Kyntra Bio (KYNB) | Biotech Resurgence Conference Replay

Añadido: 2026-05-06

[00:00:00]Coming up next is Kintara Bio, ticker KYNB on the NASDAQ. Presenting today will be Thane Wettig, the CEO. Thane, are you there?

[00:00:16]I am, Craig. How are you?

[00:00:18]Great. Thank you very much for waiting.

[00:00:21]You're showing your presentation. That's great. Let me just get some formalities out of the way and then you can dive right in.

[00:00:28]Read the safe harbor real quick here.

[00:00:31]The segment This segment may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements pertaining to future financial and/or operating results along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements.

[00:00:49]Any statements that are not historical facts should also be considered forward-looking statements. Of course, forward-looking statements involve risks and uncertainties. If you wish to write a question, you can click the bottom of your Zoom window. There is a button Q&A and then a text box will appear. Write your question there. We look forward to hearing from you. Thane, whenever you're ready, go right ahead. Yeah, a good afternoon, everyone, and thanks, Craig, and to the RedChip team for the opportunity.

[00:01:20]For those of you not familiar with Kintara Bio, we rebranded from FibroGen earlier this year to reflect the culmination of a transformation to a company that's now focused on oncology and rare disease. So, as I go through the following Kintara Bio story, I will focus on our core investment thesis of an antibody drug conjugate in metastatic castration-resistant prostate cancer that targets a novel non-PSMA epitope with a companion PET imaging agent that is currently enrolling in a phase two monotherapy trial at multiple sites in the US in the post-ARPI and the pre-chemo setting, and we expect interim results from this phase two study later this year. In addition, we have a phase three ready orally administered small molecule for lower-risk myelodysplastic syndrome, which is a high-value orphan disease with significant unmet need and substantial commercial potential. And this wholly-owned asset has the potential to start a phase three trial later this year. We have $109 million in year-end 2025 cash, no senior secured debt on our balance sheet, a cash runway into 2028, and a current market valuation of approximately $30 million.

[00:02:32]So, with that as a backdrop, I will focus first on our ADC for metastatic castration-resistant prostate cancer.

[00:02:41]And so, those of you who are familiar with prostate cancer know how significant the unmet need is and and the need for treatment options that can extend survival in later-stage disease.

[00:02:51]One out of seven or one out of eight men will be diagnosed with prostate cancer at some point in their life. There are about 65,000 patients that are drug treatable in the US annually, and unfortunately, the five-year survival in mCRPC is approximately 30%, and so there remains a substantial need for therapies that can extend survival in patients who were either ineligible for or who have progressed on ARPI therapy and/or chemotherapy. And also an unmet need for novel MOAs that can treat patients who have progressed on the available treatment options, and in our case, the potential for non-PSMA approaches, as well as predictive tools that can help inform patient selection and also help inform the optimal combination and sequencing of therapies.

[00:03:39]So, as we think about our phase two ADC for prostate cancer, a couple things that that we're really focused on. One is the target, and the second is the program itself, the ADC along with the PET imaging agent. So, when looking at the target, CD46 is a novel tumor-selective target in solid tumors that negatively regulates the complement system and therefore helps tumors evade complement-dependent cytotoxicity.

[00:04:03]It is a multifunctional protein. It's overexpressed in mCRPC. It's also overexpressed in colorectal cancer and other solid tumors and has limited expression in normal tissues, and this is a really important part of the target. This target was discovered by Bin Lu at UCSF, and and Dr. Lu in essence conducted an agnostic phase display set of experiments where he was really trying to identify targets or epitopes on the surface of of cancer cells that were overexpressed there, but were not expressed to any significant magnitude in normal tissues. The only other place that CD46 is expressed to any degree is in placenta and in prostate epithelium, and so that's why he honed in on CD46 as a non-PSMA approach for prostate cancer as well as for other solid tumors. And so, CD46 is overexpressed in mCRPC. It's upregulated as prostate cancer progresses from the localized castration castration-sensitive phase to the castration-resistant phase, and it's also further overexpressed following treatment with androgen receptor inhibitors. It's estimated that 50 to 70% of mCRPC patients have high expression of CD46, and that we also know based upon gene experiments that CD46 is expressed more homogeneously and at higher levels compared to PSMA. And so, we know the target's active based upon results that I'll show you in a moment. And so, targeting CD46, we believe has both therapeutic and diagnostic and diagnostic potential. So, FG-3246 is the therapeutic, and so it has the targeting antibody, which is depicted here in the middle of the slide, YS5, which is a fully human full-length IgG1 monoclonal antibody to to tumor-selective the epitope of CD46.

[00:05:55]In addition to the YS5 antibody, that antibody is is linked to an MMAE payload. MMAE payload is a potent anti-microtubule agent that is validated chemotherapy. In fact, it's approved in a number of other ADCs, most notably Padcev, which is the Astellas and Pfizer bladder cancer therapy, which generated about $2 billion in revenue in 2025. And again, it offers an androgen receptor agnostic and a non-PSMA approach. In addition to the ADC, we also have FG-3180, which is a PET imaging agent, and it uses the same YS5 antibody against CD46, and it's delivered with a zirconium tracer, and it has demonstrated specific uptake in CD46-positive tumors. And so, our development strategy is to combine both the FG-3246 therapeutic and the FG-3180 imaging agent to achieve a clinically differentiated profile in a disease area like prostate cancer, which is highly competitive yet highly dissatisfied at the moment. I'm going to talk about two trials that have been conducted with the ADC, first a phase one monotherapy trial, and then a combination trial that was recently disclosed at ASCO GU. The phase one monotherapy trial had the classic dose escalation and dose expansion design. 33 patients in the escalation cohort with the aim of that escalation cohort to determine the MTD to take into the dose expansion phase.

[00:07:25]That MTD was determined to be 2.7 mg/kg.

[00:07:28]There were then 23 patients treated in the in the dose expansion phase of the phase one trial. The the classic endpoints across both cohorts again, in addition to safety, were the standard efficacy measures of PSA50, ORR, duration of response, and rPFS as well.

[00:07:47]This was a heavily pretreated patient population, meeting a five prior lines of therapy. Everyone had received prior systemic therapies, androgen deprivation therapy, as well as androgen receptor inhibitors. There were about one in four patients who had previously received taxane therapy, but those were only allowed in the castration-sensitive phase, so no patients had received taxane therapy in the castration-resistant phase. In terms of results, we were very encouraged with the 8.7 months of median rPFS across the 40 patients in the efficacy evaluable population. These were patients who were at 1.2 mg/kg or and above. Again, 2.7 mg/kg was the expansion cohort dose. A PSA50 decline of 36% and ORR five out of 25 patients or 20% of patients demonstrated ORR, and the duration of response was 7.5 months.

[00:08:42]So again, we were highly encouraged by these results.

[00:08:46]When we take a look at these results relative to other programs in a similar stage of development, and again, while we can't make direct comparisons due to trial differences and study design and things of that nature, again, this gave us some encouraging signs that give us the conviction to take this ADC into the phase two portion of the program. There was a lot of excitement around JANX-007, especially given their PSA50 and PSA90 results and the T-cell engager approach.

[00:09:19]However, when JANX provided data at least on 16 patients from an rPFS perspective, it it didn't hold up to the the PSA50 results that we're seeing, and that's why we continue to be, given our non-PSMA approach, continue to be really focused on, you know, what is a registration endpoint of rPFS.

[00:09:39]We believe PSA50 is instructive, but we're going to continue to pay a lot more attention to rPFS as the program progresses. From a safety profile, the safety profile was consistent with other MMAE-based ADCs, where you see about one in three patients experiencing grade three or above neutropenia neutrophil count decrease white blood cell count decrease. Um you can see also about a third of patients had peripheral neuropathy. Only one of those patients had grade three or above peripheral neuropathy. But again, this is an adverse event that we're paying close attention to in the phase three in the phase two portion of the program given the fact that even grade two peripheral neuropathy can be problematic for a number of patients. So we'll talk about a way that we're we are mitigating the neutropenia as part of the phase two study design.

[00:10:28]Uh now turning to the phase 1b2 trial that was with FG 3246 in combination with enzalutamide. These were results that were disclosed at ASCO GU a few weeks ago. Again, the primary endpoint for the phase 1b portion was to determine the MTD to take into phase two and then the classic secondary endpoints PSA 50 ORR RPFS etc. I think it's important to note that the MTD for this particular trial was 2.1 mg per kilogram of our of FG 3246 juxtaposed to the 2.7 mg per kilogram from the phase one monotherapy trial. This trial also had 160 mg per day of enzalutamide. In terms of the results across the entire cohort seven months of median RPFS a 22% PSA 50 response. I think it's really important to note that a majority of patients had progressed on two or more prior ARPIs.

[00:11:25]So you think about the design of this trial 60 plus percent of patients had progressed on let's say an abiraterone and an enzalutamide and then they were retried with enzalutamide or vice versa enzalutamide followed by abiraterone and then put on enzalutamide again. And so it three or more ARPIs if you include enzalutamide in combination with our ADC. When Dr. Rahul Agrawal who was the PI of the phase one monotherapy trial and also ran this IST UCSF when he looked at patients who had progressed on only one prior ARPI he was very encouraged with the 10.1 months of median RPFS in the PSA 50 response of 40%. So again encouraging anti-tumor activity in patients with mCRPC consistent with the phase one monotherapy trial. And when we think about how this compares to other similar stage trials when we think about the 8.7 months of RPFS seen in the monotherapy trial and the 10.1 months in patients who had progressed on only one prior ARPI in the combination with enzalutamide trial we again think it compares very favorably. And the benchmark that we have have set for the phase two monotherapy trial is the 9.3 months of RPFS that Pluvicto demonstrated in the post one ARPI pre-chemo setting that led to the recent indication in that same setting for Pluvicto. So again we think that we need to achieve about a 10 month RPFS in the phase two study to [clears throat] get have a high degree of conviction to take it into the phase three portion of the program.

[00:13:06]In terms of the adverse event profile from Dr. Agrawal's IST again focusing on the neutropenia which as as you may recall from a few slides ago about a third of patients had grade three or above neutropenia in the phase one monotherapy trial.

[00:13:21]You can see that about 5% of patients had grade three or above neutropenia in his IST.

[00:13:28]>> [clears throat] >> And the reason is is because Dr. Agrawal utilized prophylactic GCSF to mitigate [clears throat] the neutropenia the grade three and above neutropenia that was so common in the phase one monotherapy trial and in fact led to a number of patients having to be dose interrupted or downward titrated in the phase one monotherapy trial. And so this is an important design element that we're take that we've taken forward into the phase two monotherapy trial.

[00:13:54]Um the last point from this [clears throat] particular IST and something that we were really encouraged by is that the higher tumor uptake of FG 3180 was associated with PSA 50 response which underscores the potential for FG 3180 as a companion pet biomarker for patients patient selection. On the right hand side of the slide is an example of a PET image from the IST that was captured after treatment with FG 3180. So a patient would have entered the trial.

[00:14:25]They would have given FG 3180. They would have come back six days later for the PET scan and then immediately started into the IST.

[00:14:33]And so we think this that image right there is a perfect example of how multiple tumors and lesions that express CD46.

[00:14:42]The table on the left shows the correlation between tumor uptake of FG 3180 and PSA 50 response. So patients with a higher average maximum standardized uptake value which is SUV of a target lesion when normalized to the SUV of the blood pool demonstrated a trend to greater response to FG 3246 as measured by PSA 50 versus those with a lower SUV with a nominal P value that just missed being statistically significant despite a small number of patients. And so we believe this data demonstrates for the first time an association between FG 3246 expression and response to FG 3246.

[00:15:23]And we we're going to be further evaluating this as as an important component of the phase two ongoing phase two trial. Now turning to the ongoing phase two trial monotherapy trial we expect interim results from this trial in the second half of this year.

[00:15:39]It is actively enrolling in the United States.

[00:15:43]There will be 75 patients treated 25 at each one of the three different dose cohorts 1.8 2.4 and 2.7 mg per kilogram.

[00:15:51]And again, we will use primary prophylaxis across all 75 patients. The interim analysis will look at a futility analysis and other available efficacy safety PK and exposure response data. The futility analysis will be a composite response of PSA 50 and ORR.

[00:16:11]We've set a hurdle that is similar to the composite response from the phase one monotherapy trial because again, we're most interested given the non-PSMA approach not looking at PSA 50 but we're most interested in looking at more fully mature RPFS data and the correlation between response to the ADC and the expression of CD46 as measured by the PET imaging agent which will occur in the in the 2027 time frame.

[00:16:38]And so as we think about that that 10 month RPFS that we're shooting for relative to the 8.7 months of RPFS that was demonstrated in the phase one monotherapy trial there there are some important design elements that we think will give us the potential to achieve an RPFS that we think would be competitive if we were to then take it into the phase three portion of the program. Use of the three highest doses of the ADC 1.8 2.4 and 2.7 mg per kilogram. And again, this is based upon the exposure response that was established during the phase one escalation and expansion trial. The use of primary prophylaxis prophylaxis with GCSF which I spoke about to mitigate the grade three and above neutropenia which again led to dose interruption and dose reduction in the phase one monotherapy trial. We're really trying to keep patients on the dose in the early portion of the trial without interruption or dose reduction so that they can get the maximum cytotoxic effect early in the course of of therapy. And then moving up line to patients who have have not been treated with a median of five prior lines of therapy but who have progressed on one prior ARPI. So think about it either in the first line or second line mCRPC setting. So we think that these design elements could very well allow us to build upon the encouraging 8.7 months worth of RPFS that was demonstrated in the phase one monotherapy trial. So again, we're really focused on a couple of things. First the interim analysis in the second half of this year and then the more fully mature RPFS data which given the open label design of the trial we'll be able to look at as patients are are randomized and as patients and and data is accrued.

[00:18:20]So in summary, we believe that our phase two program of FG 3246 and FG 3180 presents a unique opportunity in mCRPC.

[00:18:30]We believe that the novel mechanism and the novel target of CD46 presents a unique opportunity for for us.

[00:18:38]Encouraging results in two previous studies that show not only is CD46 an active target but the the ADC does a very nice job at at taking advantage of that target and and generating encouraging RPFS results. The safety profile which is entirely consistent with the safety profile demonstrated with other MMAE based ADC therapies. Substantial opportunity given the high unmet need in metastatic castration resistant prostate cancer. And again, the importance of the CD46 biomarker diagnostic or FG 3180 as a potential PET biomarker imaging agent designed for screening patient selection and potential enrichment. If we are able to show a correlation in the phase two monotherapy trial between the expression levels of CD46 and response to the ADC that would allow us then in the phase three portion of the program to enrich that population for those patients who are higher expressers of CD46 with the knowledge that they had a better response to the ADC in the phase two trial.

[00:19:44]So that is the phase two asset the ADC targeting CD46 for for prostate cancer.

[00:19:52]And now I'll shift to roxadustat.

[00:19:53]Roxadustat is the asset that FibroGen formerly FibroGen or Kintara Bio is most well known for. It's an oral hypoxia-inducible factor pH inhibitor.

[00:20:03]The science behind roxadustat was the basis for the 2019 Nobel Prize in physiology or medicine based upon the discovery of how cells sense and adapt and adapt to oxygen availability.

[00:20:17]We it clearly increases hemoglobin by mimicking the body's natural response to low oxygen, and it is approved in more than 40 countries for the treatment of anemia associated with chronic kidney disease. In fact, well over a million patients have been treated globally with roxadustat since it was approved first in 2019.

[00:20:37]Our focus going forward now is on roxadustat for anemia associated with lower-risk myelodysplastic syndrome. So, there was a previous phase three trial conducted by Kintara Bio in collaboration with Astellas and AstraZeneca um that looked at roxadustat for anemia associated with lower-risk myelodysplastic syndrome. In that phase three trial, we showed a numerical advantage of transfusion independence but missed statistical significance because of an abnormally high placebo response rate that was driven by the enrollment of a large number of patients who had a low transfusion burden at baseline, one or two units over an 8-week period of time. Many of these patients will spontaneously become transfusion independent just because of the the low transfusion requirement. Uh when we did a post hoc subset analysis of patients who had a high transfusion burden at baseline, we saw a very meaningful response with roxadustat relative to placebo both as measured by 8-week RBC transfusion independence within 28 weeks and 52 weeks. And these are results that that were very similar to the results from the registration programs for the two most recently approved products in this category, luspatercept and imetelstat. And so, this is what gave us the conviction to approach the FDA in the middle of last year through a type C meeting process where we reached alignment on essentially all the elements of the phase three design, which I'll speak to in a moment.

[00:22:01]So, when we think about roxadustat and how it would then compare relative to or compete with and against the the currently available therapies, typically clinicians are making their choice based upon what's called the ring sideroblast status, RS positive or RS negative. RS negative is the is the majority of patients who have lower-risk MDS. RS positive is a smaller proportion. Luspatercept, which is Reblozyl, which is a BMS product, which generated $2.3 billion in revenue in 2025, about $1.8 billion of that revenue in the US and the majority of that in lower-risk MDS. They also have a beta thalassemia indication, but they've really um positioned themselves in first-line therapy, especially in the RS positive population where they have shown a meaningful benefit head-to-head against the ESAs. They really haven't demonstrated much evidence of of efficacy in the RS negative population, but they still get utilization there.

[00:22:57]We think [clears throat] roxadustat, if we were able to mimic the subset results from the previous phase three trial and enrolled in the right patient population, we think we can compete very effectively in the second-line setting and in the third-line setting, and it doesn't take a lot of share in the second or third-line setting to to generate a meaningful amount of revenue.

[00:23:19]So, so this is our whole focus going forward into the the phase three design.

[00:23:23]Again, we've reached alignment with the FDA on the patient population, patients requiring four or more RBC units in two consecutive 8-week periods who are refractory to, intolerant to, or ineligible for prior ESAs.

[00:23:36]Efficacy either 8 or 16-week RBC transfusion independence. Um the safety, um we're going to manage uh potential thrombotic risk through eligibility, dose modification, and discontinuation criteria. Uh and then we've aligned with the FDA on both the starting dose and the maximum dose along with the with the titrate titration algorithm. We submitted the final protocol in December of 2025, and that we are in the process of of responding back to FDA feedback, and we'll have that feedback back to the FDA here in short order. So, in summary, we think there's a substantial opportunity for roxadustat as a phase three ready asset in anemia associated with lower-risk myelodysplastic syndrome. Um we think that the differentiated profile with a potentially superior tolerability and convenient dosing and administration, so roxadustat would be would be administered three times a week orally at home by the patient. Um the currently available therapies require either in-office IV infusion or sub-Q injection every three or four weeks. And for this particular patient population, 75 years of age um with with uh severe anemia and a poor quality of life, many of these patients have trouble making it into the clinic that often for the administration of the currently available therapies. We also received orphan drug designation for anemia or for lower-risk myelodysplastic syndrome, which will give us at least 7 years of regulatory exclusivity in the US. And again, with the worldwide market that's expected to exceed $4 billion in 5 years, we believe we've got an opportunity for at least a $500 million in peak sales. In fact, a recent opportunity assessment that was conducted with ZS Associates uh give us a lot of confidence that we can we can reach a peak revenue of close to a billion dollars if in fact we hit the the product profile based upon the subset analysis from the previous phase three trial.

[00:25:30]And so, in summary, Kintara Bio a phase two ongoing metastatic castration-resistant prostate cancer trial with our ADC and a phase three ready asset with roxadustat. And so, we're incredibly excited about the the potential opportunity for the firm, especially on the backdrop of a current $30 million market capitalization. So, with that I'll open it up to questions.

[00:25:53]Thank you very much. Let's get started.

[00:25:55]With cash expected to fund operations into, as you said, 2028, how are you prioritary Sorry, prioritizing capital allocation, and what steps are you taking to ensure that shareholder value is protected even as the company advances its pipeline?

[00:26:14]Yeah, so I I I tend to think about shareholder value not being protected but being but being catapulted. Um and we're going to you know, we're going to we've got the the phase two ADC is fully funded, and their cash runway allows us to get to the answer for the phase two monotherapy trial.

[00:26:30]We are doing and funding all of the phase three enabling activity for roxadustat for lower-risk myelodysplastic syndrome.

[00:26:37]Right [clears throat] now, our cash runway does not contemplate us being able to fund the phase three trial for roxadustat. So, we at the moment are having conversations with both strategics for the typical kind of business development licensing approach as well as project-based financing firms uh with the aim that in the next 60 to 90 days, we have made the determination on the path forward for roxadustat either on our own or through a strategic. Our strong conviction and strong desire is to do it on our own, so we will need to raise capital in order to do that. Uh but again, with a with a phase three ready wholly-owned asset with close to a billion-dollar peak revenue potential, um we hope that we'll be able to to be able to raise the capital in order to do so.

[00:27:21]Roxadustat, it represents a different kind of opportunity compared to FG-3246.

[00:27:27]How do you think about its role within Kintara's overall strategy, and what would success in LR-MDS anemia look like from both a clinical and commercial perspective?

[00:27:38]Yeah, so I'll take the second question first. So, you know, success would be that you know, we see results that are similar to the results that were generated by the other two most recently approved therapies in the category. So, transfusion independence for eight straight weeks of let's say 35 to 40% against a placebo response rate of you know, 10% or so.

[00:27:59]And when we tested that particular profile in the most recent opportunity assessment that ZS ran, uh we see peak revenue close to a billion dollars. Um and so, you that's how we think about the commercial opportunity and then the clinical based upon the clinical benefit of transfusion independence. How we think about that relative to the the ADC is you know, the ADC, if we think about evaluation, has a larger um value inflection opportunity just because of the size of the prostate cancer marketplace. That being said, it is a phase two asset with a with a different probability of success than a phase three ready asset. And so, we get the question we get often is um which one's more important? And it really depends upon you know, which lens you're using. If you're an oncology-focused investor, you're probably going to be more excited about the ADC. If you're thinking more about probability of success, then you're probably going to be thinking of and and and like rare disease, you're going to be thinking more about roxadustat. And then we we hear we hear from investors that say, we actually don't like a company if they've only got one shot on goal. We like it if you've got you're moving both assets forward. And so, that's how we tend to think about it is just the the optionality that we have with two really exciting assets, a phase an ongoing phase two asset and a phase three ready asset as well.

[00:29:15]Perfect timing, Thane. We are right at the bottom of the hour. Thank you very much.

[00:29:22]Appreciate it, Craig. Thanks, guys. See you soon. Take care.

[00:29:26]>> For more information on Kintara Bio, reach us at 1-800-RedChip or email us at [email protected].