On October 28, 2025 Harbour BioMed (HKEX: 02142), a global biopharmaceutical company focused on the discovery and development of novel antibody therapeutics in immunology and oncology, reported the launch of its first fully human Generative AI HCAb (Heavy Chain-Only Antibody) Model powered by its Hu-mAtrIx AI platform, built upon the Harbour Mice platform. This platform establishes a closed-loop process integrating AI design, intelligent screening, and wet-lab validation.

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The announcement was made at the company’s Global R&D Day 2025 event in Shanghai. During the event, the Global AI + Pharmaceutical Ecosystem Alliance was also officially launched. The alliance brings together leading expertise, cutting-edge technologies, and resources from various sectors, aiming to systematically reshape the entire drug R&D process through artificial intelligence technologies, accelerating the development of innovative therapies for the benefit of patients worldwide.

AI-Driven Precision for Faster and More Accurate Target Identification

The newly released AI HCAb Model establishes a closed-loop workflow that integrates AI-driven sequence generation, design and screening, and wet-lab validation. This end-to-end process transforms antibody discovery from blind screening to AI-driven intelligent selection, dramatically improving efficiency and accuracy in antibody discovery.

HCAb, known for its simple structure and low molecular weight, holds strong potential for advanced applications like bispecific/multi-specific antibodies, ADCs, CAR-T, and mRNA therapies. However, conventional human VH domains struggle to maintain stability without VL pairing.

Harbour BioMed’s Harbour Mice platform — the world’s first transgenic mouse platform that produces fully human functional HCAb — laid the foundation for overcoming this challenge. The key AI design lies in accurately identifying functional, autonomous HCAbs from vast datasets, given the high structural and sequential similarity between HCAb VH sequences and those of conventional (H2L2) antibodies. Harbour BioMed’s HCAb generation model is trained on 9 million NGS-derived HCAb sequences and extensive public data. Using a fine-tuned protein large language model, it enables de novo generation of high-potential HCAb sequences, with secondary optimization for target specificity.

These AI-generated sequences then undergo a multi-stage intelligent screening process, which includes:

AI Classification Model to filter non-HCAb sequences
Multimodal AI Developability Prediction Model (achieving SOTA performance) to assess key developability parameters such as stability, solubility, and aggregation tendency
Only candidates that pass rigorous screening proceed to synthesis and wet-lab validation.

AI HCAb Model Delivers Higher Binders Diversity and Binding Success Rate

The AI HCAb Model demonstrated a tenfold increase in candidate generation and significantly improved success rates. Data show that among 107 de novo generated binder sequences produced by AI HCAb model, 78.5% successfully hit the target, while 20 molecules were further validated in wet-lab assays, demonstrating high activity, purity, yield, and specificity.

These AI-designed binders demonstrated outstanding developability profiles, with an average yield exceeding 700 mg/L. Multiple candidate sequences showed nanomolar-level binding affinity while maintaining binding activity against relevant targets in both humans and cynomolgus monkeys.

Accelerating the Development of Next-Generation Therapies

Harbour BioMed’s AI platform is designed as a self-evolving innovation flywheel driven by continuous learning and feedback: AI design – automated validation – AI re-learning.

This iterative process enables generative AI to create diverse new molecules, while high-throughput automation rapidly characterizes and feeds back experimental data to continuously enhance model intelligence and R&D efficiency.

With this foundation, the AI HCAb Model is expected to accelerate applications of fully human HCAbs across next-generation therapeutic areas — including multi-specific antibodies, XDCs, in vivo CAR-T, and inhaled or oral large-molecule drugs — helping redefine the landscape of biologics discovery.

Building the Future AI + Pharmaceutical Ecosystem

At the event, Harbour BioMed also announced the official establishment of the Global AI + Pharmaceutical Ecosystem Alliance, which brings together leading experts, technology partners, and investors to reshape the entire drug discovery pipeline through AI innovation.

The alliance has gained strong support from government bodies, industry associations, and investors including Fortera Capital, Insilico Medicine, Molecular Mind, Evinova, INNOVEL, Fenglin Group, Taimei Technology, EClinCloud, Deep Intelligent Pharma, and Harbour BioMed.

Dr. Jingsong Wang, Founder, Chairman, and CEO of Harbour BioMed, said: "The Global AI + Pharmaceutical Ecosystem Alliance aims to create an open, collaborative, and win–win ecosystem. Through shared value and collective innovation, we seek to address the toughest challenges in drug discovery and bring transformative therapies to patients faster and more effectively."

(Press release, Harbour BioMed, OCT 28, 2025, View Source [SID1234657079])

On October 28, 2025 Pinetree Therapeutics, a preclinical-stage biotechnology company pioneering targeted protein degraders for drug-resistant cancers, reported the successful closing of an oversubscribed $47 million Series B financing. The proceeds will advance its lead preclinical oncology programs to Phase I clinical studies and accelerate development of Pinetree’s AbReptor platform. Participating investors include existing investors DSC Investment, WIDWIN Investment, STIC Ventures, Samho Green Investment, Atinum Investment, S&S Investment, SJ Investment Partners, Smilegate Investment, and Gauss Capital Management, as well as new investors Korea Investment Partners and SV Investment.

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Founded in 2019, Pinetree is an industry leader in the targeted protein degradation (TPD) field, developing a novel class of modular bispecific and multispecific protein degraders designed to selectively eliminate disease-driving membrane-bound and extracellular proteins. The AbReptor platform has demonstrated compelling preclinical efficacy and safety in degrading receptor tyrosine kinases (RTKs) that drive tumor growth and resistance, including targets refractory to existing tyrosine kinase inhibitors (TKIs) and immune checkpoint therapies.

"The AbReptor platform has repeatedly demonstrated durable activity across a broad range of RTK targets, including models resistant to standard-of-care therapies, and has unlocked the potential to develop a novel class of degrading antibody drug conjugates (ADCs), which aim to overcome the limitations of traditional ADCs, including tolerability and limited durability. Our platform also offers new therapeutic approaches for targeting inflammatory cytokines in disease tissues, representing a potential alternative to current immunology and inflammation therapies," said Ho-Juhn Song, Ph.D., Founder and CEO of Pinetree Therapeutics. "We are thrilled by the strong support from our investors and look forward to using these proceeds to advance differentiated therapies that address significant unmet clinical needs."

The Series B financing will support IND-enabling studies and Phase I clinical trials for Pinetree’s lead programs, expand its multispecific degrader portfolio, and enable strategic collaborations to unlock new target classes. "We are excited to continue our partnership with Pinetree as they advance a highly differentiated platform with the potential to meaningfully impact patients with RTK-driven tumors," said Yohan Kim, Executive Director of DSC Investment. "We see significant promise in Pinetree’s approach to overcoming resistance and improving tolerability, particularly for patients with limited treatment options."

This financing follows a July 2024 collaboration with AstraZeneca, which included an exclusive global license option for Pinetree’s preclinical EGFR degrader candidate, valued at over $500 million in potential milestones and royalties, reflecting growing interest in AbReptor therapeutics. Pinetree continues to expand its pipeline and strategic partnerships to deliver novel TPD programs that aim to transform treatment for patients with hard-to-treat cancers and other diseases.

(Press release, PineTree Therapeutics, OCT 28, 2025, View Source [SID1234657078])

On October 28, 2025 Alexion and AstraZeneca reported that its rare disease Koselugo (selumetinib), an oral, selective MEK inhibitor, has been approved in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1).

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The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) and is based on results from KOMET, the largest and only placebo-controlled global Phase III trial in this patient population, which were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and published in The Lancet.2

NF1 is a rare, progressive, genetic condition usually diagnosed in early childhood, but often progressing into adulthood, that can impact every organ system.3,4 Up to 50% of people living with NF1 may develop a type of non-malignant tumour called PN that may affect the brain, spinal cord and nerves.4,5 PN may appear later in a person’s life and can grow and become large, leading to pain, disfigurement and muscle weakness, among other debilitating symptoms.4,5

Prof. Pierre Wolkenstein, MD, PhD, Head of the Department of Dermatology at Henri Mondor Hospital, APHP, Paris East University (UPEC), and National Coordinating Investigator of the KOMET trial in Europe, said: "The approval of Koselugo for adults with NF1 PN in Europe offers patients and physicians a meaningful approach to close treatment gaps beyond childhood. As demonstrated in the KOMET Phase III trial, the most robust late-stage clinical trial conducted in this patient group to-date, adults administered Koselugo saw significant tumour volume reduction with a safety profile consistent with its established use in paediatric patients, validating the clinical benefits of Koselugo for newly diagnosed adults and those transitioning to adult care."

Marc Dunoyer, Chief Executive Officer, Alexion, said: "The European Commission approval extends the life-changing potential of Koselugo to adults with NF1 PN in the region, including continuity of care into adulthood. This milestone, along with our pioneering leadership in NF1 PN treatment landscape, embodies Alexion’s unwavering commitment to addressing the unmet needs in the rare disease community. We look forward to bringing Koselugo to those adults in need across Europe as soon as possible."

In the primary analysis of the trial, Koselugo showed a statistically significant objective response rate (ORR) of 20% (n=14/71, 95% CI: 11.2, 30.9) compared to 5% with placebo (n=4/74, 95% CI: 1.5, 13.3; p=0.01) by cycle 16. After 12 cycles, patients on placebo were switched to Koselugo and patients on Koselugo remained on treatment for an additional 12 cycles.2

The safety profile of Koselugo in the KOMET Phase III trial was consistent with its known profile and established use in paediatric patients.2

Koselugo has been recently approved in Japan and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN based on data from the KOMET Phase III trial, and additional regulatory reviews are ongoing.

Notes

NF1
NF1 is a rare, progressive, genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene.3,4 NF1 is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and, in up to 50% of patients, tumours called plexiform neurofibromas (PN) may develop on the nerve sheaths.4,5 These PN can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.4,5

KOMET
KOMET is a global Phase III randomised, double-blind, placebo-controlled, multicentre trial designed to evaluate the efficacy and safety of Koselugo in adults with NF1 who have symptomatic, inoperable PN. The trial enrolled 145 adults from 13 countries across North America, South America, Europe, Asia and Australia, with participants’ baseline characteristics, including gender and distribution of PN, reflective of the global adult NF1 patient population. Patients were enrolled and randomised to receive Koselugo or placebo (1:1) for 12 28-day cycles. Participants were required to have diagnosis of NF1, at least one symptomatic, inoperable PN measurable by volumetric MRI analysis, chronic PN pain score documented during screening, adequate organ and marrow function and stable chronic PN pain medication use at enrolment.2,6

The primary endpoint is confirmed objective response rate (ORR) by cycle 16 as assessed by ICR. ORR is defined as the percentage of patients with confirmed complete response (disappearance of PNs) or partial response (at least 20% reduction in tumour volume). Secondary endpoints include improved PN-related pain and health-related quality of life (HRQoL) at cycle 12.2,6

After 12 cycles, patients on placebo were switched to Koselugo and patients on Koselugo remained on treatment for an additional 12 cycles. Patients who had the opportunity to complete 24 cycles of treatment have the option to participate in a long-term extension period and continue to receive Koselugo.2,6

Koselugo

Koselugo (selumetinib) is a kinase inhibitor that blocks specific enzymes (MEK1 and MEK2), which are involved in stimulating cells to grow. In NF1, these enzymes are overactive, causing tumour cells to grow in an unregulated way creating so-called plexiform neurofibromas (PN). By blocking these enzymes, Koselugo slows down the growth of tumour cells and, therefore, the PN growth.

Koselugo is approved in the US, EU, Japan, China and other countries for the treatment of certain paediatric patients with NF1 who have symptomatic, inoperable PN.

Koselugo is approved in the EU, Japan and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN, and additional regulatory reviews are ongoing.

Koselugo has been granted Orphan Drug Designation in the US, EU, Japan and other countries for the treatment of NF1.

(Press release, AstraZeneca, OCT 28, 2025, View Source [SID1234657076])

On October 28, 2025 BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported it will participate in fireside chats at two upcoming investor conferences:

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Guggenheim Second Annual Healthcare Innovation Conference on Monday, November 10, 2025 at 9:30 am EST; and
Jefferies Global Healthcare Conference in London on Monday, November 17, 2025 at 4:00 pm GMT/11:00 am EST
Live webcasts of these events can be accessed from the investors section of the Company’s website at View Source, View Source, View Source Archived replays will be available on the Company’s website.

(Press release, BeOne Medicines, OCT 28, 2025, View Source/news/beone-medicines-to-present-at-upcoming-investor-conferences/2a85b5e9-db56-4488-b325-acec8a08ff0a [SID1234657075])

On October 28, 2025 Imugene Limited (ASX: IMU), a clinical-stage immuno-oncology company, reported the first efficacy results from the CAR T-naïve niche indication portion of its ongoing Phase 1b trial of azer-cel (azercabtagene zapreleucel) — an off-the-shelf, allogeneic CD19 CAR T therapy being evaluated across a spectrum of B-cell lymphomas.

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Of six evaluable CAR T-naïve patients (patients with no previous CAR T treatment), five (83%) achieved an overall response including three (50%) complete responses. The result of the sixth patient’s follow-up scan is pending.

A total of ten patients have been treated in this cohort thus far, with further results to come upon patient follow-up. These initial results encompass several rare lymphoma subtypes, notably WM, MZL and PCNSL — areas of significant unmet clinical need where no CAR T products are currently approved.

Azer-cel continues to demonstrate a manageable safety profile consistent with previously reported results in relapsed diffuse large B-cell lymphoma (DLBCL). Importantly, enrolment into the CAR T-naïve expansion is proceeding significantly faster than the earlier CAR T-relapsed DLBCL cohort, reflecting the substantial clinical demand and investigator enthusiasm for an allogeneic approach.

Imugene has an FDA Type C meeting in November to discuss pivotal study design options for azer-cel in CAR T-relapsed DLBCL, while also advancing the CAR T-naïve program to generate a broader evidence base across additional indications, including rare lymphomas to expand registrational options.

Chief Executive Officer Leslie Chong said "We are encouraged by these early signals of efficacy in the CAR T-naïve population, with at least five of the first six evaluable patients responding to treatment. As best responses can be seen up to 90 days or more after treatment, we look forward to further data on the depth and durability of these outcomes. Enrolment in this portion of the trial is proceeding at a remarkable pace, positioning us to explore an expedited development pathway for azer-cel."

In the CAR T relapsed DLBCL portion of the same Phase 1b study, azer-cel has achieved an 81% overall response rate to date, including multiple complete and partial responses, with several patients remaining in durable remission beyond a year. Patients in this cohort had typically failed an average of three prior lines of therapy, including autologous CAR T. These findings reinforce azer-cel’s potential as an allogeneic alternative for patients who relapse following existing CAR T treatments and complement the strong early data now emerging in the CAR T-naïve setting.

(Press release, Imugene, OCT 28, 2025, View Source [SID1234657074])