Loxo Oncology Announces Initial LOXO-292 Clinical Data to be Presented as Late-Breaking Presentation at the IASLC 18th World Conference on Lung Cancer

On September 13, 2017 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported that its investigators will present initial clinical data for LOXO-292 at the International Association for the Study of Lung Cancer (IASLC) 18th World Conference on Lung Cancer to be held October 15-18, 2017, in Yokohama, Japan (Press release, Loxo Oncology, SEP 13, 2017, View Source [SID1234520505]). The oral presentation will include case reports for two patients with RET fusion lung cancer, previously treated with multikinase inhibitors (MKIs), who received LOXO-292, Loxo Oncology’s highly selective RET inhibitor.

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The schedule for the late-breaking oral presentation is as follows:

Presentation Date: October 18, 2017
Title: LOXO-292, a potent, highly selective RET inhibitor, in MKI-resistant RET fusion-positive lung cancer patients with and without brain metastases
Session Title: Emerging Genomic Targets
Presenter: Vamsidhar Velcheti, M.D.

About LOXO-292
LOXO-292 is a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase. RET fusions have been identified in approximately 2% of non-small cell lung cancer, 10-20% of papillary thyroid cancer, and a subset of colon and other cancers. RET point mutations account for approximately 60% of medullary thyroid cancer. Both RET fusion and select RET mutated cancers are primarily dependent on this single activated kinase for their proliferation and survival. This dependency, often referred to as "oncogene addiction," renders such tumors highly susceptible to small molecule inhibitors targeting RET. LOXO-292 was designed to inhibit native RET signaling as well as anticipated acquired resistance mechanisms that could otherwise limit the activity of this therapeutic approach. LOXO-292 is currently being studied in a Phase 1 trial. Interested patients and physicians can contact the Loxo Oncology Physician and Patient RET Clinical Trial Hotline at 1-855-RET-4-292 or email [email protected].

Iovance Biotherapeutics Announces Approval of First Clinical Trial Application by Competent Authority in Netherlands for a Phase 2 Trial of LN-145 for the Treatment of Patients with Cervical Cancer

On September 13, 2017 Iovance Biotherapeutics, Inc. (NASDAQ:IOVA), a biotechnology company developing novel cancer immunotherapies based on tumor-infiltrating lymphocyte (TIL) technology, reported the approval of the Company’s Clinical Trial Application (CTA), by the competent authority (CA) in the Netherlands, for a Phase 2 trial of LN-145 for the treatment of patients with recurrent, metastatic or persistent cervical carcinoma (Press release, Iovance Biotherapeutics, SEP 13, 2017, View Source [SID1234520504]). Iovance initiated the submission of CTAs in multiple countries in Europe starting in August 2017 in support of clinical trials in cervical carcinoma and metastatic melanoma.

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"We intend to use the data from our Phase 2 trials in support of global registration for TIL therapy to treat patients with melanoma and cervical carcinoma. We are very enthusiastic to have our first CTA approved by the CA and look forward to initiating our trials in Europe to offer TIL therapy to these metastatic melanoma and cervical carcinoma patients in addition to those in the US. The encouraging data generated by the National Cancer Institute showing responses in three of the nine cervical cancer patients treated with TIL therapy, with two continuing to have a complete response at 46 months and 54 months of follow up, supports the potential for LN-145 in the treatment of cervical cancer," said Dr. Maria Fardis, PhD, MBA, President and Chief Executive Officer of Iovance Biotherapeutics.

LN-145 is an adoptive cell transfer (ACT) therapy that utilizes an autologous TIL manufacturing process. C-145-04 is a Phase 2, multicenter, single-arm, open-label interventional study that will enroll up to 47 patients and will assess the safety and efficacy of LN-145 for the treatment of patients with recurrent, metastatic, or persistent cervical carcinoma. The cell transfer therapy used in this study involves patients receiving a non-myeloablative (NMA) lymphocyte depleting preparative regimen, followed by infusion of autologous TIL and the administration of a regimen of up to six doses of IL-2.

Clovis Oncology’s Rucaparib ARIEL3 Study Data Published in The Lancet

On September 13, 2017 Clovis Oncology, Inc. (NASDAQ: CLVS) reported that comprehensive data from the Phase 3 ARIEL3 study of rucaparib for maintenance treatment of advanced ovarian cancer were published online today in The Lancet (Press release, Clovis Oncology, SEP 13, 2017, View Source [SID1234520503]). The ARIEL3 study successfully achieved its primary and key secondary endpoints – improved progression-free survival (PFS) by both investigator review and blinded independent central review (BICR), respectively – in each of the three populations studied, as well as its exploratory endpoints.

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ARIEL3 is a double-blind, placebo-controlled, phase 3 trial of rucaparib that enrolled 564 women with platinum-sensitive, high-grade ovarian, fallopian tube, or primary peritoneal cancer. The primary efficacy analysis evaluated three prospectively defined molecular sub-groups in a step-down manner: 1) tumor BRCA mutant (tBRCAmut) patients, inclusive of germline and somatic mutations of BRCA (n=196); 2) HRD patients, including BRCA-mutant patients and BRCA wild-type with high loss of heterozygosity, or LOH-high patients (n=354), and, finally, 3) the intent-to-treat population, or all patients treated in ARIEL3 (n=564). The study achieved its primary endpoint of improved PFS by investigator review in each of three populations. PFS was also improved in the rucaparib group compared with placebo by BICR, a key secondary endpoint, in all three populations. In addition, rucaparib improved objective response rate vs placebo among evaluable trial participants in all three study populations.

"The publication of the ARIEL3 data in this prestigious, peer-review journal reinforces the importance of identifying new therapies that provide meaningful clinical benefit to women with advanced ovarian cancer, and speaks to the high quality of the study design and the data we were able to deliver," said Patrick J. Mahaffy, President and CEO of Clovis Oncology. "We extend our sincere thanks to the study investigators and authors, as well as the many patients, who supported and participated in ARIEL3."

"The extension in PFS in the ARIEL3 intent-to-treat population demonstrates that patients with platinum-sensitive ovarian carcinoma can derive robust clinical benefit from rucaparib maintenance treatment, regardless of their mutational status," said Robert L. Coleman, M.D., professor and vice chair, clinical research, in the Department of Gynecologic Oncology and Reproductive Medicine at The University of Texas MD Anderson Cancer Center and the U.S. principal investigator for the ARIEL3 study. "Additionally, the ARIEL3 study was intentionally designed to deliver multiple, key insights that will help inform treatment decisions and management of advanced ovarian cancer patients going forward."

According to the paper published today, treatment emergent adverse events (TEAEs) in the ARIEL3 rucaparib group were generally managed with dose modifications and not associated with increased mortality or morbidity compared with the placebo group. Safety data from ARIEL3 demonstrate consistency with prior rucaparib studies.

In December 2016, Rubraca became the first PARP inhibitor approved by the U.S. Food and Drug Administration (FDA) as monotherapy for treatment of patients with deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer who have been treated with two or more prior chemotherapies. During the fourth quarter of 2016, a Marketing Authorization Application (MAA) was submitted and accepted in Europe for Rubraca in the same ovarian cancer-treatment indication.

Based on the ARIEL3 findings, Clovis Oncology plans to submit a supplemental New Drug Application (sNDA) to the U.S. FDA for a second line or later maintenance treatment indication in ovarian cancer by the end of October 2017. In early 2018, the Company plans to file an MAA in Europe for the maintenance treatment indication upon receipt of a potential approval for the treatment indication.

About the ARIEL3 Clinical Trial

The ARIEL3 pivotal study of rucaparib is a confirmatory randomized, double-blind study comparing the effects of rucaparib against placebo to evaluate whether rucaparib given as a maintenance treatment to platinum-sensitive ovarian cancer patients can extend the period of time for which the disease is controlled after a complete or partial response to platinum-based chemotherapy. The study enrolled 564 patients with high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer. To be eligible for the study, participants had to have received at least two prior platinum-based treatment regimens, been sensitive to the penultimate platinum regimen, and achieved a complete or partial response to their most recent platinum-based regimen. There were no genomic selection criteria for this study. Trial participants were randomized 2:1 to receive 600 milligrams of rucaparib twice daily (BID) or placebo.

About Rucaparib

Rucaparib is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in ovarian cancer as well as several additional solid tumor indications. In December 2016, rucaparib became the first PARP inhibitor approved by the U.S. Food and Drug Administration (FDA) as monotherapy for treatment of patients with deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer who have been treated with two or more prior chemotherapies. Studies open for enrollment or under consideration include ovarian, prostate, breast, pancreatic, gastroesophageal, bladder, lung and urothelial cancers. Clovis is also developing rucaparib in patients with mutant BRCA tumors and other DNA repair deficiencies beyond BRCA – commonly referred to as homologous recombination deficiencies, or HRD. Clovis holds worldwide rights for rucaparib.

About Ovarian Cancer

Ovarian cancer is the sixth deadliest cancer amongst women in Europe,i where more than 65,000 women are diagnosed annually.ii Ovarian cancer is challenging to treat, and most women will relapse after surgery and chemotherapy. The 80 to 85 percent of women diagnosed in the later stages of the disease (III and IV) have particularly poor outcomes.iii Approximately one in four women with ovarian cancer have a germline or somatic BRCA mutation,iv and new treatment options are needed to treat unique patient populations.

bluebird bio Announces First Patient Treated in Expansion Cohort of CRB-401, Phase 1 Study of Anti-BCMA CAR T Therapy bb2121

On September 13, 2017 bluebird bio, Inc. (NASDAQ: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, reported that the expansion cohort of the CRB-401 Phase 1 study of bb2121, an anti-BCMA CAR T therapy, has been initiated (Press release, bluebird bio, SEP 13, 2017, View Source [SID1234520502]). The objective of the CRB-401 study is to evaluate the safety and efficacy of bb2121 in patients with relapsed/refractory multiple myeloma and determine a recommended Phase 2 dose. bluebird bio and Celgene Corp. are jointly developing bb2121.

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"The high response rate and sustained benefit seen with bb2121 in the recent data presented at the ASCO (Free ASCO Whitepaper) annual meeting in June are particularly gratifying given the limited therapeutic options available for the heavily pretreated patients with relapsed/refractory multiple myeloma participating in our study," said David Davidson, MD, chief medical officer, bluebird bio. "In the expansion stage of the CRB-401 study, we will be treating an additional cohort of patients with a dose range shown to be active in the prior dose escalation stage of the study to gain more experience with the safety, efficacy and durability of response of bb2121."

Patients in the expansion cohort will be treated at a dose range of 150 to 450 x 106 CAR+ T cells and will be required to have prior exposure to a proteasome inhibitor, an immunomodulatory agent and daratumumab.
About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D product candidate, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-thalassemia, and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bb2121 is currently being studied in a Phase 1 trial for the treatment of relapsed/refractory multiple myeloma.

bluebird bio also has discovery research programs utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline.

bluebird bio has operations in Cambridge, Massachusetts, Seattle, Washington and Europe.

AVEO Oncology Announces Receipt of Payments from EUSA Pharma and CANbridge

On September 13, 2017 AVEO Oncology (NASDAQ:AVEO) reported the receipt of a $4 million research and development payment from EUSA Pharma related to the approval of FOTIVDA (tivozanib) for the treatment of adult patients with advanced renal cell carcinoma in Europe, and a $0.5 million milestone payment from CANbridge related to manufacturing development activities for AV-203, AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate (Press release, AVEO, SEP 13, 2017, View Source;p=RssLanding&cat=news&id=2300267 [SID1234520501]).

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"These payments further strengthen our balance sheet while demonstrating the value of our partnerships in advancing AVEO’s pipeline," said Michael Bailey, president and chief executive officer of AVEO. "Additionally, these payments add to our current cash on hand, which was expected to fund operations into the fourth quarter of 2018. We look forward to several additional key milestones in the coming quarters, including the expected presentation of tivozanib-nivolumab combination Phase 1 TiNivo study results this fall, and the anticipated readout of our pivotal Phase 3 TIVO-3 trial in the first quarter of 2018."

Under the terms of their December 2015 agreement, EUSA Pharma has agreed to pay AVEO up to $390 million in future milestone payments and research and development funding, assuming successful achievement of specified development, regulatory and commercialization objectives. In addition, a tiered royalty will be due to AVEO ranging from a low double-digit up to mid-twenty percent on net sales of tivozanib in the agreement’s territories. With European approval, AVEO will be eligible for up to $12 million in milestones from EUSA based on reimbursement and regulatory approvals. In the territories licensed to EUSA, thirty percent of milestone and royalty payments received by AVEO, excluding research and development payments such as the one announced today, are due to Kyowa Hakko Kirin (KHK) as a sublicensing fee. In the territories retained by AVEO, the royalty obligation to KHK ranges from the low- to mid-teens on net sales.

In March 2016, AVEO announced an exclusive collaboration and license agreement, granting CANbridge worldwide rights to AV-203, excluding the United States, Canada, and Mexico. Under the terms of the collaboration and license agreement, AVEO is eligible to receive up to $132 million in future reimbursement and milestone payments, assuming the successful achievement of specified development, regulatory and commercialization objectives, as well as a tiered royalty, with a percentage range in the low double digits, on net sales of AV-203 in the partnered territories.

AVEO has retained North American rights to tivozanib and AV-203.