Ionis partner to evaluate tominersen for Huntington’s disease in new phase 2 trial

  • Post-hoc analysis of data from the GENERATION HD1 study showed that tominersen may have potential benefits in a subset of patients
  • Ionis partner Roche is in the early stages of designing a trial for a younger adult patient population with less disease burden
  • Partner to participate in a series of webinars for the scientific and patient communities starting January 20, 2022

CARLSBAD, Calif., January 18, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced that its partner, rock, is designing a new phase 2 trial to evaluate tominersen in Huntington’s disease (HD). After discontinuation of dosing in the Phase 3 GENERATION HD1 study, exploratory post-hoc analyzes suggest that tominersen may benefit younger adult patients with lower disease burden. These results need to be confirmed by a randomized, placebo-controlled study.

“These findings are promising and warrant a new study designed to test tominersen in this specific patient group. We are pleased that Roche has determined that there is a pathway to move the tominersen development program forward,” said C. Frank Bennett, Ph.D., Ionis Executive Vice President, Chief Scientific Officer and Franchise Leader for Neurological Programs. “This is an encouraging development for the HD community. We and Roche are grateful for the HD community’s continued partnership, which has led to this important information and a new scientific hypothesis.”

Roche is in the early stages of designing the Phase 2 clinical trial to explore different doses of tominersen in a younger adult patient population with less disease burden. Roche will share more details on the design of the new phase 2 trial at future scientific meetings. Starting with the scientific community on January 20, 2022, Roche will participate in a series of webinars to discuss the results of their post-hoc analysis of data from the GENERATION HD1 study and next steps for the tominersen program.

About tominersen and clinical trials

Tominersen, formerly IONIS-HTTRx or RG6042, is an investigational antisense drug designed to reduce the production of all forms of the protein huntingtin (HTT), including its mutated variant, mHTT. In December 2017, Roche has licensed Ionis’ investigational drug. The tominersen clinical development program included the following studies:

  • GENERATION HD1: a randomized, multicenter, double-blind, placebo-controlled phase 3 clinical study evaluating the efficacy and safety of tominersen treatment in people with overt HD for 25 months. Study participants were randomized to receive either 120 mg every two months, 120 mg every four months with intrathecal injections of tominersen, or a placebo. The study recruited 791 participants from 18 countries around the world. In March 2021, Roche announced that the assay will be discontinued in tominersen’s Phase 3 GENERATION HD1 study following a recommendation from the Independent Data Monitoring Committee (iDMC) based on an overall benefit/risk assessment. The study is ongoing without dosing to allow participants to be monitored for safety and clinical outcomes. The study will end at the last visit of the last patient, as planned in March/April 2022.
  • GEN-EXTEND: an open-label extension study for participants from any Roche HD study. Participants received 120 mg of tominersen every two months or every four months in the study. The study is ongoing without dosing to allow participants to be monitored for safety and clinical outcomes. The study will end in March/April 2022.
  • GEN-PEAK: a phase I study to better understand the pharmacokinetics of tominersen and how tominersen affects levels of mHTT and other markers in cerebrospinal fluid and blood, investigating different doses of tominersen over two administrations . The study will now be terminated as all patients have completed Part 1 of the study and Part 2 will not be conducted.

Study participants should contact their local clinical trial site for any questions or assistance regarding their situation.

About Huntington’s disease

Huntington’s disease is a rare, progressive genetic disease that causes nerve cells in the brain to break down, causing problems with the ability to think, move and function, leading to increased disability and loss of independence. It has a devastating impact on people living with the disease, and the hereditary nature of HD means it deeply affects entire families for generations. Survival varies from approximately 10 to 20 years after the motor onset of the disease. There is no known cure for HD and no approved treatment that treats the underlying cause.

About the Ionis Neurology Franchise

The Ionis neurology franchise covers all major brain regions and types of central nervous system, currently has three drugs in phase 3 studies and 12 drugs in clinical development, three of which are 100% owned. Ionis is leading the way in treating the root causes of many neurological diseases and in developing antisense drugs for common diseases, such as Alzheimer’s disease and Parkinson’s disease, rare diseases such as ALS, Huntington’s disease and Alexander’s disease. Medicines marketed by Ionis for neurological diseases include SPINRAZA®, the global care base for spinal muscular atrophy (SMA) and TEGSEDI®, the first and only self-administered subcutaneous treatment for hereditary ATTR amyloidosis polyneuropathy in adults.

About Ionis Pharmaceuticals

For more than 30 years, Ionis has been the leader in RNA-targeted therapy, opening new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed drugs and a leading late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become one of the most successful biotechnology companies.

To learn more about Ionis, visit and follow us on Twitter @ionispharma.

Ionis forward-looking statement
This press release contains forward-looking statements regarding Ionis’ business, Ionis technologies, tominersen and other products in development. Any statement describing Ionis’ objectives, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. These statements are subject to certain risks and uncertainties, including those relating to the impact that COVID-19 may have on our business, and including, but not limited to, those relating to our commercial products and pharmaceuticals. our pipeline, and in particular those inherent in the process of discovering, developing and commercializing safe and effective drugs for human therapeutic use, and in building a business around such drugs. Ionis’ forward-looking statements also involve assumptions which, if they never materialize or prove to be incorrect, 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, such statements are based solely on facts and factors currently known to Ionis. Accordingly, you are cautioned not to rely on these forward-looking statements. These and other risks relating to Ionis’ programs are further described in Ionis’ Annual Report on Form 10-K for the fiscal year ended. December 31, 2020, and the most recent quarterly filing of Form 10-Q, which are filed with the SEC. Copies of these and other documents are available from the Company

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

Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. Spinraza is marketed by Biogen.

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SOURCEIonis Pharmaceuticals, Inc.

Company Codes: NASDAQ-NMS:IONS

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