Slide 1: PTC Q2 2018 Earnings and Corporate Update
PTC Q2 2018 Earnings and Corporate Update August 7, 2018 Aug-18 Page 1
Slide 2: Forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995
Forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 All statements, other than those of historical fact, contained in this presentation are forward-looking statements, including the information provided relating to our "2018 Guidance", including with
Slide 3: Delivering on our strategic vision
Delivering on our strategic vision VISION PTC is a fully integrated, innovative rare disorder company leveraging research capabilities and core technology platforms, building out world-class commercial capabilities, and being an ideal partner for late-stage, ultra-orphan disorders for which there is
Slide 4: Growing global DMD franchise
Growing global DMD franchise Translarna™ & Emflaza® DMD Net Sales ($ in millions) $260-295 $320 $280 In Q2 DMD $240 $174 franchise $200 grew 17% year $160 over year $120 $80 $40 $0 2014 2015 2016 2017* 2018E Aug-18 Page 4
Slide 5: Continuing to drive long term growth of DMD franchise
Continuing to drive long term growth of DMD franchise Approved EU label Earlier Increase disease expansion for diagnosis awareness Translarna™ in 2-5 year-old patients Strategy to bring Emflaza® to younger patients in the U.S. Improving Genotyping standards of care Aug-18 Page 5
Slide 6: Transformative transactions build out late stage and commercial pipeline
Transformative transactions build out late stage and commercial pipeline hATTR DMD *DMD Commercial Amyloidosis FCS AADC SMA Deficiency Late develop. Aniridia Dravet / CDKL5 Friedreich BMI1 Early develop. Ataxia Angelman DHODH 1 Syndrome Cognitive Preclinical Disorder HD FD DHODH 2 Gene Therapy 1 Emf
Slide 7: Agilis acquisition highlights
Agilis acquisition highlights CNS micro-dosing Immediate clinical Low doses of vector required Efficient, scalable Strategic partnership manufacturing manufacturing with MassBiologics capabilities as well as the Low manufacturing hurdles using existing systems Laboratories potential to expand
Slide 8: In-licensing opportunity of Tegsedi and Waylivra
In-licensing opportunity of TegsediTM and WaylivraTM Leverages PTC’s Latin America commercial infrastructure Diversifies our rare Waylivra launch: will utilize our disease portfolio patient support in Latin America and revenues Similar economic opportunity to Translarna in Latin America Tegsedi best
Slide 9: CHOP-INTEND total score
Cure SMA presentation showed motor muscle improvement and included video of 3 sitting SMN1 babies CHOP-INTEND Score: Individual Patient Plots Show Continuous Improvement from Baseline 40 Median change from baseline in CHOP-INTEND score was: Updated SMA data CHOP-INTEND total score 30 Aged 3-5 Aged 5
Slide 10: PTC's value creation opportunities
PTC’s value creation opportunities Establish Emflaza® as standard of care & grow TranslarnaTM globally Initiate launch activities for TegsediTM & WaylivraTM in Latin America SMA Firefish & Sunfish continued data for potential registration File BLA for AADC and IND for FA gene therapy programs Niche