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Myovant Sciences to Host Webcast and Conference Call at 8:30 a.m. Eastern Time Tuesday, November 19 to Discuss Results from Phase 3 Study Evaluating Once-Daily, Oral Relugolix in Men with Advanced Prostate Cancer

On November 18, 2019 Myovant Sciences (NYSE: MYOV), a healthcare company focused on developing innovative treatments for women’s health and prostate cancer, reported it will hold a webcast and conference call beginning at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time on Tuesday, November 19, 2019, to discuss results from the Phase 3 HERO study of once-daily, oral relugolix (120 mg) in men with advanced prostate cancer (Press release, Myovant Sciences, NOV 18, 2019, https://investors.myovant.com/news-releases/news-release-details/myovant-sciences-host-webcast-and-conference-call-830-am-1 [SID1234551475]).

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Webcast/Teleconference Details
To participate in the live conference call, please dial 1-800-532-3746 for domestic callers and +1-470-495-9166 for international callers. A live webcast of the conference call will also be available on the investor relations page of Myovant’s website at investors.myovant.com and will remain archived on Myovant’s website for at least 30 days.

Ascendis Pharma A/S Reports Third Quarter 2019 Financial Results

On November 18, 2019 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported financial results for the quarter ended September 30, 2019 (Press release, Ascendis Pharma, NOV 18, 2019, View Source [SID1234551454]).

"Our achievements this year reflect our ability to bring a product from idea stage all the way through clinic, as we near the finish line for TransCon hGH and approach our planned regulatory filings in the United States (U.S.) and Europe," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "Our recent progress in the TransCon hGH program, including both in Europe and China, also support our strategy to establish global reach. The power of the TransCon technology is enabling us to build a leading fully integrated patient-focused biopharma company, and advance towards our ‘Vision 3×3’ goals."

He continued, "One of our driving values at Ascendis is patient focus. In response to the recent recall of NATPARA in the U.S., we recently expanded our TransCon PTH Phase 2 PaTH Forward Trial with a protocol addendum to help expedite participation of some of those patients affected by the recall. We believe the expanded trial will provide meaningful clinical data to help patients, both naïve to PTH and previously treated with PTH, and further demonstrate the value of our unique approach to product development."

Corporate Highlights & Progress

Completed the last subject visit for the long-term clinical database for TransCon hGH, paving the way for the company’s planned Biologics License Application to the U.S. Food and Drug Administration in the first half of 2020 and the Marketing Authorisation Application to the European Medicines Agency in the second half of 2020. TransCon hGH is a long-acting prodrug of human growth hormone (hGH) in phase 3 development as a once-weekly therapy for pediatric growth hormone deficiency (GHD).
Completed the manufacturing of drug product Process Performance Qualification (PPQ) batches required to support the planned regulatory filings in 2020 for TransCon hGH. The company is now finalizing the associated analytics and preparing qualification reports.
Advanced the TransCon hGH program in Greater China with VISEN Pharmaceuticals, who initiated a phase 3 trial for TransCon hGH in pediatric GHD.
Received Orphan Designation from the European Commission for TransCon hGH, which is provided to therapies aimed at treating, preventing or diagnosing a disease that is life-threatening or chronically debilitating, affects no more than five in 10,000 persons in the European Union and which may provide significant additional benefit over existing therapies.
Announced a protocol addendum in the U.S. designed to expedite enrollment of subjects previously treated with NATPARA in the TransCon PTH PaTH Forward Trial, a global phase 2 trial evaluating the safety, tolerability and efficacy of TransCon PTH in adult subjects with hypoparathyroidism (HP). TransCon PTH is an investigational long-acting prodrug of PTH in development as a potential once-daily replacement therapy for HP, designed to provide physiologic levels of PTH for 24 hours a day, seven days a week. Under the addendum, patients previously treated with NATPARA in the U.S. will now have an expedited pathway to enroll in PaTH Forward. As a result, the company expects to exceed targeted enrollment of 40 subjects, and plans to report top-line data from the expanded trial in first quarter of 2020. The goal of PaTH Forward is to evaluate TransCon PTH control of serum and urinary calcium, and identify a titration regimen for complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements).
Initiated the ACcomplisH Trial, a global, phase 2, randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of TransCon CNP, a long-acting prodrug of C-type natriuretic peptide (CNP), at escalating doses in children with achondroplasia (ACH). TransCon CNP is designed to provide continuous exposure to CNP at therapeutic levels with once-weekly dosing.
Presented preclinical data for TransCon TLR7/8 Agonist, a product candidate in development for oncology, at the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting. TransCon TLR7/8 Agonist is prodrug of resiquimod, a small molecule with immune-activating and anti-tumor properties that is transiently conjugated to a hydrogel carrier via a TransCon linker. Administered as an intratumoral injection, TransCon TLR7/8 Agonist delivered sustained local release of resiquimod over weeks directly to the tumor site and demonstrated potent anti-tumor activity as a monotherapy, as well as in combination with interleukin-2 (IL-2).
Ended the third quarter of 2019 with cash and cash equivalents of €658.7 million.
Third Quarter 2019 Financial Results

For the third quarter, Ascendis Pharma reported a net loss of €25.1 million, or €0.53 per share (basic and diluted) compared to a net loss of €34.0 million, or €0.81 per share (basic and diluted) for the same period in 2018.

Revenue for the third quarter was €2.2 million compared to €20 thousand in the same quarter of 2018. The increase relates to our November 2018 strategic investment in VISEN Pharmaceuticals.

Research and development (R&D) costs for the third quarter were €46.3 million compared to €31.5 million during the same period in 2018. Higher R&D costs in 2019 reflect an increase in personnel-related costs to support development and manufacturing of TransCon hGH, TransCon PTH and TransCon CNP, increasing clinical trial costs for the PaTH Forward Trial and the ACcomplish Trial, as well as increased costs for other research programs, including oncology.

General and administrative expenses for the third quarter were €10.0 million compared to €6.8 million during the same period in 2018. The increase is primarily due to higher personnel-related costs and other increasing costs of expanding the company and preparing to become a commercial organization.

As of September 30, 2019, Ascendis Pharma had cash and cash equivalents of €658.7 million compared to €690.4 million as of June 30, 2019. As of September 30, 2019, Ascendis Pharma had 47,739,647 ordinary shares outstanding.

Conference Call and Webcast information

Ascendis Pharma will host a conference call and webcast today at 4:30 p.m. ET to discuss its third quarter 2019 financial results. Details include:

Date Monday, November 18, 2019
Time 4:30 p.m. ET
Dial In (U.S.) 844-290-3904
Dial In (International) 574-990-1036
Access Code 5897398
A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will also be available on this website shortly after conclusion of the event for 30 days.

Fosun to Pay up to $148 Million for China Rights to MimiVax Glioblastoma Treatment

On November 18, 2019 Shanghai Fosun Pharma reported a $148 million agreement to acquire China rights to SurVaxM, a novel immunotherapy aimed at glioblastoma, from MimiVax of Buffalo, New York (Press release, ChinaBio, NOV 18, 2019, View Source [SID1234551445]). SurVaxM, a novel peptide mimic vaccine, stimulates the immune system to kill tumor cells that contain survivin, a protein that helps cancer cells resist conventional treatments. In a US Phase II trial among glioblastoma patients, SurVaxM showed improved efficacy combined with an acceptable safety profile. MimiVax, which notes that survivin is present in many cancers, is also conducting a multiple myeloma trial.

Medivir Reports Last Patient Recruited to the Phase Ia Study of MIV-818 in Liver Cancer Patients and Initial Dose Determined for phase Ib

On November 18, 2019 Medivir AB (Nasdaq Stockholm: MVIR) reported that the ninth and final patient was recruited to the phase Ia study of MIV-818 in patients with advanced liver cancer (Press release, Medivir, NOV 18, 2019, View Source [SID1234551443]). Based on data from phase Ia, the initial dose of MIV-818 for the phase Ib study has been determined at 200 mg / week. The dosage is given as 40 mg / day for five days.

The phase I study with MIV-818, evaluating safety, tolerability and pharmacokinetics, consists of two parts: Phase Ia is an interpatient study while phase Ib has a classic 3 + 3 intrapatient dose-escalating design. A recommended dose for phase II will be based on the data in the phase Ib study. Medivir has previously reported that the intended liver-directed effect of MIV-818 could be verified already in the analysis of data from the first six patients. Efficacy data from the complete phase Ia study will be presented next year at a scientific conference.

"The initial data observed in the phase I study support our view that MIV-818 has the potential to provide a clear and much needed improvement in treatment for patients with liver cancer," said Dr. Uli Hacksell, CEO of Medivir. "MIV-8I8 is the most advanced drug candidate in a series of proprietary and wholly owned prodrugs that we intend to develop for the treatment of various cancer indications."

For further information, please contact:

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.

RhoVac Reports Interim Results on Immunological Response with RV001

On November 18, 2019 RhoVac AB ("RhoVac") reported interim results from ongoing immunological studies at University of Tübingen, Germany concluding that treatment with RV001 can activate CD4+ T-cells and also CD8+ T-cells.

In July 2019 RhoVac published results on the follow-up phase of the phase I/II clinical study and in relation to the immunological response it could be concluded that 18 patients showing significant treatment related immunological response at completion of treatment. All 18 patients still showed significant response at 3-, 6- and 9-months follow-up. At the 12-months follow-up, 17 out of the 18 responding patients still showed significant immunological response.

Ongoing immunological studies at the University of Tübingen now confirm that treatment with RV001 can activate CD4+ T-cells and also CD8+ T-cells. Confirmation that the drug candidate RV001 can activate CD4+ T cells is important for the clinical value of the treatment. This has been demonstrated in a number of scientific publications of which some are referenced below:

Doonan et al (2010), demonstrated that "activation of CD4+ T cells is required to stimulate and prolong CD8+ T cell activity, and for the development of long-lasting tumor specific memory T cells".
Quezeda et al (2010) concluded in their study that "results highlight the relevance of the CD4+ T cell compartment, and particularly of cytotoxic CD4+ T cells, in cancer immunotherapy, while providing evidence that T cell differentiation in vivo may afford advantages over current approaches using differentiation and expansion in vitro".
Haabeth et al (2014) also concluded that beside CD8+ T cells commonly known as being able to eliminate target cells, CD4+ T cells also have the ability to directly eliminate tumors via T Cell Receptor and peptide:MHC-class II interaction (the pathway linked to CD4+ T cells) and/or to eliminate tumor cells indirectly via the release of immunomodulatory molecules activating surrounding immune cells.
For these reasons the MHC class II tumor antigen presentation pathway (the pathway linked to CD4+ T cells) must be utilized for therapeutic cancer vaccines to have a high clinical value, and interim results strongly indicate that RV001 is well capable of this.

Comment from RhoVac’s CEO, Anders Månsson:

Confirmation of the immunological response concluded based on interim results is obviously important data for the ongoing development of the drug candidate RV001. I am looking forward to the continued collaboration with the team at University of Tübingen.