On May 9, 2024 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported its first quarter 2024 results and provided a business update (Press release, argenx, MAY 8, 2024, View Source [SID1234642902]).

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"The team at argenx has made significant progress executing across the ambitious plan we set out at the beginning of the year," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "We are driven by our commitment to provide patients with the broadest gMG product offering that consistently delivers on safety and efficacy. VYVGART SC played a key role in our growth over the quarter, expanding the breadth of our prescriber base and reaching new patients. The relationships we have built and key market learnings in gMG position us for success as we scale the organization and prepare for CIDP. After generating the data required for filing, we are also excited to advance the development of our pre-filled syringe, which should further enhance the patient experience."

"The clinical opportunity ahead is expansive – we are preparing for registrational trials across multiple programs including empasiprubart in MMN and efgartigimod in Sjogren’s disease, in addition to those already underway in TED and seronegative gMG. We look forward to deepening our understanding of FcRn with additional Phase 2 data points expected this year, while rapidly working to deliver on our promise of innovation by bringing the next wave of molecules to the clinic."

FIRST QUARTER 2024 AND RECENT BUSINESS UPDATE

Reaching More Patients with VYVGART

VYVGART (efgartigimod alfa-fcab) is a first-in-class antibody fragment targeting the neonatal Fc receptor (FcRn), and is now the first FcRn antagonist approved in two indications. VYVGART is approved in more than 30 countries globally for the treatment of generalized myasthenia gravis (gMG) and is approved in Japan for the treatment of primary immune thrombocytopenia (ITP). VYVGART subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) is approved in the U.S. (as VYVGART Hytrulo), Japan (as VYVDURA) and Europe, making VYVGART the only gMG treatment available as both an IV and simple SC injection.

Generated global net product sales (inclusive of both VYVGART and VYVGART SC) of $398 million in the first quarter of 2024
VYVGART approved in Japan for treatment of ITP on March 26, 2024, marking first global approval for ITP
Additional VYVGART and VYVGART SC regulatory decisions on approval expected for gMG in 2024, including VYVGART in Switzerland, Australia, Saudi Arabia and South Korea, and VYVGART SC in China through Zai Lab
Multiple VYVGART SC regulatory submissions under review or planned for chronic inflammatory demyelinating polyneuropathy (CIDP), including:
FDA review of Supplemental Biologics License Application (sBLA) ongoing with Prescription Drug User Fee Act (PDUFA) target action date of June 21, 2024
Regulatory submissions completed in China and Japan
Regulatory submissions expected in Europe and Canada by end of 2024
Registrational study of VYVGART in seronegative gMG patients ongoing with aim to expand label into broader MG populations
FDA submission for VYVGART SC prefilled syringe for gMG and CIDP expected in second quarter of 2024, following positive data outcomes from bioequivalence and human factor studies
Advancing Current Pipeline

argenx continues to demonstrate breadth and depth within its immunology pipeline and is advancing multiple pipeline-in-a-product candidates. argenx is solidifying its leadership in FcRn biology and expects that efgartigimod will be approved or under evaluation in at least 15 indications by 2025. argenx is also advancing its earlier stage pipeline programs, including empasiprubart (C2 inhibitor) with Phase 2 studies ongoing in multifocal motor neuropathy (MMN), delayed graft function (DGF) and dermatomyositis (DM). In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

Decision announced to advance development of efgartigimod in primary Sjogren’s disease (SjD) to Phase 3 following analysis of topline data from Phase 2 RHO study
Topline data from Phase 2 ALPHA study of efgartigimod in post-COVID-19 postural orthostatic tachycardia syndrome (PC-POTS) expected in second quarter of 2024
Topline data from seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM)), anti-synthetase syndrome (ASyS), and DM expected in second half of 2024
Update on BALLAD study development plan evaluating efgartigimod in bullous pemphigoid (BP) expected by end of 2024
Registrational studies ongoing of efgartigimod in thyroid eye disease (TED)
Decision made to discontinue planned development of efgartigimod in ANCA-associated vasculitis (AAV) following risk assessment of all ongoing studies based on learnings from ADDRESS (pemphigus) and ADVANCE SC (ITP) studies
Proof-of-concept studies ongoing with efgartigimod in membranous nephropathy (MN) and lupus nephritis (LN) with studies expected to start this year in antibody mediated rejection (AMR) and newly nominated indication, systemic sclerosis (SSc)
Full Phase 2 topline data (cohorts 1 and 2) from ARDA study of empasiprubart in MMN expected in 2024; cohort 2 ongoing to determine dose response ahead of Phase 3 study start
Phase 1b/2a trials of ARGX-119 to assess early signal detection in patients with CMS and ALS expected to start in 2024
Leveraging Repeatable Innovation Playbook to Drive Long-Term Pipeline Growth

argenx continues to invest in its discovery engine, the Immunology Innovation Program (IIP), to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX-213 targeting FcRn and further solidifying argenx’s leadership in this new class of medicine; ARGX-121 and ARGX-220, which are first-in-class targets broadening argenx’s focus across the immune system; and ARGX-109, targeting IL-6, which plays an important role in inflammation. Investigational new drug (IND) applications for each program are expected to be filed by end of 2025.

Appointment of Brian L. Kotzin, MD as Non-executive Director to Board of Directors

Dr. Brian Kotzin has been appointed as non-executive director to the Board of Directors and Chair of the Research & Development Committee for a term of four years. He is currently a consultant for companies developing therapeutics for autoimmune and inflammatory diseases. His prior roles include Chief Medical Officer for Nektar Therapeutics and Vice President of Global Clinical Development, Head of the Inflammation Therapeutic Area and Vice President and Head of Medical Sciences at Amgen.

FIRST QUARTER 2024 FINANCIAL RESULTS

argenx SE

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF PROFIT OR LOSS

Earnings 2023

DETAILS OF THE FINANCIAL RESULTS

Total operating income for the three months ended March 31, 2024, was $413 million compared to $230 million for the same period in 2023, and consists of:

Product net sales of VYVGART and VYVGART SC for the three months ended March 31, 2024, were $398 million compared to $218 million for the same period in 2023.
Collaboration revenue for the three months ended March 31, 2024, was $3 million compared to $1 million for the same period in 2023. Collaboration revenue for the three months ended March 31, 2024, includes $2 million in royalty revenue from VYVGART sales in China.
Other operating income for the three months ended March 31, 2024, was $12 million compared to $11 million for the same period in 2023. The other operating income for the three months ended March 31, 2024 and 2023, primarily relates to research and development tax incentives.
Total operating expenses for the three months ended March 31, 2024, were $506 million compared to $334 million for the same period in 2023, and mainly consists of:

Cost of sales for the three months ended March 31, 2024, was $43 million compared to $18 million for the same period in 2023. The cost of sales was recognized with respect to the sale of VYVGART and VYVGART SC.
Research and development expenses for the three months ended March 31, 2024, were $225 million compared to $166 million for the same period in 2023. The research and development expenses mainly relate to external research and development expenses and personnel expenses incurred in the clinical development of efgartigimod in various indications and the expansion of other clinical and preclinical pipeline candidates.
Selling, general and administrative expenses for the three months ended March 31, 2024, were $236 million compared to $149 million for the same period in 2023. The selling, general and administrative expenses mainly relate to professional and marketing fees linked to the commercialization of VYVGART and VYVGART SC, and personnel expenses.
Financial income for the three months ended March 31, 2024, was $39 million compared to $17 million for the same period in 2023. The increase in financial income is mainly due to an increase in interest income coming from an increase of cash, cash equivalents and current financial assets as a result of the July 2023 financing round.

Exchange losses for the three months ended March 31, 2024, were $19 million compared to $11 million of exchange gains for the same period in 2023. Exchange gains/losses are mainly attributable to unrealized exchange rate gains or losses on the cash, cash equivalents and current financial assets denominated in Euro.

Income tax for the three months ended March 31, 2024, was $13 million of income tax benefit compared to $47 million of income tax benefit for the same period in 2023. Income tax benefit for the three months ended March 31, 2024, consists of $6 million of current income tax expense and $19 million of deferred tax benefit, compared to $11 million of current income tax expense and $58 million of deferred tax benefit for the comparable prior period.

Net loss for the three months ended March 31, 2024, was $62 million compared to $29 million for the same period in 2023. On a per weighted average share basis, the net loss was $1.04 and $0.52 for the three months ended March 31, 2024 and 2023, respectively.

Cash, cash equivalents and current financial assets totalled $3.1 billion as of March 31, 2024, compared to $3.2 billion as of December 31, 2023. The decrease in cash and cash equivalents and current financial assets result from net cash flows used in operating activities.

FINANCIAL GUIDANCE

Based on its current operating plans, argenx expects its combined Research and development and Selling, general and administrative expenses in 2024 to be less than $2 billion. argenx expects to utilize up to $500 million of net cash in 2024 on these anticipated operating expenses as well as working capital and capital expenditures.

EXPECTED 2024 FINANCIAL CALENDAR

July 25, 2024: Q2 2024 financial results and business update
October 31, 2024: Q3 2024 financial results and business update
CONFERENCE CALL DETAILS

The first quarter 2024 financial results and business update will be discussed during a conference call and webcast presentation today at 2:30 PM CET/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.

Dial-in numbers:

Please dial in 15 minutes prior to the live call.

Belgium 32 800 50 201
France 33 800 943355
Netherlands 31 20 795 1090
United Kingdom 44 800 358 0970
United States 1 800 715 9871
Japan 81 3 4578 9081
Switzerland 41 43 210 11 32

On May 8, 2024 Celaid Therapeutics reported that it has raised an additional 460 million yen in its Series A extension round (Press release, Celaid Therapeutics, MAY 8, 2024, View Source [SID1234649811]). This round was led by The University of Tokyo Edge Capital Partners (UTEC) and OSAKA University Venture Capital (OUVC) with participation from Techno Science and Joyo Capital Partners. This brings Celaid’s total raise to approximately 1.2 billion yen which includes existing Series A funding of 500 million yen and the recent 200 million yen from the NEDO Deep Tech Startup grant.

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Celaid will apply the funding to advancing CMC process development in its lead pipeline asset for non-malignant hematologic disease in preparation for IND submission in the US. In parallel, Celaid will continue collaborations with partners exploring applications of its technology to HSC gene therapies and allogeneic immune cell therapies which include those utilizing iPSC-derived products.

"We were able to secure the necessary funds for future research and development in our Series A funding round thanks to new participating investors’ confidence in Celaid’s HSC-based platform and pipeline," said Nobuyuki Arakawa, President and Chief Executive Officer of Celaid.
"We are committed to delivering our unique HSC products to patients around the world as soon as possible."

On May 8, 2024 Xspray reported its financial report for the first quarter 2024 (Press release, Xspray, MAY 8, 2024, View Source [SID1234649581]).

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On May 8, 2024 Pacylex Pharmaceuticals Inc. (Pacylex) is a clinical-stage pharmaceutical company developing N-myristoyltransferase (NMT) inhibitors as targeted therapies for the treatment of hematologic cancers and solid tumors, reported the publication of a new study in the Journal of Translational Medicine, "Multiomics analysis identifies oxidative phosphorylation as a cancer vulnerability arising from myristoylation inhibition," describing how NMT inhibition by zelenirstat affects cancer cells, with broad implications for multiple liquid and solid tumor cancers (Press release, Pacylex Pharmaceuticals, MAY 8, 2024, View Source [SID1234645050]).

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In order to understand how zelenirstat kills cancer cells, Pacylex researchers used mass spectrometry to identify the proteins most affected by zelenirstat treatment. They discovered that NDUFAF4, a protein essential for mitochondrial energy production from oxygen (oxidative phosphorylation), was the most severely affected of all. Inhibiting myristoylation of NDUFAF4 shut down oxidative phosphorylation in cancer cells. The study also produced an in vitro myristoylation inhibition sensitivity signature comprising 54 genes (MISS-54) enriched in hematologic cancers as well as testis, brain, lung, ovary, and colon cancers.

"The ability of a myristoylation inhibitor to limit oxidative phosphorylation and also inhibit cancer cell signaling appears to explain how zelenirstat selectively kills cancer cells, especially those with high MISS-54 scores," said Dr. Luc Berthiaume, CSO of Pacylex Pharmaceuticals. "Since normal cells readily use other non-oxidative metabolic processes to produce energy, while many types of cancer cells including relapse causing cancer stem cells cannot, zelenristat could lead to more tolerable and durable cancer treatments."

"This new mechanism of action may explain why zelenirstat treatment shows activity across multiple patients with unrelated cancers. The phase I population was an unselected group that had no remaining standard therapy options, yet zelenirstat therapy produced prolonged stable disease or better in 57% of the patients receiving the recommended Phase 2 dose," said Dr. John Mackey, CMO of Pacylex Pharmaceuticals.

Zelenirstat is currently being dosed in patients in two Phase 2a studies, in refractory and relapsed B-cell non-Hodgkin’s lymphoma and in advanced refractory colorectal cancer patients, at 4 clinical sites in Canada.

On May 8, 2024, Coherus BioSciences, Inc., a Delaware corporation (the "Company"), reported to have entered into a senior secured term loan facility of up to $38.7 million (the "Term Loan"), all to be funded on May 8, 2024 (the "Effective Date"), with Ankura Trust Company, LLC, as administrative agent (in such capacity, the "Agent"), and the lenders signatory thereto (collectively, the "Lenders") (Filing, Coherus Biosciences, MAY 8, 2024, View Source [SID1234643586]). The proceeds of the Term Loan will be used by the Company to help repay in full the existing outstanding indebtedness owed by the Company to BioPharma Credit, PLC, BPCR Limited Partnership, and Biopharma Credit Investments V (Master) LP, acting by its general partner, BioPharma Credit Investments V GP LLC pursuant to the senior secured term loan facility that was entered into January 5, 2022 (as amended on April 7, 2022, February 6, 2023 and February 5, 2024, the "Prior Loan Agreement").

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The Term Loan is governed by a loan agreement, dated as of the Effective Date, by and among the Company, the Agent and the Lenders (the "Loan Agreement"). The Term Loan will mature on May 8, 2029. The amounts borrowed under the Term Loan accrue interest at a per annum rate equal to 8.00% per annum, plus a three month SOFR rate (the "Interest Rate"). The Term Loan provides for interest-only payments on a quarterly basis until maturity. The Company may prepay the Term Loan in full or in part provided that the Company (i) provides at least three (3) business days’ prior written notice to the Agent, (ii) pays on the date of such prepayment (A) all outstanding principal to be prepaid plus accrued and unpaid interest, (B) a prepayment fee of (x) 10.00% of the Term Loans so prepaid if paid on or after the first anniversary of the Effective Date and before the second anniversary of the Effective Date; (y) 5.00% of the Term Loans so prepaid if paid after the second anniversary of the Effective Date and on or before the third anniversary of the Effective Date; and (z) 0.00% of the Term Loans so prepaid if paid after the third anniversary of the Effective Date and (C) all other sums, if any, that shall become due and payable under the Loan Agreement, including interest at the default rate with respect to any past due amounts. Amounts outstanding during an event of default shall accrue interest at an additional rate of 4.00% per annum, which interest shall be payable on demand in cash.

The Term Loan is secured by a lien on substantially all of the assets of the Company, including intellectual property, subject to customary exclusions and exceptions. The Loan Agreement contains customary representations and warranties, covenants and events of default, including a financial covenant commencing on the Effective Date, which requires the Company to maintain certain levels of cash and cash equivalents. The Loan Agreement also contains other customary provisions, such as expense reimbursement, as well as indemnification rights for the benefit of the Agent and the Lenders.

The foregoing description of the material terms of the Loan Agreement is qualified in its entirety by the full terms and conditions of the Loan Agreement, which will be filed with the Securities and Exchange Commission (the "SEC") as an exhibit to the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2024.

In connection with the entry into the Loan Agreement, on the Effective Date, the Company repaid in full all outstanding indebtedness and terminated all commitments under the Prior Loan Agreement, the material terms of which have been disclosed previously. The aggregate principal amount of the loan outstanding under the Prior Loan Agreement was $75 million at the time of repayment. The Company did not incur any penalties, but did incur a prepayment fee and paid a make-whole amount, as a result of the foregoing.

Revenue Participation Right Purchase and Sale Agreement

On May 8, 2024, the Company entered into a revenue participation right purchase and sale agreement (the "Revenue Purchase and Sale Agreement") with Coduet Royalty Holdings, LLC, as administrative agent and each buyer named in an annex thereto (collectively, the "Purchaser"). Under the terms of the Revenue Purchase and Sale Agreement, in exchange for the Purchaser’s payment to the Company of a purchase price of $37.5 million, in the

aggregate subject to certain conditions at closing (the "Purchase Price"), the Company has agreed to sell to the Purchaser its right to receive payment in full of a mid-single digit percentage of U.S. net sales of UDENYCA and LOQTORZI (the "Revenue Payment") for each calendar quarter commencing on the effective date of the Revenue Purchase and Sale Agreement. The Purchaser’s right to receive the Revenue Payment terminates and the Company no longer has the obligation to pay Purchaser Revenue Payments once the Purchaser receives the amount equal to 2.25 times the Purchase Price. The Company may also buy-out the Purchaser’s rights to receive the Revenue Payments by paying Purchaser such multiple on the Purchaser Price.

The Revenue Purchase and Sale Agreement contains various representations and warranties, including with respect to organization, authorization, and certain other matters, certain covenants with respect to payment, reporting, intellectual property, in-licenses, out-licenses, and certain other actions, indemnification obligations and other provisions customary for transactions of this nature.

The foregoing description of the material terms of the Revenue Purchase and Sale Agreement is qualified in its entirety by the full terms and conditions of the Revenue Purchase and Sale Agreement, which will be filed with the SEC as an exhibit to the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2024.

Item 1.02 Termination of a Material Definitive Agreement

The information set forth above in Item 1.01 of this Current Report on Form 8-K regarding the repayment and termination of the Prior Loan Agreement is incorporated into this Item 1.02 by reference.