Autolus outlines path to FDA after CAR-T cell therapy trial hits primary endpoint

Autolus outlines path to FDA after CAR-T cell therapy trial hits primary endpoint

Autolus Therapeutics is almost ready to make a late play for the CD19 CAR-T cell therapy space. The biotech now has phase 2 data showing its asset hit the primary endpoint at an interim analysis and is setting out plans for further data to file for FDA approval next year and try to join Bristol Myers Squibb, Gilead Sciences and Novartis on the market.

Novartis’ Kymriah came to market in 2017, and Gilead won FDA approval for Tecartus in Autolus’ lead indication last year. But, having applied technologies designed to reduce toxicity and T-cell exhaustion to its asset, Autolus is trying to set its therapy apart from the pack and carve out a slice of the market. The company took a step toward that goal with the release of top-line data from its phase 2 FELIX trial after the market closed on Thursday.

At a preplanned analysis of 50 adult patients with relapsed or refractory acute lymphoblastic leukemia, investigators linked the CD19 cell therapy, obe-cel, to a 70% response rate, meaning the study met its primary endpoint.

Across the 92-subject safety data set, 3% of patients had grade 3 or higher cytokine release syndrome and 8% experienced grade 3 or higher immune effector cell-associated neurotoxicity syndrome, both of which are associated with other CAR-T cell therapies.

Autolus did not provide a more detailed breakdown of the primary endpoint and safety data nor share a look at the secondary endpoints. The only other tidbit is that expansion and initial persistence are comparable to the results of the earlier ALLCAR19 study after a median follow-up of 6.4 months.

Having chalked up the primary endpoint success, Autolus now plans to gather longer follow-up data on the cell therapy before filing for FDA approval by the end of next year. The biotech, leveraging the perks of its regenerative medicine advanced therapy designation, met with the FDA earlier in the year. While the FDA has recently taken a tougher line in some areas, Autolus plans to seek approval in an indication covered by an approved CD19 CAR-T cell therapy on the strength of data from a single-arm clinical trial.

Autolus shares fell after the release of the data, although the stock offering, which the biotech disclosed minutes after the trial results, may have driven the decline. By the end of the day, Autolus had confirmed investor fears by pricing a $150 million stock offering at $2. The stock closed at $2.99 before the data drop. Autolus’ shares fell roughly 30% in premarket trading to around $2.10.

The biotech held the stock offering despite the clinical readout strengthening its finances. The study data triggered a $35 million payment from Blackstone Life Sciences, as did the achievement of a manufacturing milestone

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