Vertex gains FDA fast track designation for diabetes stem cell therapy

The US Food and Drug Administration (FDA) has granted Vertex’s investigational stem cell therapy VX-880 a fast track designation for type 1 diabetes (T1D).

Vertex has announced that it has initiated a phase 1/2 clinical trial for VX-880 in T1D patients with impaired hypoglycemic awareness and severe hypoglycemia.

The single-arm, open-label study will evaluate the safety and efficacy of different doses of VX-880, with approximately 17 patients to be enrolled in the trial.

Vertex gained VX-880 from its previous acquisition of Semma Therapeutics for $950m in September 2019.

VX-880 is a first-in-class, allogeneic human stem-cell derived islet cell therapy, which has the potential to restore the body’s ability to regulate glucose levels by restoring pancreatic islet cell function, including insulin production, according to Vertex.

The phase 1/2 clinical trial will evaluate an infusion of ‘fully differentiated’ functional islet cells along with the chronic administration of concomitant immunosuppressive therapy.

This will help to protect the islet cells from immune rejection, Vertex said in a statement.

“This programme has its roots in the groundbreaking work that began in Dr Doug Melton’s lab, progressed at Semma Therapeutics, and has been accelerated and brought to the clinic by the team at Vertex,” said Bastiano Sanna, executive vice president and chief of cell and genetic therapies at Vertex.

“Ours is the only approach that produces fully differentiated and fully functional insulin-secreting pancreatic islets. We are very pleased to have received FDA’s fast track designation, which facilitates the development and expedites the review of drugs that treat serious conditions and fill an unmet medical need. We continue to work with urgency to bring this innovative therapy to patients,” he added.

The first VX-880 clinical trial sites are now open for enrolment across three locations in the US , with additional sites set to be activated this year.

Vertex is also looking to develop a CRISPR/Cas9 gene-editing therapy for the treatment of severe haemoglobinopathies in partnership with CRISPR therapeutics.

This includes CTX001 – an investigational gene therapy – which has already demonstrated promising early data in transfusion-dependant beta thalassaemia (TDT) and severe sickle cell disease (SCD) patients.

Data announced in December 2020 included analysis of seven TDT patients and three SCD patients.

The TDT patients, prior to receiving the gene therapy, required regular blood infusion to control their symptoms. All seven patients were tranfusion-independent at three to 18 months after receiving a single infusion of CTX001.

In addition, the three SCD patients had not experienced any vaso-occlusive crises (VOCs) in the three to 15-month follow-up period post treatment.