Diamyd Medical has confirmed terminating its Phase III program for the diabetes therapy Diamyd®, and will instead focus all its resources on its clinical-stage pain candidates and CNS drug compounds. Formal discontinuation of the diabetes program, which recently failed in both U.S. and European Phase III studies, means the majority of the firm’s employees in Sweden will lose their jobs, as most were working on the Diamyd studies and in related areas.
Diamyd Medical has now shifted its priorities to its chronic pain portfolio, which is based on a platform known as nerve targeting drug delivery system (NTDDS), designed to deliver gene-based drugs directly to nerve cells. The firm says in addition to its use in pain relief, the technology has potential applications in the treatment and prevention of nervous system disorders such as neuropathy, erectile dysfunction, neurodegenerative diseases, and for the treatment of cancers such as glioma, by carrying cytotoxic drugs directly to the tumor.
Lead NTDDS candidate, NP2 Enkephalin, is in Phase II trials for the treatment of severe cancer pain, and results are scheduled around the end of the year. A second candidate, NG2 GAD, for indications including diabetes pain, is being prepared for clinical development. NTDDS-related R&D is being carried out primarily at Diamyd Medical’s U.S. subsidiary in Pittsburgh.
Gene-based NTDDS are designed to be injected into the skin over the area of pain. The drug is then transported along local peripheral nerve fibers to the spinal cord, where it acts to stop the transmission of pain signals from the peripheral nerves to the nerves of the spinal cord, Diamyd Medical explains. As a gene-based approach, NTDDS harnesses the nerve cell's production machinery to continually deliver the painkilling substance locally on site at the spinal cord.
The firm maintains the platform has a range of advantages over other pain relief strategies. NTDDS treatment is nerve-specific and acts locally, which reduces the risk of systemic side effects. The NTDDS drugs are also not integrated into host cell chromosomes and so don’t induce an immune reaction, again reducing the risk of side effects compared with other gene therapies.
The Diamyd program accounted for about two-thirds of Diamyd Medical’s costs, and as a result of its discontinuation, the firm projects it will have about SEK 400 million in liquid assets at the end of the calendar year. “Having plenty of cash on hand gives us valuable strategic leeway, not least when we get the results from the Phase II study with NP2 Enkephalin,” remarks Peter Zerhouni, president and CEO. “The shift of the company’s primary development focus to the unique NTDDS technology gives us a fresh start before fall.”
The GAD65 technology on which the Diamyd therapy is based has not been abandoned, the firm adds. Researchers are trying to work out why the Diamyd trials failed, and what can be learned to guide future development of a GAD65-based diabetes therapy. One potential is to start therapy before the onset of disease. To this end, an externally funded and researcher-initiated Phase II study with Diamyd has been ongoing since 2008 to evaluate use of the treatment in preventing the development of type 1 diabetes in high-risk children.