Failing to meet the primary endpoint in its confirmatory phase III Embark trial, Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec), which received accelerated approval in June and was priced at $3.2 million, has one of three fates in its future, all of which are dependent on how the U.S. FDA perceives the data. Based on secondary endpoints showing statistical significance and a recent positive meeting with the agency, Sarepta could continue to market Elevidys under its current label for 4- and 5-year-old ambulatory Duchenne muscular dystrophy (DMD) patients; Sarepta is filing the postmarketing requirement needed to transition from accelerated to full approval.
With Deciphera Pharmaceuticals Inc.’s positive top-line data in hand from the pivotal phase III study called Motion, Wall Street speculation turned to the comparative safety and regulatory odds for vimseltinib (which analysts often refer to as “vim”) in patients with tenosynovial giant cell tumor (TGCT) not amenable to surgery.
The pivotal phase III study of brilaroxazine from Reviva Pharmaceuticals Holdings Inc. for adults with schizophrenia hit its primary endpoint and two key secondary endpoints, following positive phase II data in 2021. The serotonin-dopamine signaling modulator, the company’s lead candidate, is a once-daily treatment. Results from the study showed brilaroxazine demonstrated reductions in all major symptom domains for the patients and the secondary endpoints at week 4 when comparing the 50-mg dose with placebo.
Oncolys Biopharma Inc.’s lead compound Telomelysin (suratadenoturev, OBP-301) met the primary endpoint of exceeding the predefined threshold of local complete response, confirming clinical benefit in a phase II study in locally advanced esophageal cancer combined with radiation.
The Congress of the European Society of Medical Oncology (ESMO) held in Madrid from Oct. 20 to 24, 2023, could well add the letter W to its acronym after the four sessions on gynecological cancer that could represent a milestone in the lives of all women who suffer from cervical, ovarian, or endometrial cancer.
Novartis AG’s radioligand therapy, Pluvicto, a bestseller for the company that has been restructuring itself, continues to show strong growth in the third quarter of 2023, according to its most recent financial results. Sales were up 217% from the same quarter in 2022 at $256 million. Analysts are predicting blockbuster sales. Adding to the momentum were new data from the phase III PSMAfore study of Pluvicto (lutetium [177Lu] vipivotide tetraxetan), in second-line cancer, hitting its primary endpoint of radiographic progression-free survival in treating prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer after treatment with androgen receptor pathway inhibitor therapy.
Chimeric Therapeutics Ltd.‘s chlorotoxin (CLTX) CAR T therapy, CHM-1101, which is derived from scorpion toxin, saw a disease control rate of 55%, exceeding the historical disease control rates of 20% to 37% in heavily pre-treated patients with glioblastoma.
Harpoon Therapeutics Inc. provided updated interim monotherapy data from 71 patients in its phase I/II trial testing HPN-328 in small-cell lung cancer (SCLC) and other neuroendocrine tumor types in a poster at the European Society of Medical Oncology Congress (ESMO). Wall Street immediately began comparing the compound, which takes aim at delta-like ligand 3 (DLL3) with a similar prospect from Amgen Inc. that recently yielded promising results.
This summer’s phase III news from Qilu Pharmaceutical Co. Ltd. with iruplinalkib – an oral inhibitor of ALK and ROS1 tyrosine kinase – reinforced an ongoing interest in the pair of oncology targets, where a handful of developers remain busy. Most notably of late is Nuvalent Inc., which rolled out stock-boosting data Oct. 4.
A six-week treatment regimen of Paradigm Biopharmaceuticals Ltd.’s Zilosul (injectable pentosan polysulfate/iPPS) resulted in improvement of both the symptoms of osteoarthritis and preserved or regenerated joint tissues in the knee, according to phase II trial results.
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