On August 10, 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 second quarter ended June 30, 2020, and provided a business update on recent corporate and clinical developments (Press release, Protalix, AUG 10, 2020, View Source [SID1234563270]).

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"This quarter, we delivered on two very important milestones for the company: announcing positive topline results in our BRIDGE phase III clinical trial of PRX-102 for the treatment of Fabry disease and subsequent BLA submission to the U.S. Food and Drug Administration (FDA). We were able to accomplish these goals even as we faced the challenging headwinds from the global COVID-19 pandemic, and I am very proud of our entire team for their commitment and dedication," said Dror Bashan, Protalix’s President and Chief Executive Officer. "As we look towards an exciting second half of the year, we are continuing to build Protalix for the long term. We augmented our research and development team with highly qualified and seasoned veterans to support and enhance our pipeline, and we announced a new partnership to explore the development of PRX-110." Mr. Bashan concluded, "We are gratified to have a balance sheet supporting our strategic plans and look forward to continuing to execute as we move towards the anticipated commercial launch of PRX-102 for the treatment of Fabry disease."

Recent Business Highlights

·Submitted a Biologics License Application (BLA) to the FDA for PRX-102 (pegunigalsidase alfa) for the treatment of adult patients with Fabry disease on May 27, 2020. The BLA was submitted under the FDA’s accelerated approval pathway in collaboration with the Company’s development and commercialization partner, Chiesi Farmaceutici S.p.A. On July 28, 2020, the FDA informed Chiesi that the BLA had been filed for review and that the FDA was working on the 74-day letter. In addition, the FDA informed Chiesi that no "Refuse To File" will be issued for the PRX-102 BLA.

Announced positive topline results from our BRIDGE Phase III clinical trial of PRX-102 for the treatment of Fabry disease. The study was an open-label, switch-over trial designed to evaluate the safety and efficacy of 1 mg/kg PRX-102 infused every two weeks, in Fabry patients. The trial 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.

·Enhanced the executive management team with the appointment of Yael Hayon, Ph.D. as Vice President, Research and Development. Dr. Hayon brings over a decade of pharmaceutical research and development experience in both the scientific operations and administrative functions.

·Entered into a non-binding term sheet with SarcoMed USA, Inc. to explore the development and commercialization of PRX-110 (alidornase alfa) in the treatment of Pulmonary Sarcoidosis and related diseases. SarcoMed USA was formed in 2017 to investigate if a novel DNase 1 compound could influence the chronic pulmonary inflammation seen in Pulmonary Sarcoidosis patients.

Second Quarter 2020 Financial Highlights

The Company recorded revenues from selling goods of $3.6 million during the three months ended June 30, 2020, an increase of $0.2 million, or 6%, compared to revenues of $3.4 million for the same period of 2019.

Revenues from license and R&D services for the three months ended June 30, 2020, were $7.3 million, a decrease of $1.5 million, or 17%, compared to revenues of $8.8 million for the same period of 2019. Revenues from license and R&D services are comprised primarily of revenues the Company recognized in connection with its license and supply agreements with Chiesi. The decrease is primarily due to the completion of two out of the three phase III clinical trials of PRX-102 as well as lower costs related to the Company’s phase III BALANCE clinical trial of PRX-102 for the treatment of Fabry disease.

Cost of goods sold was $1.8 million for the three months ended June 30, 2020, a decrease of $0.9 million, or 32%, from cost of goods sold of $2.7 million for the same period of 2019. The decrease is primarily due to a change in the cost structure as well as lower royalties paid to the Israeli Innovation Authority.

Research and development expenses were $9.2 million for the three months ended June 30, 2020, a decrease of $4.1 million, or 31%, compared to $13.3 million of research and development expenses for the same period of 2019. The decrease is primarily due to the completion of two out of the three phase III clinical trials of PRX-102 and reduced costs related to the Company’s phase III BALANCE clinical trial as well as a decrease in costs related to manufacturing of the Company’s drug in development as some of the manufactured drug product and related costs have been recorded as inventory.

Selling, general and administrative expenses were $2.2 million for the three months ended June 30, 2020, an increase of $0.1 million, or 6%, compared to $2.1 million for the same period of 2019.

Cash and cash equivalents at June 30, 2020 was $4.8 million, with $35.2 million in bank deposits.

Conference Call and Webcast Information

The Company will host a conference call on Monday, August 10, 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: 13706783
Webcast: View Source

The conference call will be broadcast live and also 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.

On August 10, 2020 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the second quarter ended June 30, 2020 and recent company developments (Press release, TG Therapeutics, AUG 10, 2020, View Source [SID1234563261]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "We are extremely pleased by the progress made thus far in 2020 and are looking forward to an impactful end of year and into 2021. Completing the NDA rolling submission this past June for umbralisib in previously treated Marginal Zone Lymphoma and Follicular Lymphoma was a major milestone for our Company." Mr. Weiss continued, "With one completed NDA submission, positive topline data from our UNITY-CLL Phase 3 trial, and a healthy balance sheet with over $275 million in cash, we are well positioned to execute on our remaining milestones for this year as well as transition from a development stage company to a fully-integrated commercial organization. For the remainder of this year, we look forward to reporting topline data from our Phase 3 ULTIMATE program in Multiple Sclerosis, full data presentations from the UNITY-NHL FL and MZL single agent umbralisib cohorts, data presentation from the UNITY-CLL Phase 3 trial, as well as updated data from our triple combination trials, which we believe set the stage for the future of U2 in CLL and non-Hodgkin’s Lymphoma."

Recent Developments and Highlights

Marginal Zone Lymphoma & Follicular Lymphoma:
° In June 2020, completed rolling submission of New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for umbralisib as a treatment for patients with previously treated marginal zone lymphoma (MZL) and follicular lymphoma (FL).

Chronic Lymphocytic Leukemia:
° In May 2020, reported positive topline results from the Company’s UNITY-CLL Phase 3 trial evaluating U2 (the combination of umbralisib and ublituximab) in patients with previously untreated and relapsed/refractory chronic lymphocytic leukemia (CLL). The trial met its primary endpoint of improved progression-free survival (PFS) (p<.0001), as determined by an Independent Review Committee (IRC).
° In May 2020, reported final results from the GENUINE Phase 3 study evaluating the combination of ublituximab plus ibrutinib compared to ibrutinib alone in patients with relapsed/refractory CLL with high-risk cytogenetics. Results indicated the addition of ublituximab to ibrutinib as compared to ibrutinib alone improved PFS, overall response rate, complete response rate and the number of patients with undetectable minimal residual disease.

Multiple Sclerosis:
° In May 2020, announced the publication of results from the multicenter Phase 2 trial evaluating ublituximab in patients with relapsing forms of multiple sclerosis (RMS) in the Multiple Sclerosis Journal.

Bolstered Balance Sheet:
° In May 2020, strengthened balance sheet with more than $240 million in gross proceeds through a public offering and the Company’s At-the-Market (ATM) facility.

Publication:
° In July 2020, published preclinical data describing the unique immunomodulatory effects of umbralisib, in Blood Advances, a Journal of the American Society of Hematology (ASH) (Free ASH Whitepaper).

Board of Directors & Management:
° In May 2020, appointed Sagar Lonial, MD, FACP, Professor and Chair of the Department of Hematology and Medical Oncology at the Emory University School of Medicine, as well as the Chief Medical Officer at Winship Cancer Institute of Emory University, to the Company’s Board of Directors.
° In May 2020, strengthened executive team with the addition of Owen A. O’Connor, MD, PhD as Chief Scientific Officer. Dr. O’Connor most recently served as a Professor of Medicine and Experimental Therapeutics, the Director of the Center for Lymphoid Malignancies, and Co-Program Director of the Lymphoid Development and Malignancy Program in the Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center.
Key Objectives for Remainder of 2020 and Early 2021

Report topline results from the Phase 3 ULTIMATE I & II trials in Multiple Sclerosis.
Present full data from the UNITY-CLL Phase 3 trial and present full data from the FL and MZL single agent umbralisib cohorts of the UNITY-NHL trial at a major medical meeting.
Target an NDA/Biologics Licensing Application (BLA) submission of U2 for the treatment of patients with CLL (including both previously untreated and relapsed/refractory patients)
Continue to advance our early pipeline candidates including our anti-PD-L1 monoclonal antibody, cosibelimab (TG-1501), our Bruton’s Tyrosine Kinase (BTK) inhibitor, TG-1701, and our anti-CD47/CD19 bispecific antibody, TG-1801.
Financial Results for the Three and Six Months Ended June 30, 2020

R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation) was $34.9 million and $68.9 million for the three and six months ended June 30, 2020, respectively, compared to $31.4 million and $62.3 million for the three and six months ended June 30, 2019, respectively. The modest increase in R&D expense during the three and six month periods of 2020 is primarily attributable to our ongoing clinical development programs as well as preparations for regulatory filings and potential commercialization. We expect our R&D expenses to decrease during the remainder of 2020 as costs associated with our main pivotal clinical trials continue to decline, partially offset by expenses associated with the expected NDA/BLA filing for U2 in CLL.

G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $8.6 million and $13.8 million for the three and six months ended June 30 2020, respectively, as compared to $2.3 million and $4.3 million for the three and six months ended June 30, 2019, respectively. The increase in other G&A expenses is primarily due to increased personnel and other general and administrative costs, associated with preparations for a potential commercial launch. We expect G&A expenses to increase modestly during the remainder of 2020 in preparation for potential launch.

Net Loss: Net loss was $52.9 million and $104.0 million for the three and six months ended June 30, 2020, respectively, compared to a net loss of $36.2 million and $71.4 million for the three and six months ended June 30, 2019, respectively. Excluding non-cash compensation, the net loss for the three and six months ended June 30, 2020 was approximately $45.5 million and $85.6 million, respectively, compared to a net loss of $34.5 million and $67.7 million for the three and six months ended June 30, 2019, respectively.

Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $275.6 million as of June 30, 2020. The Company believes its cash, cash equivalents and investment securities on hand as of June 30, 2020, as well as future availability under the Company’s debt and ATM facility, will be sufficient to fund the Company’s planned operations through the end of 2021.

On August 10, 2020 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported that it has entered into a clinical trial collaboration and supply agreement with Eli Lilly and Company to evaluate the safety and efficacy of combining Pieris’ PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, with Lilly’s ramucirumab, a VEGFR2 antagonist FDA-approved for multiple types of solid tumors, and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a phase 2 study (Press release, Pieris Pharmaceuticals, AUG 10, 2020, View Source [SID1234563259]).

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Under the terms of the agreement, Lilly will supply Pieris with ramucirumab for the study as well as collaborate on data from the trial. Pieris is working towards initiation of a phase 2 single-arm combination study for the second-line treatment of HER2-positive gastric cancer later this year.

"We have seen impressive single-agent activity in the phase 1 trial of PRS-343, including a complete response and many patients experiencing a clinical benefit, and believe there is a compelling biology and clinical rationale to adding PRS-343 to the current standard of care regimen for advanced or metastatic gastric cancer in the second line, ramucirumab and paclitaxel," said Stephen S. Yoder, President and Chief Executive Officer of Pieris. "Today’s announcement further supports exploring this clinical rationale while managing costs efficiently."

On August 10, 2020 Hutchison China MediTech Limited ("Chi-Med") (Nasdaq/AIM: HCM) reported that it received scientific advice from the European Medicines Agency’s ("EMA") Committee for Medicinal Products for Human Use ("CHMP") for surufatinib for the treatment of patients with advanced neuroendocrine tumors ("NET") (Press release, Hutchison China MediTech, AUG 10, 2020, https://www.chi-med.com/chi-med-plans-to-submit-maa-for-surufatinib/ [SID1234563258]). Based on the CHMP advice, we have concluded that the completed SANET-ep (non-pancreatic NET) and SANET-p (pancreatic NET) studies, along with existing data from surufatinib in U.S. non-pancreatic and pancreatic NET patients, could form the basis to support a marketing authorization application ("MAA"). Given that no filing issues were identified, the MAA submission is planned for 2021, following submission for the U.S. Food and Drug Administration ("FDA") new drug application ("NDA").

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About Surufatinib
Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor (VEGFR) and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies, where there may be synergistic anti-tumor effects.

Chi-Med currently retains all rights to surufatinib worldwide.

About Surufatinib Development
NET in the U.S., Europe and Japan: In the U.S., surufatinib was granted Fast Track Designations for development in pancreatic and non-pancreatic (extra-pancreatic) NET in April 2020, and Orphan Drug Designation for pancreatic NET in November 2019. A U.S. FDA NDA submission is being prepared, to be followed by a MAA submission to the EMA in Europe. All such interactions are based on the robust data from the two positive Phase III studies of surufatinib in NET in China, and the ongoing multi-cohort Phase Ib study in the U.S. that began in November 2015 (clinicaltrials.gov identifier: NCT02549937).

Non-pancreatic NET in China: In November 2019, a NDA for surufatinib for the treatment of patients with advanced non-pancreatic NET was accepted for review by the China National Medical Products Administration ("NMPA") and granted Priority Review status in December 2019. The NDA is supported by data from the successful SANET-ep study, a Phase III study of surufatinib in patients with advanced non-pancreatic NET in China for whom there is no effective therapy. A 198-patient interim analysis was conducted in June 2019, leading the Independent Data Monitoring Committee ("IDMC") to determine that the study met the pre-defined primary endpoint of progression-free survival ("PFS") and should be stopped early. The positive results of this trial were highlighted in an oral presentation at the 2019 European Society for Medical Oncology Congress ("ESMO") (clinicaltrials.gov identifier: NCT02588170).

Pancreatic NET in China: In 2016, we initiated the SANET-p study, which is a pivotal Phase III study in patients with low- or intermediate-grade, advanced pancreatic NET in China. Following an interim analysis review conducted in January 2020 by the IDMC that recommended the registrational study be terminated early as the pre-defined primary endpoint of PFS had already been met (clinicaltrials.gov identifier: NCT02589821), we submitted our second NDA to the China NMPA and are now waiting for formal acceptance. The results of this study will be presented at ESMO (Free ESMO Whitepaper) 2020.

Biliary tract cancer in China: In March 2019, we initiated a Phase IIb/III study comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy. The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier NCT03873532).

Immunotherapy combinations: We have entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-PD-1 monoclonal antibodies, including with tislelizumab (BGB-A317, developed by BeiGene, Ltd.), Tuoyi (toripalimab, developed by Shanghai Junshi Biosciences Co. Ltd.) and Tyvyt (sintilimab, developed by Innovent Biologics, Inc.), which are approved in China.

On August 10, 2020 Epigenomics AG (Frankfurt Prime Standard: ECX, OTCQX: EPGNY; the "Company") reported that a study published by the NCI-sponsored cancer intervention and surveillance modeling network (CISNET) in the Journal of the National Cancer Institute reported that by comparing the incremental cost-effectiveness of CTC, PillCam, mtSDNA (Cologuard) and mSEPT9 (Epi proColon), the study revealed that of these CRC screening alternatives annual screening with Epi proColon is cost-effective (Press release, Epigenomics, AUG 10, 2020, View Source [SID1234563257]). Annual screening with Epi proColon had an incremental cost-effectiveness ratio (ICER) of $63,253 per QALYG. Other efficient strategies were CTC screening every 5 years (ICER: $1,092 per QALYG) and annual (but not every three years) mtSDNA screening (ICER: $214,974 per QALYG), which were not optimal given the willingness-to-pay threshold ($100,000 per QALYG).

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Jorge Garces, President and CSO of Epigenomics AG: "CISNET microsimulation models are the gold-standard by which the American Cancer Society (ACS), United States Preventative Services Task Force (USPSTF), and other clinical societies base their guideline recommendations for CRC screening. This study supports the findings from another recent study published in Cancer Medicine and adds to the mounting evidence indicating that Epi proColon administered annually can reduce the incidence and mortality of colorectal cancer as effectively or better than other approved methods and most importantly highlights the opportunity for the Epi proColon blood test to serve as the test of choice for those currently resistant to colonoscopy and stool-based screening methods."

The JNCI publication analyzed the clinical effectiveness and performance of various screening strategies under five different scenarios:

1. Screening from age 50 through 75 years in an-average risk population, with perfect adherence to screening, diagnostic follow-up and surveillance recommendations.

2. Assuming screening from age 45 to age 75 with outcomes presented per 1,000 45-year olds at 100% adherence.

3. Using the model version that was used to inform the 2016 USPSTF screening guidelines, in which screening starts at 50 years of age and does not account for the increasing incidence. 100% adherence assumed.

4. Assuming that 90% of the people that participated in a previous round would participate in a subsequent round, and 15% of the people that did not participate in the previous round would participate in the subsequent round. An 80% adherence to diagnostic follow-up and surveillance was assumed.

5. Assuming 12% of advanced adenomas and 18% of colorectal cancers are systematically missed by the mSEPT9.

Under all scenarios examined, annual Epi proColon was more cost-effective than Cologuard. The authors also conclude that ultimately, the best test is the "one that gets done".

Greg Hamilton, CEO of Epigenomics AG: "This is an important publication as it further validates the clinical and cost-effectiveness of Epi proColon. It is also timely as we await the preliminary National Coverage Determination (NCD) from CMS later this month".

As the JNCI authors clearly state: "A well-established microsimulation model demonstrates that for people who are unwilling to be screened with FIT or colonoscopy, annual screening with the mSEPT9 is the test of choice given its cost-effectiveness profile compared to CTC, PillCam and mtSDNA."

Epigenomics will hold a conference call on Tuesday August 11, 2020 at 9:30am ET (3:30pm CET) to discuss the publication in more detail and answer questions. Please use the link in the Financial Calendar on the Epigenomics.com website to join the conference call.