Applied Therapeutics APLT announced that the FDA has issued a Complete Response Letter (CRL) for the new drug application (NDA) seeking approval for APLT’s lead candidate, govorestat, to treat classic galactosemia. The CRL stated that the FDA has reviewed the application and cannot approve the NDA in its current form due to deficiencies in the clinical study results submitted in support of the application.

Applied Therapeutics is evaluating the FDA's feedback. It intends to promptly request a meeting to discuss the requirements for a potential NDA resubmission or appeal of the decision, and appropriate next steps. Shares of the company tanked 77.3% in the after-market hours, following the disappointing news.

APLT’s govorestat (also known as AT-007) is a novel central nervous system penetrant ARI, which is currently being developed for three rare neurological diseases. The NDA for govorestat for the galactosemia indication was accepted by the FDA in late February under its Priority Review pathway. 

Year to date, shares of Applied Therapeutics have skyrocketed 155.8% against the industry’s 7.8% decline.

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Per APLT, clinical study results submitted in support of the govorestat NDA showed rapid and sustained reductions in galactitol upon treatment with the candidate, leading to meaningful clinical benefits for pediatric patients and a favorable safety profile. In the phase III ACTION-Galactosemia Kids study in children with galactosemia aged 2-17 years, govorestat improved daily activities, behavior, cognition, fine motor skills and tremor. It also significantly reduced plasma galactitol levels in adults and children with galactosemia. Additional studies provided strong efficacy and safety data across 185 classic galactosemia patients over three years.

Per Applied Therapeutics, approximately 3,300 patients in the United States with 80-100 new births per year and approximately 4,400 patients in the EU with 120 new births per year suffer from galactosemia, a rare genetic metabolic disease.

A regulatory filing for govorestat to treat classic galactosemia is also currently under review in the EU. The company expects a decision by the EU regulatory body early in the first quarter of 2025.

Other Programs in APLT’s Clinical-Stage Pipeline

Apart from thegalactosemia indication, Applied Therapeutics is currently evaluating govorestat for two other indications, namely sorbitol dehydrogenase (SORD) deficiency and phosphomannomutase 2 deficiency (PMM2-CDG).

The company is gearing up to submit an NDA with the FDA for govorestat to treat the SORD indication early in the first quarter of 2025. Applied Therapeutics clarified that the review and potential approval of govorestat for treating SORD is independent of the ongoing review of govorestat for classic galactosemia.

Govorestat enjoys the Orphan Medicinal Product designation in the EU for both galactosemia and SORD deficiency indications. Govorestat has also received Orphan Drug designation from the FDA for the treatment of galactosemia, PMM2-CDG and SORD deficiency, Pediatric Rare Disease designation for galactosemia and PMM2-CDG and Fast Track designation for galactosemia.

Besides govorestat, APLT’s pipeline comprises two other investigational candidates — AT-001 is being studied for the treatment of diabetic cardiomyopathy, while AT-003 is being developed for diabetic retinopathy.

Applied Therapeutics Inc. Price and Consensus

Applied Therapeutics Inc. price-consensus-chart | Applied Therapeutics Inc. Quote

Zacks Rank & Stocks to Consider

Applied Therapeutics currently carries a Zacks Rank #4 (Sell).

Some better-ranked pharma stocks are Allogene Therapeutics ALLO, Pfizer PFE and Gilead Sciences GILD, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Over the past 60 days, Allogene Therapeutics’ estimates for loss per share have narrowed from $1.40 to $1.35 for 2024, and that for 2025 has narrowed from $1.46 to $1.34. ALLO shares have lost 21.5% year to date.

Allogene Therapeutics’ earnings beat estimates in three of the trailing four quarters and matched once, delivering an average surprise of 9.42%.

Over the past 60 days, Pfizer’s earnings estimates have risen from $2.62 to $2.91 per share for 2024, while that for 2025 has increased from $2.84 to $2.91. PFE shares have lost 10.6% year to date.

Pfizer’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 74.50%.

Over the past 60 days, Gilead Sciences’ earnings estimates have risen from $3.79 to $4.32 per share for 2024 while that for 2025 has increased from $7.24 to $7.38. GILD shares have risen 12.8% year to date.

Gilead Sciences’ earnings beat estimates in three of the trailing four quarters and missed the same in one, delivering an average surprise of 15.46%.

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