Life Science Today

Life Science Today 068 – Eli Lilly + ProQR, Sanofi + Kadmon, Lycia, BlueBird + 2seventy, Mammoth

September 14, 2021 Noah Goodson, PhD Season 2 Episode 68
Life Science Today
Life Science Today 068 – Eli Lilly + ProQR, Sanofi + Kadmon, Lycia, BlueBird + 2seventy, Mammoth
Show Notes Transcript

Originally Published as The Niche Podcast

$1.5B deal, $1.9B merger, protein degraders get backing, an old bird has new tricks, and another billion-dollar valuation for CRISPR

Find out more at
https://thenichepod.com

Story References
https://tinyurl.com/Niche-068-1
https://tinyurl.com/Niche-068-2
https://tinyurl.com/Niche-068-3
https://tinyurl.com/Niche-068-4
https://tinyurl.com/Niche-068-5
https://tinyurl.com/Niche-068-6

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.

 

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, $1.5B deal, $1.9B merger, protein degraders get backing, an old bird has new tricks, and another billion-dollar valuation for CRISPR


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.
 

Note to Listeners

Hi folks, sorry for the delay in this episode. I went on a vacation for the first time since 2018. If you haven’t tried it, I’ve got to say they come highly recommended. I love my work and it is exciting to keep up with what is happening in the industry, but a week staring at the ocean was absolutely the kind of refreshment I needed. If you didn’t get a chance to listen, I posted an interview with Sam Searcy and I definitely recommend you check it out! He is a businessmen, entrepreneur and former North Carolina State Senator. Now, I’m happy to be back to our normal schedule.


Eli Lilly Makes $1.3B Deal with ProQR

Get ready to keep hearing about RNA-based innovations for the next decade. Of course we are all familiar with RNA vaccines at this point, but there is a ton of other gene-therapy-like technologies sequestered in think-tanks and universities that are only now beginning to emerge into potential therapies.

ProQR has a Axiomer RNA editing pipeline that allows for single nucleotides of RNA to be edited by coopting cells natural RNA editing abilities. Adenosine denaminse action on RNA (ADAR) is present in human cells and can convert adenosine (A’s) to the oligonucleotide inosine (I). When RNA is being translated to a protein the “I” is read by the cell as a guanosine (G). This A-to-I technology therefore functions as an A-to-G mechanism. The real power of ProQR’s Axiomer technology is that it uses a manufactured Editing Oligonucleotide to attract the endogenous ADAR technology. This is basically targeted A-to-I technology, allowing for specific edits in 1000s of diseases where single nucleotide polymorphisms are a problem. Unlike “Gene therapy”, this acts on the RNA, meaning it can be dosed, controlled, and is likely reversible.

According to ProQR there are more than 20,000 conditions where a single A-to-I edit could provide a treatment. This therapeutic panacea may not be quick to market, but it does present significant possibilities for extremely rare diseases to be treated through a customizable pipeline. I’m not sure regulatory agencies will be prepared for that level of customization in this decade. Instead, ProQR will pick top targets and generate therapies for them to substantiate the effectiveness of ADAR-based technologies in treating single-nucleotide polymorphism diseases. I think one of the promising aspects of ProQRs approach is that it largely coopts an existing biological mechanism. While there are potential issues with this, for example changes in cellularly regulation of ADAR over time in the treated, there is also major potential advantages by only plugging in a single piece to a larger cellular process to drive therapeutic modifications. This might seem like basic small molecule medicine that has been around for a long time, but in the genetic diseases space, therapy is rarely this straightforward. ProQR has chosen to approach diseases of the eye with their lead candidate treated CEP290 Leber’s congenital amaurosis. I am hugely biased based on my PhD, but the eye represents and incredible venue for many of these therapies. There are automatic lateral controls (you have two eyes), it’s easy to inject therapies intra-ocularly, and it’s by-in-large an immune privileged tissue. I’m personally excited to see the results from ProQRs current phase II/III study, as well as their broad pipeline.

Eli Lilly clearly sees similar potential. That’s why they’ve signed a deal giving ProQR $50M upfront with milestones up to $1.25B and royalties after commercialization. ProQR may be early in the process, but this kind of backing provides the security they need for commercial development.


Sanofi $1.9B Merger with Kadmon Holdings

Sanofi has announced the $1.9B acquisition of Kadmon Holdings. Kadmon’s primary asset REZUROCK, which has been approved by the FDA to treat adults and children 12+ with chronic Graft-Versus-Host-Disease (cGVDH). cGVDH impacts a high percentage of hematopoietic cell transplant when immune cells attack the grafted cells. There are approximately 5000 new patients with the condition each year and REZUROCK suppresses this autoimmune response and attenuates the fibrotic disease process. For Sanofi this expands their portfolio in the general medicine immune suppression space. REZUROCK is currently in additional clinical studies for the treatment of diffused cutaneous systemic sclerosis. The deal is pending shareholder approval and due process.


Lycia Therapeutics Raises $70M Series B + Eli Lilly Partnership

Lycia Therapeutics has closed a $70M series B. This raise comes close on the heals of the partnership with Eli Lilly Announced in August to develop targeted protein degradation that gave an initial $35M investment. Lycia’s platform is based on the degradation of extracellular proteins, including cell-surface signaling molecules and secreted proteins. Currently, there is little information about their targets of choice. With this funding round, the expectation is that they develop a clear pipeline for commercialization across the next year before garnering additional funding for clinical trials. After an individual pipeline investment and participation in the series B, Eli Lilly will probably remain a major stakeholder. Now the question is, what proteins are they hoping to degrade? 


BlueBird Raises $75M for 2seventy Spinoff

BlueBird Bio has been through a lot over the last few years. Once a rising star in the gene therapy space, their shares have since fallen from a peak of $231 in 2018 to just of $18 today. This rise and fall have been driven by challenges to gain approval, delays in approval by the FDA in some of their lead products, and most recently a rapid backpedal from European markets, immediately after their therapy eli-cel was approved. Without getting into details they are largely in a slow spin trying to gain approval and generate cash-flow again.

In the midst of this, they’ve determined to spinoff a company, 2seventy, to cover their growing oncology portfolio. With BlueBird failing to take flight and a ripe oncology pipeline, this is probably a mechanism of protecting the eggs in their portfolio through a novel company to ensure investment growth and shave off some of the R&D costs currently dragging BlueBird down. While there may be issues of corporate governance or strategy, I think this is more likely a cautionary tale for investors. Gene therapy is incredibly complex and a largely unknown space. There are huge challenges ahead that we as a scientific community simply do not know about because we haven’t hit the brick wall of biological implications yet. I’m not sure about the fate of BlueBird, but I do expect additional fits-and-starts across the industry as solutions run into biology. If successful in the spin-off, 2seventy will probably try to gain wider industry partners to fund the pipeline though commercialization. However, I can’t imagine BlueBird Bio investors feeling super keen on 2seventy flying to coop only to become wildly successful.

Mammoth Biosciences Raises $150M Series D at $1B Valuation

The CRISPR technology creator Mammoth Biosciences has raised a $150M series D with a $1B valuation. The company has been hard at work producing novel CRISPRs for a vast range of diagnostic and therapeutic applications. They now have Cas12, Cas13, Cas14, and Casɸ (which I think is pronounces CasPhi). Both Cas14 and Casɸ are tiny, presenting numerous additional applications and delivery mechanisms. How Mammoth will deploy or license these technologies is yet to be determined. Like other companies positioned in the bioscience space, they appear open to application far beyond healthcare, including agriculture and according to their website “biothreats.”

Innovative specialized gene editing solutions will continue to expand across industries in the coming decade, and Mammoth wants to help lead that charge.


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.