Novo + Forma, Sanofi, Boehringer Ingelheim, Arcturus

A $1.1B acquisition, two FDA approvals, and grants for mRNA vaccines. 

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Story References
Novo + Forma
Sanofi
Boehringer Ingelheim
Arcturus


About the Show
Life Science Today is your source for stories, insights, and trends across the life science industry. Expect weekly highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It’s news, with a dash of perspective, focused on the life science industry. 

Transcript

Introduction

Welcome to Life Science Today, your source for stories, insights, and trends across the life science industry. I’m your host, Dr. Noah Goodson. This week, a $1.1B acquisition, two FDA approvals, and grants for mRNA vaccines.

Disclaimer 

The views expressed on Life Science Today are those of the host and guests. They do not necessarily reflect the opinions of any organizations with which they are affiliated. 

Novo Nordisk Acquires Forma Therapeutics for $1.1B

Novo Nordisk announced the $1.1B acquisition of Forma Therapeutics last week. The deal brings Forma’s full pipeline into Novo with a key focus on their lead candidate Etavopivat, which is entering late-phase studies for Sickle Cell Disease along with earlier clinical work focused on a range of rare hematologies. Forma as has three additional pre-clinical molecules and four early clinical stage candidates. Just over a month ago Forma announced a post-NDA partnership with Rigel Therapeutics to develop, commercialize, and manufacture their oncology therapy olutasidenib. The deal was much smaller with just $2M upfront but with more than $215M for future milestone payments. 

The choice, by Novo, to move toward Sickle Cell targets is interesting. On the one hand, they have clear experience in Hemophilia and a late-stage pipeline aligned with blood disorders. They also have a very early-stage Sickle Cell candidate. But the Sickle Cell is fairly crowded right now with a wide range of therapeutics and even gene therapies targeting the condition. But Novo seems to think there is room for a range of therapies to target this condition. With an investment of $1.1B, I suspect Novo sees more than a single therapeutic opportunity here. Instead, I think they flagged Forma as a relatively safe bet, after all they have clinical partnerships for two of their candidates. The Forma pipeline pushes Novo further into rare hematology’s. It’s not clear if Novo will pass off, sell, or continue to develop the early clinical oncology therapies from inside Forma. If Novo wants to win in the Sickle Cell space, they will need to be quick on the draw as a number of other companies are moving in, for example Pfizer’s $5.4B acquisition of Cerevel we covered just a few weeks ago.

Sanofi Ultra-Rare FDA Approval

Sanofi received FDA approval to treat adults and children with the rare genetic disease acid sphingomyelinase deficiency (ASMD) with Xenpozyme. This is the first and only treatment approved that is specific to ASMD. However, with just 120 patients across the entire US this is truly an ultra-rare disease. Like so many ultra-rare diseases, the clinical development was years in the making, taking more than a decade to launch and eventually enroll sufficient patients within a clinical trial to validate any kind of meaningful therapeutic value, along with the safety of the medication. The enzyme replacement therapy should hit the market later this year and will hopefully extend life and quality of life for those suffering from ASMD. 

Boehringer Ingelheim FDA Approval

In a second rare disease approval, Boehringer Ingelheim has gotten the greenlight from the FDA to treat generalized pustular psoriasis (GPP) flares with Spevigo. During the clinical trial after a single intravenous dose, 54% of participants showed no pustules compared to just 6% in the placebo control. GPP impacts approximately 1/10,000 people making it rare, but impacts a far larger number than ultra-rare diseases like ASMD before. GPP is a severe condition that is not only painful, but can be life threatening making this approval critical for those impacted by the condition. Spevigo works on GPP by blocking interleukin-36, which plays a key role in auto-inflammatory conditions. This marks Boehringer’s first approved medication in a dermatological condition, though BI classifies it as an immunological product. Whether they will look to expand their dermatology pipeline in future is yet to be seen.   

Arcturus $63M BARDA Award

Long before the COVID19 pandemic, governments, and particularly the US government made investments into the pharmaceuticals industry to directly prepare for potential infectious biological events, for example stock-piling small-pox vaccines. This investment saw significant expansion during the pandemic for vaccines. From an economics perspective, the investment into COVID vaccines and therapeutics are relatively small compared to the larger cost of COVID response and many economic models argue they are disproportionately effective in their ROI in retaining a  healthy and productive population. While this may be up for debate, what isn’t is that governments will continue to invest in vaccines for the future.

While we have moved into a new phase where the pandemic is steadily becoming endemic, preparedness for the next global event is underway. In line with this, the biotechnology company Arcturus Therapeutics has received a $63M grant from the US government through BARDA to develop a pre-clinical mRNA vaccine platform, specifically focused on influenza and ultimately take this product through phase I clinical trials across the three years of the grant. The goal here is not just to develop a novel influenza vaccine, though that is part of the process, but to prepare a mRNA platform for rapid development and scalability in response to potential future pandemics. The award represents a significant fiscal win for the biotechnology company looking to raise capital in a tight market. It is likely funds will continue to flow to similar organizations in the next several years as pandemic preparedness remains a high priority target for national defense, as well as scientific and pharmaceutical development. 

Closing Credits

Thanks for joining me for Life Science Today, your source for stories, insights, and trends across the life science industry. Learn more at LifeScienceTodayPodcast.com. If you like what you hear, please tell a friend. Once again, I’m Dr. Noah Goodson, I’ll see you next week. 

About the Show

Life Science Today is your source for stories, insights, and trends across the life science industry. Expect weekly highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It’s news, with a dash of perspective, focused on the life science industry.