On May 14, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported a new strategic collaboration to advance Boehringer Ingelheim’s growing cancer pipeline (Press release, Tempus, MAY 14, 2025, View Source [SID1234653101]). The new, multi-year collaboration builds on foundational work the two companies have conducted in the last few years, leveraging data and AI to further advance therapeutic research and development.

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Despite significant breakthroughs in the understanding, prevention, and treatment of cancer, the disease remains a major challenge, impacting millions of lives and straining healthcare systems. Boehringer Ingelheim is committed to changing this by advancing the discovery and development of new breakthrough treatments for people living with hard-to-treat cancers. With this new collaboration, Boehringer will have access to Tempus’ de-identified database containing molecular, clinical, and imaging data and its analytical platform, Lens. The aim is to explore data derived from patient cohorts to guide biomarker development and patient stratification, develop drug combination hypotheses, support novel target discovery efforts, and educate on patients’ healthcare journey.

"By combining internal, pre-clinical experimental data, with real-world data from Tempus on its AI-powered platform, we can profoundly deepen our understanding of cancer biology and accelerate our drug discovery and development efforts," said Mark Paul Petronczki, Head of Oncology Research at Boehringer Ingelheim.

"Leveraging real-world patient data is crucial for strengthening our data foundation and harnessing advanced AI methodologies, ultimately accelerating drug development. Integrating these comprehensive datasets with our internal data and techniques not only drives innovation but also enhances the precision and efficiency of pharmaceutical research, paving the way for groundbreaking medical advancements. I am very excited to continue our collaboration with Tempus," stated Jan Nygaard Jensen, Head of Computational Innovation at Boehringer Ingelheim.

"We look forward to expanding our work with the Boehringer team and the opportunity to deploy multiple of our solutions to their growing oncology pipeline," said Ryan Fukushima, Chief Operating Officer at Tempus. "We both are firmly committed to applying the power of data and AI to this important research to bring novel treatments to patients faster."

On May 14, 2025 Oncolytics Biotech reported its first quarter financial results (Filing, 3 mnth, MAR 31, Oncolytics Biotech, 2025, MAY 14, 2025, View Source [SID1234654256]).

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On May 13, 2025 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, reported business highlights and financial results for the first quarter ended March 31, 2025 (Press release, Fate Therapeutics, MAY 13, 2025, View Source [SID1234652965]).

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"We continue to be pleased with the initial clinical profile of our FT819 off-the-shelf CAR T-cell program in patients with moderate-to-severe SLE, and we look forward to sharing new clinical data from our ongoing FT819 Phase 1 study at the EULAR Conference in June," said Bob Valamehr, Ph.D. MBA, President and Chief Executive Officer of Fate Therapeutics. "Our primary focus remains on driving patient enrollment and achieving therapeutic differentiation in SLE, including administration of FT819 with fludarabine-free conditioning and as an add-on to maintenance therapy without conditioning. We plan to work closely with the FDA under our FT819 RMAT designation to align on a registrational pathway for FT819 in SLE, and we have now initiated regulatory submissions in Europe to expand the geographic reach of the program for lupus patients. The expansion of patient population and reach is well supported by the ability to produce FT819 in large quantities from a master cell bank, ensuring consistent on-demand product supply for a large number of patients."

FT819 iPSC-derived 1XX CAR T-cell Program

Phase 1 Study Ongoing using Flu-free Conditioning Regimen for SLE. The Company’s ongoing multi-center, Phase 1 clinical trial of FT819 for moderate-to-severe systemic lupus erythematosus (SLE) (NCT06308978) is designed to evaluate the safety, pharmacokinetics, and efficacy of a single dose of FT819 following a fludarabine (flu)-free conditioning regimen, consisting of either bendamustine alone or cyclophosphamide alone. The Company is currently enrolling patients at two dose levels – a single dose of 360 million cells and a single dose of 900 million cells – with the intent of identifying a recommended dose for later-stage development. In addition, the Company is assessing the safety, pharmacokinetics, and anti-B cell activity of a single dose of FT819 at 360 million cells as an add-on to maintenance therapy without conditioning chemotherapy. The Company plans to present new clinical data from its FT819 Phase 1 study during an oral session at the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress in Barcelona, Spain on June 11.
RMAT Designation Received from the FDA for SLE. In April 2025, the Company was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for FT819 to treat moderate-to-severe SLE. The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases or conditions. The Company’s RMAT application included initial clinical safety and activity data from patients treated with FT819 in its ongoing multi-center, Phase 1 clinical trial. The Company plans to pursue differentiated treatment approaches, including treatment of patients in community centers without hospitalization, and novel registrational strategies with the FDA under its RMAT designation.
Expanded Phase 1 Study to Include Multiple Additional B Cell-mediated Autoimmune Diseases. In December 2024, the Company reached agreement with the FDA to allow for clinical investigation of multiple B cell-mediated autoimmune diseases under its current Phase 1 clinical trial of FT819. The Company has submitted an amended clinical protocol to the FDA that enables the conduct of independent dose-expansion cohorts for anti-neutrophilic cytoplasmic antibody-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). The Company plans to initiate dose-expansion cohorts in each of AAV, IIM, and SSc in 2025.
FT825 / ONO-8250 iPSC-derived CAR T-cell Program

Phase 1 Study Ongoing for Advanced Solid Tumors. Under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono), the Company is conducting a multi-center, Phase 1 study to assess the safety, pharmacokinetics, and activity of FT825 / ONO-8250, a multiplexed-engineered CAR T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2), in patients with advanced solid tumors (NCT06241456). Dose escalation is currently ongoing, with each patient administered conditioning chemotherapy and a single dose of FT825 / ONO-8250 either as monotherapy or in combination with epidermal growth factor receptor (EGFR)-targeted monoclonal antibody therapy. FT825 / ONO-8250 has demonstrated a favorable safety profile with no dose-limiting toxicities (DLTs) to date.
Next-generation iPSC-derived CAR T-cell Programs

FT836 MICA/B-targeted CAR T-cell Program. FT836 is the Company’s multiplexed-engineered CAR T-cell product candidate uniquely targeting major histocompatibility complex (MHC) proteins A (MICA) and B (MICB). The expression of MICA/B cell-surface proteins is induced by cellular stress or malignant transformation and is detectable across many types of cancer cells with limited expression on healthy tissue. At the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting being held in New Orleans on May 13-17, the Company plans to present preclinical data showing that FT836 exerted potent and durable anti-tumor activity in vivo across a broad array of solid tumors. FT836 is the Company’s first product candidate to incorporate its novel Sword & Shield technology that couples its novel Alloimmune Defense Receptor (ADR) technology with the complete knock-out of CD58 (CD58KO), which is uniquely designed to both target and evade host alloreactive immune cells and to reduce or eliminate the need for administration of conditioning chemotherapy to patients receiving cell therapies. In January 2025, the Company secured a $4 million award from the California Institute of Regenerative Medicine (CIRM) to support IND-enabling activities for FT836.
FT839 Dual CAR T-cell Program. FT839 is the Company’s dual CAR T-cell product candidate that incorporates its novel Sword & Shield technology and is designed to express two unique CARs: a first CAR targeting CD19+ B cells, and a second CAR targeting additional disease-causing cells. At the ASGCT (Free ASGCT Whitepaper) Annual Meeting in May, the Company plans to present preclinical data demonstrating iPSC-derived CAR T cells targeting CD19 and the cell-surface glycoprotein CD38 specifically eliminated a variety of malignant cell types, including CD19+ lymphoma and CD38+ multiple myeloma cell lines. In addition, using unmatched peripheral blood mononuclear cells sourced from a patient with SLE, dual CAR T cells showed robust eradication of aberrant CD19+ B cells, CD38+ plasma cells, and CD38+ activated T cells.
FT522 iPSC-derived CAR NK Cell Program

New Phase 1 Translational Data to be Presented at ASGCT (Free ASGCT Whitepaper). FT522 is the Company’s off-the-shelf CAR NK cell product candidate and its first to incorporate Alloimmune Defense Receptor (ADR) technology. The FDA has allowed the Company’s Investigational New Drug (IND) application to assess the safety, pharmacokinetics, and activity of FT522 across a basket of B cell-mediated autoimmune diseases. The Company is currently evaluating opportunities and timelines for the clinical development of FT522 in autoimmunity without administration of conditioning chemotherapy. At the ASGCT (Free ASGCT Whitepaper) Annual Meeting in May, the Company plans to present new clinical and translational data from its multi-center, Phase 1 clinical trial of FT522 in patients with relapsed / refractory B-cell lymphoma (BCL) (NCT05950334), where initial translational data demonstrated the potential of FT522 to persist and function in the presence of an unmatched, fully-intact immune system.
First Quarter 2025 Financial Results

Cash & Investment Position: Cash, cash equivalents, and investments as of March 31, 2025 were $272.7 million.
Total Revenue: Revenue was $1.6 million for the first quarter of 2025, which was derived from the conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under the Company’s collaboration with Ono Pharmaceutical.
Total Operating Expenses: Total operating expenses were $42.9 million for the first quarter of 2025, including research and development expenses of $29.1 million and general and administrative expenses of $13.8 million. Such amount included $7.4 million of non-cash stock-based compensation expense.
Shares Outstanding: As of March 31, 2025, common shares outstanding were 114.6 million, pre-funded warrants outstanding were 3.9 million, and preferred shares outstanding were 2.8 million. Each preferred share is convertible into five common shares.

On May 13, 2025 Monopar Therapeutics Inc. ("Monopar" or the "Company") (Nasdaq: MNPR), a clinical‐stage biopharmaceutical company focused on developing innovative treatments for patients with unmet medical needs, reported first quarter 2025 financial results and recent developments (Press release, Monopar Therapeutics, MAY 13, 2025, View Source [SID1234652983]).

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Recent Developments

ALXN1840 for Wilson Disease

On May 7, 2025, Monopar presented long-term efficacy and safety data for ALXN1840 (tiomolybdate choline) at the European Association for the Study of the Liver ("EASL") International Liver Congress 2025, a leading global conference in liver disease.

The data support ALXN1840 as a potential treatment for Wilson disease, a rare genetic disorder that causes toxic copper buildup in organs like the liver and brain. Pooled results from three clinical trials (n=255) showed sustained clinical benefits over a median treatment duration of 2.63 years. Safety data, which included an additional Phase 2 study (n=266), confirmed a favorable safety profile with fewer than 5% of patients experiencing a drug-related serious adverse event ("SAE") and no renal or urinary system SAEs.

Sustained neurological improvement as assessed by the physician as well as the patient using the Unified Wilson Disease Rating Scale ("UWDRS") was observed, as was sustained increased copper mobilization. Patients reported greater convenience and effectiveness when treated with ALXN1840 compared to standard of care, and improvement in the New Wilson Index (a prognostic indicator of the status of the liver) was also observed.

The Company is preparing to submit a New Drug Application ("NDA") to the U.S. Food and Drug Administration ("FDA") in early 2026.

MNPR‐101 for Radiopharmaceutical Use

The Company’s MNPR-101-Zr Phase 1 (imaging and dosimetry) and MNPR-101-Lu (therapeutic) Phase 1a clinical trials in advanced cancers are active and enrolling in Australia. Monopar continues its preclinical work with MNPR-101-Ac (therapeutic) with plans to enter the clinic in the future.

Financial Results for the First Quarter Ended March 31, 2025, Compared to the First Quarter Ended March 31, 2024

Cash and Net Loss

Cash, cash equivalents and investments as of March 31, 2025, were $54.6 million. Monopar expects that its current funds will be sufficient to continue operations at least through December 31, 2026, in order to: (1) assemble a regulatory package and file an NDA for ALXN1840; (2) continue to conduct and conclude its first-in-human imaging and dosimetry clinical trial with MNPR-101-Zr; (3) continue to conduct its first-in-human therapeutic clinical trial of MNPR-101-Lu; (4) advance its preclinical MNPR-101-Ac program into the clinic; and (5) invest in internal research and development projects to expand its radiopharmaceutical and rare disease pipeline.

Net loss for the first quarter of 2025 was $2.6 million or $0.38 per share compared to net loss of $1.6 million or $0.51 per share for the first quarter of 2024.

Research and Development ("R&D") Expenses

R&D expenses for the first quarter of 2025 were $1,643,000, compared to $966,000 for the first quarter of 2024. This represents an increase of $677,000 attributed to (1) a $611,000 increase in R&D personnel expenses including stock-based compensation and (2) a $69,000 increase in clinical trial site activity related to MNPR-101 for radiopharmaceutical use, partially offset by (3) a net decrease of $3,000 in other R&D expenses.

General and Administrative ("G&A") Expenses

G&A expenses for the first quarter of 2025 were $1,578,000, compared to $757,000 for the first quarter of 2024. This represents an increase of $821,000 primarily attributed to (1) a $416,000 increase in Board compensation resulting from the grant of stock options during the quarter ended March 31, 2025 (no stock options were granted during the quarter ended March 31, 2024), (2) a $291,000 increase in G&A personnel expenses including stock-based compensation, (3) a $73,000 increase in legal fees and (4) a $41,000 increase in insurance expenses.

Interest Income

Interest income for the first quarter of 2025 increased by $515,000, compared to the first quarter of 2024. The increase is attributed to interest earned on U.S. Treasury securities and higher bank balances in 2025, resulting from over $55 million of funds raised in the fourth quarter of 2024.

On May 13, 2025 Gate2Brain, a Barcelona-based biotech innovator in drug delivery across biological barriers, reported that its lead candidate, G2B-002, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) (Press release, Gate2Brain, MAY 13, 2025, View Source [SID1234652966]). This follows the European Medicines Agency’s ODD approval in late 2024 and highlights G2B-002’s potential in treating rare and aggressive pediatric and adult brain tumors.

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G2B-002 is designed to target diffuse intrinsic pontine glioma (DIPG) and pediatric glioblastoma (pGBM), two of the most lethal childhood brain cancers with very limited treatment options. By leveraging Gate2Brain’s proprietary peptide-based delivery technology, G2B-002 enhances drug penetration across the blood–brain barrier, aiming to improve efficacy while reducing systemic side effects.

Endowed with this FDA designation, G2B-002 benefits from enhanced regulatory support and strategic momentum, smoothing its transition from advanced preclinical development into clinical trial phase.

"Receiving ODD from both the EMA and US FDA reaffirms our conviction in G2B-002’s potential to address critical unmet needs in pediatric oncology," said Meritxell Teixidó, CEO & CSO of Gate2Brain. "This dual recognition brings us a step closer to clinical trials and, ultimately, to offering new hope for young patients and their families."

Founded in 2020 as a spin-off from IRB Barcelona, University of Barcelona, and Sant Joan de Déu Pediatric Hospital, Gate2Brain remains dedicated to transforming the treatment landscape for pediatric brain tumors through innovative delivery solutions.

The company has received support from institutions including Fundación Botín (Mind the Gap), Banco Sabadell (BStartup Health), CDTI (Neotec, Multipaís), the European Innovation Council (EIC Accelerator), Startup Capital d’ACCIÓ and CaixaResearch Consolidate de la "Fundació la Caixa".