Developing a new drug for orphan CNS disorders: the path from benchside to clinic

Version imprimable
December 1, 2017
Lisa McKerracher

On December 8th, 2017, the CRCHUM Conferences invites Lisa McKerracher, Founder and CEO, BioAxone BioSciences and Adjunct Professor, Department of Neurology and Neurosurgery at McGill University. During this conference she will review bed to bedside translation of their lead drug, a biologic drug to treat acute spinal cord injury.


There are no FDA-approved drugs to decrease progression of disease for many orphan neurological conditions, including the diseases focus on spinal cord injury (SCI) and cerebral cavernous malformations (CCM). As opposed to approaches relying on the re-purposing of already approved drugs, the generation and development of a new chemical entity (NCE) for any indication presents a series of development and regulatory hurdles that must be addressed before clinical trial and through the clinical trial process.  Up to and through Phase 1 clinical studies, which are safety studies in healthy volunteers, the regulatory pathways are relatively clear. After Phase 1, many additional regulatory considerations must be considered so that the clinical trial meets the FDA requirements for drug approval. Studies with drug repositioning can proceed without meeting regulatory requirements for drug approval if they stay within already-established safety parameters. This is why many drug repositioning trials do not lead to clear guidelines for changing standard of care. 

In her talk, Lisa McKerracher will review bed to bedside translation of our lead drug, a biologic drug to treat acute spinal cord injury. This drug, now called VX-210, is in Phase 2b/3 clinical study. They are also developing BA-1049, an NCE, for use in treating CCM. Knowing that the mechanism of action of this compound is through its selective inhibition of ROCK2 helps with pre-clinical study design and the leveraging of transgenic mouse and other models to understand potential benefit. However, significantly more information is needed from the regulatory perspective to inform the development process beyond proof-of-concept in animal models. Safety pharmacology, pharmacokinetic and exposure/tolerability studies are all required well before clinical trial for testing efficacy in humans can be considered. She will compare and contrast the development process for biologic drugs and small molecule NCE’s.  For Phase 2 and 3 studies to show efficacy of new NCEs, regulatory authorities require quantifiable endpoints that measure meaningful functional benefit for patients as efficacy outcome measures. Since there are no approved drugs for SCI nor for CCM, preclinical experiments help inform on possible clinical trial design and she will discuss how a basic research can be used to inform this process.

This conference will be presented in English.

Friday, December 8th, 2017, at Noon.
Auditorium of the CRCHUM, R05.210 and R05.220
900 Saint-Denis Street, 5th floor 
Montreal (Quebec)  H2X 0A9

Lisa McKerracher, PhD
Founder and CEO,
BioAxone BioSciences, Cambridge, MA
Adjunct Professor, Department of Neurology and Neurosurgery,
McGill University


The CRCHUM Conferences are free weekly meetings featuring renowned scientific speakers from various disciplines. These conferences are accredited by the Royal College of Physicians and Surgeons of Canada as continuing professional development activities. The CRCHUM conferences are made possible thanks to the financial support of our partners: AstraZeneca, Banque Nationale, Biron, Charles River, GSK Canada, Lilly, Médicaments novateurs Canada, Merck, Pfizer, Québecor, Roche & Servier.


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