Deciphera Pharmaceuticals Initiates Pivotal Phase 3 Clinical Study of Ripretinib (DCC-2618) in Second-line Patients with Gastrointestinal Stromal Tumors (“INTRIGUE” Study)

On December 20, 2018 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported that it has initiated a pivotal Phase 3 clinical study, "INTRIGUE", to evaluate the efficacy and tolerability of ripretinib (DCC-2618), a broad-spectrum KIT and PDGFRα inhibitor, compared to sunitinib in second-line gastrointestinal stromal tumor (GIST) patients (Press release, Deciphera Pharmaceuticals, DEC 20, 2018, View Source [SID1234532194]).

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"We are extremely pleased that the INTRIGUE Phase 3 study of ripretinib is now open to enroll second-line GIST patients, regardless of their mutational status, who have progressed on, or are intolerant to front-line therapy with imatinib," said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. "If successful, we believe this Phase 3 study could serve as the basis for a regulatory submission for broad use in all second-line GIST patients."

"INTRIGUE is the second pivotal Phase 3 study of ripretinib that Deciphera has initiated in less than one year. As recently announced, we expect to report top-line data from our first Phase 3 clinical study, INVICTUS, in fourth-line and fourth-line-plus GIST patients in mid-2019," continued Dr. Taylor.

"While imatinib is an effective treatment for most patients with early-stage GIST, in almost all patients the disease will eventually progress due to the development of secondary drug resistance mutations," said Professor Michael Heinrich, MD, Cell and Developmental Biology, OHSU Knight Cancer Institute. "A well-tolerated therapy with broad coverage and efficacy across the spectrum of KIT and PDGFRα mutations would represent a much-needed improvement over currently approved therapies for patients with GIST."

About the INTRIGUE Phase 3 Study
The INTRIGUE Phase 3 clinical study is an interventional, randomized, global, multicenter, open-label study to evaluate the safety, tolerability and efficacy of ripretinib compared to sunitinib in patients with GIST previously treated with imatinib. This study was designed to support regulatory approvals in second-line GIST patients in the United States, Europe and other major markets. Patients will be randomized 1:1 to either 150 mg of ripretinib once daily or 50 mg of sunitinib once daily for four weeks followed by two weeks without sunitinib. The primary efficacy endpoint is median progression-free survival (mPFS) as determined by independent radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST). Secondary endpoints as determined by independent radiologic review using modified RECIST include Objective Response Rate (ORR) and Overall Survival (OS). See www.clinicaltrials.gov for further information (NCT03673501).

About the INVICTUS Phase 3 Study
The INVICTUS Phase 3 clinical study is a randomized, double‑blind, placebo-controlled, global, multicenter trial to evaluate the safety, tolerability, and efficacy of ripretinib compared to placebo in patients with advanced GIST whose previous therapies have included at least imatinib, sunitinib, and regorafenib. This fully enrolled study was designed to support regulatory approvals in fourth-line and fourth-line-plus GIST patients in the United States, Europe and other major markets. Patients were randomized 2:1 to either 150 mg of ripretinib or placebo once daily. The primary efficacy endpoint is median progression-free survival (mPFS) as determined by independent radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST). Secondary endpoints as determined by independent radiologic review using modified RECIST include Objective Response Rate (ORR), Time to Tumor Progression (TTP), and Overall Survival (OS). See www.clinicaltrials.gov for further information (NCT03353753).

About Ripretinib
Ripretinib (DCC-2618) is an investigational KIT and PDGFRα kinase switch control inhibitor in clinical development for the treatment of KIT and/or PDGFRα-driven cancers, including gastrointestinal stromal tumors, or GIST, systemic mastocytosis, or SM, and other cancers. Ripretinib was specifically designed to improve the treatment of GIST patients by inhibiting a broad spectrum of mutations in KIT and PDGFRα. Ripretinib is a KIT and PDGFRα inhibitor that blocks initiating and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18, involved in GIST as well as the primary D816V exon 17 mutation involved in SM. Ripretinib also inhibits primary PDGFRα mutations in exons 12, 14 and 18, including the exon 18 D842V mutation, involved in a subset of GIST.

Chi-Med Announces Progress in Savolitinib Lung Cancer Program and Update on Kidney Cancer Strategy

On December 20, 2018 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported a full update on the savolitinib development programs in both lung cancer and kidney cancer (Press release, Hutchison China MediTech, DEC 20, 2018, View Source [SID1234532210]).

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"This important progress on the savolitinib / Tagrisso combination in lung cancer is a product of the close collaboration that Chi-Med has with AstraZeneca," commented Christian Hogg, Chief Executive Officer of Chi-Med. He added, "We are also making rapid progress in China on MET Exon 14 deletion lung cancer where we now expect to complete enrollment of our registration intent study in 2019. In addition, we continue to believe that there is a role for a selective MET inhibitor in kidney cancer, and will use the deep body of clinical and epidemiological data that we have amassed to adapt our savolitinib strategy to the fast changing treatment landscape."

Mene Pangalos, Executive Vice-President of AstraZeneca’s Innovative Medicines and Early Development Biotech Unit, commented that, "Recent data in lung cancer has further confirmed the importance of MET as both a resistance mechanism to EGFR inhibitors and as a target in its own right. Savolitinib has the potential to provide benefit for cancer patients who have MET-driven tumors, including those with the Exon 14 deletion. The initiation of the SAVANNAH study is an important step in our goal of bringing novel targeted therapies forward which selectively inhibit key oncogenic drivers. I am delighted to see the successful collaboration with Chi-Med deliver more important data that has the potential to redefine the way in which MET-driven disease is treated."

LUNG CANCER DEVELOPMENT UPDATE:

TAGRISSO – Tagrisso (osimertinib). Since its first approval in 2015, Tagrisso has been established as a new standard of care in the treatment of Epidermal Growth Factor Receptor mutation ("EGFRm") non-small cell lung cancer ("NSCLC"), and has now been approved in over 80 countries. AstraZeneca recently announced that Tagrisso would be added to China’s national drug reimbursement program as second-line treatment for NSCLC patients in 2019;

Understanding the mechanism of acquired resistance following EGFR tyrosine kinase inhibitor ("TKI") therapy is a key clinical question to inform the next treatment choice;
At the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress ("ESMO") 2018, AstraZeneca presented the first results on the acquired resistance spectrum detected in patient plasma after progression in the first-line (FLAURA) and second-line T790M (AURA3) Phase III studies. In both studies, MET-amplification was among the most frequent mechanisms of acquired resistance to Tagrisso. 15% of patients in the FLAURA study, and 19% of patients in AURA3, had evidence of MET amplification as a mechanism of acquired resistance in their plasma samples following progression on Tagrisso; and
The frequency of MET amplification is expected to be higher than the above plasma rates when assessed in tumor tissue due to the different diagnostic tests that are available.
TATTON – The combination of Tagrisso / savolitinib is being explored as a treatment option for MET+ EGFRm NSCLC.

Initial data from the TATTON (NCT02143466) study assessing the safety and preliminary efficacy of the Tagrisso/savolitinib combination was presented at the World Conference on Lung Cancer ("WCLC") in 2017;
Confirmed partial responses ("PRs") were seen in 10/33 (Objective Response Rate ("ORR") 33%) of patients with MET+ EGFRm NSCLC (local testing) who had been previously treated with a third generation EGFR TKIs, primarily Tagrisso. The majority of these patients had received at least four lines of prior therapy;
Confirmed PRs were seen in 14/23 (ORR 61%) of patients with MET+ T790M- EGFRm NSCLC (local testing) who had been previously treated with a first or second generation EGFR TKI. The majority of these patients had received one prior line of therapy; and
Patients continue to be enrolled to the TATTON study and the clinical data has continued to mature, consistent with the WCLC 2017 presentation. A presentation of the complete TATTON dataset is planned for a scientific conference in 2019.
SAVANNAH – Based on these encouraging results, Chi-Med and AstraZeneca have initiated SAVANNAH, a global Phase II study of Tagrisso / savolitinib combination in patients with MET+ EGFRm NSCLC who have progressed following Tagrisso.

SAVANNAH is a single arm study designed to enroll approximately 170 patients with MET+ EGFRm NSCLC, with at least 50% having progressed following first-line Tagrisso therapy;
The primary data completion is anticipated in 2021; and
SAVANNAH will be closely aligned to benefit from the molecular profiling in the ORCHARD study announced by AstraZeneca during ESMO (Free ESMO Whitepaper) 2018. ORCHARD is an open-label, multi-center, multi-drug Phase II platform trial in patients with advanced EGFRm NSCLC whose disease has progressed on first-line therapy with Tagrisso. Platform studies such as ORCHARD that offer targeted treatment options for all patients are typically expected to have high enrollment rates. By aligning the two studies, patients identified by ORCHARD to have MET+ EGFRm NSCLC and meet relevant inclusion criteria will be prioritized for the SAVANNAH study.
Further opportunities identified in EGFRm NSCLC:

Separately, a Phase I study combining AstraZeneca’s first generation EGFR TKI Iressa with savolitinib has been completed in China and the results will be reported at a scientific meeting in 2019; and
Chi-Med and AstraZeneca are reviewing the data from both the TATTON and Iressa / savolitinib combination studies, and anticipate announcing plans for further studies during 2019.
MET Exon 14 deletion first-line NSCLC:
MET Exon 14 deletion first-line NSCLC is present in 2-3% of NSCLC patients. The China Phase II study of savolitinib monotherapy is currently enrolling in NSCLC patients with MET Exon 14 deletion who have failed prior systemic therapy, or are unwilling or unable to receive chemotherapy:

Following early 2018 regulatory authority dialogue and a subsequent protocol amendment, our current China Phase II study, if successful, will be sufficient to support a New Drug Application ("NDA") submission in China;
Enrollment is expected to complete during mid-late 2019;
Preliminary data expected to be published at a major scientific conference during 2019; and
Subject to positive Phase II outcome, we intend to submit China NDA in 2020.
We believe MET Exon 14 deletion NSCLC has the potential to be the first savolitinib approval world-wide.

KIDNEY CANCER DEVELOPMENT UPDATE:

Kidney cancer treatment landscape. The treatment landscape of renal cell carcinoma ("RCC") has evolved rapidly in recent years. Monotherapy treatment with second generation vascular endothelial growth factor receptor ("VEGFR") TKIs as well as programmed cell death protein-1 ("PD-1") monoclonal antibodies ("mAb") are improving patient outcomes with ORRs increasing to >30% and in first-line clear cell RCC ("ccRCC"). Lately, VEGFR TKI / PD-1 mAb combinations have been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) driven by their ORRs of >70% in first-line ccRCC patients.

Papillary renal cell carcinoma ("PRCC") continues to be a difficult sub-type of RCC to treat and has been shown to harbor MET-driven disease in between 40-70% of patients. As a result, while PRCC does favor the introduction of a MET TKI, enthusiasm for VEGFR TKI / PD-1 mAb monotherapy and combinations in PRCC is high.

Molecular Epidemiology ("MES") update – We have recently completed the largest MES study conducted on PRCC patients, which was aimed at developing a more comprehensive understanding of the role of MET-driven disease in PRCC. Archived tissue samples from over 200 PRCC patients in the US, Canada, France and Asia (Korea) were screened using our companion diagnostic to identify MET-driven disease. Historical medical records from these patients were then used to determine if MET-driven disease is predictive of worse outcome for patients treated with sunitinib, in terms of progression free survival ("PFS"), time to treatment failure (TTF) and overall survival (OS). Preliminary MES findings are as follows:

MES analysis was conducted on patients who were diagnosed before December 31, 2015, so the impact of the recent evolution in treatment landscape (e.g. PD-1) was not observed;
Sutent (sunitinib) (VEGFR TKI) in first-line PRCC was effective, providing similar tumor control across MET positive and negative patients. Sutent was heavily used as first-line PRCC therapy with minimal usage in second-line and above setting.
Incidence of MET positive PRCC patients was lower than previous scientific publications and the savolitinib US Phase II study; and
Full MES findings will be presented at a major scientific conference in 2019.
CALYPSO study in PRCC – An independently sponsored Phase II study of savolitinib monotherapy and in combination with Imfinzi (durvalumab), AstraZeneca’s anti Programmed death-ligand 1 ("PD-L1") mAb, is underway in both PRCC and ccRCC patients (NCT02819596; U.K./Spain; sponsor: Queen Mary’s University, London).

CALYPSO explores combination (savolitinib / Imfinzi) use in an all-comer PRCC population (both MET-driven and MET-independent; PD-L1 expression agnostic); and
Preliminary data from this study is planned for presentation at a future scientific conference.
SAVOIR Phase III study – SAVOIR (NCT03091192) is a global Phase III registration study of savolitinib versus Sutent in MET-driven metastatic PRCC patients. The study was initiated in June 2017 with a primary endpoint for efficacy of PFS. The SAVOIR protocol was designed, with regulatory endorsement, to include an adjustment at the time of MES readout to enable rebalancing the ratio of first-line vs. second-line and above MET positive PRCC patients.

MES implications on SAVOIR study:

Likelihood of SAVOIR success in first-line MET positive PRCC is now considered as low given outcome of MES;
Rebalancing SAVOIR to second-line and above patients is operationally not practical given minimal use of Sutent beyond first-line as well as lower than expected incidence of MET positive patients; and
The outcome of a molecular epidemiology study, along with recent novel therapy approvals, is leading to a change in savolitinib kidney cancer strategy.
As a result, the savolitinib registration strategy for PRCC is being reassessed, to take into account these findings as well as the rapidly changing RCC treatment landscape. Enrollment in the SAVOIR study has been suspended.

About Savolitinib

Savolitinib is a potential first-in-class inhibitor of c-MET, an enzyme which has been shown to function abnormally in many types of solid tumors. Chi-Med designed savolitinib to be a potent and highly selective oral inhibitor, which, through chemical structure modification, addresses human metabolite-related renal toxicity, the primary issue that halted development of several other selective c-MET inhibitors. In clinical studies to date, involving over 700 patients, savolitinib has shown promising signs of clinical efficacy in patients with c-MET gene alterations in PRCC, NSCLC, colorectal cancer (CRC) and gastric cancer with an acceptable safety profile. Chi-Med is currently testing savolitinib in partnership with AstraZeneca in Phase Ib/II studies, in multiple solid tumor indications, both as a monotherapy and in combinations.

GT BIOPHARMA, INC. TO PRESENT AT BIOTECH SHOWCASE™ 2019

On December 20, 2018 GT Biopharma, Inc. (OTCQB: GTBP and Euronext Paris GTBP.PA) ("GT Biopharma" or the "Company"), an immuno-oncology biotechnology company focused on innovative treatments based on the Company’s proprietary NK-engager and Bispecific Antibody Drug Conjugate platforms, reported that Raymond W Urbanski, M.D., Ph.D., Chief Executive Officer and Chairman, will present at Biotech Showcase 2019 on Tuesday, January 8, 2019 at 11:30 AM PST in San Francisco, CA (Press release, GT Biopharma , DEC 20, 2018, View Source [SID1234539517]).

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As part of his presentation, Dr. Urbanski will provide a corporate update and discuss GT Biopharma’s pipeline of immuno-oncology products based off the Company’s proprietary Tri-specific Killer Engager (TriKE), Tetra-specific Killer Engager (TetraKE) and bi-specific Antibody Drug Conjugate (ADC) technology platforms.

The Company’s most advanced bi-specific ADC in development, GTB-1550, targets CD19+ and/or CD22+ hematological malignancies and is currently in the Phase 2 component of a Phase 1/2 Non-Hodgkin’s Lymphoma (NHL)/Acute Lymphocytic Leukemia (ALL) trial which is an open-label, investigator-led study. GT Biopharma expects to announce topline results from the Phase 2a trial of GTB-1550 in the first quarter of 2019.

Additionally, the Company recently announced its Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) is now open and it is authorized to initiate a first-in-human Phase 1 study with GTB-3550 (formerly OXS-3550), its first-in-class (TriKE), for the treatment of acute myelogenous leukemia (AML), myelodysplatic syndrome (MDS) and mastocytosis. The study, which is expected to commence in the first half of 2019, will be led by Principal Investigator, Sarah A. Cooley, MD, MS, Associate Professor, Division of Hematology, Oncology and Transplantation at Masonic Cancer Center, University of Minnesota. The Company believes that GTB-3550 could serve as a relatively safe, cost-effective, and easy-to-use therapy for refractory/relapsed AML, high-risk MDS and advanced systemic mastocytosis and could also be combined with chemotherapy and/or other agents as frontline therapy thus targeting a much larger patient population.

In addition to the presentation, Dr. Urbanski will also be available to participate in one-on-one meetings with qualified members of the investor community who are registered to attend the conference. For more information about the conference, please click here to visit the conference website.

A live audio webcast of the presentation will be available on the Events page of the Investors section of the Company’s website (www.gtbiopharma.com). A webcast replay will be accessible for 90 days following the live presentation.

Replimune to Present at the 37th Annual J.P.Morgan Healthcare Conference

On December 20, 2018 Replimune Group Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immunotherapies derived from its Immulytic platform, reported that Robert Coffin, Ph.D., Chief Executive Officer and Director of Replimune, will present at the 37th Annual J.P.Morgan Healthcare Conference on Wednesday, January 9, 2019 at 8:00 AM PT at the Westin St. Francis Hotel in San Francisco, CA (Press release, Replimune, DEC 20, 2018, View Source [SID1234532180]).

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A simultaneous webcast will be available in the Investors section of Replimune’s website at www.replimune.com. A replay will be available for 30 days following the conference.

Relay Therapeutics Secures $400 Million in Series C Financing

On December 20, 2018 Relay Therapeutics, a new breed of company at the intersection of computation and biotechnology, reported the successful completion of a $400 million Series C financing (Press release, Relay Therapeutics, DEC 20, 2018, View Source [SID1234532195]).

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Relay Therapeutics combines unprecedented computational power with leading-edge experimental approaches across the fields of structural biology, biophysics, chemistry and biology. The integration of these disparate disciplines, tools and cultures enables Relay Therapeutics to overcome challenges that prior attempts have failed to solve and to design therapies against validated but previously intractable targets. Relay Therapeutics’ initial discovery programs in cancer have led to the development of highly selective inhibitors of disease-causing proteins in genomically defined patient populations.

The Series C financing was led by the SoftBank Vision Fund and included participation from additional new investors including Foresite Capital, Perceptive Advisors and Tavistock Group. Existing investors GV, Casdin Capital, BVF Partners, EcoR1 Capital, Alexandria Venture Investments, and an affiliate of D.E. Shaw Research also participated in the round.

Proceeds from this financing are anticipated to be used to accelerate the implementation of Relay Therapeutics’ long-term strategy. It will support the expansion of the company’s discovery efforts, advance existing programs into the clinic and bolster its broad platform and diverse team.

"We are at a unique moment in the evolution of drug discovery where we can realize the promise of integrating ever more powerful experimental and computational discovery tools to tackle previously undruggable protein targets. The success of our early programs validates the potential of our platform to create breakthrough therapies that address a broad range of diseases," said Sanjiv Patel, M.D., President and Chief Executive Officer of Relay Therapeutics.

"A financing of this magnitude allows Relay Therapeutics to significantly scale and advance both its platform and its pipeline. We are thrilled by the strong support of our investors for our mission, vision and strategy," said Alexis Borisy, Chairman of Relay Therapeutics and Partner at Third Rock Ventures.

"We are proud to support Relay Therapeutics’ world-class team expand its efforts across a larger number of programs and therapeutic areas to develop tomorrow’s life-saving treatments," said Deep Nishar, Senior Managing Partner at SoftBank Investment Advisers, investment adviser to the SoftBank Vision Fund.