A group of Alzheimer’s disease scientists from the Roskamp Institute in Sarasota, Fla., including several responsible for discovering the role of beta-amyloid accumulation in the disease, this week announced the launch of Archer Pharmaceuticals to commercialize therapeutics for Alzheimer’s disease based on discoveries made at the institute.
Archer is the first spinout for the Roskamp Institute, which prior to 2003 was affiliated with the University of South Florida, but has since operated as an independent non-profit research institute supported by the Roskamp Foundation, a trust founded by philanthropists Robert and Diane Roskamp.
The new company’s lead drug candidates are also primarily a product of research that was supported by the Roskamp Foundation and by private investors through a for-profit entity called Roskamp Research that was also set up in 2003 to manage the commercialization of certain pieces of intellectual property developed at the institute.
Michael Mullan, CEO of Archer and director of the Roskamp Institute, told BTW this week that Roskamp Research will now be dissolved, and its assets — essentially a portfolio of more than a dozen patents and applications — will be transferred to Archer.
As such, both private investors and the Roskamp Institute will also receive an undisclosed equity stake in Archer, and stand to benefit financially if the company can successfully commercialize its Alzheimer’s treatment, Mullan said.
The Roskamp Foundation originally founded the Roskamp Institute in 1998 at USF with a gift of $5 million. Mullan, who was part of a team of researchers at USF who in the early 1990s discovered the link between beta-amyloid accumulation and Alzheimer’s development, headed the institute since its inception.
However, Mullan left USF in 2003 for personal reasons, and many of the scientists from the institute, which was affiliated with USF but owned and operated by the Roskamp Foundation, followed him, setting up shop in nearby Sarasota as an independent, non-profit research institute. This group included Fiona Crawford, who currently serves as associate director of the institute and chief technical officer at Archer.
When the institute started anew in 2003, it set up Roskamp Research with “the idea that there was the potential [to commercialize] some of this work,” Mullan told BTW.
“I don’t think all the details were in place yet,” Mullan said. “The institute was really set up to pursue several academic questions in relation to Alzheimer’s. The for-profit entity was set up at the same time with the idea that IP could be generated from the [research].”
He estimated that undisclosed individual investors together with the Roskamp Foundation invested between $6 million and $10 million in Roskamp Research, which then disbursed the funds to support particularly promising work that the institute was studying, particularly developing compounds that could clear beta-amyloid from the brain.
“The benefit of doing it the way we’ve done it is that the for-profit has leaned heavily on the resources and infrastructure of the non-profit from day one.”
“The institute received funds generated on a contract basis in the same way that we get funds from other entities,” Mullan said. In particular, the private funding served as a sort of gap funding to help expand basic IP or create new IP around the researchers’ discoveries.
“The benefit of doing it the way we’ve done it is that the for-profit has leaned heavily on the resources and infrastructure of the non-profit from day one,” Mullan said. “Of course, Archer makes the chemicals that we’ll take to the clinical trials, and manufactures and formulates the drugs, which is very different from anything Roskamp does. But a lot of the initial infrastructure was provided by the institute, and I think that has really helped Archer on its way.”
Archer now has several compounds that are ready to enter clinical development, including lead candidates ARC029 and ARC031, both of which are intended to reduce soluble amyloid proteins in the brain. In addition, Archer has several other agents in the pre-clinical stage, including gamma secretase inhibitors and BACE inhibitors, which may complement the lead compounds by attacking amyloid in a different way.
The company said that it selected ARC029 as its first-line treatment from approximately 2,000 agents because it was one of the most proficient at lowering amyloid levels in preclinical models through largely unexplored biological mechanisms.
“The two compounds that we’re advancing into the clinic have a unique mode of action that hasn’t been seen or heard of before,” Mullan told BTW this week. “They clear amyloid from the brain, across the blood-brain barrier, and into the blood.”
A phase I/IIA clinical trial currently underway in Europe was designed to evaluate the safety of nilvadipine, the active ingredient of ARC029, in Alzheimer’s patients. The study has shown that the drug was well tolerated in Alzheimer’s patients, to the extent that “we certainly feel comfortable enough to take it to US-based clinical trials,” Mullan said.
Archer hopes to go ahead with US clinical trials within the next few months, and intends to fast-track ARC029 and ARC031, while continuing to work on other therapeutic approaches. It is seeking additional private investment to the tune of $25 million to $30 million to support its endeavor.
“We anticipate two stages,” Mullan said. “Stage one will probably cost about $2 million over the next year, and then the rest will be in the subsequent two years, and cost about $20 million or so.” Mullan said that the company is exploring all fundraising avenues, including investment by private individuals and venture capital firms.
Archer is also exploring the idea of partnering with larger pharmaceutical companies to move the drug candidates through clinical trials, Mullan said.
One unlikely partner for Archer moving forward will be the Roskamp Institute.
“There will be pretty strict lines, as a matter of fact, on a number of parameters between the institute and [Archer],” Mullan said. “Right now the connection is that Archer is [renting] space from the institute. But it’s run as a completely separate entity. The benefit to the institute in the long run is that it has a shareholder position in Archer.”
He added that in general, scientists involved with the drug’s development now typically either work for the institute or for Archer, but not both. “Some people have appointments at both, but they’re not working at the institute and then leaning across to Archer,” he said. “It’s very important to separate the two entities, I think.”
The major exceptions, of course, are Mullan and Crawford, both of whom maintain dual roles with the institute and the company.
“At this point, which is probably a transitional point, we both have roles with both entities,” Mullan said. “We’ll see how long that lasts. I suspect that at some point we’ll have to make the decision to either hire management to replace us at Archer; or else we’ll make a transition to Archer completely. It’s not clear completely at this point, but those options are both out there.”