With a fix for the longstanding problem of non-compliance in dosing exenatide to type 2 diabetes patients, Intarcia Pharmaceuticals Inc. reaped a potential $1 billion partner in Les Laboratoires Servier for ITCA 650, a once-per-year pump that can be implanted in the doctor's office.
"They have every territory outside the U.S. and Japan," said Kurt Graves, CEO of Boston-based Intarcia, which stands to collect as much as $401 million in near-term payments, including $171 million up front and three early stage regulatory milestones totaling $230 million, followed by as much as $650 million if the compound reaches development, regulatory and sales goals.
"'Near-term' means over the next two or two and a half years, based on finishing some of our studies, filing the product and getting it approved," Graves told BioWorld Today. Servier will handle regulatory matters outside the U.S. and Japan, with support from Intarcia, which upon commercial success will get tiered net sales-related payments ranging from the low double digits up to the mid-30s as a percentage of sales of product made available through the manufacturing and supply agreement.
Phase III trials are still under way and on track to support filings around the world in the first half of 2016. "This deal really puts us in a position to maintain our freedom and independence, and continue building a disruptive biotech company that's going to be focused on the U.S. first," Graves said, adding that the firm will seek another partner in Japan. "We haven't communicated a timeline around that. We just closed this one, so I'm going to take a little bit of a breather."
Under the terms, Intarcia and Servier, of Neuilly-sur-Seine, France, will share future global development-related investments for the life-cycle management of ITCA 650, including head-to-head superiority studies against other diabetes drugs and new combination regimens.
Bydureon, from Amylin Pharmaceuticals Inc., of San Diego, exenatide given by weekly injections. With ITCA 650, the glucagon-like peptide-1 (GLP-1) agonist is given once or twice yearly in a mini-pump about the size of a matchstick that can be placed subdermally into the patient's abdomen by a doctor, nurse or physician's assistant.
"We're providing the marketplace the flexibility of choice," Graves said. The three-month pump, "kind of a titration step," gives a lower dose of exenatide, when is then upped to 60 mcg per day a dose that patients can maintain for six months or 12 months. "A lot of people are going to end up on once-a-year pumps," he said. "That's what locks in control, compliance and adherence. It's a little miracle."
The pump is designed for "zero-order delivery: a smooth consistent delivery of the medicine 24/7, 365 days a year," Graves said. As phase III trials are testing ITCA 650 in humans, "we also developed an in vitro system where thousands of our pumps have been tested in body-like conditions, submerged in fluid at body temperature," he said. "In every manufacturing lot we make, some of those pumps go into our in vitro system so that we can validate that the pumps work flawlessly. We've never had one fail."
NEXT: OBESITY PEPTIDES-PLUS
Titanium was used to make the pump because "we wanted to have a material that, if by some chance, someone forgot to take it out, or moved to another country and couldn't get it out, the material we have is the same that is used for knees, hips, shoulders, [with] no safety consequences long term if, for whatever reason, a handful of people had these never taken out," Graves said. "We also wanted to have the mini-pump be virtually indestructible. You don't want something delivering a medicine that could be bent or cracked. I've never seen one of these be bent or cracked, and a lot of people have tried."
Graves predicted eager adoption if the ITCA 650 is approved. "I'm sure there will be some people who may not want to have a mini-pump in them," he said, but many diabetics come around "when you show them the HbA1c reductions we've seen, the weight loss we've seen, and the fact they can do something once a year and avoid lifelong injections."
Behind ITCA 650, Intarcia has ITCA 880 and ITCA 884 that work similarly in obesity to deliver weight-regulating endocrine peptides that work in synergy with the GLP-1 drug.
"If chapter one for the company is a game-changing drug for diabetes, chapter two is hopefully going to be a game-changing drug that works for obesity and diabetes together," Graves said. "What we're working on is the combination of gut peptides known not to just cause HbA1c reductions but pretty profound weight loss. Of the ones that appear to work the [best], we're taking the multiple-shot-on-goal approach" and could, if early data hold true, reduce weight by 20-plus percent, he said.
"From a technical point of view, we know we can do it we've done it," Graves said. "This is really the next level. We're going to be running multiple studies in humans to figure out the best combinations of these peptides and the best dosages. We've found a few things that we're even more excited about" that are yet to be made public, he added.
Earlier this year, Intarcia reaped a $200 million financing that included $57 million from the Foresite Capital Fund II, a growth equity stash that jumped aboard the Intarcia bandwagon after getting the January peek at interim phase III data. Graves called the Servier agreement "one of the largest ex-U.S. partnering deals ever in the biotechnology industry." Another bolus of encouraging phase III results from the FREEDOM trials rolled out about a month ago, and Intarcia predicted a partnership would turn up in the second half of this year. (See BioWorld Today, April 2, 2014.)