T cell replacement therapy

Unassigned

New Medicines

Graft versus host disease (GvHD) in patients with haematological cancers - adjuvant therapy after haematopoietic stem cell transplant

Information

Advanced therapy medicinal product (ATMP)
Kiadis
Kiadis

Development and Regulatory status

Pre-registration (Filed)
Pre-registration (Filed)
None
Yes
Yes

Dec 18: Kiadis intends to commercially launch ATIR101 itself in a first EU member state in H2 19, and notes there are only 64 major transplant clinics in 4 EU countries (Germany, UK, Italy & France). In the US, they will need to file PIII data (currently study is ongoing), and notes that transplant provision is very concentrated with 27 clinics doing 50% of all transplants. Kiadis is setting up its own manufacturing facility, building up its own commercial organisation [10].


Jan 18: The EMA has previously granted an Advanced Therapy Medicinal Product certificate to ATIR 101 for manufacturing quality and non-clinical data [9].


Jan 18: Also has orphan drug status in US for reduction of transplant related mortality caused by graft versus host disease (GvHD) and/or infections following allogeneic bone marrow transplantation and as a therapy for immune reconstitution and prevention of GvHD following allogeneic bone marrow transplantation [9].


Jan 18: Kiadis Pharma expects to receive a positive opinion and a conditional approval from the EMA during H2 2018 and launch the product in the EU in 2019 [9].


Sep 17: ATIR101 granted regenerative medicine advanced therapy (RMAT) designation by the US FDA for the treatment of blood cancers [9].


Apr 17: Filed in EU under the centralized procedure as an adjunctive treatment for haematopoietic stem cell transplantation (HSCT) for the treatment of malignant disease. The application was based on results of a single dose pivotal PII clinical trial and following positive interactions with the EMA Rapporteur and Co-Rapporteur [9].


Jun 16: Kiadis intends to gain approval of the drug in blood cancers, & expects to launch the product in 2018. Kiadis also intends to file a marketing authorisation application with the FDA and Health Canada in 2019 [6].


Jun 16: Kiadis Pharma intends to file a MAA with the EMA in Q1 2017 [6].


Jun 16: ATIR101 is a designated orphan drug in the EU [5].

Category

ATIR101 utilises the TH 9402 compound in conjunction with its TheraluxTM light exposure technology to selectively eliminate specific alloreactive T cells ex vivo from haploidentical donor samples before infusing the donor sample in the patient.
In 2013/14, 1,060 patients in the UK were transplanted with unrelated donor stem cells. It is estimated that between 20% and 80% of patients undergoing an allogeneic HSCT will develop some form of GvHD. In 2014-15, there were 2,144 admissions for bone marrow transplant rejection (ICD-10 T86.0) in England [1-3].
Graft versus host disease (GvHD) in patients with haematological cancers - adjuvant therapy after haematopoietic stem cell transplant
Intravenous infusion

Trial or other data

Jul 17: PII study (NCT02500550) is still recruiting. Due to complete collecting primary outcome data in Apr 18 [8].


Aug 16: PII (NCT02500550) study is still recruiting, & data collection should complete Mar 17 [7].


Dec 15: First patient enrolled in an open-label, PII study, which is designed to assess the safety and efficacy of a two-dose regimen of ATIR101 immunotherapy in patients with haematologic malignancies, who received a CD34-selected haematopoietic stem cell transplantation from a haploidentical donor (CR-AIR-008; NCT02500550). Recruitment of approximately 15 patients is ongoing in Belgium, Canada, and Germany, and will expand to the UK. Incidence of acute graft versus host disease (GvHD) grade III/IV will be investigated as the primary endpoint. Kiadis intends to continue the PII trial into a randomised, controlled PIII pivotal study in the H2 2016 [6].


Mar 13: PII (NCT01794299) study to determine whether ATIR is safe and effective in reducing transplant-related mortality and improving overall survival, when infused in patients with a hematologic malignancy following a T-cell depleted stem cell graft from a related haploidentical donor begins. 31 adults will be recruited in Belgium, Canada, Germany and the UK. Primary outcome is transplant-related mortality (TRM) at 6 months post HSCT; collection of these data should complete Jun 16 [4].