On June 22, 2017 Repare Therapeutics Inc. reported a US$68 million Series A financing to advance its platform and pipeline of novel medicines that target genetically defined weaknesses of cancers. Founding investor Versant Ventures co-led the round with MPM Capital (Press release, Repare Therapeutics, JUN 22, 2017, View Source [SID1234520721]). They were also joined by other syndicate investors including Fonds de solidarité FTQ, Celgene Switzerland LLC, an affiliate of Celgene Corporation, and BDC Capital’s Healthcare Venture Fund.

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With this financing, Repare emerges from Versant’s Discovery Engines after an 18-month stealth period during which the company advanced its leading CRISPR-enabled synthetic lethality drug discovery platform, identified several promising oncology targets and moved multiple programs into preclinical development. Founders Daniel Durocher, Ph.D., Agnel Sfeir, Ph.D., and Frank Sicheri, Ph.D., and their respective institutions, played instrumental roles in the company’s formation and growth in partnership with Versant.

"Versant’s commitment to and confidence in Repare’s distinct science has enabled the company to build the team, operations and initial programs away from the spotlight," said Repare CEO Lloyd M. Segal. "With the added leadership of MPM and this syndicate, we are financed to achieve our goal of testing our multiple new, precision oncology therapeutics in a clinical setting."

The core concept of synthetic lethality is that while a tumor can tolerate individual defects in its DNA, there exist combinations of defects that lead to the malignancy’s destruction. The recent approvals of several PARP inhibitors provide clear proof-of-principle for the approach of inducing synthetic lethality. Repare and its founders have developed large-scale and novel methods for discovering additional drug targets that, when inhibited, may induce synthetic lethality. New drugs directed at these targets hold promise to improve cancer treatment both as single therapies and in combination with existing drugs and treatments.

In addition to Segal, a Versant Entrepreneur-in-Residence, Repare’s seasoned management team also includes R&D Head Michael Zinda, Ph.D., who built and led AstraZeneca plc’s Oncology iMed Bioscience group in Boston; and VP of Discovery Cameron Black, Ph.D., an accomplished 18-year leader of Merck Frosst’s medicinal chemistry efforts. Repare has a 20-member team based in Montreal and Boston. To further guide this important investment both Jerel Davis, Ph.D., managing director at Versant, and Todd Foley, managing director at MPM Capital, will join Repare’s board of directors.

"The fields of synthetic lethality and DNA repair have a rich, 20-year history and are poised to deliver impactful new cancer treatments," said Davis. "We are impressed by the speed and precision with which Repare, in collaboration with its founders and scientific advisors, generated impressive insights and multiple novel targets."

Repare’s first disclosed program targets DNA-directed DNA polymerase theta (PolQ), a central component of a pathway that repairs double-strand breaks in cancer cells. NYU School of Medicine has licensed to Repare exclusive rights to drug discovery work targeting PolQ, developed by Dr. Sfeir with the support of NYU Office of Therapeutics Alliances (OTA). This unique polymerase is highly expressed in ovarian, breast and a number of other cancers. In parallel, Repare is progressing several additional programs, with an aim to put its first compound in the clinic in 2019.

"We evaluated nearly every opportunity in the synthetic lethality space and have complete conviction that Repare, its founders and its SAB members represent the leaders in the field," said Foley. "MPM is dedicated to investing in and building companies that seek to find cures for cancer and save lives and we look forward to the advancement of Repare’s programs and the development of these meaningful medicines."

Repare’s founders and scientific advisors are leaders in synthetic lethality and tumor repair machinery research. In addition to Dr. Sfeir from the Department of Cell Biology and the Skirball Institute of Biomolecular Medicine at NYU Langone Medical Center and Drs. Durocher and Sicheri from Toronto’s Lunenfeld-Tanenbaum Research Institute, Repare has assembled a world-class scientific advisory board, including:

Samuel Aparicio, Ph.D., chair of the Breast Cancer Program at BC Cancer Agency and professor of pathology and laboratory medicine at UBC, Vancouver.
Jim Carmichael, M.D. FRCP, lead of the Protein Homeostasis Thematic Center of Excellence at Celgene Corporation. He previously was U.K. regional director of medical science at AstraZeneca plc following its acquisition of KuDOS, where he was CMO and responsible for clinical development of olaparib.
Ronny Drapkin, M.D., Ph.D., director of the Penn Ovarian Cancer Research Center and director of gynecologic cancer research at the Basser Center for BRCA at the University of Pennsylvania.
Laurie Glimcher, M.D., president and CEO of the Dana-Farber Cancer Institute, Richard and Susan Smith Professor of Medicine at Harvard Medical School.
Mark Pegram, M.D., director of the breast cancer oncology program at Stanford Women’s Cancer Center and co-director of Stanford’s molecular therapeutics program.
Richard Wood, Ph.D. FRS, Grady F. Saunders Distinguished Professor in Molecular Biology at the University of Texas MD Anderson Cancer Center.

About Repare

Repare is developing new, precision oncology drugs for patients that target specific vulnerabilities of tumor cells. Its approach integrates insights from several fields of cell biology including DNA repair and synthetic lethality. Repare’s platform combines a proprietary, high-throughput, CRISPR-enabled gene editing target discovery method with high-resolution protein crystallography, computational biology and clinical informatics. The company is backed by leading global healthcare investors including founding investor Versant Ventures and MPM Capital. For additional information, please visit www.reparerx.com.

About Versant

Versant Ventures is a leading healthcare investment firm committed to helping exceptional entrepreneurs build the next generation of great healthcare companies. The firm invests across the healthcare sector and at all stages of company development, with an emphasis on the discovery and development of novel therapeutics. With $2.3 billion under management and offices in North America and Europe, Versant has built a team with deep investment, operating, and scientific expertise that enables a hands-on approach to company building. Since the firm’s founding in 1999, more than 65 Versant companies have achieved successful acquisitions or IPOs. For more information, please visit www.versantventures.com.

About MPM Capital

MPM Capital is an early‐stage life sciences venture firm founding and investing in companies that seek to cure major diseases by translating scientific innovations into positive clinical outcomes. MPM’s portfolio of companies aims to revolutionize the face of medicine across multiple areas including cancer, neuroscience, metabolic disorders, and regenerative medicine. With its experienced and dedicated team of operating executives and medical and scientific advisory board, MPM is powering novel medical breakthroughs that transform patients’ lives. MPM has more than $2.6 billion dollars in assets under management and is currently investing from two funds ─ the BV2014 and UBS Oncology Impact Fund. For further information, please visit www.mpmcapital.com.

About the Fonds de solidarité FTQ

The Fonds de solidarité FTQ is a development capital fund that channels the savings of Quebecers into investments. As at November 30, 2016, the organization had $12.2 billion in net assets, and through its current portfolio of investments has helped create and protect over 187,000 jobs. The Fonds is a partner in more than 2,600 companies and has nearly 618,000 shareholder-savers. fondsftq.com.

About BDC Capital

With more than $2 billion under management, BDC Capital is the investment arm of BDC, serving as a strategic partner to Canada’s most innovative and high potential firms. It offers a range of equity, venture capital and flexible growth and transition capital solutions to help Canadian entrepreneurs scale their businesses into global champions. To find out more, visit bdc.ca/capital.

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.

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.