Marriage for MS treatment

THE WOODLANDS, Texas—Comprehensive expertise in T celltherapy, experience in personalized clinical treatment and the ability to get adrug candidate for the treatment of multiple sclerosis (MS) to Phase IIclinical trials attracted pharmaceutical giant Merck Serono to sign a deal withOpexa Therapeutics Inc. that could generate up to $225 million in payments toOpexa.

Additionally, Neil K. Warma, president and CEO of OpexaTherapeutics—which develops patient-specific cellular therapies for thetreatment of autoimmune diseases— believes that the key factors that sold MerckSerono on Opexa were the drug's superior safety profile, encouraging efficacyand disease specifity.

From Opexa's perspective, Merck Serono has a deepunderstanding of MS and can take Opexa's drug candidate through regulatoryapproval and commercialization, Warma says. He adds, "having a partner likeMerck speaks well for us."

Susan Herbert, head of global business development andstrategy at Merck Serono, says, "This agreement illustrates Merck Serono'scommitment to employ creative ways of accessing external innovation to developpotential next-generation multiple sclerosis treatments, especially insecondary progressive multiple sclerosis (SPMS)."

The current MS market is $10 billion to $11 billion. MS, achronic inflammatory condition of the central nervous system, is the mostcommon, non-traumatic, disabling neurological disease in young adults. It isestimated that approximately two million people have MS worldwide. Symptomsinclude blurred vision, numbness or tingling in the limbs and problems withstrength and coordination. Forty percent of MS patients suffer from SPMS, forwhich there is currently no treatment.

The two companies recently agreed to an option and licensingagreement for Opexa's lead therapeutic candidate, Tcelna (imilecleucel-T), forthe treatment of multiple sclerosis. Opexa retains a number of key rightsthrough the deal (manufacturing, Japanese market, all indications outside of MSand a co-development option).

Merck Serono gets an option for an exclusive license fromOpexa to develop and commercialize Tcelna (imilecleucel-T), an investigationalT-cell therapy for patients suffering from multiple sclerosis (MS). Opexa getsan upfront payment of $5 million for granting the option to Merck Serono, whichwould pay an upfront license fee of either $25 million or $15 million forworldwide development and commercial rights to Tcelna in MS, excluding Japan.Merck Serono is wholly responsible for funding clinical development, subject toOpexa's co-funding option, as well as regulatory and commercialization activitiesfor the MS program. Additional financial considerations include development andcommercial milestone payments to Opexa of up to $195 million and a tieredroyalty rate based on net sales payable to Opexa.

Tcelna is manufactured using ImmPath, Opexa's proprietarymethod for the production of a patient-specific T-cell immunotherapy. The drugcandidate is derived from T cells isolated from peripheral blood, expanded ex vivo and reintroduced into thepatients via subcutaneous injections. This process triggers a potent immuneresponse against specific subsets of autoreactive T cells known to attackmyelin.

Tcelna, which is currently in a Phase IIb clinical trial inpatients with SPMS, is being developed as a personalized therapy specificallytailored to each patient's individual disease profile. It has been evaluated inPhase I and II clinical studies in MS that included SPMS patients and hasreceived Fast Track Designation from the U.S. Food and Drug Administration as apotential treatment for SPMS. Initially developed at Baylor University Collegeof Medicine, Tcelna has the advantage of "being able to cross the blood/barrierand become localized to treat chronic inflammation."

Opexa's Phase IIb study of Tcelna is a randomized,double-blind, placebo-controlled clinical study in patients who demonstrateevidence of disease progression without associated relapses. The trial isexpected to enroll 180 patients at approximately 30 leading clinical sites inthe U.S. and Canada with each patient receiving two annual courses of Tcelnatreatment consisting of five subcutaneous injections per year. The trial'sprimary efficacy outcome is the percentage of brain volume change (atrophy) at24 months. Study investigators will also measure several important secondaryoutcomes commonly associated with MS.

Merck Serono, the biopharmaceutical division of Merck,offers leading brands in 150 countries to help patients with cancer, multiplesclerosis, infertility, endocrine and metabolic disorders as well ascardiovascular diseases. In the United States and Canada, EMD Serono operatesas a separately incorporated subsidiary of Merck Serono. Merck is a globalpharmaceutical and chemical company with total revenues of $13.4 billion in2011, a history that began in 1668.

"First and foremost, Tcelna appears to be one of thebest therapies for MS," Warma concludes. "Hopefully, the platform can beleveraged for other diseases as well."