Cornerstone Therapeutics Inc. Release: New Analysis Shows Lower Mortality Rates and Less Need for Redosing When Treating Neonatal Respiratory Distress Syndrome With CUROSURF(R) vs. Survanta(R)

CARY, NC--(Marketwire - January 26, 2012) - Cornerstone Therapeutics (NASDAQ: CRTX), a specialty pharmaceutical company focused on acquiring, developing and commercializing proprietary products for the hospital and respiratory markets, announced a new analysis published in the eFirst Pages section of the Pediatrics website reporting significant benefits in mortality rate and the need for redosing when treating neonatal respiratory distress syndrome (RDS) with Cornerstone's CUROSURF® (poractant alfa) Intratracheal Suspension(1). The objective of the independently performed meta-analysis was to compare the efficacy of CUROSURF versus Survanta® (beractant) and Infasurf® (calfactant) with respect to clinical outcomes among preterm infants with RDS. The analysis was led by Neetu Singh, M.D., at Dartmouth Hitchcock Medical Center.

About the Study
Researchers included the data following a systematic literature search using the methods of the Cochrane Collaboration for Systematic Review of Interventions. As a result, five published randomized controlled trials (RCTs) comparing CUROSURF and Survanta were included in the analysis; enrolling a total of 529 infants. There were no trials that met the designated criteria comparing Infasurf to CUROSURF.

In this review, it is important to note the initial surfactant dose administered to infants in each included RCT. The FDA approved initial dose for CUROSURF is 200 mg/kg versus 100 mg/kg for Survanta. Two of the five RCTs included a subgroup of infants that received a low initial dose of CUROSURF (100 mg/kg). This dose is not approved in the United States therefore the results of that subgroup are not reported in this release.

Following the analysis, authors concluded that the 200 mg/kg initial dose of CUROSURF may result in superior short-term clinical outcomes, compared with Survanta, when used for the treatment of preterm infants with established RDS. There were no significant differences in respect to complications of prematurity between CUROSURF and Survanta.

Key findings of the subgroup comparing the 200 mg/kg initial dose of CUROSURF to the 100 mg/kg of Survanta included:

• A significant reduction in mortality rates was found in infants treated with the 200 mg/kg initial dose of CUROSURF compared with those treated with the 100 mg/kg initial dose of Survanta. Those treated with CUROSURF experienced a relative risk reduction of 70% (RR: 0.29 [95 percent CI: 0.12-0.66], p=.004)
• The need for redosing was significantly lower with the 200 mg/kg initial dose of CUROSURF, with a relative risk reduction of 36 percent compared with the 100 mg/kg initial dose of Survanta (RR: 0.64 [95 percent CI: 0.53-0.83]; p=0.0008)

As with any meta-analysis, there are a few important limitations to note. This systematic review yielded a small number of relevant articles on surfactants that were considered sufficiently rigorous to be included (7 of the 92). In addition, each study included a relatively small patient population. Due to this, the largest study (Ramanathan R, et al 2004) contributed approximately 55% of all patients included in the analysis, as well as most of the weight and average of the summary treatment effect. Lastly, the included studies analyzed clinical outcomes only until hospital discharge and did not examine long-term outcomes such as neurodevelopmental outcomes.

The authors also noted that the results of this systematic review and meta-analysis are consistent with previously published reviews on this subject. Most recently, a retrospective study compared all-cause, in-hospital mortality in more than 14,000 preterm infants with RDS(2). This retrospective study found that the group receiving a 200 mg/kg initial dose of CUROSURF was associated with a significantly reduced likelihood of death compared to Infasurf and a trend toward reduced mortality when compared with Survanta.

"Cornerstone is delighted to see the growing body of data supporting the role of CUROSURF in treating preterm infants struggling with RDS," said Craig A. Collard, Cornerstone's Chief Executive Officer. "Our goal is that this analysis, along with previous published studies, will provide neonatologists the data they need to make an appropriate treatment decision at that critical point in the young patient's life."

About Respiratory Distress Syndrome
Neonatal RDS is a complication of prematurity that makes breathing difficult for infants with underdeveloped lungs. The condition is caused by a lack of adequate endogenous surfactant -- a protective substance that helps to reduce surface tension in the lungs. Without adequate surfactant, it is extremely difficult for infants to fully inflate their lungs and to avoid alveolar collapse. With approximately 550,000 premature births in the United States per year(3), the National Heart Lung and Blood Institute reports that 10 of every 100 premature babies develop neonatal RDS, with nearly all infants born before 28 weeks of pregnancy developing RDS(4).

To treat this condition, physicians will often administer an exogenous surfactant shortly after birth. This surfactant is meant to reduce surface tension, helping to improve oxygenation and lung function. Today, physicians have a choice of three different animal-derived surfactants in the United States: CUROSURF, Infasurf or Survanta. Each features a unique composition, volume requirements and surfactant dosage.

Of Note
The 100 mg/kg initial dose is not approved for use in the U.S. Per the product labeling, the approved initial dose for CUROSURF is 200mg/kg.

Cornerstone licensed CUROSURF U.S. rights from Chiesi Farmaceutici S.p.A. (Parma, Italy) as part of a broader transaction it completed with Chiesi in May 2009.

Indication
CUROSURF® Intratracheal Suspension is indicated for the treatment (rescue) of Respiratory Distress Syndrome (RDS) in premature infants. CUROSURF® Intratracheal Suspension reduces mortality and pneumothoraces associated with RDS.

Important Safety Information
CUROSURF is intended for intratracheal use only. THE ADMINISTRATION OF EXOGENOUS SURFACTANTS, INCLUDING CUROSURF, CAN RAPIDLY AFFECT OXYGENATION AND LUNG COMPLIANCE. Therefore, infants receiving CUROSURF should receive frequent clinical and laboratory assessments so that oxygen and ventilatory support can be modified in response to respiratory changes.

CUROSURF should only be administered by those trained and experienced in the care, resuscitation, and stabilization of preterm infants.

TRANSIENT ADVERSE EFFECTS SEEN WITH THE ADMINISTRATION OF CUROSURF INCLUDE BRADYCARDIA, HYPOTENSION, ENDOTRACHAEL TUBE BLOCKAGE, AND OXYGEN DESATURATION. These events require stopping CUROSURF administration and taking appropriate measures to alleviate the condition. After the patient is stable, dosing may proceed with appropriate monitoring.

Please see full prescribing information at www.curosurf.com.

References

1. Singh, N, et al. Pediatrics. 128(6): December 1, 2011; e1588 -e1595
2. Ramanathan R, et al. Journal of Perinatology. Advance online publication: September 1, 2011; doi: 10.1038/jp.2011.125
3. National Vital Statistics Reports (2009, March 18). Vol. 57, (12, p. 14)
4. National Heart Lung and Blood Institute (2009, September 01). What is Respiratory Distress Syndrome? Retrieved August 30, 2011, from http://www.nhlbi.nih.gov/health/health-topics/topics/rds

About Cornerstone Therapeutics
Cornerstone Therapeutics Inc. (NASDAQ: CRTX), headquartered in Cary, N.C., is a specialty pharmaceutical company focused on developing, acquiring, and commercializing products for the respiratory, hospital and related specialty markets. Key elements of the Company's strategy are to focus on identifying therapeutic niches within respiratory, hospital and related specialty markets to leverage existing business and create new opportunities; promote the Company's current products to high prescribing physicians through the Company's respiratory sales force and to hospital-based healthcare professionals through the Company's hospital sales force; license or acquire rights to existing patent- or trade secret-protected, branded products, which can be promoted through the same channels to generate on-going high-value earnings streams; advance the Company's development projects and further build a robust pipeline; and generate revenues by marketing approved generic products through the Company's wholly owned subsidiary, Aristos Pharmaceuticals, Inc.

CUROSURF® is a registered trademark of Chiesi Farmaceutici, S.p.A.
Survanta® is a registered trademark of Abbott Laboratories, Inc.
Infasurf® is a registered trademark of ONY, Inc.