IONIS PHARMACEUTICALS, INC.

BridgeBio's acoramidis, a transthyretin stabilizer, may soon compete with Pfizer's tafamidis for treating transthyretin amyloid cardiomyopathy (ATTR-CM). Acoramidis, with an FDA action date of Nov. 29, could see approval, potentially challenging Pfizer's established position in the $5.2 billion ATTR-CM market. BridgeBio's Phase III trial showed acoramidis improved survival rates and reduced hospitalizations, though it faces challenges like twice-daily dosing and lack of mortality benefit data compared to tafamidis. Emerging RNA therapies, like Alnylam's Amvuttra and Intellia's nex-z, also show promise in treating ATTR-CM.

Two new drugs, an siRNA and an oral agent, reduced Lp(a) by 80% in phase II studies. Zerlasiran, the siRNA, lowered Lp(a) by 85% over 36 weeks, while muvalaplin, the oral agent, reduced Lp(a) by 70% with a traditional assay and over 85% with an intact assay. Both therapies offer potential for treating cardiovascular disease, with muvalaplin providing an oral alternative to injectables.

Muvalaplin, an oral drug, significantly reduced lipoprotein(a) (Lp[a]) by 70% in a traditional blood test and 85.5% in a new test, according to a phase 2 study presented at the 2024 AHA Scientific Sessions. The drug, developed by Eli Lilly, targets Lp(a), a genetically driven variant of LDL cholesterol linked to cardiovascular disease, with no current treatments. Muvalaplin disrupts the bonding of apo(a) to apoB, preventing Lp(a) formation, and also reduced oxidized phospholipids. The study involved doses of 10 mg, 60 mg, and 240 mg over 12 weeks, with 97% of participants achieving Lp(a) levels below 125 nmol/L. Phase 3 trials and cardiovascular outcomes trials are next steps before market potential.

The North America RNA Therapeutics Market is projected to grow from USD 12.95 billion in 2023 to USD 17.79 billion by 2029, driven by advancements in RNA technology and personalized medicine. Key players include Moderna, Alnylam Pharmaceuticals, and Pfizer Inc.

FDA accepts new drug application for donidalorsen, a prophylactic RNA-targeting drug to prevent hereditary angioedema attacks, with an action date set for Aug. 21, 2025.

New Huntington's disease therapies are nearing R&D finish lines, aiming to be the first disease-modifying treatments. Despite high-profile failures, companies like Wave, Prilenia, and uniQure are progressing towards regulatory approval. The field has gained momentum after decades of trial and error, with several mid-stage clinical trials underway. The ideal therapy would selectively lower mutant huntingtin and be widely distributed in the brain, though none currently exist.

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Ionis Pharmaceuticals announces Phase 3 study design for ION582, following FDA alignment, with Bayley-4 expressive communication as the primary endpoint. The study is planned to begin in H1 2025 and will enroll 200 AS patients with maternal UBE3A gene deletion or mutation. Ionis will provide an update on the Phase 3 program at the FAST Global Science Summit in November.

Praxis Precision Medicines reports significant progress in pipeline, including advancing relutrigine to late-stage development and finalizing plans for ulixacaltamide's interim analysis. Positive results from EMBOLD cohort 1 highlight relutrigine's potential as a first- and best-in-class therapy for DEEs. Financials show $411.2 million in cash, cash equivalents, and marketable securities as of September 30, 2024, with a net loss of $51.9 million for Q3 2024.