On July 25, 2024 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported its half year 2024 results and provided a second quarter business update (Press release, argenx, JUL 25, 2024, View Source [SID1234645066]).

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"We were excited to unveil our ambition for the future of argenx – Vision 2030 – last week, outlining our plan to develop and deliver continued and sustainable innovation for patients," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "We are already delivering on this promise with impressive commercial execution throughout the first half of the year, expanding our patient reach in MG, and launching in CIDP with our broad FDA label. Our development pipeline is stronger than ever, driven by our unique innovation engine. And we are well-positioned to capture the sizeable growth opportunity before us as we seek to reach earlier-line MG patients over the next 12-18 months with potential label expansions and a pre-filled syringe."

Vision 2030

During its R&D Day on July 16, 2024, argenx unveiled its ‘Vision 2030’ as part of its long-term commitment to transform the treatment of autoimmune diseases by strengthening its leadership in neonatal Fc receptor (FcRn) biology, investing in its continuous pipeline of differentiated antibody candidates, and scaling in a disciplined way to ensure innovation remains core to the argenx mission. ‘Vision 2030’ includes the following goals:

50,000 patients globally on treatment with an argenx medicine
10 labeled indications across all approved assets, including VYVGART and potentially empasiprubart and ARGX-119
Five new molecules in Phase 3 development indicating ongoing investment in internal discovery engine, the Immunology Innovation Program
Reaching 50,000 Patients Globally

VYVGART (efgartigimod alfa-fcab) is a first-in-class antibody fragment targeting FcRn and is now approved for both intravenous use and subcutaneous injection (SC) (efgartigimod alfa and hyaluronidase-qvfc) in three indications, including generalized myasthenia gravis (gMG) globally, primary immune thrombocytopenia (ITP) in Japan, and chronic inflammatory demyelinating polyneuropathy (CIDP) in the U.S.

Generated global net product sales (inclusive of both VYVGART and VYVGART SC) of $478 million in second quarter of 2024
National Medical Products Administration (NMPA) approved VYVGART SC for treatment of gMG in China through Zai Lab on July 16, 2024
Additional VYVGART regulatory decisions on approval expected for gMG in 2024, including in Switzerland, Australia, and Saudi Arabia
Launched VYVGART Hytrulo in CIDP in U.S. with first patients injected in July
Multiple VYVGART SC regulatory submissions under review or planned for CIDP, including:
Regulatory submissions completed in China, Japan, and Europe with decisions on approval expected in 2025
Regulatory submission filing in Canada by end of 2024
Announced plan to evaluate VYVGART in ocular MG with registrational study (ADAPT OCULUS) to start by end of year; OCULUS to support label-expansion strategy into broader MG populations along with ongoing ADAPT SERON study in seronegative MG
Regulatory submission filed and under review for VYVGART SC pre-filled syringe (PFS) for gMG and CIDP
Advancing Pipeline to Achieve 10 Labeled Indications by 2030

argenx continues to demonstrate breadth and depth within its immunology pipeline, advancing multiple pipeline-in-a-product candidates. argenx is solidifying its leadership in FcRn biology, with efgartigimod currently in development in 15 indications. argenx is also advancing its first-in-class C2 inhibitor, empasiprubart, which is being evaluated in multifocal motor neuropathy (MMN), delayed graft function (DGF), dermatomyositis (DM), and CIDP. In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

Registrational studies ongoing of efgartigimod in thyroid eye disease (TED)
Advancing development of efgartigimod in primary Sjogren’s disease (SjD) with Phase 3 study to begin by end of 2024
Following alignment meeting with FDA, confirmatory study of VYVGART (IV) in primary ITP to start by end of 2024 to enable registration in U.S.
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 fourth quarter of 2024
Update on BALLAD study development plan evaluating efgartigimod in bullous pemphigoid (BP) expected by end of 2024
Proof-of-concept studies ongoing with efgartigimod in membranous nephropathy (MN) and lupus nephritis (LN) with studies expected to initiate this year in antibody mediated rejection (AMR) and systemic sclerosis (SSc)
Phase 3 study of empasiprubart for MMN to initiate in fourth quarter of 2024
CIDP nominated as fourth empasiprubart indication, recognizing opportunity to bring multiple innovative treatments to patients
Phase 1b/2a studies of ARGX-119 to assess early signal detection in patients with CMS and ALS to start by end of 2024
Investing in Immunology Innovation Program to Support Five New Molecules in Phase 3 by 2030

argenx continues to invest in its 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, a first-in-class molecule targeting IgA; ARGX-109, targeting IL-6, which plays an important role in inflammation, and ARGX-220, a first-in-class sweeping antibody for which the target has not yet been disclosed.

Phase 1 studies of ARGX-213 and ARGX-121 expected to start in second half of 2025
Investigational new drug (IND) applications for ARGX-220 and ARGX-109 on track to be filed by end of 2025
SECOND QUARTER 2024 FINANCIAL RESULTS

argenx SE

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF PROFIT OR LOSS

Argenx earnings 2024

DETAILS OF THE FINANCIAL RESULTS

Total operating income for the three and six months ended June 30, 2024 was $489 million and $902 million compared to $281 million and $511 million for the same periods in 2023, and mainly consists of:

Product net sales of VYVGART and VYVGART SC for the three and six months ended June 30, 2024 were $478 million and $876 million compared to $269 million and $487 million for the same periods in 2023.
Other operating income for the three and six months ended June 30, 2024 was $12 million and $23 million compared to $10 million and $21 million for the same periods in 2023. The other operating income for the three and six months ended June 30, 2024 and 2023, primarily relates to research and development tax incentives.
Total operating expenses for the three and six months ended June 30, 2024 were $535 million and $1,041 million compared to $383 million and $717 million for the same periods in 2023, and mainly consists of:

Cost of sales for the three and six months ended June 30, 2024 was $52 million and $96 million compared to $24 million and $42 million for the same periods 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 and six months ended June 30, 2024 were $225 million and $450 million compared to $196 million and $361 million for the same periods 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 and six months ended June 30, 2024 were $256 million and $492 million compared to $162 million and $311 million for the same periods 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 and six months ended June 30, 2024 was $39 million and $78 million compared to $20 million and $37 million for the same periods 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 and six months ended June 30, 2024 were $8 million and $27 million compared to $2 million exchange losses and $9 million of exchange gains for the same periods 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 and six months ended June 30, 2024 was $44 million and $57 million of income tax benefit, respectively, compared to $11 million of income tax expense and $37 million of income tax benefit for the same periods in 2023.

Net Result for the three and six months ended June 30, 2024 was $29 million profit and $33 million loss compared to $94 million and $123 million loss for the same periods in 2023. On a per weighted average share basis, the basic profit was $0.49 and diluted profit was $0.45 for the three months ended June 30, 2024, compared to a basic and diluted loss of $1.69 for the same period in 2023. On a per weighted average share basis, the basic and diluted loss was $0.55 for the six months ended June 30, 2024, compared to a basic and diluted loss of $2.21 for the same period in 2023.

Cash, cash equivalents and current financial assets totalled $3.1 billion as of June 30, 2024, compared to $3.2 billion as of December 31, 2023. The decrease in cash and cash equivalents and current financial assets result from a 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 updated its cash burn guidance and now expects to utilize less than $500 million of net cash1 in 2024 on anticipated operating expenses as well as working capital and capital expenditures.

EXPECTED 2024 FINANCIAL CALENDAR

October 31, 2024: 3Q 2024 financial results and business update
February 27, 2025: Full-year 2024 financial results and 4Q 2024 business update
CONFERENCE CALL DETAILS

The half-year 2024 financial results and second quarter 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 and replay may be accessed on the Investors section of the argenx website at argenx.com/investors.

On July 24, 2024 GeneCentric Therapeutics, a company making precision medicine more precise through RNA-based diagnostics, reported that the American Medical Association (AMA) has granted a PLA code for the PurISTSM pancreatic cancer test (Press release, GeneCentric Therapeutics, JUL 24, 2024, View Source [SID1234645075]). PurIST is commercially available under a collaboration agreement with Tempus AI, Inc. (NASDAQ: TEM) and run on its xR platform. The PurIST PLA code is the first CPT code created to describe an algorithm-only analysis from previously sequenced, LDT, transcriptomic (RNA) data and represents a meaningful step toward reimbursement for AI-enabled algorithms.

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"Receipt of a PurIST-specific PLA test code further supports the significance of GeneCentric’s pipeline of next-generation AI-derived tests," said Michael Milburn, PhD, President and CEO of GeneCentric Therapeutics. "This is a key step toward reimbursement and successful commercialization for PurIST, and we’re excited about the importance of this test in guiding first-line treatments for pancreatic cancer patients."

Pancreatic cancer has one of the highest mortality rates among all major cancers, and there has been limited availability of validated diagnostic tests or biomarkers to guide first-line treatment selection. The PurIST test classifies the tumors of patients with unresectable stage III or stage IV pancreatic ductal adenocarcinoma (PDAC) as either a basal or classical subtype and can help guide first-line therapy.

About the PurIST Test

PurIST identifies the molecular subtype of patients with unresectable stage III or stage IV PDAC and classifies patients with PDAC into either a basal or classical subtype and may help inform first-line therapy management. Tempus and GeneCentric recently completed a new clinical validation study demonstrating that PurIST can be used to help predict overall survival of classical patients between standard-of-care first-line therapies, FOLFIRINOX and gemcitabine nab-paclitaxel. The PurIST test is available for ordering as part of the on-line Tempus HUB or via paper requisition forms for orders in the United States. Providers and patients can learn more about the PurIST test on the Tempus website.

About Pancreatic Cancer

Pancreatic cancer is the 10th most commonly diagnosed cancer in the U.S. and the 12th most common cancer worldwide. The National Cancer Institute estimates there will be over 64,440 new cases and more than 51,750 deaths in the U.S. alone in 2024, making it the third-leading cause of cancer-related deaths in the U.S. with one of the highest mortality rates of all major cancers. More than 90% of pancreatic cancers are pancreatic ductal adenocarcinoma (PDAC), and the 5-year overall survival for resectable/borderline-resectable and non-resectable PDAC is approximately 20% and 1-3%, respectively. The three main treatments for PDAC are surgical resection (if diagnosed early), radiation therapy and chemotherapy. For resectable tumors, surgical resection is provided with curative intent, however, for non-resectable tumors, chemotherapy such as FOLFIRINOX or gemcitabine with nab-paclitaxel is often used.

On July 24, 2024 Dren Bio, Inc. ("Dren Bio" or the "Company"), a privately held, clinical-stage biopharmaceutical company developing antibody therapeutics for cancer, autoimmune, and other serious diseases, reported that it has entered into a strategic collaboration with Novartis Pharma AG, a subsidiary of Novartis AG (NYSE: NVS) (Press release, Dren Bio, JUL 24, 2024, View Source [SID1234645074]). The collaboration will focus on the discovery and development of therapeutic bispecific antibodies for cancer using Dren Bio’s proprietary Targeted Myeloid Engager and Phagocytosis Platform.

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"Our agreement with Dren Bio is a promising opportunity to discover novel bispecific antibody therapies for cancer, building on our longstanding expertise in immuno-oncology science at Novartis," said Shiva Malek, Ph.D., Global Head of Oncology for Biomedical Research at Novartis. "We’re excited to collaborate to bring forward new therapeutic options for patients living with cancer, complementing our strategic efforts across a wide range of modalities, including targeted therapies, biologics, radioligand therapies and CAR-Ts."

"We are thrilled to establish this new collaboration with Novartis, a global leader in oncology," said Nenad Tomasevic, Ph.D., Chief Executive Officer of Dren Bio. "Combining the proven capabilities of Novartis in oncology drug development with Dren Bio’s novel platform could enable the advancement of important new therapies for patients."

Amit Mehta, Ph.D., Chief Operating Officer and Chief Business Officer of Dren Bio, added, "Dren Bio’s Targeted Myeloid Engager and Phagocytosis Platform is designed to deplete various disease-causing agents and has led to a rich and diverse pipeline. This collaboration will benefit from Novartis’ impressive track record of developing novel medicines and help further expand the reach of our platform."

Under the terms of the agreement, Dren Bio will receive a total upfront consideration of $150 million from Novartis, which includes a $25 million equity investment in the Company. Dren Bio is also eligible to receive up to $2.85 billion in additional cash payments upon achieving certain preclinical, clinical, regulatory, and commercial milestones, as well as tiered royalties on future net sales of any commercialized products resulting from the collaboration. Dren Bio and Novartis will collaborate to advance selected targeted myeloid engager programs in oncology through clinical candidate selection, at which point Novartis will assume full responsibility for all remaining development, manufacturing, regulatory, and commercialization activities.

The agreement is subject to customary closing conditions including regulatory clearance.

On July 24, 2024 OS Therapies, Inc. (NYSE-A: OSTX), a clinical-stage oncology-focused immunotherapy company developing cancer vaccines and antibody drug conjugate (ADC) therapeutic candidates, reported positive data from a Phase 1 clinical trial of OST-HER2 in patients with HER2-expressing solid tumors in breast cancer and other cancers (Press release, OS Therapies, JUL 24, 2024, View Source [SID1234645073]). Additionally, the Company announced positive preclinical efficacy data for OST-HER2 in multiple models of breast cancer.

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The FDA has granted Rare Pediatric Disease Designation (RPDD), Orphan Drug Designation (ODD), and Fast Track Designation (FTD) for OST-HER2 in Osteosarcoma. OST-HER2, a biologic therapeutic candidate, is a Lm (Listeria monocytogenes) vector-based off-the-shelf immunotherapeutic vaccine designed to prevent metastasis, delay recurrence, and increase overall survival in patients with osteosarcoma and other solid tumors. The proposed OST-HER2 mechanism of action is based on innate and adaptive immune stimulating responses activated by the Lm vector. This treatment generates T cells that can eliminate or slow potential micrometastases that can grow into recurrent osteosarcoma and other solid tumors, including breast cancer. T cell responses home in on HER2 expressed by the tumor and then kill the cell, releasing additional tumor targets. There are currently no approved adjuvant treatments for recurrent osteosarcoma in the United States.

A total of twelve (12) patients with a history of HER2 positive cancer were enrolled in the Phase 1b clinical study, comprised of ten (10) patients with breast cancer, one (1) patient with esophageal cancer and one (1) patient with GE junction cancer. OST-HER2 was determined to be safe and well tolerated in patients with HER2-expressing solid tumors, including at the dose currently being used in the Company’s ongoing Phase 2b clinical trial in recurrent, resected osteosarcoma (AOST-2121, NCT04974008). The Company previously announced positive AOST-2121 interim data and is now in active discussions with the US FDA regarding a Breakthrough Therapy (BTD) designation application submitted for OST-HER2 based on the interim results. The Company is focused on approval for OST-HER2 in osteosarcoma, and thereafter plans to expand development into breast cancer and other HER2 expressing cancers such as esophageal cancer and colorectal cancer.

In addition, the Company announced positive data from multiple preclinical models of breast cancer. The key data for OST-HER2 include:

78% reduction in tumor size (3mm for OST-HER2 treated vs. 14mm for control arm) in FVB/N HER2 transgenic mouse model of breast cancer treatment at day 75
33% prevention of breast cancer in OST-HER2 treated mice vs. 0% prevention of breast cancer in FVB/N HER2 transgenic model of breast cancer prevention at week 50
20% reduction of tumor size for OST-HER2 plus HER2-targeted antibody vs. HER2-targeted antibody alone Tg tumor regression model of breast cancer at day 42
65% reduction cellular concentration of metastatic cells for OST-HER2-treated mice compared with controls in brain metastasis model of primary breast cancer

On July 24, 2024 enGene Holdings Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage genetic medicines company whose non-viral, intravesical lead product candidate, EG-70, is in a pivotal study for BCG-unresponsive high-risk non-muscle invasive bladder cancer (NMIBC), reported the grant of an inducement equity award to Ron Cooper, the Company’s newly-appointed Chief Executive Officer (Press release, enGene, JUL 24, 2024, View Source [SID1234645072]).

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The inducement award consists of a non-qualified stock option to purchase 1,250,000 of the Company’s common shares. The option has an exercise price of $8.81 per share, which is equal to the closing price of the Company’s common shares on July 22, 2024, the date of grant. The stock option has a 10-year term and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the grant date and the remainder vesting in equal amounts monthly for three years thereafter, subject to Mr. Cooper’s continued service as an employee of, or other service provider to, the Company through the applicable vesting dates.

The stock option was granted by the Company’s independent Compensation Committee of the Board of Directors as an inducement material to Mr. Cooper entering into employment with the Company in accordance with NASDAQ Listing Rule 5635(c)(4). While the stock option was granted outside of the Company’s Amended and Restated enGene Holdings Inc. 2023 Incentive Equity Plan, it will have terms and conditions consistent with those set forth under the Plan.