Published in Pipeline

Adverum Biotechnologies raises $127.5 million for gene therapy pipeline

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5 min read

In a move to fund its gene therapy pipeline plans through late 2025, Adverum Biotechnologies, Inc. is selling an estimated 106.25 million shares of its common stock in a private placement.

Give me a refresh on the company.

Launched in 2006 and based in Redwood City, California, Adverum Biotechnologies (formerly known as Avalanche Biotechnologies) is a publicly traded clinical-stage gene therapy company focused on establishing gene therapy candidates as a new standard of care for highly prevalent ocular diseases.

Through its proprietary intravitreal (IVT) platform, the company has been developing durable, single-administration therapies designed for in-office use.

Now this funding.

Adverum’s $106.25 million shares of common stock (or pre-funded warrants, which allow a warrant holder to purchase a specified number of a company's securities at an equivalent price) are being sold to a “select group of institutional and accredited health specialist investors in a private placement,” according to the company.

  • Price per share: $1.20*
  • Price of pre-funded warrants per share: $1.1999

See here for details on the company’s new and existing investors who participated in this funding, which includes Frazier Life Sciences and Venrock Healthcare Capital Partners.

*Note: This is reported to represent a premium of an estimated 20% of Adverum’s 30-day volume-weighted average price.

Hold on a second… didn’t I just hear about Frazier Life Sciences?

Great memory!

Frazier Life Sciences formed a strategic partnership recently with Ascendis Pharma A/S to spin-off a new standalone company called Eyconis, Inc., focusing on the development, manufacturing, and commercialization of the TransCon (transient conjugation) ophthalmology assets across the globe.

Now back to Adverum’s package.

The company expects the gross proceeds from the private placement financing to be approximately $127.5 million—prior to deducting any offering-related expenses, that is.

When do they expect to close?

February 8, 2024 (subject to standard closing conditions).

So talk about this gene therapy pipeline.

The company’s novel gene therapy candidate—ixoberogene soroparvovec (Ixo-vec), originally named ADVM-022—is a one-time, IVT injection that incorporates Adverum’s proprietary adeno-associated virus vector capsid (AAV.7m8) to treat patients with wet age-related macular degeneration (AMD).

Ixo-vec is a vectorized therapeutic protein (aflibercept) that has reportedly demonstrated sustained durability and a promising safety profile from one injection.

Further, the majority of treated patients have demonstrated to be free of supplemental injections at 1+ years.

Give me more on this AAV.7m8.

Per Adverum, the AAV.7m8 transgene delivery vector technology “encodes retinal cells to become biofactories that provide robust, sustainable amounts of therapeutic proteins throughout the retina.”

And how does it interact with Ixo-vec?

The lead candidate incorporates AAV.7m8 to transport a codon optimized sequence for aflibercept (the anti-vascular endothelial growth factor [VEGF] protein) in order to “transduce its production in retina cells.”

The resulting effect: Ixo-vec could deliver long-term VEGF inhibition following just one IVT injection in wet AMD patients.

Gotcha. Any clinical data on it so far?

There is! Ixo-vec was evaluated in Adverum’s OPTIC clinical program, which is investigating its safety and efficacy for the first 2 years post-treatment with the dose-ranging OPTIC trial (NCT03748784) and then evaluating long-term patient outcomes up to 5 years (OPTIC extension study).

The OPTIC trial findings:

  • Ixo-vec was generally well tolerated with dose-dependent inflammation responsive to topical steroids.
  • Participants had an 81% to 98% reduction in annualized anti-VEGF injections + continuous therapeutic aflibercept protein levels demonstrated through 3 years in extension study.

And the extension study:

  • Continued long-term benefit from Ixo-vec through 3 years of follow-up: maintenance of vision and sustained reduction in anti-VEGF treatment burden
  • Aflibercept protein levels sustained through 4.5 years post-treatment

So what’s the end goal?

The company is developing Ixo-vec to have the potential to provide a durable, well-tolerated, and cost-effective in-office treatment option that meets the clinical needs of wet AMD patients.

What’s next?

Preliminary safety and efficacy data from the ongoing phase 2 LUNA study, which is evaluating two doses of Ixo-vec, will be presented at the 47th annual meeting of the Macula Society on Thursday, February 8, 2024, at 8 am EST.

Stay tuned for the data readout!