Life Science Today 008 – J&J, Momenta, White House, FDA, Decentralized Trials
Originally Published as The Niche Podcast
Introduction
Welcome to The Niche Podcast – Your weekly 5-minute rundown of the biotech, clinical research, and applied science industries. I’m your host, Dr. Noah Goodson. This week, Johnson & Johnson acquires Momenta, the White House and FDA disagree, and the emergence of decentralized clinical trial management.
Johnson & Johnson Acquires Momenta
Momenta entered a definitive agreement to sell all shares to Johnson & Johnson for $6.5 billion at $52.50/share. The purchase agreement will bring all of Momenta’s assets and pipelines into the Johnson & Johnson family. While there are several potential therapies in development, the purchase is primarily focused on Nipocalimab, a potentially class leading autoimmune targeting antibody.
Momenta received orphan drug status for Nipocalimab to treat Hemolytic Disease of Fetus and Newborns in January of 2020 and launched a phase II/III clinical trial around the same treatment . The same month, they released results from a phase 2 clinical trial of Nipocalimab to treat generalized myasthenia gravis (gMG). And most importantly they are late in progress of a phase II/III trial using Nipocalimab to treat Warm Autoimmune Hemolytic Anemia.
How can one drug work in so many diseases? Nipocalimab is an engineered human anti-FcRn antibody. The FcRn receptor keeps IgG autoantibodies in circulation and perpetuates the symptoms of certain auto-immune diseases. By blocking these receptors, the disease-causing antibodies decrease in number and the symptoms of the disease are significantly eased. One of the remarkable findings in the studies of Nipocalimab so far, is the lack of serious adverse events combined with robust decrease in disease symptoms. The drug requires a two-hour infusion every other week, but for Johnson and Johnson that all adds to the cost of treatment and the potential value of their therapy. Provided the data align from the late-stage trials, we can expect several Nipocalimab approvals in 2021 and 2022. While Nipocalimab probably won’t hit Humira status, it’s still likely to be worth several billion a year by the mid-2020s.
The White House & FDA Disagree
The White House has moved against the FDA while accusing them of playing politics. The FDA withheld approval to use convalescent plasma from recovered COVID19 patients last week. The treatment was close to emergency approval, but it was placed on hold while the data are more thoroughly reviewed. Plasma from COVID survivors has only been employed in a few small studies and these presented inconclusive results. Because of the variability in the quality of plasma and the lack of antibody standardizations, therapies employing this method can vary wildly in results and without a proper control group, clear conclusions can be challenging to make.
When the FDA indicated that they wanted to wait on the scientific evidence before approving a therapy, the White House revoked FDA authority over certain kinds of lab tests. Specifically, they removed the ability of the FDA to regulate lab developed tests. In effect this allows organizations from small labs to massive organizations to peddle unapproved tests to clinicians, who can then utilize those tests and results. The FDA has approved 35 lab developed tests for emergency use testing of COVID19 since March. Despite these moves, the FDA is getting the same messages as NOAA and the EPA: The White House does not approve of regulations or science that opposes their opinion.
Sponsors
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Decentralized Clinical Trials
Decentralized clinical trial management was all the rage in 2019. It was a branding statement from emergent CROs, and a phrase bandied about at industry conferences. In 2020, it became a necessity. Historically, clinical trials are operated at sites. Staff or supporting researchers will be placed and trained at those sites and individuals will be recruited into site-specific pipelines. For rare diseases at major centers and involved therapies like those targeting cancer, this is a reasonable model. However, for many clinical trials, these pipelines are wildly inefficient. The functional result of the historic model is that sponsors and CROs spend a lot of time and energy recruiting and training various sites only to have large variability in the effectiveness of the sites in executing the trials.
On smaller trials or less involved treatments, this centralized node-model creates enormous financial and temporal waste. Hence the development of decentralized and remote trial operations. Last week Science37, a decentralized trial enabler, oversubscribed a $40 million funding round to expand the development of a global clinical trial platform. As they claim, “we have proven to enroll faster, retain patients at a higher rate, and reach a more representative population.” Regulatory shifts are rarely rapid, and decentralized trials certainly have some barriers to overcome before becoming the mainstay in clinical research. However, with COVID19 burned into the psyche of regulators, physicians, sponsors, and investors, more companies like Science37 will emerge and drag clinical trials into the 21st century.
Closing Credits
Thanks for joining me on The Niche Podcast; your weekly 5-minute summary of top news in the biotech, clinical trials, and applied science industries. You can find us on your favorite podcast app. Like, comment, subscribe, and most of all share with your friends in the industry. If you like what you hear, please rate and review, it really helps us. Once again, I’m Dr. Noah Goodson, I’ll see you next week.
Sponsors
Story References
https://www.momentapharma.com/investors-and-news/press-releases/press-releases-details/2020/Momenta-Enters-Definitive-Agreement-with-Johnson--Johnson/
https://www.hhs.gov/coronavirus/testing/recission-guidances-informal-issuances-premarket-review-lab-tests/index.html
http://www.science37.com/solutions/
Music by Luke Goodson