Treosulfan is coming to America for BMT in 6 months; warranted discussions on P-3058 as it leads MOB-015's Canadian launch by 12 months.

Nothing's perfect or certain, but you can see out in front with some light on a subject

Hello!

It’s been a while since I talked about Medexus in any substantial way. There are a few pieces I’m proud of for having done on this blog. Piclidenoson and Treosulfan are one of them, to include some of the Moberg content.

The drug is up for an FDA decision by October.

Improving the standard of care for leukemia amongst other conditions in America is an important thing that we should do. The vast majority of the world has been dealing with BMT conditioning for many years with Treosulfan. It’s just part of the US system, but on a personal level I find it all deeply unethical and overly formal to have kept this drug away from this population for as long as the FDA has. There’s at least 10,000 people that should be alive that aren’t. I’d warrant it’s more like 30,000 but I’m not going to make these estimates too high. Especially because a subsection of like 1/3 of these people are under 18.

This is made even more important so as new indications ramp up their use of BMT for new conditions that gene editing may treat, like Sickle Cell Disease or Beta Thalassemia with Casgevy. There’s no reason to expose these patients to the hepatotoxicity caused by busulfan as it means we’re needlessly accepting that some of them will also die from the transplant caused by a rare side effect. If you’re reading this, you’ve probably never gone through multi-organ failure, but it’s not pleasant. There’s another drug called defibrotide under the brand name Defitelio that does low 9 figure annual sales volumes that this could nearly but not entirely zero out (it’s held within a much larger company — Jazz pharmaceuticals).

My hope is that the entire medical infrastructure of the USA to include physicians and insurance realize how stupid it is that we give a medication that has a slim chance to kill you and then spend > 100M USD to rescue these folks with expensive medications that cost like $200-300K USD on top of all of the hospital staff, etc when the SOC is something that just avoids the issue due to differences in drug metabolism...

OBVIOUSLY Medexus comes with other risks. I don’t like Ken very much or the IR at Medexus. I owe you guys a new writeup on P-3058.

This will be out soon, but I wanted to make sure you’re updated on this prior to the weekend.