On April 21, 2020 Tarveda Therapeutics, Inc., a clinical stage biopharmaceutical company developing a new class of potent and selective precision oncology medicines, which it refers to as Pentarin miniature drug conjugates, for the treatment of patients with various solid tumor malignancies, reported that the company will present data from its first in human study of PEN-866 at the 2020 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting, occurring April 27-28, 2020 (Press release, Tarveda Therapeutics, APR 21, 2020, View Source [SID1234556471]).

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PEN-866 is the initial clinical program from Tarveda’s HSP90 binding miniature drug conjugate platform, which is designed to bind to activated Heat Shock Protein 90 (HSP90) to accumulate and release its potent topoisomerase 1 inhibitor (SN-38) payload in solid tumors.

Details of the poster presentation are as follows:

Title: Characterization of PEN-866, a Heat Shock Protein 90 (HSP90) binding conjugate of SN-38, in patient plasma and tumors from the first in human study
Date: Monday, April 27, 2020
Time: 9:00 AM – 6:00 PM ET
Location: AACR (Free AACR Whitepaper) Virtual Annual Meeting I accessible on the AACR (Free AACR Whitepaper) website at www.aacr.org

On April 21, 2020 Blue Earth Diagnostics Ltd, a Bracco company focused on molecular imaging diagnostics, reported expanded access to Axumin (fluciclovine (18F)) in Europe (Press release, Blue Earth Diagnostics, APR 21, 2020, View Source [SID1234556470]). The first commercial delivery of Axumin was made in Slovakia in April 2020. This was possible through the Blue Earth Diagnostics’ distribution partner in Slovakia, MGP, spol. s r. o. Axumin is a PET imaging agent approved in the European Union for use in men with suspected recurrence of prostate cancer. Axumin is commercially available in Belgium, Luxembourg, Italy, France, Norway, the Czech Republic, The Netherlands, Austria and, now, Slovakia, with further European countries set to follow soon.

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Prostate cancer is a leading cause of cancer death in men. While most primary prostate cancer can be successfully treated, recurrence occurs in up to one-third of patients. Recurrent disease is typically detected by a rise in PSA levels but often the location and extent of the disease cannot be detected by conventional imaging. Of those who suffer biochemical recurrence, approximately one-third develop metastatic prostate cancer. Axumin was developed to target the increased amino acid transport that occurs in many cancers, including prostate cancer. It is labelled with the radioisotope (18F), enabling it to be visualized in the body with PET imaging.

Dr. Jonathan Allis, Chief Executive Officer of Blue Earth Diagnostics said, "We are very pleased that Axumin is now available in Slovakia. Expansion of supply into this new European country demonstrates the productive relationships that we have with our manufacturing and distribution partners, and supports our mission to make Axumin commercially available to clinicians and their patients across Europe."

Karel Zeleny, Chief Executive Officer of MGP commented, "We are proud of our continuing partnership with Blue Earth Diagnostics, and are happy to be able to distribute Axumin to another new market, contributing to the management of patients with prostate cancer recurrence."

On April 21, 2020 Myovant Sciences (NYSE: MYOV), a healthcare company focused on redefining care for women’s health and prostate cancer, reported that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for once-daily, oral relugolix (120 mg) for the treatment of men with advanced prostate cancer (Press release, Myovant Sciences, APR 21, 2020, https://investors.myovant.com/news-releases/news-release-details/myovant-sciences-submits-new-drug-application-nda-fda-once-daily [SID1234556469]). Myovant also announced that it expects to submit its NDA for once-daily, oral relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) for women with heavy menstrual bleeding associated with uterine fibroids in May 2020.

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TWEET THIS: "The submission of our NDA for prostate cancer is a major step towards providing a one pill, once a day potential new treatment option for men with advanced prostate cancer," said Lynn Seely, M.D., chief executive officer of Myovant Sciences. "Based on the robust efficacy and safety data from the Phase 3 HERO study, we believe relugolix, if approved, could provide men an important oral alternative to leuprolide injections, the current standard of care."

The NDA submission is supported by positive results from the Phase 3 HERO study, a randomized pivotal study comparing relugolix versus leuprolide acetate. Relugolix met the primary efficacy endpoint with 96.7% of men achieving sustained testosterone suppression to castrate levels (< 50 ng/dL) through 48 weeks. Six key secondary endpoints demonstrated superiority to leuprolide acetate, including sustained testosterone suppression to castrate levels through 48 weeks, rapid suppression of testosterone at Day 4 and at Day 15, profound suppression of testosterone (< 20 ng/dL) at Day 15, rapid suppression of prostate-specific antigen (PSA) at Day 15, and suppression of follicle-stimulating hormone (FSH) at Week 24 (all p-values < 0.0001). The overall incidence of adverse events in the relugolix and leuprolide acetate groups was comparable (92.9% vs. 93.5%, respectively). Major adverse cardiovascular events were reported in 2.9% of men in the relugolix group versus 6.2% of men in the leuprolide acetate group. These events included non-fatal myocardial infarction, non-fatal stroke, and all-cause mortality.

"We made the decision to prioritize this NDA submission and potentially accelerate the availability of an oral treatment option for men with advanced prostate cancer," said Juan Camilo Arjona, M.D., chief medical officer of Myovant Sciences. "This is of particular importance in the current environment and for the foreseeable future due to COVID-19 and the need for men with advanced prostate cancer to go to a clinic to receive injections in person."

About the Phase 3 HERO Program in Advanced Prostate Cancer
Myovant’s Phase 3 clinical program for advanced prostate cancer consisted of a randomized, open-label, parallel-group, multinational clinical study designed to evaluate the safety and efficacy of relugolix in men with androgen-sensitive advanced prostate cancer who required at least one year of continuous androgen deprivation therapy. Men enrolled in the study were randomized 2:1 to receive a single loading dose of relugolix 360 mg followed by relugolix 120 mg once daily, or to treatment with leuprolide acetate 3-month depot injection, respectively.

Approximately 1,100 men are planned to be enrolled in this study, including approximately 430 men with metastatic prostate cancer to support the analysis of a secondary endpoint of castration resistance-free survival, data which are expected in the third quarter of 2020, and 90 Chinese men (enrolled in China and Taiwan) to support registration in China.

About Prostate Cancer
Prostate cancer is the second most prevalent form of cancer in men and the second leading cause of death due to cancer in men in the U.S. Cardiovascular mortality is the leading cause of death in men with prostate cancer and accounts for 34% of deaths in men with prostate cancer in the U.S. Approximately three million men in the U.S. are currently living with prostate cancer, and approximately 170,000 men are estimated to be newly diagnosed in 2019. Advanced prostate cancer is prostate cancer that has spread or come back after treatment and may include men with biochemical recurrence (rising PSA in the absence of metastatic disease on imaging), locally advanced disease, or metastatic disease. Treatment for advanced prostate cancer typically involves androgen deprivation therapy, which reduces testosterone to very low levels, commonly referred to as castrate levels. GnRH receptor agonists, such as leuprolide acetate, or slow-release injections are the current standard of care for androgen deprivation therapy. However, GnRH receptor agonists may be associated with mechanism-of-action limitations, including the potentially detrimental initial rise in testosterone levels that can exacerbate clinical symptoms, which is known as clinical or hormonal flare, and delayed testosterone recovery after the drug is discontinued.

About Relugolix
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist that reduces testicular testosterone production, a hormone known to stimulate the growth of prostate cancer, and ovarian estradiol production, a hormone known to stimulate the growth of uterine fibroids and endometriosis. Myovant is developing relugolix as a monotherapy tablet (120 mg once daily) for men with advanced prostate cancer. Myovant is also developing a relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) for women with uterine fibroids and for women with endometriosis.

On April 21, 2020 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported that the U.S. Patent and Trademark Office has issued a composition of matter patent for cosibelimab (formerly referred to as CK-301), Checkpoint’s high affinity, fully-human IgG1 monoclonal antibody that directly binds to programmed death ligand-1 ("PD-L1") and blocks the PD-L1 interaction with the programmed death receptor-1 ("PD-1") and B7.1 receptors. U.S. Patent No. 10,590,199 specifically covers the antibody, cosibelimab, or a fragment thereof, providing protection through at least May 2038, exclusive of any additional patent-term extensions that might become available (Press release, Checkpoint Therapeutics, APR 21, 2020, View Source [SID1234556468]).

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Cosibelimab is currently being studied in an ongoing, multicenter, registration-enabling Phase 1 clinical trial intended to support a potential Biologics License Application ("BLA") submission for the initial indication of metastatic cutaneous squamous cell carcinoma ("CSCC"). Earlier this year, Checkpoint announced that the U.S. Food and Drug Administration had confirmed the registration submission pathway for cosibelimab in CSCC based on the ongoing clinical trial, which is over one-third enrolled. Cosibelimab is potentially differentiated from currently marketed PD-1 and PD-L1 antibodies with a half-life that supports sustained >99% tumor target occupancy and the additional benefit of a functional Fc domain capable of inducing antibody-dependent cell-mediated cytotoxicity ("ADCC") for possible enhanced efficacy in certain tumor types.

"This important issued U.S. patent for cosibelimab affords broad, foundational composition of matter protection for our antibody," said James F. Oliviero, President and CEO of Checkpoint. "We intend to continue expanding and fortifying our intellectual property portfolio for cosibelimab in the U.S. and internationally as we advance toward the completion of our clinical development program in CSCC, which initial indication for cosibelimab offers a $1-2 billion potential market opportunity."

About Cutaneous Squamous Cell Carcinoma
Cutaneous squamous cell carcinoma ("CSCC") is the second most common human cancer in the United States, with an estimated annual incidence of 700,000 cases. While most cases are localized tumors amenable to curative resection, approximately 8% of patients will experience a local recurrence, 5% of patients will develop nodal metastases, and an estimated 2% of patients will die from their disease. Ten-year survival rates are less than 20% for patients with regional lymph-node involvement. For those patients who develop distant metastases, the median survival time is estimated to be less than two years. In addition to being a life-threatening disease, CSCC causes significant functional morbidities and cosmetic deformities based on tumors commonly arising in the head and neck region and invading blood vessels, nerves and vital organs such as the eye or ear.

About Cosibelimab
Cosibelimab (formerly referred to as CK-301) is a high affinity, fully-human monoclonal antibody of IgG1 subtype that directly binds to programmed death ligand-1 (PD-L1) and blocks the PD-L1 interaction with the programmed death receptor-1 (PD-1) and B7.1 receptors. PD-L1 is an immune-inhibitory checkpoint molecule expressed on epithelial and vascular endothelial cells, as well as by a number of immune cells, and is utilized by tumor cells as an immune escape mechanism. Cosibelimab’s primary mechanism of action is based on the inhibition of the interaction between PD-L1 and its receptors PD-1 and B7.1, which removes the suppressive effects of PD-L1 on anti-tumor CD8+ T-cells to restore the cytotoxic T cell response. Cosibelimab is potentially differentiated from the currently marketed PD-1 and PD-L1 antibodies with a half-life that supports sustained >99% target tumor occupancy and the additional benefit of a functional Fc domain capable of inducing antibody-dependent cell-mediated cytotoxicity ("ADCC") for potential enhanced efficacy in certain tumor types.

On April 21, 2020 4SC AG (4SC, FSE Prime Standard: VSC) reported the Q1 Announcement 2020, presenting all material developments up to 31 March 2020 and the Company’s current outlook (Press release, 4SC, APR 21, 2020, View Source [SID1234556467]). The full communication is available for download on 4SC’s website.

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Jason Loveridge, Ph.D., CEO of 4SC, commented: "Whereas the Company had made good progress on both its development programs in Q1, including the FDA accepting our IND for the MERKLIN 2 study, most recently our team has been totally engaged with minimizing the impact of the global coronavirus pandemic on our Company and our clinical studies. Reflecting the sector more broadly, 4SC is also facing a temporary suspension of new recruitment in all of its active clinical studies and our clinical team is working overtime to ensure the safety of patients and the quality of data in our ongoing studies. 4SC is well financed and can, to a certain degree, weather the inevitable delay to study completions, however it is currently difficult to predict when study recruitment will return to normal. At this very challenging time our focus is first and foremost to ensure that patients in our active clinical studies are safe and may continue to receive medication, and to prepare as best we can for when circumstances will normalise".

Key highlight in Q1 2020 and beyond

FDA accepted Investigational New Drug (IND) application for domatinostat in combination with avelumab (Bavencio) in the MERKLIN 2 study.
2020 Business outlook

Domatinostat

Publish updated data from the SENSITIZE study in melanoma patients refractory to checkpoint blockade
Complete Phase IIa part of EMERGE study in micro-satellite stable gastrointestinal cancer
Initiate (first patient enrolled) of the Phase IIb part of the EMERGE study
Initiate the MERKLIN 2 study in Merkel cell carcinoma patients who are refractory to checkpoint blockade
Recruitment of the DONIMI study of domatinostat in the neoadjuvant setting in melanoma
Resminostat

Advance recruitment in the pivotal RESMAIN study so as to see the 125 events required to unblind the study in 2021
Development of cash balance in Q1 2020 and financial forecast

As of 31 March 2020, 4SC holds cash balance/funds of €40.350 million as compared to €45.765 million as of 31 December 2019. The monthly use of cash from operations amounted to €1.677 million on average in the first quarter of 2020 (Q1 2019: €1.263 million) and was below the range of €2.2 million and €2.6 million forecast for 2020.

The increase in the monthly use of cash as compared to Q1 2019, and the decrease in cash balance/funds in the first quarter of 2020 as compared to the end of 2019, were both predominantly due to costs for the ongoing clinical studies RESMAIN and SENSITIZE and mainly a result of the expansion of clinical programs for domatinostat, especially for the preparation of the clinical activities for the MERKLIN 2 study.

The Management Board of 4SC believes that the funds should be sufficient to finance 4SC into the second half of 2021.