Why battle? They know. Receive RMAT (FDA-Fastrack)&PPS SKYROCKETS!~SIMPLE On regulatory news, following the passing of the 21st Century Cures Act, U.S. Stem Cell, Inc. has applied to the FDA for Regenerative Advanced Therapy or RAT Designation. Thanks to the REGROW component of the Cures Act, The FDA will grant RAT designation for a regenerative medicine therapy that is intended to treat, modify, reverse, or cure a serious or life-threatening disease and demonstrates preliminary clinical evidence that the product has the potential to address unmet medical needs for a disease. We believe that our MyoCell® product meets these requirements as we have demonstrated clinical efficacy in both preclinical and clinical studies including our most recent MARVEL Phase II/III trial. If RAT designation is granted, this could expedite the approval process with the FDA. More information on the FDA’s new RAT Designation can be found here: https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm537670.htm
Quote NO LOL, "Wrong. Phase 2 has been completed. RMAT allows you to essentially go into commercialization, providing data as you go. Essentially skipping phase 3 "
NO, they never completed a full Phase II trial for Myocell. They WERE, PAST TENSE in the MARVEL trial, a phase II/III trial that stalled out in 2010 for LACK OF FUNDING. They have a phase IIa, which is HALF a phase II completed trial. Phase IIa gets safety/efficacy info, phase IIb is needed for DOSING, mega critical for any remote chance at commercial use. If you don't know what damn dose(s) to give a patient, then you ain't got squat. To low a dose probably does nothing, and to high a dose may kill um.
"Our most recent completed clinical trials of MyoCell are the SEISMIC Trial, a 40-patient, randomized, multicenter, controlled, Phase II-a study conducted in Europe and the MYOHEART Trial, a 20-patient, multicenter, Phase I dose-escalation trial conducted in the United States.
We were approved by the FDA, to proceed with a 330-patient, multicenter Phase II/III trial of MyoCell in North America and Europe, or the MARVEL Trial. We completed the MyoCell implantation procedure on the first patient in the MARVEL Trial on October 24, 2007. Thus far, 20 patients, including 6 control patients, have been treated. Initial results for the 20 patients were released at the Heart Failure Society of American meeting in September, 2009, showing a significant (35%) improvement in the 6 minute walk for those patients who were treated, and no improvement for those who received a placebo. On the basis of these results, we have applied for and received approval from the FDA to reduce the number of additional patients in the trial to 134, for a total of 154 patients.
The SEISMIC, MYOHEART and MARVEL Trials have been designed to test the safety and efficacy of MyoCell in treating patients with severe, chronic damage to the heart. Upon regulatory approval of MyoCell, we intend to generate revenue in the United States from the sale of MyoCell cell-culturing services for treatment of patients by qualified physicians.
We received approval from the FDA in July of 2009 to conduct a Phase I safety study on 15 patients of a combined therapy (Myocell with SDF-1), which we believe was the first approval of a study combining gene and cell therapies. We initially commenced work on this study, called the REGEN Trial, during the first quarter of 2010. We suspended activity on the trial in 2010 while seeking additional funding necessary to conduct the trial. We are seeking to secure sufficient funds to reinitiate enrollment in the MARVEL and REGEN trials. If we successfully secure such funds, we intend to re-engage a contract research organization, or CRO, investigators and certain suppliers to advance such trials. We have initiated and enrolled our first patient in the MIRROR trial in 2013. The trial is very similar to the MARVEL trial but focuses on sites outside the US. We will continue enrollment in the MIRROR trial once we have secured sufficient funds." 20 PATIENTS treated (they need somewhere on the order of 130 minimum), THAT IS IT. SO they ain't even remotely close to ever having reached "full enrollment" meaning no way in H they ever hit their endpoints or have complete data from that MARVEL TRIAL. NOPER.
ALSO, THEY HAD SERIOUS ADVERSE EVENTS IN THEIR HEART TRIALS IN THE PAST, so the idea this is some FDA "slam dunk" is pure BS.
"Our product candidates may never be commercialized due to unacceptable side effects and increased mortality that may be associated with such product candidates.
Possible side effects of our product candidates may be serious and life-threatening. A number of participants in our clinical trials of MyoCell have experienced serious adverse events potentially attributable to MyoCell, including six patient deaths and 18 patients experiencing irregular heartbeats. A serious adverse event is generally an event that results in significant medical consequences, such as hospitalization, disability or death, and must be reported to the FDA. The occurrence of any unacceptable serious adverse events during or after preclinical and clinical testing of our product candidates could temporarily delay or negate the possibility of regulatory approval of our product candidates and adversely affect our business. Both our trials and independent trials have reported the occurrence of irregular heartbeats in treated patients, a significant risk to patient safety. We and our competitors have also, at times, suspended trials studying the effects of myoblasts, at least temporarily, to assess the risk of irregular heartbeats, and it has been reported that one of our competitors studying the effect of myoblast implantation prematurely discontinued a study because of the high incidence of irregular heartbeats. While we believe irregular heartbeats may be manageable with the use of certain prophylactic measures including an ICD, and antiarrhythmic drug therapy, these risk management techniques may not prove to sufficiently reduce the risk of unacceptable side effects.
Although our early results suggest that patients treated with MyoCell do not face materially different health risks than heart failure patients with similar levels of damage to the heart who have not been treated with MyoCell, we are still in the process of seeking to demonstrate that our product candidates do not pose unacceptable health risks. We have not yet treated a sufficient number of patients to allow us to make a determination that serious unintended consequences will not occur."
THINK THE DAMN FDA doesn't know about the freaking PATIENT DEATHS and serious "irregular heartbeat" problems, aka "arrhythmias" which can induce heart attack or death, etc?
NOTHING about this RAT, or RMAT or "cure act" blah, blah, blah MITIGATES or REMOVES the FDA's requirement for ROBUST and COMPLETE CLINICAL TRIALS, NOTHING. It's a total fallacy being propagated here and other places. The FDA is gonna be just as rigorous as they have been in the past, and they've made public statements stating just that. "some" red tape gets removed to try and speed the process, but they're NOT gonna give a "free pass" on needing clinical, robust, scientific DATA and WELL CONSTRUCTED, STATISTICALLY RELEVANT CLINICAL TRIALS before they "approve" anything. THEN, the company would still need to fund and complete further trials for full approval.
WHICH PART OF THEIR $990 bucks of R&D spent in 2016 are they gonna use to fund a major clinical trial, LOL??? Or which part of their $270K TOTAL CASH LEFT ON-HAND will fund a major trial, a trial they COULD NOT GET FUNDED TO COMPLETION FOR THE PAST SEVEN DAMN YEARS, during the greatest bull market and "easy money" period in all of world history????
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