FRIMLEY, England, September 17 /PRNewswire/ --

- New data, updated since ASCO, show time without tumour growth now reaches nearly 5 months with RAD001 vs. 1.9 months with placebo(1)

- One quarter of patients in trial remained progression-free beyond ten months of treatment(1)

- RAD001 is first drug to show significant benefit after failure of initial therapy, Sutent(R) (sunitinib) or Nexavar(R) (sorafenib)**, with potential to address unmet medical need(1)

- RAD001 under review by regulatory authorities; potential to be first once-daily oral mTOR therapy for advanced kidney cancer

New data continue to demonstrate the potential benefit of RAD001 (everolimus) for patients with advanced kidney cancer who have failed standard therapies.

Updated study findings from the RECORD-1 (REnal Cell cancer treatment with Oral RAD001 given Daily) study show that patients receiving RAD001 had no tumour growth for nearly 5 months vs. 1.9 months for patients receiving placebo (hazard ratio = 0.33 with 95% CI 0.25 to 0.43; p-value < 0.001). In addition, after more than 10 months of treatment with RAD001, 25% of patients still had no tumour growth.(1)

The updated RECORD-1 data were presented today at the 33rd European Society for Medical Oncology (ESMO) Congress in Stockholm, Sweden.

"These updated results demonstrate that RAD001 has the potential to delay tumour progression over an extended period of time in patients with advanced kidney cancer that progressed despite treatment with standard therapies," said Dr Peter Harper, Consultant Medical Oncologist, Guy's and St Thomas'. "These data reinforce my belief that RAD001 may provide a much-needed new treatment option for this very ill patient population."

RAD001 has the potential to fill a currently unmet medical need and become the first approved therapy to demonstrate significant benefit in patients with advanced kidney cancer after failure of standard treatment, including Sutent(R) (sunitinib) or Nexavar(R) (sorafenib), or both.

Earlier this year, interim results from RECORD-1 presented at the American Society of Clinical Oncology Congress were used to submit regulatory applications for RAD001 as a treatment for metastatic renal cell carcinoma (RCC) in Europe and the United States (US).

"Every year there are about 6,000 newly diagnosed cases of kidney cancer in the UK - that's 16 new cases per day or one every 90 minutes - and the incidence is steadily increasing,"(5) said Pat Hanlon, Kidney Cancer UK. "In such a difficult to treat cancer, these results are exciting and we hope that RAD001 may provide a new treatment option for patients who have few other avenues left open to them."

RAD001 is a once-daily oral therapy that may offer a new approach to cancer treatment by continuously inhibiting the mTOR protein, a central regulator of cell division and tumour blood vessel growth. Data suggest RAD001 has clinical activity as a single agent or in combination with other therapies in multiple types of cancer, including pancreatic neuroendocrine tumours, breast, gastric, lung and lymphoma.(2)

About renal cell carcinoma (RCC)

Renal cell cancer accounts for 2% of all new cancer cases worldwide with occurrence rates rising steadily around the world.(3) In the UK, RCC has increased by 22% over the last ten years. Each year, approximately 6,600 people in the UK are diagnosed with kidney cancer, which also causes around 3,600 deaths each year.(4,5) There are several types of RCC, but the most common, called clear cell, accounts for 80% of diagnoses.(4) In RCC, cancer cells develop in the lining of the kidney's tubes and grow into a tumour.

About RAD001

RAD001, an oral once-daily inhibitor of mTOR, is an investigational drug being studied in multiple tumour types. In cancer cells, RAD001 provides continuous inhibition of mTOR, a protein that acts as a central regulator of tumour cell division, cell metabolism and blood vessel growth. If approved, RAD001 will become the first oral, once-daily therapy that targets mTOR to treat advanced kidney cancer.(6)

The safety and efficacy profile of RAD001 has not yet been established in oncology and there is no guarantee that RAD001 will become commercially available for oncology indications. The active ingredient in RAD001 is everolimus.

In addition to renal cell carcinoma (RCC), RAD001 is being evaluated as a single agent or in combination with existing therapies in neuroendocrine tumours, lymphoma, breast, gastric, lung and other cancers, as well as tuberous sclerosis.(2)

RECORD-1 results

RECORD-1 is the largest Phase III clinical trial investigating the effects of an oral mTOR inhibitor in metastatic RCC. It is a randomised, double-blind, placebo-controlled multicentre trial of more than 400 patients with RCC whose cancer worsened despite prior treatment including Nexavar or Sutent, or both.(1) In addition, prior therapy with Avastin, interferon and interleukin-2 was allowed.(1)

The primary endpoint of RECORD-1 was progression-free survival (PFS) assessed via a blinded, independent central review and defined as the amount of time between randomisation and first documented disease progression or death due to any cause.1 Results of the study demonstrated a statistically significant improvement in PFS for RAD001 compared to placebo (hazard ratio = 0.33 with 95% CI 0.25 to 0.43; p-value < 0.001; median PFS 4.9 months vs. 1.9 months, respectively).(1)

Secondary endpoints included comparison of overall survival, objective response rate, quality of life and safety. There was no significant difference in overall survival between the RAD001 and placebo groups (hazard ratio = 0.82 with 95% CI 0.57 to 1.17; p-value = 0.137). The study design allowed patients to be unblinded at the time of radiological disease progression; patients receiving placebo were allowed to cross over to receive RAD001. There was no significant difference in objective response rate between the RAD001 and placebo groups (2% vs. 0% of responders). However, in a central review among patients evaluable for best percentage change in target lesions (223 and 107 in RAD001 and placebo arms, respectively), tumour shrinkage was observed in 50% of patients receiving RAD001 during the double-blind portion of the study vs. 8% of patients receiving placebo. Quality of life measurements taken throughout the study showed no significant difference between the RAD001 and placebo groups.(1)

Safety findings in the study were consistent with those seen in prior Phase II studies. The most frequent adverse events in patients who took RAD001 included mouth sores (36%), rash (28%), feelings of weakness (23%) and tiredness (22%). There was a low incidence of grade 3 or 4 drug-related adverse events (> 1% of patients listed): infection (4%), mouth sores (3%), tiredness (3%), feelings of weakness (2%), lung inflammation (2%), diarrhea (2%), mucosal inflammation (1%), vomiting (1%) and difficulty breathing (2%). The trial had a low rate of adverse drug reactions leading to discontinuation among patients who took RAD001 (7%).(1)

About Novartis

Novartis AG (NYSE: NVS) is a world leader in offering medicines to protect health, cure disease and improve well-being. Our goal is to discover, develop and successfully market innovative products to treat patients, ease suffering and enhance the quality of life. We are strengthening our medicine-based portfolio, which is focused on strategic growth platforms in innovation-driven pharmaceuticals, high-quality and low-cost generics, human vaccines and leading self-medication OTC brands. Novartis is the only company with leadership positions in these areas. In 2006, the Group's businesses achieved net sales of USD 37.0 billion and net income of USD 7.2 billion. Approximately USD 5.4 billion was invested in R&D. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 100,000 associates and operate in over 140 countries around the world. For more information, please visit

**Sutent is a registered trademark of Pfizer Inc. Nexavar is a registered trademark of Bayer HealthCare Pharmaceuticals, Inc.

For more information, please visit


1. Escudier, B et al. 72O - Phase III Randomised Trial of Everolimus (RAD001) vs Placebo in Metastatic Renal Cell Carcinoma. Presented at the European Society for Medical Oncology (ESMO) 33rd Congress, Stockholm, Sweden on 16 September 2008.

2. Novartis data on file.

3. McLaughlin JK, Lipworth L, Tarone RE. Epidemiological aspects of renal cell carcinoma. Semin Oncol. 2006 Oct;33(5):527-33. [Abstract]

4. UK Kidney Cancer Incidence Statistics. Cancer Research UK. Lat accessed on 12 September 2008 from

5. UK Kidney Cancer Mortality Statistics. Cancer Research UK. Last accessed on 12 September 2008 from

For more information, please contact: Sally Robinson, Novartis Pharmaceuticals UK Ltd, Tel: +44(0)1276-692255, Fax: +44(0)1276-698605,; Pip Rogan, Red Health, Tel: +44(0)207-025-6566, Fax: +44(0)207-025-6499, Email: