June 10, 2015
By Alex Keown and Riley McDermid, BioSpace.com Breaking News Staff
Genentech is currently enrolling patients in a Phase III trial for its drug Actemra, a day after the U.S. Food and Drug Administration (FDA) awarded Breakthrough Therapy Designation to the company to use the drug for systemic sclerosis, the company told BioSpace Wednesday.
The drugmaker is currently enrolling patients for the systemic sclerosis (NCT02453256) trial, spokesperson Allison Neves told BioSpace this morning. Although Actemra has previously been approved for treatment for rheumatoid arthritis, that does not necessarily mean the Phase III trial will be shortened, she said.
“The current RA indication does not speed up the trial but the breakthrough therapy designation does help expedite the review process once the Phase III data has been submitted to the FDA,” Neves said.
Actemra, a humanized interleukin-6 (IL-6) receptor for the treatment of adult patients with moderately to severely active rheumatoid antagonist arthritis, was approved by the FDA in 2010. In 2013 a subcutaneous formulation of Actemra was approved by the FDA for moderately to severely active rheumatoid arthritis. In all, the drug has been approved six times by the FDA.
The Breakthrough Therapy Designation is intended to expedite the development and review of medicines with early signals of potential clinical benefit in serious diseases and to help ensure patients have access to them as soon as possible.
“This Breakthrough Therapy Designation underscores the unmet need in systemic sclerosis and the potential of Actemra to help patients with this debilitating autoimmune disorder,” Sandra Horning, chief medical officer and head of global product development, said in a statement.
Systemic sclerosis is a chronic disorder characterized by blood vessel abnormalities, as well as degenerative changes and scarring in the skin, joints and internal organs. Systemic sclerosis affects approximately 75,000 to 100,000 people in the United States with the vast majority being women, primarily 30 to 50 years old. Systemic sclerosis has the highest mortality of any autoimmune rheumatic disease.
There is currently no FDA-approved treatment for systemic sclerosis. There are treatments for some aspects of this disease, but none are used to stop or reverse the key symptom of skin thickening and hardening.
Systemic sclerosis patient Stephen Elrod was diagnosed with the disease in 2007, putting his job as a firefighter in jeopardy and making basic tasks difficult. Elrod, who now sits on the board of the Scleroderma Foundation, was excited about the Breakthrough Therapy Designation.
“Not many people will go through what I went through and come back like I did but the research happening in this area is exciting. It gives hope to me and others who may be diagnosed with scleroderma,” Elrod said in a statement.
The Breakthrough Therapy Designation for Actemra was granted based on data from the Phase II faSScinate study. Swiss drugmaker Roche , the parent company of Genentech, will present new data from the U-ACT-EARLY and TENDER studies in patients with early rheumatoid arthritis, as well as results from the Phase II faSScinate study, at the annual congress of the European League Against Rheumatism this month.
That data will show that, while the primary endpoint of improvement in skin thickening at 24 weeks was not met, a meaningful trend was observed, the company said. In the second part of the study there was continued improvement in skin thickening between weeks 24 and 48
“The breadth of our study results at EULAR, ranging from arthritis in adults and children to a rare inflammatory disorder, underscores our commitment to helping people with debilitating autoimmune diseases,” Horning said.
When Will Pfizer’s Breakup Happen?
Speculation that the revamping of Pfizer Inc. ’s internal business structure could happen as soon as this year has biotech wondering just when this Big Pharma company could see changes.
Last week an analyst with J.P. Morgan said he thinks there will be a much faster timeline than most of Wall Street had predicted for Pfizer’s stated mission to refocus its efforts on new medicines.
Pfizer initially announced in 2012 that it would be shedding units that were non-essential to that goal. It then promptly sold its nutrition silo to Nestle for $11.85 billion, which was rapidly accompanied by a public spin-off of its animal health business for $2.2 billion.
“While a Pfizer break-up would likely be a 2017 event, we see potential catalysts in 2015-2016,” said Chris Schott, an analyst at J.P. Morgan. “Three years of audited financial statements (2014-2016) are required before any part of Pfizer can be spun off, and we also see 2017 as an attractive time for action as investors see Pfizer’s innovative pipeline clearly contributing to growth and the established business having transitioned to a more stable profile.”
BioSpace wants to know what you think: Will Pfizer be a changed company by the end of 2015?
