Taysha, Prothena, Pipeline and Neuroplast Launch Clinical Trials

neurological disorder

Neurological diseases are notoriously difficult to treat for many reasons, including the complexity of the diseases and the difficulty of getting therapies past the blood-brain barrier. Four companies announced launches of clinical trials in the CNS space today. 

Prothena's PRX-12 for Alzheimer's Disease


The U.S. Food and Drug Administration cleared Prothena's Investigational New Drug Application for a Phase I trial. It will begin the ascending dose trial around mid-year PRX-12 for Alzheimer's disease. The drug is a potential best-in-class anti-amyloid beta antibody designed to allow subcutaneous dosing on what the company describes as a "patient-friendly, convenient administration schedule."

Preclinical data have demonstrated that the drug binds to beta-amyloid plaques and oligomers. Clinically achievable levels of clearance of both pyroglutamate modified and unmodified amyloid-beta plaque in brain tissues were possible.

"With Alzheimer's affecting more than 50 million people worldwide, we are committed to bringing a paradigm-shifting treatment to patients as quickly as possible," Gene Kinney, Ph.D., president and chief executive officer of Prothena, said.

Pipeline's PIPE-307 in Relapsing-Remitting Multiple Sclerosis

Pipeline Therapeutics reported the FDA had cleared it to launch a Phase Ib/IIa trial of PIPE-307 in patients with relapsing-remitting multiple sclerosis (RRMS). PIPE-307 is an oral, highly selective antagonist of the muscarinic M1 receptor, the company's lead program for myelin restoration. In a recently completed Phase I trial in healthy volunteers, data from the PET study demonstrated the tested doses hit a level of uptake in the human brain associated with remyelination seen in preclinical studies.

"FDA clearance to initiate our Phase Ib/IIa clinical study of PIPE-307 is a major regulatory milestone for Pipeline, as it allows us to proceed with the evaluation of PIPE-307 in RRMS patients," Carmine Stengone, president and chief executive officer of Pipeline, said. "Today, approved medicines for M.S. patients are focused on immune modulation but do not address the fundamental M.S. disease pathology that leads to long-term decline in neurological function - chronic demyelination. Our objective with PIPE-307 is to develop the first effective treatment that can restore myelin and improve outcomes for patients."

Neuroplast's Neuro-Cells for Traumatic Spinal Cord Injury

Neuroplast, headquartered in Geleen, Netherlands, enrolled the first patient in its Phase II trial of Neuro-Cells for acute Traumatic Spinal Cord Injury (TSCI). It is being run in collaboration with Hospital Nacional de Paraplejicos in Toledo, Spain. Neuro-Cells contains non-substantially manipulated bone marrow-derived hematopoietic and mesenchymal stem cells. They are generated from a patient's bone marrow, meaning the donor and recipient are the same person. They remove inflammatory-inducing components and pathogens during the production process.

"The start of this Phase II trial marks another important milestone in our mission to bring back perspective to people who suffer from neurodegenerative diseases for which no effective treatments are available," Johannes de Munter, M.D., chief executive officer of Neuroplast, said.

Taysha's TSHA-102 for Rett Syndrome

Taysha Gene Therapies initiated the clinical development of TSHA-102 for Rett syndrome. The company is based in Dallas, Texas, but the study will be conducted at Sainte-Justine Mother and Child University Hospital Center in Montreal, Quebec. TSHA-102 is a self-complementary intrathecally delivered AAV9 gene replacement therapy. It uses a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform to regulate transgene expression genotypically on a cell-by-cell basis. The therapy has received rare pediatric diseases designation and orphan drug designation from the FDA and orphan drug designation from the European Commission.

Rett syndrome is a severe genetic neurodevelopmental disorder. It results from a mutation in the X-linked MECP2 gene, which is necessary for neuronal and synaptic function in the brain. It primarily affects females and is one of the most common genetic causes of severe intellectual disability around the globe.

"Initiation of clinical development is a significant milestone for the TSHA-102 program and Rett syndrome community," Suyash Prasad, M.D., chief medical officer and head of Research and Development for Taysha, said. "Treating Rett syndrome by gene replacement therapy requires an approach that can safely regulate transgene expression in a genotypic manner on a cell-by-cell basis without causing deleterious effects associated with overexpression."

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