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Historically, there has never even been a glimmer of hope for stopping the autoimmune condition type 1 diabetes (T1D) in its tracks. But now, Swedish biotech company Diamyd Medical is moving forward with large-scale clinical studies of a vaccine that works to “reprogram” immune cells to prevent the destruction of insulin-producing cells in the pancreas.

Diamyd has been working on this complex immunotherapy vaccine for two decades. Despite some previous bumps in the road and delays, the latest studies showed promising results.

Their large-scale Phase III trials are set to kick off during 2021, at 50 sites across Europe and the United States. The study will include roughly 330 children and young adults (ages 12 to 28), recently diagnosed with T1D.

Notably, this will be the first approach of its kind based on precision medicine, meaning the therapy is personalized to individuals’ specific physiology.

“We have put great effort into designing this latest trial together with our collaboration partners, digging deep into the data to make sure we do not cut any corners,” Diamyd CEO Ulf Hannelius told DiabetesMine. “Without all the data and the knowledge of how to use it, we wouldn’t be where we are today.”

The Diamyd vaccine aims to stop the destruction of insulin-producing beta cells that lead to T1D.

The active ingredient in the vaccine is GAD65 (glutamic acid decarboxylase-65), an enzyme that occurs naturally in the pancreatic beta cells that helps them work properly and continue producing insulin. A majority of people with T1D have GAD autoantibodies that target this enzyme, leading the immune system to attack the cells that make insulin, shutting off insulin production.

Diamyd’s vaccine supplements the GAD65 enzyme, aiming to stop this destructive process. It could thwart or delay the onset of T1D by helping the beta cells continue to produce insulin.

In earlier clinical trials, the Diamyd vaccine was injected directly into the lymph nodes of children and young adults (ages 12 to 24) who had been diagnosed with T1D within the past 6 months. They received three or four injections over the course of 15 months. Results showed a “significant effect on C-peptide retention,” meaning it preserved or improved insulin secretion in the body.

In the upcoming Phase III trials, subjects will be randomized to receive either three injections of the Diamyd vaccine or three injections of a placebo one month apart. The outcomes will be measured after 24 months. Based on efficacy data from previous trials, the company is confident that C-peptide levels will be preserved, and participants will see lower A1C results (indicating improved blood sugar levels).

No, it wouldn’t. But it’s a start to learning more about what leads to T1D and how to delay it, and could hold major clues to preventing the autoimmune condition from developing down the road, Hannelius says.

Right now, Diamyd is focusing its research on young people who are newly diagnosed with T1D within the past 6 months.

But Diamyd is also working on a vaccine tailored specifically to individuals with latent autoimmune diabetes in adults (LADA), as well as an oral tablet called Remygen that could stimulate the growth and function of insulin-producing beta cells in both autoimmune T1D and in type 2 diabetes.

Precision medicine is a newer approach for disease treatment and prevention that focuses on people’s genes, environment, or lifestyle in order to tailor their treatment to them individually.

Ulf Hannelius

In Diamyd’s case, precision medicine allows them to hone in on newly-diagnosed T1Ds who have a very specific gene type that’s been proven to be more responsive to this particular treatment. It’s called the HLA DR3-DQ2 haplotype, which researchers note has a central role in immunity. Diamyd’s Hannelius says their research shows it significantly influences the effect of the vaccine.

For the clinical studies, Diamyd is able to use blood tests to detect this particular haplotype, to determine if someone is eligible for this precision medicine vaccine.

“This is the definition of precision medicine, bringing the right treatment for the right person at the right time,” Hannelius says. “I believe this will be driving the future of pharmaceutical development.”

As noted, Diamyd has been on the diabetes research scene for more than two decades, and this line of GAD-specific research goes back to the early 2000s.

In 2011-12, there were headlines about disappointing clinical trial results that led to investors fleeing — including Johnson and Johnson, which had once backed the vaccine development. But for the past several years, Diamyd has been quietly plugging ahead on its research, with results continuing to look more and more promising.

Hannelius became CEO in 2016. And when Medtronic acquired smart insulin pen startup Companion Medical in August 2020, Diamyd — as a previous shareholder — benefited with a multi-million dollar payout.

The name Diamyd actually has its roots in a personal connection to diabetes. It’s a mashup of “Diabetes My GAD” — with “My” being the name of the youngest daughter of the company founder, Anders Essen-Möller, who lives with T1D. Her diagnosis was a pivotal event that led this biomedical engineer to form the company and start work on a potential vaccine.

“The fact that we are now ready to go into Phase III development with a precision medicine approach is very exciting and a fantastic achievement by the team,” CEO Hannelius said. “It is very exciting to see that we are growing as a company and investing into our own pharmaceutical manufacturing. There is so much competence in the company. I am both proud and amazed by the enthusiasm and how much the team has achieved to advance our programs.”