When invited to the University of Chicago for a day three years ago as a visiting professor, Rifat Pamukcu didn't expect to come away with intellectual property that would form the basis of his new company, Midway Pharmaceuticals Inc.
At the time of his visit to Chicago, Pamukcu was the chief scientific officer of Horsham, Pa.-based Cell Pathways Inc., a company he co-founded, which was about to be acquired by OSI Pharmaceuticals Inc., of Melville, N.Y., in a stock deal valued at $32 million. (See BioWorld Today, Feb. 11, 2003.)
"I was out there that day and my scientific mentor, Gene Chang, pulled me aside and said, 'You know, we've got some technology we'd like you to look at,'" Pamukcu said.
The technology centered around the relationship between the gastrointestinal (GI) tract and GI bacteria in the emergence and recurrence of GI diseases, such as inflammatory bowel disease, and the potential use of polymer-based therapeutics to prevent pathogenic activity.
"There's a lot of data coming out recently," Pamukcu said, suggesting a connection between bacteria "in the gut and the triggering of flares in Crohn's disease and ulcerative colitis."
Those types of diseases have long been attacked "from an immunological point of view," he added, "with the Remicades of the world, corticoid steroids," and other therapies designed to suppress the immune system.
Remicade (infliximab), a monoclonal antibody that blocks tumor necrosis factor-alpha, was approved in 1998 to treat Crohn's disease. It was developed by Malvern, Pa.-based Centocor Inc.
The IP from the University of Chicago included a lead product candidate, now designated MDY-1001, designed to augment those existing immunological therapies to "see if we can prevent the flares from recurring after they've been cooled off with something else," Pamukcu said.
Midway is hoping to move the MDY-1001 polymer into the clinic within the next year to 18 months, targeting inflammatory bowel diseases, such as Crohn's. Beyond that, the IP provides "plenty of potential" for discovering additional molecules and second-generation polymers, he said.
"For a start-up, you'd always like to be in range of the clinic," Pamukcu said. The fact that the IP consisted of both an identified compound and a research platform "was a winning combination, in my mind."
With the university's support, Pamukcu was able to establish Midway last year. The company is based in Spring House, Pa.
It has brought in a small amount of seed funding to conduct ongoing animal safety work, as well as additional efficacy trials in inflammatory bowel disease models.
Last month, the firm received $500,000 from BioAdvance, the Biotechnology Greenhouse of Southeastern Pennsylvania. BioAdvance invests in health and life sciences industries, using the state's share of the nationwide tobacco settlement.
"These sorts of funds play an absolutely critical role to help start-ups get off the ground before they hit the end of the runway," Pamukcu said. "Seed money is incredibly difficult to come by these days."
For now the company is staffed by Pamukcu, with support from three founding scientists. Most of the laboratory, animal safety and toxicology work is subcontracted out, though some research still is being conducted at the University of Chicago.
Midway will have to secure its first venture round before moving MDY-1001 in Phase I and Phase II trials, adding additional employees and "building more of an infrastructure," he said.
"And we will need a research team when we begin delving deeper into the research pipeline to identify new molecules" aimed at indications such as other bacteria-derived infections, Pamukcu added.
The company also expects to investigate the MDY-1001 polymer as a potential delivery system. Some in vitro results have suggested that the polymer's ability to attach to the gut wall would make it an ideal tool for locally delivering other molecules.
"After taking it forward on its own, there's likely to be good scenarios where we could combine [MDY-1001] with either existing drugs or new agents," Pamukcu said.
Additional early studies also pointed to the ability of the polymer to prevent bacteria from becoming virulent and infectious in patients who are immunocompromised or who have had bone marrow transplants.
"There's much more of a molecular phenomenon here that just the polymer acting as a barrier and Saran Wrapping the gut," Pamukcu said.
"For many years, polymers were looked at as being pretty inert," he added. "Now, to see how they're behaving like drugs is exciting."
The potential for polymer-based therapeutics already has been seen at Genzyme Corp., which has Renagel (sevelamer hydrochloride), a phosphate binder for end-stage renal disease. The Cambridge, Mass.-based firm recently began a pivotal Phase III trial for the second product to emerge from its polymer platform: tolevamer, a non-absorbed polymer therapeutic for Clostridium difficile-associated diarrhea. If approved, tolevamer would be the first non-antibiotic to treat that condition.
Once Midway moves MDY-1001 into Phase II, it would begin looking for a development and commercialization partner to fund the larger trials and provide a sales force.
"I grew up in an era when companies could aspire to be the next Amgen," Pamukcu said, "but capital markets no longer allow for that sort of thing. Our goal here is to become the pipeline for those larger firms."