The FDA saw a liver related death in a small 18 patient ET trial where 90% of the participants experienced liver issues related to the test drug – imetelstat. Their only concern is how this will translate to a larger 200+ patient Phase II trial and what might they expect if the drug is eventually used in the general patient population.
The company's product Gencaro is an investigational and pharmacological unique beta-blocker and mild vasodilator. At present, there are no beta-blockers that have been approved by the U.S. Food and Drug Administration.
ARCA Biopharma President and Chief Executive Officer, Michael Bristow, said that if the development program of Gencaro would be successful, it would bring a new phase of pharmacogenetically developed cardiovascular treatment. As such, this would present a good opportunity for the company to improve the options available in the market today.
It is estimated that the company has IP monopoly with an estimated net present value of $15 billion. Even if a portion of these intellectual properties will be realized, this would be a blockbuster gain for the company relative to its current market capitalization of only $31 million.
The company does not generate revenues yet. It has net cash of around $21 million in the bank; equivalent to 68 percent of the market cap. Its annual cash burn of $8 million a year is something that investors should seriously consider. At that rate, the company has at least two and half years before they run out of cash.
While there are clear signs pointing to the enormous success of Gencaro, uncertainties over the outcome of the drug have weighed down on ARCA's share price. However, enterprising investors could ignore uncertainties and put more emphasis on the company's optionality. This is where the ten-baggers lie.
ARCA Biopharma (NASDAQ: ABIO), a company focused on producing genetically-targeted remedies for cardiovascular diseases, disclosed that the first patient has already been screened in its GENETIC-AF design clinical trial. The trial will evaluate its flagship product, Gencaro (generic: bucindolol hydrochloride) as a potential cure for the prevention of atrial fibrillation (NYSE:AF) in patients with heart failure and/or left ventricular dysfunction.
ARCA and Medtronic, Inc. (NYSE: MDT) sealed a collaboration in May 2013 to support GENETIC-AF clinical trial. According to the company, the clinical trial is a Phase 2B/3, multi-centered, randomized, double-blind clinical trial comparing Gencaro to Toprol-XL for the prevention of symptomatic AF in patients with heart failure.
According to the American Heart Association research, heart failure costs are estimated to double over the next 20 years in an aging population. Further studies from the same organization also show that AF incidence will also increase during the same period.
AF is a global epidemic cardiovascular disease, which increases the risk of stroke and contributes to heart failure. In a separate report, the global AF market is expected to reach an estimated value of $14.8 billion in 2019, or a compounded annual growth rate of 13.4 percent from 2013 to 2019.
Pharmacological treatment procedures are currently the preferred method of treatment. It is a low cost alternative to non-pharmacological procedures. Some of the top-tiered pharmaceutical companies have already floated pharmacological treatments in the market. This includes Bristol-Meyers Squibb (NYSE: BMY) and Merck & Co (NYSE: MRK).
We initiated screening of patients for GENETIC-AF in April 2014. We plan to activate approximately 50-60 clinical trial sites in the US and Canada for the Phase 2B portion of the trial. Currently there are 20 active clinical trial sites. We anticipate having the majority of the remaining clinical sites active by the end of 2014. During the second quarter of 2014 we made modifications to certain entry criteria and policies for the trial that we believe will facilitate patient enrollment.
GOOGLE "Jeremy Smith MPN" and click the first link that comes up. This is the guy who posts as jsandresen1 on Seeking Alpha. Read the article at the link and you'll see why this guy has a hard -- on for Dr. Tefferi.
The guy has been with seven companies over the last twenty years. Apparently he doesn’t last long at any of his jobs. Human Genome Sciences hired him as VP Strategy and Corporate Development in August 2011. He didn’t last long there and he didn’t last long at Geron.
This is great news. Looks like GERN is planning to run the new MF trial under the existing Mayo IND and not their own IND. The company's IND is on hold but Mayo's isn't. The goal is probably to get the FDA to agree that since the Mayo MF IND is not on hold, GERN should be allowed to go forward with the Phase II MF trial under that IND. Sounds like it might work.
Adam is technically correct. If the trial was statistically powered for 148 patients, the missing 8 patients is probably enough to make the results not statistically significant for the intent-to-treat population (148). Per protocol population means those who actually took the treatment (143). Good enough to move it to Phase III no doubt. But the FDA always looks at the intent-to-treat population. We need more data.
What you should really be embarrassed about is not knowing how to spell Kool-Aid.
That’s an excellent point. Scarlett has never indicated what set off the FDA hold in the first place. I simply assumed that as part of the GERN sponsored MF Phase II trial IND application, the company included the ET trail data and the FDA flagged it.
It’s possible that’s not how it came about. The company would have been required to report the patient’s death to the FDA because the ET trial was still ongoing. This may have been the cause of the FDA hold. This would be an excellent question to ask Scarlett or the company to clarify.
Don't know any shrinks in Little Rock, but do know a witch doctor outside Hot Springs. Specializes in voodoo cleansing rituals. Might be able to help with the cheese impaction.
Gentleman was sick and elderly with very complicated medical history. Had taken many prior treatments that could have affected his liver. Hepatitis = cirrhosis.
They had to say imetelstat was "possibly" related because it could not be 100% ruled out.