Important clinical trials in the first half of 2021

Important clinical trials in the first half of 2021. Clinical trials are testing grounds for new and experimental drugs. Good clinical trial results may be approved by regulatory authorities and change the way the disease is treated, while poor clinical results may dash hopes for many new drugs and new therapies and end testing.

For biotechnology and pharmaceutical companies, clinical trial results are also a high-quality currency for them to convince investors to raise funds and conduct transactions.

As the coronavirus pandemic continues to worsen in the United States and other countries in the world, the risks of many clinical trials this year are higher than ever. The successful application of vaccines, antibody drugs and antiviral drugs will help save lives and make COVID-19 an easier-to-control disease.

There are also important clinical tests for drugs for depression, hemophilia, lung cancer, and a rare disease (called alpha-1 antitrypsin deficiency). As the first six months of this year progress, please pay attention to the following clinical trials:

1 Companies: Johnson & Johnson, Novartis, AstraZeneca

• Disease: COVID-19 Pneumonia
• Treatment type: vaccine
• Trial: ENSEMBLE (J&J), UK Phase 3 (Novavax), D8110C00001 (AstraZeneca)

Two coronavirus vaccines from Moderna and its partners Pfizer and BioNTech have been authorized in the United States and other regions. However, both are difficult to apply on a large scale, and it turns out that the launch speed is slower than expected. However, if the above three phase 3 clinical trials have produced positive results, it may soon be relieved.

By the end of March, Johnson & Johnson, AstraZeneca and Novavax should all report the efficacy data of their vaccines. The success or failure of each test result is important for different reasons. For example, Johnson & Johnson is testing a single-use vaccine program, which will bring good news to the global vaccine supply.

Novavax is developing a protein-based vaccine, which is a more traditional approach than vaccines on the market. AstraZeneca (the drug’s injection has been used in the UK) has pre-sold about 300 million doses to the United States, while pre-sold hundreds of millions of doses to other countries in the world. In particular, the upcoming Phase 3 data of the 34,000 participating AstraZeneca vaccines from the United States may help explain questions about its effectiveness.

Novavax and Johnson & Johnson may release results before the end of January. AstraZeneca is expected to follow.

2 Company: Biomarin Pharmaceutical

• Disease: Hemophilia A
• Type of treatment: gene therapy
• Trial: NCT03370913

Roctavian may become the first gene therapy approved for hemophilia, which is the result of decades of research that can treat chronic blood diseases at once.

But the FDA unexpectedly rejected Roctavian in August, one of the most shocking decisions in recent memory. However, the regulator did not completely withdraw. Instead, they pointed to differences in results between the two Roctavian studies. Compared with earlier tests, in the initial participants of BioMarin’s Phase 3 clinical trial, the effect of the therapy appears to be weaker and gradually weakens over time.

At the same time, in the European Union, BioMarin withdrew its application after the European Medicines Agency was asked to provide more information.

Both regulators want to see more data, which is where BioMarin’s upcoming updated data is important. In January, the company expects to report follow-up data from its Phase 3 study. If Roctavian’s positive role continues, BioMarin may resubmit its approval application in Europe in the second quarter.

However, Roctavian has a longer road to return to FDA review. The FDA requires a two-year follow-up, and BioMarin’s results may not be available until November.

3 Company: Amgen

• Disease: lung cancer
• Treatment type: small molecule
• Trial: Codebreak 100

Drug makers have spent decades trying to design a drug that can block KRAS to treat cancer. The gene is usually mutated in several common tumor types. Amgen’s sotorasib has a chance to be the first, which is why the drug has quickly become the most valuable asset in biotechnology research and development.

Sotorasib’s most promising results so far have appeared in patients with advanced lung cancer, especially those with non-small cell lung cancer with a specific mutation (called KRAS G12C). For example, early data shows that the remission rate of treatment is approximately twice that of chemotherapy.

In October, Amgen issued a statement claiming that the drug showed similar efficacy in Phase 2 testing, and was safe and well tolerated, but did not reveal any other details. These data have been included in the company’s approval application submitted to the FDA and EMA in December.

Amgen will announce the results of the second phase of sotorasib monoclonal antibody at this month’s World Lung Cancer Conference, and will finally announce these results this month. These details can not only determine the scope of the drug’s use in lung cancer, but also determine how it compares with potential competitors such as Mirati Therapeutics. Amgen is also testing sotorasib monoclonal antibody in combination with other drugs (including immunotherapy), making it an important supplement to monotherapy.

4 Companies: Merck, Pfizer, Atea Pharmaceuticals / Roche

• Disease: COVID-19 Pneumonia
• Type of treatment: antiviral drugs
• Trial: END-COVID (Merck), NCT04535167 (Pfizer), NCT04396106 (Atea / Roche)

The clinical success of the coronavirus vaccine has made other methods of treating or preventing COVID-19 also become the focus of attention. However, despite the worsening of the pandemic, the introduction of vaccines in the United States has been slow and supply is expected to be limited.

Therefore, at the same time, any measures that can help COVID-19 patients, shorten the hospital stay or prevent organ damage will become the driving force for drug research. This makes the set of experimental antiviral drugs provided by Pfizer, Merck & Co., and partners Atea Pharmaceuticals and Roche worthy of attention.

One of these drugs is a new-purpose Ebola drug that has moderate benefits for hospitalized COVID-19 patients and must be injected intravenously for several days, which limits its wide application potential. Pfizer’s drug is a new type of antiviral drug that has shown potential against multiple coronaviruses. Merck’s drugs are developed together with Ridgeback biotherapy, which can provide options for early treatment outside the hospital. The same is true for Atea’s prospects, which prompted the company to enter one of the best biotech IPOs of 2020 and establish an alliance with Roche.

According to the federal government’s database, Pfizer’s Phase 1b study may produce results in April, while interim data from Merck and Atea therapy may appear in March.

5 Company: Sage Therapeutics, Biogen

• Disease: antidepressant
• Treatment type: small molecule drugs
• Trial: WATERFALL

Last year was a difficult year for Sage. Massachusetts Biotech reorganized, laid off half of its employees and replaced its CEO. Its depression drug zuranolone failed its phase 3 clinical trial in December.

But managers claim that this drug still has a chance to become a valuable drug. Biogen obviously also agrees with this view. In November last year, Biogen and Sage established a $3 billion partnership, which includes some rights of zuranolone.

Sage executives attributed zuranolone’s failure in major depression in 2019 to several underlying factors and have since designed a series of new phase 3 studies (including two studies in MDD) to prove this point . Three of these trials are expected to have results in 2021. The first is a study called Waterfall, which is testing zuranolone as an acute treatment for MDD. The data should be released in the first half of this year, and it will go a long way in judging whether zuranolone does have a future.

6 Company: Vertex Pharmaceuticals (Vertex Pharmaceuticals)

• Disease: Alpha-1 antitrypsin deficiency
• Treatment type: small molecule
• Trial: NCT04474197

In the mid-2010s, after the once promising hepatitis C drug franchise was cancelled, Vertex was able to recover quickly because the biotechnology company was ready to develop the cystic fibrosis business.

Part of the reason for its success is through a strategy of simultaneously developing multiple similar CF drugs and selecting the best CF drugs to proceed. The plan has created two companies, Symdeko and Trikafta, which are now selling top-tier drugs to pharmaceutical manufacturers in Boston.

Vertex’s goal is to follow its CF blueprint and develop another rare disease called alpha-1 antitrypsin deficiency. To this end, it has co-developed multiple drugs.

But the plan has been tested. Last year, in the worrying second phase of the trial, Vertex unexpectedly cancelled a drug called VX-814 due to worrying safety. This setback coexisted with fast-developing competition. Vertex also has another drug VX-864 in its candidates, which is said to be structurally different from its failed predecessor.

The Phase 2 trial should produce results in the first half of 2021, and its results may have a significant impact on the development of Vertex.

(source:internet, reference only)

Disclaimer of medicaltrend.org