NEWTON, Mass. and JERUSALEM, Israel, Aug. 17, 2015 /PRNewswire/ -- Chiasma, Inc. (Nasdaq: CHMA), a U.S. late-stage biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company's New Drug Application (NDA) for the marketing and sale of octreotide capsules, an oral drug proposed for the maintenance therapy of adult patients with acromegaly. The FDA is expected to inform the Company of the Prescription Drug User Fee Act (PDUFA) date by the end of August. The PDUFA date is the target date for the FDA to complete its review of the NDA.
"We are very pleased that the FDA has accepted this filing for our octreotide capsules product candidate, as well as conditionally accepted the proposed trade name of Mycapssa™," said Mark Leuchtenberger, chief executive officer of Chiasma. "Based on our Phase 3 results and feedback from patients and healthcare providers, we believe that octreotide capsules have the potential to be an important treatment option for adults living with acromegaly, whose current standard treatment consists of lifelong and painful injections with an array of undesirable side effects. We look forward to working closely with the FDA in the coming months."
The application, submitted on June 15, 2015, is supported by a multicenter Phase 3 study that evaluated patients for biochemical and symptomatic disease control over a period of up to 13 months following treatment with octreotide capsules, comprised of a 7-month core treatment phase and an optional 6-month extension phase. Sixty-five percent of patients in the modified intent to treat (mITT) population who received octreotide capsules twice-a-day were classified as responders, as measured by circulating concentrations of insulin-like growth factor-1 (IGF-1) and growth hormone (GH) at the end of the 7-month core treatment period, the primary endpoint of the trial. Sixty-two percent of patients in the mITT population were classified as responders at the end of the 6-month extension phase. In the study, the severity and incidence of acromegaly symptoms improved while patients were on octreotide capsules, compared to baseline. The Phase 3 results were published in the Journal of Clinical Endocrinology & Metabolism.
The NDA was submitted utilizing the FDA's 505(b)(2) regulatory pathway. The 505(b)(2) pathway enables a sponsor to rely, in part, on the FDA's prior findings of safety and efficacy for a previously approved product, or published literature, in support of the NDA. The FDA has also conditionally accepted the Company's proposed trade name of Mycapssa™, a decision that is expected to be finalized upon potential approval of the NDA.
If approved, octreotide capsules would be the first oral somatostatin analog approved for acromegaly. An oral therapy for acromegaly has the potential to provide an alternative to the chronic injections currently used for a majority of acromegaly patients. These injections have historically been associated with pain and injection-site reactions during or after injection, as well as breakthrough acromegaly symptoms near the end of dosing intervals. Octreotide capsules have been granted orphan designation in the U.S. and the EU for the treatment of acromegaly.
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. Common features of acromegaly are facial changes, intense headaches, joint pain, impaired vision and enlargement of the hands, feet, internal organs and tongue. Serious health conditions associated with the progression of acromegaly include type 2 diabetes, hypertension, cardiac and cerebrovascular disease, and respiratory disorders.
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, the current standard of care. Currently available somatostatin analogs require large-bore needles for injections into muscle (octreotide) or deep into the tissue underlying the skin (lanreotide).
Researchers estimate that three to four out of every million people develop acromegaly each year. According to the U.S. National Institutes of Health, acromegaly occurs in approximately 60 people per million in the U.S. 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.
About Octreotide Capsules
Octreotide capsules are an investigational oral form of the peptide octreotide, a somatostatin analog that is currently available only by injection. Octreotide capsules use a proprietary technology developed by Chiasma, Inc., called Transient Permeability Enhancer (TPE®) that facilitates gastrointestinal absorption of unmodified drug into the bloodstream. Chiasma is independently leading the clinical and regulatory development and commercialization of octreotide capsules for the potential treatment of acromegaly and neuroendocrine tumors.
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 octreotide capsules for the orphan condition acromegaly. 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 using the Company's TPE® technology. Chiasma is a Delaware corporation with a wholly owned Israeli subsidiary.
Additional information can be found at www.ChiasmaPharma.com.
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the safety, efficacy and potential clinical benefits of oral octreotide, statements concerning the conditional approval of the tradename Mycapssa™ and statements regarding the timing of the FDA's review of Chiasma's NDA. Acceptance of the NDA filing does not represent final evaluation of the adequacy of the data submitted in the NDA and is not a guarantee of approval, nor is conditional acceptance of a tradename a guarantee of NDA approval or of final approval of such tradename. There also can be no assurance that the FDA will complete its review by the PDUFA target date, once determined. 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 determine that the data included in the NDA are insufficient for approval and that we must conduct additional clinical trials, or nonclinical or other studies, before oral octreotide can be approved; the risk that the results of previously conducted studies involving oral octreotide 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 oral octreotide, 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 oral octreotide is approved. For a discussion of 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. 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.
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SOURCE Chiasma, Inc.