When I last wrote about Catalyst Pharmaceuticals, they were pursuing their plan to charge a lot of patients money for an existing drug, currently being provided without cost, which plan was somehow to everyone’s benefit. The FDA, though, sent them a refusal-to-file letter, and the agency has now clarified their position: Catalyst has to run another Phase III trial, as well as several toxicology studies, so this could take a while. This sounds, from the outside, like a case of very poor coordination with the agency, at the very least – being told to go run another Phase III is never a good sign, and this for a drug that people are already taking.
I also wanted to update the lesinurad story. Back in 2012, AstraZeneca bought Ardea to get this gout drug candidate, but things have apparently not gone very well since then. The drug was recently approved as Zurampic, but AZ has now sold off the US rights to the drug to Ironwood for “up to 265 million” in payments, plus single-digit royalties. That does not sound like a very good return on the roughly one billion dollars they spent to acquire it and the money they’ve spent since then to get it to market. There’s another Ardea gout drug from the original deal, but apparently Ironwood is getting some rights to that one, too.
Finally, many will remember the disastrous first-in-man trial of TGN1412 (a drug that is in the process of making what would be the most unlikely comeback since thalidomide). I remember wondering at the time what it must have been like to be one of the clinicians involved, and now we get to find out. It was just as horrible as you might have imagined:
After I’d given the dose to the seventh man, a nurse told me one of the men had a headache, which is common. While dosing the eighth, the nurse returned and told me that the first man was feeling worse and that a second was throwing up. Then they tumbled like dominoes. One man tried to walk to the toilet and collapsed. The wards became chaotic, with blood, vomit, and staff and patients shouting. It was clear which two had been given the placebos.
Yeah, that one was unblinded pretty quickly. Everyone survived, barely, and the physician involved says he’s never done another first-in-man trial since (which I can well believe). He also says that he has wondered since then about how the participants in this trial have been doing, but doesn’t think that they would want to hear from them. He may be wrong about that, actually – I hope he does hear from some of them after his article, and that it helps him with coming to terms with what happened.