TRACON Pharmaceuticals - Q2 2023

Executive Summary

• Q2 2023 was defined by a one-time $9.0M collaboration revenue from the termination of the TJ4309 license and continued progress of the ENVASARC pivotal trial; net loss was $6.3M ($0.20/sh), with a $13.0M arbitration-related gain to be recognized in Q3 on a pro forma basis Q2 would have been profitable ($6.7M, $0.22/sh).
• Management reiterated a double-digit ORR for single-agent envafolimab (ENVA) with no >Grade 2 drug-related AEs, confirmed by central review; the combination cohort with Yervoy showed no synergy and was discontinued, reducing costs and accelerating timelines.
• Cash was $1.9M at 6/30/23, but the July collection of a $22M arbitration award drove pro forma cash to ~$9.0M and extends runway into early 2024; an additional $4.4M remains in a trust pending legal fee resolution.
• Catalysts: second and final IDMC interim efficacy assessment in Q3 (futility bar already met), full accrual of single-agent cohort in Q4 2023, and final ENVASARC data mid-2024; management also aims to monetize its CRO-independent platform for non-dilutive capital in 2023 (call correction vs press release).

What Went Well and What Went Wrong

• What Went Well

  • Clinical efficacy: “ongoing double-digit ORR for single agent envafolimab… without any > Grade 2 drug related toxicity,” exceeding the futility rule heading into the interim efficacy analysis.
  • IDMC threshold: “we need at least 3 responses… we’ve exceeded that threshold,” with confirmed responses by investigator and central review.
  • Cost discipline/platform efficiency: fully burdened per-patient cost in ENVASARC “less than $90,000,” significantly below typical CRO bid estimates (~$300,000).

• What Went Wrong

  • Combination strategy: “The combination of ENVA with YERVOY did not demonstrate synergy… and we therefore terminated enrollment in Cohort D,” narrowing upside from combo efficacy.
  • Liquidity tightness at quarter-end: cash and equivalents were $1.9M at 6/30/23 before arbitration collection, highlighting balance-sheet stress pre-award.
  • One-time costs: $4.4M success fee related to arbitration legal fees recorded in Q2, elevating operating expenses for the period.

Transcript

Operator (participant)

Good day, ladies and gentlemen, and welcome to the TRACON Pharmaceuticals Second Quarter 2023 Earnings Conference Call. At this time, all callers are in a listen-only mode. After the speaker's prepared remarks, we'll 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 of regulatory activities, financing opportunities, future expenses, and cash runway, our development plans and strategy, and with the recovery of the award from our arbitration with I-Mab. These statements are subject to various risks that 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 31st, 2022, and subsequent quarterly reports on Form 10-Q.

You are cautioned not to place undue reliance on these forward-looking statements, and 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 Theuer, President and CEO of TRACON Pharmaceuticals. Dr. Theuer?

Charles Theuer (President and CEO)

Good afternoon, thank you for joining TRACON's Second Quarter 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 discuss our financial results for the three and six months ended June 30, 2023. Finally, we will conclude by taking your questions. I'll begin with an update on our continued progress with the ongoing ENVASARC pivotal trial. In June, the Data Monitoring Committee reviewed interim safety and efficacy data for more than 80 patients, equally randomized into cohort C of single-agent envafolimab treatment, or cohort D of envafolimab given in combination with Yervoy. Patients in cohort C, who had at least two on-study CT scans, continued to demonstrate a double-digit objective response rate assessed by investigator and blinded independent central review.

Envafolimab was generally well-tolerated without a single greater than Grade 2 drug-related adverse event. The combination of envafolimab with Yervoy did not demonstrate synergy when compared to single-agent envafolimab treatment, we therefore terminated enrollment in cohort D. This decision has resulted in a reduction in trial costs and acceleration of the timeline to final ENVASARC data. We expect full accrual of the 80 patients into cohort C of treatment with single-agent envafolimab in the fourth quarter and final data, including duration of response by mid-2024. In addition, a protocol-mandated DMC review will be conducted and reported after the 46 patients treated with envafolimab has completed a minimum of 12 weeks of efficacy evaluations.

We expect the DMC review to occur this quarter, as the ENVASARC trial has enrolled 180 patients to date, including 56 of the 80 expected patients in cohort C of single-agent envafolimab treatment. This DMC review includes a futility threshold that has already been achieved based on responses seen to date. As a reminder, the primary endpoint in ENVASARC is objective response rate by RECIST, confirmed by blinded independent central review with nine out of 80 objective responses on 11.25% objective response rate, defines the level of response that satisfies the primary endpoint of the study. To statistically exceed the 4% objective response rate of Votrient, the only FDA-approved treatment for patients with refractory UPS or MFS.

Therefore, a double-digit response rate at the time of interim analysis is meaningful, indicating that we are on track to achieve the primary endpoint of the study. Notably, Votrient is a drug with a black box warning for fatal liver toxicity. Our goal in ENVASARC, therefore, is to demonstrate that envafolimab has the potential to be both safer and more efficacious than Votrient. Based on data from trials of other checkpoint inhibitors in refractory UPS or MFS, we are targeting a 15% response rate for single-agent envafolimab. 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 envafolimab in the sarcoma subtypes of UPS and MFS 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 ENVASARC. These designations provide important advantages that might expedite regulatory review and commercialization of envafolimab. ENVASARC is designed to provide safety and efficacy data for the approval of envafolimab in the refractory sarcoma subtypes of UPS and MFS. We also have a strategy for the approval of envafolimab in frontline sarcoma. Based on expected synergy between envafolimab and Yervoy, we licensed YH001, a potential best-in-class CTLA-4 antibody from Eucure Biopharma in October 2021 and began a phase I/II clinical trial evaluating a triplet that included YH001, envafolimab, and doxorubicin chemotherapy for the treatment of frontline sarcoma.

Given the lack of synergy observed in ENVASARC between envafolimab and the CTLA-4 antibody Yervoy and available data from patients treated with envafolimab and YH001, we have decided to end enrollment into the trial as originally designed. We now plan to initiate a modified trial of envafolimab and doxorubicin in the frontline setting of common sarcoma subtypes, including UPS and MFS, following completion of enrollment in the pivotal ENVASARC trial. The goal of that modified trial will be to determine the subtypes of sarcoma that best respond to the combination of envafolimab and doxorubicin. Assuming positive results in the ENVASARC pivotal trial and potential accelerated approval of envafolimab, we expect the FDA will require a randomized trial to demonstrate a survival benefit. We now expect this potential phase III post-approval trial will compare a single agent, doxorubicin, to doxorubicin with envafolimab, 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 shown to respond to therapy with envafolimab 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 ENVASARC for potential accelerated approval of envafolimab in refractory sarcoma. It is important to understand the sales potential in sarcoma with envafolimab at parity pricing is not solely the forecasted $200 million in peak annual envafolimab revenues anticipated following approval in refractory UPS and MFS. Our clinical development strategy is designed to create the opportunity for envafolimab to broadly benefit patients with sarcoma in the frontline, adjuvant, and neoadjuvant settings by seeking supplemental BLA approvals.

We will now turn to our DNA damage repair inhibitor, TRC102, that is supported through a cooperative research and development agreement with the National Cancer Institute. The NCI is sponsoring an ongoing randomized phase II trial assessing TRC102 in Stage III 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 durvalumab maintenance treatment. The primary endpoint of the trial is progression-free survival. The trial is designed to detect an improvement in PFS at one year from 56% to 75%. Nine sites are open for enrollment in the U.S., and final results are expected in 2025. Next, I will provide an update on the collection of our arbitration award from I-Mab.

On April 24th, we were informed the tribunal ruled in our favor for certain claims and rendered an award to TRACON in the aggregate amount of approximately $23 million. Among other findings, the tribunal declared the TJ004309 trial complete, which entitled TRACON to $9 million, and also awarded legal fees and costs to TRACON. In July, we collected $22 million from I-Mab in satisfaction of the International Chamber of Commerce award to TRACON, announced in April. $10.5 million of the collected amount was used to repay our litigation financing facility in full, and net proceeds collected to date of $7.1 million are expected to fund the company's operations as currently planned into the first half of 2024.

An additional $4.4 million of the arbitration award remains in a client trust account administered by our law firm at this time, the disbursement of which is predicated on discussions as to the amount of success-based deferred legal fees the firm is due. Following these discussions, disbursement of all or a portion of that amount is expected later this year. We expect to further extend our runway by securing non-dilutive capital through leveraging our CRO-independent product development platform that we believe positions us as one of the most efficient clinical development organizations. As an example of our efficiency, our fully burdened per-patient cost for dosing patients in the ENVASARC trial is less than $90,000 per patient. This compares favorably with typical CRO bid estimates of $300,000 per patient, that may be even more expensive if change order charges are considered.

In addition, by managing all regulatory filings internally, we believe we significantly shorten trial duration by expediting the approval of protocol amendments and consent forms. We also emphasize quality through the use of a team of reliable site monitors who are highly experienced with relevant oncology response criteria to ensure accurate data reporting. These attributes of our product development platform have been the basis for capital infusions from partners like Johnson & Johnson, who have collaborated with us to run trials of their drug candidates. Our current focus is to work with companies in one of two ways. First, by replacing a CRO to lower their anticipated costs, but still generate a substantial profit for TRACON. Two, to teach our operational capabilities to a company with an emerging pipeline who plan to conduct multiple trials.

This offering would include our platform of advanced clinical trial management, data management, and safety reporting that will enable our collaborator to conduct trials at a cost of less than 1/3 of what they may otherwise pay to a CRO. Leveraging our cost-efficient, CRO-independent product development platform to generate non-dilutive capital before the end of this year is a top priority for the company. In that regard, please note the corrected press release. Let me reiterate, we expect to generate non-dilutive capital before end of this year by leveraging our CRO-independent product development platform. At this time, Scott will provide an update on our financials.

Scott Brown (CFO)

Thank you, Charles. Good afternoon, everyone. Collaboration revenue was $9 million for the three and six months ended June 30, 2023, compared to 0 for the comparable periods of 2022. The increase in revenue was from the pre-specified $9 million termination fee for the TJ4309 license in conjunction with the arbitration outcome. TRACON's research and development expenses were $3.5 million and $8.5 million for the three and six months ended June 30, 2023, compared to $2.9 million and $5.9 million for the comparable periods of 2022. The increase was related to envafolimab drug purchase in the first quarter of 2023, as well as higher enrollment in ENVASARC.

General and administrative expenses were $1.9 million and $4.3 million for the three and six months ended June 30, 2023, compared to $3.3 million and $9.8 million for the comparable periods of 2022. The decrease was due to lower legal expenses. We incurred a success fee of $4.4 million in the three and six months ended June 30, 2023, related to legal fees for the arbitration and collection of the award in July. As Charles mentioned, this amount remains in a client trust account administered by our law firm, the disbursement of which is predicated on discussions as to the amount of the success-based deferred legal fees the firm is due. Following these discussions, disbursement of all or a portion of that amount is expected later this year.

Our net loss was $6.3 million and $14.8 million for the three and six months ended June thirtieth, 2023, compared to $6.2 million and $15.7 million for the comparable periods of 2022. We will record a gain of $13 million related to the collection of the arbitration award in the third quarter of this year. Had this been recognized in the three and six months ended June 30, 2023, it would have resulted in net income of $6.7 million for the three-month period and a net loss of $1.8 million in the six-month period on a pro forma basis.

Turning to the balance sheet at June 30, 2023, our cash, cash equivalents, and restricted cash totaled $1.9 million, compared to $17.5 million at December 31st, 2022. As Charles mentioned, we collected the arbitration award in July, and net proceeds to date are approximately $7.1 million, which, had this been collected prior to June 30, 2023, would have resulted in ending cash of $9 million on a pro forma basis. With the award collected, we expect our current capital resources to fund the company into early 2024. With that, I will turn the call back over to Charles.

Charles Theuer (President and CEO)

Thank you, Scott. As you have heard, our corporate strategy is proceeding as planned. Allow me to recap two key expected events. First, in the third quarter, we expect to report the second and final mandated DMC interim efficacy assessment in ENVASARC that includes the first 46 patients dosed with 600 mg of envafolimab. The interim efficacy assessment includes a futility threshold that has already been achieved based on responses seen to date. Second, this year, we also expect to further leverage our unique product development platform to enable companies to benefit from our capabilities and realize for themselves the substantial clinical trial time and cost savings we enjoy at TRACON, while allowing TRACON to generate non-dilutive capital. Thank you for your time and attention. We are now available to answer your questions.

Operator (participant)

Thank you. As a reminder, to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. Please stand by while we compile the Q&A roster. One moment for our first question. Our first question comes from Joel Beatty from Baird. Your line is now open.

Joel Beatty (Biotechnology Equity Research Analyst)

Great. Thanks for taking the questions. The first question is on the, the comment that the futility threshold for the second interim efficacy assessment has already been met. Can you elaborate on, on exactly what that means? I mean, does, does that mean at an earlier point in time, you saw the, the percent response needed to hit that assessment, or, or there's, you actually kinda hit the, the numerator, that you were looking to see, you know, as if the denominator had been 46?

Charles Theuer (President and CEO)

Yeah, Joel, thanks for the question. With respect to the futility threshold, at the 46 patient evaluation, we need at least three responses in order to meet the threshold futility bar. We had disclosed, even at the DMC review in June, that, based on the data at that time, which continues to be the case, we've exceeded that threshold.

Joel Beatty (Biotechnology Equity Research Analyst)

Terrific. Then are, are those, all confirmed responses?

Charles Theuer (President and CEO)

Yeah, we disclosed at the June meeting that, we had seen confirmed responses both by investigator and, central review that generated double-digit response rate.

Joel Beatty (Biotechnology Equity Research Analyst)

Terrific. Thinking ahead to the final analysis, is that something that you anticipate announcing kinda at the end of the trial, or could you announce it at an earlier point in time once, I believe it's nine responses have been seen?

Charles Theuer (President and CEO)

Yeah. No, great question, Joel, and I'm glad you brought this up. We had been very, I think, general about response rates, saying we've seen double-digit response rates both at the DMC meeting, I believe that was in December, and then also in June, because we didn't want to bias potential accrual, or we didn't want to bias patients who may be assigned in a randomized trial to one arm versus another, where if there were a differential response rate, and we report that response rate, and a patient doesn't get assigned to the preferred arm, if you will, they might drop out of the study. As you know, now we're dosing a single-arm trial.... based on the single arm being the cohort C of single agent envafolimab.

With respect to moving forward, but I don't feel like we're restricted with respect to disclosing exact response rates. Going forward, do expect us to disclose exact response rates because we don't have to, if we will protect the integrity of the randomization, given we're no longer enrolling the cohort of envafolimab plus Yervoy.

Scott Brown (CFO)

Great, thank you.

Charles Theuer (President and CEO)

To answer your question, do expect us to report data moving forward on a more routine basis with explicit response rates that heretofore we were unable to report.

Operator (participant)

Thank you. If you would like to ask a question, that is star one, one. Again, if you would like to ask a question, that is star one, one. One second for our next question. Our next question comes from Ed White, from H.C. Wainwright.

Ed White (Managing Director and Senior Biotechnology Analyst)

Good afternoon. Thanks for taking my questions. Just a couple of questions for Scott. How should we be thinking of research and development costs going forward, now that the combo arm of this study has been discontinued? Are there some costs that we'll see in the third quarter for sort of ramping down the trial, so we should see more of an impact in the fourth quarter? How should we be thinking of that?

Scott Brown (CFO)

Thanks for the question, Ed. We'll see some impact in the third quarter and the fourth quarter, but not too much. I mean, I would expect them to go down slightly, but not that much considering, you know, our per-patient cost is so low.

Ed White (Managing Director and Senior Biotechnology Analyst)

Okay, thank you. Another question on expenses. You know, how should we be thinking of general administrative costs going forward now that the arbitration is over? Is this sort of the runway that we saw this quarter? Should we think that that's going to continue like that? Or, you know, any guidance you can give us there will be appreciated. Thank you.

Scott Brown (CFO)

Yeah. No, I, I would expect this, this amount that we had in Q2 to be our cost going forward. It should be right around there, barring anything else, until we do ramp up commercial activities once we file for potential approval of envafolimab.

Ed White (Managing Director and Senior Biotechnology Analyst)

Okay, thanks for taking my questions.

Scott Brown (CFO)

Yep. Thanks, Ed.

Charles Theuer (President and CEO)

Thanks, Ed.

Operator (participant)

Thank you. I am showing no further questions. I would now like to turn the call back over to Dr. Theuer for closing remarks.

Charles Theuer (President and CEO)

Thank you for your time and attention. We look forward to updating you next quarter. Have a great day.

Operator (participant)

This concludes today's conference call. Thank you for participating. You may now disconnect.