On August 16, 2019 Celgene Corporation (NASDAQ: CELG) reported the U.S. Food and Drug Administration (FDA) has approved INREBIC (fedratinib) for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (Press release, Celgene, AUG 16, 2019, View Source [SID1234538819]).1

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"The approval of INREBIC is another important milestone for Celgene and underscores our commitment to people living with blood cancers," said Jay Backstrom, M.D., M.P.H., Chief Medical Officer for Celgene. "We are excited to provide INREBIC as a new treatment option that may be used in patients with myelofibrosis, including patients previously treated with ruxolitinib."

"Myelofibrosis can cause patients to suffer in many ways, including experiencing debilitating symptoms," said Ruben Mesa, M.D., FACP, Director of the Mays Cancer Center at UT Health San Antonio Cancer Center MD Anderson. "There has not been a new treatment approved for this disease in nearly a decade. With INREBIC, physicians and patients now have another option available for myelofibrosis."

The INREBIC development program consisted of multiple studies (including JAKARTA and JAKARTA2) in 608 patients who received more than one dose (ranging from 30 mg to 800 mg),1 of whom 459 had myelofibrosis,1 including 97 previously treated with ruxolitinib.1 The JAKARTA study evaluated the efficacy and safety of once-daily oral doses of INREBIC compared with placebo in patients with intermediate-2 or high-risk, primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis who were previously untreated with a JAK inhibitor, had enlarged spleens (a condition known as splenomegaly), and had a platelet count of ≥50 x 109/L (median baseline platelet count was 214 x 109/L; 16% <100 x 109/L and 84% ≥100 x 109/L).1,2 In the JAKARTA study, spleen volume was reduced by 35% or greater, when assessed from baseline to the end of cycle 6 (week 24), with a 4-week follow-up scan, in 37% (35 of 96) of patients treated with INREBIC 400 mg versus 1% (1 of 96) of patients who received placebo (p<0.0001).1 INREBIC also improved the Total Symptom Score as measured by the modified Myelofibrosis Symptoms Assessment Form (MFSAF) v2.0 diary2 (night sweats, itching, abdominal discomfort, early satiety, pain under ribs on left side, bone or muscle pain) by 50% or greater when assessed from baseline to the end of cycle 6 in 40% of (36 of 89) patients treated with 400 mg, versus 9% (7 of 81) of patients who received placebo (p<0.0001).1

INREBIC has a Boxed Warning for serious and fatal encephalopathy, including Wernicke’s. Serious encephalopathy was reported in 1.3% (8 of 608) of patients treated with INREBIC in clinical trials and 0.16% (1 of 608) of the cases were fatal. Wernicke’s encephalopathy is a neurologic emergency resulting from thiamine (Vitamin B1) deficiency. Thiamine levels should be assessed in all patients prior to starting INREBIC, periodically during treatment, and as clinically indicated.1 Do not start INREBIC in patients with thiamine deficiency; replete thiamine prior to treatment initiation. If encephalopathy is suspected, immediately discontinue INREBIC and initiate parenteral thiamine. Monitor until symptoms resolve or improve and thiamine levels normalize.

In the JAKARTA study, serious adverse reactions occurred in 21% of patients treated with INREBIC 400 mg once daily (n=96), with the most common (≥2%) being cardiac failure (5%) and anemia (2%).1 Fatal adverse reactions of cardiogenic shock occurred in 1% of patients.1 Permanent discontinuation due to an adverse reaction occurred in 14% of patients. The most frequent reasons for permanent discontinuation in ≥2% of patients receiving INREBIC included cardiac failure (3%), thrombocytopenia, myocardial ischemia, diarrhea, and increased blood creatinine (2% each).1

Dosage interruptions due to an adverse reaction during the randomized treatment period occurred in 21% of patients who received INREBIC. Adverse reactions requiring dosage interruption in >3% of patients who received INREBIC included diarrhea and nausea. Dosage reductions due to an adverse reaction during the randomized treatment period occurred in 19% of patients who received INREBIC. Adverse reactions requiring dosage reduction in >2% of patients who received INREBIC included anemia (6%), diarrhea (3%), vomiting (3%), and thrombocytopenia (2%).

"INREBIC is a much-welcomed new treatment for the myelofibrosis community," said Ann Brazeau, Chief Executive Officer and Founder, MPN Advocacy and Education International. "This FDA approval marks an important milestone for people living with myelofibrosis as we embark on making greater strides in the diagnosis, understanding and treatment of this disease."

About Myelofibrosis

Myelofibrosis is a serious and rare bone marrow disorder that disrupts the body’s normal production of blood cells. Bone marrow is gradually replaced with fibrous scar tissue, which limits the ability of the bone marrow to make blood cells.3 The disorder can lead to anemia, weakness, fatigue and enlargement of the spleen and liver, among other symptoms.3 Myelofibrosis is classified as a myeloproliferative neoplasm, a group of rare blood cancers that are derived from blood-forming stem cells.4 In the U.S., between 16,000 and 18,500 are living with myelofibrosis,5 and 1.5 of every 100,000 people will be diagnosed with myelofibrosis each year.6 Both men and women are affected, and while the disease can affect people of all ages, the median age at diagnosis ranges from 60 to 67 years.7,8

About JAKARTA

JAKARTA was a pivotal Phase 3, multicenter, randomized, double-blind, placebo-controlled trial evaluating the efficacy of once-daily oral doses of INREBIC compared with placebo in patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with splenomegaly and a platelet count of ≥50 x 109/L2 (median baseline platelet count was 214 x 109/L; 16% of patients had a platelet count <100 x 109/L and 84% of patients had a platelet count ≥100 x 109/L) who were previously untreated with a JAK inhibitor.1,2 The study included 289 patients randomized to receive either INREBIC 500 mg (n=97) or 400 mg (n=96) or placebo (n=96)1 across 94 sites in 24 countries.

The primary endpoint was spleen response rate, defined as the proportion of patients achieving greater than or equal to a 35% reduction from baseline in spleen volume at the end of cycle 6 as measured by magnetic resonance imaging (MRI) or computerized tomography (CT) with a follow-up scan 4 weeks later. Secondary endpoints included symptom response rate, defined as the proportion of patients with a 50% or greater reduction in Total Symptom Score when assessed from baseline to the end of cycle 6 as measured by the modified Myelofibrosis Symptoms Assessment Form (MFSAF) v2.0 diary2 (night sweats, itching, abdominal discomfort, early satiety, pain under ribs on left side, bone or muscle pain).1

About INREBIC

INREBIC (fedratinib) is an oral kinase inhibitor with activity against wild type and mutationally activated Janus Associated Kinase 2 (JAK2) and FMS-like tyrosine kinase 3 (FLT3). INREBIC is a JAK2-selective inhibitor with higher potency for JAK2 over family members JAK1, JAK3 and TYK2. Abnormal activation of JAK2 is associated with myeloproliferative neoplasms, including myelofibrosis and polycythemia vera. In cell models expressing mutationally active JAK2 or FLT3, INREBIC reduced phosphorylation of signal transducer and activator of transcription (STAT3/5) proteins, inhibited cell proliferation, and induced apoptotic cell death. In mouse models of JAK2V617F-driven myeloproliferative disease, INREBIC blocked phosphorylation of STAT3/5, increased survival and improved disease-associated symptoms, including reduction of white blood cells, hematocrit, splenomegaly and fibrosis.1

INDICATION

INREBIC (fedratinib) is indicated for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF).

IMPORTANT SAFETY INFORMATION

WARNING: ENCEPHALOPATHY INCLUDING WERNICKE’S

Serious and fatal encephalopathy, including Wernicke’s, has occurred in patients treated with INREBIC. Wernicke’s encephalopathy is a neurologic emergency. Assess thiamine levels in all patients prior to starting INREBIC, periodically during treatment, and as clinically indicated. Do not start INREBIC in patients with thiamine deficiency; replete thiamine prior to treatment initiation. If encephalopathy is suspected, immediately discontinue INREBIC and initiate parenteral thiamine. Monitor until symptoms resolve or improve and thiamine levels normalize.

WARNINGS AND PRECAUTIONS

Encephalopathy, including Wernicke’s: Serious and fatal encephalopathy, including Wernicke’s encephalopathy, has occurred in INREBIC-treated patients. Serious cases were reported in 1.3% (8/608) of patients treated with INREBIC in clinical trials and 0.16% (1/608) of cases were fatal.

Wernicke’s encephalopathy is a neurologic emergency resulting from thiamine (Vitamin B1) deficiency. Signs and symptoms of Wernicke’s encephalopathy may include ataxia, mental status changes, and ophthalmoplegia (e.g., nystagmus, diplopia). Any change in mental status, confusion, or memory impairment should raise concern for potential encephalopathy, including Wernicke’s, and prompt a full evaluation including a neurologic examination, assessment of thiamine levels, and imaging. Assess thiamine levels in all patients prior to starting INREBIC, periodically during treatment, and as clinically indicated. Do not start INREBIC in patients with thiamine deficiency; replete thiamine prior to treatment initiation. If encephalopathy is suspected, immediately discontinue INREBIC and initiate parenteral thiamine. Monitor until symptoms resolve or improve and thiamine levels normalize.

Anemia: New or worsening Grade 3 anemia occurred in 34% of INREBIC-treated patients. The median time to onset of the first Grade 3 anemia was approximately 2 months, with 75% of cases occurring within 3 months. Mean hemoglobin levels reached nadir after 12 to 16 weeks with partial recovery and stabilization after 16 weeks. Red blood cell transfusions were received by 51% of INREBIC-treated patients and permanent discontinuation of INREBIC occurred due to anemia in 1% of patients. Consider dose reduction for patients who become red blood cell transfusion dependent

Thrombocytopenia: New or worsening Grade ≥3 thrombocytopenia during the randomized treatment period occurred in 12% of INREBIC-treated patients. The median time to onset of the first Grade 3 thrombocytopenia was approximately 1 month; with 75% of cases occurring within 4 months. Platelet transfusions were received by 3.1% INREBIC-treated patients. Permanent discontinuation of treatment due to thrombocytopenia and bleeding that required clinical intervention both occurred in 2.1% of INREBIC-treated patients. Obtain a complete blood count (CBC) at baseline, periodically during treatment, and as clinically indicated. For Grade 3 thrombocytopenia with active bleeding or Grade 4 thrombocytopenia, interrupt INREBIC until resolved to less than or equal to Grade 2 or baseline. Restart dose at 100 mg daily below the last given dose and monitor platelets as clinically indicated.

Gastrointestinal Toxicity: Gastrointestinal toxicities are the most frequent adverse reactions in INREBIC-treated patients. During the randomized treatment period, diarrhea occurred in 66% of patients, nausea in 62% of patient and vomiting in 39% of patients. Grade 3 diarrhea 5% and vomiting 3.1% occurred. The median time to onset of any grade nausea, vomiting, and diarrhea was 1 day, with 75% of cases occurring within 2 weeks of treatment. Consider providing appropriate prophylactic anti-emetic therapy (e.g., 5-HT3 receptor antagonists) during INREBIC treatment. Treat diarrhea with anti-diarrheal medications promptly at the first onset of symptoms. Grade 3 or higher nausea, vomiting, or diarrhea not responsive to supportive measures within 48 hours, interrupt INREBIC until resolved to Grade 1 or less or baseline. Restart dose at 100 mg daily below the last given dose. Monitor thiamine levels and replete as needed.

Hepatic Toxicity: Elevations of ALT and AST (all grades) during the randomized treatment period occurred in 43% and 40%, respectively, with Grade 3 or 4 in 1% and 0%, respectively, of INREBIC-treated patients. The median time to onset of any grade transaminase elevation was approximately 1 month, with 75% of cases occurring within 3 months. Monitor hepatic function at baseline, periodically during treatment, and as clinically indicated. For Grade 3 or higher ALT and/or AST elevations (greater than 5 × ULN), interrupt INREBIC dose until resolved to Grade 1 or less or to baseline. Restart dose at 100 mg daily below the last given dose. If re-occurrence of a Grade 3 or higher elevation of ALT/AST, discontinue treatment with INREBIC.

Amylase and Lipase Elevation: Grade 3 or higher amylase 2% and/or lipase 10% elevations developed in INREBIC-treated patients. The median time to onset of any grade amylase or lipase elevation was 15 days, with 75% of cases occurring within 1 month of starting treatment. One patient developed pancreatitis in the fedratinib clinical development program (n=608) and pancreatitis resolved with treatment discontinuation. Monitor amylase and lipase at baseline, periodically during treatment, and as clinically indicated. For Grade 3 or higher amylase and/or lipase elevations, interrupt INREBIC until resolved to Grade 1 or less or to baseline. Restart dose at 100 mg daily below the last given dose.

ADVERSE REACTIONS: The most common adverse reactions for INREBIC treated vs. placebo were diarrhea (66% vs. 16%), nausea (62% vs. 15%), anemia (40% vs. 14%), and vomiting (39% vs. 5%). Dosage interruptions due to an adverse reaction during the randomized treatment period occurred in 21% of patients who received INREBIC. Adverse reactions requiring dosage interruption in >3% of patients who received INREBIC included diarrhea and nausea. Dosage reductions due to an adverse reaction during the randomized treatment period occurred in 19% of patients who received INREBIC. Adverse reactions requiring dosage reduction in >2% of patients who received INREBIC included anemia (6%), diarrhea (3%), vomiting (3%), and thrombocytopenia (2%).

DRUG INTERACTIONS: Coadministration of INREBIC with a strong CYP3A4 inhibitor increases fedratinib exposure. Increased exposure may increase the risk of adverse reactions. Consider alternative therapies that do not strongly inhibit CYP3A4 activity. Alternatively, reduce the dose of INREBIC when administering with a strong CYP3A4 inhibitor. Avoid INREBIC with strong and moderate CYP3A4 inducers. Avoid INREBIC with dual CYP3A4 and CYP2C19 inhibitor. Coadministration of INREBIC with drugs that are CYP3A4 substrates, CYP2C19 substrates, or CYP2D6 substrates increases the concentrations of these drugs, which may increase the risk of adverse reactions of these drugs. Monitor for adverse reactions and adjust the dose of drugs that are CYP3A4, CYP2C19, or CYP2D6 substrates as necessary when coadministered with INREBIC.

PREGNANCY/LACTATION: Consider the benefits and risks of INREBIC for the mother and possible risks to the fetus when prescribing INREBIC to a pregnant woman. Due to the potential for serious adverse reactions in a breastfed child, advise patients not to breastfeed during treatment with INREBIC, and for at least 1 month after the last dose.

RENAL IMPAIRMENT: Reduce INREBIC dose when administered to patients with severe renal impairment. No modification of the starting dose is recommended for patients with mild to moderate renal impairment. Due to potential increase of exposure, patients with preexisting moderate renal impairment require more intensive safety monitoring, and if necessary, dose modifications based on adverse reactions.

HEPATIC IMPAIRMENT: Avoid use of INREBIC in patients with severe hepatic impairment.

On August 16, 2019 Oncolytics Biotech Inc. (NASDAQ:ONCY)(TSX:ONC) (the "Company"), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported the closing of its previously announced underwritten public offering (the "Offering") (Press release, Oncolytics Biotech, AUG 16, 2019, View Source [SID1234538818]). Oncolytics sold 4,619,773 common shares and warrants to purchase up to 4,619,773 common shares, at a purchase price of USD $0.81 (approximately CDN $1.07) per share and warrant, for gross proceeds of approximately USD $3.7 million (approximately CDN $4.9 million). Oncolytics expects to use the net proceeds from the Offering for research and development activities and working capital purposes.

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Each warrant entitles the holder thereof to purchase one common share, for a period of 5 years following the closing date of the Offering, at an exercise price of USD $0.90 per common share.

Ladenburg Thalmann & Co. Inc., a subsidiary of Ladenburg Thalmann Financial Services Inc. (LTS), acted as the sole book-running manager in connection with the Offering.

The Offering was made pursuant to a U.S. registration statement on Form F-10, declared effective by the United States Securities and Exchange Commission (the "SEC") on May 7, 2018 (the "Registration Statement"), and the Company’s existing Canadian short form base shelf prospectus (the "Base Shelf Prospectus") dated May 4, 2018. The prospectus supplements relating to the Offering (together with the Base Shelf Prospectus and the Registration Statement, the "Offering Documents") have been filed with the Alberta Securities Commission in Canada, and with the SEC in the United States. No common shares or warrants were offered or sold to Canadian purchasers. The Offering Documents contain important detailed information about the securities offered. Before you invest, you should read the Offering Documents and the other documents the Company has filed with the SEC for more complete information about the Company and the Offering. Copies of the Offering Documents will be available for free by visiting the Company’s profiles on the SEDAR website maintained by the Canadian Securities Administrators at www.sedar.com or the SEC’s website at www.sec.gov. Alternatively, when available, copies of the final prospectus supplement can also be obtained from Ladenburg Thalmann & Co. Inc., Attn: Prospectus Department, 277 Park Avenue, 26th Floor, New York, New York 10172 or by email at [email protected].

This press release does not constitute an offer to sell or the solicitation of an offer to buy securities, nor will there be any sale of the 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 August 16, 2019 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported the pricing of an underwritten public offering of 575,000 shares of its Series 1 preferred stock and warrants to purchase up to 57,500,000 shares of its common stock (Press release, Bellicum Pharmaceuticals, AUG 16, 2019, View Source [SID1234538817]). Each share of Series 1 preferred stock is being sold together with one warrant to purchase 100 shares of common stock (or, in certain circumstances, one warrant to purchase one share of Series 1 preferred stock) at a combined price to the public of $100.00. The warrants will be immediately exercisable and will expire seven years from the date of issuance. The aggregate offering size, before deducting the underwriting discounts and commissions and other offering expenses, is expected to be approximately $57.5 million.

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The offering is expected to close on or about August 21, 2019, subject to customary closing conditions.

Jefferies and Wells Fargo Securities are acting as the book-running managers for the offering. Ladenburg Thalmann is acting as a co-manager.

The securities described above are being offered by Bellicum pursuant to a shelf registration statement filed by Bellicum with the Securities and Exchange Commission (SEC), which was declared effective on July 30, 2019. A preliminary prospectus supplement related to the offering was filed with the SEC and is available for free on the SEC’s website at View Source Copies of the preliminary prospectus supplement and the accompanying prospectus related to this offering may be obtained from: Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 821-7388, or by e-mail at [email protected]; or Wells Fargo Securities, LLC, 375 Park Avenue, New York, NY 10152, Attn: Equity Syndicate Department, by phone at (800) 326-5897, or by email at [email protected].

Concurrent with the pricing of the public offering, Bellicum entered into an agreement with certain institutional investors providing for a private placement exempt from the registration requirements of the Securities Act of 1933, as amended, pursuant to which Bellicum has agreed to sell at two or more separate closings, each at the option of the investors and subject to certain conditions, shares of its Series 2 preferred stock and warrants to purchase common stock, and shares of Series 3 preferred stock and warrants to purchase common stock, for aggregate gross proceeds of up to $70.0 million. In connection with the agreement for the private placement, the investors in the private placement will pay Bellicum an upfront option fee of approximately $12.1 million.

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 state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

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.