Core Viewpoint - Applied Therapeutics, Inc. is focused on advancing its lead drug candidate, govorestat, for the treatment of rare diseases, particularly CMT-SORD and Classic Galactosemia, while preparing for upcoming regulatory interactions and clinical data presentations [2][6]. Recent Highlights - The company will present full 12-month clinical data and new topline 18-month and 24-month data from the INSPIRE Phase 3 trial of govorestat at the Peripheral Nerve Society 2025 Annual Meeting [5][6]. - Ongoing reviews of govorestat development programs for Classic Galactosemia and CMT-SORD Deficiency are progressing as planned [5][6]. Financial Results - Cash and cash equivalents and short-term investments totaled $50.8 million as of March 31, 2025, down from $79.4 million at December 31, 2024 [6]. - Research and development expenses for Q1 2025 were $7.8 million, a decrease from $12.2 million in Q1 2024, primarily due to reduced clinical and regulatory expenses [6]. - General and administrative expenses increased to $17.7 million in Q1 2025 from $9.1 million in Q1 2024, mainly due to higher legal and professional costs [6]. - The net loss for Q1 2025 was $21.8 million, or $0.15 per share, compared to a net loss of $83.9 million, or $0.67 per share, in Q1 2024 [6][13].

Core Insights - Applied Therapeutics, Inc. announced the presentation of full 12-month clinical results from the INSPIRE Phase 3 trial of govorestat (AT-007) at the Peripheral Nerve Society 2025 Annual Meeting, along with new topline 18-month and 24-month data [1] - The INSPIRE Phase 3 trial is a double-blind, placebo-controlled study evaluating govorestat's effect on patients with Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease [1] - Govorestat is a central nervous system penetrant Aldose Reductase Inhibitor being developed for multiple rare diseases, including CMT-SORD, Classic Galactosemia, and PMM2-CDG [2][4] Presentation Details - The oral presentation titled "INSPIRE CMT-SORD Clinical Trial: Month 18 and Month 24 INSPIRE Trial Results and Designing the Next Randomized Study" will be presented by Dr. Evan Bailey on May 18, 2025 [2] - The presentation is scheduled for 12:25 - 12:40 p.m. BST [2] Regulatory Designations - Govorestat has received Orphan Medicinal Product Designation from the European Medicines Agency for both Galactosemia and CMT disease [3] - It has also received Orphan Drug Designation from the U.S. FDA for Galactosemia, PMM2 deficiency, and SORD Deficiency, along with Rare Pediatric Disease designation for Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia [3] Company Overview - Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel drug candidates targeting rare diseases [4] - The lead drug candidate, govorestat, is aimed at treating CNS rare metabolic diseases, including Classic Galactosemia, CMT-SORD, and PMM2-CDG [4]

Rhythm Pharmaceuticals, Inc. (RYTM) came out with a quarterly loss of $0.81 per share versus the Zacks Consensus Estimate of a loss of $0.69. This compares to loss of $2.35 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -17.39%. A quarter ago, it was expected that this company would post a loss of $0.69 per share when it actually produced a loss of $0.72, delivering a surprise of -4.35%.Over the last four quarters, the compan ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-38898 Applied Therapeutics, Inc. (Exact name of registrant as specified in its charter) Delaware 81-3405262 (State ...

SIGA Tech则收涨11.07%，CorMedix涨11.70%，Pacira Pharmaceuticals涨14.71%。 | D 名称 ÷ | | 最新 ⇒ | 醫學 → | 磨促 ⇒ | 涨跌幅 ⇒ | 交易量 | 看涨 P | 时间 ÷ | | --- | --- | --- | --- | --- | --- | --- | --- | --- | | ■ | 트 Pacira Pharmaceuticals | 26.28 | 27.64 | 25.51 | +14.71% | 3.7M | ■日 | 03:59:59 0 | | D | | | | | | | | | | | = CorMedix | 6.780 | 7.700 | 6.695 | +11.70% | 3.95M | 印刷台 | 03:59:59 0 | | D | SIGA Tech | 5.920 | 6.400 | 5.590 | +11.07% | 1.15M | ■■合 | 03:28:20 G | | L | 三 阿玛琳 | 0.430 | 0.460 | 0.396 | +5.34% | 3.05M | 图图 ...

NEW YORK, Jan. 30, 2025 (GLOBE NEWSWIRE) -- The law firm of Kirby McInerney LLP reminds investors of the February 18, 2025, deadline to seek the role of lead plaintiff in a federal securities class action filed on behalf of investors who acquired Applied Therapeutics, Inc. (“Applied Therapeutics” or the “Company”) (NASDAQ:APLT) securities during the period from January 3, 2024, through December 2, 2024 (“the Class Period”). [LEARN MORE ABOUT THE CLASS ACTION] On November 27, 2024, Applied Therapeutics issu ...

Core Viewpoint - A class action securities lawsuit has been filed against Applied Therapeutics, Inc. due to alleged securities fraud affecting investors between January 3, 2024, and December 2, 2024 [1] Group 1: Case Details - On November 27, 2024, Applied Therapeutics announced it received a Complete Response Letter (CRL) from the FDA for its New Drug Application (NDA) for govorestat, indicating deficiencies in the clinical application [2] - Following the CRL announcement, the stock price of Applied Therapeutics dropped from $10.21 per share on November 26, 2024, to $8.57 on November 27, 2024, and further declined to $2.03 on November 29, 2024, and $1.75 on December 2, 2024, marking a total decline of over 80% [2] - After disclosing a "warning letter" from the FDA on December 2, 2024, regarding clinical trial issues, the stock price fell further to $1.69 on December 3, 2024, and continued to decline to $1.38 on December 4, 2024, and $1.29 on December 5, 2024 [2] Group 2: Next Steps for Investors - Investors who suffered losses during the specified timeframe have until February 18, 2025, to request the Court to appoint them as lead plaintiff, although participation in any recovery does not require serving as a lead plaintiff [3] - Class members may be entitled to compensation without any out-of-pocket costs or fees [3] Group 3: Firm Background - Levi & Korsinsky, LLP has a history of securing hundreds of millions of dollars for shareholders and has been recognized as one of the top securities litigation firms in the United States for seven consecutive years [4]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________ to ______________ | --- | --- | |----------------------------------------------------------------------------|------------- ...

Exhibit 99.1 Applied Therapeutics Reports Third Quarter 2024 Financial Results - NDA and MAA for govorestat for treatment of Classic Galactosemia under FDA Priority Review and EMA review; PDUFA target action date of November 28, 2024, and EMA decision expected in Q1 2025 - NDA submission for govorestat for the treatment of SORD Deficiency under Accelerated Approval expected in early Q1 2025 NEW YORK, November 7, 2024 – Applied Therapeutics, Inc. (Nasdaq: APLT) (the "Company"), a clinical-stage biopharmaceut ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________ to ______________ Commission File Number: 001-38898 Applied Therapeutics, Inc. (Exact Name of Registrant as Specified in its Cha ...