Dive Brief:
- An experimental cancer cell therapy developed by Legend Biotech helped reduce or eliminate signs of disease in people with lymphoma enrolled in a clinical trial, sparking hopes the “in vivo” medicine might compete with personalized, marketed treatments like Novartis’ Kymriah, the company said Tuesday.
- At the higher of the two dose levels tested so far, all six people treated with the therapy, LB2501, responded. Five had no evidence of lymphoma lesions, according to trial details released ahead of a medical meeting.
- Legend is one of many companies seeking to develop therapies that fight cancer by reprogramming immune cells inside the body. If successful, these therapies would sidestep an extensive, “ex vivo” process that involves extracting cells and modifying them in a lab.
Dive Insight:
Legend’s shares jumped 30% Tuesday following the news. Multiple Wall Street analysts praised the data, with RBC Capital Markets’ Leonid Timashev noting that a successful “in vivo” product could record blockbuster sales while “ex vivo” counterparts face “logistical bottlenecks” that limit their commercial potential.
Legend is already a prominent cell therapy player, having successfully developed the personalized multiple myeloma treatment Carvykti that it now sells with Johnson & Johnson. It’s now turning to “in vivo” work, an area that’s seen an explosion in venture investments as well as a string of recent drugmaker acquisitions.
With LB2501, Legend uses a modified virus to deliver into immune cells instructions to find and attack cells expressing the proteins CD19 and CD20. Those proteins are found on the surface of malignant B cells and are known targets in lymphoma. Importantly, the treatment doesn’t require a chemotherapy step to prepare the body for treatment, as other cell therapies do.
The data being reported at the European Hematology Association meeting this month comes from a first-in-human trial conducted in China that’s testing LB2501 in people whose disease had progressed after at least two lines of treatment. An abstract posted ahead of that meeting involves results in 12 patients with large B cell lymphoma, follicular lymphoma or mantle cell lymphoma who received one of two tested doses.
No responses were observed at the lower dose, even though there was evidence of activity in five of the six recipients. At the higher dose, though, remissions — defined as a reduction or elimination of lymphoma lesions — were registered in all six recipients. Five had “complete” responses, Legend said.
Eight of the enrollees had an immune response called cytokine release syndrome that is often seen with cell therapies, but only one needed an intervention beyond basic symptomatic treatments for fever, pain or nausea. No patients had an immune response that affected the brain or nerves, another side effect associated with ex vivo cell therapies.
In his note, Timashev wrote further data, such as a six-month follow-up, will be necessary to see how Legend’s therapy stacks up against others, such as an experimental treatment from Lyell Immunopharma. But the news is nonetheless “exciting both for their clinical value of the product, and strategically, because it puts forth a credible second act for [Legend] beyond Carvykti.”
