Shares of Applied Therapeutics APLT surged 68.8% on Wednesday after it announced a regulatory update regarding the ongoing new drug application (NDA) review of APLT’s lead candidate, govorestat, for treating classic galactosemia.

Per the press release, in a late-cycle review meeting with the FDA, the regulatory body communicated to Applied Therapeutics that it no longer requires the opinion of an Advisory Committee. The Advisory Committee meeting was previously tentatively scheduled for Oct. 9, 2024.

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.

A filing designated as a Priority Review reduces the review period by four months. A final decision from the regulatory body was originally expected on Aug. 28, 2024. However, in March 2024, Applied Therapeutics announced that the FDA has extended the review period of the govorestat NDA by three months, assigning a new date for communicating its decision, Nov. 28, 2024.

Per the latest press release, the FDA has informed APLT that it remains on track to announce its decision regarding the govorestat NDA on Nov. 28, 2024, despite not holding an advisory committee meeting to discuss the application. Year to date, shares of Applied Therapeutics have skyrocketed 134.3% compared with the industry’s 0.5% growth.

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APLT Likely to Benefit From the Latest Regulatory Update

Subject to approval, govorestat would be the first medication indicated for the treatment of galactosemia and Applied Therapeutics’ first commercial product. Following a successful launch, the drug’s sales will provide the company with a regular source of revenues to fund other ongoing clinical programs.

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.

Per Applied Therapeutics, approximately 3,000 patients in the United States with 80 new births per year and approximately 4,000 patients in the EU with 120 new births per year suffer from galactosemia, a rare genetic metabolic disease.

Govorestat enjoys the Orphan Medicinal Product designation in the EU for both galactosemia and sorbitol dehydrogenase (SORD) deficiency indications. Govorestat has also received Orphan Drug designation from the FDA for the treatment of galactosemia, phosphomannomutase 2 deficiency (PMM2-CDG) and SORD deficiency, Pediatric Rare Disease designation for Galactosemia and PMM2-CDG and Fast Track designation for Galactosemia.

Other Programs in APLT’s Clinical-Stage Pipeline

Apart from thegalactosemia indication, Applied Therapeutics is currently evaluating govorestat for two other indications, namely SORD deficiency and PMM2-CDG.

Currently, the company is also gearing up for a regulatory filing with the FDA for govorestat to treat the SORD indication early in the first quarter of 2025. The planned regulatory submission will be based on positive interim 12-month results from its ongoing phase III INSPIRE registrational study of the candidate for the same indication.

Besides govorestat, Applied Therapeutics’ pipeline comprises two other investigational candidates — AT-001 is being studied for the treatment of diabetic cardiomyopathy and diabetic peripheral neuropathy, 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 #3 (Hold).

Some better-ranked stocks in the biotech sector are Illumina, Inc. ILMN, Krystal Biotech, Inc. KRYS and Fulcrum Therapeutics, Inc. FULC, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Illumina’s 2024 EPS have moved up from $1.84 to $3.63. The consensus estimate for 2025 earnings has improved from $3.22 to $4.43. Year to date, shares of ILMN have lost 5.9%.

ILMN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 463.46%.

In the past 60 days, estimates for Krystal Biotech’s 2024 EPS have increased from $2.09 to $2.38. The consensus estimate for 2025 earnings has improved from $4.33 to $7.31. Year to date, shares of KRYS have jumped 47.3%.

KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 45.95%.

In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from $1.33 to 28 cents. The consensus estimate for 2025 loss per share has narrowed from $1.71 to $1.14. Year to date, shares of FULC have plunged 49%.

FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.

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