Biogen Idec (NASDAQ:BIIB)
Shares of Biogen soared as much as 10% on Friday March 20, 2015 after the company reported that it had achieved a positive outcome in an early phase 1b trial in patients with Alzheimer’s. Matter in fact many Wall Street analysts were expecting a weak efficacy outcome but Biogen’s drug BIIB037 went above and beyond the expected outcome. By that we mean that the drug was able to produce two positive clinical results in these patients with Alzheimer’s. Alzheimer’s is a cognitive disease whereby the memory deteriorates and other cognitive functions diminish over time. Alzheimer’s affects about 15 million people worldwide therefore getting a drug approved for this disease is still a major necessity.
The phase 1b trial enrolled about 166 patients and each of these patients were split up into five different dose groups. Four of the dose groups were with different dose increments of Biogen’s drug BIIB037 but the fifth and final dose group consisted of a placebo compound. Although one thing to note is that the highest efficacy achieved was with the highest dose group which included the 10 mg dose level. In this highest dose given to patients Biogen reported that their drug was not only able to reduce the cognitive decline that is associated with Alzheimer’s but in addition they were able to reduce the amount of amyloid plaque levels in the brain. With this phase 1b trial being successful though it puts Biogen right on track to possibly treat Alzheimer’s better than any other drug currently on the market.
It’s too early to be too excited for this trial as it only was a phase 1b trial and the patient population was small in comparison to other larger trials that treated Alzheimer’s patients in the past. Still the company had exceeded a lot of Wall Streets’ expectations and so the stock closed at about $476 per share. Biogen reported these results at the 12th International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders in France. The BIIB037 drug was well tolerated so the company doesn’t have to worry about having too much of a negative impact on these patients who desperately need this treatment. One can even say that upon successful completion of a late-stage Alzheimer’s trial with positive outcomes will be welcomed with open arms by both investors and Wall Street itself. The company plans to start a phase 3 trial using BIIB037 which will begin recruiting patients in late 2015. Although the trial being phase 3, the trial may last up until the year 2019 before any results are read out. So investors definitely have to be patient to see how these results play out, Still Biogen is in good shape as it has many other popular drugs such as Tecfidera which treats Multiple Sclerosis — MS– to keep it going for awhile.
Esperion Therapeutics (NASDAQ:ESPR)
Shares of Esperion soared 29% on Tuesday March 17, 2015 after the company reported positive phase 2b results for its drug ECT-1002 in combination with Statin therapy in a disease known as Hypercholesterolemia. Hypercholesterolemia is a disease whereby a person has too much cholesterol running through the bloodstream of their body. A person needs a moderate amount of cholesterol in the body to allow the body to produce hormones and aid in fat digestion in the gut as well. The problem is that these patients with Hypercholesterolemia have an abundance of cholesterol in the body which can lead to many debilitating problems with the heart. Such a problems would be patients getting heart disease which is often characterized by many different ailments such as: Heart attack, narrowing of blood vessels, stroke, and other heart related problems.
The phase 2b trial using the ECT-1002 drug in conjunction with Statin therapy was known as the ECT-1002-09 trial. The trial recruited approximately 134 patients who were randomized into three different dosing groups. The first dose group was 120mg of ECT-1002, 180mg of ECT-1002 or a placebo compound respectively. Patients in this clinical study were followed for about 12-weeks and then analyzed to determine how much of a cholesterol reduction they achieved depending upon what dosing group they were in. The results were extremely positive because Esperion’s drug of 120 mg and 180 mg both achieved a much higher efficacy outcome than placebo. ECT-1002 120 mg obtained a 17% reduction in LDL cholesterol, ECT-1002 180 mg obtained a 24% reduction in LDL cholesterol, and placebo only obtained a 4% reduction LDL cholesterol. By LDL cholesterol we mean bad cholesterol which is something that a person’s body doesn’t need a lot of. But alas the good news doesn’t stop there because Esperion reported a bonus news item. The company also reported a reduction of 30% in the high-sensitivity C-reactive protein hsCRP. You might ask what is a hsCRP protein ? Well the hsCRP protein is a marker of inflammation in coronary disease.
This positive result in phase 2 is important for many reasons but the main reason is that many patients either can’t take higher doses of Statins because of the associated side effects or sometimes the Statins don’t do anything to help them with their excessive cholesterol build up. Esperion Therapeutics has gone up roughly 49% since the phase 2b results were announced about a week ago and it seems that the momentum may continue for quite some time. ETC-1002 is an oral pill that can be taken once daily and doesn’t have very many negative side effects like other drugs that lower LDL cholesterol. This coupled with the fact that the drug has two positive functions will more than likely allow Esperion to beat out future competition in the high cholesterol drug space.
Amicus Therapeutics (NASDAQ:FOLD)
Shares of Amicus were up 30% on Thursday March 19, 2015 after the company stated that it had positive meetings with the FDA and EMA for its migalastat drug in treating Fabry disease. Fabry disease is an inherited disorder caused by a deficiency of an enzyme known as a-galactosidase A — a-Gal A which is responsible for degrading lipids in the body. This enzyme also causes a problem with an accumulation of globotriaosylceramide — GL3. When this acumulation of GL3 occurs it causes the very many symptoms that are associated with Fabry Disease. Such symptoms associated with Fabry disease are heart problems, pain, kidney failure. The disease is a rare disease so it has already received orphan drug designation from regulatory authorities in the U.S., Japan, and European Union.
In Europe the application for approval for a drug is known as receiving a marketing authorization application — MMA . Although The European Union assigns what’s called Rapporteurs who collectively analyze data and help companies with their next steps in the application for approval. This meeting in Europe with the Rapporteurs went so well that Amicus will be able to file its MMA earlier than expected by Q2 2015 instead of having to wait till the 2nd half of 2015.
The FDA on the other hand, here in the U.S., is a little more strict with its approval process. It really wants to approve migalastat because it treats Fabry disease which is an unmet medical need but it will still require that the company provide surrogate endpoints for approval. Surrogate endpoints are clinical endpoints established by the FDA for early efficacy measures to determine whether a drug should be approved early or not. In addition Amicus must satisfy the FDA with a confirmatory phase 4 clinical trial in order to market migalastat. The good news though is that the company can run this confirmatory phase 4 trial after approval so it doesn’t have to rush the trial in any way. The downside is that the FDA will not be able to meet with the company in a pre-NDA meeting until the 2nd half of 2015 which is a little ways off and will delay the actual NDA filing. In either case the company has received positive talks with both regulatory bodies so it seems more likely than not that both approvals should happen sometime in the future.