Latest news with #HollyKordasiewicz
Yahoo
8 hours ago
- Business
- Yahoo
Ionis (IONS) Announces Leadership Transition as R&D Veteran Richard Geary Prepares to Retire
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) is one of the 10 biotech stocks screaming a buy. On June 12, the company announced the upcoming retirement of Richard Geary, Ph.D., its executive vice president and chief development officer, effective January 2026. Holly Kordasiewicz, Ph.D., currently senior vice president of neurology, will take over the role. Geary has been with Ionis since 1995 and played a pivotal role in bringing six medicines to regulatory approval, including the company's first independently commercialized drug, TRYNGOLZA. A biotechnologist pouring liquid into a test tube and analyzing its components in a lab. Dr. Kordasiewicz, who joined Ionis in 2011, brings over 20 years of experience in R&D, particularly in neurology. She leads the company's neurology program, covering treatments for conditions such as Alzheimer's disease, Angelman syndrome, and Alexander disease. Her work has also contributed to key partnered programs with Biogen, including the development of QALSODY® and IONIS-MAPTRx. As Dr. Geary transitions out of his role, he will continue as a strategic consultant through 2026 to ensure continuity. Ionis leadership praised both Geary's legacy and Kordasiewicz's appointment as a pivotal step in driving the company's commitment to developing transformational therapies for patients with serious diseases. Ionis Pharmaceuticals, Inc. is a U.S.-based commercial-stage biotech company specializing in RNA-targeted therapies. Its approved products include TRYNGOLZA for FCS, WAINUA, and TEGSEDI for ATTRv-PN, SPINRAZA for spinal muscular atrophy, QALSODY for ALS, and WAYLIVRA for rare lipid disorders. The company has a robust pipeline, including late-stage programs like Olezarsen (for hypertriglyceridemia), Donidalorsen (for hereditary angioedema), and Zilganerse (for Alexander disease), along with several mid-stage treatments for neurological and metabolic conditions. Ionis also collaborates with leading pharma firms, including Biogen, GSK, AstraZeneca, Novartis, Roche, and Metagenomi, expanding its reach in developing transformative RNA therapies. While we acknowledge the potential of IONS as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 13 Best Software Stocks to Buy Now and 11 Must-Buy AI Stocks Analysts Are Betting On. Disclosure: None.
Yahoo
12-06-2025
- Business
- Yahoo
Ionis doses first subject in Phase III trial of Angelman syndrome therapy
Ionis Pharmaceuticals has dosed the first subject in the global, randomised Phase III REVEAL trial of investigational ION582, aimed at treating individuals with Angelman syndrome (AS), a rare neurodevelopmental disorder. The placebo-controlled, double-blind trial will recruit nearly 200 AS adults and children with a maternal UBE3A gene deletion or mutation. Over a 52-week treatment period, subjects will be randomised in a 2:1 ratio and given either the therapy or a placebo. Quarterly doses of either 40mg or 80mg of ION582 will be administered to those in the active treatment groups. After the initial treatment phase, eligible subjects will have the opportunity to enter a long-term extension segment of the trial, where the therapy will be administered for up to two years. The primary goal of the trial is to assess improvements in expressive communication using the Bayley Scales for Infant and Toddler Development-4 (Bayley-4), a direct clinician-administered assessment tool. Expressive communication deficits are notably the most burdensome symptoms for caregivers of individuals with AS. The trial will also evaluate secondary endpoints, including overall disease severity, communication, cognition, motor functioning, sleep, and daily living skills, as well as other exploratory endpoints. ION582 has previously shown promise in the multiple ascending dose (MAD) portion of the Phase I/II open-label HALOS trial, demonstrating clinical improvement across various functional domains such as cognition, motor function, and communication. The RNA-targeted antisense medicine, ION582, works by inhibiting the expression of the UBE3A antisense transcript (UBE3A-ATS) to potentially increase the UBE3A protein production, to treat AS. Ionis Pharmaceuticals neurology senior vice-president Holly Kordasiewicz: 'This placebo-controlled study will evaluate ION582 in both children and adults with either UBE3A deletion or mutation and builds on earlier encouraging findings from our Phase I/II HALOS study. 'With ION582, we continue to advance our leading wholly owned neurology pipeline, which includes eight medicines in clinical development across a range of rare and more prevalent diseases.' Last year, Ionis announced the design for the Phase III trial of ION582 for the treatment of AS, following discussions with the US Food and Drug Administration. "Ionis doses first subject in Phase III trial of Angelman syndrome therapy" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
11-06-2025
- Business
- Business Wire
First patient dosed in pivotal Phase 3 REVEAL clinical study of ION582 in Angelman syndrome
CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the first participant has been dosed in the global Phase 3 REVEAL study, which is designed to evaluate the efficacy and safety of ION582, an investigational medicine for the treatment of people living with Angelman syndrome (AS), a serious and rare neurodevelopmental disorder that leads to significant physical and cognitive impairments. 'Dosing the first person with Angelman syndrome in our pivotal REVEAL study marks an important milestone for this underserved community, who currently have no approved disease modifying treatments,' said Holly Kordasiewicz, Ph.D., senior vice president of neurology, Ionis. 'This placebo-controlled study will evaluate ION582 in both children and adults with either UBE3A deletion or mutation and builds on earlier encouraging findings from our Phase 1/2 HALOS study. This milestone reflects our commitment to those impacted by Angelman syndrome and builds on our legacy of innovation in neurology including the development of SPINRAZA and QALSODY. With ION582, we continue to advance our leading wholly owned neurology pipeline, which includes eight medicines in clinical development across a range of rare and more prevalent diseases.' REVEAL (NCT06914609) is a global, randomized, double-blind, placebo-controlled Phase 3 study that will enroll approximately 200 children and adults with AS that have a maternal UBE3A gene deletion or mutation. During the 52-week treatment period, participants will be randomized 2:1 to receive either ION582 or placebo. Participants in the active treatment groups will receive quarterly 40 mg or 80 mg doses of ION582. Following the treatment period, eligible participants will transition into the long-term extension portion of the study where all participants will receive ION582 for up to 2 years. The primary endpoint is improvement in expressive communication as assessed by the Bayley Scales for Infant and Toddler Development-4 (Bayley-4), an objective and direct clinician-administered assessment of clinical functioning. Deficits in expressive communication are reported to be the symptoms most challenging to caregivers of people with AS. Secondary endpoints include overall disease severity, cognition, communication, sleep, motor functioning and daily living skills, in addition to other exploratory endpoints. In the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label HALOS study, ION582 provided consistent and encouraging clinical improvement on all functional domains including communication, cognition and motor function and showed favorable safety and tolerability at all dose levels in the study. For more information on the REVEAL clinical study, please visit and About ION582 ION582 is an investigational RNA-targeted antisense medicine designed to inhibit the expression of the UBE3A antisense transcript (UBE3A-ATS) and increase production of UBE3A protein, for the potential treatment of Angelman syndrome (AS). The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) granted Orphan Drug designation to ION582. Additionally, the FDA granted Fast Track and Rare Pediatric designations to ION582. About Angelman Syndrome (AS) AS is a rare, genetic neurological disease that affects an estimated 1 in 21,000 people worldwide and is caused by the loss of function of the maternally inherited UBE3A gene. AS typically presents in infancy and is characterized by profound intellectual disability, balance issues, motor impairment and debilitating seizures. Most people with AS are unable to speak. Individuals with AS have a normal lifespan but require complete care from a caregiver. Some symptoms can be managed with existing medicines; however, there are no approved disease modifying therapies. About Ionis Neurology Ionis has been at the forefront of discovering and developing leading neurological disease medicines, including SPINRAZA ® (nusinersen), the first approved treatment for spinal muscular atrophy, WAINUA™ (eplontersen), a medicine to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), and QALSODY ® (tofersen) for SOD1-ALS. The clinical-stage portfolio includes 13 therapies, of which eight are wholly owned by Ionis. Ionis' investigational portfolio includes medicines for which there are few or no disease modifying treatments, such as rare diseases including Prion disease and Alexander disease and more common conditions such as Alzheimer's and Parkinson's disease. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology, and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit and follow us on X (Twitter), LinkedIn and Instagram. Ionis Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of ION582, our commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, 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, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals ® is a trademark of Ionis Pharmaceuticals, Inc. SPINRAZA ® and QALSODY ® are registered trademarks of Biogen. WAINUA™ is a registered trademark of the AstraZeneca group of companies.
