Life Science Today 088 – Eli Lilly + Innovent Biologics, Agios, TC Biopharm, Blue Water Vaccines, Meihua

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, the PD1 that lost, biopharma earns FDA approval, and the Biotech market is cool not dead.
 

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. 

Inclusion, Representation, and Cost

Two weeks ago FDA advisors voted 14 to 1 against approval of the PD-1/PD-L1 oncology immunotherapy sintilimab to treat non-squamous non-small cell lung cancer (NSCLC). Lilly, partnered the Chinese drug creator Innovent Biologics to bring sintilimab to the US market for this indication. They had been cognizant of potential challenges around earning a first-line PD-1 FDA approval. Their strategy was to approach the approval by offering data out of a clinical trial from China and saying they were willing to compete pretty heavily on price in a fairly expensive PD1 inhibitor market. The panel is saying no to this for a number of reasons. First, the study was conducted in mainland China with a large number of the participants being Chinese men. The panel said hey, that doesn’t really represent the generalized US population with NSCLC. And they’re right. They also had some real concerns about the selected endpoints. The ORIENT-11 study choose progression-free survival (PFS) as the primary endpoint and overall survival (OS) as the secondary endpoint. The terms may sound similar but PFS measures how long you are on a treatment before a cancer starts to grow, where as OS measures actual human survival. PFS is a great early-stage indicator of potential success, but In general other oncology immunotherapies have relied on OS. 

Lilly recognized that perhaps the trial did not have the same strength of data as existing therapies and offered an approval pipeline focused on driving down prices through undercutting current sky high therapy costs. In fact, the one FDA board member who did thumbs up the treatment pointed to costs as a good reason. And all this is underpinned by the fact that sintilimab does have reasonable good outcomes data published and is approved to treat NSCLC in China. So this is not some random new drug with sketchy data we are talking about. 

The panel’s rejection raises that possibility that Lilly will be forced to complete another Phase III trial in the US. That brings with it the challenges of gaining a participate population against approved drugs for the same indication. That could make it challenging to complete the study because of barriers to successful recruitment. Two or three more years down the road they may still lack an approval. If they gain it, they are less likely to want the “cost savings” approach. Now there are probably some bridge approaches here using smaller trials, RWD and second line treatment approvals but this presents several core conundrums of our industry:

First is the question of inclusion. It’s easy to see that the population of China doesn’t generalize to the United States, and yet with the US and EU leading trials for so many years, the reverse conclusion that drugs approved here are probably fine in other places has happened again and again. The FDA is trying to change this in some ways. And the industry is slowly becoming more truly global. This push back against a trial conducted only in China is part of this push for inclusion. But with extremely expensive options on the market, would things improve if another option were available at a fraction of the cost? Do longer more expensive trials bring better drugs to market or repress the market by limiting options and allowing only the most expensive solutions to emerge? With the US leading the way economically in not pushing for more inclusion for years it’s exciting to see that happening. But I can’t help thinking it would be nice if we were doing a better job at it all along. 

Finally, like all FDA moves that slide conservative, if the votes of the panel results in a full FDA rejection, the decision is likely to have far reaching consequences. Yes in the circumstances it’s not a crazy or wildly surprising choice for the panel to thumbs down sintilimab. But expect other risk-adverse organizations to shy away from more cost-efficient trials in emerging regions in favor of more expensive approval routes. This also signals that you can’t save a little on trials and then pass on savings by undercutting markets. Who doesn’t want the very best most expensive most well tested therapy. But at what cost? 

The core takeaway here should perhaps be that there will not be a change in the status quo around oncology at the FDA. Something that others have noted in a broader context with the appointment of Robert Califf as commissioner. So sintilimab is facing an uphill battle for approval. Lilly clearly suspected this was the case based on the strategy but the process itself is taking a big highlighter pin to the challenges and tensions that currently surround drug approval in the United States. 

Agios Earns first FDA Approval

Agios Pharmaceuticals has been granted FDA approval to treat adults with Pyruvate Kinase Deficiency (PKD). The therapy mitapivat is being sold as PYRUKYND. This first approved disease-modifying therapy works to combat the hemolytic anemia caused by PKD. The oral therapy works to directly activate the defective PK enzymes and stop the degradation of red blood cells that is a hallmark of the disease. For those living with PKD this therapy represents a major opportunity for improved quality of life and decreased transfusions. This is not the end of the pipeline for mitapivat. There are ongoing studies across PK deficiency, Thalassemia, and Sickle Cell disease. As Agios first approval this is a big move from clinical to commercial stage company with a robust pipeline of potential. 

Three Biotech’s heading for modest IPOs

While cooled from 2020, the biotech market is not dead yet. Last week three biotech’s announced modest IPOs. TC Biopharm announced the closing of a $17.5M IPO. The funds will be used to advance pivotal trials for acute myeloid leukemia, and a second late-stage treatment for COVID19 injections. They also have a pipeline of CAR therapies focused on solid tumors, but I doubt this raise will be enough to get all of these across the line. Unfortunately the stock dropped more than 40% from it’s closing costs to just $1.43 a share at recording. 

Blue Water Vaccines also announced plans to go public and raise $20M for their universal flu vaccine. Originally priced at $9/share, Blue Water skyrocketed to over $57/share at closing Friday, marking a pretty startling growth curve in current markets. Though perhaps excitement will be dampened when US markets re-open Tuesday this investor enthusiasm for a vaccine maker is pretty shocking. 

Finally, Meihua International Medical Technologies raised $36M to increase their capacity to manufacture medical devices through opening a new facility and pursuing new R&D activities. The Chinese based medical device as a range of Class I, II, and III consumables. This probably represents a perfect company to go public right now since their value hangs on production of consumable on-demand supplies like disposable catheters and not from a high-risk pipeline. While initially up, shares settled down around $8.16 by the end of the week. 

So there are no crazy $200M IPOs with billion dollar valuations. But there are still companies with both risky and stable pipelines entering the market and keeping us on our toes. 

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.

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Story References
Eli Lilly + Innovent Biologics
Agios
TC Biopharm
Blue Water Vaccines
Meihua


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.