On February 25, 2021 MannKind Corporation (NASDAQ:MNKD) reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Mannkind, FEB 25, 2021, View Source [SID1234575627]).

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"Our fourth quarter produced exceptional results, with $10.1 million in Afrezza net revenue and positive clinical data for Tyvaso DPI from the BREEZE study conducted by United Therapeutics," said Michael Castagna, Chief Executive Officer of MannKind Corporation. "UT also announced their plan to submit a new drug application for Tyvaso DPI to the FDA in April 2021. During the fourth quarter, we solidified our new direction with the acquisition of QrumPharma, which added a nebulized version of clofazimine to our pipeline of therapies for orphan lung diseases, and we entered into a collaboration agreement with Vertice for the co-promotion of Thyquidity, which is indicated for hyperthyroidism and is expected to expand our reach into endocrine diseases."

Fourth Quarter 2020 Results

Total revenues were $18.4 million for the fourth quarter of 2020, reflecting Afrezza net revenue of $10.1 million and collaborations and services revenue of $8.4 million. Afrezza net revenue increased 30% compared to $7.8 million in the fourth quarter of 2019, primarily driven by higher product demand with a more favorable mix of Afrezza cartridges and more favorable gross-to-net deductions. Collaborations and services revenue increased $0.2 million compared to the fourth quarter of 2019.

Afrezza gross profit for the fourth quarter of 2020 was $6.4 million compared to $3.1 million in the same period of 2019, an increase of $3.3 million, or 105%, that was driven by a combination of increased Afrezza revenue and a reduction in cost of goods sold.

In-process research and development expense for the fourth quarter of 2020 was $13.2 million, reflecting the acquisition of QrumPharma for approximately $12.8 million in total consideration and approximately $0.4 million in transaction costs. The acquisition of QrumPharma was accounted for as an asset acquisition and expensed on the date of acquisition as substantially all of the fair value of the assets acquired was concentrated in a single asset that consisted of in-process research and development in a pre-clinical development state.

Research and development expenses for the fourth quarter of 2020 were $1.5 million compared to $2.0 million for the fourth quarter of 2019. This decrease was mainly related to lower clinical trial spending.

Selling, general and administrative expenses for the fourth quarter of 2020 were $17.1 million compared to $15.7 million for the fourth quarter of 2019. This increase of $1.4 million, or 9%, was primarily attributable to a $1.2 million increase in personnel costs related to the expansion of our sales and medical field force.

During the fourth quarter of 2020, loss on foreign currency translation for insulin purchase commitments, which are denominated in Euros, was $4.0 million compared to $2.6 million for the fourth quarter of 2019. The fluctuation was due to a change in the U.S. dollar to Euro foreign exchange rate.

Interest expense on debt for the fourth quarter of 2020 was $2.4 million compared to $2.3 million for the fourth quarter of 2019.

The net loss for the fourth quarter of 2020 was $26.4 million, or $0.11 per share, compared to $14.3 million in the fourth quarter of 2019, or $0.07 per share. The increase in the net loss of $12.1 million was primarily due to the write-off of in-process research and development related to the acquisition of QrumPharma. On a non-GAAP basis, excluding the expense incurred for the acquisition of QrumPharma, the net loss for the fourth quarter of 2020 was $13.2 million, or $0.06 per share.

Twelve Months Ended December 31, 2020

Total revenues were $65.1 million for the year ended December 31, 2020, reflecting Afrezza net revenue of $32.3 million and collaborations and services revenue of $32.8 million. Afrezza net revenue increased 28% compared to $25.3 million for the year ended December 31, 2019, primarily driven by higher product demand with a more favorable mix of Afrezza cartridges, a price increase and more favorable gross-to-net deductions, all of which was partially offset by a reduction in sales to Biomm (Brazil). Collaborations and services revenue decreased $4.9 million compared to the full year ended December 31, 2019, primarily driven by a $5.8 million decrease in revenue recognized from the UT Research Agreement, which was substantially completed in the second quarter of 2019.

Afrezza gross profit was $17.2 million for the year ended December 31, 2020, an increase of $12.0 million, or 230%, compared to a gross profit of $5.2 million in the same period in 2019, primarily due higher commercial product sales combined with a reduction in cost of goods sold.

In-process research and development expense for the year ended December 31, 2020 was $13.2 million, reflecting the research and development acquired and expensed from the acquisition of QrumPharma for approximately $12.8 million in total consideration and approximately $0.4 million in transaction costs.

Research and development expenses for the year ended December 31, 2020 were $6.2 million compared to $6.9 million for the year ended December 31, 2019. This decrease of $0.7 million, or 9%, was primarily attributable to lower clinical trial spending.

Selling, general and administrative expenses for the year ended December 31, 2020 were $59.0 million compared to $74.7 million for the year ended December 31, 2019. This decrease of $15.6 million, or 21%, was primarily attributable a $9.3 million decrease in costs for television advertising for Afrezza, a $4.1 million decrease in promotional and marketing activities in response to the COVID-19 pandemic and a $2.5 million decrease in professional fees.

An impairment of $1.9 million was recognized for the year ended December 31, 2020 on a commitment asset and debt issuance costs related to future funding commitments of the MidCap Credit Facility. There were no asset impairments for the year ended December 31, 2019.

For the year ended December 31, 2020, foreign currency translation for insulin purchase commitments, which are denominated in Euros, resulted in a loss of $8.0 million compared to a gain of $1.9 million for the year ended December 31, 2019. The fluctuation was due to a change in the U.S. dollar to Euro foreign exchange rate.

Interest expense on debt for the year ended December 31, 2020 was $9.5 million compared to $10.9 million for the year ended December 31, 2019. This $1.4 million decrease was primarily attributable to a $3.4 million milestone obligation to Deerfield that was achieved in the third quarter of 2019 and a decrease of $0.8 million of interest expense related to the Deerfield Credit Facility, which was extinguished in the third quarter of 2019. This decrease was partially offset by an increase in interest expense from the MidCap Credit Facility of $2.3 million and an increase in interest expense from our Mann Group promissory notes of $0.6 million in 2020.

The net loss for the year ended December 31, 2020 was $57.2 million, or $0.26 per share, compared to $51.9 million net loss for the year ended December 31, 2019, or $0.27 per share. The higher net loss was mainly attributable to the write-off of in-process research and development related to the acquisition of QrumPharma and a loss on foreign currency translation related to insulin purchase commitments denominated in Euros, offset by a decrease in selling, general and administrative expenses. On a non-GAAP basis, excluding the expense incurred for the acquisition of QrumPharma, the net loss for the year ended December 31, 2020 was $44.0 million, or $0.20 per share.

Cash, cash equivalents, restricted cash, and short-term investments at December 31, 2020 was $67.2 million compared to $50.2 million at December 31, 2019.

Debt Reductions Subsequent to December 31, 2020

Pursuant to the terms of the senior convertible notes, the Company forced the conversion of all $5.0 million in principal of such notes into 1,666,667 shares of the Company’s common stock.

In addition, the Mann Group converted $9.6 million of principal and $0.4 million of accrued interest on its convertible promissory note into 4.0 million shares of the Company’s common stock. As of the date hereof, $53.4 million in principal remains outstanding under the promissory notes held by the Mann Group ($18.4 of which is convertible).

Sale-Leaseback of the Danbury Manufacturing Facility

Subsequent to December 31, 2020, the Company entered into a non-binding letter of intent ("LOI") with a third party to sell and lease back a portion of the Company’s Danbury manufacturing facility and administrative offices. The terms of the LOI include a sales price of approximately $95 million – $105 million, a lease term of 20 years with four 5-year renewal options, and annual rent of approximately $10 million – $11 million at the beginning of the lease. If the transaction is completed, the Company intends to use the proceeds for general corporate purposes, and may also pay down a portion of its senior secured debt. The completion of the transactions contemplated by the LOI is subject to certain conditions, including the negotiation of satisfactory definitive agreements and satisfactory results of the buyer’s inspections and other investigations, all of which are anticipated to be completed during the first quarter of 2021. However, there can be no assurances that this proposed transaction will be completed in the timeframe or on the principal terms set forth above, or at all.

Non-GAAP Measures

Certain financial information contained in this press release is presented on both a reported basis (GAAP) and a non-GAAP basis. Reported results were prepared in accordance with GAAP whereas non-GAAP measures exclude items described in the reconciliation tables below. Non-GAAP financial information is intended to portray the results of our baseline performance, supplement or enhance management, analysts and investors overall understanding of our underlying financial performance and facilitate comparisons among current and past periods. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP.

Conference Call

MannKind will host a conference call and presentation webcast to discuss these results today at 9:00 a.m. Eastern Time. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at View Source under News & Events. A replay will be available on MannKind’s website for 14 days.

On February 25, 2021 Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, reported financial results for the fourth quarter and full-year of 2020 (Press release, Pacira Pharmaceuticals, FEB 25, 2021, View Source;991.htm [SID1234575626]).

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"Our nation’s opioid crisis has escalated under the shadow of the COVID-19 pandemic, as isolation and lack of access to healthcare has exacerbated mental health challenges, particularly addiction. I am delighted to report that EXPAREL-based protocols are expanding opioid-sparing pain management for a variety of procedures where historically poor postsurgical pain management fueled such addictions. Despite these challenging times, we have quickly adapted to a virtual world and delivered record revenues for 2020," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "Looking ahead to the balance of the year, we remain steadfast in our commitment to providing an opioid alternative to as many patients as possible and redefining the role of opioids as a rescue medication while enabling the migration to hospital outpatient and ambulatory surgery centers for elective surgery."

2020 Full-Year and Fourth Quarter Financial Highlights

Full-year revenues of $429.6 million and fourth quarter revenues of $131.0 million.
Full-year GAAP net income of $145.5 million or $3.41 per share (basic) and $3.33 (diluted).
Fourth quarter GAAP net income of $14.5 million or $0.33 per share (basic) and $0.32 (diluted).
Full-year non-GAAP Adjusted EBITDA of $112.6 million and fourth quarter non-GAAP Adjusted EBITDA of $42.9 million.
Recent Business Highlights

Equity investment in GeneQuine Biotherapeutics.
In January 2021, Pacira announced an equity investment in GeneQuine Biotherapeutics GmbH. Under the terms of the agreement, Pacira made an initial investment of €2.0 million with an additional €4.0 million investment predicated upon GeneQuine achieving certain prespecified near-term milestones related to its lead gene therapy product candidate, GQ-303. Up to €2.5 million of the total Pacira investment will be in the form of a convertible note. In addition, Pacira is entitled to appoint one member to GeneQuine’s board of directors. GeneQuine Biotherapeutics is a privately held biopharmaceutical company advancing a gene therapy platform for the treatment of osteoarthritis (OA) and other musculoskeletal disorders. GeneQuine’s product candidates are next-generation gene transfer vehicles that are highly efficient in entering joint cells to confer multi-year gene expression.

European Commission approves EXPAREL for the treatment of postsurgical pain. In November 2020, the European Commission granted marketing authorization for EXPAREL as a brachial plexus block or femoral nerve block for treatment of post-operative pain in adults, and as a field block for treatment of somatic post-operative pain from small- to medium-sized surgical wounds in adults. The European Commission approval was based on the results of four pivotal Phase 3 studies that demonstrated improvements in pain reduction and opioid use. These studies include: lower extremity nerve block, upper extremity nerve block, and infiltration studies in hard and soft tissue surgeries. The European Commission decision is applicable to all 27 European Union member states plus the United Kingdom, Iceland, Norway and Liechtenstein. Commercial planning is underway, with an anticipated launch in the second half of 2021.
Fourth Quarter 2020 Financial Results

Total revenues were $131.0 million in the fourth quarter of 2020, a 7% increase over the $122.4 million reported for the fourth quarter of 2019.
EXPAREL net product sales were $125.3 million in the fourth quarter of 2020, a 7% increase over the $116.9 million reported for the fourth quarter of 2019.
Fourth quarter 2020 iovera° net product sales were $2.4 million, a 25% decrease versus the $3.2 million reported in the fourth quarter of 2019.
Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $2.0 million in the fourth quarter of 2020, compared to $1.7 million in 2019.
Fourth quarter 2020 royalty revenue was $1.2 million compared to $0.6 million in 2019.
Total operating expenses were $112.2 million in the fourth quarter of 2020, compared to $120.7 million in the fourth quarter of 2019.
Research and development (R&D) expenses were $15.3 million in the fourth quarter of 2020, compared to $19.7 million in the fourth quarter of 2019. The company’s R&D expenses include $5.2 million and $8.7 million of product development and manufacturing capacity expansion costs in the fourth quarters of 2020 and 2019, respectively.
Selling, general and administrative (SG&A) expenses were $52.8 million in the fourth quarter of 2020, compared to $54.2 million in the fourth quarter of 2019.
GAAP net income was $14.5 million, or $0.33 per share (basic) and $0.32 (diluted), in the fourth quarter of 2020, compared to a GAAP net loss of $4.9 million, or $0.12 per share (basic and diluted), in the fourth quarter of 2019.
Non-GAAP net income was $38.8 million, or $0.89 per share (basic) and $0.87 per share (diluted), in the fourth quarter of 2020, compared to non-GAAP net income of $23.8 million, or $0.57 per share (basic) and $0.56 per share (diluted), in the fourth quarter of 2019.
Adjusted EBITDA was $42.9 million in the fourth quarter of 2020, a 48% increase over $29.1 million in the fourth quarter of 2019.
Pacira had 43.5 million basic and 44.7 million diluted weighted average shares of common stock outstanding in the fourth quarter of 2020.
Full-Year 2020 Financial Results

Total revenues were $429.6 million in 2020, a 2% increase over the $421.0 million reported in 2019.
EXPAREL net product sales were $413.3 million in 2020, a 1% increase over the $407.9 million reported in 2019.
Full-year iovera° net product sales were $8.8 million, a 12% increase over the $7.9 million reported in 2019. Pacira began recognizing sales of iovera° in April 2019 after completing its acquisition of MyoScience, Inc., a privately held medical technology company.
Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $4.5 million in 2020, compared to $3.2 million in 2019.
Full-year royalty revenue was $3.0 million compared to $2.1 million in 2019.
Total operating expenses were $383.3 million in 2020, compared to $410.5 million in 2019.
Research and development (R&D) expenses were $59.4 million in 2020, compared to $72.1 million in 2019. The company’s R&D expenses include $23.5 million and $29.7 million of product development and manufacturing capacity expansion costs in 2020 and 2019, respectively.
Selling, general and administrative (SG&A) expenses were $193.5 million in 2020, compared to $200.8 million in 2019.
GAAP net income was $145.5 million, or $3.41 per share (basic) and $3.33 per share (diluted) in 2020, compared to a GAAP net loss of $11.0 million, or $0.27 per share (basic and diluted) in 2019.
Non-GAAP net income was $96.6 million, or $2.26 per share (basic) and $2.21 per share (diluted), in 2020, compared to non-GAAP net income of $70.7 million, or $1.70 per share (basic) and $1.67 per share (diluted), in 2019.
Adjusted EBITDA was $112.6 million in 2020, a 26% increase over $89.2 million in 2019.
Pacira had 42.7 million basic and 43.7 million diluted weighted average shares of common stock outstanding in 2020.
See "Non-GAAP Financial Information" below.

Financial Guidance

The company’s 2021 product sales continue to be negatively impacted by the COVID-19 pandemic, which mandated significant postponement or suspension in the scheduling of elective surgical procedures resulting from public health guidance and government directives. Elective surgery restrictions began to lift on a state-by-state basis in April 2020. In order to provide greater transparency, the company will continue to report monthly intra-quarter unaudited net product sales until it has gained enough visibility around the impacts of COVID-19 to reinstate financial guidance.

Today’s Conference Call and Webcast Reminder

The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Thursday, February 25, 2021, at 8:30 a.m. ET. To participate in the conference call, dial 1-877-845-0779 and provide the passcode 4864607. International callers may dial 1-720-545-0035 and use the same passcode. In addition, a live audio of the conference call will be available as a webcast. Interested parties can access the event through the "Events" page on the Pacira website at investor.pacira.com.

For those unable to participate in the live call, a replay will be available at 1-855-859-2056 (domestic) or 1-404-537-3406 (international) using the passcode 4864607. The replay of the call will be available for one week from the date of the live call. The webcast will be available on the Pacira website for approximately two weeks following the call.

Non-GAAP Financial Information

This press release contains financial measures that do not comply with U.S. generally accepted accounting principles (GAAP), such as non-GAAP net income, non-GAAP net income per share, non-GAAP cost of goods sold, non-GAAP gross margins, non-GAAP research and development (R&D) expense and non-GAAP selling, general and administrative (SG&A) expense and adjusted EBITDA, because such measures exclude acquisition-related charges, product discontinuation costs and other expense; stock-based compensation; amortization of debt discount; loss on early extinguishment of debt, amortization of acquired intangible assets; an income tax benefit and a step-up in basis of inventory in connection with the acquisition of MyoScience, Inc., (gain) loss on investment and other non-operating income and the reversal of a deferred tax valuation allowance.

These measures supplement Pacira’s financial results prepared in accordance with GAAP. Pacira management uses these measures to better analyze its financial results, estimate its future cost of goods sold, gross margins, R&D expense and SG&A expense outlook for 2021 and to help make managerial decisions. In management’s opinion, these non-GAAP measures are useful to investors and other users of our financial statements by providing greater transparency into the operating performance at Pacira and its future outlook. Such measures should not be deemed to be an alternative to GAAP requirements or a measure of liquidity for Pacira. Non-GAAP measures are also unlikely to be comparable with non-GAAP disclosures released by other companies. See the tables below for a reconciliation of GAAP to non-GAAP measures, including adjusted EBITDA.

On February 25, 2021 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it will host a live conference call and webcast at 8:30 a.m. ET on Thursday, March 4, 2021 to report its fourth quarter and full year 2020 financial results and provide a corporate update (Press release, Syros Pharmaceuticals, FEB 25, 2021, View Source [SID1234575625]).

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To access the live conference call, please dial 866-595-4538 (domestic) or 636-812-6496 (international), and refer to conference ID 4472467. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

On February 25, 2021 Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, reported its financial results for the fourth quarter and year ended December 31, 2020 and provided a business update (Press release, Gossamer Bio, FEB 25, 2021, View Source [SID1234575624]).

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"The Gossamer team enters 2021 excited and focused on clinical trial execution," said Faheem Hasnain, Co-Founder, Chairman and Chief Executive Officer of Gossamer. "Both seralutinib and GB004 are potentially paradigm shifting product candidates in indications of significant unmet patient need, and I am very proud of our team’s ongoing efforts to conduct our Phase 2 proof-of-concept studies for these programs despite the challenges of the pandemic."

Clinical-Stage Product Candidate Updates

Seralutinib (GB002): Inhaled PDGFR, CSF1R and c-KIT Inhibitor for PAH

Enrollment ongoing in the TORREY Study, a Phase 2 clinical trial in patients with PAH whose disease has progressed despite standard-of-care therapy. The primary endpoint is change in pulmonary vascular resistance (PVR) from baseline at week 24. Topline data from the TORREY study are expected in the first half of 2022, subject to developments in the ongoing COVID-19 pandemic.
Key opinion leader-led webcast and presentation regarding PAH and seralutinib (GB002) held on December 15, 2020 available through the "Events / Presentations" page in the "Investors" section of the Company’s website at www.gossamerbio.com.
GB004: Oral HIF-1α Stabilizer for Inflammatory Bowel Disease (IBD)

Enrollment ongoing in the SHIFT-UC Study, a Phase 2 clinical trial in patients with active UC despite treatment with 5-ASAs. The primary endpoint is proportion of patients with clinical remission at week 12. Topline data from the SHIFT-UC study are expected in the first half of 2022, subject to developments in the ongoing COVID-19 pandemic.
Key opinion leader-led webcast and presentation regarding IBD and GB004 held on February 18, 2021 available through the "Events / Presentations" page in the "Investors" section of the Company’s website at www.gossamerbio.com.
GB1275: Oral CD11b Modulator for Oncology Indications

Enrollment ongoing in a Phase 1 expansion cohort studying the recommended Phase 2 dose in KEYNOTE-A36, a Phase 1/2 clinical trial, including patients with gastric or esophageal cancer who have progressed after initial response to anti-PD-1 therapy and patients with advanced MSS colorectal cancer. Further Phase 1 data expected from this study in 2021.
GB001: Oral DP2 Antagonist for Eosinophilic Asthma

Gossamer engaged with the FDA and the EMA about the clinical development path in asthma, and based off those interactions, Gossamer believes that there is a viable clinical development path for GB001, or its backup molecule, in asthma. Gossamer does not currently plan to move forward with GB001, or its backup molecule, in further clinical trials without a partner.
Financial Results for Quarter and Full Year Ended December 31, 2020

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of December 31, 2020, were $512.6 million. As a result, we currently expect cash, cash equivalents and marketable securities, and access to our debt facility will be sufficient to fund operating and capital expenditures into the second half of 2023.
Research and Development (R&D) Expenses: For the quarter ended December 31, 2020, R&D expenses were $38.9 million compared to R&D expenses of $42.6 million for the same period in 2019. R&D expenses for the full year ended December 31, 2020, were $160.9 million compared to $143.4 million for the full year ended December 31, 2019. The increases were primarily due to an increase in expenses for GB004, seralutinib, GB1275 and preclinical programs.
In-Process Research and Development (IPR&D) Expenses: For the quarter ended December 31, 2020, IPR&D expenses were $5.3 million compared to $1.6 million for the same period in 2019. IPR&D expenses for the full year ended December 31, 2020, were $23.4 million compared to $3.6 million for the full year ended December 31, 2019. The increases were primarily attributable to a $15.0 million payment to Aerpio in connection with the amendment to the in-license agreement of GB004 in 2020 and a milestone payment of $5.0 million in connection with the initiation of the first Phase 2 clinical trial of seralutinib in 2020.
General and Administrative (G&A) Expenses: For the quarter ended December 31, 2020, G&A expenses were $15.9 million compared to $11.6 million for the same period in 2019. G&A expenses for the full year ended December 31, 2020, were $49.7 million compared to $39.1 million for the full year ended December 31, 2019. The increases were primarily attributable to an increase in stock-based compensation costs and an increase in personnel-related costs.
Net Loss: Net loss for the three months ended December 31, 2020, was $64.6 million, or $0.88 per share, compared to a net loss of $54.7 million, or $0.89 per share, for the same period in 2019. Net loss for the full year ended December 31, 2020, was $243.4 million, or $3.55 per share compared to a net loss of $180.3 million, or $3.29 per share, for the full year ended December 31, 2019.
Conference Call and Webcast

Gossamer’s management team will host a conference call and live audio webcast at 8:30 a.m. ET today, Thursday, February 25, to discuss its fourth quarter and full year 2020 financial results and provide a corporate update.

The live audio webcast may be accessed through the "Events / Presentations" page in the "Investors" section of the Company’s website at www.gossamerbio.com. Alternatively, the conference call may be accessed through the following:

On February 25, 2021 Exelixis, Inc. (NASDAQ: EXEL) reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to cabozantinib (CABOMETYX) as a potential treatment for patients with differentiated thyroid cancer (DTC) that has progressed following prior therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) (Press release, Exelixis, FEB 25, 2021, View Source [SID1234575623]). 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. To qualify for this designation, preliminary clinical evidence must indicate that the drug may demonstrate substantial improvement on at least one clinically significant endpoint over existing therapies.

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"Receiving Breakthrough Therapy Designation is a testament to both the urgent need for effective treatments for patients with differentiated thyroid cancer who progressed after prior therapy and the promising data demonstrating cabozantinib significantly improved progression-free survival for these patients," said Gisela Schwab, M.D., President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis. "We look forward to submitting our regulatory application in 2021 and to working closely with the FDA during the review process, with the goal of bringing cabozantinib to this patient population with a high unmet medical need for whom there is currently no available standard of care."

In December 2020, Exelixis announced that at a planned interim analysis, the phase 3 COSMIC-311 pivotal trial met the co-primary endpoint, demonstrating a significant reduction in the risk of disease progression or death of 78% with cabozantinib versus placebo (HR 0.22, 96% CI 0.13 – 0.36; p<0.0001) in patients with radioactive iodine-refractory differentiated thyroid cancer who have progressed after up to two prior VEGFR-targeted therapies. The safety profile was consistent with that previously observed for cabozantinib.

About COSMIC-311

COSMIC-311 is a multicenter, randomized, double-blind, placebo-controlled phase 3 pivotal trial that aimed to enroll approximately 300 patients at 150 sites globally. Patients were randomized in a 2:1 ratio to receive either cabozantinib 60 mg or placebo once daily. The co-primary endpoints are progression-free survival and objective response rate. More information about this trial is available at ClinicalTrials.gov.

About Differentiated Thyroid Cancer

Approximately 44,000 new cases of thyroid cancer will be diagnosed in the U.S. in 2021.1 Nearly three out of four of these cases will be in women, and the disease is more commonly diagnosed at a younger age compared to most other adult cancers.1 While cancerous thyroid tumors include differentiated, medullary and anaplastic forms, differentiated thyroid tumors make up about 90 percent of cases.1 These include papillary, follicular and Hürthle cell cancer.1 Differentiated thyroid cancer is typically treated with surgery followed by ablation of the remaining thyroid tissue with radioiodine, but approximately 5% to 15% of cases are resistant to radioiodine treatment. 2,3 For these patients, life expectancy is only three to six years from the time metastatic lesions are detected.4,5,6

About CABOMETYX (cabozantinib)

In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC; for the treatment of patients with HCC who have been previously treated with sorafenib; and for patients with advanced RCC as a first-line treatment in combination with nivolumab. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the United States.

CABOMETYX is not indicated for radioiodine-refractory differentiated thyroid cancer.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hemorrhage: Severe and fatal hemorrhages occurred with CABOMETYX. The incidence of Grade 3 to 5 hemorrhagic events was 5% in CABOMETYX patients in RCC and HCC studies. Discontinue CABOMETYX for Grade 3 or 4 hemorrhage. Do not administer CABOMETYX to patients who have a recent history of hemorrhage, including hemoptysis, hematemesis, or melena.

Perforations and Fistulas: Fistulas, including fatal cases, occurred in 1% of CABOMETYX patients. Gastrointestinal (GI) perforations, including fatal cases, occurred in 1% of CABOMETYX patients. Monitor patients for signs and symptoms of fistulas and perforations, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a Grade 4 fistula or a GI perforation.

Thrombotic Events: CABOMETYX increased the risk of thrombotic events. Venous thromboembolism occurred in 7% (including 4% pulmonary embolism) and arterial thromboembolism in 2% of CABOMETYX patients. Fatal thrombotic events occurred in CABOMETYX patients. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or serious arterial or venous thromboembolic events that require medical intervention.

Hypertension and Hypertensive Crisis: CABOMETYX can cause hypertension, including hypertensive crisis. Hypertension was reported in 36% (17% Grade 3 and <1% Grade 4) of CABOMETYX patients. Do not initiate CABOMETYX in patients with uncontrolled hypertension. Monitor blood pressure regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume at a reduced dose. Discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy or for hypertensive crisis.

Diarrhea: Diarrhea occurred in 63% of CABOMETYX patients. Grade 3 diarrhea occurred in 11% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 diarrhea, Grade 3 diarrhea that cannot be managed with standard antidiarrheal treatments, or Grade 4 diarrhea.

Palmar-Plantar Erythrodysesthesia (PPE): PPE occurred in 44% of CABOMETYX patients. Grade 3 PPE occurred in 13% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 PPE or Grade 3 PPE.

Hepatotoxicity: CABOMETYX in combination with nivolumab can cause hepatic toxicity with higher frequencies of Grades 3 and 4 ALT and AST elevations compared to CABOMETYX alone.

Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes than when the drugs are administered as single agents. For elevated liver enzymes, interrupt CABOMETYX and nivolumab and consider administering corticosteroids.

With the combination of CABOMETYX and nivolumab, Grades 3 and 4 increased ALT or AST were seen in 11% of patients. ALT or AST >3 times ULN (Grade ≥2) was reported in 83 patients, of whom 23 (28%) received systemic corticosteroids; ALT or AST resolved to Grades 0-1 in 74 (89%). Among the 44 patients with Grade ≥2 increased ALT or AST who were rechallenged with either CABOMETYX (n=9) or nivolumab (n=11) as a single agent or with both (n=24), recurrence of Grade ≥2 increased ALT or AST was observed in 2 patients receiving CABOMETYX, 2 patients receiving nivolumab, and 7 patients receiving both CABOMETYX and nivolumab.

Adrenal Insufficiency: CABOMETYX in combination with nivolumab can cause primary or secondary adrenal insufficiency. For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold CABOMETYX and/or nivolumab depending on severity.

Adrenal insufficiency occurred in 4.7% (15/320) of patients with RCC who received CABOMETYX with nivolumab, including Grade 3 (2.2%), and Grade 2 (1.9%) adverse reactions. Adrenal insufficiency led to permanent discontinuation of CABOMETYX and nivolumab in 0.9% and withholding of CABOMETYX and nivolumab in 2.8% of patients with RCC.

Approximately 80% (12/15) of patients with adrenal insufficiency received hormone replacement therapy, including systemic corticosteroids. Adrenal insufficiency resolved in 27% (n=4) of the 15 patients. Of the 9 patients in whom CABOMETYX with nivolumab was withheld for adrenal insufficiency, 6 reinstated treatment after symptom improvement; of these, all (n=6) received hormone replacement therapy and 2 had recurrence of adrenal insufficiency.

Proteinuria: Proteinuria was observed in 7% of CABOMETYX patients. Monitor urine protein regularly during CABOMETYX treatment. Discontinue CABOMETYX in patients who develop nephrotic syndrome.

Osteonecrosis of the Jaw (ONJ): ONJ occurred in <1% of CABOMETYX patients. ONJ can manifest as jaw pain, osteomyelitis, osteitis, bone erosion, tooth or periodontal infection, toothache, gingival ulceration or erosion, persistent jaw pain, or slow healing of the mouth or jaw after dental surgery. Perform an oral examination prior to CABOMETYX initiation and periodically during treatment. Advise patients regarding good oral hygiene practices. Withhold CABOMETYX for at least 3 weeks prior to scheduled dental surgery or invasive dental procedures, if possible. Withhold CABOMETYX for development of ONJ until complete resolution.

Impaired Wound Healing: Wound complications occurred with CABOMETYX. Withhold CABOMETYX for at least 3 weeks prior to elective surgery. Do not administer CABOMETYX for at least 2 weeks after major surgery and until adequate wound healing is observed. The safety of resumption of CABOMETYX after resolution of wound healing complications has not been established.

Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS, a syndrome of subcortical vasogenic edema diagnosed by characteristic findings on MRI, can occur with CABOMETYX. Evaluate for RPLS in patients presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue CABOMETYX in patients who develop RPLS.

Embryo-Fetal Toxicity: CABOMETYX can cause fetal harm. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Verify the pregnancy status of females of reproductive potential prior to initiating CABOMETYX and advise them to use effective contraception during treatment and for 4 months after the last dose.

ADVERSE REACTIONS

The most common (≥20%) adverse reactions are:

CABOMETYX as a single agent: diarrhea, fatigue, decreased appetite, PPE, nausea, hypertension, vomiting, weight decreased, constipation, and dysphonia.

CABOMETYX in combination with nivolumab: diarrhea, fatigue, hepatotoxicity, PPE, stomatitis, rash, hypertension, hypothyroidism, musculoskeletal pain, decreased appetite, nausea, dysgeusia, abdominal pain, cough, and upper respiratory tract infection.

DRUG INTERACTIONS

Strong CYP3A4 Inhibitors: If coadministration with strong CYP3A4 inhibitors cannot be avoided, reduce the CABOMETYX dosage. Avoid grapefruit or grapefruit juice.

Strong CYP3A4 Inducers: If coadministration with strong CYP3A4 inducers cannot be avoided, increase the CABOMETYX dosage. Avoid St. John’s wort.

USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed during CABOMETYX treatment and for 4 months after the final dose.

Hepatic Impairment: In patients with moderate hepatic impairment, reduce the CABOMETYX dosage. Avoid CABOMETYX in patients with severe hepatic impairment.