All treatment groups in Vertex Pharmaceuticals Inc.’s phase II study of the non-opioid VX-548 showed meaningful reductions in pain from baseline in treating diabetic peripheral neuropathy (DPN), pointing the company toward pivotal phase III studies and prompting analysts to applaud enthusiastically.
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.
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.
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.”
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.
Injecting a combination of cisplatin and vinblastine – engineered by way of the company’s Dfuserx platform – turned out to be just the ticket for early stage breast cancer in the phase II study called Invincible conducted by Intensity Therapeutics Inc. Shares of the Shelton, Conn.-based firm (NASDAQ:INTS) closed Dec. 8 at $6.88, up $2.68, or 63%, having traded as high as $11.44, thanks to Wall Street’s satisfaction with the data testing INT230-6, which emerged during the San Antonio Breast Cancer Symposium.
Three giants have produced new phase III study data for their already approved, big name therapies. Two were positive and the third was stopped for futility.
While early stage and involving a relatively small patient population, the interim phase Ib readout from the combination cohort testing estrogen receptor (ER)-targeting candidate vepdegestrant in combination with CDK4/6 inhibitor Ibrance (palbociclib) in heavily pretreated patients with ER-positive/HER2-negative breast cancer was impressive enough to prompt partners Arvinas Inc. and Pfizer Inc. to expand development work on the program. The results also struck a chord on the Street, with shares of Arvinas (NASDAQ:ARVN) gaining 31% on the day.
Pharvaris NV’s phase II top-line data from the Chapter-1 trial testing oral bradykinin B2 receptor antagonist deucrictibant in hereditary angioedema (HAE) wowed Wall Street and sparked speculation regarding where the drug might fit in the increasingly competitive landscape.
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