VBL Therapeutics Inc.’s phase III study of its gene therapy, ofranergene obadenovec, in treating ovarian cancer missed its primary endpoints, prompting the company to discontinue the trial and investors to pull way back. Shares of the Tel Aviv, Israel, and New York-based company’s stock (NASDAQ:VBLT) plunged 79% on July 20 to close at 43 cents per share. The $1.62 per share drop in value made it a penny stock. VBL shares hit their high on Nov. 8, closing at $2.53 each. Top-line data for ofra-vec, also known as VB-111, showed no statistically significant improvement in progression-free survival or overall survival.
Japan’s Kyowa Kirin Co. Ltd. has stopped development of its adenosine A2a receptor antagonist, KW-6356, in Parkinson’s disease. Discovered internally by Kyowa Kirin, KW-6356 was studied in phase II trials in Japan for Parkinson’s disease, with results showing it was “potentially effective in relieving motor and non-motor symptoms both as a monotherapy and in combination with levodopa-containing therapy,” Kyowa said. However, development is now being discontinued after conducting an evaluation of the global regulatory landscape, development hurdles and potential timelines for market entry.
New interim data from Ultragenyx Pharmaceutical Inc. and Genetx Biotherapeutics LLC’s phase I/II study of GTX-102 in Angelman’s syndrome didn’t do much to bolster investor confidence, as Ultragenyx shares sagged considerably July 19. The Novato, Calif.-based company’s stock (NADAQ:RARE) closed 13% downward at $52.89 per share. That is nearly half the price shares fetched in late August. At $102.40 per share, that was the stock’s highest value in the past 12 months.
There is no drug that will halt the inevitable process of getting older each year. But biopharmaceutical research can have a positive impact on preventing diseases that come with aging, thereby extending life for the masses, and more importantly, extending quality of life. Part one of BioWorld’s multipart series on extending the human lifespan looks at the increasing development and investment in the space.
The data safety monitoring board overseeing Contrafect Corp.’s phase III study of exebacase, a hydrolase stimulator for treating Staphylococcus aureus bacteremia, recommended the study be stopped. In an interim analysis, the board said the conditional power of the study was below the prespecified threshold for futility.
The U.S. FDA has given Sonalasense Inc. a green light to conduct two clinical trials in life-threatening brain tumors using sonodynamic therapy (SDT) and SONALA-001, an intravenous proprietary formulation of 5-aminolevulinic acid, or ALA. The studies – in diffuse intrinsic pontine glioma (DIPG) and recurrent glioblastoma (rGBM) – mark the first time SDT will have been used in clinical trials.
Atara Biotherapeutics Inc.’s eagerly awaited update on the phase II Embold study testing ATA-188 in progressive multiple sclerosis (MS) left investors scratching their heads, and shares (NASDAQ:ATRA) closed at $3.89, down $4.77, or 55%.
Lenzilumab, Humanigen Inc.’s lead candidate, undershot statistical significance on the primary endpoint in the U.S. NIH-sponsored ACTIV-5/BET-B study of treating hospitalized COVID-19 patients. The Short Hills, N.J.-based company’s stock (NASDAQ:HGEN) crumpled in the wake of the results.
Aldeyra Therapeutics Inc. CEO Todd Brady said that, with new data from a crossover trial with reproxalap in dry eye disease (DED), the question of “approvability has been put to bed,” and the company plans a pre-NDA meeting with U.S. FDA in the third quarter of this year.
Areteia Therapeutics Inc. has launched to put its lead candidate, dexpramipexole, an oral treatment for eosinophilic asthma, through a phase III trial. Areteia was created by Knopp Biosciences LLC, which has put the small-molecule eosinophil maturation inhibitor through a phase II study, and also by Population Health Partners. Areteia has both the development and commercial rights to dexpramipexole.
RSS
