Janssen Biotech, Inc. ini­ti­ates rolling sub­mission of BLA to U.S. FDA for dara­tu­mu­mab in double refractory multiple myeloma

Submission based on data from Phase II study (Sirius MMY2002)

Copenhagen (Press Release) – Genmab A/S (OMX: GEN) announced today its licensing partner Janssen Biotech, Inc. has ini­ti­ated a rolling sub­mission of a Biologics License Application (BLA) to the U.S. Food and Drug Admin­istra­tion (FDA) for dara­tu­mu­mab. The sub­mission is for dara­tu­mu­mab as a treat­ment for patients with multiple myeloma who have received at least three dif­fer­en­t lines of ther­apy in­­clud­ing both a pro­tea­some inhibitor and an immuno­modu­la­tory agent (IMiD) or who are double refractory to a pro­tea­some inhibitor and an IMiD. A rolling sub­mission allows com­pleted portions of the appli­ca­tion to be submitted to the FDA on an ongoing basis. The FDA grants this type of review if the agency de­ter­mines after a pre­lim­i­nary evaluation of clin­i­cal data that the breakthrough ther­apy may be effective and there­fore, will con­sider reviewing portions of an appli­ca­tion before the sub­mission is com­plete. In August 2012, Genmab and Janssen Biotech, Inc. entered an agree­ment which granted Janssen a world­wide exclusive license to develop, manu­fac­ture and com­mer­cial­ize dara­tu­mu­mab. Janssen is cur­rently the sponsor of all but one study globally.

The sub­mission in­cludes data from the Phase II study (Sirius MMY2002) of dara­tu­mu­mab in multiple mye­lo­ma patients who have received at least three prior lines of ther­apy in­­clud­ing both a pro­tea­some inhibitor and an IMiD or who are double refractory to a pro­tea­some inhibitor and an IMiD. However, safety and efficacy data from the Phase I/II study (GEN501) and safety data from three other studies, have also been in­cluded in the BLA sub­mission. Dara­tu­mu­mab received a Break­through Therapy Desig­na­tion (BTD) for this indica­tion from the FDA in May 2013.

"Daratumumab is a highly inno­va­tive anti­body that holds promise for patients with multiple myeloma, a dis­ease for which there is cur­rently no cure. Today, patients that are double refractory have run out of treat­ment options and we are very pleased that dara­tu­mu­mab offers the poten­tial to help this group of patients. The initiation of this rolling BLA sub­mission is a landmark in the devel­op­ment of dara­tu­mu­mab and we are work­ing together with Janssen to bring this new treat­ment option to patients as quickly as we can," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

About multiple myeloma

Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is char­ac­ter­ized by an excess proliferation of plasma cells.¹ Multiple myeloma is the third most common blood cancer in the United States (U.S.), fol­low­ing only leukemia and lym­phoma.² Approximately 26,850 new patients will be diag­nosed with multiple myeloma and approx­i­mately 11,240 people will die from the disease in the U.S. in 2015.³ Globally, it is esti­mated that 114,251 people will be diag­nosed and 80,019 will die from the disease.⁴ While some patients with multiple myeloma have no symp­toms at all, most patients are diag­nosed due to symp­toms which can in­clude bone problems, low blood counts, cal­cium elevation, kidney problems or in­fec­tions.⁵

About dara­tu­mu­mab

Daratumumab is an inves­ti­ga­tional human IgG1k mono­clonal anti­body (mAb) that binds with high affinity to the trans­mem­brane ectoenzyme, CD38, on the surface of multiple myeloma cells. It induces rapid tumor cell death through diverse mech­a­nisms of action. Five Phase III clin­i­cal studies with dara­tu­mu­mab in re­lapsed and frontline settings are cur­rently ongoing. Additional studies are ongoing or planned to assess its poten­tial in other malignant and pre-malignant diseases on which CD38 is ex­pressed, such as smol­der­ing myeloma and non-Hodgkin's lym­phoma. Dara­tu­mu­mab has been granted Break­through Therapy Desig­na­tion from the US FDA.

About Genmab

Genmab is a publicly traded, inter­na­tional bio­technology com­pany specializing in the creation and devel­op­ment of dif­fer­en­ti­ated human anti­body thera­peutics for the treat­ment of cancer. Founded in 1999, the com­pany cur­rently has one mar­keted anti­body, Arzerra® (ofatumumab) for the treat­ment of certain chronic lym­pho­cytic leukemia indi­ca­tions and dara­tu­mu­mab in clin­i­cal devel­op­ment for multiple myeloma and non-Hodgkin's lym­phoma, in addi­tion to other clin­i­cal pro­grams, and an inno­va­tive pre-clinical pipe­line. Genmab's tech­nology base consists of val­i­dated and pro­pri­e­tary next generation anti­body tech­nolo­gies - the DuoBody® plat­form for generation of bispecific anti­bodies, and the HexaBody™ plat­form which creates effector function en­hanced anti­bodies. Genmab's deep anti­body expertise is ex­pec­ted to provide a stream of future prod­uct can­di­dates. Partnering of selected inno­va­tive prod­uct can­di­dates and tech­nolo­gies is a key focus of Genmab's strategy and the com­pany has alliances with top tier pharma­ceu­tical and bio­technology com­pa­nies. For more in­­for­ma­tion visit www.genmab.com.

This Company Announcement con­tains for­ward looking state­ments. The words "believe", "expect", "antic­i­pate", "intend" and "plan" and similar ex­pres­sions identify for­ward looking state­ments. Actual results or per­for­mance may differ ma­teri­ally from any future results or per­for­mance ex­pressed or implied by such state­ments. The im­por­tant factors that could cause our actual results or per­for­mance to differ ma­teri­ally in­clude, among others, risks asso­ci­ated with pre-clinical and clin­i­cal devel­op­ment of prod­ucts, un­cer­tain­ties related to the out­come and conduct of clin­i­cal trials in­­clud­ing un­fore­seen safety issues, un­cer­tain­ties related to prod­uct manu­fac­tur­ing, the lack of mar­ket acceptance of our prod­ucts, our in­abil­ity to man­age growth, the competitive en­viron­ment in rela­tion­ to our business area and mar­kets, our in­abil­ity to attract and retain suitably qualified per­son­nel, the un­en­force­ability or lack of protection of our patents and pro­pri­e­tary rights, our rela­tion­ships with affiliated entities, changes and devel­op­ments in tech­nology which may render our prod­ucts obsolete, and other factors. For a further discussion of these risks, please refer to the risk man­agement sections in Genmab's most recent financial reports, which are avail­able on www.genmab.com. Genmab does not under­take any obli­ga­tion to update or revise for­ward looking state­ments in this Company Announcement nor to con­firm such state­ments in rela­tion­ to actual results, unless required by law.

Genmab A/S and its sub­sid­i­aries own the fol­low­ing trademarks: Genmab®; the Y-shaped Genmab logo®; Genmab in com­bi­na­tion with the Y-shaped Genmab logo™; the DuoBody logo®; the HexaBody logo™; HuMax®; HuMax-CD20®; DuoBody®; HexaBody® and UniBody®. Arzerra® is a trademark of Novartis Pharma AG.

References

1 American Cancer Society. "Multiple Myeloma Overview" http://www.cancer.net/cancer-types/multiple-myeloma/overview. Accessed November 24, 2014.
2 National Cancer Institute. "A Snapshot of Myeloma." Available at www.cancer.gov/research/progress/snapshots/myeloma. Accessed May 19, 2015.
3 American Cancer Society. "What are the key statistics about multiple myeloma?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-key-statistics. Accessed February 5, 2015.
4 GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide. Available at http://globocan.iarc.fr/Pages/fact_sheets_population.aspx. Accessed May 19, 2015.
5 American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed November 24, 2014.

Source: Genmab.