On June 5, 2016 Mirati Therapeutics, Inc. (NASDAQ: MRTX) reported an update on three current ongoing clinical programs in patients with non-small cell lung cancer (NSCLC) and other solid tumors (Filing, 8-K, Mirati, JUN 6, 2016, View Source [SID:1234513142]).

"We are making significant progress in the development of our pipeline as our ongoing clinical programs are showing meaningful results," said Charles M. Baum, M.D., Ph.D., president and CEO. "We have confirmed responses including sustained activity in our glesatinib program, and encouraging signs of early efficacy from sitravatinib. These data demonstrate our ability to advance our targeted oncology drug candidates through our differentiated development approach. Today we also announced the start of our mocetinostat combination trial, our first in immuno-oncology. We are excited about the potential to deliver meaningful data points on all three of our clinical programs in 2016 and into 2017."

Glesatinib Program Update

Glesatinib Phase 1b Trial

As of May 20, 2016, 28 patients with MET and AXL alterations were enrolled in the Phase 1b trial across multiple tumor types, including 22 NSCLC patients. A majority of these patients were heavily pretreated with multiple lines of prior therapy including radiation and/or surgery. Eleven of the NSCLC patients had genetic driver alterations comparable to the criteria in our ongoing Phase 2 trial, including two NSCLC patients with MET amplifications, eight NSCLC patients with MET exon14 deletion mutations, and one NSCLC patient with AXL amplification.

The results demonstrate tumor regression in the majority of patients including confirmed responses to glesatinib:

· 3 of 11 NSCLC patients had partial responses (PRs) confirmed per RECIST, including patients with a MET amplification, MET exon14 deletion mutation and AXL amplification
· Patients with confirmed responses were on study for 39 weeks, 23 weeks and 56 weeks, with the 23 and 56 week patients continuing on study
· Tumor regression was seen in 10 of the 11 patients, three of which had confirmed PRs

During the course of the Phase 1b trial, the majority of the NSCLC patients (nine of 11) experienced dose reductions and/or dose interruptions. These events may have resulted in decreased exposure levels needed to fully inhibit MET throughout the treatment cycle.

Of the nine patients whose doses were reduced during the trial, two had a second dose reduction; four patients had dose interruptions and three had multiple dose interruptions while on study. Of the patients no longer on trial, five discontinued due to disease progression, two were related to AEs (one incidence of nausea and vomiting and one diarrhea) and one patient withdrew consent.

The dose reductions and interruptions were due primarily to episodes of diarrhea, potentially associated with the original miglyol (an oil-based excipient similar to castor oil) formulation of glesatinib administered during the trial, which may have contributed to or exacerbated diarrhea.

A new formulation of glesatinib is being implemented in the ongoing Phase 2 trial to reduce dose reductions and interruptions and to optimize exposure levels throughout the treatment regimen. The new spray-dried dispersion (SDD) formulation of glesatinib was evaluated in a dose escalation arm of the Phase 1b study and a recommended Phase 2 dose of 750mg BID was established.

The patient with a MET exon14 deletion and confirmed PR has shown significant tumor regression as well as improved tolerability after moving to the new formulation at a dose of 500mg BID. Following two full cycles of treatment with the new formulation, the patient experienced a deepening PR, from 45% to 66%, and remains on study.

Data from the Phase 1b trial has shown the SDD formulation to have several advantages including: (i) fewer tablets per dose; (ii) better relative bioavailability than the original formulation supporting full target inhibition; (iii) improved tolerability; and (iv) manufacturing advantages.

"We believe that the combination of improved tolerability and bioavailability of the new formulation will allow patients to remain on the intended dose, extend the duration of treatment and possibly increase response rates," continued Baum. "Because development of the new formulation was already in process for integration into the Phase 2 trial for use commercially, the change has already been discussed with the FDA and is moving forward."

Glesatinib Phase 2 Trial

The Phase 2 trial for glesatinib continues in NSCLC patients with MET genetic alterations of interest who were previously treated with platinum-based chemotherapy and those who may also have had prior treatment with a checkpoint inhibitor. Enrollment is ongoing and the new formulation is being introduced this month. Patients who started on the original formulation will be transitioned to the new formulation. An interim update on response rates in patients from the Phase 2 study on the new formulation will be provided once a meaningful number of patients have been treated and are evaluable.

Patient screening continues to increase and 55 clinical sites are active globally, with approximately 130 sites planned in total. In addition to clinical screening at study sites, unique patient finding and outreach collaborations with Foundation Medicine and Guardant Health have identified more than 160 additional patients with MET amplification and exon14 deletion in the first three months. Our experience confirms the prevalence of these patient populations and the value of these collaborations, which expand our clinical reach beyond our dedicated trial sites.

The Company is exploring development of glesatinib for patients with AXL genetic alterations based upon the NSCLC AXL amplification patient who has now had a durable confirmed response to glesatinib for over a year and continues on trial.

Sitravatinib Program Update

Initial data from the Phase 1b trial of sitravatinib show early signs of clinical activity, including a confirmed PR in a Renal Cell Carcinoma (RCC) patient, as well as durable tumor regressions in multiple other tumor types, including NSCLC patients with RET mutations. The Phase 1b trial in sitravatinib continues to enroll patients with RET, CHR4q12, CBL, TRK and DDR genetic alterations in NSCLC and other solid tumors at sites in the U.S. and Korea.

A poster entitled, "A first in human Phase 1 study of receptor tyrosine kinase (RTK) inhibitor MGCD516 in patients with advanced solid tumors" presented at the 2016 ASCO (Free ASCO Whitepaper) Annual Meeting includes this initial clinical data, which further demonstrates the safety and tolerability of sitravatinib. The poster can be found at www.mirati.com.

The Phase 1b trial includes multiple cohorts to explore the safety and efficacy of sitravatinib in genetically selected patients with NSCLC, as well as cohorts in certain solid tumors where the profile of sitravatinib may provide clinical benefit. Based upon our experience to date, sitravatinib is generally well tolerated at the recommended Phase 2 dose of 150mg, administered once daily (QD).

An additional update on this trial is expected by the end of the year, when a greater number of patients have been enrolled.

Mocetinostat Program Update

The Company has initiated a trial for the combination study of mocetinostat, an HDAC (histone deacetylase) inhibitor, with the AstraZeneca/MedImmune anti-PD-L1 checkpoint inhibitor, durvalumab, in patients with NSCLC.

This trial is exploring the potential of mocetinostat to enhance the effectiveness of checkpoint inhibitors in NSCLC. The dual effect of Class I HDACs on tumor cells, as well as on immune cells, may enhance the effect of checkpoint inhibitors in all indications where checkpoint inhibitors have demonstrated efficacy.

The Company plans to provide an update on this Phase 2 trial as progress continues, with the potential to see initial signals of activity by early 2017.

About Glesatinib (MGCD265)

Glesatinib (MGCD265) is a tyrosine kinase inhibitor that potently and selectively targets tumors in patients with driver alterations in MET (mutations and gene amplification) and Axl (rearrangements and gene amplification) that occur in approximately 8% of patients with non-small cell lung cancer (NSCLC). Genetic alterations in these targets have been implicated as drivers of tumor growth and disease progression in NSCLC and other solid tumors. Glesatinib is being evaluated in a Phase 2 trial in NSCLC patients with MET genetic alterations to confirm and extend the data that supports the clinical benefit of glesatinib in patients with driver mutations in MET. Mirati retains worldwide rights to glesatinib.

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About Sitravatinib (MGCD516)

Sitravatinib (MGCD516) is being evaluated in a Phase 1b dose expansion cohort in selected patients with specific genetic alterations that are drivers of tumor growth, with an initial focus on NSCLC and in other solid tumors where sitravatinib may confer a benefit. Sitravatinib is a tyrosine kinase inhibitor with demonstrated potent inhibition of a closely related spectrum of tyrosine kinases, including RET, CBL, CHR4q12, DDR and Trk, which are key regulators of signaling pathways that lead to cell growth, survival and tumor progression. Mirati retains worldwide rights to sitravatinib.

About Mocetinostat (MGCD103)

Mocetinostat (MGCD103) is an orally-bioavailable, spectrum-selective Class I & IV HDAC inhibitor currently being studied in a Phase 2 trial as a combination therapy with durvalumab, targeting the programmed death ligand 1 (PD-L1) pathway, which has been implicated in advanced lung cancers. In preclinical models, mocetinostat with durvalumab demonstrated significant reduction in tumor volume compared to either agent alone.

About Durvalumab

Durvalumab is an investigational human monoclonal antibody directed against programmed death ligand-1 (PD-L1). PD-L1 expression enables tumors to evade detection from the immune system through binding to PD-1 on cytotoxic T lymphocytes. Durvalumab blocks PD-L1 interaction with both PD-1 and CD80 on T cells, countering the tumour’s immune- evading tactics. Durvalumab is being developed alongside other immunotherapies to activate the patient’s immune system to attack the cancer. Durvalumab is being investigated in an extensive clinical trial programme, as monotherapy or in combination with tremelimumab, in NSCLC, bladder, head and neck, gastric, pancreatic, HCC and blood cancers. In 2015, durvalumab received Fast Track Designation for the treatment of patients with PD-L1—positive metastatic SCCHN, and in 2016, durvalumab was granted Breakthrough Designation by the U.S. Food and Drug Administration as a potential treatment for metastatic urothelial bladder cancer.

On June 6, 2016 Peloton Therapeutics, Inc., a drug discovery and development company focused on advancing first-in-class, small molecule cancer therapies targeting unexploited molecular vulnerabilities, reported first-in-human Phase 1 clinical data on its lead investigational candidate, PT2385, at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago, IL (Press release, Peloton Therapeutics, JUN 6, 2016, View Source [SID:1234513120]). PT2385 is the first clinical stage antagonist of hypoxia inducible factor-2α (HIF-2α), a transcription factor implicated in the development and progression of kidney and other cancers.

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In an oral presentation titled "A Phase 1 Dose-Escalation Trial of PT2385, a first-in-class oral HIF-2α Inhibitor, in Patients with Advanced Clear Cell Renal Cell Carcinoma," it was shown that PT2385 was well tolerated with no dose-limiting toxicities and had pharmacologic activity with encouraging clinical efficacy.

Patients with advanced clear cell renal cell carcinoma (ccRCC) and at least one prior therapy with a VEGF inhibitor were treated with PT2385 to determine the recommended Phase 2 dose and evaluate safety, pharmacokinetics and pharmacodynamics.
Twenty-six patients were enrolled in dose escalation and received PT2385 from 100 to 1800 mg twice per day. Patients were heavily pretreated prior to study entry, with greater than 50 percent having four or more prior therapies. Exposure increased with doses up to the 800 mg cohort without a further increase from 800 to 1800 mg. No dose limiting toxicities were observed at any dose level. Circulating plasma levels of the HIF-2α transcriptional target erythropoietin (EPO) rapidly decreased with treatment with PT2385 and remained suppressed. Based on safety, pharmacokinetic and EPO pharmacodynamic data, 800 mg twice per day was selected as the recommended Phase 2 dose. An additional 25 patients were enrolled in an expansion cohort at the recommended Phase 2 dose.

Among the 51 patients in total, the most common all-grade adverse events (AEs) were anemia, peripheral edema and fatigue. No cardiovascular AEs were noted. To date, one patient had a complete response, three patients had a partial response, and 16 patients had stable disease for 16 or more weeks. Ten percent of patients remain in this ongoing clinical trial for at least one year.
"Research at our institution has shown than HIF-2α is strongly implicated in renal cell carcinoma. PT2385 is the first agent to target this transcription factor and is very well tolerated. Its efficacy in patients with advanced clear cell renal cell carcinoma is promising," said Toni Choueiri, M.D., from the Dana-Farber Cancer Institute and Harvard Medical School, the senior author of the study.
"We are encouraged by the initial efficacy results in this heavily pretreated population and the fact that PT2385 does not have the cardiovascular toxicities of most other kidney cancer treatments, most notably VEGFR tyrosine kinase inhibitors. In light of this favorable safety profile and preclinical evidence of synergistic activity of our HIF-2α antagonists in combination with immune checkpoint inhibitors shown at the recent AACR (Free AACR Whitepaper) conference, we have opened a clinical trial of PT2385 in combination with nivolumab (Opdivo). Additionally, we are planning clinical studies of PT2385 as monotherapy and with other combination regimens," said John Josey, Ph.D., Peloton’s Chief Executive Officer.

About PT2385
PT2385 is a first-in-class small molecule inhibitor of hypoxia-inducible factor-2α (HIF-2α), a transcription factor implicated in the development and progression of kidney cancer. It is currently being investigated in a Phase 1 clinical trial for the treatment of advanced or metastatic clear cell renal cell carcinoma (ccRCC). Loss of the von Hippel-Lindau tumor suppressor (VHL) is the key oncogenic event in up to 95% of patients with ccRCC. With the loss of the VHL protein (pVHL), the transcription factor HIF-2α accumulates and drives the unbalanced expression of numerous gene products. Preclinical data indicate that orally bioavailable PT2385 disrupts HIF-2α activity in ccRCC and thereby blocks the expression of multiple tumorigenic factors responsible for unrestrained cancer cell growth and proliferation, tumor angiogenesis, and suppression of anti-tumor immune responses characteristic of ccRCC.

About Renal Cell Cancer
The American Cancer Society estimates that more than 62,000 new cases of kidney cancer will be diagnosed and more than 14,000 people will die from this disease this year. The National Cancer Institute reports that the prognosis for any treated renal cell cancer patient with progressing, recurring, or relapsing disease is poor, regardless of cell type or stage.
About Peloton Therapeutics

On June 6, 2016 Aeterna Zentaris Inc. (NASDAQ: AEZS; TSX: AEZ) (the "Company") reported its commitment to LHRH-receptor targeted therapy and its expectation that the pivotal, phase 3 trial for Zoptrex (zoptarelin doxorubicin) in women with advanced, recurrent endometrial cancer, is expected to be completed in the third quarter of 2016 (Press release, AEterna Zentaris, JUN 6, 2016, View Source [SID:1234513119]). During the 2016 Annual Meeting of the American Society of Clinical Oncologists ("ASCO"), the Company also discussed its plans to develop Zoptrex for additional indications, based upon achieving a positive outcome in the current clinical program.

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Zoptrex (zoptarelin doxorubicin), a novel synthetic peptide carrier linked to doxorubicin as a New Chemical Entity (NCE), is the Company’s lead oncology compound. Zoptrex is currently in a fully-enrolled Phase 3 clinical trial in endometrial cancer. The Company expects to complete the Phase 3 clinical trial in the third quarter of 2016 and, if the results of the trial warrant doing so, to file a new drug application for Zoptrex in the first half of 2017.

Commenting on the significance of Zoptrex to oncologists and their patients, Dr. Richard Sachse, the Company’s Chief Scientific Officer, explained, "Zoptrex is the first targeted oncological therapy using a peptide as the targeting agent and, therefore, it represents potentially a new tool in the treatment of tumors that overexpress the LHRH receptor. The design of the compound allows for the specific binding and selective uptake of the cytotoxic conjugate by LHRH receptor-positive tumors, typically found in gynecological cancers, prostate cancer and some forms of breast cancer. Potential benefits of this targeted approach may include enhanced efficacy and a more favorable safety profile with lower incidence and severity of adverse events, as compared to doxorubicin. If Zoptrex is approved as a therapy for endometrial cancer, we intend to develop it for these additional indications. In addition, based on the results of Phase 2 studies, we believe that Zoptrex holds promise to prove its efficacy for the treatment of ovarian and prostate cancer."

During the ASCO (Free ASCO Whitepaper) Annual Meeting, the Company provided an update regarding its progress with Zoptrex during one-on-one meetings with oncological key opinion leaders. Dr. Sachse described his interactions with ASCO (Free ASCO Whitepaper) attendees as promising, stating as follows: "I’m pleased and gratified by the continuing expressions of support for our work on Zoptrex. The oncologists with whom we met were pleased to learn of our progress toward the completion of our Phase 3 clinical trial and seemed to be as excited as we are about the contribution that our new targeted oncology therapy could make to the treatment of one of the most severe forms of cancer."

On June 6, 2016 Aprea AB, a privately held, clinical-stage biopharmaceutical company developing novel anticancer therapies targeting the tumor suppressor protein p53, reported clinical data from the Phase Ib part of the ongoing PiSARRO Phase Ib/II trial in collaboration with the European Network for Translational Research in Ovarian Cancer (EUTROC) (Press release, Aprea, JUN 6, 2016, View Source [SID:1234513118]). Aprea’s PiSARRO trial is investigating the safety and efficacy of APR-246 in combination with carboplatin and pegylated liposomal doxorubicin (PLD) in patients with relapsed high-grade serous ovarian cancer. Results presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) showed:

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APR-246 can be combined with standard chemotherapy at relevant doses, allowing the highest tested dose to be selected for continuing the study in a randomized Phase II trial.
All 21 patients treated in the study that are evaluable according to RECIST criteria have stable disease or better. In addition, 15 out of 18 evaluable patients had a GCIG CA-125 (tumor antigen biomarker) response after three treatment cycles. Overall response rate (GCIG or RECIST) was 18/24 (75%).
APR-246 showed linear pharmacokinetics with no accumulation and low intra patient variability and no indication of interaction between APR-246 and chemotherapy, supporting the combination of APR-246 with carboplatin and doxorubicin at relevant doses.
The main related treatment-emergent Grade 3-4 adverse events have been neutropenia, anemia and vomiting. The most frequent treatment-emergent adverse events have been low-grade gastrointestinal (nausea/vomiting), central nervous system (dizziness) and hematological (neutropenia and thrombocytopenia) events. The hematological side effects can be attributed to the chemotherapy, although a contribution from the addition of APR-246 cannot be ruled out at this time. No new safety concerns have emerged in the study.
Prof. Charlie Gourley, Chair of Medical Oncology and Honorary Consultant in Medical Oncology at the University of Edinburgh, said of the results: "APR-246 is an extremely exciting new agent because it targets tumors with mutant forms of the p53 gene, which is the gene most frequently altered in human cancer. This study shows that APR-246 can be successfully combined with standard chemotherapy for ovarian cancer with minimal additional toxicity. The percentage of patients whose cancer responded to this treatment regime was encouraging and we look forward to validating these findings in a larger clinical trial."

Dr. Mikael von Euler, Chief Medical Officer of Aprea, said: "We are very pleased with the results of the Phase Ib trial and to be able to move this exciting drug forward into a randomized Phase II trial in the third quarter of this year. It is especially important that the patients who have more difficult-to-treat disease seem to get as much benefit as those with less aggressive disease. The current safety profile combined with the evidence of clinical activity suggests that APR-246 might become a very important drug for patients with ovarian cancer. Furthermore, the mechanism suggests that APR-246 might have relevance in other tumor types and we look forward to pursing those opportunities."

About p53 and APR-246

The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anticancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

APR-246 has been shown to reactivate mutant p53 protein – by reconverting mutant p53 into wild-type p53 conformation and function – and thereby induce programmed cell death in human cancer cells. APR-246 has demonstrated compelling pre-clinical antitumor activity in a wide variety of solid and hematological (blood) tumors, including ovarian cancer, small cell lung cancer, esophageal cancer and AML (acute myeloid leukemia), among others. Additionally, strong synergy has been seen with both traditional anticancer agents, such as chemotherapy, as well as newer mechanism-based anticancer drugs. In addition to pre-clinical testing, a Phase I clinical study has been completed, demonstrating a favorable safety profile and both biological and clinical responses in hematological tumors with mutations in the p53 gene. APR-246 is currently in a Phase Ib/II clinical trial in patients with high-grade serous ovarian cancer. The Phase Ib part has completed. In the Phase II clinical study, Aprea will enroll up to 400 ovarian cancer patients in Europe and the US. Patients will be randomized between carboplatin and pegylated liposomal doxorubicin with or without APR-246; the primary endpoint for the study is progression-free survival (PFS). The company is also expecting to begin additional clinical studies of APR-246 in other cancer indications.

On June 6, 2016 Takeda Pharmaceutical Company Limited (TSE: 4502) and Roivant Sciences Ltd. reported the formation of Myovant Sciences Ltd. ("Myovant"), a biopharmaceutical company focused on delivering innovative women’s health and prostate cancer solutions by efficiently advancing new medicines to market that have the potential to improve the lives of millions of patients (Press release, Takeda, JUN 6, 2016, View Source [SID:1234513109]). In addition, Lynn Seely, MD, an endocrinologist who led the development of XTANDI (enzalutamide) for the treatment of prostate cancer as the Chief Medical Officer of Medivation from 2005 to 2015, was named the President & Chief Executive Officer of Myovant Sciences, Inc.

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Myovant Partnership Summary
Takeda has granted Myovant an exclusive, worldwide license (excluding Japan and certain other Asian countries) to relugolix (TAK-385), a phase 3 drug candidate that has been evaluated in over 1,300 patients to date. Relugolix has successfully demonstrated significant clinical benefit and was generally well-tolerated across multiple, large, randomized phase 2 clinical trials in three different indications. Relugolix is being developed as an oral, once-daily, potential best-in-class gonadotropin-releasing hormone (GnRH) receptor antagonist for uterine fibroids, endometriosis and prostate cancer. Takeda will retain commercial rights for relugolix in Asian countries, including Japan, where Takeda is actively conducting two phase 3 registration studies for the treatment of uterine fibroids. Takeda has also granted Myovant an exclusive, worldwide license to RVT-602 (TAK-448), a novel, oligopeptide kisspeptin receptor agonist as a product candidate for the treatment of infertility in females. Financial terms of the partnership were not disclosed.

"With Takeda strengthening its focus around the core therapeutic areas of oncology, gastroenterology and central nervous system diseases, as well as establishing a strategy that embraces innovative partnerships, it is important that we seek alternatives to further develop and create value around promising assets that are either outside these areas of focus or where strategic partnership makes more sense for our business," said Andrew Plump, M.D., Ph.D., Chief Medical and Scientific Officer of Takeda. "The formation of Myovant Sciences represents such an innovative partnership arrangement to further advance relugolix by relying on Roivant’s and Myovant’s in-house development capabilities in the major markets where they are building deep expertise, while leveraging Takeda’s commercial presence in certain Asian territories."

"We are very pleased to partner with Takeda to meet the needs of patients suffering from hormone-driven diseases and disorders," said Vivek Ramaswamy, CEO of Roivant Sciences, Inc. "The creation of Myovant enables a ‘win-win’ outcome for both our partner and for patients by launching a company to address major unmet medical needs in women’s health, prostate cancer and beyond."

Appointment of Dr. Lynn Seely as President & Chief Executive Officer
Lynn Seely, MD, brings over 20 years of drug development and biopharmaceutical company leadership to her role as the President & Chief Executive Officer of Myovant Sciences, Inc. Most recently, Dr. Seely served as Chief Medical Officer of Medivation, Inc. from its early stages in March 2005 through October 2015. She served on the Executive Committee and led the development of XTANDI for the treatment of metastatic castration-resistant prostate cancer from IND-enabling studies through to NDA approval and post-approval clinical studies. Dr. Seely was responsible for building the clinical organization at Medivation, as well as the regulatory, quality, project management, medical affairs and biologics manufacturing functions. Dr. Seely currently serves on the board of directors of Blueprint Medicines Corporation, and she previously served as Vice President of Clinical Development at Anesiva, Inc. (formerly Corgentech) and at Cytyc Health Corporation. Dr. Seely received a medical degree from the University of Oklahoma College of Medicine and completed her residency in internal medicine at Yale-New Haven Hospital. After serving as Chief Resident in Internal Medicine at Yale University School of Medicine, she completed her basic science and clinical fellowship in endocrinology and metabolism at the University of California, San Diego.
"I look forward to delivering on Myovant’s mission to bring innovative new treatments to women suffering from diseases such as uterine fibroids and endometriosis and to men with prostate cancer," stated Dr. Lynn Seely, President & CEO of Myovant Sciences, Inc. "Relugolix and our partnership with Takeda represent an exceptional foundation on which to build this exciting new company."

About Relugolix
Relugolix is a once-daily, orally administered, potential best-in-class gonadotropin-releasing hormone (GnRH) receptor antagonist for the treatment of uterine fibroids and endometriosis, and a potential best- and first-in-class treatment for hormone-sensitive prostate cancer (HSPC). Relugolix has been evaluated in over 1,300 patients to date, and has successfully demonstrated significant clinical benefit and was generally well-tolerated across multiple, large, randomized phase 2 clinical trials in three different indications. Takeda is currently enrolling two phase 3 clinical trials of relugolix for registration in Japan for the treatment of uterine fibroids.

By inhibiting GnRH receptors in the pituitary gland, relugolix rapidly reduces circulating sex hormone levels leading to suppression of estrogen and testosterone. Suppression of these sex hormones improves the symptoms of women with uterine fibroids and endometriosis, and decreases prostate-specific antigen (PSA) levels in men with HSPC.

In a 216-patient phase 2 study for the treatment of uterine fibroids, women who received relugolix had a significant reduction in menorrhagia, or abnormally heavy bleeding during menstruation. In a 487-patient phase 2 study for the treatment of endometriosis, women who received relugolix had a significant reduction in both non-menstrual and menstrual pelvic pain. Based on these results, two phase 3 registration studies in women with uterine fibroids are underway in Japan (NCT02655237, NCT02655224). In two phase 2 studies in approximately 225 men with HSPC, which included control arms of either an injectable GnRH agonist (leuprolide acetate) or a GnRH antagonist (degarelix), respectively, oral once-daily relugolix suppressed serum testosterone to castrate levels and decreased PSA. The safety of relugolix across all phase 2 studies was generally well-tolerated, consistent with the mechanism of action.

The clinical experience to date, supported by an extensive preclinical development program, suggests that oral relugolix, administered once-daily, could be an important advancement in the treatment options available for women suffering from endometriosis and uterine fibroids. In addition, relugolix could provide men with HSPC an important new treatment option as the first oral GnRH receptor antagonist that offers an alternative to injectable therapies.

About RVT-602
RVT-602 (TAK-448) is a kisspeptin analog that acts to stimulate the physiologic release of GnRH and downstream hormones important in fertility such as luteinizing hormone. Recent evidence from trials performed in women undergoing In-vitro Fertilization (IVF) revealed that the native kisspeptin peptide has the potential to act as an alternative to human chorionic gonadotropin (hCG) or GnRH agonists in triggering egg maturation, an essential step in every IVF cycle.

About Myovant Sciences
Myovant Sciences is focused on innovative treatments for women’s health conditions and prostate cancer. The company’s lead program is relugolix, a phase 3 drug candidate for multiple indications, including uterine fibroids, endometriosis and prostate cancer. Myovant was formed through a strategic partnership between Roivant Sciences and Takeda. Additional information about Myovant Sciences is available through its website, www.myovant.com