On February 4, 2021 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that it will report fourth quarter and full year 2020 financial results on Thursday, February 11, 2021 (Press release, Karyopharm, FEB 4, 2021, View Source [SID1234574652]). Karyopharm’s management team will host a conference call and audio webcast at 8:30 a.m. ET on Thursday, February 11, 2021, to discuss the financial results and other company updates.

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To access the conference call, please dial (888) 349-0102 (local) or (412) 902-4299 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.

On February 4, 2021 Elucida Oncology, a biotechnology company based on C Dots – ultra-small nanoparticles developed at Cornell that show promise in identifying and fighting cancer – reported that secured $44 million in financing, in addition to $28 million raised in 2018 (Press release, Elucida Oncology, FEB 4, 2021, View Source [SID1234575245]).

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A rendering of the molecular structure of a Cornell dot, which is smaller than 10 nanometers.
C Dots, originally called Cornell dots, were created more than 15 years ago in the lab of Uli Wiesner, the Spencer T. Olin Professor of Engineering in the Department of Materials Science and Engineering. Wiesner has been working to put C Dots to use in the fight against cancer ever since.

As a result of their size, C Dots proved safe and effective for use in human beings as both an imaging and a diagnostic tool in early clinical studies. The new funding will help the company gain regulatory approval as a targeted cancer therapeutic, and to expand its management team and its laboratory capabilities.

"This vote of support from investors means a lot to me," Wiesner said. "Given C Dots’ broad applicability, I have been pushing efforts in health care-focused startups since 2005 to commercialize this technology. At the beginning it was a reagent company. Then we started a company focusing on diagnostics, and now we have a company emphasizing therapeutics."

This evolution shows the flexibility and the value of the C Dot platform, he said.

Elucida was founded in 2014 and co-founders include Wiesner, Kai Ma, Ph.D. ’15, and Dr. Michelle Bradbury, director of intraoperative imaging at Memorial Sloan Kettering Cancer Center and professor of radiology at Weill Cornell Medicine.

In the C Dots, each silica-based nanoparticle has an embedded fluorescent molecule in the interior for optical detection, as well as several organic ligands and functional groups on the surface. This makes the C Dot platform more flexible: Specific ligands prevent attack from the body’s defenses, while targeting groups cause the C Dot to specifically bind with tumor cells when they come into contact.

In this configuration, C Dots are currently being tested as a diagnostic tool in ongoing clinical trials at Memorial Sloan Kettering and Weill Cornell Medicine. When they are injected into a person with cancer, the glowing C Dots attach to cancer cells and can be seen by the surgeon with the use of a fluorescent camera.

In order to gain regulatory approval as a targeted cancer therapeutic, the company will need to complete ongoing toxicology studies and file an Investigational New Drug application with the Food and Drug Administration, according to Geno Germano, CEO and president of Elucida. The latest round of funding will support these moves.

"We have an excellent technology, an incredible team at Elucida, and a world-class board to help us navigate the highly complex landscape around cancer therapeutics," Wiesner said.

Elucida is planning to start clinical trials this summer; by the spring of 2022, the company expects to have the first data from those trials.

"If this trial validates our therapeutic approach the way we think and hope it will," Wiesner said, "our C Dot nanoparticle platform will give us a whole battery of disease-fighting products."

Chris Dawson is a writer for the College of Engineering.

On February 4, 2021 Kyowa Kirin Co., Ltd. reported its Financial Summary (IFRS) Fiscal 2020 (Press release, Kyowa Hakko Kirin, FEB 4, 2021, View Source [SID1234574597]).

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1. Consolidated Financial Results for the Fiscal Year Ended December 31, 2020 (from January 1, 2020 to December 31, 2020)
(1) Consolidated operating results
(2) Consolidated financial position
(3) Consolidated cash flows

3. Consolidated Earnings Forecasts for the Fiscal Year Ending December 31, 2021 (from January 1, 2021 to December 31, 2021)
(1) Non-consolidated operating results(2) Non-consolidated financial position These financial results reports are exempt from audit conducted by certified public accountants or an audit corporation.

* Notice regarding the appropriate use of the earnings forecasts and other special comments The forward-looking statements, including earnings forecasts, contained in these materials are based on the information currently available to the Company and on certain assumptions deemed to be reasonable by management. As such, they do not constitute guarantees by the Company of future performance. Actual results may differ materially from these projections for a wide variety of reasons. For more information regarding our suppositions that form the assumptions for the earnings forecasts, please see pages 17 and 18 of the attachment, "

(5) Outlook for Fiscal 2021" in "
1. Summary of Business Performance and Financial Position."
1. Summary of Business Performance and Financial Position

Responding to the massive changes to the business and social environments occurring as a result of the global spread of the novel coronavirus disease (COVID-19), the Kyowa Kirin Group (the "Group") has been striving to provide stable supply of pharmaceuticals, which is a core mission of a pharmaceutical company, as an utmost priority, and while paying meticulous attention to preventing infection, carrying out activities such as information provision.

Furthermore, as this fiscal year is the final year of our FY2016-2020 Mid-term Business Plan, we set our sights on achieving a further leap forward as a global specialty pharmaceutical company through various initiatives including efforts to maximize the value of three global strategic products, strengthen global governance, and research and development for future growth. In addition to changes to healthcare environments and restraints on business activities across the globe due to the COVID-19 pandemic, the Group faced other extremely difficult environments, such as the lowering of drug price standards in Japan.

Nevertheless, the Group increased its revenues mainly due to the penetration of three global strategic products in US/EU market. In Japan, the Group launched Duvroq, an oral treatment for renal anemia, in August 2020. By utilizing our abundant experience in the field of renal anemia, we carried out activities to provide information on proper use of medication, giving utmost attention to safety.

The Group is seeing steady progress for the three global strategic products. Regarding Crysvita, we obtained approvals for its additional indication for tumor induced osteomalacia in the United States and for extending its indication to include X-linked hypophosphatemia in older adolescents and adult patients in Europe and there was an increase in formulations for self-administration at home in Japan. Regarding the treatment for mycosis fungoides and Sézary syndrome, POTELIGEO, we commenced sales in Europe, beginning with Germany in June 2020.

Furthermore, regarding NOURIANZ (generic name: Istradefylline (product name in Japan: NOURIAST)), which has already been launched in the United States, our application for approval regarding its indication for combination therapy for Parkinson’s disease was accepted in Europe. Concerning the voluntary recall of mitomycin that occurred in 2019, the Group received the report on the investigation from the Group Investigation Committee spearheaded by a third-party in January 2020, and formulated the recurrence prevention measures.

As matters of the highest priority for management, the Group has formulated three key management priorities to strengthen our foundation as a global specialty pharmaceutical company: creation of a strong production and quality assurance system, improvement of risk management and reformation of corporate culture.

The Group will work on those management priorities continuously and sincerely over the five-year mid-term business plan commencing 2021.

(1) Summary of Business Performance in Fiscal 2020
1) Overview of results The Group now applies the International Financial Reporting Standards ("IFRS") in line with its policy of expanding business globally, and adopts "core operating profit" as a level of profit that shows the recurring profitability from operating activities. Core operating profit is calculated by deducting "selling, general and administrative expenses" and "research and development expenses" from "gross profit," and adding "share of profit (loss) of investments accounted for using equity method" to the amount.

For the fiscal year ended December 31, 2020, revenue was ¥318.4 billion (up 4.1% compared to the previous fiscal year) and core operating profit was ¥60.0 billion (up 1.0%). Profit attributable to owners of parent was ¥47.0 billion (down 29.9%).

 The increase in revenue was the result of steady growth of global strategic products in North America and EMEA and strong sales in Asia, mainly in China, despite the impact of lower revenue in Japan from the reduction in drug price standards and the switching to Darbepoetin Alfa Injection Syringe [KKF], an authorized generic of NESP, a renal anemia treatment drug, among others. The negative effect on revenue from foreign exchange was ¥2.9 billion.
 The increase in core operating profit was the result of an increase in gross profit due to an increase in overseas revenue, despite an increase in selling, general and administrative expenses, and a decrease in share of profit (loss) of investments accounted for using equity method. The negative effect on core operating profit from foreign exchange was ¥1.3 billion. 
Profit attributable to owners of parent decreased as a result of the absence of the profit from discontinued operations recorded in the previous fiscal year, despite lower business restructuring expenses and impairment losses in addition to an increase in core operating profit.

2) Revenue by regional control function
 Revenue in Japan decreased year on year because of the significant impact of switching to Darbepoetin Alfa Injection Syringe [KKF], an authorized generic of NESP, a renal anemia treatment drug whose patent has expired, in addition to the impact of the reductions in drug price standards implemented in October 2019 and April 2020, despite the growth in sales of new product groups.
 Darbepoetin Alfa Injection Syringe [KKF] achieved rapid progress in switching from NESP, a renal anemia treatment drug.
 Duvroq, an oral treatment for renal anemia, was launched in August 2020, and it is penetrating the market favorably.  Revenue from Patanol, anti-allergy eye drops, and ALLELOCK, an anti-allergy agent, decreased as a result of smaller pollen counts and the impact of the suppression of examinations, etc. due to COVID-19.
 Revenue from ORKEDIA, a treatment for secondary hyperparathyroidism, increased. Meanwhile, revenue from REGPARA, a treatment for secondary hyperparathyroidism, decreased due to factors such as switching to ORKEDIA and the impact of rival products.
 Revenue from ROMIPLATE, a treatment for chronic idiopathic thrombocytopenic purpura, increased as a result of receiving approval of its indication for treatment of patients with aplastic anemia who have had an inadequate response to conventional therapy, in June 2019.
 Firm growth in revenue was realized for G-Lasta, an agent for decreasing the incidence of febrile neutropenia, and Rituximab BS [KHK], an anticancer agent.
 In December 2019, Crysvita, a treatment for FGF23-related diseases, and HARUROPI, a Parkinson’s disease treatment patch, were launched and they have been penetrating the market favorably.
 Revenue in North America increased year on year due to the steady growth of global strategic products.
 Revenue from Crysvita, a treatment for X-linked hypophosphatemia, has been growing steadily since its launch in 2018. Approval for additional indication for treatment of tumor induced osteomalacia was acquired in June 2020.
 Revenue from POTELIGEO, an anticancer agent, stayed at the same level as in the previous fiscal year, due to the impact of the COVID-19 pandemic.
 NOURIANZ (product name in Japan: NOURIAST), an antiparkinsonian agent which was launched in October 2019, has been penetrating the market favorably.
 Revenue in EMEA increased year on year due to the steady growth of global strategic products.
 Revenue from Crysvita, a treatment for X-linked hypophosphatemia, has been growing steadily as the number of countries where it has been released has been increasing since its launch in 2018. Approval for sale with the extended indication for older adolescents and adults was acquired in September 2020.
 In Germany, sales of POTELIGEO an anticancer agent, was launched in June 2020, and it has been penetrating the market favorably as the number of countries where it has been released has been increasing.
 Revenue in Asia/Oceania increased year on year, reflecting strong sales particularly in China.
 Revenue from REGPARA, a treatment for secondary hyperparathyroidism, increased year on year due to market expansion in China.
 Revenue from Others decreased year on year.
 Revenue decreased year on year due to a decline in other income such as original equipment manufacturing despite an increase in technology out-licensing such as royalties revenue from AstraZeneca in relation to benralizumab. 3) Core operating profit
 Core operating profit increased year on year due to an increase in overseas revenue mainly from global strategic products, despite a lower gross profit due to a decrease in revenue in Japan, and an increase in selling, general and administrative expenses associated with sales of global strategic products.

(2) Summary of Consolidated Financial Position for Fiscal 2020
 Assets as of December 31, 2020, were ¥801.3 billion, an increase of ¥16.8 billion compared to the end of the previous fiscal year.
 Non-current assets increased by ¥23.0 billion to ¥358.8 billion, due mainly to increases in purchase of intangible assets associated with in-licensing of development products, and in deferred tax assets.
 Current assets decreased by ¥6.1 billion to ¥442.5 billion, due mainly to a decrease in cash reserves (total of cash and cash equivalents and loans receivable from parent) due in part to the purchase of intangible assets, despite large increases in cash and cash equivalents due to the impact of shifting the entire amount of loans receivable from parent to the loans with loan periods of three months or less included in the scope of cash and cash equivalents.
 Liabilities as of December 31, 2020, were ¥102.9 billion, a decrease of ¥3.3 billion compared to the end of the previous fiscal year, due mainly to a decrease in income taxes payable.
 Equity as of December 31, 2020, was ¥698.4 billion, an increase of ¥20.1 billion compared to the end of the previous fiscal year, due mainly to an increase due to the recording of profit attributable to owners of parent, despite a decrease due to the payment of dividends as well as a decrease in exchange differences on translation of foreign operations resulting from the impact of exchange rates, etc. As a result, the ratio of equity attributable to owners of parent to total assets was 87.2%, an increase of 0.7 percentage points compared to the end of the previous fiscal year.

(3) Cash Flow Summary for Fiscal 2020
 Cash and cash equivalents as of December 31, 2020, were ¥287.0 billion, an increase of ¥266.3 billion compared with the balance of ¥20.8 billion as of December 31, 2019, mainly as a result of the impact of shifting the entire amount of loans receivable from parent to the loans with loan periods of three months or less included in the scope of cash and cash equivalents. The main contributing factors affecting cash flow during the current fiscal year were as follows:
 Net cash provided by operating activities was ¥39.5 billion, a 26.4% decrease compared to the previous fiscal year. Major inflows included profit before tax from continuing operations of ¥52.3 billion and depreciation and amortization of ¥20.5 billion. Major outflows included income taxes paid of ¥28.7 billion.
 Net cash provided by investing activities was ¥252.6 billion, compared with net cash used in investing activities of ¥0.9 billion in the previous fiscal year. Major inflows included a net decrease of ¥285.6 billion in loans receivable from parent. Major outflows included ¥25.1 billion for purchase of intangible assets, and ¥10.1 billion for purchase of property, plant and equipment.
 Net cash used in financing activities was ¥26.0 billion, a 45.1% decrease compared to the previous fiscal year. Major outflows included dividends paid of ¥23.6 billion.

(4) Research and Development Activities The Group continuously and actively invests resources in research and development activities. We aim to advance both a technological pillar that can build a platform for applying various modalities and discovering innovative drugs and a disease pillar that continues to provide "only-one value drugs" for diseases for which there are no effective treatments while utilizing the disease science accumulated by the Group thus far, build a highly competitive pipeline, and provide new drugs with life-changing value worldwide. For the fiscal year ended December 31, 2020, the Group’s research and development expenses totaled ¥52.3 billion, and our progress in the respective disease fields of our main late-stage development products are as follows. ("◆" indicates the progress made during the fourth quarter of fiscal 2020.) Nephrology KRN321 (product name in Japan: NESP)

 In June 2020, we obtained approval of its indication for treatment of renal anemia in patients receiving hemodialysis in China. Oncology KRN125 (product name in Japan: G-Lasta)
 In February 2020, we started a phase I clinical study in Japan related to the development of an automated injection device for decreasing the incidence of febrile neutropenia in patients receiving cancer chemotherapy. ME-401 (generic name: Zandelisib)
 In North America, Europe, Asia, and Oceania, we are currently conducting a phase II clinical trial for treatment of follicular lymphoma. (In April 2020, we concluded an agreement with MEI Pharma on global license, development, and commercialization.)
 In October 2020 in Japan, we started a phase II clinical trial for its indication for treatment of relapsed or refractory indolent B-cell non-Hodgkin’s lymphoma (excluding small lymphocytic lymphoma, lymphoplasmacytic lymphoma, and Waldenström’s macroglobulinemia). KW-0761 (product name in Japan, U.S. and Europe: POTELIGEO)
 In December 2020, we applied for approval of its indication for treatment of mycosis fungoides and Sézary syndrome in South Korea. Immunology and allergy KHK4827 (product name in Japan: LUMICEF)
 In June 2020, we obtained approval of its indication for treatment of plaque psoriasis in China.
 In November 2020 in Japan, we obtained partial change approval for approved indications relating its treatment of ankylosing spondylitis and non-radiographic axial spondyloarthritis. Central nervous system (CNS) KW-6002 (product name in Japan: NOURIAST; product name in U.S.: NOURIANZ)
 In Europe, an application for approval of its indication for combination therapy with levodopa-based regimens for adult patients with Parkinson’s disease experiencing "off" episodes is currently under review (application accepted in January 2020). Other KRN23 (product name in Japan, U.S. and Europe: Crysvita)
 In February 2020, in the U.S., we obtained approval for partial changes to our biologics license application for approval of its indication for treatment of tumor induced osteomalacia that cannot be curatively resected or localized, and in June 2020, we obtained approval of its indication for treatment of tumor induced osteomalacia that cannot be curatively resected or localized for adult patients and pediatric patients who are two years of age or older.
 In September 2020, we obtained approval of its indication for treatment of X-linked hypophosphatemia in adolescent and adult patients in Europe.
 In September 2020, we obtained approval of its indication for treatment of FGF23-related hypophosphatemic rickets and osteomalacia in South Korea.
 In September 2020, we applied for approval of its indication for treatment of tumor induced osteomalacia in China.
 In December 2020, we applied for partial change approval for our biologics license regarding its indication for treatment of tumor induced osteomalacia in Europe.

(5) Outlook for Fiscal 2021
 Consolidated financial earnings forecasts for fiscal 2021 are for revenue of ¥351.0 billion (up 10.3% compared to the current fiscal year), core operating profit of ¥65.0 billion (up 8.4%), profit before tax of ¥64.0 billion (up 22.5%), and profit attributable to owners of parent of ¥50.0 billion (up 6.3%).
 Although we expect impacts such as a reduction in drug price standards scheduled for April 2021 in Japan, revenues are expected to increase compared to the current fiscal year due to significant growth in the global strategic products Crysvita, POTELIGEO and NOURIANZ overseas. Moreover, although we are planning to incur an increase in selling, general and administrative expenses in order to maximize the value of global strategic products and rapidly establish competitive global business bases and a significant increase in research and development expenses in association with advancements, etc. in late-stage development projects (R&D expense ratio will increase from 16% to 19%), core operating profit is expected to increase due to growth in overseas revenue.
 A year-on-year increase is forecasted for profit before tax as a result of a decrease in other expenses in addition to an increase in core operating profit.
 A year-on-year increase is forecasted for profit attributable to owners of parent despite an expected increase in income tax expense.
 Concerning cash flows from operating activities, net cash provided is expected to be higher in the next fiscal year than the current fiscal year as profit before tax is expected to be higher and the payment of income taxes is expected to be lower compared to the current fiscal year.
 Concerning cash flows from investing activities, the Company expects a decrease in net cash used compared to the current fiscal year mainly because of an expected decrease in cash used in the purchase of intangible assets. Regarding strategic partnering, M&A and other strategic investments for acquiring drug discovery technologies and pipelines, the Company will evaluate and conduct investment using a flexible approach.
 Concerning cash flows from financing activities, the Company expects net cash used to be at the same level as the current fiscal year. As regards the purchase of treasury shares and the sourcing of funds, we will remain flexible and act as appropriate for the economic and funding environment. As a result of the above, cash and cash equivalents as of the end of fiscal 2021 are expected to be higher compared to the end of fiscal 2020.

(6) Basic Policy on Profit Distribution: Fiscal 2020 and Fiscal 2021 Dividends The Company regards the return of profits to its shareholders as one of its key management priorities. The basis of the Company’s policy regarding the distribution of profits is to pay dividends stably in light of a comprehensive consideration of factors including consolidated results and dividend payout ratio for each fiscal year, while also increasing its retained earnings for future business development and other purposes. We plan to improve our capital efficiency by acting rapidly with regards to purchase of treasury shares.

The Company intends to use internal reserve funds for investments required to drive new growth, such as those in research and development, capital expenditures, and our development pipeline’s expansion that are expected to contribute to the improvement of our future corporate value. Concerning the dividend policy, in the FY2016-2020 Mid-term Business Plan, the Company sets its target consolidated dividend payout ratio at 40% and sets a policy of ensuring stable and continuous increase in the level of dividend payment in line with growth in profits. In accordance with the above-mentioned policy, the Board of Directors has resolved to pay a year-end dividend for fiscal 2020 of ¥22 per share.

As a result, we expect to increase dividends for the fourth year in a row. The annual dividend is expected to be ¥44, an increase of ¥2 compared to the previous fiscal year, including an interim dividend of ¥22. With respect to the year-end dividend, we plan to submit a proposal at the 98th Ordinary General Meeting of Shareholders to be held on March 24, 2021. As the dividend policy in the FY2021-2025 Mid-term Business Plan, the Company sets its target consolidated dividend payout ratio on core EPS at 40%. The Company intends to ensure stable and continuous increase in the level of dividend payment in line with growth in profits. In accordance with the above policy, for the fiscal year ending December 31, 2021, we expect to pay an annual dividend of ¥46 per share, an increase of ¥2 compared to the current fiscal year, consisting of an interim dividend of ¥23 and a year-end dividend of ¥23. For details of the "core EPS," refer to "(5) Outlook for Fiscal 2021."

2. Basic Rationale for Selection of Accounting Standards The Group has applied IFRS from fiscal 2017 to enhance the international comparability of its financial information in the capital markets, and unify the process of the Group’s accounting.

3. Consolidated Financial Statements and Significant

Notes Thereto
(1) Consolidated Statement of Financial Position
(1) Consolidated Statement of Financial Position (continued)
(2) Consolidated Statement of Profit or Loss and Consolidated Statement of Comprehensive Income Consolidated Statement of Profit or Loss
(3) Consolidated Statement of Changes in Equity
(3) Consolidated Statement of Changes in Equity (continued)
(4) Consolidated Statement of Cash Flows
(5) Notes to Consolidated Financial Statements Notes on going concern assumption No applicable items. Segment information, etc.

(1) Outline of reportable segments As the Bio-Chemicals business was categorized as a discontinued operation effective from the previous fiscal year, the Group omitted information by reportable segment as the Group consists of only the one reportable segment, which is the Pharmaceuticals business.

(2) Information about products and services Breakdown of revenue from external customers by product and service is as follows.

(3) Information about geographical areas

(4) Information about major customers

On February 4, 2021 Bristol Myers Squibb (NYSE:BMY) reported results for the fourth quarter and full year of 2020, which reflect robust sales, strong operating performance and advancement of the company’s product pipeline (Press release, Bristol-Myers Squibb, FEB 4, 2021, View Source [SID1234574618]).

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"In our first full year as a new company we delivered solid operational and financial results, and laid a strong foundation for the future," said Giovanni Caforio, M.D., board chair and chief executive officer, Bristol Myers Squibb. "I am grateful to our team whose resilience and continued focus enabled us to grow our inline business, launch promising new drugs and significantly advance our pipeline while keeping our colleagues safe and maintaining the supply of our medicines to patients. The growth opportunities from our in-line and launch portfolios combined with a robust product pipeline and disciplined business development strategy strongly position the company to accelerate the renewal of our portfolio and achieve long-term sustainable growth."

*The pro forma revenues assume the company’s acquisition of Celgene Corporation (Celgene Acquisition) and its divestiture of Otezla to Amgen Inc. (Otezla Divestiture) occurred on January 1, 2019 and exclude foreign currency hedge gains and losses. Management believes that measuring revenue rates on a comparable pro forma basis is an appropriate way for investors to best understand the underlying performance of the business. The pro forma revenue is presented for informational purposes only and does not purport to project the company’s revenue, results of operations or financial position for any future period or as of any future date. See "Worldwide Pro Forma Revenue" in Quarterly Package of Financial Information for this quarter and full year of 2020, which is available on bms.com/investors/financial-reporting/quarterly-results, for information on the revenue of the company and Celgene on a stand-alone basis for the prior-year period. Otezla is a trademark of Amgen Inc.

**In excess of +100%

FOURTH QUARTER FINANCIAL RESULTS

All comparisons are made versus the same period in 2019 unless otherwise stated.

Bristol Myers Squibb posted fourth quarter revenues of $11.1 billion, an increase of 39% on a reported basis and 10% on a pro forma basis. The increase was driven primarily by the impact of the Celgene Acquisition, which was completed on November 20, 2019.
U.S. revenues increased 43% to $6.8 billion in the quarter. International revenues increased 34% to $4.3 billion in the quarter. When adjusted for foreign exchange impact, international revenues increased 30%.
Gross margin increased from 68.6% to 73.7% in the quarter primarily due to product mix, lower unwinding of inventory purchase price accounting adjustments, partially offset by an impairment charge related to Inrebicmarketed product rights.
Marketing, selling and administrative expenses increased 57% to $2.7 billion in the quarter primarily due to $400 million of costs associated with the broader portfolio resulting from the Celgene Acquisition, as well as higher advertising and promotion expenses and cash settlement of MyoKardia unvested stock awards.
Research and development expenses increased 79% to $3.8 billion in the quarter primarily due to $500 million of costs associated with the broader portfolio resulting from the Celgene Acquisition, as well as license and acquisition charges related to Dragonfly, an in-process research and development (IPR&D) impairment charge related to the discontinuation of the orva-cel program development and cash settlement of MyoKardia unvested stock awards.
Amortization of acquired intangible assets increased to $2.5 billion in the quarter reflecting the full quarter amortization from the Celgene Acquisition.
IPR&D charge of $11.4 billion was included in the quarter due to the MyoKardia transaction being accounted for as an asset acquisition.
The effective tax benefit rate was 4.1% in the current quarter and includes the impact of the non-deductible MyoKardia IPR&D charge. Income taxes were $931 million despite pre-tax loss of $129 million in the same period a year ago primarily due to the Otezla divestiture, certain non-deductible expenses and purchase price adjustments.
The company reported net loss attributable to Bristol Myers Squibb of $10.0 billion, or $4.45 per share, in the fourth quarter, compared to net loss of $1.1 billion, or $0.55 per share, for the same period a year ago. The results in the current quarter include costs and expenses resulting from the IPRD charge related to the MyoKardia asset acquisition, purchase price accounting from the Celgene Acquisition, contingent value rights fair value adjustments, equity investment gains, intangible assets impairment charges and other acquisition and integration expenses.
The company reported non-GAAP net earnings attributable to Bristol Myers Squibb of $3.3 billion, or $1.46 per share, in the fourth quarter, compared to non-GAAP net earnings of $2.4 billion, or $1.22 per share, for the same period a year ago. A discussion of the non-GAAP financial measures is included under the "Use of Non-GAAP Financial Information" section.

*In excess of +100%. Product rights were acquired as part of the Celgene Acquisition.

**Pro forma product revenues assume the Celgene Acquisition and the Otezla Divestiture occurred on January 1, 2019 and exclude foreign currency hedge gains and losses. Management believes that measuring product revenue rates on a comparable pro forma basis is an appropriate way for investors to best understand the underlying performance of the business. The pro forma product revenue is presented for informational purposes only and does not purport to project product revenue for any future period or as of any future date. See "Worldwide Pro Forma Revenues" in the Quarterly Package of Financial Information for this quarter and full year of 2020, which is available on bms.com/investors/financial-reporting/quarterly-results, for information on the product revenue of the company and Celgene for the prior-year period. Otezla is a trademark of Amgen Inc.

*In excess of +100%. Product rights were acquired as part of the Celgene Acquisition.

**Pro forma product revenues assume the Celgene Acquisition and the Otezla Divestiture occurred on January 1, 2019 and exclude foreign currency hedge gains and losses. Management believes that measuring product revenue rates on a comparable pro forma basis is an appropriate way for investors to best understand the underlying performance of the business. The pro forma product revenue is presented for informational purposes only and does not purport to project product revenue for any future period or as of any future date. See "Worldwide Pro Forma Revenues" in the Quarterly Package of Financial Information for this quarter and full year of 2020, which is available on bms.com/investors/financial-reporting/quarterly-results, for information on the product revenue of the company and Celgene for the prior-year period. Otezla is a trademark of Amgen Inc.

FOURTH QUARTER PRODUCT AND PIPELINE UPDATE

Oncology

Opdivo

Regulatory

In January, the company announced that the U.S. Food & Drug Administration (FDA) approved OPDIVO(nivolumab) in combination with CABOMETYX(cabozantinib), for the first-line treatment of patients with advanced renal cell carcinoma. The approval is based on the Phase 3 Checkmate -9ER trial. (link)
In January, the company announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental Biologics License Application (sBLA) for Opdivo, in combination with fluoropyrimidine- and platinum-containing chemotherapy, for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer (GEJC) or esophageal adenocarcinoma (EAC), based on results from the CheckMate -649 trial. The U.S. FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of May 25, 2021.(link)
In January, the company announced that the U.S. FDA has accepted its supplemental sBLA for Opdivo for the treatment of patients with resected esophageal or gastroesophageal junction (GEJ) cancer in the adjuvant setting, after neoadjuvant chemoradiation therapy (CRT), based on results from the Phase 3 CheckMate -577 trial. The U.S. FDA granted the application Priority Review and assigned a PDUFA goal date of May 20, 2021.(link)
In January, the company announced that the European Medicines Agency (EMA) validated its Marketing Authorization Application (MAA) for Opdivo,based on results from the Phase 3 CheckMate -577 trial, as an adjuvant treatment for esophageal or GEJ cancer in adult patients with residual pathologic disease after neoadjuvant chemoradiotherapy (CRT) and resection. (link)
In January, the EMA validated the Type II Variation MAA for Opdivo in combination with fluoropyrimidine- and platinum-based combination chemotherapy for the first-line treatment of adult patients with advanced or metastatic gastric cancer (GC), GEJ cancer or esophageal adenocarcinoma (EAC). The filing was based on the Phase 3 CheckMate -649 trial. (link)
In November, the company announced that the European Commission (EC) has approved Opdivo for the treatment of adults with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based combination chemotherapy. (link)
In November, the company announced that the EC, based on results from the Phase 3 CheckMate -9LA trial, has approved Opdivo plus Yervoy (ipilimumab) with two cycles of platinum-based chemotherapy for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have no sensitizing epidermal growth factor receptor (EGFR) mutation or anaplastic lymphoma kinase (ALK) translocation. (link)
Clinical

In December, the company announced that CheckMate -548, a Phase 3 trial evaluating the addition of Opdivo to the current standard of care (temozolomide and radiation therapy) in patients with newly diagnosed glioblastoma multiforme (GBM) with O6-methylguanine-DNA methyltransferase (MGMT) promoter methylation following surgical resection of the tumor, did not meet its primary endpoint of overall survival (OS) in patients with no baseline corticosteroid use or in the overall randomized population. (link)
Hematology

Revlimid

Patent Update

In December, the company announced that its wholly owned subsidiary, Celgene, and Cipla Limited (Cipla) have settled their litigation related to patents for REVLIMID (lenalidomide). (link)
Inrebic

Regulatory

In December, the company announced the Committee for Medicinal Products for Human Use (CHMP) of the EMA has recommended approval of Inrebic(fedratinib) for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adult patients with primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis, who are Janus Associated Kinase(JAK) inhibitor naïve or have been treated with ruxolitinib. The CHMP recommendation will now be reviewed by the EC, which has the authority to approve medicines for the EU. (link)
Medical Conferences

In December, at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, the company announced important new data and analysis from its hematology portfolio:

QUAZAR AML-001: a study evaluating Onureg(azacitidine tablets; CC-486), an oral hypomethylating agent, as a treatment for adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or CR with incomplete blood count recovery following intensive induction chemotherapy. (link)
TRANSCEND CLL 004: longer-term follow-up from the Phase I study evaluating liso-cel in relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma with liso-cel as monotherapy and initial results from the combination cohort with ibrutinib. (link)
TRANSCEND NHL 001: safety and efficacy results in the cohort of patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL) treated with liso-cel. (link)
OUTREACH: initial results evaluating outcomes of treatment with liso-cel for patients with relapsed or refractory large B-cell lymphoma (LBCL) across inpatient and outpatient settings. (link)
First efficacy and safety results from a triplet combination study including iberdomide, a cereblon E3 ligase modulator (CELMoD) agent, with daratumumab or bortezomib and dexamethasone in patients with heavily pretreated R/R multiple myeloma. (link)
The following data were also presented at the 2020 ASH (Free ASH Whitepaper) Annual Meeting by the company and bluebird bio, Inc. (Nasdaq: BLUE):

Phase 1 CRB-401: longer-term data from the original Phase 1 CRB-401 study evaluating the companies’ investigational B-cell maturation antigen (BCMA) directed chimeric antigen receptor (CAR) T cell therapy, idecabtagene vicleucel (ide-cel) in relapsed and refractory multiple myeloma (RRMM). (link)
Phase 2 KarMMA: analyses from the Phase 2 KarMMA study of patients with triple-class exposed relapsed and refractory multiple myeloma (RMM). (link)
Immunology

Deucravacitinib (BMS-986165; TYK2 inhibitor)

Clinical

In February, the company announced results from POETYK PSO-2, the second Phase 3 trial evaluating deucravacitinib, a novel, oral, selective tyrosine kinase 2 (TYK2) inhibitor, for the treatment of patients with moderate to severe plaque psoriasis. POETYK PSO-2 met both co-primary endpoints evaluating deucravacitinib versus placebo, with significantly more patients achieving Psoriasis Area and Severity Index (PASI 75) and Physician’s Global Assessment (sPGA) scales and met multiple key secondary endpoints versus Otezla (apremilast). (link)
Zeposia

Clinical

In February, the company announced that U.S. FDA has accepted its supplemental New Drug Application (sNDA) for Zeposia for the treatment of adults with moderately to severely active ulcerative colitis (UC). The FDA granted Priority Review to the application and assigned a PDUFA goal date, or target action date, of May 30, 2021. (link)
In December, the company announced that the EMA has validated its MAA for Zeposia (ozanimod) for the treatment of adults with moderately to severely active ulcerative colitis (UC). (link)
Medical Conferences

In November, at the American College of Rheumatology (ACR) Convergence 2020, the company announced important new data and analysis across its Immunology portfolio:

deucravacitinib (BMS-986165): results from an ongoing Phase 2 study evaluating the safety and efficacy of deucravacitinib (BMS-986165) compared with placebo in adults with active psoriatic arthritis met the primary endpoint. (link)
Iberdomide: results from a Phase 2b trial in patients with active systemic lupus erythematosus (SLE) assessing iberdomide met its primary endpoint in patients with high Type 1 interferon or Aiolos gene expressions. (link)
Business Development

In November, the company announced that it has successfully completed its acquisition of MyoKardia (MyoKardia Acquisition) in an all cash transaction for approximately $13.1 billion. (link)
Capital Allocation

The company continues to maintain a consistent, balanced approach to capital allocation focused on prioritizing investment for growth through business development along with reducing debt, commitment to dividend growth and share repurchase.

Today, the company announced a debt tender offer for an aggregate purchase price of up to $4.0 billion. (link)
In January 2021, the company announced that its Board of Directors has authorized incremental share repurchases of up to an additional $2 billion of the company’s outstanding shares of common stock. With this increase, the remaining share repurchase capacity under the company’s share repurchase program was approximately $6.4 billion. During 2021, the company plans to repurchase $3.0-$4.0 billion of its shares. (link)
Commitment to Sustainability, Diversity and Inclusion

In December, the company announced it is strengthening its commitment to environmental sustainability on a global basis by setting new 2030 and 2040 goals. By 2030, the company will purchase 100% of the electricity it uses from renewable sources, and by 2040, it will be carbon neutral in its Scope 1 (direct) and Scope 2 (indirect) emissions and reach the targets of equitable water use, zero waste to landfill and 100% electric vehicles in its fleet. (link)
In November, the Bristol Myers Squibb Foundation and National Medical Fellowships announced that they will leverage $100 million of the previously announced commitment from Bristol Myers Squibb and the Bristol Myers Squibb Foundation to diversity and inclusion to develop a program to extend the reach of clinical trials into underserved patient populations in urban and rural U.S. communities. (link)
COVID-19 Pandemic Response

During the current world health crisis, the company continues to take all necessary actions to promote public health by carrying out its mission of providing life-saving medicines to the patients who depend on the company and supporting relief efforts across the globe. (link)

In February, the company and The Rockefeller University announced that they have entered into a definitive agreement under which Bristol Myers Squibb has been granted a global exclusive license to develop, manufacture, and commercialize Rockefeller’s novel monoclonal antibody ("mAb") duo treatment that neutralizes the SARS-CoV-2 virus for therapy or prevention of COVID-19. (link)
Financial Guidance

Bristol Myers Squibb is providing 2021 GAAP EPS guidance in the range of $3.12-$3.32 and is increasing its non-GAAP EPS guidance range from $7.15 – $7.45 to $7.35 – $7.55. Both GAAP and non-GAAP guidance assume current exchange rates. Key 2021 GAAP and non-GAAP line item guidance assumptions are:

Worldwide revenues increasing in the high-single digits.
Gross margin as a percentage of revenue to be approximately 80.5%.
Marketing, selling and administrative expenses to be in-line with 2020 levels for GAAP and increasing in the low-single digit range for non-GAAP.
Research and development expenses decreasing in the high-single digits for GAAP and increasing in the mid-single digits for non-GAAP.
An effective tax rate of approximately 22% for GAAP and approximately 16% for non-GAAP.
The 2021 financial guidance excludes the impact of any potential future strategic acquisitions and divestitures, and any specified items that have not yet been identified and quantified. The 2021 non-GAAP EPS guidance is explained and further excludes other specified items as discussed under "Use of Non-GAAP Financial Information." The financial guidance is subject to risks and uncertainties applicable to all forward-looking statements as described elsewhere in this press release.

Long-term Financial Targets

Bristol Myers Squibb is also affirming 2020-2025 long-term financial targets communicated in January 2021 (link):

Expects low to mid-single digit revenue CAGR and low double-digit revenue CAGR excluding Revlimid & Pomalyst at constant exchange rates
Expects to maintain low to mid-40s percent non-GAAP operating margin
Expects significant cash flow generation of $45-$50 billion dollars from 2021 -2023.
This financial guidance excludes the impact of any potential future strategic acquisitions and divestitures as well as any specified items as discussed under "Use of Non-GAAP Financial Information." There is no reliable or reasonably estimable comparable GAAP measures for this non-GAAP financial guidance. The financial guidance is subject to risks and uncertainties applicable to all forward-looking statements as described elsewhere in this press release.

Company and Conference Call Information

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook, and Instagram.

There will be a conference call on February 4 at 10 a.m. ET during which company executives will review financial information and address inquiries from investors and analysts. Investors and the general public are invited to listen to a live webcast of the call at View Source or by dialing in the U.S. toll free 800-458-4121 or international +1 313-209-6672, confirmation code: 4441406, or using this link which becomes active 15 minutes prior to the scheduled start time and entering your information to be connected. Materials related to the call will be available at the same website prior to the conference call.

A replay of the call will be available beginning at 1:30 p.m. ET on February 4 through 1:30 p.m. ET on February 18, 2021. The replay will also be available through View Source or by dialing in the U.S. toll free 888-203-1112 or international 719-457-0820, confirmation code: 4441406.

Use of Non-GAAP Financial Information

This earnings release contains non-GAAP financial measures, including non-GAAP earnings and related EPS information that are adjusted to exclude certain costs, expenses, gains and losses and other specified items that are evaluated on an individual basis. Reconciliations of these non-GAAP financial measures to the most comparable GAAP measures are provided in the accompanying financial tables and also available on the company’s website at www.bms.com.

These non-GAAP items are adjusted after considering their quantitative and qualitative aspects and typically have one or more of the following characteristics, such as being highly variable, difficult to project, unusual in nature, significant to the results of a particular period or not indicative of future operating results. Similar charges or gains were recognized in prior periods and will likely reoccur in future periods, including amortization of acquired intangible assets beginning in the fourth quarter of 2019, including product rights that generate a significant portion of our ongoing revenue, unwind of inventory fair value adjustments, acquisition and integration expenses, restructuring costs, accelerated depreciation and impairment of property, plant and equipment and intangible assets, R&D charges or other income resulting from upfront or contingent milestone payments in connection with the acquisition or licensing of third-party intellectual property rights, costs of acquiring a priority review voucher, IPRD charge resulting from the MyoKardia acquisition, divestiture gains or losses, stock compensation resulting from accelerated vesting of Celgene awards, certain retention-related employee compensation charges related to the Celgene Acquisition, pension, legal and other contractual settlement charges, interest expense on the notes issued in May 2019 incurred prior to the Celgene Acquisition and interest income earned on the net proceeds of those notes, equity investment and contingent value rights fair value adjustments and amortization of fair value adjustments of debt acquired from Celgene in our 2019 exchange offer, among other items. Deferred and current income taxes attributed to these items are also adjusted for considering their individual impact to the overall tax expense, deductibility and jurisdictional tax rates. Certain other significant tax items are also excluded such as the impact resulting from internal transfer of intangible assets and the Otezla Divestiture. This earnings release also provides international revenues excluding the impact of foreign exchange.

Non-GAAP information is intended to portray the results of the company’s baseline performance, supplement or enhance management, analysts and investors overall understanding of the company’s underlying financial performance and facilitate comparisons among current, past and future periods. For example, non-GAAP earnings and EPS information are indications of the company’s baseline performance before items that are considered by us to not be reflective of the company’s ongoing results. In addition, this information is among the primary indicators that we use as a basis for evaluating performance, allocating resources, setting incentive compensation targets and planning and forecasting for future periods. This information is not intended to be considered in isolation or as a substitute for net earnings or diluted EPS prepared in accordance with GAAP and may not be the same as or comparable to similarly titled measures presented by other companies due to possible differences in method and in the items being adjusted. We encourage investors to review our financial statements and publicly-filed reports in their entirety and not to rely on any single financial measure.

In connection with presenting our outlook, we are also providing revenue (ex-FX) and non-GAAP operating margin guidance for 2020-2025. There are no reliable or reasonably estimable comparable GAAP measures for this because we are not able to reliably predict the impact of specified items or currency exchange rates beyond the next twelve months. As a result, the reconciliation of these non-GAAP measures to the most directly comparable GAAP measures is not available without unreasonable effort. In addition, the company believes such a reconciliation would imply a degree of precision and certainty that could be confusing to investors. The variability of the specified items may have a significant and unpredictable impact on our future GAAP results.

Website Information

We routinely post important information for investors on our website, BMS.com, in the "Investors" section. We may use this website as a means of disclosing material, non-public information and for complying with our disclosure obligations under Regulation FD. Accordingly, investors should monitor the Investors section of our website, in addition to following our press releases, SEC filings, public conference calls, presentations and webcasts. We may also use social media channels to communicate with our investors and the public about our company, our products and other matters, and those communications could be deemed to be material information. The information contained on, or that may be accessed through, our website or social media channels are not incorporated by reference into, and are not a part of, this document.

On February 4, 2021 Tyme Technologies, Inc. (NASDAQ: TYME), an emerging biotechnology company developing cancer metabolism-based therapies (CMBTs), reported financial and operating results for its third quarter ended December 31, 2020 (Press release, TYME, FEB 4, 2021, View Source [SID1234574635]). During the quarter, TYME continued to build its leadership team with the announcement of Richie Cunningham as the new CEO; the Company continued to grow its global patent portfolio; expanded its body of peer-reviewed data presentations on SM-88 (racemetyrosine) at ASCO (Free ASCO Whitepaper) GI; continued enrolling patients in multiple studies including, second and third-line pancreatic cancer trials and the HopES Sarcoma Phase II trial; proceeded with next steps for initiation of the proof-of-concept trial (RESPOnD) evaluating TYME-19 as a potential new approach against COVID-19; advanced opportunities for clinical trials in metastatic breast cancer and hematological cancers.

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"TYME is at an exciting juncture in its history. As I am getting acquainted with the many facets of the Company, I am deeply impressed with the spirit of innovation and dedication towards the development of products that improve the lives of people," said Richie Cunningham, Chief Executive Officer of TYME. "I am currently fully engaged in an ongoing strategic business review with a focus on setting forth our operational priorities to determine the best path forward to maximize stakeholder value and look forward to sharing our corporate vision at the next business update."

Third Quarter Fiscal 2021 Financial Results

As of the third quarter ended December 31, 2020, the Company had approximately $13.5 million in cash and cash equivalents compared to $19.4 million as of the second quarter ended September 30, 2020. TYME’s operational cash burn rate for the third quarter of fiscal year 2021 was $5.9 million compared to $6.6 million for the second quarter of fiscal year 2021 and $4.5 million for the third quarter of fiscal year 2020. The burn rate was below current estimates and predominantly reflected expenses associated with the ongoing clinical trials in pancreatic and sarcoma cancers.

Based on active clinical trials in pancreatic and sarcoma cancers and other business developments, TYME continues to anticipate that its quarterly cash usage, or "cash burn rate", will average approximately $6.0 to $6.5 million per quarter during fiscal year 2021.

Anticipated Upcoming Key Events

At present, TYME currently expects the following key events in calendar year 2021:

Continue to advance enrollment in the HopES Sarcoma Phase II Trial; expect data readout in calendar year 2021
Present clinical data on SM-88 at a major medical meeting
Continue to advance enrollment in TYME-88-Panc pivotal study; full enrollment and data readout are not expected before calendar year 2022
Continue to advance enrollment in PanCAN’s Precision Promise℠ adaptive randomized Phase II/III registration-intent trial in patients with pancreatic cancer using oral SM-88 in second-line monotherapy
Initiate the proof-of-concept trial (RESPOnD) to evaluate TYME-19 as a potential new approach against COVID-19
Corporate Developments

On November 30, 2020, TYME announced that Richie Cunningham, former Icagen CEO and Boehringer Ingelheim executive was appointed as TYME’s Chief Executive Officer. The Company also announced that Steve Hoffman remains in the role of Chairman of the board of directors and continues as the Company’s Chief Science Officer, after his successful tenure as Chief Executive Officer since 2015.

Summary of Recent Developments

TYME Presented Data From TYME-88-Panc Study at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Gastrointestinal Cancers Symposium

On January 17, 2021, TYME presented preliminary supporting data from the TYME-88-Panc pivotal study, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual 2021 Gastrointestinal Cancers Symposium. The new data demonstrate the potential role of SM-88 (racemetyrosine) in advanced pancreatic cancer through the analysis of circulating tumor cells (CTCs) and passively acquired biometrics data from a wearable device. TYME is presently enrolling patients in a multicenter randomized controlled pivotal trial evaluating SM-88 in patients with third-line pancreatic cancer. Learn more at View Source

CTCs correlate with poor prognosis at baseline and throughout treatment may be a clinically relevant biomarker for patients with metastatic pancreatic cancer. These results demonstrated that CTC collection and enumeration were feasible for correlation with traditional trial outcomes. Furthermore, passively acquired biometrics from a wearable device can be collected for correlation with other clinically meaningful outcomes. Also, given that the longest lesion diameter was correlated with CTCs at baseline, additional radiologic feature analysis may prove to validate the importance of CTCs as a predictive biomarker for patients with pancreatic ductal adenocarcinoma.

As of September 15, 2020, the study reported that SM-88 was well tolerated with no treatment-related Grade 4 or 5 adverse advents (AEs). There was one Grade 3 AE that was deemed possibly related to SM-88. The poster presentation is available on our website at (www.tymeinc.com/data-publications).

The select data from the TYME-88-Panc trial are from an investigational study. SM-88 is not approved for the treatment of patients with any disease condition. Patients and physicians can access www.TYMETRIALS.com for more information about ongoing SM-88 clinical trials.

TYME Granted U.S. Patent Claims Covering Use of TYME-19 to Treat COVID-19 Infections

On February 3, 2021, TYME announced that it received notification that the United States Patent and Trademark Office had granted additional patent claims related to the Company’s metabolomic technology platform. U.S. Patent No. 10,905,698 is directed to methods for treating COVID-19 with TYME-19. This patent expands TYME’s patent portfolio to nearly 200 global granted and/or pending patents, broadly covering compositions, methods, manufacturing and use of the Company’s pipeline to 2032, and beyond.

TYME-19 is an investigational compound that is not approved in the U.S. for any disease indication. TYME intends to initiate the appropriate clinical trials to substantiate the safety and efficacy of TYME-19.

About SM-88

SM-88 is an oral investigational modified proprietary tyrosine derivative that is believed to interrupt the metabolic processes of cancer cells by breaking down the cells’ key defenses and leading to cell death through oxidative stress and exposure to the body’s natural immune system. Clinical trial data have shown that SM-88 has demonstrated encouraging tumor responses across 15 different cancers, including pancreatic, lung, breast, prostate and sarcoma cancers with minimal serious grade 3 or higher adverse events. SM-88 is an investigational therapy that is not approved for any indication in any disease. Learn more.

About TYME-18

TYME-18 is a CMBT compound under development that is delivered intratumorally. TYME-18 leverages a member of the bile acid family to create a potential treatment for inoperable tumors. Preliminary observations of the local administration of TYME-18, a combination of a proprietary surfactant system and natural sulfonic acid, suggested its potential as an important regulator of energy metabolism that may impede the ability of tumors to increase in size, which, in addition to its lytic functionality, could prove useful in difficult-to-treat cancers. In initial preclinical xenograft mouse studies, TYME-18 was able to completely resolve over 90 percent (11/12 mice) of established colorectal tumors within 12 days versus an average of over 600 percent growth in the control animals. Learn more.

About TYME-19

TYME-19 is an oral synthetic member of the bile acid family that the Company also uses in its anticancer compound, TYME-18. Because of its expertise in metabolic therapies, the Company was able to identify TYME-19 as a potent, well characterized antiviral bile acid and has performed preclinical experiments establishing effectiveness against COVID-19. Bile acids have primarily been used for liver disease; however, like all steroids, they are messenger molecules that modulate a number of diverse critical cellular regulators. Bile acids modulate lipid and glucose metabolism and can remediate dysregulated protein folding, with potentially therapeutic effects on cardiovascular, neurologic, immune, and other metabolic systems. Some agents in this class also have antiviral properties. In preclinical testing, TYME-19 repeatedly prevented COVID-19 viral replication without attributable cytotoxicity to the treated cells. Previous preclinical research has also shown select bile acids like TYME-19 have had broad antiviral activity.

About TYME-88-Panc Pivotal Trial

The TYME-88-Panc pivotal trial applies the latest advances in the field of cancer metabolism by evaluating the efficacy and safety of an oral investigational compound that targets the metabolic mechanisms of the disease at its source. A prospective, open label pivotal trial in metastatic pancreatic cancer for patients who have failed two lines of any prior systemic therapy. The trial is designed to evaluate the safety and efficacy of SM-88 used with MPS (methoxsalen, phenytoin and sirolimus) in advanced pancreatic cancer and will measure multiple endpoints, including overall survival, progression free survival, relevant biomarkers, quality of life, safety, and overall response rate. Learn more.