A RandomIzed, Double-Blind, Placebo-CoNtrolled, Two-Part Study to Evaluate the Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With SoRbitol Dehydrogenase (SORD) DEficiency

This study is designed to assess the efficacy and safety of AT-007 treatment in patients with SORD Deficiency. This randomized, double-blind study will assess the effect of AT-007 compared to Placebo in SORD Deficiency patients for 24 months.

Start Date01 Jan 2022

Sponsor / Collaborator

Applied Therapeutics, Inc.

NCT05418829

/ Unknown statusPhase 3

An Open-Label Study to Evaluate the Long-Term Safety and Pharmacodynamic Efficacy of AT-007 in Adult Subjects With Classic Galactosemia (CG)

This study is a 12-month open-label extension (OLE) study of AT-007 in adult subjects with CG who previously participated in Study AT-007-1001 Part D and/or Part D Extension.
The study is designed to assess the long-term safety of AT-007 in subjects with CG as well as the pharmacodynamics (PD) (inhibition of galactitol) and PK of AT-007. The effect of 12-month treatment with AT-007 on the levels of galactose and other galactose metabolites in subjects with CG will also be evaluated.

Start Date01 Oct 2021

Sponsor / Collaborator

Applied Therapeutics, Inc.

NCT04902781

/ Active, not recruitingPhase 2/3

A Sequential, Two-Part Study to Evaluate the Clinical Benefit, Safety, Pharmacokinetics, and Pharmacodynamics of AT-007 in Pediatric Patients With Classic Galactosemia (CG)

This study is designed to assess the clinical benefit as well as the safety, pharmacokinetics (PK), and pharmacodynamics (PD) (reduction of galactitol levels) of AT-007 in pediatric subjects with Classic Galactosemia (CG).

Start Date20 Mar 2021

Sponsor / Collaborator

Applied Therapeutics, Inc.

100 Clinical Results associated with Govorestat

100 Translational Medicine associated with Govorestat

100 Patents (Medical) associated with Govorestat

Literatures (Medical) associated with Govorestat

01 Nov 2024·The Journal of Clinical Pharmacology

Safety, Pharmacokinetics, and Pharmacodynamics of the New Aldose Reductase Inhibitor Govorestat (AT‐007) After a Single and Multiple Doses in Participants in a Phase 1/2 Study

Article

Author: Shendelman, Shoshana ; Perfetti, Riccardo ; Mohanlal, Ramon ; Bailey, Evan ; Wang, Stella ; Mills, Richard

AbstractIn classic galactosemia (CG) patients, aldose reductase (AR) converts galactose to galactitol. In a phase 1/2, placebo‐controlled study (NCT04117711), safety, pharmacokinetics (PK), and pharmacodynamics (PD) of govorestat were evaluated after single and multiple ascending doses (0.5‐40 mg/kg) in healthy adults (n = 81) and CG patients (n = 14). Levels of govorestat in plasma and cerebrospinal fluid (CSF) and blood levels of galactitol, galactose, and galactose‐1‐phosphate (Gal‐1p) were measured for population PK and PK/PD analyses. Govorestat was well tolerated. Adverse event frequency was comparable between placebo and govorestat. Govorestat PK displayed a 2‐compartment model with sequential zero‐ and first‐order absorption, and no effect of demographic factors. Multiple‐dose PK of govorestat was linear in the 0.5‐40 mg/kg range, and CSF levels increased dose dependently. Elimination half‐life was ∼10 h. PK/PD modeling supported once‐daily dosing. Change from baseline in galactitol was −15% ± 9% with placebo and −19% ± 10%, −46% ± 4%, and −51% ± 5% with govorestat 5, 20, and 40 mg/kg, respectively, thus was similar for 20 and 40 mg/kg. Govorestat did not affect galactose or Gal‐1p levels. In conclusion, govorestat displayed a favorable safety, PK, and PD profile in humans, and reduced galactitol levels in the same magnitude (∼50%) as in a rat model of CG that demonstrated an efficacy benefit on neurological, behavioral, and ocular outcomes.

22 May 2023·JCI insight

Sorbitol reduction via govorestat ameliorates synaptic dysfunction and neurodegeneration in sorbitol dehydrogenase deficiency.

Article

Author: Züchner, Stephan ; Rebelo, Adriana P ; Yanick, Christopher ; Saporta, Mario ; Ortiz-Vega, Natalie ; Zhu, Yi ; Zhai, R Grace ; Shendelman, Shoshana ; Lobato, Amanda G ; Shy, Michael ; Canic, Tijana ; Perfetti, Riccardo ; Syed, Sheyum ; Tao, Xianzun

Sorbitol dehydrogenase (SORD) deficiency has been identified as the most frequent autosomal recessive form of hereditary neuropathy. Loss of SORD causes high sorbitol levels in tissues due to the inability to convert sorbitol to fructose in the 2-step polyol pathway, leading to degenerative neuropathy. The underlying mechanisms of sorbitol-induced degeneration have not been fully elucidated, and no current FDA-approved therapeutic options are available to reduce sorbitol levels in the nervous system. Here, in a Drosophila model of SORD deficiency, we showed synaptic degeneration in the brain, neurotransmission defect, locomotor impairment, and structural abnormalities in the neuromuscular junctions. In addition, we found reduced ATP production in the brain and ROS accumulation in the CNS and muscle, indicating mitochondrial dysfunction. Applied Therapeutics has developed a CNS-penetrant next-generation aldose reductase inhibitor (ARI), AT-007 (govorestat), which inhibits the conversion of glucose to sorbitol. AT-007 significantly reduced sorbitol levels in patient-derived fibroblasts, induced pluripotent stem cell-derived (iPSC-derived) motor neurons, and Drosophila brains. AT-007 feeding in Sord-deficient Drosophila mitigated synaptic degeneration and significantly improved synaptic transduction, locomotor activity, and mitochondrial function. Moreover, AT-007 treatment significantly reduced ROS accumulation in Drosophila CNS, muscle, and patient-derived fibroblasts. These findings uncover the molecular and cellular pathophysiology of SORD neuropathy and provide a potential treatment strategy for patients with SORD deficiency.

01 Jan 2017·Journal of Pathogens

A Laboratory Assessment of Two Local Strains of the Beauveria bassiana (Bals.) Vuill. against the Tetranychus urticae (Acari: Tetranychidae) and Their Potential as a Mycopesticide

Article

Author: Algur, Omer Faruk ; Ortucu, Serkan

This study was conducted to assess highly pathogenic Beauveria bassiana isolates to be used in biocontrol and to determine their potentials as mycopesticide. For this purpose, two B. bassiana isolates, which were locally isolated from T. urticae, were chosen. Firstly, three suspensions were investigated at the degree of humidity of 65 ± 5% and 100% RH. Secondly, these strains were selected according to their tendency to mass production, tolerance to UV radiation, and capability of producing spore at the different temperatures. Finally, identification of the selected isolate was performed by using ITS rDNA analysis. Both tested fungal isolates were pathogenic to the T. urticae. Mycelial growths of isolate AT076 at 20°C and 30°C were found to be greater than isolate AT007. It was observed that isolate AT076 had more spore production with 1.61 × 107 spore/disc at 30°C and 44.33% germination after UV radiation for 15 min. The numbers of spores per 5 mm disk area for isolates AT076 and AT007 were found to be 1.2 × 106 and 1.0 × 106. These results show that isolate AT076 was more virulent and more UV-tolerant and had higher tendency to mass production compared to isolate AT007 against T. urticae. As a result of this study, isolate AT076 can be used in the biocontrol as mycopesticide.

News (Medical) associated with Govorestat

20 Jan 2025

·www.prnewswire.com

Shareholders that lost money on Applied Therapeutics, Inc.(APLT) Urged to Join Class Action - Contact The Gross Law Firm to Learn More

NEW YORK, Jan. 20, 2025 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Applied Therapeutics, Inc. (NASDAQ: APLT). Shareholders who purchased shares of APLT during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any recovery. CONTACT US HERE: CLASS PERIOD: January 3, 2024 to December 2, 2024 ALLEGATIONS: According to the complaint, on November 27, 2024, Applied Therapeutics issued a press release announcing that it had received a Complete Response Letter (CRL) for the New Drug Application (NDA) for govorestat, the Company's lead drug candidate. The CRL indicated that the FDA completed its review of the application and determined that it was unable to approve the NDA in its current form citing deficiencies in the clinical application. Following this news, the price of Applied Therapeutics' common stock declined dramatically. From a closing market price of $10.21 per share on November 26, 2024, Applied Therapeutics' stock price fell to $8.57 per share on November 27, 2024 before falling further to $2.03 on November 29, 2024 and $1.75 per share on December 2, 2024, a total decline of more than 80%. After market hours on December 2, 2024, Applied Therapeutics disclosed it received a "warning letter" from the FDA referring to the clinical trial issues underlying the CRL. Applied Therapeutics' disclosure of the "warning letter" prompted a further decline in the stock price as investors discovered the seriousness and severity of the Company's clinical trial errors. From a closing market price of $1.75 per share on December 2, 2024, Applied Therapeutics' stock price fell to $1.69 per share on December 3, 2024 before falling further to $1.38 per share on December 4, 2024 and $1.29 per share on December 5, 2024. DEADLINE: February 18, 2025 Shareholders should not delay in registering for this class action. Register your information here: NEXT STEPS FOR SHAREHOLDERS: Once you register as a shareholder who purchased shares of APLT during the timeframe listed above, you will be enrolled in a portfolio monitoring software to provide you with status updates throughout the lifecycle of the case. The deadline to seek to be a lead plaintiff is February 18, 2025. There is no cost or obligation to you to participate in this case. WHY GROSS LAW FIRM? The Gross Law Firm is a nationally recognized class action law firm, and our mission is to protect the rights of all investors who have suffered as a result of deceit, fraud, and illegal business practices. The Gross Law Firm is committed to ensuring that companies adhere to responsible business practices and engage in good corporate citizenship. The firm seeks recovery on behalf of investors who incurred losses when false and/or misleading statements or the omission of material information by a company lead to artificial inflation of the company's stock. Attorney advertising. Prior results do not guarantee similar outcomes. CONTACT: The Gross Law Firm 15 West 38th Street, 12th floor New York, NY, 10018 Email: [email protected] Phone: (646) 453-8903 SOURCE The Gross Law Firm WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NDA

16 Jan 2025

·www.prnewswire.com

The Gross Law Firm Notifies Shareholders of Applied Therapeutics, Inc.(APLT) of a Class Action Lawsuit and an Upcoming Deadline

NEW YORK, Jan. 16, 2025 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Applied Therapeutics, Inc. (NASDAQ: APLT). Shareholders who purchased shares of APLT during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any recovery. CONTACT US HERE: CLASS PERIOD: January 3, 2024 to December 2, 2024 ALLEGATIONS: According to the complaint, on November 27, 2024, Applied Therapeutics issued a press release announcing that it had received a Complete Response Letter (CRL) for the New Drug Application (NDA) for govorestat, the Company's lead drug candidate. The CRL indicated that the FDA completed its review of the application and determined that it was unable to approve the NDA in its current form citing deficiencies in the clinical application. Following this news, the price of Applied Therapeutics' common stock declined dramatically. From a closing market price of $10.21 per share on November 26, 2024, Applied Therapeutics' stock price fell to $8.57 per share on November 27, 2024 before falling further to $2.03 on November 29, 2024 and $1.75 per share on December 2, 2024, a total decline of more than 80%. After market hours on December 2, 2024, Applied Therapeutics disclosed it received a "warning letter" from the FDA referring to the clinical trial issues underlying the CRL. Applied Therapeutics' disclosure of the "warning letter" prompted a further decline in the stock price as investors discovered the seriousness and severity of the Company's clinical trial errors. From a closing market price of $1.75 per share on December 2, 2024, Applied Therapeutics' stock price fell to $1.69 per share on December 3, 2024 before falling further to $1.38 per share on December 4, 2024 and $1.29 per share on December 5, 2024. DEADLINE: February 18, 2025 Shareholders should not delay in registering for this class action. Register your information here: NEXT STEPS FOR SHAREHOLDERS: Once you register as a shareholder who purchased shares of APLT during the timeframe listed above, you will be enrolled in a portfolio monitoring software to provide you with status updates throughout the lifecycle of the case. The deadline to seek to be a lead plaintiff is February 18, 2025. There is no cost or obligation to you to participate in this case. WHY GROSS LAW FIRM? The Gross Law Firm is a nationally recognized class action law firm, and our mission is to protect the rights of all investors who have suffered as a result of deceit, fraud, and illegal business practices. The Gross Law Firm is committed to ensuring that companies adhere to responsible business practices and engage in good corporate citizenship. The firm seeks recovery on behalf of investors who incurred losses when false and/or misleading statements or the omission of material information by a company lead to artificial inflation of the company's stock. Attorney advertising. Prior results do not guarantee similar outcomes. CONTACT: The Gross Law Firm 15 West 38th Street, 12th floor New York, NY, 10018 Email: [email protected] Phone: (646) 453-8903 SOURCE The Gross Law Firm WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NDA

14 Jan 2025

·www.prnewswire.com

Levi & Korsinsky Reminds Shareholders of a Lead Plaintiff Deadline of February 18, 2025 in Applied Therapeutics, Inc. Lawsuit - APLT

NEW YORK, Jan. 14, 2025 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in Applied Therapeutics, Inc. ("Applied Therapeutics" or the "Company") (NASDAQ: APLT) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of Applied Therapeutics investors who were adversely affected by alleged securities fraud between January 3, 2024 and December 2, 2024. Follow the link below to get more information and be contacted by a member of our team: APLT investors may also contact Joseph E. Levi, Esq. via email at [email protected] or by telephone at (212) 363-7500. CASE DETAILS: According to the complaint, on November 27, 2024, Applied Therapeutics issued a press release announcing that it had received a Complete Response Letter (CRL) for the New Drug Application (NDA) for govorestat, the Company's lead drug candidate. The CRL indicated that the FDA completed its review of the application and determined that it was unable to approve the NDA in its current form citing deficiencies in the clinical application. Following this news, the price of Applied Therapeutics' common stock declined dramatically. From a closing market price of $10.21 per share on November 26, 2024, Applied Therapeutics' stock price fell to $8.57 per share on November 27, 2024 before falling further to $2.03 on November 29, 2024 and $1.75 per share on December 2, 2024, a total decline of more than 80%. After market hours on December 2, 2024, Applied Therapeutics disclosed it received a "warning letter" from the FDA referring to the clinical trial issues underlying the CRL. Applied Therapeutics' disclosure of the "warning letter" prompted a further decline in the stock price as investors discovered the seriousness and severity of the Company's clinical trial errors. From a closing market price of $1.75 per share on December 2, 2024, Applied Therapeutics' stock price fell to $1.69 per share on December 3, 2024 before falling further to $1.38 per share on December 4, 2024 and $1.29 per share on December 5, 2024. WHAT'S NEXT? If you suffered a loss in Applied Therapeutics during the relevant time frame, you have until February 18, 2025 to request that the Court appoint you as lead plaintiff. Your ability to share in any recovery doesn't require that you serve as a lead plaintiff. NO COST TO YOU: If you are a class member, you may be entitled to compensation without payment of any out-of-pocket costs or fees. There is no cost or obligation to participate. WHY LEVI & KORSINSKY: Over the past 20 years, the team at Levi & Korsinsky has secured hundreds of millions of dollars for aggrieved shareholders and built a track record of winning high-stakes cases. Our firm has extensive expertise representing investors in complex securities litigation and a team of over 70 employees to serve our clients. For seven years in a row, Levi & Korsinsky has ranked in ISS Securities Class Action Services' Top 50 Report as one of the top securities litigation firms in the United States. CONTACT: Levi & Korsinsky, LLP Joseph E. Levi, Esq. Ed Korsinsky, Esq. 33 Whitehall Street, 17th Floor New York, NY 10004 [email protected] Tel: (212) 363-7500 Fax: (212) 363-7171 SOURCE Levi & Korsinsky, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NDA

100 Deals associated with Govorestat

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Galactosemias	NDA/BLA	EU	Applied Therapeutics, Inc.	01 Dec 2023
Sorbitol dehydrogenase deficiency	Phase 3	GB	Applied Therapeutics, Inc.	01 Jan 2022
Sorbitol dehydrogenase deficiency	Phase 3	US	Applied Therapeutics, Inc.	01 Jan 2022
Sorbitol dehydrogenase deficiency	Phase 3	IT	Applied Therapeutics, Inc.	01 Jan 2022
Sorbitol dehydrogenase deficiency	Phase 3	CZ	Applied Therapeutics, Inc.	01 Jan 2022
Congenital Disorder of Glycosylation Type 1A	Phase 1	-	Applied Therapeutics, Inc.	-

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05397665 (INSPIRE, Biospace) Manual	Phase 2/3	Sorbitol dehydrogenase deficiency	56	Govorestat	(ltvgifjilq) = Demonstrated statistically significant correlation between sorbitol level and the prespecified CMT-FOM composite clinical endpoint (10-meter walk-run test, 4 stair climb, sit to stand test,6-minute walk test and dorsiflexion) (p=0.05). tdwzworeyx (kqjdamiqvc ) Met View more	Positive	15 Feb 2024
				Placebo
Corporate Publications Manual	Phase 3	Galactosemias	-	AT-007	rkkgxokhto(ovgknxsoon) = Review of the data at 12 months of treatment by the DMC indicated that while the study primary endpoint has not yet reached statistical significance, a trend exists favoring AT-007 vs. placebo. xcbtxdqgdm (jkmddxmxoy )	Positive	06 Oct 2022
				Placebo

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