Urogen Pharma Ltd. shares (NASDAQ:URGN) closed June 13 at $17.50, up $4.78, or 37%, on word of an 82.3% 12-month duration of response data by Kaplan-Meier estimate from its phase III Envision study with UGN-102. The finding was made in in low-grade, intermediate-risk, non-muscle invasive bladder cancer patients who achieved complete response at three months after the first instillation of the drug for intravesical solution.
Evercore ISI analyst Umer Raffat recently called orexin a “red hot neuropsychiatry target,” and the recent Sleep 2024 meeting in Houston bolstered such a view. Also known as hypocretin, the neuropeptide orexin is known to play a crucial role in regulating wakefulness, arousal, and appetite. It’s made in the hypothalamus, and was discovered in the late 1990s. Investigators found that people with narcolepsy can show a deficiency of orexin due to the loss of neurons.
After the phase IIa failure at lowering intraocular pressure to a statistically significant degree with SBI-100, Skye Bioscience Inc. is dropping work with the ophthalmic emulsion, meant to treat primary open-angle glaucoma or ocular hypertension. Resources are turning to the firm’s metabolic program, which includes nimacimab, targeting the cannabinoid 1 receptor, due to start a phase II trial in obesity during the third quarter of this year.
Phase II-stage Rapport Therapeutics Inc. began trading on Nasdaq June 7 under the ticker RAPP after pricing its IPO of 8 million shares at $17 each to raise $136 million, gaining $3.80, or 22.4%, to close its first day at $20.80. With offices in Boston and San Diego, Rapport is developing drugs for central nervous system (CNS) disorders. The IPO is expected to close June 10.
Trophoblast cell-surface antigen 2 (TROP2) antibody-drug conjugates became a topic of talk during the recent American Society of Clinical Oncology meeting in Chicago.
An expert in hepatitis delta virus said Vir Biotechnology Inc.’s phase II findings with the monoclonal antibody tobevibart and the small interfering ribonucleic acid elebsiran in chronic disease could lead to a “change [in] the entire care cascade and paradigm.” Shares of Vir (NASDAQ:VIR) ended June 5 at $12.66, up $2.08, or 19.7%, on the clinical news.
Bridgebio Pharma Inc.’s data from the phase II study with infigratinib in achondroplasia “swings the pendulum of debate sharply in favor” of the firm, competing with Biomarin Pharmaceutical Inc. in the space, Leerink analyst Mani Foroohar said in a report.
While Servier Pharmaceuticals LLC awaits this summer’s PDUFA date for the IDH-mutant glioma drug vorasidenib, acquired in the buyout of oncology assets from Agios Pharmaceuticals Inc., the latter unveiled positive data from a global phase III study with oral mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia.
As the company began a rolling NDA submission to the U.S. FDA for its drug combo in low-grade serous ovarian cancer, Verastem Oncology Inc. popped the lid off phase I/II data in pancreatic tumors, but Wall Street seemed uncertain about the news. Boston-based Verastem disclosed upbeat outcomes from the Ramp 205 study testing the RAF/MEK clamp avutometinib when paired with focal adhesion kinase inhibitor defactinib in combination with gemcitabine plus nab-paclitaxel as first-line therapy for metastatic pancreatic cancer.
Shares of Dyne Therapeutics Inc. (NASDAQ:DYN) closed May 20 at $35.38, up $7.70, or 28%, on word of positive data from the phase I/II Achieve trial of DYNE-101 in myotonic dystrophy type 1 (DM1) and the phase I/II Deliver effort with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. CEO John Cox, who joined Waltham, Mass.-based Dyne eight weeks ago, said he “couldn’t be more proud to be part of this team.” Studies are ongoing, but new data with regard to DM1 as well as DMD showed a “compelling” impact, Dyne said, plus satisfying safety profiles.
