On March 27, 2025 INmune Bio Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage inflammation and immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, reported its financial results for the year ended December 31, 2024 and provides a business update (Press release, INmune Bio, MAR 27, 2025, View Source [SID1234651524]).

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Full Year 2024 and Recent Corporate Highlights

DN-TNF Platform Highlights (XPro1595, XPro):

● Announced completion of enrollment for its Phase 2 trial on Friday, 27 September, 2024. This global, blinded, randomized Phase 2 trial (the "AD02 trial") is focused on patients with Early Alzheimer’s Disease (AD) and biomarkers of elevated neuroinflammation. Final enrollment of 208 patients exceeded the trial’s target enrollment of 201 patients.

● Completed two separate interim analyses of blinded data from its AD02 trial. The analyses demonstrated exceptional performance of the novel cognitive measure EMACC, as well as highly significant correlation between baseline EMACC and Clinical Dementia Rating-Sum of Boxes (CDR-SB). Key findings of the analysis included:

o Statistical Correlation: An independent review confirmed a significant correlation (p<0.001) between baseline scores on EMACC and CDR-SB, the key cognitive endpoints in the AD02 trial.

o Reliability: The correlation of EMACC when measured during the screening process and again at the first study visit before treatment was found to be 0.93. Higher precision produces results that are more robust and replicable with smaller sample sizes.

o Differentiation Capability: The difference in EMACC performance between patients with CDR global ratings of 0.5 (prodromal AD) and those rated 1.0 (mild dementia) was very large, with an effect size (Cohen’s d) of 0.87 (p<.0001). This demonstrates EMACC’s ability to accurately differentiate between disease stages, highlighting its sensitivity and precision.

● Hosted a webinar titled "Why EMACC is the Optimal Tool for Measuring Cognitive Change in Early Alzheimer’s Trials," that explained the development of EMACC and its advantages in assessing cognitive changes over time in early AD patients while also covering the regulatory landscape for Alzheimer’s disease drug development and the role of the CDR-SB clinical scale. The replay can be found here or with this link: View Source

● Announced publication in Cell Reports, "Microglia Regulate Cortical Remyelination via ΤNFR1-Dependent Phenotypic Polarization." Myelin is necessary for fast and efficient communication between neurons. Loss of myelin compromises neuron function and communication and is a key step in the neurodegenerative process of many CNS diseases, including Alzheimer’s Disease. Data from the publication identifies soluble TNF as a critical cytokine checkpoint that converts microglia from a reparative, remyelinating cell to a damaging, demyelinating cell. These data suggest that blocking soluble TNF is a promising strategy for treating demyelinating diseases.

● Announced new phase 1 study data presented at the annual Alzheimer’s Association International Conference on 29 July 2024 demonstrating dose-dependent effect of XPro on Proteins that regulate synapses in Alzheimer’s patients. The new analysis revealed that a 12-week treatment with XPro resulted in a significant change in synaptic proteins, which are essential for communication between neurons.

● Announced statistically significant improvements in electroencephalography (EEG), a biomarker of brain function, in patients with moderate to severe Alzheimer’s Disease treated with XPro for four weeks.

● Demonstrated 24-month stability validation of XPro for phase III readiness and commercial supply chain modeling & announced development of novel immunogenicity assay.

CORDStrom Platform

● Reported results of a double-blinded, randomized, placebo-controlled, cross-over study, known as "MissionEB," investigating CORDStrom for treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) in pediatric patients, which evidenced a favorable benefit-risk profile.

● FDA granted CORDStrom a Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) for treatment of epidermolysis bullosa (EB).

● Data from the MissionEB trial show that CORDStrom was easily delivered, extremely well tolerated, with no serious adverse events related to CORDStrom reported at either 3-months or 6-months post-treatment across all age and RDEB-severity patient sub-types. In children with severe disease, CORDStrom reduced itch at 3-months and led to a sustained reduction of over 27% at 6-months in some patients. These results demonstrate that a clinically meaningful reduction in itch severity is sustained over time. In children with intermediate disease severity, CORDStrom provided a broader range of improvements, including reduced skin involvement and less pain, as well as a large reduction in itch. In younger children with RDEB (age <10yrs), CORDStrom provided improvements in skin scores, indicating better skin integrity and reduced disease activity. Interviews with subjects and caregivers strongly support the clinical benefits of CORDStrom as both caregivers and patients were able to correctly identify which treatment had been CORDStrom and which had been placebo in this cross-over study.

● The Company plans to initiate a 12-month open label study at GOSH, including all patients enrolled in the MissionEB study, where patients will receive 3 cycles of CORDStrom therapy at 0, 4 and 8 months. Each cycle of CORDStrom is a single infusion of CORDStrom 14 days apart.

INKmune Platform:

● Announced that INKmune demonstrates excellent safety and increased NK-Cell activity in the first dosing cohort, in its Phase I/II trial (the "CaRe PC" trial) for men with metastatic Castration-Resistant Prostate Cancer (mCRPC). Blinded analysis of the monitoring blood samples from the first three patients showed changes in the phenotype and function of the patient’s NK cells. Although this is the lowest dose cohort, 2 of 3 patients showed an increase in circulating activated NK cells and all three showed increased NK cell function sustained for more than 40 days after the final INKmune infusion.

● The CaRe PC trial completed dosing of all patients in the phase I part of the trial and commenced dosing phase II patients at the intermediate and high dose cohorts. Interim analysis of the first three patients treated at the lowest dose was presented at the Innate Killer Summit conference in San Diego earlier this month and reported changes in all biomarkers consistent with NK cell activation in vivo post INKmune treatment which mirrored that seen in patients with AML/MDS treated previously.

● Published landmark paper in Journal Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) senior-authored by Mark Lowdell, PhD, Chief Scientific Officer, titled, "Proteomic and phenotypic characteristics of memory-like Natural Killer cells for cancer immunotherapy." The study demonstrates that memory-like natural killer (mlNK) cells, generated by either cytokine or INKmune priming, show increased cytotoxicity against multiple tumor types, offering promising potential for cancer immunotherapy. Importantly, while most studies are conducted on NK cells from healthy volunteers, this study demonstrated that mlNK from cancer patients are equally as potent as those generated from healthy volunteers further supporting INKmune’s in vivo treatment methodology. The research also provides new insights into the metabolic and physiological mechanisms underlying NK cell memory, paving the way for innovative treatments in both hematological malignancies and solid tumors.

● Announced new format of INKmune that supports highest trial dose with single bag administration and expansion of bioreactor capacity in preparation of scalable manufacturing. An IND amendment with the improved formulation has been submitted to the FDA that also includes additional validation data supporting an alternative critical reagent used in INKmune manufacturing, improving supply chain redundancy.

● Safety of INKmune remains excellent. There have been more than 30 administrations of INKmune in the mCRPC study given on an out-patient basis, with no significant drug related adverse events or episodes of cytokine release syndrome (CRS). Combining the experience with INKmune from the MDS/AML and mCRPC trials, over 40 infusions of INKmune have been given safely without the need for conditioning therapy, pre-medication, or cytokine support.

Corporate:

● Completed repayment of outstanding Silicon Valley Bank term loan in December.

● Executed securities purchase agreements with new and existing institutional investors and certain directors, officers and employees of the Company for total gross proceeds of approximately $27.5 million. As part of the offerings, the company issued approximately 3.9 million warrants to purchase common stock. The term of the warrants may be accelerated with positive AD02 data as defined in the warrant agreements, which if exercised for cash will raise approximately $30 million dollars.

● Sold shares of common stock through the ATM program during 2024 for total gross proceeds of approximately $2.4 million. Between January 1, 2025, and March 27, 2025, sold shares of common stock through the ATM program for total gross proceeds of approximately $5.4 million.

● Joined the broad-market Russell 3000 Index at the conclusion of the 2024 Russell US Indexes annual reconstitution, effective as of July 1, 2024.

● Received a research and development rebate from Australia in July of approximately $2.5 million USD.

Upcoming Events and Milestones:

● Top line cognitive results and secondary endpoints from the AD02 trial in Alzheimer’s Disease will be available in June 2025.

● Data from the ongoing INKmune trial in mCRPC will be released as they become available. The next data set should be released in Q2 or Q3, 2025.

● A Phase II trial of XPro in patients with Treatment-Resistant Depression will begin enrollment soon once the NIH releases funds for the trial.

● Anticipate filing a BLA in RDEB in 2025 or early 2026 followed by MAA application in the UK and EU. If approved on or prior to September 2026, CORDstrom could be eligible for a Priority Review Voucher.

Financial Results for the Year Ended December 31, 2024:

● Net loss attributable to common stockholders for the year ended December 31, 2024 was approximately $42.1 million, compared to approximately $30.0 million during the year ended December 31, 2023.

● Research and development expenses totaled approximately $33.2 million for the year ended December 31, 2024, compared to approximately $20.3 million during the year ended December 31, 2023.

● General and administrative expenses were approximately $9.5 million for the year ended December 31, 2024, compared to approximately $9.6 million during the year ended December 31, 2023.

● Other income, net, was approximately $0.6 million for the year ended December 31, 2024, compared to other expense, net, of approximately $0.3 million during the year ended December 31, 2023.

● As of December 31, 2024, the Company had cash and cash equivalents of approximately $20.9 million.

● As of March 27, 2025, the Company had approximately 22.9 million common shares outstanding.

Earnings Call Information

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call. Please ask for the INmune Bio 2024 Year End Conference Call when reaching an operator.

Date: March 27, 2025
Time: 4:30 PM Eastern Time
Participant Dial-in: 1-800-225-9448 or 1-203-518-9708 (international): 1-203-518-9808
Conference ID: INMUNE

A live audio webcast of the call can be accessed by clicking here or using this link: View Source;tp_key=4727e947f4

A transcript will follow approximately 24 hours from the scheduled call. A telephone replay will also be available for approximately 30 days by dialing 1-844-512-2921 or 1-412-317-6671 (international) and entering PIN no. 11157984.

About XPro

XPro is a next-generation inhibitor of tumor necrosis factor (TNF) that is currently in clinical trial and acts differently than currently available TNF inhibitors in that it neutralizes soluble TNF (sTNF), without affecting trans-membrane TNF (tmTNF) or TNF receptors. XPro could have potential substantial beneficial effects in patients with neurologic disease by decreasing neuroinflammation. For more information about the importance of targeting neuroinflammation in the brain to improve cognitive function and restore neuronal communication visit this section of the INmune Bio’s website.

About CORDStrom

CORDStrom is a patent-pending cell medicine comprising aseptic, allogeneic, pooled human umbilical cord-derived mesenchymal stromal cells (hucMSCs) in suspension for injection or infusion. The CORDStrom platform leverages, among other things, proprietary screening, pooling and expansion techniques to create off-the-shelf, allogeneic, pooled hucMSCs as medicines to treat complex inflammatory diseases. CORDStrom products are designed to provide high-quality, off-the-shelf, batch-to-batch consistent, scalable, cGMP manufactured, potent cellular medicines that can be produced at low cost and with repeatable specification independent of donor characteristics. The CORDStrom product platform shares many similarities, including reagents, equipment, and procedures, with the Company’s INKmune oncology product, enabling the Company to leverage economies of scale, experienced staff, and other resources to strategically manufacture both products in a rotational campaign with resource and environmental efficiencies.

Initially developed at the INKmune manufacturing facilities utilizing UK academic grant funding, CORDStrom is an MSC product platform that shows promise as a first systemic therapy for potentially treating RDEB and many other debilitating conditions. While the first generation CORDStrom product is agnostic to disease indication, the platform enables creation of indication-specific products, which can be tuned for optimization of anti-inflammatory, immunomodulatory, wound healing, and other characteristics.

On March 27, 2025 Immatics N.V. (NASDAQ: IMTX, "Immatics" or the "Company"), a clinical-stage biopharmaceutical company active in the discovery and development of T cell-redirecting cancer immunotherapies, reported a business update and announced financial results for the quarter and full year ended December 31, 2024 (Press release, Immatics, MAR 27, 2025, View Source [SID1234651523]).

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"2025 will be marked by milestones across our TCR-T and TCR Bispecifics clinical portfolio, including advancing two of our main objectives for this year: firstly, reporting data on solid cancer types beyond melanoma, such as ovarian cancer, head and neck cancer and others and secondly, demonstrating that our next-generation, half-life extended TCR Bispecifics can deliver meaningful response rates in advanced solid cancer patients," said Harpreet Singh, Ph.D., CEO and Co-Founder of Immatics. "Additionally, the initiation of SUPRAME, the Phase 3 trial for our lead TCR-T cell therapy, IMA203, represents a transformative step in Immatics’ journey towards becoming a commercial-stage enterprise. We believe IMA203 offers patients and their treating physicians a cell therapy with impressive response rates and favorable tolerability in advanced melanoma. Notably, it requires no surgery or biopsy, has a fast turnaround time and a high manufacturing success rate. We are committed to rapidly delivering the first TCR therapeutic targeting PRAME to the market and to cancer patients, serving their unmet medical needs."

Full Year 2024 and Subsequent Company Progress

ACTengine Cell Therapy Programs

ACTengine IMA203 (PRAME)
IMA203 is Immatics’ lead TCR-T cell therapy, currently being evaluated in a Phase 3 trial (SUPRAME) in patients with previously treated advanced melanoma. IMA203 has the potential to become the first TCR therapeutic targeting PRAME to enter the market. In parallel, Immatics is priming its in-house, state-of-the-art TCR-T manufacturing facility to serve its planned commercial supply. In addition to maximizing the PRAME cell therapy opportunity, Immatics plans to expand IMA203 into uveal melanoma through the ongoing Phase 1b clinical trial. The current addressable patient population of PRAME/HLA-A*02:01-positive 2L unresectable or metastatic cutaneous melanoma in the US and EU52 is ~7,300 plus ~1,300 uveal melanoma patients in the US and EU5.

Clinical and commercial development plan for ACTengine IMA203 TCR-T
Based on the positive Phase 1b clinical data presented in 2024 and supported by the FDA RMAT designation3, Immatics has advanced its lead TCR-T product candidate, IMA203 targeting PRAME, into a randomized-controlled Phase 3 trial, called "SUPRAME" (NCT06743126). The trial commenced in December 2024. The first patient was randomized in the United States and enrollment continues as planned.

SUPRAME is a prospective, multicenter, open-label, randomized-controlled Phase 3 clinical trial evaluating the efficacy, safety and tolerability of IMA203 TCR-T in patients with unresectable or metastatic cutaneous melanoma who have received prior treatment with a checkpoint inhibitor. 360 HLA-A*02:01-positive patients will be randomized 1:1 to treatment with IMA203 or investigator’s choice of selected approved treatments in the 2L setting (nivolumab/relatlimab, nivolumab, ipilimumab, pembrolizumab, lifileucel (US), chemotherapy). Based on the Company’s discussions with the FDA, the primary endpoint for seeking full approval will be blinded independent central review ("BICR")-assessed (RECIST v1.1) progression-free survival (PFS). Given the expected median PFS of 2-3 months in this patient population4, as well as the median PFS of 6 months (> 1 year in patients with deep responses) observed in the data from the IMA203 Phase 1b trial, the Company has determined that utilizing PFS as the primary endpoint is the fastest pathway to seeking full approval and presents a more attractive commercial positioning as compared to objective response rate (ORR). Secondary endpoints for the trial include ORR, safety, DOR, OS and patient-reported outcomes.

The trial is planned to run internationally with approximately 50 sites in the United States and Europe.

Patient enrollment for SUPRAME is forecasted to be completed in 2026. A pre-specified interim data analysis will be triggered upon the occurrence of a defined number of events for PFS (progressive disease or death)5 anticipated to occur after approximately 200 patients are enrolled in 1Q 2026. The final analysis is planned for 4Q 2026. Immatics aims to submit a Biologics License Application (BLA) in 1Q 2027 for full approval and to launch IMA203 in 3Q 2027.

In addition to cutaneous melanoma, Immatics intends to expand the IMA203 TCR-T opportunity to treat uveal melanoma patients and will continue to evaluate IMA203 in this patient population through the ongoing Phase 1b trial.

Manufacturing capabilities
Immatics’ proprietary manufacturing process, timeline, capabilities and facility support late-stage clinical and commercial cell therapy development and supply.

IMA203 products are manufactured from a patient’s leukapheresis (with no surgery required) within 7 days, followed by 7-day QC release testing at >95% success rate6 to achieve the target dose (1-10×109 TCR-T cells). The Company’s state-of-the-art ~100,000 sq. ft. R&D and GMP manufacturing facility in the Houston Metropolitan Area was built with a modular design for efficient and cost-effective scalability (total of 8 manufacturing suites, plus further expansion space) to serve early-stage and registration-directed clinical trials as well as planned commercial supply. Through in-house manufacturing and QC testing, Immatics aims to better control the manufacturing process, shorten the turnaround time, ensure the manufacturing success rate and quality of the product and realize potential cost efficiencies, including manufacturing capacity optimization through scalability for a competitive and profitable commercial cell therapy product.

Clinical data on ACTengine IMA203 TCR-T as of October 2024
On October 10, 2024, Immatics provided a data update on IMA203 monotherapy in 28 heavily pretreated metastatic melanoma patients from the ongoing Phase 1b dose expansion part of the clinical trial in which patients were treated at the recommended Phase 2 dose (RP2D, 1 to 10 billion total TCR-T cells).

As of the data cut-off on August 23, 2024, treatment with IMA203 monotherapy in this melanoma patient population has demonstrated:

Confirmed objective response rate of 54% and an objective response rate of 62%
Disease control rate of 92% and tumor shrinkage in 88% of patients
12.1 months median duration of response, 6 months median progression-free survival and >1-year median progression-free survival in patients with deep responses
Median overall survival has not yet been reached at a median follow-up time of 8.6 months
IMA203 monotherapy has maintained a favorable tolerability profile with no treatment-related Grade 5 events in the entire safety population (N=70 Phase 1a and Phase 1b patients across all dose levels and all tumor types).

Immatics plans to present updated clinical data from the Phase 1b trial, including patients reported previously with longer follow-up and additional uveal melanoma patients, in 2025.

ACTengine IMA203CD8 TCR-T (GEN2) Monotherapy (PRAME)
IMA203CD8 is the Company’s second-generation cell therapy product candidate targeting PRAME. Given its pharmacology profile, once the target dose is reached, the Company intends to pursue the clinical development of this product in PRAME-positive solid cancers beyond melanoma, starting with gynecologic cancers.

On November 8, 2024, Immatics announced updated Phase 1 dose escalation clinical data on its next-generation ACTengine IMA203CD8 TCR-T cell therapy in 44 heavily pretreated HLA-A*02:01 and PRAME-positive patients with solid tumors, thereof 41 patients being evaluable for efficacy. Of note, these patients had been treated at substantially lower doses than IMA203 (GEN1), i.e. in a range of 0.2-0.48×109 TCR-T cells/m2 BSA (dose level 3) to 0.801-1.2×109 TCR-T cells/m2 BSA (dose level 4c) T cells infused.

As of the data cut-off on September 30, 2024, treatment with IMA203CD8 monotherapy demonstrated:

Confirmed objective responses observed in 41% of patients (at low doses, dose escalation ongoing)
Median duration of response of 9.2 months at a median follow-up of 13.1 months
Tumor shrinkage of target lesions in 84% of patients and disease control rate at week 6 of 85%
10 out of 17 responses were ongoing, of which three confirmed responses were ongoing at 14+, 15+ and 24+ months
Deep responses with ≥50% tumor size reduction were observed in 11 out of 17 responders. This group included two patients with complete response of target lesions, of which one patient showed a complete metabolic response according to a PET-CT scan

IMA203CD8 monotherapy has maintained a manageable tolerability profile in the 44 patients treated.

Based on the enhanced pharmacology of IMA203CD8 demonstrated in this trial, the evaluation of higher doses of IMA203CD8 in the ongoing dose escalation trial opens the possibility of addressing hard-to-treat solid tumor indications with both high- and medium-level PRAME copy numbers, such as ovarian cancer, uterine cancer, squamous non-small cell lung carcinoma, triple negative breast cancer and others.

The next clinical data update including dose escalation and ovarian cancer is planned in 2025.

TCR Bispecifics Programs

TCER IMA402 (PRAME)

To expand the PRAME opportunity to additional solid cancer types and earlier lines of treatment, the Company is focusing on its half-life extended TCR Bispecific, IMA402. Upon delivering clinical proof-of-concept ("PoC") in last-line melanoma, Immatics plans to explore its potential in gynecologic cancers, sqNSCLC, breast cancer and other solid tumor indications as well as earlier lines of solid cancers, such as first-line (1L) cutaneous melanoma.

On November 18, 2024, Immatics announced the first clinical data update from the ongoing Phase 1 dose escalation trial evaluating its next-generation, half-life extended TCR Bispecific molecule, TCER IMA402 targeting PRAME, in 33 heavily pretreated (3 median lines of prior therapies) HLA-A*02:01-positive patients with recurrent and/or refractory solid tumors.

As of the data cut-off on November 6, 2024, treatment with IMA402 demonstrated a favorable tolerability profile in the 33 patients treated.

Early pharmacokinetic data indicated a median half-life of approximately seven days, potentially enabling bi-weekly dosing. Initial signs of clinical anti-tumor activity have been observed and are associated with PRAME expression and IMA402 dose levels administered (up to 4 mg at DL8).

In the PRAME-negative patient population across all doses and indications, only one patient out of seven (14%) showed tumor shrinkage of -2.9%
25% (3/12) of patients (PRAME+ or not tested) treated at low doses (DL1-6) showed tumor shrinkage, including one unconfirmed partial response in cutaneous melanoma
78% (7/9) of patients (PRAME+ or not tested) treated at relevant doses (8 patients at DL7 and 1 patient at DL8) experienced shrinkage of their target lesions, including one confirmed partial response in melanoma ongoing at 3 months and three patients with ongoing stable disease at 6+ weeks (cut. melanoma), 3 months (ovarian cancer), 8+ months (uveal melanoma)

Based on the initial signs of dose-dependent and PRAME target expression-dependent clinical activity observed during dose escalation, the Company will continue to evaluate IMA402 at higher dose levels to determine the optimal therapeutic dose.

As of March 27, 2025, dose escalation remains ongoing at DL10 (8 mg) with MTD not reached.

The next update on the Phase 1a trial with clinical data at relevant dose levels in second-line and later melanoma is planned in 2025.

TCER IMA401 (MAGEA4/8)
Immatics is further harnessing the potential of its proprietary bispecific platform to develop innovative therapeutics and unlock more cancer types. The Company’s half-life extended TCR Bispecific, IMA401 targeting MAGEA4/8, is progressing through a Phase 1 trial in patients with sqNSCLC, HNSCC, bladder cancer and other solid tumor indications, with the primary goal of developing this product candidate in earlier treatment lines.

On September 16, 2024, Immatics announced the proof-of-concept clinical data for the first candidate of its next-generation, half-life extended TCR Bispecifics platform, TCER IMA401 (MAGEA4/8), during an oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024.

As of data cut-off on July 23, 2024, 35 heavily pretreated patients with recurrent and/or refractory solid tumors were treated with IMA401 monotherapy across nine escalating dose levels. The treated patient population was composed of patients with 16 different solid tumor indications who were both HLA-A*02:01 and MAGEA4/8-positive, had received a median of four and up to eight lines of prior systemic treatments and the majority had an ECOG performance status of ≥ 1.

Proof-of-concept clinical data from the Phase 1a first-in-human dose escalation basket trial showed initial anti-tumor activity in multiple tumor types, durable objective responses, including confirmed responses ongoing at 13+ months, a manageable tolerability profile and a half-life of 14+ days.

Treatment with IMA401 monotherapy in patients with relevant IMA401 doses and MAGEA4/8high levels (N=17) demonstrated:

Objective response rate of 29% with confirmed responses observed in 25% of patients
Disease control rate of 53% and tumor shrinkage of 53%
As the clinical trial progresses, the Company aims to further leverage the potential of IMA401 by focusing on the enrollment of indications with high MAGEA4/8 target expression, such as lung and head and neck cancer patients, seeking to optimize the treatment schedule. By further combining IMA401 with a checkpoint inhibitor, Immatics aims to generate relevant clinical data to position IMA401 as a combination therapy in earlier treatment lines.

The next update on IMA401 Phase 1a data, with a focus on head and neck cancer, is expected in 2025, and the Company plans to share data with a focus on non-small cell lung carcinoma in 2026.

Corporate Development

IMA203 and mRNA Combination (Moderna collaboration): In February 2025, the FDA granted IND clearance for a Phase 1 trial evaluating Immatics’ IMA203 PRAME TCR-T in combination with Moderna’s PRAME adaptive immune modulating therapy. The objective of the combination is to further enhance IMA203 T cell responses with the potential to significantly reduce turn-around time and costs through the infusion of a much lower cell dose. The first-in-human, Phase 1a/1b trial is a multicenter, open-label, dose escalation/de-escalation (adaptive design) trial evaluating the safety, tolerability and efficacy of the combination therapy in up to 15 patients with advanced or recurrent cutaneous melanoma and synovial sarcoma. Immatics is responsible for conducting the Phase 1 trial. Each party retains full ownership of its investigational PRAME compound, and the parties will fund the clinical study on a cost sharing basis. In November 2024, Immatics presented preclinical proof-of-concept data at SITC (Free SITC Whitepaper) supporting this combination.
In September 2024, Immatics regained full clinical development and commercialization rights to IMA401 due to ongoing portfolio prioritization efforts within Bristol Myers Squibb. The Phase 1 trial with IMA401 is ongoing and will continue to be conducted by Immatics.
Full Year 2024 Financial Results

Cash Position: Cash and cash equivalents as well as other financial assets total $628.0 million1 (€604.5 million) as of December 31, 2024, compared to $442.5 million1 (€425.9 million) as of December 31, 2023. The increase is mainly due to the public offering in January and October 2024, partly offset by ongoing research and development activities.

Revenue: Total revenue, consisting of revenue from collaboration agreements, was $161.9 million1 (€155.8 million) for the year ended December 31, 2024, compared to $56.1 million1 (€54.0 million) for the year ended December 31, 2023. The increase is mainly the result of the one-time revenue associated with the termination of the IMA401 and ACTallo collaborations by Bristol Myers Squibb during the year ended December 31, 2024.

Research and Development Expenses: R&D expenses were $153.9 million1 (€148.1 million) for the year ended December 31, 2024, compared to $123.3 million1 (€118.7 million) for the year ended December 31, 2023. The increase mainly resulted from costs associated with the advancement of the product candidates in clinical trials.
General and Administrative Expenses: G&A expenses were $48.2 million1 (€46.4 million) for the year ended December 31, 2024, compared to $39.7 million1 (€38.2 million) for the year ended December 31, 2023.

Net Profit and Loss: Net profit was $15.8 million1 (€15.2 million) for the year ended December 31, 2024, compared to a net loss of $98.3 million1 (€94.6 million) for the year ended December 31, 2023. The net profit largely resulted from the one-time revenue from collaborations, offset by ongoing expenses.

Full financial statements can be found in our Annual Report on Form 20-F filed with the Securities and Exchange Commission (SEC) on March 27, 2025, and published on the SEC website under www.sec.gov.

Upcoming Investor Conferences

Bank of America Healthcare Conference, Las Vegas (NV) – May 13 – 15, 2025
Jefferies Global Healthcare Conference, New York (NY) – June 3 – 5, 2025
To see the full list of events and presentations, visit View Source

On March 27, 2025 Genmab A/S (Nasdaq: GMAB) reported that the Japan Ministry of Health, Labour and Welfare has approved TIVDAK (tisotumab vedotin) for the treatment of advanced or recurrent cervical cancer that has progressed on or after cancer chemotherapy (Press release, Genmab, MAR 27, 2025, View Source [SID1234651522]). TIVDAK is the first and only ADC to be approved for people living with cervical cancer in Japan.

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In recent years, cervical cancer incidence and mortality rates have increased in Japan, particularly among women under age 50.i,ii,iii Moreover, patients with recurrent or metastatic cervical cancer whose disease has progressed after first-line therapy have limited treatment options.

"Patients with advanced or recurrent cervical cancer, in general, have a poor prognosis. The advent of new treatment options, especially for second-line or later treatment, is much needed," said Aikou Okamoto, M.D., Ph.D., Chief Professor, Department of Obstetrics and Gynecology at The Jikei University School of Medicine. "Cervical cancer treatment has advanced in recent years, but it is very meaningful that the approval of tisotumab vedotin as an ADC has increased the number of treatment options with a new mechanism of action that is expected to prolong overall survival. This is good news for patients and healthcare professionals."

The approval is based on data from the randomized, open-label, global Phase 3 innovaTV 301 clinical trial that evaluated the efficacy and safety of TIVDAK compared to chemotherapy in patients with advanced or recurrent cervical cancer who were previously treated with chemotherapy. The trial included 502 patients, 101 of which were Japanese. The trial met its primary endpoint of overall survival (OS), demonstrating a 30% reduction in risk of death (HR: 0.70 [95% CI: 0.54-0.89], two-sided p=0.0038) compared to chemotherapy. Median OS was 11.5 months [95% CI: 9.8-14.9] among patients treated with TIVDAK compared to 9.5 months [95% CI: 7.9-10.7] for patients who received chemotherapy. Secondary endpoints of progression-free survival (PFS) and confirmed objective response rate (ORR) were also met.

Adverse drug reactions occurred in 219 (87.6%) of 250 patients (including 50 Japanese patients) treated with TIVDAK. The most common (≥20%) adverse reactions included conjunctivitis (n=76; 30.4%), nausea (n=73; 29.2%), peripheral sensory neuropathy (n=67; 26.8%), alopecia (n=61; 24.4%), and epistaxis (n=57; 22.8%), at the data cutoff date of July 24, 2023.

"As a company, we understand the urgent need for patients with advanced cervical cancer whose disease has progressed," said Judith Klimovsky, M.D., Executive Vice President and Chief Development Officer of Genmab. "This approval marks an important step forward in transforming the treatment paradigm in Japan, ultimately bringing new hope and possibility to patients and their loved ones."

About the innovaTV 301 Trial
The innovaTV 301 trial (NCT04697628) is a global, 1:1 randomized, open-label Phase 3 trial evaluating tisotumab vedotin versus investigator’s choice of single agent chemotherapy (topotecan, vinorelbine, gemcitabine, irinotecan or pemetrexed) in 502 patients with recurrent or metastatic cervical cancer who received one or two prior systemic regimens in the recurrent or metastatic setting.

Patients with recurrent or metastatic cervical cancer with squamous cell, adenocarcinoma or adenosquamous histology, and disease progression during or after treatment with chemotherapy doublet +/- bevacizumab and an anti-PD-(L)1 agent (if eligible) are included. The primary endpoint was overall survival. The main secondary outcomes were progression-free survival and objective response rate.

The study was conducted by Seagen, which was acquired by Pfizer in December 2023, in collaboration with Genmab, European Network of Gynaecological Oncological Trial Groups (ENGOT, study number ENGOT cx-12) and the Gynecologic Oncology Group (GOG) Foundation (study number GOG 3057), as well as other global gynecological oncology cooperative groups. For more information about the Phase 3 innovaTV 301 clinical trial and other clinical trials with tisotumab vedotin, please visit www.clinicaltrials.gov.

About Tisotumab Vedotin
Tisotumab vedotin (approved under the brand name TIVDAK in the U.S. and Japan) is an antibody-drug conjugate (ADC) composed of Genmab’s human monoclonal antibody directed to tissue factor (TF) and Pfizer’s ADC technology that utilizes a protease-cleavable linker that covalently attaches the microtubule-disrupting agent monomethyl auristatin E (MMAE) to the antibody. Nonclinical data suggest that the anticancer activity of tisotumab vedotin is due to the binding of the ADC to TF-expressing cancer cells, followed by internalization of the ADC-TF complex and release of MMAE via proteolytic cleavage. MMAE disrupts the microtubule network of actively dividing cells, leading to cell cycle arrest and apoptotic cell death. In vitro, tisotumab vedotin also mediates antibody-dependent cellular phagocytosis and antibody-dependent cellular cytotoxicity.

On March 27, 2025 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers, reported financial results for the fourth quarter and full year ended December 31, 2024, and provides an overview of recent and upcoming business highlights (Press release, Plus Therapeutics, MAR 27, 2025, View Source [SID1234651521]).

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"Over the last twelve months, Plus has reported very promising safety and efficacy data for our lead drug REYOBIQ administered in our two most advanced CNS cancer programs," said Marc H. Hedrick, M.D., Plus Therapeutics President and Chief Executive Officer. "The recently raised capital, coupled with existing grant support, enables us to progress both our therapeutic programs to key clinical and regulatory milestones as well as commercially launch our CNSide diagnostic platform. The year 2025 has the potential to be transformational at Plus as we anticipate transitioning to an operational revenue generating company with the launch of CNSide. We are highly appreciative of our investors, partners and other stakeholders for their continued commitment to Plus as we deliver on our objectives and drive value."

Q4 2024 & RECENT HIGHLIGHTS AND MILESTONES

Corporate

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Raised $15.0 million in a private placement financing, enabling the Company to regain compliance with Nasdaq minimum stockholders’ equity requirement and extending runway into 2026
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Obtained a $2.0 million grant award advance from the Company’s existing $17.6 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT) to accelerate the development of REYOBIQ for our leptomeningeal metastases (LM) program
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Strengthened management team with key leadership appointments:
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Dr. Michael Rosol as Chief Development Officer – Dr. Rosol will lead the Company’s clinical, pre-clinical, and biomarker development activities
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Mr. Russell Bradley as President and General Manager of Plus Therapeutics’ wholly owned subsidiary, CNSide Diagnostics, LLC ("CNSide Diagnostics") – Mr. Bradley will provide leadership at CNSide with an immediate focus on commercialization of the diagnostic platform
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Dr. Jonathan Stein as Medical Director, CNSide Diagnostics – Dr. Stein will provide technical leadership to support CNSide Diagnostics, having experience in all aspects of diagnostic operations, compliance and regulatory affairs

REYOBIQ

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Received U.S. FDA agreement for the brand name REYOBIQ (Rhenium Re186 Obisbemeda) for the Company’s lead radiotherapeutic
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Published Phase 1 clinical trial results for REYOBIQ in the peer-reviewed publication Nature Communications, demonstrating safety and potential efficacy in treating recurrent glioblastoma (GBM), with patients receiving a radiation dose >100 Gy achieving a median overall survival of 17 months, more than double the standard of care. Additional details can be found here
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Granted U.S. FDA Orphan Drug Designation for REYOBIQ for the treatment of LM in patients with lung cancer

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Completed ReSPECT-LM Phase 1 single dose administration trial and determined the maximum feasible and recommended Phase 2 doses. Additional details can be found here
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Presented positive ReSPECT-LM Phase 1 interim data for LM at the 2024 SNO Annual Conference. Additional details can be found here
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Presented positive ReSPECT-LM Phase 1 interim data for breast cancer patients with LM at the 2024 San Antonio Breast Cancer Symposium. Additional details can be found here
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Expanded strategic agreement with Telix IsoTherapeutics Group, securing a reliable supply of cGMP Rhenium-186 for late-stage clinical trials and future commercialization of REYOBIQ. Additional details can be found here

CNSide

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Presented positive FORESEE clinical trial summary demonstrating utility of the CNSide Cerebrospinal Fluid Assay Platform ("CNSide") in diagnosis and clinical management of patients with LM. Additional details can be found here
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Presented real world, multi-institutional, longitudinal data showing the commercial utility of CNSide at the 2024 Society for Neuro-Oncology Annual Meeting. Additional details can be found here

UPCOMING EXPECTED EVENTS AND MILESTONES

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Full commercial launch of CNSide on track for 2025
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Presentations planned for the following upcoming medical conferences:
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Nuclear Medicine and Neuro-oncology Symposium (NMN) in Vienna, Austria (May 9-10, 2025); Title: "Diagnostic and Therapeutic Innovations in the Era of Precision Medicine – Nuclear Medicine Meets Neuro-Oncology" on May 9, 2025 by Dr. Andrew Brenner, M.D, Ph.D.
o
Society for Neuro-Oncology/American Society of Clinical Oncology (SNO/ASCO) CNS Metastases Conference in Baltimore, Maryland (August 14-16, 2025): Corporate Key Opinion Leader symposium, title to be determined
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Complete enrollment of Cohort 1 in the ReSPECT-LM Phase 1 multiple dose administration trial in 2025
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Complete end of Phase 1 meeting with the U.S. FDA for the ReSPECT-LM trial and determine next clinical steps in 2025
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Complete ReSPECT-GBM Phase 2 enrollment in 2025
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Obtain IND approval for the ReSPECT-PBC Phase 1/2 trial of REYOBIQ for pediatric ependymoma and high-grade glioma in H2 2025

FULL YEAR 2024 FINANCIAL RESULTS

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The Company’s cash and investments balance was $3.6 million at December 31, 2024 compared to $8.6 million at December 31, 2023.
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The Company recognized $5.8 million in grant revenue in the year ending December 31, 2024 and $4.9 million for the year ending December 31, 2023, which in both periods represents CPRIT’s share of the costs incurred for REYOBIQ development for the treatment of patients with LM
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Total operating loss for the year ending December 31, 2024 was $14.7 million versus $13.3 million for the year ending December 31, 2023. The increase is primarily due to increased expenditures related to the ReSPECT-LM trial

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Net loss for the year ending December 31, 2024 was $13.0 million, or $(1.95) per basic share versus $13.3 million, or $(4.24) per basic share, for the year ending December 31, 2023
FOURTH QUARTER & FULL YEAR 2024 RESULTS CONFERENCE CALL

The Company will hold a conference call and live audio webcast at 5:00 pm Eastern Time today to discuss its financial results and provide a general business update.

The live audio webcast will be available at ir.plustherapeutics.com/events.

Participants may also pre-register any time before the call here. Once registration is completed, participants will be provided a dial-in number with a personalized conference code to access the call. Please dial in 15 minutes prior to the start time.

Following the live call, a replay will be available on the Company’s website under the ‘For Investors’ section. The webcast will be available on the Company’s website for 90 days following the live call.

On March 27, 2025 Boundless Bio (Nasdaq: BOLD), a clinical-stage oncology company interrogating extrachromosomal DNA (ecDNA) biology to deliver transformative therapies to patients with previously intractable oncogene amplified cancers, reported financial results and business highlights for the fiscal quarter and full year ended December 31, 2024 (Press release, Boundless Bio, MAR 27, 2025, View Source [SID1234651520]).

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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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"We made important strides in 2024 as we became a public company and continued to advance BBI-355, our oral, selective CHK1 inhibitor in the Phase 1/2 POTENTIATE trial in patients with oncogene amplified cancers, and we look forward to reporting preliminary proof-of-concept data in the second half of this year," said Zachary Hornby, President and CEO of Boundless Bio. "Additionally, we have continued to identify new targets and are advancing an oral degrader of a novel kinesin identified by our Spyglass platform. We are on track to nominate a development candidate for our Kinesin program by mid-year, with the intention to submit an IND in the first half of 2026. We look forward to the year ahead as we continue to advance our pipeline to address the significant unmet need in patients with oncogene amplified cancers."

Research and Development Highlights and Upcoming Milestones

BBI-355, a novel, oral, potent CHK1 inhibitor designed to target replication stress in oncogene-amplified cancers

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Enrollment in the Phase 1/2 POTENTIATE clinical trial evaluating BBI-355 as a monotherapy and combination agent in patients with locally advanced or metastatic solid tumors with oncogene amplifications is ongoing.
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ECHO, a proprietary diagnostic for the detection of ecDNA amplified oncogenes, is in use in the POTENTIATE trial.
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Boundless expects to report preliminary clinical proof-of-concept safety and antitumor activity data in the second half of 2025.

Novel Kinesin program targeting ecDNA segregation and inheritance

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Boundless is advancing a preclinical program targeting a previously undrugged kinesin that is essential for proper ecDNA segregation and inheritance during cell division.
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Boundless expects to nominate a development candidate by mid-2025 and submit an investigational new drug application (IND) to the FDA in the first half of 2026.

Recent Corporate Highlights

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In February 2025, Boundless appointed Robert Doebele, M.D., Ph.D., as Chief Medical Officer. Dr. Doebele is a medical oncologist and previously served as Chief Medical Officer and Chief Scientific Officer at Rain Oncology, where he led the early and late-stage development of multiple oncology programs using biology-based, tumor-agnostic strategies.

Fourth Quarter and Full Year 2024 Financial Results

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Cash Position: Cash, cash equivalents, and short-term investments totaled $152.1 million as of December 31, 2024.
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Research and Development (R&D) Expenses: R&D expenses were $13.3 million for the fourth quarter of 2024 and $55.3 million for the full year 2024, compared to $10.4 million and $42.6 million for the same periods in 2023.
•
General and Administrative (G&A) Expenses: G&A expenses were $5.0 million for the fourth quarter of 2024 and $18.0 million for the full year 2024, compared to $3.4 million and $12.2 million for the same periods in 2023.
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Net Loss: Net loss totaled $16.4 million for the fourth quarter of 2024 and $65.4 million for the full year 2024, compared to $12.1 million and $49.4 million for the same periods in 2023.