On November 12, 2020 BioXcel Therapeutics, Inc. ("BTI" or the "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, reported its quarterly results for the third quarter ended September 30, 2020 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, NOV 12, 2020, View Source [SID1234570774]).

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"Building on the successful data readout from the Phase 3 SERENITY trials earlier this year, we have continued to make substantial progress with BXCL501’s development as a treatment for agitation across neuropsychiatric conditions," stated Vimal Mehta, Chief Executive Officer of BTI. "Recently, we completed our pre-NDA meeting with the FDA and have initiated rolling submission of the NDA. This is a key milestone for our neuroscience franchise, laying a strong foundation for multiple follow-on indications and a catalyst for the ongoing build of our commercial infrastructure. While the TRANQUILITY and RELEASE trials advance, we are preparing to initiate a Phase 2 trial for agitation associated with delirium. Together, we believe that these trials, along with our strong cash position, will help to support our long-term strategy of establishing BXCL501 as an innovative treatment for agitation regardless of the patient’s underlying diagnosis."

Dr. Mehta continued, "We are also making excellent strides advancing our immuno-oncology program, with two combination therapy trials progressing well. Earlier this week, we presented encouraging preliminary efficacy and safety data at SITC (Free SITC Whitepaper) from both the Phase 1b/2 trial of BXCL701 and KEYTRUDA for the treatment of advanced prostate cancer and the MD Anderson-led Phase 2 basket trial in advanced solid tumors. Across both trials, we have already seen promising signals of activity in numerous difficult-to-treat tumors."

Third Quarter 2020 and Recent Highlights

BXCL501-Neuroscience Program

BXCL501 is an investigational, proprietary, orally dissolving, sublingual thin film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist, designed for the treatment of agitation and opioid withdrawal symptoms. The Company believes BXCL501 may directly target a causal agitation mechanism.

Following the recently announced positive topline results from the Phase 3 SERENITY trials, last month, BTI completed a successful pre-NDA meeting with the FDA for BXCL501 for the acute treatment of agitation in patients with schizophrenia and bipolar disorders. The FDA agreed to a rolling review of the NDA and BTI has already submitted part of the application to the FDA, with plans to submit the complete application in the first quarter of 2021.
The Company initiated the third dose cohort (90 mcg) in the TRANQUILITY study, a Phase 1b/2 trial of BXCL501 for the acute treatment of agitation associated with dementia. Based on the findings, the Company expects to report topline results in the fourth quarter of 2020, or, if needed, proceed to an additional dose cohort.
The RELEASE study, a Phase 1b/2 trial of BXCL501 for the treatment of opioid withdrawal symptoms, is ongoing, with enrollment of the dose cohorts progressing well. The Company expects to report topline results from the study in the first quarter of 2021.
In October, BTI received FDA clearance of its Investigational New Drug ("IND") application for BXCL501 for the treatment of hospitalized patients with agitation associated with delirium, including COVID-19 patients. The Company plans to initiate a Phase 2 trial within the next several months.
BTI recently analyzed topline data from a cross-over study in healthy volunteers comparing the bioavailability of BXCL501 administered sublingually with the film placed drug-side down under the tongue, compared to the film placed drug-side up under the tongue, and administered buccally (between the lower lip and the gum). It was determined that all three administrations of BXCL501 are bioequivalent and were rapidly absorbed into the systemic circulation. Also, drinking water starting at 15 minutes following sublingual administration did not have any affect on bioavailability.
The Company was issued U.S. patent No. 10,792,24 on October 6, 2020, which covers film formulations containing Dex and methods of treating agitation using such film formulations. The patent is expected to extend intellectual property ("IP") protection until 2039.
BXCL701-Immuno-Oncology Program

BXCL701 is an orally-delivered small molecule, innate immunity activator designed to inhibit dipeptidyl peptidase (DPP) 8/9 and block immune evasion by targeting Fibroblast Activation Protein (FAP). It has shown single agent activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients.

The Phase 2 portion of the Phase 1b/2 trial of BXCL701 in combination with pembrolizumab (KEYTRUDA) for treatment emergent Neuroendocrine Prostate Cancer ("tNEPC") and castrate-resistant prostate cancer ("CRPC") is advancing. Data from the 1b portion of this trial was presented recently at SITC (Free SITC Whitepaper), with an additional efficacy update planned.
The MD Anderson-led Phase 2 open label basket trial evaluating the combination of BXCL701 and KEYTRUDA in patients with advanced solid tumors is progressing well, and has already moved to the stage-2 efficacy phase. Preliminary efficacy data on 14 patients (5 patients in the checkpoint naïve cohort and 9 patients in the checkpoint pretreated cohort) was presented earlier this week at SITC (Free SITC Whitepaper).
The BXCL701 phase of the triple combination study of BXCL701, bempegaldesleukin (NKTR-214, Nektar Therapeutics, Inc.) and BAVENCIO (avelumab, Merck KGaA, Darmstadt, Germany and Pfizer) in second line pancreatic cancer was planned to initiate following Nektar and Pfizer’s Phase 1B dose-escalation trial of bempegaldesleukin and avelumab, which was delayed. All parties have agreed to discontinue activities on the triple combination study and instead reallocate resources to other studies and development programs. The parties terminated their clinical trial collaboration agreement, effective November 10, 2020.
Corporate Highlight

In July 2020, the Company raised net proceeds of approximately $187 million in connection with its common stock offering. BTI believes that the proceeds from this offering, together with current reserves, provide the cash runway to fund key clinical, regulatory, operational, and commercial activities well into 2022.
Third Quarter 2020 Financial Results

BTI reported a net loss of $24.8 million for the third quarter of 2020 compared to a net loss of $9.0 million for the same period in 2019. The third quarter 2020 results include approximately $5.3 million in non-cash stock-based compensation compared to $0.8 million for the same period in 2019.

Research and development expenses were $16.3 million for the third quarter of 2020, compared to $7.1 million for the same period in 2019. The increase was primarily attributable to increased clinical trial costs and professional research related to the acceleration of the Company’s research and development activities, primarily related to its SERENITY I and II clinical trials as well increased costs associated with its TRANQUILITY and RELEASE clinical trials for BXCL501. These amounts were partially offset by reduced costs related to the BXCL701 pancreatic cancer trial.

Personnel costs also increased, primarily related to the growth of BTI’s clinical team as the Company continues to expand its clinical programs, and in preparation of the potential commercial launch of BXCL501 in the U.S. Non-cash stock-based compensation also increased as result of the additional personnel combined with increased grant date fair values arising from higher market prices of the Company’s common stock.

General and administrative expenses were $8.5 million for the third quarter of 2020, compared to $2.0 million for the same period in 2019. The increase was primarily due to increased non-cash stock-based compensation and personnel costs related to the growth of BTI’s operations combined with increased grant date fair values arising from higher market prices of the Company’s common stock. Professional fees also increased which is primarily attributable to increased corporate legal and investor relations fees combined with increased insurance premiums.

Total operating expenses for the third quarter of 2020 were approximately $24.8 million, compared to total operating expenses of approximately $9.1 million for the same period in 2019.

As of September 30, 2020, cash and cash equivalents totaled approximately $233.4 million.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call, please dial 877-407-2985 (domestic) and 201-378-4915 (international). A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The replay will be available through at least November 26, 2020.

On November 12, 2020 Agile Therapeutics, Inc. (Nasdaq: AGRX), a women’s healthcare company, reported financial results for the three and nine months ended September 30, 2020 and provided a corporate update (Press release, Applied Therapeutics, NOV 12, 2020, View Source [SID1234570770]).

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"With our commercial launch of Twirla expected by the end of the year, we remain on track to deliver on our plan. The final validation of our commercial manufacturing process continues to progress as all three of our batches are expected to be released for commercial use in December 2020. By taking steps to build out an experienced sales force, secure major wholesaler agreements and increase market access for Twirla, we believe we are well-prepared to hit the ground running. Having our first FDA-approved product nearing launch marks an exciting time for Agile and we look forward to bringing to the market another choice for women to help fulfill their contraceptive needs," said Al Altomari, Chairman and Chief Executive Officer of Agile.

Third Quarter 2020 and Other Recent Corporate Developments:

Twirla Commercialization Update

The Company intends to begin shipping product to wholesalers by year-end 2020.

Agile remains on track to finalize the validation of its commercial manufacturing process of Twirla.
• All three validation batches are expected to be released for commercial use in December 2020.

The Company continued to build its distribution network by entering into an agreement with a third major U.S. wholesaler.

The Company continues to build awareness among prescribers and health care providers to gain market access for Twirla.
• Product sampling expected to begin at launch.

Through Syneos Selling Solutions, the Company’s contract sales force partner, Agile completed the hiring of an initial sales team of 73 persons.
• The sales force initiated discussions with healthcare providers after it was fully deployed in mid-October.
Strengthened Executive Leadership Team

In August 2020, the Company appointed Paul Korner, MD, MBA, as Chief Medical Officer. Dr. Korner is a board-certified obstetrician and gynecologist with more than 20 years of pharmaceutical and biotech industry experience, including significant experience within women’s healthcare.
Launched I’m So Done – A National Unbranded Campaign

In September 2020, the Company launched I’m So Done, an education and empowerment platform that encourages women to think about their current contraceptive method and decision-making journey.
Financial Guidance

The Company narrowed its operating expense guidance for the full year 2020 to be in the range of $52 million to $54 million, with general and administrative expenses accounting for approximately 70% of the spending as it builds out its commercial infrastructure. The Company’s operating expenses guidance includes $2.7 million to $3 million of non-cash stock compensation expense. The Company expects its gross revenue in the fourth quarter of 2020, reflecting expectations of initial stocking of Twirla by wholesalers, to be approximately $1 million.

Based on the Company’s current business plan and pending launch of Twirla, the Company believes that its cash, cash equivalents and marketable securities as of September 30, 2020 will be sufficient to meet its projected operating requirements through the end of 2021. If the COVID-19 pandemic or other factors impact the Company’s current business plans or its ability to generate revenue from the launch of Twirla, the Company believes it has the ability to revise its commercial plans, including curtailing sales and marketing spending, to allow it to continue to fund its operations.
Third Quarter Financial Results

Cash, cash equivalents and marketable securities: As of September 30, 2020, Agile had $71.9 million of cash, cash equivalents and marketable securities compared to $34.5 million of cash and cash equivalents as of December 31, 2019.

Research and development (R&D) expenses: R&D expenses were $3.7 million for the quarter ended September 30, 2020, compared to $2.4 million for the comparable period in 2019. The increase in R&D expenses was primarily due to costs to conduct validation work for commercial manufacturing of Twirla by Corium, the Company’s contract manufacturer.

General and administrative (G&A) expenses: G&A expenses were $11.0 million for the quarter ended September 30, 2020, compared to $2.1 million for the comparable period in 2019. The increase in G&A expenses was primarily due to higher costs associated with the Company’s pre-commercialization activities for Twirla, such as brand building, advocacy, market research and consulting. The increase in G&A expenses was also attributable to activities related to building out the commercial organization and included higher salaries and higher professional fees related to recruiting fees and consultants, and an increase in stock compensation expense.

Net loss: Net loss was $15.5 million, or $0.18 per share, for the quarter ended September 30, 2020, compared to a net loss of $4.4 million, or $0.08 per share, for the comparable period in 2019.

Shares Outstanding: As of September 30, 2020, Agile had 87,434,604 shares of common stock outstanding.
Conference Call and Webcast
Agile Therapeutics will host a conference call and webcast to discuss financial results for the third quarter ended September 30, 2020 today at 4:30pm ET. Investors interested in listening to the conference call may do so by dialing (877) 407-2991 for domestic callers or (201) 389-0925 for international callers. A live webcast will be available in the Events and Presentations section of the Investor Relations page at View Source, or by clicking here.

Please log in approximately 10 minutes prior to the scheduled start time. The archived webcast will be available in the Events and Presentations section of the Company’s website.

About Twirla
Twirla (levonorgestrel and ethinyl estradiol) transdermal system is a once-weekly combined hormonal contraceptive (CHC) patch that contains the active ingredients levonorgestrel (LNG), a type of progestin, and ethinyl estradiol (EE), a type of estrogen. Twirla is indicated for use as a method of contraception by women of reproductive potential with a body mass index (BMI) < 30 kg/m2 for whom a combined hormonal contraceptive is appropriate to prevent pregnancy. Healthcare providers (HCPs) are encouraged to consider Twirla’s reduced efficacy in women with a BMI ≥ 25 to <30 kg/m2 before prescribing. Twirla is contraindicated in women with a BMI ≥ 30 kg/m2. Twirla is designed to be applied once weekly for three weeks, followed by a week without a patch.

On November 12, 2020 TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage immunotherapy company with a pipeline of novel T cell therapies for patients suffering from cancer, reported financial results for the third quarter ended September 30, 2020 and provided a corporate update (Press release, TCR2 Therapeutics, NOV 12, 2020, View Source [SID1234570769]).

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"The third quarter was marked by consistency in the clinical benefit provided by our lead TRuC-T cell program, TC-210, previously shown to have produced RECIST partial responses at its very first dose level, as well as the completion of a successful financing," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "As we prepare for the expansion portion of the TC-210 trial, we are excited to bring on additional clinical sites to increase patient recruitment and we have also launched a strategic partnership with ElevateBio, whose state-of-the-art manufacturing center will support the Phase 2 clinical trial of TC-210 and planned investigator-initiated studies. We look forward to providing a clinical data update on TC-210 in the fourth quarter."

Recent Developments

TCR2 announced a partnership with ElevateBio, LLC, a Cambridge, MA-based creator and operator of a portfolio of innovative cell and gene therapy companies. The agreement with ElevateBio provides TCR2 access to ElevateBio BaseCamp, its centralized 140,000 square foot, world-class cell and gene therapy manufacturing facility based in Waltham, MA. The BaseCamp partnership enables TCR2 Therapeutics to establish additional manufacturing capacity and technical capabilities in the U.S., in addition to its existing Stevenage, UK, manufacturing facility, and will support the Phase 2 expansion portion of the TC-210 Phase 1/2 clinical trial once a recommended Phase 2 dose is defined.
Anticipated Milestones

TCR2 anticipates an interim update from the Phase 1 portion of the TC-210 Phase 1/2 clinical trial for patients with mesothelin-expressing solid tumors in 4Q20.
TCR2 anticipates an interim update from the Phase 1 portion of the TC-110 Phase 1/2 clinical trial for patients with CD19+ non-Hodgkin lymphoma or adult acute lymphoblastic leukemia in 2021.
TCR2 anticipates certification of its manufacturing facility in Stevenage, UK, in 4Q20.
TCR2 anticipates an IND filing for a third TRuC-T cell program in 2021.
Financial Highlights

Cash Position: TCR2 ended the third quarter of 2020 with $246.7 million in cash, cash equivalents, and investments compared to $158.1 million as of December 31, 2019. Net cash used in operations was $10.8 million for the third quarter of 2020 compared to $10.1 million for third quarter of 2019. TCR2 projects net cash use of $65-70 million for 2020.

R&D Expenses: Research and development expenses were $12.8 million for the third quarter of 2020 compared to $11.3 million for the third quarter of 2019. The increase in R&D expenses were primarily related to increases in headcount, activities related to the Phase 1/2 clinical trial of TC-210 and activities related to the Phase 1/2 clinical trial of TC-110.

G&A Expenses: General and administrative expenses were $4.4 million for the third quarter of 2020 compared to $3.5 million for the third quarter of 2019. The increase in general and administrative expenses was primarily due to an increase in personnel costs and costs associated with operations as a public company.

Net Loss: Net loss was $16.9 million for the third quarter of 2020 compared to $13.8 million for the third quarter of 2019, driven predominantly by increased personnel expenses.
Upcoming Events

TCR2 Therapeutics management is scheduled to participate at the following upcoming conferences.

Jefferies Virtual London Healthcare Conference: Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics, will provide a company update using a virtual platform on Wednesday, November 18, 2020 at 9:40am ET
Piper Sandler 32nd Annual Virtual Healthcare Conference: Ian Somaiya, Chief Financial Officer of TCR2 Therapeutics, and Alfonso Quintás Cardama, M.D., Chief Medical Officer of TCR2 Therapeutics, will participate in a fireside chat using a virtual platform on Monday, November 23, 2020 at 10:00am ET

On November 12, 2020 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, reported financial results for the third quarter ended September 30, 2020. The company also highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Forma Therapeutics, NOV 12, 2020, View Source [SID1234570767]).

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"We are very pleased with our strong pipeline progress during the quarter amid such challenging times," said Frank Lee, President and Chief Executive Officer of Forma. "We look forward to presenting new data from our ongoing Phase 1 trial of FT-4202 in sickle cell disease at the ASH (Free ASH Whitepaper) meeting in December, as well as beginning enrollment of patients for our Phase 1 trial of FT-7051 in men living with metastatic castration-resistant prostate cancer. The recent positive top-line results from the olutasidenib registrational Phase 2 clinical trial in relapsed/refractory acute myeloid leukemia with an IDH1 mutation further underscores our commitment to developing transformative therapies for patients."

Key Business and Clinical Highlights

PKR Program in Sickle Cell Disease (SCD):

Both planned dose cohorts enrolling in the multiple ascending dose (MAD) trial. The MAD1 cohort is designed to dose 9-12 SCD patients with 300 mg of FT-4202. Clinical measures being assessed include change in hemoglobin, indirect bilirubin, reticulocytes and lactate dehydrogenase, as well as the monitoring of tolerability and safety during the 14-day dosing and 7-day follow-up period. The MAD2 cohort is assessing a higher 600 mg dose and is now enrolling patients. Patients completing the 600 mg MAD2 cohort may enter the 12-week Open Label Extension (OLE) portion of the trial.
FT-4202 abstract selected for oral presentation at the virtual 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition December 5-8, 2020. The FT-4202 abstract describes blinded data from three patients receiving the 300 mg dose, measuring changes in parameters over the 14-day treatment and 7-day follow-up period including hemoglobin and reticulocytes, as well as tolerability and safety. Updated data will be presented at the ASH (Free ASH Whitepaper) annual meeting on December 7, 2020.
CPB/p300 Program in Prostate Cancer:

Phase 1 clinical trial of FT-7051 for the treatment of metastatic castration-resistant prostate cancer (mCRPC) on track to start by year end. This trial will enroll patients who have progressed while on standard anti-androgen therapy. Patients’ prostate cancer will be profiled for mutations in the androgen receptor (AR)-signaling pathway that drive resistance to AR-receptor antagonists, such as ARv7 mutations.
IDH1 Program in AML and Glioma:

Announced positive data for olutasidenib in relapsed/refractory acute myeloid leukemia (R/R AML). In October 2020, Forma announced positive results from the planned interim analysis (IA2) of the Phase 2 registration trial in R/R AML patients with isocitrate dehydrogenase 1 gene mutations (IDH1m). Olutasidenib demonstrated a favorable tolerability profile as a monotherapy, and for the primary efficacy endpoint of composite complete remission (CR+CRh, or complete remission plus complete remission with partial hematologic recovery), achieved a rate of 33.3% (30% CR and 3% CRh). While a median duration of CR/CRh has not been reached, a sensitivity analysis (with a hematopoietic stem cell transplant as the end of a response) indicates the median duration of CR/CRh to be 13.8 months. Safety results are consistent with previously reported Phase 1 clinical trial results.
Olutasidenib is also being evaluated in an exploratory Phase 1 trial for glioma as presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting in June 2020, as well as in other IDH1m solid tumor indications.
Corporate:

In September 2020, Forma announced the appointment of industry veteran Thomas G. Wiggans to Forma’s board of directors. Mr. Wiggans has led successful biopharmaceutical companies from start-up stage into the clinic and later global commercialization, served on the boards of numerous public and private companies, and was instrumental in the formation of the Biotechnology Industry Organization, now Biotechnology Innovation Organization (BIO).
Upcoming Milestones

Results from the ongoing randomized placebo-controlled multicenter Phase 1 trial evaluating FT-4202 in patients with SCD to be presented at ASH (Free ASH Whitepaper). Clinical data on the safety results, PK/PD and laboratory measurements in 9-12 patients from the 300 mg MAD1 cohort will be presented during an oral presentation at the virtual, 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition December 5-8, 2020. Subsequently, results from the MAD2 600 mg cohort are expected in the first quarter of 2021, and 12-week OLE results are anticipated in the second quarter of 2021.
Initiation of registrational trial of FT-4202 for people living with SCD: The global pivotal Phase 2/3 trial is expected to initiate in the first quarter of 2021. This adaptive, randomized, placebo-controlled, double-blind, multi-center study will enroll approximately 344 adults and adolescents with SCD. The trial will evaluate FT-4202 doses of 200 mg and 400 mg administered once daily in the Phase 2 portion. Primary endpoints in the Phase 3 portion of the trial are hemoglobin response rate at week 24 (increase of > 1 g/dL from baseline), and annualized vaso-occlusive crisis rate during the 52-week blinded treatment period.
Initiation of FT-7051 Clinical Development in mCRPC: Patient enrollment in the Phase 1 trial of FT-7051 in mCRPC patients is expected to begin prior to the end of 2020. Safety and tolerability data from the trial are anticipated in 2021 and clinical activity results in 2022.
Non-core Partnering Strategy: Following the recent positive registrational trial results in R/R AML with an IDH1 mutation, Forma remains focused on partnership opportunities for olutasidenib, as well as for the non-core FASN inhibitor for NASH (FT-8225).
Possibility of COVID-19 Impact: The COVID-19 pandemic remains a factor in the successful completion of these milestones. Many clinical trials across the biopharma industry have been impacted by the COVID-19 pandemic, with clinical trial sites implementing new policies in response to COVID-19, resulting in potential delays to enrollment of clinical trials or changes in the ability to access sites participating in clinical trials.
Upcoming Investor Events

Dec. 7, 2020: Forma will conduct a conference call and webcast on Dec. 7 at 6 p.m. Eastern Standard Time (EST) to discuss updated results from the ongoing Phase 1 trial of FT-4202 in SCD, as well as an overview of the company’s development plans for FT-4202. A live webcast will be available in the "News & Investors" section of Forma’s website www.formatherapeutics.com.
Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $384.3 million as of September 30, 2020, as compared to $173.2 million as of December 31, 2019.
Research and Development (R&D) Expenses: R&D expenses were $24.8 million for the quarter ended September 30, 2020, compared to $27.6 million for the quarter ended September 30, 2019. The decrease was primarily due to planned reductions in spending on FT-2102, FT-4101, FT-8225, research activities, and internal R&D personnel-related costs, which were partially offset by increases in FT-4202 expenses to conduct the Phase 1 trial, clinical product manufacturing, and preparations for the pivotal Phase 2/3 trial.
General and Administrative (G&A) Expenses: G&A expenses were $7.5 million for the quarter ended September 30, 2020, compared to $7.0 million for the quarter ended September 30, 2019. The increase in general and administrative expense was primarily attributable to a $1.3 million increase in equity-based compensation, and a $0.6 million increase in insurance related expense, and a $0.5 million increase in other related general and administrative costs, partially offset by a reduction of $2.0 million related to legal, consulting and other professional fee expenses.
Net Income/Loss: Net loss was $27.6 million for the quarter ended September 30, 2020, compared to $31.0 million for the quarter ended September 30, 2019.

On November 12, 2020 Myovant Sciences (NYSE: MYOV), a healthcare company focused on redefining care for women and for men, reported corporate updates and financial results for the second quarter fiscal year 2020 (Press release, Myovant Sciences, NOV 12, 2020, View Source [SID1234570766]).

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"I am very pleased with the significant progress we made during the second fiscal quarter in advancing both relugolix monotherapy tablet and relugolix combination tablet toward potential regulatory approvals, while preparing for commercialization in advanced prostate cancer, uterine fibroids, and endometriosis," said Lynn Seely, M.D., chief executive officer of Myovant Sciences, Inc. "We have assembled a strong and highly-experienced team, spanning medical affairs, market access, commercial operations, marketing, and sales, which will enable us to rapidly and efficiently deliver relugolix monotherapy tablet to urologists, medical oncologists, and their patients, if approved, for our first commercial launch."

Second Quarter Fiscal Year 2020 and Recent Corporate Updates

Relugolix Clinical Programs

Prostate Cancer:

Relugolix monotherapy tablet is under Priority Review by the U.S. Food and Drug Administration (FDA) and is on track for a decision by its December 20, 2020 target action date. The NDA is supported by the positive Phase 3 HERO study results, including a 97% responder rate and six positive key secondary endpoints. Relugolix also demonstrated a lower incidence of major adverse cardiovascular events compared to leuprolide acetate, the current standard of care. The Phase 3 HERO study results were published in the New England Journal of Medicine on June 4, 2020.

On September 29, 2020, Myovant announced results of an additional secondary endpoint of castration resistance-free survival assessed in the subgroup of men with metastatic prostate cancer from the Phase 3 HERO study of relugolix monotherapy in advanced prostate cancer. Relugolix monotherapy had a similar rate of castration resistance-free survival compared to leuprolide acetate (74% vs. 75%, respectively), and did not achieve statistical superiority (p = 0.84).

On October 19, 2020, Myovant presented an economic analysis of the Phase 3 HERO data at the Academy of Managed Care Pharmacy (AMCP) Nexus 2020 Virtual Meeting, demonstrating that treatment with oral relugolix may prevent one major adverse cardiovascular event for every 31 patients treated versus patients receiving leuprolide injections.

Uterine Fibroids:

In August 2020, the FDA accepted Myovant’s NDA for once-daily, oral relugolix combination tablet for the treatment of women with heavy menstrual bleeding associated with uterine fibroids, setting a target action date of June 1, 2021.

On September 14, 2020, Myovant announced one-year data on bone mineral density (BMD) from the Phase 3 LIBERTY program. The BMD results from the LIBERTY program demonstrated maintenance of BMD through one year and were consistent with those observed in a separate prospective observational study of untreated, age-matched women with uterine fibroids. These findings were presented at the American Society for Bone and Mineral Research (ASBMR) 2020 Annual Meeting Virtual Event, held on September 11-15, 2020.

On October 21, 2020, Myovant announced the presentation of data at the American Society for Reproductive Medicine (ASRM) 2020 Virtual Congress, including long-term extension data in women with symptomatic uterine fibroids. Results indicated that women experienced, on average, a 90% reduction in menstrual blood loss from baseline at one year. Additionally, 87.7% of women achieved the responder criteria for reduction in menstrual blood loss at one year, and lumbar spine and total hip BMD were maintained over one year. A poster presentation also described a validated exposure-response model simulating long-term effects of relugolix combination therapy on BMD at the lumbar spine that projected maintenance of BMD for at least three years. Other data from the LIBERTY program – including improvement in quality of life, reduction in menstrual blood loss in the first treatment cycle, and reduction in uterine fibroid-associated pain – were also presented.

Additional data from the Phase 1 ovulation inhibition study from 67 healthy women treated with relugolix combination therapy, resulting in 100% ovulation inhibition and 100% return to ovulation or menses after discontinuation of treatment, were also presented at the ASRM 2020 Virtual Congress.

Endometriosis:

Data from the replicate Phase 3 SPIRIT 1 and SPIRIT 2 studies were presented at the ASRM 2020 Virtual Congress on October 20, 2020 in an oral presentation named the Prize Paper by the Endometriosis Special Interest Group. Relugolix combination therapy resulted in clinically meaningful reductions in dysmenorrhea and non-menstrual pelvic pain, compared with placebo over 24 weeks of therapy (p < 0.0001) in each study. Changes in BMD over 24 weeks were minimal in the relugolix combination therapy groups.
COVID-19 Pandemic Environment

Myovant’s priorities during the COVID-19 pandemic are protecting the health and safety of its employees and patients while continuing its mission to redefine care for women and for men. To date, the impact of the COVID-19 pandemic on Myovant’s ability to advance its clinical studies, regulatory activities, and preparation for the potential commercialization of its product candidates has been limited, and all of Myovant’s publicly announced milestones remain on track. However, if the COVID-19 pandemic persists, and depending on the further evolution of the pandemic and its effects on Myovant’s activities, Myovant may experience more significant impacts on its business operations.
Expected Upcoming Milestones

Relugolix monotherapy tablet for advanced prostate cancer FDA target action date of December 20, 2020.

Data from the LIBERTY randomized withdrawal study expected in the first quarter of calendar year 2021.

One-year efficacy and safety data from the SPIRIT extension study expected in the first quarter of calendar year 2021.

Relugolix combination tablet for uterine fibroids FDA target action date of June 1, 2021.

Marketing Authorization Application (MAA) submission to European Medicines Agency (EMA) for relugolix monotherapy tablet for advanced prostate cancer in the first half of calendar year 2021.

NDA submission for relugolix combination tablet for the treatment of women with endometriosis-associated pain expected in the first half of calendar year 2021.

European Commission decision on the uterine fibroids MAA expected in 2021. If approved, this launch will be executed by Gedeon Richter, Myovant’s commercialization partner for relugolix combination tablet for the uterine fibroids and endometriosis indications in Europe and certain other international markets.

MAA submission to EMA for relugolix combination tablet for the treatment of women with endometriosis-associated pain expected in 2021. Gedeon Richter will be the MAA sponsor.
Second Quarter Fiscal Year 2020 Financial Summary

Research and development (R&D) expenses in the three months ended September 30, 2020, were $40.5 million compared to $50.8 million for the comparable prior year period. The decrease in R&D expenses reflects a decrease in clinical study costs as a result of the completion and continued wind down of Myovant’s Phase 3 LIBERTY, HERO, and SPIRIT studies. This decrease was partially offset primarily by increased expenses by the medical affairs organization in preparation for Myovant’s anticipated commercial launches, if approved, of relugolix monotherapy tablet for men with advanced prostate cancer and relugolix combination tablet for the women’s health indications, as well as an increase in personnel expenses.

General and administrative (G&A) expenses in the three months ended September 30, 2020, were $31.3 million compared to $16.6 million for the comparable prior year period. The increase was primarily due to increases in expenses related to commercial readiness activities, personnel-related expenses, and other general overhead expenses to support Myovant’s organizational growth and anticipated commercial launches, if approved, of relugolix monotherapy tablet for men with advanced prostate cancer and relugolix combination tablet for the women’s health indications.

Interest expense was $2.1 million in the three months ended September 30, 2020, compared to $3.8 million in the comparable prior year period. The decrease in interest expense was driven by lower interest rates associated with the Sumitomo Dainippon Pharma Loan Agreement as compared to Myovant’s previously outstanding debt obligations, which were repaid in December 2019.

Interest income in the three months ended September 30, 2020, was less than $0.1 million compared to $0.9 million for the comparable prior year period. The decrease was primarily due to decreases in interest rates and lower balances in cash equivalents and marketable securities.

Other (income) expense, net in the three months ended September 30, 2020, was income of $6.7 million compared to expenses of $0.1 million for the comparable prior year period. This was primarily the result of a foreign currency exchange gain on Myovant’s outstanding balance under the Sumitomo Dainippon Pharma Loan Agreement during the three months ended September 30, 2020 for which there was no such gain in the comparable prior year period.

Net loss for the three months ended September 30, 2020, was $67.1 million compared to $70.6 million for the comparable prior year period. On a per common share basis, net loss was $0.75 and $0.79 for the three months ended September 30, 2020, and 2019, respectively.

Capital resources: Cash, cash equivalents, marketable securities, and committed amounts available under the Sumitomo Dainippon Pharma Loan Agreement totaled $257.6 million as of September 30, 2020, and consisted of $111.3 million of cash, cash equivalents, and marketable securities and $146.3 million of available borrowing capacity under the Sumitomo Dainippon Pharma Loan Agreement.

On August 5, 2020, Myovant obtained a debt commitment letter (amended on September 29, 2020) from Sumitomo Dainippon Pharma, pursuant to which, subject to the terms and conditions set forth therein, Sumitomo Dainippon Pharma has committed to provide Myovant with an additional $200.0 million, low-interest, five-year term loan, which, subject to negotiation of a definitive agreement, will bring its total financing support for Myovant to $600.0 million. Including the additional debt commitment, cash and committed funding as of September 30, 2020 totaled approximately $460.0 million.

Conference Call
As previously announced, Myovant will hold a webcast and conference call at 8:30 a.m. Eastern Time (5:30 a.m. Pacific Time) today, November 12, 2020, to discuss corporate updates and financial results for its second fiscal quarter 2020, ended September 30, 2020. Investors and the general public may access a live webcast of the call by visiting the investor relations page of Myovant’s website at investors.myovant.com. Institutional investors and analysts may also participate in the conference call by dialing 1-800-532-3746 in the U.S. or +1-470-495-9166 from outside the U.S.

The webcast will be archived on Myovant’s Investor Relations website following the call.

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