UPDATED: Takeda inks another biotech pact as it pays $400M+ to TiGenix

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Less than a week after stumping up cash for a one-drug-wonder biotech (and an option to buy it out), Japan’s Takeda has struck another biotech deal, this time with Belgium-based TiGenix. But it's not all good news as TiGenix announces that it has withdrawn a marketed stem cell drug from the EU while consequently killing off several other deals.

First, to the research pact, which sees €25 million ($27.8 million) paid upfront from Takeda with around €355 million ($395 million) plus royalties also on the table, allows the pharma rights (outside of the U.S.) to license, develop and sell Cx601--a suspension of allogeneic adipose-derived stem cells injected intralesionally for the treatment of complex perianal fistulas in patients with Crohn's disease.

TiGenix said that its first milestone payment, worth €15 million, will be handed out if it gains European approval for Cx601. And it shouldn’t have to wait too long as the biotech submitted its drug for EMA approval back in March, and has since 2009 had an orphan designation for its candidate. A decision by the regulator is expected by mid-2017.

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Should the EMA green light Cx601, Takeda will under the pact then become the marketing authorization holder and be responsible for all commercialization and regulatory activities.

The Japanese company will also be accountable for any further studies of Cx601 in complex perianal fistulas in Crohn's disease. TiGenix will, however, retain the rights to develop Cx601 in new indications.

FDA approval is further down the road, but TiGenix said it is “preparing to develop Cx601 for the U.S. market” after having reached an agreement with the FDA through an SPA on its proposed protocol last August.

Takeda has clearly been wooed by some strong data from the drug, which the biotech published in the fall of last year and used for its EMA submission.

In the data, which boosted its share price by 20% on the day they were posted, the 289-person Phase III trial showed that in patients with complex perianal fistulas, about half of the participants who received Cx601 were in remission after 24 weeks.

If TiGenix can win EMA approval and replicate these results in the real world, it could provide a new therapy option for people who have failed to respond to other treatments.

Stem cells products, so full of promise and hype, have in recent years failed to live up to these expectations in the clinic, with many biopharmas posting disappointing data in later stages of its research.

Takeda is betting that Cx601’s achievements in the lab can be replicated in the real world and be one of the few stem cell success stories, with analysts betting that the candidate could see blockbuster sales--something that has become more likely given that the marketing might of Takeda will now be behind it.

"TiGenix is pleased to partner with Takeda, a global pharmaceutical company with a strong track record and strong leadership position in gastroenterology. This agreement reduces the investment risks associated with building a pan-European marketing and selling infrastructure, and helps get this much-needed treatment option to patients and gives to Cx601 the best partner with the needed capabilities and resources to secure its commercial success," said Eduardo Bravo, CEO of TiGenix.

"This agreement further provides TiGenix with the financial strength to move forward with the clinical development of Cx601 in the U.S., which represents approximately 50% of the world's Crohn's market."

This comes only a few days after Takeda said it would help out the single-asset biotech Alto in its early-stage study for ATC-1906, which is being tested for certain aspects of gastroparesis (GP)--a chronic condition in which the stomach cannot empty itself in the normal way.

Takeda actually went one step further with this deal as it also holds an option to buy out the tiny Californian biotech at the end of Phase I testing--at which point it would take over complete control of ATC-1906’s development future.

These deals dovetail with Takeda’s gastro portfolio, which includes its recently launched Entyvio (vedolizumab) for ulcerative colitis and Crohn’s disease, which is set for $2 billion in sales at peak. It will hope to find the next-gen version of these types of gastro meds with its biotech investments.  

Elissa Johnsen, head of global product & pipeline communications at Takeda, told FierceBiotech that it the pharma is now “focused in Europe on becoming a specialty care company”, and therefore moving away from primary care.

Johnsen said that the clinical data for Cx601 “are very encouraging” and “formed part of the reasoning behind the collaboration.” When asked about whether, as with Altos, Takeda may look to buy out TiGenix, Johnsen was tight-lipped, saying only that Takeda “does not comment on business development activities.”

TiGenix is also developing stem cell platforms with several Phase II product candidates targeting large indications such as heart attack and sepsis currently ongoing. The biotech filed with Nasdaq for a $58 million IPO earlier this year.

But the company also decided to sneak out another release, separate from its Takeda deal, which this morning announced that it has now in fact withdrawn its EU license for the autologous chondrocyte implant ChondroCelect, which was indicated for patients with cartilage lesions of the knee.

ChondroCelect is a cell-based medicinal product that was used in autologous chondrocyte implantation in which cells are taken from the patient’s own knee, multiplied to reach a large quantity, and then re-implanted at the site of the defect.

In a statement, the biotech blamed a tough regulatory environment, a lack of ROI in Europe and a “tough competitive environment” as the key reasons for its withdrawal.

This has also had the knock-on effect that the biotech will kill off its commercial deal with Sobi and its manufacturing agreement with PharmaCell. 

Bravo said: “To deliver shareholder value we need to focus all our efforts and internal resources on the upcoming Cx601 Phase III U.S. trial while advancing with our other clinical stage assets, namely AlloCSC-01 in acute myocardial infarction and Cx611 in severe sepsis.” 

- read TiGenix’ ChondroCelect withdrawal statement
- check out the release

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