Roche Gives Arrowhead RNAi Assets and Facility in Madison, WI

Company gains three delivery platforms and three primary siRNA formats plus broad IP.

Arrowhead Research has acquired RNA therapeutics assets from Roche, including delivery technologies, three primary siRNA formats, as well as infrastructure and personnel. Roche obtained a minority stake in Arrowhead as well as rights to negotiate for certain future products, milestone payments, and royalties on sales. 

“The combination of these assets and Arrowhead’s existing RNAi technologies results in what we believe to be the broadest RNAi therapeutics company in the world, with unparalleled delivery solutions and licenses granting broad freedom to operate within the three primary siRNA formats,” remarks Christopher Anzalone, Ph.D., president and CEO of Arrowhead.

The deal with Roche gives Arrowhead Roche Madison, formerly Mirus Bio, providing an RNAi delivery platform known as Dynamic PolyCunjugates™ (DPCs). Roche bought Mirus Bio in 2008 for $125 million for this very technology. Arrowhead also gains access to a liposomal nanoparticle (LNP) RNAi delivery system developed by Roche and a license from Tekmira Pharmaceuticals for its SNALP RNAi delivery.

“Together with our technologies from Calando and Leonardo, we now have five RNAi delivery systems,” Dr. Anzalone points out. “We believe this transaction positions Arrowhead as the leader in delivery, which remains the limiting factor of therapeutic RNAi. We have the platforms to optimize delivery based on tissue type, disease state, target, and siRNA chemistry.”

Additionally, Arrowhead has obtained a license from Alnylam Pharmaceuticals for its RNAi IP and Canonical siRNA structures; from City of Hope providing access to Dicer substrate siRNA structures; and from MDRNA (now Marina Biotech) for Meroduplex siRNA structures.

“We now have broad access to use the three primary siRNA structures: canonical, dicer, and meroduplex,” Dr. Anzalone continues. “No other company has the ability to optimize efficiency by optimizing the type of siRNA format in this way, and this is extremely powerful.”

Under terms of the agreement, Roche also transferred to Arrowhead all its existing RNAi operations and research facilities in Madison, WI, along with its employees, equipment, related technology licenses, and intellectual property covering current and planned development programs. Arrowhead says that it gains a team of over 40 RNAi scientists.

“Acquiring three new delivery technologies gives us even greater flexibility and substantially more power across multiple disease areas,” Dr. Anzalone comments. “For instance, the Madison team has developed multiple generations of the DPC technology and generated large amounts of small animal and nonhuman primate data. We have not seen any RNAi delivery technology with better efficiency or tolerability.

“In addition, the new RNAi chemistry IP moves Arrowhead from being a force in delivery to being a complete RNAi drug developer and partner. Together, these factors put our business on stronger and broader footing than ever before. As large pharmaceutical companies are increasingly choosing to outsource RNAi development because of the delivery challenge, we are uniquely positioned to compete for partnerships that could bring substantial nondilutive capital.”

Separately, Arrowhead closed on $4 million of new funding in addition to the $6 million financing that was recently announced. In addition, the company entered into an agreement with Lincoln Park Capital (LPC) to provide up to $15 million to support the new operations. Under the LPC agreement, Arrowhead may draw all or part of the $15 million commitment at its sole discretion over a three-year period. There are no warrants associated with the facility and pricing is based on closing market prices.

“This provides Arrowhead with an efficient means to minimize dilution to shareholders by bringing in equity capital in a relatively inexpensive way and only if/when necessary,” remarks Dr. Anzalone.
Arrowhead currently has two majority-owned subsidiaries, Calando and Ablaris, and has minority investments in two early-stage companies, Nanotope and Leonardo Biosystems. Ablaris is focused on anti-obesity therapeutics and Nanotope on nanotechnology-based regenerative medicine.

Calando and Leonardo are both in the RNAi space. Calando is developing targeted, siRNA-containing therapeutics using its three-part RNAi/oligonucleotide nanoparticle delivery (Rondel) technology. CALAA-01 is Calando’s lead RNAi therapeutics program based on Rondel delivery. Leonardo Biosystems has a multistage delivery platform that has been shown in animal models to be effective in targeting delivery of siRNA and small molecule drugs.