Last Updated 3/2/2023
Verve is one of the two companies working with Base Editing. They licensed the technology from Beam Therapeutics. They have the right to pick several gene targets for cardiovascular diseases. They have picked PCSK9 and ANGPTL3 so far, and have a deal to pick more. They have a license for CRISPR CAS12 which they license from Broad Institute and Harvard. Verve has a development platform for guided LNP delivery. They have a delivery technology for their liver targets which is designed to target GalNac. Beam licenses this guided LNP from them for some of its liver programs. They are also working on their own gene editing platform.
I haven't been following Verve or Sekar Kathiresan all that long. I do think he is a good CEO, but I think he overestimates the potential for these therapies to take market share for existing therapies. They are just starting in the clinic so we will have to wait to see the data. I do think they are doing a good job of managing the balance sheet. They seem to focus on innovation with a new gene editor in the works.
VERV-101 is a gene editing therapy that knocks out the PCSK9 gene. This is a gene associated with cardiovascular disease. They have some promising early data for the knock down of LDL. The early data in the preclinic showed about 67% LDL-C reduction. We will have to wait and see how that translates into humans with the phase 1 data. We should see some data in this program later this year.
VERV-201 is a gene editing therapy that knocks out the ANGPTL3 gene. This gene plays a role in triglyceride metabolism. The animal data here showed about a 96% reduction in the ANGPTL3. They are working on the IND enabling studies and hope to have the IND in 2024.
Lp(a) is a target for early lipoprotein which is still in early discovery.
Cash $554 million
VERV-101 I think this will be a very competitive space competing with other PCSK9 antibodies and RNAi therapies. I think they will get only a small market share of the patient is HeFH. I would estimate about 20,000 patients a year at a price of $40,000. That comes out to about $800 million in sales. I would give them a .1 multiplier for being in early phase 1. That comes to $80 million value.
VERV-201 I think this program will compete with other very competitive cholesterol therapies. It is being tested in 2 different cholesterol diseases with one of them being HoFH. I think it could manage about 20,000 patients a year at a price of $40,000. That would make this program about $800 million in sales. I would give them .1 multiplier for the preclinical stage of development. That comes out to another $80 million value.
All in, that is a $714 million market cap. Based on the 61.8 million shares outstanding, that comes to $11.55.
Beam license is for 3 targets right now. The first 2 are PCSK9 and ANGPTL3, of which Beam has the option to opt into these programs and participate in 50% of the US sales. For the 3rd gene target, Beam has the right to opt into this program for 35% of the world wide sales. Any areas that are not affected under these opt in rights, will receive royalties in the low to mid single digits.
Vertex collaboration for 4 years to develop gene editing therapies toward an undisclosed liver target. They can get up to $340 million per program along with tiered single digit royalties.
VERV-101 Initial Data in 2H 2023 outside US
VERV-101 On Clinical Hold in US
VER-201 Trial Initiation Expected in 2024
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* These are my Opinions and Estimates. They should not be considered financial advice.