Monrovia Nursery Co., Inc.

November 13, 2015 By Mark Terry , BioSpace.com Breaking News Staff Analysts are again refocusing their attention toward what companies Thousand Oaks, Calif.-based Amgen might consider buying. This time, the focus is particularly on Cambridge, Mass.-based Alnylam Pharmaceuticals . Inside sources indicated to The Financial Times early this week that Amgen was ready to join this year’s merger and acquisition frenzy with a big acquisition. Mostly the company has avoided big acquisitions, instead buying or licensing drugs in early stages of development. It has 11 experimental drugs in early-stage clinical trials, five in mid-stage trials and seven in late-stage trials. In August, Amgen announced a strategic collaboration deal with Monrovia, Calif.-based Xencor to develop and market cancer drugs and inflammatory drugs. But the sources indicate Amgen is ready to look at companies in the $10 billion range. Possible acquisition targets have included Seattle Genetics, Inc. , Dr. Reddy’s Laboratories Ltd. , and Aspen Pharmacare Ltd. . Alnylam was also cited. There were rumors in January 2015 that Paris-based Sanofi SA was looking at Alnylam , but to date nothing has happened. On Nov. 11, Alnylam announced that, working with The Medicines Company , it had positive results from a Phase I clinical trial of ALN-PCSsc, an investigational RNAi therapeutic that targets PCSK9, a protein regulator of LDL receptor metabolism—essentially, the drug appears to be effective in treating high cholesterol. “An efficacious and well tolerated bi-annual, low volume, subcutaneous dosing regimen could address the unmet needs for hypercholesterolemia management in a large, at-risk, often non-adherent population worldwide,” said John Kastelein , professor of Medicine and chairman of the Department of Vascular Medicine at the Academic Medical Center (AMC) of the University of Amsterdam , in a statement. “By harnessing the natural pathway of RNAi, ALN-PCSsc has the potential to offer a genetically validated approach for testing ASCVD patients with elevated LDL-C, a cardiovascular disease risk factor, to get to LDL target.” There are a couple interesting things about this drug. First, it’s given twice a year. Compare that to two new PCSK9 inhibitors launched recently by Amgen , Repatha and Praluent. They are given once (Repatha) or twice (Praluent) monthly. Although all three drugs block PCSK9 proteins, they do it differently— Anlylam ’s drug blocks the creation of PCSK9 in the liver using RNA interference, while Amgen ’s drugs block the PCSK9 from acting in the blood. Alnylam ’s drug also appears to be more effective than Repatha and Praluent. “These data show that significant and clamped lowering of LDL is achieved for over 180 days,” said Akshay Valshnaw , Alnylam ’s executive vice president of research and development, and chief medical officer, in a statement. Of course, all of these drugs have a fundamental pricing problem. Praluent and Repatha are priced at about $14,000 per year. The standard of care for high cholesterol is currently statins, which are typically priced about $50 per month, or $600 per year. Not surprisingly, insurance companies are a little skeptical about paying for these new therapies, although Vaishnaw says, “We think payers will like it because they don’t mind paying for medicines if patients will actually take it.” Writing for BidnessEtc, Hannah Ishmael points out that if the Alnylam drugs hit the market, they would be a big competitor to Amgen , and that its main rival, Sanofi , has a big stake in the company. Getting to Alnylam first would eliminate that competition and strengthen its position against Sanofi . Marko Kozul , an analyst with Leerink Partners , has projected that Alnylam could bring in almost $1 billion in 2020 sales through rare disease treatments alone. Alnylam also has a 300 mg dose cholesterol medication that it expects to begin Phase II clinical testing on in 2016, with approval hoped for in 2017.