Ionis announces expanded licensing agreement with Otsuka in Asia Pacific for investigational medicine donidalorsen in hereditary angioedema

Rhea-AI Summary

Ionis Pharmaceuticals has entered an expanded licensing agreement with Otsuka Pharmaceutical for the investigational RNA-targeted medicine donidalorsen, aimed at treating hereditary angioedema (HAE).

Under the agreement, Otsuka gains exclusive rights to commercialize donidalorsen in the Asia-Pacific region while also handling regulatory filings and localized development. Ionis will continue to manage global development and plans to submit a New Drug Application to the FDA this year for U.S. approval, aiming for an independent U.S. launch.

Previously, Otsuka secured licensing for Europe and is preparing a Marketing Authorization Application to the EMA this year. Ionis will receive a $20 million upfront payment and milestone payments from Otsuka, along with tiered royalties.

Recent positive results from Phase 3 trials were presented at the EAACI Annual Congress and published in The New England Journal of Medicine.

Positive

• Ionis will receive a $20 million upfront payment from Otsuka.
• Milestone payments based on regulatory and sales targets will be made to Ionis.
• Ionis is eligible for tiered royalties under the agreement.
• Positive Phase 3 trial results for donidalorsen were recently presented.
• Ionis maintains primary responsibility for global development and plans a U.S. FDA submission this year.
• Otsuka brings expertise in regional regulations and rare disease medicine commercialization.

Negative

• Commercialization for donidalorsen in Asia-Pacific and Europe is dependent on Otsuka, which may introduce uncertainties.
• Financial reliance on milestone and royalty payments from Otsuka, which are contingent upon regulatory and sales success.
• Ionis faces the risk of delays or rejections in regulatory approvals both in the U.S. and internationally.

Financial Analyst

The expanded licensing agreement between Ionis Pharmaceuticals and Otsuka Pharmaceutical Co. offers a significant boost to Ionis' financial outlook. The $20 million upfront payment, along with potential milestone payments and royalties, provides immediate and future revenue streams. This deal effectively de-risks Ionis' financial exposure in the Asia-Pacific region while allowing them to focus more of their resources on the U.S. market.

For retail investors, it's important to understand that milestone payments are contingent on achieving regulatory and sales targets, which introduces some risk. However, the recent positive Phase 3 results for donidalorsen reduce the uncertainty regarding these milestones. The financial benefits from this deal are likely to support Ionis' valuation in the short term and add to its cash reserves, which could be important for future R&D activities and market expansions.

Key Takeaway: This licensing agreement not only provides immediate financial relief but also strategically diversifies Ionis' revenue streams, which is beneficial for their long-term financial health.

Market Research Analyst

From a market perspective, this deal with Otsuka significantly bolsters Ionis' geographic reach. Otsuka's established presence and expertise in the Asia-Pacific market will expedite the commercialization process of donidalorsen, ensuring faster access to a potentially lucrative market. This partnership also strategically complements Ionis' focus on the U.S. market, where they plan to independently launch donidalorsen pending FDA approval.

Given that the Asia-Pacific region has diverse regulatory environments, Otsuka's local knowledge and established commercial infrastructure are invaluable. This collaboration not only increases the likelihood of successful regulatory approvals but also ensures efficient market penetration. With positive Phase 3 results already in hand, the timing of this agreement is optimal, leveraging recent clinical successes to boost market confidence.

Key Takeaway: The partnership with Otsuka leverages their regional expertise to accelerate market entry, reducing the time to market and potentially capturing early market share.

Medical Research Analyst

The positive Phase 3 results for donidalorsen are a significant milestone for Ionis, given the competitive nature of the rare disease market. Hereditary angioedema (HAE) is a niche market with significant unmet medical needs and successful clinical outcomes are important for market acceptance and adoption. The robust data from the OASIS-HAE and OASISplus studies, presented at a reputable conference and published in a leading medical journal, add scientific credibility to the drug's efficacy and safety profile.

This clinical success not only supports the regulatory filings in the U.S., Europe and Asia-Pacific but also positions donidalorsen as a potentially best-in-class therapy for HAE. Investors should note that regulatory submissions are imminent and successful approvals could lead to rapid market uptake, especially given the current lack of effective prophylactic treatments for HAE.

Key Takeaway: The solid clinical data provide a strong foundation for regulatory approvals and market acceptance, positioning donidalorsen as a potentially transformative treatment for HAE.

• Otsuka will be responsible for commercialization efforts for donidalorsen across both Asia Pacific and Europe
• Ionis plans to independently bring donidalorsen to U.S. patients, if approved

CARLSBAD, Calif., June 18, 2024  /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has entered into a license agreement with Otsuka Pharmaceutical Co., Ltd. (Otsuka) under which Otsuka obtains exclusive rights across the Asia-Pacific region for donidalorsen, an investigational RNA-targeted prophylactic medicine for hereditary angioedema (HAE). Ionis will maintain primary responsibility for the development of donidalorsen, while Otsuka will be responsible for territory-specific development, regulatory filings and commercialization in the Asia-Pacific region and Europe.

Ionis plans to file a New Drug Application with the U.S. Food and Drug Administration (FDA) this year, and will independently launch donidalorsen in the U.S., if approved. Ionis and Otsuka previously announced a licensing agreement for donidalorsen in Europe, and Otsuka is also preparing to submit a Marketing Authorization Application to the European Medicines Agency this year.

"Ionis and Otsuka share a steadfast commitment to bring donidalorsen to as many people living with HAE as possible," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "We look forward to working alongside Otsuka to advance regulatory discussions across Europe and the Asia-Pacific region based on our positive Phase 3 results for donidalorsen, which were presented late last month. In the U.S., we've built a robust commercial infrastructure in preparation for our near-term anticipated launches for olezarsen in familial chylomicronemia syndrome and donidalorsen in HAE."

Under the terms of the Asia-Pacific agreement, Ionis will receive a $20 million upfront payment and milestone payments based on achievement of regulatory and sales targets. Ionis is also eligible to earn tiered royalties, with similar economic terms to the previous agreement for Europe. Originating in Japan, Otsuka brings deep knowledge of regional and local regulations across the Asia-Pacific region, as well as global expertise in delivering rare disease medicines to patients and a robust commercial infrastructure.

Ionis recently reported positive results from the Phase 3 OASIS-HAE and OASISplus studies, presented at the 2024 European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress in Valencia, Spain and published in The New England Journal of Medicine.  

About Hereditary Angioedema (HAE)
HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect more than 20,000 patients in the U.S. and Europe. In the U.S., doctors frequently use prophylactic treatment approaches to prevent and reduce the severity of HAE attacks in patients. 

About Donidalorsen
Donidalorsen is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to target prekallikrein (PKK), which plays an important role in activating inflammatory mediators associated with acute attacks of hereditary angioedema (HAE). By reducing the production of PKK, donidalorsen could be an effective prophylactic approach to preventing HAE attacks. 

About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients.

To learn more about Ionis, visit Ionis.com and follow us on X (Twitter) and LinkedIn.

Ionis Forward-looking Statements
This press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of our commercial medicines, donidalorsen, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2023, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals^® is a registered trademark of Ionis Pharmaceuticals, Inc.

Ionis Investor Contact:
D. Wade Walke, Ph.D.
ir@ionis.com
760-603-2331

Ionis Media Contact:
Hayley Soffer
media@ionis.com
760-603-4679

 

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SOURCE Ionis Pharmaceuticals, Inc.

FAQ

What is donidalorsen?

Donidalorsen is an investigational RNA-targeted prophylactic medicine for hereditary angioedema (HAE).

When will Ionis submit donidalorsen for FDA approval?

Ionis plans to submit a New Drug Application to the FDA this year.

What are the financial terms of the Ionis and Otsuka agreement?

Ionis will receive a $20 million upfront payment, milestone payments, and tiered royalties from Otsuka.

Where were the Phase 3 trial results for donidalorsen presented?

The Phase 3 trial results were presented at the 2024 EAACI Annual Congress and published in The New England Journal of Medicine.

What regions are covered under the Ionis and Otsuka licensing agreement?

The agreement covers the Asia-Pacific region for donidalorsen commercialization by Otsuka.

What responsibilities does Otsuka have under the agreement?

Otsuka is responsible for territory-specific development, regulatory filings, and commercialization in the Asia-Pacific region and Europe.

Will Ionis independently launch donidalorsen in the U.S.?

Yes, Ionis plans to independently launch donidalorsen in the U.S., if approved by the FDA.