On August 5, 2021 bluebird bio, Inc. (NASDAQ: BLUE) reported that the Company will report financial results for the second quarter ended June 30, 2021 on Monday, August 9, 2021 and host a conference call to discuss the quarterly update on Monday, August 9, 2021 at 8 a.m. ET (Press release, bluebird bio, AUG 5, 2021, View Source [SID1234585860]). Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 9698691.

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Additionally, members of the management team will present at the following upcoming investor conferences:

2021 Wedbush PacGrow Healthcare Conference, Wednesday, August 11, at 12:00 p.m. ET as part of the panel titled Miss Con-GENE-iality – Updates in Gene Tx
To access the live webcasts of bluebird bio’s presentations, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source Replays of the webcasts will be available on the bluebird bio website for 90 days following the events.

On August 5, 2021 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the second 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, AUG 5, 2021, View Source [SID1234585859]).

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

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"In the second quarter of 2021, Exelixis delivered a 59 percent year over year increase in CABOMETYX net product revenue growth and record total revenues, based on the broad adoption of the CABOMETYX and OPDIVO combination regimen as a preferred treatment in first-line renal cell carcinoma," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. "Following this strong commercial performance, we are raising our net product and total revenue guidance for 2021. In parallel, we also made significant progress across our development activities, with the filing of a supplemental New Drug Application for cabozantinib in differentiated thyroid cancer based on the COSMIC-311 study, and announcements of top-line results from both the COSMIC-312 trial in first-line advanced hepatocellular carcinoma and the expanded Cohort 6 of the COSMIC-021 trial in patients with advanced prostate cancer. In addition, on the pipeline front, we broadened the development program of XL092 with a new clinical collaboration with Bristol Myers Squibb and initiated the Phase 1 trial for XB002, our first antibody-drug conjugate. I’d like to thank the entire Exelixis team for a very strong first half of 2021 and look forward to providing further updates on our progress in the second half of the year."

Second Quarter 2021 Financial Results

Total revenues for the quarter ended June 30, 2021 were $385.2 million, compared to $259.5 million for the comparable period in 2020.

Total revenues for the quarter ended June 30, 2021 included net product revenues of $284.2 million, compared to $178.7 million for the comparable period in 2020. The increase in net product revenues was primarily related to an increase in sales volume that was 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 2021.

Collaboration revenues, composed of license revenues and collaboration services revenues, were $100.9 million for the quarter ended June 30, 2021, compared to $80.7 million for the comparable period in 2020. The increase in collaboration revenues was primarily related to increases in development cost reimbursements earned, and 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), which was partially offset by a decrease in the recognition of milestone-related revenues.

Research and development expenses for the quarter ended June 30, 2021 were $148.8 million, compared to $114.9 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, personnel expenses and stock-based compensation expense, which was partially offset by decreases in clinical trial costs.

Selling, general and administrative expenses for the quarter ended June 30, 2021 were $98.5 million, compared to $59.8 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.

Provision for income taxes for the quarter ended June 30, 2021 was $28.8 million, compared to $13.9 million for the comparable period in 2020, primarily due to an increase in pre-tax income.

GAAP net income for the quarter ended June 30, 2021 was $96.1 million, or $0.31 per share, basic and $0.30 per share, diluted, compared to GAAP net income of $66.8 million, or $0.22 per share, basic and $0.21 per share, diluted, for the comparable period in 2020.

Non-GAAP net income for the quarter ended June 30, 2021 was $117.9 million, or $0.38 per share, basic and $0.37 per share, diluted, compared to non-GAAP net income of $79.4 million, or $0.26 per share, basic and $0.25 per share, 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 $1.7 billion at June 30, 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.

(1)

Guidance updated on August 5, 2021 from previously provided guidance on May 6, 2021.

(2)

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

(3)

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

(4)

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

(5)

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 $284.2 million during the second quarter of 2021, up 25 percent over the prior quarter, with net product revenues of $275.6 million from CABOMETYX and $8.6 million from COMETRIQ (cabozantinib). Exelixis earned $24.9 million in royalty revenues during the quarter ended June 30, 2021, pursuant to collaboration agreements with our partners, Ipsen and Takeda.

Announcement of Phase 1b Results from Cohort 6 of COSMIC-021 Trial in Patients with Metastatic Castration-Resistant Prostate Cancer (CRPC). In May, Exelixis announced results from the metastatic CRPC cohort 6 of COSMIC-021, the phase 1b trial of cabozantinib in combination with atezolizumab in patients with locally advanced or metastatic solid tumors. The analysis included 132 patients, 101 of whom had high-risk disease, defined as measurable visceral and/or extra-pelvic lymph node metastases. Based on these promising results, Exelixis intends to discuss the data with the FDA to determine next steps toward a potential regulatory submission for the combination regimen for patients with high-risk metastatic CRPC and plans to present detailed results at a medical meeting in the second half of 2021.

Cabozantinib Presentations at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO 2021) Demonstrate Consistent Results in Renal Cell Carcinoma (RCC). In June, cabozantinib was the subject of multiple RCC data presentations at ASCO (Free ASCO Whitepaper) 2021, which included: a post-hoc exploratory analysis demonstrating consistent efficacy benefits across subgroups in the phase 3 CheckMate -9ER pivotal trial with CABOMETYX in combination with OPDIVO compared with sunitinib as a first-line treatment for advanced RCC; results from another post-hoc analysis of the CheckMate -9ER trial demonstrating that CABOMETYX in combination with OPDIVO resulted in a statistically significant and clinically meaningful increase in quality-adjusted survival (Q-TWiST) compared with sunitinib; and positive phase 2 results from an investigator-sponsored trial evaluating cabozantinib in combination with nivolumab in patients with advanced or metastatic non-clear cell RCC.

Detailed Results from Phase 3 COSMIC-311 Pivotal Trial of Cabozantinib in Patients with Previously Treated Radioactive Iodine (RAI)-Refractory Differentiated Thyroid Cancer (DTC) Also Presented at ASCO (Free ASCO Whitepaper) 2021. In June, Exelixis and Ipsen announced detailed results from the phase 3 COSMIC-311 pivotal trial of cabozantinib in patients with previously treated RAI-refractory DTC, presented at ASCO (Free ASCO Whitepaper) 2021. Results from the trial, which met the primary endpoint of demonstrating significant improvement in progression-free survival (PFS) assessed by a blinded independent radiology committee, were recently published in The Lancet Oncology and served as the basis for the supplemental New Drug Application (sNDA) submitted to the FDA in June.

Announcement of Top-line Results of Phase 3 COSMIC-312 Pivotal Trial in Patients with Previously Untreated Advanced Hepatocellular Carcinoma (HCC). In June, Exelixis and Ipsen announced that COSMIC-312, the ongoing phase 3 pivotal trial evaluating cabozantinib in combination with atezolizumab versus sorafenib in patients with previously untreated advanced HCC, met one of the primary endpoints, demonstrating significant improvement in PFS at the planned primary analysis. A prespecified interim analysis for the second primary endpoint of overall survival (OS), conducted at the same time as the primary analysis for PFS, showed a trend favoring the combination of cabozantinib and atezolizumab but did not reach statistical significance. The trial will continue as planned to the final analysis of OS; results are anticipated in early 2022. Exelixis plans to present the trial results at a future medical meeting and intends to discuss the results with the FDA to determine next steps toward a potential regulatory submission for the combination regimen for patients with previously untreated advanced HCC.

FDA Accepts sNDA for CABOMETYX for Patients with Previously Treated RAI-Refractory DTC. In August, Exelixis announced that the FDA accepted the company’s sNDA for CABOMETYX as a treatment for patients 12 years and older with DTC who have progressed following prior therapy and are RAI-refractory (if RAI is appropriate). The FDA granted Priority Review designation and assigned a Prescription Drug User Fee Act goal date, or target action date, of December 4, 2021.

Pipeline Highlights

FDA Accepts Investigational New Drug Application (IND) and Phase 1 Trial Initiated for Tissue Factor-Targeting Antibody-Drug Conjugate (ADC) XB002 in Patients with Advanced Solid Tumors. In April, 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, and in June, a phase 1 trial was initiated. As a next-generation tissue factor-targeting ADC, XB002 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.

Exelixis and Bristol Myers Squibb (BMS) Enter Clinical Trial Collaboration and Supply Agreement to Evaluate XL092 in Combination with Immuno-oncology Therapies in Advanced Solid Tumors. In June, Exelixis announced a clinical trial collaboration and supply agreement with BMS for STELLAR-002, a new phase 1b trial evaluating XL092 in combination with immuno-oncology therapies in advanced solid tumors. The objective of the study is to evaluate the safety, tolerability and efficacy of XL092, Exelixis’ novel next-generation tyrosine kinase inhibitor, in combination with: nivolumab; nivolumab and ipilimumab (YERVOY); and nivolumab and bempegaldesleukin, an investigational CD122-preferential IL-2–pathway agonist developed by Nektar Therapeutics (Nektar). The trial is anticipated to begin enrolling patients in the second half of 2021. Nektar will supply bempegaldesleukin to BMS through their existing global development and commercialization collaboration, which is evaluating nivolumab in combination with bempegaldesleukin.

Corporate Updates

Exelixis Expands its Biotherapeutics Portfolio with Acquisition of Anti-Müllerian Hormone Receptor 2 (AMHR2) Program from GamaMabs Pharma SA (GamaMabs). In May, 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 Files Lawsuit to Enforce Its Intellectual Property Rights for CABOMETYX against Abbreviated New Drug Application (ANDA) Filers. In June, Exelixis filed a patent lawsuit against Teva Pharmaceuticals Development, Inc. and Teva Pharmaceuticals USA, Inc. (individually and collectively referred to as Teva), along with Teva Pharmaceutical Industries Limited, following receipt of two Paragraph IV certification notice letters from Teva informing Exelixis that it had filed an ANDA with the FDA requesting approval to market a generic version of CABOMETYX tablets. Teva’s notice letters 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 is seeking, among other relief, an order that the effective date of any FDA approval of the ANDA would be a date no later than the expiration of all of U.S. Patent Nos. 9,724,342, 10,034,873 and 10,039,757, the latest of which expires on July 9, 2033, and equitable relief enjoining Teva and Teva Pharmaceutical Industries Limited from infringing these patents.

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 July 2, 2021, January 1, 2021 and July 3, 2020 are indicated as being as of and for the periods ended June 30, 2021, December 31, 2020, and June 30, 2020, respectively.

Conference Call and Webcast

Exelixis management will discuss the company’s financial results for the second 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, August 5, 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 7296685 to join by phone.

A telephone replay will be available until 8:00 p.m. EDT on August 7, 2021. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 7296685. A webcast replay will also be archived on www.exelixis.com for one year.

On August 5, 2021 Acorda Therapeutics, Inc. (Nasdaq: ACOR) reported its financial results for the second quarter 2021 (Press release, Acorda Therapeutics, AUG 5, 2021, View Source [SID1234585858]).

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"We were pleased to see a 36% increase in INBRIJA net sales in the second quarter of 2021 over the same period in 2020. We also saw increases in total prescriptions and dispensed cartons. As Inbrija is an on-demand medication and prescription can range from one to five boxes, we believe that dispensed cartons are the best indicator of demand for the product. These are encouraging signs that the impact of the pandemic is moderating, though it is still too early to project how long it will take for prescribing patterns to return to pre-pandemic levels," said Ron Cohen, M.D., Acorda’s President and Chief Executive Officer. "We also were delighted to enter into an agreement with Esteve to commercialize INBRIJA in Spain, providing people with Parkinson’s access to this important medication to address their OFF periods. We also are in active discussions with several parties for commercialization of INBRIJA in other territories in Europe and the rest of the world."

Second Quarter 2021 Financial Results

For the quarter ended June 30, 2021, the Company reported INBRIJA net revenue of $6.4 million, compared to $4.7 million for the same quarter in 2020.

For the quarter ended June 30, 2021, the Company reported AMPYRA net revenue of $21.8 million compared to $26.1 million for the same quarter in 2020. In September 2018, AMPYRA lost its exclusivity and generics entered the market. Consequently, the Company expects AMPYRA revenue to continue to decline.

Research and development (R&D) expenses for the quarter ended June 30, 2021 were $2.4 million, including $0.2 million of share-based compensation compared to $5.3 million, including $0.4 million of share-based compensation for the same quarter in 2020.

Sales, general and administrative (SG&A) expenses for the quarter ended June 30, 2021 were $32.4 million, including $0.7 million of share-based compensation compared to $38.7 million, including $1.5 million of share-based compensation for the same quarter in 2020.

Change in fair value of derivative liability for the quarter ended June 30, 2021 was $(0.8) million compared to $(8.9) million for the same quarter in 2020.

Benefit from income taxes for the quarter ended June 30, 2021 was $0.5 million compared to a provision for income taxes of $0.6 million for the same quarter in 2020.

The Company reported a GAAP net loss of $22.9 million for the quarter ended June 30, 2021, or $2.29 per diluted share. GAAP net loss in the same quarter of 2020 was $17.4 million, or $2.19 per diluted share.

Non-GAAP net loss for the quarter ended June 30, 2021 was $18.7 million, or $1.87 per diluted share. Non-GAAP net loss in the same quarter of 2020 was $16.6 million, or $2.08 per diluted share. This quarterly non-GAAP net loss measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, changes in the fair value of acquired contingent consideration, changes in the fair value of derivative liability related to our 2024 convertible senior secured notes, and expenses that pertain to non-routine corporate restructurings. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At June 30, 2021, the Company had cash, cash equivalents, and restricted cash of $71 million, compared to $103 million at year end 2020. Restricted cash includes $25 million in escrow related to the 6% semi-annual interest portion, payable in cash or stock, of the 2024 convertible senior secured notes. If the Company elects to pay interest due in stock, the restricted cash will be released from escrow.

Financial Guidance

For the full-year 2021, Acorda continues to expect AMPYRA net revenue to be $75 – $85 million, and operating expenses to be $130 – $140 million. The operating expense guidance is a non-GAAP projection that excludes restructuring costs and share-based compensation as more fully described below under "Non-GAAP Financial Measures."
Due to uncertainties caused by past and potential future impacts of the COVID-19 pandemic and other factors, the Company is not providing projected peak U.S. annual net revenue of INBRIJA at this time.
Webcast

The Company will host a webcast in conjunction with its second quarter 2021 update and financial results today at 4:30 p.m. EDT.

To register for the webcast, use the link below:
View Source
Once you have registered, you will receive a confirmation email with webcast details. You will receive an email with the link to join the webcast 2 hours prior to the start time. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 7:30 p.m. EDT on May 6, 2021 until 11:59 p.m. EDT on June 3, 2021. To access the replay, please dial (800) 585-8367 (domestic) or (416) 621-4642 (international); reference code 2996776. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP) and also certain historical and forward-looking non-GAAP financial measures. In particular, Acorda has provided non-GAAP net loss, adjusted to exclude the items below, and has provided 2021 operating expense guidance on a non-GAAP basis. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, the Company believes that the presentation of non-GAAP net loss, when viewed in conjunction with actual GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because this measure excludes (i) non-cash compensation charges and benefits that are substantially dependent on changes in the market price of our common stock, (ii) non-cash interest charges related to the accounting for our convertible debt which are in excess of the actual interest expense owing on such convertible debt, as well as non-cash interest related to the Fampyra royalty monetization and acquired Biotie debt, (iii) changes in the fair value of acquired contingent consideration which do not correlate to our actual cash payment obligations in the relevant periods, (iv) asset impairment charges that are not routine to the operation of the business, (v) expenses that pertain to corporate restructurings which are not routine to the operation of the business, and (vi) changes in the fair value of derivative liability relating to the 2024 convertible senior secured notes, which is a non-cash charge and not related to the operation of the business. The Company believes its non-GAAP net loss measure helps indicate underlying trends in the Company’s business and is important in comparing current results with prior period results and understanding projected operating performance. Also, management uses this non-GAAP financial measure to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

In addition to non-GAAP net loss, we have provided 2021 operating expense guidance on a non-GAAP basis, as the guidance excludes restructuring costs and share-based compensation charges. Due to the forward looking nature of this information, the amount of compensation charges needed to reconcile this measure to the most directly comparable GAAP financial measure is dependent on future changes in the market price of our common stock and is not available at this time. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, the Company believes that the presentation of this non-GAAP financial measure, when viewed in conjunction with actual GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because it excludes (i) expenses that pertain to non-routine corporate restructurings, and (ii) non-cash charges that are substantially dependent on changes in the market price of our common stock. We believe this non-GAAP financial measure helps indicate underlying trends in the Company’s business and is important in comparing current results with prior period results and understanding expected operating performance. Also, management uses this non-GAAP financial measure to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

On August 5, 2021 Galapagos NV (Euronext & NASDAQ: GLPG) reported its progress on earlier-stage programs as well as its commercial launch of filgotinib in Europe. Following recent setbacks, the company is moving forward with its revised strategy and operational restructuring announced in May (Press release, Galapagos, AUG 5, 2021, View Source [SID1234585857]). The unaudited H1 financial and operational results are further detailed in the H1 2021 report available on the website, www.glpg.com.

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"Multiple assets are moving through clinical development, and we recently reported positive topline data on our TYK2 compound GLPG3667. In a Phase 1b trial in psoriasis (Pso), clinical activity was observed at 4 weeks, combined with an encouraging safety and tolerability profile. We currently are running an extended dose escalation study in healthy volunteers, and plan to progress GLPG3667 to a Phase 2b dose finding study in Pso as well as a Phase 2 study in ulcerative colitis (UC) in 2022.

We continue to develop our SIK portfolio of molecules, and recently reported encouraging early data from the first patient studies with SIK2/3 inhibitor GLPG3970. In a Phase 1b trial in Pso (CALOSOMA), clinical activity was observed at 6 weeks, and in a Phase 2a trial in UC (SEA TURTLE), biologically important effects were observed on a number of objective endpoints, both of which point to the potential of SIK inhibition as a novel mode of action in inflammation. No activity was observed in the LADYBUG study in rheumatoid arthritis (RA). GLPG3970 was generally safe and well tolerated. Based on these encouraging data, we work on optimizing the pharmacology of follow-up compounds from our SIK portfolio, and plan to bring an improved SIK2/3 molecule into the clinic in 2022.

On the commercial side, we report rapid progress in establishing our commercial operations for Jyseleca across Europe, with 11 countries launched to date. Reimbursement procedures are on track, and we are on target to achieve our commercial objectives.

We remain excited about the potential of our target discovery platform, our drug development capabilities, and the strength of our teams. We want to thank our shareholders for their continued support and patience as we are working hard to build our pipeline and establish Galapagos as a fully integrated European biopharma," said Onno van de Stolpe, CEO of Galapagos.

Bart Filius, President and COO, added, "Following the strategic exercise announced at Q1, we are focused on advancing our pipeline, implementing our savings program, and diligently evaluating business development opportunities. At the same time, we have been delivering on the launch of Jyseleca, building out our organization in new European markets. In line with our review, we reiterate our 2021 operational cash burni guidance of between €580 million and €620 million. We believe that the decisions and actions taken put us on the strongest footing for the future. We look forward to a busy second half of the year, not the least of which is the expected outcome of the regulatory review in Europe of Jyseleca in UC."

(*) The 2020 comparatives have been restated to consider the impact of classifying the Fidelta business as discontinued operations in 2020.

Details of the financial results
Due to the sale of our fee-for-service business (Fidelta) to Selvita on 4 January 2021 for a total consideration of €37.1 million (including customary adjustments for net cash and working capital), the results of Fidelta are presented as "Net profit from discontinued operations" in our unaudited condensed consolidated income statements for the six months ended 30 June 2021 and 30 June 2020.

Revenues and other income from continuing operations
Our revenues and other income from continuing operations for the first six months of 2021 increased to €277.2 million compared to €217.2 million in the first six months of 2020. Our revenues from the Gilead collaboration in the first six months of 2021 (€253.2 million) related to (i) the exclusive access to our drug discovery platform (€115.7 million), (ii) the filgotinib revenue recognition (€136.1 million) and (iii) royalties (€1.4 million).

Our deferred income balance on 30 June 2021 includes €1.9 billion allocated to our drug discovery platform that is recognized linearly over 10 years, and €0.7 billion allocated for the filgotinib development (including considerations for the previous and the renegotiated collaboration combined) that is recognized over time until the end of the development period.

Results from continuing operations
We realized a net loss from continuing operations of €77.2 million for the first six months of 2021, compared to a net loss of €169.2 million for the first six months of 2020.

We reported an operating loss amounting to €97.6 million for the first six months of 2021, compared to an operating loss of €134.4 million for the same period last year.

Our R&D expenditure in the first six months of 2021 amounted to €268.8 million, compared to €262.9 million for the first six months of 2020. This increase, primarily related to our filgotinib program and our Toledo program, was compensated by a decrease for ziritaxestat, the osteoarthritis (OA) program with GLPG1972, and the program in atopic dermatitis (AtD) with MOR106. Personnel costs increased due to an increase in headcount compared to the same period last year and increased costs of our subscription right plans. This factor, and the increased cost of the commercial launch of filgotinib in Europe, contributed to the increase in our S&M and G&A expenses, which were respectively €29.1 million and €76.9 million in the first six months of 2021, compared to respectively €26.9 million and €61.8 million in the first six months of 2020.

We reported a non-cash fair value gain from the re-measurement of initial warrant B issued to Gilead, amounting to €2.8 million, mainly due to the decreased implied volatility of the Galapagos share price and its evolution between 31 December 2020 and 30 June 2021.

Net other financial income in the first six months of 2021 amounted to €17.1 million, compared to net other financial loss of €13.0 million for the first six months of 2020, which was primarily attributable to €33.4 million of currency exchange gain on our cash and cash equivalents and current financial investments in U.S. dollars, to €8.7 million of negative changes in (fair) value of current financial investments and financial assets and to €4.4 million of interest expenses. The other financial expenses also contained the effect of discounting our long term deferred income of €4.8 million.

Results from discontinued operations
The net profit from discontinued operations for the six months ended 30 June 2021 consisted of the gain on the sale of Fidelta, our fee-for-services business, for €22.2 million.

Group net results
We reported a group net loss for the first six months of 2021 of €55.0 million, compared to a group net loss of €165.6 million for the first six months of 2020.

Cash position
Current financial investments and cash and cash equivalents totaled €5,006.6 million on 30 June 2021, as compared to €5,169.3 million on 31 December 2020.

Total net decrease in cash and cash equivalents and current financial investments amounted to €162.7 million during the first six months of 2021, compared to a net decrease of €214.3 million during the first six months of 2020. This net decrease was composed of (i) €223.2 million of operational cash burn, (ii) offset by €2.6 million of cash proceeds from capital and share premium increase from exercise of subscription rights in the first six months of 2021, (iii) €5.8 million negative changes in (fair) value of current financial investments and €35.0 million of mainly positive exchange rate differences, (iv) €28.7 million cash in from disposal of subsidiaries, net of cash disposed.

Finally, our balance sheet on 30 June 2021 held a receivable from the French government (Crédit d’Impôt Rechercheiv) and a receivable from the Belgian Government for R&D incentives, for a total of both receivables of €142.7 million.

Outlook 2021
In 2021, we expect the European regulatory assessment of filgotinib for the treatment of UC and anticipate both an opinion from the Committee for Medicinal Products for Human Use (CHMP) and a decision from the European Commission later this year. We also expect additional reimbursement decisions for filgotinib in RA across a number of European countries. We are on track to complete the transition from our collaboration partner Gilead to us of the full European commercial operations for filgotinib by year-end, and we anticipate reporting on our own European sales of filgotinib starting in the second half of the year.

Completion of the recruitment in the global DIVERSITY Phase 3 trial with filgotinib in Crohn’s disease by our collaboration partner Gilead is also expected later this year.

With regard to our SIK portfolio, we are advancing our SIK3 inhibitor GLPG4399 in healthy volunteers this year, and we aim to advance a follow-up SIK2/3 preclinical candidate into the clinic in 2022.

Following the positive topline data from our TYK2 inhibitor, GLPG3667, we currently are running an extended dose escalation study in healthy volunteers, and we are preparing for a Phase 2b trial in Pso and a Phase 2 trial in UC next year.

In our other programs, by year-end we intend to finalize recruitment into the GLPG2737 Phase 2a trial in polycystic kidney disease.

Following the previously announced review of our plans for 2021, we reiterate our guidance for full year 2021 operational cash burn of €580 to €620 million.

First half-year report 2021

Galapagos’ financial report for the first half-year ended 30 June 2021, including details of the unaudited consolidated results, is accessible via www.glpg.com/financial-reports.

Conference call and webcast presentation

Galapagos will conduct a conference call open to the public tomorrow, 6 August 2021, at 14:00 CET / 8 AM ET, which will also be webcasted. To participate in the conference call, please call one of the following numbers ten minutes prior to commencement:

A question and answer session will follow the presentation of the results. Go to www.glpg.com to access the live audio webcast. The archived webcast will also be available for replay shortly after the close of the call.

On August 5, 2021 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Arvinas, AUG 5, 2021, View Source [SID1234585856]).

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"Arvinas has made tremendous progress throughout the first half of 2021 and July, highlighted by our global collaboration with Pfizer to co-develop and potentially co-commercialize our estrogen receptor-targeting PROTAC degrader, ARV-471. This collaboration has the potential to be transformational, combining Arvinas’ leadership in targeted protein degradation with Pfizer’s global capabilities to enhance and potentially accelerate the development of ARV-471," said John Houston, Ph.D., President and Chief Executive Officer at Arvinas.

"Through the rest of the year, we look forward to advancing our entire pipeline of PROTAC targeted protein degraders and to several important data milestones, including interim data from the ARDENT Phase 2 trial of our androgen receptor-degrading PROTAC degrader, ARV-110, and Phase 1 dose escalation data from ARV-471 and ARV-110," continued Dr. Houston.

Business Highlights and Recent Developments

Announced a global collaboration with Pfizer to co-develop and co-commercialize ARV-471 for the treatment of patients with ER+ breast cancer. The deal includes an upfront payment to Arvinas of $650 million, a $350 million equity investment from Pfizer, and $1.4 billion in potential milestone payments. Arvinas and Pfizer will equally share worldwide development costs, commercialization expenses, and profits.
Initiated the first-in-human study of ARV-766, an androgen receptor (AR) degrader with a differentiated profile from ARV-110, in patients with metastatic castration-resistant prostate cancer.
Completed dose escalation in the Phase 1 study of ARV-471 for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer.
Completed dose escalation in the Phase 1 study of ARV-110 for the treatment of metastatic castrate-resistant prostate cancer.
Anticipated Milestones and Expectations

ARV-471

Presentation of Phase 1 dose escalation trial data (at the San Antonio Breast Cancer Symposium, December 2021)
Initiation of a neoadjuvant study in early breast cancer (2H21)
Initiation of a combination trial of ARV-471 and everolimus in 2L/3L metastatic breast cancer (2H21)
ARV-110

Presentation of completed Phase 1 dose escalation data (2H21)
Presentation of interim data from the ARDENT Phase 2 dose expansion at 420 mg (2H21)
Initiation of combination trial with abiraterone (2H21)
Second Quarter Financial Results

Cash, Cash Equivalents, and Marketable Securities Position: As of June 30, 2021, cash, cash equivalents, and marketable securities were $605.1 million as compared with $688.5 million as of December 31, 2020. The cash, cash equivalents and marketable securities amounts do not reflect the global Pfizer collaboration agreement upfront payment of $650 million that was received in July 2021, or the equity investment by Pfizer of $350 million (which is subject to certain conditions including clearance under the Hart-Scott Rodino Antitrust Improvements Act of 1976, as amended). The decrease in cash, cash equivalents and marketable securities of $83.4 million for the first six months of 2021 was primarily related to net cash used in operating activities of $86.4 million (net of $4.0 million received from two collaborators), the purchase of lab equipment and lease-hold improvements of $1.5 million and net accretion of discounts/premiums and unrealized loss on marketable securities of $3.6 million, partially offset by cash provided from the exercise of stock options of $8.1 million.

Research and Development Expenses: Research and development expenses were $43.0 million for the quarter ended June 30, 2021, as compared with $23.4 million for the quarter ended June 30, 2020. The increase in research and development expenses of $19.6 million for the quarter was primarily related to increases in clinical trial and CMC expenses associated with our AR program of $7.3 million and our ER program of $3.4 million, in addition to increases in preclinical expenses of $8.9 million associated with exploratory programs and investments in platform research.

General and Administrative Expenses: General and administrative expenses were $14.4 million for the quarter ended June 30, 2021, as compared to $8.8 million for the quarter ended June 30, 2020. The increase of $5.6 million was primarily related to an increase in personnel costs, facility related costs and professional fees.

Revenue: Revenue was $5.5 million for the quarter ended June 30, 2021, as compared with $5.7 million for the quarter ended June 30, 2020. Revenue is generated from the license and rights to technology fees and research and development activities related to the collaboration and license agreement with Bayer that was initiated in July 2019, the collaboration and license agreement with Pfizer that was initiated in January 2018, and the amended and restated option, license and collaboration agreement with Genentech that was initiated in November 2017. The decrease of $0.2 million for the quarter was primarily related to a collaborator adding new targets in previous periods that extended the period of revenue recognition for that collaboration agreement.

Interest and Other Income: Interest and other Income was $1.6 million for the quarter ended June 30, 2021 as compared with $1.3 million for the quarter ended June 30, 2020. The increase of $0.3 million was primarily due to forgiveness of debt of $1.0 million, related to a State of Connecticut loan which was partially forgiven upon our satisfaction of certain jobs criteria, partially offset by lower interest income and lower refundable research and development credits from the State of Connecticut.

Net Loss: Net loss was $50.3 million for the quarter ended June 30, 2021, as compared with $25.2 million for the quarter ended June 30, 2020. The increase in net loss for the quarter was primarily due to increased research and development expenses and increased general and administrative expenses.

About ARV-110
ARV-110 is an investigational orally bioavailable PROTAC protein degrader designed to selectively target and degrade the androgen receptor (AR). ARV-110 is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer.

ARV-110 has demonstrated activity in preclinical models of AR mutation or overexpression, both common mechanisms of resistance to currently available AR-targeted therapies.

About ARV-471
ARV-471 is an investigational orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer.

In preclinical studies, ARV-471 demonstrated near-complete ER degradation in tumor cells, induced robust tumor shrinkage when dosed as a single agent in multiple ER-driven xenograft models, and showed superior anti-tumor activity when compared to a standard of care agent, fulvestrant, both as a single agent and in combination with a CDK4/6 inhibitor. In July 2021, Arvinas announced a global collaboration with Pfizer for the co-development and co-commercialization of ARV-471; Arvinas and Pfizer will equally share worldwide development costs, commercialization expenses, and profits.

About ARV-766
ARV-766 is an investigational orally bioavailable PROTAC protein degrader designed to selectively target and degrade androgen receptor (AR). In preclinical studies, ARV-766 degraded all resistance-driving point mutations of AR tested, including L702H, a mutation associated with treatment with abiraterone and other AR-pathway therapies.

ARV-766 is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer. ARV-766 has demonstrated activity in preclinical models of resistance to currently available AR-targeted therapies.