I walked into the Tuesday afternoon Clinical Trials session at ICAD half an hour early, and got one of the last seats in the room. Conference attendees were literally running into the room to try to get a seat. The press was there in full force. Women complained about having to choose between using the restroom (which given the long lines would have meant a 10-15 minute delay) and getting in to this session. A film crew was roving around, blocking the aisles and adding to the carnival atmosphere.
This was definitely the best-attended session of the conference, and the Alzheimer's Association set up an overflow room with a video feed for those who could not get in.
So it seemed like a bit of an anti-climax when the first speaker, Dr. Robert Green of Boston University,matter-of-factly presented data from what everyone already knew was an unsuccessful trial of Myriad's Flurizan. According to Dr. Green, Phase 2 trials had shown enticing evidence that the drug worked, but only in the subset of participants with mild Alzheimer's. Myriad then launched what Dr. Green said was the largest Phase 3 trial ever completed, with 133 trial sites and more than 1600 participants with mild Alzheimer's. Only about 1000 people completed the 18 month trial, though, with 33% of the placebo group and 39% of the Flurizan group withdrawing from the trial.
Unfortunately, there were no differences between the treatment group and the placebo group in performance on the ADAS-Cog cognitive test or in "activities of daily living," and the placebo group actually improved on another cognitive test.
Trial participants taking Flurizan had higher rates of anemia, infection and ulcers than those in the placebo group, but the drug was "generally well-tolerated."
Although unsuccessful, the trial was "successful in answering questions," Dr. Green said, and added that he was proud to be part of such a well-run trial.
Myriad spent a reported $200 million developing Flurizan, and the Danish company Lundbeck paid $100 million for rights to the drug just five weeks before the Phase 3 failure was announced. Disappointing results for investors, but also for the millions of people with memory loss and their families who had read the optimistic reports after the end of the Phase 2 trials.
Privately, researchers at the conference were talking about what the failure of Flurizan and other amyloid-targeting drugs meant. Will the amyloid hypothesis be proven wrong? Were there simply methodological problems with these trials? I'll write more on this later. But one thing is clear: don't pin your hopes on medicines that haven't been tested in Phase 3 trials.