In 2019, Zolgensma, a gene therapy launched by Novartis AxeVis to treat children under 2 years old with spinal muscular atrophy ® Listed at a new record price of 2.1 million US dollars. This has again raised concerns about the price of treatment and the ability of politicians to address this issue. With the advent of the US presidential election season, this issue has gradually heated up.
In addition, with the outbreak of the COVID-19 at the end of 2019 and its continued spread in the world, the first treatment method for Alzheimer’s disease patients, as well as the wave of mergers between gene therapy contract developers and manufacturers, have also become new trends in 2020. The following is a summary of eight biopharmaceutical related trends mentioned by experts and insiders in the biopharmaceutical field in news interviews and other public statements.
presidential election
President Trump of the United States and congressional leaders of both parties seem to have found common ground on one issue. In recent years, they have all voiced the need to curb the rise in the price of prescription drugs. Whether or not all these remarks will turn into action will depend on who wins the White House and Congress in the November elections.
According to the Congressional Budget Office, Nancy Pelosi, Speaker of the US House of Representatives, released a bill in October last year that allows the plan to negotiate the price of 250 brand treatments, which is expected to save 345 billion dollars for medical insurance in 10 years. Republicans in Congress rejected Pelosi’s plan, believing that she was promoting a socialist bill to appease her most extreme members. In the United States Senate, Chuck Grassley, chairman of the Finance Committee, and Ron Wyden, senior member, had previously proposed the 2019 prescription drug price reduction bill, which stipulated that the out of pocket expenses of medical insurance beneficiaries should be capped at $3100. If the drug price rises above inflation, biopharmaceutical companies will be punished.
M&A
According to the data of Informa Pharma Intelligence, there were 242 M&A transactions in the first three quarters of 2019, with a total value of $195 billion. Two thirds (70%) of the total value (US $137 billion) came from two transactions. Bristol Myers Squibb (BMS) completed its US $74 billion acquisition of Celgene on November 20. Five months later, AbbVie agreed to acquire Allergan for US $63 billion, which is scheduled to be completed in early 2020.
The driving force of the merger and acquisition transaction includes that biopharmaceutical companies hope to purchase about half of the drug pipelines from outside, rather than through internal research and development, especially for candidate drugs under research and development. M&A is not necessarily the best way to purchase pre clinical or clinical trials, but it is definitely a good way to purchase revenue sources or late stage drugs, especially if they may have just been approved, and enterprises need business partners to maximize value.
Gene therapy CDMO
In 2019, two major acquisitions took place, merging the number of contract development and manufacturing organizations (CDMO). Both were carried out by buyers seeking to expand the manufacturing capacity of gene therapy, because the first batch of therapy was approved and entered the market: Thermo Fisher Technology acquired Brammer Bio at a price of 1.7 billion dollars in the transaction completed on May 1, while Catalent acquired Paragon Bioservices at a price of 1.2 billion dollars in the transaction completed on May 20.
It is expected that there will be more such acquisitions in 2020. Informa Pharma Intelligence analysts pointed out that the top five CDMOs only account for 15% of the total activities in this business area, compared with more than half (CRO) of the total activities generated by the top five clinical research organizations.
In the next year or two, we may see more activities. It is unlikely that the largest CDMO will buy all small CDMO. Medium sized CDMOs are more likely to increase their business by acquiring smaller CDMOs, while large CDMOs will acquire medium sized CDMOs.
Alzheimer’s disease
Biogen disclosed in its 2019 financial report that it planned to approve the application of aducanumab, an Alzheimer’s syndrome drug jointly developed with Eisai, to FDA, which surprised many market analysts. However, in April this year, according to the quarterly report information of Q1 2020, Biogen announced that it would delay the submission of biological product licensing application (BLA) for aducanumab until Q3 2020.
The new analysis of the larger data set in the trial shows that one study (NCT02484547) shows sufficient effective pharmacological and clinical activities, which is also supported by some results of another study (NCT02477800).
Venture capital transaction
In the third quarter of 2019, venture capital (VC) continued to flow to early companies, including newly established start-ups. However, although the number of M&A transactions remained stable compared with the third quarter of 2018, compared with the same period last year, the dollar value of these transactions declined significantly in the first quarter of 2019. With the slowdown of economic growth in the United States, Europe and Asia, this trend is expected to continue.
According to the quarterly MoneyTree report prepared by PwC and CB Insights, in the biotechnology industry, the dollar value of early financing in the third quarter of last year increased by 36% from $393 million in the third quarter of 2018 to $536 million, although the number of transactions was stable at 19. However, this quarter masked the overall year-on-year decline of 9 months, in which, the total transactions from January to September 2019 decreased by 27% to $1.625 billion from $2.228 billion in the first 9 months of 2018, and the number of transactions decreased by 9% to 60 from 66.
Genome editing application
At present, the application of genome editing technology is accelerating development, and this technology has been more widely used outside the traditional human drug development.
For example, in some fields where gene editing and even gene control were rarely seen before, such as livestock, animal health and companion animal health, some companies have also applied livestock gene editing and related technologies to create animal products that can be used for biomedical research, regenerative medicine and agriculture. Historically, many drugs for animals were developed in the process of developing drugs for humans. Some more exotic methods, such as cell therapy and gene therapy, are also used for companion animals.
As for gene therapy applied in humans, gene editing users will pay more and more attention to the efficiency of gene editing. If a person is conducting gene therapy, or even just cell therapy, whether targeted therapy in cells is really effective has to be considered. In particular, minimizing the miss effect has always been a worrying issue.
Prime editing
An important example of how genome editing surpassed CRISPR-Cas9 is the launch of prime medicine, which was co founded by Dr. David Liu of Harvard University and the Broad Institute of MIT.
In October last year, Liu and his colleagues introduced a new genome editing mechanism in detail. It does not produce double strand breaks in the target sequence, nor does it use donor DNA templates. It is called “Prime editing”. Liu is currently one of the co founders committed to commercializing this technology, and their startup company is named Prime Medicine. Prime has granted Beam Therapeutics an exclusive license to develop Prime editing technology for creating or editing any single base mutation and treating sickle cell disease. At present, Beam has been using its base editing technology to conduct research on these two aspects.
COVID-19
At the end of 2019, COVID-19 epidemic caused by novel coronavirus (SARS-CoV-2) broke out, and then spread to the world within two months, forming a global pandemic. By the end of June 2020, the number of infected people in the world had exceeded 10 million. Affected by the COVID-19, the A-share pharmaceutical sector has risen by 36.2% since 2020, ranking the first among all industries. The turnover and the premium rate of the sector also continue to hit new highs.
Since the therapeutic drugs related to COVID-19 are currently in the stage of emergency research and development and clinical trials, especially vaccine and antibody research and development, there are 15 vaccines in the world that have entered the stage of clinical trials, and hundreds of vaccines are under pre clinical evaluation. In addition, four kinds of therapeutic antibodies against COVID-19 have also entered clinical trials, and are expected to be put into emergency use before the end of the year.
To sum up, with the upcoming U.S. election, enterprise mergers and acquisitions, gene therapy and the COVID-19 epidemic, etc., will become important drivers of the biopharmaceutical industry.
Post time: Oct-21-2022