Deciphera Pharmaceuticals, Inc. Announces Second Quarter 2021 Financial Results

On August 3, 2021 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported financial results for the first quarter ended June 30, 2021 and provided a corporate update (Press release, Deciphera Pharmaceuticals, AUG 3, 2021, View Source [SID1234585603]).

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"We made significant progress against our goals in the first half of the year and we look forward to carrying this momentum through the rest of 2021," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "Building on the successful launch of QINLOCK for fourth-line GIST in the U.S., this best-in-class medicine has now been approved in China and Hong Kong and we expect approval from the European Medicines Agency later this year. In the fourth quarter, we look forward to reporting top-line data from the INTRIGUE Phase 3 study in second-line GIST. In addition, we expect to report updated data from the vimseltinib and rebastinib programs at the upcoming ESMO (Free ESMO Whitepaper) congress as well as finalize pivotal development plans for both programs later this year."

Mr. Hoerter continued, "We also recently achieved a key milestone treating the first patient in our Phase 1 trial of DCC-3116, a first-in-class ULK kinase inhibitor designed to address mutant RAS and RAF cancers through the inhibition of autophagy."

Second Quarter 2021 Highlights and Upcoming Milestones

QINLOCK (ripretinib)
Recorded $22.0 million in QINLOCK net product revenue in the second quarter of 2021, including $20.7 million in U.S. sales of QINLOCK and $1.3 million in ex-U.S. sales of QINLOCK.
Launched commercially in China, via our collaboration with Zai, for the treatment of adult patients with fourth-line GIST.
Presented data for QINLOCK patients undergoing intra-patient dose escalation after disease progression in the INVICTUS Phase 3 study at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June.
Expects to announce top-line results from the INTRIGUE Phase 3 study in the fourth quarter of 2021.
Expects potential approval from the European Medicines Agency (EMA) for QINLOCK 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
Expects to present updated data from the ongoing Phase 1/2 study in patients with TGCT at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 in September.
Plans to finalize the pivotal development plan for vimseltinib in patients with TGCT in the second half of 2021.
Rebastinib
Expects to present updated data from the ongoing Phase 1b/2 study of rebastinib in combination with paclitaxel in the platinum-resistant ovarian cancer cohort at the ESMO (Free ESMO Whitepaper) Congress 2021 in September.
Plans to finalize the pivotal development plan for rebastinib in combination with paclitaxel in the second half of 2021.
DCC-3116
Initiated the Phase 1 study of DCC-3116 in June 2021. The study is evaluating DCC-3116 as a single agent and in combination with trametinib in patients with advanced or metastatic tumors with a mutant RAS or RAF gene. Currently, expansion cohorts are planned in patients with advanced or metastatic pancreatic ductal adenocarcinoma with KRAS or BRAF mutations, non-small cell lung cancer with KRAS, NRAS, or BRAF mutations, colorectal cancer with KRAS, NRAS, or BRAF mutations, and melanoma with NRAS or BRAF mutations.
Expects to present preclinical data on DCC-3116 in combination with approved, targeted oncology agents in multiple tumor models at an upcoming medical meeting in the second half of 2021.
Recent Corporate Updates

Today announced an agreement with Sprint Bioscience to exclusively in-license worldwide rights to a research-stage program targeting VPS34, a key kinase in the autophagy pathway, strengthening the company’s leading position in the development of regulators of autophagy for the potential treatment of cancer. VPS34 is involved in the endosomal trafficking of cellular cargo targeted for lysosomal degradation in cancer cells. Targeting VPS34 may provide an additional approach to regulating autophagy that is complementary to inhibition of ULK kinase by blocking VPS34-mediated immunosuppression in tumors.
Upcoming Scientific Congress Presentations

ESMO Congress 2021, September 16-21. The following will be e-poster presentations and will be available on-demand via the ESMO (Free ESMO Whitepaper) Congress 2021 website beginning on September 16 at 8:30 AM CEST / 2:30 AM EST.
QINLOCK
Ripretinib as ≥4th-line treatment in patients with advanced gastrointestinal stromal tumor: long-term update from the Phase 3 INVICTUS study.
Phase 1 study of ripretinib, a broad-spectrum KIT and PDGFRA inhibitor, in patients with KIT-mutated or KIT-amplified melanoma.
Vimseltinib
Safety and preliminary efficacy of vimseltinib in tenosynovial giant cell tumor (TGCT).
Rebastinib
A Phase 1b/2 study of rebastinib and paclitaxel in advanced/metastatic platinum-resistant ovarian cancer.
Second Quarter Financial Results

Revenue: Total revenue for the second quarter of 2021 was $23.6 million, which includes $22.0 million of net product revenue from sales of QINLOCK and $1.5 million of collaboration revenue comprised of commercial supply and royalty revenue under our license agreement with Zai Lab. Total revenue for the second quarter of 2020 was $7.1 million, which includes $4.8 million of net product revenue from sales of QINLOCK and $2.3 million of collaboration revenue.
Cost of Sales: Cost of sales was $1.3 million in the second quarter of 2021, which includes $0.4 million in cost of net product revenue for QINLOCK and $0.9 million in cost of collaboration revenue. Cost of sales was less than $0.1 million for the second quarter of 2020. Cost of net product sales is not expected to be significant until the initial pre-launch inventory is depleted, and additional inventory is manufactured and sold.
R&D Expenses: Research and development expenses for the second quarter were $60.0 million, compared to $46.1 million for the same period in 2020. The increase was primarily due to personnel and preclinical costs and an increase in clinical trial expenses related to our ongoing Phase 1/2 study of vimseltinib. Non-cash, stock-based compensation was $5.6 million and $5.3 million for the second quarters of 2021 and 2020, respectively.
SG&A Expenses: Selling, general, and administrative expenses for the second quarter of 2021 were $32.8 million, compared to $29.9 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 in key European markets to support a potential launch of QINLOCK in Europe, if approved. Non-cash, stock-based compensation was $6.8 million and $5.3 million for the first quarters of 2021 and 2020, respectively.
Net Loss: For the second quarter of 2021, Deciphera reported a net loss of $70.4 million, or $1.21 per share, compared with a net loss of $67.2 million, or $1.20 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 a full quarter of product sales during the second quarter of 2021.
Cash Position: As of June 30, 2021, cash, cash equivalents, and marketable securities were $451.0 million, compared to $502.2 million as of March 31, 2021. Based on its current operating plans, Deciphera expects its current cash, cash equivalents, and marketable securities together with anticipated product and royalty revenues, excluding any potential future milestone payments or other payments under its collaboration or license agreements, 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, August 3, 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 9943767. 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.

Amgen Reports Second Quarter 2021 Financial Results

On August 3, 2021 Amgen (NASDAQ:AMGN) reported financial results for the second quarter of 2021 (Press release, Amgen, AUG 3, 2021, View Source [SID1234585602]). Key results include:

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Total revenues increased 5% to $6.5 billion in comparison to the second quarter of 2020, driven by higher unit demand, partially offset by lower net selling prices.
Product sales increased 3% globally, driven by double digit volume growth across a number of our products including Prolia (denosumab), Repatha (evolocumab) and our biosimilar products MVASI (bevacizumab-awwb) and KANJINTI (trastuzumab-anns).
GAAP earnings per share (EPS) decreased 73% to $0.81 driven by the write-off of $1.5 billion in acquired in-process research & development (acquired IPR&D) associated with our acquisition of Five Prime Therapeutics, partially offset by increased revenues.
GAAP operating income decreased 64% to $0.8 billion and GAAP operating margin decreased 25.8 percentage points to 13.5%.
Non-GAAP EPS increased 4% to $4.38 driven by increased revenues and the impact of fewer weighted average shares outstanding.
Non-GAAP operating income decreased 4% to $3.1 billion and non-GAAP operating margin decreased 4.1 percentage points to 50.9%.
The Company generated $1.7 billion of free cash flow in the second quarter versus $2.7 billion in the second quarter of 2020 driven by a difference in the timing of tax payments.
2021 total revenues guidance reaffirmed at $25.8-$26.6 billion; EPS guidance revised to $8.84-$9.90 on a GAAP basis, and reaffirmed at $16.00-$17.00 on a non-GAAP basis.

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

COVID-19 update: Compared to the first quarter of 2021, we have seen gradual recovery from the impacts of the COVID-19 pandemic. Patient visits and lab test procedure trends continued to improve but remained below pre-COVID-19 levels. The cumulative decrease in diagnoses over the course of the pandemic has suppressed the volume of new patients starting treatment, which we expect to continue to impact our business during the second half of the year.

Total product sales increased 3% for the second quarter of 2021 versus the second quarter of 2020. Unit volumes grew 8% while net selling price declined 5%. On a sequential basis, product sales grew 9% quarter-over-quarter, driven by 6% volume growth.

Results for individual products are as follows:

Prolia sales increased 24% year-over-year for the second quarter, driven by 20% volume growth as new and repeat patient volumes continued to recover from the pandemic. With osteoporosis diagnosis rates remaining at approximately 90% of pre-COVID-19 levels in the quarter, we are focused on driving patient growth and are optimistic about Prolia’s continued strength in the second half of the year.
EVENITY (romosozumab-aqqg) sales increased 30% year-over-year for the second quarter, driven by 32% volume growth. U.S. sales nearly doubled year-over-year, driven by 97% volume growth as we continued to focus on new patient activation. Rest of world (ROW) sales decreased 15% year-over-year due in part to the timing of purchases by our partner Astellas during the first half of 2020.
Repatha sales increased 43% year-over-year for the second quarter, driven by 49% volume growth. In the U.S., volumes grew 37% year-over-year, and outside the U.S. volumes grew 66% year-over-year. Volume growth in the quarter was partially offset by lower net selling price as a result of an increase in the number of U.S. Medicare Part D patients receiving Repatha and entering the coverage gap. We expect further reduction in the net selling price on a sequential basis as the number of Medicare Part D patients receiving Repatha increases. Repatha has now been prescribed for more than one million patients.
Aimovig (erenumab-aooe) sales decreased 16% year-over-year for the second quarter. Unit volume growth of 11% was offset by lower net selling price and unfavorable changes to estimated sales deductions. Aimovig retains strong payer coverage and remains the segment leader within the preventive calcitonin gene-related peptide (CGRP) class. To date, more than 500,000 patients worldwide have been prescribed Aimovig for the preventive treatment of migraine.
Otezla (apremilast) sales decreased 5% year-over-year for the second quarter, primarily driven by unfavorable changes to estimated sales deductions and lower net selling price, partially offset by 5% volume growth. In the U.S., Otezla continued to maintain first-line share leadership in psoriasis. New-to-brand prescription (NBRx) volumes grew 10% year-over-year, even as patient visits to dermatologists remained 15% below pre-pandemic levels. The number of new patients that started treatment with Otezla in Q2 was near pre-pandemic levels, but those gains were largely offset by a lower percentage of 90-day prescriptions and lower prescription refill rates. We expect that recovery in the dermatology segment will continue to progress over the coming quarters. Looking forward, we are preparing for the anticipated approval of the mild-to-moderate psoriasis indication in the U.S., and continued geographic expansion, including the launch in China.
Enbrel (etanercept) sales decreased 8% year-over-year for the second quarter, primarily driven by lower net selling price and unfavorable changes in estimated sales deductions. On a year-over-year basis volumes declined 1%. Going forward, we expect net selling price to continue to decline year-over-year.
AMGEVITA (adalimumab) sales increased 73% year-over-year for the second quarter, primarily driven by volume growth. AMGEVITA continued to be the most prescribed adalimumab biosimilar in Europe.
LUMAKRASTM (sotorasib) has been well received by the oncology community. LUMAKRAS has been added to the National Comprehensive Cancer Network (NCCN) guidelines and unaided awareness among oncologists has increased significantly since launch. KRAS testing of patients with metastatic non-small cell lung cancer (NSCLC) now stands at approximately 70%, driven by increased next generation sequencing (NGS) utilization and KRAS G12C educational efforts, and 46 of the 50 top testing laboratories are now identifying the KRAS G12C mutation as actionable in their lab reports.
KYPROLIS (carfilzomib) sales increased 11% year-over-year for the second quarter, primarily driven by volume growth and net selling price. For the remainder of the year, we expect continued growth from Kyprolis use in combination with CD38 antibodies.
XGEVA (denosumab) sales increased 12% year-over-year for the second quarter, driven by volume growth as the segment recovered from the earlier effects of the pandemic.
Vectibix (panitumumab) sales increased 23% year-over-year for the second quarter, driven by 20% volume growth that benefited from increased shipments to Takeda, our partner in Japan. We expect lower demand from Takeda in the third quarter.
Nplate (romiplostim) sales increased 27% year-over-year for the second quarter, driven by 17% volume growth and higher inventory levels.
BLINCYTO (blinatumomab), our BiTE immunotherapy, sales increased 16% year-over-year for the second quarter, driven by 18% volume growth as we benefited from broader adoption in the community hospital setting.
MVASI sales increased 71% year-over-year for the second quarter, driven by strong volume growth, partially offset by lower net selling price. In the U.S., MVASI continued to hold leading volume share with 50% of the bevacizumab segment in the quarter. Sales were flat quarter-over-quarter as volume growth was offset by unfavorable changes to estimated sales deductions. Going forward on a sequential basis, we expect continued worldwide volume growth to be more than offset by declines in net selling price due to increased competition.
KANJINTI sales increased 27% year-over-year for the second quarter, primarily driven by volume growth, partially offset by lower net selling price. In the U.S., KANJINTI continued to hold leading volume share with 42% of the trastuzumab segment in the quarter. Sales decreased 3% quarter-over-quarter, primarily driven by unfavorable changes to estimated sales deductions. Going forward, we expect sales to decline sequentially in the second half of the year driven by net selling price.
Neulasta (pegfilgrastim) sales decreased 18% year-over-year for the second quarter, driven by declines in both net selling price and volume, partially offset by a $75 million year-over-year benefit from favorable changes in reimbursement mix, resulting from the comparison of a $39 million favorable adjustment to estimated sales deductions in the quarter to a $36 million unfavorable adjustment in the second quarter last year. In the long-acting granulocyte colony-stimulating factor (G-CSF) segment, Neulasta Onpro continued to be the preferred choice for physicians and patients with volume share of 52% in the quarter. The most recent published Average Selling Price for Neulasta in the U.S. declined 35% year-over-year and 12% quarter-over-quarter. Going forward, we expect increased competition to result in further declines in net selling price.
NEUPOGEN (filgrastim) sales increased 4% year-over-year for the second quarter, driven by favorable changes in estimated sales deductions, partially offset by volume declines.
EPOGEN (epoetin alfa) sales decreased 19% year-over-year for the second quarter, driven by volume declines and lower net selling price resulting from our contractual commitment with DaVita.
Aranesp (darbepoetin alfa) sales decreased 5% year-over-year for the second quarter, driven by lower net selling price due to competition.
Parsabiv (etelcalcetide) sales decreased 62% year-over-year for the second quarter, driven by volume declines. With Parsabiv’s inclusion in the U.S. end-stage renal disease (ESRD) bundled payment system, dialysis clinics rapidly implemented new treatment protocols, switching patients from Parsabiv to generic oral cinacalcet. As a result, we expect a 50-60% year-over-year Parsabiv sales decline in 2021. For patients on hemodialysis, Parsabiv is the only IV-administered calcimimetic that treats secondary hyperparathyroidism and provides the opportunity to reduce patient pill burden.
Sensipar/Mimpara (cinacalcet) sales decreased 70% year-over-year for the second quarter, primarily driven by volume declines in response to generic competition.
Operating Expense, Operating Margin and Tax Rate Analysis

On a GAAP basis:

Total Operating Expenses increased 47% primarily driven by the recent acquisition of Five Prime Therapeutics. Cost of Sales margin increased 1.6 percentage points primarily due to product mix, including COVID-19 antibody shipments to Eli Lilly and Company (Lilly) that began this quarter, profit share and royalties, partially offset by lower amortization expense from acquisition-related assets. Research & Development (R&D) expenses increased 12% primarily due to higher research and early pipeline spend and late-stage development program spend, including our recent business development activities. Acquired IPR&D expenses in 2021 were driven by the Five Prime Therapeutics acquisition. Selling, General & Administrative (SG&A) expenses increased 7% driven by marketed product support due to increased customer engagement in response to the pandemic recovery and investment in new product launches.
Operating Margin as a percentage of product sales decreased 25.8 percentage points to 13.5%.
Tax Rate increased 5.6 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 15%. Cost of Sales margin increased 4.1 percentage points primarily due to product mix, including COVID-19 antibody shipments to Lilly that began this quarter, profit share and royalties. R&D expenses increased 11% primarily due to higher research and early pipeline spend and late-stage development program spend, including our recent business development activities. SG&A expenses increased 6% driven by marketed product support due to increased customer engagement in response to the pandemic recovery and investment in new product launches.
Operating Margin as a percentage of product sales decreased 4.1 percentage points to 50.9%.
Tax Rate decreased 1.0 percentage points primarily driven by a prior year change in foreign loss utilization.
Cash Flow and Balance Sheet

The Company generated $1.7 billion of free cash flow in the second quarter of 2021 versus $2.7 billion in the second quarter of 2020, driven by a difference in the timing of tax payments.
The Company’s second quarter 2021 dividend of $1.76 per share was declared on March 3, 2021, and was paid on June 8, 2021, to all stockholders of record as of May 17, 2021, representing a 10% increase from 2020.
During the second quarter, the Company repurchased 6.5 million shares of common stock at a total cost of $1.6 billion. At the end of the second quarter, the Company had $3.9 billion authorization remaining under its stock repurchase program.
Cash and investments totaled $8.1 billion and debt outstanding totaled $32.8 billion as of June 30, 2021.
2021 Guidance

For the full year 2021, the Company now expects:

Total revenues in the range of $25.8 billion to $26.6 billion, unchanged from previous guidance.
On a GAAP basis, EPS in the range of $8.84 to $9.90 and a tax rate in the range of 13.0% to 14.5%.
Previously, the Company expected GAAP EPS in the range of $9.11 to $10.71 and a tax rate in the range of 14.0% to 15.5%.
On a non-GAAP basis, EPS in the range of $16.00 to $17.00, unchanged from previous guidance and a tax rate in the range of 13.5% to 14.5%, also unchanged from previous guidance.
Capital expenditures to be approximately $900 million.
Share repurchases at the upper end of our previous guidance of $3.0 billion to $5.0 billion.
Second Quarter Product and Pipeline Update

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

LUMAKRAS

In May, the U.S. Food and Drug Administration (FDA) approved LUMAKRAS for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic NSCLC, as determined by an FDA-approved test, who have received at least one prior systemic therapy. LUMAKRAS received accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Regulatory reviews continue in Europe, Japan and other jurisdictions.
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.
Primary results from the Phase 2 monotherapy study in patients with advanced KRAS G12C-mutated colorectal cancer (CRC) have been submitted for publication.
Additional data from the Phase 1/2 CodeBreaK 100 monotherapy study in advanced NSCLC have been accepted for presentation at the World Conference on Lung Cancer in September, including biomarker analyses and post hoc analyses of efficacy and safety in patients with stable brain metastases. Enrollment continues in a cohort of patients with active brain metastases in the CodeBreaK 101 study.
Exploration of LUMAKRAS in multiple Phase 1b combination cohorts continues to progress.
Initial data from LUMAKRAS in combination with Vectibix in patients with advanced KRAS G12C-mutated CRC have been accepted for presentation at the European Society for Medical Oncology Congress in September.
Initial data from LUMAKRAS in combination with a mitogen-activated protein kinase kinase (MEK) inhibitor and LUMAKRAS in combination with an oral EGFR inhibitor are planned to be submitted for presentation at a Q4 2021 medical conference.
The Company is collaborating with Novartis on a LUMAKRAS combination study with their SHP-2 inhibitor TNO155. A cohort is expected to initiate in the CodeBreaK 101 study in Q3 2021. Enrollment continues in a combination cohort with Revolution Medicine’s SHP-2 inhibitor RMC-4630.
Initiation of a Phase 2 study is planned for Q3 2021 in first-line patients with KRAS G12C mutated NSCLC whose tumors express < 1% programmed death-ligand 1 (PD-L1) and/or serine/threonine kinase 11 (STK11) mutations.
Data from the Phase 2 monotherapy study in patients with KRAS G12C-mutated solid tumors other than NSCLC and CRC are expected in H1 2022.
BLINCYTO

In June, the European Commission approved an expanded indication for the use of BLINCYTO in the treatment of pediatric patients aged 1 year or older with high-risk first relapsed Philadelphia chromosome negative CD19 positive B-precursor acute lymphoblastic leukemia as part of the consolidation therapy.
Bemarituzumab

The Company has initiated discussions with regulators on the Phase 3 study design for bemarituzumab, a first-in-class anti-fibroblast growth factor receptor 2b (FGFR2b) antibody for the treatment of patients with human epidermal growth factor receptor 2 (HER2) negative, FGFR2b-positive gastric and gastroesophageal junction cancer. Initiation of the registrational program is planned for Q4 2021.
Bemarituzumab has been granted Breakthrough Therapy Designation by the FDA as first-line treatment for patients with at least 10% FGFR2b overexpression and HER2-negative metastatic and locally advanced gastric and gastroesophageal adenocarcinoma in combination with modified FOLFOX6 (fluoropyrimidine, leucovorin, and oxaliplatin).
Planning is underway for bemarituzumab clinical studies in other solid tumors, including squamous NSCLC.
Acapatamab (AMG 160)

A dose expansion cohort of acapatamab, a half-life extended (HLE) BiTE molecule targeting prostate-specific membrane antigen (PSMA), has completed enrollment 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 has initiated for patients with NSCLC expressing PSMA.
A master protocol evaluating combinations of acapatamab with AMG 404, an anti-programmed cell death 1 (PD-1) antibody, or the novel hormone therapies enzalutamide or abiraterone, continues to enroll patients with earlier-line mCRPC.
Tarlatamab (AMG 757)

The Company has begun planning a potentially pivotal Phase 2 study and will initiate discussions with regulators for tarlatamab, an HLE BiTE molecule targeting delta-like ligand 3 (DLL3), in patients with relapsed or refractory small cell lung cancer.
A Phase 1b study of tarlatamab has begun recruiting patients with neuroendocrine prostate cancer.
A Phase 1b study of tarlatamab in combination with AMG 404 is planned to initiate in Q3 2021 for patients with small cell lung cancer.
Additional Phase 1 Oncology Programs

AMG 509, a bivalent T-cell engager XmAb 2+1 antibody targeting six transmembrane epithelial antigen of the prostate 1 (STEAP1) was recently granted Fast Track designation by the FDA and continues to enroll patients with mCRPC.
Pavurutamab (AMG 701), an HLE BiTE molecule targeting B-cell maturation antigen (BCMA), has resumed enrolling patients with relapsed or refractory multiple myeloma.
AMG 330, a BiTE molecule targeting CD33, continues to enroll patients with acute myeloid leukemia.
Enrollment has been paused in the Phase 1 study of AMG 427, a BiTE molecule targeting fms-like tyrosine kinase 3 (FLT3) for patients with acute myeloid leukemia.
HLE BiTE molecules AMG 199 and AMG 910, targeting mucin 17 (MUC17) and claudin 18.2 (CLDN18.2), respectively, continue to enroll patients with gastric and gastroesophageal junction cancer.
AMG 176, a small molecule inhibitor of myeloid cell leukemia 1 (MCL-1), continues to enroll patients with hematologic malignancies.
AMG 256, a multispecific interleukin-21 receptor agonist, continues to enroll patients with PD-1 positive solid tumors.
Tezepelumab

In July, the FDA accepted the Biologics License Application and granted Priority Review for tezepelumab for the treatment of asthma. Regulatory reviews are also underway in the EU and Japan.
A Phase 3 study has begun enrolling 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.
Otezla

In May, the Company announced that the FDA accepted the supplemental New Drug Application for Otezla for the treatment of adults with mild-to-moderate plaque psoriasis who are candidates for phototherapy or systemic therapy. The FDA has assigned a Prescription Drug User Fee Act action date of December 19, 2021.
Phase 3 planning is underway for Otezla for the treatment of Japanese patients with palmoplantar pustulosis.
The Company has stopped enrollment in the Otezla arms of ongoing platform trials designed to evaluate the efficacy and safety of potential treatments for patients hospitalized with COVID-19.
AMG 451 / KHK4083

The Company expects to commence discussions with regulators soon for AMG 451, an anti-OX40 monoclonal antibody, for the treatment of atopic dermatitis, with Phase 3 development anticipated to begin in H1 2022.
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, is enrolling patients with SLE.
Data from a Phase 1b SLE study have been submitted to a Q4 2021 medical conference.
A Phase 2 study of efavaleukin alfa is planned to initiate in H2 2021 for patients with ulcerative colitis.
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

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 in patients with elevated lipoprotein(a) are expected in H1 2022 with publication expected in H2 2022.
Aimovig

In June, the Japanese Ministry of Health, Labour and Welfare granted marketing approval for Aimovig for the suppression of onset of migraine attacks in adults.
Biosimilars

A Phase 3 study of ABP 654, a biosimilar candidate to STELARA (ustekinumab), has completed enrollment.
A Phase 3 study of ABP 938, a biosimilar candidate to EYLEA (aflibercept) continues to enroll patients.
A Phase 3 study of ABP 959, a biosimilar candidate to SOLIRIS (eculizumab), is ongoing.
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.
XmAb is a registered trademark of Xencor, Inc.

U.S. Manufacturing Facilities

In anticipation of future demand for our medicines, we will invest approximately $1 billion to build two new manufacturing facilities – a packaging plant in Ohio and a drug substance plant in North Carolina. Both of these facilities will be built faster and at a lower cost than traditional plants, and both also will utilize cutting-edge technologies to be more efficient and environmentally friendly than traditional plants.

U.S. Tax Petition

In July 2021, we filed a petition in the U.S. Tax Court to contest notices of deficiencies received from the IRS during the quarter for 2010, 2011 and 2012. These notices seek to increase our U.S. taxable income by an amount that would result in additional federal tax of approximately $3.6 billion, plus interest. Any additional tax that could be imposed would be reduced by up to approximately $900 million of repatriation tax previously accrued on our foreign earnings. We firmly believe that the IRS’s positions in the notices are without merit and we will vigorously contest the notices through the judicial process.

Non-GAAP Financial Measures

In this news release, management has presented its operating results for the second quarters of 2021 and 2020, in accordance with U.S. Generally Accepted Accounting Principles (GAAP) and on a non-GAAP basis. In addition, management has presented its full year 2021 EPS and tax rate guidance in accordance with GAAP and on a non-GAAP basis. These non-GAAP financial measures are computed by excluding certain items related to acquisitions, restructuring and certain other items from the related GAAP financial measures. Reconciliations for these non-GAAP financial measures to the most directly comparable GAAP financial measures are included in the news release. Management has also presented Free Cash Flow (FCF), which is a non-GAAP financial measure, for the second quarters of 2021 and 2020. FCF is computed by subtracting capital expenditures from operating cash flow, each as determined in accordance with GAAP.

The Company believes that its presentation of non-GAAP financial measures provides useful supplementary information to and facilitates additional analysis by investors. The Company uses certain non-GAAP financial measures to enhance an investor’s overall understanding of the financial performance and prospects for the future of the Company’s ongoing business activities by facilitating comparisons of results of ongoing business operations among current, past and future periods. The Company believes that FCF provides a further measure of the Company’s liquidity.

Beginning January 1, 2021, we began to exclude the gains and losses on our investments in equity securities from our non-GAAP measures that are recorded to Other income (expense). This exclusion will not apply to our share of the earnings and losses of our strategic investments in corporations accounted for under the equity method of accounting, such as our investment in BeiGene. The Company will be excluding gains and losses from equity investments for the purpose of calculating the non-GAAP financial measures presented because the Company believes the results of such gains and losses are not representative of our normal business operations. We are making this change beginning in 2021 because, as we have increased our investments in these companies, we recognized that the resulting variability can impede comparability between periods of our financial performance for our ongoing business operations. 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.

The Company uses the non-GAAP financial measures set forth in the news release in connection with its own budgeting and financial planning internally to evaluate the performance of the business, including to allocate resources and to evaluate results relative to incentive compensation targets. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP.

Curis Reports Second Quarter 2021 Financial Results

On August 3, 2021 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported its financial results for the second quarter ended June 30, 2021 (Press release, Curis, AUG 3, 2021, View Source [SID1234585601]).

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"In the second quarter of 2021, we continued executing on planned milestones across our pipeline of next generation targeted cancer therapies designed to meaningfully improve and extend patients’ lives. We drove important progress with our first-in-class, small molecule IRAK4 inhibitor CA-4948, expanding our clinical trials into nine distinct patient populations across AML/MDS and B cell cancers. In June, we presented updated data from our Phase 1/2 relapsed/refractory (R/R) AML/MDS study at EHA (Free EHA Whitepaper), which showcased continued improvements in clinical activity and robust tolerability in extremely fragile patients," said James Dentzer, President and Chief Executive Officer of Curis. "At the 300mg BID dose we are seeing clear anticancer activity and are achieving pharmacokinetic (PK) exposure in patients that correlates to 98% inhibition in preclinical models. In addition, we are encouraged by a predictable and manageable safety profile with no dose-limiting toxicities related to myelosuppression and no overlapping dose-limiting toxicities with existing anticancer therapies planned for dosing in combination with CA-4948. The data at EHA (Free EHA Whitepaper) also highlighted that we have identified a subset of patients with specific mutations that may make their disease highly amenable to CA-4948 monotherapy; all 4 of 4 evaluable patients with spliceosome or FLT3 mutations experienced an objective response. With further expansion of this group of patients, we may be able to identify a rapid path to regulatory approval for CA-4948. We also reported that even in patients without these specific mutations, CA-4948 demonstrated consistent tumor burden reduction, providing opportunities for combination development in a broader population. We expect to begin enrolling patients later this year in combination therapy evaluating CA-4948 with azacitidine or venetoclax. In total, we are exploring CA-4948 in four distinct cohorts in AML/MDS in addition to lower risk MDS, which is the subject of a separate Investigator Sponsored Trial (the LUCAS IST). We are also exploring CA-4948 in combination with ibrutinib across four additional cohorts in B cell cancers."

Mr. Dentzer added, "Lastly, we are also pleased with the continuing dose escalation in our ongoing Phase 1 study of CI-8993, our first-in-class monoclonal anti-VISTA antibody for the treatment of patients with R/R solid tumors.

"We look forward to providing an update on enrollment progress for all studies, including data updates for the spliceosome cohort in AML/MDS and the VISTA study, later this year."

Second Quarter 2021 and Recent Operational Highlights

Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):

At EHA (Free EHA Whitepaper), Curis presented interim data from the ongoing Phase 1/2 study of CA-4948 monotherapy in patients with R/R AML/MDS. The data were as of April 30, 2021 (the cut-off date) and were consistent with previously announced findings, including:
Objective responses observed in 4 of 4 (100%) evaluable patients in the enriched population of patients with a targeted mutation (spliceosome or FLT3 mutation); these enriched populations will be studied in monotherapy expansion cohorts
Marrow blast reductions, or maintenance of blast level for patients in the normal range at baseline, in 9 of 11 (82%) patients without spliceosome or FLT3 mutations; this broader population will be studied in combination therapy in expansion
Efficacy confirmed with positive safety findings:
Clear efficacy observed at 300mg twice daily dosing
MTD not exceeded until 500mg BID
No overlap in dose-limiting toxicities with azacitidine and venetoclax, which are planned for combination with CA-4948
No dose-limiting toxicities related to myelosuppression
Dose-limiting side effect at higher doses consists of uncomplicated rhabdomyolysis (elevated CPK and muscle soreness), which was manageable, quickly and easily detected, readily reversible, and did not limit further treatment at a reduced dose level
Of note, those patients who experienced rhabdomyolysis at higher doses generally had predisposing factors, such as concomitant administration of statins or strenuous exercise
Alongside the EHA (Free EHA Whitepaper) data, Curis determined 300mg BID to be the recommended Phase 2 dose as it demonstrated clear anticancer activity, a manageable and predictable safety profile and PK exposure in patients that correlates to near complete target engagement in preclinical models (98%)
Also at EHA (Free EHA Whitepaper), Curis reported non-clinical data demonstrating synergistic antitumor activity of CA-4948 in combination with azacitidine and venetoclax in leukemia cells, providing supportive rationale for clinical studies evaluating of combination therapy for AML/MDS patients
The Phase 1/2 study of CA-4948 in AML/MDS was expanded to include both a monotherapy dose expansion and a combination dose escalation:
Monotherapy:
R/R MDS patients with/without spliceosome mutation
R/R AML patients with/without FLT3 mutation
Combination therapy:
CA-4948 + azacitidine, for patients naïve to HMA
CA-4948 + venetoclax, for patients naïve to venetoclax
The Phase 1/2 study of CA-4948 in B cell cancers was expanded to include a combination dose escalation and dose expansion across four patient cohorts
Combination therapy:
BTKi-naïve patients with marginal zone lymphoma
BTKi-naïve patients with primary CNS lymphoma
BTKi-naïve patients with ABC-DLBCL
Patients who have developed adaptive resistance to ibrutinib
Immuno-oncology, CI-8993 (anti-VISTA antibody; ImmuNext collaboration):

Enrollment is continuing in the ongoing Phase 1 dose escalation study of CI-8993, Curis’s first-in-class monoclonal anti-VISTA antibody for the treatment of R/R solid tumors
Curis expects to report initial safety data from this trial later in 2021
In June 2021, Curis hosted a virtual symposium entitled: VISTA: A New Immune Checkpoint in Cancer, Autoimmunity, and Beyond, gathering thought-leaders in industry and academia to discuss emerging understanding and opportunities surrounding the immune checkpoint
Upcoming Planned Milestones

In the second half of 2021, initiate dosing in the combination stage of the Phase 1/2 study of CA-4948 plus azacitidine and CA-4948 plus venetoclax
By year-end 2021, report additional clinical data from the Phase 1/2 monotherapy study of CA-4948 in AML/MDS patients with spliceosome mutations that result in aberrant splicing of oncogenic IRAK4-L
By year-end 2021, report initial safety data from the ongoing Phase 1 monotherapy study of CI-8993 for the treatment of R/R solid tumors
In the first half of 2022, report initial data at a medical meeting from the ongoing Phase 1/2 combination study of CA-4948 plus ibrutinib in patients with B cell cancers
Second Quarter 2021 Financial Results

For the second quarter of 2021, Curis reported a net loss of $10.8 million or $0.12 per share on both a basic and diluted basis, as compared to a net loss of $6.7 million, or $0.17 per share on both a basic and diluted basis for the same period in 2020. Curis reported a net loss of $20.8 million, or $0.23 per share on both a basic and diluted basis, for the six months ended June 30, 2021, as compared to a net loss of $16.4 million, or $0.44 per share on both a basic and diluted basis, for the same period in 2020.

Revenues for the second quarter of 2021 and 2020 were $2.3 million and $2.4 million, respectively. Revenues for the six months ended June 30, 2021 were $4.5 million, as compared to $5.1 million for the same period in 2020. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses for the second quarter of 2021 were $12.9 million, as compared to $7.8 million for the same period in 2020. Operating expenses for the six months ended June 30, 2021 were $23.9 million, as compared to $19.0 million for the same period in 2020, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.1 million for the second quarter of 2021, as compared to $0.1 million for the same period in 2020. Cost of royalty revenues for the six months ended June 30, 2021 were $0.2 million, as compared to $0.2 million for the same period in 2020.

Research and Development Expenses. Research and development expenses were $8.8 million for the second quarter of 2021 as compared to $5.3 million for the same period in 2020. The increase in direct research and development expenses for the quarter is primarily attributable to increased clinical and manufacturing costs for our programs. Additionally, employee related costs increased by $1.4 million, primarily attributable to increased stock compensation and personnel costs as a result of additional headcount. Research and development expenses were $15.5 million for the six months ended June 30, 2021 as compared to $12.8 million for the same period in 2020.

General and Administrative Expenses. General and administrative expenses were $4.1 million for the first second quarter of 2021, as compared to $2.4 million for the same period in 2020. The increase in general administrative expense was driven primarily by higher costs for stock-based compensation, personnel, professional and consulting services, and legal services. General and administrative expenses were $8.2 million for the six months ended June 30, 2021, as compared to $6.0 million for the same period in 2020.

Other Expense, Net. For the second quarter of 2021 and 2020, net other expense was $0.2 million and $1.3 million, respectively. Net other expense primarily consisted of imputed interest expense related to future royalty payments, partially offset in the second quarter of 2021 by a gain of $0.9 million related to extinguishment of debt. Net other expense was $1.3 million for the six months ended June 30, 2021, as compared to $2.5 million for the same period in 2020.

As of June 30, 2021, Curis’s cash, cash equivalents and investments totaled $160.7 million, and there were approximately 91.6 million shares of common stock outstanding. Curis expects that its existing cash, cash equivalents and investments should enable it to maintain its planned operations into 2024.

Conference Call Information

Curis management will host a conference call today, August 3, 2021, at 4:30 p.m. ET, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412-317-5252 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.

Sprint Bioscience licenses the cancer drug program Vps34 to Deciphera Pharmaceuticals

On August 3, 2021 Sprint Bioscience AB (publ) reported that the company has licensed its cancer drug program Vps34 to the US pharmaceutical company Deciphera Pharmaceuticals (Press release, Sprint Bioscience, AUG 3, 2021, View Source [SID1234585600]). The license agreement covers exclusive global rights to the program. The agreement has a total potential value of up to USD 277 million, plus additional tiered percentage royalties ranging from the mid-single-digits to the low-double-digits on the sales of a future drug from the program, if approved. On signing of the agreement, Sprint Bioscience will receive an upfront payment of USD 4 million, which will be entered into the accounts for the third quarter of 2021.

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The chosen partner, Deciphera Pharmaceuticals Inc., founded in 2003, is a pharmaceutical company focused on delivering important new medicines to patients for the treatment of cancer. The company has an approved product on the market in multiple geographies, as well as three product candidates in clinical development. The company is listed on the Nasdaq Global Select Market with a market cap of approximately USD 2 billion. With a clear focus on improving the lives of people living with cancer and demonstrated expertise in bringing a new drug to market, Deciphera is considered to be an extremely attractive partner for the Sprint Biosciences Vps34 program. Deciphera will assume full responsibility for resource allocation as it continues research efforts, and for taking the program to the clinic and further onto the market.

"Today’s announcement represents Deciphera’s focus to discover, develop, and deliver important new medicines to patients for the treatment of cancer, as well as an effort to complement our robust internal research programs with partnership opportunities such as this one. This Vps34 program fits well with our growing pipeline, and enhances our efforts to explore the potential for regulating autophagy in cancer," said Dan Flynn, Executive Vice President and Chief Scientific Officer (CSO) of Deciphera Pharmaceuticals.

Deciphera will now take over the program’s continued development and all associated costs.

"The licensing agreement with Deciphera Pharmaceuticals is the most comprehensive to date that Sprint Bioscience has signed and further proof of our ability to continuously generate groundbreaking programs with great commercial potential. We have an increasing number of programs in our portfolio where well-funded partners take over responsibility and all costs for continued development at an early stage, while we are eligible for milestone payments when the programs achieve predetermined goals. As the number of out-licensed programs increases and further develops, we create more and more opportunities for revenues. The total revenue potential from our active licensing agreements has now increased to approximately USD 747 million, plus royalty payments," said Erik Kinnman, CEO of Sprint Bioscience.

In addition to the upfront payment, Sprint Bioscience is entitled to payments linked to predefined milestones during the project’s further development, regulatory process and commercialization of a drug. With the upfront, the total value of these payments amounts up to USD 277 million.Should the program result in an approved drug, Sprint Bioscience is also entitled to tiered percentage royalties ranging from the mid-single-digits to the low-double-digits on the sales of a future drug from the program, if approved.

This disclosure contains information that Sprint Bioscience is obliged to make public pursuant to the EU Market Abuse Regulation (EU nr 596/2014). The information was submitted for publication, through the agency of the contact person, on 03-08-2021 22:15 CET.

EVERSANA Sponsors Patient Assistance & Access Programs 2021 Virtual Event

On August 3, 2021 EVERSANA and other industry leaders reported at the PAP – Patient Assistance & Access Programs 2021 Virtual Event August 17–19 as we discuss how to break through affordability barriers to streamline operational complexities, enhance patient access and optimize stakeholder collaboration (Press release, EVERSANA, AUG 3, 2021, View Source [SID1234585599]).

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Backed by 22 years of content, PAP is the premier event for influential stakeholders to collaborate on how to further access to care, understand current changes in policy and optimize PAPs for better patient outcomes. With a blend of live and on-demand content, this three-day event is designed to provide you with the latest industry updates and opportunities to expand your network and collaborate with a multi-stakeholder audience.

EVERSANA is proud to participate in this year’s session, "Industry Disruptors — Stay on the Pulse of Trends in Co-pay Exclusions, Accumulators and Maximizers," featuring Joe Bachstadt, Vice President of Patient Services Affordability.

Joe Bachstadt

Vice President, Patient Services Affordability

On Thursday, August 19, at 1 pm EST, Joe will discuss the:

Evolution and current landscape of co-pay accumulator and maximizer models.
Patient and business impact of accumulators in the ability to access drugs, as well as the legal challenges.
New workarounds entering the market that are adding pressure to manufacturer PAPs.