Pfizer, Medivation, Aera and Azalea

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Hello everyone. Damian here with an update on the biotech market, a look at the future of genome editing and the evolving definition of ‘insider trading’.

Biotech is back where it started

After a volatile three months marked by some enthusiasm from JP Morgan and several billion-dollar trades, biotech stocks are trading almost exactly where they started the year.

The closely watched XBI biotech index closed yesterday at $90.40, just 17 cents higher than on January 2. The XBI rose as much as 14% in February, driven by a series of acquisitions and a prevailing sense that more was in the works. Cards. When these deals failed to materialize, biotech gradually declined in March. Now, at the beginning of the second quarter, this has completely leveled off.

The good news, after the prolonged recession of 2023, is that the sector has not collapsed. The XBI is up about 19% over the past twelve months and almost 40% since hitting a six-year low in October.

The quest to make CRISPR more precise

The world’s top gene-editing labs these days seem less interested in developing new gene editors than in devising new systems to safely deliver those editors to the right cells in a patient. Feng Zhang launched a startup called Aera, built around cargo transport particles hidden in the human genome. Jennifer Doudna has quietly launched a company, Azalea, that uses particles made from the hollowed-out shells of viruses.

And, as STAT reported last year, David Liu has raised $100 million from several blue-chip companies for a startup called Nvelop, also based on engineered viral shells. Today, Nvelop announced it’s coming out of stealth. The company, led by former Bluebird executives Jeff Walsh and Melissa Bonner, declined to say much about the future, such as the diseases it would target or when the technology would be ready for testing. But Nvelop did say that it has made progress in making Liu’s virus-like particles at scale — a major hurdle for any new platform — and that it had also acquired a gene delivery technology from Keith Joung, another prominent researcher.

The goal is to build a system that can safely place even large CRISPR systems into specific cell types. Today, companies can only reliably deliver CRISPR to the liver, and even then not without risk. “The delivery technologies now available to the field can address a certain number of disease indications, but they really cannot cover everything effectively,” Bonner said. With these two technologies, “I think we have a huge opportunity in front of us.”

Pfizer’s plan to win the RSV vaccine race

Pfizer has said it wants to expand the market for its RSV vaccine, Abrysvo, to adults ages 18 to 59 who have chronic health conditions that put them at higher risk if they contract respiratory syncytial virus.

The company said Tuesday that top-line data from its MONet study – a safety and immunogenicity study involving 681 volunteers – showed the vaccine generated as strong an immune response in adults under 60 who have chronic conditions as in adults aged 60 and older . . The vaccine was well tolerated in younger adults, the company said. Pfizer said it would submit the data to regulators and seek an expansion of its existing licenses, which are intended for adults 60 and older.

GSK, its rival in the RSV field, has already been requested to the FDA for an expansion of the license of its vaccine, Arexvy, to adults 50 to 59 years old at increased risk for RSV. In addition, GSK used a priority review voucher to speed the application through the FDA, which is scheduled to rule on June 7. Although GSK is asking for a smaller extension, in terms of age group, the two-month lead time and expedited review could give GSK a chance at a significant advantage, prompting the Centers for Disease Control and Prevention’s expert vaccine advisory group at a scheduled meeting in June could vote to recommend the use of Arexvy in people aged 50 to 59 – in time for a fall vaccination ahead of the next RSV season.

Expanding the use of RSV vaccines will undoubtedly spark a debate about the risks and benefits in younger adults, as there appears to be a link between receipt of these vaccines and a neurological condition known as Guillain-Barré -syndrome. GBS causes muscle weakness and sometimes paralysis. Most people recover completely over time, but some suffer permanent nerve damage. A Pfizer spokesperson said no GBS-like events occurred in the MONet trial.

Biotech innovates in insider trading

If you work at a biotech company, it is undoubtedly illegal to buy your company’s stock after learning of a not-yet-public plan to acquire it. But is it illegal to buy shares of a competing company that has no interest in the merger?

The answer is yes, according to the jury who convicted a former Medivation director for this. The case dates back to 2016, when a business development leader at Medivation learned that Pfizer had agreed to acquire his company. He then bought more than $100,000 worth of options on Incyte, one of Medivation’s competitors, reasoning that the company’s stock would rise once the buyout news became public. He was right and doubled his money in the process.

He then spent two years fighting the SEC, which argued that while he had not engaged in classic insider trading, his investment in Incyte was based on information not known to the public and had therefore broken the law . And now his conviction represents a meaningful expansion of the SEC’s ability to police the market.

Read more

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