On August 16, 2019 Celyad (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell-based therapies, reported that the Company will report first half 2019 financial results and business highlights on the evening of Thursday, August 22, 2019 (Press release, Celyad, AUG 16, 2019, View Source [SID1234538816]).

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Following the press release, Celyad management will host a conference call on Friday, August 23 at 2pm CEDT / 8am EDT to discuss first half 2019 results and provide an update on the Company’s recent progress and upcoming milestones.

Participants may access the conference call by dialing any of the following numbers:

Belgium, Brussels +32 (0) 24 01 70 35

France, Paris +33 (0)1 76 72 89 28

United States: +1 917 720 0181

International: +44 (0) 2071 928501

Conference ID: 3547725

On August 15, 2019 Hemispherx Biopharma, Inc. (NYSE American: HEB), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers and immune-deficiency diseases, reported the filing of its report 10Q for the second quarter, which ended June 30, 2019, and provides a business update (Press release, AIM ImmunoTech, AUG 15, 2019, View Source [SID1234540960]).

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Management Commentary

"The company’s stockholder equity increased by $2,618,000 during the second quarter to $6,548,000 and we expect to continue on this positive trajectory. We look forward to the results from our ongoing clinical trials over the next 18 months, seeing them as strong value drivers for the stock. Thus far, we have had strong, steady and consistent clinical progress in immuno-oncology. I believe this is critical to creating long-term stockholder value, as success in any one of our ongoing or upcoming clinical trials has the potential to be a strong market catalyst," said Hemispherx CEO Thomas K. Equels. "Especially significant are the larger clinical trials, such as our Phase 2 recurrent ovarian cancer trial combining Ampligen and pembrolizumab. All our trials are paid for by third parties without Hemispherx conceding any rights for development. I believe that third parties’ fronting of trial costs shows that it’s not just Hemispherx that believes in Ampligen, but that major cancer research centers, governmental grantors and the pharmaceutical industry grantors also see the potential of Ampligen as a combination therapy."

Balance Sheet Highlights

As of June 30, 2019, Hemispherx had cash, cash equivalents and marketable securities of $2,864,000 compared with $1,825,000 as of December 31, 2019.

Second Quarter Financial Highlights

Research and development manufacturing-related expenses for the second quarter of 2019 were $1,096,000, compared with $1,341,000 million for the second quarter of 2018. The decrease was due primarily to a general reduction of Ampligen manufacturing costs.

General and administrative expenses for the second quarter of 2019 were $1,942,000, compared with $1,733,000 for the second quarter of 2018. The increase was mainly due to an increase in stock compensation expense and an increase in public relations expenses.

The net loss from continuing operations for the second quarter of 2019 was $2,029,000, or $1.07 per share, compared with $2,415,000, or $2.38 per share, for the second quarter of 2018.

Ongoing Ampligen Clinical Studies

Advanced Recurrent Ovarian Cancer – A Phase 1/2 clinical trial of intraperitoneal chemo-immunotherapy in advanced recurrent ovarian cancer. This trial is being conducted at the University of Pittsburgh Medical Center by Dr. R. Edwards. Eleven patients in the Phase 1 portion have been enrolled; 10 have completed treatment and one is undergoing treatment and evaluation. An interim report from Dr. Edwards’ team was received and they are drafting a manuscript on the biological and clinical outcomes from the Phase 1 portion of the trial. Dr. Edwards’ team has issued an updated report supporting the clinical safety of using Ampligen on an intraperitoneal basis in combination therapy, thus allowing a large Phase 2 trial combining Ampligen and pembrolizumab to begin. Separately, Dr. Edwards has reported that the Phase 1/2 clinical trial generated positive survival data, to be addressed in the manuscript. See ClinicalTrials.gov.
Advanced Recurrent Ovarian Cancer – A follow-up study to the Phase 1/2 above is a 45-patient, Phase 2 clinical trial combining Ampligen, cisplatin and pembrolizumab in the treatment of advanced recurrent ovarian cancer. This trial is being conducted at the University of Pittsburgh Medical Center by Dr. R. Edwards. The first patient was treated on June 11, 2019 and treatment of patients is underway. See ClinicalTrials.gov.
Stage 4 Colorectal Cancer Metastatic to the Liver – A 12-patient, Phase 2a clinical trial combining Ampligen, Intron A and celecoxib in Stage 4 colorectal cancer. This trial is being conducted at Roswell Park Comprehensive Cancer Center by Dr. P. Boland. To date, seven subjects have been enrolled and completed treatment, and the trial is progressing well. Data from this trial is expected in 2020. See ClinicalTrials.gov.
Stage 4 Metastatic Triple-Negative Breast Cancer – A Phase 1, 6-patient, open-label clinical trial combining Ampligen, Intron A, celecoxib and pembrolizumab in the treatment of Stage 4 triple-negative breast cancer. This trial is being conducted at Roswell Park Comprehensive Cancer Center by Dr. M. Opyrchal. Patients have been enrolled and are in early treatment. See ClinicalTrials.gov.
Early-Stage Prostate Cancer – A 60-patient, Phase 2 randomized, three-arm clinical trial of aspirin and Ampligen with or without Intron A compared with no drug treatment in patients with early-stage prostate cancer before undergoing radical prostatectomy. This trial is being conducted at Roswell Park Comprehensive Cancer Center by Dr. G. Chatta. IRB and FDA approval have been received. Roswell expects first enrollment in the immediate future. See ClinicalTrials.gov.
Expanded Access Program

Advanced Pancreatic Cancer – An Early Access Program (EAP) approved by the Inspectorate of Healthcare in the Netherlands for Stage 4 pancreatic cancer at Erasmus Medical Center has been ongoing for two years. The team at Erasmus is making progress under the supervision of Prof. Casper van Eijck, MD, a world-renowned specialist in this dread malignancy, and Diba Latifi, MD. The Dutch government has approved and extended the therapeutic program for an additional year. Early progress was reported in a published abstract from Erasmus. The abstract was part of a larger original report covering a variety of medical topics, which can be found at View Source The last two patients planned to be enrolled for a planned paper are nearing the end of treatment. The paper analyzing the data is expected in 2020.

Clinical trials planned for initiation in 2019/2020

Stage 4 Brain Metastatic Breast Cancer – A multi-site Stage 4 brain metastatic breast cancer study testing Ampligen as an immunological agent increasing efficacy is awaiting funding.
Stage 4 Refractory Metastatic Colorectal Carcinoma – Phase 2 study that will evaluate Ampligen in combination with pembrolizumab in Stage 4 refractory metastatic colorectal carcinoma at Roswell Park Comprehensive Cancer Center. Dr. P. Boland, PI. Roswell Park is reviewing the budget and preparing it for institutional submission.
Stage 4 Urothelial (Bladder), Melanoma and Renal Cell Carcinoma – Phase 2 study of Stage 4 urothelial (bladder), melanoma and renal cell carcinoma, resistant to checkpoint blockade, that will evaluate Ampligen in combination with a checkpoint blockade therapy at Roswell Park Comprehensive Cancer Center. Protocol design currently being finalized. Hemispherx Biopharma signed a clinical trial agreement with Roswell Park Comprehensive Cancer Center to study Ampligen in combination with checkpoint inhibitors in a Phase 2a study in urothelial carcinoma, renal cell carcinoma and melanoma. Hemispherx is awaiting the study protocol, which is under review at Roswell Park, and confirmation of third-party funding.
Non-Small Cell Lung Cancer – First-line therapy for non-small cell lung cancer with SOC chemotherapy that will evaluate Ampligen in combination with pembrolizumab at University of Nebraska Medical Center. Dr. V. Ernani, PI. Study design and budget being developed. However, we now anticipate an extended delay at Nebraska because we were unable to obtain funding yet for the large trial. However, we are working on a pilot trial program at Roswell Park to create sufficient data for a large, multi-site non-small cell lung cancer Phase 2 clinical study.
Advanced Pancreatic Cancer – Phase 2 study in advanced pancreatic cancer using checkpoint blockade plus Ampligen at University of Nebraska Medical Center. Dr. K. Klute, PI. Protocol and budget being developed. Based upon success in the initial animal studies, an additional round of more extensive and comprehensive pre-clinical animal pancreatic cancer studies are being conducted at University of Nebraska to reconfirm results, test additional PC tumor types, examine anti-PD-1 in addition to the prior anti-PD-L1 analysis, then fine tune the focus of the proposed future pancreatic cancer clinical trial and reduce the chances of error in clinical trial design. This information will also be used to formulate proposed future combination therapy clinical activity in the Kingdom of the Netherlands.
Early-Stage Triple Negative Breast Cancer – Phase 1 study of chemokine modulation plus neoadjuvant chemotherapy in patients with early-stage triple negative breast cancer is awaiting FDA authorization. The objective of this study is to evaluate the safety and tolerability of a combination of Ampligen, celecoxib with or without Intron A, when given along with chemotherapy. The goal of this approach is to increase survival.

On August 15, 2019 The U.S. Food and Drug Administration granted accelerated approval to Rozlytrek (entrectinib), a treatment for adult and adolescent patients whose cancers have the specific genetic defect, NTRK (neurotrophic tyrosine receptor kinase) gene fusion and for whom there are no effective treatments (Press release, Bonnie J Addario Lung Cancer Foundation, AUG 15, 2019, View Source [SID1234538896]).

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"We are in an exciting era of innovation in cancer treatment as we continue to see development in tissue agnostic therapies, which have the potential to transform cancer treatment. We’re seeing continued advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine," said FDA Acting Commissioner Ned Sharpless, M.D. "Using the FDA’s expedited review pathways, including breakthrough therapy designation and accelerated approval process, we’re supporting this innovation in precision oncology drug development and the evolution of more targeted and effective treatments for cancer patients. We remain committed to encouraging the advancement of more targeted innovations in oncology treatment and across disease types based on our growing understanding of the underlying biology of diseases."

This is the third time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated. The approval marks a new paradigm in the development of cancer drugs that are "tissue agnostic." It follows the policies that the FDA developed in a guidance document released in 2018. The previous tissue agnostic indications approved by the FDA were pembrolizumab for tumors with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) tumors in 2017 and larotrectinib for NTRK gene fusion tumors in 2018.

"Today’s approval includes an indication for pediatric patients, 12 years of age and older, who have NTRK-fusion-positive tumors by relying on efficacy information obtained primarily in adults. The FDA continues to encourage the inclusion of adolescents in clinical trials. Traditionally, clinical development of new cancer drugs in pediatric populations is not started until development is well underway in adults, and often not until after approval of an adult indication," said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "Efficacy in adolescents was derived from adult data and safety was demonstrated in 30 pediatric patients."

The ability of Rozlytrek to shrink tumors was evaluated in four clinical trials studying 54 adults with NTRK fusion-positive tumors. The proportion of patients with substantial tumor shrinkage (overall response rate) was 57%, with 7.4% of patients having complete disappearance of the tumor. Among the 31 patients with tumor shrinkage, 61% had tumor shrinkage persist for nine months or longer. The most common cancer locations were the lung, salivary gland, breast, thyroid and colon/rectum.

Rozlytrek was also approved today for the treatment of adults with non-small cell lung cancer whose tumors are ROS1-positive (mutation of the ROS1 gene) and has spread to other parts of the body (metastatic). Clinical studies evaluated 51 adults with ROS1-positive lung cancer. The overall response rate was 78%, with 5.9% of patients having complete disappearance of their cancer. Among the 40 patients with tumor shrinkage, 55% had tumor shrinkage persist for 12 months or longer.

Rozlytrek’s common side effects are fatigue, constipation, dysgeusia (distorted sense of taste), edema (swelling), dizziness, diarrhea, nausea, dysesthesia (distorted sense of touch), dyspnea (shortness of breath), myalgia (painful or aching muscles), cognitive impairment (confusion, problems with memory or attention, difficulty speaking, or hallucinations), weight gain, cough, vomiting, fever, arthralgia and vision disorders (blurred vision, sensitivity to light, double vision, worsening of vision, cataracts, or floaters). The most serious side effects of Rozlytrek are congestive heart failure (weakening or damage to the heart muscle), central nervous system effects (cognitive impairment, anxiety, depression including suicidal thinking, dizziness or loss of balance, and change in sleep pattern, including insomnia and excessive sleepiness), skeletal fractures, hepatotoxicity (damage to the liver), hyperuricemia (elevated uric acid), QT prolongation (abnormal heart rhythm) and vision disorders. Health care professionals should inform females of reproductive age and males with a female partner of reproductive potential to use effective contraception during treatment with Rozlytrek. Women who are pregnant or breastfeeding should not take Rozlytrek because it may cause harm to a developing fetus or newborn baby.

Rozlytrek was granted accelerated approval. This approval commits the sponsor to provide additional data to the FDA. Rozlytrek also received Priority Review, Breakthrough Therapy and Orphan Drug designation. The approval of Rozlytrek was granted to Genentech, Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

On August 15, 2019 The U.S. Food and Drug Administration granted accelerated approval to Rozlytrek (entrectinib), a treatment for adult and adolescent patients whose cancers have the specific genetic defect, NTRK (neurotrophic tyrosine receptor kinase) gene fusion and for whom there are no effective treatments (Press release, US FDA, AUG 15, 2019, View Source [SID1234538858]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are in an exciting era of innovation in cancer treatment as we continue to see development in tissue agnostic therapies, which have the potential to transform cancer treatment. We’re seeing continued advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine," said FDA Acting Commissioner Ned Sharpless, M.D. "Using the FDA’s expedited review pathways, including breakthrough therapy designation and accelerated approval process, we’re supporting this innovation in precision oncology drug development and the evolution of more targeted and effective treatments for cancer patients. We remain committed to encouraging the advancement of more targeted innovations in oncology treatment and across disease types based on our growing understanding of the underlying biology of diseases."

This is the third time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated. The approval marks a new paradigm in the development of cancer drugs that are "tissue agnostic." It follows the policies that the FDA developed in a guidance document released in 2018. The previous tissue agnostic indications approved by the FDA were pembrolizumab for tumors with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) tumors in 2017 and larotrectinib for NTRK gene fusion tumors in 2018.

"Today’s approval includes an indication for pediatric patients, 12 years of age and older, who have NTRK-fusion-positive tumors by relying on efficacy information obtained primarily in adults. The FDA continues to encourage the inclusion of adolescents in clinical trials. Traditionally, clinical development of new cancer drugs in pediatric populations is not started until development is well underway in adults, and often not until after approval of an adult indication," said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "Efficacy in adolescents was derived from adult data and safety was demonstrated in 30 pediatric patients."

The ability of Rozlytrek to shrink tumors was evaluated in four clinical trials studying 54 adults with NTRK fusion-positive tumors. The proportion of patients with substantial tumor shrinkage (overall response rate) was 57%, with 7.4% of patients having complete disappearance of the tumor. Among the 31 patients with tumor shrinkage, 61% had tumor shrinkage persist for nine months or longer. The most common cancer locations were the lung, salivary gland, breast, thyroid and colon/rectum.

Rozlytrek was also approved today for the treatment of adults with non-small cell lung cancer whose tumors are ROS1-positive (mutation of the ROS1 gene) and has spread to other parts of the body (metastatic). Clinical studies evaluated 51 adults with ROS1-positive lung cancer. The overall response rate was 78%, with 5.9% of patients having complete disappearance of their cancer. Among the 40 patients with tumor shrinkage, 55% had tumor shrinkage persist for 12 months or longer.

Rozlytrek’s common side effects are fatigue, constipation, dysgeusia (distorted sense of taste), edema (swelling), dizziness, diarrhea, nausea, dysesthesia (distorted sense of touch), dyspnea (shortness of breath), myalgia (painful or aching muscles), cognitive impairment (confusion, problems with memory or attention, difficulty speaking, or hallucinations), weight gain, cough, vomiting, fever, arthralgia and vision disorders (blurred vision, sensitivity to light, double vision, worsening of vision, cataracts, or floaters). The most serious side effects of Rozlytrek are congestive heart failure (weakening or damage to the heart muscle), central nervous system effects (cognitive impairment, anxiety, depression including suicidal thinking, dizziness or loss of balance, and change in sleep pattern, including insomnia and excessive sleepiness), skeletal fractures, hepatotoxicity (damage to the liver), hyperuricemia (elevated uric acid), QT prolongation (abnormal heart rhythm) and vision disorders. Health care professionals should inform females of reproductive age and males with a female partner of reproductive potential to use effective contraception during treatment with Rozlytrek. Women who are pregnant or breastfeeding should not take Rozlytrek because it may cause harm to a developing fetus or newborn baby.

Rozlytrek was granted accelerated approval. This approval commits the sponsor to provide additional data to the FDA. Rozlytrek also received Priority Review, Breakthrough Therapy and Orphan Drug designation. The approval of Rozlytrek was granted to Genentech, Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

On August 15, 2019 Delcath Systems, Inc. ("Delcath," the "Company", "we", "our" or "us" (OTCQB: DCTH) reported that it has entered into a definitive agreement for gross proceeds of approximately $9.5 million at a combined price of $1,000 per Unit (Press release, Delcath Systems, AUG 15, 2019, View Source [SID1234538835]). Each Unit consists of one preferred share initially convertible into 16,667 shares of common stock at an initial conversion price of $0.06 per share and a common stock purchase warrant. Each whole warrant entitles the holder to purchase one share of common stock at an initial exercise price of $0.06 for a period of five years from the date of the Company’s anticipated reverse stock split.

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Roth Capital Partners is acting as the sole placement agent for the offering. After the placement agent fees and estimated offering expenses payable by the Company, the Company expects to receive net proceeds of approximately $8.6 million. The offering is expected to close on August 19, 2019, subject to customary closing conditions.

The securities offered in the private placement have not been registered under the Securities Act of 1933, as amended or applicable under state securities laws. Accordingly, the securities may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. As part of the transaction, the Company has agreed to file a resale registration statement on Form S-1 with the Securities and Exchange Commission by August 21, 2019 for purposes of registering the resale of the shares of common stock issuable upon conversion of the preferred shares and upon exercise of the warrants issued in the private placement.

This notice 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. Any offering of the securities under the resale registration statement will only be by means of a prospectus.