2019 series: zolgensma becomes the listed drug of the year AZ wins the pharmaceutical company of the year

Biopharma dive, a foreign biopharma website, recently launched its 2019 series, which includes the annual biotechnology company, the annual pharmaceutical company, the annual CEO, the annual clinical data, the annual transaction destruction agency, the annual transaction, the annual new drug, etcThe following is an excerpt of some annual projects< br / > biotech company of the year - < br / > Galapagos < br / > maintaining independence is a challenge for small biotech companies, but the CEO of Galapagos believes that this is the key to the company's success< br / > for small biotech companies, it's rare to attract billions of dollars from a large partner without sacrificing independence, but Galapagos does! In July, the company signed an agreement with Geely to acquire up to $5.1 billion in cash and equity investment, as well as an unusual guarantee that Geely will not attempt to take over the company for at least the next decade< br / > despite gaining the autonomy of the company, now Galapagos needs to prove that Gilead's stake is favorable< br / > analysts of SVB leerink said that "Galapagos is likely to be the next regenerator or or gene Tek"However, to join the ranks of regenerant and Genentech, Galapagos must produce at least six drugs on the marketIt's also a challenge for large pharmaceutical companies, but Galapagos and gillid think it's possible< br / > the first real test to test Galapagos's R & D capability will be filgotinib, a JAK inhibitor anti-inflammatory drug, which is currently under regulatory review in Japan and Europe, and is planned to be submitted to US regulatory authorities for the treatment of rheumatoid arthritis (RA) by the end of the year< br / > although there are three kinds of JAK inhibitors on the market, the side effects of these drugs are lingeringHowever, some wall street people believe that Galapagos products are safer and will enter the list of heavyweight products after being launchedStifel, the investment bank, predicted that the peak sales of filgotinib in the RA field would reach US $2.5 billionThe key is whether Galapagos can obtain better label information than competitive products< br / > it's impossible to lie down and win with only one drug, Galapagos needs to produce more decent productsIn addition to filgitinib, at least six other drugs have entered the clinical stagePerhaps the most promising one is a secret project called Toledo, to which nearly half of the research organizations of Galapagos have been deployedSo far, investors only know that TOLEDO is the code name of a new class of anti-inflammatory drugs discovered by Galapagos, two of which are in phase I clinical The company intends to disclose the target of the project next year after launching phase II clinical < br / > now, everyone is curious about the Toledo project, and investors are also very interested in Galapagos When it was launched in partnership with Gilead, the company's share price surged 26 per cent, more than doubling since the beginning of the year As of December 6th, Galapagos had a market capitalization of $14 billion, comparable to some Biotech Corp in the commercialization stage, such as Alnylam and BioMarin < br / > Geely sees the value of Galapagos, too According to the company's executives, what attracts Geely is not just a certain asset, but Galapagos's chemical, biological and rapid drug development capabilities One of Geely's goals is to expand the pipeline, not only in terms of scale, but also in terms of diversity < br / > in fact, the two sides have also started negotiations on the acquisition before The main issue is the prepayment In June, the acquisition seems to have been impossible Geely eventually offered $3.95 billion for all current and future licenses and options for Galapagos drugs in markets outside Europe, plus $1.1 billion in equity investment < br / > Van de stolpe, CEO of Galapagos, said, "it doesn't matter whether Geely pays us $3 billion, 4 billion or 5 billion If the alliance succeeds, Galapagos will gain more benefits, and if Galapagos culture is maintained, the alliance is more likely to succeed." < br / > some analysts point out that although Galapagos believes that there is an opportunity to succeed in a deal that does not need Geely, it is still beneficial to have a large business partner, especially when drug pricing and filgotinib competition are imminent If one of Galapagos's riskier projects fails, the deal could provide a buffer But on the whole, it's smart for the company to choose to grow independently without tightening its belt < br / > pharmaceutical company of the year - < br / > AstraZeneca < br / > after several years of depression, AstraZeneca began to grow and take pride in a final delivery pipeline < br / > to understand AstraZeneca's position in 2019, you can first go back to 2012 when Pascal soriot took over as CEO At the time, AstraZeneca was also a company that relied on gastrointestinal, neuroscience, respiratory and cardiovascular medicine, many of which were facing shrinking sales < br / > by 2019, the cancer business led by tagrisso, imfinzi and lynparza is close to the peak sales of Crestor, AstraZeneca's powerful cholesterol lowering statins - more than $6 billion < br / > in this year, 5 of the 7 blockbuster drugs sold by AstraZeneca increased, while 5 of the 7 blockbuster drugs sold decreased in 2012 And revenue in the first nine months of this year was $2.2 billion more than in the same period in 2018 < br / > AstraZeneca's performance has also won new supporters Tim Anderson, an analyst at Wolfe research, described AstraZeneca as the "best long-term growth company" in the pharmaceutical industry and predicted that its revenue growth would "last until 2028" Andrew Berens, an analyst at SVB leerink, sees it as "one of the most powerful cancer drug makers" at the moment < br / > these may be the reasons why AstraZeneca's share price is up 30% this year (large pharmaceutical companies are up 6% on average) and Pascal soriot is one of the only two biopharmaceutical executives among the 100 best CEOs in the world in the Harvard Business Review < br / > at present, AstraZeneca has cut its business and R & D business, focusing on three main areas: Oncology, respiratory system, cardiovascular & kidney and metabolic diseases Within this range, the pipeline also favors cancer, accounting for 97 of 164 clinical research projects < br / > in 2019, lung cancer drug tagrisso has become the best-selling drug of AstraZeneca Although tagrisso's sales are far behind Crestor's sales at its peak, the advantage of the drug is that it has become the preferred drug for lung cancer targeted treatment, which requires far less marketing than Crestor At the same time, lynparza has become the best drug to treat ovarian cancer, breast cancer and other diseases < br / > compared with other large pharmaceutical companies, AstraZeneca ranked in the top three in terms of the number of candidate drugs and the number of treatment plans recently submitted to regulators, according to a statistical data compiled by Cowen, the investment bank At the same time, its forecast growth for 2018-2024 is ahead of all pharmaceutical companies < br / > in recent years, AstraZeneca has also sold the rights of brand drugs once cherished by seroquel, Nexium, etc to raise funds to focus on brands with more growth potential This strategy will give the impression of "self defeating", but it may be a necessary step to get rid of the burden of declining products The turning point came in 2018, when Pascal soriot announced its first revenue growth in three years < br / > in addition, for AstraZeneca, the Chinese market is very important Tim Anderson, an analyst at Wolfe research, said AstraZeneca's presence in China was "stronger than any of our other companies." For example, Amgen and Xinji enter the Chinese market through cooperation with Baiji Shenzhou, but AstraZeneca has its own business and research business, and further expands the latter to cooperate with local companies < br / > in the first nine months of 2019, AstraZeneca's sales in China reached US $3.7 billion, accounting for 61% of emerging market sales and 21% of total sales, an increase of more than one third compared with the same period in 2018 < br / > If AstraZeneca's weakness is that its sales volume is far lower than that of US $33 billion in 2010, another question is whether it can achieve the annual sales volume of US $40 billion by 2023, which is also the target Pascal soriot promised when it boycotted Pfizer's acquisition in 2014 Wolfe research and Cowen forecast that AstraZeneca's annual sales in 2023 will be $34 billion and $37 billion, respectively The commitment of < br / > 40 billion US dollars is based on the assumption that three oncology ace products are successful when there is still a great possibility of clinical failure Despite high-profile forecasts for imfinzi and brilinta at the time, it now looks easier to achieve than it did five years ago, with many bets paying off < br / > over compromise may lead to shareholder dissatisfaction and executive shuffle, but for AstraZeneca, at least in the next few years, it will not encounter this problem The company is on the cusp of good fortune and investors are eager to get involved < br / > annual drug launch - < br / > zolgensma < br / > this is a gene therapy with a price of up to $2.1 million, which is considered as a potential cure for SMA infants < br / > SMA is a serious neuromuscular disease Progressive muscle weakness and paralysis are caused by motor neuron death Three years ago, in addition to some daily care, there was no treatment for SMA infants < br / > this situation has changed dramatically in 2016, and Bojian's drug spinraza has been approved by the US FDA as the first treatment for SMA In May, Novartis' zolgensma gene therapy was also approved by the FDA < br / > unlike spinraza, zolgensma is a potential one-time cure The approval of the drug is a major achievement, and the biomedical industry has been working for decades to develop potential cures for devastating genetic diseases such as SMA < br / > the drug can prevent the disease progression by continuously expressing SMN protein after a single and one-off intravenous infusion, solve the root cause of SMA, and improve the quality of life of patients for a long time In phase I clinical trials that support the approval of the drug, SMA infants receiving treatment can stand and stand independently, even within months and years after treatment Whether zolgensma can really cure SMA will take years to come to a conclusion In theory, however, it is possible to cure SMA infants by giving them early enough treatment before the affected neurons begin to die < br / > a commitment to lifetime benefits is the core reason Novartis has priced zolgensma at $2.1 million In the first year, the treatment cost of Bojian spinraza was 750000 yuan, and then 375000 dollars per year If zolgensma is really curable at one time, $2.1 million seems acceptable in the long run In order to alleviate the concerns of insurance companies, Novartis has proposed to share zolgensma's expenses for five years, and is trying to establish a payment agreement to link reimbursement with patient outcomes However, no insurance company has yet accepted Novartis's installment proposal < br / > however, zolgensma and Novartis have received great attention recently due to data manipulation scandals According to previous reports from fiercepharma, Novartis included test data from preclinical mouse studies when submitting the application for approval, which were manipulated by senior officials of avaxis (or at the request of avexis) Novartis management knew in March that the data was fake, but it still submitted it The FDA said civil or criminal penalties could be imposed after the investigation is completed, but it confirmed the positive benefit risks of zolgensma, which can continue to be marketed < br / > after months of debate, the FDA partially halted a zolgensma study because of concerns about toxicity observed in experimental animals, a regulatory action that has rekindled the previous