Seeks to Demonstrate Non-Inferiority of Mycapssa™ to Monthly Injections
Approximately 150 Patients Targeted for Enrollment
NEWTON, Mass., March 14, 2016 (GLOBE NEWSWIRE) -- Chiasma, Inc., (NASDAQ:CHMA), a late-stage biopharmaceutical company developing Mycapssa (octreotide) capsules, an investigational new oral drug for the potential maintenance therapy of adult patients with the orphan disease acromegaly, today announced the initiation of a second Phase 3 clinical trial to investigate the non-inferiority of octreotide capsules to once-a-month injectable somatostatin analogs. The results are expected to support the filing of a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA).
“The initiation of this global Phase 3 trial marks a significant milestone for us as we continue to build our comparative efficacy, safety, and patient reported outcome data set for Mycapssa,” said Mark Leuchtenberger, president and chief executive officer of Chiasma. “We are pleased to have opened our first sites in this trial and believe this innovative oral form of octreotide has the potential to alleviate chronic injectable treatment burdens for many patients.”
The MPOWERED™ study is utilizing an open-label, randomized, active-controlled design comparing the efficacy, safety and patient reported outcomes of Mycapssa to monthly somatostatin analog injections. The primary objective of this study will be to demonstrate the non-inferiority of Mycapssa compared to injectable somatostatin analogs as measured through the time-weighted average of insulin-like growth factor-1 (IGF-1) levels during the nine-month controlled phase. After completing the randomized controlled phase, all eligible patients will have the option of entering an extension phase in which they would receive Mycapssa until product marketing or study termination.
“For the past two decades, most acromegaly patients have been primarily prescribed injectable somatostatin analogs for their chronic treatment requirements,” said Maria Fleseriu, MD, FACE, professor and director of NW Pituitary Center, Oregon Health & Science University, Portland, USA, the global principal trial investigator. “MPOWERED has been designed to provide unique and invaluable insights into how today’s standard of care compares with a new oral product candidate, octreotide capsules. We look forward to the learnings that will be gleaned from this international trial.”
Chiasma plans to open approximately 50 U.S. and international sites in the MPOWERED study and to enroll 150 patients in total. Similar to Chiasma’s initial Phase 3 trial, all patients will receive Mycapssa during a six-month run-in phase. Patients identified as responders to octreotide capsules after six months will then be randomized at a ratio of three to two to either continue on Mycapssa or revert to their previous injections, respectively, for nine months. A non-responder sub-study will also be included in the trial to determine the efficacy of a combination of Mycapssa plus the drug cabergoline.
“Acromegaly is a challenging chronic disease that forces many patients to plan their lives around their injections, which must be administered by healthcare professionals in order to manage their disease,” said Christian J. Strasburger, MD, Chief of Clinical Endocrinology at the Department of Endocrinology, Diabetes and Nutritional Medicine, Charite Universitaetsmedizin in Berlin, Germany. “There continues to be an unmet need within the global acromegaly community for new treatment paradigms that would enable patients to manage their disease without needles.”
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. According to the U.S. National Institutes of Health, acromegaly occurs in approximately 60 people per million in the U.S. In addition, researchers estimate that three to four out of every million people develop acromegaly each year. Because symptoms often develop slowly, diagnosis may be delayed by years or decades, making it difficult to determine the total number of people with the disease.
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.
Mycapssa™ is an investigational new oral drug proposed for the maintenance therapy of adult patients with acromegaly. If approved, octreotide capsules would be the first oral somatostatin analog approved for acromegaly. Chiasma submitted an NDA on June 15, 2015 for this product candidate. The FDA has accepted the NDA for filing and the PDUFA date is April 15, 2016. The PDUFA date is the target date for the FDA to complete its review of the NDA. Octreotide capsules have been granted orphan designation in the United States and the European Union for the potential treatment of acromegaly.
Octreotide capsules are an investigational drug that have not been approved for use in any jurisdiction. The trade name Mycapssa™ has been conditionally accepted by the FDA and EMA as the proprietary name for Chiasma’s octreotide capsules product candidate.
Chiasma is a late-stage biopharmaceutical company focused on improving the lives of patients suffering from orphan diseases by developing and commercializing novel oral forms of therapies that are available today only by injection. The company’s lead product candidate is Mycapssa™ (octreotide) capsules, an investigational new drug under FDA review for the orphan condition acromegaly, developed with Chiasma’s Transient Permeability Enhancer (TPE®) technology to facilitate gastrointestinal absorption of unmodified drug into the bloodstream safely. Mycapssa™ is a proposed tradename, and this investigational new drug has not been approved for use in any jurisdiction. Using TPE® technology, Chiasma is evaluating additional proteins, peptides and small molecule drugs that are currently only available by injection but could potentially be converted to oral delivery. TPE® technology is potentially well suited for drugs with chronic indications, where frequent dosing is required and the need for an oral alternative is greatest. Chiasma is a Delaware corporation with a wholly owned Israeli subsidiary. Mycapssa™ and TPE® are trademarks of Chiasma.
Additional information can be found at www.ChiasmaPharma.com.
Readers should note that Chiasma communicates with investors and the public using its website (www.ChiasmaPharma.com) and its investor relations website (http://ir.chiasmapharma.com), including but not limited to company disclosures; investor presentations and FAQs; Securities and Exchange Commission filings; press releases; public conference calls and webcasts. The information that the company posts on these websites could be deemed to be material information. As a result, Chiasma encourages investors, the media, and others interested to review the information that is posted there on a regular basis. The contents of the company’s website shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
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 number of sites and patients to be enrolled in the trial and the company’s ultimate ability to submit a Marketing Authorization Application to the EMA. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks associated with the regulatory review process generally; the risk that the FDA may not make a decision on Chiasma’s NDA by the PDUFA target date; the risk that the FDA may determine that that the data included in the NDA are insufficient for approval and that Chiasma must conduct additional clinical trials, or nonclinical or other studies before octreotide capsules can be approved; the risk that the results of previously conducted studies involving octreotide capsules or other product candidates will not be repeated or observed in ongoing or future studies or following commercial launch, if such product candidates are approved; risks associated with obtaining, maintaining and protecting intellectual property; risks associated with Chiasma’s ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties; the risk that octreotide capsules, if approved, will not be successfully commercialized; the risk of competition from currently approved therapies and from other companies developing products for similar uses; risk associated with Chiasma’s ability to manage operating expenses and/or obtain additional funding to support its business activities; and risks associated with Chiasma’s dependence on third parties, including with respect to the manufacture of commercial supply in anticipation of commercial launch, if octreotide capsules are approved. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in the final prospectus related to Chiasma’s initial public offering filed with the Securities and Exchange Commission pursuant to Rule 424(b) of the Securities Act of 1933, as amended, as well as discussions of potential risks, uncertainties and other important factors in Chiasma’s subsequent filings with the Securities and Exchange Commission, including our most recent Quarterly Report on Form 10-Q filed on November 16, 2015. All information in this press release is as of the date of the release, and Chiasma undertakes no duty to update this information unless required by law.