Rusfertide trial success

[EPguy]

The trial completed last month and the sponsor issued this press release right away. When they don't it's sign of a dud or iffyness

takeda.com/newsroom/newsrel...

"The primary endpoint of the study was the proportion of patients achieving a response, which was defined as the absence of phlebotomy eligibility." 77% got there on Rusf, 33% on Placebo. That is a big success by typical definitions.

"The majority of adverse events were grade 1-2 injection site reactions and all serious adverse events reported were deemed to be not drug related"

Sounds like a winner. I suspect it will be often be used as an adjunct to our other treatments, for example IFN pts not controlling HCT. This might allow a lower dose of IFN (or Rux, HU...) for some pts.

[hunter5582]

It will be good news if Rusfertide is approved. It will give us another option. I expect that it will be able to replace venesection for those using a venesection protocol and, as you suggest, be used in combination with cytoreductive medications.

[Bluetop]

Thanks for posting. Good to see another option emerging.

[Luthorville]

For a while I didn't really see a place for this drug. It seems seems quite limited for those that are on other therapies, but where HCT isn't controlled. But it's a second injection and doesn't appear to be disease moderating. If Besremi is unable to control HCT, for those instances this could be helpful in conjunction with it. There's an argument that it might be better suited for me rather than Jakafi. But this will depend on the long term ramifications of exposure to this drug.

[EPguy in reply to Luthorville]

I agree it's not the huge advance it would have been 10 years ago when phlebs were a main standard of care. As adjunct does seem its likely use, esp with many IFN pts having trouble with HCT. Compared to Rux, Rusf is not likely to have disease modification ability, so Rux should hold that advantage. But in your case it could be an option for blood counts since you don't have plt problem. In my case for example, PLT is my larger issue with my ET like PV, so Rusf would be of minimal benefit right now.

[Luthorville in reply to EPguy]

For those reasons it seems quite niche. If I were a physician, I guess I would perhaps want to see if the Besremi worked by itself and if not add this on after a year or 2. I supposed it could get to the point where both are offered together. It’s really for a subset of interferon users.