{"id":84363,"date":"2023-11-14T09:15:10","date_gmt":"2023-11-14T14:15:10","guid":{"rendered":"https:\/\/ifintechworld.com\/investing\/heart-disease-breakthrough-by-gene-editor-verve-knocks-its-stock-for-a-loop\/"},"modified":"2023-11-14T09:15:11","modified_gmt":"2023-11-14T14:15:11","slug":"heart-disease-breakthrough-by-gene-editor-verve-knocks-its-stock-for-a-loop","status":"publish","type":"post","link":"https:\/\/ifintechworld.com\/?p=84363","title":{"rendered":"Heart Disease Breakthrough by Gene-Editor Verve Knocks Its Stock for a Loop"},"content":{"rendered":"
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The medical breakthrough announced Sunday by
\n Verve Therapeutics
\n seemed like bullish news. In the first human test of a genetic technology called \u201cbase-editing\u201d, Verve\u2019s one-time treatment durably reduced patients\u2019 high cholesterol. <\/p>\n

In Monday\u2019s market, however, the stock (ticker: VERV) sank 41%, to $9.29. It wasn\u2019t the reaction that chief executive Sekar Kathiresan had wanted. But it has been an unfriendly stock market for biotechs.<\/p>\n

\n

\u201cAny data released in this market is going to make a stock go down,\u201d Kathiresan told Barron\u2019s<\/em>. \u201cThere is no room for nuance or taking a look at details.\u201d<\/p>\n

The stock\u2019s drop was probably the wrong interpretation of Verve\u2019s news.<\/p>\n

Data that Verve presented Sunday\u2014at the American Heart Association\u2019s annual science sessions\u2014showed that a single infusion of Verve\u2019s genetic treatment led to drops of 40% to 55% in the \u201cbad\u201d LDL cholesterol of patients with inherited high levels of the stuff that can kill them at an early age. Verve\u2019s therapy uses the Nobel Prize-winning technology called Crispr to zero in on the DNA defect that leaves certain families with cholesterol that is four-times the healthy level. Then Verve\u2019s treatment inactivates the defective gene, by using base editing to gently swap one letter in the gene\u2019s sequence for another.<\/p>\n

In 10 patients with the cholesterol disorder, the Verve-101 treatment cut LDL cholesterol levels by up to 55%. That\u2019s on a par with Leqvio from
\n Novartis
\n (NVS), Repatha from
\n Amgen
\n (AMGN), and Praluent from
\n Regeneron Pharmaceuticals
\n (REGN)\u2014approved drug treatments that patients must take their whole lives.<\/p>\n

Monday\u2019s stock drop was probably a response to the heart attacks suffered by two of the Verve-101 test subjects. One was fatal.<\/p>\n

But Kathiresan notes that regulators restricted this first-in-humans study to older patients with very advanced heart disease. The patients were already at high risk for cardiac events. The other safety measures in the study were satisfactory.<\/p>\n

The U.S. Food and Drug Administration reviewed the safety data and cleared Verve to expand its Phase 1 with five more patients. The trial\u2019s results were also reviewed by
\n Eli Lilly
\n (LLY) before its October decision to buy an option to partner on the Verve treatment.<\/p>\n

Lilly will decide whether to team up on the cholesterol treatment next year, after seeing results from the completed Phase 1 and that of another Verve treatment that will use a different vehicle to get the base-editor to the relevant liver cells.<\/p>\n

Lilly has also optioned a third program in which Verve will target a second genetic defect, and could reduce LDL cholesterol by an additional 50% in high-cholesterol families.<\/p>\n

Verve\u2019s ultimate ambition is a one-and-done treatment to lower cholesterol in the 50 million adults who are at-risk for cardiovascular disease. Despite the availability of statins and new treatments like Leqvio, only a small portion of patients reduce their lifetime cholesterol to healthy levels\u2014because of cost, poor adherence to the chronic treatments, or inconsistent access to healthcare.<\/p>\n

\u201cThose agents are theoretically able to lower LDL,\u201d said Kathiresan. \u201cBut in practice the actual effect is zero, because very few patients are actually on those treatments.\u201d<\/p>\n

Now that Verve has shown an effect and acceptable safety for its base-editing treatment, Kathiresan said that regulators will allow its studies to enroll patients who are less sick and prone to cardiac events. A true test of the treatment\u2019s effect on heart attacks must await the placebo-controlled trial scheduled for 2025.<\/p>\n

William Blair analyst Myles Minter called the Sunday results from Verve \u201ctrailblazing\u201d and wasn\u2019t concerned about the two cardiac events. He reiterated his Buy rating. So did Seamus Fernandez at Guggenheim, who\u2019s got a $55 price target on Verve stock.<\/p>\n

Verve\u2019s Phase 1 study was the first human trial of base-editing\u2014a variant of Crispr editing that was developed in the lab of Harvard chemist David Liu, and licensed for other illnesses to
\n Beam Therapeutics
\n (BEAM).<\/p>\n

So the Verve results also answer the question of whether base-editing can produce treatments. \u201cIt works!\u201d said Kathiresan.<\/p>\n

Write to Bill Alpert at william.alpert@barrons.com<\/p>\n<\/p><\/div>\n<\/div>\n

Read the full article here<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

The medical breakthrough announced Sunday by Verve Therapeutics seemed like bullish news. In the first human test of a genetic technology called \u201cbase-editing\u201d, Verve\u2019s one-time treatment durably reduced patients\u2019 high cholesterol. In Monday\u2019s market, however, the stock (ticker: VERV) sank 41%, to $9.29. It wasn\u2019t the reaction that chief executive Sekar Kathiresan had wanted. But […]<\/p>\n","protected":false},"author":1,"featured_media":34096,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"video","meta":{"footnotes":""},"categories":[239],"tags":[83],"class_list":["post-84363","post","type-post","status-publish","format-video","has-post-thumbnail","hentry","category-investing","tag-featured","post_format-post-format-video"],"yoast_head":"\nHeart Disease Breakthrough by Gene-Editor Verve Knocks Its Stock for a Loop | iFintechWorld<\/title>\n<meta name=\"description\" content=\"The medical breakthrough announced Sunday by Verve Therapeutics seemed like bullish news. 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