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Avidity Bio Nabs Another $460M

BIOPHARMA: Surges on Positive MD Treatment Data

SAN DIEGO – Just four months after receiving positive trial results for one of its investigational drugs, local RNA therapeutics developer Avidity Biosciences (Nasdaq: RNA) has received more positive reports from a study of its program to reverse a rare form of muscular dystrophy.

The news generated a surge in the company’s stock, bringing about $461 million to Avidity, which had just raised $400 million in a private placement earlier this year in March.

The Torrey Pines-based company is pursuing three clinical-stage programs in three distinct rare muscle diseases.

Its latest study of one program found a reduction by more than 50% of the gene that causes facioscapulohumeral muscular dystrophy (FSHD), a rare, hereditary disorder that causes life-long loss of muscle function, significant pain, fatigue and progressive disability and with no approved treatments.

The company’s stock soared about 33% at the June 12 announcement and share prices hovered around $40 in the following week.

On Monday, Avidity announced it had closed its underwritten public offering of 12,132,500 shares of its common stock at a price to the public of $38 a share. All of the shares in the offering were sold by Avidity, which received about $461 million in gross proceeds before deducting underwriting discounts and commissions and other expenses.

TD Cowen, Leerink Partners, Cantor, Barclays and Wells Fargo Securities were joint bookrunning managers for the offering.

The revenue influx this year has not gone unnoticed by Wall Street. Year to date, the company’s stock has increased about 332%.

Leading in AOCs

Avidity is developing a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs).

The latest positive news on the FSHD program came from Phase 1/2 of what Avidity is calling its FORTITUDE study of the effects of the RNA therapeutic AOC 1020 on adults living with the disease.

The approved international nonproprietary name of AOC 1020 is delpacibart braxlosiran, which is abbreviated as del-brax.

Sarah Boyce
CEO
Avidity Biosciences

“With the unprecedented del-brax data from the FORTITUDE trial, we are now focused on accelerating our registrational plans as we understand the urgency to develop a treatment for people living with FSHD who have no treatment options,” said Sarah Boyce, president and chief executive officer at Avidity.

“By directly targeting the root cause of FSHD, we believe that del-brax has the potential to be a first-in-class, best-in-class therapy for people living with FSHD. This is our third rare muscle disease program to show delivery to muscle and target engagement and second therapy to provide signs of functional improvement in patients with rare neuromuscular diseases, further reinforcing the promise of our AOC platform to profoundly improve people’s lives by revolutionizing the delivery of RNA therapeutics.”

The root cause of FSHD is the abnormal expression of the gene called double homeobox 4 or DUX4. The study showed an unprecedented and consistent reduction by more than 50% of the expression of genes that are regulated by DUX4.

All participants in the study who were treated with del-brax showed more than 20% reductions in DUX4-regulated genes.

Other trends in the study were increased strength in upper and lower limb
muscles, and muscle function as measured by reachable workspace.

“We understand the urgency to develop a treatment for people living with FSHD who have no treatment options,” the company wrote in an open letter to the FSHD community following the release of the study.

“With this encouraging data, we plan to accelerate initiation of registrational cohorts in the FORTITUDE trial starting in the second half of this year,” the letter continued. “The Phase 1/2 cohorts of the FORTITUDE trial are fully enrolled. Data generated from the upcoming registrational cohorts will be used as part of the data package evaluated by regulatory agencies for new drug approval.”

Other Promising Programs

The other two types of muscular dystrophy being addressed by the company are myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44).

Earlier this year, the company announced it had positive trial results of a drug being developed to reverse the progression of DM1.

Avidity’s most recent financial results were from the first quarter ended March 31, 2024, which we reported in May 2024. Avidity has cash, cash equivalents and marketable securities totaled $915.9 million as of March 31, 2024, which reflects a gross $400 million raise from a private placement in February 2024. Avidity’s collaboration revenue totaled $3.5 million in the first quarter of 2024 related to partnerships with Bristol Myers Squibb and Eli Lilly, compared to $2.2 million related to a partnership with Eli Lilly in the first quarter of 2023. There was no revenue related to the partnership with Bristol Myers Squibb in 2023.

Avidity Biosciences
FOUNDED: 2013
PRESIDENT & CEO: Sarah Boyce
HEADQUARTERS: Torrey Pines
EMPLOYEES: 270
BUSINESS: Biopharma
ENTERPRISE VALUE: $910 million
STOCK: Nasdaq: RNA
WEBSITE: aviditybio.com
CONTACT: info@aviditybio.com
NOTABLE: The company’s stock has increased about 332% this year and it has raised about $800 million.

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