GEN released the latest “Seven Biomedical Trends to Watch in 2021”

GEN released the latest “Seven Biomedical Trends to Watch in 2021”

GEN released the latest “Seven Biomedical Trends to Watch in 2021”.   Seven biomedical trends worth watching in 2021.

Last year, when GEN released the article “Eight Trends in Biomedicine in 2020”, who could foresee the death, suffering and economic collapse caused by the COVID-19 pneumonia virus this year? Who can foresee that the industrial and academic research fields will compete to develop hundreds of new vaccines and drugs? Although the biopharmaceutical industry has been affected by the COVID-19 pneumonia and has promoted a surge in related research and business activities, there have also been some developments in the industry itself that will enable it to continue to grow after the virus is contained.

The epidemic and other hot treatment areas have pushed venture capital financing to new heights, and at the same time promoted a new round of mergers and acquisitions (M&A) activities and an increase in initial public offerings (IPOs). Traditional drug research and development, as well as clinical and commercial activities for cell therapy and gene therapy have increased. Investors are keen on emerging technologies that have the potential to provide new therapies, including genome editing and synthetic biology.

The following are seven biomedical trends that are worth paying attention to in 2021 proposed by experts:

1. COVID-19 pneumonia: looking to the finish line

With Pfizer and BioNTech’s Phase III vaccine data being positive, hopes of finally ending the new coronavirus pneumonia pandemic ignited in November, and the two companies quickly sought emergency authorization for the BNT162b2 vaccine. This hope was strengthened when Moderna (a US biotech company that developed innovative treatments for a series of diseases) reported on the progress of its mRNA-1273 vaccine. Other encouraging developments include the FDA’s approval of the first treatment for new coronavirus pneumonia, the antiviral drug remdesivir launched by Gilead Sciences.

In December last year, Moderna’s mRNA-1273 and Pfizer/Biotech’s BNT162b2 vaccines, like remdesivir, were approved by the FDA. In addition to these vaccines, there may be several other vaccines, including AZD 1222 (AstraZeneca/University of Oxford), JNJ-78436735 (Johnson & Johnson’s Janssen Pharmaceuticals), NVX-CoV2373 (Novavax), MRT5500 (Translate Bio and Sanofi), V591 (Merck & Co.), and an unnamed vaccine being developed by Sanofi and GlaxoSmithKline. Jefferies securities analyst Michael J. Yee and three colleagues pointed out in a study on November 18, 2020 that with the approval of multiple new vaccines, the vaccination capacity is expected to increase nearly 150 times, from the fourth quarter of this year. 35 million people increased to 5 billion in the fourth quarter of 2021.

In terms of drugs, remdesivir may enter the market through two antibody therapies, namely, the bamlanivimab antibody (Lilly) and REGEN-co v2 antibody (Regeneron Pharmaceuticals) for mild to moderate COVID-19. In November last year, the FDA granted emergency use rights to Eli Lilly and Regeneron Pharmaceuticals, paving the way for the final approval of the two companies’ drugs. Remdesivir and Eli Lilly’s Olumiant combination therapy is also about to be approved, and the therapy is mainly suitable for hospitalized patients with new coronavirus pneumonia.

2. Gene therapy: big drug factory, generous

Large pharmaceutical companies have truly become interested in gene therapy and plan to expand their channels in 2021 and beyond. Bayer agreed in October to acquire Asklepios BioPharmaceutical for a maximum of US$4 billion. Two days later, Novartis acquired Vedere Bio, an ophthalmological company, for a maximum of US$280 million. Roche also agreed to spend US$1.8 billion to develop a new generation of adeno-associated viruses using Dynao Therapeutics’ CapsidMap platform (AAV) vector, the platform will be used for gene therapy of central nervous system diseases and liver targeted therapy.

Three days after homologous data from a phase I/II trial (NCT03952156) of pheNIX, a gene therapy drug for adults with phenylketonuria, showed positive, Pfizer invested $60 million in Homology Medicines. Among the biotech giants, Biogen and Sangamo Therapeutics are cooperating to develop and commercialize Sangamo gene regulation therapy drugs, which may bring Sangamo more than $2.7 billion in revenue.

A continuing challenge in 2021 will be to prevent tragedies from gene therapy clinical trials. In October this year, Lysogene disclosed that a five-year-old girl with mucopolysaccharidosis type IIIA (MPS IIIA) died in a phase II/III trial (NCT03612869) designed to evaluate the company’s LYS-SAF302. Between May and July, Audites Therapeutics, which acquired Astellas Pharma for USD 3 billion in January 2020, admitted 3 patients died in a phase I/II trial (NCT03199469). To evaluate its X-linked myotube disease (XLMTM) drug candidate AT-132. These 3 people belonged to 17 patients who received AT132 at high dose (3 × 1014 vg/kg). Audentes said that all three patients who died showed significant characteristics, including older age, heavier weight, and evidence of existing liver and gallbladder disease. “This is undoubtedly a wake-up call to remind the industry to consider dose escalation more seriously and pay more attention to CMC [chemistry, manufacturing and control] rather than simply improving efficacy,” said Penelope Booth Rockwell, Professor of Biomedical Research, University of Massachusetts Medical School. Said Dr. Guangping Gao, director of the Gene Therapy Center and Viral Vector Core Project, and co-director of the China Institute of Rare Diseases.

According to data from the Regenerative Medicine Alliance, gene therapy financing is currently increasing nearly three-fold year-on-year, increasing by 178% in the third quarter of 2020, reaching 3.5 billion US dollars. From January to September 2020, the amount of financing increased by 114% to US$12 billion. The alliance recorded 373 gene therapy trials in the third quarter of 2020, a 4% increase from 359 in the second quarter. Of these trials, 115 were in the first stage, 223 were in the second stage, and 32 were in the third stage.

3.Genome editing: CRISPR

From November 2020, genome editing has also brought huge amounts of cooperation. Eli Lilly has committed to spend US$2.7 billion to use Precision BioSciences’ ARCUS genome editing platform to research and develop potential in vivo treatments for genetic diseases, the first is Duchenne muscular dystrophy. Bayer’s investment subsidiary Leaps by Bayer provided $65 million in Series A financing for Metagenomi, a developer of CRISPR-based gene editing systems designed to develop cell and gene therapies.

CRISPR Therapeutics and Vertex Pharmaceuticals will continue to study CTX001, which is a CRISPR-Cas9 gene editing therapy. In two phase I/II trials in June, two patients with transfusion-dependent β-thalassemia proved its concept. Its effectiveness was proven in another sickle cell disease patient, which is the first clinical study on gene editing sponsored by a US company.

A market report released by a business research company on November 20 predicts that by 2023, the global CRISPR technology market will grow from $1.65 billion this year to $2.57 billion, and it will jump to $6.7 billion in 2030.

Once the legal dispute over who invented CRISPR-Cas9 is resolved, the market is expected to grow faster. This fierce dispute is the core of the second patent infringement lawsuit by the Patent Trial and Appeal Board (PTAB). In September 2020, PTAB ruled that the Massachusetts Institute of Technology (MIT) and Harvard University (Harvard) BROAD Institute as the winners, and gave the University of California, CRISPR Nobel Prize winner Dr. Emmanuelle Charpentier and the University of Vienna a relative disadvantaged position , Making the whole thing even more confusing. As to which party provided the strongest evidence that it was the first to demonstrate the effectiveness of CRISPR in eukaryotic cells, the PTAB did not immediately make a statement.

4. Financing: Excellent venture capital, M&A and IPO activities

Record-setting venture capital data shows that capital has flowed into biomedical companies. It is expected that this trend will continue in 2021, and the M&A stock market, IPO and the entire stock market will rise equally.

The “MoneyTree Report” quarterly report released by PricewaterhouseCoopers and CB Insights shows that in the third quarter of 2020, investment in biomedicine-related industries reached a record US$5.9 billion, of which only 104 in the field of life sciences Investments accounted for $3.9 billion, more than double the 74 investments of $1.9 billion in the third quarter of 2019. PricewaterhouseCoopers partner Ousmane Caba told GEN, “The COVID-19 seems to have little impact on the biotechnology and life sciences industries, and the virus has not prevented IPOs, transactions and mergers and acquisitions. Caba said that two factors have been driving financing in recent months. One is the ability to apply data to biology. The largest venture capital target in the third quarter was Recursion, a digital biology company that completed a D round of financing led by Bayer and oversubscribed US$239 million; another factor It is the long-term interest of drug developers and investors in the fight against cancer, especially technologies applicable to many forms of cancer.

Caba pointed out that in 2020, targeted cancer therapy has facilitated a series of valuable M&A transactions. Gilead Sciences completed its $21 billion acquisition of immunomedics in October, providing a first-class breast cancer treatment for its oncology product portfolio. In April 2020, the company received accelerated approval from the FDA. Illumina also plans to acquire Grail, a cancer liquid biopsy early screening company, for US$8 billion. This transaction will be completed in 2021. Grail’s ability to apply data also makes the company more attractive.

The IPO market will also perform strongly in 2020. EvaluatePharma’s data shows that in the first three quarters of 2020, biomedical companies raised a total of US$9.32 billion through 51 IPOs, more than twice the US$3.6 billion of 41 IPOs in the first three quarters of 2019. Overall, biotech stocks will grow well in 2020. As of November 20, the NASDAQ Biotechnology Index (NASDAQ Biotechnology Index) was US$4,364.15, an increase of 22% from US$3581.05 in the same period last year.

Morningstar (Morningstar) healthcare strategist Karen Andersen said: “We believe that by the end of 2020, the value of pharmaceutical and biotech stocks is undervalued.” “Overall, we have made progress in several therapeutic areas. Especially in the field of neurology, new progress has been made in the development of drugs for the treatment of amyotrophic lateral sclerosis, Parkinson’s disease and Alzheimer’s disease.”

5.Drug development: Alzheimer’s disease

In 2021, one of the most concerned drug development cases will be whether the FDA approves aducanumab, a drug for Alzheimer’s disease jointly developed by Biogen and Eisai. Aducanumab is currently undergoing priority evaluation with a target date of 20213 On the 7th.

In November last year, an advisory group recommended against approving aducanumab, after which the company encountered unexpected obstacles on its way to obtain FDA approval. The FDA’s Peripheral and Central Nervous System Drug Advisory Committee is hesitant to support aducanumab. In March 2020, Biogen discontinued EMERGE and ENGAGE (both plans are phase III trials of the drug) after analysis showed that the trial was unlikely to achieve the expected results. Then, in October, Biogen reported that the EMERGE data set was already available. Biogen also pointed out that in the ENGAGE trial, data from some patients supported new conclusions about the EMERGE trial.

“We think aducanumab will receive a complete response in early 2021 and may require us to conduct another large-scale trial before getting approval. We think the probability of approval in 2024 is 40%.” Andersen said: “If Biogen gets a complete In response, they will have to decide whether to continue investing in aducanumab or to focus on the development of a similar antibody BAN2401. Currently, the company and its partner Eisai are conducting two phase III studies.” Andersen added that aducanumab will either make people more interested Focusing on Alzheimer’s disease projects, such as those of Roche and Eli Lilly, will either inhibit people’s enthusiasm for treatments based on the removal of amyloid plaques. “In any case, we hope to continue to invest in other mechanisms of action, especially tau, and other ways to better cross the blood-brain barrier, such as Denali’s leucine-rich repeat kinase 2 small molecule inhibitor (by Biogen authorized), or RNA-based treatments, such as the therapies jointly developed by Ionis Pharmaceuticals and Biogen.”

According to Andersen, drug developers are also continuing to observe progress in oncology. She listed antibody-drug conjugates, such as the conjugate of Seagen (formerly Seattle Genetics), and two therapies with breast cancer indications-Enhertu and tru elvy of AstraZeneca. Gilead Sciences will complete the investigation of Immunomedics. The two therapies are inherited after the acquisition. “We will see more data about PD-1/PD-L1 antibodies, from the bispecific antibody project or other projects.” 

6. Cell therapy: big and small buyers

The developer is expected to extend last year’s cell therapy development to 2021. In November last year, PerkinElmer (the world’s largest manufacturer of analytical instruments) agreed to acquire Horizon Discovery Group for approximately US$383 million. The deal was designed to add gene editing and gene editing to the buyer’s portfolio of automated life science discovery and applied genomics solutions. Gene regulation tools. Sanofi bought Kiadis Pharma, a developer of natural killer cell therapies, for approximately US$364 million in November last year. Most of these therapies focus on cancer, but there is a pre-clinical treatment for COVID-19.

The two pharmaceutical giants have completed development facilities focused on cell therapy. In September, Takeda Pharmaceutical opened a new R&D cell therapy production plant in its research headquarters in Boston; in October, Astellas Pharma opened a US$120 million dedicated facility to be used by the Astellas Institute for Regenerative Medicine (AIRM) , And focus on the production and research and development of cell therapy.

The financing of cell therapy continues to grow at an unimaginable rate. According to the Regenerative Medicine Alliance, as of November 2020, cell therapy financing reached US$3 billion in the third quarter and reached US$11 billion this year, an increase of 97% and 242%, respectively. The number of Phase I trials evaluating cell therapy has increased from 41 to 50 (respectively in the first three quarters of 2019 and the first three quarters of 2020), but has declined in Phase II and Phase III. Overall, as of the third quarter, the number of clinical trials decreased from 218 to 202.

7. Synthetic biology: the time for financing

SynBioBeta recently shared some figures, indicating that synthetic biology is becoming increasingly attractive to investors. In the first half of 2020, the total financing in this field reached US$3.041 billion (56 companies). In the first half of 2019, this figure was only US$1.9 billion (65 companies). The largest financing of US$700 million came from Sana Biotechnology, a developer based on engineered cell therapy.

In September 2020, Zymergen raised US$300 million to promote its expansion into the US$3 trillion chemical and materials industry; accelerate the production of bio-composite transparent films for electronic applications; and will have advantages in electronics, agriculture, and consumer care. And commercialization of additional products for applications in healthcare.

The founder of SynBioBeta, Dr. John Cumbers, recently commented in Forbes magazine: “You must know that the coronavirus has not hindered the estimated 4 trillion dollar bio-economic development driven by synthetic biology.”

COVID-19 is helping to promote the development of synthetic biology and will continue in 2021. In May 2020, Ginkgo Bioworks raised $70 million to develop a large-scale testing infrastructure. In July 2020, the company announced that it had received approximately US$40 million in funding from the NIH RADx-ATP platform to scale up the NGS-based SARS-CoV-2 detection automation technology.

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