Forecast: 9 major events in the field of biomedicine in 2021!

In 2020, mankind has made a series of exciting progress in the field of biomedicine. For example, the COVID-19 vaccine/therapy has been developed at a record speed, and the new generation of AlphaFold artificial intelligence system has made a blockbuster in the protein structure prediction competition... etc. In 2021, what important events will the biomedical field usher in? Recently, BioSpace published the top 10 events that are worth looking forward to in the field of biomedicine in 2021. This article will briefly summarize the nine predicted events.

1. The COVID-19 epidemic is expected to usher in an inflection point

When will the COVID-19 pandemic end? This topic of concern may have an answer in 2021. At present, many countries and regions around the world have approved relevant vaccine products to market. For example, the inactivated virus vaccine of Sinopharm has been approved in China. In addition, three vaccines from companies such as Pfizer/BioNTech, Moderna, and AstraZeneca have shown a protective effect of 90% or more. The birth of these vaccines has brought hope for effective control of the COVID-19 epidemic.

According to the article’s prediction, the pandemic in 2021 will be brought under control and may basically disappear. Although most experts believe that the more likely outcome in the short term is to "control" the epidemic.

2. AstraZeneca/Oxford University's COVID-19 vaccine is expected to be approved

In the field of vaccine research and development for COVID-19, the new crown vaccine AZD1222 jointly developed by AstraZeneca and Oxford University has always attracted attention. Previously, it has been approved for emergency use in the UK for active immunization of adults over 18 years of age. According to reports, this is a new coronavirus vaccine based on the replication-deficient chimpanzee adenovirus vector. An important feature of it is that it can be stored in a refrigerated environment at 2-8°C for up to 6 months, for the storage, transportation and use of the vaccine. Provides great convenience.

The clinical results of the Phase 3 trial of AZD1222 have been published in The Lancet. Test data show that AZD1222 achieves 70.4% protection against COVID-19 disease with symptoms. Further analysis of the test data shows that adjusting the vaccination plan of AZD1222 may further improve the protective ability of the vaccine. A study published in The Lancet pointed out that the protective effect of the vaccine reached 90.0% in the group of half-dose vaccination first, and full-dose vaccination one month later.

3. Johnson & Johnson's COVID-19 vaccine is expected to bring a single-dose vaccine

JNJ-78436735 (also known as Ad26.COV2.S) is a new coronavirus vaccine candidate developed by Janssen, a subsidiary of Johnson & Johnson, based on non-replicating adenovirus vectors. In December 2020, Johnson & Johnson announced that the Phase 3 ENSEMBLE trial has completed all registrations, with approximately 45,000 subjects. They hope to obtain preliminary data from the trial before the end of January 2021. If the vaccine is shown to be safe and effective, the company plans to submit an emergency use authorization (EUA) to the FDA in February 2021.

Public information shows that JNJ-78436735, developed with Janssen AdVac technology, is expected to maintain stability for two years at -20°C and at least three months at 2-8°C. According to the article, the vaccine is different in that it only requires a single injection. If approved, there will be a single-dose vaccine that can be widely used.

4. Alzheimer's disease (AD) therapy aducanumab is expected to be approved

Aducanumab (code name: BIIB037) is an under-development monoclonal antibody targeting beta amyloid. Biogen received its research and development license from Neurimmune earlier. Since October 2017, Bojian and Eisai have jointly developed and commercialized aducanumab globally. The article predicts that the approval of the drug aducanumab for Alzheimer's disease may become a major event in 2021.

Previously, aducanumab has been shown in the final analysis of phase 3 clinical trials to significantly reduce the rate of cognitive decline in AD patients. Based on these findings, Biojian has submitted its Biological Products Licensing Application (BLA) to the FDA and obtained priority review qualifications. Since 2003, the FDA has not approved any new drugs for the treatment of AD, and many amyloid-targeting therapies under investigation have not shown significant efficacy in phase 3 clinical trials. Aducanumab not only represents the hope of delaying the disease progression of tens of millions of AD patients worldwide, it may also bring new life to the "amyloid hypothesis".

5. New opportunities in the M&A market

Affected by the epidemic, the overall M&A market in the biomedical field will decline in 2020. Recently, Yahoo Finance announced some potential acquisition targets in the biomedical field in 2021.

6. Venture capital (VC) in the field of biomedicine will increase

According to PitchBook forecasts, venture capital activities in 2021 will exceed $20 billion. This is mainly due to the increase in venture capital investors who want to enter the biomedical field and the development of the initial public offering (IPO) market. Although some pharmaceutical companies were forced to suspend clinical trials in 2020, with the successive launch of the new crown vaccine and related therapies, the epidemic is expected to be controlled in mid-2021 or earlier, and normal drug development activities will resume and continue. .

7. Hemophilia treatment Roctavian is expected to be approved

Roctavian is the flagship product of BioMarin. It is a gene therapy product for the treatment of hemophilia A. A functional copy of the gene encoding factor VIII is delivered to the patient through the AAV5 virus vector, thereby helping the patient to restore the ability to produce coagulation factor VIII. Relevant studies have shown that the drug can eliminate bleeding events to a large extent, enabling patients to significantly reduce their dependence on expensive clotting factor replacement therapy.

In August 2020, the FDA issued a Roctavian Complete Response Letter (CRL) to BioMarin, suggesting to obtain data from the ongoing Phase 3 BMN 270-301 study for another two years, with annual bleeding rate (ABR) as the primary endpoint assessment Its long-lasting effect in treating hemophilia A. This shows that BioMarin is expected to commercialize the drug as early as 2021. This will be an important moment worthy of attention for the emerging field of gene therapy for hemophilia.

8. CAR-T therapy targeting BCMA is expected to be approved for the treatment of relapsed/refractory multiple myeloma

Idecabtagene vicleucel (ide-cel) is a CAR-T cell immunotherapy targeting BCMA jointly developed by Bristol-Myers Squibb (BMS) and bluebird bio. BCMA is an important potential target of multiple myeloma. CAR-T cells targeting this target can be expanded in the human body after a single infusion, which may give the body lasting immunity against cancer cells, which means The patient is expected to achieve a one-time administration and obtain a "cure". Previously, ide-cel has been awarded the breakthrough therapy designation by the FDA and the priority drug designation (PRIME) granted by the European Medicines Agency (EMA).

In September 2020, the two companies announced that the FDA has accepted ide-cel's biological product license application, and the indication is for the treatment of adult patients with relapsed/refractory multiple myeloma (R/R MM). The FDA also granted priority review qualifications, and the PDUFA date is March 27, 2021. If approved, it will be bluebird bio's first commercialized drug. In a key single-arm, open-label phase 2 clinical trial called KarMMa, the candidate drug showed an objective response rate of up to 73.4%, showing the potential of this therapy in addressing the unmet needs of MM patients.

9. A new generation of PI3Kδ inhibitor umbralisib is expected to be approved

Umbralisib developed by TG Therapeutics is a new-generation PI3Kδ inhibitor and has been approved by the FDA as a breakthrough therapy. It is reported that the specificity of this product to PI3Kδ is better than other PI3Kδ inhibitors and will not inhibit PI3γ. And it can inhibit the protein called CK1-ε to prevent disturbing the function of regulatory T cells, thereby improving drug tolerance.

In August 2020, the FDA accepted the company's New Drug Application (NDA) for umbralisib, and the indications are patients with treated marginal zone lymphoma (MZL) and follicular lymphoma (FL). The application for MZL indications was also granted priority review by the FDA. According to the PDUFA date, the application for MZL is expected to be answered before February 15, 2021, and the application for FL is expected to be answered before June 15, 2021. The NDA is mainly based on the results of the Phase 2b trial called UNITY-NHL. Each cohort in the study reached the primary endpoint of ORR, which reached the company's 40%-50% range and was approved by the Independent Review Committee (IRC) Confirmed.