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 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."

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 June 20, 2017 Almac Discovery (www.almacgroup.com/discovery), a biopharmaceutical company focused on discovering and identifying innovative therapeutics for the treatment of cancer, and Debiopharm International SA (Debiopharm – www.debiopharm.com), a Switzerland based specialty pharma company, reported a collaboration to pursue the development of Almac Discovery’s Wee-1 kinase inhibitor program (Press release, Debiopharm, JUN 20, 2017, View Source [SID1234525171]).

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Under the terms of the license agreement, Almac Discovery has granted exclusive global rights to its Wee-1 patent families and related subject matter expertise to Debiopharm International SA. Almac Discovery is eligible to receive an upfront payment with further development and commercial milestone payments in addition to receiving royalties from Debiopharm.

The area of DNA damage response and cell cycle regulation has seen notable activity from the pharmaceutical industry recently, including some high profile drug approvals for cell cycle kinase inhibitors. Many cancer treatments such as radiation, antimetabolites, alkylating agents, DNA topoisomerase inhibitors and platinum compounds damage DNA in cells. The cellular response is to arrest the cell cycle temporarily to allow for DNA repair, hence reducing the efficacy of chemotherapy.

The Wee-1 kinase is a key regulator of a number of cell cycle checkpoints and inhibition of Wee1 can force arrested cells through the cell cycle leading to cell death. The Almac Discovery Wee-1 inhibitors exhibit good drug-like properties, high potency, excellent selectivity and demonstrate potent anti-proliferative activity in vitro and in vivo, both as single agents and in combination with genotoxics, in several cancer models.

"We are thrilled to pursue the outstanding work of Almac Discovery on this promising target." said Dr. Bertrand Ducrey, CEO of Debiopharm International SA, who added "Almac Discovery’s compelling expertise in medicinal chemistry and drug discovery is very complementary with Debiopharm business model and we look forward to developing these compounds to offer an innovative treatment for patients with high medical need."

Dr Stephen Barr, Managing Director & President, Almac Discovery commented "We are extremely pleased to announce this agreement with Debiopharm who have proven expertise and an impressive track record of developing drug candidates. The professionalism, dedication and commitment of their team is aligned perfectly with our own core values making them the number one choice to develop our Wee-1 inhibitor. Agreements of this nature enable both companies to continue to strive to improve human health worldwide and we look forward to working with them."

On June 19, 2017 Seattle Genetics, Inc. (Nasdaq: SGEN), a global biotechnology company, reported that it is discontinuing the phase 3 CASCADE clinical trial of vadastuximab talirine (SGN-CD33A) in frontline older acute myeloid leukemia (AML) patients (Press release, Seattle Genetics, JUN 19, 2017, View Source;p=RssLanding&cat=news&id=2281531 [SID1234519607]).

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Seattle Genetics took this action following consultation with the Independent Data Monitoring Committee (IDMC) and after reviewing unblinded data on June 16, 2017. The data indicated a higher rate of deaths, including fatal infections in the vadastuximab talirine-containing arm versus the control arm of the trial. Based on available data, the safety concerns in this trial do not appear related to hepatotoxicity. Seattle Genetics is suspending patient enrollment and treatment in all of its vadastuximab talirine clinical trials including the ongoing phase 1/2 clinical trial in frontline high risk myelodysplastic syndrome (MDS). Seattle Genetics will closely review the data and consult with the U.S. Food and Drug Administration (FDA) to determine future plans for the vadastuximab talirine development program.

"This is a disappointing and unexpected result for the CASCADE trial. Patient safety is our highest priority, and we will closely review the data and evaluate next steps. AML is a devastating disease with a poor prognosis in most patients, and there is a great need for therapeutics against this disease. We thank the patients, caregivers and investigators for their support of this trial," said Clay Siegall, Ph.D., President and Chief Executive Officer at Seattle Genetics. "We are enthusiastic about the many opportunities across our broad pipeline, including ADCETRIS (brentuximab vedotin), enfortumab vedotin (ASG-22ME) and SGN-LIV1A. Notably, we are looking forward to reporting data from our ADCETRIS phase 3 ECHELON-1 trial in frontline Hodgkin lymphoma, and we are on track to advance enfortumab vedotin into a pivotal trial in metastatic urothelial cancer in the second half of 2017 under our collaboration with Astellas."

The phase 3 CASCADE clinical trial is a randomized, double-blind, placebo-controlled study evaluating vadastuximab talirine in combination with the hypomethylating agents (HMAs) azacitidine or decitabine compared to an HMA alone in older patients with newly diagnosed AML. Vadastuximab talirine is a novel investigational ADC targeted to CD33 utilizing Seattle Genetics’ proprietary ADC technology. CD33 is expressed on most AML and MDS blast cells.