FDA Activity Recap: October 2024 Features IND Clearances and Company Alignment on Plans in FDA Meetings

Last month, October 2024, the CGTLive^® team was diligently tracking the FDA's activities related to the development of cell and gene therapies for the treatment of rare, complex, and otherwise challenging diseases and disorders.

The agency has continued to ramp up its activities around these therapies as more of them progress through the pipeline in tandem. Last month proved no different, with the FDA clearing investigational new drug (IND) applications for an allogeneic chimeric antigen receptor (CAR)-engineered gamma delta T-cell therapy in idiopathic inflammatory myopathy (IIM) and stiff person syndrome (SPS), the first IND clearance for a circular RNA therapy, and a number of meetings held with companies looking to submit biologics license applications (BLAs) for various advanced therapeutics currently in clinical development. Our team has highlighted these below.

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

Nanoscope Announces BLA Submission Plans for Retinitis Pigmentosa Gene Therapy MCO-010 Following Meeting With FDA

October 13, 2024 — Nanoscope Therapeutics has stated that it plans to file a BLA for MCO-010 (sonpiretigene isteparvovec), its investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy intended to treat retinitis pigmentosa (RP), following a meeting with the FDA that it characterized as “productive”.

The company noted that it intends to pursue a rolling BLA submission for MCO-010 in severe vision loss due to RP, with reference to the fast track designation that the program previously has received from the FDA. Nanoscope intends to go forward with the BLA submission in the first quarter of next year. The company additionally noted that the FDA had provided regulatory feedback in the meeting and acknowledged the planned subsequent steps.

“We are pleased with the positive interactions we have had with FDA as a result of the exceptional expertise and tireless commitment of the Nanoscope team,” Sulagna Bhattacharya, B. Tech, MBA, the cofounder and CEO of Nanoscope, said in a statement. “Our shared goal is to change lives, and together, we have advanced MCO-010 to the point of BLA submission. With every step forward, we are focused on the patients who are waiting for meaningful sight restoration. Our team looks forward to continuing the important work we have begun, along with our partners, to bring this therapy to patients who have significant unmet need.”

Adicet's T-Cell Therapy ADI-100 Snags Clearance for Evaluation in Idiopathic Inflammatory Myopathy and Stiff Person Syndrome

October 17, 2024 — Adicet Bio’s ADI-100, an investigational allogeneic CAR-engineered gamma delta T-cell therapy currently being evaluated in a phase 1 clinical trial (NCT06375993) for lupus nephritis (LN), has received clearance from the FDA of an amendment to its IND application, allowing for the recruitment of patients with IIM and SPS into the trial.

Originally, the phase 1 clinical trial was only cleared for the recruitment of patients with lupus nephritis (LN). Although, a prior IND amendment allowed for the recruitment of patients with systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and antineutrophil cytoplasmic autoantibody associated vasculitis (AAV). Adicet noted that the trial will divide patients into an arm for LN and SLE, an arm for SSc, an arm for AAV, and an arm for IIM and SPS. The company stated that patients with IIM subtypes dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, polymyositis, and overlap myositis will be eligible for participation. The treatment of patients in the IIM and SPS arm is expected to commence in the first quarter of next year.

“The FDA’s acceptance of our IND amendment to evaluate ADI-001 in patients with IIM and SPS builds on our recent momentum in autoimmune diseases, expanding our efforts to 6 autoimmune indications as we aim to bring our differentiated gamma delta T-cell therapy candidates to more patients in need of new treatment options,” Chen Schor, the president and chief executive officer at Adicet Bio, said in a statement. “Following our recent announcement highlighting clinical biomarker data which demonstrated robust B-cell depletion and preferential trafficking to tissues and organs, we believe in ADI-001’s best-in-class potential for the treatment of autoimmune diseases, and we look forward to initiating patient enrollment in IIM and SPS in the first quarter of 2025 in our ongoing phase 1 clinical program.”

Sangamo Reaches Accord With FDA on Plans for Accelerated Approval Pathway for Fabry Disease Gene Therapy ST-920

October 24, 2024 — Sangamo Therapeutics has emerged from a Type B interaction with the FDA having come into alignment with the agency on plans to pursue an accelerated approval pathway for isaralgagene civaparvove (ST-920), an investigational adeno-associated virus vector-based gene therapy product intended to treat Fabry disease.

The company noted that based on the interaction it intends to use 1 year posttreatment estimated glomerular filtration rate (eGFR) slope data from patients treated in the ongoing phase 1/2 STAAR clinical trial (NCT04046224) as an intermediate clinical end point for a planned BLA submission. The company noted that it expects to submit a BLA in the second half of next year and that the use of the accelerated approval pathway has potential to bring the therapy to market about 3 years sooner than a traditional approval pathway would allow for because an additional clinical trial will not be necessary. It also stated that the complete data set to be used to support the BLA will be reported in the first half of next year.

According to Sangamo, among 18 patients who were treated with ST-920 in STAAR who had greater than 1 year of follow-up a positive annualized eGFR slope emerged from statistically significant improvements in the mean and median eGFR levels that were recorded. The FDA noted that the eGFR slope at 104 weeks of follow-up may later be utilized for confirmation of clinical benefit.

RiboX's Circular RNA Therapy RXRG001 Cleared for Phase 1/2a Trial in Radiation-Induced Xerostomia and Hyposalivation

November 3, 2024 — RiboX Therapeutics has received clearance of an IND application from the FDA for RXRG001, an investigational circular RNA therapy, enabling a phase 1/2a clinical trial that the company will refer to as the SPRINX-1 Study, in patients with radiation-induced-xerostomia (RIX) and hyposalivation.

Notably, RiboX stated that this is the first IND clearance the FDA has granted for any circular RNA therapy. The company noted that RIX and hyposalivation are common adverse effects produced in patients who have undergone radiation therapy for head and neck cancers (HNC).

"RIX is a devastating life-long health issue for patients with HNC,” Yizhen Xu, MD, PhD, the chief medical officer of RiboX, said in a statement. “Although radiation therapy improves patients' survival, its damage to the salivary glands leads to an impaired quality of life of many cancer survivors. RXRG001 may offer a potential effective and sustained therapeutic option for RIX patients. We are excited to conduct clinical trials to further evaluate its efficacy and safety in patients."