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2020 JP Morgan Healthcare Conference Stuart W. Peltz, Ph.D., CEO
lookingstatement Th i s pr e s en tat i o n co n ta i ns for w ar d - lo o k in g s ta tem en t s wi t h in the m ean i ng o f
T he P r i va te S e c u r i ti e s Li ti g a ti o n R efor m Ac t o f 1995 . Al l s ta tem en t s c on ta i ned in t h is pr
e s en tat i on , o ther than s ta tem en ts of h i s tor i c f a c t, ar e for w ar d - l oo k i ng s t at em ent s , i nc l
ud i ng s t at em ent s r e l a ted to pr el i m i n ar y unau d i ted 2019 f i nan c i al i nfor m ati on wi t h r e sp e ct
to 2019 ne t pr odu c t r e v enue of T r ans l ar na f or the tr eatm ent of n mD MD and E MFL A Z A f or the tr eatm ent of
D u c he nne m u sc u l a r dy s tr o ph y , s tatem en t s wi t h r e sp e ct to 202 0 ne t pr odu c t r e v enue gu i da nc
e, s tatem ent s wi t h r e s pec t to the 2020 oper a t i ng e x pend i tur e gu i dan c e and s tatem en t s r egar di ng :
the futur e e x pe c tat i on s , p l a ns an d pr o s pec t s for PT C ; e x p e c ta ti o ns wi t h r e sp e ct to P T C' s
gene ther ap y p l atf or m , i n cl ud i n g an y p oten t i al r eg u l ator y s ubm i s s i on s and m an ufa c t ur i ng c
ap ab i l i t i es ; ad v a nc em en t o f P T C' s j o i n t c o l l abor a t i on pr ogr am in SM A, i ncl u d i n g an y p
o t e nti al r egu l a tor y s ubm i s s i on s , co m m e r c ia l i za t io n or r oy a l ty or m i le st o n e p a y m e nts
; PT C ' s e x p e ct a ti o n s wi t h r e s p ect to the l i c e ns i ng, r e gu l at or y s ubm i s s i on s a nd po tent i
a l co m m e r c ia l i za t io n o f T e gs e di and Wa y liv r a ; e x pa ns i on o f c om m er c i a l i z ati on o f T r ans
l ar na and E m fl a z a and r e l a ted r eg ul a tor y su b m i ssi o ns ; P T C' s s tr ateg y , fu tur e o per at i on s ,
fu tur e fi n a n ci al po s i t i on, fut ur e r e v enu es , pr o j e c ted cos ts ; and the o b j e cti v es of m anag em en
t . Ot her for w ar d - lo o k in g s ta tem en t s ma y be id e n t if ie d by th e wo r d s " gu i danc e " , "
p l a n," " a n ti ci p at e ," " b e l i ev e," " e s ti m a t e," " e x p e ct ,"
" i n tend ," "m a y, " " t a r ge t," " p ote n t i a l ," "w ill, " "
w o ul d ," " co ul d ," " sh ou l d , " " co nti n u e ," a nd s im ila r e x p r e ssi o
n s . P T C' s a c t u a l r e s u l t s, per for m an c e or a c h i ev e m e n ts c oul d d i ffer m a t e r ia lly fr om th
os e e x p r e ss e d or im p lie d by for w ar d - lo o ki n g s tatem en ts it m a ke s as a r e su l t of a v ar i ety of r
i s ks and u n c e r tai n ti es , i ncl u d i n g th o s e r e l a ted to : the ou tc om e o f pr i c i ng , c o v er age an
d re i mb u rs e me n t negot i at i on s wi t h t h ir d par t y pa y or s for P T C's pr o duc t s or pr odu c t c and
i date s that PT C co m m e r c ia l i ze s or ma y co m m e r c ia l i ze in t he fu tur e ; e x pe c tat i on s wi t h r e sp
e ct to P T C's gen e ther apy p l a tfor m , i ncl u di n g an y p o t e nti al r egu l ator y s ubm i s s i ons a nd p
o t e nti al appr ov a l s , m anu fa c t ur i ng c a p a bi l i ti e s and th e p o t e nti al fi n a n ci a l im p a ct and
bene f i t s of it s le a se d b i o l og i c s f a ci l i ty and th e po ten t i al a ch ie ve m e n t o f de v e l opm en t,
r egu l a tor y and sal e s m i l es to ne s and c on t i ngen t p a y m e nts tha t PT C m ay be ob l i g ated to m ak e ; th
e e nr o l l m en t, c ondu c t , and r es u l ts o f s tudi es un der th e SM A c o l l abor a t i on and ev ent s dur i ng ,
or a s a r e su l t of , the s tud i es tha t co u ld d e l ay or pr e v en t fur ther de v e l opm en t u nder the pr ogr am
, i ncl u d i n g an y poten ti al r egu l ator y s ubm i s s i ons and poten t i a l co m m e r ci al i z ati o n wi t h r eg
ar d s to r i sd ip la m ; P T C' s a b ili t y to c om p l ete a d y s tr oph i n s tud y nec e s s ar y to s upp or t a re -
s ubm i s s i on of it s T r ans l ar na NDA for the tr eatm en t of non s en s e m uta ti on D u c henne m us c u l ar d y s
tr oph y ( n mD MD ) to th e FD A , and P T C' s a b ilit y to per for m an y n e c e ss a r y add i t i ona l c lin ic a l tr
i al s , non - cl i n i ca l st u di e s , and CM C ass e ss m e nt s or a n al yses at s i gn i f i c ant co s t ; P T C' s a
b ilit y to m a i n t a i n i ts m ar k et i ng a uthor i z at i on of Tra n s l a rn a for the tr e atm en t of n mD MD in the
E ur opean E co n o m ic A r ea ( EE A) , i nc l ud i ng w h et her the E ur opean M e di ci n e s A gen c y ( EM A) de ter m
i ne s in f ut ur e annu a l r e n ew a l cy c l es that the be nef i t - ri s k ba l an c e of T r an s l ar na au thor i z at
i on s u ppor t s r ene w a l o f su ch a uthor i z at i on ; P T C' s a b ilit y to e n r ol l , fund , co m p le t e a nd ti
m el y s ubm i t to the EM A the r e su lt s of S tu dy 041 , a r an dom i z ed , 18 - m ont h, p l ac ebo - c ontr o l l ed c
lin ic a l t r ia l o f T r ans l ar na for the tr e atm en t of n mD MD fo l l o w ed b y an 18 - m onth o pen - l abe l e x
t e n s i o n, wh i ch is a sp e ci fi c ob l i ga t i on to c on t i nued m ar k et i ng au thor i z at i on in th e EE A ; ex
p e ct a ti o ns wi t h r es pe c t to the co m m e r c ia l i za t io n of T eg s ed i and Wa y liv r a ; si g n i f i ca n t
b us i nes s e f f e c ts, i nc l ud i ng the ef f e c ts o f i nd us tr y , m ar k et , ec onom i c , p o l i ti cal or r e gu
l a tor y c ond i t i on s ; c hange s in tax an d o ther la ws , r e g ul a ti o ns , r ate s and p o l i ci es ; the e li g
ib le p a ti e nt b a se and co m m e r c ia l p o t e nti al of T r an s l ar n a, E m f l az a, P TC - AAD C , T e g s e d i
, Wa y liv r a , r i sd ip la m or an y of P T C' s ot her pr odu c t c and i d ates ; P T C' s sc i e nti fi c appr oa c h an
d gen er a l de v e l opm en t pr ogr e s s ; P T C's a b il it y to s at i s fy it s ob l i gati on s under t he ter m
s of th e le a se agr eem ent f or it s l ea s ed b io lo g ics f a ci l i ty ; P T C' s a b ilit y to s ati s fy i ts ob l i
gat i on s under the t e rms of the se n io r s ec ur ed ter m l oan f a c ilit y wi t h M id C a p F in a n ci a l ; the su f
fi ci e nc y o f P T C' s cas h r e s o u r c es an d i ts a b il it y to ob ta i n adequate f i nan c i ng in th e fut ur e f
or it s for e s ee ab l e and un for e s eeab l e o per at i ng ex p e n s e s and c ap i ta l e x p end i tur e s ; a nd the
fa c tor s d i sc u ss e d in the "Ris k F a c tor s " se cti o n of P T C' s m o st r ec ent Quar t er l y R e por
t o n Fo r m 10 - Q and A nnua l R e por t on Fo r m 1 0 - K, as we l l as an y updat es to th e s e r i sk fa c tor s f i le
d fr om t im e to t im e in P T C' s o ther f ili n g s wi t h the SEC . Yo u a r e ur ge d to car ef ul l y co nsi d e r a ll
such facto r s . As wi t h any ph ar m ac eu t i c a l und er de v e l opm en t, ther e ar e si gn i fi c a nt r i sk s in the
de v e l opm en t, r e gu l a tor y appr o v a l a nd c om m er c i a l i z at i on o f ne w pr odu c ts . T her e a re no gu
ar antee s t hat an y pr odu c t w ill r e ce i ve or m a i n t a i n r egu l ator y appr o v a l in an y t er r i tor y , or
p r o ve to be co m m e r ci al l y s ucc ess f u l , i ncl u d i n g T r an s l ar n a, E m f l az a, PT C - AAD C , T e gse
d i , Wa y liv r a o r r i sd i p l am . T he for w ar d - l o o ki n g s ta tem ent s c on ta i ne d her e i n r epr e s en t
P T C' s vi e ws on l y as of the d ate o f th i s pr e s en tat i on an d PT C doe s no t und er ta k e or p la n to upd ate
or r e v i s e any suc h for w ar d - l o o ki n g s ta tem ent s to r e fl e c t a c tua l r e s u l ts or c h ange s in pl a
ns , p r o s p ec ts, as s um pt i on s , es ti m at es or p r o j ec ti o n s, or other c ir cu m st a n ce s o c cu r r in g
after the d ate of thi s pr ese nt a ti o n e x ce pt as r e q ui r ed b y la w . 2
global, commercial, diversified, biopharmaceutical company focused on innovative therapies for rare genetic disorders 3
commercial capabilities & infrastructure Offices in 20 countries Footprint in >50 countries 800+ employees 4
financial performance supports future growth ~$306M 2019 Unaudited Net Revenue New product launches and approvals could also contribute
2020 revenue to $291M 2019 DMD Franchise Net Product Unaudited Net Revenue (e.g.,Tegsedi, commercial Waylivra EAP, PTC-AADC and
risdiplam) $320-$340M 2020 DMD Franchise Net Product Revenue Guidance $545-$575M 2020 Opex Guidance PTC Therapeutics
Provides Corporate Update and Highlights Pipeline Progress at 2020 J.P. Morgan Healthcare Conference 5
success-based revenue through remaining risdiplam milestones and royalties Total remaining regulatory milestone-based payments
Potential 2020 risdiplam milestone payments to PTC $72.5M Milestone Payment Up to NDA Filing in Japan $ 7,500,000 mid-teen royalties
Total sales-threshold-based payments Annual, tiered potential royalties-based on net sales PTC Therapeutics Provides Corporate
Update and Highlights Pipeline Progress at 2020 J.P. Morgan Healthcare Conference 6 First US Commercial Sale$ 20,000,000 Total$
42,500,000 MAA Filing with EMA$ 15,000,000
approach builds diversified pipeline SCIENTIFIC PLATFORMS & RESEARCH * Investigator-initiated study with NYU 7 Potential registrational
studies Commercial SMA AADC Clinical HD FA PTC857 GBA-PD Angelman Undisclosed IRDs Research Cog Disorders Undisclosed PTC299 AML
PTC596 LMS PTC596 DIPG PTC743 MEDS PTC743 FA US Dystrophin Aniridia CDKL5/Dravet* LGMD2i Oncology Deflazacort Nonsense Mutation
LatAm Commercial Redox Gene Therapy Splicing
Sustainable innovation drives continuous value creation 2023 DMD Franchise, PTC-AADC, risdiplam, Tegsedi ,
Waylivra 2020* DMD Franchise (risdiplam, Tegsedi , Waylivra , PTC-AADC) REVENUE Beyond 2023 Nonsense Mutation Gene
Therapy Splicing Redox 2019 DMD Franchise (unaudited) $1.5B 2016 Translarna $320 - $340M 2015 Translarna $291M
$81M $34M Angelman, ataluren (aniridia), FA, GBA-PD, HD, LGMD2i, MEDS, PTC-FA, undisclosed redox, gene therapy, and splicing Angelman,
ataluren (US DMD, aniridia), FA, GBA-PD, HD, LGMD2i, MEDS, PTC-FA Emflaza HD, PTC-AADC, PTC-FA, risdiplam, HD, risdiplam
Risdiplam Tegsedi , Waylivra INNOVATION 8
Multiple potential value driving 1Q20 events in 2020 4Q19 2Q20 3Q20 4Q20 ales through early access
program Clinical Regulatory Commercial 9 Analyst Day Nonsense Mutation Aniridia data US dystrophin data Splicing Risdiplam NDA
filed SUNFISH Part 2 topline SUNFISH Part 2 data FIREFISH Part 2 topline Risdiplam MAA filing Risdiplam PDUFA Risdiplam US launch
HD IND filed Gene Therapy AADC MAA submitted AADC BLA submitted FA enters clinic Redox Initiate PTC743 MEDS trial Initiate PTC857
Ph1 trial Initiate PTC743 FA trial LGMD2i data ANVISA approval hATTR launch FCS s ANVISA approval
Multiplatform approach builds diversified pipeline SCIENTIFIC PLATFORMS & RESEARCH * Investigator-initiated
study with NYU 1 0 Potential registrational studies Commercial SMA AADC Clinical HD FA PTC857 GBA-PD Angelman Undisclosed IRDs
Research Cog Disorders Undisclosed PTC299 AML PTC596 LMS PTC596 DIPG PTC743 MEDS PTC743 FA US Dystrophin Aniridia CDKL5/Dravet*
LGMD2i Oncology Deflazacort Nonsense Mutation LatAm Commercial Redox Gene Therapy Splicing
Translarna demonstrates long-term benefit in DMD patients EU5 nmDMD patients treated with Translarna
Compliance STRIDE is a real-world, long-term registry of patients receiving Translarna Translarna treatment slowed disease progression
in nmDMD compared to matched natural history patients 2.2% Translarna-treated pts had an FVC<50% compared to 32.1% 3 years
Extension in ability to stand from supine in less than 5 seconds 3.5 years Delay in loss of ambulation 1 1
Growth opportunities in DMD franchise Translarna - Increased penetration in existing territories