinal Trials Group - Clinical Trialists Consortium (Australia & New Zealand) WARPNINE - Foundation (Australia) Additional partnership opportunities exist for many combinations with certepetide By indication, modality of co-administered drug(s), and/or geography Partnerships 14
15 Certepetide (formerly LSTA1) Strong Scientific Foundation and Rationale
Certepetide selective tumor targeting & penetration mechanism of action Certepetide - Strong Scientific Foundation and Rationale 161 Ding et al., Nature Comm, 2019. 1 Integrin binding 2 Proteolytic cleavage 3 Neuropilin-1 binding 4 Resulting tumor penetration Bound certepetide is proteolytically- cleaved in the tumor microenvironment (TME) resulting in a C-end Rule (CendR) linear peptide fragment. The CendR fragment binds with high affinity and specificity to neuropilin-1 (NRP-1), an adjacent receptor on the same or nearby cell, activating the CendR active transport pathway¹ and triggering tumor penetration. Certepetide is a 9-amino acid cycle peptide with high binding specificity and affinity for αvβ3 and/or αvβ5 integrins that are upregulated on target cells. *Tumor cells and tumor vascular endothelial cells (components of the tumor stroma) CendR pathway actuation triggers encapsulation of circulating co-administered anti-cancer drugs, ferrying them through the stroma into the tumor. [Not pictured] Certepetide depletes immunosuppressive T cells and enhances cytotoxic T cells in the TME, while inhibiting metastases. Simplified rendition. Microvesicles can fuse to form channels across single cells.
Certepetide selectively and efficiently facilitates intratumoral penetration 1 Braun et al., Nature Mater. 2014. 2 Liu, Braun et al., Nature Comm. 2017. 17 Certepetide - Strong Scientific Foundation and Rationale Whole body imaging of mice with pancreatic ductal adenocarcinoma (arrow) dosed with Fluorescent Quantum Dots (FQDs) with and without certepetide Circulating FQDs result in whole body fluorescence Etching solution quenches fluorescence in circulation Certepetide provides targeted tumor penetration tumor tumor FQDs + Etching solution All FQDs in circulation Certepetide + FQDs + Etching solution All FQDs in tumor
Certepetide/iRGD activity & broad applicability consistently demonstrated Sampling of >350 scientific publications showing improved survival with certepetide/iRGD 18 Breast cancer + Herceptin®Lung cancer + gemcitabine Zhang, et al., Plos One, 2015 Breast cancer + nanoparticle Abraxane GI cancer + adoptive cell therapy Ding, et al., Nature, 2019 PDAC + irinotecan nanoparticles Orthotopically transplanted KPC PDAC tumors iRGD + irinotecan nanoparticles (i.v. co-admin) Liu X et al., J Clin Invest, 2017 PDAC + gemcitabine KPC mice genetically engineered to develop PDAC iRGD + gemcitabine (i.v. co-admin) Hurtado de Mendoza et al, Nature Comms, 2021 Sugahara, et al., Science, 2010Sugahara, et al., Science, 2010 Certepetide - Strong Scientific Foundation and Rationale
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 Gemcitabine + Nab-paclitaxel1 N=431 8.5 mos. 5.5 mos. 23% (99) 0.2% (1) 23% (98) 27% (118) 20% (86) 48% 61% Endpoints 1 Von Hoff D, et al., New England Journal of Medicine, 2013. 2 Dean A, et al., The Lancet Gastroenterology & Hepatology, 2022. N=31 13.2 mos. 9.7 mos. 59% (17) 3.4% (1) 55% (16) 31% (9) 10.3% (3) 79% 96% Certepetide + Gemcitabine + Nab-paclitaxel2 19 Certepetide Ph 1b/2a results: Compelling improvement of SoC efficacy Certepetide well-tolerated with no dose-limiting toxicities Safety of certepetide + SoC consistent with SoC alone First-line, mPDAC patients from 3 sites in Australia Certepetide - Strong Scientific Foundation and Rationale
1 Von Hoff D, et al., New England Journal of Medicine, 2013. 2 Dean A, et al., The Lancet Gastroenterology & Hepatology, 2022 1.0 3.0 5.0 7.0 9.0 11.0 13.0 Median Overall Survival (Months) Median Progression Free Survival (Months) M on th s Gemcitabine + Nab-paclitaxel Gemcitabine + Nab-paclitaxel + certepetide 13.2 months 8.5 months 5.5 months 9.7 months 55% Improvement in median OS 76% Improvement in median PFS Certepetide Ph 1b/2a results: Improved survival vs. SoC alone 20 1 2 Certepetide - Strong Scientific Foundation and Rationale
0.0% 10.0% 20.0% 30.0% 40.0% 50.0% 60.0% 70.0% 80.0% 90.0% 100.0% Complete Response Partial Response Objective Response Rate Disease Control Rate at 16 weeks CA19-9 >20% drop Gemcitabine + Nab-paclitaxel Gemcitabine + Nab-paclitaxel + certepetide 1 Von Hoff D, et al., New England Journal of Medicine, 2013. 2 Dean A, et al., The Lancet Gastroenterology & Hepatology, 2022 59% 23% 3.4%0.1% 55% 23% 48% 79% 61% 96% 21 Certepetide Ph 1b/2a results: Consistent improvement across associated endpoints 1 2 Certepetide - Strong Scientific Foundation and Rationale
Evidence of certepetide activity in other solid tumors Certepetide potentiated a complete response in metastatic gastroesophageal adenocarcinoma (mGEAC) FDG-PET* scan June 2022 FDG-PET scan Sept. 2022 Reduction in FDG activity demonstrated1 53-year-old male with mGEAC with significant (> 5cm) nodal metastases (June 2022) SoC combination chemotherapy (FOLFIRINOX) and radiotherapy, with immunotherapy (pembrolizumab) later added resulting in partial response Certepetide added to above regimen at cycle 7 and exploratory laparoscopy after cycle 18 (September 2022) showed no discernable disease – deemed a complete response 221 Buck, K.K, Dean, A., McSweeney, T. LSTA1 Potentiates Complete Response in Metastatic Gastroesophageal Adenocarcinoma. Oncol Cancer Case Rep. 2023, 9(6), 001-003 Certepetide - Strong Scientific Foundation and Rationale *Fluorodeoxyglucose (FDG)-positron emission tomography (PET)
23 Certepetide Clinical/Regulatory Development Portfolio
Certepetide – Clinical / Regulatory Development Portfolio Certepetide regulatory designations and implications 24 FDA Fast Track Designation Certepetide received Fast Track Designation from FDA for pancreatic cancer More frequent communication with and program-specific guidance from FDA Eligible for Accelerated Approval, Priority Review and Rolling Review Orphan Drug Designation Certepetide received Orphan Drug Designation from FDA and EMA for pancreatic cancer, from FDA for malignant glioma, and from FDA for osteosarcoma Incentives such as tax credits, marketing exclusivity, fee waivers and grant eligibility to support clinical trials Specialized regulatory assistance from FDA's Office of Orphan Products Development FDA Rare Pediatric Disease Designation Certepetide received Rare Pediatric Disease Designation from FDA for osteosarcoma Eligible for Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application, or may be sold or transferred Historically, vouchers have sold for $350 million USD and, more recently, have sold for $75-$100 million USD
Certepetide capital efficient development plan Current Phase DescriptionIndicationSponsor(s) Phase 3Phase 2Phase 1 ASCEND: Phase 2b, placebo-controlled trial (N=158) Gemcitabine/nab-paclitaxel + certepetide or placebo Australia/New Zealand First-line mPDACAGITG/Lisata BOLSTER: Phase 2a, placebo-controlled trial (N=80) 1L CCA: Gemcitabine/cisplatin/durvalumab with certepetide or placebo 2L CCA: FOLFOX with certepetide or placebo United States First- and Second-line Cholangiocarcinoma (CCA)Lisata CENDIFOX: Phase 1b/2a, open-label trial (N=51) FOLFIRINOX + panitumumab* + certepetide United States Pancreatic, Colon, and Appendiceal Cancers KUCC/Lisata Investigator-initiated trial Phase 1b/2a, open-label trial (N=41) Gemcitabine/nab-paclitaxel + certepetide China First-line mPDACQilu/Lisata iLSTA: Phase 1b/2a, open-label trial (N=30) Gemcitabine/nab-paclitaxel/durvalumab + certepetide Australia Locally advanced, non- resectable PDACWARPNINE/Lisata Phase 2a, placebo-controlled trial (N=30) Temozolomide +/- certepetide Estonia/Latvia First-line Glioblastoma Multiforme (GBM) Tartu University/Lisata Investigator-initiated trial Phase 2, placebo-controlled trial (N=120) Gemcitabine/nab-paclitaxel + certepetide China First-line mPDACQilu/Lisata FORTIFIDE: Phase 1b/2a placebo-controlled trial (N=30) Gemcitabine/nab-paclitaxel + continuous infusion of certepetide/placebo United States Second-line mPDACLisata Enrollment complete Enrolling Enrollment complete Enrolling *Panitumumab may be added for colorectal or appendiceal patients without Ras mutation. Certepetide – Clinical / Regulatory Development Portfolio 25 Enrolling Enrolling 1L CCA Enrollment complete 2L CCA Enrolling soon Enrolling soon
Development Milestones 26
A wealth of anticipated key certepetide milestones 27 1Q 20264Q 20253Q 20252Q 20251Q 20254Q 20243Q 20242Q 20241Q 20244Q 20233Q 20232Q 2023 ASCEND [AUS, NZ] First-line mPDAC BOLSTER [USA] First- and second-line CCA CENDIFOX [USA] Pancreatic, Colon and Appendiceal Cancers Qilu: Phase 1b/2a [CHN] First-line mPDAC iLSTA [AUS] Non-resectable mPDAC Phase 2a [EST, LATV] First-line GBM Qilu: Phase 2 [CHN] First-line mPDAC FORTIFIDE [USA] Second-line mPDAC Development Milestones PDAC cohort Colon/Appendiceal cohortsEnrolling Final 6-month PFS/OS data Enrolling Cohort A top-line data Final PFS/OS data Futility analysisEnrolling Preliminary PFS data Final data ORR dataEnrolling *Several of these studies are investigator-initiated trials. Lisata has limited control and thus, timelines and expectations may be subject to change. PFS: Progression-free Survival OS: Overall Survival ORR: Objective Response Rate First patient in Last patient in Interim analysis Data Final data Milestone achievedSafety run-in analysis Final PFS/OS data Enrolling Safety run-in analysis 1L & 2L OS data1L CCA FPI 1L CCA LPI Achieved 6 months early 2L CCA LPI2L CCA FPI Biomarker Data Final PFS/OS data
Financial Highlights 28
Capital projected to fund all clinical programs to data Financial Highlights 29 $38.3M Cash & Investments As of 6/30/2024 $0 Debt 1Q2026 Projected Cash Runway Into 8.3 million sharesCommon Shares Outstanding (6/30/2024): 1.5 million shares Options Outstanding (6/30/2024): Exercise Price: $0.02 - $4.22 = 1,216,100 shares Exercise Price: > $4.22 = 237,800 shares 1.4 million shares Warrants Outstanding (6/30/2024): Weighted Average Exercise Price: $42.51
Investment Thesis Promising asset based on a body of compelling data Rational and focused development program Highly experienced management team Financially stable company 30
* As of 6/30/2024; includes investments Key factors supporting investment in Lisata Therapeutics 31 Strong Investment Rationale PEOPLE Seasoned management with successful international development experience and expertise INTELLECTUAL PROPERTY Proprietary field-leading technology in underserved global indications MILESTONES Multiple projected product and business milestones over the next 18 months CAPITAL $38.3 million cash*- no debt; Development funded through critical data milestones PARTNERING Platform technology “validated” by existing partnerships with potential for many others
© L i s a t a T h e r a p e u t i c s , I n c . 2 0 2 4 . A l l r i g h t s r e s e r v e d . Investor Relations Contact: John D. Menditto VP, IR & Corporate Communications o: (908) 842-0084 | e: jmenditto@lisata.com Nasdaq: LSTA | www.lisata.com Targeted Therapy Delivered
Appendix 33
Strategic Rationale Stage of Development Indication and Trial Product/Comparator Development Partner(s) [Development Venue] Corroborate Phase 1b results in a placebo-controlled trial and evaluate 2 dose regimens of certepetide for dose optimization Phase 2b (ASCEND) First-line mPDAC; Gemcitabine/nab-paclitaxel with certepetide or placebo Lisata/AGITG [Australia/New Zealand] Assess certepetide safety and effectiveness in cholangiocarcinoma in a placebo-controlled trial (proof-of-concept) Phase 2a (BOLSTER) First- and Second-line Cholangiocarcinoma (CCA); 1L CCA: Gemcitabine/cisplatin/durvalumab + certepetide or placebo 2L CCA: FOLFOX + certepetide or placebo Lisata [United States] Tumor immuno-profiling pre- & post- treatment and certepetide effectiveness assessment in combination with chemo and an EGFR inhibitor (open-label) Phase 1b/2a (CENDIFOX) Pancreatic, Colon & Appendiceal Cancers; FOLFIRINOX + panitumumab** with certepetide KUCC/Lisata* [United States] Assess safety, PK and therapeutic effect of certepetide in Chinese patients (open-label) Phase 1b/2a First-line mPDAC; Gemcitabine/nab-paclitaxel + certepetide Qilu [China] Assess certepetide safety and effectiveness in combination with IO & Chemo in locally advanced PDAC; determine if inoperable tumors can become operable (open-label) Phase 1b/2a (iLSTA) Locally Advanced, Non-Resectable PDAC; Gemcitabine/nab-paclitaxel/durvalumab + certepetide WARPNINE/Lisata [Australia] Assess certepetide safety and effectiveness in additional tumor type (GBM) in a placebo-controlled trial Phase 2a First-line Glioblastoma Multiforme (GBM); Temozolomide +/- certepetide Tartu University/Lisata* [Estonia/Latvia] Continue development of certepetide in China (placebo controlled) Phase 2b First-line mPDAC; Gemcitabine/Nab-paclitaxel + certepetide Qilu [China] Evaluate the safety, tolerability, and efficacy of a 4-hour continuous infusion of certepetide in combination with SoC in subjects with mPDAC who have progressed on FOLFIRINOX. Haystack MRD technology to measure ctDNA for early efficacy exploration. Phase 1b/2a (FORTIFIDE) Second-line mPDAC; Gemcitabine/nab-paclitaxel + continuous infusion of certepetide or placebo Lisata [United States] *Investigator-initiated trial **Panitumumab may be added for colorectal or appendiceal patients without Ras mutation Appendix Certepetide capital efficient development plan 34
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) Sponsor/Partner Corroborate Phase 1b results in a placebo-controlled study Determine if a second dose of certepetide further improves patient outcomes Objective Phase 2b randomized, double-blind study in mPDAC testing gemcitabine + nab-paclitaxel SoC with one of two certepetide dose regimens or placebo Design N=158 (~30 sites in Australia and New Zealand)Study Size Primary: Progression Free Survival Secondary: AEs, SAEs, Overall Survival, Objective Tumor Response Rate Endpoints Enrollment completed December 2023 Earliest possible data 4Q24 Timing ASCEND: Phase 2b, blinded, randomized trial in mPDAC 35 Appendix
R INTERVENTION Arm (N=60) • Nab-paclitaxel 125 mg/m2 IV • Certepetide 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 certepetide Placebo IV • Gemcitabine 1000 mg/m2 IV Dose on days 1, 8, 15 every 28 days INTERVENTION Arm (N=40) • Nab-paclitaxel 125 mg/m2 IV • Certepetide 3.2 mg/kg IV • Gemcitabine 1000 mg/m2 IV • Certepetide 3.2 mg/kg IV 4 hours later Dose on days 1, 8, 15 every 28 days INTERVENTION Arm (N=20) • Nab-paclitaxel 125 mg/m2 IV • Matching certepetide Placebo IV • Gemcitabine 1000 mg/m2 IV • Matching Placebo certepetide IV 4 hours later Dose on days 1, 8, 15 every 28 days R R Cohort A Cohort B 1:1 2:1 2:1 One dose of certepetide assessed Two doses of certepetide assessed Endpoints • Progression Free Survival (PFS) • ORR • OS • Safety • QoL • Exploratory Endpoints • Sponsor/Partner: AGITG in collaboration with the NHMRC Clinical Trial Centre at the University of Sydney • LSTA funded • Timing: Enrollment completed December 2023; Earliest possible data 4Q24Phase 2b randomized, double- blind study in mPDAC testing gemcitabine + nab-paclitaxel (SoC) with two certepetide dose regimens or placebo ASCEND: Phase 2b, blinded, randomized trial in mPDAC Appendix 36
Qilu Pharmaceutical (funds all development in China)Sponsor/Partner Evaluate safety, pharmacokinetics and preliminary efficacy of certepetide added to SoC in Chinese patients with mPDAC Objective Phase 1b/2a open-label study in advanced mPDAC patients of Chinese ethnicity testing SoC chemotherapy (gemcitabine + Qilu-produced nab-paclitaxel) in combination with certepetide Design N=50 (~15 sites)Study Size Primary: AEs, SAEs, Objective Response Rate, Duration of Response, Disease Control Rate, Overall Survival, and Progression Free Survival Secondary: Pharmacokinetic parameters Endpoints Final data anticipated 2H2024Timing Appendix 37 Phase 1b/2a open-label trial in mPDAC in China
7 Day Safety Evaluation Certepetide 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 certepetide Day 1 Phase 1b/2a study evaluating the safety, pharmacokinetics, and preliminary efficacy of certepetide for injection in Chinese patients with advanced metastatic pancreatic ductal adenocarcinoma mPDAC 3.2 mg/kg certepetide Day 1 7 Day Safety Evaluation Certepetide 3.2 mg/kg + nab-pac* + gem Days 1, 8, and 15 every 28 days Phase 1b N=3 N=3 Phase 2 Extension N=10-12 N=10-12 N=30-50 Disease Progression Response rates PFS OS Safety • Sponsor/Partner: Qilu Pharmaceutical (funds all development in China) • Timing: Final data anticipated 2H2024 *Qilu produced Phase 1b/2a open-label trial in mPDAC in China Appendix 38
University of Kansas Medical Center (Investigator initiated trial in U.S.) KUCC funded; Lisata provides certepetide Sponsor/Partner Evaluate the safety and therapeutic effect of certepetide in combination with neoadjuvant FOLFIRINOX- based therapies and an EGFR inhibitor for the treatment of pancreatic, colon and appendiceal cancers and determine immuno-profiling in tumor pre- & post- treatment Objective Phase 1b/2a open-label study in resectable pancreatic, colon with oligo metastases and appendiceal with peritoneal metastases cancers testing SoC chemotherapy (neoadjuvant FOLFIRINOX-based therapies) with certepetide ± panitumumab Design N=51 (21 PDAC, 15 colon and 15 appendiceal) Study Size Primary: Drug Safety Secondary: Overall Survival, Disease-free Survival, Overall Response Rate, RO Resection Rate, Pathological Response Rate Endpoints Enrollment completion target 4Q24Timing CENDIFOX: Phase 1b/2a open-label trial in PDAC and other cancers Appendix 39
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 Colon and appendiceal cancer with peritoneal mets COHORT 3 Colon cancer with oligo metastatic disease Repeat Biopsy ~72 hours after C3D1 tx Tissue immune profiling FOLFIRINOX (± Panitumumab if RAS/BRAF wildtype - Cohorts 2, 3) + Certepetide X 3, 6, or 9 Cycles Resume Standard of Care Phase 1b/2a open-label trial of certepetide in combination with neoadjuvant FOLFIRINOX based therapies in pancreatic, colon and appendiceal cancers (CENDIFOX) • Sponsor/Partner: University of Kansas Medical Center (ITT) • KUCC funded: Lisata provides certepetide • Timing: Enrollment completion target 4Q24 CENDIFOX: Phase 1b/2a open-label trial in PDAC and other cancers Appendix 40
Lisata (U.S.)Sponsor/Partner Evaluate the preliminary efficacy, safety and tolerability of certepetide in combination with standards of care in subjects with first- and second-line cholangiocarcinoma Objective Phase 2 randomized, double-blind, placebo-controlled, proof-of-concept trial in first- and second-line cholangiocarcinoma testing corresponding SoC with certepetide or placebo Design N=80 (N=40 per tumor type) 1:1 SoC + certepetide or SoC + placebo Study Size Primary: OS Secondary: Safety, ORR, PFS Endpoints Enrollment completed for 1L CCA Enrollment anticipated July 2024 for 2L CCA Timing BOLSTER: Phase 2 blinded, randomized trial in Cholangiocarcinoma Appendix 41
Dosed on Days 1, 8 every 21 days X 8 cycles Disease Progression Response rates Safety Confirm Eligibility Informed Consent Survival Analysis Certepetide + cisplatin/gemcitabine/durvalumab 72-hour run-in without SoC 1st line Cholangiocarcinoma (CCA) Placebo + cisplatin/gemcitabine/durvalumab N=20 N=20 R Phase 2a, double-blind, placebo-controlled, multi-center, randomized study evaluating certepetide when added to standard of care (SoC) versus standard of care alone in subjects with first- and second-line cholangiocarcinoma • Sponsor: Lisata • Timing: • Enrollment completed for 1L CCA • Enrollment anticipated July 2024 for 2L CCA BOLSTER: Phase 2 blinded, randomized PoC trial in various cancers Appendix 42 Dosed every 14 days Disease Progression Response rates Safety Confirm Eligibility Informed Consent Survival Analysis Certepetide + FOLFOX 72-hour run-in without SoC 2nd line Cholangiocarcinoma (CCA) Placebo + FOLFOX N=20 N=20 R
Qilu Pharmaceutical (funds all development in China)Sponsor/Partner Further evaluate safety and therapeutic efficacy of certepetide when added to SoC in Chinese patients with locally advanced unresectable mPDAC Objective Phase 2b, double-blind, placebo-controlled, randomized study evaluating certepetide + SoC (Qilu-produced nab-paclitaxel and gemcitabine) vs. placebo + SoC Design N=120 (1:1 SoC + certepetide or SoC + placebo)Study Size Objective response rate, progression free survival, duration of response, disease control rate, overall survival Safety Endpoints Trial initiated 2Q24Timing Appendix 43 Phase 2 double-blind, placebo-controlled trial in mPDAC in China
Days 1, 8, 15 and every 28 days Disease Progression Response rates PFS Safety Gemcitabine + Qilu produced nab-paclitaxel + certepetide 3.2 mg/kg Confirm Eligibility Informed Consent 1:1 Survival AnalysismPDAC R Gemcitabine + Qilu produced nab-paclitaxel + placebo N=60 N=60 Phase 2b, double-blind, placebo-controlled, randomized, multicenter study evaluating the safety and efficacy of certepetide when added to standard of care (nab-paclitaxel and gemcitabine) vs. standard of care alone and placebo in Chinese subjects with locally advanced unresectable mPDAC • Sponsor/Partner: Qilu Pharmaceutical (funds all development in China) • Timing: Trial initiated 2Q24 Phase 2 blinded, placebo-controlled trial in mPDAC in China Appendix 44
WARPNINE, Inc. (registered charity in Australia) is funding trial Lisata providing study drug Sponsor/Partner Evaluate safety and therapeutic effect of LSTA1 in combination with IO & Chemo in locally advanced non-resectable pancreatic ductal adenocarcinoma (PDAC); determine if inoperable tumors can become operable Objective Phase 1b/2a proof-of-concept safety and early efficacy study of LSTA1 in combination with durvalumab, gemcitabine and nab-paclitaxel, as first-line treatment in locally advanced non-resectable pancreatic adenocarcinoma Design N=30Study Size Safety and tolerability; 28-day DLTs Objective response rate, PFS, OS, duration of response, immune cell infiltration Endpoints Enrollment commenced April 2023Timing Appendix 45 iLSTA: Phase 1b/2a trial in locally advanced PDAC with chemo & IO
Cohort 2 Gemcitabine + nab-paclitaxel + LSTA1 + Placebo durvalumab N=5 Cohort 1 Gemcitabine + nab-paclitaxel + Placebo LSTA1 + Placebo durvalumab N=5 Cohort 3 Gemcitabine + nab-paclitaxel + LSTA1 + durvalumab N=up to 20 12 weeks 8 weeks tumor burden assessments until 24 months or recurrence 12 month Follow-up Primary Endpoint 24 months Follow-up completion EUS Biopsy Screening EUS & Biopsy At weeks 12-16 Tumor burden assessment at screening, cycle 2, and then 8-weekly thereafter. Patients are treated with 28-day cycles until surgery, definitive radiotherapy, unacceptable toxicity, progression or death Endpoints: safety, DLT, ORR, PFS, OS, DoR, immune profiling R an do m iz e Phase 1b/2a proof-of-concept safety and early efficacy study of LSTA1 in combination with durvalumab, gemcitabine and nab-paclitaxel, as first-line treatment in locally advanced non-resectable pancreatic ductal adenocarcinoma • Sponsor: WARPNINE, Inc. - funding trial • Timing: Enrollment commenced April 2023 Appendix 46 iLSTA: Phase 1b/2a trial in locally advanced PDAC with chemo & IO
Tartu University Hospital (Investigator initiated trial in Estonia) Lisata providing study drug and funding trial Sponsor/Partner Evaluate safety, tolerability, and therapeutic effect of certepetide in combination with standard-of-care (temozolomide) in patients with previously untreated Glioblastoma Multiforme Objective Phase 2a proof-of-concept, double-blind, placebo-controlled, randomized study evaluating certepetide when added to standard of care (temozolomide) versus SoC and placebo in subjects with newly diagnosed Glioblastoma Multiforme (GBM) Design N=30 total (N=3 safety run-in, N=18 in main study schema)Study Size Safety, tolerability ORR, PFS, OS, disease control rate Endpoints Enrollment commenced December 2023Timing Appendix 47 Phase 2a trial of certepetide with SoC in first-line GBM
Days 1, 2, 3, 4, 5 and every 28 days for 6 cycles Disease Progression Response rates Safety Temodar® + certepetideConfirm Eligibility Informed Consent 2:1 Survival Analysis 72-hour Run-in without SoC Newly Diagnosed GBM R Temodar® + certepetide matching placebo N=18 N=9 Phase 2a proof-of-concept double-blind, placebo-controlled, randomized, proof-of-concept study evaluating certepetide when added to standard of care (temozolomide) versus temozolomide and matching certepetide placebo in subjects with newly diagnosed GBM • Sponsor: Tartu University Hospital; Estonia • Funding: Lisata • Timing: Enrollment commenced December 2023 Appendix 48 Phase 2a trial of certepetide with SoC in first-line in GBM Main Study Schema Safety Lead-in Schema Confirm Eligibility Informed Consent Newly Diagnosed GBM 3-Day Run-in N=3 TMZ + certepetide Safety AnalysisCertepetide on Days 1, 2 which is repeated after 28 days for 2 cycles * TMZ + certepetide Days 1, 2, 3, 4, 5 and every 28 days for 4 remaining cycles • TMZ will be administered on Days 1, 2, 3, 4, and 5 of each cycle Disease Progression Response rates Safety Survival Analysis
Lisata (U.S. only)Sponsor/Partner Evaluate the safety, tolerability, pharmacodynamics, pharmacokinetics, and efficacy of certepetide when given as a 4-hour continuous infusion in combination with SoC in subjects with second-line mPDAC who have progressed on FOLFIRINOX. Haystack Oncology MRD technology to measure ctDNA for early efficacy exploration. Objective Phase 1b/2a, double-blind, placebo-controlled, three-arm, randomized study evaluating the following treatment arms in subjects with second-line mPDAC who have progressed on FOLFIRINOX: an intravenous push of certepetide with continuous 4-hour infusion + SoC a single intravenous push of certepetide with continuous infusion of matching placebo + SoC an intravenous push of matching placebo with a continuous infusion of matching placebo + SoC Design N=30Study Size Safety and tolerability PFS, OS Endpoints First patient treated target 4Q24Timing Appendix 49 FORTIFIDE: Phase 1b/2a continuous infusion study of certepetide
FORTIFIDE: Phase 1b/2a continuous infusion study of certepetide Subjects with 2L mPDAC Obtain Informed Consent Confirm Eligibility Gemcitabine, certepetide 3.2 mg/kg single IV push/ 4-hour continuous placebo infusion + nab-paclitaxel Days 1, 8, and 15 every 28 days Gemcitabine, certepetide placebo single IV push/ 4-hour continuous placebo infusion + nab-paclitaxel Days 1, 8, and 15 every 28 days N=10 N=10 Gemcitabine, certepetide 3.2 mg/kg single IV push/4-hour continuous certepetide infusion + nab-paclitaxel Days 1, 8, and 15 every 28 days N=10 R Safety Biomarkers PK Disease Progression Response rates PFS OS Primary Endpoint: • Safety Secondary Efficacy Endpoints: • Mean change from baseline in CA19-9 and CEA • Mean change from baseline in ctDNA (quantitative) • Pharmacokinetics of certepetide • ORR, PFS, OS Secondary Efficacy Endpoints to be assessed: • At baseline • C1 Day 1 (2 and 4 hours after dosing) • C1 Days 8, and 15 pre-dosing for Cycle 1 • Day 1 of each subsequent cycle until disease progression 72 hour Run-in without SoC 72 hour Run-in without SoC 72 hour Run-in without SoC 50