On February 25, 2025 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel oncology therapies that address emerging targets in oncology, reported a strategic reorganization that will extend Ryvu’s cash runway to H2 2026 to focus on driving the RVU120 clinical program and the early pipeline to key data inflection points (Press release, Ryvu Therapeutics, FEB 25, 2025, View Source [SID1234650558]).

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Paweł Przewięźlikowski, Chief Executive Officer and the largest shareholder of Ryvu said:
"We remain focused on advancing our first-in-class clinical blood cancer program RVU120, as well as promising early-stage assets. Considering our cash position, revenue projections, cost structure and the demanding market environment, we decided to optimize expenses so that the company has sufficient cash runway to generate key data for RVU120 and the preclinical pipeline, expecting that our achievements over the next year will support future cash inflows."

Pipeline Focus in 2025-2026

Ryvu continues to pursue its mission of delivering efficacious medicines to cancer patients and, accordingly, will continue to focus on first-in-class and best-in-class novel therapeutics that have the potential to help patients and deliver value to shareholders.

RVU120: emphasis on rapid study enrollment and quality data generation in 2025

Three Phase II studies of RVU120 are in progress as planned: RIVER-81 (combination study with venetoclax in patients with AML), POTAMI-61 (monotherapy/combination study with ruxolitinib in patients with myelofibrosis) and REMARK (monotherapy study in patients with LR-MDS).
The Phase II RIVER-52 study of RVU120 monotherapy in patients with AML or HR-MDS, will not enroll new patients to focus investment on the other RVU120 development paths. Currently enrolled patients will continue to receive treatment per protocol.
The decision to progress RIVER-81 and suspend enrollment in RIVER-52 was based on data analysis and feedback from advisory boards in February 2025.
The next data update for RVU120 is planned in Q2 2025.
RVU305: IND/CTA-enabling studies are ongoing and planned to be completed in H2 2025.

Preclinical discovery and research: Ryvu will pursue a dual-pronged strategy, each of which has the potential to generate multiple oncology medicines.

ONCO Prime – novel small molecule precision medicine: as part of its proprietary ONCO Prime platform, Ryvu will continue to advance several novel precision oncology targets, including synthetic lethality targets. ONCO Prime combines data from patient-derived cells and isogenic cell lines to discover first-in-class oncology targets in defined patient populations. ONCO Prime is supported by a grant of approximately PLN 26 million from the Polish Agency for Enterprise Development.
ADCs (antibody-drug conjugates) with novel payloads: Ryvu will continue to develop ADCs with next-generation novel payloads, including synthetically lethal and immunomodulatory mechanisms. Ryvu will work on novel ADCs internally and through the existing collaboration with Exelixis (STING-based ADCs). The WRN program, which previously was focused on standalone development, will be developed as a novel ADC payload to differentiate on efficacy, resistance profile and safety versus competitors. Ryvu’s ADC research and development will also be supported by grant funding totaling approximately PLN 13 million.
Ryvu continues to advance three key biopharma partnerships; in each of these partnerships, Ryvu is fully reimbursed for its expenses and has the potential to earn multiple financial milestones:

Menarini: On behalf of and in partnership with Menarini, Ryvu is conducting the Phase II JASPIS-01 study of dapolsertib (MEN1703, SEL24) in patients with diffuse large B-cell lymphoma (DLBCL). Dapolsertib is a PIM/FLT3 inhibitor discovered by Ryvu and licensed to Menarini.
BioNTech: BioNTech and Ryvu continue their multi-target research collaboration on undisclosed targets within the small molecule immune-modulatory field.
Exelixis: Exelixis and Ryvu are working collaboratively to advance novel ADCs with STING payloads licensed from Ryvu.
Operational Reorganization and Cash Position

To focus on achieving meaningful catalysts, Ryvu is extending its cash runway from Q1 to H2 2026. As part of the pipeline focus outlined above, Ryvu is reducing its current workforce by approximately 30%. After these reductions, Ryvu will employ approximately 200 colleagues. Ryvu is dedicated to providing affected employees with support during this reorganization. With this cash runway, Ryvu expects to deliver key clinical data on RVU120 and progress in the preclinical pipeline that could potentially support partnering or internal development.
As of February 23, 2025 Ryvu held approximately €46 million (PLN 192 million) in cash and other financial assets. In addition, the company has secured approximately €22 million (PLN 91 million) in non-dilutive grant funding.

Investor Webinar

Ryvu management will host a webinar today, February 25, at 10:15 am CET. The webinar can be accessed at the following link: View Source

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."