On December 10, 2020 Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines, reported that the first patient has been dosed in the multi-dose portion of the Phase 1 study evaluating [225Ac]-FPI-1434 (FPI-1434) in patients with advanced solid tumors (Press release, Fusion Pharmaceuticals, DEC 10, 2020, View Source [SID1234572635]). FPI-1434 is a radioimmunoconjugate that utilizes Fusion’s proprietary Fast-Clear linker to connect a humanized monoclonal antibody targeting the insulin-like growth factor 1 receptor (IGF-1R), with the alpha-emitting isotope actinium-225, creating a targeted alpha therapy (TAT).

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The Phase 1, multi-center, open-label clinical trial is designed to investigate the safety, tolerability and pharmacokinetics of FPI-1434 in patients with solid tumors expressing IGF-1R. The trial is also designed to establish the maximum tolerated dose for FPI-1434 and the recommended Phase 2 dose. As part of the precision medicine approach, prior to receiving the therapeutic injection of FPI-1434, patients are administered an indium-111 imaging analogue, [111In]-FPI-1547 (FPI-1547). The images collected are used to confirm the presence of tumor uptake and to ensure that estimated radiation doses to organs and tissues are below protocol-specified safety limits.

The multi-dose study follows completion of the single-dose portion of the Phase 1 study, which showed that FPI-1434 was generally well tolerated with no dose limiting toxicities or treatment-related serious adverse events reported to date. The multi-dose portion of the study is expected to enroll patients at sites in Canada, the United States and Australia. The initial patient cohort is being dosed with FPI-1434 at 75kBq/kg with repeat cycles every six weeks up to allowable limits.

"We are pleased with the results of the single-dose portion of our Phase 1 study of FPI-1434 which, following the evaluation of the Safety Review Committee, support initiating the multi-dosing portion of the study," said Chief Executive Officer John Valliant, Ph.D. "This is a critical next step in the FPI-1434 development program as data from the multi-dose portion of the study may provide important insights on potential anti-tumor activity. The multi-dosing trial will also inform the design of the Phase 2 program and assist in the selection of tumor indications to be pursued in planned expansion cohorts. This is especially important given the broad expression of IGF-1R across multiple tumor types."

For additional detail about the study, please visit View Source

About FPI-1434

FPI-1434 is a radioimmunoconjugate designed to target and deliver alpha emitting medical isotopes to cancer cells expressing IGF-1R, a receptor that is overexpressed on many tumor types. FPI-1434 utilizes Fusion’s Fast-Clear linker to connect a human monoclonal antibody that targets IGF-1R with actinium-225, a powerful alpha-emitting isotope with desirable half-life and decay chain properties.

Acknowledgement of US DOE and Actiunium-225 Supply

The actinium-225 used in this research was supplied by the United States Department of Energy Office of Science by the Isotope Program in the Office of Nuclear Physics.

On December 10, 2020 Menarini Silicon Biosystems, a pioneer of liquid biopsy technology, reported new data from the DETECT study program (Press release, Menarini Silicon Biosystems, DEC 10, 2020, View Source [SID1234572634]). These data focused on women with HER2 negative metastatic breast cancer (MBC) based on primary tumor biopsy, and HER2+ CTCs. The results of this large program demonstrate that screening for HER2+ CTCs, in the blood samples of these patients, is an important tool to guide therapy decisions and improve patient outcomes. Furthermore, the randomized DETECT III phase III study, discussed in a poster spotlight session during the 2020 San Antonio Breast Cancer Symposium, shows that adding reference HER2 targeted TKI (tyrosine kinase inhibitor) lapatinib to standard therapy has a positive impact on OS (overall survival) in this particular patient population. DETECT program is the largest screening analysis to-date on the added value of taking into consideration HER2+ overexpression by CTCs in women with a primary HER2 negative breast cancer tumor biopsy, to optimize patient management.

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Prof. Dr. med. Tanja N. Fehm, full Professor and Head of the Department of Gynecology & Obstetrics at the Heinrich-Heine University of Düsseldorf, Germany, said: "The results obtained in the overall DETECT program clearly indicate that CTC HER2 status is an additional important prognostic and predictive tool to CTC enumeration, because it allows us to better understand, beyond the information gathered from the primary tumor biopsy, what our most critical patients need, so that we can provide them with better care." Metastatic disease remains, indeed, the principal cause of cancer related deaths. CTCs have repeatedly been shown to represent qualified biomarkers to decipher various clinical and molecular complexities of advanced cancers. "The opportunity provided by the DETECT III study, to optimize treatment for patients with metastatic disease who are initially diagnosed with HER2 negative- breast cancer and therefore logically deprived of the HER2 therapy they may actually need, is likely to remove a significant roadblock in the search for a cure to the high burden of this distant recurrent type of breast cancer," she added.

More than 1,900 patients with HER2 negative MBC were screened for CTC enumeration and phenotype using Menarini Silicon Biosystems’ CELLSEARCH CTC System. The CTCs were labeled with an anti HER2 antibody*, stained and classified according to staining intensity. HER2 status of CTCs was assessed in 1,159 CTC-positive patients. A total of 174 (15.0%) patients had a CTC count ≥1 with strong HER2 staining. This situation was significantly associated, in a univariate analysis, with shorter OS.

Finally 105 patients with moderate-to-strong positive CTC-HER2 staining were enrolled in the DETECT III study and randomized to either the lapatinib in combination with standard therapy arm or standard therapy alone arm. Patients in the lapatinib arm had a significantly improved OS. In addition, patients with no evidence of CTC (CTC0), at the time of first follow-up within a median of 73 days, showed better OS compared to patients with CTCs (HR 0.36; 95% CI 0.17 – 0.76; p-value= 0.005). The main conclusion of the DETECT program is that HER2 over expression on CTCs, in patients with metastatic HER2 negative primary breast cancer, provides a key signal to physicians to consider a more appropriate therapeutic option.

"Breast cancer is a heterogenous disease with an increasing number of therapeutic strategies that physicians can prescribe depending on individual patient characteristics and for which they are eager to obtain appropriate noninvasive diagnostic tools to help them optimize their choices in real-time," said Cecilia Simonelli, MD and Global Medical Affairs Head at Menarini Silicon Biosystems. "We are particularly committed to leveraging our technology so that this becomes possible and so that we can contribute to the ultimate goal of allowing even the most difficult cancers to become curable."

About the DETECT study program

DETECT is the largest study program on the role of Circulating Tumor Cell (CTC) count and HER2 phenotype assessment to personalize treatment strategies for HER2 negative MBC. The aim of this program is to evaluate the impact of adapting therapeutic interventions based on CTC phenotypes in patients with a discordant HER2 negative primary tumor biopsy and HER2+ over expression of CTCs in the metastatic setting. The study results consistently show the importance of adding HER2 targeted therapy in this patient population to optimize patient outcomes.

About CELLSEARCH

CELLSEARCH is the first and only clinically validated blood test cleared by the U.S. Food & Drug Administration (FDA) for detecting and counting CTCs to aid physicians in managing patients with metastatic breast, prostate, and colorectal cancers when used in conjunction with other clinical methods of monitoring. The test is also approved by the China National Medical Products Administration (NMPA) for use in monitoring patients with Metastatic Breast Cancer. The CELLSEARCH System is the most extensively studied CTC technology, with research published in more than 650 peer-reviewed publications.

CELLSEARCH Circulating Tumor Cell Kit is not cleared or approved for use to guide treatment decisions. For more information on the full intended use and limitations of the CELLSEARCH system, please refer to the Instructions for Use at View Source

*The CELLSEARCH Tumor Phenotyping Reagent (HER-2/neu) is for Research Use Only. Not for Use in Diagnostic Procedures. The performance characteristics and safety have not been established and are not cleared or approved by the FDA.

On December 10, 2020 AcelRx Pharmaceuticals, Inc. (NASDAQ: ACRX) (AcelRx), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in healthcare institutions, reported it has entered into an agreement with three life sciences-focused investment funds that are existing investors and new investors in AcelRx, for the sale of 8,333,333 shares of common stock at $1.20 per share (Press release, AcelRx Pharmaceuticals, DEC 10, 2020, https://www.prnewswire.com/news-releases/acelrx-announces-10-million-registered-direct-common-stock-offering-301190326.html [SID1234572633]). AcelRx estimates gross proceeds from the offering of approximately $10.0 million. The closing of the transaction is expected to occur by December 11, 2020, subject to satisfaction of customary closing conditions.

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The securities described above are being offered by AcelRx pursuant to a shelf registration statement previously filed with the Securities and Exchange Commission (the "SEC"), which the SEC declared effective on July 8, 2020. 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.

On December 10, 2020 LIPAC Oncology LLC and Huons Co., Ltd. reported the achievement of a major development milestone in their partnering agreement by finalizing its Clinical Study Report for a Phase 2A marker lesion clinical trial in patients with low-grade highly recurrent Non-Muscle Invasive Bladder Cancer (NMIBC) treated with LiPax (paclitaxel) (Press release, Lipac Oncology, DEC 10, 2020, View Source [SID1234572632]). LiPax, a proliposomal paclitaxel formulation in development for intravesical instillation in the treatment of NMIBC, demonstrated a 63 percent responder rate in highly recurrent and heavily pre-treated patients.

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"This is an important milestone for LIPAC as we prepare for our Type B meeting with the U.S. Food and Drug Administration in early January to validate our Phase 2B/3 clinical study approach," said TR Thirucote, Ph.D., Chairman and CEO of LIPAC.

In September 2019, LIPAC and Huons announced that they entered into an exclusive licensing agreement to develop, manufacture, and commercialize LiPax for all indications in South Korea. This is the first milestone payment to LIPAC based on specific development, regulatory and commercial milestones and royalty payments based on sales.

"We are delighted to have reached this milestone in our agreement with LIPAC that brings us a step closer to making this essential therapy available to thousands of patients in Korea," said Mr. Key An UM, CEO of Huons. "Our interest in LiPax extends well beyond NMIBC as we continue our collaboration with LIPAC in additional orphan-designated programs for Upper Tract Urothelial Carcinoma, Ovarian Cancer, Mesothelioma, and Malignant Plural Effusion for Breast Cancer."

"Non-Muscle Invasive Bladder Cancer is difficult to treat and highly recurrent. By pairing a simple outpatient procedure (Transurethral Resection of Bladder Tumor or TURBT) with LiPax, we have an opportunity to substantially improve both clinical outcomes and quality of life for patients globally," said Michael Oefelein, M.D., Chief Medical Officer of LIPAC Oncology. "We were also pleased to present our Phase 2A data at the Society of Urological Oncology meeting earlier this month."

Based on a patient’s biopsy results obtained from TURBT, NMIBC is stratified into three risk categories: low, intermediate, and high risk. To reduce recurrence and prevent progression, the American Urological Association NMIBC guidelines recommend intravesical therapy after TURBT. The low to intermediate risk category targeted by LiPax is estimated to comprise 90,000 Americans, yet no intravesical agent is approved by the U.S. Food and Drug Administration for this disease.

About LiPax
LiPax is a novel, investigational formulation of paclitaxel in Phase 2 development for the treatment of NMIBC. LIPAC Oncology’s proprietary formulation utilizes their proliposomal technology platform to enhance the persistence and penetration of bladder tissue by paclitaxel. LiPax is designed to enhance the standard of care of outpatient endoscopic tumor removal through a histological risk assessment followed by intravesical instillation using a standard urinary catheter. LIPAC Oncology completed a Phase 2A clinical trial in August 2020 and intends to advance the program to a pivotal study to further investigate LiPax in the treatment of this condition.

On December 10, 2020 Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in class therapeutics in hematology and oncology, reported that the National Medical Products Administration (NMPA) has accepted the Investigational New Drug (IND) application for ATG-010 (selinexor), an oral Selective Inhibitor of Nuclear Export (SINE) compound, in combination with bortezomib and dexamethasone for the treatment of patients with relapsed/refractory multiple myeloma (rrMM) in China (Press release, Antengene, DEC 10, 2020, View Source [SID1234572630]).

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The trial is a randomized, controlled, open-label, multicenter, Phase 3 trial, aiming to evaluate the efficacy and safety of ATG-010, bortezomib and dexamethasone (SVd) regimen against bortezomib and dexamethasone (Vd) regimen in Chinese adult patients with rrMM who have received one to three prior lines of therapy. A total of 150 patients will be randomized in a 2:1 ratio to receive SVd or Vd treatment.

ATG-010 is a first-in-class and only-in-class oral selective inhibitor of nuclear export (SINE) and it is now the first and only drug approved by the Food and Drug Administration (FDA) for use in both multiple myeloma and diffuse large B-cell lymphoma. In China, Antengene is conducting a registrational Phase 2 clinical trial of ATG-010 for rrMM (MARCH). The trial is expected to complete enrollment by the end of 2020.

"The result of the BOSTON study has demonstrated that the SVd regimen reduced the risk of disease progression or death with lower doses of bortezomib and dexamethasone in the triplet combination when compared to the standard Vd regimen. Addition of ATG-010 to Vd may be more convenient and provides a more significant therapeutic effect in patients with rrMM." said Dr. Jay Mei, Founder, Chairman and CEO of Antengene. "This planned Phase 3 trial, which is the registrational study based on BOSTON, is going to validate the SVd regimen’s efficacy and safety in Chinese population."

About ATG-010 (selinexor, XPOVIO)

ATG-010 (selinexor, XPOVIO) is a first-in-class and only-in-class oral selective inhibitor of nuclear export compound, developed by Antengene and Karyopharm Therapeutics Inc. (NASDAQ: KPTI). In July 2019, the US Food and Drug Administration (FDA) approved ATG-010 in combination with low-dose dexamethasone for the treatment of relapsed/refractory multiple myeloma (rrMM) and in June 2020 approved ATG-010 as a single-agent for the treatment of relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL). ATG-010 is so far the first and only oral SINE compound approved by the FDA. ATG-010 is also being evaluated in several other mid-and later-phase clinical trials across multiple solid tumor indications, including liposarcoma and endometrial cancer. In November 2020, at the Connective Tissue Oncology Society 2020 Annual Meeting (CTOS 2020), Antengene’s partner, Karyopharm Therapeutics, presented positive results from the Phase 3 randomized, double blind, placebo controlled, cross-over SEAL study evaluating single agent, oral ATG-010 versus matching placebo in patients with liposarcoma. Karyopharm also recently announced that the ongoing Phase 3 SIENDO study of ATG-010 in patients with endometrial cancer passed planned interim futility analysis and that Data and Safety Monitoring Board (DSMB) recommended the study should proceed as planned without any modifications. Top-line SIENDO study results are expected in the second half of 2021.

Antengene is conducting two registrational Phase 2 clinical trials of ATG-010 in China for relapsed refractory multiple myeloma (MARCH) and for relapsed refractory diffuse large B-cell lymphoma (SEARCH), and has initiated clinical trials for high prevalence cancer types in the Asia Pacific region including peripheral T-cell lymphoma and NK/T-cell lymphoma (TOUCH) and KRAS-mutant non-small cell lung cancer (TRUMP).