I came across this article a few days ago featured on IBIA titled: "Drug Development in Traumatic Brain Injury" (see full article http://internationalbrain.org/?q=node/100) and just wanted to share my thoughts...
Whilst the article is a nice summary, there's not much new in it. But do hope that they're successful.
I've been thinking about 'point # 5' in particular ie: 'the lack of interest in TBI therapeutics within the industry', and with respect to this, I think this reflects the relatively small market (from a big pharma perspective) compared to anti-hypertensives, pain or depression. While the numbers are large, most patients will only receive a single treatment rather than a life time of prescriptions. That's why oral NNZ-2566 is so promising. The one treatment per patient pattern simply doesn't fit the big pharma marketing approach.
The bigger companies also have cut their R&D expenditures substantially and they never had the degree of innovation that smaller companies do.
(see here: http://www.fiercebiotech.com/special-reports/worlds-biggest-rd-spenders)
With the pressure on big pharma sales and profits, I believe more and more of them are focusing on niche indications and even orphan diseases where the marketing is driven by a handful of key opinion leaders and there's less resistance to premium pricing.
"Mr Glass said that eight of the 20 US key opinion leaders were investigators in the trial and the other 12 were on Neuren’s advisory board."
(See full feature on NEU from Biotechdaily dated October 20, 2011 below.)
Further, when we talk about the pharmaceutical industry, many of us tend to focus on the top 10, many of which have given up on CNS altogether. Within the next tier down, though, there are many specialty pharma companies that are doing quite well pursuing niche indications and that have a greater tolerance for risk.
Those include companies like Shire, UCB Pharma, Cephalon (before the Teva acquisition), Genzyme (even after the Sanofi merger), Forest Labs, Lundbeck, etc... As with the big pharmas, they still tend to rely on smaller companies (like NEU) for innovation and early clinical development but they're doing deals earlier and pricing them more aggressively.
Neuren has told the ASX that a Bell Potter analysis may have pushed its share price 81.25 percent to 2.9 cents on strong volumes.
The ASX queried the price rise from 1.6 cents on October 18 to a high today of 2.9 cents. The company said the analysis by Stuart Roberts had been posted on its website, concluding with a target share price of 12 cents.
Mr Roberts and Neuren chief executive officer Larry Glass met investors and analysts in Melbourne today and told Biotech Daily the company’s phase II trials had no financial risk.
Mr Roberts said Mr Glass was well-connected to the US Army’s biomedical research “three-star general” officials and the US Army spent $2.8 billion a year on research.
Mr Roberts said Neuren had $23 million in US Army and Australian National Health and Medical Research Council non-dilutive funding for its two phase II trials.
Mr Glass said the company was conducting a phase II trial of Motiva (nefiracetam) for post-stroke depression and apathy, which could also have application for Parkinson’s and Alzheimer’s disease.
Mr Glass said that Motiva had “an extraordinary safety profile” having been tested in 2,700 people, but an earlier trial by another company had design faults that meant there was a question of whether it was treating apathy and depression or apathy alone.
Mr Glass said that Neuren’s 122-patient phase II trial of Motiva for stroke with apathy but not depression would inform a phase III trial design.
Mr Glass said that intra-venous NNZ-2566 was in a 260-patient dose-ranging phase II trial for moderate to severe traumatic brain injury, with more than 50 patients enrolled.
“All the direct costs, manufacturing and clinical trials are covered by the Army,” Mr Glass said, adding that enrolment should be completed by the end of 2012, with results in 2013.
But Mr Glass said that the US Food and Drug Administration had agreed that if the trial was successful, the company would be required to conduct a single pivotal phase III trial with 500 to 600 patients, rather than the usual two trials and if all went smoothly, the pivotal trial would be completed in 2015 or 2016.
Mr Glass said the company was formulating an oral version of NNZ-2566 for mild traumatic brain injury. He said that NNZ-2566 was a synthetic analog of Insulin-like growth factor 1 (IGF-1) a naturally occurring neuro-peptide. Mr Glass said that after a brain injury there was an up-regulation of inflammatory cytokines which led to a cascade of inflammation and neuronal cell death.
He said NNZ-2566 inhibited the up-regulation of the cytokines and was hoped to reduce the non-convulsive, or silent, seizures that caused further brain damage.
Mr Glass said the trial had three primary endpoints including the Glasgow Coma Scale, the Portland Adaptability Index and biological outcomes including seizures as measured by electro-encephalogram, blood biomarkers of cell death and intra-cranial pressure.
He said the FDA had approved the trial meeting any one of the three efficacy endpoints.
Mr Glass said there was a $4 billion US market for traumatic brain injury with no approved drugs and only one competitor in development, the hormone progesterone.
Mr Glass said that eight of the 20 US key opinion leaders were investigators in the trial and the other 12 were on Neuren’s advisory board.
He said NZ-2566 had “many other potential indications including penetrating brain injury, stroke, cardiac arrest, perinatal asphyxia and silent seizures in other injuries.
Mr Glass said pre-clinical work had shown NNZ-2566 potential for use in a form of autism known as Rett’s syndrome, with non-growing cell dendrites resuming growth and longterm potentiation, with the possibility of bringing children “back to normal”.
Neuren was up 0.1 cents or 4.35 percent at 2.4 cents with 61.1 million shares traded.
NEU Price at posting:
2.3¢ Sentiment: LT Buy Disclosure: Held
(20min delay)