Tonix Pharmaceuticals stock halted ahead of FDA approval news
On Thursday, Raymond (NSE:RYMD) James maintained a Strong Buy rating on Ionis Pharmaceuticals (NASDAQ:IONS) but slightly reduced the price target from $61.00 to $60.00. Currently trading at $31.81 with a market capitalization of $5 billion, the stock is near its 52-week low of $30.23. According to InvestingPro data, analyst targets range from $37 to $78, suggesting significant potential upside. The firm’s analyst, Gary Nachman, provided several key updates on the company’s progress and outlook.
Ionis has seen a successful U.S. launch of Wainua for the treatment of ATTR-PN, with approximately $10 million in royalties earned in the last quarter, contributing to the full year’s $20 million. The drug, which is being commercialized by AstraZeneca (NASDAQ:AZN), has gained a new-to-brand share of around 40%. It is being adopted in both centers of excellence and community settings by neurologists and cardiologists for both naïve and switch patients, with the ease of self-administration being a key factor for switching. The UK launch is ongoing, and European Union approval is anticipated following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP).
Nachman also noted that the Phase 3 trial for ATTR-CM is on track for data in the second half of 2026, with the trial progressing smoothly and no safety issues reported. In addition, Ionis is expecting to read out Phase 3 studies for Olezarsen in severe hypertriglyceridemia (SHTG) in the second half of 2025, with safety trial data expected in mid-2025. The company is actively seeking partnerships outside the U.S. for FCS and SHTG.
For Tryngolza in familial chylomicronemia syndrome (FCS), the launch is ramping up with no resistance from payers regarding the price, and Ionis’s educational efforts are increasing awareness. Donidalorsen, which is being developed for hereditary angioedema (HAE), is on track with FDA discussions, with a Prescription Drug User Fee Act (PDUFA) date set for August 21, 2025, and no advisory committee meeting expected. The analyst remains optimistic about the drug’s label, which will include data from Phase 3 and long-term extension studies.
The cardiovascular disease outcomes data for Pelacarsen, partnered with Novartis (SIX:NOVN) (NVS), has been delayed to the first half of 2026 due to slower event rate accumulation. InvestingPro analysis shows Ionis maintains a strong liquidity position with a current ratio of 8.51 and operates with moderate debt levels, providing financial flexibility for its development programs. However, if the target is achieved and the data is positive, a New Drug Application (NDA) filing is expected in 2026, with Ionis set to receive $50 million upon NDA acceptance.
Lastly, Ionis is preparing to initiate a Phase 3 trial for ION582 in Angelman syndrome in the first half of 2025, with strong support from patient advocacy groups and high anticipation for the trial. Ionis is approximately 6-12 months behind competitor Ultragenyx Pharmaceutical (NASDAQ:RARE)’s GTX-102 in this area. While the company faces near-term challenges with a revenue decline of 10.49% over the last twelve months, InvestingPro subscribers can access detailed financial health scores and 8 additional ProTips to better understand the company’s investment potential. For comprehensive analysis, check out the Pro Research Report, available exclusively to subscribers, which provides deep-dive insights into Ionis’s financial health and growth prospects.
In other recent news, Ionis Pharmaceuticals reported its fourth-quarter 2024 earnings, showcasing a stronger-than-expected financial performance. The company achieved an earnings per share (EPS) of -0.66, surpassing the forecasted -1.11, and revenue reached $227 million, exceeding the anticipated $135.58 million. This robust performance was driven by significant revenue growth from SPINRAZA royalties and WAYNEUWA product sales. Stifel analysts responded to these developments by adjusting their outlook on Ionis Pharmaceuticals, reducing the price target from $45.00 to $38.00 while maintaining a Hold rating. The adjustment was attributed to increased spending assumptions, despite the company meeting earnings expectations.
Ionis Pharmaceuticals is also advancing its pipeline, with the launch of TRINGOLZA for Familial Chylomicronemia Syndrome (FCS) underway, and preparations for the launch of donidalorsen pending FDA approval. The company projects revenue of over $600 million for 2025, with substantial growth anticipated through product launches and strategic partnerships. Ionis is preparing for multiple significant milestones, including the phase 3 study of a drug for Angelman Syndrome and the potential market introduction of treatments for severe hypertriglyceridemia (sHTG) in 2026. Analysts at Stifel highlighted the importance of these developments, particularly the ongoing phase 3 study of TTR-CM, with results expected in the second half of 2026.
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