Hemab (COAG) Q3 2026 Earnings Call Transcript

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DATE

Nov. 10, 2026

CALL PARTICIPANTS

• Chief Executive Officer - Benny Sorensen
• Chief Financial Officer - Mads Behrndt

TAKEAWAYS

• Cash and Marketable Securities -- $430.2 million as of Sept. 30, 2026, reflecting cash used for operating activities compared to $457.5 million as of June 30, 2026.
• Research and Development Expenses -- $24.6 million for the third quarter, representing an increase from $14.6 million in the prior year period driven by clinical program advancement and higher personnel costs.
• General and Administrative Expenses -- $5.1 million for the third quarter, compared to $2.5 million in the prior year due to professional services and other costs associated with operating as a public company.
• Net Loss -- $27.3 million, or $0.58 per share, for the quarter ending Sept. 30, 2026.
• Cash Runway -- Into 2029, based on current cash reserves intended to fund operations and capital expenditures.
• SOLARA Phase III Enrollment -- Approximately 75 participants aged 12 to 70 with Glanzmann thrombasthenia, aiming to provide each participant as their own control through a 24-week run-in period.
• SOLARA Dosing Regimen -- 0.2 milligrams per kilogram weekly, administered subcutaneously to optimize bleed reduction while managing peak exposure levels.
• Sutacimig Phase II Performance -- 80% mean annualized treated bleed rate reduction in the low-dose weekly cohort, reflecting sustained efficacy over a median exposure of 6.9 months.
• HMB-002 Proof of Mechanism -- 2.5-fold peak increase in Von Willebrand Factor and Factor VIII, exceeding the target product profile goal of a 1.5-fold increase.
• HMB-002 Bleed Reduction -- 1.6 mean annualized treated bleed rate compared to a baseline mean of 20.1, reflecting descriptive efficacy observations from the VELORA Pioneer trial.
• HMB-002 Enrollment Progress -- 110 patients in the VELORA Discover prospective run-in, providing a natural history data set for future interventional trials.
• HMB-003 Targeted Population -- More than 23 million women in the United States, representing a significant unmet need for non-hormonal heavy menstrual bleeding treatments.
• HMB-003 Clinical Timeline -- Mid-2027, for the anticipated release of initial Phase I clinical data from healthy volunteers to inform dose selection.
• Sutacimig Factor VII Applicability -- 88% binding across severe-to-moderate Factor VII variants, suggesting broad applicability based on preclinical data.
• Factor VII Deficiency Prothrombin Time -- Shortened by more than 50% in two participants receiving a single subcutaneous dose, validating the molecular mechanism in humans.
• Factor VII Deficiency Endogenous Accumulation -- Fivefold increase in endogenous total Factor VII for participants with available pharmacokinetic data, with exposure maintained through day 22.
• Glanzmann Thrombasthenia Disease Burden -- 88% of natural history study participants reported at least one bleed in the previous week, underscoring the lack of current prophylactic options.
• Glanzmann Thrombasthenia Hospitalization -- 65% of participants required a bleed-related hospital visit within a six-month period prior to trial entry.
• Sutacimig Addressable Markets -- Approximately 10,000 patients across Glanzmann thrombasthenia and Factor VII deficiency in key geographic regions.
• HMB-002 Addressable Market -- Approximately 120,000 diagnosed Von Willebrand Disease patients in key markets, with over 50,000 requiring treatment in the United States alone.

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RISKS

• Sorensen stated, "The FDA recommended that we consider a randomized, controlled trial. However, the FDA acknowledged our concern about the feasibility of randomization in Glanzmann thrombasthenia," noting a divergence from standard registrational pathways.
• Sorensen reported, "Three participants had Grade 2 thromboembolic events in higher exposure cohorts and/or with multiple risk factors," during earlier Phase II sutacimig studies.

SUMMARY

Hemab Therapeutics Holdings, Inc. (NASDAQ:COAG) is transitioning into a late-stage clinical biotechnology organization focused on blood coagulation disorders. Management reported the initiation of its first pivotal trial, SOLARA, which evaluates sutacimig for Glanzmann thrombasthenia following Phase II data that showed significant bleed reductions. The company stated that it is also advancing subcutaneous prophylactic candidates for Von Willebrand Disease and heavy menstrual bleeding, with clinical data updates expected through mid-2027. CFO Behrndt indicated that current cash and marketable securities provide a multiyear runway to support these clinical milestones into 2029.

• CEO Sorensen stated, "SOLARA is a landmark for the Glanzmann thrombasthenia community. The first participants have been consented and are scheduled to enter the run-in before end of the year."
• Management reported that HMB-002 clinical results from the VELORA Pioneer trial demonstrated durability supporting potential monthly subcutaneous dosing.
• The company stated that the Phase I trial of HMB-003 will inform clinical development plans for additional high unmet need bleeding conditions beyond heavy menstrual bleeding.
• CEO Sorensen noted that the Factor VII deficiency program aims to use molecular diagnosis as a predictor of patient response to sutacimig.
• Management indicated that the natural history data set from more than 100 patients in VELORA Discover provides a foundation for upcoming interventional trials in Von Willebrand Disease.
• The company appointed Keli Walbert to its Board of Directors to leverage her experience in commercial launches for rare and common disease medicines.

INDUSTRY GLOSSARY

• ATBR: Annualized treated bleed rate, a standard clinical metric for measuring the frequency of bleeding events requiring medical intervention over one year.
• Fibrinolysis: The enzymatic breakdown of fibrin in blood clots, which HMB-003 is designed to inhibit.
• Glanzmann thrombasthenia: A rare, severe congenital bleeding disorder caused by a defect in platelet aggregation.
• HMB-002: A monovalent antibody designed to raise levels of Von Willebrand Factor and Factor VIII by shielding them from degradation.
• HMB-003: A fatty-acid-conjugated peptide being developed as a non-hormonal treatment for heavy menstrual bleeding.
• PK/PD: Pharmacokinetics and pharmacodynamics, representing how a drug moves through the body and the body's biological response to it.
• Prothrombin time: A laboratory test that measures how long it takes for a blood sample to clot.
• SOLARA: The pivotal Phase III clinical trial of sutacimig in patients with Glanzmann thrombasthenia.
• Sutacimig: A bispecific antibody (formerly HMB-001) that recruits Factor VIIa to activated platelets to facilitate clot formation.

Full Conference Call Transcript

Operator: Welcome to Hemab Therapeutics Third Quarter 2026 Conference Call. Leading the call from management are Benny Sorensen, Chief Executive Officer; and Mads Behrndt, Chief Financial Officer. [Operator Instructions] Please be advised that today's conference is being recorded. Before we get started, I would like to remind everyone that some of the comments that management may make on this call about the company's future expectations, plans and prospects constitute forward-looking statements for purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995.

Actual results and events could differ materially from those expressed or implied in any forward-looking statements as a result of various important risks, uncertainties and other factors, including those set forth in Hemab's most recent filings with the SEC, including its quarterly report on Form 10-Q for the quarterly period ended September 30, 2026, and any other future filings that the company may make with the SEC. Any forward-looking statement represents our view only as of today and should not be relied upon as representing our view as of any subsequent date. You are cautioned not to place any undue reliance on these forward-looking statements, and Hemab disclaims any obligation to update such statements, except as required by law.

A replay of this call will be available on the Investors section of the Hemab website approximately 2 hours after the conclusion of the call. I will now hand the call over to Benny Sorensen, Chief Executive Officer.

Benny Sorensen: Thank you, operator. And thank you all for joining Hemab's first quarterly financial results call. We're glad you're here, and we look forward to using this forum every quarter to share our progress. At Hemab, we are developing targeted therapies that reimagine the treatment of blood coagulation disorders to sustain life and human resilience. Our philosophy is simple. All patients matter and people living with serious blood coagulation conditions should not have to live with inadequate treatment options. Let me start with the 3 pieces of news that matter the most for the coming months. First, yesterday, we announced the initiation of our pivotal Phase III trial of sutacimig in Glanzmann thrombasthenia.

Second, last week, we also announced the first clinical data from our Phase IIb trial of sutacimig in Factor VII deficiency and new menstrual bleed data in nonhuman primates treated with HMB-003. Updated data from the Factor VII deficiency trial will be presented at the ASH Annual Meeting on December 14 and the HMB-003 data on December 13. And third, in February 2027, at the 20th EAHAD Congress, we will present new data from our HMB-002 program in Von Willebrand Disease. These updates follow a remarkable stretch for Hemab. Since our IPO in May, we have not slowed down.

In July, we put our science on the global stage at ISTH with 9 presentations and data updates across all 3 of our programs. We unveiled HMB-003 and dosed the first participant in our Phase I trial. And we have now initiated SOLARA, our pivotal Phase III trial of sutacimig in Glanzmann thrombasthenia. It's our first pivotal trial, and it's the first pivotal trial of a subcutaneous prophylactic treatment in Glanzmann thrombasthenia, truly a defining moment for Hemab and for the Glanzmann community. As we move into late-stage development, we are building the organization to match our ambition. In September, we welcomed Keli Walbert to our Board.

Keli brings 2 decades of commercial leadership, including launches of medicines for conditions ranging from rare to very common, making her the perfect strategic match. We have also begun building our commercial strategy and team so we can position ourselves to bring our medicines to patients as quickly as possible. Across our programs, we listen to patients' lived experience, we quantified the neglected unmet need, we apply our deep coagulation expertise, and we use validated technologies as well as high-quality clinical development strategies. We believe a different future is possible, one where prevention, not just management becomes the standard of care. That vision comes to life across our first 3 programs.

Sutacimig is being developed for both Glanzmann thrombasthenia and Factor VII deficiency with approximately 10,000 patients in our key markets. HMB-002 targets Von Willebrand Disease, the most common inherited bleeding disorder with approximately 120,000 diagnosed patients in the same key markets. And HMB-003 targets heavy menstrual bleeding, which affects more than 23 million women in the United States alone. I'll walk through each program. Mads will review the financials, and then we will take your questions. HMB-003 is a fatty-acid-conjugated peptide that inhibits plasmid directly at its active site to stabilize clots.

Preclinically, it showed greater activity than tranexamic acid, today's primary non-hormonal therapy for heavy menstrual bleeding and PK properties supporting potential once-per-cycle dosing potentially offering an improvement over current short-acting therapies. We are initially developing HMB-003 for heavy menstrual bleeding, which has been called The Missed Vital Sign and is too often ignored. It is reported in roughly one in 3 women of reproductive age and is the leading cause of iron deficiency and anemia in this population. According to data compiled by Wellcome Leap, affected adult women miss an average of 3.6 weeks of work annually, costing the U.S. economy an estimated almost $100 billion.

Millions of women and girls have no approved non-hormonal, customized hemostatic treatment option for heavy menstrual bleeding, and that is the gap we are working to close with HMB-003. In September, we dosed the first participant in our randomized, placebo-controlled, single-ascending-dose Phase I trial in healthy volunteers, which will inform dose selection and clinical development plans for HMB-003 in heavy menstrual bleeding and across additional high unmet need bleeding conditions. We expect initial clinical data from the Phase I trial in mid-2027. We've also announced new preclinical data from a cynomolgus monkey model of menstrual bleeding in which HMB-003 reduced the proportion of severe bleeding days and shifted bleeding towards lower intensity compared with baseline.

We'll present these data at ASH on December 13. HMB-002 is a subcutaneous prophylactic monovalent antibody designed to shield von Willebrand Factor from degradation, raising the body's own levels of both von Willebrand Factor and Factor VIII without compromising function. At ISTH, data from the VELORA Pioneer Phase I/II trial showed clear proof of mechanism and candidly, the results exceeded our own expectations. We had set a 1.5-fold increase as the target product profile, but we saw peak increases of approximately 2.5-fold in both von Willebrand Factor and Factor VIII with normalized thrombin generation and APTT and durability supporting potentially monthly dosing. Most adverse events were mild-to-moderate with no serious or thrombotic events.

The single-ascending-dose portion was not designed to measure efficacy, so the early clinical observations were descriptive in nature. Still, we find them encouraging. Based on prospectively collected data, 8 out of 9 evaluable patients had zero treated bleeds in the 28 days after dosing with a mean annualized treated bleed rate of 1.6 compared with a baseline mean of 20.1 prior to treatment. VELORA Discover continues its successful enrollment with now 110 patients in prospective run-in and identified for future interventional trials. At EAHAD in February, we expect to present initial data from Part B, monthly subcutaneous dosing at 150 milligram and additional Part A data from single dosing at 300 milligram.

These data will cover patients with both Type 1 and Type 2a disease, adding data from 8 to 10 new patients with a total above 20 patients already. Lastly, let me turn to sutacimig, a bispecific antibody that binds and stabilizes the body's own Factor VIIa and recruits it to activated platelets to facilitate the formation of hemostatic plugs and control bleeding. In Glanzmann thrombasthenia, it holds Fast Track, Orphan Drug, and Breakthrough Therapy Designations from the FDA, along with regulatory designations in Europe and the U.K. Starting with Factor VII deficiency, a severe bleeding disorder affecting about one in 500,000 people where bleeds are recurrent, unpredictable and potentially life-threatening.

Our Phase IIb trial is ongoing at a single site in the U.K. At ISTH, preclinical data showed binding to 22 of 25 tested severe-to-moderate Factor VII variants or 88%, supporting a broad patient applicability. Last week, we announced our first clinical data from the single-ascending-dose cohort. As of July 24, 2 participants with severe congenital Factor VII deficiency had received a single subcutaneous dose. Adverse events were low grade and assessed as not related to treatment. Baseline prothrombin time of 180 seconds or more shortened by more than 50% in both participants. In the participants with PK and PD data available, sutacimig produced sustained multifold accumulation of endogenous total Factor VII with exposure maintained through day 22.

Updated data from all those participants will be presented at ASH on December 14. And now Glanzmann thrombasthenia. And here is the part that matters the most. There is no approved prophylactic treatment and care today is reactive rather than preventive. The burden is felt week after week. In Glanzmann's 360, an international natural history study, 88% of participants reported at least one bleed in the previous week and 65% required a bleed-related hospital visit in the prior 6 months. These patients or these people, they have kind of normalized living with unpredictable bleeds. Against that backdrop, our Phase II extension data from ISTH gives us real conviction in this program.

They showed sustained bleed reduction in 34 participants at a median of 6.9 months and up to 15.9 months of exposure. 92% of participants who bled during the run-in had fewer treated bleeds on sutacimig. The mean high-intensity annualized treated bleed rate was reduced by 62%. And in the low-dose weekly cohort, the mean annualized treated bleed rate fell by approximately 80%. There were no Grade 3 or higher related adverse events. Three participants had Grade 2 thromboembolic events in higher exposure cohorts and/or with multiple risk factors, all managed with routine anticoagulation therapy.

Based on these data, we have now initiated SOLARA, an open-label, single-arm, multicenter trial enrolling approximately 75 people with Glanzmann thrombasthenia aged 12 to 70 with at least 4 treated bleeds in the prior 12 months. After a 24-week run-in on current treatment, participants received 48 weeks of once-weekly subcutaneous sutacimig at 0.2 milligrams per kilogram, a regimen chosen with the goal to optimize bleed reduction while avoiding the high peak exposures associated with the observed thromboembolic events. The primary endpoint compares the annualized treated bleed rate on sutacimig with the run-in period with each participant serving as their own control.

Secondary endpoints include high-intensity bleed rates, safety, tolerability, immunogenicity and pharmacokinetics, and participants who complete treatment will have the opportunity to continue sutacimig in a separate open-label extension study. SOLARA is being conducted under Hemab's active IND. SOLARA is designed as a single-arm trial using intra-patient comparisons. The FDA recommended that we consider a randomized, controlled trial. However, the FDA acknowledged our concern about the feasibility of randomization in Glanzmann thrombasthenia. In light of substantial variability in treated bleed rates, the heterogeneity of the patient population and the practical consideration of randomized trials in rare diseases, we've determined to proceed with the single-arm trial. SOLARA is a landmark for the Glanzmann thrombasthenia community.

The first participants have been consented and are scheduled to enter the run-in before end of the year. With that, I'll hand it over to Mads.

Mads Behrndt-Eriksen: Thanks, Benny. I'll do a brief financial review. Cash, cash equivalents and marketable securities were $430.2 million as of September 30, compared with $457.5 million as of June 30. We continue to expect our cash to fund operations into 2029. R&D expenses were $24.6 million for the third quarter of '26 compared with $14.6 million for the third quarter of '25. The increase was primarily driven by advancement of our clinical programs and higher personnel costs. G&A expenses were $5.1 million for the third quarter of '26 compared with $2.5 million for the same period last year.

Net losses for the third quarter was $27.3 million or $0.58 per share compared with $17.1 million or $18.04 per share for the same period last year. With that, I'll now hand over the call to the operator to open the line for questions.

Operator: [Operator Instructions] Our first question comes from David Nierengarten with Wedbush Securities.

David Nierengarten: I had a couple. Just on the first one with the FDA feedback on the sutacimig study in Glanzmann, did they give any other rationale for their desire to have a randomized, controlled trial besides being a gold standard, so to speak. It just seems to be hard to match patients in this population. And then the second one was as you reported out before in previous data, the VTE, one patient had VTE, was obese in that study in Glanzmann. And I was just wondering if you had a weight range for inclusion into the study.

Benny Sorensen: Let me elaborate a little on the SOLARA and the regulatory path there. As I just mentioned, SOLARA is designed as a single-arm trial using an intra-patient comparison, which is a common registrational path in bleeding disorders. And in particular, for sutacimig in Glanzmann thrombasthenia, we are really following the observed clinical data from the 34 patients in our Phase II. As you all may recall, in a bleeding disorder like Glanzmann, that really has been honestly deprived from any innovation for decades, unfortunately, the treatment guidance, the practices, they vary very significantly and a single-arm trial is really the strongest way to control for that variability.

You guys also know that we have a breakthrough therapy designation, Fast Track, orphan drug and we do enjoy really close dialogues with the agency on how best to advance sutacimig in Glanzmann thrombasthenia. So with the extensive amount of dialogue we have had, we have implemented lots of the constructive feedback. Yes, the FDA recommended that we consider a randomized controlled trial. However, at the same time, acknowledge the variability that we are seeing and the concern about feasibility of randomization.

And in particular, given the substantial variability in treated bleed rates, the heterogeneity in the patient population and some of the practical considerations for a randomized trial, we believe this is what the data is telling us is the right thing to do going forward. And just lastly, as you all know, plenty of precedents for our single-arm trials in bleeding disorders, just to mention a few. In Von Willebrand Disease, both Vonvendi and Wilate did single-arm HYMPAVZI in hemophilia A and B. Tretten, which is concentrated in Factor XIII deficiency also, all ran prophy registrational trials as single-arm studies. So we have a high degree of confidence this is the right path forward.

To your second question, David, on weight ranges, I forgot what the top weight range is in the eligibility criteria. But here's the good news. The study will shortly be available on clinicaltrials.gov, and I'm sure that specific eligibility is probably in there, David. I'll have to follow-up with that.

Operator: Our next question comes from Cory Kasimov with Evercore ISI.

Unknown Analyst: This is [ Josh ] on for Cory. Thanks for taking our question. Ours is on Von Willebrand. Probably the most common investor question we get is what we should be expecting as far as amount of follow-up, and any of the efficacy markers or PK/PD data that we should be focusing on into that data.

Benny Sorensen: Yes, let me elaborate on that, too. Excited to kind of give an update on the HMB-002 data at EAHAD in February. And just kind of like go through it step by step. There will be additional single-ascending-dose data at 300 milligram. We are hoping to see a maximum accumulation plateau around two to threefold. And I think that the 300 milligram will be particularly informative for that. And if we then look at the PK that we already have on 20 milligrams, 50 milligrams and 150 milligrams, we might also see that the 300 milligrams is a dose that could last a little longer. But hey, let's see the data, what it tells us.

On the multiple-ascending-dose, it will be 150 milligram. Similarly here, we are hoping to see an average of two to threefold accumulation of von Willebrand Factor and a support for the once-monthly dosing. With regards to the clinical activity, we have said all along, right, we want to see more than 50% reduction in the annualized treated bleed rates. Yes, more is better, and it's still somewhat early days. And I think the last thing to emphasize, we will also be presenting data from both patients with Type 1 and Type 2a. And when you look into the population, that is really covering the vast majority of patients with Von Willebrand Disease.

Operator: Our next question comes from Rich Law with Goldman Sachs.

Jin Law: So I'm actually kind of surprised that the FDA asked for the randomized, controlled trial since we saw many pivotal trials in heme A and VWD using single-arm trials as you pointed out. I mean, at this point, do you have full alignment with the FDA, or do you believe you're taking some risk with a single-arm trial? And also like how did you and the FDA ended that discussion? And then I have a follow-up.

Benny Sorensen: Yes, I think as I just alluded to, Rich, I think, by the way, there is -- we have a high degree of confidence this is the right trial, in particular, taking into account the patient population, the empirical data we generated in Phase II, giving us deep insight to the variability of bleeds. And our regulatory friends here, they really acknowledge that, right? I think when they are saying recommend consider, that is probably for good order. And I think it's equally important that they acknowledge the concern in running a conventional randomized, controlled trial, in particularly in a bleeding disorder that has never ever had a prophylactic drug developed. So I think the risk is very low here.

And good news is we will continue dialogues with regulatory agencies, including the FDA going forward. The more we talk, the more we align. So I'm very confident that this is the right path forward, Rich. And you had a second question, right?

Jin Law: Yes. The second question is that at the ASH. I think we saw the ASH abstract you showed, I think there were 2 patients for that Factor VII deficiency. Just want to see is there any more that we're going to see? What's the endpoint? What's the final sample size that we should see at the ASH presentation?

Benny Sorensen: Yes. Let me also go into that. I'm very excited about sharing our Factor VII data at ASH.

And we have really aimed here to generate what I call like a binary data readout in a very small number of patients, pretty much trying to recapitulate the nonhuman primate study in patients with Factor VII deficiency and at the same time, validate all of the molecular biology studies that we have done we further aim to kind of show that our entire preclinical package with the preclinical NHP, the molecular insight into mutations leading to Factor VII deficiency and that knowledge of what mutations generate Factor VII variants that can bind to sutacimig and what mutations are less likely to bind, pretty much hoping to see that the molecular diagnosis can be an immediate predictor of when you have a responder or whether you have a nonresponder.

We are not elaborating on the number of patients, but I think I've been clear here that it's a very small number of patients, a study conducted just at one site in the United Kingdom. But I truly believe that this data is going to be sufficient for us, by the way, to make a substantial additional investment in Factor VII deficiency going forward.

Operator: Our next question comes from Maury Raycroft with Jefferies.

Maurice Raycroft: I was going to ask one on Glanzmann as well for the Phase III. Just wondering if there were just minimum ATBR reduction thresholds, running bleed rates for intra-patient control or any statistical aspects discussed with FDA that you could talk more about today? And what bars for success would be that would give you confidence around the potential regulatory risk?

Benny Sorensen: Yes. I think, by the way, very shortly, Maury, the entire eligibility criteria will be available on clinicaltrials.gov. We have like a minimum annualized treated bleed rate of 4 going in to make sure we can't completely eliminate a zero, but this should substantially reduce any patient coming in with zero.

Maurice Raycroft: And maybe a follow-up on, just with ASH coming up, we're expecting longer-term insights Star open-label extension data there. There's likely going to be compare and contrast with your 002 EHA data. So I'm wondering if you think we'll get perspective into differentiation at EHA on bleed control, maybe safety profile or stronger activity across different subtypes for Von Willebrand.

Benny Sorensen: Yes. As I said, we will be presenting data on Type 1 and Type 2a. As you also know, it is really, really hard for us to do a direct comparison between HMB-002 and other ongoing programs as we have taken kind of distinctly different kind of approaches in the way that we are collecting the data. I think in particular, we count bleeds that need treatment, and we are collecting those prospectively. We know that this is an endpoint that is used in registrational trial and bleeding disorders, so we're giving us a high degree of confidence.

And I think it's just very important for us to emphasize a treated bleed is one that is serious enough that the patient needed medical intervention to stop it. Importantly, we collect this prospectively using a validated electronic bleed diary. We have at least 4 months of baseline run-in. And what you will see at ASH is -- not at ASH at EAHAD is up to 4 months follow-up afterwards on treatment with HMB-002.

And I think the other thing I want to emphasize is, right now, enrolling both Type 1 and Type 2a, our VELORA Discover study with more than 100 patients kind of prospectively collected natural history data is really a gold mine for Hemab right now and giving us an increasing amount of confidence on being really data-driven and smart in our next steps of development.

Operator: Our next question comes from Joyce Zhou with JPMorgan.

Joyce Zhou: For SOLARA, can you provide any additional color on discussions with the agency around safeguards with respect to cardiovascular risk screening? Anything that came up in your discussions that was new relative to your expectations? Or was everything fairly in line?

Benny Sorensen: Yes. Joyce, I'm super proud of the way we have approached this. We have got a lot of praise from the regulatory authorities here in the way that we have designed our safety monitoring plan, the way that we have developed the eligibility criteria to be for all patients with Glanzmann thrombasthenia, but of course, safeguarding the molecule with the knowledge that we have. So I would say absolutely no surprises with regards to the way we have designed eligibility and safety monitoring. And I'm super proud of that because that means we have done the right thing from a clinical development point of view.

Operator: Our next question comes from Gaurav Maini with LifeSci Capital.

Gaurav Maini: So one thing that KOLs noted to us and that they really appreciated from our combos is that you can actually measure von Willebrand Factor given the mechanism of HMB-002. And some points to note here, right, titrating inadequate responders, confirming patients are pushed to, say, several hundred percent et cetera. I guess with this, how do you think about potential differentiation here, especially as we think about other late-stage competitors out there? And then I do have a quick follow-up.

Benny Sorensen: I think this is probably the ultimate strength of HMB-002. It is simple, it's intuitive. It directly targets, it directly corrects the underlying pathophysiology, and it is readily available for the clinicians to monitor whether the treatment is having the intended pharmacological effect. So I think that, that is a key differentiator. And remember, we are pretty much standing on the shoulders of 50 years of clinical experience with regards to this as a mechanism because this is exactly what you can do with DDAVP. You can give DDAVP, you can monitor von Willebrand Factor, you can monitor Factor VIII.

We know that's a very effective way of controlling an acute bleed management, but of course, not possible at all to use as a prophylactic therapy. But mechanistically, this is right in the wheelhouse of practicing hematologists in bleeding disorders. So I think that's a huge differentiator. We can simply just measure what they've always measured, von Willebrand Factor and Factor VIII. I'm excited about the data we presented at ISTH and just reflecting a bit more on those, give or take, 2.5-fold increase in von Willebrand Factor and Factor VIII. The population we are studying on average, have about 20% von Willebrand Factor and about 35% Factor VIII.

So you do that 2.5x that, you get on average into the normal range of a von Willebrand Factor and Factor VIII. That would be for a treating clinician, I think that could be very reassuring for a lot of patients. So I think you are spot on. I think that is a very clearly differentiating part of the molecule.

Gaurav Maini: Yes. And I appreciate the color there, Benny. A quick follow-up. So for Von Willebrand Disease as well, this kind of bar that the company has been guiding to ATBR of at least 50% reduction. Can you just remind us where that number comes from?

Benny Sorensen: Yes. That comes from the last, I guess, 45 years of development of drugs for bleeding disorders. It has been a clinically meaningful criteria, it's been a regulatory criteria and more than a 50% reduction in treated bleeds is considered clinically meaningful. And then, hey, more is better. There's no doubt about that. But while this is still a young ongoing Phase I/II trial, I think we'll keep it there. If the data looks much more favorable, we can always dial up the expectations for that. But I think for now, that's the right guidance to give.

Operator: Our next question comes from Michael King with Rodman & Renshaw.

Michael King: Congrats on your progress. I hate to drag you back to the regulatory discussions, but I just had a couple of follow-up questions about it. Benny, was there any consideration given to alternative randomized trials such as either 2:1, 3:1 randomization or brief periods where patients would be treated with tranexamic acid and then proceed on to sutacimig or any other types of protocols that might have been compromised with the agency?

Benny Sorensen: I can say it like this, we have turned every single possible stone to kind of make sure that we design the best possible trial with the highest probability of technical success here. So yes, I think we have elaborated on all possible trial designs. The reality is, as I keep coming back to that with the empirical data we have, we can acknowledge very substantial variability in the bleeding patterns and in the treatment patterns. And one can then say, oh, couldn't you just dial down that variability, assume that it will be better in the future. And then you could have designed all sorts of other designs.

But Michael, here's the reality when you do a Phase II trial in about 15, 16 sites, you get that variability. When we dial it up to a pivotal trial with approximately 75 patients enrolled across multiple countries, multiple sites, variability is rarely going down. It is more likely to stay the same. So after a lot of iteration in that data and a lot of dialogue with our regulatory friends, this is the right study design to carry forward to give patients and us the highest probability of a drug going all the way.

Michael King: And then was there any consideration given to geographic split of the trials? I know that obviously, this is something that affects consanguineous population. So was there any minimums, maximums or expectation of distribution of different geographies to make sure that there's at least some variability in the populations?

Benny Sorensen: Yes. By the way, we have really only selected countries and sites that fulfilled the extensive feasibility that we have conducted. So there will be a reflection of the variability of the population across geographic regions around the world. But we have been pretty strict with regards to countries and sites, investigator qualifications to make sure that we somewhat control the study conduct process. So yes, right now, the study is open here. It's already approved in a couple of other regulatory jurisdictions. And we'll kind of keep on updating you as we progress with the trial on what that distribution will look like over time.

Yes, right now, go on the SOLARA website and see our recruitment activities here in the United States. I'm super proud of the way we are prepared for this and the way we are conducting ourselves for this trial.

Michael King: Okay. Great. And then one quick one on von Willebrand. Insights has got data at ASH in Phase III right now. How do you anticipate navigating around their presence in the clinical trial setting?

Benny Sorensen: I think this is where I usually say innovation is good for patients. So I am, first and foremost, cheerleading for HMB-002. I believe we have the molecule with a more intuitive, simple mechanism of action. I think we have a molecule that you can monitor. I'm super excited about the data we have so far. I really don't pay much attention to where our competitors are, emphasizing instead the qualities and the strength of our molecule. And I think what we have heard from the clinical community is that there's a high degree of excitement for HMB-002. So that's the way we are running. I'm not worried about competition in this particular space here.

Operator: That concludes today's question-and-answer session. I'd like to turn the call back to Benny Sorensen for closing remarks.

Benny Sorensen: Thanks. Let me just end here say I could just not be more excited about where Hemab is today and where we are going. With SOLARA, Hemab has now begun its transition to a late-stage clinical company. Over the coming year, we expect updates across all our programs, starting with ASH in December, EAHAD in February. We believe we are well positioned to reset the standard of care across coagulation conditions of every size. At Hemab, all patients matter, and we are driven to give people living with serious coagulation conditions better options than they have today, and we are just getting started.

Thank you to the Hemab team, our partners and collaborators and most importantly, to all of the healthy volunteers and patients who participate making this work possible. Thank you all.

Operator: This concludes today's conference call. Thank you for participating. You may now disconnect.

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