Roche's Actemra Clears Hurdle On Way To Approvals

Drugmaker Roche Holding AG (RHHBY) Friday said Actemra, an experimental drug that treats rheumatoid arthritis in a new way, showed positive results and was safe in a key study, paving the way to file the drug for regulatory approval in Europe and the U.S. later this year.

Roche, based in Basel, Switzerland, said the study, dubbed OPTION, showed that a greater proportion of patients treated with Actemra in combination with traditional drugs achieved a significant improvement in disease signs and symptoms after 24 weeks, compared to those treated with the traditional drugs alone.

Sufferers of rheumatoid arthritis, or RA, begin to experience progressive joint damage early in the disease. The disease differs from what is popularly known as arthritis, typically associated with age-related joint pain; RA is a far more serious inflammatory disease that leads to destruction of cartilage and bone, and can lead to disability.

Patients have a big number of treatment options to chose from, but many drugs merely relieve pain, in some cases at the cost of severe long-term side effects including osteoporosis and high blood pressure.

Actemra works by inhibiting the activity of interleukin-6, a protein that plays a major role in the inflammation process of rheumatoid arthritis; it's generally well tolerated, Roche said.

If approved, Actemra will compete with other biological drugs called anti-TNF medicines. These medicines target a compound known as tumor necrosis factor, which is overproduced in many patients with inflammatory disease.

Johnson & Johnson's (JNJ) Remicade and the drug Enbrel, which is co-marketed by Amgen Inc. (AMGN) and Wyeth (WYE), are all part of this class.

In the study, four times the number of patients in the Actemra group experienced a 50% improvement in disease symptoms compared to the control group, while more than ten times the number of Actemra patients achieved 70% improvement in disease signs and symptoms compared to the control group. In addition, 28% of patients achieved the ultimate goal of remission in the Actemra group against only 1% of patients in the control group.

At 0950 GMT, Roche shares were up CHF1.60, or 0.7%, at CHF219.90, in a higher broader market.

Actemra, also known as tocilizumab, was developed by Japanese pharmaceutical company Chugai Pharmaceutical Co. (4519.TO), which is controlled by Roche. Roche owns the rights to market Actemra outside Japan.

The drug has shown very good results in trials conducted in Japan, and Roche hopes to replicate these early data from the Japanese trial in large American and European studies. The study presented Friday, is is the first of five international late-stage trials that are investigating Actemra's efficacy and safety.

Roche plans to file Actemra for regulatory approval in Europe and the U.S. by the end of 2007. It usually takes around a year for regulators to review data submitted by a company and decide on approving a new drug.

In Japan, where the approval process usually takes somewhat longer, Actemra was filed for regulatory approval in April 2006.

Gilead Says FDA Approves Drug To Treat PAH

Gilead Sciences Inc. (GILD) said the U.S. Food and Drug Administration late Friday approved its drug ambrisentan for the treatment of a lethal form of hypertension that strikes without warning and can kill within three years if untreated.

The drug, which will carry the U.S. trade name Letairis, is designed to treat a disease known as pulmonary arterial hypertension (PAH), in which arteries in the lungs constrict and thicken, raising blood pressure and eventually causing heart failure. Patients typically go to doctors complaining of exhaustion, breathlessness, and inability to walk distances or perform activities of normal daily living. Often no cause can be identified, although the condition also can be triggered by diseases like scleroderma or advanced HIV, or by certain medicines like the diet drug combo Fen-Phen.

Letairis belongs to a class of drugs called ERAs or endothelin receptor antagonists, compounds that relax the vessels, lowering blood pressure and lightening the load on the heart and lungs. However, such drugs can't be taken by pregnant women due to risk of major birth defects, and can cause liver damage.

In studies, the drug improved patients exercise capacity, as measured by a six-minute walking distance test. Gilead said it has priced the drug at $3,940 a month, comparable with other oral drugs for the disease. Gilead said it established a program to ensure greater access to patients who are underinsured or face high co-payment requirements.

Because of the risks of liver damage and birth defects, the drug's label will carry strong cautionary language in a black box, as do other drugs of its class. Gilead says its studies show that incidence of liver damage is less with Letairis than with other products in this class. The company said Letairis will be available only through a special restricted program to educate patients and doctors about safe usage.

Gilead acquired the drug as a result of its merger with the drug's developer Myogen Inc. in November 2006, and plans to make it the centerpiece of a third branch of its business in the cardio-pulmonary field. Gilead's core business has focused mainly on products for HIV and hepatitis.

Based on market research, Gilead said it estimates about 75,000 to 90,000 people in the U.S. suffer from PAH, and 200,000 people worldwide, although fewer than half of them are currently believed to be diagnosed. The condition is often misdiagnosed as exercise-induced asthma or other respiratory ailments.

Letairis will be marketed in the U.S. by Gilead, and by GlaxoSmithKline PLC (GSK) outside the U.S.

Basilea Superbug Filed For European Approval

Basilea Pharmaceutica AG (BSLN.EB) said Monday its superbug antibiotic has been submitted for European approval, prompting a milestone payment of 12 million Swiss Francs ($9.8 million) for the Basel-based company.

Basilea said the anti-infective treatment ceftobiprole was submitted to the European Medicines Agency by a subsidiary of Johnson & Johnson (JNJ), with whom Basilea will sell the treatment in the U.S. and major European countries.

"Today's marketing authorization application submission as well as the recent new drug application filing in the USA reflect our view that ceftobiprole could play an important role globally in the future management of important medical problems posed by methicillin-resistant Staphylococcus aureus," Basilea's Chief Executive Anthony Man said in a statement.

The filing, in keeping with Basilea's pledge to submit the treatment this year, follows favorable study date from January, where the treatment was shown to be effective against complicated skin infections.

In January, Basilea said ceftobiprole demonstrated high cure rates in a range of patients, including those with methicillin-resistant Staphylococcus aureus, or MRSA, and diabetics with foot infections.

Basilea shares, which jumped earlier this year after the most recent data from ceftobiprole, have gained 28% this year, giving the company a market capitalization of $2 billion.

The stock closed at CHF272 Friday; trading resumes at 0700 GMT Monday.

Ceftobiprole is in Phase-III testing to treat patients who develop pneumonia while in hospital, as well as community-acquired pneumonia, for which Basilea expects results in the latter half of 2007.

New Option For Rare Condition

People with a rare and lethal high blood pressure now have more options for convenient treatment with less drug toxicity.

Late Friday the Food and Drug Administration approved Letairis, a pill made by Gilead Sciences Inc. for the treatment of pulmonary arterial hypertension, in which lung vessels constrict, raising blood pressure and leading to heart failure.

Mysterious and often misdiagnosed, PAH can arise without warning. It also can result from diseases like scleroderma or advanced HIV, or the diet drug combo known as fen-phen. Based on market research, Gilead, of Foster City, Calif., estimates 75,000 to 90,000 people in the U.S. have PAH, over half of them undiagnosed.

About a half-dozen PAH drugs exist, all of them expensive, including round-the-clock infusions and some oral drugs that risk birth defects and liver damage. Some patients require lung transplants, though drug therapy can stave off the need for it. Letairis's label carries a black-box warning about such risks, but the company and some analysts say it has the best safety profile of any oral drug to date. Shashi Sahgal, a 42-year-old homemaker and mother of two teenagers in Mission Viejo, Calif., was hiking and bounding up stairs two at a time back in 2002. When she felt winded, doctors thought she had exercise-induced asthma. By the end of 2002, exhaustion and an enlarged heart led doctors to perform a right-heart catheterization. That nailed the diagnosis of PAH.

Without treatment, PAH can be fatal within three years. Referred to lung specialist Ronald Oudiz of Harbor UCLA Medical Center in Torrance, Calif., Mrs. Sahgal entered a trial of the new Gilead drug, then known by its generic name ambrisentan. "It made a huge difference," she says.

Gilead acquired ambrisentan as part of its merger with Myogen Inc. last November, and it now plans to launch sales in the U.S. this week. Under a co-marketing agreement with GlaxoSmithKline PLC, GSK is seeking approval to market the drug outside the U.S. On Friday, the FDA delayed action on another PAH drug, Thelin by Encysive Pharmaceuticals Inc. of Houston, urging more studies.

The first drug approved for the condition in 1995 was GSK's Flolan, an infusion given continuously through a chest catheter. Originally designed as a bridge to a transplant, the drug treatment alone has extended some of his patients' lives by 13 or 14 years, says UCLA's Dr. Oudiz. Gilead now sells Flolan in the U.S., while GSK markets it outside the US.

United Therapeutics Corp., Silver Spring, Md., markets a similar drug, Remodulin, which is given as a subcutaneous or intravenous infusion. A pill form is being studied.

Swiss drug maker Actelion Ltd. markets an inhaled drug, Ventavis. It also markets a pill, Tracleer, part of an oral-drug family that expands vessels but can cause liver damage or birth defects.

Gilead's Letairis is also part of the same drug family, known as ERAs (endothelin receptor antagonists, which work by relaxing blood vessels). Gilead says its studies show Letairis has a lower incidence of liver toxicity than rival products. Common side effects include peripheral edema (or swelling), nasal congestion, flushing and palpitations. Patients must have liver enzymes monitored to detect any signs of damage, and women -- who account for most PAH patients -- must avoid becoming pregnant.

Pfizer Inc. entered the field after discovering its erectile-dysfunction drug, Viagra, could benefit vessels of the lungs as well. The New York company rechristened the drug as Revatio for PAH. Another such drug, Indianapolis-based Eli Lilly & Co.'s Cialis, is now in tests for PAH.

Like other orphan drugs for rare but serious diseases, PAH treatment is costly. A year of Flolan can run $100,000, while Tracleer tops $40,000, according to Gilead officials.

On Friday, Gilead said it set the price of Letairis at $3,940 a month, or $47,280 a year, using Tracleer's pricetag as a benchmark. The company plans to offer patient assistance with reimbursement, as well as some discounts and free drugs to patients lacking coverage.

A year of Revatio given as a thrice-daily 20-milligram tablet is about $10,000, says Dr. Oudiz. But for uninsured patients who are taking higher doses, he says it is less costly to take the drug as Viagra.

Dr. Oudiz has received funding support from Actelion, Gilead, Pfizer, United Therapeutics, Encysive and Lilly.

Like many patients who move to combination therapy, Mrs. Sahgal now also takes Revatio, along with the blood-thinner coumadin to prevent clots.

"I still can't go uphill fast, and I'm not going to climb Mt. Everest," says Mrs. Sahgal, now 46. But she adds, "If all goes well, we're looking at 20-plus years."