New drugs: Top 15 potential Blockbusters
Young and promising
Evgenia Lukyanchuk, "Weekly Pharmacy" www.apteka.ua based on FiercePharma, Financial Times and Bloomberg
The development of a new drug, although a very high-tech process, still somewhat resembles a roulette game. Pharmaceutical companies study the market, develop innovative active ingredients, conduct clinical research and at the same time make plans for the future, but they will not find out soon whether their drug is waiting for a truly stellar career. So far they can only guess. Next, 15 medicines that are at the final stage of clinical trials will be considered, which, according to the forecasts of analysts and representatives of development companies, have every chance of becoming blockbusters.
Dalcetrapib (dalcetrapib)Dalcetrapib ("Roche") is designed to increase cholesterol levels in high-density lipoproteins in patients with dyslipidemia.
Representatives of the company predict that the revenue from sales of this drug may reach $ 10 billion per year.
During phase IIb studies of the drug, it was found that the level of cholesterol associated with high-density lipoproteins, the so-called good cholesterol, in patients taking dalcetrapib increased by 31% compared with those taking placebo. In addition, there was no increase in the risk of developing cardiovascular diseases.
The prospects of dalcetrapib are due to the fact that it can be indicated for the control of cholesterol levels associated with high-density lipoproteins in patients with dyslipidemia due to diabetes mellitus. It should be noted that in studies conducted with the participation of 30 thousand patients with type II diabetes by Kaiser Permanente, a healthcare company, it was shown that the absence of significant fluctuations in the level of high-density lipoprotein cholesterol contributes to improving the prognosis of the course of the disease. And if we take into account the scale of the epidemic of diabetes, it becomes clear why the drug is predicted to have such a stellar future.
Analysts also predict the future of the megablockbuster for dalcetrapib, however, with a maximum sales volume of less than $ 10 billion per year. Andrew Weiss, an analyst at Bank Vontobel, estimates the potential sales volume of the drug at $ 5 billion per year, and Gbola Amusa, an analyst at UBS Bank, predicts that this figure could reach $ 6.8 billion per year.
Anacetrapib (anacetrapib)David Maris, an analyst at Credit Agricole Securities, predicts that annual sales of anacetrapib (Merck &Co) may exceed $10 billion.
The reason for such optimistic forecasts were the results of a study indicating that the use of anacetrapib leads to an increase in high-density lipoprotein cholesterol by 138% and a decrease in low-density lipoprotein cholesterol by 40% in patients taking statins.
However, along with a good clinical effect, developers need to prove to regulatory authorities that the drug has a favorable safety profile. This stage, at which even the smallest mistake cannot be made, because the stakes are too high, may delay the application for approval of the drug for several more years.
Bapineuzumab (bapineuzumab)At the dawn of the creation of bapineuzumab (Johnson & Johnson, Pfizer and Elan), intended for use in Alzheimer's disease, he was predicted a sales volume worthy of a megablockbuster, but everything changed in 2008, when it became known about cases of brain edema in patients participating in drug research.
After studying the causes of this pathology, the US Food and Drug Administration (FDA) decided to limit the participation in studies of patients who had previously had brain hemorrhages. However, this significantly limited the number of study participants, so after a while the FDA revised its decision and simplified the rules for selecting patients to study bapineuzumab. This was the reason for the revival of optimistic forecasts regarding future sales of the drug.
According to Guillaume van Renterghem, an analyst at UBS, the risk of failure is still quite high and it is too early to make any forecasts, although with a favorable outcome, the sales volume of bapineuzumab may reach 8 billion per year.
Solanezumab (solanezumab)According to forecasts of Tony Butler, an analyst at Barclays Capital, the maximum annual sales of solanezumab ("Eli Lilly"), intended for the treatment of Alzheimer's disease, may amount to $ 5 billion.
In addition, the market for related medicines is expected to grow from $5.4 billion in 2010 to $14.3 billion in 2020.
It is assumed that the results of phase III studies of solanezumab will be made public in Q3 2012.
Eliquis® (apixaban)In August 2011, the results of phase III studies involving patients with atrial fibrillation of the drug Eliquis® (apixaban, Pfizer and Bristol-Myers Squibb), intended for the prevention of thrombosis, were published.
Along with the fact that the use of the drug shows better results compared to the use of warfarin, it also reduces the risk of stroke or systemic embolism by 21%, bleeding by 31% and mortality by 11%.
Another reason why sales of Eliquis® are projected at $4-4.5 billion is the expected growth of the market for new antiplatelet drugs to $ 9 billion, and thanks to the favorable safety profile, Eliquis® has a chance to accumulate about half of this amount.
Seamus Fernandez, an analyst at the investment company Leerink Swann, predicts that the sales volume of Eliquis® will peak in 2017 and amount to $4.2 billion per year.
Bardoxolone (bardoxolone)The drug was developed by the American company Reata Pharmaceuticals and became the subject of a licensing agreement between it and Abbott Pharma, which paid $450 million to Reata Pharmaceuticals.
According to the creators of bardoxolone, it can be a breakthrough in the development of methods for the treatment of chronic kidney diseases, because unlike most other drugs that only slightly slow down the progression of the disease, bardoxolone improves the condition of patients. It is expected that the launch of bardoxolone will take place in 2014.
The drug has demonstrated good results of phase IIb studies. Abbott Pharma is currently organizing Phase III clinical trials. It is planned that 1.6 thousand patients with various kidney diseases at a late stage or with type II diabetes mellitus will take part in them. It is expected that the use of this drug will help to increase the period that these patients will be able to live without dialysis.
Abbott Pharma is currently negotiating with Reata Pharmaceuticals to jointly promote the drug on the U.S. pharmaceutical market, which could significantly increase the previously projected maximum sales volume of the drug by approximately $1 billion to $2 billion per year.
QVA149QVA149 ("Novartis" and "Vectura") is a combination of indacaterol and NVA237 (glycopyrronium bromide, "Vectura") and is intended for the treatment of chronic obstructive pulmonary disease.
Indacaterol is registered in the USA and the EU and is a representative of the class of long-acting β2-agonists. It relaxes the smooth muscles of the bronchi, thereby reducing the severity of the symptoms of the disease and improving the quality of life of patients with chronic obstructive pulmonary disease.
Currently, QVA149 is in the final phase of research. One of the problems to be solved by Novartis is the coordination with regulatory authorities of the dosage in which the drug will be produced. A dose of 150 mg of indacaterol is more effective, but regulatory authorities are more likely to approve a dosage of 75 mg for safety reasons.
In light of this, Novartis representatives announced their intention to conduct additional studies that would allow them to apply to the FDA for approval of QVA149 with a dosage of indocaterol at the level of 150 mg. This, however, will lead to a delay in bringing NVA237 to market. The company plans to submit an application for approval of QVA149 in the EU and other countries of the world, excluding the USA, in 2012.
According to the forecasts of Karl-Heinz Koch, an analyst at Helvea, the sales volume of the combination of indacaterol and NVA237 can reach up to $ 4 billion per year, a similar figure, according to the representatives of JP Morgan, will be at the level of $ 5 billion. However, according to Andrew Weiss, an analyst at Vontobel Bank, if it is not possible to register a drug with a dosage of indacaterol at the level of 150 mg, the revenue from sales of the combined drug may amount to $ 2.8 billion.
Xarelto®/Xarelto® (rivaroxaban)Anticoagulant Xarelto® ("Bayer" and "Johnson & Johnson") has already been approved by US regulatory authorities as a drug intended for the prevention of deep vein thrombosis in patients who have undergone surgery on the knee or hip joint.
Currently, the development companies have applied for approval of such a new indication for the use of Xarelto® as stroke prevention, which will allow the drug to reach the sales volume characteristic of a blockbuster. In addition, recently obtained data on the effectiveness of Xarelto® in acute coronary syndrome, which makes this drug even more promising.
It is expected that the revenue from sales of innovative drugs with higher efficacy and a favorable safety profile compared to warfarin may reach $20 billion. At the same time, Xarelto® can accumulate a significant share of these funds – about $2.9 billion per year.
T-DM1It is expected that the maximum sales volume of T-DM1 ("Roche"), intended for the treatment of breast cancer, may reach $2-5 billion.
The reason for such predictions was the results of phase II studies, according to which breast cancer patients who took T-DM1 statistically significantly lived longer without disease progression. In addition, fewer side effects were noted compared to patients taking a combination of Herceptin®/Herceptin (trastuzumab) and Taxotere™/Taxotere™ (docetaxel).
According to the developers, the more favorable safety profile of the T-DM1 is due to its structure. T-DM1 consists of antibodies to the epidermal growth factor Receptor (Human Epidermal growth factor Receptor 2 – HER2), similar to the antibodies included in Herceptin®, to which the chemotherapeutic agent DM1 is attached using a linker. This allows you to reduce the damage to healthy tissues of the patient's body during chemotherapy.
Currently, the company is conducting phase III studies, the results of which are expected to be included in the application for approval of the drug. It is planned that the application will be submitted in 2012. One of the phase III studies is EMILIA, during which T-DM1 will be tested as a second-line therapy for patients with HER2-dependent breast cancer. HER2 mutations are the cause of 20% of all breast cancer cases.
BG-12 (dimethyl fumarate)It is expected that the maximum annual sales volume of the drug BG-12 ("Biogen Idec"), intended for the treatment of multiple sclerosis, will amount to $ 2-3 billion.
The main goal of pharmaceutical companies in the field of creating medicines for the treatment of multiple sclerosis is to develop drugs for oral use that can reduce the likelihood of recurrence of the disease. BG-12 is a drug for oral use, which, according to the developers, can replace injectable drugs, which currently dominate the market of drugs for the treatment of this pathology.
At the end of 2011, the results of the first Phase III DEFINE study were presented, in which more than 1.2 thousand people participated. Thus, in patients with multiple sclerosis who took BG-12 twice a day, there was a decrease in the number of relapses by 53% during the year, and the risk of disability due to disease progression decreased by 38%.
Tofacitinib (tofacitinib)The world market of drugs for the treatment of rheumatoid arthritis, the volume of which is estimated at $ 20 billion, is currently also dominated by injectable drugs.
Therefore, the creation of an effective drug for oral administration is very much in demand. Clinical trials of tofacitinib (Pfizer), intended for the treatment of rheumatoid arthritis, are in the final stage. Their results indicate that tofacitinib, which is an inhibitor of the enzyme JAK (Janus kinase) intended for oral use, is characterized by the same efficacy as injectable drugs based on tumor necrosis factor already present on the market. It is expected that the maximum annual sales volume of tofacitinib will be $ 2.6 billion.
AMR101AMR101 ("Amarin") is a drug based on omega–3 fatty acids designed to reduce the level of triacylglycerides in the blood, a high value of which is associated with an increased risk of developing cardiovascular diseases.
It is expected that it will be approved by the US regulatory authorities in 2012.
Phase III clinical studies have shown that in patients taking the drug at a dose of 4 and 2 g / day, the level of triacylglycerides decreased by 21.5 and 10.1%, respectively. At the same time, there was no increase in the level of low-density lipoproteins and "bad" cholesterol, which gives AMR101 some advantage over those drugs for reducing the level of triacylglycerides, the use of which causes an increase in the content of low-density lipoproteins and "bad" cholesterol in the blood.
According to the estimates of the analytical company "Leerink Swann", the maximum annual sales of the drug may reach $ 3.2 billion in 2021.
Bydureon® (exenatide)Bydureon® ("Amylin Pharmaceuticals", "Eli Lilly" and "Alkermes") is intended for the treatment of type II diabetes mellitus and, according to forecasts, its maximum annual sales volume may amount to $ 1-3 billion.
The competition in the market of long-acting drugs based on glucagon-Like peptide-1 receptor agonists (Glucagon–Like Peptide-1 - GLP-1) is quite high, however, due to the fact that Bydureon® is enough to be administered once a week, it can count on a sales volume worthy of a blockbuster. The FDA is expected to announce its decision on the application for approval of this drug at the end of January 2012.
Pertuzumab (pertuzumab)Pertuzumab ("Roche") is a monoclonal antibody drug designed to treat HER2-positive breast cancer.
The results of the CLEOPATRA study indicate that simultaneous administration of pertuzumab, Herceptin™ / Herceptin (trastuzumab) and a chemotherapeutic drug statistically significantly slowed the progression of breast cancer compared with the use of Herceptin™ and a chemotherapeutic drug without pertuzumab.
According to the forecasts of analysts of Vontobel Bank, the maximum sales volume of the drug may amount to $ 1.8 billion per year.
QuadQuad ("Gilead") is a combination of the experimental active ingredient elvitegravir with the drug Truvada®/ Truvada (emtricitabine +tenofovir), and is intended for the treatment of HIV.
According to analysts from Decision Resources, the maximum annual sales of Quad is expected to reach $2.5 billion in 2018. A similar figure, according to Maged Shenouda, an analyst at Stifel Nicolaus, may amount to only $800 million in 2015.
Ideas are in the airIt should be noted that the attention of many pharmaceutical companies is focused on several of the most promising niches from the point of view of creating megablockbusters.
So, the list includes 2 drugs at once to control cholesterol levels in the blood, for the treatment of Alzheimer's disease and breast cancer. In addition, the development of drugs for the treatment of cardiovascular diseases that would be more effective and have a better safety profile compared to warfarin is very popular among the R&D departments of pharmaceutical companies.
However, despite the increasing competition, developers and experts are full of optimism, but whether their forecasts will be justified, we, and they themselves, will not know soon.
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10.01.2012