TearSolutions Receives FDA Orphan Drug and Fast Track Designations for Lacripep in Neurotrophic Keratitis, Initiates Phase 2 Trial
核心洞察
TearSolutions received FDA Orphan Drug and Fast Track designations for Lacripep, a first-in-class synthetic peptide therapy for neurotrophic keratitis (搜索) (NK), a rare degenerative corneal disease.
The company has initiated a multicenter, randomized, vehicle-controlled Phase 2 clinical trial (NCT07568730) enrolling approximately 54 patients with Stage 1 NK across three U.S. sites.
Lacripep is a synthetic 10-amino acid fragment of the human tear protein lacritin, designed to repair corneal epithelium and restore sensory nerve function via topical eye drop administration.
TearSolutions, Inc., a clinical-stage biotechnology company based in Charlottesville, Virginia, announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Fast Track Designation for Lacripep, its investigational ophthalmic therapeutic for neurotrophic keratitis (搜索) (NK). Concurrently, the company has initiated a Phase 2 clinical trial with the first patients now dosed.
The dual regulatory designations underscore the significant unmet medical need in NK, a rare degenerative corneal disease characterized by reduced or absent corneal sensitivity and severely impaired corneal healing, which can lead to persistent epithelial defects and vision loss.
"Receiving both Orphan Drug and Fast Track designations from the FDA is a significant milestone that underscores the unmet medical needs that still exist in neurotrophic keratitis (搜索) and the potential for Lacripep to transform how this disease is treated," said Anil Asrani, Chief Executive Officer of TearSolutions. "These designations validate our approach and afford us the opportunity to work closely with the FDA to accelerate our clinical development and bring this much-needed therapeutic option to patients sooner."
The FDA's Orphan Drug Designation provides incentives including tax credits for clinical testing and up to seven years of U.S. market exclusivity upon regulatory approval. The Fast Track Designation facilitates more frequent interactions with the FDA and enables a rolling review of the New Drug Application, expediting the development and review process for drugs that treat serious conditions.
Lacripep: A First-in-Class Peptide Therapy
Lacripep is a novel, first-in-class synthetic peptide derived from the human tear protein lacritin. Specifically, it is a synthetic 10-amino acid fragment of the naturally occurring lacritin protein, which was discovered to be selectively deficient in most forms of ocular surface disease (OSD), including dry eye and corneal disease such as NK.
The therapeutic was discovered at the University of Virginia with National Eye Institute (NEI) funding as a naturally occurring fragment of the larger 119-amino acid protein lacritin, identified in an unbiased screen for novel factors capable of addressing ocular surface disease. TearSolutions was originally formed in 2013 as a spin-off from the University of Virginia, based on NEI-funded research into tear composition led by co-founder and Chief Scientific Officer Gordon W. Laurie, PhD, FARVO, a professor of cell biology and ophthalmology.
Lacripep's mechanism of action involves dual neurotrophic and prosecretory functions aimed at resolving underlying nerve damage, reactivating physiological basal tear secretion, and restoring health to the corneal epithelium. Administered topically as an eye drop, the therapy is designed to repair the corneal epithelium's barrier function and restore the normal function of sensory nerves, with the goal of re-establishing natural production of all three tear film layers: aqueous, lipid, and mucin.
In preclinical animal models, topically administered Lacripep restored basal tearing without irritation and healed the corneal surface. More recently, Lacripep was evaluated in a completed first-in-human Phase 1/2 clinical trial for primary Sjögren's Syndrome (搜索), where results demonstrated rapid improvement in corneal health and reduction in symptoms appearing as early as two weeks.
Phase 2 Clinical Trial Design
The newly initiated Phase 2 clinical trial (NCT07568730) is a multicenter, randomized, vehicle-controlled, double-masked-to-open-label study being conducted at three clinical site locations across the United States. Approximately 54 subjects aged 18 and older diagnosed with Stage 1 NK will be enrolled.
Key inclusion criteria, which must be present in at least one eye, include: Grade 3 corneal fluorescein staining (cCFS) using the NEI scale (0–3 in one-point increments); corneal sensitivity of 4 cm or less in the central zone as measured by Cochet-Bonnet aesthesiometer; best-corrected distance visual acuity (BCDVA) of +0.2 to +1.0 logMAR (Snellen equivalent 20/32 to 20/200); and intraocular pressure (IOP) of 21 mmHg or less.
The study design includes a 2-week run-in period with open-label vehicle, after which eligible patients are randomized to receive either Lacripep (as a 4 µM ophthalmic solution) or vehicle, dosed three times daily (TID) in both eyes for 8 weeks. Clinic visits occur at Weeks 2, 4, and 8. At Week 8, all patients are assigned to open-label treatment with Lacripep for an additional 4 weeks through Week 12, allowing exploration of both shorter and longer treatment periods—4 weeks of active treatment for those originally on vehicle, and 12 total weeks for those originally randomized to Lacripep.
The primary efficacy endpoint, measured from baseline to Week 8, is the change in cCFS in the study eye. The secondary efficacy endpoint is the change in central corneal sensitivity in the study eye. The study will also evaluate Lacripep's effect on visual function and quality of life among patients with Stage 1 NK.