On March 13, 2025 Blue Earth Therapeutics reported further promising developments for its radiohybrid lutetium labelled, PSMA targeted, investigational radioligand therapy (Press release, Blue Earth Therapeutics, MAR 13, 2025, View Source [SID1234651138]). Radiation dosimetry and pharmacokinetic data from the 13 metastatic castrate resistant prostate cancer patients enrolled in the Phase 1 portion of a Phase 1/2 clinical trial (NCT05413850) of Lutetium (177Lu) rhPSMA-10.1 Injection showed proportionately higher absorbed radiation doses in tumours than in critical healthy tissues such as the kidneys. The data compares favourably to published data on first-generation PSMA-targeted radioligand therapies.1,2

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The Phase 1 data shows that Lutetium (177Lu) rhPSMA-10.1 Injection has high ratios for absorbed radiation dose to tumours vs. dose to healthy tissues, with a measured mean tumour to salivary gland ratio of 73 and tumour to kidney ratio of 32. Median absorbed radiation dose to tumours defined by SPECT imaging was 8.9 Gy for each GBq of administered radioactivity. Mean absorbed radiation dose to the kidneys was 0.27 Gy/GBq; to salivary glands, 0.13Gy/GBq.

Underlying these results, the mean biological half-life in tumors for the Lutetium (177Lu) rhPSMA-10.1 Injection was 338 hours. When paired with the 6.7-day physical half-life of 177Lu, this gives an effective mean half-life of 91.4 hours. This allows delivery of radiation to tumors over many days: one and a half to twice the reported data for established agents in this class1. This prolonged retention of the drug in tumors without proportionate increases to retention in normal tissues helps to explain the positive radiation dosimetry data.

Following recent consultation with regulatory authorities, and sharing of the preliminary data, this now opens the way for the Phase 2 portion of the Phase 1/2 trial to test innovative dosing regimens, with the goal of optimising outcomes for patients. This Phase 2 portion of the study will explore the following dosing concepts:

Administration of significantly higher overall injected radioactivity in comparison to recent Phase 3 clinical trials of other PSMA-targeted radioligand therapies
Front loading of administered radioactivity in early cycles; and
Extending the duration of administration of radioactivity beyond 36 weeks to provide longer time on treatment.
In combination with the promising Phase 1 data for Lutetium (177Lu) rhPSMA-10.1 Injection, these design factors should further support the aim of maximizing treatment response and therefore may enable delivery of better outcomes for patients. The Phase 2 portion of the study is expected to start this quarter.

David Gauden DPhil, CEO of Blue Earth Therapeutics said, "The Phase 1 data provides strong validation of the innovative approach taken on optimizing radioligand therapy by Blue Earth Therapeutics and the inventors of the rhPSMA technology. The relative ratios of tumour to healthy organ absorbed radiation doses are key metrics in establishing a better profile of the risks and potential benefits of radioligand therapies. With radioligand therapies, normal organ toxicity considerations gate the total amount of radioactivity that can be administered, so the more of the radioactivity that accumulates in tumours, the better. Our goal is to substantially increase the potential for prostate cancer patients to benefit compared to available radioligand therapy, and completion of this study moves us closer to making that goal a reality."

About metastatic prostate cancer
In 2025 it is estimated that there will be 50,055 new cases of metastatic prostate cancer in the United States (de novo diagnoses plus recurrence from earlier stage diagnoses).3 Five-year survival for newly diagnosed metastatic prostate cancer is low, 36.6%.4 While death rates from prostate cancer have declined over the past three decades4, there is still considerable room to improve patient outcomes.

About Radiohybrid Prostate–Specific Membrane Antigen (rhPSMA)
rhPSMA compounds are referred to as radiohybrid ("rh"), as each molecule possesses four distinct domains. The first consists of a Prostate–Specific Membrane Antigen–targeted receptor ligand. It is attached to two labelling moieties which may be radiolabeled with diagnostic isotopes such as 18F or 68Ga for PET imaging, or with therapeutic isotopes such as 177Lu or 225Ac for radioligand therapy, all of which are joined together by a modifiable linker which can be used to modulate important pharmacokinetic characteristics. Radiohybrid PSMA offers the potential for targeted treatment for men with prostate cancer and originated at the Technical University of Munich, Germany. Blue Earth Diagnostics acquired exclusive worldwide rights to rhPSMA diagnostic imaging technology from Scintomics GmbH in 2018, and therapeutic rights in 2020, and has sublicensed the therapeutic application to its sister company Blue Earth Therapeutics.

On March 13, 2025 Immorta Bio Inc., a scientific longevity company focused on "Treating Diseases of Aging and Treating Aging as Disease," reported that its Chairman and CEO, Dr. Boris Reznik, presented the latest findings from its SenoVax Program at the LSX Investival Showcase on March 11th at the Miami Beach Convention Centre (Press release, Immorta Bio, MAR 13, 2025, View Source [SID1234651137]).

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The presentation disclosed advancements in the Company’s oncology pipeline, highlighting animal studies showing the regression of brain, lung, skin, pancreas, and breast cancers by SenoVax, the Company’s lead product. Additionally, cellular mechanisms by which this "longevity therapeutic" targets a broad range of cancers were discussed.

"It has been widely demonstrated that senescent cells play a critical role in initiating, promoting and protecting cancer," said Dr. Thomas Ichim, President and Chief Scientific Officer of the Company. "By amplifying the body’s natural immunological activities to kill senescent cells, SenoVax induces regression of tumors."

The Company, together with the University of California San Diego, George Washington University, Calidi Biosciences, and Cedars Sinai, demonstrated in a peer-reviewed publication that SenoVax kills cancer in part by inducing antibody and T cell responses that eliminate senescent cells¹.

"We are developing multiple programs focused on radical extension of human healthspan including our senolytic immunotherapy platform," said Dr. Boris Reznik, Chairman and CEO of Immorta Bio. "Our development strategy is to apply longevity-associated technologies to address unmet medical needs that can provide answers in months not decades."

On March 13, 2025 Enliven Therapeutics, Inc. (Enliven or the Company) (Nasdaq: ELVN), a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapeutics, reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a business update, including highlights of pipeline progress (Press release, Enliven Therapeutics, MAR 13, 2025, View Source [SID1234651136]).

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"We are very pleased with the ongoing progress we are making as a Company. We continue to hear significant enthusiasm and excitement from investigators on both of our programs. The momentum of ELVN-001 has accelerated since the data presentation at the ESH-iCMLf conference," said Sam Kintz, Co-founder and Chief Executive Officer of Enliven. "2025 is a big year for Enliven and we are excited for the upcoming program updates. We are focused on continued clinical execution and preparing for the potential start of a pivotal trial for ELVN-001 in 2026."

Recent Research and Development Highlights and Upcoming Milestones

ELVN-001 is a potent, highly selective, small molecule kinase inhibitor designed to specifically target the BCR::ABL gene fusion

In 2024, the Company announced positive initial data from the ongoing Phase 1 clinical trial that compared favorably to the precedent Phase 1 trials for the approved BCR::ABL1 tyrosine kinase inhibitors (TKIs).
The U.S. Food and Drug Administration (FDA) granted orphan drug designation to ELVN-001 for the treatment of chronic myeloid leukemia (CML).
The first patient was dosed in the Phase 1 trial evaluating ELVN-001 in Japanese patients with CML (NCT06787144).
The Company plans to report additional data from the ongoing Phase 1a/b trial in the middle of 2025.
ELVN-002 is a potent, highly selective, central nervous system (CNS) penetrant and irreversible HER2 inhibitor with activity against wild type HER2 and various HER2 mutations

Enliven continued to enroll patients in its Phase 1 trial evaluating ELVN-002 as a monotherapy agent in patients with HER2+ and HER2 mutant tumors and its exploratory cohort in combination with Kadcyla (an approved HER2 antibody drug conjugate) in patients with HER2+ metastatic breast cancer (MBC) (NCT05650879).
Additionally, the Company continued to enroll patients in its Phase 1 trial evaluating ELVN-002 in combination with trastuzumab +/- chemotherapeutic agents in patients with HER2+ MBC and colorectal cancer (CRC) and began dosing patients in the Phase 1b trial specifically evaluating patients with CRC (NCT06328738).
Enliven plans to report data from the ongoing Phase 1 trials in the second half of 2025.
Fourth Quarter and Full Year 2024 Financial Results

Cash Position: As of December 31, 2024, the Company had cash, cash equivalents and marketable securities totaling $313.4 million, which is expected to provide cash runway into mid-2027.
Research and development (R&D) expenses: R&D expenses were $20.7 million for the fourth quarter of 2024, compared to $17.9 million for the fourth quarter of 2023. R&D expenses were $80.8 million for the full year 2024, compared to $64.6 million for the full year 2023.
General and administrative (G&A) expenses: G&A expenses for the fourth quarter of 2024 were $6.2 million, compared to $4.8 million for the fourth quarter of 2023. G&A expenses were $23.8 million for the full year 2024, compared to $19.0 million for the full year 2023.
Net Loss: Enliven reported a net loss of $23.2 million for the fourth quarter of 2024, compared to a net loss of $19.4 million for the fourth quarter of 2023. Total net loss for the full year 2024 was $89.0 million, compared to $71.6 million for the full year 2023.

On March 13, 2025 Medigene AG (Medigene, FSE: MDG1, Prime Standard), an oncology platform company focused on the research and development of T cell receptor (TCR)-guided therapies for the treatment of cancer, reported the submission of a patent for a novel natural killer (NK) cell-specific TCR construct to the European Patent Office. With that, Medigene is advancing its TCR-guided strategy by expanding the application of its proprietary 3S (sensitive, specific, and safe) TCRs into NK cells (Press release, MediGene, MAR 13, 2025, View Source [SID1234651135]). This patent application marks a major expansion of Medigene’s intellectual property portfolio, broadening its therapeutic reach and reinforcing its commitment to developing innovative, off-the-shelf immunotherapies for cancer treatment.

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To enable this innovation, Medigene combined TCR-guided precision targeting with innate NK cell killing. The Company has developed a proprietary universal scaffold that allows its 3S TCRs to function in NK cells without requiring CD3 co-expression. This novel NK-TCR system enhances NK cell production and therapeutic potential by facilitating the efficient transfer of all 3S TCRs into NK cells, streamlining the manufacturing process for future allogeneic TCR-NK therapies. By eliminating CD3 dependency, this approach overcomes a critical limitation in TCR-based NK cell therapy development, delivering a scalable, clinically viable solution that accelerates therapeutic timelines and reduces costs.

"This innovative approach supports our strategy to apply Medigene’s 3S TCRs into new TCR-guided modalities, such as TCR-TCEs and now TCR-NKs, to create additional value for both patients and our shareholders," said Selwyn Ho, CEO of Medigene. "Expanding our TCR-guided therapies to include NK cells aligns with our commitment to developing off-the-shelf, highly specific, and effective immunotherapies for cancer treatment."

The global NK cell therapeutics market is rapidly expanding, driven by rising cancer prevalence, an aging population, and demand for innovative immunotherapies. Valued at ~$0.55B in 2024, it is projected to reach $2.13B by 2033 (Source:Business Research Insights). Medigene’s proprietary TCR-NK technology is well-positioned to drive this growth with highly specific, scalable NK cell-based therapies.

Medigene continues to strengthen its intellectual property portfolio through the generation of new 3S TCRs, development of advanced technologies, and strategic expansion of existing patents across additional geographies. With over 29 unique patent families worldwide, Medigene safeguards its proprietary TCR and End-to-End Platform technologies, ensuring a strong competitive position in the immunotherapy space.

On March 13, 2025 aTyr Pharma, Inc. (Nasdaq: ATYR) ("aTyr" or the "Company"), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, reported fourth quarter and full year 2024 results and provided a corporate update (Press release, aTyr Pharma, MAR 13, 2025, View Source [SID1234651134]).

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"2024 was an important year for aTyr, as we completed enrollment in our global pivotal Phase 3 EFZO-FIT study, which is the largest interventional study ever to be conducted in pulmonary sarcoidosis, a disease where incidence and prevalence are rising yet there are still limited treatment options," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "The stage is set for 2025 to be a potential milestone year as we look toward reporting topline data for EFZO-FIT in the third quarter. Leading into this pivotal readout, we are very pleased with the execution of the study to date and the continued favorable safety profile demonstrated for efzofitimod, including our most recent positive DSMB review, which recommended the study continue as designed. Efzofitimod is poised to potentially become a safe and effective alternative to the current standard of care and improve patients’ lives."

Fourth Quarter 2024 and Subsequent Period Highlights

Enrollment complete in the global pivotal Phase 3 EFZO-FIT study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a randomized, double-blind, placebo-controlled, 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously monthly for a total of 12 doses. The study enrolled 268 patients with pulmonary sarcoidosis at 85 centers in nine countries. The trial design incorporates a forced steroid taper. The primary endpoint of the study is steroid reduction. Secondary endpoints include measures of sarcoidosis symptoms and lung function. Topline data from the study are expected in the third quarter of 2025. Patients who complete the study and wish to receive treatment with efzofitimod outside of the clinical trial are eligible to participate in an Individual Patient Expanded Access Program.
Held a Type C meeting with the U.S. Food and Drug Administration (FDA) to discuss the statistical analysis plan (SAP) for the Phase 3 EFZO-FIT study. The main objective of the meeting was to finalize the way in which the study endpoints are assessed from a statistical standpoint. Based on FDA feedback, steroid reduction will be measured as the absolute change from baseline to week 48.
Announced the fourth positive data and safety monitoring board (DSMB) review for the Phase 3 EFZO-FIT study. The independent DSMB recommended that the study continue without modifications based on the pre-planned analysis.
Enrollment ongoing in the Phase 2 EFZO-CONNECT study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with limited and diffuse systemic sclerosis (SSc, or scleroderma)-related interstitial lung disease (SSc-ILD). This proof-of-concept study is a randomized, double-blind, placebo-controlled, 28-week study consisting of three parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or placebo dosed intravenously monthly for a total of six doses. The study intends to enroll up to 25 patients at multiple centers in the United States. Patients who complete the study are eligible to participate in a 24-week open-label extension. Interim data from the study are expected in the second quarter of 2025 and will focus on skin assessments measured at baseline and week 12 in approximately 8 patients, including patients on drug and placebo. The data will include skin histopathology, including immune biomarkers, and the modified Rodnan skin score.
Manuscript demonstrating the immunomodulatory activity for efzofitimod in lung inflammation and fibrosis published in the peer-reviewed journal Science Translational Medicine. The publication describes efzofitimod’s unique anti-inflammatory effect on macrophages through neuropiln-2 (NRP2) and details the preclinical data supporting its development. The publication, which is entitled, "A human histidyl-tRNA synthetase splice variant therapeutic targets NRP2 to resolve lung inflammation and fibrosis," is available on the journal’s website and at: View Source
Posters for efzofitimod accepted for presentation at the upcoming American Thoracic Society (ATS) 2025 International Conference. The conference is scheduled to take place May 16 – 21, 2025, in San Francisco, CA.
Poster 9320 – Real-World Treatment Patterns Among Pulmonary Sarcoidosis Patients with Parenchymal Involvement in the US on Sunday May 18, 2025, at 11:30 a.m. PDT.
Poster 6808 – EFZO-FIT, Largest Placebo-Controlled Trial in Pulmonary Sarcoidosis – Trial Design and Patient Characteristics on Monday, May 19, 2025, at 11:30 a.m. PDT.
Poster 9092 – Incidence, Prevalence, and Mortality of Pulmonary Sarcoidosis with Parenchymal Involvement in the US on Tuesday May 20, 2025, at 11:30 a.m. PDT.
Presented preclinical research demonstrating anti-fibrotic effects of ATYR0101 in lung and kidney fibrosis in two posters at the Keystone Symposia on Fibrosis: Inflammation, Drivers, and Therapeutic Resolution. The findings demonstrated that ATYR0101 interacts with LTBP-1 to induce myofibroblast apoptosis through a novel anti-fibrotic mechanism and suggest that ATYR0101 has the potential to be a next generation anti-fibrotic drug for lung and kidney fibrosis.
Appointed Eric Benevich to the Company’s Board of Directors. Mr. Benevich currently serves as Chief Commercial Officer at Neurocrine Biosciences, Inc., where he is responsible for all aspects of commercial development, marketing, and sales of the Neurocrine product portfolio.
Year Ended 2024 Financial Highlights and Cash Position

Cash & Investment Position: Cash, cash equivalents, restricted cash and available-for-sale investments as of December 31, 2024, were $75.1 million. Subsequent to the end of the fourth quarter 2024, the Company raised approximately $18.8 million in gross proceeds from its at-the-market (ATM) offering with Jefferies LLC.
Financial Guidance: The Company updated its prior guidance and believes its cash runway will be sufficient to fund its operations for a period of one year following the Phase 3 EFZO-FIT readout, which includes the potential filing of a Biologics License Application (BLA) for efzofitimod in pulmonary sarcoidosis.
R&D Expenses: Research and development expenses were $54.4 million for the year ended 2024, which consisted primarily of clinical trial costs for the Phase 3 EFZO-FIT and Phase 2 EFZO-CONNECT studies, manufacturing costs for the efzofitimod program and research and development costs for the efzofitimod and discovery programs.
G&A Expenses: General and administrative expenses were $13.8 million for the year ended 2024.
Collaboration and License Revenue: Collaboration and license revenue related to the Kyorin Agreement was $0.2 million for the year ended 2024, which consisted of drug product material sold to Kyorin for the Japan portion of the EFZO-FIT study.
Conference Call and Webcast Details

aTyr will host a conference call and webcast today at 5:00 p.m. EDT / 2:00 p.m. PDT to discuss its financial results and provide a corporate update. Interested parties may access the call by registering here in order to obtain a dial in, personalized passcode and webcast information. Links to a live audio webcast and replay may be accessed on the aTyr website Events page at: View Source An audio replay will be available for at least 90 days following the event.

About Efzofitimod

Efzofitimod is a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. aTyr is currently investigating efzofitimod in the global Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, a major form of ILD, and in the Phase 2 EFZO-CONNECT study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.