Bluebird, FDA, Resilience, Corvia, Code, Mineralys, Tessa

Gene therapy, minor legislation, a $625M series D, and four companies raise $373M.

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Story References
BlueBird
FDA
Resilience
Corvia, Code, Mineralys, Tessa

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, gene therapy, minor legislation, a $625M series D, and four companies raise $373M.

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. 

Big move for BlueBird Bio

BlueBird Bio has received good news. The beleaguered biotech company has watched their stocks fall over the last few years as they pushed the boundaries on the development gene therapy treatments, but faced numerous challenges and setbacks. Despite their success in obtaining an approval in Europe for their gene therapy Zynteglo, sales never materialized as EU agencies failed to come to pricing agreements for the $1.8M therapy. In July 2021, BlueBird received further EU approval for Skysona as a second gene therapy, but in October last year they decided to fully wind down European operations. Then, they spun off their entire oncology wing into the newly minted 2seventy Bio. All these of these forward and backward steps have shown that markets are not always gentle to companies pushing forward the world of gene-therapy. And it’s hard to know what to say. Sure, $1.8M is a lot for a therapy, but it’s always one time for people who will die without it. It’s also notable that BlueBurd invested billions over decades to see these therapies become a reality. So what kind of payout does this investment merit? 

Anticipating unknown consequences last week, they placed their stocks on hold pending the release of a vote from the FDA on their gene therapy treatment. The Cellular, Tissue, and Gene Therapies Advisory Committee of the FDA voted 15/0 that the benefits of eli-cel (Skysona in Europe ) outweigh the risks to treat people with cerebral adrenoleukodystrophy (CALD). This is not yet an FDA approval, nor does it fully rescue BlueBird and their entire pipeline, but it is a strong endorsement. There are certainly some risks associated with the therapy, but lack of treatment is a known death sentence and patient-advocacy groups played a role in pleading for the FDA. How the US healthcare system will respond to a nearly $2M therapy is hard to predict. But markets were optimistic with stocks risking in contrast to a plummeting market Monday.  

Accelerated Approvals May Change

The US House has passed bill H.R. 7667 which supports the FDA in gathering fees from users to support their work in evaluating drug safety. For the most part, the bill maintains the status quo, however there are some notable sections. One of the changes in the bill is to push for increased accountability around the process of validating accelerated approvals. Currently, companies who receive an accelerated approval are often required to show ongoing clinical efficacy. However, they may lack temporal motivation to complete the required studies in reasonable times. Under the new bill certain provisions may increase the pressure on companies who receive accelerated approval to generate proof of clinical efficacy by having trials underway before approval is granted. While this may disadvantage certain organizations, it really serves to allow a net increase in rapid approvals which can be removed quickly if they lack safety and efficacy. In a world of increasing clinical timelines and cost, anything that truncates delays may provide value. 

An additional area of note is sections 501-505 which have reasonable and flexible requirements for a diversity plan in phase III or pivotal studies as well is in advanced device studies . While a small move to drive health-equity this bill  backs recent FDA draft guidance with legislative requirements. It is unlikely the bipartisan bill will see significant change in the senate. 

Resilience Big $625M Series D

The biomanufacturing firm Resilience has announced a $625M series D. Resilience has been on a fundraising tear bringing in $600M less than a year ago. With all this capital, they are build ing out infrastructure, purchasing manufacturing facilities, and making acquisitions. The capital investments and efficiencies required for advanced bioprocess manufacturing continue to place pipeline strains across the biotech space. The pandemic and geopolitical pressures together are likely to increase these pressures in the near term for companies to develop in regional proximity, and there remain a wide range of biotech organizations that will need long-term development and manufacturing partners. There are certainly pressures in every industry, but for a company like Resilience I suspect a key discipline is remaining focused on efficient institution building and targeted customer segmentation more than a lack of market demand. In other words, there is a plenty of business, the question is, how well do you execute? In other market times, I’d expect a move to public in 6 months, but in these bear times it’s hard to say where Resilience will head. Now with over $2B raised since 2020 they certainly haven’t slowed down with other portions of the Market. 

Four Companies Raise $373M

Four early phase organizations raised $373M this week.

First, Corvia Medical has raised a $54M internal investment from their existing investors. The funds will be used for a confirmatory trial on their Atrial Shunt. With a target market of millions, their latest trial in 626 participants showed a 55% improvements in quality of life across and 45% reduction in heart failure incidents in those with the shunt compared to shame controls. This next round of funding should see them through research to commercialization and, I suspect a hopeful sale to a large medical device company.

Code Therapeutics has raised a $75M series A to develop targeted non-viral gene therapy technologies and specifically move toward IND their leading assets. Like many gene therapy biotech’s, Code combines a platform technology approach with niche rare-disease targets. Key to their approach is an attempt to circumvent some of the ongoing immune challenges associated with viral delivery mechanisms.

Mineralys has raised $118M series B to advance a clinical stage small molecule inhibitor of aldosterone synthase to treat uncontrolled hypertension. Their phase I data showed a strong safety profile. The company is now in a place where meaningful phase II data could skyrocket or scuttle their approach. Unlike some other biotech’s this is really a one-trick pony. They have a therapy, it will work or it won’t. They are now positioned to ask the question.

Finally, Tessa Therapeutics raised a $126M Series A to advance their clinical stage oncology cell-therapies. Their two CAR-T programs are directed at lymphomas with promising early clinical data including a 57% complete response in one indication. They hope to advance to pivotal studies later this year and will be manufacturing the therapies at their own facility located in Singapore.

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.