Cytokinetics / Astellas Pharmaceutical Information

LATEST UPDATE 

• I received a phone call from one of the doctors at Cytokinetics early last week. Apparently, the representative that I've been speaking with at Cytokinetics asked one of the doctors to call me to give me any updated information. Before we began our conversation, the doctor explained that this phase 2 clinical trial that was scheduled to end in February of this year is still in an active state because one or two of their patients are just now receiving their final dosing. Once they had received their final dosing, which should be no later than the end of March, they will be able to start gathering statistical data regarding this particular clinical trial, and they will make a determination at that point as to whether or not to move this to a phase 3 pivotal trial. The doctor went on to explain that if they did move this to a phase 3 pivotal trial, this will be the last opportunity they have to gather good statistical data to present to the FDA for final approval or denial for their potential treatment. The doctor I spoke with is actively involved with their phase 2 clinical trial. The first thing that the doctor told me was that the treatment we know as CK-2127107, now has an official name which is Reldesemtiv. The doctor explained that this drug works on the muscular level by getting the muscle to contract. When the muscle contracts, the nerve endings are exposed and when the nerve endings are exposed, this is when they absorb the protein that is needed to sustain and build muscle. Biogen's Spinraza works at the cellular level and their goal is to force the SMN2 gene to produce either more protein, or to produce a full protein. After explaining this, the doctor also stated that those of us that are currently on treatment with Spinraza should be able to register to see if we would qualify to participate in their phase 3 pivotal trial when and if it gets to that point. While the majority of us may not qualified to participate in this phase 3 pivotal trial because of the inclusion/exclusion criteria, we can at least speak to our doctors to get their opinions as to whether or not this pivotal trial would be right for us.

MY OPINION

• If Cytokinetic's drug Reldesemtiv works at the muscular level, and Biogen's treatment Spinraza works at the cellular level, this may be something that is beneficial for all of us. While the majority of us are getting good results with regards to our treatment with Spinraza, we also need to keep an open mind that the possibility of using another treatment along with Spinraza, may give us added benefits that could give us more of an opportunity to sustain and build muscle. Under no circumstance should any of us call Spinraza, or any other potential treatment that could be approved by the FDA a cure. Remember, Spinraza was never touted as a cure, it was given to us as a treatment. My belief is that if we can get one treatment to work in the muscular level, and one treatment to work at the cellular level, we will have the best of both worlds working for us so that we can maybe, someday, take back control of our bodies from this devastating disease.

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About Cytokinetics / Astellas Pharmaceuticals

**** Copied verbatim from the Wikipedia. (Link in HELPFUL LINKS page) ****

• Cytokinetics, Inc. is a publicly traded biopharmaceutical company based in South San Francisco, California, that develops muscle activators as potential treatments for people with diseases characterized by impaired or declining muscle function. 
• In collaboration with Astellas, Cytokinetics is also developing CK-2127107, a next-generation fast skeletal muscle troponin activator (FSTA). CK-2127107 has been granted orphan drug designation by the FDA for the potential treatment of SMA, or Spinal Muscular Atrophy. It is currently being studied in three Phase 2 clinical trials, one in spinal muscular atrophy (SMA), one in chronic obstructive pulmonary disease (COPD), and one in ALS, as well as a Phase 1b clinical trial in elderly adults with limited mobility. Astellas holds an exclusive license for the development and commercialization of CK-2127107 worldwide.

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About Cytokinetics / Astellas' PK-2127107

**** Copied verbatim from the Cure SMA website. (Link in HELPFUL LINKS page) ****

• Cytokinetics has opened enrollment for a Phase 2 study testing CK-2127107 in teens and adults with SMA type II, III or IV. We are excited to see the continued progress of this drug into the next phase of clinical trials, and particularly excited to see this trial focused on teens and adults. This latest trial announcement speaks to two of Cure SMA’s primary goals: pursuing a breadth of treatment options, and ensuring we have treatments for all types, all ages, and all stages of SMA.
• The clinical trial is designed to assess the effect of CK-2127107 on multiple measures of muscle function in both ambulatory and non-ambulatory patients with SMA, a severe, genetic neuromuscular disease that leads to debilitating muscle function and progressive, often fatal, muscle weakness. In collaboration with Astellas, Cytokinetics is developing CK-2127107 as a potential treatment for people living with SMA and certain other debilitating neuromuscular and non-neuromuscular diseases and conditions associated with skeletal muscle weakness and/or fatigue.
• The primary objective of this double-blind, randomized, placebo-controlled clinical trial is to determine the potential pharmacodynamic effects of a suspension formulation of CK-2127107 following multiple oral doses in patients with Type II, Type III, or Type IV SMA. Secondary objectives are to evaluate the safety, tolerability and pharmacokinetics of CK-2127107. The trial will enroll seventy-two patients in two sequential, ascending dose cohorts (two cohorts of 36 patients each, half ambulatory and half non-ambulatory). Each cohort will be stratified by ambulatory versus non-ambulatory status to receive CK-2127107 dosed twice daily for 8 weeks.
• “Initiating this first Phase 2 trial of CK-2127107 represents a major step forward given our interests to serve the many adolescents and adults who are living with SMA, a disorder with few treatment options,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “We look forward to working closely with the investigators and clinical trial sites to evaluate the effects of our next-generation skeletal muscle activator, which we believe holds promise for the potential treatment of patients battling this devastating disease.” 

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Ongoing Clinical Trials

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

• A Study of CK-2127107 in Patients With Spinal Muscular Atrophy: The primary objective of this study is to demonstrate a pharmacodynamic effect of CK-2127107 on measures of skeletal muscle function or fatigability in patients with Spinal Muscular Atrophy Types II-IV.  This is the first study being conducted in these patients and is designed to assess the effect of 8 weeks of dosing of CK-2127107 on measures of muscle function in both ambulatory and non-ambulatory patients with SMA. The plasma concentration of CK-2127107 will be measured at selected time points during the course of dosing and the plasma concentrations obtained in this study may be used to conduct exposure-response analysis. The trial identifier is  NCT02644668   Link to the clinical trials website for this particular trial:  https://clinicaltrials.gov/ct2/show/NCT02644668?term=CK-2127107&rank=4

Estimated Enrollment: 72 participants 

Actual Study Start Date:  December, 2015

Estimated Primary Completion Date: February, 2018

Estimated Study Completion Date:   February, 2018

Ages Eligible for Study:  12 years or older (Child, Adult, Senior)