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CStone receives CTA approval in China to start Phase I trial for FGFR4 inhibitor BLU-554 (CS3008)

On January 28, 2019 CStone Pharmaceuticals (CStone) reported that the National Medical Products Administration (NMPA) recently approved the clinical trial application (CTA) to start a Phase I clinical trial in China for BLU-554 (CS3008), an inhibitor of fibroblast growth factor receptor 4 (FGFR4) discovered by the company’s partner Blueprint Medicines (Press release, CStone Pharmaceauticals, JAN 28, 2019, View Source [SID1234532931]). CStone has exclusive rights to develop and commercialize BLU-554 in Mainland China, Hong Kong, Macau and Taiwan.

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The trial is part of a global Phase I trial for BLU-554 in patients with advanced hepatocellular carcinoma (HCC) not previously treated with a tyrosine kinase inhibitor (TKI). The aim is to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of BLU-554 in this patient subset. CStone and Blueprint Medicines expect to enroll the first patient in the Phase I clinical trial in China soon.

HCC is the most common form of liver cancer. In China, HCC is the third most significant cause of cancer-related death, with approximately 466,000 new diagnoses and 422,000 deaths caused by the disease each year. It is estimated that approximately 30% of patients with HCC have tumors with aberrantly activated FGFR4 signaling. The majority of Chinese HCC patients (55%) are not diagnosed until the advanced stages of the disease. Currently, treatment options for advanced HCC patients remain limited.

BLU-554 is a potent and highly selective small-molecule inhibitor of FGFR4. Blueprint Medicines announced preliminary Phase I clinical data of BLU-554 at the European Society of Clinical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress, as of a data cutoff date of August 18, 2017. In heavily pre-treated patients with FGFR4-driven HCC (n=38), the data showed an objective response in 6 patients (16%; 1 complete response and 1 partial response pending confirmation), while disease control was achieved by 26 patients (68%), and 18 patients (49%) had a reduced tumor burden. In 5 patients who had not previously received TKI therapy, preliminary evidence of prolonged disease control was observed. BLU-554 was well-tolerated and most adverse events reported by investigators were Grade 1 or 2.

Dr. Frank Jiang, CEO and chairman of CStone, commented: "Compared with current treatments, BLU-554 has produced encouraging data in terms of tolerability and disease control rates. We also plan to conduct a Phase I trial of BLU-554 in combination with CS1001 for the treatment of advanced HCC patients in China in the second half of 2019. Our hope is to make more effective treatment options available to HCC sufferers."

About BLU-554

BLU-554 is an oral, highly selective and irreversible inhibitor of FGFR4 developed by Blueprint Medicines, with a precise selectivity for FGFR4 that spares the paralog kinases FGFR1, FGFR2 and FGFR3. Blueprint Medicines is developing BLU-554 for the treatment of HCC caused by FGFR4 activation, estimated to account for approximately 30% of patients with HCC tumors. BLU-554 has been granted orphan drug status by the U.S. Food and Drug Administration.

In June 2018, CStone and Blueprint Medicines entered into a license and collaboration agreement in which Blueprint Medicines granted CStone exclusive rights to develop and commercialize BLU-554 in Greater China. Blueprint Medicines retains development and commercial rights for BLU-554 in the rest of the world.

NMPA Accepts New Drug Application for IBI-305, a Biosimilar Product Candidate of Bevacizumab (Avastin®)

On January 28, 2019 Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality innovative medicines for the treatment of oncology, autoimmune and other major diseases, reported that the National Medical Products Administration (NMPA) has accepted its new drug application (NDA) for a biosimilar product candidate of bevacizumab (Avastin) (Press release, Innovent Biologics, JAN 28, 2019, View Source [SID1234532930]). This NDA by Innovent is the third that has been accepted by the NMPA following Tyvyt (sintilimab injection, marketing approval granted on December 24, 2018) and IBI-303 (a biosimilar product candidate of adalimumab).

IBI-305 is a recombinant humanized anti-vascular endothelial growth factor (anti-VEGF) monoclonal antibody independently developed by Innovent for the treatment of non-small cell lung cancer (NSCLC), colorectal cancer and other malignant tumors. Bevacizumab (marketed under the trade name Avastin in China) has been approved globally for the treatment of multiple types of malignant tumors including NSCLC and has a favorable safety and efficacy profile. Despite the huge demand for effective cancer therapies in China, the adoption rate of bevacizumab (Avastin) is relatively low due to its rather low affordability. Innovent’s biosimilar product candidate of bevacizumab, IBI-305, is expected to offer a high-quality and affordable alternative to patients in China.

The NDA is based on clinical data generated from two clinical studies, namely a Phase 3 comparative study of efficacy and safety in advanced non-squamous NSCLC patients and a pharmacokinetics (PK) study in healthy subjects. Both studies directly compare IBI-305 to bevacizumab and have met their pre-defined primary endpoints.

"Two clinical studies have been conducted with bevacizumab injection as the control drug. Based on the high-quality clinical data, the NDA application of IBI-305 has been accepted by NMPA. We believe that the high quality bevacizumab biosimilar will improve drug availability and benefit more patients and their families," said Professor Li Zhang from Cancer Hospital of Sun Yat-sen University.

"We are delighted that IBI-305 has become our third NDA successfully accepted by the NMPA. At the present time we have thirteen products in clinical development stage and four products in Phase 3 clinical trials. Tyvyt, our first commercial product, was recently approved by NMPA and our team will continue to deliver high quality biopharmaceutical drugs from our rich pipeline to benefit more ordinary people in China and globally," said Michael Yu, Founder, Chief Executive Officer and Chairman of Innovent.

About NSCLC and Colorectal Cancer

The report on the development of malignant tumors in China (2017), compiled by China Anti-Cancer Association, points out that the incidence of malignant tumors in China accounts for about 22% of the world’s incidences, and the number of cases of malignant tumors ranks first in the world. Moreover the incidence and mortality of lung cancer in China accounted for 35.78% and 37.56% of the world, respectively. China has become the country with the largest incidence of lung cancer in the world and lung cancer is the fastest growing malignant tumor in China. NSCLC, accounts for 80% of all lung cancer cases. The incidence of advanced colorectal cancer is also relatively high and continues rising.

Basic and clinical research has shown that angiogenesis plays an important role in pathogenesis of malignant tumors. The good efficacy and safety of the branded bevacizumab in seven solid tumors including NSCLC and colorectal cancer have been confirmed in multiple clinical studies. Branded bevacizumab was approved for the treatment of NSCLC and colorectal cancer by NMPA in China.

About IBI-305 (bevacizumab biosimilar)

IBI-305 has been designed as a biosimilar to bevacizumab and is a recombinant humanized anti-VEGF monoclonal antibody for injection. Vascular endothelial growth factor (VEGF) is an important factor in angiogenesis that is highly expressed by the endothelial cells in most human tumors. An anti-VEGF antibody binds VEGF selectively with high affinity and blocks its binding to VEGF receptors on the surface of vascular endothelial cells, thereby inhibiting signaling pathways such as PI3K-Akt/PKB and Ras-Raf-MEK-ERK. Bevacizumab produces anti-tumor effects by inhibiting the growth, proliferation and migration of vascular endothelial cells, blocking angiogenesis, reducing vascular permeability, blocking the blood supply to tumor tissues, inhibiting the proliferation and metastasis of tumor cells and inducing apoptosis in tumor cells.

Pulmatrix, Inc. Announces Pricing of Public Offering of Common Stock

On January 28, 2019 Pulmatrix, Inc. ("Pulmatrix," the "Company," "we," "our" or "us") (NASDAQ: PULM) reported the pricing of an underwritten public offering of 1,561,177 shares of its common stock at a price to the public of $0.17 per share (Press release, Pulmatrix, JAN 28, 2019, View Source [SID1234532929]). Pulmatrix expects to receive aggregate gross proceeds of approximately $265,400 from the offering. The offering is expected to close on or about January 31, 2019, subject to customary closing conditions.

H.C. Wainwright & Co. is acting as the sole book-running manager for the offering.

Pulmatrix currently intends to use the net proceeds from the offering for working capital and general corporate purposes.

A shelf registration statement on Form S-3 (Registration No. 333-212546) relating to the public offering of the shares of common stock described above was filed with the Securities and Exchange Commission ("SEC") and was declared effective on August 3, 2016. A preliminary prospectus supplement describing the terms of the offering was filed with the SEC on January 28, 2019 and is available on the SEC’s website at View Source Copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained, when available, from H.C. Wainwright & Co., LLC, 430 Park Avenue 3rd Floor, New York, NY 10022, or by calling (646) 975-6996 or by emailing [email protected] or at the SEC’s website at View Source

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction. Any offer, if at all, will be made only by means of the prospectus supplement and accompanying prospectus forming a part of the effective registration statement.

EpimAb Biotherapeutics Achieves Key Milestone with Lead Cancer Program EMB01

On January 28, 2019 EpimAb Biotherapeutics, an emerging Shanghai-based biopharmaceutical company specializing in bispecific antibodies, reported its completed recruitment of the first dose cohort in a global Phase I/II clinical study for its most advanced therapeutic program EMB01 (Press release, EpimAb Biotherapeutics, JAN 28, 2019, View Source [SID1234532928]). EpimAb is investigating EMB01 for the treatment of solid tumors in about 60 patients in the US and China. The respective IND applications were simultaneously submitted in the US and China at the end of August 2018. The U.S. Food and Drug Administration (FDA) approved the trial within 28 days after filing and the National Medical Products Administration (NMPA) in China within 45 business days.

"Our development team is excited to see rapid progress with our lead program EMB01. The short turn-around times and swift approval from the regulatory authorities in two of the most important pharmaceutical markets underscore the quality of our data set and our trial applications," commented Dr. Bin Peng, CMO of EpimAb Biotherapeutics. "We are now looking forward to obtain first results demonstrating the safety and preliminary efficacy of EMB01 in cancer patients. EMB01 represents a potential first-in-class approach in solid tumors with a novel mode-of-action unattainable with other drug classes such as monoclonal antibodies."

EMB01 is a bispecific antibody based on EpimAb’s proprietary FIT-Ig (Fabs-In-Tandem Immunoglobulin) technology capable of generating bispecific molecules with superior properties. EMB01 simultaneously targets two receptors, which are widely expressed on cancer cells, EGFR and cMET, and has shown significant and long-lasting activity in multiple preclinical solid tumor models by co-degradation of the two receptors that are required for tumor growth. This novel mechanism of action uniquely available to EGFR/cMet bispecifics could enable EMB01 to treat a much wider patient population than with currently available EGFR or cMet treatments.

While EMB01 advances through clinical studies, EpimAb is constantly growing its proprietary pipeline based on its FIT-Ig platform and is advancing the next FIT-Ig-based program towards IND filing. This second asset is focused on an immuno-oncology approach in areas of high medical need in cancer.

Pulse Biosciences to Report Fourth Quarter and Full Year 2018 Operational Highlights and Financial Results

On January 28, 2019 Pulse Biosciences, Inc. (NASDAQ:PLSE), a novel medical therapy company bringing to market its proprietary CellFX System reported that the Company will report fourth-quarter and full-year 2018 operational highlights and financial results on Thursday, February 7, 2019 (Press release, Pulse Biosciences, JAN 28, 2019, View Source [SID1234532927]). Pulse Biosciences management will host a conference call at 4:30 p.m. Eastern Time (ET) / 1:30 p.m. Pacific Time (PT).

Analysts and investors can participate in the conference call by dialing (844) 494-0190 (domestic) and (508) 637-5580 (international) using the conference ID# 5196202. The conference call can also be accessed live on the Investor Relations section of the Pulse Biosciences website at www.pulsebiosciences.com.