- Tocagen's lead drug candidate failed to meet all primary and secondary goals in a pivotal study for an aggressive, hard-to-treat type of brain tumor, the San Diego biotech said Thursday.
- The setback is of nearly existential consequence for Tocagen, which has focused its efforts on the experimental treatment. SVB Leerink analyst Daina Graybosch called the study "a make-it or break-it moment" for Tocagen, which saw its stock open at $0.75 Thursday, an 82% decline from yesterday's close.
- Per topline results, a combination of the biotech's immunotherapy Toca 511 and the chemotherapy Toca FC yielded a median survival time of about 11 months, one month less than the median for patients on standard treatment. CEO Marty Duvall declined to give further study details on a Thursday investor call, but said the company will begin an operational review as it analyzes the data.
In the high-risk, high-reward world of biotech, Tocagen's result leaves the company struggling to show a clear way forward.
Since its 2007 beginning, the biotech "has devoted substantially all its efforts to developing its gene therapy platform and its lead product candidate, Toca 511 & Toca FC," the company stated in its most recent financial filing.
The data disclosed Thursday are unambiguous, however, showing patients on Tocagen's treatment did worse than those in the comparator arm. No meaningful difference was observed on any secondary measure.
Cantor Fitzgerald analyst Elemer Piros expects further clarity on the company's future to come by year's end. In a note to investors, Piros suggested Tocagen could present more detailed findings at either the Society for Immunotherapy of Cancer and the Society for Neuro-Oncology, both conferences taking place in November.
Piros cut his price target for the biotech to $1 share, but added that any findings from a data review "could serve as a catalyst as to next steps for Tocagen's supporting pipeline."
The company is studying Toca 511 and Toca FC in studies dubbed Toca 6 and NRG-BN006, the latter of which is expected to be run by NRG Oncology, a nonprofit research organization, through a grant from the National Cancer Institute, according to Tocagen's website.
While the company did not elaborate on its planned operational review, layoffs often follow all-in biotech bets that go south. At the end of 2018, Tocagen had 79 full-time employees with 63 focused on R&D activities. The biotech also reported cash, equivalents and marketable securities worth $68 million as of the end of June.