On August 21, 2017 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of disease-driving genes, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SY-1425 for the treatment of acute myeloid leukemia (AML) (Press release, Syros Pharmaceuticals, AUG 21, 2017, View Source [SID1234520301]). SY-1425, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently in a Phase 2 clinical trial in genomically defined subsets of patients with AML and myelodysplastic syndrome (MDS).

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"Treatment of AML remains a significant unmet medical need, with many patients lacking adequate therapeutic options," said David A. Roth, M.D., Syros’ Chief Medical Officer. "We believe that SY-1425 may provide a meaningful benefit for subsets of AML patients whose disease is driven by abnormally high expression of the RARA or IRF8 genes. Receiving orphan drug designation is an important regulatory milestone in the development of SY-1425. We’re pleased with the continued progress of the ongoing Phase 2 clinical trial, and we look forward to presenting initial clinical data in the fourth quarter of this year."

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

Using its gene control platform, Syros discovered subsets of AML and MDS patients with super-enhancers associated with RARA or IRF8. Syros identified proprietary biomarkers related to these super-enhancers. These super-enhancers are believed to drive overexpression of the RARA or IRF8 genes, locking cells in an immature, undifferentiated and proliferative state, leading to disease. In preclinical studies, SY-1425 promoted differentiation of AML cells with high RARA or IRF8 expression and inhibited tumor growth and prolonged survival in patient-derived xenograft models of AML with high RARA expression. Syros estimates that about one-third of AML and MDS patients have either the RARA or IRF8 biomarker, or both.

The ongoing Phase 2 clinical trial of SY-1425 is assessing the safety and efficacy of SY-1425 as a single agent in four AML and MDS patient populations, as well as in combination with azacitidine, a standard-of-care therapy, in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy. All patients in the trial are prospectively selected using biomarkers for high expression of RARA or IRF8. Additional details about the trial can be found using the identifier NCT02807558 at www.clinicaltrials.gov.

On August 21, 2017 Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, reported that updated data from its ongoing Phase 1 clinical trial evaluating BLU-554 in patients with advanced hepatocellular carcinoma (HCC) will be presented in oral presentations at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress on September 10, 2017 in Madrid, Spain and at the 11th International Liver Cancer Association (ILCA) Annual Conference on September 17, 2017 in Seoul, South Korea (Press release, Blueprint Medicines, AUG 21, 2017, View Source;p=irol-newsArticle&ID=2294896 [SID1234520303]). BLU-554 is a potent and highly selective inhibitor of FGFR4.

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At ESMO (Free ESMO Whitepaper) and ILCA, Blueprint Medicines anticipates presenting updated safety and clinical activity data from its ongoing Phase 1 clinical trial of BLU-554. As of the most recent data cutoff date of August 18, 2017, 38 patients with FGFR4 pathway activation, as indicated by FGF19 overexpression, were evaluable for clinical activity, and an objective response rate of 16 percent (95 percent confidence interval 6-31 percent) was observed in this population.

"The preliminary BLU-554 data announced today continue to provide support for selective FGFR4 inhibition as a novel targeted treatment approach in biomarker-selected patients with hepatocellular carcinoma," said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. "As we evaluate the results for BLU-554 in the context of historical data for currently approved therapies showing response rates of less than 10 percent, we are encouraged that radiographic tumor reductions were observed in a refractory population with progressive disease and few or no remaining therapeutic options. We look forward to sharing a comprehensive update on the ongoing BLU-554 clinical trial, as well as the path forward for further development, in September."

Details of the presentations are as follows:

2017 ESMO (Free ESMO Whitepaper) Congress

Presentation Title: Phase 1 Safety and Clinical Activity of BLU-554 in Advanced Hepatocellular Carcinoma (HCC)
Session Title: Developmental Therapeutics
Date & Time: Sunday, September 10, 2017 from 5:30 – 5:45 p.m. CET (11:30 – 11:45 a.m. ET)
Abstract ID: 365O
Location: Cordoba Auditorium, IFEMA, Feria de Madrid

11th ILCA Annual Conference

Presentation Title: Clinical Activity Of BLU-554, A Potent, Highly-Selective FGFR4 Inhibitor In Advanced Hepatocellular Carcinoma (HCC) With FGFR4 Pathway Activation
Session Title: General Session 5
Date & Time: Sunday, September 17, 2017 from 11:30 a.m. – 1:00 p.m. KST (Saturday, September 16, 2017 from 10:30 p.m. – 12:00 a.m. ET)
Abstract No: O-032
Location: Grand Hyatt Seoul

On August 17, 2017 Cancer Research UK* and Newcastle University reported a three year extension to their major strategic drug discovery alliance with Astex Pharmaceuticals, a pharmaceutical company dedicated to the discovery and development of novel small molecule therapeutics (Press release, Cancer Research Technology, AUG 17, 2017, View Source [SID1234523162]).

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The alliance, which was formed five years ago, brings together world-leading researchers in structural and cellular biology, and medicinal chemistry with the innovative fragment-based small molecule drug discovery and development capabilities of Astex.

The researchers at the Cancer Research UK Drug Discovery Programme at the Northern Institute for Cancer Research (NICR), Newcastle University, will work to identify and develop new cancer drugs and associated biomarkers.

The existing portfolio of research consists of multiple projects spanning target validation and early stage medicinal chemistry, with projects progressing towards the more advanced stages of preclinical development.

The new agreement also includes provisions for further extension of the alliance towards the end of the new three year term.

Astex retains the right to an exclusive worldwide licence to take the most promising projects forward into pre-clinical and clinical drug development. In return, Cancer Research UK and Newcastle University are eligible to receive milestone and royalty payments on any compounds that Astex takes into clinical development and successfully commercialises.

Dr Iain Foulkes, Cancer Research UK’s executive director of research and innovation, said: "We’re delighted to extend this major collaboration which accelerates the development of Cancer Research UK’s world class work into new treatments for patients.

"Promising new compounds resulting from this partnership are now progressing towards the next stage of development. Multi-project alliances like this are powerful engines for innovation and drug discovery and this announcement underlines Cancer Research UK’s exceptional track record of bringing these together successfully."

Steve Wedge, Professor of Stratified Cancer Medicine Discovery at Newcastle University, said: "The innovative academic-industry collaborative model pioneered with Astex has been a genuine success and we are thrilled to be able to continue working in partnership on our drug discovery research.

"The alliance benefits significantly from complementary expertise and provides a route to progress promising novel therapies towards clinical use."

Dr Harren Jhoti, President and Chief Executive Officer of Astex, said: "The extension of our agreement with Newcastle and Cancer Research UK underlines the success of our existing alliance and the importance we place on collaboration with world leading academic research groups to strengthen our efforts to discover new treatments for patients.

"We look forward to continuing our important work and to continued success in bringing new compounds into development."

On August 17, 2017 WuXi Biologics (2269.HK), a global leading open-access biologics technology platform company offering end-to-end solutions for biologics discovery, development and manufacturing, and its Chinese partner Harbin Gloria Pharmaceuticals Co Ltd (002437.CN), reported that an exclusive license to the anti-PD-1 antibody GLS-010 has been granted to Arcus Biosciences, a US-based biotechnology company focused on the discovery and development of innovative cancer immunotherapies (Press release, WuXi Biologics, AUG 17, 2017, View Source [SID1234521934]).

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Gloria contracted WuXi Biologics to discover and develop GLS-010, a novel anti-PD-1 antibody, using Ligand’s transgenic rat platform OmniRat. GLS-010 is currently being evaluated in cancer patients in phase I clinical studies in China. Arcus has licensed the exclusive development and commercialization rights of GLS-010 in North America, Europe, Japan and certain other territories.

Arcus plans on developing GLS-010 as a combination product with the other product candidates in its portfolio. Based on the terms of the agreement, Arcus will pay $18.5mm in upfront payments as well as development and regulatory milestones which could total up to $422.5mm for the development and approval of 11 products that include GLS-010 as a component. WuXi Biologics and its partner Gloria, through an existing agreement will also receive commercial milestones of up to $375mm which could result in aggregate payments from Arcus of $816mm. Arcus will pay tiered royalties that range from the high single-digits to low double-digits on net sales of GLS-010. In addition, WuXi Biologics and Arcus intend to enter into an exclusive 3-year agreement for the development of Arcus’ biologics portfolio. WuXi Biologics also will be the exclusive manufacturer for GLS-010 in the licensed territories for a specified period of time.

"We are pleased that our integrated platform has enabled companies such as Gloria to enter into biologics with an exciting program. We are also excited to enter into this agreement to expedite biologics development to treat patients globally," commented Dr. Chris Chen, CEO and executive director of WuXi Biologics. "This new partnership continues to reinforce the value of our integrated service platform, the global quality WuXi Biologics commits to, and the success of our ‘follow-the-molecule’ strategy.

"We are thrilled to gain access to GLS-010 in our territories," commented Dr. Tim Sullivan, Vice President of Business Development at Arcus. "This molecule will enable us to fully exploit the potential of our other immuno-oncology agents for the benefit of patients. Working with WuXi Biologics also ensures high-quality clinical supply of our biologics, an essential operational component of our strategy to develop a series of novel and best-in-class combination therapies for the treatment of cancer."

"Gloria has recently been focusing its R&D research on biologics, especially in the immuno-oncology area. We hope our efforts can bring more innovative medicines into the Chinese market in order to fill unmet medical needs," said Mr. Hongbing Yang, Chief Executive Officer of Harbin Gloria Pharmaceuticals. "It is the first time that an antibody envisioned by Gloria has the potential to reach patients worldwide. We are extremely pleased that, with our development in China and Arcus’ exclusive license in many other countries in the world, each working with WuXi, that GLS-010 may become available in both China and worldwide."

About GLS-010

GLS-010, also referred to as WBP3055, is an investigational fully human monoclonal antibody that belongs to a class of immuno-oncology agents known as immune checkpoint inhibitors. It is designed to bind to PD-1, a cell surface receptor that plays an important role in the downregulation of the immune system by preventing the activation of T-cells. Other anti-PD-1 antibodies have been approved by the US FDA in multiple cancer settings. It is estimated that more than 500 clinical trials are ongoing to continue to investigate this class of biologics for more than 20 different cancer indications.

On August 17, 2017 Atreca, Inc., a biotechnology company focused on developing novel therapeutics based on a deep understanding of the human immune response, reported the completion of its Series B round of financing with a total investment of $35 million (Press release, Atreca, AUG 17, 2017, View Source [SID1234522949]). The financing was co-led by new investor Wellington Management Company LLP and by a large US-based, healthcare-focused fund, participating as an existing Atreca investor. Additional participation included new investor Cormorant Asset Management, based in Boston, as well as other new and existing investors.

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Proceeds from the financing will be used to accelerate and broaden Atreca’s discovery and development of antibody-based therapeutics that initiate, shape, and drive the anti-tumor immune response in cancer patients, as enabled by the Company’s proprietary Immune Repertoire Capture (IRC) technology. Atreca’s technology provides unbiased and virtually error-free antibody and T cell receptor (TCR) sequences at high throughput from single B and T cells of active human immune responses, enabling the identification, generation, and analysis of functional human antibodies and TCRs directly from such responses.

"We are very pleased with the continued support of our existing investors, as well as the commitment of our new, high-caliber investors," said Tito A. Serafini, Ph.D., Atreca’s President, Chief Executive Officer, and Co-Founder. "With their participation, we continue to build a foundation for long-term growth. This funding enables us to accelerate the acquisition of immune response data central to cancer immunotherapy and to expand and advance our pipeline of novel antibody therapeutics based on those data." To date, Atreca has built a library of over 400 patient-derived antibodies that bind non-autologous tumor tissue as the foundation for its pipeline and anticipates nominating a clinical candidate in its lead oncology program by the end of the year.

"Atreca’s novel and differentiated approach has the potential to address broad, compelling unmet needs in diverse immuno-oncology applications, a global market that is anticipated to exceed more than $100 billion within five years," said Brian Atwood, Atreca’s Chairman. "We are excited by the Company’s continued momentum and the top-tier financing syndicate participating in the Series B round."