Cell and gene therapy companies continue moving away from traditional treatment modalities into a future that’s often unclear. A panel of CEOs said at the Biotech Showcase in San Francisco that, instead of aiming for developing silver bullet therapies that knock out indications in a single blow, they tend to only be able to take incremental steps in development.
Blueprint Medicines Corp. shifted away from two early clinical therapies for EGFR-mutant non-small-cell lung cancer and dropped development and commercialization of lung and thyroid cancer drug Gavreto (pralsetinib) for areas outside of the U.S. and greater China, reducing its operating expenses, as it prepares for increasing Ayvakit sales and prioritizes development of other assets.
The zeal for antibody-drug conjugates (ADCs), a prominent focus for dealmaking in 2023, shows no signs of waning, as Johnson & Johnson greeted attendees of the J.P. Morgan Healthcare Conference (JPM) with news of its $2 billion buyout of Ambrx Biopharma Inc., picking up rights to an ADC platform along with a promising candidate targeting advanced prostate cancer. Under the terms, J&J agreed to acquire all outstanding shares of San Diego-based Ambrx for $28 apiece, marketing a 105% premium to the firm’s Jan. 5 closing price. Unsurprisingly, Ambrx’s stock (NASDAQ:AMAM) gained 101.5% to close Jan. 8 at $27.47.
MSD had a banner year at the ESMO Asia Congress earlier this month, presenting 14 abstracts on eight different types of cancers, including gastric, esophageal, colorectal, biliary tract, kidney, urothelial, breast and gynecological cancers. Ten of these studies were focused on Asian-related data. Roche AG, meanwhile, presented Asia-specific results from the phase III Alina study in patients with ALK-positive early stage non-small-cell lung cancer.
Multiple studies at the 65th American Society of Hematology Annual Meeting 2023 have the potential to change the treatment paradigm for first-line treatment of multiple myeloma.
It’s not every day you see a small drug company’s presentations get picked for both the plenary session and the late-breaker session at a conference, but Syndax Pharmaceuticals Inc. managed to do just that at the 65th American Society of Hematology Annual Meeting 2023 – with a little help from a friend.
Katy Rezvani received this year’s E. Donnall Thomas Prize for her work on natural killer (NK) cells at the annual meeting of the American Society of Hematology (ASH). It was not love at first sight, though.
Biomea Fusion Inc.’s diabetes treatment produced enhanced glycemic control at week 26 courtesy of its 200-mg cohort. It’s the latest advance for the company’s candidate that also has strong prospects in treating leukemia. Top-line data from the ongoing phase II Covalent-111 study of BMF-219, a covalent menin inhibitor for regenerating insulin-producing beta cells, demonstrated that about 40% of participants, four of 11 patients, in the 200-mg cohorts showed a durable reduction, 1% or more, in the amount of blood sugar attached to the type 2 diabetes (T2D) patients’ hemoglobin. The data came from participants who had received the last dose in a four-week treatment.
CAR T-cell therapies have worked well at curing blood malignancies, but a group out of the University Hospital of Erlangen have repurposed the technology as a treatment for autoimmune diseases. The expansion into new diseases has required cooperation between multiple departments, with CAR T experts taking the lead on treatment and potential side effects, and rheumatologists measuring the outcomes of the treatment.
Spirits were high at the 2023 annual meeting of the American Society of Hematology (ASH), buoyed by the U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego. The addition of gene therapy to the therapeutic arsenal for SCD is “phenomenal,” Adetola Kassim, director of the Adult Sickle Cell Disease Program and professor of medicine at the Vanderbilt-Ingram Cancer Center, told BioWorld. Nevertheless, at a Saturday, Dec. 9, session titled, “Improving Outcomes for Individuals with Sickle Cell Disease: Are We Moving the Needle?,” which Kassim chaired, the answer remained “maybe.”