Panbela Therapeutics, Inc.

Greetings. Welcome to Panbella Therapeutics' fourth quarter 2023 earnings call. At this time, all participants are in a listen-only mode. A question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. Please note this conference is being recorded. I will now turn the conference over to your host, James Carbonara, Investor Relations. James, you may begin.

Thank you, Operator. With me on the call are Jennifer Simpson, Chief Executive Officer, and Sue Horvath, Chief Financial Officer. Before I turn the call, please note that statements made on this call that are not historical facts may be forward-looking statements. Significant risks and uncertainties that could cause actual results to differ from those expressed or implied in the forward-looking statements are detailed in the company's annual report on Form 10-K, and supplemented by subsequently filed reports on Form 10-Q, as well as in other reports that the company has filed with the SEC. Any forward-looking statements made on this call are made only as of today's date, and the company does not undertake any obligation to update or supplement any such statements to reflect subsequent developments. Now, I would like to turn the call over to Jennifer Simpson, CEO of Panvala. Jennifer, please proceed.

Thank you, James, and thank you, everyone, for joining. I will begin the call with a review of our clinical development program, recent accomplishments, and upcoming milestones. Stu will then follow with a review of the financial results, and then we will open it up for Q&A. So let's start with our phase three initiative, specifically the ASPIRE Global Clinical Trial. ASPIRE is a global, randomized, double-blind, placebo-controlled study designed to assess ibospemin, or SBP101, in conjunction with gemcitabine and Mabpaclitaxel for patients with untreated metastatic pancreatic ductal adenocarcinoma. During Q4, the Independent Data Safety Monitoring Board, or DSMB, completed its second pre-specified review of safety data for treated patients, which included the first 214 patients in the trial. The DSMB has recommended that the study continue without modification. Then, in January, we surpassed 50% enrollment for the ASPIRE trial, moving faster than originally projected. As we have reached the full complement of sites open and enrolling, we have seen a steady cadence of enrollment, expecting full enrollment to be completed by the first quarter of 2025. We are looking forward to the interim data analysis based on overall survival. We originally projected this to occur in mid-2024, However, we have not seen enough deaths at this time. With patients living longer, we are evaluating the data and working to update the expected timing for the interim analysis. There also has been positive movement in the metastatic pancreatic cancer treatment landscape. Based on the NAPLI-3 trial, Onabide was approved in February for use within the Neller Fox regimen for first-line metastatic pancreatic cancer. This approval was based on a 1.9-month median overall survival benefit, 11.1 months in the treatment arm versus 9.2 months for the control arm, which was gemcitabine and abraxane. This approval is significant to Pembella for a number of reasons. It is the first approval in first-line metastatic pancreatic cancer in approximately 11 years. The 1.9-month survival benefit is similar to the approval of gemcitabine and abraxane versus gemcitabine in the MPACT trial. Additionally, for the ASPIRE trial, it helps to validate the assumed median survival of the control arm, which has not changed greatly since its original approval 11 years ago with a median survival of 8.5 months in the MPACT trial. With enrollment of the ASPIRE trial moving faster than anticipated, we look forward to the overall survival interim analysis and the completion of the trial in the hope that our product may be a potential option for patients in the future. Turning to familial adenomatous polyposis, or FAP, PAMBELLA remains committed to collaborating with the FDA, EMA, and the FAP community to move this program forward with the goal of bringing a novel treatment option for FAP patients. Once we obtain consensus on a global registration plan among the FDA and EMA, We plan to advance this initiative while upholding our established cost model. Concurrently, we intend to explore ways to maximize the value of this asset. Moving on to the PACES trial, our phase three double-blind, placebo-controlled study of Sympovy aims to prevent the recurrence of high-risk adenomas and second primary colorectal cancers in patients diagnosed with stage zero to three colorectal cancer. As a reminder, the PACEY trial receives funding from the National Cancer Institute, or NCI, and is being run by the Southwest Oncology Group, also known as SWOG. The study successfully cleared a planned futility analysis, and enrollment is complete. We expect data by the second half of 2026. Shifting gears to Phase II studies, in July, we announced an agreement with U.S. World Meds to divest assets from the Aflornitine Pediatric Neuroblastoma Program for non-dilutive payments of up to $9.5 million. Pembela has already received an initial upfront payment of $400,000 and stands to receive further payments as U.S. World Meds achieves key milestones related to aflornithine's clinical advancement, regulatory approval, and commercial sales. To that end, in December, U.S. World Meds received FDA approval of its new drug application, or NDA, for aflornithine, marking the first FDA approval of an NDA for any polyamine targeted therapy and a cancer indication. As mentioned, PAMBELLA benefits financially from this continued advancement of the program. Additionally, this approval validates the role polyamines can play in cancer therapy as we look forward to data from our ongoing programs that we are discussing today in metastatic pancreatic cancer, colorectal cancer, non-small cell lung cancer, and prostate cancer, as well as the advancement of preclinical programs in ovarian cancer and multiple myeloma. Continuing with phase two trials, in September, we entered into a clinical trial agreement for a phase two trial in castration-resistant metastatic prostate cancer, or CRPC. Leveraging preclinical models that demonstrate a potential role for polyamines in androgen-resistant prostate cancers, this clinical trial will determine if the addition of a fluornithine to the treatment regimen will demonstrate further efficacy in these difficult-to-treat patients. The study is actively enrolling. Staying with phase two investigations, the phase two trial for CPP1X, or aflornadine, is led by Indiana University School of Medicine and financially supported by the Juvenile Diabetes Research Foundation, or JVRF. Results show that DFMO or aflornadine treatment may preserve beta cell function, reflected by C-peptide levels in patients with type 1 diabetes through the modulation of urinary polyamines, in particular putrescine. There is a pressing need for the development of safe and effective treatments addressing the underlying cause of early-stage disease, and we are thrilled to champion this initiative with JDRF and the Indiana University School of Medicine. This trial continues to enroll patients. As a reminder, this study referred to as the TAGPOL study, is a multicenter, double-blind, placebo-controlled, two-to-one random assigned phase two trial for individuals with recent onset type 1 diabetes. The study is enrolling approximately 70 patients, and an interim analysis is anticipated next year. In phase one development, we have three programs that we plan to initiate. We are currently engaged in a clinical trial collaboration with Moffitt Cancer Center for a phase 1-2 program designed specifically for patients with SICK11 mutant non-small cell lung cancer. In the phase 1 trial's initial stages, our objective is to determine the maximum tolerated dose of aflornadine in conjunction with pembrolizumab while simultaneously evaluating its efficacy. Following this phase, our intention is to advance into a phase 2 trial to further assess effectiveness. The study is open and screening patients, and we anticipate enrolling our first patient in the first half of this year with plans to initiate the phase two trial in the latter half of the year. Our second phase one program, scheduled to begin in the first half of this year, will focus on evaluating ibuprofen in the platinum resistant ovarian cancer population. This endeavor underscores the ongoing collaboration between the company and Johns Hopkins University School of Medicine. Third, we are also engaged in an ongoing collaborative research initiative with the University of Texas MD Anderson Cancer Center, focusing on evaluating polyamine metabolic inhibitor therapies along CAR-T cell therapies and bispecific monoclonal antibodies in preclinical models. Recently, we announced the acceptance of an abstract detailing research on SPP-101, or IVOS-7, and CPP-1X, or flornithine, in multiple myeloma cell lines for online publication at the American Society of Hematology Meeting, or ASH meeting, and it was included in the November supplemental issue of the journal Blood. PAMBELLA's new programs place focus on the modulation of the immune system by polyamines. Our initial clinical proof of concept involves polyamine-targeted therapy combined with a checkpoint inhibitor for patients with STIK11 mutant non-muscle lung cancer. We are excited about expanding this research collaboration to explore the potential benefits of polyamines in immune modulation for hematologic malignancies. Finally, we are currently engaged in collaborative efforts with key opinion leaders to start the neoadjuvant pancreatic investigator initiative. We are in the advanced stages of securing the necessary institutional approvals to initiate the trial in the first half of this year. Turning briefly to our intellectual property or IP efforts, We strengthen our portfolio by announcing the issuance of new patents in China, Australia, and Europe. These patents cover claims of a novel process for the production of SBP101, a product developed in collaboration with FinGene International Limited. In closing, our unyielding commitment is to advance our development program for the benefit of patients worldwide. To summarize our projected milestones, We expect in the first half of 2024 to open the neoadjuvant pancreatic cancer trial, enroll our first patient in the non-small cell lung cancer phase one trial, to open the phase one ovarian trial, to announce gastric cancer prevention phase two results, publication of the final phase one 1B metastatic pancreatic trial data, and the overall survival interim analysis of the phase three Aspire trial, which we originally projected for the middle of 2024. However, with not enough events or deaths at present, we are working to evaluate the data so that we may update the projected timing. And in the second half of 2024, we anticipate to obtain feedback from the FDA and EMA for global registration in FAP and to open the non-small cell phase two trial, non-small cell lung cancer phase two trial. In summary, the fourth quarter and year to date have marked significant strides for PAMBELLA. We are enthusiastic about the ongoing creation of value for our shareholders as we progress in 2024. I will now turn it over to Sue.