ArQule has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, ArQule, AUG 4, 2017, View Source [SID1234520138]).

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On August 3, 2017 Kite Pharma, Inc., (Nasdaq:KITE), a leading cell therapy company, reported the publication of the first large-scale multi-institutional analysis of outcomes of patients with refractory aggressive non-Hodgkin lymphoma (NHL) in the latest electronic edition of BLOOD (Press release, Kite Pharma, AUG 3, 2017, View Source [SID1234520014]). The study, SCHOLAR-1 (Retrospective Non-Hodgkin Lymphoma Research), showed outcomes in patients with refractory aggressive NHL subtypes including diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL) or primary mediastinal B-cell lymphoma (PMBCL) following treatment with currently available therapies:

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Objective response rate of 26 percent
Complete response rate of 7 percent
Median overall survival of 6.3 months
These findings highlight the unmet medical need and provide an important benchmark for studies that address this refractory patient population.

"SCHOLAR-1 demonstrates the uniformly poor treatment outcomes for patients with aggressive non-Hodgkin lymphoma and emphasizes the need for breakthrough therapies for these refractory patients," said Christian Gisselbrecht, MD, Professor of Hematology, Saint Louis Hospital at Diderot University Paris 7, and corresponding author of the study. "Although 60 to 70 percent of non-Hodgkin lymphoma patients survive five years after rituximab-based chemotherapy and autologous stem cell transplant, nearly half of them either do not respond or relapse shortly after transplant. SCHOLAR-1 provides a rigorous measure of outcomes for these patients who do not benefit from currently available therapies, and this landmark study will serve as an important historical control for evaluating new therapeutic candidates in the field of non-Hodgkin lymphoma."

Key points and findings:

SCHOLAR-1 uses pooled, patient-level data from two of the largest randomized controlled studies in NHL, the Canadian Cancer Trials Group study Ly.12 and the Lymphoma Academic Research Organization (LYSARC) Collaborative Trial in Relapsed Aggressive Lymphoma (CORAL) study, and from two observational cohorts from MD Anderson Cancer Center and the Molecular Epidemiology Resource of the University of Iowa/Mayo Clinic Lymphoma Specialized Program of Research Excellence (IA/MC).
The analysis includes 636 patients who met criteria for refractory DLBCL, TFL and PMBCL. Refractory status was defined as progressive disease or stable disease as best response to last chemotherapy, or relapse ≤12 months post-ASCT.
While patients with relapsed/refractory NHL, a more broadly defined patient population studied in Ly.12 and CORAL, have heterogeneous outcomes to subsequent therapy, the subgroup of patients with strictly refractory NHL, as studied in SCHOLAR-1, have uniformly poor outcomes and greater unmet need.
In the refractory NHL patient pool studied in SCHOLAR-1, objective response rate was 26 percent, complete response rate was 7 percent, and median overall survival was 6.3 months.
Outcomes for the refractory NHL patient pool studied in SCHOLAR-1 were consistently poor across patient subgroups.
SCHOLAR-1 was funded through an unrestricted grant from Kite.

On August 3, 2017 Aclaris Therapeutics, Inc. (NASDAQ:ACRS), a dermatologist-led biopharmaceutical company focused on identifying, developing and commercializing innovative and differentiated therapies to address significant unmet needs in medical and aesthetic dermatology, reported it has submitted a Marketing Authorization Application (MAA) with the Medicines Product Agency (MPA) in Sweden for its product candidate A-101 40% Topical Solution for the treatment of seborrheic keratosis (Press release, Aclaris Therapeutics, AUG 3, 2017, View Source [SID1234525294]). The MPA will act as the reference member state in this decentralized procedure for review of the MAA for potential marketing approval throughout Europe. If approved, A-101 40% Topical Solution would be available to be commercialized in 16 countries in the European Union.

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Seborrheic keratosis (SK) lesions are common, non-cancerous skin lesions which can have a negative physical and emotional impact on patients because they may be perceived as cosmetically unattractive and associated with aging. Existing treatments are often painful, invasive and can have undesirable outcomes such as pigmentary changes or scarring. Fewer than 10% of people [in the United States] with SK lesions currently receive treatment.

Positive results from two pivotal Phase 3 trials – SEBK-301 and SEBK-302 – were reported in late 2016 and provide the clinical basis for this MAA submission. In these trials, A-101 40% Topical Solution (A-101 40%) met all primary and secondary endpoints, achieving clinically and statistically significant clearance of SK lesions. The two trials, which were identical in design and together enrolled 937 patients, evaluated the safety and efficacy of A-101 compared with vehicle (placebo) in patients with four target SK lesions on the face, trunk, and extremities.

"This MAA submission represents a major step toward our goal of delivering a novel, topical treatment to address a significant unmet need in the dermatology market," said Dr. Neal Walker, President and Chief Executive Officer of Aclaris. "If approved, we believe A-101 will have broad appeal across aesthetic and medical dermatology patients – both men and women."

Acceleron Pharma has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Acceleron Pharma, AUG 3, 2017, View Source [SID1234520054]).

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On August 2, 2017 AstraZeneca and its haematology research and development centre of excellence, Acerta Pharma, reported that the US Food and Drug Administration (FDA) has accepted and granted priority review for the New Drug Application (NDA) for acalabrutinib, a highly-selective, potent, Bruton tyrosine kinase (BTK) inhibitor (Press release, AstraZeneca, AUG 2, 2017, View Source [SID1234519987]).

The NDA is based on results from the Phase II ACE-LY-004 clinical trial, which evaluated the safety and efficacy of acalabrutinib in patients with relapsed/refractory mantle cell lymphoma (MCL) who have received at least one prior therapy. This follows the FDA’s recent Breakthrough Therapy Designation for acalabrutinib.

Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer, said: "FDA’s acceptance of the acalabrutinib application and Priority Review illustrates the impact it could have on patients with relapsed or refractory mantle cell lymphoma as we work to bring this potential medicine to those in need as quickly as possible."

Priority Review is granted to applications for medicines that, if approved, would offer a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.[1] The Prescription Drug User Fee Act (PDUFA) date is during the first quarter of 2018.

Flavia Borellini, PhD, Acerta Pharma Chief Executive Officer, said: "We believe acalabrutinib has the potential to be a very important treatment option for patients with this life-threatening blood cancer. The FDA’s NDA acceptance exemplifies our progress in the acalabrutinib development programme and continues our momentum as we seek to transform care for people with haematologic malignancies."

Results from the ACE-LY-004 clinical trial will be submitted for presentation at a forthcoming medical meeting. The acalabrutinib development programme includes both monotherapy and combination therapy strategies in a broad range of blood cancers and solid tumours. The programme includes the Phase III ACE-LY-308 clinical trial evaluating acalabrutinib as a 1st-line treatment for patients with MCL.[2]

About mantle cell lymphoma (MCL)
Mantle cell lymphoma (MCL) is an aggressive B-cell non-Hodgkin lymphoma (NHL) with poor prognosis.[3],[4],[5],[6] MCL accounts for approximately 3% to 6% of new NHL cases in Western countries each year, with an annual incidence of 0.5 per 100,000 persons and an estimated prevalence of 3.5/100,000.5,[7] The median age at diagnosis is 68 years, with a 3:1 male predominance.5

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About acalabrutinib
Acalabrutinib is a highly-selective, potent, covalent inhibitor of Bruton tyrosine kinase (BTK) with minimal off-target activity observed in pre-clinical trials.[8],[9],[10] This potential new medicine is in development for the treatment of multiple B-cell and other cancers. The acalabrutinib development programme includes both monotherapy and combination therapy strategies in chronic lymphocytic leukaemia (CLL), MCL, Waldenström macroglobulinemia (WM), follicular lymphoma, diffuse large B-cell lymphoma, and multiple myeloma, as well as monotherapy and combination trials in solid tumours. In total, more than 25 acalabrutinib clinical trials with more than 2,000 patients are underway or have completed. Acalabrutinib was granted Orphan Drug Designation by the FDA for the treatment of patients with MCL in September 2015 and by the European Commission in March 2016 for the treatment of patients with CLL, MCL and WM. Acalabrutinib was granted Breakthrough Therapy Designation by the FDA in August 2017 for the treatment of patients with MCL who have received at least one prior therapy. Acalabrutinib is a potential new medicine not approved for any current use.