On February 7, 2017 CANCER RESEARCH TECHNOLOGY, Cancer Research UK’s commercial arm, Tusk Therapeutics, an immuno-oncology company, and UCL (University College London) reported that they have signed a licence and collaboration agreement to research, develop and commercialise an antibody-based therapeutic against a target that plays a key role in immune suppression in cancer (Press release, Cancer Research Technology, JUL 7, 2017, View Source [SID1234523169]).

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Under the terms of the agreement, Tusk Therapeutics will receive an exclusive world-wide licence from CRT to develop and commercialise therapeutic antibodies against the target, originating from the Cancer Research UK-funded research of Dr Sergio Quezada and Professor Karl Peggs at UCL. In return, CRT will receive an upfront payment, future success-based milestones and royalty payments, which will be shared with UCL.

Tusk Therapeutics has additionally entered into a three-year collaboration with CRT and UCL to part-fund a programme of preclinical work, led by Dr Quezada, to evaluate candidate antibodies in various cancer models prior to initiation of formal preclinical and clinical development. At the end of the collaboration, Tusk Therapeutics will assume responsibility for accelerating the progress of selected antibody candidates into the clinic.

Dr Quezada said: "It is extremely exciting seeing years of preclinical work move closer to a potential real therapy for patients. We are very happy with the work to date and that which we will continue doing with the Tusk Therapeutics’ team."

Luc Dochez, CEO of Tusk Therapeutics, said: "We are proud to work together with Cancer Research UK and the group of Dr Quezada. The collaboration fits Tusk’s strategy of working with top researchers in the immune-oncology field and to bring promising assets from early stage discovery through development and to the clinic."

Dr Phil L’Huillier, CRT’s director of business management, said: "This collaboration brings together Cancer Research UK’s and UCL’s world-leading immune-oncology expertise with Tusk Therapeutics’ growing industry reputation for developing promising immune-modulating antibodies. It’s one of several projects now in our portfolio focused on the up-and-coming field of immune-oncology that we hope will accelerate progress towards exciting new treatments for cancer patients."

On February 6, 2017 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, reported a progress update on the clinical and regulatory status of ATIR101, the Company’s lead product to address the key risks and limitations of hematopoietic stem cell transplantation (HSCT) in blood cancer (Press release, Kiadis, FEB 6, 2017, View Source [SID1234517649]).

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Initiation of pivotal Phase III trial with ATIR101
Following regulatory approval from the national authority in Canada (Health Canada), the Company is pleased to announce the initiation of its randomized, controlled, transatlantic Phase III trial with ATIR101 (CR-AIR-009). Approximately 195 patients with acute leukemia will be enrolled in total in the Phase III trial and randomized 1:1 to receive a haploidentical allogeneic HSCT using either the Kiadis Pharma approach with a single dose of ATIR101 or the post-transplant cyclophosphamide approach (also known as the Baltimore Protocol). The trial protocol has also been submitted for approval and is being evaluated by the United States FDA (Food and Drug Administration) as well as several European regulatory authorities. Following approval the trial will be rolled out to additional study centers in the United States and Europe.

CTI Clinical Trial and Consulting Services, Inc., an international clinical contract research organization, has been appointed to work with Kiadis Pharma to support the clinical part of the trial and PCT, LLC, A Caladrius Company, a leading external manufacturing partner to the cell therapy industry, will manufacture ATIR101 for the United States and Canada. The German Red Cross Blood Donor Service, Baden-Wuerttemberg-Hessen, will remain the manufacturer in Europe. All 15 study centers that participated in the Company’s Phase II trials with ATIR101 (CR-AIR-007 and CR-AIR-008) have confirmed their intention to participate in the Phase III trial (CR-AIR-009). Kiadis Pharma is actively and rapidly aligning more sites with the aim of having more than 40 sites in North America and Europe participating in the Phase III trial.

Ongoing CR-AIR-008 trial continues to confirm safety profile of ATIR101
Infusing a single dose of ATIR101 continues to be safe in the ongoing Phase II trial with ATIR101 (CR-AIR-008) which, as previously announced, is continuing to treat patients with a single dose of ATIR101 according to the clinical protocol and the recommendation of the Independent Data Monitoring Committee (IDMC). Five patients in this trial have now been treated with a single dose of ATIR101 of which three were infused more than 120 to 150 days ago. None of these patients have shown any symptoms of severe Graft-versus-Host-Disease (GVHD), with none having received any prophylactic immunosuppression.

MAA submission to EMA for ATIR101 progressing well
Following the Company’s decision to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the use of ATIR101 in blood cancers, Kiadis Pharma confirms that the preparations for submitting the dossier are progressing well. The MAA will be based on the results from the Company’s successful single dose Phase II trial (CR-AIR-007) which confirmed that ATIR101 can be safely infused and shows statistically significant benefits on overall survival and reducing death from GVHD and infections, compared to an observational control group of patients having received a similar T-cell depleted stem cell transplant from a haploidentical family member but without the addition of ATIR101.

Kiadis Pharma has previously received an Advanced Therapy Medicinal Product (ATMP) certificate from EMA for manufacturing quality and pre-clinical data, recognizing that this data generated for ATIR101 meets the stringent standards imposed by the agency in evaluating an MAA.

Contingent on approval from EMA, Kiadis Pharma anticipates launching ATIR101 in to the European market in 2018.

Phase I/II trial with ATIR201
As previously announced, the Company’s Phase I/II clinical trial with its product ATIR201 for thalassemia (CR-BD-001) has received regulatory approval from the national authority in the United Kingdom (the MHRA, the Medicines and Healthcare products Regulatory Agency) as well as approval from the Ethics Committees of the Royal Manchester Children’s Hospital and the Birmingham Children’s Hospital. In addition, approval from the Ethics Committee of the University of Regensburg in Germany has now been received, with regulatory approval from the national authority in Germany pending. Kiadis Pharma expects the first clinical trial data to become available in the second half of 2017.

Manfred Rüdiger, PhD, Chief Executive Officer of Kiadis Pharma, commented: "I am very excited that we have initiated our pivotal Phase III trial with ATIR101. We have significant momentum now and the preparation of our MAA submission to EMA is progressing well. This work will bring our potentially life-saving treatment, which is also protected by Orphan Drug Designations in both Europe and the US, one step closer to patients with an anticipated launch in Europe in 2018."

About ATIR101
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.

On February 6, 2017 Cellectis (Alternext: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells (UCART), reported it has received an Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) to conduct Phase 1 clinical trials with UCART123, the Company’s most advanced, wholly owned TALEN gene-edited product candidate, in patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN) (Press release, Cellectis, FEB 6, 2017, View Source [SID1234517650]). This marks the first allogeneic, "off-the-shelf" gene-edited CAR T-cell product candidate that the FDA has approved for clinical trials. Cellectis intends to initiate Phase 1 trials in the first half of 2017.

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UCART123 is a gene-edited T-cell investigational drug that targets CD123, an antigen expressed at the surface of leukemic cells in AML, tumoral cells in BPDCN. The clinical research for AML will be led, at Weill Cornell, by principal investigator Dr. Gail J. Roboz, Director of the Clinical and Translational Leukemia Programs and Professor of Medicine. The UCART123 clinical program for BPDCN will be led, at the MD Anderson Cancer Center, by Dr. Naveen Pemmaraju, MD, Assistant Professor, and Professor Hagop Kantarjian, MD, Department Chair, Department of Leukemia, Division of Cancer Medicine.

AML is a devastating clonal hematopoietic stem cell neoplasm that is characterized by uncontrolled proliferation and accumulation of leukemic blasts in bone marrow, peripheral blood and, occasionally, in other tissues. These cells disrupt normal hematopoiesis and rapidly cause bone marrow failure and death. In the U.S. alone, there are an estimated 19,950 new AML cases per year, with 10,430 estimated deaths per year.

BPDCN is a very rare and aggressive hematological malignancy that is derived from plasmacytoid dendritic cell precursors. BPDCN is a disease of bone marrow and blood cells but also often affects skin and lymph nodes.

"The FDA’s approval of Cellectis’ UCART123 – the first "off-the-shelf" CAR T-cell product candidate to enter clinical trials in the U.S. – is a major milestone not only for the Company but also for the medical community, global biotech and pharmaceutical industries at large," said Dr. Loan Hoang-Sayag, Cellectis Chief Medical Officer. "Cellectis’ allogeneic UCART products have the potential to create an important shift with regard to availability, and cost-effectiveness, to make these therapies widely accessible to patient population across the world."

"After the National Institutes of Health’s Recombinant DNA Advisory Committee (RAC)’s unanimous approval of two Phase 1 study protocols for Cellectis’ UCART123 in December 2016, the FDA’s approval of Cellectis’ IND is a new major regulatory milestone achieved, for having UCART123 proceed into clinical development and reaching cancer patients in need," added Stephan Reynier, Chief Regulatory and Compliance Officer, Cellectis.

Information about ongoing clinical trials are publically available on dedicated websites such as:

www.clinicaltrials.gov in the U.S.

www.clinicaltrialsregister.eu in Europe

On February 6, 2017 Aura Biosciences, a biotechnology company developing a new class of therapies to target and selectively destroy cancer cells using viral nanoparticle conjugates, reported that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug application (IND) for the company’s lead program, light-activated AU-011 in ocular melanoma (OM) (Press release, Aura Biosciences, FEB 6, 2017, View Source [SID1234517651]). This active IND enables Aura to begin initial clinical testing of AU-011, a unique targeted therapy that could transform the primary treatment of patients with OM, a rare and life-threatening disease.

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"Early detection of ocular melanoma, combined with the administration of AU-011 as a potential vision-sparing therapy, could transform the treatment of patients with this devastating disease," said Brian Marr, M.D., Director of the Ophthalmic Oncology Service at Columbia University Medical Center. Dr. Marr is the principal investigator for the AU-011 clinical trial and also is a member of Aura’s Clinical Advisory Board.

"Receiving IND clearance to enter the clinic for AU-011 is an important step in the development pathway for this novel class of drugs, and I’m thankful to our team of dedicated employees, as well as to our distinguished scientific and clinical advisors, for their contributions that have propelled us to this point," said Elisabet de los Pinos, Ph.D., founder and CEO of Aura. "With the advancement of AU-011, we are opening the door for innovation in a completely new therapeutic area where there are no FDA drugs approved today. Our hope is that AU-011 could be used to treat small primary melanomas early, with the potential to eliminate the tumor and preserve vision for patients."

The Phase 1b open-label, single ascending dose clinical trial currently enrolling is designed to evaluate the safety, immunogenicity and preliminary efficacy of two dose levels of AU-011 for the treatment of small-to-medium primary OM. Screening procedures for eligible patients are underway at five clinical trial sites across the country. For more information, visit www.clinicaltrials.gov or contact [email protected].
About ocular melanoma (OM)
Ocular melanoma (OM), also known as uveal or choroidal melanoma, develops in the uvea, or uveal tract, of the eye, and is an aggressive and rare eye cancer. No targeted therapies are currently available, and current treatments are associated with serious morbidities. The most common treatment today is placing an invasive radioactive plaque against the exterior of the eye near the tumor, which requires multiple surgeries and can lead to cataracts, retinopathy and loss of vision. The alternative is enucleation, the removal of the eye. OM metastasizes to the liver in about half of all cases (source: OMF), and only 15 percent of patients whose OM has metastasized survive beyond five years after diagnosis (source: ACS).

About light-activated AU-011
AU-011 is a first-in-class targeted therapy in development for the primary treatment of ocular melanoma (OM), also known as uveal or choroidal melanoma, a rare and life-threatening disease. The therapy consists of viral nanoparticle conjugates that bind selectively to cancer cells in the eye. AU-011 has a necrotic mechanism of action and is administered through an intravitreal injection into the eye. Upon activation with an ophthalmic laser, the drug rapidly and specifically destroys the membranes of tumor cells while sparing key eye structures, which may allow for the potential of preserving patients’ vision. AU-011 for OM has been granted orphan drug designation by the U.S. Food and Drug Administration and is currently in clinical testing.

On February 6, 2017 Genmab A/S (Nasdaq Copenhagen: GEN) reported that it has updated its 2016 Financial Guidance (Press release, Genmab, FEB 6, 2017, View Source [SID1234517639]). Based on preliminary reporting, Genmab is improving its 2016 financial guidance published on December 20, 2016 .

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The table below summarizes our revised and previous guidance.
MDKK Revised Guidance Previous Guidance
Revenue 1,790 — 1,840 1,720 — 1,770
Operating expenses (750) — (800) (800) — (850)
Operating income 1,015 — 1,065 895 — 945
Cash position at end of year* 3,850 — 3,950 3,650 — 3,750
*Cash, cash equivalents, and marketable securities
The revenue range has increased primarily due to higher royalties on net sales of DARZALEX by Janssen and achievement of additional DuoBody milestones. Among other items, the operating expense range has decreased due to timing of clinical and pre-clinical expenses. The operating income range has increased due to the combination of higher revenue and lower expenses. Our cash position has increased mainly due to timing of operating expenses and other working capital adjustments.
As previously announced, the annual report for 2016 will be published on February 22, 2017.