On February 13, 2020 Foundation Medicine, Inc. and Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that they have entered into an agreement with the National Cancer Center (NCC) for the use of FoundationOne Liquid, Foundation Medicine’s laboratory-developed liquid biopsy test, in the third stage of SCRUM-Japan, the largest cancer genomic screening consortium in Japan (Press release, Chugai, FEB 13, 2020, View Source [SID1234554251]). The multinational program provides genomic screening in collaboration with hospitals on a regional scale in Japan and other countries in Asia, and aims to accelerate the development of innovative biomarker-driven precision medicine cancer therapies.

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The third stage of SCRUM-Japan is structured in two programs – LC-SCRUM-Asia and MONSTAR-SCREEN. LC-SCRUM-Asia is investigating genomic changes with the aim of delivering precision medicine to lung cancer patients. MONSTAR-SCREEN is investigating genomic changes across all types of advanced solid tumors, expanding beyond gastrointestinal cancer which was the focus of the second stage.

"The SCRUM-Japan program is a model of how collaboration between industry and academia is making precision medicine a reality for people in need of new treatment approaches," said Brian Alexander, chief medical officer of Foundation Medicine. "Utilization of FoundationOne Liquid in this program underscores its value in informing potential therapy selection for advanced-stage cancer patients. We look forward to continuing to expand access to comprehensive genomic profiling through this collaboration."

"SCRUM-Japan is a groundbreaking program to find therapies for patients with advanced cancer. There is an increasing need for blood-based genomic testing in patients who cannot give tissue samples including those who are unable to undergo invasive tumor biopsy," said Dr. Minoru Watanabe, Vice President, head of Chugai’s Foundation Medicine Unit. "We believe that this collaboration with the NCC, which has led a genome screening in Japan will pave the way to realize true precision medicine across the country."

"With the aim of delivering optimal treatments to patients, SCRUM-Japan was started with a view to detect cancer genomic alterations. The important achievements we saw from the first two stages include registration of over 10,000 patients’ clinical and genomic data, and approval of five therapeutic drugs and six in vitro diagnostics products based on clinical studies conducted by utilizing the data," said Atsushi Ohtsu, M.D., Ph.D. Director of National Cancer Center Hospital East and Representative of SCRUM-Japan. "Cancers remain leading causes of deaths in Japan and lung cancer has been ranked as the first leading cause of death among all cancer types. By incorporating FoundationOne Liquid into LC-SCRUM-Asia and MONSTAR-SCREEN, we believe the third stage of SCRUM-Japan will further prove the benefit of comprehensive genomic profiling tests such as FoundationOne Liquid."

Lung and gastrointestinal cancers are among the leading causes of cancer-related deaths in Japan, accounting for over 72 percent of cancer deaths in 2018 according to the World Health Organization. Through this collaboration, Foundation Medicine and Chugai will provide FoundationOne Liquid to academic centers participating in LC-SCRUM-Asia and MONSTAR-SCREEN.

In April 2018, Foundation Medicine received Breakthrough Device Designation from the U.S. Food and Drug Administration (U.S. FDA) on a forthcoming version of Foundation Medicine’s liquid biopsy test, which is currently under U.S. FDA review. Chugai and Foundation Medicine are preparing for the regulatory filing of this version of the test in Japan with the intention that the product will be approved for use under the National Health Insurance coverage in Japan. The parties intend that both LC-SCRUM-Asia and MONSTAR-SCREEN will transition from the existing FoundationOne Liquid test to the forthcoming version of Foundation Medicine’s liquid biopsy test following its anticipated approval by the U.S. FDA and subject to the terms of the agreement.

About SCRUM-Japan
SCRUM-Japan is the largest cancer genomic screening consortium in Japan and aims to accelerate the development of innovative biomarker-driven precision medicine cancer therapies. Since its launch in 2015, more than 10,000 patients with advanced cancers have participated in SCRUM-Japan. The third stage of SCRUM-Japan started in June 2019, and includes two programs – LC-SCRUM-Asia and MONSTAR-SCREEN. LC-SCRUM-Asia is investigating genomic changes with the aim of delivering precision medicine to lung cancer patients. More than 200 hospitals in Japan and Taiwan have joined the program and its scope area is expanding across Asia. MONSTAR-SCREEN is investigating genomic changes across all types of advanced solid tumors including gastrointestinal cancer. 28 hospitals have registered in Japan, and it aims for patients with various types of cancer to participate in the program.

On February 13, 2020 Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products reported updated results from the combination arm of KEYNOTE-46 (Part B), the Company’s ongoing Phase 1/2 study investigating ADXS-PSA with KEYTRUDA (pembrolizumab) in patients with metastatic, castrate-resistant prostate cancer (mCRPC) at the ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium in San Francisco, California (Press release, Advaxis, FEB 13, 2020, View Source [SID1234554279]). The KEYNOTE-46 trial was conducted in conjunction with Merck (known as MSD outside the U.S. and Canada) and evaluated ADXS-PSA, one of Advaxis’ Listeria monocytogenes (Lm)-based immunotherapies, alone and in combination with KEYTRUDA, Merck’s anti-PD-1 therapy.

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"The presented survival data in patients with visceral metastases strengthens our confidence that ADXS-PSA in combination with KEYTRUDA has the potential to provide meaningful increases in median overall survival in patients with advanced, metastatic, castration-resistant prostate cancer," said Kenneth A. Berlin, President and Chief Executive Officer of Advaxis. "Importantly, these demonstrated impacts on survival have not been previously observed with immunotherapy in this advanced patient population leading us to actively assess next steps for the program with the hope of providing a much-needed new treatment for these patients with limited options."

Key findings presented by Mark N. Stein M.D., FACS, Associate Professor of Medical Oncology at Columbia University Medical Center and lead study investigator, titled, "KEYNOTE-046 (Part B): Effects of ADXS-PSA in combination with pembrolizumab on survival in metastatic, castration-resistant prostate cancer patients with or without prior exposure to docetaxel" include:

●Median overall survival (95% CI) of 33.7 months (15.4-NR) for patients treated with ADXS-PSA in combination with KEYTRUDA (n=37)
●Median overall survival (95% CI) of 16.0 months (6.4-34.6) for patients with prior docetaxel (n=20)
●Median overall survival (95% CI) of 16.4 months (4.0-NR) for patients with prior visceral metastasis (n=11; 10 of who had prior docetaxel)
72.4% (21/29) of evaluable patients showed stable disease
●38% of patients had PSA declines and 27% had >= 30% PSA decline from baseline
●The combination of ADXS-PSA and pembrolizumab appeared safe and tolerable in this heavily pretreated, unselected population of patients with MSI-High-negative mCRPC
●Treatment-related adverse events were mostly Grade 1-2, with no additive toxicity observed with combination therapy

Mark N. Stein M.D., FACS said, "These data are encouraging given the advanced nature of the patient population which includes those who have failed next generation hormonal agents and/or docetaxel, and now those with visceral metastasis." He added, "I am particularly enthusiastic to see increases in median overall survival to 16.4 months as compared to standard of care, which tends to be closer to 11 months in patients with measurable disease/visceral metastasis. This improvement, delivered with a generally safe and well-tolerated treatment regimen, warrants additional evaluation in larger studies and I look forward seeing the potential of a continued evaluation of ADXS-PSA in combination with KEYTRUDA."

KEYNOTE-046 was an open-label, multicenter, dose-determining safety and tolerability Phase 1/2 trial of 50 heavily pretreated patients conducted in two parts (Part A and Part B), with a Phase 2 expansion cohort. The objective of the study was to evaluate ADXS-PSA alone (Part A) and in combination with KEYTRUDA (Part B) for primary endpoints that include safety, tolerability and dosing. Secondary endpoints included anti-tumor activity, progression-free survival and overall survival, and exploratory endpoints that include associations between biomarkers of immunologic response (serum PSA) with clinical outcomes. Enrollment in the study has been completed and the database lock occurred on January 28, 2020. The majority of treatment-related adverse events consisted of transient and reversible Grade 1-2 chills/rigors, fever, hypotension, nausea and fatigue. The combination of ADXS-PSA and KEYTRUDA has appeared to be well-tolerated, to date, with no additive toxicity observed.

About KEYNOTE-046

KEYNOTE-046 (NCT02325557) was a Phase 1/2 open-label, multicenter, dose-determination and expansion trial that evaluates the safety, tolerability and preliminary clinical activity of ADXS-PSA as monotherapy (Part A; n=14 [13 treated]), and in combination with KEYTRUDA (Part B; n= 37) in heavily pretreated patients with progressive and refractory mCRPC.

On February 13, 2020 Trovagene, Inc. (Nasdaq: TROV), a clinical-stage, oncology therapeutics company developing onvansertib for the treatment of various cancers including prostate, colorectal, and leukemia, reported positive data from its ongoing Phase 2 trial of onvansertib in combination with Zytiga (abiraterone – Johnson & Johnson)/prednisone, all administered orally, for the treatment of patients with Zytiga-resistant metastatic castration-resistant prostate cancer (mCRPC) (Press release, Trovagene, FEB 13, 2020, View Source [SID1234554296]).

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The clinical data, featured in a poster presentation today at the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Genitourinary Cancers Symposium in San Francisco, further demonstrates the efficacy of onvansertib in patients who develop resistance to first-line treatment with Zytiga. Onvansertib addresses Zytiga resistance across known androgen receptor (AR) resistance mechanisms. In patients with AR alterations, 86% had decreases in PSA levels with the addition of onvansertib to daily Zytiga.

"Metastatic castration-resistant prostate cancer is the second leading cause of cancer-related death among men in the United States, and unfortunately many patients are in desperate need of new treatment options," said study principal investigator Dr. David Einstein, Genitourinary Oncology Program, Beth Israel Deaconess Medical Center. "These data show that adding onvansertib to abiraterone in metastatic castration-resistant prostate cancer patients with an early resistance to abiraterone validates pre-clinical studies and shows potential as a new therapeutic option."
"We are very encouraged by the significant decreases in circulating tumor cells (CTCs) with the addition of onvansertib, given that these changes in CTCs are an accepted surrogate prognostic factor for efficacy and survival," said Dr. Mark Erlander, Chief Scientific Officer at Trovagene. "In addition, observing efficacy in patients that have tumors exhibiting known mechanisms of resistance to ARS inhibitor, Zytiga, suggests that onvansertib’s activity could extend to overcoming resistance to other ARS inhibitors such as Xtandi and Erleada."
Key Presentation Highlights:
Efficacy

Overall, across both arms (A and B), a 63% (12 of 19) response (Stable Disease – SD and Partial Response – PR) was observed in patients evaluable for efficacy (completed 12 weeks of treatment with onvansertib + Zytiga (abiraterone)/prednisone); 6 patients have been on treatment for ≥7 months

Arm B (onvansertib dosed daily on days 1-5 in a 14-day cycle)

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80% (4 of 5) patients had SD at 12 weeks, with 3 patients achieving the efficacy endpoint (PSA stabilization) and 3 patients remain on treatment

60% (3 of 5) patients have or had progression-free survival of >7 months

Arm A (onvansertib dosed daily on days 1-5 in a 21-day cycle)

57% (8 of 14) patients had SD or PR at 12 weeks, with 5 patients achieving the efficacy endpoint (PSA stabilization) and 4 patients remain on treatment

21% (3 of 14) patients have or had progression-free survival; 2 patients remain on treatment for >1 year

Onvansertib-induced circulating tumor cell (CTC) decrease is associated with progression-free survival

CTC count, reported as favorable or unfavorable (<5 versus ≥5 CTC/7.5mL of blood, respectively) is a prognostic factor for survival in CRPC and the conversion from unfavorable to favorable is associated with improved survival
o
At baseline, 25 (78%) patients had unfavorable CTC count with median of 19 CTC/7.5mL

10 of the unfavorable patients were re-analyzed after 12 weeks of treatment

5 (50%) patients had a ≥80% CTC decrease, including 2 AR-V7+ patients

4 (40%) patients converted from unfavorable to favorable CTC level (<5 CTC/7.5mL)

3 (30%) patients had no detectable CTC

Median time on treatment for patients with decrease CTC (n=4) is 7 months to-date, with 4 patients remaining on treatment

Conversely, median time on treatment for patients with increase CTC (n=5) was 5 months, and none of these patients remain on treatment

Efficacy observed in patients with Zytiga-resistant androgen receptor (AR) alterations

AR mechanisms of resistance to abiraterone include the expression of the constitutively active AR splice variant AR-V7 and the AR gain-of function point mutation T878A

Among the 19 patients who completed the 12-week treatment (Arm A + B):

5 patients were AR-V7+ at baseline

2 patients had AR T878A mutations at baseline

Onvansertib showed efficacy in patients with AR alterations (N=7):

6 (86%) patients had a decrease in PSA levels with the addition of onvansertib

4 (57%) patients had SD or PR at 12 weeks with 3 (43%) patients achieving the primary efficacy endpoint

3 patients have or had progression-free survival of >7 months, 2 patients remain on treatment

Safety

Safety lead-in cohort was completed in Arm A at 24 mg/m2 and is ongoing in Arm B at 18 mg/m2

Most frequent G3/G4 AEs were expected, on-target, reversible hematological (anemia, neutropenia, thrombocytopenia and leukopenia), associated with the mechanism of action of onvansertib

Hematological AEs were reversible and effectively managed by dose delay, dose reduction and/or growth factor support

Grade 3 hypophosphatemia was reported in 3 patients, next cycle treatment was delayed for 2 patients to allow recover
Conclusions

Overall, across both arms (A and B), a 63% (12 of 19) response (SD + PR) was observed in patients evaluable for efficacy (completed 12 weeks of treatment); 6 patients have been on treatment for ≥7 months

Onvansertib induced profound CTC decreases in patients with unfavorable CTC count (>80% decrease in 5 of 10 patients tested); CTC decrease was associated with prolonged response to treatment and progression-free survival

6 of 7 patients with AR alterations (AR-V7+ or AR T878A) had an immediate decrease in PSA following onvansertib treatment; efficacy (SD+PR) was achieved in 57% (4 of 7) patients

In both arms (A and B) onvansertib in combination with abiraterone was safe and well-tolerated

A more continuous dosing schedule (Arm C – onvansertib 12 mg/m2 on days 1-14 of a 21-day cycle) is planned to evaluate safety and efficacy

Adding onvansertib to abiraterone in mCRPC patients resistant to abiraterone (rising PSA) validates pre-clinical studies and shows promise as a new therapeutic option
About the Phase 2 Trial of Onvansertib in Metastatic Castration-Resistant Prostate Cancer
The trial is a Phase 2 open-label study of onvansertib in combination with Zytiga (abiraterone acetate)/prednisone, all administered orally, in patients with metastatic castration-resistant prostate cancer (mCRPC), showing signs of disease progression demonstrated by two rising PSA values separated by at least one week, while on Zytiga (NCT03414034). The primary efficacy endpoint is the proportion of patients achieving disease control after 12 weeks of study treatment, as defined by lack of prostate specific antigen (PSA) progression in patients who are showing signs of early progressive disease (rise in PSA but minimally symptomatic or asymptomatic) while currently receiving abiraterone acetate and prednisone. The trial is being conducted by Beth Israel Deaconess Medical Center (BIDMC), Dana-Farber Cancer Institute (Dana-Farber), and Massachusetts General Hospital Cancer Center (MGH). David Einstein, MD, Genitourinary Oncology Program at BIDMC, is the principal investigator for the trial.
About Onvansertib

Onvansertib is a first-in-class, third-generation, oral and highly-selective adenosine triphosphate (ATP) competitive inhibitor of the serine/threonine polo-like-kinase 1 (PLK1) enzyme, which is over-expressed in multiple cancers including leukemias, lymphomas and solid tumors. Onvansertib targets the PLK1 isoform only (not PLK2 or PLK3), is orally administered and has a 24-hour half-life with only mild-to-moderate side effects reported. Trovagene believes that targeting only PLK1 and having a favorable safety and tolerability profile, along with an improved dose/scheduling regimen will significantly improve on the outcome observed in previous studies with a former panPLK inhibitor in AML.
Onvansertib has demonstrated synergy in preclinical studies with numerous chemotherapies and targeted therapeutics used to treat leukemias, lymphomas and solid tumor cancers, including irinotecan, FLT3 and HDAC inhibitors, taxanes and cytotoxins. Trovagene believes the combination of onvansertib with other compounds has the potential to improve clinical efficacy in acute myeloid leukemia (AML), metastatic castration-resistant prostate cancer (mCRPC), non-Hodgkin lymphoma (NHL), colorectal cancer and triple-negative breast cancer (TNBC), as well as other types of cancer.
Trovagene has three ongoing clinical trials of onvansertib: A Phase 2 trial of onvansertib in combination with Zytiga (abiraterone acetate)/prednisone in patients with mCRPC who are showing signs of early progressive disease (rise in PSA but minimally symptomatic or asymptomatic) while currently receiving Zytiga (NCT03414034); a Phase 1b/2 Study of onvansertib in combination with FOLFIRI and Avastin for second-line treatment in patients with mCRC with a KRAS mutation (NCT03829410) and a Phase 1b/2 clinical trial of onvansertib in combination with low-dose cytarabine or decitabine in patients with relapsed or refractory AML (NCT03303339). Onvansertib has been granted orphan drug designation by the FDA in the U.S. and by the EC in the European Union for the treatment of patients with AML.
Trovagene licensed onvansertib (also known as NMS-1286937 and PCM-075) from Nerviano Medical Sciences (NMS), the largest oncology-focused research and development company in Italy, and a leader in protein kinase drug development. NMS has an excellent track record of licensing innovative drugs to pharma/biotech companies, including Array (recently acquired by Pfizer), Ignyta (acquired by Roche) and Genentech.

On February 13, 2020 Riptide Bioscience, Inc., reported the publication in Science Translational Medicine of a seminal article establishing a novel immune checkpoint, potentially useful in the treatment of both cancer and fibrosis (Press release, Riptide Bioscience, FEB 13, 2020, View Source [SID1234554312]).

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"We’re now engaging with larger pharmaceutical companies to move this important program into clinic. I see a real opportunity, similar in many ways to the discovery of the first checkpoint inhibitors, to make a major impact on clinical practice."

The article is entitled "Mannose receptor (CD206) activation in tumor-associated macrophages enhances adaptive and immune antitumor immune responses." (View Source). Riptide originated the development program and collaborated with Tuskegee University’s Cancer Research Center and the National Institutes of Health under separate research agreements. First author, Dr. Jesse Jaynes, Professor of Integrative Biosciences at Tuskegee, was joined by 42 co-authors from the National Cancer Institute, the National Center for Advancing Translational Sciences, and Tuskegee in this publication.

The article documents extensive research to evaluate the efficacy and confirm the mechanism of Riptide drug candidate RP-182. RP-182 is the first of several engineered peptides emerging from a years-long effort to enhance the activity of naturally occurring host defense peptides (HDPs) with immunomodulatory properties.

HDPs have the capacity to directly kill invasive microbes by forming pores in their membranes. However, certain HDPs also activate the immune system to fight the invaders – an activity which Riptide scientists discovered could be turned against "non-self" cancer cells.

Jaynes commented, "We did an extensive search for the cellular target of these peptides. Surprisingly it turned out to be CD206, the canonical biomarker for M2-polarized tumor-associated macrophages (TAMs). That made these small proteins really promising as potential drugs, since CD206 is many times more highly expressed on M2 macrophages than on any other somatic cell type."

TAMs are the most common immune cells in the tumor microenvironment, often outnumbering cancer cells themselves in solid tumors. TAMs produce a tumor-promoting tissue environment. Riptide scientists demonstrated that engaging a specific set of amino acids in a particular domain of CD206, returned the macrophage population from M2-dominant to M1-dominant, restoring a tumor-inhibiting microenvironment.

Jaynes’ colleague, Dr. Clayton Yates, Director of Tuskegee’s Cancer Research Center, added, "CD206 is a perfect narrow target for an exquisitely specific therapy. Our team was the first to show that this receptor could serve as a ‘toggle switch’ between macrophage phenotypes. We think it’s accurate to consider it a novel immune checkpoint – a completely natural mechanism that can have a profound effect on tumor etiology, without significant toxicity."

Yates continued, "We demonstrated that both in vitro and in vivo, the RP class of peptides are able to repolarize the macrophage population, and that once repolarized, the macrophages return to phagocytic activity and begin consuming cancer cells. They also send downstream signals to the T-cell, NK-cell and B-cell populations to attack the tumor."

Riptide President Dr. George Martin commented, "The results in animal models of organ cancers have been spectacular. RP-182, administered in combination with gemcitabine, produced the best results ever shown in NCI’s KRAS-p16 pancreatic cancer model. Also, with its distinctive mechanism, it has been shown to powerfully complement both chemotherapy and checkpoint inhibitor immunotherapy."

Dr. Martin continued, "Riptide has built very solid intellectual property around this program, and following the promising early work with RP-182, has developed two other peptide candidates with even better efficacy and excellent pharmacokinetics. These have demonstrated striking results in preclinical models of colon, breast, prostate, and skin cancers."

Riptide Executive Vice President Dr. Henry Lopez added, "Also, since M2 macrophages are important in the progress of certain fibroses, including scleroderma and IPF, we anticipated – and have recently confirmed – potent efficacy in animal models of those diseases."

Dr. Martin concluded, "We’re now engaging with larger pharmaceutical companies to move this important program into clinic. I see a real opportunity, similar in many ways to the discovery of the first checkpoint inhibitors, to make a major impact on clinical practice."

Riptide Bioscience, Inc., with laboratories in Vallejo, California, maintains an intensive program of research into peptide-based therapeutics

On February 13, 2019 Medivir AB reported Year End Report January-December 2019 (Press release, Medivir, FEB 13, 2020, View Source [SID1234554328])

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Preclinical data showing that in addition to its direct effect on cancer cells, MIV-818 also modulates the anti-tumor immune response, presented at the AACR (Free AACR Whitepaper)-NCI-EORTC conference in Boston .
The ninth and final liver cancer patient was included in the phase Ia study with MIV-818. Based on safety and tolerability as well as pharmaco-kinetics and positive biomarker data, it was decided to initiate the phase Ib part of the study.
In December, an investigator-initiated phase II clinical trial of remetinostat was started in patients with squamous cell carcinoma. The study is conducted at the Stanford University School of Medicine in the United States.
The first patient was included in a phase I study evaluating the safety and tolerability of a combination of birinapant and radiation therapy in patients with recurrent Head and Neck Squamous Cell Carcinoma. The study is sponsored and funded as part of the National Cancer Institute’s Cancer Treatment Evaluation Program.
A futility analysis of the phase II combination study with birinapant and Keytruda in colorectal cancer patients was performed. Medivir decided to end the study since the results of the analysis indicated that the study’s goals were unlikely to be achieved.
The first milestone payment for the candidate drug MIV-701 in veterinary medicine was received in October.
Financial summary for the quarter

Net turnover amounted to SEK 1.4 (13.6) million.
The loss before interest, tax, depreciation and amortization (EBITDA) amounted to SEK -30.3 (-96.6) million. Basic and diluted earnings per share amounted to SEK -1.32 (-4.72) and SEK -1.32 (-4.72) respectively.
Cash flow from operating activities amounted to SEK -22.6 (-72.4) million.
Liquid assets and short-term investments at the end of the period amounted to SEK 134.6 (286.3) million.
January – December
Financial summary

Net turnover amounted to SEK 8.7 (23.9) million.
The loss before interest, tax, depreciation and amortization (EBITDA) amounted to SEK -118.9 (-326.5) million. Basic and diluted earnings per share amounted to SEK -5.08 (-14.62) and SEK -5.08 (-14.62) respectively.
Cash flow from operating activities amounted to SEK -148.5 (-320.5) million.
Liquid assets and short-term investments at the end of the period amounted to SEK 134.6 (286.3) million.
Significant events after the end of the period

The phase II study of MIV-711 in patients with osteoarthritis was published in Annals of Internal Medicine (DOI: 10.7326/M19-0675).
Conference call for investors, analysts and the media
The Year End Report January – December 2020 will be presented by Medivir’s President & CEO, Uli Hacksell.

Time: Thursday, February 13, 2020, at 14.00 (CET).

Phone numbers for participants from:
Sweden +46-8-505-583-52
Europe +44-33-3300-9268
US +1-833-5268-396

The conference call will also be streamed via a link on the website: www.medivir.com
The presentation will be available on Medivir’s website after completion of the conference.
ombined with other medications and exhibits synergistic anticancer activity in preclinical models.