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Iksuda Therapeutics Receives FDA IND Clearance for IKS014

On June 30, 2025 Iksuda Therapeutics (Iksuda), the developer of class-leading antibody drug conjugates (ADCs) with clinically validated tumour-selective payload release formats, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for IKS014, a human epidermal growth factor receptor 2 (HER2) ADC targeting patients with advanced HER2+ solid tumours, enabling the expansion of ongoing clinical trials (Press release, Iksuda Therapeutics, JUN 30, 2025, View Source [SID1234654184]).

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IKS014 is currently being investigated in an open-label phase 1 dose-escalation study (View Source) designed to evaluate the safety and tolerability of increasing dose levels of IKS014 and establish a recommended phase 2 dose. Preliminary data from this study has demonstrated promising clinical activity across multiple tumour types, including breast, ovarian, gallbladder, and oesophageal cancers, and including patients who have relapsed after prior treatment with Enhertu. Gaining access to U.S. centres for the dose expansion stage of this phase 1 trial will enable efficient confirmation of the role of IKS014 in this important patient sub-set.

Dr Dave Simpson, Chief Executive Officer, Iksuda Therapeutics, said:

"The FDA clearance of our IND application for IKS014 represents a significant milestone in our mission to address the critical unmet needs of patients with HER2-positive cancers, particularly those who have exhausted current standard-of-care options. Early clinical data has been extremely encouraging, and we are excited to expand our program to reach more patients.

"The early efficacy signals that have been observed, particularly in patients that have relapsed after receiving prior treatments of Kadcyla and Enhertu, suggest IKS014 could potentially offer a new treatment option for patients who currently have limited alternatives. We look forward to working with our clinical partners to further evaluate the potential of IKS014."

The first stage of this phase 1 trial, to determine the recommended phase 2 dose for dose expansion, is nearing completion. The expansion phase will assess several patient cohorts including those with HER2-positive breast cancer who are refractory to or cannot tolerate Enhertu, patients with HER2-low breast cancer and patients with HER2-positive positive gastric cancer. The IND clearance will allow Iksuda to access patients across sites in the United States, alongside sites in Australia, New Zealand, and Singapore. The phase 1 trial is expected to complete in 2H 2026.

About IKS014

IKS014 is a potential best-in-class antibody drug conjugate, benefiting from tumour selective activation and release of the cytotoxic agent monomethyl auristatin F (MMAF). In preclinical trials, it displayed impressive activity in high- and low-HER2 expressing tumours with a favourable Therapeutic Index vs other HER2-directed drugs. Iksuda gained exclusive world-wide rights (excluding Greater China and South Korea) to IKS014 from LigaChem Biosciences (View Source).

OS Therapies Presents Statistically Significantly Positive 1-Year Event Free Survival, Overall Survival and Safety Clinical Data Updates for OST-HER2 at the MIB Agents Factor Osteosarcoma Conference

On June 30, 2025 OS Therapies (NYSE-A: OSTX) ("OS Therapies" or "the Company"), a clinical-stage immunotherapy and Antibody Drug Conjugate (ADC) biopharmaceutical company, reported positive 1-year event free survival (EFS), overall survival and safety clinical trial data updates from the 40 patient treatment arm of its Phase 2b clinical trial of immunotherapy candidate OST-HER2 in the prevention or delay of recurrence in fully resected, lung metastatic osteosarcoma (Press release, OS Therapies, JUN 30, 2025, View Source [SID1234654183]). In data presented by Principal Investigator Dr. Damon Reed at the MIB Factor Osteosarcoma Conference held in Salt Lake City, Utah on Saturday June 28, 2025 the following data updates were released:

35% (14 out of 40) of OST-HER2-treated patients achieved 1-year event free survival (EFS) compared with 20% of patients from peer-reviewed publication selected by Children’s Oncology Group (COG)1 as equivalent to osteosarcoma patient subpopulation enrolled (p = 0.0194);
A total of 13 out of 40 patients were reported to have experienced severe adverse events (SAEs) during the trial, of which 7 patients’ adverse events were treatment-associated adverse events (TSAEs). All of the 7 patients’ TSAEs were grade 3 TSAEs; 0 patients experienced grade 4 or grade 5 TSAEs. None of the patients for whom TSAEs were reported discontinued the study.
"The updated OST-HER2 data presented at MIB Factor that showed EFS data statistically significantly favoring OST-HER2 treated patients when compared with the leading peer-reviewed publications on historical event free survival outcomes in this subset of the pulmonary metastatic osteosarcoma patient population," said Dr. Robert Petit, Chief Medical & Scientific Officer of OS Therapies. "The favorable safety profile of OST-HER2 compared with standard of care is also an important quality of life factor when assessing potential new treatment options for this difficult to treat patient population."

Additionally, the Company reported a regulatory update regarding the submission of preliminary external control data and comprehensive plans for the finalization of this data package to the US Food & Drug Administration’s ("FDA"). In accordance with the provisions of prevailing Guidance to Industry – including ICH E10 Choice of Control Group in Clinical Trials; Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products 2019; and Rare Diseases – Natural History Studies for Drug Development 2019 – to support a Biologics Licensing Application (BLA) marketing authorization under the US Food & Drug Administration’s ("FDA") Accelerated Approval Program ("Accelerated Approval").

Based on guidance from FDA from its recent Type D Meeting and the submission of subsequent responses to FDA arising from the Meeting, the Company is providing updated superiority peer-reviewed historical external control data, matched external control data and/or real-world external control data. This data, individually or collectively at FDA’s discretion, may be sufficient for FDA to support a marketing authorization in a setting where randomization may not be feasible. Such an approach is common in rare diseases – such as Prevention or Delay of Recurrence in Fully Resected, Lung Metastatic Osteosarcoma – for which the company holds US FDA Orphan Disease Designation (ODD), Fast Track and Rare Pediatric Disease Designation (RPDD).

The Company has submitted clarifying information to FDA Type D Meeting feedback requests regarding proposed statistical methods to be used in FDA’s assessment of external control arm(s) suitable to support Accelerated Approval. The Company is awaiting response from FDA to its End of Phase 2 meeting request where, if granted, the Company will seek full alignment on the individual and/or collective data sets necessary to support Accelerated Approval. The Company has collected multiple sources of potential case matched external control and real-world data potentially suitable, individually or together with historical external control data at FDA’s discretion, to support Accelerated Approval.

"The feedback received from FDA regarding the use of external control comparators in settings where placebo-controlled randomization trials are not feasible increases the avenues available for OST-HER to gain Accelerated Approval," said Paul Romness, Chairman & CEO of OS Therapies. "We have now responded to the follow-up questions from our recent positive Type D Meeting with FDA positioning us to soon be granted an End of Phase 2 meeting. We were also very pleased with the reception the presentation received from the osteosarcoma community."

Mr. Romness continued: "Recent interactions we have had with FDA are consistent with public statements from FDA leadership prioritizing the safety profile of potential new products under consideration for Accelerated Approval that are intended to treat deadly rare diseases where randomized trials may not be feasible, especially in pediatric cancer where no alternative treatment options are approved."

Concurrent with this announcement, the United Kingdom’s (UK) Medicines and Healthcare products Regulatory Agency (MHRA) has agreed to support the Company in utilizing its Clinical Practice Research Datalink (CRPD) to assist the Company in developing data necessary to support potential worldwide marketing authorizations – including in the UK, US and EU – for OST-HER2. The CPRD collects anonymized patient data from a network of healthcare practitioners and institutions across the UK. Primary care data are linked to a range of other health related data to provide a longitudinal, representative population health dataset. The data encompass 60 million patients, including 18 million currently registered patients. Follow-up from the Company’s July 31st, 2025 Scientific Advice Meeting with MHRA is expected by mid-August 2025. (View Source)

OST-HER2 has received Rare Pediatric Disease Designation (RPDD) for osteosarcoma from the US FDA. If the Company receives Accelerated Approval prior to September 30, 2026, it will become eligible to receive a Priority Review Voucher (PRV) that it intends to sell. The most recent PRV sale, valued at $160 million, occurred in June 2025.

OST-HER2, an immunotherapy for osteosarcoma that uses a HER2-bioengineered form of the bacterium Listeria monocytogenes to trigger a strong immune response against HER2-expressing cancer cells, is featured in the movie Shelter Me: The Cancer Pioneers. The movie offers a look into canine comparative oncology, a field that compares treatment of cancers in dogs to those in people and covers developing treatments for rare forms of cancer. The movie is available via streaming on PBS’ website.

The most recent data updated regarding the OST-HER2 canine osteosarcoma program is available at this link.

NeoGenomics Announces PanTracer Tissue and PanTracer Tissue + HRD Now Available to Support More Informed and Timely Cancer Care

On June 30, 2025 NeoGenomics, Inc. (NASDAQ: NEO), a leading provider of oncology diagnostic solutions that enable precision medicine, reported the launch of PanTracer Tissue, a next-generation solid tumor profiling assay, including the option to add testing for homologous recombination deficiency (HRD) (Press release, NeoGenomics Laboratories, JUN 30, 2025, View Source [SID1234654182]). These new options can provide faster, actionable insights to help physicians navigate complex treatment decisions more confidently.

PanTracer Tissue evaluates over 500 cancer-related genes and aligns with clinical guidelines, covering key biomarkers recommended for therapy selection and additional genomic insights for clinical trial enrollment. Results may be delivered in as little as 8 days, enabling physicians to rapidly initiate treatment strategies. Minimal specimen requirements make it suitable for a wide range of tumor types and practice settings. The assay builds on the company’s tissue-based CGP platform, previously known as NeoComprehensive Solid Tumor.

The addition of PanTracer Tissue + HRD offers enhanced tumor profiling by incorporating homologous recombination deficiency analysis into a single, guideline-aligned test for ovarian cancer. The PanTracer Tissue + HRD offering includes BRCA mutation status and a genomic instability score—critical biomarkers that can help guide the use of PARP inhibitors and other therapies targeting DNA repair pathways. The combined approach streamlines ordering and can shorten time to actionable results by capturing a broader range of clinically relevant genomic alterations, gene fusions, and DNA repair deficiencies.

"With PanTracer Tissue and PanTracer Tissue + HRD we’re unlocking a more complete genomic view from a single sample, giving physicians clearer answers, sooner," said Warren Stone, President and Chief Operating Officer at NeoGenomics. "With the addition of PanTracer Tissue + HRD, we are expanding our portfolio to address the unmet need for ovarian cancer therapy selection with the objective of improving patient care."

PanTracer Tissue and PanTracer Tissue + HRD are part of NeoGenomics’ broader portfolio of precision oncology solutions designed to improve patient outcomes through high-quality, guideline-aligned testing. Revealed at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, the continually expanding PanTracer portfolio reflects the company’s dedicated focus on advancing diagnostic tools that support more informed, personalized care.

Zai Lab Announces Positive Topline Phase 3 Results for Bemarituzumab in Fibroblast Growth Factor Receptor 2b (FGFR2b) Positive First-Line Gastric Cancer

On June 30, 2025 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported the Phase 3 FORTITUDE-101 clinical trial evaluating first-line bemarituzumab plus chemotherapy (mFOLFOX6) met its primary endpoint of overall survival (OS) at a pre-specified interim analysis (Press release, Zai Laboratory, JUN 30, 2025, View Source [SID1234654181]).

Bemarituzumab plus chemotherapy demonstrated a statistically significant and clinically meaningful improvement in OS as compared to placebo plus chemotherapy in people living with unresectable locally advanced or metastatic gastric or gastroesophageal junction (G/GEJ) cancer with FGFR2b overexpression and who are non-HER2 positive. FGFR2b overexpression was defined as 2+/3+ staining in ≥10% of tumor cells by centrally performed immunohistochemistry (IHC) testing.

Gastric cancer is the fifth leading cause of cancer-related death worldwide, with nearly one million new cases and over 650,000 deaths globally each year 1, highlighting a critical unmet medical need. In China, there are over 350,000 new cases each year. The disease is associated with a poor prognosis, particularly in advanced stages where the five-year survival rate is less than 10%. There are currently no approved therapies specifically targeting FGFR2b overexpression in gastric cancer in China.

"Bemarituzumab is the first FGFR2b inhibitor to demonstrate a statistically and clinically significant overall survival benefit in a randomized Phase 3 trial for the first-line treatment of FGFR2b-positive gastric cancer," said Dr. Rafael Amado, M.D., President, Head of Global Research and Development at Zai Lab. "The success of the global Phase 3 FORTITUDE-101 study highlights the potential of bemarituzumab to redefine the standard of care for a patient population that has faced poor outcomes with existing therapies. We are proud to have contributed meaningfully to this pivotal trial, including a substantial number of patients enrolled in China. Based on these results, and the regulatory Breakthrough Designation, we plan to move rapidly toward regulatory submission in China to bring this transformative therapy to patients as quickly as possible."

The most common treatment-emergent adverse events (>25%) in patients treated with bemarituzumab plus chemotherapy were reduced visual acuity, punctate keratitis, anaemia, neutropenia, nausea, corneal epithelium defect and dry eye. While ocular events were consistent with the Phase 2 experience and observed in both arms, they occurred with greater frequency and severity in the Phase 3 bemarituzumab arm.

Detailed results from the trial will be shared at a future medical meeting.

Zai Lab holds the development and commercialization rights for bemarituzumab for mainland China, Hong Kong, Macau, and Taiwan. Bemarituzumab has been granted Breakthrough Therapy designation by the China Center for Drug Evaluation of the National Medical Products Administration for the treatment of FGFR2b-positive gastric and gastroesophageal junction adenocarcinoma.

A Phase 3 study of bemarituzumab plus chemotherapy and nivolumab is also ongoing in patients with first-line gastric cancer, with data readout anticipated in H2 2025.

About FGFR2b

The FGFR2b protein (also known as fibroblast growth factor receptor 2b) is an emerging biomarker which, when overexpressed, promotes aberrant signaling leading to tumor cell proliferation.2

The FGFR2b protein is overexpressed by G/GEJ tumor cells in approximately 38% of patients with advanced G/GEJ cancer. FGFR2b protein overexpression is defined as 2+/3+ staining intensity on tumor cell membrane, as detected by immunohistochemistry (IHC) testing. In approximately 16% of patients with advanced G/GEJ cancer, FGFR2b protein overexpression is observed on ≥10% of tumor cells by IHC.3

About FORTITUDE-101

FORTITUDE-101 is a randomized, multi-center, double-blind, placebo-controlled Phase 3 study of bemarituzumab plus mFOLFOX6 versus placebo plus mFOLFOX6 as first-line therapy in advanced G/GEJ cancer with FGFR2b overexpression. The FORTITUDE-101 trial spanned 300 sites across 37 countries, with 547 patients enrolled.

The primary outcome measure of the trial is overall survival in patients with FGFR2b ≥10% 2+/3+ tumor cell staining. Key secondary outcome measures include progression-free survival and overall response rate. Candidates were excluded from the trial if they were known to be human epidermal growth factor receptor 2 (HER2) positive. FORTITUDE-101 included more comprehensive ocular-related monitoring than previous studies of bemarituzumab.

About Gastric Cancer in China

Gastric cancer is the fifth most common cancer worldwide, while China bears one of the highest gastric cancer burdens in the world with an estimated 358,700 new cases and 260,400 deaths annually.4 In China, approximately 80% of gastric cancer patients are diagnosed at an advanced or metastatic stage5. For those diagnosed with Stage IV gastric cancer, the overall 5-year survival rate is less than 10%6. Patients with advanced gastric and GEJ cancer that overexpress the FGFR2b protein may be associated with poor prognosis.

Nektar Therapeutics Announces Proposed Public Offering

On June 30, 2025 Nektar Therapeutics (Nasdaq: NKTR), a clinical-stage biotechnology company focused on the development of innovative medicines in the field of immunotherapy, reported that it has commenced an underwritten public offering of shares of its common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase shares of its common stock (Press release, Nektar Therapeutics, JUN 30, 2025, View Source [SID1234654180]). All of the shares of common stock and pre-funded warrants to be sold in this offering are being offered by Nektar. In addition, Nektar intends to grant the underwriters a 30-day option to purchase up to an additional fifteen percent (15%) of shares of its common stock at the public offering price per share, less underwriting discounts and commissions. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

Nektar intends to use the net proceeds from the offering for general corporate purposes, which may include research and development, clinical development and manufacturing costs to support the advancement of its drug candidates, as well as other general corporate purposes.

Jefferies and Piper Sandler are acting as joint bookrunning managers for the offering.

The securities described above are being offered pursuant to a shelf registration statement on Form S-3 (No. 333-286222) that was filed with the U.S. Securities and Exchange Commission (the "SEC") on March 28, 2025 and declared effective on April 1, 2025. This offering is being made only by means of a prospectus supplement and an accompanying prospectus that form a part of the registration statement.

A preliminary prospectus supplement related to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website located at www.sec.gov. Copies of the preliminary prospectus supplement and an accompanying prospectus related to the offering may also be obtained, when available, from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at (877) 821-7388, or by email at [email protected]; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, or by telephone at (800) 747-3924, or by e-mail at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that state or jurisdiction.