On December 12, 2018 Gibson Oncology, LLC ("Gibson"), a privately held clinical stage company developing a novel class of oncology drugs for treating adult and pediatric cancers resistant to traditional cancer drugs, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to LMP-400, Gibson’s novel small molecule topoisomerase 1inhibitor for the treatment of Ewing sarcoma, a rare pediatric cancer (Press release, Gibson Oncology, DEC 12, 2018, View Source [SID1234532034]). LMP-400 has successfully completed multiple phase I human clinical trials as a single agent in advanced stage cancer patients with demonstrated anti-cancer activity and improved safety attributes over first generation topoisomerase inhibitors.

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Mr. Randall Riggs, President & CEO of Gibson Oncology, stated that "Gibson Oncology is excited that the U.S. FDA has granted LMP-400 RPDD, which has the potential to greatly benefit kids diagnosed with Ewing Sarcoma. We intend to conduct phase 2 trials with LMP-400 that will employ a recently discovered biomarker called Schlafen11 to select pediatric patients most likely to respond."

In a previous study performed at the National Cancer Institute (NCI) by Dr. Pommier et. al., cancer cells expressing high levels of Schlafen11 were hypersensitive to LMP-400. Ewing Sarcoma patients commonly have very high expression levels of Schlafen11 and are therefore excellent candidates for treatment with LMP-400.

The FDA grants RPDD for diseases that primarily affect children from birth to age 18, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

About Ewing Sarcoma

Ewing sarcoma is the second most common bone malignancy among children and adolescents. According to a study published in the Journal of Hematology/Oncology, the incidence is about 3 cases per 1 million per year in children younger than age 20. Despite the favorable prognosis, an American Cancer Society study showed that approximately 30-40% of patients develop metastases or local recurrence, and the long-term survival rate for refractory or recurrent disease is only 22-24%. The relapsed and refractory statistics underscore the need for new treatment options.

On December 12, 2018 Apexian Pharmaceuticals reported is shedding light on the question of how pre-leukemic cells transform into full-blown leukemia (Press release, Apexian Pharmaceuticals, DEC 12, 2018, View Source [SID1234532134]).

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Genetic mutations by themselves are rarely enough to flip the switch. Inflammation also plays a role. But, until now, the question of "how" remained unanswered.

Apexian Chief Science Officer Mark Kelley, PhD, and his colleagues presented their findings at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s December 1, 2018 meeting in San Diego, California. When a putative tumor-suppressor gene called TET2 does not function well, acute inflammation or infection can enhance the production of myeloid stem and progenitor cells in the bone marrow that are precursors to circulating mature myeloid cells. The TET2 loss of function amplifies; inflammatory proteins increase, and myeloid cells rapidly mature, increasing in sheer numbers as well as developing resistance to programmed cell death.

Myeloid cells, which contribute to immunity, are normally very short-lived. However, when those cells live too long or become too numerous, dangerous levels of inflammation can result. Using a mouse model, Kelley and his colleagues demonstrated that Apexian’s flagship compound APX3330 can prevent precancerous cells from proliferating and block cells from making inflammatory proteins.

Such anti-inflammatory therapy could be of clinical value in people carrying TET2 mutations.

"Apexian continues to develop a robust portfolio of APE1/Ref-1 compounds that have broad utility in oncology, hematology and other diseases", says Steve Carchedi, President and CEO. "We are excited by the recent scientific findings and continue to expand our research and development beyond solid tumors"

APX3330 also has clinical utility with solid tumors. A Phase 1 trial for patients with advanced solid tumors is concluding.

On December 12, 2018 Checkmate Pharmaceuticals Inc., a clinical stage biopharmaceutical company focused upon activation of innate immunity to treat advanced cancer, reported that it has appointed Barry Labinger as President and CEO. Arthur M. Krieg, MD, Checkmate’s founder and current President and CEO, will assume the newly-created role of Chief Scientific Officer (Press release, Checkmate Pharmaceuticals, DEC 12, 2018, View Source [SID1234532035]). Both Mr. Labinger and Dr. Krieg will serve on Checkmate’s Board of Directors.

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Checkmate also announced the completion of a $22 million financing led by a new investor, Decheng Capital. The financing also included participation from existing investors, Sofinnova, venBio, and F-Prime.

"Checkmate’s encouraging early clinical data provide a strong foundation for its further growth as a leading cancer immunotherapy company with broad potential. Barry’s deep experience in oncology drug development and commercialization, along with this additional funding, will support Checkmate’s further progress," commented Mike Powell, Chairman of the Board of Checkmate, and General Partner, Sofinnova Ventures. "Art has successfully led the company to this critical stage in its growth, including this planned leadership transition. We are delighted to have Barry as CEO and Art as CSO. We are confident that they will successfully guide Checkmate to its next stage of evolution," continued Dr. Powell.

Mr. Barry Labinger brings to Checkmate nearly three decades of pharmaceutical and biotech industry experience, most recently serving as President, CEO, and Director of Biothera Pharmaceuticals, a clinical stage cancer immunotherapy company focused on innate immune activation. Prior to Biothera, he was Executive Vice President and President, Biosciences Division, at Emergent BioSolutions Inc., where he led the development and commercialization of oncology, immunology, and hematology products and product candidates. He previously held leadership roles at Human Genome Sciences, 3M Pharmaceuticals, and Immunex.

"I firmly believe in the importance of innate immune activation to enhance the benefits of other cancer immunotherapeutics," said Mr. Labinger. "CMP-001 has demonstrated best-in-class potential, and I’m excited to join Art, the Checkmate team, and our Board to maximize the potential of CMP-001 to improve outcomes for cancer patients."

"My initial goal in founding Checkmate was to determine if intratumoral TLR9 activation could induce tumor regression in anti-PD-1 refractory advanced cancer patients," Dr. Krieg commented. "Now that we have achieved that goal, I am delighted to turn over Checkmate’s leadership to Barry, so that I can focus my own efforts on the further scientific development of our platform. I believe that our complementary experience and our shared vision will enable us to accelerate and maximize Checkmate’s further development."

Proceeds from the financing will support ongoing clinical development of Checkmate’s lead product CMP-001 in advanced melanoma and non-small cell lung cancer (NSCLC) refractory to prior anti-PD-1 therapy, along with expansion into additional solid tumor types.

On December 12, 2018 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company focused on developing a pipeline of first-in-class AXL kinase inhibitors to treat multiple cancer indications, reported that the Company will be presenting key results from its Phase II clinical programme with selective AXL inhibitor bemcentinib at the DNB’s 9th Annual Nordic Healthcare Conference in Oslo, Norway, today at 10:15-10:40 CET (Press release, BerGenBio, DEC 12, 2018, View Source [SID1234532017]).

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The slides are available for download at the Company’s website: www.bergenbio.com/investors/presentations/

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "We have made significant progress during 2018 establishing clinical proof of concept for the remarkable utility of bemcentinib in cancers with high unmet clinical need and large market potential. We have shown that our once-a-day, oral selective AXL inhibitor is well tolerated as a monotherapy and in combination with immune-, targeted and chemotherapies. What is more, consistently we have seen that efficacy scales with AXL biomarker expression leading to superior response rates in AXL biomarker positive relapsed/refractory AML and MDS as a monotherapy as well as non-small cell lung cancer where we are combining with the immunotherapy blockbuster Keytruda. We have met all our operational milestones this year and are looking forward to starting a randomised phase II programme towards the end of H1 2019 based on key results obtained this year".

On December 12, 2018 Apexian Pharmaceuticals reported is shedding light on the question of how pre-leukemic cells transform into full-blown leukemia (Press release, Apexian Pharmaceuticals, DEC 12, 2018, View Source [SID1234532036]).

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!

Genetic mutations by themselves are rarely enough to flip the switch. Inflammation also plays a role. But, until now, the question of "how" remained unanswered.

Apexian Chief Science Officer Mark Kelley, PhD, and his colleagues presented their findings at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s December 1, 2018 meeting in San Diego, California. When a putative tumor-suppressor gene called TET2 does not function well, acute inflammation or infection can enhance the production of myeloid stem and progenitor cells in the bone marrow that are precursors to circulating mature myeloid cells. The TET2 loss of function amplifies; inflammatory proteins increase, and myeloid cells rapidly mature, increasing in sheer numbers as well as developing resistance to programmed cell death.

Myeloid cells, which contribute to immunity, are normally very short-lived. However, when those cells live too long or become too numerous, dangerous levels of inflammation can result. Using a mouse model, Kelley and his colleagues demonstrated that Apexian’s flagship compound APX3330 can prevent precancerous cells from proliferating and block cells from making inflammatory proteins.

Such anti-inflammatory therapy could be of clinical value in people carrying TET2 mutations.

"Apexian continues to develop a robust portfolio of APE1/Ref-1 compounds that have broad utility in oncology, hematology and other diseases," says Steve Carchedi, President and CEO. "We are excited by the recent scientific findings and continue to expand our research and development beyond solid tumors."

APX3330 also has clinical utility with solid tumors. A Phase 1 trial for patients with advanced solid tumors is concluding