On November 12, 2019 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing therapeutics and pharmaceutical products, reported financial results for the third quarter and nine months ended September 30, 2019 (Press release, CASI Pharmaceuticals, NOV 12, 2019, View Source [SID1234550949]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "We launched EVOMELA in China during the third quarter and completed our transition to a commercial organization. The sales revenue since the launch and the demand for the drug were greater than our initial expectations and we are now working with our partner and manufacturer to accelerate the production timeline of EVOMELA due to this demand. Our successful launch was driven by our strong commercial team which continues to gain significant insight into the hematology oncology market which we believe will be invaluable as we continue launching additional products in this space. We are excited about our momentum and will continue evaluating additional strategic assets to tactically grow our pipeline."

Third Quarter 2019 Financial Results

·Revenues consisted primarily of product sales of EVOMELA that launched in August 2019. Revenue from EVOMELA was $2.7 million for the third quarter 2019. As commercial sales began in August 2019, revenue for the third quarter 2019 only consisted of approximately 1.5 months of sales.

·Costs of goods sold were $2.6 million for the third quarter 2019. Cost of goods sold have been impacted by a transitional supply agreement that is in the process of being modified with an alternate manufacturer. Cost of goods sold also was impacted by certain non-recurring charges associated with the startup production for the commercial launch. With the alternate supply line and the passing of start-up related charges, we expect the Cost of goods sold to be considerably reduced in the future.

CASI Pharmaceuticals, Inc. / 9620 Medical Center Drive / Suite 300 / Rockville, MD 20850

Phone 240.864.2600 / Fax 301.315.2437

·Research and development expenses for the third quarter 2019 were $1.8 million, compared with $1.8 million for the same period in 2018.

·General and administrative expenses for the third quarter 2019 were $8.0 million, compared with $6.9 million for the same period in 2018. The increase is primarily due to support costs related to the launch of EVOMELA that occurred during August 2019.

·Selling and marketing expenses for the third quarter 2019 were $975,000. The increase is due to selling cost related to commercial sales of EVOMELA that began during August 2019.

·Net loss for the third quarter 2019 was $9.7 million compared to $8.8 million for the same period in 2018.

·As of September 30, 2019, CASI had cash and cash equivalents of $63.2 million compared to $84.2 million as of December 31, 2018. The decrease in cash is primarily due to the Black Belt and Juventas investments made during the second quarter 2019, along with normal operating expenses.

Further information regarding the Company, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, can be found at www.casipharmaceuticals.com.

About EVOMELA

EVOMELA (melphalan hydrochloride for injection) is an alkaline drug that is approved and in China is indicated for use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma, and for the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate. EVOMELA was previously granted priority review by the China NMPA because multiple myeloma is classified as a rare disease in China, there is no melphalan in any formulation available in China to address this unmet medical need; and EVOMELA has clear therapeutic advantage to currently available therapeutics.

On November 12, 2019 Coherus BioSciences, Inc. ("Coherus", Nasdaq: CHRS), reported that senior management will present at two upcoming investor healthcare conferences (Press release, Coherus Biosciences, NOV 12, 2019, View Source [SID1234550948]).

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Management will deliver a company presentation at the Jefferies 2019 London Healthcare Conference on Thursday, November 21st at 9:20 a.m. GMT taking place in London, UK.
Management will deliver a company presentation at the Piper Jaffray 31st Annual Healthcare Conference on Tuesday, December 3rd at 1:30 p.m. ET taking place in New York, New York.
The audio portion of the presentations will be available on the investors page of the Coherus BioSciences website at View Source

On November 12, 2019 Applied Therapeutics, Inc. (NASDAQ: APLT) (the "Company"), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported it has entered into a definitive securities purchase agreement, dated as of November 7 2019, for the sale of its common stock, par value $0.0001 per share (the "Shares"), in a private placement (the "Private Placement") expected to result in gross proceeds to the Company of approximately $20 million, before deducting placement agent commissions and other offering expenses (Press release, Applied Therapeutics, NOV 12, 2019, View Source [SID1234550947]).

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The Private Placement is expected to close on or about November 12, 2019, subject to the satisfaction of customary closing conditions. Additional details regarding the Private Placement will be included in a Form 8-K to be filed by the Company with the Securities and Exchange Commission ("SEC").

The Company intends to use the net proceeds to fund development of its drug candidates.

Cowen and UBS Investment Bank acted as placement agents in the transaction (the "Placement Agents"). Skadden, Arps, Slate, Meagher & Flom LLP acted as legal counsel for the Company, and Davis Polk & Wardwell LLP acted as legal counsel for the Placement Agents.

The securities being sold in the Private Placement have not been registered under the Securities Act of 1933, as amended, or state securities laws and may not be offered or sold in the United States absent registration with the SEC or an applicable exemption from such registration requirements. The Company has agreed to file a registration statement with the SEC covering the resale of the Shares issuable in connection with the Private Placement.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, 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.

On November 12, 2019 Neon Therapeutics, Inc. (Nasdaq: NTGN), a clinical-stage immuno-oncology company developing neoantigen-based therapeutics, reported financial results for the third quarter ended September 30, 2019 and provided a business update (Press release, Neon Therapeutics, NOV 12, 2019, View Source [SID1234550946]).

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"We continue to make important progress in advancing our leadership in the field of neoantigen-targeted therapies and are pleased to have recently presented updates across our personalized cancer vaccine and adoptive T cell therapy programs at the SITC (Free SITC Whitepaper) Annual Meeting. In the remaining months of 2019, we look to continue building on our leadership position by completing our process development work that will enable our planned Clinical Trial Application in Europe for Phase 1 development of our neoantigen-targeted T cell therapy candidate, NEO-PTC-01," said Hugh O’Dowd, Neon’s Chief Executive Officer.

Recent Highlights

RECON Bioinformatics Platform: Neon scientists published in the scientific journal Immunity a breakthrough process for predicting which neoantigens will be presented by MHC class II molecules in the tumor microenvironment.

Predicting the relevant cancer-specific antigens is a crucial precursor to developing immunotherapies that effectively train T cells to traffic to the tumor and destroy malignant cells. In the paper, titled "Defining HLA-II ligand processing and binding rules with mass spectrometry enhances cancer epitope prediction," Neon’s proprietary mono-allelic profiling technology called MAPTAC facilitated the development of convolutional neural network-based predictors.

These algorithms achieved up to a 61-fold improvement in predicting MHC class II peptides compared to publicly available tools.

This MHC class II technology will be integrated into Neon’s RECON bioinformatics platform and is expected to improve the efficacy of immunotherapies developed by Neon by predicting recruitment of CD4+ T cells, which are believed to be in controlling tumor growth.

NEO-PV-01: Neon presented updated results at the SITC (Free SITC Whitepaper) Annual Meeting from the ongoing, multicenter Phase 1b NT-001 clinical trial evaluating NEO-PV-01, Neon’s personal neoantigen vaccine candidate, in combination with OPDIVO (nivolumab) in patients with advanced or metastatic melanoma, smoking-associated non-small cell lung cancer (NSCLC) and bladder cancer.

Across all three distinct tumor types, results demonstrated prolonged and consistent improvements in progression-free survival (PFS) and overall survival (OS) that compare favorably to that observed with checkpoint inhibitor monotherapy, based on historical benchmark data.

Further, neoantigen-specific immune responses and epitope spread to RECON-predicted targets were associated with longer PFS, and major pathological responses post-administration of NEO-PV-01 in melanoma patients were also associated with longer PFS.

The safety data for NT-001 were consistent with the safety profile for OPDIVO monotherapy. These updated results come from 82 patients who received at least one dose of OPDIVO in the Phase 1b NT-001 trial.

NEO-PTC-01: Neon presented at the SITC (Free SITC Whitepaper) Annual Meeting an update on preclinical and process development work for NEO-PTC-01, its personal neoantigen-targeted T cell therapy candidate consisting of multiple T cell populations targeting the most therapeutically relevant neoantigens from each patient’s tumor.

NEO-PTC-01 leverages Neon’s RECON bioinformatics platform to individually select a set of neoantigen targets for each patient, and NEO-STIM, its proprietary process to directly prime, activate and expand neoantigen-targeting T cells ex vivo. Neon believes that this approach will allow NEO-PTC-01, a non-engineered product that leverages peripheral blood mononuclear cells (PBMCs) as starting material, to specifically target each patient’s individual tumor with T cells that can drive a robust and persistent anti-tumor response.

In the SITC (Free SITC Whitepaper) update, Neon demonstrated that it can reproducibly generate a potent T cell product from PBMCs of melanoma patients, as well as at therapeutic scale using a healthy donor sample. This process development work showed that NEO-PTC-01 induced multiple CD8+ and CD4+ T cell responses from both the memory and the naïve T cell compartments.

Neon is focusing the initial clinical development of NEO-PTC-01 in patients with solid tumors that are refractory to checkpoint inhibitors. Neon expects to file a clinical trial application, or CTA, in Europe by the end of 2019 to evaluate NEO-PTC-01 in the solid tumor setting.

Expected Near-Term Milestones

NEO-PTC-01: Planned European CTA filing to evaluate NEO-PTC-01 in a refractory solid tumor setting (2H 2019).

NEO-PTC-01: Planned Phase 1 initiation in a refractory solid tumor setting (1H 2020).

NEO-PV-01: Planned clinical results and correlative immune data, including 12-month follow-up, from NT-002 Phase 1b trial in first-line metastatic NSCLC (Q3 2020).

Third Quarter 2019 Financial Results and Financial Guidance:

R&D Expenses: Research and development expenses were $14.1 million for the third quarter of 2019, compared to $14.4 million for the same period last year. The decrease was primarily due to a reduction in clinical development and manufacturing costs, partially offset by an increase in personnel-related costs and costs related to the preparation for the planned CTA filing in Europe for Phase 1 development of NEO-PTC-01.

G&A Expenses: General and administrative expenses were $5.1 million for the third quarter of 2019, compared to $4.6 million for the same period last year. The increase was primarily due to personnel-related costs and costs associated with being a public company.

Net Loss: Net loss was $19.0 million for the third quarter of 2019, compared to $18.4 million for the same period last year.

Cash Position: As of September 30, 2019, cash, cash equivalents and marketable securities were $44.3 million, as compared to cash, cash equivalents and marketable securities of $103.3 million as of December 31, 2018.

Financial Guidance: Based on its current operating plan, Neon expects that its existing cash and cash equivalents will enable the Company to fund its operating expenses and capital expenditure requirements into June 2020.

OPDIVO is a registered trademark of Bristol-Myers Squibb Company.

On November 12, 2019 PharmaCyte Biotech (OTCQB: PMCB) reported that has reduced a long list of FDA-required items to just one last important component—release testing—before it can submit an Investigational New Drug application (IND) to the U.S. FDA (Press release, PharmaCyte Biotech, NOV 12, 2019, View Source [SID1234550944]). PharmaCyte’s IND application will request permission from the FDA to conduct a Phase 2b clinical trial in locally advanced, inoperable pancreatic cancer (LAPC). With the FDA’s approval to begin a clinical trial, PharmaCyte’s opportunities for funding improve dramatically.

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PharmaCyte has already conducted and completed a long list of tests and entered the data from those tests into the IND application. PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said of completing the IND for submission to the FDA, "Most of the work needed for PharmaCyte to submit an IND to the FDA has been completed."

PharmaCyte recently announced that it has encapsulated the live cells from its Master Cell Bank (MCB) into its live-cell encapsulation technology, Cell-in-a-Box, in what is the second of two staggered and back-to-back manufacturing runs. In that announcement the company’s CEO stated that after reviewing pictures of the growing cells, he is confident that those cells are growing as well as those in the first run, which ended successfully after a host of changes were implemented to improve the manufacturing process.

And, with the second manufacturing run well underway and growing as expected, this final run should end in success over the next week or so, and then the release testing for this second run combined with the release testing from the first run will complete what has been a long and arduous path to submitting the much-anticipated IND.

Now, let’s walk through each of the tests (release testing) that are required by cGMP Validation, the company that is taking responsibility for "releasing" the clinical trial product into the U.S. for use in PharmaCyte’s upcoming clinical trial. Many of these tests are also FDA-required tests that must be completed in order to obtain the data necessary to complete and submit the IND and for PharmaCyte to receive a Certificate of Analysis (CoA) from Austrianova. A CoA is a document issued by Austrianova’s Quality Assurance and Quality Control Department (QA/QC) that confirms that PharmaCyte’s product meets its product specifications. The CoA will contain the actual results obtained from the testing performed as part of the QA/QC of a representative sample of PharmaCyte’s clinical trial product.

After a manufacturing run is completed successfully, 300 Cell-in-a-Box capsules are placed into 400 of PharmaCyte’s clinical trial syringes and then frozen. A representative sample of those syringes will be thawed and undergo release testing.

All release testing related to "safety" of the encapsulated cells is being outsourced by PharmaCyte to independent third-party laboratories in Europe. They include tests for sterility, endotoxin, pH, mycoplasma and cell identity.

A sterility test confirms that all syringes are free from the presence of viable microorganisms or microbial contamination.

An endotoxin test of a biologic product is necessary to ensure the product is endotoxin free. This means that the cells do not contain bacterial toxins.

A pH test will confirm how much hydrogen is in the product and that the product meets the specified pH balance for a human being.

A mycoplasma test confirms that the syringes are free from mycoplasma. Mycoplasma are small, simple bacteria which lack a cell wall and represent one of the most prevalent and serious sources of cell line contamination.

A cell identity test confirms that the cell line used in production is positively identified as the 22P1G cell line. This is the human cell line that PharmaCyte cloned from the original cell line used in the first two clinical trials using the Cell-in-a-Box technology.

All release testing related to "functionality" of the encapsulated cells is being conducted by Austrianova at its GMP facility in Thailand. Those tests include enzymatic activity, cell viability, capsule count in each syringe, and capsule diameter.

An enzymatic activity test shows that the requisite enzymes are being produced by the cells inside the capsules to create the desired chemical reaction in the body. In PharmaCyte’s case, this means that the correct enzymes are being produced to convert the cancer-killing prodrug, ifosfamide, from its inactive form to its active form. Ifosfamide is the chemotherapy drug that PharmaCyte uses with the encapsulated cells that are implanted in a patient as close to the tumor as possible.

A cell viability test measures the number of cells that remain alive and viable after thawing a syringe of encapsulated cells.

A capsule count test confirms that each syringe contains 300 capsules. One syringe full of the encapsulated cells is implanted in a pancreatic cancer patient before ifosfamide is given intravenously to treat LAPC.

A capsule diameter test confirms that the diameter of each capsule inside a syringe of encapsulated cells meets a specified range.

Additionally, a pyrogenicity test must be completed to ensure that the capsules themselves do not produce a fever. This FDA-required test is not in the list of release tests. The pyrogenicity test is being conducted in the United States.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, watch the company’s documentary video complete with medical animations at: View Source