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Tekmira Touts Advances with Lipid Nanoparticle Technology

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By Doug Macron

Tekmira Pharmaceuticals' top official this week offered a few new details about refinements being made to the company's core lipid nanoparticle delivery technology, stating that efforts are underway to add antibody-based targeting moieties and to enable respiratory administration.

Tekmira also released its 2010 financial results, posting a jump in its net loss despite increased revenues.

According to Tekmira President and CEO Mark Murray, the company is working on next-generation versions of LNPs that have been shown to “achieve therapeutic indices superior to any currently available.

“We will be applying these new LNP formulations to a number of targets in our early pipeline … as we consider them for product development,” he said during a conference call held to discuss the 2010 financial results.

He did not provide details about what drugs might be next in Tekmira's pipeline, but noted that the company is exploring the possibility to re-introducing its hypercholesterolemia candidate TKM-ApoB with a new LNP formulation.

The drug is designed to inhibit apolipoprotein B and was Tekmira's first clinical candidate. However, in early 2010, the company put a phase I study on hold after a patient experienced side effects consistent with immune stimulation triggered by the siRNA component (GSN 1/14/2010). Although the trial was expected to restart later that year, disappointing preclinical testing with different siRNAs led Tekmira to keep the program shelved indefinitely (GSN 9/16/2010).

This week, Murray said Tekmira would provide new guidance on the drug once the company finishes evaluating next-generation LNPs and other product opportunities.

Meanwhile, Tekmira has been working with an undisclosed antibody company to test the use of antibody fragments to target LNPs to specific cell-surface receptors. He did not provide additional details, but said that the firm has established the feasibility of the approach.

The company has also developed “novel LNP chemistries” that enable LNP particles to survive the process of nebulization, Murray said during the call. “This will open therapeutic potential for our LNP technology for respiratory disease, and is of interest to a major pharmaceutical company [with which] ...we have recently initiated a collaboration.”

He added that Tekmira and the undisclosed partner are planning to test the nebulized particles in in vivo models, but did not elaborate.

RIP, SNALP

Although Tekmira has transitioned into a full-fledged RNAi drug developer, its primary asset is its lipid-based delivery technology. The company formally referred to the stable nucleic-acid lipid particles as SNALPs, but it has more recently started using the more generic lipid nanoparticle moniker.

The LNP approach has been the driver behind a number of collaborations for Tekmira, including ones with Takeda Pharmaceutical, Alnylam Pharmaceuticals, and Roche, which has since been discontinued following the company's withdrawal last November from the RNAi space (GSN 11/18/2010).

At the same time, Tekmira is using the technology in its own pipeline programs TKM-PLK1, an siRNA-based cancer drug currently in phase I testing, and TKM-Ebola, a preclinical effort to develop a treatment for Ebola infection being conducted with funding from the US Department of Defense Chemical and Biological Defense Program (GSN 10/14/2010).

Murray said this week that initial data on TKM-PLK1 is expected before the end of 2011, while an investigational new drug application filing on TKM-Ebola is slated for the second half of the year.

Financials

In 2010, Tekmira's net loss rose to $12.4 million, or $1.20 per share, from a loss of $8.7 million, or $0.85 a share, the year before. The increase primarily reflects higher research, development, collaboration, and contract spending for both internal and partnered drug-development programs.

Specifically, the company's expenses in these areas jumped to $22.1 million in 2010 from $17.8 million in 2009. Meanwhile, general and administrative costs climbed to $4.8 million from $4.2 million, largely as a result of costs associated with Tekmira's listing of its shares on the Nasdaq.

Revenues for 2010 jumped to $21.4 million from $14.4 million year over year, which included $6.3 million from Alnylam and $3.6 million from the US government related to the Ebola program.

As of Dec. 31, Tekmira had cash and cash equivalents totaling $12.3 million.

Looking ahead, Tekmira said that 2011 collaboration and contract revenues are expected to exceed 2010 levels, with payments related to the ebola program offsetting the loss of Roche funding. Total R&D spending is also expected to increase this year, again related to costs tied to the ebola program.

General and administrative costs in 2011 may also exceed 2010 levels, but this depends on the outcome of Tekmira's lawsuit against Alnylam.

In March, Tekmira announced that it had sued Alnylam for allegedly misappropriating trade secrets and other information related to its LNP technology (GSN 3/17/2011). Among the charges in the suit is that Alnylam used the trade secrets “for its own internal purposes and to replicate a competing technology.”

“Alnylam repeatedly went so far as to use our proprietary delivery technology to apply for patents based on our confidential information, claiming as its own the very technology that it stole,” Murray said at the time the suit was disclosed.

During this week's call, he said he would not comment on the litigation beyond stating that Tekmira would pursue it “without interruption to our core business activities, and we intend to fulfill all of our manufacturing obligations to Alnylam.”

Based on its current funds and expected income from partners, Tekmira said its cash position is sufficient to fund continued product development into 2012.


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