Data Roundup: April 2024 Features Updates from AACR, AAN, in Muscular Dystrophy and Cancer

Last month, April 2024, the CGTLive® team was diligently tracking the latest data readouts and published literature on cell and gene therapies within oncology, ophthalmology, and rare diseases.

As more and more innovative therapies enter the clinical trial field, more data is accrued every month, buoying excitement in the field and sometimes making or breaking the fates of small biotech companies. Last month delivered promising data updates presented at the 2024 American Association for Cancer Research (AACR) Annual Meeting and 2024 American Academy of Neurology Annual Meeting, for oncologic indications and neurological disease. Our team has highlighted these and other updates below.

Click the read more buttons for more details and information about each update.

Allogeneic CAR-T Yields Some Responses in Multiple Myeloma After Anti-BCMA Therapy

April 5, 2024 - Allogeneic P-BCMA-ALLO1 chimeric antigen receptor (CAR) T-cell therapy yielded some responses in patients with relapsed/refractory multiple myeloma whose disease had progressed after prior BCMA-targeted therapy.

"New treatment options are urgently needed for these patients, which is why I'm encouraged by these impressive Phase 1 subgroup results, which may be the first report of an allogeneic CAR-T therapy showing clinical activity in heavily pretreated patients whose myeloma has progressed after multiple BCMA-targeted immunotherapies,” Bhagirathbhai Dholaria, MD, Associate Professor of Medicine (Hematology/Oncology), Vanderbilt-Ingram Cancer Center in Nashville, Tennessee, said.

Dholaria and colleagues found that P-BCMA-ALLO1 was well tolerated with no dose limiting toxicities or cases of graft versus host disease. Three participants developed cytokine release syndrome (grade 2) and 1 developed grade 2 immune effector cell neurotoxicity syndrome. Three participants (60%) had a response with a best response of very good partial response (VGPR). The 2 participants who did not respond had previously received and failed to achieve clinical response with teclistamab. One participant who achieved a VGPR had previously received both teclistamab and CAR T-cell therapy.

ASC Therapy Does Not Show Significant Difference Over Placebo in Treating Xerostomia

April 5, 2024 - Adipose-derived mesenchymal stem/stromal cell (ASC) therapy did not significantly improve xerostomia in patients with previous head and neck cancer compared with placebo, according to results of a single-center, phase 2, randomized trial (NCT04776538).

“We could not confirm superiority of the ASC relative to placebo. ASC therapy significantly improved UWS in previous patients with head and neck cancer, whereas placebo resulted in an insignificant increase,” Kathrine Kronberg Jakobsen, MD, PhD, Department of Otorhinolaryngology, Head and Neck Surgery & Audiology, Copenhagen University Hospital- Rigshospitalet, and Section for Biostatistics and Evidence-Based Research, the Parker Institute, Copenhagen University Hospital- Bispebjerg and Frederiksberg, Denmark, and colleagues wrote.

The investigators found that UWS statistically significantly increased by 0.04 mL per minute (38%) after ASC therapy (95% CI, 0.02-0.06) compared with baseline. UWS did not significantly increase from baseline after placebo treatment (increase, 0.01 mL/minute; 21%; [95% CI, 0.01-0.04])

RGX-202 Is Well-Tolerated At Multiple Doses Among Patients With DMD

April 16, 2024 - RGX-202 gene therapy appears to be well-tolerated at both dose levels assessed with no serious adverse events (SAEs) at the 25-week point post-administration, as of the February 28, 2024, cut-off date.

“Robust RGX-202 microdystrophin expression was observed at both dose levels in all ages. Encouraging observations of early improvements in daily activities associated with strength and function in clinic and caregiver videos,” Aravindhan Veerapandiyan, MD​, child neurologist and assistant professor, Arkansas Children’s Hospital​ (ACH), and director, Comprehensive Neuromuscular Program, and codirector, Muscular Dystrophy Association Care Center, ACH, said.

Microdystrophin expression at the 3-month mark was deemed “robust” at both dose levels by the investigators, with levels of 38.8%, 83.4%, and 11.1% among those aged 4 to 5 years (Patient 1, age 4.4 years), 6 to 7 years (Patient 3, age 6.6 years), and 8 to 11 years at screening (Patient 2, age 10.5 years) compared with control, respectively, for those who received the lower dose. Among the 1 patient with follow-up who received the higher dose (8 to 11 years group), the expression was 75.7% compared with control levels.

First Patient Treated With Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Shows Swallowing Improvements

April 18, 2024 - The first patient treated in Benitec Biopharma’s phase 1b/2a clinical trial (NCT06185673) evaluating BB-301, its investigational adeno-associated virus (AAV) vector-based silence and replace gene therapy for the treatment of oculopharyngeal muscular dystrophy (OPMD)-related dysphagia, has shown improvements in swallowing at 90 days posttreatment.

We are highly encouraged by these early clinical trial results and for the hope that they may offer to patients and caregivers, and we look forward to reporting additional results and continuing to treat patients as they enter the dosing portion of the study from the Natural History observational lead-in period,” Jerel A. Banks, MD, PhD, the executive chairman and CEO of Benitec Biopharma, said.

At the 90 day time point, the patient showed an improvement at 90 days posttreatment from their natural history study average for thin liquid (5.2 units improvement, -57.1%), moderately thick liquid (5.2 units, -26.0%), extremely thick liquid (3.3 units, -15.1%), and solid food (7.1 units, 39.4%) on the videofluoroscopic swallowing studies assessments of pharyngeal area at maximum constriction to determine pharyngeal constrictor muscle function during swallowing, as well as improvements on cold water timed drinking test and the Sydney Swallow Questionnaire (SSQ).