Praxis Precision Medicines announced positive Phase 1/2 trial results for elsunersen (PRAX-222), an antisense oligonucleotide treatment for SCN2A developmental and epileptic encephalopathy. The drug demonstrated a 77% placebo-adjusted seizure reduction, with 71% of treated patients achieving >50% seizure reduction. All elsunersen-treated patients showed improvements in sleep, motor function, and attention, with no serious adverse events reported.

Ionis Pharmaceuticals announced a significant price reduction for its triglyceride-lowering drug Tryngolza (olezarsen) from $595,000 to $40,000 annually, effective April 1. The repricing positions the therapy competitively ahead of an expected FDA decision on label expansion into severe hypertriglyceridemia (sHTG) by June 30. Analysts view the move positively as it aligns with payer contracting cycles and could drive substantial revenue upside, with peak sales projections potentially exceeding $2 billion.

The article examines two stocks experiencing insider selling but maintaining bullish long-term outlooks. Waste Management insiders sold ~$25M after hitting all-time highs, but institutional accumulation and growing dividends support the outlook. Ionis Pharmaceuticals faces heavier insider and institutional selling, though analysts see 25% upside driven by Olezarsen's commercial ramp. Both stocks have pulled back from early 2026 highs, creating potential entry points.

Boone Capital Management liquidated its entire $13.57 million stake in Cogent Biosciences (945,042 shares) in Q4 2026, despite the stock surging 372% over the past year. The fund reallocated capital to earlier-stage biotech opportunities like TYRA, which has gained 40% in 2026, suggesting a strategic shift toward higher-risk, higher-upside clinical-stage investments.

The global ALS therapeutics market is valued at USD 812 million in 2024 and is projected to reach USD 1,964 million by 2035, growing at an 11.5% CAGR. Growth is driven by innovations in genetic therapies, gene-silencing technologies, improved diagnostics, and increased disease awareness. Key players include Biogen, Ionis Pharmaceuticals, Amylyx Pharmaceuticals, and others developing disease-modifying treatments.

The global amyloidosis therapeutics market is experiencing strong growth, valued at $2.95 billion in 2024 and expected to reach $6.37 billion by 2033 with a 9.20% CAGR. Growth is driven by accelerating ATTR cardiomyopathy diagnosis through imaging technologies, rapid uptake of disease-modifying therapies in North America, and expanded reimbursement in emerging markets like China and Brazil. Five core compounds dominate the market, with chemotherapy remaining the cost-effective backbone for AL amyloidosis treatment.

GSK and Ionis Pharmaceuticals announced positive Phase 3 trial results for bepirovirsen, an investigational antisense oligonucleotide for chronic hepatitis B. The B-Well 1 and B-Well 2 studies, involving over 1,800 patients, met their primary endpoint with statistically significant functional cure rates. If approved, bepirovirsen could become the first finite, six-month therapeutic option for CHB. Regulatory submissions are planned for Q1 2026.

Biogen has acquired Alcyone Therapeutics, a clinical-stage biotechnology company, to enhance its CNS therapy delivery solutions through the ThecaFlex DRx™ drug delivery system, which aims to provide an alternative to repeat lumbar punctures for neurologic disorder treatments.

Ionis Pharmaceuticals reported positive results from a clinical trial of zilganersen for Alexander disease, showing statistically significant stabilization of gait speed and the first evidence of disease modification in this rare neurological condition.

Antisense oligonucleotide (ASO) therapeutics are emerging as a promising treatment approach for rare genetic and neuromuscular disorders, with over 70 pharmaceutical companies actively developing 90+ pipeline therapeutics targeting specific genetic mechanisms.

The FDA approved Ionis Pharmaceuticals' Dawnzera, the first RNA-targeted medicine for preventing hereditary angioedema (HAE) attacks in patients 12 and older, demonstrating significant attack rate reduction in clinical trials.

Biogen will present scientific data on lecanemab and tau-targeted therapies at the 2025 Alzheimer's Association International Conference, showcasing long-term clinical trial results and potential new treatment approaches for Alzheimer's disease.