FDA shoots down United Therapeutics’ pill -- Gazette.Net


United Therapeutics, which already sells intravenous, subcutaneous and inhaled versions of a drug to treat pulmonary arterial hypertension, has been working for years to gain regulatory approval for an oral version.

On Tuesday, the Food and Drug Administration shot down the Silver Spring company's plans to have the pill on the market any time soon.

United Therapeutics stock took a pounding on the news, dropping 15.3 percent.

"It's kind of back to the future here," CEO Martine Rothblatt said in a conference call with investors, referring to the regulatory hurdles the company faced in securing approval for the other versions of the drug. "At the 11th hour, we were very excited about oral trepostinil, but more questions were raised."

Specifically, the FDA had concerns that the drug didn't show enough improvement in patients who took it in clinical trials, as measured by how far they could walk in six minutes. The agency suggested that another phase 3 trial should possibly involve fixed and more frequent dosing, such as three times a day versus twice daily, COO Roger Jeffs said in the call.

Some of the agency's concerns came as a surprise, Jeffs said, as they hadn't been raised in regulators’ discussions with the company.

For now, United Therapeutics’ executives are seeking a sit-down with FDA officials to review their concerns and come up with a plan to pursue approval of the pill, Rothblatt said.

"We have great belief in the trepostinil molecule," she said. "It's had a transformative effect on many patients" in its other forms.

"Our goal is to satisfy the FDA," Rothblatt said. "Whatever it takes, we will do. It's a great molecule ... the gold standard." There is a way to win regulatory approval for the oral form, "but we haven't found it."

The company has projected sales of about $1 billion for the other forms of the drug next year, and with 30,000 Americans being treated for pulmonary arterial hypertension, Rothblatt said the oral version shows much sales promise.

The potential market of about 20,000 patients for oral trepostinil represents "luscious and delectable low-hanging fruit," she said. "Most companies would salivate to have [the oral version] in their pipeline."

In other Maryland bioscience industry news:

Supernus Pharmaceuticals enjoyed more regulatory success than United Therapeutics this week.

The Rockville drug-maker reported that the FDA approved its epilepsy drug Oxtellar XR , a novel once-daily extended release formulation of oxcarbazepine.

Supernus stock rose 10.1 percent on the news Monday, before falling later in the week.

"This is excellent news for Supernus and patients with epilepsy," CEO Jack Khattar said in a statement. "We are very excited for having obtained two [new drug application] approvals since our [initial public offering] in May.”

The FDA approved Trokendi XR, also for treating epilepsy, in June.

"We will now focus on completing the build-out of our commercial organization including, hiring, training and deploying our field sales force to launch Oxtellar XR in the first quarter of 2013," Khattar said.

Oxtellar XR is approved for treating partial seizures in adults and in children 6 to 17 years old.

Shareholders of Nabi Biopharmaceuticals on Monday overwhelmingly approved the Rockville biotech's acquisition of an Australian drug-maker that will result in the Melbourne company as the surviving entity with headquarters in Rockville.

More than 80 percent of Nabi shareholders OK'd the deal with Biota Holdings of Melbourne. That company produces influenza vaccine-related products Relenza, which is licensed to GlaxoSmithKline, and Inavir.

Last month, Nabi headed off a showdown with a major stockholder that had opposed the deal. Mangrove Partners, with 4 percent of Nabi stock, had been urging stockholders to reject the deal when they were initially scheduled to vote on it in September.

Mangrove had argued that shareholders would get a better deal if Nabi — whose experimental smoking vaccine failed in clinical trials in November, and then again this month — returned “substantially all of the cash” on its balance sheet to shareholders. Some analysts had suggested the company liquidate and return the proceeds to shareholders.

But Nabi and Biota agreed to give stockholders a greater return than they originally proposed when they announced the deal in April. And Mangrove supported the new terms.

When it announced the original deal, Nabi said it planned to pay $54 million to Biota to create a new company, Biota Pharmaceuticals, leaving Nabi stockholders with 26 percent of the new company.

Under the revised terms, Nabi is to pay Biota shareholders $27 million in Nabi stock, with Nabi stockholders receiving about $28 million to $31 million.

Before the vote, two independent proxy advisory firms, Institutional Shareholder Services of Rockville and Glass Lewis of San Francisco — both of which previously advised against the deal — recommended that Nabi stockholders approve the revised transaction.

Biota sees the deal as a way to access the “larger and deeper” U.S. marketplace, its chairman, Jim Fox, said during his company’s conference call in April.

He said Biota had been looking to get into the U.S. for a year and plans to have a U.S.-based CEO to give it a “direct and more clear connection to the U.S. market,” Fox said.

The new company’s board is to comprise two Nabi directors and six Biota directors.

Intrexon, which focuses on a range of bioscience products including human therapeutics, protein production and agricultural biotechnology, is expanding its Germantown presence.

The Blacksburg, Va., company signed a multiyear lease for 55,000 square feet of laboratory and office space at 20358 Seneca Meadows Parkway in the Seneca Meadows Corporate Center, according to Scheer Partners of Rockville, which represented the company. The landlord is Minkoff Development of Germantown.

In 2009, Intrexon purchased substantially all of the equipment and assumed the lease of Avalon Pharmaceuticals’ bioassay facility at 20358 Seneca Meadows Parkway, which Intrexon made the home of its human therapeutics division. That site also now houses its animal science division.

Rockville vaccine developer Novavax reported raising $27 million through the sale of 12.3 million shares of its common stock to RA Capital Management, Camber Capital Management and Ayer Capital Management.

Net proceeds of about $26.9 million will be spent on general corporate purposes, including working capital, product development and capital expenditures, the company said in a statement.

Sequella has licensed its B-Smart technology to Laboratory Corporation of America Holdings, which plans to develop a new laboratory-based antibiotic susceptibility assay for Mycobacterium tuberculosis for commercial use in the U.S.

Terms were not disclosed. Sequella retains all rights to commercialize the technology worldwide for indications other than tuberculosis, and for territories other than the U.S. for tuberculosis, according to a company statement.

Sequella develops antibiotics to treat infectious diseases, especially those with drug resistance.

Chesapeake Perl of Savage won a federal contract to develop the human nerve agent bioscavenger butyrylcholinesterase as a potential nerve agent countermeasure for the Defense Threat Reduction Agency.

The company said in a statement that it will work with the Army Medical Research Institute of Chemical Defense at Aberdeen Proving Grounds to test the circulatory stability and anti-nerve agent protective efficacy of the compound, with the goal of advancing it into clinical trials.

Chesapeake Perl also reported winning a Small Business Innovation Research grant from the National Cancer Institute to continue developing a possible treatment for lung cancer.