Q2 2024 Earnings Summary
- After a low Q1, FYARRO demand rebounded strongly in Q2 across all key segments, and the company remains very positive on the outlook for continued incremental growth in Q3 and Q4.
- There is palpable excitement and strong support from the medical community for nab-Sirolimus in endometrial cancer (EEC), with specialists hopeful that it can replace chemotherapy and fill a significant treatment gap.
- Physicians have shown excellent reactions to nab-Sirolimus for TSC1 and TSC2 mutations, particularly for late-line patients with identifiable mutations, indicating strong interest in the therapy's potential.
- Limited Market for FYARRO in PEComa: The CEO acknowledged that PEComa is a very rare indication with only 200-300 patients in the U.S., and the company has penetrated most of the marketplace, indicating that any further growth will be incremental. This severely limits the potential for significant sales growth of FYARRO in this indication.
- Challenges in Maintaining Consistent FYARRO Sales Growth: The company experienced a low Q1 for FYARRO sales, which the CEO attributed to possible cannibalization at large centers. Although there was a rebound in Q2, this suggests potential challenges in sustaining consistent sales growth for FYARRO.
- Uncertainty Around PRECISION1 Trial Outcomes: The company's future growth is heavily dependent on the success of the PRECISION1 trial. However, the CEO stated that they are not drawing any anticipation regarding the direction of the interim data and will have to wait for the report, indicating uncertainty about the trial's outcome and the potential path to regulatory approval. ,
| Metric | Period | Previous Guidance | Current Guidance | Change |
|---|---|---|---|---|
Cash Runway | Q2 2024 | $88.3 million to fund operations into Q4 2025 | $78.6 million to fund operations into Q4 2025 | lowered |
PRECISION1 Trial | Q2 2024 | Fully enrolled with the next interim readout in Q3 2024 covering 80 patients and study completion expected by end of 2024 | Fully enrolled with second interim data planned in Q3 2024 and completion expected by end of 2024 | no change |
EEC/NET Trials | Q2 2024 | Initial data from the endometrioid-type endometrial cancer (EEC) and NET trials expected by end of 2024 | Initial data from Phase II trials for endometrial cancer and NET expected later in 2024 | no change |
FYARRO Sales | Q2 2024 | FYARRO net product sales were $5.4 million in Q1 2024, representing an 8.8% decrease with expectations to return to growth in Q2 2024 | The company now expects incremental growth in FYARRO sales in Q3 & Q4 2024 following a strong rebound in Q2 2024 | raised |
Market Opportunity for TSC1/TSC2 Mutations | Q2 2024 | no prior guidance | Projected $300 million to $600 million market opportunity in the U.S. for nab‑Sirolimus in TSC1/TSC2-mutated cancers | no prior guidance |
| Topic | Previous Mentions | Current Period | Trend |
|---|---|---|---|
PRECISION1 Trial | Emphasized as a major growth driver in Q4 2023 and Q1 2024 , with full enrollment and Q3 2024 interim readout anticipated. | Remains a key focus, with the next interim readout in Q3 2024 and full results expected in early 2025. | Continued positive emphasis, no new uncertainties introduced. |
FYARRO Sales Performance | Q4 2023 sales were $6.3M, stabilizing at $6–$6.5M. In Q1 2024, sales dipped to $5.4M (−8.8% YoY), attributed to ordering patterns and fewer new patient starts. | Rebounded to $6.2M, a 15% increase from Q1, with a 14% rise in ordering accounts. | Returned to growth after Q1 dip. |
EEC (Endometrial) | Focused on a Phase II trial with nab-Sirolimus + letrozole in Q4 2023 and Q1 2024. Aim to improve on prior mTOR combos and offer a chemo alternative. | Emphasized rapid enrollment and strong community support; potential to replace chemotherapy in advanced or recurrent EEC. | Increasing optimism around replacing chemo with nab-Sirolimus combo. |
Shifts in FYARRO Sentiment | Q4 2023 stable around $6–$6.5M. Q1 2024 saw a decline to $5.4M, but management remained confident in rebound. | Confidence restored with strong Q2 rebound, noting sustainable incremental growth. | Positive shift after Q1 shortfall. |
PEComa Growth Limits | Recognized as ultra-rare in Q4 2023, with potential market saturation. No explicit mention in Q1 2024. | Management reiterated limited growth due to a very small patient population (200–300 patients in the U.S.) and near saturation. | Consistent acknowledgment of constrained potential. |
PRECISION1 Uncertainty | No Q4 2023 or Q1 2024 discussion of uncertainty; only optimism about results and regulatory path. | No mention of new concerns in Q2 2024; continued optimism for interim and final data. | No emergence of uncertainty; outlook remains positive. |
Mirati Collaboration | Mutual termination mentioned in Q1 2024 to prioritize nab-Sirolimus trials. In Q4 2023, collaboration was still ongoing with no new data shared. | Not mentioned in Q2 2024. | Omission after prior termination note. |
Cannibalization Concern | Not discussed in Q4 2023. In Q1 2024, concern noted as some large centers enrolled patients in trials instead of commercial use. | Mentioned as a factor for low Q1 sales but noted rebound in Q2 across key segments. | Still relevant, though recent sales rebound offset the impact. |
Impact of TSC1/TSC2, EEC, NETs | Q4 2023 and Q1 2024 highlighted large future potential for nab-Sirolimus in these indications, underscoring multibillion-dollar opportunities. | Reiterated significant upside if upcoming data are positive, supporting multi-indication expansion and addressing major unmet needs. | High-impact potential remains central to Aadi’s long-term growth strategy. |
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PRECISION1 Trial and FDA Discussions
Q: What are the expectations for the second interim data of the PRECISION1 trial and potential FDA discussions?
A: The second interim data for PRECISION1, our primary endpoint analysis on two-thirds of patients (80 patients, 40 in each arm), will be reported in Q3. This will include independently assessed overall response rate after a minimum of six months follow-up, key demographics, and select secondary data. We anticipate that this data will serve as a good foundation for data-driven discussions with the FDA about a potential path to submission. -
Trial Design Alterations Post-Interim Analysis
Q: Could the design of PRECISION1 change based on interim results?
A: With the trial now fully enrolled at 120 patients, we do not anticipate making adjustments to the trial design based on the interim results. We expect to report the full data set in early 2025 after patients complete their follow-up period. -
FYARRO Sales Growth Expectations
Q: Do you expect continued incremental growth for FYARRO sales?
A: Yes, after a low Q1, we saw a strong rebound in demand across all key segments in Q2, and this positive trend is sustaining into the next quarter. We believe we will continue to see incremental growth in Q3 and Q4. -
EEC and NET Studies Data Updates
Q: What should we expect from the initial data update for the EEC and NET studies later this year?
A: For the endometrial cancer (EEC) trial, enrollment has been rapid. We have completed the entire first cohort and are well into the second cohort. By the end of the year, we'll provide a full summary report on the first cohort and partial information on the second. For the neuroendocrine tumor (NET) program, we also expect to report complete data on the first cohort by the end of the year. -
Future Expansion and Combinations for nab-Sirolimus
Q: Are there plans for future drug combinations or target indications for nab-Sirolimus?
A: We are exploring opportunities for future combinations that build on the preferential tumor uptake of nab-Sirolimus. While it's premature to comment on specifics, we're looking forward to the results from our initial trials over the second half to set the path for the future. -
KOL Feedback on TSC1/2 vs. Indication-Specific Approaches
Q: What is the KOL community's excitement level for the TSC1/2 pan-tumor approach versus indication-specific approaches like EEC and NET?
A: Excitement is high in both areas but manifests differently. In the PRECISION1 trial, physicians are interested in finding solutions for late-line patients through targeted mutations. In the EEC trial, the community is highly enthusiastic about potentially replacing chemotherapy as frontline treatment, and enrollment has been rapid due to strong support. -
Tumor Types in PRECISION1 Trial
Q: Is the PRECISION1 trial favoring any particular tumor types?
A: No, PRECISION1 is a truly tumor-agnostic trial. We enroll any patients who qualify regardless of tumor type based on their TSC1/TSC2 status. The FDA has provided no guidance to bias the trial toward specific indications, and we anticipate the trial will be reviewed as tumor-agnostic. -
Disclosing Data Based on Lines of Treatment
Q: Will you disclose data distinguishing patients by lines of treatment in the next release?
A: We will certainly disclose the overall response rate. Whether we see a meaningful difference between lines of treatment will depend on the data. In our early interim analysis in December, we didn't see any particular pattern, with responses spread across different lines of treatment. -
Comparing EEC Trial to Other mTOR Inhibitor Data
Q: How does the EEC trial compare to previous studies with other mTOR inhibitors, and what should we pay attention to regarding patient demographics?
A: Our EEC study is based on earlier trials that established platinum and paclitaxel as standard care, and the combination of everolimus and letrozole showing promising results. In chemo-naive patients, everolimus and letrozole had a progression-free survival (PFS) of 28 months, doubling the 14 months seen with chemotherapy. We aim to replicate or improve on these results. It's important to compare patient demographics appropriately, and the community has been very supportive of this approach. -
Expectations from NET Phase II Program
Q: What are you looking for in the NET Phase II program regarding efficacy and safety?
A: Historically, mTOR inhibitors have shown benefits in neuroendocrine tumors by extending progression-free survival, though with low overall response rates. We hope to see improved initial response rates with nab-Sirolimus, which could lead to better long-term outcomes for these patients. -
Maximum Sales Potential in PEComas
Q: Do you have an updated view on maximum sales in PEComas in the U.S.?
A: PEComa is a very rare indication with about 200 to 300 patients in the U.S. We are focused on finding these patients and ensuring they receive the right treatment. It's hard to predict exactly where sales can land over time, and any further growth will be incremental. -
Timing and Venue of Interim Data Release
Q: When and where will the second interim data be released?
A: We plan to report the second interim data later in Q3, and we anticipate this will be through a company presentation rather than at a specific conference. -
Future Combinations Based on Preferential Tumor Uptake
Q: Given the preferential tumor uptake of nab-Sirolimus, are you considering any drug combinations?
A: Yes, we're exploring opportunities for combinations that build on this advantage, but it's premature to comment on specifics. We're looking forward to the results from our initial trials to set the future path.