July 15, 2015
By Mark Terry, BioSpace.com Breaking News Staff
South San Francisco-based Cytokinetics, Inc. , announced today that that The ALS Association had granted $1.5 million to the company in support of its Phase III clinical trial of tirasemtiv.
In addition to the grant, this sets up a collaboration between Cytokinetics, The ALS Association and the Barrow Neurological Institute. Patients enrolled in the clinical trial will also donate plasma samples that will be added to The Northeastern ALS Consortium (NEALS) Repository, which academic research groups access for research into identifying biomarkers that might be of use in assessing disease progression and research into the causes of ALS.
“We are grateful to The ALS Association and everyone who donated to the Ice Bucket Challenge for this grant to assist the funding of VITALITY-ALS and our collaboration with Barrow Neurological Institute for biomarker research,” said Robert Blum, president and chief executive officer of Cytokinetics in a statement. “This collaboration is an extraordinary opportunity to advance the development of tirasemtiv as well as scientific understanding of biomarkers that underlie the progression of ALS at the molecular level.”
The Ice Bucket Challenge was a social media phenomenon in the summer of 2014, in which people were challenged to either have a bucket of ice water dumped on their heads or donate money to The ALS Association. Social media, especially YouTube, Facebook and Twitter, were flooded with often amusing and occasionally painful videos of people having ice water poured over them. It was further driven by numerous celebrities undergoing the challenge in many creative and entertaining ways.
Although it isn’t entirely clear how many of the people in the videos also donated money, the challenge has, according to The Foundation, raised $115 million, and is being promoted again for this August.
ALS, or amyotrophic lateral sclerosis, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. As the voluntary muscle action deteriorates and motor neurons die, patients lose the ability to talk, eat, move and breathe. The disease is also known as Lou Gehrig’s disease, named after the famous baseball player who died from it. ALS affects about 20,000 people in the U.S. and usually affects individuals between the ages of 40 and 70. There is no cure and no treatments that extend life. Patients usually die within three to five years after diagnosis.
Tirasemtiv works by selectively activating the fast skeletal muscle troponin complex. In preclinical studies and early clinical trials, it showed an increase in skeletal muscle force. In a recent Phase IIb study, it appeared to cut the decline of slow vital capacity, which is a key measure of respiratory function in ALS patients.
Cytokinetics was initially a cancer drug biotech company, then switched to muscle research in 2007. The tirasemtiv program had problems in 2014 when a contractor error resulted in some patients being inadvertently shifted to the placebo group from the tirasemtiv group.
Although that Phase IIb study did not show statistical significance on a functional rating scale, about 178 patients on the drug showed a lower median rate of decline in “slow vital capacity” compared to the 210 patients on placebo. This particular measurement, which evaluates the maximum amount of air that can be exhaled slowly, is accepted by European drug regulators and the U.S. Food and Drug Administration (FDA) as an indication of an ALS drug’s effectiveness.
The Phase III clinical trial will take place in 80 medical centers in at least 10 countries.
Cytokinetics stock took the news fairly well. It traded for $6.01 on July 8, 2015 and is currently trading for $6.77. On Oct. 10, 2014, shares traded for $3.07, rose to $6.90 on Dec. 30, 2014 and hit a high of $8.17 on Feb. 17, 2015.
Ritu Baral of Cowen and Company, in a research note today, rated the Cytokinetics as “Outperform” with a $9 target. Baral also noted the company has a lead program in Phase IIb of omecamtiv for acute and chronic heart failure in a partnership with Amgen . It also had a Phase IIa study partnered with Astellas for spinal muscle atrophy.
Of tirasemtiv, Baral wrote, “Tirasemtiv, a skeletal troponin calcium sensitizer for ALS just entered Phase III. Phase I/II efficacy data was noisy, but generally shows some biological impact. … We think CYTK has general accord with the FDA on design of the pivotal Phase III VITALITY trial, which given the unmet need in ALS, may have a low bar for success. We see about 33 percent success for VITALITY given previous data and the generally poor track record of ALS trials.”
As New Jersey Biotech Booms, Will It Overtake Other States As Prime Location?
A week after Celgene Corporation announced it is officially the mystery buyer of Merck & Co. ’s former 1 million-square-foot R&D site in Summit, N.J., it quickly became our most popular story last week.
The company announced last Wednesday that it is buying the space, ending months of speculation about what Big Pharma company might move into the neighborhood.
The Summit, N.J. site is zoned research/office. The New Jersey site would put operations closer to some of the major biotech and pharmaceutical hubs on the East Coast.
But, by far, the most tempting part of doing business in the state remains New Jersey’s operating tax credit, which allows companies to sell their net operating losses to the New Jersey Treasury. One of the state’s most recognizable biotechs, Celgene, used the program until it became profitable, which was key to it staying in the state, said local officials.
That has BioSpace is wondering if New Jersey is becoming the new face of biotech. What do you think? Can the Garden State compete with other longtime stalwarts like California or Boston?
