FibroGen Q4 2024 Earnings Report

FibroGen EPS Results

• Actual EPS: -$2.00
• Consensus EPS: -$2.00
• Beat/Miss: Met Expectations
• One Year Ago EPS: N/A

FibroGen Revenue Results

• Actual Revenue: $3.14 million
• Expected Revenue: $24.91 million
• Beat/Miss: Missed by -$21.77 million
• YoY Revenue Growth: N/A

FibroGen Announcement Details

• Quarter: Q4 2024
• Date: 3/17/2025
• Time: After Market Closes
• Conference Call Date: Monday, March 17, 2025
• Conference Call Time: 5:00PM ET

FibroGen (NASDAQ:FGEN)., headquartered in San Francisco, California, is a biopharmaceutical company focused on the discovery, development and commercialization of novel therapies for serious unmet medical needs. Since its founding in 1993, the company has built a diversified portfolio encompassing small molecules and biologics that address anemia, fibrotic diseases and oncology.

The company’s lead product, roxadustat, is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor designed to treat anemia in chronic kidney disease. Roxadustat has received regulatory approval in China, Japan and several other markets, and is being developed globally through strategic partnerships with Astellas Pharma and AstraZeneca. In addition to roxadustat, FibroGen is advancing pamrevlumab, a monoclonal antibody in late-stage trials for idiopathic pulmonary fibrosis and other fibrotic disorders, as well as early-stage oncology assets aimed at modifying the tumor microenvironment.

FibroGen operates research and development centers across North America, Europe and Asia, leveraging both in-house expertise and external collaborations to drive clinical progress. The company’s global footprint enables efficient regulatory engagement and commercialization in multiple jurisdictions, from China’s rapidly growing pharmaceutical market to established regulatory regions such as the United States and Europe.

Under the leadership of President and Chief Executive Officer Tom Neff, FibroGen emphasizes rigorous science and strategic partnerships to accelerate its pipeline. The management team combines biotech innovation with industry experience, steering the company through clinical milestones and regulatory approvals while seeking to bring transformative medicines to patients worldwide.

FibroGen Q4 2024 Earnings Call Transcript

Key Takeaways

• The sale of FibroGen China to AstraZeneca for approximately $160 million simplifies operations, enables payoff of the Morgan Stanley term loan and extends FibroGen’s cash runway into 2027.
• Phase I monotherapy data for the CD46-targeting ADC FG3246 in heavily pretreated mCRPC patients showed a median rPFS of 8.7 months, 20% overall response rate and 36% ≥50% PSA reductions, prompting a Phase II dose-optimization trial to start by mid-2025.
• Interim results from the FG3246 plus enzalutamide combination study demonstrated a preliminary rPFS of 10.2 months and PSA declines in 71% of evaluable patients, with top-line results expected in H2 2025.
• Subgroup analyses from the Phase III MATTERHORN study indicate roxadustat improves transfusion independence in lower-risk MDS, and an FDA meeting in Q2 2025 will define a ~200-patient trial to pursue U.S. orphan drug designation.
• Operating expenses fell 51% in 2024 versus 2023 and are forecast to decrease another 58% in 2025, aided by headcount reductions and a shift to a virtual work model, further preserving cash for pipeline advancement.

Presentation

[Operator]

Welcome to the FibroGen Fourth Quarter and Full Year 2024 Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To participate, you will need to press Star one one on your telephone. You will then hear a message advising your hand is raised. To withdraw your question, simply press Star one one again. Please be advised that today's conference is being recorded. Now, it's my pleasure to turn the call over to Joanne Greller. The floor is yours.

[Joanne Greller, Head of Investor Relations at LifeSci Advisors]

Thank you, Operator. Good afternoon, everyone. Thank you for joining today to discuss FibroGen's fourth quarter and full year 2024 financial and business results. I'm Joanne Greller from LifeSci Advisors. Joining me on today's call are Thane Wettig, our Chief Executive Officer, and David DeLucia, our Chief Financial Officer. Following the prepared remarks, we will open the call to your questions. I would like to remind you that remarks made on today's call include forward-looking statements about FibroGen. Such statements may include, but are not limited to, our collaborations with AstraZeneca and Astellas, financial guidance, the initiation, enrollment, design, conduct, and results of clinical trials, our regulatory strategies and potential regulatory results, our research and development activities, commercial results and results of operations, risks related to our business, and certain other business matters.

[Joanne Greller, Head of Investor Relations at LifeSci Advisors]

Each forward-looking statement is subject to risks and uncertainties that could cause actual results and events to differ materially from those projected in that statement. A more complete description of these and other material risks can be found in FibroGen's filings with the SEC, including our most recent Form 10-K and Form 10-Q. FibroGen does not undertake any obligation to update publicly any forward-looking statements, whether as a result of new information, future events, or otherwise. The press release reporting the sale of FibroGen China and a webcast of today's conference call can be found on the investor section of FibroGen's website at www.fibrogen.com. With that, I'd like to turn the call over to our CEO, Thane Wettig. Thane.

[Thane Wettig, CEO at FibroGen]

Thank you, Joanne. Good afternoon, everyone, and welcome to our Fourth Quarter and Full Year 2024 Earnings Call. On today's call, I will provide a status update on the transformation of FibroGen, which includes the divestiture of FibroGen China and a laser focus on our U.S. pipeline opportunities, which includes the exciting prospects for FG-3246 and FG-3180, our potential first-in-class antibody-drug conjugate targeting CD46 and our PET imaging agent in metastatic castration-resistant prostate cancer, and for roxadustat in the treatment of anemia due to lower-risk myelodysplastic syndrome. David DeLucia, our CFO, will review the financials, after which we will open the call for your questions. On slide three, I would like to highlight the strategic priorities for our company. First, the announcement of the sale of FibroGen China to AstraZeneca for approximately $160 million.

[Thane Wettig, CEO at FibroGen]

This transaction simplifies our operations, allows for the payoff of our term loan facility with Morgan Stanley Tactical Value, and provides the most efficient pathway to access the company's net cash held in China, extending the company's cash runway into 2027. This is a truly transformative transaction for our company, which we are expecting to close by mid-year. Second, advancing FG-3246 and FG-3180 in mCRPC remains a key priority. In the second quarter of 2024, we shared important data from two phase I studies, highlighting the potential of FG-3246 as both monotherapy and in combination with enzalutamide. I'll provide a more detailed overview of where we are with the program and the upcoming 2025 catalysts in a moment. Third, we believe that roxadustat represents an important potential therapy for patients with anemia associated with lower-risk MDS.

[Thane Wettig, CEO at FibroGen]

We plan to meet with the FDA in the second quarter of 2025 to further explore this opportunity, which we are considering developing on our own or via a potential partnership. We believe the regulatory interaction with the FDA next quarter will provide important clarity on the best path forward, with the aim of realizing additional value for roxadustat and an indication of significant unmet need. Altogether, we are confident that our refined focus, multiple near-term catalysts across both programs, and our existing strong foundation position us well to create value for shareholders now and in the future. I will now provide a brief overview of our FG-3246 and FG-3180 programs in mCRPC. Slide five highlights the high unmet need in late-stage prostate cancer. There are approximately 290,000 men diagnosed with prostate cancer each year in the U.S.

[Thane Wettig, CEO at FibroGen]

Of these, there are 65,000 drug-treatable patients where the cancer has metastasized and become castrate-resistant, resulting in a grim five-year survival rate of approximately 30%. There remains a significant opportunity for new treatments that can extend survival for these men, with a total addressable market of over $5 billion in annual sales. FG-3246 could be this new treatment option. Turning to slide six, we highlight the novelty of our target, a tumor-selective epitope of CD46. CD46 and this specific CD46 epitope have several distinguishing features. CD46 is upregulated during tumorogenesis and helps tumors evade complement-dependent cytotoxicity. The CD46 epitope is highly expressed in mCRPC tissues with lower interpatient variability and higher median expression compared with PSMA, as depicted in the graph on the right-hand portion of the slide.

[Thane Wettig, CEO at FibroGen]

Importantly, the expression of CD46 is upregulated in the progression from localized castration-sensitive prostate cancer to metastatic castration-resistant prostate cancer and further overexpressed following treatment with androgen signaling inhibitors. The CD46 epitope is also overexpressed in colorectal cancer and other solid tumors. I would also like to highlight that CD46 was referenced at this year's ASCO GU meeting as one of the promising non-PSMA targets in advanced prostate cancer due to its high sensitivity, stable expression, and positive correlation to tumor burden. Turning to slide seven, FG-3246 is a potential first-in-class ADC in development for metastatic castration-resistant prostate cancer with a novel targeting antibody, YS5, which binds to the tumor-selective epitope of CD46 and an MMAE payload. MMAE is a validated payload that is approved as part of a number of ADCs and other oncology indications.

[Thane Wettig, CEO at FibroGen]

FG-3246 represents an androgen receptor agnostic approach, clinically differentiating it from other prostate cancer treatments currently in development. A companion PET imaging agent, FG-3180, utilizes the same YS5 targeting antibody as FG-3246 and is also under clinical development. In preclinical studies, the PET imaging agent has demonstrated specific targeting of and uptake by CD46-positive tumor cells. We believe that having a patient selection biomarker would not only allow us to better enrich the patient population in the phase III portion of the clinical development program, it would also enable differentiation of FG-3246 in the prostate cancer treatment paradigm. In addition, FG-3180 could represent an important commercial opportunity as a companion diagnostic to FG-3246, similar to the existing PSMA PET agents. Slide eight recaps the top-line results from the phase I monotherapy study reported in the second quarter of 2024.

[Thane Wettig, CEO at FibroGen]

The completed monotherapy study included a total of 56 metastatic castration-resistant prostate cancer patients who were biomarker unselected and were heavily pretreated, receiving a median of five lines of therapy prior to FG-3246. In the efficacy-available population of 40 patients, we observed a median radiographic progression-free survival of 8.7 months, overall response rate of 20% confirmed by RECIST 1.1, and PSA reductions of greater than 50% in 36% of the patients. Adverse events were consistent with those observed with other MMAE-based ADC therapies. The manuscript describing the phase I monotherapy trial has been submitted, and we anticipate publication soon. On slide nine, we highlight the performance of FG-3246 in its phase I study versus other comparable early-stage studies. As highlighted on the previous slide, the phase I study of FG-3246 demonstrated an rPFS of 8.7 months across a robust sample size of 40 heavily pretreated patients.

[Thane Wettig, CEO at FibroGen]

While we cannot make direct comparisons to these trials due to differences in study design and prior prostate cancer treatments, we are encouraged by the rPFS results, which is a recognized regulatory endpoint in prostate cancer trials. On slide ten, we highlight interim results of the phase Ib portion of the ongoing investigator-sponsored combination study with enzalutamide as reported at ASCO in June of 2024. These interim results included data on 17 biomarker unselected patients, 70% of which were pretreated with at least two prior ARSIs. In addition to establishing the phase II dose of FG-3246, the IST also demonstrated an encouraging preliminary estimate of 10.2 months of radiographic progression-free survival, with PSA declines observed in 71% of available patients.

[Thane Wettig, CEO at FibroGen]

The trial is continuing to enroll and is now set for top-line results in the second half of 2024, which will also include data on CD46 expression on patients treated with FG-3180, our PET biomarker, during the phase II portion of the IST. On slide 11, we depict a comparison of the initial results from the monotherapy trial in heavily pretreated patients and the combination trial for FG-3246 versus the rPFS results from second-line therapies in late-stage trials. Again, while we cannot make direct comparisons to these trials due to differences in study design and previous prostate cancer treatments, we are encouraged that FG-3246 demonstrates what we believe to be competitive rPFS results. Slide 12 highlights the phase II monotherapy dose optimization trial design that is based on our discussion with the FDA.

[Thane Wettig, CEO at FibroGen]

We plan to enroll 75 patients in the post-ARSI pre-chemo setting across three dose levels to determine the optimal dose for phase III based on efficacy, safety, and PK parameters. It is important to note that FG-3180 will be an important part of the study as we seek to demonstrate the correlation between CD46 expression and response to the ADC in this all-comers population. One other important design element is the primary prophylaxis with G-CSF, which is intended to mitigate adverse events associated with neutropenia commonly seen with MMAE payloads. The addition of G-CSF may enable a better tolerated and more consistent treatment with the ADC, thereby extending duration of therapy and potentially enhancing efficacy of the ADC.

[Thane Wettig, CEO at FibroGen]

We are planning an interim analysis in mid-2026, which will include efficacy, safety, PK, and exposure response data, and we intend to share relevant data to all stakeholders as they become available, given the open-label design. Slide 13 highlights the development strategy for FG-3246 and FG-3180, which we believe provides significant optionality in prostate cancer. We have a robust phase II monotherapy trial in the pre-chemo setting in mCRPC to further build upon the compelling efficacy data of 8.7 months of rPFS in 40 heavily pretreated biomarker unselected patients from the phase I monotherapy study. The phase II monotherapy trial is designed to select the optimal dose for phase III based upon the benefit-risk profile from this phase II trial. We believe there are three factors that could drive rPFS even higher than was observed in the phase I monotherapy trial.

[Thane Wettig, CEO at FibroGen]

First, preliminary evidence of an exposure-response relationship, which allows us to focus our phase II study on three of the highest tolerated doses from the phase I dose escalation and expansion study. Second, utilizing primary prophylaxis with G-CSF to combat against neutropenia and allow patients more consistent exposure to the ADC with fewer dose interruptions or adjustments. Third, enrolling healthier patients in earlier lines of therapy versus the median by prior lines of therapy in the phase I trial. In addition, this study will explore the correlation between CD46 expression and response to the ADC, potentially validating FG-3180 as a predictive patient selection biomarker in future studies.

[Thane Wettig, CEO at FibroGen]

We are confident that our development pathway for FG-3246 unlocks sequential or parallel registrational pathways as FG-3246 will be evaluated in multiple lines of therapy, in monotherapy or in combination with an ARSI, and in an all-comers population or patients with high expression of CD46. Slide 14 shows the recent and upcoming catalysts for the FG-3246 program. We have potential value inflection points in the near term with the anticipated initiation of the phase II dose optimization study in mCRPC by mid-2025, and the top-line results from the phase II portion of the combination study with enzalutamide, which are expected in the second half of 2025. To summarize on slide 15, FG-3246 targets a novel epitope on prostate cancer cells with first-in-class potential. It has already demonstrated promising efficacy signals with an acceptable safety profile both in monotherapy and in combination settings.

[Thane Wettig, CEO at FibroGen]

We are excited for the upcoming milestones and look forward to updating you on the program as the studies progress. Slide 17 highlights the unmet need and the potential for roxadustat in patients with anemia associated with lower-risk MDS. There is a lack of effective second-line and beyond treatments, given that the currently available therapies are only effective in approximately 50% of patients. In addition, there are no oral options available or in late-stage development, which could be a meaningful differentiator for roxadustat and potentially translate into a significant commercial opportunity. Moving on to slide 18, in late 2023, subgroup analysis from the phase III Matterhorn study of roxadustat in patients with anemia of lower-risk MDS were presented at the American Society of Hematology annual meeting.

[Thane Wettig, CEO at FibroGen]

In patients with anemia associated with lower-risk MDS who entered the trial with a higher transfusion burden, roxadustat demonstrated a meaningful difference in transfusion independence versus placebo, results that are highly similar to the pivotal trials for two recently approved therapies for anemia associated with lower-risk MDS. On slide 19, we highlight the significant opportunity for roxadustat in low-risk MDS. Based on other lower-risk MDS development programs, we believe the indication would support an orphan drug designation, which would provide seven years of data exclusivity in the U.S. This potential exclusivity, combined with an attractive market opportunity and efficient commercial model, provides a significant economic opportunity for further development of roxadustat in the U.S. We look forward to our FDA meeting planned for the second quarter of 2025, which could pave the way for developing roxadustat for anemia associated with lower-risk MDS, either on our own or through a partnership.

[Thane Wettig, CEO at FibroGen]

With that, I will now turn the call over to Dave to discuss the company's financials. Dave?

[David DeLucia, CFO at FibroGen]

Thank you, Thane. I will first review the FibroGen China transaction details and then provide the company's financial performance for the fourth quarter and full year 2024. Given the announced sale of FibroGen China, our China operations are now reflected as discontinued operations throughout our financials. We will continue to report our China operations and discontinued operations moving forward. On slide 21, we highlight the summary of key financial terms of the transaction. Under the terms of the agreement, FibroGen will receive an enterprise value of $85 million, plus FibroGen net cash held in China at closing, estimated to be approximately $75 million, totaling approximately $160 million.

[David DeLucia, CFO at FibroGen]

The value of FibroGen net cash in China includes FibroGen's portion of Falikang net cash, which is the joint distribution entity owned by FibroGen and AstraZeneca. Importantly, FibroGen will continue to accrue cash generated in China until the closing of the transaction. The transaction is expected to close by mid-2025, pending customary closing conditions, including regulatory review in China. The transaction does not include the Eluminex license agreement, whose rights will continue to be retained by FibroGen going forward. This transaction is truly transformative for FibroGen and allows the company to pay down its senior term loan facility with Morgan Stanley Tactical Value, fully access our cash in China, and extend the company's runway into 2027 to support U.S. development initiatives. Now on to the company's financials for the fourth quarter and full year 2024.

[David DeLucia, CFO at FibroGen]

For the fourth quarter of 2024, total revenue was $3.1 million, compared to $3.6 million for the same period in 2023. For full year 2024, total revenue was $29.6 million, compared to $46.8 million in full year 2023. In the fourth quarter of 2024, we recorded $0.4 million in development revenue, compared to $2.6 million during the fourth quarter of 2023. For full year 2024, we recorded $1.9 million in development revenue, compared to $18.4 million during full year 2023. In the fourth quarter of 2024, we recorded $2.7 million of drug product revenue, compared to $1.1 million during the fourth quarter of 2023. For full year 2024, we recorded $27.7 million of drug product revenue, compared to $18.8 million during full year 2023. Now for full year 2025, we expect total revenue to be between $4 million and $8 million.

[David DeLucia, CFO at FibroGen]

Now moving down the income statement, total operating costs and expenses for the fourth quarter of 2024 were $10.3 million, compared to $66.3 million for the fourth quarter of 2023, a decrease of $56 million, or 84% year over year. Total operating costs and expenses for full year 2024 were $180 million, compared to $369.5 million for full year 2023, a decrease of $189.5 million, or 51% year over year. R&D expenses for the fourth quarter of 2024 were $6.9 million, compared to $48.7 million in the fourth quarter of 2023, a decrease of $41.8 million, or 86% year over year. R&D expenses for full year 2024 were $95.7 million, compared to $266.5 million in full year 2023, a decrease of $170.8 million, or 64% year over year.

[David DeLucia, CFO at FibroGen]

SG&A expenses for the fourth quarter of 2024 were $8.3 million, compared to $16.4 million in the fourth quarter of 2023, a decrease of $8.1 million, or 49% year over year. SG&A expenses for full year 2024 were $49.3 million, compared to $86.5 million in full year 2023, a decrease of $37.2 million, or 43% year over year. During the fourth quarter of 2024, we recorded a net loss from continuing operations of $8.7 million, or $0.08 net loss per basic and diluted share, as compared to a net loss of $62.5 million, or $0.63 per basic and diluted share for the fourth quarter of 2023.

[David DeLucia, CFO at FibroGen]

During full year 2024, we recorded a net loss from continuing operations of $153.1 million, or $1.53 net loss per basic and diluted share, as compared to a net loss of $323 million, or $3.32 per basic and diluted share for the full year 2023. As we have previously stated, we initiated a significant cost reduction plan in the second half of 2024 to become laser-focused on our FG-3246, FG-3180, and roxadustat assets. We have reduced our headcount by approximately 5% in the U.S. We have moved to a virtual work environment after terminating our lease and substantially reduced our operating costs to maximize our cash runway. I am happy to announce that we have completed our restructuring efforts, and the team will continue to work diligently to find operational efficiencies throughout 2025.

[David DeLucia, CFO at FibroGen]

Now for full year 2025, we expect our total operating costs and expenses, including stock-based compensation, to be between $70 million and $80 million, which at the midpoint represents a 58% reduction from full year 2024. Now shifting towards cash. As of December 31st, we reported $51 million in cash, cash equivalents and accounts receivable in the U.S., and $121.1 million in total consolidated cash, cash equivalents and accounts receivable, when including balances in China. Given that the company will continue to accrue cash from its China operations until the close of the sale transaction, we expect the company to be cash flow positive on a consolidated basis in the first quarter of 2025. Upon close of the China transaction, we plan to pay off our senior secured term loan with Morgan Stanley Tactical Value, resulting in a cash outflow of approximately $80 million.

[David DeLucia, CFO at FibroGen]

This includes the $75 million principal balance, accrued and unpaid interest, and an applicable prepayment penalty. Post the payoff of our MSTV term loan, we expect the company to have cash runway into 2027. Thank you, and I will now turn the call back over to Thane.

[David DeLucia, CFO at FibroGen]

Thank you, Dave. To conclude, we believe the sale of FibroGen China is a transformative transaction for FibroGen, and we are excited about the next chapter in the company's story. We are confident that the shift to a lean U.S. organization focused on high-value indications in oncology and oncology-related diseases has the potential to create tremendous value for patients and stakeholders alike.

[David DeLucia, CFO at FibroGen]

With an extended cash runway into 2027, we plan to advance our exciting pipeline in the coming months, initiating the phase II monotherapy study for FG-3246 and FG-3180 in mCRPC, and meeting with the FDA to determine the potential development path for roxadustat in the treatment of anemia associated with lower-risk MDS. These events will set the stage for the remainder of 2025 and beyond, which include top-line results from the phase II portion of the IST for FG-3246 in combination with enzalutamide in the second half of 2025, as well as interim results from the phase II monotherapy study in mid-2026.

[David DeLucia, CFO at FibroGen]

In summary, as a leaner and more focused organization, we will continue to execute on our strategic plan with the aim of achieving evaluation that we believe is more reflective of our first-in-class phase II ready CD46 targeting ADC and our potential phase III ready opportunity in anemia associated with lower-risk MDS, bolstered by our strengthened balance sheet and extended cash runway. We look forward to providing further updates to our stakeholders over the coming months. I would now like to turn the call over to the operator for Q&A.

[Operator]

Thank you so much. As a reminder to ask a question, simply press star one one on your telephone and wait for your name to be announced. To remove yourself, press star one one again. Please stand by while we compile the Q&A roster. Our first question comes from the line of Andy Hsieh with William Blair. Please go ahead.

[Andy Hsieh, Research Analyst at William Blair]

Thank you for taking our questions and congratulations on that transformative deal. Got two quick ones for the ADC franchise. You mentioned about the futility analysis that's coming up in mid-year 2026. I'm just curious if you can characterize the level of stringency for that futility. In other words, maybe from an investor perspective, would the passing of that futility analysis be a very good sign from an efficacy standpoint? Second one, it's a quick one. I'm just curious. It's not very clear on the clinicaltrials.gov website, but curious when you said pre-chemo but post-ARPI, what's the qualification for radioligand? Would a patient who had experience with radioligand be eligible for the trial? On the clinicaltrials.gov website, it only says no radioligand prior, basically within the prior 28 days. I have a follow-up on MDS. Thank you.

[Thane Wettig, CEO at FibroGen]

Thanks, Andy, for the questions. Hope you're doing well. I'll answer the last one first. In terms of maybe setting the stage for this post-ARSI pre-chemo setting, we've learned a lot from some of the KOLs in the past several months about what the sweet spot is for an opportunity like FG-3246. With the ARSIs moving into the castration-sensitive phase, we've also learned that, especially at academic centers, you're seeing less of an ARSI switch regimen. In other words, it is not uncommon for a patient to be treated with one ARSI and then to be switched to another. I think what we're hearing, especially from the academic centers, is that the incremental benefit of switching from one to another is not that great. At the same time, patients are desiring to put off chemotherapy for as long a period of time as possible.

[Thane Wettig, CEO at FibroGen]

We think in this castration-resistant space, post-ARSI, pre-chemo, that's exactly where the trial is designed to recruit patients. We are allowing patients who have been treated with Pluvicto previously, just not within the prior 28 days. If they have been treated with Pluvicto and are no longer responding, then those patients would be eligible to be enrolled in our phase II monotherapy trial. In terms of the futility analysis, we're not going to speak very much about that at this point in time. Just suffice it to say that we're going to ensure that when we look at the data, and this will be data on 12 patients at each one of the dose cohorts, that the therapy is being appropriately tolerated and that we're not seeing any untoward adverse events. We're also going to be looking at efficacy parameters at that point in time.

[Thane Wettig, CEO at FibroGen]

Clearly, if we're seeing efficacy results combined with safety results that are not favorable to the patient, then that would be an important consideration. We are not going to go into detail at this point in time in terms of exactly what that futility analysis is.

[Andy Hsieh, Research Analyst at William Blair]

Okay. That's very helpful. For MDS, I'm curious about the ability to leverage some of the safety database from roxadustat's prior clinical programs. I'm just trying to get a sense of how big that MDS program would have to be. I'm just curious if you can speak to the ability to leverage prior clinical results.

[Thane Wettig, CEO at FibroGen]

Yeah. Thanks for that question. I think we'll learn a lot. I know we'll learn a lot when we speak with the FDA next quarter. Anemia associated with lower-risk myelodysplastic syndrome, that patient population is very different than the patient population of anemia associated with chronic kidney disease. We think that just based upon not only trials that have been done for luspatercept and MDS, but also a recent phase III trial that just started, we think the trial size would be roughly about 200 patients. We're going to get some feedback from the FDA, both in terms of dosing as well as the patient population that we would aim to target, but about 200 patients. The previous safety database for roxadustat and CKD anemia would be informative, but I don't think that instructive relative to this particular patient population.

[Thane Wettig, CEO at FibroGen]

I think it's pretty apparent that based upon previous trials, there will be some important safety follow-up. Not only looking at transfusion independence in the first 28 weeks of the trial, then looking at the same efficacy parameter over 52 weeks, but then some safety follow-up that would extend beyond 52 weeks as well.

[Andy Hsieh, Research Analyst at William Blair]

That's very helpful. Thank you so much for taking our questions.

[Thane Wettig, CEO at FibroGen]

Thank you, Andy.

[Operator]

Thank you. Our next question comes from Matthew Keller with H.C. Wainwright. Please go ahead.

[Matthew Keller, Equity Research Associate at H.C. Wainwright]

Yeah. Good afternoon, everyone. Congrats on the quarter, and thanks for the update. Just two, again, quick questions from us as well. The first one, I was wondering if the recent cost-saving measures that you commented about and really a strong cash balance open up any possibility for possibly some new assets or any possible new indications in the future?

[Thane Wettig, CEO at FibroGen]

Yeah. Matthew, one more time on that. I couldn't catch the last part of that.

[Matthew Keller, Equity Research Associate at H.C. Wainwright]

Oh, yeah. No, it's okay. I was wondering with your strong balance sheet and the cost-saving measures that you're leaving open the possibility or if you're considering any new possible assets to add to the pipeline or possible new indications to consider as well going forward?

[Thane Wettig, CEO at FibroGen]

Yeah. Thanks for the question. Not at this time. We've got a laser focus, as we stated on the call, advancing FG-3246 and FG-3180 into the phase II monotherapy trial, waiting for the results of the FG-3246 trial in combination with enzalutamide, and then seeking FDA feedback on the potential to take roxadustat in the further development in lower-risk MDS anemia. That is where we're going to stay focused. We think if we tried to branch outside of that, it just wouldn't benefit the programs that we have in front of us right now. We need to stay laser focused on those two programs.

[Matthew Keller, Equity Research Associate at H.C. Wainwright]

Yeah. No, no, totally makes sense. Just the second one, if I may, switching gears to Roxa, anemia MDS. I was wondering if you could maybe provide a little more color or highlight maybe what your expectations are if you have a wish list kind of going into your upcoming meeting with the FDA.

[Thane Wettig, CEO at FibroGen]

Yeah. It's a great question. I think our wish list would be that based upon the previous phase II/ phase III trial, the Matterhorn trial, which was conducted where there was a small amount of kind of dose-finding work across 24 patients at three different doses, 1.5 mg per kg, 2 mg per kg, and 2.5 mg per kg of roxadustat, eight patients in each one of those three dose cohorts. 2.5 mg per kg was chosen to be the dose that was then taken into the phase III portion of the trial.

[Thane Wettig, CEO at FibroGen]

There was an algorithm based upon hemoglobin levels that then allowed the clinician to further titrate from there with a maximum of 3.5 milligrams per kilogram. Our preferred response from the FDA is that we've done the appropriate amount of dose-finding work across the more than 100 patients that were treated in the phase II/ phase III trial, and that we would be able to go in right with a 2.5 milligram per kilogram dose and then not have to do additional dose-finding work as part of another phase II/ phase III trial. That's the preferred approach. That's what our request is going to be. In terms of the specific patient population, we'll have a key question that we'll ask the agency on the most appropriate patient population for this particular trial.

[Thane Wettig, CEO at FibroGen]

We desire to do a placebo-controlled trial just because of the fact that by doing a non-inferiority trial versus physician's choice, that would be a much larger and much more expensive trial. The treatment paradigm has changed a bit since we started the Matterhorn trial a few years ago. You've had luspatercept approved, and you've had roxadustat approved. What we're going to find out from the agency is in this kind of ESA refractory patient population who has been tried on one additional therapy, let's call it luspatercept, we would then like them to be randomized to either roxadustat or placebo and not have to go through another therapy before they would be randomized to roxadustat and placebo. It is what we would call the second/third line in lower-risk MDS.

[Thane Wettig, CEO at FibroGen]

That would allow us to then do a direct comparator versus placebo and size the trial, as I said to Andy's question, with about 200 patients or so.

[Matthew Keller, Equity Research Associate at H.C. Wainwright]

Yeah. Totally makes sense. That was very helpful. Thanks again for taking our questions. And congrats again on the quarter.

[Thane Wettig, CEO at FibroGen]

Yeah. Thanks, Matt.

[Operator]

Thank you. This concludes Q&A session. I will turn it back to Thane Wettig for his final remarks.

[Thane Wettig, CEO at FibroGen]

Yeah. Thanks, Carmen. Thanks to everybody for joining us today for our fourth quarter earnings call and your continued interest in FibroGen. Enjoy the rest of your day. Thank you.

[Operator]

Thank you all who participated in today's conference. You may now disconnect.

Participants

Executives

• Thane Wettig, CEO
• David DeLucia, CFO

Analysts

• Matthew Keller, Equity Research Associate at H.C. Wainwright
• Andy Hsieh, Research Analyst at William Blair
• Joanne Greller, Head of Investor Relations at LifeSci Advisors