Epilepsies, Myoclonic

EPIDIOLEX has significant market potential due to the growing demand for alternative therapies and its effectiveness in treating rare seizure disorders like Lennox-Gastaut syndrome and Dravet syndrome. As awareness around the benefits of CBD in treating neurological conditions increases, EPIDIOLEX stands to benefit from expanding indications and market reach. LAS VEGAS, May 1, 2025 /PRNewswire/ -- DelveInsight's ' EPIDIOLEX Market Size, Forecast, and Market Insight Report' highlights the details around EPIDIOLEX, the first FDA-approved prescription cannabidiol (CBD) to treat seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or tuberous sclerosis complex (TSC) in patients 1 year of age or older. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of EPIDIOLEX. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Jazz Pharmaceuticals' EPIDIOLEX (cannabidiol) Overview EPIDIOLEX (formerly known as GWP42003-P and marketed as EPIDYOLEX in Europe) is the first plant-based, prescription cannabis-derived oral medication developed by GW Pharmaceuticals. It represents a new category of antiepileptic drugs with a unique mechanism of action. The active compound, cannabidiol (CBD), is a naturally occurring cannabinoid found in the Cannabis sativa L. plant. While the exact way EPIDIOLEX works to reduce seizures in humans remains unclear, it is distinct from traditional anticonvulsants. CBD does not act through CB1 receptors or by blocking voltage-gated sodium channels, which are typical pathways for other antiepileptic drugs. Instead, CBD is thought to work by influencing multiple internal pathways, potentially producing a combined anticonvulsant effect. These include enhancing neuronal inhibition (through synaptic and extrasynaptic GABA channels), affecting intracellular calcium levels (via TRPV channels, VDAC, and GPR55), and reducing inflammation, possibly via adenosine modulation. Importantly, CBD does not directly bind to or activate the CB1 or CB2 receptors at levels relevant to its seizure-reducing effects. Ongoing research is examining other possible mechanisms such as interactions with adenosine, glycine, GABAergic systems, and serotonin receptors. EPIDIOLEX has received regulatory approval in numerous countries, including the U.S. (FDA), Europe (European Commission), Great Britain (MHRA), Australia (Therapeutic Goods Administration), Switzerland (Swissmedic), Israel (Ministry of Health), and New Zealand (Medicines and Medical Devices Safety Authority). It is formulated as an oral solution containing highly purified CBD. In the U.S., it is approved to treat seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) in patients aged one year and older. In the European Union, under the name Epidyolex, it is approved as an add-on treatment with clobazam for LGS and DS in patients two years and older, and for TSC-associated seizures as well. Both the FDA and EMA have granted Orphan Drug Designation (ODD) for EPIDIOLEX/EPIDYOLEX for these conditions. EPIDIOLEX/EPIDYOLEX net product sales rose by 15% to USD 972.4 million in 2024 and by 14% to USD 275.0 million in the fourth quarter of 2024, compared to the corresponding periods in 2023. Learn more about EPIDIOLEX/EPIDYOLEX projected market size for DEE @ EPIDIOLEX Market Potential Developmental and epileptic encephalopathies (DEE) encompass a group of severe neurological conditions marked by developmental delays and persistent epilepsy. These disorders typically present early in life and are linked to significant cognitive deficits. According to DelveInsight's analysis, there were approximately 287K diagnosed prevalent cases of DEE across the 7MM in 2024. This number is projected to increase due to the rise in neurological disorders, including epilepsy, as well as environmental and maternal health factors contributing to a higher DEE prevalence throughout the forecast period (2025−2034). The US FDA has approved a range of medications for conditions like LGS, Dravet Syndrome, and CDKL5 Deficiency Disorder, including various drug classes such as sodium channel modulators, GABA receptor modulators, calcium channel blockers, receptor blockers, and others. Some of the available anti-epileptic drugs (AEDs) include FINTEPLA, EPIDIOLEX, TOPAMAX, BANZEL, LAMICTAL, FELBATOL, ONFI, KLONOPIN, DIACOMIT, SABRIL, and more. In 2024, the total market size for DEE in the 7MM was around USD 2.3 billion, and this figure is expected to grow by 2034, driven by wider adoption of approved therapies due to label expansions and the introduction of emerging treatments. Discover more about the DEE market in detail @ Developmental and Epileptic Encephalopathy Market Report Emerging Competitors of EPIDIOLEX Some of the drugs in the pipeline include EPX-100 (Harmony Biosciences), Bexicaserin (Lundbeck), Zorevunersen (Stoke Therapeutics/Biogen), Relutrigine (Praxis Precision Medicines), and others. In August 2024, the FDA removed a partial clinical hold as the company advances toward a Phase III registrational study of zorevunersen (STK-001) in children and adolescents with Dravet syndrome. In July 2024, Longboard Pharmaceuticals announced that the FDA had granted Breakthrough Therapy designation for its investigational drug bexicaserin for the treatment of seizures associated with DEEs for patients 2 years of age or older. To know more about the number of competing drugs in development, visit @ EPIDIOLEX Alternatives Key Milestones of EPIDIOLEX In August 2024, Jazz Pharmaceuticals reported top-line results from a Phase 3 open-label, single-arm study conducted in Japan, assessing the safety and effectiveness of its cannabidiol oral solution (marketed globally as EPIDIOLEX/EPIDYOLEX) as an add-on therapy for seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC). While the trial did not achieve its primary efficacy goal—defined as a pre-specified percentage reduction in seizure frequency during the up-to-16-week treatment period compared to baseline in Japanese pediatric patients—numerical improvements were noted in both the primary and several secondary measures. The study did not identify any new safety concerns. In May 2021, Jazz Pharmaceuticals acquired EPIDIOLEX as part of the acquisition of GW Pharmaceuticals plc, or GW, which expanded its growing neuroscience business with a global, high-growth childhood-onset epilepsy franchise. In April 2021, EPIDYOLEX was approved for adjunctive therapy of seizures associated with TSC for patients 2 years of age and older in the EU. In July 2020, EPIDYOLEX was approved for the treatment of seizures associated with TSC in patients one year of age and older. FDA also approved the expansion of all existing indications, LGS and DS, to patients one year of age and older. In September 2019, the European Commission approved EPIDYOLEX for use as adjunctive therapy of seizures associated with LGS or DS, in conjunction with clobazam, for patients two years of age and older. In June 2018, EPIDIOLEX was approved in the US for the treatment of seizures associated with two rare and severe forms of epilepsy, LGS and DS, in patients two years of age and older. EPIDIOLEX Launch Status EPIDIOLEX was launched on November 1, 2018, in the US market after FDA approval for the treatment of seizures associated with LGS or Dravet syndrome in patients two years of age and older. EPIDYOLEX was launched in 2019 in Germany and the UK market for use as adjunctive therapy for seizures associated with LGS or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older (GW Pharmaceuticals, 2019b). In 2021, EPIDYOLEX was launched in Spain. In the first quarter of 2022, the company launched EPIDYOLEX for LGS, Dravet syndrome, and TSC in Ireland, for TSC in Scotland and Wales. In the third quarter of 2022, the company launched EPIDYOLEX for TSC in Italy and Switzerland. In the fourth quarter of 2022, the company successfully completed the pricing and reimbursement process and commercial launch of EPIDYOLEX in France. Discover how EPIDIOLEX/EPIDYOLEX is shaping the DEE treatment landscape @ EPIDIOLEX CBD EPIDIOLEX Market Dynamics EPIDIOLEX, the first FDA-approved cannabidiol (CBD)-based medication for the treatment of rare forms of epilepsy, has had a significant impact on the pharmaceutical market since its approval in 2018. The drug is primarily used to treat seizures associated with Dravet syndrome and Lennox-Gastaut syndrome, two rare and severe forms of epilepsy. The market dynamic for EPIDIOLEX is shaped by several factors, including increasing awareness of its therapeutic potential, the expanding medical cannabis industry, and the growing demand for alternative treatments to traditional anti-epileptic drugs. One of the main drivers of the EPIDIOLEX market is the rising acceptance of CBD-based therapies. As medical cannabis becomes more mainstream and research into its medicinal properties continues to grow, patients and healthcare providers are increasingly turning to EPIDIOLEX as a treatment option. The growing body of clinical evidence supporting its effectiveness and safety has reinforced this trend. Additionally, as the stigma around cannabis-derived medications continues to fade, it opens up more opportunities for EPIDIOLEX in both pediatric and adult patient populations. However, the market for EPIDIOLEX is not without its challenges. High treatment costs and insurance coverage issues can limit accessibility for some patients, especially in markets outside the United States. Despite its clinical efficacy, the price point for EPIDIOLEX remains a point of contention, as it may not be affordable for all families, especially when compared to other conventional anti-epileptic drugs. Furthermore, competition from other cannabinoid-based therapies, as well as new developments in the broader epilepsy drug market, could impact the drug's market share over time. Looking ahead, the market dynamics for EPIDIOLEX will likely continue to evolve as the global regulatory landscape changes and new clinical trials further establish its benefits in other neurological conditions. With increasing research into CBD's potential applications, there may be opportunities for EPIDIOLEX to expand its indications, providing a solid foundation for its continued growth. However, market growth will also depend on the ability to address pricing concerns, improve insurance coverage, and navigate potential competitive pressures from both traditional and emerging therapies. Dive deeper to get more insight into EPIDIOLEX/EPIDYOLEX's strengths & weaknesses relative to competitors @ EPIDIOLEX Market Drug Report Table of Contents Related Reports Developmental and Epileptic Encephalopathies Market Developmental and Epileptic Encephalopathies Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DEE companies, including vid Therapeutics, Stoke Therapeutics, Biogen, Harmony Biosciences, Lundbeck (Longboard Pharmaceuticals), Praxis Precision Medicines, SK Life Science, Cerecin Neurosciences, Encoded Therapeutics, Praxis Precision Medicines, among others. Developmental and Epileptic Encephalopathies Pipeline Developmental and Epileptic Encephalopathies Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key DEE companies, including Denovo Biopharma, Cantex Pharmaceuticals, CNS Pharmaceuticals, CANbridge Pharmaceuticals, Vaximm, Inovio Pharmaceuticals, Mustang Bio, Bullfrog AI Holdings, Cantex, Chimeric Therapeutics, Philogen, Boehringer Ingelheim, Photonamic GmbH, Berg Pharma, Beyond Bio, Genenta Science, Polaris Pharmaceuticals, Telix Pharmaceuticals, Shanghai Simnova Biotechnology, NEONC Technologies, among others. Dravet Syndrome Market Dravet Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Dravet syndrome companies including Biocodex, GW Pharmaceuticals, Zogenix, Ovid Therapeutics, PTC Therapeutics, among others. Lennox-Gastaut Syndrome Market Lennox-Gastaut Syndrome 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 Lennox-Gastaut syndrome companies, including GlaxoSmithKline, Meda Pharmaceuticals, Roche, Lundbeck, Greenwich Biosciences, Janssen Pharmaceuticals, Eisai, Zogenix, Takeda, Ovid Therapeutics, among others. 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 . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

– Five presentations highlight TERLIVAZ research in adults with HRS with rapid reduction in kidney function,1 also known as HRS-AKI2 – DUBLIN, April 23, 2025 /PRNewswire/ -- Mallinckrodt plc, a global specialty pharmaceutical company, today announced the presentation of four posters and one oral presentation on TERLIVAZ® (terlipressin) for injection in patients with hepatorenal syndrome (HRS) with rapid reduction in kidney function,1 also known as HRS-acute kidney injury (AKI),2 at Digestive Disease Week (DDW), taking place in San Diego, CA from May 3-6, 2025. TERLIVAZ is the first and only FDA-approved product indicated to improve kidney function in adults with HRS with rapid reduction in kidney function,1 also known as HRS-AKI,2 an acute and life-threatening condition requiring hospitalization.3 HRS-AKI is estimated to affect more than 42,000 Americans annually, approximately 0.01% of the U.S. population,4 making it a very rare condition; and rates of HRS hospitalizations are increasing.5 Please see Limitation of Use and Important Safety Information, including Boxed Warning, below. "We'll be sharing the results of five clinical data analyses at DDW, as we continue to evaluate TERLIVAZ's impact in real-world situations," said Peter Richardson, MRCP (UK), Executive Vice President & Chief Scientific Officer. "We look forward to connecting with digestive disease colleagues in San Diego and discussing treatment options like TERLIVAZ for appropriate patients." Abstract #4243460: Treatment and Outcomes of Terlipressin in Hospitalized Adults with Hepatorenal Syndrome-Acute Kidney Injury (HRS-AKI): Real-world Evidence in Patients by Transplant Waitlist Status6 Embargoed Until: Monday, May 5, 2025; 12:01 a.m. PDT Presenter: Kavish R. Patidar Session Type: Oral Presentation Session Title: SOA - Cirrhosis in the Obese Patient Session Date and Time: Monday, May 5, 2025; 4:45 – 5:00 p.m. PDT Abstract #4253811: A Comparative Analysis of Real-World HRS-AKI Treatment and Outcomes in the UK and US: Raw Clinical Data and Propensity Score-Matched Evidence7 Embargoed Until: Tuesday, May 6, 2025; 12:01 a.m. PDT Presenter : Stevan A. Gonzalez Session Type: Poster Session Session Title: Portal Hypertension and Non Bleeding Complications of Cirrhosis: Encephalopathy, Ascites, and HRS Session Date and Time: Tuesday, May 6, 2025; 12:30 – 1:30 p.m. PDT Abstract #4253534: Real-World Treatment and Outcomes in Hospitalized Patients with Hepatorenal Syndrome-Acute Kidney Injury and Alcohol-Associated Liver Disease8 Embargoed Until: Tuesday, May 6, 2025; 12:01 a.m. PDT Presenter: Robert Wong Session Type: Poster Session Session Title: Portal Hypertension and Non Bleeding Complications of Cirrhosis: Encephalopathy, Ascites, and HRS Session Date and Time: Tuesday, May 6, 2025; 12:30 – 1:30 p.m. PDT Abstract #4230360: Transforming Hepatorenal Syndrome-Acute Kidney Injury Treatment: A Win Ratio Analysis of Terlipressin Versus Placebo9 Embargoed Until: Tuesday, May 6, 2025; 12:01 a.m. PDT Presenter: Kavish R. Patidar Session Type: Poster Session Session Title: Portal Hypertension and Non Bleeding Complications of Cirrhosis: Encephalopathy, Ascites, and HRS Session Date and Time: Tuesday, May 6, 2025; 12:30 – 1:30 p.m. PDT Abstract #4253824: Improving the Benefit-to-risk Profile: Efficacy Outcomes in Patients with Hepatorenal Syndrome-Acute Kidney Injury When Selected According to the Terlipressin Prescribing Information10 Embargoed Until: Tuesday, May 6, 2025; 12:01 a.m. PDT Presenter: Michael P. Curry Session Type: Poster Session Session Title: Portal Hypertension and Non Bleeding Complications of Cirrhosis: Encephalopathy, Ascites, and HRS Session Date and Time: Tuesday, May 6, 2025; 12:30 – 1:30 p.m. PDT These analyses were sponsored by Mallinckrodt Pharmaceuticals. INDICATION AND LIMITATION OF USE TERLIVAZ is indicated to improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function. Patients with a serum creatinine >5 mg/dL are unlikely to experience benefit. IMPORTANT SAFETY INFORMATION WARNING: SERIOUS OR FATAL RESPIRATORY FAILURE TERLIVAZ may cause serious or fatal respiratory failure. Patients with volume overload or with acute-on-chronic liver failure (ACLF) Grade 3 are at increased risk. Assess oxygenation saturation (e.g., SpO2) before initiating TERLIVAZ. Do not initiate TERLIVAZ in patients experiencing hypoxia (e.g., SpO2 <90%) until oxygenation levels improve. Monitor patients for hypoxia using continuous pulse oximetry during treatment and discontinue TERLIVAZ if SpO2 decreases below 90%. Contraindications TERLIVAZ is contraindicated: In patients experiencing hypoxia or worsening respiratory symptoms. In patients with ongoing coronary, peripheral, or mesenteric ischemia. Warnings and Precautions Serious or Fatal Respiratory Failure: Obtain baseline oxygen saturation and do not initiate TERLIVAZ in hypoxic patients. Monitor patients for changes in respiratory status using continuous pulse oximetry and regular clinical assessments. Discontinue TERLIVAZ in patients experiencing hypoxia or increased respiratory symptoms. Manage intravascular volume overload by reducing or discontinuing the administration of albumin and/or other fluids and through judicious use of diuretics. Temporarily interrupt, reduce, or discontinue TERLIVAZ treatment until patient volume status improves. Avoid use in patients with ACLF Grade 3 because they are at significant risk for respiratory failure. Ineligibility for Liver Transplant: TERLIVAZ-related adverse reactions (respiratory failure, ischemia) may make a patient ineligible for liver transplantation, if listed. For patients with high prioritization for liver transplantation (e.g., MELD ≥35), the benefits of TERLIVAZ may not outweigh its risks. Ischemic Events: TERLIVAZ may cause cardiac, cerebrovascular, peripheral, or mesenteric ischemia. Avoid use of TERLIVAZ in patients with a history of severe cardiovascular conditions or cerebrovascular or ischemic disease. Discontinue TERLIVAZ in patients who experience signs or symptoms suggestive of ischemic adverse reactions. Embryo-Fetal Toxicity: TERLIVAZ may cause fetal harm when administered to a pregnant woman. If TERLIVAZ is used during pregnancy, the patient should be informed of the potential risk to the fetus. Adverse Reactions The most common adverse reactions (≥10%) include abdominal pain, nausea, respiratory failure, diarrhea, and dyspnea. Please click here to see full Prescribing Information, including Boxed Warning. ABOUT DIGESTIVE DISEASE WEEK® (DDW) Digestive Disease Week® (DDW) is the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery. Jointly sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA), the American Society for Gastrointestinal Endoscopy (ASGE) and the Society for Surgery of the Alimentary Tract (SSAT), DDW is an in-person and online meeting from May 3-6, 2025. The meeting showcases more than 5,600 abstracts and hundreds of lectures on the latest advances in GI research, medicine and technology. More information can be found at . ABOUT HEPATORENAL SYNDROME (HRS) Hepatorenal syndrome (HRS) is a life-threatening condition that occurs in people with advanced liver disease.3 HRS is classified into two distinct types – a rapidly progressive type that leads to acute renal failure where patients are typically hospitalized for their care (HRS-acute kidney injury (AKI)) and a more chronic type that progresses over weeks to months (HRS-chronic kidney disease (CKD)).3 HRS-AKI is estimated to affect more than 42,000 Americans annually, approximately 0.01% of the U.S. population,4 making it a very rare condition; and rates of HRS hospitalizations are increasing.5 If left untreated, HRS-AKI has a median survival time of less than two weeks and greater than 80% mortality within three months.2 ABOUT MALLINCKRODT Mallinckrodt is a global business consisting of multiple wholly owned subsidiaries that develop, manufacture, market and distribute specialty pharmaceutical products and therapies. The Company's Specialty Brands reportable segment's areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, hepatology, nephrology, pulmonology and ophthalmology; neonatal respiratory critical care therapies; and gastrointestinal products. Its Specialty Generics reportable segment includes specialty generic drugs and active pharmaceutical ingredients. To learn more about Mallinckrodt, visit . CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTS This release contains forward-looking statements, including with regard to TERLIVAZ®, its potential to improve health and treatment outcomes, and its potential impact on patients. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: the effects of Mallinckrodt's recent emergence from bankruptcy; satisfaction of, and compliance with, regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; issues with product quality, manufacturing or supply, or patient safety issues or adverse side effects or adverse reactions associated with TERLIVAZ; and other risks identified and described in more detail in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Mallinckrodt's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and other filings with the SEC, all of which are available on its website. The forward-looking statements made herein speak only as of the date hereof and Mallinckrodt does not assume any obligation to update or revise any forward-looking statement, whether as a result of new information, future events and developments or otherwise, except as required by law. CONTACT Media Inquiries Green Room Communications 908-577-4531 [email protected] Investor Relations Derek Belz Vice President, Investor Relations 314-654-3950 [email protected] Mallinckrodt, the "M" brand mark, TERLIVAZ, and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company. Other brands are trademarks of a Mallinckrodt company or their respective owners. ©2025 Mallinckrodt. US-2500183 04/25 References 1 TERLIVAZ® (terlipressin) for injection. [Prescribing Information]. Mallinckrodt Hospital Products Inc. 2023. 2 Flamm S.L., Brown K., Wadei H.M., et al. The Current Management of Hepatorenal Syndrome–Acute Kidney Injury in the United States and the Potential of Terlipressin. Liver Transpl. 2021;27:1191-1202. 3 National Organization for Rare Disorders. Hepatorenal Syndrome. Available at:. Accessed March 2025. 4 United States Census Bureau: Quick Facts. Available at: . Accessed March 2025. 5 Singh J., Dahiya D.S., Kichloo A., et al. Hepatorenal Syndrome: A Nationwide Trend Analysis from 2008 to 2018. Annals of Med. 2021;53:1. 2018-2024 doi.org/10/1080/07853890. 6 Patidar K.R., Barritt A.S. IV, Wong R., et al. Treatment and Outcomes of Terlipressin in Hospitalized Adults with Hepatorenal Syndrome-Acute Kidney Injury (HRS-AKI): Real-world Evidence in Patients by Transplant Waitlist Status. Abstract to be presented in an oral presentation at the Digestive Disease Week Meeting. May 2025. 7 Gonzalez S.A., Allegretti A.S., Chirikov V.V., et al. A Comparative Analysis of Real-World HRS-AKI Treatment and Outcomes in the UK and US: Raw Clinical Data and Propensity Score-Matched Evidence. Abstract to be presented in a poster presentation at the Digestive Disease Week Meeting. May 2025. 8 Wong R., Patidar K.R., Barritt A.S. IV, et al. Real-World Treatment and Outcomes in Hospitalized Patients with Hepatorenal Syndrome-Acute Kidney Injury and Alcohol-Associated Liver Disease. Abstract to be presented in a poster presentation at the Digestive Disease Week Meeting. May 2025. 9 Patidar K.R., Hernaez R., Cullaro G., et al. Transforming Hepatorenal Syndrome-Acute Kidney Injury Treatment: A Win Ratio Analysis of Terlipressin Versus Placebo. Abstract to be presented in a poster presentation at the Digestive Disease Week Meeting. May 2025. 10 Curry M.P., Pappas S.C., Marden M., et al. Improving the Benefit-to-risk Profile: Efficacy Outcomes in Patients with Hepatorenal Syndrome-Acute Kidney Injury When Selected According to the Terlipressin Prescribing Information. Abstract to be presented in a poster presentation at the Digestive Disease Week Meeting. May 2025. SOURCE Mallinckrodt plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

In February 2025, the global biopharmaceutical industry remains active in clinical-stage drug financing and collaboration activities. For instance, XtalPi Holdings successfully raised over HKD 3.2 billion via two equity placements, which will be used to further develop its AI+CRO business model. Additionally, in the realm of innovative drug partnership deals, CSPC Group’s subsidiary signed an exclusive licensing agreement with Radiance Biopharma for the antibody-drug conjugate (ADC) SYS6005, which includes up to $150 million in potential development and regulatory milestone payments as well as up to $1.075 billion in potential sales milestone payments. Innovent Biologics outlined a global exclusive collaboration and licensing agreement with Roche for the next-generation ADC candidate IBI3009, with potential milestone payments reaching up to $1 billion. 1. Nxera Pharma and Viatris Collaborate to Advance the Development and Commercialization of an S1P1 Receptor Modulator in the Asia-Pacific Region On February 28, 2025, Nxera Pharma reached a significant agreement with Viatris Inc. and Idorsia Pharmaceuticals Ltd regarding the rights to develop and commercialize Cenerimod in Japan, South Korea, and other Asia-Pacific regions (excluding China). Cenerimod, a novel oral S1P1 receptor modulator, holds significant potential as a first-line or best-in-class therapy for various autoimmune diseases. Under the terms of the agreement, Nxera Pharma will receive an upfront payment of $10 million from Viatris and will be eligible for regulatory milestone payments upon Cenerimod's approval in Japan. Additionally, Nxera Pharma will collect royalties based on net sales within the designated territories. Cenerimod is a highly selective S1P1 receptor modulator that primarily works by controlling lymphocyte migration to reduce inflammation. Prior to this, Viatris had already secured exclusive global rights to develop and commercialize Cenerimod outside of Japan and the Asia-Pacific region (excluding China) through a 2024 research and development agreement with Idorsia. This new partnership with Nxera Pharma further reinforces Viatris's global positioning in drug development and provides robust support for Cenerimod's ongoing research and development. The mechanism of Cenerimod lies in its selective targeting of the S1P1 receptor. By inhibiting lymphocyte egress from peripheral lymphoid organs into systemic circulation, Cenerimod effectively suppresses inflammatory responses. This allows it to address the abnormally elevated levels of type I interferons (IFN-1) and other pro-inflammatory cytokines observed in autoimmune diseases such as systemic lupus erythematosus (SLE). This collaboration underscores the critical role of cross-company partnerships in accelerating the development of innovative medicines. It not only expedites market entry for novel drugs to meet unmet medical needs but also effectively distributes research and development risks, fostering sustainable growth within the pharmaceutical industry. For Cenerimod, this collaboration promises broader market access and technical support, paving the way for potential breakthroughs in the treatment of autoimmune diseases. 2. Shuangqi Pharmaceutical and Anaerobic Biotechnology Collaborate to Advance the Development and Commercialization of a Quadrivalent Lactobacillus Vaginal Live Biotherapeutic Capsule Inner Mongolia Shuangqi Pharmaceutical has entered into a significant agreement with Anaerobic Biotechnology concerning an innovative live biotherapeutic product, KAL-001, for vaginal use. This microecological therapy aims to restore a healthy vaginal environment by regulating its microbiota, specifically targeting vaginal inflammatory conditions in women of reproductive age. KAL-001 is composed of four specific strains of Lactobacillus, identified after a complex process of screening, isolation, preservation, and evaluation by Anaerobic Biotechnology. KAL-001 works by replenishing beneficial bacterial flora, i.e., Lactobacillus, in the vagina to restore and maintain a healthy microbial balance. This therapeutic approach differs from traditional antibiotic treatments. Instead of directly killing pathogens, it inhibits the growth of harmful microorganisms by increasing the population of beneficial microbes. Lactobacilli produce metabolic byproducts such as lactic acid, which lower vaginal pH and create an inhospitable environment for pathogenic organisms. Additionally, Lactobacillus may competitively exclude other potential pathogens and enhance the host’s local immune response through immunomodulatory mechanisms. The collaboration extends beyond product development and includes the transfer of commercialization rights. According to the licensing agreement, Shuangqi Pharmaceutical will pay Anaerobic Biotechnology an upfront fee of RMB 25 million and commit to milestone payments of up to RMB 170 million based on development progress and sales performance, along with royalties derived from net sales. This arrangement reflects the confidence both parties have in the market potential of KAL-001 and highlights their commitment to supporting R&D investments. 3. Sino Biopharmaceutical Collaborates with Sciwind Biosciences to Advance the IL-29 Project; CPX102 Expected to Become a Breakthrough Treatment for RSV Sino Biopharmaceutical has announced the signing of an exclusive strategic collaboration agreement with Sciwind Biosciences for the development and commercialization of the human interleukin-29 (IL-29, also known as interferon-λ1) project. The deal grants exclusive licensing and cooperation rights in 19 countries, including China (including Hong Kong, Macau, and Taiwan), Brazil, Saudi Arabia, Thailand, and Singapore. The partnership aims to expedite the development of CPX102 and expand its applications in the global market. CPX102 is an inhalable aerosol solution developed through protein engineering based on IL-29. As a type III interferon, it does not directly kill or inhibit viruses but induces antiviral protein production by activating cell surface receptors, thereby preventing viral replication. Additionally, it can activate immune cells, such as cytotoxic T lymphocytes (CTLs) and natural killer (NK) cells, thus enhancing the body's innate immune response against viral infections. This treatment is particularly effective for respiratory syncytial virus (RSV) infections in children, as it directly targets the epithelial cells in the lungs via inhalation, allowing for rapid onset of action and significantly improving the clearance of the virus. As the world's first aerosolized inhalation medication for RSV treatment, CPX102 offers high patient acceptability, particularly for children. Its local administration results in systemic exposure that is only about 1/100 of the injection dosage, drastically reducing the occurrence of systemic side effects. This not only helps avoid the discomfort associated with injections but also makes the treatment process safer and more comfortable, an especially critical factor for pediatric patients with pulmonary viral infections. As a broad-spectrum antiviral drug, CPX102 activates the host immune system to clear various respiratory viruses, including RSV, SARS-CoV-2, and influenza viruses, and is not affected by viral mutations. Given the current lack of effective treatments for RSV, particularly in high-incidence countries like China, CPX102 has vast market potential. It is estimated that RSV causes approximately 330 million respiratory infections globally each year, with China being one of the countries with the highest incidence rate. Currently, CPX102 is undergoing Phase IIb clinical trials in China and is expected to become the first Class 1 innovative drug in the country approved for the nebulized treatment of RSV infections in children. Considering the unmet demand for RSV therapies both in China and globally, along with the promising clinical results of CPX102, this drug has the potential to become a blockbuster product in the respiratory and anti-infective therapeutic area, bringing tremendous market value and social benefits. 4. Ligand Pharmaceuticals Invests $75 Million to Support Castle Creek Biosciences’ D-Fi Gene Therapy On February 25, Ligand Pharmaceuticals announced the completion of a $75 million royalty financing agreement with Castle Creek Biosciences to fund the Phase III clinical development of D-Fi (FCX-007), a gene therapy for dystrophic epidermolysis bullosa (DEB), through its initial data readouts. Of the $75 million investment, Ligand provided $50 million, while leading a consortium—including Paragon Biosciences, Valor Equity Partners, and new investor XOMA Royalty Corporation—that contributed the remaining $25 million. In return, the investors will receive single-digit percentage royalties on global sales of D-Fi. D-Fi is an autologous gene-modified cell therapy designed to treat DEB, a rare genetic skin disorder caused by mutations in the COL7A1 gene. This condition results in insufficient production of type VII collagen (COL7), which is critical for anchoring the dermis to the epidermis. To address this, D-Fi uses dermal fibroblasts harvested from the patient, modifies them ex vivo with a self-inactivating (SIN) lentiviral vector encoding the COL7A1 gene to produce COL7 protein, and locally injects the treated cells into chronic wounds. This promotes the formation of anchoring fibrils within the skin. Clinical studies have shown that D-Fi is well-tolerated, with the primary adverse reactions being localized injection site responses. This collaboration underscores Ligand's commitment to investing in breakthrough treatments that have the potential to transform patients’ lives while also diversifying its income-generating asset portfolio. Matthew Gantz, President and CEO of Castle Creek Biosciences, expressed support for the partnership, emphasizing its significance as a critical step towards addressing the unmet needs of patients with DEB. 5. Eli Lilly Acquires Organovo's FXR Program, Including Key Therapy FXR314 On February 25, 2025, Organovo Holdings announced that Eli Lilly and Company would acquire its FXR program, including its core asset FXR314. This transaction marks a significant milestone for Organovo in advancing drug development for inflammatory bowel disease (IBD) using their proprietary 3D human tissue models. FXR314 is a potentially "best-in-class" oral farnesoid X receptor (FXR) agonist, initially developed to target metabolic dysfunction-associated steatohepatitis (MASH). However, recognizing the immense market potential of inflammatory bowel disease and the limitations of existing therapeutic options, Organovo deemed FXR314 equally compelling for the IBD space. Preclinical studies have demonstrated that FXR314 improves key indicators of ulcerative colitis in a dose-dependent manner using an adoptive T-cell transfer model. Under the agreement terms, Organovo will receive an upfront payment from Eli Lilly, in addition to milestone payments as FXR314 progresses through critical regulatory and commercial achievements. Moreover, Eli Lilly will obtain worldwide rights to develop and commercialize Organovo’s FXR program, including all associated intellectual property. Organovo anticipates reporting results from the Phase IIa clinical trial of FXR314 for ulcerative colitis in the first half of 2026. Through this acquisition, Eli Lilly not only strengthens its portfolio in the inflammatory bowel disease field but also underscores its commitment to innovative therapies. Previously, Eli Lilly successfully launched the IL-23 monoclonal antibody Mirikizumab for treating ulcerative colitis and Crohn’s disease, achieving significant results. With the addition of FXR314 from Organovo, Eli Lilly is well-positioned to further expand its impact in IBD treatment, offering new hope to patients. 6. CARsgen Therapeutics Partners with SoftBank Xinchuang to Accelerate the Development of Off-the-Shelf CAR-T Cell Therapies in China On February 25, 2025, CARsgen Therapeutics announced a strategic partnership with SoftBank Xinchuang, jointly investing in Ukaize Biopharmaceuticals. This collaboration aims to accelerate the development and commercialization of CARsgen’s two off-the-shelf CAR-T cell therapy products in mainland China. These products include a BCMA CAR-T therapy targeting multiple myeloma and plasma cell leukemia, as well as a CD19/CD20 CAR-T therapy for B-cell malignancies. Through this partnership, CARsgen seeks to leverage external capital to expedite innovation in the CAR-T therapy field. As part of the agreement, a SoftBank Xinchuang-managed fund will subscribe to RMB 80,000,000 of newly issued registered capital in Ukaize Biopharmaceuticals, acquiring an 8% equity stake upon transaction completion. This financing not only provides essential funds for Ukaize but also reduces CARsgen’s ownership in Ukaize from 100% to 92%. The capital investment will be allocated to support Ukaize’s research, production, and commercialization efforts. For a rapidly evolving biotech company, such funding is vital to advancing clinical trials and shortening the time required to bring new products to market. CARsgen’s two CAR-T cell therapy products target key molecular markers: BCMA (B-cell maturation antigen) and CD19/CD20. The BCMA CAR-T therapy is primarily designed to treat multiple myeloma and plasma cell leukemia—both of which are challenging, refractory hematologic malignancies. Meanwhile, the CD19/CD20 CAR-T therapy is developed to combat B-cell-derived malignancies. These therapies work by genetically engineering patients' T cells to recognize and destroy cancer cells, offering a new form of personalized treatment. This approach represents a significant advancement in the field of cancer treatment by enabling precision-targeted therapy against cancer cells while minimizing damage to normal tissues. 7. CSPC Licenses ROR1-Targeting ADC to Radiance Biopharma On February 19, 2025, CSPC Pharmaceutical Group announced that it has granted Radiance Biopharma exclusive rights for the development and commercialization of its proprietary ROR1-targeting antibody-drug conjugate (ADC), SYS6005, in the United States and other major markets. Under the terms of the agreement, Radiance Biopharma will pay an upfront fee of $15 million, with additional development and regulatory milestone payments of up to $150 million, and sales milestone payments totaling up to $1.075 billion. Furthermore, Radiance Biopharma will pay tiered royalties based on the annual net sales of the product in the licensed territories. This collaboration marks another major milestone for CSPC in its global market expansion. SYS6005 is a monoclonal antibody-drug conjugate specifically designed to bind to the ROR1 receptor on the surface of tumor cells. Through endocytosis, the ADC delivers the potent mitotic inhibitor MMAE into cancer cells, where it releases its cytotoxic payload to effectively kill the cancer cells. This targeted design enhances therapeutic efficacy while minimizing damage to healthy tissues. SYS6005 received approval from the Chinese National Medical Products Administration (NMPA) to enter clinical trial stages by the end of 2024, with indications including hematological malignancies, ovarian cancer, non-small cell lung cancer, and other advanced malignancies. These developments highlight the broad potential application of SYS6005 in oncology. 8. Brexogen and BMI Korea Enter Technology Transfer Agreement for Exosome-Based Therapy On February 19, 2025, Brexogen, a biotechnology company specializing in exosome-based therapies, announced a technology transfer and licensing agreement with BMI Korea for its innovative injectable therapeutic, BxC-I17e. BxC-I17e, an exosome-based therapy currently undergoing clinical trials for the treatment of atopic dermatitis in the United States, is at the forefront of Brexogen’s pipeline. Under the terms of the agreement, BMI Korea gains rights to develop additional potential indications, excluding immune-related diseases, for domestic and major export markets. Financially, Brexogen will receive a non-refundable upfront payment of 30 billion KRW (approximately $23 million) and will be eligible for milestone payments tied to clinical, regulatory, and commercial achievements, alongside double-digit percentage royalties on net annual sales. Additionally, if BMI Korea enters sublicensing agreements with third parties, Brexogen will share in the resultant licensing revenues. BMI Korea, a company generating over 100 billion KRW in annual revenue, brings robust pharmaceutical manufacturing capabilities, ensuring high-quality and scalable production for BxC-I17e. BxC-I17e is a key asset in Brexogen's pipeline, leveraging the company’s proprietary exosome technology, which allows precise control over the therapeutic cargo carried by exosomes to enable targeted disease treatment. Currently undergoing Phase I clinical trials for atopic dermatitis, BxC-I17e demonstrates broad therapeutic potential. BMI Korea plans to explore its application in indications outside the immunology field, offering promising new therapeutic possibilities. At present, no exosome-based therapies have received commercial approval globally, making this collaboration a significant step forward in advancing innovation in exosome therapeutic development and setting new benchmarks within the industry. Brexogen is not only exploring dermatological treatments with BxC-I17e but has already successfully commercialized products like BREXTEM-S for skin regeneration and BREXTEM-H for hair restoration. Looking ahead, Brexogen is preparing for its anticipated 2026 IPO on KOSDAQ, which is expected to accelerate the clinical and commercial adoption of exosome-based therapies. 9. Biogen and Stoke Therapeutics Collaborate on Developing a Treatment for Dravet Syndrome On February 18, 2025, Biogen and Stoke Therapeutics announced a collaboration to develop and commercialize zorevunersen, a potentially first-in-class therapeutic for the treatment of Dravet syndrome. Dravet syndrome is a rare genetic epilepsy disorder characterized by treatment-resistant seizures and neurodevelopmental delays. Under the agreement, Stoke will continue to lead global development and retain exclusive development and commercialization rights in the U.S., Canada, and Mexico, while Biogen will have exclusive commercialization rights in other territories worldwide. The partnership leverages Biogen’s extensive expertise in neurological disorders and its global commercialization capabilities, aiming to bring new therapeutic hope to patients with Dravet syndrome. As part of this collaboration, Stoke will receive an upfront payment of $165 million, and both companies will share external clinical development costs, with Biogen contributing 30% and Stoke covering 70%. Additionally, Stoke is eligible for up to $385 million in development and commercial milestone payments. If zorevunersen achieves commercial success globally, Stoke will receive tiered royalties on net sales, ranging from low double digits to high teens. This financial structure not only provides Stoke with the necessary funding support but also lays the groundwork for subsequent research and development activities. Zorevunersen is an investigational antisense oligonucleotide (ASO) targeting the SCN1A gene, which is the root cause of most Dravet syndrome cases. The drug has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) based on preliminary clinical evidence suggesting it can provide significant improvements unmet by existing therapies. In Phase I/IIa and open-label extension (OLE) studies, patients treated with zorevunersen experienced a sustained reduction in the frequency of convulsive tonic-clonic seizures, as well as improvements across multiple cognitive and behavioral metrics. These findings support the potential for zorevunersen to progress to pivotal global Phase III trials, potentially becoming the first treatment to alter the course of Dravet syndrome. Currently, there are no approved disease-modifying therapies for Dravet syndrome, making the development of zorevunersen highly significant. The pivotal Phase III EMPEROR trial is expected to commence in the second quarter of 2025, with primary data readouts anticipated in the second half of 2027 to support global regulatory submissions. This progress is crucial for addressing the significant unmet medical needs of patients with Dravet syndrome and may herald a new era in the treatment of severe genetic disorders. 10. Foundation Medicine Partners with Sumitomo Pharma America to Advance Innovative Treatments for Acute Leukemia On February 18, 2025, Foundation Medicine, a genomics company dedicated to transforming cancer care, announced a collaboration with Sumitomo Pharma America (SMPA) to develop FoundationOne® Heme as a companion diagnostic tool to identify acute leukemia patients with KMT2A rearrangements or NPM1 mutations. These patients may be eligible for treatment with SMPA’s investigational menin inhibitor, enzomenib (DSP-5336). The collaboration aims to leverage advanced comprehensive genomic analysis to develop novel targeted therapies for hematologic malignancies. Acute leukemia is a condition requiring urgent intervention due to its rapid proliferation of blood cells, leading to the swift onset of symptoms. Approximately 30% of acute myeloid leukemia (AML) patients carry NPM1 mutations, while 5–10% of AML patients present with KMT2A (MLL) rearrangements. Menin inhibitors are gaining focus as a targeted therapeutic option for these specific genetic alterations. Menin plays a critical role in regulating hematopoiesis through its interaction with the KMT2A gene. The mechanism of action of menin inhibitors involves disrupting this protein-protein interaction, thereby inhibiting the proliferation of leukemia cells. Enzomenib (DSP-5336), developed by Sumitomo Pharma, is a menin inhibitor designed to treat patients with KMT2A rearrangements or NPM1 mutations in acute leukemia. The drug works by interrupting the interaction between menin and KMT2A, aiming to inhibit the growth of leukemia cells. Preclinical studies have shown promising results for this therapeutic approach in patients with the aforementioned genetic variants. This partnership is expected to drive the development of enzomenib and accelerate its clinical application. The FoundationOne® Heme platform combines DNA sequencing with RNA sequencing technologies, enabling the detection of all types of genomic alterations, including base substitutions, insertions and deletions, copy number variations, and gene fusions. This platform provides physicians with testing options for both blood and tissue samples, helping tailor personalized treatment plans based on a patient's specific genomic alterations. Read the Rest of the Series Highlights of February 2025: Biopharmaceutical Clinical-Stage Drug Investment, Financing, and Collaboration Deals (2) How to get the latest progress on drug deals? If you would like to access the latest transaction event information, you can click on the 'Deal' module from the homepage of the Synapse database. Within the Deal module, you can search for global pharmaceutical transaction information using labels such as Drugs, Organization, Target, Drug Type, Deal Date.  Furthermore, you can obtain the original link to the transaction coverage by clicking on the "Deal Name." In the analysis view, you can see the most active assignors, assignees, popular targets, and other dimensions of analysis, as well as the distribution of research and development statuses at the time of the transaction, to help you better understand the search results. The Synapse database also supports the ability to view current transactions from the dimension of "drugs" (by selecting "drugs" from the "Adjust Dimension" dropdown menu above). Targeting transactions involving renowned pharmaceutical companies that are of interest to the industry, such as Merck, Roche, etc., Synapse has identified a group of "leading companies" through drugs that have achieved global sales exceeding 1 billion US dollars in 2022. Transactions involving drugs from these leading companies can be filtered by clicking on the "Leading Company" tag on the left-hand side. In addition to the drug transaction module, you can also view related transaction history on the drug detail page and the institution detail page. Click on the image below to explore new pharmaceutical funding transactions!