Nightstar Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for NSR-REP1 in Choroideremia
June 14, 2018
- First gene therapy RMAT designation for an inherited retinal disease
- STAR Phase 3 registrational trial ongoing and
- RMAT designation enables closer and more frequent multidisciplinary interaction with
FDAwith all of the benefits of breakthrough therapy designation
“Receiving RMAT designation for NSR-REP1 highlights the potential of this gene therapy to maintain and improve visual acuity in choroideremia,” said
Established under the 21st Century Cures Act, the RMAT designation is an expedited program for the advancement and approval of regenerative medicine products. A regenerative medicine is eligible for the designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such a disease or condition. RMAT allows companies developing regenerative medicine and gene therapies to work more closely and frequently with the FDA, and grants all of the benefits of Breakthrough Therapy Designation, including eligibility for priority review, rolling review and accelerated approval. In
RMAT designation for NSR-REP1 was based on clinical data supporting the maintenance and improvement of visual acuity from completed Phase 1/2 trials in choroideremia patients treated with NSR-REP1 and disease progression in untreated patients in the ongoing NIGHT natural history observational study.
CHM is a rare, degenerative, X-linked genetic retinal disorder primarily affecting males, with no treatments currently available and represents a significant unmet medical need. CHM presents in childhood as night blindness, followed by progressive constriction of the visual fields, generally leading to vision loss in early adulthood and total blindness thereafter. CHM is caused by mutations in the CHM gene, which encodes REP1, a protein that plays a key role in intracellular protein trafficking and the elimination of waste products from retinal cells. Absence of functional REP1 leads to death of the RPE cells and degeneration of the overlying retina, which contains the retinal photoreceptors required to convert light into visual signals. Thus, the loss of REP1 function in retinal cells caused by CHM results in progressive vision loss and blindness.
About Nightstar and NSR-REP1
Nightstar is a leading clinical-stage gene therapy company focused on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness. Nightstar’s lead product candidate, NSR-REP1, is comprised of an AAV2 vector containing recombinant human complementary DNA, or cDNA, that is designed to produce REP1 inside the eye. NSR-REP1 is currently in Phase 3 development for the treatment of patients with choroideremia, a rare, degenerative, genetic retinal disorder that has no current treatments and affects approximately one in every 50,000 people. Positive results from Phase 1/2 trials of NSR-REP1 were published in The Lancet in 2014 and in
For more information about Nightstar or its clinical trials, please visit www.nightstartx.com.
Cautionary Language Concerning Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. The words “believe,” “anticipate,” “intend,” “estimate,” “will,” “may,” “should,” “expect” or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements contained in this press release other than statements of historical facts are forward-looking statements, including, without limitation: statements about our planned and ongoing clinical trials for NSR-REP1, including our Phase 3 STAR trial in choroideremia, the continued clinical development of our pipeline, the timelines associated with our research and development programs including the timing of patient enrollment and the release of data from ongoing clinical trials and studies, whether the receipt of regenerative medicines advanced therapy designation for NSR-REP1 in choroideremia will meaningfully impact the development and review of NSR-REP1 by the
Source: Nightstar Therapeutics