1 unchanged sentence
This discussion and analysis should be read in conjunction with our financial statements and the accompanying notes included in this report and the audited financial statements and accompanying notes included in our Annual Report on Form 10-K for the year ended December 31, 2019.
−Removed: Our financial results for the three and six months ended June 30, 2020 are not necessarily indicative of results that may occur in future interim periods or for the full fiscal year.
+Added: Our financial results for the three and nine months ended September 30, 2020 are not necessarily indicative of results that may occur in future interim periods or for the full fiscal year.
This Quarterly Report on Form 10-Q contains statements indicating expectations about future performance and other forward-looking statements within the meaning of Section 27A of the Securities Act and Section 21E of the Exchange Act, that involve risks and uncertainties.
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Our pioneering research focuses on signaling pathways that are critical to disease mechanisms.
−Removed: Our first U.S.
−Removed: Food and Drug Administration (FDA) approved product is TAVALISSE ® (fostamatinib disodium hexahydrate), the only oral spleen tyrosine kinase (SYK) inhibitor, for the treatment of adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.
−Removed: The product has also been approved by the European Commission (EC) for the treatment of chronic ITP in adult patients who are refractory to other treatments and is marketed in Europe under the name TAVLESSE ® (fostamatinib).
−Removed: Fostamatinib is currently being studied in an investigator-sponsored trial (IST) conducted by Imperial College London for the treatment of COVID-19 pneumonia.
−Removed: Our clinical programs include a Phase 3 study of fostamatinib in warm autoimmune hemolytic anemia (AIHA);
−Removed: completed Phase 1 study of R835, a proprietary molecule from our interleukin receptor associated kinase (IRAK 1/4) inhibitor program;
−Removed: and an ongoing Phase 1 study of R552, a proprietary molecule from our receptor-interacting protein kinase (RIP1) inhibitor program.
−Removed: In addition, we have product candidates in clinical development with partners BerGenBio ASA (BerGenBio), Daiichi Sankyo (Daiichi), Aclaris Therapeutics (Aclaris), and AstraZeneca AB (AZ).
+Added: Our first product approved by the United States Food and Drug Administration (FDA) is TAVALISSE ® (fostamatinib disodium hexahydrate) tablets, the only oral spleen tyrosine kinase (SYK) inhibitor, for the treatment of adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.
+Added: The product has been approved by the European Commission (EC) for the treatment of chronic ITP in adult patients who are refractory to other treatments and is marketed in Europe under the name TAVLESSE ® (fostamatinib).
+Added: Fostamatinib is currently being studied in a Phase 3 trial for the treatment of warm autoimmune hemolytic anemia (AIHA);
+Added: a NIH/NHLBI-Sponsored Phase 2 trial for the treatment of hospitalized COVID-19 patients, in collaboration with Inova ® Health System;
+Added: and a Phase 2 trial for the treatment of COVID-19 pneumonia being conducted by Imperial College London.
+Added: Additionally, we plan to study fostamatinib in a Phase 3 clinical trial for the treatment of hospitalized COVID-19 patients which is expected to launch in the fourth quarter of 2020.
+Added: Other clinical trials include an ongoing Phase 1 study of R835, a proprietary molecule from its interleukin receptor associated kinase (IRAK) inhibitor program;
+Added: and an ongoing Phase 1 study of R552, a proprietary molecule from its receptor-interacting protein kinase (RIP) inhibitor program.
+Added: In addition, we have product candidates in clinical development with partners AstraZeneca (AZ), BerGenBio ASA (BerGenBio), and Daiichi Sankyo (Daiichi).
Business Update
−Removed: In the first half of 2020, net product sales of TAVALISSE increased by 52% year over year to $27.7 million.
−Removed: During the first six months, we experienced typical first quarter reimbursement issues such as the resetting of co-pays and the Medicare donut hole, and sales were also impacted negatively by the COVID-19 pandemic as further discussed below.
+Added: In the first nine months of 2020, net product sales of TAVALISSE increased by 47% year over year to $43.9 million.
+Added: During the first nine months, our sales were negatively impacted by the COVID-19 pandemic as further discussed below.
Due to the evolving effect of the COVID-19 global pandemic, resources have been deployed to enable our field-based employees to continue to engage remotely with health care providers.
These virtual engagements have enabled our field team to support existing prescribers as well as partner with new prescribers to identify appropriate patients for TAVALISSE.
−Removed: In July 2020, we announced a Phase 2 IST with Imperial College London in order to evaluate the efficacy of fostamatinib for the treatment of COVID-19 pneumonia.
−Removed: The IST is a two-stage, open label, controlled clinical trial with patients randomized (1:1:1) to fostamatinib, ruxolitinib, or standard of care.
−Removed: Treatment will be administered twice daily for 14 days and patients will receive a follow-up assessment at day 14 and day 28 after the first dose.
−Removed: The primary objective will be to determine the efficacy of fostamatinib and the efficacy of ruxolitinib compared to standard of care to reduce the proportion of hospitalized patients progressing from mild or moderate to severe COVID-19 pneumonia.
−Removed: Recent in vitro studies led by the Amsterdam University Medical Center at the University of Amsterdam, showed that R406, the active metabolite of fostamatinib, blocked macrophage hyper-inflammatory responses to a combination of immune complexes formed by anti-Spike IgG in serum from severe COVID-19 patients.
−Removed: Anti-Spike IgG levels are known to correlate with the severity of COVID-19.
−Removed: These results suggest that by inhibiting anti-Spike IgG-mediated hyperinflammation, R406 could potentially play a role in the prevention of cytokine storms as well as pulmonary edema and thrombosis associated with severe COVID-19.
−Removed: Post-hoc data analysis from our Phase 3 clinical program, which highlights the potential benefit of using TAVALISSE in earlier lines of therapy in adult patients with chronic ITP was published in the British Journal of Haematology.
+Added: Post-hoc data analysis from our Phase 3 clinical program, which highlights the potential benefit of using TAVALISSE in earlier lines of therapy in adult patients with chronic ITP was published in the July 2020 British Journal of Haematology.
Inclusion in one of the leading peer-reviewed journals in the field of hematology underscores the significance of the 78% (25/32) response rate defined as at least one platelet count of at least 50,000/µL when TAVALISSE was used as a second-line therapy in our Phase 3 clinical program.
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Our sales force is now sharing this analysis with physicians.
+Added: We recently launched FORTE, an observational study to further evaluate fostamatinib as a second-line treatment for adult chronic ITP.
+Added: The study goal is to generate additional data on patient quality of life and financial expenditures relative to the healthcare of ITP patients.
Our FORWARD study, a pivotal Phase 3 clinical trial in warm AIHA has enrolled 57 of the 90 patients targeted for enrollment.
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We continue to evaluate enrollment timing in light of COVID-19 impacts, and at this time, we are unable to provide an update on anticipated enrollment completion.
−Removed: In June 2020, at the European League Against Rheumatism (EULAR) 2020 E-Congress, we presented two oral and two poster presentations highlighting its investigational compound R835, a potent and selective inhibitor of both IRAK1 and IRAK4.
+Added: In November 2020, we announced that we will launch a Phase 3 clinical trial to evaluate the safety and efficacy of fostamatinib in hospitalized COVID-19 patients without respiratory failure that have certain high-risk prognostic factors.
+Added: This multi-center, double-blind, placebo-controlled, adaptive design study will randomly assign either fostamatinib plus standard of care (SOC) or matched placebo plus SOC (1:1) to over 300 evaluable patients.
+Added: Treatment will be administered orally twice daily for 14 days with follow up to day 60.
+Added: The primary endpoint of this study is the proportion of subjects who progress to severe/critical disease within 29 days.
+Added: In September 2020, we announced a Phase 2 clinical trial to be sponsored by the NIH/NHLBI in order to evaluate the safety of fostamatinib for the treatment of hospitalized COVID-19 patients.
+Added: This multi-center, double-blind, placebo-controlled study will randomly assign fostamatinib or matched placebo (1:1) to approximately 60 evaluable patients.
+Added: Treatment will be administered orally twice daily for 14 days.
+Added: There will be a follow-up period to day 60.
+Added: The primary endpoint of this study is cumulative incidence of serious adverse events (SAE) through day 29.
+Added: The trial also includes multiple secondary endpoints designed to assess the early efficacy and clinically relevant endpoints of disease course.
+Added: In November 2020, we announced that the trial has enrolled 9 patients.
+Added: In July 2020, we announced a Phase 2 clinical trial sponsored by Imperial College London in order to evaluate the efficacy of fostamatinib for the treatment of COVID-19 pneumonia.
+Added: This is a two-stage, open label, controlled clinical trial with patients randomized (1:1:1) to fostamatinib plus SOC, ruxolitinib plus SOC, or standard of care alone.
+Added: Treatment will be administered twice daily for 14 days and patients will receive a follow-up assessment at day 14 and day 28 after the first dose.
+Added: The primary endpoint of this study is progression from mild to severe COVID-19 pneumonia within 14 days in hospitalized patients.
+Added: In November 2020, we announced that the Imperial College London-sponsored clinical trial began enrolling patients.
+Added: The Broad Institute of the Massachusetts Institute of Technology (MIT) and Harvard led a recent screen to identify FDA-approved compounds that reduce mucin-1 (MUC1) protein abundance.
+Added: MUC1 is a biomarker used to predict the development of ALI and ARDS and correlates with poor clinical outcomes.
+Added: Of the 3,713 compounds that were screened, fostamatinib was the only compound identified which both decreased expression of MUC1 and is FDA approved, and so allows for rapid repurposing for patients with COVID-19 lung injury.
+Added: Fostamatinib demonstrated preferential depletion of MUC1 from epithelial cells without affecting cell viability.
+Added: The research was focused on drug repurposing for the much lower risk of toxicity and the ability of FDA-approved treatments to be delivered on a shortened timescale, which is critical for patients afflicted with lung disease resulting from COVID-19.
+Added: Recent in vitro studies led by the Amsterdam University Medical Center at the University of Amsterdam, showed that R406, the active metabolite of fostamatinib, blocked macrophage hyper-inflammatory responses to a combination of immune complexes formed by anti-Spike IgG in serum from severe COVID-19 patients.
+Added: Anti-Spike IgG levels are known to correlate with the severity of COVID-19.
+Added: These results suggest that by inhibiting anti-Spike IgG-mediated hyperinflammation, R406 could potentially play a role in the prevention of cytokine storms as well as pulmonary edema and thrombosis associated with severe COVID-19.
+Added: In June 2020, at the European League Against Rheumatism (EULAR) 2020 E-Congress, we presented two oral and two poster presentations highlighting our investigational compound R835, a potent and selective inhibitor of both IRAK1 and IRAK4.
In multiple pre-clinical rodent models of acute and chronic inflammation, R835 administration resulted in reduced inflammation, and in Phase 1 trials, it showed encouraging pharmacokinetic (PK) properties.
In February 2020, we received a $20.0 million payment from Grifols.
−Removed: The payment was received upon the EC approval of the MAA for fostamatinib for the treatment of chronic ITP in adult patients who are refractory to other treatments.
+Added: The payment was received upon the EC approval of the Marketing Authorization Application (MAA) for fostamatinib for the treatment of chronic ITP in adult patients who are refractory to other treatments.
In addition, as a result of the EC approval, the $25.0 million of the $30.0 million upfront fee that we previously received from Grifols will no longer be repayable by us to Grifols.
−Removed: Fostamatinib is marketed in Europe under
−Removed: the brand name TAVLESSE™ (fostamatinib).
−Removed: Grifols launched TAVLESSE™ in the UK and Germany in July 2020, and expects to launch in Italy, Spain and France in 2021.
+Added: Fostamatinib is marketed in Europe under the brand name TAVLESSE™ (fostamatinib).
+Added: Grifols launched TAVLESSE™ in the UK and Germany in July 2020, and expects a phased roll-out over the next 18 months across Europe.
We currently anticipate no disruption related to the COVID-19 pandemic in the supply of TAVALISSE tablets and drug substance to meet the needs for our U.S.
ITP sales, as well as for our collaborative partners and clinical trials worldwide.
−Removed: With our cash and cash equivalents and short term investments as of June 30, 2020 of approximately $92.5 million and expected cash flow from operations, we believe our sources of liquidity and capital will be sufficient to finance our continued operations and growth strategy for at least the next twelve months.
+Added: With our cash and cash equivalents and short term investments as of September 30, 2020 of approximately $72.8 million and expected cash flow from operations, we believe our sources of liquidity and capital will be sufficient to finance our continued operations and growth strategy for at least the next twelve months.
In May 2020, we accessed the second $10.0 million tranche from our $60.0 million credit facility with MidCap.
1 unchanged sentence
Additionally, on August 4, 2020, we entered into an Open Market Sale Agreement SM (Sales Agreement) with Jefferies LLC (Jefferies), as our sole sales agent, pursuant to which we may sell, from time to time, through Jefferies, shares of our common stock having an aggregate offering price of up to $65.0 million.
−Removed: See “Other Information” in Item 5 of Part II of this Quarterly Report on Form 10-Q for more information.
Management Update
On August 4, 2020, we announced the appointment of David Santos as our new executive vice president and chief commercial officer.
−Removed: Santos is expected to join us on August 10, 2020, and brings over 30 years of commercial experience in the biopharmaceutical industry with companies such as Bristol-Meyers Squibb, Lilly, Genentech, and most recently Jazz Pharmaceuticals, where he led the Hematology/Oncology Business Unit.
−Removed: He has a robust track record of success in sales and marketing leadership roles, building and commercial capabilities, and growing brands in the hematology-oncology area, where he has spent most of his career.
+Added: Santos joined us on August 10, 2020, and brings over 30 years of commercial experience in the biopharmaceutical industry with companies such as Bristol-Myers Squibb, Lilly, Genentech, and most recently Jazz Pharmaceuticals, where he led the Hematology/Oncology Business Unit.
+Added: He has a robust track record of success in sales and marketing leadership roles, building commercial capabilities, and growing brands in the hematology-oncology area, where he has spent most of his career.
+Added: On October 2, 2020, Nelson D.
+Added: Cabatuan, delivered notice of his resignation from his position as Vice President, Finance and Principal Accounting Officer, and employment with the Company, to be effective on October 16, 2020.
Update on Current and Potential Future Impact of COVID-19 on our Business
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As of the date of this filing, significant uncertainty exists concerning the duration and severity of the COVID-19 pandemic.
−Removed: We have undertaken, and plan to continue to undertake, safety measures to keep our staff, patients, investigators and stockholderssafe and to help the communities where we live and work reduce the number of people exposed to the virus.
−Removed: We have previously implemented work-from-home policies for certain employees and closed our office in South San Francisco requiring most of our personnel, including our administrative employees to work remotely, restricted on-site staff to only those personnel performing essential activities.
+Added: We have undertaken, and plan to continue to undertake, safety measures to keep our staff, patients, investigators and stockholders safe and to help the communities where we live and work reduce the number of people exposed to the virus.
+Added: We have previously implemented work-from-home policies for certain employees and restricted on-site staff at our office in South San Francisco to only those personnel performing essential activities.
In March 2020, through our existing Crisis Management Team (CMT), we also activated our business continuity plans to prevent or minimize business disruption and ensure the safety and well-being of our personnel.
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Resources have been deployed to enable our field team to have virtual engagements to support existing prescribers as well as partner with new prescribers to identify appropriate patients for TAVALISSE.
−Removed: As such, our field- based employees are continuing to engage remotely with health care providers.
+Added: As such, our field-based employees primarily engage remotely with health care providers.
Other commercial related activities, such as our marketing programs, speaker bureaus, and market access initiatives that were in live forums have been conducted virtually, delayed or cancelled as a result of the COVID-19 pandemic.
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We have a number of ongoing clinical trials, one of which is a global Phase 3 clinical study in warm AIHA.
−Removed: A number of our clinical trial investigators have paused, postponed or delayed new patient enrollment and restricted site visits of existing patients enrolled, but since May 2020, some have resumed patient screening .
+Added: A number of our clinical trial investigators had paused, postponed or delayed new patient enrollment and restricted site visits of existing patients enrolled, but since May 2020, trial sites have resumed screening and enrolling patients .
We are making decisions country-by-country to minimize risk to the patients and clinical trial sites.
2 unchanged sentences
At this time, however, we cannot currently fully forecast the scope of impacts that the COVID-19 pandemic may have on our ability to continue to treat patients enrolled in our trials, enroll and assess new patients, supply study drug, obtain complete data points in accordance with study protocol and overall impact on clinical study results including the timing thereof.
−Removed: In addition, our partner, Kissei, is currently conducting a Phase 3 clinical trial for fostamatinib in ITP in Japan the timing and completion of which could be delayed due to the COVID-19 pandemic.
+Added: In addition, our partner, Kissei, is currently conducting a Phase 3 clinical trial
+Added: for fostamatinib in ITP in Japan the timing and completion of which could be delayed due to the COVID-19 pandemic.
The delays may potentially delay future royalties on sales, as well as, receipt of future potential milestones.
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Taken in tablet form, fostamatinib blocks the activation of SYK inside immune cells.
−Removed: ITP is typically characterized by the body producing antibodies that attach to healthy platelets in the blood stream.
+Added: ITP is typically characterized by the body producing antibodies that attach to healthy platelets in the blood
Immune cells recognize these antibodies and affix to them, which activates the SYK enzyme inside the immune cell, and triggers the destruction of the antibody and the attached platelet.
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The most frequent adverse events were gastrointestinal-related, and the safety profile of the product was consistent with prior clinical experience, with no new or unusual safety issues uncovered.
+Added: A paper presenting this data analysis, as well as accompanying commentary have been published in the July 2020 British Journal of Haematology.
+Added: We recently launched FORTE, an observational study to further evaluate fostamatinib as a second-line treatment for adult chronic ITP.
+Added: The study goal is to generate additional data on patient quality of life and financial expenditures relative to the healthcare of ITP patients.
TAVALISSE was approved by the FDA in April 2018 for the treatment of chronic ITP in adult patients who have had an insufficient response to a previous treatment, and successfully launched in the U.S.
In January 2020, the EC granted our MAA in Europe for fostamatinib for the treatment of chronic ITP in adult patients who are refractory to other treatments.
−Removed: Grifols launched TAVLESSE TM in the UK and Germany in July 2020 and expects to launch in Italy, Spain and France in 2021.
+Added: Grifols launched TAVLESSE TM in the UK and Germany in July 2020, and expects a phased roll-out over the next 18 months across Europe .
Commercial launch activities, including sales and marketing
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To facilitate our commercial activities in the U.S., we also enter into arrangements with various third-parties, including advertising agencies, market research firms and other sales-support-related services as needed.
−Removed: We believe that our
−Removed: commercial team and distribution practices are adequate to ensure that our marketing efforts reach our target customers and deliver our products to patients in a timely and compliant fashion.
+Added: We believe that our commercial team and distribution practices are adequate to ensure that our marketing efforts reach our target customers and deliver our products to patients in a timely and compliant fashion.
Also, to help ensure that all eligible patients in the U.S.
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We are responsible for performing and funding certain development activities for fostamatinib for ITP and AIHA and Grifols is responsible for all other development activities for fostamatinib in such territories.
−Removed: We remain responsible for the manufacture and supply of fostamatinib for all development and commercialization activities under
−Removed: the agreement.
+Added: We remain responsible for the manufacture and supply of fostamatinib for all development and commercialization activities under the agreement.
In December 2019, we entered into a Drug Product Purchase Agreement with Grifols wherein we agreed to supply and sell to Grifols the drug product requested under an executed first and only purchase order until Grifols enters into a supply agreement directly with a third-party drug product manufacturer.
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With this approval, we received a $20.0 million payment as described above.
−Removed: Grifols launched TAVLESSE TM in the UK and Germany in July 2020 and expects to launch in Italy, Spain and France in 2021.
+Added: Grifols launched TAVLESSE TM in the UK and Germany in July 2020, and expects a phased roll-out over the next 18 months across Europe.
Fostamatinib in Japan/Asia
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The buyback provision if exercised would require both parties to mutually agree on commercially reasonable terms for us to purchase back the rights, taking into account Medison’s investment and the value of the rights, among others.
+Added: Pursuant to this exclusive commercialization license agreement, in August 2020, we entered into a commercial supply agreement with Medison.
Clinical Stage Programs
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AIHA is a rare, serious blood disorder where the immune system produces antibodies that result in the destruction of the body's own red blood cells.
−Removed: Symptoms can include fatigue, shortness of breath, rapid
−Removed: heartbeat, jaundice or enlarged spleen.
+Added: Symptoms can include fatigue, shortness of breath, rapid heartbeat, jaundice or enlarged spleen.
While no medical treatments are currently approved for AIHA, physicians generally treat acute and chronic cases of the disorder with corticosteroids, other immuno-suppressants, or splenectomy.
12 unchanged sentences
In May 2019, we enrolled the first patient in the FORWARD study.
−Removed: We have enrolled 44 patients of the 90 patients targeted for enrollment.
−Removed: Currently, the FORWARD study has over 90 active clinical trial sites established across 22 countries and a number of clinical trial sites have resumed screening patients after a temporary pause due to the ongoing COVID-19 pandemic.
+Added: Currently, we have enrolled 57 patients of the 90 patients targeted for enrollment.
+Added: The FORWARD study has over 90 active clinical trial sites established across 22 countries and a number of clinical trial sites have resumed screening patients after a temporary pause due to the ongoing COVID-19 pandemic.
Given the uncertainty of the COVID-19 pandemic, we are unable to provide an update on anticipated enrollment completion.
In January 2018, the FDA granted our request for Orphan Drug designation for fostamatinib for the treatment of AIHA.
+Added: Fostamatinib—in Hospitalized COVID-19 patients
+Added: Disease background.
+Added: COVID-19 is the infectious disease caused by Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2).
+Added: SARS-CoV-2 primarily infects the upper and lower respiratory tract and can lead to acute respiratory distress syndrome (ARDS).
+Added: Additionally, some patients develop other organ dysfunction including myocardial injury, acute kidney injury, shock resulting in endothelial dysfunction and subsequently micro and macrovascular thrombosis.
+Added: Much of the underlying pathology of SARS-CoV-2 is thought to be secondary to a hyperinflammatory immune response associated with increased risk of thrombosis.
+Added: SYK is involved in the intracellular signaling pathways of many different immune cells.
+Added: Therefore, SYK inhibition may improve outcomes in patients with COVID-19 via inhibition of key Fc gamma receptor (FcγR) and c-type lectin receptor (CLR) mediated drivers of pathology such as inflammatory cytokine release by monocytes and macrophages, production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation.
+Added: Furthermore, SYK inhibition in neutrophils and platelets may lead to decreased thromboinflammation, alleviating organ dysfunction in critically ill patients with COVID-19.
+Added: Orally available fostamatinib program.
+Added: In November 2020, we announced that we will launch a Phase 3 clinical trial to evaluate the safety and efficacy of fostamatinib in hospitalized COVID-19 patients without respiratory failure that have certain high-risk prognostic factors.
+Added: This multi-center, double-blind, placebo-controlled, adaptive design study will randomly assign either fostamatinib plus standard of care (SOC) or matched placebo plus SOC (1:1) to over 300 evaluable patients.
+Added: Treatment will be administered orally twice daily for 14 days with follow up to day 60.
+Added: The primary endpoint of this study is the proportion of subjects who progress to severe/critical disease within 29 days.
+Added: In September 2020, we announced a Phase 2 clinical trial to be sponsored by the NIH/NHLBI in order to evaluate the safety of fostamatinib for the treatment of hospitalized COVID-19 patients.
+Added: This multi-center, double-blind, placebo-controlled study will randomly assign fostamatinib or matched placebo (1:1) to approximately 60 evaluable patients.
+Added: Treatment will be administered orally twice daily for 14 days.
+Added: There will be a follow-up period to day 60.
+Added: The primary endpoint of this study is cumulative incidence of serious adverse events (SAE) through day 29.
+Added: The trial also includes multiple secondary endpoints designed to assess the early efficacy and clinically relevant endpoints of disease course.
+Added: In November 2020, we announced that the trial has enrolled 9 patients.
+Added: In July 2020, we announced a Phase 2 clinical trial sponsored by Imperial College London in order to evaluate the efficacy of fostamatinib for the treatment of COVID-19 pneumonia.
+Added: This is a two-stage, open label, controlled clinical trial with patients randomized (1:1:1) to fostamatinib plus SOC, ruxolitinib plus SOC, or standard of care alone.
+Added: Treatment will be administered twice daily for 14 days and patients will receive a follow-up assessment at day 14 and day 28 after the first dose.
+Added: The primary endpoint of this study is progression from mild to severe COVID-19 pneumonia within 14 days in hospitalized patients.
+Added: In November 2020, we announced that the Imperial College London-sponsored clinical trial began enrolling patients.
+Added: Researchers at MIT and Harvard led a recent screen to identify FDA-approved compounds that reduce mucin-1 (MUC1) protein abundance.
+Added: MUC1 is a biomarker used to predict the development of ALI and ARDS and correlates with poor clinical outcomes.
+Added: Of the 3,713 compounds that were screened, fostamatinib was the only compound identified which both decreased expression of MUC1 and is FDA approved.
+Added: Fostamatinib demonstrated preferential depletion of MUC1 from epithelial cells without affecting cell viability.
+Added: The research was focused on drug repurposing for the much lower risk of toxicity and the ability of FDA-approved treatments to be delivered on a shortened timescale, which is critical for patients afflicted with lung disease resulting from COVID-19.
+Added: In addition, recent in vitro studies led by the Amsterdam University Medical Center at the University of Amsterdam, showed that R406, the active metabolite of fostamatinib, blocked macrophage hyperinflammatory responses to a combination of immune complexes formed by anti-Spike IgG in serum from severe COVID-19 patients.
+Added: Anti-Spike IgG levels are known to correlate with the severity of COVID-19.
+Added: These results suggest that by inhibiting anti-Spike
+Added: IgG-mediated hyperinflammation, R406 could potentially play a role in the prevention of cytokine storms as well as pulmonary edema and thrombosis associated with severe COVID-19.
R835, an IRAK1/4 Inhibitor for Autoimmune and Inflammatory Diseases
18 unchanged sentences
In addition, we intend to search for a central nervous system molecule to potentially advance into the clinic.
−Removed: Investigator-Sponsored Clinical Program
−Removed: Fostamatinib—COVID-19 Pneumonia
−Removed: In July 2020, we announced a Phase 2 IST with Imperial College London to evaluate the efficacy of fostamatinib, our oral SYK inhibitor, for the treatment of COVID-19 pneumonia.
−Removed: SYK is a key mediator of immunoreceptor signaling in a host of inflammatory cells.
−Removed: Studies of severe acute respiratory syndrome (SARS) and other acute viral respiratory infections suggest that the pathogenesis relies on a series of SYK-dependent events involving activation of C-type lectin receptors (CLR) and immunoglobulin Fcg receptors (FcgR) in multiple cell types.
−Removed: Such SYK-mediated processes result in excessive cytokine and chemokine release, neutrophil activation associated with extensive NETosis (a highly inflammatory and thrombogenic type of cell death), and endothelial cell stimulation leading to vascular endothelium leakage and edema in the lungs.
−Removed: Together, these events can contribute to acute respiratory distress syndrome (ARDS), micro-thrombosis and associated systemic complications.
−Removed: A hallmark of severe COVID-19 are hypoxemia and a radiological pattern of acute lung injury (ALI) that share features with ARDS.
−Removed: By inhibiting SYK, fostamatinib may specifically inhibit the infiltration and activation of monocytes and neutrophils in the lungs that are prominent in COVID-19.
−Removed: Recent in vitro studies led by the Amsterdam University Medical Center at the University of Amsterdam, showed that R406, the active metabolite of fostamatinib, blocked macrophage hyper-inflammatory responses to a combination of immune complexes formed by anti-Spike IgG in serum from severe COVID-19 patients.
−Removed: Anti-Spike IgG levels are known to correlate with the severity of COVID-19.
−Removed: These results suggest that by inhibiting anti-Spike IgG-mediated hyperinflammation, R406 could potentially play a role in the prevention of cytokine storms, as well as pulmonary edema and thrombosis associated with severe COVID-19.
−Removed: In addition, researchers at The Broad Institute of the Massachusetts Institute of Technology (MIT) and Harvard led a recent screen to identify FDA-approved compounds that reduce mucin-1 (MUC1) protein abundance.
−Removed: MUC1 is a biomarker used to predict the development of ALI and ARDS and correlates with poor clinical outcomes.
−Removed: Of the 3,713 compounds that were screened, fostamatinib was the only compound identified which both decreased expression of MUC1 and is FDA approved, and so allows for rapid repurposing for patients with COVID-19 lung injury.
−Removed: Fostamatinib demonstrated preferential depletion of MUC1 from epithelial cells without affecting cell viability.
−Removed: The research was focused on drug repurposing for the much lower risk of toxicity and the ability of FDA-approved treatments to be delivered on a shortened timescale, which is critical for patients afflicted with lung disease resulting from COVID-19.
−Removed: The IST will be a two-stage open label, controlled clinical trial with patients randomized (1:1:1) to fostamatinib, ruxolitinib, or standard of care.
−Removed: Treatment will be administered twice daily for 14 days and patients will receive a follow-up assessment at day 14 and day 28 after the first dose.
−Removed: The primary objective will be to determine the efficacy of fostamatinib and the efficacy of ruxolitinib compared to standard of care to reduce the proportion of hospitalized patients progressing from mild or moderate to severe COVID-19 pneumonia.
−Removed: We will provide support for this trial along with Novartis.
Partnered Clinical Programs
27 unchanged sentences
In June 2020, BerGenBio confirmed dosing the first COVID-19 patient with bemcentinib at the University Hospital Southampton NHS Foundation Trust.
−Removed: The Phase 2 trial has commenced in seven more sites across the UK, with the plan to recruit approximately 120 subjects to assess safety and efficacy of bemcentinib as an add-on therapy to standard of care in approximately 60 hospitalized COVID-19 patients with the other approximately 60 control group
−Removed: patients receiving standard of care.
+Added: The Phase 2 trial has commenced in seven more sites across the UK, with the plan to recruit approximately 120 subjects to assess safety and efficacy of bemcentinib as an add-on therapy to standard of care in approximately 60 hospitalized COVID-19 patients with the other approximately 60 control group patients receiving standard of care.
Bemcentinib has exhibited potent anti-viral activity in preclinical models against several enveloped viruses, including Ebola and Zika virus and as of recently, to the COVID-19 virus.
6 unchanged sentences
This is an open label, multi-center, intra-tumoral tissue PK study of bemcentinib in patients with recurrent GBM for whom a surgical resection is medically indicated.
−Removed: The trial intends to enroll up to 20 recurrent GBM patients, at up to 15 sites in the U.S.
+Added: The trial intends to enroll up to 20 recurrent GBM
+Added: patients, at up to 15 sites in the U.S.
The end points of the study include an evaluation of bemcentinib’s ability to cross the blood brain barrier, AXL expression, PK, safety and tolerability, as well as efficacy assessments including PFS and Overall Survival.
+Added: In October 2020, BerGenBio announced first patient enrolled in Phase II trial assessing bemcentinib as a potential treatment for COVID-19 patients in South Africa.
DS-3032 - Daiichi
2 unchanged sentences
Evaluation of additional dosing schedules of DS-3032 is underway and combination studies with fostamatinib are currently being conducted by Daiichi.
+Added: For the three and nine months ended September 2020, we recognized $2.1 million of revenue as a result of the achievement of a milestone in accordance with the Amended Collaboration Agreement dated April 20, 2005 with Daiichi.
+Added: All deliverables under the agreement had been previously delivered, as such the above payment has been recognized as revenue in the third quarter of 2020.
+Added: We received the milestone payment from Daiichi in October 2020.
AZ-D0449 – AZ
6 unchanged sentences
We conduct research and development programs independently and in connection with our corporate collaborators.
−Removed: As of June 30, 2020, we are a party to collaboration agreements with ongoing performance obligations with Kissei for the development and commercialization of fostamatinib in Japan, China, Taiwan and the Republic of Korea and with Grifols to commercialize fostamatinib in all indications, including chronic ITP and AIHA, in Europe and Turkey and with Medison Pharma Ltd.
+Added: As of September 30, 2020, we are a party to collaboration agreements with ongoing performance obligations with Kissei for the development and commercialization of fostamatinib in Japan, China, Taiwan and the Republic of Korea and with Grifols to commercialize fostamatinib in all indications, including chronic ITP and AIHA, in Europe and Turkey and with Medison Pharma Ltd.
(Medison) to commercialize fostamatinib in all indications, including chronic ITP and AIHA in Canada and Israel.
−Removed: As of June 30, 2020, we are also a party to collaboration agreements, but do not have ongoing performance obligations, with Aclaris for the development and commercialization of JAK inhibitors for the treatment of alopecia areata and other dermatological conditions, AZ for the development and commercialization of
−Removed: R256, an inhaled JAK inhibitor, BerGenBio for the development and commercialization of AXL inhibitors in oncology, and Daiichi to pursue research related to MDM2 inhibitors, a novel class of drug targets called ligases.
+Added: As of September 30, 2020, we are also a party to collaboration agreements, but do not have ongoing performance obligations, with Aclaris for the development and commercialization of JAK inhibitors for the treatment of alopecia areata and other dermatological conditions, AZ for the development and commercialization of R256, an inhaled JAK inhibitor, BerGenBio for the development and commercialization of AXL inhibitors in oncology, and Daiichi to pursue research related to MDM2 inhibitors, a novel class of drug targets called ligases.
Under these agreements, which we entered into in the ordinary course of business, we received or may be entitled to receive upfront cash payments, payments contingent upon specified events achieved by such partners and royalties on any net sales of products sold by such partners under the agreements.
−Removed: Total future contingent payments to us under all of these agreements could exceed $610.7 million if all potential product candidates achieved all of the payment triggering events under all of our current agreements (based on a single product candidate under each agreement).
+Added: Total future contingent payments to us under all of these agreements could exceed $607.2 million if all potential product candidates achieved all of the payment
+Added: triggering events under all of our current agreements (based on a single product candidate under each agreement).
Of this amount, up to $67.5 million relates to the achievement of development events, up to $163.7 million relates to the achievement of regulatory events and up to $376.0 million relates to the achievement of certain commercial or launch events.
3 unchanged sentences
Due to the COVID-19 pandemic, the commercial launch of fostamatinib in Europe by our partner, Grifols, was delayed and undertaken in a virtual manner.
−Removed: Grifols expects to launch in Italy, Spain and France in 2021.
+Added: Grifols expects a phased roll-out over the next 18 months across Europe.
In addition, our partner, Kissei is currently conducting a Phase 3 clinical trial for fostamatinib in ITP in Japan the timing and completion of which could be delayed due to the COVID-19 pandemic.
8 unchanged sentences
With this approval, we received in February 2020 a $20.0 million non-refundable payment, which is comprised of a $17.5 million payment for EMA approval of fostamatinib for the first indication and a $2.5 million creditable advance royalty payment, based on the terms of our collaboration agreement with Grifols.
−Removed: The above milestone payment will be allocated to the distinct performance obligation in the collaboration agreement with Grifols.
+Added: The above milestone payment was allocated to the distinct performance obligations in the collaboration agreement with Grifols.
We accounted for this agreement under ASC 606 and identified the following distinct performance obligations at inception of the agreement:
(a) granting of the license, (b) performance of research and regulatory services related to our ongoing long-term open-label extension study on patients with ITP, and (c) performance of research services related to our Phase 3 study in AIHA.
−Removed: In addition, we will enter into a commercial supply agreement for the licensed territories.
+Added: In October 2020, we entered into a commercial supply agreement for the licensed territories.
We concluded each of these performance obligations is distinct.
4 unchanged sentences
We allocated the updated transaction price to the distinct performance obligations in our collaboration agreement based on our best estimate of the relative standalone selling price as follows:
−Removed: (a) for the license, we estimated the standalone selling price using the adjusted market assessment approach to estimate its standalone selling price in the licensed
+Added: (a) for the license, we estimated the standalone selling price using the adjusted market assessment approach to estimate its standalone selling price in the licensed territories;
(b) for the research and regulatory services, we estimated the standalone selling price using the cost plus expected margin approach.
1 unchanged sentence
The remaining future variable consideration of $277.5 million related to future regulatory and commercial milestones were fully constrained due to the fact that it was probable that a significant reversal of cumulative revenue would occur, given the inherent uncertainty of success with these future milestones.
−Removed: We will recognize revenues related the research and regulatory services throughout the term of the respective clinical programs using the input method.
+Added: We will recognize revenues related
+Added: the research and regulatory services throughout the term of the respective clinical programs using the input method.
For sales-based milestones and royalties, we determined that the license is the predominant item to which the royalties or sales-based milestones relate.
1 unchanged sentence
We will re-evaluate the transaction price in each reporting period and as uncertain events are resolved or other changes in circumstances occur.
−Removed: During the three ended June 30, 2020, we recognized no revenues related to the licensed rights in intellectual property and $396,000 in revenues related to the research services performed.
−Removed: During the six months ended June 30, 2020, we recognized $39.9 million in revenues related to the licensed rights in intellectual property and $3.6 million in revenues related to the research services performed.
−Removed: Deferred revenues as of June 30, 2020 was $1.8 million.
−Removed: During the three and six months ended June 30, 2020, we also recognized $651,000 in revenues for a one-time delivery of drug supply to Grifols for commercialization.
+Added: During the three months ended September 30, 2020, we recognized no revenues related to the licensed rights in intellectual property and the research services performed.
+Added: During the nine months ended September 30, 2020, we recognized $39.9 million in revenues related to the licensed rights in intellectual property and $3.6 million in revenues related to the research services performed.
+Added: Deferred revenues related to the performance of research services as of September 30, 2020 was $1.8 million.
+Added: During the nine months ended September 30, 2020, we also recognized $651,000 in revenues for a one-time delivery of drug supply to Grifols for commercialization.
Kissei License Agreement
16 unchanged sentences
For sales-based milestones and royalties, we determined that the license is the predominant item to which the royalties or sales-based milestones relate to.
−Removed: Accordingly, we will recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of
−Removed: the royalty has been allocated has been satisfied (or partially satisfied).
+Added: Accordingly, we will recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied).
We will re-evaluate the transaction price in each reporting period and as uncertain events are resolved or other changes in circumstances occur.
−Removed: We did not recognize any revenues during the three and six months ended June 30, 2020.
−Removed: At June 30, 2020, deferred revenues related to the unsatisfied performance obligations related to the supply of fostamatinib and material right associated with discounted fostamatinib supply was $1.4 million.
−Removed: Other license agreements
−Removed: As of June 30, 2020, we have accounts receivable of $500,000 relative to the first amendment to the license and collaboration agreement with Aclaris executed in the fourth quarter of 2019.
+Added: We did not recognize any revenues during the three and nine months ended September 30, 2020.
+Added: At September 30, 2020, deferred revenues related to the unsatisfied performance obligations related to the supply of fostamatinib and material right associated with discounted fostamatinib supply was $1.4 million.
+Added: Medison Commercial and License Agreements
In October 2019, we entered into two exclusive commercial and license agreements with Medison for the commercialization of fostamatinib for chronic ITP in Israel and in Canada pursuant to which we received a $5.0 million upfront payment under our agreement in Canada.
6 unchanged sentences
As such this arrangement is accounted for as a financing arrangement.
−Removed: Accrued interest related to this financing arrangement as of June 30, 2020 is immaterial.
+Added: Accrued interest related to this financing arrangement as of September 30, 2020 is immaterial.
+Added: Other license agreements
+Added: For the three and nine months ended September 2020, we recognized $2.1 million of revenue as a result of the achievement of a milestone in accordance with the Amended Collaboration Agreement dated April 20, 2005 with Daiichi.
+Added: All deliverables under the agreement had been previously delivered, as such the above payment has been recognized as revenue in the third quarter of 2020.
+Added: We received the milestone payment from Daiichi in October 2020.
Results of Operations
−Removed: Three and Six Months Ended June 30, 2020 and 2019
+Added: Three and Nine Months Ended September 30, 2020 and 2019
Three Months Ended
−Removed: Six Months Ended
+Added: Nine Months Ended
+Added: September 30,
+Added: September 30,
(in thousands)
3 unchanged sentences
Total revenues
−Removed: The following table summarizes revenues from each of our customers and collaboration partners who individually accounted for 10% or more of our total revenues for the three and six months ended June 30, 2020 and 2019 (as a percentage of total revenues):
+Added: The following table summarizes revenues from each of our customers and collaboration partners who individually accounted for 10% or more (wherein * denotes less than 10%) of our total revenues for the three and nine months ended September 30, 2020 and 2019 (as a percentage of total revenues):
Three Months Ended
−Removed: Six Months Ended
+Added: Nine Months Ended
+Added: September 30,
+Added: September 30,
ASD Healthcare and Oncology Supply
McKesson Specialty Care Distribution Corporation
−Removed: Cardinal Healthcare
−Removed: Product sales during the three and six months ended June 30, 2020 and 2019 related to sales of TAVALISSE in the U.S.
+Added: Product sales during the three and nine months ended September 30, 2020 and 2019 related to sales of TAVALISSE in the U.S.
and represent increasing sales volume since we launched in May 2018.
−Removed: For the three and six months ended June 30, 2020, the increase in product sales was mainly due to TAVALISSE sales volume increases of 28% and 32%, respectively, compared to the same periods in 2019, as well as increases in the selling price of TAVALISSE.
+Added: For the three and nine months ended September 30, 2020, the increase in product sales was mainly due to TAVALISSE sales volume increases of 27% and 30%, respectively, compared to the same periods in 2019, as well as increases in the selling price of TAVALISSE.
TAVALISSE has been prescribed across all lines of therapy in steroid refractory patients in ITP.
−Removed: It has been utilized by an increasing broad base of prescribers and community physicians, with growing early line use and continued strong refill rates.
+Added: been utilized by an increasing broad base of prescribers and community physicians, with growing early line use and continued strong refill rates.
We recognize product sales, net of discounts and allowances, as described in “Note 3” to our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q.
−Removed: Contract revenues from collaborations of $1.0 million and $44.1 million, respectively, in the three and six months ended June 30, 2020 relate to revenue from the upfront fee we previously received from Grifols in the first quarter of 2019, as well as the milestone payment received from Grifols in the first quarter of 2020 upon EC approval of the MAA for fostamatinib in Europe.
−Removed: For the same periods in 2019, we recognized contract revenues of $234,000 and $4.8 million primarily related to the portion of the upfront fees from our collaboration agreements with Grifols and Kissei, respectively, recognized as revenue upon our performance of certain research and development services.
+Added: Contract revenues from collaborations of $2.1 million in the three months ended September 30, 2020 relate to a milestone payment under our collaboration agreement with Daiichi.
+Added: Contract revenues from collaborations of $46.2 million in the nine months ended September 30, 2020 relate to revenue from the upfront fee previously received from Grifols in the first quarter of 2019 as well as the milestone payment received from Grifols in the first quarter of 2020 upon EC approval of the MAA for fostamatinib in Europe and the milestone payment under our collaboration agreement with Daiichi.
+Added: For the same periods in 2019, we recognized contract revenues of $9.1 million and $13.9 million primarily related to the license and milestone revenues from our collaboration agreements with Aclaris and Celgene as well as portions of the upfront fees from our collaboration agreements with Grifols and Kissei recognized as revenue from licenses and upon our performance of certain research and development services and supply of fostamatinib.
Our potential future revenues may include product sales from TAVALISSE, payments from our current partners and from new partners with whom we enter into agreements in the future, if any, the timing and amount of which is unknown at this time.
W e cannot currently fully forecast the extent of the impacts that the COVID-19 pandemic may have on our product sales.
−Removed: As of June 30, 2020, we had deferred revenues of $3.2 million which we will recognize as revenue upon satisfaction of our remaining performance obligations under our collaboration agreements with Grifols and Kissei.
+Added: As of September 30, 2020, we had deferred revenues of $3.2 million which we will recognize as revenue upon satisfaction of our remaining performance obligations under our collaboration agreements with Grifols and Kissei.
Cost of Product Sales
Three Months Ended
−Removed: Six Months Ended
+Added: Nine Months Ended
+Added: September 30,
+Added: September 30,
(in thousands)
1 unchanged sentence
Cost of product sales
−Removed: We recognized $279,000 and $434,000, respectively, in cost of product sales during the three and six months ended June 30, 2020 related to our product, TAVALISSE.
+Added: We recognized $140,000 and $574,000 in cost of product sales during the three and nine months ended September 30, 2020 related to our product, TAVALISSE.
Prior to the FDA approval, manufacturing and related costs were charged to research and development expense.
−Removed: Therefore, these costs were not capitalized and as a result, are not fully reflected in the costs of product sales during the three and six months ended June 30, 2020 and 2019.
+Added: Therefore, these costs were not capitalized and as a result, are not fully reflected in the costs of product sales during the three and nine months ended September 30, 2020 and 2019.
We will continue to have a lower cost of product sales that excludes the cost of the active pharmaceutical ingredient (API) that was produced prior to FDA approval until we sell TAVALISSE that includes newly manufactured API.
3 unchanged sentences
Three Months Ended
−Removed: Six Months Ended
+Added: Nine Months Ended
+Added: September 30,
+Added: September 30,
(in thousands)
2 unchanged sentences
Stock-based compensation expense included in research and development expense
−Removed: The increase in research and development expense for the three months ended June 30, 2020, compared to the same period in 2019, was primarily due to an increase of $2.1 million in research and development costs mainly for our on-going Phase 3 study in warm AIHA, Phase 1 trial of our RIP1 inhibitor program and Phase 1 trial in our IRAK 1/4
−Removed: inhibitor program partially offset by the decreases of $453,000 in stock-based compensation expense, $365,000 in research and laboratory supplies, $164,000 in personnel-related expenses and $130,000 in various third party costs.
−Removed: The increase in research and development expense for the six months ended June 30, 2020, compared to the same period in 2019, was primarily due to an increase of $7.1 million in research and development cost for our on-going Phase 3 trial in warm AIHA, Phase 1 trial of our RIP1 inhibitor program and Phase 1 trial in our IRAK 1/4 inhibitor program partially offset by the decreases of $546,000 in stock-based compensation expense and $366,000 in research and laboratory supplies.
+Added: The increase in research and development expense for the three months ended September 30, 2020, compared to the same period in 2019, was primarily due to the increases of $235,000 in personnel-related expenses and $161,000 in
+Added: third-party costs partially offset by the decrease of $259,000 in research and development costs mainly for the Phase 1 trial of our RIP1 inhibitor program and Phase 1 trial in our IRAK 1/4 inhibitor program.
+Added: The increase in research and development expense for the nine months ended September 30, 2020, compared to the same period in 2019, was primarily due to the increases of $6.7 million in research and development cost for our on-going Phase 3 trial in warm AIHA, Phase 1 trial in our RIP 1 inhibitor program and Phase 1 trial in our IRAK 1/4 inhibitor program and $275,000 in personnel-related expenses partially offset by the decreases of $500,000 in stock-based compensation expense and $150,000 in third-party costs.
We expect our research and development expense for the remainder of 2020 to increase as we continue our activities in our Phase 3 warm AIHA studies, RIP1 and IRAK 1/4 programs and other fostamatinib programs.
25 unchanged sentences
Three Months Ended
−Removed: Six Months Ended
+Added: Nine Months Ended
+Added: September 30,
+Added: September 30,
From January 1, 2007*
−Removed: to June 30, 2020
+Added: to September 30, 2020
* We started tracking research and development expense by category on January 1, 2007.
−Removed: “Other” expenses mainly represent allocated facilities costs of approximately $1.5 million each for the three months ended June 30, 2020 and 2019 and allocated stock-based compensation expense of approximately $458,000 and $911,000 for the three months ended June 30, 2020 and 2019, respectively.
−Removed: For the six months ended June 30, 2020 and 2019, allocated facilities costs were approximately $3.0 million and $3.1 million, respectively, and allocated stock-based compensation expense were approximately $1.2 million and $1.7 million, respectively.
−Removed: For the three and six months ended June 30, 2020 and 2019, a major portion of our total research and development expense was associated with our AIHA, RIP1, and IRAK programs, salaries of our research and development personnel and allocated facilities costs.
+Added: “Other” expenses mainly represent allocated facilities costs of approximately $1.5 million each for the three months ended September 30, 2020 and 2019 and allocated stock-based compensation expense of approximately $532,000 and $487,000 for the three months ended September 30, 2020 and 2019, respectively.
+Added: For the nine months ended September 30, 2020 and 2019, allocated facilities costs were approximately $4.5 million and $4.6 million, respectively, and allocated stock-based compensation expense were approximately $1.7 million and $2.1 million, respectively.
+Added: For the three and nine months ended September 30, 2020 and 2019, a major portion of our total research and development expense was associated with our AIHA, RIP1, and IRAK programs, salaries of our research and development personnel and allocated facilities costs.
Selling, General and Administrative Expense
Three Months Ended
−Removed: Six Months Ended
+Added: Nine Months Ended
+Added: September 30,
+Added: September 30,
(in thousands)
2 unchanged sentences
Stock-based compensation expense included in selling, general and administrative expense
−Removed: The increase in selling, general and administrative expense for the three months ended June 30, 2020 compared to the same period in 2019 was primarily due to the increases of $1.8 million in costs of consultants and third party services and $690,000 of personnel-related costs partially offset by the decreases of $1.1 million in travel-related commercial activities, $443,000 in stock-based compensation expense and $236,000 in various expense items.
−Removed: The decrease in selling, general and administrative expense for the six months ended June 30, 2020 compared to the same period in 2019 was primarily due to the decreases of $1.4 million in travel-related commercial activities and $1.3 million in stock-based compensation expense offset by the increases of $1.4 million in personnel-related costs, $200,000 in rent and $295,000 in various expense items.
−Removed: We expect our selling, general and administrative expense to increase as we continue to expand our commercial activities for TAVALISSE.
−Removed: As discussed above, resources have been deployed to enable our field-based employees to continue to engage remotely with healthcare providers during the ongoing COVID-19 pandemic.
−Removed: These virtual
−Removed: engagements have enabled our field team to support existing prescribers as well as partner with new prescribers to identify appropriate patients for TAVALISSE.
+Added: The decrease in selling, general and administrative expense for the three months ended September 30, 2020 compared to the same period in 2019 was primarily due to the decreases of $2.1 million in costs related to commercial activities and $259,000 in stock-based compensation expense partially offset by the increases of $1.4 million in personnel-related costs and $268,000 costs of consultants.
+Added: The decrease in selling, general and administrative expense for the nine months ended September 30, 2020 compared to the same period in 2019 was primarily due to the decreases of $2.1 million in costs of consultants and third party services, $1.5 million in stock-based compensation expense and $784,000 in costs related to commercial activities partially offset by the increases of $2.8 million in personnel costs and $88,000 in various expense items.
+Added: We expect our selling, general and administrative expense to increase for the remainder of 2020 as we continue to expand our commercial activities for TAVALISSE.
+Added: As discussed above, resources have been deployed to enable our
+Added: field-based employees to continue to engage remotely with healthcare providers during the ongoing COVID-19 pandemic.
+Added: These virtual engagements have enabled our field team to support existing prescribers as well as partner with new prescribers to identify appropriate patients for TAVALISSE.
However, we are not currently able to fully forecast the scope of impacts that the COVID-19 pandemic may have on our commercial activities and sales of TAVALISSE.
1 unchanged sentence
Three Months Ended
−Removed: Six Months Ended
+Added: Nine Months Ended
+Added: September 30,
+Added: September 30,
(in thousands)
2 unchanged sentences
Interest income results from our interest-bearing cash and investment balances.
−Removed: The decreases in interest income for the three and six months ended June 30, 2020 as compared to the same period in 2019 were primarily due to decrease in yield on our investments.
+Added: The decreases in interest income for the three and nine months ended September 30, 2020 as compared to the same period in 2019 were primarily due to decrease in yield on our investments.
Interest Expense
Three Months Ended
−Removed: Six Months Ended
+Added: Nine Months Ended
+Added: September 30,
+Added: September 30,
(in thousands)
1 unchanged sentence
Interest expense
−Removed: Interest expense for the three and six months ended June 30, 2020 was related to the outstanding balance on our term loan from Midcap.
−Removed: In May 2020, we received funding for the second tranche of $10.0 million.
+Added: Interest expense for the three and nine months ended September 30, 2020 was related to the outstanding balance on our term loan from Midcap.
Critical Accounting Policies and the Use of Estimates
12 unchanged sentences
We have consumed substantial amounts of capital to date as we continue our research and development activities, including preclinical studies and clinical trials and our ongoing commercial launch of TAVALISSE.
−Removed: As of June 30, 2020, we had approximately $92.5 million in cash, cash equivalents and short-term investments, as compared to approximately $98.1 million as of December 31, 2019, a decrease of approximately $5.6 million.
−Removed: The decrease was primarily attributable to payments associated with funding our operating expenses during the six months ended June 30, 2020.
+Added: As of September 30, 2020, we had approximately $72.8 million in cash, cash equivalents and short-term investments, as compared to approximately $98.1 million as of December 31, 2019, a decrease of approximately $25.3 million.
+Added: The decrease was primarily attributable to payments associated with funding our operating expenses during the nine months ended September 30, 2020.
In September 2019, we entered into a $60.0 million term loan credit facility with MidCap.
3 unchanged sentences
In August 2020, we entered into a Sales Agreement with Jefferies, pursuant to which we may sell, through Jefferies, up to an aggregate of $65.0 million in shares of our common stock.
+Added: In October 2020, we received $2.1 million representing payment for a milestone under our collaboration agreement with Daiichi.
In October 2018, we entered into an exclusive license and supply agreement with Kissei to develop and commercialize fostamatinib in all current and potential indications in Japan, China, Taiwan and the Republic of Korea, in which we received an upfront payment of $33.0 million.
6 unchanged sentences
Effective July 2017, the sublease agreement was amended primarily to extend the term of the sublease through January 2023.
−Removed: During the six months ended June 30, 2020, we received approximately $2.7 million of sublease income and reimbursements.
+Added: During the nine months ended September 30, 2020, we received approximately $4.0 million of sublease income and reimbursements.
We expect to receive approximately $10.7 million in future sublease income (excluding our subtenant’s share of facility’s operating expenses) through January 2023.
3 unchanged sentences
Our operations will require significant additional funding for the foreseeable future.
−Removed: Unless and until we are able to generate a sufficient amount of product, royalty or milestone revenue, we expect to finance future cash needs through public and/or private offerings of equity securities, debt financings and/or collaboration and licensing
−Removed: arrangements, and to a much lesser extent through the proceeds from exercise of stock options and interest income earned on the investment of our excess cash balances and short-term investments.
+Added: Unless and until we are able to generate a sufficient amount of product, royalty or milestone revenue, we expect to finance future cash needs
+Added: through public and/or private offerings of equity securities, debt financings and/or collaboration and licensing arrangements, and to a much lesser extent through the proceeds from exercise of stock options and interest income earned on the investment of our excess cash balances and short-term investments.
However, the COVID-19 pandemic continues to rapidly evolve and has already resulted in a significant disruption of global financial markets.
20 unchanged sentences
Insufficient funds may require us to delay, scale back or eliminate some or all of our commercial efforts and/or research or development programs, to lose rights under existing licenses or to relinquish greater or all rights to product candidates at an earlier stage of development or on less favorable terms than we would otherwise choose or may adversely affect our ability to operate as a going concern.
−Removed: For the three and six months ended June 30, 2020 and 2019, we maintained an investment portfolio primarily in money market funds, U.S.
+Added: For the three and nine months ended September 30, 2020 and 2019, we maintained an investment portfolio primarily in money market funds, U.S.
treasury bills, government-sponsored enterprise securities, and corporate bonds and commercial paper.
3 unchanged sentences
Cash Flows from Operating, Investing and Financing Activities
−Removed: Six Months Ended June 30,
+Added: Nine Months Ended September 30,
(in thousands)
4 unchanged sentences
Net (decrease) increase in cash and cash equivalents
−Removed: Net cash used in operating activities was approximately $17.1 million for the six months ended June 30, 2020, compared to approximately $17.2 million for the six months ended June 30, 2019.
−Removed: Net cash used in operating activities for the six months ended June 30, 2020 was related to our research and development programs and our ongoing commercialization of TAVALISSE, partially offset by the $20.0 million payment received from Grifols and proceeds from sale of TAVALISSE.
−Removed: Net cash used in operating activities for the six months ended June 30, 2019 was related to our research and development programs and our commercialization of TAVALISSE partially offset by the $30.0 million upfront fee received from Grifols.
+Added: Net cash used in operating activities was approximately $36.6 million for the nine months ended September 30, 2020, compared to approximately $31.9 million for the nine months ended September 30, 2019.
+Added: Net cash used in operating activities for the nine months ended September 30, 2020 was related to our research and development programs and our ongoing commercialization of TAVALISSE, partially offset by the $20.0 million payment received from Grifols and proceeds from sale of TAVALISSE.
+Added: Net cash used in operating activities for the nine months ended September 30, 2019 was related to our research and development programs and our commercialization of TAVALISSE partially offset by the $30.0 million upfront fee received from Grifols.
The timing of cash requirements may vary from period to period depending on our ongoing commercial activities related to TAVALISSE, timing of collaboration revenues, our ability to access additional funds from our credit facility with MidCap, our research and development activities, including our planned preclinical and clinical trials, and future requirements to establish commercial capabilities for any products that we may develop.
−Removed: Net cash provided by investing activities was approximately $19.2 million for the six months ended June 30, 2020, compared to net cash used in investing activities of approximately $15.7 million for the six months ended June 30, 2019.
−Removed: Net cash provided by investing activities during the six months ended June 30, 2020 related to net maturities of short-term investments, partially offset by capital expenditures.
−Removed: Net cash used in investing activities during the six months ended June 30, 2019 related to net purchases of short-term investments and capital expenditures.
−Removed: Capital expenditures were approximately $563,000 for the six months ended June 30, 2020, compared to approximately $492,000 for the same period in 2019.
−Removed: Net cash provided by financing activities was approximately $11.9 million for the six months ended June 30, 2020, compared to approximately $872,000 for the six months ended June 30, 2019.
−Removed: Net cash provided by financing activities for the six months ended June 30, 2020 related to the proceeds from funding of the second $10.0 million tranche from our term loan credit facility with MidCap and exercise of stock options and participation in the Purchase Plan.
−Removed: Net cash provided by financing activities for the six months ended June 30, 2019 related to the proceeds from exercise of stock options and participation in the Purchase Plan.
+Added: Net cash provided by investing activities was approximately $38.8 million for the nine months ended September 30, 2020, compared to net cash used in investing activities of approximately $16.0 million for the nine months ended September 30, 2019.
+Added: Net cash provided by investing activities during the nine months ended September 30, 2020 related to net maturities of short-term investments, partially offset by capital expenditures.
+Added: Net cash used in investing activities during the nine months ended September 30, 2019 related to net purchases of short-term investments and capital expenditures.
+Added: Capital expenditures were approximately $758,000 for the nine months ended September 30, 2020, compared to approximately $844,000 for the same period in 2019.
+Added: Net cash provided by financing activities was approximately $11.9 million for the nine months ended September 30, 2020, compared to approximately $10.7 million for the nine months ended September 30, 2019.
+Added: Net cash provided by financing activities for the nine months ended September 30, 2020 related to the proceeds from funding of the second $10.0 million tranche from our term loan credit facility with MidCap and exercise of stock options and participation in the Purchase Plan.
+Added: Net cash provided by financing activities for the nine months ended September 30, 2019 related to the funding of the first $10.0 million tranche from our term loan credit facility with MidCap and proceeds from exercise of stock options and participation in the Purchase Plan.
Off-Balance Sheet Arrangements
−Removed: As of June 30, 2020, we had no off-balance sheet arrangements (as defined in Item 303(a)(4)(ii) of Regulation S-K under the Exchange Act).
+Added: As of September 30, 2020, we had no off-balance sheet arrangements (as defined in Item 303(a)(4)(ii) of Regulation S-K under the Exchange Act).
Contractual Obligations
−Removed: We conduct our commercial activities and research and development programs internally and through third parties that include, among others, arrangements with collaboration partners, vendors, consultants, contract research
−Removed: organizations (CRO) and universities.
+Added: We conduct our commercial activities and research and development programs internally and through third parties that include, among others, arrangements with collaboration partners, vendors, consultants, contract research organizations (CRO) and universities.
We have contractual arrangements with these parties, however our contracts with them are cancelable generally on reasonable notice within one year and our obligations under these contracts are primarily based on services performed.
6 unchanged sentences
In addition, these agreements may, from time to time, be subjected to amendments as a result of any change orders executed by the parties.
−Removed: As of June 30, 2020, we do not have material contractual commitments with respect to the arrangements discussed above, but we had the following contractual commitments related to our facilities lease and credit facility:
+Added: As of September 30, 2020, we do not have material contractual commitments with respect to the arrangements discussed above, but we had the following contractual commitments related to our facilities lease and credit facility:
Payment Due By Period
13 unchanged sentences
Quantitative and Qualitative Disclosures About Market Risk
−Removed: During the six months ended June 30, 2020, there were no material changes to our market risk disclosures as set forth in Part II, Item 7A, “Quantitative and Qualitative Disclosures About Market Risk,” of our Annual Report on Form 10-K for the year ended December 31, 2019.
+Added: During the nine months ended September 30, 2020, there were no material changes to our market risk disclosures as set forth in Part II, Item 7A, “Quantitative and Qualitative Disclosures About Market Risk,” of our Annual Report on Form 10-K for the year ended December 31, 2019.
Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.