A Look at the Biggest Biopharma Stories of 2021

The biggest story in biopharma, at least in 2021, isn’t really one story, but many. Going through the most newsworthy stories of the year, BioSpace found trends more than one big story, topics that just kept rising again and again. Here’s a look.

#1. COVID-19. Not surprisingly, the number 1 story wasn’t one story, but many, many stories, all about COVID-19. Although everyone hoped 2021 would be less focused on the pandemic, it still dominated the news. Big COVID-19 stories included the vaccine rollouts by Pfizer–BioNTech and Moderna, which actually occurred at the very end of 2020, and then the early problems with that rollout. The Pfizer-BioNTech vaccine was fully approved by the U.S. Food and Drug Administration in August and has received authorization for expanded age groups and for booster shots. Unfortunately, as it seemed we were getting a handle on the pandemic, the Delta variant arose in June and eventually became the dominant strain around the world. Now, toward the end of the year, worries are growing over the highly infectious Omicron variant and what appears to be a fourth surge of the virus. There’s also been new hope, as on December 1, the FDA authorized Merck’s molnupiravir, an oral antiviral that appears moderately effective in treating COVID-19. Pfizer is also seeking Emergency Use Authorization (EUA) for its own antiviral, Paxlovid, which has been shown to reduce the risk of hospitalization of death by 89% compared to placebo in high-risk adults who were not hospitalized for treatment. And those were only some of the top stories domestically about COVID-19.

#2. COVID-19 Misinformation. Sadly, an underlying theme in the year’s COVID-19 coverage was what the World Health Organization (WHO) has called a “pandemic of misinformation.” Dr. Francis Collins, director of the National Institutes of Health (NIH), noted that scientists and public health officials underestimated how misinformation would undermine the “astounding achievement” of the vaccines. “Deaths continue … most of them unvaccinated, most of the unvaccinated because somebody somewhere fed them information that was categorically wrong and dangerous.”

Over and over again, stories were published in an attempt to debunk rumors about the vaccines, such as the idea that they caused menstrual changes or infertility, or the use of ivermectin as a cure-all. And in May, the FDA published an unusual public scolding of CytoDyn, accusing the company of misrepresenting its clinical trial results for leronlimab as a treatment for COVID-19.

#3. Biogen’s Aduhelm (aducanumab). In June, the FDA approved Biogen’s Aduhelm (aducanumab), the first drug approved for Alzheimer’s in almost 20 years. It was controversial prior to the approval but became even more so afterward. Members of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted against recommending it, and three members resigned over the approval. Investigations into the approval process have been launched by the Health and Human Services’ Office of Inspector General and the House of Representatives, and the drug has been very slow to gain traction in the marketplace.

#4. Gene Therapy Setbacks. Although gene therapy is just about as cutting-edge as you can get today in biopharma, with many expected to be approved in the coming years, 2021 marked a number of setbacks in the field. These included a clinical hold on BioMarin Pharmaceutical’s BMN 307 Phearless Phase I/II trial for phenylketonuria (PKU) after preclinical safety data found liver tumors in lab animals; a clinical hold on Rocket Pharmaceutical’s gene therapy for Danon disease, which was eventually lifted in August; a partial hold on Novartis’ OAV-101 intrathecal clinical trials placed in 2019 was also lifted in August; a clinical hold on bluebird bio’s elivaldogene autotemcel gene therapy for cerebral adrenoleukodystrophy (CALD), and others.

#5. Merck Buys Acceleron. The year wasn’t noted for any one big acquisition. One of the largest was Merck’s acquisition of Acceleron Pharma for $11.5 billion, which wrapped in November. There are rumors that Novartis might sell off its generic unit Sandoz, but to date, nothing has solidified in that area. Other significant deals included Jazz Pharmaceuticals’ acquisitionof GW Pharmaceuticals for $7.2 billion, Sanofi’s buyout of Translate bio for $3.2 billion and Horizon Therapeutics’ acquisition of Viela Bio for $3.05 billion.

#6. Bluebird bio Spinout. In January, darling of the gene therapy space bluebird bio announced plans to spin out its oncology business and stay focused on severe genetic diseases. Nick Leschly, the “chief bluebird,” is heading the oncology company while staying on as executive chair for bluebird bio. Andrew Obenshain took over as chief executive officer of bluebird bio.

Source: BioSpace

A strategic review led to the decision to split the two businesses, benefiting from independent leadership and separate teams. Leschly noted, “Specifically, we believe it is the right time to double down on the respective businesses to fully enable and optimize the continued innovation, development and deployment of transformative gene and cell therapies for the patients we serve.”

#7. FDA Cracks Down on Accelerated Approvals. An Accelerated Approval is when the FDA approves a drug based on biomarker evidence, rather than clinical benefit. The companies are then required to run a post-approval clinical study to determine if the drugs actually improve patients’ health. In March, it looked like the FDA was getting tougher on these approvals and in April, it was reported that the agency was going to review products approved under the pathway for specific cancers, particularly Merck’s Keytruda (pembrolizumab), Bristol Myers Squibb’s Opdivo (nivolumab), and Roche’s Tecentriq (atezolizumab), all checkpoint inhibitors.

#8. COVID-19 Origins. Unlike the more traditional biopharma stories about COVID-19, this year marked intelligence agencies, researchers and journalists digging into the origins of COVID-19. It falls predominantly into two camps: natural emergence from animals, probably bats, which transferred to humans, possibly at a wet market in Wuhan; and manmade, likely originating at the Wuhan Institute of Virology (WIV) where there was a lab leak. Unless the Chinese government becomes unexpectedly transparent about opening their records at WIV, the world may never fully determine the source of the virus, and at this time the evidence seems about evenly split between the “wet market” and “lab leak” theories.

#9. Several SPACs. Special purpose acquisition companies (SPACs) are shell companies that raise money in an initial public offering (IPO), which is then put into a trust until they merge with a private company to take it public. This was an unusually common practice this year, including Roivant Sciences combining with Montes Archimedes Acquisition Corp (MAAC); Aerami Therapeutics Holdings and FoxWayne Enterprises Acquisition Corp.; Blade Therapeutics and SPRIM Global Investments affiliate Biotech Acquisition Company; Science 37 and LifeSci Acquisition II Corp. Others include 23andMe and Virgin Group, Tango Therapeutics and BCTG Acquisition Corp, EQRx and CM Life Sciences III, Nautilus Biotechnology and SPAC Arya Sciences Acquisition III and many others.

#10. Viral Mutations. At the risk of throwing in yet another COVID-19-related topic, never before has the scientific community and general public paid so much attention to viral mutations. One wonders if the general public has ever paid any attention to viral mutations prior to this year. Although it’s probably worth pondering if some of the attention paid by the media to new variants is excessive — Delta-plus, for example — there’s no doubt that stories about new SARS-CoV-2 variants, ranging from Alpha (B.1.1.7) to Delta, Delta-plus and the new Omicron variant have caught the media and public’s attention.