On March 13, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported interim clinical data of BPX-501 in pediatric patients with acute myeloid leukemia (AML) and primary immunodeficiencies (PIDs). BPX-501 is an adjunct T cell therapy incorporating CaspaCIDe administered after haploidentical hematopoietic stem cell transplant (haplo-HSCT) for the treatment of hematologic cancers and inherited blood diseases (Press release, Bellicum Pharmaceuticals, MAR 13, 2018, View Source [SID1234524699]).

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Data from the ongoing BP-004 trial suggest that BPX-501 T cells may contribute to a durable anti-leukemic effect in patients with AML. In the study, 38 pediatric AML patients in their first (n=13) or second (n=25) complete response underwent a haplo-HSCT followed by treatment with BPX-501. With a median follow-up of one year, rates of relapse-free survival and overall survival were 91.5% and 97.3%, respectively. This compares to one-year rates reported in the literature in pediatric AML patients undergoing alternate-donor HSCT of 60% to 80%.

"The recurrence of cancer is one of the most serious risks to AML patients receiving a stem cell transplant. These impressive results in children with AML suggest that BPX-501 may be effectively reducing or eradicating residual cancer cells following a haplo-transplant procedure," commented Neena Kapoor, M.D., Director of the Blood and Marrow Transplantation Program at Children’s Hospital of Los Angeles and an investigator in the BP-004 trial.

Also from the BP-004 study, the Company reported high rates of disease-free survival and overall survival in pediatric patients with PIDs, including Severe Combined Immunodeficiency ("bubble boy disease"), Wiskott-Aldrich syndrome, Chronic Granulomatous Disease, and other rare immune deficiencies. Of 59 pediatric PID patients undergoing a haplo-HSCT and treatment with BPX-501, disease-free survival was reported at 88.1% and overall survival was reported at 88.6% with a median follow-up of one year.

Continued Dr. Kapoor: "Delayed immune reconstitution can lead to severe infectious complications, a major cause of morbidity and mortality in PID patients who undergo a T-depleted haplo-HSCT. BPX-501 donor T cells administered after a transplant support immune recovery in these patients, and the CaspaCIDe safety switch engineered into BPX-501 may provide a critical safety net to address the risk of uncontrolled GvHD from donor T cells."

Based on these clinical data, Bellicum is working with the investigators and the U.S. Food and Drug Administration to develop a protocol for a potential U.S. registration study in pediatric patients. Pending regulatory clearances, the Company expects to initiate the study by the end of 2018. These clinical data have been submitted for presentation at an upcoming medical meeting.

About BPX-501
BPX-501 is an adjunct T cell therapy administered after allogeneic HSCT, comprising genetically modified donor T cells incorporating Bellicum’s CaspaCIDe safety switch. It is designed to provide a safety net to eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD or other T-cell mediated complications occur. This enables physicians to more safely perform stem cell transplants by administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections and enhance Graft-versus-leukemic effect without unacceptable GvHD risk. The ongoing BP-004 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.

About CaspaCIDe
CaspaCIDe (also known as inducible Caspase-9, or iC9) is the Company’s safety switch, incorporated into certain Bellicum product candidates. The CaspaCIDe switch consists of the Chemical Induction of Dimerization, or CID, binding domain coupled to the signaling domain of Caspase-9, an enzyme that is an integral part of the apoptotic, cell death pathway. If a patient experiences a serious side effect, the activator agent, rimiducid, is administered to trigger dimerization and activation of the safety switch, which in turn leads to selective apoptosis of the CaspaCIDe-expressing cells.

On March 13, 2018 IDEAYA Biosciences, Inc., an oncology-focused biotechnology company committed to the discovery of breakthrough synthetic lethality medicines and immuno-oncology therapies, reported the appointment of Julie Hambleton, M.D., as senior vice president and chief medical officer, head of development (Press release, Ideaya Biosciences, MAR 13, 2018, View Source [SID1234525130]). Dr. Hambleton brings more than 20 years of experience in clinical drug development, ranging from pre-clinical studies through Phase 4 and post-marketing studies. She has extensive experience working with regulatory agencies, including the U.S. FDA and the European Medicines Agency (EMA), and in filing of investigational new drug applications (INDs), biologics license applications (BLAs), and special protocol assessments (SPAs).

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"Dr. Hambleton is an accomplished industry executive and a leading oncology drug developer, and we welcome her to the leadership team at IDEAYA," said Yujiro S. Hata, chief executive officer of IDEAYA. "Dr. Hambleton’s unique clinical background in both immuno-oncology and synthetic lethality will strengthen our organization’s capabilities as we advance multiple first-in-class programs into the clinic, leveraging our clinical strategy in patient selection and combinations."

Julie Hambleton, M.D.

"Over the past two decades, I have been involved in the development of numerous targeted and biomarker enabled therapies, including Avastin and Rubraca, and first-in-class immuno-oncology agents that have been developed in combination with checkpoint inhibitors," said Dr. Hambleton. "IDEAYA’s diverse pipeline of biomarker enabled synthetic lethality programs and immunotherapies targeting immuno-metabolism and innate immunity have the potential to transform oncology care and deliver on our mission to bring innovative personalized therapies to cancer patients."

Dr. Hambleton was previously vice president, head of US medical at Bristol-Myers Squibb, overseeing medical & health economic and outcomes research activities in support of the oncology, immuno-oncology, specialty and cardiovascular marketed portfolios. From 2012 to 2016, she was executive vice president and chief medical officer at Five Prime Therapeutics, where she led clinical, clinical operations, regulatory, preclinical and translational and process development and was a member of the executive committee. From 2010 to 2012, Dr. Hambleton was vice president, clinical development, at Clovis Oncology, where she oversaw clinical development programs and managed clinical and pharmacokinetic functions, including the development of Rubraca, a PARP inhibitor, developed with a diagnostic for BRCA and HRD. From 2003 to 2010, she was at Genentech, most recently as group medical director, global clinical development, leading a cross-functional group conducting multiple Phase 2 and 3 trials of Avastin.

Prior to joining industry, Dr. Hambleton served from 1993 to 2003 in academic positions in the division of hematology-oncology at the University of California, San Francisco (UCSF), most recently as associate professor of clinical medicine. She completed post-graduate training at UCSF, where she served as medical resident, chief medical resident and fellow in the division of hematology-oncology. She received a B.S. from Duke University in nursing, an M.D. from Case Western Reserve University School of Medicine, and was board-certified in hematology and internal medicine.

On March 13, 2018 GT Biopharma Inc. (OTCQB: GTBP) (Euronext Paris: GTBP.PA) reported that Shawn Cross, Chairman and Chief Executive Officer, and Raymond Urbanski, Chief Medical Officer, will present a company overview at the Roth Capital Partners 30th Annual Conference on Tuesday, March 13, at 11:30 a.m. PT (Press release, GT Biopharma , MAR 13, 2018, View Source [SID1234539532]).

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A live audio webcast of the event will be available via the ‘Investor Relations’ page of the GT Biopharma website, View Source Please log on through GT Biopharma’s website approximately 10 minutes before the scheduled start time. A replay of the webcast will be archived on Gt Biopharma’s website for 90 days following the call.

On March 13, 2018 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that its shareholders approved all proposals of the Board of Directors at its ordinary Annual General Meeting (AGM) (Press release, Hoffmann-La Roche, MAR 13, 2018, View Source [SID1234524733]). The 880 attending shareholders, representing 86,42% of the total of 160,000,000 shares, approved the Management Report, the Financial Statements and the Consolidated Financial Statements for 2017.

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Christoph Franz was confirmed as Chairman of the Board of Directors with 99,89% of the votes.

"2017 was a good year for Roche. The launch of Ocrevus for people with multiple sclerosis and the approval of Hemlibra for the treatment of people with haemophilia A were outstanding. In particular, we made significant progress in the areas of personalised healthcare and digitization. In this field, we have entered into important partnerships that complement our own expertise. I believe Roche is very well prepared for the future," said Christoph Franz, Chairman of Roche’s Board of Directors.

Shareholders also authorised the ratification of the Board of Directors’ actions and approved an increase in the gross dividend for the past financial year to 8.30 Swiss francs (gross) per share and non-voting equity security. This is the 31st consecutive dividend increase.

Shareholders endorsed the total amount of bonuses paid to the Corporate Executive Committee for 2017 with 99,62% of the votes, as well as the bonus paid to the Chairman of the Board of Directors for 2017, which was approved by 99,51% of the votes. They also approved the maximum total amount of future remuneration of the Board of Directors, which was confirmed by 99,62% of the votes, and the maximum total amount of future remuneration for the Corporate Executive Committee until the 2019 ordinary Annual General Meeting by 99,64% of the votes.

In addition to Christoph Franz, all other Board members who stood for re-election were confirmed for a term of one year:

André Hoffmann
Professor Sir John Bell
Julie Brown
Paul Bulcke
Anita Hauser
Professor Dr Richard P. Lifton
Dr Andreas Oeri
Bernard Poussot
Dr Severin Schwan
Dr Claudia Süssmuth Dyckerhoff
Peter R. Voser

The following Board members were elected to the Remuneration Committee:

Dr Christoph Franz
André Hoffmann
Professor Dr Richard P. Lifton
Bernard Poussot
Peter R. Voser

KPMG AG was elected as external auditor for the financial year 2018 and BDO AG was elected as independent voting proxy until the conclusion of the next regular AGM in 2019.

On March 12, 2018 Eleven Biotherapeutics, Inc. (NASDAQ: EBIO), a late-stage clinical company advancing next-generation antibody-drug conjugate (ADC) therapies for the treatment of cancer, reported that the company has completed enrollment in the VISTA Phase 3 registration trial of Vicinium in patients with non-muscle invasive bladder cancer (NMIBC) who have been previously treated with bacillus Calmette-Guérin (BCG) (Press release, Eleven Biotherapeutics, MAR 13, 2018, View Source [SID1234524701]). Bladder cancer is the sixth most common cancer in the United States and approximately 80 percent of bladder cancer patients are diagnosed with NMIBC. Vicinium is a fusion protein, designed to be a next-generation ADC, which specifically targets the epithelial cell adhesion molecule (EpCAM) antigens on the surface of bladder cancer cells to deliver a potent cytotoxin to those cells.
"Bladder cancer is one of the most prevalent cancers in the United States, yet there has been limited development of new therapeutic options for patients in more than 30 years," commented Donald Lamm, M.D., University of Arizona professor, director of BCG oncology and an investigator in the VISTA trial. "Today’s standard-of-care for NMIBC provides initial responses in many patients; however, after BCG is no longer effective, there are no meaningful FDA-approved options except surgical removal of the bladder in high-risk patients. I am encouraged by the data demonstrated with Vicinium in prior trials and its potential to offer my patients an alternative to radical cystectomy."

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"We believe Vicinium is the most advanced candidate in development for NMIBC and has the potential to be an effective and tolerable treatment for patients who have been previously treated with BCG," said Stephen Hurly, president and chief executive officer of Eleven Biotherapeutics. "The complete response rate and favorable safety seen in our Phase 2 trial were encouraging, and based on learnings from that trial, we modified the dosing regimen to potentially further enhance responses in the VISTA trial. With Phase 3 recruitment complete, we are on-track to report topline, three-month data in mid-2018, and look forward to further assessing Vicinium’s potential in treating patients with this devastating cancer."

About the VISTA Clinical Trial
The VISTA trial is an open-label, multicenter, single-arm Phase 3 clinical trial evaluating the efficacy and tolerability of Vicinium in patients with high-risk non-muscle invasive bladder cancer (NMIBC) that is carcinoma in situ (CIS, cancer found on the inner lining of the bladder that has not spread into muscle or other tissue) or papillary (cancer that has grown from the bladder lining out into the bladder but has not spread into muscle or other tissue), who have been previously treated with bacillus Calmette-Guérin (BCG). The primary endpoint of the trial is the complete response rate in patients with CIS with or without papillary disease. Patients in the

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study receive locally administered Vicinium twice a week for six weeks, followed by once-weekly treatment for another six weeks, then treatment every other week for up to two years. Topline data assessing responses and durability of responses at three-months on treatment are expected in mid-2018, with 12-month data anticipated in mid-2019. For more information, please visit www.mybladdercancer.com.

About Vicinium
Vicinium, Eleven Biotherapeutics’ lead product candidate, is a next-generation antibody-drug conjugate developed using the company’s proprietary Targeted Protein Therapeutics platform. Vicinium is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A (ETA). Vicinium is constructed with a stable, genetically engineered linker to ensure the payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical studies conducted by Eleven Biotherapeutics, EpCAM has been shown to be overexpressed in non-muscle invasive bladder cancer (NMIBC) cells with minimal to no EpCAM expression observed on normal bladder cells. Eleven Biotherapeutics is currently conducting the Phase 3 VISTA trial, designed to support the registration of Vicinium for the treatment of NMIBC in patients who have previously received two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Topline, three-month data from the trial are expected in mid-2018. The activity of Vicinium in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.

About Non-Muscle Invasive Bladder Cancer
Bladder cancer is the sixth most commonly diagnosed cancer in the United States, and approximately 80 percent of patients have non-muscle invasive bladder cancer (NMIBC). In NMIBC, cancer cells are in the lining of the bladder or have grown into the lumen of the bladder, but have not spread into muscle or other tissue. NMIBC primarily affects men and is associated with carcinogen exposure. Initial treatment includes surgical resection; however, there is a high rate of recurrence and more than 60 percent of all patients diagnosed with NMIBC will receive bacillus Calmette-Guérin (BCG) immunotherapy. While BCG is effective in many patients, challenges with tolerability have been observed and many patients will experience recurrence of disease. If BCG is not effective or a patient can longer receive BCG, the recommended option for treatment is radical cystectomy, the complete removal of the bladder.