Originally Published as The Niche Podcast
Sequencing technology, decentralized Alzheimer’s trials, busts in gene therapy and microbiomes, and a $2.4B oncology collaboration
Find out more at
https://thenichepod.com
Story References
https://tinyurl.com/Niche-060-1
https://tinyurl.com/Niche-060-2
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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.
Originally Published as The Niche Podcast
Sequencing technology, decentralized Alzheimer’s trials, busts in gene therapy and microbiomes, and a $2.4B oncology collaboration
Find out more at
https://thenichepod.com
Story References
https://tinyurl.com/Niche-060-1
https://tinyurl.com/Niche-060-2
https://tinyurl.com/Niche-060-3
https://tinyurl.com/Niche-060-4
https://tinyurl.com/Niche-060-5
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.
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 week, sequencing technology, decentralized Alzheimer’s trials, busts in gene therapy and microbiomes, and a $2.4B oncology collaboration.
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.
PacBio to Acquire Omniome – Futures in Genetic Testing and Sequencing
Genetic sequencing technology is a cornerstone of the life science industries. Not only is sequencing itself worth billions of dollars, but fundamental advancements from basic science through drug delivery rely on rapid and accurate genetic sequencing. Without digging into the minutia, suffice to say the differences in sequencing methods are meaningful and they do matter. You might think a DNA sequence is a DNA sequence, but what you want to learn can significantly inform which technology to use.
For PacBio, their Single-Molecule Real-Time (SMRT) sequencing produces relatively long reads and has specific value, especially in basic research, but hasn’t been the #1 technology with a market cap today of about $6B. It was no surprise to see market leader Illumina (market cap $72B) announce the acquisition of PacBio back in 2018. However, after a lengthy and challenging journey, the companies ultimately were not able to merge and the deal was broken off at the end of last year. This left PacBio figuring out how to capture a larger share of the market and shore up their weaknesses. Last week they announced the acquisition of Omniome in an $800M deal backed by stocks and debt. Omniome’s technology supposedly contains some polymerase driven mechanisms of exceptional sequencing accuracy. PacBio will probably marry this novel sequencing tech to their SMRT system. They have made the pivot from acquisition target to market competition.
Decentralized Trials Heading for Main Stage
Decentralized clinical trials (DCTs) are on the rise and a recent announcement from Eli Lilly suggests they’re heading for the main stage. On the back of Aduhelm’s FDA clearance, other Alzheimer’s pipelines have seen a boost across the market. For Lilly, this means their Alzheimer’s prospect donanemab is headed for phase III clinical trials soon. Almost a month ago Lilly announced the plan to file for accelerated FDA approval in line with Biogen’s success. Now they’ve announced a new clinical research partner in the form of Banner Alzheimer’s Institute. Practically this just means Lilly will be leveraging Banner’s network and probably any other recruitment strategies they can find to fill out their clinical trial population. It’s basic PR and not that interesting. However, the part of the statement that caught my eye was the intention to use a more “virtual” (read decentralized) approach to this trial. For those, like myself who are watching the clinical research industry the recruitment strategy is basically normal – it’s the attempt to incorporate decentralized components that represent the movement of decentralized clinical research to the main stage. While it should be noted that this will likely remain a hybrid trial design, it’s still a big move. DCTs are not just for rare diseases or registries, they’re for pivotal studies on potential blockbusters.
Gold Rush Means Boom Bust
Gene therapy is like a gold rush. There is real genuine gold in them hills. In this case, in the form of incredible treatments that change people’s lives forever and also potentially cost $1M bucks. But all gold rushes have boom bust cycles. In this case, the busts can come from poor strategy, poor execution, lack of clinical validation, or unforeseen safety challenges.
The last of these has occurred to Adverum’s investigational gene therapy candidate ADVM-022. Adverum has been in early phase clinical trials for both diabetic macular edema (DME) and wet age-related macular degeneration (AMD). The AMD trial has shown reasonable safety, but the DME group showed signs of toxicity including some concerning adverse events, like severe drops in intraocular pressure. Appropriate safety measures have been taken in response. Adverum has now announced they will not proceed with DME. However, a phase II AMD trial is being planned, and has FDA fast-track designation. Detangling the drivers of decreased intraocular pressure in response to a high dose gene therapy in a DME population may be too challenging. But all this should serve as a warning of the fragility of a gold-rush economy. At the end of Q1 Adverum still had $400M in assets and a burn rate of just about $30M a quarter, leaving them some room to continue exploring their options. However, with a limited opportunities in the pipeline, significant pressure will be riding on the phase II AMD trial for ADVM-022.
Microbiomes Hit a Snag
Probiotics are hot stuff. The market has 7.7% growth rate and an estimated expenditure of more than $50B per year. But clinically validated probiotic solutions that meet FDA standards have yet to materialize. There is abundant clinical evidence supporting the central importance of a proper microbiome balance in healthy skin and gut, and we know they can be rebalanced to some degree. What researchers have yet to show is that a given probiotic-like solution meets the standards for an FDA approved therapy. Seres Therapeutics has set out to deliver complex and specifically designed microbiomes to fight infectious and inflammatory diseases. They also have an early-stage pipeline focused on restoring gut balance in oncology. Unfortunately, their phase II trial for SER-287, a mixture targeting Ulcerative Colitis did not meet appropriate endpoints. It’s not a total loss for Seres, who is likely to earn an FDA approval for SER-109 to treat C. difficile in the near future. There is a chance they could be the first FDA approved “probiotic” as early as this year.
Long run, there are some exciting integrations between gut microbiome disbalance and a variety of diseases. There are still some fairly sizeable knowledge gaps in how our gut microbiome is formed, maintained, and shifts with our bodies over time. What is clear is that everything from weight to auto-immune disease could have a gut-component. We’re likely to see more creative solutions as science advances. Companies like Seres may be able to earn limited approvals for certain conditions, but I think probiotics will remain largely a supplements industry for the near future.
Pfizer Expands Massive Deal with Arvinas for Protein Degrader
Pfizer announced a $2.4B collaboration with Arvinas to see their oral oncology product ARV-471 through clinical trials for the treatment of breast cancer. The therapy targets and degrades estrogen receptors. Early trials have focused on locally advanced metastatic breast cancer. There is potential for ARV-471 to become a market leader in some of the most common breast cancers, but they’ve started with the toughest targets. Even in patients with heavy pretreatment and advanced cancers, ARV-471 showed significant promise. This has spurred Pfizer to put in $650M upfront with a $350M equity investment. This $1B front loading is followed by a generous $1.4B in milestones and 50/50 global profit sharing. This is absolutely a generous deal and suggests Pfizer things Arvinas really has something. And I guess if you can capture a large portion of the global breast cancer market in the next decade, you do.
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.