Vertex looks beyond cystic fibrosis with $950m Semma buy

Vertex Pharma is all-but synonymous with cystic fibrosis (CF) treatment but wants to diversify into new areas, including diabetes with a just-announced acquisition of Semma Therapeutics.

The all-cash, $950 million deal centres on Semma’s stem cell-based therapies that have been billed as potentially a cure for type 1 diabetes, the autoimmune form of the metabolic disease that causes the insulin producing beta cells in the pancreas to be destroyed.

Semma – formed five years ago by Harvard stem cell researcher Douglas Melton – has shown that it can stimulate stem cells to differentiate into beta cells that produce insulin when implanted into animals, including non-human primates.

It’s also developed a delivery system that encapsulates and protects the beta cells from the immune system of the recipient, sidestepping the need for immunosuppressive drugs, and is now in the preparation phase for human trials next year.

Vertex chief executive Jeffrey Leiden (pictured) said the company sees “substantial opportunity” to transform the treatment of type 1 diabetes, which has seen no new treatments since insulin started to be used nearly 100 years ago. He hopes to complete the acquisition by the end of the year.

Semma isn’t the only player in this area, with other developers seemingly further ahead, although data so far is limited.

An encapsulated stem cell therapy developed by Viacyte – called PEC-Encap – has already reached the human testing stage in both the US and Europe, using a subtherapeutic dose initially to guard against side effects, while Canada’s Sernova also has a candidate in clinical trials.

Buying Semma can however be viewed as a platform deal that could in time develop beyond type 1 diabetes into other therapeutic categories that might respond to generative stem cell treatment.

Vertex’ primary focus on CF has resulted in three marketed products that have transformed care of people with the genetic disorder and brought in blockbuster sales for the company but have also attracted no little controversy over pricing.

The deal with Semma is just the latest in a series penned by the company to try to diversify into other areas, and to avoid a repeat of what happened with its lucrative hepatitis C franchise a few years back.

Its blockbuster product Incivek (telaprevir) was effectively dismantled within a few months after newer, more effective antiviral drugs from Gilead reached the market.

Earlier this year, Vertex paid $70 million to form a joint venture with protein degradation specialist Kymera Therapeutics to develop drugs for rare diseases, with another $1 billion in milestones potentially in the offing.

It also recently teamed up with CRISPR Therapeutics on a gene-editing treatment for sickle cell disease and beta thalassaemia, two rare blood disorders, and another gene-editing deal with the buyout of Exonics, which focuses on diseases like Duchenne muscular dystrophy.

Leiden is due to step down as CEO of the company in the coming months, but has previously said Vertex will continue to seek out bolt-on deals while bringing forward in-house projects in areas like alpha-1 antitrypsin (AAT) deficiency.