1stOncology™: Cancer Intelligence Service – Page 15754 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

SCIB1 Drug Product Supply

On June 17, 2016 Scancell Holdings Plc, (AIM:SCLP), the developer of novel Immunotherapies for the treatment of cancer, reported that it is suspending dosing with the current clinical trial supplies of SCIB1 with immediate effect (Press release, Scancell, JUN 17, 2016, View Source [SID:1234513447]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

Ongoing quality control analysis has revealed that the stored drug product is no longer within the original specification. In discussion with the MHRA Clinical Trials Unit, and with patient safety of primary importance,
the Company has concluded that it is no longer suitable for further use, although no new side effects have emerged.

The suspension of dosing affects eight patients in the long term extension arm of the Phase 1/2 trial, SCIB1- 001 (out of the 35 patients that have been dosed), investigating SCIB1 as a monotherapy for the treatment
of melanoma. All study investigators have been informed and their patients will be notified as soon as possible.

SCIB1-001 was originally started in 2010 with a prospectively planned treatment period of only six months. However, continuing encouraging results and an excellent side effect profile led to successive amendments
to the clinical trial protocol to investigate increasing doses of the drug and eventually to examine a long term dosing regimen. As a result, some of the trial materials have now been stored for over 7 years.

The Company is planning to make a fresh batch of SCIB1 and has recently signed an agreement with a new GMP manufacturer. The primary reason for this is to support a new study of SCIB1 in combination with a
checkpoint inhibitor, as previously announced. However, the Company also intends to make this material available to patients currently in the long term extension of SCIB1-001 who wish to continue receiving the
drug. While it is expected that there will be a delay of approximately 9-12 months before the new SCIB1 material will be available for clinical use, it is anticipated that the anti-tumour response induced by SCIB1 should persist and eliminate any remaining tumour cells. Further doses of SCIB1 have been given for added protection to ensure the immune cells continue to patrol for any emerging cancer cells; subsequent administration of the new SCIB1 material should reactivate these memory immune cells to eliminate any recurring tumours.

Prof Poulam Patel, Chief Investigator for the SCIB1-001 clinical trial and Professor of Clinical Oncology at the University of Nottingham commented: "Results from the SCIB-001 trial to date have been very
encouraging and SCIB1 clearly warrants further investigation as a potential treatment for melanoma.

However as patient safety is our primary concern, the deterioration of stored clinical trial material from the original specification necessarily means that dosing of SCIB1 must be suspended until new drug product
becomes available."

Dr Richard Goodfellow, CEO of Scancell added: "Patient safety has always been our primary responsibility. Although we have seen no new adverse events it is unfortunate, but nevertheless appropriate, that we
suspend dosing of SCIB1 at this time while we work as quickly as possible to secure new supplies of this promising potential treatment for melanoma. Starting further efficacy studies with SCIB1 is only possible due to the results we have seen so far in the long-running SCIB1-001 study and we would again like to convey our thanks to the patients in that trial for their participation and support over the past 6 years."

MorphoSys Reports Publication of Clinical Case Report of a Blood Cancer (DLBCL) Patient Showing Long-Lasting Complete Remission of Currently 26 Months under MOR208 Treatment

On June 17, 2016 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) reported that a case study report from an ongoing clinical phase 2a study with its proprietary drug candidate MOR208 in patients with different subtypes of relapsed or refractory non-Hodgkin’s lymphoma (NHL) has been published in the Journal of Medical Case Reports (Press release, MorphoSys, JUN 17, 2016, View Source [SID:1234513435]).

The case report shows that 3rd line monotherapy with the anti-CD19 antibody MOR208 has resulted in an ongoing complete response (CR), of currently more than 26 months’ duration, in a patient suffering from a morphological variant of diffuse large B-cell lymphoma (DLBCL). DLBCL – an aggressive type of blood cancer where B cells from the body’s immune system become malignant – is the most common form of NHL. As further reported, quality of life and performance status of the patient have remained high under MOR208 therapy, as evaluated by WHO grade 0 and Karnofsky score of 100%. MOR208 could be administered on an out-patient basis. Before being enrolled in the study with MOR208, the patient had a very dismal prognosis after early relapse to two prior lines of treatment, both including chemotherapy in combination with an anti-CD20 therapy with rituximab.

"Patients with NHL, who are refractory or show early relapse after previous therapies containing anti-CD20 treatment, have very limited treatment alternatives and usually a very poor prognosis. This reported case is one out of several examples of heavily pre-treated blood cancer patients showing long-lasting and still ongoing complete responses under anti-CD19 treatment with MOR208. This further encourages us to develop MOR208 as a potential new therapy for patients suffering from B cell malignancies, in particular DLBCL, in phase 2 combination studies," said Dr. Arndt Schottelius, Chief Development Officer of MorphoSys AG.

As previously reported, in the DLBCL subgroup overall response rate (ORR) was 36% (based on evaluable patients). In DLBCL, median duration of response (Kaplan-Meier estimates) was 20 months with three ongoing responses of up to more than 26 months. Patient evaluation and data analysis of the clinical phase 2a trial are ongoing. 92 patients were enrolled in the trial.

The full article published in the Journal of Medical Case Reports 2016 can be found here.

Further details of the case report
The patient, a 33 year-old male suffering from T-cell/histiocyte-rich large B-cell lymphoma, a morphological variant of DLBCL, had previously experienced early relapses after both, first-line treatment with rituximab combined with chemotherapy (R-CHOP) (relapse after 5 months) and second-line salvage chemotherapy including rituximab consolidated with an autologous stem-cell transplant (relapse after 6 months). Subsequently, the patient has been enrolled in the phase 2a trial of single-agent MOR208. In the study, the patient was treated for 12 weeks with 12mg/kg MOR208, administered intravenously once a week, followed by an ongoing maintenance therapy with administration of MOR208 every second week. Three months after starting 3rd line treatment with MOR208, the patient PET-CT assessments demonstrated a partial response (PR). Nine months later, during the ongoing maintenance therapy, PET-CT confirmed a complete response (CR). As stated in the case report, the patient experienced only few adverse events, which were all limited to infections of the respiratory tract. The quality of life and performance status of the patient has remained high during treatment, as measured by WHO grade 0 and Karnofsky score of 100%, with MOR208 treatment being administered on an out-patient basis. The patient’s complete response is ongoing, with a current duration of more than 26 months (24 months duration at the time of manuscript acceptance). The full article published in the Journal of Medical Case Reports 2016 10:123 can be found here.

About MOR208
MOR208 is an anti-CD19 antibody with a proprietary modification to the Fc portion in clinical development to treat B cell malignancies.

An open-label, phase 2a, multicenter study was designed to assess the activity and safety of weekly doses of 12 mg/kg MOR208 as a single agent in 92 pre-treated patients with various subtypes of relapsed/refractory NHL patients including diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), and other indolent NHL (iNHL) including follicular lymphoma (FL). All patients had received at least one prior rituximab-containing therapy. According to the data observed, MOR208 showed a low level of infusion reactions. According to a subgroup analysis data presented at the ASCO (Free ASCO Whitepaper) 2016 annual meeting, a disease control rate (CR + PR + SD) of 40% in DLBCL and 73% in iNHL patients was observed. Progression-free survival (PFS) with MOR208 therapy was shown to be comparable in rituximab refractory and non-refractory NHL patients (median PFS 5.3 versus 6.6 months, HR 0.85, 95% CI 0.45-1.6, p=0.59) thus demonstrating in this trial an activity of MOR208 independent of the response to a prior anti-CD20 therapy. Updated data for the overall trial population furthermore revealed that after 12 months the PFS rate was 40% in both DLBCL and iNHL patients. Nine patients treated with MOR208 were still in remission (7 CRs, 2 PRs), the longest responses currently ongoing for 26 months. The overall response rate (ORR) of MOR208 reached 36% in the DLBCL subgroup and 33% in iNHL patients (both based on evaluable patients). Based on all patients with DLBCL and iNHL in the study, the ORR was 26% and 29%, respectively.

In April 2016, the first patient was dosed in a phase 2 combination trial (L-MIND study) investigating safety and efficacy of MOR208 in combination with lenalidomide in 80 patients with relapsed or refractory DLBCL. Furthermore, MorphoSys in 2016 plans to start the safety part of a clinical trial evaluating MOR208 in combination with the chemotherapeutic agent bendamustine in DLBCL which is intended to lead into a pivotal study to start in 2017. In addition, at the ASCO (Free ASCO Whitepaper) 2016 Annual Meeting the trial design of a phase 2 study with MOR208 was presented which trial is planned to evaluate MOR208 in combination with idelalisib in chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) in patients no longer responding to or no longer tolerating Bruton’s tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib). After the discontinuation of several combination trials of idelalisib with other compounds, this planned trial is currently under review and discussions with regulatory authorities are ongoing. MorphoSys is currently exploring alternative study designs to evaluate MOR208 in a combination trial in CLL/SLL patients previously treated with a BTK inhibitor.

OXiGENE Announces Name Change to Mateon Therapeutics

On June 17, 2016 OXiGENE, Inc. (Nasdaq:OXGN), a biopharmaceutical company developing vascular disrupting agents (VDAs) for orphan oncology indications, reported that it has changed its name to Mateon Therapeutics, Inc., effective immediately (Press release, OXiGENE, JUN 17, 2016, View Source [SID:1234513451]).

On the NASDAQ Capital Market, the name change will be effective for trading purposes as of market open on June 20, 2016. At that time, shares of Mateon’s common stock will begin trading under the new ticker symbol "MATN". In connection with the name change, Mateon’s common stock has been assigned a new CUSIP number of 57667K109.

"Today’s name change signals a new day for vascular targeted therapies and our commitment to integrate them into the cancer treatment paradigm," said William D. Schwieterman, M.D., President and Chief Executive Officer of Mateon. "Over the past year, we redefined our clinical and regulatory strategies to position the company and our lead product candidate, CA4P, on what we believe to be the shortest path towards approval in an indication with the highest probability of success."

Since joining the company in May 2015, Dr. Schwieterman has built a world-class management and clinical development team with deep expertise in drug development. Dr. Schwieterman concluded, "With our team and strategy now firmly in place, we are ready to build upon the work of the past and are committed to realizing the full potential of combination vascular targeted therapy in orphan oncology indications."

OXiGENE was named for its founding technology of oxygen-mediated radiosensitizers. The new company name, Mateon Therapeutics, is derived from the company’s location in San Mateo County, the birthplace of biotechnology.

Cell Medica and Baylor College of Medicine announce exclusive licensing agreement and co-development partnership to create next generation cellular immunotherapy products for the treatment of cancer

On June 17, 2016 Cell Medica, a leader in developing, marketing and manufacturing cellular therapeutics for cancer and infections, reported a ground-breaking co-development partnership with Baylor College of Medicine (Baylor) to develop next-generation technologies for engineering immune cells with enhanced functions for the treatment of solid tumors (Press release, Cell Medica, JUN 17, 2016, View Source [SID:1234513448]).

The collaboration provides Cell Medica with an exclusive license over several Baylor cell and gene technologies and an option to license new products introduced into the co-development partnership by Baylor’s leading research teams in the field of genetically engineered immune cells.

Next generation technology

The collaboration will build on the recent clinical success of advanced chimeric antigen receptors (CARs) to enable human immune cells to recognize and kill cancer cells expressing tumor-associated antigens. The development plan will apply the CAR technology to natural killer T (NKT) cells as a novel immune cell type with biological properties that may be particularly effective for targeting solid tumors. NKT cells are known to infiltrate tissues where solid tumors arise and kill both malignant cells and cancer-enabling cells such as tumor-associated macrophages. The development plan also includes a genetically engineered T cell receptor (TCR) for use in NKT cells and T cells. The next-generation product concepts will combine the targeting aspects of CAR and TCR technologies with functional engineering to enable the modified immune cells to counteract the powerful inhibitory mechanisms that tumor cells deploy to evade the immune response. Cell Medica expects the collaboration to generate a significant number of new products for its cellular immunotherapy pipeline.

Co-development structure

The collaboration will accelerate the pioneering work of Dr. Leonid Metelitsa, Professor of Paediatrics – Oncology, with CAR-modified NKT cells. In published research, Metelitsa and his team have shown the potential therapeutic advantages of functionally enhanced CAR-modified NKT cells in pre-clinical cancer models. Metelitsa’s research team is part of Texas Children’s Cancer Center and the Center for Cell and Gene Therapy (CAGT) at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist Hospital. The CAGT has more than 20 years of experience working with genetically modified immune cells for the treatment of cancer and has conducted more than 40 clinical studies investigating cellular immunotherapies for the treatment of cancer.

Within the co-development structure, Baylor will conduct the pre-clinical and Phase I clinical research under the guidance of the Joint Steering Committee including members from both organizations. Cell Medica will work in parallel to support early product development and will use its substantial experience in manufacturing clinical-grade cell therapies since 2008 to establish robust production processes suitable for industrial scale-up. Following completion of successful Phase I studies, the products will transfer to Cell Medica for later-stage clinical development and commercialization.

The co-development funding will be available to research teams at Baylor with respect to new technologies, which may be utilized to create therapeutic products using modified NKT cells and other immune cells or to improve manufacturing systems. Baylor’s Innovation Development Center (IDC), led by Andrew Wooten, helped to structure this unique co-development partnership and will be responsible for Baylor’s alliance management function. As part of these responsibilities, the IDC will identify complementary technologies from ongoing Baylor research programs that may be included within the co-development plan.

Five product programs have been defined for the initial development plan in addition to a general process technology program. The preclinical development work for two of these programs will be executed by Drs. Gianpietro Dotti and Barbara Savoldo at the Lineberger Comprehensive Cancer Center, University of North Carolina. The Joint Steering Committee for the collaboration will review new product opportunities on a regular basis.

The co-development partnership builds upon the ongoing successful collaboration between Baylor and Cell Medica, supported by the Cancer Prevention and Research Institute of Texas (CPRIT), to develop baltaleucel-T for the treatment of a range of cancers associated with the oncogenic Epstein Barr virus.

License, Option and Co-development Arrangements

Cell Medica has entered into a License and Option Agreement for two platform patents related to engineered NKT cells, three target cancer antigens for CAR-modified NKT cells and a T cell receptor (TCR) technology. In addition, Baylor and Cell Medica have signed a co-development agreement under which Cell Medica will fund research aimed at new products as well as concepts/technologies in both oncology and non-oncology applications. Cell Medica will have an exclusive option to license future products within the co-development plan. Cell Medica has paid an up-front fee for the exclusive licensing arrangements and will make additional payments to exercise its exclusive option to license future products. Baylor is eligible to receive further payments related to late-stage clinical, regulatory approval and sales milestones, as well as single digit royalties for the successful development of specific products. As part of the up-front consideration, Baylor will receive Cell Medica Preference Shares, which are convertible into Common Shares. Specific financial terms have not been disclosed.

"We are very pleased to partner with Cell Medica in a collaboration aimed at unlocking the huge potential of cellular immunotherapy for the benefit of cancer patients," said Dr. Adam Kuspa, senior vice president and dean for research at Baylor College of Medicine. "The co-development partnership represents a novel aspect of this collaboration which will fully engage Baylor in the early stage development work, including Phase I studies".

"This collaboration with Baylor College of Medicine will place Cell Medica at the forefront of new product concepts for CAR-modified immune cells," noted Gregg Sando, CEO of Cell Medica. "Baylor’s leading research capability in this field should add significantly to our pipeline of high value products targeting cancer types that do not respond to conventional treatments."

ARIAD Initiates Submission of New Drug Application for Brigatinib to the U.S. Food and Drug Administration Ahead of Plan

On June 17, 2016 ARIAD Pharmaceuticals, Inc.(NASDAQ:ARIA) reported the initiation of a New Drug Application (NDA) submission for its investigational anaplastic lymphoma kinase (ALK) inhibitor, brigatinib, to the U.S. Food and Drug Administration (FDA) (Press release, Ariad, JUN 17, 2016, View Source [SID:1234513446]).

ARIAD is seeking U.S. marketing approval of brigatinib for patients with ALK+ non-small cell lung cancer (NSCLC) who are resistant to crizotinib. Brigatinib received Breakthrough Therapy designation from the FDA for the treatment of patients with ALK+ NSCLC whose tumors are resistant to crizotinib, and was granted orphan drug designation by the FDA for the treatment of ALK+ NSCLC. The Company is seeking accelerated approval for brigatinib from the FDA and plans to request a priority review of the application.

"We are ahead of our previously announced schedule for initiating the submission of our brigatinib NDA to the FDA and we are grateful to have breakthrough status from the FDA, which provides the opportunity to utilize the rolling submission process," stated Paris Panayiotopoulos, president and chief executive officer of ARIAD. "If approved, we believe that brigatinib will become an important new medicine for ALK+ NSCLC patients who have become resistant or intolerant to prior crizotinib therapy and will offer additional hope to these patients and their families."

ARIAD’s NDA is a rolling submission which will occur in three parts. The initial submission contains all nonclinical portions of the NDA and will be followed by submissions of the chemistry, manufacturing and controls (CMC) and clinical data. The rolling NDA submission is expected to be complete in the third quarter of 2016.

Phase 2 ALTA Results Presented at ASCO (Free ASCO Whitepaper)

Results from the ongoing Phase 2 ALTA trial of brigatinib reported at this year’s annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) earlier this month showed that, in patients on the 180 mg regimen (Arm B, n=110) with a median follow-up of 8.3 months (range 0.1—20.2), 54 percent achieved a confirmed objective response, the trial’s primary endpoint. In this arm, the median progression free survival (PFS) exceeded one year (12.9 months) in this post-crizotinib setting. Additionally, a confirmed intracranial objective response rate (ORR) of 67 percent (12/18) was achieved in patients with measurable brain metastases.

The most common treatment-emergent adverse events (TEAEs; ≥ 25% of all patients, [Arm B/A]), regardless of relationship to treatment, were nausea (40%/33%), diarrhea (38%/19%), cough (34%/18%), and headache (27%/28%). TEAEs, ≥ grade 3, occurring in ≥ 5 percent of all patients (Arm B/A), were increased blood creatine phosphokinase (9%/3%) and hypertension (6%/6%).

About Brigatinib

Brigatinib is an investigational, targeted cancer medicine discovered internally at ARIAD. It is in development for the treatment of patients with anaplastic lymphoma kinase positive (ALK+) non-small cell cancer (NSCLC) whose disease is resistant to crizotinib. Brigatinib is currently being evaluated in the global Phase 2 ALTA trial that is the basis for its initial regulatory review. ARIAD has also initiated the Phase 3 ALTA 1L trial to assess the efficacy of brigatinib in comparison to crizotinib.