Empirico, Ionis collaborate to develop antisense oligonucleotide therapeutics

Under the three-year collaboration, Empirico will use its Precision Insights Platform to determine therapeutic targets for indications and tissues, which are responsive to antisense technology.

The Precision Insights Platform, which was purpose-built for therapeutic target discovery, integrates one of the largest datasets of its kind in the world to assess the role of genes and proteins in health and disease and find opportunities for new therapeutic interventions.

As per terms of the deal, Ionis has the chance to advance ten targets identified by Empirico, as well as take responsibility for all preclinical and clinical development activities.

The platform combines expert data curation, customised data models, and statistical and machine learning algorithms to systematically generate, interrogate, and prioritise high-confidence therapeutic hypotheses, which are then validated experimentally.

Both firms will also integrate human genetics evidence into ongoing efforts with existing Ionis programmes, comprising work on target validation, indication and biomarker selection, and patient stratification.

As part of the agreement, Empirico has secured a $10m equity investment from Ionis. Empirico will also receive up to $30m based on the achievement of certain operational and preclinical milestones.

Empirico is provided with an option to secure more than $620m for the achievement of clinical development, regulatory and commercial milestones, and royalties on net sales.

In addition, the deal will allow Empirico to licence, develop and commercialise an Ionis development candidate aimed at collaboration target for which Ionis will secure milestone payments and royalties on net sales.

Empirico CEO Omri Gottesman said: “Empirico’s approach to human genetics provides a much-needed opportunity to improve the success rate of drug discovery and development by leveraging experiments of nature.

“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.”

In April 2018, Biogen entered into a ten-year collaboration agreement with Ionis Pharmaceuticals for the development of antisense drug candidates to treat a range of neurological diseases.