Orphan oncology products


Primary liver cancer

Hepatocellular carcinoma (primary liver cancer) is the sixth most common cancer in terms of incidence (782,000 new cases per year) and the second leading cause of cancer deaths worldwide. It occurs in 80-90% of cases in patients with liver cirrhosis mainly due to viruses (virus B and C), alcohol or metabolic syndrome. It is a highly chemotherapy-resistant cancer, often diagnosed at an advanced stage and for which there is a very strong unmet therapeutic need today.

The product

 Livatag® is a nanoparticle formulation of doxorubicin (Transdrug™ technology) developed for the treatment of primary liver cancer.

Transdrug™ technology uses nanoparticles to help fight against chemotherapy resistance, a major mechanism in the failure of some anticancer drugs, that facilitate the penetration of the drug into the tumor cell and increase the cellular exposure to the drug.

Livatag® uses this technology along with doxorubicin in the treatment of advanced primary liver cancer. It allows bypassing the mechanisms of multi-drug resistance developed by tumour cells. Encapsulated in the form of nanoparticles, doxorubicin can then reach  its  therapeutic target and exert its cytotoxic activity.

Livatag® has obtained orphan drug status in two major territories, Europe and the USA, thus enhancing the value of this key asset for the Company. Livatag® has also obtained Fast Track status (accelerated review procedure)  from the FDA for the treatment of hepatocellular carcinoma (primary liver cancer) after treatment with Sorafenib (Nexavar®). This status recognizes that a drug is being developed for a severe or life-threatening pathology with a significant medical need.

The Phase II clinical trial showed that intra-arterial administration of Livatag®  in patients suffering from hepatocellular carcinoma provided a median survival of 32 months, an increase of 17 months when compared to 15 months for patients getting current best of care (TACE transarterial chemoembolisation with a cytotoxic drug).

Based on this data, an international, open randomised Phase III clinical trial (ReLive) comparing repeated intravenous administration of Livatag® to the best standard of care has been implemented in 400 patients with advanced-stage hepatocellular carcinoma, after failure or intolerance to sorafenib. A committee of independent experts has regularly reviewed  the tolerance of the treatment administered.

The company announced on September 11, 2017 that ReLive did not meet its primary endpoint of improving survival over the comparative group. The major reason is an unexpected high survival in the comparative group. Indeed, the study was not placebo controlled and patients in the comparative group could receive other anticancer agents (including oxaliplatin, gemcitabine or tyrosine kinase inhibitors) which might explain the high survival rate of the control arm. Livatag®, as single agent, showed a similar effect as the one observed in that comparative group with active treatments. There was no difference in efficacy between the two arms (Livatag® 20mg/m² and 30mg/m²).  The overall safety and tolerability profile of Livatag® in ReLive was favorable with a fully manageable toxicity profile in both groups of Livatag (20mg/m² and 30mg/m²) including in those patients who underwent the longest treatment periods, over one year. The overall tolerability was comparable to the one observed in the comparative group. The main results from the ReLive study were presented at the 11th Annual Conference of the International Liver Cancer Association in Seoul, South Korea (ILCA 2017 – September 15-17, 2017).

Pleaser refer to the press release and data presentation for aditional information.

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Peripheral T-Cell Lymphoma (PTCL)

Lymphoma is the most common blood cancer*. Hodgkin’s and non-Hodgkin’s lymphoma are the main two forms of lymphoma. The lymphoma survives when the lymphocytes, a type of white blood cell, increase abnormally and accumulate in one or more lymphatic ganglions or in lymphatic tissue. Two types of lymphocytes may develop: B lymphocytes (B cells) and T lymphocytes (T cells). Peripheral T-Cell Lymphoma (PTCL) is a sub-type of non-Hodgkin’s lymphoma which affects T cells. In the United States, PTCL accounts for around 10 to 15% of non-Hodgkin’s lymphoma and its global incidence is estimated at 12,000 cases each year. Currently no 2nd line treatment is available in Europe once the disease has progressed after a 1st line of treatment.
*Lymphoma Research Foundation (www.lymphoma.org)

The product

Belinostat is a histone deactylase inhibitor (HDACi). It has been assessed in several clinical trials as a sole treatment (monotherapy) or in combination with other anti-cancer treatments for hematological cancers and solid tumors. Its anti-cancer activity is associated with the inhibition of cell proliferation, the induction of apoptosis (programmed cell death), the inhibition of angiogenesis and the induction of cellular differentiation.

The company also plans to instigate in the coming months a development program on one or more potential indications based on the clinical results already obtained.

Since 2010, Beleodaq® has been licensed to an American partner, Spectrum Pharmaceuticals, Inc. (SPPI), for the USA and India. Spectrum Pharmaceuticals is responsible for the co-development of Beleodaq® for the United States and for promoting it to oncology and hematology specialists.

Beleodaq® benefits from industrial protection until at least 2026. Its protection (commercial exclusivity) is furthermore strengthened by its status as an orphan drug in Europe and the United States.

Developed to tackle relapsed or refractory peripheral T-cell lymphoma (PTCL), with a positive Phase II, in early July 2014 Beleodaq® obtained New Drug Authorization from the Food and Drug Administration for this indication. This authorization was given within the context of an accelerated registration program which conditionally approves a drug designed for the treatment of a life-threatening disease based on predictive elements of a clinical benefit. The registration is based on the results of the BELIEF clinical trial of 129 patients suffering from peripheral T-cell lymphoma, resistant or in relapse after at least one initial systemic treatment which showed a response level of 25% with a median duration of response of 8.3 months and a good tolerance profile.  Beleodaq® has been available to patients since July 2014, promoted by specialist Spectrum Pharmaceuticals oncology sales teams in the United States.

A clinical Phase III trial is planned to assess the efficacy of Beleodaq® in combination with the CHOP treatment, against CHOP in the 1st line of treatment of peripheral T-cell lymphoma (PTCL).

belinostat is also developed by Onxeo as an oral formulation. This would enable a significant extension of the intellectual property and constitute a real competitive advantage in terms of patient quality of life and compliance. Most importantly, it would be an asset to use belinostat in combination with other anti-cancer agents, which are mostly administered by systemic route. The company has undertaken an extensive preclinical program to assess the potential of some combinations and plan to file for a phase 1 clinical trial of the oral formulation by the end of 2017.

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The product

First-in-class signal-interfering (siDNA) product candidate, AsiDNA™ breaks the cycle of tumor DNA repair by interfering at the core of DNA damage, blocking multiple repair pathways, while sparing healthy cells. AsiDNA™ and its technology originate from three major French academic centers: Curie Institute, CNRS, and the Museum of Natural History. It offers a potential new treatment option for patients suffering from various types of cancer.

ENG lille

DNA damages of cancer cells are caused by cytotoxics (chemo- and radiotherapies) or occur spontaneously in the case of certain genetically unstable tumors. Cancer cells have the ability to recognize DNA damage and activate multiple repair pathways or proteins to survive DNA damages. Approaches to prevent DNA repair mechanisms have been identified as one of the most promising new avenues in cancer treatment.

AsiDNA™ is a short double-stranded DNA molecule that acts as a decoy, mimicking double-strand breaks in the cell’s native DNA. When introduced into a cell, AsiDNA™ molecules trigger false DNA break signals to activate and attract DNA repair proteins, which prevents the recruitment of repair enzymes to the site of actual DNA damage. As a result, the damage of a cell’s native DNA remains unrepaired. As cancer cells have lost the ability to regulate cell division, they will continue dividing with damaged DNA, ultimately leading to cancer cell death (mitotic death). Healthy cells, on the other hand, will halt cell division until the compound is no longer present and damaged DNA can be repaired.

This approach is significantly differentiated from others in the field as it does not inhibit specific enzymes (such as the PARP-inhibitors) but targets the entire DNA repair system.


A first-in-human Phase I trial performed in metastatic melanoma demonstrated that the siDNA molecules showed good tolerance and safety when administered intra-tumorally and subcutaneously around the tumors.

Onxeo plans to initate the development of the product by the systemic route, and to assess safety and tolerance in monotherapy and in combination with other DNA-damaging agents in various solid tumors. In parallel, the Company is also optimizing the manufacturing process.

Recent positive preclinical proof-of-concept results confirmed AsiDNA™ activity via systemic administration in a murine model of triple negative breast cancer (TNBC). The Company now prepares a phase I trial via systemic (intravenous) administration, expected to be submitted to the regulatory authorities by the end of 2017.

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