Download AAVP Biosystems, Inc.

AAVP Biosystems, Inc.
Maximizing Efficacy and Minimizing Toxicity Using Targeted Therapeutics
DESCRIPTION:
AAVP Biosystems (“AAVP”) is an early stage biotechnology
company developing a targeted gene delivery vector that aims to
direct therapy selectively to tumor cells while sparing normal cells
and tissue. The core technology is fully and exclusively licensed from
MD Anderson Cancer Center, and is undergoing IND-enabling
studies in preparation for a late-2015 FDA filing.
TECHNOLOGY:
Adeno-associated virus phage or AAVP, is a hybrid vector platform
developed by combining the targeting capability of bacteriophage
and the sustained gene expression of adeno-associated virus.
Bacteriophage is a virus that infects bacteria, but cannot infect
human cells and has been used clinically for decades as an antimicrobial agent. AAVP has successfully developed a version of
bacteriophage that can infect, but not replicate, in specific human
cells. That specificity can be tailored to a cell of choice, such as a
tumor cell or tumor associated cell. Normally, if a bacteriophage
infects a human cell, no gene expression takes place. However,
AAVP’s hybrid AAVP can express a gene product in a human cell
based on the fact that it carries the gene expression machinery of an
adeno-associated virus, but not the adeno-associated virus itself.
This avoids the limitations well known to adeno-associated virus
including the need for a helper virus and robust immunogenicity
limiting clinical efficacy.
The result is a platform that can be tailored to target any human cell
and express exclusively in that cell, any gene of interest. The
company has generated AAVP that infect a variety of cell types and
express a variety of gene products, both for therapy and for imaging.
AAVP’s lead therapeutic, RGD-AAVP-TNF, is designed to selectively
infect tumor associated blood vessels and express TNF exclusively
in the tumor vascular bed resulting in tumor necrosis. The company
has validated this tumor targeting and selective gene expression, as
well as therapeutic efficacy and absence of toxicity, in small and large
animals. In fact, RGD-AAVP-TNF has been administered in the
context of two veterinary clinical trials in dogs demonstrating safety
and anti-tumor activity. AAVP has generated substantial pre-clinical
data on stability, dosing and lack of toxicity and is readying to submit
an FDA IND filing in preparation for a first in human clinical trial in
patients with advanced stage malignancies.
DEVELOPMENT PLAN:
AAVP’s next steps are producing cGMP grade RGD-AAVP-TNF,
completing an IND filing for the FDA, and initiating a Phase I clinical
trial. AAVP has identified organizations capable of performing the
cGMP production, assist with IND required toxicity studies, and build
the IND package. IND-enabling projects will run through 2015, at
which point the company anticipates requesting a pre-IND meeting.
In late-2015, AAVP will focus on raising a round of capital, between
$6 and $8 million, followed by the filing of the IND request to accrue
the planned dose escalating Phase I clinical trial. In 2016, upon
closing of the second capital raise, the company anticipates the start
of enrollment of a ~30 patient first in human study, which should
conclude in 2017.
To accelerate IND-enabling activity, AAVP is in the process of leasing
incubator research space at the Los Alamos National Laboratory.
This fully-equipped research facility will house our key scientific
advisors, as well as two research associates. This facility provides
the Company with a greater level of control over its research
endeavors at a greatly reduced cost than what would be required if
this research was performed by contracted research organizations or
in university
laboratories. Furthermore, with two of the Company’s key scientific
officers overseeing this research, the risks of erroneous or mistargeted data generation is greatly reduced.
FINANCING:
AAVP has raised over $600 thousand in initial seed funding, expects
to close on the first part of a $3 million Series A round in early-2015,
and the remainder of which it aims to close upon mid-year. Funding
has been sourced from institutional investors and high-net worth
family offices focused on early stage biotechnology companies.
Cash needs through 2018 are approximately $11 million. These
funds will support development through a first in human clinical trial.
Over the next 18 months it is projected that $3 million of this total will
be allocated to cGMP production, regulatory consultants, and
support for the filing of an IND. The remaining funds are projected to
carry through 2018 to support the completion of the Phase I clinical
trial.
KEY PERSONNEL:
Jason Rifkin, J.D., M.S., President & CEO has managed all
aspects of early stage biotechnology company operations. Prior to
his role at AAVP, Jason was President of PreScience Labs, an
early stage oncology company where he oversaw the build-up of a
Phase I clinical trial program. Prior to joining PreScience, Jason
held a number of executive clinical and commercial operations
positions at Delcath Systems, Inc., a specialty pharmaceutical and
medical device company focused on oncology.
Timothy Rothwell, J.D., Chairman of the Board is also the
Chairman of Emisphere Technologies Inc. and prior to that a
Director of the Company. Mr. Rothwell is also Chairman of
Speracura LLC, a healthcare consulting company and Chairman of
the Pheo-Para Alliance. Mr. Rothwell was formerly Chairman of
Sanofi in the U.S. from February 2007 to October 2009 and served
as Chief Executive Officer and President of Sanofi-Aventis from
September 2004 to February 2007.
Steven Libutti, M.D., Founder and Director is the Director of the
Montefiore-Einstein Center for Cancer Care, Professor and ViceChairman of the Department of Surgery and Professor in the
Department of Genetics at the Albert Einstein College of Medicine
and the Montefiore Medical Center. Dr. Libutti is Editor-in-Chief of
the Nature Journal Cancer Gene Therapy.
Renata Pasqualini, Ph.D., Founder is Associate Director for
Translational Research at the University of New Mexico Cancer
Center and co-leader of the UNM Cancer Center’s Program in
Experimental Therapeutics and Drug Discovery. Dr. Pasqualini is
also Professor in the Department of Internal Medicine at the
University of New Mexico School of Medicine.
Wadih Arap, M.D., Ph.D., Founder is the Deputy Director of the
UNM Cancer Center and Chief of the Division of Hematology and
Oncology in the Department of Internal Medicine in the University of
New Mexico School of Medicine.
CONTACT INFORMATION
Jason Rifkin
T: (917) 733-1542 Fax: (914) 723-1419
E-mail: [email protected]
Website URL: www.aavpbiosystems.com