CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) and AstraZeneca (NASDAQ:AZN) have received Fast Track designation from the U.S. Food and Drug Administration (FDA) for their collaborative drug candidate, eplontersen. This designation is aimed at expediting the development and review process for treatments of serious conditions with unmet medical needs, such as transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.
ATTR-CM is a fatal condition caused by accumulation of misfolded transthyretin (TTR) protein in the heart, leading to progressive heart failure. It affects an estimated 300,000 to 500,000 patients globally and can result in death within three to five years of onset. Eplontersen, which is designed to inhibit the production of TTR protein, is currently being evaluated in the CARDIO-TTRansform Phase 3 study, the largest of its kind for this patient population with more than 1,400 participants. Results from the study are expected as early as 2025.
The drug has already been approved in the United States under the brand name WAINUA™ for treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Ionis and AstraZeneca are also seeking regulatory approval in Europe and other regions for this indication. WAINUA has been granted Orphan Drug Designation in the U.S. and the EU for the treatment of ATTR.
Eugene Schneider, M.D., executive vice president and chief clinical development officer at Ionis, expressed confidence in eplontersen's potential as a transformative treatment for ATTR-CM, highlighting the significance of the FDA's Fast Track designation. Sarah Walters, vice president at AstraZeneca, reiterated their commitment to addressing unmet needs in amyloidosis through innovation.
The Fast Track status underscores the urgent need for new therapies in the treatment of ATTR-CM and could potentially shorten the time it takes for eplontersen to reach patients if it proves to be safe and effective.
This news is based on a press release statement from Ionis Pharmaceuticals.
InvestingPro Insights
Amidst the promising developments in the treatment of ATTR-CM by Ionis Pharmaceuticals and AstraZeneca, it's worth noting AstraZeneca's current financial health and market performance. According to real-time data from InvestingPro, AstraZeneca (NASDAQ:AZN) boasts a sizeable market capitalization of 192.19 billion USD, reflecting its substantial presence in the pharmaceutical industry. The company's P/E ratio stands at 34.76, with an adjusted P/E ratio for the last twelve months as of Q4 2023 at 17.95, indicating a valuation that may be attractive relative to near-term earnings growth.
Further bolstering investor confidence, AstraZeneca has maintained a gross profit margin of 82.45% over the last twelve months as of Q4 2023. This impressive margin showcases the company's ability to manage its costs effectively and hints at robust financial health. Additionally, AstraZeneca has been a reliable dividend payer, with a history of maintaining dividend payments for 31 consecutive years—a testament to its financial stability and commitment to shareholder returns.
For those looking to delve deeper into AstraZeneca's financials and market potential, InvestingPro offers additional insights. There are 14 more InvestingPro Tips available, which include an analysis of the company's stock price volatility, debt levels, and cash flow capabilities. Subscribers to InvestingPro can access these valuable tips to make more informed investment decisions. Use coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription and gain comprehensive insights into AstraZeneca's market standing and future prospects.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.