Efficacy/Studies
Scores >2 if ALSFRS-R, FVC >80%, Disease Duration <2 yrs & Age 20-75
What is Study 19 entry criteria?
Page 9
IV & ORS Indication
What is Radicava's indication: for the treatment of amyotrophic lateral sclerosis
Page 1
Contusion 15%, Gait Disturbance 13%, Headache 10% and Fatigue 7.6%
What are Radicava ORS adverse events?
Page 18 & 20
Radicava ORS Initial Treatment Dose
What is 5ml (105mg) orally or feeding tube 14 days, followed by 14 drug free days.
Page 22
79% or 4 in 5 patients
% of patients continued ORS between Jun 22 - Feb 23
pg 3 & 28
33% & 2.49
What is Study 19 less change in ALSRS-R scores from baseline vs placebo @ 24 weeks?
What is on average number fewer points lost on the ALSFRS-R for patients on Radicava vs placebo?
Page 10
11,200 & 1.4 million days & 5,300+
What is # of Radicava pts treated and total days of treatment, since approval and patients prescribed ORS
Page 3 & 28
7.6%
What is Radicava ORS percentage fatigue adverse event?
Page 20
Radicava ORS Subsequent Treatment
What is 10 out of 14 days of treatment followed by 14 drug free days?
Page 22
2,000+ and 1,400+
# of hcp's that have prescribed Radicava and 1400 were general neurologists
Page 3
91.3%
What is the percentage of patients on Radicava in Study 19 that were also on riluzole?
Page 11
Conducted (evaluated) across 7...
What is number of Radicava clinical trials?
Page 29
2.9% and 5.9%
What are discontinue rates for Radicava IV & Radicava ORS?
Page 19 & 20
High Fat, Low Fat, Caloric supplement Meal Fasting Hours Guidance with ORS
What is 8, 4 & 2 hrs prior respectively, no food except water for one hour post dose?
Page 23
80 trials & 1 treatment
1971-2013 # of ALS trials & 1 treatment approved (riluzole)
Page 28
.0013
What is the P Value in Study 19?
Page 10 & 11
Significance of the positive results from a phase 3 trial/1995
What is "Since 1995 , Radicava is the only FDA-approved treatment for ALS with positive results from a phase 3 trial despite more than 125 clinical trials from 2008-2019."
Page 29
1.1%
What is the discontinuation rate due to GI in Radicava ORS?
Page 20
Sorbitol
What is added to Radicava ORS that gives sweet taste?
Page 21 & 22
Over a decade
How long MTPA R&D
Pag 28
41.9/41.8; 37.5/34.9
What are Radicava/Placebo ALSFR-S at baseline & at 24 weeks?
Page 11
Alternate route of administration phase 1 studies
What are two phase 1 studies in Radicava ORS one in ALS patients with PEG tubes and one in healthy subjects with NG tubes?
Page 29
Only 4 patients out of 185
What is the number of patients that discontinued (A/E) in the first 2 months on Radicava ORS? (4/185)
Page 20
Xanthan Gum
Food additive used as a thickening agent in Radicava ORS?
No Page
Room temperature
Temp ORS stored
Page 3
39.1% (3x); lost 2 pts or less
What is the percentage of Radicava patients that declined 2 pts or less. (distribution of change) vs placebo 13.2%
Page 12
20%-25% or greater change
What is NEALS Consortium conclusion that a treatment is clinically meaningful when it results in 20% to 25% or greater change in the slope of ALSFRS-R reflecting the slowing of functional decline?
Page 5 & 10
# of Radicava known drug interactions
What is NONE!
Page 21
Radicava IV & Radicava ORS Dose Adjustments
What is NONE? Including Hepatic & Renal
Page 23 & Page 16
-9.26 (3.95)
Treatment difference in total score using ALSAQ-40, which is patient self reported
Page 15
185 patients
What is the safety 6 month, phase 3, open-label clinical trial?
Page 2 & 20
Results of equivalent Plasma Levels
What are the results from a phase 1 pharmacokinetic study in healthy subjects showing equivalent plasma levels with Radicava ORS and Radicava IV?
Page 6
Radicava IV & ORS contraindication
What is hypersensitivity to Edaravone or it's active ingredients.
Page 30 & 21 most complete, but literally every other page
Neutral to slightly sweet
What is patients description of Radicava ORS taste?
Page 21 & 22
Specific Populations
Pharmokenitics, Pregnancy, Lactation, Pediatric, Renal Impairment, Hepatic Impairment
Page 16
# of Countries with clinical trials for Radicava?
What is 6? US, Canada, France, Germany, Italy and Japan
No page, was in old MVA
1.1 mg
What is the amount of Sodium in Radicava?
Page 21
Sulfite Allergic Reaction
What is Radicava IV & ORS important safety information? Contains sodium bisulfite, a sulfite that may cause allergic-type reactions.
Page 37 most complete, but literally every other page
Must do for 30 seconds
What is how long to shake ORS?
Page 3
More than 15 ongoing or completed
What are 15 ongoing or already completed clinical studies.
Page 28
4 Domains of ALSFRS-R
Bulbar, Fine Motor, Gross Motor & Respiratory
Page 4 & 14
45% & >50 miles
What is the percentage of people with ALS living greater than 50 miles from an ALS Clinic?
No page was in our old MVA
Radicava MOA
What is Unknown? It is believed, based on preclinical studies, to reduce oxidative stress
Page 29
15 days & 30 days
What are the # of days after opening bottle or # of days from date of shipment indicated on the carton pharmacy label to discard ORS?
Page 3
% patients that discontinued due to GI
1.1% d/c ue to GI, .5% diarrhea & .5% dysphagia
Page 20
Points Lost per month based of what database
what is 1 and PRO-ACT
Page 5 & 4
Service-connected Disease
What is ALS with veterans?
Page 27
Patients twice as likely to develop ALS
What are military/VA patients?
Page 27
NG or Peg tube
flexibile routes of administration
Page 3
HUB Name
JourneyMate
Page 26