On March 12, 2020 Novocure (NASDAQ: NVCR) reported that preclinical data on Tumor Treating Fields in combination with anti-PD-1 therapy were published in the peer-reviewed journal, Cancer Immunology, Immunotherapy (Press release, NovoCure, MAR 12, 2020, View Source [SID1234555511]). The published study suggests that Tumor Treating Fields therapy can induce anticancer immune response and provide the first evidence for the immunostimulatory effects of Tumor Treating Fields-induced cell death.

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The published study evaluated whether Tumor Treating Fields-mediated cell death can elicit antitumor immunity. The data demonstrated that damage-associated molecular patterns were released in Tumor Treating Fields-treated cancer cells, and calreticulin was exposed on the cell surface. In addition, Tumor Treating Fields therapy promoted the engulfment of cancer cells by dendritic cells and dendritic cell maturation in vitro, as well as recruitment of immune cells in vivo. The combination of Tumor Treating Fields with anti-PD-1 therapy resulted in a significant decline in tumor volume and an increase in the percentage of tumor-infiltrating leukocytes in two animal tumor models.

The data demonstrated a potential therapeutic advantage for combining Tumor Treating Fields and anti-PD-1 therapy, highlighting that this combination may be a viable treatment regimen to enhance clinical outcomes. Novocure is currently testing the combination of Tumor Treating Fields with immune checkpoint inhibitors in its phase 3 pivotal LUNAR trial in patients with stage 4 non-small cell lung cancer who progressed during or after platinum-based therapy.

About Tumor Treating Fields

Tumor Treating Fields is a cancer therapy that uses electric fields tuned to specific frequencies to disrupt cell division, inhibiting tumor growth and causing affected cancer cells to die. Tumor Treating Fields does not stimulate or heat tissue and targets dividing cancer cells of a specific size. Tumor Treating Fields causes minimal damage to healthy cells. Mild to moderate skin irritation is the most common side effect reported. Tumor Treating Fields is approved in certain countries for the treatment of adults with glioblastoma and in the U.S. for mesothelioma, two of the most difficult cancer types to treat. The therapy shows promise in multiple solid tumor types – including some of the most aggressive forms of cancer.

On March 12, 2020 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 full year 2019 financial and operating results on the evening of Tuesday, March 24, 2020 (Press release, Celyad, MAR 12, 2020, View Source [SID1234555510]).

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Following the press release, Celyad management will host a conference call on Wednesday, March 25 at 1 p.m. CET / 8 a.m. ET to discuss full year 2019 results and provide an update on the Company’s recent progress and upcoming milestones.

Participants may access the conference call by dialing +44 (0) 2071 928501. The conference ID for the call is 1392585. Alternatively, participants may access the conference call by dialing the following local numbers: Belgium 02 401 70 35, France 01 76 72 89 28, Netherlands 020 7188527 and United States 1 917 720 0181.

To access the subsequent archived recording, visit the "Events & Webcasts" section of the Celyad website.

Additionally, in consideration of growing concerns over the spread of coronavirus disease, COVID-19, the Company has decided to postpone its upcoming Research & Development Day, previously scheduled for March 18th, until later in the year.

On March 12, 2020 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported the latest in its series of Q&A articles that are conducted with some of the key management members of PharmaCyte’s research and development team related to its upcoming clinical trial in locally advanced, inoperable pancreatic cancer (LAPC) (Press release, PharmaCyte Biotech, MAR 12, 2020, View Source [SID1234555509]).

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This interview-style Q&A article with Dr. Gerald W. Crabtree, PharmaCyte’s Chief Operating Officer, aims to address the company’s recent FDA-required tests and the submission process of its Investigational New Drug application (IND) to the U.S. Food and Drug Administration (FDA). Dr. Crabtree has spent almost 50 years working in academia and biotech and pharmaceutical companies, with the majority of that experience being in the development of drugs and treatments for cancer. He has supervised and coordinated the development of multiple drug candidates, prepared clinical protocols, investigator brochures, monographs and research and review articles.

A highlight of Dr. Crabtree’s professional career was his tenure as Director of Project Planning and Management (Oncology and Immunology) at Bristol-Myers Squibb (BMS) from 1990 to 1997. While at BMS, Dr. Crabtree established and directed a department that monitored and coordinated the development of all oncologic and immunologic drugs from initial discovery through regulatory approval within BMS and served as Project Manager for the development of the major anticancer agent, Taxol, the leading product candidate under development at BMS at that time. Taxol ultimately became a multi-billion-dollar drug for BMS and is still widely used in combination with other drugs to treat a variety of cancers.

Currently PharmaCyte is awaiting the testing results from its pyrogenicity test, an FDA-required test, that has been discussed in the past but that wasn’t part of the release testing. Can you tell us what this test is and why this test is important?

Dr. Crabtree: "In short, the U.S. FDA wants to know if the Cell-in-a-Box capsules that we use in our treatment for LAPC have any fever-inducing properties, and a pyrogenicity test is how we provide this necessary data to the FDA.

"The pyrogenicity test is important to the FDA because, to my knowledge, no other treatment for a particular tumor combines both a cancer prodrug and a biologic where that biologic consists of live, genetically engineered human cells that have been encapsulated using a unique cellulose-based technology. Because of this, the FDA wants to know whether the capsules made using the cellulose-based Cell-in-a-Box technology have any fever-inducing properties.

"In order for PharmaCyte to conduct this test, hundreds of empty Cell-in-a-Box capsules had to be produced by our partner Austrianova at its manufacturing facility in Thailand. During this time consuming and expensive process, Austrianova filled a number of syringes (identical to the syringes it used for our clinical trial product) with the empty Cell-in-a-Box capsules and then froze those syringes using the same freezing medium that we used for our clinical trial product as though the capsules were full of live cells. Following the production of the empty capsules, the resultant material was shipped to the U.S. where the pyrogenicity test is being conducted.

"To conduct the test, a protocol had to be developed, and PharmaCyte had to wait its turn in the queue to get underway. The actual test, which uses three rabbits that are injected with a matrix made up of crushed Cell-in-a-Box capsules and saline, only takes a few days to complete; however, the overall timeline to prepare for and complete the tests, and then prepare an audited report of the results that will be submitted to the FDA as part of PharmaCyte’s IND package, takes over a month in total after the frozen empty capsules arrived at the laboratory in the U.S."

Given your experience in bringing drugs to market through FDA clinical trials, can you explain what goes into an IND submission?

Dr. Crabtree: "The complexity and size of an IND most often depends upon the stage of development of a particular product. For example, if an IND is filed requesting permission to conduct an initial Phase 1 clinical trial and the product in question is a new drug, the IND contains information on how the drug was made, preclinical and animal testing data that may give some initial information on the type of tumor to attack and perhaps some idea of the dose range of the new drug that might be effective, as well as some idea of the type and severity of side effects caused by the drug. That’s pretty much it.

"Meanwhile, those requirements are in stark contrast to the IND that we will be submitting to the FDA. We are seeking FDA approval to conduct a ‘late-phase’ Phase 2 clinical trial that combines a well-known, established cancer prodrug (ifosfamide) and a biologic component that consists of genetically engineered human cells that have been encapsulated using a very unique cellulose-based, cell encapsulation technology (Cell-in-a-Box). Much of the IND that we will submit will be concerned with the latter. For example, how were the genetically engineered human cells made, where in the genome of those cells was the genetic alteration placed, etc.

"Also, we must completely address the Cell-in-a Box technology. How was the technology developed? Each step of the technology’s development must be explained in detail and with verified and validated reports. What is the history of this technology? Signed and verified reports of everything must be included in PharmaCyte’s IND package.

"Then, we must fully document the manufacturing of the final product. Each and every change made along the way to the final successful manufacturing ‘runs’ of the product must be documented in detail. All of the tests on the final product must also be fully documented.

"But, even with all of this information, the IND is not finished yet because we still have to supply the FDA with major medical documents, such as (i) the clinical trial protocol, (ii) the Investigator’s Brochure, (iii) the Informed Consent Form that each patient will review with their oncologist and sign before he or she can participate in the trial, (iv) the Angiography Guidelines to instruct interventional radiologists on how to place the Cell-in-a-Box capsules with the live human cells inside them as close to the pancreas tumor as possible; (v) a Pharmacy manual and (vi) at least three months of stability study data to show the frozen clinical trial product will work as it was designed to work months after it was manufactured."

Who is assisting PharmaCyte in the IND submission process and has the process to prepare the IND already begun?

Dr. Crabtree: "From the above, I hope our shareholders can better understand that our IND submission will be broad in scope and massive in size and that PharmaCyte could not do this alone. We are most grateful for the efforts put into assembling the IND by our consultants at Austrianova, Facet Life Sciences, cGMP Validation, Medpace, Practical Clinical and Dr. Manual Hidalgo and Dr. Matthias Löhr.

"Preparations for the IND began quite some time ago and are still on-going. We are going as fast as responsibly possible. Facet Life Sciences, our regulatory affairs consultant, will be submitting the completed IND application to the FDA on PharmaCyte’s behalf."

Does PharmaCyte pay a fee to submit the IND?

Dr. Crabtree: "The FDA does not require a fee for the filing of an IND. This has been verified by Facet Life Sciences, our regulatory affairs consultant."

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

On March 12, 2020 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics to treat chronic diseases and extend healthy lifespan, reported that the company will hold its planned investor meetings virtually at the 32nd Annual ROTH Conference following the decision by ROTH to hold the conference online on March 17 (Press release, CohBar, MAR 12, 2020, View Source [SID1234555508]). The company was previously invited to speak on a panel titled "Great Oaks Come from Little Acorns in NASH, CVD and CKD", hosted by the Co-Head of Biotechnology Research, Yasmeen Rahimi, PhD. However, all panel sessions have since been cancelled.

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On March 12, 2020 Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx plant cell-based protein expression system, reported financial results for the fourth quarter and full year ended December 31, 2019, and provided a business update on recent corporate and clinical developments (Press release, Protalix, MAR 12, 2020, View Source [SID1234555506]). The Company will discuss the clinical, corporate and financial highlights on a conference call and live webcast, scheduled for Thursday, March 12th, 2020 at 8:30 am EDT.

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"2019 was a pivotal year for Protalix, as we successfully expanded on our strong foundation and entered into a new phase of development as a world-class recombinant therapeutic company," said Dror Bashan, Protalix’s President and Chief Executive Officer. "With this continued forward momentum toward commercialization of our Fabry program, we believe Protalix is positioned for both near- and long-term success," he continued.

"We are increasingly enthusiastic about our PRX-102 asset now that we have three ongoing, fully-enrolled Phase III clinical trials of PRX-102, and as we anticipate our BLA submission to the U.S. Food and Drug Administration under the Accelerated Approval pathway next quarter," added Mr. Bashan. "Furthermore, we anticipate the final results of our BRIDGE and BRIGHT trials in the first and second halves of 2020, respectively, which will further support our portfolio of data regarding PRX-102. Protalix is firing on all cylinders right now, and the energy among the entire staff reached a new pinnacle in 2019."

"We anticipate 2020 to be a banner year for Protalix as we increase our focus on advancing our clinical pipeline, expanding sales in Brazil of Elelyso, our enzyme replacement therapy for the treatment of Gaucher disease, and leveraging commercial opportunities to expand our global footprint in the treatment of Fabry disease," he concluded.

Conference Call and Webcast Information

The Company will host a conference call on Thursday, March 12, 2020, at 8:30 am, Eastern Daylight Time, to review the clinical, corporate and financial highlights. To participate in the conference call, please dial the following numbers prior to the start of the call:

Domestic: 877-423-9813
International: 201-689-8573
Conference ID: 13699970
Webcast: View Source

The conference call will also be broadcast live and will be available for replay for two weeks in the Events Calendar of the Investors section of the Company’s website, www.protalix.com. Please access the Company’s website at least 15 minutes ahead of the conference call in order to register, download and install any necessary audio software.

2019 Full-Year and Recent Business Highlights

Clinical Advancements

·The Company and its collaboration partner, Chiesi Farmaceutici S.p.A., or Chiesi, plan the submission of a BLA for PRX-102 via the FDA’s Accelerated Approval pathway in the second quarter of 2020, based on data from the completed Phase I/II clinical trial of PRX-102 for the treatment of Fabry disease and the ongoing Phase III BRIDGE clinical trial.
Results from the Company’s Phase I/II clinical trial of PRX-102 were published in an article in the May 2019 edition of the Journal of Inherited Metabolic Disease.

·The Company announced positive 12-month interim on-treatment data from the BRIDGE study. The interim data demonstrate a mean improvement in kidney function in both male and female patients when switched from agalsidase alfa (Replagal) to PRX-102. The data will be included in the anticipated BLA filing to help to support the application.

·The Company and Chiesi announced the completion of enrollment in the Phase III BALANCE clinical trial. The head-to-head BALANCE study is designed to evaluate the safety and efficacy of PRX-102 compared to agalsidase beta (Fabrazyme) on renal function in Fabry patients with progressing kidney disease previously treated with agalsidase beta. To date, more than 66 patients are being treated in the Company’s various extension studies after opting to continue treatment with PRX-102 after completion of an initial study.

·Enrollment was completed in the Phase III BRIGHT clinical trial of PRX- 102, via intravenous infusions of 2 mg/kg administered every 4 weeks. Preliminary pharmacokinetic (PK) data showed PRX-102 to be well-tolerated; and infusion of 2 mg/kg PRX-102 administered every 4 weeks resulted in the presence of continuous active enzyme throughout the entire infusion interval. Infusions every 2 weeks is the current standard of care for the treatment of Fabry disease.

Corporate & Financial Developments

·The Company yesterday successfully secured securities purchase agreements to raise proceeds equal to $43.7 million through a private financing with a number of leading Israeli and U.S.- based investors, including Psagot Investment House, More Investment House, Highbridge Capital, UBS O’Connor, Rosalind Capital, and Alrov Properties and Lodging, among others. Rosario Capital and Houlihan Lokey served as financial advisors in the private placement.

·In December 2019, the Company held a special meeting of stockholders to propose the following two critical financial amendments, which stockholders ultimately approved:
oA reverse stock split (1-for-10); and
oA reduction in the total number of shares of the Company’s common stock that the Company is authorized to issue from 350 million to 120 million shares.

·In December 2019, the Company enhanced its Board of Directors with addition of two accomplished biopharmaceutical executives, Pol F. Boudes, M.D., and Gwen A. Melincoff.

·In August 2019, the Company’s Board of Directors unanimously elected Zeev Bronfeld, an independent director, as Chairman of the Board.

·In July 2019, the Company appointed Eyal Rubin as Senior Vice President and Chief Financial Officer.

·In May 2019, the Company appointed Dror Bashan as President and Chief Executive Officer.

Regulatory Advancements

·In February 2020, Protalix and Chiesi announced the receipt of an agreement letter from the FDA for the Initial Pediatric Study Plan (iPSP) for PRX-102 for the treatment of Fabry disease, outlining an agreed-upon approach to address the needs of pediatric Fabry patients.

Financial Results

For the year ended December 31, 2019, compared to the year ended December 31, 2018

·The Company recorded revenues from selling goods of $15.9 million for the year ended December 31, 2019, an increase of $6.9 million, or 77%, compared to revenues of $9.0 million for the same period of 2018. The increase is primarily due to higher sales of Elelyso in Brazil as well as an increase in sales of drug substance to Pfizer.

·Research and development expenses, net, were $44.6 million for the year ended December 31, 2019, an increase of $11.3 million, or 34%, compared to $33.3 million for the same period of 2018. The increase resulted primarily from an increase of $9.1 million in clinical trial related costs as well as a decrease of $2.1 million in grants received from the Israeli Innovation Authority.

·Selling, general and administrative expenses were $9.9 million for the year ended December 31, 2019, a decrease of $1.0 million, or 9%, compared to $10.9 million for the same period of 2018. The decrease resulted primarily from costs related to the Company’s U.S. Exclusive License and Supply Agreement that the Company entered into in 2018, which were not incurred in 2019.

·Net loss was $18.3 million for the year ended December 31, 2019, or $1.23 per share, basic and diluted, compared to a net loss of $26.5 million, or $1.80 per share, basic and diluted, for the same period of 2018.

·At December 31, 2019, the Company had $17.8 million in cash and cash equivalents.

For the three months ended December 31, 2019, compared to the three months ended December 31, 2018

·The Company recorded revenues from selling goods of $3.8 million during the three-month period ended December 31, 2019, an increase of $2.0 million, or 111%, compared to revenues of $1.8 million for the same period of 2018. The increase is primarily due to higher sales of drug substance to Pfizer Inc. as well as higher sales of Elelyso in Brazil.

·Research and development expenses, net, were $9.6 million for the three-month period ended each of December 31, 2019 and December 31, 2018.

·Selling, general and administrative expenses were $3.0 million for the three-month period ended December 31, 2019, an increase of $0.8 million, or 36%, compared to $2.2 million for the same period in 2018. The increase is primarily due to costs related to the efforts to evaluate and pursue strategic alternatives, business development advisory fees, and legal fees and costs related to replacement of the Chief Executive Officer.

·Net profit for the three months ended December 31, 2019 was $0.3 million, or $0.02 per share, basic and diluted, compared to a net loss of $5.4 million, or $0.40 per share, basic and diluted, for the three months ended December 31, 2018.