Thanks for the reassuring message and calming me down.
After the BMRN CRL, how the playing field will change. The support for SRPT from reluctant advocates will be enormous. Everyone wants at least 1 option.
The smaller trial could actually help. Their data was a mess. The pt testimony was powerful on the board and I suspect it will be no less so for SRPT. Ultimately that could not swamp bad and misleading data; SPRT does not have that. I could see all 12 and some of the new boys there, that would be wonderful.
After yesterday, anyone that thinks BMRN is any kind of competitive threat is dreaming. I expect they get a CRL, but if they don't, with the SAEs, paralysis, 74% drop out rate, narrow label, etc; they will be non existent. I feel for the boys and hope the FDA moves quickly to get this over with for their sake. However they were thorough, professional and determined yesterday to make sure their ducks were in a row; I expect no less from SRPTs Adcom.
I believe its 60+20+20 (on drug) and the others not on an active drug for their condition. That is one fo the things BMRN was playing # games with, 534 patient years, vs active + placebo years.
I was amazed at the comment from Temple (that Thig mentioned earlier), Its targeted(precise) and it must do that 1 thing or its not precise and whats the use then. Also small victory when Farkas(?); reminded everyone that the 7% increase was a % INCREASE, not an absolute #. He mentioned it went from 1.00% to 1.07% NOT 7.00%. And for D which many have a hard time believing is accurately measurable when below 10%. While applicable to BMRN, maybe a shot across the bow of other so called precise medicines.
Still can't get over the drop out rate, 74 +/-%. Some here say they would take Dris if the only drug out there, but many already voted with their feet (no pun intended).
And finally, Paralysis, Good Lord! No one had the sense to make that material public information (GSK, RNA, BMRN)? As long as you say its not your drug, its not your fault, so sweep it under the rug. Many SAE, still not resolved at time of the end of study!
This was study 349, pg 97. The more one reads, the worse it looks.
The dropout of subjects during the study and the lack of control make this study uninterpretable,
even if the analyses are on parent studies. A total of 26%, 43% and 76% of the subjects dropped out
from the study at 24 weeks, 48 weeks and 72 weeks due to AE or termination of the study.
It will get punished for this failure, but also for the next several Exons in development. Ask them how the AEs are on their current trials and see what answers you get if any. Its not their sequence, its their backbone.
I am skeptical of the BMRN numbers, so I recreated my own, using individual yr male births, incidence of 1 in 3500 and US X51=13%. Granted this is not perfect, but it would appear nothing is wrt determining this population.
1st, why would BMRN overestimate this #, remember they used 2340 in their NDA, the FDA likely flowed that thru. My only good answer is either RNA fluffed it up to make the purchase look more juicy or BMRN fluffed it up to convince themselves/investors they were geniuses.
2nd, some possible errors in my numbers; female DMD, counted as 0 and is very rare but no clue what it is, immigration after birth, early mortality. wrt mortality I assumed a factor for each successive year after assumed non ambulation which frankly is a guess.
non diagnosed yet: 390:
non ambulant: 678
total DMD X51: 1800
So I'm saying I believe the BMRN # (and FDA #) stated in the BD is BS. Its possible their 2340 # included those not diagnosed yet, but IMO that's a bit fishy and misleading. With better awareness, perhaps SRPT can reach some of those, but that will take time.
Anyway, I thing 1400 is the correct #. Using 1400 vs 2340 does not change my conviction any wrt what SRPT is worth; the #s are so big and this is only X51 and only US.
I hope no suitor as I know the potential true value and don't want it taken before realizing a lot of that value; However if the suitor can hasten the development and other processes and pay up for the responsibility to do that; I could be convinced.
Most here know it works and works well. Once X51 is approved, its imperative to roll quickly on the remaining boys and remaining geographies.
The 50/50 split the market scenario was basically taken out yesterday. If they are somehow approved, they are behind SPRT with the safety stuff and the consensus is they take most of the market. However, its more likely SRPT either gets 100% or 0%. IF SRPT gets a CRL(0%), they'll be back when the phase 3 is done and the data (my expectations based on 3+ yrs of no AEs and mounting benefit) picture is complete. BMRN is at least 1 year behind and with a new 2 yr phase 3 trial, more than 1 yr; they are not a competitive threat either way. The patent picture does not change that much, 10% of rev gone to those shysters, does not change the big picture.
So we are left with an extremely high probability that SRPT gets 100% of the market(USX51). The market has not priced in the other Xs YET, nor the fact that BMRN other Xs will act similar to their X51.
Fair enough. What if its 7.5; PT of US X51 is 117; for ONLY USX51.
I do not know the exact # and its unknowable; however its clear to me that based on the 2340 USX51 pts reported by BMRN/FDA, its extremely under appreciated how much revenue this could bring in. The 2340 is almost double the previous # assumed, 1200.
What I'm trying to keep focused on is not only X51 approval, but the next 10 approvals, EU, ROW. $7 is nice, but with a couple years patience, we could be talking 100s of $ of gain. Granted we don't have X51 approval yet, so I have 1 eye on that and 1 eye on the bigger prize.
Because I was being conservative. BMRN is 18 right now after thier recent drop; supposed high rev growth co, lots of promise, etc. I think BMRN is way extended even w/o Dris, its mostly JJs fluff.
The IBB P/S is 6.7 with mostly larger established companies relative to SRPT.
I don't know what the right # is, but somewhere about 10 seems right to me. Once X51 is approved, the growth factor will come into play and as a result should see a richer valuation for a couple years based on that growth. I don't think it hits this P/S overnight, heck the rev will take months to happen. But once X51 is rolling, the template for these X's will be laid.
What the market took away from BMRN yesterday($1B), they did not give all to SRPT($.3B)
So what did BMRN lose:
* very -ve BD, approval is extremely indoubt; so maybe the analysts from 90% to maybe 10%.
* if approved, no REMS but safety monitoring; procedures and protocols need to be written. agreed, implemented; 2 month lead GONE. SRPT was hammered on the 2 month lead, we've not seen that value shift back into SRPT yet.
* subset; BMRN not tested in older boys, nor not sufficiently in younger boys per BDs. So BMRNs population is not the 2340 mentioned in another thread, it much smaller. SRPT is of course trailing older and younger boys, so if those trials count and are used in eventual approval, SPRTs population is 2340+/-.
* BMRN lost considerable time; if both drugs are CRLd, SRPT has a huge time advantage on ALL the trials in place and DOSING. I would guess easily a year advantage. While a SRPT CRL would hurt in the near term, the +1 year facts would not change and SRPT would be back in a couple quarters.
* Credibility; JJ lost a lot and IMO their revenue stream and practices are suspect. Ed and the moms stand above it all.
* FDA, despite the decision 2 years ago and the lambasting they get here daily, did a good job of laying out the +s (few) and -(s).
OK I was curious so I went ahead to do the math on the other X's..
To keep it simple, multiple the $156 X 6.38 for ALL US DMD X's. Yes that is level pricing, but I'm keeping it simple. Also keeps P/S level, but of course when SRPT starts rolling these Xs out the P/S will be closer to BMRNs 20 (or excuse me 18 now). To include EU, multiple by 1.35 as they have a larger population and diagnosis/SOC will even out over time. Yes this is a huge # and I've not factored in growth, because of course these boys will live much longer when on SRPTs drug.
Incidence: The incidence of DMD is 13 to 35 per 100,000 yearly or about 1 in 3,500 live male births
globally. The estimated prevalence in the US is 18,000, with an additional 15,000 cases in EU (McNeil
2009). Mutations that are correctable by skipping exon 51 are thought to make up around 13% of the
DMD population, resulting in a prevalence of 2340 boys in the US and 1950 boys in the EU.
This was in BMRN reports as well as the FDAs. The #s were slightly different; I will assume the FDA and BMRN know better then me (using simple math to calculate the # ot DMD pts), the addressable market. Also don't see much benefit from BMRN to spike the #s with this audience to make them look higher than reality. Also stated several places about 1/2 of these are ambulatory.
So bottom line, these #'s are generally 50% higher than what I thought, US is higher, assumed to be due to better diagnoses/detection.
Anyway, I've laboriously churned these numbers in my super computer and arrived at a new PT based on P/S multiple and 100% penetration (discount that as you wish).
2340(pts) * 300000(price) * 10(P/S multiple) / 45,000,000 (shares) = $156 (US X51 target price)
Notice I did not count (yet) EU X51, US X45, US X53, Other Xs in the pipeline nor other X's in the EU.