It’s a big day for privately held Empirico Inc. as it embarks on a massive combo pharma deal with Ionis Pharmaceuticals Inc. while racking up a $12.5 million series A-2 financing.
In the collaboration, Empirico will identify the targets and Ionis can advance as many as 10 of them. The two companies plan to use human genetics data in their efforts with programs Ionis already has in the works, and that includes target validation, the indication and biomarker selection, and patient stratification.
In the three-year collaboration, Ionis made a $10 million equity investment in Empirico, with near-term commitments of as much as $30 million, based on operational and preclinical milestones. Empirico could receive more than $620 million by achieving clinical development, regulatory and commercial milestones, plus royalties on net sales.
Empirico gets the option to license, develop and commercialize an Ionis development candidate directed toward a collaboration target. Ionis will receive milestone payments and royalties on net sales based on that target.
At the heart of the collaboration is Empirico’s platform, designed to interpret the role of genes and proteins, then search for interventional strategies. The technology uses human genetics, data science and programmable biology. It has had multiple iterations since the company’s founding in a little more than two years ago, when management’s goal was to build a next-generation therapeutics company from the ground up. That meant spending a good amount of time building the platform, validating its approach and accumulating proof of concept data.
“Empirico was founded in late 2017 but we began in earnest in early 2018,” Omri Gottesman, Empirico’s CEO, told BioWorld. “We’re now on the fourth version of the platform.”
Gottesman said he strongly believes that target discovery is a discipline that relies upon translational scientists. Without them, he said, finding the right targets is the “proverbial need in a haystack” as researchers sift through billions of datasets. The company takes “an agnostic machine learning approach,” he added, as it combs for potential targets.
“Antisense oligonucleotides are an ideal translational partner for human genetics-focused target discovery, allowing us to precisely mimic or interfere with the mechanisms by which functional genetic variants influence health and disease,” he said.
The motivation behind the collaboration with Ionis, Gottesman said, is that while Empirico has the ability to identify potential targets and the capacity to pursue them, but Ionis has the “significant expertise” needed to drive a product to market. It’s in the nature of Empirico’s business model to work with others. As Gottesman noted, Empirico doesn’t have the resources to pursue everything on its own. It does however have its own internal programs that have been initiated in asthma, skin disease and glaucoma.
The collaboration and series A-2 fundraise will allow Empirico to add to its current staff roster of 14 employees, which are evenly split between temperate San Diego and intemperate Madison, Wis., where the wet lab and development group are situated. Empirico is actively hiring computational and wet lab experts and expects to have 25 employees on its roster when the year ends.
The company name is a source of pride for Gottesman.
“I’m a big fan of our name,” he said. “It’s intended to be a portmanteau for an empirical company. We are devout about everything we do.”
Today’s fundraiser for Empirico, led by Ionis with participation by DCVC Bio and Neotribe Ventures, is a follow-on round to its $12.5 million series A financing completed in November 2018. That round was co-led by DCVC Bio and Neotribe Ventures.
Shares of Ionis (NASDAQ:IONS) closed 2% upward Wednesday, before the collaboration was publicly announced, at $62.44 each.
Ionis is also collaborating with Akcea Therapeutics Inc., a majority owned affiliate of Ionis. They are in a phase II study of AKCEA-APO(a)-LRx1 (TQJ-230), an antisense drug designed to inhibit production of apolipoprotein for cardiovascular disease patients with elevated Lp(a). Recent results published in The New England Journal of Medicine showed dose-dependent reductions in Lp(a) at all doses studied, with an up to 80% reduction at highest cumulative dose regimen; about 98% of patients on 80-mg monthly dose achieved Lp(a) levels below 125 nmol/L.