Ionis Pharmaceuticals

Six companies—Ionis Pharmaceuticals, Isarna Therapeutics, Regulus Therapeutics, Sarepta Therapeutics, Secarna Pharmaceuticals, and Wave Life Sciences—are leading in the development of antisense oligonucleotides, a targeted treatment for various diseases. Ionis, a pioneer with over 40 drugs in its pipeline, recently priced a $500 million IPO. Isarna's ISTH0036 targets TGF-β for ophthalmic conditions. Regulus focuses on microRNA targeting with its lead candidate RGLS8429 for ADPKD. Sarepta has three approved PPMO therapies for DMD and a recent $1 billion deal with Arrowhead Pharmaceuticals. Secarna's SECN-15 targets NRP1 for oncology. Wave Life Sciences' WVE-003, for Huntington's disease, recently reported positive phase 1b/2a trial results. The antisense oligonucleotide market is projected to grow to $5,519 million by 2033.

BridgeBio Pharma's acoramidis (Attruby) wins FDA approval for treating ATTR cardiomyopathy, competing with Pfizer's tafamidis. Attruby, a near-complete TTR stabilizer, aims to reduce cardiovascular death and hospitalization. Despite initial trial setbacks, 30-month data supported approval, positioning Attruby as the first oral stabilizer with near-complete stabilization claims. BridgeBio estimates a $15-20 billion market, dominated by Pfizer, with Alnylam and AstraZeneca also entering the space. Attruby's pricing is competitive but above cost-effectiveness benchmarks, with ongoing European review and a Bayer licensing deal.

FDA approves BridgeBio Pharma's Attruby (acoramidis) for transthyretin amyloidosis cardiomyopathy, priced at $244,000 annually. The drug stabilizes misfolded TTR protein, showing survival and hospitalization benefits. Market competition heats up with Pfizer's tafamidis and potential rivals. BridgeBio aims for 30% market share, planning head-to-head trials.

DelveInsight's report on Nonalcoholic Steatohepatitis (NASH) provides insights into historical and forecasted epidemiology, market trends, current treatment practices, emerging drugs, and market size from 2019 to 2032 in the United States, EU5, and Japan. Key companies and therapies in the NASH market are highlighted, including recent developments like Aligos Therapeutics' Phase 1 data presentation.

Huntington's disease treatments face challenges due to rarity and trial failures, but recent milestones like Wave's WVE-003, Roche-Ionis' tominersen, uniQure's AMT-130, and PTC Therapeutics' PTC518 offer hope. Prilenia's pridopidine is under EMA review, while Annexon's ANX005 and Sage's dalzanemdor faced setbacks. Despite these, cautious optimism remains for effective treatments.

Eli Lilly's muvalaplin, an oral cholesterol-lowering drug, significantly reduced Lp(a) levels in a 12-week phase II study, meeting primary and secondary endpoints. Muvalaplin disrupts apolipoprotein(a) and apolipoprotein(b) interaction, potentially reducing cardiovascular risk. Lilly's stock has risen 24.7% this year.

IgA nephropathy affects 25-50 per million annually, with 20–40% progressing to end-stage kidney disease. Diagnosed cases in the US are expected to reach 135,000 by 2030. Treatments include ACE inhibitors, ARBs, corticosteroids, and immunosuppressants. FDA-approved treatments are TARPEYO, FILSPARI, and FABHALTA. The IgA nephropathy market is projected to reach ~USD 4.1 billion by 2034, driven by novel therapies like Novartis’ Atrasentan, Otsuka/Visterra’s Sibeprenlimab, Vertex/Alpine’s Povetacicept, and others.

Despite decades of research, Huntington’s disease lacks a disease-modifying treatment. Clinical trials face challenges due to limited patient numbers and geographic barriers. Prilenia Therapeutics, Sage Therapeutics, uniQure, Wave Life Sciences, and Roche-Ionis are developing potential treatments, though some have faced setbacks. Prilenia’s pridopidine and Sage’s dalzanemdor showed mixed results, while uniQure’s AMT-130 and Wave’s WVE-003 demonstrated promising reductions in mutant huntingtin protein. Roche-Ionis’ tominersen faces ongoing development challenges.

Ionis Pharmaceuticals plans a phase 3 trial for ION582, targeting Angelman syndrome (AS), with enrollment starting in H1 2025. The placebo-controlled study will assess ION582's efficacy and safety in 200 AS patients with maternal UBE3A gene issues over 12 months, using Bayley-4 for expressive communication as the primary endpoint. ION582, an antisense oligonucleotide, aims to increase UBE3A protein production, addressing AS's underlying pathology. Positive phase 2 results from HALOS support ION582's potential.