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Aduro Biotech Announces Advancement of ADU-S100 into Global Combination Trial With PDR001 for the Treatment of Solid Tumors and Lymphomas

On September 26, 2017 Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, reported that the first patient has been dosed in the Phase 1b dose escalation and dose expansion clinical trial (see www.clinicaltrials.gov, identifier NCT03172936) designed to evaluate the safety and efficacy of ADU-S100 (also known as MIW815), a novel STING pathway activator, in combination with PDR001, Novartis’ investigational PD-1 checkpoint inhibitor, for the treatment of advanced/metastatic solid tumors or lymphomas (Press release, Aduro Biotech, SEP 26, 2017, View Source [SID1234520631]). The trial, which is being conducted in collaboration with Aduro’s partner, Novartis, is expected to enroll approximately 175 patients at sites located in the United States, Europe, Canada, Australia and Japan.

“We are pleased with our early progress in the ongoing Phase 1 dose escalation trial of ADU-S100 as a single agent and are eager to expand our investigation into a separate clinical trial to evaluate the effect of ADU-S100 used in combination with the PDR001 checkpoint inhibitor,” said Natalie Sacks, M.D., chief medical officer of Aduro Biotech. “As a leader in STING activation, we look forward to gaining more insight into the potential therapeutic application of this novel combination therapy.”

Clinical Design of Phase 1b ADU-S100 (MIW815)/PDR100
The Phase 1b multi-center, open-label study is designed to evaluate the safety and efficacy of ADU-S100 (MIW815) in combination with PDR001 in patients with accessible solid tumors or lymphomas. The trial will evaluate two treatment schedules of ADU-S100 in dose escalation. One group will receive a fixed dose of intravenous PDR001 on day 1 and an intratumoral injection of ADU-S100 three times in a 28-day cycle. Another group will receive a fixed dose of intravenous PDR001 on day 1 and intratumoral injection of MIW815 (ADU-S100) on day 1 of every 28-day cycle. Once the maximum tolerated dose and/or recommended dose for expansion is determined, the expansion part of the study will open.

Ongoing Phase 1 Dose Escalation Trial of ADU-S100 (MIW815) in Multiple Tumor Types
In May 2016, Aduro announced the initiation of the ongoing Phase 1, multicenter, dose escalation study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and anti-tumor activity of ADU-S100 (MIW815) in patients with cutaneously accessible metastatic solid tumors or lymphomas (see www.clinicaltrials.gov, identifier NCT02675439). The trial is ongoing.

About STING Pathway Activator Platform
The Aduro-proprietary STING pathway activator product candidates, including ADU-S100 (MIW815), are synthetic small molecule immune modulators that are designed to target and activate human STING. STING is generally expressed at high levels in immune cells, including dendritic cells. Once activated, the STING receptor initiates a profound innate immune response through multiple pathways, inducing the expression of a broad profile of cytokines, including interferons and chemokines. This subsequently leads to the development of a systemic tumor antigen-specific T cell adaptive immune response.

About Aduro/Novartis Collaboration
In March 2015, Aduro and Novartis entered into a collaboration and license agreement relating to the global research, development and commercialization of immuno-oncology products derived from Aduro’s proprietary STING pathway activator platform technology. Under the terms of the agreement, Aduro received an upfront payment from Novartis of $200 million and a $35 million development milestone upon initiation of the Phase 1 trial for ADU-S100 in May 2016, and if all development and regulatory milestones are met, is eligible to receive up to an additional aggregate amount of $465 million. The collaboration is guided by a joint steering committee with both Aduro and Novartis having final decision making authority regarding specified areas of development or commercialization. Pending regulatory approval, Aduro will lead commercialization activities and will book sales in the United States for any products developed and commercialized pursuant to this collaboration, and Novartis will lead commercialization activities in all other regions. The companies will share in profits, if any, in the United States, Japan and major European countries. Novartis will pay Aduro a mid-teens royalty for sales in the rest of the world. Aduro maintains rights to its STING platform technology in all therapeutic areas outside of oncology, including infectious disease and autoimmunity, among others.

BioLineRx Announces Initiation of Phase 1b/2 Trial for BL-8040 in AML Under Immunotherapy Collaboration

On September 26, 2017 BioLineRx Ltd. (NASDAQ/TASE:BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported the initiation of a Phase 1b/2 trial for BL-8040 in combination with atezolizumab (TECENTRIQ), an anti-PDL1 cancer immunotherapy from Genentech, a member of the Roche Group (Press release, BioLineRx, SEP 26, 2017, View Source [SID1234520628]). The trial, known as the BATTLE study (NCT03154827), will focus on the maintenance treatment of patients with intermediate- and high-risk acute myeloid leukemia (AML) who have achieved a complete response (CR) following induction and consolidation therapy.

Up to 60 patients are planned to be enrolled in this multicenter, single arm, open-label study, to evaluate the relapse-free survival, minimal residual disease status, safety and tolerability of the combination of BL-8040 and atezolizumab for maintenance treatment in AML patients. The study’s primary endpoint is to assess whether the combination of BL-8040 and atezolizumab prolongs relapse free survival. In addition, the effect of the combination therapy on minimal residual disease, multiple immunological parameters, and potential biomarkers will be evaluated. The trial is planned to take place at approximately 22 sites in the US, Europe and Israel.

The BATTLE study is part of BioLineRx’s cancer immunotherapy collaboration with Genentech to conduct several Phase 1b/2 studies investigating the combination of BL-8040 with atezolizumab in multiple cancer indications, announced in September 2016.

Philip Serlin, Chief Executive Officer of BioLineRx, stated, “We are excited to commence yet another clinical study under this significant immuno-oncology collaboration, following the recent initiation of a study to evaluate the combination of the same two drugs for the treatment of pancreatic cancer. The BATTLE study is the first study under our collaboration in hematologic malignancies, and we are hopeful that combining atezolizumab with BL-8040 will demonstrate the potential to establish a new treatment for AML patients that would extend the duration of remission following induction treatment, in particular for patients for whom stem-cell transplantation is inappropriate. We look forward to the initiation of additional combination studies under this collaboration, all planned by the end of this year.”

BL-8040, BioLineRx’s lead oncology platform, is a CXCR4 antagonist that has been shown, in a successful Phase 2a study in relapsed and refractory AML patients, to be a robust mobilizer of immune and tumor cells and to be effective in inducing direct tumor cell death.

These two effects, when combined with atezolizumab-induced blockade of the interaction between PD-L1 with PD-1 and B7.1, are hypothesized to have a beneficial effect on the minimal residual disease (MRD) status of AML patients. Specifically, this combined approach could potentially reduce an AML patient’s MRD status from positive to negative, and possibly have a favorable effect on disease outcome. This study’s regimen aims at further prolonging the period of remission, exploring a novel maintenance approach to these patients.

About BL-8040
BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and stem cell mobilization. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells from the bone marrow, thereby sensitizing these cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing cell death (apoptosis). In addition, BL-8040 has also demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, T, B and NK cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Acute Myeloid Leukemia (AML)
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow and is the most common type of acute leukemia in adults. According to the American Cancer Society, approximately 20,000 new cases of AML were diagnosed in the United States in 2016, and the median age of AML patients was 67 years old. The first treatment line for patients with AML includes a combination of chemotherapy drugs and is called induction treatment. The median survival for AML patients receiving induction chemotherapy is less than two years, with shorter survival for patients over the age of 60 or for those with certain gene or chromosome aberrations. Due to relapsed or refractory disease (where the disease is not responsive to standard treatments), the overall five-year survival rate for AML is between 10 and 40 percent.

SpringWorks Therapeutics Launches with $103M in Series A Funding and Rights to Four Clinical Programs

On September 25, 2017 SpringWorks Therapeutics, a mission-driven medicines company dedicated to developing innovative potential new treatments for underserved patient communities, reported its launch with a completed $103 million Series A financing funded by Bain Capital Life Sciences, Bain Capital Double Impact, OrbiMed, Pfizer (NYSE:PFE) and LifeArc (formerly known as MRC Technology) (Press release, SpringWorks Therapeutics, SEP 25, 2017, View Source [SID1234529339]). SpringWorks Therapeutics also has rights to four clinical-stage experimental therapies from Pfizer.

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"SpringWorks Therapeutics will pursue the development of medicines across therapeutic areas, focused on diseases where there is an urgent need and the potential for the greatest impact for patients," said Lara S. Sullivan, M.D., MBA, Founder and President of SpringWorks Therapeutics and a former Vice President at Pfizer. "We initially have rights to four very promising experimental therapies and, over time, plan to expand our pipeline by partnering with other life science companies and academic institutions who share in our mission."

New Approach to Drug Development

SpringWorks Therapeutics was originally conceived by Pfizer as an innovative way to advance investigational therapies that may hold significant promise for underserved patients. SpringWorks Therapeutics’ collaborative business model is designed to deliver both social and financial returns via partnerships with a variety of stakeholders, including scientists, biopharmaceutical partners, patient groups, funders and philanthropists. Pfizer’s contribution consists of both equity capital and royalty- and milestone-bearing licenses to experimental therapies.

"Pfizer sees SpringWorks Therapeutics as a groundbreaking new model for collaboration to deliver on the promise of medical research and development, so that more people have the potential to overcome disease. We hope that our investment in SpringWorks Therapeutics will, over time, enable us to realize even more value for patients and society," said Freda Lewis-Hall, M.D., DFAPA, Executive Vice President and Chief Medical Officer, Pfizer. "SpringWorks Therapeutics started as an idea about a new way to get things done with—and for—patients, it’s been a tremendous team effort, and we and our partners are excited to see it become a reality."

Promising Pipeline with Four Clinical-Stage Experimental Therapies

SpringWorks Therapeutics is focused on underserved patient populations where there is great medical need. The company plans to move forward potential programs for four diseases, all of which currently have no cure. SpringWorks Therapeutics plans to expand its pipeline by partnering with other life science companies and academic institutions who share in the company’s mission.

DESMOID TUMOR

A desmoid tumor is a rare, non-metastatic tumor of connective tissue cells, which can cause severe morbidity, pain and loss of function in children and adults. Desmoid tumors can show up in almost any part of the body, and desmoids that are faster growing or located near vital organs can cause life-threatening problems. Approximately 900-1,200 people are diagnosed with desmoid tumors each year in the U.S.1 Currently available treatments include unapproved medical therapy, radiation therapy, thermal ablation and surgery, which can be dangerous, costly and offer limited effectiveness. SpringWorks Therapeutics is planning to initiate a Phase 3 program to establish safety and efficacy of nirogacestat (PF-03084014), its gamma-secretase inhibitor, and will work collaboratively with the Desmoid Tumor Research Foundation to enable the needs of the patient community to be addressed.

NEUROFIBROMATOSIS

Neurofibromatosis (NF) refers to three genetic disorders—NF1, NF2 and schwannomatosis—which cause tumors to grow on nerves throughout the body and can lead to blindness, deafness, disfigurement, cancer, bone abnormalities, learning disabilities and severe pain. NF1 affects one in 3,000 individuals and usually is diagnosed in childhood when symptoms begin to appear.2 MEK inhibitors have shown encouraging activity in reducing tumor size in clinical Phase 1-2 studies in patients with plexiform neurofibromatosis, one of the many manifestations of NF1. SpringWorks Therapeutics is planning to initiate a Phase 3 program to establish safety and efficacy of its MEK 1/2 inhibitor (PD-0325901) in the NF1 population and will work collaboratively with the Children’s Tumor Foundation to enable the needs of the patient community to be addressed.

HEREDITARY XEROCYTOSIS

Hereditary xerocytosis (HX) is a genetic disorder in which red blood cells become dehydrated due to loss of potassium and cell water. The fragility of the dehydrated red cells can lead to a ranging severity of anemia and can cause complications including jaundice, fatigue, splenomegaly and gallstones. In some cases, it will lead to severe anemia that requires frequent blood transfusions. HX affects an estimated one in 10,000 people, and symptoms begin shortly after birth.3 There is no approved therapy for this disease. Senicapoc (PF-05416266) has demonstrated a good safety/tolerability profile in previous Phase 1-3 studies in other indications. SpringWorks Therapeutics plans to assess the potential activity of senicapoc in hereditary xerocytosis.

POST-TRAUMATIC STRESS DISORDER

Post-traumatic stress disorder (PTSD) is a chronic condition that some people develop after experiencing traumatic or life-threatening events, serious injury or sexual violence. PTSD involves the persistent re-experiencing of the traumatic event, which results in avoidance of trauma-related stimuli, as well as negative feelings and heightened anxiety-like symptoms. PTSD is often seen in military veterans, first responders, rape and battery victims, and abused children. Around 8.6 million people in the U.S. between the ages of 18-64 have been diagnosed with the disease.4 Over 200,000 veterans in the U.S. live with PTSD, which is currently treated with antidepressants such as SSRIs and trauma-focused psychotherapy; however, the disease can result in suicide even with treatment. SpringWorks Therapeutics’ FAAH inhibitor (PF-0445784) has demonstrated a good safety/tolerability profile in previous Phase 1 studies. The effectiveness of PF-0445784 in PTSD is to be determined. Cohen Veterans Bioscience (CVB) and SpringWorks Therapeutics plan to assess patient populations that could benefit from this mechanism.

Experienced Leadership Team

SpringWorks Therapeutics is led by a preeminent team of industry veterans, including:

Daniel S. Lynch, Executive Chairman, who has over 25 years of industry experience serving in management and board positions for a number of top-tier biotechnology and pharmaceutical companies.
Lara S. Sullivan, M.D., Founder and President, who brings more than two decades of senior leadership experience in biopharmaceuticals, healthcare and life sciences, most recently at Pfizer, where she had previously led the portfolio strategy for the company’s early stage pipeline and its Medical collaboration funding platform.
Stephen Squinto, Ph.D., Acting Head of Research & Development, Member of the Board of Directors, who brings over 25 years of biotechnology industry experience as both a scientist and senior executive.
Saqib Islam, JD, Chief Financial Officer and Chief Business Officer, who brings over 25 years in international business management with a focus on business development, global strategic planning and capital markets in the healthcare sector. Most recently, within the biotechnology industry, Saqib was an Executive Vice President and Chief Strategy Officer at Alexion Pharmaceuticals and Chief Business Officer at Moderna Therapeutics.
L. Mary Smith, Ph.D., Vice President, Clinical Research and Development, who brings more than 20 years of research and clinical development experience from both pharmaceutical and biotech companies. Most recently, Smith was the Vice President of Product Development at United Therapeutics and led the development and approval of Unituxin for high-risk neuroblastoma, a rare pediatric cancer.
In addition to Lynch, Sullivan and Squinto, SpringWorks Therapeutics has appointed the following seasoned directors to the board:

Carl L. Gordon, Ph.D., CFA – Partner, OrbiMed
Peter Keen – Trustee, LifeArc
Freda Lewis-Hall, M.D., DFAPA – Executive Vice President and Chief Medical Officer, Pfizer
Deval Patrick – Managing Director, Bain Capital Double Impact
Jeffrey Schwartz – Managing Director, Bain Capital Life Sciences

10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

(Filing, 10-K, Palatin Technologies, 2017, SEP 25, 2017, View Source [SID1234520832])

Salubris Pharmaceuticals Invests $5 Million in GO Therapeutics

On September 25, 2017 GO Therapeutics reported that Salubris Pharmaceutical Limited has made a $5M investment in GO Therapeutics, a Cambridge, MA-based company exploiting new advances in glycoproteomics to develop novel, multimodal first-in-class cancer therapeutics against intractable targets (Press release, GO Therapeutics, OCT 25, 2017, View Source [SID1234521293]).

The generation of solid tumor targeting domains that are specific to cancer cells remains a significant challenge in the drug development industry. GO Therapeutics’ targeting platforms and technologies open a novel class of tumor-specific antigens that promise to realize the full potential of antibody-drug conjugates, bi-specific T-cell engagers, and immune-based cell therapies.

"This investment from Salubris will accelerate the development of our pre-clinical antibody programs, and help support the discovery of new antibodies against prioritized targets of interest. Additionally, Salubris offers future downstream market access to China, which is expected to become a significant consumer of innovative cancer therapies," said Constantine Theodoropulos, Chief Executive Officer, GO Therapeutics.

"Novel, cancer-specific targets are imperative to widening the therapeutic window for powerful, cutting-edge cancer therapeutic modalities such as T-cell engagement and ADCs that offer tremendous promise for patients around the world. GO Therapeutics has the opportunity to generate significant value by opening an exciting new class of cancer-specific targets that will underpin transformative cancer therapies," said Sam Murphy, VP and Head of International Business Development for Salubris, who will join GO Therapeutics’ board.

Salubris’ strategic investment extends the company’s expansion to the U.S. and evolution towards leading-edge innovations in oncology and cardiovascular disease. Earlier this year Salubris announced the opening of the SalubrisBio research facility outside of Washington D.C. where the focus is building the company’s internal portfolio of NME biologic product candidates (www.salubrisbio.com).