On February 27, 2025 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2024 (Press release, Arcellx, FEB 27, 2025, View Source [SID1234650687]).

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"2024 was a transformational year for Arcellx as our ASH (Free ASH Whitepaper) data presentations for anito-cel, in partnership with Kite, continued to demonstrate anito-cel’s differentiated clinical profile for the potential treatment of patients with relapsed or refractory multiple myeloma," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "In our Phase 1 and iMMagine-1 studies, data demonstrate that anito-cel has the potential to provide a meaningful benefit to a broad population of patients. Our Phase 1 study demonstrated that the deep responses observed with anito-cel also translated to durable benefit for patients with a 30.2 month median progression-free survival in a challenging patient cohort and the preliminary data from our pivotal iMMagine-1 study demonstrated anito-cel also delivered deep responses for late line myeloma patients with a 97% overall response rate and 62% complete response rate at a median follow-up of 9.5 months. Most notably, as presented during ASH (Free ASH Whitepaper), the safety profile for anito-cel continues to be manageable with no delayed neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome in the155 patients dosed across the Phase 1 and iMMagine-1 studies. Physician engagement and enthusiasm for anito-cel continues to build following these data presentations and as more sites gain experience with anito-cel through iMMagine-3, our earlier-line study. Kite is manufacturing for iMMagine-3, and turnaround times are consistent with Kite commercial products. This year, we look forward to presenting updated data from our iMMagine-1 study mid-year, preparing for the commercial launch of anito-cel in multiple myeloma in 2026, enrolling our program in generalized myasthenia gravis, and expanding our ARC-SparX program in acute myeloid leukemia to include an additional SparX antigen target. Thank you to our Arcellx team who are willing to embrace challenges and think creatively to make an impact for the patients we serve."

Recent Business Progress

Presented positive preliminary data for the Phase 2 pivotal iMMagine-1 study of anito-cel in patients with relapsed or refractory multiple myeloma (RRMM) at the 66th ASH (Free ASH Whitepaper) Annual Meeting and Exposition. The data presented on December 9, 2024 demonstrated deep and durable responses with a predictable and manageable safety profile in a high-risk fourth-line or higher (4L+) RRMM population, including triple- and penta-class refractory disease. The data were from an October 31, 2024 data cutoff date, with a median follow-up of 9.5 months for the 86 patients who were evaluable for efficacy based on a follow-up of at least two months after treatment with anito-cel, and 98 patients were evaluable for safety based on a follow-up of at least one month after treatment with anito-cel.

Overall response rate was 97% (83/86) with a complete response/stringent complete response rate of 62% (53/86) and a very good partial response or higher rate of 81% (70/86), per International Myeloma Working Group (IMWG) criteria as investigator-assessed. Of those evaluable for minimal residual disease (MRD) testing, 93.1% (54/58) achieved MRD negativity at a minimum of 10 -5 sensitivity. Median progression-free survival (mPFS) and overall survival (OS) were not reached; 6-month PFS and OS rates were 93.3% and 96.5%, respectively, and 12-month PFS and OS rates were 78.5% and 96.5%, respectively. No delayed or non-ICANS neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, were observed in the 155 patients dosed with anito-cel.

Fourth Quarter and Full Year 2024 Financial Highlights

Cash, cash equivalents, and marketable securities:

As of December 31, 2024, Arcellx had cash, cash equivalents, and marketable securities of $625.7 million. Arcellx anticipates that its cash, cash equivalents, and marketable securities will fund its operations into 2027.

Collaboration revenue:

Collaboration revenue were $15.3 million and $63.1 million for the quarters ended December 31, 2024 and 2023, respectively, a decrease of $47.8 million. This decrease was primarily driven by the December 2023 expansion to the license and collaboration agreement with Kite Pharma, Inc. Collaboration revenue were $107.9 million and $110.3 million for the twelve months ended December 31, 2024 and 2023, respectively, a decrease of $2.4 million.

R&D expenses:

Research and development expenses were $44.6 million and $28.8 million for the quarters ended December 31, 2024 and 2023, respectively, an increase of $15.8 million. Research and development expenses were $157.1 million and $133.8 million for the twelve months ended December 31, 2024 and 2023, respectively, an increase of $23.3 million. The increases were primarily driven by increased personnel costs, which include non-cash stock-based compensation expense, and increased costs relating to anito-cel and other pipeline programs.

G&A expenses:

General and administrative expenses were $23.8 million and $19.4 million for the quarters ended December 31, 2024 and 2023, respectively, an increase of $4.4 million. General and administrative expenses were $88.4 million and $66.4 million for the twelve months ended December 31, 2024 and 2023, respectively, an increase of $22.0 million. The increases were primarily driven by increased personnel costs, which include non-cash stock-based compensation expense, increased costs relating to commercial readiness, and increased depreciation expense.

Net losses:

Net losses were $47.1 million and $19.8 million for the quarters ended December 31, 2024 and 2023, respectively. Net losses were $107.3 million and $70.7 million for the twelve months ended December 31, 2024 and 2023, respectively.

About Multiple Myeloma

Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. Multiple myeloma is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering.

About anitocabtagene autoleucel (anito-cel)

Anitocabtagene autoleucel (anito-cel, previously CART-ddBCMA) is the first BCMA-directed CAR T-cell therapy to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact binder known as the D-Domain. The small, stable D-Domain binder enables high CAR expression without tonic signaling and is designed to quickly release from the BCMA target. This combination may allow for the effective elimination of multiple myeloma cells without severe immunotoxicity. Anito-cel has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.

On February 27, 2025 IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage company in late-stage development with its DNA-mediated immunotherapy, reported financial results for the year ended December 31, 2024 and highlighted recent business updates including progress in advancing the IMNN-001 development program toward initiation of a Phase 3 clinical trial in advanced ovarian cancer (Press release, IMUNON, FEB 27, 2025, View Source [SID1234650706]).

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"2024 was a pivotal year for IMUNON. We reported robust and unprecedented data from our Phase 2 OVATION 2 Study, demonstrating that IMNN-001 is the first immunotherapy to consistently show clinical benefits in both progression-free and overall survival in ovarian cancer when combined with chemotherapy," said Stacy Lindborg, Ph.D., president and chief executive officer of IMUNON. "Treated patients achieved an overall survival of 13 months, compared to the current standard of care. Even more remarkable, the survival extension was greater among patients treated with IMNN-001 plus PARP inhibitors."

"We stand at the threshold of a historic advance in the frontline treatment of women with advanced ovarian cancer, a group with limited options and a desperate need for safe, effective treatments," Dr. Lindborg continued. "The most recent advances in ovarian cancer treatment have focused on maintenance treatment for those who have already responded to chemotherapy. However, our results in newly diagnosed patients with advanced disease are unprecedented and highly encouraging. The rapid clinical progress we have made reflects our compelling data and the strong support from trial investigators, patients, regulators, and global scientific leaders. We have engaged with the U.S. Food and Drug Administration through an End-of-Phase 2 meeting to finalize the design of our planned registrational study. As we look forward to an exciting year ahead, we are preparing to initiate a Phase 3 pivotal study of IMNN-001 in the first quarter of 2025."

RECENT DEVELOPMENTS

IMNN-001 Immunotherapy

Translational Data from OVATION 2 Study reinforce dose-dependent mechanism with IMNN-001 100mg/m2 dose and continue to validate TheraPlas technology, demonstrating DNA-mediated production of key anti-cancer immune cytokines following treatment – On February 19, 2025, IMUNON announced new translational data from ongoing analyses of results from the Company’s Phase 2 OVATION 2 Study of IMNN-001 for the treatment of newly diagnosed advanced ovarian cancer. Results demonstrated a 20% increase in IL-12 levels in women treated with IMNN-001 (100 mg/m2 administered intraperitoneally weekly) plus standard-of-care (SoC) neoadjuvant and adjuvant chemotherapy (NACT) compared to IL-12 levels in women treated with IMNN-001 (79 mg/m2). In this analysis increases in IL-12 levels were sampled in the peritoneal fluid cavity, which is the primary tumor microenvironment. Little to no changes were observed in the systemic blood stream of treated patients. In addition, the rise in IL-12 levels was accompanied by local increases in interferon-gamma (IFN-γ) and tumor necrosis factor-alpha (TNF-α), key downstream anti-cancer immune cytokines. Results showed no reports of serious immune-related adverse events including cytokine release syndrome.

Positive CMC Meeting with FDA for IMNN-001 – On December 19, 2024, IMUNON announced the positive outcome of a Type C Chemistry, Manufacturing, and Controls (CMC) meeting with the U.S. Food and Drug Administration (FDA) regarding production of IMNN-001 for the treatment of women with newly diagnosed advanced ovarian cancer. The goal of the meeting was to seek alignment and agreement with the FDA on key CMC topics to support IMNN-001 production for the planned Phase 3 pivotal trial and a potential future new biologics license application (BLA) submission. The meeting with the FDA included a review of IMUNON’s current good manufacturing practice (cGMP) clinical-scale and commercial manufacturing processes for IMNN-001, conducted at the Company’s manufacturing facility based in Huntsville, Alabama. The FDA agreed that IMUNON’s potency assay, which measures interferon-gamma (IFN-γ), is acceptable for the Phase 3 trial and for use in a commercial setting for release of drug product. The agency also agreed with the Company’s strategy to establish comparability of the core components of IMNN-001 produced by IMUNON with product previously produced through an external contract development and manufacturing organization.

Continued Improvement in Overall Survival Data from OVATION 2 Study of IMNN-001 – On December 10, 2024, the Company announced additional clinical data based on ongoing analyses of results from the Phase 2 OVATION 2 Study of IMNN-001 in the treatment of advanced ovarian cancer. The updated results, which were based on an additional seven months of patient monitoring, showed the hazard ratio (HR) decreased from 0.74 to 0.69, with an increase in median overall survival (OS) from 11.1 to 13 months following treatment with IMNN-001 plus standard-of-care (SoC) neoadjuvant and adjuvant chemotherapy (NACT) versus SoC alone. More than one-third of patients in the trial survived more than 36 months from the point of study enrollment, with 62% of those surviving patients from the IMNN-001 treatment arm and 38% from the SoC arm. More than 10% of trial participants reached 48 months or beyond at the time of this data assessment. Results also continued to demonstrate a favorable safety and tolerability profile, with no reports of cytokine release syndrome or any other serious immune-related adverse events. Initial results from the OVATION 2 Study were reported in July 2024 and results were presented in a late-breaking session at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting.

End-of-Phase 2 Meeting with the FDA for IMNN-001 Clinical Program – On November 25, 2024, IMUNON announced a positive outcome of its End-of-Phase 2 in-person meeting with the FDA, supporting the advancement of IMNN-001 for the treatment of advanced ovarian cancer into a Phase 3 pivotal study. IMUNON remains on track to initiate the Phase 3 trial in the first quarter of 2025. The interaction with the FDA included an extensive review of data generated to date, including the positive results from the Phase 2 OVATION 2 Study.

IMUNON Ovarian Cancer R&D Day – On September 18, 2024, the Company held an Ovarian Cancer R&D Day in New York City that included presentations from executive management and a panel of renowned leaders in oncology research and patient care including:

Sid Kerkar, M.D., T cell biology review editor, Frontiers in Immunology. Dr. Kerkar discussed the important role of interleukin-12 (IL-12) in treating cancer.

William Bradley, M.D., Professor, Obstetrics and Gynecology, Gynecologic Oncology, Medical College of Wisconsin. Dr. Bradley discussed the safety and efficacy of IMNN-001.

L.J. Wei, Ph.D., Professor of Biostatistics, Harvard T.H. Chan School of Public Health. Dr. Wei discussed the opportunity to combine progression-free survival (PFS) and overall survival (OS) to provide a clinically interpretable evaluation of the IMNN-001 treatment effect.

Amir Jazaeri, M.D., Vice Chair for Clinical Research, Director, Gynecologic Cancer Immunotherapy Program, Department of Gynecologic Oncology and Reproductive Medicine, University of Texas MD Anderson Cancer Center. Dr. Jazaeri discussed the ongoing Phase 1/2 study of IMNN-001 in combination with bevacizumab in advanced ovarian cancer, for which he serves as principal investigator, including the importance of minimal residual disease and early translational insights.

Premal Thaker, M.D., Interim Chief of Gynecologic Oncology, David & Lynn Mutch Distinguished Professor of Obstetrics & Gynecology, Director of Gynecologic Oncology Clinical Research, Washington University School of Medicine, and the OVATION 2 Study Chair. Dr. Thaker discussed the OVATION 2 top-line results and their clinical significance.

A webcast of the Ovarian Cancer R&D Day is available here.

PlaCCine: Next Generation Vaccine Proof of Concept

Data from PlaCCine DNA Vaccine Phase 1 Proof-of-Concept Clinical Study Demonstrate Persistent Immunogenicity in Trial Participants, Show an Acceptable Safety Profile and Further Validate PlaCCine Technology – On February 26, 2025, the Company announced safety and immunogenicity data from the Company’s first Phase 1 proof-of-concept clinical trial of IMNN-101, its investigational DNA plasmid vaccine based on the Company’s proprietary PlaCCine technology platform. The Phase 1 study was conducted in 24 healthy volunteers as a seasonal COVID-19 vaccine, targeting the SARS-CoV-2 Omicron XBB1.5 spike antigen. IMNN-101 was administered as a single dose vaccine without a booster dose in study participants who were previously vaccinated against the Omicron XBB1.5 variant. Results demonstrated that IMNN-101 is safe and well-tolerated with no serious adverse effects. IMNN-101 induced a persistent 2- to 4-fold increase in serum neutralizing antibody (NAb) titers from baseline through Week 4, further increasing NAb titers between Week 2 and Week 4. The immune response was observed against the XBB1.5 variant and many newer variants following treatment, demonstrating the IMNN-101 vaccine’s cross-reactivity. The participants in the Phase 1 trial had high baseline immune characteristics presumably from prior infection and multiple previous vaccinations against COVID-19 and ongoing infection as evidenced by the rise in viral nucleocapsid antigen during the study period. Modest increases in T cell responses were observed in this setting of trial participants having received multiple immunizations prior to the study.

The Phase 1 clinical data of IMNN-101 is consistent with strong evidence of immunogenicity and protection for the PlaCCine platform in rodents and non-human primates, with prior preclinical results showing that protection exceeded 95% in non-human primates, which is comparable to mRNA vaccines. The robust immunogenicity profile, expected durability of protection, comparative ease of manufacturing, and stability at workable temperatures (up to one year at 4°C and one month at 37°C) suggest that a vaccine based on the PlaCCine technology platform may be a potential viable alternative to available messenger RNA (mRNA) vaccines. The Company plans to seek potential partners for further development.

Corporate Development

Addition to Leadership Team to Support Future Clinical Programs – On February 10, 2025, Douglas V. Faller, M.D., Ph.D. was appointed Chief Medical Officer effective February 18, 2025. Dr. Faller joins IMUNON with more than 30 years of industry, academic and laboratory experience, with specialized expertise in oncology and immunology. Dr. Faller will lead the Company’s clinical strategy including advancing the IMNN-001 program for the treatment of newly diagnosed advanced ovarian cancer.

Dr. Faller joins IMUNON with more than 30 years of experience at biotechnology and pharmaceutical companies leading strategies across discovery, preclinical, clinical and regulatory stages of small molecule development in several therapeutic areas including oncology, immunology and hematology. He also brings more than 25 years of experience in academic clinical and laboratory research settings with a focus on drug discovery and development, oncology and hematology, and cell and molecular biology. Dr. Faller most recently served as chief medical officer at Skyhawk Therapeutics, where he was responsible for global clinical and regulatory development of novel small molecule RNA-splicing modifiers for the treatment of hematological and solid tumors and rare neurological diseases. Before that, he served as chief medical officer at Oryzon Genomics, Inc. Previously, he worked at Takeda for more than five years in roles of increasing responsibility, most recently serving as executive medical director where he led the development of multiple late-stage therapies including a CAR-T program for leukemias and lymphomas and solid tumor programs including in gynecologic oncology.

Dr. Faller received an M.D. from Harvard Medical School and a Ph.D. and B.S. from the Massachusetts Institute of Technology. He was professor of medicine at Harvard Medical School, and subsequently he founded and served as first director of Boston University Comprehensive Cancer Center where he was also Grunebaum Professor for Cancer Research and professor of medicine, biochemistry, pediatrics, microbiology, pathology and laboratory medicine. Dr. Faller is the scientific founder of multiple biotechnology and pharmaceutical companies.

Financial Results for the Year Ended December 31, 2024

IMUNON reported a net loss for 2024 of $18.6 million, or $1.62 per share compared with a net loss for 2023 of $19.5 million, or $2.16 per share. Operating expenses were $19.1 million for 2024, a decrease of $1.9 million or 9% from $21.0 million for 2023. The Company recognized tax benefits from the sale of its New Jersey net operating losses of $1.3 million in 2023.

Research and development (R&D) expenses were $11.6 million for 2024, a decrease of $0.3 million from $11.3 million for 2023. Costs associated with the OVATION 2 Study were $1.4 million and $1.2 million for 2024 and 2023, respectively. Costs associated with our PlaCCine vaccine initiative were $1.4 million in 2024. Other clinical and regulatory costs, which include start up costs for OVATION 3, were $2.4 million for 2024 compared with $1.8 million for 2023. R&D costs associated with the development of IMNN-001 to support the OVATION 2 Study were $1.8 million for 2024 compared with $1.5 million for 2023. The development of the PlaCCine DNA vaccine technology platform decreased to $2.6 million in 2024 compared to $4.5 million in 2023. CMC costs were $2.0 million for 2024 compared with $2.3 million for 2023 due to the development of in-house cGMP manufacturing capabilities for DNA plasmids and nanoparticle delivery systems and product development costs for OVATION 3.

General and administrative expenses were $7.5 million for 2024 compared with $9.7 million for 2023. This 23% decrease was primarily attributable to lower legal costs ($1.4 million), lower employee-related costs ($0.4 million), lower non-cash stock compensation expense ($0.3 million), lower public company franchise expenses ($0.2 million) and lower insurance costs ($0.1 million), offset by higher consulting fees ($0.2 million).

Other non-operating income was $0.5 million for 2024 compared with $0.2 million for 2023. This increase was primarily attributable to the following:

Investment income from the Company’s short-term investments was $0.5 million for 2024 compared with $1.2 million for 2023.

In June 2021, the Company entered into a $10.0 million loan facility with Silicon Valley Bank (SVB). IMUNON immediately used $6.0 million from this facility to retire all outstanding indebtedness with Horizon Technology Finance Corporation. In connection with the loan facility, the Company incurred $0.2 million in interest expense in the first half of 2023. In the second quarter of 2023, the Company terminated the SVB Loan Facility, paid early termination and end-of-term charges and recognized $0.3 million as a loss on debt extinguishment.

Net cash used for operating activities was $18.9 million for 2024 compared with $19.0 million for 2023. Cash provided by financing activities of $9.1 million for 2024 resulted from an at-the-market equity offering in July 2024 and sales under the Company’s At-the-Market Equity Facility compared with cash used in financing activities of $3.6 million for 2023 resulting from the pay-off of the SVB loan ($6.4 million), offset by sales under the Company’s At-the-Market Equity Facility ($2.8 million).

The Company ended 2024 with $5.9 million in cash and cash equivalents. The Company believes it has sufficient capital resources to fund its operations into late second quarter of 2025.

Conference Call and Webcast

The Company is hosting a conference call to review 2024 financial results and provide business updates today at 11:00 a.m. ET. To participate in the call, please dial 833-816-1132 (Toll-Free/North America) or 412-317-0711 (International/Toll) and ask for the IMUNON 2024 Earnings Call. A live webcast of the call will also be available here.

The call will be archived for replay until March 12, 2025. The replay can be accessed at 877-344-7529 (U.S. Toll-Free), 855-669-9658 (Canada Toll-Free) or 412-317-0088 (International Toll), using the replay access code 7147564. An audio replay of the call will also be available here for 90 days.

On February 27, 2025 Merus N.V. (Nasdaq: MRUS) (Merus, the Company, we, or our), an oncology company developing innovative, full-length multispecific antibodies and antibody drug conjugates (Biclonics, Triclonics and ADClonics), reported financial results for the fourth quarter and full year and provided a business update (Press release, Merus, FEB 27, 2025, View Source [SID1234650727]).

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"We believe that petosemtamab’s receipt of two Breakthrough Therapy designations by the FDA – previously as monotherapy in the 2L+ treatment of r/m HNSCC and very recently, based on updated clinical efficacy, durability and safety of petosemtamab in combination with pembrolizumab in 1L PD-L1+ r/m HNSCC, indicates the potential for these treatment regimens to demonstrate substantial improvement over available therapies," said Bill Lundberg, M.D., President, Chief Executive Officer of Merus. "We look forward to sharing the updated clinical data, including durability, for petosemtamab with pembrolizumab in 1L PD-L1+ r/m HNSCC, for the full phase 2 cohort, in the first half of 2025."

Petosemtamab (MCLA-158: EGFR x LGR5 Biclonics): Solid Tumors
LiGeR-HN1 phase 3 trial in 1L PD-L1+ r/m head and neck squamous cell carcinoma (HNSCC) and LiGeR-HN2 phase 3 trial in 2/3L r/m HNSCC enrolling – we expect both trials to be substantially enrolled by YE25; clinical update on phase 2 trial in combination with pembrolizumab in 1L PD-L1+ r/m HNSCC planned for 1H25; phase 2 trial in 1L, 2L and 3L+ metastatic colorectal cancer (mCRC) enrolling; mCRC initial clinical data planned for 2H25

In February 2025, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation (BTD) for petosemtamab in combination with pembrolizumab for the first-line treatment of adult patients with r/m PD-L1+ HNSCC with CPS ≥ 1. This designation was detailed in our press release, Petosemtamab granted Breakthrough Therapy designation by the U.S. FDA for 1L PD-L1 positive head and neck squamous cell carcinoma (February 18, 2025).

In September 2024, Merus announced the first patient was dosed in LiGeR-HN1, a phase 3 trial evaluating the efficacy and safety of petosemtamab in combination with pembrolizumab in 1L PD-L1+ r/m HNSCC compared to pembrolizumab. In this trial, patients will be randomized to petosemtamab plus pembrolizumab or pembrolizumab monotherapy. This was detailed in our press release, Merus Announces First Patient Dosed in LiGeR-HN1, a Phase 3 Trial Evaluating Petosemtamab in Combination with Pembrolizumab in 1L r/m HNSCC (September 30, 2024).

Merus provided an interim clinical update on petosemtamab with pembrolizumab in 1L PD-L1+ r/m HNSCC at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting 2024, demonstrating a 67% response rate among 24 evaluable patients. The oral presentation was detailed in our press release, Merus’ Petosemtamab in Combination with Pembrolizumab Interim Data Demonstrates Robust Response Rate and Favorable Safety Profile in 1L r/m HNSCC (May 28, 2024). A clinical update on this cohort is planned for 1H25.

In July 2024, Merus announced the first patient was dosed in LiGeR-HN2, a phase 3 trial evaluating the efficacy and safety of petosemtamab in 2/3L HNSCC compared to standard of care. In this trial, patients will be randomized to petosemtamab monotherapy or investigator’s choice of single agent chemotherapy or cetuximab. This was detailed in our press release, Merus Announces First Patient Dosed in LiGeR-HN2, a Phase 3 Trial Evaluating Petosemtamab in 2/3L r/m HNSCC – Merus (July 24, 2024).

Merus provided updated interim clinical data on petosemtamab in 2L+ r/m HNSCC at the European Society for Medical Oncology Asia Congress, demonstrating a 36% response rate among 75 evaluable patients. The oral presentation was detailed in our press release, Merus’ Petosemtamab Monotherapy Interim Data Continues to Demonstrate Clinically Meaningful Activity in 2L+ r/m HNSCC (Dec. 7, 2024).

In May 2024, the FDA granted BTD for petosemtamab for the treatment of patients with recurrent or metastatic HNSCC whose disease has progressed following treatment with platinum based chemotherapy and an anti-programmed cell death receptor-1 (PD-1) or anti-programmed death ligand 1 (PD-L1) antibody. This designation was detailed in our press release, Petosemtamab granted Breakthrough Therapy Designation by the U.S. FDA (May 13, 2024).

Merus believes a randomized registration trial in HNSCC with an overall response rate endpoint could potentially support accelerated approval and the overall survival results from the same study could potentially verify its clinical benefit to support regular approval.

In the third quarter 2024, Merus announced the first patient was dosed in a phase 2 trial evaluating petosemtamab in combination with standard chemotherapy in 2L mCRC. This was detailed in our press release, Merus Announces First Patient Dosed in Phase 2 Trial of Petosemtamab in 2L CRC (July 8, 2024). In the fourth quarter 2024, Merus announced the first patient was dosed in a phase 2 trial evaluating petosemtamab monotherapy in heavily pretreated (3L+) mCRC. This was detailed in our press release, Merus announces First Patient Dosed in Phase 2 Trial of Petosemtamab in 3L+ mCRC (Dec. 16, 2024). In January 2025, the first patient was dosed in a phase 2 trial evaluating petosemtamab in combination with standard chemotherapy in 1L mCRC. We expect to provide initial clinical data for petosemtamab in mCRC in 2H25.

BIZENGRI (zenocutuzumab-zbco: HER2 x HER3 Biclonics)
Approved by FDA for adults with pancreatic adenocarcinoma or non–small cell lung cancer (NSCLC) that are advanced unresectable or metastatic and harbor a neuregulin 1 (NRG1) gene fusion who have disease progression on or after prior systemic therapy

In December 2024, the FDA approved BIZENGRI (zenocutuzumab-zbco), the first and only treatment indicated for adults with pancreatic adenocarcinoma or NSCLC that are advanced unresectable or metastatic and harbor a NRG1 gene fusion who have disease progression on or after prior systemic therapy. These indications are approved under accelerated approval based on overall response rate (ORR) and duration of response (DOR). Continued approval for these indications may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). BIZENGRI has a Boxed WARNING for Embryo-Fetal Toxicity and warnings for infusion-related reactions (IRRs), hypersensitivity and anaphylactic reactions, interstitial lung disease (ILD)/pneumonitis, and left ventriculardysfunction.1 See Important Safety Information below. This was detailed in our press release, Merus Announces FDA Approval of BIZENGRI (zenocutuzumab-zbco) for NRG1+ Pancreatic Adenocarcinoma and NRG1+ Non–Small Cell Lung Cancer (NSCLC) Based on Safety and Efficacy Data From the eNRGy Study (December 4, 2024).

Merus has exclusively licensed to Partner Therapeutics the right to commercialize BIZENGRI for the treatment of NRG1+ cancer in the U.S. This was detailed in our press release, Merus and Partner Therapeutics Announce License Agreement for the U.S. Commercialization of Zenocutuzumab in NRG1 Fusion-Positive Cancer (December 2, 2024).

MCLA-129 (EGFR x c-MET Biclonics): Solid Tumors

Investigation of MCLA-129 is ongoing in METex14 NSCLC; phase 2 trial in combination with chemotherapy in 2L+ EGFR mutant (EGFRm) NSCLC enrolling

In the third quarter 2024, Merus announced the first patients were dosed in the phase 2 trial evaluating MCLA-129 in combination with chemotherapy in 2L+ EGFRm NSCLC, with a cohort receiving MCLA-129 and paclitaxel and carboplatin, and another cohort receiving MCLA-129 and docetaxel. We remain interested in partnering MCLA-129 to sufficiently resource the development of MCLA-129 and the potential benefit it may have for patients.

MCLA-129 is subject to a collaboration and license agreement with Betta Pharmaceuticals Co. Ltd. (Betta), which permits Betta to develop MCLA-129, and potentially commercialize exclusively in China, while Merus retains global rights outside of China.

Collaborations

Incyte Corporation
Since 2017, Merus has been working with Incyte Corporation (Incyte) under a global collaboration and license agreement focused on the research, discovery and development of bispecific antibodies utilizing Merus’ proprietary Biclonics technology platform. For each program under the collaboration, Merus receives reimbursement for research activities and is eligible to receive potential development, regulatory and commercial milestones and sales royalties for any products, if approved.

Eli Lilly and Company
In January 2021, Merus and Eli Lilly and Company (Lilly) announced a research collaboration and exclusive license agreement to develop up to three CD3-engaging T-cell re-directing bispecific antibody therapies utilizing Merus’ Biclonics platform and proprietary CD3 panel along with the scientific and rational drug design expertise of Lilly. The collaboration is progressing well with three programs advancing through preclinical development.

Gilead Sciences
In March 2024, Merus and Gilead Sciences announced a collaboration to discover novel antibody based trispecific T-cell engagers using Merus’ patented Triclonics platform. Under the terms of the agreement, Merus will lead early-stage research activities for two programs, with an option to pursue a third. Gilead will have the right to exclusively license programs developed under the collaboration after the completion of select research activities. If Gilead exercises its option to license any such program from the collaboration, Gilead will be responsible for additional research, development and commercialization activities for such program. Merus received an equity investment by Gilead of $25 million in Merus common shares and an upfront payment of $56 million.

Ono Pharmaceutical
In 2018, the Company granted Ono Pharmaceutical Co., Ltd. (Ono) an exclusive, worldwide, royalty-bearing license, with the right to sublicense, research, test, make, use and market a limited number of bispecific antibody candidates based on Merus’ Biclonics technology platform directed to an undisclosed target combination. During the third quarter of 2024, Merus achieved and received a milestone payment based on the filing of an Investigational New Drug (IND) application in Japan.

Biohaven
In January 2025, Merus and Biohaven announced a research collaboration and license agreement to co-develop three novel bispecific antibody drug conjugates (ADCs), leveraging Merus’ leading Biclonics technology platform, and Biohaven’s next-generation ADC conjugation and payload platform technologies. Under the terms of the agreement, Biohaven is responsible for the preclinical ADC generation of three Merus bispecific antibodies under mutually agreed research plans. The agreement includes two Merus bispecific programs generated using the Biclonics platform, and one program under preclinical research by Merus. Each program is subject to mutual agreement for advancement to further development, with the parties then sharing subsequent external development costs and commercialization, if advanced.

Cash Runway, existing cash, cash equivalents and marketable securities expected to fund Merus’ operations into 2028

As of December 31, 2024, Merus had $724.0 million cash, cash equivalents and marketable securities. Based on the Company’s current operating plan, the existing cash, cash equivalents and marketable securities are expected to fund Merus’ operations into 2028.

Full Year 2024 Financial Results

Collaboration revenue for the year ended December 31, 2024 decreased $7.8 million as compared to the year ended December 31, 2023, primarily as a result of decreases in Lilly revenue of $8.4 million and Incyte revenue of $6.4 million, offset by increases in Gilead revenue of $4.8 million, and Other revenue of $2.2 million. The decrease in Lilly revenue is primarily the result of decreases in upfront payment amortization of $4.8 million and reimbursement revenue of $3.6 million. The decrease in Incyte revenue is primarily the result of decreases in milestone revenue of $5.0 million and reimbursement revenue of $1.4 million. Gilead revenue increased due to the start of the collaboration agreement in 2024 which resulted in an increase in upfront payment amortization of $4.8 million. The increase in Other revenue is primarily the result of increases in milestone revenue of $2.1 million.

Research and development expense for the year ended December 31, 2024 increased $84.7 million as compared to the year ended December 31, 2023, primarily as a result of increases in external clinical services and drug manufacturing costs of $66.6 million, which primarily includes costs to advance our petosemtamab program and costs to fulfill our obligations under our collaboration agreements related to our programs, increases in personnel related expenses including share-based compensation of $11.8 million due to an increase in employee headcount and an increase in share price, consultancy expenses of $5.5 million, facilities expenses and other related expenses of $0.7 million, and consumables expenses of $0.2 million, offset by decreases in depreciation and amortization of $0.1 million.

General and administrative expense for the year ended December 31, 2024 increased $23.0 million as compared to the year ended December 31, 2023, primarily as a result of increases in personnel related expenses including share-based compensation of $12.6 million due to an increase in employee headcount and an increase in share price, consultancy expenses of $6.8 million, legal expenses of $1.8 million, facilities and depreciation expense of $1.2 million, and intellectual property and licenses expenses of $0.7 million. Other income, net consists of interest earned on our cash and cash equivalents held on account, accretion of investment earnings and net foreign exchange gains or losses on our foreign denominated cash, cash equivalents and marketable securities, and payables and receivables.

MERUS N.V.
CONSOLIDATED BALANCE SHEETS
(Amounts in thousands, except share and per share data)

2024 2023
ASSETS
Current assets:
Cash and cash equivalents $ 293,294 $ 204,246
Marketable securities 243,733 150,130
Accounts receivable 1,261 2,429
Prepaid expenses and other current assets 30,784 12,009
Total current assets 569,072 368,814
Marketable securities 187,008 57,312
Property and equipment, net 10,770 12,135
Operating lease right-of-use assets 9,254 11,362
Intangible assets, net 1,679 1,800
Deferred tax assets 1,520 1,199
Other assets 3,390 2,872
Total assets $ 782,693 $ 455,494
LIABILITIES AND SHAREHOLDERS’ EQUITY
Current liabilities:
Accounts payable $ 4,164 $ 4,602
Accrued expenses and other liabilities 43,957 38,482
Income taxes payable 7,317 1,646
Current portion of lease obligation 1,704 1,674
Current portion of deferred revenue 29,934 22,685
Total current liabilities 87,076 69,089
Lease obligation 8,208 10,488
Deferred revenue, net of current portion 39,482 19,574
Total liabilities 134,766 99,151
Commitments and contingencies (Note 10)
Shareholders’ equity:
Common shares, €0.09 par value; 105,000,000 and 67,500,000 shares authorized at December 31, 2024 and 2023, respectively; 68,828,749 and 57,825,879 shares issued and outstanding at December 31, 2024 and 2023, respectively 6,957 5,883
Additional paid-in capital 1,664,822 1,126,054
Accumulated deficit (968,387 ) (753,061 )
Accumulated other comprehensive (loss) income (55,465 ) (22,533 )
Total shareholders’ equity 647,927 356,343
Total liabilities and shareholders’ equity $ 782,693 $ 455,494

MERUS N.V.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(Amounts in thousands, except share and except per share data)

Year Ended December 31,
2024 2023 2022
Collaboration revenue $ 36,133 43,947 41,586
Total revenue 36,133 43,947 41,586
Operating expenses:
Research and development 225,368 140,658 149,424
General and administrative 82,832 59,836 52,200
Total operating expenses 308,200 200,494 201,624

Operating loss (272,067 ) (156,547 ) (160,038 )
Other income (loss), net:
Interest (expense) income, net 30,789 14,510 2,722
Foreign exchange (losses) gains, net 34,103 (9,710 ) 26,022
Other (losses) gains, net — — 1,059
Total other income (loss), net 64,892 4,800 29,803

Loss before income tax expense (207,175 ) (151,747 ) (130,235 )
Income tax expense 8,151 3,192 959
Net loss $ (215,326 ) $ (154,939 ) $ (131,194 )
Other comprehensive income (loss):
Currency translation adjustment (32,932 ) 7,915 (21,227 )
Comprehensive loss $ (248,258 ) $ (147,024 ) $ (152,421 )
Net loss per share allocable to common shareholders:
Basic and diluted $ (3.35 ) $ (3.00 ) $ (2.92 )
Weighted-average common shares outstanding:
Basic and diluted 64,220,765 51,605,444 44,919,084

Please see full Prescribing Information, including Boxed WARNING, at BIZENGRI.com/pi.

Reference: 1. BIZENGRI. Prescribing information. Merus N.V.; 2024.

On February 27, 2025 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) reported financial results for the three and twelve months ended December 31, 2024 and provided an update on recent corporate progress (Press release, Aurinia Pharmaceuticals, FEB 27, 2025, View Source [SID1234650688]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Fourth Quarter 2024 Financial Results

Total Revenue: For the three months ended December 31, 2024, total revenue was $59.9 million, up 33% from $45.1 million in the same period of 2023.
– Net Product Sales: For the three months ended December 31, 2024, net product sales of LUPKYNIS, the first FDA-approved oral therapy for the treatment of adult patients with active lupus nephritis, were $57.6 million, up 36% from $42.3 million in the same period of 2023.

– License, Collaboration and Royalty Revenue: For the three months ended December 31, 2024, license, collaboration and royalty revenue, which includes manufacturing services revenue and royalties from Aurinia’s collaboration partner, Otsuka, was $2.3 million, down 18% from $2.8 million in the same period of 2023.

Net Income (Loss): For the three months ended December 31, 2024, net income (loss) was $1.4 million, compared to $(26.9) million in the same period of 2023.
Cash Flow Provided by Operating Activities: For the three months ended December 31, 2024, cash flow provided by operating activities was $30.1 million, up 110% from $14.3 million in the same period of 2023.
Full Year 2024 Financial Results

Total Revenue: For the twelve months ended December 31, 2024, total revenue was $235.1 million, up 34% from $175.5 million in 2023.
– Net Product Sales: For the twelve months ended December 31, 2024, net product sales were $216.2 million, up 36% from $158.5 million in 2023.

– License, Collaboration and Royalty Revenue: For the twelve months ended December 31, 2024, license, collaboration and royalty revenue, which includes a milestone payment, manufacturing services revenue and royalties from Otsuka, was $18.9 million, up 11% from $17.0 million in 2023.

Net Income (Loss): For the twelve months ended December 31, 2024, net income (loss) was $5.8 million, compared to $(78.0) million in 2023.
Cash Flow Provided by (Used in) Operating Activities: For the twelve months ended December 31, 2024, cash flow provided by (used in) operating activities was $44.4 million, compared to $(33.5) million in 2023.
Cash Position

As of December 31, 2024, Aurinia had cash, cash equivalents, restricted cash and investments of $358.5 million, compared to $350.7 million at December 31, 2023. For the year ended December 31, 2024, the Company repurchased 6.1 million of its common shares for $41.0 million.

Full Year 2025 Total Revenue and Net Product Sales Guidance

For 2025, Aurinia expects total revenue in the range of $250 million to $260 million and net product sales in the range of $240 million to $250 million.

Research and Development Update

In September 2024, Aurinia initiated a Phase 1 study of AUR200, its potentially best-in-class dual inhibitor of B cell activating factor (BAFF) and a proliferation inducing ligand (APRIL). BAFF and APRIL are cytokines that stimulate B cell proliferation and activity and are upregulated in many autoimmune diseases. In preclinical studies, AUR200 potently inhibited B cell proliferation and production of IgA and IgM antibodies and exhibited pharmacokinetic and pharmacodynamic properties consistent with once-monthly dosing. Aurinia expects to report initial results from its Phase 1 study of AUR200 in the second quarter of 2025.

"We are pleased to have delivered strong LUPKYNIS sales growth in 2024," stated Peter Greenleaf, President and Chief Executive Officer of Aurinia. "We expect 2025 to be an exciting year for Aurinia. We remain focused on increasing LUPKYNIS’s adoption among the many lupus nephritis patients who could benefit from this important treatment, while, at the same time, advancing our important pipeline product, AUR200, which has the potential to treat a wide range of autoimmune diseases."

Webcast & Conference Call Details

A webcast and conference call will be hosted today, February 27th, at 8:30 a.m. ET. The link to the audio webcast is available here. To join the conference call, please dial 877-407-9170/+1 201-493-6756. A replay of the webcast will be available on Aurinia’s website.

On February 27, 2025 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported that members of its executive team will present and host 1×1 meetings at the Leerink Partners 2025 Global Healthcare Conference being held on March 10 – 12, 2025 in W Hotel South Beach in Miami, Florida (Press release, Innate Pharma, FEB 27, 2025, View Source [SID1234650707]).

The executive team will participate in a fireside chat scheduled Tuesday, March 11, 2025, from 3:00 – 3:30 pm ET.
A live webcast and a replay of the presentation will be available on the Events page in the Investors section of Innate Pharma website.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!