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.

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.