On April 17, 2025 BigHat Biosciences ("BigHat"), a biotechnology company with a machine learning (ML)-guided antibody discovery and development platform, reported a strategic collaboration with Eli Lilly and Company ("Lilly") (Press release, BigHat Biosciences, APR 17, 2025, View Source [SID1234651981]). In this collaboration, BigHat will deploy its Milliner platform, a suite of state-of-the-art ML technologies integrated with a synthetic biology-based high-speed wet lab, to design and engineer therapeutic antibodies with superior functionality.

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BigHat and Lilly will collaborate to design and discover next-generation antibodies for up to two antibody therapeutic programs. This partnership aims to design, engineer, and develop antibodies with enhanced functionality and improved biophysical properties to create therapeutics that will benefit patients with chronic disease.

"Partnering with Lilly represents an exciting opportunity to harness the full potential of AI-driven biologic design. By combining Lilly’s deep expertise in drug discovery and development with BigHat’s machine learning-powered Milliner platform, we can accelerate the advancement of truly differentiated, next-generation protein therapeutics," said Peyton Greenside, CEO and Co-founder of BigHat.

This collaboration builds on BigHat’s strategy to engage in value-generating strategic partnerships balanced with an exciting internal pipeline of proprietary therapeutics in the areas of oncology and immunology. BigHat’s ML-powered platform is designed to tackle molecular engineering challenges and unlock the development of novel therapeutics for the improvement of patient outcomes. In addition to supporting the development of its partners’ therapeutic programs, BigHat has built and advanced a pipeline of next-gen antibody-drug conjugates (ADCs) and functionally differentiated T-cell engagers (TCEs) across indications in both oncology and immunology. BigHat is advancing a next-generation ADC for GI cancers into the clinic in 2026. Lilly Catalyze360 will provide support, while BigHat retains full global rights and control to the program.

In addition to the antibody discovery collaboration and support for BigHat’s ADC for GI cancers, Lilly will also make an equity investment in BigHat. This collaboration is part of the Lilly Catalyze360 model, which is a comprehensive approach to empower early-stage biotech startups across all therapeutic areas by providing access to funding as well as world-class lab space and/or drug development talent and resources through its three pillars: Lilly Ventures, Lilly Gateway Labs, and Lilly ExploR&D.

On April 17, 2025 Kairos Pharma, Ltd. (NYSE American: KAPA) a clinical stage company involved in treating EGFR-driven lung cancer patients with ENV105 in combination with osimertinib after they fail to respond to single-agent osimertinib in a Phase 1 trial, reported that based on recent breakthroughs in understanding non-small cell lung cancer mechanism of resistance to first line osimertinib treatment, the U.S. Department of Defense ("DoD") is providing $876,000 to advance a strategy to identify patients that are starting to develop resistance at an early stage (Press release, Kairos Pharma, APR 17, 2025, View Source [SID1234651982]). The grant was awarded to identify biomarkers for the Company’s clinical study to address the major challenge in achieving a lasting cure for lung cancer patients. The grant was awarded to Cedars-Sinai Medical Center to support research in Dr. Neil Bhowmick’s laboratory to identify biomarkers of patients with non-small cell carcinoma who have developed resistance to Osimertinib. This will provide a means to identify those patients who will benefit from ENV105.

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Kairos Pharma CEO John Yu, M.D., stated, "Peer-reviewed support like this DoD grant is a testament to the sound scientific basis for our current Phase 1 trial in lung cancer patients receiving ENV105. It is particularly gratifying in the current environment, in which these grants are harder to achieve, underscoring the potential for ENV105 to help ensure effective therapy in this lung cancer form common to non-smokers. Grants like this enable us to progress with our trials while minimizing expense and managing our cash burn."

The DoD grant is designed to contribute to a strategy to limit resistance to osimertinib at its most early stages, by identifying patients that would be best helped by ENV105 treatment. In the near term, the Company expects that the funded study may lead to more effective monitoring and early detection of resistance development, allowing for more timely interventions to improve overall survival and quality of life for patients.

On April 17, 2025 Norgine reported that the Australian Therapeutic Goods Administration (TGA) has approved the registration of IFINWIL (eflornithine) for the treatment of adults and paediatric patients with high-risk neuroblastoma (HRNB), who have responded to prior multiagent, multimodality therapy (Press release, Norgine, APR 16, 2025, View Source [SID1234651964]).

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Neuroblastoma Australia welcomed the news today:

"On behalf of all families of children impacted by neuroblastoma, we welcome the TGA’s decision to approve IFINWIL. We urgently need treatments for children diagnosed with neuroblastoma and this milestone marks a step in the right direction towards a better future for children and their families. We thank the Federal Government for taking action to ensure access and we look forward to continued support for children with aggressive cancers." said Lucy Jones, CEO Neuroblastoma Australia.

High Risk Neuroblastoma is a rare but aggressive form of cancer, predominantly affecting children and most commonly presenting in the first 5 years of life2. Each year in Australia, approximately 50 children are diagnosed with neuroblastoma, with about half of these cases being classified as high risk neuroblastoma3,4. Neuroblastoma originates in the body’s nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands5. It is considered an aggressive tumour because it often spreads to other parts of the body (metastasizes). In most cases, it has spread by the time it is diagnosed5.

"We are committed to improving the lives of children and their families living with high-risk neuroblastoma" said Gus Rudolph, General Manager, Norgine, Australia. "This rare childhood cancer has devastating consequences for those impacted and while more needs to be done to improve treatment outcomes, we would like to recognise the TGA for their work to-date on this approval. Norgine will continue to engage with the relevant stakeholders to bring IFINWIL to patients as quickly as possible."

"This approval, as part of the Project Orbis initiative, represents a vital step forward in ensuring access to innovative cancer treatments for patients around the world," said Dr. David Gillen, Chief Medical Officer, Norgine. "By working collaboratively with international regulatory partners, we are able to help bring promising therapies to paediatric patients sooner – a goal that is especially important when time is critical. We’re proud to support Project Orbis as we strive to expedite access to high-impact oncology medicines."

Receiving the TGA approval is an important milestone on the path to realising sustainable and equitable access. IFINWIL is not currently included on the PBS.

Please refer to the IFINWIL Consumer Medicines Information (CMI) for full safety information on risks, side effects and precautions including the risk of low red blood cells (anaemia), low neutrophils (blood cells that fight infection), low platelets (clotting cells), increase in liver enzymes, and hearing loss or problems balancing.1

Notes to Editors:

About IFINWIL

IFINWIL has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy.6 IFINWIL is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines, which are important to tumour growth and development 7.

For more information on IFINWIL, find the CMI here: IFINWIL Consumer Medicine Information (CMI) or the Therapeutic Goods Administration at View Source or speak to your healthcare practitioner.

High-Risk Neuroblastoma (HRNB) Treatment Background

Children diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapse and death.8 Although there have been some improvements in survival, children with high risk neuroblastoma still face a 30% chance of recurrence (relapse) within the first 5 years post maintenance, and have an extremely poor prognosis and low likelihood of long term survival9 (e.g. estimates as low as 15% of patients will live for five years after relapsing).10 Avoiding relapse is key to long-term survival, and until now, there have been no approved therapies for the post maintenance treatment period in major markets outside of the United States11.

About Project Orbis

Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health).

In April 2024, Norgine submitted an application for approval of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. This milestone supports Norgine’s efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology.

On April 16, 2025 Bayer reported that it will present new data for NUBEQA (darolutamide) in prostate cancer at the upcoming American Urological Association (AUA) Annual Meeting taking place in Las Vegas from April 26-29, 2025 (Press release, Bayer, APR 16, 2025, View Source [SID1234651965]). These data support the potential of NUBEQA as a treatment option across the prostate cancer disease spectrum and in diverse patient populations.

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NUBEQA is indicated in the U.S. for the treatment of adult patients with metastatic hormone-sensitive prostate cancer (mHSPC) in combination with docetaxel and for the treatment of adult patients with non-metastatic castration-resistant prostate cancer (nmCRPC).1

The company will present post-hoc analyses of ultra-low prostate-specific antigen (PSA) responses (<0.02 ng/mL) and the correlation of ultra-low PSA responses with clinical outcomes from the Phase III ARANOTE trial evaluating NUBEQA and androgen deprivation therapy (ADT) in mHSPC.

An additional ARANOTE analysis on the efficacy and safety of NUBEQA plus ADT in Black men with mHSPC will be presented.

Additionally, data from the Phase III ARASENS Rollover trial evaluating the long-term safety and tolerability benefit of extended treatment with NUBEQA will be presented.

Other key data to be presented include the North American subgroup analysis from the third interim analysis of the Darolutamide Observational (DAROL) trial evaluating the safety and efficacy of NUBEQA in a real-world setting in patients with nmCRPC, and an update on the in progress Phase III ARASTEP trial, evaluating NUBEQA plus ADT in patients with high-risk biochemical recurrence.

A separate presentation will highlight results from a U.S.-based quantitative survey on the challenges and unmet needs of caregivers of men with prostate cancer.

Details on selected abstracts from Bayer at the AUA 2025 Annual Meeting follow:

NUBEQA (darolutamide):

Ultra-low PSA Response (<0.02 ng/mL) with Darolutamide Plus ADT in ARANOTE Correlates with Greatly Improved Clinical Outcomes
Interactive Poster Session: IP26-07; April 29, 9:30-11:30 a.m. PDT
Efficacy and Safety of Darolutamide Plus Androgen-Deprivation Therapy (ADT) in Black Patients with Metastatic Hormone-Sensitive Prostate Cancer (mHSPC) from the Phase 3 ARANOTE Trial
Moderated Poster: MP16-01; April 27, 1:00-3:00 p.m. PDT
Long-Term Safety and Tolerability of Extended Treatment with Darolutamide in Metastatic Hormone-Sensitive Prostate Cancer (mHSPC): Insights from ARASENS Rollover Study
Interactive Poster: IP26-06; April 29, 9:30-11:30 a.m. PDT
Prespecified Third Interim Analysis of the Darolutamide Observational (DAROL) Study in Patients with Nonmetastatic Castration-Resistant Prostate Cancer (nmCRPC): North American (NAm) Subgroup Analysis
Interactive Poster: IP26-25; April 29, 9:30-11:30 a.m. PDT
Darolutamide Plus Androgen-Deprivation Therapy (ADT) in Patients with High-Risk Biochemical Recurrence (BCR) of Prostate Cancer: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study (ARASTEP)
Clinical Trials in Progress Presentation: April 28, 2:44- 2:52 p.m. PDT
Prostate Cancer:

Challenges and Unmet Needs of Caregivers for Patients with Prostate Cancer: A US-based Quantitative Survey
Abstract IP04-31; April 26, 9:30-11:30 a.m. PDT
About NUBEQA (darolutamide)1

NUBEQA (darolutamide) is an androgen receptor inhibitor (ARi) with a distinct chemical structure that competitively inhibits androgen binding, AR nuclear translocation, and AR-mediated transcription.

NUBEQA is developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company.

NUBEQA is an androgen receptor inhibitor indicated for the treatment of adult patients with:

Non-metastatic castration-resistant prostate cancer (nmCRPC)
Metastatic hormone-sensitive prostate cancer (mHSPC) in combination with docetaxel
IMPORTANT SAFETY INFORMATION

Warnings & Precautions

Ischemic Heart Disease – In a study of patients with nmCRPC (ARAMIS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA versus 2.5% receiving placebo, including Grade 3-4 events in 1.7% vs. 0.4%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA vs. 0.2% receiving placebo. In a study of patients with mHSPC (ARASENS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA with docetaxel vs. 2% receiving placebo with docetaxel, including Grade 3-4 events in 1.3% vs. 1.1%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA with docetaxel vs. 0% receiving placebo with docetaxel. Monitor for signs and symptoms of ischemic heart disease. Optimize management of cardiovascular risk factors, such as hypertension, diabetes, or dyslipidemia. Discontinue NUBEQA for Grade 3-4 ischemic heart disease.

Seizure – In ARAMIS, Grade 1-2 seizure occurred in 0.2% of patients receiving NUBEQA vs. 0.2% receiving placebo. Seizure occurred 261 and 456 days after initiation of NUBEQA. In ARASENS, seizure occurred in 0.6% of patients receiving NUBEQA with docetaxel, including one Grade 3 event, vs. 0.2% receiving placebo with docetaxel. Seizure occurred 38 to 340 days after initiation of NUBEQA. It is unknown whether antiepileptic medications will prevent seizures with NUBEQA. Advise patients of the risk of developing a seizure while receiving NUBEQA and of engaging in any activity where sudden loss of consciousness could cause harm to themselves or others. Consider discontinuation of NUBEQA in patients who develop a seizure during treatment.

Embryo-Fetal Toxicity – Safety and efficacy of NUBEQA have not been established in females. NUBEQA can cause fetal harm and loss of pregnancy. Advise males with female partners of reproductive potential to use effective contraception during treatment with NUBEQA and for 1 week after the last dose.

Adverse Reactions

In ARAMIS, serious adverse reactions occurred in 25% of patients receiving NUBEQA vs. 20% of patients receiving placebo. Serious adverse reactions in ≥1% of patients who received NUBEQA included urinary retention, pneumonia, and hematuria. Fatal adverse reactions occurred in 3.9% of patients receiving NUBEQA vs. 3.2% of patients receiving placebo. Fatal adverse reactions in patients who received NUBEQA included death (0.4%), cardiac failure (0.3%), cardiac arrest (0.2%), general physical health deterioration (0.2%), and pulmonary embolism (0.2%). The most common adverse reactions (>2% with a ≥2% increase over placebo), including laboratory test abnormalities, were increased AST, decreased neutrophil count, fatigue, increased bilirubin, pain in extremity and rash. Clinically relevant adverse reactions occurring in ≥2% of patients treated with NUBEQA included ischemic heart disease and heart failure.

In ARASENS, serious adverse reactions occurred in 45% of patients receiving NUBEQA with docetaxel vs. 42% of patients receiving placebo with docetaxel. Serious adverse reactions in ≥2% of patients who received NUBEQA with docetaxel included febrile neutropenia (6%), decreased neutrophil count (2.8%), musculoskeletal pain (2.6%), and pneumonia (2.6%). Fatal adverse reactions occurred in 4% of patients receiving NUBEQA with docetaxel vs. 4% of patients receiving placebo with docetaxel. Fatal adverse reactions in patients who received NUBEQA included COVID-19/COVID-19 pneumonia (0.8%), myocardial infarction (0.3%), and sudden death (0.3%). The most common adverse reactions (≥10% with a ≥2% increase over placebo with docetaxel) were constipation, rash, decreased appetite, hemorrhage, increased weight, and hypertension. The most common laboratory test abnormalities (≥30%) were anemia, hyperglycemia, decreased lymphocyte count, decreased neutrophil count, increased AST, increased ALT, and hypocalcemia. Clinically relevant adverse reactions in <10% of patients who received NUBEQA with docetaxel included fractures, ischemic heart disease, seizures, and drug-induced liver injury.

Drug Interactions

Effect of Other Drugs on NUBEQA – Combined P-gp and strong or moderate CYP3A4 inducers decrease NUBEQA exposure, which may decrease NUBEQA activity. Avoid concomitant use.

Combined P-gp and strong CYP3A4 inhibitors increase NUBEQA exposure, which may increase the risk of NUBEQA adverse reactions. Monitor more frequently and modify NUBEQA dose as needed.

Effects of NUBEQA on Other Drugs – NUBEQA inhibits breast cancer resistance protein (BCRP) transporter. Concomitant use increases exposure (AUC) and maximal concentration of BCRP substrates, which may increase the risk of BCRP substrate-related toxicities. Avoid concomitant use where possible. If used together, monitor more frequently for adverse reactions, and consider dose reduction of the BCRP substrate.

NUBEQA inhibits OATP1B1 and OATP1B3 transporters. Concomitant use may increase plasma concentrations of OATP1B1 or OATP1B3 substrates. Monitor more frequently for adverse reactions and consider dose reduction of these substrates.

Review the Prescribing Information of drugs that are BCRP, OATP1B1, and OATP1B3 substrates when used concomitantly with NUBEQA.

For important risk and use information about NUBEQA, please see the accompanying full Prescribing Information.

About Metastatic Hormone-Sensitive Prostate Cancer

Prostate cancer is the second most common cancer in men and the fifth most common cause of cancer death in men worldwide.2 In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, including almost 300,000 cases in the U.S., and about 375,000 died from the disease worldwide.3,4

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy. Upon relapse when the disease will metastasize or spread, androgen deprivation therapy (ADT) is the cornerstone of treatment for this hormone-sensitive disease. Approximately 10% of men will already present with mHSPC when first diagnosed.5,6,7 Men with metastatic hormone-sensitive prostate cancer (mHSPC) will start their treatment with hormone therapy, such as ADT, androgen receptor inhibitor (ARi) plus ADT or a combination of the chemotherapy docetaxel and ADT. Despite this treatment, most men with mHSPC will eventually progress to castration-resistant prostate cancer (CRPC), a condition with limited survival.

On April 16, 2025 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX: BCT) ("BriaCell" or the "Company"), a clinical-stage biotechnology company that develops novel immunotherapies to transform cancer care, reported new positive survival data in its Phase 2 study of Bria-IMT plus check point inhibitors (CPI), outperforming ADC drugs in hormone receptor positive (HR+) metastatic breast cancer (MBC) patients (Press release, BriaCell Therapeutics, APR 16, 2025, View Source [SID1234651969]).

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In BriaCell’s Phase 2 clinical study in late-stage MBC, 25 of 37 patients treated with the ongoing pivotal Phase 3 Bria-IMT formulation were identified as having HR+ breast cancer. As shown in Table 1, the survival data of these 25 patients (17.3 months) exceeds those of the current ADC standard of care TRODELVY (14.4 months). The survival data for the Bria-IMT regimen + immune check point inhibitor in the triple negative breast cancer (TNBC), characterized by the absence of estrogen (ER), progesterone (PR) and human epidermal growth factor (HER2) receptors, was similar to TRODELVY but still markedly higher (70%) than those of chemotherapy.

"We are truly impressed with the survival benefit data of the regimen that exceeds or meets those of TRODELVY in HR+ and TNBC metastatic breast cancer patients, respectively. Bria-IMT appears to be very well-tolerated," stated Dr. William V. Williams, BriaCell’s President and CEO. "We look forward to further confirming this clinical data in our ongoing pivotal Phase 3 study with overall survival as its primary endpoint."

"HR+ and TNBC metastatic breast cancer represent a significant proportion of the patient population and are the most difficult patient groups to treat. They have limited therapeutic options and overall survival of only a few months," commented Dr. Giuseppe Del Priore, BriaCell’s Chief Medical Officer. "Our clinical data supports our hypothesis that the Bria-IMT regimen + CPI has the potential to address the unmet medical needs of HR+ and TNBC MBC patients and provide an effective and well-tolerated therapeutic option."

Table 1: Comparable Analysis of median overall survival (estimated using the Kaplan-Meier method) for the BriaCell Phase 2 study of BriaCell’s Bria-IMT plus CPI versus other drugs in MBC patient subsets

* Patients treated with the Phase 3 formulation
** Number of prior chemotherapy-containing regimens
1. View Source

Abbreviations:
HR+: hormone receptor-positive
TNBC: Triple-negative breast cancer (lacks the estrogen receptor, progesterone receptor, and lacks or has low levels of human epidermal growth factor receptor 2 (HER2))

The Phase 2 study enrolled 54 heavily pre-treated metastatic breast cancer patients (median number of prior treatments = 6) who received the Bria-IMT regimen plus checkpoint inhibitor. Of these 54 patients, 37 were treated with the formulation currently being used in BriaCell’s ongoing pivotal Phase 3 study in metastatic breast cancer (listed on ClinicalTrials.gov as NCT06072612 ). No Bria-IMT related discontinuations have been reported to date.