LENZ Therapeutics Announces Positive Topline Data from Phase 3 CLARITY Presbyopia Trials

Rhea-AI Summary

LENZ Therapeutics, Inc. announces positive Phase 3 CLARITY study results for LNZ100 eye drops to treat presbyopia, with 71% achieving significant vision improvement at 3 hours, rapid onset, and long duration. New Drug Application planned for mid-2024.

Medical Research Analyst

The announcement of positive topline results from LENZ Therapeutics' Phase 3 CLARITY study for its investigational formulations LNZ100 and LNZ101 is significant within the biopharmaceutical sector, particularly for those companies developing treatments for presbyopia. The reported efficacy of LNZ100 in improving near vision without compromising distance vision could mark a substantial advancement in presbyopia treatment, potentially reducing the reliance on reading glasses or surgical interventions. The market for presbyopia correction is large, with an estimated 128 million people affected in the United States alone, indicating a significant commercial opportunity for LENZ upon FDA approval and successful commercialization.

Investors should note the impressive responder rates, rapid onset and duration of effect, which are key factors in patient compliance and satisfaction with a therapeutic product. The lack of serious treatment-related adverse events over 30,000 treatment days is also a strong indicator of the drug's safety profile, an essential aspect for FDA approval. The anticipated NDA submission in mid-2024 suggests a potential market entry in the latter half of 2025, which could be a major catalyst for the company's stock. However, it is important to monitor the company's progress towards the NDA submission and any regulatory hurdles that may arise.

Market Research Analyst

From a market perspective, the data presented by LENZ Therapeutics positions LNZ100 as a strong contender in the presbyopia treatment landscape. The high rate of patient willingness to continue using the eye drops post-study (75%) and the anticipated frequency of usage (4-7 days a week) suggest a high level of product acceptance and a recurring revenue model for the company. These factors could drive investor interest and potentially increase the company's valuation as it moves closer to commercialization.

Moreover, the patient demographic for presbyopia treatments, primarily individuals aged 45 to 75, is a growing segment due to the aging global population. This demographic trend could further expand the market for LNZ100. The company's strategy to focus on the commercialization of LNZ100, while supported by solid clinical data, will require careful execution to capture market share, especially considering the competitive landscape and any existing or emerging alternative treatments.

Healthcare Economist

From an economic standpoint, the development of non-invasive treatment options like LNZ100 has the potential to reduce overall healthcare costs associated with vision correction for presbyopia. By providing an alternative to reading glasses or surgery, aceclidine-based eye drops could lead to cost savings for both patients and insurance providers. The economic implications extend beyond direct treatment costs to include increased productivity and quality of life for individuals who would otherwise struggle with near vision tasks.

It is, however, important to consider the pricing strategy LENZ Therapeutics will adopt for LNZ100, as it could influence the adoption rate and insurance coverage decisions. The company's ability to negotiate favorable reimbursement rates will be a key factor in the drug's accessibility and success in the market. Additionally, the long-term impact on the company's financials will depend on the drug's market penetration and the competitive dynamics of the presbyopia treatment market, including potential generic competition in the future.

– LNZ100 selected as lead candidate

– Primary endpoint was met with 71% of participants dosed with LNZ100 achieving three-lines or greater improvement at 3 hours

– Rapid onset and long duration shown with 71% of participants achieving three-lines or greater improvement at 30 minutes and 40% at 10 hours

– New Drug Application submission anticipated in mid-2024

– Company to host a conference call and webcast today at 8:00 a.m. ET

SAN DIEGO--(BUSINESS WIRE)-- LENZ Therapeutics, Inc. (Nasdaq: LENZ or “LENZ” or the “Company”), a late clinical-stage biopharmaceutical company focused on developing the first aceclidine-based eye drop to improve near vision in people with presbyopia, today announced positive topline results from its Phase 3 CLARITY study of two investigational formulations of aceclidine, LNZ100 and LNZ101, for the treatment of presbyopia, the inevitable loss of near vision that impacts the daily lives of nearly all people over 45.

In Phase 3 safety and efficacy trials (CLARITY 1 and 2), our lead product candidate LNZ100 (1.75% aceclidine) achieved the primary endpoints and key secondary endpoints, with statistically significant three-lines or greater improvement in Best Corrected Distance Visual Acuity (BCDVA) at near, without losing one-line or more in distance visual acuity. In the vehicle-controlled CLARITY 2 trial, the Day 1 results showed (all p<0.0001):

• Rapid onset: 71% achieved three-lines or greater improvement at 30 minutes.
• Primary endpoint: 71% achieved three-lines or greater improvement at 3 hours.
• Long duration: 40% achieved three-lines or greater improvement at 10 hours.

Near vision improvement was reproducible and consistent across both CLARITY 1 and 2 throughout the four-week study periods.

LNZ100 was well-tolerated with no serious treatment-related adverse events observed in the over 30,000 treatment days across all three CLARITY trials.

LNZ101 showed similar results, including achieving primary and secondary endpoints in both CLARITY 1 and 2, but did not show superiority to LNZ100. Based on these results, LENZ selected LNZ100 as its lead product candidate, for which it plans to submit a New Drug Application (NDA) in mid-2024.

“We are very pleased with the outcome of the CLARITY trials, and most importantly the strong efficacy and safety profile of LNZ100 observed in patients with presbyopia. We would like to thank our investigators, clinical sites, and all participants in our study,” said Eef Schimmelpennink, President and Chief Executive Officer of LENZ Therapeutics. “We believe these data support LNZ100 as a potential best-in-class therapy for the treatment of presbyopia. The high responder rate, rapid onset and long duration across a broad range of presbyopes ranging from 45 to 75 years of age and having a refractive range from -4.0 to +1.0D SE are consistent with features that patients are expecting from an effective treatment option. Based on these highly encouraging data, we will direct our focus towards our NDA submission in mid-2024 for LNZ100, and preparations for commercialization in second half of 2025 upon FDA approval, with the goal of moving closer to helping many of the 128 million people experiencing symptoms of presbyopia in the United States.”

“These positive CLARITY study data build on the compelling results from our INSIGHT trials, demonstrating a robust safety and efficacy profile for the use of aceclidine to treat presbyopia,” said Marc Odrich, Chief Medical Officer of LENZ Therapeutics. “Presbyopes often experience an abrupt change in their daily life as the symptoms become progressively pronounced starting in their mid-40s, when reading glasses or other corrective aids often become necessary to read, text or conduct close-up work. The statistically significant data and clinically meaningful outcomes observed in the CLARITY trials support the potential paradigm-shifting impact LNZ100 can have as an alternative and convenient therapeutic option to reading glasses.”

CLARITY Phase 3 Study Topline Data Highlights:

CLARITY is a Phase 3 multi-center, double-masked, randomized, controlled, efficacy and safety study for LNZ100 and LNZ101 for the treatment of presbyopia. It is comprised of two six-week efficacy trials, CLARITY 1 and 2, and a six-month safety trial, CLARITY 3. The trials enrolled a total of 1,059 participants ranging from ages 45 to 75, and a refractive range of -4.0D SE to +1.0D SE, and included users who previously had LASIK surgery or are pseudophakes. The primary efficacy endpoint in both CLARITY 1 and 2 is the percentage of participants who achieve three-lines or greater improvement in BCDVA at near without losing one-line (5 letters or more of distance vision) at 3 hours post-treatment.

In both the vehicle-controlled CLARITY 2 trial and the brimonidine-controlled CLARITY 1 trial, our lead product candidate LNZ100 (1.75% aceclidine) achieved all primary and secondary near vision improvement endpoints, including demonstrating (in all cases, p<0.0001):

• Rapid onset: at 30 minutes, for CLARITY 2, 71% and 91% of participants achieved three- and two-lines or greater improvement, respectively, and for CLARITY 1, 72% and 87% of participants achieved three- and two-lines or greater improvement, respectively;
• At 3 hours (primary endpoint for three-lines): for CLARITY 2, 71% and 91% of participants achieved three- and two-lines or greater improvement, respectively, and for CLARITY 1, 64% and 83% of participants achieved three- and two-lines or greater improvement, respectively; and
• Long duration: at 10 hours, for CLARITY 2, 40% and 69% of participants achieved three- and two-lines or greater improvement, respectively, and for CLARITY 1, 27% and 61% of participants achieved three- and two-lines or greater improvement, respectively.

Near vision improvement was reproducible and consistent across both CLARITY 1 and 2 throughout the four-week study periods.

Additionally, in CLARITY 2, nearly all (95%) participants who received LNZ100 achieved the clinically meaningful two-lines or greater improvement (p<0.0001) at 1 hour post-treatment.

LNZ100 also demonstrated statistically significant (p<0.0001) improvement of 2-4 letters on distance vision in normal light and no negative impact to distance vision in low light at all time points.

LNZ100 was well-tolerated with no treatment-related serious adverse events observed in the over 30,000 treatment days across all three CLARITY trials. The only reported adverse events with an incidence at 5% or more were installation site irritation, visual impairment and hyperemia which were 100% characterized by participants as mild, and headaches which were characterized as mild by 89% of participants. We believe these adverse events were transient, consistent with those observed in previous trials.

On day 28 of the CLARITY 1 and 2 trials, 223 participants who received LNZ100 were surveyed on their experience. 90% indicated they noticed an improvement in their near vision and 75% said they would want to continue to use these eye drops after the study, of which 81% noted they expected to use them 4-7 days a week. We believe these patient survey outcomes further confirm the potential commercial opportunity for LNZ100.

Based on these results, LENZ Therapeutics anticipates submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for LNZ100 as a treatment for presbyopia mid-2024.

Additional results from the CLARITY study will be presented at future medical meetings.

Conference Call Information

The Company will host a conference call and webcast today, Wednesday, April 3, 2024, at 8:00 a.m. ET to discuss the topline results. The live webcast and materials from today’s conference call can be accessed here and on the LENZ Therapeutics website at www.LENZ-tx.com in the Investors & Media section or by calling 877-315-3033 or 215-268-9883. A replay of the webcast will be archived and available for 30 days following the event.

About Presbyopia

Presbyopia is the inevitable loss of near vision associated with aging and impacts the daily lives of nearly all people over 45. In the United States, the estimated addressable population who suffer from this condition, known as presbyopes, is 128 million, almost four times the number of individuals suffering from dry eye disease and three times the number of individuals suffering from childhood myopia, macular degeneration, diabetic retinopathy, and glaucoma combined.

About LENZ Therapeutics

LENZ Therapeutics is a late clinical-stage biopharmaceutical company focused on developing the first aceclidine-based eye drop to improve vision in patients diagnosed with presbyopia. LENZ’s product candidate LNZ100 is a preservative-free, single-use, once-daily eye drop containing aceclidine. The Phase 3 CLARITY trials of LNZ100 were completed in March 2024 and NDA submission is planned in mid-2024. LENZ is committed to commercializing a potential best-in-class pharmaceutical presbyopia solution that enhances vision for “all eyes, all day.” LENZ is headquartered in San Diego, California. For more information, visit: LENZ-tx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of federal securities laws. You can identify forward-looking statements by words such as “may,” “will,” “could,” “can,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “poised,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, but not all forward-looking statements will contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding the potential of LNZ100 to have best-in-class performance; our plans relating to the clinical development of our product candidates, including statements regarding the timing, presentation and reporting of data from our clinical trials and studies and the timing of a potential NDA submission for LNZ100; the size of the addressable population for our product candidates; our expectations regarding the commercial opportunity and beneficial characteristics of our product candidates; and our plans regarding commercialization of LNZ100, if approved. These statements are based on numerous assumptions concerning the development of LENZ’s products and target markets and involve substantial risks, uncertainties and other factors that may cause actual results, levels of activity, performance or achievement to be materially different from the information expressed or implied by these forward-looking statements, including those risk factors described in the final 424B3 proxy statement/prospectus filed with the SEC on February 13, 2024. We cannot assure you that the forward-looking statements in this press release or the assumptions upon which they are based will prove to be accurate. The forward-looking statements in this press release are as of the date of this press release. Except as otherwise required by applicable law, LENZ disclaims any duty to update any forward-looking statements. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240403843686/en/

Dan Chevallard

LENZ Therapeutics

IR@LENZ-Tx.com

Janhavi Mohite

Stern Investor Relations, Inc.

janhavi.mohite@sternir.com

Source: LENZ Therapeutics, Inc.

FAQ

What are the key highlights of LENZ Therapeutics' Phase 3 CLARITY study for LNZ100?

The study showed 71% of participants achieving significant vision improvement at 3 hours, rapid onset at 30 minutes, and long duration at 10 hours.

What is the primary endpoint met by LNZ100 in the Phase 3 study?

71% of participants dosed with LNZ100 achieved three-lines or greater improvement at 3 hours.

When is the New Drug Application submission expected for LNZ100?

The submission is anticipated in mid-2024.

What is the focus of LENZ Therapeutics as a late clinical-stage biopharmaceutical company?

Developing the first aceclidine-based eye drop to improve near vision in people with presbyopia.

What is the goal of LENZ Therapeutics upon FDA approval of LNZ100?

To move closer to helping many of the 128 million people experiencing symptoms of presbyopia in the United States.

What percentage of participants achieved rapid onset with LNZ100 in the CLARITY study?

71% achieved three-lines or greater improvement at 30 minutes.

How was the safety profile of LNZ100 in the CLARITY trials?

LNZ100 was well-tolerated with no serious treatment-related adverse events observed.

What percentage of participants noticed an improvement in their near vision with LNZ100?

90% of participants indicated they noticed an improvement in their near vision.

What were the most common adverse events reported with LNZ100 in the CLARITY trials?

The most common adverse events were installation site irritation, visual impairment, hyperemia, and headaches.

Where can more information about the conference call discussing the topline results be accessed?

The webcast and materials can be accessed on the LENZ Therapeutics website or by calling the provided numbers.

What is the potential commercial opportunity for LNZ100 based on patient survey outcomes?

Patient survey outcomes confirm the potential commercial opportunity for LNZ100.

What is the role of aceclidine in the treatment of presbyopia according to LENZ Therapeutics?

Aceclidine is intended to provide an alternative and convenient therapeutic option to reading glasses.

What is the refractive range of participants in the CLARITY study?

Participants ranged from ages 45 to 75 and had a refractive range of -4.0D SE to +1.0D SE.

How many participants were enrolled in the CLARITY trials?

A total of 1,059 participants were enrolled in the trials.

What is the significance of the Phase 3 CLARITY study results for LNZ100?

The results support LNZ100 as a potential best-in-class therapy for the treatment of presbyopia.

What is the timeline for commercialization of LNZ100 upon FDA approval?

Preparations for commercialization are planned in the second half of 2025 upon FDA approval.