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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that it will report its first quarter 2019 financial results on Tuesday, April 30, 2019 after the financial markets close (Press release, Vertex Pharmaceuticals, APR 12, 2019, View Source [SID1234535114]). The company will host a conference call and webcast at 4:30 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The conference call will be webcast live and a link to the webcast can be accessed through Vertex’s website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company’s website.

On April 12, 2019 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and, through our license to Santen Pharmaceutical Co. Ltd., wet age-related macular degeneration, reported that its Phase 3 TAPPAS trial evaluating TRC105 in combination with Votrient (pazopanib) in patients with advanced or metastatic angiosarcoma was terminated for futility based on the recommendation of the Independent Data Monitoring Committee (IDMC) following its review of interim unblinded safety and efficacy data from more than 120 patients enrolled in the trial at the time of the analysis (Press release, Tracon Pharmaceuticals, APR 12, 2019, View Source [SID1234535113]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

TRACON will work with investigators to appropriately conclude the study in a manner consistent with the best interests of each patient. Data from this study will be analyzed and submitted for presentation at an upcoming scientific congress.

"We are disappointed that TRC105 in combination with Votrient did not demonstrate clinically meaningful efficacy in patients with advanced or metastatic angiosarcoma," said Charles Theuer, M.D. Ph.D., President and CEO of TRACON. "Given these data, we will terminate further enrollment in company sponsored trials of TRC105 in oncology. We will continue to support our partner, Santen, in their development of DE-122 in wet AMD, where the anti-angiogenic and anti-fibrotic properties of endoglin inhibition may be more relevant than in oncology. We will also continue to develop our other drug candidates, including TRC253 in partnership with Janssen and TJ004309 (also known as TJ-D5) in partnership with I-Mab, and, intend to advance candidates within I-Mab’s broad bispecific pipeline into the clinic in the US as early as the beginning of next year. We will also continue our business development efforts to source additional innovative products to fortify our pipeline through risk share and cost share arrangements. On a financial note, as a result of the expected savings to be generated from terminating TRACON sponsored trials of TRC105, we anticipate our current cash runway will now extend into the third quarter of 2020."

Investor Conference Call

The Company will hold a conference call today at 8:30 a.m. EDT / 5:30 a.m. PDT to provide further details on the TAPPAS trial and TRACON’s clinical stage pipeline. The dial-in numbers are (877) 407‑9039 for domestic callers and (201) 689-8470 for international callers. Please use passcode 13689814. A live webcast of the conference call will be available at View Source

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About the TAPPAS trial in Advanced or Metastatic Angiosarcoma

The TAPPAS trial was designed to compare treatment with TRC105 and Votrient to treatment with single agent Votrient in up to 340 patients with advanced or metastatic angiosarcoma. Patients were randomized in equal numbers and the primary endpoint was progression free survival by RECIST 1.1. Key secondary endpoints included objective response rate, overall survival, safety and tolerability.

About TRC105 (carotuximab)

TRC105, the oncology formulation of carotuximab, is a novel, clinical stage antibody to endoglin, a protein overexpressed on proliferating endothelial cells that is essential for angiogenesis, the process of new blood vessel formation. TRC105 has received orphan designation for the treatment of soft tissue sarcoma in both the US and EU. The ophthalmic formulation of TRC105, DE-122, is currently being studied in the randomized Phase 2 AVANTE trial in patients with wet AMD. For more information about the clinical trials, please visit TRACON’s website at www.traconpharma.com/clinical_trials.php.

About DE-122 (carotuximab)

DE-122, a novel ophthalmic formulation of carotuximab, is active in preclinical choroidal neovascularization (CNV) models and expected to enhance the effect of approved VEGF inhibitors used to treat wet AMD. DE-122 is being investigated in the Phase 2 randomized AVANTE trial assessing the efficacy and safety of intravitreal injections in combination with Lucentis (ranibizumab) compared to Lucentis monotherapy in patients with wet AMD.