On March 23, 2022 Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported financial results for the fourth quarter and year ended December 31, 2021, and provided clinical development and operational highlights (Press release, Aura Biosciences, MAR 23, 2022, View Source [SID1234610764]).

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"We have begun 2022 with strong momentum, being on track to advance AU-011 in the clinic in multiple indications with significant unmet medical need. We look forward to initiating the pivotal trial in patients with early stage choroidal melanoma, which is the first indication in our ocular oncology franchise, in the second half of this year," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. "We are excited to have received Orphan Drug Designation from the European Commission, further validating the important role that AU-011 could play in the treatment of patients with this life-threatening disease globally."

Dr. de los Pinos continued: "We are also excited to expand our pipeline into additional solid tumors by initiating our Phase 1 trial in non-muscle invasive bladder cancer (NMIBC) in the second half of this year. We are encouraged by the NMIBC preclinical data that we presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancer Symposium, which supports this indication, as well as the preclinical data that will be presented at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting that further supports the broad oncology potential of our VDC platform. Underscoring our pipeline advancement is a solid balance sheet, with our cash position supporting operations into 2024."

Recent Pipeline Developments

AU-011 is being developed for the treatment of early stage choroidal melanoma (CM), a life threatening rare disease with no approved drugs. Aura plans to select the route of administration and treatment regimen to initiate the pivotal program in CM in the second half of 2022.
Orphan Drug Designation granted to AU-011 by the European Commission for the treatment of uveal melanoma (includes CM). The European Commission grants Orphan Drug Designation for medicinal products intended to treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union (EU) and when no satisfactory method of diagnosis, prevention, or treatment of the condition can be authorized. The designation provides certain benefits and incentives in the EU, including protocol assistance, fee reductions, and ten years of market exclusivity once the medicine is on the market.
Leveraging the broad tumor targeting capabilities of the VDC platform, Aura is planning to pursue clinical development of AU-011 in NMIBC.
Preclinical data demonstrating applicability of AU-011 in bladder cancer was presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancer Symposium. Preclinical results demonstrated that AU-011’s targeting of bladder cancer cells through heparan sulfate proteoglycans is tumor grade agnostic. Tumor binding and distribution of AU-011 was evident in both ex vivo human bladder cancer tissues and in an in vivo murine bladder cancer model. Collectively, these results support further investigation of the use of AU-011 in patients with NMIBC.
NMIBC is an area of high unmet need with no approved targeted therapies. The AU-011 mechanism of action supports the opportunity for use as a first-line treatment following initial diagnosis and/or for the treatment of Bacillus Calmette-Guerin (BCG) refractory disease. The planned Phase 1 trial will evaluate the safety and early proof of mechanism, exploring local necrosis and evidence of immune activation, and Aura expects to initiate the trial in the second half of 2022.
Aura is investigating additional potential indications for AU-011.
Preclinical data highlighting the ability to target a broad number of tumor types will be presented as part of the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data that will be presented at the AACR (Free AACR Whitepaper) annual meeting support AU-011’s potential use to target modified heparan-sulfate proteoglycans that are overexpressed on the tumor cell surface. Activity was observed in every tumor type tested, indicating that there are numerous solid tumors to be considered for AU-011 in the clinic, particularly those derived from neural or epithelial lineages. The AACR (Free AACR Whitepaper) annual meeting is being held April 8-13, 2022 in New Orleans, LA.
Recent Corporate Updates

John Maraganore, Ph.D., joins as a strategic advisor. Dr. Maraganore, former founding Chief Executive Officer (CEO) of Alnylam Pharmaceuticals (Alnylam) and biopharma industry leader, has joined Aura as a strategic advisor. Dr. Maraganore served as the founding CEO and a Director of Alnylam from 2002 to 2021, where he built and led the company from an early technology platform based on RNA interference through global approval and commercialization of the first four RNAi therapeutic medicines, ONPATTRO, GIVLAARI, OXLUMO, and LEQVIO. At Alnylam, he also led the company’s value creation strategy, achieving $25 billion in market capitalization, including over 20 major pharmaceutical alliances.
Recent Events

Aura hosted a virtual Investor Day to discuss AU-011 in ocular oncology on Tuesday, March 22, 2022. The program included an overview of CM, AU-011’s unique mechanism of action, and a summary of the clinical data of AU-011 in CM to date. This was followed by a moderated Q&A with ocular oncology leaders Dr. Carol Shields, Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University, and Dr. Hans Grossniklaus, Senior Professor of Ophthalmology and Founding Director of the Ocular Oncology and Pathology Service at Emory University. The webcast is available here.
Full Year and Fourth Quarter 2021 Financial Results

As of December 31, 2021, Aura had cash and cash equivalents totaling $149.1 million. Aura believes its current cash and cash equivalents are sufficient to fund its operations into 2024.
Research and development expenses increased to $8.0 million and $25.2 million for the three months and full year ended December 31, 2021, respectively, from $3.5 million and $18.0 million for the three months and full year ended December 31, 2020, respectively, primarily due to ongoing manufacturing development costs for AU-011 and higher personnel expenses from growing headcount due to the progression of clinical trials.
General and administrative expenses increased to $3.6 million and $10.1 million for the three months and full year ended December 31, 2021, respectively, from $1.4 million and $4.2 million for the three months and full year ended December 31, 2020, respectively. General and administrative expenses include $0.9 million and $0.1 million of stock-based compensation for the three months ended December 31, 2021 and 2020, respectively. The increase was primarily related to personnel expenses due to an increase in headcount, as well as general increases in audit, legal, consulting, insurance, regulatory, and facilities expenses related to operating as a public company.
Net loss for the three months and full year ended December 31, 2021, was $11.6 million and $35.3 million, respectively, compared to $4.9 million and $22.2 million for the three months and full year ended December 31, 2020, respectively.

On March 23, 2022 Eikon Therapeutics, Inc., a California-based biotechnology company that applies advanced engineering and high-performance computing to the identification of important new medicines, reported the appointment of Roy D. Baynes, MB, BCh, MMed, PhD, as Executive Vice President and Chief Medical Officer, effective July 11, 2022 (Press release, Eikon Therapeutics, MAR 23, 2022, View Source [SID1234610763]). A gifted physician-scientist and leading oncologist, Dr. Baynes is among the most experienced – and most successful – clinical development leaders in the biopharmaceutical industry. Dr. Baynes will serve as a consultant to Eikon Therapeutics beginning April 1, 2022 before transitioning to his full-time role as Executive Vice President and Chief Medical Officer in July.

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Currently Senior Vice President, Global Clinical Development and Chief Medical Officer at Merck, Dr. Baynes received his MB, BCh (Bachelor of Medicine and Surgery) from the University of Witwatersrand, where he also received an MMed (specialist registration), and completed a PhD exploring mechanisms of human iron metabolism. After additional clinical training in Johannesburg, and service on the faculty of the Department of Medicine at the University of Witwatersrand Medical School, Dr. Baynes moved to the United States and joined the Division of Hematology in the Department of Medicine at the University of Kansas Medical Center where he served as Professor of Medicine in both hematology and bone marrow transplantation. From 1997 until 2002, Dr. Baynes led the bone marrow transplantation service at the Karmanos Cancer Institute at Wayne State University in Detroit, where he was also a Professor of Medicine (Hematology and Oncology), and the Charles Martin Professor of Cancer Research. Thereafter, Dr. Baynes joined Amgen, Inc., where he became Vice President of Global Development and head of the hematology/oncology development team. He left Amgen in 2012 to become Senior Vice President at Gilead, Inc., and joined Merck in 2013. At Merck he was responsible for the development of the entire clinical portfolio in Merck Research Laboratories, and was the architect of the development strategy for dozens of important new medicines including Keytruda, a drug that has revolutionized cancer treatment around the world. Dr. Baynes has received innumerable awards for his activities as a physician/scientist, and is currently a board member at Travere, Inc. (NASDAQ: TVTX), Atara, Inc. (NASDAQ: ATRA), and Natera, Inc. (NASDAQ: NTRA).

"Roy is both a deeply knowledgeable physician and an extraordinary leader, who has assembled top-flight clinical teams in multiple different settings, and has led the development of pioneering new treatments across a broad range of therapeutic areas including cardiovascular medicine, metabolism, infectious disease research, and of course, oncology," said Roger M. Perlmutter, MD, PhD, Chairman and CEO of Eikon Therapeutics. "I consider it a great privilege to have the opportunity to work alongside Roy once again, and to pursue our shared goal of bringing significant new medicines to patients suffering from grievous illness. By any measure, Roy ranks among the most important leaders of clinical development in our industry."

In his new role, Dr. Baynes will assume responsibility for building the company’s clinical research and development team and, eventually, serve as the site leader for a new Eikon Therapeutics facility on the eastern seaboard.

"I am delighted to have the opportunity to work with Roger again, to join the remarkably talented Eikon Therapeutics team, and to take advantage of their extraordinary skill in applying super-resolution microscopy to the identification of important new medicines," said Dr. Baynes. "I expect that the computational expertise of Eikon’s scientists and engineers across machine learning and artificial intelligence will both promote the identification of novel compounds, and the analysis of clinical trial data in powerful new ways that together enable the company to advance new and potentially life-extending medicines."

On March 23, 2022 BiomX Inc. (NYSE American: PHGE) ("BiomX" or the "Company"), a clinical-stage microbiome company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, reported that the Company will host a conference call and a live audio webcast on Wednesday, March 30th, 2022, at 8:00 a.m. EDT, to report fourth quarter and full year 2021 financial results and provide business updates (Press release, BiomX, MAR 23, 2022, View Source [SID1234610762]). To participate in the conference call, please dial 1-877-407-0724 (U.S.), 1-809-406-247 (Israel) or 1-201-389-0898 (International). The live and archived webcast will be available in the Investors section of the Company’s website at www.biomx.com.

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On March 23, 2022 Novocure (NASDAQ: NVCR) reported the results of a pre-specified interim analysis for the phase 3 pivotal INNOVATE-3 study evaluating the safety and efficacy of Tumor Treating Fields (TTFields) together with paclitaxel for the treatment of patients with platinum-resistant ovarian cancer (Press release, NovoCure, MAR 23, 2022, View Source [SID1234610761]).

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An independent data monitoring committee (DMC) reviewed the safety data for all platinum-resistant ovarian cancer patients enrolled on the trial. In addition, an analysis of overall survival was performed on the first 540 patients randomized. The interim analysis did not indicate a need to increase the sample size and the DMC recommended that the study should continue to final analysis as planned.

"Completion of the DMC interim analysis represents the next milestone in our journey to address the significant unmet need for patients diagnosed with platinum-resistant ovarian cancer," said Dr. Ely Benaim, Novocure’s Chief Medical Officer. "I would like to thank our investigators and collaborators, ENGOT and The GOG Foundation, as well as our patients for their passion and bravery. We look forward to reviewing final data next year."

The INNOVATE-3 study is designed to evaluate the safety and effectiveness of TTFields together with paclitaxel in patients with platinum-resistant ovarian cancer. There remains a significant unmet need for treatments for platinum-resistant ovarian cancer, for which approximately 16,000 patients are diagnosed each year in the U.S. The primary endpoint of INNOVATE-3 is overall survival. Secondary endpoints include progression-free survival, objective response rate, severity and frequency of adverse events, time to undisputable deterioration in health-related quality of life or death, and quality of life. The study accrued 540 patients as of October 2021 and data will be reviewed in 2023, following an 18 month follow-up period.

The European Network for Gynaecological Oncological Trial groups ("ENGOT") and The GOG Foundation, Inc. ("GOG"), third-party clinical trial networks, are collaborating with Novocure on the trial. Both ENGOT and GOG were involved in the development and facilitation of the trial at leading cancer centers in Europe and the United States.

About Ovarian Cancer
In the U.S., ovarian cancer ranks fifth in cancer deaths among women, with approximately 24,000 women diagnosed each year. Ovarian cancer incidence increases with age, and the median age at time of diagnosis is 63 years old.

Physicians use different combinations of surgery and pharmacological therapies to treat ovarian cancer, depending on the stage of the disease. Surgery is usually used in early stages of the disease and is usually combined with chemotherapy, including paclitaxel and platinum-based chemotherapy. Unfortunately, the majority of patients are diagnosed at an advanced stage when the cancer has spread outside of the ovaries to include regional tissue involvement and/or metastases. Platinum-based chemotherapy remains part of the standard of care in advanced ovarian cancer, but most patients with advanced ovarian cancer will have tumor progression or, more commonly, recurrence. Almost all patients with recurrent disease ultimately develop platinum resistance, and the prognosis for this population remains poor.

About Tumor Treating Fields
Tumor Treating Fields, or TTFields, are electric fields that disrupt cancer cell division. Fundamental scientific research extends across more than two decades and, in all preclinical research to date, TTFields have demonstrated a consistent anti-mitotic effect. TTFields therapy is intended principally for use together with other standard-of-care cancer treatments. There is a growing body of evidence that supports TTFields’ broad applicability with certain other cancer therapies, including radiation therapy, certain chemotherapies and certain immunotherapies. In clinical research and commercial experience to date, TTFields therapy has exhibited no systemic toxicity, with mild to moderate skin irritation being the most common side effect. The TTFields global development program includes a network of preclinical collaborators and a broad range of clinical trials across all phases, including four phase 3 pivotal trials in a variety of tumor types. To date, more than 22,000 patients have been treated with TTFields therapy.

On March 23, 2022 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its President and Chief Executive Officer, Rodney Varner, will provide an overview of the Company’s gene therapies for cancer and diabetes to investors at the 2022 BIO Europe Spring Investor Conference (Press release, Genprex, MAR 23, 2022, View Source [SID1234610760]).

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Conference Details:

Event: BIO Europe Spring Investor Conference
Conference Dates: March 28-31, 2022
Presentation Time: Available on-demand throughout duration of the conference
Presenter: Rodney Varner, President and Chief Executive Officer
Conference Registration: https://bit.ly/3hNGjcW

At the event, Mr. Varner will be available to participate in virtual one-on-one meetings with registered participants. A recording of this presentation will be available for replay on Genprex’s website for a period of time.