Chi-Med Presents Phase I/II Clinical Data for Selective VEGFR Inhibitor Fruquintinib at the 2017 Gastrointestinal Cancers Symposium

On January 16, 2017 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported that data from the ongoing Phase I/II clinical trial of fruquintinib in combination with paclitaxel (Taxol) in second-line patients with advanced gastric cancer will be presented at the 2017 Gastrointestinal Cancers Symposium sponsored by the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO-GI"), being held in San Francisco, California from January 19 to 21, 2017 (Press release, Hutchison China MediTech, JAN 16, 2017, http://www.chi-med.com/chi-med-presents-phase-iii-clinical-data-for-selective-vegfr-inhibitor-fruquintinib-at-the-2017-gastrointestinal-cancers-symposium/ [SID1234517411]). Fruquintinib is a highly selective and potent oral inhibitor of vascular endothelial growth factor receptors ("VEGFR").

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Chi-Med completed a Phase Ib dose finding study of fruquintinib in combination with paclitaxel, which established a combination regimen that was well tolerated, and continued to enroll patients in this trial to expand the data-set. Additional details about this study may be found at clinicaltrials.gov, using identifier NCT02415023.

The most recent results of the study will be presented in detail as follows:

Presentation Title:

A Phase I/II trial of Fruquintinib in Combination with Paclitaxel for Second-line Treatment in Patients with Advanced Gastric Cancer

Authors:
Ruihua Xu, Dongsheng Zhang, Lin Shen, Jin Li, Jing Huang, Yang Zhang, Jifang Gong, Weijian Guo, Songhua Fan, Ke Li, Ye Hua and Weiguo Su

Abstract No:

128

Session:

Poster Session A: Cancers of the Esophagus and Stomach

Date & Time: Thursday, January 19, 2017, 12:30 PM-6:30 PM (PST)
Once presented, the presentation will be available at www.chi-med.com/news. Further information about ASCO (Free ASCO Whitepaper)-GI is available at gicasym.org.

ABSTRACT
A Phase I/II Trial of Fruquintinib in Combination with Paclitaxel for Second-line Treatment in Patients with Advanced Gastric Cancer

Ruihua Xu, Dongsheng Zhang, Lin Shen, Jin Li, Jing Huang, Yang Zhang, Jifang Gong, Weijian Guo, Songhua Fan, Ke Li, Ye Hua and Weiguo Su

Background

Advanced gastric cancer is a major public health problem, particularly in Asian countries. The treatment options are limited in patients who failed standard first-line chemotherapy. This Phase I/II study is aimed to evaluate the tolerability, pharmacokinetics ("PK") and preliminary efficacy of fruquintinib, a selective oral VEGFR inhibitor, combined with paclitaxel as second-line therapy in Chinese patients with advanced gastric cancer.

Patients and methods

This open arm Phase I/II trial (NCT02415023) consisted of dose finding and dose expansion stages. In the dose finding stage, three dose levels of fruquintinib (2, 3, 4mg once daily; three-weeks-on and one-week-off) were evaluated in combination with standard 80mg/m2 paclitaxel (once weekly on day 1, 8 and 15) in a 28-day cycle until the maximum tolerated dose ("MTD") or recommended phase II dose ("RP2D") was reached. Additional patients were enrolled at dose expansion phase with fruquintinib RP2D regimen to assess further the efficacy, safety and PK profile.

Results

As of September 10, 2016, a total of 32 patients were enrolled and dosed with fruquintinib in combination with weekly paclitaxel. The RP2D of fruquintinib was determined to be 4 mg daily.

Two patients at 4 mg experienced dose-limiting toxicity, both with febrile neutropenia. Grade 3 or 4 treatment emergent adverse events ("TEAE") were neutropenia (40.6%), leukopenia (28.1%), decreased hemoglobin (6.25%), hand-foot skin reaction (6.25%), neurophlegmon (6.25%), and hypertension (6.25%), with higher frequencies in the 4mg cohort as compared with lower doses.

At steady state, fruquintinib drug exposure, i.e. the area under the curve (AUCss), increased dose-proportionally and was within the same range as given as a single agent. Paclitaxel exposure at fruquintinib RP2D (4mg) however, increased by approximately 30% as compared to that of single agent.

28 of 32 patients were evaluable for tumor response, and of these, 10 patients achieved confirmed partial response (objective response rate, ("ORR") = 35.7%), 9 patients experienced stable disease for at least 8 weeks (disease control rate, ("DCR") = 67.9%). At fruquintinib RP2D, ≥16w progression free survival ("PFS") = 50% and ≥7m overall survival ("OS") = 50%.

Conclusion

Combination therapy of fruquintinib and paclitaxel appeared to be generally well-tolerated with promising tumor response in the second-line setting in advanced gastric cancer. Further evaluation of fruquintinib in a randomized control trial is warranted.

About Gastric Cancer
Every year, it is estimated that approximately one million new patients around the world are diagnosed with gastric cancer, according to Frost & Sullivan, and in 2015 China represented approximately 44% of all newly diagnosed gastric cancer cases worldwide. The very high prevalence of gastric cancer in China as compared to the rest of the world is thought to be linked in part to food preparation habits, such as the use of certain preservatives. In 2015 there were an estimated 679,100 incidence gastric cancer cases and 498,000 mortality cases in China, according to the National Central Cancer Registry of China.

Gastric cancer is the third of most lethal cancer worldwide. As it is often diagnosed at an advanced stage, prognosis is poor with a median OS of less than 12 months. Although targeted therapy is under development in China, chemotherapy remains the mainstay of treatment for gastric cancer patients and confers only a moderate survival advantage. Accordingly, we see a high medical need for new targeted treatment options.

About Fruquintinib
Fruquintinib (HMPL-013) is a highly selective small molecule drug candidate that has been shown to inhibit VEGFR 24 hours a day via an oral dose, without known off-target toxicities. It is currently under the joint development in China by Chi-Med and its partner Eli Lilly and Company. Two late-stage, pivotal Phase III registration studies are ongoing in colorectal cancer (FRESCO) and lung cancer (FALUCA) along with the currently reported gastric cancer trial.

Colorectal: The FRESCO trial is a randomized, double-blind, placebo-controlled, multicenter, Phase III pivotal trial in patients with locally advanced or metastatic colorectal cancer who have failed at least two prior systemic antineoplastic therapies, including fluoropyrimidine, oxaliplatin and irinotecan. Enrollment was completed in May 2016. 416 patients were randomized at a 2:1 ratio to receive either: 5mg of fruquintinib orally once per day, on a three-weeks-on / one-week-off cycle, plus best supportive care ("BSC"); or placebo plus BSC. The primary endpoint is OS, with secondary endpoints including PFS, ORR, DCR and duration of response. Additional details of the FRESCO study may be found at clinicaltrials.gov, using identifier NCT02314819.

Lung: The FALUCA trial is a randomized, double-blind, placebo-controlled, multi-center, Phase III registration study targeted at treating patients with advanced non-squamous NSCLC, who have failed two lines of systemic chemotherapy. Enrollment began in December 2015. Patients are randomized at a 2:1 ratio to receive either: 5mg of fruquintinib orally once per day, on a three-weeks-on / one-week-off cycle, plus BSC; or placebo plus BSC. The primary endpoint is OS, with secondary endpoints including PFS, ORR, DCR and duration of response. Chi-Med plans to enroll approximately 520 patients in about 45 centers across China. Additional details about this study may be found at clinicaltrials.gov, using identifier NCT02691299.

Hope Biosciences Licenses nuc-Gemcitabine, A ‘Trojan Horse’ Anti-nucleolin-Gemcitabine Aptamer Drug Conjugate Against Cancer

On January 13, 2017 HOPE BIOSCIENCES reported that it has acquired the exclusive rights to develop and commercialize nuc-gemcitabine (APTA-12/HOPE-888) through a licensing agreement with AptaBio Therapeutics, a Korean pharmaceutical company (Press release, Hope Biosciences, JAN 13, 2017, View Source [SID1234609541]).

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nuc-Gemcitabine consists of Antisoma’s AS1411*, a clinically-tested DNA aptamer against surface nucleolin found on many cancer cells, and dFdCMP, an activated form of gemcitabine. Unlike ADCs (antibody drug conjugates) or SMDCs (small molecule drug conjugates), which require ‘linker’ conjugation of the cytotoxic payload to the drug, nuc-gemcitabine is created by incorporating a single activated gemcitabine molecule in lieu of a thymidine molecule during the one-step solid phase synthesis of the DNA aptamer.

Clinical trials in over 80 cancer patients showed AS1411 to have modest anti-cancer activity and a favorable safety profile. nuc-Gemcitabine is 100-1,000x more potent compared to AS1411 or conventional gemcitabine (Gemzar) in pre-clinical studies. Composition of matter patent has been granted in major countries, including the United States.

"Nucleolin is constantly and abundantly expressed on the cell surface of tumor cells where it serves as a binding protein for a variety of ligands implicated in cancer growth and angiogenesis," said Dr. SungHwan Moon, President and Chief Scientific Officer of AptaBio. "High nucleolin expression is correlated with decreased survival. nuc-Gemcitabine binds to surface nucleolin with high specificity and affinity, leading to internalization of the aptamer-drug into tumor cells, where it mediates cell death. We have demonstrated significantly greater anti-cancer activity at low doses of nuc-gemcitabine containing the equivalent of 3 mg/kg gemcitabine versus 80-100 mg/kg of Gemzar in gemcitabine-resistant pancreatic cancer xenograft mouse models."

"HOPE-888 has demonstrated impressive anti-cancer activity in preclinical models. It could truly be a game changing treatment strategy for pancreatic cancer and other difficult-to-treat malignancies," said George Uy, CEO and Founder of HOPE. "Our goal is to file IND application in the next 12-18 months and bring it into the clinic soon thereafter. We intend to truly personalize nuc-gemcitabine therapy by screening patients for nucleolin-expression with a biomarker kit."

Aptevo Therapeutics Receives $20 Million Payment From Emergent BioSolutions

On January 13, 2017 Aptevo Therapeutics Inc. (Nasdaq:APVO), a biotechnology company focused on developing novel oncology and hematology therapeutics, reported that it has received a $20 million cash payment from Emergent BioSolutions pursuant to a promissory note granted as a result of the spin-off of Aptevo from Emergent, effective August 1, 2016 (Press release, Aptevo Therapeutics, JAN 13, 2017, View Source;p=irol-newsArticle&ID=2237367 [SID1234517410]).

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With the additional $20 million cash payment announced today, Emergent has provided a total of $65 million in cash contributions to fund Aptevo’s operations as a newly-launched oncology and hematology-focused company with a broad pipeline of commercial, clinical and preclinical assets and a proprietary bispecific technology platform, ADAPTIR, focused on the development of immuno-oncology therapeutics.

"The achievement of this final payment from Emergent marks another important milestone in Aptevo’s progress," said Marvin L. White, President and Chief Executive Officer. "With the generous funding provided by Emergent, and a strong portfolio of revenue-generating commercial assets, Aptevo is solidly positioned to achieve our near-term goals. Specifically, these include: capturing increased market share for our newly-launched Hemophilia B therapeutic, IXINITY; generating additional data from our two clinical-stage programs, otlertuzumab and MOR209/ES414; and rapidly advancing new ADAPTIR immuno-oncology candidates into clinical development."

About Aptevo Therapeutics Inc.

Aptevo Therapeutics Inc. is a biotechnology company focused on novel oncology and hematology therapeutics to meaningfully improve patients’ lives. Our core technology is the ADAPTIR (modular protein technology) platform. Aptevo has four commercial products in the areas of hematology and infectious diseases, as well as various investigational stage product candidates in immuno-oncology.

Heron Announces Submission of CINVANTI™ NDA for the Prevention of Chemotherapy-Induced Nausea and Vomiting (CINV)

On January 12, 2017 Heron Therapeutics, Inc. (NASDAQ:HRTX), a commercial-stage biotechnology company focused on developing novel best-in-class treatment solutions to address some of the biggest unmet patient needs, reported submission of the New Drug Application (NDA) for CINVANTI (HTX-019), the first polysorbate 80-free, intravenous formulation of aprepitant for the prevention of CINV, to the U.S. Food and Drug Administration (FDA) (Press release, Heron Therapeutics, JAN 12, 2017, View Source;p=RssLanding&cat=news&id=2237084 [SID1234517367]). Aprepitant belongs to a class of agents known as NK1 receptor antagonists, which are often used in combination with 5-HT3 receptor antagonists for the prevention of CINV.

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The NDA filing includes data demonstrating the bioequivalence of CINVANTI to EMEND IV (fosaprepitant), supporting its efficacy for the prevention of both acute and delayed CINV with both moderately emetogenic chemotherapy (MEC) and highly emetogenic chemotherapy (HEC). Results also showed CINVANTI was better tolerated than EMEND IV, with significantly fewer adverse events reported with CINVANTI.

"The filing of the NDA for CINVANTI is an important milestone, bringing us one step closer to a new NK1 receptor antagonist treatment option for cancer patients suffering from the debilitating side effects of chemotherapy," said Kimberly J. Manhard, Executive Vice President, Drug Development at Heron Therapeutics. "We look forward to working closely with the FDA during the review of the NDA for CINVANTI in anticipation of FDA approval in late 2017."

"CINVANTI is based on the most widely used NK1 receptor antagonist, with almost 9 years of safety and efficacy experience," said Barry D. Quart, Pharm.D., Chief Executive Officer of Heron Therapeutics. "Since NK1 receptor antagonists are used in combination with 5-HT3 receptor antagonists, CINVANTI offers a strong strategic and operational fit with Heron’s existing commercial product, SUSTOL, our extended-release, injectable product that incorporates the 5-HT3 receptor antagonist granisetron and is also indicated for the prevention of CINV."

About CINVANTI (HTX-019) for CINV
CINVANTI is a proprietary intravenous formulation of aprepitant, a NK1 receptor antagonist for the prevention of CINV. NK1 receptor antagonists are typically used in combination with 5-HT3 receptor antagonists. Currently, the only injectable NK1 receptor antagonist approved in the U.S. contains polysorbate 80, a surfactant, which may cause hypersensitivity reactions, infusion site reactions or other adverse reactions in some patients. Heron’s formulation for CINVANTI does not contain polysorbate 80 and may have a lower incidence of certain types of adverse reactions than reported with the other commercially available injectable NK1 receptor antagonist.

About SUSTOL (granisetron) extended-release injection
SUSTOL is indicated in combination with other antiemetics in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of MEC or anthracycline and cyclophosphamide (AC) combination chemotherapy regimens. SUSTOL is an extended-release, injectable 5-HT3 receptor antagonist that utilizes Heron’s Biochronomer polymer-based drug delivery technology to maintain therapeutic levels of granisetron for ≥5 days. The SUSTOL global Phase 3 development program was comprised of two, large, guideline-based clinical trials that evaluated efficacy and safety in more than 2,000 patients with cancer. The efficacy of SUSTOL in preventing nausea and vomiting was evaluated in both the acute phase (day 1 following chemotherapy) and the delayed phase (days 2-5 following chemotherapy). Please see full prescribing information, including additional important safety information, available at www.SUSTOL.com.

Kitov Enters Immuno-Oncology Field Through Acquisition of TyrNovo

On January 12, 2017 Kitov Pharmaceuticals Holdings Ltd. (NASDAQ: KTOV; TASE: KTOV), an innovative biopharmaceutical company, reported the expansion of its pipeline through the acquisition of a majority stake in TyrNovo Ltd., a privately held developer of novel small molecules in the immuno-oncology therapeutic field (Press release, Kitov Pharmaceuticals , JAN 12, 2017, View Source [SID1234517379]).

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The expansion into developing immuno-oncology drugs comes as Kitov plans to file its New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for its flagship combination drug, KIT-302, which is intended to treat osteoarthritis pain and hypertension simultaneously, in Q1 2017, with commercial launch anticipated for the first half of 2018. Kitov plans to harness its development and regulatory capabilities in proceeding towards submitting an investigational new drug (IND) application with the U.S. FDA and initiate clinical trials for its newly acquired drug, NT219.

The market for immuno-oncology treatments is believed to become worth $14 billion by 2019 and to grow to $34 billion by 2024, driven by long-term and durable tumor responses to immuno-oncology drugs, according to Global Data.

Kitov will initially acquire an approximately 56% equity stake in TyrNovo from its majority shareholder, for consideration of $2 million in cash and $1.8 million equivalent ordinary shares of Kitov based on the closing price of Kitov’s shares on the TASE on January 11, 2017. Following the closing of this initial acquisition, which is expected to take place on January 13, 2017, Kitov anticipates that it may acquire additional equity stakes in TyrNovo from all or part of TyrNovo’s additional minority shareholders for consideration consisting of ordinary shares of Kitov in such amounts as to be agreed with the shareholders.

"The TyrNovo acquisition represents a major milestone in Kitov’s strategic vision of establishing a diverse pipeline that we believe will secure long term value creation for its shareholders. We are excited about NT219 and its prospects in the oncology field. NT219 can be developed as a platform combination drug for overcoming multi-drug resistance often observed in various tumors. It has the potential to be developed as a combination therapy with several approved oncology drugs for multiple types of tumors," stated Dr. Paul Waymack, Kitov’s Chairman and Chief Medical Officer.

"The Kitov regulatory team has a proven track record in the development and approval of drugs for oncology as well as other indications required to develop NT219 towards submission of an IND and initiation of clinical trials," Waymack added.

TyrNovo is led by its CEO, Dr. Hadas Reuveni, a co-inventor of the TyrNovo technology, who received her Ph.D., Summa Cum Laude, for anti-cancer drug discovery from the Hebrew University of Jerusalem and has nearly two decades of R&D experience in Biotechnology. Dr. Reuveni commented, "I am excited to join Kitov and its team to expedite the development of NT219, which addresses a true unmet medical need for numerous oncology indications. Cancer drug resistance is the major reason for failure in anti-cancer drug treatment. Preventing resistance to the drugs is critical for improving effective cancer treatment."

About NT219

NT219 is a small molecule that presents a new concept in cancer therapy by promoting the degradation of two oncology-related checkpoints, Insulin Receptor Substrates (IRS) 1 and 2 as well as the inhibition of signal transducer and activator of transcription 3 (STAT3). While targeted anti-cancer drugs inhibit the "ON" signal, NT219 activates the "OFF" switch, extensively blocking major oncogenic pathways. In pre-clinical trials, NT219, in combination with several approved cancer drugs, displayed potent anti-tumor effects and increased survival in various cancers by preventing the tumors from developing multi-drug resistance and ameliorating multi-drug resistance after resistance is acquired. NT219, in combination with various approved oncology drugs, demonstrated potent anti-tumor effects and increased survival in various cancer models including sarcoma, melanoma, pancreatic, lung, ovarian, head & neck, prostate and colon cancers, through the prevention of acquired resistance and regression of resistant tumors.