On May 14, 2021 AskAt reported that received a notice of allowance dated April 7, 2021 of a use patent for its EP4 receptor antagonist in the treatment of NASH-associated liver cancer (Press release, AskAt, MAY 14, 2021, View Source [SID1234579973]). The notice was issued by the China National Intellectual Property Administration (CNIPA), in connection with Chinese Patent Application No. 201780004254.2 (Filing Date: November 2, 2017). In addition to China, the use patent has been granted in Canada, Europe, Japan, Mexico, and the U.S.

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!

On May 14, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, reported financial results for the first quarter 2021 and provided a business update (Press release, argenx, MAY 14, 2021, View Source [SID1234579972]).

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!

"We’ve had a strong start to 2021 with the acceptance for review of the BLA and J-MAA for efgartigimod in gMG by the regulatory agencies in the U.S. and Japan. The submissions in China and the EU are on track and we are well-positioned for a global launch of our first-in-class FcRn antagonist. We are building an exceptional team with significant launch experience in neurology and rare disease and hope to reach patients this year," said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

"Efgartigimod has the potential to help people living with gMG as well as several other severe autoimmune diseases mediated by IgG autoantibodies. Our team is advancing registrational trials across four indications with plans to start enrollment in two additional efgartigimod indications this year. We are also broadening our reach within autoimmunity with our first-in-class C2 inhibitor, ARGX-117, from which we will have Phase 1 data mid-year. To complement our clinical pipeline, we continue to invest in our discovery capabilities through our Immunology Innovation Program and strategic technology partnerships that position us well to generate long-term value for shareholders. We are closer each day to building an integrated, innovative, global immunology organization with the goal of impacting the lives of patients," concluded Mr. Van Hauwermeiren.

FIRST QUARTER 2021 AND RECENT BUSINESS UPDATE

Commercial preparations on-track for global launch of IV efgartigimod for gMG, including regulatory submissions, initial salesforce hires and key stakeholder engagement efforts

Biologics License Application (BLA) for IV efgartigimod for treatment of gMG accepted for review by U.S. Food and Drug Administration (FDA) with target action date of December 17, 2021 under Prescription Drug User Fee Act (PDUFA)
J-MAA submitted to Japan’s PMDA and accepted for review with anticipated Japan commercial launch in 2022
MAA expected to be filed with European Medicines Agency (EMA) in second half of 2021
Zai Lab Limited to discuss potential accelerated regulatory pathway for approval in China with National Medical Products Administration (NMPA)
Commercial readiness activities on-track, including:
Continued build-out of global commercial organization, including hiring of U.S. regional business directors during first quarter
Launched pre-approval access (PAA) program in March 2021 in U.S., Canada and Europe to open availability of efgartigimod to people living with gMG who meet the pre-approval access program criteria

Immunology pipeline advancing with five ongoing registrational trials of efgartigimod and initial upcoming data from second potential pipeline-in a product candidate, ARGX-117

Enrollment ongoing in five registrational trials across four indications, including ADAPT-SC (gMG), ADHERE (chronic inflammatory demyelinating polyneuropathy or CIDP), ADVANCE and ADVANCE-SC (primary immune thrombocytopenia or ITP), and ADDRESS (pemphigus)
Go-forward decision confirmed in February 2021 in ADHERE trial evaluating subcutaneous (SC) efgartigimod in CIDP based on evaluation of interim safety and efficacy assessments that surpassed pre-defined threshold
Enrollment in trials for fifth and sixth indications to begin in 2021
Additional efgartigimod indications to be evaluated as part of collaboration with Zai Lab Limited
Data expected mid-year from Phase 1 trial of C2 inhibitor, ARGX-117; Phase 2 dosing plan to be identified for indications, including multifocal motor neuropathy (MMN)
Phase 2 trial of MMN on track to start by end of 2021
Combination trials of cusatuzumab remain ongoing for treatment of acute myeloid leukemia (AML) as part of global collaboration and licensing agreement with Cilag GmbH International, an affiliate of Janssen
Decision to initiate additional cusatuzumab studies under collaboration will be determined following review of all available data
Immunology Innovation Program (IIP) continues to grow pipeline through wholly-owned development, partnered opportunities, asset-centric spinoff companies and the addition of strategic technology capabilities

Preclinical work ongoing in early-stage pipeline, including ARGX-118, ARGX-119 and ARGX-120
15-20 discovery programs under evaluation at any point in time that have emerged from IIP
Initiated collaboration and license agreement with Elektrofi to explore new subcutaneous formulations for current and future pipeline candidates, including efgartigimod
Secured exclusivity for FcRn and one additional target
Ongoing development of ARGX-112 (LEO Pharma), ARGX-114 (AgomAb), ARGX-115 (ABBV-151, AbbVie) and ARGX-116 (Staten Biotech) by IIP collaboration partners

Strong balance sheet and expanded Board of Directors support transition into integrated, global immunology organization

Completed public offering of 3,593,750 ordinary shares in February 2021 with gross proceeds of $1.15 billion
Implemented transition agreement for Chief Financial Officer Eric Castaldi as part of evolution to commercial-stage company; recruitment efforts ongoing for U.S.-based successor
Proposed resolutions presented during Annual General Meeting of Shareholders were approved, including:
Appointment to Board of Directors of Yvonne Greenstreet, President and Chief Operating Officer of Alnylam
Re-appointment of Anthony Rosenberg to Board of Directors
Approval of new remuneration policy
argenx to host virtual R&D Day on July 20, 2021 to share long-term corporate vision, disclose additional potential efgartigimod indications and provide updates across immunology pipeline.

As of January 1, 2021, the Company changed its functional and presentation currency from euro to U.S. dollars, which results in reporting its 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 $2,907.4 million on March 31, 2021, compared to $1,996.5 million on December 31, 2020. The increase in cash, 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 in the form of newly issued Zai Lab shares received as part of the strategic collaboration for efgartigimod in Greater China, partially offset by (iii) the payment of $98.0 million related to the purchase of a priority review voucher from Bayer HealthCare Pharmaceuticals and other net cash flows used in operating activities.

Total operating income increased by $141.6 million for the three months ended March 31, 2021 to $167.4 million, compared to $25.8 million for the three months ended March 31, 2020. The increase was primarily due to 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 $17.7 million for the three months ended March 31, 2021 to $122.3 million, compared to $104.7 million for the three months ended March 31, 2020. The increase in the first three months of 2021 resulted primarily from higher external research and development expenses, mainly related to the efgartigimod program in multiple 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 $56.3 million for the three months ended March 31, 2021, compared to $27.6 million for the three months ended March 31, 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 efgartigimod.

The increase in fair value on non-current financial assets amounted to $11.2 million for the three months ended March 31, 2021, which is the result of the closing of a Series B financing round of AgomAb Therapeutics, for which the Company maintains a profit share in exchange for granting the license for the use of HGF-mimetic antibodies from the SIMPLE Antibody platform.

Exchange losses totaled $28.8 million for the three months ended March 31, 2021, compared to an exchange gain of $23.0 million for the three months ended March 31, 2020. As a result of the change in the Company’s functional and presentation currency, the exchange losses for the three months ended March 31, 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 2021 cash burn to approximately double from 2020. The increased spend will support 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 seven expected global trials of efgartigimod, and the continued investment in its Immunology Innovation Program.

EXPECTED 2021 FINANCIAL CALENDAR

July 29, 2021: HY 2021 financial results and business update
October 28, 2021: Q3 2021 financial results and business update
CONFERENCE CALL DETAILS
The first 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.

On March 14, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted priority review for the company’s New Drug Application (NDA), which was submitted in March (Press release, Astellas, MAY 14, 2021, View Source [SID1234579945]). If approved, enfortumab vedotin would be the first antibody-drug conjugate (ADC) available in Japan for the treatment of patients with locally advanced or metastatic urothelial cancer that has progressed after anti-cancer medication.

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!

Priority reviews are granted by MHLW for applications based on their clinical usefulness and the seriousness of the diseases for which they are indicated.1 The NDA includes data from the phase 3 EV-301 trial and the phase 2 EV-201 trial, both global clinical trials with investigational sites in Japan.

"The decision by the Ministry of Health, Labour and Welfare to evaluate enfortumab vedotin under priority review reflects the urgent need for new medicines to treat advanced urothelial cancer in Japan, where an estimated 9,500 people die from urothelial cancer each year," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head, Astellas.2

Urothelial cancer makes up approximately 90 percent of cases of bladder cancer.3 Locally advanced or metastatic urothelial cancer is an aggressive disease that is associated with poor survival and high healthcare costs.4

About the EV-301 Trial
The EV-301 trial (NCT03474107) is a global, multicenter, open-label, randomized phase 3 trial designed to evaluate enfortumab vedotin versus physician’s choice of chemotherapy (docetaxel, paclitaxel or vinflunine) in approximately 600 patients with locally advanced or metastatic urothelial cancer who were previously treated with a PD-1/L1 inhibitor and platinum-based therapy.5 The primary endpoint is overall survival and secondary endpoints include progression-free survival, overall response rate, duration of response and disease control rate, as well as assessment of safety/tolerability and quality-of-life parameters.

About the EV-201 Trial
The EV-201 trial (NCT03219333) is a single-arm, dual-cohort, pivotal phase 2 clinical trial of enfortumab vedotin for patients with locally advanced or metastatic urothelial cancer who have been previously treated with a PD-1 or PD-L1 inhibitor, including those who have also been treated with a platinum-containing chemotherapy (cohort 1) and those who have not received a platinum-containing chemotherapy in this setting and who are ineligible for cisplatin (cohort 2). The trial enrolled 128 patients in cohort 1 and 91 patients in cohort 2 at multiple centers internationally.6 The primary endpoint is confirmed objective response rate per blinded independent central review. Secondary endpoints include assessments of duration of response, disease control rate, progression-free survival, overall survival, safety and tolerability.

About Enfortumab Vedotin
Enfortumab vedotin is an antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.7,8 Nonclinical data suggest the anticancer activity of enfortumab vedotin is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).7

About the Astellas and Seagen Collaboration
Astellas and Seagen Inc. are co-developing enfortumab vedotin under a 50:50 worldwide development and commercialization collaboration. In the United States, Astellas and Seagen co-promote enfortumab vedotin under the brand name PADCEV (enfortumab vedotin-ejfv). In the Americas outside the US, Seagen holds responsibility for commercialization activities and regulatory filings. Outside of the Americas, Astellas holds responsibility for commercialization activities and regulatory filings.

On May 13, 2021 Interline Therapeutics, a Foresite Labs incubated company focused on systematically elucidating protein communities to define molecular mechanisms of disease, reported that the company has raised $92 million (Press release, Interline Therapeutics, MAY 13, 2021, View Source [SID1234627668]). Foresite Capital and ARCH Venture Partners co-led the financing, which will be used to expand the research platform and advance six preclinical therapeutic programs.

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!

Dysfunctional protein communities drive disease

Proteins have generally been studied as isolated entities, but in nature function in protein communities – interconnected networks of dynamic protein interactions. Medical researchers have been limited by an incomplete understanding of how these communities impact common diseases, lowering the likelihood of successful drug development.

Reshaping protein communities to treat common diseases

Interline Therapeutics uses genomics, proteomics, structural biology and computational chemistry to systematically map and modulate protein communities. Interline is leveraging recent advancements in these technologies, as well as collaborations with leading academic groups, to develop a precision medicine platform focused on three essential areas:

Genomics: Prioritize genetic variants that drive disease by altering protein communities
Communities: Identify the specific molecular mechanisms through which these variants change protein community dynamics, enabled by the application of machine learning
Modulators: Discover and characterize drugs that reshape these communities
The discovery platform will allow the company to identify new medicines targeting genetically validated signaling pathways and ensure that drug candidates comprehensively correct dysfunctional disease networks.

Interline was co-founded by CEO Zachary Sweeney, a scientist with a track record of growing and leading successful drug discovery groups. Company leaders bring decades of experience discovering innovative medicines at Amgen, Denali Therapeutics, Genentech, Novartis and Nurix. The team includes Nick Galli, Chief Operating Officer, Don Kirkpatrick, Chief Technology Officer, Mario Cardozo, Vice President Computational Chemistry and Cheminformatics, Anj Saha, Vice President Discovery Pharmacology and David Tully, Vice President Medicinal Chemistry.

"Biomedical research is newly enabled by advances in human genomic and proteomic technologies," said Zachary Sweeney, CEO. "We are excited to collaborate with leading research groups to develop a protein-focused roadmap for precision medicine."

The company will continue to collaborate with Foresite Labs in the fields of data science and genomics. Interline also launches with foundational collaborations with University of California San Francisco and Memorial Sloan Kettering Cancer Center, as well as an internationally recognized team of founding scientific advisors, including:

John Chodera, PhD, Associate Member and Laboratory Director, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center
Wade Harper, PhD, Chairman and Bert and Natalie Vallee Professor of Molecular Pathology and Cell Biology, Harvard Medical School
Nevan J. Krogan, PhD, Professor, Cellular and Molecular Pharmacology, UCSF, Director, Quantitative Biosciences Institutes, and Senior Investigator, Gladstone Institutes
Brenda Schulman, PhD, Director, Molecular Machines and Signaling, Max Planck Institute of Biochemistry
Mike Varney, PhD, Former Executive Vice President Research and Early Development of Genentech
"Interline has assembled a world-class set of scientific advisors and an outstanding leadership team," said Mike Varney, member of the company’s Board of Directors. "The company has made great progress toward mapping and understanding the perturbations in protein dynamics caused by genetic mutations, and leveraging these insights to correct the protein communities found in cancer and inflammation."

"By creating a platform to discover, understand and re-engineer protein interactions, we will find new approaches to treat debilitating diseases," commented Vikram Bajaj, PhD, co-founder and CEO of Foresite Labs. "Zach and the Interline team possess an incredible breadth of experience, and we are thrilled to help accelerate Interline’s growth."

Jay Parrish, PhD and Venture Partner at ARCH Venture Partners said, "Our investment in Interline Therapeutics reflects our commitment to their vision of protein-centric systems biology drug discovery. The platform allows for unique insights into mechanisms of serious illnesses with high unmet need, while delivering novel paths and high-impact therapeutics to tackle validated disease targets."

For more information about Interline Therapeutics, please visit www.interlinetx.com.

Dr. Chodera has served as a scientific advisor for and has financial interests in Interline Therapeutics, and has also provided consulting and advisory services to Foresite Labs. Memorial Sloan Kettering Cancer Center (MSK) has intellectual property rights and associated financial interests related to Interline Therapeutics by virtue of licensing agreements between MSK and Interline Therapeutics.

On May 13, 2021 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Decibel Therapeutics, MAY 13, 2021, View Source [SID1234585162]).

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!

"The start of 2021 marked a pivotal time for Decibel as we successfully closed our upsized IPO. With this strong financial foundation, we are in a position to further advance our exciting pipeline of clinical, preclinical and discovery programs with the overarching goal of bringing the privileges of hearing and balance to people in need. In early 2021, we announced encouraging preclinical results on our lead gene therapy program, DB-OTO, for the treatment of people with otoferlin deficiency. We also established a critical cGMP manufacturing and development relationship with Catalent," said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. "Beyond DB-OTO, we remain encouraged by the progress in our gene therapy programs for congenital, monogenic hearing loss and look forward to announcing the program target for our AAV.104 program in 2021."

Company Highlights

Closed Second Tranche of Series D Financing and Upsized Initial Public Offering: In February 2021, Decibel completed its Series D financing, closing the second tranche and receiving net proceeds of $27.4 million. Additionally, Decibel completed its Initial Public Offering (IPO) of 7,662,000 shares of common stock at a public offering price of $18.00 per share. Net proceeds from the offering, after deducting underwriting discounts and offering expenses, were approximately $124.8 million.
Pipeline Progress

Gene Therapies for Congenital, Monogenic Hearing Loss

Continued Development of Lead Gene Therapy Program, DB-OTO, for Otoferlin (OTOF): In February 2021, Decibel established manufacturing capabilities for its lead gene therapy product candidate, DB-OTO. Under the new agreement, Catalent will provide Decibel with cGMP manufacturing and process and analytical development of DB-OTO.
Presented New Preclinical Data on DB-OTO and Surgical Approach at the 44th Annual Association for Research in Otolaryngology Conference (ARO) and the Annual Scientific and Technology Conference of the American Auditory Society: In the first quarter of 2021, Decibel presented new preclinical findings to support the development of DB-OTO. Presentations highlighted data demonstrating that the Company’s proprietary dual vector technology and cell-selective promoter enabled expression of OTOF in hair cells and durably restored hearing in mice and drove highly selective expression of a reporter gene in hair cells of non-human primates across the cochlear length.
On Track for DB-OTO Key Milestones in 2022: Decibel expects to submit an investigational new drug application (IND) with the U.S. Food and Drug Administration (FDA) and/or a Clinical Trials Application (CTA) in Europe and initiate a Phase 1/2 clinical trial for DB-OTO in pediatric patients with congenital hearing loss in 2022.
Preclinical Pipeline Expansion Continues: Decibel expects to announce the program target for its AAV.104 discovery program in patients with autosomal recessive hearing disorders in 2021.
Gene Therapies for Hair Cell Regeneration

Preclinical Pipeline Expansion Continues: Decibel continues to advance DB-ATO and AAV.201, its gene therapy programs for regeneration of hair cells in the vestibule for the treatment of bilateral vestibulopathy, and its gene therapy program to regenerate hair cells in the cochlea for the treatment of sensorineural hearing loss. Based on findings from recently completed behavioral studies of DB-ATO, the Company did not see sufficient functional recovery to continue to move DB-ATO to development candidate in 2021. The Company plans to announce the program target for AAV.201 in 2022.
Otoprotection Therapeutic

Updated Timeline to Report Interim Results from Phase 1b Proof-of-Concept Trial of DB-020 for the Treatment of Cisplatin-Induced Hearing Loss: The reporting of interim results from the ongoing Phase 1b clinical trial of DB-020 in patients with cisplatin-induced hearing loss is now expected in the first half of 2022 due to continued impact of the COVID-19 pandemic on the pace of patient recruitment in the United States. Due to COVID-19 restrictions, sites in the United States have been delayed in recruiting, but are now open and actively recruiting for the trial, along with the active sites in Australia.
First Quarter 2021 Financial Results:

Cash Position: As of March 31, 2021, cash, cash equivalents and available-for-sale securities were $191.1 million, compared to $54.3 million as of December 31, 2020. The increase in cash, cash equivalents and available-for-sale securities was due to the sale of the Company’s Series D convertible preferred stock and common stock in the Company’s IPO completed in February 2021.
Research and Development Expenses: Research and development expenses were $6.0 million for the first quarter of 2021, compared to $7.4 million for the first quarter of 2020. The decrease in research and development expenses for the first quarter of 2021 was primarily due to $1.8 million decrease in personnel-related costs due to reduced headcount, driven primarily by a reduction-in-force conducted in January 2020 and a $0.8 million decrease in expenses incurred for our DB-020 program driven by decreased activity as a result of delays due to the COVID-19 pandemic, partially offset by an increase of $1.2 million in other indirect research and development expenses.
General and Administrative Expenses: General and administrative expenses were $4.9 million for the first quarter of 2021, compared to $4.2 million for the same period in 2020. The increase in general and administrative expenses for the first quarter of 2021 was primarily attributable to $1.0 million increase in professional fees, driven primarily by expenses related to consulting, accounting advisory and audit services incurred as a result of becoming a public company in February 2021.
Financial Guidance:

Based on its current operating and development plans, Decibel believes that its existing cash, cash equivalents and available-for-sale securities will fund its pipeline programs and operating expenses into 2024.