A lot of exciting stuff in the world cystic fibrosis to discuss from the last week or so…

Drug Pipeline Update

A few medications are charging through the drug pipeline that could one day, very soon, broadly impact the CF community. First we have this guy, SPX-101. SPX-101 is an ENaC Modulator. Without getting too complex, it helps patients clear mucus from their lungs. Last week the sponsor company, SPYRYX Bio Sciences, released Phase 2 data, which showed that patients on live drug compared to placebo saw an average of 5.2% increase in FEV1. Further, patients with moderate lung disease, “FEV1 >55%” saw a gain of 8.3%. To me, the most shocking part of this is that patients saw this change at day 7 of treatment, and were able to maintain that gain through the 28 days of the trial. For relative comparison (and although this works through an entirely different mechanism), these FEV1 gains are more significant than both Symdeko and Orkambi. This medication, if approved, would work alongside CFTR modulators. SPX-101 is not genetic specific, meaning it could be a viable treatment for all patients.

Proteostasis announced some lackluster data from their investigational CFTR Modulator, PTI-801. Although they are claiming the medication is having positive results, analysts seem to think otherwise, and I, unfortunately, agree with them. Proteostasis does have a pretty decent looking pipeline with a bunch of potential candidates to compete with Vertex, but it seems we are still a ways away from that coming to fruition. An ongoing phase 1 study of PTI-801 taken in conjunction with Orkambi, showed minimal change in baseline FEV1. Granted Orkambi really isn’t the miracle medication that people want it to be, and can create some complications of it’s own (and maybe it did with these investigational cases), the results from this are just so bleh. I realize it’s phase 1, and efficacy really isn’t the defining goal, but FEV1 gains of 1.9%, 2.5% and 0.6% in respective ascending doses don’t seem so spectacular. I don’t want to say I’m giving up on PTI-801 or the Proteostasis pipeline, but this looks underwhelming compared to some of the FEV1 gains we’re are seeing in other ongoing trials.

A Livable Cure (?)

It’s hard not to look at the big picture when we start seeing some of these positive trial results, and quite frankly, how many successes we really are seeing. The CFTR Modulators and the Triple-Combos from Vertex have made the biggest splash so far, but they’re just a piece of the puzzle (a really enormous piece at that). Imagine for a moment we can marry the impending Triple-Combo medications, the investigational anti-inflammatory from Corbus, the above ENaC modulator form Spyryx and (hopefully) a new antimicrobial with existing treatments. Suddenly disease prognosis looks very different. It’d almost be like a livable cure. Now I realize that is a lot of “if’s” (basically continued success for the listed meds and FDA approval), but realistically speaking, even if we are able to achieve half of that list, we are quickly charging deep into the dreams that we’ve all had – long, successful lives. I think there is reason for all of us to be quite optimistic, but as with any point of drug development… only time will tell.


I did something pretty radical and shaved a mohawk. I think it looks pretty good. I’ve always wanted to give it a try, and finally I did it… after years of desiring it. I finally had the confidence to do it.


….and that’s what I’m thinking about