Cartesian Therapeutics has announced updated efficacy and safety data from the Phase IIb trial of its mRNA cell therapy candidate for generalised myasthenia gravis (MG), Descartes-08. The results demonstrated durable responses at month 12.
The double-blind, placebo-controlled, crossover trial (NCT06038474) was open-label and included 36 heavily pre-treated, highly symptomatic patients, half of whom received a placebo with the other half receiving Descartes-08. Both were administered as six weekly outpatient infusions without preconditioning chemotherapy.
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Cartesian’s study identified that Descartes-08-treated patients experienced deepening responses over time.
At month four, the 12 treated patients included in the primary efficacy dataset reported an average 5.5-point reduction on the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale. In the same month, particularly deep responses were observed in the seven patients who had no prior exposure to biologic therapies.
By the sixth month, responses were observed to further deepen in four out of 12 patients in the primary efficacy dataset, and four out of seven patients without prior exposure to biologic therapies.
Responses were observed to be durable through month 12, and four of the five patients from the primary efficacy dataset evaluable for their month-12 assessment maintained a clinically meaningful response, defined as a reduction in MG-ADL score of at least two points.
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By GlobalDataTwo participants with no prior exposure to biologic therapy had reached their month-12 evaluation. Both maintained a clinically meaningful response, according to Cartesian, with one continuing to demonstrate minimum symptom expression.
Cartesian also announced that Descartes-08 demonstrated a safety profile supporting outpatient administration.
The updated data follows Cartesian Therapeutics’ announcement in July that the trial had met its primary endpoint. Ten of 14 patients achieved at least a five-point improvement in their MG-ADL score while two patients were lost to follow-up before the release of updated Phase IIb results. In the placebo group, only three in 12 achieved the same level of improvement.
Following the positive results, Cartesian Therapeutics is now planning its pivotal Phase III study: AURORA, which is on track to commence in the first half of 2025. It will assess approximately 100 patients with acetylcholine receptor autoantibody-positive (AChR Ab+) MG.
“Current standards of care are associated with broad immunosuppression and limited efficacy, and patients with MG, which is a rare and incurable autoimmune disorder, are in dire need of new treatment options,” said James F Howard Jr, professor of neurology, medicine, and allied health at the University of North Carolina School of Medicine and investigator in the Phase IIb trial.
“These updated results continue to show deep responses durable for months after cessation of treatment in both heavily pre-treated participants and, notably, participants without prior exposure to biologic drugs, underscoring the potential for Descartes-08 to emerge as a meaningful addition to available MG treatments.”
The primary endpoint of Cartesian’s AURORA trial will be to assess the proportion of Descartes-08-treated patients that demonstrate an improvement of over three points on the MG-ADL scale at month four, relative to placebo. Secondary endpoints will be the proportion of patients with a reduction of four points or more on the MG Composite score, as well as improvements in other MG severity scales. It will also assess safety and tolerability.
