U.K. NICE RECOMMENDS ANTICANCER AGENT HALAVEN(R) AS TREATMENT FOR ADVANCED BREAST CANCER

On November 4, 2016 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that its in-house developed anticancer agent Halaven (eribulin mesylate, "eribulin") has been recommended by the U.K. National Institute for Health and Clinical Excellence (NICE) as a treatment for patients with locally advanced or metastatic breast cancer who have received at least two chemotherapeutic regimens for advanced disease (prior therapy may have included an anthracycline or a taxane, and capecitabine) in NICE’s Final Appraisal Determination (FAD) (Press release, Eisai, NOV 4, 2016, View Source [SID1234516247]). 1 Eribulin is the first breast cancer treatment to be recommended by NICE since 2007.
2 Following the issue of the FAD by NICE, eribulin will be eligible for reimbursement for this indication via the National Health Service in England (NHS England).

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 Appraisal Committee considered that the models suggest Eribulin offers a mean overall survival benefit of more than 3 months. According to the FAD, "In light of the short life expectancy at this stage of breast cancer, the committee considered this overall survival benefit to be substantial. The committee concluded that eribulin met the end-of-life criteria objectively and robustly and that it can be considered a life-extending, end-of-life treatment." Eribulin was approved in Europe in March 2011 as a treatment for patients with locally advanced or metastatic breast cancer who have received at least two chemotherapeutic regimens for advanced disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting. The agent was launched in the U.K. in April 2011. Furthermore, eribulin was approved in June 2014 for an expanded indication to include patients with locally advanced or metastatic breast cancer who have progressed after at least one chemotherapeutic regimen for advanced disease (prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting, unless patients were not suitable for these treatments).

Eisai regards oncology as a key therapeutic area and is aiming to discover revolutionary new medicines with the potential to cure cancer. Eisai remains committed to providing further clinical evidence and expanding patient access for eribulin, and by maximizing the value of the drug, seeks to contribute further to addressing the diverse needs of, and increasing the benefits provided to, patients with cancer, their families, and healthcare providers.

About Halaven (eribulin mesylate, "eribulin") Eribulin is the first in the halichondrin class of microtubule dynamics inhibitors with a novel mechanism of action. Structurally eribulin is a simplified and synthetically produced version of halichondrin B, a natural product isolated from the marine sponge Halichondria okadai. Eribulin is believed to work by inhibiting the growth phase of microtubule dynamics which prevents cell division. In addition, recent non-clinical studies showed that eribulin is associated with increased vascular perfusion and permeability in tumor cores.3 Eribulin promotes the epithelial state and decreases the capacity of breast cancer cells to migrate.4 Eribulin was first approved in November 2010 in the United States as a treatment for patients with metastatic breast cancer who have received at least two chemotherapeutic regimens for the treatment of metastatic disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting. Eribulin is currently approved for use in the treatment of breast cancer in approximately 60 countries worldwide, including Japan and countries in Europe, the Americas and Asia. In Japan, eribulin has been approved to treat inoperable or recurrent breast cancer and was launched in the country in July 2011. In addition, eribulin has been approved in countries in Europe and Asia indicated as a treatment for patients with locally advanced or metastatic breast cancer who have progressed after at least one chemotherapeutic regimen for advanced disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting, unless patients were not suitable for these treatments.
Regarding soft tissue sarcoma, eribulin was approved in the United States for the treatment of patients with unresectable or metastatic liposarcoma who have received a prior anthracycline-containing regimen in January 2016, approved in Japan for the treatment of soft tissue sarcoma in February 2016, and approved in Europe for the treatment of adult patients with unresectable liposarcomas who have received prior anthracycline containing therapy (unless unsuitable) for advanced or metastatic disease in May 2016. Applications seeking approval for use in the treatment of soft tissue sarcoma are currently under review in countries including Switzerland, Australia, Brazil and Malaysia.

2. About NICE’s New Approach to the Appraisal and Funding of Cancer Drugs The former Cancer Drugs Fund (CDF) was established in 2009 as a means to improve patients’ access to new cancer drugs including those which were "Not Recommended" by NICE. These drugs would be evaluated and listed in the CDF with their costs reimbursed through the fund. However, due to a severe increase in financial burden, a new scheme for NICE appraisal and funding of cancer drugs, including a new CDF, came into operation on July 29, 2016. All novel cancer drugs that had been newly approved would undergo NICE appraisal while cancer drugs that were listed in the former CDF would be eligible for reappraisal by NICE under the new scheme depending on the judgment of each company. The NICE appraisal process consists initially of an Appraisal Consultation Document, the issue of a Final Appraisal Determination, and ultimately the setting of Final Guidance. While cancer drugs that are "Recommended" are eligible for reimbursement through the NHS England, drugs that are "Not Recommended" require an Individual Funding Request (IFR) to be deliberated on a case-by-case basis, and therefore their use is greatly limited. Cancer drugs that could possibly be recommended but are judged to lack sufficient evidence can be given a "Limited Recommendation under the CDF", with provisional access secured under the CDF for a maximum of two years. After receiving this designation, NICE conducts a reappraisal based on the new evidence, and ultimately determines whether to either "Recommend" or "Not Recommend" the drug.

Cancer Research UK and Cancer Research Technology partner with TYG Oncology to develop next generation cancer vaccine

On November 4, 2016 Cancer Research UK and Cancer Research Technology (CRT), the charity’s commercial arm, reported an agreement with TYG oncology Ltd (TYG) to take its new antigen-specific, active checkpoint control cancer vaccine TYG100, into clinical trials in cancer patients with advanced solid tumours (Press release, Cancer Research Technology, APR 4, 2016, View Source [SID1234523176]).

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!

TYG100 is among a range of new treatments that use a patient’s own immune system to target hormones that encourage the growth and spread of solid tumours. It works by triggering the production of antibodies that specifically target two members of the gastrin hormone family* that help to fuel tumour growth.

Under the agreement, Cancer Research UK and TYG will share the cost of the process development and production of TYG100 for the clinic.

Cancer Research UK’s Centre for Drug Development (CDD) will then sponsor and manage a Phase I clinical trial of TYG100 in cancer patients with advanced solid tumours, mainly to evaluate drug safety and toxicity. It will take place across the Experimental Cancer Medicine Centres (ECMC) network, a nationwide initiative funded by Cancer Research UK and the UK’s four Health Departments.

The collaboration forms part of Cancer Research UK’s Clinical Development Partnership (CPD) scheme, meaning that at the end of the trial, TYG oncology has the option of either retaining rights to the new treatment or transferring them to Cancer Research UK to continue development through a new partner.

Fred Jacobs, president of TYG oncology, said: "We are very pleased to be working in partnership with the clinical experience and expertise from Cancer Research UK to advance TYG100. Cancer Research UK’s sponsorship of the Phase I clinical trial is a significant technical and financial validation of our novel therapeutic vaccine. TYG100 will be attacking some of the most difficult and deadliest solid tumours, including pancreatic and gastro-oesophageal cancers."

Dr Nigel Blackburn, Cancer Research UK’s director of drug development, said: "We hope that next generation cancer vaccines like this one will build on the widespread success of existing hormonal treatments – such as those targeting oestrogen in breast cancer and testosterone in prostate cancer. It’s a very promising area of research and we’re excited to be working with TYG oncology to take this to the next stage."

"Our Clinical Development Partnerships are a novel approach that allows companies to benefit from Cancer Research UK’s extensive drug development resources in a way that is mutually beneficial and that ultimately helps new treatments reach patients more quickly."

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

vTv Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, vTv Therapeutics, 2017, NOV 3, 2016, View Source [SID1234521321]).

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!

Moleculin Biotech, Inc. Reports Financial Results for the Third Quarter Ended September 30, 2016

On November 21 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical and clinical-stage pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center ("MD Anderson"), reported its financial and operating results for the third quarter ended September 30, 2016 (Press release, Moleculin, NOV 3, 2016, View Source [SID1234516729]).

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!

During the third quarter and year to date, key activities included:
Updated the Annamycin clinical strategy to add a Phase I arm to its next Phase II trial that leverages a potential increase in the maximum tolerable drug dose, which could significantly increase the chance for positive outcomes. Despite some likely cost increases, which the Company believes will be offset by the Dermin supply agreement, as well as a likely extension in approval timing by several months, the Company believes that it remains on track to generate useful Phase II data by the second half of 2017;
Secured an agreement with Dermin Sp. Zo. O. to utilize its supply of Annamycin for the Company’s clinical trial, substantially reducing expenditures required of Moleculin for drug product and shortening the time required to produce clinical supplies;
Announced it had received verbal positive guidance from the FDA regarding its planned IND submission indicating that the Company may incorporate by reference the IND established by a prior developer;
Benefitting from additional grant funded research at MD Anderson for WP1066;
Announced promising initial results on the preclinical toxicology work for WP1122. Preliminary escalating single dose toxicity testing in mice was successfully completed. No toxic death was observed and the drug was well tolerated;
Appointed a new CFO, Jonathan P. Foster; and
Completed successful initial public offering and bridge financing.
Planned activities and milestones for the remainder of 2016 include:
Receive pre-IND guidance from FDA for liposomal Annamycin, an anthracycline for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML);
Submit IND for liposomal Annamycin based on FDA guidance;
Strengthen license and IP portfolio; and
Continue development of pipeline assets, including drug and other molecular portfolio.
Walter Klemp, Chairman and CEO of Moleculin stated: "We continue to make progress towards executing on our clinical programs and are pleased with recent developments that allow us to more cost effectively fund our activities and potentially improve target outcomes, while maintaining our milestone to report Phase II data by the second half of 2017. We continue to believe we have sufficient funds to pursue our planned operations through the generation of Phase II data for Annamycin through the end of the third quarter of 2017."
Unaudited Financial Results for the Third Quarter Ended September 30, 2016
Research and development expense was $496,659 and $38,409 for the three months ended September 30, 2016 and 2015, respectively. The increase of approximately $458,000 mainly represents accrued license fees to MD Anderson for approximately $40,000, $37,500 for research performed by HPI, and approximately $228,000 related to MD Anderson sponsored research.
General and administrative expense was $924,041 and $184,344 for the three months ended September 30, 2016 and 2015, respectively. The expense increase of approximately $740,000 was mainly attributable to additional payroll and related expenses of approximately $459,000 related to a full three months of our Chief Financial Officer’s, Chief Operating Officer’s and Chief Executive Officer’s salaries and compensation for our Board of Directors. Also, included in this quarter’s expense was $118,000 related to the severance of the former Chief Financial Officer. The Company also incurred approximately $289,000 of expenses related to investor relations, audit and accounting, and insurance costs.
Interest expense included expense accrued on the Company’s convertible promissory notes issued in 2015 and 2016 bearing interest at the rate of 8% per annum.
The Company’s net loss for the three months ended September 30, 2016 amounted to $1,432,079.
As of September 30, 2016, the Company had $6,183,783 in cash. During the period from January 1, 2016 through May 2, 2016, the Company sold 234,296 common shares for $702,888. On May 31, 2016, the Company completed its initial public offering, pursuant to which it sold 1,540,026 shares of common stock at $6.00 per share for net proceeds of $8,464,183 after deducting underwriting discounts and commissions and direct offering expenses.
Net cash used in operating activities was $2,596,647 for the nine months ended September 30, 2016 and mainly included payments made for payroll, travel, insurance and professional fees to the Company’s consultants, attorneys and accountants for services related to becoming a publicly traded company and related filing fees, along with payments made to MD Anderson for license and maintenance fees. Additionally, prepayments were made for insurance.
Net cash used in investing activities was $109,793 for the nine months ended September 30, 2016 and primarily represents the cash paid to acquire Moleculin, LLC.
Net cash provided by financing activities was $8,862,132 for the nine months ended September 30, 2016. The Company received $8,464,183 net proceeds from its IPO stock issuance, $702,888 from issuance of common shares at $3 per share, and $165,000 from issuance of convertible notes. Net cash used in financing activities included approximately $470,000 for payments of notes payable.
(Tables to follow)
Restatement of the Unaudited Financial Results for the Second Quarter Ended
June 30, 2016
Today, the Company filed its restated unaudited financial statements for the Second Quarter Ended June 30, 2016 with the Security and Exchange Commission ("SEC") on Form 10-Q/A.
The Company identified the following non-cash errors due to an error in the accounting for the business combination of Moleculin, LLC. The impact of the correction of the error was as follows:
1 – The net loss for the three and six months ended June 30, 2016 was overstated by approximately $256,889 due to amortization of an intangible which was recorded in error. Upon correction, the net loss for the period will be $738,727 and $1,070,968, respectively.
2 – A liability in the amount of $750,000 should not have been reflected in the balance sheet as of June 30, 2016. Upon correction for this and item 1 above, the total for Liabilities and Stockholders’ Equity was $18,740,288.
3 – Intangibles were overstated by $750,000 before the amortization mentioned above. Upon correction, total assets was $18,740,288.

Poseida Therapeutics to Present Three Abstracts on Novel BCMA-Specific CAR-T Therapy at American Society of Hematology 2016 Annual Meeting

On November 3, 2016 Poseida Therapeutics Inc. ("Poseida"), a San Diego-based company translating best-in-class gene editing technologies into lifesaving therapeutics, reported that three abstracts detailing preclinical data on P-BCMA-101, the company’s BCMA-specific CAR-T drug candidate, have been selected for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Poseida Therapeutics, NOV 3, 2016, View Source [SID1234516624]). The studies explore utility and performance of Poseida’s proprietary CAR-T modifications using its piggyBac non-viral gene delivery system and Centyrin binding scaffold. Data will be presented at the 2016 ASH (Free ASH Whitepaper) Annual Meeting in San Diego on December 3rd and 5th. Abstracts are currently available online at View Source

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 following abstracts will be presented:

Presentation Title: A Novel BCMA-Specific, Centyrin-Based CAR-T Product for the Treatment of Multiple Myeloma
Abstract Number: 2127
Date and Time: 5:30 p.m. – 7:30 p.m., Saturday, December 3, 2016
Location: San Diego Convention Center, Hall GH

Presentation Title: piggyBac-Produced CAR-T Cells Exhibit Stem-Cell Memory Phenotype
Abstract Number: 2167
Date and Time: 5:30 p.m. – 7:30 p.m., Saturday, December 3, 2016
Location: San Diego Convention Center, Hall GH

Presentation Title: Development of Novel Non-Immunoglobulin Centyrin-Based CARs (CARTyrins) Targeting Human BCMA
Abstract Number: 4557
Date and Time: 6:00 p.m. – 8:00 p.m., Monday, December 5, 2016
Location: San Diego Convention Center, Hall GH