Panbela Therapeutics, Inc. (NASDAQ:PBLA) Q2 2022 Earnings Conference Call August 15, 2022 4:30 PM ET
Company Participants
Jennifer Simpson – Chief Executive Officer
Sue Horvath – Chief Financial Officer
Conference Call Participants
Tony Butler – ROTH Capital
Operator
Good afternoon, ladies and gentlemen. Thank you for standing by. Welcome to the Panbela Therapeutics Second Quarter 2022 Earnings Call. At this time, all participants are in a listen-only mode. After management’s prepared remarks, there will be a question-and-answer session.
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 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 Panbela. Jennifer, please proceed.
Jennifer Simpson
Thank you, and thank you, everyone, for joining. I will begin the call by touching on Q2 and our recent significant accomplishments. Sue will then follow with the review of the financial results, and then we will open it up for Q&A.
So starting with Q2 and recent highlights, since our last earnings call, we have closed on our acquisition of Cancer Prevention Pharmaceuticals or CPP for short, as we’ve shared, a combined company will have a much larger pipeline targeting approximately a $5 billion aggregate market opportunity for the areas of initial focus.
Acquiring CPP has substantially advanced our mission of treating diseases, where there are unmet medical needs, through a diversified pipeline, addressing numerous targets and thus expanding the potential of a combined company. CPP’s primary assets eflornithine or CPP-1X is being evaluated in clinical trials in combination, and in two additional forms, a tablet and a sachet.
In a Phase 3 study, the efficacy and safety of a combination of eflornithine and sulindac for Flynpovi as compared with either eflornithine or sulindac alone in adults with familial adenomatous polyposis also known as FAP was completed in 2019, a total of 171 patients under one – underwent randomization. The primary endpoint was an event driven composite endpoint, which the study failed to show a statistical difference.
In a post-hoc analysis, none of the patients in the combination arm progressed to a need for lower gastrointestinal or lower GI surgery for up to 48 months compared with 7% or 13.2% and 8% or 15.7% of the patients in the sulindac and eflornithine arms respectively. These data corresponded to risk reductions for the need for lower GI surgery approaching a 100% between combination and either monotherapy.
Given the statistical significance of the lower GI group, a new drug application or NDA was filed with the FDA. As the study fails [ph] to meet the primary endpoint and the NDA was based on the results of an exploratory analysis, a complete response letter was issued. To address this deficiency concern, we intend to submit the results of one or more adequate and well-controlled clinical trials that demonstrate an effect on a clinical endpoint.
CPP has a license agreement with One-Two Therapeutics Assets Limited or One-Two. Under the license agreement, One-Two has licensed the North American development and commercialization rights for Flynpovi. As described in the company’s IND application, CPP has transferred the IND for the product’s licensing partner through the agreement.
The agreement provided upfront payments, which were recognized by CPP and the year ended December 31, 2021. The agreement also calls for CPP receive a milestone payment upon regulatory approval of Flynpovi by the FDA and royalties on net sales of Flynpovi in the licensed territories.
Payment of the milestone payment and net sales royalties will be reduced on a dollar per dollar basis by amounts funded by the licensing partner for its direct cost associated with any development activities necessary to secure FDA approval. We expect that the FAP registration trial will be initiated by mid-2023.
We will be collaborating closely with One-Two Therapeutics as we look to finalize the development plan to seek approval for FAP in Europe. We also have an ongoing Phase 3 double-blind placebo-controlled trial of the combination of eflornithine and sulindac prevent recurrence of high risk adenomas and second primary colorectal cancers in patients with Stage 0 through 3 colon or rectal cancer.
The trial title is preventing adenomas of the colon with a eflornithine and sulindac and is also known as the PACES trial. The purpose of this study is to assess whether the combination of a eflornithine and sulindac prepared to the corresponding placebos have asked, has efficacy against colorectal lesions or total colorectal events. The PACES trial is funded by the National Cancer Institute or NCI in collaboration with the Southwest Oncology Group also known as SWOG.
Additionally, there are trials evaluating eflornithine sachet relapsed/refractory neuroblastoma supported by the Children’s Oncology Group or COG and the NCI, which is ongoing and a non-small cell lung cancer trial in the STK11 [ph] mutation patients scheduled to begin this year. For eflornithine tablets, a Phase 2 trial in type 1 onset diabetes is scheduled to begin this year.
Turning to SBP-101. In May of 2022, we were notified that the United States Adopted Names Council or USAN had adopted ivospemin as a USAN for SBP-101. After August 1 of 2022, the USAN information on ivospemin will be scheduled for posting on the USAN website.
Regarding clinical development, as a reminder earlier in the year, we announced the initiation of a double-blind placebo-controlled global randomized clinical trial for SBP-101 in combination with gemcitabine and nab-paclitaxel versus gemcitabine, nab-paclitaxel, and placebo in patients with untreated metastatic pancreatic ductal adenocarcinoma, and is referred to as the ASPIRE trial. This month, we announced expansion of the ASPIRE trial into Australia, as well as the first patient enrolled.
Moving ahead, we are planning to have approximately 90 additional sites activated by early 2023. The trial was originally designed as a Phase 2/3 with a smaller sample size of 150 patients to support the events required for an interim analysis based on progression-free survival and a primary endpoint of overall survival.
In response to European and FDA regulatory feedback, we amended the study to include the total trial sample size of 600 patients and modify the design to utilize the primary endpoint of overall survival for the interim analysis as well. PFS will also be analyzed to provide additional efficacy evidence. This amendment was supported by the final data from the Phase 1a/b first line metastatic pancreatic trial, which completed enrollment in December of 2020.
The study will enroll 600 subjects and is anticipated to take approximately 36 months for complete enrollment with the interim analysis still available in early 2024. The interim analysis allows us to assess the efficacy and safety enabling us to ensure optimal resource utilization. Also note, we have completed preclinical work necessary to begin a pancreatic cancer neoadjuvant trial. We are working with the key opinion leaders to finalize the protocol and obtain the necessary institutional approvals to open this investigator initiated trial by year end.
Turning to our work on SBP-101 for ovarian cancer, during the quarter we held an R&D Day, and we were joined by leading experts from the Sidney Kimmel Comprehensive Cancer Center at the Johns Hopkins University School of Medicine for a deep dive on SBP-101 as a polyamine metabolism modulator in ovarian cancer.
We were also pleased to have completed a poster presentation highlighting the results for SBP-101 in ovarian cancer at the American Association for Cancer Research or AACR conference, which took place April 8 through 13 of this year. The poster presentation points out that the treatment of immune competent mice injected with VDID8-positive ovarian cancer with SBP-101 has significantly prolong survival and decrease overall tumor burden.
This data supports their efforts to initiate an ovarian cancer clinical program late this year into early next year. The mature data was later published in the International Journal of Molecular Sciences titled expanded potential of the polyamine analogue SBP-101 as a modulator of polyamine metabolism and cancer therapeutics.
The publication highlighted that in vitro studies determined that SBP-101 reduced cellular viability across a broad range of cancer cell types with an exceptionally strong reduction in ovarian adenocarcinoma viability resulting in a 42% increase in median survival in the VDID8-positive ovarian cancer mouse model.
Turning to milestones, as I mentioned, we closed on the CPP acquisition and announced the first patient enrolled in our ASPIRE trial, as well as expansion outside the U.S. Additionally, in the second half of this year, we anticipate announcing the final data from our Phase 1 trial in untreated metastatic pancreatic cancer patients and the opening of the neoadjuvant pancreatic cancer investigator-initiated trial.
We also intend to initiate the ovarian cancer clinical program for SBP-101 by early 2023. With the closing of the CPP transaction, we also anticipate achievement of additional milestones for 2022 that include initiation of the non-small cell lung cancer trial with eflornithine in combination with KEYTRUDA initiation of a Phase 2 trial in Type 1 onset diabetes, and a futility analysis in the PACES trial by early 2023.
In summary, we have made tremendous progress in Q2 and year-to-date. We are excited to enhance stockholder value as we move ahead in the second half of 2022 by executing against our milestones.
I will stop here and turn it over to Sue to review the financials.
Sue Horvath
Thank you, Jennifer. General and administrative expenses were $1.3 million in the second quarter of 2022, compared to $1.2 million in the second quarter of 2021. The changes due primarily to legal fees associated with the acquisition of CPP. Research and development expenses were $20 million in the second quarter of 2022, inclusive of a one-time non-cash expense of $17.7 million. This expense was the write-off of in process research and development or IPR&D.
The company has accounted for the acquisition of CPP as an asset purchase. IPR&D represents the asset purchase and GAAP accounting requires writing off this asset immediately after the acquisition. The remaining R&D expense in the quarter of approximately $2.3 million compares to $1 million in the second quarter of 2021.
This increase in R&D expense is related to spending on our clinical studies as we ramped up efforts to activate up to 90 clinical sites around the world in the ASPIRE clinical trial. Net loss in the second quarter of 2022 was $22.1 million or $1.51 per diluted share compared to a net loss of $2.2 million or $0.22 per diluted share in the second quarter of 2021.
Total cash was $2.5 million as of June 30, 2022. Total current assets were $3.4 million and current liabilities were $6.2 million as of that same date. At June 30, 2022, total non-current assets consisting of cash deposits held by our contract research organization were $3.1 million. As a result of the CPP acquisition, we now have debt on our balance sheet, totaling $6.9 million as of June 30, 2022. This includes principle on two notes of $6.2 million and 650,000 along with accrued interest.
Both notes accrued simple interest at a rate of 5% per annum. The smaller note plus accrued interest is due at the end of the year. The remaining note totaling $6.2 million has the following payment terms. Annually, on or before January 31, 2023 through January 31, 2026, $1 million plus accrued but unpaid interest. Second, the final remaining principle plus accrued interest is due on or before January 31, 2027, a portion not to exceed the $1 million plus interest of the first payment will be prepaid if the company raises capital prior to the first payment due date.
The rationale for this acquisition of CPP is strong. Not only has the new combined entity significantly expanded our first, our total addressable market, but it also came with a fully funded registration trial for Flynpovi in FAP and collaboratively funded investigator led clinical trials in several other indications, only requiring that we provide product.
It is not expected that the acquisition will add significantly to our cash used in operations for the balance of 2022. Panbela acquired CPP via merger for non-cash consideration consisting of 7.3 million shares of common stock for upfront payment and holdback, plus 1.9 million shares subject to rights to acquire consisting of assumed options and warrants. CPP stockholders are eligible to receive contingent cash payments, totaling a maximum of $60 million payable from future milestones and royalty payments associated with the potential approval in commercialization of the CPP lead asset.
Panbela stockholders retained a majority of the outstanding shares of the post merger holding company. Looking to the cap table, we now have 20.8 million of common shares outstanding and including shares reserved for options and warrants, we were at 30.3 million shares. The shares reserved number includes all outstanding equity awards, including stock options, which were held primarily by insiders in all warrants to purchase common stock.
Our cash used in operations for the six months just ended totals approximately $8.7 million. During the first half, the company paid $2.6 million in cash deposits to our global contract research organization, which will be held to pay for clinical expenses at the end of the ASPIRE trial. This cash outlay for CRO deposits is not expected to repeat. Ramped up activity in the randomized trial and cost associated with our acquisition drove the remaining cash used in operations.
As discussed in our last earning call, we project that cash will take us into early Q4 of 2022. We will continue to focus our cash on those items in our plan, which will drive value for our stockholders, such as the ASPIRE trial. In early August, the company launched an ATM offering, enabling potential gross proceeds of up to $8.4 million. While our daily trading volume has not yet supported sales under the ATM, we intend to take advantage of its availability under appropriate conditions to assist with our operating cash needs.
That concludes my remarks. Operator, can you please open the phone lines for Q&A and poll for questions?
Question-and-Answer Session
Operator
Certainly. The floor is now open for questions. [Operator Instructions] Your first question is coming from Tony Butler with ROTH Capital. Please pose your question. Your line is live.
Tony Butler
Jennifer, thanks very much. Jennifer, this is – the question is really around ASPIRE in the 36 months to fully enroll. I understand the pace of getting sites on board and they having signed all appropriate documents, et cetera. I guess, the question is the anticipation of the pace of enrollment per site. And I’m just curious how you arrived at that 36 months, if you could provide some color on it. And importantly, if you have 150 patients for the futility, the question becomes, is it a matter of that number of patients or the 104 events? Thanks very much.
Jennifer Simpson
Sure. Thank you, Tony. So when you look at clinical trials kind of an average and certainly one – the average that we see in pancreatic cancer is to assume roughly point to patients per site per month. So that gives you an idea of one of the reasons why we have so many sites. And so we have factored that in and with the primary – with the interim analysis being overall survival, that’ll be – unfortunately, it’s a pretty finite endpoint, right?
So when we hit that number of events, we won’t need to go very far past that for our conclusion for our valuation, because the patients will either be alive or dead. So that’s one there’s – I think it’s important, because we’re saying consistent now with both the primary endpoint, as well as the interim efficacy of evaluation, right. We’re using overall survival for both, because we’re using overall survival also for the interim analysis. That’s one of the reasons that we don’t have to necessarily over enroll, because you’ll have the extra data available almost immediately.
And that really gives us the ability to have the DSMB take a look, evaluate, ensure that both the safety and the efficacy are positive so that we can ensure appropriate resource utilization. And of course, more importantly, that – if we see a benefit for the patients that we are able to continue.
Tony Butler
Thanks. Thanks, Jennifer. Appreciate that.
Jennifer Simpson
Certainly.
Operator
There appear to be no further questions in queue at this time. Thank you, ladies and gentlemen. This does conclude today’s conference call. You may disconnect your phone lines this time and have a wonderful day. Thank you for your participation.
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