Lexeo Down Despite Upbeat Rare Disease Gene Therapy Data

Shares of Lexeo Therapeutics LXEO lost 26% on Monday after it reported positive interim data from two cohorts of the phase I/II SUNRISE-FA study evaluating LX2006, an investigational gene therapy for treating Friedreich ataxia (FA) cardiomyopathy.

Data from the SUNRISE-FA study showed that treatment with LX2006 achieved a mean reduction in left ventricular mass index (LVMI) of 11.4% at 12 months and 18.3% at 18 months in participants with elevated LVMI at baseline. LVMI is an estimator of cardiac morbidity and mortality. Treatment with the therapy was also well-tolerated across all study participants, with no treatment-related serious adverse events.

The interim data also showed that treatment with LX2006 also showed improvement in other key measures of cardiac status, including left ventricular wall thickness (an early indicator of an inefficient heart) and troponin I (a biomarker of myocardial injury), in the majority of participants at 12 months.

Based on the above results, management sees a path for seeking the FDA's approval for LX2006 under the accelerated pathway.

Lexeo intends to report additional data at a future medical meeting later this year. Management is currently dosing patients in the third dose cohort of the SUNRISE-FA study, which is also enrolling study participants.

While Wall Street analysts were impressed with the results, some investors raised concerns about the data. They stated that the results were not enough to warrant accelerated approval from the FDA. The data also did not show that patients' function improved after treatment with LX2006. This was likely the reason for the share price decline.

Year to date, Lexeo's shares have lost 3.0% compared with the industry's 3.6% fall.

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FA is an ultra-rare genetic, progressive, neurodegenerative movement disorder that affects nearly 5,000 diagnosed patients in the United States. FA cardiomyopathy is the most common cause of mortality in FA patients. Currently, there are no approved therapies for FA cardiomyopathy.

The only approved treatment for FA indication is Biogen's BIIB Skyclarys, which is approved to treat FA in individuals aged 16 years and older. This drug, which was approved by the FDA last year, was added to Biogen's portfolio following the September 2023 acquisition of Reata Pharmaceuticals. However, the drug is effective in slowing neurological disease progression and does not impact the heart.

Zacks Rank & Key Picks

Lexeo currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include Arcutis Biotherapeutics ARQT and Compugen CGEN. While CGEN sports a Zacks Rank #1 (Strong Buy), ARQT carries a Zacks Rank #2 (Buy) at present.

In the past 60 days, estimates for Compugen's 2024 earnings per share have risen from 2 cents to 5 cents. During the same period, loss estimates per share for 2025 have improved from 27 cents to 11 cents. Year to date, CGEN's shares have lost 3.5%.

Earnings of Compugen beat estimates in three of the last four quarters while missing the mark on one occasion. CGEN delivered a four-quarter average earnings surprise of 5.79%.

In the past 60 days, estimates for Arcutis Biotherapeutics' 2024 loss per share have narrowed from $2.22 to $1.60. During the same period, the loss estimates per share for 2025 have narrowed from $1.37 to $1.14. Year to date, shares of Arcutis have surged 233.4%.

Earnings of Arcutis Biotherapeutics beat estimates in three of the last four quarters while missing the mark on one occasion. Arcutis delivered a four-quarter average earnings surprise of 14.93%.

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