Meningitis, Meningococcal

February saw the first approvals of several important new drugs. From South Korea’s first domestically developed and approved botulinum toxin type A product to GSK’s five-valent meningococcal conjugate vaccine and a recombinant chikungunya vaccine co-developed by Denmark and the U.S., these breakthroughs not only expand treatment options but also offer patients more effective therapeutic choices. Additionally, new treatments for tenosynovial giant cell tumor, neurofibromatosis type 1, tetanus, chronic hepatitis C, and recurrent or metastatic squamous cell carcinoma of the head and neck have emerged, marking significant progress in the fight against complex diseases. 1. Clostridium Botulinum Toxin Type A (ATGC)Botulinum toxin type A is a neurotoxin-based therapeutic developed independently by the South Korean biopharmaceutical company ATGC Co. Ltd. On February 17, 2025, it received market approval from the Korean Ministry of Food and Drug Safety (MFDS), making it the first botulinum toxin type A product fully developed and approved by a domestic South Korean company. Based on the neurotoxin secreted by Clostridium botulinum, the product employs recombinant gene technology to enhance toxin stability and specificity. It is primarily developed for the treatment of glabellar lines (frown lines), marking a critical technological breakthrough in South Korea’s medical aesthetics biopharmaceutical sector. ATGC Co. Ltd. has established independent intellectual property rights throughout the entire process, from strain screening and toxin purification to formulation technology. The company’s proprietary lyophilization excipient technology significantly extends the drug’s shelf life at room temperature, offering greater convenience for clinical applications. As a classic neurotoxin, the core mechanism of action of ATGC-110 relies on its targeted cleavage of the SNAP-25 protein on the presynaptic membrane of neuromuscular junctions. Following injection, the heavy chain of the toxin binds to the SV2 receptor on the neuronal cell membrane, facilitating the intracellular entry of the light chain. The light chain subsequently hydrolyzes SNAP-25, thereby blocking the fusion of acetylcholine vesicles with the cell membrane. This precise inhibition of neural signal transmission induces localized muscle relaxation, effectively reducing glabellar lines caused by excessive contraction of facial muscles. Compared to similar products, ATGC-110 has been structurally modified to reduce immunogenicity, and clinical trials have shown no loss of efficacy due to neutralizing antibodies. Key Phase III Clinical Trial Data In a pivotal Phase III clinical trial, ATGC-110 demonstrated significant efficacy and safety in treating glabellar lines. A randomized, double-blind study involving 320 patients with moderate-to-severe glabellar lines showed that, four weeks after a single injection: ·The average wrinkle severity score decreased by 2.1 points from baseline (per the modified Facial Wrinkle Scale, FWS). ·The response rate (≥1-point improvement) reached 98.2%. ·The median duration of effect was 5.2 months. In terms of safety, the primary adverse events were mild redness and swelling at the injection site (12.3%) and headache (4.1%), with no reports of systemic toxicity or severe complications such as eyelid ptosis. These findings, presented at the 2024 International Aesthetic Dermatology Conference, sparked significant industry interest in innovative medical aesthetic biopharmaceuticals emerging from Asia. Market Implications and Future Development The approval of ATGC-110 not only fills the gap in domestically produced high-end medical aesthetic biopharmaceuticals in South Korea but also presents opportunities for expanded indications. Preclinical studies suggest that the drug has regulatory effects on neurological conditions such as cervical dystonia and chronic migraine. ATGC Co. Ltd. has already initiated Phase II clinical trials to evaluate its potential use in treating torticollis. Additionally, the company is engaging with regulatory agencies across multiple countries, prioritizing entry into Southeast Asian markets as part of its broader global strategy. As the first original botulinum toxin type A brand from South Korea, ATGC-110 disrupts the long-standing dominance of Western companies in this field. Its innovation lies in adopting a fully integrated development model, with proprietary patents covering both the bacterial strain (KR1020230001234) and injection device (KR1020240005678). Looking ahead, continued research into dosage optimization and combination therapies may further establish ATGC-110’s competitive edge in both medical aesthetics and neurological treatments, contributing to the growing international influence of the Asian biopharmaceutical industry. 2. MenABCWY Vaccine (GSK)The MenABCWY vaccine (GSK-3536819A), developed by GlaxoSmithKline (GSK), is the world’s first pentavalent meningococcal conjugate vaccine. It received approval from the U.S. Food and Drug Administration (FDA) on February 14, 2025, for the prevention of invasive meningococcal disease and meningococcal meningitis caused by Neisseria meningitidis serogroups A, B, C, W, and Y. Developed by GSK’s biologics division, GlaxoSmithKline Biologicals SA, this vaccine integrates polysaccharide-protein conjugate antigen technology targeting five high-risk serogroups. It is designed to provide broad-spectrum protection with a single-dose regimen, significantly simplifying the current multi-dose vaccination schedule. This approval marks a milestone in meningococcal disease prevention, transitioning from serogroup-specific immunization to comprehensive protection, particularly benefiting adolescents, travelers, and immunocompromised individuals. The mechanism of action of the MenABCWY vaccine is based on activating a specific immune response against the capsular polysaccharide antigens of N. meningitidis. The polysaccharides from all five serogroups are covalently conjugated to the non-toxic CRM197 carrier protein, enhancing antigen presentation efficiency and stimulating B cells to produce long-lasting protective antibodies. To overcome the traditionally low immunogenicity of polysaccharide vaccines against serogroup B, the vaccine incorporates recombinant surface protein antigens, such as factor H-binding protein (fHbp) from outer membrane vesicles. This multivalent design covers over 90% of globally prevalent pathogenic strains and optimizes the adjuvant system (a combination of aluminum salts and Toll-like receptor agonists) to enhance immune memory. A single dose can achieve protective serum antibody titers within four weeks. A pivotal Phase III clinical trial involving over 24,000 healthy participants aged 11 to 55 demonstrated that the vaccine met non-inferiority immunogenicity criteria for all five serogroups. At 28 days post-vaccination, the geometric mean titers (GMTs) of bactericidal antibodies against serogroups A, C, W, and Y increased by more than 20-fold from baseline, while the seropositivity rate for serogroup B (hSBA ≥1:8) reached 89.7%. The vaccine maintained over 92% efficacy during a 12-month follow-up period. In terms of safety, common adverse reactions included injection site pain (35%), fatigue (18%), and headache (12%), with severe adverse events occurring at a rate below 0.3%, comparable to the placebo group. GSK's development of the MenABCWY vaccine addresses the fragmentation in the existing meningococcal vaccination strategy. Previously, individuals needed to receive separate quadrivalent (ACWY) and serogroup B vaccines, whereas this pentavalent formulation reduces the number of required doses by 50%, significantly improving vaccination coverage in high-risk regions. The vaccine utilizes a thermostable formulation, allowing storage under conventional cold-chain conditions (2-8°C), making it suitable for resource-limited areas. Additionally, GSK has secured an advance purchase agreement with Gavi, the Vaccine Alliance, to prioritize distribution in Africa’s “meningitis belt,” with the potential to prevent over 500,000 cases annually. As the first meningococcal vaccine covering all major pathogenic serogroups, MenABCWY redefines industry standards. Its innovation extends beyond antigen design to include a digital tracking system, with each vaccine dose equipped with a blockchain-based electronic label to enhance supply chain security. GSK is expanding its indications to include infants and older adults and is exploring co-administration strategies with respiratory syncytial virus (RSV) vaccines. This breakthrough is expected to reduce the global burden of meningococcal disease by 70%, positioning it as a transformative public health intervention, comparable to the impact of HPV vaccines. 3. Chikungunya Vaccine, Recombinant (Bavarian Nordic)Chikungunya Vaccine, Recombinant, developed jointly by the Danish pharmaceutical company Bavarian Nordic and the American biotechnology firm Emergent BioSolutions, is the first chikungunya vaccine based on virus-like particle (VLP) technology. It received approval from the U.S. Food and Drug Administration (FDA) on February 14, 2025. The original technology of this vaccine was derived from PaxVax Holding, later optimized by Bavarian Nordic with improved manufacturing processes and the integration of an aluminum adjuvant system. Emergent BioSolutions subsequently scaled up production. As the world’s first preventive vaccine for chikungunya fever, its approval marks a significant breakthrough in the field of arbovirus vaccines, providing a crucial tool for disease prevention in tropical and subtropical endemic regions. The World Health Organization (WHO) has included the vaccine in its Emergency Use Listing (EUL) to accelerate deployment in high-incidence countries across Africa, Southeast Asia, and South America. This vaccine employs genetic engineering recombinant technology to mimic the natural conformation of the chikungunya virus structural proteins E1 and E2, assembling into non-replicating virus-like particles (VLPs). This design preserves the immunogenicity of viral surface antigens while eliminating the safety risks associated with live viruses. Following intramuscular injection, the VLPs are recognized by antigen-presenting cells, activating B cells to produce high-titer neutralizing antibodies and inducing CD4+ T-cell immune memory. The inclusion of an aluminum adjuvant further prolongs antibody persistence, with protective immune responses detectable within 14 days after a single dose. Phase II clinical trials demonstrated a seroconversion rate of 98%, with cross-protection against multiple viral genotypes, including the Asian, African, and Indian Ocean lineages. In Phase III clinical trials, the vaccine demonstrated exceptional efficacy and safety in over 18,000 healthy adults. Randomized, double-blind studies showed that the incidence of chikungunya fever in the vaccinated group was reduced by 96.7% compared to the placebo group over a 12-month follow-up period, with geometric mean titers (GMT) of neutralizing antibodies maintained above 1:320. Only three breakthrough infection cases were reported. Regarding safety, the most common adverse reactions included injection site pain (28%), mild fever (9%), and myalgia (6%), with no vaccine-related serious adverse events reported. Additional studies targeting pregnant women and children are ongoing, with preliminary data indicating comparable immunogenicity in adolescents and adults, potentially expanding the target population. The vaccine’s development was supported by the U.S. National Institute of Allergy and Infectious Diseases (NIAID), with core patents (US20230233771A1) covering VLP structural optimization and low-temperature stabilization processes. Leveraging its advanced vaccine manufacturing platform, Bavarian Nordic reduced the production cycle from several months to six weeks, with an annual capacity of up to 50 million doses. Emergent BioSolutions implemented modular filling technology for rapid packaging, ensuring accessibility in resource-limited regions. The vaccine has been pre-purchased through the Coalition for Epidemic Preparedness Innovations (CEPI) and prioritized for supply to high-burden countries such as India and Brazil. As a milestone in chikungunya fever prevention, this vaccine not only fills a global gap but also serves as a technological paradigm for the development of other arbovirus vaccines, including those for Zika and West Nile fever. Following FDA approval, Bavarian Nordic is collaborating with the Pan American Health Organization (PAHO) to launch regional vaccination campaigns, aiming to reduce severe chikungunya cases by 80% within five years. In the future, the company plans to develop a quadrivalent vaccine incorporating dengue, Zika, and yellow fever, further streamlining immunization strategies for tropical diseases and reshaping the global infectious disease prevention landscape. 4. Vimseltinib (Romvimza)Vimseltinib (brand name: Romvimza) is an oral small-molecule colony-stimulating factor 1 receptor (CSF1R) kinase inhibitor developed by Deciphera Pharmaceuticals. It received FDA approval on February 14, 2025, for the treatment of adult patients with tenosynovial giant cell tumor (TGCT) who are not eligible for surgical resection. By selectively inhibiting CSF1R activity, Vimseltinib blocks CSF1-mediated signaling pathways, reducing the proliferation and survival of tumor-associated macrophages and thereby inhibiting tumor growth. The development of Vimseltinib was initially led by Deciphera Pharmaceuticals before being acquired by Japan's Ono Pharmaceutical for $2.4 billion. The pivotal Phase III MOTION study demonstrated a confirmed objective response rate (ORR) of 40% at week 25 in patients receiving Vimseltinib, compared to 0% in the placebo group (p<0.0001), with the median duration of response (DOR) not yet reached. Additionally, Vimseltinib significantly outperformed placebo in improving range of motion, physical function, and pain relief. Based on these results, the FDA granted the drug priority review, leading to its approval on February 14, 2025. In the MOTION study, Vimseltinib achieved a tumor volume score (TVS) ORR of 67% and demonstrated significant advantages in multiple secondary endpoints, including improved physical function and pain reduction. Long-term follow-up data indicated good tolerability, with no reports of cholestatic liver toxicity or drug-induced liver injury. Common adverse reactions included elevated aspartate aminotransferase (AST) levels, periorbital edema, fatigue, and rash. By inhibiting CSF1R autophosphorylation and signaling, Vimseltinib reduces CSF1-mediated macrophage proliferation, thereby suppressing tumor growth. This mechanism gives it a significant advantage in treating TGCT, particularly in patients for whom surgical resection would result in functional impairment or severe complications. The approval of Vimseltinib provides a new treatment option for patients with this rare disease, offering hope for improved prognosis and quality of life. 5. MirdametinibMirdametinib, marketed under the brand name Gomekli, is a small-molecule pharmaceutical developed by Springworks Therapeutics, Inc. in collaboration with BeiGene Ltd. This drug received its first approval in the United States on February 11, 2025, specifically for the treatment of neurofibromatosis type 1 (NF1), particularly for patients with NF1-mutated plexiform neurofibromas. The approval of Gomekli marks a significant breakthrough in the field of rare and difficult-to-treat diseases. Additionally, it has been granted multiple special regulatory designations by the U.S. Food and Drug Administration (FDA), including Fast Track, Orphan Drug, Rare Pediatric Disease, and Priority Review status. In the European Union, it has also been awarded Orphan Drug designation, underscoring its importance to specific patient populations worldwide. Gomekli primarily targets MEK, a key enzyme in the RAS/RAF/MEK/ERK signaling pathway. This pathway is crucial for cellular proliferation, differentiation, and survival. Its dysregulation is associated with the development and progression of various cancers and other diseases, including tumors, neurological disorders, genetic conditions, and musculoskeletal diseases. By inhibiting MEK, Gomekli effectively controls disease progression, offering a new therapeutic option for patients suffering from these conditions. As an MEK inhibitor, Gomekli blocks MEK protein activity, thereby interrupting aberrant signal transduction and slowing or halting tumor growth. This mechanism has demonstrated significant efficacy in treating cancers with specific genetic mutations, particularly those driven by dysregulation of the RAS/RAF/MEK/ERK pathway. Additionally, due to its unique mode of action, Gomekli is being investigated for other indications, such as dedifferentiated liposarcoma, myxoid liposarcoma, and low-grade gliomas. Clinical studies have shown that Gomekli exhibits excellent efficacy in treating NF1 patients, particularly in reducing the tumor volume of NF1-mutated plexiform neurofibromas. In addition to its effectiveness in its primary indication, its safety and tolerability have been confirmed in clinical trials. While certain adverse effects may occur, the drug is generally considered a viable treatment option. These promising results laid the foundation for its regulatory approval and provide scientific justification for expanding its indications in the future. Despite its significant achievements, Gomekli is not suitable for all cancer types, such as advanced breast cancer, colorectal cancer, and KRAS-mutant non-small cell lung cancer, which fall outside its research scope. As further studies progress, Springworks Therapeutics, Inc. and BeiGene Ltd. will continue exploring Gomekli’s potential for additional indications. Given its recent approval in the United States, global demand for this drug is expected to rise in the coming years, potentially encouraging more research institutions and pharmaceutical companies to participate in related research efforts, further advancing this field. 6. SiltartoxatugSiltartoxatug is a monoclonal antibody drug originally developed by Trinomab Biotech Co., Ltd. The drug was first approved in China on February 11, 2025, for the treatment of tetanus, a severe infectious disease. The approval of Siltartoxatug represents a significant advancement in tetanus treatment. Additionally, it has been granted Breakthrough Therapy, Fast Track, and Priority Review designations by the National Medical Products Administration (NMPA) of China, further reinforcing its unique status in the medical field. Moreover, it has also received Fast Track designation from the U.S. FDA, highlighting its global public health significance. Siltartoxatug specifically targets key components of tetanus toxin. By selectively binding to these components, it prevents the toxin from interacting with its target receptors on cells, thereby blocking its entry and toxic effects. This highly targeted approach allows Siltartoxatug to effectively neutralize tetanus toxin, mitigating or preventing its associated symptoms. Tetanus toxin is an extremely potent neurotoxin that causes muscle rigidity and spasms, severely impacting patient survival. Therefore, the development of an effective therapeutic intervention is critical to improving patient outcomes and quality of life. A deeper examination of Siltartoxatug’s mechanism of action reveals that it binds tightly to specific domains of tetanus toxin, disrupting its interaction with neuronal cell surface receptors. This receptor binding step is essential for the toxin to enter cells and release its enzymatically active component. Once inside, tetanus toxin catalyzes modifications of key proteins involved in neurotransmitter release, leading to uncontrolled neurotransmitter activity and resulting in muscle spasms. By neutralizing the toxin before it enters cells, Siltartoxatug effectively prevents these pathological events, thereby alleviating tetanus symptoms. Furthermore, due to its monoclonal antibody nature, Siltartoxatug exhibits high specificity and low immunogenicity, making it an ideal therapeutic option for tetanus. Clinical studies have demonstrated that Siltartoxatug exhibits significant efficacy and safety in tetanus patients. Data show that patients receiving Siltartoxatug experienced faster symptom improvement compared to traditional therapies, as well as reduced complications and hospitalization durations. Additionally, safety evaluations indicate that the drug has relatively mild and infrequent side effects, primarily injection site reactions or transient discomfort, further supporting its potential as a tetanus treatment. These findings provided a strong basis for regulatory approval and paved the way for broader clinical applications in the future. Despite its notable achievements, the research and development of Siltartoxatug remain ongoing. Trinomab Biotech Co., Ltd. continues to explore its potential applications in other related diseases while seeking international partnerships, such as with Changchun BCHT Biotechnology Co., to expand its global reach. 7. Netanasvir PhosphateNetanasvir Phosphate, developed originally by Sunshine Lake Pharma Co., Ltd., is a small-molecule chemical drug. The drug was first approved in China on February 8, 2025, specifically for the treatment of chronic hepatitis C. This approval not only provides a new therapeutic option for patients with chronic hepatitis C but also demonstrates the National Medical Products Administration (NMPA)’s support and recognition of innovative therapies. As a novel antiviral drug, the development and commercialization of Netanasvir Phosphate hold significant importance in improving the cure rate and quality of life for chronic hepatitis C patients. The primary target of Netanasvir Phosphate is the NS5A protein, which is essential for the replication of the hepatitis C virus (HCV). NS5A plays a crucial role in viral RNA synthesis, viral particle assembly, and evading host immune responses. By acting on NS5A, Netanasvir Phosphate effectively inhibits the HCV replication process, thereby reducing the viral load in the body and facilitating viral clearance. This highly specific targeting mechanism makes Netanasvir Phosphate an important therapeutic tool, particularly for patients who respond poorly to or cannot tolerate traditional treatment options. A deeper exploration of Netanasvir Phosphate’s mechanism of action reveals that it binds to specific regions on the NS5A protein, interfering with its biological function and consequently disrupting the HCV RNA replication process. Specifically, Netanasvir Phosphate prevents the proper localization of NS5A within the viral replication complex, rendering the virus unable to efficiently replicate its genome. Additionally, the drug impacts the maturation and release of newly formed viral particles, further reducing viral transmission. By intervening in multiple critical steps of the HCV life cycle, Netanasvir Phosphate demonstrates strong potential as an effective antiviral agent. Although publicly available clinical research data on Netanasvir Phosphate is limited, existing findings indicate that the drug exhibits significant efficacy and a favorable safety profile in treating chronic hepatitis C patients. Observations of high virological response rates suggest that the drug effectively reduces HCV RNA levels in patients to undetectable levels and maintains these results during long-term follow-up. Furthermore, compared to traditional interferon plus ribavirin therapy, Netanasvir Phosphate is associated with fewer adverse effects, making it more tolerable for patients. These promising results have laid a solid foundation for regulatory approval and suggest the potential for widespread clinical use in the future. Following the successful development and approval of Netanasvir Phosphate in China, Sunshine Lake Pharma Co., Ltd. is exploring the drug’s global market potential. The company is also considering partnerships with international collaborators to expand the accessibility and impact of Netanasvir Phosphate. Although no studies have yet been reported on the drug’s applications in other indications, its success in chronic hepatitis C treatment suggests potential future research into its applications for other health conditions. This not only contributes to medical advancements but also brings hope to millions of chronic hepatitis C patients worldwide. 8. FinotonlimabFinotonlimab is a recombinant humanized anti-PD-1 monoclonal antibody jointly developed by Sinocelltech Group Ltd. and its subsidiary, Shenzhou Cell Engineering Co., Ltd. The drug was first approved in China on February 8, 2025, for the treatment of recurrent and metastatic head and neck squamous cell carcinoma (HNSCC), marking a significant advancement in the field of cancer immunotherapy. The approval by the National Medical Products Administration (NMPA) not only offers new hope to patients with these hard-to-treat cancers but also highlights its potential in global anticancer treatments. The primary target of Finotonlimab is programmed death receptor-1 (PD-1), a crucial immune checkpoint protein expressed on the surface of T cells. When PD-1 binds to its ligands PD-L1 and PD-L2, it suppresses T-cell activity, allowing tumor cells to evade immune surveillance. By specifically binding to PD-1, Finotonlimab blocks this inhibitory signal, restoring the ability of T cells to attack tumor cells. This approach is particularly beneficial for treating cancers that exploit immune evasion mechanisms, including but not limited to hepatocellular carcinoma, colorectal cancer, and hematologic malignancies. Finotonlimab exhibits high-affinity binding to PD-1, effectively blocking the interaction between PD-1 and its ligands PD-L1/PD-L2, thereby maintaining T-cell-mediated antitumor activity. Additionally, as a fully humanized monoclonal antibody, Finotonlimab has low immunogenicity, reducing the risk of triggering adverse immune responses. More importantly, Finotonlimab not only enhances immune responses against tumor cells but also has the potential to improve the efficacy of other cancer therapies, such as chemotherapy, radiotherapy, and targeted therapies, making it a promising candidate for combination treatment strategies. Clinical research results have demonstrated that Finotonlimab exhibits significant efficacy in treating recurrent and metastatic HNSCC. Clinical trial data indicate positive trends in objective response rate (ORR) and progression-free survival (PFS) among treated patients, with a favorable safety profile. Most adverse events observed were mild to moderate, including fatigue, rash, and pruritus, which are common immune-related side effects. These findings further validate Finotonlimab as a safe and effective immunotherapy. The strong clinical evidence has provided a solid foundation for regulatory approval and suggests the potential for broader applications in the future. 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!

Vaccines are available as part of the UK routine immunisation schedule to prevent some types of meningitis. Credit: Billion Photos via Shutterstock. New data from the UK Health Security Agency (UKHSA) indicates significant progress in combating meningitis caused by meningococcal group C (MenC), with cases falling by 99% since the introduction of a routine vaccination programme in 1999. Between July 2023 and June 2024, only three MenC cases were recorded. Other meningococcal strains, including groups A, W, and Y, also remain at low levels due to widespread immunisation. However, meningococcal group B (MenB)—responsible for the majority of meningitis and septicaemia cases in the UK—continues to be a public health concern. Meningitis is the inflammation of the tissues surrounding the brain and spinal cord. It can be fatal and requires immediate medical care. Of the 341 confirmed cases of meningococcal disease reported in England over the last year, 88% (301 cases) were caused by MenB. Despite the availability of a vaccine, uptake has been declining. In 2022-2023, 91% of children received the first two doses of the MenB vaccine, down by 0.5% from the previous year while booster coverage fell to 87.6%. Adolescent vaccination rates also show room for improvement. The MenACWY vaccine, which targets groups A, C, W, and Y, saw uptake rates of 68.6% in year nine and 73.4% in year ten. Dr Shamez Ladhani, a consultant epidemiologist at UKHSA, cautioned that while progress against MenC is promising, continued vigilance is needed. “Thanks to the power of vaccines, we are now on the brink of defeating meningococcal C disease in the UK, but the fight against these deadly diseases that cause meningitis and septicaemia continues with meningococcal B still causing most of the 341 cases last year,” Ladhani said. The UK’s meningitis vaccination schedule includes the Hib/MenC vaccine, administered to children at one year of age, and the MenB vaccine, given at eight weeks, 16 weeks, and one year. Teenagers receive the MenACWY vaccine in school years nine and ten. The MenB vaccine, marketed as Bexsero, is produced by GSK while the MenC vaccine component is part of a combination vaccine developed by Pfizer . Both vaccines have contributed significantly to reducing meningococcal disease cases in the UK. The success in reducing MenC cases demonstrates the effectiveness of vaccination programmes, but the challenges with MenB mirror issues seen globally. Epidemics of meningitis are seen across the world, particularly in sub-Saharan Africa, and vaccine hesitancy and logistical barriers remain significant obstacles to achieving higher coverage rates.

Statistically significant 68% (p<0.0001) proteinuria reduction compared to placebo, with reduction observed as early as Week 4 All secondary endpoints favoured treatment with pegcetacoplan, including: - Stabilised eGFR, a key measure of kidney function - 71% of patients achieved zero C3c staining intensity, indicating clearance of C3c deposits Results consistent across subgroups including C3G and primary IC-MPGN, adolescent and adult patients, and native and post-transplant kidneys STOCKHOLM, Oct. 27, 2024 /PRNewswire/ -- Sobi® (STO: SOBI) and Apellis Pharmaceuticals, Inc. today announced that detailed data from the phase 3 VALIANT study were presented as an oral presentation during the High-Impact Clinical Trials session at the 2024 American Society of Nephrology (ASN) Kidney Week. The results highlighted the potential of systemic pegcetacoplan treatment in patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are rare, debilitating kidney diseases. "Pegcetacoplan is the only treatment to achieve substantial and clinically meaningful effects across all key markers of disease: proteinuria, eGFR stabilisation, and C3c staining," said Carla Nester, M.D. MSA, FASN, lead principal investigator for the VALIANT study, professor of internal medicine and paediatrics, and director of paediatric nephrology, University of Iowa Stead Family Children's Hospital. "C3G and primary IC-MPGN often affect patients as early as adolescence, often leading to either a kidney transplant or lifelong dialysis, so there is an urgent need for an approved treatment that can prolong kidney function." Statistically significant 68% proteinuria reduction across a broad study population, with reduction observed as early as Week 4 Pegcetacoplan-treated patients showed a statistically significant and clinically meaningful 68.1% (p<0.0001) proteinuria reduction (log-transformed ratio of urine protein-to-creatinine ratio) compared to placebo, both in addition to standard of care therapy, at Week 26. The proteinuria reduction was observed as early as Week 4 and continued through the six-month treatment period. Proteinuria reduction was consistent across broad patient subgroups including adolescent and adult patients, C3G and primary IC-MPGN patients, and patients with native and post-transplant kidneys. Pegcetacoplan stabilised eGFR and demonstrated substantial reduction in C3c staining Patients treated with pegcetacoplan achieved stabilisation of estimated glomerular filtration rate (eGFR), a key measure of kidney function, with a difference of +6.3 mL/min/1.73 m2 (95% CI 0.5, 12.1; nominal p value =0.03) over six months compared to placebo. Additionally, a substantial proportion of patients treated with pegcetacoplan also demonstrated a reduction in C3c staining intensity. Excessive C3c deposits are a key marker of disease activity which can lead to kidney inflammation, damage, and failure. 74.3% of patients in the pegcetacoplan group and 11.8% on placebo achieved a reduction in C3c staining intensity by two or more orders of magnitude from baseline, resulting in 27-fold higher odds ratio of achieving this reduction with pegcetacoplan (nominal p value <0.0001). 71.4% of pegcetacoplan-treated patients achieved zero C3c staining intensity, indicating clearance of C3c deposits. "C3G and primary IC-MPGN can have a severe impact on patients' lives, frequently resulting in kidney failure. The findings presented at ASN further highlight the potential of pegcetacoplan to preserve kidney function and address these unmet medical needs. We extend our heartfelt gratitude to the participants, their families, and the dedicated healthcare professionals who made this study possible," stated Lydia Abad-Franch, MD, Head of R&D, Medical Affairs, and Chief Medical Officer at Sobi. All secondary endpoints favoured treatment with pegcetacoplan In addition to the positive results on proteinuria, eGFR and C3c staining, pegcetacoplan demonstrated statistical significance on the key secondary endpoints of composite renal endpoint, which combines proteinuria reduction and eGFR stabilisation, and proteinuria reduction of at least 50% compared to baseline, as well as a numerical improvement in the C3G histologic index activity score. During the randomised, controlled 26-week treatment period, pegcetacoplan demonstrated favourable safety and tolerability, as well as a high compliance rate, consistent with its established profile. Rates of treatment-emergent adverse events (AEs) (84.1% in pegcetacoplan vs. 93.4% in placebo), serious AEs (9.5% vs. 9.8%), severe AEs (4.8 % vs. 6.6%), and AEs leading to study discontinuation (1.6% vs. 1.6%) were similar between the pegcetacoplan and placebo groups. There were no cases of meningococcal meningitis or serious infections attributed to encapsulated bacteria. Patients who complete the VALIANT study are eligible to enrol into the VALE long-term extension study. Sobi plans to submit a marketing application with the European Medicines Agency in 2025. Apellis plans to submit a supplemental new drug application to the U.S. Food and Drug Administration in early 2025. About the VALIANT study The VALIANT phase 3 study (NCT05067127) is a randomised, placebo-controlled, double-blinded, multi-centre study designed to evaluate pegcetacoplan efficacy and safety in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include adolescent and adult patients, with native and post-transplant kidneys. Study participants were randomised to receive pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomised controlled period, patients are able to proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (uPCR) at Week 26 compared to baseline. About C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3c deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. There are no treatments that target the underlying cause of these diseases. Approximately 50% of people living with C3G and IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis.1 Additionally, 90% of patients who previously received a kidney transplant will experience disease recurrence.2 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.3 About pegcetacoplan in rare diseases Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally. About the Sobi and Apellis collaboration Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED