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Business Upturn
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Duchenne Muscular Dystrophy Market to Witness Significant Expansion During the Forecast Period (2025–2034) Amidst Advances in Gene Therapy and Novel Drug Approvals
New York, USA, July 28, 2025 (GLOBE NEWSWIRE) — Duchenne Muscular Dystrophy Market to Witness Significant Expansion During the Forecast Period (2025–2034) Amidst Advances in Gene Therapy and Novel Drug Approvals | DelveInsight The Duchenne muscular dystrophy market is witnessing steady growth driven by advancements in gene therapies, exon-skipping drugs, and corticosteroid alternatives. Increasing diagnosis rates, rising awareness, and supportive regulatory pathways have accelerated therapy approvals. Additionally, key players continue to expand their pipelines, with several novel therapies in late-stage clinical trials. DelveInsight's Duchenne Muscular Dystrophy Market Insights report includes a comprehensive understanding of current treatment practices, emerging Duchenne muscular dystrophy drugs, market share of individual therapies, and current and forecasted Duchenne muscular dystrophy market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Key Takeaways from the Duchenne Muscular Dystrophy Market Report According to DelveInsight's analysis, the total Duchenne muscular dystrophy market size is expected to grow positively by 2034. The United States accounts for the largest market size of Duchenne Muscular Dystrophy, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan. In 2024, the United States had the highest prevalence of DMD among the 7MM countries, with approximately 17,000 cases. This number is expected to grow at a steady CAGR. cases. This number is expected to grow at a steady CAGR. Prominent companies, including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, and others, are actively working on innovative Duchenne muscular dystrophy drugs. and others, are actively working on innovative Duchenne muscular dystrophy drugs. Some of the key Duchenne muscular dystrophy therapies in the pipeline include NS-089/NCNP-02 (brogidirsen), TAS-205 (pizuglanstat), RGX-202, and others. These novel Duchenne muscular dystrophy therapies are anticipated to enter the Duchenne muscular dystrophy market in the forecast period and are expected to change the market. Discover which Duchenne muscular dystrophy medications are expected to grab the market share @ Duchenne Muscular Dystrophy Market Report Duchenne Muscular Dystrophy Market Dynamics The Duchenne muscular dystrophy market dynamics are anticipated to change in the coming years. Glucocorticosteroids remain the mainstay treatment option for Duchenne muscular dystrophy, despite their associated adverse side effects, while newer corticosteroids like EMFLAZA face cost-effectiveness challenges in the United States; moreover, there is currently no cure or disease-reversing therapy available for non-ambulant DMD patients. However, ongoing research in gene therapy and genetic technologies offers promising opportunities for developing targeted and personalized treatments, alongside a wider commercial potential for therapies such as Capricor's CAP-1002 and Antisense's ATL1102, which are specifically aimed at improving upper limb function in DMD patients and addressing a broader patient population. Furthermore, many potential therapies are being investigated for the treatment of Duchenne muscular dystrophy, and it is safe to predict that the treatment space will significantly impact the Duchenne muscular dystrophy market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate is expected to drive the growth of the Duchenne muscular dystrophy market in the 7MM. However, several factors may impede the growth of the Duchenne muscular dystrophy market. The diagnosis of Duchenne muscular dystrophy is often complicated due to inadequate patient history, the use of inappropriate medical tests, and the presence of multiple comorbidities, frequently leading to delayed or incorrect diagnosis; additionally, the high cost of current and emerging therapies may limit patient access globally, while the rarity of DMD and its occurrence in the paediatric population contribute to a low availability of participants for clinical trials, posing further challenges to therapeutic development. Moreover, Duchenne muscular dystrophy treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Duchenne muscular dystrophy market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Duchenne muscular dystrophy market growth. Duchenne Muscular Dystrophy Treatment Market The primary treatment approaches for Duchenne muscular dystrophy focus on restoring dystrophin production through gene replacement or mutation-targeted genetic therapies, stabilizing muscle membranes, enhancing compensatory protein expression, reducing inflammation, and promoting muscle regeneration. In the United States, several therapies have received approval for DMD management, including AGAMREE (vamorolone), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), VILTEPSO (viltolarsen), and ELEVIDYS (delandistrogene moxeparvovec), among others. DUVYZAT (givinostat), a histone deacetylase (HDAC) inhibitor, has been authorized for use in DMD patients aged six and older. Notably, it is the first FDA-approved non-steroidal drug applicable to all genetic forms of DMD. Givinostat works by inhibiting HDAC enzymes, which regulate gene expression and contribute to muscle inflammation and damage. By blocking these enzymes, givinostat seeks to reduce inflammation, support muscle repair, and slow muscle degeneration in DMD patients. AMONDYS 45, or casimersen, developed by Sarepta Therapeutics, is an antisense oligonucleotide designed for patients with a specific DMD gene mutation suitable for exon 45 skipping. Casimersen employs exon-skipping technology, targeting the dystrophin gene pre-mRNA to exclude exon 45 during processing. This allows the creation of a shortened but functional dystrophin protein, which helps maintain muscle fiber structure and function, aiming to restore the disrupted reading frame and partially compensate for the dystrophin deficiency. AGAMREE is a prescription therapy for children aged two and older with DMD. It is a novel anti-inflammatory drug that selectively binds the glucocorticoid receptor but modulates downstream effects, bypassing the 11β-HSD enzyme pathway associated with steroid-induced toxicity. This distinct mechanism potentially provides the anti-inflammatory benefits of corticosteroids while minimizing their harmful side effects, making AGAMREE a safer treatment option for pediatric and adolescent patients. AGAMREE was granted Orphan Drug designation in August 2014. Learn more about the Duchenne muscular dystrophy treatment options @ Duchenne Muscular Dystrophy Treatment Guidelines Duchenne Muscular Dystrophy Emerging Drugs and Companies The Expected launch of potential therapies may increase the market size in the coming years, assisted by an increase in the diagnosed prevalent population of DMD. Owing to the positive outcomes of several products during the developmental stage by key players such as NS Pharma/Nippon Shinyaku (NS-089/NCNP-02), Taiho Pharma (TAS-205), REGENXBIO (RGX-202), and others. NS-089/NCNP-02 is an antisense oligonucleotide jointly developed by Nippon Shinyaku and the National Center of Neurology and Psychiatry (NCNP). It is being explored as a treatment option for Duchenne muscular dystrophy (DMD) patients whose dystrophin gene mutations can be addressed through exon 44 skipping. In January 2025, NS Pharma announced that NCNP published the outcomes of a first-in-human, investigator-led study of NS-089/NCNP-02 (brogidirsen) in Cell Reports Medicine. This therapy is currently being investigated in a global Phase II clinical trial (NCT05996003) for DMD. TAS-205 (pizuglanstat), discovered by Taiho Pharmaceutical, is a selective inhibitor of hematopoietic prostaglandin D synthase (HPGDS). It is under development for DMD treatment and works independently of the type of dystrophin gene mutation. By inhibiting HPGDS, which contributes to inflammation in muscle tissue, TAS-205 aims to slow the progression of motor decline in DMD patients. This drug is currently undergoing Phase III trials. RGX-202 is a gene therapy designed to deliver a transgene encoding a novel microdystrophin that retains critical functional components of the C-terminal domain present in natural dystrophin. RGX-202 is in a Phase II/III pivotal trial. The therapy has been granted Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation by the FDA. In November 2024, REGENXBIO announced that the AFFINITY DUCHENNE trial of RGX-202 has progressed to its pivotal stage, with the first patient receiving the treatment. The company shared positive safety and efficacy results from its earlier Phase I/II study, which included functional outcomes. REGENXBIO has also reached an agreement with the FDA on the pivotal study design and the potential for accelerated approval, with a Biologics License Application (BLA) filing targeted for 2026. The pivotal trial is currently enrolling ambulatory patients aged 1 year and older. The anticipated launch of these emerging Duchenne muscular dystrophy therapies are poised to transform the Duchenne muscular dystrophy market landscape in the coming years. As these cutting-edge Duchenne muscular dystrophy therapies continue to mature and gain regulatory approval, they are expected to reshape the Duchenne muscular dystrophy market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about new treatment for Duchenne muscular dystrophy, visit @ Duchenne Muscular Dystrophy Management Recent Developments in the Duchenne Muscular Dystrophy Market In May 2025, Capricor Therapeutics completed a mid-cycle review meeting with the FDA for its Biologics License Application (BLA) for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA confirmed no significant deficiencies in the review and affirmed that the application is on track for a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025. An advisory committee meeting is also planned, but no date has been set yet. completed a mid-cycle review meeting with the FDA for its Biologics License Application (BLA) for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA confirmed no significant deficiencies in the review and affirmed that the application is on track for a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025. An advisory committee meeting is also planned, but no date has been set yet. In March 2025, the FDA accepted Capricor Therapeutics' application for approval of deramiocel, a cell therapy developed to treat heart muscle disease in individuals with Duchenne muscular dystrophy (DMD), and granted it priority review to accelerate the agency's decision. the FDA accepted Capricor Therapeutics' application for approval of deramiocel, a cell therapy developed to treat heart muscle disease in individuals with Duchenne muscular dystrophy (DMD), and granted it priority review to accelerate the agency's decision. In January 2025, Capricor Therapeutics announced the submission of its Biologics License Application (BLA) to the FDA for full approval of deramiocel, an investigational cell therapy for treating Duchenne muscular dystrophy cardiomyopathy. announced the submission of its Biologics License Application (BLA) to the FDA for full approval of deramiocel, an investigational cell therapy for treating Duchenne muscular dystrophy cardiomyopathy. In November 2024, Regenxbio is advancing its gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) into pivotal development, with plans for a BLA submission by 2026. The company has secured FDA alignment on an accelerated approval pathway, proposing RGX-202 as a one-time treatment, potentially challenging Sarepta's Elevidys in the DMD market. is advancing its gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) into pivotal development, with plans for a BLA submission by 2026. The company has secured FDA alignment on an accelerated approval pathway, proposing RGX-202 as a one-time treatment, potentially challenging Sarepta's Elevidys in the DMD market. In November 2024, Cumberland Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation and Rare Pediatric Disease Designation to Ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). Duchenne Muscular Dystrophy Overview Duchenne muscular dystrophy (DMD) is a severe, progressive genetic disorder characterized by muscle degeneration and weakness. It primarily affects boys, with symptoms typically becoming evident between the ages of 2 and 5. DMD is caused by mutations in the DMD gene located on the X chromosome, which encodes for dystrophin, a crucial protein that helps stabilize and protect muscle fibers during contraction. The absence or dysfunction of dystrophin leads to muscle damage, inflammation, and eventual replacement of muscle tissue with fat and fibrotic tissue. Clinically, children with Duchenne Muscular Dystrophy often present with delayed motor milestones, difficulty running or climbing stairs, frequent falls, and a characteristic waddling gait. As the disease progresses, they may develop calf muscle pseudohypertrophy, scoliosis, and cardiac or respiratory complications due to the involvement of the heart and diaphragm muscles. Cognitive difficulties and learning disabilities are also reported in some cases. The diagnosis of DMD typically involves a combination of clinical evaluation and laboratory tests. Elevated levels of creatine kinase (CK) in the blood are indicative of muscle damage and often prompt further investigation. Genetic testing is the gold standard for confirming mutations in the DMD gene. Muscle biopsy, though less commonly performed today, can reveal dystrophin deficiency. Additional assessments, such as electromyography (EMG) and cardiac evaluations, help monitor disease progression and guide management. Early diagnosis enables timely intervention with corticosteroids, physical therapy, and supportive care aimed at improving quality of life and prolonging mobility. Duchenne Muscular Dystrophy Epidemiology Segmentation The Duchenne muscular dystrophy epidemiology section provides insights into the historical and current Duchenne muscular dystrophy patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Duchenne muscular dystrophy market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Duchenne Muscular Dystrophy Age-specific Cases of Duchenne Muscular Dystrophy Ambulatory and Non-ambulatory Cases of Duchenne Muscular Dystrophy Mutation-specific Cases of Duchenne Muscular Dystrophy Associated Comorbidities in Duchenne Muscular Dystrophy Download the report to understand which factors are driving Duchenne muscular dystrophy epidemiology trends @ Duchenne Muscular Dystrophy Treatment Algorithm Duchenne Muscular Dystrophy Report Metrics Details Study Period 2020–2034 Duchenne Muscular Dystrophy Report Coverage 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] Key Duchenne Muscular Dystrophy Companies FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, and others Key Duchenne Muscular Dystrophy Therapies NS-089/NCNP-02 (brogidirsen), TAS-205 (pizuglanstat), RGX-202, VYONDYS 53, EXONDYS 51, AGAMREE, VILTEPSO, VILTEPSO, and others Scope of the Duchenne Muscular Dystrophy Market Report Duchenne Muscular Dystrophy Therapeutic Assessment: Duchenne Muscular Dystrophy current marketed and emerging therapies Duchenne Muscular Dystrophy current marketed and emerging therapies Duchenne Muscular Dystrophy Market Dynamics: Conjoint Analysis of Emerging Duchenne Muscular Dystrophy Drugs Conjoint Analysis of Emerging Duchenne Muscular Dystrophy Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Duchenne Muscular Dystrophy Market Access and Reimbursement Discover more about Duchenne muscular dystrophy drugs in development @ Duchenne Muscular Dystrophy Clinical Trials Table of Contents 1. Duchenne Muscular Dystrophy Market Key Insights 2. Duchenne Muscular Dystrophy Market Report Introduction 3. Duchenne Muscular Dystrophy Market Overview at a Glance 4. Duchenne Muscular Dystrophy Market Executive Summary 5. Disease Background and Overview 6. Duchenne Muscular Dystrophy Treatment and Management 7. Duchenne Muscular Dystrophy Epidemiology and Patient Population 8. Patient Journey 9. Duchenne Muscular Dystrophy Marketed Drugs 10. Duchenne Muscular Dystrophy Emerging Drugs 11. Seven Major Duchenne Muscular Dystrophy Market Analysis 12. Duchenne Muscular Dystrophy Market Outlook 13. Potential of Current and Emerging Therapies 14. KOL Views 15. Unmet Needs 16. SWOT Analysis Related Reports Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key DMD companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, among others. Duchenne Muscular Dystrophy Epidemiology Forecast Duchenne Muscular Dystrophy Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Duchenne muscular dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Facioscapulohumeral Muscular Dystrophy Market Facioscapulohumeral Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 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 FSHD companies, including Fulcrum Therapeutics, Dyne Therapeutics, Hoffmann-La Roche, aTyr Pharma, Inc., Avidity Biosciences, Inc., among others. Spinal Muscular Atrophy Market Spinal Muscular Atrophy Market Insights, Epidemiology, and Market Forecast – 2034 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 SMA companies, including Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Therapeutics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics, among others. Becker Muscular Dystrophy Market Becker Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 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 Becker muscular dystrophy companies, including Italfarmaco, Immunoforge Co., Ltd., Edgewise Therapeutics, Epirium Bio, Ultragenyx, Strykagen, among others. DelveInsight's Pharma Competitive Intelligence Service: Through its CI solutions, DelveInsight provides its clients with real-time and actionable intelligence on their competitors and markets of interest to keep them stay ahead of the competition by providing insights into the latest therapeutic area-specific/indication-specific market trends, in emerging drugs, and competitive strategies. These services are tailored to the specific needs of each client and are delivered through a combination of reports, dashboards, and interactive presentations, enabling clients to make informed decisions, mitigate risks, and identify opportunities for growth and expansion. Other Business Pharmaceutical Consulting Services Healthcare Conference Coverage Pipeline Assessment Healthcare Licensing Services Discover how a mid-pharma client gained a level of confidence in their soon-to-be partner for manufacturing their therapeutics by downloading our Due Diligence Case Study About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Connect with us on LinkedIn|Facebook|Twitter Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash


Business Insider
17-06-2025
- Business
- Business Insider
FibroGen trading halted, news pending
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Yahoo
13-05-2025
- Business
- Yahoo
Q1 2025 FibroGen Inc Earnings Call
Joanne Geller; Investor Relations; LifeSci Advisors Thane Wettig; Chief Executive Officer; FibroGen Inc David DeLucia; Chief Financial Officer; FibroGen Inc Andy Hsieh; Analyst; William Blair Matthew Keller; Analyst; H.C. Wainwright & Co Operator Good day, and thank you for standing by. Welcome to FibroGen first quarter 2025 earnings conference call. (Operator Instructions). Please be advised today's conference is being recorded. I would not like to end the conference over to your speaker today, Joanne Geller. Please, go ahead. Joanne Geller Thank you, operator. Good afternoon, everyone. Thank you for joining today to discuss FibroGen's first quarter 2025 financial and business results. I'm Joanne Geller from LifeSci Advisors. Joining me on today's call are Thane Wettig, our Chief Executive Officer; and David DeLucia, our Chief Financial Officer. Following the prepared remarks, we will open the call to your questions. I would like to remind you that remarks made on today's call include forward-looking statements about FibroGen. Such statements may include, but are not limited to, our collaborations with AstraZeneca and Astellas, financial guidance, initiation, enrollment, design, conduct, and results of clinical trials, our regulatory strategies and potential regulatory results, our research and development activities, commercial results, and results of operations, risks related to our business, and certain other business matters. Each forward-looking statement is subject to risks and uncertainties that could cause actual results and events to differ materially from those projected in that statement. A more complete description of these and other material risks can be found in FibroGen's filings with the SEC, including our most recent Form 10-K and Form 10-Q. FibroGen does not undertake any obligation to update publicly any forward-looking statements, whether as a result of new information, future events, or otherwise. The press release reporting the company's financial results and business update, and a webcast of today's conference call, can be found on the investor section of FibroGen's website at With that, I'd like to turn the call over to CEO, Thane Wettig. Thane? Thane Wettig Thank you, Joanne. Good afternoon, everyone, and welcome to our first quarter 2025 earnings call. On today's call, I will provide a status update on the transformation of FibroGen, which includes the divestiture of FibroGen China and a laser focus on our US pipeline opportunities, specifically the exciting prospects for FG-3246 and FG-3180, our potential first-in-class antibody drug conjugate targeted in CD46, and our PET imaging agent in metastatic castration-resistant prostate cancer. And for roxadustat in the treatment of anemia due to lower-risk myelodysplastic syndrome. Then, David DeLucia, our CFO, will review the financials, after which we will open the call for your questions. On slide 3, I would like to highlight the strategic priorities we've set forth for FibroGen this year. I'll begin by providing an update on the sale of FibroGen China to AstraZeneca. As we've stated previously, this is a truly transformative transaction for FibroGen as it simplifies our operations, allows for the payoff of our term loan facility with Morgan Stanley Tactical Value, and provides the most efficient pathway to access the company's net cash held in China. At the time of the announcement in February, the total consideration for the sale was expected to be approximately $160 million, which included an equity value of $85 million and expected net cash in China of approximately $75 million. We are pleased to share that we expect the total consideration to now be approximately $185 million, which is a $25 million increase from our initial guidance due to greater than expected net cash in China at closing. Importantly, the increase in expected proceeds extends the company's cash runway into the second half of 2027. We now expect the transaction to close in the third quarter of this year. Second, we remain hyper-focused on advancing FG-3246 and FG-3180 in metastatic castration-resistant prostate cancer or mCRPC, in which we continue to make important progress. We recently announced in March the publication of the full trial results from the Phase 1 monotherapy study of FG-3246 in patients with mCRPC in the Journal of Clinical Oncology, which highlights the promising potential of its anti-cancer activity, especially when considering the unselected, heavily pretreated patient population. We believe the trial results demonstrate that the CD46 target is active and provide key insights into the potential clinical impact of targeting CD46-expressing tumors. We are excited to share that we recently received notification from the FDA clearing the IND for FG-3180, our companion PET imaging agent. This marks an important achievement for FibroGen as it paves the pathway for FG-3180 to be used alongside FG-3246 in the upcoming Phase 2 dose optimization monotherapy trial, which is expected to start in the third quarter. Third, for roxadustat, FibroGen recently filed a type C meeting request with the FDA to gain feedback on the potential path forward for roxadustat in anemia associated with lower-risk myelodysplastic syndromes, an indication with significant unmet medical need. In the post hoc subgroup analysis from the MATTERHORN Phase 3 trial, roxadustat showed promise in reducing transfusion dependence in patients with a higher transfusion burden at baseline. We expect FDA feedback in the third quarter that will provide important clarity on the path forward for roxadustat in the US, with the aim of realizing additional value for this wholly owned asset. Altogether, we are confident that our refined focus, multiple near-term catalysts across both clinical programs, and our existing strong foundation position us well to create value for shareholders now and in the future. I will now provide a brief overview of our FG-3246 and FG-3180 programs in mCRPC. Slide 5 highlights the high unmet need in late-stage prostate cancer. There are approximately 290,000 men diagnosed with prostate cancer each year in the US. Of these, there are 65,000 drug-treatable patients where the cancer has metastasized and become castrate-resistant, resulting in a grim five-year survival rate of approximately 30%. There remains a significant opportunity for new treatments that can extend survival for these men, with a total addressable market of well over $5 billion in annual sales. FG-3246 could become a non-PSMA treatment option that is so desperately needed given the significant unmet need in mCRPC. Turning to slide 6, we highlight the novelty of our target, a tumor-selective epitope of CD46. CD46 and this specific CD46 epitope have several distinguishing features. First, CD46 is upregulated during tumorigenesis and helps tumors evade complement-dependent cytotoxicity. The CD46 epitope is highly expressed in mCRPC tissues with lower interpatient variability and higher median expression compared with PSMA, as depicted in the graph on the right-hand portion of the slide. Importantly, the expression of CD46 is upregulated in the progression from localized castration-sensitive prostate cancer to metastatic castration-resistant prostate cancer and further overexpressed following treatment with androgen signaling inhibitors. The CD46 epitope is also overexpressed in colorectal cancer and other solid tumors. Turning to slide 7, FG-3246 is a potential first-in-class ADC in development for mCRPC with a novel targeting antibody YS5, which binds to the tumor-selective epitope of CD46 along with an MMAE payload. MMAE is a validated payload that is approved as part of a number of ADCs and other oncology indications. FG-3246 represents an androgen receptor agnostic approach, clinically differentiating it from other prostate cancer treatments currently in development. A companion PET imaging agent, FG-3180, utilizes the same YS5 targeting antibody as FG-3246 and is also under clinical development. In preclinical studies, the PET imaging agent has demonstrated specific targeting of and uptake by CD46-positive tumor cells. We believe that having a patient selection biomarker would not only allow us to better enrich the patient population in the Phase 3 portion of the clinical development program, but it would also enable differentiation for FG-3246 in the prostate cancer treatment paradigm. In addition, FG-3180 could represent an important commercial opportunity as a companion diagnostic to FG-3246, similar to the existing PSMA PET agents such as PYLARIFY. We are excited to announce that we have recently received IND clearance for FG-3180, paving the way for FG-3180 to be an important component for the upcoming Phase 2 dose optimization study that I will touch on in a moment. Slide 8 recaps the top-line results from the Phase 1 monotherapy study with full details published in the peer-reviewed Journal of Clinical Oncology in March of this year. The completed monotherapy study included a total of 56 metastatic castration-resistant prostate cancer patients who were biomarker unselected and were heavily pre-treated, receiving a median of five lines of therapy prior to FG-3246. In the efficacy evaluable population of 40 patients, a median radiographic progression-free survival of 8.7 months was observed. There was an overall response rate of 20% confirmed by RECIST 1.1, and PSA reductions of greater than 50% were achieved in 36% of patients. Adverse events were consistent with those observed with other MMAE-based ADC therapies. Additional highlights from the JCO publication include the expression of CD46 being observed in 80% of evaluable biopsies in patients enrolled during the dose expansion phase, which is consistent with results previously reported from a prospectively obtained cohort of patients who underwent metastatic CRPC biopsy. 20 patients had evaluable circulating tumor DNA at baseline, on treatment defined as before the cycle 2 day 1 dose, and at the end of treatment or disease progression. In 9 of these 20 patients, or 45% of those evaluable, there was a greater than 50% decline from baseline in ctDNA fraction after just one cycle of treatment. A tighter dose-response relationship appeared to be observed for objective tumor response by imaging as opposed to serum PSA decline, which may be related to the independence of CD46 from the androgen signaling pathway and expression of CD46 in androgen receptor-independent tumor clones. Finally, anti-tumor activity was observed in patients who had received more than one previous line of ARPI therapy, as well as those who had received taxane chemotherapy in the metastatic castration-sensitive setting, the latter being notable given similar mechanisms of action of taxane and MMAE. Based on the totality of the data from the Phase 1 monotherapy trial, we are encouraged by the clinical activity of FG-3246 in targeting CD46 in mCRPC. On slide 9, we highlight the rPFS results of FG-3246 in its Phase 1 study versus other comparable early-stage studies. As covered on the previous slide, the Phase 1 study of FG-3246 demonstrated an rPFS of 8.7 months across a robust sample size of 40 heavily pre-treated patients. While we cannot make direct comparisons to these trials due to the differences in study design and prior prostate cancer treatments, we are encouraged by these rPFS results, which is a recognized regulatory endpoint in prostate cancer trials. On slide 10, we highlight previously reported preliminary efficacy data from the Phase 1b portion of the ongoing investigator-sponsored combination study with enzalutamide. These interim results included data on 17 biomarker unselected patients, 70% of whom were pre-treated with at least two prior ARSIs. In addition to establishing the Phase 2 dose of FG-3246, the IST also demonstrated an encouraging 10.2 months of radiographic progression-free survival, with PSA declines observed in 71% of evaluable patients. We expect to report the Phase 2 top-line results in the fourth quarter of this year, which will also include data on CD46 expression in patients treated with FG-3180, our PET biomarker, during the Phase 2 portion of the IST. On slide 11, we depict a comparison of the initial results from the monotherapy trial in heavily pre-treated patients and the combination trial for FG-3246 versus the rPFS results from second-line therapies in late-stage trials. Again, while we cannot make direct comparisons to these trials due to the differences in study design and previous prostate cancer treatments, we are encouraged that FG-3246 demonstrates what we believe to be competitive rPFS results. Slide 12 highlights the Phase 2 monotherapy dose optimization trial design that is based on our discussion with the FDA. We plan to initiate the study next quarter and expect to enroll 75 patients in the post-ARSI pre-chemo setting across three dose levels to determine the optimal dose for Phase 3 based on efficacy, safety, and PK parameters. It is important to note that FG-3180 will be an integral part of the study as we seek to demonstrate the correlation between CD46 expression and response to the ADC in this all-comers population. One other important design element is the use of UCSF as primary prophylaxis to mitigate Grade 3 or greater adverse events associated with neutropenia commonly seen with MMAE payloads. The addition of UCSF may enable a better tolerated and more consistent treatment with the ADC, minimizing dose interruptions or dose reductions, extending duration of therapy, and potentially enhancing the efficacy of the ADC. We are planning an interim analysis in the second half of 2026, which will include efficacy, safety, PK, and exposure-response data, and we intend to share relevant data with all stakeholders as they become available, given the open-label design. Slide 13 highlights why we are so optimistic about the potential for the Phase 2 study to further build upon the strong efficacy seen in the Phase 1 study. We believe there are three factors that could drive our PFS even higher than the 8.7 months observed in the Phase 1 monotherapy trial. First, preliminary evidence of an exposure-response relationship allows us to focus our Phase 2 study on three of the highest tolerated doses from the Phase 1 dose escalation and expansion study. Second, utilizing primary prophylaxis with UCSF to combat neutropenia potentially allows patients more consistent exposure to the ADC with fewer dose interruptions or adjustments. Third, enrolling patients in earlier lines of therapy versus the median five prior lines of therapy in the Phase 1 trial. We believe that these design elements have the potential to improve upon the Phase 1 results and achieve an rPFS in the 10 to 12-month range, which we believe is the benchmark for commercial competitiveness. On slide 14. We show our long-term development strategy for FG-3246 and FG-3180, which we believe provides significant optionality in prostate cancer. We have a robust Phase 2 monotherapy trial in the pre-chemo setting in mCRPC to further build upon the compelling efficacy data of 8.7 months of rPFS in 40 heavily pretreated biomarker-unselected patients from the Phase 1 monotherapy study. In addition, this study will explore the correlation between CD46 expression and response to the ADC, potentially validating FG-3180 as a predictive patient selection biomarker in future studies. We are confident that our development pathway for FG-3246 unlocks sequential or parallel registration pathways, as FG-3246 will be evaluated in multiple lines of therapy in monotherapy and/or in combination with an ARSI, and in an all-comers population or patients with high expression of CD46. Slide 15 shows the recent and upcoming catalysts for the FG-3246 and FG-3180 program. We are very pleased to have received IND clearance for FG-3180, as this marks an important milestone as we explore its potential to be used as a diagnostic tool and potential biomarker for patient selection in the treatment of mCRPC. We plan to initiate the Phase 2 monotherapy trial in the third quarter, which will include FG-3180 to enable assessment of its diagnostic performance and the potential correlation between CD46 expression and response to FG-3246. To summarize on slide 16, FG-3246 targets a novel epitope on prostate cancer cells with first-in-class potential. It is important to note that there are no other CD46-targeted projects in clinical development. Targeting CD46 with FG-3246 has already demonstrated promising early efficacy signals with an acceptable safety profile both in monotherapy and combination settings. We are excited for the upcoming milestones and look forward to updating you on the program as the studies progress. Turning to roxadustat, slide 18 highlights the unmet need and the potential for roxadustat in patients with anemia associated with lower-risk MDS. There is a lack of effective second-line and beyond treatments, given that the currently available therapies are only effective in approximately 50% of patients. In addition, there are no oral options available or in late-stage development, which could be a meaningful differentiator for roxadustat and potentially translate into a significant commercial opportunity. Moving on to slide 19, we highlight data from the Phase 3 MATTERHORN study of roxadustat in a subgroup of patients with anemia of lower-risk MDS who entered the trial with a higher transfusion burden. In this post hoc analysis, roxadustat demonstrated a meaningful difference in transfusion independence versus placebo, results that are highly similar to the pivotal trials for two recently approved therapies for anemia associated with lower-risk MDS. On slide 20, we highlight the significant opportunity for roxadustat in lower-risk MDS. Based on other lower-risk MDS development programs, we believe the indication would support an orphan drug designation, which would provide seven years of data exclusivity in the US. This potential exclusivity, combined with an attractive market opportunity and efficient commercial model, provides a significant economic opportunity for further development of roxadustat. We look forward to near-term discussions with the FDA, which could pave the way for developing roxadustat for anemia associated with lower-risk MDS on our own or through a potential partnership. With that, I will now turn the call over to Dave to discuss the company's financials. Dave? David DeLucia Thank you, Thane. I will first review the updated FibroGen China transaction details and then provide the company's financial performance for the first quarter of 2025. As a reminder, our China operations are reflected as discontinued operations throughout our financials. We will continue to report our China operations in continued operations moving forward. On slide 22, we highlight the summary of key financial terms of the transaction. Under the terms of the agreement, FibroGen will receive an enterprise value of $85 million plus FibroGen net cash held in China at closing, estimated to now be approximately $100 million, totaling approximately $185 million. This is a $25 million increase from our initial net cash guidance in February. Given the company's current market capitalization of approximately $30 million, we believe this increase in expected net cash received upon the close of the transaction represents a meaningful outcome for shareholders. As a reminder, the value of FibroGen net cash in China includes FibroGen's portion of Falikang net cash, which is the joint distribution entity owned by FibroGen and AstraZeneca. Importantly, FibroGen will continue to accrue cash generated in China until the closing of the transaction, which is expected in the third quarter of 2025, pending customary closing conditions including regulatory review in China. This transaction is truly transformative for FibroGen and allows the company to pay down its senior term loan facility with MSTV, fully access our cash in China, and extend the company's runway into the second half of 2027 to support US development initiatives. Now, on to the company's financials for the first quarter. For the first quarter of 2025, total revenue was $2.7 million compared to $25.4 million for the same period in 2024. For the full year 2025, we reiterate total revenue to be between $4 million and $8 million. Now, moving down the income statement, total operating costs and expenses for the first quarter of 2025 were $17.7 million compared to $74.5 million for the first quarter of 2024, a decrease of $56.8 million or 76% year-over-year. R&D expenses for the first quarter of 2025 were $9.2 million compared to $36.5 million in the first quarter of 2024, a decrease of $27.3 million or 75% year-over-year. SG&A expenses for the first quarter of 2025 were $8.1 million compared to $16.7 million in the first quarter of 2024, a decrease of $8.6 million or 51% year-over-year. During the first quarter of 2025, we recorded a net loss from continuing operations of $16.8 million or $0.16 net loss per basic and diluted share, as compared to a net loss of $49 million or $0.49 per basic and diluted share for the first quarter of 2024. For the full year 2025, we reiterate our guidance for our total operating costs and expenses, including stock-based compensation, to be between $70 million and $80 million, which at the midpoint represents a 58% reduction from the full year 2024. Now, shifting towards cash, as of December 31, we reported $33.8 million in cash, cash equivalents, and accounts receivable in the US, and $128.4 million in total consolidated cash, cash equivalents, and accounts receivable when including balances in China. The company was cash flow positive in the first quarter of 2025, generating a total of $7.3 million in cash flow on a total consolidated basis when including balances in China. Given that the company will continue to accrue cash from its China operations until the close of the sale transaction, we expect the company to again be cash flow positive on a consolidated basis in the second quarter of 2025. Upon close of the China transaction, we plan to pay off our senior secured term loan with Morgan Stanley Tactical Value, resulting in a cash outflow of approximately $80 million. This includes the $75 million principal balance, accrued and unpaid interest, and an applicable prepayment penalty. Post the payoff of our MSTV term loan, we expect the company to have runway into the second half of 2027. Thank you, and we'll now turn the call back over to Thane. Thane Wettig Thank you, Dave. To conclude, we are extremely excited about the future prospects for FibroGen with a number of important catalysts in the coming months. We plan to advance our exciting pipeline, initiating the Phase 2 monotherapy study next quarter for FG-3246 and FG3-180 in mCRPC. We will gain important feedback from the FDA regarding the potential development of roxadustat in lower-risk MDS, and we anticipate the close of the FibroGen China sale, payoff of the MSTV term loan, and extension of our cash runway into the second half of 2027. In summary, we are committed to driving significant shareholder value by advancing our US development initiatives, supported by our strong balance sheet. We look forward to providing further updates to our stakeholders over the coming months. I would now like to turn the call over to the operator for Q&A. Operator (Operator Instructions) Andy Hsish, William Blair. Andy Hsieh Thanks for taking our questions. Congratulations on the higher-than-expected proceeds from the AstraZeneca deal and the JCO publication. So, we have three questions. One has to do with clinical development on FG-3246 and so this has to do with some of the market dynamic changes after the Pluvicto approval based on the PSMA 4 study in the pre-chemo setting. I'm curious if you have contemplated potentially running the monotherapy study or the pivot program in the Pluvicto experienced population just to mitigate some of the risk associated with a highly heterogeneous population and maybe targeting a higher unmet medical need. So, that's question number one. Question number two, has to do with some of the macro disruptions that we have read about in the news or reported by the media. I'm just curious if you can comment on some of the recent FDA correspondence or communication, especially with the Roxadustat study and the opportunity. And third, I think this is something you have mentioned a couple of times in previous calls. Given the cash infusion from AstraZeneca, is it worthwhile, maybe from a capital allocation perspective, to conduct some feasibility studies in colorectal cancer, especially in light of [CytomX's] success this morning? Thank you very much Thane Wettig Hey, thanks, Andy, for the comments and the questions. Appreciate that. So, let me touch on the first one and then I'll ask Dave to comment as well. As it relates to clinical development for FG-3246 and some market dynamic changes with Pluvicto's new indication, as part of our synopsis or protocol for the Phase 2 monotherapy trial, we are going to allow Pluvicto experienced patients who happen to progress while on Pluvicto in that pre-chemo or post-ARSI pre-chemo setting to be entered into our trial. We don't think it makes sense to only or exclusively study those patients who are post-Pluvicto because, as I'm sure you can appreciate, Andy, you never have rapid adoption of an agent with a new indication. It usually takes time for clinicians to begin to adopt it. So, if we would require all patients who would be entered into our Phase 2 monotherapy trial to have been Pluvicto experienced, we just think that would create too much of an enrollment challenge. But we will be allowing patients who have been treated with Pluvicto to be entered into our Phase 2 monotherapy trial. Dave, anything to add to that? David DeLucia Nothing to add there, thanks. Thane Wettig Okay, and then in terms of the macro news and a lot of what's going on with respect to (inaudible) and FDA and the like, the only thing that we can point to are recent interactions that we've had with the agency. Maybe I'll give you just a flavor of that. The first one was when we filed the IND for FG-3180, which is our PET imaging agent. Everything progressed exactly on schedule. The questions, there were just a few of them, they came in a timely way. We answered them, the IND got cleared right on time. The next example that we have for the agency was the reactivation of the roxadustat IND in the US. We had deactivated it once we got the license back from AZ a little over a year ago. We needed to reactivate it in order to file the Type C meeting request. We requested the reactivation, that was also achieved right on time. And then in terms of the Type C meeting request that we filed, typically the FDA has 21 days or so to get back to the sponsor once a Type C meeting request has been filed. If they accept the Type C meeting request, there is then a date that has been set. We filed that Type C meeting request a week ago and have already heard back from the agency on the date that the Type C meeting is set for. So, the experiences that we have, Andy, I think are very favorable in terms of the FDA continuing to keep to certain timelines. In fact, we haven't experienced anything that's different than that. And then finally, in terms of cash from AZ and potentially doing a feasibility study for FG-3246 in mCRPC, I think we're going to hold off on that for now. We continue to evaluate lifecycle opportunities. The most important thing that we need to do is get the Phase 2 monotherapy trial started and then we'll be evaluating other opportunities that we think make sense from a lifecycle perspective. Dave, anything to add to that? David DeLucia No, I think you hit the nail on the head there, Thane. I think for us, obviously, we have identified colorectal cancer as a potential opportunity for FG-3246 given the expression of CD46 in those patients. But at the same point, I think our goals for this calendar year are really to close the transaction for China, kick off our Phase 2 studies, and really get the ball rolling around the development pathway for roxadustat in MDS as well. So, great question, Andy. Andy Hsieh Great, thank you so much. Thane Wettig Any other follow-up questions, Andy? Andy Hsieh I think that's it from us. Thank you so much. Thane Wettig Okay. Yeah, thanks for the questions. Appreciate it. Operator (Operator Instructions) Matthew Keller, HCW. Matthew Keller Hey everyone, congrats on the quarter and thanks for taking our questions. Just two quick ones from us. The first one, I was wondering if you have any additional or any rate-limiting steps ahead of the upcoming Phase 2 monotherapy study? And then secondly, with the updates on today's call, I was wondering how we should start to view the evolving commercial opportunity for FG-3180? Thane Wettig Thanks, Matt, for the questions. I'll take them and then ask Dave to add some additional commentary as well. Originally, as it relates to the Phase 2 monotherapy trial, we weren't certain how quickly we were going to get the FG-3180 IND cleared. So, there was a point in time where we thought that the majority of the patients in the Phase 2 monotherapy trial, the 75 patients, would be able to be treated with the PET biomarker in addition to the ADC. Now, with the clearing of the IND, I wouldn't call it a rate limiter, but what that allows us to do is to get an amendment in front of the sites that includes FG-3180 as part of the Phase 2 monotherapy study. So that all of the patients in the Phase 2 trial will be able to be treated with the PET biomarker in advance of receiving the ADC. We're going through that process right now as we speak. The rate limiter is the close of the China transaction so that we can move forward rapidly to then start the Phase 2 monotherapy trial. In terms of the question around the evolving landscape, what was that one question again? Matthew Keller Kind of like how we should view the commercial opportunity for FG-3180 as it evolves, both as a stand-alone product or in combination with some of the other things you guys have going on as well. Thane Wettig Yeah, it's a really important question. The Phase 2 trial will tell us a lot. It will give us even more information relative to what we have now. There have been about 25 or 27 patients who have been treated with the PET imaging agent in the Phase 1/2 investigator-sponsored trial at UCSF in combination with enzalutamide. We've seen those scans. It's clear that the target lights up with the PSMA imaging agent, and as you know, it uses the same YS5 antibody that is part of the ADC. The scans look really clear. The Phase 2 trial will then tell us if we continue to see the scans appropriately light up the lesions and what we can learn in terms of the assessment of the expression level of CD46 in response to the ADCs. That will be a correlation analysis that we will do during the course of the trial and at the end of the trial so that we can understand the diagnostic performance of the agent itself. We have looked at the radioligand space, we've looked at the PSMA PET space. Clearly, with the PSMA PET imaging agents from Lantheus and from Telix that generate well over $1.5 billion in annual revenue, there is a clear commercial opportunity. But we're going to have to assess the performance of our PET imaging agent to determine what that potential commercial opportunity could be. Dave, please add to that. David DeLucia Yeah, I think the one thing that I want to add around the Phase 2 and the importance of being able to dose all 75 patients is that we're really trying to get to a point where we can have a large swath of patients being able to be tested with FG-3180. The fact that we could have 75 patients in the Phase 2, we have the Phase 2 portion of the combination therapy and the IST with UCSF. The more and more patients that we can get treated with FG-3180 allows us to get really smart around what the levels of expression would be that could correlate with efficacy and allows us to better understand how it can be used in future studies. Matthew Keller Yep, no, totally makes sense. Thanks again for taking our questions. Appreciate you guys. Thane Wettig Thank you, Matt. Operator I'm not showing any further questions at this time. I turn the call back over to Thane for closing remarks. Thane Wettig We appreciate everybody's participation today and continued interest in FibroGen. We look forward to updating you over the coming weeks and months on the important catalysts that are ahead of us. Thank you for your time today. Operator Ladies and gentlemen, this concludes today's presentation. You may now disconnect and have a wonderful day. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
13-05-2025
- Business
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FibroGen (FGEN) Reports Q1 Loss, Tops Revenue Estimates
FibroGen (FGEN) came out with a quarterly loss of $0.16 per share versus the Zacks Consensus Estimate of $0.03. This compares to loss of $0.33 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -633.33%. A quarter ago, it was expected that this biotech drug developer would post a loss of $0.08 per share when it actually produced a loss of $0.08, delivering no surprise. Over the last four quarters, the company has surpassed consensus EPS estimates two times. FibroGen , which belongs to the Zacks Medical - Drugs industry, posted revenues of $2.74 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 71.19%. This compares to year-ago revenues of $55.9 million. The company has topped consensus revenue estimates three times over the last four quarters. The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call. FibroGen shares have lost about 41.8% since the beginning of the year versus the S&P 500's decline of -3.8%. While FibroGen has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock? There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately. Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions. Ahead of this earnings release, the estimate revisions trend for FibroGen: mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. It will be interesting to see how estimates for the coming quarters and current fiscal year change in the days ahead. The current consensus EPS estimate is -$0.09 on $1.7 million in revenues for the coming quarter and -$0.60 on $6 million in revenues for the current fiscal year. Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Drugs is currently in the top 27% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1. Another stock from the same industry, MediWound (MDWD), has yet to report results for the quarter ended March 2025. The results are expected to be released on May 21. This developer of treatments for burns and hard-to-heal wounds is expected to post quarterly loss of $0.65 per share in its upcoming report, which represents a year-over-year change of -66.7%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days. MediWound's revenues are expected to be $5.1 million, up 2.7% from the year-ago quarter. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report FibroGen, Inc (FGEN) : Free Stock Analysis Report MediWound Ltd. (MDWD) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
08-05-2025
- Business
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Duchenne Muscular Dystrophy (DMD) Pipeline Insight Report 2025, Featuring Analysis of Vamorolone (Santhera), Givinostat (Italfarmaco), and Pamrevlumab (Fibrogen)
Dublin, May 08, 2025 (GLOBE NEWSWIRE) -- The "Duchenne Muscular Dystrophy - Pipeline Insight, 2025" clinical trials has been added to report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in Duchenne Muscular Dystrophy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this HighlightsCompanies and academics are working to assess challenges and seek opportunities that could influence R&D Duchenne Muscular Dystrophy. The therapies under development are focused on novel approaches to treat/improve Duchenne Muscular Muscular Dystrophy Emerging Drugs Vamorolone: SantheraVamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors1,2. This has the potential to 'dissociate' efficacy from typical steroid safety concerns and therefore could emerge as a valuable alternative to corticosteroids, the current standard of care in children and adolescent patients with DMD. There is a clear unmet medical need in this patient group as high dose corticosteroids have significant systemic side effects that detract from patient quality of ItalfarmacoGivinostat, is an HDAC inhibitor (HDACi), a principle candidate, currently being developed for the treatment of DMD and BMD. Since Givinostat acts on the pathogenetic events downstream of the genetic defects, it is potentially a treatment for the whole DMD and BMD population and to counter the disease pathogenetic events in all muscular FibrogenPamrevlumab is a first-in-class antibody developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is advancing towards Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and pancreatic cancer and has been granted Orphan Drug Designation (ODD) in each of these indications, and is currently in a Phase 2 trial for Duchenne muscular dystrophy (DMD).Duchenne Muscular Dystrophy: Therapeutic AssessmentThis segment of the report provides insights about the Duchenne Muscular Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:Major Players in Duchenne Muscular DystrophyThere are approx. 75+ key companies which are developing the therapies Duchenne Muscular Dystrophy. The companies which have their Duchenne Muscular Dystrophy drug candidates in the most advanced stage, i.e phase III include RochePhases The report covers around 75+ products under different phases of clinical development like Late stage products (Phase III) Mid-stage products (Phase II) Early-stage product (Phase I) along with the details of Pre-clinical and Discovery stage candidates Discontinued & Inactive candidates Route of AdministrationDuchenne Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Molecule Type Products have been categorized under various Molecule types such as Small molecule Cell Therapy Peptides Polymer Small molecule Gene therapy Product TypeDrugs have been categorized under various product types like Mono, Combination and Mono/ ActivitiesThe report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Duchenne Muscular Dystrophy therapeutic drugs key players involved in developing key ActivitiesThe report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Duchenne Muscular Dystrophy drugs. Key Questions Current Treatment Scenario and Emerging Therapies: How many companies are developing Duchenne Muscular Dystrophy drugs? How many Duchenne Muscular Dystrophy drugs are developed by each company? How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Duchenne Muscular Dystrophy? What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Duchenne Muscular Dystrophy therapeutics? What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies? What are the clinical studies going on for Duchenne Muscular Dystrophy and their status? What are the key designations that have been granted to the emerging drugs? Key Players Santhera Pharmaceuticals Sarepta Therapeutics Italfarmaco Wave Life Sciences Ltd. FibroGen EDG 5506 Edgewise Therapeutics Fordadistrogene movaparvovec Daiichi Sankyo Sarepta Therapeutics, Inc. ENCell Taiho Pharmaceutical Solid Biosciences Capricor Nippon Shinyaku Hansa Biopharma Key Products Vamorolone Delandistrogene moxeparvovec Givinostat WVE N531 Pamrevlumab EDG 5506 Fordadistrogene movaparvovec Renadirsen SRP 5051 EN 001 TAS-205 SGT 001 CAP 1002 NS 089/NCNP 02 UX 810 For more information about this clinical trials report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900Sign in to access your portfolio