Astria Therapeutics at Jefferies Conference: Strategic Pipeline Insights

Astria Therapeutics (NASDAQ:ATXS) presented its strategic pipeline at the Jefferies Global Healthcare Conference 2025 on Wednesday, 04 June 2025. The company discussed its promising developments in treatments for hereditary angioedema (HAE) and atopic dermatitis, highlighting both opportunities and challenges. While Astria aims for leadership in the HAE market, it faces competition and regulatory hurdles.

Key Takeaways

• Astria is advancing Nevenibart for HAE with a Phase III trial, targeting a significant market share by 2030.
• STAR310 aims to address unmet needs in atopic dermatitis, focusing on patients unresponsive to existing treatments.
• The company holds a strong cash position of just under $300 million, expected to last until mid-2027.
• Astria plans independent commercialization in the US for Nevenibart, with potential partnerships in Europe and Japan.
• The HAE market is projected to reach $5.4 billion by 2030, driven by early diagnosis and geographic expansion.

Financial Results

• Astria ended the last quarter with just under $300 million in cash.
• The current cash position is expected to sustain operations and key milestones until mid-2027.
• The HAE market is anticipated to grow to $5.4 billion by 2030, with Astria positioning Nevenibart as a leading product.

Operational Updates

• Nevenibart (HAE):

- Phase III trial began in February 2025, enrolling patients in the US and Canada, with plans to expand globally.

- Top-line data from the trial is expected in early 2027.

- Long-term safety and efficacy data from the Alpha Solar extension trial are anticipated mid-year.

• STAR310 (Atopic Dermatitis):

- Phase 1a trial data on safety and tolerability is expected within the next 12 to 18 months.

- The drug aims to differentiate itself by minimizing adverse effects associated with current treatments.

Future Outlook

• Nevenibart (HAE):

- Astria aims for Nevenibart to become the market-leading HAE treatment.

- Plans for independent commercialization in the US are underway, with potential partnerships in Europe and Japan.

• STAR310 (Atopic Dermatitis):

- Targets the 40% of patients not adequately responding to current treatments like Dupixent.

- Focuses on the OX40 pathway to offer a novel treatment approach.

Q&A Highlights

• HAE Market and Competition:

- Nevenibart’s efficacy is measured by attack rate reduction compared to placebo, aiming to attract both switch and newly diagnosed patients.

• Phase III Trial Design:

- The trial includes three Nevenibart arms and a placebo arm, each compared individually to demonstrate statistical significance.

• STAR310 Differentiation:

- Designed to avoid common adverse effects of OX40 antagonists, offering a safer profile for higher dosing.

In conclusion, Astria Therapeutics is strategically positioned to advance its pipeline, with promising prospects in the HAE and atopic dermatitis markets. For a detailed discussion, refer to the full transcript below.

Full transcript - Jefferies Global Healthcare Conference 2025:

Anqi Yu, Team member of biotech research team, Jefferies: Alright. Good afternoon, everyone. My name is Anqi Yu. I’m a team member of biotech research team here at Jefferies. Today, we’re happy to have Astraya and Joe with us to give us a fireside chat about Astraya.

Welcome, Thank you. Glad to be here. All right. For those who are not quite familiar, could you please give us an overview for Astraea?

Joe, Astraea: Absolutely. So at Astraea we are focused on bringing first choice products to people living with allergic and immunologic diseases. And what we mean by first choice is that patients and physicians will choose our products first because of their competitive efficacy, good safety tolerability profile and importantly very low treatment burden. Our lead program at Astraea is Nevenibart which is a monoclonal antibody inhibitor of an enzyme called plasmacallicrin. Plasmocalikrin is a clinically and commercially validated target for the preventative treatment of hereditary angioedema and we’re currently in Phase III with Nivenabart.

What excites us about Nivenabart is the efficacy profile that we’ve produced to date in our proof of concept trial in patients, which showed a greater than ninety percent attack rate reduction in patients when dosed once or twice during six months. And so that profile, that data supports our potential for dosing Nivenabart as infrequently as every six months. And that’s what we’ve taken forward into our phase three clinical trial, both a Q3 month dosing regimen and a Q6 month. We also have a second program in the pipeline, as you know, STAR310, which is a monoclonal antibody antagonist of the OX40 receptor, a mechanism that’s important in T cell biology. And we expect to have phase one healthy volunteer data later this year.

Unidentified speaker: Fantastic. And maybe for the moment, let’s focus on HAE. This is a very active space. So the from the clinical learnings, you have shown more than 90% of attack rate reduction and also attack free rate is pretty compelling. Can you share with us so far what do you hear from physician communities?

How do they evaluate across two different efficacy endpoints? And what how to define best in class given there are so many competing programs in this space?

Joe, Astraea: Yeah. Great questions. So from an efficacy endpoint, certainly what physicians look at as they look at programs, of course, is the endpoint that is used in all pivotal phase three studies. So time normalized attack rate reduction compared to placebo. And that’s really what physicians, we believe, will be looking at as they think about Nivenabart versus other programs.

And of course there are other factors that come into play as well. Certainly attack free is very important and that’s something that we are monitoring and is a key secondary endpoint in our pivotal phase three because of course patients want to remain attack free. And we believe Nivenabart has a good chance of producing a highly competitive attack free rate in this phase three as well.

Unidentified speaker: Fantastic. Also the data update from the long term open label trial in HAE of alpha solar is expected in mid of this year. Could share with us how should we set expectation and what kind of read through to the future pivotal trial readout?

Joe, Astraea: Yeah. So Alpha Solar is the long term extension trial from our phase 1b trial. And so patients, all of the patients from our phase 1B2 trial chose to enroll into Alpha Solar so we took that as a good sign. What we expect to show is long term safety and efficacy data. And we would expect to see similar to what we saw in that phase 1B2 trial in terms of attack rate reduction and the safety tolerability profile.

And we’re on track to share that data mid this year, so coming up. Would

Unidentified speaker: you be able to help us to narrow down, like, how to define mid year of this year? Because we are already in June.

Joe, Astraea: Yes. So certainly, we’re in June and that’s right around midyear. So we haven’t disclosed publicly where we’ll be releasing it. But, yes, we are on track for midyear.

Unidentified speaker: Okay. Fantastic.

Joe, Astraea: And you asked about read through to the phase three. Of course, we expect the data will be in line with what we’ve seen in the phase 1btwo trial for Novenebart. And the design of our phase three, of course, was designed from the results that we’ve achieved to date. We would expect to our premise has been for nivenabart that we believe we could support. And they need a 90% attack rate reduction in that pivotal phase three.

And we’ll certainly be looking to see what we see on attack free.

Unidentified speaker: And do we expect some difference from three months versus a six months cohort?

Joe, Astraea: We don’t. So we’ve designed the doses going into our pivotal phase three based on the data we’ve generated to date. And we believe the efficacy range will both, for both cohorts will be in that eighty to ninety percent attack rate reduction range.

Unidentified speaker: And also does this data have some implication in terms of the timing of regulatory filing post the phase three readout in another way like shed light on the follow-up time of pivotal trial?

Joe, Astraea: Are you asking about the Alpha Solar Yes. Alpha Solar, that data certainly will be part of the package that ultimately goes with our filing to the FDA and other regulatory authorities, but is not the Alpha Solar data is not critical path to that.

Unidentified speaker: Okay. Great. Thanks for clarification. And also talking about HE market opportunity, you be able to help us to put a number on that? And how does this market evolve for this couple of years?

Joe, Astraea: Yeah. So the HAE market is large as far as rare diseases go. And it’s been growing. We expect the market to grow to about $5,400,000,000 by 02/1930. And we expect that growth to be largely driven by patients being diagnosed earlier, geographic expansion of drugs, as well as more patients going on to prophylactic therapy.

So then when we get into how the market is shaping up in terms of across the different therapeutics, you know, I think there are oral therapeutics. There are the long acting injectables like Novenebart and the other injectables. I think Novenebart and the profile that we believe we can support in this phase three has the potential to allow Novenebart to become the market leading product. And certainly the market research we’ve done with physicians has suggested that navenibart will take a large market share of not only switch patients, so patients who are already on a prophylactic and switching off to go on to a drug like navenibart, but also newly diagnosed patients coming on to therapy for the first time.

Unidentified speaker: Fantastic. For the investors who not super familiar with your Phase III trial, could you elaborate on the trial design? Initially you plan to start a three months first and then six months cohort. Now actually going to start simultaneously. Can you tell us like what’s the advantage does it offer?

Joe, Astraea: Yeah. So we were able to include both the Q3 month and Q6 month dosing regimens in a single pivotal phase three trial. And that trial was initiated in February of this year. And I think the advantages that we see for bringing both of these dosing regimens forward is it gives patients and physicians more options. And I think create the opportunity to capture the most market share by being able to offer both at time of launch.

And certainly our original premise before we were able to get in front of regulators around the world was that we might run these in two separate phase three trials. But of course, there’s a cost savings to be able to do them in a single trial as well, not only operational costs for running the trials, but also as we think about other costs that we can have synergistic effects of running the two simultaneously.

Unidentified speaker: I see. And also, do you see six months and three months dosing regimen might target a slightly different patient population? Or in another word, how do you think a physician’s going to balance the efficacy outcome to the treatment convenience if actually two different dosing regimen actually turns out to have a little bit of different treatment outcome?

Joe, Astraea: Yeah, so it’s interesting. What we there is great interest in both the Q3 month and the Q6 month. And when we’ve done market research with patients, we’ve seen almost equal interest in the two different dosing regimens which at first we found interesting because we originally anticipated most may want the less frequent dosing, every six months. But what we’ve come to learn is that many patients and many physicians might want to start Nivenabart every three months if that drug works for them, if Nivenabart provides the level of attack rate reduction that meets the needs of the patient, that then they might want to try the longer dosing regimen every six months. And so we anticipate that we might have most patients starting on a Q3 month and transitioning to a Q6 month.

Unidentified speaker: Great. And how is the trial designed to show status significance for the endpoints?

Joe, Astraea: Yeah. So we have three treatment arms, three Neventybart arms and a placebo arm. And each of those treatment arms will be compared individually to placebo.

Unidentified speaker: And also the Phase III trial started enrolling in the first quarter of this year. Can you provide us some color on the enrollment pace?

Joe, Astraea: Yeah, so I will say, yes, so the trial started in February. We are now enrolling in both The US and Canada and opening sites in the rest of the world presently. I will say we are quite pleased with the enthusiasm that we’re seeing from patients and physicians. So everything remains on track at this point for early twenty seven for top line data.

Unidentified speaker: Great. And also, could you actually share some more details on the timing of regulatory filing on top of phase three success?

Joe, Astraea: Yeah, we haven’t worked out those details or shared those publicly yet but of course we’re going to be working very efficiently, as efficiently as possible to make sure that we can have this filed as soon after data as possible.

Unidentified speaker: And there will be around 10 adolescent patients in the Phase III trial. What’s your regulatory strategy for this patient and also patient population also pediatric HAE given there is no placebo arm for adolescents?

Joe, Astraea: Yeah, so that design, so the adolescents, we will enroll 10 adolescents. And you’re right, they will not have a placebo control and they will not be part of the primary analysis for the phase three. And that was based on regulatory feedback that in order to have the adolescents in the label, we just needed to include them in the pivotal phase three. But did not need to include a placebo arm for the patient, for the adolescents.

Unidentified speaker: I see. Could you also discuss the market research you’ve done on the potential market penetration of three versus six months dosing? And also how your drug makes a difference across the HAE landscape?

Joe, Astraea: Yeah, so we think based on the profile for Nivenabart, you know, it’s a trusted mechanism. It’s trusted modality. So the mechanism and modality are the same as the current market leading product, Tekxiro. And so we think that gives it a strong advantage as we think about going into the market and penetrating that market. In addition to that, the data to date has shown a very strong efficacy profile, very good safety tolerability.

And to date our injections have not been associated with pain, which we think is something that’s important certainly for patients. And so we think our with that profile that we believe we can support with the phase three, we think that we have the opportunity to become the market leading product and take share not only from products like the current market leader, TEKHZYRO, but also from the other categories as well.

Unidentified speaker: And how physicians are receptive to make a switch to Nivenibart from TEKHZYRO, Takeda’s drug for HAE if the both the three months and the six months cohort showing similar treatment outcome in pivotal trial from your early phase trial?

Joe, Astraea: Yeah. I think with data similar to what we produced in the proof of concept trial in patients, I think it’s an easy conversation for a physician to have with a patient that’s already that’s on TAXIRO. So same mechanism, same modality, but just much less frequent dosing. And we also hope a better experience for patients in terms of no injection site pain. That’s been a target as well for us.

And we think that’s an easy conversation and hence why we are bullish about our potential to really capture that market leading position with Nivenabart should we be able to support that profile.

Unidentified speaker: Still early days, but would you like to discuss the commercial plan if you decided to do it by yourself? And also, are you open to partnership for future commercial path?

Joe, Astraea: Yeah. What we’ve talked about is that we intend to commercialize Nivenabart in The US on our own. But we are considering what to do outside of The US. And that could involve partnerships in Europe. It could involve partnerships in Japan.

And so that strategy is being discussed now.

Unidentified speaker: Great. And switching to atopic dermatitis for the OX40 antagonist program. This is also competitive space with many approved drugs and the pipeline candidates. What is the unmet needs you see there for your program?

Joe, Astraea: Yeah. I think atopic dermatitis is a very large market but it’s growing and growing massively. And I think there’s also a growing appreciation in use of biologics and mechanism based biologics. Right now, DUPIXENT is clearly performing incredibly well atopic dermatitis and serving patients well. However, there are about 40 of patients that don’t respond adequately to Dupixent and drugs with that mechanism that targets selectively a Th2 population of T cell mediated disease.

We see that remaining, that forty percent of patients as a good initial target population for a drug like STAR310 that targets the OX40 pathway. And OX40, one of the exciting pieces about OX40 is it has a broad effect on broad T cell populations and so we believe can reach patients that may be inadequately controlled by drugs that target just one of these T cell populations. I think the other interesting piece about the OX40 mechanism is the potential for disease modification. And we’re really excited to test that potential.

Unidentified speaker: What is the key takeaway on both safety and efficacy front OX40 targeting strategy from a couple of late stage programs?

Joe, Astraea: Yeah. So you’re referring to so both Amgen has a roclatilumab, their roclatilumab program and Sanofi has their amlatilumab program. So Amgen has released results from three phase threes in atopic dermatitis with roclotilimab. Interestingly, their phase 2B which was a well controlled atopic dermatitis trial with their three hundred milligram every two week dose of roclotilimab. They saw efficacy on par with dupilumab, DUPIXENT in AD patients.

However when they went into phase three they took that dose and cut it in half. So they went to three hundred milligrams every four weeks which led to lower efficacy. They still hit their endpoints and showed strong effects in atopic dermatitis but not as strong as they saw in phase 2B. And I think that caused a lot of interest and concern from people in this space. I will say roclotilimab has been, was engineered for high ADCC, so high killing of T cells.

That was in the engineering of that antibody because it was believed you had to kill the T cell to have the efficacy. And I think what Amgen is trying to do is thread a needle. So balance the AE profile, the fever chills associated with ADCC, but maintain the efficacy. And that was when we started our STARR310 program knowing the ADCC activity of roclotilimab, that was built into the design of our molecule to have a molecule that hit the receptor as hard, as potent as roclotilimab but without that T cell killing. So to avoid the fever chills AE profile so that we could open up the therapeutic window, really drive the doses to drive the efficacy.

And so that’s where we hope one of the areas we hope that STARR310 will differentiate.

Unidentified speaker: Fantastic. And in the upcoming initial results from your Phase III in healthy volunteer trial, What investors should be looking for? What kind of data sets do we anticipate? For example, the Th2 biomarkers would be included in this update?

Joe, Astraea: So what we’ll be looking at, there are two key sets of data we’ll be focused on. One is the AE profile, the tolerability profile. We know from the roclotilimab program in healthy subjects they saw a greater than fifty percent of subjects having fever associated with dosing. We’re clearly going to be looking for a differentiation there. We also have engineered STAR-three ten for a very long half life, so we’ll be looking at PK because the idea with STAR-three ten is not only can we drive efficacy to greater levels, but we also can have very infrequent dosing.

And so we’ll be looking at that. We will be testing exploratory biomarkers. Of course, these are healthy subjects so they don’t have activated T cells and so we’ll be doing some exploratory biomarkers.

Unidentified speaker: Great. And maybe lastly, could you help us to understand the balance sheet and lay out the most meaningful catalyst over the next twelve to eighteen months?

Joe, Astraea: Sure. Yeah, so we ended the last quarter with just under $300,000,000 in cash and that cash position will take us into mid twenty twenty seven. So importantly through major catalyst for nivenabart program, our lead program, so beyond our top line phase three data. So over the next twelve to eighteen months we’ll have the data from Alpha Solar, the long term extension with nivenabart, so patients who have been on drug for twelve to eighteen months and we’re looking at both the Q3 month and the Q6 month regimen there. We’ll have our Phase 1a healthy subject data for the STAR-three 10 program and again we believe that data will read on the potential for differentiation of that program.

And then beyond that, we’ll be looking straight to the phase three data.

Unidentified speaker: Fantastic. Thanks for a very insightful discussion. And thanks, everyone, for joining

Joe, Astraea: us. Thank you.

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