5 Bio-Pharmas On The Rise With Potential Groundbreaking And Lifesaving Treatments

by: StockMatusow

A lot of BioPharmas that have been attracting a lot of attention lately that have potential ground-breaking and lifesaving treatments. These companies offer what I feel are cutting edge technological advancements in medicine.

The companies mentioned here might have groundbreaking treatments and potential cures for Duchenne muscular dystrophy, Aplastic anemia, rare Leukemias, and age-related macular degeneration.

These are serious ailments in our society and many biotechs have been working hard to advance medicine world-wide. For this write-up, let's take a look at a few of these companies that have been making serious strides that investors might find worth-while to invest in.

Pluristem Therapeutics Inc. (NASDAQ:PSTI)

Pluristem is a developer of placenta-based cell therapies. The company's patented PLX (PLacental eXpanded) cells are a drug delivery platform that releases a cocktail of therapeutic proteins in response to a host of local and systemic inflammatory and ischemic diseases.

The company's main technology, The PluriX Bioreactor System is designed to be a system of stromal cell cultures and substrates that create an artificial physiological environment where adherent stromal cells (ASCs) can grow and reproduce outside of the human body. Unlike conventional two-dimensional (2D) culturing methods, the company's PluriX Bioreactor creates a three-dimensional (3D) microenvironment that closely resembles the structure and function of the body's bone marrow environment. By mimicking the natural environment that exists within human bones, the system tricks stem cells into growing and reproducing in the same way they would in living organs. Because the size and scale of the PluriX Bioreactor is larger than that of human bone marrow, stem cell growth can be greatly expanded.

On August 6th, the company announced that the life of a patient suffering from bone marrow failure in which there was a dangerous reduction in the number of red blood cells, white blood cells, and platelets (pancytopenia) has been saved using Placental eXpanded cells. This is the second time in the past three months that a patient suffering from bone marrow failure was successfully treated in a compassionate use treatment with PLX cells with a return of bone marrow function.

An announcement from Pluristem on Aug. 13, 2012 indicated that those afflicted with peripheral arterial disease, or PAD, may soon have an alternative to Bristol-Myer Squibb's (BMY) troubled anti-clotting drug Plavix, whose drawbacks prompted the FDA to assign a black box label two years ago. People who have reduced functioning of their CYP2C19 liver enzyme cannot effectively convert Plavix to its active form. As a result, Plavix may be less effective in altering platelet activity in those people. These "poor metabolizers" may not receive the full benefit of Plavix treatment and may remain at risk for heart attack, stroke, and cardiovascular death.

Pluristem announced in the middle of September that it engaged in a secondary offering of new shares, raising enough money to give them at least five years of cash at their current burn rate with a comfortable capital position of $70 million.

At first I was critical of this raise because I thought the company would be best served to focus on trying to get a Phase II-III pivotal trial for its platform in treating a-plastic bone marrow. After talking to several analysts and people "in the know," I was informed that the company engaged in this offering to ensure it could make the transition from being an Israeli based company to a United States company. Also, I was informed that the company does plan to go for a Pivotal Phase II-III trial with outside compassionate use in regards to a-plastic bone marrow treatment with its platform. This kind of treatment is potentially world changing if ultimately proven to work via a pivotal clinical trial.

It has been nearly a month and Pluristem is expecting to hear back from the FDA soon in regards to its filed request for Orphan Drug Status concerning a plastic bone marrow, having successfully received orphan drug status from the FDA for their PLX cell therapy in the treatment of Buerger's disease. Should this status be granted (which is highly likely in my opinion), this could be a nice trade with short term returns. For those interested in the company as a long term bet, PSTI could be in position to begin marketing that treatment by the end of 2013.

Placenta based stem cell treatments could have the potential to change the world for the better by helping to save lives.

Pluristem closed yesterday's trading session at $4.07 Up $0.08 (2.01%).

Ariad Pharma (NASDAQ:ARIA) seems to have attracted a good deal of philanthropist investment over the last year or so. Ariad is currently finishing up a Pivitol Phase II-III for Ponatinib, an investigational pan BCR-ABL inhibitor, for the treatment of patients with hematologic cancers, including chronic myeloid leukemia and Philadelphia positive acute lymphoblastic leukemia. Ponatinib is the kind of drug that is potentially life-changing.

Ponatinib has fully enrolled its pivotal Phase II PACE (Ponatinb Ph+ ALL and CML Evaluation) trial in patients with resistant or intolerant CML and Ph+ ALL. Results from the Phase II PACE trial of ponatinib were reported at the annual meeting of the American Society of Clinical Oncology (ASCO) and the 2012 European Hematology Association (EHA) annual congress. ARIAD's Phase III EPIC trial comparing ponatinib to imatinib is continuing for newly diagnosed chronic-phase CML patients.

Ponatinib has really made a difference in the lives of a few "lucky" people suffering from the forms of leukemia mentioned above. This because the FDA has allowed Ariad to treat people suffering from these forms of leukemia via compassionate use, which allows certain qualified patients to try the drug outside of clinical trials. Pluristem is also interested in taking a similar route for a-plastic bone marrow treatment.

In July 2012, ARIAD submitted a New Drug Application (NDA) for ponatinib to the U.S. Food and Drug Administration. ARIAD is seeking U.S. marketing approval of ponatinib in patients with resistant or intolerant CML Ph+ ALL. The company is seeking accelerated approval for ponatinib by the FDA and has requested a priority review of the application.

Ariad expects an FDA approval decision for the drug sometime in Q1, 2013. The company's stock price has risen at a parabolic rate from around $0.45 in a little over 3 years, to a recent 52 week and all-time high of $25.39.

Sarepta Therapeutics (SRPT) is certainly another such company attracting a lot of philanthropist investors lately.

On 10/3/12, Sarepta announced that it received positive Phase IIb clinical data for its Duchenne muscular dystrophy (DMD) drug eteplirsen.

Sarepta said eteplirsen slowed the progress of the disease and increased patients' levels of the protein dystrophin in an extension of a midstage trial. Low levels of that protein are the cause of Duchenne muscular dystrophy. The stock exploded from a prior day's closing price of $14.99 to an intra-day high the next trading session of $45.00.

DMD is one of the most common fatal genetic disorders to affect children around the world. Approximately one in every 3,500 boys worldwide is affected with DMD. Girls are rarely affected by the disorder. DMD is a devastating and incurable muscle-wasting disease associated with specific inborn errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Symptoms usually appear in children by age three. Progressive muscle weakness of the legs and pelvis eventually spreads to the arms, neck and other areas. By age 10, braces may be required for walking, and most patients require full-time use of a wheelchair by age 12. Eventually, this progresses to complete paralysis and increasing difficulty in breathing due to respiratory muscle dysfunction requiring ventilatory support, and cardiac muscle dysfunction leading to heart failure. The condition is terminal, and death usually occurs before the age of 30. The outpatient cost of care for a non-ambulatory DMD patient is very high. There is currently no cure for DMD, but for the first time ever there are promising therapies in, or moving into, development.

Sarepta is definitely the kind of company that could be on the verge of a groundbreaking treatment here. To date, there is no known treatment for Duchenne muscular dystrophy.

Sarepta closed yesterday's trading session at $32.85 Down $1.45 (4.23%).

Amicus Therapeutics, Inc. (FOLD) is a company with the potential to attract a good deal of philanthropist investors.

Amicus is utilizing a new technology in the development of treatments for genetic diseases. Pharmacological chaperone technology involves the use of small molecules that selectively bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity.

In its Fabry program, the company is investigating the use of AT1001 to bind to destabilized α-galactosidase A enzyme (α-GAL) and thereby restore its intended biological function.

Fabry disease is a rare X-linked (inherited) lysosomal storage disease, which can cause a wide range of systemic symptoms. It is a form of sphingolipidosis, as it involves dysfunctional metabolism of sphingolipids.

There has been historic difficulty in finding successful treatments for rare genetic diseases such as Fabry's. If Amicus can show successful phase III data here, it could very well be like a "Kodak" moment in medicine's war against genetic diseases -- groundbreaking.

Amicus closed yesterday's trading session at $5.18 Up $0.19 (3.81%).

One company that might not yet be on the philanthropist investor radar yet is StemCells Inc. (STEM).

StemCells engages in the research, development, and commercialization of stem cell therapeutics, and related tools and technologies for academia and industry. It develops cell-based therapeutics for the central nervous system and liver.

StemCells priority focus at this time is on age-related macular degeneration (AMD), a medical condition which usually affects older adults and results in a loss of vision in the center of the visual field (the macula) because of damage to the retina. It occurs in "dry" and "wet" forms. AMD afflicts approximately 30 million people worldwide and is the leading cause of vision loss and blindness in people over 55 years of age.

Macular degeneration can make it difficult or impossible to read or recognize faces, although enough peripheral vision remains to allow other activities of daily life.

Starting from the inside of the eye and going towards the back, the three main layers at the back of the eye are the retina, which contains the nerves; the choroid, which contains the blood supply; and the sclera, which is the white of the eye.

StemCells announced on October 4th that the first patient in its Phase I/II clinical trial in dry AMD has been enrolled and transplanted. The trial is designed to evaluate the safety and preliminary efficacy of the company's proprietary HuCNS-SC product candidate (purified human neural stem cells) as a treatment for dry AMD.

StemCell's premise is to use tissue-derived stem cells only in that same organ, i.e., a homologous approach. For example, to use CNS-derived neural stem cells for the treatment of CNS disorders and liver-derived cells for the treatment of liver disorders. These multi-potent, tissue-derived "adult" stem cells are naturally pre-programmed to become the mature functional cells of the organ in which they are found, so they are directly transplantable into the same type of organ. Conversely, embryonic stem (ES) and induced pluripotent stem (IPS) cells have the potential to differentiate into any of the various cell types of the body, and so they would need to be re-programmed or genetically modified in order to reduce the risk of transplanting unwanted cell types.

While StemCells is a cheaper stock selling for $2.04 a share with a market cap of $62.34M, philanthropist investors might be beginning to take notice of this one -- I think deeper due diligence might be in order for StemCells.

The companies mentioned here in this article are but a few that offer hope for many who suffer from various forms of cancer and other serious diseases and ailments. As society further progresses in technological advancement, the hope to find treatments and cures increases.

Disclosure: I am long FOLD, PSTI. 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.

Disclaimer: This article is intended for informational and entertainment use only, and should not be construed as professional investment advice. They are my opinions only. Trading stocks is risky -- always be sure to know and understand your risk tolerance. You can incur substantial financial losses in any trade or investment. Always do your own due diligence before buying and selling any stock, and/or consult with a licensed financial adviser.