On June 1, 2020 Shanghai Henlius Biotech, Inc. (2696.HK) and Accord Healthcare Limited (Accord) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency ( European Medicines Agency, EMA) issued a favorable opinion that recommended the market for the HLX02 biosimilar (trastuzumab injection) for the treatment of early HER-2-positive breast cancer, HER2-positive metastatic breast cancer and previously untreated HER-2-positive metastatic cancer Contains adenocarcinomas of the stomach or the transition area of ​​the esophagus into the stomach (Press release, Shanghai Henlius Biotech, JUN 1, 2020, View Source;816056005.html [SID1234560743]). According to the EMA-CHMP summary of this positive opinion, HLX02 is the reference product Herceptin (Trastuzumab) very similar. Data show that HLX02 is comparable to Herceptin in terms of quality, safety and effectiveness .

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HLX02 is a monoclonal biosimilar antibody (mAb) developed and manufactured by Henlius in accordance with biosimilar guidelines and will be marketed and marketed by Accord in Europe. The CHMP’s opinion is now being reviewed by the European Commission. A regulatory decision is expected over the next two to three months. As soon as a license is granted , a centralized marketing authorization is granted for all EU member states, Iceland, Norway and Liechtenstein .

Dr. Scott Liu , co-founder and CEO of Henlius , commented: "We are proud of CHMP’s favorable opinion on Henlius, as is the fact that Henlius’ capabilities in developing and manufacturing biological products are recognized by an international drug agency. We look forward to the launch of HLX02 as an alternative, high quality and affordable treatment option for patients with HER-2 positive breast cancer and gastric cancer. We will continue to work with Accord, make HLX02 more available in Europe and other regions, and continue to strive to support patients around the world. "

Dr. James Burt , Executive Vice President of Accord Europe and MENA added: "We are committed to providing oncology patients with access to safe, inexpensive drugs. The CHMP’s positive opinion is due to our in-depth expertise and our extensive portfolio of oncological therapies. Our agreement with Henlius reflects our longstanding commitment to biopharmaceutical development, research and manufacturing. "

On June 1, 2020 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported the presentation of two posters at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Meeting (Press release, ERYtech Pharma, JUN 1, 2020, View Source [SID1234560760]).

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Trial in Progress Poster for the ongoing Phase 3 TRYbeCA1 trial (Abstract # TPS4666)

TRYbeCA-1 is a randomized, controlled Phase 3 clinical trial evaluating eryaspase in second-line metastatic pancreatic cancer. The trial is planned to enroll approximately 500 patients in approximately 100 clinical sites in Europe and the U.S. Eligible patients are randomized 1-to-1 to receive eryaspase in combination with standard chemotherapy (gemcitabine/nab paclitaxel or an irinotecan based regimen) or chemotherapy alone. The primary endpoint of TRYbeCA1 is overall survival (OS). The trial is designed to identify a OS hazard ratio (HR) of 0.72 with close to 90% power. An interim efficacy analysis is planned for when approximately two- thirds of events have occurred. To date, approximately 80% of the patients have been enrolled.

Prof Pascal Hammel, MD, PhD, gastroenterologist-oncologist at Beaujon Hospital in Paris and co-principal investigator of the study, commented, "Patients with advanced pancreatic cancer need new treatment options, particularly in second line treatments after failure of gemcitabine-Nab-paclitaxel or FOLFIRINOX combinations. The Trial-in-Progress poster demonstrates that we are making excellent progress toward completing enrolment of this important international study and I look forward to the outcome of the planned interim analysis for superiority towards the end of 2020."

ctDNA is prognostic and potentially predictive of eryaspase efficacy in patients with advanced pancreatic adenocarcinoma (Abstract #4617)

Eryaspase is composed of L-asparaginase encapsulated in erythrocytes and has demonstrated significant efficacy in a randomized Phase 2 trial1. Prognostic and predictive value of circulating tumor DNA (ctDNA) investigated in this trial.

The analysis from this prospective randomized trial confirmed that the presence of ctDNA at baseline is a strong prognostic factor, and that the early change of ctDNA correlates with treatment outcome. ctDNA could therefore be a potential predictive biomarker of eryaspase efficacy.

Dr Bachet from Sorbonne Université, UPMC Université, IUC, Paris France commented, " The feasibility of this approach and its potential prognostic value provides a rationale for stratifying patients in future clinical trials. Our results suggest that presence of ctDNA could be a predictive biomarker of eryaspase efficacy which needs to be confirmed from the ongoing phase 3 clinical trial."

"We are excited about this state-of-the art ctDNA analysis. We will continue our endeavour to investigate ctDNA and other biomarker platforms in the ongoing Phase 3 trial to further understand the efficacy of eryaspase in different patient subsets." said Dr. Iman El-Hariry, Chief Medical Officer of ERYTECH Pharma.

1 Hammel P, Fabienne P, Mineur L, et al. Erythrocyte-encapsulated asparaginase (eryaspase) combined with chemotherapy in second-line treatment of advanced pancreatic cancer: An open-label, randomized Phase IIb trial. Eur J Cancer. 2019;124:91- 101

"We continue to be encouraged by the progress of our Phase 3 trial, despite the unprecedented times due to COVID-19. We are thankful to all our investigators who continued to enroll patients in the study and adhere to our guidelines dealing with potential COVID-19 impact."

Both posters are available to view online at the ASCO (Free ASCO Whitepaper) Virtual meeting website: View Source

About pancreatic cancer

Pancreatic cancer is a disease in which malignant (cancer) cells are found in the tissues of the pancreas. Every year there are about 150,000 new cases of pancreatic cancer diagnosed in Europe and the United States. Pancreatic cancer is a particularly aggressive cancer, with a five-year survival rate of less than 10% and is currently the fourth most common cause of cancer death in the EU for men and women.

On June 1, 2020 Genor Biopharma, a Shanghai company with ten biologic candidates in clinical trials, reported that it completed a $160 million Series B financing led by Beijing’s Hillhouse Capital Group (Press release, Genor Biopharma, JUN 1, 2020, View Source [SID1234560728]). The company’s PD-1 molecule is currently in four pivotal Phase II trials. Its other clinical stage assets are mAbs and recombinant fusion proteins, a combination of novel drugs and biosimilars that target indications in cancer and autoimmune diseases. In late 2019, Genor was rumored to be planning a $200 million IPO on the Hong Kong Exchange, but that offering has not been officially announced.

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On June 1, 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 first quarter ended March 31, 2020, and provided a business update on recent corporate and clinical developments (Press release, Protalix, JUN 1, 2020, View Source [SID1234560744]). The Company’s management will discuss the financial results and provide a clinical, corporate and financial highlights on a conference call and live webcast scheduled for Monday, June 1, 2020 at 8:30 am Eastern Daylight Time (EDT).

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"The first quarter of 2020 has most certainly been transformational for Protalix, despite the COVID-19 pandemic that affected the global markets," said Dror Bashan, Protalix’s President and Chief Executive Officer. "I am proud to say that despite the pandemic, Protalix was able to keep the company running smoothly and adapt quickly to the changing environment."

"During the quarter, we were able to close a $43.7 million private placement," he continued. "Furthermore, the topline results from the completion of our Phase III BRIDGE study and the subsequent BLA submission for PRX–102 announced in May prove that Protalix has actually gained momentum by leaning into this unprecedented challenge. I am convinced now more than ever that our team is positioned for long-term success and look forward to continuing our momentum through the rest of this year and into 2021."

Conference Call and Webcast Information

The Company will host a conference call on Monday, June 1, 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:

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

First Quarter 2020 and Recent Business Highlights

Clinical and Regulatory Advancements

On May 28, 2020, the Company and its development and collaboration partner, Chiesi Global Rare Diseases, a unit of Chiesi Farmaceutici S.p.A., or Chiesi, announced the submission on May 27, 2020 of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for pegunigalsidase alfa, or PRX–102, for the treatment of adult patients with Fabry disease via the FDA’s Accelerated Approval pathway. PRX–102 was granted Fast Track designation by the FDA in January 2018. Upon the BLA approval, if approved, the Company will be eligible to receive a milestone payment from Chiesi.
On May 11, 2020, the Company announced positive topline results following the completion of its Phase III BRIDGE clinical trial of PRX–102 for the treatment of Fabry disease. The Phase III BRIDGE clinical trial, a 12-month open-label, single arm switch-over study evaluating the safety and efficacy of PRX–102, 1 mg/kg infused every two weeks, met its main objectives for safety and efficacy, and topline analysis indicated substantial improvement in renal function as measured by mean annualized estimated Glomerular Filtration Rate (eGFR slope) in patients switched from agalsidase alfa to PRX–102.
On February 6, 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.
Corporate & Financial Developments

On March 16, 2020, the Company announced that it has agreed to conduct a feasibility study with Kirin Holdings Company, Limited, or Kirin, to evaluate the production of a novel complex protein utilizing ProCellEx. The Company received a non-refundable payment of $1.0 million and Kirin will provide research funding for the Company’s scientists to conduct cell line engineering and protein expression studies on the target protein.
On March 12, 2020, the Company entered into securities purchase agreements with certain existing and new institutional and other accredited investors in a private placement. Pursuant to such agreements, the Company issued and sold to the purchasers an aggregate of approximately 17.6 million unregistered shares of its common stock at a price per share of $2.485, or aggregate net committed proceeds equal to approximately $41.3 million. Each share of the Company’s common stock issued in the transaction was accompanied by a warrant to purchase an additional share of common stock at an exercise price equal to $2.36.
Financial Results

For the three months ended March 31, 2020, compared to the three months ended March 31, 2019

The Company recorded revenues from selling goods of $5.0 million during the three months ended March 31, 2020, an increase of $1.5 million, or 43%, compared to revenues of $3.5 million for the same period of 2019. The increase resulted primarily from an increase of $0.8 million in sales of drug product to Brazil as well as an increase of $0.7 million in sales of drug substance to Pfizer Inc.
Revenues from license and R&D services for the three months ended March 31, 2020, were $16.6 million, an increase of $9.7 million, or 140%, compared to revenues of $6.9 million for the same period of 2019. Revenues from the license agreements represent the revenues recognized in connection with previously announced agreements with Chiesi. The increase is primarily due to revenues recognized in connection with the progress of the Company’s clinical trial that have been performed, and with revenues recognized in connection with an updated costs estimation throughout the trials until completion in the amount of $6.7 million.
Cost of goods sold was $3.4 million for the three months ended March 31, 2020, an increase of $1.4 million, or 68%, from cost of goods sold of $2.0 million for the same period of 2019. The increase is primarily due to an increase in sales of goods.
Research and development expenses were $10.3 million for the three months ended March 31, 2020, a decrease of $1.4 million, or 12%, compared to $11.7 million of research and development expenses for the same period of 2019. The decrease was primarily due to a decrease in costs related to manufacturing of our drug in development.
Selling, general and administrative expenses were $3.2 million for the three months ended March 31, 2020, an increase of $1.0 million, or 43%, compared to $2.2 million for the same period of 2019. The increase resulted primarily from a $0.6 million increase in compensation related costs and a $0.2 million increase in professional fees.
Net income for the three months ended March 31, 2020 was $1.7 million, or $0.10 per share, basic and diluted, compared to a net loss of $7.3 million, or $0.50 per share, basic and diluted, for the same period of 2019.

On May 31, 2020 Taiho Oncology, Inc. reported efficacy and safety results of an interim analysis of FOENIX-CCA2, a single-arm multicenter Phase 2 study evaluating futibatinib (TAS-120) in patients with intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 gene fusions or other rearrangements, who have failed at least one line of therapy (Press release, Taiho, MAY 31, 2020, View Source [SID1234558753]). The data were presented online at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting from 10:30 a.m.-12:00 p.m. ET on Sunday, May 31, 2020.

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"The interim analysis demonstrated that treatment with the covalently-binding FGFR inhibitor futibatinib may lead to meaningful clinical benefit in patients with refractory iCCA with FGFR2 gene fusions or other rearrangements," said medical oncologist Lipika Goyal, MD , MPhil of the Massachusetts General Hospital Cancer Center. "In a disease with limited treatments, this drug could be an effective and well-tolerated option for patients and the oncologists that care for them."

In the FOENIX-CCA2 trial, 103 patients with locally advanced or metastatic unresectable iCCA harboring FGFR2 gene rearrangements including fusions who had received one or more prior lines of systemic therapy received futibatinib 20 mg once daily until disease progression or unacceptable toxicity. The primary endpoint of the trial is independent central radiology reviewed objective response rate (ORR). Secondary endpoints include disease control rate (DCR), duration of response (DOR) and safety. The interim analysis reported data for 67 patients (65%) with a minimum of 6 months of follow up and found the ORR was 37.3% (1 CR=1.5%; 24 PR=35.8%). Median duration of response was 8.31 months. The most common treatment-related adverse events (all grades, grade 3) at the time of analysis were hyperphosphatemia (80.6%; 26.9%), diarrhea (37.3%; 0%), and dry mouth (32.8%; 0%). There were no grade 4 treatment related adverse events. Final results from the trial will be presented at a future medical meeting.

"FOENIX-CCA2 adds to the body of evidence supporting futibatinib as a potential treatment option for patients living with intrahepatic cholangiocarcinoma," said Martin J. Birkhofer, MD, Senior Vice President and Chief Medical Officer, Taiho Oncology, Inc. "We are pleased to see the interim results of the FOENIX-CCA2 trial, which point to the efficacy and tolerability of futibatinib in these patients, and we look forward to sharing the final results and progressing this investigational compound."

In May 2018, the U.S. Food and Drug Administration Office of Orphan Drug Development granted futibatinib orphan drug status for the treatment of cholangiocarcinoma.

About Cholangiocarcinoma
Cholangiocarcinoma (CCA), also known as bile duct cancer, is not common. About 8,000 people in the U.S. are diagnosed with CCA each year.1 This includes both intrahepatic (inside the liver) and extrahepatic (outside the liver) cancers. CCA can occur at younger ages, but it is seen mainly in older people. The average age of people in the U.S. diagnosed with cancer of the intrahepatic bile ducts is 70, and for cancer of the extrahepatic bile ducts it is 72.2 The five-year survival rates of localized iCCA is 24%.1

The main treatment for CCA is surgery. Radiation therapy and chemotherapy may be used if the cancer cannot be entirely removed with surgery and in cases where the edges of the tissues removed at the operation show cancer cells (also called a positive margin). Both stage III and stage IV cancers cannot be completely removed surgically. Currently, standard treatment options are limited to radiation, palliative therapy, liver transplantation, surgery, chemotherapy and interventional radiology.2

About Futibatinib (TAS-120)
Futibatinib (TAS-120) is an investigational, oral, potent, selective, and irreversible small-molecule inhibitor of FGFR1, 2, 3, and 4 being studied as a potential treatment for patients with advanced solid tumors with FGFR1-4 genetic aberrations, including cholangiocarcinoma, who were previously treated with chemotherapy or other therapies. Futibatinib selectively and irreversibly binds to the ATP binding pocket of FGFR1-4 resulting in the inhibition of FGFR-mediated signal transduction pathways, reduced tumor cell proliferation and increased tumor cell death in tumors with FGFR1-4 genetic aberrations.