On February 27, 2025 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, reported fourth quarter and full year 2024 financial results and corporate updates (Press release, Iovance Biotherapeutics, FEB 27, 2025, View Source [SID1234650709]).

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "In 2024, we successfully drove strong early adoption for our U.S. commercial launch of Amtagvi for patients with previously treated advanced melanoma. Strong demand and growth are continuing and on track to accelerate for both Amtagvi and Proleukin in 2025 and beyond in the U.S. and globally. Our top commercial priorities are to drive broader adoption and utilization, increase patient referrals, add large community practices to our authorized treatment center (ATC) network, expand the U.S. market, and secure regulatory approvals in three new markets outside the U.S. I am confident that Iovance is well positioned to remain the global leader in innovating, developing, and delivering current and future generations of TIL cell therapy for patients with cancer."

Fourth Quarter and Full Year 2024 Financial Results, Corporate Guidance, and Updates

Product Revenue and Guidance

Fourth Quarter 2024 Total Product Revenue: Iovance recognized total revenue of $73.7 million from sales of Amtagvi and Proleukin during the fourth quarter ended December 31, 2024.
Amtagvi Revenue: Product revenue was $48.7 million from U.S. Amtagvi sales in the fourth quarter of 2024, reflecting strong adoption with increasing demand. Amtagvi revenue is recognized upon patient infusion.
Proleukin Revenue: Product revenue also included $25.0 million in Proleukin sales in the fourth quarter of 2024. Proleukin is used in the Amtagvi treatment regimen and other commercial, clinical, manufacturing, and research settings, which provide additional revenue. Proleukin revenue is generally recognized upon delivery to distributors and ATCs.
Full Year 2024 Total Product Revenue: Total product revenue was $164.1 million and achieved the high end of the company’s guidance range of $160 to $165 million for the full year 2024. Full year product revenue included the first three quarters of sales following the U.S. launch of Amtagvi on February 20, 2024. The full year 2024 product revenue for Amtagvi and Proleukin was $103.6 million and $60.5 million, respectively.
Significant Amtagvi Growth Potential at Approximately 70 ATCs in 2025: Amongst current ATCs, 76% completed tumor resections, 64% infused one or more patients, and 13% infused more than 10 patients, highlighting significant growth potential at existing ATCs.
Full Year 2025 Total Product Revenue Guidance: Iovance is reaffirming total product revenue guidance within the range of $450 to $475 million for 2025, the first full calendar year of Amtagvi sales. Amtagvi adoption is on track to continue accelerating throughout 2025 with broader utilization, higher demand, and growth in community referrals. Iovance also expects significant growth in total product revenue for full year 2026, and beyond.
Gross margins are expected to increase over time and remain on track to surpass 70% over the next several years. In line with anticipated growth in Amtagvi demand, Proleukin revenue is also expected to increase significantly in 2025 and beyond.
Full Year 2025 Expense Guidance: Cash burn for full year 2025 is expected to be under $300 million, including completion of construction of the Iovance Cell Therapy Center (iCTC) manufacturing expansion.
Cash Position: As of February 26, 2025, Iovance had cash, cash equivalents, investments, and restricted cash of approximately $422 million. The current cash position and anticipated product revenue are expected to be sufficient to fund current and planned operations, including manufacturing expansion, into the second half of 2026.
Amtagvi (Lifileucel) U.S. Launch Highlights in Advanced Melanoma

The U.S. FDA approved Amtagvi (lifileucel) on February 16, 2024, as the first treatment option for patients with advanced melanoma after anti-PD-1 and targeted therapy. Amtagvi is the first FDA-approved T cell therapy for a solid tumor indication.
Approximately 70 U.S. ATCs are active across 32 states and 95% of addressable patients live within 200 miles of an ATC. Additional U.S. ATCs will be added steadily throughout 2025, focusing on quality ATCs with a high volume of eligible patients, including large community practice ATCs.
Community referral activities are increasing throughout the U.S. to drive additional patient volume to these ATCs. Large community practices are currently onboarding, creating a new and significant opportunity for more patients to receive Amtagvi after frontline therapy.
Manufacturing turnaround time is aligning with launch expectations of approximately 34 days from inbound to return shipment to ATCs. Efforts are underway to shorten the turnaround time in 2025. The commercial manufacturing experience remains consistent with prior clinical experience.
Amtagvi is a preferred second-line or subsequent therapy in the National Comprehensive Cancer Network guidelines for treatment of cutaneous melanoma.
Reimbursement remains successful, with an average financial clearance time of about three weeks.
Approximately 75% of Amtagvi patients are covered by private payers. To date, payers or plans covering more than 250 million lives have added Amtagvi to policies since its launch.
Launch Expansion into New Markets

Amtagvi has the potential to address more than 20,000 patients annually with previously treated advanced melanoma across the U.S. and initial global markets with significant populations of previously treated advanced melanoma patients.1
Regulatory dossiers are under review, submitted, or planned across multiple international markets for lifileucel for the treatment of adult patients with unresectable or metastatic melanoma after anti-PD-1 and targeted therapy. If approved, lifileucel will be the first and only approved therapy in this treatment setting in all markets.
A marketing authorization application (MAA) was submitted to the Medicines and Healthcare products Regulatory Agency in the United Kingdom for potential approval in the first half of 2025.
A new drug submission (NDS) to Health Canada was accepted for a prioritized 200-day review process through the Notice of Compliance with Conditions (NOC/c) policy for potential approval in mid-2025.
An MAA for all EU member states was accepted for review by the European Medicines Agency for potential approval in the second half of 2025.
Named patient programs are planned in the UK, France, Germany, Canada, Switzerland, and Australia in 2025 to provide reimbursed access to treatment prior to approval or final pricing and are also expected to provide initial revenue from these markets.
Additional regulatory submissions remain on track in 2025 and 2026, including Australia in the first half of 2025 and Switzerland in the second half of 2025.
A total of 15 active ATCs are targeted by year-end to support initial launch markets outside the U.S.
Recent Iovance TIL Cell Therapy Pipeline Highlights

Lifileucel in Frontline Advanced Melanoma
Strong momentum continues with global site activation and patient enrollment in the registrational TILVANCE-301 trial, which is intended to support accelerated and full U.S. approvals of Amtagvi in combination with pembrolizumab in frontline advanced melanoma, as well as full approval of Amtagvi in post-anti-PD-1 melanoma.
Lifileucel in Previously Treated Advanced Non-Small Cell Lung Cancer (NSCLC)
Iovance expects to share additional data in the second half of 2025 from the IOV-LUN-202 registrational Phase 2 trial in post-anti-PD-1 NSCLC.
The IOV-LUN-202 trial is intended to support a potential accelerated approval of lifileucel in post-anti-PD-1 NSCLC in the U.S., with an anticipated regulatory decision in 2027. The FDA previously provided positive regulatory feedback on the proposed potency matrix in NSCLC and the IOV-LUN-202 clinical trial design.
Enrollment in IOV-LUN-202 continues with high demand at clinical sites in the U.S., Canada, and Europe with additional site activations underway in new regions with strong track records for enrollment in NSCLC studies.
Lifileucel in Frontline Advanced NSCLC
Iovance is pursuing a frontline therapy strategy to integrate lifileucel plus pembrolizumab following chemotherapy for patients with EGFR wild type NSCLC, representing most patients with an unmet medical need in this setting. This regimen will be investigated in a new Cohort 3D in IOV-COM-202 trial. Cohort 3D results will inform a registrational and confirmatory trial design in frontline advanced NSCLC.
Lifileucel in Endometrial Cancer
Iovance is actively enrolling in the IOV-END-201 Phase 2 trial which is investigating lifileucel for advanced endometrial cancer patients who have progressed after platinum-based chemotherapy and anti-PD-1 therapy regardless of mismatch repair (MMR) status.
IOV-END-201 is supported by preclinical and manufacturing success data, as well as positive feedback from gynecological oncology experts. Initial results from IOV-END-201 are expected in the second half of 2025. There are no currently approved therapies for endometrial cancer following frontline post-anti-PD-1 therapy and chemotherapy, representing a significant opportunity for TIL cell therapy to address an additional unmet medical need in the post-anti-PD-1 treatment setting.

Next Generation TIL Pipeline

PD-1 Inactivated TIL Cell Therapy (IOV-4001): The Phase 2 efficacy portion of the IOV-GM1-201 trial in previously treated advanced melanoma and NSCLC continues to enroll rapidly. Iovance utilizes the TALEN technology licensed from Cellectis to develop other investigational gene-edited TIL cell therapies with multiple knockout targets to potentially improve efficacy.
Next Generation Interleukin-2 (IL-2) for TIL Treatment Regimen: A Phase 1/2 clinical trial was initiated to investigate IOV-3001, a second-generation, modified IL-2 analog for use in the TIL therapy treatment regimen. Non-human primate and IND-enabling studies of IOV-3001 demonstrated the potential for improved safety with strong effector T cell expansion.
Next Generation, Cytokine-Tethered TIL Therapy: IND-enabling studies are proceeding for IOV-5001, a genetically engineered, inducible, and tethered interleukin-12 (IL-12) TIL cell therapy. A clinical trial of a prior generation IL-12 TIL therapy at the National Cancer Institute showed improved efficacy with low cell doses, without the use of IL-2, and provides the rationale for modifications in IOV-5001 to enhance TIL efficacy while optimizing safety. In preclinical studies, IOV-5001 drove superior antitumor activity in a simulated tumor microenvironment. Iovance plans to submit an IND in 2025 to support clinical development for multiple indications.
Manufacturing Capacity Expansion

The iCTC, and an FDA-approved legacy contract manufacturer, currently have capacity to treat several thousand patients annually.
The company completed annual scheduled maintenance at the iCTC and successfully restarted full production.
Expansion is currently underway for the iCTC campus to supply TIL cell therapies to more than 5,000 patients annually in the next few years.
Iovance is also developing a manufacturing network to address more than 10,000 patients annually.
Corporate Updates

Dan Kirby joined Iovance’s Executive Leadership Team in the newly created role of Chief Commercial Officer in February 2025.
Raj Puri, M.D., Ph.D., was promoted to the newly created role of Chief Regulatory Officer in November 2024.
Iovance currently owns more than 250 granted or allowed U.S. and international patents and patent rights for Amtagvi and other TIL-related technologies that are expected to provide Amtagvi with exclusivity through at least 2042. This patent portfolio covers TIL compositions and methods of treatment and manufacturing in a broad range of cancers, with Gen 2 patent rights expected to provide exclusivity for Amtagvi into 2038 and additional patent rights, including methods of treating melanoma and compositions and methods for potency assays, expected to provide exclusivity into 2039 and 2042, respectively. Iovance also owns an industry-leading patent portfolio covering TIL products produced with genetic engineering, using core biopsies and peripheral blood as starting material, and using combinations of TIL products with checkpoint inhibitors, as well as Iovance’s proprietary IovanceCares system. More information on Iovance’s patent portfolio is available on the Intellectual Property page on www.iovance.com.
Fourth Quarter and Full Year 2024 Financial Results

As of February 26, 2025, Iovance’s cash position is approximately $422 million. The current cash position and anticipated product revenue are expected to be sufficient to fund current and planned operations into the second half of 2026.

Net loss for the fourth quarter of 2024 was $78.6 million, or $0.26 per share, compared to a net loss of $116.4 million, or $0.45 per share, for the fourth quarter of 2023. Net loss for the full year 2024 was $372.2 million, or $1.28 per share, compared to a net loss of $444.0 million, or $1.89 per share, for the full year 2023.

Revenue was $73.7 million for the fourth quarter of 2024 and consisted of product revenue from Amtagvi and Proleukin sales. Iovance recognized $48.7 million in revenue from Amtagvi infusions that were completed during the fourth quarter of 2024 and $25.0 million in global revenue for Proleukin. An additional $0.5 million in cash was received in the fourth quarter of 2024 for Amtagvi sales that will be recognized as revenue in the first quarter of 2025. Revenue for the fourth quarter of 2023 was $0.5 million for global sales of Proleukin.

Revenue for the full year 2024 was $164.1 million and reflected product revenue of $103.6 million from Amtagvi and $60.5 million from Proleukin. Revenue for the prior full year period 2023 was $1.2 million for global sales of Proleukin which Iovance began to recognize during the three-month period ended June 30, 2023.

The increases in revenue in the fourth quarter and full year 2024 over the prior year periods were primarily attributable to the U.S. launch of Amtagvi, including revenue recognized for Amtagvi, as well as significant growth in U.S. Proleukin revenue for use in the Amtagvi treatment regimen and global Proleukin sales.

Cost of sales includes inventory, overhead and related cash and non-cash expenses that are directly associated with sales of Amtagvi and Proleukin, as well as manufacturing costs for Amtagvi. Cost of sales for the three months ended December 31, 2024 was $45.5 million, primarily attributed to $9.1 million in period costs associated with patient drop off and manufacturing success rates, $5.9 million for non-cash amortization expense for intangible assets and fair value mark up of inventory, and $6.0 million in royalties payable on product sales. Cost of sales for the three months ended December 31, 2023 was $4.4 million, primarily related to non-cash amortization for intangible assets.

Cost of sales for the full year 2024 was $124.0 million, primarily related to $26.3 million in certain costs associated with patient drop off and manufacturing success rates, $26.2 million in non-cash amortization expense for intangible assets and fair value mark-up of inventory, and $14.2 million royalties payable on product sales. Cost of sales for the full year 2023 was $10.8 million, primarily related to non-cash amortization for intangible assets.

Increases in cost of sales in the fourth quarter and full year 2024 over the prior year periods were primarily attributable to the initiation of product sales, commercial manufacturing, and related cash and non-cash expenses tied to the U.S. launch of Amtagvi that began during the first quarter of 2024.

Research and development expenses were $72.2 million for the fourth quarter of 2024, a decrease of $15.3 million compared to $87.5 million for the fourth quarter of 2023. Research and development expenses were $282.3 million for the full year 2024, a decrease of $61.8 million compared to $344.1 million for the full year 2023.

The decreases in research and development expenses in the fourth quarter over the prior year period were primarily attributable to the transition of Amtagvi to commercial manufacturing. This decrease was partially offset by increases in headcount and related costs, including stock-based compensation, and clinical trial costs. The decrease in research and development expenses in the full year 2024 over the prior full year period was primarily attributable to the transition of Amtagvi to commercial manufacturing and lower clinical costs. These decreases were partially offset by increases in headcount and related costs, including stock-based compensation and lab and consumable costs.

Selling, general and administrative expenses were $42.5 million for the fourth quarter of 2024, an increase of $12.6 million compared to $29.9 million for the same period ended December 31, 2023. Selling, general and administrative expenses were $153.0 million for the full year 2024, an increase of $46.1 million compared to $106.9 million for the prior full year period.

The increase in selling, general and administrative expenses in the fourth quarter and full year 2024 compared to the prior year periods was primarily attributable to increases in headcount and related costs, including stock-based compensation, to support the growth in the overall business and related corporate infrastructure, as well as legal costs and costs incurred to support the commercialization of Amtagvi and Proleukin.

For additional information, please see the Company’s Selected Consolidated Balance Sheets and Statements of Operations below.

Webcast and Conference Call

Management will host a conference call and live audio webcast to discuss these results and provide a corporate update today at 4:30 p.m. ET. To listen to the live or archived audio webcast, please register at View Source The live and archived webcast can be accessed in the Investors section of the Company’s website, IR.Iovance.com, for one year.

On February 27, 2025 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, reported operational highlights and financial results for the fourth quarter and year ended December 31, 2024 (Press release, Intellia, FEB 27, 2025, View Source [SID1234650708]).

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"We are off to an excellent start in 2025 with renewed focus and strong operational execution across our three, pivotal Phase 3 studies," said Intellia President and Chief Executive Officer John Leonard, M.D. "We are excited by the clinical data presented during the fourth quarter. Our Phase 1/2 results in HAE suggest that NTLA-2002 could represent a functional cure for patients with HAE – for the first time a patient has the potential to be both free from attacks and free from chronic therapy. Similarly, the rapid, deep and durable reductions in serum TTR demonstrated to date in our Phase 1 study of nex-z in ATTR represent a highly differentiated profile that may offer patients an opportunity to stabilize or improve their clinical readouts in an otherwise unrelenting, progressive disease."

Fourth Quarter 2024 and Recent Operational Highlights

Hereditary Angioedema (HAE)

NTLA-2002: NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based therapy designed to knock out the KLKB1 gene in the liver, with the goal of lifelong control of HAE attacks after a single dose.
In January, Intellia announced the first patient was dosed with NTLA-2002 in the global Phase 3 HAELO study. The Company expects to complete enrollment in the second half of 2025.
The Company plans to submit a Biologics License Application in the second half of 2026 to support plans for a U.S. launch in 2027.
Intellia expects to present longer-term data from the ongoing Phase 1/2 study in 2025. The data will include patients in the Phase 2 portion who initially received a 25 mg dose or placebo and were subsequently given the 50 mg dose of NTLA-2002 selected for the Phase 3 study.
Transthyretin (ATTR) Amyloidosis

Nexiguran ziclumeran (nex-z, also known as NTLA-2001): Nex-z is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene in liver cells, thereby preventing the production of transthyretin (TTR) protein for the treatment of ATTR amyloidosis. Nex-z offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose. Nex-z has been generally well tolerated across all patients and at all dose levels tested. The most common treatment-related adverse event was an infusion reaction, which were mild or moderate; all patients were able to receive the intended dose of nex-z. Intellia leads development and commercialization of nex-z in collaboration with Regeneron.
ATTR Amyloidosis with Cardiomyopathy (ATTR-CM):
Enrollment in the pivotal Phase 3 MAGNITUDE trial is progressing ahead of our target projections and we anticipate enrollment to exceed 550 total patients by year end.
Intellia presented data from the ongoing Phase 1 study at the 2024 American Heart Association (AHA) Scientific Sessions and published the findings online in the New England Journal of Medicine. Across all patients (n=36), a single dose of nex-z led to consistently rapid, deep and sustained serum TTR reduction, regardless of baseline levels, through the latest follow-up. At month 12, the mean serum TTR reduction was 90%, and the mean absolute residual serum TTR concentration was 17 µg/mL. With 11 patients who have reached 24 months of follow-up, all patients continued to show a sustained response with no evidence of a waning effect over time. The consistently low levels of serum TTR are anticipated to reduce the rate of ongoing amyloid formation and potentially allow for amyloid clearance and improvement in cardiac function. Nex-z was generally well tolerated across all patients.
Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN):
We are actively screening patients for the Phase 3 MAGNITUDE-2 study and are on track to dose the first patient in the first quarter of 2025.
Intellia presented data from the ongoing Phase 1 study in November. At month 12, patients who received a dose of 0.3 mg/kg or higher (n=33) had a mean serum TTR reduction of 91% and mean absolute residual serum TTR concentration of 20 µg/mL. For the 16 patients who reached 24 months of follow-up, there was no change to their post-dose TTR levels. It is anticipated that greater TTR reduction may lead to a greater clinical benefit in patients with ATTRv-PN. Nex-z was generally well tolerated across all patients and at all dose levels tested.
In November, Intellia announced the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) to nex-z for the treatment of ATTRv-PN.
Intellia expects to present longer-term data from both ATTR-CM and ATTRv-PN patients in the Phase 1 study in 2025. The data will include updated measures of clinical efficacy and safety.
Platform Update

Intellia continues to apply novel technologies, such as CRISPR-based gene editing technologies and lipid nanoparticle (LNP) delivery technologies, to develop in vivo and ex vivo product candidates. Treating—and potentially curing—a broad range of severe diseases requires the application of multiple technologies. With Intellia’s proprietary technology at the core of the platform, the Company continues to research and develop new gene editing and delivery technologies to expand the therapeutic opportunities, furthering progress on the frontier of genetic medicine.
Corporate Update

On January 9, 2025, the Company announced that, after a strategic review of its business, it elected to prioritize late-stage programs – NTLA-2002 for HAE and nex-z for ATTR amyloidosis – and select research investments to focus on near-term value creation. As a result, the Company discontinued NTLA-3001 and other, undisclosed programs, and is reducing its workforce by approximately 27% in 2025. The Company expects to incur charges of approximately $8.0 million for severance and other employee termination-related costs in the first quarter of 2025.
Upcoming Events

The Company will participate in the following events during the first quarter of 2025:

AAAAI/WAO Joint Congress, March 1, San Diego
TD Cowen 45th Annual Health Care Conference, March 4, Boston
Leerink 2025 Global Biopharma Conference, March 10, Miami
Barclays 27th Annual Global Healthcare Conference, March 11, Miami
Jefferies Biotech on the Beach Summit, March 12, Miami
Fourth Quarter and Full-Year 2024 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $861.7 million as of December 31, 2024, compared to $1.0 billion as of December 31, 2023. The Company’s cash position as of December 31, 2024, is expected to fund operations into the first half of 2027.
Collaboration Revenue: Collaboration revenue was $12.9 million during the fourth quarter of 2024, compared to negative $1.9 million during the fourth quarter of 2023. The $14.8 million increase was mainly driven by collaboration revenue received under the Regeneron agreements.
R&D Expenses: Research and development (R&D) expenses were $116.9 million during the fourth quarter of 2024, compared to $109.0 million during the fourth quarter of 2023. The $7.9 million increase was primarily driven by the advancement of our lead programs. Stock-based compensation expense included in R&D expenses was $24.4 million for the fourth quarter of 2024.
G&A Expenses: General and administrative (G&A) expenses were $32.4 million during the fourth quarter of 2024, compared to $29.0 million during the fourth quarter of 2023. The $3.4 million increase was primarily related to stock-based compensation. Stock-based compensation expense included in G&A expenses was $15.2 million for the fourth quarter of 2024.
Net Loss: Net loss was $128.9 million for the fourth quarter of 2024, compared to $132.2 million during the fourth quarter of 2023.
Conference Call to Discuss Fourth Quarter and Full-Year 2024 Results

The Company will discuss these results on a conference call today, Thursday, February 27 at 8 a.m. ET.
To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.
Please visit this link for a simultaneous live webcast of the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com, beginning on February 27 at 12 p.m. ET.

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.

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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 Immutep reported results for half-year ended 31 December 2024 (Press release, Immutep, FEB 27, 2025, View Source [SID1234650705]).

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