Actelion surges as positive PhIII macitentan data reignites buyout buzz

Posted under Actelion, Blog, Clinical Trials, Companies, Diagnostics, Funding, macitentan, Medical Devices, Medical Supply, Pharmaceuticals, Phase III, Pipeline, Startups, Tracleer, Universities, Videos by john

Actelion cleared a crucial hurdle today, reporting that its late-stage study of macitentan for pulmonary arterial hypertension beat the primary endpoint. The data clears a path to the regulatory agencies, signaling to investors that the Swiss company can be ready for the loss of patent protection on its primary earner with the introduction of another blockbuster. And Actelion shares ($ATLN) jumped more than 20% on the news.

News of the positive Phase III provides a clean win for founding CEO Jean-Paul Clozel, who has had to fend off repeated claims that the company was on the road to ruin. Actelion gets 90% of its revenue from Tracleer, which loses patent protection in 2015. If macitentan--a successor to Tracleer--can clear a regulatory review, the drug can replace lost revenue.

According to investigators, a 10 mg dose of macitentan reduced the risk of mortality and morbidity 45% compared with a placebo, while the 3 mg dose cut the same risks 30%. It managed those results without evidence of liver toxicity, giving it an edge on Tracleer. And in this heated M&A environment, the promising Phase III quickly triggered fresh speculation of a possible buyout.  

"The results of SERAPHIN suggest the market's assumption that its business will simply disappear has been overly negative and are likely to make chances the company becomes an M&A target realistic," noted Richard Parkes, an analyst at Deutsche Bank, according to a Reuters report.

"Essentially 'best case' Phase III results for macitentan should ensure a long-term future for Actelion's PAH franchise and shift the disease treatment paradigm," noted Jefferies.

- read the press release
- here's the Reuters article

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PRA snags Amgen’s PhIII biosimilars contract

Posted under Amgen, biosimilars, Blog, Clinical Trials, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Merck, Partnering, Pharmaceuticals, Phase III, PRA, Startups, Universities, Videos, Watson Pharmaceuticals by john

PRA has snagged an exclusive deal to run Amgen's worldwide Phase III biosimilar studies. The contracting coup comes four months after Amgen ($AMGN) tied up with Watson ($WPI) on an ambitious pact to develop biosimilars of some leading cancer therapies. Watson signed on with a capped investment of $400 million over the next 7 years, while Amgen has an open-ended commitment to the deal. And analysts were intrigued by the notion of a leading biologics company jumping into an arena that has already attracted a lineup of Big Pharma players like Merck ($MRK) as well as a group of multinationals. Story

Bayer bolsters blockbuster case for regorafenib with new PhIII data

Posted under Bayer, Blog, Cancer, Clinical Trials, colorectal cancer, Companies, Diagnostics, Funding, gastrointestinal tumors, Medical Devices, Medical Supply, Nexavar, Onyx Pharmaceuticals, Pharmaceuticals, Phase III, Pipeline, regorafenib, Startups, Universities, Videos by john

Bayer has racked up a fresh set of preliminary late-stage data that will bolster its plans to market regorafenib for a range of cancers. The treatment, which bears a striking resemblance to sorafenib (Nexavar), registered a successful response for gastrointestinal stromal tumors in Phase III, adding to the positive data already gathered on colorectal cancer.

Just a few months ago Bayer agreed to pay Onyx ($ONXX) $160 million and a hefty royalty slice on regorafenib to settle the biotech's claim that Bayer scientists had secretly created an analog closely matched to sorafenib, their blockbuster cancer medication. Now that that dispute has been replaced by a partnership, Bayer is eager to move ahead on a new drug program that promises to deliver more than a billion dollars a year in new revenue. Bayer has set its sights on applying for an approval on colorectal cancer with plans to expand its use for other cancers.

In this latest trial Dana-Farber's George Demetri studied regorafenib in advanced cancer patients who weren't responding to existing medications. Bayer says the treatment hit its primary endpoint on progression-free survival. The data will be detailed at an upcoming scientific meeting.

About 200 patients were enrolled in the study, with a 2-to-1 mix getting 160 mg of regorafenib daily, three weeks on and one week off. And patients in the control arm whose disease progressed were offered regorafenib.

"GIST is a difficult disease to treat. Patients treated with currently available therapies have a high likelihood that their disease will progress, and there are no remaining treatment options for those patients whose prior therapies have failed," said Ted W. Love, the EVP for R&D at Onyx. "These clinical data will show regorafenib's activity in patients with previously treated GIST."

- get the press release
- here's the story from Bloomberg

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UPDATED: Roche readies for approvals of previously snubbed breast cancer drug

Posted under Blog, breast cancer, Clinical Trials, Companies, Diagnostics, Eli Lilly, Funding, Herceptin, ImmunoGen, Medical Devices, Medical Supply, Pharmaceuticals, Phase III, Pipeline, Roche, Sanofi, Startups, T-DM1, Universities, Videos by rmcbride

Roche ($RHHBY) has turned in positive data from a key Phase III study of its blockbuster hopeful T-DM1 and later this year plans to seek green lights to sell the breast cancer drug in the U.S. and Europe, the company announced today.

The Swiss drug giant revealed top-line results from its 991-patient "EMILIA" study, saying that T-DM1, or trastuzumab emtansine, met one of the main goals of the study in providing a significant boost in survival without patients' cancer getting worse compared with patients who took the two cancer drugs lapatinib and Xeloda. The company didn't have available data on overall survival, which is the second primary endpoint of the study. Detailed results of the trial are expected at an upcoming scientific meeting.

Roche, which boasts the world's largest cancer drug business, sees T-DM1 as an encore to its drug Herceptin in treating HER-2 breast cancers, which account for 15% to 20% of all breast cancers. The experimental drug links Herceptin, an antibody drug, to the chemo agent DM1, providing what the company hopes will be a one-two punch that is more powerful in combating breast cancer than Herceptin or even Herceptin and separate doses of chemo. All patients in the EMILIA trial were previously treated with Herceptin and chemo.

The FDA turned down Roche's early bid for approval of T-DM1 in 2010, saying that the company's Phase II data weren't enough to win the agency's stamp. Roche is obviously upbeat about going back to regulators this year with Phase III results added to its data package for the program.

"We are excited about the EMILIA results because trastuzumab emtansine is our first antibody drug conjugate, and it may help people who still need more treatment options for this aggressive disease," Dr. Hal Barron, Roche's chief medical officer and head of product development, stated. "We will work to submit these data to regulatory authorities as quickly as possible."

ImmunoGen ($IMGN), which licenses its linker tech and the chemo agent for T-DM1 to Roche, could get its first revenue from sales of an antibody-drug conjugate if the drug is approved. To date, the Waltham, MA-based biotech relied on revenue from partnerships with Roche, Sanofi ($SNY), Eli Lilly ($LLY) and others. Biotech commentator Adam Feuerstein noted on Twitter this morning that ImmunoGen's royalty on T-DM1 sales is 5%.

- here's Roche's release
- check out the Reuters report
- get more from Dow Jones

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Roche trial buoys antibody-based delivery of cancer drugs
Roche's T-DM1 generates a fresh blast of positive PhII data
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Corrected and updated: ImmunoGen doesn't license T-DM1 to Roche, as initially reported in this article. ImmunoGen licenses the linker technology and chemo agent DM1 used for T-DM1 to the drug company. We apologize for any confusion. The story was updated after the correction to add that ImmunoGen also licenses DM1 to Roche.