Some bad news from Cambridge-based Tolerx Inc.: The biotechnology company and a partner, GlaxoSmithKline PLC, report their experimental drug for Type 1 diabetes failed to meet the main clinical goal of a late-stage study.
The 272-patient study measured how well newly diagnosed adults were able to improve production of insulin a year after an eight-day course of the treatment, a monoclonal antibody called otelixizumab. The goal was for patients to show a change in a measure of insulin production called C-peptide.
The companies are reviewing the results but have suspended recruitment and dosing of a separate late-stage trial of the treatment.
Otelixizumab, the most advanced drug in Tolerx’s pipeline, is supposed to protect insulin-producing cells in the pancreas from immune system attacks, which hamper the body’s ability to control blood sugar levels.
In 2007, the company and Glaxo allied to develop otelixizumab for Type 1 diabetes and other diseases. Tolerx has no approved drugs on the market but has several in its pipeline for cancer and other diseases in which the immune system plays a role.
Forma Therapeutics Inc. could choose to do a lot of things after spending its first couple of years raising $50 million, forming partnerships, and assembling a staff of 100 people with all kinds of skills in the cancer drug R&D business.
But Cambridge-based Forma has decided to place one of its early bets on a new drug that interferes with the overactive metabolism of cancer cells — a fancy way of saying it wants to kill tumors by starving them.
“This is a hot area,’’ says Forma’s cofounder and chief executive, Steven Tregay.
Forma isn’t disclosing much yet, though Tregay mentioned the program in interviews in January. It’s the first hint of where Forma intends to go as a drug developer. The company spent its first two years building an unusually potent research and development group, when most venture-backed biotechnology companies were pinching pennies.
The company was founded in January 2009 by scientists from the Broad Institute of MIT and Harvard. Forma has had a whirlwind ride since, clinching partnerships with Cubist Pharmaceuticals Inc., the Experimental Therapeutics Centre of Singapore, and the Leukemia & Lymphoma Society.
Forma has been busy building a pipeline of drug candidates, Tregay says.
It has bucked the trend toward outsourcing and “virtual’’ drug development to build a group of people with lots of the same skills Big Pharma companies have. This focused effort has resulted in a richer pipeline than at a typical biotech company, which often concentrates on a single lead program, or maybe two.
“With these core capabilities, you can be very efficient,’’ Tregay says. “The kind of productivity we’re seeing is not something you can outsource and manage effectively.’’
The most exciting program so far is in cancer metabolism, Tregay says. He is not disclosing the precise molecular target the company is going after. But he said an early drug candidate against this target was able to shrink tumors in mice for as long as 30 days, even when given in tiny doses (10 to 15 milligrams per kilogram of body weight) for about five days.
“This is an incredibly potent compound,’’ Tregay says. “We are seeing really dramatic efficacy. You rarely see things like that.’’ It’s unlikely Forma will take this lead drug program into clinical trials this year, but “we might be close.’’
How was Forma able to plow ahead so aggressively? In 2009, there were lots of talented people, lab space, and cheap equipment available as many other biotech companies cut back. By raising money early and building a network of partnerships, Forma was able to grow. A big part of the strategy was convincing the staff a recession was a great time to step on the gas.
Six patients with the rare genetic disorder Fabry disease have sued Cambridge-based Genzyme Corp. and the Mount Sinai School of Medicine over a rationing system for Genzyme’s Fabry treatment, agalsidase beta (Fabrazyme), the online publication Pharmalot reported. Genzyme, which is being bought by the French drug giant Sanofi-Aventis, started rationing its Fabry therapy after a temporary closing in 2009 of its Allston plant where the treatment is made. Mount Sinai, of New York, licensed the treatment to Genzyme and “went along with the rationing plan,’’ Pharmalot wrote.
This report was compiled by the editors of Xconomy, an online news website focused on the business of technology. For more New England coverage, visit www.Xconomy.com/boston.