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Hemab Therapeutics, led by Alnylam vet Benny Sorensen and chaired by John Maraganore, has raised $135 million from a new crop of investors as it begins pushing its blood disorder programs into the clinic. The raise gives Hemab’s a cash runway into 2025, and the biotech has a slate of goals it wants to check off before then, including having five clinical candidates by 2025. Maraganore said in an interview with Endpoints News that the biotech wants to be in Phase III trials by then as well. For now, however, the company has just gotten started with human trials. Hemab just began a Phase I/II trial of a bispecific antibody dubbed HMB-001, which it’s testing for Glanzmann thrombasthenia, a rare blood clotting disorder. In January, the biotech announced it had dosed its first patient with the drug. The trial is currently running in the UK, though Hemab said it plans on expanding that to the US and other European countries. Early data from the trial are expected later this year. The biotech is also working on a pre-clinical treatment for von Willebrand disease. For von Willebrand disease, Takeda’s enzyme replacement therapy Vonvendi was approved early last year. Vega Therapeutics, which like Hemab is backed by RA Capital, is also working on a treatment for the bleeding disorder and it hopes to start clinical trials this year. Importantly, Hemab is not working on hemophilia A or B, Sorensen emphasized during the joint interview, and is instead developing treatments for neglected bleeding disorders that currently have few or no approved options. Hemab said it hopes its Series B will bankroll the current study on HMB-001 and the start of a pivotal trial, as well as a Phase I/II trial for the von Willebrand disease therapy. Hemab’s Series B was led by Access Biotechnology, followed by Deep Track Capital, Avoro Capital, Invus, Rock Springs Capital and Danish fund Maj Invest Equity — all new investors. Investors from the biotech’s $55 million Series A — Novo Holdings, RA Capital Management, and HealthCap — also returned for the round. Hemab could have raised over $200 million, Maraganore said, but took the $135 million it needed to reach its 2025 goals. So what happens in 2025? “At that point in time, we can choose to go public or we can choose to raise another round privately, but at a higher valuation,” Maraganore said.

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that the U.S. Food & Drug Administration (FDA) approved VONVENDI® [von Willebrand factor (Recombinant)] for routine prophylaxis to reduce the frequency of bleeding episodes in patients with severe Type 3 von Willebrand disease (VWD) receiving on-demand therapy. VONVENDI is the only recombinant von Willebrand factor (VWF) replacement therapy, and the first and only treatment to reduce the frequency of bleeding episodes for severe Type 3 VWD approved by the FDA for routine prophylactic use. VONVENDI is now indicated for routine prophylaxis in adults with severe Type 3 VWD receiving on-demand therapy, as well as on-demand and perioperative bleed management in adults with VWD. “This approval is a major advancement for those living with severe Type 3 VWD and is a testament to Takeda's commitment to improve VWD care,” said Heather Dean, Vice President, U.S. Hematology Franchise Head, Takeda. “With routine prophylactic treatment, there is now a proactive strategy available for management of bleeding episodes and may offer people living with severe Type 3 VWD hope that bleed reduction is possible.” VWD is an inherited disorder that affects women and men equally.1 It is caused by a deficiency or defective function of VWF, one of several types of proteins in the blood that are needed for proper blood clotting.1 Due to this defective function or deficiency, blood is not able to clot effectively in people with VWD. VONVENDI is an infused product that is specifically designed to replace the body's missing or dysfunctional VWF.1 “VWD is a complex disease where both patients and providers may experience stress and uncertainty due to the unpredictable disease course and limited treatment options,” said Miguel A. Escobar, M.D., professor in the Department of Pediatrics and Internal Medicine at the McGovern Medical School at The University of Texas Health Science Center at Houston and an investigator in the VONVENDI prophylaxis study*. “A prophylactic treatment option may allow for greater disease control and the potential to enhance the standard of care.” The approval is based on data from a prospective, open-label, international multicenter study to evaluate efficacy and safety of prophylactic treatment in reducing the frequency of bleeding episodes in 10 adult patients diagnosed with severe Type 3 VWD who were previously treated on-demand. Based on descriptive statistics, the median annualized bleeding rates (ABR) for all bleeds (treated and untreated spontaneous and traumatic bleeding events) was reduced from historical median ABR 5.0 (range: 3.0, 159.0) to an on-study median ABR of 2.3 (range: 0, 157.9), which is a 54.7% reduction. The most common adverse reactions (≥2% of subjects) observed in adult patients treated with VONVENDI in clinical trials were headache, vomiting, nausea, dizziness, arthralgia, joint injury, vertigo, ALT increased and generalized pruritus. VONVENDI [von Willebrand factor (Recombinant)] Important Information What is VONVENDI? VONVENDI is used in adults (age 18 years and older) diagnosed with von Willebrand disease to: treat and control bleeding episodes prevent excessive bleeding during and after surgery reduce the number of bleeding episodes when used regularly (prophylaxis) in adults with severe Type 3 von Willebrand disease receiving on-demand therapy. Detailed Important Risk Information Who should not use VONVENDI? You should not use VONVENDI if you: Are allergic to any ingredients in VONVENDI. Are allergic to mice or hamsters. Tell your healthcare provider if you are pregnant or breastfeeding because VONVENDI may not be right for you. How should I use VONVENDI? Your first dose of VONVENDI for each bleeding episode may be administered with a recombinant factor VIII as instructed by your healthcare provider. Your healthcare provider will instruct you whether additional doses of VONVENDI with or without recombinant factor VIII are needed. What should I tell my healthcare provider before I use VONVENDI? You should tell your healthcare provider if you: Have or have had any medical problems. Take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements or herbal remedies. Have any allergies, including allergies to mice or hamsters. Are breastfeeding. It is not known if VONVENDI passes into your milk and if it can harm your baby. Are pregnant or planning to become pregnant. It is not known if VONVENDI can harm your unborn baby. Have been told that you have inhibitors to von Willebrand factor (because VONVENDI may not work for you). Have been told that you have inhibitors to blood coagulation factor VIII. What else should I know about VONVENDI and von Willebrand Disease? Your body can form inhibitors to von Willebrand factor or factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may stop VONVENDI or factor VIII from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to von Willebrand factor or factor VIII. What are the possible side effects of VONVENDI? You can have an allergic reaction to VONVENDI. Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting. Side effects that have been reported with VONVENDI include: headache, nausea, vomiting, tingling or burning at infusion site, chest discomfort, dizziness, joint pain, joint injury, increased liver enzyme level in blood, hot flashes, itching, high blood pressure, muscle twitching, unusual taste, blood clots and increased heart rate. Tell your healthcare provider about any side effects that bother you or do not go away. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. About von Willebrand disease (VWD) VWD is the most common inherited bleeding disorder, affecting up to one percent of the U.S. population. VWD is caused by a deficiency or dysfunction of von Willebrand factor (VWF), one of several types of proteins in the blood that are needed to facilitate proper blood clotting. Due to this defect or deficiency in VWF, blood is not able to clot effectively in people with VWD which may result in heavy menstrual periods, easy bruising, or frequent nose bleeds. Bleeding caused by VWD varies greatly among patients with this disease.1 About Takeda Hematology Following its recent acquisition of Shire, Takeda is a leader in hemophilia with the longest heritage and market-leading portfolio, backed by established safety and efficacy profiles with decades of real-world experience. We have 70+ years driving innovation for patients and a broad portfolio of 11 products across multiple bleeding disorders. Our experience as leaders in hematology means we are well prepared to meet today’s needs as we pursue future developments in the care of bleeding disorders. Together with the hematology community, we are raising expectations for the future, including earlier diagnosis, earlier and full protection against bleeds, and more personalized patient care.2 About Takeda Pharmaceutical Company Limited Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. Important Notice For the purposes of this notice, “press release” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. 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