GTx Inc. (GTXI): GTx is a Memphis biopharmaceutical company whose pipeline is largely dedicated to cancer treatments and other cancer related drugs.
The company developed SARM (selective andular receptive modulators) in the late 90s, and recently re-acquired the rights to this technology (from 2010 form 10-K). The SARM program includes one of GTx's main pipeline products, Ostarine, which began two separate pivotal phase III trials this July. The results should be announced in the fourth qurter of next year. Ostarine is intended for prevention and treatment of muscle-wasting in patients with non-small cell lung cancer. The company says that roughly 70% of patients with this cancer suffer from moderate to severe muscle loss. NSCL cancer accounts for roughly 85% of the 220,000 new cases of lung cancer per year. Based on these numbers, 131,000 patients suffer from muscle-wasting; a very large market indeed.
Muscle-wasting is a serious issue when treating patients, as it reduces the ability of patients to undergo chemotherapy. An inability to accept treatment is a common cause of death.
The company met with the FDA after the completion of its phase II trials, where it received guidance on its phase III trials. GTx's phase II trials met significant endpoints, and harsh side-effects were uncommon.
The company is also developing Capasaris, which is a first-line treatment for prostate cancer.
Financially, the company is in good shape. Their only marketed drug, Fareston, helped them reach $14 million in revenues in 2010, and the company has a wonderful $90 million in cash, along with no debt to speak of. The stock currently trades at $4.18.
NPS Pharmaceuticals (NPSP): NPS Pharmaceuticals primarily focuses on developing treatments for patients with uncommon gastrointestinal issues, and endocrine disorders. The market is largely untapped thus far.
GATTEX, NPS' leading candidate, treats short-bowel syndrome. SBS typically results from Crohn's disease or other bowel disorders; SBS is severe damage to the intestines which results in malnutrition, severe diarrhea, dehydration, fatigue, and severe weight-loss.
GATTEX has had fantastic results from its phase III trial, including recent news that it met its primary efficacy endpoint in the trial; "reducing parenteral nutrition dependence in adults with short-bowel syndrome." The "parenteral nutrition dependence" is a $100,000 annual procedure that repairs the intestines in needy patients. The issues of the procedure of course include cost, that it doesn't improve the body's ability to actually absorb nutrients itself (it simply delivers them through an IV), and that it can lead to fatal complications.
GATTEX has an orphan drug designation from the FDA.
NPSP558 is also being developed by the company. The drug is intended for usage in patients hypoparathyroidism, "a rare endocrine disorder in which the body produces insufficient amounts of parathyroid hormone. The company's 10-K report states that at least 60,000 U.S. cases are prevalent yearly.
The stock trades at $6.75.
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.



