Panbela Therapeutics, Inc.

And welcome to the PanBella Therapeutics third quarter 2023 earnings call. At this time, all participants are in a listen-only mode and a question and answer session will follow the formal presentation. You may enter the queue anytime by pressing star then one on your keypad. Should you wish to remove yourself from queue, you can press star then two. Please note this conference is being recorded. I will now turn the conference over to your host, James Carbonara, Investor Relations at PanBella. 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 over to Dr. Simpson, 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 quarterly 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 Pembella. 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. We 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 Trials. ASPIRE is a global, randomized, double-blind, placebo-controlled study designed to assess ibospemin, or SCP-101, in conjunction with gemcitabine and nabpaclitaxel for patients with untreated metastatic pancreatic ductal adenocarcinoma. During Q3, we initiated enrollment in both the UK and Germany, successfully activating all intended countries for the ASPIRE trial, which are now actively enrolling patients. Additionally, In July, the Independent Data Safety Monitoring Board, or DSMB, completed its scheduled safety review for treated patients in the trial and recommended the study's continuation without any changes. This achievement, alongside the full activation of all countries and DSMB's green light, provides substantial encouragement as we propel the trial forward. We anticipate interim data around mid-year 2024. Addressing familial adenomatous polyposis, or FAP, Pambella is dedicated to working with the FDA, EMA, and the FAP community to move this initiative forward, aiming to introduce a novel treatment option for FAP patients. Upon securing consensus on a global registration plan from the FDA and EMA, we intend to progress this endeavor while adhering to our existing cost structure. Simultaneously, we'll assess avenues to optimize the value of this asset. Shifting focus to the PACES trial, it's our Phase III Double-Blind Placebo-Controlled Study of Sympovy. This trial aims to prevent the recurrence of high-risk adenomas and second primary colorectal cancers in patients diagnosed with stage 0 to 3 colorectal cancer. The PACES trial receives funding from the NCI and is being performed in conjunction with the Southwest Oncology Group, also known as SWOG. The study's purpose is to assess the combination of aflornithine and Sulandac in reducing the three-year event rate of adenomas and second primary colorectal cancers in patients previously treated for stages zero through three colon or rectal cancer. We've successfully cleared a planned futility analysis and will continue the study. Transitioning to phase two studies, in July we disclosed that we would be receiving a total of up to $9.5 million in non-dilutive funding through the divestiture of assets from the Aflornithine Pediatric Neuroblastoma Program to U.S. World Meds. We look forward to collaborating with U.S. World Meds in the ongoing FDA review of a new drug approval. PAMBELLA 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. This agreement not only broadens our range of partner-supported programs, but also holds the potential for significant development milestone payouts. We warmly welcome U.S. World Meds to our network of partners dedicated to advancing our product candidates. One final item on this program and partnership. After the quarter ended, the FDA's Oncology Drug Advisory Committee, or ODAC, voted that there was sufficient evidence to conclude aflornadine, or DFMO, reduces the risk of relapse in pediatric patients with high-risk neuroblastoma who are in remission and have completed multi-agent, multi-modal therapy. This recommendation is the first-ever ODAC approval supported by a clinical trial that relied on an external control arm using patient-level data extracted from another trial. The FDA stated that the high unmet medical need for patients with neuroblastoma, the specific external control data source, and the results of the propensity score matched analysis impacted their willingness to consider an external control design in this circumstance. We view this ruling as additional validation of the potential clinical efficacy and safety of the drug and value that we expect to derive from its continued development. Staying with Phase II trials, in September, we entered into a clinical trial agreement for a Phase II trial in castration-resistant metastatic prostate cancer, or CRPC. The goal of the androgen and polyamine elimination alternating with Xtandine or APEX trial will be to evaluate if treatment with a combination of DFMO or Florentine and high dose testosterone will improve the prostate specific antigen or PSA response rate in patients with metastatic CRPC compared with historical controls. The study is actively enrolling. 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 fluorine to the treatment regimen will demonstrate further efficacy in these difficult-to-treat patients. If the trial is successful, it opens the door for combining polyamine-targeted therapies, such as a fluorine and ibuprofen, with bipolar androgen therapy, or VAT therapy, as a new treatment option for metastatic CRPC. There is a huge unmet need for new therapies for castration-resistant metastatic prostate cancer patients. Previous clinical studies demonstrated the efficacy of VAT as a monotherapy and its ability to sensitize to subsequent androgen inhibition. Three clinical studies showed that superphysiological levels of androgen can upregulate ornithine decarboxylase suggesting a role of aflornithine as a combination therapy. The trial will determine if modulating polyamines can enhance the efficacy of VAP therapy in these patients. Continuing with Phase II investigations, the Phase II trial for CPP1X or aflornithine, overseen by Indiana University School of Medicine and financially supported by the Juvenile Diabetes Research Foundation, or JDRF, continues to enroll patients. JDRF stands as the foremost global entity propelling transformative breakthroughs for type 1 diabetes. We are enthusiastic about backing the newly launched phase 2 trial for early stage type 1 diabetes run by Indiana University School of Medicine and funded by JDRF. The objective is to create innovative and effective treatments for patients facing significant medical challenges. This trial, based upon the recently published phase 1 trial and preclinical data, in the journal Cell Reports Medicine investigated the mechanism of polyamines and polyamine inhibition by CPP1X on beta cell stress that plays a role in the onset of type 1 diabetes. Results showed that DFMO or a fluoroethanol 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. According to SIMS et al., although therapy of type 1 diabetes has improved, the morbidity, mortality, and cost continue to impact the quality of life for those affected, highlighting the need for safe and effective therapies that address the underlying pathology. In phase 1 development, we have three programs that we will be starting. We have an ongoing clinical trial partnership with Moffitt Cancer Center, focusing on a phase 1-2 program tailored for patients with STIC-11 mutant non-small cell lung cancer. Initially, the phase one trial aims to establish the highest tolerated dose of aflornadine while also assessing its effectiveness. Subsequently, we plan to progress into a phase two efficacy trial. We expect to have data from the phase one trial early next year with the intention to commence the phase two trial immediately following completion of the phase one trial. Concurrently, our second phase one program that's commenced later this year or early next year will center on the assessment of ibuprofen in the platinum-resistant ovarian cancer demographic. This effort exemplifies the company's continuous partnership with Johns Hopkins University School of Medicine. Additionally, we have an ongoing collaborative research effort with the University of Texas MD Anderson Cancer Center. focusing on the evaluation of polyamine metabolic inhibitor therapies in conjunction with CAR-T cell therapies using preclinical models. This research aims to determine whether treatments involving a fluorine and or ibospemin can enhance CAR-T-induced cytotoxicity against CD19-positive large B-cell lymphoma cell lines. A metabolite panel predominantly consisting of polyamines was identified as a predictor of limited response to anti-CD19 CAR-T cell therapy in cases of relapsed refractory large B-cell lymphoma. Moreover, there is an observed upregulation of the polyamine uptake transport system in both large B-cell lymphoma and multiple myeloma. These findings collectively indicate the potential for targeted polyamine therapy in conjunction with CAR-T therapies. Recently, we announced that an abstract about SBP101, or ibospemin, and CPP1X, or aflornithine, research in multiple myeloma cell lines has been accepted for an online publication on the American Society of Hematology, or ASH, meeting site and will be printed in the November supplemental issue of the journal Blood. Pembella places significant emphasis on polyamine modulation of the immune system. Our initial clinical proof of concept involves polyamine therapy combined with a checkpoint inhibitor for STIC11 mutant non-flossful lung cancer patients. We are enthusiastic about extending this research collaboration to assess the potential advantages of polyamines in immune modulation for hematologic malignancies. Lastly, we're in the process of collaborating with key opinion leaders to complete the protocol for the neoadjuvant pancreatic investigator initiative We're also in the final stages of obtaining the required institutional approval to launch this trial by the end of the year. Briefly turning to IP, we fortified our intellectual property portfolio, announcing the issuance of new patents in China and Australia for claims of a novel process for the production of SBP 101, developed in collaboration with Syngene International Limited. Additionally, we announced the issue of a new patent in Chile for claims of a novel process for the production of Lympovy, developed in collaboration with Sanopy. In closing, despite a very challenging biotech market, we are committed to advancing our development program for the benefit of patients around the world. To summarize our projected milestones, as we continue to progress with our development initiative, we foresee the initiation of a neoadjuvant pancreatic cancer trial and an ovarian cancer trial by end of year early next year, We anticipate the phase one non-small cell lung cancer data early next year, which will guide the phase two segment of the non-small cell lung cancer trial expected to be initiated immediately following the phase one trial. Our focus for the FAP program is to obtain feedback from the FDA and EMA for global harmonization on a registration protocol. Additionally, we anticipate data on our polyamine metabolic inhibitors in combination with CAR T therapy in preclinical lymphoma and multiple myeloma models. Finally, we look forward to the interim analysis of the ASPIRE trial mid-year 2024. To sum up, Q3 and year to date, we have seen remarkable progress. We are eager to continue creating value for our shareholders as we move ahead in the coming months and into 2024. I will now turn it over to Sue.