On March 31, 2024 Harbour BioMed ("HBM" or the "Company"; HKEX: 02142), a global biopharmaceutical company committed to the discovery and development of novel antibody therapeutics in immunology and oncology, reported its financial results for the year ended December 31, 2024 (Press release, Harbour BioMed, MAR 31, 2025, View Source [SID1234651697]).

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Dr. Jingsong Wang, Founder, Chairman, and CEO of Harbour BioMed, commented: "The year 2024 was a pivotal period for Harbour BioMed, marked by significant progress in research and development, strategic business expansion, and operational excellence. Despite a challenging macroeconomic environment, the Company demonstrated remarkable resilience, navigating inflationary pressures, global supply chain disruptions, and geopolitical uncertainties while remaining focused on its long-term growth objectives.

Throughout the year, Harbour BioMed advanced its strategic transition toward becoming a sustainably profitable global biotech engine. The Company expanded its research and development pipeline with a strong focus on immunology, autoimmune diseases, and inflammation therapeutics. Several key clinical-stage programs progressed significantly, reinforcing our vision of delivering life-changing medicines to patients worldwide.

The Company also made substantial investments in its technology platforms to ensure its core antibody discovery capabilities remain at the forefront of biopharmaceutical innovation. The strategic growth of Nona Biosciences, a subsidiary of Harbour BioMed, further solidified our position as a leading partner in the global biotech ecosystem. Additionally, we strengthened our business collaborations, securing multiple licensing and co-development agreements with top-tier pharmaceutical companies to drive sustainable revenue growth.

As we look ahead to 2025, we remain committed to expanding our global presence and delivering breakthrough therapies to patients."

Full Year 2024 Financial Highlights

Harbour BioMed reported total revenue of US$38.1 million for the year ended December 31, 2024, with an overall profit of approximately US$2.7 million. The company has been profitable for two consecutive years, demonstrating the uniqueness and resilience of its business model.

Compared to 2023, the company’s revenue sustainability has continued to improve. Annual recurring revenue increased from US$5.7 million to US$16.9 million, reflecting a growth rate of 196.5%. Cash profit[1] reached a record high of US$30.68 million. Meanwhile, Harbour BioMed has maintained a strong financial position. As of December 31, 2024, the company’s cash and cash equivalents totaled approximately US$166.8 million, providing a solid financial foundation for future growth.

Advancing a Robust and Differentiated Pipeline

Harbour Therapeutics, a sub-brand parallel to Nona Biosciences, is now individually responsible for the development of the Company’s products pipeline. With a growing focus on immunology, inflammation and oncology, Harbour Therapeutics manages a highly differentiated portfolio that includes multiple innovative drug candidates in both clinical and Investigational New Drug (IND)/IND-enabling stages.

In inflammation and immunology, the Company has built a robust preclinical pipeline encompassing bispecific and multi-specific antibodies generated using the HCAb-based Bispecific Immune Cell Antagonist (HBICATM) technology, as well as ultra long-acting biotherapeutics for immune-related diseases.

In oncology, the Company is leveraging its HBICE platform to develop bispecific and multi-specific antibodies with novel designs and differentiated mechanisms, such as HBM9027 (PD-L1xCD40) and HBM7004 (B7H4xCD3). In addition, the Company is utilizing its Harbour Mice and XDC platforms to explore multiple therapeutic modalities, including HBM9033, a mesothelin-targeted ADC, and other ADC/RDC programs in early-stage development.

Main products in the clinical stage include:

Batoclimab (HBM9161) is the first anti-FcRn monoclonal antibody completed Phase I to pivotal trials in China. As a novel, fully human anti-FcRn monoclonal antibody, batoclimab has the potential to be a breakthrough treatment option for a wide range of autoimmune diseases. In December 2023, the Company voluntarily planned to include additional long-term safety data and re-submitted the Biologic License Application (BLA) for batoclimab to the National Medical Products Administration of China (NMPA) in June 2024. In July 2024, NMPA accepted the BLA for batoclimab for the treatment of gMG. The Phase III pivotal clinical trial results of batoclimab were published in JAMA Neurology in March 2024, demonstrating sustained efficacy and safety with long-term use of batoclimab in the treatment of gMG.

HBM9378 is a fully human monoclonal antibody against thymic stromal lymphopoietin (TSLP) generated from the H2L2 Harbour Mice platform. It is a co-development project conducted by the Company and Kelun-Biotech, with both parties equally sharing the rights. HBM9378 has fully human sequences with a lower immunogenicity risk and better bioavailability compared to other TSLP-targeting competitors. Its long half-life optimization and outstanding biophysical properties support favourable dosing and formulation advantages.

The Company received IND approval of HBM9378 for moderate-to-severe asthma from the NMPA in February 2022 and completed a Phase I clinical trial in healthy subjects in China. In November 2024, the Company submitted an IND application for HBM9378 for chronic obstructive pulmonary disease (COPD) to the NMPA, which was approved in February 2025.
In January 2025, the Company and Kelun-Biotech entered into an exclusive license agreement with Windward Bio, under which Windward Bio was granted an exclusive license for the research, development, manufacturing and commercialization of HBM9378 globally (excluding Greater China and several Southeast and West Asian countries). Currently, Windward Bio is preparing a global Phase II clinical trial for HBM9378.
Porustobart (HBM4003) is a next-generation, fully human heavy-chain-only anti-CTLA-4 antibody discovered and developed using the HCAb Harbour Mice platform. It is also the first fully human heavy-chain-only antibody which entered clinical development globally. Compared with conventional CTLA-4 antibodies, porustobart has unique, favourable properties, including significant Treg cell depletion and optimized pharmacokinetics for improved safety. Additionally, by enhancing antibody-dependent cellular cytotoxicity (ADCC), porustobart increases the potential to selectively deplete intratumoral Treg cells, helping to overcome the efficacy and toxicity bottleneck of current CTLA-4 therapies. The Company has implemented a global development plan for multiple types of solid tumors with an adaptive treatment design for porustobart. Positive efficacy and safety data have been observed in the monotherapy trial targeting advanced solid tumors, as well as in combination trials with PD-1 inhibitors for melanoma, CRC, NEN and HCC.

HBM1020 is a first-in-class fully human monoclonal antibody generated from the H2L2 Harbour Mice platform targeting B7H7. As a newly discovered member of the B7 family, B7H7 expression is found to be non-overlapping with PD-L1 expression in multiple tumor types, potentially playing a more significant role in tumor immune evasion. With its excellent product design and target features, HBM1020 presents great potential to address significant unmet medical needs for solid tumors. In September 2024, the Company presented the latest clinical data for patients with advanced solid tumors at the ESMO (Free ESMO Whitepaper) Congress 2024. The data demonstrated excellent safety and tolerability profiles for HBM1020. Of the 15 patients who received post-treatment tumor assessments, 7 patients (46.7%) achieved stable disease (SD), with two patients showing tumor shrinkage of 11% and 25%.

Main products in IND and IND-enabling stages include:

HBM7020 is a BCMAxCD3 bispecific antibody generated using the fully human HBICE bispecific technology and Harbour Mice platform. HBM7020 can crosslink targeted cells and T cells by binding to BCMA and CD3 on the cell surface, leading to potent T cell activation and cell elimination. By incorporating dual anti-BCMA binding sites for optimal cell targeting and monovalent-optimized CD3 activity to minimize CRS, HBM7020 demonstrated potent cytotoxicity with broad applications in both immunological and oncology diseases. In August 2023, HBM7020 obtained IND clearance from the NMPA to commence a Phase I trial for cancer in China. In 2024, the Company restructured its development strategy to target immunological diseases and is currently preparing an IND application.

HBM9027 is a novel PD-L1xCD40 bispecific antibody developed using the HBICE bispecific antibody technology and Harbour Mice platform. The development of PD-L1xCD40 bispecific antibody further expands the Company’s bispecific immune cell engager into the cutting-edge DC/myeloid cell engager field, showcasing the HBICE platform’s versatile geometry formats and plug-and-play advantages. In January 2024, HBM9027 obtained IND approval from the Food and Drug Administration (FDA) to initiate a Phase I clinical trial in the U.S.

HBM7004 is a novel B7H4xCD3 bispecific antibody. Using HBICE bispecific technology and Harbour Mice platform, this bispecific antibody was designed to provide innovative solutions for cancer immunotherapy from both efficacy and safety perspectives. The development of B7H4xCD3 bispecific HBICE further consolidates the Company’s bispecific immune cell engager platform, demonstrating the HBICE platform’s versatility and plug-and-play advantages. In preclinical studies, HBM7004 demonstrated an intratumor B7H4-dependent T cell activation manner. In multiple animal models, HBM7004 showed strong anti-tumor efficacy, remarkable in vivo stability, and reduced systemic toxicity. Additionally, in preclinical models, HBM7004 exhibited a strong synergistic effect when combined with a B7H4x4-1BB bispecific antibody at a low effector-to-target cell ratio, indicating an encouraging therapeutic window. The Company is currently conducting IND-enabling studies for HBM7004.

HBM9014 is a first-in-class, fully human antibody targeting leukemia inhibitory factor receptor (LIFR) for cancer treatment. It was discovered using the Harbour Mice platform. HBM9014 blocks multiple IL-6 family cytokine pathways via LIFR to inhibit their function in promoting tumor progression, metastasis and chemoresistance. In preclinical studies, HBM9014 showed significant in vivo antitumor efficacy and enhanced efficacy in combination with cisplatin in multiple tumor models. In addition, HBM9014 exhibited strong tolerability in toxicology studies conducted on primates. In 2025, the Company will continue to actively explore drug development strategies and seek collaboration opportunities.

Strategic Business Collaborations Maximizing Platform Value

Harbour BioMed’s ongoing commitment to global partnerships is critical to driving both scientific and commercial success. In 2024, the Company continued expanding its business collaborations with leading academic institutions and select industrial partners, focused on driving innovation and efficiency worldwide.

Leveraging its technological advantages, Harbour BioMed established Nona Biosciences to better empower industry innovators and support collaborators from Idea to IND (I to ITM). Nona Biosciences is a global biotechnology company committed to providing integrated solutions for partners worldwide, across academia, biotechnology startups, and biopharmaceutical giants. Since its launch in late 2022, Nona Biosciences has achieved remarkable success, securing numerous international collaborations across various innovative formats. The subsidiary has established four leading technology units based on HCAb, including protein engineering, conjugation technology, delivery technology, and cell therapy, to accelerate the development of next-generation therapies.

Nona Biosciences signed a global out-license and option agreement with AstraZeneca in May 2024 for preclinical monoclonal antibodies to be used in developing targeted oncology therapies. In December 2024, Nona Biosciences entered a research collaboration and license agreement with Candid Therapeutics to discover next-generation T-cell engagers. Over the past year, Nona Biosciences has also formed research collaborations with multiple partners across various therapeutics areas, including Boostimmune, Alaya.bio, Umoja Biopharma, Alkyon Therapeutics, OverT Bio, and Kodiak Sciences.

In January 2025, the Company announced a license agreement with Windward Bio for HBM9378, an anti-TSLP fully human antibody for immunological diseases. In February 2025, HBM Alpha Therapeutics, an innovative biotechnology company incubated by the Company, announced a strategic collaboration and license agreement with a business partner to advance novel therapies targeting corticotropin-releasing hormone (CRH) for various disorders.

These collaborations further highlight the Company’s unique strengths in pushing technological boundaries and exploring new innovation pathways. With its industry-leading technology platforms and a flexible business model, the Company will continue seeking new opportunities to expand its collaboration network and maximize the value of its platforms.

Incubating Breakthrough Collaborations for the Future

To fully harness the potential of its unique platform technologies, Harbour BioMed continues to explore the scalability of its platform applications. The Company is incubating several joint ventures focused on next-generation therapeutics, ranging from multivalence antibodies to cell therapies. These ventures aim to broaden the application of platform technologies and create additional value for the Company. This innovative incubation model facilitates the integration of incremental resources for the diversified development of next-generation innovations, requiring minimal marginal investment while offering a high return on value growth. Representative projects include HBM Alpha Therapeutics and Shanghai NK Cell Technology Limited.

2025 Outlook: Expanding Global Presence and Driving Innovation

Looking ahead to 2025, Harbour BioMed will continue driving business growth and accomplishing its mission through two key pillars, Harbour Therapeutics and Nona Biosciences. Harbour Therapeutic will advance multiple clinical trials of its internal pipeline to fully advance the global clinical development project, while Nona Biosciences will continue providing integrated discovery solutions to biotech and pharmaceutical companies, ultimately fostering an innovation ecosystem that promotes biological advancement.

A range of products derived from the Company’s Harbour Mice technology platform and T-cell engager technology will be advanced in immunology, and the ADC platform will be upgraded to the next generation. Through a combination of in-house development and business collaborations, Harbour BioMed will build a portfolio of products with differentiated competitive advantages both in immunology and immuno-oncology.

Maximizing the value of its platform through strategic collaborations will continue to drive the Company’s global expansion. In 2025, as the preclinical product pipeline matures, the company expects to expand its collaboration network even further and enter into broader global partnerships, solidifying its leading position in the global market.

On March 31, 2025 BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that intends to change its name to BeOne Medicines Ltd., reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued a positive opinion recommending approval of TEVIMBRA (tislelizumab), in combination with etoposide and platinum chemotherapy, as a first-line treatment for adult patients with extensive-stage small cell lung cancer (ES-SCLC) (Press release, BeiGene, MAR 31, 2025, View Source [SID1234651660]).

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"The aggressive nature of extensive-stage small cell lung cancer makes it an extremely difficult type of lung cancer to treat, and currently available treatments may not adequately control disease progression," said Prof. Silvia Novello, M.D., Ph.D., President Women Against Lung Cancer in Europe (WALCE) and Head of Medical Oncology Unit of San Luigi Hospital in Orbassano, Italy. "The compelling data from the RATIONALE-312 study demonstrates the potential of TEVIMBRA plus chemotherapy as a further first-line treatment option to extend overall survival for patients with ES-SCLC."

The extension of indication for ES-SCLC is based on results from BeiGene’s RATIONALE-312 (NCT04005716), a randomized, double-blind, placebo-controlled, multicenter, Phase 3 study to evaluate the efficacy and safety of TEVIMBRA, in combination with platinum (investigator’s choice of cisplatin or carboplatin) plus etoposide, as first-line treatment in adult patients with ES-SCLC. The study, which randomized 457 patients, met its primary endpoint, exhibiting a statistically significant and clinically meaningful improvement in overall survival (OS) with TEVIMBRA in combination with chemotherapy, compared with placebo plus chemotherapy in the intent-to-treat (ITT) population. As reported in the Journal of Thoracic Oncology, at the protocol-defined final analysis, the median OS was 15.5 months for TEVIMBRA with chemotherapy versus 13.5 months for placebo plus chemotherapy (HR 0.75 [95% CI: 0.61–0.93]; one-sided p = 0.0040), resulting in a 25% reduction in the risk of death. TEVIMBRA plus chemotherapy was generally well tolerated, with no new safety signals identified.

"Today’s positive CHMP opinion marks another important step for TEVIMBRA to potentially expand its indications in a fourth disease area in Europe to reach more patients affected by cancer," said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeiGene. "TEVIMBRA is the cornerstone of our solid tumor portfolio with 58 regulatory approvals in 18 months and is being studied in combination with multiple novel molecules with the potential to herald the next wave of cancer therapeutics."

The pooled safety data in this extension of indication included more than 3,900 patients who received TEVIMBRA as either monotherapy (n=1,952) or in combination with chemotherapy (n=1,950) at the approved dosing regimen. The most common Grade 3 or 4 adverse reactions (≥ 2%) for TEVIMBRA given in combination with chemotherapy were neutropenia, anemia, thrombocytopenia, hyponatremia, hypokalemia, fatigue, pneumonia, lymphopenia, rash, decreased appetite, increased aspartate aminotransferase, and increased alanine aminotransferase.

TEVIMBRA is currently approved in the EU as a first-line treatment for eligible patients with unresectable esophageal squamous cell carcinoma (ESCC) and gastric or gastroesophageal junction (G/GEJ) adenocarcinoma in combination with chemotherapy, as a second line treatment in unresectable, locally advanced or metastatic ESCC after prior platinum-based chemotherapy, and for three non-small lung cancer (NSCLC) indications covering both the first- and second-line settings.

The Company recently announced its intent to change its name to BeOne Medicines, reaffirming its commitment to develop innovative medicines to eliminate cancer by partnering with the global community to serve as many patients as possible.

About Extensive-Stage Small Cell Lung Cancer (ES-SCLC)

Lung cancer is the leading cause of cancer-related deaths worldwide.1 SCLC is an aggressive, high-grade cancer that accounts for 15% of all lung cancers,2 and is typically classified as limited-stage or extensive-stage disease.3 Approximately 70% of SCLC patients are diagnosed with extensive-stage disease,4 defined as cancer that has spread throughout or beyond the lungs, or exceeding an area that can be treated with radiation alone.5 In Europe, the estimated prevalence of SCLC is 1-5 per 10,000 people.6 ES-SCLC is associated with a very poor prognosis with a median OS of 8 to 13 months and an expected 2-year survival rate of only 5%.7

About TEVIMBRA (Tislelizumab)

TEVIMBRA is a uniquely designed humanized immunoglobulin G4 (IgG4) anti-programmed cell death protein 1(PD-1) monoclonal antibody with high affinity and binding specificity against PD-1. It is designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors.

TEVIMBRA is the foundational asset of BeiGene’s solid tumor portfolio and has shown potential across multiple tumor types and disease settings. The global TEVIMBRA clinical development program includes almost 14,000 patients enrolled to date in 35 countries and regions across 70 trials, including 21 registration-enabling studies. TEVIMBRA is approved in 45 countries, and more than 1.3 million patients have been treated globally.

Important Safety Information

The current European Summary of Product Characteristics (SmPC) for TEVIMBRA is available from the European Medicines Agency.

This information is intended for a global audience. Product indications vary by region.

On March 31, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported it will present new non clinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place on April 25-30, 2025 in Chicago, Illinois (Press release, MaaT Pharma, MAR 31, 2025, View Source [SID1234651679]).
MaaT034 is the Company’s next-generation full ecosystem synthetic microbiome therapy and the first candidate from its proprietary MET-C platform. Developed using a co-culturing technology optimized for large-scale production, MaaT034 is designed to improve patient responses to immunotherapy in combination with Immune Checkpoint Inhibitors.

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The key findings, obtained from studies conducted in germ-free mice, demonstrate that MaaT034:

Effectively engrafts, thus ensuring an enduring presence of beneficial bacteria in the gut environment.
Potentiates anti-tumor effects mediated by anti-PD-1 checkpoint blockade.
Leads to the production of key microbial-derived metabolites in germ-free mice.
Improves gastrointestinal physiological functions.
In parallel, the Company is developing donor-derived microbiome therapies that are being explored in multiple clinical trials. Among these, MaaT013 has been tested in patients with acute Graft-versus-Host Disease (aGvHD) and advanced melanoma receiving immune checkpoint inhibitors (PICASSO trial).

"While the PICASSO trial builds on MaaT013’s established safety and efficacy to potentially unlock significant new market opportunities in oncology, our next-generation candidate, MaaT034 — to be featured at AACR (Free AACR Whitepaper) — is designed with an optimized microbiome composition that eliminates the need for donors and incorporates defined and reproducible immunomodulatory properties, offering enhanced therapeutic potential across a broader range of oncology indications," stated Hervé Affagard, CEO and co-founder of MaaT Pharma.

AACR Poster Presentation details:

Title: MaaT034, a new co-cultured microbiome ecosystem therapy candidate, is capable to safely colonize the gastro-intestinal tract of germ-free mice to restore a healthy gut physiology and to stimulate immunity
Abstract Number: 2209
Session Category: Immunology
Session Title: Microbiome, Inflammation, and Cancer
Session Date/ Time: Monday April 28th, 2025 – 9:00:00 AM U.S Central Time
About Picasso

PICASSO is a Phase 2a clinical trial sponsored by AP-HP and in collaboration with INRAE and Institut Gustave Roussy, evaluating MaaT013 in combination with immune checkpoint inhibitors, ipilimumab (Yervoy) and nivolumab (Opdivo). The trial is fully recruited and have enrolled 70 patients. Key study endpoints include MaaT013 safety profile and best-overall response rate vs placebo as add-on treatment to Ipilimumab + Nivolumab. Clinical Trials.gov: NCT04988841

About MaaT034

MaaT034, currently in preclinical development, is a next-generation donor-independent full ecosystem synthetic microbiome therapy, dedicated to improving patient responses to immunotherapy in combination with Immune Checkpoint Inhibitors. Developed using the Company’s co-culturing proprietary MET-C platform, MaaT034 is optimized for large-scale production in oncology. Previous presented preclinical data showed that MaaT034 produced key metabolites, recognized as promoting gut barrier restoration and modulating immune responses, such as Short-Chain Fatty Acids (SCFA), secondary bile acids, and tryptophan derivatives. These data support the role of MaaT034 in gut barrier repair and in T cell reactivation either in combination with anti-PD1 or with anti-PD-L1. By enhancing gut barrier repair and modulating immune responses, MaaT034 is expected to complement the action of these immunotherapeutic agents, potentially improving their efficacy in treating solid tumors cancer.

On March 31, 2025 Hoth Therapeutics, Inc. (NASDAQ: HOTH), a patient-focused clinical-stage biopharmaceutical company, reported that it has received an official Filing Receipt from the United States Patent and Trademark Office (USPTO) for a new patent application related to the proprietary formulation of HT-001, the Company’s lead clinical asset (Press release, Hoth Therapeutics, MAR 31, 2025, https://www.prnewswire.com/news-releases/hoth-therapeutics-receives-uspto-filing-receipt-for-ht-001-formulation-patent-application-expanding-existing-patent-coverage-302416196.html [SID1234651698]).

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This newly filed application seeks to expand the Company’s intellectual property portfolio surrounding HT-001 and builds upon Hoth’s already approved patent for the compound. The formulation-specific filing is aimed at further protecting the unique composition and delivery method of HT-001, which is being developed as a novel topical treatment to alleviate dermatologic side effects commonly experienced by cancer patients undergoing epidermal growth factor receptor (EGFR) inhibitor therapy.

"The receipt of this Filing Receipt from the USPTO marks another critical milestone in the development of HT-001," said Robb Knie, Chief Executive Officer of Hoth Therapeutics. "Expanding our patent protection to include formulation-specific claims reinforces the strength of our IP position and our long-term commitment to patients and shareholders."

The USPTO Filing Receipt confirms that the patent application has been formally accepted and will proceed through the examination process. This development adds an important layer of protection as Hoth continues advancing HT-001 through clinical development.

On March 31, 2025 BioLineRx Ltd. (NASDAQ/TASE: BLRX), a development stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, reported its audited financial results for the year ended December 31, 2024, and provided a corporate update (Press release, BioLineRx, MAR 31, 2025, View SourceArchives/edgar/data/1498403/000117891325001124/exhibit_1.htm" target="_blank" title="View SourceArchives/edgar/data/1498403/000117891325001124/exhibit_1.htm" rel="nofollow">View Source [SID1234651661]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"It has been just over four months since we implemented a major strategy shift, highlighted by the transformational exclusive licensing agreement that we entered into with Ayrmid Ltd., granting it the rights to commercialize APHEXDA (motixafortide) in all non-solid-tumor indications and all territories other than Asia," said Philip Serlin, Chief Executive Officer of BioLineRx. "Since then, we implemented cost efficiencies across the Company, including the shutdown of our U.S. commercial operations, that have resulted in an approximate 70% reduction in our operating expense base, which, together with recent financings, have put us on a firm footing with a cash runway through the second half of 2026."

"As we return to our roots as a lean drug development company, with a highly validated development platform focused on oncology and rare diseases, we believe these actions help ensure that we remain nimble and capable of seizing the opportunities in front of us. Our strategy moving forward is to in-license additional assets over the next year that we can advance through clinical proof-of-concept, funded in part by milestones and royalties from our out-licensing transactions. To that end, I am pleased to report that we are evaluating numerous promising candidates. This process is methodical and steady to ensure that our due diligence is thorough as we look for new chemical entities. Based on our deep and validated experience in drug development, I believe we are well positioned to create sustained value for our shareholders. I am excited about what the future holds for our Company this year and beyond," Mr. Serlin concluded.

Corporate Updates

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Executed license agreement with Ayrmid Pharma Ltd. to develop and commercialize APHEXDAâ (motixafortide) in all indications except solid tumors, and across all territories except Asia

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License agreement included a $10 million upfront payment, up to $87 million in potential commercial milestones, and royalties on net sales ranging from 18% to 23%

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Announced receipt of a Notice of Allowance from the U.S. Patent & Trademark Office (USPTO) for a patent, titled "Composition of BL-8040," which strengthens BioLineRx’s robust intellectual property (IP) estate and extends its patent protection on motixafortide (BL-8040) in the U.S. through December 2041

Financial Updates

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Completed two financings in past few months which raised combined gross proceeds of $19 million

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Reduced operating expense run rate by approximately 70% beginning January 1, 2025 through the APHEXDA program transfer to Ayrmid and the resulting shutdown of the Company’s U.S. commercial operations in Q424, as well as additional headcount and other operating expense reductions

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Significantly reduced outstanding debt and restructured the remainder on favorable terms to the Company

APHEXDA 2024 Performance Update

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Aphexda achieved 10 percent market share of total CXCR4 inhibitor usage in the U.S., which compares APHEXDA to branded MOZOBIL and generic plerixafor in all indications

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BioLineRx generated more than $6 million in net product sales year-to-date through the November 2024 completion of the Ayrmid out-licensing transaction

Clinical Updates

Motixafortide

Pancreatic Ductal Adenocarcinoma (mPDAC)

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Additional trial sites activated for the CheMo4METPANC Phase 2b clinical trial being led by Columbia University. Full enrollment in the randomized trial targeting 108 patients is anticipated in 2027, with a prespecified interim futility analysis planned when 40% of PFS events are observed

Sickle Cell Disease (SCD) & Gene Therapy

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First patient dosed in the multi-center Phase 1 clinical trial evaluating motixafortide for the mobilization of CD34+ hematopoietic stem cells (HSCs) used in the development of gene therapies for patients with Sickle Cell Disease (SCD). The trial is sponsored by St. Jude Children’s Research Hospital.

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Oral presentation delivered at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition detailing initial results from a Phase 1 clinical trial evaluating motixafortide as monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies in SCD. Sponsored by investigators at Washington University in St. Louis, the findings from this proof-of-concept study suggest motixafortide alone, and in combination with natalizumab, could support the collection of the large number of stem cells required by gene therapies for sickle cell disease within a single apheresis cycle.

Financial Results for the Year Ended December 31, 2024

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Revenues for the year ended December 31, 2024 were $28.9 million, an increase of $24.1 million, or 502.1%, compared to $4.8 million for the year ended December 31, 2023. The revenues in 2024 primarily reflect a portion of the up-front payment received, and a milestone payment achieved, under the Gloria license, which collectively amounted to $15.0 million, as well as the up-front payment received under the Ayrmid license and $6.0 million of net revenues from product sales of APHEXDA in the U.S. The revenues in 2023 (all of which were recorded in the fourth quarter of 2023) primarily reflect a portion of the up-front payment received under the Gloria license of $4.6 million, as well as $0.2 million of revenues from product sales of APHEXDA in the U.S.

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Cost of revenues for the year ended December 31, 2024 were $9.3 million, an increase of $5.6, or 151.4%, compared to $3.7 million for the year ended December 31, 2023. The cost of revenues in 2024 primarily reflects amortization of intangible assets, Biokine’s share of the up-front payment received under the Ayrmid license, sub-license fees accrued on a milestone payment recorded under the Gloria license, as well as royalties on net product sales of APHEXDA in the U.S. and cost of goods sold on product sales. The cost of revenues in 2023 primarily reflects Biokine’s share of the up-front payment received under the Gloria license and of the net sales.

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Research and development expenses for the year ended December 31, 2024 were $9.2 million, a decrease of $3.3 million, or 26.4%, compared to $12.5 million for the year ended December 31, 2023. The decrease resulted primarily from lower expenses related to motixafortide NDA supporting activities, termination of the development of AGI-134 and a decrease in payroll and share-based compensation.

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Sales and marketing expenses for the year ended December 31, 2024 were $23.6 million, a decrease of $1.7 million, or 6.7%, compared to $25.3 million for the year ended December 31, 2023. The decrease resulted primarily from the shutdown of U.S. commercial operations in the fourth quarter of 2024 following the Ayrmid license.

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General and administrative expenses for the year ended December 31, 2024 were $6.3 million, similar to the year ended December 31, 2023.

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Net non-operating income amounted to $18.4 million for the year ended December 31, 2024, compared to net non-operating expenses of $10.8 million for the year ended December 31, 2023. Non-operating income for the year ended December 31, 2024 primarily relates to non-cash, fair-value adjustments of warrant liabilities on the Company’s balance sheet, as a result of changes in the Company’s share price, offset by warrant offering expenses. Non-operating expenses for the year ended December 31, 2023 primarily relate to non-cash, fair-value adjustments of warrant liabilities on the Company’s balance sheet.

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Net financial expenses amounted to $7.3 million for the year ended December 31, 2024, compared to net financial expenses of $0.1 million for the year ended December 31, 2023. Net financial expenses for both periods primarily relate to interest paid on loans, which increased in 2024 due to a one-time $4.0 million charge to interest expense in connection with the November 2024 amendment to the loan agreement with BlackRock, partially offset by investment income earned on bank deposits.

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Net loss for the year ended December 31, 2024 was $9.2 million, compared to $60.6 million for the year ended December 31, 2023.

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As of December 31, 2024, the Company had cash, cash equivalents, and short-term bank deposits of $19.6 million (approximately $29.0 million on a pro-forma basis, following the financing completed at the beginning of January 2025).

A copy of the Company’s annual report on Form 20-F for the year ended December 31, 2024 has been filed with the U.S. Securities and Exchange Commission at View Source and posted on the Company’s investor relations website at View Source Company will deliver a hard copy of its annual report, including its complete audited consolidated financial statements, free of charge, to its shareholders upon request at [email protected].

Conference Call and Webcast Information
To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0685 internationally. A live webcast and a replay of the call can be accessed through the event page on the Company’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast. The call replay will be available approximately two hours after completion of the live conference call. A dial-in replay of the call will be available until April 2, 2025; please dial +1-888-295-2634 from the US or +972-3-925-5904 internationally.