Unlike most visions for the future, Helix BioPharma’s is focused on the near term, with longer-term goals shaped by the success and learning that come from acting now. The reason is simple: time is a luxury that many of us don’t have; while the science is advancing, outcomes are not keeping pace with the real, immediate needs of those facing a diagnosis today. Innovation takes time, especially in oncology, where cancer is not a single disease but a complex group of more than 200 distinct types, each with its own biology, behavior, and response to treatment.
The incidence of cancer is projected to increase by 77% by 2050, meaning that for every person diagnosed with cancer today, there will soon be nearly two. This trend raises serious concerns about whether turning innovation into widely adoptable, effective therapies can keep up with this growing, global emergency for humanity. Meeting the needs of a larger population affected by cancer in the future hinges on our ability to meet the needs of people living with cancer today; and these patients have already waited long enough for better treatment options. At Helix, next-generation cancer therapies are exactly that: what comes next, not what comes years down the line.
That’s why our focus is on turning proven science into near-term, high-impact solutions. We do this by taking a pragmatic approach to innovation (innovating from strength): selecting high-potential candidates where we can deliver difference, rather than investing valuable time and resources in proving premise; developing therapies designed to elevate and fit seamlessly into existing care algorithms; and leveraging insight and know-how to identify value-based opportunities and strategically accelerate their development, to bring urgently-needed therapies to those who need them most.
Our recent acquisition of LEUMUNA™ and GEMCEDA™ reflects this strategy in action: two next-generation candidates that address hard-to-treat cancers, build on proven science, and offer a direct path to meaningful impact.
i) LEUMUNA™
LEUMUNA™ (LR 09, ulodesine hemiglutarate) is a novel, patented, oral immune checkpoint modulator with the potential to significantly extend progression-free survival for patients relapsing with leukemia after allogeneic stem cell transplantation (allo-SCT). Allo-SCT is a physically and emotionally taxing, often life-threatening procedure, which patients willingly endure because it represents the only potentially curative treatment for acute leukemia and other hematological malignancies. If the procedure is successful, patients with hematological cancers have a 20% to 30% chance of relapsing within 24 months, delivering a devastating blow to morale and a grim prognosis of just four months. In this scenario, the treatment landscape is fragmented and options are largely ineffective, with most offering limited durability and few achieving long-term remission in specific patient subsets, making leukemia relapse a hard-to-treat cancer, today.
In preclinical models of B-cell acute lymphoblastic leukemia relapse conducted at the Fred Hutchinson Cancer Center, LEUMUNA has demonstrated the ability to significantly reduce the risk of leukemic death by indirectly activating T cells and triggering a graft-versus-leukemia (GvL) effect. This immune-mediated mechanism, in which donor immune cells recognize and eliminate residual host leukemia cells post-transplant, is the most effective path to long-term remission. Yet despite its potential, the GvL response has remained under-leveraged in post-transplant relapse treatment. LEUMUNA aims to change that, and it brings a head start and distinct advantages that made its acquisition a strategic fit for Helix.
LEUMUNA exemplifies our strategy of innovating from strength, in that it builds on a strong foundation of prior clinical research while introducing a novel formulation that unlocks new therapeutic potential. The active compound in LEUMUNA is ulodesine hemiglutarate, a patented, novel salt form of ulodesine that retains the pharmacodynamic properties of its predecessor while offering distinct advantages in safety, manufacturing, and intellectual property protection.
Ulodesine (BCX-4208) was originally developed by BioCryst Pharmaceuticals in collaboration with Roche as an immunosuppressant for autoimmune conditions, such as psoriasis. However, its mechanism of action was not fully understood at the time; whereas the goal of ulodesine’s initial development was to suppress immune activity, subsequent research revealed that PNP inhibition selectively depletes immunosuppressive regulatory T cells (Tregs), effectively enhancing anti-tumor immune responses by unmasking effector T cell activity. After failing to meet efficacy endpoints in a Phase IIa psoriasis trial, Roche discontinued the program in 2008. BioCryst then pursued ulodesine as a treatment for gout, completing Phase IIb trials that confirmed a strong safety profile in over 500 volunteers, but the program was ultimately shelved for strategic reasons.
Further validating LEUMUNA’s mechanism as an immune checkpoint modulator is the case of forodesine hydrochloride, an analogous PNP inhibitor also out-licensed by BioCryst to Mundipharma International in 2006. Forodesine showed early clinical efficacy in various hematological malignancies, with two patients achieving complete remission after relapsing with T-cell acute lymphoblastic leukemia after allo-SCT. One of these patients was a three-year-old little girl, whose patient case titled “When a Drug Becomes a Child’s Last Hope for T-Cell Leukaemia” is available on YouTube. Forodesine was eventually approved in Japan for peripheral T-cell lymphoma (PTCL) under the trade name Mundesine™, but its broader clinical development was hampered by a complex manufacturing process, followed by patent expiry.
LEUMUNA not only mirrors forodesine’s therapeutic potential—it improves upon it. With a simplified five-step synthesis, a clean clinical safety record via ulodesine, and a long intellectual property runway to 2041, LEUMUNA offers a scalable, efficient, and biologically targeted treatment approach for a devastating and underserved cancer relapse scenario.
Its re-development as ulodesine hemiglutarate was initiated by Laevoroc Immunology AG, the company from which Helix acquired the asset, in a research collaboration with UCLA. Laevoroc Immunology was co-founded by hemato-oncologist Thomas Mehrling, MD, PhD, now CEO of Helix BioPharma, who previously oversaw the development of forodesine during his tenure at Mundipharma, and Shanta Bantia, PhD, a recognized expert in PNP biology who was instrumental in advancing both forodesine and ulodesine at BioCryst Pharmaceuticals. This continuity of insight—from compound to clinical strategy—positions Helix to unlock LEUMUNA’s full therapeutic potential with a rare combination of scientific, developmental, and operational strength. LEUMUNA has additionally been granted Orphan Drug Designation (ODD) by the US FDA in December 2022, further reinforcing Helix’s strategy of innovating from strength by enabling a more capital-efficient path to market, regulatory incentives, and validating the therapeutic potential of the compound in a critically underserved population.
ii) GEMCEDA™
GEMCEDA (LR 06B, an oral gemcitabine prodrug combined with cedazuridine) is a first-in-class, oral chemotherapy agent designed to transform the administration of gemcitabine—a cornerstone of cancer treatment and a World Health Organization (WHO) Essential Medicine.
Intravenous (IV) gemcitabine remains a mainstay of cancer treatment, indicated as first-line therapy alone or in combination with other cytotoxic drugs for advanced or metastatic pancreatic cancer, advanced or metastatic non-small cell lung cancer (NSCLC), and metastatic breast cancer after failure of adjuvant chemotherapy, among other indications. These are among the most challenging cancers to treat—aggressive, often diagnosed late, and associated with poor prognosis and limited durable treatment options. In addition to its cytotoxic effects, gemcitabine has immune modulating effects on tumor cells within the tumor microenvironment (TME) and enhances innate anti-tumor responses, making it a promising candidate for combinations with immunotherapies like checkpoint inhibitors. Low-dose, metronomic therapy with IV gemcitabine combined with a PD-1 inhibitor in 61 patients with advanced lung, pancreatic, breast and other cancers has been shown to be well tolerated and to significantly improve treatment outcomes, particularly in post-operative patients. However, the full potential of gemcitabine as a combination therapy and metronomically dosed agent is limited by its IV administration, due to its rapid degradation, short half-life, and spiking peak-trough levels, which together reduce treatment flexibility, and require frequent hospital visits that add to the burden of people fighting advanced cancers.
Oral chemotherapy is increasingly used in the treatment of cancer; a total of 88 oral chemotherapy agents have been approved by the FDA over the past 20 years, with over 30% of the 900 chemotherapy agents currently in development being oral formulations. However, there are currently no oral forms of gemcitabine, largely due to its poor bioavailability (less than 10%), which has made it nearly impossible to achieve the necessary drug concentrations in the body for an effective therapeutic effect. Numerous efforts to develop oral gemcitabine have been discontinued due to challenges in achieving sufficient systemic exposure.
In 2022, Laevoroc Chemotherapy AG announced the development of a novel active pharmaceutical ingredient (API), LR 06B (GEMCEDA): a rationally-designed, novel chemical entity to enable new uses of gemcitabine as a single agent in oral metronomic/maintenance therapy and/or combinations with oral targeted therapies. The compound was developed under a joint venture between Laevoroc Chemotherapy and Lipomed AG, succeeding a collaboration agreement between the two companies. When combined with cedazuridine, a cytidine deaminase inhibitor, GEMCEDA achieved a staggering 90% bioavailability in a pharmacokinetic study in large animal models—near-matching the bioavailability of IV gemcitabine.
Gemcitabine’s long-standing use across multiple tumor types, combined with its well-established safety and efficacy profile, significantly de-risks the clinical development of GEMCEDA. As a novel, rationally-designed prodrug of a chemotherapy agent with a validated mechanism of action and improved oral bioavailability, GEMCEDA may qualify for development via the FDA’s 505(b)(2) regulatory pathway, which allows for abridged timelines and reduced development costs by referencing existing data with gemcitabine. The compound benefits from an established synthetic route and scalable manufacturing process, designed to enable cost-effective production from the outset. Importantly, GEMCEDA is a patented new chemical entity with composition-of-matter protection extending to 2042, offering Helix and a potential future partner a long IP runway and commercial defensibility. An added strength for Helix is that Dr. Davide Guggi, Chief Technology Officer (CTO) of Helix and an expert in Chemistry, Manufacturing, and Controls (CMC), is one of the inventors of LR 06B, bringing deep knowledge of the compound’s formulation and production.
GEMCEDA may be used as a single agent for oral maintenance therapy or in combination with oral targeted or immunotherapies—including PARP inhibitors, ATR inhibitors, and PD-1/PD-L1 checkpoint inhibitors—presenting new ways to fight and better ways to live for people with advanced cancers. The program has already attracted early interest from leading generics companies seeking strategic partnerships around next-generation oral chemotherapies.
iii) Broadening the Horizon with Our New Acquisitions
The strategic addition of LEUMUNA and GEMCEDA to our asset portfolio expands the reach of our pipeline to deliver urgently needed therapies for hard-to-treat cancers. These assets complement our CEACAM6-targeting platform (consisting of our lead candidate, Tumor Defence Breaker™, L-DOS47, and CEACAM6-targeting antibody-drug conjugates, currently in discovery) in that they, too, transform hard-won learnings into faster, smarter and more accessible solutions for some of cancer’s biggest, most urgent challenges.
For us, next-generation cancer therapies are exactly that: advancements standing on the shoulders of what came before. Innovation is a continuum, and innovating from strength is simply being honest about that and intentional in how we move forward.
Reference:
1 https://www.cancerresearch.org/blog/december-2023/exploring-the-different-types-of-cancer-and-treatment-options
2 https://acsjournals.onlinelibrary.wiley.com/doi/10.3322/caac.21834
3 https://www.thenewswire.com/press-releases/1BOxF12pw-helix-biopharma-tsx-hbp-otc-pink-hbpcd-frankfurt-hbp0-secures-pre-ind-candidates-leumuna-tm-and-gemceda-tm-in-strategic-acquisition-from-the-laevoroc-group.html
4 https://pubmed.ncbi.nlm.nih.gov/18026156/; https://haematologica.org/article/view/8281
5 https://pmc.ncbi.nlm.nih.gov/articles/PMC9850970/; https://www.sciencedirect.com/science/article/pii/S0006497119620185
6 https://link.springer.com/article/10.1007/s12185-024-03793-1
7 See unpublished data on p. 17 of our non-confidential deck: https://www.helixbiopharma.com/wp-content/uploads/2025/06/Helix-Public-Deck_June-2025.pdf
8 https://pmc.ncbi.nlm.nih.gov/articles/PMC10978651/
9 https://ir.biocryst.com/static-files/f139814f-6bdc-4843-a901-312f08d139f7
10 https://www.jci.org/articles/view/160852
11 https://ir.biocryst.com/news-releases/news-release-details/biocryst-reports-results-bcx-4208-phase-iia-trial-subjects
12 https://ir.biocryst.com/news-releases/news-release-details/biocryst-announces-positive-results-two-ulodesine-phase-2-trials; https://www.sec.gov/Archives/edgar/data/882796/000117184315004473/gfpf10q_080715.htm
13 https://ir.biocryst.com/news-releases/news-release-details/biocryst-announces-presentation-and-update-results-intravenous; https://www.sciencedirect.com/science/article/abs/pii/S0093775407002242?via%3Dihub
14 https://newdrugapprovals.org/2018/03/06/forodesine-hydrochloride/
15 https://www.nature.com/articles/d43747-023-00010-6
16 https://laevoroc.com/28-02-2023-laevoroc-immunology-announces-fda-orphan-drug-designation-granted-to-lr-09-a-novel-metabolic-immune-checkpoint-inhibitor-for-the-treatment-of-leukemia-relapse-after-allogeneic-st/
17 https://list.essentialmeds.org/recommendations/409
18 https://www.medicines.org.uk/emc/product/7298/smpc#gref
19 https://www.scientificarchives.com/article/gemcitabine-in-the-era-of-cancer-immunotherapy
20 https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2022.882172/ful
21 https://www.hoparx.org/documents/288/Oral_Chemotherapy_Issue_Brief_-_Updated_02.22.24.pdf
22 https://pubs.acs.org/doi/10.1021/acsnano.3c04793
23 https://pubmed.ncbi.nlm.nih.gov/32603666/ ; https://aacrjournals.org/clincancerres/article/14/11/3477/72692/Oral-Administration-of-Gemcitabine-in-Patients; https://aacrjournals.org/clincancerres/article/17/18/6071/76510/Phase-I-Study-of-Oral-Gemcitabine-Prodrug; https://pmc.ncbi.nlm.nih.gov/articles/PMC7606647/
24 https://laevoroc.com/18-03-2022-laevoroc-oncology-announces-evolution-of-former-subsidiary-into-joint-venture-with-lipomed-ag/