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Provention Bio wins PRIME designation for type 1 diabetes prevention drug

Meanwhile J&J partnered Crohn's drug flops in phase 2

Provention Bio

Provention Bio has announced its lead candidate PRV-031 (teplizumab) has been granted Priority Medicines (PRIME) designation from the European Medicines Agency (EMA).

Back in June, Provention Bio saw its share price double after releasing data which showed that the experimental immunotherapy could delay the onset of type 1 diabetes in children and adults by a median of two years.

The phase 2 trial in 76 patients at risk of developing T1D were given PRV-031, which demonstrated a significant delay in the onset and diagnosis of the chronic condition. Compared to placebo, children and adults who were considered high-risk of developing T1D saw it delayed by a median of two years.

The results were even more impressive for a subgroup of patients – around 60% - who did not develop the condition at all in the study period.

Now, the biotech has been given a PRIME designation by the EMA, which is awarded to promising medicines that demonstrate the potential to address substantial unmet medical need based on clinical data.

A PRIME designation gives medicines priority and provides them with special support – this includes enhanced interaction and dialogue, and an accelerated evaluation and review.

“We are very pleased the EMA recognises the transformative potential of PRV-031 and has granted this groundbreaking therapy PRIME designation", said Ashleigh Palmer, CEO of Provention Bio.

“Following on the heels of Breakthrough Therapy Designation from the FDA in August, an EMA PRIME further validates the rationale for PRV-031 to prevent or delay the onset of clinical T1D in at-risk subjects. We are committed to working closely with both regulatory agencies to bring PRV-031, as quickly as possible, to the many thousands of T1D at-risk individuals who currently have no options to prevent or delay this catastrophic disease,” he added.

The US biotech was established only three years ago, and acquired rights to PRV-031 from MacroGenics, which suspended its development of the drug in 2010 after a clinical trial run with Eli Lilly showed it failed to show sufficient efficacy in T1D patients.

Teplizumab works by binding to the pro-inflammatory T cell co-receptor CD3, which in turn prevents them from activating the T cells that attack and kill the pancreas’ beta cells.

Stumble in Crohn’s study

On the flip-side, Provention also announced that one of its J&J partnered drugs has failed a mid-stage study.

The experimental therapy, PRV-6527, was designed to inhibit colony-stimulating factor 1 receptor (CSF-1R) signalling. The drug was being studied in a phase 2a study in moderate-to-severe Crohn’s disease patients, with its primary endpoint being to demonstrate a change in the Crohn’s Disease Activity Index (CDAI) score at week 12.

This endpoint was not met, with the drug proving to be only as effective in improving symptoms as placebo. According to Provention, the ‘high placebo response’ was linked to background medication used in a significant number of the patient population.

"Despite the PRINCE study's high placebo effect, single dose level, limited 12-week duration, and relatively small sample size, the initial top-line results highlight how PRV-6527's CSF-1R inhibition intercepts the migration of inflammatory cells to the gut. This rapid go/no-go, signal finding trial indicates a potential role for PRV-6527 as a well-tolerated, oral, first-line therapy in early Crohn's and, potentially, for the prevention of relapse," stated Ashleigh Palmer, CEO of Provention Bio.

He added: “Under the terms of our in-license agreement, Janssen now has 90 days to exercise its option to re-acquire this asset to support further development in inflammatory bowel disease.”

Article by
Lucy Parsons

28th October 2019

From: Research



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