On August 1, 2018 RhoVac AB ("RhoVac") reported positive top-line results on safety and on immune activation in their phase I/II clinical trial RhoVac-001 in prostate cancer patients (Press release, RhoVac, AUG 1, 2018, View Source [SID1234528369]).

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In totally 22 prostate cancer patients received RV001 treatment over a period of approximately 30 weeks. The regimen was well tolerated and there were no treatment related grade 3, 4 or 5 reactions according to CTCAE (Common Terminology Criteria for Adverse Events). The primary end-point of the study was therefore achieved. No related allergic reactions to treatment and no treatment related SAEs (Serious Adverse Events) were reported. Local injection site reactions were noted; however, these reactions were all expected and the study confirmed that the reactions are reversible. In conclusion, the study meets its endpoint.

Totally 21 of the 22 enrolled patients were eligible for immune response assessment based on IFNγ ELIspot analysis, while analytical results from one patient were non-conclusive and therefore excluded from the final assessment. The immune response was analysed before -, two time during – and once, one month after completion of treatment. The result is that 86% (18 of 21 of the eligible patients) show a significant immune response to RV001 in samples taken during or after treatment. To qualify as a Confirmed Immune Responder in RhoVac-001 protocol, patients are required to show a significant response in two of the three samples taken during or after treatment. This was the case for all 18 patients showing significant immune response. In conclusion, the results confirm that a vaccine mediated immune response is established following treatment with RV001 and the dose administered in the study is biologically active.

Comments from RhoVac´s CEO, Anders Ljungqvist
-We are very excited about the results! In addition to the very high responder rate to the vaccination, we also saw that the responses were consistent over time, and we found that the vaccinations were safe and well tolerated by the patients. At this time, I would like to thank the Copenhagen Prostate Cancer Center at the University Hospital, Copenhagen, the phase-I unit Zelo at Bispebjerg and Frederiksberg Hospital plus DanTrials ApS and the T-cell monitoring group, Department of Immunology at the University of Tübingen, for a highly professional, timely and dedicated work. It has been an outstanding collaboration. Finally, but not least, I would like to thank the enrolled patients for participating in this clinical trial. Your commitment and compliance to treatment and planned visits have ensured that the trial have been completed on time and that data can be clearly interpreted.

The clinical trial
The clinical trial RhoVac-001 (ClinicalTrials.gov identifier: NCT03199872) is a phase I/II first-in-man trial studying the therapeutic cancer vaccine RV001. Patients prostatectomised, due to histologically verified adenocarcinoma of the prostate gland who were not being treated, were enrolled in the study and recruitment of the 22 patients was completed in July 2017. The primary endpoint of the study is to evaluate the safety and tolerability of the RV001 cancer vaccine and the secondary endpoint is to investigate the RV001-specific immunological response before, during and after vaccination. In addition to the now reported results, all patients treated are monitored for duration of immune response over a 12-month period following completion of treatment. This part of the study is ongoing and the results are expected to be reported mid-2019.

RV001 cancer vaccine
RV001 is a peptide based therapeutic cancer vaccine targeting the protein RhoC, which is known to be overexpressed in practically all cancer cells having a metastatic potential. The basic concept with RV001 cancer vaccine is to develop a treatment against initial formation and spread of metastatic cancer cells, specifically targeting the prevention against cancer relapse.

Prostate cancer
Prostate cancer is the fourth most common cancer in both sexes combined and the second most common cancer in men. An estimated 1.1 million men worldwide were diagnosed with prostate cancer in 2012, accounting for 15% of the cancers diagnosed in men, with almost 70% of the cases (759,000) occurring in more developed regions. The RV001 cancer vaccine is focussing on patients having completed treatment of the primary tumor, which commonly is surgical removal of all or part of the prostate gland. The adjuvant treatment is aiming at blocking or limiting relapse of prostate cancer.

On August 1, 2018 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to quizartinib, an investigational FLT3 inhibitor, for the treatment of adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML) (Press release, Daiichi Sankyo, AUG 1, 2018, View Source [SID1234528390]).

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"There have been limited advances over the past several decades for the treatment of relapsed/refractory FLT3-ITD AML, a very aggressive form of the disease associated with poor prognosis. Quizartinib is the first FLT3 inhibitor to significantly improve overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory AML with FLT3-ITD, an underlying driver of this subtype of AML," said Arnaud Lesegretain, Vice President, Oncology Research and Development and Head, AML Franchise, Daiichi Sankyo. "We are excited that quizartinib has received Breakthrough Therapy designation and we look forward to working closely with the FDA to bring this potential new treatment option to patients as quickly as possible."

Breakthrough Therapy designation is designed to expedite the development and regulatory review of medicines that may demonstrate substantial benefit over currently approved treatments, in order to more quickly bring new treatment options to patients with serious diseases. Significant unmet medical need exists in relapsed/refractory AML, as available treatment options are limited and there are no approved targeted therapies for patients with relapsed/refractory FLT3-ITD AML.

The designation was granted based on the results of the pivotal phase 3 QuANTUM-R study of quizartinib, which were presented during the plenary program at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June 2018. QuANTUM-R is the first randomized phase 3 study to show that a FLT3 inhibitor, quizartinib, prolongs overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory FLT3-ITD AML.

The safety profile observed in QuANTUM-R appears consistent with that observed at similar doses in the quizartinib clinical development program. Incidence of treatment-emergent adverse events was comparable between patients who received single agent quizartinib (n=241) and those who received salvage chemotherapy (n=94). The most common adverse events (>30 percent, any Grade) in patients treated with quizartinib included nausea, thrombocytopenia, fatigue, musculoskeletal pain, pyrexia, anemia, neutropenia, febrile neutropenia, vomiting and hypokalemia.

About Quizartinib
Quizartinib, the lead investigational agent in the AML Franchise of the Daiichi Sankyo Cancer Enterprise, is an oral selective FLT3 inhibitor currently in phase 3 development for relapsed/refractory (QuANTUM-R) and newly-diagnosed (QuANTUM-First) FLT3-ITD AML in the U.S., EU and Japan, and phase 2 development for relapsed/refractory FLT3-ITD AML in Japan.

In addition to Breakthrough Therapy designation, quizartinib has been granted Fast Track designation by the FDA for the treatment of relapsed/refractory AML. Quizartinib also has been granted Orphan Drug designation by both the FDA and the European Medicines Agency (EMA) for the treatment of AML. Quizartinib is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.

About FLT3-ITD Acute Myeloid Leukemia
AML is an aggressive blood and bone marrow cancer that causes uncontrolled growth and accumulation of malignant white blood cells that fail to function normally and interfere with the production of normal blood cells.1 In the U.S. this year, it is estimated that there will be more than 19,000 new diagnoses of AML and more than 10,000 deaths from AML.2 The five-year survival rate of AML reported from 2005 to 2011 was approximately 26 percent, which was the lowest of all leukemias.1

FLT3 gene mutations are one of the most common genetic abnormalities in AML.3 FLT3-ITD is the most common FLT3 mutation, affecting approximately one in four patients with AML.4,5,6,7 Patients with FLT3-ITD AML have a worse overall prognosis, including an increased incidence of relapse, an increased risk of death following relapse and a higher likelihood of relapse following hematopoietic stem cell transplantation as compared to those without this mutation.8,9

On August 1, 2018 Selecta Biosciences, Inc. (Nasdaq: SELB), a clinical-stage biopharmaceutical company focused on unlocking the full potential of biologic therapies by mitigating unwanted immune responses, reported that it plans to issue its second quarter 2018 financial results before the open of the U.S. financial markets on Wednesday, August 08, 2018 (Press release, Selecta Biosciences, AUG 1, 2018, View Source [SID1234528289]).

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At 8.30 a.m. ET that day, Selecta Biosciences will host a conference call via live webcast to discuss these results. Investors and the public can access a live and archived webcast of this call via the Investors & Media section of the company’s website, View Source Individuals may also participate in the live call via telephone by dialing (844)-845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10122287.

On August 1, 2018 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage biopharmaceutical company focused on creating innovative cancer immunotherapies, reported that Terry Rosen, Ph.D., Chief Executive Officer, will participate in a fireside chat at the 2018 Wedbush PacGrow Healthcare Conferenceon Tuesday, August 14, 2018 at 2:30 pm ET at the Parker New York, in New York City, NY. (Press release, Arcus Biosciences, AUG 1, 2018, View Source [SID1234528352]).

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On August 1, 2018 Atara Biotherapeutics, Inc. (Nasdaq:ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases reported financial results for the second quarter of 2018 and recent operational highlights (Press release, Atara Biotherapeutics, AUG 1, 2018, View Source [SID1234528632]).

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"The future of T-cell immunotherapy is both off-the-shelf and across multiple therapeutic areas," said Isaac Ciechanover, M.D., Chief Executive Officer and President of Atara Biotherapeutics. "During the second quarter, we continued to advance our robust T-cell immunotherapy pipeline, highlighted by our ongoing Phase 3 studies of tab-cel in patients with EBV+ PTLD and Phase 1 study of ATA188 in patients with progressive multiple sclerosis. In parallel, we continue to build Atara’s global commercial and operational capabilities in anticipation of the first tab-cel Phase 3 results and submission of an EU conditional marketing authorization application in the first half of 2019. We are also preparing to expand our pipeline with the development of the next generation of chimeric antigen receptor T cell (CAR T) technologies. This is an exciting time for Atara as we enter the next phase of the Company’s growth as a leader in off-the-shelf, allogeneic T-cell immunotherapy."

Recent Highlights and Anticipated Upcoming Milestones

Tab-cel (tabelecleucel)

Two Phase 3 clinical studies are ongoing (MATCH and ALLELE) to evaluate tab-cel (tabelecleucel) in patients with Epstein-Barr virus associated post-transplant lymphoproliferative disorder (EBV+ PTLD) who have failed rituximab following hematopoietic cell transplant (HCT) or solid organ transplant (SOT).
11 clinical sites for the MATCH and 13 for the ALLELE studies are now open for enrollment in the U.S. with additional sites expected to open in the U.S. and other geographies.

Presented positive long-term outcomes including durable remissions and encouraging safety findings from two Phase 2 studies of tab-cel in EBV+ PTLD at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).
One- and three-year overall survival (OS) for tab-cel treated patients with EBV+ PTLD following HCT who failed rituximab (n=35) was 68% and 55%, respectively. Median OS was not reached after a median of 23.3 months of follow-up in this patient group.
In patients with EBV+ PTLD following SOT who failed rituximab, the one- and three-year OS after treatment with tab-cel (n=14) was 64% and 43%, respectively. Median survival in this patient group was 21.3 months.
None of the EBV+ PTLD patients who had complete or partial responses (CR or PR) after treatment with tab-cel died of EBV+ PTLD. Two-year OS for these responding patients was 83% and 86% following HCT (n=24) and SOT (n=7), respectively.
Tab-cel was associated with durable objective response rate (CR plus PR) of 69% and 50% in patients with EBV+ PTLD following HCT and SOT, respectively, who have failed rituximab.

U.S. Food and Drug Administration (FDA) accepted IND to initiate a Phase 1/2 clinical study of tab-cel in combination with Merck’s anti-PD-1 (programmed death receptor-1) therapy, KEYTRUDA (pembrolizumab), in patients with platinum-resistant or recurrent EBV-associated nasopharyngeal carcinoma (NPC) that Atara plans to initiate in the second half of 2018.

Expect to present updated tab-cel results in patients with EBV+ cancers in the second half of 2018.
ATA188 & ATA190 for Multiple Sclerosis (MS)

Announced publication of new research findings advancing the understanding of Epstein-Barr Virus (EBV) infection in the MS-affected brain.
The findings were reported in an article online and published in the July 2018 print issue of Neurology: Neuroimmunology & Neuroinflammation, an official journal of the American Academy of Neurology.

A Phase 1 clinical study to evaluate off-the-shelf, allogeneic ATA188 in patients with progressive MS is also underway across clinical sites in the U.S. and Australia.
The primary objective of the Phase 1 study is to assess the safety of ATA188 in patients followed for at least one year after the first dose. Key secondary endpoints in the study include measures of clinical improvement such as expanded disability status scale (EDSS) and annualized relapse rate (ARR), as well as MRI imaging.
The first results from the ongoing ATA188 Phase 1 study in patients with progressive MS are expected in the first half of 2019.
Plan to initiate a randomized autologous ATA190 study in progressive MS patients in 2019.
Development Pipeline

Plan to rapidly advance novel gene-edited CAR T development programs from recently expanded T-cell immunotherapy collaboration with Memorial Sloan Kettering Cancer Center (MSK), leveraging our existing off-the-shelf T-cell immunotherapy technology platform, manufacturing expertise and research and development capabilities.
Expect to start Phase 1 study for ATA621 targeting both JC and BK viruses in 2019.
Corporate

Commenced operations at Atara T Cell Operations & Manufacturing (ATOM) facility in the second quarter of 2018, with completion to support clinical production expected in 2019.
Appointed Utpal Koppikar as Chief Financial Officer. Utpal has an accomplished track record in global biotechnology financial operations.
In June 2018 we exercised our option under a license agreement with QIMR Berghofer to an exclusive, worldwide license to develop and commercialize additional T-cell immunotherapy programs including ATA190, as well as the option to license additional technology.
Second Quarter 2018 Financial Results

Cash, cash equivalents and short-term investments as of June 30, 2018 totaled $417.0 million, which we believe will enable us to expand our near-term pipeline and accelerate pre-commercial activities as well as fund our previously planned operations to mid-2020. In the second quarter of 2018, we sold approximately 1.0 million shares of common stock pursuant to our "at-the-market" (ATM) facility for net proceeds of $47.6 million, after deducting commissions and other offering expenses.
We reported net losses of $50.9 million, or $1.15 per share, for the second quarter of 2018, as compared to $27.4 million, or $0.94 per share, for the same period in 2017.
Research and development expenses were $33.4 million for the second quarter of 2018, as compared to $18.3 million for the same period in 2017. The increase in the second quarter of 2018 was due to costs associated with our continuing expansion of research and development activities, including:
clinical trial, manufacturing and outside service costs related to the two Phase 3 clinical trials of tab-cel in patients with EBV+ PTLD and the Phase 1 clinical trial of allogeneic ATA188 in patients with MS;
higher payroll and related costs from increased headcount, and
an increase in allocated facilities and information technology expenses.
Research and development expenses include $3.4 million and $2.0 million of non-cash stock-based compensation expenses in the second quarters of 2018 and 2017, respectively.
General and administrative expenses were $19.2 million for the second quarter of 2018, as compared to $9.6 million for the same period in 2017. The increase in the second quarter of 2018 was primarily due to increases in professional services costs and payroll and related costs driven by increased headcount to support the Company’s expanding operations. General and administrative expenses include $4.6 million and $3.7 million of non-cash stock-based compensation expenses in the second quarters of 2018 and 2017, respectively.