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With Funds from R&D Partners, Traversa Advances In-House Programs


By Doug Macron

With revenues coming in from "several" ongoing research and development collaborations, Traversa Therapeutics continues to advance its own in-house drug-development programs.

However, the company will most likely require an additional infusion of capital before it can move any of these into the clinic, Traversa President and CEO Hans Petersen told RNAi News.

Last week, Traversa announced that it had forged a deal with Sanofi-Aventis to explore the use of its so-called PTD-DRBD technology to delivery siRNAs against undisclosed targets (see RNAi News, 3/25/2010).

According to Petersen, the deal is one of "several" that Traversa has signed with large pharmaceutical firms. And, like those other deals, it includes an option through which Sanofi-Aventis could license the technology for use against specific targets.

The arrangements are "providing us with significant revenue to grow the company," he said. They are "also providing us with resources at these companies to continue to develop the platform and to actually develop specific lead candidates."

In collaboration with one of the companies, Traversa has optimized two siRNA drug candidates that are "on their way toward the clinic. In [that] collaboration, we are hopefully moving toward [investigational new drug application]-enabling studies and [product] manufacturing," Petersen said.

He declined to estimate when an IND might be filed, noting that such guidance would be difficult to provide since the programs are "on their timeline more than ours."

The funding received from its R&D partners, as well as a $5 million Series B financing round closed last year (see RNAi News, 4/2/2009), has provided Traversa with " plenty of cash to keep [work] going" through the third quarter of next year, Petersen said.

That work, in addition to the projects with its big pharma partners, includes the characterization and formulation of PTD-DRBD-delivered siRNAs, including an in vivo study examining systemic drug delivery, he noted.

At the same time, the company is exploring intranasal delivery and has in vitro validation of an antiviral effect against respiratory syncytial virus; intraperitoneal delivery; ocular delivery; and intrathecal delivery for pain management indications in critical care settings. This work is being conducted through a series of earlier-stage collaborations, Petersen noted.

In terms of in-house R&D, the company's longstanding interest has been cancer, and "we're still interested in glioblastoma, metastatic ovarian cancer, [and] leukemia," Petersen said. But to advance a program into human testing, he noted, would require some sort of new money-making deal.

"As soon as we come up with a formulation we're happy with and a final molecule, we'll move straight and quickly into IND-enabling studies," he said. However, "to complete a phase I, we'd need either another … research collaboration [such as the one inked with Sanofi-Aventis], we would need [an existing partner] to move forward on a licensing option, or [the company would have to] raise a third round."

Another potential source of funding would be a deal for the reagent rights to the PTD-DRBD technology, which comprises protein transduction domains linked with a double-stranded RNA-binding domain.

According to Traversa, an siRNA coated with PTD-DRBD molecules binds to cell-surface proteoglycans, which stimulates macropinocytosis. The drug then enters the cell inside a macropinosome, at which point the pH inside the vesicle drops and the siRNA is released from the PTD-DRBD molecules into the cytoplasm.

Last year, Integrated DNA Technologies picked up non-exclusive, worldwide rights to distribute the technology for research purposes (see RNAi News, 6/4/2009). IDT markets it in kits under the Transductin name.

Despite having found a partner in IDT, Traversa still wanted to find another distributor. "Different distributors have different specialties," Petersen said at the time. "In the case of IDT, they're very strong on the high-throughput screening side [and] … on the institutional side."

He said this week that Traversa is currently in discussions with another undisclosed company about a distribution deal, but noted that consummating such an arrangement has proven difficult.

"We're seeing companies going through organizational changes," Petersen said. "So occasionally we'll have a term sheet, or an agreement in place in some cases, [but] the champion [of the deal at the company] moves or resigns, or the entire division is closed.

"Any time a structural change occurs, it's like pressing the reset button," he added. "That's what we're running into quite a bit."

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