EMA committee endorses Autolus’ leukemia therapy obe-cel

LONDON - Autolus Therapeutics plc (NASDAQ:AUTL), a biopharmaceutical company specializing in programmed T cell therapies with a market capitalization of $407 million, has received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for its leukemia treatment, obecabtagene autoleucel (obe-cel). According to InvestingPro analysis, the company appears undervalued based on its Fair Value metrics, despite its stock showing significant volatility with a 15% gain in the past week. This recommendation is for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL).

The CHMP’s endorsement is based on the results of the FELIX clinical trial, which demonstrated that obe-cel achieved a 76.6% Complete Response/Complete Response with Incomplete Hematological Recovery (CR/CRi) rate in a pivotal cohort of 94 patients. The company maintains a strong liquidity position with a current ratio of 9.24, and InvestingPro data shows it holds more cash than debt on its balance sheet, providing financial flexibility to support its commercialization efforts. The median duration of response was 21.2 months, with median event-free survival (EFS) at 11.9 months. Notably, the treatment was associated with manageable toxicity levels, with cytokine release syndrome occurring in 68.5% of patients, but only 2.4% experiencing grade 3 or higher events.

This recommendation follows the therapy’s approval by the U.S. Food and Drug Administration (FDA) on November 8, 2024, and a conditional marketing authorization by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) on April 25, 2025. The European Commission (EC) is expected to make a final decision on the conditional marketing authorization application (MAA) for obe-cel within approximately two months, which would extend the treatment’s availability to all 27 European Union Member States as well as Iceland, Norway, and Liechtenstein.

Obe-cel is an autologous CD19 CAR T cell therapy, developed by a team led by Dr. Martin Pule at University College London. It is designed to mimic physiological T-cell receptor interactions for potentially improved treatment outcomes.

The positive opinion from the CHMP is significant for adult r/r B-ALL patients in Europe, who currently face poor survival rates with conventional treatments. Approximately 6,000 new cases of ALL are diagnosed annually in Europe, with up to 50% of adult B-ALL patients relapsing after frontline treatment.

Autolus Therapeutics is focused on delivering next-generation T cell therapies for cancer and autoimmune diseases, with obe-cel being its first FDA approved and MHRA licensed product. The company continues to develop a pipeline of product candidates for hematological malignancies, solid tumors, and autoimmune diseases. Analysts anticipate sales growth in the current year, though InvestingPro data indicates the company is not expected to be profitable this year. For deeper insights into Autolus Therapeutics’ financial health and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers, along with 10+ additional ProTips and extensive financial metrics.

This article is based on a press release statement from Autolus Therapeutics plc.

In other recent news, Autolus Therapeutics plc has reported a net loss of $220.7 million for the fourth quarter of 2024, an increase from the previous year’s $208.4 million loss. Despite the losses, the company saw a significant boost in cash reserves, reaching $588 million by the end of 2024, primarily due to a $600 million collaboration with BioNTech. The UK Medicines and Healthcare products Regulatory Agency has conditionally approved Autolus’s AUCATZYL® (obecabtagene autoleucel) for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia, based on the FELIX clinical trial results. Meanwhile, Truist Securities has adjusted its price target for Autolus to $10.00 while maintaining a Buy rating, reflecting a cautious outlook as they await further details on the company’s AUTO4 program. Mizuho Securities continues to support Autolus with a $12.00 price target and an Outperform rating, following the company’s fourth-quarter earnings call. Autolus has successfully authorized 33 U.S. treatment centers for Aucatzyl, surpassing its initial target and potentially covering 60% of the U.S. patient population. The company is also preparing for significant developments in the second half of 2025, including approval decisions for Aucatzyl in the UK and Europe and the release of initial pediatric data.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.