Outcome Biotech Market Movers: Wide-Ranging Late-Stage Pipeline Propels Ionis Pharmaceutical, Inc.

By: Nick Frame, PhD, Vice President

Leveraging differentiated therapeutic platforms, biotech companies progress comprehensive product pipelines across various indications, attracting multiple strategic partnerships to enable rapid growth.

Ionis Pharmaceuticals, Inc (NASDAQ:IONS), developing antisense RNA-targeting medicines, held their Q2/22 earnings call Tuesday morning (8/9/2022). Leading up to the call, Ionis saw a >10% stock price appreciation, reaching highs of >$46/share, after a month of sitting under $40/share. The company’s strong performance supported the increase, closing today (8/10/2022) at $46.11. In Q2, Ionis reported revenues of $134M from the commercial products and partnered programs. With this quarter’s performance Ionis reported $276M in 1H revenues. Importantly, Ionis signified that best seller, Spinraza (in partnership with Biogen), continues to show encouraging signs for future growth. Of note, Ionis disclosed high-profile partnerships with Biogen, AstraZeneca and Roche accounted for $57M, $37M and $22M, respectively, in 1H revenue.

Despite management signaling that Ionis remain on target to meet stated FY22 goals, the focus of the call was predominantly on the company’s late-stage pipeline. As founder and CEO, Brett Monia shared, “[Ionis] made great progress in advancing our key priority to grow our commercial pipeline and deliver abundant new transformational medicines to the market. We’re looking forward to potentially adding eplontersen and tofersen to our commercial portfolio as early as next year. We continue to make excellent progress in advancing our other near-term opportunities, olezarsen and donidalorsen and with multiple positive mid-stage data readouts, we’re well positioned to expand our Phase III pipeline to these medicines across 10 indications.”

Ionis’s most advanced experimental medicine, tofersen, in partnership with Biogen (NASDAQ: BIIB), has been developed for the treatment of a form of ALS caused by genetic mutations in SOD-1, and is currently under priority review with the potential to be approved early in 2023. The team emphasized to investors that they have developed a comprehensive data package supporting a disease-modifying effect. Richard Geary, Chief Development Officer, communicated “The filing includes new integrated data from the Phase III VALOR study and ongoing OLE study, which shows tofersen significantly slow decline across multiple measures of ALS disease progression and importantly, led to robust and sustained reductions in neurofilament, a marker for neurodegeneration”.

Eplontersen, developed in collaboration with AstraZeneca (NASDAQ: AZN), represents a potential second near-term approval for Ionis. The Company communicated their intention to file a New Drug Application (NDA) with the FDA in Q3 of this year. While the initial discussion focused on the positive data readouts of Eplontersen in TTR polyneuropathy, which is the first indication Ionis will seek approval for, the conversation shifted to the much larger market opportunity of ATTR cardiomyopathy.  Ionis and AstraZeneca are currently undergoing a Phase III trial (CARDIO-T TRANSFORM) to support approval in this indication, which was indicated to be progressing well.

Finally, the late-stage pipeline of Phase IIb and Phase III assets, positions Ionis to have additional high-profile clinical readouts in the near-term. In fact, the company stated that just this year three positive read-outs in Phase IIb trials have led Ionis and their partners (GSK and Roche) to continue the development of additional programs in Phase III.  Taken together, Elizabeth Hougen, CFO, communicated; “By investing in our strategic priorities, including advancing our goal to deliver a steady stream of new medicines to the market, [Ionis is] poised to deliver significant future growth.”

Key Takeaways

  • Strategic partnerships, underpinned by differentiated platforms, drive growth and reduce investor risks
  • Indication/disease expertise required for effective clinical development and commercialization
  • R&D collaborations offer substantial risk mitigation and revenue opportunities for pre-commercial programs

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