Shire bags option on device-enabled oral FVIII drug

The Rani Pill (Rani Therapeutics)

Shire has teamed up with Rani Therapeutics to develop an oral factor VIII therapy. The project is built upon a device Rani designed to enable the oral delivery of proteins and other molecules that are currently administered via injections.  

Scientists have strived for decades to make such formulations using pharmaceutical techniques but have been bedeviled by low bioavailability. Rani thinks its mechanical solution can succeed where these earlier efforts failed. 

Rani’s device, dubbed the Rani Pill, looks like an ordinary capsule when it is swallowed but hides a balloon and needles inside its shell. 

The delivery vehicle stays in its conventional capsule form until it has passed through the stomach and into the small intestine, at which point the change in pH causes the outer cellulose shell to dissolve. This triggers a carbon dioxide-producing reaction in a balloon, which in turn causes the balloon to inflate and force drug-loaded dissolvable needles into the intestinal wall.  

Rani is banking that the lack of sharp-pain receptors in the intestine will ensure the patient feels no pain and that the dense network of blood vessels in the organ's wall will result in fast absorption of the drug. Shire is one of many major companies to be wooed by the idea. 

In Shire’s case, the device is enticing because it creates an opportunity to provide hemophilia A patients with a new, more convenient way of preventing bleeding episodes. 

“With this technology, though early in development in hemophilia, we hope to improve compliance, quality of life and outcomes for patients with hemophilia by offering a painless and more convenient oral delivery,” Rani CEO Mir Imran said in a statement. 

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Shire has embarked on multiple projects with similar goals, leading to the approval of a twice-weekly formulation but also setbacks such as the failure of a once-weekly candidate earlier this year.

An oral formulation would blow these earlier injectable efforts out of the water in terms of ease of use and convenience. Shire has moved to seize the opportunity and protect its franchise by landing an exclusive option to negotiate a license for an oral factor VIII therapy enabled by Rani’s pill.

The agreement cements Rani’s position at the forefront of attempts to make oral formulations of existing injectable therapies. Rani lists AstraZeneca, Novartis and GV among its investors and has tapped these and other backers for $100 million to date. AstraZeneca and Novartis are testing the device with certain drugs.