The revenue ramp of ETEP is something I continue to look at and try to make sure I understand.

I
posted something the other day about it, and wanted to follow up a bit. The dichotomy of opinion on
whether this was a positive of negative is amazing. I choose to look and feel it is a great positive what
is going on.

Q Q4 Q1 Q2 Q3 Q4 Q1(Est)
Revenue(M) 5.4 16.3 33.0 47.7 57.2 63.9
Run rate(M) 44.0 85.3 161.9 215.8 238.7 269.8
RunRate/Pot rev 8% 16% 30% 40% 44% 50%

So what I do is marry this data with the fact that the total potential revenue right now for ETEP is about
543M. Note my run rate calc, is slightly modified to account for boys newly added on for a partial Q
and not a full Q; its close to x4, but not exactly. Its not rocket science, but I think its slightly more
accurate. Also note, the 543M is today and grows slowly as boys live longer, mature and there are new
births.

So IMO whats going on, is SRPT has been very successful at getting the mid weight boys(amb and non
amb) on drug, that they have or are very near crossing the 50% of potential revenue for US X51. So by
definition, they have a smaller revenue pool each succeeding Q to be able to get on drug. More opinion
than fact is what that revenue pool is comprised of; the farther out ages; older and much younger. So
while they have ½ the revenue potential, they only have ~1/3 the boys(including the undiagnosed);
Hence DIs comments several times about long runway. So they need to continue to work the doctors
and their message to reach these boys/parents. That is critical, as critical as them looking at the more
rare Xs, is finding and reaching these hard to reach X51 boys.

Q Q4 Q1 Q2 Q3 Q4 Q1
Est Boys on Drug 4% 9% 20% 26% 30% 34%

Also note, Anthem reversed their stance (although privately they were apparently covering) on non
coverage and that may boost the new adds for a Q or 2.

I don't mean to say SRPT are such great salesman. They might be; however, I expect what's actually
going on is the boys/parents are beating a path to the SRPT door. That bodes well for all the follow on
Tx; GOLO, CASI, PPMO, GT, etc looking forward.

So it appears ETEP has crossed(or extremely close) the 50% of total market in 6 Qs. So what does that
imply for GOLO if approved:
1) since genetic testing (DecodeDuchenne) is ongoing and they are finding many X51 boys, they
are also finding many X53 boys; this will dramatically speed up initial conversions.
2) Certainly there will be some initial inertia on INS coverage, but they(and shorts that tried to
spread FUD) will lose some of their arguments:
1. N=25 vs N=12 from a smaller pt pool.
2. active PBO arm for 2 yrs.
3. about 3x the D production vs Etep
4. trial(active and PBO(not NH)) performed in EU, same geography.
5. FDA guidance document clearly supports D production as a means to approval.
3) ESSENCE, the assumed confirmatory trial is or will be nearly fully enrolled with N=~40(on
X53 drug).
4) Word of mount(awareness) from parent to parent is very high.
So if SRPT was able to reach 50% of potential revenue in 6Q, what might they be able to do on GOLO,
if approved. A complete guess is maybe 4Qs or 3Qs, certainly not more than 6Qs. It also bodes well
for any later approval of PPMO and GT, for all the bullets mentioned above on genotyping and having
the market identified, etc.

I would urge anyone interested to read the following wrt the GOLO test in question, which is now
ongoing on drug for almost (or just past) 2 years on drug. The link 'SKIP-NMD' is dead as the SKIP
program completed their time period and were not funded further; however, SRPT continued the trials
for the duration.

https://cordis.europa.eu/result/rcn/189889_en.html