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FDA Denies Cornerstone Therapeutics Inc.'s Lixivaptan; Requests More Info  
11/1/2012 7:39:45 AM

Cornerstone Therapeutics Inc. ( NASDAQ : CRTX ), a specialty pharmaceutical company focused on commercializing products for the hospital and related specialty markets, announced today that the Company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) for lixivaptan for the treatment of symptomatic hypervolemic and euvolemic hyponatremia associated with heart failure and syndrome of inappropriate antidiuretic hormone (SIADH), respectively.

The Complete Response Letter requests additional clinical and non-clinical information in order for the FDA to consider approval of lixivaptan. The Company intends to request an End-of-Review meeting with the Division of Cardiovascular and Renal Products to better understand the contents of the CRL.

"We are reviewing the Complete Response Letter and will determine the most appropriate next steps for the Lixivaptan program," said Craig A. Collard, Cornerstone's Chief Executive Officer. "We believe we have strong clinical data supporting lixivaptan for the treatment of hyponatremia and that a significant unmet need remains for an alternative vaptan in the hospital and community setting."

Lixivaptan is a highly potent, non-peptide, oral capsule that works by reducing the action of a hormone (vasopressin) that blocks fluid excretion. Lixivaptan acts specifically on the vasopressin-2 receptor in the kidneys, causing water to be excreted while sparing sodium without affecting other electrolytes. Three large clinical trials, two evaluating lixivaptan for SIADH and one for heart failure, met the primary endpoint of producing a significant increase in serum sodium concentration compared to placebo at treatment day seven.

About Hyponatremia

Hyponatremia is a metabolic condition that occurs when there is not enough sodium (salt) in the blood. It is estimated to affect up to six million people in the U.S. with direct medical costs ranging between $1.6 and $3.6 billion annually. It is the most common electrolyte disorder among hospitalized patients and is often diagnosed in patients with heart failure. In the U.S. alone, there are five million heart failure patients, and each year one-fourth of them develop hyponatremia. Other causes of hyponatremia include burns, diuretic medications, kidney disease, liver cirrhosis and SIADH.

About Cornerstone Therapeutics

Cornerstone Therapeutics Inc. ( NASDAQ : CRTX ), headquartered in Cary, N.C., is a specialty pharmaceutical company focused on commercializing products for the hospital, niche respiratory and related specialty markets. Key elements of the Company's strategy are to focus its commercial and development efforts in the hospital and related specialty product sector within the U.S. pharmaceutical marketplace; continue to seek out opportunities to acquire companies and marketed and/or registration-stage products that fit within the Company's focus areas; and generate revenues by marketing approved generic products through the Company's wholly-owned subsidiary, Aristos Pharmaceuticals, Inc. For more information, visit www.crtx.com.

Safe Harbor Statement

Statements in this press release regarding the progress and timing of our product development programs and related trials; our future opportunities; our strategy, future operations and opportunities, including our plans and expectations regarding the manner and timing for the regulatory approval, manufacture and sale of our cardiovascular products, anticipated financial position, future revenues and projected costs; our management's prospects, plans and objectives; and any other statements about management's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including, without limitation, statements containing the words "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "plan," "project," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause our actual results or events to differ materially from those indicated by such forward-looking statements, including risks relating to our "critical accounting estimates"; our ability to develop and maintain the necessary sales, marketing, supply chain, distribution and manufacturing capabilities to commercialize our products; our ability to replace the revenues from our marketed unapproved products, which we ceased manufacturing and distributing at the end of 2010, our propoxyphene products, which we voluntarily withdrew from the U.S. market in November 2010 at the request of the U.S. Food and Drug Administration, or FDA, and our anti-infective products, which we divested in March 2012; the adverse impact of returns of previously sold inventory; patient, physician and third-party payer acceptance of our products as safe and effective therapeutic products; our heavy dependence on the commercial success of a relatively small number of currently marketed products; our ability to maintain regulatory approvals to market and sell our products; our ability to obtain FDA approval to manufacture, market and sell our products and product candidates, including our lixivaptan compound, CRTX 080, and RETAVASE; our ability to enter into additional strategic licensing, product acquisition, collaboration or co-promotion transactions on favorable terms, if at all; our ability to manage and control unknown liabilities in connection with any acquisitions; our ability to successfully manage growth or integrate acquired businesses and operations; our ability to maintain compliance with NASDAQ listing requirements; adverse side effects experienced by patients taking our products; difficulties relating to clinical trials, including difficulties or delays in the completion of patient enrollment, data collection or data analysis; the results of preclinical studies and clinical trials with respect to our product candidates and whether such results will be indicative of results obtained in later clinical trials; our ability to develop and commercialize our product candidates before our competitors develop and commercialize competing products; our ability to satisfy FDA and other regulatory requirements; our substantial indebtedness and debt covenants; and our ability to obtain, maintain and enforce patent and other intellectual property protection for our products and product candidates and the other factors described in Item 1A (Risk Factors) of our Annual Report on Form 10-K filed with the SEC on March 6, 2012 and in our subsequent filings with the SEC. If one or more of these factors materialize, or if any underlying assumptions prove incorrect, our actual results, performance or achievements may vary materially from any future results, performance or achievements expressed or implied by these forward-looking statements.

In addition, the statements in this press release reflect our expectations and beliefs only as of the date of this release. We anticipate that subsequent events and developments will cause our expectations and beliefs to change. However, while we may elect to update these forward-looking statements publicly at some point in the future, we specifically disclaim any obligation to do so, whether as a result of new information, future events or otherwise, except as required by law. Our forward-looking statements do not reflect the potential impact of any acquisitions, mergers, dispositions, business development transactions, joint ventures or investments that we may make or enter into. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this release.

Contact:

Investor Relations
Josh Franklin
Vice President, Strategy and Business Development
919-678-6520
josh.franklin@crtx.com

Media Relations
Fleishman-Hillard
Andrea Moody
919-457-0743
andrea.moody@fleishman.com

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