DEERFIELD, Ill.—In keeping with the rise of orphandisease-focused partnerships, Baxter International Inc. and OnconovaTherapeutics Inc. have announced the signing of a European licensing agreementfor rigosertib, a novel targeted anti-cancer compound that is currently beingevaluated in a Phase III study for the treatment of Myelodysplastic Syndromes(MDS), a group of rare hematological malignancies, and in a Phase II/III studyfor pancreatic cancer.
Per the agreement, Baxter will pay Onconova $50 millionupfront, for which it will receive commercialization rights in the EuropeanUnion and other countries in Europe. If certain precommercial development andregulatory milestones are met for the initial indications, Onconova stands toreceive up to $515 million in milestone payments, as well as sales milestonesand royalties. Baxter will have the option to participate in developing and commercializingrigosertib in additional indications, and has an existing equity investmentwith Onconova of $50 million.
"Rigosertib's first anticipated indication would be anatural complement to Baxter's existing treatments for patients managing rare hematologicconditions, and will allow us to expand the product portfolio of our existinghematologic sales force," Dr. Ludwig Hantson, president of Baxter's BioSciencebusiness, said in a press release. "Our collaboration with Onconova will allowus to further expand our pipeline and extend our legacy in disease areas withcritical needs."
A spokesman for Baxter said the company was attracted toOnconova as a partner because the Newtown, Pa.-based company "demonstrated verystrong potential to build a broader relationship beyond the anticipated EUlicensing opportunity." The agreement, they add, allows Baxter "to furtherexpand our pipeline and extend our legacy in disease areas with critical needsby focusing resources on a potential solution for MDS and pancreatic cancer,two conditions with very limited options today."
Rigosertib targets two pathways—PI-3k and PLK—known to playsignificant roles in the growth of cancer cells, and has demonstrated activityin treating solid tumors as well as hematological malignancies. The compound'scurrent Phase III trial consists of 270 MDS patients who have failed orrelapsed after undergoing current treatment options, and results are expectedin the second half of next year. The Phase II/III trial is testing rigosertibin patients with previously untreated metastatic pancreatic cancer, and an oralformulation of the compound is being evaluated in a Phase II study intransfusion-dependent low or intermediate-1 risk MDS patients. So far, Onconovahas secured orphan drug designation for MDS in the United States and Europe,and for pancreatic cancer in the United States.
Ramesh Kumar, CEO of Onconova, says Baxter emerged as"almost a perfect match for the product and for us," given the pharma's "stronglegacy in hematology," its interest in growing its oncology business and thefact that nearly a third of its revenue comes from its dealings in Europe.
"They have a strong commercial presence in Europe, they havea strong desire in oncology and most of all, they're a specialist inhematology. The more we looked into it—and because we wanted to do a regionalpartnership, not a global one—Baxter appeared to us ideally suited as apartner," says Kumar.
He notes that the partnership enhances Onconova's pipelineand "reinforces our commitment to cancer in general," noting that "rigosertib,because of its mechanism, is potentially very widely useful." Additionalpotential indications include other hematological cancers such as acute myeloidleukemia (AML), and solid tumors such as ovarian cancer and head and neckcancer, he adds.
"The only way we can maximize the potential of the drug isfirst to get it approved and secondly to expand the utility by doing additionaltrials," says Kumar. "And our relationship with Baxter, as well as ourrelationship with SymBio and the Leukemia & Lymphoma Society, enables us tobroadly explore the potential value of the drug by focusing on the speedyapproval for the first indication in MDS."
MDS encompasses a variety of bone marrow disorders associatedwith increased risk of bleeding, infection and progression to AML. Patientsoften require multiple blood transfusions and significant supportive care andface poor survival rates, with an annual incidence in Europe of roughly threeper 100,000 people. Pancreatic cancer is highly aggressive and difficult todiagnose early due to vague symptoms. The disease has a low survival rate, andrecent estimates place the mortality rate in Europe between eight in 100,000 inmen and five in 100,000 in women.