Chinese player Legend Biotech reached a new turning point last month when its Johnson & Johnson-partnered Carvykti became the first BCMA-targeted CAR-T cell therapy to be approved by the FDA for second-line treatment of multiple myeloma. From its original roots in China to its first FDA approval in 2022, the company has remained uniquely focused on CAR-T cells targeting the BCMA protein.


Humble Beginnings and a Breakthrough Approval

Back in 2014, a team of Chinese scientists began investigating cell therapies for cancer under the banner of the “Legend Project,” operating in what the company calls “a room the size of a freight elevator.”

After making the decision to focus its research solely on chimeric antigen-receptor T-cells (CAR-T) targeting the BCMA protein in 2015, Legend began conducting its first clinical trials in 2016.

The pioneering biotech landed its first major vote of confidence from the global life sciences community in 2017, landing a partnership with Janssen (now Johnson & Johnson Innovative Medicine). And, just five years after revealing initial clinical data for its BCMA-targeting CAR-T therapy, Legend became a commercial stage operation when the FDA approved Carvykti for the treatment of multiple myeloma after four previous therapies.

Having set up its headquarters in the US and built manufacturing capacity, Legend launched the drug, achieving USD 500 million in sales in Carvykti’s first year on the market.

“With worldwide sales of half a billion dollars in its first full year of commercialization, our rapid, successful launch of Carvykti reinforces its position as a leading CAR-T therapy for patients with relapsed and refractory multiple myeloma,” said Legend CEO Ying Huang. “Our accomplishments in 2023, through our strategic partnership with Johnson & Johnson, created the foundation for strong growth and uptake of Carvykti, positioning us to bring Carvykti to more patients in need of treatment going forward.”


Manufacturing Bottleneck

Producing enough of the newly approved therapy soon became an issue. Due largely to a global shortage of the viral vectors needed to deliver cell and gene therapies, Legend and J&J struggled to meet the strong demand for Carvykti. To deal with the bottleneck, the companies reached out to fellow cell therapy specialist Novartis and last year signed a tech transfer and manufacturing deal for the therapy.

At the same time the partner companies ramped up their own production capacity, announcing that they would double investments in their Raritan, New Jersey facility. A new manufacturing site in Ghent, Belgium has also been built with clinical production beginning last year and in its 2023 full year results report, Legend said it planned to deliver production capacity of 10,000 annual doses by the end of 2025.


Major CAR-T Deal with Novartis

Legend continues to attract attention in the cell and gene therapy arena and its manufacturing partner Novartis last year paid up USD 100 million for a selection of its promising CAR-T candidates, including LB2102 for lung cancer.

Novartis, a cell therapy expert in its own right, set out to develop the candidates with the T-Charge CAR-T cell therapy manufacturing platform it has already used for two of its in-house candidates and which aims to reduce the need for extensive culture time outside the body.


Bumped up to Second-line Status

In the biotech’s next big milestone, last month Legend and its partner J&J broke into the earlier treatment of multiple myeloma when Carvykti became the first and only BCMA-directed therapy for patients who have received one prior line of therapy.

“The expanded label of Carvykti has the potential to transform the treatment paradigm for multiple myeloma by providing patients and physicians with a personalized immunotherapy that can be used earlier in the treatment regimen. Multiple myeloma is an incurable and progressive hematologic cancer that causes patients to relapse and become refractory to treatment, so new, innovative options are desperately needed,” said Huang about the new approval.