Reports of brain damage call a halt to Bellicum trials: industry news round-up

Written by Roisin Conneely

Pharmaceutical policy announcements, promising drug trial data and more: check out our round-up of the biggest news from the neuroscience and neurology industry this week.
Our pick of the headlines this week:

  • Bellicum Pharmaceuticals forced to halt drug trials after three cases of brain damage reported
  • Positive results from Phase III eslicarbazepine acetate epilepsy trial
  • Pfizer confirms cull of neuroscience programs
  • Sunovion announce Phase III Parkinson’s trial results for apomorphine
  • Sage Therapeutics present findings from latest investigation into insomnia drug

Bellicum Pharmaceuticals forced to halt drug trials after three cases of brain damage reported

The US FDA has imposed a clinical hold on Bellicum Pharmaceuticals’ (TX, USA) leading stem cell therapy product following reports of encephalopathy in three patients, deemed possibly related to the treatment.

The experimental drug, BPX-501, had been hailed as a safer option for stem cell transplants, with the drug planned to be administered to patients following allogeneic hematopoietic stem cell transplants. Primary aims of the drug are: fighting infection, aiding engraftment and preventing disease relapse.

Bellicum has stated that encephalopathy has previously been reported in allogeneic hematopoietic stem cell research, whilst also emphasizing that the three patients in question have a “number of potential confounding factors”, including immunodeficiency and concurrent infection.

The company is awaiting further instructions from the US FDA to determine the circumstances required to restart trials.

Positive results from Phase III eslicarbazepine acetate epilepsy trial

BIAL and Eisai (Porto, Portugal and Hatfield, UK) have announced results from their Phase III investigations into daily Zebinix® (eslicarbazepine acetate) monotherapy for newly diagnosed epilepsy.

The trial of 815 patients indicated that monotherapy with the drug is non-inferior to the standard treatment: twice-daily, controlled-release carbamazepine.

Zebinix met its primary endpoint of non-inferiority, with 71.1% of patients treated with the drug remaining seizure-free throughout the duration of the 26-week trial, whilst 75.6% of carbamazepine patients were seizure-free for 6 months or more.

The experimental voltage-gated sodium channel blocker was also observed to be well-tolerated, with rates of treatment-emergent adverse effects remaining similar between Zebinix and carbamazepine (73.3% and 79.6%, respectively).

“For this patient population in particular, it is important that the physician takes into consideration individual factors and tailors treatment to the individual. We therefore welcome the news that another treatment has been shown to be effective for these patients,” lead author of the study Eugen Trinka (Paracelsus Medical University, Salzburg, Austria) commented.

Pfizer confirms cull of neuroscience programs

Following Pfizer Inc.’s (NY, USA) announcement last month that they would be cutting their neuroscience drug development programs, the pharma giant has now confirmed that all neuroscience development will be cut except for its late-stage pain program allied with Eli Lilly and Company (IN, USA).

Drugs being cut include a GABA-A receptor agonist for epilepsy and a dopamine 1 activator for cognitive disorders.

Investigations into several potential cancer drugs are also being halted, including an IDO1 inhibitor and PD-L1 checkpoint Bavencio® for third line gastric cancer, following failure in earlier trials.

Read more about the initial announcement

Sunovion announce Phase III Parkinson’s trial results for apomorphine

The findings from Sunovion’s (London, UK) Phase III apomorphine trial have been released, indicating superior efficacy of the drug in patients with Parkinson’s disease.

Study CTH-300 recruited 109 Parkinson’s disease patients, aiming to reduce their rate of OFF episodes. Participants who were given apomorphine sublingual film, APL-130277, exhibited a significant reduction in the Movement Disorder Society Unified Parkinson’s Disease Rating Scale after 12 weeks of therapy, compared with those receiving a placebo.

The US FDA has approved Fast Track Designation for APL-130277, and data from the trial will be used to support Sunovion’s submission for a New Drug Application, anticipated in spring 2018.

Discover more about apomorphine as a Parkinson’s therapeutic here

Sage Therapeutics present findings from latest investigation into insomnia drug

A placebo-controlled trial carried out by Sage Therapeutics (MA, USA) has yielded promising results for SAGE-217 as a potential treatment for insomnia.

The drug met primary endpoints of improved sleep efficiency and improvements in maintaining sleep, in comparison with a placebo.

The Phase I/II study enrolled healthy volunteers and used polysomnography to create a 5-hour advance model of insomnia. SAGE-217, administered at either 30 or 45mg, was observed to raise sleep efficiency levels to 85% and 88%, respectively. Total sleep time also demonstrated significant improvements and the drug was generally well-tolerated, with no serious adverse effects.

Based on these results, the company expects to begin clinical development of SAGE-217 as a sleep disorder therapy in 2018. Chief research officer at Sage Therapeutics, Jim Doherty, commented on the announcement: “Our evaluation of SAGE-217 in multiple clinical trials, across several indications, suggests that the drug’s mechanism of action may rebalance fundamental brain circuitry, therefore supporting SAGE-217’s development across a broad variety of disorders. These findings suggest that SAGE-217 has the potential to assist sleep maintenance, and we were pleased with the tolerability demonstrated for both doses in this trial.”