BridgeBio Pharma and Affiliate ML Bio Solutions Announce Dosing of First Patient in Phase 2 Trial of BBP-418 in Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)

SAN FRANCISCO, CA, USA I February 19, 2021 I BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, and affiliate ML Bio Solutions today announced that the first patient has been dosed in a Phase 2 trial of BBP-418 in patients with LGMD2i. BridgeBio and ML Bio’s BBP-418 is the first-ever oral disease-modifying investigational treatment for LGMD2i. BBP-418 was granted Orphan Drug Designation for LGMD2i by the US Food and Drug Administration (FDA) in 2019, and for LGMD by the European Medicines Agency (EMA) in 2020.

LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein (FKRP). FKRP is an enzyme that adds a sugar molecule to a critical muscle cell structural protein called alpha-dystroglycan (αDG). Due to defective FKRP enzyme function, muscle cells of patients affected by LGMD2i lack the structural integrity that is provided by fully glycosylated αDG protein. This leads to chronic muscle injury and loss, muscle weakness and disability. Many affected patients ultimately lose their ability to walk, and some develop a need for ventilatory support or die from heart failure. Currently, there are no disease-modifying therapies for people with LGMD2i. 

BBP-418 is designed to bypass the metabolic defect in LGMD2i by providing the FKRP enzyme with precursor sugar molecules that supplement the body’s natural sugars used by FKRP to glycosylate the αDG protein on muscle cells. BBP-418 represents a novel substrate rescue approach with the potential to improve muscle strength and function. 

The Phase 2 trial is expected to enroll up to 16 patients with a genetically-confirmed diagnosis of LGMD2i. In addition to safety, key efficacy measurements include changes in muscle αDG glycosylation levels, and changes in functional measures including 10 meter walk and North Star for Dysferlinopathy (NSAD).

“The start of our Phase 2 trial is a key milestone in our effort to develop a safe and efficacious therapy for patients with LGMD2i who lack treatment options,” said Dr. Douglas Sproule, M.D. M.Sc., Chief Medical Officer of ML Bio Solutions. “This trial launch is an outgrowth of our strong partnership with the GRASP-LGMD consortium based at Virginia Commonwealth University, where we are currently enrolling a lead-in study to define LGMD2i-specific biomarkers and functional endpoints. Our hope is that the ongoing lead-in study, together with early data from our Phase 2 trial, will hasten the successful execution of a future registrational trial and ultimately advance a meaningful medicine for patients in need.” 

“We are pleased to continue our collaboration with ML Bio Solutions on the lead-in study that is currently enrolling and look forward to further extending it by launching the first-in-patient treatment study for BBP-418. Our clinical partnership is seeking to validate the biomarker assays and establish the registrational clinical outcome measures that are needed to bring BBP-418 to LGMD2i patients,” said Dr. Nicholas Johnson M.D. M.S.C.I., Associate Professor of Neurology and Chair of Research at Virginia Commonwealth University and Director of the GRASP-LGMD consortium.  

About GRASP-LGMD (Genetic Resolution and Assessments Solving Phenotypes in LGMD) Consortium  
The GRASP-LGMD consortium assembles an international team of neuromuscular specialists, basic scientists, physical therapists, geneticists, informaticians, and patient advocates to address issues related to: diagnostics; outcome measure development, patient engagement; and therapeutic development, to advance the state of LGMD research and readiness to support translation of science into therapeutic development.  For more information visit: http://www.grasp-lgmd.org 

About ML Bio Solutions 
ML Bio Solutions, an affiliate of BridgeBio Pharma based in a Charlotte, NC, is a biotechnology company focused on developing a small molecule as an oral substrate supplementation therapy for LGMD2i. ML Bio Solutions is led by a team of veteran biotechnology executives, and together with patients and physicians, aims to bring safe, effective treatments to market as quickly as possible. For more information, visit mlbiosolutions.com. 

About BridgeBio Pharma 
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com

SOURCE: BridgeBio Pharma

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