PKU

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.

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 8, 2024

SOUTH PLAINFIELD, N.J., Jan. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, at 10:30am EST/7:30am PST. Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2023 accomplishments and highlight potential 2024 value-creating milestones. Preliminary 2023 unaudited financial results and 2024 financial guidance will also be provided. The presentation is being webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com.

Key Points: 
  • Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2023 accomplishments and highlight potential 2024 value-creating milestones.
  • The presentation is being webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com .
  • PTC announced strategic portfolio prioritizations, which resulted in reductions in both operating expenses and headcount of approximately 25% and 30%, respectively.
  • PTC maintains all economics associated with up to $250 million in the remaining commercial sales milestones associated with Evrysdi global net sales.

Synlogic Provides Corporate Update and Outlook for 2024

Retrieved on: 
Thursday, January 4, 2024

CAMBRIDGE, Mass., Jan. 04, 2024 (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 summarized accomplishments for 2023 and anticipated key milestones for 2024.

Key Points: 
  • “We are pleased with the important corporate progress we achieved in 2023 - highlighted by the initiation of our global pivotal Synpheny-3 study evaluating our potentially transformative treatment option for PKU,” said Aoife Brennan, M.B.
  • Ch.B., Synlogic President and Chief Executive Officer.
  • “We expect 2024 to be similarly momentous, with the achievement of key milestones, including the upcoming Data Monitoring Committee review of initial study data.
  • More information on the Synpheny-3 study is available at www.clinicaltrials.gov, identifier NCT05764239 and also by visiting pkuresearchstudy.com.

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.

ARTBIO Announces Appointment of Nick Pullen, Ph.D., as Chief Scientific Officer

Retrieved on: 
Thursday, October 5, 2023

CAMBRIDGE, Mass. and OSLO, Norway and LONDON and BASEL, Switzerland, Oct. 5, 2023 /PRNewswire/ -- ARTBIO, Inc. (ARTBIO), a clinical-stage radiopharmaceutical company developing a new class of targeted alpha radioligand therapies (ART), today announced the appointment of Nick Pullen, Ph.D., as Chief Scientific Officer. Dr. Pullen brings over 20 years of leadership experience in the biopharmaceutical space and has led drug discovery programs spanning multiple gene classes, modalities and indications, including oncology, neuroscience and immunology. In this role, Dr. Pullen will be responsible for overseeing all scientific research efforts to further leverage ARTBIO's scientific approach to radiopharmaceuticals and progress the company's differentiated pipeline.

Key Points: 
  • and OSLO, Norway and LONDON and BASEL, Switzerland, Oct. 5, 2023 /PRNewswire/ -- ARTBIO, Inc. (ARTBIO), a clinical-stage radiopharmaceutical company developing a new class of targeted alpha radioligand therapies (ART), today announced the appointment of Nick Pullen, Ph.D., as Chief Scientific Officer.
  • In this role, Dr. Pullen will be responsible for overseeing all scientific research efforts to further leverage ARTBIO's scientific approach to radiopharmaceuticals and progress the company's differentiated pipeline.
  • "We are pleased to welcome Nick to our team at this pivotal time for ARTBIO.
  • Nick brings a wealth of highly relevant and valuable expertise in discovery and development of innovative therapies across multiple disease areas," said Emanuele Ostuni, Ph.D., Chief Executive Officer of ARTBIO.