Fitusiran

Hemab’s Kendall Square office in Cambridge, Massachusetts, gives visitors a rare view above the Charles River and downtown Boston. It’s an enviable backdrop for realizing the company’s vision of becoming the premier clotting biopharma. The view isn’t the only thing keeping spirits high at the newly public company, which completed an upsized $301.5 million IPO in May to fund its pipeline of therapies aimed at helping patients living with bleeding disorders. In addition to underwriters helping lift the final IPO proceeds to $346.7 million, the biotech's stock had tripled in value to $53.48 by August 12, up from a May debut of $18. The company ended June with $457.5 million in the bank, which Hemab expects to pave a cash runway into 2029. The biopharma has modestly expanded its workforce since the IPO, and now employs 80 people. With the post-IPO quiet period having come to an end, CEO Benny Sørensen, M.D., Ph.D., was free to sit down with Fierce to discuss Hemab’s future as a publicly listed company. Sørensen called the Nasdaq listing both a “natural step” and a “dream come true.” The IPO was a result of years of hard work and an expected part of the plan, but he was pleased with the scale of the response. The company, which has dual headquarters in Copenhagen, Denmark and Cambridge, has raised several rounds of funding over the years. This included a $55 million series A in 2021, a $135 million series B in 2023 and a $157 million series C last year. “It was clear that to advance the company, build out the portfolio, and be serious about this mission of building the ultimate coagulation company, the natural next step was a public offering to access those capital markets,” Sørensen explained. Fortunate that the company’s data and progress aligned with a wide-open IPO window, Sørensen said he encountered excitement for Hemab’s vision and progress when the company set out on its roadshow. “We had good interest from very high-quality investors,” he said. “Not just a few, but multiple—positioning us for building a book that is highly concentrated with top-notch investors. It felt like a dream come true.” Sørensen said Hemab had already achieved its initial goal of having five projects in development by 2025. The company recently shared data on three of those programs, including its bispecific antibody sutacimig, which is in phase 2 studies for patients with the rare inherited bleeding disorders Glanzmann thrombasthenia (GT) and factor VII deficiency (FVIID). At the International Society on Thrombosis and Haemostasis Congress last month, the company unveiled phase 2 data from a long-term extension study of sutacimig in GT that it said demonstrated the therapy's “potential to shift people ... from reactive, IV-dependent treatment to subcutaneous prophylaxis.” Hemab is gearing up for a phase 3 trial later this year. This summer also saw Hemab read out data for its phase 2-stage antibody HMB-002. The subcutaneous prophylactic injection targets the lifelong inherited bleeding disorder von Willebrand disease (VWD), and has been tied to a 2.4-fold or greater peak increase in von Willebrand factor, a glycoprotein that helps blood clot. “The hardworking may be allowed to be lucky too,” said Sørensen as he reflected on the findings. “The HMB-002 data were much better than anyone had hoped for or thought they would be.” Last month, Hemab unveiled HMB-003, a non-hormonal direct plasmin inhibitor designed to reduce bleeding across multiple settings, beginning with heavy menstrual bleeding. The asset is set to enter the clinic later this year. The fatty acid-conjugated peptide resembles Wegovy or Mounjaro in its delivery platform, but is tailored for women with heavy menstrual bleeding, Sørensen said. “Taboo and stigma hold on to this, and [heavy bleeding] generates iron deficiency and anemia,” the CEO explained. “On average, a woman with heavy menstrual bleeding is losing 3.6 weeks from work every year.” Sørensen emphasized the company’s commitment to women’s health, pointing out that Hemab has made an effort to ensure women are well represented in its trials. A total of 86% of the participants in the phase 1 study of HMB-001 were female, according to the biotech, with women accounting for half of the patients enrolled in the phase 2 trial for sutacimig in GT. “There's a recognition that this is a completely underappreciated, unmet need,” Sørensen said. “It requires modern treatment options to empower women to control this.” Sørensen has dedicated much of his career to hematology and coagulation. Before joining Hemab as CEO in 2021, his previous roles included senior director of clinical research at Alnylam Pharmaceuticals, where he led development of Qfitlia and other hematology products, as well as global medical director at Baxter Healthcare, where he helped develop the hemophilia therapy Adynovate and the VWD drug Vonvendi, which were acquired by Takeda in the 2019 Shire buyout. Before joining the biotech industry, he was the director of the hemostasis research unit and honorary lecturer at Guy’s and St. Thomas’ Hospital and King’s College London School of Medicine. Sørensen said two of Hemab's existing five pipeline programs have yet to be unveiled, and he already has his sights set on launching two commercial products, advancing two new late-stage programs and entering two other drugs into phase 1/2 by 2030. This will give Hemab “the flavor of a multi-product coagulation company,” he explained. The CEO is hopeful about the future for patients living with bleeding disorders. Sørensen pointed to the success of Genentech’s clotting medicine, which transformed hemophilia “from being a debilitating disease to being put on Hemlibra today.” These medical advances mean “you are not going to die from hemophilia,” he pointed out. Still, there is plenty of unmet need in the bleeding disorder space. “We left so many behind, and now we're on the mission of trying to close the gap,” Sørensen said.

The FDA has expanded its approval for Pfizer's hemophilia drug Hympavzi. It now will be available to a younger and broader patient population. The FDA has expanded the label for Pfizer’s subcutaneous hemophilia drug Hympavzi, now including patients age 6 and older who have hemophilia A or B.The anti-tissue factor pathway inhibitor was initially approved in October of 2024 for those age 12 and older with hemophilia A or B who have not developed the antibodies—also known as inhibitors—produced by the immune system that block or destroy infused clotting factor medications.The new expansion covers all patients 6 and older, regardless of their inhibitor status. The new nod also opens up the treatment to those 12 and older who have developed the inhibitors.Hympavzi, which is the first hemophilia medicine administered by way of a pre-filled, auto-injector pen, is administered weekly and is used as a prophylactic to reduce the risk of the painful bleeding episodes that accompany the disorder.    “For children who have to deal with bleeding episodes from an early age and for people living with hemophilia who develop inhibitors, treatment options have been limited and are often burdensome,” Guy Young, M.D., the Director of the Hemostasis and Thrombosis Center at Children’s Hospital in Los Angeles, said in a release.Hympavzi provides control with “a level of simplicity this community has long needed,” Young added.Hemophilia, which prevents blood from clotting properly and can include painful bleeding in the joints, impacts more than 800,000 people worldwide, Pfizer said. Childrens’ joints have growing cartilage and bone, which makes them particularly susceptible to damage caused by the bleeding episodes, the company added.Inhibitor antibodies develop in roughly 20% of those with hemophilia A and 3% of those with hemophilia B and are associated with an increased risk of uncontrolled bleeding. Factor replacement therapies can become ineffective in those who are classified as inhibitors.  The phase 3 Basis trial backed the approval of Hympavzi as a treatment for those age 12 and older with inhibitors, showing a 93% reduction in the annualized bleeding rate after patients switched from prior regimens. In the phase 3 Basis Kids trial, which included patients ages 6 to 17, annualized bleeding rates were also significantly reduced for those with and without inhibitors.Pfizer did not break out sales of Hympavzi in its first-quarter earnings report. Hympavzi was one of two hemophilia treatments Pfizer gained approval for in 2024. The other was hemophilia B gene therapy Beqvev, which was priced at $3.5 million and was removed from the market in February of last year after it failed to drum up much interest from patients or doctors. Roche’s Hemlibra is the top-selling branded hemophilia drug on the market, generating sales of 4.5 billion Swiss francs ($5.7 billion) last year. It is for those who have hemophilia A. Novo Nordisk’s once-daily subcutaneous Alhemo, for patients with hemophilia A or B, was approved in 2024. Last year, Sanofi and Alnylam gained FDA approval for Qfitlia, which is administered subcutaneously every one or two months and can be used by those with either type of hemophilia and regardless of inhibitor status.