Biotech Values

[mouton29]

Verve Therapeutics (VERV) - I am starting to look at this, it is another gene editing company, in fact licensing from BEAM, among others. Its first drug is in phase 1 in the UK and New Zealand and is designed to make a single A to G change in the PCSK9 gene in the liver, downregulating PCSK9 and thereby upregulating LDLR, which clears LDL in the liver, essentially what a PCSK9 inhibitor does via an MAB but in Verve’s case via a genetic manipulation. People with heterozygous familial hypercholesterolemia have one of three types of mutuations, most commonly their production of LDLR is decreased. Preclinical results were encouraging; this article in the MIT Technology Review discusses it. https://www.technologyreview.com/2023/01/19/1067074/next-for-crispr/

Other details here https://www.vervetx.com/pipeline/

Stock is down 75% from highs, at least partly in reaction to a clinical hold from the FDA, discussed in an 8-K:

On December 2, 2022, Verve received a clinical hold letter from the FDA that outlined the information required to resolve the clinical hold, including additional preclinical data relating to: (i) potency differences between human and non-human cells, (ii) risks of germline editing, and (iii) off-target analyses in non-hepatocyte cell types. The FDA also requested available clinical data from the ongoing heart-1 trial. In addition, the FDA has requested that Verve modify the trial protocol in the United States to incorporate additional contraceptive measures and to increase the length of the staggering interval between dosing of participants. Verve intends to submit a response as expeditiously as possible.
Verve continues to enroll patients in the heart-1 clinical trial in New Zealand and the United Kingdom and plans to report initial safety and pharmacodynamic data from the dose-escalation portion of the heart-1 trial in the second half of 2023.

https://www.sec.gov/ix?doc=/Archives/edgar/data/1840574/000119312522297968/d422129d8k.htm