Nacuity Pharmaceuticals announced that the FDA granted Fast Track designation (FTD) to NPI-001 (N-acetylcysteine amide [NACA]) tablets, its investigational therapy for retinitis pigmentosa (RP) treatment.
First up: this company.
Launched in 2017 and based in Fort Worth, Texas, Nacuity is a clinical-stage pharmaceutical company developing targeted therapies for oxidative stress.
Supported by Foundation Fighting Blindness and the RD (Retinal Degeneration) Fund, the company currently has several highly differentiated clinical programs for:
- RP (in Usher syndrome)
- Cataract
- Cystinosis
- Neurological disease
Now this candidate.
NPI-001 is a proprietary, Good Manufacturing Practice (GMP)-grade formulation of NACA tablets designed to target oxidative stress associated with such diseases as RP.
What it does: Based on preclinical research, the therapeutic has been found to boost glutathione to prevent chemically-aggressive oxygen molecules from causing retinal cell damage.
And what does FTD mean for it?
Quick refresh: FTD is granted to a company in order to facilitate the development and expedite the review of a drug to treat serious medical conditions (such as RP) and fill an unmet need (plus potentially be eligible for Priority Review).
- Quite simply: It’s a way to make new drugs available to patients faster.
Keep in mind: Unlike the FDA’s Breakthrough Therapy designation, FTD can be requested with non-clinical data and/or preliminary clinical evidence.
And this isn’t NPI-001’s first designation, right?
Indeed. The FDA already granted NPI-001 Orphan Drug designation earlier this year, according to the company.
What this means: NPI-001 is eligible for a new drug application (NDA), which would give Nacuity 7 years of U.S. FDA exclusivity.
Let’s circle back to that clinical data … how has it performed so far?
According to Nacuity, NPI-001 preserved photoreceptor cells and functionality in preclinical animal studies. A later phase 1 study of the candidate among healthy participants was reported to be completed “with no serious adverse events.”
- More recently: The phase 1/2 SLO-RP trial (NCT04355689) is evaluating the safety and efficacy of NPI-001 (in tablet form) among patients diagnosed with RP associated with Usher syndrome.
Talk more about this phase 1/2 study.
- What it is: A randomized, placebo-controlled, double-masked, multicenter trial
- Initiation: September 2020
- Participants: 48 patients (aged 18+) diagnosed with Usher syndrome (see inclusion criteria)
- The setup: Participants randomized to receive either:
- Oral NPI-001 tablets (250 mg), twice daily (BID)
- Oral placebo tablet (BID)
- Duration: 24 months
- The outcome measure: Change from baseline for retinal sensitivity, as assessed via microperimetry of active versus placebo (measured at 24 months)
Its significance?
When the study was initially launched, G. Michael Wall, PhD, senior vice president and chief scientific officer at Nacuity, noted SLO-RP to be “an important milestone in our efforts to understand the potential of this treatment for RP and a prelude to treating other conditions of the eye involving oxidative stress.”
Has any data been reported yet?
Last we heard: The company achieved its target enrollment (48 participants, which was later updated in November 2024 to 49) for the study in May 2023.
At the time of announcing this target number, Nacuity also stated it planned to release an interim analysis (including efficacy data) by the end of 2023.
- Take note: As far as we can tell, this was not reported.
So you know what that means … stay tuned!