On February 7, 2019 Myovant Sciences (NYSE: MYOV), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of women’s health and endocrine diseases, reported corporate updates and reported financial results for the third fiscal quarter ended December 31, 2018 (Press release, Myovant Sciences, FEB 7, 2019, View Source [SID1234533141]).

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"As we enter this new year, we look forward to our first Phase 3 data from our LIBERTY program in uterine fibroids. The recent approval by Takeda for Relumina (relugolix 40 mg tablets) for the treatment of uterine fibroids in Japan, further validates our belief in relugolix as a critical treatment for people with uterine fibroids in the U.S. and Europe," said Lynn Seely, M.D., President and Chief Executive Officer of Myovant Sciences. "We continue to be on track to deliver data from both our LIBERTY and HERO programs this year, however enrollment for our SPIRIT endometriosis studies has taken longer than anticipated. While we remain confident that enrollment will be completed in 2019, we now anticipate top-line data for SPIRIT will be available in the first quarter of next year."

Recent Business Highlights and Upcoming Milestones

Relugolix Phase 3 Clinical Programs

Completed patient enrollment in the Phase 3 LIBERTY 2 trial evaluating relugolix in combination with estradiol and a progestin in women with heavy menstrual bleeding associated with uterine fibroids.
Expect top-line data from LIBERTY 1 and LIBERTY 2 Phase 3 trials evaluating the safety and efficacy of relugolix in combination with estradiol and a progestin in women with heavy menstrual bleeding associated with uterine fibroids in Q2 and Q3 of 2019, respectively, and assuming positive data, submission of the New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in Q4 of 2019.
Expect top-line data from the HERO Phase 3 trial evaluating the safety and efficacy of relugolix in men with advanced prostate cancer in Q4 2019, and assuming positive data, submission of the NDA to the FDA in early 2020.
Expect completion of enrollment in both SPIRIT 1 and SPIRIT 2 Phase 3 trials evaluating the safety and efficacy of relugolix in women with pain associated with endometriosis this year, with top-line data expected in Q1 2020.
On January 8, 2019, Takeda Pharmaceutical Company Limited and ASKA Pharmaceutical Co., Ltd. announced that Takeda has obtained marketing authorization in Japan from the Ministry of Health, Labour and Welfare for Relumina Tablets 40 mg (generic name: relugolix) for the improvement of symptoms of uterine fibroids (heavy menstrual bleeding, lower abdominal pain, lower back pain, and anemia).
MVT-602 Clinical Program

Completed enrollment in the dose-finding pharmacokinetic/pharmacodynamic Phase 2a study of MVT-602, a kisspeptin-1 receptor agonist, in healthy women undergoing a controlled ovarian stimulation protocol. Top-line results are expected to be reported in the first half of 2019.
Corporate

Raised proceeds of $92.0 million pursuant to our existing financing arrangement with NovaQuest in late December 2018.
Third Fiscal Quarter 2018 Financial Summary

Research and development (R&D) expenses for the quarter ended December 31, 2018, were $58.4 million compared to $34.9 million for the comparable period in 2017. The increase for the quarter primarily reflects the progress of Myovant’s ongoing Phase 3 clinical trials of relugolix, which were initiated in 2017, as well as additional personnel-related expenses and MVT-602 clinical trial expenses.

General and administrative (G&A) expenses for the quarter ended December 31, 2018, were $10.7 million compared to $6.6 million for the comparable period in 2017. The increase for the quarter primarily reflects increases in personnel-related expenses, professional service fees, and other administrative expenses to support Myovant’s headcount growth and expanding operations.

Net interest expense for the quarter ended December 31, 2018, was $1.6 million compared to $0.9 million in the comparable prior year period. Net interest expense consists of interest expense related to financing agreements with NovaQuest and Hercules Capital, Inc., as well as the associated non-cash amortization of debt discount and issuance costs, partially offset by interest income earned on cash equivalents.

Net loss for the quarter ended December 31, 2018, was $70.6 million, compared to $41.8 million for the comparable period in 2017. On a per common share basis, net loss was $1.04 and $0.70 for the quarters ended December 31, 2018, and 2017, respectively. The increases in the net loss and net loss per common share for the quarter were driven primarily by the increase in costs outlined above.

Capital resources: Cash and cash equivalents totaled $183.0 million at December 31, 2018. An additional $40.6 million of capacity remains available under the "at-the-market" equity offering program that Myovant initiated in April 2018.

About Relugolix

Relugolix is an oral, once-daily, small molecule that acts as a gonadotropin-releasing hormone, or GnRH, receptor antagonist. More than 2,150 study participants have received treatment with relugolix in Phase 1, Phase 2 and Phase 3 clinical trials. In completed trials, relugolix was generally well tolerated and suppressed estrogen and progesterone levels in women and testosterone levels in men. Common side effects observed were consistent with suppression of these hormones.

In the ongoing Phase 3 LIBERTY clinical trials in women with heavy menstrual bleeding associated with uterine fibroids and the ongoing Phase 3 SPIRIT clinical trials in women with pain associated with endometriosis, relugolix is undergoing evaluation in combination with estradiol and norethindrone acetate, a progestin, and as monotherapy. Myovant is studying whether the combination optimizes estradiol levels to the range required to treat the signs and symptoms of endometriosis and uterine fibroids while minimizing the side effects associated with low estrogen levels, which include bone mineral density loss and hot flashes. The ongoing Phase 3 HERO study is evaluating relugolix monotherapy in men with advanced prostate cancer.

About MVT-602

MVT-602 is an oligopeptide kisspeptin-1 receptor agonist. Kisspeptin, the ligand, is a naturally-occurring peptide that stimulates GnRH release and is required for puberty and maintenance of normal reproductive function, including production of sperm, follicular maturation and ovulation, and production of estrogen and progesterone in women and testosterone in men. A Phase 2a clinical trial in healthy female volunteers to characterize the dose-response curve in the controlled ovarian stimulation setting has completed enrollment.

On February 7, 2019 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of autoimmune diseases, asthma and allergic diseases and cancer, reported that company management will participate in fireside chats at two upcoming conferences (Press release, Xencor, FEB 7, 2019, View Source [SID1234533180]):

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Guggenheim Healthcare Talks Forum | Oncology Day
Date: Thursday, February 14, 2019
Location: New York, NY
Presentation Time: 1:30 p.m. ET

8th Annual SVB Leerink Global Healthcare Conference
Date: Thursday, February 28, 2019
Location: New York, NY
Presentation Time: 11:00 a.m. ET

Live webcasts of both events will be available under "Events & Presentations" in the Investors section of the Company’s website located at View Source A replay of the events will be posted on the Xencor website approximately one hour after the live events and will be available for 90 days following the events.

On February 7, 2019 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported that Michael Taylor, Ph.D., President and Chief Executive Officer, will present at the 1st Annual Guggenheim Healthcare Talks Idea Forum Oncology Day on Thursday, February 14 at 3:30 PM ET at the St. Regis Hotel in New York (Press release, Deciphera Pharmaceuticals, FEB 7, 2019, View Source [SID1234533108]).

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A live webcast of the event will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following the presentation.

On February 7, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA) is scheduled to review data supporting the Company’s New Drug Application (NDA) requesting accelerated approval for selinexor, a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound, at a meeting on February 26, 2019 at 12:30 p.m. ET (Press release, Karyopharm, FEB 7, 2019, View Source [SID1234533125]). The proposed indication to be discussed at this upcoming ODAC meeting is for selinexor in combination with dexamethasone for the treatment of patients with refractory multiple myeloma who have received at least three prior therapies and whose disease is refractory to at least one proteasome inhibitor (PI), one immunomodulatory agent (IMiD), and one anti-CD38 monoclonal antibody, and to their most recent treatment regimen.

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The ODAC is an independent panel of experts that evaluates data concerning the efficacy and safety of marketed and investigational products for use in the treatment of cancer and makes appropriate recommendations to the FDA. Although the FDA will consider the recommendation of the panel, the final decision regarding the approval of the product is made by the FDA solely, and the recommendations by the panel are non-binding.

Karyopharm’s NDA seeking accelerated approval for oral selinexor in combination with dexamethasone as a treatment for patients with triple class refractory multiple myeloma who have received at least three prior therapies is under Priority Review by FDA with an action date of April 6, 2019, under the Prescription Drug User-Fee Act (PDUFA).

The Company has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for selinexor requesting conditional approval for the treatment of patients with relapsed or refractory multiple myeloma who have received at least three prior lines of therapy and whose disease is refractory to at least one PI, one IMiD, and one anti-CD38 monoclonal antibody, and to their most recent treatment regimen. The selinexor MAA has been granted accelerated assessment by the EMA’s Committee for Medicinal Products for Human Use.

About Selinexor

Selinexor is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. In 2018, Karyopharm reported positive data from the Phase 2b STORM study evaluating selinexor in combination with low-dose dexamethasone in patients with penta-refractory multiple myeloma. Selinexor has been granted Orphan Drug Designation in multiple myeloma and Fast Track designation for the patient population evaluated in the STORM study. Karyopharm’s New Drug Application (NDA) has been accepted for filing and granted Priority Review by the FDA, and oral selinexor is currently under review by the FDA as a possible new treatment for patients with penta-refractory multiple myeloma. The Company has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) with a request for conditional approval and was granted accelerated assessment. Selinexor is also being studied in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). In 2018, Karyopharm reported positive top-line results from the Phase 2b SADAL study evaluating selinexor in patients with relapsed or refractory DLBCL after at least two prior multi-agent therapies and who are ineligible for transplantation, including high dose chemotherapy with stem cell rescue. Selinexor has received Fast Track designation from the FDA for the patient population evaluated in the SADAL study. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade (bortezomib) and low-dose dexamethasone (BOSTON), as a potential backbone therapy in combination with approved therapies (STOMP), in liposarcoma (SEAL), and an investigator-sponsored study in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

On February 7, 2019 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for its second quarter ended December 31, 2018 (Press release, MEI Pharma, FEB 7, 2019, View Source [SID1234533142]).

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"MEI remains in a strong position to continue to build on the clinical data presented at ASCO (Free ASCO Whitepaper) 2018 and at ASH (Free ASH Whitepaper) 2018 across our pipeline of four clinical-stage oncology candidates – including two candidates in clinical studies that may support future submissions for potential marketing approval by FDA," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "In particular, ME-401 continues to attract increasing notice from physicians for its emerging clinical profile, and there’s been a heightened scientific focus on PI3K delta as a target with the potential to mechanistically match BTK inhibition in importance for targeting B-cell malignancies."

Upcoming Milestones and Recent Program Highlights

Upcoming Milestones

Dosing of the first patient in the global Phase 2 study to support accelerated approval of ME-401 in relapsed or refractory follicular lymphoma in the first calendar quarter of 2019.
Updates and presentations of clinical data from the ME-401 clinical development program, including at select 2019 medical/oncology meetings.
Initial clinical results from the ongoing Phase 1 study of voruciclib, including single agent dose ranging data and results from the combination with venetoclax in patients with B-cell malignancies and relapsed and refractory acute myeloid leukemia, by end of 2019.
Results from the investigator-initiated study of ME-344 in combination with bevacizumab (marketed as Avastin) in patients with breast cancer at a medical/oncology meeting mid-year.
Pracinostat results from the Phase 2 clinical trial evaluating patients with myelodysplastic syndrome, including response and 1-year survival, expected to be available by the end of 2019.
Clinical Development Highlights

In December 2018, the Company opened the first clinical trial sites in its Phase 2 study that will evaluate both a continuous (CS) and intermittent (IS) dosing schedule of ME-401 in patients with third-line follicular lymphoma. The Phase 2 study is intended to support MEI’s accelerated approval registration strategy, if successful.
In December 2018, at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, the Company presented data from three clinical programs:
Interim results presented from the ongoing Phase 1b study evaluating ME-401, a selective phosphatidylinositol 3-kinase ("PI3K") delta inhibitor, support the complementary potential of intermittent and continuous dosing schedules of ME-401 to optimize the clinical risk-benefit ratio in patients with relapsed/refractory follicular lymphoma. The data demonstrate that ME-401, as both a single agent and in combination with rituximab, continues to be associated with overall high objective response rates. In addition, low rates of Grade 3 immune-related adverse events (irAEs) were observed in patients on the intermittent dosing schedule while maintaining a high level of clinical response.
Interim results presented from the ongoing Phase 2 study evaluating pracinostat, a histone deacetylase inhibitor, in combination with azacitidine for the treatment of patients with IPSS-R high/very high-risk of Myelodysplastic Syndrome ("MDS") demonstrate a 9% discontinuation rate due to adverse events, a substantially lower rate than observed in an earlier study. Additionally, the data presented demonstrated an encouraging 36% complete response rate among patients receiving at least 6 cycles of treatment.
Preclinical data presented demonstrate that voruciclib, MEI’s orally available CDK9 inhibitor, synergistically induced apoptosis at clinically relevant concentrations when combined with venetoclax (marketed as Venclexta) in human derived acute myeloid leukemia ("AML") cells lines and patient samples. Voruciclib is currently being evaluated in a Phase 1b dose ranging study in patients with B-cell malignancies and AML.
In October 2018, MEI entered into a clinical collaboration to evaluate the safety and efficacy of MEI’s ME-401, an investigational PI3K delta inhibitor, in combination with BeiGene’s zanubrutinib, an investigational Bruton’s tyrosine kinase ("BTK") inhibitor, for the treatment of patients with B-cell malignancies.
Corporate Highlights

In November 2018, MEI entered into a license, development and commercialization agreement granting Kyowa Hakko Kirin ("KHK") exclusive rights to develop and commercialize ME-401 in Japan. Under the terms of the agreement, MEI received a $10.0 million upfront payment and is eligible to receive up to $87.5M in additional development and commercialization milestones, and royalties on sales.
Financial Highlights

As of December 31, 2018, MEI had $93.4 million in cash, cash equivalents and short-term investments, with no outstanding debt.
For the three months ending December 31, 2018, cash expenditures for operating activities were $7.2 million, compared to $4.7 million for 2017. For the six months ending December 31, 2018, cash expenditures for operating activities were $20.0 million, compared to $11.3 million for 2017. The increase in cash used for the six months ended December 31, 2018 primarily relates to changes in working capital associated with our clinical development programs, including start-up costs related to the ME-401 Phase 2 study.
Research and development expenses were $9.1 million for the quarter ended December 31, 2018, compared to $3.4 million for the same period in 2017. Research and development expenses primarily reflect increased costs associated with the development of ME-401.
General and administrative expenses were $3.8 million for the quarter ended December 31, 2018, compared to $2.4 million for the same period in 2017. The increase primarily relates to increased salary and share-based compensation associated with increased headcount, and increased professional services expenses.
The Company recognized revenues of $2.0 million for the quarter ended December 31, 2018, compared to $0.4 million for the same period in 2017. The increase is related to new license revenues from our agreement with KHK, and to higher levels of research and development activities performed pursuant to the Helsinn license agreement.
Net income for the quarter ended December 31, 2018, was $12.0 million, or $0.17 per share compared to a net loss of $6.1 million, or $0.16 per share for the same period in 2017. The Company had 71,131,486 shares of common stock outstanding as of December 31, 2018, compared with 37,052,361 shares as of December 31, 2017.
The adjusted net loss, excluding non-cash expenses related to changes in the fair value of the warrants issued in connection with the May 2018 financing (a non-GAAP measure) for the quarter ended December 31, 2018, was $11.4 million.