Cefepime hydrochloride

SHANGHAI, March 25, 2025 /PRNewswire/ -- Everest Medicines (HKEX 1952.HK, "Everest", or the "Company"), a biopharmaceutical company focused on the discovery, clinical development, manufacturing and commercialization of innovative medicines and vaccines, today announced its financial results for the full year ended December 31, 2024, along with a corporate update. "In 2024, Everest continued to strength the execution of our 'Dual-Engine' strategy, having evolved from a license-in model to a balanced approach that combines in-house discovery with global partnerships and in-licensing, with a focus on high-value programs and the development of first-in-class or best-in-class assets," commented Rogers Yongqing Luo, CEO of Everest Medicines. "Our total revenue reached RMB706.7 million, representing a 461% year-over-year increase and exceeding our revenue guidance of RMB700 million. Operating expenses as a percentage of revenue decreased significantly by 562%. Notably, our non-IFRS net loss narrowed by 25%, and our gross margin, excluding non-cash items, reached 83%. We ended the year with a strong cash balance of RMB1.6 billion. Importantly, we achieved commercial-level profitability for the full year of 2024. The highlight of our commercial execution in 2024 was the successful launch of NEFECON® in mainland China in May, marking the beginning of a new era in the treatment of IgA nephropathy (IgAN). The product generated RMB353.4 million in revenue for the year, representing a year-over-year increase of 1,581%. NEFECON® was also included in China's National Reimbursement Drug List (NRDL), helping to address a significant unmet medical need among Chinese patients. In addition, it received regulatory approvals in Singapore, Hong Kong SAR, Taiwan region, and South Korea during 2024, expanding its regional footprint in Asia. We also delivered robust growth with XERAVA® (eravacycline), a first-in-class fluorocycline antibiotic, which generated RMB352.8 million in revenue in 2024, representing a 256% year-on-year increase. This strong performance was driven by its differentiated clinical profile and rising market demand. In our autoimmune portfolio, we achieved significant progress with VELSIPITY® (etrasimod), which received regulatory approvals in Macau SAR and Singapore in April 2024. The product was subsequently commercially launched in December in Guangdong province under the 'Hong Kong and Macau Medicine and Equipment Connect' policy. In parallel, New Drug Applications (NDAs) for VELSIPITY® have been officially accepted in both mainland China and Hong Kong in December 2024." "We continued to advance our pipeline of assets with global rights, focusing on creating value in high-potential therapeutic areas. EVER001, a covalent reversible BTK inhibitor, delivered positive results from the preliminary analysis of our Phase 1b/2a trial and is progressing steadily through global development. We also achieved a major milestone by advancing our mRNA platform from early-stage research into global clinical development, underscoring both our innovation capabilities and the strategic potential of our AI-powered mRNA technology. EVM16, our personalized therapeutic mRNA cancer vaccine, completed first patient dosing in an investigator-initiated clinical trial (IIT) in March 2025. EVM14, our off-the-shelf tumor-associated antigen (TAA) vaccine, received Investigational New Drug (IND) approval from the U.S. FDA, and we plan to submit an IND application to China's NMPA in the first half of this year. The in vivo CAR-T program is expected to achieve preclinical candidate milestone later this year. These programs are supported by our commercial-scale, GMP-compliant manufacturing facility in Jiashan, Zhejiang, which provides integrated R&D, production, and commercialization capabilities across mRNA and other pipeline platforms. In 2025, we will continue to enhance our commercial excellence and advance our first-in-class and best-in-class assets with global rights to maximize synergies. A key priority will be accelerating the sales of NEFECON®, leveraging its unique position as the only approved IgAN therapy in China and successfully included in the NRDL. With global rights to EVER001, we will actively explore partnership opportunities outside of China to leverage international expertise and optimize commercial value, while further enhancing Everest's global visibility and presence. Our 'Dual-Engine' strategy has entered a new chapter. Under the Board's guidance, Everest will leverage its established efficient commercial platform to solidify our position in key therapeutic areas, advance innovation on our mRNA platform, and strive to become a leading biopharma in the Asia-Pacific region by 2030—delivering greater value for shareholders and breakthrough therapies for patients worldwide." Luo concluded. Recent Key Product Highlights and Anticipated Milestones RENAL PRODUCTS PORTFOLIO Nefecon® 2024 - In March 2024, the Company's partner Calliditas Therapeutics AB ("Calliditas") (which was acquired by Asahi Kasei Corporation in September 2024 ) announced the U.S. FDA has granted an Orphan Drug exclusivity period of seven years for NEFECON®, expiring in December 2030 based on Calliditas obtaining full approval for this drug product in December 2023. - In March 2024, the Singapore Health Sciences Authority ("HSA") approved NEFEGAN®, known in other Everest territories as NEFECON®, for the treatment of primary IgAN in adults at risk of disease progression. Singapore marks the third region in Everest territories that received NDA approval after Macau and mainland China. - In April 2024, Calliditas published additional sub-analyses of NEFECON®'s Phase 3 NefIgArd study. One of the sub-analyses showed that during the 2-year period (9 months of treatment with NEFECON®, followed by a 15-month observation period after discontinuation), significant benefits in estimated glomerular filtration rate ("eGFR") were observed regardless of baseline UPCR levels (<0.8 g/g or ≥0.8 g/g). This study also showed that patients with UPCR <0.8 g/g who received NEFECON® achieved an eGFR slope of -0.25 mL/min/1.73 m3 per year, supporting the treatment target of <1 ml/min/1.73 m3 per year with the objective to avoid kidney failure in their lifetime. Another sub-analyses demonstrated that, irrespective of baseline UPCR levels and use of rescue medication, time to confirmed 30% eGFR reduction or kidney failure was significantly delayed. - In April 2024, Calliditas announced that the global open-label extension (OLE) study to the Phase 3 NefIgArd study showed a treatment response consistent with the NefIgArd study for the endpoints of urine protein to creatinine ratio ("UPCR") and eGFRat 9 months in all IgAN patients, including those who had previously received NEFECON® in the NefIgArd study. The safety data after 9 months of treatment or retreatment with Nefecon® in patients who completed the NefIgArd study were consistent with previously reported safety data. - In May 2024, the Hong Kong Department of Health had approved NEFECON® for the treatment of primary IgAN in adults at risk of disease progression. Hong Kong marks the fourth region in Everest territories where Nefecon® has received NDA approval after Singapore, Macau and mainland China. - In May 2024, Everest announced the successful launch of NEFECON® in mainland China, which has the highest prevalence of primary glomerular diseases in the world. The commercialization of NEFECON® in the Chinese market represents a significant milestone for Everest and a breakthrough for IgAN patients in China. The first prescription of NEFECON® was issued through an internet hospital, enhancing speed and convenience of delivering medication to patients and improving accessibility to the treatment. - In June 2024, Calliditas announced an additional efficacy analysis of NEFECON® in IgAN at the 61st European Renal Association Congress. An anchored matching-adjusted indirect comparison (MAIC) was performed to estimate the relative effect of NEFECON® or sparsentan on the absolute eGFR change from baseline at 9, 12, and 24 months, with common comparators of optimized renin-angiotensin system inhibition for NefIgArd and irbesartan from the phase 3 PROTECT study. Using patient-level data from NefIgArd and trial-level data from PROTECT, the study demonstrated that treatment with NEFECON® 16 mg/day for 9 months was associated with greater eGFR benefit compared with continuous treatment with sparsentan 400 mg/day over 2 years. - In July 2024, China's NMPA accepted the submission of a supplemental New Drug Application seeking full approval of NEFECON® based on the complete clinical data from the global Phase 3 NeflgArd study. - In September 2024, NEFECON® was included in the "KDIGO 2024 Clinical Practice Guideline for the Management of Immunoglobulin A Nephrophthy (IgAN) and Immunoglobulin A Vasculitis (IgAV)" draft for public review. The draft guideline notes that NEFECON® is the only treatment to date proven to reduce the levels of pathogenic forms of IgA and IgA immune complexes and recommends treatment with a 9-month course of NEFECON® for IgAN patients who are at risk of progressive kidney function loss. - In October 2024, the Taiwan Food and Drug Administration approved NEFECON® indicated "to reduce the loss of kidney function in adult patients with primary IgAN who are at risk for disease progression". Taiwan was the fifth region in Everest Medicines' authorized area to approve NEFECON® after Macau, Mainland China, Singapore, and Hong Kong. - In October 2024, "Kidney 360" magazine published the complete two-year subpopulation data from Chinese patients in the Phase 3 NefIgArd clinical trial of NEFECON® under the title "Efficacy and Safety of Nefecon in Patients with Immunoglobulin A Nephropathy from Mainland China: 2-Year NefIgArd Trial Results". The article states that during the 2-year treatment and observation period, the Chinese subpopulation data showed improvements in kidney protection, proteinuria reduction, and microhematuria that were numerically greater than the outcomes of global population. - In October 2024, additional analyses of the NefIgArd study for NEFECON® were presented at the 2024 American Society of Nephrology Kidney Week (ASN Kidney Week 2024). One of the analyses showed that the ability of NEFECON® to specifically modulate pathogenic Gd-IgA1 production in the GALT while leaving systemic IgA responses and total IgA and IgG levels unchanged, supports its use as a generally well tolerated, targeted, locally-acting treatment option for IgAN. Another analysis demonstrated that real-world use of NEFECON® was well-tolerated and can reduce loss of kidney function in patients who receive ≥9 months of treatment. - In November 2024, NEFECON® received full approval from the Ministry of Food and Drug Safety ("MFDS") in South Korea, indicated for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.8 g/g). - In November 2024, NEFECON® was included in NRDL in mainland China which takes effect on January 1st, 2025. NEFECON®'s inclusion in the NRDL acknowledges its significant clinical value in improving patient care and with approximately 5 million IgAN patients in China and over 100,000 newly diagnosed patients annually, there is a significant unmet clinical demand in the country. - In December 2024, the first prescription for NEFECON® was issued at Hong Kong Gleneagles Hospital, marking a new era for the targeted treatment of IgAN in the region. It represents the third commercial launch of NEFECON® in the year, following its launch in Mainland China and Singapore. 2025 - On January 1, 2025, with the official implementation of the latest update of NRDL, NRDL pricing now applies to NEFECON® which will benefit more IgAN patients. NEFECON® is the only approved treatment for primary IgAN in adults at risk of disease progression in China. - In March 2025, the HSA has granted full approval of NEFEGAN® indicated "to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.8 g/g)". - We expect to commercially launch NEFECON® in Taiwan region and South Korea in 2025. - We expect to receive NEFECON® full approval from NMPA in 2025. - We expect inclusion of NEFECON® in the KDIGO 2025 guidelines as well as in the first Chinese guideline for IgAN in 2025.® EVER001 is a next-generation covalent reversible Bruton's tyrosine kinase (BTK) inhibitor with potentially best-in-class characteristics for the treatment of autoimmune renal diseases. Compared to covalent irreversible BTK inhibitors, EVER001 offers improved selectivity while maintaining high potency, thereby potentially avoiding many of the side effects associated with earlier-generation BTK inhibitors. Everest Medicines holds global rights to EVER001 for the treatment of renal diseases. 2024 - In December 2024, Everest announced positive results in the ongoing Phase 1b/2a clinical trial for the treatment of pMN with EVER001. In an analysis of the data available as of September 13th, 2024, we observed that of the patients in the low-dose cohort who have completed 36 weeks of treatment, 9 out of 11 (81.8%) achieved overall clinical remission and 10 out of 11 (91%) achieved immunological complete remission (ICR). In the high dose cohort, 6 out of 7 (85.7%) patients achieved overall clinical remission and all patients achieved ICR by week 24. EVER001 was generally safe and well tolerated. No clinically significant adverse events typically associated with earlier-generation BTK inhibitors, such as bleeding, arrhythmia, severe infection, leukopenia, thrombocytopenia, or severe liver function impairment, were reported. 2025 - We expect to report one-year follow up data of the Phase 1b/2a clinical trial in 2025. INFECTIOUS DISEASE PORTFOLIO XERAVA® (eravacycline) 2024 - In January 2024, eravacycline's clinical breakpoint was officially approved by the Expert Committee of the National Health Commission on Antimicrobial Susceptibility Testing and Standard Research (ChinaCAST) for clinical use in China. - In November 2024, data on eravacycline (XERAVA®), were presented in Los Angeles, California at IDWeek 2024. The first study evaluated the in vitro antimicrobial activity of eravacycline against Carbapenem-resistant Acinetobacter baumannii (CRAB). Using the recently approved, ChinaCAST breakpoint of 1 µg/mL the results showed a high susceptibility rate of eravacycline against CRAB. The second study evaluated the in vitro activity of eravacycline against 23,127 global clinical isolates of major Gram-positive and Gram-negative bacteria, including drug-resistant strains collected from various regions such as Asia, Europe, and North America from 2018 to 2022. Since its approval in 2018, eravacycline has consistently maintained a high level of susceptibility against clinically relevant pathogens across diverse geographic regions and infection sites which supports eravacycline's use in treating complicated intra-abdominal infections caused by both Gram-negative and Gram-positive bacteria. - In November 2024, in a real-world clinical evaluation led by the National Health Commission's Expert Committee on Clinical Use of Antimicrobials and Evaluation of Antimicrobial Resistance, the final report showed that the overall efficacy rate of eravacycline treatment for 3 days was 91.1%, and the overall treatment efficacy rate at the end of treatment was 90.1%. Cefepime-taniborbactam is an investigational agent that is a combination of cefepime, a fourth-generation cephalosporin, and the novel beta-lactamase inhibitor (BLI), taniborbactam, that exhibits broad coverage of both serine- and metallo-beta-lactamases. In combination with cefepime, taniborbactam is under development as a new treatment option for patients with serious bacterial infections caused by difficult-to-treat drug resistant gram-negative pathogens, including carbapenem-resistant Enterobacterales (CRE) and carbapenem-resistant or multidrug-resistant Pseudomonas aeruginosa (CRPA/MDR-PA). 2024 - In February 2024, The New England Journal of Medicine ("NEJM") published the results of the CERTAIN-1 Phase 3 clinical study of the investigational agent cefepime-taniborbactam for the treatment of adult patients with complicated urinary tract infections ("cUTI"), including acute pyelonephritis. The results showed that cefepime-taniborbactam was superior to meropenem for the treatment of complicated UTI that included acute pyelonephritis, with a similar safety profile to meropenem. 2025 - We expect to submit an NDA for cefepime-taniborbactam in cUTI to China NMPA in 2025. AUTOIMMUNE DISEASE PORTFOLIO VELSIPITY® (etrasimod) 2024 - In February 2024, our licensing partner Pfizer Inc. announced that the European Commission ("EC") granted marketing authorization for VELSIPITY® in the European Union to treat patients 16 years of age and older with moderately to severely active UC who have had an inadequate response, lost response, or were intolerant to either conventional therapy, or a biological agent. - In March 2024, the Pharmaceutical Administration Bureau of the Macau Special Administrative Region, China, accepted Everest's NDA for VELSIPITY® for the treatment of adult patients with moderately to severely active UC. In April, the bureau approved the NDA, marking the first approval of VELSIPITY® in Everest territories. - In July 2024, Everest announced positive topline data results of the maintenance period from a multi-center Phase 3 clinical trial of VELSIPITY® in Asia for the treatment of subjects with moderately-to-severely active UC. The data of maintenance treatment confirmed that, after 40 weeks of treatment of 2mg once-daily VELSIPITY or placebo, VELSIPITY® demonstrated significant clinical and statistical improvements over placebo in the primary and all key secondary endpoints (p<0.0001), and other secondary endpoints (including mucosal healing and endoscopic normalization, both p<0.0001). The safety profile of VELSIPITY® was consistent with previous studies, with no new safety signals observed. - In October 2024, Everest announced that, through the "Hong Kong and Macau Medicine and Equipment Connect" policy, VELSIPITY® was officially been approved for patients with moderately to severely active UC by the Guangdong Provincial Medical Products Administration and the first prescription for VELSIPITY® was written at Foshan Fosun Chancheng Hospital in Guangdong in December. VELSIPITY® is now Everest's third commercialized product. - In November 2024, the full induction period data results from a multi-center Phase 3 clinical trial of VELSIPITY® in Asia on the treatment of moderately to severely active UC with etrasimod was presented in an oral presentation at the 32nd United European Gastroenterology Week (UEGW 2024). The induction period results showed that all primary and key secondary efficacy endpoints in the etrasimod treatment group achieved statistically significant and clinically meaningful improvements compared to the placebo group. The treatment differences in clinical remission, endoscopic improvement, and clinical response rates were 20.4%, 28.6%, and 32.0%, respectively (all P-values <0.0001). Patients treated with etrasimod achieved clinically meaningful and statistically significant improvements in mucosal healing (P<0.0001) and endoscopic normalization (P=0.0003). - In December 2024, the Department of Health of the Government of the Hong Kong Special Administrative Region, China, accepted Everest's NDA for VELSIPITY® for the treatment of adult patients with moderately to severely active UC. - In December 2024, the first prescription of VELSIPITY® in Macau was issued at Kiang Wu Hospital and VELSIPITY® was included in the Catalog of Pharmaceutical and Medical Devices Imported from Hong Kong and Macau for the Nine Municipalities in Guangdong Province within the Guangdong-Hong Kong-Macau Greater Bay Area, published by the Guangdong Provincial Medical Products Administration and Health Commission of Guangdong Province. - In December 2024, the NMPA of China accepted the NDA for VELSIPITY® for the treatment of patients with moderately to severely active UC. We expect to receive NDA approval in 2026. 2025 - In February 2025, Everest announced the presentation of maintenance data from its multi-center Phase 3 clinical trial of etrasimod in Asia at the 20th European Crohn's and Colitis Organization Congress (ECCO 2025). The results demonstrated that treatment with etrasimod 2 mg resulted in a clinically meaningful and statistically significant improvement in the primary and all secondary endpoints at the end of maintenance period. A statistically significant greater proportion of etrasimodtreated patients achieved clinical remission at Week 40 compared with placebo. A statistically significant greater proportion of etrasimod-treated patients achieved endoscopic improvement and clinical response at week 40 compared with placebo. Other secondary endpoints of mucosal healing, endoscopic normalization, and histological remission also significantly favored patients treated with etrasimod compared with placebo. Notably, mucosal healing as measured by a central read endoscopic subscore ≤1 (excluding friability) with a Geboes Index score <2.0, was achieved in 51.9% of the etrasimod treated patients compared to 8.8% in the placebo group (2-sided p-value <0.0001). The safety profile of etrasimod during the maintenance period was consistent with previous studies, with no new safety findings observed. - In March 2025, Everest announced the launch of a construction project for etrasimod's local production at Jiashan factory. The project is expected to achieve an annual production capacity of 50 million tablets upon full operation. The planned supply regions include mainland China, Hong Kong, Macau, Taiwan, South Korea, and Singapore, covering Everest's licensing regions in Asia. - We expect to receive VELSIPITY® NDA approval in UC in Hong Kong in 2025. - We expect to submit NDA for VELSIPITY® in UC in Taiwan region and South Korea in 2025. - We will continue to expand VELSIPITY®'s accessibility in the Greater Bay area through the "Hong Kong and Macau Medicine and Equipment Connect" policy in 2025. Zetomipzomib is a novel, first-in-class, selective immunoproteasome inhibitor currently being evaluated for immune- mediated disorders. It was licensed from Kezar Life Sciences in 2023. Everest and Kezar collaborate on the development of zetomipzomib in autoimmune diseases such as autoimmune hepatitis. - In February 2024, Everest announced that the Center for Drug Evaluation ("CDE") of China's NMPA approved Kezar's IND application for initiation of the Phase 2b PALIZADE trial in China of zetomipzomib in patients with lupus nephritis ("LN"). - In July 2024, Everest announced that the first patient had been dosed in China with zetomipzomib in the global Phase 2b PALIZADE trial for the treatment of active LN. - In September 2024, PALIZADE was placed on clinical hold following the recommendation of the Independent Data Monitoring Committee. In October, Kezar made the strategic decision to terminate the PALIZADE trial and focus clinical development efforts on zetomipzomib in autoimmune hepatitis (AIH). - Zetomipzomib is currently being tested in PORTOLA, a placebo-controlled, randomized, double-blind Phase 2a clinical trial evaluating the efficacy and safety of the molecule in patients with AIH. AIH is a rare chronic disease in which the immune system attacks the liver and causes inflammation and tissue damage, severely impacting patients' physical health and quality of life. The study has completed enrollment of 24 patients, and Kezar expects to report topline results in 1H 2025. mRNA PLATFORM Everest has built end-to-end capabilities across its proprietary mRNA platform. Our R&D team is developing multiple mRNA-based therapeutic products including personalized cancer vaccines, tumor-associated antigen vaccines, immunomodulatory cancer vaccines, in vivo CAR-T program, as well as next generation lipid nanoparticle ("LNP") delivery systems to enhance cell-mediated immune response. Our mRNA manufacturing facility in Jiashan, Zhejiang Province in China is designed to comply with global good manufacturing practice ("GMP") standards and is able to produce at clinical- and commercial-scale. Everest owns full global intellectual property rights to its mRNA therapeutic programs. In 2024, the Company advanced its self-developed mRNA pipeline products, the first of which is EVM16, a novel personalized therapeutic mRNA cancer vaccine independently developed by Everest. It contains neoantigens with high immunogenicity potential, predicted based on the unique tumor mutations of each patient using Everest's proprietary AI-based neoantigen prediction algorithm, EVER-NEO-1. The vaccine is designed to encode dozens of tumor neoantigens. This platform will be an important part of our discovery efforts going forward. 2024 - In Feb. 2024, Everest announced the termination of the collaboration and license agreements with Providence Therapeutics Holdings Inc. Everest continues to develop its own therapeutic vaccine products utilizing the mRNA platform. - In August 2024, Everest announced the launch of an IIT for a personalized mRNA cancer vaccine, EVM16, under the study EVM16CX01, at the Peking University Cancer Hospital and Fudan University's Cancer Hospital. This trial is designed to assess the safety, tolerability, immunogenicity, and preliminary efficacy of EVM16 injection as a monotherapy and in combination with PD-1 antibody for patients with advanced or recurrent solid tumors. EVM16CX01 is the first-in-human ("FIH") trial for EVM16. 2025 - In March 2025, Everest announced that the first patient has been dosed with the Company's internally developed personalized mRNA cancer vaccine EVM16 at Peking University Cancer Hospital in the investigator-initiated clinical trial (IIT) EVM16CX01. - In March 2025, the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for EVM14, a TAA vaccine. EVM14 is Everest's first internally developed mRNA therapeutic vaccine to receive FDA IND approval, marking a significant milestone in the Company's efforts to develop innovative mRNA therapeutics in oncology. - We expect to submit IND application for EVM14 in China in 2025. PRODUCT PIPELINE Everest has built a strong product pipeline across renal, anti-infective, and autoimmune diseases that are all potentially first-in-class treatment or best-in-class assets. These programs encompass short-term, mid-term and long-term opportunities which are collectively expected to generate significant revenue growth for the Company and create value for its shareholders. Pipeline Outlook 2025 promises to be a year of important data readouts and significant advancement in our best-in-class programs which carry global rights. In 2024 we were pleased to report positive data from a preliminary analysis of a Phase 1b/2a clinical trial of EVER001, a novel BTK Inhibitor for the treatment of primary membranous nephropathy. We expect to advance this trial further in 2025 and plan to report one-year follow-up data in the second half of 2025. Among our self-developed programs, the first patients have been dosed in our IIT for personalized cancer vaccine and preliminary data on safety and immunogenicity are expected this year. In parallel, we have received FDA's IND approval for EVM14, our off-the-shelf TAA cancer vaccine. We also plan to submit IND application to China's NMPA for EVM14 in the first half of this year. It marks the first U.S. IND submission and approval for Everest, and the first IND applications for Everest's self-developed pipeline. With respect to our late-stage programs, we plan to submit the NDA for cefepime-taniborbactam to the NMPA in mainland China for the treatment of cUTI in 2025. We will also submit NDAs for VELSIPITY® in South Korea and Taiwan and expect to receive NDA approval for this product in Hong Kong in the second half of the year. Commercialization Our commercial organization continued to flourish in 2024 as we launched and grew three commercial products, NEFECON®, XERAVA®, and VELSIPITY®. In our renal franchise, our team successfully launched our leading drug product, NEFECON® in mainland China in May 2024. We employed a broad array of sales channels, including traditional in-person detailing of hospitals as well as the innovative channel of online prescribing, to meet the urgent and significant unmet needs of an estimated 5 million IgAN patients. Our 150-person sales team had penetrated more than 600-700 hospitals by the end-of 2024, which encompass over 60% of the addressable IgAN population. To further broaden IgAN patient access, NEFECON® was included in China's 2024 NRDL which went into effect on January 1, 2025. We expect this significantly increased affordability to drive rapid penetration of NEFECON® throughout its patient population. Another highlight for NEFECON® in 2024 was its inclusion in the "KDIGO 2024 Clinical Practice Guideline for the Management of Immunoglobulin A Nephrophthy (IgAN) and Immunoglobulin A Vasculitis (IgAV)" draft for public review. The draft guideline points out that NEFECON® is the only treatment to date proven to reduce the levels of pathogenic forms of IgA and IgA immune complexes and recommends treatment with a 9-month course of NEFECON® for patients who are at risk of progressive kidney function loss with IgAN. It also suggests that many patients may need either repeated 9-month treatment cycles or a reduced-dose maintenance regimen in order to produce a sustained clinical response in terms of proteinuria reduction or stabilization of eGFR. The safety and efficacy of repeated treatment was also supported by our partner Calliditas' global OLE study which showed consistent treatment response across endpoints of UPCR and eGFR at 9 months across all IgAN patients, including those who had previously received NEFECON® in the NefIgArd study. In our anti-infective portfolio, Everest continued to grow XERAVA® sales through deeper penetration of our covered core hospitals and we began partnering with Contract Sales Organizations to benefit patients outside of our core target hospitals, although we still anticipate our own commercial team to generate the majority of sales for XERAVA®. Eravacycline's clinical breakpoint was officially approved by ChinaCAST for clinical use in China, and more than 100 hospitals in China have already adopted the new breakpoint in 2024, facilitating wider use of the drug in clinical practice. Inclusion of eravacycline in the Catalogue of Hierarchical Management of Clinical Application of Antimicrobial Drugs in Shanghai, Beijing and Guangdong last year underscores recognition of the drug's clinical benefits by China's key opinion leaders. In addition, eravacycline was included into The Surgical Infection Society Guidelines on the Management of Intra-Abdominal Infection: 2024 Update and China's Clinical Diagnosis and Treatment Guidelines for Multidrug-resistant Bacterial Infections in Renal Transplantation, Infectious Diseases Society of America 2024 Guidance on the Treatment of Antimicrobial-Resistant Gram-Negative Infections and Chinese Expert Consensus on the Diagnosis and Treatment of Pneumonia in the Elderly (2024 Edition). Inclusion in these guidelines is expected to broaden physician awareness of XERAVA® while also encouraging broader product utilization. With the accumulation of clinical experience and the conduct of clinical studies by Chinese doctors, the following articles were published in 2024. These publications have significantly enhanced awareness and provided more references for broader clinical applications. We were pleased to have launched our first autoimmune therapeutic, VELSIPITY® which was approved in Macau in April and in Singapore in May, and successfully launched for sales in those regions. Under the "Hong Kong and Macau Medicine and Equipment Connect" policy ("the Connect Policy"), VELSITPITY® was qualified for early access in nine cities in Guangdong province and its first prescription was written at Foshan Fosun Chancheng Hospital in December. To date, VELSIPITY® has been made available at five designated medical institutions in Guangdong (First Affiliated Hospital of Sun Yat-sen University, Foshan Fosun Chancheng Hospital, Shenzhen Hospital of Southern Medical University, Guangzhou United Family Healthcare and Shenzhen Qianhai Shekou Free Trade Zone Hospital) to benefit patients in mainland China with urgent needs for advanced innovative medicines in UC. In 2025 we will continue to build on the strong commercial foundation established in 2024. In our renal franchise, we will work to rapidly ramp NEFECON® sales after its inclusion on the NRDL which makes the medicine much more affordable and broadly accessible to patients. We are targeting rapid progress in NRDL implementation through either hospital listing or dual-channel pharmacies in the first quarter of 2025. We are also aiming to expand our list of core coverage hospitals to about 800 in 2025, which includes over 80% of the potential NEFECON® market with about 200 sales representatives. Everest also anticipates official inclusion of NEFECON® in the 2025 revised Kidney Disease Improving Global Outcomes (KDIGO) guidelines as well as the first Chinese guideline for IgAN as a first-line treatment for IgAN patients. This is expected to help further grow our sales volumes by giving treating physicians an industry standard reference which instills in them more confidence in NEFECON®. While we focus on maximizing the NRDL opportunity in mainland China, we also plan to expand availability of this first-in-disease medication to more IgAN patients across other valuable Asian regions including Taiwan and South Korea. Within our anti-infective portfolio, we will continue to drive penetration of XERAVA® among our core hospitals by enhancing doctors' awareness, which would lead to more prescriptions at hospitals with significant growth potential. We will also drive early and appropriate use of XERAVA® to establish the medicine as a fundamental option in empirical multi-drug resistant bacteria infection therapy. In addition to our own sales network, we will further optimize promotion through the contract sales organization model in non-core markets where we believe XERAVA® prescription can be broadened. VELSIPITY® is our newest launch and in 2025, while we will facilitate the NDA review by NMPA with approval expected in 2026, we will also accelerate its availability in designated medical institutions under the Connect Policy for early access to VELSIPITY® in mainland China. Additionally, VELSIPITY®'s real-world study is planned in Greater Bay to provide physicians with more clinical guidance with the product. Discovery 2024 marked a remarkable year of progress in our mRNA platform as we started the first in-human trial with an investigator-initiated program for our personalized cancer vaccine, EVM16 in two top cancer hospitals in China. EVM16 is independently developed by Everest which contains neoantigens with high immunogenicity potential, predicted based on the unique tumor mutations of each patient using Everest's proprietary AI-based neoantigen prediction algorithm, EVER-NEO-1. The vaccine is designed to encode dozens of tumor neoantigens, and uses an LNP delivery system to efficiently deliver neoantigen-encoded mRNA in vivo, activating neoantigen-specific tumor-killing T cells and inhibiting tumor growth. In preclinical studies, vaccination with EVM16 stimulated a strong neoantigen-specific T cell response in different mouse models and showed significant tumor growth inhibition in the syngeneic B16F10 mouse melanoma model. EVER-NEO-1, the AI-based neoantigen prediction algorithm developed in-house by Everest, can identify the majority of reported tumor neoantigens, as well as several previously unreported neoantigens. Furthermore, the neoantigen prediction capability of EVER-NEO-1 was shown to be either comparable to or superior to leading industry algorithms in multiple independent validation studies. We are expecting a catalyst-rich 2025 with a preliminary human data readout from EVM16, for which we achieved first patient dosing in March. Additionally, we have received U.S. FDA's IND approval for EVM14, our off-the-shelf tumor-associated antigen vaccine, marking Everest's first internally discovered program and the first mRNA therapeutic cancer vaccine to be cleared for global clinical development. With IND application to China's NMPA expected in the first half of this year, this asset is designed to treat various cancers, including non-small cell lung cancer and head and neck cancer. In preclinical studies, EVM14 induced a dosedependent antigen-specific immune response in mice and significantly inhibited tumor growth in multiple syngeneic tumor models. Lastly, the in vivo CAR-T program is also expected to achieve preclinical candidate milestone later this year, which may create a pathway to future global partnership opportunities. Business Development With the promising data generated by EVER001, our most advanced product with global rights, we will look to potentially engage in out-licensing activities to create value for all our early-stage assets with global rights. For these assets, we plan to pursue global partnership opportunities and seek ways to maximize shareholder value with entities who have global expertise and deals that offer attractive economics. Our business development strategy for in-licensing remains focused on first-in-class or best-in-class assets in less crowded, high value therapeutic areas such as renal diseases, autoimmune disorders, and anti-infective categories. We intend to leverage the strength of our existing commercial platforms to pursue commercial-stage or near-commercial-stage assets in China to create operational synergies and build scale. In parallel, we plan to evaluate earlier-stage assets with global rights, where we can deliver clinical proof-of-concept results and build substantial shareholder value. Financial Highlights IFRS Numbers: Revenue for the year ended 31 December 2024 increased significantly by RMB580.7 million, or 461%, to RMB706.7 million, compared with RMB125.9 million for the year ended 31 December 2023. The revenue growth was primarily driven by the strong ramp up of XERAVA® sales and the successful launch of NEFECON® in mainland China. Additionally, in markets outside of Mainland China, XERAVA® sales continued to build in Hong Kong and Singapore, NEFECON® was successfully launched in Hong Kong and Singapore, and VELSIPITY® was first launched in Macau and made available in Guangdong province through the "Hong Kong and Macau Medicines and Equipment Connect" policy. Gross profit margin rose from 72.7% for the year ended 31 December 2023 to 74.6% for the year ended 31 December 2024. Excluding the amortisation of intangible assets, the gross profit margin increased from 79.9% in 2023 to 82.9% in 2024. The improvement was mainly due to the commercial launch of NEFECON® and the optimisation of product costs. R&D expenses for the year ended 31 December 2024 amounted to RMB528.0 million, decreasing slightly from RMB540.1 million for the year ended 31 December 2023. The Company remains committed to strategic R&D investments across various product pipelines to support long-term sustainable growth. General and administrative expenses increased by RMB84.9 million, from RMB165.2 million for the year ended 31 December 2023 to RMB250.1 million for the year ended 31 December 2024. This increase was primarily due to higher remuneration expenses and professional service expenses in order to support the Company's business expansion and continued pipeline growth. Distribution and selling expenses increased by RMB276.7 million from RMB231.4 million for the year ended 31 December 2023 to RMB508.1 million for the year ended 31 December 2024, primarily due to expansion of the commercial team and additional commercial activities, which helped the successful launch of new products and encouraged the growth of existing product sales. Our commercial operational efficiency increased as commercialization expenses-to-sales ratio decreased by 111.9%, with our continued efforts to build a more efficient and focused commercialization model. The ratio of total operating expenses (including general and administrative expenses, research and development expenses, and distribution and selling expenses) to sales decreased by 561.8%, showing operational efficiency improvement. Net loss for the year increased by RMB196.9 million from RMB844.5 million for the year ended 31 December 2023 to RMB1,041.4 million for the year ended 31 December 2024. This increase was primarily due to a one-time, non-recurring impairment loss from an intangible asset related to mRNA COVID-19 vaccines in the first half of 2024. Excluding this impairment loss of an intangible asset, net loss narrowed by RMB107.5 million, from RMB792.5 million for the year ended 31 December 2023 to RMB685.0 million for the year ended 31 December 2024. This was primarily due to the strong product sales and improvements in operational efficiency. Cash and cash equivalents and bank deposits amounted to RMB1,603.3 million as of 31 December 2024. Non-IFRS Measure: Adjusted loss for the year narrowed by RMB176.1 million, from RMB713.6 million for the year ended 31 December 2023 to RMB537.6 million for the year ended 31 December 2024, primarily excluding the one-time and non-recurring loss on impairment of an intangible asset, and non-cash expenses of share-based compensation and amortization of intangible assets. About Everest Medicines Everest Medicines is a biopharmaceutical company focused on discovering, developing, manufacturing and commercializing transformative pharmaceutical products and vaccines that address critical unmet medical needs for patients in Asian markets. The management team of Everest Medicines has deep expertise and an extensive track record from both leading global pharmaceutical companies and local Chinese pharmaceutical companies in high-quality discovery, clinical development, regulatory affairs, CMC, business development and operations. Everest Medicines has built a portfolio of potentially global first-in-class or best-in-class molecules in the company's core therapeutic areas of renal diseases, infectious diseases and autoimmune disorders. For more information, please visit its website at . Forward-Looking Statements: This news release may make statements that constitute forward-looking statements, including descriptions regarding the intent, belief or current expectations of the Company or its officers with respect to the business operations and financial condition of the Company, which can be identified by terminology such as "will," "expects," "anticipates," "future," "intends," "plans," "believes," "estimates," "confident" and similar statements. Such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, or other factors, some of which are beyond the control of the Company and are unforeseeable. Therefore, the actual results may differ from those in the forward-looking statements as a result of various factors and assumptions, such as future changes and developments in our business, competitive environment, political, economic, legal and social conditions. The Company or any of its affiliates, directors, officers, advisors or representatives has no obligation and does not undertake to revise forward-looking statements to reflect new information, future events or circumstances after the date of this news release, except as required by law. SOURCE Everest Medicines WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

2024’s novel drug approvals offered a wide array of new cancer drugs, rare disease treatments, a potential Alzheimer’s blockbuster, and about 10 fewer approvals than 2023. While 2022 and 2023 saw major approvals of new cell and gene therapies, 2024 did not stick as cleanly to a theme — instead featuring a grab-bag of long-awaited approvals. That included Eli Lilly’s amyloid-targeted Alzheimer’s drug, the first two treatments for the rare Niemann-Pick disease, and the first-ever mesenchymal stromal cell therapy. Several approvals in 2024 also deployed new ways to treat familiar diseases. For instance, Pfizer’s new hemophilia A or B drug Hympavzi is the first to target a particular protein in the blood-clotting process. Bristol Myers Squibb’s Cobenfy, approved for schizophrenia, is the first antipsychotic that targets cholinergic receptors and not dopamine receptors. What to watch for in 2025: more cancer drug approvals and major label expansions. And answers to questions like: Will the incoming Trump administration push to overturn some drug and vaccine approvals or EUAs? Or attempt to make the approval process more difficult? — Zachary Brennan Active ingredient: berdazimer sodium Indication: Molluscum contagiosum in adults and pediatric patients 1 year of age and older Snapshot : Ligand Pharmaceuticals scored FDA approval in January for berdazimer sodium, marketed as Zelsuvmi, to treat the viral skin infection molluscum contagiosum. The topical gel was approved for at-home treatment in both adult and pediatric patients ages 1 one year and older and is the first novel drug for these types of infections. The approval came after Ligand acquired the assets of Novan Pharmaceuticals, which had initially overseen the drug’s development and filed for bankruptcy in July 2023. — Lia DeGroot Active ingredient: lifileucel Indication: Adult patients with unresectable or metastatic melanoma who have been previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. Snapshot : An idea more than 40 years in the making finally went commercial in February with the approval of the first treatment based on tumor-infiltrating lymphocytes. Iovance’s bespoke therapy, Amtagvi, is made by dissecting out immune cells from a patient’s tumor, bolstering their numbers in a lab, and reinfusing the cells into a patient to help keep cancer at bay. The company is testing the approach in other cancers and as a frontline therapy for melanoma. The Biosecure Act threatened Iovance’s relations with the Chinese contract manufacturer WuXi, which helped make Amtagvi, but sales of the therapy were still off to a strong start with $54.9 million in total revenue by the end of the third quarter. — Ryan Cross Active ingredient: cefepime and enmetazobactam Indication: Complicated urinary tract infections in patients 18 years and older Snapshot : Exblifep, a combination of cefepime and enmetazobactam delivered intravenously, was approved in February for adults with complicated urinary tract infections, including pyelonephritis, or kidney infection. Enmetazobactam was discovered by Orchid Pharma, which licensed the ex-India rights to Allecra in 2013. — Nicole DeFeudis Active ingredient: letibotulinumtoxinA-wlbg Indication: To treat moderate-to-severe frown lines in adults Snapshot : Hugel Aesthetics’ Botox competitor Letybo was approved in March for the treatment of moderate to severe frown lines. Letybo was initially rejected in 2022 over manufacturing issues. The FDA turned it down again the following year, and Hugel refiled it in September 2023 after “completing a review of the facility management and supplementary work.” Despite a growing number of Botox rivals on the market, AbbVie’s chief commercial officer Jeffrey Stewart said at the 2024 JP Morgan Healthcare Conference that the company has been “incredibly efficient” at maintaining Botox’s lead. — Nicole DeFeudis Active ingredient: resmetirom Indication: Noncirrhotic non-alcoholic steatohepatitis (NASH) with moderate-to-advanced liver scarring in adults Snapshot : Madrigal Pharmaceuticals’ resmetirom won accelerated approval in March as the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), the debilitating liver disease also called NASH. The approval is likely to be seen as a crucial milestone in MASH, where the standard of care was previously diet and exercise. Rezdiffra, as the drug is marketed, is indicated for patients with stage 2 or 3 fibrosis who have not progressed to cirrhosis. — Nicole DeFeudis Active ingredient: tislelizumab Indication: Unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor Snapshot : BeiGene had licensed US rights to its PD-1 inhibitor twice during the development journey, once to Celgene and the other time to Novartis. By the time the biotech clinched FDA approval for Tevimbra, it became the sole owner again. While Novartis and BeiGene had first filed for approval more than two years prior, the FDA was unable to inspect BeiGene’s manufacturing site in China amid the pandemic, leading to a lengthy review delay. As with the many checkpoint drugs already on the market, BeiGene hopes esophageal cancer will be the first of many indications Tevimbra will eventually be approved to treat, both on its own and in combination with other medicines. The drug was first approved in China in 2019. — Amber Tong Active ingredient: atidarsagene autotemcel Indication: Children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy Snapshot : With a $4.25 million price tag, Lenmeldy is on the list of the world’s most expensive medicines. The gene therapy is for an ultra-rare and fatal disease called early-onset metachromatic leukodystrophy, which affects fewer than 40 children a year in the US. Lenmeldy was already approved under a different brand name in the EU and UK and was shown in clinical trials to keep the majority of children who received it alive and with normal cognitive development. Japanese pharmaceutical company Kyowa Kirin completed its purchase of the company that developed the therapy, Orchard Therapeutics, last January after Orchard had been struggling to build a sustainable business around commercializing these complex treatments for rare diseases with small numbers of patients. — Lei Lei Wu Active ingredient: aprocitentan Indication: Hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs Snapshot : The FDA approved Idorsia’s Tryvio for patients who aren’t adequately treated by current blood pressure medications. Johnson & Johnson backed away from its partnership on the drug in 2022, and while the pharma will still receive royalties, Idorsia needs a new partner to sustain its operations through this year. Idorsia said in November that it entered exclusive negotiations with an undisclosed third party for global rights to the drug, netting $35 million, and that it could lay off approximately 270 staffers. The drug carries a black box warning for embryo-fetal toxicity and is only available via a restricted safety program. The FDA only approved the lower dose that Idorsia studied in its key clinical trial. — Lei Lei Wu Active ingredient: givinostat Indication: Duchenne muscular dystrophy in patients 6 years of age and older. Snapshot : The FDA’s approval of Duvyzat marked the first commercial nonsteroidal drug for all genetic variants of Duchenne muscular dystrophy. The treatment, an oral histone deacetylase inhibitor developed by Italfarmaco, was approved for Duchenne patients aged 6 and older. Unlike genetic medicines for Duchenne, such as exon-skipping drugs or gene therapy, Duvyzat aims to counteract the downstream effects of missing dystrophin, a crucial muscle protein, rather than trying to restore it. In clinical trials, Duvyzat led to improved performance on a stair climb test that’s used to measure motor function in patients with muscle diseases. — Lei Lei Wu Active ingredient: sotatercept Indication: Pulmonary arterial hypertension in adults Snapshot : Merck’s $11.5 billion Acceleron deal appeared to pay off with the much-anticipated approval of its pulmonary arterial hypertension drug Winrevair. The rare disease medicine, which had a “successful resurrection” around mid-stage data in 2020, is expected to become a quick blockbuster treatment as the New Jersey pharma looks to fill the eventual patent gap with Keytruda. The under-the-skin drug, when combined with background PAH therapy, helped patients walk farther than patients on placebo and background treatment in a Phase 3 trial. “The increase in six-minute walk distance capability was gargantuan,” Eliav Barr, chief medical officer at Merck Research Laboratories, said in an interview with Endpoints News . — Kyle LaHucik Active ingredient: vadadustat Indication: Anemia from chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months Snapshot : After securing approval in more than 35 other countries, Akebia’s anemia drug finally got the FDA’s blessing in March, almost two years to the day after it was initially rejected by the agency. The once-daily Vafseo tablets were greenlit for adults who have been on dialysis for at least three months and indicated for anemia due to chronic kidney disease. The drug belongs to a class of medicines known as hypoxia-inducible factor prolyl hydroxylase inhibitors. They’ve had a precarious history. In 2021, FibroGen’s drug was also rejected by the FDA. GSK, meanwhile, won US approval in February 2023, but wound up ditching plans to seek European approval. Akebia doesn’t plan on making the drug available in the US until this month. — Kyle LaHucik Active ingredient: danicopan Indication: Add-on therapy for extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH) Snapshot : The oral factor D inhibitor Voydeya from AstraZeneca scored approval in the US in April 2024 as an add-on therapy to Soliris and Ultomiris for the treatment of a rare blood disorder called extravascular hemolysis. Voydeya is also being developed as a monotherapy for an advanced form of age-related macular degeneration. — Katherine Lewin Active ingredient: ceftobiprole Indication: Adults with Staphylococcus aureus bloodstream infections; adults with acute bacterial skin and skin structure infections; and adult and pediatric patients 3 months to less than 18 years old with community-acquired bacterial pneumonia Snapshot : The FDA approved Zevtera for staph infections, acute bacterial skin and skin structure infections, and community-acquired bacterial pneumonia. The approval came later than planned — Basilea initially said that it would submit the drug to the FDA in early 2023, but pushed back those plans after manufacturing issues. — Katherine Lewin Active ingredient: nogapendekin alfa inbakicept-pmln Indication: Adult patients with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors Snapshot : Anktiva was approved in combination with Bacillus Calmette-Guérin (BCG) to treat patients with a subtype of BCG-unresponsive non-muscle invasive bladder cancer. ImmunityBio was hit with an FDA rejection in May 2023, citing manufacturing issues with a third party. After the eventual approval, ImmunityBio received $100 million as part of a royalty deal and private placement with Oberland Capital. — Katherine Lewin Active ingredient: tovorafenib Indication: Children 6 months of age and older with low-grade glioma that has stopped responding to or returned after previous treatment Snapshot : Day One Biopharmaceuticals’ goal is to develop new drugs for children first — reversing the industry’s age-old practice of first winning approval of medicines in adults and then (and only on occasion) in children. The company’s first approved drug, Ojemda, is a targeted treatment called a BRAF inhibitor. It was approved for children 6 months of age and up with a form of slow-growing brain cancer called low-grade glioma. “We now have shown that you can develop drugs intentionally for children at the speed of biotech,” Day One’s then-R&D chief Sam Blackman told Endpoints around Ojemda’s approval. — Lei Lei Wu Active ingredient: pivmecillinam Indication: Uncomplicated urinary tract infections caused by susceptible isolates of Escherichia coli , Proteus mirabilis and Staphylococcus saprophyticus , in female patients 18 years and older Snapshot : In April, Utility Therapeutics’ Pivya became the first new treatment for uncomplicated urinary tract infections in more than two decades. The oral antibiotic induced a composite response in 62% of patients versus just 10% for placebo in one of Utility’s three registrational trials. The UK-based drugmaker bought the US rights to pivmecillinam — the active ingredient in Pivya — from Leo Pharma as part of a 2018 deal with undisclosed financial terms. — Ayisha Sharma Active ingredient: fidanacogene elaparvovec-dzkt Indication: Moderate-to-severe hemophilia B in adults Snapshot : Pfizer became the second company to nab FDA approval for a one-time gene therapy to treat hemophilia B. And just like the US market’s first entrant, CSL, Pfizer priced the treatment at an eye-popping $3.5 million. Pfizer’s Beqvez was found to eliminate bleeding in 60% of patients in an open-label Phase 3 study, compared to 29% of patients receiving prophylaxis with a factor IX inhibitor. While it won approval for Beqvez, Pfizer has taken a step back from the gene therapy field, selling early assets in 2023 and dropping its Duchenne gene therapy after a failed trial. — Lei Lei Wu Active ingredient: mavorixafor Indication: WHIM syndrome in patients 12 and older Snapshot : Xolremdi was approved for an ultra-rare immune disorder, WHIM, named for its symptoms: warts, hypogammaglobulinemia, infections, and myelokathexis. X4 Pharmaceuticals’ drug is a CXCR4 inhibitor that is meant to address the underlying cause of the disease and improve patients’ ability to fight infections. X4 is also exploring Xolremdi’s use in treating chronic neutropenia. — Lei Lei Wu Active ingredient: tarlatamab-dlle Indication: Extensive-stage small cell lung cancer with disease progression on or after platinum-based chemotherapy. Snapshot : Imdelltra’s approval marked a major milestone for bispecific T cell engagers, a class of antibody-based drugs that bring T cells to tumors in hopes that they will attack the cancer. Following Immunocore’s Kimmtrak, it became the second T cell engager approved to treat solid tumors. Imdelltra was approved for extensive-stage small cell lung cancer that progressed despite chemotherapy, and Wall Street analysts project that the therapy has blockbuster potential if it can move into earlier lines of treatment. — Lei Lei Wu Active ingredient: RSV vaccine Indication: Prevention of lower respiratory tract disease caused by respiratory syncytial virus (RSV) in individuals 60 and older Snapshot : Moderna expects its RSV vaccine, its first non-Covid approval, to play a significant role in the company’s return-to-growth strategy by 2026 and reverse the $4.7 billion loss it recorded in 2023. The first full RSV vaccine season (winter 2023-24) was highly successful for Moderna’s competitors, GSK and Pfizer, and analysts anticipate the market will grow to $10 billion over the next few years. — Max Gelman Active ingredient: imetelstat Indication: Adults with low- to intermediate- risk myelodysplastic syndromes with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents Snapshot : Geron’s imetelstat cleared an adcomm and then secured an FDA nod in June for certain anemic myelodysplastic syndrome patients who are dependent on blood transfusions. Branded as Rytelo, the telomerase inhibitor carries a $2,471 price tag for a 47 mg vial and costs $9,884 for a 188 mg vial. The approval came after a panel of experts voted 12 to 2 in March that the drug’s benefits outweigh its risks. — Ayisha Sharma Active ingredient: elafibranor Indication: Primary biliary cholangitis in combination with ursodeoxycholic acid in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA Snapshot : Ipsen’s $136 million bet on Genfit’s elafibranor paid off in June when the drug clinched US approval for primary biliary cholangitis, a rare disease that can lead to liver failure. The drug, sold as Iqirvo, is a first-in-class dual agonist of the alpha and delta isoforms of PPAR and could slow disease progression, according to Ipsen. PBC affects around 100,000 people in the US. — Ayisha Sharma Active ingredient: immune globulin intravenous, human-dira Indication: Primary humoral immunodeficiency (PI) in patients 2 or older Snapshot : Yimmugo was approved in June for patients 2 years and older with primary humoral immunodeficiency, a condition that affects the immune system and can leave patients at an increased risk of infections. Italy-based Kedrion Biopharma announced in July that it struck an agreement for the full commercialization and distribution of Yimmugo in the US. — Nicole DeFeudis Active ingredient: pneumococcal 21-valent conjugate vaccine Indication: For prevention of invasive pneumococcal disease and pneumococcal pneumonia. Snapshot : Merck’s 21-valent pneumococcal vaccine Capvaxive was specifically designed for adults. The strains covered by the vaccine were responsible for 84% of invasive pneumococcal disease in patients 50 years and older between 2018 and 2021, according to Merck. That includes 10 strains in common with Pfizer’s rival 20-valent shot Prevnar 20, plus 11 unique strains. — Nicole DeFeudis Active ingredient: sofpironium Indication: Primary axillary hyperhidrosis in adult and pediatric patients 9 years of age and older Snapshot : Botanix Pharmaceuticals unlocked a potential market of 10 million people in the US with the June approval of sofpironium for primary axillary hyperhidrosis, or excessive underarm sweat. Sold as a gel with the brand name Sofdra, Botanix launched an early patient experience program in the third quarter, followed by a broader launch in the fourth quarter, which is when it also expected its first revenues from the product. — Ayisha Sharma Active ingredient: crovalimab-akkz Indication: Adult and pediatric patients 13 years and older with paroxysmal nocturnal hemoglobinuria and body weight of at least 40 kg Snapshot : The FDA approval for Genentech’s C5 inhibitor crovalimab for people with paroxysmal nocturnal hemoglobinuria in June came with a significant caveat — the drug can only be given under a REMS pathway due to the serious risk of meningococcal infections. Two months after its US approval, crovalimab, sold as PiaSky, secured an EU green light for the rare disease. When left untreated, PNH causes intravascular hemolysis, thromboembolic events and cytopenia. — Ayisha Sharma Active ingredient: ensifentrine Indication: Maintenance treatment of chronic obstructive pulmonary disease in adult patients Snapshot : Verona Pharma launched its COPD drug Ohtuvayre in 2024, taking a step into a titan-filled arena. Known generically as ensifentrine, Ohtuvayre is a maintenance treatment for COPD, taken via nebulizer twice a day, that can be added to standard COPD therapies. Verona aims to carve out a niche for Ohtuvayre as an add-on treatment rather than competing directly with major pharma companies in COPD like GSK and AstraZeneca. — Lei Lei Wu Active ingredient: donanemab Indication: Alzheimer’s disease Snapshot : Eli Lilly has invested more than $8 billion over more than three decades in its search for an Alzheimer’s drug. It finally found success in July when the FDA approved Kisunla . Kisunla is the second amyloid-targeting antibody to get full FDA approval after Eisai and Biogen won approval for Leqembi last year. Experts once worried about Alzheimer’s drugs bankrupting Medicare, but sales of the drugs are off to a slower-than-expected start , partly due to the risk of potentially fatal brain bleeding and swelling — which occurred in about 1 in 300 people in Lilly’s study. Lilly hopes the drug will be safer and more effective when taken earlier, before symptoms of dementia begin. A large study testing that hypothesis is underway and is expected to wrap up in 2027. — Ryan Cross Active ingredient: deuruxolitinib Indication: Adults with severe alopecia areata Snapshot : Sun Pharma’s $576 million acquisition of Concert Pharmaceuticals in 2023 paid off with the approval of the JAK inhibitor Leqselvi in severe alopecia areata. Sun’s drug could compete against some big players, including Eli Lilly’s JAK inhibitor Olumiant and Pfizer’s Litfulo. However, the drug’s launch was delayed after Incyte, which makes a similar drug, secured an injunction against its sale in November. Sun said it planned to appeal. — Katherine Lewin Active ingredient: benzgalantamine Indication: Mild to moderate dementia of the Alzheimer’s type in adults Snapshot : Zunveyl is a new twist on an old pill that’s commonly used to help reduce some symptoms of Alzheimer’s disease, but potentially with fewer gastrointestinal side effects. The compound is a prodrug of galantamine, an acetylcholinesterase inhibitor approved in 2001 that blocks breakdown of a key neurochemical that brain cells use to communicate with each other. Alpha Cognition, a tiny Canadian company that won approval for Zunveyl in July based on bioavailability studies, said it will begin selling Zunveyl in early 2025. — Ryan Cross Active ingredient: afamitresgene autoleucel Indication: Unresectable or metastatic synovial sarcoma in adults have received prior chemotherapy, are HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive, and whose tumor expresses the MAGE-A4 antigen as determined by FDA authorized companion diagnostic devices. Snapshot : Tecelra became the first cell therapy based on T cell receptors and the second cell therapy for a solid tumor cancer to win approval in the US in 2024. The therapy, known as afami-cel, is meant for a subsection of synovial sarcoma patients whose cancer has spread throughout their body or can’t be removed via surgery, and who have already received chemotherapy. Patients need to be tested for expression of a protein called MAGE-A4, which is found in a range of tumors, as well as an immune marker called HLA-A*02. — Lei Lei Wu Active ingredient: vorasidenib Indication: For adult and pediatric patients 12 years and older with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, sub-total resection, or gross total resection. Snapshot : Brain cancer is getting its targeted therapies, too. Servier’s Voranigo was approved for people with slow-growing gliomas that bear an IDH mutation. Patients are eligible for the drug after surgery for their brain tumor. Over 80% of grade 2 gliomas bear an IDH mutation, and the FDA said this was the first approved systemic drug for this subset of patients. In a clinical trial versus placebo, the treatment staved off cancer progression for a median 16.6 months longer, which physicians called a “striking” result. — Lei Lei Wu Active ingredient: denileukin diftitox-cxdl Indication: Adult patients with relapsed or refractory stage I-III cutaneous T cell lymphoma after at least one prior systemic therapy Snapshot : Following a delay from a CRL over manufacturing issues in 2023, Lymphir gained FDA approval in August. In order to support the launch of the treatment for a rare cancer, the company’s cancer subsidiary, Citius Oncology, combined with TenX Keane Acquisition. — Katherine Lewin Active ingredient: palopegteriparatide Indication: Hypoparathyroidism in adults Snapshot : Ascendis rebounded from an earlier FDA rejection and subsequent delay of its new application to snag FDA approval of hypoparathyroidism drug Yorvipath. It was already approved in Austria and Germany, generating €1.5 million ($1.6 million) in the first quarter. Yorvipath’s US entry replaces a void left by Takeda’s Natpara, some two years after it was pulled off the market. Ascendis said at the time of approval that it was completing manufacturing of the product and expecting the first batch of supply to be available in the first quarter of 2025. — Max Bayer Active ingredient: nemolizumab Indication: Prurigo nodularis in adults Snapshot : In August, Galderma secured first-in-class status with the US approval of its IL-31 antibody nemolizumab for prurigo nodularis. The green light is the first step in unlocking Nemluvio’s blockbuster potential, much of which lies in eczema, according to Galderma. The Swiss drugmaker earned a label expansion for moderate-to-severe atopic dermatitis in December, with Jefferies analysts projecting $2.1 billion in global peak sales for the drug. — Ayisha Sharma Active ingredient: seladelpar Indication: Primary biliary cholangitis in combination with ursodeoxycholic acid, in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA Snapshot : After acquiring seladelpar in the $4.3 billion purchase of CymaBay Therapeutics, Gilead snagged FDA accelerated approval to treat PBC, boosting its liver portfolio. The drug joins three others on the market, though its ability to treat itch symptoms gives Gilead hope that it can edge out competitors. That potential benefit has also contributed to Livdelzi’s price, which is slightly higher than the other two approved therapies. — Max Bayer Active ingredient: axatilimab-csfr Indication: Chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg Snapshot : Thanks to the help of Syndax Pharmaceuticals, Incyte was able to get another cGVHD treatment approval in August. It already markets Jakafi. The newly approved CSF-1 receptor-blocking antibody will also be studied alongside Jakafi and in combination with steroids. Incyte has said it plans to target the approximately 6,500 people who have failed two prior drugs. It also hopes to get the medicine greenlit for idiopathic pulmonary fibrosis. — Kyle LaHucik Active ingredient: lazertinib Indication: Locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test Snapshot : Lazcluze was approved in August in combination with Johnson & Johnson’s Rybrevant for certain non-small cell lung cancer patients with two common EGFR mutations. The approval puts the combination therapy in direct competition with AstraZeneca and its blockbuster Tagrisso, which has been approved in the same lung cancer population since 2018. Tagrisso also recently won a label expansion as part of a chemotherapy combination regimen in that indication. — Nicole DeFeudis Active ingredient: lebrikizumab Indication: Moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable, in adult and pediatric patients 12 years and older who weigh at least 40 kg Snapshot : Following an FDA rejection over manufacturing issues in 2023 (and no concerns with safety or labeling), Lilly scored approval for Ebglyss in September for moderate-to-severe eczema. Between two studies, 38% of patients on Lilly’s drug achieved clear or almost-clear skin at 16 weeks. — Katherine Lewin Active ingredient: arimoclomol Indication: Neurological manifestations of Niemann-Pick disease type C in adult and pediatric patients 2 and older, in combination with miglustat Snapshot : Zevra Therapeutics won approval for Miplyffa, known generically as arimoclomol, for people with the rare genetic disorder Niemann-Pick disease type C who are aged 2 years and older. Miplyffa’s approval came after the FDA previously rejected the drug in 2021, but Zevra returned to ask for approval with a reanalysis of clinical trial data that showed improvements in swallowing, which can be affected by the disease. FDA hosted an advisory committee ahead of its decision that voted in favor of the drug for the disease, a lysosomal storage disorder in which the body can’t properly recycle certain fatty substances. — Lei Lei Wu Active ingredient: levacetylleucine Indication: Neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients weighing ≥15 kg Snapshot : In September, IntraBio’s levacetylleucine became the second drug to clinch FDA approval for Niemann-Pick disease type C in the span of just one week. Branded Aqneursa, the oral drug can be taken up to three times daily with weight-dependent dosing. — Ayisha Sharma Active ingredient: xanomeline and trospium chloride Indication: Schizophrenia in adults Snapshot : Cobenfy’s approval gives Bristol Myers a significant foothold in the neuroscience and psychiatry fields, as it brings to market the first new schizophrenia drug in decades. It also validates the company’s $14 billion acquisition of Karuna, with execs predicting peak sales in the multibillion-dollar range. Researchers hope to broaden the use of Cobenfy in at least half a dozen other indications. — Max Gelman Active ingredient: inavolisib Indication: For adults with endocrine-resistant, PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy Snapshot : Roche’s Itovebi, which the company says is the first targeted therapy approved for people with HR-positive disease, got the green light from the FDA in October for patients with PIK3CA-mutated HR-positive HER2-negative breast cancer in combo with chemotherapy and Pfizer’s Ibrance. Patients on the drug saw progression-free survival of 15 months and a 58% overall response rate. — Katherine Lewin Active ingredient: marstacimab-hncq Indication: For routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years and older with: hemophilia A without factor VIII inhibitors, or hemophilia B without factor IX inhibitors Snapshot : Pfizer had a few successes in hemophilia in 2024, first with a gene therapy approval in April and then the nod for Hympavzi. The drug reduces a naturally occurring anticoagulation protein, unlike other treatments, which replace a clotting factor. But the results over routine prophylaxis were “modest,” TD Cowen analysts have said. Pfizer will likely have to rely on its packaging as a pre-filled, auto-injector pen to get traction in the market, the analysts said. — Kyle LaHucik Active ingredient: zolbetuximab Indication: In combination with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adults with locally advanced unresectable or metastatic human epidermal growth factor receptor 2-negative gastric or gastroesophageal junction adenocarcinoma whose tumors are Claudin18.2-positive, as determined by an FDA-approved test Snapshot : Vyloy, which targets a protein called Claudin18.2, scored an approval in combination with chemotherapy for the first-line treatment of locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma. The drug was approved in Europe and Japan as well, making it the first in its class to get worldwide approval. — Katherine Lewin Active ingredient: sulopenem etzadroxil and probenecid Indication: Uncomplicated urinary tract infections caused by the designated microorganisms Escherichia coli , Klebsiella pneumoniae , or Proteus mirabilis in adult women who have limited or no alternative oral antibacterial treatment options Snapshot : Iterum’s oral sulopenem was approved in October for certain patients with uncomplicated urinary tract infections (uUTIs). The drug, to be marketed as Orlynvah, is indicated specifically for patients who have limited or no alternative oral options. While the company had applied for broad use in adults with uUTIs, Iterum CEO Corey Fishman said the FDA’s label restrictions are “very consistent with how we’ve been thinking the whole time.” The agency had previously raised concerns that “inappropriate and/or extensive use” of an oral penem could result in cross-resistance with carbapenems, a class of antibiotics used for severe infections. — Nicole DeFeudis Active ingredient: obecabtagene autoleucel Indication: Adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia Snapshot : Autolus is challenging one of Gilead’s CAR-T cell therapies, Tecartus, with its recently approved treatment Aucatzyl in adult B-ALL. Aucatzyl will be listed at $525,000, making it pricier than Tecartus, but Autolus is hoping that its therapy’s safety profile will win over payers and providers. — Lei Lei Wu Active ingredient: eladocagene exuparvovec-tneq Indication: Adult and pediatric patients with aromatic 13 L-amino acid decarboxylase deficiency Snapshot : Known as Upstaza in Europe and Israel, PTC Therapeutics’ rare disease gene therapy finally won approval in the US in November under the brand name Kebilidi. The gene therapy is for AADC deficiency, an ultra-rare disease that impacts the nerve cells and brain. In the US, the therapy’s approval included children and adults across “the full spectrum of disease severity,” whereas the treatment’s previous approvals outside the country included patients with severe AADC deficiency. — Lei Lei Wu Active ingredient: revumenib Indication: Relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene translocation in adult and pediatric patients 1 year and older Snapshot : Revuforj, also known as revumenib, is the first drug approved in a new class of medicines called menin inhibitors. It’s used for an aggressive form of leukemia driven by rearrangement of the KMT2A gene. However, it carries a boxed warning for differentiation syndrome, a potentially life-threatening reaction to some blood cancer treatments. Syndax plans to seek approval in 2025 for leukemia patients with a different and more common genetic alteration called NPM1. — Nicole DeFeudis Active ingredient: zanidatamab Indication: Adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer (BTC), as detected by an FDA-approved test Snapshot : Jazz Pharmaceuticals’ HER2-targeted bispecific antibody zanidatamab will be the first of its class to hit the market for biliary tract cancer. The drug, to be marketed as Ziihera, won accelerated approval in November for previously treated patients who scored high on an immunohistochemistry test measuring HER2 expression. A Phase 3 confirmatory trial is underway in the first-line setting, which executives say could support both a full approval in the second-line setting and a supplemental application for frontline patients. — Nicole DeFeudis Active ingredient: acoramidis Indication: Cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization Snapshot : Attruby, approved in November, is expected to compete with Pfizer’s Vyndaqel franchise and Alnylam’s Amvuttra, should the FDA greenlight the latter ahead of its March 23 decision date. BridgeBio and Alnylam also traded blows as the drugs moved through the clinic (Vyndaqel was approved in 2019). Notably, Attruby’s label said the drug reduces the likelihood of both hospitalization and death, putting it on the same ground as Vyndaqel. — Max Gelman Active ingredient: zenocutuzumab-zbco Indication: Pancreatic adenocarcinoma or NSCLC that harbors NRG1 gene fusions and are advanced unresectable or metastatic Snapshot : The new cancer drug marked the first commercial product for Merus. But it will be Partner Therapeutics that actually commercializes the product in the US, after paying for rights in 2024. — Max Bayer Active ingredient: cosibelimab-ipdl Indication: Adults with locally advanced or metastatic cutaneous squamous cell carcinoma who cannot receive curative surgery or radiation Snapshot : A new PD-L1 drug in 2024? Apparently so. Checkpoint Therapeutics won approval late in the year for its checkpoint inhibitor cosibelimab, which will be branded as Unloxcyt. The drug won approval for a form of advanced skin cancer, where Merck’s Keytruda and Regeneron’s Libtayo are already approved. Checkpoint was part of an earlier wave of companies that wanted to disrupt how the blockbuster class of cancer drugs are priced by selling their treatments at a discount compared to currently approved options, but those aspirations have failed to become reality. Checkpoint now says it believes its drug can be priced “at or near parity with other approved anti-PD-(L)1 therapies,” and the company is seeking a partner or business deal before launching the drug commercially. — Lei Lei Wu Active ingredient: crinecerfont Indication: To be used together with glucocorticoids to control androgen levels in adults and pediatric patients 4 years and older with classic congenital adrenal hyperplasia Snapshot : Neurocrine’s second approved product marked the first new drug for congenital adrenal hyperplasia in 70 years. It also brings some of the work of the company’s late founder, Wylie Vale, to fruition: Vale, who died in 2012, identified the compound that this drug works to block. Analysts at William Blair believe the drug could reach nearly $1.5 billion in peak sales by 2030. — Max Bayer Active ingredient: ensartinib Indication: Adult patients with anaplastic lymphoma kinase-positive locally advanced or metastatic non-small cell lung cancer who have not previously received an ALK inhibitor Snapshot : Xcovery won its first-ever FDA approval for a first-line treatment of adults with anaplastic lymphoma kinase (ALK)-positive locally advanced or metastatic non-small cell lung cancer. The approval was based on a trial of 290 patients who had not previously received an ALK-targeted therapy, and FDA said Ensacove demonstrated a statistically significant progression-free survival improvement compared to Pfizer’s Xalkori. Xcovery is also testing Ensacove in other cancers. — Zachary Brennan Active ingredient: remestemcel-L-rknd Indication: Steroid-refractory acute graft-versus-host disease in pediatric patients 2 months and older Snapshot : Ryoncil took a winding path to approval, including two rejections and concerns from regulators about the design of its single-arm trial. An approval came more than four years after regulators accepted Mesoblast’s first application. But CEO Silviu Itescu believes it was worth the wait. Ryoncil is now the first mesenchymal stromal cell therapy to be approved by the FDA. It will be available for pediatric patients 2 months and older with acute graft-versus-host disease, a complication of allogeneic transplant in which donor cells attack the recipient. — Nicole DeFeudis Active ingredient: olezarsen Indication: As an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome Snapshot : Ionis’ antisense oligonucleotide olezarsen is the first approved treatment for familial chylomicronemia syndrome, a rare metabolic disorder that prevents the body from breaking down fats called triglycerides. The approval marks an important transition for Ionis, as Tryngolza will be the company’s first independent launch following years of partnerships. While FCS affects few people in the US, CEO Brett Monia hopes that a potential label expansion to a broader condition called severe hypertriglyceridemia could make Tryngolza a blockbuster. — Nicole DeFeudis Active ingredient: concizumab-mtci Indication: For routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A or B with inhibitors Snapshot : The anti-tissue factor pathway inhibitor Alhemo finally came good for Novo Nordisk at the tail end of 2024 following its rejection by the FDA in May 2023. It is the first daily subcutaneous treatment for hemophilia A or B patients with inhibitors — antibodies that neutralize clotting factors — to be delivered via a pen injector. It will compete with Pfizer’s similar weekly shot Hympavzi, but could reach more patients since Hympavzi is only approved for hemophilia A and B patients without inhibitors. — Elizabeth Cairns Active ingredient: vanzacaftor, tezacaftor, and deutivacaftor Indication: Cystic fibrosis in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene Snapshot : Alyftrek is Vertex’s “vanza triple” combination treatment for cystic fibrosis, a modulator of the CFTR gene. It’s the fifth such therapy Vertex has pushed across the finish line, but it comes with a boxed warning for potential drug-induced liver injury and failure. The treatment succeeded in its Phase 3 trials earlier this year, and Vertex used a priority review voucher to reduce the FDA’s review time to six months. — Max Gelman