Might as well include the good news flowing into the pages of the AFR as well today ...

Biotech sensation Neuren rallies to record on clinical trial results

https://www.afr.com/markets/equity-markets/biotech-sensation-neuren-rallies-to-record-on-clinical-trial-results-20231218-p5es8l

and for those outside the paywall ...

Neuren Pharmaceuticals shares soared 29.5 per cent on Monday, giving the biotech group a record $2.8 billion valuation after it revealed positive phase-two clinical trial results for its drug to treat the cognitive disorder Phelan-McDermid syndrome in children.

The Melbourne-headquartered company’s trial success for the NNZ-2591 drug backs up its launch of DAYBUE, an oral form of a drug to treat Rett syndrome, a rare developmental disorder also in children, in the United States.

Neuren shares closed at $22.20 after coming out of a trading halt in place since Thursday.

“We’re very happy,” said Neuren chief executive Jon Pilcher. “This phase-two trial was a really important hurdle for our second drug. We couldn’t have got a better result. The amount of improvement seen in patients in 13 weeks of treatment has exceeded the expectations of both physicians and caregivers. Important things changed in [patients’] behaviour and communication.”

Neuren is separately conducting phase-two trials for NNZ-2591 to treat other neurodevelopmental disorders such as Phelan-McDermid, for which there is no or little approved treatment: Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome. The drug uses chemical compounds to improve connections between brain cells for tasks such as hand movements or visual communication.

The biotech’s clinical success has powered its shares 17-fold in two years as it earns royalty payments from US sales of DAYBUE, which partner Acadia expects to reach up to $US87.5 million ($130 million) in the December quarter alone. That would be worth $13.7 million to Neuren.

Mr Pilcher said he had no firm timeline on a phase-three trial for the Phelan-McDermid syndrome drug, with contingencies including a potential partnership to fund the trial with a deep-pocketed biotech partner. The phase-two trial was conducted with 18 participants.

“But we’ve got more than $200 million cash on our balance sheet, so we can fund it ourselves if we need to,” he added.

Investors including Andrew Mitchell of Ophir Asset Management are backing Neuren to succeed.

“Today’s results were very well received for good reason,” Mr Mitchell said. “We’re still excited about Neuren’s prospects.”

Listened to the brief snippet of the talk on "The Call" that was posted on twitter (I'll listen to the full show later).

Bell Potter expected CGI of 3.5 and said it "fell short of expectations" when the result was 2.4. But isn't 2.4 a better score than 3.5???

Guess it shows how poorly some people read into the results... Or am I reading the results wrong here..

Disc: Held IRL and on Strawman.

Great result. Jon Pilcher on the call now say "We had extremely high expectations of NNZ-2591. The results have exceeded those expectations." He is clearly very pleased.

Share price action blew past my "buy" order .... I'm not too sad. $NEU is now my second largest RL holding. I'll leave the order in, as there is often some pull back after the initial pop.

56% clinicians report patients are "much improved" or "very much improved". This bodes very well for Phase 3.

This de-risking of NNZ-2591, which may yet prove to be effective in multiple neurological conditions, significantly ups the risked value of $NEU IMHO.

Disc: Held in RL and SM

50% of the children scored much improved benefits. Quite amazing really for a P2 trial. With some slight adjustments on amounts, delivery timing etc P3 will hopefully see even better results. Always nice to see companies developing products to help those less fortunate.