On May 16, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported the presentation of updated clinical data for its potent and highly selective investigational medicines avapritinib and BLU-667 (Press release, Blueprint Medicines, MAY 16, 2019, View Source [SID1234536441]).

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At the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and 24th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), Blueprint Medicines plans to present:

Data from the expansion portion of the registration-enabling ARROW trial of BLU-667 in patients with RET-altered non-small cell lung cancer (NSCLC) and thyroid cancers
Data from the registration-enabling NAVIGATOR trial of avapritinib in patients with PDGFRA Exon 18 mutant and fourth-line gastrointestinal stromal tumors (GIST), which will be used for planned marketing applications in the United States and Europe
Data from the registration-enabling EXPLORER trial of avapritinib in patients with advanced systemic mastocytosis (SM)
"Our upcoming presentations highlight data supporting our plans for multiple marketing applications in the United States and Europe over the next 18 months and demonstrate continued progress on our ‘2020 Blueprint’ strategy," said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. "The datasets reflect the rapid development of avapritinib and BLU-667, including in four indications that have received Breakthrough Therapy Designation from the FDA. These presentations for avapritinib and BLU-667 highlight the breadth and speed of development of our portfolio, as well as our commitment to rapidly deliver new precision therapies to cancer patients with limited or no effective treatment options."

The accepted abstracts are listed below and are now available online on the ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) conference websites, respectively: View Source and View Source

2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting
May 31-June 4, 2019; Chicago, Illinois

Oral Presentation

Presentation Title: Clinical activity and tolerability of BLU-667, a highly potent and selective RET inhibitor, in patients (pts) with advanced RET-fusion+ non-small cell lung cancer (NSCLC)
Session Title: Lung Cancer—Non-Small Cell Metastatic
Session Date & Time: Monday, June 3, 2019 from 8:00 a.m. – 11:00 a.m. CT (9:00 a.m. – 12:00 p.m. ET)
Abstract Number: 9008

Poster Discussion Presentations

Presentation Title: Activity and tolerability of BLU-667, a highly potent and selective RET inhibitor, in patients with advanced RET-altered thyroid cancers
Session Title: Head and Neck Cancer
Poster Session Date & Time: Saturday, June 1, 2019 from 1:15 p.m. – 4:15 p.m. CT (2:15 p.m. – 5:15 p.m. ET)
Poster Discussion Session Date & Time: Saturday, June 1, 2019 from 4:30 – 6:00 p.m. CT (5:30 p.m. – 7:00 p.m. ET)
Abstract Number: 6018

Presentation Title: Clinical activity of avapritinib in ≥ fourth line (4L+) and PDGFRA Exon 18 gastrointestinal stromal tumors (GIST)
Session Title: Sarcoma
Poster Session Date & Time: Saturday, June 1, 2019 from 8:00 a.m. – 11:00 a.m. CT (9:00 a.m. – 12:00 p.m. ET)
Poster Discussion Session Date & Time: Saturday, June 1, 2019 from 3:00 p.m. – 4:30 p.m. CT (4:00 p.m. – 5:30 p.m. ET)
Abstract Number: 11022

24th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper)
June 13-16, 2019; Amsterdam, The Netherlands

Oral Presentation

Presentation Title: Avapritinib, a potent and selective inhibitor of KIT D816V, induces complete and durable responses in patients (pts) with advanced systemic mastocytosis (AdvSM)
Session Title: New Agents in MPN
Presentation Date & Time: Saturday, June 15, 2019 from 12:00 p.m. – 12:15 p.m. CEST (6:00 a.m. – 6:15 a.m. ET)
Abstract Number: S830

Investor Event and Webcast Information

Blueprint Medicines will host an investor event on Monday, June 3, 2019 beginning at 6:30 p.m. CT (7:30 p.m. ET) in Chicago to provide a portfolio update, including a review of updated clinical data from the ongoing ARROW trial of BLU-667 in patients with RET-altered cancers and the ongoing registration-enabling NAVIGATOR trial in patients with PDGFRA Exon 18 mutant and fourth-line GIST. The event will be webcast live and can be accessed under the "Investors & Media—Events & Presentations" section of Blueprint Medicines’ website at www.BlueprintMedicines.com. A replay of the webcast will be available approximately two hours after the event and will be available for 30 days following the event.

On May 16, 2019 Fusion Pharmaceuticals, a clinical-stage biopharmaceutical company developing targeted alpha-particle radiotherapeutics for treating cancer, reported that it will present a poster at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting, which is being held from May 31 to June 4, 2019, at the McCormick Place Convention Center in Chicago, Illinois (Press release, Fusion Pharmaceuticals, MAY 16, 2019, View Source [SID1234536410]). The abstract is available on the ASCO (Free ASCO Whitepaper) meeting website at View Source

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The Company will present a poster highlighting a Phase 1 study evaluating its lead candidate [225Ac]-FPI-1434, a radioimmunoconjugate consisting of a humanized monoclonal antibody that binds to the external domain of IGF-1R, a bifunctional chelate, proprietary linker, and the alpha-emitting radionuclide actinium-225 (Ac-225). The indium-111 analog, [111In]-FPI-1547, which contains the identical antibody and bifunctional chelate is used for patient selection and quantification of IGF-1R expressing targets prior to therapy.

The Phase 1 study has a primary objective of evaluating the safety and tolerability of [111In]-FPI-1547 Injection and [225Ac]-FPI-1434 Injection in patients with advanced refractory solid tumors and to determine the maximum tolerated dose of a single [225Ac]-FPI-1434 Injection. The study is currently enrolling patients in Canada.

Details of the poster presentation are below:

Abstract Title: A Phase I study of [225Ac]-FPI-1434 radioimmunotherapy in patients with IGF-1R expressing solid tumors.

Abstract Number for Publication: TPS3152

Session Title: Developmental Therapeutics and Tumor Biology (Nonimmuno)

Date: Saturday, June 1, 2019, 8:00 AM-11:00 AM CDT

Location: McCormick Place, Hall A

On May 16, 2019 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that a total of four abstracts have been accepted for presentation at the 24th Congress of European Hematology Association (EHA) (Free EHA Whitepaper) meeting (June 13-16, Amsterdam, Netherlands) (Press release, Oncopeptides, MAY 16, 2019, View Source [SID1234536426]).

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Updated data from the Phase 2 HORIZON study evaluating melflufen for the treatment of relapsed/refractory multiple myeloma (RRMM) patients with no or limited remaining treatment options will be presented as an oral presentation by Professor Paul G. Richardson. Three poster presentations, one of which is from the ongoing Phase 1/2 ANCHOR study, will also be presented.

The data in the abstracts regarding the ANCHOR and HORIZON studies is from February 6th 2019 and consequently similar to the data already presented at ASH (Free ASH Whitepaper) 2018. Additional data supporting melflufen’s activity have been generated during the spring of 2019 and will be presented in full at EHA (Free EHA Whitepaper).

CEO
"With the abstracts accepted at EHA (Free EHA Whitepaper), we have now already more than doubled the amount of abstracts accepted for presentations in 2019 compared to the full year of 2018. This underlines the increase in clinical experience that we are generating with melflufen for the treatment of patients with RRMM, and that melflufen continues to show strong activity alone or in combination with other myeloma drugs in this patient population. As always, the abstract book contains quite old data from the ongoing studies and we are excited about the opportunity to present the full data-sets at EHA (Free EHA Whitepaper)", said Jakob Lindberg CEO of Oncopeptides.

Upcoming presentations at EHA (Free EHA Whitepaper)
The HORIZON data will be presented as an oral presentation by Professor Paul G. Richardson, in the session "Novel strategies in multiple myeloma" on June 16 at 08.45 (CET) in the auditorium. Paul G. Richardson, MD, is Professor of Medicine at Harvard Medical School and Clinical Program Leader, Director of Clinical Research at the Jerome Lipper Multiple Myeloma Center Dana-Farber Cancer Institute in Boston, Massachusetts, USA.

The posters will made public presented on Friday, June 14 at 09.30 and presented between 17.30 – 19.00 (CET).

Oncopeptides will also host a symposium at EHA (Free EHA Whitepaper) on June 13th between 18.45 – 20.45 (CET) at the Amsterdam RAI Hall 3B, under the title "Challenging the Treatment Paradigm in Multiple Myeloma".

The four abstracts can be found on the company webpage under:
www.oncopeptides.com / Investors & Media / Presentations / EHA (Free EHA Whitepaper) Abstracts 2019

HORIZON (OP-106): Updated efficacy and safety of melflufen in relapsed / refractory multiple myeloma (RRMM) patients refractory to daratumumab (DARA) and/or pomalidomide (POM)
Topic: Myeloma and other monoclonal gammopathies – Clinical
Session Title: Novel strategies in multiple myeloma
Abstract Code – S 1605

ANCHOR (OP-104): A phase 1/2 study update of melflufen and dexamethasone plus bortezomib or daratumumab in in relapsed / refractory multiple myeloma patients refractory to an IMiD or a proteasome inhibitor
Topic: Myeloma and other monoclonal gammopathies – Clinical
Session Title: Myeloma and other monoclonal gammopathies – Clinical
Abstract Code – 608

O-12-M1: An evaluation of time to next treatment in melflufen and dexamethasone treated patients with relapsed / refractory multiple myeloma
Topic: Myeloma and other monoclonal gammopathies – Clinical
Session Title: Myeloma and other monoclonal gammopathies – Clinical
Abstract Code – PF 628

Tolerability of treatments for relapsed / refractory multiple myeloma: A systematic review
Topic: Quality of life, palliative & supportive care, ethics and health economics
Session Title: Quality of life, palliative & supportive care, ethics and health economics
Abstract Code – PF 726

For further information, please contact:
Jakob Lindberg, CEO of Oncopeptides
E-mail: [email protected]
Telephone: +46 (0)8 615 20 40

Rein Piir, Head of Investor Relations at Oncopeptides
E-mail: [email protected]
Cell phone: +46 (0)70 853 72 92

This information was submitted for publication at 15.00 CET, May 16, 2019.

About melflufen
Melflufen is a lipophilic peptide-conjugated alkylator that rapidly delivers a highly cytotoxic payload into myeloma cells through peptidase activity. It belongs to the novel class Peptidase Enhanced Cytotoxics (PEnC), which is a family of lipophilic peptides that exhibit increased activity via peptidase cleavage and have the potential to treat many cancers. Peptidases play a key role in protein homeostasis and feature in cellular processes such as cell-cycle progression and programmed cell death. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately cleaved by peptidases to deliver an entrapped hydrophilic alkylator payload. In vitro, melflufen is 50-fold more potent in myeloma cells than the alkylator payload itself due to the peptidase cleavage, and induces irreversible DNA damage and apoptosis. Melflufen displays cytotoxic activity against myeloma cell lines resistant to other treatments, including alkylators, and has also demonstrated inhibition of DNA repair induction and angiogenesis in preclinical studies.

On May 16, 2019 Zymeworks Inc. (NYSE/TSX: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported that GSK has expanded its 2016 licensing and collaboration agreement with Zymeworks for the research, development, and commercialization of bispecific antibodies across multiple disease areas (Press release, Zymeworks, MAY 16, 2019, View Source [SID1234536442]). Under the expanded agreement, GSK will now have access to Zymeworks’ unique heavy-light chain pairing technology, part of its proprietary Azymetric platform. Zymeworks’ Azymetric platform enables the development of bispecific and multifunctional therapeutics while maintaining the characteristics of naturally-occurring human antibodies.

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"We are pleased to see GSK broaden their technology license and believe that it reflects their increased commitment to develop differentiated bispecific therapeutics," said Ali Tehrani, Ph.D., President and CEO of Zymeworks. "As part of this expansion, GSK expects to use our Azymetric technology to develop bispecifics for the treatment of infectious diseases, which highlights the utility of our platform beyond traditional indications like oncology and inflammatory disease."

Under the updated terms of the expanded agreement, GSK will have the option to develop and commercialize bispecific drugs across different disease areas and Zymeworks will be eligible to receive increased preclinical, development and commercial milestone payments. If all six programs are developed and commercialized, the new potential value of the collaboration would be up to US$1.1 billion. Additionally, Zymeworks is eligible to receive increased tiered royalties on worldwide sales.

About the Azymetric Platform

The Azymetric platform enables the transformation of monospecific antibodies into bispecific antibodies, giving the antibodies the ability to simultaneously bind two different targets. Azymetric bispecific technology enables the development of multifunctional biotherapeutics that can block multiple signaling pathways, recruit immune cells to tumors, enhance receptor clustering degradation, and increase tumor-specific targeting. These features are intended to enhance efficacy while reducing toxicities and the potential for drug resistance. Azymetric bispecifics have been engineered to retain the desirable drug-like qualities of naturally occurring antibodies, including low immunogenicity, long half-life, and high stability. In addition, they are compatible with standard manufacturing processes with high yields and purity, potentially significantly reducing drug development costs and timelines.

On May 16, 2019 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported positive data from the Phase I/II STARTRK-NG study, evaluating the investigational medicine entrectinib in children and adolescents with recurrent or refractory solid tumours with and without neurotrophic tyrosine receptor kinase (NTRK), ROS1 or anaplastic lymphoma kinase (ALK) gene fusions (Press release, Hoffmann-La Roche, MAY 16, 2019, View Source [SID1234536388]). The study showed entrectinib shrank tumours (objective response rate; ORR) in all children and adolescents who had NTRK, ROS1 or ALK fusion-positive solid tumours (11 of 11 patients), including two patients achieving a complete response.1 Of the 11 patients, five patients with primary high-grade tumours in the central nervous system (CNS) had an objective response, including one patient with a complete response.1 The safety profile of entrectinib was consistent with that seen in previous analyses.1 Data will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago on Sunday, 2 June, 2019, from 8:00 – 8:12 am CDT (Abstract 10009), and was part of yesterday’s official ASCO (Free ASCO Whitepaper) presscast.

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"We are encouraged by the results we have seen with entrectinib in children with paediatric and adolescent cancers, including those with tumours in the brain," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "The STARTRK-NG study underscores the importance of combining comprehensive genomic profiling with targeted therapies and supports our approach to providing people with personalised medicines developed specifically for their type of cancer."

Additional data for entrectinib across different tumour types and patient populations will also be presented at ASCO (Free ASCO Whitepaper), highlighting the company’s unique approach to personalised healthcare through advances in targeted therapies, diagnostics and data analytics:

Initial results from an integrated analysis of the Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA-372-001 trials, evaluating the efficacy of entrectinib in adults with solid tumours and CNS metastases, will be presented on Saturday, 1 June, 2019, in a poster session from 3:00 – 4:30 pm CDT (Abstract 3017).
Results from a Real World Data study, evaluating time-to-treatment discontinuation and progression-free survival as endpoints for comparative efficacy analysis of clinical trials of entrectinib and crizotinib for the treatment of people with ROS1-positive non-small cell lung cancer (NSCLC), will be presented during a poster session on Sunday, 2 June, 2019, from 8:00 – 11:00 am CDT (Abstract 9070).
The FDA recently granted Priority Review for entrectinib for both the treatment of paediatric and adult patients with NTRK fusion-positive, locally advanced or metastatic solid tumours who have either progressed following prior therapies or as an initial therapy when there are no acceptable standard therapies, and for the treatment of people with metastatic ROS1-positive NSCLC.2 These NDAs are based on results from the integrated analysis of the Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA-372-001 trials, and data from the STARTRK-NG study. The FDA is expected to make a decision on approval by 18 August, 2019.2

About the STARTRK-NG study
STARTRK-NG is a Phase I/II open-label dose-escalation and expansion study evaluating the safety and efficacy of entrectinib in children and adolescent patients with no curative first-line treatment option, recurrent or refractory extracranial solid tumours or primary CNS tumours, with or without NTRK, ROS1 or ALK fusions.1 Response, assessed by Investigator, was classified as complete response (CR), partial response (PR), stable disease (SD) or progressive disease (PD) using Response Assessment in Neuro-Oncology (RANO) for CNS tumours, Response Evaluation Criteria in Solid Tumors (RECIST), and Curie score (CS) for NBL.1 The study enrolled 29 children and adolescents aged 4.9 months through to 20 years (median age of 7 years) who had recurrent or refractory solid tumours, and 28 were evaluated for response.1 Of the 28 children and adolescents evaluated, 11 children were identified to have tumours with NTRK, ROS1 or ALK fusions and one with ALK F1174L-mutated neuroblastoma (NBL).1 A summary of the results are included below.

Complete responses were observed in 2 patients with tumours harbouring NTRK and ALK fusions: 1 with an NTRK fusion-positive primary CNS tumour and 1 with an ALK fusion-positive inflammatory myofibroblastic tumour. Another complete response was observed in 1 neuroblastoma patient with an ALK F1174L mutation.1
Partial responses were observed in 9 patients, 3 unconfirmed at the time of the clinical cut-off date, across NTRK, ROS1 and ALK fusion-positive primary CNS (n=4) and extracranial (n=5) solid tumours.1
Median duration of therapy for confirmed fusion-positive responders was 10.51 months (3.8 to 17.7 months), and median time to response was 1.89 months (1 to 1.9 months).1
The safety profile of entrectinib was consistent with that seen in previous analyses. Treatment-related adverse events were most frequently National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events Grade 1 or 2, leading to discontinuation in 6.9% of patients.1
About NTRK fusion-positive cancer
Neurotrophic tyrosine receptor kinase (NTRK) fusion-positive cancer occurs when the NTRK1/2/3 genes fuse with other genes, resulting in altered TRK proteins (TRKA/TRKB/TRKC) that can activate signaling pathways involved in proliferation of certain types of cancer. NTRK gene fusions are tumour-agnostic, meaning they are present in tumours irrespective of site of origin. These fusions have been identified in a broad range of solid tumour types, including breast, cholangiocarcinoma, colorectal, gynaecological, neuroendocrine, non-small cell lung, salivary gland, pancreatic, sarcoma and thyroid cancers.3

About entrectinib
Entrectinib (RXDX-101) is an investigational, oral medicine in development for the treatment of locally advanced or metastatic solid tumours that harbour NTRK1/2/3 or ROS1 gene fusions. It is a selective tyrosine kinase inhibitor designed to inhibit the kinase activity of the TRK A/B/C and ROS1 proteins, whose activating fusions drive proliferation in certain types of cancer.4,5 Entrectinib can block ROS1 and NTRK kinase activity and may result in the death of cancer cells with ROS1 or NTRK gene fusions.4,5 Entrectinib is being investigated across a range of solid tumour types, including breast, cholangiocarcinoma, colorectal, gynaecological, neuroendocrine, non-small cell lung, salivary gland, pancreatic, sarcoma and thyroid cancers.2,3

Entrectinib has been granted Breakthrough Therapy Designation (BTD) by the FDA; Priority Medicines (PRIME) designation by the European Medicines Agency (EMA); and Sakigake designation by the Japanese health authorities for the treatment of NTRK fusion-positive, locally advanced or metastatic solid tumours in adult and paediatric patients who have either progressed following prior therapies or have no acceptable standard therapies.2