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.
In a remarkable demonstration of leadership and community service, two British-Nigerians, Opeyemi Bright and Jason Jackson, have been sworn in as mayors in London, marking a historic moment for the Nigerian diaspora. Their achievements have not only highlighted the contributions of Nigerians abroad but have also set new benchmarks for young leaders everywhere.
Opeyemi Bright, at the age of 29, has become the youngest-ever Civic Mayor of Barking and Dagenham, a testament to her dedication and hard work. Her journey in public service began at the age of 22 when she was elected as the borough's youngest Labour councillor. Similarly, Jason Jackson has made history as the first Nigerian-born Mayor of Islington since the borough's establishment in 1900. His rapid ascent from councillor to chair of the Homes and Communities Committee before becoming mayor showcases his commitment to public service.
President Bola Tinubu of Nigeria has extended his congratulations to both mayors, praising their achievements as a reflection of the excellence and integrity that Nigerians in the diaspora continue to embody. The President's message underscored the importance of their roles in inspiring young Nigerians both at home and abroad to pursue excellence and serve their communities with distinction.
The appointments of Bright and Jackson are a clear indication of the growing influence and positive impact of the Nigerian diaspora in global leadership roles. Their stories serve as a beacon of hope and inspiration, proving that with dedication and hard work, barriers can be broken and new heights can be achieved. As they embark on their new roles, the expectations are high, but so is the confidence in their abilities to lead and make a significant difference in their respective boroughs.