UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
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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 May 9, 2024, Lisata Therapeutics, Inc. (the "Company") issued a press release in connection with its financial results for the first quarter ended March 31, 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 May 9, 2024 | |||||
| Lisata Therapeutics, Inc. Corporate Presentation, May 9, 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: May 9, 2024
Exhibit 99.1
Lisata Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Update
Seminal Phase 2b ASCEND trial top-line data expected in fourth quarter of 2024
Projected available cash to fund planned operations into early 2026 covering all studies through data
Conference call scheduled for today at 4:30 p.m. Eastern Time
BASKING RIDGE, NJ (May 9, 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 three months ended March 31, 2024.
“2024, a pivotal year for Lisata, is off to a very strong start,” stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Lisata. “Although we project multiple data readouts over the next 18 months, topline results from the Phase 2b ASCEND trial later this year have transformative potential for the Company. These results will be instrumental in determining the future of Lisata, and we plan to use them to explore conditional approvals with various regulatory agencies and/or to design an optimized Phase 3 program in pancreatic ductal adenocarcinoma. Since the start of the year, we have received both U.S. FDA Orphan Drug and Rare Pediatric Disease designations for certepetide, previously known as LSTA1, in osteosarcoma, further validating the broad therapeutic potential of this innovative therapy. We are energized by the progress we are making and excited about Lisata’s prospects.“
Dr. Mazzo added, “Our continued prudent financial management allows us to reaffirm our projection that currently available cash will fund operations into early 2026, providing a solid foundation to fund all ongoing and planned trials through to completion. More than ever, we remain confident in our ability to execute our development activities with the goal of reaching critical milestones at the earliest possible juncture.”
Development Portfolio Highlights
Certepetide as a treatment for solid tumors in combination with other anti-cancer agents
Certepetide is an investigational drug designed to activate the CendR uptake pathway that allows co-administered or molecularly bound anti-cancer drugs to target and penetrate solid tumors more effectively. Certepetide is designed to actuate 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, to the exclusion of normal tissues. In preclinical models, certepetide has also shown the ability to modify the tumor microenvironment, leading to the expectation that tumors will become more susceptible to immunotherapies and inhibiting the metastatic cascade (i.e., the spread of cancer to other parts of the body). Lisata and its development partners have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies, and RNA-based therapeutics. To date, certepetide has also demonstrated favorable safety, tolerability and activity in completed and ongoing clinical trials designed to test its ability to enhance delivery of standard-of-care (“SOC”) chemotherapy for metastatic pancreatic cancer (“mPDAC”). Certepetide has been granted orphan drug designation for pancreatic cancer in the U.S. and Europe as well as for glioblastoma multiforme (“GBM”) and osteosarcoma in the U.S. It also received a Fast Track designation from the FDA for pancreatic cancer and, just recently, a Rare Pediatric Disease designation from the FDA for osteosarcoma. 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:
•ASCEND: Phase 2b double-blind, randomized, placebo-controlled clinical trial evaluating two dosing regimens of certepetide in combination with gemcitabine/nab-paclitaxel SOC chemotherapy in patients with 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-line cholangiocarcinoma. The trial is actively enrolling with enrollment completion expected by the end of the third 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 continues to make steady progress with enrollment completion for all three arms expected 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 recently announced, Qilu has begun treating patients in their Phase 2 placebo-controlled trial in mPDAC. The study is planned to take approximately 18 months to complete enrollment and another 13 months for patient follow-up and data analysis and reporting.
•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 standard-of-care gemcitabine and nab-paclitaxel chemotherapy versus standard-of-care 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 standard-of-care 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.
First Quarter 2024 Financial Highlights
For the three months ended March 31, 2024, operating expenses totaled $6.6 million, compared to $6.8 million for the three months ended March 31, 2023, representing a decrease of $0.2 million or 3.6%.
Research and development expenses were approximately $3.2 million for the three months ended March 31, 2024, compared to $3.2 million for the three months ended March 31, 2023, representing an essentially unchanged spend. The minor increase of $62,000 or 2.0% was primarily due to an increase in expenses associated with enrollment activities in the current year for the BOLSTER trial, partially offset by a reduction in expenses associated with the Phase 2b ASCEND trial which completed enrollment in the prior year.
General and administrative expenses were approximately $3.4 million for the three months ended March 31, 2024, compared to $3.7 million for the three months ended March 31, 2023, representing a decrease of $0.3 million or 8.3%. This was primarily due to a decrease in staffing costs associated with the elimination of the Chief Business Officer position on May 1, 2023, a reduction in option assumption equity expense in connection with the Company’s merger with Cend Therapeutics, Inc., a decrease in directors and officers insurance premiums, and a reduction in spend on consulting and legal fees partially offset by one-off settlement-related costs.
Overall, net losses were $5.4 million for the three months ended March 31, 2024, compared to $6.2 million for the three months ended March 31, 2023.
Balance Sheet Highlights
As of March 31, 2024, Lisata had cash, cash equivalents, and marketable securities of approximately $43.3 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, May 9, 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 lead product candidate, certepetide, is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to target and penetrate solid tumors more effectively. Based on Lisata’s CendR Platform® Technology, Lisata has already established noteworthy commercial and R&D partnerships. The Company expects to announce numerous clinical study and business milestones over the next two years and has projected that its current business and development plan is funded with available capital through these milestones and into early 2026. 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 strategy, future operations, future financial position, future revenue, projected expenses and capital, 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, 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 and Media:
Lisata Therapeutics, Inc.
John Menditto
Vice President, Investor Relations and Corporate Communications
Phone: 908-842-0084
Email: jmenditto@lisata.com
- Tables to Follow -
Lisata Therapeutics, Inc.
Selected Financial Data
(in thousands, except per share data)
| Three Months Ended March 31, | |||||||||||||||||
| 2024 | 2023 | ||||||||||||||||
| (unaudited) | (unaudited) | ||||||||||||||||
| Statement of Operations Data: | |||||||||||||||||
| Research and development | $ | 3,241 | $ | 3,179 | |||||||||||||
| General and administrative | 3,360 | 3,665 | |||||||||||||||
| Total operating expenses | 6,601 | 6,844 | |||||||||||||||
| Operating loss | (6,601) | (6,844) | |||||||||||||||
| Investment income, net | 589 | 670 | |||||||||||||||
| Other expense, net | (187) | (13) | |||||||||||||||
| Net loss before benefit from income taxes and noncontrolling interests | (6,199) | (6,187) | |||||||||||||||
| Benefit from income taxes | (798) | — | |||||||||||||||
| Net loss | (5,401) | (6,187) | |||||||||||||||
| Less - net income attributable to noncontrolling interests | — | — | |||||||||||||||
| Net loss attributable to Lisata Therapeutics, Inc. common stockholders | $ | (5,401) | $ | (6,187) | |||||||||||||
| Basic and diluted loss per share attributable to Lisata Therapeutics, Inc. common stockholders | $ | (0.65) | $ | (0.77) | |||||||||||||
| Weighted average common shares outstanding | 8,294 | 7,987 | |||||||||||||||
| March 31, 2024 | December 31, 2023 | ||||||||||||||||
| (unaudited) | |||||||||||||||||
| Balance Sheet Data: | |||||||||||||||||
| Cash, cash equivalents and marketable securities | $43,349 | $50,535 | |||||||||||||||
| Total assets | 48,240 | 54,694 | |||||||||||||||
| Total liabilities | 5,497 | 6,800 | |||||||||||||||
| Total equity | 42,743 | 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| May 9, 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.
Lisata at a Glance Company Overview 3
A clinical stage therapeutics development company rapidly advancing a novel solid tumor targeting and penetration technology to improve the efficacy of anti-cancer drugs Seasoned management with successful international drug development experience and expertise Proprietary field- leading technology in underserved global indications Multiple product and business milestones projected over the next 24 months Platform technology “validated” by existing partnerships with potential for many others Projected cash runway into 2026, funding all development programs through to data Company Overview 4 Lisata Therapeutics (Nasdaq: LSTA)
Therapeutic Focus and Rationale Problem, Solution and Approach 5
Improved solid tumor cancer treatment is a vital global need Therapeutic Focus and Rationale 6 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 tumor cancers1 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 tumor cancers include cancers of the lung, breast, pancreas, liver, bile duct, kidneys, ovaries, brain, colon, prostate, esophagus, and head & neck projected
7 Therapeutic Focus and Rationale Current solid tumor treatments are suboptimal 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 therapy generally leads to intolerable off-target side effects Diagram: Abizanda-Campo, S. et al, Microsyst Nanoeng 9, 154 (2023) A challenging tumor microenvironment complicates “targeting” and “penetration”
Harnessing the C-end Rule (CendR) transport mechanism for solid tumor penetration Therapeutic Focus and Rationale 8 Improving selective solid tumor penetration to maximize treatment effects RGD peptides target tumor cells, but do not enhance penetration and delivery Internalizing RGD (iRGD) peptides combine targeting and penetration enhancement Certepetide (LSTA1) is an iRGD peptide that triggers the CendR active transport mechanism to selectively target and deliver anti-cancer drugs to solid tumors Certepetide is in mid- to late-stage clinical development for solid tumor treatment
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. Therapeutic Focus and Rationale Certepetide promises optimized solid tumor treatment 9 Certepetide converts tumor stroma from a barrier to a conduit for anti-cancer drugs Certepetide combats resistance and metastases1 • Preclinically, certepetide selectively depletes immunosuppressive T cells, enhances cytotoxic T cells, and inhibits the metastatic cascade Certepetide is agnostic to the modality of the companion anti-cancer therapy • 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 prolonged compound exclusivity
Certepetide development strategy is composed of two main pillars Therapeutic Focus and Rationale 10 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 & Other Advanced Solid Tumor Cancers 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 tumor cancers • Multiple Phase 1b/2a studies underway
Partnerships Noteworthy existing relationships and potential for many more 11
Existing partnerships support certepetide’s promise and broad applicability Strategic commercial partnership in China with Qilu Pharmaceutical 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 Collected $15 million in milestones to date Potential for additional $221 million in milestones plus royalties on sales Development alliances contribute resources without 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 12
13 Certepetide (formerly LSTA1) Strong Scientific Foundation and Rationale
Tumor or Tumor Vascular Endothelial Cell αvβ3 β5 Certepetide (LSTA1) A) Integrin binding Certepetide is a 9 amino acid cyclic peptide with high binding affinity and specificity to αvβ3/β5 integrins that are upregulated on tumor cells and tumor endothelial cells (i.e., tumor stroma) B) Proteolytic cleavage NRP1 Once bound to αvβ3 & β5 integrins, certepetide is cleaved by proteases in the tumor microenvironment (TME) releasing a C-end Rule (CendR) linear peptide fragment αvβ3 β5 C) Neuropilin-1 binding CendR Fragment NRP1 αvβ3 β5 NRP1 The CendR fragment binds with high affinity and specificity to neuropilin-1 (NRP1), an adjacent receptor on the same or nearby cell, activating the CendR transport pathway1 and triggering tumor penetration CendR Fragment D) Transcytosis plus TME modification CendR Transport Mechanism Co-administered anti-cancer drugs CendR pathway actuation triggers tumor penetration of circulating co-administered anti-cancer drugs Certepetide-induced TME modification provokes reduction of immunosuppressive T cells, augmentation of cytotoxic T cells, and inhibition of metastases 14 Certepetide - Strong Scientific Foundation and Rationale Certepetide selective tumor targeting & penetration mechanism of action 1 Ding et al., Nature Comm, 2019. Cleavage
Certepetide selectively and efficiently facilitates intratumoral penetration 1 Braun et al., Nature Mater. 2014. 2 Liu, Braun et al., Nature Comm. 2017. 15 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 16 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 17 Certepetide Ph 1b/2a results: Compelling improvement of SoC efficacy Certepetide well-tolerated, 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 18 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% 19 Certepetide Ph 1b/2a results: Consistent improvement across associated endpoints 1 2 Certepetide - Strong Scientific Foundation and Rationale
Clinical 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 radiotherapy, chemotherapy (FOLFIRINOX), and immunotherapy (pembrolizumab) resulted in partial response Certepetide added to SoC regimen at cycle 7 Exploratory laparoscopy after cycle 18 (September 2022) showed no discernable disease – complete response 201 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)
21 Certepetide Clinical/Regulatory Development Portfolio
Certepetide – Clinical / Regulatory Development Portfolio Certepetide regulatory designations and implications 22 FDA Fast Track Designation More frequent communication with and program-specific guidance from FDA Eligible for Accelerated Approval, Priority Review and Rolling Review Certepetide received Fast Track Designation from FDA for pancreatic cancer Orphan Drug Designation 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 Certepetide received Orphan Drug Designations from FDA and EMA for pancreatic cancer, from FDA for malignant glioma, and from FDA for osteosarcoma FDA Rare Pediatric Disease Designation 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 received Rare Pediatric Disease Designation from FDA for osteosarcoma
*Panitumumab may be added for colorectal or appendiceal patients without Ras mutation First-line mPDAC Gemcitabine/nab-paclitaxel with certepetide or placebo N=158 AGITG/Lisata Phase 2b (ASCEND) Placebo-controlled Enrollment complete Australia & New Zealand First-line Cholangiocarcinoma (CCA) Gemcitabine/cisplatin/durvalumab with certepetide or placebo N=40 Lisata Phase 2a (BOLSTER) Placebo-controlled Enrolling USA Pancreatic, Colon, & Appendiceal Cancers FOLFIRINOX + panitumumab* with certepetide N=50 KUCC/Lisata Phase 1b/2a (CENDIFOX) Open-label Enrolling USA First-line mPDAC Gemcitabine/nab-paclitaxel + certepetide N=41 Qilu/Lisata Phase 1b/2a Open-label Enrollment complete China Locally advanced, non-resectable PDAC Gemcitabine/nab-paclitaxel/durvalumab + certepetide N=30 WARPNINE/Lisata Phase 1b/2a (iLSTA) Open-label Enrolling Australia Sponsors/Partners Region Indication and Test Articles Status 23 Certepetide capital efficient development plan Certepetide – Clinical / Regulatory Development Portfolio
First-line Glioblastoma Multiforme (GBM) Temozolomide +/- certepetide N=30 Tartu University/ Lisata Phase 2a Placebo-controlled Enrolling Estonia & Latvia Peritoneal Carcinomatosis (Colon & Ovarian) HIPEC* intraoperative intraperitoneal lavage + certepetide N=21 UCSD/Lisata Phase 1 Open-label Enrolling USA First-line mPDAC Gemcitabine/Nab-paclitaxel + certepetide N=120 Qilu/Lisata Phase 2 Placebo-controlled Enrolling China Locally advanced, non-resectable Gastroesophageal Adenocarcinoma Nivolumab/FOLFIRINOX + certepetide N=40 WARPNINE/Lisata Phase 1b/2a (iGoLSTA) Open-label Pending initiation Australia *Hyperthermic intraperitoneal chemotherapy Sponsors/Partners Region Indication and Test Articles Status Certepetide capital efficient development plan 24 Certepetide – Clinical / Regulatory Development Portfolio
Development Milestones 25
A wealth of anticipated key milestones 26 2Q 2023 3Q 2023 4Q 2023 1Q 2024 2Q 2024 3Q 2024 4Q 2024 1Q 2025 2Q 2025 3Q 2025 4Q 2025 1Q 2026 ASCEND [AUS, NZ] First-line mPDAC BOLSTER Trial [USA] First-line CCA CENDIFOX [USA] Pancreatic, Colon and Appendiceal Cancers Qilu: Phase 1b/2a [CHN] First-line mPDAC iLSTA [AUS] Non-resectable mPDAC Futility analysis OS data Colon/Appendiceal Cohorts PDAC Cohort Cohort A Topline data Preliminary PFS data Final PFS/OS data Final data Final 6-month PFS/OS data • PFS: Progression-free Survival • OS: Overall Survival • ORR: Objective Response Rate Enrolling First patient in Last patient in Interim Analysis Data Final data Milestone Achieved Enrolling Enrolling Enrolling Development Milestones *Several of these studies are investigator-initiated trials. Lisata has limited control and thus, timelines and expectations may be subject to change.
A wealth of anticipated key milestones (contd.) 2Q 2023 3Q 2023 4Q 2023 1Q 2024 2Q 2024 3Q 2024 4Q 2024 1Q 2025 2Q 2025 3Q 2025 4Q 2025 1Q 2026 Phase 2a [EST, LATV] First-line GBM Phase 1 [USA] Peritoneal Carcinomatosis Qilu: Phase 2 [CHN] First-line mPDAC Final PFS/OS data Tumor Penetration data Final data ORR data Enrolling Enrolling • PFS: Progression-free Survival • OS: Overall Survival • ORR: Objective Response Rate First patient in Last patient in Interim Analysis Data Final data Milestone Achieved 27 Development Milestones *Several of these studies are investigator-initiated trials. Lisata has limited control and thus, timelines and expectations may be subject to change. Enrolling
Financial Highlights 28
Capital projected to fund all clinical programs to data Financial Highlights 29 $43.3M Cash & Investments As of 3/31/2024 $0 Debt 1Q2026 Projected Cash Runway Into Common Shares Outstanding (3/31/2024): 8.3 million shares Options Outstanding (3/31/2024): Exercise Price: $0.02 - $4.22 = 1,216,100 shares Exercise Price: > $4.22 = 237,800 shares 1.5 million shares Warrants Outstanding (3/31/2024): Weighted Average Exercise Price: $42.51 1.4 million shares
Investment Thesis Rational and focused development program Highly experienced management team Derisked asset based on a body of data Fiscally stable company 30
* As of 3/31/2024; includes investments Key factors supporting investment in Lisata Therapeutics 31 Strong Investment Rationale PEOPLE Seasoned management with successful international development experience and expertise TECHNOLOGY Proprietary field-leading technology in underserved global indications MILESTONES Multiple projected product and business milestones over the next 24 months CAPITAL $43.3 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
Development Partner(s) [Development Venue] Indication and Trial Product/Comparator Stage of Development Strategic Rationale Lisata/AGITG [Australia/New Zealand] First-line mPDAC; Gemcitabine/nab-paclitaxel with certepetide or placebo Phase 2b (ASCEND) Corroborate Phase 1b results in a placebo-controlled trial and evaluate 2 dose regimens of certepetide for dose optimization Lisata [United States] First-line Cholangiocarcinoma (CCA); Gemcitabine/cisplatin/durvalumab with certepetide or placebo Phase 2a (BOLSTER) Assess certepetide safety and effectiveness in cholangiocarcinoma in a placebo-controlled trial (Proof-of-Concept) KUCC/Lisata [United States] Pancreatic, Colon & Appendiceal Cancers; FOLFIRINOX + panitumumab* with certepetide Phase 1b/2a (CENDIFOX) Tumor immuno-profiling pre- & post- treatment and certepetide effectiveness assessment in combination with chemo and an EGFR inhibitor (open label) Qilu [China] First-line mPDAC; Gemcitabine/nab-paclitaxel + certepetide Phase 1b/2a Assess safety, PK and therapeutic effect of certepetide in Chinese patients (open label) WARPNINE/Lisata [Australia] Locally advanced non-resectable PDAC; Gemcitabine/nab-paclitaxel/durvalumab + certepetide Phase 1b/2a (iLSTA) Assess certepetide safety and effectiveness in combination with IO & Chemo in locally advanced PDAC; determine if inoperable tumors can become operable (open label) WARPNINE/Lisata [Australia] Locally advanced non-resectable Gastroesophageal (GE) adenocarcinoma; Nivolumab/FOLFIRINOX + certepetide Phase 1b/2a (iGoLSTA) Assess certepetide safety and effectiveness in combination with IO & chemo in locally advanced GE AdenoCa; determine if inoperable tumors can become operable (open label) *Panitumumab may be added for colorectal or appendiceal patients without Ras mutation Appendix Certepetide capital efficient development plan 34
Development Partner(s) [Development Venue] Indication and Trial Product/Comparator Stage of Development Strategic Rationale Tartu University/Lisata [Estonia/Latvia] First-line Glioblastoma Multiforme; Temozolomide +/- certepetide Phase 2a Assess certepetide safety and effectiveness in additional tumor type (GBM) in a placebo- controlled trial UCSD/Lisata [United States] Peritoneal Carcinomatosis HIPEC* intraoperative intraperitoneal lavage + certepetide Phase 1 Assess safety and intraoperative tumor penetration of HIPEC in combination with certepetide (open label) Qilu [China] First-line mPDAC; Gemcitabine/Nab-paclitaxel + certepetide Phase 2b Continue development of certepetide in China (placebo controlled) 35 Certepetide capital efficient development plan Appendix
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 Corroborate Phase 1b results in a placebo-controlled study Determine if a second dose of certepetide further improves patient outcomes Design Phase 2b randomized, double-blind study in mPDAC testing gemcitabine + nab-paclitaxel SoC with one of two certepetide dose regimens or placebo Study Size N=158 (~30 sites in Australia and New Zealand) Endpoints Primary: Progression Free Survival Secondary: AEs, SAEs, Overall Survival, Objective Tumor Response Rate Timing Enrollment completed December 2023 Earliest possible data 2024 ASCEND: Phase 2b, blinded, randomized trial in mPDAC 36 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 2024Phase 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 37
Sponsor/Partner Qilu Pharmaceutical (funds all development in China) Objective Evaluate safety, pharmacokinetics and preliminary efficacy of certepetide added to SoC in Chinese patients with mPDAC Design 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 Study Size N=50 (~15 sites) Endpoints Primary: AEs, SAEs, Objective Response Rate, Duration of Response, Disease Control Rate, Overall Survival, and Progression Free Survival Secondary: Pharmacokinetic parameters Timing Preliminary data expected 1H23 Appendix 38 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: Preliminary data expected 1H23 *Qilu produced Phase 1b/2a open-label trial in mPDAC in China Appendix 39
Sponsor/Partner University of Kansas Medical Center (Investigator initiated trial in U.S.) KUCC funded; Lisata provides certepetide Objective 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 Design 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 Study Size N=50 (20 PDAC, 15 colon and 15 appendiceal) Endpoints Primary: Drug Safety Secondary: Overall Survival, Disease-free Survival, Overall Response Rate, RO Resection Rate, Pathological Response Rate Timing Enrollment completion target 4Q23 Data readouts possible throughout 2023 with complete results expected 2024 CENDIFOX: Phase 1b/2a open-label trial in PDAC and other cancers Appendix 40
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 4Q23; data readouts possible throughout 2023; complete results expected 2024 CENDIFOX: Phase 1b/2a open-label trial in PDAC and other cancers Appendix 41
Sponsor/Partner Lisata (U.S.) Objective Evaluate the preliminary efficacy, safety and tolerability of certepetide in combination with standards of care in subjects with first-line cholangiocarcinoma Design Phase 2 randomized, double-blind, placebo-controlled, proof-of-concept trial in first-line cholangiocarcinoma testing corresponding SoC with certepetide or placebo Study Size N=40 (1:1 SoC + certepetide or SoC + placebo) Endpoints Primary: OS Secondary: Safety, ORR, PFS Timing Trial initiation target: 2Q23 Enrollment commenced September 2023 BOLSTER: Phase 2 blinded, randomized trial in Cholangiocarcinoma Appendix 42
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-line cholangiocarcinoma • Sponsor: Lisata • Timing: Trial initiation target 2Q23 BOLSTER: Phase 2 blinded, randomized PoC trial in various cancers Appendix 43
Sponsor/Partner Qilu Pharmaceutical (funds all development in China) Objective Further evaluate safety and therapeutic efficacy of certepetide when added to SoC in Chinese patients with locally advanced unresectable mPDAC Design Phase 2b, double-blind, placebo-controlled, randomized study evaluating certepetide + SoC (Qilu-produced nab-paclitaxel and gemcitabine) vs. placebo + SoC Study Size N=120 (1:1 SoC + certepetide or SoC + placebo) Endpoints Objective response rate, progression free survival, duration of response, disease control rate, overall survival Safety Timing Trial initiation target 2Q24 Appendix 44 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 initiation target 2Q24 Phase 2 blinded, placebo-controlled trial in mPDAC in China Appendix 45
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 non-resectable pancreatic ductal adenocarcinoma (PDAC); determine if inoperable tumors can become operable Design 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 Study Size N=30 Endpoints Safety and tolerability; 28-day DLTs Objective response rate, PFS, OS, duration of response, immune cell infiltration Timing Trial initiation target 2Q23 Enrollment commenced April 2023 Appendix 46 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 Ra nd om 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: Trial initiation target 2Q23 Appendix 47 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 certepetide safety & therapeutic effect in combination neoadjuvant chemo in operable gastroesophageal (GE) cancers. Evaluate certepetide safety and therapeutic effect in combination with immunotherapy and chemotherapy for advanced non-resectable GE cancers Design Phase 1b/2a proof-of-concept, two cohort, 6 arm safety and early efficacy study of certepetide in combination with chemo as treatment in resectable GE cancers as well as in combination with chemotherapy and immunotherapy in advanced non-resectable GE cancers Study Size N=40 (20 per cohort) Endpoints Safety and tolerability Objective response rate, PFS, OS, duration of response, immune cell infiltration Timing Trial initiation target 3Q23 Appendix 48 iGoLSTA: Phase 1b/2a trial in operable/inoperable GEC with chemo & IO
N=10 NALIFIROX + Nivolumab + certepetide N=5 NALIFIROX + Nivolumab N=5 mFOLFIRINOX + Nivolumab N=10 mFOLFIRINOX + certepetide N=5 mFOLFIRINOX N=5 FLOT Operable Neo-adjuvant Locally Advanced PET CT every 8 weeks Surgery after 8 cycles Biopsy at Screening 16 weeks Biopsy Every 8-week tumor assessment until progression Phase 1b/2a proof-of-concept safety and early efficacy study of certepetide in combination with chemotherapy and immunotherapy in resectable and locally advanced non-resectable gastroesophageal cancers Gastro- Esophageal Cancers iGoLSTA: Phase 1b/2a trial in operable/inoperable GEC with chemo & IO Appendix 49
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 certepetide in combination with standard-of-care (temozolomide) in patients with previously untreated Glioblastoma Multiforme Design 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) Study Size N=40 Endpoints Safety, tolerability ORR, PFS, OS, disease control rate Timing Trial initiation target 3Q23 Enrollment commenced December 2023 Appendix 50 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 1:1 Survival Analysis 72-hour Run-in without SoC Newly Diagnosed GBM R Temodar® + certepetide matching placebo N=20 N=20 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: Trial initiation target 3Q23 Appendix 51 Phase 2a trial of certepetide with SoC in first-line in GBM
Sponsor/Partner University of California, San Diego (Investigator initiated trial) Objective Evaluate safety of certepetide in combination with hyperthermic intraperitoneal chemotherapy (HIPEC) or HIPEC alone (without certepetide) in patients with peritoneal metastases Design Phase 1 single-center, unblinded, randomized trial to determine the safety and tolerability of certepetide administered intraperitoneally in patients with peritoneal metastases from colorectal, appendiceal, or ovarian cancer undergoing Cytoreductive Surgery (CRS) and HIPEC. Participants will be randomized 2:1 to receive certepetide with HIPEC versus HIPEC alone after CRS. Study Size N=21 Endpoints Safety and tolerability PFS, OS Timing First patient treated target 4Q23 Appendix 52 Phase 1 trial of certepetide + HIPEC in Peritoneal Carcinomatosis
Safety Tumor drug concentration PFS OS CRS-HIPEC + certepetideConfirm Eligibility Informed Consent 2:1 5-year Patient Record Review for Survival Analysis Candidate for CRS-HIPEC Non- mucinous appendiceal, colorectal, ovarian carcinoma with ≥ 5 mm peritoneal nodule(s) R CRS-HIPEC N=14 N=7 A Phase I, single center, unblinded, randomized controlled trial of Intraperitoneal Certepetide in Patients Undergoing Cytoreductive Surgery and HIPEC for Peritoneal Surface Malignancy • Sponsor: Tartu University Hospital; Estonia • Funding: Lisata • Timing: Trial initiation target 4Q23 Appendix 53 Phase 1 trial of certepetide + HIPEC in Peritoneal Carcinomatosis Day 30