Applied Therapeutics, Inc. has taken a significant step forward in the treatment of Charcot-Marie-Tooth disease caused by Sorbitol Dehydrogenase deficiency (CMT-SORD), with the presentation of full 12-month clinical results and new topline data from 18 and 24 months at the Peripheral Nerve Society 2025 Annual Meeting. The data underscores the potential of govorestat (AT-007) to alter the course of this rare, progressive neuromuscular disease, offering hope to patients worldwide.

The findings revealed a slowing of disease progression observed via MRI at 24 months, alongside improvements in outcomes and a reduction in sorbitol levels maintained through the same period. Notably, a statistically significant correlation was found between the absolute reduction in sorbitol and changes in the 10MWRT and CMT-FOM Lower Limb domain at 12 months. Although the primary endpoint of 10MWRT at 12 months was not statistically significant, the company has since removed this measure from the CMT-FOM clinical outcomes assessment based on the ACT-CMT study results.

Further bolstering the case for govorestat, treatment in CMT-SORD-Null rats showed a highly statistically significant 37% reduction of sorbitol in the sciatic nerve, mirroring the peripheral neuropathy experienced by patients. The drug has been generally safe and well tolerated, with the company remaining on track for a potential NDA submission in 2025. This commitment reflects Applied Therapeutics' dedication to addressing unmet needs in rare diseases.

The positive data has not gone unnoticed by the investment community, with Applied Therapeutics' shares experiencing a notable surge. Retail traders, in particular, have shown heightened interest, as evidenced by a 629% surge in 24-hour message volume on Stocktwits. The optimism among investors underscores the potential market impact of govorestat, should it receive regulatory approval, marking a pivotal moment for both the company and patients affected by CMT-SORD.

The UK is on the cusp of experiencing some of the highest temperatures of the year, with weather maps indicating a potential peak of 33C in parts of England. This scorching forecast, expected around June 26, could see regions such as the East of England, South East, and Greater London basking in intense heat. The Met Office and other weather modelling services highlight the possibility of this heatwave surpassing previous highs, marking a significant climatic event for the country.

Amidst the anticipation of soaring temperatures, the UK Health Security Agency (UKHSA) has issued its first yellow heat-health alert of the year. This alert, covering London, the southeast, East Anglia, and the East Midlands, underscores the heightened health risks for vulnerable populations, including the elderly and those with pre-existing conditions. Dr. Agostinho Sousa of the UKHSA emphasizes the importance of taking sensible precautions during this period to mitigate the adverse effects of the heat.

However, the weather narrative is not solely about the heat. The Met Office has also issued yellow thunderstorm warnings for various parts of the UK, including Northern Ireland, Wales, and South West England. These storms, potentially carrying Saharan dust, could lead to localized flooding, power cuts, and travel disruptions. The phenomenon, partly attributed to a Spanish plume, brings an added layer of complexity to the UK's weather dynamics, blending extreme heat with the threat of severe thunderstorms.

As the UK navigates through these contrasting weather extremes, the focus remains on preparedness and resilience. The combination of record-breaking temperatures and unpredictable storms presents a unique challenge for communities across the country. With the right precautions and awareness, the public can safely enjoy the summer's warmth while staying vigilant against the potential hazards posed by the upcoming weather conditions.