Efficacy AND Safety

MYCAPSSA Combines Proven Efficacy and an Established Safety Profile With a Twice-Daily, Oral Capsule

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Consistent, proven control of IGF-I levels, and a decrease in breakthrough symptoms1-3*

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Generally well tolerated for acromegaly treatment1,2

IGF-I, insulin-like growth factor 1; iSSA, injectable somatostatin analog.
*Data are representative of patients responding to both MYCAPSSA and iSSAs.
Photo of Jim, an actual MYCAPSSA patient Photo of Jim, an actual MYCAPSSA patient

Jim, an actual MYCAPSSA patient

The safety and efficacy of MYCAPSSA was studied in1-5:

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3 Clinical Trials

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329 People Living With Acromegaly

who previously responded to and tolerated injectable SSA treatment with octreotide or lanreotide.
Study name Treatments studied Number of participants Outcome
Phase 3 Open-Label Study MYCAPSSA N=155
  • Assessed efficacy and safety profiles of MYCAPSSA
OPTIMAL MYCAPSSA vs placebo N=56
  • Established efficacy and safety profiles of MYCAPSSA
  • Led to the FDA approval of MYCAPSSA
MPOWERED MYCAPSSA vs injectable SSA N=146
  • Demonstrated that MYCAPSSA maintained biochemical control
Study name
Phase 3 Open-Label Study
Treatments studied
MYCAPSSA
Number of participants
N=155
Outcome
  • Assessed efficacy and safety profiles of MYCAPSSA
Study name
OPTIMAL
Treatments studied
MYCAPSSA vs placebo
Number of participants
N=56
Outcome
  • Established efficacy and safety profiles of MYCAPSSA
  • Led to the FDA approval of MYCAPSSA
Study name
MPOWERED
Treatments studied
MYCAPSSA vs injectable SSA
Number of participants
N=146
Outcome
  • Demonstrated that MYCAPSSA maintained biochemical control

FDA, US Food and Drug Administration; SSA, somatostatin analog; ULN, upper limit of normal.


Twenty-eight patients in OPTIMAL received placebo.

Primary endpoint of noninferiority assessment of the proportion of patients maintaining biochemical response (IGF-I <1.3 x ULN using time-weighted average) throughout the randomized controlled treatment phase.

EFFICACY

OPTIMAL Trial: Study Design2

The OPTIMAL phase 3 trial was a 9-month, randomized, double-blind, placebo-controlled study in 56 patients with acromegaly and IGF-I ≤1 × ULN at screening.

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Starting dose: 40 mg (20 mg AM + 20 mg PM)

Dose titration was performed during the first 6 months of the study to a dose of 60 mg (40 mg AM + 20 mg PM) and up to a maximum dose of 80 mg daily (40 mg AM + 40 mg PM) based on biochemical results or symptoms.

Patients then maintained a fixed dose until end of treatment.
Primary Endpoint
  • Proportion of patients who maintained biochemical response

Response was defined as IGF-I ≤1.0 × ULN based on average of week 34 and 36 measurements.

Secondary Endpoints
  • Proportion of patients who maintained GH response at week 36 (average GH <2.5 ng/mL)
  • Time to loss of IGF-I response defined as either IGF-I >1.0 or ≥1.3 × ULN for 2 consecutive visits
  • Proportion of patients who reverted to prior SSA up to and including week 36 measurements
GH, growth hormone.

In the OPTIMAL Trial,
Majority of MYCAPSSA-Treated Patients Maintained IGF-I Control2

58% of patients on MYCAPSSA maintained IGF-I response§ vs 19% of patients on placebo.

The mean IGF-I level was maintained within normal range in the group receiving MYCAPSSA at the end of oral treatment.

Bar chart depicting mean levels of IGF-I at baseline vs end of treatment with MYCAPSSA or placebo. At baseline, the mean IGF-I level was 0.84 in the placebo group vs 0.80 in the MYCAPSSA group. At the end of treatment at 9 months, IGF-I levels were 1.69 in the placebo group vs 0.97 in the MYCAPSSA group.Bar chart depicting mean levels of IGF-I at baseline vs end of treatment with MYCAPSSA or placebo. At baseline, the mean IGF-I level was 0.84 in the placebo group vs 0.80 in the MYCAPSSA group. At the end of treatment at 9 months, IGF-I levels were 1.69 in the placebo group vs 0.97 in the MYCAPSSA group.
§IGF-I response was defined as an average of weeks 34 and 36 IGF-I ≤1.0 × ULN (P =.008).
MPOWERED Trial: Study Design3
15-month, open-label study with a 9-month randomized, active-controlled phase


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Primary Endpoint
  • Noninferiority assessment of the proportion of patients maintaining biochemical response (IGF-I <1.3 x ULN using time-weighted average) throughout the randomized controlled treatment (RCT) phase

Selected Secondary Endpoints
  • Proportion of patients who maintain or reduce the overall number of active acromegaly symptoms
  • Incidence and severity of adverse events
  • Change in IGF-I and GH levels during RCT phase
  • Number of acromegaly symptoms per patient and proportion of patients with each symptom
||Dose escalations to 60 mg or 80 mg until adequate response achieved.

In the MPOWERED Trial, MYCAPSSA Demonstrated Consistent Control

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IGF-I Levels

Biochemical control with MYCAPSSA was consistent with results observed in both open-label and OPTIMAL studies.2,3,5#
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Breakthrough Symptoms

MYCAPSSA-treated patients experienced a decrease in breakthrough symptoms in the run-in phase.
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Acromegaly Symptoms

In 2 separate studies, MYCAPSSA-treated patients demonstrated consistent control of several common symptoms of acromegaly.3,5
Data are representative of patients responding to both iSSAs and MYCAPSSA.

#Response rates at the completion of the studies in those patients responding after the dose titration phase was 92% of patients in the pivotal phase 3 trial and 85% of patients in the open-label study.2,5

THE PRIMARY ENDPOINT OF NONINFERIORITY TO iSSA WAS MET3

Patients in both groups maintained biochemical control3**
91% of MYCAPSSA-treated patients maintained biochemical control†† vs 100% of those treated with iSSAs.

Bar graph depicting the proportion of patients that were biochemical responders. 100% of patinets responded to the iSSA (n=37/37; 95% CI, 34-37). 91% of patients responded to MYCAPSSA (n=50/53; 95% CI, 44-53)Bar graph depicting the proportion of patients that were biochemical responders. 100% of patinets responded to the iSSA (n=37/37; 95% CI, 34-37). 91% of patients responded to MYCAPSSA (n=50/53; 95% CI, 44-53)

95% confidence intervals (CI) represent number of patients.
**Data are representative of patients responding to both iSSAs and MYCAPSSA.
††Biochemical control was defined as IGF-I <1.3 × ULN using time-weighted average.
MYCAPSSA-treated patients experienced a decrease in breakthrough symptoms in the run-in phase3
Bar graph depicting the proportion of patients that reported breakthrough symptoms. 25% of patients reported breakthrough symptoms at baseline of run-in, and 7% of patients reported symptoms at the end of run-in.Bar graph depicting the proportion of patients that reported breakthrough symptoms. 25% of patients reported breakthrough symptoms at baseline of run-in, and 7% of patients reported symptoms at the end of run-in.
Acromegaly Symptoms

Across multiple studies, MYCAPSSA-treated patients demonstrated consistent symptom control3,5

Open-Label Trial‡‡
Bar
MPOWERED Trial§§
Bar
‡‡Proportion of patients with active individual symptoms from baseline to end of treatment. Fixed-dose population (n = 110). For patients not completing the core treatment period, the last assessment during the core treatment period was used. For patients not completing the extension treatment period, the last assessment during the extension treatment period was used.
§§Proportion of patients with active individual symptoms at indicated timepoint during run-in phase.
SAFETY
MYCAPSSA Safety Profile Compared to Placebo
Treatment-related TEAEs occurring ≥5% in either treatment group during the randomized phase by system organ class and preferred term##
Adverse Event Data in Patients Who Responded to MYCAPSSA & to iSSAs
MYCAPSSA Safety Profile
Compared to Placebo1
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Adverse Event Data in Patients Who
Responded to MYCAPSSA and to iSSAs3¶¶

Treatment-related TEAEs occurring ≥5% in either treatment group during the randomized phase by system organ class and preferred term##

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‖‖Includes blood glucose increased, hyperglycemia, and glycosylated hemoglobin increased.
¶¶Data are representative of patients responding to both iSSAs and MYCAPSSA.
##An adverse event was considered related if the relationship to study drug was reported as possibly related or related.

Talk to Patients About GI-Related Side Effects

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GI side effects were mostly transient and resolved in less than 2 weeks

GI AEs were reported in up to 68% of MYCAPSSA patients.1

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GI AEs were mostly mild to moderate for acromegaly treatment

AEs occurred mostly during the initial 3 months of treatment and resolved on treatment within a median duration of less than 2 weeks after onset.1

AEs, adverse events; GI, gastrointestinal; TEAEs, treatment-emergent adverse events.
REFERENCES: 1. MYCAPSSA [package insert]. Amryt Pharmaceuticals; 2022. 2. Samson SL, et al. J Clin Endocrinol Metab. 2020;105(10):1-13. 3. Fleseriu M, et al. Lancet Diabetes Endocrinol. 2022;10(2):102-111. 4. Efficacy and safety of octreotide (MYCAPSSA [formerly Octreolin]) for acromegaly. ClinicalTrials.gov. Updated August 17, 2017. Accessed November 21, 2023. https://classic.clinicaltrials.gov/ct2/show/study/NCT01412424 5. Melmed S, et al. J Clin Endocrinol Metab. 2015;100(4):1699-1708.

Important Safety Information

CONTRAINDICATIONS

Hypersensitivity to octreotide or any of the components of MYCAPSSA. Anaphylactoid reactions, including anaphylactic shock, have been reported in patients receiving octreotide.

WARNINGS AND PRECAUTIONS

MYCAPSSA can cause problems with the gallbladder. Monitor patients periodically. Discontinue if complications of cholelithiasis are suspected.

Blood sugar, thyroid levels, and vitamin B12 levels should be monitored and treated accordingly.

Bradycardia, arrhythmia, or conduction abnormalities may occur. Treatment with drugs that have bradycardia effects may need to be adjusted.

ADVERSE REACTIONS

The most common adverse reactions (incidence >10%) are nausea, diarrhea, headache, arthralgia, asthenia, hyperhidrosis, peripheral swelling, blood glucose increased, vomiting, abdominal discomfort, dyspepsia, sinusitis, and osteoarthritis.

DRUG INTERACTIONS

The following drugs require monitoring and possible dose adjustment when used with MYCAPSSA: cyclosporine, insulin, antidiabetic drugs, calcium channel blockers, beta blockers, lisinopril, digoxin, bromocriptine, and drugs mainly metabolized by CYP3A4. Counsel women taking an oral contraceptive to use an alternative non-hormonal method of contraception or a back-up method when taking MYCAPSSA.

Patients taking proton pump inhibitors, H2-receptor antagonists, or antacids concomitantly with MYCAPSSA may require increased dosages of MYCAPSSA.

PREGNANCY

Advise premenopausal females of the potential for an unintended pregnancy.

Please contact Chiesi Farmaceutici S.p.A. at 1-888-661-9260 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information, including Medication Guide.


Indication

INDICATION AND USAGE

MYCAPSSA (octreotide) delayed-release capsules, for oral use, is a somatostatin analog indicated for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.

Important Safety Information

CONTRAINDICATIONS

Hypersensitivity to octreotide or any of the components of MYCAPSSA. Anaphylactoid reactions, including anaphylactic shock, have been reported in patients receiving octreotide.

WARNINGS AND PRECAUTIONS

MYCAPSSA can cause problems with the gallbladder. Monitor patients periodically. Discontinue if complications of cholelithiasis are suspected.

Blood sugar, thyroid levels, and vitamin B12 levels should be monitored and treated accordingly.

Bradycardia, arrhythmia, or conduction abnormalities may occur. Treatment with drugs that have bradycardia effects may need to be adjusted.

ADVERSE REACTIONS

The most common adverse reactions (incidence >10%) are nausea, diarrhea, headache, arthralgia, asthenia, hyperhidrosis, peripheral swelling, blood glucose increased, vomiting, abdominal discomfort, dyspepsia, sinusitis, and osteoarthritis.

DRUG INTERACTIONS

The following drugs require monitoring and possible dose adjustment when used with MYCAPSSA: cyclosporine, insulin, antidiabetic drugs, calcium channel blockers, beta blockers, lisinopril, digoxin, bromocriptine, and drugs mainly metabolized by CYP3A4. Counsel women taking an oral contraceptive to use an alternative non-hormonal method of contraception or a back-up method when taking MYCAPSSA.

Patients taking proton pump inhibitors, H2-receptor antagonists, or antacids concomitantly with MYCAPSSA may require increased dosages of MYCAPSSA.

PREGNANCY

Advise premenopausal females of the potential for an unintended pregnancy.

Please contact Chiesi Farmaceutici S.p.A. at 1-888-661-9260 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information, including Medication Guide.


Indication

INDICATION AND USAGE

MYCAPSSA (octreotide) delayed-release capsules, for oral use, is a somatostatin analog indicated for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.