Friday, September 30, 2022

561.316.3330

Biotechnology News Magazine

MBX Biosciences Receives Orphan Drug Designation for Lead Candidate MBX 2109 for the Treatment of Hypoparathyroidism

Latest Posts

City of Hope to Accelerate Immunotherapy Research & Treatment Innovation with $15 Mil Gift from Ted Schwartz Family

Ted Schwartz, who is now cancer free, achieved complete remission at City of Hope in 2020 with the center's leading CAR T cell therapy after a 16-year battle with lymphoma, provided the gift to City of Hope to advance treatment options that offer better outcomes and quality of life for people living with cancer.

Neurocrine Biosciences Appoints Dr Ingrid Delaet as Chief Regulatory Officer

Prior to joining Neurocrine Biosciences, Dr Ingrid Delaet served as Senior Vice President, Regulatory Affairs at Intercept Pharmaceuticals, which she joined in 2016.

Astrea Bioseparations Introduces Nereus LentiHERO, a Fit-for-purpose Solution for Lentiviral Vector Purification

“We believe that AstreAdept will be a game-changer,” explained Astrea Bioseparations’ CEO Terry Pizzie. “Our approach was to rapidly develop and incorporate this material into the Nereus LentiHERO, a simple, fit-for-purpose device that radically transforms how lentivirus can be purified [in terms of speed, recovery, and efficiency].

At Pack Expo, Schreiner MediPharm to Debut Functional Labels Designed from More Sustainable Materials

Schreiner MediPharm advises he new label concepts are based on existing items in Schreiner MediPharm’s roster of functional labeling solutions.

MBX Biosciences, Inc., a clinical stage biopharmaceutical company developing precision endocrine peptide therapeutics to treat endocrine disorders, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to its lead therapeutic product candidate, MBX 2109, for the treatment of hypoparathyroidism.

“This is an exciting achievement for MBX and underscores the significant need to expand treatment options for hypoparathyroidism,” said Kent Hawryluk, President and Chief Executive Officer of MBX Biosciences. “We believe MBX 2109, currently in a Phase 1 clinical trial, has the potential to overcome limitations of the current standard of care with fewer administrations necessary due to its long-acting profile. Our goal is to improve the outcomes and quality of life for patients with this chronic condition.”

Orphan drug designation is designed to support the development of medicines addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.

MBX 2019 is an investigational long-acting parathyroid hormone (PTH) prodrug. It is currently undergoing evaluation in a Phase 1 randomized, double blind, placebo-controlled trial to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses of MBX 2109 in healthy adult subjects. For additional information about the trial, please visit clinicaltrials.gov using the identifier NCT05158335.

About Hypoparathyroidism

Hypoparathyroidism (HP) is a rare endocrine disorder caused by a deficiency of parathyroid hormone (PTH) that results in decreased calcium and increased phosphorus levels in the blood. There are approximately 200,000 individuals worldwide with HP. The clinical manifestations are broad affecting a large number of organ systems, especially the kidney and bone which are major target organs for PTH. The persistence of symptoms and development of complications often diminish a person’s quality of life. In contrast to most hormone deficiencies for which hormone replacement therapy is the mainstay of therapy, HP has conventionally been treated with calcium supplements and active vitamin D. This approach can maintain the serum calcium level but may require high doses thereby contributing to the risk of long-term kidney complications.

About MBX 2109

MBX 2109 is an investigational long-acting parathyroid hormone (PTH) prodrug in development as a PTH replacement therapy. It was designed with the company’s novel, proprietary prodrug chemistry platform to provide sustained PTH activity with a convenient once-weekly dosing regimen to simplify an individual’s disease management, while relieving both the symptoms of the disorder and long-term complications.

Latest Posts

Learn More

spot_img

Subscribe

spot_img

Our Sister Publication

Medical Device News Magazine