Ionis Pharmaceuticals

FDA accepts Ionis Pharmaceuticals' donidalorsen NDA for hereditary angioedema, sets PDUFA date for August 21, 2025. Clonidine hydrochloride extended-release liquid approved for ADHD in pediatric patients. PDUFA date for Dermavant's tapinarof cream extended to March 12, 2025. MenACWY vaccination program reduced meningococcal disease incidence by 59% among 11-23 year-olds.

The global spinal muscular atrophy market is projected to grow from USD 2,013.5 million in 2024 to USD 4,823.9 million by 2031, at a CAGR of 13.3%. Key drivers include FDA approvals for drugs like Zolgensma and Evrysdi, rising awareness, and increasing gene therapies. North America is expected to dominate due to its established healthcare infrastructure.

FDA approved Attruby (acoramidis) for transthyretin amyloidosis, a deadly heart condition, marking a significant step in treatment. Priced at $244,000 annually, it competes in a lucrative market against drugs like Pfizer's tafamidis. Despite investor skepticism, BridgeBio Pharma aims for a 30% market share, highlighting Attruby's potential advantages in survival and hospitalization rates.

Ionis Pharmaceuticals (IONS) reported a narrower-than-expected Q3 2024 loss of 95 cents per share, with total revenues of $134 million, beating estimates. The company's shares have declined 22.9% YTD. Ionis receives royalties from Biogen for Spinraza and co-markets Qalsody with Biogen. Wainua, co-marketed with AstraZeneca, was approved in the U.S. and the UK, generating $30 million in milestone payments. Commercial revenues were $76 million, missing estimates. Ionis maintained its 2024 financial guidance, expecting total revenues over $575 million and an adjusted operating loss of less than $475 million.

Ionis Pharmaceuticals plans Phase III REVEAL trial of ION582 for Angelman syndrome, enrolling 200 with UBE3A mutation/deletion. Primary endpoint is expressive communication improvement via Bayley-4. Trial includes long-term extension phase and assesses multiple secondary endpoints. CEO Brett Monia anticipates trial start in H1 2025.

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting progress with WAINUA U.S. launch, olezarsen FDA action date in December 2024, and donidalorsen HAE PDUFA date in August 2025. The company is on track to achieve 2024 financial guidance and increased cash guidance to $2.2 billion. Key pipeline milestones include positive CHMP opinion for WAINUA in the UK, QALSODY approval in China, and ongoing pivotal development programs for olezarsen and donidalorsen.

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting successful U.S. launch of WAINUA, upcoming FDA action dates for olezarsen and donidalorsen, and reaffirmed 2024 P&L financial guidance with increased cash guidance to $2.2 billion.

Praxis Precision Medicines reports progress in essential tremor treatment with ulixacaltamide, seizure-free outcomes in DEEs with relutrigine, and advances in focal epilepsy and generalized epilepsy treatments with vormatrigine. Financial results show increased cash reserves and operational expenses, with a net loss for Q3 2024.