Page 8 - 2020 Spring CMTA Report

HALTS ACE-083 TRIALS

cceleron Pharmaceuticals patients with neuromuscular cle in the lower leg involved in
announced March 9 that it diseases.” Dable thanked all of the ankle dorsiflexion (raising the
is discontinuing Phase 2 patients, families, caregivers and foot at the ankle). Part 1 was an
trials of ACE-083 in CMT investigators for their support and open-label, dose-escalation study,
A patients. While the drug participation. He also acknowl- with ACE-083 administered by
demonstrated an increase in mus- edged the Acceleron team’s “hard injection into the TA muscles
cle volume, those increases did not work and commitment to execut- of 18 patients once every three
translate to statistically significant ing robust Phase 2 trials that have weeks to evaluate safety and
improvements in any of the func- provided us the data necessary to increases in muscle volume over
tional or quality of make the difficult but a three-month treatment period.
life endpoints, such informed investment Part 2 was a randomized, double-
as muscle strength, Increases decision to discon- blind, placebo-controlled study
the company said. in muscle tinue the program.” using the optimal dose level
Habib Dable, volume did not Acceleron plans selected in Part 1. A total of 44
Acceleron president translate to to present results of patients were randomized and
and CEO, said in a the study at the next treated with either placebo or
statement, “Unfortu- functional American Academy of ACE-083 in Part 2 and were
nately, over the improvements. Neurology Annual evaluated for changes in muscle
course of multiple Meeting. volume, fat fraction, strength,
clinical trials, our The two-part function, quality of life and
myostatin-plus hypothesis has Phase 2 clinical trial was designed safety over a six-month primary
not resulted in the functional to evaluate ACE-083 in CMT treatment period, followed by a
outcomes necessary to provide patients with muscle weakness in six-month open-label treatment
clinically meaningful benefits for the tibialis anterior (TA), a mus- period. h

CMTA Advisory Board Member Clark Semmes, a participant in the ACE-083 trials,
shares his feelings at their discontinuation.

I AM A LAB RAT NO MORE…

began the clinical trial over a year ago when I received an invitation to participate from the
Neurology Department at the University of Pennsylvania, where my neurologist practices.
I I received four injections into the lower front of each leg every three weeks for a year.
In addition to the injections, I also gave blood and urine samples, received MRIs and had
my performance measured on various physical tests that included speed walking, running and
my ability to maintain my balance. I will not lie, the travel was frequently grueling and the running and speed walking often left
me sore and depleted, but the staff administering the clinical trial could not have been nicer or more accommodating.
While I was disappointed to hear the news that the drug was ineffectual and the clinical trial was ending, I have to admit I was
not completely surprised. Neither I, nor a handful of friends with CMT who were also in the trial, felt as though the injections we
received were making any life-changing impact. As my friend and fellow participant Angela Cretekos Dethloff said, “Kudos to all of
us for trying: lab rats, doctors, staff and even partners. We have to continue our search and efforts on something else.”
As we texted one another following the announcement, there was one emotion that we all felt and expressed, and that was
pride. I think I speak for all of us when I say I am proud to have taken part in this clinical trial. I am proud to have volunteered
my bent, but not broken, body in the name of science. I am proud to have stepped forward in the effort to find a treatment for
CMT. I am proud to have been a CMT lab rat. I would do it all again in a heartbeat.
I think it is important to remember that delay does not mean defeat. This was just one of the CMTA’s many research projects.
While this effort may not have hit pay dirt, we will, one day, find the gold mine. In the words of the late Senator Edward
Kennedy, “The work goes on, the cause endures, the hope still lives, and the dream shall never die.”

8 THE CMTA REPORT SPRING 2020