Annexon (NASDAQ:ANNX) President and CEO Doug Love outlined a series of anticipated clinical and regulatory milestones during the 25th Annual Needham Healthcare Conference, describing 2026 as a “win year” after more than a decade of work developing therapies that target the classical complement pathway.
Key milestones highlighted for 2026
Love said the company expects multiple catalysts across its pipeline over the course of the year, led by programs in Guillain-Barré syndrome (GBS), geographic atrophy (GA), and an oral small-molecule candidate.
- GBS: Love said Annexon has filed for regulatory approval in Europe following a “very successful phase III pivotal study” and anticipates an approval decision in the “first part of 2027.” He added that the company anticipates filing for U.S. approval with the FDA “later this year,” describing submission of the BLA as a key milestone.
- ANX1502: Love said the company expects a proof-of-concept readout this year for ANX1502, which he described as “the first small molecule targeting the classical pathway.”
- Geographic atrophy: Love said the company’s phase III program for vonaprument, a classical-pathway-targeting therapy, is expected to read out in Q4 of this year.
Why Annexon is targeting C1q
In GA, Love said C1q localizes on photoreceptor synapses and drives inflammation that damages photoreceptors, which are critical for visual acuity. “Our approach is simply to block C1q right where it’s localized on diseased tissue,” he said, characterizing it as stopping inflammation “before it starts.” He added that the company has observed “very differentiated outcomes” versus downstream approaches across multiple diseases, including GA, GBS and ALS.
GA landscape and vonaprument’s design
Love argued that the GA field has evolved from viewing the disease as neurodegeneration to focusing heavily on retinal pigment epithelium (RPE) structure—an emphasis he said was enabled by imaging tools such as fundus autofluorescence. While he said regulators’ willingness to consider RPE-related biomarkers helped attract investment and development, he contended that RPE-focused approaches have not demonstrated functional vision preservation and that European regulators have not approved drugs on RPE structure alone.
Love described vonaprument as a 50 kDa Fab fragment derived from the company’s full-length monoclonal antibody tanruprubart, which he said has been “well-tolerated” in programs including GBS, Huntington’s disease, and ALS. He said vonaprument is “non-PEGylated with low viscosity and high potency,” and noted its 5 mg dose administered in 25 μl, which he said is “about a quarter of the size” of approved GA drugs that are typically delivered at about 100 μl. Love also said Annexon demonstrated C1q target engagement in the eye by measuring aqueous humor prior to initiating phase II.
Phase II results and the phase III ARCHER II design
Needham senior analyst Joey Stringer noted that the phase II ARCHER study missed its 12-month primary endpoint on lesion growth but showed improvements across visual measures. Love said the trial showed a trend on lesion growth that strengthened in the second half of the study, which he attributed to Annexon’s “top-down” strategy of protecting photoreceptors before downstream RPE preservation becomes more evident.
On vision-related outcomes, Love said the phase II study demonstrated “significant preservation” of photoreceptors across the macula, with more pronounced protection in the central retina. He said the study also met a key pre-specified secondary endpoint: best-corrected visual acuity (BCVA) 15-letter loss. Love said the company reduced the risk of 15-letter loss at month 12 by 73% and also saw protection in low-light visual acuity measures. He highlighted the study’s six-month off-treatment follow-up, saying that when dosing stopped, patients began to lose vision again rather than “rebounding” immediately, which he viewed as supporting a biological treatment effect.
For the ongoing phase III study, Love said ARCHER II includes monthly dosing versus sham with 2:1 randomization and a primary endpoint at 15 months. He said the primary endpoint is BCVA 15-letter loss at month 15, which he called the first GA phase III program he is aware of with vision preservation as the primary endpoint and said is aligned “across the globe” with regulators. He also described two enrichment changes from phase II: a baseline BCVA cutoff of 45 letters and enrolling roughly half foveal patients, which he said have more aggressive disease.
On powering, Love said the company has not disclosed specific figures but characterized the study as “really well-powered,” drawing assumptions from the phase II trial and Roche’s lampalizumab natural history dataset. He said Annexon took a conservative approach on sham and treatment event-rate assumptions. Love also said the company is tracking event rates in a blinded fashion and is “on top of our targeted event rates,” adding that compliance has been high and withdrawals low in a population with an average age around 80. He said the DSMB has been meeting regularly and that “so far everything has gone really well.”
GBS regulatory path and ANX1502 update
In GBS, Love emphasized the severity of the disease and said Annexon’s phase III study was placebo-controlled and “highly statistically significant” on the primary endpoint and other measures. He noted that there are no approved therapies for GBS.
Love said the company filed a marketing application in Europe and is conducting the FORWARD study in the U.S. and Europe to provide additional PK/PD and efficacy data requested by the FDA to support a BLA. He said the company expects to submit the FDA package this year with FORWARD data included, while noting that final outcomes depend on FDA review.
For ANX1502, Love said the company is dosing patients in a proof-of-concept study in cold agglutinin disease and expects to provide a data update this year. He said the program has shown a food effect in which taking the drug with food reduces exposure, and that current patients are being dosed in a fasted state. He said an ideal profile would be twice-daily dosing with normalization of complement markers and objective measures such as bilirubin, alongside acceptable safety and tolerability. Love added that the company may pursue antibody-mediated diseases where the classical pathway is a key effector, citing myasthenia gravis and CIDP as potential starting points.
Love also said the company ended 2025 with about $238 million in cash, which he expects to fund operations well into the second half of 2027 and through anticipated catalysts including the GA phase III readout, ANX1502 proof-of-concept data, and potential GBS approvals in Europe and the U.S.
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.
