On June 23, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the US Food and Drug Administration (FDA) approved Rituxan Hycela (rituximab and hyaluronidase human) for subcutaneous (under the skin) injection, for the treatment of adults with the following blood cancers: previously untreated and relapsed or refractory follicular lymphoma, previously untreated diffuse large B-cell lymphoma (DLBCL), and previously untreated and previously treated chronic lymphocytic leukaemia (CLL) (Press release, Hoffmann-La Roche, JUN 22, 2017, View Source [SID1234519664]). This new treatment includes the same monoclonal antibody as intravenous Rituxan (rituximab) in combination with hyaluronidase human, an enzyme that helps to deliver rituximab under the skin.

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"With today’s approval of Rituxan Hycela, people with three of the most common blood cancers now have a new treatment option which provides efficacy comparable with intravenous Rituxan and can be delivered under the skin in minutes instead of hours through IV infusion," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "People who benefit from Rituxan may receive years of repeated treatments for their blood cancer, so an option that reduces the administration time can be important."

The FDA approval is based on results from clinical studies, which demonstrated that subcutaneous administration of
Rituxan Hycela resulted in non-inferior levels of rituximab in the blood (pharmacokinetics) and comparable clinical efficacy outcomes compared to intravenous Rituxan. One of the studies showed the majority (77%) of patients preferred Rituxan Hycela over intravenous Rituxan, with the most common reason being that administration required less time in the clinic. People can only receive Rituxan Hycela after at least one full dose of intravenous Rituxan.

With the exception of local skin (cutaneous) reactions, the incidence and profile of adverse reactions for Rituxan Hycela were comparable with those for intravenous Rituxan. The most common (≥20%) adverse reactions observed with Rituxan Hycela in people with follicular lymphoma were infections, low white blood cell count (neutropenia), nausea, constipation, cough and fatigue. The most common adverse reactions in people with DLBCL were infections, neutropenia, hair loss (alopecia), nausea and low red blood cell count (anemia). The most common adverse reactions in people with CLL were infections, neutropenia, nausea, low platelet count (thrombocytopenia), fever (pyrexia), vomiting and reddening of the skin (erythema) at the injection site.

Rituxan Hycela will be available to people in the United States within one to two weeks, and intravenous Rituxan will continue to be available.

About the Rituxan Hycela Clinical Development Program
The approval of Rituxan Hycela is based on results from clinical studies that together represented nearly 2,000 people. The studies were the following:
SABRINA (NCT01200758): Phase III combination with chemotherapy and maintenance study in previously untreated follicular lymphoma

SAWYER (NCT01292603): Phase Ib study in previously untreated chronic lymphocytic leukaemia (CLL)
MabEase (NCT01649856): Phase III study in previously untreated diffuse large B-cell lymphoma (DLBCL)
PrefMab (NCT01724021): Phase III patient preference study in previously untreated follicular lymphoma and DLBCL

About MabThera/Rituxan (rituximab)
MabThera/Rituxan is a therapeutic monoclonal antibody that binds to a particular protein – the CD20 antigen – on the surface of normal and malignant B-cells. It then recruits the body’s natural defenses to attack and kill the marked B-cells. Stem cells (B-cell progenitors) in bone marrow lack the CD20 antigen, allowing healthy B-cells to regenerate after treatment and return to normal levels within several months.

Rituxan first received FDA approval for the treatment of relapsed indolent non-Hodgkin Lymphoma (NHL) in 1997 and was the first targeted cancer medicine approved by the U.S. Food and Drug Administration (FDA). MabThera was approved in the EU in June 1998, and has since been used to treat more than 2.7 million people with specific blood cancers. For more than 15 years, the efficacy and safety of MabThera/Rituxan has been documented in more than 300 phase II/III clinical studies. MabThera/Rituxan has been approved for the treatment of several blood cancers, specifically, certain types of NHL and for chronic lymphocytic leukaemia (CLL). It continues to be studied in other types of blood cancers and disease areas where CD20-positive cells are believed to play a role.

MabThera is known as Rituxan in the United States, Japan and Canada. Genentech, a member of the Roche Group, and Biogen collaborate on Rituxan in the United States, and Roche markets MabThera in the rest of the world, except Japan, where MabThera is co-marketed by Chugai and Zenyaku Kogyo Co. Ltd.

About Rituxan Hycela
Rituxan Hycela is a co-formulation of the same monoclonal antibody as intravenous MabThera/Rituxan and Halozyme Therapeutics’ proprietary hyaluronidase human, an FDA-approved enzyme that facilitates the delivery of a large volume of medicine under the skin. Rituxan Hycela can be administered in five to seven minutes, compared to 1.5 to four hours for intravenous MabThera/Rituxan. It is known as the subcutaneous (SC) formulation of MabThera (rituximab) in the European Union.

About Follicular Lymphoma
Follicular lymphoma is the most common indolent (slow-growing) form of non-Hodgkin lymphoma (NHL), accounting for about one in five cases of NHL1. It is considered incurable and relapse is common. Every day, more than 50 people in Europe are diagnosed this type of NHL2. It is estimated that each year, more than 75,000 people are diagnosed with follicular lymphoma worldwide2.

About Diffuse Large B-Cell Lymphoma (DLBCL)
Diffuse large B-cell lymphoma (DLBCL) is the most common subtype of non-Hodgkin lymphoma (NHL), accounting for about one in three cases of NHL3. DLBCL is an aggressive (fast-growing) type of NHL, which is generally responsive to treatment in the frontline4. However, as many as 40% of patients will relapse, at which time salvage therapy options are limited and survival is short4. Approximately 123,000 people worldwide are estimated to be diagnosed with DLBCL each year5.

About Chronic Lymphocytic Leukaemia (CLL)
Chronic lymphocytic leukaemia (CLL) is the most common type of leukaemia in the Western world6. CLL mainly affects men and the median age at diagnosis is about 70 years7. Worldwide, the incidence of all leukaemias is estimated to be over 350,0006 and CLL is estimated to affect around one-third of all people newly diagnosed with leukaemia8.

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.

OSE-172 (Effi-DEM) is a monoclonal antibody: a new generation checkpoint inhibitor which blocks the generation of pro-tumor suppressor cells and restores their effector function.

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OSE-172 blocks SIRP-alpha (Signal Regulatory Protein Alpha), on strategic SIRP-alpha/CD47 pathway, a receptor strongly expressed by myeloid and macrophage suppressor cells. It restores effector functions of these suppressor cells, an activity which promotes the immunosurveillance (Hanna R.N. et al ; Science 2015). OSE-172 may also be combined with other immunotherapies, in particular with checkpoint inhibitors acting on T lymphocytes, e.g. checkpoint inhibitors targeting the PD-1/PD-L1 axis or products triggering a stimulation of the immune system.

This innovative product helps modifying Tumor Associated Macrophages (TAM) and Myeloid Derived Suppressor Cells (MDSC) associated with a poor prognosis, by blocking and transforming them into cells with a good prognosis (in blue in the above diagram).
Proof of concept obtained in in vivo models

Proof of concept has been obtained in models of aggressive cancers such as primary liver cancer, melanoma and breast cancer. These studies have confirmed a therapeutic effect of OSE-172 (Effi-DEM) as well as a potential long-lasting effect, in both monotherapy or combined with another checkpoint inhibitor, or with an immune system stimulator.

The therapeutic effect can be considered as long-lasting as reinsertion of a new tumor in animals treated by OSE-172 shown to be impossible : animals had developed an anti-tumor immunization. A treatment with OSE-172 as a monotherapy and combined with other immunotherapies induces a strong and long-lasting anti-tumor effect.

OSE-172 could be developed in all cancers involving TAM and MDSC cells, key cells in the progression of inflammatory cancers. Cytokines secreted by suppressor cells foster this mechanism (IL-10, IL-1β, TGFβ). This therapeutic strategy could be applied to cancers linked to a chronic inflammation such as primary liver cancer or colon cancer (Zamarron B.F. et 2011) (Mallmann MR et al. 2012).

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.