On February 25, 2025 Alpheus Medical, Inc., a private, clinical-stage oncology company pioneering sonodynamic therapy (SDT) for the treatment of solid body cancers, reported the publication of a study in the Journal of Neuro-Oncology showcasing its proprietary SDT treatment in three patients with newly diagnosed glioblastomas that were not candidates for gross total tumor resection (Press release, Alpheus Medical, FEB 25, 2025, View Source [SID1234650557]). The study reported no adverse effects with an immediate positive imaging and histopathological response indicating cancer cell death, with no impact on healthy brain tissue, after a single dose of SDT. The Company shared that it expects to begin a randomized control trial in patients with newly diagnosed glioblastoma later this year.

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"The early clinical results are very encouraging for advancing treatment options in patients with newly diagnosed glioblastoma, a disease with few effective therapies," commented Walter Stummer, Professor and Director of the Department of Neurosurgery of the University Hospital Münster, Germany, and the senior author of the study. "Sonodynamic therapy’s ability to selectively induce immediate tumor cell death while sparing healthy brain tissue is unprecedented. Additionally, its diffuse nature allows treatment across the entire hemisphere, including the peripheral invasive zone and beyond, a major challenge in neuro-oncology. These factors suggest that SDT could be a significant breakthrough in glioblastoma treatment."

"SDT’s ability to selectively induce immediate tumor cell death while sparing healthy brain tissue is unprecedented."

Alpheus Medical’s novel SDT therapy delivers low-intensity diffuse ultrasound (LIDU) combined with oral 5-aminolevulinic acid (5-ALA) to selectively target and destroy cancer cells across the entire brain hemisphere. This non-invasive treatment, performed without the need for imaging or sedation, offers a revolutionary new approach to cancer care in an outpatient setting.

This news builds on the previously presented first-in-human positive results from the Company’s Phase 1/2 trial in recurrent, high-grade glioma patients, a population with very short survival expectancy. The open-label, multicenter study demonstrated a greater than twofold increase in median overall survival and a threefold improvement in progression-free survival for patients treated with SDT, underscoring its potential as a transformative therapy for this aggressive disease.

On February 25, 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 that management will participate in a fireside chat at the TD Cowen 45th Annual Health Care Conference on Tuesday, March 4, 2025, at 10:30 a.m. ET (Press release, Enliven Therapeutics, FEB 25, 2025, View Source [SID1234650556]).

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The fireside chat will be webcast live and can be accessed by visiting the investor relations section of the Company’s website at View Source The webcast will be archived for a period of 90 days following the conclusion of the live event.

On February 25, 2025 Novita Pharmaceuticals, Inc. ("Novita"), a privately held, clinical-stage pharmaceutical company dedicated to developing novel cancer drugs through its proprietary fascin inhibitor technology, reported additional results from its Phase 2 study (NCT05023486) evaluating NP-G2-044 in combination with SOC anti-PD-1 therapy in patients with advanced solid tumors resistant to prior anti-PD-1 therapy at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Immuno-oncology (AACR IO) Annual Meeting (Press release, Novita Pharmaceuticals, FEB 25, 2025, View Source [SID1234650555]). The data was presented in a poster presentation titled "Phase 2 Study of NP-G2-044, a Novel Fascin Inhibitor, in Combination with Anti-PD-1 Therapy in Patients with Solid Tumors Resistant to Prior Anti-PD-1 Therapy." Findings indicate that NP-G2-044 provides a novel therapeutic opportunity when combined with ICIs.

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"We are very pleased with the findings generated to date in our Phase 2 trial of NP-G2-044 both as a monotherapy and in combination with anti-PD-1 immune checkpoint inhibitors (ICIs) in ICI-resistant patients with advanced and metastatic solid tumors," said Jillian Zhang, Ph.D., President & Chief Scientific Officer of Novita. "The strong safety and durable efficacy we have observed with our first-in-class fascin inhibitor further support the simultaneous inhibition of metastasis and enhancement of cancer immunotherapy as a promising and innovative approach in cancer treatment with broad applications for many solid tumors. We look forward to sharing additional data from the Phase 2 expansion cohort of NP-G2-044 in combination with ICI in the second half of 2025."

Among the 45 patients treated with NP-G2-044 as of the last data cutoff (Oct. 2024), 80% had progressed on prior anti-PD-(L)1 therapies. The anti-PD-1 Combination RP2D for NP-G2-044 was 1600 mg QD with 4-week cycles. The primary endpoint was objective response rate (ORR), and secondary endpoints included progression-free survival (PFS), metastasis-free interval (MFI), overall survival (OS), safety, and tolerability.

Key highlights include:

A Disease Control Rate of 76% (includes patients with Stable Disease and Objective Responses)
An ORR of 21% (95% CL 9-38.9%) including 4 patients with Partial Response (PR) and 3 patients with Complete Responses (CR) including Pathologic Complete Response
Results indicate durable responses and tumor control in a significant proportion of patients across at least seven cancer types, including cases converted from ICI-non-responsive to ICI-responsive.
Long lasting objective responses have been observed.
Notable outcomes include a CR in a cervical cancer patient, target lesion CR in an endometrial cancer patient, pathological CRs in a pancreatic cancer patient and a patient with gastroesophageal junction adenocarcinoma and PRs in cutaneous squamous cell carcinoma, non-small cell lung cancer, and cholangiocarcinoma.
Seven patients are still on treatments, with the longest duration of 18+ months in an endometrial cancer patient and a patient with pancreatic cancer.
An amendment to the study is underway to open additional cohorts. These new cohorts aim to further evaluate the combination of NP-G2-044 with anti-PD-1 therapy across patient populations and solid tumor subtypes, providing a broader understanding of its therapeutic potential. Future analysis will also explore biomarkers for response prediction and mechanisms of resistance, guiding personalized approaches in treatment-resistant cancer. Novita is on track to begin enrollment in its pivotal Phase 3 study of NP-G2-044 + PLD in platinum resistant ovarian cancer in the third quarter of 2025.

On February 25, 2025 HBM Alpha Therapeutics (HBMAT), Inc., an innovative biotechnology company incubated by Harbour BioMed (HKEX: 02142), reported a strategic collaboration and license agreement with a business partner to advance novel therapies targeting corticotropin-releasing hormone (CRH) for various disorders (Press release, Harbour BioMed, FEB 25, 2025, View Source [SID1234650554]).

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Under the agreement, the partner gains exclusive global rights, excluding Greater China (mainland China, Taiwan, Hong Kong, and Macau), to develop and commercialize HAT001 (designated as HBM9013 by Harbour BioMed), a potent and selective anti-CRH-neutralizing antibody. In return, HBMAT is eligible to receive up to $395 million, including upfront, development, regulatory and commercial milestone payments, as well as tiered royalties on future net product sales. Additionally, HBMAT is also entitled to a warrant to receive minority interest in the partner.

HAT001/HBM9013 is designed to neutralize CRH for various disorders, including congenital adrenal hyperplasia (CAH). CAH is a group of autosomal recessive diseases due to mutations in genes that encode for enzymes necessary for synthesis of key adrenal hormones, which lead to serious health consequences. Current standard of care therapy of CAH was introduced more than 70 years ago. It has not changed significantly since and still has huge unmet medical needs. HAT001/HBM9013 aims to dramatically improve standard of care and improve patient outcomes. It has demonstrated strong preclinical efficacy in downregulating CRH-mediated induction of adrenocorticotropic hormone (ACTH) and is being advanced toward clinical development.

"This collaboration represents a pivotal step for HBMAT in our mission to deliver innovative solutions for patients facing challenging endocrine disorders. The completion of this transaction also marks Harbour BioMed’s partial exit from the first global NewCo we incubated," said Dr. Jingsong Wang, Founder, Chairman & CEO of Harbour BioMed, and Chairman of BOD of HBMAT. "HAT001/HBM9013 has the potential to be a transformative therapy. With their expertise in drug development, our partner is well-positioned to help bring this therapy to patients worldwide."

On February 25, 2025 Telix Pharmaceuticals Limited (ASX: TLX, Nasdaq: TLX, Telix, the Company) reported that the United States (U.S.) Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for its breakthrough investigational kidney cancer PET[1] imaging agent TLX250-CDx (Zircaix[2], 89Zr-DFO-girentuximab), granted a Priority Review and provided a PDUFA[3] date of 27 August 2025, paving the way for a U.S. commercial launch in 2025 (Press release, Telix Pharmaceuticals, FEB 25, 2025, View Source [SID1234650553]).

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If approved, TLX250-CDx will become the first commercially available imaging agent to accurately and non-invasively diagnose and characterize clear cell renal cell carcinoma (ccRCC), the most common and one of the most aggressive sub-types of kidney cancer. It works by specifically binding to carbonic anhydrase IX (CAIX), a validated target protein expressed on 95% of ccRCC cells to produce images with high tumor-to-background ratio and high intra- and inter-reader consistency.

The BLA is based on Telix’s successful global Phase 3 ZIRCON[4] study, which demonstrated a sensitivity of 86%, specificity of 87% and a positive predictive value (PPV) of 93% for ccRCC, including in very small, difficult-to-detect lesions[5]. The results of this study were published in The Lancet Oncology in September 2024, in a peer-reviewed manuscript by Professor Brian Shuch (University of California, Los Angeles, UCLA) and colleagues[6]. The paper outlines the critical unmet need for a new, non-invasive technique that can accurately detect and differentiate ccRCC from other renal masses in patients and concluded that TLX250-CDx meets this need and ‘has the potential to be practice changing.’

Kevin Richardson, Chief Executive Officer, Precision Medicine, said, "We are delighted that the FDA has accepted this BLA as it moves us one step closer to bringing our breakthrough product to patients. We are aiming to revolutionize the management of kidney cancer, just as PSMA-PET/CT[7] scanning has changed the management of prostate cancer. By providing a more definitive clinical diagnosis for renal masses, we believe that Zircaix[2] will help physicians make more timely and confident patient management decisions and more quickly provide patients with a clear understanding of their disease and treatment options. Building further on Telix’s successful urology franchise, we are preparing to bring this powerful precision medicine product to market in 2025[8]."

About TLX250-CDx

TLX250-CDx (Zircaix[2]) is an investigational PET agent that is under development for the diagnosis and characterization of ccRCC. Telix’s pivotal Phase 3 ZIRCON trial evaluating TLX250-CDx in 300 patients, of whom 284 were evaluable, met all primary and secondary endpoints, including showing 86% sensitivity and 87% specificity and a 93% PPV for ccRCC across three independent radiology readers[5]. Telix believes this demonstrated the ability of TLX250-CDx to reliably detect the clear cell phenotype and provide an accurate, non-invasive method for diagnosing and characterizing ccRCC. Confidence intervals exceeded expectations amongst all three readers, showing evidence of high accuracy and consistency of interpretation.

As part of Telix’s commitment to access to medicine, the Company operates an expanded access program (EAP) in the U.S.[9], named patient programs (NPPs) in Europe, and a special access scheme (SAS) in Australia to allow continued access to TLX250-CDx outside of a clinical trial, to patients for whom there are no comparable or satisfactory alternate options. TLX250-CDx has not received a marketing authorization in any jurisdiction and is for investigational use only.