Q4 2023 Theriva Biologics Inc Earnings Call

Participants

Steven A. Shallcross; CEO, CFO, Treasurer, Corporate Secretary & Director; Theriva Biologics, Inc.

Presentation

Operator

And greetings, and welcome to the Theriva Biologics full year 2023 investor conference call.
At this time, all participants are in a listen only mode. As a reminder, this conference is being recorded. I would now like to turn the call over to your host Chris Calabrese with LifeSci Advisors. Thank you. You may begin.

Thank you, operator, and good morning, everyone. Welcome to the Theriva Biologics full year 2023 investor conference call. Leading the call today will be Steven Shallcross, Chief Executive and Chief Financial Officer of Theriva Biologics; Dr. Medel, Qwest guy of General Director of the RevA biologics, European subsidiary, and Dr. Vince wager, Head of Corporate and Product Development of Spiriva biologics are also on the call and will be available to answer questions during the Q&A session.
The review Biologics issued a press release this morning, which provided operational highlights and included the financial results for the full year ending December 31, 2023. The press release can be found in the Investors section of the Company's website at w. w. w. dot Areva bio.com, together with the annual report on Form 10-K for full year ended December 31, 2023, which we plan to file today with the Securities and Exchange Commission. In addition to the phone line, this call is being streamed live via webcast, which will be archived on the Company website, w. w. w. dot Areva bio.com for 90 days.
During this call, certain forward-looking statements regarding Theriva Biologics and DCM Biosciences, current expectations and projections about future events will be made. Generally, the forward-looking statements can be identified by terminologies such as may, should, expects, anticipates, intends, plans, believes, estimates and similar expressions. These statements are based upon current beliefs, expectations and assumptions and are subject to a number of risks and uncertainties, including those set forth in three-V Biologics' filings with the SEC, many of which are difficult to predict.
No forward-looking statements can be guaranteed, and actual results may differ materially from such statements. The information on this call is provided only as of the date of this call and to review. Biologics undertakes no obligation to update any forward-looking statements contained on this conference call on account of new information, future events or otherwise, except as required by law.
With that, I'd like to turn the call over to Steve.

Steven A. Shallcross

Thank you, Chris, and good morning. I appreciate everyone for taking the time to join us today. In 2023, we continued to make steady progress to drive forward our oncology forward focused portfolio designed to address unmet needs for difficult to treat cancers. Our primary efforts and resources are focused on pursuing multiple therapeutic opportunities for our lead clinical candidate, VCN-01.
As a reminder, VCN-01 is a systemically administered oncolytic adeno virus designed to selectively replicate within the tumor, the grade the tumor matrix increase tumor immunogenicity. We believe these multiple modes of action position, VCN-01 for optimized tumor killing in combination with chemotherapy and immuno-oncology products and otherwise refractory solid tumors.
We have shown that repeated systemic dosing of VCN-01 is feasible from a safety perspective, and we can now focus on whether the repeated dose VCN, Reg VCN-01 regimen may lead to improved clinical outcomes for patients beyond VCN-01, we are pursuing new oncolytic virus candidates to leverage our novel human shield technology, which is designed to protect systemically administered oncolytic viruses from the host immune system and may facilitate more frequent repeated administration of oncolytic virus therapies. This movement may enable our pipeline of products to be used in standardized treatment cycles that are well established in cancer, chemotherapy and immunotherapy.
Additionally, as part of our oncology focus portfolio. We continue to screen and enroll patients in the second cohort of the Phase Ib IIa clinical trial of sin for design to prevent potentially fatal adverse outcomes in patients who undergo alginate committed periodic cell transplant ACT. to treat hematologic cancers. With our cash runway into the first quarter of 2025, we believe we're well positioned to execute on our corporate objectives and remain on track to achieving multiple value-enhancing milestones.
With this brief introduction, I'd like to expand on key pipeline updates. Starting with our lead program. VCN-01 BCL. one has been administered to more than 100 patients across diverse indications, which speaks to the broad therapeutic potential, including pancreatic ductal adenocarcinoma or PDX retinol BLISS, stoma, head and neck squamous cell carcinoma, colorectal cancer and ovarian cancer.
VCN-01 has been granted orphan drug designation in the US and Europe for the treatment of pancreatic cancer and in the US for retinol, but Soma, providing additional opportunities for regulatory engagement and if approved market exclusivity, our most advanced program for VCN-01 is in PDX for which incidence continues to rise in an indication that has one of the lowest survival rates among all cancers.
It is well established that the pediatric tumor matrix is one of the key reasons for the overall poor therapeutic outcomes for these patients. We believe the CNO one's differentiated mechanism of action has the potential to address the urgent need for new treatment options for patients with PTAC. by degrading the twofer matrix and increasing tumor access by co administering cancer therapies. We are pleased to report that dosing is well underway for Viraj, our Phase 2b trial of VCN-01 in combination with standard of care chemotherapy, gemcitabine and nab-paclitaxel, which is being evaluated as a first-line therapy for patients with PDX with six sites open in the US and nine sites opened in Spain. Faraj remains on track to complete enrollment in the first half of 2024.
In the first quarter of 2024, we completed the first safety review with the Independent Data Monitoring Committee or IDMC with a positive recommendation from the IDMC. Faraj will continue to enroll patients without any changes to the protocol. Notably, intravenous VCN-01 has been well tolerated and demonstrated a safety profile consistent with prior clinical trials.
Importantly, no additional toxicities were observed in patients receiving a second dose of VCN-01, providing the first clinical evidence of the feasibility of repeated systemic dosing. As a reminder, primary endpoints for the Virage trial include overall survival and BCNO. one safety and tolerability. Additional endpoints include progression-free survival objective response rate measures of VCN-01 biodistribution replication, immune response and measures of the quality of life of treated patients. Since this is an open-label trial, progress will be monitored very closely in steps to accelerate the clinical program may be implemented if supported by emerging data.
More broadly, the Virage trial will enable us to determine the feasibility of repeated dosing of VCN-01 This could shift the approach to standardize treatment cycles that are well established in cancer chemotherapy and immunotherapy and may lead to improved clinical outcomes for patients with PDX and other difficult to treat solid tumors.
In addition to advancing the Virage PDN trial, we continue to work closely with key opinion leaders in the US, Europe and Central and South America, as well as with regulatory agencies to refine our clinical strategy in Renovo stoma. We believe intravitreal VCN-01 has the potential to treat vitreous seeds in children with retinol Bluestone.
Since current clinical practice varies in, there's no regulatory guidance specific to retinol plus stoma drug development. We held a pre IND meeting with the FDA in the fourth quarter of 2023 to discuss the development pathway for VCN-01 is an adjunct to chemotherapy in pediatric patients with advanced renal milestones. During our meeting with the FDA, we were provided some guidance on the potential endpoints in patient population for an advanced clinical trial and encouraged to submit a formal protocol under a US IND.
In order to provide a more detailed commentary for this program, we are encouraged by interactions with the FDA and look forward to driving this program forward.
In parallel with company-sponsored studies, the potential utility of VCN-01 is being explored and a number of investigator-sponsored studies that are underway at leading oncology research institutions around the world, notably collaborators at St. Johns and Dale have completed patient treatment in the Phase one investigator sponsored trial evaluating the safety and activity of intravitreal BC and one in pediatric patients with refractory retinol Bluestone.
The trial evaluated escalating doses of VCN-01 administered by two intravitreal injections separated by 14 days and remains on track to complete patient follow-up in the first half of 2024, which will help to inform the planned Phase 2 trial and design and the protocol. As a reminder, preclinical data has shown that topotecan treatment enhanced BCNO. one oncolytic activity against rentable stoma and more broadly reinforced VCN-01 possibility as an adjunct to intravitreal chemotherapy in patients who fail currently available treatments. We remain encouraged by the potential of this novel combination approach to provide superior clinical benefits for children with this devastating cancer.
Additionally, the University of Pennsylvania continues to enroll and treat patients in their Phase one investigator sponsored trial, administering VCN-01 with your CAR T muscle cells to patients with ovarian or pancreatic cancers. VCN-01 designed to increase tumor immunogenicity and improve access by additional therapies such as UCargo muscle cells while cell-based immunotherapies have had limited efficacy against solid tumors to date, we are encouraged by initial results highlighting the feasibility of administering VCN-01 with this type of CAR T therapy. These preliminary results were presented last year at the Society for Immunotherapy of Cancer Annual Meeting or cities. European investigators are continuing to explore the optimal dosing regimen for VCN-01 co-administered with your card muscle cells, and we look forward to further data from the study in 2024.
Turning to our ongoing Phase 2b 2a clinical trial of Washington University evaluating same for or write-backs. The trials designed to evaluate the therapeutic potential of sin for two fatal to reduce fatal adverse events related to IV beta-lactam antibiotic use and allogeneic HCT recipients, including acute graft versus host disease or AGVHD. in overgrowth and infection by pathological organisms such as C. difficile and Vanco myosin resistant and tear Koksay. The Phase 1b 2a study is designed to assess the feasibility of using sense for and consist of three sequential cohorts comparing different IV beta-lactam antibiotics following conditioning therapy in each cohort eight patients will receive and four and four receive placebo. While the data remain blinded, interim analysis suggests it's in four is well tolerated and was not observed in the blood samples of a majority of development patients.
Our second cohort is underway and is designed to evaluate SIN for combination with piperacillin tazobactam. The trial is on track to complete enrollment in the second cohort in the second quarter of 2024. This cohort will provide important additional safety information. In particular, whether oral sema four has the potential to alter IV antibiotic levels in this patient population. We look forward to sharing this data in the second half of 2024.
Overall, we are encouraged by the progress across our pipeline in the growing clinical data that underscore the promise of our systemically administered oncolytic virus in key indications and combinations. We remain focused on driving our clinical programs forward in exploring opportunities to leverage our novel albumin shield technology and exciting additional technologies from Rovi discovery platform. I'm confident that the Company's upcoming catalysts will provide a solid foundation for execution and value creation.
Specifically we remain on track to complete enrollment for the Virage study in the first half of 2024, complete follow-up in the Phase 1 investigator sponsored trial evaluating the safety and activity of intravitreal, the VCN-01 in pediatric patients with refractory reasonable stoma and the first half of 2024 and complete enrollment in the second cohort of our Phase 1b 2a clinical study of seven four for the prevention of AGVHD. in bone marrow transplant patients in the second quarter of 2024.
Now I briefly turn to our financial results for the first full year ended at December 31, 2023, general and administrative expenses decreased to $7.1 million for the year ended December 31, 2023 from $9.9 million for the year ended December 31, 2022. This decrease of 28% is primarily comprised of the decrease in the fair value of the contingent consideration of $2.8 million, along with lower salary, investor relations, legal costs, consulting fees related to the VCN. acquisition and director and officer insurance, offset by higher audit fees and other consulting fees.
The charge related to stock-based compensation expense was $0.4 million for the year ended December 31, 2023 compared to $0.4 million for the year ended December 31, 2022. Research and development expenses increased to $14.3 million for the year ended December 31, 2023 from $11.7 million for the year ended December 31, 2022.
This increase of 22% is primarily the result of higher clinical trial expenses related to our Virage Phase 2 clinical trial of VCN-01 and PDx offset by lower expenses related to our Phase 1b 2a clinical trial of sin for an allogeneic HCT recipients. The completed phase one, a clinical trial have seen 20 decreased manufacturing expenses related to our Phase 1a clinical trials in 20 and lower other indirect costs.
We anticipate research and development expense to increase as we continue enrollment in our Virage Phase two clinical trial of VCN-01 and P. deck in our ongoing Phase 1 clinical trial and renewable stoma, expand GMP manufacturing activities for VCN-01 and continue supporting RBC and 11 on other preclinical and discovery initiatives.
Research and development expense also includes a charge related to noncash stock-based compensation expense of $165,000 for the year ended December 31, 2023, compared to $112,000 for the year ended December 31, 2022. Other income was $1,442,000 for the year ended December 31st, 2023, compared to other income of $471,000 for the year ended December 31, 2022. Other income for the year ended December 31, 2024, was primarily comprised of interest income of $1,439,000 and an exchange gain of $3,000. Other income for the year ended December 31, 2022 is primarily comprised of interest income of $512,000, offset by an exchange loss of $41,000.
Cash and cash equivalents totaled $23.2 million as of December 31, 2023, compared to $41.8 million as of December 31, 2022. We remain deeply committed to improving patient outcomes for these very hard-to-treat cancers.
And before we conclude today's call, I want to extend my sincere appreciation and gratitude for the foundational work that has brought us closer to delivering on our mission.
I'd like to thank the entire Theriva team, our investors and the many people who have been supportive along the way, including our patients and their families.
With that, we're happy to take questions.

Question and Answer Session

Operator

Thank you. And at this time, we'll be conducting a question and answer session. (Operator Instructions)
James Molloy, Alliance Global Partners.

Yes, good morning. Thank you very much for taking my questions. Pillow had a question looking below Stephen, Alex, Christopher, just even your mental on looking across the multiple IST.s, which are very cost efficient way of getting trials done excellent. Excellent use of capital, which you guys see as sort of most promising of like all the volatile and of course, of which seems like it might be maybe growing the task. We look across the thrust of the plethora of IST as you guys have got underway.

Steven A. Shallcross

So let me let me just highlight where what we've got going on once again. And then Manav, you could talk specifically about some of the exciting findings that we found of had disclosed at various scientific conferences. So as you're aware, we put out some incredible head and neck cancer data at asthma last October. This is using BC and one in combination with checkpoint inhibitors in patients that were refractory to previous rounds of checkpoint inhibitor therapy on someone else could talk a little bit about that.
We've got the retinol Bluestone, the program on which we've just concluded, and now we're in the middle of follow-up. And then we've got the ongoing New Penn study, which is down to of their organization, trying to isolate the right dose take forward. So we would expect to see some more of data sometime this year related to the work they're doing there.

So now maybe you could just highlight the important findings on those three studies that are that are ongoing or recently completed?

Yes, sure, sure. So obviously, the muscle complete data, let's say, from the trial in head and neck had a good percentage of asthma in October as you know, this was a trial combining VISION one with the development and efficient refractory to the action of immune checkpoint inhibitors in head and neck carcinoma.
Okay. And we have previously presented safety data that demonstrated that additional one has an acceptable safety profile when administered prior to durvalumab. And in this new presentation that we conducted in October, we presented data on efficacy.
And we have seen that the patients treated with Vision Show with an increase of restaurants, two, a subset of chemotherapy treatments after progressions and style. But specifically, we have also seen that the survival of patients has been large for a number of patients. And in fact, some patients are still alive more than four years after we didn't participate in the trial, which in my view, it's quite remarkable because those patients were entering the trial had some effect on shelf life of between three or four months. And what we are seeing obviously, it's really interesting.
And moreover, it's not just a question of efficacy that does a very good correlation between the restaurants and the biological data we are getting from the tumor biopsies of these patients. And in fact, we have seen down-regulation of the more massive genes. We have been observing increasing levels of immune markers in the tumor biopsies. And interestingly, there was a correlation between the survival and with the CPS score and a day after vision one for investigator scoring, it's a kind of marker about immunological status and what looks in our biological, the that you said that vision one treatment change in this environment and definite correlation between the magnitude of the change that we use and the survival of patients, which is I think exciting.
Obviously, it's use a proportion, but it's exciting in regional customer. We have been treating more patients. We have seen some patients with reductions that we do see it. But specifically, we have been also collecting data of combination with the chemotherapy, the chemotherapy that is using rational estimate, it's topotecan. And in this trial, we have seen not derailing the clinical patients. But in preclinical work that we have done with biopsies and from a human cells, we have been observing that there is synergy between the action of this in one and a couple of Beacon, which is very encouraging and opens the door for a combination approach in our Phase two program that is massively with cash and with FDA in our meeting.
Okay.
So basically, the data we are collecting, it's very exciting. And in my view, it's very exciting to see that in different indications, we can confirm the mechanism of action. And we can see initial evidence of activity in happening in RightNow customer, which is also the same thing that we have observed in our Phase one program in pancreatic cancer so that the data is quite consistently that way.

Thank you very much. Taking my quick follow-up on the on the retina. Last on the ISTI. when you anticipate potentially filing an IND and starting the Phase two three again, presuming the Phase one wraps up as you hope.
And then a follow-up to the Phase IIb Virage, I know the enrollment completed in first half 2024, which we just data final data top line data for us.

So Manoj, why don't you take the first one and then Vince, you could take the second guess our four original restaurant model right now we are just following the a lot of patients.
Okay. As you know, our clinical trial to treat the patient that part, it's already finished that we need to follow the patients for six months after the last dosing. So we are expecting to finish our database with data of the trial in mid-2024, probably. And after that, we need to rise to interest a rep or and start that discussion with FDA. The final design of the and the so I don't expect to go for an IND until 2024, 2025, sorry by shore because obviously we need to finish some activities. We got before submitting any AMD for RightNow customer base. We want to give some color on the pancreatic program.

Thanks, Menno. So I'll as Steve indicated, a Virage study is enrolling and we anticipate completing enrollment in the first half of this year with the with the patients only. And then we will be very closely monitoring the emergence of our data. And this there's two key outcomes one. Obviously, our primary endpoint of survival will be following those patients and the longer the better from our perspective because we want to have a good effect.
So the primary endpoint data. The survival won't read out until next year as I kind of peak when we would like to say that to be potentially in the second half, the longer as long as these patients keep going. We want patients on our drug to do well.
In the interim, we will be looking at our data to see if there's something that we can around which we can build a formal interim analysis to review with the FDA and discuss next steps how we can potentially advance our program quickly into a pivotal trial. And as we know, as you know, we've got the orphan drug designation, so we'll leverage that strategy. But again, we can't necessarily predict the timing other than later in the year for a potential interim analysis if we choose to do one. But the overall endpoints for the primary endpoint of survival will be next year

Thank you very much. And maybe a follow-up questions from me on Cyno forking giving you actually sort of the endgame for CNO for where a so what though which we anticipate sort of coming to a conclusion on that on the data, what sort of timing on that? And then can you talk a little bit about how the partnership and characterize the partnership environment currently for Finjan licensing OTOR. for linear compounds?

Steven A. Shallcross

Sure. So on, as you know, the same for trials, a single single-site study at Wash U and those partners have been outstanding on fortunately or unfortunately, on the time it takes to enroll a trial like that is subject to the number of patients that actually meet the screening criteria so they can be brought into the trial and we're going to complete that trial, as I stated earlier on in relative short order.
And then we'll obviously have some disclosure around that. This cohort is pretty important because a sin for does degrade the combination of pepper so on and take it back to him. And obviously, we're monitoring the data in this trial and we want to make sure that the antibiotic is not and interfered with in this more fragile patient population.
So once we have that data in hand, we'll make a decision about whether or not we advance into the third cohort or whether or not we have enough and to answer our questions that were brought to us by the FDA on then this asset ties in more broadly to the initiatives we have underway to identify potential partners across our entire pipeline. So we've hired some outside advisors on one group specific to help us finding a home for person for and we've had engagement.
And again, a lot of this has to do about when we have the data on. We've also have a group we're working with and outreach to potential partners for the VCNO. one platform. And once again, we've had multiple engagements and interest and we will continue those discussions. And once again, data is key and we've also have some folks that we're working with trying to find a home for the Cine 20 program.
And again, those discussions are ongoing. I think the environment has gotten a bit better recently on, I think over the last couple of years, given sort of the bear market that biotech and small and micro-cap biotech has been and has hindered a lot of discussion on, but I am more and more optimistic things seem to be picking up a little bit. And in keeping with our strategy and how we view these ongoing discussions we're not going to talk about specific interactions and when we have something, obviously we'll make a disclosure about that, of course.

Thank you very much taking questions.

Operator

Thank you. And as a reminder, if you'd like to ask a question, please press star one on your telephone keypad or pause a moment to allow for any other question. Mr. Shah cross.
I'm not showing any other questions at this time. I'll turn the floor back to you for any final.

Steven A. Shallcross

Okay. Thanks, Melissa. Well, thanks again, everyone, for taking the time to join us today. I hope you sense that we're incredibly focused on driving all of our programs forward.
We're doing this in a way which I think we're very good stewards with our cash and making and stretching that dollar the best we can to get as much data and as many clinical outcomes and results as possible.
Will continue is we just ended with Jim here to evaluate our strategic options, and we'll continue to look for ways to drive additional value for our shareholders and for the long-term success of what we're trying to do namely delivering, you know, promising treatments for very, very difficult-to-treat cancers.
Thanks again for joining us, and we look forward to keeping you updated on our progress.

Operator

Thank you. This concludes today's conference. You may disconnect your lines at this. Thank you for your participation.