Research and Markets: Cytokinetics' CK-2017357 - Expanding the muscle contractility pipeline to skeletal muscle disorders
Research and Markets (http://www.researchandmarkets.com/research/9tctm2/cytokinetics) has announced the addition of the "Cytokinetics' CK-2017357 - Expanding the muscle contractility pipeline to skeletal muscle disorders" report to their offering.
Cytokinetics' CK-2017357 is a small molecule that improves skeletal muscle contraction. Although CK-2017357 does not increase the maximal force of contraction, it increases the response to weaker neuronal signals that cause disability in many neuromuscular diseases. CK-2017357 is now in phase II testing for two orphan neuromuscular diseases: amyotrophic lateral sclerosis (ALS) and myasthenia gravis, as well as a third indication, peripheral vascular disease with claudication.
In this report, we review the science, the rationale, the early evidence of activity, the emerging safety profile, the market opportunity across the various indications and the prospects for success in clinical development for this young and promising drug.
Key Topics Covered:
INTRODUCTION AND BACKGROUND
1. Neuromuscular diseases - a diverse medical need
2. A primer on skeletal muscle biology
CK-2017357, A FAST SKELETAL MUSCLE TROPONIN ACTIVATOR
1. CK-2017357 clinical indications
- Amyotrophic lateral sclerosis (ALS, also called Lou Gehrig's disease)
- Myasthenia gravis (MG)
- Peripheral artery disease (PAD) with claudication
1. Phase IIa - ALS (completed)
2. Phase IIa - PAD with claudication (ongoing)
3. Phase IIa - generalized MG (ongoing)
4. Upcoming studies
CLINICAL AND REGULATORY DISCUSSION
1. CK-2017357 has a novel mechanism of action with great clinical potential
2. Early clinical studies with CK-2017357 produced promising results
3. CK-2017357 appears safe but some adverse events are worrying
4. The impact of CK-2017357 on neuromuscular disease remains unknown
5. How might CK-2017357 fare in phase III?
1. Market opportunity in amyotrophic lateral sclerosis (ALS)
2. Market opportunity in myasthenia gravis (MG)
3. Market opportunity in peripheral artery disease (PAD) with claudication
4. Potential upside in other diseases
- Post-polio syndrome (PPS)
- Charcot-Marie-Tooth disease (CMT)
- Multiple sclerosis (MS)
- Guillain-Barré syndrome (GBS)
5. Market summary
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