Q4 2022 Panbela Therapeutics Inc Earnings Call
Okay.
Good day, everyone and welcome to the <unk> Therapeutics fourth quarter 2022 earnings call.
At this time, all participants have been placed on a listen only mode and we will open the floor for your questions and comments after the presentation.
It is now my pleasure to turn the floor over to your host James Carbonara, Sir the floor is yours.
Thank you and once again welcome to <unk> fourth quarter 2022 earnings call 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 Companys annual report on Form 10-K, and supplemented by the subsequently filed quarterly reports on Form 10-Q.
As well as in other reports that the company.
From time to time 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.
Subsequent developments now I would like to turn the call over to Jennifer Simpson CEO of Penn Bella Jennifer. Please proceed.
Thank you and thank you everyone for joining I will begin the call with a review of our clinical development program recent accomplishments and upcoming milestones.
Steve will then review the financial results and we will open it up for Q&A.
Starting with our phase III programs in November we enrolled our first patient in Europe for our aspire global clinical trial on the first line treatment of metastatic pancreatic cancer.
As a reminder, aspire is a global randomized double blind placebo controlled clinical trial to evaluate Ivo salmon or S. E. T 101 in combination with Gemcitabine and Nab paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma.
We're really excited after just recently receiving regulatory approval for the opening of trial sites in Europe . So we have had our first patient enrolled in Italy.
Having European enrollment underway is highly encouraging as we continue to ramp up the trial.
We continue global enrollment and just last week enrolled our first patient in South Korea.
We are focused on the country and plate and initiation.
Your trial as we aim to have the full complement of sites on board by the middle of this year.
Having south Korea underway. It's also encouraging as we continue to advance the trial.
With approximately 95 five plan throughout the United States, Europe , Australia, and South Korea, we are continuing to focus on site initiation enrollment.
Ultimately deliver a more effective treatment for pancreatic cancer.
Deadly disease with few treatment options.
The trial sample size is approximately 600 patients and the primary endpoint will be overall survival.
We will use overall survival for the interim analysis as well.
It is anticipated to take approximately 36 months to complete enrollment with the interim analysis still available in early 2024.
The interim analysis allows us to assess the merits of continuing the study as well as to ensure optimal resource utilization.
We are excited to have these recent approvals as we move forward towards the interim analysis.
And a little over one year from now.
On a related note in January the European Medicines agency or EMA Committee for orphan medicinal products has issued the adoption of commission implementing decision relating to the designation of Idaho sermon FTP 101, as an orphan medicinal product in combination with Gemcitabine.
And Nab paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma.
The designation of orphan drug status within the EU is an important achievement as we continue to advance the global aspire trial with the potential that this may be an option for patients with first line metastatic pancreatic cancer in the future.
Orphan drug designation in the European Union is granted by the European Commission based on the positive opinion issued by the EMA Committee for orphan medicinal products.
The EMA orphan designation is available to companies developing treatments for life, threatening or chronically debilitating condition that affect fewer than five and 10000 persons in the E U.
Medicines that meet the EMA orphan designation criteria may qualify for financial and regulatory incentive.
Including a 10 year period of market exclusivity in the EU after product approval.
Protocol assistance from the EMA reduce fees during the product development phase and access to centralized marketing authorization.
Turning to familial adenomatous polyposis or at the registration trial with some Tobey it's anticipated to begin in the second half of 2023, and it's funded by our licensing partner once you therapeutics.
We are working closely with once you to ensure alignment with the FDA on all key aspects of the trial before the trial commences.
As we anticipate that the trial will utilize European clinical sites and we retained the rights to Toby outside of the U S and Canada, we will seek advice from European authorities regarding the utilization of the registration trial for approval in Europe .
As a reminder, our prior phase III study evaluating a quarantine them to them back or when Kobe.
Either agent alone showed a 100% risk reduction in the need for surgery and the lower gastrointestinal group.
As there are currently no approved drug therapies for the treatment of S E T.
Glad I was explaining in a lower Gi group is intended to be a focus of the new registration trial.
Lastly, we in partnership with once you also have an ongoing phase three double blind placebo controlled trial of from Tobi again, the combination of our quantity insulin dock to prevent recurrence of high risk Adenomas and second primary colorectal cancers in patients with stage, three colon and rectal cancer.
This trial known as the pace trial is funded by the National Cancer Institute or the MTI in collaboration with the southwest Oncology group also known as swaps and we look forward to a futility analysis in the first half of 2023.
Moving to phase two studies first that there is an ongoing trial in relapsed refractory neuroblastoma uniform utilizing our Florida team caches.
This trial is funded through the children's oncology group or Cogs and the NCI.
We also announced the start of a phase two double blind randomized study to evaluate or anything tablet for recent onset type one diabetes in collaboration with Indiana University School of Medicine, and funded by J D. O S, leading global type one diabetes research and advocacy organization.
Indiana University School of Medicine, we expect to enroll 70 patients in the phase two clinical trial at approximately six centers in the United States.
Study eligibility will be for patients with recent onset type one diabetes.
Participants will be randomized two to one to affirmative tablets administered orally with food twice daily at a 1000 milligram per meter squared dose or placebo over six months, followed by a six month washout period to assess durability of response.
The primary objective will be to determine the difference between the treated and placebo to our area under the curve or AUC mean, using the la Nina C peptide at the six month end of treatment period.
Secondary objectives will include C peptide AUC fasting and stimulated.
Excuse me accumulated pro insulin C peptide ratios, a biomarker of beta cell stress and the urine pining contact at three nine and 12 month time point.
We are pleased to have begun the phase two trial for our Florida and tablets with Indiana University School of Medicine, and J D or F.
Which is the leading global organization advancing life changing breakthrough for type one diabetes.
Approximately 1.45 million Americans living with type one diabetes.
In the United States and there is approximately a 16 billion dollar type one diabetes associated health care expenditure and lost income.
Our realized annually.
Moreover, less than one third of people with type one diabetes in the U S are consistently achieving target blood glucose control levels.
We are excited to move forward with a phase two trial, along with India in Indiana University School of Medicine, and J D are off to provide better treatment options for this patient population.
In phase one development, we have three programs that we will be starting.
First a quantity sashays will be evaluated in combination with keytruda and the strict 11 mutation population of non small cell lung cancer.
I think 11 mutant non small cell lung cancer respond poorly to checkpoint inhibitors and bio informatic analysis suggests that alterations in polymer metabolic pathways may play a role in the resistance to immunotherapy.
In preclinical tumor models of Florida think treatment improved anti PD, one efficacy by increasing tumor specific cytotoxic T cell population as well as increased expression of PD, one on tumor associated CDA positive T cells.
The phase one portion is fully funded and we look forward to the trial starting as it will be the first clinical proof of concept trial focused on the relationship between poly means immune system.
If this trial is positive and opens the possibility for combining with a checkpoint inhibitor in other tumors as well as the possibility of combining with other immunotherapies such as car T therapy to improve response rates.
Our second Phase one program, which is scheduled to begin in the first half of 2023, we'll focus on the evaluation of Ivo stemming in the platinum resistant ovarian cancer population.
As a reminder, we collaborated with Johns Hopkins University School of Medicine to identify this indication.
Additionally, a poster on the treatment of immune competent mice injected with beat it eight positive ovarian cancer with Irish famine was presented and showed a significantly prolong survival and decrease in overall tumor burden.
The mature data with a lot of with later published in the International Journal of molecular sciences titled Extra.
Expanded potential.
The timing analogue SVP 101, as a modulator of Piney metabolism and cancer therapeutics.
The publication highlighted that in vitro studies determine the F. B P 101 reduced cellular viability across a broad range of cancer cell type with an exceptionally strong reduction in ovarian adenocarcinoma viability, resulting in a 42% increase in median survival and the data eight positive ovarian cancer.
Your mouse model.
We recently announced the acceptance of the abstract titled evaluating the efficacy of affirming analog Irish woman SPP went to one in combination with chemotherapy in ovarian cancer for poster presentation at the American Association for clinical research or ACR.
Meeting, which is occurring April 14th through the 19th of this year.
The data supports our efforts to initiate an ovarian cancer clinical program.
Lastly, we have completed the preclinical work necessary to begin to pancreatic cancer Neo adjuvant investigator initiated trial, we are working with the key opinion leaders to finalize the protocol and obtain the necessary institutional approvals to open this trial in the first half of this year.
To recap the milestones as we continue to execute our development program. We anticipate the opening of the non small cell lung cancer. It's taken a lot of mutation phase one trial.
The ACR poster presentation of the ovarian preclinical work.
A futility analysis for the swab colon cancer risk reduction trial in the first half of this year.
The opening of the Neo adjuvant trial in ovarian cancer trial.
The phase III trial to open in the second half of this year.
Phase one non small cell lung cancer data in the second half of this year, which one form the phase two portion of the non small cell lung cancer trial, which we hope to open by year end.
Final data from the phase one programs in first line metastatic pancreatic cancer and early onset type one diabetes.
And finally, the interim analysis of the aspire trial in early 2024.
In summary, we have made tremendous progress in Q4 and year to date, we are excited to enhance stockholder value as we move ahead in 2023 and onward by executing against our milestones I will stop here and turn it over to Steve to review the financials.
Thank you Jennifer.
General and administrative expenses for $1.7 million in the fourth quarter of 2022 compared to $1 3 million in the fourth quarter of 2021 the.
The increase is due to severance cost associated with the acquisition and integration of cancer prevention pharmaceuticals or CPP.
Research and development expenses were $3 5 million in the fourth quarter of 2022 compared to 2 million in the fourth quarter of 2021. The increase is due to spending on our clinical studies as we ramped up efforts activate up to 95 clinical sites around the world and the aspire clinical trial.
As a reminder, as a result for the year ended December 31, 2022 include approximately $17 7 million of in process research and development otherwise known as IP R&D.
R&D represents the asset we purchased in the acquisition of CPP and GAAP accounting required writing off this asset to research and development expense immediately after the acquisition in quarter and the quarter ended June 30th.
Net loss in the fourth quarter of 2022 was $4 7 million or $5.68 per diluted share compared to a net loss of $3 5 million or $10.64 per diluted share in the fourth quarter of 2021.
On January 13th 2023, we affected a reverse stock split at a ratio of one for 40 shares of the company's common stock all share and per share amounts of our common stock presented here and in our report 10-K have been retroactively adjusted to reflect the war.
For 40 reverse stock split.
In Q4 of 2022, the company completed the sale of common stock and warrants for gross proceeds of approximately $6 million.
As of December 31, 2022, total cash was approximately $1 3 billion.
Also as of that date total current assets were $1 8 million and current liabilities were $7 8 million.
At December 31, total non current assets, consisting primarily of cash deposits held by our contract research organization with $3 2 million.
Our cash number as of December 31, 2022 does not include gross proceeds of approximately $15 million from our public offering which was priced and closed in early Q1 of 2023.
As a result of the C. P. P X ish acquisition, we had debt and accrued interest on our balance sheet totaling approximately $7 2 million as of December 31 2022.
This includes two notes with principal balances of $6 2 million and 650000, along with accrued interest.
<unk> notes accrue simple interest at a rate of 5% per annum.
Subsequent to December 31, 2022, two payments were made to noteholders per the terms of the notes.
After payment of the principal balance on the first dose was now $5 2 million in the second note was paid in full.
Looking to the cap table as of December 31, 2022, we had approximately 1 million common shares outstanding including shares reserved for options and warrants we were at approximately 2 million shares.
The shares reserved number includes all outstanding equity awards, including stock options, which were held primarily by insiders and all warrants to purchase common stocks.
That's reflected in the Form 10-K, we filed earlier today as of March 13, 2023, we were just under $14 6 million shares of common stock outstanding.
As of that same date, an additional four 8 million shares were reserved for the exercise of outstanding options and warrants.
We cannot be sure.
When or whether holders of these warrants may elect to exercise any or all of the remaining warrants if the trading price for our common stock is less than the exercised price of any warrants, we believe holders would be unlikely to exercise for cash.
Our cash used in operations for the year ended 2022 totaled approximately $15 3 million during the year. The company paid $2 6 million and cash deposits to our global contract research organization, which will be held to pay clinical expenses at the end of the ASP.
Fair trial.
Ramped up activity in the randomized trial and costs associated with their acquisition drove the remaining increase in cash used in operations.
We project that cash with the Q1 'twenty two 'twenty three capital raise will take us into early Q3 2023.
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.
Operator can you. Please open the phone lines for Q&A and poll for questions.
Sure <unk>.
Time will be conducting a question and answer session. If you have any questions or comments. Please press star one on your phone at this time.
We do ask them what posing a question. Please pickup your handset if you're listening on speaker phone to provide optimum sound quality.
Once again, if you have any questions or comments. Please press star one on your phone please.
Please hold while we poll for questions.
Your first question is coming from Jonathan Aschoff from Roth.
Your line is live.
Hi, Thank you Hi, Jennifer ensue sounds like you've got a full plate.
I have a specific question about the strategy.
The phase III futility analysis, Brooklyn, Povey colon cancer risk prevention.
At this analysis goes from Povey need to be numerically superior to.
To the Controle to proceed.
Or does it just need to be performing within home you know plus.
Plus or minus five or 10% margin goal to control at interim to pursuit.
So Jonathan Thank you so much for your question and we actually will have two registration program. So the first in F. P. We are still finalizing that trial design. So that is the one that we'll use some tobey. So once that that design is locked up and we've had the.
Locked it with the input from the FDA with our partner and then we can certainly look to answer questions along those lines our.
Or the aspire trial you know this is again a.
Randomized double blind placebo controlled trial and the D. S. M. B will have the oversight. So obviously if the sponsor we will not know the results I'm only the D. F N B will know that.
And the threshold in there I I will have to look I do not believe that has been put.
Put out into the public so according to the S. E T. Within the protocol that is what will be SMB will follow them.
But it is a pretty.
Conservative.
Hurdle, if you will knowing the patient population and knowing that it's important for us to ensure two things one is that you.
The patients are truly getting a benefit.
And the second that as a company.
We are utilizing our resources appropriately.
So that's one good net analysis does happen we will get.
The.
The thumbs up or the hold noticed from the SMB and that is something that we would announce.
Okay.
Regarding the early 2024.
I will spend the interim phase III analysis.
Metastatic PD.
Is that for futility, just like a phone Kobe analysis or is that for something else where data will be disclosed.
Yeah, So again for the.
For the aspire trial, because it's a double blind.
Placebo controlled.
The only the F&B will know the data for the aspire trial. So they will basically just tell us to proceed or stock.
Based on the clinical analysis plan and the data that they are reviewing.
Okay, and you don't wish to make a public comment on the stringency kind of like my first question for this futility analysis, either right, yeah, I mean, well so.
The when I was talking about the stringency of I was referring to the inspire trial because we don't have the final design for the trial yet.
That is something that our licensing partner as you know we are working with them, but we need the feedback from the FDA and so we do not have a final protocol yet so can't comment on an interim analysis or stringency at this point for the S. E T trial or the aspire trial, though absolutely.
We did make sure that it was a a reasonable hurdle that gives us confidence that this is truly doing something for patients.
That is first and foremost the most important thing for us as a company.
And then again it also translates into utilizing our resources appropriately.
Okay. Lastly, you know what kind of treatment effect are you looking for viable sperm into demographics.
At the final analysis, you know to compel a market uptake.
Yeah, you know so if if you if that's an interesting question actually because when you depending on who you talk to your right clinicians regulatory bodies.
I think the kind of you know.
Base base case would be at least a three month improvement in survival. You know when you look at the approval of a chairman of Brac thing.
Over jumps out of being alone.
You went from roughly $6 seven to about $8 five.
We certainly believe that we would like to see a more meaningful benefit of at least three months.
This is an area that really is in need of options, but we are anticipating to see at least a three months that'll that'll obviously higher would be great. Thank.
Thank you very much.
Currently.
Yeah.
Thank you that concludes our Q&A session. Everyone. This concludes today's event you may disconnect at this time and have a wonderful day.
Thank you for your participation.
Yeah.