Art Levin is liv­ing his “sci­en­tist’s dream come true.”

The Avid­i­ty Bio­sciences CSO set out years ago to build a new class of oligonu­cleotide-based ther­a­pies for se­vere mus­cle dis­eases, like Duchenne mus­cu­lar dy­s­tro­phy (DMD) and my­oton­ic dy­s­tro­phy (DM1) — rare, ge­net­ic dis­or­ders that caus­es pro­gres­sive mus­cle weak­ness.

“As you might imag­ine, in the 15 or 16 years I’ve been work­ing on this, I’ve seen boys go from be­ing tod­dlers, to be­ing wheel­chair-bound, or even ven­ti­la­tor-bound,” he said, re­fer­ring to DMD. “It would be re­al­ly … a sci­en­tif­ic dream of mine to ac­tu­al­ly make a dif­fer­ence for this pa­tient pop­u­la­tion.”

At JP Mor­gan, Avid­i­ty is up­dat­ing in­vestors that their DM1 can­di­date is of­fi­cial­ly off to the races — head­ed for a Phase I/II tri­al in the sec­ond half of this year. The team is al­so div­ing in­to a part­ner­ship with Bris­tol My­ers Squibb sub­sidiary MyoKar­dia to ex­plore their ap­proach in car­diac tis­sue.

“It’s re­al­ly grat­i­fy­ing that this tech­nol­o­gy that we built and en­gi­neered in-house our­selves is com­ing to fruition,” Levin said.

DM1 is caused by hun­dreds of thou­sands of nu­cleotide re­peats at the end of a gene called DMPK. When the DMPK gene is tran­scribed in­to RNA, the RNA al­so car­ries the nu­cleotide re­peats, which form a hair­pin loop struc­ture, mak­ing the RNA tox­ic to the cell.

“Us­ing the tech­nol­o­gy that we’re talk­ing about, you can re­verse that and re­place these mis­placed pro­teins or mis­s­pliced RNA with prop­er­ly spliced RNA,” Levin told End­points back in 2019. “In the ac­tu­al treat­ment of pa­tients, we should ac­tu­al­ly be able to re­verse some of the na­ture of this dis­ease.”

The idea that you can use oligonu­cleotides to mod­i­fy hu­man RNA isn’t new. The prob­lem has al­ways been de­liv­ery, Levin said.

“Cells have evolved over eons to try and keep out RNA and keep out for­eign DNA,” he said. “So you re­al­ly have to trick the cell in­to want­i­ng to take up our oligonu­cleotide ther­a­peu­tic.”

The team is work­ing on An­ti­body Oligonu­cleotide Con­ju­gates (AOCs) — oligonu­cleotides com­bined with mon­o­clon­al an­ti­bod­ies in­to a sin­gle con­ju­gate that can be more eas­i­ly di­rect­ed to pre­cise tis­sues.

Avid­i­ty al­so has AOC pro­grams for fa­cioscapu­lo­humer­al mus­cu­lar dy­s­tro­phy (FSHD), Duchenne mus­cu­lar dy­s­tro­phy (DMD), and mus­cle at­ro­phy (MA). The FSHD pro­gram is head­ed for IND en­abling stud­ies this year, and the biotech ex­pects to file for a clin­i­cal tri­al in 2022. There are three pro­grams with­in DMD, the leader of which tar­gets Ex­on 44. The com­pa­ny plans on sub­mit­ting for per­mis­sion to en­ter the clin­ic in 2022.

In June, the biotech land­ed a near­ly $300 mil­lion IPO to make it all hap­pen. They tagged $65 mil­lion for their DM1 can­di­date, $35 mil­lion for DMD, and $25 mil­lion for MA, ac­cord­ing to an S-1/A. 

“We al­so in­tend to pur­sue the de­vel­op­ment of AOCs in cell types in ad­di­tion to mus­cle. In pre­clin­i­cal mod­els, we ob­served the abil­i­ty of AOCs not on­ly to de­liv­er to the liv­er, skele­tal and car­diac mus­cle, but al­so to oth­er tis­sue and cell types, in­clud­ing im­mune cells,” the doc­u­ment states.

Eli Lil­ly holds 6.6% of Avid­i­ty’s stock, ac­cord­ing to the S-1/A. Back in 2019, Avid­i­ty inked a li­cens­ing and re­search deal with Lil­ly, snag­ging $20 mil­lion up­front and an in­vest­ment of $15 mil­lion. Lil­ly is of­fer­ing up to $405 mil­lion more in mile­stones, plus roy­al­ties.

Levin kept mum about what the com­pa­ny is work­ing on with MyoKar­dia, on­ly re­veal­ing that they’re de­vel­op­ing a “sin­gle ther­a­peu­tic en­ti­ty.” The com­pa­nies are keep­ing the fi­nan­cial terms of the deal un­der wraps for now.

“We’re now re­al­ly now see­ing the fruits of all those labors come true,” Levin said. “We can build on peo­ple’s new ap­pre­ci­a­tion for what is a fun­da­men­tal build­ing block of life: RNA. Peo­ple are fi­nal­ly be­gin­ning to re­al­ize that this is a re­al way to cre­ate ther­a­peu­tic en­ti­ties.”

A cor­rec­tion has been made to clar­i­fy that Levin was re­fer­ring to DMD, not DM1, in his quote in the third para­graph.

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Three years after first launching Calquence as a second generation BTK inhibitor, AstraZeneca continues to tout new data to compete with J&J and AbbVie’s first generation blockbuster Imbruvica.

The British pharma announced on Monday that Calquence passed a head-to-head Phase III study against Imbruvica in chronic lymphocytic leukemia, proving non-inferior — i.e. just as good — as the older drug. Although AstraZeneca did not break down any of the numbers, they said the drug proved superior on safety, triggering fewer cases of atrial fibrillation, an irregular heartbeat that can lead to stroke or heart failure.

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Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.