Roche Pharmaceutical Information

About Roche Pharmaceuticals

**** Copied verbatim from the Roche Pharmaceutical website. (Link in HELPFUL LINKS page) ****

• Headquartered in Basel, Switzerland, Roche is a leader in research-focused healthcare with combined strengths in pharmaceuticals and diagnostics. Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and neuroscience. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Roche’s personalized healthcare strategy aims at providing medicines and diagnostics that enable tangible  improvements in the health, quality of life and survival of patients. Founded in 1896, Roche has  been making important contributions to global health for more than a century. Twenty-four medicines developed by Roche are included in the World Health Organisation Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and chemotherapy.

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About Olesoxime

**** Copied verbatim from the Roche Pharmaceutical website. (Link in HELPFUL LINKS page) ****

• Olesoxime (TRO19622) is an investigational medicine designed to protect the health of motor nerve cells. Results of a pivotal phase II study of olesoxime in Type II and non-ambulatory Type III SMA patients from the ages of three to 25 years were first presented in April 2014 at the 66th American Academy of Neurology (AAN), Philadelphia, PA, USA. Trophos’s development program was supported by the French Muscular Dystrophy Association. Olesoxime has been granted ‘Orphan Medicinal Product’ designation for the treatment of SMA by the European Medicines Agency and orphan drug designation by the US Food and Drug Administration.

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Ongoing Clinical Trials

**** Copied verbatim from ClinicalTrials.gov website (Link in HELPFUL LINKS page) ****

• Investigate Safety, Tolerability, PK, PD and Efficacy of RO7034067in Infants With Type1 Spinal Muscular Atrophy (FIREFISH): Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD),and efficacy of RO7034067 in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts,an exploratory dose finding part (Part 1) and a confirmatorypart (Part 2) which will investigate RO7034067 for 24-monthsat the dose selected in Part 1.  The trial identifier is NCT02913482 BP39056  Link to the clinical trials website for this particular trial:  https://www.clinicaltrials.gov/ct2/show/NCT02913482?term=NCT02913482+BP39056&rank=1

Estimated Enrollment: 48 participants 

Actual Study Start Date:  December 24, 2016

Estimated Primary Completion Date: November 30, 2020

Estimated Study Completion Date:   January 1, 2021

Ages Eligible for Study:  1 Month to 7 Months (Child)

• A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants (SUNFISH): Multi-center, randomized, double-blind, placebo-controlled, Phase II study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RO7034067 in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1) of RO7034067 for 12 weeks and a confirmatory part (Part 2) of RO7034067 for 24 months.  The trial identifier is NCT02908685 BP39055  Link to the clinical trials website for this particular trial:  https://www.clinicaltrials.gov/ct2/show/NCT02908685?term=NCT02908685+BP39055&rank=1

Estimated Enrollment: 219 participants

Actual Study Start Date:  October 20, 2016

Estimated Primary Completion Date:  May 31, 2019

Estimated Study Completion Date:  July 16, 2020

Ages Eligible for Study:  2 Years to 25 Years (Child, Adult)

• A Study of RO7034067 in Adult and Pediatric Participants With Spinal Muscular Atrophy (Jewelfish): This is a multi-center, exploratory, non-comparative and open-label study to investigate the safety, tolerability, pharmacokinetic (PK) and PK/pharmacodynamic (PD) relationship of RO7034067 in adults and children with Type 2 and Type 3 Spinal Muscular Atrophy (SMA) who have been previously enrolled in a study with a survival of motor neuron 2 (SMN2)-targeting therapy.  The trial identifier is NCT03032172 BP39054  Link to the clinical trials website for this particular trial:  https://www.clinicaltrials.gov/ct2/show/NCT03032172?term=NCT03032172++BP39054&rank=1

Estimated Enrollment: 24 participants 

Actual Study Start Date:  March 3, 2017

Estimated Primary Completion Date:  April 5, 2019

Estimated Study Completion Date:  April 5, 2019

Ages Eligible for Study:  12 Years to 60 Years (Child, Adult)