Cue Biopharma Inc.’s Kenneth Pienta, acting chief medical officer, called “very strong” the latest phase Ib data with IL-2-based candidate CUE-101 in HPV-positive, recurrent head and neck squamous cell carcinoma (HNSCC), and “by midyear, we're going to have enough data to go forward to talk to the FDA to develop a strategy with them” for a registrational study, likely in third-line patients. Meanwhile, Wall Street sifted through the latest findings from Cambridge, Mass.-based Cue, which closed (NASDAQ:CUE) at $7.99, down $2.61, or 24%. The stock had been posting a gradual run-up for several days.
Sierra Oncology Inc. plans an NDA in the second quarter of this year for momelotinib (MMB), thanks to data from the phase III Momentum study with the drug in myelofibrosis (MF) patients who are symptomatic and anemic after treatment with an approved JAK inhibitor. Momentum, which pitted momelotinib against the androgen danazol, met all of its primary and key secondary endpoints. MMB inhibits JAK1/2 and ACVR1/ALK2. Shares of San Mateo, Calif.-based Sierra (NASDAQ:SRRA) closed Jan. 25 at $22.68, up $7.17, or 46%.
With data due the first half of this year from the phase III trial testing Opdivo (nivolumab, Bristol Myers Squibb Co.) plus Nektar Therapeutics Inc.’s bempegaldesleukin (bempeg) in metastatic melanoma, the possibilities for engineered cytokines such as the latter remain alluring in many quarters.
When, late last year, Daiichi Sankyo Co. Ltd. won breakthrough status from the FDA for HER3-targeting lung cancer prospect patritumab deruxtecan, Wall Street began taking stock of other candidates in the space. “The winning strategy to therapeutically target HER3 remains to be seen,” noted a recent paper in Clinical Cancer Research, “but HER3 is a promising drug target, and the era of drugging the ‘undruggables’ has already started.”
Treatment-emergent adverse events (TEAEs) may have soured investors on otherwise-positive new data from Cardiff Oncology Inc.’s phase Ib/II study with PLK1 inhibitor onvansertib in second-line, KRAS-mutated metastatic colorectal cancer (mCRC). Cardiff, however, is forging ahead with a pivotal experiment.
Targeting VEGF and DLL4 at the same time in solid tumors is an approach that continues to entice researchers, with such firms as Abbvie Inc., Compass Therapeutics Inc. and Oncxerna Therapeutics Inc. working to come up with oncology solutions.
Drug sponsors should not focus on influencing legislators about how to price rare-disease and orphan therapies, Ovid Therapeutics Inc. CEO Jeremy Levin said during a panel talk at Biotech Showcase. “The game is not in Washington,” he said. “The game is on the ground in each state, where you need to convince the public, the individuals, the patients that, in fact, you are bringing value.”
“There isn’t a better place to be” now than in Alzheimer’s disease (AD) drug development, said Phyllis Ferrell, global head of external engagement in AD and neurodegeneration at Eli Lilly and Co., during Biotech Showcase’s panel talk titled, “Aduhelm: Stimulating the Next Generation of AD Treatment.”
Having unveiled more data from the ongoing, global phase III Gener8-1 study with Roctavian (valoctocogene roxaparvovec, also known as valrox), Biomarin Pharmaceutical Inc. remains on track to file a regulatory submission with the FDA in the second quarter of this year for the gene therapy to treat adults with severe hemophilia A. The EMA is already reviewing a marketing authorization application.
Harmony Biosciences Holdings Inc.’s plan disclosed at the start of last December to launch a phase III study with Wakix (pitolisant) for idiopathic hypersomnia (IH) during the first half of this year brought renewed attention to the sleep space, where a handful of players are jostling for position.
