On June 21, 2017 ImmunoCellular Therapeutics, Ltd. ("ImmunoCellular") (NYSE MKT: IMUC) reported an update on the strategic review of its financing and development strategies for ICT-107, its patient-specific, dendritic cell-based immunotherapy for patients with newly diagnosed glioblastoma (Press release, ImmunoCellular Therapeutics, JUN 21, 2017, View Source [SID1234519640]).

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ImmunoCellular has determined that the Company is unable at this time to secure sufficient additional financial resources to complete the phase 3 registration trial of ICT-107. As a result, the Company intends to suspend further patient randomization in the ICT-107 trial while it continues to seek a collaborative arrangement or acquisition of its ICT-107 program. The Company plans to actively work with current collaborators to ensure that patients already randomized and receiving treatment in the phase 3 trial can be appropriately supported and followed. The suspension of the phase 3 registration trial of ICT-107 is expected to reduce the amount of cash used in the Company’s operations.

While maintaining the focus on its Stem-to-T-Cell research programs, ImmunoCellular continues its evaluation of financing and strategic alternatives for its immuno-oncology research and development pipeline and technology platform, which may include a potential merger, consolidation, reorganization or other business combination, as well as the sale of the Company or the Company’s assets.

On June 21, 2017 FibroGen, Inc. (NASDAQ:FGEN), a science-based biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation status to pamrevlumab, the company’s first-in-class antibody, for the treatment of pancreatic cancer (Press release, FibroGen, JUN 21, 2017, View Source;p=RssLanding&cat=news&id=2282394 [SID1234519642]).

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"This is an important regulatory milestone for FibroGen, as pamrevlumab continues to show promise in the treatment of pancreatic cancer," said Tom Neff, Chief Executive Officer of FibroGen. "Phase 2 clinical studies of pamrevlumab have produced initial positive data on median and one-year survival for patients with advanced pancreatic cancer (88% metastatic). An ongoing study in locally advanced non-resectable pancreatic cancer has shown promise in converting pancreatic cancer from non-resectable to surgically viable. In this current open-label Phase 2 randomized trial, we are evaluating pamrevlumab in combination with chemotherapy standard-of-care versus chemotherapy alone. We look forward to sharing results by early next year."

Orphan Drug Designation qualifies the sponsor for various development incentives of the Orphan Drug Act, including tax credits for qualified clinical testing, to advance the evaluation and development of products that demonstrate promise for the diagnosis and treatment of rare diseases or conditions. Orphan Drug Designation can also convey up to seven years of marketing exclusivity if the compound receives regulatory approval from the FDA. FibroGen previously received Orphan Drug Designation for pamrevlumab for the treatment of idiopathic pulmonary fibrosis (IPF).

About Pamrevlumab
Pamrevlumab (formerly FG-3019) is an investigational therapeutic antibody developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in chronic fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. FibroGen is currently conducting clinical studies of pamrevlumab in idiopathic pulmonary fibrosis, pancreatic cancer, and Duchenne muscular dystrophy (DMD). In desmoplastic or fibrotic cancers, such as pancreatic cancer, CTGF in the extensive fibrous stroma associated with the tumor promotes abnormal proliferation of stromal cells and tumor cells. For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.

On June 21, 2017 Rubius Therapeutics, a biotechnology company pioneering the development of a new class of extraordinarily active and differentiated cell therapies reported that it has raised $120 million in a highly oversubscribed private financing (Press release, Rubius Therapeutics, JUN 21, 2017, View Source [SID1234520731]).

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Rubius has developed the technology to grow, genetically engineer and mature long-circulating red cell therapeutics which will provide transformational clinical benefits to a wide range of patients across multiple therapeutic areas. These allogeneic, off-the-shelf products offer the additional advantage of extended stability and storage to allow for rapid, universal access by the medical community. Proceeds will be used to accelerate the advancement of Rubius’ breakthrough Red-Cell Therapeutics (RCT) product portfolio, to further build out the team and to prepare to enter human clinical trials in 2018. This financing follows the successful achievement of several key milestones, including the clinical scale production of cultured red cells in bioreactors and the generation of preclinical proof-of-concept data for several lead programs.
"With this financing, Rubius is well positioned to focus on the continued development of our platform to advance a broad range of therapeutic candidates that have the potential to make a significant difference in the lives of patients," said Torben Straight Nissen, Ph.D., President of Rubius Therapeutics.
"Rubius has achieved multiple scientific and manufacturing milestones in the first six months of this year. Those achievements plus the trust that Flagship and our new investors have put in Rubius with this financing sets us up to turn our RCTs into important new treatment options," said David Epstein, Chairman of Rubius Therapeutics and Executive Partner of Flagship Pioneering.
The RCT platform allows Rubius to express enzymes, agonists, antagonists, binders and combinations in their natural conformation on or inside of red cells. Lead RCT programs include enzyme replacement therapies as well as therapies targeting solid tumors and hematological cancers. Further, Rubius has demonstrated that RCTs provide potential applications across additional therapeutic areas, such as autoimmune disease, infectious disease, metabolic disease and rare diseases, including hemophilia.
"We are excited to expand our support of Rubius in this next round of funding as the company continues to advance its technology and programs — following in the footsteps of Flagship’s family of successful multiproduct platform companies," said Noubar Afeyan, CEO of Flagship Pioneering and Co-Founder of Rubius. "The team at Rubius has made great strides over the past three years and we are pleased to support the company’s continued growth. This new financing, together with a leadership team with unparalleled expertise, positions Rubius to deliver on the promise of the RCT platform and bring transformative therapies to patients."
Rubius was conceived, launched and funded by Flagship VentureLabs, the innovation foundry of Flagship Pioneering. Joined by undisclosed large institutional investors, Flagship Pioneering significantly increased its investment in this financing.
About Red-Cell Therapeutics Red-Cell Therapeutics are genetically engineered, enucleated red cells that provide allogeneic, off-the-shelf therapies to patients across multiple therapeutic areas. RCT advantages over other therapies include immuno-privileged presentation of proteins within or on the red cell, high target avidity and affinity resulting in highly potent and selective therapies, and long circulation half-life. Rubius RCTs exhibit fundamentally unique biology and have been engineered to replace missing enzymes for patients living with a variety of rare diseases, to kill tumors, and upregulate or downregulate the immune system to treat both cancer and autoimmune disorders.
About Rubius Therapeutics Rubius Therapeutics is developing Red-Cell Therapeutics (RCTs) as a new class of medicines to address a wide array of indications, with leading applications in cancer, rare and autoimmune disease, as well as additional potential in hemophilia, infectious and metabolic diseases. The company was founded and launched in 2014 by Flagship VentureLabs, the innovation foundry of Flagship Pioneering. Rubius has successfully engineered and manufactured red cells that express therapeutic proteins for use in the treatment of serious diseases. The company is now demonstrating that these newly equipped high performing, off-the-shelf Red-Cell Therapeutics have pre-clinical activity across a spectrum of medical applications. Rubius has generated more than 200 prototypes to date.

On June 20, 2017 Merck, a leading science and technology company, reported its corporate venture arm Merck Ventures created iOnctura SA, Geneva, Switzerland. This immuno-oncology spin-out company was formed around two assets from the Healthcare R&D portfolio of Merck and three assets from Cancer Research Technology (CRT) (Press release, Cancer Research Technology, 20 20, 2017, View Source [SID1234523165]). CRT is the commercial arm of Cancer Research UK, London, UK. Merck Ventures will manage the investment and will be represented on iOnctura’s board of directors.

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"Our goal is to modulate key culprits of immunosuppression in the tumor microenvironment to maximize the therapeutic potential of checkpoint inhibitors for patients," said Catherine Pickering, CEO and co-founder of iOnctura. "Through our key alliances with CRT and Merck, we are optimally positioned to explore novel combination therapies and advance them quickly to cancer patients."

iOnctura aims to develop a pipeline of selected assets that target and modulate mechanisms that drive immunosuppression in the tumor microenvironment (TME). Such immunosuppression has been shown to be one of the main causes behind a significant number of patients not responding to first generation checkpoint inhibitors. iOnctura, through its alliances with Merck and CRT, has already built a pipeline of promising programs and entered a research collaboration with CRT Discovery Laboratories. In exchange for the exclusive global option to license three immuno-oncology assets from CRT, iOnctura will provide CRT with an initial equity holding in the company and will make further payments for the achievement of late development and approval milestones as well as royalties on net sales. iOnctura has also secured access to future supply of avelumab, being co-developed and co-commercialized by Merck and Pfizer, which will enable acceleration into initial clinical proof of concept studies.

Stuart Farrow, CRT’s director of biology, said: "We’re delighted that these three potential new cancer treatments, partly developed by our drug discovery laboratories alongside leading Cancer Research UK-funded scientists, have been prioritized for further development through the formation of this new company. The ongoing support by our drug discovery laboratories will hopefully help build a strong development pipeline for iOnctura."

Merck Ventures, the strategic venture investment arm of Merck, is providing the initial seed funding to iOnctura. Hakan Goker, Senior Investment Director at Merck Ventures, and Keno Gutierrez will represent Merck Ventures on iOnctura’s board of directors.

On July 17, 2017 Takeda Pharmaceutical Company Limited (TSE: 4502) and Schrödinger Inc., a privately-held company dedicated to revolutionizing drug discovery through advanced computational methods, reported the formation of a multi-target research collaboration directed to diseases that align with Takeda’s core therapeutic areas of interest (Press release, Takeda, JUN 20, 2017, View Source [SID1234539393]). The unique collaboration will combine Schrödinger’s in silico platform-driven drug discovery capabilities with Takeda’s deep therapeutic area knowledge and expertise in structural biology. With a focus on simplicity, speed and agility, Schrödinger will lead the multi-target discovery effort with Takeda providing protein crystal structures to aid Schrödinger in using its computational platform to guide the design of new chemical entities. Schrödinger will be responsible for its discovery costs.

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"Schrödinger’s agility, drug discovery track record and revolutionary computational chemistry platform are key strengths that attracted Takeda to Schrödinger as a strategic partner," said Stephen Hitchcock PhD, Head of Research for Takeda. "Takeda’s business model is focused on creating mutually beneficial partnerships that capitalize on the complementary capabilities of both parties in order to accelerate the discovery of new therapies for patients. In this case, Schrödinger is taking the lead role in discovery, with Takeda playing a supporting role, leveraging its in-house structural biology team and therapeutic area expertise."

Under the terms of the collaboration, Takeda will have the option to exclusively license the programs from Schrödinger later in discovery at established economic terms, including pre-clinical, clinical, and commercial milestones of up to $170M per program, as well as royalties on future sales. Additional financial details are not being disclosed.

"We are very much looking forward to partnering with Takeda and marshalling its biology and development expertise with our world class computational chemistry and drug discovery capabilities to advance these programs," said Ramy Farid, Ph.D., Schrödinger’s president and chief executive officer. "This alliance allows us the opportunity to fully exploit the power of our platform to accelerate the delivery of novel therapeutics to patients."