• Ongoing Phase 1/2a Study of CD19 CAR-NK with Pivotal Study Expected to Enroll Patients in 2021
• Potential to be the First CAR Cell Therapy Approved for Outpatient Admin­istra­tion

{{image}}Houston, TX and Osaka, Japan (Press Release) – The University of Texas MD Anderson Cancer Center and Takeda Pharma­ceu­tical Com­pany Limited (TSE:4502/NYSE:TAK) (“Takeda”) to­day an­nounced an ex­clu­sive license agree­ment and re­search agree­ment to de­vel­op cord blood-derived chi­meric an­ti­gen re­cep­tor-directed natural killer (CAR NK)-cell ther­a­pies, ‘armored’ with IL-15, for the treat­ment of B-cell malig­nan­cies and other cancers.

Under the agree­ment, Takeda will re­ceive access to MD Anderson’s CAR NK plat­form and the ex­clu­sive rights to de­vel­op and com­mer­cial­ize up to four pro­grams, in­clud­ing a CD19-targeted CAR NK-cell ther­apy and a B-cell maturation an­ti­gen (BCMA)-targeted CAR NK-cell ther­apy. Takeda and MD Anderson will also conduct a re­search col­lab­o­ration to fur­ther de­vel­op these CAR NK pro­grams.

“Our vision is to im­prove upon existing treat­ments by devel­op­ing armored CAR NKs that could be admin­istered off-the-shelf in an outpatient setting—enabling more patients to be treated ef­fec­tively, quickly and with minimal toxicities,” said Katy Rezvani, M.D., Ph.D., pro­fessor of Stem Cell Trans­plan­ta­tion and Cellular Ther­a­py at MD Anderson. “With their ex­per­tise in hema­to­logic malig­nan­cies and com­mitment to devel­op­ing next-gener­a­tion cell ther­a­pies, Takeda is the ideal col­lab­o­rator to help our team ad­vance CAR NK-cell ther­a­pies to patients in need of treat­ments.”

A Novel Approach to Delivering Off-the-Shelf CARs in an Outpatient Setting

MD Anderson’s allo­geneic CAR NK plat­form isolates NK cells from umbilical cord blood and engi­neers them to express CARs against specified cancer targets. CAR NK cells are modified with a retroviral vector to de­liver genes and en­hance their ef­fec­tiveness to attack spe­cif­ic tumors. A CD19 CAR in­creases the cells’ spe­cif­icity for B-cell malig­nan­cies while the immunocytokine IL-15 en­hances the pro­lif­er­a­tion and sur­vival of the CAR NK cells in the body.

In contrast to current CAR T-cell ther­a­pies that uti­lize a patient’s own ge­net­ic­ally modified T-cells and re­quire a multi-week manu­fac­tur­ing process, CAR NK cells are in­tended to be manu­fac­tured from a non-related donor source and stored for off-the-shelf use, allow­ing treat­ment to be de­liv­ered more rapidly.

It is antic­i­pated that the CD19 CAR NK-cell ther­apy could be admin­istered in an outpatient setting. In an on­go­ing phase 1/2a clin­i­cal study treating patients with re­lapsed and re­frac­tory B-cell malig­nan­ces, the CD19 CAR NK-cell ther­apy has not been asso­ci­ated with the severe cytokine release syn­drome (CRS) or neurotoxicity observed with existing CAR-T ther­a­pies.

The devel­op­ment of MD Anderson’s CAR NK plat­form is led by Dr. Rezvani and is fur­ther sup­ported by the adoptive cell ther­apy plat­form, Chronic Lym­phocytic Leukemia Moon Shot^® and B-Cell Lym­phoma Moon Shot^®, all part of the in­sti­tu­tion’s Moon Shots Program^®, a col­lab­o­rative effort to rapidly de­vel­op scientific discoveries into meaningful clin­i­cal ad­vances that save patients’ lives.

Takeda: Accelerating the De­vel­op­ment of Multiple Next-Generation CAR Platforms

“MD Anderson’s CAR NK plat­form rep­re­sents the curative poten­tial of cell ther­a­pies, which is why we are estab­lish­ing the CD19 CAR NK as our lead cell ther­apy can­di­date in on­col­ogy,” said Andy Plump, M.D., Ph.D., Pres­i­dent of Research and De­vel­op­ment at Takeda. “We need to work swiftly and with pur­pose, and as such, we in­tend to ini­ti­ate a pivotal study of the CD19 CAR NK in 2021.”

In addi­tion to CAR NK-cell ther­a­pies, Takeda and its part­ners are investigating mul­ti­ple ap­proaches to im­prov­ing the safety, ef­fi­cacy and accessibility of first-generation CAR T-cell ther­a­pies in­clud­ing gamma delta CAR Ts, induced pluripotent stem cell-derived CAR Ts, CAR Ts targeting solid tumors, and other next-gener­a­tion ap­proaches. Takeda plans to ad­vance five on­col­ogy cell ther­a­pies to the clinic by the end of FY20.¹ These plat­forms are being devel­oped both with part­ners and by applying the ex­per­tise of Takeda’s trans­la­tional cell ther­apy engine which provides bio­engineer­ing, chemistry, manu­fac­tur­ing and con­trol (CMC), clin­i­cal and trans­la­tional capabilities in a single footprint to over­come many of the manu­fac­tur­ing chal­lenges ex­peri­enced in cell ther­apy devel­op­ment.

Takeda is responsible for the devel­op­ment, manu­fac­tur­ing and com­mer­cial­iza­tion of CAR NK prod­ucts re­sult­ing under the agree­ment. MD Anderson will re­ceive an up­front pay­ment and is eli­gible to re­ceive devel­op­ment and com­mer­cial mile­stones for each target as well as tiered royalties on net sales of any such CAR NK prod­uct.

MD Anderson and Takeda will con­tinue re­search for the addi­tional targets and CAR NK plat­form under the direction of a joint re­search com­mit­tee. MD Anderson will implement an In­sti­tu­tional Conflict of Interest Management and Monitoring Plan for this re­search.

About MD Anderson

The University of Texas MD Anderson Cancer Center in Houston ranks as one of the world's most respected centers focused on cancer patient care, re­search, education and prevention. The in­sti­tu­tion’s sole mis­sion is to end cancer for patients and their families around the world. MD Anderson is one of only 50 com­pre­hen­sive cancer centers designated by the National Cancer Institute (NCI). MD Anderson is ranked No.1 for cancer care in U.S. News & World Report’s “Best Hospitals” survey. It has ranked as one of the nation’s top two hos­pi­tals for cancer care since the survey began in 1990, and has ranked first 15 times in the last 18 years. MD Anderson re­ceives a cancer center sup­port grant from the NCI of the National Institutes of Health (P30 CA016672).

About Takeda Pharma­ceu­tical Com­pany Limited

Takeda Pharma­ceu­tical Com­pany Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven bio­pharma­ceu­tical leader headquartered in Japan, com­mit­ted to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative med­i­cines. Takeda focuses its R&D efforts on four thera­peutic areas: Oncology, Gastroenterology (GI), Rare Diseases and Neuro­science. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on devel­op­ing highly inno­va­tive med­i­cines that con­trib­ute to making a dif­fer­ence in people's lives by ad­vanc­ing the frontier of new treat­ment op­tions and leveraging our en­hanced col­lab­o­rative R&D engine and capabilities to create a robust, modality-diverse pipe­line. Our employees are com­mit­ted to im­prov­ing quality of life for patients and to work­ing with our part­ners in health care in approx­i­mately 80 countries and regions. For more in­for­ma­tion, visit https://www.takeda.com

Forward-Looking State­ments

This press release and any ma­teri­als distributed in con­nec­tion with this press release may con­tain for­ward-looking state­ments, beliefs or opinions re­gard­ing Takeda’s future business, future position and results of op­er­a­tions, in­clud­ing esti­mates, forecasts, targets and plans for Takeda. In par­tic­u­lar, this press release con­tains forecasts and man­agement esti­mates related to the fi­nan­cial and op­er­a­tional per­for­mance of Takeda, in­clud­ing state­ments re­gard­ing forecasts for Revenue, Operating profit, Adjusted EBITDA, Profit before income taxes, Net profit attributable to owners of Takeda, Basic earn­ings per share, Amortization and im­pair­ment and other income/expense, Underlying Revenue, Underlying Core Earnings margin, Underlying Core EPS and Net Debt. Without lim­i­ta­tion, for­ward looking state­ments often in­clude the words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “will”, “may”, “should”, “would”, “could” “anticipates”, “estimates”, “projects” or words or terms of similar sub­stance or the neg­a­tive thereof. Any for­ward-looking state­ments in this doc­u­ment are based on the current assump­tions and beliefs of Takeda in light of the in­for­ma­tion cur­rently avail­able to it. Such for­ward-looking state­ments do not rep­re­sent any guar­an­tee by Takeda or its man­agement of future per­for­mance and in­volve­ known and unknown risks, un­cer­tainties and other factors, in­clud­ing but not limited to: the economic cir­cum­stances surrounding Takeda’s business, in­clud­ing general economic con­di­tions in Japan, the United States and world­wide; competitive pres­sures and devel­op­ments; appli­cable laws and reg­u­la­tions; the success of or failure of prod­uct devel­op­ment pro­grams; de­ci­sions of regu­la­tory author­i­ties and the timing thereof; changes in ex­change rates; claims or con­cerns re­gard­ing the safety or ef­fi­cacy of mar­keted prod­ucts or prod­ucts can­di­dates; and post-merger integration with acquired com­pa­nies, any of which may cause Takeda’s actual results, per­for­mance, achieve­ments or fi­nan­cial position to be ma­teri­ally dif­fer­en­t from any future results, per­for­mance, achieve­ments or fi­nan­cial position ex­pressed or im­plied by such for­ward-looking state­ments. For more in­for­ma­tion on these and other factors which may affect Takeda’s results, per­for­mance, achieve­ments, or fi­nan­cial position, see “Item 3. Key In­for­ma­tion—D. Risk Factors” in Takeda’s Reg­is­tra­tion State­ment on Form 20-F filed with the U.S. Se­cu­ri­ties and Ex­change Com­mis­sion, avail­able on Takeda’s website at: https://www.takeda.com/investors/reports/sec-filings/ or at www.sec.gov. Neither Takeda nor its man­agement gives any assurances that the ex­pec­ta­tions ex­pressed in these for­ward-looking state­ments will turn out to be correct, and actual results, per­for­mance or achieve­ments could ma­teri­ally differ from ex­pec­ta­tions. Persons re­ceiv­ing this press release should not place undue reliance on for­ward looking state­ments. Takeda under­takes no obli­ga­tion to up­date any of the for­ward-looking state­ments con­tained in this press release or any other for­ward-looking state­ments it may make. Past per­for­mance is not an indicator of future results and the results of Takeda in this press release may not be indicative of, and are not an esti­mate, forecast or pro­jec­tion of Takeda’s future results.

Footnotes

¹ Takeda’s 2020 fiscal year begins April 1, 2020 and ends March 31, 2021.

Source: MD Anderson and Takeda.