The company said that Govorestat slowed disease progression and improved patient outcomes in CMT-SORD, a rare hereditary neuropathy. The company plans to submit an NDA in 2025 amid strong retail investor interest.

Retail buzz surrounding Applied Therapeutics Inc spiked late Sunday after the company revealed full 12-month clinical results alongside new 18- and 24-month topline data from a midstage trial of govorestat to treat Charcot-Marie-Tooth disease caused by Sorbitol Dehydrogenase deficiency (CMT-SORD).

MRI observations showed that govorestat treatment slowed disease progression at 24 months, while keeping blood sorbitol levels low and improving functional outcomes and patient-reported metrics.

The 12-month data revealed a statistically significant increase in the CMT-Health Index (CMT-HI) and a connection between sorbitol reductions and enhanced mobility and balance. 

CMT-SORD is a rare, progressive neuromuscular disorder that leads to nerve damage, muscle weakness, and mobility impairment.

The company acknowledged that the primary endpoint of the 10MWRT at 12 months failed to show statistical significance, which led to its removal from the CMT-FOM clinical outcomes tool.

Patients demonstrated good tolerance for govorestat, maintaining safety for two years. 

90% of eligible patients continued participating in the open-label extension study. 

Applied Therapeutics said it will submit a New Drug Application (NDA) for govorestat by 2025.

The company’s shares surged 22.8% to close at $0.60 on Friday, with a further 1.2% gain in after-hours trading.

On Stocktwits, retail sentiment was ‘extremely bullish’ amid ‘a 629% surge in 24-hour message volume.

Some retail traders expressed optimism about Applied Therapeutics' prospects, with one suggesting that the stock’s sharp decline in December may have been overdone and that the current low valuation presents a “monster opportunity.”

Another pointed to the positive impact of govorestat on disease progression in CMT-SORD, noting that in a rare condition with no approved treatments, the available data could support regulatory approval.

The stock has declined 33% so far in 2025.

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