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spk03: Good day, ladies and gentlemen, and welcome to the TriCon Pharmaceuticals' fourth quarter and year-end 2023 earnings conference call. At this time, all callers are in a listen-only mode. After the speaker's prepared remarks, we will conduct a question and answer session, and instructions will be given at that time. During today's call, we will be making certain forward-looking statements, including statements regarding expected timing of clinical trials and results, regulatory activities, financing opportunities, our development plan and strategies, potential cost savings, and other benefits deliverable through our product development platform, or PDP. the ability to enter into additional license agreements, and expectations regarding in the FLOBNAB treatments continuing to generate a double-digit objective response rate. These statements are subject to risk and are described in our filings made with the Securities and Exchange Commission, including our annual report on Form 10-K for the year ended December 31, 2023, and subsequent reports on Forms 10-Q, you are cautioned not to place undue reliance on these forward-looking statements unless required by applicable law. We disclaim any obligation to update such statements. Now I would like to turn the call over to Dr. Charles Thor, President and CEO of Tracon Pharmaceuticals. Dr. Thor, please begin.
spk10: Good afternoon, and thank you for joining Tracon's fourth quarter and year-end 2023 Financial Results and Business Update call. I will begin with an update on our pipeline and then review our recent activities. Following that, Scott Brown, our Chief Financial Officer, will review the Financial results for the three and 12 months into December 31st, 2023. Finally, we will conclude by taking your questions. I'll begin with an update on our continued progress with the ongoing phase two and the SART pivotal trial. In December, we reported updated interim safety and efficacy data from 46 patients in cohort C of single agent and the treatment. The objective response rate in the initial 46 patients treated with single agent ENVA was 15% by investigator review and 8.7% by blinded independent central review, or four responses. ENVA monotherapy was generally well tolerated without a single drug-related serious adverse event. Importantly, median duration response by central review was greater than six months. We are on track to complete accrual of the ENVASARC pivotal trial later this quarter. and expect to report updated response data shortly thereafter and prior to the end of this quarter. As a reminder, in order to statistically exceed the 4% objective response rate of Votrien, the only FDA-approved treatment for patients with refractory UPS or MFS, the primary endpoint in NVSARC must show objective responses in nine out of 80 patients or an 11.25% objective response rate confirmed by central review. Median duration response of greater than six months is a key secondary endpoint. Our goal in ENVISARC is to demonstrate that ENVA has the potential to be both safer and more efficacious than Votrien, a drug with a black box warning for fatal liver toxicity. Based on data from trials of other checkpoint inhibitors in refractory UPS or MFS, we are targeting a 15% response rate for single agent ENVA. Furthermore, we plan to approach the FDA to discuss a BLA filing strategy as soon as we determine nine responses. As a reminder, we have received FAST-TRACK designation for ENVA in the sarcoma subtypes of UPS and NFS that have progressed on one or two prior lines of therapy and received orphan drug designation in soft tissue sarcoma based on activity observed in ENVA-SARC. These designations provide important advantages that might expedite regulatory review of ENVA. ENVASARC is designed to provide safety and efficacy data in the refractory sarcoma subtypes of UPS and MFS. We also have a strategy to pursue the approval of ENVA in frontline sarcoma. Doxorubicin is the most common approved therapy used for the treatment of newly diagnosed sarcoma patients. We therefore plan to initiate a trial of ENVA and doxorubicin in the frontline setting of the common sarcoma subtypes, including UPS and MFS, following the completion of enrollment in the pivotal ENVASARC trial, and prior to the expected BLA submission, subject to positive results from ENVASARC. The goal of that trial will be to determine the subtypes of sarcoma that best respond to the combination of ENVA and doxorubicin. Assuming positive results in the ENVASARC pivotal trial, we expect the FDA will require a randomized trial to demonstrate a survival benefit. We expect this potential phase three post-approval trial will compare single-agent doxorubicin to doxorubicin with ENVA with progression-free survival as the endpoint. This trial would be expected to enroll patients with UPS and MFS as well as other sarcoma subtypes expected to respond to therapy with ENVA and doxorubicin. We expect to discuss the design of a frontline trial with the FDA at the time of our expected pre-BLA meeting to review the expected submission of data from ENVA-SARC for potential accelerated approval of ENVA in refractory sarcoma. It is important to understand the sales potential in sarcoma with ENVA at parity pricing is not solely the forecast $200 million in peak annual revenues anticipated following approval in refractory UPS and MFS. Our clinical development strategy is designed to create the opportunity for ENVA to broadly benefit patients with sarcoma in the frontline, adjuvant, and neoadjuvant settings by seeking supplemental BLAs. We will now turn to our DNA damage repair inhibitor, TRC102, a program that is financially supported through a cooperative research and development agreement with the National Cancer Institute. The NCI is sponsoring an ongoing randomized phase two trial assessing TRC102 in stage three, non-squamous, non-small cell lung cancer, in combination with chemoradiation. The two-arm trial will enroll 78 patients to assess the benefit of adding TRC102 to current standard of care treatment of pemetrexed, cisplatin, and radiation therapy, followed by consolidated drivalumab maintenance treatment. The primary endpoint of the trial is progression-free survival, and the trial is designed to detect an improvement in progression-free survival at one year from 56% to 75%. 12 sites are now open for enrollment in the US, and the final results are expected in 2025. I will now shift from our pipeline update to discuss our product development platform, or PDP, of CRO independent research. We executed a license of our PDP for an upfront payment of $3 million in November of last year to a biotech company that recognized the value of internalizing its clinical operations to reap the benefits of CRO independent clinical trial implementation that we enjoy at TRACON. The license of the PDP is expected to allow them to run clinical trials as we do at TRACON. for an estimated cost of approximately $100,000 per patient. As a typical CRO charges $300,000 or more per patient, the potential savings from licensing our PDP on a 100-patient trial could be up to approximately $20 million for our partner, in addition to the expected advantages of increased speed of trial execution and pace of enrollment that we enjoy at TRACON by running trials using our in-house team. As we have noted in the past, we expect to further supplement our CAST position through opportunities for non-dilutive capital enabled through our CRO-independent PDP that we believe positions us as one of the most efficient clinical development organizations. We expect to continue to leverage our platform in two ways that provide for potential non-dilutive capital to TRACON. We plan to continue to evaluate drug candidates where TRACON performs clinical trials at a lower fixed cost compared to a CRO, but still at a premium to our cost using a pay for performance model. This is an aligned structure we used in the past, for example, with Johnson & Johnson. Second, we plan to continue to execute non-transferable licenses to our PDP, whereby we are paid to share our proprietary capabilities and know-how to enable another company to independently internalize clinical operations and use these new capabilities to avoid contracting with CROs to execute clinical trials. As has been the experience at TRACON, we believe such an investment could result in substantial time and cost savings for our partner. We believe that over time our PDP has earned strong credibility as a compelling solution for companies who wish to become CRO independent and reap the rewards of conducting trials faster at higher quality and at lower cost compared to trials typically contracted to CROs. At this time, Scott will provide an update on our financials.
spk06: Thank you, Charles, and good afternoon, everyone. Revenue was $3 million and $12 million for the three and 12 months ended December 31, 2023, compared to zero for the comparable periods of 2022. The increase in revenue for the three-month period is related to the PDP license for $3 million, and the increase in the 12-month period is due to the pre-specified $9 million termination fee for the TJ4309 license in conjunction with the previously announced arbitration outcome with IMAP. TRACON's research and development expenses were $1.5 million and $12.3 million, for the three and 12 months ended December 31st, 2023, compared to 3.9 million and 13.9 million for the comparable periods of 2022. The decrease was due to enrollment only in cohort C of NVISARC and the corresponding termination of cohort D of the NVISARC pivotal trial. General and administrative expenses were 1.1 million and 6.7 million for the three and 12 months ended December 31, 2023, compared to $2 million and $14 million for the comparable periods of 2022. The decrease was due to lower legal expenses. Our net income was $0.4 million for the three months ended December 31, 2023, and our net loss was $3.6 million for the 12 months ended December 31, 2023. compared to net losses of $7 million and $29.1 million for the comparable periods of 2022. We recorded other income of $13 million in the 12 months ended December 31st, 2023 due to the arbitration award being collected in the third quarter. Turning to the balance sheet, at December 31st, 2023, our cash, cash equivalents, and restricted cash totaled $8.6 million compared to $17.5 million at December 31st, 2022. With that, I will turn the call back over to Charles.
spk10: Thank you, Scott. As you have heard, our corporate strategy is proceeding as planned. Allow me to recap two key expected events. First, later this quarter, we expect to report updated response data from the NVISAR pivotal trial. Second, we expect to continue to leverage our product development platform to generate non-dilutive capital through either an additional license or through fees captured by replacing a CRO and executing clinical trials for partners at a lower cost compared to a CRO, but still at a premium to our cost using a pay-for-performance model. Thank you for your time and attention, and we are now available to answer your questions.
spk03: Thank you. To ask a question at this time, please press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again. One moment while we compile our Q&A roster. And our first question is going to come from the line of Somit Roy with Jones Research. Your line is open. Please go ahead.
spk07: Hello, everyone, and thank you for taking my question.
spk08: On the NVASAR trial, have you started doing some market research on what would be the What approval rate or response rate would really cause a proper adoption of this drug? Do you expect this response rate to be greater than 20, 25 percent, or physicians are willing to take on a lower response rate because of better safety profile and better, faster infusion? Any color would be helpful.
spk10: Hi, Sharman. Yeah, thanks for the question. Yeah, I think in order to answer your question, I think it's important to understand kind of where the treatment for refractory sarcoma stands. As an example, for diseases like or subtypes like leiomyosarcoma and liposarcoma, there are second-line treatments approved like epirubicin that has a 1% response rate. In the case of UPS and MFS, as we've discussed, the approved standard of care, Votrien, has a 4% response rate. So based on our market research of achieving our target product profile, which is a 15% response rate, we actually expect significant adoption of the therapy. And you have to consider that a 15% response rate, for example, in refractory sarcoma is almost as high as the response rate for doxorubicin, which is about 17% in frontline sarcoma. So I think when you think about sarcoma and response rates, it's a very different dynamic than some larger tumors where there have been much more significant advances in the standard of care. And I guess the bottom line to think about how slow progress has been in sarcoma is to consider the fact that doxorubicin is still the most effective therapy across the board for sarcoma. It's a drug that was approved in 1975, and as I mentioned, even in a frontline setting, only has a 17% response rate. So based on our market research and achieving our target product profile, we do feel that Envifolmab would be adopted by the investigators that are currently using it in the clinical trial, which is another advantage for us. The fact that we're enrolling this trial at 29 sites in the US, with almost every significant key opinion leader in the field, and they have experience with the drug, I think would also be a big positive in terms of potential adoption of the therapy commercially.
spk08: Great, that's really helpful. Second question on the discussion with the FDA on the BLA. Now that the trial has only one arm, what has been the conversation from before if the FDA would like you to run a larger phase three trial with a control arm, or can this be filed?
spk10: Yeah, so our discussion with the FDA were to file the NVISARC data based on achieving the primary endpoint. as a single-arm study with respect to accelerated approval. And the way the trial was designed initially, to be clear, there was a single arm of envafolamab, which is cohort C currently, and there was a combination of combining envafolamab with ipilimumab, which is cohort D, as you know. Given we didn't see superior responses in cohort D compared to cohort C, we took down cohort D in the study. But to be clear, the opportunity for having cohort D in the study was potentially to have dual approval. But that would have only been achieved if cohort D had clearly outperformed cohort C and both of the cohorts had independently achieved the primary endpoint of an 11.25% response rate. So the fact that cohort D did not show increased activity doesn't change with respect to cohort C and the opportunity for approval based on achieving the primary endpoint.
spk08: Right. And the upcoming data at the end of this quarter, could you give us any color on how many patient data will that be and what kind of details will be provided? Thank you.
spk10: Sure. Yeah, so we had announced in December that we enrolled in excess of 70 patients at that time. And our opportunity for providing an updated response rate data is that we want to follow each enrolled patient for at least three months to give them the opportunity to respond to therapy. At this time, we would expect an update on approximately 70 patients, so that would be the additional patients beyond the 46 in December to a total of potentially 70 or so patients in whom we would have at least the opportunity to evaluate for three months. And we would expect to update response rate and also duration response to finish that thought showment. Great. Thank you again. Congratulations. Appreciate your question, Shoma. Thank you.
spk03: Thank you. And one moment for our next question. Our next question is going to come from the line of Ed White with HC Wainwright. Your line is open. Please go ahead.
spk04: Hey, this is Steve. I'm for Ed. So the first question, for the final data from the NSARC study, I think that used to be expected in mid-2024, and now it's second half. So just wondering about the timing.
spk10: Yeah, no, good question. So as I mentioned as part of the call, we do expect full accrual this quarter, which means that we should have response data by mid-year. You know, we mentioned third quarter just because the other key endpoint for the trial is not just the objective response rate, but also duration of response with the key endpoint being there, a median duration response of more than six months being the goal. So in order to have significant data for each patient being on trial at least six months, given we expect full accrual this quarter, we feel that data would then be in third quarter of 2024.
spk04: Okay, yeah, that makes sense. And then for your product development platform, do you have any deals that are being worked through right now, and how would pricing for future deals look like?
spk10: Great question. I can't comment on specific potential transactions, but I would just reiterate the fact that we've clearly monetized the PDP in the past in one of two ways. One is through now licensing the actual technology through a non-transferable license that we accomplished in November. That was for $3 million up front. I think even more potentially substantial would be actually performing services for a company to replace a CRO and give them the access to our in-house platform. And, you know, to make clear what that potentially could mean to TRACON, you know, if it's a phase one study, and, for example, we can do a 30-patient phase one study at $3 million, and we know Acira will charge typically $9 million or more, you know, if we charge $9 million and guarantee the price so it won't be more, that's potentially $6 million in revenue to TRACON. If it's a bigger trial, let's say it's a 100-patient trial, that we can do a TRACON at, say, $10 million, which is $100,000 a patient. Again, a CERO might bid that study at $300,000 a patient. Likely, the final cost to the potential partner company would be more than that. So we're talking about a $30 million cost plus. If we can go to that company and say, we'll guarantee the price at $30, knowing we can do it at $10, you could see that substantial potential revenue for TRACON. So both those are opportunities for TRACON, and that gives you some idea of the potential economics around each of those potential opportunities.
spk05: Okay, thank you. Thank you, Steve. Yeah, please.
spk04: Yeah, can you provide the cash runway and any cost-cutting efforts and just how to support SG&A going forward?
spk10: Sure, just in general. So I think we've got it to mid this year. And I think, as you've seen, we have definitely decreased expenses, I think, related to two things. So one is they no longer have expenses related to the arbitration that was a significant expense the last two years. And second of all, we have, as I mentioned, almost fully enrolled the NVISARC trial. So that will decrease expenses going forward as well. And, you know, potential revenue through licensing or leveraging the platform could result in further quarters in the future. where we're actually income positive. And, you know, I would point out we were net income positive both in quarter four and also quarter three related to leveraging the platform.
spk04: All right. Thank you very much. Thank you, Steve.
spk03: Thank you. And, again, if you would like to ask a question at this time, please press star 1-1 on your telephone.
spk01: One moment.
spk03: And I'm showing no further questions at this time, and I would like to turn the conference back over to Dr. Thur for closing remarks.
spk10: Well, many thanks for the questions, and thank you to the audience for your time and attention, and we look forward to updating you next quarter. Have a great day.
spk03: This concludes today's conference call. Thank you for participating. You may now disconnect. Thank you. Thank you. Good day, ladies and gentlemen, and welcome to the TriCon Pharmaceuticals' fourth quarter and year-end 2023 earnings conference call. At this time, all callers are in a listen-only mode. After the speaker's prepared remarks, we will conduct a question and answer session, and instructions will be given at that time. During today's call, we will be making certain forward-looking statements, including statements regarding expected timing of clinical trials and results, regulatory activities, financing opportunities, our development plan and strategies, potential cost savings, and other benefits deliverable through our product development platform, or PDP. ability to enter into additional license agreements and expectations regarding in the flow of NAB treatments continuing to generate a double-digit objective response rate. These statements are subject to risk and are described in our filings made with the Securities and Exchange Commission, including our annual report on Form 10-K for the year ended December 31, 2023, and subsequent reports on Forms 10-Q, you are cautioned not to place undue reliance on these forward-looking statements unless required by applicable law. We disclaim any obligation to update such statements. Now I would like to turn the call over to Dr. Charles Thor, President and CEO of Tracon Pharmaceuticals. Dr. Thor, please begin.
spk10: Good afternoon, and thank you for joining Tracon's fourth quarter and year-end 2023 Financial Results and Business Update call. I will begin with an update on our pipeline and then review our recent activities. Following that, Scott Brown, our Chief Financial Officer, will review the Financial results for the three and 12 months into December 31st, 2023. Finally, we will conclude by taking your questions. I'll begin with an update on our continued progress with the ongoing phase two and the SART pivotal trial. In December, we reported updated interim safety and efficacy data from 46 patients in cohort C of single agent and the treatment. The objective response rate in the initial 46 patients treated with single agent ENVA was 15% by investigator review and 8.7% by blinded independent central review, or four responses. ENVA monotherapy was generally well tolerated without a single drug-related serious adverse event. Importantly, median duration response by central review was greater than six months. We are on track to complete accrual of the ENVASARC pivotal trial later this quarter. and expect to report updated response data shortly thereafter and prior to the end of this quarter. As a reminder, in order to statistically exceed the 4% objective response rate of Votrien, the only FDA-approved treatment for patients with refractory UPS or MFS, the primary endpoint in NVSARC must show objective responses in 9 out of 80 patients or an 11.25% objective response rate confirmed by central review. Median duration response of greater than six months is a key secondary endpoint. Our goal in ENVISARC is to demonstrate that ENVA has the potential to be both safer and more efficacious than Votrien, a drug with a black box warning for fatal liver toxicity. Based on data from trials of other checkpoint inhibitors and refractory UPS or MFS, we are targeting a 15% response rate for single agent ENVA. Furthermore, we plan to approach the FDA to discuss a BLA filing strategy as soon as we determine nine responses. As a reminder, we have received FAST-TRACK designation for ENVA in the sarcoma subtypes of UPS and NFS that have progressed on one or two prior lines of therapy and received orphan drug designation in soft tissue sarcoma based on activity observed in ENVA-SARC. These evaluations provide important advantages that might expedite regulatory review of ENVA. ENVASARC is designed to provide safety and efficacy data in the refractory sarcoma subtypes of UPS and MFS. We also have a strategy to pursue the approval of ENVA in frontline sarcoma. Doxorubicin is the most common approved therapy used for the treatment of newly diagnosed sarcoma patients. We therefore plan to initiate a trial of ENVA and doxorubicin in the frontline setting of the common sarcoma subtypes, including UPS and MFS, following the completion of enrollment in the pivotal ENVASARC trial, and prior to the expected BLA submission, subject to positive results from ENVASARC. The goal of that trial will be to determine the subtypes of sarcoma that best respond to the combination of ENVA and doxorubicin. Assuming positive results in the ENVASARC pivotal trial, we expect the FDA will require a randomized trial to demonstrate a survival benefit. We expect this potential phase three post-approval trial will compare single-agent doxorubicin to doxorubicin with ENVA with progression-free survival as the endpoint. This trial would be expected to enroll patients with UPS and MFS as well as other sarcoma subtypes expected to respond to therapy with ENVA and doxorubicin. We expect to discuss the design of a frontline trial with the FDA at the time of our expected pre-BLA meeting to review the expected submission of data from ENVA-SARC for potential accelerated approval of ENVA in refractory sarcoma. It is important to understand the sales potential in sarcoma with ENVA at parity pricing is not solely the forecast $200 million in peak annual revenues anticipated following approval in refractory UPS and MFS. Our clinical development strategy is designed to create the opportunity for ENVA to broadly benefit patients with sarcoma in the frontline, adjuvant, and neoadjuvant settings by seeking supplemental BLAs. We will now turn to our DNA damage repair inhibitor, TRC102, a program that is financially supported through a cooperative research and development agreement with the National Cancer Institute. The NCI is sponsoring an ongoing randomized phase two trial assessing TRC102 in stage three, non-squamous, non-small cell lung cancer, in combination with chemoradiation. The two-arm trial will enroll 78 patients to assess the benefit of adding TRC102 to current standard of care treatment of pemetrexed, cisplatin, and radiation therapy, followed by consolidated drivalumab maintenance treatment. The primary endpoint of the trial is progression-free survival, and the trial is designed to detect an improvement in progression-free survival at one year from 56% to 75%. 12 sites are now open for enrollment in the US, and the final results are expected in 2025. I will now shift from our pipeline update to discuss our product development platform, or PDP, of CRO independent research. We executed a license of our PDP for an upfront payment of $3 million in November of last year to a biotech company that recognized the value of internalizing its clinical operations to reap the benefits of CRO independent clinical trial implementation that we enjoy at TRACON. The license of the PDP is expected to allow them to run clinical trials as we do at TRACON. for an estimated cost of approximately $100,000 per patient. As a typical CRO charges $300,000 or more per patient, the potential savings from licensing our PDP on a 100-patient trial could be up to approximately $20 million for our partner, in addition to the expected advantages of increased speed of trial execution and pace of enrollment that we enjoy at TRACON by running trials using our in-house team. As we have noted in the past, we expect to further supplement our CAST position through opportunities for non-dilutive capital enabled through our CRO-independent PDP that we believe positions us as one of the most efficient clinical development organizations. We expect to continue to leverage our platform in two ways that provide for potential non-dilutive capital to TRACON. We plan to continue to evaluate drug candidates where TRACON performs clinical trials at a lower fixed cost compared to a CRO, but still at a premium to our cost using a pay-for-performance model. This is an aligned structure we used in the past, for example, with Johnson & Johnson. Second, we plan to continue to execute non-transferable licenses to our PDP whereby we are paid to share our proprietary capabilities and know-how to enable another company to independently internalize clinical operations and use these new capabilities to avoid contracting with CROs to execute clinical trials. As has been the experience at TRACON, we believe such an investment could result in substantial time and cost savings for our partner. We believe that over time our PDP has earned strong credibility as a compelling solution for companies who wish to become CRO independent and reap the rewards of conducting trials faster at higher quality and at lower cost compared to trials typically contracted to CROs. At this time, Scott will provide an update on our financials.
spk06: Thank you, Charles, and good afternoon, everyone. Revenue was $3 million and $12 million for the three and 12 months ended December 31, 2023, compared to zero for the comparable periods of 2022. The increase in revenue for the three-month period is related to the PDP license for $3 million, and the increase in the 12-month period is due to the pre-specified $9 million termination fee for the TJ4309 license in conjunction with the previously announced arbitration outcome with IMAP. TRACON's research and development expenses were $1.5 million and $12.3 million, for the three and 12 months ended December 31st, 2023, compared to 3.9 million and 13.9 million for the comparable periods of 2022. The decrease was due to enrollment only in cohort C of NVISARC and the corresponding termination of cohort D of the NVISARC pivotal trial. General and administrative expenses were 1.1 million and 6.7 million for the three and 12 months ended December 31, 2023, compared to $2 million and $14 million for the comparable periods of 2022. The decrease was due to lower legal expenses. Our net income was $0.4 million for the three months ended December 31, 2023, and our net loss was $3.6 million for the 12 months ended December 31, 2023. compared to net losses of $7 million and $29.1 million for the comparable periods of 2022. We recorded other income of $13 million in the 12 months ended December 31st, 2023 due to the arbitration award being collected in the third quarter. Turning to the balance sheet, at December 31st, 2023, our cash, cash equivalents, and restricted cash totaled $8.6 million compared to $17.5 million at December 31st, 2022. With that, I will turn the call back over to Charles.
spk10: Thank you, Scott. As you have heard, our corporate strategy is proceeding as planned. Allow me to recap two key expected events. First, later this quarter, we expect to report updated response data from the NVISAR pivotal trial. Second, we expect to continue to leverage our product development platform to generate non-dilutive capital through either an additional license or through fees captured by replacing a CRO and executing clinical trials for partners at a lower cost compared to a CRO, but still at a premium to our cost using a pay-for-performance model. Thank you for your time and attention, and we are now available to answer your questions.
spk03: Thank you. To ask a question at this time, please press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again. One moment while we compile our Q&A roster. And our first question is going to come from the line of Somit Roy with Jones Research. Your line is open. Please go ahead.
spk07: Hello, everyone, and thank you for taking my question.
spk08: On the NVASAR trial, have you started doing some market research on what would be the – What approval rate or response rate would really cause a proper adoption of this drug? Do you expect this response rate to be greater than 20-25% or physicians are willing to take on a lower response rate because of better safety profile and better, faster infusion? Any color would be helpful.
spk10: Hi, Sharman. Yeah, thanks for the question. Yeah, I think in order to answer your question, I think it's important to understand kind of where the treatment for refractory sarcoma stands. As an example, for diseases like or subtypes like leiomyosarcoma and liposarcoma, there are second-line treatments approved like epirubicin that has a 1% response rate. In the case of UPS and MFS, as we've discussed, the approved standard of care, Votrien, has a 4% response rate. So based on our market research of achieving our target product profile, which is a 15% response rate, we actually expect significant adoption of the therapy. And you have to consider that a 15% response rate, for example, in refractory sarcoma is almost as high as the response rate for doxorubicin, which is about 17% in frontline sarcoma. So I think when you think about sarcoma and response rates, it's a very different dynamic than some larger tumors where there have been much more significant advances in the standard of care. And I guess the bottom line to think about how slow progress has been in sarcoma is to consider the fact that doxorubicin is still the most effective therapy across the board for sarcoma. It's a drug that was approved in 1975, and as I mentioned, even in a frontline setting, only has a 17% response rate. So based on our market research and achieving our target product profile, we do feel that Envifolmab would be adopted by the investigators that are currently using it in the clinical trial, which is another advantage for us. The fact that we're enrolling this trial at 29 sites in the US, with almost every significant key opinion leader in the field, and they have experience with the drug, I think would also be a big positive in terms of potential adoption of the therapy commercially.
spk08: Great, that's really helpful. Second question on the discussion with the FDA on the BLA. Now that the trial has only one arm, what has been the conversation from before if the FDA would like you to run a larger phase three trial with a control arm, or can this be filed?
spk10: Yeah, so our discussion with the FDA were to file the NVISARC data based on achieving the primary endpoint. as a single-arm study with respect to accelerated approval. And the way the trial was designed initially to be clear, there was a single arm of envafolamab, which is cohort C currently, and there was a combination of combining envafolamab with ipilimumab, which is cohort D, as you know. Given we didn't see superior responses in cohort D compared to cohort C, we took down cohort D in the study. But to be clear, the opportunity for having cohort D in the study was potentially to have dual approval. But that would have only been achieved if cohort D had clearly outperformed cohort C and both of the cohorts had independently achieved the primary endpoint of an 11.25% response rate. So the fact that cohort D did not show increased activity doesn't change with respect to cohort C and the opportunity for approval based on achieving the primary endpoint.
spk08: Right. And the upcoming data at the end of this quarter, could you give us any color on how many patient data will that be and what kind of details will be provided? Thank you.
spk10: Sure. Yeah, so we had announced in December that we enrolled in excess of 70 patients at that time. And our opportunity for providing an updated response rate data is that we want to follow each enrolled patient for at least three months to give them the opportunity to respond to therapy. At this time, we would expect an update on approximately 70 patients, so that would be the additional patients beyond the 46 in December to a total of potentially 70 or so patients in whom we would have at least the opportunity to evaluate for three months. And we would expect to update response rate and also duration response to finish that thought showment. Great. Thank you again. Congratulations. Appreciate your questions, gentlemen. Thank you.
spk03: Thank you. And one moment for our next question. Our next question is going to come from the line of Ed White with HC Wainwright. Your line is open. Please go ahead.
spk04: Hey, this is Steve. I'm for Ed. So the first question, for the final data from the NSARC study, I think that used to be expected in mid-2024, and now it's second half. So just wondering about the timing.
spk10: Yeah, no, good question. So as I mentioned, as part of the call, we do expect full accrual this quarter, which means that we should have response data by mid-year. You know, we mentioned third quarter just because the other key endpoint for the trial is not just the objective response rate, but also duration of response with the key endpoint being there, a median duration response of more than six months being the goal. So in order to have significant data for each patient being on trial at least six months, given we expect full accrual this quarter, we feel that data would then be in third quarter of 2024.
spk04: Okay, yeah, that makes sense. And then for your product development platform, do you have any deals that are being worked through right now, and how would pricing for future deals look like?
spk10: No, great question. So I can't comment on specific potential transactions, but I would just reiterate the fact that we've clearly monetized the PDP in the past in one of two ways. One is through now licensing the actual technology through a non-transferable license that we accomplished in November, and that was for $3 million up front. I think even more potentially substantial would be actually performing services for a company to replace a CRO and give them the access to our in-house platform. And, you know, to make clear what that potentially could mean to TRACON, you know, if it's a phase one study, and, for example, we can do a 30-patient phase one study at $3 million, and we know Acira will charge typically $9 million or more, you know, if we charge $9 million and guarantee the price so it won't be more, that's potentially $6 million in revenue to TRACON. If it's a bigger trial, let's say it's a 100-patient trial, that we can do a TRACON at, say, $10 million, which is $100,000 a patient. Again, a CRO might bid that study at $300,000 a patient. Likely, the final cost to the potential partner company would be more than that. So we're talking about a $30 million cost plus. If we can go to that company and say, we'll guarantee the price at $30, knowing we can do it at $10, you could see that substantial potential revenue for TRACON. So both those are opportunities for TRACON, and that gives you some idea of the potential economics around each of those potential opportunities.
spk05: Okay, thank you.
spk04: Thank you, Steve. Yeah, please. Yeah, can you provide the cash runway and any cost-cutting efforts and just how to think about SG&A going forward?
spk10: Sure, just in general. So I think we've got it to mid this year. And I think, as you've seen, we have definitely decreased expenses, I think, related to two things. So one is they no longer have expenses related to the arbitration that was a significant expense the last two years. And second of all, we have, as I mentioned, almost fully enrolled the NVISARC trial. So that will decrease expenses going forward as well. And potential revenue through licensing or leveraging the platform could result in further quarters in the future. where we're actually income positive. And I would point out we were net income positive both in quarter four and also quarter three related to leveraging the platform.
spk04: All right. Thank you very much. Thank you, Steve.
spk03: Thank you. And again, if you would like to ask a question at this time, please press star one one on your telephone.
spk01: One moment.
spk03: And I'm showing no further questions at this time, and I would like to turn the conference back over to Dr. Thor for closing remarks.
spk10: Well, many thanks for the questions, and thank you to the audience for your time and attention, and we look forward to updating you next quarter. Have a great day.
spk03: This concludes today's conference call. Thank you for participating. You may now disconnect.
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