BioWorld International Correspondent

Diamyd Medical AB is on track to begin a Phase III clinical trial of its therapeutic vaccine for Type I diabetes this spring. The Stockholm, Sweden-based firm said it replied to the sole question it received from the FDA on its IND filing, which it had completed in December.

The FDA's queried a minor aspect of the manufacturing process for the vaccine, which is also called Diamyd. "It's a very small formality, really," Diamyd CEO Elisabeth Lindner told BioWorld International. "One analytical value we had not given, so we had to provide that value, that's all," she said. The company expects to get a green light from the FDA within 30 days and to begin the 300-patient study shortly afterward. "That's our expectation definitely," she said. The trial will last for 15 months, with a follow-up period of another 15 months.

The company licensed rights to the vaccine from inventors Allan Tobin and Daniel Kaufman at the University of California, Los Angeles. It comprises an alum-adjuvanted formulation of a recombinant form of the enzyme glutamate decarboxylase (GAD-65), a major auto-antigen in Type I diabetes. It is designed to elicit immunological tolerance in patients and slow the autoimmune destruction of insulin-secreting beta cells in the pancreas.

"It brings to the market the possibility for intervention in diabetes. You can protect the remaining beta cells," Lindner said.

Diamyd has two manufacturing partners involved in the project. Protein Sciences Corp., of Meriden, Conn. - in which Diamyd holds a 6.7 percent stake - is producing the active drug substance, using its Baculovirus-based protein expression system. The protein is produced in a cell line derived from the insect species Spodoptera frugiperda, which, said Lindner, provides high levels of productivity without the complexity of mammalian production systems. The insect background does not represent any potential problems, she said. "The functionality of this protein is not dependent on the glycosylation." Octoplus Technologies NV, of Leiden, the Netherlands, is carrying out the formulation of Diamyd and fill/finish.

In parallel with the Phase III study in the U.S., the company is commencing a similar trial in Europe, for which it filed an initial application with the Swedish Medical Products Agency in Uppsala last month. In addition, the National Institute of Diabetes and Digestive and Kidney Diseases is sponsoring a 126-patient Phase IIb clinical trial of the vaccine in order to examine whether it can help preserve the endogenous insulin production in recently diagnosed Type I diabetes patients.

Diamyd, which currently is valued at about SEK771 million (US$120 million), reported a cash balance of SEK49.8 million Nov. 30. But raising additional cash to finance its clinical programs is not the immediate priority. "We have communicated to the market that we are looking for partnerships," said Lindner, who joined the company in December. "I think it could be this year."