Perrigo Reports Third Quarter 2020 Financial Results

On November 4, 2020 Perrigo Company plc (NYSE; TASE: PRGO), a leading provider of Quality, Affordable Self-Care Products, reported financial results for the third quarter ended September 26, 2020 (Press release, Perrigo Company, NOV 4, 2020, View Source [SID1234569855]).

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President and CEO, Murray S. Kessler commented, "Results for the first nine months of this year reflect the strength of Perrigo’s diversified business model, the successful execution of our transformational activities and the dedication and agility of our 11,000 team members worldwide who are successfully navigating this horrific global pandemic."

Kessler continued, "Year to date, Worldwide Consumer net sales have grown high-single-digits, and importantly, organic growth is well above our 3% net sales goal. Consumer Self-Care Americas once again led the way with robust growth in all major franchises, resulting in record third quarter net sales as the segment benefited from channel shifting to e-Commerce and successful new product launches. Better than expected recoveries in both the CSC International and RX base businesses also contributed as our branded self-care and generic prescription product categories that were negatively impacted by consumer/patient buying dynamics surrounding COVID-19 lock-downs improved to near pre-COVID-19 levels. I am pleased that the strength of our businesses allowed us to reaffirm guidance, despite the disappointing RX albuterol recall."

Kessler concluded, "Our self-care evolution has accelerated Perrigo’s growth over the past 18 months and has us very well positioned in a ‘New World’ where self-care, value and e-Commerce will be more important than ever before. While there is still work to be done on our transformation, our team is energized about the future and remains committed to our long-term growth targets. Looking ahead, we will continue to leverage our unique and durable capabilities, along with more than $1 billion in committed investments to date, to recapture the Perrigo Advantage and create long-term value for our shareholders."

Third Quarter Financial Highlights

Consolidated third quarter net sales were $1.2 billion, an increase of 1.3% compared to the prior year quarter.
Worldwide Consumer third quarter net sales grew 3.6% compared to the prior year. Excluding the impact of currency and divested businesses, net sales increased 4.2%. Organic net sales grew 1.6%.
Consumer Self-Care Americas ("CSCA") achieved record third quarter net sales of $664 million, up 7.3% versus the prior year. Organic net sales grew 4.0% as consumer demand in the U.S. remained strong.
Consumer Self-Care International ("CSCI") third quarter net sales of $339 million were down 2.9% versus the prior year quarter, due primarily to European consumer dynamics surrounding COVID-19.
Reported diluted net loss per share for the third quarter was $1.13 as compared to EPS of $0.67 in the prior year, due primarily to a $202 million dollar impairment of goodwill in the RX segment.
Adjusted diluted EPS for the third quarter of 2020 decreased 10.6% to $0.93 as compared to $1.04 per diluted share in the prior year. Diluted EPS included a $0.14 charge from the third quarter RX albuterol recall.
Year-to-Date 2020 Financial Highlights

Consolidated year to date net sales were $3.8 billion, up 7.8% compared to the prior year. Excluding the impact of currency and divested businesses, net sales increased 9.7%. Organic net sales grew 4.4%.
Worldwide Consumer net sales increased 8.9% compared to the prior year. Excluding the impact of currency and divested businesses, net sales were 11.3% higher year-over-year. Organic net sales grew 4.6%.
CSCA achieved year-to-date net sales of $2.0 billion, 13.2% higher versus the prior year, or 14.9% higher excluding the impact of currency and divested businesses. Organic net sales grew 6.7%.
CSCI year-to-date net sales increased 1.5% versus the prior year, or 5.0% higher excluding divested businesses and the impact of currency. Organic new sales grew 1.0%.
Cash flow from operations as a percentage of adjusted net income was 124%.

(1) Unless otherwise noted, all comparisons of operating results against the prior year period include the previously disclosed third quarter 2019 net sales adjustments for the market withdrawal of Ranitidine, of which $7.4 million was included in the CSCA segment and $1.8 million was included in the CSCI segment, as well as $1.8 million in operating results attributable to the then held-for-sale animal health business in our CSCA segment. See attached Appendix for additional details.

(2) See attached Appendix for details. Organic net sales growth excludes the effects of acquisitions and divestitures and the impact of currency.

Refer to Tables I – IV at the end of this press release for a reconciliation of non-GAAP adjustments to the current year and prior year periods and additional non-GAAP information. The Company’s reported results are included in the attached Condensed Consolidated Statements of Operations, Balance Sheets and Statements of Cash Flows.

Third Quarter 2020 Consolidated Results Versus Third Quarter 2019

Consolidated net sales for the third quarter of calendar year 2020 were $1.2 billion, an increase of $15 million or 1.3%. Organic net sales declined 0.4% and included a 0.7 percentage point net negative impact from RX albuterol sulfate as product sales early in the quarter were more than offset by the establishment of the estimated albuterol reserve associated with the September recall.

The $15 million increase in consolidated net sales was driven by 1) $48 million in year over year growth in the CSCA segment driven by $24 million of increased demand across most product categories reflecting continued consumer channel shifting from traditional brick and mortar outlets to e-Commerce and $24 million from the Dr. Fresh oral self-care acquisition, and 2) $10 million in net favorable currency movements. These consolidated gains were partially offset by 1) a $20 million decline in the RX segment as $23 million in net sales of albuterol sulfate in the quarter were more than offset by a $31 million impact from the reserve for the estimated albuterol sulfate recall costs and $9 million in lower-margin discontinued products, 2) $15 million from divested businesses, and 3) a decline of $8 million in CSCI mainly driven by categories impacted by COVID-19 lock-downs.

Reported net loss was $155 million, or $1.13 per diluted share, versus net income of $92 million, or $0.67 per diluted share in the prior year period. Excluding certain charges as outlined in Table I, third quarter 2020 adjusted net income was $128 million, or $0.93 per diluted share, versus $142 million, or $1.04 per diluted share, for the same period last year resulting in a 10.6% decrease in adjusted diluted EPS.

Worldwide Consumer Self-Care Third Quarter 2020 Results Versus Third Quarter 2019

Worldwide Consumer is comprised of the CSCA segment, the CSCI segment and Corporate.

Worldwide Consumer Self-Care third quarter net sales increased $35 million, or 3.6%, to $1.0 billion with organic net sales growth of 1.6%, driven by growth in CSCA.

Third quarter reported gross profit margin was 37.0%. Adjusted gross profit margin of 39.2%, was 120 basis points lower year-over-year due to strong growth in the lower gross margin store brand business and the net gross margin impact of acquisitions and divestitures.

Reported operating margin was 8.5%. Adjusted operating margin decreased 180 basis points year-over-year to 14.0% due primarily to gross margin flow-through and higher variable compensation costs.

CSCA Third Quarter 2020 Results Versus Third Quarter 2019

Consumer Self-Care Americas achieved record third quarter net sales of $664 million, an increase of 7.3% or $45 million, which included $24 million from the acquisition of Dr. Fresh oral self-care and unfavorable currency movements of $3 million. Organic net sales grew 4.0% driven by increased demand across most categories that benefited from robust e-Commerce growth as consumers continued to shift purchasing towards online where Perrigo has greater market share, more than offsetting lower traditional brick and mortar purchases as measured through IRI MULO.

In OTC, increased demand was led by the pain, allergy, and digestive health categories resulting from strong e-Commerce growth and incremental volume from new products including Prevacid(R) and the store brand versions of Diclofenac Gel 1% and Esomeprazole Mini. Partially offsetting these were lower net sales of cough and cold products within the upper respiratory category and normal pricing pressure.

As a component of transforming to a consumer self-care company, Perrigo has made investments to enhance its consumer analytical and digital capabilities. Historically, CSCA measured its market share using only IRI’s MULO or Multi-Outlet data, which does not capture certain retailers and sales through the e-commerce channel. For the first time, the Company has the capabilities to measure, analyze, and benchmark omnichannel performance for Perrigo, store brand and the overall OTC category using IRI MULO point-of-sale (POS) data, IRI panel e-commerce data and POS data from Perrigo’s leading customers.

Total Perrigo omnichannel POS data was up 3.5% for the 13-weeks ending October 4, 2020. Total OTC omnichannel POS data was estimated to be up 1.2% in the categories in which Perrigo competes, resulting in a Perrigo share gain of 70 basis points.

In Nutrition, higher net sales were led by the December 2019 store brand infant formula launch at a major retailer, greater shipments in the infant formula contract manufacturing business and growth in customer e-Commerce activities. In Oral Self-Care, net sales were driven by the Dr. Fresh acquisition and strong e-Commerce growth.

Third quarter reported gross margin was 32.7%. Adjusted gross margin of 33.5% was 50 basis points lower than the prior year as higher margin new products were more than offset by normal pricing pressure. Third quarter 2020 adjusted gross margin was 60 basis points higher and 210 basis points higher than the second and first quarters of 2020, respectively.

Reported operating margin was 18.6%. Adjusted operating margin increased 30 basis points to 20.1%, due primarily to operating leverage on gross margin flow-through.

CSCI Third Quarter 2020 Results Versus Third Quarter 2019

Consumer Self-Care International net sales were $339 million, a decrease of $10 million, or 2.9%. Organic net sales were lower by 2.7%.

The decline in net sales was due primarily to 1) divested businesses of $15 million and discontinued products of $3 million, 2) lower sell-in activities to customers of cough and cold OTC brands within the upper respiratory category, and 3) lower consumer demand for anti-parasite products within the skincare & personal hygiene category due primarily to COVID-19 related school closings. These were partially offset by gains in 1) new products, including line extensions in the ACO dermatology product line and new innovations to the XLS Forte Five weight management brand, 2) $12 million in favorable currency movements, and 3) higher net sales in the pain and VMS categories, both of which benefited from consumer behavior surrounding COVID-19.

Reported gross margin was 45.5%. Adjusted gross margin of 50.4% declined 150 basis points due primarily to divested businesses, less favorable product mix and higher input costs on a particular OTC brand.

Reported operating margin was 3.0%. Adjusted operating margin decreased 310 basis points to 15.1% due to gross profit flow-through and divested businesses.

RX Third Quarter 2020 Results Versus Third Quarter 2019

RX net sales were $211 million, $20 million or 8.5%, lower than the prior year. Net sales from albuterol sulfate in the quarter of $23 million were more than offset by a $31 million reserve for the estimated impact from the albuterol sulfate recall and $9 million in lower-margin discontinued products. The RX base business, which excludes albuterol sulfate and discontinued products in both years, was down 1.2% compared to the prior year and reflected a faster than anticipated recovery in dermatology prescriptions from the second quarter of 2020.

Reported gross margin was 27.0% and adjusted gross margin was 37.1%. The 380 basis point decline in adjusted gross margin was due primarily to impact from the reserve for the estimated albuterol sulfate recall costs.

Reported operating margin was (85.7)%, driven primarily by the $202 million goodwill impairment charge taken in the quarter. Adjusted operating margin was 20.7%, down 330 basis points as lower gross profit was partially offset by lower R&D and administration expenses.

Fiscal 2020 Outlook

The Company reaffirms its fiscal 2020 outlook with expected net sales growth of 6% to 7% with organic net sales growth of at least 3%. Adjusted diluted EPS is expected to be in the range of $3.95 to $4.15. The reaffirmed guidance reflects strong business fundamentals and a lower expected tax rate for the year, offsetting $0.12 – $0.15 per adjusted diluted share of incremental COVID-19 related costs, $0.14 per share impact from the RX albuterol sulfate recall, and $0.06 per share from the divested Rosemont Rx business, which the Company sold on June 19, 2020. Future waves of COVID-19 could present both incremental risks as well as opportunities for the Company’s business.

The Company cannot reconcile its expected adjusted diluted earnings per share to diluted earnings per share under "Fiscal 2020 Outlook" without unreasonable effort because certain items that impact net income and other reconciling metrics are out of the Company’s control and/or cannot be reasonably predicted at this time.

MannKind Corporation Reports 2020 Third Quarter Financial Results

On November 4, 2020 MannKind Corporation (NASDAQ:MNKD) reported financial results for the quarter and nine months ended September 30, 2020 (Press release, Mannkind, NOV 4, 2020, View Source [SID1234569854]).

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"Our employees continue to execute in a tumultuous Covid-19 environment," said Michael Castagna, Chief Executive Officer. "As we head toward year-end, our focus remains on two significant drivers of value for shareholders: enabling healthcare providers to increase their prescribing of Afrezza and supporting United Therapeutics as they move the TreT clinical program to completion, including manufacturing TreT clinical supplies."

Third Quarter 2020 Results

Total revenues were $15.4 million for the third quarter of 2020, reflecting Afrezza net revenue of $7.3 million and collaboration and services revenue of $8.1 million. Afrezza U.S net revenue of $7.3 million increased 27% compared to $5.7 million in the third quarter of 2019, driven by higher product demand, a favorable mix of cartridges, and price. The first sale of Afrezza to the Company’s marketing partner in Brazil was completed in the third quarter of 2019 for $0.7 million; there were no international sales in the third quarter of 2020. Collaboration and services revenue for the third quarter of 2020 decreased $0.1 million compared to the third quarter of 2019.

Afrezza gross profit for the third quarter of 2020 was $3.7 million compared to a loss of $0.7 million in the same period of 2019, an increase of $4.4 million that was driven primarily by higher Afrezza revenue combined with a reduction in cost of goods sold, primarily attributable to a $2.8 million amendment fee associated with our insulin supply agreement in 2019. Non-GAAP gross margin in the third quarter of 2020 increased to 51% from 32% for the same quarter in 2019.

Selling, general and administrative expenses for the third quarter of 2020 were $13.9 million compared to $16.7 million for the third quarter of 2019. This 17% decrease was primarily due to a $1.2 million reduction in promotional and marketing activities and a $0.8 million decrease in professional costs.

Interest expense for the third quarter of 2020 was $2.4 million compared to $5.3 million for the third quarter of 2019. This $2.9 million decrease was primarily attributable to a $3.4 million milestone obligation that occurred in the third quarter of 2019, partially offset by interest expense from the MidCap Credit Facility.

A loss on foreign currency translation for the third quarter of 2020 was $3.9 million compared to a $3.8 million gain for the third quarter of 2019. The fluctuation is related to the change in the U.S. dollar to Euro foreign exchange rate on the recognized loss on insulin purchase commitments, which are denominated in Euros.

The net loss for the third quarter of 2020 was $11.3 million, or $0.05 per share, compared to a $10.4 million net loss, or $0.05 per share in the third quarter of 2019. The higher net loss was mainly attributable to the change in foreign currency translation from a gain in 2019 to a loss in 2020, partially offset by the reduction in cost of goods sold, selling, general and administrative expenses and interest expense.

Nine Months Ended September 30, 2020

Total revenues were $46.7 million for the nine months ended September 30, 2020, reflecting Afrezza net revenue of $22.3 million and collaboration and services revenue of $24.4 million. Afrezza net revenue increased 27% compared to $17.5 million for the nine months ended September 30, 2019, primarily driven by higher product demand, a more favorable mix of cartridges and price, partially offset by a reduction in sales to Biomm (Brazil). Collaboration and services revenue for the nine months ended September 30, 2020 decreased $5.1 million compared to the same period in the prior year, primarily due to a $5.7 million decrease in revenue recognized from the UT Research Agreement, which was substantially completed in 2019.

Afrezza gross profit for the nine months ended September 30, 2020 was $10.8 million compared to $2.1 million in the same period of 2019, an increase of $8.7 million, or +416%, that was driven primarily by a reduction in cost of goods sold combined with higher commercial product sales. Cost of goods sold decreased by $4.0 million, primarily attributable to a $2.8 million amendment fee associated with our insulin supply agreement in 2019, $0.8 million of increased 2020 manufacturing activities which resulted in a greater amount of costs capitalized to inventory and $1.1 million in reduced 2020 manufacturing-related spending, partially offset by $0.5 million of 2020 inventory write-offs and $0.4 million in costs associated with higher commercial product sales. Non-GAAP gross margin for the nine months ended September 30, 2020 increased to 49% from 28% for the same period in 2019, primarily due to higher Afrezza revenue and lower cost of goods sold.

Selling, general and administrative expenses for the nine months ended September 30, 2020 were $41.9 million compared to $58.9 million for the same period in 2019. This 29% decrease was primarily due to $9.3 million spent on direct-to-consumer television advertising in 2019 (which was not repeated in 2020), a $4.1 million decrease in promotional and marketing activities, a $1.3 million decrease in consulting costs, and a $1.2 million decrease in personnel and employee related costs.

The loss on foreign currency translation for the nine months ended September 30, 2020 was $4.0 million compared to a $4.5 million gain for the same period in 2019. The fluctuation is related to the change in the U.S. dollar to Euro foreign exchange rate on the recognized loss on insulin purchase commitments, which are denominated in Euros.

The net loss for the nine months ended September 30, 2020 was $30.8 million, or $0.14 per share, compared to a $37.6 million net loss, or $0.20 per share for the same period in 2019. The lower net loss was mainly attributable to a decrease selling, general and administrative expenses, partially offset by the change in the foreign currency translation from a gain in 2019 to a loss in 2020. The reduction in the net loss per share was also impacted by a greater number of outstanding shares.

Cash, cash equivalents and restricted cash at September 30, 2020 was $52.7 million compared to $50.2 million at December 31, 2019, which also included short-term investments of $20.0 million. The increase was primarily due to a milestone payment from United Therapeutics of $12.5 million, $15.2 million of net proceeds received from at-the-market offerings, $11.6 million received from warrant exercises and the origination of a Paycheck Protection Program loan for $4.9 million, offset by non-GAAP net cash used in operating activities of $40.9 million.

Non-GAAP Measures

Certain financial information contained in this press release is presented on both a reported basis (GAAP) and a non-GAAP basis. Reported results were prepared in accordance with GAAP whereas non-GAAP measures exclude items described in the reconciliation tables below. Non-GAAP financial information is intended to portray the results of our baseline performance, supplement or enhance management, analysts and investors overall understanding of our underlying financial performance and facilitate comparisons among current and past periods. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP.

Spectrum Pharmaceuticals Reports Third Quarter 2020 Financial Results and Corporate Update

On November 4, 2020 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported financial results for the three-month period ended September 30, 2020 (Press release, Spectrum Pharmaceuticals, NOV 4, 2020, View Source [SID1234569853]).

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"The third quarter was marked by significant progress in our drug development programs and a strengthened financial position," said Joe Turgeon, President and CEO, Spectrum Pharmaceuticals. "Our team is preparing for the upcoming pre-NDA meeting with the FDA for poziotinib and actively working to obtain an approval for ROLONTIS as soon as possible."

Pipeline Updates

Poziotinib, an irreversible tyrosine kinase inhibitor targeting EGFR and HER2 mutations

Spectrum has a pre-NDA meeting with the FDA to review the positive results from Cohort 2 and the path forward for poziotinib registration for the treatment of patients with previously treated locally advanced or metastatic NSCLC with HER2 exon 20 insertion mutations.
Spectrum presented the results from Cohort 2 at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020 in September. This marked the first presentation to the medical and scientific community of the positive results from this registrational cohort from the ZENITH20 clinical trial.
Spectrum expects to provide poziotinib update, including dosing strategy and topline results from Cohort 3 of the ZENITH20 trial in first-line EGFR NSCLC patients, by year-end 2020.
ROLONTIS (eflapegrastim), a novel long-acting G-CSF

The FDA deferred its action on the BLA for ROLONTIS, due to an inability to inspect the Hanmi Bioplant in South Korea citing travel restrictions related to the COVID-19 pandemic.
Spectrum has confirmed with the FDA that the deferral is not a Complete Response Letter (CRL). The company is actively working to find a way to expedite the plant inspection.
Three-Month Period Ended September 30, 2020 (All numbers are from Continuing Operations and are approximate)

GAAP Results

Spectrum recorded a net loss of $48.5 million, or $0.37 loss per basic and diluted share, in the three-month period ended September 30, 2020, compared to a net loss of $26.6 million, or $0.24 loss per basic and diluted share, in the comparable period in 2019. Total research and development expenses were $24.5 million in the quarter, as compared to $17.2 million in the same period in 2019. Selling, general and administrative expenses were $15.1 million in the quarter, compared to $13.1 million in the same period in 2019.

The company ended the quarter with cash, cash equivalents, and marketable securities of $198.3 million.

Non-GAAP Results

Spectrum recorded a non-GAAP net loss of $35.2 million, or $0.27 loss per basic and diluted share, in the three-month period ended September 30, 2020, compared to a non-GAAP net loss of $24.5 million, or $0.22 per basic and diluted share, in the comparable period in 2019. Non-GAAP research and development expenses were $23.3 million, as compared to $16.1 million in the same period of 2019. Non-GAAP selling, general and administrative expenses were $12.3 million, as compared to $9.9 million in the same period in 2019.

Conference Call and Webcast

Spectrum’s management will host a webcast and conference call today, November 4, 2020, at 4:30 p.m. ET / 1:30 p.m. PT to discuss the financial results and provide a corporate update. The live call may be accessed by dialing (877) 837-3910 for domestic callers and (973) 796-5077 for international callers and entering the conference ID#: 1281757. A live webcast of the call will be available from the Investor Relations section of the company’s website at View Source and will be archived there shortly after the live event.

MacroGenics Announces Flotetuzumab and Tebotelimab Presentations at the ASH Annual Meeting 2020

On November 4, 2020 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported six clinical and preclinical abstracts related to acute myeloid leukemia (AML) and flotetuzumab, an investigational bispecific CD123 × CD3 DART molecule, and one abstract related to tebotelimab, an investigational bispecific PD-1 × LAG-3 DART molecule, to be presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, December 5-8, 2020 (Press release, MacroGenics, NOV 4, 2020, View Source [SID1234569852]).

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"We look forward to presenting clinical and biomarker results for flotetuzumab in patients with relapsed or refractory acute myeloid leukemia at the 2020 ASH (Free ASH Whitepaper) annual meeting," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "The first of our two oral presentations will address results of the role of immune senescence and exhaustion-related RNA profiles in predicting outcomes in AML. The second oral presentation will provide the results on the use of flotetuzumab as salvage therapy in AML patients who failed induction therapy or experienced an early relapse. Together with our four posters, these presentations add to the growing medical and scientific interest in the potential of flotetuzumab in AML. Finally, we are very pleased to present data from the cohort of diffuse large B-cell lymphoma (DLBCL) patients who were treated in the dose expansion study of tebotelimab."

Oral Presentations

An Immune Senescence and Exhaustion-related RNA Profile Predicts Clinical Outcomes in Acute Myeloid Leukemia
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics, and Molecular Markers in Diagnosis and Prognosis: Single Cell Profiling and Novel molecular Markers
Session Date: Saturday, December 5, 2020
Session Time: 7:30 AM – 9:00 AM
Presentation Time: 8:15 AM
Flotetuzumab as Salvage Therapy for Primary Induction Failure and Early Relapse Acute Myeloid Leukemia
Session Name: 613. Acute Myeloid Leukemia: Novel Therapies and Treatment Approaches
Session Date: Sunday, December 6, 2020
Session Time: 9:30 AM – 11:00 AM
Presentation Time: 9:45 AM

Poster Presentations

Prophylactic Ruxolitinib for Cytokine Release Syndrome (CRS) in Relapse/Refractory (R/R) AML Patients Treated with Flotetuzumab
Session Name: 613. Acute Myeloid Leukemia: Clinical Studies: Poster III
Date: Monday, December 7, 2020
Tp53 Abnormalities Correlate with Immune Infiltration and Associate with Response to Flotetuzumab Immunotherapy in Acute Myeloid Leukemia
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics, and Molecular Markers in Diagnosis and Prognosis: Poster II
Date: Sunday, December 6, 2020
Immune Senescence and Exhaustion Correlate with Response to Flotetuzumab, An Investigational CD123 × CD3 Bispecific DART Molecule, In Acute Myeloid Leukemia
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics, and Molecular Markers in Diagnosis and Prognosis: Poster III
Date: Monday, December 7, 2020
Flotetuzumab and other Cellular Immunotherapies Upregulate MHC Class II Expression on Acute Myeloid Leukemia Cells In Vitro and In Vivo
Session Name: 704. Immunotherapies: Poster III
Date: Monday, December 7, 2020
A Phase 1, Open-Label Study of MGD013, a Bispecific DART Molecule Binding PD-1 and LAG-3 in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma
Session Name: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster III
Date: Monday, December 7, 2020
The above abstracts were published today on the ASH (Free ASH Whitepaper) website at View Source

About Flotetuzumab

Flotetuzumab (also known as MGD006) is a clinical-stage bispecific, investigational DART molecule that recognizes both CD123 and CD3. CD123, the interleukin-3 receptor alpha chain, has been reported to be over-expressed on malignant cells in AML and other hematologic malignancies. The primary mechanism of action of flotetuzumab is believed to be its ability to redirect T lymphocytes to kill CD123-expressing cells. To achieve this, the DART molecule combines a portion of an antibody recognizing CD3, an activating molecule expressed by T cells, with an arm that recognizes CD123 on the target cells. MacroGenics is conducting a single-arm, registration-enabling clinical study to evaluate flotetuzumab in up to 200 patients with primary induction failure / early relapse (PIF/ER) AML, with complete remission (CR) and CR with partial hematological recovery (CRh) as the primary endpoint. The study will be conducted as a continuation of the ongoing Phase 1/2 study (NCT02152956). The FDA has granted orphan drug designation to flotetuzumab for the treatment of AML.

About Tebotelimab

Tebotelimab (also known as MGD013) is a clinical-stage bispecific, investigational DART molecule designed to independently or coordinately block PD-1 and LAG-3 checkpoint molecules to sustain or restore the function of exhausted T cells for the treatment of cancer. Data from the Phase 1 dose escalation study in cohorts of patients with advanced solid tumors at escalating flat doses of 1-1200 mg every two weeks were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program. A maximum tolerated dose was not identified. At the same meeting, it was reported that LAG-3 expression on immune effector cells was shown to be enhanced by margetuximab, MacroGenics’ investigational Fc-engineered monoclonal antibody targeting HER2. Given the early signal of activity and acceptable safety profile observed in an initial, small expansion cohort of patients, the Company is evaluating the combination of tebotelimab and margetuximab in patients with HER2-positive tumors. The Company believes that combining Fc-engineering and checkpoint blockade has the potential to engage both innate and adaptive immune responses against a broad range of tumors with varied tumor microenvironments. For more information about the study design, please visit ClinicalTrials.gov (NCT03219268).

NanOlogy Enrolls First Patient in Phase 2 Clinical Trial of NanoPac® for Intratumoral Treatment of Prostate Cancer

On November 4, 2020 NanOlogy LLC, a clinical-stage oncology company advancing intratumoral therapy for solid tumors,reported that has enrolled the first patient in a Phase 2 clinical trial of NanoPac (sterile nanoparticulate paclitaxel) for suspension via intratumoral injection for local prostate cancer (Press release, NanOlogy, NOV 4, 2020, View Source [SID1234569851]). The single arm trial is evaluating up to 3 monthly injections of the investigational drug in patients scheduled for prostatectomy approximately 90 days after the first injection. Patients will be followed for safety and tumor response. Immune effects will be evaluated via multiplex immunohistochemistry and flow cytometry.

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Craig G. Rogers, MD (Henry Ford Cancer Institute), a clinical investigator for the Phase 2 trial, commented: "NanoPac completed a first-in-human clinical safety trial in 2019. In this innovative second trial, we will be evaluating both safety and efficacy of multiple intratumoral injections of NanoPac in patients with prostate cancer. We will also be analyzing tissue and blood for immune response and what therapeutic potential NanoPac may offer patients suffering from local disease. This trial aligns with our vision of offering precision medicine approaches along with cutting edge clinical trials at Henry Ford Cancer Institute."

The multicenter Phase 2 trial expects to enroll up to 18 patients with localized prostate cancer (tumors classified as

This second clinical trial follows completion of a first-in-human phase 2a dose-rising trial (NCT03077659) at a single institution to establish the safety of a single focal injection of NanoPac into the tumor-bearing lobe of patients scheduled for prostatectomy. Selected findings from the first trial:

No drug related SAEs including no prostatitis reported
Highest dose up to 75mg of NanoPac well tolerated
Mean reductions in tumor volume, PSA-density, and percent adenocarcinoma in biopsy
Peak paclitaxel plasma concentration was well below toxicity threshold at all timepoints, while drug was detected in all prostatectomy tissue specimens following prostatectomy.
A summary of study findings submitted to FDA can be found under NCT03077659 in the clinicaltrials.gov database.

Prostate cancer affects about 3 million men in the USA with 191,930 new cases and 33,330 deaths estimated for 2020 by the American Cancer Society. Patients at higher risk for disease progression may face surgical removal of the prostate or radiation therapy. Unfortunately, these patients may suffer incontinence or impotence, which significantly decreases quality of life. If the cancer becomes metastatic, patients have an estimated 5-year survival rate of only 31%.

In addition to prostate cancer, NanOlogy clinical programs are advancing in other genitourinary cancers, as well as gastrointestinal, peritoneal, lung, and dermal cancers. Data from preclinical and clinical studies in a variety of solid tumors have shown persistent tumor kill, antitumoral immune response, and minimal local or systemic toxicity.

The NanOlogy large surface area particle therapeutic platform is based on a proprietary supercritical precipitation technology that converts taxane API crystals into stable particles of pure drug with disproportionate size to surface area ratio. The particles are covered by two composition of matter patents (US 9,814,685) and (10,507,195) both valid until 2036 in the US and pending globally, forming the foundation of an extensive intellectual property portfolio protecting the investigational drugs and technology.