Submission of rolling BLA to US FDA for dara­tu­mu­mab in multiple myeloma com­pleted by Janssen Biotech, Inc.

Completion of sub­mission triggers USD 15 million mile­stone pay­ment to Genmab

Copenhagen, Denmark (Press Release) — Genmab A/S (OMX: GEN) announced its licensing partner Janssen Biotech, Inc. has com­pleted the rolling sub­mission of the 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 prior lines of ther­apy in­­clud­ing both a pro­te­a­some inhibitor (PI) and an immuno­modu­la­tory agent (IMiD) or who are double refractory to a PI and an IMiD. In May, 2013, dara­tu­mu­mab was granted a Break­through Therapy Desig­na­tion (BTD) in this pop­u­la­tion. The completion of the sub­mission triggers a mile­stone pay­ment of USD 15 million to Genmab from Janssen. The mile­stone was in­cluded in Genmab's financial guidance for 2015, which was updated on May 20, 2015. In August 2012, Genmab granted Janssen Biotech, Inc. an exclusive world­wide license to de­vel­op, manu­fac­ture and com­mer­cial­ize dara­tu­mu­mab.

A request for Priority Review has been submitted by Janssen with this BLA. The FDA will inform Janssen whether a Priority Review has been granted by calendar day 60 of their review starting today. If the FDA grants Priority Review the review period may not exceed 6 months from that date.

If dara­tu­mu­mab receives FDA approval, Genmab will receive a mile­stone pay­ment from Janssen of USD 45 million asso­ci­ated with the first commercial sale of the prod­uct in the United States. However, it is not possi­ble to precisely predict the timing of a poten­tial mar­ket­ing approval and first commercial sale; there­fore, this mile­stone has not been in­cluded in the 2015 financial guidance at this time.

"The rapid completion of the BLA sub­mission brings us a sig­nif­i­cant step closer to the poten­tial regu­la­tory approval of dara­tu­mu­mab," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab. "Daratu­mu­mab received Break­through Therapy Desig­na­tion from the FDA for this indi­ca­tion for multiple myeloma pa­tients who have no other treat­ment options avail­able, and we are proud that our partner Janssen has com­pleted the sub­mission in record time."

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 PI and an IMiD, or who are double refractory to a PI 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.

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.