10-Q – Quarterly report [Sections 13 or 15(d)]

XBiotech has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, XBiotech, 2017, NOV 14, 2016, View Source [SID1234521567]).

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!

bluebird bio to Present New Data from Novel Anti-BCMA CAR T Cell Therapy bb2121 at EORTC-NCI-AACR Molecular Targets and Cancer Therapies Symposium

On November 14, 2016 bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported that interim data from its study of bb2121, the company’s anti-BCMA CAR T cell therapy, currently in a Phase 1 trial of patients with relapsed/refractory multiple myeloma will be presented at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Molecular Targets and Cancer Therapies Symposium in Munich, Germany (Press release, bluebird bio, NOV 14, 2016, View Source;p=RssLanding&cat=news&id=2222159 [SID1234516581]).

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!

Clinical remissions and limited toxicity in a first-in-human multicenter study of bb2121, a novel anti-BCMA CAR T cell therapy for relapsed/refractory multiple myeloma

Date: Thursday, December 1, 2016, 17:40 CET (11:40 am ET)
Session: Plenary Session 7

RedHill Biopharma Reports 2016 Third Quarter Financial Results

On November 14, 2016 RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) ("RedHill" or the "Company"), a biopharmaceutical company primarily focused on development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, reported its financial results for the quarter ended September 30, 2016 (Press release, RedHill Biopharma, NOV 14, 2016, View Source [SID1234516721]).

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!

The Company will host a conference call on Monday, November 14, 2016, at 9:00 am EST to review the financial results and business highlights, dial-in details are included below.

Financial highlights for the quarter ended September 30, 20161

Research and Development Expenses for the third quarter of 2016 were $7.0 million, an increase of $3.1 million, compared to $3.9 million in the third quarter of 2015 and an increase of $1.0 million, compared to $6.0 million in the second quarter of 2016. Research and Development Expenses for the nine months ended September 30, 2016 were $17.7 million, an increase of $4.9 million, compared to $12.8 million in the comparable period of 2015. The increase in 2016 resulted primarily from the ongoing Phase III MAP US study with RHB-104 (Crohn’s disease), the ongoing Phase II and Phase III studies with BEKINDA (IBS-D and gastroenteritis, respectively) and from preparations for several Phase I/II studies with YELIVA(TM) for multiple indications.

General, Administrative and Business Development Expenses in the third quarter of 2016 were $1.4 million, an increase of $0.7 million, compared to $0.7 million in the third quarter of 2015 and an increase of $0.2 million, compared to $1.2 million in the second quarter of 2016. General, Administrative and Business Development Expenses for the nine months ended September 30, 2016 were $3.8 million, an increase of $1.4 million, compared to $2.4 million in the comparable period of 2015. The increase was mainly due to enhanced business development and investor relations activities.

Operating Loss in the third quarter of 2016 was $8.5 million, an increase of $3.9 million, compared to $4.6 million in the third quarter of 2015 and an increase of $1.3 million, compared to $7.2 million in the second quarter of 2016. Operating Loss for the nine months ended September 30, 2016 were $21.6 million, an increase of $6.4 million, compared to $15.2 million in the comparable period of 2015. The increase was mainly due to increases in Research and Development Expenses, as detailed above.

Net Cash Used in Operating Activities in the third quarter of 2016 was $7.4 million, an increase of $3.7 million, compared to $3.7 million in the third quarter of 2015 and an increase of $1.7 million, compared to $5.7 million in the second quarter of 2016. Net Cash Used in Operating Activities for the nine months ended September 30, 2016 was $18.1 million, an increase of $6.3 million, compared to $11.8 million in the comparable period of 2015. The increase mainly reflects the increase in Operating Loss, as detailed above.

Net Cash Used in Investment Activities in the third quarter of 2016 was $10.7 million, compared to Net Cash Used in Investment Activities of $2.4 million in the third quarter of 2015. Net Cash Used in Investment Activities for the nine months ended September 30, 2016 was $3.2 million, an increase of $4.3 million, compared to Net Cash Provided by Investment Activities of $1.1 million in the comparable period of 2015. The increase in Net Cash Used in Investment Activities was due to an increase in bank deposits and marketable securities in 2016.

Cash Balance2 as of September 30, 2016 was $40.5 million, a decrease of $17.6 million, compared to $58.1 million as of December 31, 2015. The decrease was a result of the ongoing operations, mainly related to research and development activities.

"We are very pleased with the important operational milestones achieved during this quarter, including the significant enhancements to the RHB-104 Phase III development program for Crohn’s disease and the initiation of several clinical studies with our novel Phase II oncology drug YELIVA(TM)" said Mr. Micha Ben Chorin, RedHill’s CFO. "We maintained a debt-free balance sheet and a cash position of $40.5 million at the end of the third quarter of 2016 as we continue to advance several Phase III and Phase II gastrointestinal programs toward important data points in the coming months. We also continue to advance our strategic plan to build a U.S. specialty GI pharmaceutical company."

Conference Call and Webcast Information:

The Company will host a conference call on Monday, November 14, 2016, at 9:00 am EST to review the financial results and business highlights.

To participate in the conference call, please dial the following numbers 5-10 minutes prior to the start of the call: United States: +1-877-280-2342; International: +1-646-254-3367; and Israel: +972-3-721-9510. The access code for the call is 2341628.

The conference call will be broadcasted live and available for replay on the Company’s website, View Source, for 30 days. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

Recent operational highlights:

On July 5, 2016, RedHill and its co-development partner, IntelGenx Corp. (IntelGenx), announced the signing of an exclusive license agreement with Grupo JUSTE S.A.Q.F (Grupo JUSTE), for the commercialization of RIZAPORT oral thin-film for acute migraines. Under the terms of the agreement, RedHill granted Grupo JUSTE the exclusive rights to register and commercialize RIZAPORT in Spain and a right of first refusal for a predefined term for additional territories. RedHill and IntelGenx received an upfront payment and are entitled to receive additional milestone payments upon the achievement of certain predefined regulatory and commercial targets, as well as tiered royalties. Further financial terms of the agreement were not disclosed. Grupo JUSTE recently filed a national Marketing Authorization Application for RIZAPORT in Spain and commercial launch in Spain is expected to take place in the second half of 2017.

On July 13, 2016, RedHill announced the signing of a research collaboration agreement with the U.S. National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), intended to evaluate RedHill’s proprietary experimental therapy for the treatment of Ebola virus disease. The new research collaboration follows encouraging results from preliminary non-clinical studies conducted in conjunction with the NIAID using RedHill’s proprietary experimental therapy. If successful, this study is intended to provide supportive data for discussions with the FDA for potential use of the Animal Rule pathway for approval. Ebola virus disease is a severe and often fatal illness, which can cause severe hemorrhagic fever in humans and has a mortality rate ranging from 25% to 90%3. There is currently no FDA-approved treatment for Ebola virus disease.

On July 21, 2016, RedHill announced that it had received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a new patent covering RHB-105. The patent application, entitled "Pharmaceutical Compositions For The Treatment Of Helicobacter Pylori"expands RedHill’s patent portfolio covering RHB-105 and is expected to be valid until 2034, once granted.

On August 1, 2016, RedHill announced that the last patient completed the final scheduled follow-up visit in the Phase IIa proof-of-concept clinical study evaluating RHB-104 in patients treated for relapsing-remitting multiple sclerosis (the CEASE-MS study). The analysis of the study is ongoing, with top-line final results expected in Q4/2016. Previously announced interim results after completion of the 24-week RHB-104 treatment period of the study demonstrated positive safety and efficacy signals and support further clinical development.

On August 29, 2016, RedHill announced that it had received from the European Patent Office a Notice of Intention to Grant for a new patent covering the use of RHB-104 in the treatment of multiple sclerosis. Upon grant by the European Patent Office, the patent is expected to be valid until 2032 and can be officially validated in up to 38 European countries.

On September 8, 2016, RedHill announced that a Phase Ib/II clinical study evaluating YELIVA(TM) in patients with refractory or relapsed multiple myeloma was initiated. The open-label, dose escalation Phase Ib/II study is being conducted at Duke University Medical Center and will enroll up to 77 patients with refractory or relapsed multiple myeloma who have previously been treated with proteasome inhibitors and immunomodulatory drugs. The study is supported by a $2 million grant from the National Cancer Institute Small Business Innovation Research Program awarded to Apogee Biotechnology Corp., in conjunction with Duke University, with additional support from RedHill.

On September 12, 2016, RedHill announced a research collaboration with Stanford University School of Medicine for the evaluation of YELIVA(TM). The research collaboration is intended to complement RedHill’s planned Phase Ib clinical study to evaluate YELIVA(TM) as a radioprotectant for prevention of mucositis in head and neck cancer patients undergoing therapeutic radiotherapy. Results from the research collaboration are expected in mid-2017.

On September 21, 2016, RedHill and IntelGenx, announced that they had entered into a binding term sheet with Pharmatronic Co., granting Pharmatronic Co. the exclusive license to commercialize RIZAPORT in the Republic of Korea (South Korea). Under the term sheet, RedHill and IntelGenx are to receive an upfront payment and will be eligible to receive additional milestone payments upon achievement of certain predefined regulatory and commercial targets, as well as tiered royalties. Subject to the satisfaction of the remaining conditions, the parties will endeavor to enter into a definitive agreement within 60 days of the execution of the term sheet. Further financial terms of the term sheet were not disclosed.

On October 5, 2016, RedHill announced that a Phase II clinical study evaluating YELIVA(TM) in patients with advanced hepatocellular carcinoma (HCC) was initiated at the Hollings Cancer Center at the Medical University of South Carolina (MUSC), subject to IND clearance by the FDA. The study is supported by a grant from the U.S. National Cancer Institute (NCI) awarded to MUSC, with additional support from RedHill. The HCC study protocol is still under FDA review. Enrolment in the study is expected to begin following successful completion of the FDA’s review process, anticipated by end of 2016.

On October 6, 2016, RedHill provided an update on the RHB-104 Phase III Crohn’s disease development program, planned enhancements to the MAP US first Phase III study and expected milestones, including:

An increase in the total number of patients planned to be enrolled in the study from 270 to 410 and the addition of an open-label extension study offering all patients who complete 26 weeks of study participation and remain out of remission (Crohn’s disease active index (CDAI) > 150) the opportunity to receive treatment with RHB-104 for a 52-week period.

A first safety-focused independent DSMB meeting is on track to take place in the fourth quarter of 2016. A second independent DSMB meeting is expected in the second quarter of 2017 and will include safety and interim efficacy analysis, with evaluation of an early stop for success for overwhelming efficacy, under pre-specified efficacy criteria.

Two small-scale, open-label ex-U.S. clinical studies with RHB-104, each with up to 20 Crohn’s disease patients, are planned to be initiated and are intended to provide additional supportive clinical data to potential future marketing applications, as well as to evaluate RHB-104’s efficacy in newly diagnosed and treatment-naïve Crohn’s disease patients, and as an add-on therapy to current standard-of-care.

RedHill will remain blinded to the interim and ongoing results from the Phase III study and no changes are planned to the MAP US Phase III study’s primary endpoint or 90% power.

Assuming enrollment of all 410 planned subjects, completion of patient recruitment in the MAP US Phase III study is expected by the end of 2017.
On October 18, 2016, RedHill announced that it had received from the Japan Patent Office a Notice of Allowance for a new patent covering RHB-104 for the treatment of multiple sclerosis, which is expected to be valid until 2032, once granted.

On November 1, 2016, RedHill announced that it had intended to offer its American Depositary Shares (ADSs), each representing 10 of its ordinary shares, in an underwritten public offering. RedHill subsequently announced, on November 2, 2016, that it had withdrawn the proposed underwritten public offering of its ADSs due to market conditions.

On November 1, 2016, RedHill announced that it had entered into a non-binding term sheet with a pharmaceutical company as part of its potential strategic vertical integration plan to build a U.S. specialty pharmaceutical company by establishing a commercial presence and capabilities. Under the term sheet, RedHill would be granted the right to exclusively promote a specialty gastrointestinal product in certain territories in the U.S. The parties would share revenues generated in such territories based on an agreeable split between the parties. RedHill is not required to make any upfront or milestone payments under the term sheet. Although RedHill’s goal is to complete the transaction pertaining to the commercial asset in the fourth quarter of 2016, the term sheet is non-binding and there is no certainty as to the execution nor timing of execution of a definitive agreement between RedHill and its potential partner. There is no assurance that satisfactory due diligence will be completed or that the parties will obtain all necessary corporate approvals.

On November 3, 2016, RedHill announced that top-line results from both the ongoing Phase III clinical study with BEKINDA 24 mg for acute gastroenteritis and gastritis (the GUARD study) and the ongoing Phase II clinical study with BEKINDA 12 mg for diarrhea-predominant irritable bowel syndrome (IBS-D) are expected in mid-2017. Over two-thirds of the planned total of 320 subjects have been enrolled in the Phase III GUARD study with BEKINDA 24 mg for acute gastroenteritis and gastritis in the U.S. Approximately half of the planned total of 120 subjects have been enrolled in the Phase II clinical study with BEKINDA 12 mg for the treatment of IBS-D in the U.S.

On November 10, 2016, RedHill announced that it has concluded a positive Type B Meeting with the U.S. Food and Drug Administration (FDA) discussing the chemistry, manufacturing and controls (CMC) aspects of the RHB-105 Phase III development program. The confirmatory Phase III study with RHB-105 for H. pylori infection is planned to be initiated in H1/2017, after completion of the ongoing supportive PK program. Subject to a successful outcome, the confirmatory Phase III study and the supportive PK program are expected to complete the package required for a U.S. NDA for RHB-105, including clinical data and CMC.

Inovio’s Cancer Immunotherapy (INO-3112) Generates T Cell Immune Responses in Tumor Tissue and Peripheral Blood in Patients with HPV-Associated Head and Neck Cancer

On November 14, 2016 Inovio Pharmaceuticals, Inc. (NASDAQ:INO), reported an interim data analysis showing that its INO-3112 cancer immunotherapy product generated antigen-specific CD8+ killer T cell responses measured both in tumor tissue and in peripheral blood from subjects with head and neck cancer associated with human papillomavirus (HPV) (Press release, Inovio, NOV 14, 2016, View Source [SID1234516589]). The immunology results show that INO-3112 treatment generated robust HPV16/18 specific CD8+ T cell responses in peripheral blood in four of five subjects who also showed increased T cell activation in resected tumor tissue samples. These four subjects remained disease free in continuing follow-up that ranged from nine to 24 months at the time of analysis. One subject with only minimal increases in T cell immune responses developed progressive disease at 11 months post start of the study. These results were presented November 12th at the 2016 Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in National Harbor, Maryland.

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!

Dr. J. Joseph Kim, Inovio’s President and CEO, said, "In immuno-oncology, it’s all about the T cells. We now have evidence in cancer patients that our immunotherapy product can generate antigen-specific CD8+ killer T cell responses in the tumor, a major step forward towards an effective immunotherapy. This study gives us an important opportunity to evaluate a novel treatment approach using a DNA vaccine platform to increase immune activation by generating a robust in-vivo T cell response, especially in the tumor, and potentially decreasing tumor recurrence in HPV positive head and neck cancer patients."

INO-3112, an active immunotherapy targeting HPV 16/18 combined with a DNA plasmid for IL-12 as an immune activator, is designed to activate patients’ immune responses to specifically target and kill HPV associated tumors. This open label phase I/IIa study has fully enrolled twenty-two subjects with HPV-positive head and neck squamous cell carcinoma and is intended to assess the safety, tolerability, and immunogenicity of INO-3112 in two treatment groups. Additionally, the study is evaluating the anti-tumor response and progression free survival of patients. The first group enrolled six subjects who were treated with INO-3112 before and after resection of their tumor. One subject withdrew consent after surgery, leaving five evaluable subjects in this group. All of these subjects received one dose of INO-3112 (averaging 14 days and ranging 7 to 28 days) prior to definitive surgery plus three additional doses post-surgery. The second group enrolled sixteen subjects who received four doses of INO-3112 after at least two months following completion of definitive chemoradiation or surgery and adjuvant chemoradiation therapy.

This poster presentation provided immune response and disease free survival data from the first treatment group. CD8+ and FoxP3 T cell expression were evaluated in tumor samples obtained before and after surgery. In addition, ELISpot analysis was performed to determine the number of T cells capable of secreting IFN-γ in response to HPV antigen stimulation. Four of five subjects had robust T cell response as measured by blood ELISpot assay and the same four subjects also showed an average increase of 60% of CD8+ to FoxP3 ratio measured by immunohistochemistry post vaccination, demonstrating increased infiltration of CD8+ T cells as well as reduction of regulatory T cells measured by FoxP3 expression in tumor tissue. These four subjects remained disease free with follow-up ranging from nine to 24 months to date. One subject with only a marginal increase in ELISpot response magnitude to HPV and no increase in CD8+/FoxP3 ratio in tumor tissue post INO-3112 developed progressive disease at 11 months post-treatment.

Overall the characteristics of these immune response data mirrored those previously observed in a phase IIb clinical study of VGX-3100 for HPV-associated cervical dysplasia. In that study, strong CD8+ T cell immune responses were positively correlated with achievement of primary and secondary efficacy endpoints. VGX-3100 is the first therapy to demonstrate that activated killer T cells induced in the body have the power to clear neoplastic lesions as well as the virus which caused the disease.

Inovio is continuing subject monitoring and comprehensive immune analyses for both cohorts of this study and expects multiple reports of additional data throughout 2017.

In August 2015, Inovio licensed INO-3112 to MedImmune, the global biologics research and development arm of AstraZeneca, for an upfront payment of $27.5 million, $700 million in potential development and commercial milestone payments, and royalties on INO-3112 product sales.

About HPV-Caused Head & Neck Cancer

Human papillomavirus (HPV) is the most common sexually transmitted disease in the United States, currently infecting about 79 million Americans. HPV is known to play a major role in the development of head and neck cancers, which include cancers of the oral cavity, oropharynx, nose/nasal passages and larynx. In 2016 an estimated 48,330 persons will get oral cavity or oropharyngeal cancer in the U.S. New cases of head and neck cancer occur nearly three times more often in men as in women. Incidence rates of head and neck cancers have been on the rise, especially HPV-associated oropharyngeal cancer in men, and are expected to continue growing.

10-Q – Quarterly report [Sections 13 or 15(d)]

Syros Pharmaceuticals has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Syros Pharmaceuticals, 2017, NOV 14, 2016, View Source [SID1234521272]).

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!