BioNTech secures new revenue with FDA approval of Autolus' CAR-T for ALL

The FDA has granted approval for Autolus' CD19-targeted cell therapy, Aucatzyl (obecabtagene autoleucel), marking it the second CAR-T therapy authorized for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). This decision is based on results from the Phase Ib/II FELIX trial, which demonstrated that 42% of the 65 ALL patients who received the therapy achieved a complete response (CR) within three months. The median duration of CR was noted to be 14.1 months.

Further data shared by Autolus at the American Society of Clinical Oncology (ASCO) meeting showed a broader study involving 127 patients, revealing an overall response rate of 78% for Aucatzyl. Currently, the only other CAR-T therapy approved by the FDA for adult ALL patients is Gilead's Tecartus (brexucabtagene autoleucel). Additionally, Novartis' Kymriah (tisagenlecleucel) was approved in 2017 but is limited to ALL patients up to 25 years old.

This regulatory approval is also notable for BioNTech, which invested $50 million earlier this year to secure royalty rights on Aucatzyl. The biotech firm, known for its COVID-19 vaccine Comirnaty, has been positioning itself to expand into oncology leveraging the revenues from its vaccine success.

In terms of safety, like other CAR-T cell therapies, Aucatzyl includes a boxed warning for potential severe side effects such as cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and T cell malignancies. Autolus reported that severe (Grade 3 or higher) CRS and ICANS occurred in 3% and 7% of patients treated with Aucatzyl, respectively. Common adverse reactions, affecting more than 20% of patients, included symptoms like musculoskeletal pain, fever, nausea, diarrhea, and fatigue.

Meanwhile, Gilead is optimistic about its new BCMA-targeting CAR-T therapy, anitocabtagene autoleucel (anito-cel), which might circumvent some of the neurotoxicities associated with CAR-T therapies like ICANS. According to an abstract released before the American Society of Hematology (ASH) meeting in December, none of the patients who received anito-cel experienced delayed neurotoxicities such as parkinsonism, cranial nerve palsies, or Guillain-Barré syndrome.

In summary, the FDA's approval of Aucatzyl for treating adult ALL patients is a significant advancement in CAR-T cell therapies. With promising response rates and a safety profile that is being closely monitored, Aucatzyl provides a new therapeutic option for patients who have relapsed or are refractory to traditional treatments. This decision not only enhances treatment possibilities for ALL but also represents a strategic development for companies like BioNTech that are expanding their focus into oncology.