OK, why the post? What did they do?

Today NTI CEO Mr Duthy presented the full results of their 12 wk open label trial carried out on 14 girls with an average age of around 8 years using their lead cannabis derived drug NTI1164 on the conditions Rhett syndrome.

Where the results any good?

There was one adverse health event in the trial and it met the primary end point

Very roughly using the standard CGI-1 test they achieved a 10% improvement over 9 “exploratory anchors” conditions

Using 4 “core anchor” conditions they achieved a 23% improvement (p=0.04)

What are you talking about?

CGI-1 is the standard cognitive/behavioral test used to evaluate drug efficacy in conditions like Rhett. It was used by NEU to obtain their Daybue FDA registration.

When they talk about “4 core conditions”, they are exercising their right to focus in on just the best results. This is perfectly legitimate and what makes NEU’s PhIII crack at FDA approval for PMS so interesting.  They will legitimately zero in on the conditions they did best at in the earlier trials to frame the later trials.

What else?

 The other key results was their RSBQ result which showed a 13.4 decrease over the baseline (p <0.001) – a 30% improvement.  Impressive. So impressive they showed a slide highlighting that NEU with Daybue in their Lavender Phase 3 trial had only shown a reduction of 4.9. (To be honest about it, they should have also shown a NTI164/Daybue comparison slide for the CGI-1 test).

Agreed that looks pretty good, what else?

CEO Mr Duthy was chipped in the webinar that it was only open label with just 14 subjects, so by inference pretty meaningless. 

His response was that with NTI's other trials, when they had gone to larger blinded NTI164 trials the earlier results held up.

So what do you want me to do?

Nothing, just watch. 

Mr Duthy said NTI is committed to the orphan conditions of Rhetts, PANS and CP even though Autism is a much larger market and NTI last month got some pretty results for their PhII/III trial.  They want to push the Autism results with the TGA and get an Australian provisional approval. Much cheaper and would be a real coup if they can pull it off. 

They only have around $14m and a lot on their plate. Mr Duthy provided a slide on what they are going to do in the next 8 months. Nothing really exciting.

Oh, thanks for wasting my time Scoonie!

 That’s OK, just try to be patient.  The CEO is not an idiot and they could be onto something.

And hey, why don’t you go talk to someone smarter than me on this.

As documented everywhere, NEU has had a spectacular run with their drug Daybue and the up and coming drug NNZ2591. 

The NNZ2591 Phase 2 topline results for the treatment of Phelan-McDermid Syndrome (PMS) were released to the market on the 18/12/23.

And the results were impressive, in short:

• “Clinical Global Impression of Improvement (CGI-I) – mean score of 2.4 with 16 out of 18 children showing improvement assessed by clinicians.
• Caregiver Overall Impression of Change (CIC) – mean score of 2.7 with 15 out of 18 children showing improvement assessed by caregivers.”

The NEU PMS trial was a Phase 2, 13 week, open label undertaken on just 18 children.

In the immediate days following the release of the results NEU share price went from around to $16 to a peak of $24.50.

This represented an increase in NEU’s market capitalisation of around $1b.  

However given the sales potential of a successful NNZ2591 drug for PMS and then potentially the other in-their-sights orphan conditions, we all can appreciate why.

Neurotech International (NTI), this month released Phase 2/3 results for their cannabis derived drug NT1164 on Autism Spectrum Disorder. In NTI’s release the trial design and purpose was:

“NTIASD2 is a Phase II/III Double-Blind, Randomised and Controlled-to-Open-Label Study to assess the efficacy of NTI164 up to 20mg/kg/day on the severity of spectrum disorder (ASD) in up to 54 patients aged 2-17 years (inclusive).”

The improvement results whilst not good as NEU’s PMS trial, were pretty solid:           

“NTIASD2 Phase II/III clinical trial met the primary endpoint of a statistically significant improvement in severity of illness (CGI-S) at 8 weeks between NTI164 and placebo (p<0.001) • Children in NTI164 group re-classified from markedly-severely ill (CGI-S: 5.54) at baseline to mild-moderately ill (CGI-S: 3.77) at 8 weeks, a very strong improvement ><0.001)

Children in NTI164 group re-classified from markedly-severely ill (CGI-S: 5.54) at baseline to mild-moderately ill (CGI-S: 3.77) at 8 weeks, a very strong improvement.”

The response from the market to NTI’s announcement was flaccid with the M/cap rising just $9m and since falling back to below the post announcement level.

Without trying to sound like a pal of Mr Culper, the NEU PMS Ph2 trial was open label and only tested 18 children.  And there are a lot more ASD than PMS suffers.   

Three years ago John Pilcher was running just another cash starved on-the-bones-of-its-arse biotech hopeful.

Is NTI shorthand for Not To Invest or is there something being missed?