For Patients and Caregivers

The most inclusive clinical trial program in SMA2

The most inclusive clinical trial program in
SMA2

Participants reflective of real-world SMA population1,28,30*

Over 490 SMA patients ranging from newborns to 60 years old

490+ individuals with presymptomatic, infantile-onset, and later-onset SMA†

With different levels of disease severity and functional ability

Both ambulatory and nonambulatory‡

With and without scoliosis (mild to severe)

With and without prior disease-modifying treatment§ (evaluated for safety)

Sunfish, Firefish, Rainbowfish and Jewelfish trial icons

EFFICACY DATA in patients aged 16 days to 25 years

SAFETY DATA in patients aged 16 days to 60 years

 

The first trial program of an FDA-approved SMA treatment to include patients older than 18 years, some of whom have severe scoliosis2
The first trial program of an FDA-approved SMA treatment to include patients older than 18 years, some of whom have severe scoliosis2

*Enrollment across 4 clinical trials.

†Presymptomatic SMA defined as genetically diagnosed SMA in infants aged <6 weeks at time of first Evrysdi dose who had not yet presented with symptoms; infantile-onset SMA defined as Type 1 SMA; later-onset SMA defined as Type 2 or 3 SMA.

‡Seven ambulatory patients included in SUNFISH Part 1; no ambulatory patients included in Part 2. There are 16 patients included in JEWELFISH who are ambulatory.

§Approved and investigational therapies including an SMN2-splicing modifier or gene-replacement therapy.

The Evrysdi clinical program consists of 4 trials1,30

Four trials, Sunfish, Firefish, Rainbowfish and Jewelfish
See the results

SUNFISH is a 2-part, placebo-controlled trial in later-onset (Type 2 or 3) spinal muscular atrophy (SMA): the first placebo-controlled trial in adults and children older than 9 years with SMA (ages ranged from 2 to 25 years)1,2

Sunfish study design
SUNFISH was purposely designed to include adults and children with complications, such as contractures and severe scoliosis1,2
  • Part 1 (N=51) was the exploratory, dose-finding portion
  • In Part 2:
    • Adults and children were randomized 2:1 to receive either the recommended dose of Evrysdi or placebo. After 12 months, adults and children receiving placebo were switched to Evrysdi
    • The primary endpoint was mean change from baseline in MFM-32 at 12 months

MFM-32=Motor Function Measure–32 Items.

Participants included a broad range of individuals designed to reflect the real-world SMA population1,2*

SUNFISH PART 2
  Evrysdi
(n=120)		 Placebo
(n=60)
Patient characteristics
SMA Type, % (n)
Type 2
Type 3
Median weight at baseline, kg (range)
70.0% (84)
30.0% (36)
27% (10-100)
73.3% (44)
26.7% (16)
27% (11-112)
Disease severity
Mean age at onset, months (SD) 14.1 (8.4) 18.5 (21.1)
Scoliosis, % (n)
Yes
>40° curvature
63.3% (76)
28.3% (34)
73.3% (44)
38.3% (23)
Surgery for scoliosis before screening, % (n)
Yes
No
24.2% (29)
52.5% (63)
28.3% (17)
55.0% (33)
Motor function assessment scores at baseline
MFM-32 total score, median (min-max)
n		 46.9 (16.7-71.9)
115		 47.9 (17.7-71.9)
59
RULM total score, median (min-max)
n		 19.0 (3.0-36.0)
119		 20.0 (9.0-38.0)
58
HFMSE total score, median (min-max)
n		 14.0 (0.0-48.0)
120		 13.0 (2.0-43.0)
60

*The overall baseline demographic characteristics were well balanced between Evrysdi and placebo groups, with the exception of an imbalance of patients with scoliosis (63% in the Evrysdi arm and 73% in the placebo arm).

HFMSE=Hammersmith Functional Motor Scale Expanded; MFM-32=Motor Function Measure-32 Items; RULM=Revised Upper Limb Module.

See the results

FIREFISH is a 2-part, open-label trial in infantile-onset (Type 1) spinal muscular atrophy (SMA) establishing the safety and efficacy of Evrysdi in infants 2 months and older1,14

Firefish study design
  • Part 1 determined the recommended dose
  • In Part 2, the primary endpoint was ability to sit without support for ≥5 seconds, as measured by Item 22 of the BSID-III gross motor scale
  • In Parts 1 and 2 (pooled analysis), key efficacy endpoints were:
    • The ability to sit without support for ≥5 seconds as measured by Item 22 of the BSID-III gross motor scale (recommended-dose cohort; n=58)
    • Survival without permanent ventilation (all-patients cohort; N=62)

*The first 4 enrollees received a lower dose (titration to target dose of 0.08 mg/kg/day) for 12 months; all other enrollees (n=17) had their dose adjusted to the recommended dose (0.2 mg/kg/day) before 12 months. Efficacy and safety were assessed at 12 months, after which all participants received the recommended dose (0.2 mg/kg/day).
BSID-III=Bayley Scales of Infant and Toddler Development–Third Edition.

Infants reflected those seen in a real-world setting, including age, disease progression, baseline motor function and levels of disease severity.1,2,14

FIREFISH PARTS 1 and 2
Baseline demographics
Recommended-dose cohort (n=58)
Patient characteristics2,27  
Median age at onset, months (range) 1.5 (0.9-3.0)
Median age at enrollment, months (range) 5.5 (2.2-6.9)
Median weight, kg (range) 6.6 (4.1-10.6)
Motor function assessment scores27  
CHOP INTEND, median (range) 23.0 (8.0-37.0)
HINE-2, median (range) 1.0 (0.0-5.0)

CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders; HINE-2=Hammersmith Infant Neurological Examination Module 2.

See the results

RAINBOWFISH is an ongoing, open-label, single-arm trial of Evrysdi in 26 infants who were aged 16 to 40 days at first Evrysdi dose. These infants had a genetic diagnosis of SMA, but had not yet presented with symptoms (presymptomatic SMA)1,28

Rainbowfish study design
  • Infants were <6 weeks of age at time of first Evrysdi dose, had a genetic diagnosis of SMA, but had not yet presented with symptoms
  • The primary endpoint† is the ability to sit without support for ≥5 seconds at 12 months, as measured by Item 22 of the BSID-III gross motor scale
  • Efficacy is based on an interim analysis of HINE-2 at month 12 (n=6)

*Final patient number enrolled as of February 22, 2022.

†The primary efficacy population includes infants with 2 copies of the SMN2 gene and CMAP amplitude ≥1.5 mV at baseline (n≥5). At the time of the interim analysis there were not enough participants to evaluate the primary endpoint.
BSID-III=Bayley Scales of Infant and Toddler Development–Third Edition; CMAP=compound muscle action potential; HINE-2=Hammersmith Infant Neurological Examination-Module 2; mV=millivolt.

RAINBOWFISH demographics

RAINBOWFISH
  Infants enrolled2,28
(n=18)		 Infants who received Evrysdi for ≥12 months1,2
(n=7)*
Patient characteristics
Age at first dose, days, median (range) 26.5 (16-40) 35 (16-40)
SMN2 copy number, % (n)
  2
>2
39% (7)
61% (11)†
57% (4)
43% (3)‡
Gender, % (n)
Female
Male
56% (10)
44% (8)
71% (5)
29% (2)
Weight at first dose, grams, median (range) 3984
(3076-5726)		 4030
(3210-5726)
Baseline CMAP amplitude,§ mV, median (range)
Baseline value <1.5 mV, % (n)
Baseline value ≥1.5 mV, % (n)		 3.6 (0.5-6.7)
17% (3)
83% (15)		 3.0 (0.5-6.6)
29% (2)
71% (5)

As of clinical cut-off date: July 1, 2021.

*Infants who had 2 or 3 SMN2 copies were included in the interim efficacy analysis (n=6). The 7th infant who had >3 SMN2 copies was excluded from the interim analysis.

†Includes 7 infants with 3 SMN2 copies and 4 infants with ≥4 SMN2 copies.

‡Includes 2 infants with 3 SMN2 copies and 1 infant with ≥4 SMN2 copies.

§The primary efficacy population includes infants with 2 SMN2 copies and CMAP amplitude value ≥1.5 mV at baseline.
CMAP=compound muscle action potential; mV=millivolt.

Explore safety results

JEWELFISH is an open-label safety trial in adults, children, and infants aged 1 to 60 years with Type 1, 2, or 3 spinal muscular atrophy (SMA) taking Evrysdi who have received previous treatment with approved or investigational therapies30

Jewelfish study design
  • This trial is investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of Evrysdi in both ambulatory and nonambulatory patients
    • Ambulatory and nonambulatory patients: 9% (n=16) walkers, 57% (n=99) sitters, and 34% (n=59) nonsitters
  • Participants have received previous treatment with approved or investigational therapies* including an SMN2-splicing modifier (≥90 days prior to screening) or gene-replacement therapy (≥12 months prior to screening)

*Investigational at the start of JEWELFISH. All but 3 patients enrolled in JEWELFISH received previous treatment; these 3 patients were previously enrolled in the MOONFISH trial, received placebo, and were never switched to RG7800.30

A broad and heterogeneous population with a high degree of motor impairment at baseline30

PREVIOUS TREATMENT
    RG7800* (MOONFISH)
n=13†		 Nusinersen
n=76‡		 Olesoxime*
n=71		 Onasemnogene abeparvovec
n=14§		 All patients
N=174
Patient characteristics
Age at enrollment, years Median (range) ≥18 years, % (n) 30.0 (16-58)
85% (11)		 12.0 (1-60)
28% (21)		 16.0 (11-36)
44% (31)		 2.0 (1-5)
0%		 14.0 (1-60)
36% (63)
Gender, % (n) Male 69% (9) 53% (40) 49% (35) 79% (11) 55% (95)
SMA type, % (n) 1
2
3		 0%
39% (5)
62% (8)		 12% (9)
57% (43)
32% (24)		 3% (2)
70% (50)
27% (19)		 29% (4)
71% (10)
0%		 9% (15)
62% (108)
29% (51)
SMN2 copy number, % (n) 1
2
3
4
 0%
8% (1)
46% (6)
46% (6)
 0%
13% (10)
72% (55)
15% (11)
 0%
1% (1)
92% (65)
7% (5)
 21% (3)
7% (1)
71% (10)
0%
 2% (3)
8% (13)
78% (136)
13% (22)
Disease severity
Scoliosis, % (n) Yes
>40° curvature		 69% (9)
23% (3)		 84% (61/73)||
37% (27/73)||		 93% (66)
51% (36)		 27% (3/11)||
0%		 83% (139/168)||
39% (66/168)||
Hip subluxation or dislocation, % (n) Yes 15% (2) 34% (25/73)|| 28% (20) 36% (4/11)|| 30% (51/168)||
Motor function assessment scores at baseline
Motor function at baseline, % (n) Nonsitters
Sitters
Walkers		 54% (7)
23% (3)
23% (3)		 28% (21)
55% (42)
17% (13)		 41% (29)
59% (42)
0%		 14% (2)
86% (12)
0%		 34% (59)
57% (99)
9% (16)
Baseline HFMSE total score <10, % (n) Yes 62% (8) 48% (35/73)|| 83% (59) 27% (3/11)|| 63% (105/168)||

*Investigational therapies; not approved by FDA for any use.

†In the MOONFISH trial, 3 patients were treated with placebo and were not switched to RG7800.30

‡Three patients had also received olesoxime previously.

§One patient received treatment with onasemnogene abeparvovec first, followed by nusinersen. Ten patients were enrolled in STRONG, three patients in STR1VE, and one patient in STR1VE EU prior to enrollment in JEWELFISH.

||Only reported for patients aged 2 to 60 years.

For patients younger than 2 years, baseline motor milestones were evaluated by the Hammersmith Infant Neurological Examination–Module 2.
FDA=Food and Drug Administration; HFMSE=Hammersmith Functional Motor Scale Expanded; SMN=survival motor neuron.

Important Safety Information and Indication

Indication

Evrysdi is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

 

Interactions with Substrates of MATE Transporters

  • Based on in vitro data, Evrysdi may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K, such as metformin
  • Avoid coadministration of Evrysdi with MATE (multidrug and toxin extrusion) substrates. If coadministration cannot be avoided, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug if needed

Pregnancy & Breastfeeding

  • Evrysdi may cause embryofetal harm when administered to a pregnant woman. In animal studies, administration of Evrysdi during pregnancy and/or lactation resulted in adverse effects on development. Advise pregnant women of the potential risk to the fetus
  • Pregnancy testing is recommended prior to initiating Evrysdi. Advise female patients to use contraception during treatment with Evrysdi and for at least 1 month after the last dose
  • There is a pregnancy exposure registry that monitors pregnancy and fetal/neonatal/infant outcomes in women exposed to Evrysdi during pregnancy. Physicians are encouraged to register patients and pregnant women are encouraged to register themselves by calling 1-833-760-1098 or visiting www.evrysdipregnancyregistry.com
  • The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Evrysdi and any potential adverse effects on the breastfed infant

Potential Effects on Male Fertility

  • Counsel male patients that fertility may be compromised by treatment with Evrysdi. Male patients may consider sperm preservation prior to treatment

Most Common Adverse Reactions

  • The most common adverse reactions in later-onset SMA (incidence in at least 10% of patients treated with Evrysdi and more frequent than control) were fever, diarrhea, and rash
  • The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, adverse reactions with an incidence of at least 10% were upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough
  • The safety profile for presymptomatic patients is consistent with the safety profile for symptomatic SMA patients treated with Evrysdi in clinical trials

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

Please see full Prescribing Information for additional Important Safety Information.

 

 

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      Kletzl H, Cleary Y, Grimsey P, Gerber M, Scalco RS. Risdiplam: pharmacokinetic, pharmacodynamic, safety and efficacy exposure response analyses. Poster presented at: Cure SMA 2022 Research and Clinical Care Meeting; June 15-17, 2022; Anaheim, CA.

    • Iannaccone ST, To TM, Dickendesher T, Shapouri S, Pineda ED. A retrospective analysis of adherence and persistence among risdiplam-treated patients with spinal muscular atrophy (SMA). Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 19-22, 2023; Dallas, TX.

      Iannaccone ST, To TM, Dickendesher T, Shapouri S, Pineda ED. A retrospective analysis of adherence and persistence among risdiplam-treated patients with spinal muscular atrophy (SMA). Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 19-22, 2023; Dallas, TX.