September 9, 2024 - 🧬 [nGram] Today’s Neurology Scoop: FDA Supports αSyn-SAA, Novocure at ESMO, MBX IPO

1. FDA issues letter of support encouraging use of synuclein-based biomarker, αSyn-SAA, in clinical trials in Parkinson's and related diseases

   • The FDA issued a 'Letter of Support' for the alpha-synuclein seed amplification assay (αSyn-SAA) biomarker for Parkinson's and related diseases.
   • The biomarker, discovered in 2023, can detect early biology related to Parkinson's and Dementia with Lewy Bodies, even before symptoms emerge.
   • The support follows collaboration among The Michael J. Fox Foundation for Parkinson's Research and the Critical Path Institute.
   • The FDA's decision is based on significant evidence from the Parkinson's Progression Markers Initiative (PPMI) study, which validated the biomarker.
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2. MBX Biosciences announces launch of initial public offering

   • MBX Biosciences has launched its initial public offering (IPO) of 8,500,000 shares of common stock.
   • The estimated IPO price is between $14.00 and $16.00 per share, with a 30-day option for underwriters to purchase an additional 1,275,000 shares.
   • Proceeds will fund the development of MBX 2109 and MBX 1416, preclinical program MBX 4291, discovery research, and general corporate purposes.
   • Shares will be listed on the Nasdaq Global Select Market under the symbol 'MBX'.
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3. Novocure to present real-world data at ESMO 2024 demonstrating improved survival outcomes for newly diagnosed glioblastoma patients with increased use of tumor treating fields therapy

   • Novocure will present a real-world analysis of Tumor Treating Fields (TTFields) therapy at ESMO 2024.
   • The analysis suggests higher TTFields device usage is linked to improved survival in newly diagnosed glioblastoma (ndGBM) patients.
   • Presentation details: Poster #459, September 16, ESMO Congress 2024, Barcelona, Spain.
   • Novocure is also sponsoring an ESMO Colloquium on the role of alternating electric fields in treating advanced non-small cell lung cancer.
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4. Cartesian Therapeutics receives FDA rare pediatric disease designation for Descartes-08 for the treatment of juvenile dermatomyositis

   • FDA grants Rare Pediatric Disease Designation to Descartes-08 for juvenile dermatomyositis (JDM).
   • Descartes-08 is an autologous mRNA-engineered CAR-T therapy targeting BCMA, designed without preconditioning chemotherapy.
   • IND filing for Phase 2 pediatric basket study on track for year-end, focusing on neurology and rheumatology autoimmune indications.
   • Rare Pediatric Disease Designation may qualify Cartesian for a priority review voucher if Descartes-08 is approved for JDM.
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5. PureTech founded entity Vor Bio announces new clinical data validating approach of using shielded transplants to deliver targeted therapies

   • Vor Bio announced new clinical data from its ongoing Phase 1/2 VBP101 study for relapsed/refractory AML patients.
   • The data showed reliable engraftment, shielding from Mylotarg on-target toxicity, and early evidence of patient benefit.
   • 18 patients were treated with trem-cel, with 10 receiving Mylotarg, demonstrating high CD33 editing efficiency and robust platelet recovery.
   • Vor Bio plans to discuss a pivotal trial design with the FDA by year-end and continues to explore other synergistic opportunities.
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6. Roivant provides update on Graves’ disease development program

   • High dose batoclimab achieved a 76% response rate in patients uncontrolled on antithyroid drugs (ATDs) at week 12.
   • 56% of patients achieved an ATD-free response rate at week 12 with high dose batoclimab.
   • Strong correlation observed between IgG lowering and clinical outcomes, indicating potential best-in-class opportunity for IMVT-1402.
   • IND cleared with initiation of IMVT-1402 pivotal trial in Graves’ Disease expected by the end of 2024.
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