Efgartigimod/Hyaluronidase

VYVDURA ® now approved for at-home self-injection in Japan for both generalized myasthenia gravis and CIDP argenx’s VYVGART ® and VYVDURA portfolio approved in Japan for three indications – first country globally with access across three indications AMSTERDAM, The Netherlands I December 27, 2024 I argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved VYVDURA for adults with chronic inflammatory demyelinating polyneuropathy (CIDP). VYVDURA is approved for CIDP as a once weekly 30-to-90 second subcutaneous injection, which can be self-administered at home, and is the first and only neonatal Fc receptor (FcRn) blocker approved for the treatment of CIDP. “CIDP is a rare and debilitating disease for which there has been little innovation in treatment in 30 years,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx. “With VYVDURA, CIDP patients in Japan now have access to a novel therapy with a focused mode of action offering a convenient 30-to-90 second at-home self-injection option with an established efficacy and safety profile, as demonstrated by the ADHERE trial and real-world evidence. By extending the reach of this transformational therapy to thousands more patients, argenx continues to bring efgartigimod, our first-in-class FcRn blocker, to more patients in Japan and around the world suffering from severe autoimmune disease.” CIDP is a progressive, immune-mediated rare and debilitating neuromuscular disorder of the peripheral nervous system. Patients experience a range of disabling mobility and sensory issues, including trouble standing from a seated position, pain and fatigue, and frequent tripping or falling. Many patients become wheelchair bound and are unable to work as the disease progresses. Currently, 85% of patients require ongoing treatment and nearly 88% of treated patients experience residual impairment and disability. The MHLW approval is based on the ADHERE Study, the largest clinical trial to date studying CIDP. In the ADHERE study, 69% (221/322) of patients treated with VYVDURA, regardless of prior treatment, demonstrated evidence of clinical improvement, including improvements in mobility, function and strength. ADHERE met its primary endpoint (p<0.0001) demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse versus placebo. Ninety-nine percent of trial participants elected to participate in the ADHERE+ open-label extension. The safety results were generally consistent with the known safety profile of VYVDURA in previous clinical studies and real-world use. VYVDURA was approved by the MHLW for manufacturing and marketing in January 2024 and launched in April 2024 for the treatment of generalized myasthenia gravis (gMG). In March 2024, VYVDURA was designated as an Orphan Drug for the treatment of CIDP by the MHLW. See FDA-approved Important Safety Information below and full Prescribing Information for VYVDURA, which is marketed as VYVGART Hytrulo in the United States, for additional information. What is VYVGART® HYTRULO (efgartigimod alfa and hyaluronidase-qvfc)? VYVGART HYTRULO is a prescription medicine used for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP). IMPORTANT SAFETY INFORMATION Do not use VYVGART HYTRULO if you have a serious allergy to efgartigimod alfa, hyaluronidase, or any of the other ingredients in VYVGART HYTRULO. VYVGART HYTRULO can cause serious allergic reactions and a decrease in blood pressure leading to fainting. VYVGART HYTRULO may cause serious side effects, including: Tell your doctor if you have signs or symptoms of an infection, allergic reaction, or infusion-related reaction. These can happen while you are receiving your VYVGART HYTRULO treatment or afterward. Your doctor may need to pause or stop your treatment. Contact your doctor immediately if you have signs or symptoms of a serious allergic reaction. Before taking VYVGART HYTRULO, tell your doctor if you: What are the common side effects of VYVGART HYTRULO? The most common side effects in efgartigimod-alfa-fcab-treated patients were respiratory tract infection, headache, and urinary tract infection. Additional common side effects with VYVGART HYTRULO are injection site reactions, including rash, redness of the skin, itching sensation, bruising, pain, and hives. These are not all the possible side effects of VYVGART HYTRULO. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088. Please see the full Prescribing Information for VYVGART HYTRULO and talk to your doctor. About ADHERE Trial Design The ADHERE trial was a multicenter, randomized, double-blind, placebo-controlled trial evaluating VYVDURA® (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP). ADHERE enrolled 322 adult patients with CIDP who were treatment naïve (not on active treatment within the past six months or newly diagnosed) or being treated with immunoglobulin therapy or corticosteroids. The trial consisted of an open-label Stage A followed by a randomized, placebo-controlled Stage B. In order to be eligible for the trial, the diagnosis of CIDP was confirmed by an independent panel of experts. Patients entered a run-in stage, where any ongoing CIDP treatment was stopped and in order to be eligible for Stage A had to demonstrate active disease, with clinically meaningful worsening on at least one CIDP clinical assessment tool, including INCAT, I-RODS, or mean grip strength. Treatment naïve patients were able to skip the run-in period with proof of recent worsening. To advance to Stage B, patients needed to demonstrate evidence of clinical improvement (ECI) with VYVDURA. ECI was achieved through improvement of the INCAT score, or improvement on I-RODS or mean grip strength if those scales had demonstrated worsening during the run-in period. In Stage B, patients were randomized to either VYVDURA or placebo for up to 48 weeks. The primary endpoint was measured once 88 total relapses or events were achieved in Stage B and was based on the hazard ratio for the time to first adjusted INCAT deterioration (i.e. relapse). After Stage B, all patients had the option to roll-over to an open-label extension study to receive VYVDURA. About VYVDURA VYVDURA is a subcutaneous combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. In binding to the neonatal Fc receptor (FcRn), VYVDURA results in the reduction of circulating IgG. It is the first-and-only approved FcRn blocker administered by subcutaneous injection for the treatment of CIDP. VYVDURA is the proprietary name in Japan for subcutaneous efgartigimod alfa and recombinant human hyaluronidase PH20. It is marketed under different proprietary names in other regions. About Chronic Inflammatory Demyelinating Polyneuropathy Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. Although confirmation of disease pathophysiology is still emerging, there is increasing evidence that IgG antibodies play a key role in the damage to the peripheral nerves. People with CIDP experience fatigue, muscle weakness and a loss of feeling in their arms and legs that can get worse over time or may come and go. These symptoms can significantly impair a person’s ability to function in their daily lives. Without treatment, one-third of people living with CIDP will need a wheelchair. About argenx argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan, Israel, the EU, the UK, Canada and China. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, Twitter, and Instagram. SOURCE: argenx

Standard treatment for many autoimmune diseases includes drugs that suppress the immune system. Even when this approach works (and it doesn’t work for everyone), chronic immunosuppression puts patients at risk of infections among other complications. Nuvig Therapeutics is developing a first-in-class drug that could offer an alternative treatment for autoimmune diseases and the startup now has $161 million to advance clinical development of its lead program. Nuvig aims to treat autoimmune disease by tapping into a built-in mechanism of the body that tamps down inflammatory pathways. The Menlo Park, California-based company’s drugs are proteins engineered to bind to type II Fc receptors, a class of receptors that regulate immune responses. The company says the binding of its drug to these receptors leads to the expansion of regulatory T cells, or Tregs, a type of immune cell that suppresses immune responses. This approach also downregulates numerous inflammatory pathways . Lead Nuvig program NVG-2089 has Phase 1 results showing it was safe and well tolerated. Also important, the results showed the drug engaged its target. In Phase 2, Nuvig will test NVG-2089 as a treatment for chronic inflammatory demyelinating polyneuropathy (CIDP), a rare disorder in which the immune system attacks myelin, the protective sheath covering nerve fibers. This disease leads to nerve and motor problems as well as progressively worsening muscle function. Consumer / Employer Health Executives on Digital Transformation in Healthcare Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare. By MedCity News Standard CIDP treatment includes intravenous immunoglobulin (IVIg), an infusion of antibodies sourced from healthy donors. This treatment provides a patient with antibodies that block the inflammatory processes that damage myelin. Immunosuppressants are another CIDP treatment option. Not all patients respond to these therapies, but there’s already a new alternative. This past June, the FDA expanded the approval of Argenx drug Vyvgart Hytrulo to include CIDP. This injectable drug, which was initially approved to treat generalized myasthenia gravis, is an antibody fragment designed to bind to and block the neonatal Fc receptor (FcRn). Consequently, disease-driving autoantibodies go to a cellular system that degrades proteins. Through the first three quarters of 2024, Argenx reported more than $1.4 billion in product sales, a 77.5% increase over the same period in 2023. These figures includes Vyvgart, the original intravenously infused formulation of the FcRn inhibitor. Argenx has touted its drug’s potential uses in a wide range of autoimmune indications and the company is in various stages of clinical development testing its FcRn-blocker in multiple diseases. Similarly, Nuvig says its drug has the potential to address many autoimmune indications. But first, the company will see how its drug works in CIDP. Nuvig chose CIDP because it is an indication where IVIg is the standard of care and there is still unmet need for a better-tolerated therapy that is not immunosuppressive, co-founder and Chief Scientific Officer Pamela Conley said in an email. She added that her company’s approach is distinct from that of the Argenx drug and other FcRn inhibitors. Most autoimmune diseases, including CIDP, are not entirely mediated by the presence of autoantibodies, she explained. Rather, most of the damage in CIDP is mediated by immune cells that infiltrate the myelin that protect nerve cells. An FcRn blocker like Argenx’s drug does nothing to reduce the infiltration of immune cells into myelin, Conley said. Furthermore, blocking FcRn leads to an up to 80% reduction in overall circulating antibodies. Chronic maintenance of these low antibody levels is immunosuppressive, putting patients at risk of infection. presented by Market Trends to Watch for Health Systems and Their Specialty Pharmacies The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients. By Jake Gaffey, MBA, Chief Strategy Officer at Shields Health Solutions Rather than being immunosuppressive, Nuvig’s drug is immunomodulatory, Conley said. The drug offers a broader approach by down regulating the response of immune cells that drive inflammation while also expanding and activating Tregs, which is similar to what happens with IVIg. Conley added that clinical tests of the Argenx drug showed the overall response of CIDP patients was less than what has been observed in CIDP studies with IVIg. “We believe that the [mechanism of action] of NVG-2089 will lead to greater efficacy in CIDP than what was observed with Vyvgart,” Conley said. Nuvig, which is based on research from The Rockefeller University, emerged from stealth in 2022 backed by a $47 million Series A round. The new Series B round was co-led by Sanofi Ventures, Blue Owl Healthcare Opportunities (formerly Cowen Healthcare Investments), and Norwest Venture Partners. Other participants include new investors B Capital, Leaps by Bayer, Global BioAccess Fund, LOTTE Holdings, Alexandria Venture Investments, and funds managed by abrdn Inc., as well as earlier investors Novo Holdings, Platanus, Bristol Myers Squibb, Digitalis Ventures, and Mission BioCapital. Here’s a recap of other recent biotech financing news: —Metabolic medications biotech Antag Therapeutics raised €80 million (about $80.6 million) to enter the clinic with obesity drug candidate AT-7687, a peptide designed to block the GIP receptor. The drug is based on an endogenous GIP receptor antagonist. The drug is intended to be dosed alongside current or future obesity drugs, including those that block the GLP-1 receptor. Compared to other drugs being developed to block GIP, Antag says its approach offers flexibility in dosing that could improve both efficacy and tolerability. The drug could also be used by itself as a maintenance treatment. In tests in monkeys, Antag said AT-7687 dosed with a GLP-1 drug produced “best-in-industry weight loss” along with improved glycemic control and lipid profiles that were independent of weight changes. Antag added that these benefits were achieved without gastrointestinal side effects. The biotech’s pipeline includes drug combinations for other targets and a follow-on molecule that could offer monthly dosing. Versant Ventures led Antag’s Series A financing. —Maze Therapeutics raised $115 million to advance clinical development of its two lead programs. MZE829, an oral drug designed to block a protein called APOL1, is on track to Phase 2 testing in the rare kidney disease focal segmental glomerulosclerosis. MZE782, an oral inhibitor of SCL6A19, is on in Phase 1 testing; South San Francisco-based Maze plans to evaluate it in chronic kidney disease and phenylketonuria. Maze’s Series D round was co-led by Frazier Life Sciences and Deep Track Capital. —Adcendo, a developer of antibody drug conjugates for cancer, recently received FDA clearance to begin a Phase 1/2 test of ADCE-D01 in patients with advanced soft tissue sarcoma. Now it has $135 million to fund the clinical research. ADCE-D01 is designed to target uPARAP, a receptor that’s overexpressed in mesenchymal cancers, including subtypes of soft tissue sarcoma. Copenhagen-based Adcendo earlier this year expanded its Series A round of funding to €98 million (about $102.5 million). The new round, a Series B financing, was led by TCGX. —Kanglin Biotechnology unveiled $20 million in Series A financing to advance to clinical development with lead program, KL003, a gene therapy for the rare blood diseases beta thalassemia and sickle cell disease. Those diseases are already addressed by FDA-approved gene therapies from Vertex Pharmaceuticals and Bluebird Bio. Hangzhou, China-based Kanglin claims its lentiviral vector-based therapy can be best in class with shorter time to transfusion independence and engraftment of the genetically modified cells. No investor details were disclosed. —35Pharma raised $53 million to support clinical development of HS135 for pulmonary hypertension and HS235 for cardiometabolic disease and obesity. Both drugs are fusion proteins designed to block activins and growth differentiation factors (GDFs), proteins associated with many diseases. Frazier Life Sciences led the Series C round for Montreal-based 35Pharma. —Startup Valora Therapeutics emerged with $30 million to advance the development of a new class of drugs it calls antibody lectin chimera, or AbLecs. These engineered antibodies target the glycol-immune checkpoint, blocking glycans, sugar molecules on the surface of cells, from interacting with lectins. This approach has potential applications to cancer and autoimmune disease. Avalon BioVentures co-led the seed round of San Diego-based Valora. —Enveda raised $130 million to support its pipeline of 10 programs and multiple discovery candidates, all from an AI technology platform that applies machine learning techniques to metabolomics data. In October, the Boulder, Colorado-based company began a Phase 1 study of ENV-294, a potential oral anti-inflammatory drug for atopic dermatitis and other inflammatory disorders. Kinnevik and FPV led the Series C round, which brings the company’s funding total to $360 million. —TRexBio closed $84 million to advance its lead internal program, TRB-061, through early clinical development. The drug is an agonist of the TNFR2 receptor, which is abundant on regulatory T cells in the skin and gut. TRB-061 is a potential treatment for atopic dermatitis and ulcerative colitis; a Phase 1 study is on track to begin in the first half of 2025. Delos Capital led TRexBio’s Series B financing. —Wellington Management and Venrock led the $215 million financing of Metsera, a clinical-stage developer of metabolic disorder drugs. The Series B round comes seven months after Metsera launched backed by $290 million. The most advanced program of the New York-based company is a long-acting GLP-1 drug administered once-monthly — a key dosing edge over currently available GLP-1 drugs that patients must inject once weekly. Metsera expects to report preliminary Phase 2 data in the first half of 2025. —Alentis Therapeutics raised $181.4 million for clinical development of two antibody drug conjugates. ALE.P02 is on track to begin Phase 1/2 testing in advanced CLDN1-positive squamous solid tumors. ALE.P03 is being readied to enter the clinic in patients with CLDN-positive tumors. Alentis last raised money in 2023, a $103 million Series C round. Orbimed led the latest round, a Series D financing, along with co-leads Novo Holdings and Jeito Capital. —Arda Therapeutics raised $43 million to take a new approach to treating chronic disease. Rather than modulating the activity of proteins produced by cells that drive disease, San Francisco-based Arda is developing targeted biologic drugs that deplete those cells. Andreessen Horowitz led Arda’s Series A financing. —Evommune raised $115 million as the biotech prepares for upcoming data readouts for two skin disease drugs. Lead program EVO756 is on expected to report data in the first half of 2025 from a Phase 2 test in chronic inducible urticaria. Phase 2 tests are also planned for EVO756 in chronic spontaneous urticaria and for EVO301 in atopic dermatitis. Data from these tests are expected in 2026. RA Capital Management and Sectoral Asset Management led the Palo Alto-based biotech’s Series C financing. —Trace Neuroscience launched with $101 million and a lead program in development for amyotrophic lateral sclerosis (ALS). The South San Francisco-based biotech says its antisense oligonucleotide drug restore a protein called UNC13A, which in turn reestablishes healthy communication between nerves and muscle cells that are affected by neurodegenerative disorders. Third Rock Ventures led Trace’s Series A financing. —Axonis Therapeutics has $115 million to support a lead program in development for treating epilepsy and pain. The Boston-based company’s drug candidate, AXN-027, targets KCC2, a chloride transporter in the central nervous system that is essential for inhibitory neurotransmission. It’s the same target being pursued by Ovid Therapeutics, which acquired its KCC2-targeting molecules from AstraZeneca. Cormorant Asset Management and venBio Partners co-led Axonis’s Series A financing. —Blue Earth Therapeutics announced its presence in the growing radiopharmaceuticals sector with a $76.5 million Series A round. The Oxford, U.K.-based company will apply the proceeds toward Phase 2 tests of its radioligand therapies that target PSMA, the same protein hit by Novartis radiopharmaceutical Pluvicto. Soleus Capital led the round, which was co-led by Sands Capital Management. —Archon Biosciences, a Seattle startup from the University of Washington lab of Nobel Prize-winning scientist David Baker, emerged with $20 million and a platform technology that designs antibodies in a way that controls their structure. These antibody cages, or AbCs, can control how a drug distributes in the body to potentially offer advantages in safety and potency. Madrona Ventures led Archon’s seed financing. —Kivu Bioscience launched with $92 million, revealing antibody drug conjugates and technology licensed from Lonza subsidiary Synaffix. The startup, named for Lake Kivu in Africa, isn’t disclosing its cancer targets, but it plans to advance its lead program into Phase 1 testing in 2025. Novo Holdings led Kivu’s Series A financing. —Agomab raised $89 million to support ongoing clinical testing of AGMB-129, which is in development as a potential treatment for fibrostenosing Crohn’s disease. In this complication of the inflammatory gut disorder, scar tissue (fibrosis) and inflammation lead to thickening of the bowel. The Antwerp, Belgium-based biotech’s drug is a small molecule designed to block ALK5, a protein involved in a signaling pathway associated with fibrosis. Agomab’s Series D round added new investors Sanofi and Invus. —Pathos AI raised $62 million as it plans to advance to mid-stage clinical development. P-500, one of two clinical-stage assets the biotech acquired in the past year, is a brain-penetrating small molecule inhibitor of a target called PRMT5. The Chicago-based company says it will use its AI-powered technology to make patient selection and clinical trial design decisions. New Enterprise Associates led Pathos AI’s Series C round. —With a lead drug candidate in Phase 1/2 testing and a new program on its heels, B-cell therapies developer Be Biopharma raised $82 million in Series C financing. The Cambridge-based biotech’s technology engineers B cells to produce therapeutic proteins. Its most advanced program, BE-101, is in development for treating hemophilia B. The next program is BE-102, a potential treatment for hypophosphatasia, a rare inherited disorder that leads to weak bones and teeth. Be Bio last raised money in 2022, a $130 million Series B round. Picture: Feodora Chiosea, Getty Images