Software quality assurance engineers know that there is no such thing as “bug-free” software. It’s about giving a verdict on when none of the outstanding flaws count as showstoppers. Even the wondrous system that makes up the human body makes mistakes. “A genetic disorder is a disease caused in whole or in part by a change in the DNA sequence away from the normal sequence,” says the NHGRI. About 5,000 diseases are caused by mutating DNA, which could be corrected by editing the genes causing the problem. That, in a nutshell, is the gene editing investment thesis, and it’s why investors have been poring over the list of 27 gene stocks to pick the winner(s). Today we’re going to look at the 28th gene-editing stock to hit our radar — Prime Medicine (PRME) — which recently announced plans for one Iinitial ppublicly Odisguise (initial public offering).
We believe Prime Editing technology has the potential to provide lifelong healing after a single treatment.
Credit: Prime Medicine
About Prime Medicine Stock
In our recent Beam Therapeutics (BEAM) article, we talked about how Prime Medicine had raised $315 million to bring the world “Prime Editing,” a new gene editing technology with the potential to improve more than 90% of known disease-causing mutations. As always, the latest gene editing technology is always touted as the best. Prime Medicine believes that while each gene editing technology is different, the barriers that prevent one or more of the existing technologies from comprehensively addressing genetic diseases include:
- Restrictions on the types of edits they can make
- Restrictions in the cell types in which you can make changes
- Limits in the accuracy of gene correction
- Reliance on double-strand breaks
- Inability to correct the mutated gene at its physiological location
Prime comes with a portfolio of 18 therapeutic programs divided into four different buckets.
- Instant Target Ads: Deliberately chosen as potentially the fastest and most direct way to demonstrate the technological success of prime editing in patients.
- differentiation target indications: Treat the underlying cause of serious genetic diseases with therapeutics that previously could not have been developed, particularly with other gene-editing approaches.
- “Blue Sky” ads: Intended to drive new and innovative technological developments in Prime Editing and expand its application beyond rare genetic data. Lower priority.
- “March up the chromosome” is approaching: Overarching vision to correct the entire set of mutations in a given gene. This category overlaps with other strategic categories of indications.
The first category interests us the most. Prime Medicine must prove its technology platform can successfully deliver an approved therapeutic, as does any gene editing company rushing to be the first to cure a disease. In order to be seen as a platform that churns out therapeutics, it’s important to get one to market as quickly as possible. You can see their pipeline below, where we’ve highlighted two therapies the company says it’s primarily focused on, one of which is the treatment for sickle cell disease, which they’re developing with Beam Therapeutics.
As you can see above, one therapy is considered approved Iinquiring Nah DCarpet (IND) from the FDA is an important next step for each of the 18 candidates in development.
The Importance of IND Submissions
Prime Medicine’s technology undergoes quality assurance measures like any other. The S-1 filing talks about how they improved it enough to start tracking IND applications.
We have focused on developing the manufacturing processes both in-house and collaborating with suppliers to ensure the quality of the Prime Editor components required for pre-clinical studies and IND submission.
They claim to have achieved quality levels that IND submissions pass, and that’s the next milestone we’re watching for.
An IND is an FDA exemption that allows an unapproved product candidate to be shipped in interstate commerce for use in a clinical investigation study. An IND application also serves as an application for FDA approval to administer investigational drugs to humans. The FDA requires a 30-day waiting period after submission of each IND to review the IND to determine whether human subjects are being exposed to unreasonable health risks. At any time during this 30-day period, the FDA may impose a clinical lockdown or a partial clinical lockdown. In this case, the IND sponsor and the FDA must resolve any outstanding concerns before clinical trials can begin.
The first IND filing to go through the FDA gauntlet will be a vote of confidence in Prime Medicine, which will likely be accompanied by stock price swings (assuming the IPO goes through, of course). The first candidate appears to be the treatment of “chronic granulomatous disease,” according to Prime’s statement below:
Based on these results, we will select a development candidate from a selection of leading prime editors for this program and initiate IND-eligible studies.
Credit: Prime Medicine
After evaluating all the technical details of their pipeline candidates (none of which can be understood without a strong technical background, which our MBAs don’t have), the above comment seems to point to the candidate most likely to see an IND filing first.
Radiation therapy and myeloid therapeutics
Several events occurred just prior to the IPO filing date that are worth noting. The first is a patent issued just eight days ago that the company describes as a “major milestone.”
The ‘770 patent is the first issued prime editing patent in our licensed patent portfolio and we believe it will help protect our prime editing platform and pipeline of gene editing programs.
Credit: Beam Therapeutics
The S-1 filing makes it an important point to talk about how much intellectual property there is and the importance of Prime’s licensing agreements with Broad Institute and Hahvahd. “As of June 2019, it has been reported that approximately 2,072 patent families worldwide relate to CRISPR gene editing inventions and their uses,” says Prime Medicine, which has previously worked with two companies – Beam Therapeutics and Myeloid. The latter is a company that provides technology that Prime may need to extend the applicability of their technology, so Prime includes the payout:
This retrotransposon-based approach complements prime editing and, if successfully deployed alongside prime editing, could expand the applicability of our technologies towards our goal of addressing human diseases more comprehensively.
The relationship between Beam Therapeutics and Prime is complicated for a number of reasons. Prime Medicine’s co-founder — world-renowned gene-editing leader David Liu — also happens to be a co-founder of Beam Therapeutics (he also co-founded Editas Medicine). There are currently no plans for Dr. Liu to become an executive or director of Prime Medicine following this offer:
He is expected to continue to provide consulting services to us pursuant to a consulting agreement, which has a current term ending September 2025, and reflects a prior commitment with respect to Beam Therapeutics Inc. that could create or appear to be a conflict of interest. He is also expected to retain his position and affiliations with the Broad Institute, Inc., Howard Hughes Medical Institute and Harvard University.
Credit: Prime Medicine
Coincidentally, the Broad Institute and Harvard are two institutions Prime Medicine licenses intellectual property from that are doing quite well out of business. In addition to standard royalty structures, Prime Medicine has pledged to donate $5 million annually to the Broad Institute and Harvard for 14 years, with a recent change stating that David Liu, who is a Fellow of the Broad Institute and a Harvard faculty member , can use the buff in his labs. There he will likely work on the next variant of gene editing that will outperform all others.
Another event shows an attempt to create a greater distance between Beam and Prime Medicine. Beam initially named its CEO, John Evans, to Prime’s board of directors, but he only resigned from that position a few weeks ago.
Conclusion
Unless primitive man embarks on the mutually assured destruction, there will come a time when all genetic diseases will be cured with a single treatment. Unless we start gene editing at the germline, these therapies will be administered to treat all people born with harmful genetic mutations. With 5,000 diseases out there attributed to mutations, there’s plenty of room for more than one winner. Prime Medicine is behind the eighth in terms of pipeline progress but claims to have the superior technology. If their technology is that good, it means their pipeline should move forward at a decent pace with few hitches. We would like to see more clinical advances and partners before considering an investment here.
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