UCSF and Accurexa Receive Response From FDA to Pre-IND Meeting Request

NEWARK, DE / ACCESSWIRE / March 24, 2016 / the "Company" or "Accurexa") ACXA, a biotechnology company focused on the development of novel neurological therapies to be directly delivered into the brain, announced today that the University of California, San Francisco ("UCSF") received a written response from the FDA (U.S. Food and Drug Administration) regarding a pre-IND meeting request to discuss a collaborative program between UCSF and Accurexa to evaluate a combined stem cell/gene transfer candidate therapeutic CNS10-NPC GDNF for the treatment of Parkinson's Disease using Accurexa's BranchPoint delivery device. CNS10-NPC are human fetal neural stem cells that express Glial-Derived Neurotrophic Factor (GDNF) in the treatment of Parkinson's Disease, and are expected to be supplied by the Cedars-Sinai Regenerative Medicine Institute in support of IND (Investigational New Drug) enabling studies.

The FDA provided guidance on the non-clinical activities that are necessary to enable the filing of a future IND application.

"The FDA provided valuable feedback that will help us to refine our development plan and we look forward to working with UCSF on this important program to develop a potential treatment for Parkinson's Disease," said George Yu, MD, Accurexa's President & CEO.

Glial-Derived Neurotrophic Factor (GDNF) is a potent trophic factor for dopamine neurons and a candidate trophic therapy for Parkinson's Disease. Although controversy surrounds the clinical use of GDNF, its neuroprotective and regenerative effects in animal models of Parkinson's Disease have been independently corroborated by several laboratories. Like other trophic factors, GDNF requires intracerebral site-specific delivery to be effective and decrease the risk of side effects.

About UC San Francisco

UCSF is the nation's leading university exclusively focused on health. Now celebrating the 150th anniversary of its founding as a medical college, UCSF is dedicated to transforming health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. It includes top-ranked graduate schools of dentistry, medicine, nursing and pharmacy; a graduate division with world-renowned programs in the biological sciences, a preeminent biomedical research enterprise and two top-tier hospitals, UCSF Medical Center and UCSF Benioff Children's Hospital San Francisco. Additional information may be found on www.ucsf.edu.

About Accurexa, Inc.

The Company is focused on developing novel neurological therapies to be directly delivered into specific regions of the brain. Its BranchPoint device was invented to deliver multiple therapeutics, such as stem cells, chemotherapy or gene therapy vectors, through the radial deployment of a flexible catheter to specific brain target areas through a single brain penetration, and licensed from UCSF (University of California, San Francisco). The current standard of care is the use of straight, rigid needles, often requiring surgeons to penetrate the brain multiple times for delivering therapeutics, which in turn may increase the risk of bleeding, stroke and reflux of therapeutics back out to the brain surface. An animal study at UCSF(1) demonstrated that the use of a straight needle was associated with reflux of at least 75% of the infusion while no reflux was found with the BranchPoint device. The development of the BranchPoint device was originally funded with $1.8 million by California's Stem Cell Agency CIRM (California Institute of Regenerative Medicine). A 510(k) application was submitted to the FDA in June 2015.

The Company is also developing its ACX-31 program to deliver two chemotherapy drugs, temozolomide in combination with BCNU, locally to brain tumor sites. The ACX-31 program is based on an issued patent licensed from Accelerating Combination Therapies LLC which is co-owned by Prof. Henry Brem, Director of the Neurosurgery Department at Johns Hopkins University. The Company is collaborating in the development of its ACX-31 program with Prof. Henry Brem who built one of the largest brain tumor research and treatment centers in the world at Johns Hopkins University. Prof. Robert Langer, who is the David H. Koch Institute Professor at MIT and the most cited engineer in history, is also advising the Company in the development of its ACX-31 program.

Additional information about the Company may be found on its website, www.accurexa.com.

Notes:

(1) Silvestrini MT, Yin D, Coppes VG, Mann P, Martin AJ, Larson PS, Starr PA, Gupta N, Panter SS, Desai TA, Lim DA. Radially branched deployment for more efficient cell transplantation at the scale of the human brain. Stereotact Funct Neurosurg. 2013;91(2):92-103.

Safe Harbor Statement

This release contains certain "forward-looking statements" relating to the business of the Company. All statements, other than statements of historical fact included herein are "forward-looking statements" including statements regarding: the ability of the Company to successfully develop and commercialize novel neurological therapies based on its BranchPoint device or U.S. Patent No. 8,895,597 B2, or its ACX-31 program and execute its business plan; the business strategy, plans, and objectives of the Company; and any other statements of non-historical information. These forward-looking statements are often identified by the use of forward-looking terminology such as "believes," "expects" or similar expressions and involve known and unknown risks and uncertainties. Although the Company believes that the expectations reflected in these forward-looking statements are reasonable, they do involve assumptions, risks, and uncertainties, and these expectations may prove to be incorrect. Investors should not place undue reliance on these forward-looking statements, which speak only as of the date of this news release. The Company's actual results could differ materially from those anticipated in these forward-looking statements as a result of a variety of factors, including those discussed in the Company's periodic reports that are filed with the Securities and Exchange Commission and available on its website (http://www.sec.gov). All forward-looking statements attributable to the Company or persons acting on its behalf are expressly qualified in their entirety by these factors. Other than as required under the securities laws, the Company does not assume any duty to update these forward-looking statements.

Contact Investor Relations:

David Burke
The Ruth Group
Tel.: 646-536-7009
dburke@theruthgroup.com

Accurexa Inc.
info@accurexa.com
Tel.: 302-709-1822

SOURCE: Accurexa Inc.