Phenylketonuria

Eton Pharmaceuticals Reports First Quarter 2024 Financial Results

Retrieved on: 
Donnerstag, Mai 9, 2024
FDA, Research and development, NDA, Food, Research, Carglumic acid, Administration, Patient, Physician, PKU, Nitisinone, Phenylketonuria, Growth, Pharmaceutical industry

DEER PARK, Ill., May 09, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today reported financial results for the quarter ended March 31, 2024.

Key Points: 
  • •  Q1 2024 revenue of $8.0 million, representing 50% growth over Q1 2023 and the 13th straight quarter of sequential product sales growth
    •  Acquired PKU GOLIKE®, launched Nitisinone, and submitted a new drug application (NDA) for product candidate ET-400
    DEER PARK, Ill., May 09, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today reported financial results for the quarter ended March 31, 2024.
  • We also launched Nitisinone and submitted an NDA for our highly anticipated ET-400 product candidate,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals.
  • Eton reported first quarter 2024 product sales of $8.0 million, representing 50% growth over the prior year, driven primarily by the ongoing momentum of ALKINDI SPRINKLE® and Carglumic Acid.
  • As previously announced, Eton Pharmaceuticals will host a its first quarter 2024 conference call as follows:

National PKU Alliance Names Catherine Warren Executive Director

Retrieved on: 
Donnerstag, Mai 2, 2024
Volunteering, University, Outline of health sciences, National Multiple Sclerosis Society, MD, Research, Jefferson College, PKU, Metabolism, Pediatrics, Phenylketonuria, Growth, Connolly, Management

NEW YORK, May 2, 2024 /PRNewswire/ --The National PKU Alliance (NPKUA), whose mission is to improve the lives of people living with phenylketonuria (PKU) and pursue a cure, today announced Catherine Warren as the organization's new Executive Director.

Key Points: 
  • NEW YORK, May 2, 2024 /PRNewswire/ --The National PKU Alliance (NPKUA), whose mission is to improve the lives of people living with phenylketonuria (PKU) and pursue a cure, today announced Catherine Warren as the organization's new Executive Director.
  • Bringing nearly two decades of healthcare nonprofit development and management experience, Catherine served as Executive Director of the Susan G. Komen Virginia Blue Ridge Affiliate and later as Senior Director of Southwest Virginia for the Virginia Breast Cancer Foundation.
  • Catherine's personal connection to PKU began twelve years ago when her nephew was diagnosed at birth.
  • "I look forward to meeting more members of the PKU community at the bi-annual NPKUA conference this July 18-21, 2024 in Chantilly, Virginia."

Tessera Therapeutics to Present New Data Across Preclinical Programs for its Gene Writing™ and Delivery Platforms at the American Society of Gene and Cell Therapy 27th Annual Meeting

Retrieved on: 
Montag, April 22, 2024
Sickle cell disease, Annual general meeting, Therapy, Gene, Cell, Society, Biotechnology, Phenylketonuria, Vaccine

SOMERVILLE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Tessera Therapeutics, a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, today announced six presentations of new preclinical data at the upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place in Baltimore, Maryland, May 7 – 11, 2024.

Key Points: 
  • SOMERVILLE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Tessera Therapeutics, a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, today announced six presentations of new preclinical data at the upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place in Baltimore, Maryland, May 7 – 11, 2024.
  • “We look forward to presenting multiple new datasets at this year’s ASGCT Annual Meeting, including our progress towards in vivo therapies for phenylketonuria, alpha-1 antitrypsin deficiency, sickle cell disease and Wilson’s disease, as well as our advancements in novel T-cell based therapies,” said Michael Severino, M.D., CEO of Tessera Therapeutics.
  • “We continue to focus on the development of transformative genetic medicines through the applications of our Gene Writing and non-viral delivery platforms.”
    Details of the Company’s ASGCT 27th Annual Meeting presentations are as follows:

Synlogic Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Dienstag, März 19, 2024
Research, AbbVie, Therapy, Roche, PKU, Phenylketonuria

CAMBRIDGE, Mass., March 19, 2024 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a biopharmaceutical company advancing novel therapeutics to transform the care of serious diseases, today reported financial results for the fourth quarter and full year ending December 31, 2023.

Key Points: 
  • CAMBRIDGE, Mass., March 19, 2024 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a biopharmaceutical company advancing novel therapeutics to transform the care of serious diseases, today reported financial results for the fourth quarter and full year ending December 31, 2023.
  • As of December 31, 2023, Synlogic had cash, cash equivalents, and short-term marketable securities of $47.7 million.
  • Revenue was $2.8 million for the three months ended December 31, 2023, compared to $0.1 million for the corresponding period in 2022.
  • Revenues were $3.4 million for the year ended December 31, 2023, compared to $1.2 million for the same period in 2022.

Agios Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Donnerstag, Februar 15, 2024
Agios Pharmaceuticals, Security, AGIO, Total, Research, New Drug Application, ASH, FDA, Research and development, PKU, Growth, Sale, Exposition, Thalassemia, Society, MDS, Phenylketonuria, Administration, Sickle cell disease, Anti-transglutaminase antibodies, Patient, Public expenditure, Net, Small RNA, IND, Pharmaceutical industry

CAMBRIDGE, Mass., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Launch: Generated $7.1 million in U.S. net revenue for the fourth quarter of 2023, a 4 percent decrease from the third quarter of 2023, primarily driven by lower customer inventory levels at the end of the fourth quarter of 2023, partially offset by favorable gross-to-net adjustments.
  • A total of 109 patients are on PYRUKYND® therapy, a 9 percent increase from the third quarter of 2023.
  • Cost of Sales: Cost of sales was $0.6 million for the fourth quarter of 2023 and $2.9 million for the full year ended Dec. 31, 2023.
  • ET to discuss fourth quarter and full year 2023 financial results and recent business highlights.

Synlogic Announces Decision to Discontinue Synpheny-3 Study and Provides Corporate Update

Retrieved on: 
Donnerstag, Februar 8, 2024
Workforce, News, Acquisition, Partnership, Investment, Therapy, Safety, Data monitoring committee, DMC, PKU, Science, Phenylketonuria, Disclosure, Management

CAMBRIDGE, Mass., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a biopharmaceutical company advancing novel therapeutics to transform the care of serious diseases, announced today the decision to discontinue Synpheny-3, its ongoing pivotal study of labafenogene marselecobac (SYNB1934) as a potential treatment for phenylketonuria (PKU). As a result, Synlogic’s management and its Board of Directors have made the decision to evaluate strategic options for the Company.

Key Points: 
  • As a result, Synlogic’s management and its Board of Directors have made the decision to evaluate strategic options for the Company.
  • The decision was not based on concerns regarding safety or tolerability.
  • Synlogic will now work with the Synpheny-3 clinical trial sites involved to implement the discontinuation.
  • "It is with a heavy heart that we share this news, and our resulting decision to end Synpheny-3.

Jnana Therapeutics Announces Positive Clinical Proof of Concept Achieved with JNT-517, a Potential First-in-Class Oral Treatment for PKU

Retrieved on: 
Dienstag, Januar 30, 2024
RAPID, Pharmacokinetics, SLC6A19, Phenylketonuria, BID, Trial of the century, Safety, Medical genetics, Ageing, Jñāna, Blood, Disease, Therapy, Science, Phenylalanine, PKU, Pharmaceutical industry

BOSTON, Jan. 30, 2024 (GLOBE NEWSWIRE) -- Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in individuals with phenylketonuria (PKU). JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes. On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517.

Key Points: 
  • JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes.
  • On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517.
  • “There is an urgent need for an oral, safe, and efficacious therapy for the more than 60% of individuals with PKU not currently on therapy.
  • The study dosed its first participant with PKU in August 2023 and is enrolling individuals aged 18 to 65 at clinical sites in the United States and Australia.

Homology Medicines Reports Third Quarter 2023 Financial Results

Retrieved on: 
Dienstag, November 14, 2023
Pfizer, Safety, Research, CDMO, Workforce, Investment, Clinical trial, Pipeline, PKU, Phenylketonuria, Environment, Pharmaceutical industry

BEDFORD, Mass., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today third quarter 2023 financial results and highlighted recent updates.

Key Points: 
  • - Continuing to Evaluate Strategic Options for the Company and the Pipeline of Genetic Medicines, including HMI-103 -
    BEDFORD, Mass., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today third quarter 2023 financial results and highlighted recent updates.
  • There was no collaboration revenue for the quarter ended September 30, 2023, compared to $0.8 million for the quarter ended September 30, 2022.
  • Research and development expenses for the quarter ended September 30, 2023 were $17.5 million, compared to $25.9 million for the quarter ended September 30, 2022.
  • General and administrative expenses for the quarter ended September 30, 2023 were $6.8 million, compared to $7.8 million for the quarter ended September 30, 2022.

Synlogic Reports Third Quarter 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Donnerstag, November 9, 2023
FDA, Research, PKU, Patent, AbbVie, PAL, International Conference on Nuclear Disarmament, Oslo, 2008, Homocystinuria, Treatment, University, Conference, Progress, Phenylketonuria, Partnership, Head, United States patent law, Methionine, Lead, Pittsburgh, Investment, Inflammatory bowel disease, Nature Metabolism, Nursing, Pharmaceutical industry, Cryptocurrency, Vitro

CAMBRIDGE, Mass., Nov. 09, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company advancing novel, oral, non-systemically absorbed biotherapeutics to transform the care of serious diseases, today reported financial results for the third quarter ended September 30, 2023, and provided a corporate update.

Key Points: 
  • Publication of Synpheny-1 Phase 2 study results for the PKU program in the journal Nature Metabolism.
  • Presentation of Synpheny-1 Phase 2 study results by lead investigator Dr. Jerry Vockley of the University of Pittsburgh at the 37th E.S.PKU Conference 2023.
  • Revenue for the three months ended September 30, 2023 was $0.4 million compared to $0.7 million for the corresponding period in 2022.
  • General and administrative expenses were $3.4 million for the three months ended September 30, 2023, compared to $4.4 million for the corresponding period in 2022.

Agios Reports Business Highlights and Third Quarter 2023 Financial Results

Retrieved on: 
Donnerstag, November 2, 2023
Anti-transglutaminase antibodies, Research, RISE, PKU, Research and development, IND, AGIO, Rare, Sickle cell disease, MDS, Public expenditure, Enrollment, SG, Pyruvate kinase deficiency, R, Society, Phenylketonuria, ASH, Thalassemia, PK, Security, Abstract, Phenylalanine hydroxylase, TMPRSS6, Agios Pharmaceuticals, PAH, Agios, Patient, Administration, Nursing, Cryptocurrency, Pharmaceutical industry, Medical device, Video game, Alnylam Pharmaceuticals

CAMBRIDGE, Mass., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the third quarter ended September 30, 2023.

Key Points: 
  • Launch: Generated $7.4 million in U.S. net revenue for the third quarter of 2023, a 10 percent increase over the second quarter of 2023.
  • A total of 160 unique patients have completed prescription enrollment forms, representing an increase of 9 percent over the second quarter of 2023.
  • Net Loss: Net loss was $91.3 million for the third quarter of 2023 compared to $81.7 million for the third quarter of 2022.
  • ET to discuss third quarter 2023 financial results and recent business activities.