Aspreva Pharmaceuticals Corp. reported disappointing results from its Phase III study of immunosuppressive drug CellCept in patients with myasthenia gravis, though the company is hoping ongoing pivotal trials in two other autoimmune indications will yield better outcomes.

The Victoria, British Columbia-based firm did not release detailed data from the failed study, in which CellCept (mycophenolate mofetil) missed its primary endpoints of maintaining or improving disease symptoms while reducing patients' need for corticosteroids. The 176-patient study was designed to evaluate the effect of CellCept over a 36-week treatment period in myasthenia gravis, a neuromuscular disorder typically characterized by chronic fatigue and muscular weakness in the face and neck.

The news sent Aspreva's stock (NASDAQ:ASPV) down $3.16 Friday, or 14 percent, to close at $18.99.

"Over the next couple of days, we're going to dig through the data and put more information together," said Sage Baker, vice president of investor relations and corporate communications, in preparation for reporting the company's third-quarter earnings, scheduled for Wednesday.

However, Baker said Aspreva has no intention of further pursuing CellCept in myasthenia gravis.

"When you have an outcome that's as clear as this one is," she told BioWorld Today, there's no reason "to go forward with a regulatory attempt."

The company emphasized that the results in the myasthenia gravis study have no immediate impact on its ongoing Phase III trials in lupus nephritis and pemphigus vulgaris. Also, Baker said, 85 percent of Aspreva's revenues relating to CellCept come from areas other than myasthenia gravis, so "although we expect some impact, it shouldn't be substantial."

Aspreva added CellCept to its pipeline in 2003, through a licensing deal with Basel, Switzerland-based F. Hoffmann-La Roche Ltd., which developed the product through approval in transplant rejection. The agreement allowed Aspreva worldwide rights to CellCept for all autoimmune disease indications.

Last month, Aspreva finished enrolling 358 patients in its Phase III study of CellCept in lupus nephritis, which is designed to assess the safety and efficacy of the immunosuppressant in inducing response and remission in patients with biopsy-proved disease. It's a two-phase induction and maintenance study, with the first phase involving an open-label comparison of CellCept to intravenous cyclophosphamide. That phase is expected to be completed in early 2007, with a potential new drug filing in the fourth quarter of 2007, Baker said.

The maintenance phase of the trial will involve a double-blind comparison of CellCept and azathioprine for up to three years.

A Phase III study testing CellCept in the skin disease pemphigus vulgaris completed enrollment in March. That trial involves 77 patients and is investigating the drug's activity over a 52-week treatment period, with a primary endpoint encompassing both minimal disease activity defined as no new persistent lesions, with a low steroid dose.

Aspreva expects to complete that study in 2007, with possible regulatory submission in 2008.

The company also is considering other indications for CellCept, possibly cardiovascular disease associated with lupus nephritis and multiple sclerosis, though Baker said plans were still in "the very early stages of looking to see whether it would make sense" from a business standpoint.

For the second quarter, Aspreva reported net income of $28 million, or 78 cents per share. As of July 31, it had cash and short-term investments totaling $192.2 million.

NicOx's Naproxcinod Meets Endpoints

Another firm reporting Phase III results Friday had better news. NicOx SA, of Sophia Antipolis, France, said top-line results from a Phase III trial of naproxcinod in patients with osteoarthritis of the knee showed that the drug, a nitric-oxide anti-inflammatory compound, demonstrated statistically significant superiority to placebo at two dose levels (750 mg and 375 mg) in all three co-primary endpoints of the study. Endpoints were assessed by the mean change in baseline at week 13 in three scales: the WOMAC pain subscale, the WOMAC function subscale and patients' overall rating of disease status.

About 250 patients were enrolled per treatment group.

Data also showed that both doses of naproxcinod decreased systolic and diastolic blood pressure, which the company believes will separate it from existing anti-inflammatory products, such as naproxen. Naproxcinod also demonstrated good overall safety.

The study, designated the 301 study, is the first of three Phase III trials for naproxcinod, and NicOx expects to follow it with the 302 and 303 studies in knee and hip osteoarthritis in 2007.