On February 4, 2020 PharmaCyte Biotech (OTCQB: PMCB) is reported to embark upon a planned U.S. FDA Phase 2b clinical trial to treat locally advanced, inoperable pancreatic cancer (LAPC) at trial sites all over the United States, and with that journey comes the need to keep its signature technology, Cell-in-a-Box, protected should it one day receive marketing approval from drug regulatory agencies in the U.S. and Europe (Press release, PharmaCyte Biotech, FEB 4, 2020, View Source [SID1234553841]). PharmaCyte’s Cell-in-a-Box technology for the treatment of pancreatic cancer is certainly protected well into the future if/when it does receive the coveted marketing approval it’s striving for upon the completion of clinical trials.

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

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

                  Schedule Your 30 min Free Demo!

Currently, PharmaCyte is revamping its provisional patent application and its strategy related to full patent protection. The idea is to present the United States Patent and Trademark Office (USPTO) with an acceptable provisional patent application that protects its therapy to treat cancerous tumors, including the therapy that will be used in its upcoming clinical trial in LAPC.

Provisional patent applications are a way to establish and protect a "date of invention" or "priority filing date" for one year. The provisional patent application was created to provide inventors with a way to begin protecting their inventions, and an approved provisional patent application will provide PharmaCyte 12 months to prepare a full patent application. This approach will offer the company an opportunity to establish an early effective filing date for a patent.

A provisional patent—and eventually a full patent—would give PharmaCyte a fresh 20 years of patent protection; however, even without it, PharmaCyte has protections in place to protect its technology that are in line with any biotechnology/pharmaceutical company developing treatments after many years. In most cases, the 20-year patent protection that biotechnology/pharmaceutical companies have in place while developing drugs/treatments, have their 20-year time frame cut in half to 10 years by the time the drug hits the marketplace.

Patents are typically awarded within a few years after the patent application submission, but a common misconception is that the patent begins only after the drug hits the market, so with this in mind, PharmaCyte will actually have longer protections in place than most biotechnology/pharmaceutical companies have upon marketing approval after years of development.

How is this possible? Well, actually PharmaCyte has two protections in place that will keep its technology protected—and unlike 20-year patent protection where the clock is ticking throughout development, these protections don’t begin until the therapy is approved. First, PharmaCyte is developing a "biologic" therapy, which offers protections under the Affordable Care Act, and second, PharmaCyte’s therapy for pancreatic cancer was granted Orphan Drug Designation from both the U.S. FDA and in the European Union by the European Medicines Agency (EMA).

PharmaCyte’s pancreatic cancer therapy was designated an orphan drug and listed in the official registry of medicinal products for rare diseases by the U.S. FDA on December 17, 2014. This orphan drug status assures marketing exclusivity for PharmaCyte’s pancreatic cancer therapy in the U.S. for 7 years after market approval by the FDA. Also, PharmaCyte has orphan drug status in the European Union (EU) for its pancreatic cancer therapy. This designation provides 10 years of marketing exclusivity in all countries in the EU following approval by the European Medicines Agency (EMA).

In addition, the Biologics Price Competition and Innovation Act (BPCIA), which was enacted as part of the Affordable Care Act in 2010, establishes a period of 12 years of "data exclusivity" for reference products to preserve incentives for future innovation. Under this framework, data exclusivity protects the data in the innovator’s regulatory application by prohibiting others, for a period of 12 years, from gaining FDA approval based in part on reliance on or reference to the innovator’s data in a biosimilar application. PharmaCyte’s 12-year exclusivity doesn’t begin until the FDA approves the company’s pancreatic cancer therapy.

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said of the protections for the company’s pancreatic cancer therapy, "If our pancreatic cancer therapy receives FDA approval, the orphan drug designation in the U.S. and the EU, together with the BPCIA data exclusivity, will give us substantial marketing exclusivity for our pancreatic cancer therapy. Any new patent application, while it does include our pancreatic cancer therapy, should really be viewed as an opportunity to dramatically broaden PharmaCyte’s ability to protect our therapy for all malignant tumors for the next 20 years."

On February 4, 2020 Celsion Corporation (NASDAQ: CLSN), an oncology drug-development company, reported Prof. Riccardo Lencioni, M.D., FSIR, EBIR delivered a presentation titled "Thermally-Sensitive Ablation Enhancers: Where Do We Stand?" at the SPECTRUM 2020 Interventional Oncology Conference held in Miami , FLA last month (Press release, Celsion, FEB 4, 2020, View Source [SID1234553840]). Dr. Lencioni is a professor in the Department of Radiology at the University of Pisa School of Medicine in Italy and is an Honorary Research Professor of Interventional Oncology at the Miami Cancer Institute. He was the lead principal investigator in Europe for the Company’s completed Phase III HEAT Study in hepatocellular carcinoma (HCC), or primary liver cancer, using radiofrequency ablation (RFA) plus ThermoDox, the Company’s lead product.

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

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

                  Schedule Your 30 min Free Demo!

The SPECTRUM Conference is the only interventional oncology conference endorsed by The American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). In his talk, Prof. Lencioni focused on the HEAT Study subgroup analysis showing the duration of RFA heating time per tumor volume of 45 minutes or longer plus ThermoDox was key to overall survival benefit in this patient population. He noted that in early-stage HCC, nearly 50% of patients with a solitary lesion of less than 5 cm on imaging have microsatellites on histology. While RFA and other energy sources are not able to treat these microsatellites, a thermosensitive drug carrier such as ThermoDox would deposit increased amounts of doxorubicin in the margins of a tumor given increased ablation time.

"The significant attention ThermoDox is receiving among key opinion leaders at important medical conferences has helped build awareness of our drug in combination with RFA for treating HCC," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "We are grateful to Prof. Lencioni for helping Celsion create the training video on RFA heating time for lesion sizes from 3 cm to 7 cm that is being used by all investigators in our current Phase III OPTIMA Study."

The Company’s OPTIMA Study was based on the prospective analysis of the HEAT Study subgroup of patients who received 45 minutes or more of RFA energy. The OPTIMA Study was fully enrolled in August 2018 with 556 subjects from 65 clinical sites in 14 countries. At its first interim analysis in November 2019 following 128 patient events, or deaths, the independent Data Monitoring Committee (iDMC) unanimously recommended the OPTIMA Study continue according to protocol based on safety and data integrity.

The Company re-affirms its projection that its second pre-planned interim efficacy analysis will occur during the second quarter of 2020, following 158 patient events, or deaths. The hazard ratio for success at 158 events is 0.70, which is below the hazard ratio of 0.65 observed for the 285 patients in the HEAT Study subgroup of patients treated with RFA of 45 minutes or longer.

Mr. Tardugno added, "We were pleased that the HCC part of the SPECTRUM program featured invited faculty with ties to Celsion, such as Dr. Josep Llovet, Founder and Director of the Liver Cancer Program and Full Professor of Medicine at the Mount Sinai School of Medicine in New York, who is chair of the OPTIMA iDMC, and Dr. Ghassan Abou-Alfa of Memorial Sloan Kettering Cancer Center and Professor, Weill Medical College at Cornell University, both in New York City, who was a member of the HEAT Study DMC. The medical community’s independent assessment, along with the National Institutes of Health’s published confirmation of the hypothesis supporting the potential for ThermoDox as a curative treatment for the largest unmet need in oncology is gratifying indeed."

On February 4, 2020 Horizon Therapeutics plc (Nasdaq: HZNP) reported that its fourth-quarter and full-year 2019 financial results will be released on Wednesday, Feb. 26, 2020 (Press release, Horizon Therapeutics, FEB 4, 2020, Horizon Therapeutics plc to Release Fourth-Quarter and Full-Year 2019 Financial Results and Host Webcast on Feb. 26, 2020 [SID1234553838]). Following the announcement, Horizon’s management will host a live webcast at 8 a.m. Eastern Time to review the Company’s financial and operating results.

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

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

                  Schedule Your 30 min Free Demo!

The live webcast and a replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay of the webcast will be available approximately two hours after the live webcast.

On February 4, 2020 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need, reported that Steven A. Shallcross, Chief Executive and Financial Officer, is scheduled to present at the BIO CEO & Investor Conference on Monday, February 10, 2020, at 11:00 a.m. (ET) at the New York Marriott Marquis (Wilder room) (Press release, Synthetic Biologics, FEB 4, 2020, View Source [SID1234553837]).

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

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

                  Schedule Your 30 min Free Demo!

A live webcast of Synthetic Biologics’ presentation may be accessed by logging onto the internet at http://www.veracast.com/webcasts/bio/ceoinvestor2020/17107426667.cfm. A replay will be archived and accessible for 90 days at the same website and at www.syntheticbiologics.com.

On February 4, 2020 Bio-Techne Corporation (NASDAQ:TECH) reported its financial results for the second quarter ended December 31, 2019 (Press release, Bio-Techne, FEB 4, 2020, View Source [SID1234553836]).

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

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

                  Schedule Your 30 min Free Demo!

Second Quarter FY2020 Snapshot

Second quarter organic revenue increased by 6% (6% reported) to $184.9 million and 9% (9% reported) in the first half of fiscal 2020 to $368.2 million.

GAAP EPS was $3.02 versus $0.45 one year ago resulting from a gain of approximately $121 million on our ChemoCentryx investment. Delivered adjusted earnings per share (EPS) of $1.08, an increase of 2% despite foreign currency exchange headwinds negatively impacting results by $0.08 or 8% in the second quarter.

Adjusted Operating Margin increased to 33.4% in the second quarter of fiscal 2020 compared to 32.5% in the second quarter of fiscal 2019.

Diagnostics and Genomics delivered organic growth of 12% in the second quarter and 14% in the first half of fiscal 2020.

As of December 1, 2019, applicable Medicare beneficiaries are now covered for the ExoDx Prostate Test.

The Company formed a new joint venture focused on providing scalable manufacturing technologies and processes needed to develop and commercialize new cell and gene therapies.

Delivered record Operating Cash Flow in the quarter and paid down $103 million of debt.
The Company’s financial statements are prepared in accordance with accounting principles generally accepted in the United States (GAAP). Adjusted EPS, adjusted earnings, adjusted gross margin, adjusted operating income, and adjusted operating margin are non-GAAP measures that exclude certain items detailed later in this press release under the heading "Use of non-GAAP Adjusted Financial Measures." A reconciliation of GAAP to non-GAAP financial measures is included in this press release.

"Q2 was a solid quarter of 6% organic growth, especially considering some one-time headwinds we had over last year," said Chuck Kummeth, President and Chief Executive Officer of Bio-Techne. "Our core retail reagents remained strong with double-digit growth in most product categories, while our Genomics’ ACD business continued growing at over 20% in Q2. As expected, China continued its trend of above 20% growth while Europe was soft year over year, especially with instrument sales. However, early in Q3, we are starting to see instrument order rates in Europe accelerate to growth rates that we are more accustomed to experiencing."

Kummeth added, "Overall, I am pleased with the team’s performance in the first half of the fiscal year. I am also encouraged by the strength in our end-markets and our excellent competitive position and look forward to executing even better in the second half of the fiscal year."

Second Quarter Fiscal 2020

Revenue

Net sales for the second quarter increased 6% to $184.9 million. Organic growth was 6% compared to the prior year, with foreign currency exchange having an unfavorable impact of 1% and acquisitions contributing 1% to revenue growth.

GAAP Earnings Results

GAAP EPS increase to $3.02 per diluted share, versus $0.45 in the same quarter last year. GAAP EPS was favorably impacted by a gain of approximately $121 million on our ChemoCentryx investment. GAAP operating income for the second quarter of fiscal 2020 increased 10% to $37.0 million, compared to $33.6 million in the second quarter of fiscal 2019. GAAP operating margin was 20.0%, compared to 19.3% in the second quarter of fiscal 2019. GAAP operating margin compared to prior year was positively impacted by volume leverage on sales growth.

Non-GAAP Earnings Results

Adjusted EPS increased to $1.08 per diluted share, versus $1.06 in the same quarter last year, an increase of 2%. Adjusted EPS increased due to revenue growth in the quarter, which was partially offset by foreign currency headwinds. Adjusted operating income for the second quarter of fiscal 2020 increased 9% compared to the second quarter of fiscal 2019. Adjusted operating margin was 33.4%, compared to 32.5% in the second quarter of fiscal 2019. Adjusted operating margin compared to the prior year was favorably impacted by volume leverage on sales growth.

Segment Results

Management uses adjusted operating results to monitor and evaluate performance of the Company’s business segments, as highlighted below. Since these results are used for this purpose, they are also considered to be prepared in accordance with GAAP.

Protein Sciences Segment

The Company’s Protein Sciences segment is one of the world’s leading suppliers of specialized proteins such as cytokines and growth factors, immunoassays, antibodies and reagents, to the biotechnology community. Additionally, the segment provides an array of platforms useful in various areas of protein analysis. Protein Sciences segment’s second quarter fiscal 2020 net sales were $141.5 million, an increase of 4% from $135.5 million for the second quarter of fiscal 2019. Organic growth for the segment was 4%, with foreign currency exchange having an unfavorable impact of 1% on revenue growth and acquisitions contributing 1% to revenue growth. Protein Sciences segment’s operating margin was 43.0% in the second quarter of fiscal 2020 compared to 43.5% in the second quarter of fiscal 2019. The segment’s operating margin compared to the prior year was negatively impacted by the acquisition of B-Mogen in Q4 of FY19 and unfavorable foreign exchange.

Diagnostics and Genomics Segment

The Company’s Diagnostics and Genomics segment provides blood chemistry and blood gas quality controls, hematology instrument controls, immunoassays and other bulk and custom reagents for the in vitro diagnostic market. The Diagnostics and Genomics segment also develops and provides in situ hybridization products as well as exosome-based diagnostics for various pathologies, including prostate cancer. The Diagnostics and Genomics segment’s second quarter fiscal 2020 net sales were $43.8 million compared to $39.3 million for the second quarter of fiscal 2019. Organic growth for the segment was 12%, with foreign currency exchange having an immaterial impact on revenue. The Diagnostics and Genomics segment’s operating margin was 2.2% in the second quarter of fiscal 2020 compared to -2.7% in the second quarter of fiscal 2019. The segment’s operating margin was favorably impacted by volume leverage.

Conference Call

Bio-Techne will host an earnings conference call today, February 4, 2020 at 8:00 a.m. CST. To listen, please dial 1-800-289-0438 or 1-323-794-2423 for international callers, and reference conference ID 2359733. A recorded rebroadcast will be available for interested parties unable to participate in the live conference call by going to:
View Source

The replay will be available from 11:00 a.m. CDT on Tuesday, February 4, 2020 until 11:00 p.m. CDT on Wednesday, March 4, 2020.

Use of non-GAAP Adjusted Financial Measures:

This press release contains financial measures that have not been calculated in accordance with accounting principles generally accepted in the U.S. (GAAP). These non-GAAP measures include:

Organic growth
Adjusted diluted earnings per share
Adjusted net earnings
Adjusted gross margin
Adjusted operating income
Adjusted operating margin
We provide these measures as additional information regarding our operating results. We use these non-GAAP measures internally to evaluate our performance and in making financial and operational decisions, including with respect to incentive compensation. We believe that our presentation of these measures provides investors with greater transparency with respect to our results of operations and that these measures are useful for period-to-period comparison of results.

Our non-GAAP financial measure of organic growth represents revenue growth excluding revenue from acquisitions within the preceding 12 months as well as the impact of foreign currency. Excluding these measures provides more useful period-to-period comparison of revenue results as it excludes the impact of foreign currency exchange rates, which can vary significantly from period to period, and revenue from acquisitions that would not be included in the comparable prior period.

Our non-GAAP financial measures for adjusted gross margin, adjusted operating margin, and adjusted net earnings, in total and on a per share basis, exclude the costs recognized upon the sale of acquired inventory, amortization of acquisition intangibles, acquisition related expenses inclusive of the changes in fair value of contingent consideration, and other non-recurring assessments. The Company excludes amortization of purchased intangible assets, purchase accounting adjustments, including costs recognized upon the sale of acquired inventory and acquisition-related expenses inclusive of the changes in fair value contingent consideration, and other non-recurring assessments from this measure because they occur as a result of specific events, and are not reflective of our internal investments, the costs of developing, producing, supporting and selling our products, and the other ongoing costs to support our operating structure. Additionally, these amounts can vary significantly from period to period based on current activity.

The Company’s non-GAAP adjusted operating margin and adjusted net earnings, in total and on a per share basis, also excludes stock-based compensation expense, which is inclusive of the employer portion of payroll taxes on those stock awards, restructuring, impairments of equity method investments, gain and losses from investments, and certain adjustments to income tax expense. Stock-based compensation is excluded from non-GAAP adjusted net earnings because of the nature of this charge, specifically the varying available valuation methodologies, subjective assumptions, variety of award types, and unpredictability of amount and timing of employer related tax obligations. Impairments of equity investments are excluded as they are not part of our day-to-day operating decisions. Additionally, gains and losses from other investments that are either isolated or cannot be expected to occur again with any predictability are excluded. Costs related to restructuring activities, including reducing overhead and consolidating facilities, are excluded because we believe they are not indicative of our normal operating costs. The Company independently calculates a non-GAAP adjusted tax rate to be applied to the identified non-GAAP adjustments considering the impact of discrete items on these adjustments and the jurisdictional mix of the adjustments. In addition, the tax impact of other discrete and non-recurring charges which impact our reported GAAP tax rate are adjusted from net earnings. We believe these tax items can significantly affect the period-over-period assessment of operating results and not necessarily reflect costs and/or income associated with historical trends and future results.