Ribitol

- Submitted New Drug Application (NDA) to US Food and Drug Administration (FDA) for acoramidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) based on positive results of Phase 3 ATTRibute-CM trial, which were published in the New England Journal of Medicine; NDA has been accepted for review with a PDUFA date of November 29, 2024; Marketing Authorization Application (MAA) for acoramidis has also been accepted by the European Medicines Agency (EMA) - Presented additional data from ATTRibute-CM at the American Heart Association Scientific Sessions, demonstrating separation at Month 3 of the placebo and acoramidis time-to-first-event Kaplan-Meier curves for a composite of all-cause mortality (ACM) and cardiovascular-related hospitalization (CVH); separation was sustained through Month 30 and represents the most rapid clinical benefit on the composite endpoint of ACM and CVH in ATTR-CM patients to the Company’s knowledge - Shared positive results of single-arm Phase 3 study of acoramidis in Japanese ATTR-CM patients, including no mortality reported over the 30 month acoramidis treatment period - PROPEL 3, the Company's Phase 3 study of infigratinib for achondroplasia continues to enroll with full enrollment expected in 2024; the Company has also announced a partnership granting Kyowa Kirin exclusive license on infigratinib for skeletal dysplasias in Japan in exchange for an upfront payment of $100 million, royalties up to the high twenties percent, and additional milestone-based payments - FORTIFY, the Company's Phase 3 study BBP-418 for limb-girdle muscular dystrophy type 2I (LMGD2I), continues to enroll, with full enrollment of interim analysis population expected in 2024 - CALIBRATE, the Company's Phase 3 study of encaleret for autosomal dominant hypocalcemia type 1 (ADH1) continues to enroll, with full enrollment expected in 2024 and topline data expected in 2025 - Secured up to $1.25 billion of capital from Blue Owl and CPP investments, including $500 million in cash in exchange for a 5% royalty on future global net sales of acoramidis and a $450 million credit facility from Blue Owl that refinanced existing senior secured credit, extending maturity from 2026 to 2029 subject to certain conditions - Ended the quarter with $393 million in cash, cash equivalents, and short-term restricted cash, and $59 million of investments in equity securities PALO ALTO, Calif., Feb. 22, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided an update on the Company’s operations. “Our focus this year is executing on the launch of acoramidis for patients with ATTR cardiomyopathy,” said Neil Kumar, Ph.D., founder and CEO of BridgeBio. “At the same time, we are also focused on fully enrolling three ongoing Phase 3 clinical trials by the end of 2024. Finally, we hope that reading out potentially exciting data from our Phase 1/2 trial in congenital adrenal hyperplasia later this year will let us take the next step in serving that patient community.” BridgeBio’s key programs: Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): The Company filed an NDA for acoramidis for the treatment of ATTR-CM with the US FDA; the NDA was accepted for review with a PDUFA date of November 29, 2024. The Company has also filed a Marketing Authorization Application for acoramidis with the EMA, which has been accepted for review.The regulatory filings were based on data from the Phase 3 ATTRibute-CM study, which met its primary endpoint (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001). Additional results from ATTRibute-CM include: An 81% survival rate on acoramidis, which approaches the survival rate in the age-matched U.S. database (~85%), and a 0.29 mean annual CVH rate on acoramidis, which approaches the annual hospitalization rate observed in the broader U.S. Medicare population (~0.26);Improvements from baseline observed for a large proportion of participants treated with acoramidis on laboratory and functional measures including n-terminal prohormone of brain natriuiretic peptide (NT-proBNP) and 6-minute walk distance;Rapid clinical benefit on the composite endpoint of ACM and CVH in participants treated with acoramidis, demonstrated by placebo and acoramidis time-to-first event Kaplan-Meier curves for a composite of ACM and CVH that separated at Month 3 and continued to diverge steadily through Month 30 as presented at the American Heart Association Scientific Sessions in November 2023; andAcoramidis was well-tolerated with no safety signals of potential clinical concern identified. The Company also shared positive results of an open-label, single-arm Phase 3 study conducted in Japan by licensing partner Alexion, AstraZeneca Rare Disease, including that no mortality was reported over the 30 month acoramidis treatment period.Additional detailed results of ATTRibute-CM are planned for presentation at 2024 medical meetings. Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia: In December 2023, the Company announced the dosing of the first child in PROPEL 3, its global Phase 3 registrational study of infigratinib in achondroplasia.In February 2024, the Company announced a partnership with Kyowa Kirin wherein the Company grants Kyowa Kirin an exclusive license to develop and commercialize infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan; in exchange, the Company will receive an upfront payment of $100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.The Company is committed to exploring the potential of infigratinib on the wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias, and anticipates initiating its clinical program for hypochondroplasia in 2024. BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9): FORTIFY, the global Phase 3 registrational trial of BBP-418, continues to enroll in the U.S. with clinical trial sites planned for Europe and Australia. Full enrollment of the interim analysis population is expected in 2024. The Company believes there is potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the FDA on the use of glycosylated αDG levels as a surrogate endpoint. Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1): CALIBRATE, the Phase 3 clinical trial of encaleret, continues to enroll; the Company anticipates sharing topline data from CALIBRATE in 2025. Recent Corporate Updates: ·Secured up to $1.25 billion of capital from Blue Owl and CPP Investments: The raise includes $500 million in cash from Blue Owl and CPP Investments available upon FDA approval of acoramidis in exchange for a 5% royalty on future global net sales of acoramidis, as well as a $450 million credit facility from Blue Owl that refinanced existing senior secured credit, extending maturity from 2026 to 2029 subject to certain conditions. Fourth Quarter and Full Year 2023 Financial Results: Cash, Cash Equivalents, Marketable Securities and Short-term Restricted Cash Cash, cash equivalents and short-term restricted cash, totaled $392.6 million as of December 31, 2023, compared to cash, cash equivalents, marketable securities and short-term restricted cash of $466.2 million as of December 31, 2022. The net decrease of $73.6 million in cash, cash equivalents, marketable securities and short-term restricted cash was primarily attributable to net cash used in operating activities of $527.7 million and $6.9 million in repurchase of shares to satisfy tax withholdings, primarily offset by net proceeds received of $449.8 million from various equity financings, $6.0 million from stock option exercises, and $3.4 million from common stock issuances under our employee stock purchase plan during the year ended December 31, 2023. Revenue Revenue for the three months and year ended December 31, 2023 were $1.7 million and $9.3 million, respectively, as compared to $1.9 million and $77.6 million for the same periods in the prior year, respectively. The net decreases of $0.2 million and $68.3 million for the three months and year ended December 31, 2023, respectively, compared to the same periods in the prior year, were primarily due to license revenue recognized in 2022 upon the transfer of the license in accordance with the Navire-BMS License Agreement which was entered into in May 2022. Operating Costs and Expenses Operating costs and expenses for the three months and year ended December 31, 2023 were $179.2 million and $616.7 million, respectively, compared to $131.1 million and $589.9 million, for the same periods in the prior year, respectively. The overall increase of $48.1 million in operating costs and expenses for the three months ended December 31, 2023, compared to the same period in the prior year, was primarily due to an increase of $39.3 million in research and development and other expenses (R&D) to advance the Company's pipeline of development programs, an increase of $15.7 million in selling, general and administrative (SG&A) expenses to support commercialization readiness efforts, offset by a decrease of $6.9 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year. The overall increase of $26.8 million in operating costs and expenses for the year ended December 31, 2023 , compared to the same period in the prior year, was primarily due to an increase of $55.2 million in R&D expenses to advance the Company's pipeline of development programs, an increase of $7.4 million in SG&A expenses to support commercialization readiness efforts, offset by a decrease of $35.8 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year. Restructuring, impairment and related charges for the three months and year ended December 31, 2023, amounted to $0.8 million and $7.9 million, respectively. These charges primarily consisted of winding down, exit costs, and severance and employee-related costs. Restructuring, impairment and related charges for the same periods in the prior year were $7.7 million and $43.8 million, respectively. These charges primarily consisted of impairments and write-offs of long-lived assets, severance and employee-related costs, and exit and other related costs. Stock-based compensation expenses included in operating costs and expenses for the three months ended December 31, 2023 were $37.1 million, of which $22.5 million is included in R&D expenses, and $14.6 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $22.6 million, of which $8.9 million is included in R&D expenses, and $13.6 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the year ended December 31, 2023 were $115.0 million, of which $61.6 million is included in R&D expenses, and $53.4 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $93.8 million, of which $38.0 million is included in R&D expenses, $54.7 million is included in SG&A expenses, and $1.2 million is included in restructuring, impairment and related charges. “Coming off of our recent royalty financing, we find ourselves well capitalized to launch acoramidis this year alongside strong new partners who share our confidence in acoramidis’ potential in the ATTR-CM market,” said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. “We are excited for this launch, as well as for the continued advancement of our late stage pipeline, which we hope will allow us to serve patients with genetic diseases both directly with the advancement of those medicines towards the market as well as by diversifying our top line revenue and enabling reinvestment into the R&D and business development opportunities that will allow us to be sustainable in the long term.” BRIDGEBIO PHARMA, INC.Condensed Consolidated Statements of Operations(in thousands, except shares and per share amounts) Three Months Ended December 31, Year Ended December 31, 2023 2022 2023 2022 (Unaudited) (Unaudited) (1) Revenue $1,745 $1,870 $9,303 $77,648 Operating costs and expenses: Research, development and other expenses 130,824 91,549 458,157 402,896 Selling, general and administrative 47,583 31,862 150,590 143,189 Restructuring, impairment and related charges 754 7,691 7,926 43,765 Total operating costs and expenses 179,161 131,102 616,673 589,850 Loss from operations (177,416) (129,232) (607,370) (512,202)Other income (expense), net: Interest income 5,578 4,092 18,038 7,542 Interest expense (20,268) (19,990) (81,289) (80,438)Gain from sale of priority review voucher, net — — — 107,946 Other income (expense), net 21,778 4,560 17,370 (7,500)Total other income (expense), net 7,088 (11,338) (45,881) 27,550 Net loss (170,328) (140,570) (653,251) (484,652)Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 2,180 2,979 10,049 3,469 Net loss attributable to common stockholders of BridgeBio $(168,148) $(137,591) $(643,202) $(481,183)Net loss per share, basic and diluted $(0.96) $(0.92) $(3.95) $(3.26)Weighted-average shares used in computing net loss per share, basic and diluted 174,462,332 149,344,380 162,791,511 147,473,076 Three Months Ended December 31, Year Ended December 31, Stock-based Compensation 2023 2022 2023 2022 (Unaudited) (Unaudited) (1) Research, development and others $22,495 $8,941 $61,647 $37,987 Selling, general and administrative 14,638 13,643 53,369 54,669 Restructuring, impairment and related charges — — — 1,172 Total stock-based compensation $37,133 $22,584 $115,016 $93,828 (1)The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date. BRIDGEBIO PHARMA, INC.Condensed Consolidated Balance Sheets(In thousands) December 31, December 31, 2023 2022 (Unaudited) (1) Assets Cash, cash equivalents and marketable securities $375,935 $428,269 Investment in equity securities 58,949 43,653 Receivable from licensing and collaboration agreements 1,751 17,079 Short-term restricted cash 16,653 37,930 Prepaid expenses and other current assets 24,305 21,922 Property and equipment, net 11,816 14,569 Operating lease right-of-use assets 8,027 10,678 Intangible assets, net 26,319 28,712 Other assets 22,625 20,224 Total assets $546,380 $623,036 Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit Accounts payable $10,655 $11,558 Accrued and other liabilities 129,061 106,195 Operating lease liabilities 13,109 15,949 2029 Notes, net 736,905 734,988 2027 Notes, net 543,379 541,634 Term loan, net 446,445 430,993 Other long-term liabilities 9,361 26,643 Redeemable convertible noncontrolling interests 478 (1,589)Total BridgeBio stockholders' deficit (1,354,257) (1,254,617)Noncontrolling interests 11,244 11,282 Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit $546,380 $623,036 (1)The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date. BRIDGEBIO PHARMA, INC.Condensed Consolidated Statements of Cash Flows(In thousands) Year Ended December 31, 2023 2022 (Unaudited) (1 ) Operating activities: Net loss $(653,251) $(484,652)Adjustments to reconcile net loss to net cash used in operating activities: Stock-based compensation 108,710 91,559 Depreciation and amortization 6,494 6,771 Noncash lease expense 4,032 5,172 Accrual of payment-in-kind interest on term loan 10,207 13,562 Loss on deconsolidation of PellePharm 1,241 — (Gain) loss from investment in equity securities, net (18,314) 8,222 Fair value of shares issued under a license agreement — 4,567 Accretion of debt 8,907 8,570 Fair value adjustment of warrants (984) 1,571 Loss on sale of certain assets — 6,261 Impairment of long-lived assets — 12,720 Gain from sale of priority review voucher, excluding transaction costs — (110,000)Gain from recognition of receivable from licensing and collaboration agreement — (12,500)Other noncash adjustments 181 604 Changes in operating assets and liabilities: Receivable from licensing and collaboration agreements 15,328 15,169 Prepaid expenses and other current assets (2,702) 7,671 Other assets (1,546) 10,971 Accounts payable 2,780 (349)Accrued compensation and benefits 7,802 (2,362)Accrued research and development liabilities (9,855) (4,309)Operating lease liabilities (4,829) (6,245)Deferred revenue (5,438) 15,262 Accrued professional and other liabilities 3,517 (7,729)Net cash used in operating activities (527,720) (419,494)Investing activities: Purchases of marketable securities (29,726) (137,493)Maturities of marketable securities 82,550 479,688 Purchases of investment in equity securities (107,538) (55,562)Sales of investment in equity securities 110,556 52,835 Decrease in cash and cash equivalents resulting from deconsolidation of PellePharm (503) — Payment for intangible asset — (1,500)Proceeds from sale of priority review voucher — 110,000 Proceeds from sale of certain assets — 10,000 Purchases of property and equipment (1,306) (4,821)Net cash provided by investing activities 54,033 453,147 Financing activities: Proceeds from issuance of common stock through Private Placement offering, net 240,796 — Proceeds from issuance of common stock through Follow-on offering, net 144,049 — Proceeds from issuance of common stock through ATM offering, net 64,965 4,852 Transactions with noncontrolling interests (801) — Repayment of term loan — (20,486)Proceeds from BridgeBio common stock issuances under ESPP 3,398 2,558 Repurchase of RSU shares to satisfy tax withholding (6,880) (1,561)Proceeds from stock option exercises, net of repurchases 6,008 666 Other financing activities — 837 Net cash provided by (used in) financing activities 451,535 (13,134)Net increase (decrease) in cash, cash equivalents and restricted cash (22,152) 20,519 Cash, cash equivalents and restricted cash at beginning of period 416,884 396,365 Cash, cash equivalents and restricted cash at end of period $394,732 $416,884 Year Ended December 31, 2023 2022 (Unaudited) (1) Supplemental Disclosure of Cash Flow Information: Cash paid for interest $61,108 $54,443 Supplemental Disclosures of Noncash Investing and Financing Information: Unpaid property and equipment $100 $47 Recognized intangible asset recorded in “Other accrued and other long-term liabilities” $— $11,000 Transfers (to) from noncontrolling interests $(10,534) $(3,512)Payment-in-kind interest added to principal of term loan $— $1,763 Reconciliation of Cash, Cash Equivalents and Restricted Cash: Cash and cash equivalents $375,935 $376,689 Short-term restricted cash 16,653 37,930 Restricted cash — Included in “Other assets” 2,144 2,265 Total cash, cash equivalents and restricted cash at end of periods $394,732 $416,884 (1)The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date. 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 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,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “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. These forward-looking statements, including statements relating to the clinical and therapeutic, market potential of our programs and product candidates, including the statements in Dr. Kumar’s and Dr. Stephenson's quotes regarding the potential commercial launch of acoramidis (if approved), continued advancement in our pipeline, including enrollments in clinical trials and anticipated readout, and other benefits resulting from recent financing; the statements related to the FDA's planned actions regarding our NDA for acoramidis for the treatment of ATTR-CM; the potential outcomes of regulatory reviews by the FDA and the EMA; the timing and success of our clinical development programs, including the progress of our clinical development program for acoramidis for patients with ATTR-CM, and our plan for, and the expected timing of, presenting additional detailed results of ATTRibute-CM study at medical meetings; the potential success of our partnership granting Kyowa Kirin an exclusive license on infigratinib for skeletal dysplasias in Japan and the potential payments we may receive under the license; the continuation of PROPEL 3, our Phase 3 study of infigratinib for achondroplasia and the expected timing for full enrollment in the study; our commitment to exploring the potential of infigratinib and the expectation and timing of the initiation of our clinical program for hypochondroplasia; the continuation and progress of FORTIFY, the Phase 3 trial of BBP-418 for LGMD2I, including the ongoing enrollment in the United States, the expectation to enroll in clinical trial sites planned in Europe and Australia, the expectation and timing of full enrollment of the interim analysis population, and the potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the FDA; the continued enrollment in CALIBRATE, the Phase 3 clinical trial of encaleret, and the expectation and timing of full enrollment and sharing topline data from CALIBRATE; the Company’s financial performance, capitalization status, strategy, business plans and goals reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations and strategies 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, initial and ongoing data from our preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations our product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for our product candidates, the FDA or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of our collaborations, the Company’s ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in our share price, uncertainty regarding any impacts due to global health emergencies such as COVID-19, including delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and rising interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of our most recent Annual Report on Form 10-K and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate 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 our 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 Bali contact@bridgebio.com (650)-789-8220

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. 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