RARE Daily

FDA Grants Fast Track Designation to Belite Bio, Graphite Bio, and Pliant for Rare Disease Treatments

May 3, 2022

The U.S. Food and Drug Administration granted Fast Track designation to three companies’ experimental therapeutics for the treatment of rare diseases: Belite Bio’s LBS-008 for the treatment of Stargardt disease; Graphite Bio’s GPH101 for the treatment of sickle cell disease, and Pliant Therapeutics’ PLN-74809 for the treatment of idiopathic pulmonary fibrosis.

FDA’s Fast Track designation is intended to facilitate and expedite the development and review of new drugs to treat serious or life-threatening conditions. To qualify, available clinical and non-clinical data need to demonstrate the potential to address unmet medical need. The benefits of Fast Track designation include opportunities for frequent meetings with the FDA to discuss trial design, development plans and data needed to support drug approval, as well as the ability to submit a New Drug Application (NDA) on a rolling basis, and eligibility for priority review, if relevant criteria are met.

Stargardt disease (STGD1) is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by a dysfunctional retina-specific gene (ABCA4) which results in massive accumulation of toxic vitamin A byproducts in the retina leading to retinal cell death and progressive loss of central vision.

Belite Bio’s LBS-008 is a novel oral therapy in late-stage development that prevents the buildup of toxins in the eye that cause STGD1 and works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), a carrier protein that transports retinol to the eye. Belite Bio

expects the next near-term data readout in its STGD1 phase 2 trial to occur in the last quarter of 2022 when all subjects have completed 12 months of treatment.

Sickle cell disease (SCD) is a serious, life-threatening inherited blood disorder and the most prevalent monogenic disease worldwide. Graphite Bio’s GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the mutation in the beta-globin gene to decrease sickle hemoglobin (HbS) production and restore adult hemoglobin (HbA) expression, thereby potentially curing SCD.

Graphite Bio is evaluating GPH101 in the CEDAR trial, an open-label, multi-center phase 1/2 clinical trial designed to assess the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics in patients with severe SCD.

Idiopathic pulmonary fibrosis (IPF) is a deadly, age-related lung disease of unknown cause with few treatment options. Approximately 60 to 80 percent of patients with IPF die within five years of diagnosis. Patients experience debilitating symptoms, including shortness of breath and difficulty performing routine functions, such as walking and talking. Currently, there is no pharmacological cure for IPF with neither of the approved two therapies demonstrating an ability to stop the progression of IPF.

Pliant Therapeutics’ lead drug candidate, PLN-74809, is currently being tested as part of the INTEGRIS-IPF phase 2a clinical trial. Pliant anticipates topline data from this randomized, double-blind, placebo-controlled trial in patients with IPF, in mid-2022.

Author: Rare Daily Staff

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