Hi Kronberg, thanks for the message.
First of all I don't think my forecasts and numbers are too optimistic.
I cannot see what patient or physician would not prefer to be on Elfabrio aside from those doing well with low ADA and low side effects from Fabrazyme. As well as the Efficacy and Safety data generated to date, one should also keep in mind earlier PK/PD data which showed significantly more enzyme going into the heart as well as kidneys. If I were a patient I might even demand to be put on Elfabrio if my insurance covers it. And given I'm probably on an isurance that covers Fabrazyme, then I would assume it would cover Elfabrio too.
I believe the above only makes sense. This is my life you're talking about, this is my family I'm going to spend time with. I want to maximise it with a few safety issues as possible.
A timeframe for 50% is too difficult to really predict as I'm sure you'll appreciate.
However I might say, quick uptake of all US & EU Fabry patients that are on very poor eGFR slopes as those recruited into the Balance trial, this being quick for both Fabrazyme & Replagal. Quick maybe 3-6 months post launch and available.
How much of the market would that be, no idea. For fun I would hazard a guess at 10% of the market.
Then we have the uptake of these doing generally poorly and considering the option with the physicians I would put that at another 20% of the market. These coming over a 6 months-1 year period.
Then those that are doing ok on eGFR but are having side-effects from Fabrazyme and want to try Elfabrio to minimise that, another 10% of the market. These 10% also coming 6 months to 1-2 years.
Then some those that are doing well on Fabrazyme eGFR and no big side effects, but that decide that they would like to switch based on the great feedback they hear from everyone else on quality of life. Anecdotal evidence that Elbabrio patients have even started sweating again, which they hadn't done so for years. it might seem small but all these things point to happier patients. And who doesn't want to be happier. These coming from the 1-2 year mark
And that is my highly speculative assumption, though is not to say that Elfabrio would not become standard of care. I only feel this chance is somewhat harder to say as I am not blind to the fact that Sanofi has plenty of expertise and cash. That said I have every confidence in both the Phase I/II and 3 Phase 3 trials data across naive and switch patients as well as all the PK/PD and organ data, as well as Chiesi capacity to maximise the value. They are a Top 50 global company with rare disease ERT experience. Very satisfied.
As far as timing of switch, yes of course this can happen 'in the middle of the year' or at anytime really. I don't quite get why you are focused on the middle of the year?? Curious??
Balance and Bright were both switch studies from patient previously on Fabrazyme and/or Replagal. So yes, any of these patients can switch over to Elfabrio at anytime.
Correct the once in 4 weeks dosing was not approved yet, and based on the clinical data I can see where the regulators were coming from. Not that things were bad, but I don't think it was specifically good enough to get the regulators to agree, especially when they can start the process with the once per 2 weeks regimen that all patients are already accustomed to. So they want more data before approving reducing the regimen by half.
First of all I think that's fine, but more importantly for us, it makes no difference to the revenues as the amount dosed over the 2 week or 4 week regimen is the same and this drug usage is the same. Where of course it could make a difference is for those patients that are doing very well on Fabrazyme or Replagal already, but just want to take less frequent dosing.
I am highly doubtful of a BO, and nor to do I wish it to happen yet, perhaps after a year of sales, and the price would be significantly higher.
But yes, once this shorting nonsense ends and the longs that were in stop freaking out, then we can see the SP take on a true image.
I expect SP to rise nicely even coming into the June strategy meeting, as this may show early indications of Chiesi commercialisation which will spook the shorts.
Just look at GMDA, just the mere suggestion of the upcoming earnings call which may show signs of market uptake or progress in commercialisation efforts has the shorts covering.
I note that yesteday on Shortablestocks, you can see that the short shares available to be seen in that site, when from 750,000 to 1.7 million. So the shorts covering significantly, and which was associated with the 20% increase in GMDA we say yesterday.
So while we will not hear of the amount of the milestone payment which will be made in the next 2-3 weeks based on the USA agreement SEC filing, I still think that much the way the chance of uncertainty and bad news in the market spooks investors tremendously, would expect that for shorts the chance of uncertainty and good news has the same effect on them and rightly so.
The future is bright for PLX and I'm just going to buy more when this nonsense bottoms out. Which could be today, let's see.
Best,
Spidey
