On May 14, 2019 Precision BioSciences (Nasdaq: DTIL), a genome editing company dedicated to improving life through the application of its proprietary ARCUS genome editing platform, reported that Christopher R. Heery, M.D. has joined Precision BioSciences as its Chief Medical Officer (Press release, Precision Biosciences, MAY 14, 2019, View Source [SID1234536273]).

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"We are thrilled to welcome Chris Heery as our Chief Medical Officer. Chris has exceptional experience in the rapidly evolving field of cell therapies and has led many first-in-human clinical trials during his time at the National Cancer Institute (NCI). We believe his skill set and experience are a perfect fit to guide our off-the-shelf immunotherapy and in vivo gene correction programs through human clinical trials," said Matt Kane, Chief Executive Officer and co-founder of Precision BioSciences.

Chief Development Officer, David Thomson, commented, "Chris brings a highly unique and sought-after skill set to Precision BioSciences. He has been a translational clinical trialist at one of the world’s preeminent research institutions, NCI, where his first-in-human immunotherapy clinical trials included a Phase 1 study of an anti-PD-L1 treatment (avelumab) that is now approved for use in the United States. Chris’s most recent experience as Chief Medical Officer at Bavarian Nordic has given him a strong understanding of regulatory requirements, clinical trial oversight, and leadership that will allow him to play a key role in helping Precision BioSciences build a successful clinical organization. We look forward to advancing our clinical pipeline under Chris’s leadership."

Prior to joining Precision BioSciences, Dr. Heery most recently served as Chief Medical Officer of Bavarian Nordic, where he oversaw clinical development programs for its immune-oncology and infectious diseases portfolios. Prior to this, he was Head of the Clinical Trials Group of the Laboratory of Tumor Immunology and Biology at NCI. He joined the NCI Medical Oncology Branch as a Medical Oncology Fellow in 2009 and also served as an Adjunct Appointment in the Genitourinary Malignancies Branch. He was part of the larger effort of the Laboratory of Tumor Immunology and Biology to create new immunotherapies for the treatment of cancer. Dr. Heery is board certified in Medical Oncology and Internal Medicine and received his M.D. from East Carolina University Brody School of Medicine. He completed his internal medicine residency at the University of Illinois at Chicago. Dr. Heery completed his undergraduate studies at Duke University, where he received a BA in English Literature.

About Precision BioSciences

On May 14, 2019 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory, cancer and other diseases, reported that Allan Reine, Senior Vice President and Chief Financial Officer of Pieris Pharmaceuticals, Inc. will present at the 2019 RBC Capital Markets Global Healthcare Conference in New York, New York on Tuesday, May 21, 2019 at 11:30 AM (EDT) (Press release, Pieris Pharmaceuticals, MAY 14, 2019, View Source [SID1234536272]). A webcast of the company’s presentation will be available at this link.

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On May 14, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with an initial focus on Myelodysplastic Syndromes (MDS), reported financial results for the first quarter of 2019 ended March 31, 2019 (Press release, Onconova, MAY 14, 2019, View Source [SID1234536271]).

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"As we progress through 2019, we are advancing the clinical development of our pipeline and executing on our collaboration strategy, as highlighted by the recently announced license agreement for investigational rigosertib in Greater China with HanX Biopharmaceuticals (HanX)," said Steven M. Fruchtman, M.D., President and Chief Executive Officer. "2019 is an important year for Onconova and we are working diligently to execute on our objectives. If successful, we believe rigosertib could be the first new treatment for higher-risk MDS (HR-MDS) in more than 15 years."

Dr. Fruchtman added, "Enrollment in our Phase 3 intravenous (IV) rigosertib trial in second-line HR-MDS patients is continuing and is our top priority. We exceeded 75 percent enrollment of the study during the first quarter and are focused on completing enrollment in the second half of 2019 and reporting top-line data following full enrollment and 288 death events. We believe the addition of sites in Brazil and China later this year could contribute significantly to achieving our goal of completing enrollment by year end. We also look forward in the future to initiating a Phase 3 trial with oral rigosertib in combination with azacitidine in first-line HR-MDS patients and to filing an IND in the U.S. for ON 123300, a first-in-class CDK4/6 + ARK5 inhibitor for the treatment of a variety of advanced tumors."

First Quarter 2019 and Recent Highlights

Entered into rigosertib license agreement with HanX for the development and commercialization of rigosertib in Greater China, which, together with an equity investment at a premium, brings $4 million in cash to the Company. This new HanX license expands the existing collaboration between the two companies. Onconova and HanX previously signed a separate agreement in December 2017 for the pipeline compound ON 123300, Onconova’s novel CDK 4/6 inhibitor, which is expected to enter a Phase 1 clinical trial in the U.S. during the second half of 2019.
Achieved over 75 percent enrollment in the INSPIRE study during the first quarter, and remain focused on completing enrollment in the second half of 2019 and reporting top-line data following full enrollment and 288 death events.
More than 140 trial sites in 23 countries across four continents are open, including 21 sites in Japan. Opened 19 new clinical trial sites in 8 already participating countries to support completion of enrollment of 360 patients in the Phase 3 INSPIRE study. Additional geographies are being opened during the coming months to add approximately 25 more sites.
Attended MDS Symposium in Copenhagen May 8-11, 2019, for which five posters related to rigosertib were accepted for presentation.
Oral Rigosertib in Combination with Azacitidine for First-Line HR MDS Trial Progress and Near-Term Milestones

In December 2018, Onconova submitted an application for a SPA to the FDA for a Phase 3 Trial of oral rigosertib in combination with azacitidine for treatment of first-line higher-risk MDS adult patients. The Company is currently in discussions with the FDA regarding the SPA. Upon agreement regarding the SPA, Onconova hopes to initiate the Phase 3 study with the support of a partnership.
Business Development Progress for Rigosertib and Pipeline Products

Onconova entered into a new license agreement with HanX to develop and commercialize rigosertib in Greater China. HanX is a China-based pharmaceutical company focused on the development, registration, and commercialization of therapeutics for China. Under the terms of the agreement, Onconova has granted to HanX an exclusive license to develop and commercialize rigosertib in Greater China. In exchange for these rights, HanX will pay a $2 million up-front payment and make an additional $2 million equity investment in Onconova stock at a premium to market. In addition, HanX will initially dedicate $2 million in local currency to fund rigosertib development in China over the next two years and will be responsible for future development costs of the product in China pursuant to a joint development plan. HanX will make additional regulatory, developmental, and sales-based milestone payments to Onconova of up to $45.5 million and pay Onconova tiered royalties up to double digits on net sales in Greater China. If approval is received, Onconova will supply the finished product to HanX for development and commercialization. HanX also will support Onconova’s other clinical trial initiatives in Greater China.
ON 123300, an investigational first-in-class dual inhibitor of CDK4/6 + ARK5 with the potential to treat a variety of cancers, continues to make progress toward clinical development in the U.S. and China in partnership with HanX. HanX has conducted toxicology studies to support an Onconova IND filing in the U.S., anticipated in the second quarter of 2019.
Collaboration with the National Cancer Institute is ongoing for preclinical studies of rigosertib for treatment of pediatric cancer associated RASopathies.
Scientific presentations related to rigosertib development and clinical trials were made at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, the MDS Symposium in Copenhagen, and the Acute Leukemia Forum in Newport, CA; upcoming presentations will be made at the Acute Leukemia Forum in China, and the European Hematology Association (EHA) (Free EHA Whitepaper) Congress. Onconova will also attend the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Annual Meeting in June.
First Quarter 2019 Financial Results

Cash and cash equivalents as of March 31, 2019, totaled $10.4 million compared to $17.0 million at December 31, 2018. The Company expects that cash and cash equivalents at March 31, 2019 will be sufficient to fund ongoing trials and operations into the fourth quarter of 2019. After receiving the upfront proceeds from the HanX rigosertib transaction, the Company expects that its cash and cash equivalents will be sufficient to fund ongoing trials and operations into the first quarter of 2020.

Net loss was $7.6 million for the quarter ended March 31, 2019, compared to $5.1 million for the first quarter ended March 31, 2018. Research and development expenses were $4.1 million for the quarter ended March 31, 2019, and $4.6 million for the comparable period in 2018. General and administrative expenses were $3.2 million for the quarter ended March 31, 2019, and $1.9 million for the comparable period in 2018.

Conference Call and Webcast Information

The Company will host a conference call today, May 14, at 9 a.m. Eastern Time, to provide a corporate update and discuss first quarter 2019 financial results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the U.S., or internationally (210) 229-8823 and using conference ID: 4275108. The call will also be webcast live. Please click here to access the webcast. A replay will be available following the live webcast.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, and to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.

About Onconova Therapeutics, Inc.

Onconova Therapeutics, Inc. is a Phase 3-stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS). Using a proprietary chemistry platform, Onconova has created a pipeline of targeted agents designed to work against specific cellular pathways that are im

On May 14, 2019 Neuralstem, Inc. (Nasdaq:CUR), a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell and small molecule compound technologies, reported its financial results for the first quarter ended March 31, 2019 (Press release, Neuralstem, MAY 14, 2019, View Source [SID1234536270]).

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"We are pleased with our progress in the first quarter of 2019 as we continue to review our current portfolio and options for business expansion." said Ken Carter, Executive Chairman of Neuralstem. "We have completed an extensive evaluation of our NSI-566 and NSI-189 programs and are developing appropriate long-term plans for these programs. Additionally, in collaboration with our strategic advisers we have identified promising preclinical and clinical assets that may be complimentary to our company’s mission as well as our recently announced pipeline expansion initiative."

Corporate Highlights

Effective January 1, 2019 Dr. Kenneth Carter was appointed as Executive Chairman by the Board of Directors. Dr. Carter succeeds Mr. Scully, former interim Neuralstem president and chief executive officer. Dr. Carter brings to Neuralstem extensive experience in the pharmaceutical and broader healthcare industry, including leadership roles in NexImmune, Noble Life Sciences and Avalon Pharmaceuticals.

On May 6, 2019 the Company announced that David. J. Mazzo will be joining the Board of Directors effective June 12, 2019. Dr. Mazzo brings 30 years of experience in the pharmaceutical industry to the company, including senior positions at Regado Biosciences, Aeterna Zentaris, and Chugi Pharma. Dr. Mazzo is currently Chief Executive Officer of Caladrius Biosciences, a clinical-stage biopharmaceutical company in the cardiovascular and autoimmune space.

Financial Results for the Quarter Ended March 31, 2019

Research and Development Expenses: R&D expenses for the quarter ended March 31, 2019 increased by $.3 million to $1.5 million, or 30% increase over the comparable period of 2018. This increase was primarily attributable to a severance payment in connection with the termination of an employee and entering into a corresponding separation agreement. The balance of the increase was driven by expenses to support the strategic evaluation and pipeline expansion initiative.

General and Administrative Expenses: G&A expenses for the quarter ended March 31, 2019 decreased by $.2 million to $.9 million , or 20% decrease over the comparable period of 2018. This decrease was driven by general expense reduction efforts across multiple areas.

Other Expense: Other expense for the quarter ended March 31, 2019 increased by $.3 million to $.3 million over the comparable period of 2018. This increase was primarily attributable to a write-off of an employee payable in connection with the termination of an employee and entering into a corresponding separation agreement.

Net Loss: Net loss for the quarter ended March 31, 2019 was $3.1 million, or $0.17 per share , compared to a loss of $2.1 million, or $0.14 per share, for the comparable period of 2018. Weighted average shares outstanding were 18.2 million shares at March 31, 2019 compared to 15.1 million shares at March 31, 2018.

Cash Position and Liquidity: At March 31, 2019, cash, cash equivalents and short-term investments was $4.0 million as compared to $5.8 million at December 31, 2018. The Company expects its existing cash, cash equivalents and short-term investments to fund its operations, based on its current operating plans, into the third quarter of 2019.

On May 14, 2019 NanOlogy LLC, a clinical-stage oncology company, reported that its abstract showing encouraging therapeutic effects from a preclinical pharmacology study on inhaled NanoPac (submicron particle paclitaxel) for treatment of lung cancer has been accepted for presentation at the 2019 American Thoracic Society International Conference (Press release, NanOlogy, MAY 14, 2019, View Source [SID1234536269]).

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The abstract, entitled Enhanced Tumor Regression and Immune Cell Infiltration by Inhaled Submicron Particle Paclitaxel in an Orthotopic Athymic Nude Rat Model of Non-Small Cell Lung Cancer (NSCLC), will be presented at the conference on May 20th, 11:15 AM to 1:00 PM, during the Oncogenic Mutations, Metastases, and Novel Therapeutics poster session at the Kay Bailey Hutchison Convention Center in Dallas.

Previously, NanOlogy conducted a pharmacokinetic study examining retention of NanoPac in healthy rat lung tissue following a single inhalation via nose-only exposure chamber. Data showed measurable amounts of drug in lung tissue 14-days post exposure, with treated lungs microscopically indistinguishable from normal lung tissue. A preclinical proof-of-concept study was then conducted to examine the therapeutic effect of inhaled NanoPac in an orthotopic athymic nude rat model of NSCLC. An abstract of that study was presented at the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting showing inhaled NanoPac achieved a significant decrease in primitive tumor cell population as well as significant tumor reduction.

The ATS abstract presents follow-on immunohistochemical analysis of lung tissue from the proof-of concept study, which showed NanoPac-treated animals had greater incidence and degree of tumor regression, immune cell infiltration, and tertiary lymphoid structures compared to untreated controls and intravenously administered nab-paclitaxel.

Describing the antitumoral immune response, Gere diZerega, MD, VP of Medical Affairs, observed, "Immunohistochemical analysis showed the immune cell infiltrates appeared to facilitate destruction of tumor and its conversion into small fibrin deposits."

NanOlogy is finalizing preclinical work of inhaled NanoPac in preparation for an investigational new drug (IND) submission to the US FDA in mid-2019 to allow for clinical trials in NSCLC.

During the conference, NanOlogy has also been selected to participate in the International Society for Aerosols in Medicine/ATS Pre-Conference session on "Current Practice and Future Developments in Aerosol Medicine." The session will be held on Saturday, May 18, from 1 PM to 4 PM at the Omni Dallas Hotel Trinity Ballroom 5-7 (Level 3).

Lung cancer is by far the leading cause of cancer death in the United States according to the American Cancer Society with more than 143,000 deaths projected this year. More people die of lung cancer annually than breast, prostate, and colon cancers combined.

In addition to lung cancer, NanOlogy clinical programs are advancing in genitourinary gastrointestinal, peritoneal, lung, and dermal cancers.

The NanOlogy submicron particle technology platform is based on a proprietary production process that reduces the size of taxane API crystals by up to 400 times into stable submicron particles of pure drug with exponentially increased surface area and unique geometry. The characteristics of the particles have recently been granted a composition of matter patent (US 9,814,685) valid in the US until 2036.