BridgeBio's LGMD2i investigational therapy is one of the Company's 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and genetically-driven cancers.
BridgeBio's first wave of programs are the now-approved drugs for Molybdenum Cofactor Deficiency (MoCD) Type A and previously-treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring an FGFR2 fusion or rearrangement. The second wave of programs includes the Company's four major near-term catalysts for its product candidates for the treatment of transthyretin (TTR) amyloidosis (ATTR), achondroplasia, congenital adrenal hyperplasia (CAH) and autosomal dominant hypocalcemia type 1 (ADH1).
LGMD2i represents one of the leading programs in BridgeBio's ongoing third wave in development, which includes a variety of programs in the cancer and mendelian space already in the clinic.
With approximately 7,000 patients with potentially treatable mutations, LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein (FKRP). FKRP is a critical enzyme that adds a specific sugar molecule to a muscle cell structural protein called alpha-dystroglycan (αDG). Due to defective FKRP enzyme function, muscle cells of patients affected by LGMD2i lack a robust cushioning system that is provided by fully glycosylated αDG proteins. Pediatric and adult patients with LGMD2i most commonly present with upper and lower extremity ("limb") and thoracic ("girdle") dysfunction ("limb-girdle" pattern of weakness), and without treatment often develop additional severe clinical manifestations, including loss of independent ambulation, severe breathing issues which can require mechanical ventilation, cardiomyopathy and premature death.
"As of now, there are no approved treatment options for people born with Limb-girdle Muscular Dystrophy Type 2i. People living with this disease can lose their ability to perform routine daily activities, and ultimately may lose the ability to walk, need ventilatory support or face the risk of heart failure," said
BBP-418 is being investigated as a treatment for LGMD2i. The investigational therapy is designed to overcome the enzymatic limitation of the defective FKRP enzyme by supplementing endogenous sugar molecules to glycosylate αDG and to improve muscle cell integrities, resulting in improved muscle strength and function for patients. Clinical trials to verify the safety and efficacy of BBP-418 are ongoing.
BBP-418 has received Orphan Drug Designation for the treatment of LGMD2i from the FDA and for LGMD from the
About Limb-girdle Muscular Dystrophy Type 2i
LGMD2i is a monogenic autosomal recessive disease caused by partial loss of function mutations in the FKRP gene, and these FKRP mutations impair glycosylation of α-DG, a protein associated with stabilizing muscle cells. LGMD2i is a disease that has pediatric symptomatic onset with most individuals developing manifestations of disease between 5 and 18 years of age. Clinical manifestations typically present as a skeletal myopathy affecting the lower and then upper limbs, which is commonly later accompanied by respiratory muscle and cardiac muscle involvement. Patients who harbor a homozygous genotype typically develop disease manifestations during late childhood with progression to loss of independent ambulation (25%), assisted ventilation (5%), and cardiomyopathy (10%) in adulthood. Cardiomyopathy is progressive, with an annual loss of 0.4% of left ventricular ejection fraction (LVEF). Patients with heterozygous genotypes have an earlier childhood onset with a more severe clinical course, rapid loss of mobility by 20 years of age, more frequent cardiac involvement (25%), and eventual respiratory failure by 30 years of age in nearly all cases.
This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the "Securities Act"), and Section 21E of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), which are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to the timing and success of ML Bio Solutions' clinical trials of BBP-418 for the treatment of LGMD2i, expectations, plans and prospects regarding ML Bio Solutions' regulatory approval process for BBP-418, the ability of BBP-418 to treat LGMD2i in humans, the potential for BBP-418 to be the first approved therapy for the treatment of LGMD2i and the timing and success of BridgeBio's clinical trials and development pipeline, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, ML Bio Solutions' ability to continue and complete its clinical trials of BBP-418 for the treatment of LDMD2i, past data from preclinical studies not being indicative of future data from clinical trials, ML Bio Solutions' ability to advance BBP-418 in clinical development according to its plans, the ability of BBP-418 to be the first approved therapy for the treatment of patients with LGMD2i, BridgeBio's ability to advance its clinical trials and development pipeline, the success of BridgeBio's approved drugs, as well as those risks set forth in the Risk Factors section of
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