2014 Highlights and perspectives
Expansion of key orphan oncology programs
Company’s key orphan oncology programs Validive®, Livatag® and Beleodaq® are high added-value programs focusing on significant and unmet medical needs, showing strong sales potential. In 2014, the team has reached important development milestones, which contributed to increase the overall company value:
Beleodaq® (belinostat)
- In July 2014, the Food and Drug Administration (FDA) granted conditional marketing authorization in the USA for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL) in 2nd line treatment after failure of standardized chemotherapy used in first line (CHOP protocol). As per contract, this approval has triggered a $25 million milestone payment from Spectrum Pharmaceuticals which was received in November. The commercialization of Beleodaq® by the Spectrum Pharmaceuticals’ oncology sales team has started in late July 2014 with positive level of sales estimated to around 5M USD, resulting in first royalties for Onxeo.
- Following this conditional marketing authorization in 2nd-line PTCL, a Beleodaq® Phase III trial is planned to be initiated H1 2016 for the same PTCL patients but in first line of treatment, combined with CHOP, expanding therefore the indication from 2nd to 1st line of treatment.
- Prior to this Phase III initiation, a phase I study with the combined treatment Beleodaq® + CHOP (BelCHOP) is ongoing to determine the optimal dose of the combination and its safety profile. This study is expected to recruit up to 28 patients by Q3 2015 and is the preliminary step of the phase III trial. .
- Beyond PTCL, Beleodaq® profile and first data advocate for the development of new promising orphan oncology indications. The company is currently discussing with its partner to finalize future product development plans.
Validive® (clonidine Lauriad®)
- End of October 2014, Onxeo reported positive preliminary top-line results of the large international Phase II trial comparing the efficacy and safety of Validive® versus placebo in the prevention of oral severe mucositis in 183 head and neck cancer patients. Validive® has shown to reduce of 16% (absolute value) the incidence of severe oral mucositis in treated patients versus placebo, to delay the occurrence of the mucositis and allow a higher intensity of radiation before appearing. The safety profile of Validive® was very good with no major safety issue.
- The study’s advisory committee, made up of internationally recognised experts, has confirmed that these data were supportive to further pursue Validive® development plan and recommended advancing its development program through a Phase III study on the same patient population. This Phase III trial evaluating Validive®’s efficacy is being prepared and the Company plans to initiate it in 2015.
- Validive®’s development will benefit from the “fast track” status obtained from the Food and Drug Administration in January 2014. This status is granted for drugs developed for life-threatening diseases for which the medical need is strong. It facilitates interactions with the FDA and optimizes review duration during development and registration.
- Oral mucositis is a very severe adverse effect of chemoradiation therapy which, at a severe stage, is highly painful and debilitating. There is no current preventing option for such disease and the need for such treatment is particularly high.
Livatag® (doxorubicin Transdrug™)
- Active recruitment in the Phase III trial ReLive in primary liver cancer, with 40 % of planned patients already randomized. In 2015, the international expansion of ReLive will be supported by broadening the ReLive trial into new regions such as MENA (Middle East North Africa) countriesto optimize recruitment rate.
- The product safety profile has been so far confirmed, twice again in 2014, by the trial’s Data Safety Monitoring Board who meets twice a year to review all the safety data of the treated patients.
- Livatag® also obtained FDA “fast track” status for treating hepatocellular carcinoma as a second-line treatment after sorafenib In addition, the product’s patent protection was reinforced in February 2014 with a new family of patents protecting its specific dosing regimen, and issued by the European Patent Office. This second patent family significantly strengthens and extends the product’s patent protection in Europe until 2032 against the marketing of generics.
Major development for the Company, the creation of Onxeo, resulting from the merger of BioAlliance Pharma and Topotarget
2014 was a cornerstone year for the Company which became Onxeo in August, through the merger between BioAlliance Pharma SA and Topotarget A/S. This was a major first step in the company growth strategy implementation .
This operation has enabled the company to gain critical mass in orphan oncology, its strategic field, as a European player with competitive advantages:
- An enlarged and advanced clinical pipeline,
- A reinforced team with strong scientific skills,
- A US based co- development and commercial partner on Beleodaq®,
Onxeo is listed on both Euronext and Nasdaq OMX in Copenhagen. The market capitalization has reached the 250 M€ threshold, which positions the company among the leading biotechs in Europe.