On May 20, 2025 PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported it will present results from the Phase 3 SURPASS-ET clinical trial in an oral session at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place May 30-June 3 in Chicago (Press release, PharmaEssentia, MAY 20, 2025, View Source [SID1234653265]).

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SURPASS-ET (NCT04285086) is evaluating ropeginterferon alfa-2b-njft as a second-line treatment for patients with essential thrombocythemia (ET). Earlier this year, PharmaEssentia announced positive topline Phase 3 results, with ropeginterferon alfa-2b-njft demonstrating a significantly higher durable clinical response rate compared to anagrelide (42.9% vs. 6.0%; p=0.0001), along with a favorable safety profile and a greater reduction in JAK2 V617F allelic burden over 12 months.

Ropeginterferon alfa-2b-njft is currently FDA-approved and marketed as BESREMi for the treatment of adults with polycythemia vera (PV). BESREMi has been recognized by the National Comprehensive Cancer Network (NCCN) as a preferred first-line cytoreductive therapy for adults with symptomatic, low-risk PV and the only preferred therapeutic option for both high-risk and low-risk (symptomatic) patients, regardless of treatment history.

"Current options for patients with ET are limited. Standard therapies like hydroxyurea have notable drawbacks and do not target the underlying biology of the disease, while anagrelide has been associated with toxicity concerns and limited efficacy," said Ruben Mesa, M.D., co-principal investigator, presenting author, and President of Atrium Health Levine Cancer Institute, the largest cancer program in the Carolinas which includes the Comprehensive Cancer Center at Wake Forest Baptist. "This marks the first registrational Phase 3 trial of a long-acting interferon in ET, demonstrating not only well-tolerated blood count control but also a measurable reduction in JAK2 mutation allele burden. These findings support further investigation of ropeginterferon as a second-line option for patients with ET who are seeking additional treatment approaches."

"The ASCO (Free ASCO Whitepaper) meeting is an important opportunity to share detailed findings of our positive data from the SURPASS-ET study with the medical community," said Albert Qin, M.D., Ph.D., Chief Medical Officer of PharmaEssentia USA. "These data highlight a significant advance in the treatment of essential thrombocythemia and reinforce our commitment to delivering innovative, non-chemotherapy options for patients living with myeloproliferative neoplasms."

Presentation Details

Title: Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial
Abstract Number: 6500
Presenter: Dr. Ruben Mesa
Session: Hematologic Malignancies — Leukemia, Myelodysplastic Syndromes, and Allotransplant
Date: Monday, June 2, 2025
Time: 3:00 p.m. – 6:00 p.m. CDT

About Essential Thrombocythemia

Essential thrombocythemia is a chronic, rare blood disorder that is the most common type of myeloproliferative neoplasm. Essential thrombocythemia is most often caused by genetic mutations that cause the bone marrow to produce too many platelets, which can obstruct blood flow and cause a stroke, heart attack or pulmonary embolism.

About BESREMi (ropeginterferon alfa-2b-njft) in polycythemia vera (PV)

BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients.

BESREMi has orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. BESREMi has been approved in more than 40 countries, with approval from the European Medicines Agency (EMA) in 2019, by the US Food and Drug Administration (FDA) in 2021, and by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023. It was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications.

BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders.

Please see full Prescribing Information, including Boxed Warning.

On May 20, 2025 4SC AG ("4SC") (Frankfurt Stock Exchange, Prime Standard: VSC; ISIN: DE000A3E5C40) reported to have received a Negative Opinion on the Company’s Market Authorization Application for Resminostat (Kinselby) for the treatment of patients with advanced stage cutaneous T-cell lymphoma (CTCL) from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) (Press release, 4SC, MAY 20, 2025, https://www.pressetext.com/news/20250523025 [SID1234653364]).

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Following an Oral Explanation on 20 May 2025, and the CHMP’s review of the data on quality, safety and efficacy for Resminostat (Kinselby) in the treatment of patients with advanced stage mycosis fungoides (MF) and Sézary syndrome (SS) that have achieved at least stable disease with at least one prior systemic therapy or Total Skin Electron Beam therapy (TSEB) – the CHMP considers by consensus that the efficacy of the above-mentioned medicinal product is not sufficiently demonstrated, and therefore, recommends the refusal of the granting of the marketing authorization for Resminostat (Kinselby).

As already communicated on 20 May 2025, 4SC will thus cease all efforts to develop and commercialize Resminostat (Kinselby). Currently, 4SC has at least a 12-month cash runway to finance the Company’s projected expenses as the Management and Supervisory Boards consider all options for the Company, including liquidation.

Jason Loveridge, Ph.D., CEO of 4SC, commented: "We had hoped that the long wait for a maintenance treatment to help manage CTCL would soon be over. We are therefore deeply disappointed with this outcome as it is a further setback for people living with this rare and incurable disease."

On May 20, 2025 Promontory Therapeutics Inc., a clinical stage pharmaceutical company advancing small molecule oncology approaches with immunotherapeutic effects, reported a successful in-person End of Phase 2 (EOP2) Meeting with the U.S. Food and Drug Administration (FDA), allowing the company to prepare and submit a registrational Phase 3 study of PT-112 monotherapy in patients with metastatic castration-resistant prostate cancer (mCRPC) (Press release, Promontory Therapeutics, MAY 20, 2025, View Source [SID1234653250]).

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Consistent with FDA Project Optimus, the FDA agreed with the proposed dosing regimen for use in a prospective, randomized controlled Phase 3 registrational study, on the basis of the completed Phase 2 clinical trial. The study design and endpoints, along with the proposed comparator and statistical framework, were agreed in principle, as were Promontory’s proposed patient population within mCRPC, and proposal for an interim analysis. The latter would allow a potential pathway for drug approval prior to the full completion of the Phase 3 study.

Promontory will now prepare its full Phase 3 study submission for final review on the basis of FDA’s guidance, and conduct similar meetings with international regulatory authorities.

The proof of concept and dose optimization Phase 2 study of PT-112 monotherapy in advanced mCRPC was conducted in the U.S. and France and included three randomized dosing arms of PT-112. Immune response biomarker data were recently presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2025 Annual Meeting, and preliminary clinical outcomes will be presented on June 2nd at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 Annual Meeting.

For more information about Promontory Therapeutics and PT-112 visit www.PromontoryTx.com.

On May 20, 2025 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported the launch of the Guardant Hereditary Cancer test, a germline test that identifies genetic variants associated with cancer risk to help cancer care teams provide optimal patient care (Press release, Guardant Health, MAY 20, 2025, View Source [SID1234653266]).

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Germline genetic testing is recommended by medical practice guidelines for patients diagnosed with cancer to enable genetically targeted treatment and identify relatives who may benefit from personalized cancer screening and prevention.1 The testing analyzes inherited genetic variants, typically present in all the cells of the body, that may predispose an individual to certain risks or diagnoses, including hereditary cancers and other genetic conditions. The new Guardant Hereditary Cancer test is a blood-based germline panel test that identifies guideline-recommended genetic variants across 82 genes associated with an increased risk for more than 12 tumor types, including breast, colorectal, prostate, endometrial, renal and others.

"Introducing a best-in-class hereditary cancer test is another important step in achieving our mission to conquer cancer with data," said Helmy Eltoukhy, Guardant Health chairman and co-CEO. "It expands the portfolio we offer to support healthcare providers across the entire continuum of cancer care and allows them to access an even broader set of precision oncology tools through a single source. The germline testing will help them develop more informed personalized treatment plans for patients, reduce risk and improve outcomes."

Physicians may order the Guardant Hereditary Cancer test for patients with a personal or family history of hereditary cancer for several reasons, such as guiding treatment or care decisions, assessing risk for secondary cancer development, and providing information that can help identify other family members who may be at increased risk for certain cancers. Discussion of risk-reducing strategies, enhanced screening, and referral to a specialist or genetic counseling is recommended.

The Guardant Hereditary Cancer test requires only a simple blood draw and can be ordered as a standalone test or added to Guardant360 liquid biopsy tests with no additional sample required. Results are available in two to three weeks.

On May 20, 2025 Clarity Pharmaceuticals (ASX: CU6) ("Clarity" or "Company"), a clinical-stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for patients with cancer, reported that it has commenced its second registrational Phase III 64Cu-SAR-bisPSMA diagnostic trial in prostate cancer, AMPLIFY (NCT06970847)1, with the initiation of the first clinical site at XCancer in Omaha, NE (Press release, Clarity Pharmaceuticals, MAY 20, 2025, View Source [SID1234653232]).

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AMPLIFY is a study of 64Cu-SAR-bisPSMA Positron Emission Tomography: A Phase 3 Study of Participants with Biochemical Recurrence of Prostate Cancer. It is a non-randomised, single-arm, open-label, multi-centre, diagnostic clinical trial of 64Cu-SAR-bisPSMA positron emission tomography (PET) in participants with rising or detectable prostate-specific antigen (PSA) after initial definitive treatment.

The aim of this Phase III trial is to investigate the ability of 64Cu-SAR-bisPSMA PET/computed tomography (CT) to detect recurrence of prostate cancer. Evaluation will be across 2 imaging timepoints, Day 1 (day of administration, same-day imaging) and Day 2 (approximately 24 hours post administration, next-day imaging).

The study will enroll approximately 220 participants at multiple clinical sites across the United States (US) and Australia, and the first participant is expected to be imaged in the coming weeks. As a pivotal trial, the final study results are intended to provide sufficient evidence to support an application to the US Food and Drug Administration (FDA) for approval of 64Cu-SAR-bisPSMA as a new diagnostic imaging agent in biochemical recurrence (BCR) of prostate cancer.

Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "We are very excited to commence our second registrational Phase III trial with the optimised SAR-bisPSMA agent in patients that are experiencing the return of their disease following treatment of the primary cancer.

"The recent devastating news that former US President, Joe Biden, has been diagnosed with aggressive prostate cancer that has metastasised to the bone is yet another reminder that no man is safe from this insidious disease, and he joins over 3.3 million men in the US who live with prostate cancer today2. Australia is also not immune. A recent public announcement revealed that MP Barnaby Joyce has recently been diagnosed with prostate cancer, and he joins over 250,000 Australian men who are living with prostate cancer today3. These large numbers highlight the need for more timely and accurate diagnosis to safely and effectively treat the cancer with suitable therapies."

The AMPLIFY trial is based on favourable preclinical and clinical data generated to date, including the Phase I/II COBRA trial in patients with BCR of prostate cancer and the Phase I PROPELLER trial in patients with confirmed prostate cancer pre-prostatectomy4,5. These earlier studies demonstrated an excellent safety profile and exciting efficacy results, especially in comparison to current standard of care imaging. PROPELLER showed improved diagnostic performance of 64Cu-SAR-bisPSMA compared to 68Ga-PSMA-11 on same-day imaging, including higher number of lesions identified and 2-3 times higher uptake and tumour-to-background ratio, favouring 64Cu-SAR-bisPSMA. The COBRA trial showed that more lesions and more patients with a positive scan were identified on 64Cu-SAR-bisPSMA PET compared to conventional scans and on next-day vs. same-day imaging. 64Cu-SAR-bisPSMA also allowed for the identification of lesions in the 2-mm range. The most recent findings from the COBRA trial demonstrated that 64Cu-SAR-bisPSMA was able to detect lesions from 29 days to more than 6 months earlier than standard-of-care (SOC) prostate-specific membrane antigen (PSMA) PET agents.

"These compelling findings, along with preliminary theranostic SECuRE trial data6, laid the foundation for the receipt of three Fast Track Designations (FTD) for SAR-bisPSMA from the US FDA within six months7-9, highlighting how impressive our science and clinical development are, the significance of the data so far and the high unmet need for better therapies and diagnostics in prostate cancer. We believe 64Cu-SAR-bisPSMA could become a best-in-class product and look forward to progressing its clinical development through several trials in prostate cancer: AMPLIFY in BCR, our first Phase III CLARIFY study in pre-prostatectomy patients, the investigator-initiated trial led by Prof Louise Emmett, Co-PSMA (head-to-head study comparing the diagnostic performance of 64Cu-SAR-bisPSMA vs. SOC 68Ga-PSMA-11 in BCR), and the theranostic SECuRE study in metastatic castration-resistant prostate cancer (mCRPC). The FTDs will enable us to accelerate the development of this comprehensive program to be used in patients with prostate cancer throughout the management of their cancer, from initial to late-stage disease, with an opportunity to completely change the entire treatment landscape for the large prostate cancer market.

"We have received a lot of interest in the AMPLIFY trial and look forward to seeing this study progress in such an important patient population. Knowing whether or not their prostate cancer has returned following initial treatment and where the cancer lesions are located are essential for the appropriate treatment of BCR patients. Identifying lesions as early as possible can arm clinicians with important information to help determine the right treatment regimen and ensure positive long-term treatment outcomes. We would like to thank all clinicians and patients who participate in our clinical trials and who trust us in delivering on our promise of developing best-in-class products. Our team and collaborators look forward to bringing this next-generation PSMA diagnostic to prostate cancer patients around the world," said Dr Taylor.

About SAR-bisPSMA
SAR-bisPSMA derives its name from the word "bis", which reflects a novel approach of connecting two PSMA-targeting agents to Clarity’s proprietary sarcophagine (SAR) technology that securely holds copper isotopes inside a cage-like structure, called a chelator. Unlike other commercially available chelators, the SAR technology prevents copper leakage into the body. SAR-bisPSMA is a Targeted Copper Theranostic (TCT) that can be used with isotopes of copper-64 (Cu-64 or 64Cu) for imaging and copper-67 (Cu-67 or 67Cu) for therapy.