Sarepta Inks Codiak Deal to Explore Combining Exosomes, Muscle Meds

Xconomy Boston — 

Tiny, naturally occurring particles called exosomes shuffle molecular payloads between cells, facilitating intercellular communication. Codiak Biosciences’ focus is using those nanoparticles to more safely deliver a range of medicines to specific cell types.

Now the Cambridge, MA-based biotech has teamed up with neuromuscular disease drug developer Sarepta Therapeutics (NASDAQ: SRPT) to explore using its engineered exosomes technologies to deliver potential gene therapy, gene editing, and RNA drugs.

The companies on Monday announced the two-year deal, which includes up to five neuromuscular targets. The alliance makes Codiak eligible for up to $72.5 million in upfront and near-term license payments, though the breakdown of those payments was not specified.

Using exosomes as a delivery system for genetic medicines is viewed as a possible way to get those drugs to cells without prompting an adaptive immune response. The idea is that since exosomes occur naturally, the immune system won’t consider the particles stuffed with a genetic payload as foreign matter. That acceptance of the delivery vehicle as non-threatening upside could also smooth the path to redosing, if later needed. An additional potential benefit of Codiak’s technology, which it calls engEx, is that exosomes can be engineered to target specific cell types, Sarepta CEO Doug Ingram said in a prepared statement. Codiak president and CEO Doug Williams, a former Biogen (NASDAQ: BIIB) research chief, elaborated.

“The development of targeted delivery systems that enable repeat-dosing to select cell types has been a challenge for the emerging field of genetic medicine, especially in diseases of the muscle,” Williams said. “Engineered exosomes may offer a solution through their potential to selectively target muscle cells.”

Under the deal terms, the companies will together design exosomes that can deliver payloads in neuromuscular indications. If Sarepta decides to exercise its option on a target, Codiak will be responsible for research and preclinical development; Sarepta, for clinical development and commercial activities.

Codiak has developed a pipeline of drug candidates across immuno-oncology, immuno-neurology and oncology, but hasn’t tested any in humans yet. This year the company has said it intends to move its two lead drug candidates into the clinic.

In addition to the upfront payment, research funding and license payments from Cambridge, MA-based Sarepta, the agreement could also bring Codiak a “significant” amount of money in payments tied to development and regulatory milestones, plus royalties, the company said Monday.

Sarepta isn’t the only biotech eying exosomes as a potential delivery vehicle for its medicines. Last year Codiak signed a similar agreement with Ireland-based Jazz Pharmaceuticals (NASDAQ: JAZZ) that brought it $56 million up front. The deal, which encompasses five oncology targets, also made Codiak eligible for up to $20 million more based on preclinical development progress, plus up to $200 million more per target based on other milestones.

Founded in 2015, Codiak in 2017 raised a $76.5 million Series C round from investors including founding backers Flagship Pioneering and Arch Venture Partners plus new participants EcoR1 Capital and Casdin Capital.

In April 2019 Codiak filed documents with the SEC indicating its intent to raise $86 million via an initial public offering. But the company withdrew its prospectus that July, citing unfavorable market conditions.

Image: iStock/EvgeniiAnd