On May 6, 2021 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the first quarter of 2021 and provided an update on progress toward achieving key corporate objectives, as well as commercial, clinical and pipeline development milestones (Press release, Exelixis, MAY 6, 2021, View Source [SID1234579345]).

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"The Exelixis team made significant progress across all components of our business in the first quarter of 2021, and delivered top-line revenue growth following the U.S. approval of CABOMETYX in combination with OPDIVO for first-line renal cell carcinoma in late January," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. "We also executed on a number of key priorities throughout the quarter, including initiating phase 1 clinical development of XL102, our novel small molecule CDK7 inhibitor, and successfully filing an Investigational New Drug application with the FDA for XB002, our first antibody-drug conjugate. We continue to focus on key 2021 development and regulatory milestones, and expect to report top-line results in the second quarter from the phase 3 COSMIC-312 pivotal trial evaluating the combination of cabozantinib and atezolizumab as a first-line treatment in advanced hepatocellular carcinoma, and file up to three supplemental New Drug Applications for cabozantinib across multiple indications by year-end. I look forward to providing updates on our progress throughout the year as we further our efforts to build a leading multi-product oncology company aiming to deliver new therapies and treatment options for the patients we serve."

First Quarter 2021 Financial Results

Total revenues for the quarter ended March 31, 2021 were $270.2 million, compared to $226.9 million for the comparable period in 2020.

Total revenues for the quarter ended March 31, 2021 included net product revenues of $227.2 million, compared to $193.9 million for the comparable period in 2020. The increase in net product revenues was primarily related to an increase in sales volume that was partially driven by strong uptake for the combination therapy of CABOMETYX (cabozantinib) and OPDIVO (nivolumab) following approval by the U.S. Food and Drug Administration (FDA) in January of 2021.

Collaboration revenues, composed of license revenues and collaboration services revenues, were $43.0 million for the quarter ended March 31, 2021, compared to $33.0 million for the comparable period in 2020. The increase in collaboration revenues was primarily related to higher royalty revenues for the sales of cabozantinib outside of the U.S. generated by Exelixis’ collaboration partners, Ipsen Pharma SAS (Ipsen) and Takeda Pharmaceutical Company Limited (Takeda), and increases in development cost reimbursements.

Research and development expenses for the quarter ended March 31, 2021 were $159.3 million, compared to $101.9 million for the comparable period in 2020. The increase in research and development expenses was primarily related to increases in license and other collaboration costs, clinical trial costs, personnel expenses and stock-based compensation expense.

Selling, general and administrative expenses for the quarter ended March 31, 2021 were $102.4 million, compared to $62.9 million for the comparable period in 2020. The increase in selling, general and administrative expenses was primarily related to increases in personnel expenses, marketing costs, corporate giving and stock-based compensation expense, which was partially offset by a decrease in the Branded Prescription Drug Fee.

Provision for (benefit from) income taxes for the quarter ended March 31, 2021 was $(3.6) million, compared to $11.4 million for the comparable period in 2020, primarily due to the change in pre-tax income (loss).

GAAP net income for the quarter ended March 31, 2021 was $1.6 million, or $0.01 per share, basic and $0.00 per share, diluted, compared to GAAP net income of $48.6 million, or $0.16 per share, basic and $0.15 per share, diluted, for the comparable period in 2020.

Non-GAAP net income for the quarter ended March 31, 2021 was $28.5 million, or $0.09 per share, basic and diluted, compared to non-GAAP net income of $59.4 million, or $0.19 per share, basic and diluted, for the comparable period in 2020. Non-GAAP net income excludes stock-based compensation, adjusted for the related income tax effect.

Cash, cash equivalents, restricted cash equivalents and investments were $1.6 billion at March 31, 2021, compared to $1.5 billion at December 31, 2020.

Non-GAAP Financial Measures

To supplement Exelixis’ financial results presented in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Exelixis presents non-GAAP net income (and the related per share measures), which excludes from GAAP net income (and the related per share measures) stock-based compensation expense, adjusted for the related income tax effect for all periods presented.

Exelixis believes that the presentation of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors. In particular, Exelixis believes that these non-GAAP financial measures, when considered together with its financial information prepared in accordance with GAAP, can enhance investors’ and analysts’ ability to meaningfully compare Exelixis’ results from period to period, and to identify operating trends in Exelixis’ business. Exelixis has excluded stock-based compensation expense, adjusted for the related income tax effect, because it is a non-cash item that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. Exelixis also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate its business and to make operating decisions.

These non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. Exelixis encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP financial information and the reconciliation between these presentations, to more fully understand Exelixis’ business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.

2021 Financial Guidance

Exelixis is maintaining the following previously provided financial guidance for fiscal year 2021:

Total revenues

$1,150 million – $1,250 million

Net product revenues

$950 million – $1,050 million

Cost of goods sold

Approximately 5% – 6% of net product revenue

Research and development expenses (1)

$600 million – $650 million

Selling, general and administrative expenses (2)

$375 million – $425 million

Effective tax rate

20% – 22%

Cash and investments (3)(4)

$1.6 billion – $1.7 billion

(1)

Includes $45 million of non-cash stock-based compensation expense.

(2)

Includes $60 million of non-cash stock-based compensation expense.

(3)

This cash and investments guidance does not include any potential new business development activity.

(4)

Cash and Investments is composed of cash, cash equivalents, restricted cash equivalents and investments.

Cabozantinib Highlights

Cabozantinib Franchise Net Product Revenues and Royalties. Net product revenues generated by the cabozantinib franchise in the U.S. were $227.2 million during the first quarter of 2021, up 13 percent over the prior quarter, with net product revenues of $223.6 million from CABOMETYX and $3.6 million from COMETRIQ (cabozantinib). Exelixis earned $23.8 million in royalty revenues during the quarter ended March 31, 2021, pursuant to collaboration agreements with our partners, Ipsen and Takeda.

FDA Approves CABOMETYX in Combination with OPDIVO for Advanced Renal Cell Carcinoma (RCC). In January 2021, Exelixis announced the FDA approved its supplemental New Drug Application (sNDA) for CABOMETYX in combination with Bristol Myers Squibb’s OPDIVO as a first-line treatment of patients with advanced RCC. The approval is based on positive results of the CheckMate -9ER phase 3 pivotal trial, which met its primary endpoint of significantly improving progression-free survival (PFS) and secondary endpoints of overall survival (OS) and objective response rate (ORR), with a favorable tolerability profile versus sunitinib.

Cabozantinib Data Presentations Demonstrate Broad Anti-Tumor Activity at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Genitourinary Cancers Symposium (ASCO GU 2021). In February 2021, cabozantinib was the subject of multiple data presentations at ASCO (Free ASCO Whitepaper) GU 2021, held virtually from February 11-13. Presentations included: updated trial results with extended follow-up and patient-reported outcomes from the CheckMate -9ER trial demonstrating continued superior PFS, OS and ORR versus sunitinib across both the subgroup of 75 patients with sarcomatoid histology and the full study population, as well as significantly improved health-related quality of life; positive results from the SWOG S1500 trial ("PAPMET") of cabozantinib versus sunitinib in metastatic papillary RCC; positive findings from an international retrospective study of cabozantinib in RCC patients with brain metastases; and positive final results from the phase 1 trial sponsored and conducted by NCI-CTEP, including seven expansion cohorts, evaluating cabozantinib in combination with either nivolumab or nivolumab plus ipilimumab in patients with refractory metastatic genitourinary tumors.

FDA Grants Breakthrough Therapy Designation to Cabozantinib for the Treatment of Patients with Previously Treated Radioactive Iodine-Refractory Differentiated Thyroid Cancer (DTC). In February 2021, Exelixis announced that the FDA granted Breakthrough Therapy Designation to cabozantinib as a potential treatment for patients with DTC who progressed following prior therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). The FDA’s Breakthrough Therapy Designation aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases. The designation was based on an interim analysis of the COSMIC-311 phase 3 pivotal trial, in which cabozantinib demonstrated significant improvement in PFS.

Exelixis’ Partner Ipsen Receives European Commission (EC) Approval for CABOMETYX in Combination with OPDIVO as First-Line Treatment for Patients with Advanced RCC. In March 2021, Exelixis announced its partner Ipsen received approval from the EC for CABOMETYX in combination with OPDIVO as a first-line treatment for advanced RCC. The approval allows for the marketing of CABOMETYX in combination with OPDIVO in this indication in all 27 member states of the European Union, Norway, Iceland and Liechtenstein. The EC approval was based on the positive results of the phase 3 CheckMate -9ER pivotal trial.

Enrollment Completed in Phase 3 COSMIC-313 Pivotal Trial of Cabozantinib in Combination with Nivolumab and Ipilimumab Versus Nivolumab and Ipilimumab in Previously Untreated Advanced RCC. In March 2021, Exelixis announced that COSMIC-313, the phase 3 pivotal trial evaluating the triplet combination of cabozantinib, nivolumab and ipilimumab versus the combination of nivolumab and ipilimumab in patients with previously untreated advanced intermediate- or poor-risk RCC, completed enrollment. COSMIC-313 is a multicenter, randomized, double-blind, controlled phase 3 pivotal trial that enrolled approximately 840 patients globally. The primary endpoint of the trial is PFS, and additional endpoints include OS and ORR.

Cabozantinib Data Presentations at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting. Cabozantinib will be the subject of 20 presentations at this year’s meeting, which has again adopted a virtual format as a result of the COVID-19 pandemic. Data presentations will include results from the COSMIC-311 and CheckMate -9ER phase 3 pivotal trials, as well as updates from externally sponsored studies.

Pipeline Highlights

Initiation of Phase 1 Clinical Trial Evaluating XL102 as a Single Agent and in Combination with Other Anti-Cancer Agents in Patients with Advanced or Metastatic Solid Tumors. In January 2021, Exelixis announced the initiation of a phase 1 clinical trial evaluating the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of XL102, both as a single agent and in combination with other anti-cancer agents, for the treatment of patients with inoperable, locally advanced or metastatic solid tumors. XL102 (formerly AUR102) is a potent, selective and orally bioavailable inhibitor of cyclin-dependent kinase 7 (CDK7) that Exelixis in-licensed from Aurigene Discovery Technologies Limited in December 2020 under the companies’ July 2019 collaboration agreement.

FDA Accepts Investigational New Drug Application (IND) for Tissue Factor-Targeting Antibody-Drug Conjugate (ADC) XB002 in Patients with Advanced Solid Tumors. In April 2021, Exelixis announced that the FDA accepted its IND to evaluate the safety, tolerability, pharmacokinetics and preliminary antitumor activity of XB002 in patients with advanced solid tumors. As a next-generation tissue factor-targeting ADC, XB002 (formerly ICON-2) has the potential for an improved therapeutic index and may provide a favorable safety profile compared with earlier-generation tissue factor-targeting ADCs. Exelixis in-licensed XB002 from Iconic Therapeutics, Inc. in December 2020 under the companies’ May 2019 collaboration agreement.

Corporate Updates

Exelixis Outlines Key Priorities and Anticipated Milestones for 2021. In January 2021, Exelixis announced its key priorities and anticipated milestones for 2021, including: the commercial launch of CABOMETYX in combination with OPDIVO as a first-line treatment of patients with advanced RCC; potential sNDA submissions for CABOMETYX in DTC, hepatocellular carcinoma and metastatic castration-resistant prostate cancer; progress and enrollment in the COSMIC and CONTACT clinical studies evaluating cabozantinib as a single agent or in combination with immune checkpoint inhibitors (ICIs); expanded clinical development activities for XL092; and multiple INDs for preclinical assets. Exelixis presented the details of its key priorities and anticipated milestones at the 39th Annual J.P. Morgan Healthcare Conference, which was held virtually from January 11-14.

Exelixis and Adagene Inc. (Adagene) Enter into Collaboration and License Agreement to Develop Novel Masked ADC Therapies with Improved Safety and Efficacy Profiles. In February 2021, Exelixis and Adagene announced a collaboration and license agreement under which Exelixis will utilize Adagene’s SAFEbody technology platform to generate masked versions of monoclonal antibodies (mAbs) from Exelixis’ growing preclinical pipeline for the development of SAFEbodies or other innovative biologics against Exelixis-nominated targets. Under the terms of the agreement, Exelixis received an exclusive, worldwide license to develop and commercialize any potential ADC products generated by Adagene with respect to an initial target, as well as a second target Exelixis may nominate during the collaboration term.

Exelixis Enters into Exclusive License Agreement with WuXi Biologics (WuXi Bio) to Support Further Expansion of its Growing Oncology Biologics Pipeline. In March 2021, Exelixis announced an exclusive license agreement with WuXi Bio to support the continued expansion of Exelixis’ oncology biologics pipeline. Under the terms of the agreement, Exelixis received an exclusive license to a panel of mAbs to a validated target, discovered based on WuXi Bio’s integrated technology platforms, for the development of ADC, bispecific, and certain other novel tumor-targeting biologics applications.

Exelixis Enters Clinical Trial Collaboration and Supply Agreement with Merck KGaA, Darmstadt, Germany (Merck KGaA) and Pfizer Inc. (Pfizer) to Evaluate XL092 and Avelumab (BAVENCIO) in Various Forms of Locally Advanced or Metastatic Urothelial Carcinoma (UC). In March 2021, Exelixis announced a clinical trial collaboration and supply agreement with Merck KGaA and Pfizer for the ongoing phase 1b dose escalation study STELLAR-001 (previously called "XL092-001"), adding three new cohorts that will evaluate the safety and tolerability of XL092, Exelixis’ novel next generation tyrosine kinase inhibitor, in combination with avelumab, an anti-PD-L1 ICI, in patients with locally advanced or metastatic UC.

Exelixis Expands its Biotherapeutics Portfolio with Acquisition of Anti-Müllerian Hormone Receptor 2 (AMHR2) Program from GamaMabs Pharma SA (GamaMabs). In May 2021, Exelixis entered into an asset purchase agreement with GamaMabs under which Exelixis will, upon the closing of the asset purchase and subject to certain conditions, acquire all rights, title and interest in GamaMabs’ antibody program directed at AMHR2, a novel oncology target with relevance in multiple forms of cancer. Exelixis believes applying its ADC capabilities to GamaMabs’ panel of antibodies against AMHR2 could yield potential additions to the company’s biotherapeutics portfolio.

Exelixis Receives Notice of Paragraph IV Certifications. In May 2021, Exelixis received a notice letter from Teva Pharmaceuticals USA, Inc. (Teva) regarding an Abbreviated New Drug Application Teva submitted to the FDA, requesting approval to market a generic version of CABOMETYX tablets. Teva’s notice letter included a Paragraph IV certification with respect to three of Exelixis’ Orange Book-listed patents: U.S. Patent Nos. 9,724,342 (formulations), 10,034,873 (methods of treatment) and 10,039,757 (methods of treatment), which expire in 2033, 2031 and 2031, respectively. Teva’s notice letter did not provide a Paragraph IV certification against any additional CABOMETYX patents. Exelixis intends to continue to vigorously defend its cabozantinib intellectual property estate.

Basis of Presentation

Exelixis has adopted a 52- or 53-week fiscal year that generally ends on the Friday closest to December 31st. For convenience, references in this press release as of and for the fiscal periods ended April 2, 2021, January 1, 2021 and April 3, 2020 are indicated as being as of and for the periods ended March 31, 2021, December 31, 2020, and March 31, 2020, respectively.

Conference Call and Webcast

Exelixis management will discuss the company’s financial results for the first quarter of 2021 and provide a general business update during a conference call beginning at 5:00 p.m. EDT / 2:00 p.m. PDT today, Thursday, May 6, 2021.

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 855-793-2457 (domestic) or 631-485-4921 (international) and provide the conference call passcode 6139476 to join by phone.

A telephone replay will be available until 8:00 p.m. EDT on May 8, 2021. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 6139476. A webcast replay will also be archived on www.exelixis.com for one year.

On May 6, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported presentations featuring TransCon PTH at two upcoming medical conferences: the 48th annual meeting of the European Calcified Tissue Society (ECTS), taking place virtually May 6-8, 2021 and the 23rd European Congress of Endocrinology taking place virtually May 22-26, 2021 (Press release, Ascendis Pharma, MAY 6, 2021, View Source [SID1234579344]).

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"We are excited to share the 26-week open-label extension (OLE) data from our phase 2 PaTH Forward Trial in adult patients with hypoparathyroidism with the European medical community," said Mark Bach, M.D., Ph.D., Senior Vice President of Endocrine Medical Sciences at Ascendis Pharma.

"These data provide evidence indicating that TransCon PTH has the potential to be a first line therapy as a hormone replacement in patients with hypoparathyroidism. The study was selected for oral presentations by both ECTS and ECE meeting organizers which we think underscores the significance of these results," continued Dr. Bach.

ECTS 2021: Presentation Details

Oral Presentation
Title Date/Time
Week 26 Results from the PaTH Forward Open-Label Extension Trial Support TransCon PTH as a Potential Hormone Replacement Therapy for Patients with Hypoparathyroidism Plenary Oral Presentations 2: Novel Aspects of Osteoporosis and Treatments
May 7, 2021
16:30 – 17:30 CEST
e-ECE 2021: Presentation Details

Oral Presentation
Title Date/Time
TransCon PTH as a Potential Hormone Replacement Therapy for Patients with Hypoparathyroidism: PaTH Forward Open-Label Extension Trial Week 26 Results Oral Communications 6: Calcium and Bone
May 25, 2021
14:30 – 15:30 CEST
The posters will be available on the Ascendis website under Selected Publications in the Pipeline section: View Source If you are a healthcare provider who would like more information, please contact: [email protected].

About TransCon Technology

TransCon refers to "transient conjugation." The proprietary TransCon platform is an innovative technology to create new therapies that are designed to optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and can be used systemically or locally.

On May 6, 2021 Acceleron Pharma Inc. (Nasdaq:XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the first quarter ended March 31, 2021 (Press release, Acceleron Pharma, MAY 6, 2021, View Source [SID1234579343]).

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"Acceleron made significant operational progress during the first quarter," said Habib Dable, President and Chief Executive Officer of Acceleron. "With the STELLAR Phase 3 trial already enrolling patients, we expect to initiate multiple mid- and late-stage clinical trials across our pipeline in pulmonary disease throughout 2021. We were thrilled to have data from the PULSAR Phase 2 trial of sotatercept in PAH recently featured in the New England Journal of Medicine. These data along with the results from our ongoing and planned Phase 3 trials will support our vision of establishing sotatercept as a backbone therapy for patients with all stages of PAH. We are looking forward to hosting a research and development day in late June to primarily discuss Acceleron’s plans and vision in rare pulmonary diseases with sotatercept in pulmonary hypertension and ACE-1334 in systemic sclerosis-associated interstitial lung disease, or SSc-ILD."

Added Mr. Dable: "In our hematology program, Acceleron and our partner, Bristol-Myers Squibb Company (Bristol Myers Squibb), are leveraging the key learnings from the early success of the commercial launch of REBLOZYL in the United States. We continue to transition from the initial bolus patient group earlier in the launch to underlying new patient demand. Bristol Myers Squibb expects launches in multiple additional countries across the globe this year as REBLOZYL receives reimbursement. Most importantly, assuming success in current and future potential indications associated with anemia, we continue to estimate that peak annual sales of REBLOZYL will surpass $4 billion."

Program Highlights

Pulmonary

Sotatercept: Pulmonary Arterial Hypertension (PAH)

Sotatercept is an investigational reverse-remodeling agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH.

In April, the New England Journal of Medicine published results from the PULSAR Phase 2 trial of sotatercept in patients with PAH.
The Company plans to present updates from the PULSAR and SPECTRA Phase 2 trials of sotatercept in patients with PAH at the American Thoracic Society (ATS) 2021 International Conference, held May 14-19.
The Company will also present preclinical research on the effects of a murine version of sotatercept in animal models of PAH and pulmonary hypertension (PH Group 2).
Acceleron plans to host a webcast and conference call for investors and analysts on May 19, 2021 to discuss highlights from the PULSAR and SPECTRA trial updates at the congress.
Enrollment is ongoing in the registrational STELLAR Phase 3 trial in patients with PAH.
The Company expects to have the HYPERION (newly diagnosed intermediate and high-risk patients) Phase 3 trial and the ZENITH (WHO Functional Class IV) Phase 3 trial in expanded PAH populations initiated by the second half of 2021.
ACE-1334: Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD)

ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2. ACE-1334 has shown robust anti-fibrotic activity in multiple preclinical models of fibrosis.

Acceleron expects to start a Phase 1b/Phase 2 study to evaluate the activity of ACE-1334 in patients with SSc-ILD in 2021.
Hematology

REBLOZYL (luspatercept-aamt):

REBLOZYL is the first and only approved erythroid maturation agent designed to promote late-stage red blood cell (RBC) production. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.

The Company recognized approximately $22.4 million in royalty revenue from approximately $112 million in net sales of REBLOZYL in the first quarter of 2021. This compares with approximately $23.0 million in royalty revenue from approximately $115 million in net sales of REBLOZYL in the fourth quarter of 2020.
In February, Acceleron and partner Bristol Myers Squibb announced that Health Canada approved REBLOZYL for the treatment of adult patients with transfusion-dependent anemia requiring at least two RBC units over 8 weeks resulting from very low- to intermediate-risk MDS who have ring sideroblasts and who have failed or are not suitable for erythropoietin-based therapy.
Bristol Myers Squibb initiated the INDEPENDENCE Phase 3 trial in patients with anemia-associated with myelofibrosis.
The Companies expect to present results from the BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta-thalassemia by the end of June 2021.
Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS, with topline results expected in 2022+.
Corporate Highlights

Acceleron is planning to host a research and development day on Tuesday, June 22, 2021 to primarily focus on its rare pulmonary disease pipeline and long-term vision.
Financial Results

Cash Position – Cash, cash equivalents and investments as of March 31, 2021 were $795.4 million, compared with $857.5 million as of December 31, 2020. Based on Acceleron’s current operating plan and projections, the Company believes that its current cash, cash equivalents and investments, along with the expected royalty revenue from REBLOZYL sales, will be sufficient to fund the Company’s projected operating requirements for the foreseeable future.
Revenue – Revenue for the first quarter of 2021 was $24.8 million, which includes $2.4 million of cost share revenue and $22.4 million of royalty revenue from net sales of REBLOZYL. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.
R&D Expenses – GAAP R&D expenses were $57.3 million for the first quarter of 2021. Non-GAAP R&D expenses were $48.2 million for the first quarter of 2021, excluding $8.2 million and $0.9 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
SG&A Expenses – GAAP SG&A expenses were $31.1 million for the first quarter of 2021. Non-GAAP SG&A expenses were $23.5 million for the first quarter of 2021, excluding $7.5 million and $0.1 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Net Loss – The Company’s GAAP net loss for the first quarter of 2021 was $63.5 million, or $1.05 per share. Non-GAAP adjusted net loss for the first quarter was $46.8 million, or $0.77 per share, excluding $15.6 million and $1.0 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Non-GAAP Financial Measures

Acceleron supplements its results of operations prepared in accordance with U.S. generally accepted accounting principles, or GAAP, with certain non-GAAP financial measures, including non-GAAP R&D expense, non-GAAP SG&A expense, adjusted net loss and adjusted net loss per share, that exclude stock-based compensation expense and depreciation and amortization expense. These results should not be viewed as a substitute for the Company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures provide investors with additional insight into underlying trends of the Company’s ongoing business, and are important in comparing current results with prior period results. Investors are cautioned that there are material limitations associated with the use of non-GAAP financial measures. In addition, other companies may report similarly titled non-GAAP measures, but calculate them differently, which reduces their usefulness as a comparative measure. In the reconciliation tables below, Acceleron presents these non-GAAP financial measures reconciled to their comparable GAAP financial measures.

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its first quarter 2021 financial results on May 6, 2021, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at www.acceleronpharma.com. To participate in the conference call, please dial 833-494-1483 (domestic) or 236-714-2620 (international) and reference code #5559908.

An archived version of the webcast will be available for replay on the Company’s website for approximately one year.

On May 6, 2021 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported first quarter 2021 financial results and provided a corporate update (Press release, Kura Oncology, MAY 6, 2021, View Source [SID1234579342]).

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"We believe KO-539 is well-positioned as a potentially best-in-class and first-in-class menin inhibitor," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "This confidence is supported by a growing body of clinical data, including compelling activity, a favorable safety and tolerability profile and a wide therapeutic window. As such, we intend to conduct a comprehensive clinical development strategy for KO-539, both as a monotherapy and in combination, aimed at providing the greatest benefit to patients with acute leukemia, and we are well funded to execute on this strategy."

"A critical component of our KO-539 development plan is the determination of an optimal Phase 2 dose," continued Dr Wilson. "Given the wide therapeutic window of KO-539, we have amended our KOMET-001 trial to include two genetically enriched Phase 1b expansion cohorts. This should enable us to maximize the benefit-risk for KO-539 in our target patient populations and better inform an optimal dose for Phase 2 and beyond. Enrollment in these Phase 1b expansion cohorts is expected to begin mid-year, and we look forward to sharing our progress as we work to bring this important therapeutic option to patients."

Recent Highlights

Enrollment in KOMET-001 Phase 1b expansion cohorts to begin shortly – KO-539 continues to demonstrate a wide therapeutic window and compelling single-agent activity in an all-comer population of patients with relapsed or refractory acute myeloid leukemia (AML), including patients with NPM1 mutations and KMT2A rearrangements. In order to better inform an optimal Phase 2 dose, Kura has amended its KOMET-001 trial of KO-539 to include two Phase 1b expansion cohorts. Both cohorts will be enriched with NPM1-mutant and KMT2A-rearranged relapsed/refractory AML patients. The Company expects to enroll at least 12 patients in each of the Phase 1b expansion cohorts and assess those patients for safety and tolerability, pharmacokinetics/pharmacodynamics and efficacy in order to determine the recommended Phase 2 dose. In addition, the amended Phase 1b protocol gives the Company flexibility to enroll up to 18 additional patients per cohort, as appropriate. Kura believes the patients enrolled in the cohort selected as the recommended Phase 2 dose have the potential to be included in the subsequent, registration-directed portion of the KOMET-001 trial. Patient enrollment in the genetically enriched Phase 1b expansion cohorts is expected to begin at existing and new clinical sites in mid-2021.

Multiple expansion opportunities in acute leukemias – Pending determination of an optimal Phase 2 dose, Kura is preparing to conduct a comprehensive clinical development plan for KO-539, both as a monotherapy and in combination, aimed at broadening the opportunity in acute leukemias. Additional opportunities include front line combination studies, additional genetic subtypes, a pediatric development strategy and other indications, such as acute lymphocytic leukemia and myelodysplastic syndrome.

Publication of tipifarnib Phase 2 data in Journal of Clinical Oncology – Data from Kura’s Phase 2 clinical trial (RUN-HN) of tipifarnib were recently published in the Journal of Clinical Oncology. These data formed the basis of the Breakthrough Therapy Designation granted by the U.S. Food and Drug Administration (FDA) earlier this year for the treatment of patients with recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC). Tipifarnib is currently being evaluated in an ongoing registration-directed clinical trial (AIM-HN) in this indication of high unmet need.

Breakthrough Device Designation for HRAS companion diagnostic – The FDA has granted Breakthrough Device Designation (BDD) to Illumina for a companion diagnostic to detect HRAS mutations in HNSCC in support of Kura’s tipifarnib program, as the device provides for more effective treatment of a life-threatening disease. The next-generation sequencing-based companion diagnostic is being developed in collaboration with Illumina leveraging the content of TruSight Oncology 500. The BDD enables frequent interactions with the FDA and prioritized review on regulatory submissions.
Financial Results

Research and development expenses for the first quarter of 2021 were $20.3 million, compared to $12.6 million for the first quarter of 2020.

General and administrative expenses for the first quarter of 2021 were $10.6 million, compared to $7.6 million for the first quarter of 2020.

Net loss for the first quarter of 2021 was $30.7 million, compared to a net loss of $19.2 million for the first quarter of 2020. This included non-cash share-based compensation expense of $5.1 million, compared to $3.2 million for the same period in 2020.

Cash, cash equivalents and short-term investments totaled $603.9 million as of March 31, 2021, compared with $633.3 million as of December 31, 2020. Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2024.
Upcoming Milestones

Initiation of genetically enriched Phase 1b expansion cohorts in KOMET-001 in mid-2021

Additional Phase 1 data from KOMET-001 in the second half of 2021

Initiation of a Phase 1/2 proof-of-concept study of tipifarnib in combination with a PI3Kα inhibitor in the second half of 2021

Nomination of a next-generation farnesyl transferase inhibitor Development Candidate in mid-2021
Conference Call and Webcast

Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, May 6, 2021, to discuss the financial results for the first quarter 2021 and provide a corporate update. The live call may be accessed by dialing (888) 771-4371 for domestic callers and (847) 585-4405 for international callers and entering the conference code: 50156205. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com.

On May 6, 2021 Dynavax Technologies Corporation (Nasdaq: DVAX), a biopharmaceutical company focused on developing and commercializing novel vaccines, reported financial results for the first quarter of 2021 (Press release, Dynavax Technologies, MAY 6, 2021, View Source [SID1234579341]).

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"The first quarter of 2021 continued to build on our successful execution in 2020. With the combined strength of opportunities from HEPLISAV-B and CpG 1018, we believe 2021 will be a transformational year for Dynavax," commented Ryan Spencer, Chief Executive Officer of Dynavax. "HEPLISAV-B continues to take market share in accounts targeted by our field sales team, reaching a new high this quarter, which reinforces our belief that it will become the standard of care in the U.S. for adult hepatitis B vaccination."

Mr. Spencer continued, "Dynavax is making progress on numerous collaborations for its proven vaccine adjuvant CpG 1018 across multiple indications, including COVID-19, pertussis, and universal flu. Our COVID-19 collaborations have advanced significantly in recent months with multiple partners targeting emergency or conditional authorization in the second half of 2021. Importantly, these collaborations are now generating significant revenue for Dynavax, with first quarter CpG 1018 revenue of $74.6 million. Additionally, last week we expanded our agreement with CEPI whereby they fund manufacturing of CpG 1018 for future sales to CEPI grantees, providing the opportunity for additional revenue in 2021 from COVID-19 collaborations. The emerging portfolio of product opportunities with CpG 1018 has the potential to drive significant revenue growth beyond this year."

HEPLISAV-B [Hepatitis B Vaccine (Recombinant), Adjuvanted]

Net product revenue for HEPLISAV-B during the first quarter 2021 was $8.3 million compared to $10.5 million for the first quarter 2020, driven by increased market share offset by a reduction in vaccine utilization due to the COVID-19 pandemic.
Market share in accounts targeted by the field sales team increased to 27%, up from 21% market share in the first quarter of 2020.
Final immunogenicity and interim safety results of the ongoing clinical trial (HBV-24) evaluating HEPLISAV-B in patients undergoing hemodialysis evaluating a new 4-dose regimen of HEPLISAV-B demonstrated a seroprotection rate of 89.3%. Interim safety data showed HEPLISAV-B is well tolerated and no safety concerns were observed. Full safety data are expected by the end of 2021.
Positive results from the post-marketing observational surveillance study (HBV-25) in over 69,000 patients demonstrated the study met the primary endpoint and showed no evidence of an increased risk of acute myocardial infarction associated with vaccination with HEPLISAV-B compared to Engerix-B.
CpG 1018 (ADVANCED VACCINE ADJUVANT)

Net product revenue for CpG 1018 during the first quarter 2021 was $74.6 million.
In February, Dynavax initiated a Phase 1 clinical trial of Tdap-1018, its tetanus, diphtheria, and acellular pertussis (Tdap) booster vaccine product candidate adjuvanted with CpG 1018.
In March, Clover Biopharmaceuticals dosed the first participant in SPECTRA, a global Phase 2/3 clinical trial for its trimeric SARS-CoV 2 spike (S) protein vaccine adjuvanted with CpG 1018.
In April, Valneva reported positive initial results for Part A of the Phase 1/2 clinical trial of its VLA2001 COVID-19 vaccine candidate adjuvanted with CpG 1018 and subsequently initiated a pivotal Phase 3 clinical trial.
In April, Medigen published positive Phase 1 clinical study data demonstrating neutralizing antibody titers 1.8 to 3.9 times that of human convalescent sera for its COVID-19 vaccine candidate adjuvanted with CpG 1018 and has completed enrollment of over 4,000 participants in its on-going Phase 2 clinical trial.
In April, CEPI expanded its agreement with the Company to provide funding to manufacture CpG 1018 for its COVID-19 vaccine grantees, increasing total funding under the loan agreement from $99 million to $176 million.
2021 MILESTONES

Multiple data readouts from our CpG 1018 COVID-19 collaboration partners throughout the year
Data from the ongoing Phase 1 clinical trial of Tdap-1018 in the fourth quarter
Launch HEPLISAV-B in the EU in the fourth quarter
FINANCIAL RESULTS
Total Revenue. Total revenues for the first quarter of 2021 were $83.3 million, including $82.9 million of net product revenue, an increase from total revenue for the first quarter of 2020 of $10.9 million.

Product Revenue, Net. HEPLISAV-B product revenue, net was $8.3 million in the first quarter of 2021 compared to $10.5 million in the same period in 2020. CpG 1018 product revenue, net was $74.6 million in the first quarter of 2021 compared to $0.0 million in the same period in 2020.

Cost of Sales – Product. Cost of sales – product for the first quarter 2021 increased to $24.6 million, compared to $2.4 million for the first quarter of 2020. The increase was primarily due to manufacturing costs for CpG 1018.

Research and Development Expenses (R&D). R&D expenses for the first quarter of 2021 increased to $7.8 million, compared to $4.7 million for the first quarter of 2020. The increase is primarily due to development activities related to process improvements at our Dusseldorf facility and higher headcount, partially offset by a decrease in business travel due to COVID-19 travel restrictions. In addition, non-cash stock-based compensation in the first quarter of 2020 included reversal of expenses related to cancellation of certain equity grants.

Selling, General and Administrative Expenses (SG&A). SG&A expenses for the first quarter of 2021 increased to $22.4 million, compared to $20.9 million for the first quarter of 2020. Compensation and related personnel costs increased due to higher headcount and an accrual of benefits for a former executive in connection with his retirement, offset by the decrease in business travel due to COVID-19 travel restrictions. Non-cash stock-based compensation increased due to higher headcount.

Income from Operations and Net Income. Income from operations for the first quarter of 2021 was $28.5 million compared to a loss of $19.3 million in the first quarter of 2020. Net income for the first quarter of 2021 was $0.9 million compared to a net loss of $12.6 million for the first quarter of 2020. Basic and diluted net income per share was $0.01 for the first quarter of 2021, compared to a basic net loss of $0.15 per share and diluted net loss per share of $0.25 in the first quarter of 2020.

Cash Position. Cash, cash equivalents and marketable securities totaled $232.7 million at March 31, 2021.

CONFERENCE CALL AND WEBCAST INFORMATION
Dynavax will hold a conference call today at 4:30 p.m. ET/1:30 p.m. PT. The live audio webcast may be accessed through the "Events & Presentations" page on the "Investors" section of the Company’s website at www.dynavax.com. Alternatively, participants may dial (866) 420-4066 or (409) 217-8237 and refer to conference ID 4533398. A replay of the webcast will be available for 30 days following the live event.

Please see Important Safety Information below.

For more information about HEPLISAV-B, visit View Source

About Hepatitis B
Hepatitis B is a viral disease of the liver that can become chronic and lead to cirrhosis, liver cancer and death. The hepatitis B virus is 50 to 100 times more infectious than HIV,I and transmission is on the rise. There is no cure for hepatitis B, but effective vaccination can prevent the disease.

In adults, hepatitis B is spread through contact with infected blood and through unprotected sex with an infected person. The U.S. Centers for Disease Control (CDC) recommends vaccination for those at high risk for infection due to their jobs, lifestyle, living situations and travel to certain areas.II Because people with diabetes are particularly vulnerable to infection, the CDC recommends vaccination for adults age 19 to 59 with diabetes as soon as possible after their diagnosis, and for people age 60 and older with diabetes at their physician’s discretion.III Approximately 20 million U.S. adults have diabetes, and 1.5 million new cases of diabetes are diagnosed each year.IV

About HEPLISAV-B
HEPLISAV-B is an adult hepatitis B vaccine that combines hepatitis B surface antigen with Dynavax’s proprietary Toll-like Receptor (TLR) 9 agonist CpG 1018 to enhance the immune response. Dynavax has worldwide commercial rights to HEPLISAV-B.

Important U.S. Product Information
HEPLISAV-B is indicated for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older.

Safety and effectiveness of HEPLISAV-B have not been established in adults on hemodialysis.

For full U.S. Prescribing Information for HEPLISAV-B, click here.

Important U.S. Safety Information (ISI)
Do not administer HEPLISAV-B to individuals with a history of severe allergic reaction (e.g., anaphylaxis) after a previous dose of any hepatitis B vaccine or to any component of HEPLISAV-B, including yeast. Appropriate medical treatment and supervision must be available to manage possible anaphylactic reactions following administration of HEPLISAV-B. Immunocompromised persons, including individuals receiving immunosuppressant therapy, may have a diminished immune response to HEPLISAV-B. Hepatitis B has a long incubation period. HEPLISAV-B may not prevent hepatitis B infection in individuals who have an unrecognized hepatitis B infection at the time of vaccine administration. The most common patient reported adverse reactions reported within 7 days of vaccination were injection site pain (23% to 39%), fatigue (11% to 17%) and headache (8% to 17%).

Important EU/EEA Product Information
HEPLISAV B is indicated for active immunisation against hepatitis B virus infection (HBV) caused by all known subtypes of hepatitis B virus in adults 18 years of age and older.
The use of HEPLISAV B should be in accordance with official recommendations.
It can be expected that hepatitis D will also be prevented by immunisation with HEPLISAV B as hepatitis D (caused by the delta agent) does not occur in the absence of hepatitis B infection.

For full EU/EEA. Prescribing Information for HEPLISAV-B, click here.

Important EU/EEA Safety information
Do not receive HEPLISAV B if you have had a sudden life-threatening, allergic reaction after receiving HEPLISAV B in the past, or if you are allergic to any of components of this vaccine, including yeast. Signs of an allergic reaction may include itchy skin, rash, shortness of breath and swelling of the face or tongue.
Appropriate medical treatment and supervision should be readily available in case of rare anaphylactic reactions following the administration of the vaccine.
The administration of HEPLISAV B should be postponed in subjects suffering from acute severe febrile illness.
Immunocompromised persons may have a diminished immune response to HEPLISAV B.
Because of the long incubation period of hepatitis B, it is possible for unrecognised HBV infection to be present at the time of immunisation. HEPLISAV B may not prevent HBV infection in such cases.
There are very limited data on the immune response to HEPLISAV B in individuals who did not mount a protective immune response to another hepatitis B vaccine.
As a precautionary measure, it is preferable to avoid the use of HEPLISAV B during pregnancy. Vaccination during pregnancy should only be performed if the risk-benefit ratio at the individual level outweighs possible risks for the fetus.
The most common patient-reported side effects reported within 7 days of vaccination were pain, swelling or redness at the injection site, feeling tired, headache, muscle aches, feeling unwell and fever.

About CpG 1018 Adjuvant
CpG 1018 is the adjuvant used in HEPLISAV-B. Dynavax developed CpG 1018 adjuvant to provide an increased vaccine immune response, which has been demonstrated in HEPLISAV-B. CpG 1018 adjuvant provides a well- developed technology and a significant safety database, potentially accelerating the development and large-scale manufacturing of a COVID-19 vaccine.