On November 28, 2018 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported that the U.S. Patent and Trademark Office (USPTO) has issued a new patent protecting the Company’s lead drug candidate, tipifarnib, a potent and selective farnesyl transferase that is currently being studied in multiple solid tumor and hematologic indications, including a registration-directed trial in HRAS mutant head and neck squamous cell carcinoma (HNSCC) and a Phase 2 trial in peripheral T-cell lymphoma (PTCL) (Press release, Kura Oncology, NOV 28, 2018, View Source [SID1234531664]).

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U.S. Patent No. 10,137,121, "Methods of Treating Cancer with Farnesyltransferase Inhibitors," includes multiple claims directed to the use of tipifarnib as a method of treating patients with angioimmunoblastic T-cell lymphoma (AITL), an aggressive form of T-cell lymphoma. The newly issued patent has an expiration date of November 2037, excluding any possible patent term extension. Kura continues to pursue U.S. and foreign patent protection in this and other indications.

"The issuance of this new patent is an important achievement for Kura and reflects our ability to expand the breadth and depth of tipifarnib’s development opportunities," said Troy Wilson, Ph.D., President and CEO of Kura Oncology. "This patent comes just six months after the USPTO issued us a patent for the use of tipifarnib as method of treating patients with certain CXCL12-expressing cancers, further strengthening our intellectual property protection for tipifarnib based on genetically defined patient populations and disease indications."

Kura is evaluating, on a prospective basis, the role of the CXCL12 pathway and markers of bone marrow homing as potential biomarkers of clinical activity for tipifarnib in hematologic malignancies. The Company’s ongoing Phase 2 trial of tipifarnib in PTCL is enrolling patients into two expansion cohorts. The first cohort is defined by histology and includes patients with AITL. The second cohort is defined by genetics and includes patients with PTCL not otherwise specified (NOS) who have the absence of a single nucleotide variation in the 3’ untranslated region of the CXCL12 gene. The Company estimates that the combined addressable populations of patients with AITL and CXCL12+ account for approximately 40% of all PTCL cases.

Kura plans to report preliminary data from both expansion cohorts in its Phase 2 trial of tipifarnib at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego on Sunday, December 2, 2018. A copy of the poster will be available on the Company’s website at www.kuraoncology.com following presentation at the meeting.

About Tipifarnib

Kura Oncology’s lead candidate, tipifarnib, is an inhibitor of farnesylation, a key cell signaling process implicated in cancer initiation and development. Tipifarnib was previously studied in more than 5,000 cancer patients and showed compelling and durable anti-cancer activity in certain patient subsets with a manageable side effect profile. Leveraging advances in next-generation sequencing as well as emerging information about cancer genetics and tumor biology, the Company is seeking to identify those patients most likely to benefit from tipifarnib. Based on positive results from a Phase 2 clinical trial in HRAS mutant HNSCC and feedback from the U.S. Food and Drug Administration, Kura recently initiated a global, registration-directed trial of tipifarnib in patients with recurrent or metastatic HRAS mutant HNSCC.

On November 28, 2018 Aclaris Therapeutics, Inc. (NASDAQ:ACRS), a dermatologist-led biopharmaceutical company committed to identifying, developing, and commercializing innovative therapies to address significant unmet needs in aesthetic and medical dermatology and immunology, reported that management will attend the following conferences (Press release, Aclaris Therapeutics, NOV 28, 2018, View Source [SID1234531663]):

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Management will host investor meetings at the 2018 Citi Global Healthcare in New York, NY on Wednesday, December 5, 2018.
Dr. Neal Walker, President and Chief Executive Officer, will present at the 12th Annual Leerink POLARxPRESS Conference in New York, NY on Tuesday, December 11, 2018.

On November 28, 2018 the U.S. Food and Drug Administration approved Truxima (rituximab-abbs) as the first biosimilar to Rituxan (rituximab) for the treatment of adult patients with CD20-positive, B-cell non-Hodgkin’s lymphoma (NHL) to be used as a single agent or in combination with chemotherapy. Truxima is the first biosimiliar to be approved in the U.S. for the treatment of non-Hodgkin’s lymphoma.

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"As part of the FDA’s Biosimilars Action Plan we’re advancing new policies to make the development of biosimilars more efficient and to enable more opportunities for biosimilar manufacturers to make these products commercially successful and competitive. Our goal is to promote competition that can expand patient access to important medicines," said FDA Commissioner Scott Gottlieb, M.D. "The Truxima approval is our third biosimiliar approval in the past month. The growing pipeline of biosimilars is encouraging. We’re seeing more biosimilar drugs gain market share as this industry matures. We’ll continue to make sure biosimilar medications are evaluated efficiently through a process that makes certain that these new medicines meet the FDA’s rigorous standards for approval."

Truxima is indicated for the treatment of adult patients with:

Relapsed or refractory, low grade or follicular, CD20-positive B-cell NHL as a single agent;
Previously untreated follicular, CD20-positive, B-cell NHL in combination with first line chemotherapy and, in patients achieving a complete or partial response to a rituximab product in combination with chemotherapy, as single-agent maintenance therapy; and
Non-progressing (including stable disease), low-grade, CD20­ positive, B-cell NHL as a single agent after first-line cyclophosphamide, vincristine and prednisone (CVP) chemotherapy.

Biological products are generally large, complex molecules and may be produced through biotechnology in a living system, such as a microorganism, plant cell or animal cell. A biosimilar is a biological product that is approved based on data showing that it is highly similar to a biological product already approved by the FDA (reference product) and has no clinically meaningful differences in terms of safety, purity and potency (i.e., safety and effectiveness) from the reference product, in addition to meeting other criteria specified by law.

The FDA’s approval of Truxima is based on a review of evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic data, clinical immunogenicity data, and other clinical data that demonstrates Truxima is biosimilar to Rituxan. Truxima has been approved as a biosimilar, not as an interchangeable product.

The most common side effects of Truxima are infusion reactions, fever, abnormally low level of lymphocytes in the blood (lymphopenia), chills, infection and weakness (asthenia). Health care providers are advised to monitor patients for tumor lysis syndrome (a complication of treatment where tumor cells are killed off at the same time and released into the bloodstream), cardiac adverse reactions, damage to kidneys (renal toxicity), and bowel obstruction and perforation. Patients should not receive vaccinations while in treatment. Women who are pregnant or breastfeeding should not take Truxima because it may cause harm to a developing fetus or newborn baby.

Like Rituxan, the labeling for Truxima contains a Boxed Warning to alert health care professionals and patients about increased risks of the following: fatal infusion reactions, severe skin and mouth reactions, some with fatal outcomes; Hepatitis B virus reactivation, that may cause serious liver problems including liver failure and death; and Progressive Multifocal Leukoencephalopathy, a rare, serious brain infection that can result in severe disability or death. This product must be dispensed with a patient Medication Guide that provides important information about the drug’s uses and risks.

The FDA granted approval of Truxima to Celltrion. Rituxan was approved in November 1997 and is manufactured by Genentech.

With the Truxima approval, the FDA has approved 15 biosimiliars. On Oct. 30, 2018, the FDA approved Hyrimoz (adalimumab-adaz), from Sandoz, as a biosimilar to Humira (adalimumab) and on Nov. 2, 2018, the FDA approved Udenyca (pegfilgrastim-cbqv) from Coherus, as a biosimilar to Neulasta (pegfilgrastim).

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

On November 28, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a company focused on the development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell ("CAR T") technology and gene therapies for rare diseases, reported that additional safety and efficacy Phase 1 data evaluating MB-102 (CD123 CAR) in relapsed or refractory acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN) have been selected for an oral presentation at the AACR (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy (Press release, Mustang Bio, NOV 28, 2018, View Source [SID1234531653]). The presentation will take place on November 30, 2018, in Miami Beach, Florida.

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Martina Sersch, M.D., Ph.D., Chief Medical Officer of Mustang, said, "We are excited about the additional data demonstrating MB-102’s (CD123 CAR T) potential to treat patients with AML and BPDCN. Initial safety and tolerability data as well as preliminary data on efficacy are very promising in a population with high unmet medical need. Based on the Phase 1 data, we expect to submit an IND filing for MB-102 and look forward to initiating a multicenter Phase 1/2 clinical trial in 2019 in patients with AML, BPDCN and high-risk myelodysplastic syndrome."

Lihua Elizabeth Budde, M.D., Ph.D., assistant professor in the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope and principal investigator for the Phase 1 trial, said, "There is increased expression of CD123 on AML blasts, leukemic stem cells and BPDCN cells compared to normal hematopoietic stem cells, and it is therefore a promising target for cellular immunotherapy. We remain encouraged by interim data showing MB-102’s potential to treat BPDCN and AML and continue to evaluate MB-102’s clinical benefits in our ongoing Phase 1 clinical trial."

Details of the oral presentation are as follows:

Title: CD123CAR Displays Clinical Activity in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Safety and Efficacy Results from a Phase 1 Study
Special Session: Novel Targets, Pathways and Tools
Date and Time: Friday, November 30, 2018; 8:00 AM – 9:30 AM ET
Location: Loews Miami Beach Hotel
Presenter: Elizabeth Lihua Budde, M.D., Ph.D., City of Hope, Duarte, CA

For more information, please visit: View Source;ItemID=733&DetailItemID=733.

Key Efficacy and Safety Findings
This single center, first-in-human Phase 1 dose-escalation clinical trial is evaluating the safety and activity of escalating doses of MB-102 in patients with relapsed or refractory AML (cohort 1) and BPDCN (cohort 2). Patients receive a single dose of MB-102 with an option for a second infusion if they continue to meet safety and eligibility criteria and still have CD123+ disease. To date, 18 patients have been enrolled and nine have been treated (seven with AML, two with BPDCN).

In the AML cohort, all seven patients had at least one prior allogeneic stem cell transplant and median of 4 (range: 4-10) prior lines of therapy. Two patients were treated at dose level 1 (50M CAR+ T). Trial investigators reported that one achieved a morphologic leukemic-free state (MLFS) that lasted 70 days. This patient received a second infusion three months later, with blast reduction from 77.9% to 0.9% (flow cytometry) after 35 days. Five patients received dose level 2 (200M CAR+ T). One patient achieved complete remission (CR) with incomplete count recovery at day 28, and one patient achieved MLFS that improved to CR at day 84. The remaining three patients had stable disease.

In the BPDCN cohort, two patients were treated with 100M CAR+ T and tolerated the treatment well, with no grade 3 or above treatment-related toxicities. One patient achieved CR with no evidence of disease in the bone marrow and skin at day 28.

Investigators found MB-102 infusions of up to 200M CAR T cells were safe. All toxicities observed to date were reversible and manageable. No patient developed grade 3 or above cytokine release syndrome or neurotoxicity. There were no treatment-related dose-limiting toxicities and no treatment-related cytopenias longer than 12 weeks. One patient with prior cutaneous graft-versus-host disease developed grade 1 rash five weeks after CAR T infusion, which resolved after clinical management. Peak of T cell expansion occurred within the first 14 days. Investigators did not observe any CD123-loss leukemic variants.

About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the myeloid line of blood cells characterized by rapid growth of abnormal white blood cells that accumulate in the bone marrow. Although AML is rare, there are approximately 20,000 new cases in the U.S. each year and 10,000 deaths. Current treatment of relapsed or refractory AML with chemotherapy or hematopoietic stem cell transplantation is associated with low rates of complete response and considerable complications.

CD123 is overexpressed on AML blasts and leukemic stem cell-enriched cell subpopulations compared to normal hematopoietic stem cells and myeloid progenitors, making CD123 an attractive target for T cell-based adoptive immunotherapy.

About Blastic Plasmacytoid Dendritic Cell Neoplasm
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and incurable blood cancer with a median survival of less than 18 months and no standard of care. High levels of CD123 expression is one of the diagnostic hallmarks of BPDCN, making CD123 an attractive target for T cell-based adoptive immunotherapy.

About MB-102 (CD123 CAR T)
MB-102 (CD123 CAR T) is a CAR T cell therapy that is produced by engineering patient T cells to recognize and eliminate CD123-expressing tumors. CD123 is widely expressed on bone marrow cells of patients with MDS, as well as in hematologic malignancies including AML, B cell acute lymphoblastic leukemia, hairy cell leukemia, BPDCN, chronic myeloid leukemia and Hodgkin’s lymphoma.

In the first-in-human clinical trial at City of Hope (NCT02159495), MB-102 has demonstrated complete responses at low doses in AML and BPDCN without dose-limiting toxicities, as reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2017. Dose escalation continues at City of Hope in both indications.

On November 27, 2018 ADC Therapeutics, an oncology drug discovery and development company that specializes in the development of proprietary antibody drug conjugates (ADCs), reported that the first patients have been dosed in a Phase I/II clinical trial evaluating the safety, tolerability, pharmacokinetics and anti-tumor activity of ADCT-602 in patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) (Press release, ADC Therapeutics, NOV 27, 2018, View Source [SID1234596072]). The trial is being led by The University of Texas MD Anderson Cancer Center.

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ADCT-602 is an ADC that incorporates a pyrrolobenzodiazepine (PBD) drug linker and targets CD22, which is a clinically validated ADC target. Preclinical studies have demonstrated that ADCT-602 has significant anti-tumor activity in a number of animal models.

Hagop Kantarjian, MD, Professor and Chair of the Department of Leukemia, and Nitin Jain, MD, Associate Professor in the Department of Leukemia, at MD Anderson, are leading the Phase I/II clinical trial of ADCT-602. The open-label trial will enroll up to 65 patients.

Dr. Kantarjian said, "There is a significant unmet need for new treatment options for adult patients with B-cell ALL who have not responded to initial treatment or whose cancer has returned after treatment. We are excited to evaluate the safety and anti-tumor activity of a CD22-targeted ADC in these patients."

Jay Feingold, MD, PhD, Chief Medical Officer and Senior Vice President at ADC Therapeutics, said, "We are delighted to be partnered with MD Anderson on this important clinical trial in adult patients with relapsed or refractory B-cell ALL, who have limited therapeutic options and for whom the prognosis is typically poor. We are hopeful that the response rates seen in our ADCT-402 and ADCT-301 lymphoma clinical trials can be replicated in the ALL patient population with ADCT-602, and that our growing portfolio of hematology-focused ADCs targeting CD19, CD25 and now CD22 can make a positive impact on patient outcomes."

For more information about this clinical trial, please visit www.clinicaltrials.gov (identifier NCT03698552).

About ADCT-602

ADCT-602 is an antibody drug conjugate (ADC) composed of a monoclonal antibody that binds to CD22 conjugated to a pyrrolobenzodiazepine (PBD) dimer toxin. Once bound to a CD22-expresing cell, ADCT-602 is internalized into the cell where enzymes release the PBD-based warhead. CD22 is an attractive and clinically validated ADC target. CD22 is highly expressed on most malignant B-cells, including expression in greater than 90% of patients with B-cell acute lymphoblastic leukemia.