This year’s American Academy of Neurology meeting included a presentation that could one day set a new treatment standard for myasthenia gravis.
After four days in Chicago, the 2026 cycle of the American Academy of Neurology’s annual meeting has wrapped up, featuring some of the newest developments in the field—some of which hold the potential to shape the treatment paradigm in the coming years.
BioSpace rounds up some of the most interesting presentations here.
J&J’s Imaavy maintains myasthenia gravis benefit over 2 years
In patients with generalized myasthenia gravis (gMG), continued treatment with Johnson & Johnson’s FcRn blocker Imaavy keeps disease symptoms at bay and maintains quality of life through two years of follow-up, according to new Phase 3 data released at AAN.
In the Phase 3 Vivacity-MG3 trial, the pharma enrolled more than 150 patients who had insufficient response to standard of care treatments and were given either Imaavy or placebo. Patients entered a 24-week double-blind phase of the study, after which they were transitioned into the ongoing open-label extension portion. Participants stayed on their standard-of-care regimens.
Data presented at AAN cover a total of 120 weeks of observation, J&J said in an April 22 news release, which is “among the longest follow-up periods reported for any FcRn blocker study in gMG.” Results showed sustained improvements on symptoms that affect patients’ daily living and continued reductions in disease severity.
Additionally, nearly 60% of patients on Imaavy were able to cut back on corticosteroid use, with some reaching daily doses of at most 5 mg.
Imaavy was approved in April last year for gMG in patients 12 years and up. J&J is continuing to develop the drug and in February filed an application for its use in warm autoimmune hemolytic anemia. The pharma is also running the first open-label, head-to-head gMG study comparing Imaavy against Argenx’s Vyvgart.
Kyverna’s CAR T therapy sets ‘new efficacy standard’ in myasthenia gravis
All patients treated with Kyverna Therapeutics’ CAR T candidate mivocabtagene autoleucel (miv-cel) saw “clinically meaningful” improvements in symptom severity, according to a Phase 2 gMG readout at AAN on Monday.
“We believe miv-cel is differentiated from current therapies and other late-stage assets by its magnitude of effect and durability, thereby setting a new efficacy standard in the field,” William Blair analysts wrote in an April 20 note. With Kyverna moving quickly toward a filing, the biotech “is poised to have the first FDA-approved CAR-T for the treatment of an autoimmune disease,” the analysts continued.
Miv-cel is an investigational autologous anti-CD19 CAR T therapy designed to stimulate CD28-bearing cells. Data presented at AAN come from seven gMG patients who received a single dose of miv-cel in the Phase 2/3 KYSA-6 study, a registrational single-arm trial. KYSA-6’s primary efficacy measure was improvements to patients’ daily life, as assessed through symptom severity and functional impairment.
Aside from improvements in patient-reported symptom burden, miv-cel treatment also led to deep depletion of B cells in all treated patients, with signs of “immune reset,” according to Kyverna. At week 24, all patients were able to stop treatment with non-steroidal immunosuppressants, high-dose steroids and FcRn and complement blockers.
Kyverna is currently enrolling patients in the Phase 3 portion of KYSA-6, the biotech said on Monday.
Capricor builds case for cell therapy ahead of August decision date for Duchenne
Capricor Therapeutics’ cell therapy deramiocel is currently undergoing FDA review for Duchenne muscular dystrophy, with a decision expected in August. While waiting, the biotech provided more data from its late-stage HOPE-3 trial, touting further functional improvements tied to the asset.
At 12 months, deramiocel slowed the deterioration of upper limb function by 54% versus placebo, as measured by a functional scale that assesses how well patients are able to perform certain tasks. This effect was statistically significant, according to a company presentation.
Using another assessment tool—one that relies on video assessments of how well a patient performs activities of daily living—HOPE-3 found that deramiocel could slow functional decline by 83%.
Deramiocel is composed of a rare subset of cardiac cells that reduce fibrosis and modulate the immune response. In July 2025, the FDA rejected deramiocel’s drug application, noting that it did not satisfy the “statutory requirement for substantial evidence of effectiveness.” Capricor refiled in December that year, boosting its data package with results from HOPE-3.
Praxis clears Phase 3 for essential tremor drug, nearing $3B+ market opportunity
Praxis Medicines is advancing the calcium channel blocker ulixacaltamide for essential tremor. At AAN, the biotech touted significant and durable improvements in activities of daily living. Jefferies analysts told investors that ulixacaltamide could secure FDA approval as early as January 2027 and access a market opportunity exceeding $3 billion at its peak, according to an April 21 note.
Praxis’ presentation at AAN comes after a topline readout in October 2025 demonstrated a 4.3-point improvement on a scale used to measure functional performance across 11 daily activities. Placebo comparators saw a 1.7-point statistically significant improvement.
These data remained unchanged in the AAN presentation, Jefferies noted.
Praxis at the conference also presented results from a withdrawal study, which randomized patients who had responded to ulixacaltamide to continue treatment or switch to placebo. According to Jefferies, 55% of those who stayed on ulixacaltamide remained responsive after 4 weeks, versus 33% of comparators who switched.
Ulixacaltamide is currently under FDA review for essential tremor, with a target action date of Jan. 29, 2027.
