Life Science Today 089 – Intellia + Regeneron, Purdue, Amicus

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, CRISPR works, the opioid epidemic makes amends (sort of), and the SPAC that wasn’t.

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. 

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. 

Intellia + Regeneron Positive Clinical

CRISPR works! I know… as a person who literally earned their PhD on the back of CRISPR technology, you’d think I’d know that by now. But it works in humans… which… actually now that I say it and think about biochemistry… pretty obvious. Ok let’s try again. New, interim data from a clinical trial suggests that CRISPR technology may result in sustained functional changes in gene expression. Data, from Intellia released Monday, show that when treated with a CRISPR therapy, people with Transthyretin Amyloidosis experienced “rapid, deep, and sustained reduction in disease-causing protein.” This includes an 86-93% reduction in protein expression at 28 days after a single dose, sustained through 12 months of follow-up. There are tons of fascinating bits from this study. First were the levels of dose escalation which basically suggest if they were dosed higher, it was more effective. Second was the incredibly low adverse events. I mean for a literally life changing gene-therapy only a single serious adverse event was reported related to vomiting. Not to downplay this event but for therapies on this scale that is practically negligible. Of course, it is important to remember this is a smaller phase I clinical trial that is primarily powered to ask if this is safe and effective from objective outcomes like changes in blood protein levels, not more clinical outcomes related to the participants experience. Other data have shown you can at times change the expression of a protein without modifying some of the experience of a disease. But, for now this is super promising data. 

In very bad news for Intellia, an important court decision calls into question patents that may relate to this very trial. But, we’ll have more on that next week. 

Embattled Purdue Earns FDA Approval

Purdue Pharma has not been popular recently. Or rather, they have been all-to-popular for many of the wrong reasons. The OxyContin producer has received significant criticism for their role in the opioid epidemic that has impacted so many globally. Last fall the company received approval to transition into a public benefit corporation focused on addressing the opioid crisis. On the one hand this is a sensible re-envisioning with a nice redemptive arch. On the other hand, it’s a clear mechanism to shield those who profited from OxyContin from some of the potential ongoing legal ramifications.

This week, Purdue received FDA approval to treat unknown or suspect opioid overdose with an injection of Nalmefene. The treatment should be used in conjunction with other emergency care for potential overdoses. While it will hopefully save some lives, it’s hard to argue that Purdue has outrun their Karma just yet.

The SPAC that Wasn’t

This should not come as a big shock to my listeners but some view this as an unfavorable time to go public. This does not upend the IPO plans of all companies, but those looking for a mega raise without some startling results to drive the capital are in for some challenging times. That is the case for Amicus Therapeutics, who was heading towards spinning off part of their pipeline through a SPAC, valued at $600M later this year. Now the deal has fell through leaving them short on a next steps trajectory and mega short on cash. Of course, this isn’t just that market conditions are challenging, Amicus revealed some less-than-promising long-term data for Batten disease. They used words like “streamlining portfolio” read, were cutting jobs and only keeping the top therapeutic options. Amicus is not completely lost in the woods; they still have an approved therapy for Fabry disease and a treatment for Pompe disease in review at the FDA and in Europe. They may just need to bide their time and weather the storm till they can re-expand their pipeline and launch back out when market conditions are more favorable. 

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. 

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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https://LifeScienceTodayPodcast.com

Story References
Intellia
Purdue Pharma
Amicus SPAC

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.