Chiasma Provides Corporate Update and Previews 2020 Milestones
MYCAPSSA®NDA resubmitted to
MYCAPSSA® U.S. commercial launch planned for the fourth quarter of 2020
Top-line MYCAPSSA® MPOWERED Phase 3 results on track for the fourth quarter of 2020
2019 Key Highlights:
- MYCAPSSA New Drug Application (NDA) Resubmitted to
FDA: On December 26, Chiasma resubmitted its NDA to the U.S. Food and Drug Administration( FDA) for its investigational octreotide capsules product candidate, MYCAPSSA, for the maintenance treatment of adults with acromegaly. The positive results from the CHIASMA OPTIMAL trial, which was conducted under a Special Protocol Assessment (SPA) agreement with the FDA, support the NDA resubmission. Chiasma expects a six-month review of the NDA resubmission. The NDA resubmission marks an important milestone in the company’s effort to bring an oral somatostatin analog to patients.
- Manufacturing Plans: Following anticipated NDA approval, and in order to have commercial supply of MYCAPSSA product for launch, Chiasma expects to submit two manufacturing supplements to provide for (i) an additional active pharmaceutical ingredient (API) manufacturer and (ii) an additional commercial-scale manufacturing site affiliated with the API manufacturer currently referenced in the NDA. Chiasma is currently procuring API from both sources in anticipation of NDA approval. Chiasma expects to have adequate product available to support its planned commercial launch in the fourth quarter of 2020, subject to FDA’s timely approval of the NDA and either of the manufacturing supplements.
- CHIASMA OPTIMAL Global Phase 3 Trial for Acromegaly Met the Primary and All Secondary Endpoints: In July, Chiasma announced that the primary endpoint of the CHIASMA OPTIMAL (Octreotide capsules vs. Placebo Treatment In MultinationAL centers) trial, the proportion of patients biochemically (IGF-1 ≤ 1.0 x ULN) controlled at the end of the trial, was achieved. Additionally, all four secondary endpoints were met. The CHIASMA OPTIMAL trial was conducted under an SPA agreement with the
FDAand evaluated MYCAPSSA for the maintenance treatment of adults with acromegaly.
- Three Posters Presented at
ENDO2019 Highlighted Significant Unmet Needs in U.S. Acromegaly Patients: Data presented concluded that patients currently treated on injectable somatostatin analog therapies experience a significant disease burden and treatment dissatisfaction even when biochemically-controlled using existing therapies.
- MPOWERED Phase 3 Trial Completed Enrollment and Randomization: The MPOWERED (Maintenance of acromegaly Patients with Octreotide capsules compared With injections – Evaluation of REsponse Durability) trial, designed to support approval of MYCAPSSA in the European Union for acromegaly, completed enrollment in
June 2019. As of January 2020, the randomization was completed. Responders to the octreotide capsules were randomized per the protocol (IGF-1 < 1.3 x ULN) into the nine-month randomized controlled phase.
- Chiasma appointed key leaders in medical affairs, market access, patient services and marketing: Chiasma began its transformation into a commercial-ready organization with the appointment of a commercially-experienced chief executive officer and the addition of experienced leaders in marketing, market access and patient services. Chiasma has also expanded its medical affairs function, including hiring a leader with strategic medical and scientific expertise.
“2019 was a successful year for Chiasma, highlighted by the positive results from the Phase 3 CHIASMA OPTIMAL trial and the NDA resubmission,” said Raj Kannan, Chief Executive Officer of Chiasma. “Looking ahead, 2020 has the potential to be a transformational year for our company as we focus on executing a successful launch of the first oral somatostatin analog for the treatment of acromegaly.”
Anticipated 2020 Milestones and Other Guidance:
- MYCAPSSA NDA Acceptance: Chiasma anticipates that within 30 days of its NDA resubmission the
FDAwill determine whether the NDA is complete and ready for review and provide the review timeline.
ENDO2020 Conference: Chiasma has been selected to present the CHIASMA OPTIMAL Global Phase 3 results in an oral presentation at the Endo2020 conference. Additional abstracts have been submitted to the conference which is being held March 28-31, 2020 in San Francisco, CA.
- Publication of CHIASMA OPTIMAL Phase 3 Results: Chiasma plans to submit the final results of the CHIASMA OPTIMAL trial to a peer-reviewed journal for expected publication in mid-2020.
- MYCAPSSA PDUFA Decision: Chiasma expects a six-month review of the MYCAPSSA NDA resubmission and a PDUFA decision from the
- MYCAPSSA U.S. Commercial Launch: Chiasma plans to launch MYCAPSSA in
the United Statesin the fourth quarter of 2020, pending FDA’s timely approval of the NDA and either of the planned manufacturing supplements to the NDA.
- Top-line MPOWERED Phase 3 Results: Chiasma expects to report top-line results from the MPOWERED Phase 3 open-label trial of MYCAPSSA in the fourth quarter of 2020.
- Pipeline Expansion Leveraging Transient Permeability Enhancer (TPE®) Technology: Chiasma plans to announce its indication and pipeline expansion plans to develop one or more oral therapies targeting rare and serious chronic diseases with unmet needs following the anticipated approval of MYCAPSSA for the maintenance treatment of adults with acromegaly.
- Cash: As of December 31, 2019, Chiasma had approximately
$92 millionin cash, cash equivalents and marketable securities, which is expected to fund its operations, as currently planned, through at least 2020, including key milestones such as the planned FDAapproval of MYCAPSSA, the anticipated U.S. commercial launch, and the release of top-line MPOWERED Phase 3 results.
Chiasma management will be available to meet with institutional investors and analysts at the 2020 LifeSci Advisors Corporate Access Event, which is being held from January 13-15, 2020, in
CHIASMA OPTIMAL Trial Design
The CHIASMA OPTIMAL trial was a randomized, double-blind, placebo-controlled, nine-month Phase 3 clinical trial of octreotide capsules that was conducted under a SPA agreement with the
MPOWERED™ Phase 3 Trial
Chiasma is also 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, ultimately leading to significant health problems. 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. We believe that approximately 8,000 adult acromegaly patients are chronically treated with somatostatin analogs in the
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 data from the CHIASMA OPTIMAL trial and whether the data and the rest of the regulatory submission will support the acceptance by the
Vice President, Investor Relations and Corporate Communications
Source: Chiasma, Inc.