Life Science Today 056 – End of Season 1, Biogen, Pfizer, Eli Lilly, Intellia (CRISPR)

Originally Published as The Niche Podcast

This is the last episode of Season 1 of the Niche Podcast. In line with that, I’ve got some announcements right at the top of the episode. Also, this week, congressional brakes, internal brakes, FDA brakes, and the first human CRISPR data.

Story References
https://tinyurl.com/Niche-056-1
https://tinyurl.com/Niche-056-2
https://tinyurl.com/Niche-056-3
https://tinyurl.com/Niche-056-4

Music by Luke Goodson
https://www.soundcloud.com/lukegoodson


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 The Niche Podcast – Your weekly rundown of the biotech, pharma, clinical research, and life science industries. I’m your host, Dr. Noah Goodson. This is the last episode of Season 1 of the Niche Podcast. In line with that, I’ve got some announcements right at the top of the episode. Also, this week, congressional brakes, internal brakes, FDA brakes, and the first human CRISPR data.

Announcements at The End of Season 1

Season 1 of the Niche Podcast is coming to a close. It has been a real honor to have so many of you listen. Several years ago, I started exploring clinical research really seriously. When I did, I realized that there just weren’t that many great resources out-there. It was really challenging to learn about roles and opportunities. More important, it was hard to parse how the complex integrations of healthcare, investment, science, and clinical research coalesced into the system we have today. The reality is that so much of the information in this field is thrown out into massive dumps of marketing written briefs or communicated behind closed doors. Because most people in the know are also insiders, people in clinical research and associated industries forget pretty quickly what it’s like to be an outside. I wanted to make a show that was accessible to business folks, graduate students, clinical research coordinators, or biotech CEOs. I wanted to provide you, the intelligent folks in my audience, brief insights that can help you with your week. If you’re trying to get a job, you might know a little more about the industry. If you’re at a work meeting, you could share a tid-bit about last weeks happenings. And, if you listen over time, you’ll get a feel for what is happening in trends across industries.

In season 2, we are making some changes. First, the show will no longer be sponsored by The Scope Method. I believe in this show and want to give it to you. It was never intended as a business funnel, and I won’t ever make it that. I’ll be taking on a new role in clinical research to help make clinical trials more efficient and I’m pretty excited about the opportunity. All this means my private consulting is going on hold. If you get this show on LinkedIn, I’m sure you’ll see what I’m up to next. For you, this listener, this doesn’t change much, but now you don’t have an ad to skip. 

Third, and related to my new role, I’ll be adding a disclaimer to the show. It’ll just let you all know that what I say on here is my opinion. It doesn’t mean everyone I work for thinks the same as me.

Finally, in the coming weeks I’ll have some website updates and new mechanisms for you to send in feedback, or stories you think might be important to the show. I’ll also continue to do interviews. We’ve got two on deck right now and I’ll launch one of them as the opener to season 2 next week.

Thanks so much for listening. Especially thanks to those of you who have given me feedback. It’s a pretty incredible honor to make something that people listen to and value. Seriously, blows my mind. Now, for the last episode of season 1.

Disclaimer

The views expressed on The Niche Podcast are those of the host and guests. They do not necessarily reflect the opinions of any organizations or companies with which they are affiliated.

The Aduhelm Saga Continues, in Congress

Biogen’s potential block-buster Alzheimer’s therapy was approved early this month under inauspicious circumstances. We mentioned at the time, the complex interplay of pressures that may have driven the FDA to grant the drug the nod. This was followed by weeks of drama as some stood up in favor, while individuals on the independent advisory committee resigned in protest. This week, the waters got muddier. The House Committee on Oversight and Reform announced Friday they are launching an investigation into the processes that lead to this approval. Two questions are at stake. First, should this drug be approved in the first place? But second, why does it cost $56K? Medicare will fit an enormous portion of the cost of Aduhelm if it remains on the market. So maybe some folks don’t want to pay. It’s not unreasonable, if probably meaningless for congress to get involved. Remember the real question at hand here is not if Aduhelm reduces amyloid plaques, a key marker for Alzheimer’s disease. The question is, does it provide clinical efficacy in reducing symptoms? Expect debate for a long time on this one.


Pfizer Pauses Chantix Sales

The long-approved smoking cessation drug, Chantix, had sales paused this last week. Pfizer, the drug creator, identified increased levels of potentially carcinogenic nitrosamines in tests supplies. It’s likely Pfizer will cross check their production pipeline, identify the problem, and get Chantix back out there as quickly as possible. This was found through an internal process, and not an FDA audit, so there is limited information on what exactly was found. With Chantix coming off patent this last year, and sales dropping during the pandemic, it’s probably the first nail in the coffin of this profit stream which posted numbers in excess of $1B for many years.


The FDA Pauses Distribution of Eli Lilly COVID Antibody

Eli Lilly has been in a losing game for a while with their COVID19 antibody combo. Data seemed to suggest that other therapies had superior results. However, Lilly scraped by with tentative approval. Recently, several states have said, hold on, this might not be working that well. The FDA has now stopped all distribution, on the back of new in vitro data showing the therapy has no impact on several common emergent variants of COVID19. As a little salt in the wound, the FDA specifically recommended using the competition. Noting that both Regeneron and the GSK/Vir showed better results across the board. Lilly was on track to sell a $1-2B of this therapy in 2021. What they’ll do next is anyone’s guess. With Regeneron, specifically, on the rise with really robust data, it’s hard to imagine them having a fighting chance. They may try to wrangle some limited approvals or sell the product globally but I think this one might be headed for the scrap heap.


CRISPR Has Been Successfully Performed in Humans
In news that strikes a deep chord with me, the first ever positive in vivo human CRISPR results are in. An interim analysis (28 days) from Intellia and Regeneron shows that a single CRISPR infusion has yielded significant results in a rare liver disease. This is it, in my mind, the first real concrete data in the new era of medicine. In case you’re new here. CRISPR is a mechanisms of editing DNA. Basically a piece of molecularly machinery targets a specific sequence of DNA based on a very precise guide and makes a cut. The easiest way I can think of to explain this is, imagine you want to delete a paragraph from a book. If you knew the first sentence of the paragraph word for word and the last sentence the same, you could search for where that paragraph started and ended. With CRISPR, you can both target those two sequences, and delete the words between, but in human DNA. Just like deleting the correct paragraph from a book can change everything, deleting something broken in human DNA cause also have massive benefits in certain conditions.

Intellia has combined a couple of key elements to get to this point. First, they targeted a rare liver disease called Transthyretin Amyloidosis (ATTR). In ATTR a protein, transthyretin,  is misfolded and piles up all over the body causing massive problems. Intellia’s therapy, NTLA-2001 targets the production of that gene. Another core piece to Intellia is their pipeline. They are using a specific packaging system to ensure all the CRISPR machinery get’s delivered. This happens in a single infusion. What’s the result? After just 28 days, the treated individuals had zero serious adverse events and showed an 87% reduction in TTR serum levels in the 0.3mg/kg dose group. What does that mean? It means a single infusion may have stopped a genetic disease in it’s tracks. Possibly forever.

There is a lot to happen before this hits the market. First, this is an interim analysis of a phase 1 trial. That means any therapy is years away. Second, long term and detailed safety profiles are an absolute must to understand how effective NTLA-2001 is, and if there are additional risks in the long term. This also does not mean every genetic disease is curable. ATTR is particularly amenable to gene therapy because of the specific genetic causes of the disease. Diseases of under expression, for example, are much more complicated to treat. But, this is a huge step in the next phase of medical development. For Intellia, the successful delivery mechanism means they are building out their pipeline in attempt to deliver other CRISPR therapies. Expect more news from them in the coming months.

Whatever you think about the complications and ethics of human gene editing, I suggest you buckle up because CRISPR is here stay.


Closing Credits

Thanks for joining me on The Niche Podcast; your weekly summary of top news in the biotech, pharma clinical research, and life science industries. You can learn more at thenichepod.com or find us on your favorite podcast app. Like, comment, subscribe, and most of all share with your friends. 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.