On August 9, 2022 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer, reported financial results for the quarter ended June 30, 2022 (Press release, Allogene, AUG 9, 2022, View Source [SID1234617909]).

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"We feel confident that we could soon initiate the industry’s first Phase 2 pivotal trial for an allogeneic CAR T product, thereby paving the road not just for ALLO-501A, but our entire portfolio," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "As the use of autologous CAR T therapy increases, we are seeing a greater need for an off-the-shelf, allogeneic CAR T option. We are keenly aware of the devastating consequences patients face when only a minority are able to access the curative potential of CAR T therapy. Clinicians have been forced into the unfathomable position of needing to choose which of their patients will receive this potentially life-saving therapy. As patients face access bottlenecks, we are determined to transform CAR T therapy from a complex individualized procedure to an off-the-shelf, on demand pharmaceutical product."

Pipeline Updates

CD19 Program

In the coming weeks, the Company expects to receive clearance from the U.S. Food and Drug Administration (FDA) to initiate a potential Phase 2 pivotal clinical trial for ALLO-501A in relapsed/refractory (r/r) large B cell lymphoma (LBCL). This includes meeting Chemistry Manufacturing and Controls (CMC) requirements to use ALLO-501A from its manufacturing facility, Cell Forge 1.

In June, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-501A in r/r LBCL. RMAT designation was based on data demonstrating the potential of ALLO-501A to address the unmet need for patients who have failed other therapies. Previously presented data support the potential of ALLO-501A to provide a safe and durable alternative to approved autologous CAR T therapies in CAR T naïve patients, including manageable safety with no dose limiting toxicities (DLTs) or graft-vs-host disease (GvHD) and minimal Grade 3 Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), or Grade 3 cytokine release syndrome (CRS). In the Phase 1 ALPHA2 study, nearly all enrolled patients were able to receive therapy with the median time from enrollment to initiation of treatment of two days.

Allogene anticipates providing an update on the Phase 1 portion of the ALPHA and ALPHA2 trials toward the end of 2022. This update will include a few additional patients enrolled in ALPHA2 and will focus on longer-term follow up of patients previously treated in the ALPHA and ALPHA2 trials.

The EXPAND trial is expected to begin in 2022 and is planned to support registration of the lymphodepleting agent ALLO-647. This trial is intended to demonstrate the contribution of ALLO-647 to the lymphodepletion regimen.

BCMA Program

Allogene plans to explore its pivotal study approach and timing for its BCMA program by year end. In parallel, the Company intends to work within the framework afforded by its RMAT designation for ALLO-715 to facilitate FDA interactions and determine the best course forward.

Enrollment continues in the Phase 1 UNIVERSAL trial on ALLO-715 in r/r multiple myeloma (MM). Toward the end of 2022, Allogene intends to provide a clinical update that will focus on the longer-term follow up of patients in UNIVERSAL treated with a single dose of ALLO-715. Allogene has made the decision not to advance ALLO-715 in combination with nirogacestat from SpringWorks Therapeutics into dose expansion cohorts. There was no clear indication that the combination would meaningfully improve the benefit-risk profile of ALLO-715 as a monotherapy. Allogene’s Clinical Trial Collaboration Agreement with SpringWorks is expected to remain in effect until the data from the combination study are fully analyzed.

The IGNITE trial on TurboCAR candidate ALLO-605 continues to enroll patients in the dose escalation portion of this Phase 1 study.

Solid Tumor Programs

ALLO-316, which targets CD70, is the Company’s first AlloCAR T candidate for solid tumors. The ongoing Phase 1 TRAVERSE trial is designed to evaluate the safety, tolerability, anti-tumor efficacy, pharmacokinetics, and pharmacodynamics of ALLO-316 in patients with advanced or metastatic clear cell renal cell carcinoma (RCC).

The FDA previously granted ALLO-316 Fast Track Designation (FTD) based on its potential to address the unmet need for patients with difficult to treat RCC who have failed standard therapies. Metastatic solid tumors have historically been a challenge regardless of treatment modality, and the five-year survival rate for patients with advanced kidney cancer is less than 15%, highlighting the need for innovation.

Corporate Highlights
CF1, Allogene’s commercial scale manufacturing facility located in Newark, California is fully operational and producing GMP material with the intent of supplying ALLO-501A for the planned pivotal study as well as other clinical trials. CF1 is projected to have the ability to manufacture approximately 20,000 ALLO-501A AlloCAR T doses annually at scale. CF1 recently earned a LEED Interior Design and Construction Gold designation from the U.S. Green Building Council (USGBS), a non-profit dedicated to sustainable building design and construction.

In July, Allogene announced the appointment of Stephen L. Mayo, Ph.D., a world-renowned expert in computational protein design, to the company’s Board of Directors. Dr. Mayo is the Bren Professor of Biology and Chemistry and Merkin Institute Professor at the California Institute of Technology in Pasadena, California. Dr. Mayo also serves on the Board of Directors of Merck & Co. and Sarepta Therapeutics, Inc.

Second Quarter Financial Results

Research and development expenses were $57.2 million for the second quarter of 2022, which includes $13.0 million of non-cash stock-based compensation expense.
General and administrative expenses were $19.5 million for the second quarter of 2022, which includes $9.9 million of non-cash stock-based compensation expense.
Net loss for the second quarter of 2022 was $74.8 million, or $0.52 per share, including non-cash stock-based compensation expense of $22.9 million.
The Company had $686 million in cash, cash equivalents, and investments as of June 30, 2022.
Updated 2022 Financial Guidance
While the Company anticipates spending to increase in the second half relative to the first half of 2022, it now expects full year GAAP Operating Expenses to be at the low end of the previous range of $360 million and $390 million. This includes estimated non-cash stock-based compensation expense of $90 million to $100 million and excluding any impact from potential business development activities. Cash burn for 2022 is now expected to be approximately $250 million.

Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss financial results and provide a business update. To access the live conference call by telephone, please dial 1 (800) 715-9871 (U.S.) or 1 (646) 307-1963 (International). The conference ID number for the live call is 7832993. The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

On August 9, 2022 Bio-Path Holdings, Inc., (NASDAQ: BPTH) a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported that it will host a live conference call and audio webcast on Tuesday, August 16, 2022 at 8:30 a.m. ET to report financial results for the second quarter ended June 30, 2022 and to provide a business overview (Press release, Bio-Path Holdings, AUG 9, 2022, View Source [SID1234617908]).

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To access the live conference call, please call (833) 630-1956 (domestic) or (412) 317-1837 (international) at least five minutes prior to the start time. A live audio webcast of the call will also be available on the Presentations section of the Company’s website, www.biopathholdings.com. An archived webcast will be available on the Bio-Path website approximately two hours after the event.

On August 9, 2022 The leaders of Case Western Reserve University and Lawrence Livermore National Laboratory (LLNL) reported that signed an agreement today to accelerate their efforts in shared areas of excellence (Press release, Case Western Reserve University, AUG 9, 2022, View Source [SID1234617907]).

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After three years of steadily growing institutional collaboration in such areas as energy, materials science and polymer processing, Case Western Reserve President Eric W. Kaler welcomed Livermore Director Kim Budil to the university’s campus.

"Both of our teams have embraced opportunities to partner with enormous enthusiasm and ingenuity," President Kaler said. "We are grateful to Congresswoman Marcy Kaptur for helping to catalyze this connection, and very much look forward to building on our strong progress to date."

Located less than an hour’s drive from San Francisco, the Livermore Lab is one of 17 laboratories around the country operating under the auspices of the U.S. Department of Energy. Their mission is "to push the boundaries of science, engineering and technology" to advance aspects of the energy department’s mission.

"We’re pleased to continue our successful collaboration with Case Western Reserve," said LLNL Director Kim Budil. "This agreement provides an important framework as we partner to advance some of today’s most transformative science and technology."

In the document signed Tuesday, the two institutions agreed to:

Exchange and share science and technology ideas;
Enable student opportunities and internships; and
Pursue research activities in areas of joint interest and benefit.
Conversations to collaboration
The two organizations began discussions 2019 at the suggestion of U.S. Rep. Kaptur (D-Ohio) and then National Nuclear Security Administration leader Lisa Gordon-Hagerty, said Grant Goodrich, executive director of the university’s Great Lakes Energy Institute.

Case Western Reserve and LLNL officials said the university and lab have benefitted over the last several years as their relationship has grown. LLNL has provided funding and expertise to advance research at the university, and CWRU provided a talent pipeline to LLNL, beginning with five internships over the last few years and the hiring of an employee in 2020.

CWRU and LLNL representatives also visited their counterpart’s campus or held virtual meetings nearly a dozen times over the last several years, including for technical presentations and seminars.

Among the other highlights:

LLNL awarded four research grants totaling more than $2 million to CWRU faculty since 2020 from its research and development arm. It funded or collaborated on research being conducted by Dustin Tyler, the Kent H. Smith II Professor of Biomedical Engineering; Rohan Akolkar, the Milton and Tamar Maltz Professor of Energy Innovation and the Ohio Eminent Scholar in Advance Energy Research at the Case School of Engineering; Matthew Willard, professor of materials science and engineering; and Laura Bruckman, associate professor of materials science and engineering.

The lab awarded a $1.1 million grant to a broad-based team led by Roger French, the Kyocera Professor in the Department of Materials Science and Engineering at the Case School of Engineering and a team of researchers to investigate material degradation and lifetime extension of advanced materials. The lab is also a member of the National Science Foundation-funded Materials Data Science for Reliability Center at CWRU, a joint project with the University of Pittsburgh—also led by French.

Shared research projects among Akolkar and LLNL researcher Scott McCall; and João Maia, a professor of macromolecular science and engineering and director of the Center for Advanced Polymer Processing and LLNL researcher Fei Zhou.

On August 9, 2022 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the second quarter ended June 30, 2022 and highlighted recent corporate progress (Press release, Avidity Biosciences, AUG 9, 2022, View Source [SID1234617906]).

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"We are very pleased with the team’s execution on our ambitious goal of having three AOC programs addressing three distinct rare diseases in the clinic by the end of this year," said Sarah Boyce, president and chief executive officer. "Our AOC 1001 program for DM1 is progressing with patients from the MARINA trial now enrolling in the recently initiated MARINA-OLE trial. We remain on track to report a preliminary assessment from MARINA in the fourth quarter. We also continue to collaborate with the FSHD and DMD communities as we work to advance AOC 1020 and AOC 1044 into the clinic by the end of this year."

"Our strong cash balance of over $400 million, inclusive of additional funds raised subsequent to June 30th, positions us to be well funded through 2024. This allows us to meaningfully advance our DM1, FSHD and DMD programs, as well as other programs in skeletal muscle and expand the platform into other tissues and cell types," said Mike MacLean, chief financial and chief business officer.

Recent Highlights
Commenced enrolling patients from the MARINA study into a Phase 2 open-label extension study (MARINA-OLE) of AOC 1001 in adults with DM1. Click here for more information on the MARINA-OLE or visit www.clinicaltrials.gov and search for NCT05479981.
Presented data supporting Avidity’s approach of directly targeting DUX4 with AOC 1020 at the 29th Annual FSHD Society International Research Congress. Key highlight from the conference:
Data from a FSHD mouse model showed that a single intravenous dose with the murine version of AOC 1020 prevented development of muscle weakness demonstrated by three functional assays – treadmill running, in vivo force and compound muscle action potential.
Second Quarter 2022 Financial Results
Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $398.2 million as of June 30, 2022, compared to $405.5 million as of December 31, 2021. In addition, subsequent to June 30th, we have raised $18.7 million through our "at the market" program.

Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company (Lilly) and totaled $2.2 million for the second quarter of 2022 compared with $2.6 million for the second quarter of 2021, and $4.0 million for the first six months of 2022 compared with $5.3 million for the first six months of 2021. The decrease was primarily due to timing of reimbursable collaboration-related research and development expenses resulting in the recognition of lower corresponding revenue under the collaboration with Lilly.

Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $39.8 million for the second quarter of 2022 compared with $22.7 million for the second quarter of 2021, and $67.5 million for the first six months of 2022 compared with $43.4 million for the first six months of 2021. The increase was primarily driven by the advancement of AOC 1001, AOC 1020 and AOC 1044, as well as internal and external costs related to the expansion of the company’s overall research capabilities.

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 $8.7 million for the second quarter of 2022 compared with $6.3 million for the second quarter of 2021, and $17.3 million for the first six months of 2022 compared with $12.2 million for the first six months of 2021. The increase was primarily due to higher personnel costs and professional fees as well as facilities costs to support the company’s expanded operations.

On August 9, 2022 Applied DNA Sciences, Inc. (NASDAQ: APDN) (the "Company"), a leader in polymerase chain reaction ("PCR")-based technologies, reported that The City University of New York (CUNY), the largest urban university in the United States, has extended its health services contract for COVID-19 testing and vaccine policy management with the Company’s wholly-owned clinical laboratory subsidiary, Applied DNA Clinical Labs, LLC (ADCL), for 12 months through July 2023 and at the prior contract terms (Press release, Applied DNA Sciences, AUG 9, 2022, View Source [SID1234617905]). ADCL’s COVID-19 testing volumes, including the CUNY contract, fueled the Company’s record fiscal 2021 revenues and consecutive quarterly record revenues in the first half of fiscal 2022 ended March 31, 2022 .

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The contract has been successfully operational since August 2021. Through a partnership with Cleared4 software platform, ADCL provides a wide range of COVID-19 related health services to help CUNY mitigate COVID-19 associated risks. These services, which are provided to CUNY’s 25 campuses and covering 300,000+ students, employees, auxiliary workers, contractors and visitors, include: (i) high-throughput RT-PCR COVID-19 testing (weekly testing and a robust randomized testing program); (ii) vaccination policy and documentation management; and, (iii) facility access control.

"We commend the CUNY Board of Trustees for taking a forward-thinking approach to ensure the continued health and safety of all CUNY stakeholders while remaining committed to the promise and value of in-person learning. As New York City grapples with a resurgence of infections fueled by the Omicron BA.5 subvariant that can potentially elude rapid test detection and more easily reinfect people, including those who have been vaccinated, boosted and/or previously infected, our ability to deliver rapid PCR-based results with actionable reporting and access management for safeCircle clients remains a standout," stated Dr. James A. Hayward, president and CEO of Applied DNA. "Given our infrastructure and client concentration in New York City, we are pleased to continue servicing CUNY while we expand our test offering and services."

About safeCircle
safeCircle is a fully integrated health testing platform that offers a customized suite of services to institutions and their personnel/members that encompasses: program design, RT-PCR and rapid antigen testing, sample kit distribution and collection, test site management, results reporting to individuals and program administrators, facilities access management, variant tracking, and vaccination documentation management.