Eplontersen sodium - Generic Drug Details

What are the generic sources for eplontersen sodium and what is the scope of freedom to operate?

Eplontersen sodium is the generic ingredient in one branded drug marketed by Astrazeneca Ab and is included in one NDA. There are four patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Eplontersen sodium has two hundred and thirty-six patent family members in thirty-six countries.

One supplier is listed for this compound.

Summary for eplontersen sodium

International Patents:	236
US Patents:	4
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
DailyMed Link:	eplontersen sodium at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for eplontersen sodium

Constraining patent/regulatory exclusivity:

TREATMENT OF THE POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS IN ADULTS

Dosage:

SOLUTION;SUBCUTANEOUS

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Pharmacology for eplontersen sodium

Drug Class	Antisense Oligonucleotide Transthyretin-directed RNA Interaction
Physiological Effect	Decreased RNA Integrity Increased Protein Breakdown

Anatomical Therapeutic Chemical (ATC) Classes for eplontersen sodium

N07XX	Other nervous system drugs
N07X	OTHER NERVOUS SYSTEM DRUGS
N07	OTHER NERVOUS SYSTEM DRUGS
N	Nervous system

US Patents and Regulatory Information for eplontersen sodium

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Astrazeneca Ab	WAINUA (AUTOINJECTOR)	eplontersen sodium	SOLUTION;SUBCUTANEOUS	217388-001	Dec 21, 2023		RX	Yes	Yes				Y
Astrazeneca Ab	WAINUA (AUTOINJECTOR)	eplontersen sodium	SOLUTION;SUBCUTANEOUS	217388-001	Dec 21, 2023		RX	Yes	Yes			Y	Y
Astrazeneca Ab	WAINUA (AUTOINJECTOR)	eplontersen sodium	SOLUTION;SUBCUTANEOUS	217388-001	Dec 21, 2023		RX	Yes	Yes			Y	Y
Astrazeneca Ab	WAINUA (AUTOINJECTOR)	eplontersen sodium	SOLUTION;SUBCUTANEOUS	217388-001	Dec 21, 2023		RX	Yes	Yes

International Patents for eplontersen sodium

Country	Patent Number	Title	Estimated Expiration
Russian Federation	2015151204	КОМПОЗИЦИИ И СПОСОБЫ
Israel	242126	תרכובות המכילות אוליגונוקליאוטיד שונה וקבוצה מצומדת (Compounds comprising a modified oligonucleotide and a conjugate group)
Slovenia	2992009
Brazil	112015027377	COMPOSTOS OLIGOMÉRICOS COM GRUPOS DE CONJUGADO, COMPOSIÇÕES COMPREENDENDO OS REFERIDOS COMPOSTOS E USOS DOS MESMOS

Market Dynamics and Financial Trajectory for Eplontersen Sodium

Introduction to Eplontersen Sodium

Eplontersen, formerly known as IONIS-TTR-LRx, is a ligand-conjugated antisense investigational medicine developed by Ionis Pharmaceuticals in collaboration with AstraZeneca. This drug is designed to reduce the production of transthyretin (TTR) protein, which is crucial for treating both hereditary and non-hereditary forms of TTR amyloidosis (ATTR), including amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN)[1][4].

Clinical Trials and Development

Eplontersen is currently in Phase III clinical trials, with Ionis leading the conduct of these trials globally. The trials include the NEURO-TTRansform study for patients with hereditary ATTR amyloidosis with polyneuropathy and the CARDIO-TTRansform study for those with cardiomyopathy. These trials have shown promising results, with eplontersen continuing to demonstrate improvement in ATTRv-PN through 85 weeks[2].

Collaboration and Commercialization

The collaboration between Ionis and AstraZeneca is pivotal for the development and commercialization of eplontersen. In the United States, both companies will jointly develop and commercialize the drug, while AstraZeneca will have exclusive rights to commercialize it in the rest of the world, except for certain countries in Latin America[1][4].

Responsibilities and Roles

• Ionis: Will continue to lead the global Phase III clinical trials, manufacture and supply eplontersen for ongoing clinical trials, and share responsibility for medical affairs and commercial activities in the U.S.
• AstraZeneca: Will be responsible for commercial supply, with transition timing to be agreed upon by both companies. AstraZeneca will also handle commercialization outside the U.S., except in specified Latin American countries[4].

Financial Considerations

The financial arrangement between Ionis and AstraZeneca is comprehensive and includes several key components:

• Upfront Payment: AstraZeneca will pay Ionis an upfront payment of $200 million.
• Conditional Payments: Additional payments of up to $485 million are conditional upon regulatory approvals.
• Sales-Related Milestones: AstraZeneca will pay up to $2.9 billion in sales-related milestones based on sales thresholds between $500 million and $6 billion.
• Royalties: AstraZeneca will also pay royalties in the range of low double-digit to mid-twenties percentage, depending on the region[1].

Market Potential and Penetration

The collaboration is expected to enable faster and deeper market penetration into the growing global TTR amyloidosis market. The combined expertise of Ionis in RNA-targeted therapeutics and AstraZeneca's global scale and leadership in cardiovascular drug development and commercialization will be crucial in reaching a broader patient population[4].

Market Size and Growth

The TTR amyloidosis market is growing, driven by the increasing prevalence of ATTR-CM and ATTR-PN. ATTR-CM is a systemic, progressive, and fatal condition leading to progressive heart failure and death within four years from diagnosis. Hereditary ATTR-PN is another significant indication, and the companies plan to seek regulatory approval for this first[1].

Revenue and Financial Projections

Ionis has seen significant revenue growth driven by partner payments, including those related to eplontersen. For the second quarter and first half of 2023, Ionis reported a 40% and 16% increase in revenue, respectively, compared to the same periods in the previous year. The company's financial health, with $2.4 billion in cash and short-term investments as of June 30, 2023, supports continued investments in advancing its pipeline, including eplontersen[2].

Ionis' Financial Performance

• Revenue: Increased by 40% and 16% for the second quarter and first half of 2023, respectively.
• Operating Expenses: Increased due to investments in advancing the pipeline and go-to-market activities.
• Cash and Short-Term Investments: $2.4 billion as of June 30, 2023, ensuring continued investments[2].

Regulatory Milestones

The companies are on track to file a new drug application (NDA) with the U.S. Food and Drug Administration (FDA) by the end of 2022 for hereditary ATTR-PN. The FDA has set a PDUFA date of December 22, 2023, for eplontersen, marking a critical regulatory milestone[1][2].

Impact on AstraZeneca’s Financials

The transaction is expected to be neutral to AstraZeneca’s core earnings in 2021 and will be accounted for as an intangible asset acquisition. The upfront payment and potential future milestone payments will be capitalized into the intangible asset as they are recognized. This collaboration aligns with AstraZeneca’s long-term growth strategy, particularly in the cardiovascular and rare disease therapy areas[1][3].

Key Takeaways

• Collaborative Development: Ionis and AstraZeneca are jointly developing and commercializing eplontersen.
• Financial Arrangements: Include an upfront payment, conditional payments, sales-related milestones, and royalties.
• Market Potential: Significant growth potential in the TTR amyloidosis market.
• Regulatory Progress: On track for NDA filing and FDA approval.
• Financial Health: Ionis and AstraZeneca are well-capitalized to support the development and commercialization of eplontersen.

FAQs

What is eplontersen, and how does it work?

Eplontersen is a ligand-conjugated antisense investigational medicine designed to reduce the production of transthyretin (TTR) protein, treating both hereditary and non-hereditary forms of TTR amyloidosis.

Who are the key partners in the development and commercialization of eplontersen?

Ionis Pharmaceuticals and AstraZeneca are the key partners in the development and commercialization of eplontersen.

What are the current clinical trial stages for eplontersen?

Eplontersen is currently in Phase III clinical trials, with Ionis leading the conduct of these trials globally.

What are the financial terms of the collaboration between Ionis and AstraZeneca?

The collaboration includes an upfront payment of $200 million, conditional payments of up to $485 million, sales-related milestones of up to $2.9 billion, and royalties in the range of low double-digit to mid-twenties percentage.

What is the expected regulatory timeline for eplontersen?

The companies plan to file an NDA with the FDA by the end of 2022, with a PDUFA date set for December 22, 2023.

Cited Sources

1. AstraZeneca and Ionis sign deal to develop and commercialise eplontersen. AstraZeneca. Retrieved December 17, 2024.
2. Ionis reports second quarter 2023 financial results. Ionis Pharmaceuticals. Retrieved December 17, 2024.
3. Annual Report 2022. AstraZeneca. Retrieved December 17, 2024.
4. Ionis and AstraZeneca to develop and commercialize eplontersen. Ionis Pharmaceuticals. Retrieved December 17, 2024.
5. Fixed-income investor update | AstraZeneca. AstraZeneca. Retrieved December 17, 2024.

Last updated: 2024-12-17