CAMBRIDGE, Mass. & SAN JOSE, Calif.—Hemophilia is a challenging chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood. Factor replacement therapy attempts to make bleeding subside when enough clotting factor has reached the source of the bleed, but no oral therapies currently exist for the treatment of hemophilia. According to a recent research report, the global hemophilia treatment drug market is expected to be worth $25.1 billion in 2024, up from $15.8 billion in 2015.
Shire plc, a biotechnology company specializing in rare diseases, and Rani Therapeutics, an InCube Labs company involved in multi-disciplinary life sciences R&D, have formed a collaboration to conduct research on the use of the Rani Pill technology for the oral delivery of factor VIII (FVIII) therapy for patients with hemophilia A. According to the collaboration agreement, Shire has acquired an exclusive option to negotiate a license to develop and commercialize the technology for delivery of FVIII therapy after completing feasibility studies.
“Rani Therapeutics was founded in 2012 with the mission of converting injectable drugs into pills and thereby radically improving patient outcomes,” explained its CEO, Mir Imran. “The Rani Oral Delivery Platform (ODP) is suitable for a wide range of molecules and across therapeutics areas including inflammatory disease, diabetes and hemophilia, to name a few.”
With its platform, the company has demonstrated absorption similar to subcutaneous injections in preclinical studies, and it has established partnerships with several pharmaceutical companies, including Novartis and AstraZeneca, according to Imran. Most recently Shire, a leader in hemophilia, was interested in exploring Rani’s technology for treating hemophilia A patients, who currently are treated with infusions. Shire has entered into a collaboration with Rani to evaluate Factor VIII on the Rani ODP.
“If successful, the Rani Pill would allow Factor VIII to be delivered orally—a first-of-its-kind innovation,” Imran said. “The Rani Pill is an oral capsule that delivers a pain-free intestinal injection of the therapy. The goal is higher levels of compliance and an improved patient experience. Shire has also made an equity investment into Rani Therapeutics as part of the collaboration.”
The novel approach for the oral delivery of large molecules, including peptides, proteins and antibodies, delivers the intestinal injection without exposing medication to digestive enzymes. Once the capsule is consumed, it stays protected until it enters the small intestine and delivers medication into the intestinal wall. Because the intestines do not have sharp pain receptors, the intestinal delivery is expected to be pain-free.
According to Fritz Scheiflinger, Shire’s head of global research, “Shire is proud to partner with Rani Therapeutics to pioneer the investigation of oral delivery of factor therapy for the hemophilia community. 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. We strive to provide hemophilia patients with innovative therapies that are effective and do not expose patients to additional risks.”
Shire attempts to develop “best-in-class therapies across a core of rare disease areas—including hematology, immunology, genetic diseases, neuroscience and internal medicine—with growing therapeutic areas in ophthalmics and oncology,” Scheiflinger added. “Our diversified capabilities enable us to reach patients in more than 100 countries.”
According to Imran, “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 has the potential to disrupt the industry with its innovative approach to delivering biologics orally,” said David Pyott, member of the board of directors for Rani Therapeutics and former chairman and CEO of Allergan Inc. “The hemophilia market is an excellent application of Rani’s technology, and we are delighted that Rani is partnering with Shire, a market leader in the space.”