The author is an analyst for NH Investment & Securities. He can be reached at pk.park@nhqv.com -- Ed.
Gene cell therapy approaches are gradually gaining renewed attention in response to recent CRISPR and CAR-T news. GCT received keen attention in 2H20 as a viral vector and mRNA-based coronavirus vaccine, but interest declined from 2021 due to poor actual performance. We advise focusing upon anticpated new market expansion in 2024.
Cell therapy product performance stengthening—important market expansion factor
Looking at global performance in Dec 2023, expectations for gene and cell therapy look to be on the rise. Following first-ever FDA approval of a CRISPR-Cas9 system drug, the chances of additional related agreements have elevated alongside news of a Vertex-Editas deal. Boasting new CRISPR-integrase technology, I-PGI technology player Tom Bio has attracted US$200mn worth of investment. The future should contain efforts to broaden target areas and to minimize off-target side effects versus existing CRISPR-Cas9-produced drugs. Meanwhile, a recently-inked MSD-C4 deal is to bring attention to the field of protein degraders and antibody conjugates.
CAR-T developments are also increasingly in the news. Having recently acquired Gracell for US$1bn, AstraZeneca (AZ) now has GC012F (a BCMA-CD19 dual target CAR-T) under its umbrella. Going forward, we advise keeping an eye on AstraZeneca’s efforts to expand to platforms of the same type. In the pursuit of allogeneic CAR-T, Roche-Poseida has unveiled BCMA target phase 1b data. The high-dose group revealed promising results, including an ORR of 82%, no rejection in terms of safety, and no major adverse reactions at the Grade-2-or-lower level. While the FDA's heightened (since end-November) bar of safety for cell therapy products is being met, concerns have emerged that efficacy dependent on patient cohort with lymphocyte removal may be less sustainable. Given such, it will be important to monitor for yet-to-be-released sustainability data becoming public.
Attention to be on large players’ market views
Conservative industry guidance for 2024 started to be reflected in Oct 2023. Having upped recently, the bio index reflects: 1) bio returns (which trailed market hopes through into end-October); and 2) expectations for earnings for 2H24 and onwards via deal expansions. That said, as big pharmas have yet to change their market views sharply, profit-taking looks likely. We advise paying attention to big pharmas and whether they change market views more positively at the upcoming JP Morgan conference in January.