On May 8, 2025 Nektar Therapeutics (Nasdaq: NKTR) reported financial results for the first quarter ended March 31, 2025 (Press release, Nektar Therapeutics, MAY 8, 2025, View Source [SID1234652753]).

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Cash and investments in marketable securities on March 31, 2025 were $220.7 million as compared to $269.1 million on December 31, 2024. Nektar’s cash and marketable securities are expected to support strategic development activities and operations into the fourth quarter of 2026.

"We are on track to report topline data in June from the Phase 2 study of rezpegaldesleukin in atopic dermatitis," said Howard W. Robin, President and CEO of Nektar. "These data will be followed by the topline data from the Phase 2 study of rezpegaldesleukin in patients with alopecia areata in December of this year. The results of both randomized studies will demonstrate the potential of rezpegaldesleukin to provide a new treatment paradigm for patients with these serious dermatological diseases. As a first-in-class T regulatory cell biologic, rezpegaldesleukin is poised to emerge as an important novel mechanism to treat millions of patients with chronic autoimmune disorders."

"We are also on track to complete our IND-enabling work for NKTR-0165, our unique antibody targeting the TNFR2 receptor, that has the potential to be developed as a treatment for multiple sclerosis, ulcerative colitis and vitiligo," continued Robin. "We are targeting the submission of the IND for NKTR-0165 at the end of this year. Additionally, we are making significant progress on our new bispecific antibody, NKTR-0166. This antibody incorporates a TNFR2 epitope with a validated antibody target and is advancing into preclinical studies."

Summary of Financial Results

Revenue in the first quarter of 2025 was $10.5 million as compared to $21.6 million in the first quarter of 2024. Revenue has decreased year over year because we no longer recognize product sales due to the sale of the Huntsville manufacturing facility in December 2024.

Total operating costs and expenses in the first quarter of 2025 were $55.0 million as compared to $57.1 million in the first quarter of 2024. Operating costs and expenses for the first quarter of 2025 decreased as compared to 2024 due to the elimination of cost of goods sold following the sale of the Huntsville manufacturing facility, partially offset by increases in R&D and G&A expenses.

R&D expense in the first quarter of 2025 was $30.5 million as compared to $27.4 million for the first quarter of 2024. R&D expense increased primarily due to an increase in expense for the development of rezpegaldesleukin, partially offset by a decrease in expense for the development of NKTR-255.

G&A expense was $24.3 million in the first quarter of 2025 and $20.1 million in the first quarter of 2024. G&A expense increased due to an increase in legal expenses, partially offset by decreases in facilities and stock-based compensation expenses.

In the first quarter of 2025, we began accounting for our investment in the new portfolio company, Gannet BioChem, under the equity method of accounting which calculates our gain or loss based on the change in our share of Gannet BioChem’s equity each quarter. This resulted in a $4.5 million non-cash loss from the equity method investment.

Net loss for the first quarter of 2025 was $50.9 million or $0.24 basic and diluted loss per share as compared to a net loss of $36.8 million or $0.19 basic and diluted loss per share in the first quarter of 2024. Excluding the $4.5 million non-cash loss from our equity method investment in Gannet BioChem, net loss, on a non-GAAP basis, for the first quarter of 2025 was $46.4 million or $0.22 basic and diluted loss per share.

Recent Business Highlights

● In April 2025, the European Hematological Association (EHA) (Free EHA Whitepaper) selected the abstract submitted by Nektar collaborators at the Fred Hutchinson Cancer Center entitled "Enhanced CAR T-cell Expansion and Durable Complete Responses with NKTR-255 Plus Lisocabtagene Maraleucel in Relapsed/Refractory Large B-cell Lymphoma" for oral presentation at the 30th annual EHA (Free EHA Whitepaper) Congress, being held in Milan, Italy from June 12-15, 2025.

● In February 2025, Nektar announced completion of target enrollment in the REZOLVE-AA 84-patient Phase 2b clinical trial of rezpegaldesleukin in severe-to-very severe alopecia areata.

● In February 2025, Nektar announced a new clinical trial agreement with TrialNet, an international clinical trial network at the forefront of diabetes research, to evaluate rezpegaldesleukin in a Phase 2 study of approximately 70 patients with new onset type 1 diabetes mellitus.

● In February 2025, the FDA granted Fast Track designation for rezpegaldesleukin for the treatment of adult and pediatric patients 12 years of age and older with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.

● In January 2025, Nektar announced completion of target enrollment in the REZOLVE-AD 396-patient Phase 2b clinical trial of rezpegaldesleukin in moderate-to-severe atopic dermatitis.

Conference Call to Discuss First Quarter 2025 Financial Results

Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time on May 8, 2025.

This press release and live audio-only webcast of the conference call can be accessed through a link that is posted on the Home Page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through June 8, 2025.

To access the conference call, please pre-register at Nektar Earnings Call Registration. All registrants will receive dial-in information and a PIN allowing them to access the live call.

On May 8, 2025 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported financial results for the first quarter of 2025, along with recent product highlights and corporate updates (Press release, Zai Laboratory, MAY 8, 2025, View Source [SID1234652774]).

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"In the first quarter, we continued to advance both our global pipeline and commercial business," said Dr. Samantha Du, Founder, Chairperson, and CEO of Zai Lab. "We are rapidly expanding our global portfolio, having recently presented promising data for two next-generation oncology therapies at AACR (Free AACR Whitepaper), and we look forward to presenting updated results for ZL-1310 (DLL3 ADC) at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting. We remain on track to initiate a pivotal study for ZL-1310 in SCLC this year, with the goal of securing FDA approval in 2027. In parallel, we are also exploring opportunities in first-line SCLC and other neuroendocrine tumors to unlock the full potential of this important global asset. On the commercial front, we continued to expand patient access for key products and are leveraging our existing infrastructure to support upcoming launches and our next wave of blockbuster opportunities. With a strong foundation in place, we are well positioned to drive growth and profitability, and to further our mission of becoming a leading global biopharmaceutical company."

"2025 marks a transformational year for Zai Lab, and we are executing against the key objectives we set at the start of the year," said Josh Smiley, President and COO of Zai Lab. "Following a seasonal slowdown of VYVGART early in the year, we saw patient volumes rebound in March and April, and we anticipate a return to strong sequential growth throughout the rest of the year. Looking ahead, our late-stage pipeline, including bemarituzumab and KarXT, is expected to fuel the next wave of topline growth alongside VYVGART. At the same time, we continue to strengthen our financial position, achieving a 20% year-over-year reduction in operating loss and a 25% year-over-year reduction in adjusted operating loss1, keeping us on track to achieve profitability1 in the fourth quarter of 2025. With a strong cash position,2 a growing commercial business, and an expanding global portfolio, Zai Lab is poised to capitalize on multiple upcoming catalysts and drive long-term shareholder value."

First Quarter 2025 Financial Results

•Product revenue, net was $105.7 million in the first quarter of 2025, compared to $87.1 million for the same period in 2024, representing 21% y-o-y growth, 23% y-o-y growth at constant exchange rate (CER). This increase was primarily driven by increased sales for VYVGART, ZEJULA, and NUZYRA.

–VYVGART and VYVGART Hytrulo were $18.1 million in the first quarter of 2025, compared to $13.2 million for the same period in 2024. Although quarter over quarter growth was impacted by seasonality, sales grew year over year driven by increasing market coverage and penetration since VYVGART’s launch in September 2023 and listing on China’s National Reimbursement Drug List (NRDL) for the treatment of generalized Myasthenia Gravis (gMG) effective January 1, 2024.

–ZEJULA was $49.5 million in the first quarter of 2025, compared to $45.5 million for the same period in 2024. ZEJULA sales remained strong as it continued to be the leading PARP inhibitor in hospital sales for ovarian cancer in mainland China.

–NUZYRA was $15.1 million in the first quarter of 2025, compared to $9.9 million for the same period in 2024. This growth was supported by increasing market coverage and penetration.

•Research and Development (R&D) expenses were $60.7 million in the first quarter of 2025, compared to $54.6 million for the same period in 2024. This increase was primarily due to upfront fees totaling $20.0 million for our license and collaboration agreements. Other R&D expenses decreased as a result of resource prioritization and efficiency efforts.

•Selling, General and Administrative expenses were $63.4 million in the first quarter of 2025, compared to $69.2 million for the same period in 2024. This decrease was primarily driven by decreased personnel costs as a result of resource prioritization and efficiency efforts.

•Loss from operations was $56.3 million in the first quarter of 2025, $37.1 million when adjusted to exclude non-cash expenses including depreciation, amortization, and share-based compensation. A reconciliation of loss from operations (GAAP) to adjusted loss from operations (non-GAAP) is included at the end of this release.

•Net loss was $48.4 million in the first quarter of 2025, or a loss per ordinary share attributable to common stockholders of $0.04 (or loss per American Deposit Share (ADS) of $0.45), compared to a net loss of $53.5 million for the same period in 2024, or a loss per ordinary share of $0.05 (or loss per ADS of $0.55). These decreases in net loss were primarily due to product revenue growing faster than net operating expenses.

•Cash and cash equivalents, short-term investments, and current restricted cash totaled $857.3 million as of March 31, 2025, compared to $879.7 million as of December 31, 2024.

Recent Pipeline Highlights

Below are key product updates since our last earnings release:

Oncology Pipeline

•ZL-1310 (DLL3 ADC):

–Second-Line+ Extensive-Stage SCLC (ES-SCLC): In April 2025, Zai Lab announced that updated data from an ongoing, global Phase 1a/1b clinical trial of ZL-1310 for the treatment of ES-SCLC, will be presented during a poster session at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The updated results will include additional patients and follow-up from the ongoing trial.

–Other neuroendocrine tumors: In April 2025, Zai Lab initiated a global Phase 1/2 study in patients with selected solid tumors, exploring its therapeutic potential beyond ES-SCLC.

•Tisotumab Vedotin (TIVDAK, Tissue Factor ADC): In March 2025, China’s National Medical Products Administration (NMPA) accepted the Biologics License Application (BLA) for TIVDAK for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after systemic therapy. Zai Lab will leverage its commercial footprint of ZEJULA in women’s cancer to accelerate patient access to this therapy in China if approved.

•Tumor Treating Fields (TTFields): In April 2025, Zai Lab partner Novocure announced that the additional results from the Phase 3 PANOVA-3 trial for pancreatic cancer will be presented as a late-breaking oral presentation at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting. Zai Lab participated in the study in Greater China (mainland China, Hong Kong, Macau and Taiwan, collectively) and plans to file for regulatory approval in China in 2025.

•Repotrectinib (ROS1/TRK): In April 2025, China’s NMPA accepted the supplemental New Drug Application for repotrectinib for patients with NTRK-positive solid tumors. Repotrectinib has the potential to become a next-generation TKI that can be used across a broad range of NTRK fusion-positive solid tumors for both TKI-naïve and TKI-pretreated patients.

•ZL-6201 (LRRC15 ADC): In April 2025, Zai Lab presented new data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025. The findings demonstrate that ZL-6201 efficiently internalizes within and kills tumor cells, while also exhibiting a strong bystander killing effect in the tumor microenvironment where the target is often expressed. Based on these findings, Zai Lab plans to initiate Investigational New Drug (IND)-enabling studies of ZL-6201 as a potential treatment for patients with sarcoma and other LRRC15-positive solid tumors, such as breast cancer and other malignancies, in 2025.

•ZL-1222 (PD-1/IL-12): In April 2025, Zai Lab presented data at the AACR (Free AACR Whitepaper) Annual Meeting 2025, marking the first public disclosure of this global asset. Findings from preclinical studies demonstrate its potent anti-tumor activity in both anti-PD-1 sensitive and resistant tumor models, with improved systemic safety. These results indicate its potential to benefit patients who are unresponsive or resistant to the current immuno-oncology therapies.

Immunology Pipeline

•Efgartigimod (FcRn): In April 2025, Zai Lab partner argenx announced the U.S. Food and Drug Administration (FDA) approved VYVGART Hytrulo prefilled syringe (PFS) for self-injection in gMG and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). It is the third administration option providing additional flexibility and convenience for patients. Zai Lab plans to submit the Chemical Manufacturing and Control (CMC) variation for PFS for these indications in China in 2025.

•ZL-1102 (IL-17 Humabody): Zai Lab has decided to discontinue the global Phase 2 clinical trial of ZL-1102 following a comprehensive review of the data from interim analysis from the first 40 enrolled participants and the subsequent recommendation of the independent Data and Safety Monitoring Board.

•Povetacicept (APRIL/BAFF): Zai Lab partner Vertex has completed enrollment of the interim analysis cohort in the global Phase 3 RAINIER study of povetacicept in IgA nephropathy. Zai Lab participated in the study in Greater China.

Anticipated Major Milestones in 2025

Upcoming Potential NMPA Submissions

•Bemarituzumab (FGFR2b): BLA submission in first-line gastric cancer.

•Tumor Treating Fields (TTFields): Marketing Authorization Application submissions in first-line pancreatic cancer.

•Efgartigimod (FcRn): submission for the CMC variation for PFS in gMG and CIDP.

Expected Clinical Developments and Data Readouts

Global Pipeline

ZL-1310 (DLL3 ADC)

•Second-Line+ ES-SCLC: Zai Lab to present updated data at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting on June 2, 2025. Zai Lab plans to initiate a pivotal study in the second half of 2025.

•First-Line ES-SCLC: Zai Lab to provide a data readout for dose escalation of ZL-1310 doublet in combination with atezolizumab.

ZL-1503 (IL-13/IL-31R)

•Zai Lab to provide a preclinical data update and advance into a global Phase 1 study in moderate-to-severe atopic dermatitis.

ZL-6201 (LRRC15 ADC)

•Zai Lab to advance into global Phase 1 development for patients with sarcoma and potentially other LRRC15-positive solid tumors, such as breast cancer and other malignancies.

Regional Pipeline

Bemarituzumab (FGFR2b)

•Zai Lab partner Amgen to provide data readout from the Phase 3 FORTITUDE-101 study of bemarituzumab combined with chemotherapy versus chemotherapy alone in first-line gastric cancer in the second quarter of 2025. Zai Lab participated in the study in Greater China.

•Zai Lab partner Amgen to provide data readout from the Phase 3 FORTITUDE-102 study of bemarituzumab plus chemotherapy and nivolumab versus chemotherapy and nivolumab in first-line gastric cancer in the second half of 2025. Zai Lab participated in the study in Greater China.

Xanomeline-Trospium (or KarXT) (M1/M4-agonist)

•Zai Lab partner Bristol Myers Squibb (BMS) to provide data readout from the Phase 3 ADEPT-2 study of KarXT in Alzheimer’s Disease Psychosis in the second half of 2025. Zai Lab participated in the study in Greater China.

Efgartigimod (FcRn)

•Seronegative gMG: Zai Lab partner argenx to provide topline results from the Phase 3 ADAPT-SERON study in seronegative gMG. Zai Lab participated in the study in Greater China.

•Lupus Nephritis (LN): Zai Lab to provide topline results from the Phase 2 study in LN.

Povetacicept (APRIL/BAFF)

•Primary Membranous Nephropathy (pMN): Zai Lab plans to partner with Vertex to execute the global pivotal Phase 2/3 study of povetacicept in pMN in Greater China.

Conference Call and Webcast Information

Zai Lab will host a live conference call and webcast today, May 8, 2025, at 8:00 a.m. ET (8:00 p.m. HKT). Listeners may access the live webcast by visiting the Company’s website at View Source Participants must register in advance of the conference call.

Details of registration links are as follows:

•For webcast: View Source

•For dial-in: View Source

All participants must use the link provided above to complete the online registration process in advance of the conference call. Dial-in details will be in the confirmation email which the participant will receive upon registering.

A replay will be available shortly after the call and can be accessed by visiting the Company’s website.

On May 8, 2025 Cidara Therapeutics, Inc. (Nasdaq: CDTX) (the Company), a biotechnology company using its proprietary Cloudbreak platform to develop drug-Fc conjugate (DFC) therapeutics, reported financial results for the first quarter ended March 31, 2025, and provided recent business updates (Press release, Cidara Therapeutics, MAY 8, 2025, View Source [SID1234652738]).

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"Coinciding with the end of the 2024-2025 flu season in the Northern Hemisphere, we have established April 30, 2025 as the data cutoff for the primary efficacy analysis of the 5,041 subjects dosed in the Phase 2b NAVIGATE clinical trial. We are expecting top-line results in late June of this year. The severity of the 2024-2025 flu season has provided an opportunity to discuss changes to the study’s statistical analysis plan with the U.S. FDA to evaluate the potential statistical significance of CD388 versus placebo," said Jeffrey Stein, Ph.D., president and chief executive officer of Cidara. "We believe CD388 is highly differentiated from vaccines and monoclonal antibodies and we believe these differences can significantly improve upon the protective efficacy against influenza in healthy, high-risk, and immunocompromised patients with a single dose per flu season."

Recent and Expected Corporate Highlights

Phase 2b NAVIGATE trial evaluating CD388 for prevention of seasonal influenza ongoing. Dosing of 5,041 subjects in the Phase 2b NAVIGATE study were completed in early December of 2024 across clinical sites in the U.S. and UK. This is a randomized, double-blind, controlled trial designed to evaluate the efficacy and safety of CD388 for the pre-exposure prophylaxis of seasonal influenza. Three CD388 dose groups, comprised of 150mg, 300mg and 450mg doses, and one placebo group were randomized and dosed in a 1:1:1:1 ratio at the beginning of the 2024-25 influenza season. Subjects were followed for efficacy through the end of April 2025 to monitor for breakthrough cases. Rates of laboratory and clinically confirmed influenza will be compared between subjects receiving the various single doses of CD388 or placebo.
Phase 2b NAVIGATE efficacy data expected in late June 2025. Due to the severity of the 2024-25 flu season, Cidara has the opportunity to discuss changes to the study’s statistical analysis plan with the U.S. FDA, to evaluate possible statistical significance of CD388 versus placebo. Assuming agreement with the FDA on the changes to the statistical analysis plan, we expect that data from this trial will enable dose selection for Phase 3 as well as the determination of statistically meaningful comparisons of each dose to the placebo arm of the study.
Published preclinical data of CD388 in Nature Microbiology in March 2025. The article, entitled "Drug-FC Conjugate CD388 targets influenza virus neuraminidase and is broadly protective in mice," highlighted the potential of CD388 as a potent, universal antiviral for influenza A and B prevention regardless of immune status. The full press release can be found here.
Presented two posters on CD388 in influenza at the 38th International Conference on Antiviral Research (ICAR) in March 2025. The Company’s presentations highlighted the study design, demographic information, and preliminary safety data from the ongoing Phase 2b NAVIGATE trial of CD388, as well as dose optimization models for its planned Phase 3 development program. The preliminary safety profile shows that CD388 is well-tolerated and the majority of injection site reactions are considered mild in severity.
Promoted Nicole Davarpanah to Chief Medical Officer and Corrina Pavetto to Senior Vice President, Clinical Development. In May 2025, Cidara announced the promotions of Nicole Davarpanah, M.D., J.D., to Chief Medical Officer and Corrina Pavetto to Senior Vice President, Clinical Development. Dr. Davarpanah and Ms. Pavetto have shown exceptional leadership in the execution of the Phase 2b NAVIGATE Study, the revision of its statistical analysis plan, and the design of the Phase 3 development and regulatory strategy for CD388. Their continued guidance and oversight will be invaluable for the successful development of CD388 as a novel option for the universal prevention of influenza.
First Quarter 2025 Financial Results

Cash, cash equivalents and restricted cash totaled $174.5 million as of March 31, 2025, compared with $196.2 million as of December 31, 2024.
Collaboration revenue was zero for the three months ended March 31, 2025, compared to $1.0 million for the same period in 2024. Collaboration revenue related to research and development (R&D) and clinical supply services provided to J&J Innovative Medicine, previously Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen), under our license and collaboration agreement with Janssen (the Janssen Collaboration Agreement). The Janssen Collaboration Agreement was terminated upon the effectiveness of our license and technology transfer agreement with Janssen on April 24, 2024.
R&D expenses were $24.6 million for the three months ended March 31, 2025, compared to $5.9 million for the same period in 2024. The increase in R&D expenses is primarily driven by higher expenses associated with our ongoing CD388 Phase 2b NAVIGATE study and higher one-time personnel costs, partially offset by lower nonclinical expenses associated with our Cloudbreak platform.
General and administrative (G&A) expenses were $6.2 million for the three months ended March 31, 2025, compared to $3.6 million for the same period in 2024. The increase in G&A expenses is primarily due to higher personnel costs, partially offset by lower legal costs.
Net loss from discontinued operations for the three months ended March 31, 2025 was zero, compared to net loss from discontinued operations of $2.1 million for the same period in 2024. On April 24, 2024, Cidara entered into an asset purchase agreement with Napp Pharmaceutical Group Limited (Napp), an affiliate of Mundipharma Medical Company, pursuant to which all rezafungin assets and related contracts were sold to Napp. All conditions of the sale were completed on April 24, 2024, and the financial results of rezafungin have been reported separately as discontinued operations.
Net loss for the three months ended March 31, 2025 was $23.5 million, compared to a net loss of $10.3 million for the same period in 2024.
First Quarter 2025 Conference Call and Webcast Details

Cidara Therapeutics management will host a conference call and webcast beginning at 5:00 pm ET / 2:00 pm PT today, May 8, 2025. A live webcast may be accessed here. The conference call can be accessed by dialing toll-free (800) 717-1738 or (646) 307-1865 (international). The passcode for the conference call is 90743.

A replay of the webcast will be archived on www.cidara.com for one year under the "Events & Presentations" tab in the Investors section of the company’s website.

On May 8, 2025 Novavax, Inc. (Nasdaq: NVAX) reported its financial results and operational highlights for the first quarter ended March 31, 2025 (Press release, Novavax, MAY 8, 2025, View Source [SID1234652754]).

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"I am pleased with the progress we have made in the first quarter on our corporate growth strategy," said John C. Jacobs, President and Chief Executive Officer, Novavax. "We remain focused on creating shareholder value as we advance our three priorities for the year – optimizing our partnership with Sanofi, advancing new and existing partnership opportunities and continuing the development of our early-stage organic pipeline."

First Quarter 2025 and Recent Highlights

Strategic Priority #1: Sanofi Partnership

•COVID-19 Biologics License Application (BLA) under review by the U.S. Food and Drug Administration (FDA). In April 2025, we received an information request for a postmarketing commitment (PMC) for a clinical trial. Discussions with the FDA regarding our proposed study design are ongoing and we believe our BLA is approvable upon alignment on the details of the PMC.

oAchievement of BLA approval triggers a $175 million milestone payment from Sanofi.
•Transfers of marketing authorization to Sanofi for U.S. and European Union (EU) markets, assuming approvals in each jurisdiction, are expected in Q4 2025 and trigger an additional $50 million in combined milestones from Sanofi.

Strategic Priority #2: Leverage our technology platform and pipeline to forge additional partnerships

•In April 2025, Novavax and Takeda Pharmaceuticals announced significantly improved terms for their partnership to support ongoing commercialization of Nuvaxovid in Japan. As part of this agreement, Novavax will receive a $20 million upfront payment, a payment related to the 2024-2025 season and is eligible to receive annual milestone payments plus royalties on net sales.
•In March 2025, Novavax signed an additional Material Transfer Agreement (MTA) for Matrix-M with a top tier pharmaceutical company, expanded the scope of the MTA signed in the fall to now include viral pathogens, and entered a preclinical collaboration with a new partner to explore the application and utility of Matrix-M with their cancer vaccine candidate.
•Completed enrollment and expect initial cohort data by mid-year for the Phase 3 trial for our COVID-19-Influenza Combination (CIC) and stand-alone seasonal influenza vaccine candidates to evaluate immunogenicity and safety in adults aged 65 and older. Novavax intends to partner these programs, and this trial reflects the material completion of investment by Novavax.
•Presented data at the April 2025 World Vaccine Congress on the potential of Novavax’s technology platform and Matrix-M adjuvant, which showcases attributes related to efficacy and tolerability. Highlights included utility of Matrix-M across multiple vaccine platforms and disease areas, underscoring breadth of potential partnership opportunities.

Strategic Priority #3: Advance our technology platform and early-stage pipeline

•In April 2025, announced preliminary results from the SHIELD-Utah study that showed Novavax’s COVID-19 Vaccine, Adjuvanted (2024-2025 Formula) targeting the JN.1 strain resulted in fewer and less severe reactogenicity symptoms, when compared with the Pfizer-BioNTech mRNA 2024-2025 vaccine.
•Continued advancement of early-stage preclinical research for H5N1 avian pandemic influenza, respiratory syncytial virus combinations, varicella-zoster virus (shingles) and Clostridioides difficile colitis vaccine candidates.
•Continued work on new potential Matrix formulations intended to improve upon and expand the utility of Matrix-M.

On May 8, 2025 Astrazeneca reported positive high-level results from the POTOMAC Phase III trial showed one year of treatment with Imfinzi (durvalumab) plus standard-of-care BCG induction and maintenance therapy demonstrated a statistically significant and clinically meaningful improvement in disease-free survival (DFS) for patients with high-risk non-muscle-invasive bladder cancer (NMIBC) compared to BCG induction and maintenance therapy alone (Press release, AstraZeneca, MAY 8, 2025, View Source [SID1234652777]).​

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The trial was not statistically powered to formally test overall survival (OS) however a descriptive analysis demonstrated no detriment.

More than 70% of bladder cancer patients are diagnosed with NMIBC, an early-stage cancer where the tumour is in the tissue that lines the inner surface of the bladder but has not invaded the muscle wall.1-2 About half of patients with NMIBC are classified as high-risk for disease progression or recurrence because of certain characteristics of their cancer, such as tumour grade, stage and specific tumour features.3

Maria De Santis, MD, Head of the Interdisciplinary Uro-Oncology Section at Charité Universitätsmedizin Berlin, Germany, and a principal investigator in the POTOMAC trial, said: "These exciting data show that adding one year of durvalumab to the current standard treatment significantly extends the time patients live without high-risk disease recurrence or progression. While most patients with non-muscle invasive bladder cancer are treated with curative intent, 80 per cent see their disease return and almost half may require life-altering surgery to remove the bladder, underscoring the urgent need to improve treatment."

Cristian Massacesi, Chief Medical Officer and Oncology Chief Development Officer, AstraZeneca, said: "The positive results for Imfinzi in the POTOMAC trial represent a significant advance that will potentially allow more patients with early-stage bladder cancer to benefit from this important immunotherapy. Building on the NIAGARA data, this outcome demonstrates our strategy of bringing novel therapies to patients with early-stage disease where there is the greatest potential for long-term benefit."

The safety and tolerability of Imfinzi plus BCG induction and maintenance therapy was consistent with the known safety profiles of the individual medicines, with no new safety concerns identified. The addition of Imfinzi did not compromise patients’ ability to complete BCG induction and maintenance therapy.

The second experimental arm evaluating Imfinzi plus BCG induction-only therapy compared to BCG induction and maintenance therapy alone did not meet the endpoint of DFS.

These data will be presented at a forthcoming medical meeting and shared with global regulatory authorities.

Imfinzi is approved in the US and other countries for patients with muscle-invasive bladder cancer (MIBC) based on results from the NIAGARA Phase III trial and continues to be investigated across early and late-stage bladder cancer in various treatment combinations, including in patients with MIBC who are ineligible or refuse to take cisplatin (VOLGA) and in locally advanced or metastatic disease (NILE).

Notes

Bladder cancer
Bladder cancer is the 9th most common cancer in the world, with more than 614,000 cases diagnosed each year.4 The most common type is urothelial carcinoma, which begins in the urothelial cells of the urinary tract.2

In 2024, an estimated 125,000 patients were treated for high-risk NMIBC, for which the current standard of care is transurethral resection of bladder tumour (TURBT) followed by administration of BCG directly into the bladder.5-6 Up to 80% of patients experience disease recurrence within five years, and rates of progression in high-risk patients can be as high as 45%.2 There is a critical need for treatment options in this curative-intent setting.

POTOMAC
POTOMAC is a randomised, open-label, multi-centre, global Phase III trial evaluating Imfinzi in combination with BCG therapy as a treatment for 1,018 patients with high-risk, BCG-naïve NMIBC who have undergone TURBT prior to randomisation. Patients were randomised 1:1:1 to receive Imfinzi plus BCG induction and maintenance therapy, or Imfinzi plus BCG induction-only therapy, versus standard-of-care BCG induction and maintenance therapy.

The trial was conducted in more than 120 centres across 12 countries including Canada and others across Europe and Asia. The primary endpoint was DFS, defined as time from randomisation to date of first recurrence of high-risk disease or death from any cause, for Imfinzi plus BCG induction and maintenance therapy compared to BCG induction and maintenance therapy alone. Secondary endpoints included DFS for Imfinzi plus BCG induction only therapy versus the comparator arm, as well as OS at five years and safety across both experimental arms of the trial.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

In addition to its indication in MIBC, Imfinzi is the global standard of care based on OS in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy (CRT). Additionally, Imfinzi is approved as a perioperative treatment in combination with neoadjuvant chemotherapy in resectable NSCLC, and in combination with a short course of Imjudo (tremelimumab) and chemotherapy for the treatment of metastatic NSCLC. Imfinzi is also approved for limited-stage small cell lung cancer (SCLC) in patients whose disease has not progressed following concurrent platinum-based CRT; and in combination with chemotherapy for the treatment of extensive-stage SCLC.

Imfinzi is also approved in combination with chemotherapy in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the European Union (EU).

In March 2025, perioperative Imfinzi added to standard-of-care chemotherapy met the primary endpoint of event-free survival in the MATTERHORN Phase III trial in resectable gastric and gastroesophageal junction cancers.

Imfinzi in combination with chemotherapy followed by Imfinzi monotherapy is approved as a 1st-line treatment for primary advanced or recurrent endometrial cancer (mismatch repair deficient disease only in US and EU). Imfinzi in combination with chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with mismatch repair proficient advanced or recurrent endometrial cancer in EU and Japan.

Since the first approval in May 2017, more than 374,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with NSCLC, bladder cancer, breast cancer, ovarian cancer and several gastrointestinal cancers.