RARE Daily

Autolus Raises $220 Million as it Reports Positive Interim Data from Pivotal Trial of CAR-T Therapy in Rare Cancer

December 9, 2022

Autolus Therapeutics, a company developing next-generation programmed T cell therapies, raised $220 million in two financings as it reported positive interim data from a phase 2 pivotal study of its CAR-T therapy obe-cel in relapsed/refractory adult acute lymphoblastic leukemia

Photo: Christian Itin, CEO of Autolus

The company said obe-cel met its primary endpoint based on a pre-planned interim analysis of 50 patients with morphological disease, as verified by an independent data monitoring committee.

At the same time, the company priced a previously announced underwritten public offering in the United States of 75 million American Depositary Shares at a public offering price of $2.00 per ADS, for total gross proceeds of $150.0 million. In addition, Autolus has granted the underwriters a 30-day option to purchase up to an additional 11.3 million ADSs at the public offering price, less underwriting discounts and commissions.

Autolus also said that Blackstone Life Sciences has committed to make two pre-agreed milestone payments of $35 million each to Autolus, totaling $70 million. The first milestone is being paid earlier than anticipated as a result of the joint steering committee’s review of Autolus’ interim analysis of the pivotal FELIX Phase 2 clinical trial. The second Blackstone milestone is a pre-agreed manufacturing milestone as a result of completion of planned activities demonstrating the performance and qualification of Autolus’ obe-cel’s manufacturing process.

These funds stem from a strategic collaboration and financing agreement in November 2021, whereby funds managed by Blackstone agreed to provide up to $250 million in equity and product financing to support Autolus’ advancement of obe-cel, its CD19 CAR T cell investigational therapy product candidate, as well as next generation product candidates of obe-cel in B-cell malignancies.

Obe-cel is a CD19 CAR-T cell investigational therapy may overcome the limitations in clinical activity and safety compared to current CD19 CAR-T cell therapies by being designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, thus potentially reducing toxicity and T cell exhaustion, which could enhance persistence and improve the ability of the programmed T cells to engage in serial killing of target cancer cells.

The primary endpoint for the FELIX phase 2 trial is the Overall Remission Rate (ORR), defined as the proportion of patients achieving Complete Remission (CR) and Complete Remission with Incomplete Blood Count Recovery (CRi) as assessed by an independent response review committee (IRRC).

Based on 50 patients evaluated for efficacy, the ORR for obe-cel was 70 percent. Obe-cel showed comparable expansion and initial persistence (median follow-up 6.4 months) to the data observed in the prior ALLCAR19 study. The safety analysis was based on 92 patients treated with obe-cel and evaluable for safety. The company observed that 3 percent of patients experienced Grade 3 or higher CRS, while 8 percent experienced Grade 3 or higher ICANS, and 23 percent of patients experienced any grade ICANS.

“We believe that we are one step closer to bringing this potentially innovative treatment to an underserved ALL patient population,” said Christian Itin, CEO of Autolus. “We look forward to supplementing this interim data with longer follow up data to more fully explore the clinical benefit of obe-cel, and to work towards the submission of a Biologics License Application (BLA) by the end of 2023 to the U.S. Food and Drug Administration (FDA).”

The trial has completed screening patients for entry into the morphological cohort as the pre-specified goal of approximately 90 patients enrolled has been reached, and Autolus plans to present the results from the FELIX trial at a medical conference in mid-2023, with longer follow up planned to be reported at the end of 2023.

Author: Rare Daily

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