Vitro Biopharma Receives FDA Authorization to Conduct a Clinical Trial Using AlloRx Stem Cells(R) to Treat Pitt Hopkins Syndrome, a rare form of Autism

GOLDEN, CO / ACCESSWIRE / November 17, 2021 / Vitro Biopharma, Inc. announced receipt of FDA authorization and clearance to enroll patients in its IND for a phase I-II, randomized, double-blind, placebo-controlled study of the safety and efficacy of therapeutic treatment with AlloRx Stem Cells® in children with Pitt Hopkins Syndrome (PTHS), a rare neurogenetic disorder that features autistic traits. PTHS results from genetic mutations/deletions of a key brain development gene, TCF4 (transcription factor 4) that controls neurogenesis. Affected children have distinctive facial features and experience moderate to severe intellectual disability, feeding difficulties, delays in reaching developmental milestones, impaired ability to speak, and can have recurrent seizures (40-50%), poor sleep, autistic features, maladaptive behaviors, and breathing pattern abnormalities (reported from age 3-7 years old). The disorder is overwhelming with prolonged episodes of crying and neuro-irritability often reported around 3 years of age. Gastrointestinal concerns are common. Management is symptomatic with early intervention, use of orthotics, anticonvulsive therapies, and psychotropic medications with disappointingly modest improvement. In addition, through the use of AlloRx Stem Cells® in adult populations for other indications, and in children with autism spectrum disorders, there is evidence that support the safety and efficacy of this product.

The FDA clearance of our IND opens an additional pathway to potentially gain FDA pre-market approval of AlloRx Stem Cells® through an Orphan Drug Designation. This is a type of accelerated FDA approval for qualified rare conditions. Our IND-authorized clinical study protocol design is a pivotal study design that when successfully completed could lead to FDA pre-market approval of AlloRx Stem Cells®. This is our second indication for AlloRx Stem Cell® therapy to have received FDA authorization to proceed to enroll patients.

"This is the next step for Vitro Biopharma's efforts in developing successful treatments in the neurologic and inflammatory space. We are excited to have received clearance for this IND, which will enable us to evaluate the safety and efficacy of AlloRx Stem Cells® in PTHS patients in the United States," said Dr. Jack Zamora, M.D., Chief Executive Officer of Vitro Biopharma, Inc. "We look forward to working with investigators to initiate the trial in the United States and meet the needs of patients with PTHS."