Neurotech Pharmaceuticals Inc.’s research efforts are paying off.
The biotech company, focused on developing treatments for chronic eye conditions, received Food and Drug Administration (FDA) approval this month for its cell therapy implant Encelto. The implant could treat idiopathic macular telangiectasia (MacTel) type 2 in adults, a rare neurodegenerative eye disease causing progressive vision loss.
It has taken the company, founded in 2000, more than 14 years to get from clinical trials to approval.
Well-known Orange County ophthalmology executive Jim Mazzo serves as executive chairman of the company, which is based in Cumberland, Rhode Island.
“The approval is a pivotal moment for Neurotech,” Mazzo told the Business Journal. “Not only for Neurotech but for physicians and patients.”
Neurotech expects to begin commercialization of the treatment in June.
Mazzo said that the company is also exploring Encelto’s potential to treat other retinal diseases and has started trials for glaucoma.
Rare Eye Disease
Encelto is a small semi-permeable capsule containing genetically modified retinal cells that is surgically implanted into the eye.
The procedure takes about 10 minutes.
“It’s a single surgical procedure,” Mazzo said. He said they’ve seen patients that have had the implant removed 14 to 15 years later, showing the same amount of drug release as the day it was implanted.
It took more than 20 years to develop the technology, which once implanted, provides sustained drug delivery of therapeutic proteins to the back of the eye.
Other retinal therapies are administered as injections or drops, which can require multiple doctor visits.
Encelto is the first treament of its kind to “address the limitations of intraocular drug delivery to the back of the eye,” according to Mazzo.
MacTel type 2 is a bilateral neurodegenerative disease in adults.
It occurs when blood vessels in the macula, the light-sensitive part of the retina, become dilated and leak, causing central vision loss, according to the Macular Society.
MacTel is an orphan disease affecting a small population.
There are about 160,000 recorded cases in the U.S., according to Mazzo.
The company believes it’s an underdiagnosed disease.
Prior to Encelto, there has been no other FDA-approved treatments for the rare eye disease.
“Overall, it’s not a huge market,” Mazzo said.
Orphan diseases often have less treatments due to not having as large returns for companies, but in Neurotech’s case, the company has received financial support from investors to fund its research.
“We’ve had a great support network to ensure we’ve got this product approved, but that’s very rare,” Mazzo said.
Mazzo didn’t disclose names of the investors nor how much the company has raised to date.
Focused U.S. Launch
Neurotech is planning a focused launch of Encelto to retinal physicians in the U.S., Mazzo said.
One of its planned sites includes University of California, Irvine’s Gavin Herbert Eye Institute, named after the founder of Irvine-based drug maker Allergan.
“It’s in our backyard here in Irvine,” Mazzo said.
In preparation for the launch, the company is scaling up from a production standpoint and educating the audience, Mazzo said.
“We’re creating awareness in the U.S. with the people and doctors that don’t know about it,” he said.
While the company’s manufacturing facilities are in Rhode Island and Boston, it will have sales representatives based in Orange County.
Neurotech is continuing to build out its commercial team.
“We have clinical, we have regulatory, so we’re a fully operational company,” Mazzo said.
