On March 27, 2019 Crown Bioscience, a global drug discovery and development services company, providing translational platforms to advance oncology, inflammation and autoimmune, cardiovascular and metabolic disease research reported that parent company, JSR Corporation, has entered into a strategic partnership with Hubrecht Organoid Technology (HUB) (Press release, JSR, MAR 27, 2019, View Source [SID1234553813]).

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The agreement provides CrownBio, a wholly owned subsidiary of JSR Corporation, with an exclusive license to provide preclinical oncology drug development and validation services using HUB Organoid Technology, including access to HUB’s highly characterized tumor organoid biobank. CrownBio and HUB will also launch a collaborative research & development program to accelerate further development of Organoid Technology, while CrownBio will establish a new operations center in Utrecht, Netherlands.

HUB’s adult stem cell-derived organoids, developed by Prof. Hans Clevers at the Hubrecht Institute in Utrecht, are generated using highly standardized, proprietary culture methods. The Organoids faithfully recapitulate tumors in patients and are extensively characterized by pathology, genomics, and sensitivity to known and experimental drugs.

"This agreement is a pivotal moment in our growth and the evolution of the company," said Dr. Jean-Pierre Wery, CEO of CrownBio. "This partnership positions CrownBio at the forefront of global preclinical research by creating an unparalleled translational platform to accelerate drug development and validation."

Building on a global leadership in Patient-Derived Xenografts (PDX), CrownBio will offer comprehensive pharmacology and translational services based on HUB Organoid Technology, establishing a unique platform that incorporates in vitro screening of Patient-Derived Organoids (PDO) and PDX derived organoids (PDXO), matched to downstream in vivo models.

"We are delighted with the increased access to HUB Organoid Technology for researchers worldwide through CrownBio’s global reach and preclinical service expertise," said Dr. Robert Vries, Executive Director of HUB. "Our partnership with CrownBio equips the drug development community with powerful new translational tools to guide the creation of novel and improved treatments for cancer."

On March 27, 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported participation in the Jefferies 6th Annual IO Cell Therapy Summit taking place in Boston (Press release, Bellicum Pharmaceuticals, MAR 27, 2019, View Source [SID1234534667]). Bellicum’s President & CEO Rick Fair and Head of Research, Aaron Foster, Ph.D., will provide an overview of the company and its development programs during a fireside chat on April 5, 2019 at 9:10 a.m. EDT.

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On March 27, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported plans to expedite development of avapritinib, BLU-667 and BLU-782 based on recent clinical progress and interactions with regulatory authorities (Press release, Blueprint Medicines, MAR 27, 2019, View Source [SID1234534710]).

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"The updates we are announcing today for avapritinib, BLU-667 and BLU-782 represent the acceleration of multiple clinical-stage programs, including planned marketing applications for avapritinib and BLU-667, and highlight our commitment to executing a portfolio-based strategy," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In addition, as we prepare to submit marketing applications for avapritinib for the treatment of PDGFRA-driven and fourth-line GIST in the United States in the second quarter and Europe in the third quarter, we are rapidly building global commercial capabilities to enable us to deliver this important new medicine, if approved, to patients in need."

Summary of Accelerated Regulatory Submission Plans and Recent Clinical Progress

Avapritinib: Gastrointestinal Stromal Tumors (GIST)

Blueprint Medicines now plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for avapritinib for the treatment of both PDGFRα D842V mutant GIST and fourth-line GIST in the third quarter of 2019.
Avapritinib: Advanced Systemic Mastocytosis (SM)

Blueprint Medicines now plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for avapritinib for the treatment of advanced SM in the first quarter of 2020, subject to continuing discussions with the FDA under its breakthrough therapy program to determine the required clinical data for an NDA submission.
BLU-667: RET-Altered Cancers

Top-line interim data from the Phase 1 ARROW clinical trial of BLU-667 for patients with RET-fusion non-small cell lung cancer (NSCLC) and RET-mutant medullary thyroid cancer (MTC) who were response evaluable and treated at the recommended Phase 2 dose of 400 mg once daily, as of November 14, 2018 with follow-up through a data cutoff date of March 1, 2019, showed the following:
In 34 patients with RET-fusion NSCLC previously treated with platinum-based chemotherapy, the overall response rate (ORR) was 62 percent.(1)
In 16 patients with RET-mutant MTC previously treated with the approved multi-kinase inhibitors cabozantinib and/or vandetanib, the ORR was 63 percent.(2)
BLU-667 was generally well-tolerated, and most adverse events (AEs) reported by investigators were mild or moderate (Grade 1 or 2). Across all doses and treatment cohorts (n=217), only eight patients discontinued treatment with BLU-667 due to treatment-related AEs, with one Grade 5 AE (pneumonia/lung infection) determined by the investigator to be possibly related to BLU-667.
Detailed clinical safety and efficacy data from the ARROW trial in RET-fusion NSCLC patients and RET-mutant MTC patients have been submitted for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2019.
The enrollment target has been reached and patient screening is now closed for the registration-enabling ARROW trial cohort for patients with previously treated RET-fusion NSCLC. Blueprint Medicines anticipates reaching the enrollment target for the registration-enabling ARROW trial cohort for patients with previously treated MTC in the second quarter of 2019.
Based on the early achievement of the enrollment target for the RET-fusion NSCLC cohort, Blueprint Medicines now plans to submit an NDA to the FDA for BLU-667 for the treatment of patients with NSCLC previously treated with platinum-based chemotherapy in the first quarter of 2020. Blueprint Medicines continues to expect to submit an NDA to the FDA for BLU-667 for the treatment of patients with RET-mutant MTC previously treated with an approved multi-kinase inhibitor in the first half of 2020.
BLU-782: Fibrodysplasia Ossificans Progressiva (FOP)

Based on the progress of the ongoing Phase 1 clinical trial in healthy volunteers and input from clinical experts, Blueprint Medicines now plans to initiate a Phase 2a clinical trial of BLU-782 in patients with FOP in the fourth quarter of 2019.
Footnotes:
(1) 95 percent confidence interval (CI): 44-78 percent; five responses pending confirmation.
(2) 95 percent CI: 35-85 percent; three responses pending confirmation.

On March 27, 2019 Syndax Pharmaceuticals, Inc. ("Syndax" or the "Company") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it has entered into an agreement with Biotechnology Value Fund, L.P. ("BVF") and other leading life sciences investors for the purchase of 4,366,666 shares of common stock at a purchase price of $6.00 per share, representing a premium of 30% to the share price as of market close on Tuesday, March 26, as well as warrants to purchase up to 2,183,331 shares of common stock at an exercise price of $12.00 per share, and warrants to purchase up to 2,183,335 shares of common stock at an exercise price of $18.00 per share (Press release, Syndax, MAR 27, 2019, View Source [SID1234534668]).

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Syndax anticipates aggregate gross proceeds from the offering will be approximately $26.2 million (excluding any proceeds to be received upon exercise of the warrants). Closing of the transaction is expected to occur on or about March 29, 2019.

The securities described above are being offered by Syndax pursuant to a shelf registration statement previously filed with the Securities and Exchange Commission (the "SEC"), which the SEC declared effective on April 20, 2017. A final prospectus supplement related to the offering will be filed with the SEC, and will be available on the SEC’s website located at View Source

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus.

On March 27, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported that it has commenced an underwritten public offering of $300,000,000 in shares of its common stock (Press release, Blueprint Medicines, MAR 27, 2019, View Source [SID1234534711]). In addition, Blueprint Medicines expects to grant the underwriters a 30-day option to purchase up to an additional $45,000,000 in shares of its common stock in connection with the public offering. All shares of common stock will be offered by Blueprint Medicines.

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Blueprint Medicines expects to use the net proceeds of the offering to further build its commercial infrastructure and operations in support of one or more anticipated commercial launches of its drug candidates, including potential commercial launches of avapritinib in the United States and Europe, subject to regulatory approval; to fund clinical trials for avapritinib in gastrointestinal stromal tumors (GIST) and systemic mastocytosis (SM), including its planned registration-enabling Phase 3 COMPASS-2L clinical trial for second-line GIST and its ongoing registration-enabling Phase 2 PIONEER clinical trial for indolent and smoldering SM, as well as future indication expansion clinical trials; to fund clinical trials for BLU-667 in RET-driven cancers, including its planned Phase 3 clinical trial for BLU-667 in first-line RET-altered non-small cell lung cancer (NSCLC) and its planned Phase 2 clinical trial for BLU-667 in combination with osimertinib in treatment-resistant, EGFR-mutant NSCLC harboring an acquired RET alteration; to fund a planned Phase 2a clinical trial for BLU-782 in patients with fibrodysplasia ossificans progressiva; to fund manufacturing costs for ongoing and anticipated drug development efforts for its most advanced drug candidates, including a potential commercial launch of avapritinib; and the balance, if any, to fund additional discovery research efforts, its other ongoing and planned clinical trials, working capital requirements and other general corporate purposes.

Goldman Sachs & Co. LLC and Cowen and Company, LLC are acting as joint book-running managers for the offering. Guggenheim Securities, LLC and Wedbush Securities Inc. are acting as co-lead managers for the offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed or as to the actual size or terms of the offering.

A registration statement on Form S-3 (File No. 333-216573) relating to these securities has been previously filed with the Securities and Exchange Commission (SEC) and has become effective. This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

The offering will be made only by means of a prospectus. A copy of the prospectus supplement relating to the offering will be filed with the SEC and may be obtained, when available, from Goldman Sachs & Co. LLC by mail at Prospectus Department, 200 West Street, New York, NY 10282, by telephone at (866) 471-2526, by fax at (212) 902-9316, or by email at [email protected], or from Cowen and Company, LLC, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, by telephone at (631) 274-2806, or by fax at (631) 254-7140