Prize for 'negative scientific results' awarded for work on experimental drug
Winners are selected especially on the basis of the impact of their findings in helping the field to optimise its resource and investment allocation and, in doing so, advance long-term treatment development.
The 2019 award goes to a paper by David Brown et al (see below), which shows that the novel compound BI409306 does not improve mental functioning in schizophrenia, but that it does have a good safety profile and merits investigation for other disease areas.
Michael Sand, co-author and Senior Clinical Program Leader CNS at Boehringer Ingelheim Pharmaceuticals in Connecticut, U.S.A., accepted the award on behalf on the research group. He answered the following questions.
- 1. What does the work prove and disprove? "The study demonstrated that while not an effective drug to improve cognition in patients with schizophrenia, it showed very clearly that is a well-tolerated drug with an acceptable safety profile—both of which are very important as Boehringer Ingelheim continues to explore other potential uses for the drug".
2. What have you done? "Boehringer Ingelheim assessed a novel compound on the cognitive function of a large (n>500), multinational (5 countries in North America, EU and Asia) population of patients living with schizophrenia in a randomized, double blind 12 week study".
3. Why did you decide to undertake this work? "Boehringer Ingelheim conducted this study because there are no medications approved to treat the cognitive impairments that people with schizophrenia live with and the mechanism of action of our compound—PDE9 inhibition—had shown promising preclinical evidence of a pro-cognitive effect".
5. What (if any) would be the next steps be for you? "As a result of the learnings from this study Boehringer Ingelheim is studying the PDE9 compound in other areas of unmet medical need in patients with, and at risk to develop, schizophrenia. Also, this experience helped us to improve the design of other studies, now ongoing, in people with cognitive problems due to schizophrenia; these studies test the potential benefits of compounds with other modes of action".cognitive problems due to schizophrenia; these studies test the potential benefits of compounds with other modes of action".
6. Anything else which you think would be interesting/relevant? "This trial used a novel methodology that helped to overcome one of the challenges in conducting and interpreting cognitive studies. Further, this trial has sparked research and analysis into the methodology the field uses to identify appropriate subjects for trials such as this. It also is the first study to use a patient-reported outcome measure where the individuals living with the problem assess how they experience their illness".
Commenting for the ECNP, Professor Eduard Vieta (Barcelona) said:
"The negative results initiative tries to encourage the publication of studies which contribute to science, even if they don't reach a positive outcome. It is very important that scientists understand not just what works, but also what doesn't work. The study by Dr. Brown, Dr. Sand and their colleagues is exactly the type of work we want to support with this prize".
Professor Vieta was not involved in this work, this is an independent comment.
Provided by European College of Neuropsychopharmacology