NEW YORK - Record-setting snowfalls from the weekend blizzard grounded transportation throughout the Northeast, and snow that topped 27 inches in Manhattan delayed the Westminster Kennel Club show here, with leading dogs unable to get to Madison Square Garden.
But it did little to slow down the Biotechnology Industry Organization’s annual CEO & Investor Conference, and by Tuesday, conference attendees filtered into the stately Waldorf Astoria Hotel, for two days of panel discussions and company pitches.
Inhibitex Inc. took the opportunity to reiterate its pricing strategy for the pediatric anti-infective drug Veronate, for which the company on Tuesday revealed it had begun a rolling biologics license application in hospital infections in highly susceptible low-birth-weight babies. In recent months, Inhibitex President and CEO William Johnston has told The Street that the drug likely would cost at least $6,000 per course of treatment, a figure he cited again at the conference. On a yearly basis, that could generate at least between $200 million and $300 million in the U.S.
Projecting gross margins that could range between 80 percent and 90 percent, Johnston added that Veronate "is going to be a very attractive product."
Top-line pivotal data from a Phase III trial are due in two months, and Inhibitex expects to complete its BLA by the end of the third quarter or early in the fourth. Pending priority review from the FDA and subsequent approval, the product could be launched late next year. Data ultimately will drive the cost of Veronate, though, an antibody-based drug that has fast-track and orphan drug status in the U.S.
Enrollment in the Phase III study is complete, totaling 2,018 infants who weigh between 500 grams and 1,250 grams.
"The smaller the patient, the greater the risk," Johnston said. That group represents an annual patient population of 43,000 babies in the U.S., said Joseph Patti, Inhibitex’s vice president of research and development and its chief scientific officer.
It is estimated that 300,000 U.S. babies every year fall into the low-birth-rate category, defined as smaller in size than 2,500 grams. They have higher chances of mental development problems, cerebral palsy, blindness and hearing loss.
The Phase III babies are treated with up to four 750-mg doses of Veronate over 15 days, a shorter duration than Phase II, but otherwise the two trials have followed "very parallel designs," Johnston said. That previous study, which showed a clear dose-response relationship, demonstrated a 63 percent reduction in the incidence of Staphylococcus aureus infections among infants treated with the drug, as well as a 67 percent reduction in Candida species. Those types of infections extend by 25 days low-birth-weight infants’ time in neonatal intensive care units.
"We’re giving antibodies to kids who don’t have antibodies," said Johnston. The trial’s primary endpoint is the reduction in S. aureus, and secondary endpoints include reductions in Candida species, Coagulase-negative staphylococci and all-cause mortality. Success in that latter measure also could dictate a larger market opportunity.
Notably, the FDA has indicated that primary endpoint success in an undefined subset of patients, as well as positive trends in the overall Phase III patient population, also could support approval.
That determination "shocked" company officials, Johnston said, but he assumed the FDA might have based its decision on outside pressure to get new drugs to pediatric patients.
Inhibitex, of Alpharetta, Ga., has submitted the chemistry, manufacturing and controls section of the BLA, and plans to file the preclinical section in April.
If approved, the company plans to build its own "relatively small sales force" of 40 to 50 representatives to visit neonatologists in the U.S., Johnston said. Inhibitex holds commercialization rights for the rest of the world and any overseas partnering plans will be "data driven," he added. In Europe, Veronate has orphan drug status.
Deeper in the company’s pipeline is Aurexis, a Phase II product being developed for adjunctive use with antibiotics for S. aureus. Also in the portfolio are earlier-stage monoclonal antibodies and a vaccine relationship with Wyeth, of Madison, N.J. With cash reserves of $93 million, Johnston said Inhibitex is "well capitalized to keep moving."
On Tuesday, its shares (NASDAQ:INHX) gained 17 cents to close at $8.60.