Full Year 2023 Theriva Biologics Inc Earnings Call
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Operator: Greetings and welcome to the Theriva Biologics full year 2023 investor conference call. At this time, all participants are in a listen-only mode.
Speaker Change: Greetings and welcome to this the Riva Biologics full year 2023 Investor Conference call. At this time, all participants are in a listen only mode.
Operator: As a reminder, this conference is being recorded. I would now like to turn the call over to your host, Chris Calabrese, with Lifesai Advisors. Thank you. You may begin.
Chris Calabrese: Reminder, this conference is being recorded I would now like to turn the call over to your host Chris calibration with life Science advisors. Thank you you may begin.
Chris Calabrese: Thank you, operator, and good morning, everyone. Welcome to Theriva Biologics' full year 2023 investor conference call. Leading the call today will be Stephen Chalcross, Chief Executive and Chief Financial Officer of Theriva Biologics. Dr. Manel Kiskayo, General Director of Theriva Biologics, Europe's subsidiary, and Dr. Vince Waitcher, head of corporate and product development of Sariva Biologics, are also on the call and will be available to answer questions during the Q&A session. Theriba Biologics issued a press release this morning that provided operational highlights and included the financial results for the full year ending December 31st, 2020. The press release can be found in the investor section of the company website at www. TherivaBio.com, together with the annual report on Form 10K for the full year ended December 31st, 2020, 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, www.
Chris Calabrese: Thank you operator, and good morning, everyone. Welcome to you to read the biologics full year of 2023 Investor Conference call, leading the call today will be Steven Shallcross, Chief Executive and Chief Financial Officer to read the biologics Dr. Mendell kits Guy out General director of series of Biologics.
Speaker Change: European subsidiary and Dr. Vince wait your head of corporate and product development series of Biologics are also on the call and will be available to answer questions. During the Q&A session to.
Speaker Change: The repo biologics issued a press release this morning, which provided operational highlights and included the financial results for the full year ending December 31 2023.
Speaker Change: Press release can be found in the investors section of the company website at Www Dot so read the bio dotcom together with the annual report on Form 10-K.
Speaker Change: For full year ended December 31st 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 www dot to Riva bio dotcom for 90 days.
Chris Calabrese: Theriva bio.com for 90 days. During this call, certain forward-looking statements regarding Cerevo Biologics and DCN Biosciences, current expectations and projections about future events will be made. Generally, forward-looking statements can be identified by terminology such as may, should, expects, anticipates, intends, plans, believes, estimates, and similar expressions.
Speaker Change: During this call certain forward looking statements regarding theories of biologics in D C and biosciences.
Speaker Change: <unk> expectations and projections about future events will be made.
Speaker Change: Generally the forward looking statements can be identified by terminology 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.
Chris Calabrese: 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 Theriva 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 those expressed. The information on this call is provided only as of the date of this call, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this conference call on account of new information, future events, or otherwise, except as required by law. Steve?
Speaker Change: Set forth in theory with biologics filings with the S E T.
Speaker Change: 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 read the biologics undertakes no obligation to update any forward looking statements contained on this conference call on account of new one.
Speaker Change: Information future events or otherwise, except as required by law with that I'd like to turn the call over to Steve 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 focus portfolio designed to address unmet needs for difficult-to-treat cancer. Our primary efforts and resources are focused on pursuing multiple therapeutic opportunities for our lead clinical candidate, BCN01. As a reminder, BCN01 is a systemically administered ocolytic adenovirus designed to selectively replicate within the tumor, degrade the tumor matrix, and increase tumor immunogenicity. We believe these multiple modes of action position BC-1 for optimized tumor killing in combination with chemotherapy and immunoncology products in otherwise refractory solid tumors.
Steve: Thank you, Chris and good morning, I appreciate everyone for taking the time to join us today.
Steve: In 2023 we continued to make steady progress to drive forward, our oncology for our focused portfolio designed to address unmet needs for difficult to treat cancers.
Steve: Our primary efforts and resources are focused on pursuing multiple therapeutic opportunities for our lead clinical candidate P. C N O one.
Steve: As a reminder, B C. N O. One is a systemically administered athletic adenovirus designed to selectively replicate within the tumor the grade the tumor matrix and increase tumor immunogenicity.
Steve: We believe these multiple nodes of action position V. C O one for optimized tumor, killing in combination with chemotherapy and immuno oncology products and otherwise refractory solid tumors.
Steven A. Shallcross: We have shown that repeated systemic dosing of VCN-O-1 is feasible from a safety perspective, and we can now focus on whether the repeated dose VCN-O-1 regimen may lead to improved clinical outcomes for patients. Beyond BCN01, we are pursuing new oncoletic virus candidates to leverage our novel Human Shield technology, which is designed to protect systemically administered oncoletic viruses from the host immune system and may facilitate more frequent repeated administration of oncoletic virus therapy. This 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 portfolio, we continue to screen and enroll patients in a second cohort of the Phase 1B2A clinical trial of CIN4, designed to prevent potentially fatal adverse outcomes in patients who undergo algenaea comitopoeotic cell transplantation (HCT) to treat hematologic cancer. 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 achieve multiple value-enhancing milestones.
Steve: We have shown that repeated systemic dosing of V. C. N O. One is feasible from a safety perspective, and we can now focus on whether the repeated dose V. C. N. Reg D. C. N O one regimen may lead to improve clinical outcomes for patients.
Steve: Beyond V C N O. One we're pursuing new athletic virus candidates to leverage our novel of Human Shield technology, which is designed to protect systemically administered athletic viruses from the host immune system and may facilitate more frequently repeated administration of alkylating virus therapies. This may.
Steve: They enable our pipeline of products to be used in standardized treatment cycles that are well established and cancer chemotherapy and immunotherapy.
Steve: Additionally, and as part of our oncology focused portfolio, we continue to screen and enroll patients in our second cohort of the phase one b to a clinical trial of syn for designed to prevent potentially fatal adverse outcomes in patients who undergo allogeneic hematopoietic cell transplant, a C T to treat hematologic cancers.
Steve: Yeah.
Steve: 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.
Steven A. Shallcross: With this brief introduction, I'd like to expand on key pipeline updates, starting with our lead program, VCN01. VcN01 has been administered to more than 100 patients across diverse indications, which speaks to its broad therapeutic potential, including pancreatic ductyl adenocarsinoma, or PEDAX, retinoblastoma, head-endax squim cell carcinoma, colorectal cancer, BCN01 has been granted orphaned drug designation in the U.S. and Europe for the treatment of pancreatic cancer and in the U.S. for retinoblasoma, providing additional opportunities for regulatory engagement and, if approved, market exclusivity. Our most advanced program for VCN01 is in PDAC, for which incidents continue to rise in an indication that has one of the lowest survival rates among all cancers. It is well established that the PEDAC tumor matrix is one of the key reasons for the overall poor therapeutic outcomes for these patients.
Steve: With this brief introduction I'd like to expand on key pipeline updates starting with our lead program P. C N O one.
Steve: B C. N O. One has been administered to more than 100 patients across diverse indications, which speaks to the broad therapeutic potential, including pancreatic ductal adenocarcinoma or Pete Act.
Steve: Retinoblastoma head and neck squamous cell carcinoma, colorectal cancer and ovarian cancer.
Steve: V C. N O. One has been granted orphan drug designation in the U S and Europe for the treatment of pancreatic cancer.
Steve: And in the U S for retinoblastoma, providing additional opportunities for regulatory engagement and if approved market exclusivity.
Steve: Our most advanced program for V. C. N O. One is in <unk> for which incidents continues to rise and an indication that has one of the lowest survival rates among all cancers.
Steve: It is well established that the pdx tumor matrix is one of the key reasons for the overall poor therapeutic outcomes for these patients.
Steven A. Shallcross: We believe UCN01's differentiated mechanism of action has the potential to address the urgent need for new treatment options for patients with PEDAC by degrading the Tupper matrix and increasing tumor access by co-administering cancer therapy. We are pleased to report that dosing is well underway for Verage, our phase 2B trial of BCN01, in combination with standard of care chemotherapy, gencytibine, and napaxytaxyl, which is being evaluated as a first-line therapy for patients with PEDAC. With six sites open in the U.S. and nine sites open in Spain, Farage remains on track to complete enrollment in the first half of 2024.
Steve: We believe you see no one's differentiated mechanism of action has the potential to address the urgent need for new treatment options for patients with Pete Act by degrading the two per matrix and increasing tumor access by co administering cancer therapies.
Steve: We are pleased to report that dosing is well underway for <unk>, our phase two b trial of V. C. N O. One in combination with standard of care chemotherapy Gen side of being in Nab, Paclitaxel, which is being evaluated as a first line therapy for patients with <unk>.
Steve: With six sites open in the U S and nine sites open in Spain for Raj remains on track to complete enrollment in the first half of 2024.
Steven A. Shallcross: 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, Varage will continue to enroll patients without any changes to the protocol. Notably, intravenous BCN01 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 BCN01, providing the first clinical evidence of the feasibility of repeated systemic doses. As a reminder, primary endpoints for the Varage trial include overall survival and BCN01 safety and tolerability. Additional endpoints include progression-free survival, objective response rate, measures of BCN01 by distribution, replication, immune response, and measures of the quality of life of patients treated. Since this is an open-label trial, progress will be monitored very closely, steps to accelerate the clinical program may be implemented if supported by emerging data.
Steve: In the first quarter of 2024, we completed the first safety review with the independent data monitoring committee or I D. M C.
Steve: With a positive recommendation from the I D M C.
Steve: Raj will continue to enroll patients without any changes to the protocol.
Steve: Notably intravenous. These you know one has been well tolerated and demonstrated a safety profile consistent with prior clinical trials.
Steve: Importantly, no additional toxicities were observed in patients receiving a second dose of V. C. N O one providing the first clinical evidence of the feasibility of repeated systemic dosing.
Steve: As a reminder, primary endpoints for the barrage trial include overall survival in B C N O one safety and Tolerability.
Steve: Additional endpoints include progression free survival objective response rate measures of D. C. N O one by a distribution replication immune response and measures of the quality of life of treated patients.
Steve: Since this is an open label trial progress will be monitored very closely and steps to accelerate the clinical program may be implemented if supported by emerging data.
Steven A. Shallcross: More broadly, the barrage trial will enable us to determine the feasibility of repeated dosing of VCN-O-1. This could shift the approach to standardized treatment cycles that are well established in cancer chemotherapy and immunotherapy and may lead to improved clinical outcomes for patients with PEDAC and other difficult-to-treat solid tumors. In addition to advancing the Verage PEDEC trial, we continue to work closely with key opinion leaders in the U.S., Europe, and Central and South America, as well as with regulatory agencies to refine our clinical strategy for retinoblastoma. We believe intravitreal Vecino-1 has the potential to treat vitrease seeds in children with retinoblastoma.
Steve: More broadly the barrage trial will enable us to determine the feasibility of repeated dosing of V. C. N O one.
Steve: This could shift the approach the standardized treatment cycles that are well established in cancer chemotherapy and immunotherapy and may lead to improved clinical outcomes for patients with <unk> and other difficult to treat solid tumors.
Steve: In addition to advancing the variety of pediatric trial, we continue to work closely with key opinion leaders in the U S Europe, and central and South America, as well as with regulatory agencies to refine our clinical strategy and retinoblastoma.
Steve: We believe intervene trio do you see no one has the potential to treat vitreous seeds in children with retinoblastoma.
Steven A. Shallcross: Since current clinical practice varies and there is no regulatory guidance specific to retinoblastoma drug development, we held a pre-I&D meeting with the FDA in the fourth quarter of 2023 to discuss the development pathway for BCN01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma. During our meeting with the FDA, we were provided some guidance on the potential endpoints and patient population for an advanced clinical trial and encouraged to submit a formal protocol under U.S. I&D in order to provide 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 VCNO1 is being explored in a number of investigator-sponsored studies that are underway at leading oncology research institutions around the world.
Steve: Since current clinical practice varies and there is no regulatory guidance specific to retinoblastoma drug development, we held a pre IND meeting with the FDA in the fourth quarter of 2023 to discuss the development pathway for D. C. N. Oman is an adjunct to chemotherapy in pediatric patients with advanced <unk>.
Steve: Retinoblastoma.
Steve: During our meeting with the FDA, we were provided some guidance on the potential endpoints in patient population for an advanced clinical trial and encourage you submit a formal protocol under a U S. I N D. In order to provide a more detailed commentary for this program.
Steve: We are encouraged by interactions with the FDA and look forward to driving this program forward.
Steve: In parallel with company sponsored studies the potential utility of V. C. N O. One is being explored in a number of investigator sponsored studies that are underway at leading oncology research institutions around the world.
Steven A. Shallcross: Notably, collaborators at San Juan de Deo have completed patient treatments in the Phase 1 Investigator-sponsored trial evaluating the safety and activity of intravitrile VCN-O-1 in pediatric patients with refractory retinoblastoma. The trial evaluated escalating doses of E.C.N.01 administered by two intravenous 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 two trial and its design and the protocol. As a reminder, preclinical data have shown that Topatikin treatment enhanced BCN01 oncolytic activity against retinoblastoma and, more broadly, reinforced BCN01's possibility as an adjunct to intravenous chemotherapy in patients who fail currently available treatment.
Notably collaborators as Sandro under Dale have completed patient treatment in the phase one investigator sponsored trial evaluating the safety and activity of <unk> in pediatric patients with refractory retinoblastoma.
Steve: The trial evaluated escalating doses of V. C. N O one administered by two intravenous injections separated by 14 days and remains on track to complete patient follow up in the first half of 'twenty 'twenty, four which will help to inform the planned phase II trial in <unk>.
Steve: Design and the protocol.
Steve: As a reminder, preclinical data have shown that took the T can treatment enhanced P. C. N O one uncle lytic activity against Retinoblastoma and more broadly reinforced V. C N O ones possibility as an adjunct to intravenous you all chemotherapy in patients who fail currently available treatments.
Steven A. Shallcross: 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 1 investigator-sponsored trial administering VCN01 with U-R-T measles cells to patients with ovarian or pancreatic cancer. BCN01 is designed to increase tumor immunogenicity and improve access by additional therapies such as Hewkar-Miesel cells. Although cell-based immunotherapies have had limited efficacy against cellar tumors to date, we are encouraged by initial results highlighting the feasibility of administering BCN01 with this type of CART therapy.
Steve: We remain encouraged by the potential of this novel combination approach to provide superior clinical benefits for children with this devastating cancer.
Steve: Additionally.
Steve: Sully the University of Pennsylvania continues to enroll and treat patients in their phase one investigator sponsored trial administering V. C. N O one with your car T missile cells to patients with ovarian or pancreatic cancers.
Steve: D C. N O. One is designed to increase tumor immunogenicity and improve access by additional therapies, such as Hugh card muscle cells.
Steve: Why cell based Immunotherapies have had limited efficacy against solid tumors to date. We are encouraged by initial results highlighting the feasibility of administering V. C. N O. One with this type of car T therapy.
Steven A. Shallcross: These preliminary results were presented last year at the Society for Immunotherapy of Cancer Annual Meeting, or SITS. The UPenn investigators are continuing to explore the optimal dosing regimen for BC-01, co-administered with UCARP mesal cells, and we look forward to further data from the study in 2024. Turning to our ongoing phase 1B2A clinical trial at Washington University evaluating CIN4 or Ryback, the trial is designed to evaluate the therapeutic potential of Sin 4 to reduce fatal adverse events related to IV beta lactam antibiotic use and alginic HETE recipients, including acute graft versus host disease or AGVHD, and overgrowth and infection by pathological organisms such as C. difficil and vancomyocin resistant and The Pays 1B2A study is designed to assess the feasibility of using Sin 4 and consists of three sequential cohorts comparing different IV beta lactam antibiotics following conditioning. In each cohort, eight patients will receive CIN4, and four will receive placebo. While the data remained blinded, interim analysis suggests that CIN4 is well tolerated and was not observed in the blood samples of a majority of the available patients.
Steve: These preliminary results were presented last year at the society for immunotherapy of cancer annual meeting or city.
Steve: The U Penn investigators are continuing to explore the optimal dosing regimen for VC no one co administered with your card muscle cells and we look forward to further data from the study in 2024.
Steve: Turning to our ongoing phase one b to a clinical trial of Washington University evaluating seeing four or write backs.
Steve: The trial is designed to evaluate the therapeutic potential of sin for.
Steve: Two fatal to reduce fatal adverse events related to IV beta lactam antibiotic use in allogeneic, <unk> recipients, including acute graft versus host disease, or a gvhd and overgrowth in infection by pathological organisms, such as C difficile and vancomycin resistant <unk>.
Steve: The phase one B two way study is designed to assess the feasibility of using <unk> for and consist of three sequential cohorts comparing different I IV beta lactam antibiotics falling conditioning therapy.
Steve: And each cohort eight patients will receive <unk> four and four will receive placebo.
Steve: While the data remain blinded interim analysis suggests that's in four is well tolerated and was not observed in the blood samples of a majority of the available patients.
Steven A. Shallcross: Our second cohort is underway and is designed to evaluate Sin 4 in combination with pepper cilin and taseabactan. The trial is on track, and complete enrollment in the second cohort will occur in the second quarter of 2024. This cohort will provide important additional safety information, in particular whether oral CIN4 has the potential to alter IBE and about uptake levels in this patient population.
Steve: Our second cohort is underway and is designed to evaluate sinful or in combination with pepper ceiling and tastes it back to him.
Steve: The trial is on track to complete enrollment in the second cohort in the second quarter of 2024.
Steve: This cohort will provide important additional safety information in particular, whether oral syn <unk> has the potential to alter IV antibiotics levels in this patient population.
Steven A. Shallcross: We look forward to sharing this data in the second half of 2024. Overall, we are encouraged by the progress across our pipeline and the growing clinical data that underscore the promise of our systemically administered oncolytic adenovirus in key indications and combinations. We remain focused on driving our clinical programs forward and exploring opportunities to leverage our novel Abume and Shield technology and exciting additional technologies from our OV Discovery. I'm confident that the company's upcoming catalyst will provide a solid foundation for execution and value creation. Specifically, we remain on track to complete enrollment for the Verage study in the first half of 2024. Complete follow-up in the Phase 1 Investigator-Sponsor trial evaluating the safety and activity of intravitrile vcina 1 in pediatric patients with refractory retinablastoma in the first half of 2024.
Steve: We look forward to sharing this data in the second half of 'twenty 'twenty four.
Steve: Overall, we are encouraged by the progress across our pipeline and the growing clinical data that underscore the promise of our systemically administered athletic adenovirus in key indications and combinations.
Steve: We remain focused on driving our clinical programs forward and exploring opportunities to leverage our novel abuse, <unk> Shield technology and exciting additional technologies from her Ob discovery platform.
Steve: I'm confident that the company's upcoming catalysts will provide a solid foundation for execution and value creation.
Steve: Specifically, we remain on track to complete enrollment for the Verizon <unk> study in the first half of 2024.
Steve: Complete follow up in the phase one investigator sponsored trial evaluating the safety and activity of intravenous <unk> in pediatric patients with refractory retinoblastoma and the first half of 2024.
Steven A. Shallcross: In complete enrollment in the second cohort of our Phase 1B2A clinical study of Sin 4 for the prevention of AGVHD and bone marrow transplant patients in the second quarter of 2025. Now, I briefly turn to our financial results for the first full year ended December 31, 2023. General Administrative expenses decreased to $7.1 million for the year ending December 31, 2023 from $9.9 million for the year ending December 31, 2022. This decrease of 28% is primarily comprised of a 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 VAC acquisition, and director and officer insurance, offset by higher audit fees and other consulting.
Steve: And complete enrollment in the second cohort of our phase one b to a clinical study of Syn <unk> for the prevention of a gvhd in bone marrow transplant patients in the second quarter of 2024.
Steve: Now I'll briefly turn to our financial results for the first full year ended December 31 2023.
Steve: 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.
Steve: This decrease of 28% is primarily comprised of the decrease in the fair value of contingent consideration of $2 $8 million, along with lower salary investor relations legal costs consulting fees related to the V C N acquisition and director and Officer insurance.
Steve: Offset by higher audit fees and other consulting fees.
Steven A. Shallcross: The charge related to stock-based compensation expense was $.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 Varage, Phase 2 clinical trial of VCN01 and PEDAC, offset by lower expenses related to our Phase 1B2A clinical trial of CIN4 and ALJNAICHCT recipients, the completed phase 1A clinical trial of CIN 20, decreased manufacturing expenses related to our phase 1A clinical trial of CIN 20, and lower other indirect costs. We anticipate research and development expenses to increase as Research and Development Expense also includes a charge related to nine cash stock bases and compensation expense of $165,000 for the year ended December 31, 2020, compared to $112,000 for the year ended December 31, 2022. Other income was $442,000 for the year ended December 31, 2021, and 2023, compared to other income of $471,000 for the year ended December 31, 2020.
Steve: The charge related to stock based compensation expense was point $4 million for the year ended December 31, 2023 compared to $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.
Steve: This increase of 22% primarily as a result of higher clinical trial expenses related to wherever Raj phase II clinical trial of <unk> and <unk>.
Steve: Offset by lower expenses related to our phase <unk> clinical trial of Syn <unk> in allogeneic H C T recipients.
Steve: The completed phase one clinical trial of Syn <unk> 'twenty decreased manufacturing expenses related to our phase one clinical trials in 'twenty and lower other indirect costs.
Steve: We anticipate research and development expense to increase as we continue enrollment in our garage phase two clinical trial of B C N O and P deck in our ongoing phase one clinical trial in retinoblastoma expand GMP manufacturing activities for <unk> and continue supporting RBC in 11.
Steve: Other preclinical and discovery initiatives.
Steve: Research and development expense also includes a charge related to noncash stock based compensation expense of $165000 for the year ended December 31, 2023 compared to $112000 for the year ended December 31 2022.
Steve: Other income was $1.442 million for the year ended December 31, 2023 compared to other income of $471000 for the year ended December 31 2022.
Steven A. Shallcross: Other income for the year ended December 31, 2020, is 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.
Steve: Other income for the year ended December 31, 2020 years, primarily comprised of interest income of $1.439 million and an exchange gain of $3000.
Steve: Other income for the year ended December 31, 2022 is primarily comprised of interest income of $512000 offset by an exchange loss of $41000.
Steve: Cash and cash equivalents totaled $23 $2 million as of December 31, 2023, compared to $41 8 million as of December 31, 2022.
Steven A. Shallcross: 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 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. Thank you. At this time, we'll be conducting a question and answer session. If you'd like to ask a question, please press Star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue.
Steve: 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.
Speaker Change: I'd like to thank the entire three the 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.
Speaker Change: Thank you at this time well be conducting a question and answer session. If you'd like to ask a question. Please press star one on your telephone keypad, a confirmation tone will indicate your line is in the question queue you.
Operator: You may press star 2 if you'd like to remove your question from the list. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the start button. One moment, please. We'll we pull for questions. Thank you. Our first question comes from the line of James Malloy with Alliance Global Partners. Please proceed with your questions. Hey guys, good morning. Thank you very much for taking my question. Hello, I had a question. Hello Stephen,
Speaker Change: You May press star two if you'd like to remove your question from the queue.
Speaker Change: For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys one.
One moment, please while we poll for questions.
Speaker Change: Thank you. Our first question comes from the line of James Molloy with Alliance Global Partners. Please proceed with your questions.
James Francis Molloy: Hey, guys. Good morning, Thank you very much for taking my questions.
James Francis Molloy: Hello, I had a question looking.
James Francis Molloy: Let's ask you, Stephen, or Manel, looking across the multiple ISTs, which are a very cost-efficient way of getting trials done, an excellent use of capital, which do you guys see is sort of the most promising? You love all your children, of course, but which seems like it might be growing tallest when you're looking across the plethora of ISTs you guys have got underway? Thank you. So, let me just highlight what we've got going on once again, and then Manel, you could talk specifically about some of the exciting findings that we've disclosed at various scientific conferences. So, as you're aware, we put out some incredible head and neck cancer data at Esmil last October. This was using BCN-O-1 in combination with checkpoint inhibitors and patients that were refractory to previous rounds of checkpoint inhibitor therapy. So Manel could talk a little bit about that.
James Francis Molloy: Steve a question.
Speaker Change: Of course, we just need a viewer menno.
Speaker Change: Looking across the.
Speaker Change: Multiple I S teas, which are very cost efficient way of getting getting trials done excellent excellent use of capital.
Speaker Change:
Steve: Which one do you guys see as sort of the most promising Oh My God you Love all your children of course, but which seems like it might be might be growing the task. When you look at across the trust of the plethora of I S. T. As you guys have got underway.
Speaker Change: So let me let me just highlight.
Speaker Change: What we've got going on and once again and then now you could talk specifically about some of the exciting findings that we've.
Speaker Change: Of had disclosed at various scientific conferences, so as Youre aware, we put out some incredible head and neck cancer data at ESMO last October this is using <unk> hundred one in combination with checkpoint inhibitors in patients that were refractory to previous rounds.
Speaker Change: A checkpoint inhibitor therapy.
Speaker Change: Someone else could talk little bit about that.
Steven A. Shallcross: We've got the retinalblastoma program, which we've just concluded, and now we're in the middle of follow-up. And then we've got the ongoing. U-PEN study, which is down to their organization trying to isolate the right dose to take forward, so we would expect to see some more data sometime this year related to the work they're doing there. Now, maybe you could just highlight the important findings in those three studies that are ongoing or have recently completed. Yes, sure, sure. So obviously, the most complete data, let's say it's from the trial in Hettenneck that we presented as amo As you know, this was a trial combining DC&1 with the development of and in patients refractory to the action of immunogenic inhibitors in Hetten Neck Arcinoma.
Speaker Change: We've got the Retinoblastoma program.
Speaker Change: Which we've just concluded and now we're in the middle of follow up and then we've got the ongoing U Penn study, which.
Speaker Change: Which is down two of their organization trying to isolate the right dose.
Speaker Change: To take forward. So we would expect to see some more data sometime this year related to the work Theyre doing there. So maybe you could just highlight the important findings in those three <unk>.
Speaker Change: Studies that are that are ongoing or recently completed.
Speaker Change: Yes sure sure.
Speaker Change: So obviously that the muscle.
Speaker Change: Complete data, let's say needs from the filing cabinets represent small in October as you don't get swaps a trial combining these kind of one way and in patients refractory to the action of being more of a checkpoint inhibitor.
Speaker Change: Actually no.
Manel Cascall: Okay, and we have previously presented safety data that demonstrated that DCNO1 has an acceptable safety profile when administered prior to Durbalamap. In this new presentation that we conducted in October, we presented data on efficacy, and we saw that patients treated with DCN showed an increased response to a subset of chemotherapy treatments after progression to this trial. But specifically, we have also seen that the survival of patients has been extended in a number of patients, and, in fact, there are some patients still alive more than four years after participating in the trial. In my view, it's quite remarkable because those patients, when entering the trial, had an expectancy of life between three or four months.
Speaker Change: Okay, and we have previously presented data that demonstrated the DCNR one has an acceptable safety profile one of Mr. <unk>.
Speaker Change: You do have a bottleneck.
Speaker Change: Indeed.
Speaker Change: No presentation that we conducted in October we presented data on that.
Speaker Change: And we have seen that the patient is treated would be can show up and we should fresh sponge to a successful chemotherapy.
Speaker Change: Once I start progressions in style, but specifically we have also seen that the Bible of patients has been a large for a number of patients and in fact, there are some patients are still alive more going forward.
Speaker Change: She didn't participate in the trial, which in my view each quite a remarkable because those patients entering the trial has some effect on shelf life could be three or four months and what we are seeing obviously its' really pricing and more work. It's not just a question of if I think I see that that's the back of good correlation between these restaurants.
Manel Cascall: And what we are seeing, obviously, is really interesting. And moreover, it's not just a question of efficacy, but there's a very good correlation between response and the biological data that we are getting from the tumor biopsies of those patients. And in fact, we have seen downregulation of tumor-matic genes. And we have been observing increasing levels of immune markers in the tumor biopsies.
Speaker Change: And biological data.
Speaker Change: Do more biopsies distributions and in fact, we have seen a downregulation of magic genes. We haven't been a sharp increase of level something you know mark Hershey into tumor biopsies and interestingly there was a correlation between the survival and with the Cps score a day after a b C and the one thing.
Manel Cascall: And interestingly, there was a correlation between survival and with the CPS score, a kind of marker about the immunological status, a day after BCNO1 treatment. And what looks like in our biological data is that BCNO1 treatment is changing this environment, and there's a correlation between the magnitude of the change that we need to use and the survival of patients, which is, I think, exciting. Obviously, it's a reduced population, but it's exciting. In retinoblastoma, we have been treating more patients. We have seen some patients with reduction in the B2C, but specifically, we have been also collecting data in combination with chemotherapy. The chemotherapy that is used in retinoblastoma is called Caputican.
Speaker Change: It's going to it's a kind of marker about immunological the steepness and what looks in our biological deal that you said, we shouldn't one friedman changing environment and desk a correlation between the magnitude of change that we can use industrial bipolar patient, which is I think exciting obviously, you've talked with you said population, but it's exciting.
Speaker Change: In retinoblastoma, we'd have been taking more patients we have seen some patients with a reduction in your seats, but specifically we have been also but I can be that of combination with.
Speaker Change: Well therapy.
Speaker Change: Chemotherapy that you've seen racking up last time I spoke with you can and in this trial, we have seen none.
Manel Cascall: And in this trial, we have seen not directly in the clinical patients, but in preclinical work that we have done with biopsies and from human cells, we have been observing that there's a synergy between the action of Cineau and Topotican, which is very encouraging and opens the door for a combination approach in our face-to-prolund approach, that is basically what we discussed with FDA in our meeting. So basically, the data we are collecting is 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 the head and neck, in the retinaldustoma, which is also the same thing that we have observed in our face one problem in pancreatic cancer.
Speaker Change: Not getting the clinical patients that in preclinical work that we have done with biopsies from human cells, and we have been observing that the synergy between the action of instead of one.
Speaker Change: A couple of Beacon, which is very encouraging and opens the door for a combination approach in our phase two program that is absolutely what we discuss it with FDA.
Speaker Change: Okay. So basically the data we are collecting it it's very exciting and in my view is it's very exciting to see that in different indications. We can confirm the mechanism of action and we can see our initial evidence of activity in head and neck in writing the rest of them.
Speaker Change: Which is also the same thing that we have observed in our phase one program in pancreatic cancer.
Manel Cascall: So that the data is quite consistent in that way. Great, thank you very much for taking that. A quick follow-up on the retina-lastoma IST.
Speaker Change: Did he say you'd see it's quite consistent in that way.
Speaker Change: Okay.
Speaker Change: Great. Thank you very much for taking my.
Speaker Change: A quick follow up on the on the retina Blastoma I S. T. A when you anticipate to potentially filing an IND and starting the phase three.
James Francis Molloy: When you anticipate potentially filing an IND and starting phase 2-3, again, presuming the phase 1 wraps up, as you hope, and then a follow-up, too, in phase 2B, Varage, I know the enrollment is completing here, first half-24. When should we anticipate final top-line data for Varage? So, Menel, why don't you take the first one, and then, Vince, you could take the second.
Speaker Change: Again presuming the phase one wraps up as you hope and then I'll have a follow.
Speaker Change: A follow up too on the face to be <unk>.
I know the enrollments completed your first half 'twenty four when should we anticipate a final top line data for rush.
Speaker Change: So why don't you take the first one and then Vince you could take the second.
Manel Cascall: Yes, for retina resoma right now, we are just following the follow-up of patients, okay? As you know, in a clinical trial, you treat the patient. That part is already finished, but we need to follow the patients for six months after the last dosing. So we are expecting to finish our database with the date of the trial in mid-2020, probably. And after that, we need to write the clinical study report and start discussing the final design of the INB with the FDA. So I don't expect to go.
Vince: Yes for the rest of the restaurant, where right now we are just following the follow up of patients. Okay. As you know.
Speaker Change: The patients that clock is already finished but we need to follow the patients for six months after the loss of Oc.
Vince: We are expecting to finish our database, we beat up the trial meet 40, 24, probably and after that we need to write the clinical study.
Vince: Right before I start discussing with FDA the final design of that and be so.
Vince: Back to to go for an AMB.
Manel Cascall: go for an IND until 2025, sorry, because obviously we need to finish some activities before submitting any I&D for writing a BLASCOM. Vince, do you want to give some color on the pancreatic forum? Yeah, thanks, Minot.
Vince: Until 'twenty 'twenty four 'twenty five sorry by short because somebody said, we need to finish them excuse me.
Vince: Before the meeting I am before wrapping up my stomach.
Vince: Vince do you want to give some color on the pancreatic program.
Vince: Yes. Thanks.
Vince Wacher: So, as Steve indicated, our Varage study is enrolling, and we anticipate completing our enrollment in the first half of this year. With the patients all in, then we will be very closely monitoring the emergence of our data, and there are two key outcomes. One, obviously, our primary endpoint is survival.
Vince: So.
Vince: As Steve indicated our garage studies enrolling and we anticipate completing enrollment in the first half of this year with our with the patients. All in then we will be very closely monitoring the emergence of our data and there's there's two key outcomes. One obviously our primary endpoint is survival will be following those patients in the long.
Vince Wacher: We'll 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 come out until next year. And I can't pick when we would like for that to be, you know, potentially in the second half of the year, as long as these patients keep going. We want patients on our drug to do well.
Vince: With a better from our perspective, because we wanted to have a good effect.
Vince: The primary endpoint at the survival, one read out until next year.
Vince: Todd pick Wham, we would like to say that to be a potentially in the second half of the of how long as long as these patients keep telling them, we want patients on our drug to do well.
Vince Wacher: In the interim, we'll be looking at our data to see if there's something that we can build a formal interim analysis around to discuss with the FDA and discuss the next steps, how we can potentially advance our program quickly into a pivotal trial. And as we know, as you know, we have the orphan drug designation. So we'll leverage that strategy.
Vince: In the interim we will be looking at I thought it 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 the next steps how we can potentially advance that program quickly into.
Vince: Pivotal trial and that's as we know as you know with the orphan drug designation. So we will leverage that strategy.
Vince Wacher: But again, we can't necessarily predict the timing other than later in the year for potential interim analyses if we choose to do one. But the overall endpoint for the primary endpoint for survival will be next year. Great, thank you very much.
Vince: But I can't we can't necessarily predict the timing of a later in the year for a potential interim analysis, if we choose to do one but the the overall endpoint. So the primary endpoint for survival will be next year.
Speaker Change: Great. Thank you very much.
James Francis Molloy: And maybe a final couple questions for me. On CIN-04, Stephen, can you walk through sort of the end game for Cyn O4? Where, so what the..., which we anticipate sort of coming to a conclusion on that, on the data, so the timing on that. And then can you talk a little bit about how the partnership and characterize the partnership environment currently for potentially out licensing, you know, O2O or really any of your compounds? Sure. So, as you know, the CIN4 trials, a single-site study at Washu, and those partners have been outstanding. You know, fortunately or unfortunately, 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 maybe a final couple of questions for me.
Speaker Change: On cinema Forking excuse me you walk through sort of the endgame for signal for where so what the.
Speaker Change: Which we anticipate sort of.
Speaker Change: Coming to a conclusion on that on the data. So the timing on that and then can you talk a little bit about how the partnership and characterize the partnership environment currently for potentially out licensing O to O or really any of your compounds.
Speaker Change: Sure.
Speaker Change: So.
Speaker Change: The <unk> trial is a single site study at wash U and those partners have been outstanding.
Speaker Change: Fortunately or unfortunately, the 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.
Steven A. Shallcross: We're going to complete that trial, as I stated earlier, in relatively short order, and then, you know, we'll obviously have some disclosure around that. This cohort is pretty important because Sin 4 does degrade the combination of peppercillin and Thasabactam. And obviously, we're monitoring the data in this trial. We want to make sure that the antibiotic is not interfered with in this more fragile patient population.
Speaker Change: We're going to complete that trial as I stated earlier in relative short order.
Speaker Change: And then we will obviously have some disclosure around that.
Speaker Change: This cohort is pretty important because soon for does degrade the combination of pepper, Ceylon and Taser backed him.
Speaker Change: Obviously, we're monitoring the data in this trial and we want to make sure that the antibiotic is not interfered with and and this more fragile patient population. So once we have that data in hand, we'll make a decision about whether or not we.
Steven A. Shallcross: So once we have that data in hand, we'll make a decision about whether or not to advance into the third cohort or whether or not we have enough to answer our questions that were brought to us by the FDA. And 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, one group specific to help us find a home for Sin 4. And, you know, we've had engagements. And again, you know, a lot of this has to do with when we have the data.
Speaker Change: Vance into the third cohort or whether or not we have enough.
Speaker Change: Two two.
Speaker Change: Answer our questions that.
Speaker Change: We are brought to us by the F D a.
Speaker Change: And then this asset highs in more broadly to the initiatives, we have underway to identify potential partners across our entire pipeline.
Speaker Change: So we've hired some outside advisors.
Speaker Change: One group specific to help is finding a home for <unk> four and <unk>.
Speaker Change: We've had engagement.
Speaker Change: And again a lot of this has to do about when we have the data.
Steven A. Shallcross: We also have a group we're working with, and we're doing outreach to potential partners for the VCNO1 platform. And once again, we've had multiple engagements and interests, and we'll continue those discussions. Once again, data is key.
Speaker Change: We've also have a group, we're working with and outreach to potential partners for the V. C N O one platform.
Speaker Change: And once again, we've had multiple engagements and interest.
Speaker Change: And we.
Speaker Change: We will continue those discussions and <unk>.
Speaker Change: Once again data is key.
Steven A. Shallcross: We've also got some folks that we're working with to try to find a home for the CIN 20 program. You know, again, those discussions are ongoing. I think the environment has gotten a bit better recently. I think over the last couple of years, given sort of the bare market that biotech and small and microcap biotech has been and has hindered a lot of discussion.
Speaker Change: We've also have some folks that were working with.
Speaker Change: To try to find a home for that.
Speaker Change: These in 'twenty program.
Speaker Change: And.
Speaker Change: Again, those discussions are ongoing I think the environment has gotten a bit better recently.
Speaker Change: I think over the last couple of years given sort of the.
Speaker Change: The bear market that biotech and small and micro cap biotech has been has has.
Speaker Change: Has hindered a lot of discussion.
Steven A. Shallcross: But I'm more optimistic. Things seem to be picking up a little bit. And in keeping, you know, with our strategy and how we view these ongoing discussions, we're not going to talk about specific interactions.
Speaker Change: But.
Speaker Change: I'm more optimistic things seem to be picking up a little bit and in keeping with.
Speaker Change: Our strategy and how we view these ongoing discussions we're not going to talk about specific.
Speaker Change: Interactions and when we have something obviously, we'll make a disclosure about that.
Steven A. Shallcross: And, you know, when we have something, obviously, we'll make a disclosure about that. Of course. Great. Thank you very much for taking the time to answer the question. Thank you. As a reminder, if you'd like to ask a question, please press Star 1 on your telephone keypad, where Paul will be for a moment to allow for any other questions. Mr. Shawcross, I'm not seeing any other questions at this time. I'll turn the floor back to you for any final questions.
Speaker Change: Of course, great. Thank you very much for taking the questions.
Speaker Change: Thank you as a reminder, if you'd like to ask a question. Please press star one on your telephone keypad will pause a moment to allow for any other questions.
Speaker Change: Mr. Schall Cross I'm lacking any other questions at this time I'll turn the floor back to you for any final comments.
Operator: Okay, thank you, 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. And we do this in a way that I think, you know, 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. We'll continue, as 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. Thank you. This concludes today's conference. You may disconnect your lines at this time. Thank you for your purchase.
Speaker Change: Thank you 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.
Speaker Change: We're very good stewards with our cash.
Melissa: And making and stretching that dollar the best we can to get as much data in as many clinical outcomes and results as possible will continue as we just ended with Jim here to evaluate our strategic options and we'll continue to look for ways to dry.
Melissa: Ive additional value for our shareholders and for the long term success of what we're trying to do namely delivering.
Melissa: 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.
Speaker Change: Thank you. This concludes today's conference you may disconnect. Your lines at this time. Thank you for your participation.