Hopes were high for tipifarnib a few years ago, when Kura Oncology Inc., of La Jolla, Calif., completed a reverse merger and closed a $60 million private placement with a syndicate of top venture funds around the same time it executed an agreement with Johnson & Johnson (J&J) unit Janssen Pharmaceutica NV, of Beerse, Belgium, for an exclusive license to develop and commercialize the oral farnesyltransferase inhibitor in multiple oncology indications.
KSQ Therapeutics Inc. nailed down $80 million in series C money to push into the clinic oncology drug candidates generated by the company's CRISPRomics discovery engine. With the new cash, KSQ will move its first program into testing within the next 18 months, and plans to advance up to three more cancer bids into IND-enabling studies. The first candidate consists of a modified adoptive T-cell immunotherapy that has shown efficacy in animal models of PD-1 resistance.
Geron Corp.'s shares took a major hit as Johnson & Johnson unit Janssen Biotech Inc. ended their collaboration and license deal, leaving the telomerase inhibitor imetelstat to go it alone into the upcoming phase III trial.
Gearing up for an NDA filing for selective adrenergic modulator Edsivo (celiprolol) in vascular Ehlers-Danlos syndrome (vEDS), Acer Therapeutics Inc. has begun drawing attention for another candidate in the pipeline: ACER-001 in urea cycle disorder (UCD), where company-watchers are placing bets on its odds of taking market share from the only approved therapy in the space, Ravicti (glycerol phenylbutyrate), from Horizon Pharma plc.
With its reactive aldehyde species (RASP) inhibitor reproxalap a winner in phase IIb results against dry eye disease (DED) and already undergoing study at the phase III stage in allergic conjunctivitis (AC), Aldeyra Therapeutics Inc. might be able to pull off a dual NDA submission.
Wall Street apparently wasn't much pleased with the boxed warning on Verastem Inc.'s first approved product, trimming shares (NASDAQ:VSTM) by 19.6 percent, or $1.74, so they closed Tuesday at $7.15, even though analysts pointed out that the side effects with Copiktra (duvelisib) can be handled by doctors.
Investors will have to wait until November to find out the details of Amarin Corp. plc's cardiovascular (CV) outcomes trial with Vascepa, but Wall Street heard enough Monday to increase the share value of the Dublin-based company by 315 percent, or $9.41, closing at $12.40.
With Blueprint Medicines Corp. due to start screening patients soon for the phase II Pathfinder trial that will test avapritinib in systemic mastocytosis (SM), investors are weighing the chances of several players in the disease and mulling how success might most rightly be judged.
This summer, Adverum Biotechnologies Inc. drew Wall Street's eyeballs to a potential gene therapy for wet age-related macular degeneration (AMD) when the company made known that its IND had become active for ADVM-022 (AAV.7m8-aflibercept).
Days after a court decision torpedoed four patents protecting Acorda Therapeutics Inc.'s Ampyra (dalfampridine) – clearing the way for generic versions of the only drug approved in the U.S. for improving walking in people with multiple sclerosis – the company learned that the FDA has extended the PDUFA date for its NDA related to Inbrija (levodopa inhalation powder) to treat off-period symptoms in people with Parkinson's disease (PD) on a carbidopa/levodopa regimen.
