Homocystinuria

Travere Therapeutics to Present Abstracts at the Society for Inherited Metabolic Disorders and Genetic Metabolic Dieticians International

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Thursday, April 4, 2024

Homocysteine, Homocystinuria, Caregiver, SAN, HCU, Society, Treatment, Therapy, Genetic, SIMD, Pharmaceutical industry

SAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for Inherited Metabolic Disorders (SIMD) in Charlotte, North Carolina from April 14-17, 2024, and Genetic Metabolic Dieticians International (GMDI) in Charlotte, North Carolina from April 17-20, 2024.

Key Points:

SAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for Inherited Metabolic Disorders (SIMD) in Charlotte, North Carolina from April 14-17, 2024, and Genetic Metabolic Dieticians International (GMDI) in Charlotte, North Carolina from April 17-20, 2024.
At SIMD and GMDI, the Company will present the trial design of the pivotal Phase 3 HARMONY Study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical HCU.
Additionally, the Company will share insights on the development of an innovative tool used for dietary management and monitoring in the Phase 3 HARMONY Study and open-label extension ENSEMBLE Study, and the positive results from cohort 6 in the placebo-controlled Phase 1/2 COMPOSE Study of pegtibatinase in classical HCU.
“Classical HCU is an isolating and devastating rare disorder with very limited treatment options, including adherence to highly restrictive diets, leaving patients and their caregivers with immense challenges,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics.

Eton Pharmaceuticals Announces Acquisition of PKU GOLIKE® for Phenylketonuria

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Friday, March 22, 2024

Brain, Taste, Nitisinone, Partnership, Education, Patient, PKU, Phenylalanine, DSM-IV codes, Insurance, Protein, APR, Diet, Homocystinuria, Patent, OTCQB, FDA, Practitioner, Pharmaceutical industry

After extensive discussions with metabolic geneticists, dieticians, and PKU patients, we believe PKU GOLIKE is the best product in the estimated $100 million U.S. PKU medical formula market.

Key Points:

After extensive discussions with metabolic geneticists, dieticians, and PKU patients, we believe PKU GOLIKE is the best product in the estimated $100 million U.S. PKU medical formula market.
PKU GOLIKE’s taste-masked, odor-free coating technology is designed to provide a better taste and a superior experience compared to alternative PKU medical formulas.
Relief launched PKU GOLIKE Granules in the United States in the fourth quarter of 2022 and the PKU GOLIKE Tropical Bar in the second quarter of 2023.
Eton plans to promote PKU GOLIKE with its existing metabolic sales force, which currently promotes Eton’s Carglumic Acid, Betaine, and Nitisinone products.

Fulcrum Therapeutics Appoints Patrick Horn M.D., Ph.D., as Chief Medical Officer

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Monday, March 18, 2024

CAMBRIDGE, Mass., March 18, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced the appointment of Patrick Horn, M.D., Ph.D., as chief medical officer, effective immediately. Dr. Horn is a seasoned executive with over 20 years of end-to-end drug development experience spanning multiple therapeutic areas, with an emphasis on rare diseases, across both large pharmaceutical and biotech companies. Interim chief medical officer, Iain Fraser, MBChB, DPhil, will continue to serve on Fulcrum’s executive leadership team as SVP of early development. Together, Drs. Horn and Fraser will be responsible for leading clinical development and overseeing regulatory strategy and execution.

Key Points:

Interim chief medical officer, Iain Fraser, MBChB, DPhil, will continue to serve on Fulcrum’s executive leadership team as SVP of early development.
Horn and Fraser will be responsible for leading clinical development and overseeing regulatory strategy and execution.
His most recent role was as the Chief Medical Officer at HemoShear Therapeutics, specializing in rare metabolic diseases.
Prior to transitioning to industry, Dr. Horn was a practicing pediatrician at major academic institutions in Chicago.

Synlogic Announces Abstract Accepted for Poster Presentation at the International Conference on Microbiome Engineering 2023

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Thursday, December 7, 2023

HCU, Homocystinuria, Poster, Conference, AbbVie

CAMBRIDGE, Mass., Dec. 07, 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, announced that an abstract outlining the SYNB1353 program as a potential treatment for homocystinuria (HCU), including findings from fermentation process improvements to increase activity of methionine degradation, has been accepted and will be presented at the International Conference on Microbiome Engineering 2023, held in Berkeley, California on December 8th to 10th.

Key Points:

CAMBRIDGE, Mass., Dec. 07, 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, announced that an abstract outlining the SYNB1353 program as a potential treatment for homocystinuria (HCU), including findings from fermentation process improvements to increase activity of methionine degradation, has been accepted and will be presented at the International Conference on Microbiome Engineering 2023, held in Berkeley, California on December 8th to 10th.
Poster presentation details are as follows:
For more information, please visit the conference website .
The poster will be available following the conference on the Synlogic website.

Synlogic Reports Third Quarter 2023 Financial Results and Provides Corporate Update

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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.

Travere Therapeutics to Present Abstracts at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023

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Thursday, August 24, 2023

DSM-IV codes, Society, Prevalence, Homocystinuria, Burden, ICC, HCU, IDT, Exhibition Hall of the Ministry of Culture, Cystathionine beta synthase, SAN, Therapy, Report of the 1st Annual Symposium on Relaxed Improvisation, Pharmaceutical industry, Metabolism

SAN DIEGO, Aug. 24, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the Company will present clinical data from the Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU), at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem, Israel, August 29 – September 1, 2023.

Key Points:

SAN DIEGO, Aug. 24, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the Company will present clinical data from the Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU), at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem, Israel, August 29 – September 1, 2023.
The Company and its collaborators will also present analyses on its prospective HCU natural history study, the prevalence of HCU, and the burden of HCU from the patient perspective highlighting the devastating nature of this rare metabolic disease over patients’ lifetimes and underscoring the urgent need for new treatments.
In addition, the Company will present data on the clinical burden of HCU and the relationship between total homocysteine (tHcy) and clinical outcomes, which has been recognized as one of the highest ranked posters at SSIEM.
Clinical Characterization of Classical Homocystinuria Due to Cystathionine-beta Synthase Deficiency: Results from the ACAPPELLA Study
Clinical Burden of Classical Homocystinuria in the United States: A Retrospective Analysis of Optum Market Clarity
Understanding the Burden of Classical Homocystinuria (HCU) from the Patient’s Perspective: A Qualitative Study

SWK Holdings Highlights Recent Achievements and Provides Portfolio Update

Retrieved on:

Monday, July 17, 2023

We view repurchasing shares at the current discount to book as a highly accretive use of capital that benefits shareholders."

Key Points:

We view repurchasing shares at the current discount to book as a highly accretive use of capital that benefits shareholders."
SWK has received $1.5mm of proceeds from the sale and anticipates receiving approximately $0.5 million in additional proceeds from the estate.
SWK will provide an update on the Flowonix finance receivable in our second quarter earnings release.
Signed a partnership with LeMaitre Vascular to distribute Aziyo's cardiovascular repair portfolio within the U.S., increasing sales representative coverage five-fold.

Synlogic Announces Data Presentations at the 2023 Synthetic Biology: Engineering, Evolution & Design (SEED) Conference

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Wednesday, May 24, 2023

Homocysteine, DSM-IV codes, Therapy, Engineering, Evolution, Publication, SEED, Research and development, Homocystinuria, HCU, Metabolism, Patient, AbbVie, Synthetic, Synthetic biology, Treatment, Pharmaceutical industry, Research

CAMBRIDGE, Mass., May 24, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), the leading company advancing therapeutics based on synthetic biology, today announced its planned presentations at the 2023 Synthetic Biology: Engineering, Evolution & Design (SEED) conference being held May 30 – June 2, 2023 in Los Angeles, CA.

Key Points:

CAMBRIDGE, Mass., May 24, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), the leading company advancing therapeutics based on synthetic biology, today announced its planned presentations at the 2023 Synthetic Biology: Engineering, Evolution & Design (SEED) conference being held May 30 – June 2, 2023 in Los Angeles, CA.
The company’s oral presentation focuses on Synlogic’s development of a Synthetic Biotic drug candidate for the potential treatment of homocystinuria (HCU), a rare metabolic disease caused by an inborn error of metabolism.
The company’s poster presentations highlight preclinical and clinical data.
“The data being presented by our team at SEED this year showcases the extraordinary progress our Synthetic Biotic platform has achieved across multiple therapeutic categories,” said Dave Hava, Chief Scientific Officer and Head of Research and Development at Synlogic.

Synlogic Reports First Quarter 2023 Financial Results and Provides Business Update

Retrieved on:

Thursday, May 11, 2023

Therapy, ODD, Roche, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, AbbVie, PKU, EMA, Homocystinuria, HCU, DSM-IV codes, Investment, Research, FDA, Conference, Synthetic biology, Food, Pharmaceutical industry, Cryptocurrency

CAMBRIDGE, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), the leading company advancing therapeutics based on synthetic biology, today reported financial results for the first quarter ended March 31, 2023, and provided an update on its pipeline programs.

Key Points:

Revenue was $0.2 million for each of the three months ended March 31, 2023 and March 31, 2022.
Research and development expenses were $12.5 million for the three months ended March 31, 2023, compared to $11.7 million for the corresponding period in 2022.
General and administrative expenses were $4.0 million for the three months ended March 31, 2023, compared to $4.3 million for the corresponding period in 2022.
Synlogic leadership will attend the Jefferies Global Healthcare Conference being held June 7-9, 2023, in New York City.

Eton Pharmaceuticals Announces Commercial Availability of Betaine Anhydrous for Oral Solution, a Generic Version of Cystadane® (betaine anhydrous for oral solution)

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Wednesday, May 10, 2023

DEER PARK, Ill., May 10, 2023 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the commercial availability of Betaine Anhydrous for Oral Solution 180 grams for the treatment of homocystinuria.

Key Points:

“We are excited to be offering a lower cost alternative1 to Cystadane while still providing full patient and provider support services.
Betaine Anhydrous is available exclusively through Optime Care, a specialty pharmacy dedicated to helping patients with rare diseases manage their conditions.
Optime Care will administer the Eton Cares Program in partnership with Eton Pharmaceuticals.
Betaine anhydrous for oral solution is indicated in children and adults for the treatment of homocystinuria to decrease high homocysteine blood levels.