TRACON Pharmaceuticals - Q1 2024

Executive Summary

• Q1 2024 was operationally focused: ENVASARC (single‑agent envafolimab) fully enrolled (82 patients) with final response assessment data expected in Q3 2024; interim ORR was 11% (investigator) and 5.5% (BICR) in 73 patients, all confirmed, with median DoR >6 months by BICR.
• OpEx fell sharply YoY as enrollment completed and Cohort D was terminated; R&D was $1.9M vs $5.0M YoY and G&A was $1.4M vs $2.3M YoY; net loss narrowed to $3.2M vs $8.5M YoY.
• Liquidity remains tight: cash and equivalents were $7.9M at March 31, 2024, funding “late into the third quarter of 2024”; management aims to generate non‑dilutive capital via PDP licensing/services; Nasdaq compliance remains a near‑term overhang (reverse split executed; equity/market‑value remedies under evaluation).
• Near‑term stock catalysts: ENVASARC pivotal final data in Q3 2024 and any PDP monetization that bolsters shareholder equity and listing compliance; management plans to approach FDA on BLA if the ENVASARC primary endpoint (≥9 BICR responses out of 82; 11%) is met.

What Went Well and What Went Wrong

What Went Well

• ENVASARC fully enrolled; interim BICR median DoR >6 months with confirmed responses. CEO: “We look forward to reporting the final response assessment data…expected in the third quarter.”.
• Material OpEx reductions: R&D fell due to trial enrollment completion/Cohort D termination; G&A declined on lower legal spend, improving the P&L trajectory YoY.
• Clear regulatory and commercial path outlined if the primary endpoint is achieved, including intent to discuss BLA strategy and plan for a potential post‑approval randomized trial in frontline sarcoma (ENVA + doxorubicin).

What Went Wrong

• Revenue remains de minimis ($0.1M), and the quarter posted a net loss of $3.2M; the business remains dependent on financing and business development.
• BICR ORR of 5.5% in 73 patients at interim was below the 11% primary endpoint threshold, keeping outcome risk for Q3 final data elevated despite investigator‑assessed 11% ORR; success requires ≥9 BICR responses among 82 patients.
• Nasdaq compliance remains unresolved: reverse split completed, but company still needs to satisfy equity/market‑value criteria by the stated deadlines; management is pursuing PDP monetization and other alternatives (press release noted June 3, while call referenced June 30, indicating potential timing uncertainty).

Transcript

Operator (participant)

Good day, ladies and gentlemen, and welcome to TRACON Pharmaceuticals' first quarter 2024 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, plans for future clinical trials, financing opportunities, our development plans and strategies, potential cost savings, and other benefits deliverable through our product development platform, or PDP, ability to enter into additional licensing agreements, ability to generate non-dilutive capital using the PDP, market size estimates, and whether the company's stock will remain listed on NASDAQ.

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 31, 2023, 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, and thank you for joining TRACON's first quarter 2024 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 months ended March 31, 2024. Finally, we will conclude by taking your questions. I will begin with an update on our continued progress with the ongoing phase II ENVASARC pivotal trial. In April, the Independent Data Monitoring Committee recommended the trial continue as planned following a review of interim safety and efficacy data from 73 patients in cohort C of single-agent ENVA treatment. The objective response rate in patients treated with single-agent ENVA was 11% by investigator review and 5.5% 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 independent central review was greater than six months. We have completed accrual of 82 patients dosed with ENVA as a single agent at 600 milligrams and expect to report final data in the third quarter. As a reminder, in order to statistically exceed the 4% objective response rate of Votrient, the only FDA-approved treatment for patients with refractory UPS or MFS, the primary endpoint in ENVASARC must show objective responses in nine out of 82 patients, or an 11% objective response rate confirmed by independent central review. Median duration response of greater than six months is a key secondary endpoint. Our goal in ENVASARC is to demonstrate that ENVA has the potential to be both safer and more efficacious than Votrient, a drug with a black box warning for fatal liver toxicity.

We plan to approach the FDA to discuss a BLA filing strategy if we determine 9 responses by independent central review. As a reminder, we have received fast-track designation for ENVA in the sarcoma subtypes of UPS and MFS that have progressed on 1 or 2 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 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 commonly 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, including achieving the primary endpoint. 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 III 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 a pre-BLA meeting to review the expected submission of data from ENVASARC for potential accelerated approval of ENVA in refractory sarcoma, assuming positive results from the ENVASARC pivotal trial. It is important to understand the sales potential in sarcoma with ENVA at parity pricing is not solely the forecasted $200 million in peak annual ENVA revenues anticipated if approved 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, that is financially 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 consolidative durvalumab maintenance treatment. The primary endpoint of the trial is progression-free survival, and the trial is designed to detect an improvement in PFS at one year from 56% to 75%. 15 sites are now open for enrollment in the U.S., and 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 execute 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 for oncology trials. As a typical CRO charges $300,000 or more per patient in this indication, the potential savings from licensing our PDP on a 100-patient trial could be up to approximately $20 million for a partner, in addition to 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 cash 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 potential non-dilutive capital to TRACON. First, we plan to continue to evaluate drug candidates whereby TRACON captures revenue by performing 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. As previously announced, on March 7, 2024, we had a hearing with Nasdaq regarding a letter of non-compliance that we received on June 8, 2023, regarding two deficiencies.

We presented a compliance plan to cure our deficiencies to Nasdaq at the hearing on March 7. On March 20, the Nasdaq hearings panel granted our request for continued listing on the Nasdaq capital market, subject to us regaining compliance with all applicable continued listing requirements. We effected a reverse stock split on April 9 to cure one of the Nasdaq deficiencies and are considering alternatives to cure the other Nasdaq deficiency, which requires us to have a market value for our listed securities of at least $35 million or meet the stockholders' equity requirement of $2.5 million by June 3. At this time, Scott will provide an update on our financials.

Scott Brown (CFO)

Thank you, Charles, and good afternoon, everyone. TRACON's research and development expenses were $1.9 million for the three months ended March 31, 2024, compared to $5 million for the comparable period of 2023. The decrease was due to termination of cohort D of the ENVASARC pivotal trial in 2023. General and administrative expenses were $1.4 million for the three months ended March 31, 2024, compared to $2.3 million for the comparable period of 2023. The decrease was due to lower legal expenses. Our net loss was $3.2 million for the three months ended March 31, 2024, compared to $8.5 million for the comparable period of 2023. Turning to the balance sheet, at March 31, 2024, our cash, cash equivalents, and restricted cash totaled $8 million compared to $8.6 million at December 31, 2023. 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 final data from the ENVASARC pivotal trial. Second, we expect to continue to leverage our product development platform to generate non-dilutive capital through either an additional license or by capturing revenue 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.

Operator (participant)

Thank you. To ask a question, 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, please, for our first question. Our first question comes from the line of James Malloy with Alliance Global Partners.

Matt Venezia (Equity Research Senior Associate)

Hi, guys. This is Matt Venezia on for Jim Malloy. I just had a couple of questions, first regarding the PDP. Can you go over a little bit more about the margins you would expect on a clinical trial that you guys would run through PDP, for you guys and for your client?

Charles Theuer (President and CEO)

Appreciate the question. Thanks so much. I can give an example that serves as a precedent case. For example, we have run in the past a phase I trial for I-Mab whereby we were paid $9 million to conduct a clinical trial, again, a phase I trial. Our total expense for that trial was a bit under $3 million. So in other words, we can run a trial at roughly $100,000 a patient in oncology. We know CROs are bidding those trials at roughly $300,000 per patient. So for example, for a 30-patient trial, if we can do it at $3 million, which is $100,000 per patient, and we can guarantee the total cost of the trial to a partner at $300,000 a patient, which for 30 patients is about $9 million, it's a benefit to our partner because that's a guaranteed price.

It won't go up, which, based on change orders, the CRO bid price of $300,000 a patient could go up substantially beyond that. We'll guarantee a $300,000 a patient, knowing the profit margin there is about 200% for us.

Matt Venezia (Equity Research Senior Associate)

Got it. All right. So 200%. Thank you. And then could you go over the potential solutions in terms of regaining compliance with the minimums by early June, I think you had talked about, and how that would align with the timeline for the final ENVASARC data?

Charles Theuer (President and CEO)

Yeah. Yeah, great question. So with respect to the Nasdaq compliance, to do one of two things: either increase the market cap to $35 million or regain compliance to increase in the stockholder equity to $2.5 million. Our preferred approach is to leverage the PDP, that if we can leverage the PDP and gain revenue by, for instance, as you just pointed out, doing a trial for someone else, including a significant upfront payment to do that, or by licensing the PDP as we did in November, that's the preferred option for us to have capital come to the company that would potentially cure the stockholder equity deficit. Other options would potentially be fundraising as well. But our preferred approach clearly is leveraging the PDP through business development.

Matt Venezia (Equity Research Senior Associate)

Gotcha. Okay. Can you go over how that would align with the timeline for ENVASARC and whether?

Charles Theuer (President and CEO)

Yeah. So ENVASARC.

Matt Venezia (Equity Research Senior Associate)

Yeah. Sorry.

Charles Theuer (President and CEO)

No, no. Appreciate the question. Sorry. Yeah. So ENVASARC data, we're expecting final data in Q3. So that's just past June, is when Q3 starts. We haven't given a more definitive time within Q3, but that would expect to be just after the NASDAQ compliance date of June 3.

Matt Venezia (Equity Research Senior Associate)

Okay. Got it. All right. Thank you for the questions. I appreciate it.

Charles Theuer (President and CEO)

Thanks for the questions.

Operator (participant)

Thank you. And once again, ladies and gentlemen, to ask a question, please press star one one on your telephone. No further questions. So with that, I'll hand the call back over to CEO and President, Dr. Charles Theuer, for any closing remarks.

Charles Theuer (President and CEO)

I'd like to thank the audience for your time and attention and your questions, and we look forward to talking with you again next quarter.

Operator (participant)

Ladies and gentlemen, thank you for participating. This does conclude today's program, and you may now disconnect.