On January 14, 2020 Idera Pharmaceuticals, Inc. (Nasdaq: IDRA), or Idera or the Company, reported an update on its tilsotolimod clinical development program and providing a corporate outlook for 2020 (Press release, Idera Pharmaceuticals, JAN 14, 2020, View Source [SID1234553159]).

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"We made tremendous progress last year with the tilsotolimod clinical development program, and we are positioned well going into this pivotal year," stated Vincent Milano, Idera’s Chief Executive Officer. "Our ILLUMINATE-301 registrational trial of tilsotolimod in anti-PD-1 refractory metastatic melanoma is enrolling at a rate that dramatically exceeded our expectations. We expect to complete enrollment this quarter, setting up the possibility of data before the end of this year. We believe the rapid enrollment in this randomized trial is a testament to the critical unmet need for this patient group as well as to the growing appreciation from global investigators for both intratumoral administration and the mechanism of action of tilsotolimod."

Continued Milano, "We also will add to the overall body of clinical evidence for tilsotolimod with additional data readouts expected in the second quarter. Final analysis of ILLUMINATE-204 will include median overall survival (OS) for the first time, and ILLUMINATE-206 will provide safety and preliminary efficacy in microsatellite-stable colorectal cancer (MSS-CRC) as a triple combination of ipilimumab, nivolumab and tilsotolimod in this immunorefractory indication."

"Lastly, we recently entered into a financing agreement that provides financial resources for these critical catalysts and beyond. Overall, we are looking forward to 2020 and delivering on the goals we set for patients and our investors when we initiated the development of tilsotolimod."

ILLUMINATE (tilsotolimod) Clinical Development Program

ILLUMINATE 301 – Randomized phase 3 trial of tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with anti-PD-1 refractory metastatic melanoma:

•Approximately 100 sites active in 11 countries;
•Planned enrollment target of 454 patients;
•As of January 13, 2020, 427 patients enrolled, representing 94% enrollment; and
•Targeting completion of enrollment during Q1 2020; and data in Q4 2020/Q1 2021.

ILLUMINATE 206 – Phase 2, open-label, multi-cohort, multi-center study to test the safety and effectiveness of tilsotolimod in combination with ipilimumab and nivolumab for the treatment of solid tumors:

•Trial initiated on September 30, 2019, leading with the MSS-CRC cohort;
•Initial safety run-in cohort of 10 patients with MSS-CRC fully enrolled; and
•Preliminary objective response rate (ORR) and safety data from this cohort expected in Q2 2020.

ILLUMINATE 204 – Phase 1/2 trial of tilsotolimod in combination with ipilimumab or pembrolizumab in patients with anti-PD-1 refractory metastatic melanoma:

•Completed enrollment with 52 patients (49 evaluable) at tilsotolimod 8 mg with ipilimumab in February 2019; and
•Final results from the ILLUMINATE 204 trial are expected to be released in Q2 2020;
oData to be presented include ORR, median OS, duration of response (DOR) and safety.

Upcoming Corporate Presentation

Idera Chief Executive Officer Vincent Milano will provide a corporate overview at the 38th Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2020 at 7:30 AM PT/ 10:30 AM ET. The conference is being held at the Westin St. Francis Hotel in San Francisco, CA. A copy of the Company’s J.P. Morgan corporate presentation will be posted on the Investors’ page of the Company’s corporate website on Wednesday January 15, 2020 prior to the opening of the U.S. markets.

Live audio webcast of Idera’s presentations will be accessible in the Investors section of Idera’s website at View Source Archived versions will also be available on the Company’s website after the event for 90 days.

About Tilsotolimod (IMO-2125)

Tilsotolimod is an investigational TLR 9 agonist that received Fast Track Designation from the U.S. Food and Drug Administration (FDA) in 2017 for the treatment of anti-PD-1 refractory melanoma, in combination with ipilimumab as well as orphan drug designation from the FDA for the treatment of melanoma Stages IIb to IV. It signals the immune system to create and activate cancer-fighting cells (T-cells) to target solid tumors. Currently approved immuno-oncology treatments, specifically check-point inhibitors, provide benefit for some patients, but these therapies are limited in patients whose immune responses are missing or weak. Intratumoral injections with tilsotolimod are designed to selectively enable the tumor-specific T-cells to recognize and attack cancers that remain elusive and unrecognized by the immune system exposed to checkpoint inhibitors alone, while limiting toxicity or impact on healthy cells in the body.

On January 14, 2020 BioLineRx Ltd. (NASDAQ/TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, reported that the European Commission (EC) has granted Orphan Drug Designation to its lead oncology candidate, Motixafortide (BL-8040), for the treatment of pancreatic cancer, based on a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) (Press release, BioLineRx, JAN 14, 2020, View Source [SID1234553158]). Last year, Motixafortide received Orphan Drug Designation for the treatment of Pancreatic Cancer from the US Food and Drug Administration (FDA).

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"The Orphan Drug status we received for Motixafortide, from both the US and European regulatory bodies, is of significant strategic importance for the development of our lead product for the treatment of pancreatic cancer, an extremely difficult to treat indication with a poor response to the currently available treatments," stated Philip Serlin, Chief Executive Officer of BioLineRx. "We recently reported very encouraging initial data from the triple combination arm of our ongoing Phase 2a COMBAT/KEYNOTE-202 study in second line metastatic pancreatic cancer patients, which served as the basis for this ODD, and we believe that this designation will maximize the potential to make this new treatment available for patients in the fastest way possible."

Motixafortide is currently being evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a collaboration agreement with Merck & Co., Inc., Kenilworth, N.J., USA (known as MSD outside the United States and Canada).

The EMA grants orphan medicinal product designation to investigational drugs intended to treat, prevent or diagnose a life-threatening or chronically debilitating disease affecting fewer than five in 10,000 people in the EU and for which no satisfactory treatment is available or, if such treatment exists, the medicine must be of significant benefit to those affected by the condition. Orphan medicinal product designation provides regulatory and financial incentives for companies to develop and market therapies, including ten years of market exclusivity, protocol assistance, fee reductions and EU-funded research.

About Motixafortide in Cancer Immunotherapy
Motixafortide is targeting CXCR4, a chemokine receptor and a well validated therapeutic target that is over-expressed in many human cancers including PDAC. CXCR4 plays a key role in tumor growth, invasion, angiogenesis, metastasis and therapeutic resistance, and CXCR4 overexpression has been shown to be correlated with poor prognosis.

Motixafortide is a short synthetic peptide used as a platform for cancer immunotherapy with unique features allowing it to function as a best-in-class antagonist of CXCR4. It shows high-affinity, long receptor occupancy and acts as an inverse agonist.

In a number of clinical and preclinical studies, Motixafortide has been shown to affect multiple modes of action in "cold" tumors, including immune cell trafficking, tumor infiltration by immune effector T cells, and reduction in immunosuppressive cells (such as MDSCs) within the tumor niche, turning "cold" tumors, such as pancreatic cancer, into "hot" (i.e., sensitizing them to immune checkpoint inhibitors and chemotherapy).

About Pancreatic Cancer
Pancreatic cancer has a low rate of early diagnosis and a poor prognosis. Its incidence rate in the US is estimated at 3.2% of new cancer cases. Each year, about 185,000 individuals globally are diagnosed with this condition, and an estimated 55,000 individuals were diagnosed with pancreatic cancer in the US during 2018. Symptoms are usually non-specific and as a result, pancreatic cancer is often not diagnosed until it reaches an advanced stage. Surgical resection does not offer adequate treatment since only 20% of patients have resectable tumors at the time of diagnosis. The overall five-year survival rate among all pancreatic cancer patients is 7-8%, which constitutes the highest mortality rate among solid tumor malignancies. The overall median survival is less than one year from diagnosis, highlighting the need for the development of new therapeutic options.

Despite advances in chemotherapeutics and immunotherapy, increases in median and overall survival rates in pancreatic cancer have been modest. Pancreatic cancer remains an area of unmet medical need, with no new approved therapies since the approval of nab-paclitaxel in combination with gemcitabine (Abraxane) for first-line treatment in 2013 and Onivyde in combination with fluorouracil and leucovorin for second-line treatment in 2015. The limited clinical benefits demonstrated by these existing standard treatment options reinforce the need for additional approaches.

On January 14, 2020 Ipsen (Euronext: IPN; ADR: IPSEY) reported the appointment of Dr. Steven Hildemann as Executive Vice President, Chief Medical Officer, Head of Global Medical Affairs and Pharmacovigilance effective March 1, 2020 (Press release, Ipsen, JAN 14, 2020, View Source [SID1234553156]). Based in Paris, France, Dr. Hildemann will report directly to Aymeric Le Chatelier, CEO, Ipsen and serve on the Executive Leadership Team .

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Aymeric Le Chatelier, CEO, Ipsen, commented, "As we advance as a leading global biopharmaceutical company focused on innovation and Specialty Care, it is my great pleasure to appoint Dr. Hildemann to lead Ipsen’s Global Medical Affairs, Patient Affairs and Pharmacovigilance organizations through patient-centric leadership, sound medical governance and business conduct. With over 20 years of service in the pharmaceutical industry and 10 years as a physician-scientist in academic medicine, he brings a wealth of experience in medical affairs, clinical operations and patient safety from major global biopharmaceutical companies. Dr. Hildemann will play a crucial role in delivering on our global medical and patient safety strategy, engaging in structured, ethical and high-quality dialogue with patients, external and internal stakeholders throughout the entire life-cycle of Ipsen’s portfolio."

Dr. Hildemann will act as a strategic partner to Ipsen’s scientific, clinical and business teams. He will provide a global medical strategy based on real value to patients and caregivers. In close partnership with R&D, Dr. Hildemann will lead the generation of post-launch clinical trial data by defining, prioritizing, optimizing or guiding studies in line with this strategy. As a member of the Executive Leadership team, he will actively contribute to the overall management and strategic leadership of the company.

Prior to joining Ipsen, Dr.Hildemann held leadership roles in science-based bioethics and built an innovative digital health startup in cancer care after serving for five years as Chief Medical Officer, Senior Vice President, Head of Global Medical Affairs and Global Patient Safety at Merck. He also held several strategic leadership positions with biopharmaceutical companies such as Pharmacia-Pfizer and Schering-Plough-MSD. Dr Hildemann is board certified in internal medicine and cardiology with broad clinical training across internal medicine including medical oncology, gastroenterology, rheumatology and pulmonary oncology at university hospitals in Munich, Germany. Throughout his career, he has engaged in part-time clinical practice, late-stage pharmaceutical research and medical teaching. Dr. Hildemann received his MD-PhD at the Albert Ludwig University of Freiburg, Germany, where he continues to serve as an adjunct Professor of Medicine.

On January 14, 2020 Masimo (NASDAQ: MASI), a global leader in noninvasive monitoring technologies, and NantHealth, Inc. (NASDAQ: NH), a next-generation, evidence-based, personalized healthcare company, reported the signing of a definitive agreement under which Masimo will acquire the Connected Care assets from NantHealth, Inc. for a $47.25 million upfront cash payment (Press release, NantHealth, JAN 14, 2020, View Source [SID1234553155]). NantHealth’s Connected Care solutions provide medical device interoperability to hospitals and health systems. The portfolio includes DCX device connectivity (formerly known as DeviceConX), VCX patient vitals software (formerly known as VitalsConX), HBox connectivity hub, and Shuttle interface cable.

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The acquisition supports Masimo’s goal to help hospitals improve the continuum of great care through hospital automation, connectivity, and innovative noninvasive monitoring technologies. The Connected Care franchise is an established leader in providing connectivity solutions to hospitals, enabling streamlined collection and storage of medical device data through a vendor-agnostic platform into the EHR or other clinical information systems.
"One of the strategic priorities for Masimo is, through our Hospital Automation solutions, to reduce clinician cognitive overload and reduce errors of omission. Through connectivity, predictive algorithms, and decision support, we hope to improve the continuum of great care. The connectivity assets we are acquiring are completely in line with our mission as they will help accelerate our internal growth initiatives in this area," said Joe Kiani, Founder, Chairman, and CEO of Masimo. "Not only does Connected Care immediately increase our customer footprint but also provides us with products which complement our current portfolio. As a result, we are very excited to welcome the Connected Care’s talented team to Masimo and look forward to realizing our vision together."

Patrick Soon-Shiong, MBBCh, MSc, FRCS (C), NantHealth Chief Executive Officer and Chairman of the Board said, "Our decision to sell the Connected Care business enables us to focus on accelerating growth for our NaviNet and Eviti SaaS solutions, our data and molecular analytics capabilities, and pursue other strategically aligned goals. We believe Masimo has the best connectivity solutions, and we are delighted to have found the right home for our Connected Care business and our committed team of employees. We are working with Masimo to ensure a smooth transition for our Connected Care customers."

The transaction is expected to close in the first quarter of 2020, subject to customary closing conditions. Masimo expects to fund the acquisition with existing cash on hand. The estimated financial impact of the transaction is included in Masimo’s full-year 2020 financial guidance.

On January 13, 2020 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported that program updates for its four drug candidates and ongoing clinical studies (Press release, Turning Point Therapeutics, JAN 13, 2020, View Source [SID1234564381]).

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"Following a transformational year in 2019, we are pleased with the progress in our ongoing clinical studies, our recent Fast Track designation for repotrectinib and nomination of our ALK inhibitor candidate for IND-enabling studies," said Athena Countouriotis, M.D., president and CEO.

"In addition, we have now dosed multiple patients with our RET/SRC-inhibitor, TPX-0046, who presented with solvent-front mutations and were previously treated with other investigational RET inhibitors. The emergence of RET solvent-front mutations is an indication of the need for a next-generation RET inhibitor given the limited treatment options for these patients."

PROGRAM UPDATES AND 2020 MILESTONES

Repotrectinib
Clinical study of repotrectinib continues to progress in the Phase 2 registrational portion of the TRIDENT-1 global study and Phase 1/2 study in pediatric patients. With sites activating globally in the TRIDENT-1 study, the company continues to anticipate reporting early interim data from initial patients in some of the registrational cohorts in the second half of the year.

The company announced today that repotrectinib has been granted Fast Track designation by the U.S. Food and Drug Administration for the treatment of ROS1-positive advanced non-small cell lung cancer (NSCLC) patients who have been previously treated with one prior line of platinum-based chemotherapy and one prior line of a ROS1 tyrosine kinase inhibitor (TKI). There are currently no approved targeted therapies for TKI-pretreated ROS1-positive NSCLC patients.

Following ongoing dialogue with health authorities, the company recently amended the TRIDENT-1 protocol for expansion cohorts 2 and 3 to clarify that ROS1-positive advanced NSCLC patients must have had one prior platinum-based chemotherapy regimen in addition to one prior TKI (cohort 2) or two prior TKIs (cohort 3). This amendment to cohort 2 is in line with the patient population granted fast track designation. Additionally, based on the potential future treatment paradigm, expansion cohort 4 has been amended to enroll only ROS1-positive advanced NSCLC patients with one prior TKI and no prior chemotherapy. This amendment is aimed at expanding enrollment in multiple ROS1-positive advanced NSCLC TKI-pretreated patient populations.

TPX-0022 – MET/CSF1R/SRC Inhibitor
Study of TPX-0022 continues to progress in a Phase 1 clinical trial in advanced solid tumor patients with MET genetic alterations. The company continues to anticipate reporting early interim data from initial patients during the second half of the year.

TPX-0046 – RET/SRC Inhibitor
Study of TPX-0046 continues to progress in a Phase 1/2 clinical trial, with early enrollment including multiple RET-positive patients with solvent-front mutations previously treated with other investigational RET inhibitors. In preclinical studies against proxy molecules for other investigational RET inhibitors, TPX-0046 showed comparable or stronger potency against wildtype and certain RET solvent-front mutations.

TPX-0131, A Next-Generation ALK Inhibitor
The company reported the advancement of TPX-0131, its next-generation ALK inhibitor candidate, into IND-enabling studies. TPX-0131 has been designed with a compact macrocyclic structure and in preclinical studies has been shown to potently inhibit wildtype ALK and numerous ALK mutations, in particular the clinically observed G1202R solvent-front mutation and G1202R/L1196M compound mutation. ALK-driven tumors are estimated to represent up to 7 percent of driver oncogenes in NSCLC and of patients who develop a resistance mutation, G1202R has been reported in approximately 42 percent.

2020 Milestones
Key milestones anticipated in 2020 include:

Additional TPX-0131 preclinical data during the first half of the year;
Present preclinical repotrectinib combination data;
Early interim data from initial patients in some of the registrational cohorts of the repotrectinib TRIDENT-1 Phase 2 study during the second half of the year;
Early interim data from initial patients treated with TPX-0022 during the second half of the year; and
Submitting the IND for TPX-0131 by early 2021.