On May 7, 2025 Merus N.V. (Nasdaq: MRUS) (Merus, the Company, we, or our), an oncology company developing innovative, full-length multispecific antibodies and antibody drug conjugates (Biclonics, Triclonics and ADClonics), reported financial results for the first quarter and provided a business update (Press release, Merus, MAY 7, 2025, View Source [SID1234652662]).

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"We are very much looking forward to sharing the robust updated interim phase 2 data, on the entire 45 patient data set at 2025 ASCO (Free ASCO Whitepaper). We believe based on these interim data that petosemtamab continues to demonstrate substantial clinical activity superior to historical controls based on the magnitude and consistency of efficacy across ORR, PFS and OS in the overall population and within important subgroups of HPV disease and PD-L1 expression levels," said Bill Lundberg, M.D., President, Chief Executive Officer of Merus. "Additionally, I’m thrilled by the team’s execution in our two phase 3 trials and expect both trials to be substantially enrolled by year end 2025."

Petosemtamab (MCLA-158: EGFR x LGR5 Biclonics): Solid Tumors
LiGeR-HN1 phase 3 trial in 1L recurrent/metastatic (r/m) head and neck squamous cell carcinoma (HNSCC) and LiGeR-HN2 phase 3 trial in 2/3L r/m HNSCC enrolling – with both trials expected to be substantially enrolled by YE25; clinical update on phase 2 trial in combination with pembrolizumab in 1L PD-L1+ HNSCC at 2025 ASCO (Free ASCO Whitepaper); phase 2 trial in 1L, 2L and 3L+ metastatic colorectal cancer (mCRC) enrolling; mCRC initial clinical data planned for 2H25

An updated analysis of the interim clinical data from the phase 2 trial of petosemtamab with pembrolizumab as 1L treatment of PD-L1+ r/m HNSCC will be presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO) (Free ASCO Whitepaper) as detailed in our press release, Merus Announces Abstract Accepted for Presentation at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting. The presentation will include data on the entire 45 patient dataset and follows the early clinical efficacy and encouraging safety data previously presented at 2024 ASCO (Free ASCO Whitepaper), which was detailed in our 2024 press release, Merus’ Petosemtamab in Combination with Pembrolizumab Interim Data Demonstrates Robust Response Rate and Favorable Safety Profile in 1L r/m HNSCC (May 28, 2024).

Merus will hold a conference call and webcast for investors on Thursday, May 22, 2025 at 5:30 p.m. ET. A replay will be available after the completion of the call in the Investors and Media section of our website for a limited time.

Date & Time: May 22, 2025 at 5:30 p.m. ET
Webcast link: Available on our website
Dial-in: Toll Free: (800) 715-9871 / International: (646) 307-1963
Conference ID: 7517301 or Merus NV call

In February 2025, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation (BTD) to petosemtamab in combination with pembrolizumab for the first-line treatment of adult patients with recurrent or metastatic programmed death-ligand 1 (PD-L1) positive HNSCC with combined positive score (CPS) ≥ 1. This designation was detailed in our press release, Petosemtamab granted Breakthrough Therapy designation by the U.S. FDA for 1L PD-L1 positive head and neck squamous cell carcinoma (February 18, 2025). BTD was also granted for petosemtamab monotherapy for the treatment of patients with recurrent or metastatic HNSCC whose disease has progressed following treatment with platinum based chemotherapy and an anti-programmed cell death receptor-1 (PD-1) or anti-programmed death ligand 1 (PD-L1) antibody, detailed in our press release, Petosemtamab granted Breakthrough Therapy Designation by the U.S. FDA (May 13, 2024).

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

LiGeR-HN1, a phase 3 trial evaluating the efficacy and safety of petosemtamab in combination with pembrolizumab in 1L PD-L1+ (CPS≥1) r/m HNSCC compared to pembrolizumab, and LiGeR-HN2, a phase 3 trial evaluating the efficacy and safety of petosemtamab in 2/3L HNSCC compared to standard of care, are enrolling and we expect both trials to be substantially enrolled by YE25. Further, the Company manufactures both drug substance and drug product in the United States and Europe, with a plan to focus on potential commercial manufacturing in the United States.

Merus believes a randomized registration trial in HNSCC with an overall response rate endpoint could potentially support accelerated approval and the overall survival results from the same study could potentially verify its clinical benefit to support regular approval for the Company’s phase 3 trial in 1L, and in phase 3 trial in 2/3L HNSCC.

A phase 2 trial evaluating petosemtamab in combination with standard chemotherapy in 1L and 2L mCRC, and as monotherapy in heavily pretreated (3L+) mCRC, is enrolling. We expect to provide initial clinical data for petosemtamab in mCRC in 2H25.

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

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

MCLA-129 (EGFR x c-MET Biclonics): Solid Tumors
Investigation of MCLA-129 is ongoing in METex14 NSCLC; phase 2 trial in combination with chemotherapy in 2L+ EGFR mutant (EGFRm) NSCLC enrolling

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

Collaborations

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

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

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

Ono Pharmaceutical
In 2018, the Company granted Ono Pharmaceutical Co., Ltd. (Ono) an exclusive, worldwide, royalty-bearing license, with the right to sublicense, research, test, make, use and market a limited number of bispecific antibody candidates based on Merus’ Biclonics technology platform directed to an undisclosed target combination.

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

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

As of March 31, 2025, Merus had $638 million cash, cash equivalents and marketable securities. Based on the Company’s current operating plan, the existing cash, cash equivalents and marketable securities are expected to fund Merus’ operations into 2028.

First Quarter 2025 Financial Results
Collaboration revenue for the three months ended March 31, 2025 increased by $18.6 million as compared to the three months ended March 31, 2024, primarily as a result of commercial material revenue this quarter and higher deferred revenue amortization.

Research and development expense for the three months ended March 31, 2025 increased by $41.5 million as compared to the three months ended March 31, 2024. The increase is primarily driven by an increase of $35.6 million in clinical trial support provided by contract manufacturing and development organizations and contract research organizations, most of which is related to the petosemtamab clinical trials.

General and administrative expense for the three months ended March 31, 2025 increased by $6.0 million as compared to the three months ended March 31, 2024, primarily as a result of increases in personnel related expenses including share-based compensation of $5.3 million; increases in consulting expenses, facilities and depreciation expense also contributed.

Other income (loss), net consists of interest earned and fees paid on our cash and cash equivalents held on account, accretion of investment earnings and net foreign exchange (losses) gains on our foreign denominated cash, cash equivalents and marketable securities. Other gains or losses relate to the issuance and settlement of financial instruments.

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

March 31,
2025 December 31,
2024
ASSETS
Current assets:
Cash and cash equivalents $ 197,199 $ 293,294
Marketable securities 261,126 243,733
Accounts receivable 14,203 1,261
Prepaid expenses and other current assets 49,744 30,784
Total current assets 522,272 569,072
Marketable securities 179,886 187,008
Property and equipment, net 10,937 10,770
Operating lease right-of-use assets 9,199 9,254
Intangible assets, net 1,703 1,679
Equity Investment 3,449 —
Deferred tax assets 364 1,520
Other assets 3,112 3,390
Total assets $ 730,922 $ 782,693
LIABILITIES AND SHAREHOLDERS’ EQUITY
Current liabilities:
Accounts payable $ 8,984 $ 4,164
Accrued expenses and other liabilities 42,799 43,957
Income taxes payable 8,015 7,317
Current portion of lease obligation 1,772 1,704
Current portion of deferred revenue 27,560 29,934
Total current liabilities 89,130 87,076
Lease obligation 8,084 8,208
Deferred revenue, net of current portion 37,589 39,482
Total liabilities 134,803 134,766
Commitments and contingencies – Note 6
Shareholders’ equity:
Common shares, €0.09 par value; 105,000,000 shares authorized at March 31, 2025 and December 31, 2024; 69,175,766 and 68,828,749 shares issued and outstanding as at March 31, 2025 and December 31, 2024, respectively 6,990 6,957
Additional paid-in capital 1,686,350 1,664,822
Accumulated other comprehensive income (32,360 ) (55,465 )
Accumulated deficit (1,064,861 ) (968,387 )
Total shareholders’ equity 596,119 647,927
Total liabilities and shareholders’ equity $ 730,922 $ 782,693

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

Three Months Ended
March 31,
2025 2024
Commercial material revenue $ 13,331 $ —
Collaboration revenue 13,148 7,889
Royalty revenue 9 —
Total revenue 26,488 7,889
Operating expenses:
Research and development 80,116 38,584
General and administrative 22,112 16,114
Total operating expenses 102,228 54,698
Operating loss (75,740 ) (46,809 )
Other income, net:
Interest income, net 7,203 4,917
Foreign exchange gains (loss) (24,316 ) 8,534
Other expense (1,766 ) —
Total other income (loss), net (18,879 ) 13,451

Net loss before income taxes (94,619 ) (33,358 )
Income tax expense 1,855 1,098
Net loss $ (96,474 ) $ (34,456 )
Other comprehensive loss:
Currency translation adjustment 23,105 (7,388 )
Comprehensive loss $ (73,369 ) $ (41,844 )
Net loss per share attributable to common shareholders:
Basic and diluted $ (1.40 ) $ (0.59 )
Weighted-average common shares outstanding:
Basic and diluted 69,017,576 58,085,416

On May 7, 2025 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported that it is scheduled to participate in the following upcoming investor events (Press release, Arrowhead Pharmaceuticals, MAY 7, 2025, View Source [SID1234652661]):

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BofA Securities 2025 Health Care Conference – May 13-15, 2025

Type: Fireside Chat Presentation
Date/Time: May 14, 2025, 11:20 a.m. PDT

2025 RBC Capital Markets Global Healthcare – May 20-21, 2025

Type: Fireside Chat Presentation
Date/Time: May 20, 2025, 2:35 p.m. EDT

Presentation webcasts may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

On May 7, 2025 Theriva Biologics (NYSE American: TOVX), ("Theriva" or the "Company"), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported positive topline outcomes from the VIRAGE Phase 2b clinical trial evaluating the Company’s lead product candidate VCN-01 (zabilugene almadenorepvec) plus standard-of-care (SoC) chemotherapy gemcitabine/nab-paclitaxel as a first line therapy for patients with metastatic pancreatic ductal adenocarcinoma (PDAC) for whom gemcitabine/nab-paclitaxel is the recommended first-line treatment option (Press release, Theriva Biologics, MAY 7, 2025, View Source [SID1234652660]). VCN-01 is a systemically-administered, tumor selective, stroma-degrading oncolytic adenovirus that has been granted Orphan Drug Designation and Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of pancreatic cancer.

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The analysis of the VIRAGE trial (see "About VIRAGE" below) includes data for first-line treatment of 96 newly-diagnosed metastatic PDAC patients:

In the primary endpoint analysis, the 48 patients treated with at least one dose of gemcitabine/nab-paclitaxel SoC had a median overall survival (OS) of 8.6 months, while the 48 patients treated with VCN-01 followed by at least one dose of gemcitabine/nab-paclitaxel SoC had a median OS of 10.8 months [Hazard Ratio (HR) = 0.57, 95% CI 0.34-0.96, p=0.0546].
The improvements in OS in the VCN-01+SoC treatment arm compared to the SoC control arm were reflected in increased progression free survival (PFS) [median PFS 7.0 vs 4.6 months; HR = 0.55, 95% CI 0.34-0.88, p= 0.0105].
The median duration of response (DoR) was 5.4 months (n=15) in the SoC control arm, while the median DoR in the VCN-01+SoC treatment arm was doubled to 11.2 months (n=19, HR = 0.22, 95% CI 0.08-0.62, p=0.0035).
The increase in OS was greater for patients who received 2 doses of VCN-01 and 4 or more cycles of gemcitabine/nab-paclitaxel SoC (n=34) compared with patients who received 4 or more cycles of gemcitabine/nab-paclitaxel SoC (n=29) [median OS 14.8 and 11.6 months respectively; HR=0.44, 95% CI: 0.21-0.92, p=0.046], suggesting that the second dose of VCN-01 (administered 3 months after the first dose) provides a meaningful additional benefit in this treatment subgroup.

"The exciting topline data from the VIRAGE Phase 2b trial demonstrate the potential opportunity for VCN-01 to benefit metastatic PDAC patients treated with gemcitabine/nab-paclitaxel SoC chemotherapy." said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "The significantly reduced hazard ratios for survival parameters in the VCN-01 treatment group provide compelling evidence that VCN-01 in combination with gemcitabine/nab-paclitaxel may extend the lives of metastatic PDAC patients. These data, combined with recent advice from the U.S. FDA and the European Medical Agency, are expected to facilitate engagement with industry partners and enable the design of a Phase 3 confirmatory trial that, if successful, may deliver an important new therapeutic option for patients suffering this rapidly fatal disease."

As previously reported, the VCN-01 adverse event (AE) profile was consistent with that observed in prior clinical trials. The most common VCN-01 related AEs (pyrexia, flu-like illness, vomiting, nausea, and elevated transaminases) were transient and reversible. These AEs were observed to be less frequent and of reduced CTCAE grade after the second VCN-01 dose (administered on day ~92) compared to the first VCN-01 dose (administered on day 1). A review by an Independent Data Monitoring Committee noted that the overall type and number of AEs in the VCN-01 treatment group was as expected for the pancreatic cancer population, the duration of treatment, and the administration of an oncolytic virus.

Theriva will host a live virtual event to review and discuss the data from the VIRAGE trial of VCN-01 on Wednesday, May 7th 2025, 8:00 AM EDT. The virtual event will feature eminent pancreatic cancer clinician/researchers Dr. Manuel Hidalgo Medina MD PhD (Chief, Division of Hematology and Medical Oncology, Weill Cornell Medical College and Attending Physician, New York-Presbyterian Hospital) and Dr. Mike Pishvaian MD PhD (Director of Gastrointestinal, Developmental Therapeutics and Clinical Research Programs for the Johns Hopkins Kimmel Cancer Center and Associate Professor at the School of Medicine). To register for the event, click HERE.

About Pancreatic Ductal Adenocarcinoma

Cancer of the pancreas consists of two main histological types: cancer that arises from the ductal (exocrine) cells of the pancreas or, much less often, cancers may arise from the endocrine compartment of the pancreas. Pancreatic ductal adenocarcinoma ("PDAC") accounts for more than 90% of all pancreatic tumors. It can be located either in the head of the pancreas or in the body/tail. Pancreatic cancer usually metastasizes to the liver and peritoneum. Other less common metastatic sites are the lungs, brain, kidney and bone. In its early stages, pancreatic cancer does not typically result in any characteristic symptoms, so in most cases it is diagnosed in its late stages (locally advanced non-metastatic or metastatic disease) when surgical resection and possibly curative treatment is not possible. It is generally assumed that only 10% of cases are resectable at presentation, whereas 30-40% of patients are diagnosed at local advanced/unresectable stage and 50-60% present with distant metastases.

About VIRAGE

VIRAGE is a two-arm, Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. Patients were enrolled at 5 sites in the U.S. and 9 sites in Spain. In both the control and VCN-01 (zabilugene almadenorepvec) treatment arms, patients received gemcitabine/nab-paclitaxel standard-of-care chemotherapy in repeated 28-day cycles until disease progression. In the VCN-01 treatment arm only, patients were also administered intravenous VCN-01 seven-days prior to starting the first and fourth cycles of gemcitabine/nab-paclitaxel treatment (study days 1 and ~92 respectively). Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, duration of response, and measures of VCN-01 biodistribution, replication, and immune response. The study was designed with 80% power to detect a HR of 0.65 for overall survival with a 1-sided alpha of 0.05 (2-sided alpha 0.1). Reported p-values are for 2-sided statistical analysis using the log rank test. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).

About VCN-01

VCN-01 (zabilugene almadenorepvec) is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 140 patients to date in clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.

On May 7, 2025 UroGen (NASDAQ: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that the U.S. Food and Drug Administration (FDA) has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting on May 21, 2025 to review the new drug application (NDA) for UGN-102 (mitomycin) for intravesical solution, an investigational treatment for recurrent LG-IR-NMIBC (Press release, UroGen Pharma, MAY 7, 2025, View Source [SID1234652658]).

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The ODAC meeting will provide an opportunity for independent clinicians and other experts to evaluate the UGN-102 data and make a recommendation to the FDA as to whether the NDA should be approved and under what conditions. The FDA carefully considers the advice of the Advisory Committee, but is not bound by its recommendations. The FDA has stated in its correspondence to the Company that they intend to complete their review in time to meet the Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025.

"We are excited to discuss our data with the Advisory Committee and broader medical community as we continue our mission to bring innovative solutions to patients suffering from bladder cancer," said Liz Barrett, President and Chief Executive Officer of UroGen. "We believe UGN-102 represents a meaningful advancement for patients facing the recurrent and challenging nature of LG-IR-NMIBC, and we look forward to the opportunity to discuss its potential."

UGN-102 is in development as a therapeutic option for patients with recurrent LG-IR-NMIBC, a condition for which there are no FDA-approved drugs. The NDA is supported by results from the pivotal Phase 3 ENVISION trial which demonstrated a 79.6% complete response (CR) rate at 3 months after first instillation of UGN-102, and duration of response of 82.5% at 12 months after 3-month CR by Kaplan-Meier analysis.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102, scheduled an ODAC meeting for May 21, 2025, and assigned a PDUFA target action date of June 13, 2025.

About Low-Grade Non-Muscle Invasive Bladder Cancer (NMIBC)

In the U.S., bladder cancer is the second most common urologic cancer in men and primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. More specifically, LG-IR-NMIBC represents approximately 23,000 newly diagnosed bladder cancer patients each year and an estimated 59,000 recurrences annually among patients diagnosed in previous years. Guideline recommendations for the management of NMIBC include trans-urethral resection of bladder tumor (TURBT) as the standard of care. Up to 70% of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures.

On May 7, 2025 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies, reported financial results and business highlights for the first quarter ended March 31, 2025 and recent corporate highlights (Press release, In8bio, MAY 7, 2025, View Source [SID1234652657]).

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"IN8bio stands at the forefront of gamma-delta T cell innovation, pioneering transformative therapies for cancer patients with urgent unmet needs. Driven by our commitment to scientific excellence, we are advancing our mission of Cancer ZeroTM with elegant, cutting-edge approaches. Our clinical data showcases robust activity, delivering extended progression-free survival and a unique safety profile across challenging cancers to date. As the biopharmaceutical industry excitedly embraces novel T cell engagers, IN8bio is once again redefining the landscape with our innovative INB-600 TCE platform. The preclinical data unveiled at AACR (Free AACR Whitepaper) represents a leap forward," said William Ho, CEO & co-founder, IN8bio. "Harnessing our deep expertise, we’ve engineered a breakthrough technology that achieves picomolar potency, enhances immune surveillance, and potentially mitigates some of the safety risks associated with conventional CD3-targeted TCEs. We’re excited to advance this platform into both oncology and autoimmune indications and continue building a differentiated pipeline that promises to reshape the future of medicine."

Corporate Highlights and Recent Developments
Expanded Pipeline with INB-600 Gamma-Delta T Cell Engager Platform

Presented new preclinical data at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting highlighting the INB-600 platform, including lead candidates INB-619 (targeting CD19) and INB-633 (targeting CD33).
Preclinical studies demonstrated strong, antigen-specific cytotoxicity against leukemia cells and primary B cells with minimal inflammatory cytokine release, potentially addressing key limitations of conventional CD3-based TCEs.
INB-600 TCE platform significantly expands both Vδ1+ and Vδ2+ subsets to address the reduced cell counts that have limited earlier γδ TCE therapies in cancer patients.
Targeted B cell elimination (INB-619) highlights potential applications in B cell–driven autoimmune diseases as well as oncology indications.
Demonstrated Additional Clinical Progress Across Gamma-Delta T Cell Therapy Pipeline

Presented Positive Phase 1 data (February 2025) for INB-100 (Allogeneic Gamma-Delta T Cell Therapy for High-Risk AML and Leukemias) at the 2025 Transplantation & Cellular Therapy (TCT) Annual Meeting showing durable remissions, with 100% of treated AML patients remaining relapse-free with 20.1 month median follow-up as of January 17, 2025.
Observed a favorable safety profile, with no cases of cytokine release syndrome (CRS) or neurotoxicity (ICANs) reported to date.
1-Year Survival Rates Exceeded Real-World Control Groups: The 1-year progression-free survival (PFS) rate across all leukemia patients is 90.9% and 1-year overall survival (OS) is 100%, outperforming comparative real-world historical control data obtained from both the Center for International Blood & Marrow Transplant Research (CIBMTR) and from the University of Kansas Cancer Center.
Participation at Immuno-Oncology 360° (IO360°) Conference

William Ho, Chief Executive Officer and co-founder of IN8bio, Co-Chaired Day 2 of the Immuno-Oncology 360° (IO360°) Conference on March 25, 2025, delivering opening remarks, leading the "State of the IO Market, Investments and Deals Plenary" session, and presenting on the promise of gamma-delta T cell engagers for oncology and autoimmune diseases, including INB-619.
Upcoming Anticipated Pipeline Milestones and Events

American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 Annual Meeting (May 30, 2025):

Upcoming oral presentation: "INB-200: Phase 1 Study of Gene-Modified Autologous Gamma-Delta (γδ) T Cells in Newly Diagnosed Glioblastoma Multiforme (GBM) Patients Receiving Maintenance Temozolomide (TMZ)" will present updated clinical data from IN8bio’s Phase 1 trial of INB-200. The data will be featured in the Central Nervous System Tumors Oral Abstract Session.

International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting (May 9, 2025):

Upcoming oral presentation: "From Donor to Therapy: How Robust Manufacturing Shapes the TCR Repertoire and Cytotoxic Power of Donor-Derived Allogeneic ex vivo Expanded and Activated γδ T Cell Products" will highlight the power of the company’s gamma-delta T cell manufacturing platform. The data will demonstrate the TCR diversity, genomic profiling and cytotoxic function of donor-derived clinical gamma-delta T cell therapies. Mariska ter Haak, Senior Director, Analytical Development at IN8bio, will present during the Oral Abstract Session – Immunotherapy.

Poster Presentation: "Selection and Implementation of the Electronic Quality Management System for High Complexity Clinical Stage Cellular Therapy Company." This will be presented by Guoling Chen, Senior Director, Quality Operations.

American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2025 Annual Meeting (May 17, 2025):

Upcoming oral presentation: "Decoding the Molecular Signature of Expanded Gamma Delta T Cell Products; TCR and Immune Gene Expression from Allogeneic Derived Products" will present new data characterizing the TCR repertoire and immune gene expression profiles of IN8bio’s clinical expanded gamma-delta T cell products. This data will show the robustness and reproducibility of the in-house developed manufacturing platform at IN8bio. Mariska ter Haak, Senior Director, Analytical Development, will deliver the presentation at the New Orleans Ernest N. Morial Convention Center.

Poster Presentation: "INB-600: A Novel T Cell Engager Platform Specific for gamma-delta (γδ) T cells" will demonstrate the use of the novel CD19 γδ TCE for driving deep B cell depletion and potential application in autoimmune disease including Lupus. This will be presented by Lawrence Lamb, Ph.D., Co-Founder and Chief Scientific Officer.

First Quarter 2025 Financial Highlights

Research and Development (R&D) expenses: R&D expenses were $3.0 million for the three months ended March 31, 2025, compared with $4.9 million in the prior year. These amounts include non-cash items such as stock-based compensation (SBC) and depreciation. The change was primarily due to decreased but some remaining clinical trial-related activities for the INB-400 program and a decrease in personnel-related costs, in each case as a result of the Company’s pipeline prioritization announced in September 2024.
General and administrative (G&A) expenses: G&A expenses were $2.7 million for the three months ended March 31, 2025, compared with $3.7 million for the comparable prior year period. These amounts include non-cash items such as SBC and depreciation. The change was primarily due to cost savings related to personnel-related costs, director and officer insurance premiums and professional services.
Net loss: The company reported a net loss of $5.6 million, or $0.07 per basic and diluted common share, for the three months ended March 31, 2025, compared with a net loss of $8.6 million, or $0.20 per basic and diluted common share, for the comparable prior year period.
Cash position: As of March 31, 2025, the Company had cash of $11.9 million, compared with $13.0 million for the comparable prior year. Subsequently, in April 2025, the Company received aggregate gross proceeds of $1.9 million from the exercise of its Series A and Series B common stock warrants.