argenx
🇧🇪Belgium
- Country
- 🇧🇪Belgium
- Ownership
- -
- Established
- 2008-01-01
- Employees
- -
- Market Cap
- $31.2B
- Website
- https://www.argenx.com/
Riliprubart Shows Promise in CIDP Treatment with Significant Neurofilament Light Reduction
• Phase 2 study results reveal that Sanofi's investigational riliprubart reduced plasma neurofilament light levels by 31% in patients with chronic inflammatory demyelinating polyneuropathy.
• Greater reductions in neurofilament light levels correlated with higher treatment response rates, with up to 69% of patients showing improvement in disability scores.
• Riliprubart, a selective inhibitor of the classical complement pathway, is now being evaluated in two global Phase 3 trials (MOBILIZE and VITALIZE) across 28 countries.
Global Thrombocytopenia Clinical Trials Market Sees Significant Growth in 2025
• The global thrombocytopenia clinical trials landscape is expanding rapidly with over 25 pharmaceutical companies developing 25+ treatment therapies, according to recent market analysis.
• Key industry players including GSK, Novartis, Amgen, and Sanofi are leading clinical development efforts, with several promising candidates in late-stage trials showing efficacy in reducing thrombocytopenia events.
• Recent advances include Takeda's TAK-755 demonstrating 60% reduction in thrombocytopenia events compared to standard of care, and HUTCHMED completing enrollment for its pivotal Phase III ESLIM-01 trial of sovleplenib for immune thrombocytopenia.
Takeda Spotlights Three Late-Stage Readouts as Potential "Inflection Point" Amid Vyvanse Generic Competition
• Takeda Pharmaceutical is positioning three upcoming late-stage clinical trial readouts as a strategic "inflection point" to offset revenue losses from Vyvanse's generic competition.
• The Japanese pharmaceutical giant is focusing on new product launches and pipeline advancement to maintain growth momentum, with particular emphasis on rare disease and neuroscience therapeutic areas.
• Industry analysts view Takeda's pipeline strategy as critical for the company's long-term financial stability, as it navigates the challenging transition period of losing exclusivity for one of its top-selling medications.
LEO Pharma's Temtokibart Shows Promising Results in Phase 2b Trial for Moderate-to-Severe Atopic Dermatitis
• LEO Pharma's investigational IL-22RA1 antagonist temtokibart achieved positive results for the primary endpoint at the three highest doses in a Phase 2b trial for moderate-to-severe atopic dermatitis.
• The treatment demonstrated a favorable safety profile with most adverse events being non-serious, mild to moderate in severity, and not considered treatment-related, supporting its potential as a novel therapeutic approach.
• Temtokibart represents a different mechanism of action by targeting the IL-22 pathway, potentially addressing unmet needs in atopic dermatitis treatment where current options may be insufficient.
Scholar Rock Appoints David L. Hallal as CEO, Strengthens Leadership Team Ahead of Apitegromab Launch for SMA
• Scholar Rock has appointed David L. Hallal as CEO, transitioning from his role as Chairman of the Board, as the company prepares for the global launch of apitegromab for spinal muscular atrophy.
• The company has bolstered its leadership team with three key appointments: Akshay Vaishnaw as President of R&D, R. Keith Woods as Chief Operating Officer, and Vikas Sinha as Chief Financial Officer.
• Apitegromab, a first-in-class muscle-targeted treatment for SMA, has received FDA priority review following successful Phase 3 trials, with potential approval and launch expected later this year.
FDA to Phase Out Animal Testing Requirement for Monoclonal Antibodies and Drugs
• The FDA announced it will phase out requirements for animal testing of monoclonal antibodies and other drugs, citing the availability of "more effective, human-relevant methods" for safety evaluation.
• The initiative aims to improve drug safety, accelerate evaluation processes, reduce R&D costs, and ultimately lower drug prices by implementing AI-based computational models, humanoid models, and real-world human data.
• This regulatory shift, enabled by the bipartisan FDA Modernization Act 2.0 of 2022, represents what FDA Commissioner Marty Makary calls "a paradigm shift in drug evaluation" that could expedite development of new treatments.
FDA Approves Pre-Filled Syringe for Self-Injection of Efgartigimod in gMG and CIDP Patients
• The FDA has approved a new pre-filled syringe formulation of efgartigimod (VYVGART Hytrulo) for self-injection in adults with generalized myasthenia gravis and chronic inflammatory demyelinating polyneuropathy.
• The self-injection option provides patients greater independence and flexibility, allowing treatment at home or while traveling, reducing the need for frequent clinic visits while maintaining the medication's established safety and efficacy profile.
• Developed through argenx's partnership with Halozyme's ENHANZE drug delivery technology, the pre-filled syringe enables rapid 20-30 second subcutaneous administration, with regulatory decisions expected in the EU, Japan, and Canada by 2025.
FDA Clears ZEISS INTRABEAM 700: Advanced Robotic-Assisted Platform for Intraoperative Radiotherapy
• ZEISS INTRABEAM 700 has received FDA 510(k) clearance, offering robotic-assisted precision for intraoperative radiation therapy in neuro-oncology and breast cancer treatment.
• The platform features a digital-first architecture with SMART Workflow Efficiency, including sterile single-use applicators, intuitive controls, and Radiance™ treatment planning software for pre-operative simulation.
• Currently being evaluated in multiple clinical trials including INTRAGO-II for glioblastoma and TARPIT-E for breast cancer, with results expected between 2025-2027.
Vertanical Achieves Phase 3 Success with Novel Non-Opioid Pain Medication
• Vertanical has announced positive Phase 3 results for its non-opioid pain management therapy, potentially offering a new alternative in the midst of the ongoing opioid crisis.
• The clinical success comes as other biotech companies face challenges, with Apriori Therapeutics implementing a 40% workforce reduction to extend its operational runway.
• Industry analysts suggest this breakthrough could significantly impact the pain management landscape, addressing the critical need for effective non-addictive alternatives to opioid medications.
Amgen's Uplizna Shows Sustained Efficacy in Myasthenia Gravis Patients Through One Year
• Amgen's Uplizna demonstrated durable efficacy in patients with acetylcholine receptor antibody-positive generalized myasthenia gravis, with 72.3% of treated patients showing significant improvement in daily living activities versus 45.2% on placebo at 52 weeks.
• The anti-CD19 antibody therapy maintained its efficacy with an infrequent dosing schedule of just two doses per year following an initial loading dose, potentially offering a competitive advantage over current treatments requiring more frequent administration.
• Regulatory filing for Uplizna in generalized myasthenia gravis is anticipated in the first half of 2025, with the FDA having already granted the therapy Orphan Drug Designation for this indication.
Patent Dispute Emerges Between Merck and Halozyme Over Injectable Keytruda Formulation
• Halozyme Therapeutics and Merck are heading into a patent battle over the development of an injectable formulation of the blockbuster cancer drug Keytruda.
• The dispute centers around Halozyme's drug delivery technology, which could potentially transform Keytruda's administration from intravenous to subcutaneous injection.
• This legal confrontation highlights the growing importance of drug delivery innovations in the pharmaceutical industry, particularly for established cancer therapeutics.
DARZALEX® Subcutaneous Regimen Receives CHMP Backing for Newly Diagnosed Multiple Myeloma Treatment
• Johnson & Johnson's DARZALEX® subcutaneous formulation combined with VRd receives positive CHMP recommendation for treating newly diagnosed multiple myeloma patients, regardless of transplant eligibility.
• The recommendation is supported by the Phase 3 CEPHEUS study, which evaluated the efficacy of daratumumab-VRd compared to VRd alone in 395 patients with newly diagnosed multiple myeloma.
• DARZALEX® has demonstrated significant impact in multiple myeloma treatment, having been used in over 618,000 patients worldwide and currently approved in eight indications.
FDA Grants Priority Review to Troriluzole for Spinocerebellar Ataxia Treatment
• Biohaven's troriluzole has received FDA Priority Review for treating spinocerebellar ataxia (SCA), potentially becoming the first FDA-approved treatment for this rare genetic neurodegenerative disease.
• Clinical trials demonstrated troriluzole slowed SCA disease progression by 50-70% over three years, representing a 1.5-2.2 year delay in disease advancement compared to controls.
• The FDA's decision on the New Drug Application is expected in Q3 2025, with Biohaven prepared to commercialize troriluzole in the US by 2025 if approved.
EMA Recommends Approval for New Cancer Treatments and Vaccines
• The European Medicines Agency (EMA) has recommended eight new products for EU-wide approval, expanding treatment options for various conditions.
• Several cancer treatments have received positive recommendations, potentially offering new hope for patients with different types of malignancies.
• A new antiparasitic combination has been endorsed for use in non-EU markets, addressing a critical need in regions affected by parasitic infections.
• The EMA is also reviewing new safety information regarding Leqembi, an Alzheimer's disease treatment, ensuring ongoing monitoring of its benefit-risk profile.
Eculizumab Shows Promise in Treating Thymoma-Associated Myasthenia Gravis
• A real-world study demonstrates that eculizumab significantly reduces Myasthenia Gravis Activities of Daily Living (MG-ADL) scores in patients with thymoma-associated myasthenia gravis.
• The research indicates a substantial decrease in the need for corticosteroids among patients treated with eculizumab by week 12.
• Clinically meaningful improvements were observed in 81.8% of patients, with initial benefits appearing within an average of 1.7 weeks after starting eculizumab.
• The study suggests eculizumab is a safe and effective treatment option for this severe myasthenia gravis subtype, warranting further investigation through larger randomized controlled trials.
Annexon's 2025 Outlook: Key Milestones for Neuroinflammatory Disease Therapies
• Annexon anticipates a Biologics License Application (BLA) submission for ANX005 in the first half of 2025, potentially transforming Guillain-Barré Syndrome (GBS) treatment.
• Enrollment in the Phase 3 ARCHER II trial for ANX007 in geographic atrophy (GA) is expected to be completed in the latter half of 2025.
• Clinical proof-of-concept data for ANX1502, an oral C1s inhibitor targeting autoimmune conditions, is anticipated in the first quarter of 2025.
• Annexon's current cash reserves are projected to sustain operations into the second half of 2026, supporting the achievement of critical developmental milestones.
Arvinas Advances Vepdegestrant into Phase 3 Trials for Breast Cancer and Updates Pipeline Milestones
• Arvinas plans to initiate two Phase 3 trials in 2025 for vepdegestrant in ER+/HER2- metastatic breast cancer, one in the first-line setting with atirmociclib and another in the second-line setting with a CDK4/6 inhibitor.
• Topline data from the Phase 3 VERITAC-2 monotherapy trial of vepdegestrant in second-line-plus ER+/HER2- metastatic breast cancer is anticipated in the first quarter of 2025.
• Arvinas is set to present initial data from the Phase 1 trial of ARV-393 in B-cell lymphomas and file an IND application for a novel PROTAC KRAS G12D degrader in 2025.
• Phase 1 trial with PROTAC LRRK2 degrader ARV-102 in patients with Parkinson’s disease has been initiated, with data expected to be presented in the first half of 2025.
Nipocalimab Receives FDA Priority Review for Generalized Myasthenia Gravis Treatment
• The FDA granted Priority Review to nipocalimab for treating gMG in antibody-positive patients, expediting its potential availability to patients.
• Phase 3 Vivacity-MG3 study results supported the application, demonstrating sustained disease control and significant MG-ADL score reduction.
• Nipocalimab, a monoclonal antibody, aims to reduce IgG autoantibodies, addressing the underlying cause of gMG without broad immunosuppression.
• Johnson & Johnson also submitted a Marketing Authorisation Application to the EMA, seeking approval of nipocalimab in gMG in Europe.
Scholar Rock Submits Apitegromab BLA to FDA for Spinal Muscular Atrophy Treatment
• Scholar Rock has submitted a Biologics License Application (BLA) to the FDA for apitegromab to treat spinal muscular atrophy (SMA).
• The BLA is supported by positive data from the Phase 3 SAPPHIRE trial, showing statistically significant motor function improvement.
• Apitegromab has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA for SMA treatment.
• Scholar Rock plans to initiate a Phase 2 trial (OPAL) in SMA patients under two years old in mid-2025.
VYVDURA Approved in Japan for Chronic Inflammatory Demyelinating Polyneuropathy
• Japan's MHLW has approved VYVDURA (efgartigimod alfa and hyaluronidase-qvfc) for treating adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP).
• VYVDURA is the first and only neonatal Fc receptor (FcRn) blocker approved for CIDP treatment, administered as a weekly subcutaneous injection.
• The approval is based on the ADHERE study, which demonstrated a 61% reduction in relapse risk compared to placebo (p<0.0001) and clinical improvement in 69% of patients.
• This approval expands treatment options for CIDP patients in Japan, offering a convenient at-home self-injection alternative.
© Copyright 2025. All Rights Reserved by MedPath