Parkinson's Disease: 3 Different Firms, 3 Novel Approaches

Includes: AMRX, FOLD, ISCO
by: Shrideep Murthy

The Parkinson's Disease Foundation estimates that as many as one million Americans suffer from Parkinson's disease and 60,000 new patients are diagnosed each year. Between seven to ten million people are affected by Parkinson's around the world. Medication costs per person are believed to be around $2,500 each year and the total economic impact is estimated to be around $25 billion in the US alone. Boxing legend Muhammed Ali established the Muhammed Ali Parkinson Center for diagnosis, therapy and research and has raised millions of dollars to find a preventive cure for the dreaded disease. There is immense scope for a company that is able to find a preventive treatment for Parkinson's which is encouraging many small and large bio-tech firms to invest in research and development.

A brief overview

Certain neurons in the part of the human brain called substantia nigra are responsible for producing a chemical called dopamine which aids in controlled muscle movement. The death of these neurons leads to a deficiency of dopamine further leading to erratic muscle movement. An early symptom of Parkinson's is a resting tremor, i.e. a small tremor in the affected body part when it is not performing action. As the disease progresses, it leads to muscle stiffness and rigidity making everyday activities difficult to perform. Though we know that the lack of dopamine is the primary cause for Parkinson's, it is not known what exactly causes the death of dopamine-producing neurons. Parkinson's UK, a research and support charity, is funding more than 40 different studies to answer this question.

Current treatments

Current treatments for Parkinson's revolve around the drug called Levodopa. The brain converts this drug to dopamine which reduces the symptoms of the disease, but does not cure it. It also does not slow the progress of the disease and the symptoms get worse over a period of time. A healthy diet, exercise, physiotherapy and occupational therapy are some other prescribed treatments.

GlaxoSmithKline plc (NYSE:GSK), Merck & Co. Inc. (NYSE:MRK), Novartis AG (NYSE:NVS) and Teva Pharmaceutical Industries Limited (NYSE:TEVA) are some of the big names that market medication to patients suffering from Parkinson's.

Adopting a completely different approach to the treatment of this condition are small biotech companies that are trying to tap the potential of stem cells or gene therapy or simple extended versions of Levodopa medication. Here I profile some companies, each with different approach, different market capitalization and different risk profiles.

International Stem Cell Corporation (OTCQB:ISCO)

International Stem Cell Corporation is a small biotech firm in the research stage. It focuses on using stem cells for treating diseases like Parkinson's, liver damage and cornea tissue implants. ISCO has conducted extensive research on using pluripotent stem cells which have the ability to convert to any type of cells in the body. The idea is to induce the pluripotent stem cells to convert themselves to dopamine-producing cells in the brain so that the chemical is produced naturally.

In an announcement on July 17th, 2012, ISCO said that they intend to start studies measuring the efficacy of stem cells developed by them in the third quarter of 2012. Studies on non-human primates (monkeys) will start in the fourth quarter of 2012 and the first set of conclusions will be available in early 2013.

The company is also using its expertise in stem cells to create a stem cell bank thus making them available for research or commercial use. Lifeline Skin Care is a wholly-owned subsidiary of ISCO that leverages on it parents expertise to build products that can provide marginal revenue streams.

ISCO trades on the OTC markets at around $0.25 and has a market cap of $25 million. ISCO has total current assets worth $9 million and current liabilities worth $2 million. ISCO is a promising early stage bio-tech firm suitable for people with a high risk appetite and long-term investment horizon.

Amicus Therapeutics, Inc. (NASDAQ:FOLD)

Amicus Therapeutics seeks to develop orally administered drugs called pharmacological chaperones to treat a broad range of diseases of neuro-degeneration. Adopting a different approach, Amicus targets the GBA1 mutation that has been identified as the most common genetic factor of Parkinson's.

Amicus is conducting preclinical studies to evaluate two pharmacological chaperones, AT2101 and AT3375, to target the GCase enzyme. Genetic mutation in GBA1 genes that encode the GCase enzyme is a root cause of Gaucher disease. Amicus plans to target Parkinson's in Gaucher carriers and then Parkinson's in general for which it received a grant from Michael J Fox foundation.

Amicus trades at around $5 and has a market cap of $238 million. At the end of the second quarter of 2012, Amicus had current assets worth $105 million and current liabilities worth $17 million. Given the number of drugs that it has in its pipeline Amicus may warrant a second look for investors with a medium term outlook plus a medium risk profile.

Impax Laboratories Inc. (IPXL)

Impax Laboratories is a larger pharma company with a diversified product portfolio and has several other drugs in various stages of development. In February Impax announced that the US FDA had accepted for filing its NDA application for treatment of Parkinson's disease. IPX066 is a patented extended release version of carbidopa-levodopa that has undergone extensive clinical development and studies in patients with Parkinson's disease. The company expects to hear back from the FDA by October 21st, 2012 and has licensed the compound to GlaxoSmithKline for development and marketing.

Impax trades at around $24 and has a market cap of $1.5 billion. It has a steady stream of income and can be a good investment for investors who do not prefer taking on much risk.

As a caveat, let me add that each company profiled may not be ideal for every investor. Some might be a good investment for a risk taker and a long-term investor while another may be an appropriate play for an investor who seeks steady growth

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.