FDA has in the past from time to time approved oncology drugs based on single-arm phase II-trials and a few more based on randomized phase II data. Phase III - by definition - is prospectively randomized two- or more arm-trials.
T-DM1, an ADC targeted for HER2+ breast cancer, had strong single-arm phase II data and got a refuse-to-file-letter from FDA in summer 2010 and, hence, needed to present randomized phase III data for filing in 2012. Approval is pending.
CLDX has over 77 mio$ in cash (acc. to the CEO's recent presentation at Lazard) which puts them in a strong position for license talks as they can last "well into 2014" (Marucci) based on current activities.
True. But: Adcetris initially was approved for patients who had exhausted all other treatment options for hodgkin's (incl. autologous stem cell transplantation) or a rare other type of lymphoma (ALCL).
T-DM1's initial filing was not accepted because FDA felt that there are still treatment options available for mBC patients in 3rd line (it was deemed not an unmet medical need) and FDA was about to approve eribulin based on a large randomized phase III.
All I'm saying: It's not excluded but FDA usually does grant approval based on single-arm phase II only for desperate indications and they may again (as in the T-DM1 case) feel that enough alternative cytotoxics are available in this clinical situation and therefore refrain from taking the risk of approving CDX011 at this stage and level of evidence. They may also want to see further proof of the GPNMB marker concept in the clinics in a well controlled setting first.
My bet: A phase III and a licensing deal are much more likely than an accelerated or fast-track approval at this point.
can 011 obtain fast track approval for triple negative BC under the revised FDA regs? A full blown PIII is not a fast track, hence one would assume that the new regs contemplate something other than a double arm PIII trial for 011 re TNBC.
Two questions i) is this likley, and ii) what is the timing of a decision, Q1 2013 or later?
the $77 mm does not include another $30 mm that Cnatior is busy raising.
IMHO: (i) it is NOT very likely (see also my answer to db998 a few minutes ago under this same topic); (ii) Q1 at the latest, because CLDX must have been looking at the results for some time and SABCS is only the first publication of the up-dated results, so they may already have sat with FDA (don't think so) or have a date for a meeting to discuss (these meetings need to be requested and prepared with the submission of data and questions to be discussed etc. usually a couple of weeks all in all depending on FDA availability and on the urgency of the issue at hand). Marucci made it sound like it is something that will happen after SABCS, though ("We will also then talk about our plans for going forward"..).
Let's wait for CLDX' press release around SABCS and hope for more clues.