Optimization of soluble gamma-secretase modulators for the treatment of Alzheimer’s disease PDE4D allosteric modulators for treating cognitive impairment Small-molecule development of PrPc antagonists for the treatment of Alzheimer’s disease Triazine-based compounds to promote regeneration in optic neuropathies Targeting cytokine-mediated pathologies for neuroprotection in treatment of AMD Small-molecule drugs for treatment of dry age-related macular degeneration It should be noted that projects from academic institutions were highly represented throughout the stages of the program (60% of projects at the onset, 80% of projects reaching the preclinical safety stage) demonstrating the quality of the starting projects and success of our mentoring from our NIH staff and consultants. The regular interactions allowed for maximum benefit of the BPN infrastructure and consultants’ expertise to the grantees, as well as focusing efforts on critical path activities. Additionally, as projects progressed, Lead Discovery Teams met face-to-face at major transition points such as start of Lead Optimization, Start of Development and Investigational New Drug preparation. Formal milestone reviews with go/no go decisions for continued funding typically occurred once or twice a year depending on the stage of the program. Through these meetings, progress was measured against milestones, and scientific direction was altered collaboratively based on emerging data in order to give the teams the best chance at meeting their milestones and advancing further. The BPN formed customized Lead Discovery Teams around each of the grantees’ project needs and worked in a collaborative fashion, typically meeting every 2 weeks via web-enabled meetings. The BPN launched 15 projects over the period 2011 to 2013, directed at 13 different disease indications in the mission of 7 different BPN ICs (listed in Table Table1). In practice, each application round was reviewed by a National Institute of Neurological Disorders and Stroke Special Emphasis Panel consisting of members with disease expertise, as well as drug discovery and development experts with industry experience in multiple disciplines. Therefore, careful consideration was given in the program to ensure that intellectual property of the contributing academic institution or small business was protected and unencumbered by the NIH in order to simplify licensing discussions with pharma and venture capital investors.Īs a first-of-its-kind program at NIH, we projected prelaunch a 50% attrition at each stage considering an overall lower success rate for central nervous system drugs, limited NIH resources, and the average industry attrition rates. Another explicit goal of the BPN was to de-risk the development of new, impactful therapies that could attract commercial interest enabling a hand-off to industry for late-stage development and additional clinical trials. The BPN’s initial target was to start up to 20 milestone-driven projects and advance those projects with the most promise of obtaining Food and Drug Administration approval to enter and execute phase I clinical trials with BPN resources. NIH = National Institutes of Health PK/Tox = pharmacokinetics/toxicology The BPN is a milestone-driven cooperative agreement program bringing together a unique blend of grant dollars, industry-standard scientific expertise, and contract resources to foster drug development from the discoveries of academic and small businesses with the goal of advancing at least one compound into clinical trials by the end of the first 5 years. To foster small-molecule neurotherapeutic development, the NIH Blueprint Neurotherapeutics Network (BPN) ( ) was launched in 2011, creating a first-of-its-kind virtual pharma network at NIH (Fig. With many biopharmaceutical companies exiting or significantly downsizing their neuroscience efforts in the last decade, it highlighted the need for NIH to provide a catalyst for neuroscience drug discovery efforts. To address the need for crosscutting neuroscience research, the NIH established the NIH Blueprint for Neurological Research ( ) comprised of 15 separate NIH ICs whose missions have neuroscience components. Neuroscience encompasses a wide variety of diseases and spans the missions of many of the National Institutes of Health (NIH) Institutes and Centers (ICs).
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