Annexon (NASDAQ:ANNX) President and CEO Doug Love outlined the company’s late-stage pipeline and regulatory plans during a presentation at the Bank of America Healthcare Conference, highlighting upcoming data in geographic atrophy and regulatory filings for Guillain-Barré syndrome.
Love described Annexon as a neuroimmunology company focused on the classical complement pathway, specifically C1q, which he said is the initiator of that pathway. He said the company’s approach is differentiated from other complement-targeting strategies because C1q “localizes and binds to disease tissue” across neuroinflammatory diseases affecting the body, brain and eye.
Geographic Atrophy Data Expected in Q4
Love said Annexon’s geographic atrophy program, which uses intravitreal administration of vonaprumet, is in a 659-patient Phase 3 study expected to read out in the fourth quarter. The study is double-masked and randomized 2-to-1 between the treatment arm and sham arm, using a 5 mg monthly dose.
According to Love, the top-line results are expected to include multiple measures of visual acuity, as well as a key structural measure: photoreceptor protection as measured by ellipsoid zone, or EZ, analysis through OCT.
Love said Annexon believes its mechanism could show a visual acuity benefit because C1q acts earlier in the disease process than downstream complement targets. He said C1q binds to photoreceptor synapses and neurons that are responsible for receiving light, driving their removal before retinal pigment epithelial, or RPE, lesion growth becomes evident.
“The downstream approaches are targeting RPE cells after you’ve already lost your vision,” Love said. “Whereas by blocking C1q, you are protecting your photoreceptor synapses and cells.”
Love said the company’s base case is success on the study’s primary endpoint, best-corrected visual acuity, specifically 15-letter loss. He also noted that the trial includes key secondary measures such as OCT and low-luminance visual acuity.
Company Sees Commercial Opportunity in Earlier GA Treatment
Asked about market uptake for currently approved geographic atrophy therapies, Love said he believes adoption has been limited because existing drugs have not demonstrated visual benefit. He said about 80% of patients in the GA market are not currently treated with available therapies, and that among treated patients, roughly one-third to one-half discontinue treatment within a year.
Love said Annexon expects that vision preservation, if confirmed in Phase 3, could drive uptake, particularly in earlier-stage disease. He pointed to Phase 2 data in which, he said, zero out of 56 patients with earlier neurodegeneration defined by low-luminance visual deficit lost vision, compared with about 17% in the sham group.
On safety, Love said Annexon has not seen the same types of events reported with some downstream complement approaches. He attributed that to the drug candidate’s characteristics, including that vonaprumet is a 50 kDa Fab fragment, non-pegylated, low viscosity and administered in a 25-microliter volume. He also said the mechanism blocks only the classical pathway, while allowing the lectin and alternative complement pathways to continue immune functions in the eye.
Guillain-Barré Syndrome Filing Underway in Europe
Love also discussed Annexon’s Guillain-Barré syndrome program, calling the disease the leading cause of acute neuromuscular paralysis. He said Annexon has completed a pivotal Phase 3 study that showed improvement within one week and durability to six months and beyond.
The company has filed for approval in Europe, and Love said Annexon has had “really good engagement” with European regulators, including initial questions and the beginning of inspection activity at sites in Southeast Asia. He said European regulators have significant familiarity with Guillain-Barré syndrome, citing multiple institutions and studies in the region.
Love said current treatment in Europe and the United States largely involves intravenous immunoglobulin, or IVIG, and plasma exchange. He said about 80% of patients receive IVIG and about 20% receive plasma exchange. IVIG is typically given over five days, while plasma exchange can involve 10 days of treatment, he said. By contrast, Love said Annexon’s therapy is designed as a single infusion administered over a matter of hours.
On European commercialization, Love said Annexon has begun country-level reimbursement discussions focused on disease burden and health economics. He said the company has conducted extensive health economics work in Guillain-Barré syndrome and has presented or plans to present related data at medical meetings.
U.S. Filing Still Expected This Year
Love said Annexon also expects to file for U.S. approval this year, though he noted the company is watching recent changes at the FDA. He said the FDA’s neurology division is familiar with the program after more than 10 interactions during development.
Because Annexon’s pivotal studies were conducted outside the U.S. and Europe, Love said the company must demonstrate both substantial evidence of effectiveness and generalizability to Western patients. He said Annexon conducted a real-world evidence analysis matching Phase 3 patients to a 2,000-patient natural history data set from countries including the U.S., Europe and Bangladesh.
The company is also running an open-label U.S. and European bridging study called FORGE. Love said the study is intended to show that patients in the U.S. and Europe progress similarly to patients in Southeast Asia and that outcomes with Annexon’s drug are similar, with a focus on pharmacokinetic and pharmacodynamic data.
Love described the U.S. market as an incidence-based rare disease opportunity, estimating about 8,000 U.S. patients and about 15,000 European patients develop Guillain-Barré syndrome each year. He said 90% to 95% of patients are currently treated with IVIG or plasma exchange.
Small Molecule Program Used as Proof of Concept
Love briefly discussed ANX1502, Annexon’s small molecule program targeting the classical complement pathway. He said the company is studying it in cold agglutinin disease as a “tool indication” because the disease allows objective measurement of hemolysis through bilirubin and complement levels.
Love said Annexon does not plan to advance commercially in cold agglutinin disease. If the proof-of-concept data are favorable, he said the next step could be a late-stage study in a neuromuscular disease such as myasthenia gravis or chronic inflammatory demyelinating polyneuropathy. He also said the company has observed a food effect with the current formulation and will determine whether reformulation is needed before advancing.
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.
This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.
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