Ionis Pharmaceuticals, Inc. - Common Stock (IONS)

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, February 19th at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2024 financial results.

Applications are based on data from the DEVOTE study, which demonstrate the potential for the investigational higher dose regimen of nusinersen to advance the treatment of SMA

CAMBRIDGE, Mass., Oct. 08, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced detailed results from Part B and Part C of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of an investigational higher dose regimen of nusinersen in spinal muscular atrophy (SMA), showing benefits in both individuals previously treated and treatment-naïve to nusinersen with infantile-onset or later-onset SMA. The investigational, higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months, compared to the approved nusinersen regimen (SPINRAZA®). Data to be presented during the World Muscle Society (WMS) 2024 Congress (Oct. 8-12, 2024 in Prague) highlight the potential of this investigational higher dose regimen to help address remaining unmet need in SMA.

BOSTON, Sept. 04, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQPRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that that it will present preclinical and clinical data from three of its epilepsy programs at the International League Against Epilepsy (ILAE) 15th European Epilepsy Congress (EEC), being held from September 7 to 11, 2024 in Rome, Italy.

CAMBRIDGE, Mass., Sept. 04, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced positive, topline data from the pivotal cohort (Part B) of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of a higher dose regimen of nusinersen in treatment-naïve, symptomatic infants with spinal muscular atrophy (SMA). The investigational higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and a higher maintenance regimen, 28 mg, every 4 months, compared to the approved nusinersen regimen (SPINRAZA). The study met its primary endpoint at six months, achieving a statistically significant improvement in motor function in infants who received the higher dose regimen as compared to a prespecified matched sham (untreated) control group from the ENDEAR study.

CAMBRIDGE, Mass. and CARLSBAD, Calif., May 16, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced the decision to terminate development of BIIB105 (ION541) an investigational antisense oligonucleotide (ASO) for amyotrophic lateral sclerosis (ALS) based on topline results from the Phase 1/2 ALSpire study. BIIB105 was designed to reduce expression of ataxin-2 (ATXN2) protein and demonstrated statistically significant cerebrospinal fluid (CSF) ATXN2 protein reductions in the study. However, over the 6-month placebo-controlled period, treatment with BIIB105 did not result in a reduction in levels of plasma neurofilament light chain (NfL), a marker of neurodegeneration and neuronal damage. Additionally, BIIB105 did not demonstrate an impact on clinical outcome measures of function, breathing, and strength.

Undervalued stocks Albemarle, Ionis, Insulet, Hess and Wynn beat earnings views, offering potential for investors to buy quality companies at discounted prices.

CAMBRIDGE, Mass., March 06, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced interim 6-month biomarker data from the initial 29 participants in the open-label RESPOND study.* The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec). The new data show that plasma neurofilament light chain (NfL) levels, an objective biomarker of axonal injury and neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA. These data will be presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 3-6, 2024).

Positive preliminary analysis of 15 mg cohort in the PRAX-628 study in epilepsy patients with Photo-Paroxysmal Response (PPR) exceeds expectations; topline results expected in 1Q 2024 after completion of 45 mg cohort

BOSTON, Nov. 16, 2023 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQPRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that the European Medical Agency (EMA) has awarded its Priority Medicines (PRIME) designation for elsunersen (PRAX-222) for the treatment of SCN2A Gain of Function (GoF) developmental and epileptic encephalopathy (DEE). The EMA’s PRIME designation provides enhanced development support for priority medicines that target an unmet need and was granted based on the Part 1 data from the EMBRAVE study that showed a reduction in seizures and improvement in seizure free days, as well as preclinical data.

Positive results from preliminary analysis of PRAX-222 Part 1 showed 44% median reduction in seizures after three doses for SCN2A-gain-of-function pediatric patients

Ionis Pharmaceuticals, Inc. (NASDAQIONS) has caught the attention of the investment community today with its bullish price action. The company’s shares are currently up 5.6% on the day to $45.05.

CAMBRIDGE, Mass., June 30, 2023 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community. The data were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA this week in Orlando, Fla.

NEW YORK and CARLSBAD, Calif., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Royalty Pharma has acquired an interest in Ionis’ royalty in Biogen’s SPINRAZA® (nusinersen) and Novartis’ pelacarsen for up to $1.125 billion, including an upfront payment of $500 million and up to $625 million in additional pelacarsen milestone payments.

CAMBRIDGE, Mass. and LOWELL, Mass., Jan. 04, 2023 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Alcyone Therapeutics (Alcyone) have entered into a license and collaboration agreement to develop Alcyone’s ThecaFlex DRx™ System, an implantable medical device intended for subcutaneous delivery of antisense oligonucleotide (ASO) therapies into the intrathecal space. Through this agreement, Biogen aims to leverage the ThecaFlex DRx™ System with a goal of improving the patient treatment experience and accessibility for a broader population of people suffering from neurological disorders, such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).

Palm Beach, FL – June 22, 2022 – FinancialNewsMedia.com News Commentary Neuroinflammatory disorder is the study of conditions where immune responses which damage components of the nervous system. It includes many rare neurological disease disorders like Alzheimer’s disease, Parkinson’s disease and Multiple Sclerosis. It is also associated with aging and Traumatic brain injury. According to […]