BeOne Medicines Receives PRIME Designation from the European Medicines Agency for BGB-16673 in Waldenstrom’s Macroglobulinemia

On July 31, 2025 BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported that the European Medicines Agency (EMA) has granted PRIority MEdicines (PRIME) designation to the Company’s investigational Bruton’s tyrosine kinase (BTK) degrader, BGB-16673, for the treatment of patients with Waldenstrom’s macroglobulinemia (WM) previously treated with a BTK inhibitor (Press release, BeOne Medicines, JUL 31, 2025, View Source [SID1234654690]).

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"This is the Company’s first PRIME designation, marking a milestone for BeOne and providing early and enhanced interaction with the EMA as we advance BGB-16673," said Julie Lepin, Senior Vice President, Chief Regulatory Affairs Officer at BeOne. "PRIME allows us to align early with the EMA on key evidence requirements and potentially accelerate our path to marketing authorization of BGB-16673 for patients with relapsed or refractory Waldenstrom’s macroglobulinemia."

In addition to the PRIME designation, the EMA’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on the EU Orphan Drug Designation (ODD) application for BGB-16673 in WM. A final decision is anticipated in the coming weeks. The U.S. Food and Drug Administration (FDA) also granted Fast Track Designation to BGB-16673 for the treatment of adult patients with relapsed or refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL), and adult patients with R/R mantle cell lymphoma (MCL).

The EMA’s CHMP granted PRIME designation to BGB-16673 based on data demonstrating its novel mechanism and anti-tumor activity in B-cell malignancies. The CHMP recognized the limited treatment options available for WM patients post-BTK inhibitor therapy and acknowledged the strong biological rationale and promising clinical data for BGB-16673 in this setting, thereby demonstrating the potential to address the unmet medical need.

The PRIME initiative, launched by the EMA in 2016, provides early, proactive, and enhanced regulatory support to developers of promising medicines. It is designed to optimize development plans and accelerate evaluation, helping innovative therapies reach patients with unmet medical needs faster.

About BGB-16673

BGB-16673 is an orally available Bruton’s tyrosine kinase (BTK) targeting protein degrader from BeOne’s chimeric degradation activation compound (CDAC) platform. BGB-16673 is designed to promote the degradation, or breakdown, of both wild-type and mutant forms of BTK, including those that commonly result in resistance to BTK inhibitors in patients who experience progressive disease. BGB-16673 is the most advanced BTK protein degrader in the clinic, with an extensive global clinical development program.

Conference on Q1 FY2025 (April 1, 2025 to June 30, 2025) Financial Results

On July 31, 2025 Sumitomo Dainippon Pharma reported Conference on Q1 FY2025 (April 1, 2025 to June 30, 2025) Financial Results.

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(Presentation, Sumitomo Dainippon Pharma, JUL 31, 2025, View Source [SID1234661679])

Quarterly Activities and Cash Flow Report Period Ending 30 June 2025

On July 31, 2025 Imugene Limited (ASX:IMU), a clinical-stage immuno- oncology company, reported its Quarterly Cash Flow report (Appendix 4C) for the quarter ended 30 June 2025 (Press release, Imugene, JUL 31, 2025, https://mcusercontent.com/e38c43331936a9627acb6427c/files/0d0ee6a1-bf9b-f0a7-5414-bc82a36b02ed/IMU_Quarterly_ActivitiesAppendix_4C_Cash_Flow_Report.pdf [SID1234654647]).

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CLINICAL UPDATES

Two Additional Complete Responses and Three Partial Responses in azer-cel CAR T Phase 1b trial

Subsequent to the end of the quarter, Imugene reported further encouraging clinical results from its Phase 1b trial of azer-cel (azercabtagene zapreleucel), an allogeneic "off- the-shelf" CD19-targeting CAR T therapy for patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

Since the Company’s last update in February 2025, five additional patients have been treated in the trial. Of these, two achieved complete responses (CRs), defined as the disappearance of all signs of cancer, and three achieved partial responses (PRs), where tumours reduced in size by at least 50%. This brings the trial’s total best response rate to six complete responses and three partial responses bringing the overall response rate (ORR) to nine out of twelve patients, corresponding to a 75% ORR and a 55% CR rate.

The first patient treated remains cancer-free 15 months after dosing, while others have sustained responses for periods of greater than 2, 5, and 11+ months. The response durability continues to mature as more data accumulates. These patients had previously failed at least three, and in some cases up to six, prior lines of therapy, including autologous CAR T-cell therapies highlighting the potential of azer-cel in a high unmet- need population that has exhausted conventional treatment options.

Unlike currently approved autologous CD19 CAR T cell products, which require patient- specific manufacturing and face logistical limitations, azer-cel is designed as an allogeneic (donor-derived), off-the-shelf therapy that could significantly improve access and treatment timelines. Patients in this study are treated with a combination of lymphodepletion, azer-cel, and interleukin-2 (IL-2), a cytokine known to enhance CAR T- cell function and longevity.

Given the strength of the data and the FDA Fast Track Designation already granted for DLBCL, Imugene intends to meet with the US Food and Drug Administration in Q4 CY25 for a Type B (End of Phase 1) meeting. This interaction will focus on discussing the design of a pivotal or registrational trial that could lead to market approval.

In parallel with these developments, the azer-cel trial will now expand its scope to include other B-cell lymphomas in CAR T-naïve patients (those who have not previously received CAR T therapy). These include rare and underserved cancers such as primary central nervous system lymphoma (PCNSL), chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone lymphoma (MZL), follicular lymphoma (FL), and Waldenström macroglobulinemia (WM). Each of these indications currently suffers from limited treatment options in the relapsed/refractory setting, especially in patients who are ineligible for or unresponsive to existing therapies.

The trial is ongoing across ten sites in the United States, with up to six Australian sites planned.

First dose level cleared in IV combination arm of Phase 1 onCARlytics trial

In April, the Company announced the clearance of the first dose level in the intravenous (IV) combination arm of its Phase 1 onCARlytics clinical trial, known as OASIS. This milestone followed the successful completion of the initial safety observation period and allows the study to progress to the next dose level.

The OASIS trial is a first-in-human study targeting adult patients with advanced or metastatic solid tumours. Its aim is to evaluate both the safety and efficacy of two administration routes; intratumoural (IT) and intravenous (IV), for delivering the onCARlytics therapy. This therapy uses an engineered oncolytic virus (CF33-CD19) to make solid tumours express the CD19 protein, a well-validated target in blood cancers. By enabling CD19 expression in solid tumours, the trial seeks to make them susceptible to treatment with existing CD19-targeted therapies, such as the bispecific monoclonal antibody blinatumomab (Blincyto), which is used in combination with onCARlytics in this trial.

MAST Phase 1 Trial for VAXINIA in Bile Tract Cancer

The Metastatic Advanced Solid Tumours (MAST) trial evaluating CF33-hNIS (VAXINIA) continues to support the potential clinical benefit of our oncolytic virotherapy.

The expansion cohort has completed its first group of three patients without any dose- limiting toxicities, and the cohort remains open. The FDA has granted Orphan Drug Designation (ODD) for VAXINIA in biliary tract cancer, providing a range of regulatory and financial incentives to support ongoing development and potential partnering opportunities.

First Patient Dosed in Australia for PD1-Vaxx Neo-POLEM Phase II trial

In June, the first patient was dosed in Australia at the Queen Elizabeth Hospital in Adelaide as part of the Phase II Neo-POLEM clinical trial investigating its PD1-Vaxx immunotherapy. This investigator-sponsored trial is focused on patients with mismatch repair-deficient or microsatellite instability-high (dMMR/MSI-high) colorectal cancer, a subtype representing approximately 15% of all colorectal cancer cases.

Neo-POLEM is a neoadjuvant study, meaning treatment is administered prior to surgery. The trial is evaluating the potential of PD1-Vaxx, a therapeutic cancer vaccine designed to elicit an immune response against the PD-1 checkpoint protein to improve treatment outcomes in patients with early-stage, resectable disease. The vaccine aims to activate the patient’s immune system to target and reduce tumours before surgery is performed.

The trial is being conducted in collaboration with the Cancer Research UK Southampton Clinical Trials Unit, Royal Surrey Hospital NHS Foundation Trust, and the Australasian Gastro-Intestinal Trial Group (AGITG). Recruitment will span both Australia and the United Kingdom.

The study’s primary objective is to assess major pathological response, specifically tumour reduction post-treatment. Secondary objectives include evaluating safety, identifying biomarkers of immune response, and measuring overall response and survival outcomes.

PATENT PROTECTIONS

US patent issued for onCARlytics

Imugene received a Notice of Allowance from the United States Patent and Trademark Office for its patent application covering the onCARlytics platform. The patent, titled "Oncolytic Virus Expressing a CAR T Cell Target and Uses Thereof," protects both the composition and method of use of the Company’s CD19-expressing oncolytic virus technology. Subsequent to the end of the quarter, Imugene received notice of issue from the USPTO with a patent term extension of 110 days resulting in protection until November 28, 2038.

PD1-Vaxx patent portfolio further strengthened

In June 2025, the PD1-Vaxx cancer vaccine patent portfolio was further strengthened after receiving a Notice of Grant from the US Patent and Trademark Office (USPTO) confirming that US patent application no. 16/966442 has now issued as a patent. The official patent number is 12311019. The patent entitled "Vaccine Composition and Uses Thereof" received a 1110 day patent term extension from the USPTO meaning the patent is in force until February 2042.

Agios Reports Second Quarter 2025 Financial Results and Provides Business Update

On July 31, 2025 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, reported financial results and updates for the second quarter ended June 30, 2025 (Press release, Agios Pharmaceuticals, JUL 31, 2025, View Source [SID1234654665]).

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"With fewer than 40 days to our PDUFA goal date, our commercial team is prepared for the potential U.S. approval of PYRUKYND for thalassemia," said Brian Goff, Chief Executive Officer, Agios. "In the second quarter, we made progress advancing our early- and mid-stage pipeline and remain on track to deliver topline results of the RISE UP Phase 3 trial for PYRUKYND in sickle cell disease by the end of the year. Collectively, our progress reflects our continued focus on delivering innovative medicines with the potential to transform the lives of those affected by rare diseases and deliver long-term shareholder value."

Second Quarter 2025 and Recent Corporate Highlights

Commercial Performance – PYRUKYND (mitapivat)

Generated $12.5 million in net revenue for the second quarter of 2025, compared to $8.6 million in the second quarter of 2024.
248 unique patients completed prescription enrollment forms, representing an increase of 6 percent over the first quarter of 2025.
142 patients are on PYRUKYND therapy, inclusive of new starts and continued therapy, representing an increase of 4 percent over the first quarter of 2025.
Entered into a distribution agreement with Avanzanite Bioscience B.V., a rapidly growing European specialty pharmaceutical company focused on rare diseases, to distribute and commercialize PYRUKYND across the European Economic Area, the United Kingdom and Switzerland.
R&D Highlights

PYRUKYND (mitapivat)
Thalassemia –
Launch preparations underway ahead of U.S. PDUFA goal date of September 7, 2025. The sNDA for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia remains under active review by the U.S. Food and Drug Administration (FDA).
Other regulatory applications remain under review by health authorities in Saudi Arabia, United Arab Emirates, and the European Union.
Sickle Cell Disease –
Topline results from RISE UP Phase 3 trial of mitapivat in sickle cell disease on track by year-end with potential U.S. commercial launch in 2026.
Tebapivat
Sickle Cell Disease –
Dosed the first patient in the Phase 2 trial investigating tebapivat in sickle cell disease. The trial is enrolling across three dose cohorts (2.5mg, 5mg, 7.5mg) and placebo and the primary endpoint will measure hemoglobin response, defined as a ≥1g/dL increase in hemoglobin concentration from week 10 to week 12, compared to baseline.
Lower-risk Myelodysplastic Syndromes (LR-MDS) –
Continue to progress patient enrollment in the Phase 2b trial for tebapivat in LR-MDS with target enrollment completion by the end of 2025.
Early Pipeline
Investigational New Drug (IND) clearance received for AG-236, an siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera (PV).
Presented new data on mitapivat and tebapivat at the 30th European Hematology Association (EHA) (Free EHA Whitepaper) Congress. A total of 14 presentations and publications, led by Agios and external collaborators, were shared, covering sickle cell disease, thalassemia, PK deficiency, and MDS.
Second Quarter 2025 Financial Results

For the quarter ended June 30, 2025, net loss was $112.0 million dollars, compared to a net loss of $96.1 million dollars for the second quarter ended June 30, 2024.

Net product revenue from sales of PYRUKYND for the second quarter of 2025 was $12.5 million, compared to $8.6 million for the second quarter of 2024.
Cost of sales for the second quarter of 2025 was $1.7 million.
Research and Development (R&D) Expenses were $91.9 million for the second quarter of 2025, compared to $77.4 million for the second quarter of 2024. The year-over-year increase was primarily attributed to a $10.0 million regulatory milestone payment to Alnylam associated with our agreement to develop and commercialize AG-236, an siRNA targeting TMPRSS6, intended for the treatment of polycythemia vera.
Selling, General and Administrative (SG&A) Expenses were $45.9 million for the second quarter of 2025 compared to $35.5 million for the second quarter of 2024. The year-over-year increase was primarily attributable to an increase in commercial-related activities, including headcount, as the company prepares for the potential approval of PYRUKYND in thalassemia.
Cash, cash equivalents and marketable securities as of June 30, 2025, were $1.3 billion compared to $1.5 billion as of December 31, 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND launches in thalassemia and sickle cell disease, advance existing programs, and opportunistically expand its pipeline through both internally and externally discovered assets.
Conference Call Information

Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s second quarter 2025 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.

Actinium Presents Data Supporting Paradigm Changing Potential of ATNM-400 in Prostate Cancer Demonstrating Its Superior Efficacy and Improved Survival in Treatment Resistant Tumor Models versus Pluvicto and ARPI Therapy, and Also Enhanced Efficacy in Combination with ARPI Therapy at the 4th Annual Targeted Radiopharmaceuticals Summit

On July 31, 2025 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, reported additional preclinical data supporting its ATNM-400 radiotherapy prostate cancer candidate at the 4th Annual Targeted Radiopharmaceuticals Summit (TRP) being held July 29 – 31, 2025 in San Diego, CA (Press release, Actinium Pharmaceuticals, JUL 31, 2025, View Source [SID1234654691]). ATNM-400 is a novel, first-in-class targeted radiotherapy designed to deliver potent Actinium-225 (Ac-225), an alpha-emitter radioisotope, to prostate cancer cells by targeting a non-Prostate Specific Membrane Antigen (PSMA), disease-driving protein overexpressed in advanced and treatment-resistant disease. Unlike PSMA-targeted agents that primarily serve as imaging and targeting tools, the ATNM-400 target is directly implicated in tumor progression, survival signaling, and resistance to androgen receptor (AR) pathway inhibitor (ARPI) therapy. The presentation titled, "Building a Transformative Ac-225 Portfolio for Next-Generation Precision Oncology" on Wednesday, July 30, 2025, highlighted new PET imaging data showing tumor-specific uptake of ATNM-400, robust tumor control and improved survival outcomes in preclinical studies.

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ATNM-400 Demonstrated Sustained Tumor-Specific Uptake

PET imaging confirmed tumor-specific uptake of the ATNM-400 antibody

The ATNM-400 antibody showed sustained tumor uptake up to 216 hours with rapid clearance from normal tissues
ATNM-400 Produced Robust Tumor Control, Improved Survival and Superior Efficacy After Pluvicto Resistance

ATNM-400 is more efficacious than Pluvicto (Lu-177-PSMA-617) with potent tumor control

ATNM-400 significantly improved survival compared to Pluvicto with continued follow-up ongoing

ATNM-400 is highly effective in prostate cancer tumors with acquired Pluvicto resistance, halting tumor growth and producing potent tumor cell killing after Pluvicto stops working, highlighting its potential in advanced disease settings that are resistant to standard treatments
Enhanced Efficacy of ATNM-400 with Enzalutamide Supports Potential for Novel Combinations

ATNM-400 demonstrated significant in vitro tumor cell killing when used in combination with AR-targeting agents, such as enzalutamide (Xtandi)

The ATNM-400 enzalutamide combination produced superior anti-tumor efficacy and durable tumor control compared to enzalutamide alone with 40% of prostate cancer tumor-bearing animals having complete cures

Actinium previously highlighted that ATNM-400 inhibited tumor growth of enzalutamide resistant tumors whereas re-treatment with Pluvicto or additional enzalutamide did not

Actinium highlighted that follow-up continues to further evaluate the durability of ATNM-400’s anti-tumor efficacy in prostate cancer with additional data expected in the second half of 2025. Actinium’s TRP presentation can be accessed via the Investor Relations page of Actinium’s website HERE.

Sandesh Seth, Actinium’s Chairman and CEO, said, "We are highly encouraged by the growing body of data supporting the therapeutic potential of ATNM-400 and the significance of its target being directly implicated in tumor progression, survival signaling, and resistance to ARPI therapy. The new data presented at TRP highlight the utility of the differentiated mechanism of action of ATNM-400 via the Ac-225 alpha-emitter payload evidenced by durable tumor control, improved survival rates compared to Pluvicto, and efficacy in enzalutamide and Pluvicto resistant models. We believe ATNM-400 has the potential to redefine the treatment paradigm in the high-value, advanced disease, and metastatic castrate-resistant prostate cancer settings, which impact tens of thousands of patients annually. We look forward to presenting additional data in the second half of this year and further highlighting ATNM-400’s differentiated potential in treatment settings with high unmet needs."

About ATNM-400

ATNM-400 is a highly innovative, first-in-class, non-PSMA targeting Actinium-225 (Ac-225) radiotherapy candidate for prostate cancer. In comparison to Pluvicto (Lu-177-PSMA-617) and the majority of radiotherapies in development for prostate cancer, which target prostate specific membrane antigen (PSMA) and are either non-differentiated or barely differentiated, ATNM-400 targets a distinct non-PSMA disease-driving protein overexpressed in advanced and treatment-resistant disease. The receptor specifically targeted by ATNM-400 is highly expressed in metastatic castration-resistant prostate cancer (mCRPC), contributes directly to disease progression, poorer survival outcomes, and continues to be expressed at a high level even after androgen receptor inhibitor (ARPI) and Pluvicto treatment. ATNM-400 leverages the alpha-particle emitter Ac-225, which can cause lethal irreversible double-stranded DNA breaks and is more potent compared to the beta-particle emitter Lutetium-177 (Lu-177) used by Pluvicto, as well as a shorter path length that could result in fewer off-target effects.

Prostate cancer is the most commonly diagnosed cancer in men, with ~1.5 million new cases globally and over 313,000 expected in the U.S. in 2025. While early-stage disease is typically managed with surgery, radiation, and ARPI therapy, up to 20% of cases progress to mCRPC – a lethal stage with limited treatment options. Targeted radiotherapy is a growing field in prostate cancer, dominated by PSMA-targeting agents like Pluvicto, which had sales of over $1.3 billion in 2024, yet many patients either lack PSMA expression or develop resistance to Pluvicto. In the U.S., 40,000–60,000 mCRPC patients annually progress after ARPIs such as Xtandi, which had sales of over $5.9 billion in 2024, highlighting a significant unmet need.