Panbela Therapeutics' Phase 3 ASPIRE Trial Gets Third DSMB Safety Approval

Panbela Therapeutics Inc. recently announced the completion of its third safety review by an independent Data Safety Monitoring Board (DSMB) for its ongoing Phase 3 ASPIRE clinical trial. This trial is evaluating ivospemin combined with gemcitabine and nab-Paclitaxel for treating metastatic pancreatic ductal adenocarcinoma (mPDAC) in first-line treatment. The DSMB recommended the continuation of the trial without any modifications, marking the third consecutive positive safety review. The safety database now includes 395 patients, a significant increase from the 214 patients recorded in November 2023.

Jennifer K. Simpson, PhD, MSN, CRNP, and President & CEO of Panbela Therapeutics, expressed satisfaction with the DSMB's recommendation, noting that the lower-than-expected event rate suggests prolonged survival among patients in the ASPIRE trial. The company anticipates interim survival analysis results to be available by early 2025. This is seen as a promising development for patients, highlighting the potential of ivospemin to address the urgent medical need in mPDAC treatment.

Key points to note include the DSMB's continued support for ivospemin's safety profile, the expanded safety database now covering 395 patients, and the indication that the lower event rate could mean longer survival for trial participants. Panbela's rapid enrollment progress positions the company to complete trial enrollment by the first quarter of 2025, which is earlier than originally expected.

Panbela also emphasized the importance of the ASPIRE trial in light of other recent advancements in mPDAC treatments, such as the approval of liposomal irinotecan (Onivyde) combined with fluorouracil, oxaliplatin, and leucovorin (NALIRIFOX). Despite this recent approval, which offered a median overall survival benefit of 1.9 months over gemcitabine and nab-Paclitaxel, the prognosis for mPDAC patients remains poor, with median survival still under 12 months.

Dr. Simpson commented on the potential of adding ivospemin to the standard regimen of gemcitabine and nab-Paclitaxel to significantly improve patient outcomes, beyond the incremental benefits observed with recently approved therapies. Panbela remains dedicated to advancing this important study and anticipates sharing the interim results in Q1 2025.

Panbela continues to focus on the ASPIRE trial, aiming to evaluate ivospemin's potential to improve patient outcomes for mPDAC, a condition still associated with a median overall survival of less than 12 months despite recent treatment advancements. The company is keenly awaiting the interim survival analysis in early 2025 to gain further insights into ivospemin's efficacy in addressing this significant unmet medical need.

Panbela’s development pipeline includes assets in clinical trials targeting various cancers such as familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention, and ovarian cancer. The pipeline promises a steady flow of catalytic events ranging from pre-clinical to registration studies.

Ivospemin (SBP-101), a proprietary polyamine analogue, is designed to inhibit polyamine metabolism by targeting pancreatic ductal adenocarcinoma and other tumors. Clinical studies have shown it can inhibit tumor growth in metastatic pancreatic cancer patients, with a median overall survival of 14.6 months and an objective response rate of 48%. These figures surpass the standards set by gemcitabine and nab-Paclitaxel alone, indicating potential complementary benefits with the current FDA-approved chemotherapy regimen. Importantly, ivospemin has not exacerbated chemotherapy-related adverse events like bone marrow suppression and peripheral neuropathy.

Flynpovi™, combining CPP-1X (eflornithine) and sulindac, has shown efficacy in preventing pre-cancerous sporadic adenomas in a Phase III trial. For FAP patients, Flynpovi demonstrated statistically significant benefits in delaying surgical events compared to single agents, supporting its continued evaluation.

CPP-1X (eflornithine) is being developed for various indications, including gastric cancer prevention, neuroblastoma treatment, and recent onset Type 1 diabetes. Early studies suggest CPP-1X is well-tolerated and potentially effective.

Panbela Therapeutics is committed to developing disruptive therapies for urgent unmet medical needs, with key assets including ivospemin (SBP-101) and Flynpovi. The company's stock is quoted on the OTCQB under the symbol “PBLA.”