On June 9, 2020 Veracyte, Inc. (Nasdaq: VCYT) reported its submission of a De Novo classification request to the U.S. Food & Drug Administration (FDA) for the nCounter Dx LymphMark Assay, a novel, genomic lymphoma subtyping test (Press release, Veracyte, JUN 9, 2020, View Source [SID1234560933]). The in vitro diagnostic test is used on the nCounter Flex Analysis System, Veracyte’s diagnostics platform, and is intended to add to Veracyte’s portfolio of tests that address complex clinical problems, helping to inform diagnoses and better treatment decisions.

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Veracyte has proposed the LymphMark test as an aid in disease characterization and prognosis to support disease management for patients newly diagnosed with diffuse large B-cell lymphoma (DLBCL). The LymphMark test utilizes gene-expression profiling of RNA extracted from formalin-fixed paraffin-embedded tissue to classify the "cell of origin" subtype of DLBCL tumors. The World Health Organization recommends gene-expression profiling for patients with DLBCL, given that certain subtypes are associated with poorer clinical outcomes, which may potentially be mitigated by more-specific treatments that are under development.1

"This submission marks an important milestone for Veracyte in our efforts to expand the menu of advanced genomic tests we provide on the nCounter system. Ultimately, our goal is to make our broader menu of tests accessible to patients worldwide through hospitals and laboratories that can perform them locally," said Bonnie Anderson, Veracyte’s chairman and chief executive officer.

Veracyte acquired the LymphMark test in December 2019 as part of its acquisition from NanoString of the exclusive global diagnostic rights to the nCounter system.

"DLBCL is an aggressive form of lymphoma with heterogeneous clinical behavior – outcomes significantly depend upon the distinct molecular subtype of each patient’s tumor," said Dr. David Scott, associate professor in the Department of Medicine at the University of British Columbia and a scientist in the Department of Lymphoid Cancer Research at BC Cancer, a program of the Provincial Health Services Authority. "The ability to determine this information from gene-expression profiling of tissue samples collected routinely in a clinical setting has the potential to transform patient care, making personalized treatment options more accessible."

Non-Hodgkin lymphoma ranks among the top-10 common cancers worldwide, with over 500,000 new cases estimated in 2018.2 DLBCL accounts for approximately 30 percent of lymphomas.3

On June 9, 2020 Rafael Holdings, Inc., (NYSE: RFL), reported revenue of $1.2 million and a loss per share of $0.14 for the fiscal quarter ended April 30, 2020 (Press release, Rafael Holdings, JUN 9, 2020, View Source [SID1234560950]).

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Q3 FY 2020 Consolidated Highlights

Revenue of $1.2 million in Q3 FY2020, generated by Rafael Holdings’ real estate portfolio, decreased from $1.4 million in the year-ago quarter. The loss per share of $0.14 increased from $0.07 in the year ago quarter largely on increased R&D expense incurred by the Barer Institute.
Rafael Pharmaceuticals

On May 26, 2020, Rafael Pharma announced positive results of a single-arm, open-label, Phase 1 study of CPI-613 (devimistat) with gemcitabine and nab-paclitaxel in patients with locally advanced or metastatic pancreatic cancer. The data was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program.
On April 28, 2020, Rafael Pharma announced the expansion of its Phase 2 clinical trial of CPI-613 (devimistat) for patients with relapsed or refractory Burkitt’s lymphoma/leukemia. The clinical trial began enrolling patients at MD Anderson Cancer Center, where Dr. Raphael Steiner serves as principal investigator.
On March 24, 2020, Rafael Pharma announced that it had enrolled more than 75% of the 500 patients needed for its pivotal Phase 3 clinical trial for metastatic pancreatic cancer (AVENGER 500). The trial is evaluating the efficacy and safety of Rafael’s lead compound CPI-613️ (devimistat) in combination with modified FOLFIRINOX (mFFX) as first-line therapy.
LipoMedix

At April 30, 2020, Rafael Holdings held 57.9% of the issued and outstanding ordinary shares of LipoMedix, a development-stage Israeli company focused on the development of an innovative, safe and effective cancer therapy based on liposome delivery.

Lipomedix was awarded a Seal of Excellence for its Promitil project by European Innovation Council of the European Union.
LipoMedix’s Phase IB study of Promitil in Israel continued to enroll patients with advanced cancer requiring palliative radiotherapy for inoperable tumors or metastatic disease.
Barer Institute

Rafael Holdings increased its investment in pharmaceutical development through its Barer Institute subsidiary. The Barer Institute is currently testing indications for lead compounds targeting cancer metabolism and has initiated a preclinical in-licensing effort on selected compounds that target the unique mechanisms of cancer.

Remarks by Howard Jonas, Chairman and CEO of Rafael Holdings

"Rafael Holdings’ key pharmaceutical investments, Rafael Pharma and LipoMedix and our wholly owned Barer Institute, continue to execute on their development and clinical programs despite the challenges posed by the worldwide Covid-19 pandemic. I am especially gratified that Rafael Pharma has surpassed the 80% enrollment milestone in its pivotal Phase 3 Avenger 500 study of patients with pancreatic cancer. The Barer Institute is evaluating promising candidates for potential clinical development programs. And finally, we continue to work to monetize our New Jersey real estate assets, while our asset in Israel is now fully leased."

On June 9, 2020 Fate Therapeutics, Inc. (the "Company" or "Fate Therapeutics") (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported the pricing of an underwritten public offering of 6,181,562 shares of its common stock at a public offering price of $28.31 per share, before underwriting discounts, for an aggregate offering of approximately $175 million (Press release, Fate Therapeutics, JUN 9, 2020, View Source [SID1234560914]). Fate Therapeutics has granted the underwriters a 30-day option to purchase up to an additional 927,234 shares of its common stock. The proceeds to Fate Therapeutics from this offering are expected to be approximately $164.2 million after deducting underwriting discounts and commissions and other estimated offering expenses but excluding any exercise of the underwriters’ option. Fate Therapeutics intends to use the net proceeds from the offering to fund clinical trials and nonclinical studies, the manufacture of its clinical product candidates, the expansion of its cGMP compliant manufacturing operations, including the construction, commissioning and qualification of its new facility, the conduct of preclinical research and development, and for general corporate purposes. All shares of common stock to be sold in the offering are being offered by Fate Therapeutics. The offering is expected to close on or about June 11, 2020, subject to customary closing conditions.

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Subject to the completion of the offering and the expiration or early termination of applicable waiting periods relating to certain antitrust filings under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, the Company also expects to sell to Johnson & Johnson Innovation-JJDC, Inc. in a private placement, 1,766,160 shares of common stock for an aggregate purchase price of approximately $50.0 million, at a price per share equal to the price to the public in the underwritten public offering.

Jefferies, SVB Leerink, Barclays and Guggenheim Securities are acting as joint book-running managers for the offering. Mizuho Securities is acting as lead manager for the offering, and H.C. Wainwright & Co. is acting as co-manager for the offering.

The securities described above are being offered by Fate Therapeutics pursuant to an automatic shelf registration statement on Form S-3 (File No. 333-228513) that was previously filed by Fate Therapeutics with the Securities and Exchange Commission (the "SEC") and automatically became effective upon filing on November 21, 2018. The securities may be offered only by means of a prospectus.

A preliminary prospectus supplement related to the offering was filed with the SEC on June 8, 2020 and is available on the SEC’s website at View Source and a final prospectus supplement related to the offering will be filed with the SEC and will be available on the SEC’s website at View Source. Copies of the final prospectus supplement and the accompanying prospectus for the securities being offered may also be obtained, when available, by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by e-mail at [email protected] or by telephone at (877) 547-6340; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525 ext. 6218 or by email at [email protected]; Barclays Capital Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (888) 603-5847 or by email at [email protected]; or Guggenheim Securities, LLC Attention: Equity Syndicate Department, 330 Madison Avenue, New York, NY 10017 or by telephone at (212) 518-5548, or by email at [email protected].

On June 9, 2020 L.E.A.F. Pharmaceuticals LLC ("LEAF"), a global pharmaceutical company focused on developing novel anticancer drugs, reported that results from a preclinical study of LEAF-1401, the Company’s lead anticancer product, were presented at the 2020 Virtual Annual Meeting of ASCO (Free ASCO Whitepaper) held from May 29 – 31, 2020 (Press release, LEAF Pharmaceuticals, JUN 9, 2020, View Source [SID1234560951]). ASCO (Free ASCO Whitepaper) is the world’s largest cancer conference which brings together, from around the world, both cancer researchers and cancer healthcare providers to review and discuss new cancer treatments being developed worldwide. Treatment with LEAF-1401 resulted in 20-fold higher intratumoral exposure levels of pentaglutamated pemetrexed (the main active form of pemetrexed) and 30-fold higher intratumor exposure levels of pemetrexed itself, when compared to treatment with currently approved pemetrexed (Alimta).

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Details of the poster are as follows:

Poster #3524-254: Intratumoral Exposure Levels of Pentaglutamated Pemetrexed following Treatment with LEAF-1401 and Pemetrexed.

A copy of the poster is available on the LEAF website (click here).

LEAF-1401, a new generation onco-immuno antimetabolite designed to disrupt dysregulated 1-carbon metabolism in cancer and the immune system, is a liposomal formulation of gamma L-pentaglutamated pemetrexed. Gamma L-polyglutamated pemetrexed has been shown to be 80-times more potent than pemetrexed in inhibiting thymidylate synthase. In Oct 2018, LEAF received positive feedback following Pre-Investigational New Drug (Pre-IND) interactions with the United States Food and Drug Administration (US FDA) about LEAF-1401, where the Agency indicated that LEAF-1401 may be acceptable for development and registration under 505(b)(2) regulatory pathway. In addition, the Agency also provided guidance on how to establish a "bridge", between this product and Alimta, the US FDA approved listed drug, for the purpose of fulfilling the 505(b)(2) registration path requirements.

"The selection of LEAF-1401 by the ASCO (Free ASCO Whitepaper) Science Committee for presentation at this year’s annual ASCO (Free ASCO Whitepaper) meeting marked an important milestone in the recognition, by world’s leading cancer experts, of the future role this new investigational product is expected to play in the treatment of cancer," says Founder, President, and CEO of L.E.A.F. Pharmaceuticals, Dr. Clet Niyikiza.

"Although pemetrexed remains a backbone of treatment regimens approved for lung cancer, an unacceptable number of lung cancer patients treated with pemetrexed alone or in combination with novel drugs, such as immunotherapy, eventually succumb to this disease," says Dr. Victor Moyo, Executive Vice President, Global Head of Research and Development and Chief Medical Officer of L.E.A.F. Pharmaceuticals. Dr. Moyo added, "The results from LEAF-1401 preclinical studies, in which significantly higher levels of pemetrexed and the more potent pentaglutamated pemetrexed are delivered to the tumor with an increased antitumor effect, point to a high likelihood that LEAF-1401 could achieve improved outcomes for patients with lung cancer."

On June 9, 2020 Menarini Silicon Biosystems, the pioneer of liquid biopsy and single cell technologies, reported that results from a new study presented at the virtual 2020 meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) suggest that circulating tumor cell (CTC) counts may be useful in determining long-term prognosis and guiding treatment selection in patients with metastatic castrate sensitive prostate cancer (mCSPC) (Press release, Menarini Silicon Biosystems, JUN 9, 2020, View Source [SID1234560952]). A second study showed predictive value of CTC counts in metastatic breast cancer. Researchers used Menarini’s CELLSEARCH Circulating Tumor Cell test,* considered the gold standard in liquid biopsy technology, to detect and count CTCs.

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A current challenge in treating mCSPC is the lack of accurate biomarkers that indicate which patients will do well with particular therapies, or how long patients will live, according to lead researcher, Amir Goldkorn, M.D., Associate Professor of Medicine at the University of Southern California’s Keck School of Medicine. With this study, researchers determined CTC counts are a non-invasive way to obtain valuable prognostic information at the start of treatment.

"These findings have important clinical implications, suggesting that patients with high initial CTC counts are less likely to respond and more likely to progress on hormonal therapy," said Dr. Goldkorn. "Though additional analysis is required, these results indicate that CTCs could become a valuable biomarker that can tell us about a patient’s long-term prognosis and help guide therapy."

The study (Abstract #5506) investigated mCSPC patients early in the disease, when participants were first being treated with hormone therapy. Researchers enumerated CTCs in 1200 men at the start of the study, and then looked at two endpoints: the level of prostate-specific antigen (PSA) after seven months, and progression-free survival (PFS) after two years.

The results showed clear prognostic value for the CTC count. The 63% of men who had no circulating tumor cells when the study began were more than six times more likely at seven months to have PSA values below 0.2, which has been shown to be highly correlated with longer survival times than higher PSA values. The men also were 3.7 times more likely to survive, with no cancer progression, after two years.

In addition to Dr. Goldkorn’s oral presentation on the study, the research was included in a live discussion among a panel of experts on the ASCO (Free ASCO Whitepaper) meeting site on Sunday, May 31.

The second study, presented as a poster at ASCO (Free ASCO Whitepaper) (Abstract #1028), examined the role of CTC counts and mutations in circulating tumor DNA (ctDNA) in predicting prognosis, treatment response and disease spread in metastatic breast cancer (MBC). Led by Massimo Cristofanilli, M.D., F.A.C.P., Associate Director for Translational Research at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, the researchers looked at 36 Stage III and 203 Stage IV breast cancer patients.

They found that CTC counts were much higher in the Stage IV patients—an average of 62.2 cells per 7.5 mL of blood compared to 14.5 cells in Stage III patients — and that within each group, high CTC counts predict worse outcomes. In addition, they discovered that mutations in one particular gene in ctDNA — known as PI3KCA — dramatically increased in Stage IV patients compared to Stage III patients, and were also highly predictive of worse prognosis and treatment outcomes.

"These new studies demonstrate the important role our rare cell technologies play in advancing precision medicine research, which could one day translate to better, more personalized treatment options for patients with prostate and breast cancer," said Fabio Piazzalunga, President and CEO of Menarini Silicon Biosystems, Inc.

CELLSEARCH is the first and only clinically validated blood test cleared by the 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 Food & Drug Administration for use in monitoring patients with MBC. The CELLSEARCH System is the most extensively studied CTC technology, with research published in more than 650 peer-reviewed publications.