First gene therapy directly infused into the brain to be delivered with ClearPoint’s SmartFlow® cannula
SOLANA BEACH, Calif., May 20, 2022 (GLOBE NEWSWIRE) — ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the “Company”), a global therapeutic platform company enabling navigation and delivery to the brain, today congratulates partnered with PTC Therapeutics on receiving a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending its gene therapy treatment Upstaza™ (eladocagene exuparvovec), formerly PTC-AADC, for the treatment of L-aromatic amino acid decarboxylase deficiency (AADC). Using ClearPoint Neuro’s CE-patented SmartFlow® The neuroventricular cannula for minimally invasive gene therapy infusion is included in the submission for Upstaza administration.
“This is an important milestone for PTC Therapeutics, for ClearPoint and for life biology researchers around the world, as it is the first-ever positive CHMP recommendation for the direct injection into the brain of a gene therapy to treat a serious, highly morbid and fatal neurological disorder,” commented Joe Burnett, President and CEO of ClearPoint Neuro. “This step is extremely important for several reasons. First, it offers a potential way forward for other therapies and sets an important precedent for regulatory approval of gene therapies delivered directly to the brain. And second, we see this as a validation of ClearPoint’s biologics partnership strategy and our rigorous laboratory, preclinical and clinical experience. We believe this announcement will give our 45 existing and future pharmaceutical, academic and biotechnology partners confidence that SmartFlow is able to pass through the necessary regulatory review and can reduce the risks of commercialization efforts for many different drugs to multiple targets. in the brain.
About Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
AADC deficiency is a fatal and rare genetic disease that usually causes severe disability and suffering from the first months of life, affecting all aspects of life – physical, mental and behavioral. The suffering of children with AADC deficiency can be exacerbated by episodes of distressing, seizure-like oculogyric crises, which can occur daily and last for hours, causing rolling eyes in the head, frequent vomiting, behavioral problems, sleep disorders and life disorders. -threatening complications such as respiratory infections and gastrointestinal problems.
There is no approved disease-modifying treatment for AADC deficiency, and the lives of affected children are severely affected and shortened, with the use of many different drugs to help manage symptoms. Ongoing physical therapy, occupational therapy, and speech therapy, as well as interventions, including surgery, to manage life-threatening complications such as infections, severe eating, and breathing problems are also often needed.
About SmartFlow® Cannula
With over 5,000 cannulas sold to date, SmartFlow is the only co-branded device to gain regulatory approval for delivery of approved gene therapy to the brain. The industry-leading cannula is used by many of ClearPoint Neuro’s 45 pharmaceutical, academic and biotechnology partners to bypass the blood-brain barrier and deliver therapeutics to regions of interest using the convection enhanced delivery (CED) under direct image guidance. The SmartFlow cannula has received 510(k) clearance from the FDA for use in the United States for aspiration of cerebrospinal fluid or injection of the chemotherapy drug Cytarabine into the ventricles. It has also been CE marked to deliver approved fluids in brain or CSF aspiration. SmartFlow is used in approved clinical and preclinical studies for various research and drug trials. This device is not intended to be implanted and is intended for single patient use only.
About ClearPoint Neuro
ClearPoint Neuro’s mission is to improve and restore the quality of life for patients and their families by enabling therapies for the most complex neurological disorders with pinpoint precision. Applications of the company’s current product portfolio include deep brain stimulation, laser ablation, biopsy, neuroaspiration, and delivery of drugs, biologics, and gene therapy to the brain. The clear point® Neuro Navigation System is FDA cleared, CE marked, and installed at over 60 active sites in the United States, Canada, and Europe. ClearPoint Neuro is partnered with approximately 45 biologic/pharmaceutical companies and academic centers, providing solutions for the direct delivery of therapeutics to the CNS in preclinical studies and clinical trials worldwide. To date, more than 5,000 cases have been treated and supported by the company’s team of field clinical specialists, who provide support and services to our customers and partners worldwide. For more information, visit www.clearpointneuro.com.
The statements herein regarding the Company’s plans, growth and strategies may include forward-looking statements within the context of federal securities laws. Statements regarding future events, developments and future performance of the Company, as well as management’s expectations, beliefs, plans, estimates or projections regarding the future, are forward-looking statements within the meaning of such laws. Uncertainties and risks may cause the Company’s actual results to differ materially from those expressed or implied by the forward-looking statements. Particular risks and uncertainties include those related to: the impact of the COVID-19 pandemic and the measures adopted to contain its spread; future revenues from sales of the Company’s ClearPoint Neuro navigation system products; the Company’s ability to market, market and achieve broader market acceptance for the Company’s ClearPoint Neuro navigation system products; and the risks inherent in the research and development of new products. More detailed information about these and other factors that could affect the Company’s actual results is described in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2021. , and in the Company’s Quarterly Report on Form 10-Q for the three months ended March 31, 2022, both of which have been filed with the Securities and Exchange Commission.
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