Applied Therapeutics Reports Second Quarter 2025 Financial Results
Applied Therapeutics (NASDAQ:APLT) reported Q2 2025 financial results and significant progress across its rare disease pipeline. The company's lead program, govorestat for CMT-SORD treatment, advances with an upcoming FDA meeting in Q3 2025 to discuss potential NDA submission. The company presented promising 24-month clinical data showing slowed disease progression and launched a sponsored Sorbitol Assay program for patient identification.
Financial highlights include cash position of $30.4M as of June 30, 2025, down from $79.4M in December 2024. Q2 2025 resulted in a net loss of $21.3M ($0.15 per share). The company secured an out-licensing agreement with Biossil for AT-001 and strengthened its leadership team with key promotions.
Applied Therapeutics (NASDAQ:APLT) ha comunicato i risultati finanziari del secondo trimestre 2025 e importanti progressi nel suo portafoglio per le malattie rare. Il programma principale, govorestat per il trattamento della CMT‑SORD, procede con un incontro programmato con la FDA nel terzo trimestre 2025 per discutere una possibile presentazione della NDA. L'azienda ha presentato promettenti dati clinici a 24 mesi che evidenziano un rallentamento della progressione della malattia e ha lanciato un programma sponsorizzato di assay del sorbitolo per l'identificazione dei pazienti.
I punti finanziari includono una posizione di cassa di $30.4M al 30 giugno 2025, in calo rispetto a $79.4M a dicembre 2024. Il Q2 2025 si è chiuso con una perdita netta di $21.3M ($0.15 per azione). L'azienda ha inoltre stipulato un accordo di out‑licensing con Biossil per AT�001 e ha rafforzato il team dirigente con promozioni chiave.
Applied Therapeutics (NASDAQ:APLT) informó resultados financieros del segundo trimestre de 2025 y avances significativos en su cartera de enfermedades raras. Su programa principal, govorestat para el tratamiento de CMT‑SORD, avanza con una reunión prevista con la FDA en el tercer trimestre de 2025 para tratar una posible presentación de NDA. La compañía presentó prometedores datos clínicos a 24 meses que muestran una desaceleración de la progresión de la enfermedad y lanzó un programa patrocinado de ensayo de sorbitol para la identificación de pacientes.
Entre los aspectos financieros destaca una posición de efectivo de $30.4M al 30 de junio de 2025, frente a $79.4M en diciembre de 2024. El Q2 2025 registró una pérdida neta de $21.3M ($0.15 por acción). La compañía aseguró además un acuerdo de out‑licensing con Biossil para AT�001 y reforzó su equipo directivo con promociones clave.
Applied Therapeutics (NASDAQ:APLT)� 2025� 2분기 재무 실적� 희귀질환 파이프라인의 주요 진전� 발표했습니다. 주력 프로그램� CMT‑SORD 치료� govorestat� NDA 제출 가능성� 논의하기 위한 FDA 미팅� 2025� 3분기� 예정되어 있으� 순조롭게 진행되고 있습니다. 회사� 질병 진행 속도가 둔화� 것으� 보이� 유망� 24개월 임상 데이�� 제시했고, 환자 선별� 위한 후원 소르비톨 분석 프로그램� 시작했습니다.
재무 하이라이트로� 2025� 6� 30� 기준 현금 보유� $30.4M� 기록했으�, 이는 2024� 12월의 $79.4M에서 감소� 수치입니�. 2025� 2분기 순손실은 $21.3M (주당 $0.15)이었습니�. 또한 회사� AT�001� 대� Biossil� 아웃라이선스 계약� 체결하고 핵심 인사 승진� 통해 경영진을 강화했습니다.
Applied Therapeutics (NASDAQ:APLT) a publié ses résultats financiers du deuxième trimestre 2025 et annoncé des progrès significatifs dans son portefeuille de maladies rares. Le programme principal, govorestat pour le traitement de la CMT‑SORD, progresse avec une réunion prévue auprès de la FDA au troisième trimestre 2025 pour discuter d’une éventuelle soumission de NDA. La société a présenté des données cliniques à 24 mois prometteuses montrant un ralentissement de la progression de la maladie et a lancé un programme sponsorisé d’analyse du sorbitol pour l’identification des patients.
Les points financiers incluent une position de trésorerie de $30.4M au 30 juin 2025, en baisse par rapport à $79.4M en décembre 2024. Le T2 2025 s’est soldé par une perte nette de $21.3M (0,15 $ par action). La société a également conclu un accord d’out‑licensing avec Biossil pour AT�001 et renforcé son équipe de direction par des promotions clés.
Applied Therapeutics (NASDAQ:APLT) veröffentlichte die Finanzergebnisse für Q2 2025 und berichtete über deutliche Fortschritte in seiner Pipeline für seltene Krankheiten. Das führende Programm, govorestat zur Behandlung von CMT‑SORD, kommt voran; ein Treffen mit der FDA ist für Q3 2025 geplant, um eine mögliche NDA‑Einreichung zu besprechen. Das Unternehmen legte vielversprechende 24′ѴDzԲٱ′ұԾ岹ٱ vor, die eine verlangsamte Krankheitsprogression zeigen, und startete ein gesponsertes Sorbitol‑Assay‑Programm zur Identifikation von Patienten.
Zu den finanziellen Eckdaten zählt ein Barmittelbestand von $30.4M per 30. Juni 2025, gegenüber $79.4M im Dezember 2024. Q2 2025 schloss mit einem Nettoverlust von $21.3M (je Aktie $0.15). Das Unternehmen sicherte sich zudem eine Out‑Licensing‑Vereinbarung mit Biossil für AT�001 und stärkte das Führungsteam durch wichtige Beförderungen.
- Secured FDA meeting in Q3 2025 to discuss potential NDA submission for govorestat in CMT-SORD
- 24-month MRI data demonstrated govorestat slowed disease progression
- Launched no-cost sponsored Sorbitol Assay program to support patient identification
- Secured out-licensing agreement with Biossil for AT-001 with upfront payment and potential future royalties
- Cash position decreased significantly from $79.4M to $30.4M in 6 months
- Net loss increased to $21.3M in Q2 2025 compared to net income of $2.9M in Q2 2024
- General and administrative expenses increased by $2.6M year-over-year
Insights
Applied Therapeutics reports $21.3M Q2 loss with cash runway concerns amid advancing rare disease pipeline and FDA engagement.
Applied Therapeutics' Q2 results reveal critical financial challenges alongside pipeline progress. The company's
The upcoming FDA meeting for govorestat in CMT-SORD represents the company's most immediate value driver. The INSPIRE trial data showed statistically significant improvements across key secondary endpoints and 24-month MRI data demonstrated slowed disease progression. These clinical advances are supported by the strategic launch of a sponsored sorbitol assay to identify potential patients, which could accelerate diagnosis and build a treatment-ready population if approved.
The out-licensing agreement with Biossil for AT-001 provides immediate non-dilutive capital through the upfront payment while retaining future royalty potential. This transaction allows Applied to concentrate resources on its rare disease pipeline while maintaining upside in the diabetes space. However, the press release lacks specifics on the upfront payment amount, making it difficult to assess the deal's immediate financial impact.
The company faces a pivotal inflection point, balancing promising clinical data against deteriorating finances. While pipeline assets show potential with several regulatory catalysts ahead, the rapid cash depletion suggests additional financing will likely be necessary in early 2026, which could result in dilution for current shareholders unless regulatory approvals generate revenue or partnerships materialize.
Applied Therapeutics advances govorestat across three rare diseases with promising CMT-SORD data and diagnostic initiatives to accelerate patient identification.
Applied Therapeutics is making significant strides in addressing multiple rare genetic disorders with govorestat, its lead asset. The INSPIRE Phase 2/3 trial data for CMT-SORD (Charcot-Marie-Tooth disease with Sorbitol Dehydrogenase Deficiency) demonstrates clinically meaningful benefits across multiple endpoints. The 12-month data showed statistically significant improvements in the CMT-Health Index and CMT-Functional Outcomes Measure Lower Limb domain, while 24-month MRI data confirmed slowed disease progression—crucial for a progressive neurodegenerative condition where halting decline represents a substantial clinical victory.
The company's launch of a sponsored Urine Sorbitol Assay is particularly noteworthy as it addresses a fundamental challenge in rare disease drug development: patient identification. By removing financial barriers to diagnosis through no-cost testing for healthcare providers and patients, Applied is simultaneously building a diagnostic infrastructure and potential patient pool ahead of possible approval. This dual clinical-diagnostic strategy maximizes the potential market penetration for govorestat if approved.
The upcoming FDA meeting in Q3 2025 to discuss NDA submission strategy for CMT-SORD represents a critical regulatory milestone that could accelerate the path to market. Additionally, the company continues pursuing parallel development in Classic Galactosemia (despite a previous Complete Response Letter) and the ultra-rare PMM2-CDG, demonstrating a focused rare disease strategy leveraging a single molecular entity across multiple indications.
While each indication represents a small patient population individually, collectively they create a more substantial market opportunity. The company's strategic focus on genetic diseases with similar underlying mechanisms (accumulation of toxic metabolites) maximizes R&D efficiency while addressing significant unmet needs in disorders currently lacking approved therapies.
- CMT-SORD program advances; meeting scheduled with the FDA in Q3 2025 to discuss govorestat for the treatment of CMT-SORD
-Presented full 12-month clinical results and new topline data from INSPIRE Phase 2/3 trial of govorestat for the treatment of CMT-SORD at PNS 2025 Annual Meeting highlighting slowed progression of disease observed via MRI at 24 months
-Launched new sponsored Sorbitol Assay to support identification of patients suspected of having CMT-SORD
-Entered into out-licensing agreement with Biossil, Inc. for AT-001
-New data on govorestat for the treatment of PMM2-CDG to be presented at the 2025 ASHG Annual Meeting
-Ongoing review of govorestat development program for Classic Galactosemia progressing as planned
NEW YORK, Aug. 13, 2025 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT) (the “Company�), a clinical-stage biopharmaceutical company dedicated to creating transformative treatments for rare diseases, today reported financial results for the second quarter ended June 30, 2025.
“The first half of 2025 has been marked by a period of important execution as we’ve strengthened our internal clinical operations and made significant progress in addressing priorities while aligning on future planning across our pipeline. We are positioned for forward momentum as we engage with the FDA in the third quarter regarding a potential NDA submission for govorestat for the treatment of CMT-SORD. Our commitment to the CMT-SORD community is highlighted by our newly launched sponsored sorbitol assay, which is available to healthcare providers for patients at no-cost, removing potential barriers to diagnosis and treatment,� said Les Funtleyder, interim CEO and CFO of the Company. “As we look to the remainder of the year, we are focused on regulatory alignment across our pipeline. Our unwavering priority continues to be transforming the lives of patients with rare diseases, specifically CMT-SORD, Classic Galactosemia and PMM2-CDG.�
Recent Highlights
CMT-SORD
- Meeting Scheduled with the FDA to Discuss Govorestat for Treatment of CMT-SORD. The Company has been granted a meeting with the U.S. Food and Drug Administration (the “FDA�) to align on its planned New Drug Application (“NDA�) submission strategy for govorestat, which is expected to be held in the third quarter of 2025. The Company plans to discuss the latest comprehensive data generated on govorestat for the treatment of Sorbitol Dehydrogenase Deficiency (“SORD�), a subtype of Charcot-Marie-Tooth (“CMT�) disease.
- Launched Sponsored Assay to Identify Patients with CMT-SORD Through Urine Sorbitol Testing. In July 2025, the Company launched a sponsored Urine Sorbitol Assay for the identification of patients with CMT-SORD. The program is intended to support health care providers in identifying suspected cases of CMT-SORD through urine-based sorbitol measurement, using a clinically validated and compliant testing workflow. The Urine Sorbitol Assay is available to healthcare providers at no cost for patients and caregivers, removing potential barriers to diagnosis, care management and treatment that patients may face.
- Full 12-Month Clinical Results and New Topline Data from INSPIRE Phase 2/3 Trial Shared in Late-Breaking Oral Presentation at Peripheral Nerve Society (“PNS�) 2025 Annual Meeting. In May 2025, the Company presented full 12-month clinical results and topline 18- and 24-month patient level analyses from the INSPIRE Phase 2/3 trial of govorestat for the treatment of patients with CMT-SORD. Govorestat continued to be generally safe and well tolerated, with similar incidence of adverse events between active and placebo-treated groups. Govorestat treatment demonstrated statistically significant improvements across key secondary endpoints at 12 months including CMT-Health Index (“HI�), correlation between absolute reduction in sorbitol and change in the 10-meter walk/run test (�10MWRT�) and the CMT-Functional Outcomes Measure (“FOM�) Lower Limb domain and lowering of blood sorbitol levels. Additionally, examination of 24-month MRI data demonstrated that govorestat slowed the progression of disease.
Classic Galactosemia
- Ongoing Review of Govorestat Development Program for Classic Galactosemia Progressing as Planned. The Company continues to evaluate its response to the Complete Response Letter (“CRL�) issued in response to the Company’s NDA for govorestat for the treatment of Classic Galactosemia, including any meeting requests with the FDA regarding potential regulatory advancement of the Company’s Classic Galactosemia program.
PMM2-CDG
- Results of Treatment of PMM2-CDG with Govorestat to be Presented at the 2025 American Society of Human Genetics (“ASHG�) Annual Meeting. In October 2025, results from an ongoing single-patient investigator-initiated trial evaluating govorestat for the treatment of phosphomannomutase 2-congenital disorder glycosylation (“PMM2-CDG�) will be shared at the 2025 ASHG Annual Meeting, being held from October 14-18, 2025, in Boston, Massachusetts. PMM2-CDG is an ultra-rare mutation of the PMM2 gene and the most common congenital disorder of glycosylation, a group of ultra-rare hereditary metabolic disorders. It is a severe disease with a high mortality rate in pediatric patients. Govorestat has received Orphan Drug Designation and Rare Pediatric Disease designation from the FDA for the treatment of PMM2-CDG.
Corporate
- Entered Into Out-licensing Agreement with Biossil, Inc. for AT-001. In July 2025, the Company entered into an out-licensing agreement with Biossil for AT-001, the Company’s investigational, novel Aldose Reductase Inhibitor (“ARI�) developed as an oral therapy for the treatment of Diabetic Cardiomyopathy (“DbCM�). Under the terms of the agreement, Biossil gained exclusive worldwide rights to develop and commercialize AT-001, excluding a select number of rare disease indications. In exchange, Applied received an upfront payment and is eligible to receive future royalties and milestone payments.
- Appointed Key Leadership Members with Promotions of Evan Bailey, M.D., to Chief Medical Officer, and Dottie Caplan to Executive Vice President, Patient Advocacy and Government Affairs. In June 2025, the Company promoted Evan Bailey, M.D., from Senior Vice President, Clinical Development to Chief Medical Officer to lead the Company’s clinical development and medical affairs activities. Additionally, Dottie Caplan was promoted from Senior Vice President, Patient Advocacy and Engagement to Executive Vice President, Patient Advocacy and Government Affairs, to lead the Company’s patient engagement and advocacy efforts as well as the regulatory, legislative, and public policy functions.
Financial Results
- Cash and cash equivalents totaled
$30.4 million as of June 30, 2025, compared with$79.4 million at December 31, 2024. - Research and development expenses for the three months ended June 30, 2025, were
$9.9 million , compared to$10.0 million for the three months ended June 30, 2024. The decrease of approximately$0.1 million was primarily related to a decrease in clinical and preclinical expenses, offset by an overall increase in stock-based compensation, travel, consulting, personnel, drug manufacturing and formulation expenses. - General and administrative expenses were
$13.2 million for the three months ended June 30, 2025, compared to$10.6 million for the three months ended June 30, 2024. The increase of approximately$2.6 million was primarily related to an increase in legal, professional, personnel, data storage and stock-based compensation expenses, offset by an overall decrease in commercial and insurance expenses. - Net loss for the second quarter of 2025 was
$21.3 million , or$0.15 per basic and diluted common share, compared to a net income of$2.9 million , or$0.02 per basic common share and a net loss of$0.13 per diluted common share, for the second quarter of 2024.
About Applied Therapeutics
Applied Therapeutics is a clinical-stage biopharmaceutical company committed to the development of novel drug candidates against validated molecular targets in rare diseases. The Company’s lead drug candidate, govorestat, is a novel central nervous system (“CNS�) penetrant Aldose Reductase Inhibitor (“ARI�) for the treatment of CNS rare metabolic diseases, including Classic Galactosemia, Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (“CMT-SORD�) and phosphomannomutase 2 congenital disorder of glycosylation (“PMM2-CDG�).
To learn more, please visit and follow the company on X at @Applied_Tx.
Forward-Looking Statements
This press release contains “forward-looking statements� that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, included in this press release regarding the strategy, future operations, prospects, plans and objectives of management, including words such as “may,� “will,� “expect,� “anticipate,� “plan,� “intend,� “predicts� and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, statements regarding (i) the Company’s business, plans and outlook, (ii) expectations about the clinical development and commercialization of govorestat, including the potential for an NDA submission in CMT-SORD, the upcoming meeting with the FDA, the data presentation at the 2025 ASHG Annual Meeting and development plans in Classic Galactosemia, (iii) the benefits of the sponsored Urine Sorbitol Assay and (iv) the benefits of the out-licensing agreement with Biossil. Forward-looking statements in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved.
Such risks and uncertainties include, without limitation, (i) our plans to develop, market and commercialize our product candidates, (ii) the initiation, timing, progress and results of our current and future preclinical studies and clinical trials and our research and development programs, (iii) our ability to take advantage of expedited regulatory pathways for any of our product candidates, (iv) our estimates regarding expenses, future revenue, capital requirements and needs for additional financing, (v) our ability to successfully acquire or license additional product candidates on reasonable terms and advance product candidates into, and successfully complete, clinical studies, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain and timing of regulatory approval of our current and future product candidates, (viii) the anticipated indications for our product candidates, if approved, (ix) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (x) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (xi) the implementation of our business model and strategic plans for our business and product candidates, (xii) our intellectual property position and the duration of our patent rights, (xiii) developments or disputes concerning our intellectual property or other proprietary rights, (xiv) our expectations regarding government and third-party payor coverage and reimbursement, (xv) our ability to compete in the markets we serve, (xvi) the impact of government laws and regulations and liabilities thereunder, (xvii) developments relating to our competitors and our industry, (xviii) our ability to achieve the anticipated benefits from the agreements entered into in connection with our partnership with Advanz Pharma and (xix) other factors that may impact our financial results. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur at all. Factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in our filings with the U.S. Securities and Exchange Commission, including those described in the “Risk Factors� section contained therein. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.
Contacts
Investors:
Julie Seidel / Andrew Vulis
(212) 600-1902
Media:
Applied Therapeutics, Inc. Condensed Balance Sheets (in thousands, except share and per share data) (Unaudited) | ||||||||
As of | As of | |||||||
June30, | December31, | |||||||
2025 | 2024 | |||||||
ASSETS | ||||||||
CURRENT ASSETS: | ||||||||
Cash and cash equivalents | $ | 30,421 | $ | 79,398 | ||||
Prepaid expenses and other current assets | 4,090 | 4,248 | ||||||
Total current assets | 34,511 | 83,646 | ||||||
Security deposits | 252 | 253 | ||||||
Operating lease right-of-use asset | 2,579 | 2,792 | ||||||
TOTAL ASSETS | $ | 37,342 | $ | 86,691 | ||||
LIABILITIES AND STOCKHOLDERS� EQUITY | ||||||||
CURRENT LIABILITIES: | ||||||||
Current portion of operating lease liabilities | $ | 447 | $ | 406 | ||||
Accounts payable | 4,861 | 4,433 | ||||||
Accrued expenses and other current liabilities | 10,668 | 16,143 | ||||||
Warrant liabilities | 1,773 | 6,314 | ||||||
Total current liabilities | 17,749 | 27,296 | ||||||
NONCURRENT LIABILITIES: | ||||||||
Noncurrent portion of operating lease liabilities | 2,155 | 2,389 | ||||||
Total noncurrent liabilities | 2,155 | 2,389 | ||||||
Total liabilities | 19,904 | 29,685 | ||||||
STOCKHOLDERS� EQUITY: | ||||||||
Common stock, | 35 | 35 | ||||||
Preferred stock, par value | � | � | ||||||
Additional paid-in capital | 634,768 | 631,181 | ||||||
Accumulated deficit | (617,365 | ) | (574,210 | ) | ||||
Total stockholders' equity | 17,438 | 57,006 | ||||||
TOTAL LIABILITIES AND STOCKHOLDERS� EQUITY | $ | 37,342 | $ | 86,691 |
Applied Therapeutics, Inc. Condensed Statements of Operations (in thousands, except share and per share data) (Unaudited) | ||||||||||||||||
Three Months Ended | Six Months Ended | |||||||||||||||
June30, | June30, | |||||||||||||||
2025 | 2024 | 2025 | 2024 | |||||||||||||
REVENUE: | ||||||||||||||||
Research and development services revenue | $ | � | $ | 144 | $ | � | $ | 334 | ||||||||
Total revenue | � | 144 | � | 334 | ||||||||||||
COSTS AND EXPENSES: | ||||||||||||||||
Research and development | 9,923 | 10,004 | 17,760 | 22,221 | ||||||||||||
General and administrative | 13,175 | 10,580 | 30,863 | 19,646 | ||||||||||||
Total costs and expenses | 23,098 | 20,584 | 48,623 | 41,867 | ||||||||||||
LOSS FROM OPERATIONS | (23,098 | ) | (20,440 | ) | (48,623 | ) | (41,533 | ) | ||||||||
OTHER INCOME (EXPENSE), NET: | ||||||||||||||||
Interest income | 364 | 628 | 985 | 1,215 | ||||||||||||
Change in fair value of warrant liabilities | 1,437 | 22,744 | 4,541 | (40,660 | ) | |||||||||||
Other expense | (33 | ) | (34 | ) | (58 | ) | (62 | ) | ||||||||
Total other income (expense), net | 1,768 | 23,338 | 5,468 | (39,507 | ) | |||||||||||
Net income (loss) | $ | (21,330 | ) | $ | 2,898 | $ | (43,155 | ) | $ | (81,040 | ) | |||||
Net income (loss) per share attributable to common stockholders - basic | $ | (0.15 | ) | $ | 0.02 | $ | (0.30 | ) | $ | (0.60 | ) | |||||
Net income (loss) per share attributable to common stockholders - diluted | $ | (0.15 | ) | $ | (0.13 | ) | $ | (0.30 | ) | $ | (0.60 | ) | ||||
Weighted-average common stock outstanding - basic | 145,091,474 | 143,934,239 | 144,940,561 | 134,627,942 | ||||||||||||
Weighted-average common stock outstanding - diluted | 145,091,474 | 152,392,748 | 144,940,561 | 134,627,942 |
