Phenylketonuria

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

Retrieved on: 
Thursday, February 15, 2024

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: 
Thursday, February 8, 2024

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: 
Tuesday, January 30, 2024

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: 
Tuesday, November 14, 2023

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: 
Thursday, November 9, 2023

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: 
Thursday, November 2, 2023

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.

MediciNova’s Collaborator Initiates Clinical Development of a Gene Therapy Product for the Treatment of Phenylketonuria

Retrieved on: 
Friday, October 27, 2023

LA JOLLA, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), today announced that its collaborator Genzyme Corporation, a subsidiary of Sanofi, has treated the first patient in a clinical trial of SAR444836 for the treatment of phenylketonuria (PKU).

Key Points: 
  • LA JOLLA, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), today announced that its collaborator Genzyme Corporation, a subsidiary of Sanofi, has treated the first patient in a clinical trial of SAR444836 for the treatment of phenylketonuria (PKU).
  • SAR444836 is a phenylalanine hydroxylase (PAH) replacement gene therapy product based on adeno-associated virus (AAV) vector technology which is covered under MediciNova’s assignment agreement with Genzyme Corporation.

Synlogic Announces Publication of Synpheny-1 Phase 2 Study of Synthetic Biotic for Phenylketonuria in Nature Metabolism

Retrieved on: 
Thursday, September 28, 2023

CAMBRIDGE, Mass., Sept. 28, 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 announced the publication of results from its Synpheny-1 Phase 2 study of its drug candidates, labafenogene marselecobac (SYNB1934) and SYNB1618 in patients with phenylketonuria (PKU) in the journal Nature Metabolism.

Key Points: 
  • CAMBRIDGE, Mass., Sept. 28, 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 announced the publication of results from its Synpheny-1 Phase 2 study of its drug candidates, labafenogene marselecobac (SYNB1934) and SYNB1618 in patients with phenylketonuria (PKU) in the journal Nature Metabolism.
  • The publication, entitled “Efficacy and Safety of a Synthetic Biotic for Treatment of Phenylketonuria: a Phase 2 Clinical Trial” is now available online at https://www.nature.com/articles/s42255-023-00897-6 .
  • The paper includes a comprehensive review of the data, showing clinically meaningful, dose-dependent reductions in plasma phenylalanine (Phe).
  • “These positive results build on a body of data supporting the role of labafenogene marselecobac for patients with PKU,” said Dr. Neal Sondheimer, Head of Clinical Development at Synlogic.

Synlogic Announces CEO Aoife Brennan Named to PharmaVoice 100

Retrieved on: 
Tuesday, September 19, 2023

CAMBRIDGE, Mass., Sept. 19, 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 announced that CEO Aoife Brennan, M.B.

Key Points: 
  • CAMBRIDGE, Mass., Sept. 19, 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 announced that CEO Aoife Brennan, M.B.
  • Ch.B., has been honored as a PharmaVoice 100 winner, a list recognizing the most inspiring people in the life sciences industry.
  • PharmaVoice is a life sciences industry publication operated by Industry Dive that strives to deliver the most important voices and ideas to industry leaders.
  • “I am honored to be selected by PharmaVoice as a leader in our industry,” said Dr. Brennan.

Jnana Therapeutics Presents Additional Data from Phase 1a Clinical Study of JNT-517 at SSIEM Annual Symposium

Retrieved on: 
Thursday, August 31, 2023

BOSTON, Aug. 31, 2023 (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 that additional data from its Phase 1a clinical trial of JNT-517 in healthy volunteers were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023. JNT-517 is a first-in-class, oral, allosteric inhibitor of the phenylalanine (Phe) transporter SLC6A19 that Jnana is developing for the treatment of phenylketonuria (PKU). The data were presented by Cary O. Harding, M.D., study investigator and Professor of Molecular and Medical Genetics at Oregon Health and Science University School of Medicine.

Key Points: 
  • JNT-517 is a first-in-class, oral, allosteric inhibitor of the phenylalanine (Phe) transporter SLC6A19 that Jnana is developing for the treatment of phenylketonuria (PKU).
  • The data were presented by Cary O. Harding, M.D., study investigator and Professor of Molecular and Medical Genetics at Oregon Health and Science University School of Medicine.
  • The Phase 1a study enrolled 64 healthy adults in a randomized, double-blind, placebo-controlled trial to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of JNT-517 across single (SAD) and multiple (MAD) ascending dose cohorts.
  • The ongoing Phase 1b study will assess the impact of JNT-517 on plasma Phe levels in individuals with PKU.