Galactosemia

Applied Therapeutics Announces Topline Results from the ARISE-HF Phase 3 Study of AT-001 in Diabetic Cardiomyopathy

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Giovedì, Gennaio 4, 2024
Cardiology, Hospital, Food, Survival, BID, Physician, FDA, EMA, MAA, Diabetes, Disease, Massachusetts General Hospital, NDA, Incidence, MD, Doctor of Philosophy, Therapy, Odds ratio, Diabetic cardiomyopathy, New Drug Application, European Medicines Agency, SGLT2, Patient, Safety, Galactosemia, Heart failure, Pharmaceutical industry

NEW YORK, Jan. 04, 2024 (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 the topline results of the ARISE-HF Phase 3 trial of AT-001 (caficrestat) in patients with Diabetic Cardiomyopathy (DbCM) at high risk of progression to overt heart failure.  

Key Points: 
  • While a trend favored active treatment, the difference between active and placebo treated groups (0.30 ml/kg/min) was not statistically significant (p=0.210).
  • The ARISE-HF study evaluated the treatment effect of AT-001 as an add-on to diabetes standard of care therapies.
  • Approximately 38% of study subjects were on SGLT2 or GLP-1 therapies for treatment of diabetes, while 62% were not.
  • Full study results will be presented at an upcoming medical conference, along with results of the Diabetic Peripheral Neuropathy sub-study, which are still being analyzed.

Applied Therapeutics Announces MAA Validation and NDA Submission of Govorestat (AT-007) for Treatment of Classic Galactosemia

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Mercoledì, Gennaio 3, 2024
Galactosemia, Food, Ansell, FDA, EMA, MAA, Hope, NDA, Doctor of Philosophy, Therapy, New Drug Application, European Medicines Agency, Patient, Galactitol, CHMP, Nursing

NEW YORK, Jan. 03, 2024 (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 has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for govorestat (AT-007) for the treatment of Classic Galactosemia. The NDA was submitted in December 2023. In addition, the Company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the fourth quarter of 2023, which was subsequently validated and accepted for review in December 2023.

Key Points: 
  • “Govorestat offers hope to many patients and families affected by Galactosemia.
  • I saw the positive impact of govorestat first-hand in my daughter, Ansell, who participated in the ACTION-Galactosemia Kids pediatric study.
  • The FDA has a 60-day filing review period to determine whether the NDA is complete and accepted for review.
  • The MAA has been validated and will move to review by the EMA’s Committee for Medicinal Products for Human Use (CHMP).

Applied Therapeutics Reports Third Quarter 2023 Financial Results

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Giovedì, Novembre 9, 2023
Massachusetts General Hospital, FDA, Research, Insurance, Harvard Medical School, Heart failure, MAA, Ongo, Treatment, MD, Cardiology, European Medicines Agency, Diabetes, Chairperson, Doctor of Philosophy, ChromeOS, EASD, Quality of life, Symposium, KOL, U.S. FDA, EMA, European Association for the Study of Diabetes, Annual general meeting, NDA, Safety, Therapy, Diabetic cardiomyopathy, Investment, Patient, Advanz Pharma, Pharmaceutical industry, Birth control, SORD, Galactosemia

NEW YORK, Nov. 09, 2023 (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, 2023.

Key Points: 
  • The Company expects topline data from the study in the fourth quarter of 2023.
  • Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics, presented the data in an oral Symposium entitled Diabetic Cardiomyopathy (DbCM): a severe complication of diabetes.
  • Cash and cash equivalents and short-term investments totaled $37.5 million as of September 30, 2023, compared with $30.6 million at December 31, 2022.
  • Net loss for the third quarter of 2023 was $42.4 million, or $0.47 per basic and diluted common share, compared to a net loss of $19.1 million, or $0.40 per basic and diluted common share, for the third quarter 2022.

Applied Therapeutics Announces Successful Pre-NDA Meeting with the FDA and Confirms Plans to Submit NDA for Govorestat (AT-007) for Treatment of Classic Galactosemia to FDA in Q4 2023

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Mercoledì, Settembre 6, 2023
Partnership, New Drug Application, NDA, Doctor of Philosophy, Food and Drug Administration, Galactitol, MD, FDA, Therapy, Food, Pharmaceutical industry, Galactosemia

Based on discussions with the FDA, the Company believes they are aligned with the FDA and plans to submit an NDA for govorestat (AT-007) for the treatment of Galactosemia in the fourth quarter of this year.

Key Points: 
  • Based on discussions with the FDA, the Company believes they are aligned with the FDA and plans to submit an NDA for govorestat (AT-007) for the treatment of Galactosemia in the fourth quarter of this year.
  • “In the pre-NDA meeting, the FDA expressed their support for a potential NDA based on the govorestat data generated to date in Galactosemia, and provided constructive recommendations for successful NDA acceptance and review,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics.
  • “If approved, govorestat will fill an important unmet need for the Galactosemia community.
  • We are excited to advance towards a regulatory submission for the first potential treatment for Galactosemia.”

Applied Therapeutics Reports Second Quarter 2023 Financial Results

Retrieved on: 
Giovedì, Agosto 10, 2023
Insurance, ChromeOS, Sorbitol dehydrogenase, Chairperson, PIP, Journal, New Drug Application, European Medicines Agency, Diabetic cardiomyopathy, NDA, Quality of life, Medicine, Doctor of Philosophy, Brain cell, EMA, INSPIRE, US, Ongo, Galactitol, Phase 3, Patient, Investment, Treatment, Annual general meeting, Advanz Pharma, MAA, Research, FDA, Therapy, ADA, American Diabetes Association, US FDA, Safety, Disease, Pharmaceutical industry, Birth control, Galactosemia, EU, Sorbitol, SORD

NEW YORK, Aug. 10, 2023 (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 second quarter ended June 30, 2023.

Key Points: 
  • “In the second quarter we continued to advance our programs across Galactosemia, SORD Deficiency and Diabetic Cardiomyopathy,” said Shoshana Shendelman, PhD, Founder, Chief Executive Officer, and Chair of the Board.
  • In June 2023, the Company presented baseline data at the 2023 Annual Meeting of the ADA from the ongoing Phase 3 ARISE-HF study of AT-001 (caficrestat) in Diabetic Cardiomyopathy (DbCM).
  • The Company expects topline data from the study in the fourth quarter of 2023.
  • Net loss for the second quarter of 2023 was $29.6 million, or $0.37 per basic and diluted common share, compared to a net loss of $25.9 million, or $0.96 per basic and diluted common share, for the second quarter 2022.

Applied Therapeutics Provides Regulatory Update on Galactosemia Program

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Martedì, Luglio 25, 2023
PIP, Galactose, New Drug Application, European Medicines Agency, NDA, Doctor of Philosophy, EMA, US, Galactitol, Patient, Advanz Pharma, MAA, FDA, Therapy, US FDA, Pharmaceutical industry, Galactosemia

NEW YORK, July 25, 2023 (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 provided a regulatory update on the Galactosemia program, including updates on the regulatory paths for govorestat (AT-007) in both the U.S. and Europe.

Key Points: 
  • NEW YORK, July 25, 2023 (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 provided a regulatory update on the Galactosemia program, including updates on the regulatory paths for govorestat (AT-007) in both the U.S. and Europe.
  • “We look forward to advancing our regulatory submissions in both the U.S. and Europe,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics.
  • “We believe that govorestat offers compelling efficacy alongside a favorable safety profile and represents a transformative treatment option for patients with Galactosemia.
  • There are currently no approved treatments for Galactosemia, and we hope to bring govorestat to patients as the first treatment for Galactosemia as soon as possible.”

The Galactosemia Foundation and National Organization for Rare Disorders (NORD) Jointly Publish First "Voice of the Patient" Report to Help Inform the FDA and Researchers

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Giovedì, Luglio 6, 2023
DSM-IV codes, NORD, Human, National Organization for Rare Disorders, Patient, Human voice, Drug development, Health, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Galactosemia, Food, Caregiver, External counterpulsation, Disease, Pharmaceutical industry, Medical device, Nursing

WASHINGTON and ALBANY, N.Y., July 6, 2023 /PRNewswire/ -- The Galactosemia Foundation and the National Organization for Rare Disorders (NORD)® today announced they have jointly published the galactosemia community's first "Voice of the Patient" report.

Key Points: 
  • WASHINGTON and ALBANY, N.Y., July 6, 2023 /PRNewswire/ -- The Galactosemia Foundation and the National Organization for Rare Disorders (NORD)® today announced they have jointly published the galactosemia community's first "Voice of the Patient" report.
  • This report provides a comprehensive overview of patient and caregiver experiences with this rare metabolic disease, which currently has no approved treatment options.
  • The report follows a historic Externally Led Patient-Focused Drug Development (EL-PFDD) meeting hosted by the Galactosemia Foundation and NORD last September.
  • The "Voice of the Patient" report is available for download now, and a recording of the entire EL-PFDD meeting on galactosemia can be viewed here .

Govorestat (AT-007) Receives Orphan Medicinal Product Designation from the EMA for Treatment of SORD Deficiency

Retrieved on: 
Giovedì, Maggio 25, 2023
Advanz Pharma, Sorbitol dehydrogenase, Central nervous system, Journal, European Medicines Agency, ATP, Medicine, Cell, Doctor of Philosophy, CNS, Brain cell, Drosophila, EMA, ROS, HIV disease progression rates, Patient, Reactive oxygen species, Sorbitol, Therapy, Adenosine triphosphate, Disease, Pharmaceutical industry, Galactosemia, EU, SORD

Additionally, the Company announced that new data has been published in the Journal of Clinical Investigation on govorestat treatment in models of SORD Deficiency.

Key Points: 
  • Additionally, the Company announced that new data has been published in the Journal of Clinical Investigation on govorestat treatment in models of SORD Deficiency.
  • Applied Therapeutics has partnered govorestat for treatment of SORD Deficiency as well as Galactosemia in Europe with ADVANZ Pharma.
  • The new data on govorestat treatment in models of SORD Deficiency was recently published in the Journal of Clinical Investigation.
  • These findings uncover the molecular and cellular pathophysiology of sorbitol toxicity in SORD neuropathy, and govorestat provides a potential treatment strategy for patients with SORD Deficiency.

Applied Therapeutics Reports First Quarter 2023 Financial Results

Retrieved on: 
Giovedì, Maggio 11, 2023
Cognition, Insurance, Partnership, Tremor, Galactose, Diabetic cardiomyopathy, NDA, Venrock, Doctor of Philosophy, Sale, Galactitol, Patient, Investment, MAA, Research, Therapy, Pharmaceutical industry, Cryptocurrency, Galactosemia, SORD

NEW YORK, May 11, 2023 (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 first quarter ended March 31, 2023.

Key Points: 
  • NEW YORK, May 11, 2023 (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 first quarter ended March 31, 2023.
  • “We are committed to bringing transformative therapies to patients with debilitating diseases and no treatment options, including Galactosemia, SORD Deficiency and Diabetic Cardiomyopathy,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics.
  • In April 2023, the Company announced consistent long-term clinical benefit from the ACTION-Galactosemia Kids Phase 3 study of govorestat.
  • Net loss for the first quarter of 2023 was $10.1 million, or $0.18 per basic and diluted common share, compared to a net loss of $23.1 million, or $0.88 per basic and diluted common share, for the first quarter 2022.

Galactosemia Foundation Urges FDA to Incorporate Voice of the Patient in Review of Potential First Treatment for Children with Life-Altering Rare Disease

Retrieved on: 
Mercoledì, Aprile 26, 2023
Cognition, Tremor, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, European Medicines Agency, New Drug Application, NDA, Hope, Medicine, Emotion, Ansell, DSM-IV codes, Behavior, EMA, Name, Patient, MAA, FDA, Therapy, Caregiver, Disease, Pharmaceutical industry, Galactosemia

The Galactosemia Foundation has shared qualitative data on the patient journey and burden of disease with the FDA on several occasions including an externally led patient-focused drug development meeting held in September 2022, patient and caregiver focus groups and direct outreach to the agency.

Key Points: 
  • The Galactosemia Foundation has shared qualitative data on the patient journey and burden of disease with the FDA on several occasions including an externally led patient-focused drug development meeting held in September 2022, patient and caregiver focus groups and direct outreach to the agency.
  • The results of the ACTION-Galactosemia Kids registrational clinical study evaluating govorestat in children with galactosemia were recently announced.
  • My youngest daughter Louise, who is 18 months old and also lives with galactosemia, may never have access.
  • The galactosemia community has gone more than a century with no approved treatment.