ALS, Other Muscle Diseases Focus of Company‘s Push to Expand Drug Candidates

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

Share this article:

Share article via email
ALS and pollutants

Cytokinetics Inc. has presented its Vision 2020: Empowering Our Future – an initiative designed to expand the company’s pipeline until 2020, as well as advance its muscle biology-directed drug candidates toward late-stage development and marketing strategies, to tackle the unmet medical needs of people living with impaired muscle function including amyotrophic lateral aclerosis (ALS).

The late-stage biopharma Cytokinetics has dedicated its attention to discover, develop, and commercialize first-in-class muscle activators as potential treatments for debilitating diseases in which muscular performance is declining or compromised. The company’s lead drug candidate, tirasemtiv, is a fast skeletal muscle troponin activator for the potential treatment of ALS. Tirasemtiv has already been granted orphan drug and fast track designation by the U.S. Food and Drug Administration (FDA) and orphan medicinal product designation by the European Medicines Agency (EMA), all for the potential treatment of ALS.

The company is also developing CK-2127107, another fast skeletal muscle activator, for the treatment of spinal muscular atrophy (SMA).

Vision 2020: Empowering Our Future includes the following key goals:

  • Lead late-stage clinical development of novel, first-in-class muscle activators for the potential treatment of ALS, SMA, heart failure and other diseases affecting muscle function;
  • Advance proprietary research focused on muscle contractility, energetics and growth into development under new partnerships and collaborations;
  • Advance next-generation skeletal and cardiac muscle activator compounds into clinical development by enhancing existing research alliances;
  • Collaborate with patient communities to support the much-needed development of new medicines for diseases affecting muscle function;
  • Mature company operations to advance the development, registration and marketing of muscle biology drug candidates across Europe.

Until 2020, Cytokinetics envisions the company’s maturation into a commercial-ready organization through the expansion of its portfolio with proprietary and joint programs and multiple, first-in-class compounds for the treatment of diseases of impaired muscle function.

In 2016, the company expects to:

  • Complete patient enrollment of VITALITY-ALS, Cytokinetics’ ongoing Phase 3 clinical trial of tirasemtiv in people with ALS;
  • Complete patient enrollment in the recently initiated Phase 2 clinical trial of CK-2127107 in people with SMA, in collaboration with Astellas;
  • Further advance omecamtiv mecarbil into Phase III clinical development in patients with heart failure, in partnership with Amgen.

“We are entering a transformative phase for the company and our shareholders and Vision 2020 provides the road map to focus our team on our near and long-term opportunities,” Cytokintics President and CEO Robert I. Blum sais in a press release. “We believe our proprietary insights into the mechanics of muscle contractility and performance can translate over the next five years into advanced medicines that may provide meaningful contributions to the treatment of some of the most devastating diseases impacting patients’ lives.  Our commitment to maintaining a diverse portfolio of drug candidates while investing in a world-class research and discovery operation sets us apart and will afford us key advantages to achieve Vision 2020.”