Gallium GA-68 Gozetotide

In March 2025, the global pharmaceutical sector witnessed a surge of innovation where technology deeply integrates with clinical needs, spanning key areas such as gene therapy, antibiotic breakthroughs, targeted therapies for rare diseases, and vaccine upgrades. From Fitusiran (the world's first RNAi therapy for hemophilia) to Gepotidacin (a novel antibiotic combating drug-resistant strains), from Diazoxide Choline (the first targeted drug for Prader-Willi Syndrome) to the quadrivalent influenza vaccine (covering a broader range of viral subtypes), these new drugs not only break through the limitations of traditional treatments but also redefine disease management paradigms through precision targeting, broad-spectrum antiviral strategies, and the cost-effectiveness of biosimilars. 1. Fitusiran On March 28, 2025, Fitusiran received approval from the U.S. Food and Drug Administration (FDA) for the first time in the United States, becoming the first RNA interference (RNAi) drug to treat hemophilia A and B by inhibiting antithrombin (AT). This milestone marks a groundbreaking advancement of gene therapy technology in the field of blood disorders. The FDA's approval was based on its efficacy in significantly reducing bleeding events in clinical trials, supported by long-term safety data. In addition to the U.S., Fitusiran has also been granted priority review status by China's National Medical Products Administration (NMPA), accelerating its commercialization in key global markets. Developed by Alnylam Pharmaceuticals, Fitusiran's R&D journey exemplifies the deep collaboration of multinational pharmaceutical companies. Alnylam partnered strategically with Sanofi and its subsidiary Genzyme Corp. to jointly advance the drug's clinical trials, optimize production processes, and facilitate global registration. This collaboration leveraged Alnylam's expertise in RNAi technology and Sanofi's extensive experience in treating blood disorders. Moreover, during the R&D process, Fitusiran, due to its innovation in targeting a rare disease, successively received the FDA's "Fast Track" designation and "Orphan Drug" status, further highlighting its value in addressing unmet medical needs. Fitusiran specifically targets antithrombin mRNA via RNAi technology, blocking its translation process to reduce antithrombin levels (by approximately 80%-90%). Antithrombin, a key natural anticoagulant protein in the blood, can overly inhibit the activity of residual clotting factors (such as VIII or IX), exacerbating the bleeding tendency in hemophilia patients. By removing this inhibition, Fitusiran allows the residual clotting factors in patients to more effectively participate in hemostasis, significantly reducing spontaneous or traumatic bleeding events. Technically, the drug employs a GalNAc (N-acetylgalactosamine)-conjugated delivery system to precisely target the liver (the primary organ for antithrombin synthesis), combined with 2'-O-methylation and locked nucleic acid (LNA) chemical modifications to enhance stability, reduce off-target effects, and minimize immunogenicity, achieving a long-acting treatment with monthly subcutaneous injections. In addition to the U.S. and China, Fitusiran is also in the accelerated review stage in major markets such as the EU and Japan, with an expectation to cover major global regions by the end of 2025. Its differentiated advantages—eliminating the need for frequent clotting factor infusions, applicable to all hemophilia subtypes and inhibitor patients—make it a significant complement to existing therapies (such as factor replacement therapy and the anticoagulant Emicizumab). Furthermore, the R&D team is exploring the potential applications of Fitusiran in a broader range of indications, including von Willebrand disease, perioperative clotting management, and prevention of bleeding risks following trauma or surgery, further expanding its clinical value. 2.Technetium Tc 99m Trofolastat Technetium Tc 99m Trofolastat is an innovative diagnostic radiopharmaceutical belonging to the category of radionuclide-conjugated drugs, primarily used for imaging diagnosis of prostate-specific membrane antigen (PSMA)-positive prostate cancer. The drug precisely labels tumor lesions by binding to PSMA, a protein highly expressed on the surface of prostate cancer cells, aiding doctors in locating primary tumors, assessing metastatic distribution, and monitoring treatment responses. Its core advantage lies in the use of the radioactive isotope technetium-99m (Tc-99m), which has a short half-life (approximately 6 hours) and high imaging sensitivity, providing clear tumor images while ensuring patient safety. On March 27, 2025, the drug was first approved in the UK, becoming the world's first radiopharmaceutical diagnostic agent based on PSMA-targeting technology, marking a significant breakthrough in prostate cancer imaging diagnostics. The mechanism of action of Trofolastat is based on the targeting of PSMA. PSMA is a membrane protein overexpressed on the surface of prostate cancer cells, particularly in metastatic or recurrent tumors. The drug specifically binds to PSMA through its chemical structure, then conjugates with Tc-99m to form a radioactive tracer. After intravenous injection into the body, PSMA-positive tumor cells take up and accumulate the drug, generating high-resolution tumor distribution maps through gamma camera imaging. Compared to traditional imaging techniques (such as CT or MRI), Trofolastat can detect PSMA-positive lesions earlier and more sensitively, especially in the assessment of bone metastases or lymph node metastases. Its short half-life reduces patient radiation exposure risks, and the widespread global availability of Tc-99m (due to its mature infrastructure) further enhances the convenience of clinical application. The drug was led in development by Progenics Pharmaceuticals, Inc., with its R&D journey exemplifying the synergistic effects of multi-party collaboration. Progenics initially discovered the targeting potential of PSMA and designed the molecular structure of Trofolastat. Subsequently, Lantheus Medical Imaging, Inc. (now part of Bracco Imaging) became a key partner, responsible for optimizing drug production, global registration, and commercial promotion, especially in the European and American markets. Additionally, the R&D team leveraged Progenics' technical accumulation in the PSMA field (such as the development experience of the therapeutic drug 177Lu-PSMA-617) to further validate the feasibility of PSMA targeting in diagnostics. Despite Progenics' acquisition by Bracco in 2023, the R&D team continued to optimize drug performance, ultimately leading to its first approval in the UK, filling the gap in PSMA-targeted diagnostic drugs. The R&D progress of Trofolastat has been rapid, entering Phase III clinical trials by 2025 and validating its diagnostic accuracy through key trials. On March 27, 2025, the UK Medicines and Healthcare products Regulatory Agency (MHRA) approved it for the diagnosis of PSMA-positive prostate cancer, making it the first PSMA-targeted radiopharmaceutical diagnostic agent approved in Europe. The current indication focuses on the localization and staging of PSMA-positive prostate cancer, with potential future expansion to monitoring the recurrence of metastatic prostate cancer and assessing treatment effectiveness. Although similar drugs (such as Ga-68 PSMA-11) have been approved in the U.S., Trofolastat's widespread availability of Tc-99m (used in 90% of global nuclear medicine imaging) and longer imaging window (suitable for primary care hospitals) demonstrate significant market competitiveness. 3.Diazoxide Choline Diazoxide Choline (brand name: VYKAT XR) is a small-molecule chemical drug that acts as a potassium channel agonist, reducing appetite by modulating the function of potassium ion channels in cell membranes. Developed by Essentialis, Inc. and subsequently advanced to the clinical stage by Soleno Therapeutics, Inc., it was first approved in the United States on March 26, 2025, for the treatment of hyperphagia and Prader-Willi Syndrome (PWS). PWS is a genetic disorder where patients often experience extreme hunger and an increased risk of obesity due to hypothalamic dysfunction. Diazoxide Choline, by targeting the appetite regulation center, becomes the first drug to address the core pathological mechanisms of this disease. Its extended-release formulation (VYKAT XR) is designed to allow once-daily dosing, significantly improving patient adherence. The mechanism of action of Diazoxide Choline is based on the activation of potassium ions (K⁺) channels. The drug activates ATP-sensitive potassium channels (KATP), inhibiting the secretion of glucagon by pancreatic α-cells while modulating the hypothalamic appetite regulation pathway to reduce hunger and suppress binge eating behavior. In patients with Prader-Willi Syndrome, hypothalamic dysfunction leads to abnormally elevated ghrelin levels and defective leptin signaling, exacerbating uncontrolled appetite. Diazoxide Choline restores the hypothalamus's regulation of energy balance through potassium channel-mediated signaling pathways, thereby reducing the impulse to overeat. Additionally, the drug has no significant impact on insulin secretion, reducing the risk of side effects such as hypoglycemia and ensuring higher safety. The drug's development began with Essentialis, Inc., and was later taken over by Soleno Therapeutics to advance it to the clinical stage. During the R&D process, Diazoxide Choline received several special review designations from the FDA, including Fast Track status, Breakthrough Therapy designation, and Orphan Drug status (both in the U.S. and EU), highlighting its importance in treating the rare disease PWS. Additionally, the drug underwent priority review by the FDA, accelerating the approval process. Although initially explored for indications such as hypertriglyceridemia, its breakthrough efficacy in PWS became the core R&D direction. Phase III clinical trial data showed that Diazoxide Choline significantly reduced binge eating behavior, weight gain, and hospitalization rates in PWS patients. In the trial, patients receiving treatment experienced a reduction in hunger scores by over 50%, with no serious drug-related adverse events reported. After its first approval in March 2025, the indication focused on hyperphagia and Prader-Willi Syndrome, with potential future expansion to other hypothalamic dysfunction-related appetite disorders. Additionally, the R&D team is exploring its potential to improve cognitive function and behavioral symptoms in PWS patients, further enhancing the drug's comprehensive therapeutic value. 4. Suraxavir Marboxil Suraxavir Marboxil is a novel small-molecule antiviral drug jointly developed by Ginkgo Pharmaceutical in collaboration with Kerui and Qingfeng Pharmaceutical. It was approved for marketing in China on March 25, 2025, becoming the first broad-spectrum anti-influenza drug targeting both influenza A and B virus infections. Its mechanism of action involves targeting key proteins of the influenza virus (presumably neuraminidase or related targets), thereby inhibiting viral replication and release. This mechanism helps to shorten the duration of illness and reduce the risk of severe complications. The approval of Suraxavir Marboxil fills a critical gap in China's broad-spectrum anti-influenza drug market and holds significant value in addressing seasonal flu outbreaks and potential pandemic threats. Its formulation (likely oral) and convenient administration make it a valuable tool for influenza treatment and prevention. Suraxavir Marboxil’s antiviral mechanism is based on the inhibition of essential viral proteins. Influenza viruses rely on neuraminidase (NA) to release newly formed viral particles from host cells. This drug is believed to bind selectively and inhibit the activity of neuraminidase, thus preventing viral spread. Compared to traditional neuraminidase inhibitors (such as oseltamivir), Suraxavir Marboxil may offer broader antiviral coverage against both influenza A and B, whereas traditional drugs typically focus on influenza A. Additionally, its molecular structure has likely been optimized to enhance efficacy against drug-resistant strains and minimize side effects. Preclinical studies have demonstrated its rapid onset, strong antiviral activity, and low toxicity in both in vitro and animal models. Clinical trial data show that Suraxavir Marboxil significantly outperforms placebo in reducing the duration of influenza symptoms and lowering the incidence of complications (such as pneumonia), with proven efficacy against both influenza A and B viruses. It has exhibited a favorable safety profile, with no reports of serious drug-related adverse events, making it suitable for children, adults, and the elderly. The current approved indication focuses on influenza A and B infections, with potential future expansion to prophylactic use in high-risk populations (e.g., immunocompromised individuals or patients with chronic illnesses). Moreover, the development team is exploring its applicability in patients with impaired liver function to expand its coverage across broader patient populations. 5. Gepotidacin Gepotidacin is a novel small-molecule antibiotic developed by GlaxoSmithKline (GSK), which received its first approval in the United States on March 25, 2025. It is indicated for the treatment of bacterial infections caused by susceptible organisms, including urinary tract infections (such as acute cystitis and gonococcal urethritis). Gepotidacin features a unique mechanism of action—dual inhibition of bacterial DNA gyrase and topoisomerase IV—which blocks bacterial DNA replication and repair, thereby rapidly eliminating pathogens. As the first approved antibiotic in the triazolopyrimidine class, Gepotidacin addresses treatment gaps caused by antibiotic resistance to traditional fluoroquinolones, especially against resistant Gram-positive and Gram-negative pathogens. Unlike conventional fluoroquinolones (e.g., levofloxacin), Gepotidacin targets both DNA gyrase (primarily in Gram-negative bacteria) and topoisomerase IV (primarily in Gram-positive bacteria), providing broad-spectrum antibacterial activity. This dual-targeted mechanism significantly reduces the risk of resistance, as bacteria must simultaneously mutate both targets to develop resistance—an event with very low probability. Preclinical studies have shown that Gepotidacin is active against multidrug-resistant organisms (such as ESBL-producing E. coli and MRSA) and lacks cross-resistance with existing antibiotics, offering a promising option for resistant infections. Its rapid bactericidal activity—reducing bacterial load within hours—also shortens the course of infection compared to traditional antibiotics. Gepotidacin's development was driven by GSK’s urgent need for novel antibiotics to address the global antibiotic resistance crisis. While no special regulatory designations (e.g., FDA Fast Track or Orphan Drug status) have been mentioned, its timely approval in 2025 was supported by positive Phase III clinical trial data and the FDA’s commitment to fostering innovation in anti-infective drug development. GSK advanced clinical trials in collaboration with global partners (e.g., Atos SE in Europe), evaluating its potential in treating complex infections, such as complicated skin and soft tissue infections. Furthermore, the drug’s oral formulation and favorable tolerability profile (with fewer gastrointestinal side effects) enhance its clinical utility. Phase III trial results indicated that Gepotidacin's efficacy in treating acute bacterial cystitis was comparable to that of levofloxacin, with better tolerability. Although its current approved indication focuses on urinary tract infections, its broad-spectrum activity suggests potential future use in complicated urinary tract infections, gonorrhea, and skin and soft tissue infections. GSK is also investigating its use in treating multidrug-resistant infections—such as carbapenem-resistant Enterobacteriaceae (CRE)—to address the growing challenge of nosocomial infections. In the future, Gepotidacin could become the preferred alternative to traditional fluoroquinolones, particularly in regions with high rates of antibiotic resistance. 6. Encofosbuvir Encofosbuvir is a novel small-molecule direct-acting antiviral (DAA) developed jointly by Sunshine Lake Pharma and YiChang HEC ChangJiang Pharmaceutical. On March 25, 2025, it was approved in China as the first domestically produced pan-genotypic treatment for hepatitis C, indicated for the treatment of chronic hepatitis C virus (HCV) infection. The drug targets a key step in the HCV lifecycle, exerting potent antiviral effects by inhibiting viral RNA replication. Encofosbuvir specifically targets the HCV NS5B RNA-dependent RNA polymerase, a core enzyme involved in viral replication. By competitively binding to the active site of NS5B polymerase, it inhibits the elongation of viral RNA chains, thereby blocking viral RNA synthesis and assembly. As a nucleotide analog prodrug, Encofosbuvir is metabolized in vivo into its active form, which is incorporated into the viral RNA chain, resulting in premature chain termination and complete suppression of viral replication. Its pan-genotypic activity stems from its broad inhibitory effect across all major HCV genotypes (1–6), and it retains efficacy against common resistance-associated substitutions (e.g., Y93H). Compared to traditional interferon-based regimens, Encofosbuvir offers a significantly higher sustained virologic response (SVR) rate and lower resistance barrier, greatly improving treatment success while reducing adverse effects. Encofosbuvir was developed from Sunshine Lake Pharma’s antiviral drug platform, with clinical development and manufacturing supported by Humanwell Healthcare. During its development, the team optimized the drug design to align with the epidemiological characteristics of hepatitis C in China (primarily genotype 1b), and efficacy and safety were validated through multicenter Phase III clinical trials. Although no special review designations were noted, the drug’s swift approval in 2025 reflects regulatory support for domestic innovation in China. Clinical trials demonstrated that Encofosbuvir, either as monotherapy or in combination with ribavirin (RBV), achieved SVR12 rates exceeding 95% in patients with chronic hepatitis C, including those with cirrhosis. It showed a favorable safety profile, with common adverse events limited to mild fatigue or headache and no reports of serious hepatotoxicity or hematological toxicity. Currently approved for pan-genotypic chronic hepatitis C, future indications may include acute HCV infection and post-liver transplantation recurrence. Additionally, fixed-dose combinations with novel antivirals, such as NS3/4A protease inhibitors, are under exploration to further simplify treatment regimens and enhance adherence. The launch of Encofosbuvir not only provides Chinese hepatitis C patients with a cost-effective therapeutic option but also represents a major milestone in the localization of global antiviral drug development. Its pan-genotypic coverage and short-course regimen can significantly improve treatment accessibility, particularly in regions with limited healthcare resources. With its broader application, China is well-positioned to accelerate progress toward the World Health Organization (WHO)’s goal of eliminating viral hepatitis as a public health threat by 2030. Moreover, the successful development of Encofosbuvir offers technical insights for antiviral drug development targeting other RNA viruses such as hepatitis B and HIV, highlighting China's growing innovation capacity in the antiviral therapeutics sector. 7. Influenza Vaccine (Split Virion, Quadrivalent) The Influenza Vaccine (split virion, quadrivalent), developed by Anhui Longcom Biologic Pharmacy, is a multivalent prophylactic vaccine approved in China on March 18, 2025, for the prevention of influenza virus infection. Utilizing a virus-splitting technique, the vaccine separates influenza viruses into key antigenic components (such as hemagglutinin and neuraminidase), removes viral nucleic acids, and retains immunogenic elements to stimulate an immune response. It induces antibody production against influenza A subtypes H1N1 and H3N2, as well as B lineages Victoria and Yamagata. As a quadrivalent formulation, it offers broader coverage than trivalent vaccines, effectively addressing the high mutation rates of circulating influenza strains, particularly the predominant seasonal strains. Its approval fills a market gap for high-end domestically produced influenza vaccines in China, providing an affordable and effective option for seasonal influenza prevention. The vaccine's mechanism of action involves antigen presentation and activation of humoral immunity. The split virion fragments, especially hemagglutinin proteins, are recognized by the immune system and presented by dendritic cells to T lymphocytes, which in turn activate B cells to produce specific IgG antibodies. These antibodies bind to hemagglutinin on the virus surface, blocking receptor binding to host cells and marking viral particles for macrophage clearance. The vaccine also induces immune memory, offering long-term protection and reducing the risk of reinfection. The quadrivalent design, covering two A and two B influenza strains, significantly enhances cross-protection against variant strains, particularly in high-risk populations such as children, the elderly, and individuals with chronic illnesses. The splitting process ensures vaccine safety by eliminating the risk of infection associated with whole-virus vaccines, while preserving antigenic epitopes for robust immune responses. Due to the segmented genome structure of influenza viruses, frequent mutations occur annually, leading to hundreds of millions of global infections and hundreds of thousands of deaths. As a populous nation, China faces considerable challenges in influenza control. The mismatch between the traditional vaccine development cycle (approximately 6–8 months) and the rapid mutation rate of the virus highlights the urgent need for broad-spectrum, efficient vaccines. Anhui Longcom Biologic Pharmacy's quadrivalent influenza vaccine was developed in alignment with China's influenza epidemiology. By dynamically monitoring strain variations and selecting WHO-recommended dominant strains for seasonal formulation, the vaccine ensures optimal protection. Clinical trial data show that the vaccine achieves a seroprotection rate (antibody titer ≥40) exceeding 90% in both adults and children, providing significant protection against covered influenza strains. Reported adverse reactions are mostly mild, including injection site pain and low-grade fever, with no reports of severe allergic events. The current approved indication is prevention of influenza virus infection for individuals aged six months and older, with strong recommendations for high-risk groups such as healthcare workers, the elderly, and people with chronic conditions. Future directions may include emergency use during outbreaks of specific variant strains, or co-administration with COVID-19 vaccines to address co-infection risks. Additionally, the development team is investigating the vaccine's immunogenicity in immunocompromised patients to optimize vaccination strategies. 8. Ivarmacitinib Sulfate Ivarmacitinib sulfate is a selective Janus kinase (JAK) inhibitor jointly developed by Jiangsu Hengrui Pharmaceuticals and Reistone Biopharma. It was approved for marketing in China on March 18, 2025, for the treatment of ankylosing spondylitis. The drug functions by targeting and inhibiting JAK kinases and their downstream signaling pathways, thereby blocking the transmission of pro-inflammatory cytokines such as IL-6 and IL-23. This suppression mitigates the inflammatory responses triggered by excessive immune system activation. As the first domestically developed JAK inhibitor in China, Ivarmacitinib received accelerated approval through “breakthrough therapy” designation, addressing a significant unmet need in targeted therapy for ankylosing spondylitis. It offers a novel treatment option, especially for patients who are unresponsive or intolerant to traditional therapies like TNF-α inhibitors. The drug is also being investigated for other autoimmune conditions such as atopic dermatitis, alopecia areata, and ulcerative colitis, demonstrating broad clinical potential. The mechanism of action of Ivarmacitinib centers on inhibition of the JAK-STAT signaling pathway. JAK kinases are key mediators in cytokine signaling (e.g., IL-6, IL-17, IL-23), which are overexpressed in inflammatory autoimmune diseases such as ankylosing spondylitis and psoriatic arthritis. By selectively inhibiting JAK1/3 isoforms, Ivarmacitinib blocks signal transduction following cytokine-receptor binding, reduces the release of pro-inflammatory cytokines, and alleviates joint inflammation, skin damage, and systemic immune responses. During development, the research team optimized the molecular structure to enhance isoform selectivity, reduce off-target effects, and improve oral bioavailability, making it suitable for long-term treatment. As a domestically developed innovative drug, Ivarmacitinib responds to the urgent demand for effective and safe targeted therapies for autoimmune diseases. Supported by Hengrui’s robust R&D capabilities, the drug was rapidly translated from laboratory research to clinical application. Phase III clinical trial results showed that Ivarmacitinib significantly improved joint pain, morning stiffness, and functional impairment in patients with ankylosing spondylitis, with a favorable safety profile (the main adverse effects being mild elevation of liver enzymes and headache). Its currently approved indication is ankylosing spondylitis, with potential future indications including moderate-to-severe atopic dermatitis (now in Phase III), alopecia areata (Phase II data showing >50% hair regrowth rate), and ulcerative colitis. The research team is also exploring its efficacy in psoriatic arthritis and non-segmental vitiligo, aiming for integrated management of multisystem diseases through immune modulation. Notably, after early collaboration with Arcutis Biotherapeutics ended, Hengrui continued clinical development independently, underscoring its leadership in domestic innovation. Compared with existing JAK inhibitors, Ivarmacitinib offers several differentiating advantages: ·Higher JAK isoform selectivity, minimizing inhibition of JAK2 (which is involved in hematopoiesis) and thereby reducing risks of thrombosis and anemia; ·Longer half-life (~24 hours), allowing for once-daily dosing and improved patient adherence; ·Localized anti-inflammatory effect, targeting affected tissues (e.g., skin, joints) while minimizing systemic immunosuppressive side effects.These features make Ivarmacitinib particularly competitive in balancing efficacy and safety, especially in the long-term management of chronic inflammatory diseases. 9. Teprotumumab N01 Teprotumumab N01 is a monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R), independently developed by Innovent Biologics. It was approved in China on March 11, 2025, for the treatment of moderate-to-severe Graves' ophthalmopathy (GO). By inhibiting the IGF-1R signaling pathway, the drug reduces orbital inflammation, fibrosis, and muscle edema, thereby improving symptoms such as proptosis, diplopia, and visual impairment. Graves' ophthalmopathy is an autoimmune thyroid-associated eye disease characterized by the activation of the IGF-1/IGF-1R pathway, which triggers downstream signaling cascades (e.g., PI3K/Akt/mTOR), leading to orbital fibroblast proliferation, collagen deposition, and cytokine release. Teprotumumab N01 binds with high affinity to the α-subunit of IGF-1R, blocking the activation of the IGF-1 pathway, suppressing fibroblast activation and inflammation, and reducing orbital tissue swelling and fibrosis. The research team enhanced the drug’s specificity and safety by optimizing the antibody structure, including modifications to the Fc region to reduce immunogenicity. Approval was based on strong Phase III trial results: compared to placebo, patients receiving Teprotumumab experienced an average reduction of 3.3 mm in proptosis (P<0.001), and 80% showed symptom improvement (e.g., reduced diplopia, stabilized vision). Adverse events were primarily mild injection site reactions. Teprotumumab N01 is indicated for patients with moderate-to-severe active Graves' ophthalmopathy (e.g., Clinical Activity Score ≥3) who are either intolerant or unresponsive to corticosteroids. Its differentiated advantages are threefold: 1.Precision targeting of the IGF-1R pathway: Specifically blocks IGF-1R activation, directly inhibiting abnormal proliferation of orbital fibroblasts and associated inflammation, thus reducing fibrosis and edema. This avoids systemic side effects of corticosteroids, such as osteoporosis, blood glucose fluctuations, and immunosuppression. 2.Robust and sustained efficacy: Phase III data showed a 3.3 mm average reduction in proptosis after six months, with marked improvement in diplopia and orbital pressure. These benefits were maintained over a 12-month follow-up period and significantly outperformed placebo. 3.Good safety and convenient administration: No severe immune-related adverse events (e.g., neutropenia or liver enzyme abnormalities) were observed; only mild injection site reactions occurred. The drug is administered via intravenous infusion, suitable for outpatient treatment and enhancing patient adherence. Looking forward, Teprotumumab N01 may be used preventatively in early or mild GO to delay disease progression and could be explored in combination with radiotherapy or surgery to optimize outcomes in severe cases. This would further expand its clinical value. The approval of Teprotumumab N01 marks a breakthrough in targeted therapy for rare diseases in China. Its development process—from antibody screening to clinical validation—demonstrates Innovent’s comprehensive capabilities in biologic innovation. Currently, the only similar product worldwide is Lilly’s Teprotumumab, approved by the U.S. FDA in 2019. However, Teprotumumab N01, through differentiated design (e.g., longer half-life enabling dosing every three weeks) and localized clinical data, significantly reduces treatment costs and benefits more Chinese patients. Furthermore, its development model provides a reference for targeted therapies in other autoimmune-related ophthalmic diseases (e.g., dry eye disease associated with Sjögren’s syndrome), enhancing China’s global competitiveness in ophthalmologic biologics. 4o 10. Denosumab-bnhtDenosumab-bnht is a biosimilar of denosumab developed by Germany-based Fresenius Kabi, approved in the United States on March 25, 2025, for the treatment of various skeletal-related disorders. As a monoclonal antibody biosimilar, it functions by targeting and inhibiting the receptor activator of nuclear factor kappa-Β ligand (RANKL), mirroring the mechanism of the reference product. By optimizing the manufacturing process, it offers a more cost-effective treatment option for patients. Structurally, functionally, and in terms of safety and efficacy, Denosumab-bnht is highly consistent with the originator drug, and is indicated for conditions such as osteoporosis, giant cell tumor of bone (GCTB), glucocorticoid-induced osteoporosis, hypercalcemia of malignancy, and multiple myeloma, particularly in cases of bone density loss or skeletal damage caused by abnormal bone metabolism. The drug works by blocking the interaction between RANKL and its receptor RANK on the surface of osteoclast precursors and mature osteoclasts. This inhibition suppresses the differentiation, activity, and survival of osteoclasts, thereby reducing bone resorption and maintaining bone density. This mechanism significantly lowers fracture risk in osteoporosis patients, particularly postmenopausal women and cancer patients at risk of bone metastases. Moreover, by modulating bone metabolism, it helps manage the progression of GCTB and alleviate tumor-related bone destruction and pain. As a biosimilar, Denosumab-bnht demonstrates equivalence to the originator in pharmacokinetics, immunogenicity, and clinical efficacy, while simplified manufacturing enhances accessibility. In clinical practice, Denosumab-bnht has broad indications: for osteoporosis, a subcutaneous injection every six months significantly improves patient compliance and reduces the risk of vertebral, non-vertebral, and hip fractures; in GCTB, it can reduce tumor size and relieve bone pain; for emergency treatment of malignancy-associated hypercalcemia, a single injection rapidly lowers serum calcium levels and improves prognosis. Additionally, the drug can prevent skeletal-related fractures in patients with multiple myeloma or solid tumors and reduce the need for radiation therapy. Phase III clinical trials have shown no statistically significant difference in efficacy compared to the originator, with a favorable safety profile. Common adverse reactions include injection site reactions and hypocalcemia, while severe allergic reactions are rare. From a development and market positioning perspective, Denosumab-bnht followed a rigorous biosimilar development pathway, including structural characterization, animal studies, and clinical comparison trials to ensure interchangeability with the reference product. Fresenius Kabi, leveraging its experience in the biosimilar field, optimized antibody production to reduce costs and provide a cost-effective option for both patients and healthcare systems. Its approval marks further expansion of biosimilars in the treatment of skeletal disorders, and in the U.S. market, its pricing advantage is expected to accelerate substitution of the originator drug, offering critical therapeutic opportunities for underserved populations, including those in developing countries. 11. Liraglutide BiosimilarThe liraglutide biosimilar developed by Zhuhai United Pharmaceutical is the first GLP-1 receptor agonist biosimilar approved in China. It was granted market approval on March 18, 2025, for the treatment of type 2 diabetes. Jointly developed by Zhuhai United Pharmaceutical and its subsidiaries, this product contains liraglutide, a recombinant glucagon-like peptide-1 (GLP-1) analog highly homologous to human GLP-1, which lowers blood glucose by mimicking endogenous GLP-1's physiological effects. As a biosimilar, it exhibits high structural, functional, safety, and efficacy similarity to the reference drug (Novo Nordisk’s Victoza), while optimized production processes and cost control provide a more affordable treatment option—particularly beneficial for China's large type 2 diabetes population. The mechanism of action centers on GLP-1 receptor activation: it stimulates glucose-dependent insulin secretion from pancreatic β-cells, suppresses glucagon secretion from α-cells to reduce hepatic glucose output, delays gastric emptying, and increases satiety, thereby lowering both blood glucose levels and body weight. With a half-life of 12–14 hours, it supports once-daily subcutaneous administration. Its fatty acid side chain modification reduces immunogenicity and lowers the risk of allergic reactions. These characteristics result in comprehensive benefits for glycemic control, cardiovascular protection, and weight management, making it suitable for patients inadequately managed by oral antidiabetic drugs or those with insulin resistance. Clinically, the liraglutide biosimilar offers multiple advantages in managing type 2 diabetes. Phase III clinical trials showed no statistically significant difference in HbA1c reduction compared to the originator drug, with average reductions ranging from 1.5% to 2.0%, and significantly lower risk of hypoglycemia due to its glucose-dependent mechanism. In addition, it promotes weight loss (average reduction of 3%–5%) and offers cardiovascular benefits by improving lipid profiles and slowing the progression of atherosclerosis. Regarding safety, the most common adverse events are mild gastrointestinal reactions (e.g., nausea, diarrhea), which tend to decrease over time; no serious immunogenic events were observed. Looking ahead, the clinical use of this biosimilar may expand to other metabolic syndrome-related conditions, such as obesity and non-alcoholic fatty liver disease (NAFLD). Long-term cardiovascular benefits observed in the originator drug’s post-marketing studies could also support its positioning as a preferred option for diabetic patients with elevated cardiovascular risk. Conclusion The global drug approvals in March 2025 highlight three trends in biomedicine: the clinical translation of gene and RNA therapies, breakthroughs in antibiotics and antivirals, and the broader accessibility of biosimilars and vaccines. In gene therapy, Fitusiran became the world’s first RNAi treatment to offer once-monthly management for hemophilia by suppressing antithrombin. Novel treatments such as Teprotumumab N01 (an IGF-1R monoclonal antibody) and Encofosbuvir (a pan-genotypic DAA for hepatitis C) target precise signaling pathways or viral replication mechanisms, offering breakthroughs for rare diseases and chronic infections. In the antibiotic field, Suraxavir Marboxil emerged as a novel weapon against resistant bacteria by dual inhibition of DNA enzymes, while Mashurashave, China’s first broad-spectrum influenza drug, addressed resistance and treatment complexity with its single-dose regimen. On the public health front, Denosumab-bnht and the liraglutide biosimilar enhance treatment accessibility for osteoporosis and diabetes through cost reductions. Meanwhile, quadrivalent influenza vaccines employing split-virus technology improve coverage against multiple viral subtypes, strengthening public health preparedness. The approvals of Diazoxide Choline (for Prader-Willi syndrome) and Ivarmacitinib (a JAK inhibitor for ankylosing spondylitis) further extend the application of targeted therapies in metabolic and autoimmune disorders. Finally, the innovation and global partnerships of Chinese pharmaceutical companies—such as Hengrui and Innovent Biologics—are accelerating the integration of global R&D resources. How to obtain the latest research advancements in the field of biopharmaceuticals? In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!

MELBOURNE, Australia and INDIANAPOLIS, April 22, 2025 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, NASDAQ: TLX, Telix, the Company) today provides an update on its commercial and operational performance for the quarter ended 31 March 2025 (Q1 2025). All figures are in $US unless stated otherwise. Strong Q1 2025 revenue growth Q1 2025 unaudited revenue of approximately $186 million represents an increase of 62% over the prior year corresponding quarter (Q1 2024: $115M) and a quarter-over-quarter increase of 31% (Q4 2024: $142 million) and includes: $151 million from global sales of Illuccix®, up 35% over the prior year corresponding quarter (Q1 2024: $112 million) and a quarter-over-quarter increase of 9% (Q4 2024: $139 million).$33 million from RLS Radiopharmacies (RLS) since the acquisition completed on 27 January 20251. FY 2025 guidance reaffirmed Telix confirms FY 2025 revenue guidance of $770 million to $800 million2.Guidance reflects revenue from Illuccix® sales in jurisdictions with a marketing authorization, and 11 months of revenue from RLS1,3.Revenue guidance is expected to be updated at the appropriate time, following and subject to reimbursement for Gozellix® in the United States (U.S.) and Illuccix® in ex-U.S. markets.Telix confirms research and development (R&D) expenditure guidance, expecting a year-over-year increased investment range for FY 2025 of 20% to 25% compared to FY 2024. Q1 2025 commentary and recent highlights Telix Managing Director and Group CEO, Dr. Christian Behrenbruch, stated, “Illuccix has continued its momentum, gaining market share and maintaining price stability in a competitive landscape. Telix is the only company with two FDA4-approved PSMA-PET5 imaging agents – Illuccix and Gozellix – enabling us to broaden patient reach and maximize choice for our customers. The expansion of our commercial portfolio and launches of Illuccix into new international markets provides a foundation to diversify and grow revenue globally, while we continue to deliver on multiple catalysts in our pipeline. This quarter also includes the first two months of revenue from RLS since completion of our acquisition, highlighting its potential as a platform to drive further growth. This strategic acquisition has significantly expanded our manufacturing footprint in the U.S., which we believe is an increasingly important consideration amid changing global trade dynamics.” Therapeutics business ProstACT™ Global, the Phase 3 trial of TLX591 (177Lu-rosopatamab tetraxetan), Telix’s prostate cancer therapy candidate, continues to recruit and remains on track to deliver a Part 1 readout (safety and dosimetry) in H1 2025.IPAX-Linz, a Phase 2 investigator-initiated trial of TLX101 (131I-iodofalan, or 131I-IPA), Telix’s brain cancer therapy candidate, reported positive preliminary results. Early efficacy from IPAX-1 was substantiated and TLX101 was found to be well tolerated in combination with external beam radiation therapy. No serious adverse events were reported in patients at first or second recurrence with high-grade gliomas (HGG), including glioblastoma, often after multiple resections6.Following consultations with the FDA, the Company remains on track to submit Investigational New Drug (IND) applications to enable pivotal trials for TLX101 and Telix’s kidney cancer therapy candidate (TLX250, 177Lu-DOTA-girentuximab) to commence this year, subject to regulatory approval.The first patient was dosed in the Phase 1 ZOLAR7 trial of TLX300-CDx (89Zr-olaratumab), which aims to validate the use of olaratumab, an antibody exclusively licensed from Eli Lilly and Company (Lilly), as a potential treatment for advanced, metastatic soft tissue sarcoma.Telix completed the acquisition of a suite of clinically validated FAP8-targeting therapeutic and precision medicine (diagnostic) radiopharmaceutical candidates. Telix has added the lead FAP-targeting therapeutic compound to its pipeline under the designation TLX400.Telix acquired the therapeutics assets of antibody engineering company ImaginAb, Inc. The transaction included a pipeline of next-generation therapeutic candidates, a proprietary novel biologics technology platform, and a protein engineering and discovery research facility in Los Angeles, California9, staffed by a talented team of discovery, protein engineering and radiopharmaceutical development experts who joined the Company on the ImaginAb acquisition closing.Telix announced that it has developed and validated a breakthrough generator technology for the production of lead-212 (212Pb) and successfully completed first production of this promising alpha-emitting therapeutic radioisotope10. The new generator technology, developed internally by Telix’s IsoTherapeutics team, significantly increases the amount of radioactivity, yield and shelf life compared to currently available 212Pb generators and provides an additional isotope supply for Telix’s next-generation targeted alpha therapy program. Precision Medicine business On 21 March 2025, the FDA approved the New Drug Application (NDA) for Gozellix®11, Telix’s next-generation PSMA-PET imaging agent for prostate cancer that provides a longer shelf life of up to six hours and an extended distribution radius compared to existing gallium-based imaging products.Commercial launch of Gozellix® in the U.S. is expected to commence during Q2 2025 where Telix has appointed Cardinal Health, Inc. (Cardinal Health)12 and RLS Radiopharmacies as distribution partners.Telix received a positive decision on the decentralized Marketing Authorization Application (MAA) for Illuccix® in the European Economic Area (EEA)13. Illuccix® also received MAA approval in the United Kingdom (UK) and Brazil. Country level approvals are now being implemented in the EEA, with approvals to date granted in Denmark, Ireland, Luxembourg, Malta, the Netherlands, Norway, and Sweden14.Illuccix® is now commercially available in Brazil, under the joint venture with R2PHARMA (Telix Innovations Brazil, Ltda.), and commercial launches will commence in the UK and in European markets where Illuccix® has received approval during Q2 2025.The FDA accepted the Biologics License Application (BLA) for Telix’s kidney cancer imaging candidate TLX250-CDx (Zircaix®15, 89Zr-DFO-girentuximab), granted a Priority Review, and provided a PDUFA16 goal date of 27 August 2025.The NDA for TLX101-CDx (Pixclara®15, 18F-floretyrosine or 18F-FET), Telix’s brain cancer imaging candidate, continues to progress with a PDUFA goal date of 26 April 2025. Telix Manufacturing Solutions (TMS) In April, the TMS Brussels South facility (Seneffe) received notice of its GMP accreditation, enabling first commercial radiopharmaceutical dose production.Telix reaffirms that it does not expect any material impact on its business or supply chain as a result of the international trade tariffs announced by the U.S. government on 2 April 2025 (subject to a three-month delay) or the subsequent inclusion of pharmaceuticals. Telix has an extensive U.S.-based manufacturing and distribution infrastructure, including third-party manufacturing sites and radiopharmacy partner networks, for the production and delivery of its FDA-approved products Illuccix® and Gozellix®17.Telix also notes that it does not rely on rare earth elements of the kind utilized in semi-conductor supply chains and is therefore not impacted by the recent export controls imposed by the Chinese government this month. Board renewal Telix announced new Director appointments as part of a Board succession planning and renewal process18: H Kevin McCann AO will retire as a Non-Executive Director (NED) and Board Chairman on 21 May 2025, immediately following Telix’s Annual General Meeting.Tiffany Olson will be appointed Board Chair following Mr. McCann’s retirement.Marie McDonald joined the Board as a NED, effective 3 March 2025 and, on appointment, immediately succeeded Mr. McCann as Chair of the People Committee.Anne Whitaker joined the Board as a NED, effective 7 April 2025, and was appointed as a member of the Audit and Risk Committee and the People Committee. Guidance disclaimer The stated revenue guidance is based on expected global and domestic economic conditions and is subject to known and unknown risks, uncertainties and other factors that may cause our actual results to differ materially. As such, investors are cautioned not to place undue reliance on this guidance and in particular Telix cannot guarantee a particular result. In compiling financial forecasts, a number of key variables that may have a significant impact on guidance have been identified and are listed below. Key variables that could cause actual results to differ materially include: the success and timing of research and development activities; decisions by regulatory authorities regarding approval of our products as well as their decisions regarding label claims; competitive developments affecting our products; the ability to successfully market new and existing products; difficulties or delays in manufacturing; trade buying patterns and fluctuations in interest and currency exchange rates; legislation, regulation, or policy that affects product production, distribution, pricing, reimbursement, access or tax; acquisitions and divestitures; research collaborations; litigation or government investigations; and Telix’s ability to protect its patents and other intellectual property. See the Legal Notices section below for additional information, risks and assumptions. About Telix Pharmaceuticals Limited Telix is a biopharmaceutical company focused on the development and commercialization of therapeutic and diagnostic radiopharmaceuticals and associated medical technologies. Telix is headquartered in Melbourne, Australia, with international operations in the United States, Brazil, Canada, Europe (Belgium and Switzerland), and Japan. Telix is developing a portfolio of clinical and commercial stage products that aims to address significant unmet medical needs in oncology and rare diseases. ARTMS, IsoTherapeutics, Lightpoint, Optimal Tracers and RLS are Telix Group companies. Telix is listed on the Australian Securities Exchange (ASX: TLX) and the Nasdaq Global Select Market (NASDAQ: TLX). Telix’s prostate imaging product, gallium-68 (68Ga) gozetotide injection (also known as 68Ga PSMA-11 and marketed under the brand name Illuccix®), has been approved by the FDA19, the Australian Therapeutic Goods Administration (TGA)20, Health Canada21, the UK Medicines and Healthcare Products Regulatory Agency (MHRA)22, by the Brazilian Health Regulatory Agency (ANVISA)23, and in multiple countries within the European Economic Area (EEA)24. Illuccix® is currently in national approval review elsewhere in the EEA following a positive decentralized procedure (DCP) opinion by the German medical regulator, BfArM25. Gozellix® (kit for the preparation of gallium-68 (68Ga) gozetotide injection) has been approved by the FDA26. Telix’s osteomyelitis (bone infection) imaging agent, technetium-99m (99mTc) besilesomab, marketed under the brand name Scintimun®, is approved in 32 European countries and Mexico. Telix’s miniaturized surgical gamma probe, SENSEI®, for minimally invasive and robotic-assisted surgery, is registered with the FDA for use in the U.S. and has attained a Conformité Européenne (CE) Mark for use in the European Economic Area. No other Telix product has received a marketing authorization in any jurisdiction. Visit www.telixpharma.com for further information about Telix, including details of the latest share price, ASX and SEC filings, investor and analyst presentations, news releases, event details and other publications that may be of interest. You can also follow Telix on LinkedIn, X and Facebook. Telix Investor Relations Ms. Kyahn WilliamsonTelix Pharmaceuticals LimitedSVP Investor Relations and Corporate CommunicationsEmail: kyahn.williamson@telixpharma.com Telix Investor Relations (U.S.) Annie KasparianTelix Pharmaceuticals LimitedDirector Investor Relations and Corporate CommunicationsEmail: annie.kasparian@telixpharma.com This announcement has been authorised for release by the Telix Pharmaceuticals Limited Disclosure Committee on behalf of the Board. Legal Notices You should read this announcement together with our risk factors, as disclosed in our most recently filed reports with the Australian Securities Exchange (ASX), U.S. Securities and Exchange Commission (SEC), including our Annual Report on Form 20-F filed with the SEC, or on our website. The information contained in this announcement is not intended to be an offer for subscription, invitation or recommendation with respect to securities of Telix Pharmaceuticals Limited (Telix) in any jurisdiction, including the United States. The information and opinions contained in this announcement are subject to change without notification. To the maximum extent permitted by law, Telix disclaims any obligation or undertaking to update or revise any information or opinions contained in this announcement, including any forward-looking statements (as referred to below), whether as a result of new information, future developments, a change in expectations or assumptions, or otherwise. No representation or warranty, express or implied, is made in relation to the accuracy or completeness of the information contained or opinions expressed in the course of this announcement. This announcement may contain forward-looking statements, including within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, that relate to anticipated future events, financial performance, plans, strategies or business developments. Forward-looking statements can generally be identified by the use of words such as “may”, “expect”, “intend”, “plan”, “estimate”, “anticipate”, “believe”, “outlook”, “forecast” and “guidance”, or the negative of these words or other similar terms or expressions. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. Forward-looking statements are based on Telix’s good-faith assumptions as to the financial, market, regulatory and other risks and considerations that exist and affect Telix’s business and operations in the future and there can be no assurance that any of the assumptions will prove to be correct. In the context of Telix’s business, forward-looking statements may include, but are not limited to, statements about: the initiation, timing, progress and results of Telix’s preclinical and clinical trials, and Telix’s research and development programs; Telix’s ability to advance product candidates into, enrol and successfully complete, clinical studies, including multi-national clinical trials; the timing or likelihood of regulatory filings and approvals for Telix’s product candidates, manufacturing activities and product marketing activities; Telix’s sales, marketing and distribution and manufacturing capabilities and strategies; the commercialization of Telix’s product candidates, if or when they have been approved; Telix’s ability to obtain an adequate supply of raw materials at reasonable costs for its products and product candidates; estimates of Telix’s expenses, future revenues and capital requirements; Telix’s financial performance; developments relating to Telix’s competitors and industry; the anticipated impact of U.S. and foreign tariffs and other macroeconomic conditions on Telix’s business; and the pricing and reimbursement of Telix’s product candidates, if and after they have been approved. Telix’s actual results, performance or achievements may be materially different from those which may be expressed or implied by such statements, and the differences may be adverse. Accordingly, you should not place undue reliance on these forward-looking statements. ©2025 Telix Pharmaceuticals Limited. Telix Pharmaceuticals, Telix Group company, and Telix product names and logos are trademarks of Telix Pharmaceuticals Limited and its affiliates – all rights reserved. Trademark registration status may vary from country to country. ______________________________________________1 Excludes revenue contribution from Illuccix® sales.2 Refer to ASX disclosures 20 February 2025. 3 See Guidance Disclaimer for further information.4 U.S. Food and Drug Administration.5 Imaging of prostate-specific membrane antigen with positron emission tomography.6 Telix ASX disclosure 16 April 2025.7 Telix media release 1 April 2025. ClinicalTrials.gov ID: NCT06537596.8 Fibroblast activation protein.9 Telix ASX disclosure 13 January 2025.10 Telix media release 13 March 2025.11 Telix ASX disclosure 21 March 2025.12 Telix media release 8 April 2025.13 Telix ASX disclosure 17 March 2025.14 Illuccix® received marketing authorization in the UK, Brazil, Denmark, Luxembourg, Malta, the Netherlands and Norway during Q1 2025. Illuccix® received marketing authorization in Ireland and Sweden on 4 and 11 April 2025, respectively. 15 Brand name subject to final regulatory approval. 16 Prescription Drug User Fee Act.   17 Telix ASX disclosure 7 April 2025.18 Telix ASX disclosure 26 February 2025.19 Telix ASX disclosure 20 December 2021.20 Telix ASX disclosure 2 November 2021.21 Telix ASX disclosure 14 October 2022.22 Telix ASX disclosure 13 February 2025.23 Telix ASX disclosure 18 March 2025.24 Denmark, Ireland, Luxembourg, Malta, the Netherlands, Norway and Sweden at time of release.25 Telix ASX disclosure 17 January 2025.26 Telix ASX disclosure 21 March 2025.