View from the Top, page-2

First 2 Last

Share
Profile

Follow
pigofsteel

10,009 Posts.

03/03/15

13:25

Post #: 14851522
Share

Originally posted by Garfus ↑

I emailed Dr T this morning to gain his perspective, below is an excerpt from his comprehensive reply which only reinforces my confidence in this outstanding company...

The FDA does not provide great detail in their written response other than what we have disclosed.
In short summary, the FDA has discretion on what statistical analysis they rely upon when making a determination of clinical evidence. It is important to recall that our Phase 2 study was designed to measure safety in the very first instance, not efficacy. We nonetheless chose to enrich the study to collect early evidence of drug efficacy where possible. By virtue of the trial design, the study was never intended to be powered statistically ( i.e number of patients and duration of treatment), in the same way that a pivotal, or phase 3 study would normally be.
Within the design limitations of our Phase 2 study, Neuren nevertheless took the opportunity to present an alternative statistical analysis in the Breakthrough Therapy (BT) application, which in our view and in the opinion of our independent experts, still presents a very compelling case. Furthermore, our approach was guided by the knowledge that a different division of the FDA had previously made use of this technique when deciding on a BT application. You will see reference in our November release to what is called the Permutation Test.
In deciding on our BT application, the Neurology division of the FDA has chosen not to rely on our statistical argument, preferring instead to rely on the more conventional technique that is associated only with much larger studies.
No Australian company has to our knowledge received BT designation and the overall success rate for all BT applications is approximately 30%. Neuren made a worthy and credible application with a view of doing all we possibly can to get trofinetide to market as soon as possible.
We will continue to work with the FDA under Fast Track designation to determine the best and quickest way to get trofinetide developed and available to Rett patients. We have not had an opportunity for discussion with the agency as yet…. and naturally we look forward to doing exactly that in the very near term. We remain confident in the knowledge that we submitted a really good case…..even if not quite enough to tick every box right now. We recognise the FDA has a lot of discretion in the determination of BT designation and we are encouraged to speak with them and get a more detailed understanding of their thinking.
As a long-term holder, you may recall that we applied for Orphan Drug designation for Rett Syndrome a couple of years ago, and were declined on the basis the data was too preliminary. Undeterred we re-applied again in Dec 2014 and OD was granted to Neuren last month. Not dissimilar with BT designation, we are able to reapply at any point in time. We believe Neuren is best served doing this once we have had an opportunity to speak directly with the Division of Neurology Products at the FDA.
kind regards
Richard

Expand

geez Garfus, what a great idea, go and ask the Chairman to explain clearly what the announcement means to shareholders... As opposed to dumping your shares .... I must put you on my favorites list... Using your common sense is a rare thing on HC..

So we now have 95m shares changed hands and a 46% drop in price before it was explained as to what the ANN meant... To those that bought and kept cool heads, well done, you will be well rewarded.

NEU Price at posting: 9.9¢ Sentiment: Buy Disclosure: Held
50 Upvote

0 Great analysis

Reply

Top Stories

HLX

Helix investor makes partial takeover bid, proposing board changes

Week 35 Wrap: HotCopper’s top watched; ‘iron ore winter’ has started; Oz inflation @ 3.5% MoM; Harris fracking flip

CCO

The Calmer Co’s Anthony Noble reflects on record breaking FY24

TBN

Tamboran kicks off Shenandoah South pilot project with SS-2H well spud

Consumer Staples Shine Amid Retail Slump – Key Stocks to Watch

There are more pages in this discussion • 64 more messages in this thread...

First 2 Last

Add NEU (ASX) to my watchlist

(20min delay)
Last $15.61		Change 0.400(2.63%)		Mkt cap ! $1.994B

Open	High	Low	Value	Volume
$15.51	$15.82	$15.46	$5.754M	368.7K

Buyers (Bids)

No.	Vol.	Price($)
9	401	$18.73

Sellers (Offers)

Price($)	Vol.	No.
$12.49	8860	2

View Market Depth

No.	Vol.	Price($)
9	401	18.730
10	8918	17.170
1	18813	17.000
1	10429	16.830
1	979	16.690

Price($)	Vol.	No.
12.490	8860	2
14.050	7199	6
14.360	35	1
14.720	16174	9
14.750	1127	2

Last trade - 15.59pm 30/08/2024 (20 minute delay)

NEU (ASX) Chart

View from the Top, page-2

Helix investor makes partial takeover bid, proposing board changes

Week 35 Wrap: HotCopper’s top watched; ‘iron ore winter’ has started; Oz inflation @ 3.5% MoM; Harris fracking flip

The Calmer Co’s Anthony Noble reflects on record breaking FY24

Tamboran kicks off Shenandoah South pilot project with SS-2H well spud

Consumer Staples Shine Amid Retail Slump – Key Stocks to Watch

Featured News

Buyers (Bids)

Sellers (Offers)

Featured News

View from the Top, page-2

Top Stories

Helix investor makes partial takeover bid, proposing board changes

Week 35 Wrap: HotCopper’s top watched; ‘iron ore winter’ has started; Oz inflation @ 3.5% MoM; Harris fracking flip

The Calmer Co’s Anthony Noble reflects on record breaking FY24

Tamboran kicks off Shenandoah South pilot project with SS-2H well spud

Consumer Staples Shine Amid Retail Slump – Key Stocks to Watch

Featured News

Buyers (Bids)

Sellers (Offers)

Featured News