BridgeBio Pharma, Inc. to Host Investor Call to Discuss Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)
PALO ALTO, CA – March 11, 2022 — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that it will host an investor call on March 14, 2022, at 8:00 AM ET to discuss data from its Phase 2 study of BBP-418 in patients with limb-girdle muscular dystrophy type 2i (LGMD2i). The results will be featured at the Muscular Dystrophy Association (MDA) 2022 Annual Meeting, taking place in Nashville, Tennessee on March 13 – 16, 2022.
With approximately 7,000 patients in the U.S. and European Union with potentially treatable mutations, LGMD2i is an inherited autosomal recessive muscular dystrophy caused by partial loss of function mutations in fukutin-related protein (FKRP) which results in hypoglycosylation of alpha-dystroglycan (αDG). BBP-418 is being investigated to function as a substrate supplementation therapy that is hypothesized to drive glycosylation of αDG and target the root cause of the disease. If the development program is successful, we believe BBP-418 could be the first approved therapy for the treatment of patients with LGMD2i.
Focused execution is BridgeBio’s top priority as it advances its pipeline of high-quality programs to help patients as quickly as possible. The Company remains dedicated to strategically developing and delivering transformative medicines for genetic diseases and cancers with unmet need.
BridgeBio will host an investor call and simultaneous webcast to discuss Phase 2 data for limb-girdle muscular dystrophy type 2i on March 14, 2022 at 8:00 AM ET. To access this call, dial 800-379-2666 and enter conference ID 3562087. A link to the webcast may be accessed from the event calendar page of BridgeBio’s website at https://investor.bridgebio.com/. A replay of the conference call and webcast will be archived on the Company’s website and will be available for at least 30 days following the event.
About Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)
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 αDG, 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.
About BridgeBio Pharma, Inc.
BridgeBio Pharma Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials, and its commercial organization is focused on delivering the company’s first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.
BridgeBio Pharma, Inc. Forward-Looking Statements
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 BridgeBio’ clinical trials of BBP-418 for the treatment of LGMD2i, expectations, plans and prospects regarding BridgeBio’ 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, BridgeBio’ ability to continue and complete its clinical trials of BBP-418 for the treatment of LGMD2i, past data from preclinical studies not being indicative of future data from clinical trials, BridgeBio’ 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 BridgeBio Pharma’s Annual Report on Form 10-K for the year ended December 31, 2021, and BridgeBio Pharma’s other SEC filings. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
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