Shares of Cogent Biosciences Inc. (NASDAQ:COGT) sank $4.77 to $3.86, or 55%, despite positive phase II data from the ongoing phase II Summit trial testing bezuclastinib in non-advanced systemic mastocytosis (NonAdvSM) at the American Society of Hematology (ASH) meeting in San Diego. The KIT D816V inhibitor turned up a rapid and continuing improvement in patient symptoms, with 57% median best improvement on Mast Cell Quality-of-Life (MC-QoL). ASH attendees also heard that 78% of NonAdvSM patients reported ≥1 point improvement on Patient Global Impression of Severity by week 20. Investors likely were displeased by the MC-QoL score with Waltham, Mass.-based Cogent’s candidate when stacked against competitor Ayvakit (avapritinib), the tyrosine kinase inhibitor from Blueprint Medicine Corp., first approved by the U.S. FDA in January 2020. Shares of Blueprint (NASDAQ:BPMC) were trading at $80.12, up $6.14.

ASH 2023: For broad reach, meaningful innovation still means small molecules

Spirits were high at the 2023 annual meeting of the American Society of Hematology (ASH), buoyed by U.S. FDA approval of the first two gene therapies for sickle cell disease the day before the conference kicked off in San Diego. The approval is “historic,” Pamela Ting told BioWorld. But it is also clear that with list prices in the millions and a harsh conditioning regimen, only a small percentage of patients will have access to gene therapies for the time being. For sickle cell disease, “we have a global market that we need to address, and we need small molecules to address that,” Betty Pace, professor of pediatrics and Francis J. Tedesco Distinguished Chair of Pediatric Hematology/ Oncology at Augusta University told the audience at Sunday’s plenary scientific session. Pace was introducing Ting’s presentation on upregulating fetal hemoglobin through targeted degradation of Wiz, a transcription factor that represses fetal hemoglobin. 

ASH 2023: CAR T therapies take on autoimmune diseases

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. At the 65th American Society of Hematology Annual Meeting and Exposition, Fabian Müller, Max Elder research group leader and head of the CAR T-cell unit at the hospital, presented data from a clinical trial of 15 patients with systemic lupus erythematosus, systemic sclerosis or idiopathic inflammatory myositis. All the patients responded to a single treatment of a CAR T therapy targeting CD19 with patients remaining in remission for as long as 28 months.

Biomea’s dose escalation in diabetes nearly doubles efficacy

Biomea Fusion Inc.’s diabetes treatment produced enhanced glycemic control at week 26 courtesy of its 200-mg cohort. Top-line data from the ongoing phase II Covalent-111 study of BMF-219, a covalent menin inhibitor for regenerating insulin-producing beta cells, about 40% of participants in the 200-mg cohorts showed a durable reduction, 1% or more, in the amount of blood sugar attached to their hemoglobin. That nearly doubled the number of patients with better results in the 100-mg cohort, which was presented at last week’s World Congress Insulin Resistance, Diabetes and Cardiovascular Disease conference in Universal City, Calif. Biomea’s stock (NASDAQ:BMEA) was taking a beating at midday, with shares down 24% to $12.50 each.

Prescient to advance PTX-100 to phase II for refractory T-cell lymphomas 

Prescient Therapeutics Pty Ltd.’s PTX-100 met primary safety endpoints and showed preliminary efficacy in a phase Ib trial in patients with relapsed and refractory T-cell lymphomas that exceeded the standard of care, and the company hopes to advance to a phase II registrational study in 2024, Prescient CEO Steven Yatomi-Clarke told BioWorld. PTX-100 (previously GGTI-2418) blocks cancer growth enzyme geranylgeranyltransferase-1 (GGT-1). Targeting the Ras pathway directly has proved elusive, and PTX-100 disrupts the Ras pathway downstream by inhibiting post-translational modification of Rho, Rac and Ral. PTX- 100 is believed to be the only GGT-1 inhibitor in clinical development, Yatomi-Clarke said.

BioWorld Insider Podcast: Six biopharma executives consider a tough 2023 and are hopeful for a better 2024

One aspect of 2023 our group of executives completely agreed on: the past year was tough financially. And they all foresee a more vibrant year ahead for the market. Giving all of them hope were technological breakthroughs such as artificial intelligence, game changing weight loss drugs, the surging fascination with ADCs and hope for lower interest rates. But uncertainty looms about the upcoming general election in the U.S. and ground-shifting gene therapies. In a preview of the annual Biotech Showcase conference, an investor conference for private and micro- to mid-cap biotech companies Jan. 8-10 in San Francisco, BioWorld spoke with Dave Bearss, CEO of Halia Therapeutics Inc., Vimal Mehta, CEO of Bioxcel Therapeutics Inc., Paul Lammers, CEO at Triumvira Immunologics Inc., Chris Pirie, COO of HDT Bio Corp., Thijs Spoor, CEO of Perspective Therapeutics Inc., and Shelley Hartman, CEO of Aegle Therapeutics Corp. They offered insights brought about by years of hard-won experience.

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Also in the news

Agenus, Alpha Cognition, Apollomics, Biomea Fusion, Bio-Thera, Bioxcel, Bristol Myers Squibb, Cadence, Camino, Cstone, Dantari, Effector, Eisai, Electra, Eli Lilly, Engeneic, Exegi, Eyepoint, Ferring, G-Niib, Hummingbird, Imunon, In8bio, Intensity, Ionctura, IPA, LSL, Menarini, Merck, Naobios, Novartis, Pfizer, Pharmabiome, Prelude, Respirerx, Springworks, Tilt, Zafrens