Chiasma Previews Important Upcoming Milestones
CHIASMA OPTIMAL Phase 3 topline data expected in Q3 2019
Company anticipates submitting U.S. NDA by year-end 2019, assuming positive CHIASMA OPTIMAL data, and if accepted for filing, further expects a six-month FDA PDUFA review classification
Key U.S. commercial readiness activities planned in 2019
“During 2018, we completed the required enrollment in both of our Phase 3 clinical trials of our investigational octreotide capsules product candidate, which we have conditionally trade-named Mycapssa®, and with those trials progressing as planned, we believe we have set the stage for a catalyst-rich 2019 including the announcement of topline data evaluating Mycapssa’s efficacy as potentially the first oral somatostatin analog for the maintenance therapy of adult acromegaly patients,” said
“Multiple publications as well as initial screening data from Chiasma’s MPOWEREDTM clinical trial have highlighted the undesirable treatment burden associated with monthly injectable somatostatin receptor ligands, the current standard of care in the chronic treatment of adults with acromegaly. Based on this data and feedback from physicians and patients, an alternative orally administered therapeutic option could alleviate this burden for some patients. We believe Mycapssa, if approved as the first oral somatostatin analog, has the potential to become a standard of care in acromegaly maintenance treatment. In anticipation of positive clinical trial results in 2019, we intend to meaningfully transition into commercial readiness planning in 2019,” Mr. Fitzpatrick concluded.
Anticipated 2019 and 2020 Milestones:
- CHIASMA OPTIMAL (for U.S. approval). The Company anticipates reporting topline data in Q3 2019 from its ongoing international CHIASMA OPTIMAL (Octreotide capsules vs. Placebo Treatment In MultinationAL centers) Phase 3 clinical trial of Mycapssa for the maintenance therapy of adult patients with acromegaly. This trial is being conducted under a Special Protocol Assessment (SPA) agreement with the
U.S. Food and Drug Administration( FDA) to support potential regulatory approval in the United States. Chiasma announced in October 2018that it completed enrollment with 56 patients in 17 countries, including 21 from the United States, which exceeded its original SPA-agreed enrollment goal of 50 patients. The enrolled trial has been progressing as planned.
- U.S. NEW DRUG APPLICATION. Data from CHIASMA OPTIMAL, if positive, will support the Company’s New Drug Application (NDA) in the U.S., which the Company anticipates submitting by year-end 2019. The Company currently expects the
FDAto complete its review of the anticipated NDA, if accepted for filing, in six months based on our expectation that the planned NDA filing will be designated a Class 2 resubmission by FDAto address the April 2016complete response letter to our original NDA.
- MPOWERED (for EU approval). Chiasma anticipates completing enrollment in Q2 2019 of up to 150 patients into the run-in phase of MPOWERED (Maintenance of acromegaly Patients with Octreotide capsules compared With injections – Evaluation of REsponse Durability). Chiasma anticipates reporting topline data in the second half of 2020 from this global Phase 3 open-label clinical trial of Mycapssa. If data from MPOWERED demonstrates that oral octreotide capsules is non-inferior to standard of care somatostatin analog injections, such data is intended to support submission of a Marketing Authorization Application for potential approval of Mycapssa in the
- COMMERCIAL READINESS. Most diagnosed adult patients with acromegaly and their caregivers are readily identified. During 2019, Chiasma intends to build on its plans and preparations to commercialize Mycapssa in 2020 with a specialty U.S. sales team.
- CASH. Chiasma ended 2018 with estimated cash and cash equivalents of approximately
$41 million. The Company anticipates that its current cash balance is sufficient to fund its operations as currently planned through CHIASMA OPTIMAL topline data, and into early 2020.
Chiasma plans to be available for meetings with investors during next week’s 37th Annual
About the CHIASMA OPTIMAL Trial
The CHIASMA OPTIMAL trial is a randomized, double-blind, placebo-controlled, nine-month clinical trial of octreotide capsules being conducted under a SPA agreement with the
About the Chiasma MPOWERED Trial
Chiasma is conducting an international Phase 3 clinical trial under a protocol accepted by the EMA for the Company’s octreotide capsules product candidate for the maintenance therapy of adult patients with acromegaly. The trial, referred to as MPOWERED, is a global, randomized, open-label and active-controlled, 15-month trial intended to support approval in the
Acromegaly typically develops when a benign tumor of the pituitary gland produces too much growth hormone (GH), ultimately leading to significant health problems and early death if untreated. There are an estimated 69,000 individuals with acromegaly worldwide. In 13 studies of acromegaly prevalence since 1980, an average of approximately 75 cases per million was determined, suggesting roughly 24,000 individuals with acromegaly in
Common features of acromegaly are facial changes, intense headaches, joint pain, impaired vision and enlargement of the hands, feet, tongue and internal organs. Serious health conditions associated with the progression of acromegaly include type 2 diabetes, hypertension, respiratory disorders and cardiac and cerebrovascular disease.
Current treatment options include surgery to remove the pituitary tumor, radiation therapy, which destroys any lingering tumor cells, and/or medical treatment in cases where these approaches are not possible or fully effective. Today's medical treatments include dopamine agonists, GH antagonists, and injectable somatostatin analogs, which are the current standard of care.
Chiasma is focused on improving the lives of patients who face challenges associated with their existing treatments for rare and serious chronic diseases. Employing its Transient Permeability Enhancer (TPE®) technology platform, Chiasma seeks to develop oral medications that are currently available only as injections. In
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the Company’s development of octreotide capsules, conditionally named Mycapssa, for the treatment of acromegaly, the Company’s efforts to potentially obtain regulatory approval in
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