May 12, 2017
By Alex Keown, BioSpace.com Breaking News Staff
LOS ANGELES – Shares of Capricor Therapeutics plunged nearly 60 percent in premarket trading after the company announced late Thursday it was laying off an unknown number of employees following the failure of a mid-stage cardiovascular drug trial.
Shares of Capricor closed at $3.05 on Thursday, but have fallen to $1.81 per share in wake of the reported failure of its Phase II Allstar trial. The 142-patient trial was assessing the efficacy of CAP-1002 (allogeneic cardiosphere-derived cells) in adults who have experienced a large heart attack with residual cardiac dysfunction. In its Thursday announcement, Capricor said the mid stage trial demonstrated a low probability of achieving a statistically-significant difference in the 12-month primary efficacy endpoint of percent change from baseline infarct size. Capricor performed a six-month data test and discovered there was no notable difference between the patients who received the treatment and those who received a placebo.
CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of cardiac progenitor cell.
Capricor said that at six months near-statistically-significant reduction of mean end-diastolic volume, as well as a trend of reduction of mean end-systolic volume, were seen in the CAP-1002 treatment group. However, the company said there was no notable difference between treatment groups with respect to the change in ejection fraction.
Following the failure, Capricor said it will continue to perform analysis of the data in order to understand the basis for the trial’s outcome, particularly since Phase I results were considered robust. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials.
With this failure, Capricor said it is being forced to tighten its belt financially, which includes the workforce reduction as well as a reduction in operations. Capricor said the cost-savings measures, which were not disclosed in its announcement, will allow the company to focus on CAP-1002 in in boys and young men with Duchenne muscular dystrophy (DMD).
In April, the company announced six month data from the Phase I/II DMD trial assessing the drug demonstrated statistically-significant improvement compared to control. Based on the trial results, Capricor said it plans to meet with the U.S. Food and Drug Administration to request Breakthrough Therapy or Regenerative Medicine Advanced Technology, or RMAT, designations for CAP-1002. The company also plans to initiate enrollment into a randomized, double-blind, placebo-controlled, repeat-dose clinical trial of intravenous CAP-1002 in DMD in the second half of 2017. The new trial will primarily evaluate skeletal (non-cardiac) muscle function.
Linda Marbán, president and chief executive officer of Capricor, said they were disappointed in the Allstar trial results, but said the favorable safety profile demonstrated in that trial supports the prospect of its chronic, repeat administration in patients with Duchenne muscular dystrophy.
“Also, the potent anti-inflammatory properties of CAP-1002 may be well-suited to mitigate DMD progression, for which chronic inflammation is believed to play a causative role,” Marbán said in a statement.
