Profile/strategy

Profile

Founded in 1997, and listed on Euronext Paris since 2005 and on Nasdaq Copenhagen in 2014, Onxeo designs, develops and brings to market innovative drugs for the treat­ment of cancer and its associated pathologies, more specifically for severe and rare orphan-status diseases.

Onxeo focuses its therapeutic strategy on fighting drug resistance which poses ever-greater challenges to treatment, further more so in orphan cancers. To do so, the Company uses innovative and patented technologies to develop tumor DNA-targeting drugs that enable targeted action on the ability of tumor cells to repair their damaged DNA, or their ability to resist to other anti-cancer treatments .

Onxeo has chosen to focus on orphan diseases which enables a targeted, synergistic and streamlined approach to developing these drugs, both in terms of development costs and time.

Onxeo’s most valuable asset is its 55 employees who have built a unique expertise in developing and registering drugs.

The Company has successfully led two programmes to registration, including with the US (FDA) and European (EMA) authorities, sold or licensed four drugs and successfully executed one international merger (Topotarget, Denmark in 2014) and one acquisition (DNA Therapeutics in 2016).

Its key expertises in preclinical and cli­nical development, regulatory affairs, as well as industrial property and business development, are major assets for Onxeo which aims to become a leading player in the field of orphan diseases in oncology by linking innovation to patient’s needs.

Strategy

Today’s Science. Tomorrow’s Cancer treatments.

Our growth strategy is driven by the development of our orphan oncology candidates which aim to answer established and unaddressed therapeutic needs in rare or resistant cancers.

Even though some of our compounds could be used in large oncology indications, we focus on products with at least one potential orphan application and long and solid IP protection as these products with high sales potential benefit from favorable pricing and reimbursement policies and accelerated registration pathways.

We seek to transform scientific opportunities into clinical breakthrough treatments through unparalleled translational expertise, developing products up to the stage where they become compelling opportunities for partners. This means moving products from preclinical stage (1 to 2 years from IND) to optimal inflexion points (usually Phase II clinical proof-of-concept). Once this proof of concept is established, we seek to license these products to pharmaceutical partners, who have the capacity to conduct the registration phases and to market them.

Our added value rests on 3 solid assets:

- A leading-edge scientific expertise in early and breakthrough research fields such as DNA-targeting ;

- Translational research capabilities (bringing a product from the preclinical stage to the proof-of-concept in man) ;

- Our ability to both identify potential targets (already 2 successful mergers / acquisitions) in order to feed our pipeline, and to conclude licensing or partnership agreements (already 4 products  sold or licensed to partners).

Onxeo’s orphan oncology products are:

Beleodaq® (belinostat): an HDAC inhibitor, already registered and sold in the U.S. in relapsed or refractory peripheral T-cell lymphoma (PTCL). We are also exploring the value of belinostat beyond PTCL, notably in an oral form, to assess its potential use in various combinations for the treatment of others liquid or solid tumors with filing for a phase I expected by the end of 2017.

AsiDNA™: A first-in-class siDNA (signal-interfering DNA) compound which has successfully undergone a proof-of-concept Phase I trial in metastatic melanoma by intratumoral administration. We are now exploring the potential of AsiDNA™ via systemic administration with the goal to file for a phase I study by the end of 2017.

Livatag® (doxorubicin Transdrug™): its ReLive Phase III study in the treatment of primary liver cancer did not meet the primary endpoint. The product however showed an efficacy similar to the active comparative arm and a good tolerability profile. The prospects of the product are currently under strategic review.

Look up the Orphan oncology products portfolio

Legacy products based on the Lauriad® technology, licensed or sold in 2017

While these products illustrate Onxeo’s undeniable development experience and skills, they are no longer at the heart of the Company’s strategy dedicated to the development of innovative drugs in orphan oncology.

Loramyc®/ Oravig®: indicated for the treatment of oropharyngeal candidiasis and Sitavig®/ Labiriad®, indicated for the treatment of recurrent herpes labialis were developed and registered in Europe and the United States. They were sold to Vectans Pharma in July 2017.

Validive® (clonidine Lauriad®): after positive results of Phase II in the treatment and prevention of radio/chemotherapy induced severe oral mucositis in patients suffering from head and neck cancer, the Company licensed Validive® to Monopar Therapeutics in September 2017.