On August 12, 2025 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells for the treatment of autoimmune disease and cancer, reported a business and financial update for the second quarter 2025 (Press release, Cue Biopharma, AUG 12, 2025, View Source [SID1234655132]).

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"We made significant progress during the second quarter with highly encouraging clinical data from our ongoing Phase 1b clinical trial, supporting our belief that CUE-101, representative of the CUE-100 series, has the potential to establish a new standard of care for HPV+ HNSCC patients," said Daniel Passeri, chief executive officer of Cue Biopharma. "These maturing data, together with the continued advancements of our lead autoimmune programs, reinforces our commitment to provide patients with more effective and well tolerated therapies to treat serious disease."

Business Highlights

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Received FDA feedback on Pre-IND Briefing Document reinforcing Company’s intention to advance IND submission for CUE-401 to address unmet need in the treatment of autoimmune disease.
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Announced strategic research collaboration and license agreement with Boehringer Ingelheim to develop and commercialize CUE-501, a differentiated B cell depletion therapy for autoimmune and inflammatory diseases.
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Upfront payment of $12 million and ~$345 million in potential milestone payments
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Raised approximately $20 million through a public offering.
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Hosted virtual investor event, Mobilizing the Immune System: Cue Biopharma’s Novel Biologics Portfolio, featuring key opinion leaders, Richard DiPaolo, PhD, and Andrew Cope, MD PhD.

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Poster presentation delivered by Dana-Farber Cancer Institute at the American Society of Gene & Cell Therapy Annual Meeting presented proof-of-concept data on CUE-101 and its potential to control CAR-T cell expansion and persistence in vivo.
Reported new complete response and confirmed 50% overall response rate (ORR) in ongoing Phase 1 trial of CUE-101 and pembrolizumab (KEYTRUDA) in recurrent/metastatic HPV+ head and neck cancer. Data observed included 12-month overall survival of 88% and median overall survival (mOS) of 32 months.

Second Quarter 2025 Financial Results
The Company reported collaboration revenue of $3.0 million and $2.7 million for the three months ended June 30, 2025 and 2024, respectively. The increase was due to the timing of revenue earned from the collaboration and license agreement with Boehringer Ingelheim International GmbH (BI).

Research and development expenses were $7.9 million and $9.5 million for the three months ended June 30, 2025 and 2024, respectively. The decrease was primarily due to decreases in clinical trials costs and employee compensation.

General and administrative expenses were $3.7 million and $3.5 million for the three months ended June 30, 2025 and 2024, respectively. The increase was primarily due to an increase in professional fees.

On August 12, 2025 Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies, reported financial results for the second quarter and year ended June 30, 2025 (Press release, Nkarta, AUG 12, 2025, View Source [SID1234655159]).

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"We remain focused on the execution of our clinical trials and continue to believe in the differentiated potential of NK cell therapy to address unmet needs in the treatment of autoimmune diseases," said Paul J. Hastings, CEO of Nkarta. "Welcoming Dr. Rose and other key new members of our clinical team with rheumatology experience has provided us with world-class medical expertise to inform our ongoing enrollment of patients across our clinical studies and investigator-sponsored trials. Dr. Rose’s track record as a seasoned rheumatologist, immunologist and accomplished drug developer in the autoimmune space will be invaluable as we continue to advance our NKX019 clinical trial programs."

NKX019 Clinical Program Progress and Upcoming Milestones

NKX019 clinical programs in autoimmune diseases continue to enroll patients. This includes Ntrust-1, Ntrust-2 and two investigator-sponsored trials.
Preliminary data from the Ntrust-1 and Ntrust-2 clinical trials is planned for release in the second half of 2025.
Other Corporate Updates

Shawn Rose, M.D. Ph.D, was appointed Chief Medical Officer and Head of Research and Development in June 2025. In previous roles with Johnson & Johnson, BMS and emerging biopharma companies, Dr. Rose brought forward more than a dozen programs from discovery into clinical development, and he has developed multiple pioneering approved medicines for autoimmune conditions.
Second Quarter 2025 and Recent Financial Highlights

Nkarta had cash, cash equivalents, restricted cash, and investments in marketable securities of $334.0 million as of June 30, 2025.
Research and development (R&D) expenses were $20.8 million for the second quarter of 2025. Non-cash stock-based compensation expense included in R&D expense was $0.9 million for the second quarter of 2025.
General and administrative (G&A) expenses were $6.4 million for the second quarter of 2025. Non-cash stock-based compensation expense included in G&A expense was $1.2 million for the second quarter of 2025.
Net loss was $23.0 million, or $0.31 per basic and diluted share, for the second quarter of 2025. This net loss includes non-cash charges of $3.4 million that consisted primarily of share-based compensation and depreciation expenses.
Financial Guidance

Nkarta expects its current cash and cash equivalents will be sufficient to fund its current operating plan into 2029.
About the Ntrust℠ Clinical Trials in Autoimmune Disease
Ntrust-1 (NCT06557265) and Ntrust-2 (NCT06733935) are multi-center, open label, dose escalation clinical trials that build on academic studies of durable, drug-free remissions in patients with autoimmune disease after CD19-targeted cell therapy. Both trials will assess the safety of NKX019 in people living with autoimmune diseases as well as its ability to enable long-term remissions via a "reset" of the immune system through the elimination of pathogenic B cells.

Ntrust-1 is initially enrolling up to 24 patients with lupus nephritis or primary membranous nephropathy. Ntrust-2 is initially enrolling up to 36 patients with systemic sclerosis, idiopathic inflammatory myopathy, or ANCA-associated vasculitis.

In both studies, patients receive a three-dose cycle of NKX019 on Days 0, 3, and 7 following lymphodepleting conditioning with either fludarabine and cyclophosphamide or cyclophosphamide alone. Leveraging the engineering of NKX019, no patients in either trial will receive supplemental cytokines or antibody-based therapeutics. This approach is designed to evaluate the single-agent activity of NKX019 and facilitate a more rapid path to regulatory approval. Patients in Ntrust-1 may also receive additional cycles, if necessary, to restore response.

About the Investigator-Sponsored Clinical Trial of NKX019 for Generalized Myasthenia Gravis
The single-arm, open-label Phase 1 investigator-sponsored clinical trial is designed to enroll patients with generalized myasthenia gravis and will evaluate safety and clinical outcomes. Translational and biomarker studies, including autoantibodies, cytokine profiles and pharmacokinetics are planned. Patients receive 3 doses of NKX019 following lymphodepletion. The clinical trial is being co-led by Ali A. Habib, M.D., Clinical Professor of Neurology at the University of California, Irvine, and other investigators.

About the Investigator-Sponsored Clinical Trial of NKX019 for Systemic Lupus Erythematosus
The single-center, single-arm, open-label Phase 1 investigator-sponsored clinical trial (NCT06518668) is designed to enroll up to 6 patients with systemic lupus erythematosus, regardless of renal involvement, and will evaluate safety and clinical outcomes in a potentially different population than Ntrust-1. Translational and biomarker studies, including autoantibodies, cytokine profiles and pharmacokinetics are planned. Patients receive 3 doses of NKX019 following lymphodepletion. The clinical trial is being led by Anca D. Askanase, M.D., M.P.H., Director, Lupus Center at Columbia University Irving Medical Center and the Director of Rheumatology Clinical Trials.

About NKX019
NKX019 is an allogeneic, cryopreserved, off-the-shelf immunotherapy candidate that uses natural killer (NK) cells derived from the peripheral blood of healthy adult donors. It is engineered with a humanized CD19-directed chimeric antigen receptor (CAR) for enhanced cell targeting and a proprietary, membrane-bound form of interleukin-15 (IL-15) for greater persistence and activity without exogenous cytokine support. CD19 is a biomarker for normal B cells as well as those implicated in autoimmune disease. Nkarta is evaluating NKX019 in multiple autoimmune conditions.

On August 11, 2025 FoRx Therapeutics, a clinical-stage biotechnology company developing precision anti-cancer therapeutics, reported the dosing of the first patient in a first-in-human clinical study of FORX-428, a novel PARG inhibitor designed to target and disrupt the DNA Damage Response (DDR) in advanced solid tumors (Press release, FoRx Therapeutics, AUG 11, 2025, View Source [SID1234655067]).

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The discovery that distinct genetic subsets of cancer are exceptionally vulnerable to drugs that interfere with the DDR led to the approval of PARP inhibitors more than 10 years ago, transforming cancer treatment. By pursuing a next-generation DDR target, called PARG, FoRx is seeking to further advance this strategy. PARG inhibition holds tremendous promise as a treatment option for patients whose cancers do not or no longer respond to PARP inhibitors.

Tarig Bashir, CEO of FoRx Therapeutics, said: "FoRx is built on the disruptive potential of PARG inhibition as a therapeutic strategy. FORX-428 has demonstrated exquisite anti-tumor efficacy in multiple preclinical in vivo tumor models, suggesting best-in-class potential. The entry of FORX-428 into clinical development is a major milestone in our mission to redefine cancer therapy by offering better treatment options for patients."

An initial data readout from the trial is expected by mid-2026. The open-label study, initially taking place in the United States, is evaluating safety, tolerability, pharmacokinetics, and preliminary efficacy in patients with advanced solid tumors who have exhausted standard-of-care options.

Manish R. Sharma, MD, Co-Director of Clinical Research at START Midwest and Principal Investigator on the trial said: "We are excited to have dosed the first patient with cancer in collaboration with FoRx Therapeutics. There is an unmet need to develop new therapies for advanced cancer patients with distinct DNA damage repair deficiencies or high replication stress. The PARG inhibitor, FORX-428, has a novel mechanism of action, and preclinical studies have shown it had impressive activity in cancers resistant to chemotherapy and PARP inhibitors."

FORX-428 received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) on June 13, the first patient first visit (FPFV) was on July 22 and the first patient was dosed on August 6.

Jens Wuerthner, MD, PhD, Chief Medical Officer of FoRx Therapeutics, said: "The efficient pace from IND clearance to dosing the first patient is a testament to the dedication and coordination of our clinical, regulatory, operational, and research teams, including the team at START Midwest. We are thrilled to have begun investigating FORX-428 in patients with advanced cancer and believe this compound could be a significant advancement in solid tumor therapy."

FORX-428 is a proprietary, orally available small molecule drug designed to inhibit poly (ADP-ribose) glycohydrolase (PARG) to cause tumor cell death. PARG is a key DNA repair enzyme necessary for the survival of certain genetically defined cancers, harboring specific DDR deficiencies or high replication stress. Preclinical studies demonstrated FORX-428 had robust anti-tumor activity across multiple solid tumor types underscoring the novel compound’s outstanding potential in both monotherapy and combination settings. Importantly, FORX-428 was well tolerated, demonstrating drug-like pharmacology and a favorable safety profile.

On August 11, 2025 Tahoe Therapeutics reported $30 million in new funding to build the definitive foundational dataset for training Virtual Cell Models (Press release, Tahoe Therapeutics, AUG 11, 2025, View Source [SID1234655088]). With this, the team will generate one billion single-cell datapoints, mapping one million drug-patient interactions, a scale previously impossible. The dataset will support the discovery of new precision medicines for cancer and beyond. Tahoe will also select a single partner to share the data and accelerate translation to clinical outcomes.

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The round was led by Amplify Partners, joined by a distinguished group of investors including Databricks Ventures, Wing Venture Capital, General Catalyst, Civilization Ventures, Conviction, Mubadala Capital Ventures, and AIX Ventures.

The raise follows the release of Tahoe‑100M, the world’s first gigascale perturbative single-cell dataset, which has become foundational for teams building virtual cell models, ranging from major AI labs to focused research institutions. Open-sourced just a few months ago, Tahoe-100M has been downloaded nearly 100,000 times. The dataset and the models trained on it have already led to the discovery of promising new therapeutic candidates for major cancer subtypes as well as novel targets across multiple modalities.

Tahoe is now expanding on that foundation: the company plans to generate one billion single-cell datapoints, mapping how tens of thousands of drug molecules interact with human biology. This new dataset will expand the boundaries of biological foundation models, aiming to reduce clinical trial failure rates and accelerate the development of precision medicines.

"Building Tahoe-100M required us to invent new ways to generate single-cell data," said Nima Alidoust, co-founder and CEO of Tahoe Therapeutics. "Now, we’re applying that superpower to go 10x further. This next phase is about using these massive datasets to bring about the GPT moment for AI models of human cells, translating insights to clinical readouts, and developing new medicines with much lower clinical failure rates."

With the new capital, Tahoe is advancing its own therapeutic programs toward the clinic, while also launching a new model of strategic collaboration. The company will select a single partner, a pharmaceutical or AI company with complementary strengths, to access the forthcoming dataset. Together, the goal is to develop the first medicines powered by virtual cell models, combining Tahoe’s data with the partner’s clinical or modeling expertise.

"While structural models have accelerated molecular design, they rarely translate to clinical success — a problem that remains one of the biggest challenges in drug development," said Sunil Dhaliwal, General Partner at Amplify Partners. "Tahoe Therapeutics is uniquely positioned to move the industry past this bottleneck by generating massive drug-patient datasets and training high-dimensional, cell-based AI models. We’re proud to back this exceptional team as they combine biology and computation to accelerate clinical impact."

Tahoe founders, Nima Alidoust, Johnny Yu, Hani Goodzari, and Kevan Shokat hold deep experience in single-cell genomics, ML, and drug discovery. The company’s platform makes large-scale, single-cell drug screening across diverse patient contexts not only possible, but scalable. Built on scientific breakthroughs at UCSF, Tahoe is creating the raw materials needed to train disease-relevant foundation models of human cells and chart a new course for precision medicine.

On August 11, 2025 GT Biopharma, Inc. (the "Company") (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company’s proprietary TriKE natural killer (NK) cell engager platform, reported initiation of dosing in Cohort 3 of its Phase 1 dose escalation trial evaluating GTB-3650 for the treatment of relapsed or refractory (r/r) CD33 expressing hematologic malignancies (Press release, GT Biopharma, AUG 11, 2025, View Source [SID1234655068]).

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The Phase 1 dose escalation trial is evaluating GTB-3650, GT Biopharma’s second-generation TriKE, for the treatment of relapsed or refractory (r/r) CD33 expressing hematologic malignancies. Cohorts 1 and 2 have now both been successfully completed and following the formal safety reviews, no safety or tolerability issues have been observed. This has allowed initiation of dosing in Cohort 3, with the first patient now having completed the first week of cycle 1.

Patients from Cohort 1 and Cohort 2 have shown encouraging early results indicative of GTB-3650’s ability to activate endogenous NK cells and induce NK cell expansion. Data from multiple blood biomarker assays from the first four patients show heightened immune activity. GT Biopharma plans on releasing initial Phase 1 results later in 2025 following completion of additional dose cohorts.

The trial plans to evaluate GTB-3650 in up to approximately 14 patients (seven cohorts) and GTB-3650 will be dosed in two-week blocks, two weeks on and two weeks off (defining a treatment cycle), for up to four months based on clinical benefit. The trial will assess safety, pharmacokinetics, pharmacodynamics, in vivo expansion of endogenous patient NK cells and clinical activity. More details can be found on clinicaltrials.gov with the identifier: NCT06594445.