Mycapssa (octreotide) / Chiesi - LARVOL DELTA

SWOG-S0518: Octreotide Acetate and Recombinant Interferon Alfa-2b or Bevacizumab in Treating Patients With Metastatic or Locally Advanced, High-Risk Neuroendocrine Tumor (clinicaltrials.gov) - P3 | N=427 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Jan 2025 ➔ Jan 2026

Trial completion date • Endocrine Cancer • Gastric Cancer • Neuroendocrine Tumor • Oncology • Solid Tumor • Thyroid Gland Carcinoma • Thyroid Gland Medullary Carcinoma

Oral Absorption of Middle-to-Large Molecules and Its Improvement, with a Focus on New Modality Drugs. (PubMed, Pharmaceutics) - "Although these two medications represent major achievements in the development of orally absorbable peptide formulations, the oral bioavailability of peptides after taking Rybelsus and Mycapssa is still only around 1%. In this article, we review the approaches and recent advances of orally bioavailable middle-to-large molecules and discuss challenges for improving their oral absorption."

Journal • Review • Acromegaly • Diabetes • Endocrine Disorders • Metabolic Disorders • Type 2 Diabetes Mellitus

Acromegaly: Pathophysiological Considerations and Treatment Options Including the Evolving Role of Oral Somatostatin Analogs. (PubMed, Pathophysiology) - "While clinical trial data supports the use of octreotide capsules (Mycapssa) in the pharmacological management of GH and IGF-1 levels, very little data exist regarding the drug's efficacy, tolerability, and use in female or pediatric-specific populations. A better understanding of the efficacy, application, and role of oral octreotide capsules in the long-term medical management of acromegaly in a diversity of populations is imperative to best determine the risks/benefits for the clinician."

Journal • Review • Acromegaly • Endocrine Disorders • Gastrointestinal Disorder • Pediatrics • IGF1 • SSTR

Gastrointestinal Permeation Enhancers for the Development of Oral Peptide Pharmaceuticals. (PubMed, Pharmaceuticals (Basel)) - "Sodium salcaprozate (SNAC) and sodium caprylate (C8) have been used as gastrointestinal permeation enhancers for semaglutide and octreotide, respectively. Herein, we briefly review two approved products, Rybelsus and Mycapssa, and discuss the permeation properties of SNAC and medium chain fatty acids, sodium caprate (C10) and C8, focusing on Eligen technology using SNAC, TPE technology using C8, and gastrointestinal permeation enhancement technology (GIPET) using C10."

Journal • Review • Acromegaly • Diabetes • Endocrine Disorders • Gastrointestinal Disorder • Metabolic Disorders • Type 2 Diabetes Mellitus

COMP Adopts Positive Opinion on Orphan Designation for Mycapssa for the Treatment of Carcinoid Syndrome Associated with Neuroendocrine Tumors (GlobeNewswire) - "Amryt...announces that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion for orphan designation for the use of Mycapssa^® in the treatment of carcinoid syndrome associated with neuroendocrine tumors (NET)....Pharmacokinetic studies with Mycapssa^® showed dose linearity from 20mg to 80mg (40mg/day to 160mg/day), hence support dosing requirements for the planned Phase 3 study in patients with carcinoid syndrome, planned to be initiated early in 2023."

European regulatory • New P3 trial • Carcinoid Tumor • Neuroendocrine Tumor • Oncology • Solid Tumor

Amryt Announces New Patent for Mycapssa (GlobeNewswire) - "Amryt...announces an update regarding the patents for its product Mycapssa^® (octreotide)....The USPTO has issued to Amryt US Patent No. 11,510,963, with claims related to the Mycapssa^® product. This patent will expire in February 2036. With the addition of this patent, Amryt has ten Orange Book-listed patents for Mycapssa^® with patent protection through December 2040."

Patent • Oncology

Amryt Reports Q3 2022 Financial and Operational Results (GlobeNewswire) - "Amryt...provides a business update and announces unaudited financial results for the third quarter ended September30, 2022....292.8% increase in Mycapssa^® revenues YoY to $5.7Min Q3 2022(Q3 2021: $1.5M) and 26.9% growth versus Q2 2022."

Sales • Carcinoid Tumor • Neuroendocrine Tumor • Oncology • Solid Tumor

"$AMYT Amryt Announces New Patent for Mycapssa® https://t.co/dgtgNdYU24 #patent" (@stock_titan)

Amryt Reports Record Q2 2022 Results (GlobeNewswire) - "Mycapssa: Mycapssa new patent announced, further strengthening our IP portfolio with protection to 2040; Received Orphan Drug Designation from the FDA for Mycapssa for the treatment of carcinoid syndrome associated with neuroendocrine tumors (NET); Pathway agreed with the FDA to initiate a Phase 3 study for NET - expected in Q1 2023....Mycapssa delivered $4.5M in Q2 2022 as compared to $3.4M in Q1 2022"

New P3 trial • Orphan drug • Patent • Neuroendocrine Tumor • Oncology

(PT10) Consistent Control for Patients with Acromegaly (ENDO 2022) - "This Product Theater is presented by: AMRYT PHARMA Session Description: - Describe the clinical characteristics and associated serious comorbidities of acromegaly - Utilizing an acromegaly case study, demonstrate how the road to diagnosis could be more accurate and efficient - Describe the risk to benefit profile of utilizing MYCAPSSA® (octreotide) for patients with acromegaly"

Clinical • Acromegaly • Endocrine Disorders

Amryt Announces Successful Bioavailability Study for Mycapssa (octreotide capsules) (GlobeNewswire) - "Amryt...announces completion of a successful pharmacokinetic (PK) study for Mycapssa® (oral octreotide). The data supports a planned Phase 3 study of Mycapssa® in the treatment of patients with carcinoid symptoms due to Neuroendocrine Tumors (NET)...Amryt is currently finalising the study protocol with the FDA and expects to initiate the Phase 3 study in Q4 2022."

Clinical data • Clinical protocol • New P3 trial • Neuroendocrine Tumor • Oncology

"$AMYT Amryt Announces Successful Bioavailability Study for Mycapssa® (octreotide capsules) https://t.co/TaOAxkmrJP" (@stock_titan)

UVA Nuclear Medicine and Oncology Teams Join Forces to Offer Neuroendocrine and Prostate Cancer Patients the Latest in Theranostic Treatments (University of Virginia School of Medicine) - "Patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are traditionally treated with the chemotherapy drugs octreotide or lanreotide. 'With neuroendocrine cancers in general, the treatment, planning and care is more of a marathon than a sprint,' says...Matthew Reilley, MD. 'Typically, we're thinking long-term care, over years. A lot of patients are asymptomatic and the cancer is picked up incidentally. The goal of treatment is to maintain the disease as effectively as possible before patients develop problems or complications.'"

Media quote

"$AMYT Amryt Announces New Patents for Oleogel-S10 and Mycapssa® https://t.co/N9eIqWAuSE #patent" (@stock_titan)

Transient Permeation Enhancer® (TPE®) technology for oral delivery of octreotide: a technological evaluation. (PubMed, Expert Opin Drug Deliv) - ": The FDA approval of oral semaglutide for type 2 diabetes (2019) and oral octreotide for acromegaly (2020) is evidence that selected niche peptides can be administered orally if formulated with selected intestinal permeation enhancers. We evaluated the oral octreotide formulation, MYCAPSSA® (Chiasma Pharmaceuticals, Needham, MA, USA)...The oral octreotide dose required to achieve these endpoints was over 200 times that of the 0.1 mg immediate-release subcutaneous injection, a reminder of the difficulty in achieving oral absorption of macromolecules. Many acromegaly patients will prefer a convenient twice-daily oral formulation of octreotide compared to monthly depot injections."

Journal • Acromegaly • Diabetes • Endocrine Disorders • Metabolic Disorders • Type 2 Diabetes Mellitus

[VIRTUAL] Injection Site Reactions and Their Impact in Patients With Acromegaly Receiving Injectable Somatostatin Receptor Ligands in the Phase 3 MPOWERED Trial (AACE 2021) - "However, injection site reactions (ISRs) are common and impact patient activities of daily living and quality of life (QOL).1-3 Recently, oral octreotide capsules (OOC; MYCAPSSA ) were approved in the United States as a new treatment option for adult patients with acromegaly previously responding to iSRLs (octreotide or lanreotide). MPOWERED is a phase 3 trial assessing maintenance of response to OOC compared to iSRLs in patients with acromegaly. Here we report ISR-related data from patients receiving iSRLs in the randomized controlled treatment (RCT) phase of the MPOWERED trial."

Clinical • P3 data • Acromegaly • Endocrine Disorders • SSTR

[VIRTUAL] Improved Quality of Life and Work Productivity in Patients With Acromegaly Receiving Oral Octreotide Capsules in the Phase 3 MPOWERED Trial (AACE 2021) - "Patient-reported outcomes (PROs) are a key treatment objective in acromegaly. Validated PROs were used to assess disease and treatment burden in the MPOWERED phase 3 trial comparing oral octreotide capsules (OOC; MYCAPSSA ) to injectable SRLs (iSRLs)."

Clinical • HEOR • P3 data • Acromegaly • Endocrine Disorders

[VIRTUAL] Effects of Prior Injectable Somatostatin Receptor Ligand Type and Dose in Patients With Acromegaly Receiving Oral Octreotide Capsules in the Phase 3 MPOWERED Trial (AACE 2021) - "Oral octreotide capsules (OOC; MYCAPSSA ) are approved in the US for treating adults with acromegaly previously responding to and tolerating injectable somatostatin receptor ligands (iSRLs). MPOWERED is a phase 3 trial assessing maintenance of response to OOC compared to iSRLs in acromegaly. Here we report outcomes in patients receiving OOC based on their prior iSRL treatment (octreotide or lanreotide)."

Clinical • P3 data • Acromegaly • Endocrine Disorders • SSTR

[VIRTUAL] Improved Quality of Life and Work Productivity in Patients With Acromegaly Receiving Oral Octreotide Capsules in the Phase 3 MPOWERED Trial (AACE 2021) - "Validated PROs were used to assess disease and treatment burden in the MPOWERED phase 3 trial comparing oral octreotide capsules (OOC; MYCAPSSA ) to injectable SRLs (iSRLs). Eligibility criteria for MPOWERED included acromegaly diagnosis, current biochemical response (insulin-like growth factor I < 1.3 upper limit of normal; mean integrated growth hormone, < 2.5 ng/mL), and 6 months iSRL treatment (octreotide or lanreotide)... PROs, including quality of life (QoL) and work productivity, were all significantly improved in the Run-in phase in patients transitioning from iSRL to OOC. As patients served as their own control, the analysis was highly sensitive in the Run-in phase. Significant differences between treatment arms were not observed during the RCT phase, potentially due to sample size and variability of the measures."

Clinical • HEOR • P3 data • Acromegaly • Endocrine Disorders

[VIRTUAL] Understanding the Management of Acromegaly with MYCAPSSAa (octreotide) Delayed-Release Oral Capsules presented by Chiasma (AACE 2021) - "Monitor patients periodically. Discontinue if complications of cholelithiasis are suspected."

Acromegaly • Dermatology • Dyspepsia • Endocrine Disorders • Gastroenterology • Immunology • Musculoskeletal Pain • Osteoarthritis • Otorhinolaryngology • Pain • Respiratory Diseases • Rheumatology • Sinusitis

[VIRTUAL] Oral Octreotide Capsules Lowered Incidence and Improved Severity of Acromegaly Symptoms Compared to Injectable Somatostatin Receptor Ligands—Results From the MPOWERED Trial (ENDO 2021) - "The phase 3 MPOWERED trial assessed control of acromegaly by oral octreotide capsules (OOC; MYCAPSSA ® ) in comparison to injectable somatostatin receptor ligands (iSRLs) in patients responding to both OOC and iSRLs...Conclusion : Results from MPOWERED show that patients receiving OOC had significant improvement in number and severity of acromegaly symptoms after switching from iSRLs. These findings validate previous results from a phase 3 study of OOC in acromegaly in which patients switching to OOC from iSRLs showed significant reduction in joint pain, extremity swelling, and fatigue."

Acromegaly • Endocrine Disorders • Fatigue • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pain • SSTR

[VIRTUAL] Improved Acromegaly Patient Satisfaction With Oral Octreotide Capsules Compared With Injectable Somatostatin Receptor Ligands in the MPOWERED Trial (ENDO 2021) - "Validated PROs were used to assess disease and treatment burden in the MPOWERED phase 3 trial in acromegaly, which also assessed safety and efficacy of oral octreotide capsules (OOC; MYCAPSSA ® ) compared to injectable SRLs (iSRLs)...Breakthrough symptoms were reported more frequently with iSRLs (31%) than OOC (15%) at the end of RCT. Conclusion : Higher patient satisfaction, convenience and emotional well-being, and improved symptom control based on the newly validated Acro-TSQ PRO reporting tool were observed with OOC compared to iSRLs in patients enrolled in the MPOWERED trial."

Clinical • Acromegaly • Endocrine Disorders • Mood Disorders • Psychiatry • SSTR

[VIRTUAL] Safety Results From MPOWERED, a Phase 3 Trial of Oral Octreotide Capsules in Adults With Acromegaly (ENDO 2021) - "Oral octreotide capsules (OOC; MYCAPSSA ® ) were recently approved in the United States... In the RCT, incidence of treatment-emergent adverse events (TEAEs) was similar between groups; 39 patients (70.9%) in the OOC group and 26 (70.3%) in the iSRL group had ≥1 TEAE. 19 patients (34.5%) in the OOC and 15 (40.5%) in the iSRL group had treatment-related TEAEs. Occurrence was similar for serious AEs (OOC, 5.5%; iSRL, 8.1%) as well as TEAEs classified as severe (OOC, 9.1%; iSRL, 10.8%)."

Clinical • P3 data • Acromegaly • Constipation • Endocrine Disorders • Gastroenterology • Gastrointestinal Disorder • Pain • SSTR

[VIRTUAL] A Phase 3 Large International Noninferiority Trial (MPOWERED): Assessing Maintenance of Response to Oral Octreotide Capsules in Comparison to Injectable Somatostatin Receptor Ligands (ENDO 2021) - "Background : MPOWERED, a large phase 3 trial, assessed maintenance of response to oral octreotide capsules (OOC; MYCAPSSA ® ) compared to injectable somatostatin receptor ligands (iSRLs) in patients with acromegaly who responded to OOC and iSRLs (octreotide or lanreotide)...For oral presentations, the abstracts are embargoed until the session begins. The Endocrine Society reserves the right to lift the embargo on specific abstracts that are selected for promotion prior to or during ENDO 2021."

Clinical • Head-to-Head • P3 data • Acromegaly • Endocrine Disorders • IGF1 • SSTR

[VIRTUAL] Addition of Cabergoline to Oral Octreotide Capsules May Improve Biochemical Control in Patients With Acromegaly Who Are Inadequately Controlled With Monotherapy (ENDO 2021) - "Background : Oral octreotide capsules (OOC; MYCAPSSA ® ) are approved in the US for individuals with acromegaly who responded to and tolerated treatment with injectable somatostatin receptor ligands (iSRLs). Addition of cabergoline to OOC yielded biochemical control improvement (IGF-I reduction) in patients inadequately controlled with OOC monotherapy. As both combination and OOC monotherapy safety profiles were similar, adjunctive cabergoline may be helpful in patients with acromegaly who do not achieve adequate biochemical control on OOC alone."

Clinical • Monotherapy • Acromegaly • Endocrine Disorders • IGF1 • SSTR