Phase III study for new imlpant puts novel treatment approach in line for FDA review
Clinical scientists from the Emory Eye Center are applauding the recent release of results from the Phase III clinical trials for a novel approach to macular telegniectasia-2 (MacTel), a disease that causes the gradual deterioration of central vision.
Emory Eye Center retinal surgeon Jiong Yan, MD has served as the principal investigator for the ECT and NT-501 clinical trials for more than a decade.
Official announcement of the results was made last month by Neurotech Pharmaceuticals, Inc., during a meeting of the MacTel Study Group held in Israel. The positive topline results mean the investigational implant, NT-501, and an associated encapsulated cell therapy (ECT) can now be reviewed by the FDA.
The multicenter, randomized, sham-controlled studies evaluated the safety and efficacy of the surgically implanted NT-501, a tiny hollow cylindrical membrane encapsulating genetically engineered human epithelial cells that continuously produce ciliary neurotropic factor (CNTF), a protein now clinically validated to slow the progression of MacTel.
After a long period of intensive research, development, and clinical trials, we are extremely gratified with our Phase 3 results,
said Thomas Aaberg Jr., MD, chief medical officer of Neurotech.
The recently completed studies demonstrated statistical significance for the pre-specified primary endpoint, rate of change in ellipsoid zone (EZ) area loss from baseline through 24 months. The rate of change in area loss translates to and demonstrates a change in the rate of photoreceptor loss.
These findings are an important step towards giving hope to those currently suffering with unrelenting vision loss and who don't have a way of stopping the progression of the disease today. We look forward to engaging with the FDA throughout the review process,
said Richard Small, chief executive officer of Neurotech.
Dr. Yan's original investigations focused on the CNTF implant’s efficacy as a treatment for retinitis pigmentosa and atrophic AMD. When researchers later decided to evaluate its impact in MacTel patients, Yan was tapped to perform the first surgical implantation, due to her vast experience with the device.
The success of that surgery prompted additional interest in ECT as a potential treatment for MacTel - a finding that launched the recently completed suite of clinical trials.
Emory has played a pivotal role in the advancement of this trial and this approach. We were able to set the surgical standards and even created a video that was used to train other surgeons,
she said.
I think Emory Eye Center was chosen as the site for that first implant because of our outstanding reputation as a clinical trial site and our longstanding commitment. We had an incredible team that worked together - like [former EEC Chair Thomas M. Aaberg, Sr., MD], former retina section director Dr. Dan Martin. , current retina section director Dr. Baker Hubbard. , and clinical coordinators like Donna Leef. who did an enormous amount of work behind the scenes on this- and on many other trials.
So, yes, it is a tremendous honor for me to have worked on something that could potentially be impactful and a game-changer. But I also know that the honor goes to a team of collaborators in the department who worked hard on this.
The positive study results are good news for MacTel II patients. Pending FDA approval, the new treatment could provide a meaningful tool for slowing the progression of their vision loss.
About Macular Telangiectasia Type 2
Macular telangiectasia type 2 (MacTel), or idiopathic juxta foveal macular telangiectasia type 2, is a rare neurodegenerative disease with characteristic alterations of the retinal vasculature and localized retinal degeneration.1 The prevalence of MacTel ranges from 0.02% - 0.06% 2,3 with most patients diagnosed in their 40s and 50s.4 MacTel typically affects both eyes and causes a gradual deterioration in central vision.
About NT-501 Implant
Designed to be implanted into the vitreous cavity of the eye, the investigational NT-501 implant is a tiny hollow cylindrical membrane which encapsulates human epithelial cells genetically engineered to produce ciliary neurotropic factor (CNTF) continuously, a protein now clinically validated in Phase 3 clinical trials to slow the progression of MacTel.
About Encapsulated Cell Therapy
Encapsulated Cell Therapy (ECT) is an investigational, first in class, platform technology that promotes continuous production of therapeutic proteins to the eye with the potential to treat a broad array of ocular diseases. It utilizes a proprietary, well-characterized retinal pigment epithelial cell line that has been genetically engineered to produce therapeutically active biologics. The cells are encapsulated in a semi-permeable membrane that allows for selective passage of therapeutic proteins.
The ECT platform is inserted during a single outpatient surgical procedure through a small scleral incision, and can also be removed through the same incision, if desired. ECT has the potential to address the current limitations of intraocular drug delivery by allowing for and ensuring patient compliance and reducing treatment burden with one surgical procedure that can deliver drug for at least 2 years.
About Neurotech Pharmaceuticals, Inc.
Neurotech Pharmaceuticals, Inc. is a private clinical stage biotech company focused on developing transformative therapies for chronic eye diseases. The core platform technology, ECT, enables continuous production of therapeutic proteins to the eye. Neurotech is currently studying in the clinic ECT candidates to treat Macular telangiectasia type 2 and glaucoma. To learn more, visit www.neurotechpharmaceuticals.com.
-Kathleen E. Moore