Hemab Therapeutics Q3 2026 Earnings Call Transcript

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Provided by Quartr
November 10, 2026

Key Takeaways

  • Negative Sentiment: Hemab initiated SOLARA, its pivotal Phase III trial of sutacimig in Glanzmann thrombasthenia, with about 75 participants and a single-arm design comparing bleed rates before and during treatment. The FDA recommended considering a randomized trial; Hemab says it believes the chosen design is appropriate, but the agency’s feedback leaves regulatory uncertainty.
  • Positive Sentiment: Hemab highlighted Phase II extension results for sutacimig in Glanzmann, including fewer treated bleeds for 92% of participants who bled during run-in and a 62% reduction in mean high-intensity treated bleed rate. The company also reported three grade 2 thromboembolic events in higher-exposure cohorts and/or participants with multiple risk factors; it selected a lower weekly dose for SOLARA.
  • Positive Sentiment: HMB-002 showed proof of mechanism in von Willebrand disease, with peak increases of about 2.5-fold in von Willebrand factor and factor VIII and durability that may support monthly dosing. Hemab said eight of nine evaluable patients had no treated bleeds in the 28 days after dosing, but noted the early observations were descriptive and the study was not designed to measure efficacy.
  • Positive Sentiment: Sutacimig’s early Factor VII deficiency data showed a greater-than-50% shortening of prolonged prothrombin time in two participants, with exposure maintained through day 22; updated data are expected at ASH. Hemab also began a Phase I trial of HMB-003 and reported preclinical monkey data showing a shift toward less severe menstrual bleeding, with initial human data expected in mid-2027.
  • Positive Sentiment: Hemab reported $430.2 million in cash, equivalents and marketable securities at September 30, and expects its cash to fund operations into 2029. Quarterly R&D and G&A expenses rose year over year as clinical programs advanced and personnel costs increased.
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Earnings Conference Call
Hemab Therapeutics Q3 2026
00:00 / 00:00

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Operator

Welcome to Hemab Therapeutics' third quarter 2026 conference call. Leading the call from management are Benny Sørensen, Chief Executive Officer, and Mads Behrndt, Chief Financial Officer. After management's prepared remarks, we will open the call to your questions. To ask a question during the session, you will need to press star one one on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star one one again. 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.

Operator

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 Investor section of the Hemab website approximately two hours after the conclusion of the call.

Operator

I will now hand the call over to Benny Sørensen, Chief Executive Officer.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Thank you, operator, and thank you all for joining Hemab's first quarterly financial results call. We are glad you are 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 three 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 II-B trial of sutacimig in Factor VII deficiency and new menstrual bleed data in non-human primates treated with HMB-003.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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. 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 nine presentations and data updates across all three 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 is our first pivotal trial, and it is the first pivotal trial of a subcutaneous prophylactic treatment in Glanzmann thrombasthenia. Truly a defining moment for Hemab and for the Glanzmann community.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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 two 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 quantify 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 three programs.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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. HMB-003 targets heavy menstrual bleeding, which affects more than 23 million women in the U.S. alone. I will 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 plasmin 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. 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.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

It is reported in roughly one in three 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.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

We have 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 will present these data at ASH on December 13th. 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.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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, eight out of nine 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 mg and additional part A data from single dosing at 300 mg. These data will cover patients with both type 1 and type 2A disease, adding data from eight to 10 new patients, with a total above 20 patients already.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

As of July 24th, two 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 14th. Now, Glanzmann thrombasthenia. 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.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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 six 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 three or higher related adverse events.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Three participants had grade two 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 four treated bleeds in the prior 12 months. After a 24-week run-in on current treatment, participants receive 48 weeks of once-weekly subcutaneous sutacimig at 0.2 mg 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 rate, safety, tolerability, immunogenicity, and pharmacokinetics.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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
Mads Behrndt
CFO at Hemab Therapeutics

Thanks, Benny. I'll do a brief financial review. Cash, cash equivalents, and marketable securities were $430.2 million as of September 30th, compared with $457.5 million as of June 30th. We continue to expect our cash to fund operations into 2029. R&D expenses were $24.6 million for the third quarter of 2026, compared with $14.6 million for the third quarter of 2025. The increase was primarily driven by advancements of our clinical programs and higher personnel costs. G&A expenses were $5.1 million for the third quarter of 2026, 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.

Mads Behrndt
Mads Behrndt
CFO at Hemab Therapeutics

With that, I'll now hand over the call to the operator to open the line for questions.

Operator

As a reminder, if you'd like to ask a question at this time, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. Our first question comes from David Nierengarten with Wedbush Securities.

David Nierengarten
Analyst at Wedbush Securities

Hey, thanks for taking the question. 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 it being a gold standard, so to speak? Because it seems to be hard to match patients in this population. The second one was, as you reported out before in previous data, that the VTEs, one patient had VTEs, was obese in that study in Glanzmann, and I was just wondering if you had a weight range for inclusion into the study. Thank you.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Thank you, David, for the question. 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. In particular, 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.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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 control trial. However, at the same time, acknowledge the variability that we are seeing and the concern about feasibility of randomization. In particular, giving 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 are telling us is the right thing to do going forward. Just lastly, as you all know, plenty of precedents for 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 the factor XIII concentrate in factor XIII deficiency also, all ran prophylaxis registration trials as single-arm studies.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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. Here is the good news. The study will shortly be available on clinicaltrials.gov, and I am sure that specific eligibility is probably in there, David. I will have to follow up with that.

David Nierengarten
Analyst at Wedbush Securities

Okay. Thank you.

Operator

Our next question comes from Cory Kasimov with Evercore ISI.

Analyst at Evercore ISI

Hey, Benny and Mads. 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. Thank you so much.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Thank you, Josh. Let me elaborate on that too. Excited to give an update on the HMB-002 data at EAHAD in February. I'm just going to go through it step by step. There will be additional single ascending dose data at 300 mg. We are hoping to see a maximum accumulation plateau around two to three-fold. I think that the 300 mg will be particularly informative for that. If we then look at the PK that we already have on 20 mg, 50 mg, and 150 mg, we might also see that the 300 mg is a dose that could last a little longer. Let's see the data, what it tells us. On the multiple ascending dose, it will be 150 mg.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Similarly here, we are hoping to see an average of two to three-fold accumulation of von Willebrand factor and a support for the once-monthly dosing. With regards to the clinical activity, we have said all along, 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. I think the last thing to emphasize, we will also be presenting data from both patients with type 1 and type 2A. When you look into the population, that is really covering the vast majority of patients with von Willebrand disease.

Analyst at Evercore ISI

Thank you.

Operator

Our next question comes from Rich Law with Goldman Sachs.

Rich Law
Rich Law
Analyst at Goldman Sachs

Hey, guys. Good morning. I am actually kind of surprised that the FDA asked for the randomized control trial since we saw many pivotal trials in hemophilia 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 are taking some risk with a single-arm trial? How did you and the FDA end that discussion? Then I have a follow-up.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Yeah, I think, as I just alluded to, Rich, I think, by the way, 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. Our regulatory friends here, they really acknowledge that, right? I think when they are saying, recommend, consider, that is probably for good order. I think it is equally important that they acknowledge the concern in running a conventional randomized control 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. Good news is we will continue dialogues with regulatory agencies, including the FDA, going forward.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

The more we talk, the more we align, so I am very confident that this is the right path forward, Rich. You had a second question, right?

Rich Law
Rich Law
Analyst at Goldman Sachs

Yes. The second question is that at the ASH, I think we saw the ASH abstract you showed, I think there were two 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? Thank you.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

I think, and let me also go into that. I'm very excited about sharing our factor VII data at ASH. We have really aimed here to generate what I call a binary data readout in a very small number of patients. Pretty much trying to recapitulate the non-human 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 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 non-responder.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

We're 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 U.K. 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.

Rich Law
Rich Law
Analyst at Goldman Sachs

Great. Thank you.

Operator

Our next question comes from Maury Raycroft with Jefferies.

Maury Raycroft
Maury Raycroft
Analyst at Jefferies

Hi. Good morning. Thanks for taking my questions. I was going to ask one on Glanzmann as well for the phase III. Just wondering if there were minimum ATBR reduction thresholds, run-in bleed rates for intrapatient 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 Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Yeah, I think, by the way, very shortly, Maury, the entire eligibility criteria will be available on clinicaltrials.gov. We have a minimum annualized treated bleed rate of four going in to make sure we cannot completely eliminate a zero, but this should substantially reduce any patient coming in with zero.

Maury Raycroft
Maury Raycroft
Analyst at Jefferies

Got it. Okay. A follow-up on, just with ASH coming up, we are expecting longer-term Insights, STAR open-label extension data there. There is likely going to be compare and contrast with your 002 EAHAD data. So I am wondering if you think we will get perspective into differentiation at EAHAD on bleed control, maybe safety profile, or stronger activity across different subtypes for von Willebrand?

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Yeah. 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 distinctly different 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. 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.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

We have at least four months of baseline run-in, and what you will see at EAHAD, is up to four months follow-up afterwards on treatment with HMB-002. I think the other thing I want to emphasize is, right now, enrolling both type 1, type 2A, our VELORA Discover study with more than 100 patients worth of prospectively collected natural history data is really a goldmine 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.

Maury Raycroft
Maury Raycroft
Analyst at Jefferies

Got it. Thanks for the perspective.

Operator

Our next question comes from Joyce Zhou with JPMorgan.

Joyce Zhou
Joyce Zhou
Analyst at JPMorgan

Hey, guys. Thanks for taking our question this morning. 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? Thanks.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Yeah, Joyce, I am 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 criterias to be for all patients with Glanzmann thrombasthenia, but of course safeguarding the molecule with the knowledge that we have. I would say absolutely no surprises with regards to the way we have designed eligibility and safety monitoring. And I am 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
Gaurav Maini
Analyst at LifeSci Capital

Hey, good morning, everyone. Appreciate the update this morning. 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 in some points you note here, titrating inadequate responders, confirming patients aren't pushed to, say, several 100%, 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 Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Yeah, Gaurav, thank you for that point and that question. I think this is probably the ultimate strength of HMB-002. It is simple, it is 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. 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 that is a very effective way of controlling an acute bleed management, but of course, not possible at all to use as a prophylactic therapy.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

But mechanistically, this is right in the wheelhouse of practicing hematologists in bleeding disorders. I think that is a huge differentiator. We can simply just measure what they have always measured, von Willebrand factor and factor VIII. I am 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. And 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 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
Gaurav Maini
Analyst at LifeSci Capital

Yeah, and 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 of ATBR of at least 50% reduction, can you just remind us where that number comes from?

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Yeah. That comes from the last, I guess, 45 years of development of drugs for bleeding disorders. It has been a clinically meaningful criteria, it has been a regulatory criteria. More than a 50% reduction in treated bleeds is considered clinically meaningful. And then, hey, more is better, there is no doubt about that. But, while this is still a young ongoing phase I/II trial, I think we will 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 is the right guidance to give.

Gaurav Maini
Gaurav Maini
Analyst at LifeSci Capital

Awesome. Thank you.

Operator

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

Michael King
Michael King
Analyst at Rodman & Renshaw

Good morning, guys. Thanks for taking the question and 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 onto sutacimig or any other types of protocols that might have been compromised with the agency?

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Yeah, I think, Michael, thank you for that question. I can say it like this. We have turned every single possible stone to kind of make sure that we designed the best possible trial with the highest probability of technical success here. Yeah, 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. 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.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

When we dial it up to a pivotal trial with approximately 75 patients enrolled across multiple countries, multiple sites, that variability is rarely going down. It is more likely to stay the same. 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
Michael King
Analyst at Rodman & Renshaw

Okay. Was any consideration given to geographic split of the trials? Obviously, this is something that affects consanguineous populations. 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 Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

Yeah. By the way, we have really only selected countries and sites that fulfilled the extensive feasibility that we have conducted. 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. Yes, right now the study is open here. It's already approved in a couple of other regulatory jurisdictions. We'll kind of keep on updating you as we progress with the trial on what that distribution will look like over time. Yeah, right now, go on the SOLARA website and see our recruitment activities here in the U.S. I'm super proud of the way we are prepared for this and the way we are conducting ourselves for this trial.

Michael King
Michael King
Analyst at Rodman & Renshaw

Okay, great. One quick one on von Willebrand. Insights got data at ASH and phase III right now. How do you anticipate navigating around their presence in the clinical trial setting?

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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

Michael King
Michael King
Analyst at Rodman & Renshaw

Thanks for taking the questions, Benny.

Operator

That concludes today's question and answer session. I would like to turn the call back to Benny Sørensen for closing remarks.

Benny Sørensen
Benny Sørensen
CEO at Hemab Therapeutics

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.

Executives
    • Benny Sørensen
      Benny Sørensen
      CEO
    • Mads Behrndt
      Mads Behrndt
      CFO
Analysts
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