On October 30, 2019 Medivir AB (Nasdaq Stockholm: MVIR) reported that a poster entitled "MIV-818 stimulates an anti-tumor immune response in vitro and enhances the effects of pembrolizumab" was presented at the AACR (Free AACR Whitepaper)-NCI-EORTC conference in Boston (Press release, Medivir, OCT 30, 2019, View Source [SID1234550082]). The data shows that MIV-818 exerts favorable immunomodulating effects in vitro by increasing immune cytokine levels in complex tumor microenvironment models, and the white blood cell mediated killing of cancer cells. These effects are further enhanced in combination with the anti-PD1 check-point inhibitor pembrolizumab.

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!

"It is exciting that MIV-818, in addition to directly targeting cancer cells, also modulates the anti-tumor immune-response. This opens the possibility for potential future combinations with immunotherapy" – says Dr Fredrik Öberg, CSO.

For further information, please contact:
Dr Uli Hacksell
CEO
Medivir AB
phone: +46(0)8-5468-3100.

About MIV-818

MIV-818 is a pro-drug designed to selectively treat liver cancers and to minimize side effects. It has the potential to become the first liver-targeted, orally administered drug to benefit patients with HCC and other forms of liver cancer. A phase I study in patients with liver cancer is ongoing.

About liver cancer

Liver cancer is the third leading cause of cancer-related deaths worldwide and hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver. Although existing therapies for advanced HCC are capable of extending the lives of patients, treatment benefits are low while death rates remain high. HCC is a very diverse disease with multiple cancer cell types and without specific mutations seen in other tumor types. This has contributed to the lack of success of molecularly targeted agents in HCC. The limited overall benefit, taken together with the poor overall prognosis for patients with intermediate and advanced HCC, results in a large unmet medical need.

On October 30, 2019 Diplomat Pharmacy, Inc. (NYSE: DPLO) reported that it will release its third-quarter 2019 operating results before market open Tuesday, Nov. 12 (Press release, Diplomat Speciality Pharmacy, OCT 30, 2019, View Source [SID1234550035]). A conference call and live webcast will be held at 8:30 a.m. ET.

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!

Shareholders and interested participants can listen to a live broadcast by calling 833.286.5805 (647.689.4450 for international callers) and entering participation code 4382088, about 15 minutes before the call. A live webcast of the conference call will be available on the investor relations section of Diplomat’s website at ir.diplomat.is. The site will host an audio recording and supplemental investor information for 90 days.

On October 30, 2019 Invivoscribe, Inc. reported that the European Commission (EC) has proceeded to authorize the drug Astellas XOSPATA (gilteritinib) as monotherapy for the treatment of adults affected by leukemia Recurrent or refractory acute myeloid (AML) presenting FLT3 mutations ( FLT3 mut +) detected with the LeukoStrat CDx FLT3 mutation analysis (Press release, Invivoscribe Technologies, OCT 30, 2019, View Source [SID1234550067]).

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 LeukoStrat test is presented as a service that comprises a range of determinations and is marketed through several subsidiaries of Invivoscribe: LabPMM LLC, of San Diego ( California , USA); LabPMM GmbH, of Martinsried (Germany), and LabPMM GK, of Kawasaki (Japan). Currently, lots of CDx FLT3 mutation analysis are also distributed in Europe, Japan, Switzerland and Australia and it is planned to do so, in the future, in the United States and China .

Invivoscribe developed the LeukoStrat CDx FLT3 , in collaboration with Astellas, as an accompanying diagnostic test that allows predicting the patient’s response to the drug XOSPATA (Gilteritinib fumarate), from Astellas, for AML. The authorization that has just been granted is based on the results of the ADMIRAL phase III trial, in which gilteritinib was studied in comparison to rescue chemotherapy in patients with relapsing or refractory FLT3 AML . Thus, patients treated with gilteritinib had a markedly greater overall survival (OS) than those who received rescue chemotherapy: the median of OS was, in the case of the first, 9.3 months, compared with 5.6 of the patients who were only given chemotherapy.

Such a milestone consolidates the position of the LeukoStrat CDx FLT3 mutation analysis as a reference method of general evaluation of the FLT3 gene in critically ill patients of AML, since this accompanying diagnostic test allows FLT3 mutations in the tyrosine kinase domain (DTC) to be identified. and that show internal tandem duplication (DIT). LeukoStrat is marketed internationally.

This authorization is complementary to the others that have already been granted by the health authorities to the analysis of the LeukoStrat CDx FLT3 mutation as an accompanying diagnostic method of XOSPATA (Gilteritinib fumarate) from Astellas; RYDAPT (midostaurin) from Novartis, and quizartinib hydrochloride from Daiichi Sankyo.

"The authorization of the European Commission constitutes a notable advance for patients with recurrent or refractory acute myeloid leukemia with a FLT3 mutation . In Invivoscribe we welcome the introduction of more therapeutic options that allow the patient to be extended and we would be delighted to partner with pharmaceutical laboratories interested in taking advantage of our Streamlined CDx program with a view to speeding up obtaining authorization for their drugs in different parts of the world, regardless of whether the treatments in question are aimed at hemopathies or solid tumors, "said Jeffrey Miller , director of Strategy and CEO of Invivoscribe.

About the LeukoStrat CDx FLT3 mutation analysis of Invivoscribe
The LeukoStrat CDx
FLT3 mutation analysis is an in vitro diagnostic test for PCR designed to detect mutations in D835 and I836 with internal tandem duplication (DIT) and in the tyrosine kinase domain (DTC) ) of the FLT3 gene , a detection that is performed on genomic DNA extracted from mononuclear cells taken from peripheral blood or bone marrow aspirates from patients who have been diagnosed with acute myelogenous leukemia.

The LeukoStrat CDx FLT3 mutation analysis is used as an auxiliary system for the evaluation of patients with AML for those considering the possibility of starting treatment with midostaurin (in the United States, Europe, Switzerland and Australia ),

The LeukoStrat CDx FLT3 mutation analysis is used as an auxiliary system for the evaluation of patients with AML for those considering the possibility of starting treatment with gilteritinib fumarate (in the United States, Europe and Japan).

The LeukoStrat CDx FLT3 mutation analysis is used as an auxiliary system for the evaluation of patients with AML for those considering the possibility of starting treatment with quizartinib hydrochloride (in Japan).

The international standardized test includes software that interprets the data and generates signal ratios of mutant and natural alleles corresponding to mutations with DIT and in the DTC. This widely validated analysis makes it possible to standardize the detection of genetic mutations in the FLT3 gene , which are among the most important oncogenic initiators of acute myeloid leukemia (AML).

On October 20, 2019 NexImmune, a clinical stage immunotherapy company developing novel T cell therapies, reported that it received Investigational New Drug (IND) clearance for the company’s first cellular therapy product (Press release, NexImmune, OCT 30, 2019, View Source [SID1234554876]). NEXI-001 is being developed for the treatment of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) patients with relapsed disease after an allogeneic hematopoietic cellular transplant (allo-HCT). Scott Carmer, NexImmune’s President and CEO, commented "we are excited to initiate clinical trials with NEXI-001, and to provide these patients with a promising new treatment option. NEXI-001 is a product that is meaningfully differentiated from other cellular therapies in that it contains multiple populations of antigen specific endogenous T cells with enhanced anti-tumor properties. Because of this, we are confident NEXI-001 could address key limitations observed with other cellular immunotherapies; specifically, tumor escape through single target down-regulation and tumor relapse due to diminished T cell persistence."

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 Phase 1/2 trial of NEXI-001 will begin enrolling patients at clinical sites across the United States, including Memorial Sloan Kettering, MD Anderson, The City of Hope, The Dana Farber Cancer Institute, and AdventHealth. The trial is a multi-center, dose-escalating, open-label, single-arm study evaluating the safety, tolerability and initial efficacy of adoptively-transferred donor-derived T-cells as a treatment for AML or MDS patients with relapsed disease after allo-HCT. The trial will evaluate two increasing dose levels, with three patients enrolled in each dose escalation cohort. After the initial safety evaluation phase, up to 20 patients will be enrolled in the dose expansion phase. All patients will be followed for at least one year.

Miguel Perales, MD, Deputy Chief of Bone Marrow Transplant Services at Memorial Sloan Kettering Cancer Center and the trial’s lead investigator, stated "for patients receiving T cell therapy after allo-HCT, the primary treatment goal is to separate the efficacy benefits of Graft-versus-Leukemia (GVL) from the toxicities associated with Graft-versus-Host Disease (GVHD). This requires the ability to deliver a cell therapy product that contains a significant proportion of leukemia antigen specific memory T cells, and few-to-no T cell subtypes with allo-reactive potential. We believe NEXI-001 represents a T cell product with potential to deliver against these key treatment goals. Our center is excited to participate in this clinical trial, and we are enthusiastic about the promise NEXI-001 represents for our post-transplant AML/MDS patients with relapsed disease."

Lee Greenberger, MD, Chief Scientific Officer at The Leukemia and Lymphoma Society, commented further, "patients who relapse after allo-HCT are left with a dismal prognosis and limited treatment options, and are in need of new treatments that offer significant benefit. LLS is proud to partner with NexImmune on the development of products like NEXI-001 through our Therapy Acceleration Program (TAP). We believe this unique technology has the potential to meaningfully benefit patients who are in urgent need of novel approaches to treating this disease."

About AML / MDS relapse after Allogeneic Hematopoietic Cellular Transplant
Among patients with Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS), allogeneic hematopoietic cellular transplantation (HCT) used as post-remission therapy for those with high-risk genetic profiles or as salvage therapy for those with disease refractory to chemotherapy offers the highest potential for long-term survival. However, relapse after allogenic HCT remains the major cause of treatment failure, is associated with an extremely poor prognosis, and represents a major therapeutic challenge. In this situation, no standard therapy is defined, but treatment options generally aim to reduce disease burden and to enforce a graft-versus-leukemia (GvL) effect. Commonly used treatment options for these patients are chemotherapy combined with cellular-based approaches, such as donor lymphocyte infusion (DLI)or second transplantation.

About NEXI-001
NEXI-001 is an endogenous cellular therapy and includes populations of primed antigen specific CD8+ cells directed at multiple leukemia relevant antigen targets. Generating T cells against multiple tumor targets minimizes the potential for tumor escape. In addition, NEXI-001 contains T cell subtypes that are predominantly memory phenotypes, with the majority being characterized as stem cell memory and central memory T cells. Importantly, each NEXI-001 product contains minimal populations of naïve T cells (average <5%). Minimizing the naïve T cell compartment reduces alloreactivity and Graft-versus-Host Disease (GvHD) in patients after an allo-HCT, while T cell products that contain high proportions of memory cells, particularly central memory and stem cell memory, have been associated with potent anti-tumor activity, long-term T cell persistence and durable anti-tumor response.

On October 30, 2019 United Therapeutics Corporation (Nasdaq: UTHR) reported its financial results for the quarter ended September 30, 2019 (Press release, United Therapeutics, OCT 30, 2019, View Source [SID1234550013]).

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!

"We are pleased to see continued growth during the quarter in the total number of U.S. patients treated with our prostacyclin product franchise, consisting of Remodulin, Tyvaso, and Orenitram," said Michael Benkowitz, President and Chief Operating Officer of United Therapeutics. "As we execute toward our commercial goals, we are also continuing to execute toward our clinical goals, which include the recent label update to indicate that Orenitram delays disease progression and continued progress toward near-term phase III clinical trial readouts for DISTINCT, the study of dinutuximab for small cell lung cancer, and INCREASE, the study of pulmonary hypertension associated with interstitial lung disease."

Revenues for the three months ended September 30, 2019 decreased by $11.2 million as compared to the same period in 2018, driven entirely by a decrease in Adcirca revenues following the onset of generic competition in 2018. Revenues for our other four commercial products grew by $33.1 million in the aggregate for the three months ended September 30, 2019. Additional details regarding each product are discussed below.

Remodulin net product sales increased by $14.7 million for the three months ended September 30, 2019, as compared to the same period in 2018. $32.7 million of the increase was due to an increase in total quantities sold, primarily to our international distributors, partially offset by a decrease of $13.9 million due to price reductions, primarily to our international distributors, and higher gross-to-net revenue deductions of $4.1 million. During the three months ended September 30, 2019, there was an increase in the number of U.S. patients being treated with Remodulin, as compared to the same period in 2018.

Tyvaso net product sales for the three months ended September 30, 2019 increased by $3.0 million, as compared to the same period in 2018, primarily due to a price increase implemented in January 2019.

Orenitram net product sales for the three months ended September 30, 2019 increased by $8.2 million as compared to the same period in 2018. This increase was primarily due to an increase in the number of patients being treated with Orenitram and a price increase implemented in January 2019, partially offset by higher gross-to-net revenue deductions.

Unituxin net product sales for the three months ended September 30, 2019 increased by $7.2 million as compared to the same period in 2018. This increase was due to an increase in the number of vials sold and a price increase implemented in April 2019.

Adcirca net product sales for the three months ended September 30, 2019 decreased by $44.3 million as compared to the same period in 2018. This decrease was primarily due to a decrease in bottles sold following the onset of generic competition for Adcirca beginning in August 2018.

Research and development expense, excluding share-based compensation. Research and development expense decreased by $9.8 million for the three months ended September 30, 2019, as compared to the same period in 2018. Research and development expense for the treatment of cardiopulmonary diseases decreased by $21.9 million for the three months ended September 30, 2019, as compared to the same period in 2018, due to: (1) a one-time $10.0 million payment under a research agreement with MannKind; and (2) an up-front payment of $10.0 million under our license agreement with Samumed, both of which occurred during the three months ended September 30, 2018. The decrease in cardiopulmonary research and development expense was partially offset by an $11.2 million increase in research and development expense for our organ manufacturing projects due to increased preclinical and clinical work on technologies designed to increase the supply of transplantable organs and tissues.

The decrease in share-based compensation expense of $27.5 million for the three months ended September 30, 2019, as compared to the same period in 2018, was primarily due to a $30.8 million decrease in STAP expense driven by a 2 percent increase in our stock price for the three months ended September 30, 2019, as compared to a 13 percent increase in our stock price for the same period in 2018; partially offset by: (1) a $1.8 million increase in stock option expense due to additional awards granted and outstanding in 2019; and (2) a $1.4 million increase in restricted stock unit expense due to additional awards granted and outstanding in 2019.

Other Expense, Net

The increase in other expense, net of $16.0 million for the three months ended September 30, 2019, as compared to the same period in 2018, was primarily due to the recognition of a net unrealized loss in publicly-traded equity securities. During the three months ended September 30, 2019, we recognized $13.0 million of net unrealized losses on these securities.

Impairment of Investment in a Privately-Held Company

During the quarter ended September 30, 2018, one of the privately-held companies in which we invested experienced an event triggering an impairment analysis to evaluate the recoverability of our investment. We determined that the fair value of our investment was lower than its carrying value, resulting in an impairment charge of $12.4 million.

Income Tax Expense

The income tax expense was $34.5 million for the three months ended September 30, 2019, as compared to $33.6 million for the same period in 2018. The income tax expense is based on an estimated effective tax rate (ETR) for the entire year. The estimated annual ETR is subject to adjustment in subsequent quarterly periods if components used to calculate the estimated annual ETR are updated or revised. Our actual ETR as of September 30, 2019 and September 30, 2018 was 33 percent and 21 percent, respectively. We recognized a loss before income taxes, and a corresponding income tax benefit, for the nine months ended September 30, 2019, as a result of the one-time $800.0 million payment to Arena Pharmaceuticals, Inc. in January 2019. As a result of this loss, our anticipated tax credits and foreign sales deduction, partially offset by non-deductible compensation expense, increased our tax benefit and resulting ETR for the nine months ended September 30, 2019, compared to the same period in 2018.

Non-GAAP Earnings

Non-GAAP earnings is defined as net income, adjusted for: (1) share-based compensation expense (including expenses relating to stock options, restricted stock units, share tracking awards and our employee stock purchase plan); (2) impairment of investment in privately-held company; (3) unrealized losses (gains) on equity securities; (4) asset impairment charges; (5) license-related fees; and (6) tax benefit on non-GAAP earnings adjustments.

Inducement Restricted Stock Units

On October 25, 2019, we granted a total of 2,604 restricted stock units under our 2019 Inducement Stock Incentive Plan to three newly hired employees. These restricted stock units vest in three equal installments on October 31, 2020, October 31, 2021 and October 31, 2022, assuming continued employment on such dates, and are subject to the standard terms and conditions we filed with the SEC as Exhibit 10.2 to our Current Report on Form 8-K on March 1, 2019. We are providing this information in accordance with Nasdaq Listing Rule 5635(c)(4).

Conference Call

We will host a half-hour teleconference on Wednesday, October 30, 2019, at 9:00 a.m. Eastern Time. The teleconference is accessible by dialing 1-877-351-5881, with international callers dialing 1-970-315-0533. A rebroadcast of the teleconference will be available for one week by dialing 1-855-859-2056, with international callers dialing 1-404-537-3406, and using access code: 7462119.

This teleconference will also be webcast and can be accessed via our website at View Source