Replies to post #20246 on US Stem Cell Inc (USRM)

[BluSkies]

MYOCELL FLAGSHIP PRODUCT COULD BE FDA APPROVED SOON!!

Estimated Primary Completion Date: February 2017 (Final data collection date for primary outcome measure)

https://clinicaltrials.gov/ct2/show?term=BIOHEART&rank=3

[Mean Weimaraner]

Gm. Best get them bids set

[Dragon Lady]

Quote NO LOL, "FDA ACCELERATED APPROVAL & $USRM.

GUESS WHAT? WE DONT NEED TO COMPLETE PHASE 3 BOYS.
MYOCELL COULD BE COMMERCIALIZED AT THE END OF THE II/III STUDY WITH AN FDA FAST TRACK APPLICATION AND UPON APPROVAL IN MY OPINON.
IT'S A PIVOTAL STUDY: STRAIGHT OUT OF THE WORDS OF COMELLA IN THIS VIDEO. "

LOL, WHAT??

Uh....HOW EXACTLY WOULD THAT "work" supposedly, when BIOHEART, now USRM, NEVER even finished enrolling that trial, and thus never advanced it or completed it to its END FREAKING POINTS??? LOL ! HOW?

The "cures act" is NOT SOME DAMN MAGIC WAND, that's gonna get company's off the hook of needing ROBUST, LARGE SCALE, WELL CONDUCTED freaking CLINICAL TRIALS that are "FDA QUALITY" and thus need robust data, statistical analysis, end-points, PROVEN scientific efficacy, etc

The MARVEL TRIAL, never re-started/re-initiated to reach the needed "full enrollment" numbers, and thus was never completed per their OWN DAMN SEC FILINGS..(A clinical trial CAN NOT meet its "END POINTS" if the statistical needed patient "count" is never reached/enrolled and treated, that's IMPOSSIBLE per the science/statistical modeling they rely on)..Marvel was paused/halted in 2010 FOR LACK OF FUNDING among "other" problems/issues.

20 patient "treated" sample size (since that's NOT the end point design of the trial) it ain't gonna get a FDA APPROVAL FANTASY. AND...they had SERIOUS ADVERSE EVENTS in them there ole "HEART TRIALS", including PATIENT DEATHS and serious "HEART BEAT IRREGULARITY" issues, deemed to be CAUSED BY THE "stem cell" injections. OTHER COMPETITORS, as well as Bioheart, STOPPED or CANCELLED trials because of these "heart beat" problems, aka "heart arrhythmias", something that can cause HEART ATTACK and often DEATH of a patient.

Further, Myocell is NO LONGER EVEN PROTECTED UNDER PATENT, and USRM now lacks key licenses and rights to OTHER PEOPLE'S TECHNOLOGY they were, PAST TENSE, using to create and make "MYOCELL" and "MYOCELL SDF-1". SEC FILINGS...it's all in there.

https://www.sec.gov/Archives/edgar/data/1388319/000118518516003859/usstemcell10k123115.htm

FILED IN MARCH 2016, SEE ANY DATES PAST 2010 IN THAT "trial update" in that 10-K???? ANY??? NO. NO UPDATE. Says LACKING FUNDING FOR TRIALS. NEVER got past an initial 20 patients, which is NOT the trial "END POINTS", thus the trial is NOT COMPLETED or going to "BE COMPLETED SOON" as per DIS-INFORMATION being spread here on this msg board, and is NOT up for "FDA DAMN APPROVAL" per some "CURES ACT" freaking fantasy. NEVER EVEN SAYS THE FDA APPROVED THEIR "request for reducing the trial total enrollment size", and that was in March 2016.


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."

SAME 10-K, PAGE 8:

"MyoCell 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 addition, many of the patent and patent applications that have been licensed to us that pertain to our other product candidates do not cover certain countries within Europe."

SAME 10-K, PAGE 28:

"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."

SEE THAT, PLAIN ENGLISH, THEY DO NOT EVEN HOLD THE NEEDED DAMN "licenses" to the tech they WERE, PAST TENSE, "licensing" which is OWNED BY OTHER PEOPLE/COMPANIES that makes "MYOCELL" what it is. They can not use "MYOCELL" w/o OWNING/PAYING for or acquiring the damn "licenses" to the tech they need, which ALL LAPSED as of March 2016, less than 1 yr ago. READ IT.

https://www.sec.gov/Archives/edgar/data/1388319/000114544313000874/d30274.htm

From prior 10-K filing, PAGE 30:
"
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."

THINK THE FDA is gonna "APPROVE" some product with serious adverse events, including PATIENT DEATHS, based on a 20 patient sample size that NEVER MET FULL ENROLLMENT and NEVER PROGRESSED TO THE TRIAL END-POINTS? LOL, REALLY? All based on some fantasy blah, blah of the magic supposed "CURES ACT"???

TOTAL NONSENSE TO ME. BS. They never completed, or even began to re-enroll to reach the 170 or 150 or 130 patients needed for the MARVEL trial, FOR LACK OF FUNDING, and NOWHERE in any subsequent SEC FILINGS or "PR" have they claimed to have "FOUND FUNDING" and then re-started the trial, which would require hiring a outside CONTRACT FIRM among many other very, very expensive steps. They also never have claimed to have resolved the SERIOUS ADVERSE EVENTS and PATIENT DEATH issues with the FDA, not in any public filing I've ever read or seen.

The MARVEL trial is not due to be "completed" next freaking week IMO, total BS NONSENSE, and 100% in contradiction to their own detailed, damned SEC filing commentary, all listed above, cut n pasted word-for-word from their own DULY FILED SEC DOCUMENTS. Show ANY "newer" commentary by USRM Management to contradict what's listed above, some of it filed as recently as March of 2016. FIND IT and POST IT, else, BS TO ME.

[stervc]

Huge, with USRM & its pending FDA Approval...

I have a feeling that USRM is going to get FDA Fast Tack Approval approval soon. What they do save lives. This is a post, courtesy of TheHungryHippo, that all should read:

http://investorshub.advfn.com/boards/read_msg.aspx?message_id=128968613
FDA ACCELERATED APPROVAL & $USRM.

GUESS WHAT? WE DONT NEED TO COMPLETE PHASE 3 BOYS.
MYOCELL COULD BE COMMERCIALIZED AT THE END OF THE II/III STUDY WITH AN FDA FAST TRACK APPLICATION AND UPON APPROVAL IN MY OPINON.
IT'S A PIVOTAL STUDY: STRAIGHT OUT OF THE WORDS OF COMELLA IN THIS VIDEO.

Video about MYOCELL And Clinical Trials. MUST WATCH

"A PIVOTAL STUDY " Which means at the end of the study we could commercialize

Under the Cures Act, “regenerative medicine and advanced therapies” are defined so as to include gene therapy, gene-modified cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products that use these therapies or products

In a statement, Robert M. Califf, the FDA commissioner welcomed the act for establishing “a new program for the development of regenerative medicine products, an important and exciting new field that deserves this special focus.” Key elements in its provisions, Califf said, were an “enhance[d] … exchange of information among FDA, researchers and developers,” and the creation of a “research network and a public-private partnership to assist developers in generating definitive evidence” that could lead to quicker approval of therapies showing that they “indeed provide clinical benefits that are hoped for.”

The FDA will receive $500 million over 10 years to implement these programs.

Under the Cures Act, “regenerative medicine and advanced therapies” are defined so as to include gene therapy, gene-modified cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products that use these therapies or products, according to an “explainer” on the new law released by the Regulatory Affairs Professionals Society or RAPS.

In its current form, the Cures Act does not allow new stem cell therapies to forgo those Phase 3 trials, when many drugs fail. Instead, it says that regenerative medicine products, which include stem cell therapies, can be granted what’s called accelerated approval by the FDA.

Under that program, regulators can green-light a therapy without waiting on a demonstrated clinical benefit. Rather, they can rely on symptoms or other measures — what are called surrogate endpoints — that might indicate the drug is working; a cancer drug, for example, could be approved if it shrinks a tumor, even if it hasn’t shown yet it extends survival.

Treatments that go to market under accelerated approval are required to be studied further to ensure that they do in fact show a real benefit as they become available to more patients. The designation is typically used for therapies for serious or threatening conditions for which there are few or no treatment options, according to the FDA.

Potential upsides to Cures for stem cell research. One possible benefit of the Cures regenerative medicine provisions is that some high quality stem cell therapies that ultimately will be proven safe and effective would likely get to patients more quickly and help patients sooner. That’s a big deal. For biotech the major plus would be that they will have to spend less money to get therapies further in the trial pipeline. Since we as a field need stem cell biotechs to be successful in order for all of us collectively to help the most patients, the benefit for biotechs would likely yield some benefit for the field. Another possible plus here with Cures is the focus in the language on the need for standards for regenerative medicine, which have been lacking in the field.

Congress passes 21st Century Cures Act, boosting research and easing drug approvals

https://www.washingtonpost.com/news/powerpost/wp/2016/12/07/congress-passes-21st-century-cures-act-boosting-research-and-easing-drug-approvals/?utm_term=.51156066f53f


PREDICTION: FDA FAST TRACK APPROVAL NEWS COMING WILL SEND THIS STOCK INTO ORBIT.

v/r
Sterling