APLT Applied Therapeutics Inc.

10.88
-0.23  -2%
Previous Close 11.11
Open 10.67
52 Week Low 10.22
52 Week High 29.8
Market Cap $285,224,792
Shares 26,215,514
Float 17,754,207
Enterprise Value $185,865,360
Volume 73,455
Av. Daily Volume 93,417
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Upcoming Catalysts

Drug Stage Catalyst Date
AT-007 (ACTION-Galactosemia kids)
Galactosemia
NDA Filing
NDA Filing
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AT-001 (ARISE-HF)
Diabetic cardiomyopathy (DbCM)
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
AT-007
Sorbitol Dehydrogenase Deficiency (SORD Deficiency)
Phase 2
Phase 2
Phase 2 trial biomarker data demonstrated substantial and significant reduction in sorbitol, noted October 25, 2021. Registrational trial expected to initiated YE 2021.

Latest News

  1. NEW YORK, Nov. 18, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, has announced a symposium and poster presentations at the 14th International Congress of Inborn Errors of Metabolism (ICIEM), November 21-23, 2021, Sydney, Australia.

    "We are pleased to present this important data at the ICIEM conference, demonstrating statistically significant correlation between toxic galactitol level and disease severity," said Shoshana Shendelman, Chief Executive Officer of Applied Therapeutics. "Additionally, new data on burden of illness in adults with Galactosemia…

    NEW YORK, Nov. 18, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, has announced a symposium and poster presentations at the 14th International Congress of Inborn Errors of Metabolism (ICIEM), November 21-23, 2021, Sydney, Australia.

    "We are pleased to present this important data at the ICIEM conference, demonstrating statistically significant correlation between toxic galactitol level and disease severity," said Shoshana Shendelman, Chief Executive Officer of Applied Therapeutics. "Additionally, new data on burden of illness in adults with Galactosemia demonstrates that the disease continues to progress through adulthood and has substantial impact of quality of life."

    Presentation Details

    Poster #504: Progressive Worsening of the Central Nervous System Phenotype in Children with Classic Galactosemia on Galactose Restricted Diet: A Cross-Sectional Analysis

    Poster #573: Qualitative Interviews of Adults with Classic Galactosemia (CG) and their Caregivers: Disease Burden and Challenges with Daily Living

    Poster #574: Galactitol Level is a Predictor of Disease Severity in Children with Classic Galactosemia on Galactose Restricted Diet

    Symposium: Targeting Toxic Galactitol for the Treatment of Classic Galactosemia

    • Date and Time: November 23, 1:30 – 2:30 PM AEDT
    • Speakers: Shoshana Shendelman, CEO and Founder of Applied Therapeutics; Riccardo Perfetti, Chief Medical Officer at Applied Therapeutics; Laura Saltonstall, VP, Medical Affairs at Applied Therapeutics

    About Applied Therapeutics

    Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company's lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of Diabetic retinopathy, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.

    To learn more, please visit www.appliedtherapeutics.com and follow the company on Twitter @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 strategy, future operations, prospects, plans and objectives of management, including words such as "may," "will," "expect," "anticipate," "plan," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. 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, 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 the "Risk Factors" 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:

    Maghan Meyers

    (212) 600-1902 or

    Media:



    Applied Therapeutics, Inc.



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  2. Data from pilot trial of AT-007 in SORD deficiency expresses substantial and significant reduction in sorbitol

    Data from the ACTION-Galactosemia Kids study demonstrates statistically significant reduction in plasma galactitol

    NEW YORK, Nov. 12, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported financial results for the third quarter ended September 30, 2021.

    "This quarter we announced positive data in both the Galactosemia pediatric study as well as our pilot SORD Deficiency study, highlighting our execution across programs and expansion…

    Data from pilot trial of AT-007 in SORD deficiency expresses substantial and significant reduction in sorbitol

    Data from the ACTION-Galactosemia Kids study demonstrates statistically significant reduction in plasma galactitol

    NEW YORK, Nov. 12, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported financial results for the third quarter ended September 30, 2021.

    "This quarter we announced positive data in both the Galactosemia pediatric study as well as our pilot SORD Deficiency study, highlighting our execution across programs and expansion into additional indications with AT-007," said Shoshana Shendelman, PhD, CEO, Founder and Chair of the Board of Applied Therapeutics. "We are excited to launch our registrational study in SORD later this year and remain focused on preparations for our anticipated commercial launch in Galactosemia in 2022."

    Recent Highlights

    • Reported biomarker data from pilot trial of AT-007 in SORD deficiency. In October 2021, the Company highlighted results from a pilot open-label study in 8 SORD Deficiency patients. In the study, AT-007 reduced blood sorbitol levels by approximately 66% from baseline through 30 days of treatment with a range of reduction from baseline of 54%-75%. AT-007 was safe and well tolerated in all patients. The Company plans to initiate a registrational study by the end of 2021.



    • Reported pediatric biomarker data from ACTION-Galactosemia Kids. In October 2021, the Company reported pediatric biomarker data from the ACTION-Galactosemia Kids study. The results demonstrated a substantial reduction in plasma galactitol of approximately 40%, which was statistically significant (p<0.001) vs. placebo. Additionally, analysis of the 47 children in the ACTION-Galactosemia Kids study demonstrated a clear correlation between baseline galactitol level and baseline clinical functional outcomes. Children with higher plasma galactitol levels displayed greater disease severity vs. children with lower plasma galactitol levels at baseline. This data is the first demonstration of correlation of a biochemical biomarker with severity of disease in Galactosemia patients. This data will be presented as a late-breaking abstract at the 14th International Congress on Inborn Errors of Metabolism (ICIEM) November 21-24, 2021.

    Financial Results

    • Cash and cash equivalents and short-term investments totaled $108.8 million as of September 30, 2021, compared with $125.6 million at June 30, 2021.



    • Research and development expenses for the three months ended September 30, 2021 were $17.6 million, compared to $19.9 million for the three months ended September 30, 2020. The decrease of $2.3 million was related to a decrease in drug manufacturing and formulation costs of $5.2 million primarily related to the completion and release of AT-001 and AT-007 drug product batches in the three months ended March 31, 2021; an increase in clinical and pre-clinical expense of $2.1 million, primarily related to the progression of the AT-007 ACTION-Galactosemia adult extension study and the AT-007 ACTION-Galactosemia Kids pediatric registrational study; an increase in personnel expenses of $0.6 million due to the increase in headcount in support of our clinical program pipeline; and an increase in regulatory and other expenses of $0.2 million.



    • General and administrative expenses were $10.8 million for the three months ended September 30, 2021, compared to $10.0 million for the three months ended September 30, 2020. The increase of $0.8 million was primarily related to an increase in commercial expenses of $0.8 million related to the expansion of the commercial department; an increase in other expenses of $0.3 million relating to increased costs of rent and other office expenses; an increase in stock-based compensation of $0.2 million; an increase in insurance expenses of $0.1 million related to increased insurance costs; and a decrease in legal and professional fees $0.6 million due to lower external legal fees.



    • Net loss for the third quarter of 2021 was $28.4 million, or $1.09 per basic and diluted common share, compared to a net loss of $29.8 million, or $1.33 per basic and diluted common share, for the third quarter 2020.

    About Applied Therapeutics

    Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company's lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of Diabetic retinopathy, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.

    To learn more, please visit www.appliedtherapeutics.com and follow the company on Twitter @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 strategy, future operations, prospects, plans and objectives of management, including words such as "may," "will," "expect," "anticipate," "plan," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, statements regarding (i) the Company's plan to initiate a registrational study in SORD by the end of 2021, (ii) the anticipated commercial launch in Galactosemia in 2022, (iii) AT-007 potential to be the first disease-modifying therapy for SORD Deficiency, (iv) the timing of the initiation and completion of our clinical trials, (v) the likelihood that data from our clinical trials will support future development of our product candidates and (vi) the likelihood of obtaining regulatory approval of our product candidates. 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 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, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain regulatory approval of our current and future product candidates, (viii) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (ix) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (x) the implementation of our business model and strategic plans for our business and product candidates, (xi) our intellectual property position and the duration of our patent rights, (xii) developments or disputes concerning our intellectual property or other proprietary rights, (xiii) our expectations regarding government and third-party payor coverage and reimbursement, (xiv) our ability to compete in the markets we serve, (xv) the impact of government laws and regulations and liabilities thereunder, (xvi) developments relating to our competitors and our industry, (xvii) the impact of the COVID-19 pandemic on the timing and progress of our ongoing clinical trials and our business in general and (xviii) 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 the "Risk Factors" 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:

    Maghan Meyers

    (212) 600-1902 or

    Media:



    Applied Therapeutics, Inc.

    Statements of Operations

    (in thousands, except share and per share data)

    (Unaudited)

                  
      Three Months Ended  Nine Months Ended  
      September 30,  September 30,  
         2021     2020     2021     2020  
    OPERATING EXPENSES:             
    Research and development $17,597  $19,945  $46,846  $47,974  
    General and administrative  10,833   10,020   31,658   22,744  
    Total operating expenses  28,430   29,965   78,504   70,718  
    LOSS FROM OPERATIONS  (28,430)  (29,965)  (78,504)  (70,718) 
    OTHER INCOME (EXPENSE), NET:               
    Interest income (expense), net  76   131   321   435  
    Other income (expense)  (64)  (10)  (242)  11  
    Total other income (expense), net  12   121   79   446  
    Net loss $(28,418) $(29,844) $(78,425) $(70,272) 
    Net loss attributable to common stockholders—basic and diluted $(28,418) $(29,844) $(78,425) $(70,272) 
    Net loss per share attributable to common stockholders—basic and diluted $(1.09) $(1.33) $(3.08) $(3.22) 
    Weighted-average common stock outstanding—basic and diluted  26,177,079   22,426,203   25,472,590   21,790,207  

    Applied Therapeutics, Inc.

    Balance Sheets

    (in thousands, except share and per share data)

            
      As of As of 
      September 30,  December 31, 
      2021  2020  
      (Unaudited)   
    ASSETS       
    CURRENT ASSETS:         
    Cash and cash equivalents $70,207  $57,466  
    Investments  38,595   39,363  
    Prepaid expenses and other current assets  8,141   5,764  
    Total current assets  116,943   102,593  
    Operating lease right-of-use asset  1,404   1,712  
    Security deposits and leasehold improvements  200   201  
    TOTAL ASSETS $118,547  $104,506  
    LIABILITIES AND STOCKHOLDERS' EQUITY        
    CURRENT LIABILITIES:        
    Current portion of operating lease liabilities $436  $406  
    Accounts payable  9,170   640  
    Accrued expenses and other current liabilities  21,237   20,189  
    Total current liabilities  30,843   21,235  
    NONCURRENT LIABILITIES:       
    Noncurrent portion of operating lease liabilities  1,003   1,332  
    Total noncurrent liabilities  1,003   1,332  
    Total liabilities  31,846   22,567  
    STOCKHOLDERS' EQUITY:         
    Common stock, $0.0001 par value; 100,000,000 shares authorized as of September 30, 2021 and December 31, 2020; 26,214,399 shares and 22,493,661 shares issued and outstanding as of September 30, 2021 and December 31, 2020, respectively  3   2  
    Additional paid-in capital  326,108   242,780  
    Accumulated other comprehensive loss  (254)  (112) 
    Accumulated deficit  (239,156)  (160,731) 
    Total stockholders' equity  86,701   81,939  
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY $118,547  $104,506  


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  3. Substantial and significant reduction in sorbitol

    Company plans to initiate registrational trial by end of 2021

    Company to host conference call and webcast today at 8:30 a.m. ET

    NEW YORK, Oct. 25, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported biomarker data from a pilot trial of AT-007 in patients with SORD Deficiency.

    Sorbitol Dehydrogenase Deficiency (SORD Deficiency) is a rare, progressive, debilitating hereditary neuropathy that affects peripheral nerves and motor neurons. SORD Deficiency affects approximately 3,300 patients in the…

    Substantial and significant reduction in sorbitol

    Company plans to initiate registrational trial by end of 2021

    Company to host conference call and webcast today at 8:30 a.m. ET

    NEW YORK, Oct. 25, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported biomarker data from a pilot trial of AT-007 in patients with SORD Deficiency.

    Sorbitol Dehydrogenase Deficiency (SORD Deficiency) is a rare, progressive, debilitating hereditary neuropathy that affects peripheral nerves and motor neurons. SORD Deficiency affects approximately 3,300 patients in the US and 4,000 patients in Europe. The disease is caused by a lack of the enzyme sorbitol dehydrogenase, responsible for metabolism of sorbitol, which causes sorbitol to accumulate at high levels and become toxic to the body. Sorbitol accumulation results in significant disability, loss of sensory function, and neuromuscular dysfunction.

    Patients with SORD Deficiency have 100 times higher sorbitol concentration in their blood compared with unaffected individuals. In a pilot open-label study in 8 SORD Deficiency patients, AT-007 reduced blood sorbitol levels by approximately 66% from baseline through 30 days of treatment. The range of reduction from baseline in patients was 54%-75%. AT-007 was safe and well tolerated in all treated patients.  

    These results, in addition to preclinical findings, demonstrate that AT-007 has the potential to be the first disease-modifying therapy for SORD Deficiency. The Company plans to initiate a registrational study by the end of 2021. In advance of the registrational study start, patients can now pre-screen to determine whether they have SORD and if they may qualify for the upcoming trial.

    "Reduction in toxic sorbitol is critically important in patients with SORD Deficiency. This data demonstrates a significant effect on the underlying cause of the disease," said Michael Shy, MD, Professor of Neurology and Director of the Division of Neuromuscular Medicine at the University of Iowa Hospital Carver School of Medicine.

    "AT-007 represents an important advancement for patients with SORD Deficiency, and a unique opportunity to meaningfully impact patients' lives. We look forward to beginning our registrational trial for this indication in the coming months," said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics.

    Increased access to screening and early diagnosis can dramatically improve patients' lives, and the Company is collaborating with the Charcot Marie Tooth Association and the Hereditary Neuropathy Foundation to improve access to SORD diagnostic testing, and to better understand the perspectives of individuals living with SORD.

    Conference Call Information

    Applied Therapeutics will host a conference call today, Monday, October 25, 2021, at 8:30 a.m. Eastern Time, to discuss data from a pilot trial of AT-007 in SORD deficiency. To access the conference call, please dial (800) 369-8554 (local) or (409) 937-8917 (international) at least 10 minutes prior to the start time and refer to conference ID 2437605. A live webcast of the call will be accessible on the Events page under the Investor Relations section of the Applied Therapeutics website at www.appliedtherapeutics.com. A replay will be available on the Company's website approximately two hours after the event.

    About Applied Therapeutics

    Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company's lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of Diabetic retinopathy, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.

    To learn more, please visit www.appliedtherapeutics.com and follow the company on Twitter @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 strategy, future operations, prospects, plans and objectives of management, including words such as "may," "will," "expect," "anticipate," "plan," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, statements regarding (i) the Company's plan to initiate a registrational study by the end of 2021, (ii) AT-007 potential to be the first disease-modifying therapy for SORD Deficiency, (iii) the timing of the initiation and completion of our clinical trials, (iv) the likelihood that data from our clinical trials will support future development of our product candidates and (v) the likelihood of obtaining regulatory approval of our product candidates. 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 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, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain regulatory approval of our current and future product candidates, (viii) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (ix) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (x) the implementation of our business model and strategic plans for our business and product candidates, (xi) our intellectual property position and the duration of our patent rights, (xii) developments or disputes concerning our intellectual property or other proprietary rights, (xiii) our expectations regarding government and third-party payor coverage and reimbursement, (xiv) our ability to compete in the markets we serve, (xv) the impact of government laws and regulations and liabilities thereunder, (xvi) developments relating to our competitors and our industry, (xvii) the impact of the COVID-19 pandemic on the timing and progress of our ongoing clinical trials and our business in general and (xviii) 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 the "Risk Factors" 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:

    Maghan Meyers

    (212) 600-1902 or

    Media:

    Patients:



    Dottie Caplan, SVP Patient Advocacy and Engagement



    617.417.8114

    Applied Therapeutics, Inc.



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  4. Substantial and statistically significant reduction in plasma galactitol of 40% with new weight-based dosing parameters

    Baseline galactitol levels clearly correlated with baseline clinical functional outcomes

    NEW YORK, Oct. 18, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported additional biomarker data from the pediatric ACTION-Galactosemia Kids study.

    As previously announced, a pharmacokinetic analysis of AT-007 plasma drug levels at day 30 revealed that pediatric dosing could be further optimized by adjusting dose based on weight rather…

    Substantial and statistically significant reduction in plasma galactitol of 40% with new weight-based dosing parameters

    Baseline galactitol levels clearly correlated with baseline clinical functional outcomes

    NEW YORK, Oct. 18, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported additional biomarker data from the pediatric ACTION-Galactosemia Kids study.

    As previously announced, a pharmacokinetic analysis of AT-007 plasma drug levels at day 30 revealed that pediatric dosing could be further optimized by adjusting dose based on weight rather than age. As such, weight-based dosing brackets were implemented, and a subset of pediatric patients were dose-adjusted for an additional 30 days.

    A new data analysis was performed on galactitol reduction across the study, including data from children who received a dose adjustment. AT-007 substantially reduced plasma galactitol by approximately 40%, which was statistically significant (p<0.001) vs. placebo. Reduction in plasma galactitol was rapid and sustained, with no impact on levels of galactose or Gal-1p, and was similar across dose groups. AT-007 was safe and well tolerated in children of all ages (2-17). The Company plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in Q4 of this year.  

    As previously announced, an analysis of the 47 children in the ACTION-Galactosemia Kids study demonstrated a clear correlation between baseline galactitol level and baseline clinical functional outcomes. Children with higher plasma galactitol levels displayed greater disease severity vs. children with lower plasma galactitol levels at baseline.   This data will be presented as a late-breaking abstract at the 14th International Congress on Inborn Errors of Metabolism (ICIEM) November 21-24, 2021.

    "We have demonstrated that toxic galactitol drives disease progression and greatly impacts quality of life in Galactosemia patients," said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. "Here we have shown that AT-007 significantly reduces galactitol levels, which we believe could transform the lives of children and adults living with this disease. We are sincerely thankful to all of the families participating in the ACTION-Galactosemia clinical program, who have made this study possible."

    "The Commercial team has made great progress in preparing for a possible launch of AT-007 in Galactosemia. Our ‘Galactosemia Together' disease state education campaign has generated tremendous interest from the physician and patient community and our website galactosemia.com has won numerous industry awards. We look forward to potentially bringing AT-007 to these patients who are in desperate need of a treatment option," said Adam Hansard, Chief Commercial Officer of Applied Therapeutics.

    About Applied Therapeutics

    Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company's lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of Diabetic retinopathy, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.

    To learn more, please visit www.appliedtherapeutics.com and follow the company on Twitter @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 strategy, future operations, prospects, plans and objectives of management, including words such as "may," "will," "expect," "anticipate," "plan," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, statements regarding (i) the timing of our NDA submission for potential approval of AT-007, which will include data from the ACTION-Galactosemia Kids trial and the 90-day safety data in adults with Galactosemia, (ii) the timing of our rare disease franchise expansion programs in SORD Deficiency, (iii) the timing of the initiation and completion of our clinical trials, including ARISE-HF, (iv) the likelihood that data from our clinical trials will support future development of our product candidates and (v) the likelihood of obtaining regulatory approval of our product candidates. 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 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, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain regulatory approval of our current and future product candidates, (viii) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (ix) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (x) the implementation of our business model and strategic plans for our business and product candidates, (xi) our intellectual property position and the duration of our patent rights, (xii) developments or disputes concerning our intellectual property or other proprietary rights, (xiii) our expectations regarding government and third-party payor coverage and reimbursement, (xiv) our ability to compete in the markets we serve, (xv) the impact of government laws and regulations and liabilities thereunder, (xvi) developments relating to our competitors and our industry, (xvii) the impact of the COVID-19 pandemic on the timing and progress of our ongoing clinical trials and our business in general and (xviii) 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 the "Risk Factors" 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:

    Maghan Meyers

    (212) 600-1902 or

    Media:

    Applied Therapeutics, Inc.



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  5. Substantial and statistically significant reduction in plasma galactitol

    Baseline analysis demonstrates clear relationship between plasma galactitol level and severity of disease in pediatric Galactosemia patients

    NEW YORK, Sept. 09, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported initial biomarker data from the pediatric ACTION-Galactosemia Kids study, as well as baseline analysis of the 47 children enrolled in the study.

    The results demonstrate a substantial reduction in plasma galactitol of approximately 35% after 30 days of treatment…

    Substantial and statistically significant reduction in plasma galactitol

    Baseline analysis demonstrates clear relationship between plasma galactitol level and severity of disease in pediatric Galactosemia patients

    NEW YORK, Sept. 09, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported initial biomarker data from the pediatric ACTION-Galactosemia Kids study, as well as baseline analysis of the 47 children enrolled in the study.

    The results demonstrate a substantial reduction in plasma galactitol of approximately 35% after 30 days of treatment, which was statistically significant (p<0.001) vs. placebo.  AT-007 was safe and well tolerated in children of all ages (2-17).  A pharmacokinetic analysis of AT-007 plasma drug levels at day 30 revealed that dosing could be further optimized by adjusting dose based on weight rather than age.  As such, weight-based dosing brackets have been implemented for the remainder of the study, and a subset of patients have been dose-adjusted. The Company expects to present additional biomarker data at the optimized drug levels pending analysis at day 30.  Due to the time required for these study changes, the Company now plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in Q4 of this year, and has adjusted projected timelines accordingly.

    Additionally, analysis of the 47 children in the ACTION-Galactosemia Kids study demonstrated a clear correlation between baseline galactitol level and baseline clinical functional outcomes. Children with higher plasma galactitol levels displayed greater disease severity vs. children with lower plasma galactitol levels at baseline. This data is the first demonstration of correlation of a biochemical biomarker with severity of disease in Galactosemia patients. Full data will be presented at a future medical conference.

    "Reduction in toxic galactitol is critically important to patients with Galactosemia," said Shoshana Shendelman, PhD, CEO, Founder and Chair of the Board of Applied Therapeutics. "AT-007 has previously demonstrated significant reduction in galactitol in adults with Galactosemia. Now, we have demonstrated a significant reduction in children with this devastating rare disease. As the first interventional clinical study in pediatric Galactosemia patients, ACTION-Galactosemia Kids has already provided important insight into disease severity and progression. This new data demonstrating correlation of galactitol level with severity of disease underscores the importance of reducing this toxic metabolite in patients with Galactosemia."

    About Applied Therapeutics

    Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company's lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of Diabetic retinopathy, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.

    To learn more, please visit www.appliedtherapeutics.com and follow the company on Twitter @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 strategy, future operations, prospects, plans and objectives of management, including words such as "may," "will," "expect," "anticipate," "plan," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, statements regarding (i) the timing of our NDA submission for potential approval of AT-007, which will include data from the ACTION-Galactosemia Kids trial and the 90-day safety data in adults with Galactosemia, (ii) the timing of our rare disease franchise expansion programs in SORD Deficiency, (iii) the timing of the initiation and completion of our clinical trials, including ARISE-HF, (iv) the likelihood that data from our clinical trials will support future development of our product candidates and (v) the likelihood of obtaining regulatory approval of our product candidates. 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 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, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain regulatory approval of our current and future product candidates, (viii) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (ix) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (x) the implementation of our business model and strategic plans for our business and product candidates, (xi) our intellectual property position and the duration of our patent rights, (xii) developments or disputes concerning our intellectual property or other proprietary rights, (xiii) our expectations regarding government and third-party payor coverage and reimbursement, (xiv) our ability to compete in the markets we serve, (xv) the impact of government laws and regulations and liabilities thereunder, (xvi) developments relating to our competitors and our industry, (xvii) the impact of the COVID-19 pandemic on the timing and progress of our ongoing clinical trials and our business in general and (xviii) 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 the "Risk Factors" 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:

    Maghan Meyers

    (212) 600-1902 or

    Media:

    Applied Therapeutics, Inc.



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