About 18,000 children in the US—and a similar number in Europe—have pediatric developmental nonverbal disorder, the majority of whom are diagnosed as young children and fall within the autism and epilepsy spectrum disorders. The existing medications that are prescribed to those with autism are psychotherapeutics that, while affecting mood and temperament, do nothing for the underlying disorder.

Q BioMed, Inc. QBIO, a New York City-based biotechnology acceleration company, has a lead program in autistic spectrum disorder drug development for nonverbal or minimally verbal children. The company’s QBM-001 compound 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. According to Q BioMed, the economic cost for toddlers that become non- or minimally verbal averages $10 million per person over a life span, and, collectively, an estimated $200 billion is spent annually on individuals who have become nonverbal in the US.

The company announced this week that Pamela Ventola, PhD, an Assistant Clinical Professor at Yale University School of Medicine, Child Study Center, has joined Q BioMed’s advisory committee for the QBM-001 development program. Dr. Ventola is a licensed clinical psychologist and an expert in the field of pediatric neuropsychology, with specialization in developmental and genetic disorders. Her clinical and research focus lies in behavioral treatment, Pivotal Response Treatment (PRT), biological markers of treatment response, and dissemination and implementation of treatment for autism spectrum disorders. Dr. Ventola’s recent research includes a study on brain-based mechanisms of treatment response in autism spectrum disorder. She is also utilizing eye-tracking paradigms as a novel outcome of clinical trials in autism spectrum disorder and is involved in studies on novel pharmacological therapeutics.

Dr. Ventola is also the Senior Science Director, Rare Disease and Pediatric Center of Excellence at Cogstate, a publicly traded Australian company. She provides pharmaceutical sponsors with strategic oversight and expert guidance throughout all stages of their study planning and execution—from endpoint selection, rater training and strategic monitoring to final statistical analysis.

“Children with ASD need safe, effective and targeted treatments that are tested under conditions that take their best interests into consideration to ensure a successful trial,” said Dr. Ventola. “I am inspired that Q BioMed has the same vision and am excited to ensure QBM-001 has the best chance to help their targeted subgroup.”

Dr. Ventola received her Doctor of Philosophy in Clinical Psychology from the University of Connecticut and completed her clinical training and Postdoctoral Fellowship at the Yale University School of Medicine, Child Study Center. She serves on the editorial review board of multiple academic journals, including the Journal of Autism and Developmental Disorders, Journal of the American Academy Child and Adolescent Psychiatry, Clinical Psychology Review, and the Autism International Journal of Research and Practice.

The addition of Dr. Ventola to the QBM-001 advisory committee is the latest step in Q BioMed’s systematic, efficient approach to accelerating the development of therapeutics. In December 2018, the company announced a strategic partnership with SRI International to provide formulation development, preclinical development and early clinical manufacturing in support of QBM-001. SRI is an independent, nonprofit research center that has led the discovery and design of innovative products and technologies for government and industry for more than 70 years. Apple’s Siri virtual assistant, online banking, medical ultrasound and cancer treatments are just a few of the advances in which SRI has played a critical role. SRI has broad expertise in solving formulation, drug delivery and characterization challenges of small molecule drugs and biologics.

Q BioMed CEO Denis Corin said, “Dr. Ventola is a leader in clinical trial design and biomarker research for the autism spectrum, which is a perfect fit for the subgroup Q BioMed is targeting. Dr. Ventola will be an asset to our development program as we prepare our Investigational New Drug Application (IND) for submission to the FDA in this important indication for which no drugs are currently available.”

In addition to QBM-001, for which the company expects to begin clinical trials this year, the other near-term catalyst for Q BioMed lies in its palliative care segment. The company acquired the branded Metastron strontium chloride Sr89 from GE Healthcare in November 2018, which provides Q BioMed with ownership of the brand, trademarks and market authorizations of the drug in 22 countries. Metastron is an FDA-approved non-opioid injectable radiopharmaceutical indicated to relieve cancer bone pain in patients with painful skeletal metastases. The drug complements Q BioMed’s existing generic version of Sr89, giving it a powerful foothold in palliative pain care, and the company believes that it also has the potential to improve survival for cancer patients, either as monotherapy or in combination with other therapies.

Q BioMed appears to be well positioned to generate revenue in 2019, while achieving continued operational and clinical milestones across its developmental programs. In addition to its lead autism spectrum and cancer pain relief programs, the company has broadcast anticipated clinical progress in its liver cancer and glaucoma programs, with additional advancements expected to be made this year.

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