On March 15, 2021 CorMedix Inc. (NASDAQ: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of infectious and inflammatory disease, reported that it has been approved by the New Jersey Economic Development Authority (NJEDA) to transfer approximately $1.3 million of the total $1.3 million of its available tax benefits to an unrelated, profitable New Jersey corporation pursuant to the Company’s application to participate in the New Jersey Technology Business Tax Certificate Transfer (NOL) program for State Fiscal Year 2020 (Press release, CorMedix, MAR 15, 2021, View Source [SID1234576645]). The Company anticipates receiving approximately $1.3 million in cash proceeds from the sale of its NOLs during the second quarter of 2021. Closing is subject to NJEDA’s typical closing conditions, which are in process.

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"We are pleased to receive an allocation from this program for the third consecutive year," said Khoso Baluch, Chief Executive Officer of CorMedix. "The funding will help us continue to advance DefenCath toward an anticipated commercial launch upon approval for the U.S. market."

The NOL program enables qualified, unprofitable NJ-based technology or biotechnology companies with fewer than 225 U.S. employees (including parent company and all subsidiaries) to sell a percentage of net operating losses and research and development (R&D) tax credits to unrelated profitable corporations. This allows qualifying technology and biotechnology companies with NOLs to turn their tax losses and credits into cash proceeds to fund growth and operations, including research and development or other allowable expenditures. CorMedix is one of 49 early-stage companies to share in approximately $54.5 million of tax credit transfers approved by NJEDA for the 2020 period.

On March 15, 2021 AVEO Oncology (Nasdaq: AVEO) reported that it will regain its rights to AV-203 outside of North America, its clinical-stage potent humanized IgG1 monoclonal antibody that targets ErbB3 (also known as HER3), following the voluntary termination of its collaboration and license agreement by CANbridge Life Sciences (Press release, AVEO, MAR 15, 2021, View Source [SID1234576644]). AVEO will regain rights to AV-203 in all territories outside of North America, and CANbridge has initiated the process to transfer all preclinical data and materials to AVEO. The transfer of rights and termination of the collaboration and license agreement will become effective on September 5, 2021.

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AV-203 is an IgG1 antibody designed to inhibit both ligand-dependent and ligand-independent ErbB3 signaling. ErbB3 is a receptor that is typically expressed in many human cancers, and AV-203 has demonstrated preclinical activity in multiple tumor models. To date, AVEO has completed a Phase 1, open-label, dose-escalation study of AV-203 in patients with advanced solid tumors (N=22). In this study, one patient had a dose limiting adverse event and the recommended phase 2 dose, or RP2D, is 20 mg/kg. One of two neuregulin positive (NRG1+) patients had a partial response. Neuregulin, the only known ligand for ErbB3, is a potential biomarker which may prove to be predictive of AV-203 anti-tumor activity.

"By reacquiring rights to AV-203 outside of North America, we add global rights to a third IgG1 antibody clinical candidate within our internally developed and diverse portfolio of oncology therapeutics," said Michael Bailey, president and chief executive officer of AVEO. "AV-203 has demonstrated early signs of activity in an NRG1+ patient that suggest it could have meaningful application in several areas of high unmet need in cancer. We look forward to advancing AV-203 in the clinic as part of our strategy for delivering long-term value from our pipeline programs. This strategy includes progress in our immunotherapy combination programs for FOTIVDA (tivozanib), potential initiation of a pivotal study of ficlatuzumab in head and neck squamous cell carcinoma, and the execution of our Phase 1 study of AV-380 for cancer cachexia."

Under their 2016 agreement, AVEO granted CANbridge Life Sciences worldwide rights, excluding the United States, Canada, and Mexico, to AV-203. CANbridge completed their manufacturing obligations under the agreement and AVEO received a $2 million development and regulatory milestone in August 2018 from CANbridge for regulatory approval from the National Medical Products Administration in China of an investigational new drug application for a clinical study of AV-203 in esophageal squamous cell cancer.

On March 15, 2021 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported recent business highlights and pipeline updates, as well as fourth quarter and full year 2020 financial results (Press release, Beam Therapeutics, MAR 15, 2021, View Source [SID1234576643]).

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"Beam has made significant progress in the last 12 months," said John Evans, chief executive officer of Beam. "Since our IPO in February 2020, we named three development candidates from our portfolio; initiated IND-enabling studies for BEAM-101; presented data highlighting our broad set of gene editing technologies; expanded access to novel delivery modalities through the acquisition of Guide Therapeutics; secured and initiated work on our build-to-suit manufacturing facility; enhanced our leadership team with the addition of Amy Simon as chief medical officer; expanded our board with the appointment of Kate Walsh; and further strengthened our capital position and operating runway. In the remainder of 2021, we plan to submit our first Investigational New Drug application for BEAM-101, initiate IND-enabling studies for BEAM-102 and BEAM-201, and nominate our first development candidate from our liver portfolio, positioning Beam to become a clinical stage company with an expansive pipeline moving quickly behind our lead assets. We are well positioned today to advance our platform and pipeline, and remain focused on achieving our vision of providing life-long cures for patients suffering from serious diseases."

Recent Business Highlights

Acquisition of Guide Therapeutics (GuideTx) Expands Novel Base Editing Capabilities: In February 2021, Beam acquired GuideTx, a developer of nonviral drug delivery vehicles for genetic medicines, further expanding the potential reach of Beam’s genetic medicines into target tissues beyond the liver. GuideTx’s technology enables the screening of hundreds of nanoparticles in a single experiment, potentially generating in vivo drug delivery data at significantly greater rates compared to traditional screens. Beam believes that the proprietary screening method will enable identification of lipid nanoparticles with novel biodistribution and high selectivity for target cells. Using this platform and a proprietary library of cationic lipids, GuideTx has identified a broad array of novel lipid nanoparticle formulations.
Balance Sheet Enhanced with $260 Million Private Placement: In January 2021, Beam sold 2,795,700 shares of its common stock to certain institutional investors in a private placement. Aggregate gross proceeds from the offering were approximately $260 million, before deducting fees to the placement agents and other offering expenses payable by the Company.
Leadership Team Strengthened by the Addition of Amy Simon, M.D., as Chief Medical Officer: In March 2021, Beam appointed Amy Simon, M.D., as chief medical officer. Dr. Simon joins Beam from Alnylam Pharmaceuticals, where she spent over a decade in various roles with increasing responsibility for the clinical development of numerous RNAi-based medicines, most recently serving as vice president, clinical development and the lead clinician developing GIVLAARI (givosiran) for acute hepatic porphyria, which was approved by FDA in 2019. Dr. Simon holds a B.A. in history and science from Harvard University and received her M.D. from Tufts University School of Medicine.
Leading Healthcare Executive, Kate Walsh, Appointed to Board of Directors: In January 2021, Kate Walsh, president and chief executive officer of the Boston Medical Center (BMC) health system, joined Beam’s board of directors. BMC is a private, not-for-profit, academic medical center with a community-based focus and is the primary teaching affiliate of Boston University School of Medicine.
Base Editing Progress

Updated Data from Novel Sickle Cell Disease Approaches Presented at 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2020): In December 2020, Beam presented updated data from the company’s complementary base editing approaches to treat hemoglobinopathies, BEAM-101 and BEAM-102, during poster sessions at ASH (Free ASH Whitepaper) 2020. The BEAM-101 data presented demonstrated precise base editing with no off-target editing observed, supporting the company’s planned IND submission in the second half of 2021. Additionally, in vivo data from the company’s Makassar base editing program demonstrated long-term engraftment and editing retention at 16 weeks, with BEAM-102 achieving greater than 90% bi- and mono-allelic Makassar editing in SCD CD34+ HSPCs in vitro.

First Data Highlighting Base Editing Program for Glycogen Storage Disease Type Ia Presented at American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting Digital Experience: In November 2020, Beam presented the first data highlighting its novel base-editing strategy for correcting disease-causing mutations underlying Glycogen Storage Disease Type Ia (GSDIa) during a poster session at AASLD, demonstrating in vivo correction of the R83C and Q347X mutations by ABEs in the livers of two strains of transgenic mice, each carrying one of the two G6PC mutations. Next-generation sequencing data from whole liver extracts revealed significant correction for both R83C and Q347X, with nearly 40% and approximately 70% A-to-G conversion efficiency, respectively, of each mutation back to the normal gene sequence. These significant levels of mutation correction greatly surpassed those expected to restore glucose homeostasis, and functional studies are ongoing to correlate pathophysiology to extent of mutation correction by base-editing. Further, these levels of in vivo correction for GSDIa by base-editing were achieved without detectable creation of double-stranded breaks. In total, these data support base-editing technology as a promising approach for precise correction of causative mutations in GSDIa.
Fourth Quarter and Full Year 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $299.7 million as of December 31, 2020, compared to $91.8 million as of December 31, 2019. This cash balance does not include the proceeds from the January 2021 private placement.
Research & Development (R&D) Expenses: R&D expenses were $32.5 million for the fourth quarter of 2020 and $103.2 million for the full year ended December 31, 2020, compared to $20.2 million for the fourth quarter of 2019 and $54.6 million for the full year ended December 31, 2019.
General & Administrative (G&A) Expenses: G&A expenses were $8.4 million for the fourth quarter of 2020 and $29.6 million for the full year ended December 31, 2020, compared to $6.2 million for the fourth quarter of 2019 and $20.6 million for the full year ended December 31, 2019.
Net Loss: Net loss attributable to common stockholders was $95.5 million, or $1.69 per share, for the fourth quarter of 2020 and $195.9 million, or $4.19 per share, for the full year ended December 31, 2020, compared to $31.1 million, or $4.35 per share, for the fourth quarter of 2019 and $91.0 million, or $14.05 per share, for the full year ended December 31, 2019.

On March 15, 2021 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the fourth quarter and year ended December 31, 2020 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAR 15, 2021, View Source [SID1234576642]).

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"In 2020, we made significant advances across our AOC pipeline and platform. Our work supports Avidity’s evolution to a clinical-stage company as we plan to advance AOC 1001 into the clinic in the second half of this year and progress our FSHD and DMD programs," said Sarah Boyce, President and Chief Executive Officer. "Our discovery efforts continue to focus on expanding our AOC platform into additional muscle diseases and other tissues as we begin to realize our vision of profoundly improving people’s lives by revolutionizing the delivery of RNA treatments."

"Last year we built a solid foundation for growth, anchored by our successful IPO and strong financial position with $328 million in cash at year-end," said Mike MacLean, Chief Financial Officer. "We are investing in our platform and have assembled an experienced team in RNA therapeutics and rare diseases to deliver on our discovery, clinical and commercial objectives."

AOC Platform and Pipeline Highlights

Advanced Lead Program, AOC 1001 for DM1, and Broad Pipeline for Untreated Rare Muscle Diseases. Avidity advanced its first-in-class, lead rare disease program, AOC 1001, toward the clinic and entered into a collaboration with Myotonic Dystrophy Clinical Research Network supporting END-DM1, a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1 (DM1). The company plans to initiate a Phase 1/2 clinical study of AOC 1001 in adults with DM1 in the second half of 2021.

Avidity also advanced additional programs in its muscle franchise including a program for facioscapulohumeral muscular dystrophy (FSHD) and three programs for Duchenne muscular dystrophy (DMD). The AOC FSHD program and the lead AOC DMD program targeting Exon 44 are the most advanced. In 2022, following additional preparatory preclinical studies and regulatory clearance, Avidity plans to commence clinical trials for both of these programs.

Demonstrated Preclinical Proof-of-Concept in Skeletal Muscle and Other Tissues; Advancing Beyond Muscle with Discovery Efforts and Partnering. In preclinical models, Avidity’s AOCs demonstrated robust mRNA reductions in skeletal muscle, cardiac muscle, activated B- and T-cells and tumor infiltrating lymphocytes, macrophages and the liver.

Avidity is advancing beyond muscle with its own discovery efforts and through partnering, as evidenced by its strategic collaborations with Eli Lilly in immunology and MyoKardia, a wholly-owned subsidiary of Bristol Meyers Squibb, in cardiac tissue.
Organizational Highlights

Appointed Experienced and Diverse Team Members to Management and Board of Directors. Avidity has assembled a full management team with deep expertise in the discovery, development and commercialization of RNA therapeutics and rare diseases. Avidity also welcomed Jean Kim and Tamar Thompson to its Board of Directors.
Fourth Quarter and Year-End 2020 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $328.1 million as of December 31, 2020, which includes net proceeds of $274.1 million from the company’s IPO in June 2020, compared to $94.6 million as of December 31, 2019.

Collaboration Revenue: Collaboration revenue solely related to our partnership with Lilly, including reimbursable expenses, was $2.1 million for the fourth quarter of 2020 compared with $1.4 million for the fourth quarter of 2019, and $6.8 million for the full year 2020 compared with $2.3 million for the full year 2019.

Research and Development (R&D) Expenses: R&D expenses, including external and internal costs associated with research activities, primarily relate to the progression of the company’s research on AOC 1001 and other muscle programs. These expenses were $13.6 million for the fourth quarter of 2020 compared with $5.6 million for the fourth quarter of 2019, and $37.6 million for the full year 2020 compared with $14.5 million for the full year 2019. The increases were primarily driven by the progression of AOC 1001 toward the clinic, as well as other programs.

General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs, and patent filing and maintenance fees. These expenses were $4.8 million for the fourth quarter of 2020 compared with $1.8 million for the fourth quarter of 2019, and $13.5 million for the full year 2020 compared with $5.1 million for the full year 2019. The increases were primarily due to higher personnel costs (including noncash stock-based compensation), professional fees and insurance costs related to being a public company, as well as higher patent filing fees.

On March 15, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported that the company will participate at the Oppenheimer 31st Annual Healthcare Conference (Press release, Ascendis Pharma, MAR 15, 2021, View Source [SID1234576641]). Company executives will provide a business overview and an update on the Company’s pipeline programs.

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Details

Event Oppenheimer 31st Annual Healthcare Conference
Location Virtual
Date Wednesday, March 17, 2021
Time 11:20 a.m. Eastern Time
A live webcast of the presentation will be available on the Investors and News section of the Company’s website at www.ascendispharma.com. A webcast replay will also be available on the Company’s website shortly after conclusion of the event for 30 days.