LENZ Therapeutics announced its New Drug Application (NDA) for LNZ100, a novel aceclidine-based eye drop for presbyopia, remains on track for its FDA decision date of August 8, 2025. The company reported fourth quarter and full year 2024 financial results while highlighting significant progress toward potential commercialization of what could become the first aceclidine-based treatment for presbyopia in the United States.
The San Diego-based biopharmaceutical company completed its mid-cycle review with the FDA in January 2025, with no significant issues identified. The regulatory agency has maintained its Prescription Drug User Fee Act (PDUFA) target action date and confirmed it does not plan to hold an Advisory Committee Meeting for the application.
"This year has been an extremely successful year for LENZ, and we have continued to make progress in the fourth quarter and recent period as we move closer to the potential approval and launch of LNZ100 in the United States," said Eef Schimmelpennink, President and Chief Executive Officer of LENZ Therapeutics.
Strong Clinical Results Support Regulatory Application
The NDA submission is supported by positive data from the pivotal Phase 3 CLARITY study. Additionally, in October 2024, LENZ and its partner CORXEL announced positive topline data from a Phase 3 study conducted in China. In this trial, LNZ100 (1.75% Aceclidine) achieved its primary endpoint with 74% of patients showing three-lines or greater improvement in Best Corrected Distance Visual Acuity (BCDVA) at near without losing one-line or more in distance visual acuity at 3 hours post-dose. The difference in efficacy was statistically significant compared to the vehicle-controlled group (p<0.0001).
Aceclidine represents a new chemical entity in the United States and is not currently approved for presbyopia treatment in any country. LENZ's formulation is designed as a pupil-selective, ciliary-body sparing miotic, which the company believes offers advantages over existing treatment approaches.
Commercial Preparations Accelerate
LENZ has significantly advanced its commercial readiness in anticipation of potential approval. The company's unbranded EYEAMSELECTIVE campaign, approaching its first anniversary, has generated over 2 million digital impressions targeting more than 30,000 Eye Care Professionals (ECPs). The campaign focuses on educating practitioners about the benefits of pupil-selective, ciliary-body sparing miotic treatments for presbyopia.
The company has expanded its commercial infrastructure, hiring key sales leadership including Regional Sales Directors and District Managers with substantial eye care and pharmaceutical sales experience. Manufacturing operations have begun production to support the potential commercial launch, which is anticipated in the fourth quarter of 2025, pending FDA approval.
"I am very pleased with the advancements made by our regulatory team with the FDA, having recently completed our mid-cycle review, which led to our manufacturing operations team initiating production to support our potential commercial launch," Schimmelpennink noted.
Addressing a Significant Market Need
Presbyopia affects an estimated 1.8 billion people globally, including 128 million in the United States alone. The condition, which typically begins around age 40, causes progressive difficulty focusing on near objects due to age-related hardening of the eye's lens.
LNZ100 is being developed as a preservative-free, single-use, once-daily eye drop containing aceclidine. The company's commercial positioning emphasizes its goal of providing a solution that enhances vision for "all eyes, all day."
Financial Position Supports Launch Plans
As of December 31, 2024, LENZ reported cash, cash equivalents, and marketable securities of $209.1 million, which the company anticipates will fund operations through commercial launch to post-launch positive operating cash flow.
For the fourth quarter of 2024, LENZ reported:
- Research and Development expenses of $5.9 million, down from $19.5 million in Q4 2023
- Selling, General and Administrative expenses of $9.4 million, up from $5.5 million in Q4 2023
- Net loss of $12.7 million, or $0.46 per share, compared to a net loss of $23.7 million, or $12.04 per share in Q4 2023
For the full year 2024, the company reported:
- R&D expenses of $29.8 million, down from $59.5 million in 2023
- SG&A expenses of $28.8 million, up from $12.9 million in 2023
- Net loss of $49.8 million, or $2.34 per share, compared to a net loss of $70.0 million, or $35.71 per share in 2023
The decrease in R&D expenses was primarily attributed to the conclusion of the Phase 3 CLARITY study in March 2024, while the increase in SG&A expenses reflected growing personnel costs and pre-commercial activities.
Looking Forward
LENZ will host a Commercial Day on April 15, 2025, to provide further details on its commercialization strategy and progress. The event will feature presentations on commercialization strategies, supply chain readiness, and perspectives from key opinion leaders and eye care professionals.
As the company approaches its PDUFA date, it continues to focus on finalizing its commercial infrastructure and branding creative. If approved, LNZ100 would represent a significant advancement in the treatment landscape for presbyopia, offering a novel pharmaceutical approach to a condition that has traditionally been managed with reading glasses or contact lenses.
