On January 29, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, "the Company") reported the financial results for the first nine months (April 1, 2020 – December 31, 2020) of the fiscal year 2020 (FY2020) ending March 31, 2021 (Press release, Astellas, JAN 29, 2021, View Source [SID1234574380]).

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1. Qualitative information on consolidated financial results for the first nine months of FY2020 (1) Business performance
Consolidated financial results (core basis) in the first nine months of FY2020 are shown in the table below.

Revenue, core operating profit and core profit decreased across the board. Revenue-Sales of main products XTANDI for the treatment of prostate cancer and XOSPATA for the treatment of acute myeloid leukemia continued to grow.

In addition, growth of the co-promotion revenue of PADCEV for the treatment of urothelial cancer, which was launched in the United States in December 2019, contributed to revenue.

-Moreover, sales of Betanis / Myrbetriq / BETMIGA for the treatment of overactive bladder ("OAB") showed steady progress, and new product group in Japan achieved sales growth, including those of EVENITY for the treatment of osteoporosis, Suglat and SUJANU Combination Tablets for the treatment of diabetes mellitus.
-However, revenue decreased mainly due to the loss of market exclusivity of Vesicare for the treatment of OAB in Europe and Celecox for the treatment of inflammation and pain in Japan, and the termination of sales agreements for Symbicort for the treatment of asthma, human vaccines of KM Biologics Co., Ltd. and Micardis family for the treatment of hypertension. Sales were also negatively impacted due to the spread of COVID-19.

As a result of the above, revenue in the first nine months of FY2020 decreased by 4.8% compared to those in the corresponding period of the previous fiscal year ("year-on-year") to ¥940.9 billion. Core operating profit / Core profit-Gross profit decreased by 1.8% year-on-year to ¥753.2 billion.

The cost-to-revenue ratio fell by 2.5 percentage points year-on-year to 20.0%, mainly due to changes in product mix.-Selling, general and administrative expenses increased by 2.7% year-on-year to ¥363.0 billion.

There was a decrease in expenses due to refraining from promotional activities, etc. due to the spread of COVID-19, but total selling, general and administrative expenses increased due to the increase of co-promotion fees associated with the growth of sales of XTANDI in the United States, and there was a one-off reducing factor on expenses from a reversal of loss allowances in the corresponding period of the previous fiscal year.-Research and development (R&D) expenses increased by 5.7% year-on-year to ¥168.8 billion.

There was a decrease in development expenses due to the impact of the spread of COVID-19 on the execution of a portion of clinical trials, but total R&D expenses increased due to an increase in development expenses for key post-POC pipeline projects, and the addition of R&D expenses from Audentes Therapeutics, Inc., which was acquired in January 2020. The R&D cost-to-revenue ratio was up 1.8 percentage points year-on-year to 17.9%.-Amortisation of intangible assets increased by 12.0% year-on-year to ¥17.3 billion.

As a result of the above, core operating profit decreased by 13.6% year-on-year to ¥203.7 billion, and core profit decreased by 13.1% year-on-year to ¥166.6 billion. 3 Impact of exchange rate on financial results The exchange rates for the yen in the first nine months of FY2020 are shown in the table below. The resulting impacts were a ¥7.3 billion decrease in revenue and a ¥3.6 billion decrease in core operating profit compared with if the exchange rates of the first nine months of FY2019 were applied.

Average rate First nine months
Consolidated financial results on a full basis in the first nine months of FY2020 are shown in the table below. Revenue, operating profit, profit before tax and profit decreased across the board. The full basis financial results include "Other income," "Other expenses," which are excluded from the core basis financial results. In the first nine months of FY2020, "Other income" was ¥7.0 billion (¥15.1 billion in the same period of the previous fiscal year) and "Other expenses" was ¥51.3 billion (¥13.4 billion in the same period of the previous fiscal year).

As "Other expenses," the Company recorded impairment losses of ¥30.2 billion in relation to the termination of development for the anti-TIGIT antibody ASP8374/PTZ-201 in the first six months of FY2020, and as a result, the decrease in profit was larger compared to the financial results on a core basis. * Prograf: Includes Advagraf, Graceptor, and ASTAGRAF XL.-Sales of XTANDI increased by 15.0% year-on-year to ¥342.7 billion. Sales increased in all regions of Japan, United States, Established Markets, Greater China, and International.-Sales of XOSPATA increased by 80.7% year-on-year to ¥17.6 billion. This was due to its sales growing in Japan and United States in addition to the contribution of its sales in Established Markets, where it was launched in November 2019.-Co-promotion revenue of PADCEV amounted to ¥9.4 billion in United States.-Sales of Betanis / Myrbetriq / BETMIGA increased by 1.0% year-on-year to ¥122.3 billion.

While sales grew in United States, Established Markets and Greater China, sales decreased in Japan and International due to decreased demand, etc. associated with the reduction of patient visits to hospitals/clinics as a result of the impact of the spread of COVID-19.-Sales of Vesicare decreased by 31.8% year-on-year to ¥24.7 billion mainly due to the impact of the effect of generic drugs resulting from the loss of market exclusivity for the drug in Europe.

-Sales of Prograf decreased by 5.4% year-on-year to ¥138.3 billion. While sales grew in Greater China, sales decreased in other regions.
-In Japan, new product group sales continued to increase, including those of EVENITY, Suglat and SUJANU Combination Tablets. On the other hand, the main factors for the decrease in sales were the loss of market exclusivity for Celecox and the termination of sales agreements for Symbicort, human vaccines of KM Biologics Co., Ltd. and Micardis family.

-In United States, sales of pharmacologic stress agent Lexiscan decreased due to decreased demand associated with the reduction of patient visits to hospitals/clinics as a result of the impact of the spread of COVID-19, mainly in the first three months of FY2020. 6 Revenue by region is shown in the table below. Revenue in United States increased, and revenue in Established Markets achieved similar levels year on year. On the other hand, revenue in Japan, Greater China and International decreased.

(2) Financial position i. Assets, equity and liabilities In the first three months of FY2020, the consolidated statement of financial position as of March 31, 2020 was retrospectively revised due to adjustments of fair value of assets acquired and liabilities assumed for Audentes Therapeutics, Inc., which was acquired in January 2020.

As a result, goodwill increased, and intangible assets and deferred tax liabilities decreased in comparison to the figures prior to the retrospective adjustment. The Company is still in the process of finalizing the fair value measurement as of December 31, 2020. An overview of the consolidated statement of financial position as of December 31, 2020 and the main changes from the end of the previous fiscal year after the retrospective adjustment are shown below. Assets Total assets as of December 31, 2020 saw a decrease of ¥18.3 billion compared to the end of the previous fiscal year to ¥2,296.8 billion.

As of December 31, 2020: ¥1,433.5 billion (a decrease of ¥14.1 billion)-Property, plant and equipment decreased by ¥13.4 billion compared to the end of the previous fiscal year to ¥255.2 billion.-Goodwill decreased by ¥10.5 billion compared to the end of the previous fiscal year to ¥267.8 billion, and intangible assets decreased by ¥27.6 billion compared to the end of the previous fiscal year to ¥697.2 billion. Intangible assets decreased mainly due to the recording of impairment losses in relation to the termination of development for the anti-TIGIT antibody ASP8374/PTZ-201 in the first six months of FY2020.

As of December 31, 2020: ¥863.3 billion (a decrease of ¥4.2 billion)-Cash and cash equivalents decreased by ¥11.9 billion compared to the end of the previous fiscal year to ¥306.5 billion.

Equity Total equity as of December 31, 2020 saw an increase of ¥79.4 billion compared to the end of the previous fiscal year to ¥1,368.6 billion, making the ratio of equity attributable to owners of the parent to gross assets 59.6%.-While profit stood at ¥132.9 billion, the Company paid ¥76.2 billion of dividends of surplus. 8 Liabilities Total liabilities decreased by ¥97.8 billion compared to the end of the previous fiscal year to ¥928.2 billion.

As of December 31, 2020: ¥292.4 billion (an increase of ¥65.1 billion)-Other financial liabilities increased by ¥69.7 billion compared to the end of the previous fiscal year to ¥199.0 billion mainly due to converting ¥80.0 billion from short-term borrowings to long-term borrowings in the first three months of FY2020.

As of December 31, 2020: ¥635.8 billion (a decrease of ¥162.9 billion)-As of December 31, 2020, the balance of bonds amounted to ¥165.0 billion. Other financial liabilities decreased by ¥156.2 billion compared to the end of the previous fiscal year to ¥189.5 billion due to the conversion from short-term borrowings to long-term borrowings stated above, repayments, etc.ii. Cash flow Cash flows from operating activities Net cash flows from operating activities in the first nine months of FY2020 increased by ¥54.9 billion year-on-year to ¥225.1 billion

.-Income tax paid decreased by ¥20.0 billion year-on-year to ¥10.5 billion. Cash flows from investing activities Net cash flows used in investing activities in the first nine months of FY2020 was ¥67.7 billion, a decrease in outflow of ¥6.7 billion year-on-year. Cash flows from financing activities Net cash flows used in financing activities in the first nine months of FY2020 was ¥171.3 billion, an increase in outflow of ¥46.1 billion year-on-year.

-While proceeds from long-term borrowings amounted to ¥80.0 billion, the balance of bonds and short-term borrowings decreased by ¥161.0 billion. Dividends paid increased by ¥2.6 billion year-on-year to ¥76.2 billion.

As a result, cash and cash equivalents totaled ¥306.5 billion as of December 31, 2020, a decrease of ¥11.9 billion compared to the end of the previous fiscal year.(3) Consolidated business forecasts for FY2020 and other forward-looking statements The Company’s business forecasts are presented on a core basis and full basis. The consolidated full-year business forecasts for FY2020 are shown below.

The Company has left its business forecasts unchanged from the consolidated full-year business forecasts announced in October 2020.Business Combinations For the nine months ended 31 December 2020 Audentes Therapeutics, Inc. On 15 January 2020, Audentes Therapeutics, Inc. became a consolidated subsidiary of the Company through a cash tender offer followed by a merger. During the three months ended 30 June 2020, further facts came to light and additional analysis was performed on the fair value measurement of the assets acquired and liabilities assumed at the acquisition date. As a result, the provisional fair values were adjusted as follows.

The initial accounting for the business combination is incomplete as of 31 December 2020 as Astellas Pharma Inc. and its subsidiaries are still in the process of finalizing the fair value measurement.Goodwill mainly comprises the value of expected synergies arising from the acquisition and future economic benefits, which is not separately recognised. Financial assets at FVTOCI (debt instruments) are included in "Other financial assets" in the condensed interim consolidated statement of financial position.

Along with this adjustment, the Company retrospectively revised the corresponding balances in the condensed interim consolidated statement of financial position as of 31 March 2020. As a result, intangible assets and deferred tax liabilities decreased by 13,734 million yen and 2,992 million yen respectively, and goodwill increased by 10,743 million yen.

On January 29, 2021 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the conditional approval for NEXPOVIO (selinexor) in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents, and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy (Press release, Karyopharm, JAN 29, 2021, View Source [SID1234574431]).

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The positive CHMP opinion is a scientific recommendation for marketing authorization and one of the final steps before the European Commission (EC) makes a decision on Karyopharm’s marketing authorization application (MAA). An EC marketing authorization through the centralized procedure is valid in all 27 European Union member countries as well as the European Economic Area countries Iceland, Liechtenstein and Norway.

"We are delighted that the CHMP has adopted a positive opinion for NEXPOVIO, which could lead to Karyopharm’s first regulatory approval in Europe," said Sharon Shacham, PhD, MBA, Founder, President and Chief Scientific Officer of Karyopharm. "This positive opinion highlights the CHMP’s recognition of the positive clinical benefit-risk profile for oral NEXPOVIO and takes Karyopharm one step closer to bringing this important medicine to European patients in need of novel multiple myeloma treatment options. We look forward to the European Commission’s final decision on the NEXPOVIO MAA, which is expected by April of 2021."

The MAA is supported by data from the Phase 2b STORM study which evaluated selinexor in patients with heavily pretreated, triple class refractory multiple myeloma and published in the New England Journal of Medicine (Chari, et al.) in August 2019.

Karyopharm intends to submit a second regulatory filing to the EMA (Type II variation) by April 2021 based on the data from the confirmatory Phase 3 BOSTON study, which evaluated once-weekly NEXPOVIO in combination with once-weekly Velcade and low-dose dexamethasone in patients with multiple myeloma after at least one prior therapy with the goal of further expanding the global reach of NEXPOVIO to additional patients in need of new treatment options.

About the Phase 2b STORM Pivotal Trial

The Phase 2b STORM trial (Selinexor Treatment of Refractory Myeloma) was an international, multi-center, single-arm, open-label study which enrolled 122 patients (Part 2 of the trial) with heavily pretreated, triple class refractory multiple myeloma. Patients in the trial had a median of seven previous therapeutic regimens, including a median of 10 unique antimyeloma agents.

For the study’s primary endpoint, oral selinexor achieved an overall response rate of 26% (95% confidence interval [CI], 19, 35) and the trial therefore met its primary endpoint. Minimal response per IMWG criteria was observed in 16 (13%) patients and 48 patients (39%) had stable disease. All responses were adjudicated by an Independent Review Committee. The median overall survival was 8.6 months in the total population studied and 15.6 months in patients who had a minimal response or better.

Karyopharm’s request for conditional approval in Europe is based upon the same patient population that served as the basis for XPOVIO’s accelerated FDA approval in the U.S. Specifically, it includes the efficacy and safety data from a pre-specified sub-group analysis of 83 patients in the STORM study whose disease was refractory to bortezomib, carfilzomib, lenalidomide, pomalidomide, and daratumumab, as the benefit-risk ratio appeared to be greater in this more heavily pre-treated population than in the overall trial population. The overall response rate in this patient population was 25.3%.

The most common adverse reactions (≥20%) were thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.

About Multiple Myeloma in Europe

Multiple myeloma (MM) is an incurable cancer with significant morbidity and the second most common hematologic malignancy. In 2020, there were approximately 51,000 new cases and 32,000 deaths from MM in Europe1. While the treatment of MM has improved over the last 20 years, and overall survival has increased considerably, the disease remains incurable, and nearly all patients will eventually relapse and develop disease that is refractory to all approved anti-MM therapies. Therefore, there continues to be a high unmet medical need for new therapies, particularly those with novel mechanisms of action.

About NEXPOVIO (selinexor)

NEXPOVIO, which is marketed as XPOVIO in the U.S., is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. NEXPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). NEXPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion. XPOVIO is approved in the U.S. in multiple hematologic malignancy indications, including in combination with Velcade (bortezomib) and dexamethasone for the treatment of patients with multiple myeloma after at least one prior therapy, in combination with dexamethasone for the treatment of patients with heavily pretreated multiple myeloma and as a monotherapy for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. A Marketing Authorization Application for NEXPOVIO for patients with penta-refractory multiple myeloma is also currently under review by the European Medicines Agency and received a positive CHMP opinion in January 2021. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including as a potential backbone therapy in combination with approved myeloma therapies (STOMP), in liposarcoma (SEAL) and 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 January 29, 2021 Gan & Lee Pharmaceuticals Co., Ltd. (hereinafter referred to as Gan & Lee) (Shanghai: 603087.SH), a global biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for GLR2007, for the treatment of patients with glioblastoma (Press release, Gan and Lee Pharmaceuticals, JAN 29, 2021, View Source;lee-has-been-granted-fast-track-designation-by-the-us-fda-301218146.html [SID1234574430]). GLR2007 is a cyclin-dependent kinase 4/6 (CDK 4/6) inhibitor that Gan & Lee is developing for the treatment of advanced solid tumors including glioblastoma, an aggressive form of brain cancer with a low survival rate. Although considered a rare disease, glioblastoma is the most common brain and central nervous system (CNS) malignancy, accounting for 45.2% of malignant primary brain and CNS tumors[1].

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The one-year survival rate for glioblastoma is 39.3%. By year two and year five post-diagnosis, the survival rate drops to 16.9% and 5.5%, respectively. The average survival time for untreated patients is only three months[2]. Current available treatments improve prognosis only by a matter of months. According to Julius Huang, Director of Global Clinical Sciences, Gan & Lee, "The poor prognosis and low survival rates for glioblastomas, demonstrate an unmet need for new treatment options." The FDA’s Fast Track Designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Receiving Fast Track Designation potentiates frequent meetings and written communication with the FDA. The GLR2007 application is also eligible for Rolling Review and may be eligible for Accelerated Approval, and Priority Review[3].

On January 29, 2021 Scopus BioPharma Inc. (Nasdaq: "SCPS") reported the closing of a $9 million follow-on public offering (Press release, Scopus BioPharma, JAN 29, 2021, View Source [SID1234574429]). The offering consisted of 1,000,000 shares of common stock at a public offering price of $9.00 per share. The company has granted the underwriters a 45-day option to purchase up to an additional 150,000 shares of common stock at the public offering price.

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Scopus is a biopharmaceutical company developing transformational therapeutics based on groundbreaking scientific and medical discoveries. The company’s lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. This drug candidate is highly distinctive, encompassing both gene therapy and immunotherapy by synthetically linking siRNA to an oligonucleotide TLR9 agonist, creating the potential for targeted gene silencing with simultaneous TLR stimulation and immune activation in the tumor microenvironment.

Scopus intends to use the proceeds of the offering principally for further development of the company’s lead drug candidate, including in combination with checkpoint inhibitors.

The Benchmark Company, LLC acted as Sole Bookrunning Manager and Joseph Gunnar & Co., LLC acted as Co-Manager for the offering.

Greenberg Traurig, LLP is acting as counsel to the company. Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C. is acting as counsel to the underwriters.

An offering statement relating to the shares of common stock was filed with the U.S. Securities and Exchange Commission and became qualified on January 26, 2021. The offering is being made only by means of an offering circular, copies of which may be obtained, when available, by contacting: The Benchmark Company, LLC, Attention: Prospectus Department, 150 E. 58th Street, 17th Floor, New York, NY 10155, by calling (212) 312-6700 or by e-mail at [email protected]; or Joseph Gunnar & Co., LLC, Attention: Prospectus Department, 30 Broad Street, 11th Floor, New York, NY 10004, by calling (212) 440-9600 or by email at [email protected]. The offering circular is also available on the U.S. Securities and Exchange Commission website at www.sec.gov.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification of these securities under the securities laws of any such state or jurisdiction.

On January 29, 2021 ImmunoGen Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that the Company will host a conference call at 8:00 a.m. ET on Friday, February 12, 2021 to discuss its 2020 operating results (Press release, ImmunoGen, JAN 29, 2021, View Source [SID1234574428]). Management will also provide a brief update on the business.

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Conference Call Information

To access the live call by phone, dial (877) 621-5803; the conference ID is 1666147. The call may also be accessed through the Investors and Media section of the Company’s website, www.immunogen.com. Following the webcast, a replay of the call will be available at the same location.