Archive for the ‘RNAi’ Category
Apr
20
Posted under
ALN-PCS,
Alnylam Pharmaceuticals,
Amgen,
Blog,
cholesterol,
Companies,
Diagnostics,
Funding,
Medical Devices,
Medical Supply,
PCSK9 drugs,
Pharmaceuticals,
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RNAi,
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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
Apr
11
Posted under
Arrowhead Research,
Biogen Idec,
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Arrowhead Research has bought Alvos Therapeutics and the small company's peptides that home in on tumor targets. Alvos is getting 315,467 restricted shares in Arrowhead ($ARWR) common stock upfront, with $23.5 million in additional stock awards tied to development and regulatory milestones.
Alvos, previously called Mercator Therapeutics, boasts founders from MD Anderson Cancer Center and veteran biotech insiders Roy Lobb, a former Biogen Idec ($BIIB) scientist and co-founder of Avila Therapeutics (sold to Celgene ($CELG) for $350 million in January) as well as Mark Leuchtenberger, who is chief executive of antibiotics developer Rib-X Pharma, a 2011 Fierce 15 company. Alvos raised $2 million in a seed round in summer 2010 to license homing peptides from MD Anderson that have shown an ability to carry drugs to tumors while limiting side effects on healthy tissues.
Arrowhead, which bought Swiss drug giant Roche's ($RHHBY) RNA-interference (RNAi) assets last year, says Alvos' homing peptides could help the company deliver RNAi treatments into cancer cells and carry a variety of other anti-cancer drugs. One of the major challenges in RNAi drug development has been delivering the gene-silencing therapies to tissues deep in the body, and earlier efforts to achieve this have fallen short. The programs under development at Alvos (which had been operating in the Boston area at the time of its 2010 seed funding) will move to Arrowhead's R&D outpost in Madison, WI, according to the buyer.
Alvos, clearly, never matured as much as Lobb and Leuchtenberger's previous companies, and a vast majority of the payout from Arrowhead will depend on the future success of the biotech's programs. Leuchtenberger hasn't returned a message left on his cell phone this morning to discuss the deal. Arrowhead is planning a conference call to review the transaction at 4:30 pm ET.
In Arrowhead's release, Lobb, the chief scientist at Alvos, said: "We were attracted to Arrowhead as an acquirer because of its ability to accelerate development of the platform through its excellent scientific staff and state-of-the-art facilities."
In 2010, Leuchtenberger told me for an article in Xconomy that a special aspect of the technology the company licensed from MD Anderson was the ability to do organ-specific screening of peptides in humans with cancer, enabling researchers to find peptide-receptor pairs that could help them develop treatments that bring targeted fights to tumors.
- here's Arrowhead's release
Related Article:
Roche hands over RNAi assets to Arrowhead Research
Jan
20
Posted under
Alnylam Pharmaceuticals,
Blog,
Companies,
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layoffs,
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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 $260 million in cash to make it happen.
"Now is the time to focus our near-term efforts and resources on what we believe to be our highest value opportunities; specifically, accelerated clinical development plans for our programs in transthyretin-mediated amyloidosis and hemophilia," Maraganore said in a statement.
- here's the company's release
- check out Dow Jones Newswires' report
- and Xconomy's article
Related Articles:
Countering skeptics, Alnylam claims pioneering first in RNAi cholesterol study
As early hype fades, pharma steadily backs away from RNAi
Jan
04
Posted under
ALN-PCS,
Alnylam Pharmaceuticals,
Blog,
Companies,
Diagnostics,
Funding,
Medical Devices,
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Pharmaceuticals,
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Videos by John Carroll
Facing off against some deep-seated skepticism about the future of RNAi drug development, Alnylam ($ALNY) has pushed out a preliminary assessment of a small Phase I study demonstrating some dramatic results in sharply reducing the levels of bad cholesterol in patients suffering from severe hypercholesterolemia. And tackling doubts about being able to effectively deliver an RNAi treatment, Alnylam touted the use of second-generation lipid nanoparticles in the study.
The bottom line on the study is impressive: Patients taking ALN-PCS experienced a reduction of LDL-C of 50% or more. And the biotech unabashedly claimed an historic first in the study.
"We are extremely pleased with these data from our ALN-PCS trial which represent what we believe is the first ever demonstration of efficacy for an RNAi therapeutic toward a clinically validated endpoint, namely LDL-C, a defined risk factor for coronary artery disease and acute myocardial infarction. Indeed, preliminary results from our ongoing study show robust, statistically significant, and dose-dependent lowering of both PCSK9 and LDL-C levels," said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. "The RNAi effects were rapid and durable after a single dose, exemplifying a compelling profile for RNAi therapeutics that we have now established in man for two disease programs."
The RNAi world has been rocked by a number of high-level defections in the past two years as pharma companies assessed the time, costs and efforts needed to get a treatment through clinical trials and on to an approval. Encouraging efficacy data from a small Phase I, while helpful, is unlikely to dramatically change anyone's mind about that. But it is a step forward for a field that has been forced to contend with a bitter chill.
- here's the Alnylam release
Related Articles:
ASCO confab gives Alnylam, RNAi a shot to shine
As early hype fades, pharma steadily backs away from RNAi
Alnylam announces 25-30% reduction of workforce