Cabaletta (trehalose) / Seelos Therap - LARVOL DELTA

HEALEY ALS Platform Trial - Master Protocol (clinicaltrials.gov) - P2/3 | N=1500 | Recruiting | Sponsor: Merit E. Cudkowicz, MD | Active, not recruiting ➔ Recruiting | Trial primary completion date: Nov 2027 ➔ Jul 2027

Enrollment open • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

HEALEY ALS Platform Trial - Master Protocol (clinicaltrials.gov) - P2/3 | N=1500 | Active, not recruiting | Sponsor: Merit E. Cudkowicz, MD | Trial completion date: Apr 2026 ➔ Aug 2028 | Trial primary completion date: Jul 2025 ➔ Nov 2027

Trial completion date • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

ISS1: CAR T Cell Therapy in Autoimmune Disease: A Potential Emerging Treatment Option (ACR Convergence 2025) - "FUNDING Developed and offered by Cabaletta Bio...Other investigational CAR T cell therapies in autoimmune diseases will also be summarized. Not an official program of the ACR."

CAR T-Cell Therapy • Immunology • Inflammatory Arthritis • Lupus • Myositis • Scleroderma • Systemic Sclerosis

Immunoglobulin G Levels in Ofatumumab-Treated Participants With Episodes of Low Immunoglobulin M: An Analysis of 7-Year Data From the ALITHIOS Extension Study (ECTRIMS 2025) - "IgG levels remained stable for up to 7 years of OMB treatment in plwRMS whose IgM level dropped 10% below LLN and was similar irrespective of whether treatment was interrupted/discontinued. Disclousure of interest: Heinz Wiendl receives research support from Deutsche Forschungsgemeinschaft (DFG), Deutsche Myasthenie Gesellschaft e.V., Europeenzyme, Idorsia, Immunic, Immunovant, INmune Bio_Syneos Health, Kohlhammer, LTS, Lundbean Union, Alexion, Amicus Therapeutics Inc., Argenx, Biogen, CSL Behring, F. Hoffmann-La Roche, Genzyme, Merck, Novartis, Roche, UCB Biopharma as well as speaker and/or consultancy fee from Alexion, Argenx, Argobio, AOCN, AstraZeneca, BGP Products Operations GmbH, Biogen, Bristol Myers Squibb, CEMCAT, Dianthus, EMD Serono Research &Development Institute, Inc., Billerica, MA, USA, an affiliate of Merck KGaA, EPG Health/Medthority, Fondazione Cariplo, Gck, Merck, MS at the Limits, Muna Therapeutics, Myrobalan Therapeutics, Neurodiem, NMSS,..."

Late-breaking abstract • CNS Disorders • Multiple Sclerosis

Unpacking FDA's Guidance on Manufacturing Changes and Comparability – Case Study on Cabaletta's Approach (ASGCT 2025) - No abstract available

Case study • Clinical

Subcutaneous Immunoglobulin (IgPro20) Dose Adjustments for Chronic Inflammatory Demyelinating Polyneuropathy Maintenance Therapy in Clinical Practice (P10-11.024). (PubMed, Neurology) - "To assess dosing of IgPro20 (immune globulin subcutaneous [human], 20% liquid, Hizentra®) in clinical practice in patients with chronic inflammatory demyelinating polyneuropathy (CIDP)...Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta...Dr. Jiang has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen ."

Journal • Retrospective data • Pain

Serostatus Testing Patterns Among Individuals with Myasthenia Gravis: Implications for Patient Care (P6-11.030). (PubMed, Neurology) - "Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio...Dr. Nowak has a non-compensated relationship as a Member of the Board of Directors with Myasthenia Gravis Foundation of America (MGFA) that is relevant to AAN interests or activities."

Journal • Observational data • Retrospective data • CNS Disorders • Dyslipidemia • Myasthenia Gravis

Effect of Rozanolixizumab on Ocular Symptoms in Patients with Generalized Myasthenia Gravis: A Post Hoc Item-level Analysis of Myasthenia Gravis-specific Outcomes in MycarinG (P1-11.006). (PubMed, Neurology) - P3 | "Habib has received research support from CabalettaBio. Bril has received intellectual property interests from a discovery or technology relating to health care. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Clinical • Journal • Retrospective data • CNS Disorders • Myasthenia Gravis

Effectiveness and Safety of Ravulizumab in Generalized Myasthenia Gravis (gMG): Updated Analysis from a Global Registry (P1-11.014). (PubMed, Neurology) - P | "MG-ADL total scores and MGFA classification were assessed in patients enrolled in the MG SPOTLIGHT Registry who received ravulizumab, including those who received ravulizumab only (ravu-only subgroup) or transitioned from eculizumab to ravulizumab (ecu-to-ravu subgroup) with data available prior to C5IT initiation ("pre-C5IT") and ≥1 assessment post-ravulizumab initiation ("post-ravu")...Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta...Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • CNS Disorders • Infectious Disease • Meningococcal Infections • Myasthenia Gravis • Rare Diseases

Correlation Between MG Symptoms PRO and Existing MG-Specific Outcome Scores in the Phase 3 MycarinG Study: Post Hoc Analysis (P1-11.026). (PubMed, Neurology) - P3 | "To evaluate the correlation between Myasthenia Gravis (MG) Symptoms Patient-Reported Outcome (MG Symptoms PRO) scale scores and MG Activities of Daily Living (MG-ADL) and Quantitative MG (QMG) subdomain scores using data from the Phase 3 MycarinG study (NCT03971422) of rozanolixizumab in patients with generalized MG...Habib has received research support from CabalettaBio...Bril has received intellectual property interests from a discovery or technology relating to health care. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Clinical • Journal • P3 data • Retrospective data • CNS Disorders • Fatigue • Myasthenia Gravis

Miglustat: A First-In-Class Enzyme Stabilizer for Late-Onset Pompe Disease (P10-2.012). (PubMed, Neurology) - "In a head-to-head study, cipa+mig had a similar safety profile to alglucosidase alfa...Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cabaletta Bio...Prof. parenti has nothing to disclose."

Journal • Fabry Disease • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Pompe Disease

Change in Concomitant Immunosuppressive Therapies for Generalized Myasthenia Gravis in Patients Receiving Complement C5 Inhibitor Therapies: A Retrospective Analysis of Registry Data (P7-11.028). (PubMed, Neurology) - "The current analysis included registry patients who transitioned from eculizumab to ravulizumab, had available con-IST (azathioprine, mycophenolate mofetil, intravenous immunoglobulin/plasma exchange, oral corticosteroid [OCS]) data, and received eculizumab and ravulizumab for ≥1 year...Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta...Narayanaswami has a non-compensated relationship as a Member, Board of Directors with AANEM that is relevant to AAN interests or activities. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • Retrospective data • CNS Disorders • Myasthenia Gravis • Rare Diseases

Assessment of Hospitalizations in Patients with Generalized Myasthenia Gravis (gMG) Before and During Treatment with Ravulizumab: Results from a Global Registry (P1-11.016). (PubMed, Neurology) - P | "Reductions in hospitalizations with ravulizumab were most pronounced in patients not previously treated with eculizumab, whereas the decrease in hospitalizations associated with eculizumab was maintained after switching to ravulizumab...Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta...Pulley has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Multiple law firms. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • CNS Disorders • Myasthenia Gravis • Rare Diseases

Outcomes for Patients with Generalized Myasthenia Gravis Prescribed Ravulizumab or Efgartigimod Treatment: A Retrospective Medical Record Analysis (P1-11.001). (PubMed, Neurology) - "Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta. Pulley has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Multiple law firms. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • Retrospective data • CNS Disorders • Myasthenia Gravis

Association Between Body Mass Index, Early B-cell Repopulation and Clinical Outcomes in Multiple Sclerosis (P11-1.001). (PubMed, Neurology) - "The same association persisted in subgroup analysis examining Ocrelizumab or Rituximab separately...Riley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta Bio...The institution of Dr. Xia has received research support from Genentech/Roche."

Clinical data • Journal • Retrospective data • CNS Disorders • Multiple Sclerosis

Pulmonary Manifestations in Patients with Dermatomyositis - Effect of IVIG Treatment: Data from the ProDERM Study (P3-11.011). (PubMed, Neurology) - "Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cabaletta Bio...Aggarwal has received research support from Q32. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Clinical • Journal • Dermatomyositis • Dysphonia • Immunology • Interstitial Lung Disease • Myositis • Pulmonary Disease • Respiratory Diseases

Thymectomy and Extrathymic Cancer in Myasthenia Gravis Patients (P1-11.027). (PubMed, Neurology) - "Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cabaletta Bio...Dr. Dimachkie has received publishing royalties from a publication relating to health care."

Journal • Observational data • Retrospective data • CNS Disorders • Immunology • Muscular Dystrophy • Myasthenia Gravis • Oncology • Solid Tumor • Thymic Carcinoma • Thymus Cancer

Effect Size Analysis of Cipaglucosidase Alfa Plus Miglustat Versus Alglucosidase Alfa in ERT-experienced Adults with Late-onset Pompe Disease in PROPEL (S21.003). (PubMed, Neurology) - P3 | "The randomized, double-blind PROPEL study (ATB200-03; NCT03729362) compared the efficacy and safety of the investigational two-component enzyme replacement therapy (ERT) cipa+mig with alg plus placebo in adults with late-onset Pompe disease (LOPD); 77% of patients had received ERT with alg before study entry (median ERT duration 7.4 years)...Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio...Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH."

Clinical • Journal • CNS Disorders • Fatigue • Muscular Dystrophy • Pompe Disease • Rare Diseases

Clinically Meaningful Improvement in Physical Fatigue and Muscle Weakness Fatigability with Rozanolixizumab: Post-hoc Analysis of MG Symptoms PRO Responder Rate in the MycarinG study (P4-11.001). (PubMed, Neurology) - P3 | "Habib has received research support from CabalettaBio. Dr. Bril has received intellectual property interests from a discovery or technology relating to health care."

Clinical • Journal • Retrospective data • CNS Disorders • Fatigue • Myasthenia Gravis

220: Achieving an Immune System RESET: The Next Act in CAR T Therapy (ACR Convergence 2024) - "Sponsored by Cabaletta Bio...Speakers will also provide an overview of the distinguishing features of CABA-201, a CD19-CAR T cell therapy candidate, and the RESET™ Ph 1/2 clinical program in several autoimmune diseases, including lupus, myositis, and systemic sclerosis. Not an official program of the ACR. Non-CME"

Immunology • Inflammatory Arthritis • Lupus • Myositis • Scleroderma • Systemic Sclerosis

Unlocking the Potential of CD19-CAR T Cell Therapy in Lupus & Myositis (EULAR 2024) - "Sponsored By Cabaletta Bio."

CAR T-Cell Therapy • Immunology • Inflammatory Arthritis • Lupus • Myositis

Resetting the Immune System of Patients with Autoimmune Disease (EULAR 2024) - "Sponsored By Cabaletta Bio."

Clinical • Immunology

Evolving the Potential of CAR T Cell Therapies to Autoimmunity (EULAR 2024) - "Sponsored By Cabaletta Bio."

CAR T-Cell Therapy • Immunology

Immune Reset: The Potential of CAR T Cell Therapy to Transform the Treatment of Patients with Autoimmune Disease (EULAR 2024) - "Sponsored By Cabaletta Bio. Learning Objective(s):· Gain insight into the rationale and considerations for delivering a targeted B cell reset in patients with autoimmune disease· Appraise the potential of CD19-CAR T cell therapy to reset the immune system through transient and deep B cell depletion· Learn about an investigational fully human 4-1BB costimulatory domain-containing CD19-CAR T cell therapy in patients with lupus and myositis"

CAR T-Cell Therapy • Clinical • Immunology • Inflammatory Arthritis • Lupus • Myositis

Welcome & Introduction (EULAR 2024) - "Sponsored By Cabaletta Bio."

Immunology