Anemia, Iron-Deficiency

CHMP recommends approval for XOANACYL, an oral therapy for use in the following therapeutic areas: Iron deficiency, chronic renal failure and hyperphosphatemiaFinal European Commission decision expected by June 2025; UK regulatory submission foreseen via MHRA’s international recognition procedureAVEROA seeking strategic commercial partners to bring XOANACYL to market across Europe Grenoble, France, April 2nd, 2025 - Averoa, a biopharmaceutical company bringing innovative therapeutic solutions to people with renal diseases, today announces a positive opinion from the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) for XOANACYL®, an oral therapy for chronic kidney disease (CKD). This milestone represents a key step toward a potential EU- approval. XOANACYL (Ferric Citrate as Coordination Complex) is an oral therapy offering a dual mechanism of action for patients with CKD. This new treatment provides an effective source of ferric iron for addressing iron deficiency and plays a pivotal role in controlling phosphorus absorption. XOANACYL offers a comprehensive approach to improving patient outcomes in CKD. XOANACYL was licensed from Akebia Therapeutics, Inc. (Akebia) in December 2022. Since then, Averoa has created an appropriate dossier, in particular by re-engineering the clinical package, to support the dual indication for the benefit of the European patients. The marketing authorization application (MAA) was filed in March 2024 through the centralized European procedure, supported by three pivotal clinical studies performed by Akebia Therapeutics. The positive opinion will now be reviewed by the European Committee (EC), which has the authority to approve medicines for European Union member states. The EC has approximately two months from the CHMP opinion to issue the final decision. Additionally, Averoa will submit a marketing authorization application to the Medicines and Healthcare products Regulatory Agency (MHRA), following the international recognition procedure (IRP) which has the authority to approve medicines for the UK market. A final MHRA decision is anticipated in the coming months. Luc-André Granier, President and Medical Director at Averoa, said: “This positive CHMP opinion is a pivotal moment for Averoa. It validates our ability to successfully navigate European regulatory pathways and brings us one step closer to offering a novel therapy to CKD patients. We are now focused on securing the right commercial partners to bring XOANACYL to market and accelerate patient access.” With EU approval expected in the coming months, Averoa is actively pursuing strategic commercial partnerships to support the launch and distribution of XOANACYL across Europe. The company is focused on collaboration models that can accelerate access to this innovative therapy and maximize its clinical and commercial impact. *** About CKDChronic Kidney Disease (CKD) describes the gradual loss of kidney function. It is a major public health problem resulting in an important burden for patients and healthcare systems. It affects millions of people with an estimated prevalence ranging from 3% to 17% in Europe. It is one of the ten leading causes of death in developed countries and can be due to multiple causes, including: high blood pressure, diabetes, high cholesterol, kidney infections, glomerulonephritis, polycystic kidney disease, genetic conditions, autoimmune diseases, kidney stones, smoking, age, and use of certain medicines. CKD induces two common debilitating disorders, Iron Deficiency Anemia (IDA) and Mineral Bone Disorders (MBD) that in turn is linked to an increase of FGF23 as a compensatory mechanism. Depending on the stage of the disease, CKD can induce cardiovascular diseases. CKD can progress to end-stage kidney failure, which is fatal without dialysis or a kidney transplant. About Xoanacyl ®Akebia Therapeutics granted to Averoa an exclusive license to develop and commercialize Xoanacyl® in the European Economic Area, Turkey, Switzerland and the United Kingdom. Xoanacyl® has been approved and is being commercialized in different regions: in the United States (US) under the brand name Auryxia® (ferric citrate) by Akebia Therapeutics, Inc.; in Japan as Riona® (ferric citrate hydrate) by Japan Tobacco Inc.; in Taiwan as Nephoxil® by Panion & BF Biotech Inc.; and in South Korea as Nephoxil® by Kyowa Kirin Korea Co. Ltd. About AveroaAveroa is a biopharmaceutical company, founded in December 2021, bringing innovative therapeutic solutions to people with renal diseases. Averoa’s goal is to build, advance and commercialize a strong pipeline of products to meet significant unmet medical needs of patients with kidney or metabolic diseases. More information is available on our website www.averoa-pharma.org and on our LinkedIn page Contacts: AveroaLuc-André GranierPresident & Medical Director Email: info@averoa-pharma.com MEDiSTRAVASandi Greenwood, Sylvie Berrebiaveroa@medistrava.com

Vafseo is currently only approved in dialysis patients with chronic kidney disease-related anemia thanks to safety concerns raised by the FDA. Despite finally winning a hard-fought FDA approval for its chronic kidney disease (CKD) anemia drug Vafseo last year, Akebia Therapeutics’ endgame remains focused on pushing the therapy into a wider pool of patients both on and off dialysis.Now, in tandem with Vafseo’s U.S. market debut, Akebia is putting its CKD expansion plans into action.Akebia on Thursday laid out plans to kick off a phase 3 clinical trial, dubbed Valor, to assess Vafseo as a potential treatment for anemia in late-stage CKD patients who aren’t on dialysis.Vafseo, which is part of the hypoxia-inducible factor–prolyl hydroxylase inhibitor (HIF-PHI) branch of meds, was approved last March to treat CKD-associated anemia in patients who have received dialysis for at least three months.Vafseo already boasts CKD anemia approvals in both dialysis and nondialysis patients in some countries outside the U.S., but safety concerns raised by the FDA—mainly around cardiovascular risks—led to the drug receiving a more limited label in the U.S.Regarding Valor, Akebia aims to recruit roughly 1,500 U.S. patients with stage 4 or 5 CKD not on dialysis for the late-stage cardiovascular outcomes trial, according to a presentation at the J.P. Morgan Healthcare Conference in January.The company said Thursday in its 2024 annual report that it has now submitted a trial protocol to the FDA and expects to meet with the regulator to discuss its designs. If all goes to plan, Akebia hopes to start the study in the second half of 2025.As for the population Akebia is targeting, stage 4 CKD is severe and points to advanced kidney damage with a high risk to progress to kidney failure. Stage 5 of the disease describes the point at which a patient’s kidneys fail, leading to dangerous buildups up fluid, electrolytes and waste in the body.Patients with end-stage CKD need dialysis or a kidney transplant to continue living, though some choose to manage their symptoms with conservative care instead.Akebia estimates there are around 550,000 patients with stage 4 or 5 CKD who are anemic and not receiving dialysis, though the company asserts that just 25% of those patients are currently treated for anemia. The company noted in its annual report that there are well-known risks to beginning dialysis with unmanaged anemia, while use of erythropoietin stimulating agents (ESAs)—a common class of CKD anemia drug—is “low and declining in the non-dialysis patient population.”“We believe the availability of an oral HIF-PH inhibitor within the non-dialysis patient population could address the issues contributing to the decline in ESA use among that patient population,” the company said in its annual filing.Akebia additionally pointed to a recent Spherix Global Insights report that found 78% of doctors said they would increase anemia treatment for nondialysis CKD patients if an HIF-PH inhibitor were approved.  A U.S. approval bid in nondialysis patients has long been part of Akebia’s overall plan for Vafseo, with the company noting at JPM that its upcoming Valor study could unlock a "multi-billion-dollar U.S. market opportunity."Meanwhile, when the company set Vafseo’s price tag notably higher than that of GSK’s rival HIF-PHI drug Jesduvroq in July, Akebia’s CEO, John Butler, explained that the company had to account for a potential label expansion under the Inflation Reduction Act (IRA), which limits how quickly drugmakers can raise a medication’s price.“[With] the IRA now, you really can’t change your price when you get a new indication,” Butler said on an investor call last summer. “You have one opportunity to price your drug, so we really have to think about that, that expanded label, as we thought about the strategic pricing and getting outside of dialysis.”Vafseo’s path to the FDA finish line, even in the more limited dialysis patient population, was anything but easy.Akebia’s original approval bid for Vafseo in both dialysis-dependent and nondialysis patients was shot down by the FDA in March 2022, with the regulator flagging safety concerns for the drug, including higher risk of thromboembolic events and liver injury.Akebia contested those concerns and soldiered on, working through a cost-cutting initiative and layoffs as it followed an FDA dispute resolution process that finally bore fruit in May 2023, Butler told Fierce Pharma around the time of the drug’s U.S. approval.Vafseo ultimately crossed the FDA finish line last March, thanks to a resubmission to the FDA girded with additional postmarketing safety data from Japanese patients.Vafseo started shipping out to U.S. patients Jan. 9 and is expected to reel in sales of $10 million to $11 million during its first quarter on the U.S. market, Akebia said in an earnings announcement Thursday.Three of the nation’s top four dialysis organizations have placed orders for Akebia’s anemia drug, and, as of the end of February, more than 500 prescribers had written Vafseo prescriptions, the company added. Akebia delivered the Vafseo progress report as it posted $46.5 million in total fourth-quarter revenue for 2024, down from the $56.2 million haul it brought home for the period in 2023. For the entire year, Akebia generated 2024 revenue of $160.2 million, down roughly 19% compared to 2023.Most of that sum came from Akebia’s phosphate binder Auryxia, which is cleared to control serum phosphorus levels in dialysis patients and as an iron replacement for treatment of iron deficiency anemia in adult CKD patients not on dialysis. The company also generated a small sum from license agreements and collaborations.

First participant with Von Willebrand Disease dosed with HMB-002, a novel subcutaneous therapy designed to increase endogenous von Willebrand Factor and Factor VIII levels COPENHAGEN, Denmark and CAMBRIDGE, MA, USA I February 27, 2025 I Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced the first participant with Von Willebrand Disease (VWD) has been dosed in Velora Pioneer, a Phase 1/2 clinical trial investigating HMB-002, a potential first-in-class subcutaneous therapy for VWD. “We are excited to advance the clinical development of HMB-002 for Von Willebrand Disease,” said Catherine Rea, MD, PhD, Vice President of Clinical Development at Hemab. “HMB-002 is the first fixed-dose subcutaneously administered antibody-based preventative therapy aiming to transform daily life for patients.” HMB-002 is a monovalent antibody uniquely designed to increase levels of von Willebrand Factor (VWF) and Factor VIII and provide a long-acting, subcutaneous prophylactic treatment for individuals with all types of VWD. Preclinical data for this investigational therapy were first presented at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), showcasing its potential to target the underlying patho-etiology of VWD by stabilizing VWF and promoting sustained hemostatic correction. “Von Willebrand Disease is characterized by a deficiency in VWF activity and sometimes associated with lowered levels of FVIII – both essential to control severe bleeding,” said Dr. Priyanka Raheja, the Royal London Hospital, Barts Health NHS Trust. “HMB-002 functions by increasing VWF and FVIII and could offer a complete disease correction for some and substantial modification for bleeding tendency for others. I am excited to be a contributing investigator on the Phase 1/2 trial.” The Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in individuals with VWD. The initial part of the study will evaluate single ascending doses of HMB-002 in a controlled setting. The first participant was dosed at Richmond Pharmacology in London under the supervision of Principal Investigator, Dr. Ulrike Lorch. The study is actively enrolling participants, with interim data anticipated later this year. “The initiation of the HMB-002 clinical program establishes Hemab as a multi-asset clinical-stage biotech and underscores our unwavering commitment to reimagine blood clotting therapies for people living with a variety of underserved bleeding disorders,” said Benny Sorensen, MD, PhD, CEO of Hemab. “Von Willebrand Disease has been documented for about 100 years. However, the current standard of care is sparse, ancient, and inconvenient. It’s time to leapfrog treatment into the 21 st century, and we believe HMB-002 can become a new back-bone therapy for all types of Von Willebrand Disease.” In addition to Velora Pioneer, Hemab is also conducting Velora Discover , a prospective natural history study collecting data on bleeds, treatments, and quality-of-life elements in people with VWD. Hemab is partnered with Haemnet, a specialist research and communications consultancy in the bleeding disorders community, to conduct VWD 360, the largest-ever natural history study in all types of VWD based on people’s detailed reported lived experience. Recent initial results were shared at EAHAD 2025. About Von Willebrand Disease Von Willebrand Disease (VWD) is the most common inherited bleeding disorder, characterized by quantitative or qualitative defects in Von Willebrand Factor (VWF), often resulting in frequent mucocutaneous bleeding events and heavy menstrual bleeding in women. The severity of bleeding ranges from low-volume events to potentially life-threatening hemorrhages. Chronic blood loss frequently leads to iron deficiency anemia, exacerbating the disease burden and reducing quality of life, particularly for those with clinically understated subtypes. Despite its prevalence, current treatment options for VWD primarily focus on managing symptoms rather than addressing the underlying defect in VWF production or function. About HMB-002 HMB-002 is a monovalent human antibody developed as the first-in-class prophylactic treatment for Von Willebrand Disease targeting the underlying root cause of the disease, a condition driven by a deficiency or defect in Von Willebrand Factor (VWF), a key regulator of hemostasis. By specifically targeting the C-terminal CK domain of VWF, which is distinct from regions critical to its essential interactions, HMB-002 shields the protein from degradation, boosting endogenous levels without compromising its function. Preclinical data suggest strong potential for meaningful therapeutic benefit. For more information, please visit clinicaltrials.gov (NCT06610201). About Hemab Therapeutics Hemab is a multiple clinical-asset biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA, and Copenhagen, Denmark, Hemab is progressing a pipeline of innovative therapeutic solutions, leveraging a variety of cutting-edge technologies and approaches to transform the treatment paradigm for patients with high unmet need. The company’s strategic guidance, Hemab 1-2-5 TM , targets building a pipeline of multiple development programs to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann thrombasthenia, Factor VII Deficiency, Von Willebrand Disease, and others. Learn more at hemab.com . Follow us on LinkedIn , Facebook , Instagram , and X . SOURCE: Hemab Therapeutics