8-K: Lisata Therapeutics Reports Positive Preliminary Phase 2b Data for Certepetide in Metastatic Pancreatic Cancer, Reinforcing Confidence for Phase 3

Lisata Therapeutics announced promising preliminary Cohort B data from its Phase 2b ASCEND trial, showing improved progression-free survival and objective response rates for certepetide in metastatic pancreatic cancer, paving the way for a global Phase 3 study.

Summary

• Lisata Therapeutics, in collaboration with AGITG and the University of Sydney, announced positive preliminary Cohort B data from the Phase 2b ASCEND trial evaluating certepetide with standard-of-care (SoC) chemotherapy in metastatic pancreatic ductal adenocarcinoma (mPDAC).
• The preliminary Cohort B data showed a six-month progression-free survival (6MPFS) of 60.8% for the certepetide-treated group compared to 25% for the placebo-treated group.
• Median progression-free survival (mPFS) in Cohort B was 7.5 months for the certepetide group versus 4.7 months for the placebo group, nearing statistical significance (HR 0.6, p=0.08).
• The objective response rate (ORR) in Cohort B was 45.2% for the certepetide group and 19% for the placebo group, with 50.0% of certepetide-treated patients having a complete or partial response compared to 21.1% in the placebo group (excluding unknown/early withdrawal/death), indicating statistical significance.
• Median overall survival (mOS) in Cohort B numerically favored certepetide at 10.32 months compared to 9.23 months for placebo.
• Cohort B data corroborates earlier Cohort A data, which showed a positive trend in overall survival (12.68 months vs. 9.72 months for placebo) and four complete responses in the certepetide group versus none in the placebo group.
• Certepetide demonstrated an attractive safety profile, with adverse events comparable across treatment groups and reflecting toxicities of concomitant cytotoxics and the disease under study.
• The company has completed an End-of-Phase 2 FDA meeting, with agreement reached on the Phase 3 study protocol and development plan, and preparations for a global Phase 3 trial are underway.
• Lisata Therapeutics projects its current capital will fund operations into the fourth quarter of 2026, encompassing anticipated data milestones from ongoing and planned clinical trials.

Sentiment

Score: 9

Explanation: The document conveys a highly positive outlook, driven by strong preliminary clinical trial results for a drug targeting a severe disease with high unmet need. The data shows clear improvements in key efficacy endpoints (PFS, ORR) and a favorable safety profile, reinforcing confidence for advancing to a global Phase 3 trial. The company's solid financial position and multiple regulatory designations further contribute to the positive sentiment.

Positives

• Preliminary Cohort B data from the ASCEND trial showed a significantly higher 6-month progression-free survival (60.8% vs. 25% for placebo) and median PFS (7.5 months vs. 4.7 months) for certepetide.
• Objective response rate (ORR) in Cohort B was substantially higher for certepetide (45.2% vs. 19% for placebo), demonstrating a statistically significant improvement.
• Median overall survival (mOS) in Cohort B numerically favored certepetide (10.32 months vs. 9.23 months), corroborating clinical benefit.
• Certepetide-treated subjects stayed on treatment longer than those on placebo (5.00 months vs. 3.22 months).
• The safety profile of certepetide is attractive, with adverse events comparable to placebo, reflecting the safety of the standard-of-care chemotherapy.
• Preliminary Cohort A data also showed a positive trend in overall survival (12.68 months vs. 9.72 months for placebo) and four complete responses in the certepetide group.
• Previous Phase 1b/2a trials (CEND1-001 in Australia and CEND1-201 in China) demonstrated that certepetide plus SoC chemotherapy improved overall survival in mPDAC, with improvements of 31% and 55% respectively, compared to SoC alone.
• Certepetide has received Fast Track Designation (U.S.) and Orphan Drug Designation for pancreatic cancer (U.S. and E.U.), malignant glioma (U.S.), osteosarcoma (U.S.), and cholangiocarcinoma (FDA), which can expedite development and provide market exclusivity.
• Rare Pediatric Disease Designation for osteosarcoma (U.S.) makes certepetide eligible for a Priority Review Voucher upon approval, which has significant monetary value.
• The company has a strong intellectual property portfolio with global IP protection extending beyond 2040, potentially to 2046 with patent term extensions.
• Lisata has $25.8 million in cash and investments as of March 31, 2025, with no debt, providing a projected cash runway into Q4 2026.
• The company has established noteworthy commercial and R&D partnerships, including Qilu Pharmaceutical (China), Kuva Labs (diagnostics), Catalent (ADCs), and GATC Health Corp., validating its CendR Platform technology.

Risks

• Results observed from preliminary data are not necessarily indicative of final results, and clinical outcomes may materially change following more comprehensive reviews of the data and as more patient data becomes available.
• Product candidates that appeared promising in early research and clinical trials may not demonstrate safety and/or efficacy in larger-scale or later clinical trials.
• The safety and efficacy of Lisata's product candidates are subject to ongoing evaluation.
• Decisions of regulatory authorities and the timing thereof, as well as the duration and impact of regulatory delays in Lisata's clinical programs, could affect development timelines.
• Lisata's ability to finance its operations and the likelihood and timing of the receipt of future milestone and licensing fees are subject to various factors.
• The future success of Lisata's scientific studies and its ability to successfully develop and commercialize drug candidates are uncertain.
• The timing for starting and completing clinical trials is difficult to predict.
• Rapid technological change in Lisata's markets could impact its competitive position.
• Lisata's ability to protect its intellectual property rights is crucial.
• Legislative, regulatory, political, and economic developments could affect the company's operations and prospects.
• Timelines and expectations for investigator-initiated trials, where Lisata has limited control, may be subject to change.

Future Outlook

Lisata Therapeutics plans to expedite the investigation of certepetide as a novel therapeutic agent for metastatic pancreatic cancer, with full study data from both ASCEND cohorts expected later this year. The company has agreed on a global Phase 3 study design with the FDA, targeting 657 patients with overall survival as the primary endpoint, and preparations are underway. Lisata also anticipates numerous product and business milestones over the next 1.5 years across its diverse clinical development portfolio for various solid tumors and non-oncologic applications.

Management Comments

• "We are pleased with the promising Cohort B data of the ASCEND trial. These data, taken with those previously reported for Cohort A, reinforce our confidence in the therapeutic promise of certepetide. Along with its attractive safety profile, we continue to believe that certepetide has the potential to transform the treatment landscape for mPDAC and many other devastating solid tumors." David J. Mazzo, PhD, President and Chief Executive Officer of Lisata.
• "The data from ASCEND provides us with critical new knowledge that will significantly enhance our understanding of how to optimally treat patients battling pancreatic cancer. We are excited by the evidence of certepetides therapeutic effect and encourage the continued development of this potentially treatment paradigm-changing compound." Dr. Andrew Dean, Study Chair.

Industry Context

Pancreatic cancer has one of the poorest prognoses among cancers, ranking as the 6th leading cause of cancer mortality worldwide and the 3rd leading cause in Australia. With a five-year survival rate of just 13%, there is a considerable and urgent need for new, more effective treatment options. Current solid tumor treatments often face challenges due to tumor morphology, the immunosuppressive tumor microenvironment (TME), and off-target side effects, leading to suboptimal patient outcomes. Certepetide aims to address these challenges by enhancing drug penetration and modifying the TME, potentially transforming the treatment landscape for mPDAC and other solid tumors where immunotherapies are generally ineffective.

Comparison to Industry Standards

• The median overall survival (mOS) of 11.1 months for certepetide + SoC in the CEND1-001 Australian trial and 13.2 months in the CEND1-201 Chinese trial compares favorably to the 5.5 months mOS reported for standard gemcitabine + nab-paclitaxel in the Von Hoff 2013 Phase 3 registration study, which established the SoC.
• The mOS of 10.32 months for certepetide in ASCEND Cohort B, while preliminary, is numerically higher than the 9.23 months for placebo and aligns with the separation observed in the NAPOLI-3 NALIRIFOX Phase 3 study data, suggesting a clinically meaningful benefit.
• The objective response rate (ORR) of 45.2% for certepetide in ASCEND Cohort B is significantly higher than the 19% for placebo, indicating a substantial improvement over SoC alone in achieving tumor shrinkage or disappearance.
• Certepetide's ability to potentiate a complete response in metastatic gastroesophageal adenocarcinoma (mGEAC) when added to FOLFIRINOX, radiotherapy, and pembrolizumab, resulting in a sustained complete response for over 33 months, highlights its potential beyond pancreatic cancer and in combination with immunotherapies, where many solid tumors remain resistant.

Stakeholder Impact

• **Shareholders:** Positive preliminary clinical data and a clear path to Phase 3 could significantly increase shareholder value and investor confidence. The strong cash runway into 4Q2026 reduces immediate dilution risk.
• **Patients:** The promising results for certepetide offer hope for improved treatment outcomes for patients suffering from metastatic pancreatic cancer, a disease with a very poor prognosis and high unmet medical need.
• **Employees:** Positive clinical progress and a robust development pipeline provide job security and potential for growth within the company.
• **Healthcare Providers:** If approved, certepetide could provide a new, more effective treatment option to improve patient care for mPDAC and potentially other solid tumors.
• **Regulatory Authorities:** The positive data and clear development plan will be closely scrutinized by regulatory bodies like the FDA and EMA as the drug progresses towards potential approval.

Next Steps

• Full study data from both Cohort A and Cohort B of the ASCEND trial are anticipated to be available later this year.
• Dr. Andrew Dean is scheduled to present the preliminary Cohort B findings at the European Society for Medical Oncology (ESMO) Gastrointestinal Cancers Congress on July 2, 2025.
• Preparations for a global Phase 3 clinical trial for certepetide in first-line mPDAC are underway, following agreement on the study design with the FDA.
• A second EMA scientific advice meeting is planned for Autumn 2025.
• Continued development of certepetide in other solid tumors and non-oncologic applications through ongoing and planned Phase 1b/2a studies (BOLSTER, CENDIFOX, iLSTA, GBM trial).
• Targeting first-in-human dosing for the GATC-1021 asset for Opioid Use Disorder in 1H 2026.
• Anticipating numerous product and business milestones over the next 12-18 months.

Key Dates

Recommendation

Keywords

Certepetide, Pancreatic Cancer, Metastatic Pancreatic Ductal Adenocarcinoma, mPDAC, ASCEND Trial, Phase 2b Clinical Trial, Oncology, Chemotherapy, Progression-Free Survival, Objective Response Rate, Overall Survival, Clinical-Stage Pharmaceutical, Solid Tumors, Drug Development, Biotechnology, SEC Filing, 8-K, LSTA