GlobeNewswire Inc | Feb 19, 2021 07:30AM EST

February 19, 2021

SAN FRANCISCO, Feb. 19, 2021 (GLOBE NEWSWIRE) -- BridgeBioPharma, 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,andaffiliateML BioSolutionstoday announcedthatthefirst patient has been dosed inaPhase2trial ofBBP-418 in patients with LGMD2i.BridgeBioand ML Bios BBP-418 isthe first-ever oral disease-modifying investigational treatment for LGMD2i.BBP-418wasgranted Orphan Drug Designation for LGMD2i by the US Food and Drug Administration (FDA) in 2019,andfor LGMD by the European Medicines Agency (EMA) in 2020.

LGMD2iis aninheritedrecessivemuscular dystrophy caused bymutation offukutin-related protein (FKRP).FKRP is an enzyme that adds a sugar moleculeto a criticalmuscle cellstructural proteincalled alpha-dystroglycan(DG). Due to defective FKRP enzyme function, muscle cells of patients affected by LGMD2ilack the structural integrity that is provided by fully glycosylatedDG protein. This leads to chronic muscle injury and loss, muscle weakness and disability.Many affectedpatientsultimatelylose their ability to walk,and somedevelop a need for ventilatory support ordie from heart failure.Currently,there are no disease-modifying therapies forpeople withLGMD2i.

BBP-418 is designed to bypass the metabolic defect in LGMD2ibyproviding theFKRPenzymewith precursor sugar moleculesthatsupplementthebodysnatural sugarsused by FKRPto glycosylate theDGproteinon muscle cells. BBP-418 represents a novel substraterescueapproachwith 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 DG 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 withLGMD2iwho lack treatment options, saidDr.Douglas Sproule, M.D. M.Sc.,ChiefMedicalOfficerof ML BioSolutions.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 defineLGMD2i-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 tocontinueourcollaboration withML Bio Solutionson the lead-instudy that is currentlyenrollingandlook forward tofurtherextending itbylaunchingthe first-in-patienttreatment study for BBP-418.Ourclinicalpartnershipis seeking tovalidate the biomarker assays andestablish the registrationalclinical outcome measures that are needed to bring BBP-418 to LGMD2ipatients, 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-LGMDconsortium.

AboutGRASP-LGMD (Genetic Resolution and Assessments Solving Phenotypes in LGMD)ConsortiumThe GRASP-LGMDconsortiumassemblesan internationalteam of neuromuscular specialists, basic scientists, physical therapists, geneticists, informaticians, and patient advocates to address issues relatedto:diagnostics; outcome measure development, patient engagement; and therapeutic development, to advance thestate 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 SolutionsML Bio Solutions,anaffiliateofBridgeBioPharmabased 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, visitmlbiosolutions.com.

AboutBridgeBioPharmaBridgeBiois a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source.BridgeBiowas 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.BridgeBiospipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visitbridgebio.com.

BridgeBioPharma Forward-Looking StatementsThis 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 tothe timing and success of ML BioSolutions Phase 2 clinical trial of BBP-418for the treatment of LGMD2i,expectations, plans and prospects regarding ML BioSolutions regulatory approval process for BBP-418,the ability of BBP-418 to treat LGMD2iin humans,andthe timing and success ofinitial top-line Phase 2 date of BBP-418, 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 bya number ofrisks, uncertainties and assumptions, including, but not limited to,MLBioSolutionsabilityto continue and complete its Phase 2 clinical trial of BBP-418for the treatment of LDMD2i,the continuing success of ML BioSolutions collaboration with the GRASP-LGMDconsortiumbased at Virginia Commonwealth University,past datafrom preclinical studiesnot being indicative of future datafrom clinical trials,ML BioSolutionsability to advanceBBP-418in clinical development according to its plans, the ability of BBP-418 to treat LGMD2i, as well as those risks set forth in the Risk Factors section ofBridgeBioPharmas most recent Quarterly Report on Form 10-Q andBridgeBioPharmas other SEC filings. Moreover,ML BioSolutionsoperates 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.

Contact:Grace Rauh,BridgeBioPharmagrace.rauh@bridgebio.com917-232-5478

Source:BridgeBioPharma