Regulus Therapeutics (NASDAQ:RGLS) & AstraZeneca (NYSE:AZN)
On April 7, 2015 Shares of Regulus were up about 8% in after hours trading after the company reported that Astrazeneca would like to collaborate with the company to develop a NASH drug. NASH stands for non-alcoholic steatohepatitis and as the name suggests it is a disease that doesn’t occur because of excessive alcohol use. What is NASH then? NASH is a fatty liver disease where deposits of fat builds up in the liver tissue causing scarring and other organ damage. The drug that will be used is known as RG-125 and it will be used to treat these NASH patients who additionally have pre-diabetes/type 2 diabetes.
According to a contract agreement back in 2012 Astrazeneca obtained rights from Regulus to be able to develop three potential areas using mRNA — messenger RNA — technology. The three key areas Astrazeneca chose were: Metabolic diseases, oncology, and cardiovascular diseases. Since Astrazeneca chose NASH it will now have the option for a third protein target. Why you ask? Well because the NASH drug uses two protein targets — both miR-103 and miR-107 respectively. With the agreement back in 2012 Regulus had received $31 million dollars. This was because at that time Regulus did its IPO for the stock market so they got $28 million for the IPO and then $3 million in an upfront payment for the deal.
With this news announced on April 7, 2015 Regulus earned an upfront payment of $2.5 million dollars from Astrazeneca, and in addition the potential to earn up to another $495.5 million dollars in milestone payments. Regulus will have the responsibility of initiating all pre-clinical trials for these product candidates, while Astrazeneca will be responsible for paying the late-stage trials and commercializing the products upon successful completion. Investors won’t have to wait long as data from the pre-clinical trial in NASH for RG-125 will be presented at an upcoming conference this year in 2015. Both Regulus and Astrazeneca expect to begin a phase 1 trial for RG-125 in NASH by the end of 2015.
Inovio Pharmaceuticals (NASDAQ:INO)
Shares of Inovio were up about 8% on April 8, 2015 after the company announced that it had received a $45 million dollar contract from the government to mass produce its ebola vaccine in large quantities. More specifically DARPA — Defense Advanced Research Projects Entity — gave Inovio $45 million dollars. The way it will work is that Inovio will provide its DNA technology and combine it with a monoclonal antibody entity to create a powerful vaccine to combat against the Ebola Virus. Medimmune which is a research and development arm of AstraZeneca will provide the monoclonal antibodies, while Inovio’s GeneOne/VGXI team will supplement the DNA vaccine.
So what is so special about Inovio’s DNA technology? Inovio is a revolutionary company because it creates something called Synthetic DNA vaccines. Believe it or not synthetic DNA vaccines are produced in water. The company utilizes a computer to create genetic code that it obtains from years of genetic research. It creates the Synthetic DNA genes to be closely similar to the disease in question but not exact — in this case ebola — and then formulates it into water. This makes the vaccine very safe. After all other vaccines currently marketed that treat a variety of diseases have a lot of toxic side effects.
The point of the DARPA agreement is that it will cover the costs of running the pre-clinical trials in addition to running phase 1 trials. Also the money will be used for manufacturing purposes to mass produce Inovio’s ebola DNA vaccine. Because Synthetic DNA can be formulated in water it makes the process of developing the vaccine fairly easy which means the company can mass produce it quickly in case of an ebola outbreak here in the U.S. or any other territory. Since the DNA vaccine is created synthetically it can be stored safely in any type of room temperature making transportation of the product fairly easy as well. Finally another good advantage for Inovio’s ebola vaccine is that it can be created to be a therapeutic — after ebola outbreak — or as a preventative vaccine — before an ebola outbreak — making it versatile in nature.
Tekmira Pharmaceuticals (NASDAQ:TKMR)
On April 10, 2015 the FDA announced that it had removed the partial clinical hold for Tekmira for its phase 1 TKM-Ebola drug trial. The FDA told the company though that it would only remove the partial clinical hold on the basis that the trial would be run with lower dosing only. That is because the TKM-ebola drug is being tested in healthy volunteers therefore it seems the FDA wanted to be more cautious before exposing these participants to inherent risks.
The phase 1 trial drug, known as TKM-Ebola, is Tekmira’s RNAi Ebola drug. Patients can only be given a maximum dose of 0.24 mg/kg per day. The patients in the study will either receive TKM-Ebola 0.24 mg/kg or a placebo compound and then followed for seven days after to determine safety of the RNAi drug compound. Tekmira expects that it will begin this phase 1 safety study in Ebola within a few weeks from now. Although the company may be able to show investors some safety results by the 2nd half of 2015.
Back in 2014 the FDA had placed the company’s phase 1 trial of TKM-Ebola on partial clinical hold because of safety concerns. One thing to note is that the TKM-Ebola drug didn’t cause any problems with patients in any trials but the FDA wanted to make sure that higher doses of the drug weren’t a danger to participants. Previously TKM-Ebola was only given to patients who had Ebola or who displayed symptoms of having Ebola. This time Tekmira can test patients who are healthy volunteers in a phase 1 trial.