On December 16, 2025 FivepHusion, an advanced clinical-stage biotechnology company, reported that Dr Marion Mateos, Co-Principal Investigator of the phase 1/2 Deflexifol at Relapse Trial (DART) proudly supported by the Kids with Cancer Foundation, recently presented a poster of the phase 1 (Part A) trial results at the Society of Neuro-Oncology (SNO) 2025 Annual Meeting in Honolulu, Hawaii, USA, which was held from the 19th – 23rd November.
The poster can be found on the FivepHusion website here.

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Key outcomes of the phase 1 trial are:

Nine participants (aged 4-14 years) with ependymoma (n=6), diffuse midline glioma (DMG) (n-2) and embryonal tumour with multilayered rosettes (n=1) were enrolled
Deflexifol is well tolerated in children, with a side effect profile consistent with adults
Deflexifol pharmacokinetic properties are similar to those reported previously in adults, and for 5-FU in children
The Deflexifol paediatric maximum tolerated dose (MTD) was defined as 525mg/m2 bolus (of delivered 5-FU) followed by a 3000mg/m2 infusion. These results reflect:
~25% greater than typical 5-FU clinical dosing in adults
~9x the previously reported 5-FU MTD in children
The Recommended Phase 2 Dose for future paediatric studies was declared as the MTD
The DART phase 2 expansion cohort (DART Part B) evaluating the activity of Deflexifol in children with ependymoma will commence soon
The phase 1/2 DART study is an Australian investigator-initiated trial, led by Principal Investigators Professor David Ziegler and Dr Marion Mateos and sponsored by the Australian and New Zealand Children’s Haematology / Oncology Group (ANZCHOG) in collaboration with FivepHusion. The trial is designed to investigate Deflexifol monotherapy as a treatment for paediatric ependymoma and other childhood brain cancers. All major paediatric oncology centres in Australia are participating in the trial, and major trial funding has been provided by the Kids with Cancer Foundation, through Sydney Children’s Hospitals Foundation, and the Robert Connor Dawes Foundation.

Dr Christian Toouli, CEO and Managing Director of FivepHusion commented, "Presentation of the DART Phase 1 results at SNO 2025 was an excellent opportunity to raise international awareness of Deflexifol and its potential use in the treatment of paediatric ependymoma and other brain cancers. Congratulations and thanks to Dr Mateos for presenting the DART study results at such a prestigious international congress."

Deflexifol is an advanced clinical-stage, next-generation co-formulation of 5-FU and leucovorin (LV), a drug that significantly enhances 5-FU activity. Deflexifol has been previously evaluated in two successfully completed clinical trials in adults with a variety of solid tumours; the DART study is the first clinical evaluation of Deflexifol in paediatric patients. FivepHusion is harnessing the proven cytotoxic activity of 5-FU together with the unique, optimised attributes of the Deflexifol co-formulation to pursue Deflexifol development in a range of strategic solid tumour indications presenting with significant unmet medical needs, including paediatric ependymoma.

Ependymomas are rare central nervous system tumours (annual incidence of ~4 patients per million) that are more common in young children 0-4 years of age. The current standard treatment for ependymoma is surgery and radiotherapy, though relapse occurs in one third of all paediatric patients and is associated with a poor prognosis. Currently, there are no therapeutic drugs approved for the treatment of ependymoma, presenting a significant unmet medical need for the development of safe and efficacious new treatments for this disease.

Previously, 5-FU has been reported as a promising drug candidate for the treatment of paediatric ependymoma by independent research groups1,2, and in a clinical trial conducted at the St Jude Children’s Research Hospital (Memphis, Tennessee, USA)3. Recently, independent studies have gained further insights into understanding the susceptibility of paediatric ependymoma to 5-FU4. Research by FivepHusion collaborators indicates that Deflexifol, as an optimised co-formulation of 5-FU and LV, may be efficacious against paediatric ependymoma and other brain cancers. Due to its improved safety, tolerability, and potentially superior anti-tumour efficacy, Deflexifol offers the exciting opportunity of addressing the limitations of current 5-FU formulations to enable development as potentially the first approved drug for ependymoma and possibly other brain tumours.

(Press release, FivepHusion, DEC 16, 2025, View Source [SID1234661451])

On December 16, 2025 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a late-stage immunotherapy company targeting cancer and autoimmune diseases, reported strong operational progress in the TACTI-004 (KEYNOTE-F91) Phase III trial evaluating eftilagimod alfa (efti) in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy, KEYTRUDA (pembrolizumab), and chemotherapy as first line therapy for advanced/metastatic non-small cell lung cancer.

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The registrational TACTI-004 trial has enrolled 289 patients (over 38% of the trial’s targeted enrolment of 756 patients), and enrolment continues at a robust pace. Additionally, the number of activated clinical sites now exceeds 120 and 27 countries have received full regulatory approvals.

This includes the United States where the first of multiple clinical sites has received full regulatory clearance following the recent completion of the FDA’s Project Optimus initiative and subsequent receipt of local and central Institutional Review Board (IRB) approvals.

As announced on 9 October 2025, TACTI-004 had enrolled the necessary 170 patients to conduct the futility analysis that remains on track for the first quarter of CY2026. Furthermore, Immutep expects to complete patient enrolment in the third quarter of CY2026.

Immutep Chief Executive Officer, Marc Voigt, said, "We are very pleased with the strong operational progress of TACTI-004 globally and the robust pace of recruitment. Growing interest in this pivotal trial has been enhanced by the recent licensing deal for efti in emerging markets with Dr Reddy’s. The Immutep team is excited about further delivering on key milestones ahead, including the futility analysis and completion of patient enrolment."

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About TACTI-004
TACTI-004 (Two ACTive Immunotherapies) is a randomised, double-blind, controlled Phase III study evaluating eftilagimod alfa (efti), a first-in-class MHC Class II agonist, in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy, KEYTRUDA (pembrolizumab), and chemotherapy as first line therapy for patients with advanced or metastatic non-small cell lung cancer with no EGFR, ALK or ROS1 genomic tumour aberrations. The global trial will enrol approximately 756 patients regardless of PD-L1 expression and with non-squamous or squamous tumours at over 150 clinical sites in over 25 countries. Patients will be randomised 1:1 to receive either efti in combination with pembrolizumab and chemotherapy in the treatment arm or pembrolizumab in combination with chemotherapy and placebo in the control arm. The study’s dual primary endpoints are progression-free survival and overall survival.

About Eftilagimod Alfa (Efti)
Efti is a novel immunotherapy that directly activates antigen-presenting cells or APCs (e.g. dendritic cells, monocytes) via the MHC Class II pathway to fight cancer. As an MHC Class II agonist, its activation of APCs engages the adaptive and innate immune system to initiate a broad anti-cancer immune response. This includes priming and activating cytotoxic T cells as well as generating important co-stimulatory signals & cytokines that further boost the immune system’s ability to combat cancer.

Efti is under evaluation for a variety of solid tumours including non-small cell lung cancer (NSCLC) in a pivotal Phase III trial called TACTI-004 (KEYNOTE-F91), as well as head and neck squamous cell carcinoma (HNSCC), soft tissue sarcoma, and breast cancer. Its favourable safety profile enables various combinations like with anti-PD-[L]1 immunotherapy, radiotherapy, and/or chemotherapy. Efti has received Fast Track designation in first line HNSCC and in first line NSCLC from the United States Food and Drug Administration (FDA).

(Press release, Immutep, DEC 16, 2025, https://www.immutep.com/immutep-announces-strong-operational-progress-in-global-tacti-004-keynote-f91-phase-iii-and-enrolment-continues-at-robust-pace/ [SID1234661432])

On December 16, 2025 Bicycle Therapeutics plc (NASDAQ: BCYC), a pharmaceutical company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported it has entered into a 15-year contract including an option to renew with the UK Nuclear Decommissioning Authority (NDA) for access to up to 400 tonnes of reprocessed uranium (RepU). RepU continually regenerates providing a potentially sustainable supply of 212Pb. Bicycle intends to utilize the RepU provided by this agreement in its development of potential lifesaving therapies.

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In addition, Bicycle announced a collaboration with United Kingdom National Nuclear Laboratory (UKNNL), pursuant to which it plans to extract 228Th from the RepU obtained from NDA. The extracted 228Th will then be further processed into 224Ra and loaded into a bespoke 212Pb generator being developed exclusively for Bicycle by SpectronRx. 212Pb is a radioisotope and one of the more potent therapeutic payloads against cancer cells known as Targeted Alpha Therapy (TAT).

Collectively, this bespoke set of arrangements is designed to support the potential discovery, development, and commercial supply of a portfolio of Bicycle Radioconjugates (BRC) containing 212Pb.

"Cancer is a disease that affects millions worldwide, and tears too many families apart. Breakthroughs in medical science are giving more cancer patients and their loved ones hope, and this unique partnership could help take that work even further. Turning nuclear material into cutting-edge cancer treatments sounds like science fiction – but thanks to the brilliance of scientists, researchers and doctors, it could be a life-saving reality. Work like this shows exactly why we’re determined to support our life sciences innovators to make groundbreaking new treatments possible," said Liz Kendall, Secretary of State.

"These new collaborations are testaments to the potential of Bicycle to advance a differentiated and exciting isotope agnostic radiopharmaceuticals portfolio. We believe we now have the resources and infrastructure we need to create the world’s first end-to-end 212Pb radiopharmaceutical ecosystem from discovery through development to commercial supply. We believe the potential of BRCs and our ability to incorporate the appropriate isotopes for specific patient needs is unique and creates significant value-creating capabilities," said Mike Hannay, D.Sc., FRPharmS, chief product and supply chain officer of Bicycle Therapeutics. "We are incredibly grateful to the UK government for their recognition of Bicycle’s potential to develop BRCs with a 15-year access agreement for reprocessed uranium, and to our partners SpectronRx with whom we are developing a bespoke 212Pb generator. We look forward to advancing these dynamic collaborations and developing our pipeline of radiopharmaceuticals with the potential of improving lives for cancer patients around the globe."

"I am delighted with the progress we are making in establishing ourselves as a potential leader in radiopharmaceutical R&D. Today’s announcement builds on our previous announcements concerning our agreement with Eckert & Ziegler to supply a range of radioisotopes for the manufacture of BRC products as well as the formation of our Research and Innovation Advisory Board (RAB) comprising some of the worlds most esteemed experts in radiopharmaceuticals," said Bicycle Therapeutics CEO Kevin Lee, Ph.D. "As we continue to advance our emerging BRC pipeline, currently with novel targets EphA2 and MT1-MMP, we look forward to presenting initial EphA2 human imaging data in the first half of 2026 and initiating our own Bicycle study in 2026."

On December 18, 2025 Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, announced today that it has entered into a securities purchase agreement to sell 13,876,032 shares of its common stock at a price of $10.81 per share to a select group of institutional and accredited healthcare specialist investors in a private placement. Vor Bio anticipates the gross proceeds from the private placement to be approximately $150 million, before deducting any offering-related expenses. Vor Bio did not engage a placement agent in connection with the private placement. The private placement is expected to close on or about December 18, 2025, subject to the satisfaction of customary closing conditions.

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The financing includes participation from new and existing institutional investors, including RA Capital Management, Forbion, Frazier Life Sciences, Caligan Partners, Logos Capital, and Venrock Healthcare Capital Partners. In addition, as part of the private placement, Forbion is being granted the right to appoint one director to Vor Bio’s board of directors.

Vor Bio intends to use the net proceeds from the private placement to advance the clinical development of telitacicept, including the ongoing global Phase 3 clinical trial for myasthenia gravis and initiation of a global Phase 3 clinical trial in primary Sjögren’s disease, and for working capital and general corporate purposes.

The offer and sale of the securities to be sold in the private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other applicable jurisdiction’s securities laws, and such securities may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws. Concurrently with entering into the securities purchase agreement, Vor Bio and the investors entered into a registration rights agreement pursuant to which Vor Bio has agreed to file a registration statement with the Securities and Exchange Commission registering the resale of the shares of common stock issued in the private placement.

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

(Press release, Vor BioPharma, DEC 15, 2025, View Source [SID1234661538])

On December 15, 2025 PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported key accomplishments from 2025 and provided an overview of strategic priorities and anticipated milestones for 2026. The Company also announced its attendance at the 44th Annual J.P. Morgan Healthcare Conference, with a presentation scheduled for Thursday, January 15 at 10:30 a.m. PT (1:30 p.m. ET).

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"PharmaEssentia delivered a year of substantial progress in 2025, driven by the strong performance of BESREMi and preparation for continued growth supported by exceptional commercial execution and important pending product milestones," said Ko-Chung Lin, Ph.D., Founder and CEO of PharmaEssentia. "BESREMi has become a key growth engine for our company with the potential to transform treatment across multiple patient populations living with serious myeloproliferative neoplasms (MPNs). In 2025, we saw strong performance in polycythemia vera (PV) and are well positioned for further acceleration. With our injection pen submitted to the FDA and a supplemental BLA under review for the essential thrombocythemia (ET) indication, we are preparing for meaningful opportunities to expand adoption and patient reach. Alongside these anticipated 2026 catalysts, we continue to advance our pipeline, including the ongoing Phase 3 study evaluating BESREMi in pre-fibrotic/early primary myelofibrosis (Early PMF), and preparing to move additional earlier-stage programs into the clinic."

2025 Major Accomplishments

Commercial Performance of BESREMi

Delivered strong commercial growth of BESREMi in PV, with substantial year-over-year revenue growth (Q3 2025 revenue of $127.8 million, representing 44% YoY growth)
Strengthened commercial and medical affairs readiness for anticipated 2026 product launches
Regulatory & Clinical Progress

Submitted the BESREMi injection pen presentation to the FDA for review
Submitted supplemental BLA seeking BESREMi label expansion for ET based on positive head-to-head Phase 3 results
Continued global enrollment in the Phase 3 trial in Early PMF
Earlier Stage Program Progress

Advanced six high value programs to the IND-enabling stage:
PEG-IL-2 and P11838 for immunology and inflammation indications
PD-1–IL-2 immunocytokine for solid tumors
Dual-targeting LILRB1/2 monoclonal antibody for solid tumors
Novel ADC candidate for solid tumors
KRAS and NY-ESO TCR-T cell therapies for solid tumors
2026 Anticipated Milestones

BESREMi

U.S. launch of BESREMi Pen expected (H1 2026)
Potential FDA approval and commercial launch of ET indication (H2 2026)
Completion of enrollment in the global Early PMF Phase 3 clinical trial (H1 2026)
Pipeline & Portfolio Growth

File IND submissions for multiple earlier-stage pipeline programs
Share initial clinical results from multiple earlier-stage programs

(Press release, PharmaEssentia, DEC 15, 2025, View Source [SID1234661449])