Sernova gets under the skin

The company believes its new combination technology can make insulin injections a thing of the past.

Thanks to the deal it signed last month with Evotec, Sernova will soon have a ready supply of off-the-shelf islet cells to add to its implanted Cell Pouch device. The combination is not yet in formal preclinical trials, but the group believes it can replace its current flagship project, which uses donor-derived islet cells, and go on to be nothing less than a functional cure for type 1 diabetes.

But many cell therapies, for diabetes and beyond, have posted indifferent efficacy or been subject to safety scares. Perhaps this is why Sernova likes to compare its new programme – in terms of scale-up, manufacturing, ease of use and potential market reach – not to a cell therapy or a device, but to a drug. 

Sernova’s Cell Pouch device is about three quarters of the size of a business card and a couple of millimetres thick. It can be rolled up and placed, via a small incision in the abdomen, under a patient’s subcutaneous fat. It then flattens out and, after around three weeks, therapeutic cells are added.

“Then tissue will continue to grow around those cells, blood vessels will come in and feed them, and the cells are sitting in an organ-like environment,” Philip Toleikis, Sernova’s chief executive, tells Evaluate Vantage. The company’s first two diabetes programmes use donor islet cells which can only be obtained post mortem. Naturally the supply of these is somewhat limited – hence the Evotec deal.

Cell mates

“The stem cell programme we’re working on with Evotec allows us to have an unlimited supply of cells,” Mr Toleikis says, which “allows us to treat millions of patients”. That is the theory at least, but the project has a long way to go first. Sernova does not expect to even enter the clinic until early 2024. 

“That might sound like a long time,” Mr Toleikis says, “but it’s a very, very short time in the pharmaceutical world to be able to get this kind of a product into clinic.”

In the meantime, Sernova is pushing ahead with the donor-derived cell products. The first is in a phase 1/2 trial in which each patient will receive at least 3,000 donor cells per kg of patient body weight, with the endpoints covering safety across one year, as well as survival of the cells and blood sugar levels. Judging by its record on clincialtrials.gov, data could emerge this year. 

The major downside of this project – along with the scarcity of donor cells – is that patients must take immunosuppressive drugs. The follow up, in preclinical trials, is designed to dispense with this requirement: it incorporates a polymer coating licensed from the University of Miami which is designed to block rejection by the immune system. The Evotec-partnered project will use this technology too.

Mr Toleikis insists that there is a place on the market for Sernova’s lead combo product, though it would probably only be used for the most severely ill patients, at least at first.

“We have the ability to get a product in the market to help patients with our Cell Pouch. But as we combine the technologies of the local immune protection and the [Evotec] stem cells, which essentially are producing an islet that is fully functional, then that programme, once we get it approved, will essentially take over all of the products.”

Perils

This presupposes a smooth route to market – something that has eluded another group active in the same arena. The phase 1/2 trial of Vertex’s stem cell-derived islet cell replacement therapy VX-880 was placed on hold at the start of May after the FDA concluded that there was insufficient information to support dose escalation with the product.

But VX-880 does not use a device to encapsulate the cells – it is a simple infusion – so Sernova believes it can dodge similar concerns. Vertex also has a device combo, but this remains preclinical two and a half years on from the acquisition of its originator, Semma

Then there are the regulatory requirements. As an innovative implanted medical device, the Cell Pouch will require a PMA in the US, but as cells are present there will also have to be oversight by the agency’s biologics division, CBER. 

As for market size, Mr Toleikis reckons that even the donor-derived products will be widely taken up. Sernova is developing a toolkit to allow surgeons in any country, in any hospital, to implant the device and transplant the cells easily. While this is clearly more complex than giving a drug, Sernova hopes that, in terms of scalability and reliability, it can emulate pharmaceuticals.

“We’re really trying to make this like a pharmaceutical product, and pharmaceutical products can treat millions,” he says. 

The technology is certainly intriguing. But Sernova’s hope that this new technology can work – and sell – as reliably and widely as a drug is an ambitious goal.

Sernova's pipeline
Product Indication Status Partner
Human donor islets (requires systemic immunosuppression) Type 1 diabetes Phase 1/2 -
Micro-encapsulated islets Type 1 diabetes Preclinical University of Miami
iPSC-based beta cells Type 1 diabetes Concept Evotec
Patient cells edited to produce factor VIII
Haemophilia A
Preclinical -
Allograft immune protected cells
Haemophilia A
Concept -
Autograft thyroid cells
Thyroid disease
Preclinical -
All programmes use the Cell Pouch device. iPSC = induced pluripotent stem cells. Source: Evaluate Medtech, company website. 

Share This Article