BASEL, Switzerland, Jan. 20, 2005 (PRIMEZONE) --Novartis (NYSE:NVS):
* 75 projects in development, including 43 new molecular entities
* Key late-stage projects progressing on track
* New data on five of ten top-priority compounds presented
* AMN107 Phase I data reveal hematological response in over 50% of Gleevec-resistant patients
* FTY720 Phase II MS data show strong efficacy as potentially first oral therapy
* QAB149 Phase IIb data underscore efficacy in asthma and COPD
* First SPP100 Phase IIb/III data confirm efficacy in high blood pressure
* ICL670 set for US/EU submission based on response in 20 mg & 30 mg doses
* New Phase III data expected on six compounds and five key submissions in 2005
At a Research & Development review today, Novartis provided an update on its industry-leading pipeline, announcing robust progress in 75 development projects that aim to address unmet medical needs.
In addition to these 75 projects in clinical development, which includes 52 in phase II, III or registration and 43 new molecular entities (NMEs), Novartis announced that it has more than 64 candidates in advanced pre-clinical testing. Key areas of development are oncology and cardiovascular diseases, with promising compounds in both primary care and specialty medicines.
"Innovation is the core of our strategy, and our strong investments of USD 3.5 billion in pharmaceutical R&D in 2004 rank among the highest in the industry. In the US alone, our investments have allowed us to bring 13 new medicines to patients over the past four years, the highest number of any pharmaceutical company," said Dr. Daniel Vasella, Chairman and Chief Executive Officer.
"We continue to focus on novel medicines. Six of our 10 highly innovative compounds in mid-to-late-stage development are potential first-in-class medicines, and I am pleased that the expertise of our scientists, improved processes and advanced technologies have contributed to maintaining a leading position in R&D productivity," Dr. Vasella said.
Pipeline highlights
Novartis has been consistently ranked by industry experts as having one of the best pipelines, highlighted by several innovative compounds. These include SPP100 (hypertension), QAB149 (asthma and COPD) and FTY720 (multiple sclerosis and transplantation) as well as LAF237 (type 2 diabetes) and PTK787 (cancer). Many of these projects have the potential to become a new standard of care and the first to market in their respective classes.
The priority development projects include:
Oncology & Hematology
* AMN107 is an investigational oral compound being studied in the small subset of advanced chronic myeloid leukemia (CML) patients who do not respond or stop responding to Gleevec/Glivec and have no treatment options available. The compound is a signal transduction inhibitor that selectively inhibits specific proteins called tyrosine kinases, including BCR-ABL and some mutant forms of this protein, that cause white blood cells to grow and divide uncontrollably. AMN107 has been shown in preclinical studies to be the most selective BCR-ABL inhibitor to date and more potent than Gleevec. Phase I data showed hematological responses of over 50% in Gleevec-resistant patients in advanced disease stages (accelerated or blast phases). Phase II trials are expected to start in the first half of 2005.
* PTK787 (vatalanib) is an oral angiogenesis inhibitor in Phase III development for metastatic colorectal cancer. First Phase III results for PTK787, which is being developed with Schering AG, are expected in Q2 2005 from the CONFIRM1 trial in colorectal cancer. Enrollment was completed in 2004 for both the CONFIRM1 and the CONFIRM2 trials, which aim to show that PTK787 has the potential to set a new standard of care in metastatic colorectal cancer. Novartis plans to initiate an adjuvant colon cancer trial in 2005 pending positive CONFIRM1 data. A broad Phase I/II program is underway to identify additional indications for further development, including prostate, non-small cell lung (NSCLC), breast, pancreatic and ovarian cancers as well as glioblastoma and hematological malignancies.
* ICL670 (deferasirox) offers the potential to revolutionize the treatment of iron overload, providing a once-daily oral formulation to replace the cumbersome infusion therapy Desferal. Iron overload is a cumulative, potentially life-threatening condition that may result from repeated blood transfusions required to treat certain types of anemias, including sickle cell disease, thalassemia and myelodysplastic syndromes. It is estimated that, of the more than 250,000 people worldwide who require frequent blood transfusions, and as many as 100,000 people may require an iron chelator to avoid dangerous iron overload. ICL670 is expected to increase treatment compliance of this debilitating and life-threatening condition, leading to better health outcomes. Phase III data showed efficacy in the group of patients that received 20-30 mg/kg doses. Based on the results, submission in the US and EU with Orphan Drug Status is planned for the first half of 2005.
Cardiovascular & Metabolism
* LAF237 (vildagliptin) is a first-in-class incretin enhancer for the treatment of type 2 diabetes. It has been shown in Phase II trials to be effective in lowering blood-sugar (HbA1c) levels. Phase III development as a monotherapy and in combination with other medicines remains on track. Phase III data are expected at the end of 2005. Submission is planned for early 2006.
* SPP100 (aliskiren) is the first in a new class of antihypertension agents called renin inhibitors that offers a once-daily treatment with efficacy and safety comparable to angiotensin-receptor blockers (ARBs), another class of high blood pressure treatments. In contrast to other antihypertensive agents, SPP100 lowers renin enzyme activity in the bloodstream, so it may have the potential to better protect against heart attacks (myocardial infarction) and kidney disease. Phase IIb/III data confirmed efficacy as a monotherapy and suggested benefits of combination with ARBs. Phase III trials are ongoing in the US, EU and Japan. Additional Phase III data are expected in Q3 2005. The first regulatory submission is planned for early 2006.
Neuroscience
* FTY720, an oral immunomodulator with a novel mechanism of action, has shown excellent efficacy in multiple sclerosis (MS) in a Phase II study. FTY720 has the potential to become the first efficacious oral therapy for MS, a condition estimated to affect more than one million people worldwide. Data from the Phase II study showed a significant reduction in the relapse rate and in the number of brain lesions detected by MRI scan as well as a longer time to first relapse. The vast majority of patients are continuing in the extension phase. One-year data are expected in mid-2005. Phase III studies are planned to start in mid-2005.
Transplantation
* FTY720 is also being developed for transplantation. Phase III data remain on track for completion in 2005. US and EU submissions are expected in early 2006.
Respiratory diseases
* QAB149 is a once-daily long-acting beta-2 agonist for asthma and Chronic Obstructive Pulmonary Disease (COPD) that offers a quick onset of action and true 24-hour control. New Phase IIb data showed strong efficacy in both asthma and COPD and confirmed safety at high doses. QAB149 is being developed first as a monotherapy for the bronchodilator market. Treatments like QAB149 are expected to maintain substantial market share in a rapidly growing asthma/COPD market. Phase III studies are planned to start in 2005. Regulatory submissions are planned for 2007. Several options for combination products are currently being evaluated in parallel.
* Xolair is on track for EU regulatory approval in 2005. This novel agent offers a breakthrough in treating asthma, particularly as a unique add-on therapy for adults and adolescents with severe persistent asthma who remain inadequately controlled with conventional medicines. Clinical data have shown Xolair offers substantial improvements in disease control and reduces clinically significant exacerbation rates. Xolair is being developed in collaboration with Genentech and Tanox.
Bone and joint treatment
* Aclasta(1) (zoledronic acid) has the potential to become the gold standard in treating osteoporosis. Having shown superior efficacy in Phase III studies in Paget's disease, a condition marked by abnormal bone growth, the single once-yearly bisphosphonate infusion has the potential to provide unsurpassed compliance and excellent bone protection. Aclasta has received priority review status in the US for Paget's disease, and a decision is expected in March 2005. EU approval is expected in the first half of 2005. Submission for postmenopausal osteoporosis is expected in 2007 following completion of ongoing Phase III trials. (1) Zoledronic acid (5 mg) is authorized to be marketed under the name Aclasta in Europe and is awaiting US approval of the name.
* AAE581 is an oral once-daily compound aiming to be the first cathepsin K inhibitor for treating osteoporosis. Well-tolerated and efficacious in Phase II trials, AAE581 has been shown to inhibit bone resorption and may have a positive effect on bone formation. Phase II trials are on schedule, and biomarker data are expected in Q4 2005. Phase III trials are planned to start in Q1 2006.
Infectious diseases
* LDT600 (telbivudine) is a once-daily tablet treatment for hepatitis B virus (HBV) infection, a disease estimated to affect about 400 million people worldwide. Phase II clinical data have shown efficacy exceeding lamivudine, the global market leader for hepatitis B therapeutics. The ongoing international Phase III GLOBE clinical trial, which includes China, is designed to evaluate telbivudine head-to-head against lamivudine. Patient enrollment was completed ahead of schedule in April 2004. Novartis holds a 57% investment in Idenix, and both companies plan to submit US, EU and international marketing applications for telbivudine beginning in Q4 2005 and extending into 2006.
Ophthalmics
* Lucentis(TM) is a recombinant antibody fragment designed as a new treatment for "wet" AMD (age-related macular degeneration), an eye condition that can cause vision loss. Developed with Genentech, which retains the right to develop and market the product in North America, Lucentis binds and inactivates VEGF (vascular endothelial growth factor), a protein that plays a role in angiogenesis (the formation of new blood vessels). Data from two Phase III trials are expected in 2005. Submission for EU approval is planned for 2006.
A total of 12 development projects were added to the portfolio, while 16 left the portfolio through approval or launch (5 projects) as well as through termination or for being put on hold (11 projects). Among the terminated projects is TCH346 in Parkinson's disease and amyotrophic lateral sclerosis (ALS) after Phase II studies did not demonstrate efficacy.
Novartis expects the EU Mutual Recognition Procedure (MRP) for Prexige, a novel anti-inflammatory medicine being developed for osteoarthritis, to resume in mid-2005 after the European Medicines Agency (EMEA) completes a review of the COX-2 inhibitor class. Data from the landmark TARGET safety outcomes study in August 2004 demonstrated a significant 79% reduction in the incidence of upper gastrointestinal (GI) ulcer complications without compromising cardiovascular (CV) safety. The TARGET study showed that Prexige had a cardiovascular profile similar to conventional non-steroidal anti-inflammatory drugs (NSAIDs). Discussions are underway with the FDA on requirements for a new cardiovascular safety study. Additional studies are already underway to support the 100 mg dose for treating osteoarthritis. However, submission for US approval is not expected before 2007.
Promising early-stage projects
A priority of Novartis R&D efforts is to expedite "proof-of-concept" trials in humans through a "translational medicine" approach to better assess a compound's clinical potential by collecting biomarker data as early as possible.
Novartis highlighted a group of nine early stage compounds currently in Phase I/II that offer significant potential based on the results of proof-of-concept testing. The richness of the early-stage pipeline illustrates the replacement power of the Novartis pipeline and demonstrates the continuing high performance of the research organization.
Among the priority early-stage projects is LBM642, a novel agonist of both PPAR-alpha and PPAR-gamma (peroxisome proliferator-activated receptor) for the treatment of metabolic syndrome, a group of risk factors that include obesity, insulin resistance, elevated cholesterol and high blood pressure. LBM642 was shown to be more efficacious in lipid lowering than fenofibrate in a first proof-of-concept trial. Proof-of-concept data in diabetes are expected in mid-2005.
Other early-stage projects include ACZ885, which offers a new mechanism for treating rheumatoid arthritis; AEE788, an anti-cancer agent with promise in many solid tumors; valopicitabine (also known as NM283), a novel therapy for hepatitis C infection being developed by Idenix, for which Novartis has an exclusive right to in-license; and AEB071, a first-in-class immunomodulator with potential in transplantation.
Important submissions planned for 2005
In 2005, Novartis anticipates making submissions for at least three new molecular entities for regulatory approval as well as making a series of applications for new indications of products already on the market, including the following:
* PTK787 in the US for colorectal cancer (pending Phase III results of CONFIRM1 trials)
* ICL670 in the US and EU as the first oral treatment for chronic iron overload
* LDT600 in the US for the treatment of hepatitis B
* Femara in the US and EU for a new indication for treating women with breast cancer in the early adjuvant (post-surgery) setting (pending initial data from the BIG 1-98 trial expected to be presented at the St. Gallen Breast Cancer Conference in January)
* Visudyne in the US for the occult form of age-related macular degeneration (AMD)
Seven major projects are currently in registration, including Aclasta for the treatment of Paget's disease in the US and EU, the novel asthma medicine Xolair in the EU following approval in the US in mid-2003 and Zelnorm/Zelmac for treating irritable bowel syndrome (IBS) in the EU. Regulatory decisions are expected in 2005.
Strategic alliances complement pipeline
Novartis has more than 100 alliances with biotechnology and academic institutions looking for a major pharmaceutical company partnership. At the same time, Novartis recognizes the value of scientific advances that are made by partners and seeks to source the best technologies and early-stage compounds. Novartis has a culture designed to build fair, effective and mutually beneficial alliances.
Our alliance strategy in 2004 focused on two key components to strengthen our pipeline: development of research alliances and the in-licensing of new compounds.
A key focus of in-licensing was the acquisition of three compounds to complement internal cardiovascular/metabolism programs. D-4F, a novel apoA-I (apolipoprotein A-I) mimetic, was acquired from the US biotechnology company BruinPharma. ApoA-I is the major protein component of HDL and is considered a promising target for atherosclerosis therapy since it has been associated with beneficial effects on cardiovascular disease. Phase I development of D-4F has been initiated. An exclusive agreement was signed with Xenon Pharmaceuticals to develop and commercialize compounds from their Stearoyl-CoA Desaturase-1 (SCD1) program, which is in pre-clinical development. This program is complementary with Novartis programs targeting type 2 diabetes and the metabolic syndrome, which are conditions characterized by risk factors including obesity, insulin resistance, elevated cholesterol and high blood pressure. An agreement was also signed with Torrent Pharmaceuticals to develop its AGE (Advanced Glycosylation End-products) breaker compound in heart disease and diabetes-related vascular events, which is also in pre-clinical development.
Complementing internal discovery efforts in oncology, Novartis selected VX-322, a protein kinase inhibitor from Vertex in 2004, and also acquired from Vernalis the exclusive rights to the UK biotechnology company's development program for Hsp90, a target implicated in a number of different cancers.
Novartis also recently acquired all rights to Triad's pre-clinical p38map kinase program, which may deliver a promising oral therapeutic for inflammatory diseases. Novartis will be responsible for full development and commercialization of all compounds covered under Triad's intellectual property.
New alliances included Morphosys (development of therapeutic antibodies), Cellzome (indentification of critical molecular pathways and interactions), Infinity Pharmaceuticals (expanding the chemical universe for new drug discovery), and the Broad Institute of MIT and Harvard (discovering the basic genetic causes of type 2 diabetes).
Disclaimer This release contains certain forward-looking statements relating to the Company's business, which can be identified by the use of forward-looking terminology such as "pipeline", "potential", "set for", "2005 newsflow", "aim to", "potential", "expected", "plans to", "planned", "being developed", "aiming to", "early-stage projects", "anticipates", "promising", "may deliver", or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, or regarding potential future revenues. Such forward-looking statements 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 any of the development projects described in this release will succeed, that any new products will be brought to market, or that any new indications will be approved for any of our existing products. Similarly, there can be no guarantee that Novartis, or any present or future product, will achieve any particular level of revenue. In particular, management's expectations could be affected by, among other things, additional analysis of clinical data; new clinical data; unexpected clinical trial results; 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; as well as factors discussed in the Company's Form 20-F filed 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 described herein as 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
Novartis AG (NYSE:NVS) is a world leader in pharmaceuticals and consumer health. In 2004, the Group's businesses achieved sales of USD 28.2 billion and net income of USD 5.8 billion. The Group invested approximately USD 4.2 billion in R&D. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 81,400 people and operate in over 140 countries around the world. For further information please consult http://www.novartis.com.
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