Biogen Joins With Alnylam On RNAi Therapies For PML.
BIOWORLD Today - Sep. 23, 2006
Byline: Jennifer Boggs, Staff Writer
Biogen Idec Inc. and Alnylam signed a potential $56 million early stage deal to discover RNAi-based treatments targeting progressive multifocal leukoencephalopathy (PML), a brain infection linked in rare cases to the use of Biogen's multiple sclerosis drug Tysabri.
PML, a disease caused by the JC virus, typically occurs in patients who are immune-suppressed, either from drug treatment or disease, and is almost always fatal.
Last year, Biogen and partner Dublin, Ireland-based Elan Corp. plc voluntarily pulled Tysabri (natalizumab) from the market following two patient deaths from PML; however, the companies resubmitted the drug for approval after further study and, in June, the FDA granted Tysabri a second chance in a limited capacity in MS patients and with an accompanying risk-management plan. (See BioWorld Today, June 6, 2006.)
"This early stage research collaboration is one of many efforts that we've undertaken to understand and manage the risk of PML," said Jose Juves, spokesman for Cambridge, Mass.-based Biogen.
Joining forces with Alnylam will allow Biogen to look at RNAi-based therapies, "which we think might work by inhibiting the production of the JC viral proteins that cause PML," Juves added.
Alnylam, also of Cambridge, already has done extensive work in antiviral RNAi drugs. It has completed two Phase I trials with its lead program, ALN-RSV01, a drug aimed at respiratory syncytial virus infection by silencing a viral gene in infected lungs. Beyond that, the company is working on an RNAi antiviral against pandemic flu.
Alnylam "has deep expertise in developing antiviral RNAi therapeutics and also in delivering RNAi to [the central nervous system] -- both the brain and the periphery," Barry Greene, Alnylam's chief operating officer told BioWorld Today. So the Biogen collaboration "is a very synergistic program, from our perspective."
Terms of the deal call for Biogen to pay Alnylam a $5 million up-front fee and fund all research and development activities. Alnylam also would be entitled to up to $51 million for reaching certain preclinical and clinical milestones, plus royalties.
In addition, Alnylam would receive utilization fees for any product resulting from the collaboration, which Greene said was "a unique structure put in place" due to the small number of patients diagnosed with PML.
"Neither company was looking at significant monetary gains from this population," Greene said, estimating that the number of patients range anywhere from "the upper hundreds the low thousands," comprising patients on immunosuppressive drugs as well as those with immune system disorders, such as HIV.
The inclusion of use fees ensures that Alnylam "can recognize significant compensation for overall effort," he added.
Collaboration work will focus on the discovery and development of RNAi therapeutics that target the JC virus, a virus that has been well sequenced, with specific genes identified that are responsible for the virus' replication.
"So the strategy here is to identify highly conserved sequences of those genes," Greene said, and then "to develop siRNAs [small-interfering RNAs] against those viruses."
The first step will be to design and optimize an RNAi drug that can be directed specifically to the JC virus genome, and then move to preclinical work. Alnylam said it's too early to disclose a timeline for advancing a product into the clinic, but said it will be working with Biogen and the FDA to determine a regulatory pathway.
Biogen is the sixth major partner Alnylam has signed, and the "first classic biotech company" to enter the RNAi space, Greene said.
Alnylam's other collaborators include large pharma firms such as Basel, Switzerland-based Novartis AG and Whitehouse Station, N.J.-based Merck & Co. Inc., and medtech firm Medtronic Inc., of Minneapolis.
To date, its collaborations have brought Alnylam $115 million in cash and continue to provide funding that the company uses to "develop our overall pipeline of RNAi therapeutics," Greene said. "It really fits with our goal to build a long term and valuable company."
Alnylam reported a net loss of $9.9 million, or 31 cents per share, for the second quarter. As of June 30, the company had a cash position of $123.3 million.
Its shares (NASDAQ:ALNY) closed at $13.48 Thursday, down 54 cents.
Biogen's stock (NASDAQ:BIIB) closed at $43.66 Thursday, unchanged.
http://www.therapeuticsdaily.com/news/article.cfm?contentValue=1089604&contentType=sentryarticle&channelID=30
Byline: Jennifer Boggs, Staff Writer
Biogen Idec Inc. and Alnylam signed a potential $56 million early stage deal to discover RNAi-based treatments targeting progressive multifocal leukoencephalopathy (PML), a brain infection linked in rare cases to the use of Biogen's multiple sclerosis drug Tysabri.
PML, a disease caused by the JC virus, typically occurs in patients who are immune-suppressed, either from drug treatment or disease, and is almost always fatal.
Last year, Biogen and partner Dublin, Ireland-based Elan Corp. plc voluntarily pulled Tysabri (natalizumab) from the market following two patient deaths from PML; however, the companies resubmitted the drug for approval after further study and, in June, the FDA granted Tysabri a second chance in a limited capacity in MS patients and with an accompanying risk-management plan. (See BioWorld Today, June 6, 2006.)
"This early stage research collaboration is one of many efforts that we've undertaken to understand and manage the risk of PML," said Jose Juves, spokesman for Cambridge, Mass.-based Biogen.
Joining forces with Alnylam will allow Biogen to look at RNAi-based therapies, "which we think might work by inhibiting the production of the JC viral proteins that cause PML," Juves added.
Alnylam, also of Cambridge, already has done extensive work in antiviral RNAi drugs. It has completed two Phase I trials with its lead program, ALN-RSV01, a drug aimed at respiratory syncytial virus infection by silencing a viral gene in infected lungs. Beyond that, the company is working on an RNAi antiviral against pandemic flu.
Alnylam "has deep expertise in developing antiviral RNAi therapeutics and also in delivering RNAi to [the central nervous system] -- both the brain and the periphery," Barry Greene, Alnylam's chief operating officer told BioWorld Today. So the Biogen collaboration "is a very synergistic program, from our perspective."
Terms of the deal call for Biogen to pay Alnylam a $5 million up-front fee and fund all research and development activities. Alnylam also would be entitled to up to $51 million for reaching certain preclinical and clinical milestones, plus royalties.
In addition, Alnylam would receive utilization fees for any product resulting from the collaboration, which Greene said was "a unique structure put in place" due to the small number of patients diagnosed with PML.
"Neither company was looking at significant monetary gains from this population," Greene said, estimating that the number of patients range anywhere from "the upper hundreds the low thousands," comprising patients on immunosuppressive drugs as well as those with immune system disorders, such as HIV.
The inclusion of use fees ensures that Alnylam "can recognize significant compensation for overall effort," he added.
Collaboration work will focus on the discovery and development of RNAi therapeutics that target the JC virus, a virus that has been well sequenced, with specific genes identified that are responsible for the virus' replication.
"So the strategy here is to identify highly conserved sequences of those genes," Greene said, and then "to develop siRNAs [small-interfering RNAs] against those viruses."
The first step will be to design and optimize an RNAi drug that can be directed specifically to the JC virus genome, and then move to preclinical work. Alnylam said it's too early to disclose a timeline for advancing a product into the clinic, but said it will be working with Biogen and the FDA to determine a regulatory pathway.
Biogen is the sixth major partner Alnylam has signed, and the "first classic biotech company" to enter the RNAi space, Greene said.
Alnylam's other collaborators include large pharma firms such as Basel, Switzerland-based Novartis AG and Whitehouse Station, N.J.-based Merck & Co. Inc., and medtech firm Medtronic Inc., of Minneapolis.
To date, its collaborations have brought Alnylam $115 million in cash and continue to provide funding that the company uses to "develop our overall pipeline of RNAi therapeutics," Greene said. "It really fits with our goal to build a long term and valuable company."
Alnylam reported a net loss of $9.9 million, or 31 cents per share, for the second quarter. As of June 30, the company had a cash position of $123.3 million.
Its shares (NASDAQ:ALNY) closed at $13.48 Thursday, down 54 cents.
Biogen's stock (NASDAQ:BIIB) closed at $43.66 Thursday, unchanged.
http://www.therapeuticsdaily.com/news/article.cfm?contentValue=1089604&contentType=sentryarticle&channelID=30
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