Trial results for novel CAD treatment now expected early 2025
Annexon's ongoing proof-of-concept study testing ANX1502 in patients
The results from Annexon Biosciences‘ ongoing proof-of-concept clinical trial of ANX1502, its novel treatment candidate for cold agglutinin disease (CAD), are now expected in early 2025, the company has announced.
That trial is testing a new tablet formulation of the oral therapy in people with CAD.
Earlier this year, when the trial was still being planned, Annexon said it hoped to have results by the end of this year. With the trial now underway, the developer says it is expecting those initial results in the first quarter of the new year, meaning data should be available by the end of March.
“We’re anticipating data from our first-in-kind oral C1s [suppressor] program, ANX1502, with an improved tablet formulation in patients with CAD in the first quarter of 2025,” Douglas Love, president and CEO of Annexon, said in a company press release announcing its latest financial results and anticipated milestones.
Developer changed formulation for CAD treatment to oral tablets
CAD is an autoimmune disease marked by the production of self-reactive antibodies that attack red blood cells at low temperatures. This abnormal immune attack activates a group of immune proteins called the classical complement pathway that ultimately drive red blood cell destruction, or hemolysis, in CAD.
ANX1502 is designed to specifically block the active form of a complement protein called C1s, which together with the C1q complement protein triggers classical pathway activation. The oral therapy is therefore expected to suppress the classical complement pathway, prevent hemolysis, and lessen disease symptoms.
Annexon’s treatment candidate originally was created in a liquid formulation, but the company revamped ANX1502 into a tablet designed to “enhance the patient experience and provide improved tolerability,” according to the release. The new tablet version is meant to be taken twice per day.
Studies in healthy volunteers have suggested that this new formulation has safety and pharmacological properties similar to, or better than, the original liquid version.
In the ongoing CAD study, participants are taking the ANX1502 oral tablet twice a day for up to four weeks. The study’s main goals are to assess the treatment’s safety and its pharmacological properties, including its effects on objective markers of complement activation and hemolysis.
Annexon did not provide details about how many people the trial has enrolled or where it is being conducted. The company did say that, if results are positive, this proof-of-concept study is expected to form the basis for further investigations of ANX1502 — not just in CAD, but also in other autoimmune disorders where abnormal activation of the complement cascade plays a central role in driving disease.
“Our portfolio of programs has provided consistent support for our approach to target C1q, a key upstream driver of neuroinflammatory diseases of the body, brain and eye, and we’re encouraged by the outlook for our programs,” Love said. “We are laser-focused to deliver on our near-term goals and achieve our mission of helping millions of patients live their best lives.”
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