Alnylam scores efficacy data with PCSK9 drug

Posted under ALN-PCS, Alnylam Pharmaceuticals, Amgen, Blog, cholesterol, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, PCSK9 drugs, Pharmaceuticals, Pipeline, RNAi, Startups, Universities, Videos by rmcbride

Alnylam Pharmaceuticals' ($ALNY) stock rose this morning after the developer of gene-silencing drugs touted early-stage trial data on its RNA-interference drug that zeros in on one of the hottest targets in biotech. The data show that the small company has a horse in the race to advance PCSK9-targeting drugs as a brand new category of treatments for lowering LDL cholesterol.

The Cambridge, MA-based company's stock had jumped 5.6% as of 10:48 a.m. ET after Alnylam revealed the Phase I trial results early this morning.

The Phase I study met its primary goal of safety and tolerability of the treatment, called ALN-PCS, which was given to patients without cholesterol-lowering statins. And patients on the drug in the 32-person study had an average drop in LDL or "bad" cholesterol of 41% compared with baseline and placebo as well as a mean reduction in PCSK9 protein levels of 68% in the highest dose group. Alnylam says it plans to find a partner for the program before moving ahead with mid-stage trials with the drug.

Alnylam isn't as advanced in clinical development with its PCSK9-targeting drug as AMG 145, Amgen's ($AMGN) contender, or REGN727, which is Sanofi ($SNY) and Regeneron's ($REGN) drug that targets the protein. However, Alnylam's program could attract a partner interested in having a stake in PCSK9 drugs, which could offer an alternative cholesterol-lowering treatment for patients who don't respond to existing drugs such as statins. Analysts say the PCSK9-targeting class could be worth billions of dollars per year if they make it onto the market.

Alnylam also noted that ALN-PCS uses the company's next-generation lipid nanoparticle delivery technology, and the upbeat results from the company's small study could bode well for other drugs in its pipeline that use the delivery tech, such as ALN-TTR02. Delivery challenges have dogged the RNAi field, and Alnylam has been working on providing data that its next-gen drugs can overcome some of the hurdles that plagued programs of the past.

- here's Alnylam's release
- and an RTTNews report

Related Articles:
Developers spotlight huge potential of new class of cholesterol drugs
Countering skeptics, Alnylam claims pioneering first in RNAi cholesterol study
Sanofi, Regeneron report stellar LDL drug data as race heats up
As early hype fades, pharma steadily backs away from RNAi

Alnylam identifies who’s getting ax

Posted under Alnylam Pharmaceuticals, Blog, Companies, Diagnostics, Funding, layoffs, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by John Carroll

Alnylam Pharmaceuticals ($ALNY) has detailed plans for laying off 52 workers, part of a broad effort to slash its RNA workforce 33%. The Boston Business Journal reports that the biotech told state officials it is axing its senior director of biotherapeutics, a senior director of business development, a senior director of pharma operations and a senior director of information technology. The Journal reports that "nine senior scientists and 14 research associates positions will also be eliminated." Story

Alnylam cutting third of jobs to devote cash to lead RNAi programs

Posted under Alnylam Pharmaceuticals, Blog, Companies, Diagnostics, Funding, layoffs, Medical Devices, Medical Supply, Pharmaceuticals, RNAi, Startups, Universities, Videos by Ryan McBride

Alnylam Pharmaceuticals ($ALNY), a developer of RNA-interference (RNAi) drugs, is axing about 33% of its workforce in a restructuring plan that aims to steer the company's dollars into developmental programs, the company said Thursday. And the layoffs come after Big Pharma outfits such as Roche and Merck ($MRK) have made major cutbacks to research in the RNAi field, which has yet to yield any marketed drugs.

With its smaller staff, Cambridge, MA-based Alnylam plans to make its early-stage RNAi therapies for transthyretin-mediated amyloidosis and hemophilia its lead candidates. Though neither of the programs has advanced beyond Phase I, according to the company's website, and the projects will require significant investment to take through clinical trials. The company expects the restructuring to save $20 million in 2012 and cost $4 million in one-time expenses.

Alnylam has shifted from a fast-growing biotech several years back when it was bagging major licensing and R&D deals with Novartis ($NVS), Roche, Takeda Pharmaceutical and others to a company that is shrinking. The company axed 25% to 30% of its workers in September 2010, shortly after its 5-year research deal with Novartis ended. Major drugmakers pledged or spent billions of dollars on RNAi drug research in the 2000s, yet more recently companies have made cutbacks in the field amid major challenges in translating the gene-silencing tech into drugs. Merck, which spent $1.1 billion to acquire the South San Francisco-based RNAi drug research group Sirna Therapeutics in 2006, closed the former Sirna shop last summer.

As one would expect, Alnylam CEO John Maraganore (who didn't even hint about the restructuring during a meeting with FierceBiotech at the J.P. Morgan Healthcare Conference last week) has kept the faith that RNAi will yield great new drugs. His recent strategy calls for the company to focus on products after years of investing in early research, and his group ended 2011 with about $260 million in cash to make it happen.

"Now is the time to focus our near-term efforts and resources on what we believe to be our highest value opportunities; specifically, accelerated clinical development plans for our programs in transthyretin-mediated amyloidosis and hemophilia," Maraganore said in a statement.

- here's the company's release
- check out Dow Jones Newswires' report
- and Xconomy's article

Related Articles:
Countering skeptics, Alnylam claims pioneering first in RNAi cholesterol study
As early hype fades, pharma steadily backs away from RNAi

Biotech execs leave J.P. Morgan with mixed feelings about year ahead

Posted under Alnylam Pharmaceuticals, Big Pharma, Biotech IPO, Blog, Companies, Concert Pharmaceuticals, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by Biotech News

 By John Carroll, Mark Hollmer and Ryan McBride

Wheeling and dealing at J.P. Morgan 2012 Launch slideshow >>

Every year everyone who either qualifies as a top dealmaker in biotech or aspires to be one converges on San Francisco at the J.P. Morgan Healthcare Conference to check the pulse of the financial markets. It's where you talk programs and deal terms, hopes and aspirations. And this year's gathering had its share of bright new ideas and pioneering technologies, tempered by the simple reality that the key financial trends we'll see in 2012 have already been playing out for several tough years.

"I don't see any significant changes," says Ernst & Young's longtime life sciences analyst Glen Giovannetti. This year, he adds, shouldn't end up all that different from 2011, or 2010 for that matter. With the Eurozone crisis adding to the financial alarms that have kept many investors sidelined since 2008, "you're not going to get mainstream investors in biotech."

That means little or no chance of a stellar IPO success in the immediate future for clinical-stage drug developers and a continued constrained environment for venture groups, which will struggle to find timely exits.

With dwindling resources for privately held drug developers, leaders are calling on Big Pharma outfits to pump dollars into the biotech ecosystem. "The ecosystem is shrinking," says Roger Tung, CEO of Concert Pharmaceuticals, a venture-backed group based in Lexington, MA. "VCs don't have the same firepower that they did 5 years ago. I think pharma is going to have to step up to feed their own ecosystem where they get a lot of their products from."

The situation has become so bad, Alnylam ($ALNY) CEO John Maraganore joined Glaxo's ($GSK) Moncef Slaoui and retired investment banker Stelios Papadopoulos to pen an op-ed piece calling on Big Pharma to join forces and pump billions of dollars into new funds that will be charged with backing biotech IPOs, giving developers the kind of financial support needed to get the industry growing again at a rapid pace.

"I worry a lot about companies not getting funded because there's no IPO window," says Alnylam's Maraganore. A revived public market could give investors the exits they need and go a long way to resolving the cash crunch.

On the other hand, despite all the grim forecasts from the end of 2011, venture groups continue to pump money into new companies and existing biotechs that can toe the line on science and data points. Canaan Partners and Flagship Ventures helped underscore that point over the course of the week, as they timed announcements on $470 million in new venture cash for the industry. Flagship's new $270 million fund was oversubscribed, but the venture group isn't in a celebratory mood. 

When we asked Flagship chief Noubar Afeyan about the mood of his investors, he underscored how discouraged many had become. "It is a tougher environment." Success will be reserved for the best and the brightest.

"In this biotech jungle," says Forbion Capital Partners Chairman Bart Bergstein, "the toughest and smartest will survive."

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