Making siRNA research a SNALP
Tekmira to use stable nucleic acid-lipid particle technology to deliver Pfizer’s siRNA molecules
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VANCOUVER, B.C.—Tekmira Pharmaceuticals Corp. recently announced the initiation of a research collaboration with Pfizer Inc. According to Mark Murray, president and CEO of Tekmira, the collaboration focuses on evaluating Tekmira's stable nucleic acid-lipid particle (SNALP) technology to deliver small-interfering RNA (siRNA) molecules provided by Pfizer.
RNAi therapeutics have the potential to treat a broad number of human diseases by "silencing" disease-causing genes. The discoverers of RNAi, a gene silencing mechanism used by all cells, were awarded the 2006 Nobel Prize for Physiology or Medicine.
RNAi therapeutics, such as "siRNAs," require delivery technology to be effective systemically. Lipid nanoparticles (LNPs) are one of the most widely used siRNA delivery approaches for systemic administration. Tekmira says its SNALP (stable nucleic acid-lipid particles) technology is the leading class of LNPs being used in clinical development.
SNALP technology encapsulates siRNAs with high efficiency in uniform lipid nanoparticles that are effective in delivering RNAi therapeutics to disease sites in numerous preclinical models. SNALP-based products have been reviewed by multiple FDA divisions for use in clinical trials. SNALP formulations comprise several lipid components that can be adjusted to suit the specific application.
"Tekmira will be responsible for preparing the SNALP formulations, and Pfizer will evaluate the formulations in preclinical models," Murray says.
The companies have not revealed financial terms of the collaboration. Specific diseases or therapeutic areas also have not been disclosed.
"We are pleased to be working with Pfizer, one of the world's leading pharmaceutical companies and an organization that has made a commitment to the development of nucleic acid therapeutics," adds Murray. "The collaboration combines Tekmira's expertise in the delivery of RNAi therapeutics with Pfizer's research excellence in nucleic acid therapeutics."
Murray points out that Tekmira continues to collaborate with pharmaceutical and biotechnology companies, thereby broadening the use of its SNALP delivery platform, with a goal to sign additional collaborative agreements in 2010.
"Tekmira's SNALP technology is the leading class of lipid nanoparticles being used in clinical development for the delivery of RNAi therapeutics," adds Murray. "SNALP technology efficiently encapsulates siRNAs in uniform lipid nanoparticles and enables the effective delivery of RNAi therapeutics to sites of disease. SNALP formulations are manufactured using a proprietary method that is robust, scalable and easily reproducible."
Pooya Hemami, an analyst with Desjardins Securities, says the collaboration adds further validity and support to Tekmira's SNALP technology, and that is what is driving investor interests.
This is Tekmira's first formal research collaboration with Pfizer. Murray notes that Tekmira also is working with Alnylam Pharmaceuticals Inc. and Swiss drugmaker Roche Holding AG, both of which are developing drugs using the SNALP technology.
"So if other companies like Bristol-Myers Squibb and Pfizer, which have initial agreements with Tekmira, were to develop more comprehensive licensing agreements, it would be very positive to the extent of the depth of the potential revenue for the technology," Hemami says.
One of the key issues raised with the SNALP technology is safety. In January, an early-stage trial for Tekmira's own internal cholesterol drug candidate using the same technology showed that one of the two patients, treated at the highest dose level, experienced flu-like symptoms.
"My main concern really, and I think for a lot of analysts covering the company, is the safety of the technology. There were concerns with the first phase study of ApoB SNALP as there was a case of flu-like response," Hemami notes.
RNAi therapeutics have the potential to treat a broad number of human diseases by "silencing" disease-causing genes. The discoverers of RNAi, a gene silencing mechanism used by all cells, were awarded the 2006 Nobel Prize for Physiology or Medicine.
RNAi therapeutics, such as "siRNAs," require delivery technology to be effective systemically. Lipid nanoparticles (LNPs) are one of the most widely used siRNA delivery approaches for systemic administration. Tekmira says its SNALP (stable nucleic acid-lipid particles) technology is the leading class of LNPs being used in clinical development.
SNALP technology encapsulates siRNAs with high efficiency in uniform lipid nanoparticles that are effective in delivering RNAi therapeutics to disease sites in numerous preclinical models. SNALP-based products have been reviewed by multiple FDA divisions for use in clinical trials. SNALP formulations comprise several lipid components that can be adjusted to suit the specific application.
"Tekmira will be responsible for preparing the SNALP formulations, and Pfizer will evaluate the formulations in preclinical models," Murray says.
The companies have not revealed financial terms of the collaboration. Specific diseases or therapeutic areas also have not been disclosed.
"We are pleased to be working with Pfizer, one of the world's leading pharmaceutical companies and an organization that has made a commitment to the development of nucleic acid therapeutics," adds Murray. "The collaboration combines Tekmira's expertise in the delivery of RNAi therapeutics with Pfizer's research excellence in nucleic acid therapeutics."
Murray points out that Tekmira continues to collaborate with pharmaceutical and biotechnology companies, thereby broadening the use of its SNALP delivery platform, with a goal to sign additional collaborative agreements in 2010.
"Tekmira's SNALP technology is the leading class of lipid nanoparticles being used in clinical development for the delivery of RNAi therapeutics," adds Murray. "SNALP technology efficiently encapsulates siRNAs in uniform lipid nanoparticles and enables the effective delivery of RNAi therapeutics to sites of disease. SNALP formulations are manufactured using a proprietary method that is robust, scalable and easily reproducible."
Pooya Hemami, an analyst with Desjardins Securities, says the collaboration adds further validity and support to Tekmira's SNALP technology, and that is what is driving investor interests.
This is Tekmira's first formal research collaboration with Pfizer. Murray notes that Tekmira also is working with Alnylam Pharmaceuticals Inc. and Swiss drugmaker Roche Holding AG, both of which are developing drugs using the SNALP technology.
"So if other companies like Bristol-Myers Squibb and Pfizer, which have initial agreements with Tekmira, were to develop more comprehensive licensing agreements, it would be very positive to the extent of the depth of the potential revenue for the technology," Hemami says.
One of the key issues raised with the SNALP technology is safety. In January, an early-stage trial for Tekmira's own internal cholesterol drug candidate using the same technology showed that one of the two patients, treated at the highest dose level, experienced flu-like symptoms.
"My main concern really, and I think for a lot of analysts covering the company, is the safety of the technology. There were concerns with the first phase study of ApoB SNALP as there was a case of flu-like response," Hemami notes.
