On October 28, 2021 Takeda Pharmaceutical Company Limited (TOKYO:4502/NYSE:TAK) ("Takeda") reported financial results for the first half of fiscal year 2021 (period ended September 30, 2021) (Press release, Takeda, OCT 28, 2021, View Source [SID1234592131]). Based on the strong first half results, the company also confirmed its fiscal year 2021 management guidance.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Fiscal year 2021, ending in March 31, 2022, continues to be a year of growth with anticipated topline acceleration driven by the company’s 14 global brands. In addition, the company is committed to the potential of its highly innovative R&D strategy built on a combination of in-house innovation and strategic partnerships that tap cutting-edge science at the source. Recent key pipeline wins, including the U.S. Food and Drug Administration (FDA) approval of EXKIVITY and unanimous recommendation for approval of maribavir by a U.S. FDA Advisory Committee further underscore its promise and potential. The company also announced today the intent to buy back shares up to 100B yen, underscoring confidence in its business strategy and commitment to delivering value to shareholders.

Christophe Weber, Chief Executive Officer, commented:

"Our strategic vision to discover and deliver life-transforming treatments will be realized by the strength of our leading products and our innovative pipeline. Takeda’s Q2 and first-half results are evidence of progress and conviction in our strategy while consistently delivering on our fundamentals. As a result, we are confirming full-year FY2021 guidance as we track toward topline growth and strong core operating profit margins."
"Takeda’s growth continues to be driven by our 14 Global Brands, which will remain our primary growth driver for the coming years. In addition, our ambitious pipeline is starting to deliver results, including the recent U.S. FDA approval of EXKIVITY. This underscores the potential of the pipeline to transform lives and our business."
"The announcement of our new share buyback program, approved by Takeda’s Board of Directors, further demonstrates commitment to delivering shareholder value and our confidence in our business strategy."
"Takeda’s strong commercial portfolio and R&D pipeline are diversified across four core therapeutic areas and represent innovative and potentially transformative benefits for patients. Altogether, we believe that the combination of these growth drivers will continue to propel our business forward and help to ensure our future growth is resilient, for not just the next quarter but the next decade."

(a) Further information on certain of Takeda’s Non-IFRS measures is posted on Takeda’s investor relations website at View Source." target="_blank" title="View Source." rel="nofollow">View Source
(b) Underlying growth compares two periods (quarters or years) of financial results under a common basis and is used by management to assess the business. These financial results are calculated on a constant currency basis and excluding the impact of divestitures and other amounts that are unusual, non-recurring items or unrelated to our ongoing operations.
(c) Core Operating Profit represents net profit adjusted to exclude income tax expenses, the share of profit or loss of investments accounted for using the equity method, finance expenses and income, other operating expenses and income, amortization and impairment losses on acquired intangible assets and other items unrelated to Takeda’s core operations, such as non-recurring items, purchase accounting effects and transaction related costs.
(d) Free Cash Flow represents cash flows from operating activities, excluding acquisition of plant, property and equipment, intangible assets and investments, and any other cash that is not available to Takeda’s immediate or general business use, and including proceeds from sales of property, plant, sales and redemption of investments and businesses, net of cash and cash equivalents divested.
View Source

Reported Revenue increased +12.8%, Underlying Core Revenue increased +6.8% vs H1 FY2020, driven by 14 global brands

Takeda’s 14 global brands, with an aggregate reported revenue of 692.2 billion yen ($6.2B), posted year-over-year underlying revenue growth of +11.4% and now represent 42% of total core revenue, with further acceleration expected in H2.
Takeda’s 5 key business areas with 1,434.6 billion yen ($12.9B) in reported revenue.
GI with 429.1 billion yen ($3.85B) in reported revenue, with underlying revenue growth of +8% spearheaded by gut-selective ENTYVIO.
Rare Diseases with 300.1 billion yen ($2.69B) in reported revenue declined -2% on an underlying basis with rare hematology decline in line with expectations due to competition and HAE growth being impacted by phasing dynamics; remain on track toward full year forecast.
Plasma Derived Therapy (PDT) Immunology with 238.0 billion yen ($2.13B) in reported revenue, with underlying revenue growth +11% driven by Immunoglobulin and ALBUMIN/FLEXBUMIN.
Oncology with 233.7 billion yen ($2.09B) in reported revenue, with underlying revenue growth of +8% driven by indication expansion across portfolio.
Neuroscience with 233.7 billion yen ($2.09B) in reported revenue, with underlying revenue growth of +9% driven by strong rebound of VYVANSE following impact of COVID-19 in prior year.
Reported Operating Profit increased +60.5%; Underlying Core Operating Profit Margin was 29.1% for H1

Reported operating profit increased +60.5% to 346.0 billion yen ($3.1B) compared to H1 FY2020, reflecting a gain on the sales of the diabetes portfolio in Japan and declining purchase price accounting and integration costs.
Core operating profit for the current period decreased -4.3% due to divestitures and increased R&D investment. On track towards full year forecast of 930.0 billion yen ($8.34B)
Important R&D Milestones as Innovative Pipeline Begins to Deliver

Received U.S. FDA approval for EXKIVITY (mobocertinib) as the first and only approved oral therapy specifically designed for patients with EGFR Exon20 insertion mutations+ NSCLC, with filing under review in China and other countries.
Received unanimous recommendation from a U.S. FDA Advisory Committee for maribavir as a treatment for post-transplant recipients with refractory CMV infection with or without resistance. A decision is expected by next month (PDUFA November 23, 2021).
Partnership with Novavax in Japan for development, manufacturing (250 million doses per year) and commercialization of TAK-019, their COVID-19 vaccine candidate with distribution in Japan expected to begin in early calendar year 2022, subject to regulatory approval.
Received approval from the Japan Ministry of Health, Labour and Welfare for Alofisel (darvadstrocel) to be manufactured and marketed for the treatment of complex perianal fistulas in patients with non-active or mildly active luminal Crohn’s disease.
Announced an exclusive collaboration and license agreement with JCR Pharmaceuticals to commercialize JR-141 (INN: pabinafusp alfa) for the treatment of Hunter syndrome (also known as Mucopolysaccharidosis type II or MPS II).
Announced intent to acquire Gamma Delta Therapeutics to accelerate the development of allogeneic gamma delta T-cell therapies with the intention to finalize deal in Q1 FY22. Closing of the transaction is contingent on completion of review under antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the U.S.
Entered into three next generation gene therapy collaborations, including with Selecta Biosciences, Poseida Therapeutics and Immusoft.
Additional Pipeline Developments

Suspended dosing of patients in two Phase 2 studies of TAK-994, an investigational oral orexin agonist for the treatment of narcolepsy type 1 (NT1), after a liver toxicity signal emerged in two clinical studies. Takeda, as well as many experts and regulatory authorities, remain excited and optimistic about the potential of orexin agonists given the transformative efficacy demonstrated by TAK-994 in NT1 patients, and Takeda has a number of differentiated molecules in the pipeline that are part of its orexin franchise.
Announced that the Phase 3 PANTHER (pevonedistat-3001) study did not achieve statistical significance for the primary endpoint of event-free survival.
Other Important Developments in H1

Broke ground in Woodlands, Singapore for company’s first building to follow the Singapore Green Mark Zero Energy certification scheme as the first ‘net zero carbon emissions’ building in its global network and first-of-its-kind investment within the biotechnology industry in Singapore.
Selected four new partners for the annual global Corporate Social Responsibility program to help strengthen health systems in low- and middle-income countries.
(a) Previously 160 yen per Share. This change reflects the recording of a tax provision involving Irish taxation of the break fee Shire received from AbbVie in connection with the terminated offer to acquire Shire made by AbbVie in 2014. This change reflects an update to Takeda’s forecasted net income attributable to owners of the Company for the fiscal year ending March 31, 2022 filed today with the Tokyo Stock Exchange. See the release entitled "Summary of Financial Statements for the Six-month Period Ended September 30, 2021 (IFRS, Consolidated)" for additional information.

Key Assumptions in FY2021 Forecast

Company guidance reflects management’s expectations for continued business momentum across Takeda’s five key business areas and underlying revenue growth of its 14 global brands, while also increasing investment in R&D.

FY2021 guidance reflects key assumptions, including (1) Takeda expects at least one 505(b)2 competitor for subcutaneous VELCADE to launch in the U.S. around mid FY2021; (2) Takeda does not expect to restart sales of NATPARA in the U.S. market in FY2021; and (3) FY2021 guidance does not include the impact of any potential further divestitures beyond what has already been disclosed by Takeda.

To date, Takeda has not experienced a material effect on its financial results as a result of the global spread of the novel coronavirus infectious disease (COVID-19). Based on currently available information, Takeda believes that its financial results for FY2021 will not be materially affected by COVID-19 and, accordingly, Takeda’s FY2021 forecast reflects this belief. However, the situation surrounding COVID-19 remains highly fluid, and future COVID-19-related developments in FY2021, including new or additional COVID-19 outbreaks and additional or extended lockdowns, shelter-in-place orders or other government action in major markets, could result in further or more serious disruptions to Takeda’s business, such as slowdowns in demand for Takeda’s products, supply chain related issues or significant delays in its clinical trial programs. These events, if they occur, could result in an additional impact on Takeda’s business, results of operations or financial condition, as well as result in significant deviations from Takeda’s FY2021 forecast.

For more details on Takeda’s H1 FY2021 results and other financial information, please visit: View Source

On October 28, 2021 CohBar, Inc. (NASDAQ: CWBR) (the "Company"), a clinical stage biotechnology company developing mitochondria based therapeutics to treat chronic diseases and extend healthy lifespan, reported the pricing of an underwritten public offering of 20,833,334 shares of the Company’s common stock and accompanying warrants to purchase 20,833,334 shares of common stock at a price to the public of $0.72 per share and accompanying warrant (Press release, CohBar, OCT 28, 2021, View Source [SID1234592130]). Each warrant will have an exercise price of $0.72 per share and be exercisable for 5 years from the closing date of the offering. The aggregate gross proceeds from this offering are expected to be approximately $15.0 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by the Company. The offering is expected to close on or about November 1, 2021, subject to customary closing conditions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Cantor Fitzgerald & Co. is acting as sole book-running manager for the offering. Brookline Capital Markets, a division of Arcadia Securities, LLC, is acting as co-manager.

CohBar intends to use the net proceeds from the offering, together with its existing cash resources, for general corporate purposes, which may include funding preclinical and clinical development of its peptides, increasing working capital, operating expenses and capital expenditures.

The securities will be issued pursuant to an effective shelf registration statement filed with the Securities and Exchange Commission (SEC) on August 24, 2020. A final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the prospectus supplement, together with the accompanying prospectus, can be obtained at the SEC’s website at www.sec.gov or from Cantor Fitzgerald & Co., Attn: Capital Markets, 499 Park Ave., 4th Floor, New York, New York 10022, or by e-mail at [email protected].

This press release does not and shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction. Any offer, if at all, will be made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement.

On October 28, 2021 Candel Therapeutics, Inc. (Nasdaq: CADL), a late clinical stage biopharmaceutical company developing novel oncolytic viral immunotherapies, reported that data on patient-reported tolerability assessment of intraprostatic injections will be presented in a virtual poster session at the 28th Annual Prostate Cancer Foundation Scientific Retreat (Press release, Candel Therapeutics, OCT 28, 2021, View Source [SID1234592129]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Date: Thursday, October 28, 2021
Presenter: Laura K. Aguilar, MD, PhD, Chief Medical Officer at Candel Therapeutics, Inc.
Presentation Title: Patient experience with intraprostatic injection of CAN-2409 or placebo followed by valacyclovir in a phase 3 clinical trial for localized prostate cancer in combination with standard of care radiation therapy with or without androgen suppression.
The data were generated from the company’s ongoing phase 3 clinical trial, which is evaluating safety and efficacy of intra-prostatic injection with CAN-2409 or placebo followed by oral valacyclovir prodrug in combination with standard of care (radiation therapy ± short-term androgen deprivation therapy) in patients with localized prostate cancer having intermediate-risk or a single NCCN high-risk factor.

Clinical trial enrollment has been completed with a diverse population of 745 patients from 51 sites in the U.S. More than 2,000 injection procedures have been performed (40% transperineal, 56% transrectal, 4% not reported). Information on the patient experience is being collected with a questionnaire, and data is available from 32 patients who completed the questionnaire within 3 months of completing treatment. For the transperineal procedure, 65% of patients reported the intra-prostatic injections to be "the same or better" tolerated than a prostate biopsy, 30% "a little harder" to tolerate and 4% "much harder" to tolerate than a biopsy. For the transrectal procedure, 89% of patients reported the injections to be "the same or better" tolerated than a biopsy and 11% "a little harder" to tolerate than a biopsy. All patients (100%) reported overall feeling positive about their involvement in the study.

"These data further support the tolerability and patient receptiveness of intra-prostatic injection of CAN-2409 and its comparability to routine biopsies being performed in this patient population," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel Therapeutics. "Listening to patients is important as we look towards the commercialization of CAN-2409, if approved. We believe CAN-2409 holds great promise as a treatment for patients with localized, non-metastatic prostate cancer, which may improve disease outcome, while eliminating the need for long-term androgen deprivation therapy and its associated side effects."

Details from the presentations will be available on the Candel website at View Source

About CAN-2409

CAN-2409, Candel’s most advanced oncolytic viral immunotherapy candidate, is a replication-deficient adenovirus that delivers the herpes simplex virus thymidine kinase (HSV-tk) gene to cancer cells. HSV-tk is an enzyme that locally converts orally administered valacyclovir into a toxic metabolite that kills nearby cancer cells. The intra-tumoral administration results in the release of tumor-specific neoantigens in the microenvironment. At the same time, the adenoviral serotype 5 capsid protein elicits a strong pro-inflammatory signal in the tumor microenvironment. This creates the optimal conditions to induce a CD8+ T cell mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity.

Because of its versatility, CAN-2409 has the potential to treat a broad range of solid tumors. Monotherapy activity as well as combination activity with standard of care radiotherapy, surgery, chemotherapy, and immune checkpoint inhibitors have previously been shown in several preclinical and clinical settings. Furthermore, CAN-2409 presents a favorable tolerability profile; more than 700 patients have been dosed to date, supporting the potential for combination with other therapeutic strategies without inordinate concern of overlapping adverse events. Currently, Candel is evaluating the effects of treatment with CAN-2409 in localized, non-metastatic prostate cancer, non-small cell lung cancer, high-grade glioma, and pancreatic cancer in ongoing clinical trials.

About the Phase 3 Clinical Trial in Prostate Cancer

The pivotal phase 3 study of CAN-2409 immunotherapy in patients with intermediate-high risk localized prostate cancer is a placebo-controlled, randomized clinical trial to evaluate CAN-2409 treatment in combination with valacyclovir added to standard of care (radiation therapy ± short-term androgen deprivation therapy). The primary endpoint of the study is disease-free survival. Secondary endpoints include prostate cancer specific survival, overall survival, freedom from biochemical failure, patient reported health-related quality of life, and safety.

On October 28, 2021 West Pharmaceutical Services, Inc. (NYSE: WST) reported its financial results for the third-quarter 2021 and updated full-year 2021 financial guidance (Press release, West Pharmaceutical Services, OCT 28, 2021, View Source [SID1234592128]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Third-Quarter 2021 Summary (comparisons to prior-year period)

Net sales of $706.5 million grew 28.9%; organic sales growth was 27.9%.
Reported-diluted EPS of $2.31 increased 112%.
Adjusted-diluted EPS of $2.06 increased 79%.
Company is raising full-year 2021 net sales guidance to a new range of $2.800 billion to $2.810 billion, compared to a prior range of $2.760 billion to $2.785 billion.
Company is raising full-year 2021 adjusted-diluted EPS guidance to a new range of $8.40 to $8.50, compared to a prior range of $8.05 to $8.20.
The Company also announced that its Board of Directors has approved a fourth-quarter 2021 dividend of $0.18 per share, a 5.9% increase over the $0.17 per share paid in each of the four preceding quarters. This is the twenty-ninth consecutive annual increase in the Company’s dividend. The dividend will be paid on November 17, 2021, to shareholders of record as of November 10, 2021.
"Adjusted-diluted EPS" and "organic sales growth" are Non-U.S. GAAP measurements. See discussion under the heading "Non-U.S. GAAP Financial Measures" in this release.

"We had robust growth in all three of our Proprietary Products market units, led by the sales of components in our High-Value Product (HVP) portfolio," said Eric M. Green, President and Chief Executive Officer. "This quarter’s strong performance came from both our base business, especially in the Biologics market unit, and COVID-19 related sales. Demand continues to grow for our premium offerings, such as NovaPure and FluroTec components, and, as a result, we are again increasing our planned capital expenditures, commencing next year, to expand HVP capacity at existing sites."

Proprietary Products Segment
Net sales grew by 36.9% to $577.0 million. Organic sales growth was 35.7%, with currency translation increasing sales growth by 120 basis points. HVP sales represented over 70% of segment sales and generated double-digit organic sales growth, led by customer demand for Westar, FluroTec and NovaPure components and Daikyo Crystal Zenith containers.

All three market units – Biologics, Generics and Pharma – had strong double-digit organic sales growth.

Contract-Manufactured Products Segment
Net sales grew by 2.4% to $129.7 million. Organic sales growth was 2.1% with currency translation increasing sales growth by 30 basis points. Segment performance was led by sales of healthcare-related medical devices.

Financial Highlights (first nine months of 2021)
Operating cash flow was $423.2 million, an increase of 30.7%. Capital expenditures were $176.9 million, an increase of 51.6% over the same period last year. Free cash flow (operating cash flow minus capital expenditures) was $246.3 million, an increase of 18.9%.

During the first nine months of 2021, the Company repurchased 479,000 shares for $137.1 million at an average share price of $286.23 under its share repurchase program.

Full-Year 2021 Financial Guidance

Full-year 2021 net sales are expected to be in a range of $2.800 billion to $2.810 billion, compared to a prior guidance range of $2.760 billion to $2.785 billion.
Organic sales growth is expected to be 28%, compared to a prior range of 24% to 25%.
Net sales guidance includes an estimated full-year 2021 benefit of $55 million based on current foreign exchange rates. This updated guidance is a reduction of $25 million, compared to a prior estimated full-year benefit of $80 million.
Full-year 2021 adjusted-diluted EPS is expected to be in a range of $8.40 to $8.50, compared to a prior range of $8.05 to $8.20.
Full-year adjusted-diluted EPS guidance range includes an estimated benefit of approximately $0.19 based on current foreign currency exchange rates. This updated guidance is a reduction of $0.08, compared to a prior estimated benefit of $0.27.
The revised guidance includes a $0.35 EPS positive impact of tax benefits from stock-based compensation from the first nine months of 2021.
For the remainder of the year, our EPS guidance range assumes a tax rate of approximately 23% and does not include potential tax benefits from stock-based compensation. Any tax benefits associated with stock-based compensation beyond those recorded in the first nine months of 2021 would provide a positive adjustment to our full-year EPS guidance.
Third-Quarter 2021 Conference Call
The Company will host a conference call to discuss the results and business expectations at 9:00 a.m. Eastern Time today. To participate on the call please dial 877-930-8295 (U.S.) or 253-336-8738 (International). The conference ID is 5587559.

A live broadcast of the conference call will be available at the Company’s website, www.westpharma.com, in the "Investors" section. Management will refer to a slide presentation during the call, which will be made available on the day of the call. To view the presentation, select "Presentations" in the "Investors" section of the Company’s website.

An online archive of the broadcast will be available at the website three hours after the live call and will be available through Thursday, November 4, 2021, by dialing 855-859-2056 (U.S.) or 404-537-3406 (International) and entering conference ID 5587559.

On October 28, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, reported its third quarter 2021 financial results and provided a business update and outlook for the remainder of the year (Press release, argenx, OCT 28, 2021, View Source [SID1234592127]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"With three parallel regulatory reviews in our key priority territories of the U.S., Japan and the EU and the simultaneous build-out of our respective commercial organizations, we are well-positioned for the planned global launch of efgartigimod for the treatment of generalized myasthenia gravis. In order to optimize our strategy to make efgartigimod available to patients in need across the world, we are pursuing innovative partnerships, such as the strategic partnership with Zai Lab in China and the agreement with Medison in Israel which we are excited to announce today," said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

"As part of our commitment to becoming a fully-integrated, global immunology company, we are expanding our efgartigimod development plan to be in at least 15 indications by 2025 while also advancing a series of additional high-potential programs emerging from our Immunology Innovation Program. This includes a first-in-class C2 inhibitor, ARGX-117, which is on track to begin the first Phase 2 trial in multifocal motor neuropathy patients by the end of this year. Our growing commercial infrastructure along with our expanding pipeline ambitions provide considerable opportunity for argenx to deliver long-term, sustainable growth," concluded Mr. Van Hauwermeiren.

THIRD QUARTER 2021 AND RECENT BUSINESS UPDATE

Three global regulatory reviews ongoing for FcRn antagonist efgartigimod for the treatment of gMG

Biologics License Application (BLA) under review with U.S. Food and Drug Administration (FDA) with target action date of December 17, 2021 under Prescription Drug User Fee Act (PDUFA)
Marketing Authorization Application (J-MAA) under review with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) with anticipated approval in first quarter of 2022
MAA under review with European Medicines Agency (EMA) with anticipated approval in second half of 2022
Zai Lab on track with expected regulatory discussions with National Medical Products Administration (NMPA) for approval in China
Signed exclusive partnership agreement with Medison to commercialize efgartigimod for gMG in Israel; under agreement, Medison will also be responsible for seeking requisite regulatory approvals
Field teams onboard in U.S. and Japan, including 70 U.S. and 24 Japan sales representatives
New data from Phase 3 ADAPT trial of efgartigimod for the treatment of gMG presented during American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Annual Meeting. Additional data to be presented at upcoming Myasthenia Gravis Foundation of America (MGFA) Scientific Session. Highlights of the new data points include:

Among acetylcholine receptor-antibody positive (AChR-Ab+) patients, minimal symptom expression or MSE (MG-ADL of 0 or 1) was achieved by 59.1% (26/44) of MG-ADL responders following efgartigimod treatment during first cycle and 44.4% (16/36) during second cycle, compared to 36.8% (7/19) and 0% (0/11), respectively, for placebo patients
Among acetylcholine receptor-antibody negative (AChR-Ab-) patients, MSE was achieved in 31.6% (6/19) of MG-ADL responders following efgartigimod treatment during first cycle compared to 15.8% (3/19) for placebo patients
gMG and pemphigus patients who were vaccinated for influenza and pneumococcus during and prior to efgartigimod treatment in recent clinical trials showed that the ability to mount an immune antibody response was not impacted
Consistent and statistically significant disease score improvements were demonstrated following efgartigimod treatment across ADAPT patient subtypes, regardless of concomitant medication or affected muscle domain (bulbar, ocular, respiratory, limb/gross motor)
Initial patient-reported data from MyRealWorld MG real-world evidence study (N = 144) showed that despite taking an average of 2.3 treatments to control symptoms, people living with gMG experience substantial negative physical, mental, social, and emotional impacts of the disease
92% of responders agreed there is a significant need for new gMG treatments and are hopeful for ones with fewer side effects (96%)
In a separate argenx-sponsored patient burden survey (N = 150), 51% of gMG patients stopped working entirely as a consequence of their disease

Efgartigimod is currently being evaluated in five ongoing registrational trials across four indications, including ADAPT-SC (gMG), ADHERE (chronic inflammatory demyelinating polyneuropathy or CIDP), ADVANCE (IV) and ADVANCE-SC (primary immune thrombocytopenia or ITP), and ADDRESS (pemphigus)

Enrollment complete in ADAPT-SC and ADVANCE (IV); topline data for both trials expected in first half of 2022
Full Phase 2 trial results of efgartigimod for treatment of pemphigus published in British Journal of Dermatology
Protocol finalized for registrational trial of efgartigimod for treatment of idiopathic inflammatory myopathy (myositis), following FDA consultation; trial on track to start in first quarter of 2022
Registrational trial of efgartigimod for treatment of bullous pemphigoid on track to start by end of 2021
ARGX-117, a first-in-class C2-inhibitor, has potential to be the next pipeline-in-a-product opportunity across multiple severe autoimmune indications

Phase 1 data showed favorable safety profile and potential for infrequent dosing schedules
Phase 2 trial for treatment of multifocal motor neuropathy (MMN) on track to start by end of 2021
Wim Parys, M.D. to retire as Chief Medical Officer (CMO) on March 31, 2022 and transition to member of Research and Development Committee of argenx Board of Directors

Succession plans underway for Luc Truyen, M.D., Ph.D., Vice President, Research & Development Operations at argenx, to assume CMO role in April 2022

DETAILS OF THE FINANCIAL RESULTS

As of January 1, 2021, the Company changed its functional and presentation currency from euro to U.S. dollar, which results in reporting financial highlights in U.S. dollar as compared to euro in prior periods. Historical financials have been converted at the average exchange rate of the related period.

Cash, cash equivalents and current financial assets totaled $2,534.0 million as of September 30, 2021, compared to $1,996.5 million on December 31, 2020. The increase in cash and cash equivalents and current financial assets resulted primarily from (i) the closing of a global offering, which resulted in the receipt of $1,092.1 million in net proceeds in February 2021, (ii) the net receipt of a $73.1 million non-creditable, non-refundable development cost-sharing payment received from Zai Lab as part of the strategic collaboration for efgartigimod in Greater China, (iii) the payment of $98.0 million related to the purchase of the priority review voucher from Bayer HealthCare Pharmaceuticals, and other net cash flows used in operating activities.

Total operating income increased by $446.9 million for the nine months ended September 30, 2021 to $494.6 million, compared to $47.7 million for the nine months ended September 30, 2020. The increase was primarily due to the recognition of the transaction price as a consequence of the termination of the collaboration agreement with Janssen, resulting in the recognition of $315.1 million and the closing of the strategic collaboration for efgartigimod with Zai Lab, resulting in the recognition of $151.9 million in collaboration revenue.

Research and development expenses increased by $136.9 million for the nine months ended September 30, 2021 to $413.3 million, compared to $276.4 million for the nine months ended September 30, 2020. The increase in the first nine months of 2021 resulted primarily from higher external research and development expenses, mainly related to the efgartigimod program in various indications and other clinical and preclinical programs. Furthermore, the research and development personnel expenses increased due to a planned increase in headcount and the increased costs of the share-based payment compensation plans related to the grant of stock options.

Selling, general and administrative expenses totaled $210.2 million for the nine months ended September 30, 2021, compared to $113.2 million for the nine months ended September 30, 2020. The increase resulted primarily from higher personnel expenses, including the costs of the share-based payment compensation plans related to the grant of stock options, and consulting fees linked to the preparation of a possible future commercialization of argenx’s lead product candidate efgartigimod.

The change in fair value on non-current financial assets amounted to $11.2 million for the nine months ended September 30, 2021, which is the result of the closing of a Series B financing round of AgomAb Therapeutics, for which argenx maintains a profit share in exchange for granting the license for the use of HGF-mimetic antibodies from the SIMPLE AntibodyTM platform.

Exchange losses totaled $36.0 million for the nine months ended September 30, 2021, compared to $65.3 million for the nine months ended September 30, 2020. As a result of the change in the Company’s functional and presentation currency, the exchange losses for the nine months ended September 30, 2021 are reflecting the unfavorable change in euro/U.S. dollar exchange rate, mainly attributable to unrealized exchange rate losses on cash, cash equivalents and current financial asset position in euro.

FINANCIAL GUIDANCE

Based on current plans to fund anticipated operating expenses and capital expenditures, argenx continues to expect its full-year 2021 cash burn to approximately double from 2020. The increased spend has supported the Company’s transition to an integrated immunology company, including the build-out of global commercial infrastructure and drug product inventory ahead of the expected launch of efgartigimod in gMG in the U.S, the advancement of its clinical-stage pipeline, including expected registrational trials of efgartigimod in six indications, and the continued investment in its Immunology Innovation Program. As argenx further expands its commercial infrastructure and differentiated pipeline of assets, it is expected that the spend associated with these activities will continue to increase.

EXPECTED 2022 FINANCIAL CALENDAR

March 3, 2022: FY 2021 financial results and business update
May 12, 2022: Q1 2022 financial results and business update
July 28, 2022: HY 2022 financial results and business update
October 27, 2022: Q3 2022 financial results and business update
CONFERENCE CALL DETAILS
The third quarter 2021 financial results and business update will be discussed during a conference call and webcast presentation today at 2:30 pm CEST/8:30 am ET. A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website.