Portfolio News
AGTC raises $11.8M from VCs to help gene therapy programs
BioWorld Today
Catherine Hollingsworth
4 May 2009
Applied Genetic Technologies Corp. has raised $11.8 million in a venture-funded financing round, money to advance the company's gene therapy programs.
The financing will be used to complete the next phase in human clinical trials for Alpha-1 antitrypsin deficiency Alpha-1, a form of emphysema, and Leber's congenital amaurosis (LCA), a form of childhood blindness.
Currently, the company is enrolling patients for an investigational new drug study for the LCA drug candidate and the emphysema drug candidate has completed a safety study and will initiate an efficacytrial in the first quarter of 2010.
The financing will advance the two clinical programs through efficacy studies, said Susan Washer, CEO and president of AGTC. The money also will be used for preclinical development of AGTC's gene therapy treatment for achromatopsia, a form of daytime blindness. The preclinical product, she said, is expected to enter the clinic in 2011.
Privately held AGTC, which was spun out of the University of Florida long ago, has raised $39.3 million to date, and will be seeking partners to further advance its programs in the future.
Other than some limited grant funding, Gainesville, Fla.-based AGTC has no other sources of revenue, Washer said. "We are very excited to be moving on to the efficacy trials at this point," she told BioWorld Today.
The financing was led by led by InterWest Partners. Other participants in the round include Intersouth Partners and MedImmune Ventures Inc. Those same investors, along with Skyline Ventures, participated in the company's last financing round in November 2003, which brought in $27 million.
In 2007, AGTC received a $2 million milestone payment from Genzyme Corp. as a result of having successfully transferred AGTC's proprietary adeno- associated virus (AAV) vector production technology to Genzyme. Under the collaboration, Genzyme gained access to AGTC's vector production expertise along with rights to certain intellectual property for the use of AAV vectors to deliver genes to patients.
AGTC uses a modified version of the naturally- occurring adeno-associated virus to deliver genes to patients. The virus has never been associated with illness in humans and more than 50 percent of the population is exposed normally to this virus. Other firms working on AAV-based therapies include Genzyme and Targeted Genetics Corp., both of which are studying different diseases than AGTC.
The Foundation for Fighting Blindness welcomed the funding for AGTC's pipeline therapies for blindness.
Stephen Rose, chief research officer for the foundation, said in a statement that the venture funding extends support from the foundation's National Neurovision Research Institute for the company's Phase I LCA trials. In addition, he said, "the support for preclinical studies for achromatopsia provides even greater potential for gene therapy to treat and cure these retinal degenerative diseases that often do not receive the attention or investment necessary to bring promising treatments to patients."
LCA is a hereditary defect that causes early onset blindness in children.
Previous trials have shown that AGTC's LCA product to be well tolerated with promising signs of efficacy. That product will be further evaluated in a dose escalation study designed to identify an optimally effective treatment regimen.
Alpha-1, a hereditary defect that causes early onset emphysema in adults, is the most common potentially lethal hereditary disease of U.S. and Northern European adults. There are about 130,000 symptomatic patients in the U.S. and Europe.
Previous human trials have shown the product to be well tolerated and can provide sustained expression of the therapeutic protein. The next planned clinical study will determine if therapeutic levels of alpha-1 anti-trypsin can be achieved by increasing the dose of AGTC's alpha-1 product administered to patients.
In other financing news, Oncolytics Biotech Inc., of Calgary, Alberta, entered an engagement agreement with Bolder Investment Partners Inc. in connection with a proposed equity financing. The offering will consist of up to 3 million units at $2 per unit, for gross proceeds of up to $6 million. Bolder will be granted an over-allotment option to purchase up to an additional 450,000 units at the same price. The offering is expected to close the week of May 11. Proceeds will be used for clinical trials, manufacturing activities in support of the trial program and for general corporate purposes. Oncolytics' clinical program includes a variety of Phase I/II and Phase II trials using Reolysin, its proprietary formulation of the human reovirus, alone and in combination with radiation or chemotherapy.
