Since Q3 and into Q4 I have tried to understand the rates of revenue added each Q and the apparent

slowdown in that rate. Some of this 'slowdown' IMO is driven by the exceptional increase in that rate
in Q2; which likely was driven by the log jam of 'insurability' being broken; or in other words, we are
heading into a steady state rate. If the insurance issues had not happened, the surge would have been
Q416 and Q117, rather it was Q217 and Q317 (6 months after approval).

To summarize that data, I track the US revenue each Q, but also the rate of NEW boys added
rev(NBAR stupid acronym so I don't have to keep typing it) each Q. In Q416, they had 5.4M of
revenue, but those boys mostly (assumed) came on in the middle of Q4, so the revenue those Q416
boys contributed in Q117, was 10.8. Therefore, that means the Q117 NBAR was 16.3 – 10.8 = 5.3 in
Q117. Each Q has weight assumptions and discontinuations assumptions, so its not a straight
calculation, but that would get one close in the short run. I build it up from the start, Q416. SRPT has
only 1 source of revenue right now, ETEP. I have assumptions based on the weight which is correlated
to revenue, cost of ETEP per KG, the timing they came on treatment(Tx), etc. We have real data on
each years # of boys based on birth records, weights for each year, etc; so a picture can be assembled to
represent the DMD population and how the reported revenue translated into boys on drug. The table
below summarizes the revenue and NBAR.

Q4 Q1 Q2 Q3 Q4 Q1(E)
Revenue(M) 5.4 16.3 33.0 47.7 57.2 63.9
NBAR(M) 5.4 5.3 11.7 7.2 3.2 4.2
Boys Added 57.6 56.5 141.6 87.3 42.7 56.0
Cumulative 57.6 114.1 255.8 343.0 385.7 441.7

With that foundation, I try to layer in the different cohorts of boys. Some other assumptions to help
you understand how the 'picture' is put together. My assumed DMD prevalence is 1:4250, right in the
middle of the often reported 1:3500 to 1:5000 live male births. Live male births by year and weight at
each age are easily available online. DMD boys on steroids are generally larger (heavier) but many
times shorter due to steroids, but my weights data comes from healthy US boys and may understate
DMD boys. Also, starting at age 15, I increase the 'mortality' factor due to their early passing due to
this nasty disease. I also have an 'insurability' factor which as you might guess affects the older boys
more.

With all that I also have the # of boys on drug Q4 end at 386, I'll round that to 400.

The DMD population in simple terms and with 'arbitrary' definitions is comprised of :
1) 0-4 YO(year olds); not yet diagnosed
2) 5-13 YO ambulatory
3) 14-25 YO non-ambulatory

Also I use the Bo snippet from the CC, included below: ..all through the year averages went back
and forth between 13 and 14, the majority of the children that are on therapy fall in really to sort
of 10 to 15 age bracket. And in 2018 we expect that as stay pretty consistent, we do get children
all over to different age groups, but when new born screenings really kicks in, you are talking just
hundreds of children, because to your point about average age of diagnostic is around hundreds
of children there un-diagnosed.
 The below table, shows the DMD cohorts and this 10-15 bracket.

Age 25 24 23 22 21 20 19 18 17 16 15 14 13 12 11 10 9 8 7 6 5 4 3 2 1 0 Totals
Non Ambulatory Ambulatory UnDiagnosed
Boys 405 572 322 1,299
On Drug (A) 200 200 0 400
Potential. Rev(M) 257(M) 229(M) 37(M) 543
15 – 10
Potential. Rev(M) 164(M) 242(M) 100(M) 37(M) 543
On Drug (B) 200 200
In cohort 121 250 322
Conversion Rate 49.38% 34.97% 0.00%

First off note, the total potential revenue is 543M, today. I have previously stated ETEP potential
revenue is in the 1B range. If these boys continue on drug, they get older, mature, live longer, new
births are added, then the 2025 potential revenue is 0.985B. Hence also, after GOLO and CASI have
been on the market about 5 years, the total potential revenue > 2.0B

So the average age on drug is about 13, this is the mean, I'm treating it roughly as the median (as Bo
stated the boys are all over the spectrum) and I realize I don't know the complete makeup of the boys
on drug, that's what I'm trying to infer and draw some conclusions about. I also realize the lines I've
drawn are not perfect definitions, but rather fuzzy.

1) The conversion of non ambulatory is about 50%; they have 405 in that group, they have about
200 on drug. That group is made up of older boys, many of which are likely quite advanced in
disease progression. So that # is not too surprising, nor do I expect it to increase dramatically,
until say a 10 YO boy today in +5 years is a 15YO. However I do expect some conversions in
that group, just not as rapidly as we are in the 1st year after approval.
2) The conversion on 10-13 YO is quite good, again my lines should be considered fuzzy, but
roughly 200 boys on drug out of a possible 250 is 80% conversion. This relies on Bo's
comment that the vast majority of boys are in the 10-15 cohort. BTW this is not a gotcha on
Bo's part, just trying to piece together the info to complete the picture.
3) There is this group of boys 5-9, where the real work is currently and conversions are likely
lower than I would like or expect. The boys have likely been diagnosed and need to be
genotyped to confirm they are ETEP amenable. This group of boys are still running and
jumping, perhaps just a little slower; educating the parents to start their boys before visible
disease progression sets in, is Bo's job. These are also parents that in the last couple years have
had their world turn upside down, recently been diagnosed and trying to understand what DMD
is. There is 1 approved Tx that is not a cure and has a high Tx burden, but also trials all over
the place and its very likely confusing and difficult to navigate. This is where PPMD (and
others) are so important; because, where are the parents of the newly diagnosed going to turn
when they try to figure out what to do.
4) The un-diagnosed group represent 322 toddlers and infants.
5) The total of not on drug; 0-13 YO is about 600 boys, about ½ those are diagnosed and
ambulatory (100M of potential revenue) and the other ½ are not even diagnosed yet(37M of
potential revenue). This represents the majority of the boys that need to be converted.
6) The 'slowdown', although I don't like that word, is a natural progression of SRPT attacking (or
doctors/pts moving) the middle cohort (10-15 which represents 242M/543M or 44% or potential
revenue) first. The older non ambulatory boys will still convert, but likely at a slower rate as
they have now have had 5 Qs to genotype and consider the effects and burden. The younger
 boys will very likely continue to convert as SRPT educates and doctors reach out, but even if
the same absolute # convert, they are the younger/smaller boys so in revenue terms it will look
'slower'. And finally, the non diagnosed is a function of educating doctors; it represents a very
small portion of revenue, but probably the most difficult to ID and reach.

I would also note, the compliance is high so the boys on drug are staying on drug. I would expect there
are some discontinuations; especially or particularly in the old boy cohort as some started late in the
progression. Also the burden of Tx, really stinks. I'm sure it is tough to do this every week, until a
PPMO (hopefully) or GT comes online.

So to summarize wrt the revenue ramp; the early adopters have been converted to drug mostly, the 10-
15 cohort is that group. The progression is visible and the parents have lived with DMD for many
years now and are knowledgeable of the options and the natural history, they are living it. Some older
boys will never convert as they are rather progressed and did not have this option when they were
younger. The young ambulatory (5-9 YO=322 boys) will likely continue to convert albeit at a slower
rate in revenue terms(as the pool is just smaller and they are just smaller boys) as parents come to terms
with the progression and become more comfortable with the Tx. The un-diagnosed(0-4 YO=433 boys)
obviously need to be diagnosed and/or as Bo stated, newborn genetic testing put in place, so
they(parents) can be educated and put on Tx before progression takes hold.

This is mostly looking at ETEP PMO revenue. Looking forward and using the same methodology,
GOLO has a potential revenue of 334M and 606M in 2025; CASI the same. These #s are all US and
do not include any EU considerations.

However, SRPT is pivoting to PPMO and GT. Other than GOLO and CASI, there are no more PMO
trials in their pipeline. They must feel very confident in PPMO as they have 6 trials planned which
would Tx 44% of the DMD population including cannibalizing some of their existing drugs. PPMO
offers a step change in pts burden and efficacy, at least that is my hope and belief. They spent a lot of
time and resources on it. So the calculus from a parents perspective will change dramatically if say
PPMO Txs is every 4 or 6 or 8 weeks, and maybe sheer volume of drug is less so a port is less needed.
So the conversion rate(and compliance) should be much more rapid with PPMO. With GT, I see a
similar quick conversion, however I still expect issues with GT in older boys and hence PPMO will still
be very relevant long into the future.