ML Bio Solutions, Inc.

Muscular dystrophies are a group of genetic muscle disorders characterized by progressive muscle weakness. There are more than 40 different variations, and the most common ones in children are Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). LAS VEGAS, Nov. 6, 2023 /PRNewswire/ -- Muscular dystrophies encompass a range of muscle disorders resulting from genetic mutations. Over time, these conditions lead to muscle weakness, which hinders everyday activities. Muscular dystrophy takes various forms, each targeting specific muscle groups and manifesting at different ages with varying degrees of severity. It can either be hereditary or occur as a spontaneous genetic mutation. Additionally, there may be multiple genetic subtypes within each specific type of muscular dystrophy, and individuals with the same subtype may exhibit varying symptoms. DelveInsight has recently released a series of epidemiology-based market reports focusing on muscular dystrophy including Becker Muscular Dystrophy, Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, and Limb-girdle Muscular Dystrophy. These reports include a comprehensive understanding of current treatment practices, historical and forecasted patient pool, emerging drugs, market share of individual therapies, and historical and forecasted market size from 2019 to 2032 segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]. Additionally, the reports feature exhaustive analysis of prominent companies working with their emerging candidates in different stages of clinical development. Let's deep dive into the assessment of these muscular dystrophy reports market individually. Becker Muscular Dystrophy Market Becker muscular dystrophy (BMD) is a genetic disorder that primarily impacts muscle structure. It leads to the gradual deterioration of limb, cardiac, and skeletal muscles. Interestingly, involuntary muscles remain unaffected. BMD is considered a milder form of Duchenne muscular dystrophy (DMD). Typically, BMD manifests in one's teenage years or early adulthood, and its progression is slower and less predictable compared to DMD. As per DelveInsight analysis, it can be observed that prevalence of BMD is high in the 7MM, and similarly, high rate of treatment is seen in patients. The approach to BMD treatment centers on symptom management since there is no definitive cure available. Instead, there are treatments designed to alleviate symptoms and enhance muscle functionality. It's essential for individuals living with BMD to maintain their physical conditioning and muscle usage. While there are no FDA-approved drugs specifically for BMD, researchers are actively investigating various therapeutic interventions. As per DelveInsight analysis, the dynamics of the becker muscular dystrophy market are anticipated to change in the coming years owing to the positive outcomes of the emerging pipeline candidates during the developmental stage by key players that are under phase III, II and I clinical development have the potential to create a significant positive shift in BMD market size. Becker Muscular Dystrophy Pipeline Therapies and Companies EDG-5506: Edgewise Therapeutics, Inc./Medpace, Inc. Vamorolone: ReveraGen BioPharma, Inc./Santhera Pharmaceuticals For a comprehensive view of the Becker muscular dystrophy market, check out the Becker Muscular Dystrophy Market Assessment Myotonic Dystrophy Market Myotonic dystrophy, an umbrella term for two rare genetic disorders with autosomal dominant inheritance patterns, impacts various bodily systems, featuring a clinical triad of progressive muscle weakness, myotonia, and early-onset cataracts. These disorders are classified into two primary types: DM type 1 (DM1) and DM type 2 (DM2). DelveInsight estimated that the total diagnosed prevalent patient population of myotonic dystrophy in the 7MM countries was close to 80K cases in 2021. As per the estimates, the US had the highest patient population of myotonic dystrophy in 2021. Although numerous pre-clinical advancements have been made, there is currently no targeted disease-modifying treatment available. The mainstay of management primarily involves monitoring for complications and adhering to standard care practices, such as utilizing assistive devices, administering hormone therapy, and providing pain relief. Several clinical studies have systematically assessed the efficacy of therapeutic agents in managing the condition. However, the scarcity of scientific evidence, coupled with the complex and diverse manifestations of the disease, poses significant challenges for healthcare providers when it comes to identifying and selecting appropriate medications. To tap the uncovered myotonic dystrophy market, few players have targeted the unmet need fortreatment. As per DelveInsight analysis, the myotonic dystrophy market is expected to grow from USD 80 million in 2021 at a significant CAGR by 2032. The current pipeline consists of both mid and late-stage candidates. The few promising therapies inthe mid and late-stage development are expected to experience significant reconstitution during the forecastperiod. Hence, more research and studies are needed to develop potential therapies that cater to the unmet need for curative and disease-modifying therapy in myotonic dystrophy patients. Myotonic Dystrophy Pipeline Therapies and Companies AMO-02 (tideglusib): AMO Pharma Limited Mexiletine: Lupin Ltd. Pitolisant: Harmony Biosciences, LLC AOC 1001: Avidity Biosciences, Inc. DYNE-101: Dyne Therapeutics To gain a deeper understanding of the myotonic dystrophy market, be sure to explore the Myotonic Dystrophya Market Outlook Facioscapulohumeral Muscular Dystrophy (FSHD) Market Facioscapulohumeral muscular dystrophy (FSHD), a genetically inherited disorder, progressively weakens muscles and significantly reduces the functional capacity of those affected. Recent years have witnessed substantial progress in our understanding of how epigenetic factors play a role in this condition. FSHD can be categorized into two subtypes based on the mechanisms of reduced methylation and failed epigenetic repression. Type 1 accounts for approximately 95% of FSHD cases and is characterized by the loss of D4Z4 repeats. The remaining 5% fall into Type 2, where methylation and epigenetic changes occur independently of deletions. Regardless of the subtype, FSHD leads to the same outcome. As per DelveInsight analysis, in the year 2021, the total prevalent cases of FSHD were 78K in the 7MM which are expected to grow during the study period, i.e., 2019–2032. As of now, there are no officially approved therapies for managing FSHD. However, individuals dealing with FSHD can find relief by utilizing orthotic devices designed to support their feet. These devices are particularly effective in addressing common FSHD symptoms like foot drop and shoulder weakness. Depending on the degree of foot drop, a custom-made ankle-foot orthosis (AFO) can provide significant benefits. In cases where knee extensor weakness may worsen gait with an AFO, alternatives like floor reaction ankle-foot orthosis (FRAFO) or advanced knee-ankle-foot orthosis (KAFO) may be considered. Regular exercise, especially hydrotherapy, plays a crucial role in preserving mobility and pain management. Additionally, weight management, potentially through dietary adjustments, is essential to reduce strain on already weakened muscles. It's important to note that there is currently no cure for FSHD, but there are treatments and devices available to alleviate many of its symptoms. Non-steroidal anti-inflammatory drugs (NSAIDs) are commonly prescribed to enhance comfort and mobility. According to DelveInsight, the FSHD market in 7MM is expected to witness a major change in the study period (2019–2032). As per estimates, in 2021, the total FSHD market size was USD 25.55 million which is expected to rise by 2032. This increase in market size is mainly due to extensive research and development activities of pharmaceutical companies, along with the expected launch of approved therapies. Facioscapulohumeral Muscular Dystrophy Pipeline Therapies and Companies Losmapimod: Fulcrum Therapeutics/GSK GYM329 (RO7204239/RG 6237): Roche Explore in-depth for a comprehensive understanding of the FSHD Clinical Trials Limb-girdle Muscular Dystrophy Market Limb-girdle muscular dystrophies (LGMD), also known as pelvofemoral muscular dystrophy or proximal muscular dystrophy, encompass a group of infrequent genetic disorders characterized by the progressive wasting and weakening of voluntary muscles located in the hip and shoulder regions. This muscle weakness and atrophy gradually advance and can eventually impact other muscle groups in the body. As per data from the National Organization for Rare Disorders, LGMD affects both males and females in roughly equal proportions. The exact prevalence of LGMD remains uncertain, but estimates suggest a range between one in 14,500 to one in 123,000 individuals. It's important to note that the age at which symptoms manifest can vary significantly, even within the same family. Additionally, the relative frequencies of the different LGMD types differ across populations, though it is worth mentioning that LGMD2G, 2H, and 2J are exceedingly rare on a global scale. Currently, there is no known cure for LGMD. However, there are supportive therapies designed to manage and alleviate its symptoms. The approach to LGMD management is individualized, focusing on addressing specific symptoms that each patient experiences. Treatment options may involve physical and occupational therapy to enhance muscle strength and prevent contractures, the utilization of assistive devices like canes, braces, walkers, or wheelchairs to aid mobility, corrective surgery for skeletal issues like scoliosis, and regular monitoring of cardiac and respiratory health for potential complications associated with certain LGMD subtypes. According to DelveInsight's' estimates, the limb girdle muscular dystrophy market in 7MM is expected to show a positive growth, during the forecast period (2023–2032), mainly attributed to anticipated launch of emerging therapy and the increasing cases of LGMD. Limb-girdle Muscular Dystrophy Pipeline Therapies and Companies SRP-6004: Sarepta Therapeutics, Inc. LION-101: Asklepios Biopharmaceutical, Inc. BBP-418 (ribitol): ML Bio Solutions, Inc. To access a complete analysis of the limb-girdle muscular dystrophy market, visit Limb-girdle Muscular Dystrophy Market Assessment Trending Genetic Disorders Reports Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DMD companies, including Pfizer, Santhera Pharmaceuticals, ReveraGen BioPharma, Taiho Pharmaceutical, FibroGen, Sarepta Therapeutics, Capricor Therapeutics, Daiichi Sankyo, Italfarmaco, Antisense Therapeutics, Solid Biosciences, among others. Alpha-1 Antitrypsin Deficiency Market Alpha-1 Antitrypsin Deficiency Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key A1ATD companies, including Arrow Head Pharmaceuticals, Mereo Biopharma, Kamada Ltd, Grifols, CSL Behring, Shire, LFB Biotechnologies, among others. Wilson Disease Market Wilson Disease Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wilson disease companies, including Orphalan, AstraZeneca, Vivet Therapeutics, Pfizer, Ultragenyx Pharmaceutical, among others. Cystic Fibrosis Market Cystic Fibrosis Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cystic fibrosis companies, including Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Santhera Pharmaceuticals, Laurent Pharmaceuticals Inc., Vertex Pharmaceuticals, among others. About DelveInsight DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

- Early assessment of increased glycosylated ⍺DG levels at 3 months predicted subsequent ambulatory improvements at 9 months, supporting the use of glycosylated ⍺DG levels as a potential surrogate endpoint in LGDM2I/R9 - Long-term data from the ongoing Phase 2 study of BBP-418 in patients with LGMD2I/R9 at Month 21 demonstrate a well-tolerated safety profile, and stabilization in 10 meter walk test (10MWT), 100-m timed test (100mTT), and the North Star Assessment (NSAD) scores - A sustained decrease of ≥80% in creatine kinase (CK), a marker of muscle breakdown, was observed with BBP-418 treatment at 21 months - The Phase 3 study continues to enroll in the U.S. and BridgeBio believes there is potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the U.S. Food and Drug Administration (FDA) on the use of glycosylated αDG levels as a surrogate endpoint PALO ALTO, Calif., Oct. 09, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, shared new long-term data from its Phase 2 trial in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) at the Annual Congress of World Muscle Society (WMS) in Charleston, South Carolina. The new long-term data remains consistent with earlier data from the Phase 2 study showing a well-tolerated safety profile and encouraging preliminary efficacy. Additionally, early changes in glycosylated ⍺DG levels at 3 months appear to be associated with ambulatory improvements at 9 months, providing support for the possible use of glycosylated ⍺DG levels as a surrogate endpoint in the ongoing Phase 3 study for accelerated approval. BridgeBio presented 21-month results from its ongoing Phase 2 trial, including: Large (≥80%), sustained reduction in creatine kinase observed over an extended (up to 21-months) treatment periodStabilization in NSAD scores and ambulatory measures observed over 21-months of BBP-418 treatmentBBP-418 continues to be well-tolerated with longer-term treatmentNo treatment-related SAEs or dose limiting toxicities observed with 21-months of BBP-418 dosing “There is a serious unmet need for those with LGMD2I/R9. The consistent improvement observed in biomarkers as well as stabilization in ambulatory measures are encouraging and help predict the clinical benefit for patients. The data for BBP-418 give me hope that similar results will be carried through in the currently enrolling Phase 3 trial, FORTIFY, for patients with LGMD2I/R9,” said Amy Harper, M.D., professor in the department of neurology at Virginia Commonwealth University (VCU) and primary investigator of the Phase 2 clinical trial in LGMD2I/R9. BridgeBio recently announced the first patient dosed in FORTIFY, its Phase 3 registrational study in patients with LGMD2I/R9 and is continuing to enroll throughout the U.S. with clinical trial sites planned for Europe and Australia. The Phase 3 FORTIFY registrational study is a randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of BBP-418. FORTIFY has a planned interim analysis at 12 months focused on assessing glycosylated αDG as a surrogate endpoint to potentially support an accelerated approval. The NSAD and secondary endpoints will be evaluated at 36 months, and results are expected to provide confirmatory clinical data. “To see that our therapy is potentially providing clinical improvements to people who otherwise could be non-ambulatory is deeply heartening for us,” said Douglas Sproule, M.D., M.Sc., chief medical officer of ML Bio Solutions, a BridgeBio affiliate that is focused on developing BBP-418 for LGMD2I/R9. “LGMD2I/R9 is a severely progressive disease and, inevitably, the weakening of the muscles causes many affected people to become fully dependent on a caregiver. Current treatments for LGMD2I/R9 are purely supportive. BBP-418 has the potential to be the first disease-modifying orally administered therapy available for patients.” More information about FORTIFY, the ongoing Phase 3 clinical trial of BBP-418 (NCT05775848) can be found here on the ClinicalTrials.gov website. About Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)LGMD2I/R9 is a monogenic autosomal recessive disease caused by partial loss of function mutations in the fukutin-related protein (FKRP) gene, and FKRP mutations impair glycosylation of αDG, a protein associated with stabilizing muscle cells. 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 L276I homozygous genotype typically develop disease manifestations during late childhood with progression to loss of independent ambulation (25%), assisted ventilation (10%), and cardiomyopathy (30%) in adulthood. Cardiomyopathy is progressive, with an annual loss of 0.4% of left ventricular ejection fraction (LVEF). Patients with other (non-L276I homozygous) 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 (60%), 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 development programs ranges from early science to advanced clinical trials. 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 BridgeBio makes 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. BridgeBio intends 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. These forward-looking statements, including statements relating to the clinical and therapeutic potential of BridgeBio’s programs and product candidates, including BBP-418 for the treatment of LGMD2I, the potential benefits of BBP-418, including that the early assessment of increased glycosylated ⍺DG levels at 3 months predicted subsequent ambulatory improvements at 9 months, supporting the use of glycosylated ⍺DG levels as a potential surrogate endpoint in LGDM2I/R9 and that BBP-418 continues to be well-tolerated with longer-term treatment , the progress of BridgeBio’s ongoing and planned clinical trials of BBP-418, including the continued enrollment of the Phase 3 study in the U.S. with clinical trial sites planned for Europe, UK, and Australia, the potential and the opportunity to pursue Accelerated Approval Pathway for BBP-418 in LGMD2I/R9 in the U.S. based on recent interactions with the FDA on the use of glycosylated αDG levels as a surrogate endpoint, the statement regarding the potential benefit of FORTIFY trial for patients with LGMD2I/R9 and of BBP-418 in the quotes of Dr. Harper and Dr. Sproule, the expectation that the results of the evaluation of secondary endpoints at 36 months will provide confirmatory clinical data, and the timing and success of BridgeBio’s clinical trials and development pipeline, among others, reflect BridgeBio’s current views about its plans, intentions, expectations, strategies and prospects, which are based on the information currently available to BridgeBio and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, BridgeBio 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’s ability to continue and complete its ongoing and planned clinical trials of BBP-418 for the treatment of LGMD2I, initial and ongoing data from clinical trials not being indicative of final data, the design and success of BridgeBio’s ongoing and planned clinical trials, the FDA or other regulatory agencies not agreeing with BridgeBio’s regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, as well as those risks set forth in the Risk Factors section of BridgeBio’s Annual Report on Form 10-K for the year ended December 31, 2022, and BridgeBio’s other SEC filings. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. 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. BridgeBio Media Contact:Vikram Balicontact@bridgebio.com (650)-789-8220

- BridgeBio met with the U.S. Food and Drug Administration (FDA) to discuss the use of glycosylated αDG levels as a surrogate endpoint; based on this meeting, the Company believes there is potential to pursue Accelerated Approval in the U.S. for BBP-418 - BridgeBio has dosed the first participant in FORTIFY, its global Phase 3 study of BBP-418 in patients with LGMD2I/R9 - FORTIFY includes an interim analysis at 12 months focused on change in glycosylated αDG levels; topline data from this analysis is expected in late 2024/early 2025 - Deficiency of glycosylated αDG is the causal molecular driver of LGMD2I/R9; in the ongoing Phase 2 study, patients treated with BBP-418 had a rapid and sustained increase of glycosylated αDG levels, concurrent with sustained decreases in creatine kinase and improvements from baseline in ambulatory and clinical function measures - If successful, BBP-418 has the potential to address serious unmet need for patients with LGMD2I/R9, a disease for which there are no approved therapies PALO ALTO, Calif., July 31, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that, based on discussions with the U.S. Food and Drug Administration (FDA), there is a potential path to pursue accelerated approval in the U.S. for BBP-418, an investigational oral substrate supplementation therapy, in patients with limb-girdle muscular dystrophy type 2I (LGMD2I/R9). BridgeBio also announced that the first patient with LGMD2I/R9 has been dosed in its Phase 3 FORTIFY clinical trial of BBP-418. To date, the Company has activated over half the planned U.S. clinical sites and is in the process of opening sites in Europe and Australia to support global registration. The Phase 3 FORTIFY registrational study is a randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of BBP-418. FORTIFY has a planned interim analysis at 12 months focused on assessing glycosylated αDG as a surrogate endpoint to potentially support an accelerated approval. The North Star Assessment for Dysferlinopathy (NSAD) and secondary endpoints will be evaluated at 36 months, and results are expected to provide confirmatory clinical data. A novel, validated bioassay was developed by BridgeBio to directly measure glycosylated ⍺DG levels, which are central to LGMD2I/R9 disease, and may enable monitoring of responses to disease-modifying therapies in LGMD2I/R9 patients. BridgeBio is committed to collaborating with the FDA to address the challenges associated with LGMD2I/R9 drug development, including the potential use of a surrogate endpoint to support an accelerated approval. “As a physician who regularly treats people with LGMD2I/R9, I found the results observed in the Phase 2 trial of BBP-418 very encouraging, and I am pleased we’ve been able to dose the first patient in the Phase 3 trial. I am hopeful that we will see impact for these patients similar to the Phase 2, because patients with LGMD2I/R9 are significantly affected by progressive, debilitating muscle weakness that impinges heavily upon quality of life and eventually leads to functional dependence,” said Tahseen Mozaffar, M.D., professor of neurology and pathology & laboratory medicine at University of California, Irvine. BBP-418 is designed to supply supraphysiological levels of an endogenous substrate upstream of the mutant FKRP enzyme to help drive residual activity of the enzyme to glycosylate αDG, with the goal of stabilizing muscle cells during contraction, and potentially halting further muscle damage. Data from an ongoing open-label Phase 2 study is consistent and encouraging, suggesting that BBP-418 may be well-tolerated and have a positive impact on key endpoints. “There are no FDA approved specific treatments for LGMD2I/R9 and current treatments are only supportive. The LGMD community is hopeful that we will soon transition from supportive care to disease modifying treatments for LGMDs. Living with a progressive disease like LGMD2I/R9 means that each of us is, day by day, losing the ability to be independent and do the things that we love. Many of us live in fear of the future. The launch of the Phase 3 study provides our community with a beacon of needed hope,” said Kathryn Bryant Knudson, founder and president of The Speak Foundation. Updated results on the Company’s novel bioassay and for the Phase 2 clinical trial will be presented at the Annual Congress of the World Muscle Society (WMS), taking place in Charleston, South Carolina on October 3 – 7, 2023. If approved, BridgeBio believes BBP-418 could be the first approved orally administered therapy for the treatment of patients with LGMD2I/R9. “Currently, our Phase 2 data suggest that glycosylated αDG levels are improved and sustained over time following treatment with BBP-418. In addition to the effect on αDG, we have also observed consistent improvement in clinical endpoints such as increased NSAD score, increased 10-meter walk test velocity and reduced time to complete 100-meter timed test compared to baseline,” said Douglas Sproule, M.D., M.Sc., chief medical officer of ML Bio Solutions, a BridgeBio affiliate that is focused on developing BBP-418 for LGMD2I/R9. “The meeting with the U.S. FDA was both positive and productive in discussing the potential use of glycosylated αDG levels as a surrogate endpoint in our clinical trial. I am encouraged by the collaboration from the FDA, including the clear feedback received regarding the information needed to support the use of αDG as a reasonably likely surrogate endpoint. We look forward to working with the Agency to bring this treatment to patients as quickly as possible.” More information about the ongoing Phase 3 clinical trial of BBP-418 (NCT05775848) can be found here on the ClinicalTrials.gov website. About Limb-girdle Muscular Dystrophy Type 2I (LGMD2I/R9)LGMD2I/R9 is a monogenic autosomal recessive disease caused by partial loss of function mutations in the fukutin-related protein (FKRP) gene, and FKRP mutations impair glycosylation of αDG, a protein associated with stabilizing muscle cells. 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 (10%), and cardiomyopathy (30%) 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 (60%), 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 development programs ranges from early science to advanced clinical trials. 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 StatementsThis press release contains forward-looking statements. Statements BridgeBio makes 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. BridgeBio intends 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. These forward-looking statements, including statements relating to the clinical and therapeutic and market potential of BridgeBio’s programs and product candidates, including BBP-418 for the treatment of LGMD2I/R9, the drug design and the potential benefits of BBP-418, including its potential to address serious unmet need for patients with LGMD2I/R9, the potential and the opportunity to pursue Accelerated Approval Pathway for BBP-418 in LGMD2I/R9 in the U.S., the expectation of BBP-418 to be well-tolerated and have a positive impact on key endpoints, the expected timeline of announcing the topline data from the interim analysis of FORTIFY at 12 months focused on assessing glycosylated αDG as a surrogate endpoint to support an accelerated approval, and the statements regarding the potential benefit of our clinical trial or of our product candidate in the quotes of Ms. Knudson and Dr. Sproule; the progress, timeline and success of BridgeBio’s ongoing and planned clinical trials of BBP-418, including the expectation of opening clinical trial sites in Europe and Australia to support global registration, , the timeline of evaluation of the NSAD and secondary endpoints at 36 months, and the expectation of providing confirmatory clinical data from such results, the plans of engaging with regulatory authorities, including the collaboration and interaction with the FDA to address the challenges associated with LGMD2I/R9 drug development, the typical clinical manifestations of LGMD2I/R9 and progression to loss of independent ambulation, assisted ventilation and cardiomyopathy in adulthood, the impact on patients with heterozygous genotypes, the potential benefits of BridgeBio’s novel bioassay, including the ability to measure glycosolated ⍺DG levels from muscle biopsy samples, and the potential for BBP-418 to be the first approved orally administered therapy for the treatment of LGMD2I/R9,among others, reflect BridgeBio’s current views about its plans, intentions, expectations, strategies and prospects, which are based on the information currently available to BridgeBio and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, BridgeBio 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’s ability to continue and complete its ongoing and planned clinical trials of BBP-418 for the treatment of LGMD2I/R9, initial and ongoing data from clinical trials not being indicative of final data, the design and success of BridgeBio’s ongoing and planned clinical trials, the U.S. Food and Drug Administration (FDA) or other regulatory agencies not agreeing with BridgeBio’s regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, as well as those risks set forth in the Risk Factors section of BridgeBio’s Annual Report on Form 10-K for the year ended December 31, 2022, and BridgeBio’s other SEC filings. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. 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. BridgeBio Contact:Vikram Balicontact@bridgebio.com(650)-789-8220