US Stem Cell Inc (USRM)

[Dragon Lady]

Quote LOL, "USRM is already passed Phase 2 "

Well....NOT REALLY..GOTTA READ THEM SEC FILINGS IN DETAIL..??? Not sure why the post was reply to me either? I didn't ask what phase trial they are in??? I already know? And the "cures act" is NOT some freaking magic panacea that's gonna let companies skip the robust clinical trial process or need for IND's and lots of other complicated and expensive things.

USRM in recent SEC filings doesn't even discuss any major FDA trials (large scale, 100 patients or more) being active, they just keep talking about their little "clinic" biz new "revenue generating biz model" thingy they announced by CEO Tomas, the ole cash-for-treatments thingy, and training courses and a little vet thingy, which doesn't seem to be doing much or generating much revenue that I can tell? And top-line "revenue" has done nothing it seems to me, not that I can see from their most recent SEC filings and balance sheet and "financials" etc, to stem their losses, or stop liabilities from increasing in yr over yr from 2015 in their most recent 10-Q, or stop their need and continual use of toxic, convertible debt borrowing, and now receivables "FACTORING" borrowing at horrific rates in a time when the world is literally sitting at near ZERO interest rates, so much so that other companies like Apple are borrowing CASH to buy-back their own shares, or change their GOING CONCERN WARNING STATUS as noted by Sr Mgt AND their Auditors.... nope, all still looked pretty much the same to me essentially as of the most recent SEC filed 10-Q, only 3 short months ago.

OH, AND..Myocell is no longer even under patent protection and "key licenses" they need to even make/use what they call "Myocell", PER THEIR OWN DAMN SEC FILINGS, those licenses have "ALL LAPSED" as of the most recent SEC filing that discussed that matter. GOTTA UNDERSTAND, they didn't "invent" and/or 'OWN" all the tech that does what they tried to do with their "STEM CELL HEART CONCEPT", clear back to found Howard Leonhardt, they were LICENSING and purchasing PATENT RIGHTS to USE OTHER PEOPLE'S INVENTIONS/TECHNOLOGY to sorta "bolt together" what they "thought might work", and then putting names on it, like a trademark of their own, and calling it, "MYOCELL" for example. BUT, if the underlying patents lapse, or aren't paid-up, or key licenses lapse and aren't paid for etc, then you don't gets to use the goo, the "stuff" that makes what you call your "MYOCELL" or whatever. READ SEC FILINGS, page by page, COVER TO COVER. It's all explained in there.

They have a Phase II-a portion they completed in Europe and it's only a small sample size of 40 patients. (a smaller sample size is common at the phase II stage; that's why a good "FDA quality" robust phase II/III trial typically moves to at least 100 or more patients, often much more than 100 patients to get any real "statistical powering" to to them). The Phase II "a" portion is only to evaluate short-term safety of a drug . The 2nd part of a phase II, the "b" portion, is to confirm clinical efficacy of a drug and determine the therapeutic dose range.

So they never appear to have done the "b" portion that I'm aware of from their own SEC filed commentaries, which is sorta, kinda, freaking IMPORTANT, cause you gotta know the dosing ranges, and "efficacy", like does the damn thing safely work at WHAT GIVEN DOSE(s)?? One dose "might" prove to have some efficacy and another dose might KILL THE PATIENT or do nothing better than a placebo. That's what the whole damn robust and blinded trial process is about.

They also had some SERIOUS ADVERSE EVENTS in their trials, like PATIENT DEATH AND SERIOUS HEART ARRYTHMIAS (irregular heart beats caused by injection of their product into the patient's heart), which tends to sorta, kinda, like get the attention of the FDA IMO. They even halted trials for these problems and some competitors at the time, cause Bioheart hasn't done these freaking trials since the 2008 to 2010 period, NOT RECENTLY, thee ole FDA sorta needs real good data and science to explain them kinds of problems.

Most recent SEC 10-K, PAGE 5:

"We have completed various clinical trials for MyoCell including 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 U.S. Food and Drug Administration, or 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. We are planning, on the basis of these results, to request the FDA to consider the MARVEL Trial a pivotal trial (pivotal from Phase II to Phase III) and to reduce the number of patients in the trial to 150. No assurances can be provided that this request will be approved. We have also initiated the MIRROR trial, which is a Phase III, double-blind placebo controlled study for centers outside the United States. The SEISMIC, MYOHEART, MARVEL and MIRROR Trials have been designed to test the safety and efficacy of MyoCell in treating patients with severe, chronic damage to the heart. 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) called the REGEN trial, during the first quarter of 2010. Advancement of the MyoCell and MyoCell SDF-1 clinical development programs is contingent, among many factors, upon the Company obtaining access to sufficient funding to execute the necessary clinical trials to achieve proof of efficacy and regulatory authorization to market such products. The Company is also presently seeking a joint development partner for its MyoCell SDF-1 product candidate."

PAGE 28:

"Risks Related to Our Intellectual Property

We hold limited patent and other intellectual property rights, and our success will be dependent in large part on safeguarding our existing intellectual property rights and obtaining patent and other proprietary protection for our product candidates.

We hold limited patent rights in our product candidates. Our MyoCath product candidate is protected by a patent, expiring in September 2017, in which we have an irrevocable co-exclusive license. Our MyoCell product candidate is no longer protected by patents, which means that competitors will be free to sell products that incorporate the same or similar technologies that are used in MyoCell without infringing our patent rights. As a result, MyoCell, if approved for use, may be vulnerable to competition in the form of products that use the same or similar technologies. We have previously licensed certain patents and patent applications relating to our MyoCell product candidate. These licenses have all lapsed as of the date of this report, although we have had discussions with the relevant licensor regarding a potential reinstatement of our rights in such licenses.
"

https://www.sec.gov/Archives/edgar/data/1388319/000114544314000356/d31044-10k.htm

Prior 10-K, PAGE 31:

"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."

SEC FILINGS. THERE FOR A GOOD REASON, IMO. SUSPENDED TRIALS for irregular heart beat "problems" aka "arrhythmias", 6 DEATHS and 18 serious irregular heartbeats, aka "arrhythmia" problems (which of course can cause heart attack/death) 6 + 18 out of only a relatively small sample size of patients (there's some STATISTICS in there, gotta crunch them numbers). AND, competitors completely halted/abandoned trial(s) for same. Sorta important info IMO.

Good luck with the magic "CURES ACT" thingy....like it's just gonna sail on off into the sunset supposedly? ME, I don't think it changes much of anything. ROBUST TRIALS and ROBUST CLINICAL TRIAL DATA EVALUATIONS by the FDA and PATIENT SAFETY are still the underpinning of the ole "CURES ACT" and I don't see any changes related to that, NONE IMO.