NOVARTIS

The OCT DACH 2024 conference in Zurich discussed EU regulations, digitalisation, AI, and ethics in clinical trials, emphasizing the DACH region's challenges and opportunities.

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.

Insilico Medicine announced results from a clinical trial of ISM001-055, designed using generative AI to treat idiopathic pulmonary fibrosis, showing improvements in lung function and quality of life. Enveda began clinical trials of ENV-294, an oral inflammatory agent for atopic dermatitis, derived from natural compounds identified using AI. Various healthcare updates include FDA approvals, licensing agreements, and collaborations in drug discovery and treatment development.

Study at MD Anderson Cancer Center (IRB-approved, ClinicalTrials.gov NCT04239157) enrolled patients with relapsed/refractory lower-risk MDS (IPSS/IPSS-R ≤3.5, hemoglobin <10 g/dL, symptomatic anemia/transfusion dependency). Open-label, single-arm, phase 2 trial used canakinumab (150 mg, then 300 mg subcutaneously) with primary endpoint of hematologic improvement (HI). Secondary objectives included safety, transfusion independence, duration of response, progression-free survival, leukemia-free survival, and overall survival. Bone marrow assessments, single-cell RNA sequencing, and cytokine/chemokine quantification were performed. Statistical analyses used modified IWG 2006 criteria, Kaplan–Meier method, Cox proportional hazards model, and R platform.

DelveInsight's 'Multiple Sclerosis Pipeline Insight 2024' report highlights 75+ companies developing 80+ therapies, including promising candidates like GA Depot, Remibrutinib, and IMU 838. Key events include Immunic's positive Phase 3 ENSURE trial interim analysis, Roche's FDA approval for Ocrevus injectable, and Sanofi's mixed results for tolebrutinib in Phase III trials. The report covers global pipeline stages, product types, molecule types, mechanisms of action, and routes of administration.

Asia's complex market for innovative therapies includes varied regulatory, market access, and healthcare infrastructures. Early access programs (EAPs) like Hainan's have facilitated rapid entry for over 400 drugs and devices, with examples like Yutiq and Lorbrena gaining formal approvals and reimbursement. China's EAPs have expanded to the Greater Bay Area and beyond, while Japan, Korea, Southeast Asia, and the Middle East also use EAPs to provide early access to innovative treatments.

Non-cystic fibrosis bronchiectasis market driven by rising prevalence, early diagnosis, and drug development. Over 15 companies, including Zambon SpA, Insmed, and AstraZeneca, are developing 15+ pipeline therapies. Key drugs in trials include Colistimethate sodium, Benralizumab, and Brensocatib.

The Nasal Drug Delivery Technology Market is projected to grow from USD 4.4 billion in 2024 to USD 7.8 billion by 2031, driven by non-invasive drug administration, technological advancements, and rising respiratory disease prevalence.

Goodwin's two-part series on Greater China's life sciences sector highlights stable M&A activity in pharmaceuticals/biotech despite market fluctuations. Notable 2024 deals include Fosun's $1.71B acquisition of Shanghai Henlius Biotech, Johnson & Johnson's $850M purchase of Proteologix, and Novartis' acquisition of SanReno Therapeutics. Trends indicate a shift towards CAR-T cell therapies and large molecule/biologics, with medical devices also gaining significance. Factors driving recovery include regulatory changes, market readjustments, and the maturing of early-stage investments.

Curocell appoints Lee Seung-won as managing director to lead domestic commercialization of next-gen CAR-T treatment CRC01 (Anbal-cel). Lee, with 20+ years in hematologic cancers and CAR-T therapeutics, aims to expedite Anbal-cel's approval and global expansion.