On November 2, 2021 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported financial results for the third quarter ended September 30, 2021, including sales of TAVALISSE (fostamatinib disodium hexahydrate) tablets for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment (Press release, Rigel, NOV 2, 2021, View Source [SID1234594099]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Rigel has expanded the commercial organization and our pipeline candidates are advancing in the clinic, setting us up for a transformative year in 2022," said Raul Rodriguez, Rigel’s president and CEO. "We recently achieved an important development milestone, completing enrollment of our pivotal FORWARD study in wAIHA, with an anticipated data readout in mid-2022. In addition, our sales force expansion is now complete, allowing us to have a greater impact in driving awareness and uptake for TAVALISSE as a novel, targeted therapy for ITP in the U.S."

"We recently achieved an important development milestone, completing enrollment of our pivotal FORWARD study in wAIHA."

Business Update

Enrollment of Rigel’s FORWARD study, a Phase 3 pivotal trial of TAVALISSE in patients with wAIHA, is complete. Rigel expects to report topline data from the 24-week study in mid-2022 and proceed with regulatory filings if the data is positive. If approved, TAVALISSE has the potential to be the first-to-market therapy for patients with wAIHA in 2023.

Rigel expanded its field sales force by the end of September, growing from 39 to 55 territory business managers to improve efficiency and increase in-person interactions.

In the third quarter of 2021, 1,710 bottles of TAVALISSE were shipped to patients and clinics, representing an increase of 5% year over year. Net product sales for the third quarter decreased 2% year over year to $16.0 million. During the third quarter, the company’s net product sales were negatively impacted by a decrease in bottles remaining in its distribution channels compared to Q2 2021.

During the quarter, partner Grifols announced its continued commercial rollout of TAVLESSE in Europe with launches in France, Italy and Spain. Grifols’ phased rollout across the rest of Europe planned over the following months is expected to include the Czech Republic, Denmark, Finland, Norway, and Sweden.

In September, results from the NIH/NHLBI-sponsored Phase 2 trial of fostamatinib in hospitalized patients with COVID-19 were published in Clinical Infectious Diseases.

Rigel’s Phase 3 clinical trial evaluating fostamatinib in high-risk patients hospitalized with COVID-19 has enrolled ~210 of the targeted 308 patients to date.
Financial Update
For the third quarter of 2021, Rigel reported a net loss of $21.0 million, or $0.12 per basic and diluted share, compared to a net loss of $14.2 million, or $0.08 per basic and diluted share, for the same period of 2020.

In the third quarter of 2021, total revenues were $21.5 million, consisting of $16.0 million in TAVALISSE net product sales, $4.5 million in contract revenues from collaborations, and $1.0 million in government contract revenue. TAVALISSE net product sales of $16.0 million in the third quarter of 2021 decreased by 2% from $16.3 million for the same period in 2020.

Contract revenues of $4.5 million from collaborations for the third quarter of 2021 consisted of $2.4 million in revenue related to the achievement of the remaining performance obligations in Rigel’s license agreement with Eli Lilly (Lilly), $1.8 million in revenue related to the achievement of a certain milestone from Daiichi Sankyo (Daiichi), $0.2 million in revenue related to the performance of certain research and development services pursuant to its collaboration agreement with Grifols, and a $0.1 million milestone payment from Medison. Government contract revenue of $1.0 million for the third quarter of 2021 was related to the income recognized pursuant to the agreement Rigel entered in January 2021 with the U.S. Department of Defense (DOD) to support Rigel’s ongoing Phase 3 clinical trial of fostamatinib in hospitalized patients with COVID-19.

Rigel reported total costs and expenses of $41.3 million in the third quarter of 2021, compared to $32.2 million for the same period in 2020. The increase in costs and expenses was primarily due to the research and development costs related to Rigel’s ongoing Phase 3 clinical trial of fostamatinib for the treatment of hospitalized patients with COVID-19, as well as increased commercial activities, including the recent sales force expansion.

For the nine months ended September 30, 2021, Rigel reported net income of $4.7 million, or $0.03 per basic and diluted share, compared to a net loss of $10.5 million, or $0.06 per basic and diluted share, for the same period of 2020.

Rigel reported total revenues of $128.8 million for the nine months ended September 30, 2021, consisting of $45.4 million in TAVALISSE net product sales, $73.9 million in contract revenues from collaborations, and $9.5 million in government contract revenues. TAVALISSE net product sales of $45.4 million increased by 3% from $43.9 million for the same period of 2020.

Total costs and expenses for the nine months ended September 30, 2021, were $119.9 million, compared to $100.3 million for the same period in 2020. The increase in costs and expenses was primarily due to increases in research and development costs related to Rigel’s various ongoing clinical studies, including its Phase 3 clinical trial of fostamatinib for the treatment of hospitalized patients with COVID-19, increased commercial activities that include the recent sales force expansion, personnel-related costs, and stock-based compensation expense.

As of September 30, 2021, Rigel had cash, cash equivalents, and short-term investments of $143.1 million, compared to $57.3 million as of December 31, 2020.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time
Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call and accompanying slides will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About ITP
In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that destroy the body’s own red blood cells. AIHA affects approximately 45,000 adult patients in the U.S. and can be a severe, debilitating disease. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients. Warm antibody AIHA (wAIHA), the most common form of AIHA, is characterized by the presence of antibodies that react with the red blood cell surface at body temperature.

About COVID-19 & SYK Inhibition
COVID-19 is the infectious disease caused by Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2). SARS-CoV-2 primarily infects the upper and lower respiratory tract and can lead to acute respiratory distress syndrome (ARDS). Additionally, some patients develop other organ dysfunction including myocardial injury, acute kidney injury, shock resulting in endothelial dysfunction and subsequently micro and macrovascular thrombosis.1 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.2

SYK is involved in the intracellular signaling pathways of many different immune cells. 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 pro-inflammatory cytokine release by monocytes and macrophages, production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation.3,4,5,6 Furthermore, SYK inhibition in neutrophils and platelets may lead to decreased thrombo-inflammation, alleviating organ dysfunction in critically ill patients with COVID-19.

For more information on Rigel’s comprehensive clinical program in COVID-19, go to: View Source

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.
Please see www.TAVALISSEUSPI.com for full Prescribing Information.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

TAVALISSE and TAVLESSE are registered trademarks of Rigel Pharmaceuticals, Inc.

On November 2, 2021 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported financial results for the third quarter ended September 30, 2021 and provided a corporate update (Press release, Deciphera Pharmaceuticals, NOV 2, 2021, View Source [SID1234594098]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In the third quarter, we made tremendous progress expanding the reach of QINLOCK for patients with GIST around the world. In addition to obtaining the approval for this important medicine in Switzerland and Taiwan, we also received a positive CHMP opinion for QINLOCK in the EU, with approval expected before the end of the year. Finally, we remain on track to report top-line results from the Phase 3 INTRIGUE study in second-line GIST later this quarter."

Mr. Hoerter continued, "At the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021, we shared very encouraging data from both the vimseltinib and rebastinib programs demonstrating significant clinical activity in two diseases with high unmet medical needs. We plan to initiate the Phase 3 MOTION study of vimseltinib in tenosynovial giant cell tumor this quarter and expect to initiate a Phase 3 study of rebastinib in combination with paclitaxel in platinum-resistant ovarian cancer next year. The body of scientific evidence continues to build supporting the potential of our first-in-class ULK inhibitor, DCC-3116, to address a broad spectrum of cancers in which autophagy is upregulated and believed to play an important role in tumor growth and survival. The Phase 1/2 study of DCC-3116 is on track and, with the first-in-class program in this field, we are well positioned to explore the full potential of inhibiting autophagy in cancer."

Third Quarter 2021 Highlights and Upcoming Milestones

QINLOCK(ripretinib)
Recorded $21.7 million in QINLOCK net product revenue in the third quarter of 2021, including $20.0 million in U.S. sales of QINLOCK and $1.7 million in ex-U.S. sales of QINLOCK.
Presented data at the ESMO (Free ESMO Whitepaper) Congress 2021:
An exploratory evaluation of primary and secondary endpoints in the Phase 3 INVICTUS study, with a cutoff date of January 15, 2021, an additional 19 months after the primary analysis, demonstrated consistent progression-free survival (PFS) with no change since the primary data cut off, and improved median overall survival (OS) among patients receiving QINLOCK.
Median PFS was 6.3 months with QINLOCK compared to 1.0 month with placebo.
Median OS was 18.2 months with QINLOCK compared to 6.3 months with placebo.
Phase 1 study in patients with KIT-mutated or KIT-amplified melanoma.
Received approvals in Switzerland and Taiwan for the treatment of adult patients with advanced GIST who have received prior treatment with three or more kinase inhibitors, including imatinib.
Expects approval from the EMA for QINLOCK in the fourth quarter of 2021.
Expects to announce top-line results from the Phase 3 INTRIGUE study in the fourth quarter of 2021.
Expects to initiate a Phase 1b/2 study of QINLOCK in combination with binimetinib, a commercially available MEK inhibitor, in post-imatinib GIST patients in the fourth quarter of 2021.
Vimseltinib
Presented updated data from the ongoing Phase 1/2 study in patients with TGCT at the ESMO (Free ESMO Whitepaper) Congress 2021, showing an encouraging ORR of 47% across all cohorts and a manageable safety and tolerability profile.
Expects to initiate the pivotal Phase 3 MOTION study of vimseltinib in the fourth quarter of 2021. MOTION is a two-part, randomized, double-blind, placebo-controlled study of vimseltinib to assess the efficacy and safety in patients with symptomatic TGCT who are not amenable to surgery. The primary endpoint of the study is ORR at week 25 as measured by RECIST v1.1 by blinded independent central review.
Granted Fast Track Designation by the FDA for the treatment of patients with symptomatic TGCT who are not amenable to surgery. This designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
Rebastinib
Presented updated data from the ongoing Phase 1b/2 study of rebastinib in combination with paclitaxel in the platinum-resistant ovarian cancer (PROC) cohort at the ESMO (Free ESMO Whitepaper) Congress 2021. Data showed promising results including median PFS of 9.1 months and an ORR of 38% (confirmed and unconfirmed) in heavily pretreated patients with PROC.
Received Orphan Drug Designation in the EU for the treatment of ovarian cancer based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP).
Announced that the Company has begun planning for a pivotal study in PROC that is anticipated to start in 2022, subject to feedback from regulators.
DCC-3116
Presented preclinical data at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) showing that DCC-3116 inhibits EGFR inhibitor-induced autophagy in multiple EGFR-mutant non-small cell lung cancer cell lines and decreased tumor burden in combination with osimertinib and afatinib in an EGFR mutant xenograft model.
Announced plans to present initial data from the dose escalation phase of the Phase 1 study in 2022.
Third Quarter Financial Results

Revenue: Total revenue for the third quarter of 2021 was $23.2 million, which includes $21.7 million of net product revenue from sales of QINLOCK and $1.5 million of collaboration revenue comprised primarily of QINLOCK supply and royalty revenue under our license agreement with Zai Lab. Total revenue for the third quarter of 2020 was $15.5 million, which included $15.2 million of net product revenue from sales of QINLOCK and $0.3 million of collaboration revenue.
Cost of Sales: Cost of sales was $0.9 million in the third quarter of 2021, which includes $0.2 million in cost of net product revenue for QINLOCK and $0.7 million in cost of collaboration revenue. Cost of sales was $0.1 million for the third quarter of 2020. Deciphera does not expect that the cost of sales as a percentage of net product sales of QINLOCK will increase significantly after the Company has sold all zero cost inventories and commenced the sales of inventories which will reflect the full cost of manufacturing. The Company expects to continue to sell the zero cost inventories of QINLOCK in the U.S. during 2021 and into 2022.
R&D Expenses: Research and development expenses for the third quarter were $66.4 million, compared to $49.2 million for the same period in 2020. The increase was primarily due to personnel and preclinical costs, and a $4.0 million up-front payment to Sprint Bioscience (Sprint) pursuant to the terms of the agreement with Sprint to exclusively in-license worldwide rights to a research-stage program targeting VPS34, and an increase in clinical trial expenses related to start-up activities for the planned Phase 3 MOTION study of vimseltinib and Phase 1b/2 study of QINLOCK in combination with binimetinib. Non-cash, stock-based compensation was $5.4 million and $4.5 million for the third quarters of 2021 and 2020, respectively.
SG&A Expenses: Selling, general, and administrative expenses for the third quarter of 2021 were $35.5 million, compared to $30.1 million for the same period in 2020. The increase was primarily due to personnel costs as well as external spend related to professional fees, including those associated with establishing a targeted commercial infrastructure and commercial preparedness in key European markets to support a launch of QINLOCK in Europe, if approved. Non-cash, stock-based compensation was $6.4 million and $5.3 million for the third quarters of 2021 and 2020, respectively.
Net Loss: For the third quarter of 2021, Deciphera reported a net loss of $79.8 million, or $1.37 per share, compared with a net loss of $63.7 million, or $1.13 per share, for the same period in 2020. The increase in net loss was primarily a result of increased R&D expenses, as described above, partially offset by increased sales volume in the U.S.
Cash Position: As of September 30, 2021, cash, cash equivalents, and marketable securities were $392.2 million, compared to $451.0 million as of June 30, 2021. Based on its current operating plans, Deciphera expects its current cash, cash equivalents, and marketable securities together with anticipated product, royalty, and supply revenues, excluding any potential future milestone payments under the Zai License Agreement, will enable the Company to fund its operating and capital expenditures into the first half of 2023.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, November 2, 2021, at 4:30 PM ET. To access the live call by phone please dial (866) 930-5479 (domestic) or (409) 216-0603 (international); the conference ID is 8178994. A live audio webcast of the event may also be accessed through the "Investors" section of Deciphera’s website at www.deciphera.com. A replay of the webcast will be available for 30 days following the event.

On November 2, 2021 Amgen (NASDAQ:AMGN) reported financial results for the third quarter of 2021 (Press release, Amgen, NOV 2, 2021, View Source [SID1234594097]). Key results include:

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Total revenues increased 4% to $6.7 billion in comparison to the third quarter of 2020, driven by higher unit demand, partially offset by lower net selling prices.
Product sales increased 4% globally, driven by double-digit volume growth across a number of our products, including Prolia (denosumab), EVENITY (romosozumab-aqqg), Repatha (evolocumab) and MVASI (bevacizumab-awwb).
GAAP earnings per share (EPS) decreased 3% to $3.31 driven by a $400 million licensing-related expense from our collaboration with Kyowa Kirin Co., Ltd. (Kyowa Kirin), partially offset by increased revenues.
GAAP operating income decreased 3% to $2.4 billion, and GAAP operating margin decreased 2.6 percentage points to 37.6%.
Non-GAAP EPS increased 11% to $4.67 driven by increased revenues and the impact of fewer weighted average shares outstanding. Total non-GAAP operating expenses increased less than 1%.
Non-GAAP operating income increased 8% to $3.5 billion, and non-GAAP operating margin increased 2.5 percentage points to 54.6%.
The Company generated $2.2 billion of free cash flow in the third quarter versus $3.2 billion in the third quarter of 2020, driven by higher collections in the third quarter of 2020 from customers who had been granted extended payment terms due to COVID-19. This increase was partially offset by the timing of tax payments in the third quarter of 2020.
2021 total revenues guidance of $25.8-$26.2 billion; EPS guidance of $9.55-$10.21 on a GAAP basis and $16.50-$17.10 on a non-GAAP basis.
"Our newest product, LUMAKRAS, a first-in-class lung cancer treatment, is off to a strong start and our robust pipeline of potential new medicines across all stages of development sets us up well to drive growth over the long term," said Robert A. Bradway, chairman and chief executive officer. "We achieved solid growth in the quarter as our medicines reached an increasing number of patients around the world."

References in this release to "non-GAAP" measures, measures presented "on a non-GAAP basis" and to "free cash flow" (computed by subtracting capital expenditures from operating cash flow) refer to non-GAAP financial measures. Adjustments to the most directly comparable GAAP financial measures and other items are presented on the attached reconciliations. For comparability of results to the prior year, non-GAAP net income and non-GAAP EPS amounts for 2020 have been revised to reflect the update to our non-GAAP policy that excludes gains and losses on certain equity investments. Refer to Non-GAAP Financial Measures below for further discussion.

Product Sales Performance

Total product sales increased 4% for the third quarter of 2021 versus the third quarter of 2020. Unit volumes grew 8% while net selling price declined 7%. In addition, this quarter includes $147 million of favorable changes to estimated sales deductions related to prior periods. In the third quarter last year, the favorable estimated sales deductions were $36 million, resulting in a $111 million year-over-year benefit in this quarter.

We continue to see gradual recovery from the impact of the COVID-19 pandemic. As we progressed through the third quarter, we saw improvement in patient visits and diagnoses. Healthcare professional activity also improved during the first half of 2021 and stabilized during the third quarter. Overall, the gap in diagnosis visits over the course of the pandemic has suppressed the number of new patients starting treatment, which we expect will continue to impact our business for the remainder of the year.

General Medicine

Prolia sales increased 15% year-over-year for the third quarter, driven by 13% volume growth. New and repeat patient visits continued to improve as osteoporosis diagnosis rates in the U.S. reached over 90% of pre-COVID levels during the quarter.
EVENITY sales increased 153% year-over-year to a record $149 million for the third quarter, primarily driven by 118% volume growth. U.S. sales grew 74% year-over-year, driven by 65% volume growth as we continued to focus on increasing the number of new patients starting treatment. Outside the U.S., year-over-year volume growth was strong, amplified by inventory drawdowns by our partner Astellas during the third quarter of 2020.
Repatha sales increased 33% year-over-year for the third quarter, primarily driven by 42% volume growth partially offset by lower net selling price. In the U.S., volumes grew 64% year-over-year, and outside the U.S., volumes grew 24% year-over-year. Volume growth in the quarter was partially offset by lower net selling price, primarily as a result of an increase in the number of U.S. Medicare Part D patients receiving Repatha and entering the coverage gap, the so-called "donut hole." The impact of the donut hole is more pronounced in the second half of the year as patients reach their plan deductibles.
Aimovig (erenumab-aooe) sales decreased 25% year-over-year for the third quarter, driven by lower net selling price.
Inflammation

Otezla (apremilast) sales increased 13% year-over-year for the third quarter. Volume grew 7% partially offset by lower net selling price. Sales in the quarter benefited from an $18 million favorable adjustment to estimated sales deductions, compared to a $24 million unfavorable adjustment in the third quarter last year, resulting in an 8% year-over-year benefit. In the U.S., Otezla continued to maintain first-line share leadership in psoriasis. Looking forward, we are preparing for the anticipated U.S. approval of the mild-to-moderate psoriasis indication.
Enbrel (etanercept) sales decreased 3% year-over-year for the third quarter, driven by declines in volume, inventory and net selling price. Sales in the quarter benefited from a $114 million favorable adjustment to estimated sales deductions, compared to a $84 million favorable adjustment in the third quarter last year, resulting in a 2% year-over-year benefit. Year-over-year volume declined by 2%, representing the second consecutive quarter of slowing volume declines. We expect the trend of year-over-year net selling price declines to continue.
AMGEVITA (adalimumab) sales increased 39% year-over-year for the third quarter, driven by 73% volume growth partially offset by lower net selling price. AMGEVITA continues to be the most prescribed adalimumab biosimilar in Europe.
Hematology-Oncology

LUMAKRAS/LUMYKRAS (sotorasib) generated $36 million of sales in the quarter and cumulative sales of $45 million through the end of the third quarter. LUMAKRAS has been prescribed by over 500 physicians in both academic and community settings. A majority of the top clinical laboratories have now updated their reports to reflect KRAS G12C as an actionable mutation and ~75% of patients with NSCLC are now being tested by their oncologists at diagnosis for the KRAS G12C mutation.
KYPROLIS (carfilzomib) sales increased 13% year-over-year for the third quarter, driven by 10% volume growth supported by physician adoption of KYPROLIS use in combination with DARZALEX (daratumumab) plus dexamethasone (DKd).
XGEVA (denosumab) sales increased 7% year-over-year for the third quarter, driven by 9% volume growth partially offset by lower net selling price.
Vectibix (panitumumab) sales increased 4% year-over-year for the third quarter, driven by 8% volume growth as Vectibix remains the EGFR inhibitor of choice across all lines of therapy.
Nplate (romiplostim) sales increased 29% year-over-year for the third quarter, primarily driven by 14% volume growth.
BLINCYTO (blinatumomab) sales increased 40% year-over-year for the third quarter, primarily driven by 30% volume growth as we continue to see broad adoption in the community hospital setting.
MVASI sales increased 19% year-over-year for the third quarter, driven by 54% volume growth partially offset by lower net selling price. In the U.S., MVASI continues to hold leading volume share with 49% of the bevacizumab segment in the quarter. We expect that continued worldwide volume growth from MVASI will be offset by declines in net selling price due to increased competition.
KANJINTI (trastuzumab-anns) sales decreased 31% year-over-year for the third quarter, driven by lower net selling price, partially offset by 18% volume growth. In the U.S., KANJINTI continues to hold leading volume share with 41% of the trastuzumab segment in the quarter. We expect net selling price to continue to decline as a result of increased competition.
Established Products

Total sales of our established products, which include Neulasta (pegfilgrastim), NEUPOGEN (filgrastim), EPOGEN (epoetin alfa), Aranesp (darbepotein alfa), Parsabiv (etelcalcetide), and Sensipar/Mimpara (cinacalcet), decreased 21% year-over-year for the third quarter, primarily driven by volume declines and lower net selling price. Going forward, we expect increased competition to result in additional net price and volume erosion across this portfolio of products.
Product Sales Detail by Product and Geographic Region

Operating Expense, Operating Margin and Tax Rate Analysis

On a GAAP basis:

Total Operating Expenses increased 9% primarily driven by a licensing-related expense from our recent collaboration with Kyowa Kirin. Cost of Sales margin decreased 0.1 percentage points primarily due to lower amortization expense from acquisition-related assets, offset by product mix. Research & Development (R&D) expenses increased 34% primarily due to a licensing-related expense from our collaboration with Kyowa Kirin, partially offset by lower late-stage program support. Selling, General & Administrative (SG&A) expenses decreased 3%.
Operating Margin as a percentage of product sales decreased 2.6 percentage points to 37.6%.
Tax Rate increased 4.2 percentage points primarily driven by the non-deductible acquired IPR&D expense arising from the acquisition of Five Prime Therapeutics.
On a non-GAAP basis:

Total Operating Expenses increased less than 1%. Cost of Sales margin increased 1.5 percentage points primarily due to product mix, including COVID-19 antibody shipments to Lilly that began last quarter. R&D expenses decreased 4% primarily due to lower late-stage program support. SG&A expenses decreased 5%.
Operating Margin as a percentage of product sales increased 2.5 percentage points to 54.6%.
Tax Rate increased 0.5 percentage points primarily driven by a prior year favorable item partially offset by a change in earnings mix.
Cash Flow and Balance Sheet

The Company generated $2.2 billion of free cash flow in the third quarter of 2021 versus $3.2 billion in the third quarter of 2020, driven by higher collections in the third quarter of 2020 from customers who had been granted extended payment terms due to COVID-19. This increase was partially offset by the timing of tax payments in the third quarter of 2020.
The Company’s third quarter 2021 dividend of $1.76 per share was declared on July 30, 2021, and was paid on September 8, 2021, to all stockholders of record as of August 17, 2021, representing a 10% increase from 2020.
During the third quarter, the Company repurchased 4.6 million shares of common stock at a total cost of $1.1 billion. At the end of the third quarter, the Company had $2.9 billion authorization remaining under its stock repurchase program. In October 2021, the Board of Directors increased the amount authorized under our stock repurchase program by an additional $4.5 billion.
Cash and investments totaled $12.9 billion and debt outstanding totaled $37.6 billion as of September 30, 2021.
2021 Guidance

For the full year 2021, the Company now expects:

Total revenues in the range of $25.8 billion to $26.2 billion.
On a GAAP basis, EPS in the range of $9.55 to $10.21 and a tax rate in the range of 12.5% to 14.0%.
On a non-GAAP basis, EPS in the range of $16.50 to $17.10 and a tax rate in the range of 13.0% to 14.0%.
Capital expenditures to be approximately $900 million.
Share repurchases at the upper end of $3.0 billion to $5.0 billion range.
Third Quarter Product and Pipeline Update

The Company provided the following updates on selected product and pipeline programs:

LUMAKRAS/LUMYKRAS

In September, marketing authorizations were granted for LUMYKRAS in Great Britain and LUMAKRAS in Canada for the treatment of second-line patients with KRAS G12C-mutated, advanced non-small cell lung cancer (NSCLC). Regulatory reviews continue in Europe, Japan and other jurisdictions.
In September, data from LUMAKRAS in combination with Vectibix in patients with advanced KRAS G12C-mutated colorectal cancer (CRC) were presented at the European Society for Medical Oncology Congress. A Phase 3 study of LUMAKRAS in combination with Vectibix in third-line colorectal cancer is expected to initiate in Q4 2021.
In September, data from the Phase 1/2 CodeBreaK 100 monotherapy study in advanced KRAS G12C-mutated NSCLC, including biomarker analyses and post hoc analyses of efficacy and safety in patients with stable brain metastases, were presented at the World Conference on Lung Cancer. Enrollment continues in a cohort of patients with active brain metastases in the CodeBreaK 101 study.
In October, data from cohorts exploring LUMAKRAS in combination with trematinib, a mitogen-activated protein kinase kinase inhibitor, and LUMAKRAS in combination with afatinib, an oral epidermal growth factor receptor inhibitor, were presented at the AACR (Free AACR Whitepaper)-NCI-EORTC 2021 Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper).
A Phase 2 study has initiated in first-line patients with KRAS G12C-mutated NSCLC whose tumors express serine/threonine kinase 11 (STK11) mutations and/or less than 1% programmed death-ligand 1.
Top-line results from the event-driven, confirmatory Phase 3 study comparing LUMAKRAS to docetaxel in patients with KRAS G12C-mutated advanced NSCLC are expected in H1 2022.
Top-line results from the Phase 2 monotherapy study in patients with KRAS G12C-mutated solid tumors other than NSCLC and CRC are expected in H1 2022.
Initial data from cohorts exploring LUMAKRAS in combination with the anti-programmed cell death 1 (PD-1) antibody pembrolizumab and LUMAKRAS in combination with the Src homology-2 domain-containing protein tyrosine phosphatase-2 (SHP2) inhibitor RMC-4630 from Revolution Medicines are expected to be presented in H1 2022.
BLINCYTO

A Phase 3 study of BLINCYTO alternating with low-intensity chemotherapy versus standard of care for older adults with newly diagnosed Philadelphia-negative B-cell precursor acute lymphoblastic leukemia has initiated.
Bemarituzumab

The Phase 3 program has initiated for bemarituzumab, a fibroblast growth factor receptor 2b antibody, in first-line advanced gastric and gastroesophageal junction adenocarcinoma. The program will explore bemarituzumab in combination with either backbone chemotherapy or chemotherapy plus a checkpoint inhibitor.
A Phase 1b signal-seeking study of bemarituzumab alone and in combination with chemotherapy for the treatment of advanced, refractory, squamous NSCLC is expected to initiate by Q1 2022. Planning is underway for signal-seeking studies in other solid tumors.
Acapatamab (AMG 160)

Data continue to mature in a dose-expansion cohort of acapatamab, a half-life extended (HLE) BiTE molecule targeting prostate-specific membrane antigen (PSMA) for the treatment of patients with metastatic castrate-resistant prostate cancer (mCRPC). Enrollment of acapatamab is ongoing in cohorts with reduced levels of monitoring during cycle one to explore outpatient administration.
An acapatamab dose-escalation study is enrolling patients with NSCLC tumors expressing PSMA.
A master protocol evaluating combinations of acapatamab with AMG 404, an anti-PD-1 antibody, or the novel hormone therapies enzalutamide or abiraterone, continues to enroll patients with earlier-line mCRPC.
AMG 340 (formerly TNB-585)

A Phase 1 dose-escalation study of AMG 340, a UniAb bispecific T-cell engager targeting PSMA, is enrolling patients with mCRPC.
Tarlatamab (AMG 757)

Initiation of a potentially pivotal Phase 2 study for tarlatamab, an HLE BiTE molecule targeting delta-like ligand 3 (DLL3), in patients with relapsed or refractory small cell lung cancer (SCLC) is planned for Q4 2021.
A Phase 1b dose-expansion cohort of tarlatamab in patients with SCLC is ongoing.
A Phase 1b study of tarlatamab continues to enroll patients with neuroendocrine prostate cancer.
A Phase 1b study of tarlatamab in combination with AMG 404 has initiated for patients with SCLC.
Tezepelumab

Tezepelumab, a thymic stromal lymphopoietin (TSLP) antibody, is under Priority Review by the U.S. Food and Drug Administration (FDA) for severe asthma with a Prescription Drug User Fee Act (PDUFA) target date in Q1 2022. Regulatory reviews are also underway in the EU, Japan, and other jurisdictions.
A Phase 3 study continues to enroll patients with chronic rhinosinusitis with nasal polyps.
A Phase 2b study continues to enroll patients with chronic spontaneous urticaria.
A Phase 2 study continues to enroll patients with chronic obstructive pulmonary disease.
Tezepelumab was granted Orphan Drug Designation by the FDA for the treatment of eosinophilic esophagitis.
Otezla

The FDA review of Otezla for the treatment of adults with mild-to-moderate plaque psoriasis continues to progress, with a PDUFA target action date of December 19, 2021.
In August, China’s National Medical Products Administration approved Otezla for the treatment of adult patients with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy.
Phase 3 initiation for the treatment of Japanese patients with palmoplantar pustulosis is expected to begin in H1 2022.
AMG 451 / KHK4083

In October, positive results from the Phase 2b study of AMG 451, an anti-OX40 monoclonal antibody for the treatment of atopic dermatitis, were presented at the European Academy of Dermatology and Venereology 30th Virtual Congress. Phase 3 development is expected to begin in H1 2022.
Biomarker analyses from the Phase 2 atopic dermatitis study will be presented at the Inflammatory Skin Disease Summit on November 4, 2021.
Rozibafusp alfa (AMG 570)

A Phase 2b study of rozibafusp alfa, a multispecific antibody-peptide conjugate that simultaneously blocks inducible T-cell costimulatory ligand (ICOSL) and B-cell activating factor (BAFF) activity, continues to enroll patients with systemic lupus erythematosus (SLE).
Efavaleukin alfa (AMG 592)

A Phase 2b study of efavaleukin alfa, an interleukin-2 mutein Fc fusion protein, continues to enroll patients with SLE.
Data from a Phase 1b study in patients with SLE will be presented at the American College of Rheumatology Convergence 2021 on November 9, 2021.
A Phase 2 study of efavaleukin alfa in patients with ulcerative colitis has initiated.
AMG 714 / PRV-015

A Phase 2b study of AMG 714, a monoclonal antibody that binds interleukin-15, continues to enroll patients with non-responsive celiac disease.
Repatha

In September, the FDA approved Repatha as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C)-lowering therapies for the treatment of pediatric patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH) to reduce LDL-C. The FDA also expanded the homozygous familial hypercholesterolemia (HoFH) indication to patients aged 10 years and older.
In October, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion recommending an update to the Marketing Authorization for Repatha for the treatment of pediatric patients aged 10 years and older with HeFH, and the expansion of treatment for pediatric HoFH patients aged 10 years and older.
A Phase 3 cardiovascular outcomes study (VESALIUS-CV) continues to enroll patients at high cardiovascular risk without prior myocardial infarction or stroke.
Olpasiran (AMG 890)

Results from a Phase 2 study of olpasiran, a lipoprotein(a) (Lp(a)) small interfering RNA molecule, in patients with elevated Lp(a) are expected in H1 2022 with publication expected in H2 2022.
Biosimilars

A Phase 3 study of ABP 938, an investigational biosimilar to EYLEA (aflibercept) continues to enroll patients, with data expected in 2022.
Phase 3 studies of ABP 654, an investigational biosimilar to STELARA (ustekinumab), and ABP 959, an investigational biosimilar to SOLIRIS (eculizumab), are ongoing, with data expected in 2022.
Phase 3 studies to support an interchangeability designation in the U.S. for ABP 654 and AMJEVITA (adalimumab-atto) are enrolling patients.
Amgenpipeline.com

A listing of additional ongoing clinical programs can be found at Amgenpipeline.com
Tezepelumab is being developed in collaboration with AstraZeneca
AMG 451 (also known as KHK4083) is being developed in collaboration with Kyowa Kirin
AMG 714 (also known as PRV-015) is being developed in collaboration with Provention Bio
DARZALEX and STELARA are a registered trademarks of Janssen Pharmaceutica NV
EYLEA is a registered trademark of Regeneron Pharmaceuticals, Inc.
SOLIRIS is a registered trademark of Alexion Pharmaceuticals, Inc.

On November 2, 2021 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that the Company will release its third quarter 2021 financial results on Tuesday, November 9, 2021, after the close of U.S. markets. Management will host a conference call on the same day at 4:30 pm ET (Press release, Curis, NOV 2, 2021, View Source,-2021 [SID1234594096]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To access the live conference call, please dial (888) 346-6389 from the United States or (412) 317-5252 from other locations, shortly before 4:30 pm ET. The conference call can also be accessed on the Curis website at www.curis.com in the ‘Investors’ section. A replay of the financial results conference call will be available on the Curis website shortly after completion of the call.

On November 2, 2021 Cerus Corporation (Nasdaq: CERS) reported financial results for the third quarter ended September 30, 2021 (Press release, Cerus, NOV 2, 2021, View Source [SID1234594095]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Recent developments and highlights include:

Third quarter 2021 total revenue of $42.1 million, reflecting a 44% increase over the prior year period. Total revenue was composed of (in thousands, except %):

As of the date of this release, the Company is increasing its 2021 annual product revenue guidance range to $127 million to $129 million (from the prior guidance of $118 million to $122 million), representing an approximate 38% to 40% increase over full year 2020 reported product revenue.
On October 1, 2021, the U.S. Food and Drug Administration’s (FDA) final guidance document on bacterial risk control strategies for platelet collection and transfusion was made effective. This guidance for the industry identifies FDA-approved pathogen reduction as a means of compliance.
Cerus achieved cumulative sales for the INTERCEPT Blood System for platelets and plasma that have surpassed 10 million treatable doses globally since the products were first commercially launched.
Cash, cash equivalents, and short-term investments were $120 million at September 30, 2021.
"Cerus’ third quarter results represent a strong commercial achievement for the Company, in particular with regard to the U.S. platelet business. With quarterly product revenue of $36.1 million and sales momentum that continues into the fourth quarter, I am pleased that adoption of the INTERCEPT Blood System for platelets is establishing a new standard of care," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer. "With solid visibility to continued demand for the balance of the year, we are raising our product revenue guidance and look forward to finishing this breakout year for Cerus on a strong note."

Revenue

Product revenue during the third quarter of 2021 was $36.1 million, compared to $23.6 million during the same period in 2020. Product revenue growth during the quarter was driven by increased sales of INTERCEPT platelet products to blood center customers across the U.S.

Third quarter government contract revenue was $6.0 million, compared to $5.6 million during the same period in 2020. Third quarter government contract revenue was comprised of funding associated with research and development (R&D) activities related to the INTERCEPT Blood System for Red Cells as well as sponsored efforts related to the development of next generation pathogen reduction technology to treat whole blood.

Product Gross Profit & Margin

Product gross profit for the third quarter 2021 was $18.5 million and was the highest in company history, reflecting a nearly $6 million increase over the same period in 2020. Product gross margin for the third quarter 2021 was 51.3% compared to 53.6% for the third quarter of 2020 and flat compared to the second quarter of 2021. The anticipated decrease in product gross margin compared to the third quarter of 2020 was tied to higher sales to U.S. customers during the quarter, who at present, primarily use the Company’s single dose platelet kits, which provide a lower product gross margin percentage compared to our double dose kits that are more frequently used internationally.

Operating Expenses

Total operating expenses for the third quarter of 2021 were $35.6 million compared to $32.2 million for the same period of the prior year. In general, the increase in operating expenses compared to the prior year period were driven by higher commercial expenses, due to sales incentive compensation and partially offset by lower R&D expenses.

Selling, general, and administrative (SG&A) expenses for the third quarter of 2021 totaled $20.4 million, compared to $16.3 million for the third quarter of 2020. The year-over-year increase in SG&A expenses was tied to increased sales costs and continued investments in our therapeutics business unit.

R&D expenses for the third quarter of 2021 were $15.3 million, compared to $15.9 million for the third quarter of 2020. Continued investments in a variety of R&D pipeline projects continued during the quarter, including those related to the Company’s LED illuminator, but were offset by lower R&D costs versus the prior year period associated with mature projects that have been completed.

Net Loss Attributable to Cerus Corporation

Net loss attributable to Cerus Corporation for the third quarter of 2021 was $12.4 million, or $0.07 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $14.1 million, or $0.08 per basic and diluted share, for the third quarter of 2020.

Balance Sheet

At September 30, 2021, the Company had cash, cash equivalents and short-term investments of $120.0 million, compared to $122.8 million at June 30, 2021 and $133.6 million at December 31, 2020. Despite continued investments in inventory to meet the anticipated growth in demand, cash used from operations continued to decline as the Company realized increased revenue contribution and significant leverage in SG&A spend.

As of September 30, 2021, the Company carried $55 million of notes due and a balance on its revolving line of credit of $10 million. The Company continues to have access to another $15 million under its term facility and capacity for an additional $10 million under its revolving line of credit.

Increasing 2021 Product Revenue Guidance

Based on the strong product revenue year to date and updated demand estimates heading into the remainder of the Company’s fourth quarter, the Company now expects 2021 product revenue to be in the range of $127 million to $129 million, compared to the prior range of $118 million to $122 million. The revised guidance range represents approximately 38% to 40% growth over 2020 reported product revenue.

Quarterly Conference Call

The Company will host a conference call at 4:30 P.M. EDT this afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook. To listen to the live webcast, please visit the Investor Relations page of the Cerus website at View Source Alternatively, you may access the live conference call by dialing (866) 235-9006 (U.S.) or (631) 291-4549 (international).

A replay will be available on Cerus’ website, or by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and entering conference ID number 3970817. The replay will be available approximately three hours after the call through November 16, 2021.