RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Breakthrough Therapy (China), Orphan Drug (South Korea), Paediatric investigation plan (European Union), Promising Innovative Medicine (United Kingdom), Priority Review (United States)

Structure/Sequence

Sequence Code 18457995

Source: *****

Clinical Trials associated with Efgartigimod Alfa

NCT06831058

/ Not yet recruitingPhase 2IIT

A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

Immune-mediated Thrombotic thrombocytopenic purpura (iTTP) is a rare, autoimmune disorder characterized by life-threatening episodes of thrombocytopenia, microangiopathic hemolytic anemia and organ damage. Patients have an unpredictable course punctuated by relapses associated with autoantibody-mediated (primarily IgG) depletion of ADAMTS13, a key regulator of coagulation. ADAMTS13 deficiency during remission has been associated with increased risk of relapse, but also, and potentially more devastating, ischemic stroke.
Until recently, it was presumed that rituximab (a monoclonal antibody targeting B cells) improved relapse-free survival in most patients, but this was based on findings from very small studies. Given concern about stroke and relapse risk, preventive immunosuppression with rituximab has also recently come into practice for patients with falling ADAMTS13 activity (ADAMTS13-relapse). It is expected that following efgartigimod therapy, there will be a rise in ADAMTS13 activity to the normal range that will be sustained during the treatment period. Following withdrawal of therapy, it is expected that most participants will experience a fall in ADAMTS13 activity, demonstrating the safety and efficacy in efgartigimod to reliably but temporarily reduce pathogenic antibodies. This would demonstrate the potential efficacy for efgartigimod as a maintenance therapy to safely prevent relapse of iTTP to be further explored in a larger efficacy study.

Start Date01 May 2025

Sponsor / Collaborator

University of Minnesota

NCT06885762

/ Not yet recruitingPhase 2/3IIT

Safety and Efficacy of Efgartigimod in Guillain-Barré Syndrome

The goal of this clinical trial is to evaluating the efficacy and safety of efgartigimod in the treatment of Guillain-Barré Syndrome and exploring the immunological mechanisms of efgartigimod therapy in Guillain-Barré Syndrome. The main questions it aims to answer are:
Will efgartigimod improve the symptoms of participants? What medical problems do participants have when using efgartigimod?
Participants will:
On day 1 and day 5 of the treatment period, drug A was administered intravenously.
Keep a diary of their symptoms

Start Date13 Apr 2025

Sponsor / Collaborator

Xinqiao Hospital

ChiCTR2500098739

/ SuspendedNot ApplicableIIT

Efficacy and Safety of Efgartigimod in Anti-NMDAR Encephalitis: A Multicenter, Randomized, Placebo-Controlled Clinical Study

Start Date16 Mar 2025

Sponsor / Collaborator-

100 Clinical Results associated with Efgartigimod Alfa

100 Translational Medicine associated with Efgartigimod Alfa

100 Patents (Medical) associated with Efgartigimod Alfa

161

Literatures (Medical) associated with Efgartigimod Alfa

01 Apr 2025·JOURNAL OF NEUROIMMUNOLOGY

Response of refractory residual ocular symptoms to efgartigimod in generalized myasthenia gravis: A real-world case series

Article

Author: Zhou, Yufan ; He, Dingxian ; Yan, Chong ; Zhang, Jialong ; Luo, Sushan ; Xi, Jianying ; Zhang, Yexin ; Zhao, Chongbo

OBJECTIVE:

To evaluate the efficacy of efgartigimod (EFG) in treating residual ocular symptoms in myasthenia gravis (MG) patients with acetylcholine receptor antibodies (AChR-Ab).

METHODS:

Five MG patients with refractory residual ocular symptoms treated with EFG at Huashan Hospital were included. The demographic and clinical information was collected, and MG Activities of Daily Living (MG-ADL) scores and Quantitative Myasthenia Gravis (QMG) scores was elevated weekly during the 8-week follow up period. The time to reach minimal symptom expression (MSE) was also recorded.

RESULTS:

After a single cycle of EFG infusion, all five patients showed response in MG-ADL (≥2 points reduction), and three patients in QMG score (≥3 points reduction). The mean ± SD MG-ADL scores decreased significantly from 5.0 ± 1.0 at baseline to 1.8 ± 1.1 at weak 4 (p = 0.0027) and 1.8 ± 0.5 at weak 6 (p = 0.0027). The mean ± SD QMG score decreased from 5.8 ± 0.5 at baseline to 2.4 ± 1.7 at week 4 (p = 0.1357) and 1.0 ± 0.7 at week 6 (p = 0.0076). The proportions of patients reaching MSE at week 4, 6 and 8 were 20 % (1/5), 20 % (1/5), and 60 % (3/5), respectively.

CONCLUSIONS:

AChR-Ab+ MG patients with residual and refractory ocular symptoms could benefit from EFG treatment, while the duration of efficacy varied in individuals.

15 Mar 2025·INTERNAL MEDICINE

Efgartigimod Successfully Ameliorated Acute Exacerbation of Myasthenia Gravis with Anti-muscle-specific Kinase Antibodies

Article

Author: Yoshikawa, Masaaki ; Suzuyama, Kohei ; Inoue, Yukako ; Hoshino, Yuki ; Tsumura, Keisuke ; Shichijo, Chika ; Tanaka, Koya ; Ide, Toshihiro ; Koike, Haruki

We herein report two patients with anti-muscle-specific kinase (MuSK) antibody-positive myasthenia gravis who experienced rapid deterioration of weakness, particularly respiratory muscle weakness, necessitating non-invasive positive pressure ventilation (NIPPV) and were treated with efgartigimod. After treatment initiation, a rapid reduction in IgG levels and recovery from clinical symptoms were observed. NIPPV was no longer required two to three weeks after the first infusion of efgartigimod. These findings suggest that the reduction of IgG levels using efgartigimod is a good treatment option in patients with myasthenia gravis positive for anti-MuSK antibodies, even during the acute phase of the disease.

01 Mar 2025·MUSCLE & NERVE

Individualized Dosing of Efgartigimod in Patients With Generalized Myasthenia Gravis: Clinical Experience at a Single Center

Article

Author: Silvestri, Nicholas J.

ABSTRACT:

Introduction/Aims:

Neonatal Fc receptor (FcRn) inhibitors represent a promising treatment option for patients with generalized myasthenia gravis (gMG); however, data on clinical use are limited. The aim of this report is to describe one center's approach to efgartigimod dosing in patients with gMG.

Methods:

Medical records of patients with acetylcholine receptor antibody‐positive (AChR‐Ab+) gMG whose symptoms were not adequately controlled by oral medications and/or intravenous immunoglobulin who received efgartigimod between January 2022 and January 2024 were retrospectively evaluated. The first three efgartigimod cycles (10 mg/kg IV) were initiated at fixed intervals (4 once‐weekly infusions, with 4 weeks between cycles). After the third cycle, initiation of subsequent treatment cycles and time between cycles were determined individually by clinical evaluation. Effectiveness was measured by the Myasthenia Gravis Activities of Daily Living (MG‐ADL) scale. Adverse events and changes to concomitant therapies were monitored.

Results:

Nineteen patients were included and received a mean of 4.4 efgartigimod cycles, including two patients who discontinued after two cycles. All patients exhibited a clinically meaningful improvement in MG‐ADL total score from baseline to the end of the last cycle, with a mean improvement of 5.8 points. Seven (37%) patients achieved minimal symptom expression (MSE, MG‐ADL 0–1). From baseline to the end of the last cycle, daily prednisone dose was decreased or it was discontinued, while two patients initiated prednisone. Efgartigimod was generally well tolerated.

Discussion:

This approach to efgartigimod dosing resulted in substantial MG‐ADL score improvement in these patients with AChR‐Ab+ gMG as well as reduced daily dose and/or discontinuation of concomitant corticosteroids.

284

News (Medical) associated with Efgartigimod Alfa

22 Mar 2025

·endpts.com

Teenager dies after receiving Sarepta therapy; Immunovant gets Phase 3 win, but shifts focus; Alnylam’s big moment; and more

Hello and welcome back to the new Endpoints Weekly. It was another busy week in the biopharma world and we’ve got all of the headlines for you below. We also had an exclusive from pharma reporter Max Bayer this week, on how a meeting of CDC vaccine advisors has been rescheduled for the middle of April. The meeting was previously scheduled for late February but was postponed amid a broader pause on panels across federal health agencies. Keep it locked in at Endpoints for biopharma news, exclusives, analysis and more. — Max Gelman A 16-year-old boy died from liver failure after receiving Sarepta’s gene therapy for Duchenne muscular dystrophy Elevidys, the company said . Testing showed the patient had a recent cytomegalovirus infection (CMV), which the treating physician said was “a possible contributing factor,” according to Sarepta. Sarepta said this is the first death possibly related to Elevidys. More than 800 patients have received the gene therapy in trials and post-approval. Acute liver injury is a known side effect of the therapy, which was granted broad approval by the FDA last year. No deaths were reported in Sarepta’s pivotal studies of Elevidys. The company said it will “continue to gather and analyze information” related to the event. Sarepta reported the death to relevant health authorities, and said it “intends to update the prescribing information to appropriately represent this event.” Duchenne muscular dystrophy is an inherited disease that causes progressive muscle degeneration and weakness. It occurs primarily in young boys. 🤔 One of Roivant’s portfolio companies, Immunovant reported Phase 3 data for a rare disease drug called batoclimab this week, saying it succeeded in its primary goal. But rather than take batoclimab to market, Immunovant will instead shift its focus toward a program farther behind in the pipeline, IMVT-1402. Researchers were testing the drug in an autoimmune disorder called myasthenia gravis, which destroys the connection between nerves and muscles. Roivant CEO Matt Gline said although he was pleased with the outcome, batoclimab’s data were not stronger than already approved drugs — most notably Vyvgart, a multibillion-dollar product from argenx. In particular, the high dose only achieved a two-point reduction over placebo on a symptom measurement scale. “Our view is, why fight that fight when we can fight the better fight in a couple of years, when we can launch ‘1402,” Gline told Endpoints this week. And Roivant won’t treat this like another TL1A scenario, with Gline saying definitively that the company is not actively looking to out-license batoclimab or house it in another subsidiary. Roivant took that route with its anti-TL1A molecule a few years ago, buying it on the cheap from Pfizer, shepherding it through a mid-stage trial and then selling it to Roche for $5 billion-plus. However, Gline did not rule out a deal entirely, if an appealing one were to come along. The outcome also raises the stakes for IMVT-1402’s eventual readout, as Immunovant believes the program can produce better efficacy results than batoclimab while also improving on safety measurements. By the time IMVT-1402 reaches its pivotal readouts, there may be other approved drugs beyond Vyvgart: Amgen and Johnson & Johnson both recorded Phase 3 wins in myasthenia gravis last year for Uplizna and nipocalimab, respectively. ✅ Alnylam received approval for its RNAi drug Amvuttra in a heart disease known as transthyretin amyloid cardiomyopathy, or ATTR-CM, this week. It comes with a broad label that executives hope to use as an advantage over its competitors Pfizer and BridgeBio. Alnylam is pricing the drug in ATTR-CM at about $476,000 per year, which is the same price used for its other approved indication, hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy. The company has never been profitable, but that could change next year. Alnylam has been gearing up to launch Amvuttra in ATTR-CM for some time, getting into a growing market that analysts expect could reach $10 billion-plus by 2030. Alnylam lost $278 million last year and the CEO, Yvonne Greenstreet, believes the company can make enough in new Amvuttra sales to put the company “on the path” to profitability by the start of 2026. But many observers are skeptical that such goals will be achieved immediately. Analysts who spoke to Endpoints described a tough road for Alnylam ahead, especially for those individuals who haven’t had their disease treated yet. Both of the Pfizer and BridgeBio drugs are taken orally while Amvuttra needs to be injected by a physician. Ultimately, how patients receive treatment may come down to their individual circumstances. Alnylam is hoping its broader label will be one of the biggest keys to profitability . 💰 AstraZeneca has inked a deal to acquire a cell therapy startup for $425 million upfront. The company, called EsoBiotec, is working on an immune-shielded lentiviral vector dubbed ESO-T01 that’s designed to reprogram T lymphocytes into BCMA CAR-T cells within the patient’s body. AstraZeneca has promised up to $575 million more if certain development and regulatory milestones are met. EsoBiotec caught AstraZeneca’s attention at the JP Morgan Healthcare Conference in January, where it presented data that AstraZeneca’s head of oncology and hematology R&D Susan Galbraith said were “really quite impressive.” ESO-T01 entered a China-based trial in multiple myeloma patients in January, and data are expected to read out in the second half of this year. That isn’t the only deal AstraZeneca made this week. The British pharma also struck two R&D deals with Harbour BioMed and Syneron Bio, each of which are potentially worth more than $3.4 billion. Roche also inked a billion-dollar deal this week when it formed a multiyear partnership with Oxford Biotherapeutics. Roche agreed to put down $36 million upfront to work with OBT on finding new targets for antibody-based cancer treatments. OBT stands to make more than $1 billion in milestones and royalties, the companies announced. OBT says its discovery platform, called OGAP-Verify, is more sensitive than immunohistochemistry-based technology and can find targets that might otherwise be missed. And Sanofi said it would pay $600 million upfront for an early-stage cancer and autoimmune disease candidate from Dren Bio. The B cell-depleting antibody is in two Phase 1 trials: one in relapsed/refractory B cell non-Hodgkin lymphoma and another in a range of autoimmune conditions. Sanofi has promised up to $1.3 billion in development and launch milestones. 🧬Just over a year into his role as CEO of Illumina, Jacob Thaysen detailed his plans to expand into multiomics, a field that involves decoding proteins, RNA and other elements of biology. DNA is still core to Illumina’s business (as evidenced by a coiled staircase that mimics a DNA strand at its San Diego headquarters). But Thaysen said biology is “much more complicated.” Many diseases involve intricate biological interactions that DNA alone can’t fully explain. Illumina has faced a series of challenges in recent months, including competition from other companies in the sequencing space, threats to NIH funding that many of Illumina’s academic customers rely on, and China’s ban on imports of Illumina’s sequencers. But Thaysen said he plans to face those challenges head-on. “You can sit on the sideline and eat the popcorn, or you can go in and be a part of the solution,” he said. You can read more about Thaysen’s plans for Illumina’s transformation in Jared Whitlock’s feature. DON’T MISS:

Phase 3Clinical ResultDrug ApprovalPhase 1

19 Mar 2025

·www.prnewswire.com

CABLIVI Market Reports Positive Growth - Strong Momentum Expected to Continue | DelveInsight

Developed by Sanofi, CABLIVI is the first FDA-approved nanobody therapy for this condition. Increasing awareness, improved diagnostic rates, and rising demand for targeted therapies in hematology drive the market growth. With its niche indication and high treatment costs, CABLIVI holds a strong position in the rare disease therapeutics segment. LAS VEGAS, March 19, 2025 /PRNewswire/ -- DelveInsight's " CABLIVI Market Size, Forecast, and Market Insight Report" highlights the details around CABLIVI, a von Willebrand factor (vWF)-directed antibody fragment. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CABLIVI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Ablynx NV's CABLIVI (caplacizumab-yhdp) Overview CABLIVI is an antibody fragment that targets von Willebrand factor (vWF) and is used to treat adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. It works by binding to the A1-domain of vWF, preventing its interaction with platelets, which helps reduce vWF-mediated platelet adhesion and consumption. CABLIVI is approved in both the US and EU for treating aTTP. In 2024, CABLIVI sales reached €249 million, reflecting a 9.7% increase, driven by a rise in the number of patients identified for suitable treatment in the US, as well as new launches in Europe and other regions. The recommended CABLIVI dosing schedule is as follows: Day 1: Administer an 11 mg intravenous bolus injection at least 15 minutes before plasma exchange, followed by an 11 mg subcutaneous injection after the exchange on the same day. During Plasma Exchange Therapy: Administer 11 mg subcutaneously once daily after each plasma exchange session. After Plasma Exchange Therapy: Continue with 11 mg subcutaneous injections once daily for 30 days after the final plasma exchange. If signs of persistent underlying disease, such as low ADAMTS13 activity, are still present, treatment can be extended for up to 28 additional days. CABLIVI treatment should be discontinued if the patient experiences more than two recurrences of aTTP while on the medication. Learn more about CABLIVI projected market size for thrombocytopenia @ CABLIVI Market Potential Thrombocytopenia is a medical condition where the platelet count in the blood drops below the normal level (<150,000/µL). Common causes include heparin-induced thrombocytopenia, chemotherapy-induced thrombocytopenia, thrombocytopenia linked to chronic liver disease, immune thrombocytopenia (ITP), and thrombotic thrombocytopenic purpura (TTP). Among the 7MM, the United States reported the highest number of thrombocytopenia cases, accounting for about 40% of the total cases in 2023, according to DelveInsight. Treatment for thrombocytopenia varies based on its cause and severity, with the primary goal of preventing bleeding-related complications and improving patient outcomes. Approved therapies for thrombocytopenia in the US include MULPLETA, DOPTELET, TAVALISSE, PROMACTA, NPLATE, CABLIVI, GAMMAPLEX, OCTAGAM, RHOPHYLAC, and PRIVIGEN. Additionally, danaparoid, argatroban, and lepirudin are approved for treating thrombosis in patients with heparin-induced thrombocytopenia. For severe thrombocytopenia related to chronic liver disease, treatment options include platelet transfusion, splenic artery embolization, splenectomy, and transjugular intrahepatic portosystemic stent shunt (TIPSS) placement. The US holds approximately 60% of the thrombocytopenia market share, surpassing the combined market size of the EU4, the UK, and Japan. Market growth is expected to be driven by an increasing patient population, the introduction of new therapies, and deeper market penetration within the 7MM. Discover more about the thrombocytopenia market in detail @ Thrombocytopenia Market Report Emerging Competitors of CABLIVI The potential therapies that can mark a significant change in the thrombocytopenia treatment landscape and give tough competition to CABLIVI include Rilzabrutinib (Sanofi), Ianalumab (Novartis), Mezagitamab (Takeda), PF-06835375 (Pfizer), Povetacicept (Vertex), Cevidoplenib (Genosco/Oscotec), and Efgartigimod (ARGX-113) (Argenx), among others. In December 2024, Sanofi shared encouraging Phase III results at ASH for rilzabrutinib, its investigational oral BTK inhibitor, in patients with ITP. In June 2024, Takeda reported late-breaking Phase IIb data on Mezagitamab, highlighting its potential to revolutionize the treatment of Primary ITP. In Novartis' Q2 2024 update, the company forecasted key trial results: VAYHIT1 (NCT05653349) for first-line therapy, expected in 2026, and VAYHIT2 (NCT05653219) for second-line therapy, anticipated in 2025. Novartis also aims to file for ianalumabin approval in both first- and second-line ITP treatment by 2027 or later. To know more about the number of competing drugs in development, visit @ CABLIVI Market Positioning Compared to Other Drugs Key Milestones of CABLIVI In June 2022, at the 27th Annual European HematologyAssociation (EHA) Congress, Sanofi presented long-term safety and efficacy results from the post-HERCULES trial (NCT02878603), a three-year prospective follow-up study of patients with acquired thrombotic thrombocytopenic purpura (aTTP) who completed the Phase 3 HERCULES trial. In February 2019, the FDA approved CABLIVI (caplacizumab-yhdp) injection as the first therapy specifically indicated for treating adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening blood clotting disorder. It is to be used in combination with plasma exchange and immunosuppressive therapy. In September 2018, the European Commission approved CABLIVI (caplacizumab) for marketing, authorizing its use in treating adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. CABLIVI is the first therapy specifically designed for aTTP treatment. In July 2017, Ablynx announced a research collaboration and global exclusive licensing agreement with Sanofi, aiming to develop and commercialize Nanobody-based therapies for treating various immune-mediated inflammatory diseases. Discover how CABLIVI is shaping the thrombocytopenia treatment landscape @ CABLIVI FDA Approval CABLIVI Market Dynamics The primary driver of CABLIVI's market growth is the increasing recognition and diagnosis of aTTP, coupled with improved access to specialized treatments. Growing awareness among healthcare providers and advancements in diagnostic capabilities have facilitated early and accurate diagnosis, thereby boosting the demand for targeted therapies like CABLIVI. The drug's ability to prevent microthrombi formation, shorten the duration of plasma exchange, and reduce the risk of relapses has positioned it as a preferred option in the treatment landscape for aTTP. Moreover, Sanofi's robust commercial infrastructure and strategic marketing efforts have further contributed to market penetration and adoption. Despite its therapeutic benefits, CABLIVI faces challenges related to its high cost and reimbursement complexities. The specialized nature of aTTP treatment, combined with the requirement for hospital-based administration and close monitoring, adds to the overall cost burden on healthcare systems and patients. Additionally, competition from alternative therapies, including plasma exchange and immunosuppressive treatments, may limit its market expansion. Regulatory hurdles and the need for long-term safety data could also impact the drug's market trajectory. The CABLIVI market is expected to grow steadily, driven by ongoing clinical research and potential label expansions into related thrombotic disorders. Increased investments in rare disease treatments and orphan drug incentives are likely to support market growth. Moreover, the rise in strategic collaborations and partnerships to improve patient access and streamline reimbursement processes could enhance market uptake. However, balancing pricing pressures with broader patient access will remain a key factor in sustaining long-term market performance. Dive deeper to get more insight into CABLIVI's strengths & weaknesses relative to competitors @ CABLIVI Market Drug Report Table of Contents Related Reports Acquired Thrombotic Thrombocytopenic Purpura Pipeline Acquired Thrombotic Thrombocytopenic Purpura Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acquired thrombotic thrombocytopenic purpura companies, including Sanofi, TargED Biopharmaceuticals, Takeda, Genentech, Alexion Pharmaceuticals, among others. Thrombocytopenia Market Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key thrombocytopenia companies including Sanofi, Principia Biopharma, Baxalta, Takeda, Argenx, Millennium Pharmaceuticals, Biotest, GC Pharma, Genosco (Subsidiary of Oscotec), Rigel Pharmaceuticals, Kissei Pharmaceutical, Shionogi & Co., Ltd, Amgen, Novartis, Zenyaku Kogyo, among others. Thrombocytopenia Pipeline Thrombocytopenia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key thrombocytopenia companies, including Takeda, Argenx, Keros Therapeutics, Principia Biopharma, Momenta Pharmaceuticals, Veralox Therapeutics, Novartis Pharmaceuticals, Pfizer, HUTCHMED, Principia Biopharma, Genosco, UCB, among others. Immune Thrombocytopenia Market Immune Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key immune thrombocytopenia companies including Rigel Pharmaceuticals, Kissei Pharmaceutical, Sobi (Dova Pharmaceuticals), Amgen, Argenx, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultLicense out/inPhase 3Drug Approval

19 Mar 2025

·endpts.com

Immunovant will not take rare disease drug to regulators despite Phase 3 success

Immunovant will not seek approval for its candidate for the autoimmune disorder myasthenia gravis (MG), despite a reasonable showing in its pivotal trial. Instead, the company said Wednesday that it will use the data from the trial of batoclimab to develop its second-generation therapy, IMVT-1402. Investors were alarmed last May when the company delayed the batoclimab trial’s readout in order to prioritize development of IMVT-1402. Now the wait is over, and batoclimab — like Vyvgart an anti-FcRn antibody — has been shown to improve MG patients’ symptoms by 5.6 points on a widely-used rating scale. This measurement was taken after three months of treatment. Leerink analysts wrote in a March 17 note that in their base case, batoclimab would show a reduction on a symptom measurement scale called the Myasthenia Gravis Activities of Daily Living, or MG-ADL, of around four to five percentage points. This would be in line with competing anti-FcRn agents and complement inhibitors. MG is caused by immunoglobulin G (IgG) antibodies that disrupt the links between nerves and muscles, causing potentially life-threatening muscle weakness. The condition can worsen, with severely affected patients having difficulty swallowing and breathing. It can be treated with generic steroids, immunosuppressants and acetylcholinesterase inhibitors as well as Alexion’s Soliris, but some patients do not respond to these. Other approved therapies include complement factor C5 inhibitors like AstraZeneca’s Ultomiris and UCB’s Zilbrysq. But most excitement in this area centers on neonatal Fc receptor (FcRn) inhibitors. FcRn works to preserve levels of IgG, so blocking it can improve MG symptoms. Vyvgart was approved in 2021 on the basis of a slightly different interpretation of the MG-ADL scale to that reported by Immunovant today. After one cycle of treatment in the ADAPT trial, 68% of treated MG patients were MG-ADL responders, versus 30% of those given placebo. Response was defined as a drop of at least two points on the scale, with the improvement sustained for at least four weeks. In a prior Phase 3 trial in China, batoclimab had yielded a 58.2% response rate on MG-ADL vs. 31.3% with placebo. However, the response here had a more stringent definition than in the Vyvgart trial, requiring a three-point reduction from baseline. Leerink analysts note that if a two-point reduction in MG-ADL score had been used as the threshold for response in the China trial, the rate would have been 65.7% for batoclimab. A subcutaneous form of Vyvgart was approved for adults with MG in 2023. Another FcRn blocker, such as UCB’s Rystiggo is also approved for adults. Meanwhile, Johnson & Johnson said in July 2024 that its FcRn inhibitor nipocalimab had generated a 4.7-point drop on the MG-ADL in its Phase 3 trial, significantly more than the 3.3 points seen with placebo. Other mechanisms are also being developed. Last September , Amgen’s anti-CD19 antibody Uplizna produced a significant reduction on MG-ADL of 4.2 points after six months, versus 1.9 points with placebo. Separately, Immunovant reported data on batoclimab in a Phase 2 chronic inflammatory demyelinating polyneuropathy (CIDP) trial but said it would not seek approval for the drug in this disease either. Editor’s note: This is an evolving story and will be updated.

Phase 3Clinical ResultPhase 2Drug ApprovalImmunotherapy

100 Deals associated with Efgartigimod Alfa

External Link

KEGG	Wiki	ATC	Drug Bank
-	Efgartigimod Alfa	-	DB15270

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Purpura, Thrombocytopenic, Idiopathic	Japan	argenx SE	26 Mar 2024
Myasthenia Gravis	United States	arGEN-X BV	17 Dec 2021

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	NDA/BLA	China	Zai Lab (Shanghai) Co., Ltd.	24 Apr 2024
Primary Sjögren's syndrome	Phase 3	United States	argenx SE	04 Dec 2024
Graves Ophthalmopathy	Phase 3	United States	argenx SE	28 Mar 2024
Graves Ophthalmopathy	Phase 3	China	argenx SE	28 Mar 2024
Graves Ophthalmopathy	Phase 3	Japan	argenx SE	28 Mar 2024
Graves Ophthalmopathy	Phase 3	Austria	argenx SE	28 Mar 2024
Graves Ophthalmopathy	Phase 3	Bulgaria	argenx SE	28 Mar 2024
Graves Ophthalmopathy	Phase 3	France	argenx SE	28 Mar 2024
Graves Ophthalmopathy	Phase 3	Italy	argenx SE	28 Mar 2024
Graves Ophthalmopathy	Phase 3	Latvia	argenx SE	28 Mar 2024

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04188379 (ADVANCE \| ADVANCE IV, CTgov)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	131	efgartigimod (Efgartigimod)	agqrqiqgvd = jmwcduuddk euzojijnkg (hexrmxdjfk, ttwppdefmh - gmwalzwcbi) View more	-	13 Mar 2025
				Placebo (Placebo)	agqrqiqgvd = topcjlvxrc euzojijnkg (hexrmxdjfk, rnvzdqyqrn - abzvyqxccp) View more
NCT04687072 (ADVANCE SC, CTgov)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	207	Efgartigimod PH20 SC (Efgartigimod PH20 SC)	nbanuffdys = nxjstwpcsz wckffikdrq (vndpuiqeep, ekdsrvjlsg - vefzpszjtq) View more	-	31 Oct 2024
				Placebo PH20 SC (Placebo PH20 SC)	nbanuffdys = voxcdivzys wckffikdrq (vndpuiqeep, kkltwmxmbb - iimzyiwivx) View more
NCT04281472 (ADHERE, CTgov)	Phase 2	Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	322	Efgartigimod PH20 (Stage A: Efgartigimod PH20 SC)	mhfudzwxfi = wbmbstgnyn bowxnhwqnh (xeidnsyquf, ykinypjnzb - vhrdhjzixb) View more	-	20 Aug 2024
				Efgartigimod PH20 (Stage B: Efgartigimod PH20 SC)	llyzmmeusf(uchbiuuoqm) = dokhzfidtz wwuqnavdst (tiwmuvfxmd, vixhxypjya - mksgfvagab) View more
NCT04735432 (ADAPT-SC, NEWS) Manual	Phase 3	Myasthenia Gravis 乙酰胆碱受体（AChR）抗体阳性	-	艾加莫德	rnvknxquuz(jrccssvhue) = gpbqfqjdwe mmpfcyybng (bzzhcxqvia ) Met	Non-inferior	17 Jul 2024
				efgartigimod IV	rnvknxquuz(jrccssvhue) = twyepqxwuq mmpfcyybng (bzzhcxqvia ) Met
EAN2024	Not Applicable	-	-	Efgartigimod IV	vpufayhrer(jsjvsjeswu) = nkaipicsuj uetluldded (uromhwipmh, 2.2)	-	28 Jun 2024
EAN2024	Not Applicable	-	-	Efgartigimod	imblgbmhfq(ygakgdpyck) = edyqeskspc qlkarugvvk (dfaxuxmort ) View more	-	28 Jun 2024
EAN2024	Not Applicable	-	-	Efgartigimod IV	wbfxzxjqke(canfjwjnvm) = ezntqookxa qqswrdmkkf (psjywfuuln )	-	28 Jun 2024
				Efgartigimod SC	wbfxzxjqke(canfjwjnvm) = ysofhkfbar qqswrdmkkf (psjywfuuln )
P4-14.016 (AAN2024)	Phase 2/3	Autoimmune Diseases IgG-mediated autoimmune disorders	-	Efgartigimod	kcpkxmyshs(xgmdsenbni) = nidfemvbva xztdsrsdwr (mhvsusdusp )	Positive	09 Apr 2024
				Placebo	kcpkxmyshs(xgmdsenbni) = donwhllssc xztdsrsdwr (mhvsusdusp )
ADAPT/ADAPT+ (AAN2024)	Phase 3	Myasthenia Gravis acetylcholine receptor antibody-positive	-	Efgartigimod	cyitrlkkrd(sbpqjedcgc) = 0.3-point increase uzjmgiaqgq (manomyuxij ) View more	Positive	09 Apr 2024
				Placebo
NCT04281472 (ADHERE, AAN2024)	Phase 2	Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	322	Efgartigimod PH20 SC 1000mg	hmgjpktogh(javbbflhys) = Most TEAEs were mild to moderate; 3 deaths occurred, none being related to treatment obcemmjlwb (iwhxyvidtj )	Positive	09 Apr 2024

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