RareCyte announces the first blood-to-result PD-L1 Circulating Tumor Cell Assay

On February 27, 2020 RareCyte reported a liquid biopsy blood test for programmed death-ligand 1 (PD-L1), enabling customers to evaluate PD-L1 expression on circulating tumor cells (CTCs) with industry leading sensitivity. PD-L1 is a biomarker that directs checkpoint inhibitor immunotherapy treatment and liquid biopsy offers a noninvasive method to evaluate biomarker expression for treatment selection and patient monitoring in clinical research (Press release, RareCyte, FEB 27, 2020, View Source [SID1234554946]).

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With the RareCyte PD-L1 assay and the AccuCyte-CyteFinder system, blood from a single tube is processed to cells for CTC enumeration and PD-L1 biomarker expression analysis, and to plasma for optional cfDNA analysis. Single CTCs can also be retrieved using the CytePicker Module for sequencing analysis. As a result, the RareCyte assay provides the first comprehensive liquid biopsy solution for PD-L1 analysis.

The PD-L1 Panel Kit was validated based on rigorous requirements set to clinical standards. Tad George, PhD, SVP of Biology R&D at RareCyte noted, "Our approach to assay development and validation is centered on creating globally deployable products that combine the sensitivity, specificity, and precision required for multi-center clinical trials for CTC enumeration and phenotyping. In addition, the RareCyte platform enables cell-free DNA analysis on the same sample, providing a comprehensive assessment of patient status."

Joe Victor, CEO of RareCyte, said "We released this new assay as a response to customer feedback from an earlier generation assay still in use today and we anticipate that this PD-L1 Panel Kit will be utilized in a variety of exploratory clinical research this year." The PD-L1 Panel Kit is now available for purchase. More information on the PD-L1 Panel Kit and the RareCyte platform is available at View Source

RareCyte products are for research use only. Not for use in diagnostic procedures.

Menarini Silicon Biosystems to Announce New Solutions for Advancing Liquid Biopsy in Clinical Research

On February 27, 2020 Menarini Silicon Biosystems, the pioneer of liquid biopsy and rare cell technologies, reported that it is developing several new products that could help advance precision medicine research by expanding the capabilities and applications of its CELLSEARCH and DEPArray technologies, for rare cells isolation and single cell analysis (Press release, Menarini Silicon Biosystems, FEB 27, 2020, View Source [SID1234554945]).

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The new solutions include an assay for the enumeration and molecular characterization of circulating multiple myeloma cells (CMMC) from peripheral blood samples, the DEPArraysystem upgraded to 9 colors and artificial intelligence software.

The company’s technological developments will be highlighted during a presentation by Mark Connelly, PhD, Chief Research and Development Officer at Menarini Silicon Biosystems Inc, as part of the Molecular Medicine Tri-Conference’s Circulating Tumor Cells & Liquid Biopsy track.

"We are continually working to develop additional tools that will facilitate the exploration of new areas of inquiry," said Connelly. "Providing researchers with more information from a wider variety of cell populations and the ability to examine tumor heterogeneity in real-time could help address some critical questions in pharmaceutical and clinical research."

The CMMC assay based on the capabilities of CELLSEARCH to enrich and enumerate rare cells allow researchers to monitor the number of CMMCs in peripheral blood; furthermore when combined with the DEPArray system, will allow to perform molecular analysis at single cell level**. Research suggests that the ability to obtain CMMCs via liquid biopsy may help clinical researchers to better monitor disease state and treatment response, though, not only the cell enumeration but also the real time cell molecular profiling. Menarini Silicon Biosystems currently offers a CMMC assay (Research Use Only) as a service through its Global Laboratory Services. The company plans to release the assay for Research Use Only in the summer of 2020.

The 9-Color DEPArray will offer more fluorescence channels to examine the heterogeneity of markers expression on various rare cells, enabling researchers to simultaneously identify and characterize multiple cell populations. The artificial intelligence software will reduce operator variability by automating cell identification and selection and will help researchers move beyond enumeration to unravel hidden information in the cell image data.

Connelly’s presentation will also feature new research supporting the potential use of CTCs as a response biomarker for prostate cancer treatment. A 2018 study published in the Journal of Clinical Oncology indicates that using CTC counts obtained from liquid biopsy after 13 weeks of treatment can provide an accurate assessment of response to treatment. PSA (prostate-specific antigen) is one of the only markers currently available for the disease but is not considered to be a sufficient indicator of treatment response.

Menarini Silicon Biosystems is working with the FDA to get this application qualified as a response biomarker. This would enable researchers to incorporate CTCs as a response biomarker into clinical trials to expedite the drug development process.

Jennifer Davids, PhD, Director of Field Scientific Support at Menarini Silicon Biosystems Inc, will also be presenting on how the DEPArray technology can overcome the challenges of sample heterogeneity by enabling rare cell sorting with single cell precision.

Presentation Details
Location: Moscone South Convention Center

Monday, March 2 at 1:05 pm
Title: Advancing Liquid Biopsy – presented by Mark Connelly, PhD
Program: Circulating Tumor Cells and Liquid Biopsy
Track: Companion Diagnostics with Liquid Biopsy

Tuesday, March 3 at 11:40 am
Title: Utilizing the Next Generation Single Cell Sorter, DEPArray – presented by Jennifer Davids, PhD
Program: Immuno-Oncology Biomarkers and Companion Diagnostics
Track: Neoantigen Approaches

"These exciting developments demonstrate our continued commitment in advancing the applications of liquid biopsy," said Fabio Piazzalunga, President of Menarini Silicon Biosystems. "Our goal at MSB is to contribute to the advancement of precision medicine through innovative rare cell technologies, and we are actively working with our partners in the research community to provide advanced integrated and automated solutions."

To learn more about the CELLSEARCH and DEPArray Systems, MMTC attendees can visit Menarini Silicon Biosystems at Booth #601.

The 27th International Molecular Medicine Tri-Conference will be held March 1-4, 2020 in San Francisco.

ANI Pharmaceuticals Reports Full Year and Fourth Quarter 2019 Results, Provides 2020 Guidance and Remains on Track to Submit Cortrophin® Gel sNDA to FDA in March 2020

On February 27, 2020 ANI Pharmaceuticals, Inc. ("ANI") (NASDAQ: ANIP) reported its financial results for the three and twelve months ended December 31, 2019 and provided its 2020 financial guidance (Press release, ANI Pharmaceuticals, FEB 27, 2020, https://www.prnewswire.com/news-releases/ani-pharmaceuticals-reports-full-year-and-fourth-quarter-2019-results-provides-2020-guidance-and-remains-on-track-to-submit-cortrophin-gel-snda-to-fda-in-march-2020-301012068.html [SID1234554944]). The Company will host its earnings conference call this morning, February 27, 2020, at 10:30 AM ET. Investors and other interested parties can join the call by dialing (866) 776-8875. The conference ID is 4783883.

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"ANI generated record net revenues and non-GAAP gross profit in 2019 while continuing to diversify its commercial product offering and pipeline opportunities. While revenues and non-GAAP gross profit for three of our important generic products were negatively impacted in recent periods, overall our business remains resilient, and we look forward to leveraging important recent launches such as Vancomycin Oral Solution and Bretylium Tosylate Injection as well as the recently acquired Amerigen portfolio in 2020. During 2019, we launched several new products, entered into collaborative arrangements with strategic partners and acquired both previously approved and development stage products we plan to launch in the future. We have utilized strong operating cash flows for asset acquisitions and investment into our portfolio of pipeline products and expect to continue to do so in the future.

During the fourth quarter, we successfully completed our fourth commercial scale batch of both Corticotropin API and the Cortrophin Gel drug product, and importantly, remain on track to submit our supplemental NDA filing in March 2020."

2020 Financial Guidance

For the twelve months ending December 31, 2020, ANI is providing guidance on net revenue, adjusted non-GAAP EBITDA, and adjusted non-GAAP diluted earnings per share. The following table summarizes 2020 guidance as compared to 2019 actual results:

In addition, we expect that adjusted non-GAAP gross margin, defined as the result of Net Revenues less Cost of Sales (excluding depreciation and amortization) as a percentage of Net Revenues, will decline from approximately 71% in 2019 to the mid 60% range in 2020, driven by the impact of negative price and product mix. Year over year changes of our Adjusted non-GAAP diluted earnings per share metric differ from our Adjusted non-GAAP EBITDA metric due to a projected increase in cash interest expense resulting from the refinancing of our convertible debt to bank debt.

ANI’s full year 2020 financial guidance reflects management’s current assumptions regarding customer relationships, product pricing, prescription trends, competition, inventory levels, cost of sales, operating costs, timing of research and development spend, taxes, and the anticipated timing of future product launches and other key events.

Cortrophin Gel Re-commercialization Update

Product

Required Filing

Expected Filing Date

Total Annual Market(d)

Cortrophin Gel

sNDA

March 2020

$1.0 billion

(d) Based on data from IQVIA

We continue to successfully progress our Cortrophin Gel re-commercialization program. Significant accomplishments since the third quarter 2019 press release (dated November 6, 2019) include:

ANI successfully completed API process validation by completing the fourth commercial scale batch of Corticotropin API. ANI also completed manufacturing for a fifth commercial scale batch of Corticotropin API. All five commercial scale batches were analytically consistent with each other and met all API release specifications. ANI expects to have 6 months stability on all API registration batches prior to the sNDA filing and by the end of first quarter 2020.
ANI successfully completed drug product process validation in the fourth quarter of 2019. ANI also completed manufacturing of a fourth commercial scale batch of Cortrophin Gel. This batch was analytically consistent with previously manufactured batches and met all drug product release specifications. ANI had already completed manufacturing for three commercial scale registration stability batches of Cortrophin Gel and expects to have 6 months stability on each prior to the sNDA filing and by the end of first quarter 2020.
ANI is on track to file the sNDA as planned by March 2020.

For further details, please see ANI’s Cortrophin Gel Re-commercialization Milestone Update in Table 5.

Fourth Quarter Results

Generic Pharmaceutical Products

Fourth Quarter Net Revenues – Results and Update

Net revenues from sales of generic pharmaceuticals decreased 14% to $29.1 million from $33.7 million in the prior period, primarily due to decreases in sales of Esterified Estrogen with Methlytestosterone ("EEMT"), Erythromycin Ethylsuccinate ("EES"), Propafenone, and Fenofibrate. These decreases were tempered by the September 2019 launch of Vancomycin HCl for Oral Solution as well as increased unit sales of Vancomycin tablets.

Branded Pharmaceutical Products

Fourth Quarter Net Revenues – Results and Update

Net revenues from sales of branded pharmaceuticals decreased 17% to $15.6 million from $18.8 million in the prior period, primarily due to decrease in sales of Inderal LA, Atacand and Atacand HCT. These decreases were tempered by increased sales of InnoPran XL.

Contract Manufacturing

Fourth Quarter Net Revenues – Results and Update

Contract manufacturing revenues decreased 28% to $2.6 million from $3.7 million in the prior year period, due to the timing and volume of orders from contract manufacturing customers in the period.

Royalty and Other

Fourth Quarter Net Revenues – Result and Update

Royalty and Other decreased 34% to $0.6 million from $0.9 million, primarily due to the timing and volume of product development and laboratory services revenue earned by ANI Canada.

Operating Expenses

Operating expenses increased to $52.6 million for the three months ended December 31, 2019, from $45.7 million in the prior year period. The increase was primarily due to the following:

$6.5 million in the build of Cortrophin pre-launch commercial inventories (which are expensed for US GAAP); there were no such activities in 2018,
$1.2 million increase in research and development expense, primarily due to Q4 2019 development-based milestone payments earned by collaborative partners, and
$0.9 million increase in depreciation and amortization expense, primarily due to additional amortization expense associated with a March 2019 asset acquisition and a January 2019 royalty buyout payment related to a prior period asset acquisition.
These increases were partially offset by a $2.3 million decrease in cost of sales as a result of the previously mentioned royalty buyout in January 2019 and a decrease in sales over the comparable periods, tempered by Q4 2019 inventory reserve charges of $4.6 million primarily related to the Company’s exit from the Methylphenidate Extended Release market.

Cost of sales as a percentage of net revenues increased to 37% during the three months ended December 31, 2019, from 35% during same period in 2018. The increase was primarily due to the inventory reserve charges recognized in the fourth quarter 2019 and negative price, which were tempered by reductions related to the 2019 royalty buyout.

Net Loss and Diluted Loss per Share

Net loss was $4.8 million for the three months ended December 31, 2019, as compared to net income of $5.4 million in the prior year period. The net loss was driven by the previously mentioned $6.5 million build of Cortrophin pre-launch commercial inventories and $4.6 million of inventory reserve charges. The effective consolidated tax benefit rate for the three months ended December 31, 2019 was 37%.

Diluted loss per share for the three months ended December 31, 2019 was $0.41, based on 11,886 thousand diluted shares outstanding, as compared to diluted earnings per share of $0.46 in the prior year period. Adjusted non-GAAP diluted earnings per share was $1.08, as compared to adjusted non-GAAP diluted earnings per share of $1.32 in the prior year period. For a reconciliation of adjusted non-GAAP diluted earnings per share to the most directly comparable GAAP financial measure, please see Table 4.

ANI Product Development Pipeline

ANI’s pipeline consists of 118 products, addressing a total annual market size of $7.0 billion, based on data from IQVIA. Of these, ANI expects that at least 52 can be commercialized based on either CBE-30s or prior approval supplements filed with the FDA.

Non-GAAP Financial Measures

The Company’s fiscal 2019 guidance for adjusted non-GAAP EBITDA and adjusted non-GAAP diluted earnings per share is not reconciled to the most comparable GAAP measure. This is due to the inherent difficulty of forecasting the timing or amount of items that would be included in a reconciliation to the most directly comparable forward-looking GAAP financial measures. Because a reconciliation is not available without unreasonable effort, it is not included in this release.

Adjusted non-GAAP EBITDA

ANI’s management considers adjusted non-GAAP EBITDA to be an important financial indicator of ANI’s operating performance, providing investors and analysts with a useful measure of operating results unaffected by non-cash stock-based compensation and differences in capital structures, tax structures, capital investment cycles, ages of related assets, and compensation structures among otherwise comparable companies. Management uses adjusted non-GAAP EBITDA when analyzing Company performance.

Adjusted non-GAAP EBITDA is defined as net income/(loss), excluding tax expense, interest expense, depreciation, amortization, the excess of fair value over cost of acquired inventory, stock-based compensation expense, expense from acquired in-process research and development, gains, losses, and expenses related to the repurchase of convertible debt, expenses related to debt financing, transaction and integration expenses, non-cash impairment charges, Cortrophin pre-launch charges, other income / expense and certain other items, including expensed milestone payments and inventory reserve charges, that vary in frequency and impact on ANI’s results of operations. Adjusted non-GAAP EBITDA should be considered in addition to, but not in lieu of, net income or loss reported under GAAP. A reconciliation of adjusted non-GAAP EBITDA to the most directly comparable GAAP financial measure is provided in Table 3.

Adjusted non-GAAP Net Income

ANI’s management considers adjusted non-GAAP net income to be an important financial indicator of ANI’s operating performance, providing investors and analysts with a useful measure of operating results unaffected by purchase accounting adjustments, non-cash stock-based compensation, non-cash interest expense, depreciation and amortization, non-cash impairment charges, Cortrophin pre-launch charges and certain other items, including expensed milestone payments and inventory reserve charges, that vary in frequency and impact on ANI’s results of operations. Management uses adjusted non-GAAP net income when analyzing Company performance.

Adjusted non-GAAP net income is defined as net income/(loss), plus the excess of fair value over cost of acquired inventory, stock-based compensation expense, transaction and integration expenses, gains, losses, and expenses related to the repurchase of convertible debt, expenses related to debt financing, non-cash interest expense, depreciation and amortization expense, expense from acquired in-process research and development, non-cash impairment charges, Cortrophin pre-launch charges and certain other items, including expensed milestone payments and inventory reserve charges, that vary in frequency and impact on ANI’s results of operations, less the tax impact of these adjustments calculated using an estimated statutory tax rate. Management will continually analyze this metric and may include additional adjustments in the calculation in order to provide further understanding of ANI’s results. Adjusted non-GAAP net income should be considered in addition to, but not in lieu of, net income reported under GAAP. A reconciliation of adjusted non-GAAP net income to the most directly comparable GAAP financial measure is provided in Table 4.

Adjusted non-GAAP Diluted Earnings per Share

ANI’s management considers adjusted non-GAAP diluted earnings per share to be an important financial indicator of ANI’s operating performance, providing investors and analysts with a useful measure of operating results unaffected by purchase accounting adjustments, non-cash stock-based compensation, non-cash interest expense, depreciation and amortization, non-cash impairment charges, Cortrophin pre-launch charges and certain other items, including expensed milestone payments and inventory reserve charges, that vary in frequency and impact on ANI’s results of operations.

Management uses adjusted non-GAAP diluted earnings per share when analyzing Company performance.

Adjusted non-GAAP diluted earnings per share is defined as adjusted non-GAAP net income, as defined above, divided by the diluted weighted average shares outstanding during the period, as adjusted for the dilutive effect of the convertible debt notes (in 2019), when applicable. Management will continually analyze this metric and may include additional adjustments in the calculation in order to provide further understanding of ANI’s results. Adjusted non-GAAP diluted earnings per share should be considered in addition to, but not in lieu of, diluted earnings or loss per share reported under GAAP. A reconciliation of adjusted non-GAAP diluted earnings per share to the most directly comparable GAAP financial measure is provided in Table 4.

Cryoport to Report Fourth Quarter and Fiscal Year 2019 Financial Results on March 5, 2020

On February 27, 2020 Cryoport, Inc. (NASDAQ: CYRX) (NASDAQ: CYRXW) ("Cryoport" or the "Company"), a global leader in life sciences solutions, reported that the Company will report financial results for the fourth quarter and fiscal year ended December 31, 2019 on Thursday, March 5, 2020 after U.S. markets close (Press release, Cryoport, FEB 27, 2020, View Source [SID1234554942]).

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In addition to the earnings release, a document titled "Cryoport 2019 Year in Review", which will provide a review of Cryoport’s recent financial and operational performance and a general business update, will be issued by management at 4:05 pm ET on Thursday, March 5, 2020. The document is designed to be read by investors before the questions and answers conference call and can be accessed at http://ir.cryoport.com/events-and-presentations.

Cryoport management will host a conference call at 5:00 pm ET on March 5, 2020. The conference call will be in the format of a questions and answers session and will address any queries investors have regarding the Company’s reported results.

Conference Call Information

Date:

March 5, 2020

Time:

5:00 p.m. ET

Dial-in numbers:

+1 (855) 327-6837 (U.S.), +1 (631) 891-4304 (International)

Confirmation code:

Request the "Cryoport Call"

Live webcast:

‘Investor Relations’ section at www.cryoport.com or at this link. Please allow 10 minutes prior to the call to visit this site to download and install any necessary audio software.

An archive of the question and answer webcast will be available approximately three hours after completion of the live event and will be accessible on the Investor Relations section of the Company’s website at www.cryoport.com for a limited time. To access the replay of the webcast, please follow this link. A dial-in replay of the call will also be available to those interested until March 12, 2020. To access the replay, dial +1 (844) 512-2921 (United States) or +1 (412) 317-6671 (International) and enter replay pin number: 10008682.

Oramed Pharmaceuticals Inc. Prices Public Offering of Common Stock for Aggregate Proceeds of $21 Million

On February 27, 2020 Oramed Pharmaceuticals Inc. (Nasdaq: ORMP) (TASE: ORMP) ("Oramed" or "Company"), a clinical-stage pharmaceutical company focused on the development of oral drug delivery systems, reported the pricing of an underwritten public offering of 5,250,000 shares of its common stock at a public offering price of $4.00 per share (Press release, Oramed Pharmaceuticals, FEB 27, 2020, View Source [SID1234554941]). All of the shares in the offering are to be sold by Oramed. Oramed has granted the underwriters a 45-day option to purchase up to 787,500 additional shares of common stock at the public offering price, less the underwriting discount. The offering is expected to close on or about March 2, 2020, subject to the satisfaction of customary closing conditions.

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The gross proceeds from the offering, before deducting the underwriting discount and estimated offering expenses payable by Oramed, are expected to be $21 million. The Company intends to use the net proceeds of the offering for its anticipated Phase 3 clinical trial in ORMD-0801 (Oral Insulin) and for other clinical trials and research and development activities as well as for general corporate purposes.

National Securities Corporation, a wholly owned subsidiary of National Holdings Corporation, is acting as the sole book running manager for the offering.

A.G.P./Alliance Global Partners is acting as financial advisor for the offering.

The shares of common stock are being offered by Oramed pursuant to its shelf registration statement on Form S-3 (File No. 333-236194) previously filed and declared effective by the Securities and Exchange Commission (the "SEC") on February 10, 2020. The offering is being made only by means of a prospectus supplement and accompanying prospectus. A preliminary prospectus supplement and accompanying prospectus relating to the underwritten public offering have been filed with the SEC and are available on the SEC’s website at View Source A final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website. When available, copies of the final prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from National Securities Corporation, Attn: Charles Wanyama, 200 Vesey Street, 25th Floor, New York, New York 10281, telephone: (212) 417-3634, or by email at [email protected].

This press release shall not constitute an offer to sell or a 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.