UNIVERSITY OF KANSAS

AskBio's AB-1005 Gene Therapy for Parkinson's Disease Gains FDA RMAT Designation and Enrolls First Phase II Participants

4 months ago

• AskBio's AB-1005, a gene therapy for Parkinson's disease, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, potentially expediting its development. • The RMAT designation was supported by positive 36-month Phase Ib data, demonstrating a favorable safety profile and encouraging trends in clinical outcome measures. • The Phase II REGENERATE-PD trial, evaluating AB-1005 in moderate-stage Parkinson's, has randomized its first participants across sites in the US, with plans to expand to Europe. • AB-1005 delivers glial cell line-derived neurotrophic factor (GDNF) to promote the survival of dopaminergic neurons, addressing a critical unmet need in Parkinson's treatment.

AbbVie's Vyalev for Parkinson's Disease Incorporates Key Innovations from KU Chemist

5 months ago

• Vyalev, a new subcutaneous drug therapy by AbbVie for Parkinson's disease, has been approved by the FDA in October 2024 after successful Phase 3 trials. • The therapy uses a wearable pump to deliver levodopa/carbidopa, providing consistent dosage and reducing motor fluctuations in Parkinson's patients. • University of Kansas chemist Valentino Stella contributed to the drug's development by discovering forms of L-dopa and carbidopa suitable for infusion. • Clinical trials showed Vyalev significantly increased 'on' time (optimal motor control) and decreased 'off' time (return of symptoms) compared to oral treatments.

FDA Approves Supernus' ONAPGO for Motor Fluctuations in Advanced Parkinson's Disease

6 months ago

• The FDA has approved ONAPGO (apomorphine hydrochloride) as the first subcutaneous apomorphine infusion device for treating motor fluctuations in advanced Parkinson's disease. • ONAPGO, a wearable device, provides continuous subcutaneous apomorphine infusion, offering more consistent control of OFF time for Parkinson's patients. • Clinical trials demonstrated that ONAPGO significantly reduced daily OFF time by 2.6 hours compared to placebo, with improvements seen as early as week 1. • Supernus plans to launch ONAPGO in the U.S. in the second quarter of 2025, supported by a dedicated nurse education program and access support.

Bristol Myers Squibb and 2seventy bio Discontinue Enrollment in Phase 3 KarMMa-9 Trial for Abecma in Newly Diagnosed Multiple Myeloma

8 months ago

• Bristol Myers Squibb and 2seventy bio halted enrollment in the Phase 3 KarMMa-9 trial evaluating Abecma (idecabtagene vicleucel) plus lenalidomide maintenance versus lenalidomide alone in newly diagnosed multiple myeloma (NDMM) patients. • The decision was driven by challenges in patient enrollment due to the evolving treatment landscape, where more intense induction therapies result in fewer eligible patients for the trial. • Advances in induction therapies now lead to complete responses or better in over 70% of NDMM patients post-transplant, reducing the eligible population for KarMMa-9, which aimed for patients with suboptimal responses. • Both companies will collaborate with investigators to determine the next steps for patients already participating in the KarMMa-9 study, while reaffirming their commitment to Abecma in multiple myeloma treatment.