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Abstract Details

Update of FORTITUDE-ALS: A Phase 2, Double-blind, Randomized, Placebo-Controlled, Study to Evaluate Efficacy, Safety and Tolerability of Reldesemtiv in Patients with Amyotrophic Lateral Sclerosis
Neuromuscular and Clinical Neurophysiology (EMG)
S5 - Therapeutics in ALS and SMA (1:11 PM-1:22 PM)
002

Reldesemtiv (CK-2127107), is a selective small molecule fast skeletal muscle troponin activator that sensitizes the sarcomere to Ca2+ by slowing Ca2+ release from troponin. Reldesemtiv increased the force generated by the tibialis anterior muscle versus placebo in response to nerve stimulation in healthy volunteers and has potential in the treatment of ALS. Derived from a different chemical scaffold and designed to limit crossing of the blood-brain barrier, it is expected to have fewer central nervous system adverse effects than tirasemtiv, a molecule with similar though less potent effects on fast skeletal muscle.

To evaluate the tolerability and preliminary efficacy of reldesemtiv in patients with amyotrophic lateral sclerosis (ALS).
Key inclusion criteria for FORTITUDE-ALS (Functional Outcomes in a Randomized Trial of Investigational Treatment with CK-2127107 to Understand Decline in Endpoints – in ALS) include diagnosis within 24 months and upright slow vital capacity (SVC) ≥60% of predicted. Participants are randomized 1:1:1:1 to placebo or reldesemtiv at 150 mg twice daily (BID), 300 mg BID, or 450 mg BID for 12 weeks. The primary endpoint is change from baseline in SVC at 12 weeks. Exploratory home-based outcomes (voice analysis and SVC) are also included. Safety and pharmacokinetic endpoints are included, as well as quantitative strength testing using hand held dynamometry and the ALS Functional Rating Scale-Revised. FORTITUDE-ALS is being conducted in the USA, Canada, Australia, Ireland, Spain, and the Netherlands.

FORTITUDE-ALS is currently ongoing but is expected to complete enrollment in November 2018.  Complete demographic information as well as top line efficacy and safety results will be presented.

Efficacy and tolerability data from FORTITUDE-ALS will be presented at the 71st Annual Meeting of the AAN. The results of this study will determine whether phase 3 studies of this compound should be conducted in ALS.
Authors/Disclosures
Jeremy M. Shefner, MD, PhD, FAAN (Barrow Neurological Institute)
PRESENTER 		Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Uniqure. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clene. Dr. Shefner has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cytokinetics. Dr. Shefner has received personal compensation in the range of $0-$499 for serving as a Consultant for Mitsubishi Tanabe Pharma America. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for annexon. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurosense. Dr. Shefner has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neuvivo. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nura BIo. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Prilenia. Dr. Shefner has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for swanbio. Dr. Shefner has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for vertex. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Shefner has received personal compensation in the range of $50,000-$99,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for UpToDate. The institution of Dr. Shefner has received research support from NIH. The institution of Dr. Shefner has received research support from Mitsubishi Tanabe Pharma America. The institution of Dr. Shefner has received research support from Amylyx. The institution of Dr. Shefner has received research support from BIogen. The institution of Dr. Shefner has received research support from Cytokinetics. The institution of Dr. Shefner has received research support from IONIS. The institution of Dr. Shefner has received research support from Healey Center for ALS. Dr. Shefner has received personal compensation in the range of $500-$4,999 for serving as a grant review chair with DOD- General Dynamics.
Jinsy Andrews, MD, FAAN (Columbia University Medical Center) Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Wave Life Sciences. Dr. Andrews has received personal compensation in the range of $0-$499 for serving as a Consultant for Cytokinetics. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Apellis . Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Revalasio . Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AL-S Pharma. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Quralis . Dr. Andrews has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for Neurosense. Dr. Andrews has received personal compensation in the range of $0-$499 for serving as an officer or member of the Board of Directors for Akava. The institution of Dr. Andrews has received research support from Amylyx. The institution of Dr. Andrews has received research support from Biogen . The institution of Dr. Andrews has received research support from Cytokinetics . The institution of Dr. Andrews has received research support from NIH . The institution of Dr. Andrews has received research support from Biohaven/Platform Trial. The institution of Dr. Andrews has received research support from Clene/Platform Trial . The institution of Dr. Andrews has received research support from Ra Pharma/Platform Trial . The institution of Dr. Andrews has received research support from Prilenia/Platform Trial . Dr. Andrews has received research support from ALS Association . Dr. Andrews has a non-compensated relationship as a Board of Trustee Member with ALS Association that is relevant to AAN interests or activities.
Angela L. Genge, MD (Mcgill University) Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AL-S Pharma. Dr. Genge has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amylyx. Dr. Genge has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Quralis. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA. Dr. Genge has received personal compensation in the range of $0-$499 for serving as a Consultant for WAVE. Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for eikonizo. Dr. Genge has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for pepgen. Dr. Genge has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for rapa.
Carlayne E. Jackson, MD, FAAN (University of Texas Health Science Center at San Antonio) Dr. Jackson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Mitsubishi Tanabe Pharma America. Dr. Jackson has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for AAN. The institution of Dr. Jackson has received research support from NIH. The institution of Dr. Jackson has received research support from Massachussetts General Hospital.
Noah Lechtzin, MD No disclosure on file
Timothy M. Miller, MD, PhD (Washington University) Dr. Miller has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Ionis Pharmaceuticals. Dr. Miller has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Disarm Therapeutics. Dr. Miller has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cytokinetics. Dr. Miller has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen IDEC. Dr. Miller has received intellectual property interests from a discovery or technology relating to health care. Dr. Miller has received intellectual property interests from a discovery or technology relating to health care.
Bettina M. Cockroft, MD, MBA No disclosure on file
Fady I. Malik (Cytokinetics) No disclosure on file
No disclosure on file
Andrew A. Wolff, MD (Cytokinetics, Inc.) No disclosure on file
Stacy A. Rudnicki, MD, FAAN Dr. Rudnicki has received personal compensation for serving as an employee of Cytokinetics. Dr. Rudnicki has stock in Cytokinetics.