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Label indication SKYCLARYS is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. SKYCLARYS is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. ( 1 ) FDA label
Also known as [14C]-Omaveloxolone, Omaveloxolone Ophthalmic Suspension 0.5%, Omaveloxolone 0.5%, Omaveloxolone Ophthalmic Suspension 1.0%, Omaveloxolone 1.0%, Omaveloxolone Lotion 0.5%, RTA 408, SKYCLARYS
Omaveloxolone · 10 trials · 14 indications
The mFARS is a validated and sensitive rating scale that was developed to quantitatively assess the severity of the neurologic features of FA in adults and adolescents. Scores on the mFARS range from 0 to 93, with lower scores indicating better neurological function. The subscales of the mFARS assessment and maximum score for each subscale are: bulbar function (Subscale A; 2 assessments of speech and cough; maximum score = 5), upper limb coordination (Subscale B; 5 assessments of coordination of movement and function in arms and hands with each limb scored individually; maximum score = 36), lower limb coordination (Subscale C; 2 assessments of coordination of movement and function of lower limbs with each limb scored individually; maximum score = 16), and upright stability (USS, Subscale E; 9 assessments of sitting posture, stance, tandem walk, and gait assessments; maximum score = 36).
The mFARS is a validated and sensitive rating scale that was developed to quantitatively assess the severity of the neurologic features of FA in adults and adolescents. Scores on the mFARS range from 0 to 93, with lower scores indicating better neurological function. The subscales of the mFARS assessment and maximum score for each subscale are: bulbar function (Subscale A; 2 assessments of speech and cough; maximum score = 5), upper limb coordination (Subscale B; 5 assessments of coordination of movement and function in arms and hands with each limb scored individually; maximum score = 36), lower limb coordination (Subscale C; 2 assessments of coordination of movement and function of lower limbs with each limb scored individually; maximum score = 16), and upright stability (USS, Subscale E; 9 assessments of sitting posture, stance, tandem walk, and gait assessments; maximum score = 36).
The C-SSRS is a low-burden measure of the spectrum of suicidal ideation and behavior that was developed to assess severity and track suicidal events through any treatment of individuals ≥ 6 years of age. The C-SSRS is a clinical interview providing a summary of both ideation and behavior that can be administered by the clinician during any evaluation or risk assessment to identify the level and type of suicidality present. The assessment includes "yes" or "no" responses for 5 questions each, related to suicidal ideation (wish to be dead, non-specific active suicidal thoughts, active suicidal ideation with any methods, active suicidal ideation with some intent, active suicidal ideation with specific plan) and suicidal behavior (preparatory acts or behavior, aborted attempt, interrupted attempt, actual attempt, suicide). Numeric ratings are provided for severity of ideation, from 1 to 5, with 5 being the most severe.
Assessment of Tanner stages (a scale of physical development) will be performed by a medical doctor experienced with this assessment. Tanner score ranges from Stage 1 (childhood) to Stage 5 (full physical maturity). Information regarding Tanner staging will be collected at baseline for all participants and will be stopped once the participant reaches Tanner Stage 5 in all gender-appropriate scales.
Assessment of Tanner stages (a scale of physical development) will be performed by a medical doctor experienced with this assessment. Tanner score ranges from Stage 1 (childhood) to Stage 5 (full physical maturity). Information regarding Tanner staging will be collected at baseline for all participants and will be stopped once the participant reaches Tanner Stage 5 in all gender-appropriate scales.
Cycle ergometry using a stationary recumbent bike was used to conduct maximal exercise testing. Peak work is defined as the workload at which patients reach maximal volition (defined as an inability to continue to exercise due to exhaustion). Change of peak workload during exercise testing was measured at baseline, Week 4, and Week 12. Change from baseline at Week 12 reported.
Cycle ergometry using a recumbent stationary bicycle was used to conduct maximal exercise testing and workload was increased incrementally. Peak work is defined as the workload at which participants reach maximal volition (defined as an inability to continue to exercise due to exhaustion). A positive change from baseline suggests an improvement.
An adverse event (AE) was any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug whether or not it is considered to be study drug related. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as any AEs, regardless of relationship to study drug, that had an onset or worsened in severity on or after the first dose of study drug.
The Friedreich Ataxia Rating Scale (FARS) is a neurological-exam-based rating scale with five sections: Bulbar (section A), Upper Limb Coordination (section B), Lower Limb Coordination (section C), Peripheral Nervous System (section D), and Upright Stability (section E). mFARS is the sum of sections A (score 0 to 11), B (score 0 to 36), C (score 0 to 16), and E (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function.
An AE was any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug whether or not it is considered to be study drug related. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as any AEs, regardless of relationship to study drug, that had an onset or worsened in severity on or after the first dose of study drug.
CTCAE Radiation dermatitis scoring: Grade 0 = No radiation dermatitis; Grade 1 = Faint erythema or dry desquamation; Grade 2 = Moderate to brisk erythema, patchy moist desquamation, mostly confined to skin folds and creases, moderate edema; Grade 3 = Moist desquamation in areas other than skin folds and creases, bleeding induced by minor trauma or abrasion; Grade 4 = Life-threatening consequences, skin necrosis or ulceration of full thickness dermis, spontaneous bleeding from involved site, skin graft indicated; Grade 5 = Death
Count of central corneal endothelial cells 12 weeks post cataract surgery, compared to baseline
Count of participants who had absence of anterior chamber cells at Day 15. White blood cells were counted. In a healthy eye, the anterior chamber should not have any blood cells present. "Yes" indicates an absence of anterior chamber cells. Data for visits after a patient was discontinued for lack of efficacy were imputed as failures and missing data were imputed using last observation carried forward.
Participants were asked to report their pain on Day 4 using the Numerical Pain Rating Scale (NPRS) for patients receiving active drug compared to patients receiving placebo. The NPRS is an 11-point numeric scale, which ranges from "0" representing "no pain" to "10" representing the "worst pain imaginable". Lower scores indicate less pain. Participants reporting '0' were recorded as "Yes" while patients reporting any other pain score were recorded as "No". Data for visits after a patient was discontinued for lack of efficacy were imputed as failures and missing data were imputed using last observation carried forward.
An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with use of a medicinal (investigational) product, whether or not related to medicinal (investigational) product. SAE is any untoward medical occurrence that at any dose results in death, in the view of investigator, places the participant at immediate risk of death (life-threatening event), requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in congenital anomaly/birth defect or is medically important event.
Vital signs, including blood pressure (BP), heart rate (HR), and oral body temperature, will be assessed.
Pharmacokinetics will be assessed by blood sampling for omaveloxolone to determine maximum observed concentration (Cmax).
Pharmacokinetics will be assessed by blood sampling for omaveloxolone to determine area under the curve (AUC).
Best overall response rate (ORR) is defined as the proportion of patients with complete or partial tumor size reduction according to RECIST v1.1 criteria. Stable disease is not a component of ORR. Complete response: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial reduction: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. The first occurrence of a response is considered an unconfirmed response. A CR or PR which persists to the next tumor burden assessment is then considered a confirmed response. Confirmed plus unconfirmed best overall response are presented. A subject may be counted twice if best unconfirmed response and best confirmed response are different.
To determine the recommended Phase 2 dose of RTA 408 following oral administration to patients with metastatic or incurable NSCLC or melanoma that is relapsed, refractory after standard of care therapy, or for which standard of care therapy is not appropriate.
| Arm | Type | Description |
|---|---|---|
| Part 1: Omaveloxolone | EXPERIMENTAL | Participants will receive a single oral dose of omaveloxolone once a day (QD) for up to 52 weeks in Part 1 of the study. |
| Part 1: Placebo | PLACEBO_COMPARATOR | Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study. |
| Part 2A Continued Efficacy Evaluation: Omaveloxolone | EXPERIMENTAL | Participants will receive a single oral dose of omaveloxolone, QD for up to 104 weeks in Part 2A of the study. |
| Part 2B Safety: Omaveloxolone | EXPERIMENTAL | Participants will receive a single oral dose of open-label omaveloxolone, QD for up to 104 weeks in Part 2B of the study. |
| omaveloxolone Capsules 2.5 mg and 5 mg | EXPERIMENTAL | omaveloxolone (RTA 408) Capsules, 2.5 mg taken orally once daily for 2 weeks, then 5 mg taken orally once daily for 10 weeks |
| omaveloxolone Capsules 10 mg | EXPERIMENTAL | omaveloxolone (RTA 408) capsules, 10 mg taken orally once daily for 12 weeks |
| Placebo Capsules | PLACEBO_COMPARATOR | Placebo capsules taken orally once daily for 12 weeks |
| omaveloxolone Capsules 20 mg | EXPERIMENTAL | omaveloxolone (RTA 408) Capsules, 20 mg taken orally once daily for 12 weeks. |
| omaveloxolone Capsules 40 mg | EXPERIMENTAL | omaveloxolone (RTA 408) Capsules, 40 mg taken orally once daily for 12 weeks. |
| omaveloxolone Capsules 80 mg | EXPERIMENTAL | omaveloxolone (RTA 408) Capsules, 80 mg taken orally once daily for 12 weeks. |
| omaveloxolone Capsules 160 mg | EXPERIMENTAL | omaveloxolone (RTA 408) Capsules, 160 mg taken orally once daily for 12 weeks. |
| Part 1: Omaveloxolone 2.5 and 5 mg | EXPERIMENTAL | Participants received omaveloxolone 2.5 mg, oral capsule, QD for 2 weeks, followed by 5 mg Omaveloxolone, oral capsule, QD for 10 weeks. |
| Part 1: Omaveloxolone 10 mg | EXPERIMENTAL | Participants received omaveloxolone 10 mg, oral capsule, QD for 12 weeks. |
| Part 1: Omaveloxolone 20 mg | EXPERIMENTAL | Participants received omaveloxolone 20 mg, oral capsule, QD for 12 weeks. |
| Part 1: Omaveloxolone 40 mg | EXPERIMENTAL | Participants received omaveloxolone 40 mg, oral capsule, QD for 12 weeks. |
| Part 1: Omaveloxolone 80 mg | EXPERIMENTAL | Participants received omaveloxolone 80 mg, oral capsule, QD for 12 weeks. |
| Part 1: Omaveloxolone 160 mg | EXPERIMENTAL | Participants received omaveloxolone 160 mg, oral capsule, QD for 12 weeks. |
| Part 1: Omaveloxolone 300 mg | EXPERIMENTAL | Participants received omaveloxolone 300 mg, oral capsule, QD for 12 weeks. |
| Part 2: Omaveloxolone 150 mg | EXPERIMENTAL | Participants received omaveloxolone 150 mg, oral capsule, QD for 48 weeks. |
| Part 2: Placebo | PLACEBO_COMPARATOR | Participants received placebo, oral capsule, QD for 48 weeks. |
| OLE: Placebo/ Omaveloxolone | EXPERIMENTAL | All eligible participants who had received either omaveloxone or placebo in Part 1 and eligible participants who received placebo in Part 2 received omaveloxone, 150 mg, oral capsule, QD, until the drug was available through commercial channels or alternate post-trial access mechanisms, or until participant withdrawal, whichever was sooner (up to approximately 370 weeks). |
| OLE: Omaveloxolone/ Omaveloxolone | EXPERIMENTAL | All eligible participants who had received omaveloxone in Part 2 continued to receive omaveloxolone, 150 mg, oral capsule, QD until the drug was available through commercial channels or alternate post-trial access mechanisms, or until participant withdrawal, whichever was sooner (up to approximately 370 weeks). |
| omaveloxolone (RTA 408) Lotion 0.5% | EXPERIMENTAL | Omaveloxolone Lotion at a fixed dose of 0.5% administered topically to the radiation area, twice daily for approximately 9 weeks (up to a maximum of 16 weeks) |
| omaveloxolone (RTA 408) Lotion 3% | EXPERIMENTAL | Omaveloxolone Lotion at a fixed dose of 3% administered topically to the radiation area, twice daily for approximately 9 weeks (up to a maximum of 19 weeks) |
| Vehicle Lotion | PLACEBO_COMPARATOR | Vehicle Lotion administered topically to the radiation area, twice daily for approximately 9 weeks (up to a maximum of 16 weeks) |
| Vehicle Ophthalmic Solution | PLACEBO_COMPARATOR | A single drop of Vehicle Ophthalmic solution was instilled into the study eye twice daily (approximately 12 hours apart) for a maximum of 28 days, beginning 3 to 7 days prior to cataract surgery and continuing on the day of surgery and for 3 weeks after surgery |
| Omaveloxolone Opthalmic Suspension 0.5% | EXPERIMENTAL | A single drop of Omaveloxolone Ophthalmic suspension 0.5% was instilled into the study eye twice daily (approximately 12 hours apart) for a maximum of 28 days, beginning 3 to 7 days prior to cataract surgery and continuing on the day of surgery and for 3 weeks after surgery |
| Omaveloxolone Opthalmic Suspension 1% | EXPERIMENTAL | A single drop of Omaveloxolone Ophthalmic suspension 1.0% was instilled into the study eye twice daily (approximately 12 hours apart) for a maximum of 28 days, beginning 3 to 7 days prior to cataract surgery and continuing on the day of surgery and for 3 weeks after surgery |
| Omaveloxolone Ophthalmic Suspension 1.0% | EXPERIMENTAL | Patients will receive a single drop of Omaveloxolone Ophthalmic suspension 1.0% instilled into the study eye twice daily (approximately 12 hours apart) for 14 days, beginning 24 ± 6 hours after surgery |
| Omaveloxolone Ophthalmic Suspension 0.5% | EXPERIMENTAL | Patients will receive a single drop of Omaveloxolone Ophthalmic suspension 0.5% instilled into the study eye twice daily (approximately 12 hours apart) for 14 days, beginning 24 ± 6 hours after surgery |
| Placebo | PLACEBO_COMPARATOR | Patients will receive a single drop of vehicle for Omaveloxolone Ophthalmic suspension was instilled into the study eye twice daily (approximately 12 hours apart) for 14 days, beginning 24 ± 6 hours after surgery |
| Part 1 and 2: Cohort A1 | EXPERIMENTAL | Cohort A1 will contain participants 12 to \<16 years of age. Participants will receive a single oral dose of omaveloxolone, 150 milligrams (mg), capsule, on Day 1 of the treatment period of part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian population pharmacokinetics (popPK) analyses. |
| Part 1 and 2: Cohort A2 | EXPERIMENTAL | Cohort A2 will contain participants 12 to \<16 years of age. Participants will receive a single oral dose of omaveloxolone, capsule, at a dosage level determined by a Bayesian popPK analysis using the data from Cohort A1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses. |
| Part 1 and 2: Cohort B1 | EXPERIMENTAL | Cohort B1 will contain participants 7 to \<12 years of age and will initiate in parallel with Cohort A2. Participants will receive a single oral dose of omaveloxolone, capsule, at a dosage level determined by a Bayesian popPK analysis using the data from Cohort A1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses. |
| Part 1 and 2: Cohort C1 | EXPERIMENTAL | Cohort C1 will contain participants 2 to \<7 years of age. Participants will receive a single oral dose of omaveloxolone, capsule, at a dosage level determined by a Bayesian popPK analysis using the data from cohorts A1, A2, and B1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses. |
| Part 1 and 2: Cohort A3 | EXPERIMENTAL | Cohort A3 will contain participants 12 to \<16 years of age. Participants will receive a single oral dose of omaveloxolone, capsule, at a dosage level determined by a Bayesian popPK analysis using the data from cohorts A1, A2, and B1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses. |
| Part 1 and 2: Cohort B2 | EXPERIMENTAL | Cohort B2 will contain participants 7 to \<12 years of age and will initiate in parallel with Cohort A3. Participants will receive a single oral dose of omaveloxolone, capsule, at a dosage level determined by a Bayesian popPK analysis using the data from cohorts A1, A2, and B1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses. |
| Part 1 and 2: Cohort C2 | EXPERIMENTAL | Cohort C2 will contain participants 2 to \<7 years of age. Participants will receive a single oral dose of omaveloxolone, capsule, at a dosage level determined by a Bayesian popPK analysis using the data from Cohorts A1, A2, A3, B1, B2, and C1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses. |
| Group 1: matched healthy subjects | EXPERIMENTAL | On the morning of Day 1, following an overnight fast of at least 10 hours, a single oral dose of 150 mg omaveloxolone (3 × 50 mg capsules) will be administered with 240 mL of water. No food will be allowed for 4 hours post dose. Pharmacokinetic samples will be obtained from pre dose until 336 hours post dose. |
| Group 2: subjects with mild hepatic impairment | EXPERIMENTAL | On the morning of Day 1, following an overnight fast of at least 10 hours, a single oral dose of 150 mg omaveloxolone (3 × 50 mg capsules) will be administered with 240 mL of water. No food will be allowed for 4 hours post dose. Pharmacokinetic samples will be obtained from pre dose until 336 hours post dose. |
| Group 3: subjects with moderate hepatic impairment | EXPERIMENTAL | On the morning of Day 1, following an overnight fast of at least 10 hours, a single oral dose of 150 mg omaveloxolone (3 × 50 mg capsules) will be administered with 240 mL of water. No food will be allowed for 4 hours post dose. Pharmacokinetic samples will be obtained from pre dose until 336 hours post dose. |
| Group 4: subjects with severe hepatic impairment | EXPERIMENTAL | On the morning of Day 1, following an overnight fast of at least 10 hours, a single oral dose of 150 mg omaveloxolone (3 × 50 mg capsules) will be administered with 240 mL of water. No food will be allowed for 4 hours post dose. Pharmacokinetic samples will be obtained from pre dose until 336 hours post dose. |
| Omaveloxolone 5 mg & ipilimumab | EXPERIMENTAL | Omaveloxolone (RTA 408) capsules, Dose1 taken orally once daily for 168 weeks, plus ipilimumab (3 mg/kg) administered at weeks 1, 4, 7, and 10. |
| Omaveloxolone 10 mg & ipilimumab | EXPERIMENTAL | Omaveloxolone (RTA 408) capsules, 10 mg taken orally once daily for 168 weeks, plus ipilimumab (3 mg/kg) administered at weeks 1, 4, 7, and 10. |
| Omaveloxolone 5 mg & nivolumab | EXPERIMENTAL | Omaveloxolone (RTA 408) capsules, 5 mg taken orally once daily for 168 weeks, plus nivolumab (240 mg) administered every two weeks as clinically indicated. |
| Omaveloxolone 10 mg & nivolumab | EXPERIMENTAL | Omaveloxolone (RTA 408) capsules, 10 mg taken orally once daily for 168 weeks, plus nivolumab (240 mg) administered every two weeks as clinically indicated. |
| Omaveloxolone 20 mg & nivolumab | EXPERIMENTAL | Omaveloxolone (RTA 408) capsules, 20 mg taken orally once daily for 168 weeks, plus nivolumab (240 mg) administered every two weeks as clinically indicated. |
| Omaveloxolone 100 mg & nivolumab | EXPERIMENTAL | Omaveloxolone (RTA 408) capsules, 100 mg taken orally once daily for 168 weeks, plus nivolumab (240 mg) administered every two weeks as clinically indicated. |
| Omaveloxolone 150 mg & nivolumab | EXPERIMENTAL | Omaveloxolone (RTA 408) capsules, 150 mg taken orally once daily for 168 weeks, plus nivolumab (240 mg) administered every two weeks as clinically indicated. |
| RTA 408 Capsules | EXPERIMENTAL | RTA 408 capsules, beginning dose 2.5 mg once daily, 28-day cycle, up to 12 cycles. Doses will increase by 100% of previous dose (e.g., 5 mg, 10 mg, 20 mg, etc.) until such time the Protocol Safety Review Committee decreases the escalation rate to 50% of the previous dose (e.g., 20 mg, 30 mg, 45 mg, etc). Dose escalation will continue until Maximum Tolerated Dose is identified. |
| Name | Type | Description |
|---|---|---|
| Omaveloxolone | DRUG | Administered as specified in the treatment arm. |
| Placebo | DRUG | Administered as specified in the treatment arm. |
| Omaveloxolone capsules, 2.5 mg | DRUG | - |
| omaveloxolone capsules, 5 mg | DRUG | - |
| omaveloxolone capsules, 10 mg | DRUG | - |
| Placebo capsules | DRUG | - |
| omaveloxolone capsules, 20 mg | DRUG | - |
| omaveloxolone capsules, 40 mg | DRUG | - |
| omaveloxolone capsules, 80 mg | DRUG | - |
| omaveloxolone capsules, 160 mg | DRUG | - |
| Omaveloxolone Capsules, 300 mg | DRUG | - |
| Omaveloxolone Capsules, 150 mg | DRUG | - |
| Omaveloxolone Lotion 0.5% | DRUG | Omaveloxolone lotion 0.5% will be applied topically twice-daily, for up to 13 weeks, during the course of radiation therapy |
| Omaveloxolone Lotion 3% | DRUG | Omaveloxolone lotion 3% will be applied topically twice-daily, for up to 13 weeks, during the course of radiation therapy |
| Vehicle Lotion | DRUG | Lotion vehicle manufactured to mimic RTA 408 lotion will be applied topically twice-daily, for up to 13 weeks, during the course of radiation therapy |
| 3D conformal radiation therapy | RADIATION | 45 50.4 Gy in 1.8 Gy per day, in addition to 10 - 16 Gy boost or 46 - 50 Gy in 2 Gy per day, in addition to 10 - 16 Gy boost, at the physician's discretion |
| Vehicle Ophthalmic Solution | DRUG | Opthalmic suspension manufactured to mimic RTA 408 suspension |
| Omaveloxolone Ophthalmic Suspension 0.5% | DRUG | 0.5% ophthalmic suspension of RTA 408 |
| Omaveloxolone Ophthalmic Suspension 1% | DRUG | 1% ophthalmic suspension of RTA 408 |
| Omaveloxolone Ophthalmic Suspension 1.0% | DRUG | - |
| Omaveloxolone Opthalmic Suspension 0.5% | DRUG | - |
| Omaveloxolone 50 mg capsules | DRUG | Capsules containing 50 mg of omaveloxolone |
| Omaveloxolone Capsules (2.5 mg/capsule) | DRUG | Capsules containing 2.5 mg of omaveloxolone per capsule to the dosage indicated in the Arm Label |
| Ipilimumab (3 mg/kg) | DRUG | Sterile solution containing ipilimumab to be delivered intravenously at 3mg/kg |
| Nivolumab (240 mg) | DRUG | Sterile solution containing nivolumab to be delivered intravenously at 240 mg |
| Omaveloxolone Capsules (10 mg/capsule) | DRUG | Capsules containing 10 mg of omaveloxolone per capsule to the dosage indicated in the Arm Label |
| Omaveloxolone Capsules (50 mg/capsule) | DRUG | Capsules containing 50 mg of omaveloxolone per capsule to the dosage indicated in the Arm Label |
Part 1: Key inclusion criteria: * Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, or GAA repeat expansion in 1 allele and with point mutations or deletions, or other non-GAA expansio...
Omaveloxolone is being studied for Friedreich ataxia, a genetic neurodegenerative disorder. It is also being evaluated in other conditions including breast cancer, corneal endothelial cell loss, hepatic impairment, mitochondrial myopathies, and inflammation and pain following ocular surgery. It is an investigational therapy and has not been approved for any indication.
Omaveloxolone targets NFE2L2, also known as Nrf2. It acts as an activator of this transcription factor, which regulates antioxidant response elements and helps cells defend against oxidative stress. This mechanism is relevant to diseases involving mitochondrial dysfunction and inflammation.
Omaveloxolone is being developed by Biogen Inc., which trades on the Nasdaq under the ticker BIIB. Biogen is the company responsible for advancing the drug through clinical trials across its various indications.
Omaveloxolone is in Phase 2 development overall. However, individual trials span different phases: a Phase 3 trial in Friedreich ataxia is active but not recruiting, and Phase 1 trials have been conducted in Friedreich ataxia, hepatic impairment, and melanoma.
Omaveloxolone has been studied in several clinical trials. Active trials include NCT06953583, a Phase 3 study in children and teens with Friedreich ataxia, and NCT06054893, a Phase 1 study in young Friedreich ataxia patients. Completed trials include NCT03902002 in hepatic impairment and NCT02259231 in melanoma.
Yes, omaveloxolone is also known as BIIB141. Other alternative names include RTA 408 and various formulation-specific names such as omaveloxolone capsules, omaveloxolone ophthalmic suspension, and omaveloxolone lotion. These refer to the same active ingredient developed by Biogen.