Applied Therapeutics will present new data from the INSPIRE trial for govorestat at the PNS Annual Meeting in May 2025.
Quiver AI Summary
Applied Therapeutics, Inc. announced that it will present the full 12-month clinical results from the INSPIRE Phase 3 trial of its drug govorestat (AT-007) at the Peripheral Nerve Society Annual Meeting in Edinburgh from May 17-20, 2025. The presentation will also feature new topline data at 18 and 24 months, prior to transitioning to an open-label extension study. The trial evaluates govorestat for treating Sorbitol Dehydrogenase Deficiency, a form of Charcot-Marie-Tooth disease. Govorestat has been recognized with various orphan drug designations by regulatory agencies for multiple rare diseases. The company emphasizes its commitment to developing innovative treatments for rare diseases, despite potential risks outlined in its forward-looking statements.
Potential Positives
- Announcement of full 12-month clinical results from the INSPIRE Phase 3 trial selected for oral presentation at a prominent medical conference, indicating strong scientific validation.
- Presentation will include new topline 18-month and 24-month data, highlighting ongoing research and potential efficacy over a longer timeframe.
- Govorestat, the lead drug candidate, has received multiple designations from regulatory bodies, underscoring its promise in treating rare diseases and enhancing the company's credibility.
Potential Negatives
- The reliance on forward-looking statements introduces significant uncertainty regarding the future success of their drug candidate, govorestat, leaving investors questioning the viability of the company's plans and objectives.
- The company's inability to guarantee regulatory approval or successful development of their product candidates raises concerns about the potential for delays or failures in bringing their treatments to market.
- The potential need for additional financing may indicate weaknesses in the company’s financial position, which could affect its ability to sustain research and development initiatives.
FAQ
What is the INSPIRE Phase 3 trial about?
The INSPIRE Phase 3 trial evaluates the effect of govorestat in patients with Sorbitol Dehydrogenase Deficiency, a subtype of CMT disease.
When will the 12-month clinical results be presented?
The full 12-month clinical results will be presented at the PNS 2025 Annual Meeting from May 17-20, 2025.
Who is presenting the INSPIRE trial results?
Evan Bailey, MD, Senior Vice President of Clinical Development at Applied Therapeutics, will present the results.
What are the designations received by govorestat?
Govorestat has received Orphan Medicinal Product Designation and Orphan Drug Designation from the EMA and FDA, respectively.
What diseases is govorestat being developed to treat?
Govorestat is being developed for treating Charcot-Marie-Tooth disease, Classic Galactosemia, and PMM2-CDG.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
$APLT Insider Trading Activity
$APLT insiders have traded $APLT stock on the open market 6 times in the past 6 months. Of those trades, 0 have been purchases and 6 have been sales.
Here’s a breakdown of recent trading of $APLT stock by insiders over the last 6 months:
- RICCARDO PERFETTI (Chief Medical Officer) has made 0 purchases and 2 sales selling 96,444 shares for an estimated $57,068.
- LESLIE D. FUNTLEYDER (See Remarks) has made 0 purchases and 2 sales selling 37,452 shares for an estimated $20,380.
- CONSTANTINE CHINOPOROS (See Remarks) has made 0 purchases and 2 sales selling 28,564 shares for an estimated $17,348.
To track insider transactions, check out Quiver Quantitative's insider trading dashboard.
$APLT Hedge Fund Activity
We have seen 64 institutional investors add shares of $APLT stock to their portfolio, and 87 decrease their positions in their most recent quarter.
Here are some of the largest recent moves:
- JANUS HENDERSON GROUP PLC removed 10,505,737 shares (-99.7%) from their portfolio in Q4 2024, for an estimated $8,995,012
- VESTAL POINT CAPITAL, LP added 10,355,000 shares (+904.4%) to their portfolio in Q4 2024, for an estimated $8,865,951
- SCHONFELD STRATEGIC ADVISORS LLC added 8,419,781 shares (+2458.3%) to their portfolio in Q4 2024, for an estimated $7,209,016
- PERCEPTIVE ADVISORS LLC removed 7,145,000 shares (-89.3%) from their portfolio in Q4 2024, for an estimated $6,117,549
- T. ROWE PRICE INVESTMENT MANAGEMENT, INC. added 5,213,370 shares (+2511.9%) to their portfolio in Q4 2024, for an estimated $4,463,687
- FRANKLIN RESOURCES INC removed 4,797,510 shares (-70.5%) from their portfolio in Q4 2024, for an estimated $4,107,628
- PROPEL BIO MANAGEMENT, LLC added 3,047,562 shares (+46.4%) to their portfolio in Q4 2024, for an estimated $2,609,322
To track hedge funds' stock portfolios, check out Quiver Quantitative's institutional holdings dashboard.
$APLT Price Targets
Multiple analysts have issued price targets for $APLT recently. We have seen 2 analysts offer price targets for $APLT in the last 6 months, with a median target of $4.5.
Here are some recent targets:
- Brian Skorney from Robert W. Baird set a target price of $5.0 on 11/29/2024
- Brian Abrahams from RBC Capital set a target price of $4.0 on 11/29/2024
Full Release
NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT) (the “Company”), a clinical-stage biopharmaceutical company dedicated to creating transformative treatments for rare diseases, today announced that it will present full 12-month clinical results from the INSPIRE Phase 3 trial of govorestat (AT-007) in a late-breaking abstract selected for oral presentation at the Peripheral Nerve Society (PNS) 2025 Annual Meeting, being held May 17-20, 2025 in Edinburgh, Scotland. The presentation will also include new topline 18-month and 24-month data prior to the transition to the open-label extension study. The INSPIRE Phase 3 trial was a double-blind, placebo-controlled registrational trial to evaluate the effect of govorestat in patients with Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease. The Company previously shared topline results from an analysis of 12-month group-level data from the INSPIRE trial in February 2024.
Presentation Details
Title:
INSPIRE CMT-SORD Clinical Trial: Month 18 and Month 24 INSPIRE Trial Results and Designing the Next Randomized Study
Oral Presentation Number:
597
Presenter:
Evan Bailey, MD, Senior Vice President, Clinical Development, Applied Therapeutics Inc.
Date and Time:
Sunday, May 18, 2025, 12:25 - 12:40 p.m. BST
About Govorestat (AT-007)
Govorestat is a central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI) being developed for the treatment of multiple rare diseases including Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD), Classic Galactosemia, and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG).
Govorestat has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for both Galactosemia and CMT disease. Govorestat has also received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Galactosemia, phosphomannomutase 2 deficiency, and SORD Deficiency; Rare Pediatric Disease designation for Galactosemia and PMM2-CDG; and Fast Track designation for Galactosemia.
About Applied Therapeutics
Applied Therapeutics is a clinical-stage biopharmaceutical company committed to the development of novel drug candidates against validated molecular targets in rare diseases. The Company’s lead drug candidate, govorestat, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Classic Galactosemia, Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD) and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG).
To learn more, please visit www.appliedtherapeutics.com and follow the company on X at @Applied_Tx.
Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, included in this press release regarding the strategy, future operations, prospects, plans and objectives of management, including words such as “may,” “will,” “expect,” “anticipate,” “plan,” “intend,” “predicts” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, expressed or implied statements regarding the (i) continued development and advancement of AT-007 and (ii) potential of AT-007 for the treatment of multiple rare diseases, including CMT-SORD, Classic Galactosemia and CDG. Forward-looking statements in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved.
Such risks and uncertainties include, without limitation, (i) our plans to develop, market and commercialize our product candidates, (ii) the initiation, timing, progress and results of our current and future preclinical studies and clinical trials and our research and development programs, (iii) our ability to take advantage of expedited regulatory pathways for any of our product candidates, (iv) our estimates regarding expenses, future revenue, capital requirements and needs for additional financing, (v) our ability to successfully acquire or license additional product candidates on reasonable terms and advance product candidates into, and successfully complete, clinical studies, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain and timing of regulatory approval of our current and future product candidates, (viii) the anticipated indications for our product candidates, if approved, (ix) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (x) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (xi) the implementation of our business model and strategic plans for our business and product candidates, (xii) our intellectual property position and the duration of our patent rights, (xiii) developments or disputes concerning our intellectual property or other proprietary rights, (xiv) our expectations regarding government and third-party payor coverage and reimbursement, (xv) our ability to compete in the markets we serve, (xvi) the impact of government laws and regulations and liabilities thereunder, (xvii) developments relating to our competitors and our industry, (xviii) our ability to achieve the anticipated benefits from the agreements entered into in connection with our partnership with Advanz Pharma and (xix) other factors that may impact our financial results. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur at all. Factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in our filings with the U.S. Securities and Exchange Commission, including those described in the “Risk Factors” section contained therein. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.
Contacts
Investors:
Julie Seidel / Andrew Vulis
(212) 600-1902 or
[email protected]
Media:
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