On March 30, 2020 Heat Biologics, Inc. ("Heat") (NASDAQ: HTBX), a clinical-stage biopharmaceutical company specialized in the development of novel therapeutic and prophylactic vaccines, including one for coronavirus COVID-19, reported that financial, clinical and operational updates for the year ended December 31, 2019 (Press release, Heat Biologics, MAR 30, 2020, View Source [SID1234555981]).

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Jeff Wolf, Chief Executive Officer of Heat Biologics, commented, "2019 was an exciting year for Heat, as we advanced our therapeutic pipeline. This past November, we presented additional positive top line Phase 2 data of HS-110 plus BMS checkpoint inhibitor nivolumab (Opdivo) in advanced NSCLC lung cancer patients at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy. We are highly encouraged by the data as we advance partnership and collaboration discussions. Earlier this year, we announced dosing the first patient in a Phase 1 clinical trial of our co-stimulatory HS-130, in combination with HS-110, for patients with advanced solid tumors refractory to standard of care, which marks a key milestone for Heat."

"We recently announced plans to develop a vaccine for preventing infection from the SARS-CoV-2 coronavirus that causes COVID-19 utilizing our robust gp-96 vaccine platform in collaboration with the University of Miami. Heat’s gp96 platform has undergone rigorous testing as a vaccine against SIV/HIV, malaria, zika and other infectious diseases in numerous National Institutes of Health (NIH) and Department of Defense (DOD)-funded mice and primate trials and has been tested in over 300 patients in multiple NIH and Heat-funded oncology trials. We believe this platform has the potential to provide broad protection against COVID-19, and possible future mutations of COVID-19 or other coronaviruses. We are encouraged by the prospects for this program and are moving forward with our partners to advance this vaccine as quickly as possible."

"Earlier this month we also announced a collaboration with the University of Miami to develop a proprietary COVID-19 point-of-care diagnostic test. The new paper-based test is being designed to provide a read-out in a fraction of the time required for most other tests, have no technical hardware requirements, have low cost of goods, be easily manufactured at scale, and provide binary readout if the patient is positive for a disease within 30 minutes. Unfortunately, current diagnostic tests, are in short supply and often take days for results. This diagnostic is being designed for early detection to provide critical and time-sensitive information to help curb the spread of the disease."

Mr. Wolf concluded, "We share our sympathies to those individuals and families impacted by COVID-19 and are committed to helping find solutions to this global pandemic. I’d like to thank all of the Heat and University of Miami professionals that have worked tirelessly to advance our therapeutic and diagnostic platforms. Importantly, we have over $25 million of cash and short-term investments as of March 23, 2020, which should put us on a solid financial footing as we advance our programs. Moreover, we believe that upcoming catalysts and milestones have the potential to drive significant shareholder value."

2019 Financial Results

Research and development expenses decreased by 20% to $13.0 million for the year ended December 31, 2019 compared to $16.2 million for the year ended December 31, 2018. The variance of approximately $3.2 million is primarily due to reduced clinical trial expenses related to HS-110 patient enrollment completion.

General and administrative expense increased approximately 36% to $9.5 million for the year ended December 31, 2019 compared to $7.0 million for the year ended December 31, 2018. The variance of $2.5 million is primarily due to the increase in personnel and stock compensation expense.

Net loss attributable to Heat Biologics was approximately $20.0 million, or ($0.60) per basic and diluted share for the year ended December 31, 2019 compared to a net loss of approximately $15.7 million, or ($0.90) per basic and diluted share for the year ended December 31, 2018.

As of December 31, 2019, the Company had approximately $14.8 million in cash, cash equivalents and short-term investments.

On March 30, 2020 Eagle Pharmaceuticals, Inc. ("Eagle" or the "Company") (NASDAQ: EGRX) reported that its marketing partner SymBio Pharmaceuticals Limited ("SymBio") has completed patient enrollment in a clinical trial for TREAKISYM rapid infusion ("RI"), a liquid bendamustine injection with a 10-minute administration time, in Japan (Press release, Eagle Pharmaceuticals, MAR 30, 2020, View Source [SID1234555980]). The study’s primary objective is to confirm the safety of the RI product. SymBio expects to obtain regulatory approval in the second half of 2022.

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SymBio intends to submit a New Drug Application ("NDA") for the RI product for all indications for which TREAKISYM is currently approved (low-grade non-Hodgkin’s lymphoma, mantle cell lymphoma, and chronic lymphocytic leukemia). As previously disclosed, SymBio filed an NDA for its TREAKISYM ready-to-dilute ("RTD") product in October 2019.

In September 2017, Eagle licensed to SymBio intellectual property necessary to develop, market and sell RTD and RI formulations of TREAKISYM in Japan. As part of the agreement, SymBio assumed responsibility for securing regulatory approval of the TREAKISYM RTD and RI injection products using the licensed technology in Japan.

Pursuant to the terms of the license with SymBio, Eagle received a $12.5 million upfront milestone payment in 2017, and is entitled to additional milestone payments, including $5 million upon approval of an NDA for either TREAKISYM RTD or RI, and other amounts upon achievement of cumulative sales thresholds. Eagle will also receive royalties on future net sales of the licensed bendamustine products.

According to SymBio, sales in Japan for TREAKISYM were $78 million in 2019.

"SymBio continues to make excellent progress in advancing TREAKISYM RTD and RI for approval in Japan as evidenced by the completion of enrollment of the safety study for the RI product. With anticipated future approvals for both the TREAKISYM RTD and RI products, we look forward to SymBio’s successful commercialization in Japan, allowing patients to benefit from these products’ key advantages," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.

On March 30, 2020 Bio-Techne Corporation (NASDAQ:TECH) reported researchers from its Munich-based laboratory, Exosome Diagnostics, in a paper published in the scientific Journal of Extracellular Vesicles (JEV) (link), showed that tumor- derived RNA was not detected in thrombocytes in samples where the tumor RNA was readily detected in exosomes (Press release, Bio-Techne, MAR 30, 2020, View Source [SID1234555979]). This finding holds significant implications for developers of liquid biopsies for cancer.

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The results from this study raise doubts about the commonly held belief that platelets include abundant tumor RNA that can be used for tumor specific RNA analysis, a process named Tumor Educated Platelets (TEPs). This has important implications for the use of platelets as a potential technology platform for liquid biopsies that aim to detect or monitor molecules of tumor origin in a biofluid sample. Such platelet-based strategies have recently been proposed by other companies and academic labs in the field.

"We wanted to find the most sensitive way to detect tumor specific RNA and were looking for ways to increase our clinical sensitivity. In light of previous publications on TEPs, we isolated RNA from exosomes as well as platelets from the same samples in hopes of improving the detection. We learned that combining the sample gave more RNA, but actually decreased the sensitivity for mutation assays," said Dr. Johan Skog, Chief Scientific Officer at Exosome Diagnostics, a Bio-Techne brand. "This finding led to this study quantifying the amount of tumor specific RNA in the platelets, if any, compared to other normal RNA". The researchers in this study applied a sensitive mutation assay but were unable to detect any tumor RNA in the platelet fraction, even when it was readily detected in the exosome fraction from the same sample. This casts doubt that keeping the platelet fraction adds value when looking at single nucleotide mutations on RNA. "There is plenty of RNA in platelets," continued Dr. Skog, "far more than in exosomes… but it does not come from the tumor."

In this carefully executed study, involving prospective collection of blood samples from patients with melanoma, whose tumor tissue had been confirmed to carry the BRAF V600E mutation, the researchers directly compared the RNA mutation signals detectable in exosomes to platelets isolated from the same blood sample. Using an exceptionally sensitive assay for detection of the BRAF V600E RNA, the mutation could be detected in 10 out of 12 exosome fractions, but not in a single platelet preparation. The researchers conclude in the article, that based on their findings, the differences in platelet RNA expression, previously published between cancer patients and normal healthy controls are unlikely to be of tumor origin. The researchers further conclude that as a consequence of their findings, it is recommended for most cases to remove platelets from a blood specimen for optimal detection of tumor-derived RNA in extracellular vesicles.

On March 30, 2020 BerGenBio, a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for multiple cancer indications, reported that Richard Godfrey, Chief Executive Officer, will present at the upcoming Solebury Trout Virtual Investor Conference (Press release, BerGenBio, MAR 30, 2020, View Source [SID1234555978]). The 25 minute presentation includes an interactive Q&A with participants prompted to submit questions electronically. Details are as follows:

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Date/Time: Tuesday March 31, at 11am EDT

Access: https://78449.themediaframe.com/dataconf/productusers/solebury/mediaframe/36522/indexl.html

Webcast archive: 24 hours following the presentation an archive copy of the presentation will be available on the Company’s website at View Source

On March 30, 2020 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported recent business highlights and full year 2019 financial results (Press release, Beam Therapeutics, MAR 30, 2020, View Source [SID1234555977]).

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"So far in 2020, we have made strides across all aspects of our business, and despite the many uncertainties we face today amid the COVID-19 pandemic, we remain committed to moving our programs forward for patients," said John Evans, chief executive officer of Beam. "Following the successful completion of our initial public offering and the recent additions to our team, we have the resources needed to steadily advance a broad and deep portfolio of base editing programs addressing a wide range of disease areas and to invest in the science of base editing, illustrated by the recent platform technology publications. As we look ahead, we are planning for additional scientific publications throughout the year and remain on track to file an initial wave of IND applications beginning in 2021, in an effort to bring a new class of precision genetic medicines to the clinic and, ultimately, to patients."

Recent Business Highlights

Leadership Team Strengthened with Key Appointments: Since the beginning of 2020, Beam has named several appointments and promotions aimed at further supporting advancement of its pipeline and expanding its business operations. These include the promotion of Giuseppe "Pino" Ciaramella, Ph.D., to president and chief scientific officer, the promotion of Manmohan Singh, Ph.D., to senior vice president of pharmaceutical sciences and delivery technologies, and the appointment of Christine Swenson as senior vice president of regulatory affairs.

Advancements in Base Editing Technology Platforms Highlighted in Scientific Publications: New research on Beam’s novel adenine base editor (ABE) and cytosine base editor (CBE) technologies were reported in multiple scientific publications. Data from these internal and collaborator publications further support that Beam’s continued evolution of ABEs and CBEs can enhance the precision of targeted A-to-G and C-to-T DNA base changes, potentially improving the range of therapeutic applications.

In a paper entitled, "ABE8: Superior adenine base editors with expanded targeting range, higher activity, and therapeutic application", accepted for publication in Nature Biotechnology, Gaudelli, et al., highlight continued evolution of Beam’s adenine base editors. These proprietary, next-generation "ABE8 variants" demonstrated improved therapeutic potential compared to previously published versions, including increased on-target activity while maintaining low-levels of off-target editing across a number of cell types, including transformed and human primary cells.

In a paper entitled, "Next-generation cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity," accepted for publication in Nature Communications by Yu, et al., highlighted proprietary next-generation versions of CBEs. Off-target editing rates with the first published version of CBEs, known as BE3, are low and within mutation rates that naturally occur with no consequence in somatic cells. These next-generation CBEs display comparable on-target editing to BE3 while further reducing the low levels of off-target editing seen with BE3.

Two papers published in Nature Biotechnology by Beam co-founder Dr. David Liu, highlight novel CBEs that diminish or eliminate the low levels of off-target editing observed with BE3s, and novel variants of base editors that are able to bind a wider range of genetic targets, expanding their therapeutic potential.

Oversubscribed $207M Initial Public Offering Completed: On February 11, 2020, Beam announced the closing of its initial public offering of 12,176,471 shares of common stock, including the exercise in full by the underwriters of their option to purchase up to 1,588,235 additional shares of common stock, at a public offering price of $17.00 per share. The net proceeds to Beam Therapeutics from the offering were approximately $188.3 million, after deducting underwriting discounts and commissions and estimated offering expenses.

Business Continuity: Beam is executing its business objectives for 2020 and beyond, while closely monitoring the impact of the COVID-19 situation. The company has established a remote operating model while prioritizing the most critical research and development activities needed to advance its portfolio of novel base editing programs.

Full Year 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $91.8 million as of December 31, 2019, which does not include the $188.3 million in net proceeds from the company’s IPO completed in February 2020. In addition, cash used to fund our operations was $72.0 million for the year ended December 31, 2019.

R&D Expenses: Research and development (R&D) expenses were $54.6 million for the full year ended December 31, 2019.

G&A Expenses: General and administrative (G&A) expenses were $20.6 million for the full year ended December 31, 2019.
Net Loss: Net loss was $78.3 million for the full year ended December 31, 2019.