On April 12, 2019 GlycoMimetics, Inc. (NASDAQ: GLYC) reported plans to initiate a clinical trial of GMI-1359 in breast cancer patients whose tumors have spread to bone (Press release, GlycoMimetics, APR 12, 2019, View Source [SID1234535115]). GMI-1359 is a dual function antagonist that targets both E-selectin and CXCR4, both of which are involved in tumor trafficking and metastatic spread. The trial will evaluate dose escalation as well as safety and pharmacodynamic markers in these patients.

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The trial’s Co-Principal Investigators are Kelly Marcom, M.D., and Dorothy Sipkins, M.D., Ph.D., both of the Duke Cancer Institute. Dr. Sipkins has previously published on the key roles of both E-selectin and CXCR4 in the trafficking of metastatic cancer cells and of their establishment as micro-metastases in bone.1

"Dr. Sipkins’ work suggests that both E-selectin and CXCR4 mediate key mechanisms that promote progression and migration of circulating cancer cells to protective niches. Importantly, her work reveals a potentially exciting approach to molecularly excise disseminated breast cancer cells with GMI-1359, which was rationally designed to inhibit both of these targets," said John Magnani, Ph.D., Senior Vice President and Chief Scientific Officer at GlycoMimetics.

"Our preclinical research in mice suggests that targeting E-selectin and CXCR4 with a single agent may potentially improve treatment of patients at risk of metastasis to bone, or whose tumors might have already spread," said Dr. Sipkins.

"If ultimately shown safe and effective in clinical trials, this agent could represent a potentially novel approach to treating metastatic cancer, and we’re pleased to begin exploring the use of this investigational therapy in individuals with metastatic cancer," added Dr. Marcom.

GlycoMimetics expects to initiate this trial in 2H 2019.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers.

On April 12, 2019 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, reported the pricing of its previously announced underwritten public offering of 5,670,000 shares of its common stock and, in lieu of common stock, pre-funded warrants to purchase 2,130,000 shares of common stock, and accompanying Class A warrants to purchase 3,900,000 shares of its common stock at a price to the public of $11.00 per share and accompanying Class A warrant (or $10.999 per pre-funded warrant and accompanying Class A warrant) (Press release, X4 Pharmaceuticals, APR 12, 2019, View Source [SID1234535116]). X4’s gross proceeds from this offering are expected to be approximately $85.8 million, before deducting underwriting discounts and estimated offering expenses. All of the securities in the offering are being sold by X4. The offering is expected to close on or about April 16, 2019, subject to customary closing conditions.

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Cowen and Stifel are acting as joint book-running managers and representatives of the underwriters for the offering. Canaccord Genuity is acting as lead manager of the offering.

The offering is being made only by means of a written prospectus and related prospectus supplement forming part of a shelf registration statement on Form S-3 that was filed with the Securities and Exchange Commission (SEC) on January 25, 2019 and declared effective by the SEC on February 19, 2019. The preliminary prospectus supplement and accompanying prospectus relating to and describing the terms of the offering were filed with the SEC on April 12, 2019. The final prospectus supplement and accompanying prospectus will be filed with the SEC and will be available at the SEC’s website located at www.sec.gov, copies of which may be obtained, when available, from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, or by telephone at 631-274-2806, and from Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, or by telephone at 415-364-2720 or by email at [email protected].

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 other jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction.

On April 12, 2019 Astex Pharmaceuticals (Astex), a pharmaceutical company dedicated to the discovery and development of novel small molecule therapeutics for oncology and diseases of the central nervous system, reported that it has received a milestone payment from Janssen Pharmaceutica N.V. (Janssen) (Press release, Astex Pharmaceuticals, APR 12, 2019, View Source [SID1234535117]). This follows the United States Food and Drug Administration’s (FDA) accelerated review and approval of a Janssen New Drug Application (NDA) for BALVERSA (erdafitinib) for the treatment of adults with locally advanced or metastatic urothelial carcinoma (mUC) which has susceptible fibroblast growth factor receptor (FGFR)3 or FGFR2 genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.

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BALVERSA (erdafitinib) is a once-daily, oral pan-FGFR inhibitor that was discovered by Astex and Janssen as part of a 2008 exclusive worldwide collaboration and licence agreement to identify novel, small molecule inhibitors of FGFR kinase, including for the treatment of cancer. Under the ongoing collaboration, Janssen is responsible for the clinical development and commercialisation of all products, including erdafitinib. Astex is eligible to receive further milestone payments on additional regulatory filings and approvals in the US and Europe and for additional indications, as well as tiered double-digit royalty payments on annual sales of erdafitinib.

BALVERSA (erdafitinib) received Breakthrough Therapy Designation from the FDA for the treatment of patients with metastatic urothelial cancer in March 2018 and Janssen announced submission of an NDA seeking its approval to the US FDA in September 2018. Urothelial cancer, particularly of the bladder, is the sixth most common type of cancer in the USA.

Before entering into its exclusive worldwide collaboration and licence agreement with Janssen, Astex performed pioneering work on FGFR with the Cancer Research UK Drug Discovery Group at the Newcastle Cancer Centre, Northern Institute for Cancer Research, Newcastle University, UK, underlining the quality of UK science and strengths in academic-biotech collaboration.

Harren Jhoti Ph.D., President and CEO of Astex, UK, said, "We are delighted with the approval of BALVERSA and congratulate our valued colleagues at Janssen for their determination and excellent work in the discovery and development of this new medicine. We also congratulate our academic collaborators at Newcastle University for their contribution to our pioneering early collaboration on FGFR that aided in its discovery. Astex continues to strive to discover new medicines for cancer patients and is very proud that BALVERSA is the second of our Pharma-partnered products to have been approved in the last two years.ˮ

On April 12, 2019 QIAGEN N.V. (NYSE:QGEN; Frankfurt Prime Standard:QIA) reported the U.S. launch of its novel therascreen FGFR RGQ RT-PCR Kit (therascreen FGFR Kit) as a companion diagnostic to help guide the use of the newly approved FGFR kinase inhibitor, BALVERSA (erdafitinib), developed by Janssen Biotech, Inc. (Janssen) (Press release, Qiagen, APR 12, 2019, View Source [SID1234535118]). The test will aid in identifying patients with urothelial cancer whose tumors have certain alterations in the fibroblast growth factor receptor 3 (FGFR3) gene. The U.S. Food and Drug Administration co-approved the new test with BALVERSA, as announced today by Janssen.

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Urothelial cancer begins in tissues lining the bladder and other genitourinary organs and is the sixth most common type of cancer in the United States. An estimated 15,000 patients a year in the United States are diagnosed with advanced or metastatic urothelial cancer, but current treatment options are limited so the prognosis is poor. A percentage of urothelial carcinoma tumors have certain FGFR alterations which are thought to be key drivers of tumor growth. Detection of these alterations utilizing the companion diagnostic will help identify patients eligible for treatment with BALVERSA. The therascreen FGFR Kit will run on QIAGEN’s Rotor-Gene Q MDx, a member of the modular QIAsymphony family of automation solutions and leverages a worldwide exclusive license for in-vitro diagnostic use of FGFR3:TACC3 fusions from Columbia University.

"We are very excited about the launch of the new therascreen FGFR Kit, the first companion diagnostic test to obtain FDA approval for detection of FGFR gene alterations to guide therapy in any cancer indication. Using our test to help guide treatment decisions in urothelial cancer will address a high unmet medical need among patients," said Thierry Bernard, Senior Vice President and Head of QIAGEN’s Molecular Diagnostics Business Area. "The new therascreen FGFR Kit and significant testing capacities at leading laboratories will be available through QIAGEN’s Day-One Lab Readiness program to accelerate the availability of innovations in Precision Medicine."

An updated list of laboratories offering the new therascreen FGFR Kit under QIAGEN’s Day-One Lab Readiness program is available on QIAGEN’s FGFR lab finder web page (www.qiagen.com/fgfr-lab-finder).

QIAGEN is a pioneer in Precision Medicine and the global leader in collaborations with pharmaceutical and biotechnology companies to co-develop companion diagnostics, which detect clinically relevant genetic abnormalities to provide insights that guide clinical decision-making on the use of drugs in diseases such as cancer. QIAGEN has an unmatched depth and breadth of technologies from NGS to PCR for companion diagnostic development and is currently working under master collaboration agreements with more than 25 companies to develop and commercialize companion diagnostic tests for their drug candidates. The therascreen FGFR Kit co-approval with BALVERSA marks the sixth FDA approval of a therapy with a QIAGEN companion diagnostic assay. For more information on QIAGEN’s companion diagnostics please visit www.qiagen.com.

On April 12, 2019 Wayshine Biopharm reported that it has received IND approval from FDA about its innovative experimental medicine WSD0922, a First-in-Class CNS penetrable EGFR/EGFRvIII inhibitor for the treatment of Glioblastoma, Anaplastic Astrocytoma and cancers with CNS metastasis patients (Press release, Wayshine Biopharm, APR 12, 2019, View Source [SID1234537799]). The WSD0922 program is under the sponsorship of Wayshine and the clinical trial, entitled "A Study to Evaluate Safety, Tolerability, Pharmacokinetics and Anti-tumor Activity of WSD0922-FU" will be performed at Minnesota, Arizona and Florida, the three campuses of Mayo Clinic. Glioblastoma is the most aggressive malignant primary brain tumor in adults and is nearly always fatal. Treatment options for GBM are scarce and recurrence is inevitable. After recurrence, no standard treatment has been established. WSD0922 could be a potential game-changing program in treating GBM and other brain cancers.

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"We are very pleased that the FDA has approved the application of IND, which is a significant development milestone in advancing our Portfolio," said Wei Zhong, Ph.D., CEO and Founder of Wayshine Biopharm. "WSD0922 is the first and only BBB penetrable EGFR/EGFRvIII inhibitor under development. The discovery of WSD0922 truly reflects our innovation and commitments and the clinical potential has been recognized and endorsed by the FDA, for this substantial unmet medical need. "

"If only one target can be picked for brain cancer, it’s EGFR/EGFRvIII." Comments from KOLs in Neuro-Oncology field.

"We make difference! WSD0922 is discovered and developed by Chinese scientists and the IND approval in US is a very important progress in its development. With the help from FDA and Mayo Clinic, we can definitely move this program forward quickly to benefit patients who are suffering Glioblastoma and the data generated from the study in cancer patients will significantly contribute to the brain cancer field." commented by Jinqiang Zhang, Ph.D., VP and co-founder at Wayshine.