On August 14, 2019 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported the pricing of its previously announced registered underwritten public offering of 10,810,810 shares of its common stock at a price to the public of $37.00 per share (Press release, Deciphera Pharmaceuticals, AUG 14, 2019, View Source [SID1234538741]). The gross proceeds to Deciphera from the offering, before deducting the underwriting discounts and commissions and other estimated offering expenses, are expected to be $400.0 million. The offering is expected to close on or about August 19, 2019, subject to customary closing conditions. In addition, Deciphera has granted the underwriters a 30-day option to purchase up to 1,621,621 additional shares of its common stock.

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J.P. Morgan, Piper Jaffray and Jefferies acted as joint book-running managers for the offering. Guggenheim Securities acted as lead manager for the offering. SunTrust Robinson Humphrey acted as co-manager for the offering.

Deciphera intends to use the net proceeds of the offering to fund: clinical trials for ripretinib, including the expansion stage of its current Phase 1 clinical trial, its ongoing pivotal Phase 3 clinical trials, and additional clinical trials, as well as clinical research outsourcing and manufacturing of clinical trial material, and pre-commercialization manufacturing process development and validation; clinical trials for DCC-3014, including the expansion stage of its current Phase 1 clinical trial, as well as clinical research outsourcing and manufacturing of clinical trial material; clinical trials for rebastinib, including its current Phase 1b/2 clinical trial, as well as clinical research outsourcing and manufacturing of clinical trial material; Investigational New Drug-enabling studies and the potential development of DCC-3116; new and ongoing research activities for future drug candidates using its proprietary kinase switch control inhibitor platform; continued growth of its commercial and medical affairs capabilities to support its transition from a development-stage company toward a commercial-stage company; and working capital purposes, including general operating expenses.

A shelf registration statement relating to the shares of common stock offered in the public offering described above was filed with the Securities and Exchange Commission (SEC) and was declared effective by the SEC on October 12, 2018. The securities may be offered only by means of a written prospectus, including a prospectus supplement, forming a part of the effective registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and are available on the SEC’s website at www.sec.gov. A final prospectus supplement and accompanying prospectus will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from J.P. Morgan Securities LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (866) 803-9204, or by email at [email protected]; Piper Jaffray & Co., 800 Nicollet Mall, J12S03, Minneapolis, Minnesota, 55402, Attention: Prospectus Department, by telephone at (800) 747-3924 or by email at [email protected]; and Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at (877) 821-7388 or by email at [email protected].

This press release does not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On August 14, 2019 M2Gen, a health informatics solutions company focused on accelerating the discovery, development and delivery of precision medicine, reported the appointment of Helge Bastian, Ph.D., as president and chief executive officer (CEO) (Press release, M2Gen, AUG 14, 2019, View Source [SID1234538740]). Bastian succeeds M2Gen Executive Chair William S. Dalton, M.D., Ph.D., who has served as interim CEO since September 2018.

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"Dr. Bastian has a proven track record of building profitable and sustainable businesses in the life sciences and healthcare sectors including many years of experience in leading clinical research and biobanking organizations supporting precision medicine approaches. We are excited to have Dr. Bastian join M2Gen and lead our company to the next level as we deliver health information solutions that link patient-specific clinical and molecular data with the objective of developing and delivering personalized medicine for patients. With Dr. Bastian at the helm, M2Gen will continue to grow and advance the company’s mission to learn from each patient and proactively develop therapeutic options to meet patient needs," said Dalton.

Dr. Bastian is an experienced executive and scientific leader with more than 27 years of international experience while contributing to the growth of leading life science and healthcare companies in Germany, Switzerland and the USA. His expertise spans, molecular and synthetic biology including game-changing technologies such as genome editing, gene synthesis and re-programming, molecular and companion diagnostics, clinical research, drug development, pharmaceutical manufacturing and biobanking. As M2Gen’s new CEO, he will focus on expanding the company’s business portfolio and growing the Oncology Research Information Exchange Network (ORIEN), an alliance of biopharmaceutical companies and leading cancer centers, powered by M2Gen’s data and informatics platforms that work in collaboration to address the greatest challenges in oncology drug development. He will also join M2Gen’s Board of Directors.

"I am very excited about M2Gen’s revolutionizing data-driven approach to precision medicine in such an important area as oncology that affects so many people’s lives. I look forward to bringing my leadership experiences from the life science and healthcare industry to M2Gen and taking the company to the next level of its exciting growth strategy," said Bastian. "Understanding a patient’s genetic predisposition and the real-time molecular patterns in a patient’s tumor in combination with his / her clinical data is paramount to unravel the molecular mechanisms responsible for cancer development, and for developing new drugs and therapies as well as for accurate patient enrolling to clinical trials and even for developing strategies for cancer prevention. M2Gen’s initiative to build this unique and growing healthcare ecosystem of cancer centers to support clinical and molecular data sharing from a large number of cancer patients via M2Gen’s platforms is designed to fundamentally change the way cancer is being studied and treated, and will accelerate the discovery and development of precision treatment options for patients."

Dr. Bastian joins M2Gen from Thermo Fisher Scientific, Inc., where he led the Sample Preparation and Synthetic Biology businesses as Vice President and General Manager since 2014. Before Thermo Fisher Scientific, Dr. Bastian held a number of senior leadership positions and served as a member of several executive leadership teams, including Managing Director and Chief Commercial Officer at Indivumed, an integrated oncology company; Vice President Global Marketing, Business Development and Strategy at Sigma-Aldrich (now Merck KGaA); Executive Vice President Life Science Services for SGS, the world’s leading inspection, verification, testing and certification company. Prior to SGS, he held various management positions during his 11-plus years at Qiagen including Vice President Molecular Diagnostics, Vice President Global Strategic Marketing, and Vice President PreAnalytiX, a QIAGEN / Becton Dickinson joint venture. Dr. Bastian earned his diploma in biology / biochemistry from Georg-August University and holds a Ph.D. in molecular and cell biology performed at the Max-Planck Institute for Biophysical Chemistry. From 2014-2018, Dr. Bastian served as a board member to the International Gene Synthesis Consortium (IGSC). Dr. Bastian has recently been appointed to the board of directors at enzymatic gene synthesis pioneer Molecular Assemblies, Inc., San Diego, and is an alumnus from the Max-Planck-Network and the Columbia Business School.

On August 14, 2019 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that the U.S. Patent and Trademark Office has issued U.S. Patent No. 10,370,452 covering compositions and uses of effector T cells expressing a chimeric antigen receptor (CAR), where such T cells are derived from a pluripotent stem cell including an induced pluripotent stem cell (iPSC) (Press release, Fate Therapeutics, AUG 14, 2019, View Source [SID1234538739]). The foundational patent, which expires in 2034, is owned by Memorial Sloan Kettering Cancer Center (MSK) and is licensed exclusively to Fate Therapeutics for all human therapeutic uses.

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"The breadth of this newly-issued patent, which is not restricted by the signaling domain of the CAR construct nor by the antigen to which the CAR targets and binds, covers off-the-shelf, iPSC-derived CAR T cell therapy for the treatment of cancer, infectious disease and immune disorders," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "This patent is a testament to the prescient work of MSK’s Dr. Michel Sadelain in combining CAR and iPSC technologies in an effort to overcome the significant challenges that limit the widespread adoption of patient- and donor-sourced engineered T-cell immunotherapy."

Fate Therapeutics and MSK are conducting research and development of T-cell product candidates derived from iPSCs. These activities include the conduct of IND-enabling studies for FT819, an off-the-shelf CAR T-cell product candidate derived from a clonal master engineered iPSC line with complete elimination of T-cell receptor (TCR) expression and a novel 1XX CAR targeting CD19 inserted into the T-cell receptor alpha constant (TRAC) locus. Fate Therapeutics recently expanded its laboratory operations to New York City to support its research and development of off-the-shelf, iPSC-derived T cells.

Fate Therapeutics has built a dominant intellectual property position broadly covering iPSC technology and iPSC-derived cell products. Its proprietary portfolio includes compositions and methods for generating iPSCs, including engineering their biological properties using CRISPR and other nucleases, and for producing genetically edited cells of the hematopoietic lineage, including NK cells and T cells, from iPSCs. Fate Therapeutics’ iPSC product platform is supported by over 250 issued patents and 150 pending patent applications.

About FT819
FT819 is an off-the-shelf, T-cell receptor (TCR)-less CD19 chimeric antigen receptor (CAR) T-cell product candidate manufactured from a clonal master iPSC line that is undergoing IND-enabling activities in collaboration with Memorial Sloan Kettering Cancer Center (MSK). FT819 is manufactured from a clonal master engineered iPSC line with complete elimination of TCR expression and a novel 1XX CD19-specific CAR inserted into the T-cell receptor alpha constant (TRAC) locus. These synthetic features are intended to mitigate the risk of graft-versus-host disease, a severe life-threatening condition that occurs when donor T cells attack a patient’s healthy tissue, and to regulate CAR expression to enhance the therapeutic potency and durability of CAR T cells. Scientists from the Company and MSK have presented preclinical data demonstrating that FT819 lacks TCR expression, is highly cytotoxic and specific to CD19 antigen and controls tumor progression comparable to peripheral blood CAR19 T cells in a mouse model of acute lymphoblastic leukemia.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is fraught with batch-to-batch and cell-to-cell variability that can affect safety and efficacy.

On August 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 technologies, reported its financial results for the second quarter ended June 30, 2019 (Press release, Neuralstem, AUG 14, 2019, View Source [SID1234538738]).

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RECENT HIGHLIGHTS & ACCOMPLISHMENTS:

Appointed Mary Ann Gray, Ph.D., to Board of Directors
Executed a 1:20 reverse share split
Completed $7.5 million underwritten public offering
Regained compliance with the NASDAQ listing requirements
"In the second quarter we took a number of actions to strengthen the company and establish a foundation to enable our future growth. The addition of Dr. Mazzo and Dr. Gray to our board of directors adds significant industry and scientific experience to our board. Additionally, the completion of our public offering resulted in a funding level that we believe will support our strategic initiatives. Lastly, we are very pleased that NASDAQ granted our request for continued listing. All of these actions, in addition to the strategic portfolio evaluation we began in the first quarter of this year positions the company to move into 2020 as a stronger company with enhanced opportunities," said Ken Carter, Chief Executive Officer of Neuralstem.

Financial Results for the Quarter Ended June 30, 2019

Research and Development Expenses: R&D expenses for the quarter ended June 30, 2019 were flat at $1.0 million versus the comparable period of 2018. Spending was dedicated to completing various clinical activities as well as pursuing the identification of strategic program opportunities.

R&D expenses for the six months ended June 30, 2019 increased by $0.3 million to $2.5 million, or 13% increase over the comparable period of 2018. This increase was primarily attributable to a $0.5 million write-off of an employee payable in connection with the termination of such employee and entering into a corresponding separation agreement.

General and Administrative Expenses: G&A expenses for the quarter ended June 30, 2019 decreased by $0.3 million to $1.0 million, or 13% decrease over the comparable period of 2018. This decrease was driven by general expense reduction efforts across multiple areas.

G&A expenses for the six months ended June 30, 2019 decreased by $0.5 million to $1.9 million, or 12% decrease over the comparable period of 2018. This decrease was driven by general expense reduction efforts across multiple areas.

Net Loss: Net loss for the quarter ended June 30, 2019 was $1.4 million, or $1.45 per share, compared to a loss of $0.6 million, or $0.83 per share, for the comparable period of 2018. Weighted average shares outstanding were 1.0 million shares at June 30, 2019 compared to .8 million shares at June 30, 2018.

Net loss for the six months ended June 30, 2019 was $4.6 million, or $4.78 per share, compared to a loss of $2.8 million, or $3.67 per share, for the comparable period of 2018. Weighted average shares outstanding were 1.0 million shares at June 30, 2019 compared to .8 million shares at June 30, 2018.

All per share numbers have been retroactively adjusted for our 1-for-20 reverse stock split.

The net loss in the first three- and six-month periods of 2018 were positively impacted by reductions in the derivative liabilities related to outstanding warrants reflected on the June 30, 2018 statement of operations of $1.4 and $1.6 million, respectively. The corresponding 2019 three- and six-month periods also reflect reduced derivative liabilities with the impact reflected on the statement of operations in the amounts of $0.4 and $0.1 million, respectively.

Cash Position and Liquidity: At June 30, 2019, cash, cash equivalents and short-term investments were $2.3 million as compared to $5.8 million at December 31, 2018. After including the capital raise completed in July 2019, the cash and cash equivalents were $8.8 million. The Company anticipates its existing cash, cash equivalents to fund its operations, based on its current operating plans, into the third quarter of 2020.

On August 14, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company discovering and developing novel products to treat cancer, with a focus on Myelodysplastic Syndromes (MDS), provided a corporate update and reported financial results for the second quarter ended June 30, 2019 (Press release, Onconova, AUG 14, 2019, View Source [SID1234538737]).

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Steven M. Fruchtman, M.D., President and Chief Executive Officer, stated, "Enrollment in our global Phase 3 INSPIRE Trial with IV rigosertib in second-line, higher-risk MDS patients is progressing. Our goal is to complete enrollment by the end of 2019 and we anticipate reporting top-line data in the first half of 2020 following full enrollment and 288 death events. If the INSPIRE Trial is successful, we believe rigosertib could be the first new treatment for higher-risk MDS in more than 15 years, and based on its unique mechanism of action, has the potential to provide clinical benefit in other Ras mutated cancers."

Dr. Fruchtman continued, "In addition to advancing the INSPIRE Trial, our Special Protocol Assessment (SPA) request to the FDA for a Phase 3 combination trial of oral rigosertib plus azacitidine in first-line higher-risk MDS patients is being pursued. We also have plans to target cancers driven by mutated Ras genes. Ras-mutated cancers represent about a third of all human cancers, and a Phase 1 study of rigosertib in combination with a PD-1 inhibitor for patients with progressive K-Ras mutated non-small cell lung cancer is expected to commence in 2019 as an investigator-initiated study. We are also working toward filing an IND for a Phase 1 trial of ON 123300, our investigational, first-in-class, dual inhibitor of CDK4/6 + ARK5, which we believe has the potential to treat various cancers including refractory metastatic breast cancer."

Second Quarter 2019 and Recent Highlights

On March 25, the Company announced that it had passed the 75 percent enrollment mark in the Phase 3 INSPIRE Trial. Onconova remains focused on our primary goal of completing enrollment by the end of 2019 and expects to report top-line survival data following full enrollment and 288 death events.

• More than 140 trial sites in 24 countries across four continents are open, including 21 sites in Japan. The Company opened new clinical trial sites in already participating countries. Additional geographies, including Brazil, are being opened during the coming months to add approximately 25 more sites. This strategy is designed to support the goal of achieving full accrual to the INSPIRE Trial in 2019. The Company anticipates reporting top-line data in the first half of 2020 following full enrollment and 288 death events.
The Company entered into a license agreement with HanX Biopharmaceuticals (HanX) for the development and commercialization of rigosertib in Greater China. The agreement provides for $4 million of upfront payments to Onconova, including a $2 million cash upfront fee and an investment totaling $2 million in shares purchased at a premium to market. In addition, HanX agreed to place $2 million in escrow in local currency for rigosertib clinical development expenses in Greater China. HanX will make additional regulatory, developmental, and sales-based milestone payments to Onconova of up to $45.5 million and will pay Onconova tiered royalties up to double digits on net sales in Greater China. If approval is received, Onconova will supply rigosertib to HanX for development and commercialization. HanX also will support Onconova’s other clinical trial initiatives in Greater China.
Onconova and Mission Bio have entered into a collaboration to utilize the Mission Bio Tapestri Platform for targeted single-cell DNA analysis to study rigosertib as part of planned clinical trials. Single-cell genomics may identify mutations with far better resolution than that of traditional sequencing methods, allowing a view into each patient’s disease at a level never before achieved. Tapestri will be utilized to identify mutations, including those of the Ras pathway, to monitor a patient’s response in clinical trials, supporting the advancement of rigosertib. By adding the Tapestri Platform to its research and development program, Onconova is including the opportunity to study single cell clones in MDS and determine the sequence of genetic events and the influence of rigosertib on these events along with clinical outcomes.

In December 2018, Onconova applied to the FDA for a Special Protocol Assessment (SPA) for a Phase 3 trial of oral rigosertib in combination with azacitidine for treatment of first-line higher-risk MDS patients. The Company expects completion of the FDA’s SPA decision before the end of 2019.

Results from the Phase 2 trial were reported in December 2018 in an oral presentation at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) meeting and updated at the 2019 European Hematology Association (EHA) (Free EHA Whitepaper) Meeting.
Additional Progress for Rigosertib and Pipeline Products

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 IND filing in the U.S.

The collaboration with the National Cancer Institute as well as one with a Center of Excellence in Juvenile Myelomonocytic Leukemia, or JMML, for preclinical studies of rigosertib for treatment of pediatric cancer associated RASopathies are ongoing.

Onconova continues to participate in important medical and investment conferences. During the second quarter, presentations related to rigosertib development and clinical trials were made at:
• 15th Annual International Symposium on MDS/Copenhagen
• Acute Leukemia Forum/Shanghai
• BIO International Forum/Philadelphia
• European Hematology Association (EHA) (Free EHA Whitepaper)/Amsterdam
Upcoming 2019 conferences include:

RAS-Targeted Drug Discovery Summit (Boston, September 17-19)
Brazilian Association of Hematology, Hemotherapy and Cellular Therapy Congress (Rio de Janeiro, November 6-9)
The American Society of Hematology (ASH) (Free ASH Whitepaper) Conference in (Orlando, December 7-10)
Second Quarter 2019 Financial Results

Cash and cash equivalents as of June 30, 2019, totaled $5.9 million compared to $17.0 million at December 31, 2018. The Company believes that cash and cash equivalents at June 30, 2019, along with additional funds to be received from the HanX license in the third quarter, will be sufficient to fund ongoing trials and operations late into the fourth quarter of 2019. The Company was notified by Nasdaq on July 26 that Nasdaq has accepted the Company’s plan to regain compliance with the stockholders’ equity listing requirement by November 18, 2019.

Net loss was $3.6 million for the quarter ended June 30, 2019, compared to $4.3 million for the second quarter ended June 30, 2018. Research and development expenses were $3.9 million for the quarter ended June 30, 2019, and $4.1 million for the comparable period in 2018. General and administrative expenses were $1.8 million for the quarter ended June 30, 2019, and $2.1 million for the comparable period in 2018.

Conference Call and Webcast Information

The Company will host a conference call today, August 14, 2019, at 9 a.m. Eastern Time, to provide a corporate update and discuss second 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 90141175. 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.