Direct Anatomic Delivery of Brain Therapeutics OTCQB: ACXA Company Presentation January 2015 – San Francisco, CA
Note on Forward-Looking Statements Statements made in this presentation that are not historical facts are forward-looking statements that involve risks and uncertainties. The inclusion of forward-looking statements, including those related to the ability of the Company to perform per its license agreement with UCSF and meet its obligations; the ability of the Company to successfully develop and commercialize the BranchPoint device and execute its business plan; the business strategy, plans, and objectives of the Company; and any other statements of non-historical information should not be regarded as a representation that any of our plans will be achieved. Actual results may differ materially from those described in this presentation due to the risks and uncertainties inherent in our business, including, without limitation, risks and uncertainties related to: our research and development efforts, including pre- clinical and clinical testing; regulation by the FDA and other government agencies; the timing of regulatory applications and product launches; our ability to successfully commercialize our products; and other risks detailed in our filings with the Securities and Exchange Commission filings, including our most recent Annual Report on Form 10-K and our most recent Quarterly Report on Form 10-Q. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. We undertake no obligation to revise or update this presentation to reflect events or circumstances that occur after the date of this presentation. Accurexa Inc. 2
Mission Our Mission is to develop and commercialize novel neurological therapies based on our proprietary BranchPoint technology delivering therapeutics directly into specific regions of the brain, to the benefit of our patients and shareholders. Accurexa Inc. 3
Investment Thesis Summary • Licensed global exclusive rights to the BranchPoint technology from UCSF with a working commercial prototype • 510(k) FDA application for BranchPoint expected to be filed in the first half of 2015 • Complete Product Development Team: Bioengineering, Manufacturing, Quality Control, Regulatory • Strong Scientific Advisory Group from UCSF, incl. neurosurgery KOLs • Revenue opportunity: Product sales of BranchPoint to clinical stem cell trials upon FDA approval • Brain Cancer program: Direct anatomic delivery of chemotherapeutics to brain tumor site • Stem Cell program: Delivery of dopamine producing, stem cell-derived neurons for the treatment of Parkinson’s UCSF: University of California, San Francisco Accurexa Inc. 4 KOLs: Key Opinion Leaders
Management and Board Management • George Yu, President & CEO – over 15 years of experience in technology start-ups, investment banking and management consulting. Previously CEO of Sinobiomed, a biopharmaceutical company focused on developing genetically engineered recombinant protein drugs and vaccines. Co-founded Bay2Peak, a financial advisory and investment management firm that executed strategic partnerships between RxD Pharmaceuticals and Pfizer, and Sinobiopharma and Lupin Pharmaceuticals. Bain, Lehman Brothers. MBA Columbia Business School, Medical Doctor University of Tuebingen, Germany. Board of Directors • Anchie Kuo – Physician, venture capitalist and entrepreneur with over 25 years of experience in healthcare. CEO of Arc Medical, first FDA registered suture manufacturer in China, Genaissaince Capital, BankAmerica Ventures, Pfizer. BA Dartmouth, MD Dartmouth Medical School. • William Callahan – more than 25 years in emerging growth companies in diagnostics, medical devices and drug delivery, establishing GMP manufacturing operations, support of regulatory FDA submissions, new product launches. Avocet Medical, Optiscan BioMedical, Cygnus Therapeutics, Powderject Technologies, Applied Biosystems, J&J Lifescan, Depomed. BS in Chemistry San Francisco State University. Accurexa Inc. 5
Advisory Group Scientific and Clinical Advisors • Dr. Daniel Lim, Assistant Professor of Neurological Surgery, UCSF – Lead inventor of BranchPoint with over 18 years of basic and clinical research experience in neural stem cell therapies, brain tumors and stereotactic neurosurgery. Co-surgeon in Stem Cells Inc.’s Phase 1 clinical trial of neural cell transplantation to PMD patients. • Prof. Krys Bankiewicz, Vice-Chair of Neurological Surgery, UCSF – over 20 years experience with convection enhanced delivery (CED) of therapeutics to brain tumor and Parkinson’s disease patients • Dr. Alastair Martin, Adjunct Professor Radiology and Biomedical Imaging, UCSF - over 20 years experience in MR imaging, pioneered MRI implantation system for the delivery of deep brain stimulator electrodes and administration of therapeutics Product Development and Regulatory Advisors • Prof. Tejal Desai, Chair Bioengineering and Therapeutic Sciences, UCSF – Co- Inventor of BranchPoint, developed implantable drug delivery devices, implantable biohybrid devices for cell encapsulation, templates for cell and tissue regeneration • Greg Mathison, Regulatory, Clinical and Quality Assurance – 35 years experience in regulatory, clinical and quality compliance management positions at Medtronic, American Medical Systems, Cardiac Pacemakers, Clarus Medical and Bacchus Vascular Accurexa Inc. 6
The Problem: Therapeutic Delivery to the Brain Device used for cell delivery today Gupta, et al, Sci Transl Med , 2012 Accurexa Inc. 7
Multiple straight penetrations with limited anatomic targeting risk increased trauma and reduce efficacy Each penetration risks 9 penetrations for the putamen hemorrhagic stroke (each hemisphere) Accurexa Inc. 8
BranchPoint enables delivery to large, complex targets through a single penetration • Platform technology that enables radially branched deployment (RBD) of cells to multiple target locations at variable radial distances and depths along the initial brain penetration tract • RBD overcomes some of the technical limitations inherent to the use of straight cannulas iMRI-guided RBD. Targeting is performed with intraoperative MRI scans, which • This platform technology could enables corrections for brain “shift,” and delivery can be monitored in “real time” with rapid MRI scans. By controlling guide cannula radial angle and depth have a major impact on the clinical and delivery catheter radial distance, cells (schematized as green and purple circles) can be delivered to larger brain target volumes that vary in shape and translation of a wide range of cell size due to individual patient anatomy and different disease states. therapeutics Silvestrini, et al, Mol Therapy, 2014 Accurexa Inc. 9
iMRI-guided BranchPoint: Leveraging advances in material sciences, modern imaging and neurosurgery • Integrated RBD with FDA-approved iMRI skull- mounted aiming device and targeting software. • “Real - time” MRI monitoring that is easy to implement in most hospitals worldwide addresses shortcomings inherent to standard stereotaxy • Enables delivery “tailored” to individual patient anatomical variation and different disease states • “Piggybacking” can reduce patient risk and the cost of procedures for a wide variety of therapeutics for brain therapy Accurexa Inc. 10
BranchPoint technology with intuitive, simple controls (a) Guide cannula with distal side port (white arrow, left) and proximal controls (right). (b) Delivery catheter with dual side infusion ports (not seen at this magnification) at the curved distal end (left) and the proximal locking hub (right). A plunger wire is fitted into the catheter lumen. (c) Positioning collet. Orange arrows indicate the rotational (radial angle) control provided by the white hub (top) and depth control provided by the yellow wheel (right). Horizontal markings (black and red lines, mm spacing) indicate changes in collet depth. Scale bars = 5 mm. (d) RBD use illustrated with a human skull model. View is from the posterior right. The RBD platform was mounted over a right frontal burr hole. (1) Attachment of positioning collet to the MRI- compatible, skull mounted aiming device; (2) insertion of guide cannula through the positioning collet; (3) insertion of delivery catheter with the proximal hub locking into the guide cannula proximal controls (white arrowhead); (4) deployment of the catheter through the opened guide cannula side port; (5) delivery of cells with plunger wire; (6) removal of delivery catheter and closure of the guide cannula side port; (7) repositioning of guide cannula side port to new depth and radial angle; (8) delivery to new location via repetition of (3 – 7). Accurexa Inc. 11
BranchPoint works at the scale of the human brain ( A ) Before SPIO delivery. Arrow indicates the region of the putamen, which is shown enlarged and outlined in the inset at the lower right. ( B,C ) After six deliveries of SPIO (numbered in red) via a single guide cannula insertion. The two panels show different axial planes to illustrate the different radial and depth locations of the SPIO deposits. ( D ) Enlarged view of the putamen (red outline) with the regions of the SPIO deposits (blue outline). White scale bars = 1 cm. iMRI, interventional magnetic resonance image; RBD, radially branched deployment; SPIO, super paramagnetic iron oxide. Accurexa Inc. 12
Recommend
More recommend
