Rare Daily Staff

Ionis Pharmaceuticals entered into two separate drug discovery and development agreements intended to leverage its expertise in antisense drug development, which targets RNA.

The company entered into a three-year collaboration with Empirico to use the company’s Precision Insights Platform, which incorporates biological data sets, human genetics, and advanced algorithmic approaches, to identify therapeutic targets for indications and tissues that are amenable to Ionis’ antisense technology.

Under the terms of the agreement, Ionis can advance up to ten targets identified by Empirico and assume responsibility for all preclinical and clinical development activities. Empirico and Ionis will also work together to incorporate human genetics evidence into ongoing efforts with existing Ionis programs, including work on target validation, indication and biomarker selection, and patient stratification.

“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,” said Omri Gottesman, CEO of Empirico. “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.”

As part of this new collaboration, Ionis has made a $10 million equity investment in Empirico, with additional near-term commitments of up to $30 million based on operational and preclinical milestones. Empirico will be eligible to receive in excess of $620 million for the achievement of clinical development, regulatory and commercial milestones, and royalties on net sales.

Empirico also has the option to license, develop, and commercialize an Ionis development candidate directed toward a collaboration target for which Ionis will receive milestone payments and royalties on net sales.

In connection with the collaboration, Empirico also announced the closing of its $17 million Series A-2 financing, led by Ionis and with participation by DCVC Bio and Neotribe Ventures. This was a follow-on round to Empirico’s $12.5 million Series A financing, co-led by DCVC Bio and Neotribe Ventures and completed In November 2018.

In a separate deal, Ionis entered into a licensing and collaboration with agreement with Aro Biotherapeutics that will allow Ionis to use Aro’s Centyrin technology to develop targeted cell- and tissue- specific delivery of antisense oligonucleotides (ASOs).

Centyrins are small, structurally simple, ultra-stable, highly soluble proteins. These characteristics enable the discovery of medicines with new mechanisms of action for cancer and other devastating diseases. This first-of-its-kind combination of properties is designed to address unmet medical needs by targeting drug payloads in high concentration to the site of disease, while lowering the toxicity to non-target organs.

Under the terms of the agreement, the companies will collaborate to create unique ASO-Centyrin conjugates that are designed to achieve tissue specific, therapeutically effective gene knockdown in extra-hepatic tissues with systemic administration.

“This important collaboration combines Aro’s strengths in protein engineering with Ionis’ leading expertise in the science of antisense technology,” said Aro Biotherapeutics co-founder and Chief Scientific Officer Karyn O’Neil. “Ionis will leverage this unique technology to develop novel ASO-Centyrin combination drug candidates targeting a variety of diseases with high unmet medical need.”

Under the terms of the agreement, the two companies will work towards advancing a defined number of ASO-Centyrin drug conjugates. Aro will be responsible for Centyrin discovery and will collaborate with Ionis to create lead ASO-Centyrin drug conjugates for further development.

Ionis will be responsible for development and will have global commercialization rights for each ASO-Centyrin drug conjugate. The parties will also collaborate on additional discovery programs.

Aro will receive an upfront cash payment, funding to support R&D efforts, and payments associated with the achievement of specific development and commercial milestones, up to $1.4 billion. In addition, Aro will receive royalties on net sales.

Photo: Aro Biotherapeutics co-founder and Chief Scientific Officer Karyn O’Neil

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