NEW YORK—Back in December, Q BioMed Inc., a biotechnology acceleration company, announced a collaborative agreement with SRI International to provide formulation development, preclinical development and early clinical manufacturing of QBM-001, in support of Q BioMed’s autistic spectrum disorder (ASD) drug development program for nonverbal or minimally verbal autistic children.
SRI has broad expertise in solving formulation, drug delivery and characterization challenges of small-molecule drugs and biologics. The formulation team, led by Dr. Gita Shankar, is experienced in the development of novel formulations that have reached clinical trials. They are currently developing a formulation for QBM-001 with relatable chemical properties and formulation requirements.
According to Denis Corin, CEO of Q BioMed, “QBM-001 is a combination therapy that is designed to reduce the levels of two molecules that are elevated in our target group by targeting multiple pathways. When these two molecules are elevated, they are known to induce neurodegeneration and inhibit speech. The elevated levels of these molecules tend to be treatment-resistant when only one pathway is targeted. One targeted pathway is adding the components that normally regulate the levels in the body, which is one focus of our combination therapy. However, past studies (not from Q BioMed) have shown this only reduces elevated levels by 20 to 30 percent. That is why we targeted another pathway with a different compound that is designed to correct a negative feedback loop.”
“The third target prevents this neuronal death by preventing the elevated levels from triggering an mTOR pathway,” Corin continues. “At the same time, this third component also helps reduce the elevated levels. Together, we feel this approach should provide an effective and much-needed therapy that should facilitate a more typical cognitive and speech development path for this subgroup within the autism spectrum.
“Equally important is the safety of QBM-001. All components are very safe. Only one has potential gastrointestinal side effects, and although our dose is not known to cause those side effects, we went ahead and developed an analog of the parent molecule that eliminates any GI issues. In addition, our formulation will further mitigate any possible GI issues.”
The preclinical studies for QBM-001 will be led by Dr. Stephen Morairty and his team in SRI’s Center for Neuroscience. Morairty and his colleagues are experienced in working with several preclinical autism models.
“As with all our outsourced needs, we first search for several potential providers that fit the criteria based on case studies with similar needs, expertise in the area needed and, of course, price. We then subject those to a rigorous request for proposal screening,” Corin notes. “During this process, SRI stood out due to their ability to provide case studies of having executed similar formulations. In addition, and equally important, was their past experience with several autism animal models, and thus they showed great interest in our approach, engaged with several questions and provided valuable insight into preclinical modeling—which really set them above the rest since in general there is not a lot of experience with autism in the service provider space, because there are no approved products and very few trials.”
Corin also mentions that while some members of the Q BioMed team have worked with SRI International in the past, this will be the first time that Q BioMed itself has worked with SRI.
“We are pleased to have found both experience with autism models and expertise in formulation of products like QBM-001 in SRI International. This important step will be the catalyst for several milestones for QBM-001 over the next few months as we prepare clinical product for the trials we anticipate starting in 2019,” he says.
QBM-001 targets toddlers with pediatric developmental nonverbal disorder, where an underlying commonality may lead to developmental delay, an autism diagnosis and eventual nonverbal or very minimally verbal capability for the rest of their lives. There are approximately 18,000 new cases of pediatric developmental nonverbal disorder in the US each year and a similar amount in Europe. The majority of the children are diagnosed as young children and fall within the autism and epilepsy spectrum disorders.
Not all individuals who become nonverbal will benefit from QBM-001. However, with validated biomarkers, testing from trained specialists and genetic testing, children who fall into this targeted population can be identified, and will have a higher likelihood of responding to treatment.
“There are no specific advocacy groups for these individuals,” Corin adds. “There are no dedicated research programs, and we are the only company working on a treatment for this subgroup in the autism spectrum. That is highly motivating, and even more so when we see the response of key physicians who are equally excited and cheer us along, and we are very grateful to the many who have provided valuable insight.”
“The formulation is underway and the first formulation batch should be available in late March. That will start the clock for our stability testing, for which we need at least four months, as that is the planned duration of our trial. From March until the beginning of June the formulation will be further fine-tuned to ensure it provides the correct, sustained levels of each compound in the blood. In parallel, preclinical testing in an autism animal model that we feel best represents our target group has just started this month as well. We anticipate formulation being complete by the end of Q2, which will allow us to then file an IND. That would put us on target to start trials towards the end of Q3 this year,” concludes Corin.