Unlocking the Potential of Single-Target Compounds in Modern Cancer Care

Cancer treatment has changed dramatically over the past two decades. One of the biggest breakthroughs has been the development of medicines that focus on a single, well-defined target inside a cancer cell. These medicines, called single-target compounds, are designed to switch off the specific molecule or pathway that helps a tumor survive and grow.

For many patients, this marks a shift from traditional chemotherapy, which works broadly across the body, to precision therapy that is tailored to the biology of their own cancer.

These treatments are not just scientific innovations. They represent hope for patients with cancers that were once considered untreatable, and they demonstrate what is possible when medicine becomes more personalized.

How Single-Target Cancer Medicines Are Discovered

The development of these drugs begins long before a patient receives them. Researchers first identify a molecule inside cancer cells that plays a key role in driving the disease. This molecule could be a mutated protein, a surface receptor, or a signaling enzyme that helps cancer cells divide uncontrollably[1].

Scientists then test thousands of compounds through laboratory screening and computer-based simulations to find one that can block this target safely and effectively. This stage has become far more advanced with the rise of genomics. Today, next-generation sequencing can reveal the exact mutations in a patient’s tumor, allowing researchers to focus on targets that matter most[2].

One of the best known examples is imatinib, developed for chronic myeloid leukemia. This drug was designed to silence a specific abnormal protein called BCR-ABL. What began as a weak lead molecule was optimized step by step until it became a therapy that transformed leukemia survival rates worldwide[3].

This story is not unique. Similar advances have been observed across lung cancer, breast cancer, melanoma, and many other malignancies, proving that when you find the right target, you can reshape the entire course of a disease.

How These Medicines Work Inside the Body

Single-target compounds fall into two main categories: small molecule inhibitors and monoclonal antibodies. Both work differently but share the same mission: to shut down the pathways tumors rely on.

Small molecule inhibitors
These medicines are tiny enough to enter cancer cells and block internal proteins known as kinases. Kinases act like switches inside the cell, turning signals on and off. When mutated, they can get stuck in the “on” position, driving cancer cells to multiply.

By blocking the kinase at its active site or a nearby allosteric site, small molecule inhibitors prevent the signals that drive cancer growth. This slows or stops the disease at the molecular level.

Monoclonal antibodies
These treatments target receptors or proteins on the surface of cancer cells. They can stop growth signals from being sent, mark cancer cells for destruction by the immune system, or trigger cell death directly.

Rituximab, trastuzumab, and many others have proven how powerful targeted antibodies can be when a tumor expresses the right marker[1].

When Single-Target Compounds Work Best

These medicines have had the greatest impact in cancers where a specific mutation or protein plays a central role. Key examples include:

• Chronic myeloid leukemia: Imatinib revolutionised survival, helping more than 80 percent of patients live for many years with good quality of life[3].

• Lung cancer: Patients with EGFR mutations or ALK fusions often respond dramatically to drugs specifically designed for these abnormalities, with response rates exceeding 60 percent[4].

• HER2-positive breast cancer: Trastuzumab improves survival when added to chemotherapy and reduces the risk of recurrence over the long term[5].

• Melanoma: Blocking BRAF and MEK simultaneously has extended survival for patients with BRAF-mutant disease[6].

These are examples of personalised medicine at its best, where knowing a single mutation can reshape treatment decisions entirely.

Why Resistance Still Happens

As effective as targeted drugs can be, they are not perfect. Many patients eventually develop resistance. Cancer cells are highly adaptive and can find new ways to survive even after their main driver is blocked.

Resistance can arise because the target mutates, the cancer activates a backup pathway, or the tumor evolves into a different cell type altogether. In some cases, small pockets of resistant cells already exist before treatment even begins.

For patients, this can feel discouraging, especially after an initially strong response. But understanding resistance has helped guide the next phase of progress.

Combination Strategies: The Future of Single-Target Therapy

To overcome resistance, researchers are now combining targeted drugs with others, with immunotherapy, or with novel agents such as SHP2 inhibitors. These combinations aim to shut down multiple pathways at once, leaving the cancer with fewer escape routes.

This approach has already shown success. BRAF and MEK inhibitors used together in melanoma deliver deeper and longer responses. In colorectal cancer with BRAF mutations, combinations involving EGFR and BRAF inhibitors outperform single-agent therapy[7].

Even more advanced strategies are being explored through artificial intelligence, high-throughput screening, and real-time genomic monitoring.

For patients, this means a future defined by treatments that are increasingly personalized, increasingly effective, and increasingly designed around the uniqueness of their own cancer.

At Helix BioPharma, we are committed to advancing this vision.

Our research focuses on therapies that directly address the molecular vulnerabilities of difficult cancers, while also overcoming the barriers that limit current targeted treatments. By integrating scientific insight with patient-centred innovation, our goal is to help move precision oncology from possibility to reality, and to bring better options to those who need them most.

Ref:

1. Min HY, Lee HY. Molecular targeted therapy for anticancer treatment. Exp Mol Med. 2022 Oct;54(10):1670-1694. doi: 10.1038/s12276-022-00864-3. Epub 2022 Oct 12. PMID: 36224343; PMCID: PMC9636149.

2. Doostmohammadi A, Jooya H, Ghorbanian K, Gohari S, Dadashpour M. Potentials and future perspectives of multi-target drugs in cancer treatment: the next generation anti-cancer agents. Cell Commun Signal. 2024 Apr 15;22(1):228. doi: 10.1186/s12964-024-01607-9. PMID: 38622735; PMCID: PMC11020265.

3. Baran Y, Saydam G. Cumulative clinical experience from a decade of use: imatinib as first-line treatment of chronic myeloid leukemia. J Blood Med. 2012;3:139-50. doi: 10.2147/JBM.S29132. Epub 2012 Nov 16. PMID: 23180974; PMCID: PMC3503471.

4. Leonetti A, Sharma S, Minari R, Perego P, Giovannetti E, Tiseo M. Resistance mechanisms to osimertinib in EGFR-mutated non-small cell lung cancer. Br J Cancer. 2019 Oct;121(9):725-737. doi: 10.1038/s41416-019-0573-8. Epub 2019 Sep 30. PMID: 31564718; PMCID: PMC6889286.

5. Early Breast Cancer Trialists’ Collaborative group (EBCTCG). Trastuzumab for early-stage, HER2-positive breast cancer: a meta-analysis of 13 864 women in seven randomised trials. Lancet Oncol. 2021 Aug;22(8):1139-1150. doi: 10.1016/S1470-2045(21)00288-6. PMID: 34339645; PMCID: PMC8324484.

6. Priantti JN, Vilbert M, Madeira T, Moraes FCA, Hein ECK, Saeed A, Cavalcante L. Efficacy and Safety of Rechallenge with BRAF/MEK Inhibitors in Advanced Melanoma Patients: A Systematic Review and Meta-Analysis. Cancers (Basel). 2023 Jul 25;15(15):3754. doi: 10.3390/cancers15153754. PMID: 37568570; PMCID: PMC10417341.

7. Liu M, Yang X, Liu J, Zhao B, Cai W, Li Y, Hu D. Efficacy and safety of BRAF inhibition alone versus combined BRAF and MEK inhibition in melanoma: a meta-analysis of randomized controlled trials. Oncotarget. 2017 May 9;8(19):32258-32269. doi: 10.18632/oncotarget.15632. PMID: 28416755; PMCID: PMC5458282.

Jacek Antas

Chief Executive Officer

Jacek Antas is a shareholder of the Company, has spent more than 25 years in the financial services industry holding various positions in sales and consulting.

Mr. Antas obtained a master’s degree from the Warsaw School of Economics and has served as a board member of various
companies throughout his career.

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James B. Murphy

Chief Financial Officer

Mr. Murphy is a certified public accountant with over thirty years of experience in finance and operations management. He is currently a consultant with Danforth Advisors LLC (“Danforth”), a leading provider of outsourced strategic and operational specialists across functions in the life sciences industry. While at Danforth, Mr. Murphy has served over fifteen private and publicly held life sciences companies as CFO and CFO Advisor, helping them secure over USD 0.5 billion in financing and successfully execute pivotal asset transactions. Mr. Murphy functions as a consultant to Helix pursuant to a consulting agreement between the Company and Danforth.

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Thomas Mehrling

Medical Adviser

Thomas Mehrling (PhD in Pharmacology and MD) has over 20 years’ experience in multinational Pharma companies developing novel oncology compounds from preclinical research through to registration. Prior to entering the industry, he spent 13 years as an MD at the University Hospital in Frankfurt, working on preclinical and translational projects. He served as Director of European Oncology at Mundipharma International (2003–2013), building the company’s first European oncology business from the ground up out of Cambridge, UK, and completing the clinical development, registration and launch of two major products in Europe, DepoCyte® and Levact® (Ribomustin® and Treanda®). In 2013, he led the establishment of the Mundipharma Group’s start-up, Mundipharma EDO, developing anti-cancer therapeutics for solid tumours out of Basel, Switzerland.

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Kim Gaspar

Director Quality Assurance

Kim is the Director of Quality Assurance at Helix BioPharma Corp. An experienced quality assurance professional with expertise in Canadian, US, and EU regulations, she has been involved in all aspects of Phase I/II biopharmaceutical product development over the years, including regulatory submissions, QC laboratory compliance, tech transfer and third-party oversight of CMC activities, clinical QA, and bioanalytical data analysis. Kim joined Helix in 2000, transitioning into QA in 2003. She holds a B.Sc in Biochemistry and a Ph.D in Veterinary Physiological Sciences, both from the University of Saskatchewan.

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Brenda Lee

Director Clinical Operations

Brenda is the Clinical Operations Director at Helix Biopharma Corp. A clinical research operations professional with 25 years of experience managing clinical trials, ranging from early Phase I to late Phase IIIb/IV studies, she brings experience in clinical study protocol writing and development, trial start-up and vendor management, and a proven track record in planning and managing clinical trials to quality standards, timelines and budget. Brenda joined Helix Biopharma Corp. in 2018, working to advance the clinical program of L-DOS47. She holds B.Sc and M.Sc. degrees from the University of Toronto, specializing in Nutritional Sciences and Human Biology.

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Jerzy Leszczynski

Director

Jerzy Leszczynski is a shareholder of the Company, has spent more than 35 years developing businesses and has served in the capacity of board member of various real estate development companies. Mr. Leszczynski obtained his Master of Science in Chemistry from the Warsaw Institute of Technology.

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Janusz Grabski

Director, Chair of Audit Committee

Janusz (John) Grabski is a lawyer specialized in corporate and real estate law with over twenty years of experience.

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Malgorzata Laube

Director

Malgorzata Laube has over 19 years of experience in nuclear medicine. In her last role with Alberta Health Services, she was the Department Supervisor, Nuclear Medicine at Royal Alexandra Hospital. Ms. Laube obtained a MSc degree in Environmental Engineering from the Warsaw University of Technology and is based in Edmonton, Alberta, Canada.

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Jacek Antas

Chairman of the Board

Jacek Antas is a shareholder of the Company, has spent more than 25 years in the financial services industry holding various positions in sales and consulting.

Mr. Antas obtained a master’s degree from the Warsaw School of Economics and has served as a board member of various
companies throughout his career.

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Jonathan Davis

Advisor, ADC Discovery

Jonathan Davis received his Ph.D. from University of California, San Francisco, where he studied protein structure and function using NMR. After a post-doc at Harvard Medical School exploring RNA selection and structure in the labs of Jack Szostak and Gerhard Wagner, he went to work at EMD Serono, where his work involved improving antibody-based therapeutics, inventing a platform technology for generating heterodimeric Fcs as a basis for multifunctional molecules, and developing a novel scaffold based on an artificially-designed protein from David Baker’s lab. In 2008 he took a job at Bristol-Myers Squibb in Waltham/Cambridge MA, working on antibody discovery and platform development in a wide range of therapeutic areas, with a particular focus on multispecific therapeutics. He moved to Madison, WI in 2019 to take on the role of VP of Innovation and Strategy at Invenra, a biotech focused on bispecific antibodies, and where he is currently head of the Scientific Advisory Board. In early 2024 he left the corporate world to found Creative Antibodies, a consulting firm that helps guide companies to successful antibody discovery and development projects, from mAbs to multispecifics, ADCs, and other formats. Outside of science, Jonathan is a conservatory trained cellist, plays numerous other instruments, and founded the UCSF Orchestra (now Symphony Parnassus) in San Francisco, where he was Music Director for six years.

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Davide Guggi

Advisor, CMC

Davide graduated as a pharmacist and received his PhD in Pharmaceutical Technology and Biotechnology from the University of Vienna. He has over 20 years of experience in the pharmaceutical industry, principally in the field of oncology. At the beginning of his career, Davide led oncology business units and commercial departments at Mundipharma and Gilead across Austria and Eastern Europe. Since over 10 years he has been working as a CMC expert, covering operational and regulatory CMC functions on behalf of over 20 different small- and medium-sized biotech companies across the world. He has served as CMC Director and CSO/CTO for several years, developing both small molecules and biologics (mABs, Fab, ADCs and Radio-immuno-conjugates) from early discovery to NDA/BLA in the US, EU and Canada, with a focus on First-in-Human and Phase I/II studies in oncology indications.

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Tumor Defense Breaker™, L-DOS47

L‑DOS47 is a first‑in‑class, clinical-stage antibody‑enzyme conjugate designed to deliver a game-changing assist to anti-cancer immunity and today’s leading cancer immunotherapies for the treatment of prevalent, hard-to-treat solid tumors. The compound precisely targets CEACAM6, a cell-surface protein overexpressed in non‑small cell lung cancer (NSCLC) and other aggressive tumors, where it delivers an enzymatic payload that raises the extracellular pH of the acidic tumor microenvironment (TME). By neutralizing tumor acidity, L-DOS47 restores immune cell infiltration and activity, helps turn immunologically “cold” tumors “hot”, and enhances the therapeutic reach of immune checkpoint inhibitors. With patented composition-of-matter coverage through 2036 and demonstrated synergy with PD-1 inhibitor, pembrolizumab, L-DOS47 is poised to significantly increase the efficacy of immune checkpoint blockade and unlock broader and more durable responses in NSCLC and other aggressive solid tumors.



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LEUMUNA™ is an oral immune checkpoint modulator designed to activate the donor immune system to recognize and fight relapsing leukemia in patients who have undergone allogeneic stem cell transplantation (allo-SCT). Although a life-saving procedure, up to 30% of patients who undergo allo-SCT see their cancer return, facing a median survival of just four months. LEUMUNA aims to offer these patients a new lease on life, by activating an immune cascade and inciting graft-versus-leukemia (GvL) effect, potentially offering long-term remission. Backed by strong preclinical data and a promising safety record from trials with its precursor compound, ulodesine, LEUMUNA offers a patient‑friendly, oral approach to a difficult-to-treat condition, with patent protection through 2041 and an Orphan Drug Designation granted by the US FDA.



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GEMCEDA™

GEMCEDA is a first-in-class oral prodrug of gemcitabine that opens up the possibility for convenient at-home administration, metronomic dosing and seamless integration into combination regimens with immune checkpoint inhibitors. To date, gemcitabine is only administered intravenously because oral forms have shown poor bioavailability of about 10%. GEMCEDA was developed as a prodrug to enable new uses of gemcitabine by combining it with cedazuridine, an enzyme inhibitor that helps boost its bioavailability to 90%. This remarkable innovation allows for greater flexibility in dosing schedules, fewer clinic visits, and a better quality of life, while achieving bioavailability on par with intravenous gemcitabine. Supported by a well‑established safety profile, scalable manufacturing, and patent coverage to 2043, GEMCEDA reimagines how chemotherapy can fit into patients’ lives.



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