FDA approval was granted for Luxturna gene therapy, which can be used to cure a genetic disease that causes blindness.
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/u…
Has anyone dug into this company much yet/before? Maybe bulwnkl?
Looking back through some of my open tabs, I see that Fuma102 mentioned ONCE in Saul’s recent “do you have any candidates (for doubling in 2018)” thread.
Here is a direct link to that post.
http://discussion.fool.com/i-have-two-candidates-one-is-twou-a-c…
Fuma said this while there is much bally-hoo about CRISPR, I have to say, show me the clinical data! Many of these companies frankly have none (I’m looking at you Editas)!
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I would suggest a basket of 50% EDIT, 25%CRSP and 25%NTLA to catch any Gene splicing machine wave. You know, like biotech, nanotech and cryptocurrency in the early days. This stuff looks real, and any success could bring in the crazies to buy up the stock.
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EDIT has a partnership with Allergan for a gene therapy blindness cure. (EDIT-101 for Leber congenital amaurosis type 10 (LCA10), the most common form of hereditary blindness. While the program won’t enter the clinic until the second half of next year, it has shown remarkable effects in almost all mice to receive a sub-retinal injection, and the program could move forward fairly quickly once it starts.)
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how come the stock has plummeted if this is such a promising approval? Is it really expensive gene therapy like CAR-T or is it a small market. I think there was bad news a recent convetion and this is clearly not enough to move the needle after that.
FYI, I am long EDIT and BLUE, but think EDIT, CRSP and maybe NTLA have huge potential. But I thought that about Human Genome and others back in the good ol’ days.
Pete.
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Pete,
EDIT has a partnership with Allergan for a gene therapy blindness cure. (EDIT-101 for Leber congenital amaurosis type 10 (LCA10), the most common form of hereditary blindness
This is pretty much copied from a newsletter without your own due diligence. LCA itself is the most common form of hereditary blindness, with about 2,000 cases of LCA10 worldwide.
Of the LCAs, spark targets one of those mutations, RPE65, with over 6000 cases worldwide.
As for why it fell, it fell because of hemophilia A results from biomarin. From previous post cited:
Spark has a hemophilia A treatment (there are many more hemophilia A patients than B) - and Spark’s recent drop was likely due to BioMarin reporting positive results. I’m not positive how this pans out. When looking at patient oriented outcomes - number of bleeding episodes, need for infusions, etc - both drugs work well. This may be a race for first mover, and price & insurance company approval may play a role here. Regardless, the hemophilia A market is now a crapshoot, but certainly proof of concept for Spark
I’m still unsure why edit is more expensive. Spark is crossed the finish line on one drug and has a second drug soon to be in p3 that should likely be accepted. both of which have a larger potential market than edit’s lead LCA10 candidate.
Edit has more hype, spark has more beef. I’m not sure why spark isn’t a higher valuation. It’s literally the leader in the genetic disease editing field!
With that said, there’s likely room for more than one winner. Just think spark is undervalued compared to it’s peers.
https://globenewswire.com/news-release/2016/06/01/845160/0/e…
http://ir.sparktx.com/news-releases/news-release-details/fda…
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expected sales of Luxturna are $400m in 2021, and a possible price tag of >1 million for a one-time dose.
and insurers complained about the cost of Hepatitis cures…
expected sales of Luxturna are $400m in 2021, and a possible price tag of >1 million for a one-time dose.
and insurers complained about the cost of Hepatitis cures…
Tough to put a price tag on being able to see. One of the things I read was that in determining how to price Luxturna, Spark had included insurance awards following people losing sight from accidents and such. A million doesn’t seem that bad for such an important sense. Smell would probably only go for maybe $75,000-150,000 comparatively, I would guess. Hearing maybe $300,000-600,000?
Fuma,
Yes, I did grab that tidbit from a trusted source with no additional research.
Spark info from a Fool news release…
There are approximately 1,000 to 2,000 patients in the U.S. with biallelic RPE65 mutation-associated retinal dystrophy
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Gene therapies are incredibly complex and Luxturna is no exception. The high cost of developing it for a small number of patients could result in its becoming one of the most expensive drugs on the planet
As of the time of this writing, Spark Therapeutics hasn’t disclosed pricing yet, but management told investors during its third-quarter earnings conference call that Luxturna could hit the market with a one-time price that’s "in excess of $1 million per patient. Management based that price on “reasonable assumptions” of the direct costs that are incurred by these patients over their lifetime. They may have a point. "
So the market for this is small to start with, even including "Of the LCAs, spark targets one of those mutations, RPE65, with over 6000 cases worldwide.
You say I’m not sure why spark isn’t a higher valuation Here is why (in my mind):
No other country will pay $1M for this treatment and I am not sure we will. Single payer systems like Canada won’t. Poor coutries won’t.
Also, pricing will be based on lifetime costs of being blind, which may not sway insurance companies here. Much of that cost is probably not covered by insurance now, so they are not saving money.
Why is EDIT more richly valued? Yes, some hype, but it could be well justified. My understanding is CRSPR technology is way simpler to use than viral vectors that companies like Spark use. My understanding is that it could leapfrog CAR-T therapies and sell a solution at a much lower price. EDIT will have partnerships and take royalties, thus sparing them from a lot of developement costs. They are also subject to great competition from CRSP (the company), so that could be bad.
For me, EDIT, CRSP are high spec stocks, but I think they broad application of the technology and the relative easy of gene splicing with them, make them the place to commit some of my high-spec money. And I did.
Pete
*P.S. are you the Fuma from the biotech boards back around 2000+ time frame?
Also, I just read this while review Vertex news…
Leiden says some of those goals came to fruition in 2017. He expects to continue on the same trajectory as Vertex sends potential triple-pill regimens to treat cystic fibrosis into pivotal trials next year and dives deeper into treating other diseases with Crispr Therapeutics (CRSP) and Moderna Therapeutics.
So a broadbased technogoly that might be used my many biotechs is very appealing to me.
No other country will pay $1M for this treatment and I am not sure we will. Single payer systems like Canada won’t. Poor countries won’t.
The questions I ask myself, looking for a broader perspective, is how will advances in the technology and techniques change the price over the next five or ten years? Likewise, are there companies around now that will make serious profits if and when those prices drop?
Too bad all I have is questions.
Too bad all I have is questions.
good questions are better than bad answers.
Looks like they’re pricing it 15% less than $1 million, at $850,000.
https://www.cnbc.com/2018/01/03/spark-therapeutics-luxturna-…
~ bump ~
Figured since Duma was talking BLUE, the approved gene therapy, ONCE, should get brought up. its been on fire since Luxterna approval, up 47% YTD.
3b market cap. Still undervalued imo.
its been on fire since Luxterna approval, up 47% YTD.
Original poster in this thread…and had not looked at ONCE in months and months.
There are lots of quite investable companies out there…more than I can keep up with for sure.
Find good companies that are in the nice part of their growth cycle, but shares, be patient (but watchful), and beating the indices is far easier than rocket science. Biggest key might simply be to believe/recognize that it can be done…which is maybe the biggest benefit of Saul’s board here.
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