Just a few weeks after completing the treatment portion of a Phase II/III trial of Fibrillex, Neurochem Inc. partnered its AA amyloidosis drug with Centocor Inc. in a deal worth up to $54 million.
Laval, Quebec-based Neurochem, through its wholly owned subsidiary, Neurochem Ltd., of Ecublens, Switzerland, signed the collaboration and distribution agreement with Malvern, Pa.-based Centocor, which is a wholly owned subsidiary of Johnson & Johnson.
"This deal has both short-term and long-term benefits," said Lise Hébert, Neurochem's vice president of corporate communications. "It really positions the program well with the key physicians that we need to reach. This is a worldwide agreement. It provides for Neurochem very good advantages, and for the patients, ultimately, we feel this partnership should accelerate their access to Fibrillex."
Centocor gains exclusive distribution rights for Fibrillex to prevent and treat AA amyloidosis. The rights are worldwide, with the exception of Canada, Switzerland, China, Japan, Taiwan and South Korea, where Neurochem continues to hold rights.
The potential $54 million consists of an up-front payment and regulatory and sales-based milestone payments. Neurochem also could receive an escalating distribution fee based on annual sales of Fibrillex over the life of the agreement.
"It's a royalty, plus supply price, plus overhead," explained Christine Lennon, the company's vice president of business development. "The distribution fee is a percentage on sales, so it's a tiered distribution rate. It's at least as good as any royalty rate."
Hébert and Lennon would not disclose more specific financial terms.
While Centocor and its affiliates will manage the marketing and sales of Fibrillex and be responsible for worldwide safety surveillance, Neurochem will take care of product approvals in the U.S. and the European Union and global manufacturing activities.
The company intends to file a new drug application in the U.S. sometime in 2005, following the release of Fibrillex's Phase II/III data in the second quarter.
The fact that Centocor has experience marketing its rheumatoid arthritis drug, Remicade (infliximab), was attractive to Neurochem. Arthritis is the most common underlying condition that leads to AA amyloidosis, so rheumatologists often are the doctors providing care for those patients. And Centocor's affiliates market to nephrologists, other specialists that encounter patients with AA amyloidosis.
"The breadth of the commercialization reach is really Centocor and its affiliates through the J&J group," Lennon told BioWorld Today.
The term of the agreement is on a country-by-country basis. Centocor will hold the rights for the longer of 10 years or the end of legal exclusivity, orphan drug exclusivity or the patent term.
Lennon said Neurochem will seek another partner for Asia, but "for Canada and Switzerland, it is our intent to bring the product forward ourselves."
The most frequent underlying diseases in patients enrolled in the Phase II/III trial of Fibrillex were rheumatoid arthritis and familial Mediterranean fever, and the mean time from onset of underlying disease until AA amyloidosis was 14.6 years. Nephrotic syndrome was present in 32 percent of the participating patients.
The trial was a two-year, randomized, double-blind and placebo-controlled study in patients suffering from AA amyloidosis confirmed by biopsy and renal involvement. It enrolled 183 patients at 27 sites across North America, Europe, North Africa and Israel.
In early December, Neurochem completed the treatment phase of the trial, and it expects to present detailed results in the second quarter.
AA amyloidosis affects about 270,000 people worldwide and occurs in patients with chronic inflammatory diseases, such as rheumatoid arthritis, ankylosing spondylitis, juvenile rheumatoid arthritis and Crohn's disease. Symptoms include renal dysfunction and gastrointestinal problems, such as diarrhea, abdominal pain and malabsorption, as well as enlargement of the liver and spleen. If left untreated, it leads to organ failure.
"Once the AA amyloidosis has developed, the survival time is very severe," Hébert said. "Fifty percent of these patients will, unfortunately, die of this condition within five years."
There is no specific treatment available for the disease. As a result, the oral drug Fibrillex (1,3-propanedisulfonate) has orphan drug status in the U.S. and Europe. It also has been selected by the Cardio-Renal Drug Product Division of the FDA to be part of the Continuous Marketing Applications Pilot 2 program, which is aimed at accelerating the development and marketing of the product. Fibrillex prevents amyloid fibril formation and belongs to the class of glycosaminoglycan mimetics.
Aside from Fibrillex, Neurochem is focused on Alzhemed (3-amino-1-propanesulfonic acid), which entered a Phase III trial in June in Alzheimer's disease. (See BioWorld Today, June 22, 2004.)
Neurochem also is studying Cerebril to prevent hemorrhagic stroke caused by cerebral amyloid angiopathy. That product has completed a Phase II trial.
Neurochem's stock (NASDAQ:NRMX) rose 37 cents Wednesday to close at $17.40.