Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
LFG316 · 6 trials · 10 indications
The primary efficacy variable for assessing the effect of LFG316 over the first 4 weeks of treatment was response rate where a patient was considered a responder if the percentage reduction from baseline in serum lactate dehydrogenase (LDH) was at least 60% at any time up to and including week 4 for that patient.
Lactate dehydrogenase (LDH) levels were measured in serum samples and the percentage change from baseline was calculated. For serum LDH, baseline was the average of all pre-dose measurements.
Response rate as defined by: 1. An improvement of 2 or more steps in vitreous haze (scale of 0 to 4), relative to baseline OR 2. An improvement of 10 or more letters in visual acuity (VA), relative to baseline OR 3. An improvement of 2 or more steps in anterior chamber cells (ACC) score (scale of 0 to 4), relative to baseline OR 4. Absence of chorioretinal lesions as determined by the investigator
Remission (complete response) was defined as any patient who had: * a vitreous haze score of 0 or 0.5 (scale of 0 to 4) in the study eye, AND * an anterior chamber cell score of 0 (scale of 0 to 4), AND * no chorioretinal lesions in the study eye, AND * was off all immune modulatory therapy (systemic, corticosteroids and topical), AND * without any worsening of uveitis during the trial.
Number or retreatments with anti-VEGF treatments will be recorded
Geographic atrophy (GA) lesion growth measured by fundus autofluorescence (FAF) from baseline to Day 505.
Number is the Estimated Difference (95% CI) in lesion size.
This primary outcome (for Part B) is reported under the Adverse Events section.
The following PK parameters were determined from the plasma concentration time profile of LFG316 using a non-compartmental method: AUClast: Area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration AUCinf: Area under the plasma concentration-time curve from time zero to infinity
| Arm | Type | Description |
|---|---|---|
| LFG316 then LNP023 | EXPERIMENTAL | During treatment periods 1 to 3, the regimen included LFG316 at a dosage of 20 mg/kg administered as an intravenous (i.v.) infusion biweekly. In treatment period 4, the patients were given: * LFG316 at a dosage of 20 mg/kg as an i.v. infusion every two weeks for four weeks, amounting to two infusions in total. * LNP023 at a dose of 200 mg administered orally twice daily (b.i.d.) for approximately 20 weeks, with four 50 mg capsules taken at each administration. |
| LFG316 -Intravitreal Injection | EXPERIMENTAL | - |
| Conventional Therapy | ACTIVE_COMPARATOR | - |
| LFG316 | EXPERIMENTAL | - |
| Sham | SHAM_COMPARATOR | - |
| LFG316 higher dose | EXPERIMENTAL | LFG316 10 mg/100 μL |
| LFG316 lower dose | EXPERIMENTAL | LFG316 5 mg/ 50 μL |
| LFG316 + IVIG | EXPERIMENTAL | - |
| LFG316 alone | EXPERIMENTAL | - |
| LFG316 0.15mg | EXPERIMENTAL | - |
| LFG316 0.5mg | EXPERIMENTAL | - |
| LFG316 1.5mg | EXPERIMENTAL | - |
| LFG316 5mg | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| LFG316 | BIOLOGICAL | LFG316 20 mg/kg was administered to all patients enrolled in the study: * Treatment Periods 1 to 3: LFG316 20 mg/kg as i.v. infusion every 2 weeks * Treatment Period 4: LFG316 20 mg/kg as i.v. infusion every 2 weeks for 4 weeks (total 2 infusions). |
| LNP023 | DRUG | Treatment Period 4: LNP023 200 mg b.i.d. for approximately 20 weeks. Four capsules (each 50 mg) were administered each time study medication was taken. |
| Conventional Therapy | DRUG | Conventional Therapy administered in accordance with its prescribing info. |
| Placebo | DRUG | Placebo will be administered as sham injections. Sham injections will involve placement of the syringe hub against the sclera, without use of a needle. |
| Sham | DRUG | Sham injection (akin to intravitreal injection but without intravitreal needle; no investigational drug given) |
| LFG316 Lower dose | DRUG | LFG316 5 mg/50 μL solution for IVT Injection |
| IVIG | DRUG | IVIG single dose |
Inclusion criteria: 1. Written informed consent obtained before any assessment were performed. 2. Male and female patients \>= 18 years old with a diagnosis of PNH prior to screening. Based on local requirements (applicable in Czech Republic) only patients between the age of 18 to 65 (inclusive) wi...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE3 | Pozelimab, Cemdisiran |
| Annexon, Inc. | ANNX | 1 | PHASE3 | Vonaprument |
| Belite Bio, Inc. ADR | BLTE | 1 | PHASE3 | Tinlarebant |
| Johnson & Johnson | JNJ | 1 | PHASE2 | JNJ-81201887 |
| AbbVie, Inc. | ABBV | 1 | PHASE1 | ABBV-6628, SYFOVRE |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR446597, Sham Comparator |
| Ocugen Inc | OCGN | 1 | PHASE1 | OCU410 |
| Apellis Pharmaceuticals, Inc. | APLS | 1 | - | Pegcetacoplan |
LFG316 is an investigational monoclonal antibody being studied for multiple conditions, including advanced age-related macular degeneration, geographic atrophy, wet AMD, paroxysmal nocturnal hemoglobinuria, non-infectious intermediate uveitis, and kidney transplantation. It is administered intravitreally for ophthalmologic indications and has been evaluated in clinical trials for these conditions.
LFG316 is a monoclonal antibody that targets complement component C5. By binding to C5, it is designed to inhibit the complement cascade, which is implicated in diseases like age-related macular degeneration and paroxysmal nocturnal hemoglobinuria. This mechanism is being investigated across several clinical trials.
LFG316 is being developed by Novartis AG, a global healthcare company traded on the New York Stock Exchange under the ticker symbol NVS. Novartis has sponsored clinical trials evaluating LFG316 for various indications, including age-related macular degeneration and paroxysmal nocturnal hemoglobinuria.
LFG316 has completed Phase 1 and Phase 2 clinical trials. A Phase 1 study in advanced age-related macular degeneration and multiple Phase 2 studies in geographic atrophy, wet AMD, and paroxysmal nocturnal hemoglobinuria have all been completed. The drug remains investigational and has not been approved by regulatory authorities.
LFG316 has been studied in several completed trials, including NCT01255462 (Phase 1, advanced AMD), NCT01527500 (Phase 2, geographic atrophy), NCT01535950 (Phase 2, wet AMD), and NCT02534909 (Phase 2, paroxysmal nocturnal hemoglobinuria). These trials enrolled a total of 158 participants across the United States, Czechia, Japan, and Lithuania.
LFG316 is a distinct monoclonal antibody targeting complement component C5, similar in mechanism to other C5 inhibitors but with its own unique structure and development program. It is being developed by Novartis for both ophthalmologic and hematologic indications, and its clinical trial results are specific to this asset.