TiGenix and Takeda announce Alofisel® (darvadstrocel) receives approval to treat complex perianal fistulas in Crohn’s disease in Europe

  • First allogeneic stem cell therapy to receive central marketing authorization approval in Europe
  • Alofisel offers a new treatment option for patientswho do not respond to current available therapies and may be subject to numerous invasive surgeries1

LEUVEN, Belgium I March 23, 2018 I OSAKA, Japan I March 24, 2018 I TiGenix NV (Euronext Brussels and NASDAQ: TIG) (“TiGenix”) and Takeda Pharmaceutical Company Limited (TSE: 4502) (“Takeda”) today announced that the European Commission (EC) has approved Alofisel (darvadstrocel), previously Cx601, for the treatment of complex perianal fistulas in adult patients with nonactive/mildly active luminal Crohn’s disease, when fistulas have shown an inadequate response to at least one conventional or biologic therapy. Alofisel should be used after conditioning of fistula.1 This marks the first allogeneic stem cell therapy to receive central marketing authorization (MA) approval in Europe.

The European approval follows a positive opinion by the European Medicines AgencyEMA Committee for Medicinal Products for Human Use (CHMP), in conjunction with the Committee for Advanced Therapies (CAT), in December 2017. The recommendation was based on results from TiGenix’s Phase III ADMIRE-CD pivotal trial, which showed that Alofisel achieved statistically significant superiority versus the control group in the primary efficacy endpoint of combined remission at 24 weeks,*1 as well as further follow-up data that indicated Alofisel maintained long-term remission of treatment refractory complex perianal fistulas in patients with Crohn’s disease over 52 weeks.2

“I am extremely excited about this approval, which brings allogeneic stem cell therapy one step closer to patients in Europe,” said Professor Julian Panés, Head of the Gastroenterology Department at the Hospital Clinic of Barcelona (Spain) and President of the European Crohn's and Colitis Organisation (ECCO). “Alofisel offers a novel, minimally invasive and well tolerated alternative treatment option for patients with Crohn’s disease who do not respond to currently available therapies, and who have until now had limited treatment options available.”

“This approval of Alofisel reflects our deep understanding and recognized leadership in the development of allogeneic stem cells and our firm commitment to developing innovative therapies for medical needs," said Dr. María Pascual, VP Regulatory Affairs and Corporate Quality at TiGenix. "We are pleased to offer the medical community an important new treatment option for patients with Crohn’s disease who do not respond to currently available therapies.

Alofisel has been licensed to Takeda for the exclusive development and commercialization outside of the US. Receipt of the MA will trigger a milestone payment from Takeda to TiGenix of €15 million, and initiation of the process of transferring MA from TiGenix to Takeda. 

“Today’s marketing authorization, the first for an allogeneic stem cell therapy, represents a positive advancement in the treatment of patients with complex perianal fistulas in Crohn’s disease,” said Dr. Asit Parikh, Head of Takeda’s Gastroenterology Therapeutic Area Unit. “We look forward to bringing this much needed treatment option to patients across Europe in the coming months.”  

The receipt of MA from the EC is one of the conditions to completion of the tender offer announced by Takeda on January 5, 2018.

The consummation of the tender offer remains subject to other conditions, including the tender into the offer (in Belgium and the US), in aggregate, of a number of shares, warrants and American Depositary Shares that, together with all shares, warrants and American Depositary Shares owned by Takeda and its affiliates, represents or gives access to 85% or more of the voting rights represented or given access to by all of the outstanding shares, warrants and American Depositary Shares of TiGenix on a fully diluted basis as of the end of the initial acceptance period

* Combined remission defined as clinical assessment of closure of all treated external openings draining at baseline, despite gentle finger compression, and absence of collections >2cm confirmed by pelvic MRI

Takeda’s Commitment to Gastroenterology

Gastrointestinal (GI) diseases can be complex, debilitating and life-changing. Recognizing this unmet need, Takeda and our collaboration partners have focused on improving the lives of patients through the delivery of innovative medicines and dedicated patient disease support programs for over 25 years. Takeda aspires to advance how patients manage their disease. Additionally, Takeda is leading in areas of gastroenterology associated with high unmet need, such as inflammatory bowel disease, acid-related diseases and motility disorders. Our GI research & development team is also exploring solutions in celiac disease, advanced liver disease and microbiome therapies

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE: 4502) is a global, research and development-driven pharmaceutical company committed to bringing better health and a brighter future to patients by translating science into life-changing medicines. Takeda focuses its R&D efforts on oncology, gastroenterology and neuroscience therapeutic areas plus vaccines. Takeda conducts R&D both internally and with partners to stay at the leading edge of innovation. Innovative products, especially in oncology and gastroenterology, as well as Takeda’s presence in emerging markets, are currently fueling the growth of Takeda. Approximately 30,000 Takeda employees are committed to improving quality of life for patients, working with Takeda’s partners in health care in more than 70 countries. For more information, visit https://www.takeda.com/newsroom/

About TiGenix

TiGenix NV (Euronext Brussels and NASDAQ: TIG) is an advanced biopharmaceutical company developing novel therapies for serious medical conditions by exploiting the anti-inflammatory properties of allogeneic, or donor-derived, stem cells. 

TiGenix´ lead product, Alofisel, successfully completed a European Phase III clinical trial for the treatment of complex perianal fistulas - a severe, debilitating complication of Crohn’s disease. A global Phase III trial intended to support a future U.S. Biologic License Application (BLA) started in 2017. TiGenix has entered into a licensing agreement with Takeda, a global pharmaceutical company active in gastroenterology, under which Takeda acquired the exclusive right to develop and commercialize Alofisel for complex perianal fistulas outside the U.S. TiGenix’ second adipose-derived product, Cx611, is undergoing a Phase I/II trial in severe sepsis – a major cause of mortality in the developed world. TiGenix is headquartered in Leuven (Belgium) and has operations in Madrid (Spain) and Cambridge, MA (USA). For more information, please visit http://www.tigenix.com.

About Alofisel (darvadstrocel)

Alofisel is a local administration of allogeneic (or donor derived) expanded adipose-derived stem cells (eASCs) for the treatment of complex perianal fistulas in adult Crohn’s disease patients that have previously shown an inadequate response to at least one conventional therapy or biologic therapy. Crohn’s disease is a chronic inflammatory disease of the intestine and complex perianal fistulas are a severe and debilitating complication for which there is currently no effective treatment. Alofisel was granted orphan drug designation by the European Commission in 2009 and by the U.S Food and Drug Administration (FDA) in 2017. TiGenix completed a European Phase III clinical trial (ADMIRE-CD) in August 2015 in which both the primary endpoint and the safety and efficacy profile were met, with patients receiving Alofisel showing a 44% greater probability of achieving combined remission compared to control (placebo).1 A follow-up analysis was completed at 52 weeks3 and 104 weeks post-treatment, confirming the sustained efficacy and safety profile of the product. The 24-week results of the Phase III ADMIRE-CD trial were published in The Lancet  in July 2016.1Based on the positive 24 weeks Phase III study results, TiGenix submitted a Marketing Authorization Application to the European Medicines Agency (EMA). A global Phase III clinical trial (ADMIRE-CD II) intended to support a future U.S. Biologic License Application (BLA) started in 2017, based on a trial protocol that has been agreed with the FDA through a special protocol assessment procedure (SPA) (clinicaltrials.gov; NCT03279081). ADMIRE-CD II is a randomized, double-blind, placebo-controlled study designed to confirm the efficacy and safety of a single administration of Alofisel for the treatment of complex perianal fistulas in Crohn's disease patients. In July 2016, TiGenix entered into a licensing agreement with Takeda, a global pharmaceutical company active in gastroenterology, under which Takeda acquired exclusive rights to develop and commercialize Alofisel for complex perianal fistulas in Crohn’s patients outside of the U.S.

References

Panés J, García-Olmo D, Van Assche G, et al., Expanded allogeneic adipose-derived mesenchymal stem cells (Cx601) for complex perianal fistulas in Crohn’s disease: a phase 3 randomized, double-blind controlled trial. The Lancet. 2016; 388(10051): 1281-1290.

2 Data on file

3 Panés J, García-Olmo D, Van Assche G, et al., Long-term Efficacy and Safety of Stem Cell Therapy (Cx601) for Complex Perianal Fistulas in Patients With Crohn’s Disease. Gastroenterology. 2017. Available at: http://www.gastrojournal.org/article/S0016-5085(17)36726-4/fulltext. Accessed February 2018.

SOURCE: Takeda Pharmaceutical Co

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