Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
QR-010 · 2 trials · 1 indication
The primary endpoint was the within-subject change from baseline in total chloride transport as measured by NPD, after the Chloride-free+isoproterenol solution (Cl-free+iso), and was based on the average measurements of both nostrils. To provide baseline stability, baseline was defined as the average of the two most recent pre-dose values, where each pre-dose value was the average of two nostrils. A negative change from baseline of Cl-free+iso shows an improvement.
Number of subjects experiencing at least one treatment emergent adverse events (TEAEs)
Assessment of severity of treatment emergent adverse events (TEAEs). Severity is graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events Modified for CF (CTCAE v4.03). For events not present in this listing the following grading was applied: Mild: Asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated; Moderate: Minimal, local, or noninvasive intervention indicated; discomfort sufficient to reduce or interfere with daily activities; Severe: Medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization may be indicated; disabling; limits self-care with significant interference with daily activities; incapacitating with inability to perform self care activities of daily living; Life-threatening: Urgent intervention indicated; immediate risk of death.
DLT's were defined as an allergic reaction, acute bronchospasm or acute AEs of interest requiring (immediate) medical intervention.
| Arm | Type | Description |
|---|---|---|
| ΔF508 Homozygous | EXPERIMENTAL | QR-010 administered intranasally as an atomized liquid 10 mg (5 mg per nostril), 3 times weekly for 4 weeks. |
| ΔF508 Compound Heterozygous | EXPERIMENTAL | QR-010 administered intranasally as an atomized liquid 10 mg (5 mg per nostril), 3 times weekly for 4 weeks. |
| QR-010 | EXPERIMENTAL | QR-010 administered via inhalation either as a single dose or three times weekly for four weeks. |
| Placebo | PLACEBO_COMPARATOR | Placebo (normal saline) administered via inhalation either as a single dose or three times weekly for four weeks. |
| Name | Type | Description |
|---|---|---|
| QR-010 | DRUG | Single-stranded RNA antisense oligonucleotide in isoosmolar solution |
| Placebo | DRUG | Normal Saline |
Inclusion Criteria: * Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test (sweat chloride) of \> 60 mmol/L * Nasal potential difference (NPD) measurement at Screening consistent with CF * Confirmation of CFTR gene mutations homozygous or compound heterozygous for t...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
QR-010 is an investigational small molecule being developed for the treatment of cystic fibrosis, specifically in patients with the ΔF508 CFTR mutation. It is designed to address the underlying genetic defect that causes the disease. QR-010 is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
QR-010 is being developed by ProQR Therapeutics N.V., a biopharmaceutical company traded on the NASDAQ under the ticker symbol PRQR. The company is conducting clinical trials to evaluate the safety and efficacy of QR-010 in patients with cystic fibrosis.
QR-010 is in Phase 1 clinical development. Two Phase 1 trials have been completed, with a total of 88 participants enrolled. The drug is investigational and has not received regulatory approval. ProQR Therapeutics is continuing to evaluate QR-010 for the treatment of cystic fibrosis.
QR-010 has been studied in two completed Phase 1 clinical trials. The first, NCT02532764, was a dose escalation study in homozygous ΔF508 cystic fibrosis patients with 70 participants. The second, NCT02564354, was an exploratory study in subjects with the ΔF508 CFTR mutation, enrolling 18 participants.
QR-010 is a distinct investigational small molecule developed by ProQR Therapeutics for cystic fibrosis. It is designed to target the underlying cause of the disease in patients with the ΔF508 CFTR mutation. It is not the same as other approved cystic fibrosis therapies, as it is still in clinical development.