BUENOS AIRES, Argentina – Andes Biotechnologies SA, a cancer drug company from Santiago, Chile, is planning to cement its presence in the U.S. as it begins to emerge from stealth mode. With $18 million in hand from home-grown Chilean venture funds, angel investors and family offices, Andes' move north is designed to help open up new funding opportunities and put the company closer to its clinical work with its most advanced antisense oligonucleotide candidate, Andes-1537.

"Almost all of our work, especially the clinical trials, is being done in the U.S.," Andes' chief operating officer, Cristian Hernández-Cuevas, told BioWorld. "We are quite active there. I don't think we are going to relocate the whole company; we are probably going to create a subsidiary, or something like that.

"I foresee future rounds of funding taking place in the U.S," he added. "We are slowly going to start a more active presence there."

Andes already rents "a really, really tiny lab in San Francisco," Hernández-Cuevas said, which handles samples for clinical trials and drug supplies. The company is in the process of finalizing the legal work – a stage it hopes to complete this year – to have a formal presence in the U.S. That will also put Andes closer to investors in Silicon Valley and the Bay Area, paving the way for better funding opportunities.

Andes was founded in 2008 by a team of Chilean researchers led by Pablo Valenzuela, who was previously a co-founder and vice president of R&D at Chiron Corp., which was later acquired by Novartis AG. Its focus is on developing antisense oligonucleotide candidates that bind to long noncoding mitochondrial RNA. with the aim of inducing apoptosis of cancer cells. Preclinical studies showed that approach was capable of selectively destroying cancer cells in in vitro and in vivo models and, notably, required no special delivery formulation.

Andes-1537, a phosphorothioate oligonucleotide that binds, or hybridizes, with the long noncoding mitochondrial RNA, was identified as the lead candidate. It "kills specifically tumor cells, without affecting normal cells," Hernández-Cuevas explained. "That is based on a discovery we made that tumor cells have a special expression of the target, a long noncoding mitochondrial RNA that is differentially expressed in tumor cells; normal cells express it in a different way and our drug targets specifically the tumor-related mitochondrial RNA."

The drug currently is in phase I testing under an IND cleared by the FDA in September 2015. According to Cortellis Clinical Trials Intelligence, the trial is designed as an open-label, dose-escalating safety and tolerability study in patients with advanced unresectable solid tumors that are refractory to standard therapy.

So far, according to Hernández-Cuevas, the trials have been positive and the company is confident the drug "is very safe."

"We have surpassed our predicted, therapeutic dose in humans, and we haven't seen any side effects yet," Hernández-Cuevas said. "That's very promising." He added that phase Ia testing is nearly complete, with data from the upcoming phase Ib trials expected to be available in 18 months to two years.

The next phase of trials will focus on three or four types of cancer and will involve solid, highly vascularized, tumors that appear in the liver, kidneys and pancreas. Tumors like those, in the peritoneal cavity, are particularly good candidates because the distribution of Andes-1537 is strong in that area of the body. However, in the future, it could also be used to treat lung or colon cancers.

Funded by Chileans

Andes-1537 is the furthest along in Andes' pipeline, where a number of other drug candidates wait their turn. That includes work on prostate, melanoma, breast cancer and also some preliminary work on hematological malignancies. "We are trying to find a super-specific drug based on the same principles as the antisense oligonucleotide hybridization for [these] types of cancers," Hernández-Cuevas said. "These are early stage works."

Andes is in a unique position, in that its efforts – and backing – come entirely from Chile. For Hernández-Cuevas, who has an MBE, or masters in bioscience enterprise, from the University of Cambridge, that is a point of pride, but also serves as a reminder that the company must continue to set its sights internationally.

"The technology was discovered and developed here, and that's quite unique because [Chile] is out of the radar, totally," he said. "[Andes] has been funded by Chileans and that is quite unusual for the Chilean ecosystem. We don't have the experience or previous expertise in the investment community, really, to invest in these high-risk, high-reward opportunities.

"Although this has been a Chilean-led endeavor, we haven't lost site of the international, or global, playing field where we need to perform," he added. "We are constantly traveling, we are meeting with key people, and we are hiring the best lawyers that we can afford.

"We have a Chilean identity but a global footprint."