Beam Therapeutics has begun human testing in the U.S. of a first-of-its-kind gene editing medicine for cancer, the company said Tuesday.
Beam, a pioneering developer of a precise gene editing technique known as base editing, said in a short statement that it’s dosed its first patient in a study of the treatment, called BEAM-201. The trial involves patients with an aggressive form of blood cancer known as T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma, or T-ALL/T-LL. It will eventually enroll about 100 participants, according to a federal database.
The study’s start makes BEAM-201 the first base editing therapy to enter clinical testing in the U.S., and marks the first time patients have received a cell therapy made by “multiplex editing,” in which several genes are edited. The edits are designed to eliminate expression of four genes known as CD7, TRAC, PDCD1 and CD52.
Beam claims this approach could lead to a more powerful and durable treatment. In its statement, the company noted BEAM-201’s potential to sidestep a variety of issues associated with cell therapies, like propensity for the modified cells to kill one another, or become weaker as time goes on.
Beam also believes the simultaneous edits could yield a more potent donor-derived, or “off the shelf,” cell therapy. Such allogeneic treatments would be more convenient than the personalized CAR-T therapies on the market, but results to date haven’t proven they’re more powerful at killing cancer cells.
“We believe that the full therapeutic potential of CAR-T therapies, including the ability to utilize an allogeneic source of T cells, will only be unlocked through higher levels of cellular engineering enabled by multiple simultaneous genetic edits,” said CEO John Evans, in a statement.
Yet Beam has struggled to meet development timelines amid Food and Drug Administration scrutiny of base editing, and in some cases is trying to catch more advanced competitors.
In sickle cell disease, the company is behind gene-based medicines from Bluebird bio and Vertex Pharmaceuticals that could be approved later this year. Beam’s “slower than expected” progress enrolling patients in an early study of its sickle cell therapy, dubbed BEAM-101, “drew a disproportionate amount of investor attention/scrutiny” when Beam reported quarterly earnings last month, wrote Stifel analyst Dae Gon Ha.
Testing of BEAM-201 has also been held up. Its first clinical study was supposed to begin last year, but the FDA paused development in August 2022 and asked for a variety of technical information from preclinical experiments before the company could proceed. The clinical hold was lifted four months later.
Beam isn’t alone in dealing with regulatory scrutiny. The FDA has slowed early U.S. tests of several emerging gene editing treatments in recent years, often requesting additional information to allay safety concerns. But the resolution of Beam’s issue and subsequent dosing of a patient could indicate “FDA’s comfort with intricate and novel editing technologies,” wrote Stifel analyst Ha.
Ha believes progress with BEAM-201 “can lay an important foundation” for Beam to progress, as the therapy could demonstrate the impact multiplex editing can have. Still, “we suspect investors also ascribe minimal, if any, value,” to the drug, he wrote.
At about $24 apiece, Beam shares have lost roughly 40% of their value this year.