Tauroursodeoxycholic Acid (TUDCA) Plus Camrelizumab and Regorafenib in Hepatocellular Carcinoma Previously Treated With Anti-PD1/PD-L1 and Bevacizumab: A Randomized, Phase 2 Study.

To evaluate the efficacy and safety of Tauroursodeoxycholic Acid (TUDCA) plus Camrelizumab and Regorafenib for patients with advanced hepatocellular carcinoma (HCC) who have progressed on prior systemic treatment with Anti-PD1/PD-L1 plus bevacizumab combination.

Start Date01 Aug 2025

Sponsor / Collaborator

Fudan University

NCT07064668

/ Not yet recruitingPhase 2IIT

Prospective Study on the Synergistic Effect of Tauroursodeoxycholic Acid Combined With Immunotherapy in Hepatocellular Carcinoma

This is a clinical trial that aims to evaluate whether adding a bile acid called tauroursodeoxycholic acid (TUDCA) can improve the effects of immunotherapy in patients with advanced liver cancer (hepatocellular carcinoma).
Immunotherapy has shown promise in treating this type of cancer, but not all patients respond well. TUDCA is known to help protect liver cells and may improve the liver's immune environment, potentially making immunotherapy more effective.
In this study, 300 patients with advanced liver cancer will be randomly assigned to receive either immunotherapy alone or immunotherapy combined with TUDCA.
Researchers will look at how well the cancer responds, whether the treatment helps more patients become eligible for surgery, and how safe the combination is.
The goal is to find a more effective and better tolerated treatment for patients with liver cancer.

Start Date30 Jul 2025

Sponsor / Collaborator

Tongji Hospital Tongji Medical College of HUST

CTR20241944

/ CompletedNot Applicable

牛磺熊去氧胆酸胶囊人体生物等效性研究

[Translation] Study on the bioequivalence of tauroursodeoxycholic acid capsules in healthy volunteers

主要目的：采用单中心、随机、开放、三周期部分重复交叉、单剂量、空腹及餐后给药设计，比较杭州沐源生物医药科技有限公司提供的牛磺熊去氧胆酸胶囊（规格：250 mg）与Bruschettini S.r.l.（意大利贝斯迪大药厂）持证DOPPEL FARMACEUTICI S.R.L.生产的牛磺熊去氧胆酸胶囊（商品名：滔罗特®/TAUROLITE®；规格：250 mg）在健康人群中吸收程度和速度的差异。次要目的：评价空腹及餐后条件下，杭州沐源生物医药科技有限公司提供的牛磺熊去氧胆酸胶囊（规格：250 mg）与Bruschettini S.r.l.（意大利贝斯迪大药厂）持证DOPPEL FARMACEUTICI S.R.L.生产的牛磺熊去氧胆酸胶囊（商品名：滔罗特®/TAUROLITE®；规格：250 mg）的安全性。

[Translation]

Primary objective: A single-center, randomized, open, three-period partially repeated crossover, single-dose, fasting and postprandial administration design was used to compare the differences in the degree and speed of absorption of tauroursodeoxycholic acid capsules (specification: 250 mg) provided by Hangzhou Muyuan Biopharmaceutical Technology Co., Ltd. and tauroursodeoxycholic acid capsules (trade name: TAUROLITE®; specification: 250 mg) produced by DOPPEL FARMACEUTICI S.R.L., a licensed pharmaceutical company of Bruschettini S.r.l. (Italy). Secondary objective: Evaluate the safety of tauroursodeoxycholic acid capsules (specification: 250 mg) provided by Hangzhou Muyuan Biopharmaceutical Technology Co., Ltd. and tauroursodeoxycholic acid capsules (trade name: TAUROLITE®; specification: 250 mg) produced by DOPPEL FARMACEUTICI S.R.L., a licensed pharmaceutical company of Bruschettini S.r.l. (Italy). Secondary objective: Evaluate the safety of tauroursodeoxycholic acid capsules (specification: 250 mg) provided by Hangzhou Muyuan Biopharmaceutical Technology Co., Ltd. and tauroursodeoxycholic acid capsules (trade name: TAUROLITE®; specification: 250 mg) produced by DOPPEL FARMACEUTICI S.R.L., a licensed pharmaceutical company of Bruschettini S.r.l. (Italy).

Start Date26 Jun 2024

Sponsor / Collaborator

Hangzhou Muyuan Biomedical Technology Co., Ltd.

100 Clinical Results associated with Tauroursodeoxycholate sodium

100 Translational Medicine associated with Tauroursodeoxycholate sodium

100 Patents (Medical) associated with Tauroursodeoxycholate sodium

3,244

Literatures (Medical) associated with Tauroursodeoxycholate sodium

01 Apr 2026·Neural Regeneration Research

Unfolded protein response in endoplasmic reticulum stress associated with retinal degenerative diseases: A promising therapeutic target.

Article

Author: Zhang, Hongbing ; Li, Xiaogang ; Mu, Yalin ; Li, Hongsong

The unfolded protein response is a cellular pathway activated to maintain proteostasis and prevent cell death when the endoplasmic reticulum is overwhelmed by unfolded proteins. However, if the unfolded protein response fails to restore endoplasmic reticulum homeostasis, it can trigger pro-inflammatory and pro-death signals, which are implicated in various malignancies and are currently being investigated for their role in retinal degenerative diseases. This paper reviews the role of the unfolded protein responsein addressing endoplasmic reticulumstress in retinal degenerative diseases. The accumulation of ubiquitylated misfolded proteins can lead to rapid destabilization of the proteome and cellular demise. Targeting endoplasmic reticulum stress to alleviate retinal pathologies involves multiple strategies, including the use of chemical chaperones such as 4-phenylbutyric acid and tauroursodeoxycholic acid, which enhance protein folding and reduce endoplasmic reticulum stress. Small molecule modulators that influence endoplasmic reticulum stress sensors, including those that increase the expression of the endoplasmic reticulum stress regulator X-box binding protein 1, are also potential therapeutic agents. Additionally, inhibitors of the RNAse activity of inositol-requiring transmembrane kinase/endoribonuclease 1, a key endoplasmic reticulum stress sensor, represent another class of drugs that could prevent the formation of toxic aggregates. The activation of nuclear receptors, such as PPAR and FXR, may also help mitigate ER stress. Furthermore, enhancing proteolysis through the induction of autophagy or the inhibition of deubiquitinating enzymes can assist in clearing misfolded proteins. Combination treatments that involve endoplasmic-reticulum-stress-targeting drugs and gene therapies are also being explored. Despite these potential therapeutic strategies, significant challenges remain in targeting endoplasmic reticulum stress for the treatment of retinal degeneration, and further research is essential to elucidate the mechanisms underlying human retinal diseases and to develop effective, well-tolerated drugs. The use of existing drugs that target inositol-requiring transmembrane kinase/endoribonuclease 1 and X-box binding protein 1 has been associated with adverse side effects, which have hindered their clinical translation. Moreover, signaling pathways downstream of endoplasmic reticulum stress sensors can contribute to therapy resistance. Addressing these limitations is crucial for developing drugs that can be effectively used in treating retinal dystrophies. In conclusion, while the unfolded protein response is a promising therapeutic target in retinal degenerative diseases, additional research and development efforts are imperative to overcome the current limitations and improve patient outcomes.

31 Dec 2025·Virulence

Dietary iron attenuates Clostridioides difficile infection via modulation of intestinal immune response and gut microbiota

Article

Author: Cui, Wenwen ; Li, Xiao ; Zang, Wanqing ; Yang, Huan ; Wu, Xiaoxiao ; Zhou, Zhou ; Chen, Ying

Clostridioides difficile (C. difficile) is one of the majors causes of antibiotic-associated diarrhea globally. Host vulnerability to C. difficile infection (CDI) is largely affected by gut microbiota, which in turn is influenced by diet. However, the mechanism underlying the interplay between diet and the gut microbiota that regulates host susceptibility to CDI remains unclear. This study aimed to investigate how a high-iron diet affects the intestinal immune response, microbiota, and metabolism in mice infected with C. difficile. We explored the specific role of the unique gut microbiota and metabolites on CDI. A mouse model of CDI was constructed with or without high dietary iron treatment. The effect of high iron levels on gut microbiota was analyzed by 16S rRNA gene sequencing, and the role of gut microbiota was confirmed by fecal microbiota transplantation (FMT). High dietary iron (400 mg/kg ferrous sulfate) alleviated CDI by decreasing C. difficile pathogenicity and altering host intestinal neutrophil recruitment. Furthermore, E. coli AVS0501, enriched in the gut microbiota of iron-treated CDI mice, showed prophylactic and therapeutic effects on CDI. Moreover, the production of L-proline and tauroursodeoxycholic acid (TUDCA) in CDI mice treated with high dietary iron influenced C. difficile colonization, toxin production, and in turn, regulates the intestinal neutrophil response. Thus, high dietary iron alleviates C. difficile induced enteritis by regulating gut microbiota maintaining gut homeostasis, suggesting that high dietary iron may be an important determinant of disease control.

01 Dec 2025·Toxicology reports

Targeting mitochondrial damage and ER stress to inhibit ferroptosis in cadmium-induced nephrotoxicity

Review

Author: Mammadova, Khayala ; Arshad, Muhammad ; Raza, Muhammad ; Fatima, Anmol ; Yousaf, Saba

This review highlights the pivotal roles of autophagy, ferroptosis, and endoplasmic reticulum (ER) stress in mediating cadmium (Cd)-induced nephrotoxicity. Cadmium exposure results in ER stress, which in turn activates major UPR pathways such as IRE1, ATF6, and PERK. By encouraging lipid peroxidation and suppressing cellular antioxidant defence, these mechanisms worsen ferroptosis and produce a feedback mechanism that increases cellular damage. There are two roles of autophagy in Cd-induced ferroptosis, which include its action in reducing cadmium-induced cytotoxicity by breaking down damaged components, and excessive autophagy, namely ferritinophagy, which promotes ferroptosis by iron dysregulation. The rise of mitochondrial ROS (MitoROS) caused by Cd-induced mitochondrial malfunction aids ferroptosis. This, in turn, causes ER stress and autophagy. This implies that focusing on mitochondrial health could be a useful treatment strategy. Effective treatment approaches include autophagy inhibitors like chloroquine, which have been shown to effectively reduce Cd-induced ferroptosis, and promising medicines that suppress ER stress, such as TUDCA. Desferrioxamine and other iron chelators effectively lower lipid peroxidation and iron dysregulation, therefore preventing ferroptotic cell death. Additionally, a multi-targeted treatment plan is suggested that targets iron metabolism, ER stress, and autophagy. In order to create tailored treatments for Cd-induced nephrotoxicity, this review emphasizes the need for additional study into the molecular pathways of Cd-induced ferroptosis, namely the ER stress-autophagy axis. The goal of future research should be to apply these mechanistic insights to clinical settings to enhance public health outcomes and create efficient therapies for renal failure brought on by cadmium toxicity.

135

News (Medical) associated with Tauroursodeoxycholate sodium

02 Jul 2025

·www.einpresswire.com

Amyotrophic Lateral Sclerosis (ALS): Competitive Intelligence Insight into Next-Gen Therapies | DataM Intelligence

ALS treatment is evolving from symptom management to disease-modifying strategies gene therapies, antisense drugs, & neuroprotective agents promise real impact ALS, once incurable, is seeing remarkable progress. With gene interventions and neuroprotective molecules entering late-stage trials, the vision of slowing or halting progression is closer than ever.” — DataM Intelligence AUSTIN, TX, UNITED STATES, July 2, 2025 / EINPresswire.com / -- Amyotrophic Lateral Sclerosis (ALS) , often known as Lou Gehrig’s disease, is a fatal neurodegenerative disorder that progressively destroys motor neurons, leading to paralysis and death. It remains one of the most complex and devastating neurological diseases, with a prognosis of just 2 to 5 years post-diagnosis for most patients. However, the landscape in 2025 reflects significant progress. With deeper genetic insights and a diversified therapeutic pipeline, ALS is transitioning from symptom management toward disease modification. Download CI Sample Report: https://www.datamintelligence.com/strategic-insights/ci/amyotrophic-lateral-sclerosis-als 🧬 Disease Overview ALS causes the degeneration of both upper and lower motor neurons, disrupting voluntary muscle activity. This leads to severe muscle wasting, difficulties in swallowing and breathing, and ultimately, respiratory failure. While about 90% of ALS cases are sporadic, the remaining 10% are familial, frequently linked to mutations in genes such as SOD1, C9orf72, TARDBP, and FUS. Diagnosis is typically clinical, supported by electromyography (EMG), nerve conduction studies, and exclusion of mimicking conditions. Biomarker development is ongoing, aiming to enable earlier diagnosis and patient stratification for clinical trials. 🌍 Epidemiology & Market Landscape Globally, ALS affects an estimated 2–5 people per 100,000, with an annual incidence of 1–2 per 100,000. The disease is more common in individuals aged 55–75 and is slightly more prevalent in men. Due to its high mortality and lack of curative therapies, ALS qualifies for orphan drug status in major markets. This regulatory incentive, combined with strong advocacy and rising R&D investment, is accelerating the therapeutic pipeline. The ALS treatment market, valued at around USD 2 billion in 2024, is projected to grow steadily with the entry of novel disease-modifying therapies over the next five years. 💊 Current Treatment Paradigm The current standard of care is limited to supportive and symptomatic therapies. These include: - Riluzole: Approved in 1995, it modestly extends survival by a few months by reducing glutamate-mediated excitotoxicity. - Edaravone: Approved in 2017, it acts as a free radical scavenger, slightly delaying functional decline in early-stage ALS. - Nuedexta: Although not approved specifically for ALS, it is commonly used to manage pseudobulbar affect (PBA), improving emotional stability. While helpful, these therapies do not halt or reverse the disease, highlighting a clear unmet need for disease-modifying approaches. Book Free CI Consultation Call: https://www.datamintelligence.com/strategic-insights/ci/amyotrophic-lateral-sclerosis-als 🔬 Pipeline Innovation and Competitive Momentum The ALS pipeline in 2025 is driven by a multi-pronged approach, including gene silencing, neuroprotection, immunomodulation, and cellular therapies. Key areas of innovation include: Gene-Targeted Therapies - Biogen’s Tofersen, an antisense oligonucleotide (ASO) targeting SOD1 mutations, has shown promise in reducing SOD1 protein levels and slowing disease progression in familial ALS. It represents the first real attempt at targeting a genetic driver in ALS with precision. - Another gene-targeted agent, BIIB100, is in early-phase trials to treat C9orf72-associated ALS, a common cause of familial and sporadic cases. These therapies are designed to address root-cause mutations and could revolutionize ALS management in genetically stratified patients. Neuroprotective and Combination Therapies - AMX0035, a fixed-dose combination of sodium phenylbutyrate and taurursodiol, showed significant survival benefit and slower functional decline in late-phase trials. With a favorable safety profile and oral administration, it’s emerging as a strong candidate for broad use across ALS populations. - Other agents in this class aim to support mitochondrial function, reduce ER stress, or modulate excitotoxicity-core mechanisms implicated in ALS pathogenesis. Immuno-inflammatory Modulators - Masitinib, a tyrosine kinase inhibitor, targets neuroinflammation and microglial activation. Now in Phase III, it could become the first oral immune-modulating agent with broad applicability in ALS if efficacy is confirmed. - Avonex (interferon-beta) and other repurposed immune therapies are being evaluated to address inflammatory aspects of ALS, particularly in patients showing elevated biomarkers of immune activation. Stem Cell and Cellular Approaches Stem cell infusions, particularly mesenchymal stem cells (MSCs), are under evaluation for their potential to protect or repair damaged motor neurons. These approaches remain experimental but are gaining attention for their regenerative promise. 🔍 Competitive Intelligence – Descriptive Landscape Tofersen is leading the field in genetically targeted therapy for SOD1-mutated ALS. Its ability to slow functional decline and reduce neurofilament biomarkers puts it ahead in terms of precision and biological targeting. In the broader ALS population, AMX0035 offers a disease-modifying option with strong survival data and ease of use. It stands out as the first combination therapy likely to gain global traction for sporadic ALS. Masitinib, still under evaluation, represents a promising oral option for patients with inflammatory phenotypes. Its oral route and broad immunologic targeting could position it as a foundational therapy if efficacy and tolerability are confirmed. Meanwhile, stem cell therapies and early-stage gene-editing solutions such as BIIB100 are advancing with hopes of establishing novel, durable interventions. These will likely serve as adjuncts or niche solutions depending on patient genotype and disease stage. 🎯 Target Opportunity Profile (TOP): What Success Looks Like For any new therapy to thrive in the ALS market, it must deliver across a range of clinical and strategic attributes: - Clinical Efficacy: Demonstrated improvement in survival and/or functional endpoints such as ALSFRS-R or time to tracheostomy. - Safety and Tolerability: Minimal systemic side effects, especially critical in a fragile patient population with reduced physiological reserve. - Mechanism Differentiation: Gene-targeted therapies should address known mutations; other candidates must tackle neuroinflammation, oxidative stress, or neuronal energy failure. - Ease of Administration: Oral or infrequent dosing regimens are preferred. Intrathecal administration may be acceptable for precision therapies like ASOs. - Payer & Access Considerations: Orphan status, cost-effectiveness, and robust real-world data will be vital for widespread adoption and reimbursement. - Patient Stratification: Success depends on biomarker-driven selection, especially for genetic or inflammatory subtype-specific therapies. Ask for Customized CI Service as per Your Business Requirement: https://www.datamintelligence.com/strategic-insights/ci/amyotrophic-lateral-sclerosis-als 🔮 Market Outlook: 2025 and Beyond The years ahead will witness critical inflection points: - Tofersen is expected to reshape treatment in genetically confirmed SOD1-ALS, catalyzing greater adoption of genetic testing. - AMX0035 will likely establish itself as the first broadly used disease-modifying therapy. - Masitinib may expand treatment choices for sporadic ALS if it confirms efficacy in ongoing trials. - Gene therapies and cell-based innovations will push the envelope toward true disease reversal or stabilization. ✅ Conclusion After decades of frustration, the ALS field is entering a breakthrough era. New therapies are moving beyond symptomatic support to targeting the underlying biology of the disease. From gene silencing to neuroprotection and immunomodulation, the coming years hold unprecedented potential to extend and improve lives. For patients, caregivers, and industry stakeholders, ALS is no longer a closed chapter-it’s a rapidly unfolding story of innovation. Read Related CI Reports: 1. Focal Segmental Glomerulosclerosis | Competitive Intelligence 2. Vitiligo | Competitive Intelligence Sai Kumar DataM Intelligence 4market Research LLP +1 877-441-4866 sai.k@datamintelligence.com Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Phase 3Drug ApprovalOrphan DrugOligonucleotideImmunotherapy

05 Jun 2025

·firstwordpharma.com

Despite study miss, Corcept makes case for ALS drug's survival benefit

Corcept Therapeutics shared long-term survival data at the European Network to Cure ALS (ENCALS) meeting Thursday that further bolster the firm's argument that its experimental amyotrophic lateral sclerosis (ALS) therapy can improve survival — despite failing to slow functional decline. The drugmaker had previously reported that in the Phase II DAZALS trial, dazucorilant did not meet its primary endpoint of achieving a significant difference on the ALS Functional Rating Scale-Revised (ALSFRS-R) scale compared with placebo. At the 24-week readout, however, Corcept noted that for the secondary measure of overall survival, none of the 83 patients who received the 300-mg daily dose of dazucorilant had died, while there were five deaths in the 82-patient placebo group. DAZALS randomised 249 patients with ALS to receive high-dose dazucorilant, a low 150-mg dose of the drug, or placebo. After the 24-week study period, participants could enrol in a long-term extension study and receive the 300-mg dose. Dazucorilant is a selective cortisol modulator that binds to the glucocorticoid receptor, but does not bind to the body’s other hormone receptors.Corcept revealed the results of an exploratory one-year survival analysis at ENCALS, which found that patients who received the high dose of dazucorilant lived significantly longer than those on placebo (hazard ratio = 0.16).Of the 83 patients taking 300-mg dazucorilant, 14 (17%) died after one year. Corcept also followed 18 patients who had received placebo during the 24-week study period and did not participate in the extension, and thus never received the experimental treatment; there were seven (39%) deaths at the one-year mark.Corcept said that dazucorilant's safety profile is "acceptable," with the most common adverse event (AE) being a mild-to-moderate, dose-related, transient abdominal pain. The company added that the rate of severe and serious AEs was similar between patients who received the experimental drug and those on placebo."Medications that can extend life for patients with ALS are urgently needed. We are working with regulatory authorities to determine the optimal path for advancing dazucorilant,” said Chief Development Officer Bill Guyer. The company said it is specifically seeking guidance from US and European regulators on a path forward. Current FDA-approved ALS treatments in the US include Mitsubishi Tanabe's Radicava (edaravone) and Biogen's Qalsody (tofersen). However, the field has been riddled with setbacks, most notably Amylyx Pharmaceuticals' decision last year to withdraw Relyvrio (sodium phenylbutyrate/taurursodiol) from the market after disappointing confirmatory trial results. For more on what's going on in the ALS space, see – Spotlight On: Trends shaping the future of ALS treatment and KOL Views Q&A: Expert views on new presentations for existing and would-be drugs in ALS.

Phase 2Clinical Result

08 May 2025

·www.amylyx.com

Amylyx Pharmaceuticals Reports First Quarter 2025 Financial Results

Pivotal Phase 3 LUCIDITY trial of avexitide, a potential first-in-class GLP-1 receptor antagonist with FDA breakthrough therapy designation, underway in post-bariatric hypoglycemia (PBH); completion of recruitment expected in 2025, with topline data anticipated in first half of 2026 Data through Week 48 from the ongoing Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome to be presented at the Joint Congress of the European Society for Pediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) taking place from May 10-13 Phase 1 LUMINA trial of AMX0114, an investigational, potent antisense oligonucleotide targeting knockdown of calpain-2, underway in people living with amyotrophic lateral sclerosis (ALS); early cohort data expected in 2025 Cash, cash equivalents, and marketable securities of $204.1 million as of March 31, 2025; cash runway expected through the end of 2026 Management to host conference call and webcast today at 8:00 a.m. Eastern Time CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial and business results for the first quarter ended March 31, 2025. “During the first quarter, we made continued progress across our pipeline, including strong clinical execution of our pivotal Phase 3 LUCIDITY trial of avexitide in post-bariatric hypoglycemia. We remain focused on avexitide and continue to expect completion of enrollment in 2025 and topline data in the first half of 2026,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. “We also made important progress in dosing the first patient in our Phase 1 LUMINA trial of AMX0114 in ALS, with early cohort data expected later this year. Additionally, we look forward to presenting new, long-term data from the Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome in the coming days, and the topline data from the Phase 2b portion of the Phase 2b/3 ORION trial of AMX0035 are expected in the third quarter. With our momentum so far this year and current anticipated cash runway through the end of 2026, we remain confident in achieving our current goals and look forward to our upcoming milestones.” First Quarter and Recent Updates: Amylyx began dosing in the pivotal Phase 3 LUCIDITY clinical trial of avexitide, a glucagon-like peptide-1 (GLP-1) receptor antagonist with FDA Breakthrough Designation, for the treatment of post-bariatric hypoglycemia (PBH). In April 2025, the Company dosed the first participant for LUCIDITY, an approximately 75-participant, randomized, double-blind, placebo-controlled clinical trial designed to evaluate efficacy and safety of avexitide in participants with PBH following Roux-en-Y gastric bypass (RYGB) surgery at approximately 20 sites in the U.S. Amylyx presented the design of LUCIDITY in December 2024, and the poster is available on the “Publications” page of the Amylyx website. Avexitide has been previously evaluated in five PBH clinical trials showing consistent, dose-dependent effects, including statistically significant reductions in hypoglycemic events. Avexitide was generally well-tolerated, with a favorable safety profile replicated across clinical trials. With these consistent data, Amylyx believes avexitide has the potential to become the first approved therapy for PBH. Amylyx began dosing in the Phase 1 LUMINA clinical trial of AMX0114, an investigational antisense oligonucleotide (ASO) targeting knockdown of calpain-2, for people living with amyotrophic lateral sclerosis (ALS).In April 2025, the Company dosed the first participant for LUMINA, a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose clinical trial designed to evaluate the safety and biological activity of AMX0114. The trial will also assess ALS biomarkers, including changes from baseline in neurofilament light (NfL) levels. Approximately 48 participants will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks, for up to four doses. Amylyx closed its underwritten public offering of 19.7 million shares of its common stock in January 2025. The net proceeds to Amylyx from this offering were approximately $65.5 million, extending the company’s expected cash runway through the end of 2026. Upcoming Expected Milestones: Completion of enrollment for the pivotal Phase 3 LUCIDITY clinical trial of avexitide in PBH expected in 2025, with a data readout anticipated in the first half of 2026 and, if approved, commercial launch anticipated in 2027. LUCIDITY is evaluating the FDA-agreed-upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16 and has similar inclusion and exclusion criteria to previous Phase 2 trials of avexitide in PBH. Amylyx will share Week 48 data from the ongoing Phase 2 HELIOS trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in Wolfram syndrome at the Joint Congress of the European Society for Pediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) on May 10-13 in Copenhagen, Denmark. The poster presentation will be made available on the “Presentations” tab of the Amylyx website on Monday, May 12, 2025. Data from participants at Week 48 and ongoing discussions with the FDA will inform the design of a Phase 3 trial of AMX0035 in Wolfram syndrome. Amylyx plans to present additional data from the Phase 2 PREVENT and Phase 2b trials of avexitide in PBH at the Endocrine Society’s annual meeting (ENDO) 2025 on July 12-15 in San Francisco, CA. The presentations will be made available on the “Presentations” tab of the Amylyx website following the conclusion of the sessions in accordance with ENDO’s embargo policy. Unblinded analysis of the Phase 2b portion of the Phase 2b/3 ORION trial evaluating AMX0035 for progressive supranuclear palsy (PSP) expected in the third quarter of 2025.ORION is an operationally seamless Phase 2b/3 clinical trial in people living with PSP. The Phase 2b portion was fully enrolled in January 2025 with a total of 139 participants randomized. Efficacy and safety Phase 2b data from an unblinded analysis when all participants have reached Week 24 will be used to inform a go/no-go decision on the Phase 3 portion of the trial. Early cohort data from the Phase 1 LUMINA clinical trial of AMX0114 in ALS expected in 2025. LUMINA clinical trial sites were activated in Canada in the beginning of 2025 and are recruiting participants. Amylyx is working to open U.S. sites for screening, enrollment, and dosing. Financial Results for the First Quarter Ended March 31, 2025 R&D Expenses: Research and development expenses for the first quarter of 2025 were $22.1 million, compared to $36.6 million for the same period in 2024. The decrease was primarily due to a decrease in spending on AMX0035 for the treatment of ALS, payroll and personnel-related costs, and a decrease in preclinical development activities. Research and development expenses include $1.8 million of stock-based compensation expense for the quarter, compared to $2.7 million of stock-based compensation expense for the same period in 2024. SG&A Expenses: Selling, general, and administrative expenses for the first quarter of 2025 were $15.7 million, compared to $57.8 million for the same period in 2024. The decrease was primarily due to a decrease in payroll and personnel-related costs and a decrease in consulting, professional services, and other expenses. Selling, general, and administrative expenses include $5.0 million of stock-based compensation expense for the quarter, compared to $7.2 million for the same period in 2024. Net Loss: Net loss for the three months ended March 31, 2025 was $35.9 million, or $0.42 per share, compared to net loss of $118.8 million, or $1.75 per share for the same period in 2024. Cash Position: Cash, cash equivalents, and marketable securities were $204.1 million at March 31, 2025, compared to $176.5 million at December 31, 2024. Based on its current operating plans, Amylyx expects its cash runway to be through the end of 2026. The cash position at March 31, 2025 reflects payments of $6.0 million in the first quarter of 2025 related to product rebates and the settlement of purchase commitments for AMX0035 that were established prior to the voluntary discontinuation of sales of RELYVRIO®/ALBRIOZA™ in April 2024. We recorded $1.4M of expense in the first quarter of 2025 related to these payments, with the remaining expense recorded in prior periods. The residual cash obligations related to the discontinuation of RELYVRIO/ALBRIOZA are $3.1 million, and the Company expects they will be paid through 2025. Investor Conference Call Information Amylyx’ management team will host a conference call today, May 8, 2025, at 8:00 a.m. ET to discuss financial results and provide an update on the business. To access the conference call, please dial +1 (800)-836-8184 (U.S. & Canada) or +1 (646)-357-8785 (international) at least 10 minutes prior to the start time and ask to be joined into the Amylyx Pharmaceuticals call. A live audio webcast of the call will be available under “Events and Presentations” in the Investor section of the Company’s website, https://investors.amylyx.com/events-presentations. The webcast will be archived and available for replay for 90 days following the event. Available Information We periodically provide other information for investors on our corporate website, https://amylyx.com, and our investor relations website, https://investors.amylyx.com. This includes press releases and other information about financial performance, information on corporate governance, and details related to our annual meeting of stockholders. We intend to use our website as a means of disclosing material non-public information and for complying with our disclosure obligations under Regulation FD. Accordingly, investors should monitor our website, in addition to following the Company’s press releases, SEC filings, and public conference calls and webcasts. About Avexitide Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist that has been evaluated in five Phase 1 and Phase 2 clinical trials for post-bariatric hypoglycemia (PBH) and has also been studied in congenital hyperinsulinism (HI). The U.S. Food and Drug Administration (FDA) has granted avexitide Breakthrough Therapy Designation for both indications, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI). Avexitide is designed to bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the effect of GLP-1 to mitigate hypoglycemia by decreasing insulin secretion and stabilizing blood glucose levels. In PBH, excessive GLP-1 can lead to the hypersecretion of insulin and subsequent debilitating hypoglycemic events. In two Phase 2 PBH clinical trials, avexitide demonstrated highly statistically significant reductions in hypoglycemic events. These events can lead to autonomic and neuroglycopenic symptoms that can have a devastating impact on daily living. About Post-Bariatric Hypoglycemia (PBH) Post-bariatric hypoglycemia (PBH) is a condition that affects approximately 8% of people in the U.S., or approximately 160,000 people, who have undergone the two most common types of bariatric surgery, which include sleeve gastrectomy and Roux-en-Y gastric bypass (RYGB). PBH is thought to be caused by an excessive glucagon-like peptide-1 (GLP-1) response leading to hypoglycemia and impaired quality of life. PBH can cause debilitating hypoglycemic events associated with inadequate supply of glucose to the brain, known as neuroglycopenia. Clinical manifestations can include impaired cognition, loss of consciousness, and seizures. PBH is also associated with a high degree of disability that can result in major disruptions to independent living. There are no approved therapies for PBH. About the LUCIDITY Trial LUCIDITY (NCT06747468) is an approximately 75-participant, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following Roux-en-Y gastric bypass (RYGB) surgery. The Phase 3 trial will be conducted at approximately 20 sites in the U.S. Participants will be randomized 3:2 to receive either 90 mg of avexitide subcutaneously once daily or placebo. The trial includes an up to six-week screening period, including a three-week run-in period, and a 16-week double-blind treatment period. Participants who complete the double-blind period will be eligible to enter an open-label extension (OLE) period with a duration of 32 weeks. The primary efficacy objective of LUCIDITY will evaluate the FDA-agreed upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability will also be evaluated. About Amylyx Pharmaceuticals At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on three investigational therapies across several neurodegenerative and endocrine diseases in which we believe they can make the greatest impact. For more information, visit amylyx.com and follow us on LinkedIn and X. For investors, please visit investors.amylyx.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx’ expectations regarding: the potential of avexitide as a treatment for PBH; and expectations regarding the timing for recruitment completion and topline data readout of the Phase 3 LUCIDITY trial of avexitide in PBH and regarding the upcoming presentation of additional data from the Phase 2 PREVENT and Phase 2b trials of avexitide; the potential of AMX0035 (sodium phenylbutyrate and taurursodiol) as a treatment for Wolfram syndrome and PSP or other neurodegenerative diseases; expectations regarding the timing of the announcement of Week 48 data from the Phase 2 HELIOS trial of AMX0035 for the treatment of Wolfram syndrome; planned discussions with the FDA related to AMX0035 for the treatment of Wolfram syndrome; expectations regarding the timing of the announcement of interim results from the Company’s Phase 2b/3 ORION trial of AMX0035 for the treatment of PSP; the potential for AMX0114 as a treatment for ALS and the expected timeline for data readout of the Phase 1 LUMINA clinical trial; and Amylyx’ expectations regarding its financial performance, cash runway and longer-term strategy. Any forward-looking statements in this press release and related comments in the Company's earnings conference call are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’ program development activities; Amylyx’ ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; Amylyx’ ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx’ operations, as well as the risks and uncertainties set forth in Amylyx’ United States Securities and Exchange Commission (SEC) filings, including Amylyx’ Annual Report on Form 10-K for the year ended December 31, 2024, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. AMYLYX PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED BALANCE SHEETS UNAUDITED (in thousands) March 31, 2025 December 31, 2024 Assets Cash, cash equivalents and marketable securities $ 204,068 $ 176,501 Accounts receivable, net 300 447 Prepaid expenses and other current assets 11,776 12,484 Other assets 3,532 4,202 Total assets $ 219,676 $ 193,634 Liabilities and Stockholders’ Equity Accounts payable and accrued expenses $ 16,877 $ 26,888 Other liabilities 1,382 1,981 Total liabilities 18,259 28,869 Stockholders’ equity 201,417 164,765 Total liabilities and stockholders' equity $ 219,676 $ 193,634 AMYLYX PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS UNAUDITED (in thousands, except share and per share data) Three Months Ended March 31, 2025 2024 Product revenue, net $ — $ 88,643 Operating expenses: Cost of sales — 5,945 Cost of sales - inventory impairment and loss on firm purchase commitments — 110,461 Research and development 22,119 36,608 Selling, general and administrative 15,684 57,759 Total operating expenses 37,803 210,773 Loss from operations (37,803 ) (122,130 ) Other income, net 1,896 3,579 Loss before income taxes (35,907 ) (118,551 ) Provision for income taxes — 242 Net loss $ (35,907 ) $ (118,793 ) Net loss per share — basic and diluted $ (0.42 ) $ (1.75 ) Weighted-average shares used in computing net loss per share — basic and diluted 85,697,108 67,854,356 Media Amylyx Media Team +1 (857) 320-6191 amylyxmediateam@amylyx.com Investors Lindsey Allen Amylyx Pharmaceuticals, Inc. +1 (857) 320-6244 Investors@amylyx.com

Phase 2Phase 3Breakthrough TherapyPhase 1Clinical Result

100 Deals associated with Tauroursodeoxycholate sodium

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KEGG	Wiki	ATC	Drug Bank
-	Tauroursodeoxycholate sodium	-	DB08834

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Cholelithiasis	China	Bruschettini Srl	25 Jul 2006

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cholestatic liver disease	Phase 3	China	Beijing Quanfu Kangjian Medicine Information Consulting Co., Ltd.	01 Aug 2009
Primary Biliary Cholangitis	Phase 3	China	Beijing Quanfu Kangjian Medicine Information Consulting Co., Ltd.	01 Aug 2009
Liver Diseases	Phase 2	-	Mitsubishi Tanabe Pharma Corp.	-
Fibrosis, Liver	Preclinical	China	Lanzhou University	09 Apr 2025
Colitis, Ulcerative	Preclinical	China	Xi'an Jiaotong University	30 May 2024
Colitis, Ulcerative	Preclinical	China	University of Macau	30 May 2024
Colitis, Ulcerative	Preclinical	China	Zhejiang Chinese Medical University	30 May 2024

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02218619 (CTgov)	Phase 2	Diabetes Mellitus, Type 1	20	Sugar Pill (placebo) (Sugar Pill (Placebo))	sqlkycxjcv(dkohwrefls) = xuzjsyoutj usbazjsdiw (cvmlkqxiki, 0.11) View more	-	05 Jun 2025
				Tauroursodeoxycholic Acid (TUDCA) (Taurourodeoxycholic Acid (TUDCA))	sqlkycxjcv(dkohwrefls) = xwnsqyprxp usbazjsdiw (cvmlkqxiki, 0.10) View more
NCT03423121 (CTgov)	Phase 1/2	Multiple Sclerosis, Chronic Progressive	59	Tauroursodeoxycholic Acid (TUDCA Treatment)	xoqttgbsaa = ecyetenfdb pjgxumsgnm (qkdyhmwglx, bnnwmfqdgu - zthbtgswmy) View more	-	03 May 2023
				Placebo oral capsule (Placebo Oral Capsule)	xoqttgbsaa = pmxvqtwdsa pjgxumsgnm (qkdyhmwglx, naydekdnda - aaqdnivdnq) View more
NCT01855360 (CTgov)	Phase 1/2	Transthyretin Amyloid Cardiomyopathy \| Senile cardiac amyloidosis	40	Doxycycline+Tauroursodeoxycholic Acid	wrxddjdkxf(ppcgqgsywo) = apkydadzpw dnfwqyetfj (aumfcanxtf, .9) View more	-	04 Dec 2020
NCT01877551 (CTgov)	Not Applicable	Insulin Resistance	27	Tauroursodeoxycholic acid (Tauroursodeoxycholic Acid)	layogmqjxh(yzvrkbshtq) = kujujnutiw ftxslotics (cuizjqqpvp, 11) View more	-	27 Jan 2020
				Placebo tablet (Placebo)	layogmqjxh(yzvrkbshtq) = uwpsaepwnx ftxslotics (cuizjqqpvp, 7) View more
NCT00771901 (TUDCA/PBA, CTgov)	Not Applicable	Insulin Resistance \| Diabetes Mellitus \| Obesity	101	placebo (Placebo)	jzfgemttgj(faeadkutbc) = fzogsoctqy xxphfzhqud (qnpwhogdzk, 7) View more	-	29 May 2018
				tauroursodeoxycholic acid (Tauroursodeoxycholic Acid)	jzfgemttgj(faeadkutbc) = loniblaiix xxphfzhqud (qnpwhogdzk, 8) View more
NCT00877604 (Pubmed \| Eur J Neurol)	Phase 2	Amyotrophic Lateral Sclerosis	34	Tauroursodeoxycholic acid	vkpbejpobl(rjgivzhies) = rixjdohapg nmqetdgryq (bxwpkyyulj )	Positive	01 Jan 2016
				Placebo	vkpbejpobl(rjgivzhies) = chpunrecjz nmqetdgryq (bxwpkyyulj )
NCT00877604 (TUDCA-ALS, CTgov)	Phase 2	Amyotrophic Lateral Sclerosis	34	tauroursodeoxycholic acid (TUDCA) (TUDCA)	rreujzhcmi = pkougpuics bnsupiwogs (pxzudiqquz, qarvoofgmh - beqdmwmbbf) View more	-	26 Nov 2014
				Placebo (Placebo)	rreujzhcmi = lyvimxfdgp bnsupiwogs (pxzudiqquz, ehsawzzhdx - bngaradcot) View more

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