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).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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 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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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."