Neuren Pharmaceuticals delivers encouraging Pitt Hopkins trial results

Neuren Pharmaceuticals’ trial of a treatment for the rare Pitt Hopkins syndrome has delivered encouraging results, sending its shares sharply higher.

Neuren shares are up strongly on positive trial results.

Neuren Pharmaceuticals’ phase two clinical trial of its drug to treat Pitt Hopkins syndrome showed statistically significant improvements in patients, sending the company’s shares more than 10 per cent higher.

The drug developer said on Monday the trial of NNZ-2591 in 16 children with the syndrome showed efficacy across all of the four measures the trial was designed to test for.

“Statistically significant improvement from baseline was observed by both clinicians and caregivers from treatment, across all four efficacy measures that were specifically designed to assess the core characteristics of Pitt Hopkins syndrome (PTHS),’’ the company said.

“There are no approved treatments for PTHS despite its severely debilitating impact on the lives of patients, as well as their parents and siblings.’’

Neuren chief executive John Pilcher said the company was “very excited’’ about the trial results.

“This underserved community has such urgent unmet need and we can now continue towards our goal of developing a first approved treatment,’’ Mr Pilcher said.

“We are very grateful to the people in the Pitt Hopkins community and at the trial sites in the United States who enabled the successful completion of this extremely challenging, but groundbreaking trial.’’

The primary endpoints of the trial were around safety, tolerability and pharmacokinetics, while the secondary endpoints were around its efficacy.

“NNZ-2591 was well tolerated and demonstrated a good safety profile. All treatment emergent adverse events were mild to moderate and most were considered not related to study drug,’’ the company said.

On the efficacy front Neuren said the results for measures rated by both clinicians and caregivers “showed a level of improvement considered clinically meaningful’’.

“Nine out of 11 children that completed the trial showed improvement measured by the PTHS Clinical Global Impression of Improvement (CGI-I), an assessment by the clinician of the child’s overall status compared with baseline,’’ Neuren said.

“The mean CGI-I score was 2.6. Five children received a score of either 1 (very much improved) or 2 (much improved).’’

PTHS is estimated to affect as many as one in 34,000 people and its symptoms include moderate-to-severe intellectual disability, behavioural differences, hyperventilation and/or breathholding while awake, seizures, gastrointestinal issues and lack of speech.

Wilsons Advisory lifted its price target on Neuren by 13 per cent to $30 on the news, saying the announcement “should elevate investor’s confidence in the reality of NNZ-2591 as a future blockbuster and appetite to reflect that in valuation now’’.

Neuren shares were up 14 per cent at $23.62 in late afternoon trade.

Originally published as Neuren Pharmaceuticals delivers encouraging Pitt Hopkins trial results