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AGTC Cleans Up with $37.5M Series B; It's Good 'Eye Gene'


By Randy Osborne
Staff Writer

Riding the wave of success at last for gene therapy, privately held Applied Genetic Technologies Corp. (AGTC) bagged $37.5 million in a Series B financing that will let the company finish two Phase II trials with separate drug candidates, and see the firm through a pair of new, Phase I/II studies with other compounds in orphan eye diseases.

Sue Washer, president and CEO of Gainesville, Fla.-based AGTC, noted that monoclonal antibodies took 25 years to ripen, and gene therapy is nearing the end of a similar timeline.

Such is the road "for brand new, cutting-edge technologies to really come into their own," she said. "Everyone is very well convinced [that gene therapy is] a safe way to treat patients. Certainly, in the last 18 to 24 months, there's been good news after good news in the gene-therapy space."

The company recently finished enrolling a Phase II trial in Leber's congenital amaurosis (LCA), a form of childhood blindness. "We're just at the point of collecting data," Washer said. (See BioWorld Today, May 4, 2009.)

Another Phase II study is under way in alpha-1 antitrypsin deficiency. Patients with that condition "are missing a protein that protects their lungs, [which] start being degraded at a very young age, and they develop emphysema and chronic obstructive pulmonary disease-like symptoms in their early 20s."

Two more orphan indications will be the subjects of Phase I/II investigations. Achromatopsia is caused by mutations in a group of genes which make the cone cells concentrated in the central retina nonfunctional.

"All of those patients are legally blind and have severe photophobia," Washer said. AGTC exploits adeno-associated virus to deliver healthy copies of genes to replace the flawed ones.

The other Phase I/II trial, in X-Linked rentinoschisis, will explore the gene therapy approach against a genetic disease in which the afflicted are missing a structural protein, "which means the layers of their retina delaminate from each other," Washer told BioWorld Today. "None of those patients can drive. Their vision is quite poor."

A scientific founder of the company was interested in the alpha-1 antitrypsin space, and others wanted to tackle the rare eye diseases. "We also did a partnership with Genzyme [Corp., now a part of Sanofi SA] working on a product for age-related macular degeneration," which provided further expertise in eye conditions, Washer said.

"We are no longer in a partnership with them," she said. "Once the product reached the clinic, we fully licensed the program out to Genzyme and they're in complete control moving it forward." Cambridge, Mass.-based Genzyme is testing an anti-VEGF compound called AAV2-sFLT01 in a Phase I trial that will enroll about 34 patients.

Gene therapy, "on the cusp of being successful," as Washer put it, has proved encouraging to the point that labs have begun testing not only in life-threatening diseases, but those with less dire potential outcomes. (See BioWorld Today, Nov. 12, 2012.)

This month, Glybera (alipogene tiparvovec) became the first gene therapy to be approved in a regulated market, when the European Commission gave its nod to the compound from uniQure BV, of Amsterdam, the Netherlands. Glybera was developed to treat the genetic disorder lipoprotein lipase deficiency. (See BioWorld Today, Nov. 7, 2012.)

Washer said AGTC will decide later how to proceed, once the proof-of-concept data are in hand. "It will depend entirely on whether we have multiple programs with positive data, or only one or two," she said. Success across the board means the company would "complete Phase III trials in a couple of programs and partner out the others. "If we only had one or two programs that were successful, we might consider selling those off and having an exit at that point," she said.

Alta Partners and S.R. One Ltd. led the financing. New investor Osage University Partners joined existing investors InterWest, Intersouth Partners and MedImmune Ventures in the round.

In other financings news:

• Compugen Ltd., of Tel Aviv, Israel, signed an agreement with a private U.S.-based investment company pursuant to which up to $15 million in milestone-related equity financing will be made available to Keddem Bioscience Ltd., a wholly owned subsidiary of Compugen. That financing will be used to further develop and commercialize a technology platform that has been designed to enable the discovery of small-molecule modulators (drugs and pesticides) for potentially any given protein target. Under the agreement, the new investor will obtain a majority equity interest in Keddem, with Compugen maintaining a minority interest and certain future preferential access rights to utilize the Keddem technology with Compugen-discovered drug targets.