In an effort to simplify the understanding of the 'value' created (likely) with some of these Gene Therapy(GT)

Treatments(Tx); I created this simple calculator more for fun than anything. I realize this is a really simple model, but
thought it would be good to have a go by or be able to get in the ball park, without a fancy spreadsheet. Some assumptions: I
assume no competition, the terminal phase assume it goes on forever, It is assumed a once and done treatment, I assumed
80% of the existing patients will eventually get GT and I assumed that would happen in a straight line 10 yr period. Also:

• i = discount rate = 10%

• t = terminal growth rate = 2%
• T = time to terminal growth= 10 years
• NM = Net Margins = 50% = most all models have this very high for GT (50% range)
• FCF = Rev * NM%; so ½ rev goes to bottom line and that's FCF(Free Cash Flow). Its not used below, but
effectively its FCF that the PV is derived from.
• I used 12.5 to determine the existing pts Txd each year vs 10 to account for only 80% of existing pts getting GT.

Present Value of this target:

PV = PV of FCF of bolus of existing + newborns for 10 yrs + PV of > 10 years annual FCF

PV = PV of level 10 years annual annuity of FCF + PV of the terminal FCF

PV =
((( Bolus /12.5)+newborns)∗$ Tx∗NM )∗(1−(1 /1+i)T )/i+(Newborns∗$ Tx∗NM )∗((1+t )/(i−t))/(1+ t)(T +1 )
putting i, t, T, NM in the formula and simplify. Could simplify further but keeping bolus and terminal stage separate.

PV = ((( Bolus /12.5)+newborns)∗$ Tx)∗3.0+(Newborns∗$ Tx)∗2.25

SMA with 21k WW existing and 900 WW newborns @ x $GT
DMD with 50k WW existing and 1350 WW newborns @ x $GT
LGMD with 20k WW existing and 500 WW newborns (swag) @ x $GT

Change the cells in green; generally leave the ones in red alone to calculate.

Existing Patients (at approval) 50,000

Newborns (annually) 1,350
Price of the once and done GT Tx (in Millions) 2.00

Present Value of bolus phase (Bs) 32.1

Present Value of terminal phase (Bs) 6.1
Total Present Value at approval (Bs) 38.2

SAMPLES ONLY
PV at approval (Bs) 1M / $GT 2M / $GT 4M / $GT
SMA 9.8 19.5 39.1
DMD 19.1 38.2 76.4
LGMD 7.4 14.9 29.7

As you may or may not know:

• SMA is comprised of 3 different phenotypes, only 1 of which (SMA-1) has the phenomenal data we've seen.
• The DMD micro Dystrophin construct will treat 100% of DMD. SRPTs current trial excludes a part of that
population to keep the trial population very homogeneous, like they do with the ages. My opinion is they have a
strategy to include all those other cohorts as they have stated before approval.
 • LGMD is comprised of more than 5 phenotypes, and the SRPT GT solutions are 5 different drugs. 3 of those are
very similar to the micro Dystrophin solution, so they are thought to a very much lower risk level. Those 3
comprise 2/3 population of the total of 5 solutions.

By the way, this was created as a fun/educational exercise, if you don't agree with some of the data, change it or tell me why
I'm wrong. It is not a dynamic formula, for example, the higher GT prices will likely result in me under estimating the
'value' as the NM will be too high. At any rate, it helps to see the ball park and see the value of being 1st to market to
capture the bolus.

Lastly if one wanted to ball park the SRPT share price with the above scenarios or any one you believe is the likely
outcome, then take the Total Present Value above and divide by 0.066 (66M shs) to get a rough idea).

GL