Shire, Rani Therapeutics Collaborate on Pill to Treat Hemophilia A

Shire and Rani Therapeutics have partnered to investigate the use of oral Rani Pill technology as the carrier system for clotting factor VIII in hemophilia A patients.

As part of Shire’s commitment, the Irish pharmaceutical company made an equity investment in Rani. Under the agreement — whose terms weren’t disclosed — Shire will be head the product’s development license and commercialization.

Hemophilia A patients are now treated mainly by factor replacement therapy, in which patients are injected with concentrates of clotting factor VIII. But this method of delivery entails significant safety and compliance challenges.

Although oral therapies for this disease don’t yet exist, Rani has already created the technology to convert injectable drugs into pills. Before drugs can be clinically effective, however, they must be absorbed. Most medications are designed to be primarily absorbed in the small intestine, due to its big surface area and permeable membranes.

However, oral drugs need to survive the stomach’s low pH and secretions from the digestive system.

The Rani Pill may look like an ordinary medication capsule that patients take orally, but it allows the drug that’s inside to navigate through the stomach without being degraded by the gastrointestinal tract secretions. Once in the small intestine, the carrier system undergoes a transformation that enables it to adhere to the intestine’s wall and inject the drug.

Because the small intestine does not have sharp pain receptors, taking this drug should be painless for patients.

“With Shire’s in-depth scientific expertise and leadership position in hemophilia and Rani Therapeutics’ deep experience in engineering and material science, we are excited by the potential of this partnership to reduce the chronic burden of hemophilia on patients’ everyday lives by researching an oral option to deliver FVIII to patients,” Fritz Scheiflinger, head of global research at Shire, said in a press release. “We strive to provide hemophilia patients with innovative therapies that are effective and do not expose patients to additional risks.”

Added Mir Imran, chairman and CEO of Rani Therapeutics: “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.”