Applied Therapeutics Undergoes Leadership Changes Amid FDA Rejection and Regulatory Challenges

Leadership Changes:
Applied Therapeutics has undergone significant leadership changes following a month of bad news, including the resignation of CEO Shoshana Shendelman, who was also the company's founder and board chair. CFO Les Funtleyder has taken over as interim CEO and board chair2.

FDA Rejection:
The FDA rejected Applied Therapeutics' rare disease drug govorestat, citing deficiencies in the application. The drug was intended to treat classic galactosemia2.

Regulatory Challenges:
The FDA issued a warning letter to Applied Therapeutics regarding issues with the clinical trial for govorestat, including concerns with electronic data capture and significant findings documented during an inspection2.

Financial Impact:
The company's shares have plummeted by 90%, from over $10 to just 88 cents, due to the FDA rejection and regulatory challenges2.

Future Plans:
Applied Therapeutics is reevaluating its regulatory applications for govorestat and pushing back planned filings. The company is also withdrawing its marketing authorization application to the European Medicines Agency for govorestat due to the need for additional data2.

Financial Risk:
The company has a short cash runway of $98.9 million as of September 30, placing it at increased financial risk2.

Industry Context:
The rare disease space faces significant challenges, including lack of funding and regulatory hurdles. Many rare disease companies have announced layoffs or restructuring, and nearly 1,400 development programs have been paused or put on hold4.

Sources:

2. https://www.biospace.com/business/after-month-of-bad-news-applied-shuffles-leadership-lays-out-uncertain-path-for-rare-disease-drug

4. https://www.biospace.com/business/facing-a-dearth-of-big-pharma-interest-rare-disease-players-get-creative-to-fund-r-d