On May 12, 2021 Marker Therapeutics, Inc. (Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported financial results for the first quarter ended March 31, 2021 (Press release, TapImmune, MAY 12, 2021, View Source [SID1234579880]).

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"We had a productive first quarter supported by our recently completed financing, strengthening our overall cash position and enabling continued growth and expansion of our Multi-TAA pipeline," said Peter L. Hoang, President & CEO of Marker Therapeutics. "In addition, we continue to make strong progress on both the clinical and manufacturing fronts. In March, we dosed the first patient in the safety lead-in portion of our Phase 2 trial in post-transplant acute myeloid leukemia, or AML, and continue to activate clinical sites. In parallel, we continue to optimize the MT-401 cell therapy manufacturing process, which we believe could result in an increase in the number of T cells available for patient administration—among other benefits—as we prepare to operationalize our new in-house cGMP facility in the first half of the year."

PROGRAM UPDATES

In March 2021, Marker dosed the first patient in the safety lead-in portion of its Phase 2 trial in AML, which is expected to enroll a total of six patients: three of which will be treated with MT-401 manufactured with a legacy reagent, and the remaining three to be treated with study drug manufactured with a new reagent from an alternate supplier.
The clinical operations team has made considerable progress in opening sites to enroll patients for the safety lead-in portion of the AML trial. The Company has also received commitments from additional clinical sites to participate in the Phase 2 AML trial following the safety lead-in phase and anticipates activating a total of approximately 20 sites.
Marker continues to streamline and simplify the MT-401 manufacturing process, which could potentially result in a product with a superior T cell phenotype and improved antigen specificity as compared to the original process. The new process improvements have been updated in the CMC section of the IND and will be used for all patients in the Marker AML Phase 2 clinical trial.
Marker presented early results of robotic automation of T cell generation for the treatment of AML at the American Society of Gene & Cell Therapy 24th Annual Meeting on May 11, 2021. The results will also be presented at the upcoming International Society for Cell & Gene Therapy 2021 Annual Meeting, taking place virtually May 26-28, 2021.
BUSINESS UPDATES

In March, Marker closed an underwritten public offering of 32,282,857 shares of its common stock at a public offering price of $1.75 per share. The gross proceeds to Marker from the offering, before deducting the underwriting discounts and commissions and other offering expenses, were approximately $56.5 million.
The Company is preparing to open its new cGMP manufacturing facility in Houston, TX, which will be used to manufacture study drug for Marker’s Phase 2 AML trial (MT-401) and for future hematological and solid tumor trials, in addition to the potential commercialization of any approved products. Marker initiated the technology transfer from Baylor College of Medicine to the cGMP facility in Q1 2021 and expects the facility to be fully operational in the first half of 2021.
ANTICIPATED PROGRAM MILESTONES

AML Trial Milestones

Complete safety lead-in (6 patients) in Q2 2021
Initiate main portion of Phase 2 trial in Q3 2021
Complete enrollment of 20 patients in main portion of Phase 2 trial in Q4 2021
Topline readout of Group 2 (active disease) in Q1 2022
Manufacturing Milestones

Receive regulatory approval for Marker cGMP in Q2 2021
Manufacture MT-401 at Marker cGMP for Phase 2 AML trial in Q3 2021
FIRST QUARTER 2021 FINANCIAL RESULTS

Cash Position and Guidance: At March 31, 2021, Marker had cash and cash equivalents of $64.5 million. The Company believes that its existing cash and cash equivalents will fund its operating expenses and capital expenditure requirements into Q1 2023.
R&D Expenses: Research and development expenses were $5.6 million for the quarter ended March 31, 2021 compared to $3.8 million for the quarter ended March 31, 2020. The increase was primarily attributable to increases in headcount-related expenses and infrastructure expenses due to growth of research and development operations.
G&A Expenses: General and administrative expenses were $3.1 million for the quarter ended March 31, 2021 compared to $2.8 million for the quarter ended March 31, 2020.
Net Loss: Marker reported a net loss of $8.8 million for the quarter ended March 31, 2021, compared to a net loss of $6.5 million for the quarter ended March 31, 2020.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first quarter 2021 financial results and provide a corporate update today at 5:00 p.m. EDT. The webcast will be accessible in the Investors section of the Company’s website at markertherapeutics.com. Individuals can participate in the conference call by dialing 877-407-8913 (domestic) or 201-689-8201 (international) and referring to the "Marker Therapeutics First Quarter 2021 Earnings Call."

On May 12, 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 first quarter ended March 31, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAY 12, 2021, View Source [SID1234579767]).

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"AOC 1001, the first program from our AOC platform, is advancing toward the clinic and is on track to begin a Phase 1/2 study in adults with DM1 in the second half of this year. Our skeletal muscle pipeline is also progressing as planned while we continue to expand our discovery efforts to other indications," said Sarah Boyce, president and chief executive officer. "We are proud to be at the forefront of RNA innovation by delivering on the promise of our AOC platform and pipeline."

Mike MacLean, chief financial officer, added, "We are off to a great start this year with a strong financial position of over $300 million at the end of the first quarter. This allows us to progress AOC 1001 while continuing to invest in our other pipeline programs and our AOC platform, which we believe is a sustainable engine with the potential to have a broad impact across both rare and other serious diseases."

AOC Platform and Pipeline Highlights

Advanced Lead Program, AOC 1001 for DM1, and Broad Pipeline for Untreated Rare Muscle Diseases.

In April 2021, Avidity presented preclinical data for AOC 1001 for myotonic dystrophy type 1 (DM1) in an oral presentation as part of the Emerging Science Session at the American Academy of Neurology (AAN) 2021 Virtual Annual Meeting. Avidity scientists have demonstrated activity and potency of siRNAs against the dystrophy protein kinase (DMPK) gene in muscle cells derived from patients with DM1. These data demonstrated activity in the nucleus and cytoplasm. The presentation also showed that single doses of AOC 1001 produced 75% reductions in DMPK mRNA expression in preclinical studies that were maintained for months post-dosing. Following regulatory clearance, Avidity remains on track with plans to begin 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. Avidity remains on track with plans to commence clinical trials for both programs in 2022, following additional preparatory preclinical studies and regulatory clearance.
Upcoming Events

Avidity’s Virtual Investor & Analyst Event Series – Volume 1. Recently, Avidity launched its Virtual Investor and Analyst Event Series. The first virtual event entitled "Engineering Antibody Oligonucleotide Conjugates" will be held on Wednesday, May 19th, 2021 at 8am PT/11am ET.

The virtual event will feature a panel discussion with leading RNA therapeutics key opinion leaders Phillip D. Zamore, Ph.D., Chair, RNA Therapeutics Institute at UMASS and Steven F. Dowdy, Ph.D., Professor of Cellular and Molecular Medicine at UCSD. To register for the event or for more information, please email [email protected].
First Quarter 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $307.9 million as of March 31, 2021, compared to $328.1 million as of December 31, 2020.
Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $2.7 million for the first quarter of 2021 compared with $1.4 million for the first quarter of 2020.
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 $20.7 million for the first quarter of 2021 compared with $5.5 million for the first quarter of 2020. The increase was primarily driven by the progression of AOC 1001 toward the clinic, as well as research for 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 $5.9 million for the first quarter of 2021 compared with $2.0 million for the first quarter of 2020. The increase was primarily due to higher personnel costs (including noncash stock-based compensation), professional fees and insurance costs related to being a public company.

On May 12, 2021 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported that Fred Schwarzer, Chief Executive Officer, will participate in a fireside chat at the 2021 RBC Capital Markets Global Healthcare Conference on May 19, 2021 at 1:55 p.m. EDT (Press release, IGM Biosciences, MAY 12, 2021, View Source [SID1234579784]). The conference will be held in a virtual meeting format.

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A live webcast of the event will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following the presentation.

On May 12, 2021 Recursion Pharmaceuticals, Inc. (Nasdaq : RXRX), a clinical-stage biotechnology company decoding biology by integrating technological innovations across biology, chemistry, automation, data science, and engineering, reported financial results for the first quarter of 2021 and provided business updates (Press release, Recursion Pharmaceuticals, MAY 12, 2021, View Source [SID1234579800]).

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"We are thrilled to have led Recursion through a successful IPO process which garnered support from new and existing healthcare and technology investors and better capitalized the company’s mission of decoding biology to radically improve lives," said Recursion Co-Founder and CEO Chris Gibson Ph.D. He continued, "We are excited about Recursion’s continued foray into inferential search, how that approach has quickly added many new programs to our portfolio in a short time, and how our new supercomputer, BioHive-1, will enable faster training and iteration of our machine learning algorithms and the ability to train on the totality of our nearly 8 petabytes of relatable biological data. And finally, we continue to make strides expanding, advancing, and executing on our pipeline of potential new medicines, including our four clinical stage programs and our first new chemical entity now headed towards clinical development."

Recursion finished the first quarter of 2021 with a portfolio of 4 clinical stage programs, 4 preclinical programs, 4 late discovery programs, and 25 early discovery programs, for a total of 37 research and development programs. Additionally, Recursion continued scaling the total number of executed phenomic experiments to over 67 million, the size of its total dataset to nearly 8 petabytes, and the number of biological inferences to over 92 billion. We accomplished this by utilizing a cellular library of 36 cell types, an in-house chemical library of over 706 thousand compounds, an in silico library of over 12 billion chemical scaffolds, and a growing team of more than 200 Recursionauts that is balanced between life scientists and computational and technical experts.

Summary of Business Highlights

Clinical Programs
Familial adenomatous polyposis (REC-4881) : We plan to initiate a Phase 2, randomized, double-blind, placebo-controlled study to evaluate efficacy and safety of REC-4881 in classical FAP patients within the next 4 to 5 quarters.
GM2 gangliosidosis (REC-3599) : We are generating additional pharmacodynamic data in an animal model of GM2 in anticipation of enrolling the first patient in an open-label Phase 2 trial in the next 4 to 5 quarters.
Neurofibromatosis type 2 (REC-2282) : The protocol has been updated based on scientific advice from the MHRA and FDA feedback from the special protocol assessment (SPA). We expect to enroll the first patient in an adaptive Phase 2/3, randomized, multicenter study within the next 4 to 5 quarters.
Cerebral cavernous malformation (REC-994) : We finalized the Phase 2 protocol and submitted it to the FDA. Subject to completion of all required CMC steps, we expect to enroll the first patient in a Phase 2, double-blind, placebo-controlled, safety, tolerability and exploratory efficacy study within the next 4 to 5 quarters.
Notable Preclinical Programs
Clostridium difficile colitis (REC-163964, REC-164014, REC-164067) : We completed exploratory, non-clinical, safety studies with three lead molecules to enable selection of a development candidate.
Immune checkpoint resistance in STK11-mutant NSCLC : We completed dose-optimization studies of REC-64151 that supported efficacy in rodent models at exposures achievable in humans.
Notable Late Discovery Programs
Neuroinflammation : This program is currently in the lead-optimization phase. The project has made progress in optimization of potency and pharmacokinetics of molecules in our lead series.
Batten disease : The program is currently in the lead-optimization phase. The project has made progress in optimization of potency and pharmacokinetics of molecules in our lead series.
Charcot-Marie-Tooth type 2A : The chemistry strategy has been constructed and relevant biochemical and cellular orthogonal assays are being developed.
Oncology – small molecule MYC Inhibitors : We have now confirmed that several molecules from multiple series exhibit activity in a c-Myc protein turnover assay and made progress in elucidating mechanisms underlying modulation of c-Myc degradation.
Partnership : There has been continued progress in our collaboration with Bayer to discover small molecule drug candidates targeting novel biology for the treatment of fibrotic diseases. Bayer’s library of approximately 500,000 compounds has been onboarded to Recursion and high throughput discovery screens have been initiated for two of the approximately 10 programs.
Inferential Search : In less than 9 months, Recursion’s transition to inferential search, which leverages its massive dataset for drug discovery, has yielded 18 programs in the area of oncology. This work has been led by Ron Alfa M.D., Ph.D., as Senior Vice President of Translational Medicine at Recursion. As a result of this success, Recursion is expanding its inferential search capabilities into neuroscience as well as inflammation and immunology.
Platform
Orthogonomics : We have made meaningful progress in both transcriptomics and proteomics at Recursion. Our transcriptomics team has standardized our protocol for extracting transcriptomic signals from experiments. Our proteomics team has been optimizing its ability to survey thousands of proteins to identify biomarkers with significant changes in level due to perturbation treatments. These technologies are now being applied to over a dozen early and late discovery programs at Recursion to quickly provide target-agnostic orthogonal validation data.
Digital Chemistry : We launched our digital chemistry-based target prediction tool which identifies potential targets for small molecules by comparing their structural fingerprints to well-annotated small molecules. This tool augments our mechanism of action deconvolution by taking chemical structure into account.
BioHive-1 : The company purchased, built, and activated BioHive-1, a purpose-built supercomputer that we believe is the 58th most powerful in the world and the most powerful supercomputer dedicated wholly to drug discovery for a single company.
Facilities : We initiated two facility expansions in Salt Lake City. First, we are expanding our current headquarters to add laboratory space that will support our plans for additional technology and future partnerships. Second, we are establishing a nearby CMC site for work in process chemistry, analytical chemistry, and drug substance production.
First Quarter 2021 Financial Results

Cash Position : Cash, cash equivalents and marketable securities were $214.1 million as of March 31, 2021 and do not include net proceeds from the company’s April 2021 IPO of $462.6 million.
Revenue : Total revenue, consisting primarily of revenue from collaborative agreements, was $2.6 million for the first quarter of 2021, compared to $60 thousand for the first quarter of 2020. The increase was primarily due to progress in our collaboration agreement with Bayer.
Research and Development Expenses : Research and development expenses were $24.1 million for the first quarter of 2021, compared to $12.8 million for the first quarter of 2020. The increase in research and development expenses over this period was primarily due to an increased number of experiments screened on the platform, an increased number of preclinical assets being validated, and the development of clinical-stage assets.
General and Administrative Expenses : General and administrative expenses were $8.9 million for the first quarter of 2021, compared to $5.6 million for the first quarter of 2020. The increase in general and administrative expenses was due to growth in size of the company’s operations including an increase in salaries and wages of $1.2 million, human resources costs, facilities costs, finance costs and other administrative costs associated with operating a growth-stage company.
Net Loss : Net loss was $30.7 million for the first quarter of 2021, compared to a net loss of $18.4 million for the first quarter of 2020.
Other Corporate Updates

IPO : Recursion upsized and completed its initial public offering in April 2021, raising gross and net proceeds of $501.8 million and $462.6 million, respectively.
Company Management : Since the end of 2020, Recursion has made several executive and board appointments aimed at further supporting the advancement of its pipeline and expanding its business operations. These appointments include naming R. Martin Chavez, Ph.D. as Chairman of our Board of Directors, Ramona Doyle, M.D. as Chief Medical Officer, and Louisa Daniels, J.D. as Chief Legal Officer and General Counsel. Additionally, Ron Alfa M.D., Ph.D. has been promoted to Senior Vice President of Research to lead the rapid creation and advancement of new early discovery programs based on our inferential search platform, and Sharath Hegde Ph.D. has resigned from his role as the company’s Chief Scientific Officer. Dr. Alfa will assume the responsibilities of Dr. Hegde. We thank Dr. Hegde for his substantial contributions to Recursion, which include accelerating our work with new chemical entities and curating our rapid repurposing library.

On May 12, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that interim data from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for high-risk B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL") has been selected for an e-poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress ("EHA2021") (Press release, Mustang Bio, MAY 12, 2021, View Source [SID1234579818]). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Research Center ("Fred Hutch").

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In the abstract posted today on the EHA (Free EHA Whitepaper)2021 website, Fred Hutch reported on 12 patients treated with MB-106, which underwent a major cell manufacturing modification after treating the first 7 patients as previously reported at the 62nd Annual American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in 2020. The 12 patients treated under the new manufacturing process were treated at dose levels ("DL") ranging from 3.3×105 to 1×107 CAR T cells/kg, and clinical responses were observed at all DLs with no dose-limiting toxicities. Cytokine release syndrome occurred in 3 patients (25%): 2 patients with grade 1 and 1 patient with grade 2. Only 1 patient required tocilizumab and dexamethasone, and no immune effector cell-associated neurotoxicity syndrome of any grade was observed. Overall response rate ("ORR") was 92% (11/12) with a complete response ("CR") rate of 58% (7/12). In 9 patients with follicular lymphoma, ORR and CR were 89% (8/9) and 67% (6/9), respectively. The patient with CLL had a PET-negative CR and undetectable measurable residual disease in peripheral blood and bone marrow by flow cytometry (10-4) (uMRD4) on day 28. Among patients who received the highest two dose levels, DL3 (3.3×106 CAR T cells/kg; n=4) and DL4 (1×107 CAR T cells/kg; n=1), CR rate was 100% (5/5). All 7 patients who achieved a CR remain in remission at a median follow-up of 4 months. CAR T expansion was robust, with median peak blood levels of CAR+ T cells of 122 CAR+ cells/μl (range 0.27-2024), corresponding to 19% (range 0.15 – 65%) of all CD3+ cells. Updated data will be presented at EHA (Free EHA Whitepaper)2021.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We are pleased that Fred Hutch will present interim data from the ongoing Phase 1/2 trial of MB-106 at EHA (Free EHA Whitepaper)2021. The data thus far indicate that MB-106 has a highly favorable safety profile at all dose levels when compared to commercially available CAR T cell therapies targeting CD19. We look forward to the continued progression of this CD20-targeted CAR T cell therapy program for patients with relapsed or refractory B-cell non-Hodgkin lymphomas and CLL."

Details of the MB-106 e-poster presentation are as follows:

Topic: 25. Gene therapy, cellular immunotherapy and vaccination – Clinical
Abstract Code: EP731
Title: Immunotherapy Using a 3rd Generation CD20 Targeted CAR T-Cell (MB-106) for Treatment of B-Cell Non-Hodgkin Lymphoma (B-NHL) and Chronic Lymphocytic Leukemia (CLL)
Presenter: Mazyar Shadman, M.D., M.P.H., Fred Hutchinson Cancer Research Center, Seattle, WA, USA
Date and Time: All e-posters will be available for viewing on June 11, 2021 at 09:00 Central European Summer Time (CEST) / 3 a.m. ET

A copy of the abstract can be viewed online through the EHA (Free EHA Whitepaper)2021 website at View Source