Rxi Pharmaceuticals (NASDAQ:RXII)
On June 9, 2015 shares of Rxi Pharmaceuticals went up 6% after the company reported that two new key patents were given to the company for use with their self-delivering sd-rxRNA technology platform. The new patents establish a few new details that enhance upon the company’s prospects:
First the new patent is for a chemical modification that allows for better systemic administration using the sd-rxRNA technology without the use of any delivery vehicle like other RNAi companies. Before Rxi could send its drug systemically but it had to do so at higher doses without a delivery vehicle or at normal doses with a delivery vehicle -standard lipid nanoparticle shield. Now the company can send multiple compounds systemically with lower doses and that creates a huge advantage for the platform
Secondly the patent was established for use in patients with the central nervous system –CNS — disorders. At this point its speculation but the CEO did mention at the Rxi Jefferies 2015 presentation on June 4, 2015 that the company was close with a collaboration deal with a company in the neurodegenerative space. This patent could potentially be specifically for that collaboration which the CEO mentioned which would be a deal similar to that of the MirImmune deal.
Finally the patent also established itself for diseases in the inflammatory and metabolic diseases areas. This is because Rxi received a patent for a protein known as MAP4K4
Most interesting is the fact that about a few weeks ago big institutional holders bought many shares of RXII common stock:
- On June 1, 2015 Empery Asset Management announced that it acquired about 4.5 million shares of RXII which is a 7.86% stake in the company
- On June 5, 2015 Frigate Ventures, LP announced that it acquired about 4.2 million shares of RXII which is a 6.9% stake in the company
- On June 5, 2015 Sabby Healthcare announced that it acquired 2.7 million shares of RXII which is a 4.2% stake in the company
- On June 8, 2015 Intracoastal Capital, LLC. announced that it acquired 3.4 million shares of RXII which is a 4.99% stake in the company
What this institutional buying signifies is that these institutional buyers believe in the long term story of the company. The company has several important catalysts coming in 2015 so investors should definitely keep an eye on this company.
Regeneron Pharmaceuticals (NASDAQ:REGN), Sanofi (NYSE:SNY), & Esperion Therapeutics (NASDAQ:ESPR)
Shares of Regeneron, Sanofi, and Esperion were all down on June 10, 2015 after an FDA panel had voted the previous day for an experimental drug. The vote was based on a drug from Regeneron and Sanofi known as Praluent which is a cholesterol-lowering type of a drug. The FDA panel voted to approve Praluent for patients with cholesterol, but the problem was that the panel only thought that the drug should be used for those patients with severe genetic defects in cholesterol.
The panel wanted more evidence for the greater population of cholesterol patients. More specifically the panel wanted the companies to perform larger long-term studies showing that the larger cholesterol patient population can reduce the risk of heart attacks by taking Praluent. While both companies didn’t get what they wanted from the FDA they should at least be happy that the panel voted 13-3 in favor of Praluent for the subset of cholesterol patients.
This Praluent drug is a new class of drugs known as PCSK9 inhibitors that are intended to help patients lower the levels of cholesterol in their bodies. The promise for this drug is the fact that some patients don’t respond to current standard of care treatments like statins. Even then some patients can’t even take statins because of the negative reactions that occur with these types of drugs. Praluent may the type of drug these patients need to help them lower cholesterol levels safely. The downside is that these heart studies that Regeneron and Sanofi need won’t be completed until 2017, so it will be a long wait until the drug is able to be branched out to other cholesterol patients in the future.
Esperion pretty much tanked down 20% that very same day because it was trading on the panel vote for Praluent as well. That is because if a non-statin drug like Praluent has to be tested for heart safety studies then more likely than not Esperion will also have to run large safety trials which will be very expensive for the company. Shares of Regeneron were down 5% on this news and shares of Sanofi were pretty much flat for the entire session.
Isis Pharmaceuticals (NASDAQ:ISIS)
On June 11, 2015 shares of Isis Pharmaceuticals were up 8% after the company reported positive phase 2 results in patients with Spinal Muscular Atrophy — SMA. This disease SMA is a rare genetic disease that affects the ability for patients to maintain the survival of motor neurons in the body. There are no treatments approved for SMA so there is a huge market opportunity in this space. Currently Isis is partnered with Biogen (NASDAQ:BIIB) for the SMA drug.
There were about 20 infants recruited in the clinical trial but only 19 were available for result analysis at the time of the efficacy testing. Patients in this clinical trial either took 6 mg of ISIS-SMNrx or 12 mg of ISIS-SMNrx. One key note to point out here is that this trial is not powered so much for efficacy because this trial was run with an active compound only and no placebo counterpart. Which is probably why many investors are questioning the efficacy of the results seen to date. The primary endpoint of the trial was event free survival in patients with SMA.
The results of the primary endpoint in median free survival events was met because of the positive clinical efficacy seen. Infants in the 6 mg group saw median-free events go from 16.3 months to 19.9 months. Infants in the 12 mg group saw median-free events go from 11.6 months to 16.7 months. In addition to this endpoint being met ISIS-SMNrx also met on other clinical trial endpoints as well such as muscle movement. Now the final phase 3 trial that should be run with a placebo compound will determine whether or not the FDA will ultimately approve the drug. Despite this Isis has a huge pipeline of drugs therefore it is still a great long-term name to own regardless.
Disclosure: Long Rxi Pharmaceuticals (RXII)
Terry writes about new and existing biotech companies in addition to big pharmaceutical companies that are innovating new treatments for many of today’s unmet medical needs. Terry has a large following on Stocktwits discussion pharmaceutical companies under the name “BiopharmaPro” http://stocktwits.com/BiopharmaPro
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