FDA Grants Humanitarian Use Device Designation to Bionaut Labs for Treatment of Dandy Walker Syndrome

LOS ANGELES, Sept. 23, 2021 /PRNewswire/ -- Bionaut Labs, a company focused on revolutionizing the treatment of central nervous system (CNS) disorders with its Bionaut™ precision medicine treatment modality, today announced that the U.S. Food and Drug Administration (FDA) has granted the Company humanitarian use device (HUD) designation for BNL-201 for the local treatment of Dandy Walker Syndrome, a rare pediatric neurological disorder.

Dandy Walker Syndrome is a congenital brain malformation usually diagnosed in childhood. It varies in severity and may cause a wide range of symptoms, including intellectual disabilities, delayed motor development, muscle stiffness and partial paralysis, seizures, hearing loss, and vision problems. The only treatment options available today involve invasive intracranial surgical procedures and are prone to infections and complications, and many patients with Dandy Walker Syndrome suffer from shorter life spans with poor quality of life. BNL-201 offers a minimally invasive local microsurgery procedure to fenestrate the cyst that causes this condition and eliminate surgical trauma, to restore patient brain function and development.

"The granting of humanitarian use device designation by the FDA for BNL-101 is an important regulatory milestone for Bionaut Labs and represents a novel treatment modality for Dandy Walker patients and their families," said Michael Shpigelmacher, co-founder and CEO, Bionaut Labs. "This designation is a critical step in getting BNL-201 into the clinic and, ultimately, to those who suffer from this debilitating disease. We look forward to working with the FDA as we pursue our mission of giving new treatment options to patients suffering from this and many other devastating CNS disorders."

A HUD designation is intended to benefit patients in the treatment or diagnosis of a condition that affects less than 8,000 people in the United States per year. The FDA's HUD program is an alternative pathway for getting market approval for medical devices that may help people with rare diseases or conditions like Dandy Walker Syndrome, which affects only one out of every 25,000 to 30,000 people. In June 2021, the Company received orphan drug designation for BNL-101, an investigational treatment modality comprised of the chemotherapy doxorubicin in combination with BNL-201 for the localized treatment of all malignant gliomas.

Aya Jakobovits, Ph.D., appointed to advisory board

Bionaut Labs also announced that Aya Jakobovits, Ph.D., former CEO of Kite Pharma, has joined its advisory board and will serve as the Company's senior corporate advisor. Dr. Jakobovits has a 25-year track record of starting and growing biotech companies that have developed and progressed therapies. 

"Dr. Jakobovits is one of the industry's most respected leaders in targeted therapeutics, and she comes to Bionaut Labs with a decades-long career of developing companies that generate novel technologies and therapeutic products," said Mr. Shpigelmacher. "Her scientific and business-development expertise will be invaluable as we continue to explore the therapeutic potential of Bionauts in treating diseases that lack conventional and successful therapies."

Dr. Jakobovits was president and CEO of Adicet Bio until March 2018. From 2010 to 2013, she was president and founding CEO of Kite Pharma, acquired in 2017 by Gilead for $11.9 billion. She has also served in executive roles at Agensys (acquired by Astellas in 2007 for $537 million) and Abgenix (acquired by Amgen for $2.2 billion).

Dr. Jakobovits serves on the Board of Directors of Dorian Therapeutics, Adicet Bio, Dyve Biosciences, UCLA Technology Development Corporation and Yeda Research and Development. She received her bachelor's degree from Hebrew University of Jerusalem and her master's in chemistry and Ph.D. in life sciences from the Weizmann Institute of Sciences in Israel. Dr. Jakobovits was a postdoctoral fellow at the University of California San Francisco and Genentech, has authored more than 50 scientific publications, and is the inventor of more than 120 issued patents.

About Bionaut™ Treatment

A Bionaut is a novel treatment modality that uses remote-controlled microscale robots to deliver biologics, nucleic acids, small molecule, gene or cellular therapies locally to targeted CNS disease areas. Through precise targeting, Bionaut™ therapeutics could offer better efficacy and safety that cannot be achieved by other traditional treatment or delivery modalities.

Bionauts can be constructed in different designs with custom geometries and surface characteristics. Smaller than a millimeter, they contain moving parts controlled remotely by a magnetic controller, allowing them to safely reach the target and release a therapeutic payload from the cargo compartment. Engineering flexibility provides a broad foundation for designing Bionaut therapies for nearly any disease of interest.

Bionaut Labs has demonstrated safe and controlled navigation of its therapeutic Bionaut to and from the treatment locus in the brain, in a large animal in vivo model. Furthermore, the Company has successfully treated human glioma tumors established in mice, utilizing guided delivery of therapeutic cargos directly into these tumors to eliminate systemic toxicity. These results pave the way to the clinical trials of the Bionaut platform.

About Bionaut Labs

Bionaut Labs is focused on revolutionizing the treatment of central nervous system disorders with its Bionaut precision medicine treatment modality that uses remote-controlled microscale robots to deliver biologics, nucleic acids, or small molecule therapies locally to targeted disease areas. With the Bionaut medical modality, Bionaut Labs can transform the way the biopharmaceutical industry develops treatments, allowing the Company to engineer the therapeutic index for better efficacy and safety. Led by a world-class team of medical, engineering, and drug development experts, Bionaut Labs' headquarters is in Los Angeles, California, with additional R&D sites in Israel and at the Max Planck Institute in Germany. For more information, please visit www.bionautlabs.com.

SOURCE Bionaut Labs