Annexon (NASDAQ:ANNX) used its presentation at the 44th Annual J.P. Morgan Healthcare Conference to outline a set of late-stage and mid-stage milestones that CEO Doug Love described as setting up 2026 as a “pivotal year” for the company. Love highlighted two registration-stage programs—ANX007 in geographic atrophy (GA) secondary to dry age-related macular degeneration (dry AMD) and ANX005 in Guillain-Barré syndrome (GBS)—alongside a proof-of-concept effort for oral small molecule ANX1502.
Platform focus: targeting the classical complement pathway upstream
Love said Annexon’s scientific approach centers on inhibiting C1q, which initiates the classical complement pathway and is implicated in harmful inflammation across multiple neuroinflammatory diseases. He contrasted this upstream strategy with “downstream first-generation approaches” targeting C3 and C5, which he said provide incomplete protection against inflammation. Annexon’s portfolio includes compartment-specific approaches: ANX005 is designed to block C1q systemically (and in the brain), ANX007 selectively targets C1q in the eye, and ANX1502 is an oral candidate intended to inhibit the classical pathway in systemic autoimmune settings.
ANX007: phase III program aims at vision preservation in geographic atrophy
In GA, Love emphasized that current approved therapies address lesion growth but have not been approved to preserve vision. He described GA as a neurodegenerative disease in which loss of photoreceptor neurons drives visual acuity decline, arguing that C1q localizes on photoreceptor cells and synapses and that upstream complement inhibition could better protect those structures.
Love reviewed phase II findings and said ANX007 showed 50% to 60% protection against loss of photoreceptors in the central retina. Functionally, he cited a dose-dependent reduction in the proportion of patients experiencing a 15-letter loss in best-corrected visual acuity (BCVA) over one year: 21% in sham versus roughly 10% with every-other-month dosing and roughly 6% with monthly dosing. He also cited low luminance visual acuity (LLVA) results, stating that 20% of sham patients lost vision versus 7.5% in the every-other-month arm and 10% in the monthly arm.
Love said monthly ANX007 reduced the risk of vision loss by 73% at month 12 and that separation emerged around month six and increased over time. He also described an on-treatment versus off-treatment analysis in which patients appeared to resume vision loss after dosing stopped, but did not “catch up” to sham, which he characterized as consistent with a disease-modifying effect.
Annexon’s phase III study, ARCHER II, is designed as a single study intended to support approval, with global regulatory alignment, Love said. He noted a total enrollment of 659 patients, an analysis at 15 months, and that the study enrolled about two months ahead of schedule and over-enrolled by 30 patients. In a Q&A exchange, Love said the study is powered at greater than 90% and that any statistically significant win on BCVA 15-letter loss would be clinically meaningful. He added that in phase II, sham patients had roughly “high teens” (17%–18%) 15-letter loss at 15 months, while treatment was about 6% in the monthly arm, and said phase III assumptions were conservative on both sham decline and treatment effect.
Love also discussed potential product presentation, saying Annexon plans a prefilled syringe and extended-release formulations. He noted ANX007’s characteristics—small 50 kD Fab fragment, low viscosity, non-PEGylated, and low injection volume (25 microliters)—as supportive of those plans. For safety, he said the company has not seen many safety events reported in other GA studies of downstream complement approaches, and he highlighted that Annexon has not observed an increased conversion to wet AMD versus sham, unlike reported 10%–15% conversion rates he cited for approved drugs.
ANX005: phase III win in Guillain-Barré syndrome and regulatory steps
Love described GBS as the leading cause of acute neuromuscular paralysis and said there are no approved therapies, with IVIG used off-label. He cited disease burden statistics including 150,000 cases worldwide and said treatment costs in the U.S. exceed $7 billion annually for about 8,000 patients.
Love said Annexon’s phase III program demonstrated a “robust win,” and he highlighted several outcomes he attributed to ANX005 treatment:
- Reduction of 25 inflammatory biomarkers within the first week, while IVIG and placebo showed no effect on those biomarkers.
- A 10-point improvement on the MRC sum score versus placebo at week one.
- A 2.5x greater likelihood of returning to a normal state by week 26.
- Return to walking 31 days sooner than placebo.
- Ventilator discontinuation 28 days earlier.
- ICU discharge seven days earlier.
He also said ANX005 was administered as a single infusion and that 90% of treated patients improved by week one, with a safety profile “like placebo.” Love noted that the company’s placebo-controlled phase III trial was the first such study in the disease area in 40 years.
On regulatory timing, Love said Annexon filed for European approval “last week” and anticipates filing with the FDA later in 2026. He said the European package included “substantial evidence” and a generalizability assessment, and that the FDA submission would be supplemented with additional U.S. and European data from the ongoing open-label FORWARD study. Love said FORWARD is currently planned for up to 30 patients, but he does not believe 30 will be necessary to support the submission, adding that Annexon expects consistent data and that the pharmacokinetic/pharmacodynamic profile is well characterized from dosing in other diseases.
When asked whether Annexon would launch in Europe before the U.S., Love said the decision is to be determined, noting that European review can take 12 to 15 months and that approvals could be close in time; he suggested the company would prioritize a U.S. launch first to establish pricing if timelines align.
ANX1502: oral program seeks proof of concept in Cold Agglutinin Disease
Love described ANX1502 as an “audacious” oral small-molecule effort aimed at autoimmune conditions typically treated with IV or subcutaneous therapies. He said earlier work identified tolerability and formulation issues: an initial film-coated formulation caused emesis, prompting a switch to an enteric-coated version that resolved emesis but introduced a food effect. Love said the current proof-of-concept study uses twice-daily dosing with patients fasted for roughly two hours before and after dosing to manage the food effect, and that the key objective measures in CAD include hemolysis markers such as bilirubin as well as complement activation. He said achieving target drug levels would be considered a “home run,” and that additional formulation work may follow to address the food effect.
Love concluded by reiterating that 2026 is expected to be a major year for Annexon, anchored by ANX007’s phase III GA readout, anticipated U.S. and EU regulatory activity for ANX005 in GBS, and proof-of-concept data for ANX1502.
About Annexon (NASDAQ:ANNX)
Annexon Inc is a clinical-stage biotechnology company focused on the discovery and development of complement-targeted therapies for patients with neurodegenerative and neuroimmune diseases. The company’s research platform centers on the inhibition of the C1 complex, a key initiator of the classical complement pathway implicated in several rare and life-threatening disorders. By selectively targeting upstream complement activation, Annexon aims to prevent the aberrant immune-mediated damage that characterizes conditions such as Guillain-Barré syndrome (GBS) and autoimmune neuropathies.
At the core of Annexon’s pipeline is ANX005, a humanized monoclonal antibody directed against the C1q subcomponent, currently in Phase 2 clinical trials for acute GBS and chronic neurodegenerative indications.
