Alnylam, Tekmira target RNAi therapeutics

In a move aimed at the discovery of novel cationic lipids and lipid nanoparticles for the systemic delivery of RNAi therapeutics, Alnylam Pharmaceuticals Inc. and Tekmira Pharmaceuticals Corp. have joined forces and launched a new company, AlCana Technologies Inc.

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CAMBRIDGE, Mass.—In a move aimed at the discovery of novelcationic lipids and lipid nanoparticles for the systemic delivery of RNAitherapeutics, Alnylam Pharmaceuticals Inc. and Tekmira Pharmaceuticals Corp.have joined forces and launched a new company, AlCana Technologies Inc.
The research collaboration will be funded by Alnylam andconducted by AlCana, a company founded by former Tekmira scientists inassociation with lipid experts in Peter Cullis' lab at the University ofBritish Columbia, which will also participate in the research collaboration.
"Strategically," notes Barry Greene, president and COO ofAlnylam, "continuously improving delivery is a critical area, and with thiscollaboration and others we are on a clear trajectory to achieve low,single-digit microgram per kilogram efficacy with lipid nanoparticles."
Lipid nanoparticle technology for the delivery of RNAi drugsis the most advanced delivery technology in clinical development, says TekmiraCEO Mark Murray.
"Tekmira's SNALP (stable nucleic acid-lipid particle)technology has been shown in preclinical models to be a safe and effective wayto deliver RNAi drugs to disease sites. Cationic lipids are one component ofTekmira's SNALP nanoparticle technology. By changing the components and ratiosof the different SNALP components, including cationic lipids, Tekmira canadjust the properties of a given formulation for delivery of RNAi drugs todifferent organs and tissues in the body," he states.
Under the terms of the two-year research collaboration,Alnylam will receive exclusive rights to all new inventions as well as rightsto sublicense any resulting intellectual property to Alnylam's current andfuture partners. Tekmira receives rights to use new inventions for their ownRNAi therapeutic programs licensed under Alnylam intellectual property throughits InterfeRx program. AlCana will focus its efforts on generating novelcationic lipids that can be incorporated into lipid nanoparticles for thedelivery of RNAi drugs.
In March 2008, Alnylam granted Tekmira InterfeRx licenses todiscover, develop and commercialize RNAi therapeutics towards seven genetargets—one of which is apolipoprotein B (ApoB). Tekmira has initiated a PhaseI human clinical trial for ApoB SNALP, which is being developed as a treatmentfor patients with high LDL cholesterol, or "bad" cholesterol, and expects tocomplete the Phase I trial in early 2010. Alnylam is eligible to receivemilestones and royalties based on the development and commercialization of theprogram. 
Alnylam also has a program in Phase I trials formulatedusing Tekmira's SNALP technology, Greene notes. Called ALN-VSP, the proprietaryAlnylam program is being developed for the treatment of liver cancers andpotentially other solid tumors. ALN-VSP targets two key genes involved in thedisease pathway of liver cancer: KSP (kinesin spindle protein), which isinvolved in cancer proliferation, and VEGF (vascular endothelial growthfactor), which is involved in cancer angiogenesis. In April 2009, ALN-VSPentered into a Phase I multi-center, open label, dose escalation study forpatients around the age of 55 with advanced solid tumors with liverinvolvement, who have failed to respond to or have progressed after standardtreatment. 
"We expect to complete enrollment and present preliminarydata from this study in mid-2010," Greene states.
Tekmira is eligible to receive milestones and royaltiesbased on the development and commercialization of any Alnylam program that usesTekmira's proprietary technologies, including SNALP.
RNAi drugs act to target the root cause of disease bysilencing genes associated with
disease, thereby acting upstream of today's therapies. Thecommercial potential will
be driven by the specific indications treated by the RNAidrugs, Murray says.
"It is too early to be more specific than that," he states."We believe RNAi represents a breakthrough in biology that has the opportunityto treat diseases in a fundamentally new way by targeting virtually any gene inthe genome involved in the causal pathway of disease, including genes that arecurrently 'undrugable' using today's medicines," Greene adds.
Alnylam is developing RNAi therapeutics for the treatment ofa wide range of diseases, including liver cancers, hypercholesterolemia,Huntington's disease and TTR amyloidosis. The company has alliances withMedtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin and Cubist.

Alnylam and Regulus receive U.S. patent covering microRNAtherapeutics
CAMBRIDGE, Mass.—Alnylam Pharmaceuticals Inc. and RegulusTherapeutics Inc. announced Aug. 11 that the U.S. Patent and Trademark Office(USPTO) awarded a notice of allowance for the Manoharan patent (Application No.11/200,703), which covers several chemical classes of anti-miRs, antisenseoligonucleotide inhibitors of microRNAs. This new patent in combination with Regulus'existing intellectual property estate broadly covers approaches to developingmicroRNA-based therapeutics.
"We are pleased to have this important recognition from theUSPTO regarding our discovery of antagomirs, a new class of anti-miRs and anovel invention in the field of microRNA-based therapeutics," says Dr. MuthiahManoharan, vice president for drug discovery at Alnylam. "With this initialgrant, we have received broad claims covering classes of anti-miRs with certainimportant chemical features for optimal delivery and in vivo pharmacology.Importantly, the inventive nature of these novel compositions was allowedindependent of any specific microRNA sequence, thereby providing a broad set ofpatent claims that add to the innovations Regulus can employ in designing novelmicroRNA therapeutics, an entire new class of medicines."
"Regulus Therapeutics is advancing a whole new frontier ofpharmaceutical research through the discovery of microRNA therapeutics, whichhave the potential to block broad disease pathways as opposed to a singletarget," says Dr. Kleanthis G. Xanthopoulos, president and CEO of RegulusTherapeutics. "Our access to the pioneering research of our academiccollaborators and Alnylam and Isis on oligonucleotide-based therapeutics, asevidenced most recently by the award of the Manoharan patent, has enabledRegulus to build a dominant intellectual property estate for microRNAtherapeutics. We believe this is a strong foundation to advance our innovativeapproach to developing novel medicines to address the needs of patients."

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