Onxeo Buys DNA Repair Technology; Delays PhIII For Validive
This article was originally published in Scrip
Paris, France-based Onxeo, a company focused on the development of orphan oncology therapeutics, has acquired privately held, clinical-stage biopharmaceutical company DNA Therapeutics for its signal-interfering DNA repair technology and first-in-class molecule.
Onxeo is acquiring DNA Therapeutics for an upfront payment of €1.7m in common shares in a deal expected to close by the end of March. Additional payment to DNA Therapeutics shareholders, in the form of milestones, will include €1m in shares upon successful initiation of a Phase II trial in a selected indication of lead molecule, DT01; as well as royalty payments on future commercial sales, up to €25m per indication developed and approved using the company's technology. DNA Therapeutics' historical shareholders have also agreed to invest €1m in Onxeo.
DT01 is a first-in-class signal-interfering DNA (siDNA) molecule with potential as a treatment for genetically unstable or resistant types of cancers. In a first-in-human Phase I/IIa trial in metastatic melanoma, DT01 demonstrated good local, systemic, and immune tolerance and safety when administered intra-tumorally and subcutaneously around the tumors. Onxeo now plans to initiate the development of DT01 by the systemic route, to assess safety and tolerance of the product both in monotherapy and in combination with other DNA-damaging agents in various solid tumors.
CEO Judith Greciet told Scrip the mechanism of action of DT01 is a "hotspot" for future cancer therapy, with a number of academic institutions looking into its potential. She added that the technology has "logical" potential alone and in combination with other cancer treatments, such as immunotherapies.
However, she said at this point in time the company needs to look more closely at the technology and where and how it can be used before big decisions on specific indications are made.
"The mechanism is extremely relevant in cancer," she said, adding that the mode of action for siDNA is "universal" with potential in various indications.
The siDNA repair antagonist technology developed by DNA Therapeutics breaks the cycle of cancer DNA repair activities by interfering at the core of DNA damage repair pathways, while sparing healthy cells. The technology was invented by Dr. Marie Dutreix, research director at The French National Centre for Scientific Research (CNRS), and Jian-Sheng Sun, professor at The French National Museum of Natural History (Museum National d'Histoire Naturelle) in Paris, and further developed in Dutreix's lab at Institut Curie.
The theory behind DT01 is that the short double-stranded DNA molecule acts as a decoy, providing a false DNA break signal to attract DNA repair proteins. This prevents the recruitment of repair enzymes to the site of actual DNA damage. As DNA damage is not repaired, cancer cells, which are not able to regulate cell division, ultimately die.
Onxeo's CEO noted that this technology acquisition fits with the company's overall strategy of internal innovation and the leveraging of external technology opportunities.
She added, though, that while this new technology will be the company's immediate focus, alongside its existing pipeline, Onxeo will seek other corporate development deals in the future. Still, finding the right dynamic arrangement is "easy to say, but harder to do," Greciet said.
Delay For Validive
Meanwhile, Onxeo announced that it won't be pursuing Phase III development of one its three lead products, Validive (clonidine lauriad), which is being developed for the treatment of severe oral mucositis in patients suffering from head and neck cancer, without a partner.
Onxeo noted that throughout 2015 it held meetings with both European and US regulators about the route to market for Validive. However, the company said in a Feb. 29 statement that "despite recognition from both agencies of Validive's interest and value to patients, these discussions have confirmed that two Phase III clinical trials will be required for registration in the US, which makes the further clinical program significantly longer and more costly than expected."
Greciet said conducting two Phase III studies for one product was a "heavy requirement for a company like us in terms of cash and resource dedication." She noted that discussions for this product are ongoing and that the company will "do its best" to secure a partner to share the economic burden and risk with for Validive.