argenex (NASDAQ:ARGX) plans to continue executing its Vision 2030 strategy while expanding beyond its core FcRn franchise, newly appointed CEO Karen Massey said during a fireside discussion at the Bank of America Healthcare Conference.
Speaking with Bank of America biotech analyst Tazeen Ahmad, Massey said the company’s long-term goal remains reaching 50,000 patients by the end of the decade, securing 10 labeled indications and advancing five molecules into late-stage development. She said argenex’s first strategic pillar has been building and establishing VYVGART, while the next phase includes extending leadership in FcRn and building a non-FcRn portfolio that can become a meaningful contributor to the company.
FcRn Strategy Focuses on Convenience and Broader Use
Massey said argenex’s FcRn strategy is built around efficacy, safety and convenience across diseases with significant unmet need, including generalized myasthenia gravis, chronic inflammatory demyelinating polyneuropathy and immune thrombocytopenia.
The company launched VYVGART as an infusion and later introduced subcutaneous administration and a pre-filled syringe. Massey said the move from healthcare-provider administration to patient administration was a major factor in expanding the prescriber base and addressable market. She said an auto-injector could be an additional differentiator, though she characterized it as a smaller step than the move to a pre-filled syringe.
Massey also discussed next-generation FcRn assets, including one she referred to as “213,” which she said could move dosing to every four weeks. She said an oral FcRn option could further improve convenience while maintaining the efficacy and safety profile associated with the mechanism.
Myositis Seen as a Major Growth Opportunity
Massey described myositis as a significant potential growth area, saying argenex views the total addressable market as roughly 70,000 patients, comparable to the company’s current view of the generalized myasthenia gravis opportunity. The clinical trial is designed to evaluate three subtypes: immune-mediated necrotizing myopathy, dermatomyositis and polymyositis.
She said argenex is particularly excited about IMNM from a commercial and unmet-need standpoint. Massey said there are no approved treatments in IMNM, no competition on the horizon and about 20,000 patients, making the opportunity larger than the addressable market for CIDP at launch and larger than the myasthenia gravis addressable market when VYVGART launched.
Massey said dermatomyositis also remains an important opportunity, though she described it as a more heterogeneous disease with more competition. She said argenex estimates dermatomyositis has a 40,000-patient total addressable market.
The company’s myositis study uses a seamless Phase 2/Phase 3 basket design, Massey said. She added that argenex did not cap enrollment by subtype and will provide more information on the enrolled populations when topline data are available.
Rheumatology Expansion Could Support Multiple Programs
If argenex advances myositis, Massey said the company would likely expand its commercial organization to better cover rheumatology. She said argenex can leverage much of its existing infrastructure, but rheumatology has a different physician footprint and may require different patient support services.
Massey also discussed Sjogren’s disease, where she said argenex sees a strong biological rationale for FcRn inhibition. She said the company used competitor data to support the rationale for the mechanism, then conducted a small, focused Phase 2 study to better understand disease drivers and trial execution.
She said Sjogren’s is a large opportunity, with about 300,000 patients, including roughly 100,000 more severe patients with serious unmet need and systemic manifestations beyond dry eyes.
Empasiprubart Readout Expected in MMN
Massey highlighted empasiprubart, argenex’s C2-targeting complement therapy, as the company’s next medicine and its first late-stage program outside efgartigimod. A pivotal study in multifocal motor neuropathy is expected to read out in the fourth quarter, according to Ahmad’s question and Massey’s confirmation.
The MMN study is a head-to-head trial against IVIG with a 24-week primary endpoint based on grip strength. Massey said grip strength is meaningful to physicians and patients because it reflects functional improvement and is easy to understand. She said Phase 2 data showed grip strength improved over time, rather than only slowing decline.
Massey also said that in Phase 2, nine out of 10 patients reported feeling better on empasiprubart than at their peak on IVIG. The pivotal study includes multiple secondary endpoints, including physician- and patient-rated quality-of-life measures and MMN-RODS.
Discussing the C2 mechanism, Massey said argenex believes C2 is an attractive point of intervention because it sits at the intersection of the classical and lectin complement pathways while leaving the alternative pathway available, which she said may offer a safety benefit.
IgA Sweeper Program to Begin Shortly
Massey also addressed argenex’s planned program in IgA nephropathy, saying the company believes there is room for a new entrant despite increasing competition. She said argenex’s IgA sweeper is differentiated by targeting only IgA and producing rapid and sustained IgA reductions.
She said the program is expected to start shortly and confirmed there would be no data this year. On safety, Massey said the company will need to see clinical data, but noted that some people genetically lack IgA without an elevated infection risk, supporting the company’s view that targeting IgA could produce a favorable safety profile.
About argenex (NASDAQ:ARGX)
argenx (NASDAQ: ARGX) is a biotechnology company focused on the discovery, development and commercialization of antibody-based therapeutics for severe autoimmune and neuromuscular diseases. The company uses its proprietary SIMPLE Antibody platform to generate differentiated antibodies and engineered Fc regions, and it pursues mechanisms that modulate the neonatal Fc receptor (FcRn) to reduce pathogenic IgG levels. Argenx’s research and development activities span target identification, preclinical development and late-stage clinical programs aimed at addressing unmet needs in immunology.
The company’s lead product, efgartigimod (marketed as Vyvgart), is an FcRn antagonist developed to reduce circulating IgG antibodies and treat IgG-mediated disorders.
