Panbela Therapeutics Q1 2024 Earnings Results & Report

Panbela Therapeutics EPS Results

• Actual EPS: -$2.28
• Consensus EPS: -$1.23
• Beat/Miss: Missed by -$1.05
• One Year Ago EPS: N/A
• EPS Beat Rate: 1 of last 4 quarters

Panbela Therapeutics Revenue Results

• Actual Revenue: N/A
• Expected Revenue: N/A
• Beat/Miss: N/A
• YoY Revenue Growth: N/A

Panbela Therapeutics (OTCMKTS:PBLA) is a biotechnology company focused on developing novel therapies for cancer and other serious diseases. The company’s research has centered on treatments that target polyamine metabolism, a biological process associated with tumor growth and cancer progression.

Panbela’s lead investigational product has been ivospemin, formerly known as SBP-101, which has been studied in combination with standard treatments for pancreatic ductal adenocarcinoma and other solid tumors. The company has also pursued eflornithine-based programs, including combinations intended to support cancer treatment and prevention strategies.

Panbela’s programs have primarily been in clinical development and have been evaluated through research and clinical-trial activities in the United States. As an investigational biotechnology company, its product candidates have not represented approved commercial therapies unless separately authorized by applicable regulators.

Panbela Therapeutics Q1 2024 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: ASPIRE Phase III enrollment has passed 50% with ~600 patients expected by Q1 2025; the interim overall survival analysis (≈170 events, 33% of 512) is now projected as early as Q1 2025 and a lower-than-expected event rate suggests prolonged patient survival.
• Negative Sentiment: Cash runway is constrained — cash on hand was about $262k at March 31, 2024 (after a $8.1M January raise), Q1 net loss was $7.1M and cash used in operations was $9.4M, and the company’s NASDAQ listing was suspended (now on OTCQB), so additional financing/uplist is needed.
• Neutral Sentiment: Multiple programs and near-term milestones are underway — PACES (Flynpovi) enrollment is complete with data expected H2 2026, the FAP program is pursuing a global registration plan with regulators, and several Phase II/I trials (eflornithine in prostate and type 1 diabetes, planned ovarian trial, NSCLC studies) are active or planned.
• Positive Sentiment: Preclinical, IP, and collaborations bolster the pipeline — AACR data show ivospemin plus doxorubicin improved outcomes in immunocompetent ovarian models, the company announced a U.S./Canada patent for eflornithine+sulindac, and ongoing partnerships with major cancer centers support development.

Presentation

[Operator]

Good afternoon, everyone, and welcome to the Panbela Therapeutics First Quarter 2024 earnings call. At this time, all participants have been placed on a listen-only mode, and we will open for questions following the presentation. If anyone should require operator assistance during this conference, please press star zero on your phone keypad. Please note, this conference is being recorded. I will now turn the conference over to your host, James Carbonara, Investor Relations. James, over to you.

[James Carbonara, Head of Investor Relations at Panbela Therapeutics]

Thank you, operator. Joining me on today's call are Jennifer Simpson, Chief Executive Officer, and Sue Horvath, Chief Financial Officer. Before we begin, please note that statements made during this call that are not historical facts may be considered forward-looking statements. Risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such forward-looking statements are detailed in the company's filings with the SEC. Any forward-looking statements made on this call speak only as of today's date, and the company does not undertake any obligation to update or revise any of these statements to reflect future events or circumstances. With that, I will turn the call over to Dr. Simpson. Jennifer, please go ahead.

[Jennifer Simpson, CEO at Panbela Therapeutics]

Thank you, James, and thank you all for joining us. I will start by discussing our clinical development programs, recent achievements, and upcoming milestones. Then Sue will review our financial results before we open up the call for Q&A. Let us begin with our Phase III ASPIRE global clinical trial. ASPIRE is evaluating ivospemin or SBP-101, in combination with gemcitabine and nab-paclitaxel for patients with untreated metastatic pancreatic ductal adenocarcinoma. We were excited to announce in January that the ASPIRE enrollment had surpassed 50%, proceeding faster than initially anticipated. With all sites now open and actively enrolling, we expect full enrollment of approximately 600 patients to be completed by the first quarter of 2025. With respect to interim data, the ASPIRE trial requires 33% of the total expected events to occur before the interim analysis can be conducted.

[Jennifer Simpson, CEO at Panbela Therapeutics]

As of the latest assessment, less than half of the required events for the interim analysis had occurred. While we initially anticipated the interim analysis to take place in mid-2024, we are encouraged by the lower-than-expected event rate, which suggests that patients in the ASPIRE trial have experienced prolonged survival. We are now projecting the interim analysis to occur as early as first quarter 2025. This is a positive development for patients and underscores the potential of ivospemin in addressing a significant unmet need in the treatment of metastatic pancreatic ductal adenocarcinoma. We also want to reiterate the significance of the ASPIRE trial in the context of recent advancements in metastatic pancreatic ductal adenocarcinoma treatment, such as the NAPOLI-3 trial, which led to the approval of liposomal irinotecan, or Onivyde, in combination with fluorouracil, oxaliplatin, and leucovorin, known as the NALIRIFOX regimen.

[Jennifer Simpson, CEO at Panbela Therapeutics]

Despite this approval, which was based on a median overall survival benefit of 1.9 months compared to gemcitabine and nab-paclitaxel, the prognosis for patients with metastatic pancreatic ductal adenocarcinoma remains poor, with median overall survival still less than 12 months. The incremental benefits in median survival have been modest in the past 11 years, with the recent approval of Onivyde in the NALIRIFOX regimen demonstrating the 1.9-month survival benefit compared to the approval of gemcitabine and nab-paclitaxel, which was based on a median overall survival benefit of 1.8 months over gemcitabine alone. We believe that the addition of ivospemin, or SBP-101, to the standard of care regimen of gemcitabine and nab-paclitaxel has the potential to significantly improve outcomes for patients with metastatic pancreatic ductal adenocarcinoma beyond the incremental benefits observed with the recently approved therapy.

[Jennifer Simpson, CEO at Panbela Therapeutics]

The early indications from the ASPIRE trial support this belief, and we remain committed to advancing this important study and look forward to sharing the interim results in the first quarter of 2025. Turning to our familial adenomatous polyposis, or FAP, program, we remain committed to collaborating with the FDA, EMA, and the FAP community to advance this initiative. Once we obtain consensus on a global registration plan, we intend to move this program forward while exploring ways to maximize its value. In our PACES trial, a Phase III study of Flynpovi for the prevention of high-risk adenomas and second primary colorectal cancers, enrollment is complete, and we anticipate data by the second half of 2026. This study, funded by the NCI and conducted by the Southwest Oncology Group, known as SWOG, successfully passed a planned futility analysis. Moving on to our Phase II studies.

[Jennifer Simpson, CEO at Panbela Therapeutics]

In September, we entered into an agreement with U.S. WorldMeds to divest assets from the eflornithine pediatric neuroblastoma program. Panbela has received an upfront payment of $400,000, with an additional $775,000 received in lieu of U.S. sales milestones in the latter years. Panbela stands to receive additional payments as U.S. WorldMeds achieves key milestones. As a reminder, in December, U.S. WorldMeds received FDA approval of its NDA for eflornithine, marking the first FDA approval of a pioneering targeted therapy in a cancer indication. This approval not only benefits Panbela financially, but also helps to validate the role polyamines can play in cancer therapy as we advance our other programs. We also entered into a clinical trial agreement for a Phase II trial of eflornithine in castration-resistant metastatic prostate cancer. This study is actively enrolling.

[Jennifer Simpson, CEO at Panbela Therapeutics]

The Phase II eflornithine trial in type one diabetes is a multicenter, double-blind, placebo-controlled, 2-to-1 randomized clinical trial led by Indiana University School of Medicine and supported by JDRF. All six centers are open and enrolling patients with an anticipated interim analysis next year. We are also planning a Phase II study evaluating ivospemin in the platinum-resistant ovarian cancer, which we hope to begin in the second half of this year in collaboration with Johns Hopkins University School of Medicine. In fact, last month at the American Association for Cancer Research, or AACR's annual meeting, we presented a poster highlighting the efficacy of ivospemin as a polyamine metabolism modulator in ovarian cancer.

[Jennifer Simpson, CEO at Panbela Therapeutics]

Ivospemin reduces the viability of human ovarian adenocarcinoma cell lines regardless of their platinum sensitivity, and we found that the combination treatment with doxorubicin increases median survival, delays tumor onset, and decreases overall tumor burden compared to either clinical or subclinical doxorubicin dosing schemes. The results suggest that SBP-101 in combination with doxorubicin may have a role in the clinical management of ovarian cancer, in particular, the difficult-to-treat platinum-resistant population, where few options exist. These studies continue to support the basis for moving into a clinical trial program in ovarian cancer, with a goal of developing effective novel therapeutics in combination with standard of care for patients with unmet medical needs. The poster provided additional details on the preclinical studies. Treatment with doxorubicin significantly increased the in vitro toxicity of SBP-101 in both cisplatin-sensitive and cisplatin-resistant ovarian cancer cell lines.

[Jennifer Simpson, CEO at Panbela Therapeutics]

SBP-101 and doxorubicin cooperatively increased polyamine catabolism and decreased overall cell survival in vitro. In an immunocompetent mouse model of ovarian cancer, the combination of SBP-101 and doxorubicin significantly increased median survival time, delayed ascites formation, and decreased overall tumor burden. Interestingly, this survival benefit was not observed in the immunodeficient mice, indicating that an intact immune system is required for the efficacy of this therapy. These promising preclinical results provide a strong foundation as we prepare to initiate our ovarian cancer clinical program later this year. Future studies will evaluate ivospemin in combination with other polyamine metabolism modulators, as well as immune modulators. In Phase I development, we have two programs. Our Phase I/II collaboration with Moffitt Cancer Center, non-small cell cancer, is open and screening.

[Jennifer Simpson, CEO at Panbela Therapeutics]

We anticipate enrolling our first patient first half of this year and initiating the Phase II trial in the first half of 2025. Finally, we are looking to initiate the ROAR Investigator Initiative in the second half of this year. In the preclinical setting, we are also engaged in an ongoing research initiative with MD Anderson Cancer Center, focusing on evaluating polyamine metabolic inhibitor therapies with CAR T-cell therapies and bispecific monoclonal antibodies in preclinical models. We recently announced the acceptance of an abstract detailing research on SBP-101 and CPP-1X eflornithine in myeloma cell lines for online publication at the ASH meeting. On the IP front, we recently announced the issuance of a new patent in the U.S. and Canada. This patent is for claims of a fixed-dose combination of eflornithine and sulindac.

[Jennifer Simpson, CEO at Panbela Therapeutics]

In summary, our projected first half 2024 milestones include enrolling our first patient in the non-small cell lung cancer Phase I trial, announcing gastric cancer prevention Phase II results. In the second half of this year, we anticipate opening the neoadjuvant pancreatic cancer trial, opening the Phase II ovarian trial, publishing the final Phase I/1b metastatic pancreatic trial data, and obtaining FDA and EMA feedback for a global registration trial in FAP. And in 2025, early on, we anticipate overall survival interim analysis for our Phase III ASPIRE trial in the first quarter and the opening of the non-small cell lung cancer Phase II trial in the first half of the year. The first quarter and year to date have marked significant clinical development strides for Panbela. We look forward to continued progress and value creation in 2024.

[Jennifer Simpson, CEO at Panbela Therapeutics]

I will now turn the call over to Sue to discuss our financial results. Sue?

[Sue Horvath, CFO at Panbela Therapeutics]

Thank you, Jennifer. General and administrative expenses were approximately $1.2 million in Q1 2024, compared to $1.4 million in Q1 2023. This decrease was primarily due to lower professional fees in 2024. Research and development expenses were approximately $5.5 million in the quarter ended March 31st, 2024, up from $3.5 million in the same quarter last year, the increase due primarily to increased enrollment in the ASPIRE trial. Net loss for the quarter was $7.1 million, or $2.28 per diluted share, compared to a net loss of $5.1 million, or $392.76 per diluted share in Q1 2023.

[Sue Horvath, CFO at Panbela Therapeutics]

Total cash as of March 31st, 2024, was approximately $260,000, which includes net proceeds of approximately $8.1 million from our public offering, which closed in January. Total current assets were $1.8 million, and current liabilities were $10.5 million at quarter end. Non-current assets, consisting primarily of cash deposits held by our CRO, were $8.7 million. During the quarter, $1 million of the current portion of our debt, plus accrued interest of approximately $260,000, was paid per the terms of the note. Regarding our capitalization, as of March 31st, 2024, we had approximately 4.85 million common shares outstanding, including shares reserved for options and warrants. Our issued and fully reserved share count was approximately 13.95 million shares.

[Sue Horvath, CFO at Panbela Therapeutics]

Cash used in operations for Q1 of 2024 totaled approximately $9.4 million. Cash used in operations included our net loss for the quarter, payments made to vendors for expenses incurred in 2023, and approximately $0.9 million for prepayments made to secure additional supply of standard of care chemo agents. In January, we closed a $9 million public offering with net proceeds of approximately $8.1 million. On April 28, 2024, the company signed an amendment to our 2023 agreement with U.S. WorldMeds. In exchange for a second non-refundable payment of approximately $0.8 million, the company has agreed to give up two potential future payments associated with future milestones. This non-dilutive payment was received by the company at the signing of the amendment.

[Sue Horvath, CFO at Panbela Therapeutics]

Per the amended terms, total potential payments remaining, if these milestones are achieved, is approximately $7.6 million. During the quarter, Panbela's common stock was suspended from trading on NASDAQ. It is now eligible for quotation on the OTCQB under the symbol PBLA. The company is pursuing a new listing of its common stock on a national securities exchange. Operator, we are now ready to take questions.

[Operator]

Thank you very much. We are now conducting our question and answer session. If you would like to ask a question, please press star one on your phone keypad now. A confirmation tone will indicate that your line is in the queue. You may press star two if you would like to remove your question from the queue. For any participants using speaker equipment, it may be necessary to pick up your handset before you press the keys. Please wait a moment while we poll for questions. Thank you. Your first question is coming from Jonathan Aschoff of Roth MKM. Jonathan, your line is live.

[Jonathan Aschoff, Managing Director and Senior Research Analyst at Roth MKM]

Thank you. Good afternoon. I was wondering, you know, can you tell us the stopping criteria for the hopefully 1Q25 ASPIRE interim, you know, for both for efficacy and for futility? Can you just close that?

[Jennifer Simpson, CEO at Panbela Therapeutics]

Hi, Jonathan. How are you today? The interim analysis is based on approximately 33% of the total events. The total events that we're looking for is 512, so basically about 170 events. It is actually a superiority, and so it would, in terms of, stopping, I think, you know, it's probably unlikely that we would be stopping. And at that point, the reality is we will have most likely completed enrollment.

[Jonathan Aschoff, Managing Director and Senior Research Analyst at Roth MKM]

And so-

[Jennifer Simpson, CEO at Panbela Therapeutics]

We had a very interesting thing happening, right? So enrollment was much faster than we anticipated, and the patients are living longer than anticipated, and so the two created an interesting dynamic by which they're both going to hit about the same time.

[Jonathan Aschoff, Managing Director and Senior Research Analyst at Roth MKM]

Okay, you know, maybe can you tell us about anything about alpha spend, just some sort of, some sort of statistic hurdle we can think about?

[Jennifer Simpson, CEO at Panbela Therapeutics]

Yeah. So, you know, the alpha spend is minimal. It really, it's, it's de minimis in terms of the spend, and we designed it that way. The study is powered at 90%, with a hazard ratio of 0.75 overall, and an alpha of 0.025 one-sided. So the alpha spend, for the interim analysis, is actually quite small.

[Jonathan Aschoff, Managing Director and Senior Research Analyst at Roth MKM]

Okay. You know, and do you think that the 1Q25 timing is at risk, or are the events occurring, you know, that are occurring, still strongly tracking to 1Q25?

[Jennifer Simpson, CEO at Panbela Therapeutics]

At this time, I'd say we're still tracking to Q1 2025. If we do start to see slippage and we think it's going to be even later, we certainly will update the public. But at this point, we are still on track for our first quarter.

[Jonathan Aschoff, Managing Director and Senior Research Analyst at Roth MKM]

Okay. Thank you very much for the update.

[Jennifer Simpson, CEO at Panbela Therapeutics]

Certainly.

[Operator]

... Thank you very much. Just as a reminder, if you do have any remaining questions, you can press star one on your phone keypad now. Your next question is coming from Joe Pantginis of H.C. Wainwright. Joe, your line is live.

[Analyst at H.C. Wainwright]

Hi, this is Josh on for Joe. I was just wondering if we could get a little bit more insight into the current cash position, and I was wondering if there's any steps that you may be taking to help strengthen the cash position?

[Sue Horvath, CFO at Panbela Therapeutics]

Sure. Well, we did raise a portion of the cash that we needed in the first quarter in January, but certainly not sufficient to keep up for our burn rate. Right now, last year, we were burning close to $6.5 million per quarter, if we excluded the Abraxane that we were required to purchase. That is the enrollment increases, that burn rate will probably jump in closer to $7 million per quarter. You might have noticed a filing of an S-1 that was completed in April. It would be our intention, as we look to find a national exchange to uplist on, to also raise the capital that's identified in that S-1.

[Joe Pantginis, Managing Director of Equity Research at H.C. Wainwright]

Okay. And I just wanted to verify if I heard this correctly. The $262,000 cash, that does include the net proceeds from the January raise?

[Sue Horvath, CFO at Panbela Therapeutics]

It was. Yes, that was included in there because those proceeds were received in January, but we burned on top of that, you know, with a loss in the quarter of $7 million. So that leaves us with a balance at the end of the quarter. What it did not include was the incremental upfront payment that we received from U.S. WorldMeds in April, which was basically a bridge to help us to our next step in fundraising.

[Joe Pantginis, Managing Director of Equity Research at H.C. Wainwright]

All right. Perfect. Thank you so much.

[Operator]

Thank you very much. Just a reminder, if there are any remaining questions, you can press star one on your phone keypad now. Okay, we don't appear to have any further questions in the queue, and I will now conclude today's conference call. You may disconnect your phone lines at this time and have a wonderful day. Thank you for your participation.

Participants

Executives

• James Carbonara, Head of Investor Relations
• Jennifer Simpson, CEO
• Sue Horvath, CFO

Analysts

• Jonathan Aschoff, Managing Director and Senior Research Analyst at Roth MKM
• Analyst at H.C. Wainwright
• Joe Pantginis, Managing Director of Equity Research at H.C. Wainwright