On May 5, 2022 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported financial results for the first quarter 2022 and provided a business update (Press release, argenx, MAY 5, 2022, View Source [SID1234613607]).

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In a separate press release issued today, argenx also announced positive results from the Phase 3 ADVANCE trial evaluating VYVGART for the treatment of adult patients with ITP. The primary endpoint, demonstrating a significantly higher proportion of VYVGART-treated patients achieved a sustained platelet response than patients receiving placebo, and additional key secondary endpoints were met.

"Our VYVGART commercial launch is off to a strong start, underscoring the significant unmet need for a new treatment option in gMG. We are very encouraged by the early demand from patients and physicians and our team continues to meet the challenge with outstanding execution and deep engagement with our key stakeholders. We look forward to our imminent commercial launch in Japan and an upcoming regulatory decision in Europe, which support our goal to make VYVGART available worldwide. We are confident that the relationships we are building today with the gMG community will establish a strong foundation to continue to deliver on behalf of patients," commented Tim Van Hauwermeiren, Chief Executive Officer of argenx.

"The positive readout from our first registrational ITP trial highlights the promise of efgartigimod as a pipeline-in-a-product with the potential to reach a variety of IgG-mediated autoimmune diseases, even beyond the ten indications we are currently evaluating. We are on track to achieve our argenx 2025 goal to build the next great immunology company while bringing breakthrough innovations to patients and creating long-term value for our stakeholders."

FIRST QUARTER 2022 AND RECENT BUSINESS UPDATE

VYVGART Launch Progress

VYVGART is the first-approved neonatal Fc receptor (FcRn) blocker in the U.S. and Japan. VYVGART is approved in the U.S. for the treatment of adult generalized myasthenia gravis (gMG) patients who are anti-acetylcholine receptor (AChR) antibody positive and in Japan for adult gMG patients. The global launch strategy is on track to make VYVGART available in Europe, China and Canada, as well as select additional regions.

Generated net product revenues of $21.2 million for first full quarter of VYVGART commercial launch in U.S.
Japan commercial launch to start this month following addition of VYVGART to National Health Insurance (NHI) drug price list on April 20, 2022
Decision from European Medicines Agency on Marketing Authorization Application expected in second half of 2022
Zai Lab to file for approval in China in mid-2022 and Medison in Israel in second quarter of 2022
Efgartigimod Research and Development

argenx is positioned to expand its leadership position in FcRn blockade to include ten total autoimmune indications by the end of 2022. Six registrational trials are ongoing with four new proof-of-concept trials to start this year across multiple therapeutic franchises.

Neuromuscular franchise
Biologics License Application (BLA) on track to be filed by end of year for subcutaneous (SC) efgartigimod for gMG, following positive topline results from Phase 3 ADAPT-SC trial
Topline data from registrational ADHERE trial of SC efgartigimod for chronic inflammatory demyelinating polyneuropathy (CIDP) expected in first quarter of 2023
Registrational ALKIVIA trial on track to start this quarter for three subtypes of idiopathic inflammatory myopathies (myositis), including immune-mediated necrotizing myopathy, anti-synthetase syndrome and dermatomyositis; interim analysis planned of first 30 patients of each subtype
Hematology franchise
Positive topline data of VYVGART for primary ITP reported today
Primary endpoint was met; significantly higher proportion of patients receiving VYVGART achieved a sustained platelet response than patients receiving placebo
Statistically significant separation from placebo in key platelet-derived secondary endpoints
Safety and tolerability profile confirmed in second indication
Topline data from second registrational ADVANCE-SC trial of SC efgartigimod for primary ITP expected in first quarter of 2023
Dermatology franchise
Enrollment expanded in registrational ADDRESS trial of SC efgartigimod for pemphigus vulgaris and foliaceus in order to manage ongoing impact of war in Ukraine; topline data now expected in second half of 2023
Registrational BALLAD trial ongoing of SC efgartigimod for bullous pemphigoid with interim analysis planned of first 40 patients
Proof-of-concept trials to be launched in collaboration with Zai Lab and IQVIA
Zai Lab to launch Phase 2 trials in lupus nephritis and membranous nephropathy in 2022 with argenx to lead global registrational programs for each potential indication
IQVIA to launch Phase 2 trials in primary Sjogren’s syndrome in second half of 2022 and COVID-19-mediated postural orthostatic tachycardia syndrome (POTS) in mid-2022
Pipeline Progress

argenx is developing ARGX-117 and ARGX-119, which both have pipeline-in-a-product potential for multiple autoimmune indications.

ARGX-117 (C2 inhibitor)
Proof-of-concept ARDA trial ongoing to evaluate safety, tolerability, and potential dosing regimen in multifocal motor neuropathy (MMN)
Phase 2 proof-of-concept trial expected to start in 2022 for prevention of delayed graft function and/or allograft failure after kidney transplantation
ARGX-119 (muscle-specific kinase (MuSK) agonist)
Phase 1 dose-escalation trial in healthy volunteers expected to start after Clinical Trial Application filing in fourth quarter of 2022
A subsequent Phase 1b trial will assess early signal detection in patients with congenital myasthenic syndrome and MuSK-associated myasthenia gravis
Upcoming Medical Meeting Presentations

14th Myasthenia Gravis Foundation of America International Conference on Myasthenia and Related Disorders (May 10-12, Miami, FL)
Society for Investigative Dermatology Annual Meeting (May 18-21, Portland, Oregon)
Annual Meeting of the Japanese Society of Neurology (May 18-22, Tokyo, Japan)
8th Congress of the European Academy of Neurology (June 25-28, Vienna, Austria)
17th International Congress on Neuromuscular Diseases (July 5-9, Brussels, Belgium)
DETAILS OF THE FINANCIAL RESULTS

Total operating income for the three months ended March 31, 2022 was $31.5 million, compared to $178.6 million for the three months ended March 31, 2021, and consists of:

Product net sales from sales of VYVGART in the U.S. for the three months ended March 31, 2022 were $21.2 million, following the approval of VYVGART by the U.S. Food and Drug Administration (FDA) on December 17, 2021. No product sales were recognized during the comparable prior period.
Collaboration revenue for the three months ended March 31, 2022 was $2.2 million, compared to $158.2 million for three months ended March 31, 2021, resulting in a decrease of $155.9 million. The collaboration revenue for the three months ended March 31, 2021 was primarily attributable to the closing of the strategic collaboration for efgartigimod with Zai Lab, resulting in the recognition of $151.9 million in collaboration revenue.
Other operating income for the three months ended March 31, 2022 was $8.1 million, compared to $20.4 million for three months ended March 31, 2021, resulting in a decrease of $12.3 million. During the three months ended March 31, 2021, the fair value of the argenx profit share in AgomAb Therapeutics NV increased by $11.2 million. There was no change in the fair value during the three months ended March 31, 2022.
Total operating expenses for the three months ended March 31, 2022 were $254.2 million, compared to $178.6 million for the three months ended March 31, 2021, and consists of:

Cost of sales for the three months ended March 31, 2022 amounted to $1.4 million. The cost of sales was recognized with respect to the sale of VYVGART in the U.S. during the first quarter of 2022. There was no cost of sales recognized in the comparable prior period.
Research and development expenses increased by $29.6 million for the three months ended March 31, 2022 to $152.0 million, compared to $122.3 million for the three months ended March 31, 2021. The increase resulted primarily from higher external research and development expenses, mainly related to the efgartigimod program in various indications and other clinical and preclinical programs.
Selling, general and administrative expenses totaled $100.9 million for the three months ended March 31, 2022, compared to $56.3 million for the three months ended March 31, 2021. The increase resulted primarily from higher professional and marketing fees linked to the commercialization of VYVGART in the U.S. and Japan and higher personnel expenses increased due to a planned increase in headcount.
Exchange losses totaled $7.2 million for the three months ended March 31, 2022, compared to $28.9 million for the three months ended March 31, 2021 and are mainly attributable to unrealized exchange rate losses on cash, cash equivalents and current financial assets position in Euro.

Income tax totaled $2.9 million of tax income for the three months ended March 31, 2022, compared to $11.2 million of tax expense for the comparable prior period. Tax income for the three months ended March 31, 2022 consists of $5.0 million of income tax expense and $7.9 million of deferred tax income, compared to $6.2 million of income tax expense and $5 million of deferred tax expense for the comparable prior period.

Net loss for the three months ended March 31, 2022 was $227.2 million compared to $40.4 million for the comparable prior year period. On a per weighted average share basis, the net loss was $4.36 and $0.81 for the three months ended March 31, 2022 and 2021, respectively.

Cash, cash equivalents and current financial assets totaled $2,855.4 million as of March 31, 2022, compared to $2,336.7 million as of December 31, 2021. The increase in cash and cash equivalents and current financial assets resulted primarily from the closing of a global offering of shares, including a U.S. offering and a European private placement, which resulted in the receipt of $761.0 million in net proceeds in March 2022, partially offset by the net cash flows used in operating activities, primarily towards the commercial launch of VYVGART in the U.S. and Japan and continued investment in pipeline expansion.

FINANCIAL GUIDANCE

As of March 31, 2022, argenx had $2.9 billion in cash, cash equivalents and current financial assets. Based on current plans to fund anticipated operating expenses and capital expenditures, argenx expects to utilize approximately $1 billion of its available cash in 2022. The increased spend will support the global VYVGART launches, clinical development of efgartigimod in 10 indications and ARGX-117 in two indications, investment in the global supply chain, and continued focus on pipeline expansion through the Immunology Innovation Program.

EXPECTED 2022 FINANCIAL CALENDAR

July 28, 2022: HY 2022 financial results and business update
October 27, 2022: Q3 2022 financial results and business update
CONFERENCE CALL DETAILS

The first quarter 2022 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.

On May 5, 2022 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, reported financial results for the first quarter 2022 and provided program updates (Press release, Aeglea BioTherapeutics, MAY 5, 2022, View Source [SID1234613606]).

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"We have started off 2022 with significant progress in both our clinical programs. With AGLE-177, we were pleased to see total homocysteine lowering in all patients in our first, low dose cohort and we look forward to sharing clinical data later this year. This program is another example of innovation with human enzyme therapies and has the potential to address a significant unmet need," said Anthony Quinn, M.B., Ch.B., Ph. D., president and chief executive officer of Aeglea. "I’m also proud of the work we have done with our pegzilarginase program enabling our recent BLA submission to the FDA with what we believe are very compelling data to support approval for the treatment of Arginase 1 Deficiency. Some of these data were presented recently at the SIMD Annual Meeting, providing additional insight into the potential positive impact of pegzilarginase treatment. We look forward to working with the FDA throughout the review of our BLA as they assess the potential effectiveness of pegzilarginase in addressing the unmet need that impacts the lives of Arginase 1 Deficiency patients and their families."

Program Updates

Pegzilarginase in Arginase 1 Deficiency

Submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D).
Requested Priority Review at the time of submission.
Presented new data from the PEACE Phase 3 clinical trial at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting, including patient-level outcomes, results from additional secondary endpoints and previously announced topline results. Highlights of the data presented include:
76.7% reduction in mean plasma arginine in pegzilarginase treated patients compared to placebo (p<0.0001); normal plasma arginine levels achieved in 90.5% of pegzilarginase treated patients compared to no patients receiving placebo.
Eleven patients (65%) treated with pegzilarginase reached or exceeded response criteria for ≥1 mobility assessment compared to four patients (44%) receiving placebo.
Eight patients (47%) treated with pegzilarginase met or exceeded prespecified clinical response criteria for ≥2 mobility assessments compared to no patients receiving placebo.
In a post hoc analysis correcting for a missed assessment that was improperly scored as 0 rather than "not assessed," the least squares mean Gross Motor Function Measure Part D score of patients treated with pegzilarginase improved from baseline by 2.25 units compared to placebo (p=0.0896).
Pegzilarginase treated patients showed statistically significant biochemical improvements in measures of ornithine and guanidino compounds compared to placebo.
On track to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in 2022.
AGLE-177 in Homocystinuria

Working to initiate U.S. sites to support enrollment in the ongoing Phase 1/2 clinical trial and a potential pivotal Phase 3 trial after opening an Investigational New Drug (IND) application with the FDA.
Expect to announce Phase 1/2 clinical data in the second half of 2022.
First Quarter 2022 Financial Results

As of March 31, 2022, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $68.6 million. The Company expects its cash, cash equivalents and investments will enable it to fund its operating expenses and capital expenditure requirements into the first quarter of 2023.

Aeglea recognized development fee revenues of $1.4 million in the first quarter of 2022, as a result of its license and supply agreement with Immedica for the commercial rights to pegzilarginase in certain territories outside the U.S. The revenues recorded in the first quarter of 2022 are related to the PEACE Phase 3 trial and BLA package. Aeglea recognized no revenue for the first quarter of 2021.

Research and development expenses totaled $17.0 million for the first quarter of 2022 and $11.9 million for the first quarter of 2021. The increase was primarily associated with expenses related to the BLA submission, management and dosing of ongoing patients in the PEACE Phase 3 trial, and increased enrollment in our Phase 1/2 trial of AGLE-177 for the treatment of patients with Homocystinuria.

General and administrative expenses totaled $8.8 million for the first quarter of 2022 and $6.4 million for the first quarter of 2021. This increase was primarily due to ramping-up the Company’s commercial capabilities and infrastructure as well as personnel expenses.

Net loss totaled $24.4 million and $18.2 million for the first quarter of 2022 and 2021, respectively, with non-cash stock compensation expense of $2.1 million and $1.8 million for the first quarter of 2022 and 2021, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is a novel recombinant human enzyme engineered to degrade the amino acid arginine and has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of people with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality.

The PEACE Phase 3 clinical trial met its primary endpoint with a 76.7% reduction in mean plasma arginine compared to placebo. Additionally, 90.5% of pegzilarginase treated patients achieved normal plasma arginine levels. The arginine lowering was accompanied by a positive trend in Gross Motor Function Measure Part E, a measure of patient mobility. Aeglea’s Phase 1/2 and Phase 2 Open-Label Extension (OLE) data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough Therapy, Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration as well as Orphan Drug Designation from the European Medicines Agency.

About AGLE-177 in Homocystinuria

AGLE-177 is a novel recombinant human enzyme, which is engineered to degrade the amino acid homocysteine and its dimer, homocystine. AGLE-177 is currently being studied in a Phase 1/2 clinical trial for the treatment of patients with Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of total homocysteine. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis), developmental delay, intellectual disability, lens dislocation and severe near sightedness. Preclinical data demonstrated that AGLE-177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received both U.S. and EU Orphan Drug Designation as well as U.S. Rare Pediatric Disease Designation.

On May 5, 2022 PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to discovering, developing and commercialising highly differentiated medicines for devastating diseases, reported that its Board of Directors has approved a share buyback programme in respect of its ordinary shares of one pence each ("Ordinary Shares") up to a maximum consideration of $50 million (the "Share Buyback Programme") (Press release, PureTech Health, MAY 5, 2022, View Source [SID1234613594]).

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The Share Buyback Programme follows consultation with shareholders and is consistent with PureTech’s capital allocation strategy1 and stated approach to drive additional value for its shareholders. The Company had consolidated cash and cash equivalents of $465.7 million and $413.2 million at December 31, 2021 and March 31, 2022, respectively. Given the strength of the Company’s balance sheet, the Board is confident that it can return this capital to shareholders while maintaining sufficient cash on hand to deliver on its strategy in line with its previous cash runway guidance into the first quarter of 2025, including the continued development and expansion of its Wholly Owned Pipeline and strategic investment in its Founded Entities.

The Share Buyback Programme will be limited to the lower of $50 million or 28,589,875 Ordinary Shares, in line with the general authority to repurchase shares, which was granted by shareholders at the most recent annual general meeting held on May 27, 2021, and it will be executed via open market purchases in accordance with applicable regulations, including those related to daily volume and price limitations. Further details of the Share Buyback Programme will follow.

This announcement includes information that is inside information as defined in Article 7 of the Market Abuse Regulation No.596/2014. The person responsible for arranging for the release of this announcement on behalf of PureTech Health plc is Bharatt Chowrira, President, Chief Business, Legal and Operating Officer, and Director.

On May 4, 2022 Singapore biotechnology startup Tetris Therapeutics reported that it has raised US$15 million from the funds managed by Trinity Innovation Bioventure Singapore (TIBS) and its affiliates to develop antibody drug conjugates (ADCs) that aim to address unmet medical needs (Press release, Axcynsis Therapeutics, MAY 4, 2022, View Source [SID1234618244]). ADCs are one of the fastest growing fields in cancer therapy with market size projected to grow at a compound annual growth rate of 24% over the period of 2022 to 2029. Global sales of currently marketed ADCs are forecast to exceed US$16.4 billion in 2026.

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"ADCs consist of three components, antibody, drug payload, and linker. We call ourselves Tetris Therapeutics because like the game of Tetris, different placement of the ADCs building blocks will result in distinct cancer treatment outcomes," said Dr. Zou Bin, founder and CEO of Tetris Therapeutics. "Although the first ADC dates back to the early 2000s, there is an urgent need to develop new generation of ADCs to further improve therapeutic window. We plan on harnessing the cell selectivity of antibodies and the cytotoxicity of small molecule toxins to selectively destroy malignant cancerous cells with a ‘magic bullet’ while sparing healthy tissues."

Established in March 2022, Tetris Therapeutics is a platform-based biotechnology company and firmly believes that ADC is the long-awaited solution to cancer. Funding from this seed round will be used to expand the team, establish the technology platform and develop a robust pipeline. Tetris Therapeutics is currently setting up research and development facilities in Singapore.

In addition to establishing Tetris Therapeutics, Dr. Zou is an adjunct associate professor at National University of Singapore and Nanyang Technological University. He is a serial entrepreneur and was the founder and CEO of Shanghai Blueray Biopharma. Under his leadership, he successfully led the team to develop several candidates into the investigational new drug (IND) stage. Prior to his venture, Dr. Zou started his career as a principal scientist at Novartis Institute for Tropical Disease and discovered Phase II clinical trial drug NITD609.

"Tetris Therapeutics is very grateful for the support of TIBS." Said Dr. Zou, "TIBS is a professional VC specializing in biotechnology investment. I am keen in working with TIBS and future investors to develop new medicines for cancer patients."

"TIBS is excited to invest in its first Singapore biotech." Said Dr. Thomas Keller, Investment Partner of TIBS, "Working with Dr. Zou Bin to nurture Tetris Therapeutics will be a wonderful journey and TIBS is looking forward to the rapid growth of Tetris Therapeutics."

On May 4, 2022 Oncopeptides reported its interim Report Q1 2022 (Presentation, Oncopeptides, MAY 4, 2022, View Source [SID1234615403]).

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