Kylin Therapeutics last week announced that it has expanded its collaboration with Purdue University to include additional in vivo studies evaluating the company’s core RNAi platform in a variety of solid tumors in animal models.
As a small company — the firm employs five people — Kylin must keep its focus on therapeutic targets that have been evaluated in recently completed in vivo studies, company Co-founder, President, CEO Eric Davis told RNAi News last week.
“But we wanted to continue to broaden the utility of the platform,” he said. To do so, collaborators at Purdue’s Bindley Bioscience Center will conduct experiments that “focus on a number of different targets in a number of different animal models [to] see if we can continue to get positive results with other [undisclosed] solid-tumor models.”
Although Kylin has maintained sponsored-research arrangements with investigators at Purdue, including company co-founder Peixuan Gou, and collaborators at the University of Cincinnati, the decision to expand its relationship with Purdue comes amid a slight change in the course Davis had charted earlier this year.
In February, after securing a $250,000 grant from the Indiana Economic Development Corporation and closing a $1.2 million round of seed financing, he told RNAi News that the company was considering adding as many as six new staffers over 2008 to support new in vivo studies.
“The academic environment can take [a technology] so far, and [Purdue] had taken it through a lot of in vitro validation and … some animal work,” Davis said at the time. “But to take it to the next level [requires] more standardized in vivo tests and in vivo results. Obviously, it takes people to do that.”
Last week, however, he said that Kylin has added just two new employees since February and that it has no plans to hire any more this year. At the same time, the company has opted to conduct certain animal studies at Purdue.
“To us, it doesn’t matter [where the researchers are employed] as long as work is getting done,” Davis said last week. “Through our sponsored-research agreement … with Purdue, we’re able to do a lot of work on a sort of outsourced basis.”
“To us, it doesn’t matter [where the researchers are employed] as long as work is getting done. Through our sponsored-research agreement … with Purdue, we’re able to do a lot of work on a sort of outsourced basis.”
Going forward, Davis said Kylin’s in-house research team will focus primarily on the programs that have thus far yielded promising data, while earlier-stage efforts will mostly be advanced by Purdue collaborators.
“When you’re a small company, you basically focus on what you know works and continue to advance that further down the pipeline,” Davis said. “But because we have a platform we think is broadly applicable, we didn’t want to stop working on other projects. A lot of that early work can be appropriately done in an academic environment.”
The expanded collaboration with Purdue “is going to allow us to really continue to broaden the platform … [by] extending things we probably would have waited to do,” he added.
Although Davis declined to disclose the exact nature of that new work, he noted last week that Guo had previously knocked down a variety of different genes using several different targeting moieties in vitro using the company’s RNAi technology.
“We haven’t, to date, been able to test all of those in an in vivo environment,” he said. “What we have been able to [test], we’re moving forward with. But we would like to continue to show the breadth of the platform by … extending some of the in vitro work that was already [completed by Guo] into an in vivo system.”
Davis declined to comment on the in vivo work that Kylin has already conducted or the specific indications the company is pursuing, but he noted that certain of the data from that work has been submitted for publication.
Gear in the Machine
Kylin’s chief technology is the so-called packaging RNA, or pRNA, which is derived from bacteriophage phi29 and characterized by the company as a “gear in the DNA packaging machinery” that can be modified with therapeutic RNA such as siRNA.
In May 2006, Purdue’s Gou published data describing the construction of folate-conjugated pRNA for the delivery of siRNAs to cancer cells.
And last year, Gou reported that pRNA could be used to escort hammerhead ribozymes into cancer cells.
For now, the company is keeping its eye on cancer, Davis said. He noted, however, that Guo and his colleagues are conducting preliminary work in other disease models. He declined to disclose the nature of the models due to the preliminary status of the work.
“We’d like to explore [those], but we have limited resources,” he added. And while Kylin intends to begin fundraising again sometime in the first half of 2009, any programs outside of cancer would likely require industry collaborators, Davis said.
He said the company does anticipate its technology will be attractive to potential partners, but stressed he wants to have more in vivo data in hand before beginning any negotiations.
With the work already done and the new research underway at Purdue, “I believe that we’re getting close to the appropriate time for pursuing those” negotiations. He said talks could be set up possibly as early as next year, though he did not elaborate.
And though all of Kylin’s work to date has been on using pRNA for therapeutic applications, Davis said that these efforts will also support the company’s bid to realize value from the technology for research purposes.
Since the true potential of pRNA for the research market lies in its use as an in vivo tool, “all the data we’re developing for the human therapeutics is going to be relevant to that group, as well. It doesn’t really change our focus.”