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

On March 26, 2019 Akrevia Therapeutics, a privately held biopharmaceutical company focused on developing highly-potent, tumor-targeted immuno-oncology therapeutics, and City of Hope, an independent research and treatment center for cancer, diabetes and other life-threatening diseases, reported that they have entered into an exclusive licensing agreement that will allow Akrevia to utilize innovative technology to engineer potent immune-activating cytokines that can be selectively activated in the tumor microenvironment (Press release, Akrevia Therapeutics, MAR 26, 2019, View Source [SID1234554019]).

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The tumor microenvironment has distinct physiological characteristics that can be exploited to help develop tools to deliver therapies that are more precise and potent than existing treatments. The new technology, licensed from the lab of John Williams, Ph.D., at City of Hope, is suited to a broad spectrum of cytokines – molecules for which current tumor activation approaches are not applicable. This novel Switchblade allows inactivated proteins to "flick open" at the point of attack, releasing potent anti-tumor agents precisely where they are needed, thus maximizing efficacy and minimizing the peripheral side effects that limit current cytokine therapies. This new platform can be applied to other highly potent biologic architectures, including diverse cytokine family members, chemokines and agonistic immune modulators which to date have proved intractable in clinical development. This new technology will augment Akrevia’s existing Aklusion platform technologies, and augments the existing technology previously licensed from City of Hope.

"Cytokines have immense promise as highly potent cancer immunotherapeutics, with well-understood biology – but their extreme potency and lack of tumor targeting have thwarted attempts to exploit this clinically," said Tim Clackson, Ph.D., president and executive vice president, R&D, Akrevia Therapeutics. "The new technology licensed from City of Hope will substantially complement our current platform portfolio, providing the ability to target tumor activation of the full range of potent, immune-stimulating molecules. Akrevia will use the newly licensed technology to aggressively advance our pipeline and build towards the next generation of targeted cancer treatments."

Under the agreement, Akrevia gains exclusive commercial rights for application of the technology to the development of proprietary products. City of Hope, internationally recognized for pioneering technology that enabled the creation of numerous breakthrough cancer drugs, developed this technology to help investigators and researchers shield their selected antibodies for targeted therapies and to improve drug delivery. The nonprofit institution is working to improve precision medicine for all patients.

"At City of Hope, our research teams focus on rapidly developing technologies that can quickly move from laboratory research to clinical treatment," said Williams, professor of Molecular Medicine, City of Hope. "Our team has developed a novel approach to unlock the potential of multiple important immuno-oncology mechanisms to deliver potent, targeted agents to patients. We look forward to our continued collaboration with Akrevia to harness the therapeutic potential of this technology with the goal of improving treatment for patients."