APLT Applied Therapeutics Inc.

14.66
+0.61  (+4%)
Previous Close 14.05
Open 14.14
52 Week Low 13.58
52 Week High 51.99
Market Cap $382,353,969
Shares 26,081,444
Float 17,620,137
Enterprise Value $272,427,288
Volume 55,792
Av. Daily Volume 93,439
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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 2a
Phase 2a
Phase 2 trial has been initiated.

Latest News

  1. Phase 2 pilot study of AT-007 initiated in patients with SORD Deficiency

    NDA submission for AT-007 in Galactosemia expected no later than Q3 2021; commercial preparations ongoing

    Completion of enrollment in ARISE-HF Phase 3 global registrational study of AT-001 in Diabetic Cardiomyopathy on track for mid-2021

    Ended 1Q21 with a strong balance sheet of $148.1 in cash and cash equivalents and short-term investments

    NEW YORK, May 11, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT) ("Applied Therapeutics" or the "Company"), 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…

    Phase 2 pilot study of AT-007 initiated in patients with SORD Deficiency

    NDA submission for AT-007 in Galactosemia expected no later than Q3 2021; commercial preparations ongoing

    Completion of enrollment in ARISE-HF Phase 3 global registrational study of AT-001 in Diabetic Cardiomyopathy on track for mid-2021

    Ended 1Q21 with a strong balance sheet of $148.1 in cash and cash equivalents and short-term investments

    NEW YORK, May 11, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT) ("Applied Therapeutics" or the "Company"), 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 first quarter ended March 31, 2021.

    "In the first quarter we continued to progress our late stage programs towards commercialization in Galactosemia and Diabetic Cardiomyopathy, while advancing our additional rare disease programs in SORD and PMM2-CDG," said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics. "We are looking forward to our planned NDA submission in Galactosemia in the third quarter of this year, and continue to focus on our primary objective – bringing drugs to patients in desperate need of treatment."

    Recent Highlights

    • Initiated Phase 2 Study of AT-007 in Patients with SORD Deficiency. The Phase 2 pilot study of AT-007 in patients with Sorbitol Dehydrogenase Deficiency (SORD Deficiency), a progressive, debilitating hereditary neuropathy that affects peripheral nerves and motor neurons, has been initiated. Preclinical findings demonstrate that AT-007 has the potential to be the first disease-modifying therapy for SORD, targeting the underlying cause of disease.

    • Presented Data on Galactosemia Disease Progression at the 2021 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics. In April 2021, the Company presented data featuring a cross-sectional analysis of nineteen pediatric patients with Classic Galactosemia, providing meaningful insight on the progressive worsening of the central nervous system phenotype with age.

    • Hosted Virtual Rare Disease Forum. In March 2021, the Company hosted a virtual forum highlighting its AT-007 development programs in Galactosemia, SORD Deficiency, and PMM2-CDG. A replay of the presentation, which featured several rare disease key opinion leaders, is available at https://www.appliedtherapeutics.com/presentations-and-publications/.

    • Announced Restart of Pediatric Galactosemia Study. In February 2021, the Company announced that the FDA lifted the clinical hold on the AT-007 ACTION-Galactosemia Kids pediatric clinical study, and the study resumed immediately. Applied Therapeutics worked closely with FDA to modify the trial, with the shared goal of ensuring that all patients have the opportunity to receive clinical benefit, and remains on target to submit an NDA no later than Q3 2021.



    • Closed Approximately $75 million Public Offering. In February 2021, the Company completed an underwritten public offering of 3,450,000 shares of its common stock, including the exercise in full of the underwriters' option to purchase 450,000 additional shares of common stock at a price of $23.00 per share, resulting in aggregate net proceeds of approximately $74.4 million.

    Financial Results

    • Cash and cash equivalents and short-term investments totaled $148.1 million as of March 31, 2021, compared with $96.8 million at December 31, 2020.



    • Research and development expenses for the three months ended March 31, 2021 were $14.4 million, compared to $7.3 million for the three months ended March 31, 2020. The increase of $7.2 million was primarily related to an increase in clinical and pre-clinical expense of $1.3 million, related to the progression of the AT-007 ACTION-Galactosemia adult extension study, the AT-007 ACTION-Galactosemia Kids pediatric registrational study and the AT-001 Phase 3 ARISE-HF clinical study; an increase in drug manufacturing and formulation costs of $5.2 million related to the progression of the manufacturing campaigns and the completion and release of AT-001 and AT-007 drug product batches; an increase in personnel expenses of $0.3 million due to the increase in headcount in support of our clinical program pipeline; an increase in stock-based compensation of $0.2 million due to new stock option and restricted stock unit grants; and an increase in regulatory and other expenses of $0.2 million relating to an increase in clinical consulting fees during the three months ended March 31, 2021.



    • General and administrative expenses were $9.8 million for the three months ended March 31, 2021, compared to $5.2 million for the three months ended March 31, 2020. The increase of approximately $4.6 million was primarily related to an increase of $1.9 million related to the establishment of a commercial department; an increase in stock-based compensation of $1.5 million and increase in personnel expenses of $0.9 million related to an increase in headcount; an increase of $0.2 million related to increased insurance costs; an increase of $0.4 million in other expenses relating to increased costs of rent and other office expenses; and a $0.4 million decrease in legal and professional fees due to lower external legal fees.
    • Net loss for the first quarter of 2021 was $24.2 million, or $1.00 per basic and diluted common share, compared to a net loss of $12.4 million, or $0.59 per basic and diluted common share, for the first 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 Galactosemia, a rare pediatric metabolic disease. The Company initiated a pivotal Phase 1/2 clinical trial in June 2019, read out positive top-line biomarker data in adult Galactosemia patients in January 2020 and announced full data from the trial in April 2020. A pediatric Galactosemia study commenced in June 2020. The Company is also developing AT-001, a novel potent ARI that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The Company initiated a Phase 3 registrational study in DbCM in September 2019. 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.

    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 and PMM2-CDG, (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 or Brendan Burns

    (212) 600-1902 or

    Media:



    Applied Therapeutics, Inc.

    Statement of Operations

    (in thousands, except share and per share data)

    (Unaudited)

            
      Three Months Ended  
      March 31,  
      2021  2020  
    OPERATING EXPENSES:       
    Research and development $14,448  $7,271  
    General and administrative  9,751   5,196  
    Total operating expenses  24,199   12,467  
    LOSS FROM OPERATIONS  (24,199)  (12,467) 
    OTHER INCOME (EXPENSE), NET:       
    Interest income (expense), net  76   122  
    Other income (expense)  (56)  (24) 
    Total other income (expense), net  20   98  
    Net loss $(24,179) $(12,369) 
    Net loss attributable to common stockholders—basic and diluted $(24,179) $(12,369) 
    Net loss per share attributable to common stockholders—basic and diluted $(1.00) $(0.59) 
    Weighted-average common stock outstanding—basic and diluted  24,135,735   20,840,658  
              

    Applied Therapeutics, Inc.

    Balance Sheet

    (in thousands, except share and per share data)

            
      As of As of 
      March 31,  December 31, 
      2021  2020  
      (Unaudited)   
    ASSETS       
    CURRENT ASSETS:         
    Cash and cash equivalents $84,067  $57,466  
    Investments  63,992   39,363  
    Prepaid expenses and other current assets  6,347   5,764  
    Total current assets  154,406   102,593  
    Operating lease right-of-use asset  1,612   1,712  
    Security deposits and leasehold improvements  201   201  
    TOTAL ASSETS $156,219  $104,506  
    LIABILITIES AND STOCKHOLDERS' EQUITY        
    CURRENT LIABILITIES:        
    Current portion of operating lease liabilities $415  $406  
    Accounts payable  3,475   640  
    Accrued expenses and other current liabilities  15,884   20,189  
    Total current liabilities  19,774   21,235  
    NONCURRENT LIABILITIES:       
    Noncurrent portion of operating lease liabilities  1,224   1,332  
    Total noncurrent liabilities  1,224   1,332  
    Total liabilities  20,998   22,567  
    STOCKHOLDERS' EQUITY:         
    Common stock, $0.0001 par value; 100,000,000 shares authorized as of March 31, 2021 and December 31, 2020; 26,010,465 shares and 22,493,661 shares issued and outstanding as of March 31, 2021 and December 31, 2020, respectively  3   2  
    Additional paid-in capital  320,282   242,780  
    Accumulated other comprehensive loss  (154)  (112) 
    Accumulated deficit  (184,910)  (160,731) 
    Total stockholders' equity  135,221   81,939  
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY $156,219  $104,506  
              



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  2. NEW YORK, April 07, 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 announced a poster presentation at the upcoming 2021 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics (ACMG) taking place April 13-16, 2021. The abstract and presentation include a cross-sectional analysis of nineteen pediatric patients with Classic Galactosemia, characterizing progressive worsening of the central nervous system (CNS) phenotype with age.

    "We are pleased to share this important data with the medical community," said Riccardo…

    NEW YORK, April 07, 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 announced a poster presentation at the upcoming 2021 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics (ACMG) taking place April 13-16, 2021. The abstract and presentation include a cross-sectional analysis of nineteen pediatric patients with Classic Galactosemia, characterizing progressive worsening of the central nervous system (CNS) phenotype with age.

    "We are pleased to share this important data with the medical community," said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. "The severity and significant decline in CNS outcomes with age underscores the urgency for a disease-modifying therapy to halt or prevent damage in Galactosemia. "

    Presentation Details

    Abstract eP011: Progressive Worsening of Central Nervous System Phenotype in Children with Classic Galactosemia: a Cross-Sectional Analysis

    The poster presentation will be available on the ACMG website.

    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 Galactosemia, a rare pediatric metabolic disease. The Company initiated a pivotal Phase 1/2 clinical trial in June 2019, read out positive top-line biomarker data in adult Galactosemia patients in January 2020 and announced full data from the trial in April 2020. A pediatric Galactosemia study commenced in June 2020. The Company is also developing AT-001, a novel potent ARI that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The Company initiated a Phase 3 registrational study in DbCM in September 2019. 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.

    Investors:

    Maeve Conneighton

    (212) 600-1902 or

    Media:



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  3. AT-007 ACTION-Galactosemia Kids pediatric clinical study recently resumed following discussions with FDA

    On track to submit NDA for AT-007 in Galactosemia no later than Q3 2021; Commercial preparations underway

    ARISE-HF Phase 3 global registrational study of AT-001 in Diabetic Cardiomyopathy on track to complete enrollment in mid-2021

    On track to initiate rare disease franchise expansion programs in SORD Deficiency and PMM2-CDG studies in the first half of 2021; Company to host Rare Disease Forum on Tuesday, March 23rd

    NEW YORK, March 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…

    AT-007 ACTION-Galactosemia Kids pediatric clinical study recently resumed following discussions with FDA

    On track to submit NDA for AT-007 in Galactosemia no later than Q3 2021; Commercial preparations underway

    ARISE-HF Phase 3 global registrational study of AT-001 in Diabetic Cardiomyopathy on track to complete enrollment in mid-2021

    On track to initiate rare disease franchise expansion programs in SORD Deficiency and PMM2-CDG studies in the first half of 2021; Company to host Rare Disease Forum on Tuesday, March 23rd

    NEW YORK, March 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 financial results for the fourth quarter and full year ended December 31, 2020.

    "The fourth quarter was a productive period of internal planning and execution," said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics. "We are excited for what lies ahead in 2021, where our focus will be on advancing our late stage assets toward commercialization while initiating clinical development of our two new rare disease programs."

    Recent Highlights

    • Announced Restart of Pediatric Galactosemia Study.   In February 2021, the Company announced that the FDA lifted the clinical hold on the AT-007 ACTION-Galactosemia Kids pediatric clinical study, and the study resumed immediately.   Applied Therapeutics worked closely with FDA to modify the trial, with the shared goal of ensuring that all patients have the opportunity to receive clinical benefit, and remains on target to submit an NDA no later than Q3 2021.



    • Announced the Launch of a Galactosemia Awareness and Education Initiative.   Galactosemia Together is the first and only industry-led Galactosemia awareness and education campaign.   Developed in partnership with the Galactosemia community, this initiative aims to address gaps in education by providing updated, reliable and credible resources to help connect, educate and support those families impacted by this disease.



    • Announced Magnetic Resonance Spectroscopy (MRS) Data from ACTION-Galactosemia Study.   In December 2020, the Company shared MRS data on reduction of galactitol levels in the brain of Galactosemia patients treated with AT-007 in the ACTION-Galactosemia adult study.   Overall, plasma reduction in galactitol correlated with brain reduction in galactitol.   At the two doses which demonstrated statistically significant reduction in plasma galactitol, 20 and 40mg/kg, 3 out of 4 patients displayed substantial galactitol reduction ranging from 61.94% to 69.80% reduction from baseline.



    • Closed Public Offering of 3,000,000 Shares of Common Stock at a Price to the Public of $23.00 Per Share. In February 2021, the Company completed an underwritten public offering of 3,450,000 shares of common stock, including the exercise in full of the underwriters' option to purchase 450,000 additional shares of common stock at a price of $23.00 per share, resulting in aggregate net proceeds of approximately $74.3 million.

    Financial Results

    • Cash and cash equivalents and short-term investments totaled $96.8 million as of December 31, 2020, compared with $38.9 million at December 31, 2019.   This does not include approximately $74.3 million in aggregate net proceeds the Company received from the underwritten public offering of common stock in February 2021.



    • Research and development expenses for the year ended December 31, 2020 were $61.8 million, compared to $32.4 million for the year ended December 31, 2019. The increase of $29.4 million related to an increase in clinical and pre-clinical expense of $11.3 million, primarily related to the progression of the AT-007 ACTION-Galactosemia adult extension and the AT-001 Phase 3 ARISE-HF clinical studies, as well as the commencement and progression of the AT-007 ACTION-Galactosemia Kids pediatric registrational study; an increase in drug manufacturing and formulation expenses of $15.4 million primarily related to the commencement of the 2020 manufacturing campaigns and the associated raw material deliveries and AT-007 and AT-001 drug product batch releases; an increase in personnel expenses of $0.6 million due to the increase in headcount in support of our clinical program pipeline; a decrease in stock-based compensation of $0.2 million due to the stock option modification expense recognized during the year ended December 31, 2019 for the acceleration of certain options vesting following the IPO; offset by an increase in expense recognized during the year ended December 31, 2020 due to new stock option and restricted stock grants; and an increase of regulatory and other expenses of $2.3 million primarily related to University of Miami license fees and increased clinical consulting fees recognized during the year ended December 31, 2020.



    • General and administrative expenses were $32.7 million for the year ended December 31, 2020, compared to $13.2 million for the year ended December 31, 2019. The increase of $19.4 million was an increase in professional and legal fees of $4.1 million due to increased operations and costs associated with being a public company for a full year; an increase of $5.7 million related to the establishment of a commercial department; an increase in personnel expenses of $4.2 million and an increase in stock-based compensation of $2.0 million due to an increase in headcount; an increase of insurance expenses of $1.8 million related to increased D&O insurance costs; and an increase in other expenses of $1.6 million, primarily relating to increased costs of rent and other office expenses.



    • Net loss for the year ended December 31, 2020 was $94.0 million, or $4.28 per basic and diluted common share, compared to a net loss of $45.5 million, or $3.55 per basic and diluted common share, for the year ended December 31, 2019.

    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 Galactosemia, a rare pediatric metabolic disease. The Company initiated a pivotal Phase 1/2 clinical trial in June 2019, read out positive top-line biomarker data in adult Galactosemia patients in January 2020 and announced full data from the trial in April 2020. A pediatric Galactosemia study commenced in June 2020. The Company is also developing AT-001, a novel potent ARI that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The Company initiated a Phase 3 registrational study in DbCM in September 2019. 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.

    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 and PMM2-CDG, (iii) the timing and effectiveness of our Galactosemia awareness and education campaign, (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:

    Maeve Conneighton

    (212) 600-1902 or

    Media:



    Applied Therapeutics, Inc.

    Statements of Operations

    (in thousands, except share and per share data)

           
      Year Ended
      December 31, 
      2020  2019 
    OPERATING EXPENSES:      
    Research and development $61,788  $32,350 
    General and administrative  32,678   13,232 
    Total operating expenses  94,466   45,582 
    LOSS FROM OPERATIONS  (94,466)  (45,582)
    OTHER INCOME (EXPENSE), NET:      
    Interest income (expense), net  559   93 
    Other income (expense)  (54)  (24)
    Total other income (expense), net  505   69 
    Net loss $(93,961) $(45,513)
    Net loss attributable to common stockholders—basic and diluted $(93,961) $(45,513)
    Net loss per share attributable to common stockholders—basic and diluted $(4.28) $(3.55)
    Weighted-average common stock outstanding—basic and diluted  21,966,326   12,831,221 
     
     

    Applied Therapeutics, Inc. 

    Balance Sheets 

    (in thousands, except share and per share data)

         
      As of As of
      December 31,  December 31,
      2020

     2019

         
    ASSETS      
    CURRENT ASSETS:      
    Cash and cash equivalents $57,466  $18,850 
    Investments  39,363   20,004 
    Prepaid expenses and other current assets  5,764   7,301 
    Total current assets  102,593   46,155 
    Operating lease right-of-use asset  1,712   2,035 
    Security deposits and leasehold improvements  201   199 
    TOTAL ASSETS $104,506  $48,389 
    LIABILITIES AND STOCKHOLDERS' EQUITY      
    CURRENT LIABILITIES:      
    Current portion of operating lease liabilities $406  $356 
    Accounts payable  640   8,793 
    Accrued expenses and other current liabilities  20,189   4,950 
    Total current liabilities  21,235   14,099 
    NONCURRENT LIABILITIES:      
    Noncurrent portion of operating lease liabilities  1,332   1,683 
    Total noncurrent liabilities  1,332   1,683 
    Total liabilities  22,567   15,782 
    STOCKHOLDERS' EQUITY:      
    Common stock, $0.0001 par value; 100,000,000 shares authorized as of December 31, 2020 and December 31, 2019; 22,493,661 shares and 18,531,560 shares issued and outstanding as of December 31, 2020 and December 31, 2019, respectively  2   1 
    Additional paid-in capital  242,780   99,378 
    Accumulated other comprehensive loss  (112)  (2)
    Accumulated deficit  (160,731)  (66,770)
    Total stockholders' equity  81,939   32,607 
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY $104,506  $48,389 



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  4. NEW YORK, March 16, 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 announced it will host a Virtual Rare Disease Forum on Tuesday, March 23rd, 2021 from 1:00pm to 3:00pm ET. The event will highlight Applied Therapeutics' development programs in Galactosemia, SORD Deficiency, and PMM2-CDG. The agenda for the event will include:

    • Update on ACTION-Galactosemia Clinical Development Program
    • Overview of SORD Deficiency, presented by Stephan L. Züchner, MD, PhD, Professor and Department Chair of Human Genetics at the University of Miami Miller School of Medicine…

    NEW YORK, March 16, 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 announced it will host a Virtual Rare Disease Forum on Tuesday, March 23rd, 2021 from 1:00pm to 3:00pm ET. The event will highlight Applied Therapeutics' development programs in Galactosemia, SORD Deficiency, and PMM2-CDG. The agenda for the event will include:

    • Update on ACTION-Galactosemia Clinical Development Program
    • Overview of SORD Deficiency, presented by Stephan L. Züchner, MD, PhD, Professor and Department Chair of Human Genetics at the University of Miami Miller School of Medicine
    • Update on SORD Deficiency Clinical Development Plans
    • Overview of PMM2-CDG, presented by Joseph Muenzer, MD, PhD, Professor of Pediatric Genetics and Metabolism at University of North Carolina Children's Research Institute
    • Update on PMM2-CDG Clinical Development Program Plans

    "We're excited to host this event to spotlight our clinical development programs in Galactosemia, SORD and PMM2-CDG," said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics. "We believe that AT-007 represents an important advancement for patients with these rare diseases, and our clinical development programs offer a unique opportunity to meaningfully impact patients' lives."

    An audio webcast of the presentation will be available live. More details will be available at http://ir.appliedtherapeutics.com leading up to the event. An archived version of the presentation will be available following 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 Galactosemia, a rare pediatric metabolic disease. The Company initiated a pivotal Phase 1/2 clinical trial in June 2019, read out positive top-line biomarker data in adult Galactosemia patients in January 2020 and announced full data from the trial in April 2020. A pediatric Galactosemia study commenced in June 2020. The Company is also developing AT-001, a novel potent ARI that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The Company initiated a Phase 3 registrational study in DbCM in September 2019. 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.

    Investors:

    Maeve Conneighton

    (212) 600-1902 or

    Media:

     



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  5. NEW YORK, Feb. 23, 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, announced today that it will present at two upcoming investor conferences in March:

    Cowen 41st Annual Health Care Conference
    Date: Tuesday, March 2, 2021
    Time: 9:10 – 9:40 a.m. ET

    Barclays Global Healthcare Conference
    Date: Tuesday, March 9, 2021
    Time: 4:45 – 5:10 p.m. ET

    Webcast information for these events will be accessible on the Events page under the Investor Relations section of the Applied Therapeutics website at www.appliedtherapeutics.com. A replay will also be available following…

    NEW YORK, Feb. 23, 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, announced today that it will present at two upcoming investor conferences in March:

    Cowen 41st Annual Health Care Conference

    Date: Tuesday, March 2, 2021

    Time: 9:10 – 9:40 a.m. ET

    Barclays Global Healthcare Conference

    Date: Tuesday, March 9, 2021

    Time: 4:45 – 5:10 p.m. ET

    Webcast information for these events will be accessible on the Events page under the Investor Relations section of the Applied Therapeutics website at www.appliedtherapeutics.com. A replay will also be available following the webcast.

    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 Galactosemia, a rare pediatric metabolic disease. The Company initiated a pivotal Phase 1/2 clinical trial in June 2019, read out positive top-line biomarker data in adult Galactosemia patients in January 2020 and announced full data from the trial in April 2020. The Company is also developing AT-001, a novel potent ARI that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The Company initiated a Phase 3 registrational study in DbCM in September 2019. 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.

    Investors:

    Maeve Conneighton

    (212) 600-1902 or

    Media:

    Gleb Sagitov

     



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