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Moleculin Receives Orphan Drug Designation for Annamycin for the Treatment of Acute Myeloid Leukemia

On March 22, 2017 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical 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, reported its lead candidate, Annamycin (also known as "Liposomal Annamycin"), an anthracycline, has received Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML) (Press release, Moleculin, MAR 22, 2017, View Source [SID1234518240]).

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Moleculin’s Chairman and CEO, Walter Klemp, commented, "We are pleased to report this key milestone and the FDA’s decision to grant Annamycin orphan drug designation. We look forward to announcing additional milestones in regard to our clinical pathway as we make further progress."
The FDA grants orphan drug designation to drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

About AML
Leukemia is a cancer of the white blood cells and the acute forms of leukemia can manifest quickly and leave patients with limited treatment options. AML is the most common type of acute leukemia in adults. It occurs when a clone of leukemic progenitor white blood cells proliferates in the bone marrow suppressing the production of normal blood cells. In order to qualify for a curative bone marrow transplant, patients must first undergo induction therapy. The current standard of care is the combining of 2 chemotherapeutic drugs, always including an anthracycline intended to induce a CR or complete response, which has not improved since it was first used in the 1970’s. We estimate that it has the same cure rate of about 20% as then. Currently, the only viable long term option for acute leukemia patients is a bone marrow transplant for those 20%, which is successful in a significant number of patients. For more information on AML click: View Source

About Annamycin
Annamycin is an anthracycline intended for the treatment of relapsed or refractory AML. Annamycin is a unique liposome formulated anthracycline (also referred to in literature as "L-Annamycin") that has been designed to produce little to no cardiotoxicity and avoid the multidrug resistance mechanisms that often defeat current anthracyclines. It has been tested in 114 patients in 6 clinical trials, 3 of which focused on leukemia, with little to no cardiotoxicity and 3 of those clinical trials focused on leukemia. The Company is working with the FDA on an investigative new drug application for a Phase I/II trial for second line treatment of relapsed or refractory AML, for which no approved therapy currently exists.

Pieris Pharmaceuticals Reports Full-Year 2016 Financial Results and Corporate Update

On March 22, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for cancer and other diseases, today reported financial results for the fourth quarter and fiscal year of 2016 and provided an update on the Company’s recent developments (Press release, Pieris Pharmaceuticals, MAR 22, 2017, View Source [SID1234518242]).

"2016 was a highly productive year for Pieris marked by: i) broad advancement of our proprietary clinical and preclinical programs, ii) completion of a $16.5 million private placement financing that strengthened our shareholder base, iii) achievement of several milestones in our collaborative programs, and iv) expansion of our Board of Directors and the appointment of a Chief Business Officer. At several R&D conferences throughout the year, including in the fourth quarter at the annual meeting of the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), we presented preclinical data demonstrating a differentiated mode of action for PRS-343, our lead 4-1BB (CD137)-based HER-2 bispecific immuno-oncology program, which remains on track for Phase I initiation in the first half of this year," said Stephen Yoder, President and CEO. "Our targeted, inhaled asthma program, PRS-060, which engages the IL4a receptor, is differentiated from systemically administered therapies, and is on track to enter a Phase I study in mid-2017. Pieris has also completed dosing of all patients in a Phase Ib single ascending dose study of our most advanced program, PRS-080, in dialysis-dependent chronic kidney disease patients and expects to present this data in the first half of this year."

"With these accomplishments behind us, we started 2017 on a very strong note, as we have already consummated a multi-target, multi-year, transformative partnership in the immuno-oncology space with Servier, the second largest pharmaceutical company in France. This alliance includes our dual checkpoint inhibitor, PRS-332, as well as four additional bispecific programs and may be expanded to eight total programs. Notably, Pieris has the option to co-develop and retain full US rights for four of these programs, including PRS-332, and is eligible to receive up to approximately $1.8 billion in total potential milestones, and up to low double-digit royalties on potential future product sales, in addition to having received an upfront payment of approximately $31.0 million. We also recently announced a regional partnership in Japan with Aska Pharmaceutical Co., Ltd. for PRS-080, which will allow us to invest in manufacturing efficiencies and drug supply for additional clinical studies beyond our planned Phase IIa study, which we believe could help set the stage for additional potential partnerships outside of Japan, following the completion of that study. Finally, we continue to advance our preclinical portfolio of novel multispecific therapeutic proteins, as well as our existing partnerships, while continuing to explore additional collaborations. We ended the fourth quarter in a solid financial position and, considering the upfront payments we have received in the first quarter of 2017, we believe we can manage our financial runway into 2019, enabling us to reach several key value inflection points along the way."

Fourth Quarter and 2016 Highlights:

Advanced PRS-080 through an ongoing Phase Ib single ascending dose study in anemia of chronic disease, having completed patient dosing in early 2017, which will assess the effect of PRS-080 on iron mobilization and transferring saturation in dialysis-dependent anemia patients.
Advanced PRS-343 through IND-enabling studies and towards a first-in-patient study for HER-2 positive cancers.
Advanced PRS-060, a novel inhaled therapeutic for moderate to severe asthma, through IND-enabling studies.
Advanced our novel multi-checkpoint blockade bispecific, PRS-332, comprised of an anti-PD-1 antibody genetically linked to an existing Anticalin against an undisclosed checkpoint, through preclinical studies.
Strengthened our Board of Directors with the addition of Julian Adams, Ph.D. and Christopher Kiritsy.
Dr. Adams is the former President of Research & Development at Infinity Pharmaceuticals. During his career, Dr. Adams has had global responsibility for multiple drug discovery programs, including the discovery and development of Velcade (bortezomib), a proteasome inhibitor for cancer therapy, and Viramune (nevirapine) for HIV. Dr. Adams has received many awards, including the 2012 Warren Alpert Foundation Prize for his role in the discovery and development of bortezomib, the 2012 C. Chester Stock Award Lectureship from Memorial Sloan-Kettering Cancer Center, and the 2001 Ribbon of Hope Award for Velcade from the International Myeloma Foundation.
Mr. Kiritsy is the Chief Executive Officer and co-founder of Arisaph Pharmaceuticals. Prior to Arisaph, Mr. Kiritsy served as Executive Vice President, Corporate Development and Chief Financial Officer of Kos Pharmaceuticals, Inc., where he played a key operating role in building the company from start-up to a highly profitable, publicly traded, commercial company.
Appointed Claude Knopf as Senior Vice President and Chief Business Officer. Prior to joining Pieris, Mr. Knopf served as Global Head Business Development & Licensing/Mergers and Acquisitions at Baxalta. Prior to joining Baxalta, a spin-off of Baxter where he held a similar position for the Baxter Bioscience Division up to the creation of Baxalta. Prior to joining Baxter, Mr. Knopf held several business development, alliance management, and licensing and marketing roles at Novartis, most recently as the Head of Business Development and Licensing, Strategic Planning, Vaccines European Region.
Fiscal Year Financial Update:

Cash Position – Cash and cash equivalents totalled $29.4 million as of December 31, 2016, compared to $29.3 million as of December 31, 2015. The increase in cash was driven primarily by the $16.5 million gross private placement financing completed in June 2016 offset by cash used in our operating activities.

R&D Expense – Research and development expenses were $19.7 million for the year ended December 31, 2016, compared to $8.2 million for the year ended December 31, 2015. The $11.5 million increase was primarily due to a $5.6 million increase in pre-clinical development and CMC costs for PRS-343 as we carry out IND enabling studies and increased development costs for our other PRS-300 series programs, and a $1.2 million increase in CMC costs associated with PRS-060 as we carry out IND enabling studies, offset by a $0.2 million decrease for our PRS-080 program due to the completion of our Phase Ia clinical trial in 2015. Other R&D expenses also increased by $4.9 million primarily due to higher personnel-related expenses including stock-based compensation expense and increased costs for license fees, as well as higher legal and consulting costs. Additionally, costs for general lab supplies increased due to an upturn in program activities.

G&A Expense – General and administrative expenses for the year ended December 31, 2016 were $8.9 million, compared to $8.4 million for the year ended December 31, 2015. The $0.5 million increase in G&A expenses is primarily due to an increase in personnel-related costs, including stock-based compensation expense, higher legal and recruiting costs, and costs associated with being a public company such as financial printing costs and transaction fees.

Net Loss – Net loss was $22.8 million or ($0.55) per share for the year ended December 31, 2016, compared to a net loss $14.1 million or ($0.41) per share for the year ended December 31, 2015.

Upcoming Milestones:

The Company expects to reach the following milestones during 2017:

PRS-080: Present Phase Ib data and initiate a multi-dose, Phase IIa study in dialysis-dependent anemia patients during the second quarter, which we estimate will be completed by the end of 2017.
PRS-343: Initiate a Phase I multi-ascending dose study involving a range of HER2-positive solid cancers representing unmet medical needs (such as breast, gastrointestinal and bladder cancers) in the first half of 2017.
PRS-332: Progress preclinical evaluation in collaboration with Servier, with IND-enabling activities planned for later in 2017.
PRS-060: Initiate a Phase I study in mid-2017.
Upcoming Scientific Presentations:

PRS-343: IND-enabling data informing the design of a first-in-patient clinical trial for PRS-343 will be presented in a poster session at next month’s Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) to be held in Washington D.C. The poster will be presented on Tuesday, April 4, 2017 in a session from 8am to 12pm EDT.

PharmaCyte Biotech Strengthens Protection of Cancer Therapy with Patent Filing

On March 22, 2017 PharmaCyte Biotech, Inc. (OTCQB:PMCB), a clinical stage biotechnology company focused on developing targeted treatments for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has filed a provisional patent application with the United States Patent and Trademark Office (USPTO) to protect its therapy to treat cancerous tumors, including the therapy that will be used in its upcoming clinical trial in locally advanced pancreas cancer (LAPC) (Press release, PharmaCyte Biotech, MAR 22, 2017, View Source [SID1234518239]).

The patent application specifically includes methods of treating all cancerous tumors, such as pancreas, liver, breast and colon, using the live-cell encapsulation of genetically modified human cells that overexpress a form of the Cytochrome P450 enzyme system normally found in the liver. These cells are encapsulated using the Cell-in-a-Box technology. Together with low doses of ifosfamide, the encapsulated cells comprise PharmaCyte’s therapy for cancerous tumors. The patent application also includes using PharmaCyte’s platform technology with cyclophosphamide, another chemotherapy drug that must be activated by the Cytochrome P450 enzyme system.

"By filing for a new patent, we have begun taking steps to obtain patent protection for 20 years to protect our therapy for all forms of malignant tumors. This is particularly important to the company as we are taking steps to embark upon a clinical trial in pancreas cancer," said PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner. "We will be filing for new patent protection in all of the countries in which PharmaCyte currently has patent protection for pancreas cancer."

Provisional patent applications are a way to establish and protect a "date of invention" or "priority filing date" for one year. The provisional patent application was created to provide inventors with a way to begin protecting their inventions. A provisional patent application provides PharmaCyte 12 months to prepare a full patent application during which it can label its inventions as "patent pending." It also enables PharmaCyte to establish an early effective filing date for a patent.

The family of patents that deal with the subject matter of the new patent application are set to expire on March 27, 2017. The new patent application is designed to continue patent protection of PharmaCyte’s therapy for cancerous tumors. It is not an extension of the existing patents. A new patent for PharmaCyte’s cancer therapy, if granted by the USPTO, will provide another 20 years of patent protection from the date of the filing of this Provisional Patent Application – March 21, 2017.

PharmaCyte therapy for pancreas cancer is already protected. PharmaCyte’s pancreas cancer therapy was designated an orphan drug and listed in the official registry of medicinal products for rare diseases by the U.S. Food and Drug Administration (FDA) on December 17, 2014. This orphan drug status assures marketing exclusivity for PharmaCyte’s pancreas cancer therapy in the U.S. for 7 years after market approval by the FDA. Similarly, PharmaCyte has orphan drug status in the European Union (EU) for its pancreas cancer therapy. This designation provides 10 years of marketing exclusivity in all countries in the EU following approval by the European Medicines Agency (EMA).

In addition, the Biologics Price Competition and Innovation Act (BPCIA), which was enacted as part of the Affordable Care Act in 2010, establishes a period of 12 years of "data exclusivity" for reference products to preserve incentives for future innovation. Under this framework, data exclusivity protects the data in the innovator’s regulatory application by prohibiting others, for a period of 12 years, from gaining FDA approval based in part on reliance on or reference to the innovator’s data in a biosimilar application. PharmaCyte’s 12-year exclusivity will begin as soon as the FDA approves the company’s pancreas cancer therapy.

Mr. Waggoner concluded by stating, "While this patent application should make our investors feel assured about the protection of our pancreas cancer therapy, they should understand that if our pancreas cancer therapy receives FDA approval, the orphan drug designation in the U.S. and the EU, together with the BPCIA data exclusivity, will give us substantial marketing exclusivity for our pancreas cancer therapy. This new patent application, while it does include our pancreas cancer therapy, should really be viewed as an opportunity to dramatically broaden PharmaCyte’s ability to protect our therapy for all malignant tumors for the next 20 years."

AVEO Reports Full Year 2016 Financial Results and Provides Business Update

On March 22, 2017 AVEO Oncology (NASDAQ:AVEO) reported financial results for the full year ended December 31, 2016 and provided a business update (Press release, AVEO, MAR 22, 2017, View Source [SID1234518237]).

"TIVO-3, our lead clinical program designed to serve as the basis for a potential U.S. registration for tivozanib as a first- and third-line treatment for renal cell cancer, continues to enroll ahead of schedule and has now completed its first safety review," said Michael Bailey, president and chief executive officer of AVEO. "We look forward to the study’s pre-planned interim futility analysis midyear 2017 and, potentially, to top line results in the first quarter of 2018 and to initial data from our Phase 1 TiNivo study of tivozanib in combination with Opdivo in the first half of 2017. We also continue to support our partner EUSA Pharma in its efforts to complete the European Marketing Authorization Application review for tivozanib as a first-line treatment for renal cell carcinoma. There remains a significant unmet need for better tolerated therapies in this disease, particularly those that enable combination treatment, and we look forward to receiving tivozanib data and to potential regulatory milestones in the coming quarters."

Mr. Bailey continued: "We also look forward to several milestones with the balance of our pipeline, including the presentation of data from two investigator sponsored studies of ficlatuzumab, a potential partnership for AV-353, and progress toward the clinic for AV-380 and AV-203."

Recent Updates

TIVO-3 Enrolling Ahead of Schedule and Passes First Safety Monitoring Committee Safety Review; Pre-Planned Interim Futility Analysis Expected Midyear 2017. In February 2017, AVEO announced that its pivotal, Phase 3 TIVO-3 trial, a randomized, controlled, multi-center, open-label study to compare tivozanib to sorafenib in subjects with refractory advanced renal cell carcinoma (RCC), has successfully completed the first safety review by the study’s Safety Monitoring Committee (SMC). The SMC concluded that no safety concern was observed for tivozanib and recommended that the study replace the small number of patients who dropped out prior to starting treatment. The Company announced just prior to the safety review that the TIVO-3 trial is enrolling substantially ahead of schedule. With the SMC recommendation to replace early dropouts, the Company still expects to complete enrollment in June 2017, ahead of its prior guidance of August 2017. A pre-planned futility analysis of the trial is expected around midyear 2017, with topline data expected in the first quarter of 2018. The TIVO-3 trial, together with the previously completed TIVO-1 trial of tivozanib in the first-line treatment of RCC, is designed to support potential regulatory approval of tivozanib in the U.S. as a third- and first-line treatment for RCC.
First Patient Dosed in Phase 1/2 TiNivo Trial Evaluating Tivozanib in Combination with Bristol-Myers Squibb’s Opdivo (nivolumab) in Advanced RCC. AVEO announced today that the first patient has been treated in the Company’s Phase 1/2 TiNivo trial evaluating tivozanib in combination with Bristol-Myers Squibb’s anti-PD-1 therapy, Opdivo (nivolumab), in advanced RCC. The study, which is led by the Institut Gustave Roussy in Paris, is under the direction of Professor Bernard Escudier, MD, Chairman of the Genitourinary Oncology Committee. The Phase 1, which the Company expects to complete in the first half of 2017, will primarily evaluate the safety of tivozanib in combination with nivolumab at escalating doses of tivozanib. If the Company receives favorable results, it expects to follow immediately with an expansion Phase 2 at the established combination dose.
Ongoing Review of the Marketing Authorization Application (MAA) in Europe for Approval of Tivozanib as a First-Line RCC Treatment Option. In February 2017, AVEO announced that its European licensee for tivozanib, EUSA Pharma, a specialty pharmaceutical company with a focus on oncology and oncology supportive care, has received the Day 180 List of Outstanding Issues (LOI) from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The Day 180 LOI signifies that the MAA is not approvable at the present time and outlines outstanding deficiencies, which are then required to be satisfactorily addressed in an oral explanation and/or in writing prior to a final application decision. EUSA has informed AVEO that it expects to submit written responses to the Day 180 LOI in April 2017, and the EMA has tentatively scheduled EUSA to provide an oral explanation to the CHMP in May 2017.
Submitted for Presentation Results from Phase 1 Studies of Ficlatuzumab in Combination with Cetuximab in Head and Neck Squamous Cell Cancer (HNSCC) and Cytarabine in Acute Myeloid Leukemia (AML). The Company announced today that results from two investigator sponsored Phase 1 studies of ficlatuzumab were submitted for presentation at an upcoming major medical meeting. The first study is designed to explore cetuximab in combination with ascending doses of ficlatuzumab in patients with cetuximab-refractory HNSCC patients. The second study is designed to explore cytarabine in combination with ascending doses of ficlatuzumab in relapsed/refractory AML. AVEO and Biodesix, Inc. have a worldwide agreement to develop and commercialize ficlatuzumab.
Full Year 2016 Financial Highlights

AVEO ended 2016 with $23.3 million in cash, cash equivalents and marketable securities as compared with $34.1 million at December 31, 2015.
Total collaboration revenue for 2016 was approximately $2.5 million compared with $19.0 million for 2015.
Research and development expense for 2016 was $23.7 million compared with $12.9 million for 2015.
General and administrative expenses for 2016 were $8.2 million compared with $14.2 million for 2015.
Net loss for 2016 was $26.9 million, or a loss of $0.39 per basic and diluted share, compared with net loss of $15.0 million for 2015, or a loss of $0.27 per basic and diluted share.
Financial Guidance

We believe that our $23.3 million in existing cash, cash equivalents and marketable securities as of December 31, 2016 could allow us to fund our planned operations into the fourth quarter of 2017; however, additional funds will be needed to extend these operations into 2018 and maintain compliance with our $10.0 million financial covenant under our loan agreement with Hercules.

Sunesis Pharmaceuticals Announces Submission of Responses to the EMA Day 180 List of Outstanding Issues for Marketing Authorization Application for Vosaroxin

On March 22, 2017 Sunesis Pharmaceuticals, Inc. (Nasdaq:SNSS) reported that it has submitted its responses to the European Medicine Agency (EMA) Day 180 List of Outstanding Issues issued by the Committee for Medicinal Products for Human Use (CHMP) as part of the centralized review process of the Marketing Authorization (MAA) for vosaroxin as a treatment for relapsed/refractory acute myeloid leukemia (AML) in patients aged 60 years and older (Press release, Sunesis, MAR 22, 2017, View Source [SID1234518235]).

"Our team has provided detailed answers to the EMA in response to the Day 180 List of Outstanding Issues," said Daniel Swisher, President and Chief Executive Officer of Sunesis. "We are preparing to go before the Scientific Advisory Group’s Oncology Division (SAG-O) in April, which will assist the CHMP in its evaluation of our application. As we approach this final phase of the European approval process, anticipating a CHMP decision by mid-year, we continue to work in parallel to qualify the best pharma partner to work with us on a European market launch of vosaroxin in the second half of 2017."

About QINPREZO (vosaroxin)

QINPREZO (vosaroxin) is an anti-cancer quinolone derivative (AQD), a class of compounds that has not been used previously for the treatment of cancer. Preclinical data demonstrate that vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis. Both the U.S. Food and Drug Administration (FDA) and European Commission have granted orphan drug designation to vosaroxin for the treatment of AML. Additionally, vosaroxin has been granted fast track designation by the FDA for the potential treatment of relapsed/refractory AML in combination with cytarabine. Vosaroxin is an investigational drug that has not been approved for use in any jurisdiction.

Vosaroxin’s Marketing Authorization Application for relapsed refractory AML is currently under review by the European Medicines Agency, and a regulatory decision regarding approval is expected in 2017.

The trademark name QINPREZO is conditionally accepted by the FDA and the EMA as the proprietary name for the vosaroxin drug product candidate.