AMSTERDAM--(BUSINESS WIRE)--Regulatory News:
Kiadis Pharma N.V. (“Kiadis Pharma” or the “Company”) (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, today announces its unaudited financial results for the six months ended June 30, 2016, which have been prepared in accordance with the IAS 34 “Interim Financial Reporting” as adopted by the European Union.
Operating highlights (including post period end)
- Positive results announced on the primary endpoint from the single dose Phase II CR-AIR-007 trial (ATIR101™)
- Decision to file for marketing authorization with the European Medicines Agency (EMA) for ATIR101™ in blood cancers – submission on course for Q1 2017
- PCT appointed for US manufacture of ATIR101™ for Phase III trial
- Orphan Drug Designation for ATIR101™ further expanded to include treatment in a hematopoietic stem cell transplantation
- Supervisory Board strengthened with appointment of Dr. Robert Soiffer and Mr. Berndt Modig
- Partnership announced with The Leukemia & Lymphoma Society around the development of ATIR101™ in acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) patients
- Preparations to commence the CR-AIR-009 (ATIR101™) Phase III trial ongoing with initiation expected on track by end 2016
- Second dose CR-AIR-008 Phase II trial ongoing – safety read-out now expected in Q1 2017
- Initiation of ATIR201™ Phase I/II thalassemia trial (CR-BD-001) on track for patient enrollment in Q4 2016
(Amounts in EUR million, except per share data)
|Total revenue and other income||-||-||-|
|Total operating expenses||(5.1)||(11.7)||6.6|
|Research and development||(3.8)||(4.7)||0.9|
|General and administrative||(1.3)||(7.0)||5.7|
|Net financial result||(1.4)||2.2||(3.6)|
|Net operating cash flow||(5.3)||(3.6)||(1.7)|
|Cash position at end of period||23.7||2.5||21.2|
|Earnings per share before dilution (EUR)||(0.48)||(0.89)||0.40|
A full financial report for the six months ended June 30, 2016 is available on Kiadis Pharma’s website at http://www.kiadis.com/financial-news/.
Commenting on the financial results, Manfred Rüdiger, CEO of Kiadis Pharma, said: “The period has been extremely busy with the progress made on our lead product ATIR101™. Our team is focused on the marketing authorization application and advancing our clinical trials, particularly the key Phase III study, as expediently as possibly whilst coordinating with both the regulators and world leading experts. We continue to be very confident in our plans and products and keep our target of helping patients whilst creating value for shareholders.”
Update on clinical product progress
- Ongoing single dose Phase II trial (CR-AIR-007) with ATIR101™
In April 2016, the Company presented positive results on the primary endpoint of its single dose Phase II trial (NCT01794299/EudraCT 2012-004461-41) with its lead product ATIR101™ at the 42nd Annual Meeting of the European Society of Blood and Marrow Transplantation (EBMT) in Valencia, Spain.
The data confirmed that ATIR101™ can be safely infused, does not cause grade III-IV Graft-versus-Host-Disease (GVHD) and shows a significant reduction in Transplant Related Mortality (TRM) and a significant improvement in Overall Survival (OS) in comparison to a historical control group of patients undergoing a T-cell depleted haploidentical donor transplantation only.
The trial is ongoing with enrollment having been completed and patients being followed up for 24 months post transplantation in order to collect further long-term outcome data.
- Preparations for Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for ATIR101™
In June 2016, the Company announced its decision to file for marketing authorization with the EMA for its lead product ATIR101™ in blood cancers and Kiadis Pharma has started compiling the dossier for this submission. The submission will be based on the results from the ongoing single dose Phase II trial and the Company expects to submit the application to EMA in Q1 2017, in line with latest guidance.
- Preparations for the Phase III trial (CR-AIR-009) with ATIR101™
Haploidentical hematopoietic stem cell transplantation (HSCT) has become a highly dynamic field of great interest and several meetings have been held with regulatory authorities, including the United States Food and Drug Administration (FDA), and with international world-leading Key Opinion Leaders (KOLs) to discuss and optimize the Phase III trial design for testing ATIR101™ in patients with blood cancer.
The Phase III study design takes into account the top line data from the single dose Phase II trial which showed a strong and statistically significant Overall Survival advantage for patients who received ATIR101™. This Phase III trial will therefore be based on a single dose treatment of ATIR101™ to reflect the previous Phase II enrollment criteria and treatment schedule and, most importantly, to minimize Phase III study risks that often result from design changes made vis-à-vis a Phase II trial.
The Phase III trial will consequently be based on a two arm study randomizing patients to receive a haploidentical HSCT according to either the post-transplant cyclophosphamide approach (the so-called “Baltimore protocol”) or the Kiadis Pharma approach using a T-cell depleted haploidentical HSCT with a single dose of ATIR101™.
The Company is currently finalizing the clinical protocol for this trial for submission in early Q4 2016 to the national authorities in Canada, Belgium, the United States and Germany. Kiadis Pharma aims to have the first clinical centers open to enroll patients, as previously guided, by the end of 2016.
- United States-based ATIR101™ manufacturer for the Phase III trial
In June 2016, Kiadis Pharma announced that it had appointed PCT, LLC, A Caladrius Company, as the Company’s contract manufacturing organization (CMO) in the United States for the supply of study medication to the clinical trial sites in the United States and Canada that are expected to participate in the Company’s planned Phase III clinical trial. With regards to the manufacture of study medication for the European clinical sites in this Phase III trial, Kiadis Pharma already has a long-running and successful manufacturing collaboration with the German Red Cross Blood Donor Service, Baden-Wuerttemberg-Hessen.
- Ongoing second dose Phase II trial (CR-AIR-008) with ATIR101™
Whilst progressing on track with the key Phase III trial preparations, the Company continues to evaluate the safety of a second dose of ATIR101™ in its ongoing Phase II trial (NCT02500550/EudraCT 2015-002821-20) in patients with blood cancer. The second dose trial is enrolling patients with currently nine out of fifteen patients recruited onto the study. Whilst physicians have expressed interest in any additional potential upside and product flexibility in administering a second dose of ATIR101™, should it be needed, they are also very keen to start treating patients in the Phase III (CR-AIR-009) trial as swiftly as possibly in order to compare the Kiadis Pharma approach with the Baltimore protocol approach. The Company now anticipates the safety read-out of the trial in Q1 2017, due to slower than anticipated enrollment.
- Preparations for Phase I/II trial (CR-BD-001) with ATIR201™
As previously guided, the planned Phase I/II trial for ATIR201™ for use in children suffering from thalassemia major is on track to start enrolling patients in hospitals in the United Kingdom and Germany in Q4 2016 and the clinical protocol is currently under review by the national authorities.
For patients suffering from blood cancers, an allogeneic hematopoietic stem cell transplantation (HSCT) is generally regarded as the most effective curative approach. During an HSCT treatment, the bone marrow, harboring the diseased cancer cells, is completely destroyed and subsequently replaced by stem cells in the graft from a healthy donor. After an HSCT treatment it usually takes the patient at least six to twelve months to recover to near-normal blood cell levels and immune cell functions. During this period, the patient is highly vulnerable to infections caused by bacteria, viruses and fungi but also to disease relapse.
ATIR101™ (Allodepleted T-cell ImmunotheRapeutics) provides for a safe donor lymphocyte infusion (DLI) from a partially matched (haploidentical) family member without the risk of causing severe Graft-versus-Host-Disease (GVHD). The T-cells in ATIR101™ will help fight infections and remaining tumor cells and thereby bridge the time until the immune system has fully re-grown from stem cells in the transplanted graft.
In ATIR101™, T-cells that would cause GVHD are eliminated from the donor lymphocytes using Kiadis Pharma’s photodepletion technology, minimizing the risk of GVHD and eliminating the need for prophylactic immune-suppression. At the same time, ATIR101™ contains potential cancer killing T-cells from the donor that could eliminate residual cancer cells and help prevent relapse of the disease, known as the Graft-versus-Leukemia (GVL) effect.
Therefore, ATIR101™, administered as an adjunctive immuno-therapeutic on top of HSCT, provides the patient with functional, mature immune cells from a partially matched family donor that can fight infections and tumor cells but that do not cause GVHD. ATIR101™ thus has the potential to make curative HSCT a viable option to many more patients.
The Company estimates that approximately 35% of patients who are eligible and in urgent need of HSCT will not find a matching donor in time. A partially matched (haploidentical) family donor, however, will be available to over 95% of patients.
ATIR101™, consisting of donor T-cells that fight infections and residual tumor cells while not eliciting severe GVHD, is designed to result in low relapse rates and low rates of death due to infections, in the absence of severe acute GVHD.
About Kiadis Pharma
Kiadis Pharma is focused on cell-based immunotherapy products for the treatment of blood cancers and inherited blood disorders. The Company’s products have the potential to address the risks and limitations connected with allogeneic hematopoietic stem cell transplantation (HSCT), namely: Graft-versus-Host-Disease (GVHD); cancer relapse; opportunistic infections; and limited matched donor availability. The Company believes that HSCT could become a first-choice treatment for blood cancers, inherited blood disorders and possibly autoimmune diseases and solid organ transplantations.
In April 2016, the Company reported positive Phase II results with its lead product ATIR101™ in patients with blood cancer. The data showed that ATIR101™ significantly reduced Transplant Related Mortality and significantly improved Overall Survival. In addition, ATIR101™ did not elicit grade III-IV GVHD in any patient. A Phase III clinical trial with ATIR101™ is expected to be initiated in the second half of 2016. ATIR101™ has been granted Orphan Drug Designations both in the US and Europe. The Company’s second product candidate, ATIR201™, addresses inherited blood disorders with an initial focus on thalassemia, a disease which results in destruction of red blood cells in patients. ATIR201™ is expected to enter Phase I/II clinical development in the second half of 2016.
Kiadis Pharma, based in Amsterdam, The Netherlands, was granted an Advanced Therapy Medicinal Product (ATMP) certificate for manufacturing quality and non-clinical data by the European Medicines Agency (EMA). The Company’s shares are listed on Euronext Amsterdam and Euronext Brussels. For more information visit www.kiadis.com
Forward Looking Statements
Certain statements, beliefs and opinions in this press release are forward-looking, which reflect Kiadis Pharma’s or, as appropriate, Kiadis Pharma’s directors’ current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, Kiadis Pharma expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither Kiadis Pharma nor its advisers or representatives nor any of its subsidiary undertakings or any such person's officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.