The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.
With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.
Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.
Autolus sold $150 million worth of its stock immediately after the readout, as part of a previously filed shelf registration. It is also collecting $70 million in milestone payments from Blackstone per a $250 million collaboration and financing deal they struck late last year.
The pivotal Phase II trial, dubbed FELIX, enrolled patients with relapsed/refractory adult acute lymphoblastic leukemia.
Jae Park, a principal investigator at Memorial Sloan Kettering Cancer Center, called it an “underserved disease setting” characterized by “the explosive growth of the leukemia and the poor condition of many patients.”
Out of 50 patients evaluated for efficacy, 70% went into either complete remission or complete remission with incomplete blood count recovery, as assessed by an independent response review committee. Among this group, obe-cel showed “comparable expansion and initial persistence” to a previous Phase I study at median follow-up of 6.4 months, Autolus reported, suggesting consistent data.
When researchers look at all 92 patients who have been dosed so far, 3% experienced grade 3 or higher cytokine release syndrome, and 8% experienced grade 3 or higher immune effector cell-associated neurotoxicity syndrome (ICANS), common side effects associated with CAR-T therapy. All told, 23% of patients experienced any grade ICANS.
“We look forward to supplementing this interim data with longer follow up data to more fully explore the clinical benefit of obe-cel,” said Itin, who leads a crew scattered across London, Munich, Basel and Rockville, MD.
Shares of the company, though, plummeted 27% to $2.17 in pre-market trading, which Mizuho analyst Mara Goldstein chalked up to the public offering. She also noted overall concerns about the commercial CAR-T market.
Obe-cel is designed to hit CD19, a familiar target in the CAR-T space, but Autolus believes that its engineering approach can limit T cell activation, keeping the immune system from going haywire while also preventing T cell exhaustion.
In the r/r ALL setting, it will be going up against Gilead’s Tecartus, which posted an ORR of 65% among 54 patients at median follow-up of 12.3 months.
Autolus plans to present more details at a conference in mid-2023.
Credit: Source link