Here are my further thoughts on the AGM. There will be some repetition with
my previous (and other people's ) comments. I agree with pretty much
everything sean64 and herro have said so far. I will mix RT's comments from
the AGM with my own opinions and I hope I keep the distinctions clear.
To start with, here is some text taken from the FDA website:
"The Office of Drug Evaluation I consists of three review divisions: The
Division of Cardiovascular and Renal Products, Division of Neurology
Products, and the Division of Psychiatry Products."
"The Division of Psychiatry Products (DPP) regulates and reviews
Investigational New Drug (IND) applications and marketing applications for
products for the treatment of psychiatric diseases and conditions, such as
bipolar disorder, schizophrenia, schizoaffective disorder, major depressive
disorder, attention deficit disorder and attention deficit hyperactivity
disorder, obsessive-compulsive disorder, panic attacks, posttraumatic stress
disorder, and generalized anxiety disorder. "
"The Division of Neurology Products (DNP) regulates and reviews
Investigational New Drug (IND) applications and marketing applications for
drug and biologic products for the treatment of neurological diseases and
conditions, such as Alzheimer’s disease, stroke, Parkinson’s disease,
Huntington’s disease, epilepsy, migraine headaches, muscular dystrophy,
amyotrophic lateral sclerosis, multiple sclerosis, cerebral palsy, dementia,
narcolepsy, lennox-gastaut syndrome, and insomnia."
So Fragile X and Rett are dealt with by different departments, so for Neuren,
talking with the FDA about FXS was starting from square one.
RETT
For the next 2-3 years this will be the one to watch. RT was clearly
emphasising that this is what has the company's attention above all else. He
emphasised the positive relationship NEU has with DNP, "happy to iterate
outside formal meetings". He commented that they may revisit BT after the
paediatric trial at the end of the year. I don't believe BT would affect the
timeline very much. RT seemed very confident that they would get this trial
done by end of year and have the results out in Q12017. They should start the
PhIII trial in H2 of 2017. The trial will have 195 patients over a year. I'm
going to pluck a number out of the air and suggest that the rett trial is
costing about $3m a quarter. So for a trial with 3 times as many subjects
and, say, twice as much clinical interaction, that's $18m a quarter. I could
be utterly wrong- feel free to throw some maths back at me- but that gives
you a feel for the challenges.
FXS
My reading of RT's comments is that dealing with DPP is a whole new ballgame
and NEU is starting from scratch in terms of agreeing on clinical outcomes
that work in Trof's favour. The fact that the DPP requires extra toxicology
and results from the Rett trial for the next FXS trial, while the DNP have
allowed a paediatric trial with much higher doses, shows that NEU are dealing
with a different bunch of people and a whole new bureaucracy. Nonetheless, RT
described FXS as being 12 months behind Rett and agreed with a questioner who
asked if pivotal Ph III would start in 2018. It was asked whether the PhIII
trial for FXS would be a single pivotal trial like Rett, and RT commented
that they would need further meetings with the DPP before being confident
about that. They have not applied for BT.
TBI and concussion
RT indicated that the US Army were still interested in Trof, his phrasing was
more positive than the rather dry way it's described in the written text. LG
seemed confident that, even though a further trial would be more restrictive
from a recruitment point of view, lack of competition from other trials would
help. They have 6 trial sites, 3 in particular high performing, that they
feel they have a good relationship with so a further trialk should go faster:
the last year of the TBI trial is a good indicator (which by my guesstimate
would mean a further trial of the same size would take 2-3 years) RT's
comments: "Things are wonderfully clear in hindsight. The army is genuinely
motivated. For them the mission is still on." RT said that while it "wasn't
approprate to headline" with the RBANS data, there was "quite a profound"
difference between drug and placebo (I was writing like fury at this point
but that word "profound" struck me).
Concussion wasn't discussed much but I recall from the last AGM that
recruitment was a real problem.
EVERYTHING ELSE
They have enough money for 2016 but will need more for 2017. RT mentioned
cash from options, tax rebates etc. My rough calcs had them running low by Q4
but I haven't included these othe income sources. RT talked of balancing
short term needs vs long term potential value. They are evaluating whether to
do a deal pre or post trial completion. RT was asked about Leerink but didn't
say anything very specific. I think the general (unsurprising) consensus was
that the company was felt to be undervalued.
So what for timelines?
There may be ongoing discussions with the FDA but I doubt anything overtly
market sensitive.
The review of TBI/ concussion will happen in Q3 so maybe news on this in Q4.
While one of the key comments was that NEU was funded through to end 2016, I
would guess further funding would be announced in Q4.
Then trial results Q1 next year.
(20min delay)