In preparation for its pivotal study with its autism diagnostic aid, Blinklab (BB1: $0.64) CEO Henk-Jan Boele said that the company's program passed the ultimate stress test.
Moving into a pivotal study, the company wanted to make sure there would be few surprises in the forthcoming study, 'avoiding risk' said Boele. Boele said the company pushed the test to its limits and it passed, in what was a very diverse group of children.
The sensitivity achieved was 83.7% and the specificity was 84.7%. This well exceeds the requirements from the FDA of greater than 65% on both measures, which was stipulated by the regulator just earlier this month.
Only 100 children (between the ages of 2-10) were expected to be enrolled. However the study assessed 485 children. The robustness of the results is not just from the high accuracy, but that the study enrolled children with no developmental disorders, children with different developmental disorders, children with combined disorders, such as autism and ADHD, and children with mild autism who are the most difficult to diagnose according to Boele.
The study was conducted at two sites in the US. It recruited in just over seven months, including some initial hiccups, indicating a strong demand for such a test and acknowledgement from clinicians of interest in the technology and the approach to assist with diagnosis of this disorder.
In a previous study in Morocco in 441 subjects, the population was more homogeneous with more typical symptoms of autism. The sensitivity and specificity achieved was 91% and 85% respectively. With such crossover in the population in the pilot study just conducted, the retained high specificity was a particularly good outcome.
Also impressive with this study outcome is that 90% of the recordings were conducted in the home setting, which supports how robust the test has become.
Pivotal Study Next
The next stage for the company is its pivotal study to gain FDA approval. This will be conducted at seven US sites that have already signed on for the study.
Given the strength of the recent results, Blinklab now expects that trial size to be substantially smaller than originally anticipated (1000), at 528 subjects. This smaller pivotal study has also been agreed to by the FDA in the recent meeting. As a result the trial will cost less and should be completed more rapidly.
Boele said that clinicians are viewing the company's technology not as a simple test, but as a diagnostic tool that can quantify sensory processing…."essentially a window into the brain." Boele said that the enthusiasm from the clinicians in the study highlights that the company is working on an important advancement in this field.
In the pivotal study as in this pilot study, at least two clinicians will make the formal assessment. If those clinicians do not agree, then a third clinician from a different site will make the final decision. Boele acknowledges that autism assessment is inherently subjective. The company is seeking to provide clinicians with the tools to improve upon the current gold standard.
There are obvious challenges in developing a diagnostic where the gold standard may not be 100% accurate itself. Providing additional colour around the patient responses, including measuring repetitive vocal responses and hand and other body movements, will assist clinicians with formal diagnosis. This sets the technology apart from approved tests, which focus on social behaviour and eye gazing.
Other Studies
Other studies continuing are in ADHD in Europe, in adults with autism (in Amsterdam), in Alzheimer's disease and dementia (in Rotterdam), and a family study with Monash University seeking to identify genetic and environmental links in autism and in ADHD.
Blinklab is capitalised at $82 million. It held cash of $8.7 million at the end of June, which gives the company around two years of funding.
Bioshares recommendation: Speculative Buy Class A
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