Genentech Inc. is paying Kiniksa Pharmaceuticals Ltd. $100 million in up-front and near-term payments for the development and commercialization rights to vixarelimab, a fully human monoclonal antibody. Kiniksa also could receive up to approximately $600 million in certain clinical, regulatory and sales-based milestones, as well as royalties on annual net sales.
After spending a lot of money and putting in seven years of hard work, Astrazeneca plc is discontinuing its phase III Interlink-1 study of monalizumab. Results from an interim futility analysis and an independent data monitoring committee’s recommendation convinced the company to bring it to a halt.
Top-line results from Vistagen Therapeutics Inc.’s Palisade-1 phase III study of PH-94B for treating social anxiety disorder showed the therapy fell shy of the primary endpoint. The South San Francisco-based company’s stock (NASDAQ:VTGN) fell dramatically on the results July 22 as shares closed 86% lower at 15 cents each.
Verve Therapeutics Inc. has packed a lot into the past few weeks. The latest is a four-year research deal with Vertex Pharmaceuticals Inc. to find and develop an in vivo gene editing program for an undisclosed liver disease. Vertex will pick up the tab for program costs as Verve does the preclinical R&D. Verve is getting an up-front $60 million from Vertex, along with a $35 million equity investment.
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.
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.
Recently launched and relatively small Avista Therapeutics Inc. has just cut a deal with comparatively massive Roche Holding AG that could bring the new Pittsburgh-based company more than $1 billion. Avista’s single cell adeno-associated virus (AAV) platform will be used to develop intravitreal AAV capsids that match a Roche-defined capsid profile. Roche will evaluate and license Avista’s capsids and conduct the preclinical, clinical and commercialization work for the gene therapy programs.
Controlling the epigenetics of a patient, figuring out what genes are expressed and understanding their level of expression, is at the center of Epic Bio, a new company founded by Stanley Qi, co-inventor on the CRISPR patent held by the University of California.
Now that Novavax Inc. has received an emergency use authorization (EUA) from the U.S. FDA allowing adults to receive the adjuvanted vaccine to ward off severe acute COVID-19, it’s time for the CDC to weigh in. Once its Advisory Committee on Immunization Practices meets July 19 to discuss the vaccine and make a policy recommendation, the vaccine will be available on the market.