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I have a Bachelors Degree In Business Administration. I have been investing in biotech stocks for many years, and I prefer to invest as a long term investor. With that In mind I seek stocks that have long term value! I primarily Like to Invest In biotechnology stocks and I accept the risks.
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  • Rxi Pharmaceutical's RXI-109 Does The Impossible Once Again!

    On Friday May 2nd 2014 Rxi Pharmaceuticals (OTC:RXII) announced results from a preclinical study involving studies of the eye in monkeys. Rxi pharmaceuticals is using RNAi to down regulate the expression of CTGF -- connective Tissue Growth Factor. RXI-109 is currently being used by Rxi Pharmaceuticals for anti-scarring against hypertrophic scars and keloids. There are zero FDA approved drugs for scarring and the possible market value for RXI-109 in scarring alone could yield $3 to $5.5 billion dollars.

    The results showed that the eyes of these monkeys were shown to reduce CTFG mRNA in a dose dependent manner in the Retina. That means that as the dosing was increased the subjects saw a greater gene knockdown of protein in the eye. These test subjects received an injection and the results were shown to be remarkable 7 days afterwards. What was most impressive was the fact that the company was only attempting this study as a dose range finding study, but instead noticed that RXI-109 was able to knock down the CTGF of the cornea as well. That's what made this press release spike Rxi Pharmaceutical's stock so high at 25%, because the company only expected to improve the retina but also improved the cornea as well. There was a selloff at the end of the day but it doesn't matter because these results bode well for the future of Rxi and its sd-rxRNA platform.

    We believe this selloff was due to investors being on the wrong end of the spectrum. These pre-clinical results that saw a huge surprise along with positive phase 1 multidose results in patients with scarring just validated the RXI-109 compound and sd-rxRNA platform even more. The company even claims in a quote from the CEO that the company can now possibly make a topical cream form of RXI-109 to improve retinal and cornea scarring of the eye seen here:

    "This finding opens up an avenue to possibly develop topical forms of RXI-109 to combat corneal scarring which often occurs secondary to trauma or infection and can lead to visual impairment including blindness"

    The best part about this study was the validation for RXI-109 because now Rxi Pharmaceuticals can move on to target other eye indications like PVR -- retinal detachment scarring of eye, Retinoblastoma -- eye cancer in children under the age of 15, macular degeneration -- vision loss in elderly, and any other possible eye candidate. As mentioned above RXI-109 is also being used for hypertrophic scars and keloids but may also show remarkable use in a big area many pharmaceutical companies want to target which is liver fibrosis. Yes RXI-109 can even be used for liver fibrosis which in itself can be a $1 to $2 billion dollar market.

    What validates Rxi's ophthalmology pipeline even more is the deal the company was able to achieve with Lincoln Park Capital. The deal called for LPC to purchase an aggregate of $20 million dollars worth or Rxi's common stock over a 30 month period. This purchase agreement was built to help fund Rxi's pre-clinical ophthalmology pipeline. LPC immediately purchased 500,000 shares of Rxi's common stock at $4.00 per share. Also when ever RXi reaches the ability to file an IND application for each ophthalmologic product LPC expects to purchase $1 million dollars worth of Rxi stock at prevailing market prices. For example that means if the share price at that later point is up in the $10 per share range or higher LPC has to purchase stock at that price. This huge capital flux is just another validation for the company and its ability to revolutionize the ophthalmology space and other unmet medical needs. With the eye indications along Rxi Pharmaceuticals could be looking to become the next Regeneron Pharmacetucals (NASDAQ:REGN) or even better as the company is also targeting liver fibrosis and scarring.

    Rxi Pharmaceuticals is expected release initial results from its phase 2a study of hypertrophic scars in the middle of 2014 which means it could come anytime now. A huge boost of RXI-109 over placebo with pictures would mean a game changing RNAi sd-rxRNA technology that could yield greater value and more licensing deals/partnerships. This Friday was the first time the company traded such high volume in one day coming in at 2.7 million shares traded for the day and with continuation of positive results we see greater share price appreciation in the coming days/months.

    Look out for this next week as tomorrow May 4th and May 8th will see Presentations at ARVO from Rxi Pharmaceuticals on their opthalmology pipeline. More importantly on Thursday May 8th will be a presentation on Retinoblastoma -- eye cancer in children under the age of 15 -- in which the presenter will announce what gene is being targeted for the disease in question. Rxi Pharmaceuticals is only getting started and we think the current share price at $3.39 per share severely undervalues the company for its massive potential. But we believe the phase 2 validation results in hypertrophic scars will greatly change that for sure!

    Disclosure: I am long RXII.

    May 03 9:02 AM | Link | 2 Comments
  • Three RNAi Companies Worth Watching In 2014

    There are three RNAi biotechnology stocks that are worth looking out for in 2014. These three biotechnology stocks are Rxi Pharmaceuticals (OTC:RXII), Tekmira Pharmaceuticals (NASDAQ:TKMR), and Arrowhead Research Corp (NASDAQ:ARWR). All these three companies have one thing in common, and that is that they are all RNAi stocks.

    RNAi -- RNA interference -- is the ability for siRNA molecules to be able to down regulate genes in a disease to stop the production of proteins from ever being created. The term siRNA means "small interfering RNA" molecules, whose sole job is to block genetic code. This halt in the production of proteins would then in turn cause a halt on the disease itself. There is however a key difference between these three RNAi biotechs. That difference is that they each have a different way of delivering the siRNA molecules to penetrate the cells surface. That means that each of these biotechs have developed their own delivery technology that sets one another apart.

    Rxi Pharmaceuticals

    Rxi Pharmaceuticals has developed a technology known as sd-rxRNA. We believe this technology has set a stronghold in the field of RNAi because the sd-rxRNA technology has been coined as a "self delivering" vehicle. This is because while all other RNAi companies have chosen to encapsulate their siRNA molecules into shields -- encapsulation of the molecule into a protective barrier -- scientists at Rxi Pharmaceuticals have figured out a way to deliver the RNAi oligonucleotide molecule to the cell without the need for a delivery vehicle. Thus far Rxi Pharmaceuticals tech relies on the patient receiving an injection into the skin for dermatological functions. Rxi hopes to achieve the same method of injection by injecting the RXI-109 compound directly intto the eye for its ophthalmologic indications. How this was accomplished is quite remarkable, and may provide a revolution in the field of RNAi medicine. What Rxi Pharmaceuticals has done is the ability to take the best prospects of RNAi and combine it with the best prospects of RNA. This combination of molecules forms a drug that can be sent through the patient's body to penetrate the targeted cell without degradation -- collapse of the molecule. With this safe and effective way to deliver RNAi molecules Rxi has chosen its first indication to be an anti-scarring drug known as RXI-109. This compound is currently in phase 2a clinical testing of patients with hypertrophic scars. The company expects to start two additional trials this year in 2014. One of the new trials will deal with keloid scars, and the other will deal with hypertrophic scars from breast reconstruction surgery. The final phase 1 multi-dose results for RXI-109 showed that it was able to reduce CTGF -- Connective Tissue Growth Factor -- mRNA by as much as 50%. One key thing to notice is that Rxi Pharmaceuticals had to use much lower dosing, and that is because with scarring you don't want the maximum potential to knock down the genes of greater than 50%. That is because you want at least some CTFG expression to occur so that patients aren't left with an open wound. Therefore many people confuse this result, because they assume 90% gene knockdown is needed to produce proper efficacy results. Rxi Pharmaceuticals expects to report preliminary phase 2a results in hypertrophic scars by mid 2014 as an update after patients receiving the RXI-109 compound for at least 3 months. Rxi Pharmaceuticals expects to have enough cash to last the company until Q2 of 2015 according to the 10-k sec filing. The current cash on the balance sheet is $12.4 million dollars. In truth Rxi Pharmaceuticals will have to either find a partner willing to give upfront money so that Rxi can advance its pipeline, or will have to do some form of dilution to raise more cash this year. The CEO recently spoke at the ROTH presentation on March 11th 2014, and said that Rxi Pharmaceuticals received interest from four big funds willing to buy Rxi shares at current market prices so the company can get an influx of cash to advance its ophthalmology pipeline in a private form of dilution shown on Rxi Presentation slides page 14.

    Tekmira Pharmaceuticals

    Tekmira Pharmaceuticals has developed its own technology to deliver siRNA molecules known as the lipid nanoparticle delivery system or LNP. Tekmira encapsulates the siRNA molecule with the LNP and then sends it systemically to reach the cells nucleus. There the siRNA molecule goes to work knocking down the genes of the disease in question. The good part about the LNP technology though is that it can efficiently deliver other nucleic acid molecules to the tissues of choice. Which is the reason why Tekmira Pharmaceuticals was able to create partnerships with other companies interested in the LNP technology. The reason for Tekmira Pharmaceuticals having to create the LNP technology is because these mRNA -- messenger RNA -- molecules are fragile so they are easily degraded by the human body. This is where RNAi failed in the past because the mRNA was unable to ever reach its target location, and even if it did reach there wasn't enough genetic material to accomplish the ability of silencing the genes. With the Lipid Nanoparticle delivery system from Tekmira Pharmaceuticals the company has been able to get the mRNA through the body with no degradation whatsoever. This means that a full mRNA molecule can reach the target tissue and down regulate the genetic code within the cells nucleus causing the disease to be diminished. Tekmira Pharmaceuticals lead product candidate is in phase I/II testing in solid tumors. This clinical trial is targeting GI-NET -- Gastrointestinal Neuroendocrine Tumors, and ACC -- Adrenocortical carcinoma. Tekmira is testing out these indications first and with positive results from this phase I/II trial the company will begin to test on patients with Hepatocellular Carcinoma or Primary liver cancer. Tekmira's lead oncology program is known as TKM-PLK1 because it targets a specific protein that is responsible for the disease known as polo-like kinase 1. The Protein PLK1 is responsible for cell proliferation -- cell dividing -- and has been a clinical cancer target for big pharmaceutical companies for many years. The results from the phase I study were very encouraging because 6 out of 15 patients -- 40% -- achieved clinical benefit. This clinical benefit was achieved with doses that were equal to and greater than 0.6 mg/kg. Breaking down the results further 3 out of 4 patients in the ACC indication achieved stable disease which indicates the potential target for PLK1. Two of the GI-NET patients enrolled showed clinical benefit as well. One GI-NET patient achieved a partial response, and the other GI-NET patient showed stable disease. TKM-PLK1 has the ability to show that RNAi can be used to target oncology indications, and positive phase I/II results should help validate Tekmira's platform even more. As of September 30, 2013 Tekmira Pharmaceuticals has cash and cash equivalents of $71.4 million dollars. This cash is sufficient to carry the company all the way until the early portion of 2016. One risk to note is that even with this cash on hand there is a possibility the company can dilute shareholders at an earlier time for an influx of additional operating cash.

    Arrowhead Research Corporation

    Like Tekmira Pharmaceuticals Arrowhead Research has developed its own delivery technology for siRNA molecules. Arrowhead's technology is known as Dynamic Polyconjugates or DPC. Arrowhead's DPC technology mimics viruses because of its ability to easily pick the right entry point to enter the cell successfully. Also for the fact that it can travel through the systemic portion of the human body without being harmed by the immune system. The main component of the DPC technology is the polymer shield that is an outside barrier responsible for encapsulating the siRNA payload molecule. This shield protects the siRNA payload molecule so it can reach its target effectively, and it also blocks the body from receiving any undue toxicity from the molecule itself. Once the DPC reaches inside into the cell's cytoplasm it releases the siRNA payload which results in the gene knockdown of the disease that is being targeted. This phenomena occurs because the endosome in the cell is able to break down the polymer shield of the DPC, which results in the releasing of the payload. The ability for the DPC to be only broken down in the endosomal portion of the cell compared to the systemic portion of the body is what makes this technology unique in its own right. With the DPC technology on hand Arrowhead is targeting the hepatitis b virus. The hepatitis b virus compound from Arrowhead is known as ARC-520. This compound though is looking to do something far different than just treating the hepatitis b virus. The company is looking to create a functional cure for the hepatitis b virus. That is to create a hepatitis b s-angiten that is responsible for creating an immune state to the hepatitis b virus. This s-antigen would be responsible for your body being able to cure the virus completely removing it from the body. In proof of concept preclinical studies it was shown that by using ARC-520, primates were shown to achieve a hepatitis b virus gene knockdown of 99% and greater. This pre-clinical result proved the mechanism of action for ARC-520 to be able to reduce HBV RNA proteins and viral DNA with a single injection. With these results Arrowhead has been cleared to initiate a phase 2 trial for ARC-520. Arrowhead was given the ability to begin its phase 2a single dose study in patients with the chronic hepatitis b virus in Hong Kong. The trial will evaluate patients with two single dose ascending dose levels. These two dose levels will be 1.0 mg/kg and 2.0 mg/kg the maximum dose tested in the phase 1 safety study. The company expects to have top-line phase 2a results for this study by Q3 2014. As of December 31, 2013 Arrowhead has cash and cash equivalents of $85.5 million dollars according to the 10q-SEC filing. This current cash is expected to carry the company for the next 12 months in which time investors should be aware of the risk of further dilution this year to increase operating cash flow.


    In closing we believe that these three RNAi companies should be watched closely by investors in 2014. They each have developed a platform that could help lead RNAi into a new revolution in the way we treat diseases. Both Tekmira Pharmaceuticals and Arrowhead Research Corp have each developed their own delivery platforms for RNAi therapeutics. In the case for Rxi Pharmaceuticals though, it has gone beyond the typical RNAi delivery, because it has developed a "self delivering" technology compound that can revolutionize the RNAi field. All these companies have great long term potential to become the next best thing in the biotechnology industry.

    Disclosure: I am long RXII. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

    Additional disclosure: no position in other stocks mentioned

    Mar 18 10:02 AM | Link | 4 Comments
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