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May 17, 2009

Tobramycin inhalation powder (TIP) improved lung function in cystic fibrosis (CF) patients with respiratory Pseudomonas aeruginosa (Pa) infection

• Pa is the most common cause of infection and lung damage, affecting more than half of patients with CF
• Phase III study met primary endpoint with TIP providing improvement in lung function over placebo
• Treatment with TIP also decreased sputum Pa density, hospitalization and other antibiotic use
• The administration time of TIP was approximately 4 to 6 minutes

East Hanover, NJ, May 17, 2009 - In a Phase III study, TIP, an inhaled investigational formulation of tobramycin, improved lung function (as measured by FEV1) in cystic fibrosis patients with Pseudomonas aeruginosa (Pa) infection, compared to placebo. The data, presented today at the American Thoracic Society (ATS) 2009 International Conference in San Diego, also demonstrated, with respect to secondary endpoints, that TIP decreased sputum Pa density, hospitalization and other antibiotic use in these patients versus placebo.

TIP is currently in Phase III development for the management of CF patients with Pa infection. TIP, a dry-powder form of tobramycin, is delivered in approximately 4 to 6 minutes via a hand-held, portable, pocket-sized inhaler device, twice daily.

More than half of the people with CF have Pa infection, a respiratory condition caused by the Pa bacteria that settle into the thick mucus trapped in the airways. Pa is the most common cause of infection and lung damage in patients with CF.

"The daily treatment routine for patients with CF can take hours. The study findings presented at ATS are promising, as a new treatment option like TIP may allow people with CF an alternative for Pa management," said lead investigator Michael Konstan, MD, Professor and Director, The LeRoy W. Matthews Cystic Fibrosis Center, Rainbow Babies and Children's Hospital and Case Western Reserve University School of Medicine.

In a Phase III placebo-controlled study, patients with CF were administered treatment with TIP 112mg twice daily or matching placebo in a 1:1 ratio during Cycle 1 (28 days on and 28 days off treatment). This was followed by 2 cycles where all patients received TIP. The primary efficacy variable was relative change in forced expiratory volume in one second (FEV1) percent predicted from Day 1 to Day 28 (Cycle 1).

At 28 days, patients on TIP showed a significant improvement in FEV1 % predicted with an average improvement of 13% versus placebo (p=.0016). At the end of the first full cycle (Day 56), improvement over placebo in predicted lung function was maintained.

With respect to secondary endpoints, TIP reduced the mean sputum Pa density by 2.59 log10 colony forming units (CFU/mL), compared with 0.24 log10 for placebo. The proportion of patients requiring other antipseudomonal antibiotics was lower with TIP versus placebo (19.6% vs. 32.7%) and the mean duration of additional antipseudomonal antibiotic use was shorter (17 vs. 31.1 days, respectively) over the 56 days of Cycle 1. There were no respiratory-related hospitalizations for patients on TIP compared with 12.2% for patients receiving placebo (average duration of 12.3 days).

Adverse events were reported by 75.5% of placebo-treated and 50% of TIP-treated patients. The most commonly reported adverse events with placebo were cough, lung disorders and productive cough. With TIP, the most common adverse events were cough, lung disorders and sore throat. There were no major changes from baseline in vital signs, hematology, blood chemistry or urine protein. Audiology tests at selected sites indicated that there were no clinically meaningful decreases in hearing thresholds1l. None of the patients reported adverse events related to hearing.

"Novartis is working closely with respiratory researchers worldwide to develop medications to treat complex respiratory diseases with limited treatment options, including cystic fibrosis," said Robert K. Zeldin, MD, VP and US Medical Franchise Head of Respiratory and Dermatology, Novartis Pharmaceuticals Corporation. "We are pleased to report the findings of the TIP Phase III study, and we look forward to additional clinical data as we work towards regulatory filing."

Disclaimer
The foregoing release contains forward-looking statements that can be identified by terminology such as "can," "promising," "may," "to develop," "look forward," or similar expressions, or by express or implied discussions regarding potential marketing approvals for TIP, the potential development of other respiratory medications, or regarding potential future revenues from TIP or such other medications. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of the Company regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that TIP will be approved for sale in any market. Nor can there be any guarantee that Novartis will successfully develop any such other respiratory medications. Neither can there be any guarantee that TIP or such other medications will achieve any particular levels of revenue in the future. In particular, management's expectations regarding such products could be affected by, among other things, unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; the impact that the foregoing factors could have on the values attributed to the Novartis Group's assets and liabilities as recorded in the Group's consolidated balance sheet, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis Pharmaceuticals Corporation
Novartis Pharmaceuticals Corporation researches, develops, manufactures and markets leading innovative prescription drugs used to treat a number of diseases and conditions, including those in the cardiovascular, metabolic, cancer, organ transplantation, central nervous system, dermatological, GI and respiratory areas. The company's mission is to improve people's lives by pioneering novel healthcare solutions.

Located in East Hanover, New Jersey, Novartis Pharmaceuticals Corporation is an affiliate of Novartis AG, which provides healthcare solutions that address the evolving needs of patients and societies. Focused solely on healthcare, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, preventive vaccines, diagnostic tools, cost-saving generic pharmaceuticals, and consumer health products. Novartis is the only company with leading positions in these areas. In 2008, the Group's continuing operations achieved net sales of USD 41.5 billion and net income of USD 8.2 billion. Approximately USD 7.2 billion was invested in R&D activities throughout the Group. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 98,000 full-time-equivalent associates and operate in more than 140 countries around the world. For more information, please visit http://www.novartis.com.

References

1 Konstan, M., et al. Tobramycin Inhalation Powder Is Effective and Safe in the Treatment of Chronic Pulmonary Pseudomonas aeruginosa (Pa) Infection in Patients with Cystic Fibrosis. American Journal of Respiratory and Critical Care Medicine. 2009; 179: A1196. Poster presented at American Thoracic Society (ATS) 2009 International Conference, May 2009.

2 Moss, RB. Administration of Aerosolized Antibiotics in Cystic Fibrosis Patients. CHEST. 2001; 120: 107S-113S.

3 Pamucku, A. Effects of Pseudomonas aeruginosa Colonization on Lung Function and Anthropometric Variables in Children with Cystic Fibrosis. Pediatric Pulmonology. 1995; 19: 10-15.

4 Cystic Fibrosis Foundation Patient Registry 2007 Report, 2008. http://www.cff.org/UploadedFiles/research/ClinicalResearch/2007-Patient-Registry-Report.pdf. Accessed 6 May 2009.

5 Davis, PB. Cystic Fibrosis Since 1938. American Journal of Respiratory and Critical Care Medicine. 2006; 173: 475-482.

6 Sawicki, GS., et al. High Treatment Burden in Adults with Cystic Fibrosis: Challenges to Disease Self-Management. Journal of Cystic Fibrosis. 2008; 3(2): 91-96.

 

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