On May 14, 2024 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-clinical stage biotechnology company pioneering nanoparticle-based therapeutic approaches to expand treatment possibilities for patients with cancer and other major diseases, reported that the U.S. Food and Drug Administration ("US FDA") issued a Study May Proceed Letter for a randomized Phase 2 study evaluating NBTXR3 for the treatment of patients with stage 3, unresectable non-small cell lung cancer ("NSCLC") (Press release, Nanobiotix, MAY 14, 2024, View Source [SID1234643232]). An IND to support this trial was submitted by the global trial sponsor, Johnson & Johnson Enterprise Innovation Inc., a Johnson & Johnson company.

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"The NBTXR3 collaboration established by our global licensing agreement with Janssen Pharmaceutica NV continues to make progress toward our goal of reaching millions of patients with cancer around the world. The US FDA acceptance of the protocol for this new Phase 2 study has the potential to expand the NBTXR3 development pipeline to a new indication where innovation could potentially provide important outcomes," said Louis Kayitalire, MD, chief medical officer at Nanobiotix. "We look forward to continuing to prepare for the launch of the study."

About NBTXR3
NBTXR3 is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. Its proof-of-concept was achieved in soft tissue sarcomas for which the product received a European CE mark in 2019. The product candidate’s physical mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.

Radiotherapy-activated NBTXR3 is being evaluated across multiple solid tumor indications as a single agent or in combination with anti-PD-1 immune checkpoint inhibitors, including in NANORAY-312—a global, randomized Phase 3 study in locally advanced head and neck squamous cell cancers. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the Phase 3 study.

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a collaboration strategy to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 Nanobiotix entered into a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to sponsor several Phase 1 and Phase 2 studies evaluating NBTXR3 across tumor types and therapeutic combinations. In 2023, Nanobiotix announced a license agreement for the global co-development and commercialization of NBTXR3 with Janssen Pharmaceutica NV.

On May 14, 2024 Mural Oncology plc (Nasdaq: MURA), a clinical-stage immuno-oncology company developing novel, investigational engineered cytokine therapies designed to address areas of unmet need for patients with a variety of cancers, reported financial results for the first quarter of 2024 and provided a business update (Press release, Mural Oncology, MAY 14, 2024, View Source [SID1234643231]).

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"We remain on track to share data readouts in the first half of next year from our two late-stage, potentially registrational studies of nemvaleukin. We are also excited about the potential of our less frequent dosing regimen of nemvaleukin that we are evaluating in a clinical trial as announced in March," said Caroline Loew, Ph.D., CEO of Mural Oncology. "As we look to further strengthen our pipeline, we have made progress with our preclinical programs for interleukin-18 (IL-18) and IL-12. Both are targets that have generated a great deal of interest in the immuno-oncology space, and we are diligently working to make candidate nominations for each program. We continue to be well capitalized to achieve our key clinical readouts and are laser-focused on the delivery of our goals."

Recent Corporate Highlights and Upcoming Milestones

The company’s late-stage clinical trials remain on track, with details as follows:
ARTISTRY-7 is a potentially registrational, phase 3 clinical trial evaluating nemvaleukin in combination with pembrolizumab compared to investigators’ choice of chemotherapy in approximately 448 patients with platinum-resistant ovarian cancer. Mural expects to report interim overall survival (OS) results based on approximately 75% of events in the first quarter of 2025. The company anticipates reporting final OS results in the second quarter of 2026.
Cohort 2 of ARTISTRY-6 is a potentially registrational, phase 2 clinical trial evaluating nemvaleukin as a monotherapy in 90 patients with mucosal melanoma. The company expects to report top-line data results from cohort 2 of ARTISTRY-6 in the first half of 2025.
Mural is evaluating a newly selected dose of less-frequent intravenous (LFIV) nemvaleukin in open-label cohorts of patients with cutaneous melanoma in ARTISTRY-6. The new dosing regimen is a shift from five daily infusions (days 1-5) to two infusions (days 1 and 8), per three-week cycle. The company expects preliminary data readouts in the monotherapy cohort in the first half of 2025, and in the combination cohort with pembrolizumab in the second half of 2025.
The company looks forward to presenting data from ARTISTRY-3, an evaluation of the LFIV dosing of nemvaleukin, at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June 2024.
Mural presented IL-18 and IL-12 preclinical data for the first time at the American Association of Clinical Research (AACR) (Free AACR Whitepaper) annual meeting in April 2024 that outlined the company’s novel approach to protein engineering. Both poster presentations are available at www.muraloncology.com/publications.
IL-18 poster synopsis: Native IL-18 is a potent immune-stimulating cytokine, but its efficacy is blunted by IL-18 binding protein (IL-18BP), a high affinity decoy receptor that binds to, and neutralizes, IL-18, thereby rendering it ineffective. Its potency is also limited by its short half-life. Mural’s IL-18 variant contains mutations designed to eliminate binding to IL-18BP while minimally impacting the native IL-18 structure. The company has also fused IL-18 to protein scaffolds to extend the half-life and increase IL-18’s exposure. Together, these may lead to a more durable immunological effect.
IL-12 poster synopsis: Native IL-12 is a highly potent pro-inflammatory cytokine, but because of its very narrow therapeutic index, it can be toxic with systemic exposure. Mural’s IL-12 variant splits the molecule into two inactive monomers: IL12p35 and IL-12p40. These individual subunits are then separately fused to antibody fragments, which deliver and concentrate IL-12 specifically in the tumor microenvironment with the goal of limiting systemic exposure.
Mural intends to nominate development candidates for these investigational engineered IL-18 and IL-12 cytokine therapies later this year.
Financial Results for the Quarter Ended March 31, 2024

Cash Position: As of March 31, 2024, cash, cash equivalents, and marketable securities were $231.7 million.
R&D Expenses: Research and development expenses were $26.9 million for the first quarter of 2024 compared to $40.4 million for the first quarter of 2023. The decrease in expenses was primarily related to decreased headcount compared to the headcount allocated to the company by the former parent prior to the separation, decreased spend on the ARTISTRY-1 and ARTISTRY-2 trials as activities related to the ARTISTRY-1 and ARTISTRY-2 trials wound down in 2023, and decreased manufacturing spend on other programs. These decreases were partially offset by increased spend on the ARTISTRY-7 trial related to increased enrollment and associated clinical trial expenses.
G&A Expenses: General and administrative expenses were $7.2 million for the first quarter of 2024 compared to $3.7 million for the first quarter of 2023. The increase in expenses was primarily due to increases in employee-related expenses and professional fees associated with operating as a standalone public company after the separation.
Net Loss: Net loss was $30.9 million for the first quarter of 2024 compared to $46.5 million for the first quarter of 2023.
Financial Guidance

The company reaffirms guidance that its cash, cash equivalents, and marketable securities are expected to fund its operations into the fourth quarter of 2025.
As noted in the 2023 year-end financial results press release, Mural anticipates reporting lower year-over-year operating expenses in 2024. Also, management forecasts higher operating expenses in 2024 versus 2025 due to the timing of clinical trial expenses.
About Nemvaleukin
Nemvaleukin alfa (nemvaleukin) is a novel, engineered cytokine designed to leverage antitumor effects of the IL-2 pathway while mitigating its hallmark toxicities that limit its use. Nemvaleukin selectively binds to the intermediate-affinity IL-2 receptor (IL-2R) and is sterically occluded from binding to the high-affinity IL-2R. Because of this molecular design, nemvaleukin treatment leads to preferential expansion of antitumor CD8+ T cells and natural killer cells, with minimal expansion of immunosuppressive regulatory T cells. Nemvaleukin is currently being evaluated in two potentially registrational late-stage trials.

About Mural Oncology’s IL-18 Program
IL-18 is a potent immune-stimulating cytokine, but its efficacy is blunted by IL-18 binding protein (IL-18BP), a high affinity decoy receptor that binds to, and neutralizes, IL-18, thereby rendering it ineffective. Native IL-18’s potency is also limited by its short half-life. Mural Oncology’s novel approach to protein engineering is designed to mitigate these issues. First, Mural introduced mutations to IL-18 that eliminate binding to IL-18BP while minimally impacting the native IL-18 structure. Second, it fused IL-18 to protein scaffolds which extend the half-life and increase IL-18’s exposure. Together, these have demonstrated more durable immunological effect in preclinical studies. Mural intends to nominate a development candidate for its IL-18 program by the end of this year.

About Mural Oncology’s IL-12 Program
Native IL-12 is a highly potent pro-inflammatory cytokine, but because of its very narrow therapeutic index, it can also be toxic with systemic exposure. To mitigate this hallmark toxicity, Mural, through its novel approach to protein engineering, split the IL-12p70 heterodimer into two inactive monomers: IL12p35 and IL-12p40. These individual subunits are then separately fused to antibody fragments and sequentially injected, which deliver and concentrate IL-12 specifically in the tumor microenvironment to limit systemic exposure. In preclinical studies, Mural’s engineered IL-12 achieved the desired reduction in serum while maintaining tumor concentrations providing the potential to reduce systemic toxicities. Mural intends to nominate a development candidate for its IL-12 program by the end of this year.

On May 14, 2024 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported that it has completed enrollment of 85 patients in the Phase 2 portion of KOMET-001, a registration-directed clinical trial of the Company’s menin inhibitor, ziftomenib (KO-539), in patients with relapsed or refractory (R/R) NPM1-mutant acute myeloid leukemia (AML) (Press release, Kura Oncology, MAY 14, 2024, View Source [SID1234643230]). The Company expects to report topline data from the trial in early 2025.

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"We are thrilled to announce this critical milestone, which brings us one step closer to delivering ziftomenib as a potentially best-in-class treatment for patients with genetically defined acute leukemias," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "Our confidence is supported by our recently announced Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA), which recognizes ziftomenib’s potential as an innovative medicine for patients with R/R NPM1-mutant AML and is intended to expedite review as we prepare for submission of a New Drug Application. We are grateful for the KOMET-001 investigators, patients and their families, and we look forward to sharing topline data from this pivotal study early next year."

Kura announced the first patients dosed in the Phase 2 portion of KOMET-001 in February 2023. The registration-directed study is designed to assess evidence of clinical activity, safety and tolerability of ziftomenib in patients with R/R NPM1-mutant AML, with a primary endpoint of complete response. The study has completed enrollment of the 85 patients necessary to support the primary endpoint analysis.

"The rapid enrollment of this study reflects the urgent need for more effective treatment options in AML as well as the potential for ziftomenib to address this need," said Eunice Wang, M.D., Chief of the Leukemia Service at Roswell Park Comprehensive Cancer Center and principal investigator of the trial. "NPM1-mutant AML represents approximately 30% of new AML cases annually and is a disease of significant unmet need for which there is no approved targeted therapy. The favorable safety profile and encouraging clinical activity demonstrated by ziftomenib to date offer the potential to transform the standard of care for these AML patients."

About NPM1-mutant AML

AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1 mutations are among the most common, representing approximately 30% of AML cases. While patients with NPM1-m AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the R/R setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A and IDH1/2, with prognosis heavily influenced by the occurrence of co-occurring mutations. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5.3 months in 3rd line and 3.5 months following the 4th line.1 There are currently no FDA-approved therapies targeting NPM1-m AML.

About Ziftomenib

Ziftomenib is a novel, once-daily, oral investigational drug candidate targeting the menin-KMT2A/MLL protein-protein interaction for treatment of genetically defined AML patients with high unmet need. In the KOMET-001 Phase 1 study, ziftomenib demonstrated an encouraging safety profile and tolerability with reported events most often consistent with features and manifestations of underlying disease. Clinical activity of ziftomenib as a monotherapy was optimal at the 600 mg daily dose and a 35% complete remission rate was observed in 20 patients with NPM1-mutant AML treated at the recommended Phase 2 dose (600 mg). Ziftomenib has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for the treatment of R/R NPM1-mutant AML. Additional information about clinical trials for ziftomenib can be found at kuraoncology.com/clinical-trials/#ziftomenib.

On May 14, 2024 Kintara Therapeutics, Inc. (Nasdaq: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company focused on the development of new solid tumor cancer therapies, reported financial results for its fiscal third quarter ended March 31, 2024, and recent corporate developments (Press release, Kintara Therapeutics, MAY 14, 2024, View Source [SID1234643229]).

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Recent Corporate Developments

Announced that Kintara had entered into a definitive merger agreement (the "Merger Agreement") with TuHURA Biosciences, Inc. ("TuHURA"), a Phase 3 registration-stage immune-oncology company developing novel technologies to overcome resistance to cancer immunotherapy, and Kayak Mergeco, Inc., Kintara’s wholly-owned subsidiary, whereby Kayak Mergeco will merge with and into TuHURA with TuHURA surviving the merger and becoming Kintara’s direct, wholly-owned subsidiary(the "Merger"). Pursuant to the terms of the Merger, stockholders of TuHURA will receive shares of Kintara common stock. Kintara’s existing stockholders will receive contingent value rights ("CVR"), entitling them to receive shares of common stock upon achievement of enrollment of a minimum of 10 patients in the REM-001 study, with such patients each completing 8 weeks of follow-up on or before December 31, 2025. Under the terms of the Merger Agreement, on a pro forma basis, Kintara’s stockholders post-Merger are expected to collectively own approximately 2.85%, or approximately 5.45% including the shares underlying the CVR if the milestone is achieved, of the common stock of the post-Merger combined company on a fully-diluted basis. The transaction is expected to close in the third quarter of 2024. (April 2024)

Announced the expansion of the inclusion criteria in the open label 15-patient REM-001 study in cutaneous metastatic breast cancer (CMBC) to include patients receiving pembrolizumab (KEYTRUDA) for at least three months at screening. (March 2024)

Announced the initiation of an open label 15-patient study in CMBC patients which is evaluating REM-001, a second-generation photodynamic therapy (PDT) photosensitizer agent, and is designed to test the 0.8 mg dose as well as optimize the study design in advance of a Phase 3 trial initiation. The primary endpoint in the study is Best Overall Objective Response Rate (bORR) (complete response or partial response) of the target treatment fields at any time from treatment up to, and including, week 24. The majority of the costs to run this study will be covered by the $2.0 million Small Business Innovation Research (SBIR) grant Kintara was awarded from the National Institutes of Health (NIH). (February 2024)

Announced that Kintara received a letter from The Nasdaq Stock Market LLC stating it had regained compliance with Nasdaq’s minimum stockholders’ equity requirement. (February 2024)
Summary of Financial Results for Fiscal Year 2023 Third Quarter Ended March 31, 2024

As of March 31, 2024, Kintara had cash and cash equivalents of approximately $6.35 million.

For the three months ended March 31, 2024, Kintara reported a net loss of approximately $2.0 million, or $0.05 per share, compared to a net loss of approximately $3.3 million, or $1.94 per share, for the three months ended March 31, 2023. The decreased net loss for the three months ended March 31, 2024, compared to the three months ended March 31, 2023, was largely attributed to lower research and development expenses which was primarily due to lower clinical development costs. General and administrative costs were higher during the same period primarily due to an increase in professional fees related to the proposed transaction with TuHURA.

Selected Balance Sheet Data (in thousands)

March 31,
2024

June 30,
2023

$

$

Cash and cash equivalents

6,351

1,535

Working capital

5,414

188

Total assets

7,446

3,979

Total stockholders’ equity

5,922

731

Selected Statement of Operations Data (in thousands, except per share data)

For the three months ended

March 31,

March 31,

2024

2023

$

$

Research and development

592

2,005

General and administrative

1,493

1,297

Other loss (income)

(74)

(38)

Net loss for the period

(2,011)

(3,264)

Series A Preferred cash dividend

(2)

(2)

Net loss for the period attributable to common stockholders

(2,013)

(3,266)

Basic and fully diluted weighted average number of shares

44,562

1,681

Basic and fully diluted loss per share

(0.05)

(1.94)

For the nine months ended

March 31,

March 31,

2024

2023

$

$

Research and development

2,562

7,235

General and administrative

3,054

4,212

Other loss (income)

(70)

(133)

Net loss for the period

(5,996)

(11,314)

Series A Preferred cash dividend

(6)

(6)

Series C Preferred stock dividend

(173)

(362)

Net loss for the period attributable to common stockholders

(6,175)

(11,682)

Basic and fully diluted weighted average number of shares

16,772

1,596

Basic and fully diluted loss per share

(0.37)

(7.32)

Kintara’s financial statements as filed with the U.S. Securities Exchange Commission can be viewed on the Company’s website at: View Source

On May 14, 2024 IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, immune-modulating therapeutic cancer vaccines based on its T-win platform, reported financial results for the first quarter ended March 31, 2024 (Press release, IO Biotech, MAY 14, 2024, View Source [SID1234643228]).

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"This will be a transformative year for IO Biotech as we approach the interim analysis of our pivotal Phase 3 study in first-line advanced melanoma, which, if supportive, could allow for a submission of a Biologics License Application (BLA) to the United States (US) Food and Drug Administration (FDA) this year," said Mai-Britt Zocca, Ph.D., President and CEO of IO Biotech. "Last year, we expanded our Phase 3 study to potentially bring in the time to reach the primary endpoint of progression free survival and we concluded enrollment in the fourth quarter of 2023 with 407 patients. As we prepare for the interim analysis, we now have greater visibility to estimate the timing of reaching 226 events needed for the PFS analysis, which we now project will occur in the first half of 2025."

Dr. Zocca continued, "With continued execution of our Phase 3 study and its planned interim analysis as our main priorities this year, we also continue both of our Phase 2 basket studies: completing enrollment in our IOB-022 study; and rapidly enrolling and expanding our newest basket study, IOB-032, in the neo-adjuvant/adjuvant settings of melanoma and squamous cell carcinoma of the head and neck (SCCHN). Finally, as we prepare for potential commercialization and partnering, I am very pleased with the strength of expertise we have added to our leadership team with the additions of Marjan Shamsaei as Senior Vice President, Commercial and Portfolio Lead, and Faiçal Miyara as Chief Business Officer. I founded IO Biotech 10 years ago and couldn’t be more proud of all that we have accomplished, and, with such a strong team in place, confident in what lies ahead."

Recent Business Highlights

•
The company recently completed enrollment of 407 patients in its pivotal Phase 3 trial (IOB-013/KN-D18) of IO102-IO103 in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in advanced melanoma. The primary endpoint of the Phase 3 trial is PFS. The PFS analysis will be conducted when 226 events have occurred in the trial, which, based on the expanded trial size, reaching full enrollment ahead of schedule and the events to date in the study, the company now projects will occur in the first half of 2025. Additionally, a planned interim analysis of ORR will be conducted when the first 225 randomized patients reach one year of treatment in June 2024. The outcome of this analysis is expected in the third quarter of 2024 and, if supportive, we believe could allow for submission of a BLA for accelerated approval in the US.

•
The independent data monitoring committee (IDMC) for the company’s IOB-013/KN-D18 Phase 3 trial convened its fourth meeting in March 2024 and recommended that the trial continue without modifications.

•
The company’s Phase 2 basket trial (IOB-022/KN-D38) evaluating IO102-IO103 in combination with pembrolizumab in patients with metastatic non-small cell lung cancer (NSCLC) or recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) completed enrollment; the company plans to submit abstracts with updates from this study to medical meetings in the fall of 2024.

•
The company’s Phase 2 solid tumor basket trial (IOB-032/PN-E40) studying treatment with IO102-IO103 in combination with pembrolizumab given before (neo-adjuvant) and after (adjuvant) surgery with curative intent in patients with resectable melanoma or SCCHN has been enrolling patients since December 2023 in cohorts A (melanoma) and B (SCCHN) in the US, European Union (EU) and Australia. Cohort A is now fully enrolled and the company recently expanded the study to include a randomized cohort C in melanoma, in which patients are randomized either to IO102-IO103 in combination with pembrolizumab or to pembrolizumab alone.

•
In April, a poster presentation of new non-clinical data further supporting the dual mechanism of action of the company’s lead cancer vaccine, IO102-IO103, was delivered at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024 in San Diego, California. While further studies are needed to fully discern the relationship between IDO1+/PD-L1+ target populations within the TME and the impact of IDO1/PD-L1 targeted vaccination, we believe the data presented support the use of a dual antigen approach to reduce the immunosuppression and enhance anti-tumor effect.

•
In April, the company strengthened its executive team with the appointments of Faiçal Miyara, Ph.D., as Chief Business Officer, and Marjan Shamsaei, Pharm.D., as Senior Vice President, Commercial and Portfolio Lead.

First Quarter 2024 Financial Results

•
Net loss for the three months ended March 31, 2024, was $19.5 million, compared to $17.0 million for the three months ended March 31, 2023.

•
Research and development expenses were $14.3 million for the three months ended March 31, 2024, compared to $11.9 million for the three months ended March 31, 2023. The increase was primarily related to clinical trial-related activities for the company’s IO102-IO103 therapeutic cancer vaccine candidate, including the continued execution of the company’s pivotal Phase 3 clinical trial. The company recognized $0.6 million in research and development equity-based compensation for the three months ended March 31, 2024, compared to $0.7 million for the three months ended March 31, 2023.

•
General and administrative expenses were $5.9 million for the three months ended March 31, 2024, compared to $6.0 million for the three months ended March 31, 2023. The company recognized $1.0 million in general and administrative equity-based compensation for the three months ended March 31, 2024, compared to $1.2 million for the three months ended March 31, 2023.

•
Cash and cash equivalents as of March 31, 2024 were $118.0 million, compared to $143.2 million at December 31, 2023. During the three months ended March 31, 2024, the company used cash, cash equivalents and restricted cash of $24.9 million. The increase in cash use was primarily driven by milestone payments and payment of other accrued expenses associated with clinical trials, as well as the payment of year-end bonuses. The company continues to expect that it will have sufficient cash to run the company into the fourth quarter of 2025.

About IO102-IO103

IO102-IO103 is an investigational off-the-shelf therapeutic cancer vaccine designed to kill both tumor cells and immune-suppressive cells in the tumor microenvironment (TME) by stimulating activation and expansion of T cells against indoleamine 2,3-dioxygenase (IDO) positive and programmed death-ligand 1 (PD-L1) positive cells. The company is currently conducting a pivotal Phase 3 trial (IOB-013/KN-D18; NCT05155254) investigating IO102-IO103 in combination with pembrolizumab versus pembrolizumab alone in patients with advanced melanoma, a Phase 2 basket trial (IOB-022/KN-D38; NCT05077709) investigating IO102-IO103 in combination with pembrolizumab as first line treatment in patients with solid tumors, and a Phase 2 basket trial (IOB-032/PN-E40; NCT05280314) investigating IO102-IO103 in combination with pembrolizumab as neo-adjuvant/adjuvant treatment of patients with solid tumors.

The clinical trials are sponsored by IO Biotech and conducted in collaboration with Merck and Merck is supplying pembrolizumab. IO Biotech maintains global commercial rights to IO102-IO103.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About the IOB-013/KN-D18 Pivotal Phase 3 Clinical Trial

IOB-013/KN-D18 (Clinical Trials.gov: NCT05155254) is an open label, randomized Phase 3 clinical trial of IO102-IO103 in combination with pembrolizumab versus pembrolizumab alone in patients with previously untreated, unresectable or metastatic (advanced) melanoma, being conducted in collaboration with Merck. Patients have been enrolled from centers across the United States, Europe, Australia, Turkey, Israel and South Africa. The primary endpoint of the study is progression free survival. Biomarker analyses will also be conducted. IO Biotech is sponsoring the Phase 3 trial and Merck is supplying pembrolizumab. IO Biotech maintains global commercial rights to IO102-IO103.

About the IOB-013/KN-D18 Clinical Trial Endpoints

The primary endpoint of the IOB-013/KN-D18 trial is progression free survival (PFS). The PFS analysis is event-driven and will be conducted when 226 events have occurred in the trial, which the company estimates will take place in the first half of 2025. Additionally, there is a planned per-protocol interim analysis of overall response rate when the first 225 randomized patients reach one year of treatment in mid-2024. The outcome of this analysis is expected in the third quarter of 2024. There is a high statistical bar for the Phase 3 interim analysis (p≤0.005), which was set to preserve most of the alpha for the primary endpoint of PFS. Regardless of the outcome of the interim analysis, the trial is designed to continue to the primary PFS endpoint.

About IOB-022/KN-D38 Phase 2 Solid Tumor Basket Trial

IOB-022/KN-D38 (NCT05077709) is a non-comparative, open label trial to investigate the safety and efficacy of IO102-IO103 in combination with pembrolizumab in first-line advanced cancers in non-small cell lung cancer (NSCLC) and squamous cell carcinoma of the head and neck (SCCHN). IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab. IO Biotech maintains global commercial rights to IO102-IO103.

About IOB-032/PN-E40 Phase 2 Solid Tumor Basket Trial

IOB-032/PN-E40 (NCT05280314) is a Phase 2 basket trial investigating the IO102-IO103 therapeutic cancer vaccine in combination with pembrolizumab as neo-adjuvant/adjuvant treatment of patients with solid tumors. The study will enroll approximately 15 patients with melanoma and 15 patients with SCCHN in cohort A and cohort B respectively as single arm cohorts receiving combination of IO102-IO103 with pembrolizumab, whereas in cohort C ≥30 melanoma patients will be randomized 1:1 to neo-adjuvant treatment with either the combination of IO102-IO103 with pembrolizumab or pembrolizumab alone. In the neo-adjuvant period, for all cohorts, treatment is every 3 weeks (Q3W) for 3 cycles (melanoma) or 2-3 cycles (SCCHN). Patients entering the study will be scheduled for surgery and begin neoadjuvant treatment 4-9 weeks prior. Surgery will be followed by adjuvant treatment with the same regimen for 15 cycles. Cohort C patients with poor pathological response to pembrolizumab alone in the neo-adjuvant phase (>10% residual viable tumor) may cross over to combination treatment post-surgery. The primary endpoint is major pathological response at surgery (≤10% residual viable tumor; central assessment). IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab.