Ligand Reports First Quarter 2020 Financial Results

On May 6, 2020 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three months ended March 31, 2020 and provided an operating forecast and program updates (Press release, Ligand, MAY 6, 2020, View Source [SID1234557126]). Ligand management will host a conference call with slides today beginning at 4:30 p.m. Eastern time to discuss this announcement and answer questions.

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"Our first quarter financial results feature strong sales of Captisol to partners evaluating remdesivir in multiple clinical trials and scaling-up for potential treatment courses for COVID-19. We continue to meet Captisol requirements to support these trials as well as manufacturing scale-up, and are proud to play a role in developing potential treatments to address the pandemic both with Captisol and with various OmniAb and Vernalis-derived product candidates," said John Higgins, Chief Executive Officer of Ligand. "We recently closed a strategic acquisition, and throughout the first quarter we added a number of Shots on Goal with new agreements for various technologies, in particular our OmniAb platform. Overall, our business is performing very well, especially given the difficult business environment due to the pandemic. As such, we are pleased to be raising our 2020 financial guidance."

COVID-19 Impact

As the COVID-19 pandemic continues to evolve, our primary concern remains the health and safety of our employees and partners globally, while we continue to take actions to help address the pandemic.

We are supporting our employees through a range of programs, have enabled working from home and staggered operations in our labs and other critical work spaces. Importantly, Ligand is committed that there will be no COVID-19 related layoffs. Our corporate structure is spread across five sites in the U.S. and England, which positions us well to operate effectively in the current remote working environment. The Ligand team is ready to support a return to full operations with appropriate social distancing measures in place, following the lifting of shelter-in-place restrictions.

We have a very strong balance sheet, and we anticipate no material operational impacts for the rest of the year due to COVID-19. During the first quarter, COVID-19 did not have a material negative impact on our underlying business, financial condition, cash collections or liquidity. During the first quarter we reduced our outstanding convertible debt by approximately one-third given the favorable pricing on the bonds and the lower interest rate environment.

Our partnership with Gilead Sciences for remdesivir resulted in an increase in sales of Captisol to supply the scale up and manufacturing of that medicine as the first new treatment for COVID-19 available under an Emergency Use Authorization. We do not anticipate supply chain disruption for Captisol production at this time given inventory levels, risk management measures and operations at multiple sites throughout the world. We believe we are well positioned to meet Captisol requirements and are planning to make further capital investments in plant and operational capacity.

We have a large portfolio of more than 200 programs fully funded by more than 125 different pharmaceutical and biotechnology companies. We recently surveyed all our partners and found that the majority of them are generally in a relatively strong capital and operating condition with limited expected long-term impact on our partnered programs. Looking ahead to the remainder of the year and after thoroughly analyzing our business, we anticipate royalty and contract revenue will be lower than originally forecasted. We believe that patient access to certain medicines around the world will be disrupted over several months, which may decrease revenue for products from which we earn royalties. In addition, we anticipate that some partners will delay trial initiations or experience a slowdown in patient enrollment. These delays and slowdowns will likely reduce milestone payments for contract revenue due to Ligand. In addition, some smaller partners may face cash constraints or difficulty raising new capital, which could impact their ability to make payments to Ligand. Nonetheless, as the economy begins to reopen we expect our partners will resume important clinical and regulatory work on a wide array of partnered programs. Additionally, to date the increase in sales of Captisol has more than offset our projected decline in royalty and contract revenues. While the mix of revenue will be different than our original outlook, we anticipate total revenue and earnings to be higher in 2020 compared to our previous guidance.

Ligand has multiple programs relating to potential treatments for COVID-19. One is with Gilead for remdesivir, a nucleotide analogue issued an Emergency Use Authorization by the U.S. Food and Drug Administration on May 1, 2020. We also have two OmniAb partners with antibody programs in discovery stage, and a heat shock protein program that was added to our R&D programs when we acquired Vernalis.

First Quarter 2020 Financial Results

Total revenues for the first quarter of 2020 were $33.2 million, compared with $43.5 million for the same period in 2019. Royalties for the first quarter of 2020 were $6.6 million and primarily consisted of royalties from Kyprolis and EVOMELA. Royalties for the first quarter of 2019 were $19.5 million and included $14.2 million in royalties from Promacta; Ligand sold its Promacta license to Royalty Pharma as of March 6, 2019. Captisol sales were $21.1 million for the first quarter of 2020, compared with $9.0 million for the same period in 2019, primarily reflecting higher sales of Captisol for remdesivir. Effective this quarter, Ligand is presenting service revenue as a separate line item. Service revenue includes revenue generated from our Vernalis, Icagen and OmniChicken businesses as we collaborate with partners on early stage discovery and development work, and was $3.4 million for the first quarter of 2020, compared with $3.9 million for the same period in 2019. Contract revenue was $2.1 million for the first quarter of 2020, compared with $11.1 million for the same period in 2019, with the change driven by the timing of partner events.

Cost of Captisol was $4.7 million for the first quarter of 2020, compared with $3.9 million for the same period in 2019. Amortization of intangibles was $3.5 million for the first quarter of both 2020 and 2019. Research and development expense was $11.9 million for the first quarter of 2020, compared with $11.3 million for the same period of 2019. General and administrative expense was $9.3 million for the first quarter of 2020, compared with $11.1 million for the same period in 2019, with the decrease primarily attributable to lower legal expenses.

Net loss for the first quarter of 2020 was $(24.1) million, or $(1.46) per diluted share, compared with net income of $666.3 million, or $31.32 per diluted share, for the same period in 2019. The net loss for the first quarter of 2020 includes a non-cash change in the value of Ligand’s investments of $(25.5) million, while net income for the first quarter of 2019 includes a $17.3 million net non-cash gain from the value of Ligand’s investments as well as a $640.3 million gain, net of taxes, from the sale of the Promacta license. Adjusted net income for the first quarter of 2020 was $15.3 million, or $0.89 per diluted share, compared with $24.7 million, or $1.16 per diluted share, for the same period in 2019. Please see the table below for a reconciliation of net income/(loss) to adjusted net income.

As of March 31, 2020, Ligand had cash, cash equivalents and short-term investments of $738.8 million. During the first quarter of 2020, Ligand repurchased $234 million in principal amount of its convertibles notes at a price of $203 million, and repurchased 878,525 common shares for $73.3 million.

2020 Financial Guidance

Ligand is raising its 2020 financial guidance. Ligand now expects 2020 total revenues to be approximately $140 million and diluted EPS to be $3.65, up from previous guidance for total revenues of approximately $133 million and diluted EPS of $3.62. This increase reflects Ligand’s revised view on the business incorporating an estimated impact from COVID-19. The revised estimate of approximately $140 million in total revenues includes higher sales of Captisol, partially offset by reductions in royalties and contract revenue.

First Quarter 2020 and Recent Business Highlights

Kyprolis (carfilzomib), an Amgen Product Utilizing Captisol

Amgen reported first quarter Kyprolis sales of $280 million. Ono Pharmaceutical Co. will report first quarter Kyprolis sales in Japan on May 11, 2020.
OmniAb Platform Updates

There are now more than 80 OmniAb-related Shots on Goal in Ligand’s partnered portfolio, representing over 40% of the Ligand pipeline.
OmniAb partners have filed or been issued more than 30 U.S. and international patent applications or patents, claiming OmniAb-derived antibodies as the primary invention.
The number of active or recently completed clinical trials that include an OmniAb-derived antibody reached 43, with new clinical trial starts in Q1 that included:
two new Phase 1/1b trials;
three new Phase 2 trials; and
two new Phase 3 trials.
Two COVID-19 antibody programs are being pursued by Ligand partners. One is a multinational Big Pharma partner that has initiated a program using OmniChicken, and the other is focused on antibodies derived from OmniRat.
Immunovant announced positive results from its Phase 2a proof-of-concept study of OmniAb-derived IMVT-1401 in thyroid eye disease. IMVT-1401 is a novel investigational anti-FcRn antibody delivered by subcutaneous injection. The results showed a 65% mean reduction in total IgG observed from baseline to end of treatment, with a pharmacodynamic response nearly identical to modeled predictions for the dosing regimen tested in the trial. IMVT-1401 was generally well-tolerated.
Janssen initiated a Phase 1b trial of OmniAb-derived teclistamab (also known as JNJ-64007957) in combination with subcutaneous daratumumab in patients with multiple myeloma to identify a Phase 2 dose regimen and assess safety of the combination.
Gloria Biosciences submitted an application for marketing approval in China for OmniAb-derived zimberelimab for the treatment of classical Hodgkin lymphoma, marking multiple OmniAb drug applications filed seeking approval.
Arcus Biosciences and Taiho Pharmaceutical announced Taiho’s exercise of its option for an exclusive license to zimberelimab (also known as AB122) for Japan and other Asian countries, excluding China.
Ligand entered into an OmniAb Platform agreement with Pandion Therapeutics, and Pandion subsequently closed an $80 million financing and announced that proceeds will support the advancement of their pipeline of modular proteins and bi-functional antibodies for the treatment of autoimmune diseases.
CStone Pharmaceuticals and Blueprint Medicines initiated a Phase 1b/2 clinical trial of fisogatinib in combination with OmniAb-derived CS1001 for patients with hepatocellular carcinoma. CStone also announced that the first patient was dosed in the global proof-of-concept study of OmniAb-derived CS1001 in combination with Bayer’s regorafenib in patients with advanced solid tumors.
Harbour BioMed raised $75 million to fund the clinical development of an OmniAb-derived anti-FcRn antibody batoclimab, among other uses. Harbour also announced first patient dosing of Phase 1b/2a study of batoclimab for treating neuromyelitis optica spectrum disorder.
OmniAb partner GenMab highlighted DuoBody-PD-L1x4-1BB (GEN1046) at the J.P. Morgan investor conference in January; the GEN1046 anti-PD-L1 is derived from OmniRat.
Captisol Business Update

Ligand’s Captisol is a patented ultra-high pure form of sulfobutylether beta-cyclodextrin (SBECD) that is built upon drug master files maintained in multiple countries and has an extensive safety record for use in intravenous, inhaled, subcutaneous, oral, and ophthalmic formulations, among others. Ligand is the sole supplier of Captisol globally.
Ligand announced that it is supplying Captisol to partners for remdesivir in clinical trials and for manufacturing scale-up as a treatment for COVID-19.
On May 1, 2020 remdesivir was the first new treatment for COVID-19 to be issued an Emergency Use Authorization. Gilead announced that it intends to donate 1.5 million doses of remdesivir. Gilead has also stated that it is expanding manufacturing to increase product supply to over 1 million treatment courses by December 2020 ahead of potential increased demand.
On April 29, 2020 Gilead announced topline results from the open-label, Phase 3 SIMPLE trial evaluating 5-day and 10-day dosing durations of the investigational antiviral remdesivir in hospitalized patients with severe manifestations of COVID-19 disease. The study demonstrated that patients receiving a 5-day treatment course of remdesivir achieved similar improvement in clinical status compared with those taking a 10-day treatment course. The study demonstrated the potential for some patients to be treated with a 5-day regimen, which could significantly expand the number of patients who could be treated with Gilead’s current supply of remdesivir. The study results complement positive data from a placebo-controlled Phase 3 study of remdesivir conducted by the National Institute for Allergy and Infectious Diseases also reported on April 29, 2020, and may help to determine the optimal duration of treatment with remdesivir.
Ligand’s Captisol network is served by manufacturing plants in two European countries and five distribution facilities around the globe, all of which remain fully operational. Ligand has substantial capacity to supply Captisol manufactured according to cGMP and its focus is to ensure sufficient supply to meet all existing and future partner needs, and to supply Gilead. Ligand is also evaluating plans with its supply partners to further increase capacity by bringing additional sites online, if needed.
During the first quarter, Ligand entered into Captisol clinical use agreements with Double-Crane Pharmaceuticals and OnKure Therapeutics.
Vernalis Business Update

Ligand entered into an exclusive worldwide license agreement with Neuritek Therapeutics to develop and commercialize V158866, a novel oral, selective fatty acid amide hydrolase inhibitor that was discovered using the Vernalis Design Platform. Neuritek plans to develop V158866 for post-traumatic stress disorder and other CNS diseases. Under the terms of the agreement, Ligand will receive an upfront license fee and is eligible to receive over $240 million in milestones and tiered royalties on net sales six to eight percent. On March 31, 2020 Neuritek announced it had secured approximately $27 million in a capital commitment from GEM Global Yield LLC SCS.
Third-party academic drug analyses suggest a potential role for heat shock protein 90 (Hsp90) inhibitors in treating COVID-19 infection. Based on these studies, Ligand is evaluating potential collaborations or partnerships relating to intravenous luminespib (AUY-922) as a potential treatment for patients with COVID-19. Luminespib is a Phase 2-ready Hsp90 inhibitor, previously investigated in clinical trials for cancer.
Additional Pipeline and Partner Developments

Retrophin announced that the first 190 patients have been enrolled in its pivotal Phase 3 DUPLEX Study evaluating the safety and efficacy of sparsentan in focal segmental glomerulosclerosis.
Nucorion Pharmaceuticals initiated a Phase 1 clinical trial of NCO-48 Fumarate (NCO-1010), an oral prodrug of the nucleotide tenofovir utilizing Ligand’s LTP Platform technology for the potential treatment of hepatitis B. The first-in-human, double-blind, placebo-controlled, randomized, ascending single oral dose study will evaluate the safety and tolerability of NCO-48 Fumarate in healthy subjects. Topline results are expected in August 2020.
Palvella Therapeutics announced the completion of enrollment in its seamless Phase 2/3 VALO Study of PTX-022 (QTORIN 3.9% rapamycin anhydrous gel) for the treatment of adults with pachyonchia congenita.
Verona Pharma reported positive topline data in its 4-week Phase 2b COPD study with nebulized ensifentrine on top of tiotropium therapy, and also reported positive efficacy and safety data with a single dose of the pressurized metered-dose inhaler (pMDI) formulation of ensifentrine in a Phase 2 COPD study. Ensifentrine has demonstrated statistically significant and clinically meaningful improvements in lung function in Phase 2 studies in COPD patients when delivered via all three widely used inhaled formulations: nebulizer, DPI and pMDI.
Corvus Pharmaceuticals presented clinical data from its Phase 1b/2 trial of ciforadenant at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Genitourinary Cancers Symposium.
Ligand regained global rights to its novel glucagon receptor antagonist (formerly known as LGD-6972 and RVT-1502) from Metavant Sciences.
Business Development and Corporate Highlights

Following the close of the first quarter, Ligand completed its acquisition of the core assets of Icagen, Inc.’s North Carolina operations, including partnered programs, proprietary ion channel screening and assay platforms, x-ray fluorescence capabilities, custom screening technologies and novel unpartnered preclinical-stage molecules for $15 million in cash.
Ligand announced the launch of its Environmental, Social and Corporate Responsibility Governance section of its corporate website. Please visit this link for current disclosures.
Adjusted Financial Measures

The Company reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation, gain on the sale of Promacta and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. However, other than with respect to total revenues, the Company only provides financial guidance on an adjusted basis and does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, stock-based compensation expense and effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

Conference Call

Ligand management will host a conference call with slides today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (833) 325-0071 from the U.S. or (720) 405-1612 from outside the U.S., using the conference ID 5190682. To participate via live or replay webcast, a link is available at www.ligand.com. Slides to accompany the conference call are available here.

MannKind Corporation Reports 2020 First Quarter Financial Results

On May 6, 2020 MannKind Corporation (NASDAQ:MNKD) reported financial results for the quarter ended March 31, 2020 (Press release, Mannkind, MAY 6, 2020, View Source [SID1234557125]).

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"We are pleased to report first quarter Afrezza net revenue of $8.0 million, which is 58% higher than the same quarter in 2019," said Michael Castagna, Chief Executive Officer of MannKind Corporation. "We believe that some of the increased revenue this quarter reflects the impact of patients stocking up on extra refills in advance of the stay-at-home orders that have since been implemented across the country. In response to the COVID-19 pandemic, we have rapidly implemented digital tools and programs to help our sales force and our Afrezza prescribers navigate this challenging time for our healthcare system. In addition, our manufacturing and development team in Connecticut remain focused on maintaining supply of Afrezza and meeting our obligations under our collaboration with United Therapeutics."

Total revenues were $16.2 million for the first quarter of 2020, reflecting Afrezza net revenue of $8.0 million and collaboration and services revenue of $8.2 million. Afrezza net revenue increased 58% compared to $5.1 million in the first quarter of 2019, primarily driven by higher product demand as well as a price increase and a more favorable mix of cartridges. Collaboration and services revenue for the first quarter of 2020 decreased $4.2 million compared to the first quarter of 2019, primarily due to the substantial completion of the research agreement with United Therapeutics in the second quarter of 2019.

Afrezza gross profit for the first quarter of 2020 was $3.8 million vs. $1.1 million in the same period of 2019, a 263% increase that was driven primarily by higher Afrezza revenue. Cost of goods sold increased by $0.1 million which included an increase related to Afrezza unit sales growth and an inventory write-off offset by a greater amount of costs capitalized to inventory due to a higher volume of manufacturing activities in the first quarter of 2020. Gross margin in the first quarter of 2020 increased to 48%, our highest gross margin to date, from 21% for the same quarter in 2019, primarily due to higher Afrezza revenue.

Selling, general and administrative expenses for the first quarter of 2020 were $14.4 million compared to $25.7 million for the first quarter of 2019. This 44% decrease was primarily due to $9.3 million spent on direct-to-consumer television advertising in 2019, which was not repeated in 2020, a $1.1 million decrease in promotional and marketing activities and $0.8 million decrease in personnel and employee related costs.

Net interest expense for the first quarter of 2020 was $2.2 million compared to $1.4 million for the first quarter of 2019. This $0.8 million increase was due to a higher balance of outstanding principal and an increase in the interest rate of certain promissory notes.

The net loss for the first quarter of 2020 was $9.3 million, or $0.04 per share, compared to a $14.9 million net loss in the first quarter of 2019, or $0.08 per share. The lower net loss is mainly attributable to a decrease in operating expenses of $7.6 million. The reduction in the net loss per share was impacted by the lower operating expenses and a greater number of outstanding shares.

Cash, cash equivalents and restricted cash at March 31, 2020 was $39.2 million compared to $50.2 million at December 31, 2019, which also included short-term investments of $20.0 million. The decrease was primarily due to net cash used in operating activities of $11.2 million in the first quarter of 2020.

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.

MannKind will host a conference call and presentation webcast to discuss these results today at 5:00 p.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 telephone replay of the call will be accessible for approximately 14 days following completion of the call by dialing (844) 512-2921 or (412) 317-6671 and use the participant passcode: 7127207#. A replay will also be available on MannKind’s website for 14 days.

Incyte Announces FDA Approval of Tabrecta™ (capmatinib) for the Treatment of Patients with Metastatic Non-Small Cell Lung Cancer with METex14

On May 6, 2020 Incyte (Nasdaq:INCY) reported that the U.S. Food and Drug Administration (FDA) has approved TabrectaTM (capmatinib) for treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to MET exon 14 skipping (METex14) as detected by an FDA-approved test (Press release, Incyte, MAY 6, 2020, View Source [SID1234557123]). This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Tabrecta, the first and only treatment approved to specifically target NSCLC with this driver mutation, is approved for first-line and previously treated patients regardless of prior treatment type.

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Novartis has exclusive worldwide development and commercialization rights to Tabrecta, and the FDA approval of Tabrecta triggers $70 million in milestone payments from Novartis to Incyte. Incyte is also eligible to receive 12-14 % royalties on net sales of Tabrecta by Novartis.

NSCLC accounts for approximately 85% of lung cancer diagnoses1. METex14 occurs in 3-4% of newly-diagnosed metastatic NSCLC cases2 and is a recognized oncogenic driver3,4.

"We are pleased that the FDA has approved Tabrecta for patients with METex14 NSCLC," said Steven Stein, M.D., Chief Medical Officer, Incyte. "Having a therapy that targets the recognized oncogenic driver will provide a much needed treatment option for patients with METex14 NSCLC who currently have limited treatment options. Tabrecta is the fourth Incyte molecule to be approved by the FDA, highlighting our world-class discovery program and commitment to bringing innovative medicines to patients in need."

The approval of Tabrecta is based on results5 from the pivotal GEOMETRY mono-1 Study. In the METex14 population (n=97), the confirmed overall response rate was 68% (95% CI, 48-84) and 41% (95% CI, 29-53) among treatment-naive (n=28) and previously treated patients (n=69), respectively, based on the Blinded Independent Review Committee (BIRC) assessment per RECIST v1.1. In patients taking Tabrecta, the study also demonstrated a median duration of response of 12.6 months (95% CI, 5.5–25.3) in treatment-naive patients (19 responders) and 9.7 months (95% CI, 5.5-13.0) in previously treated patients (28 responders). The most common treatment-related adverse events (AEs) (incidence ≥20%) are peripheral edema, nausea, fatigue, vomiting, dyspnea, and decreased appetite.

Capmatinib was previously granted Breakthrough Therapy designation by the FDA, which is designed to expedite the development and review of drugs for serious conditions that have shown encouraging early clinical results and may demonstrate substantial improvements over available medicines.

Full prescribing information for Tabrecta can be found at: View Source

About GEOMETRY mono-1

The Novartis-sponsored GEOMETRY mono-1 trial is a multi-center, non-randomized, open-label, multi-cohort Phase 2 study to evaluate the efficacy and safety of single-agent capmatinib in adult patients with EGFR wild-type, metastatic NSCLC as measured by ORR.

Patients (n=97) with metastatic NSCLC harboring mutations that lead to METex14 (centrally confirmed) were assigned to Cohorts 4 (n=69, previously treated patients) or 5B (n=28, treatment-naïve), and received 400 mg capmatinib tablets orally twice daily. The major efficacy outcome was ORR based on BIRC assessment per RECIST v1.1. An additional efficacy outcome was DOR by BIRC.

About Tabrecta

Tabrecta (capmatinib; formerly INC280) is a kinase inhibitor that targets MET discovered by Incyte and licensed to Novartis in 2009. Under the terms of the Agreement, Incyte granted Novartis exclusive worldwide development and commercialization rights to capmatinib and certain back-up compounds in all indications. Incyte is eligible for over $500 million in future milestones as well as royalties of between 12 and 14 percent on global sales by Novartis.

Novartis announces FDA approval of MET inhibitor Tabrecta™ for metastatic non-small cell lung cancer with METex14

On May 6, 2020 Novartis reported that the US Food and Drug Administration (FDA) approved Tabrecta (capmatinib, formerly INC280), an oral MET inhibitor for adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to MET exon 14 skipping (METex14) as detected by an FDA-approved test (Press release, Novartis, MAY 6, 2020, View Source [SID1234557122]). This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

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This approval fills a long-recognized and urgent need among METex14 patients who have not had a treatment option approved to specifically target the driver of their lung cancer3. Tabrecta is approved for first-line and previously treated patients, regardless of prior treatment type, and is expected to be available to patients in the coming days.

The FDA also approved FoundationOneCDx as the companion diagnostic for Tabrecta, to aid in detecting mutations that lead to MET exon 14 skipping in tumor tissue.

"Non-small cell lung cancer is a complex disease, with many different possible mutations that may encourage the cancer’s growth," said Juergen Wolf, MD, from the Center for Integrated Oncology, University Hospital Cologne and lead investigator of the GEOMETRY study. "MET exon 14 skipping is a known oncogenic driver. With today’s decision by the FDA, we can now test for and treat this challenging form of lung cancer with a targeted therapy, offering new hope for patients with NSCLC harboring this type of mutation."

Novartis was previously granted Breakthrough Therapy Designation for capmatinib. According to FDA guidelines, treatments that receive Breakthrough Therapy Designation must target a serious or life-threatening disease and demonstrate a substantial improvement over existing therapies on one or more significant preliminary research endpoints.

The approval of Tabrecta is based on results from the pivotal GEOMETRY mono-1 Phase II multi-center, non-randomized, open-label, multi-cohort study. In the METex14 population (n=97), the confirmed overall response rate was 68% (95% CI, 48-84) and 41% (95% CI, 29-53) among treatment-naive (n=28) and previously treated patients (n=69), respectively, based on the Blinded Independent Review Committee (BIRC) assessment per RECIST v1.11. In patients taking Tabrecta, the study also demonstrated a median duration of response of 12.6 months (95% CI, 5.5–25.3) in treatment-naive patients (19 responders) and 9.7 months (95% CI, 5.5-13.0) in previously treated patients (28 responders)1. The most common treatment-related adverse events (AEs) (incidence ≥20%) are peripheral edema, nausea, fatigue, vomiting, dyspnea, and decreased appetite1.

"Today, and especially during these difficult times, we are incredibly proud that Tabrecta is the first treatment approved by the FDA specifically to treat patients diagnosed with this aggressive NSCLC associated with METex14," said Susanne Schaffert, PhD, President, Novartis Oncology. "In our quest to reimagine medicine, we have worked tirelessly over the past decades to advance the understanding and treatment of NSCLC, striving to make a difference in patients’ lives, one mutation at a time. We thank all the physicians, patients and families involved in the Tabrecta clinical trials, and we remain committed to advancing innovative solutions for the patients we work to serve."

NSCLC accounts for approximately 85% of the 2 million new lung cancer diagnoses each year worldwide, including about 228,000 in the United States4-5. Nearly 70% of NSCLC patients have a genomic mutation6. METex14, a recognized oncogenic driver, occurs in approximately 3%-4% of newly diagnosed metastatic NSCLC cases (about 4,000 – 5,000 patients in the US annually)7-9, 2.

"With NSCLC, understanding whether a mutation is driving the cancer is critical, and it’s important for doctors and patients to use comprehensive biomarker testing at the time of diagnosis or progression to check for mutations like those that cause METex14," said Andrea Ferris, President and CEO of LUNGevity. "Knowing more about the molecular makeup of their tumor will help patients and their healthcare teams make informed treatment-related decisions from the start."

Novartis is committed to providing patients with access to medicines, as well as resources and support to address a range of needs. The Novartis Oncology Patient Support Program is available to help guide eligible patients through the various aspects of getting started on treatment, from providing educational information to helping them understand their insurance coverage and identify potential financial assistance options. Patients or providers can call 800-282-7630 or visit Patient.NovartisOncology.com or HCP.Novartis.com/Access to learn more about eligibility and to enroll.

Full prescribing information for Tabrecta can be found at View Source

About Tabrecta (capmatinib)
Tabrecta (capmatinib) is a kinase inhibitor that targets MET. Tabrecta is licensed to Novartis by Incyte Corporation in 2009. Under the Agreement, Incyte granted Novartis worldwide exclusive development and commercialization rights to capmatinib and certain back-up compounds in all indications.

About GEOMETRY mono-1
GEOMETRY mono-1 is a Phase II a multi-center, non-randomized, open-label, multi-cohort study in adult patients with EGFR wild-type, metastatic NSCLC as measured by ORR.

The trial evaluated 97 adult patients with metastatic NSCLC harboring mutations that lead to METex14 (centrally confirmed) who were assigned to Cohorts 4 (n=69, previously treated patients) or 5b (n=28, treatment-naive), and received capmatinib tablets 400 mg orally twice daily.

The major efficacy outcome is ORR based on BIRC assessment per RECIST v1.1. An additional efficacy outcome is duration of response by BIRC.

Novartis Commitment to Lung Cancer
Worldwide, lung cancer causes more deaths than colon, breast and prostate cancer combined, and more than 2 million new cases of lung cancer are diagnosed each year4. Despite treatment advances, many patients with NSCLC still have a poor prognosis and limited treatment options3. This includes the nearly 70% of NSCLC patients who have a genomic mutation6. To determine the most appropriate treatment, medical organizations recommend comprehensive genomic testing for patients with lung cancer as part of their upfront diagnosis7.

Novartis Oncology’s research has helped transform treatment approaches for patients living with NSCLC. Novartis continues its commitment to the global lung cancer community through ongoing studies, as well as the exploration of investigational compounds in NSCLC, including those that target genetic biomarkers and tumor promoting inflammation.

Indication
TABRECTA (capmatinib) tablets is a prescription medicine used to treat adults with a kind of lung cancer called non-small cell lung cancer (NSCLC) that has spread to other parts of the body or cannot be removed by surgery (metastatic), and whose tumors have an abnormal mesenchymal-epithelial transition (MET) gene.

The effectiveness of TABRECTA in these patients is based on a study that measured 2 types of response to treatment (response rate and duration of response). There is no clinical information available to show if patients treated with TABRECTA live longer or if their symptoms improve. There are ongoing studies to find out how TABRECTA works over a longer period of time.

It is not known if TABRECTA is safe and effective in children.

Important Safety Information
TABRECTA may cause serious side effects, such as lung or breathing problems. TABRECTA may cause inflammation of the lungs during treatment that may lead to death. Patients should be advised to contact their health care provider right away if they develop any new or worsening symptoms, including cough, fever, trouble breathing, or shortness of breath.

TABRECTA may cause abnormal blood test results, which may be a sign of liver problems. Patients should be advised that their health care provider will do blood tests to check their liver before starting and during treatment with TABRECTA. Patients should be advised to contact their health care provider right away if they develop any signs and symptoms of liver problems including the skin or the white part of their eyes turning yellow (jaundice), dark or "tea-colored" urine, light-colored stools (bowel movements), confusion, loss of appetite for several days or longer, nausea and vomiting, pain, aching, or tenderness on the right side of the stomach area (abdomen), or weakness or swelling in the stomach area.

The skin may be sensitive to the sun (photosensitivity) during treatment with TABRECTA. Patients should be advised to use sunscreen or wear clothes that cover their skin during treatment with TABRECTA to limit direct sunlight exposure.

For women of reproductive potential, TABRECTA can harm their unborn baby. They should use an effective method of birth control during treatment with TABRECTA and for 1 week after the last dose. Men who have partners who can become pregnant should use effective birth control during treatment with TABRECTA and for 1 week after the last dose.

Before taking TABRECTA, patients should tell their health care provider about all their medical conditions, including if they have or have had lung or breathing problems other than lung cancer, have or have had liver problems, or if they are pregnant or plan to become pregnant, as TABRECTA can harm their unborn babies. Females who are able to become pregnant should have a pregnancy test before they start treatment with TABRECTA and should use effective birth control during treatment and for 1 week after the last dose of TABRECTA. Patients should be advised to talk to their health care provider about birth control choices that might be right for them during this time and to tell their health care provider right away if they become pregnant or think they may be pregnant during treatment with TABRECTA. Males who have female partners who can become pregnant should use effective birth control during treatment and for 1 week after their last dose of TABRECTA.

Patients should tell their health care provider about all the medicines they take or start taking, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of TABRECTA include swollen hands, ankles, or feet (peripheral edema); nausea and/or vomiting; tiredness and/or weakness (fatigue, asthenia); shortness of breath (dyspnea); loss of appetite; changes in bowel movements (diarrhea or constipation); cough; pain in the chest; fever (pyrexia); back pain; and decreased weight.

Triumvira Announces Grant of T Cell Antigen Coupler (TAC) Technology Patent by U.S. Patent and Trademark Office

On May 6, 2020 Triumvira Immunologics (Triumvira), a privately held biopharmaceutical company developing a novel platform for engineering T-cells to attack cancers, reported the issuance of key claims in U.S. Patent No. 10640562 entitled "T cell-antigen coupler with various construct optimizations (Press release, Triumvira Immunologics, MAY 6, 2020, View Source [SID1234557121])." The newly allowed TAC-CD19 composition-of-matter claims build on other composition-of-matter claims previously granted by the US Patent and Trademark Office and expand the protection of the company’s TAC platform and therapeutic product candidates.

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"Issuance of our composition-of-matter claims further strengthens our position as a company pioneering novel engineered T cell approaches as we are bringing this innovative treatment to patients."

"Expanding intellectual property protection of our TAC platform is key to developing a solid technology platform, and this issuance confirms that the TAC is a well-differentiated technology," said Dr. Paul Lammers, Triumvira’s President and CEO. "Issuance of our composition-of-matter claims further strengthens our position as a company pioneering novel engineered T cell approaches as we are bringing this innovative treatment to patients."

Triumvira’s autologous and allogeneic preclinical data previously revealed unique biological differences of TAC-engineered T cells compared to second-generation CAR-T cells, with TAC-T cells producing greater anti-tumor efficacy and no evidence of toxicity, particularly in models of solid tumors.