Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — Technical Presentation (January 2018)
18 slides from Ptc Therapeutics's technical presentation, Jan 29, 2018
Slide 1: FIREFISH: Safety and efficacy of RG7916 in babies with Type 1 SMA
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. FIREFISH, A MULTI-CENTER, OPEN-LABEL TRIAL TO INVESTIGATE THE SAFETY AND EFFICACY OF RG7916 IN BABIES WITH TYPE 1 SMA: STUDY UPDATE AND REAL-LIFE EXPERIENCE OF STUDY IMPLEMENTATION G Baranello1, J Day2, A Klein3, E Mercuri4, L Servais5, N Deconin
Slide 2: Disclosures
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. Disclosures GB is PI in the following clinical trials in SMA: BP39055 and BP39056 (Roche); CLMI070X2201 (Novartis); AVXS-101-CL-302 (Avexis); SMA- 001 (Catalyst); PI in the following clinical trials in Duchenne Muscular Dystrophy: FOR_DMD; DSC/14
Slide 3: Introduction
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. Introduction • SMA is a severe, progressive neuromuscular disease leading to loss of motor function and reduced life expectancy1 • Increasing evidence suggests that SMA may be a multi-system disorder, where cells and tissues throughout the body,
Slide 4: RG7916 mechanism of action
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. RG7916 mechanism of action SMN1 6 8 7 DNA Pre-mRNA mRNA Functional SMN protein 6 8 7 6 8 7 SMN2 6 8 7 6 8 7 6 8 7 6 8 7 6 8 Functional SMN protein Unstable SMN protein rapidly degraded RG7916 Pinard E, et al. J Med Chem; 2017;60:4444–4457. RG7916
Slide 5: FIREFISH: Study overview
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. *Comprises minimum two dose-ranging cohorts; Patients to be enrolled in a stepwise fashion based on PK findings to minimize exposure. https://clinicaltrials.gov/ct2/show/NCT02913482, accessed Jan 2018. FIREFISH: Study overview FIREFISH Type 1 SMA
Slide 6: FIREFISH: Outcome measures
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. Part 1 Part 2 Primary endpoint • Safety, tolerability, PK and PD of RG7916 • Dose selection for Part 2 % infants sitting without support for 5 seconds at 12- months assessed by Gross Motor Scale of the BSID-III Secondary endpoints • Motor functio
Slide 7: Patient demographics and baseline characteristics from the first 13 patients and study status
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. Patient demographics and baseline characteristics from the first 13 patients and study status IQR=interquartile range. Data current as of December 7, 2017. RG7916 is an investigational medicine and benefit/risk profile has not yet been fully esta
Slide 8: Age of patients and duration of exposure to treatment
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. Age of patients and duration of exposure to treatment Study duration is measured from start date of first dose to date of data extraction. Data current as of December 7, 2017. RG7916 is an investigational medicine and benefit/risk profile has not
Slide 9: No patient has lost the ability to swallow
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. No patient has lost the ability to swallow Data current as of December 7, 2017. RG7916 is an investigational medicine and benefit/risk profile has not yet been fully established. The information presented is from early interim analysis. Visit All
Slide 10: Summary of safety outcomes
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. Summary of safety outcomes • Overall 10 (77%) out of 13 patients experienced at least one adverse event. Most events were mild in intensity and resolved despite ongoing treatment • Adverse events reported in more than one patient were pyrexia (n=
Slide 11: Tips from real-life experience during the implementation of the FIREFISH study
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. • Working with patients with Type 1 SMA presents several challenges, mainly related to the age and the severity of the disease • Challenges can affect the infants, family, and staff involved in the studies • As SMA (and especially Type 1) is a ra
Slide 12: Tips from real-life experience during the implementation of the FIREFISH study
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. • Real-life experience from investigators must be used to best support patients and families during the FIREFISH trial • The importance of support from advocacy groups and patient/family organizations • Key considerations when conducting a study
Slide 13: Family relocation
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. • Families in countries where competing trials and therapies (eg, nusinersen) are not available may ask to be recruited to studies or to access therapies • Resource limitations and cultural differences • Relocation is important for the safety of
Slide 14: SMA standard of care implementation has increased survival and improved quality of life
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. SMA standard of care implementation has increased survival and improved quality of life of children and their families Increased survival Improved quality of life Rehabilitation Orthopedic Care/ Orthosis Psycho-social support Nutritional
Slide 15: Clinical trials involving relocation should ensure multidisciplinary management
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. • A proactive and anticipatory approach is essential to modify the course of the disease • Changing phenotype should be paralleled and supported by the implementation of standard of care (eg, postural control or standing frame as long as the chil
Slide 16: Conclusions
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. Conclusions • To date, RG7916 has been safe and well tolerated at all doses and there have been no drug-related safety findings leading to withdrawal in any SMA patients exposed to RG7916 • Ophthalmologic monitoring did not show any evidence of t
Slide 17: Acknowledgments
© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. We thank all the patients who participate in these studies and their families. We thank our collaborators PTC Therapeutics and SMA Foundation. We thank the FIREFISH, SUNFISH, and JEWELFISH investigators and trial staff. Acknowledgments
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