On March 23, 2022 Exscientia plc (Nasdaq: EXAI) reported that (Press release, Exscientia, MAR 23, 2022, View Source [SID1234610838])

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Recent advancements in the Company’s pipeline, collaborations and operations as well as financial results for the fourth quarter and full year 2021 are summarised below. In addition, Exscientia will host a conference call on Thursday, March 24 at 12:30 p.m. GMT / 8:30 a.m. ET to provide an overview of the company’s end-to-end technology platform and progress towards automating drug discovery and development.

Recent Highlights

Progressed pipeline led by significant collaborations, expansion, and programme advancement

-Year-over-year pipeline progress with 11 new programmes added, including advancement of three programmes into IND-enabling studies and two programmes to late discovery
-Partnered programmes
oEstablished collaboration with Sanofi in January 2022 focused on development of up to 15 novel small molecule candidates across oncology and immunology, received $100 million upfront cash payment with the potential of $5.2 billion in total milestones plus tiered royalties; several targets have been identified since signing
oCommenced eighth drug discovery project with Bristol Myers Squibb (BMS) in early 2022 for programme against an undisclosed oncology target
oAchieved preclinical proof of concept milestone for a target within Bayer collaboration supporting advancement into late discovery
-Co-owned programmes
oEntered into IND-enabling studies for GTAEXS-617, a CDK7 inhibitor co-owned with GT Apeiron; IND/CTA submission expected by year-end 2022; additional translational data in several tumour types to be presented at the April 2022 American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting and throughout 2022
oOncology target selected with EQRx, the third in the multi-target collaboration
-Wholly and majority owned programmes
oPhase 1 healthy volunteer top-line data for EXS-21546, A2a antagonist in high adenosine signature cancers, on track for the first half of 2022; translational research on identifying adenosine specific gene signatures for patient selection to be presented at AACR (Free AACR Whitepaper) 2022
oWith support from the Bill & Melinda Gates Foundation, Exscientia has designed an Mpro molecule with pan-coronavirus activity demonstrating 200x greater potency in vitro than a commercially available oral COVID-19 antiviral; ongoing compound development with candidate nomination anticipated in the second half of 2022

Balanced business model has generated meaningful cash flows and strong balance sheet

-$85.3 million cash flow from collaborations in full-year 2021, ending 2021 with $758.9 million in cash and cash equivalents
-2021 net cash outflows from operations of approximately $9.0 million
-$100 million upfront payment from Sanofi collaboration signed in January 2022

Peer-reviewed publications and upcoming scientific meeting presence showcase impact of translational research, clinical impact of precision medicine platform and differentiated technology platform

-Three abstracts accepted for poster presentation at AACR (Free AACR Whitepaper) 2022 highlight the potential of Exscientia’s precision medicine platform and AI capabilities in designing optimised molecules, improving translational research and identifying novel pathways
-EXALT-1 clinical trial results published in Cancer Discovery demonstrate the benefit of the first AI-supported functional precision medicine platform to guide treatment selection and improve outcomes in patients with advanced haematological cancers
-Select peer-reviewed publications describe recent advancements in Exscientia’s technology platform
-Fragment Hotspot Mapping to Identify Selectivity-Determining Regions Between Related Proteins describes a computational method to map protein binding pockets and identify critical regions that can be exploited to generate selective molecules
oDeep generative design with 3D pharmacophoric constraints demonstrates the potential of a new method (DEVELOP) to utilise 3D representations of molecules and generate compounds with improved properties over previous methods
oGenerating property-matched decoy molecules using deep learning showcases a deep learning method for generating molecules to validate and improve virtual screening methods

Strengthened team to position Exscientia for future growth

-Richard Law, D. Phil, has been promoted to Chief Business Officer, effective March 4, with continued focus on driving impactful collaborations across our business models
-Professor Charlotte Deane, Ph.D, joined as Chief Scientist of Biologics AI, from the University of Oxford, focused on the application of AI, machine learning, and the design of protein structures in the discovery and develpment of novel drug candidates
-Significant progress in building out clinical organisation, including clinical operations, to prepare for pipeline expansion and future clinical trials
-Experienced drug discovery team established in Boston, Massachusetts, to focus on wholly owned pipeline

"2021 was a transformational year for Exscientia as we further validated and built-out our differentiated end-to-end AI platform. We executed across our business as shown by new and expanded collaborations, growing our pipeline with eleven new programmes. We significantly expanded our global operations and scaled our capacity. On top of these achievements, we also completed our upsized IPO on Nasdaq," said Andrew Hopkins, DPhil., Exscientia’s founder and CEO. "As we look to the future, we believe we are reaching a tipping point for realising the full potential of AI-driven drug creation. We’ve already seen historic investment across the industry. Exscientia has achieved many of the "firsts" in this field and is well-positioned to lead the way. The sheer scale that our AI systems lend to scientific discovery make it possible to keep pace with breaking science in a way never before seen. This year we are focused on bringing together the team we have built and the technologies we have invented to advance new medicine programmes toward the clinic, working with our pharma and biopharma collaborators toward audacious goals to tackle rare diseases and solve persistent scientific challenges, and to continue building out our one-of-a-kind precision medicine platform to bring truly personalised medicine to patients."

Investor Call and Webcast Information

Exscientia will host a conference call on March 24 at 12:30 p.m. GMT / 8:30 a.m. ET. A webcast of the live call can be accessed by visiting the "Investors and Media" section of the Company’s website at investors.exscientia.ai. Alternatively, the live conference call can be accessed by dialling +1 (888) 330 3292 (U.S.), +44 203 433 3846 (U.K.), +1 (646) 960 0857 (International) and entering the conference ID: 8333895. A replay will be available for 90 days under "Events and Presentations" in the "Investors and Media" section of the Exscientia website.

Fourth Quarter and Full Year 2021 Financial Results

For the convenience of the reader, the Company has translated pound sterling amounts to U.S. dollars at the rate of £1.000 to $1.350, which was the noon buying rate of the Federal Reserve Bank of New York on December 31, 2021.

Revenue: Revenue for the full year 2021, was $36.9 million, an increase of $23.9 million compared to the full year 2020, primarily due to the achievement of the opt-in milestone on the first candidate in-licensed under Exscientia’s collaboration with BMS.

R&D and cost of drug discovery: Due to various collaboration structures, R&D expenses may be included under multiple accounting line items. The tables below show how these expenses are separated across the accounting categories.

On March 23, 2022 Ichnos Sciences Inc., a global biotechnology company developing novel multispecific antibodies for the treatment of cancer using the proprietary BEAT platform1, reported that preclinical data for ISB 1442, a first-in-class 2+1 biparatopic bispecific antibody that targets both CD38 and CD47, will be presented at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, to be held in New Orleans from April 8-13, 2022 (Press release, Ichnos Sciences, MAR 23, 2022, View Source [SID1234610703]). ISB 1442 is being investigated as a treatment for relapsed/refractory multiple myeloma, T-cell acute lymphoblastic leukemia and acute myeloid leukemia.

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"The data being presented at AACR (Free AACR Whitepaper) highlight the potential for ISB 1442, our first-in-class biparatopic CD38 x CD47 bispecific antibody, in multiple myeloma," said Stefano Sammicheli, Ph.D., Director of Innate Cell Engagers, at Ichnos Sciences. "This compound has shown higher potency and greater inhibition of tumor growth relative to daratumumab in both in vitro and in vivo models, suggesting that ISB 1442 may overcome common mechanisms of resistance to other CD38 targeted therapies, which is coupled with low potential for on-target off-tumor effects seen with other CD47 directed treatments."

"We are excited to have the opportunity to present at AACR (Free AACR Whitepaper), and to share ISB 1442 data that support our plan to move this drug into a Phase 1 study in the middle of this year," said Cyril Konto, M.D., President and Chief Executive Officer of Ichnos Sciences. "Advancing ISB 1442 is particularly important to Ichnos because it is built using BEAT 2.0, which is among the most advanced antibody engineering platforms and the basis of our discovery efforts for novel multispecifics to treat hematologic malignancies and solid tumors."

Ichnos Sciences continues to advance its pipeline of agents based on the proprietary BEAT technology platform. Using this platform, Ichnos Sciences is exploring the full design space for treating cancer and engineering multispecific antibodies capable of simultaneously engaging tumor and immune cells.

Details for the poster presentation are shared below:

Session PO.IM02.10 – Therapeutic Antibodies 2
April 12, 2022, 9:00 AM – 12:30 PM

2903 / 18 – ISB 1442, a first-in-class CD38 and CD47 bispecific antibody innate cell modulator for the treatment of CD38+ malignancies

Posters will be available on-demand on the AACR (Free AACR Whitepaper) website at www.aacr.org beginning at the start of the poster session and the abstract is available to view here.

On March 23, 2022 Novartis reported that the US Food and Drug Administration (FDA) approved Pluvicto (lutetium Lu 177 vipivotide tetraxetan) (formerly referred to as 177Lu-PSMA-617) for the treatment of adult patients with a certain type of advanced cancer called prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer (PSMA-positive mCRPC) that has spread to other parts of the body (metastatic)1 (Press release, Novartis, MAR 23, 2022, View Source [SID1234610766]). These patients have already been treated with other anti-cancer treatments (androgen receptor pathway inhibition and taxane-based chemotherapy)1.

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Experience the interactive Multichannel News Release here: View Source

"The approval of Pluvicto is an important clinical advancement for people with progressing mCRPC, as it can significantly improve survival rates for those who have limited treatment options," said Oliver Sartor, MD, Medical Director at Tulane Cancer Center. "Pluvicto is a step forward in the evolution of precision medicine for prostate cancer."

Pluvicto is the first FDA-approved targeted radioligand therapy (RLT) for eligible patients with mCRPC that combines a targeting compound (ligand) with a therapeutic radioisotope (a radioactive particle)1. Pluvicto is expected to be available to physicians and patients within weeks.

The FDA has also approved Locametz (kit for the preparation of gallium Ga 68 gozetotide injection)2. After radiolabeling, this imaging agent may be used to identify PSMA-positive lesions in adult patients with mCRPC through a positron emission tomography (PET) scan2. Gallium-68 labeled Locametz can identify tumor lesions expressing the PSMA biomarker and locate where in the body tumors may have spread (eg, in soft tissue, lymph nodes, or bone), identifying patients eligible for targeted treatment with Pluvicto1,2. PSMA is highly expressed in more than 80 percent of patients with prostate cancer, making it an important phenotypic biomarker for assessing the progression of metastatic prostate cancer10. Locametz is expected to be available to physicians and patients within weeks.

"With our unique strategy to tackle cancer by leveraging four therapeutic platforms, I am thrilled that with Pluvicto, we are bringing the targeted RLT platform to bear for treating eligible patients with mCRPC," said Susanne Schaffert, PhD, President, Novartis Oncology. "Today’s approval builds upon our history in prostate cancer, a devastating disease where we believe our innovation can make a meaningful difference to patients."

FDA approval of Pluvicto is based on the results of the Phase III VISION trial which demonstrated that PSMA-positive mCRPC patients previously treated with androgen receptor (AR) pathway inhibition and taxane-based chemotherapy who received Pluvicto plus standard of care (SOC) had improved overall survival compared to SOC alone1. Participants treated with Pluvicto plus SOC had a 38% reduction in risk of death and a statistically significant reduction in the risk of radiographic disease progression or death (rPFS) compared to SOC alone1. Interpretation of the magnitude of the rPFS effect was limited due to a high degree of censoring from early drop out in the control arm1.

In addition, about a third (30%) of patients with evaluable disease at baseline demonstrated an overall response (per RECIST 1.1) with Pluvicto plus SOC, compared to 2% in the SOC alone arm1. The most common adverse events (all grades) in the Pluvicto arm of the study were fatigue (43%), dry mouth (39%), nausea (35%), anemia (low red blood cell counts) (32%), decreased appetite (21%), and constipation (20%)1.

"Prostate cancer is the second leading cause of cancer-related death in Americans with a prostate gland13. Although the treatment landscape for mCRPC continues to evolve, there is a high unmet need for additional precision medicine treatment options to improve outcomes for these patients," said Jamie Bearse, CEO and President at ZERO – The End of Prostate Cancer. "The approval of Pluvicto offers new hope to the mCRPC community."

Pluvicto and Locametz are registered products of Advanced Accelerator Applications, the radioligand business of Novartis, approved in the United States for physicians to prescribe to appropriate patients. Additional safety details for Pluvicto and Locametz, and full Prescribing Information can be found on the Novartis website.

About Pluvicto
Pluvicto (lutetium Lu 177 vipivotide tetraxetan) is indicated for the treatment of adult patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) who have already been treated with other anticancer treatments (androgen receptor pathway inhibition (ARPI) and taxane-based chemotherapy)1. It is a type of precision cancer treatment combining a targeting compound (ligand) with a therapeutic radioisotope (a radioactive particle)1. After administration into the bloodstream, Pluvicto binds to target cells, including prostate cancer cells that express PSMA, a transmembrane protein1. Once bound, energy emissions from the radioisotope damage the target cells and nearby cells disrupting their ability to replicate and/or triggering cell death1.

Novartis has submitted marketing authorization for Pluvicto to the European Medicines Agency and other health authorities.

About Locametz
Locametz (gallium Ga 68 gozetotide), diagnostic kit for radiopharmaceutical injectable preparation is indicated for positron emission tomography (PET) of PSMA-positive lesions with prostate cancer with suspected metastasis who are candidates for initial definitive therapy; with suspected recurrence based on elevated serum prostate-specific antigen (PSA) level; and for selection of patients with metastatic prostate cancer, for whom lutetium Lu 177 vipivotide tetraxetan PSMA-directed therapy is indicated2.

Novartis has submitted marketing authorization for Locametz to the European Medicines Agency and other health authorities.

About VISION
VISION is an international, prospective, randomized, open-label, multicenter, phase III study that assessed the efficacy and safety of Pluvicto (lutetium Lu 177 vipivotide tetraxetan) (7.4 GBq administered by IV infusion every 6 weeks for a maximum of 6 cycles) plus investigator-chosen standard of care (SOC) in the investigational arm, versus SOC in the control arm1. Patients with PSMA PET-scan positive mCRPC who have received androgen receptor (AR) pathway inhibition and taxane-based chemotherapy were randomized in a 2:1 ratio in favor of the investigational arm1. The alternate primary endpoints were rPFS and OS1. The study enrolled 831 patients1.

About Phenotypic Precision Medicine in Advanced Prostate Cancer
Despite advances in prostate cancer care, there is a high unmet need for new targeted treatment options to improve outcomes for patients with mCRPC. More than 80% of patients with prostate cancer highly express a phenotypic biomarker9 called prostate specific membrane antigen (PSMA)4-6,9,10, making it a promising diagnostic (through positron emission tomography (PET) scan imaging) and therapeutic target for radioligand therapy10. This differs from ‘genotypic’ precision medicine which targets specific genetic alterations in cancer cells7.

Novartis and Prostate Cancer
With more 1.4 million new cases and 375,000 deaths in 2020 alone, prostate cancer is the most frequently diagnosed cancer in 112 countries—more than half the world12.

At Novartis, we are harnessing the innovation of our world-class scientists, strategic partnerships, and one of the industry’s most competitive pipelines to explore the potential of new, targeted therapies and precision medicine platforms to address the greatest unmet needs in prostate cancer.

Through the bold science of targeted therapies, our goal is to reduce the global disease burden, extend the lives of patients with prostate cancer, and elevate current standards of care.

Pluvicto Indication
PLUVICTO (lutetium Lu 177 vipivotide tetraxetan) is a radiopharmaceutical used to treat adults with an advanced cancer called prostate-specific membrane antigen–­­­­positive metastatic castration-resistant prostate cancer (PSMA-positive mCRPC) that has spread to other parts of the body (metastatic), and has already been treated with other anticancer treatments.

Pluvicto Important Safety Information
Use of PLUVICTO involves exposure to radioactivity. Long-term, accruing radiation exposure is associated with increased risk for cancer. To minimize radiation exposure to others following administration of PLUVICTO, patients are advised to limit close contact (less than 3 feet) with household contacts for 2 days or with children and pregnant women for 7 days, to refrain from sexual activity for 7 days, and to sleep in a separate bedroom from household contacts for 3 days, from children for 7 days, or from pregnant women for 15 days.

PLUVICTO may cause low level of blood cell counts. Patients should tell their doctor right away if they develop any new or worsening symptoms, including tiredness or weakness, pale skin, shortness of breath, bleeding or bruising more easily than normal or difficulty to stop bleeding, or frequent infections with signs such as fever, chills, sore throat, or mouth ulcers. PLUVICTO may also cause problems with kidneys. Patients should tell their doctor right away if they develop any new or worsening symptoms, including passing urine less often or passing much smaller amounts of urine than usual.

Before receiving PLUVICTO, patients should tell their doctor if they have low level of blood cell counts (hemoglobin, white blood cell count, absolute neutrophil count, platelet count); if they have or have had tiredness, weakness, pale skin, shortness of breath, bleeding or bruising more easily than normal or difficulty stopping bleeding, or frequent infections with signs such as fever, chills, sore throat, or mouth ulcers (possible signs of myelosuppression); if they have or have had kidney problems; if they have or have had any other type of cancer or treatment for cancer, as PLUVICTO contributes to long-term cumulative radiation exposure; and if they are sexually active, as all radiopharmaceuticals, including PLUVICTO, have the potential to cause harm to an unborn baby. Patients should use effective contraception for intercourse during treatment with PLUVICTO and for 14 weeks after the last dose. PLUVICTO may cause temporary or permanent infertility.

Before administration of PLUVICTO patients should drink plenty of water in order to urinate as often as possible during the first hours after administration.

The most common side effects of PLUVICTO include tiredness, dry mouth, nausea, low red blood cell count, loss of appetite, changes in bowel movements (constipation or diarrhea), vomiting, low blood platelet count, urinary tract infection, weight loss, and abdominal pain.

Please see full Prescribing Information for PLUVICTO at View Source

Locametz Indication
LOCAMETZ (kit for the preparation of gallium Ga 68 gozetotide injection), after radiolabeling with gallium-68, is indicated for positron emission tomography (PET) of prostate-specific membrane antigen (PSMA)-positive lesions in men with prostate cancer:

with suspected metastasis who are candidates for initial definitive therapy
with suspected recurrence based on elevated serum prostate-specific antigen (PSA) level
for selection of patients with metastatic prostate cancer for whom lutetium Lu 177 vipivotide tetraxetan PSMA-directed therapy is indicated
Locametz Important Safety Information
LOCAMETZ PET images can be misinterpreted. Gallium Ga 68 gozetotide may be taken up into other types of cancerous and noncancerous tissue. Corroborating approaches, which may include histopathology, are recommended.

Gallium Ga 68 gozetotide contributes to a patient’s long-term cumulative radiation exposure, which is associated with an increased risk of cancer. Safe handling and preparation procedures must be ensured to protect patients and health care workers from unintentional radiation exposure. Patients should be well hydrated prior to administration and urinate immediately prior to and frequently during the first hours after image acquisition to reduce radiation exposure.

Adverse reactions that occurred at a rate of 0.5% or greater in the VISION study were fatigue, nausea, constipation, and vomiting; at a rate less than 0.5%, diarrhea, dry mouth, injection site reactions, and chills.

Please see full Prescribing Information for LOCAMETZ at View Source

Patient Access and Support
Novartis is committed to helping ensure that our medicines are accessible to as many patients as possible. With the approval of Pluvicto in the United States, we offer support and services to address a range of needs through AAA PatientCONNECT. AAA PatientCONNECT is a support center staffed by dedicated case managers who can help eligible patients throughout their treatment journey to start and stay on treatment. Patients or providers can call 1-844-638-7222 or visit AAApatientconnect.com to enroll and learn more about AAA PatientCONNECT.

On March 23, 2022 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 financial results and recent corporate highlights for the full year ended December 31, 2021 (Press release, Mustang Bio, MAR 23, 2022, View Source [SID1234610839]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "In 2021, we continued to make great strides in advancing our cell and gene therapy programs. Notably, the U.S. Food and Drug Administration ("FDA") approved two Mustang Investigational New Drug ("IND") applications, the first to initiate a pivotal multicenter Phase 2 clinical trial to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with X-linked severe combined immunodeficiency ("XSCID"), and the second to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted autologous CAR T cell therapy for relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL"). Additionally, we received a $2 million grant from the National Cancer Institute to partially fund our MB-106 Phase 2 clinical trial. Interim data from the ongoing Phase 1/2 clinical trial of MB-106 presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting ("ASH2021") by our collaborators at Fred Hutchinson Cancer Research Center ("Fred Hutch") showed a 95% overall response rate, 65% complete response rate and favorable safety profile. We also expanded our product portfolio by executing an exclusive license agreement with Leiden University Medical Centre for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID and enhanced our CAR T program with an exclusive license agreement with Mayo Clinic for a novel technology to create in vivo CAR T cells that has the potential to transform the administration of CAR T therapies and be used as an off-the-shelf therapy."

Dr. Litchman continued, "We anticipate another productive year in 2022, with several Mustang IND clinical trial initiations and data updates from our ongoing clinical programs at prominent medical conferences. In February, City of Hope presented Phase 1 data on MB-105, our prostate stem cell antigen ("PSCA") CAR T-cell therapy for the treatment of PSCA-positive metastatic castration-resistant prostate cancer ("mCRPC") at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Genitourinary ("GU") Cancers Symposium that demonstrated its potential to treat patients with mCRPC. In April, our collaborators at Fred Hutch will present interim Phase 1/2 clinical trial data on MB-106 at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy ("ASTCT") and Center for International Blood & Marrow Transplant Research ("CIBMTR") which show its compelling clinical activity and favorable safety profile. In the first half of 2022 we anticipate enrolling the first patient in Mustang’s multicenter Phase 1/2 clinical trial to evaluate MB-106, and in the second half of 2022 we expect to enroll the first patient in Mustang’s multicenter pivotal Phase 2 clinical trial to evaluate MB-107. We also plan to advance additional Mustang clinical candidates, including filing an IND for MB-109 (MB-101 autologous IL13Rα2-directed CAR T cells + MB-108 oncolytic virus) for the treatment of glioblastoma. With this steady progress across our clinical programs, and with our robust pipeline and strong cash position following completion of the debt facility with Runway Growth Capital, Mustang is poised to continue its success building an integrated cell and gene therapy company."

Financial Results:

·As of December 31, 2021, Mustang’s cash and cash equivalents and restricted cash totaled $110.6 million, compared to $98.8 million as of December 31, 2020, an increase of $11.8 million year-to-date.
·Research and development expenses were $49.9 million for the year ended December 31, 2021. This compares to $37.2 million for 2020. Non-cash, stock-based compensation expenses included in research and development were $2.3 million for the year ended December 31, 2021, compared to $1.4 million for 2020.
·Research and development expenses from license acquisitions totaled $5.8 million for the year ended December 31, 2021, compared to $10.1 million for 2020. Non-cash, stock-based compensation expenses included in research and development – licenses acquired were $4.2 million for the year ended December 31, 2021, compared to $7.6 million for 2020.
·General and administrative expenses were $11.0 million for the year ended December 31, 2021. This compares to $9.5 million for 2020. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.9 million for the year ended December 31, 2021, compared to $4.0 million for 2020.
·Net loss attributable to common stockholders was $66.4 million, or $0.76 per share, for the year ended December 31, 2021, compared to a net loss attributable to common stockholders of $60.0 million, or $1.14 per share, for 2020.

2021 and Recent Corporate Highlights:

·In February 2021, Mustang announced encouraging MB-107 and MB-207 clinical updates from its X-linked severe combined immunodeficiency ("XSCID") investigator IND trials, as well as additional consistent safety and efficacy data. In the second half of 2022, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang Bio’s IND to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with XSCID, also known as bubble boy disease. Mustang filed an IND application in December 2021 for its pivotal multicenter Phase 2 clinical trial of MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with a hematopoietic stem cell transplantation ("HSCT") and for whom re-treatment is indicated. The trial is currently on hold pending CMC clearance from FDA and, based on feedback from the Agency, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial in the first quarter of 2023.
·In May 2021, Mustang announced that the FDA approved its IND application to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted autologous CAR T cell therapy for relapsed or refractory B-NHL and CLL. Mustang intends to dose the first patient in that trial in the first half of 2022.
·Also in May 2021, Mustang announced that the first patient was dosed at City of Hope in a clinical trial to establish the safety and feasibility of administering MB-101 (autologous IL13Rα2-directed CAR T cells) to patients with leptomeningeal brain tumors (e.g., glioblastoma, ependymoma or medulloblastoma).
·In June 2021, Mustang announced that MB-106 CD20-targeted CAR T cell therapy data were presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress. Dr. Mazyar Shadman of Fred Hutch presented updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL, which showed a favorable safety profile and compelling clinical activity, with a 93% overall response rate and 67% complete response rate in patients treated with a modified cell manufacturing process.
·Also in June 2021, Mustang hosted a key opinion leader webinar featuring a presentation from Dr. Shadman, who discussed interim results from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for B-NHL and CLL.
·Additionally in June 2021, Mustang announced that it was one of 28 recipients awarded tax incentives from the Massachusetts Life Sciences Center. The $300,000 tax incentive amount that Mustang was awarded was based on a hiring commitment of 20 net new full-time equivalent employees for calendar year 2021 and retaining that headcount level through 2025.
·In August 2021, Mustang announced that the European Medicines Agency granted Priority Medicines ("PRIME") designation to MB-107, a lentiviral gene therapy for the treatment of XSCID in newly diagnosed infants.
·Also in August 2021, Mustang announced an exclusive license agreement with Mayo Clinic for a novel technology to create in vivo CAR T cells that may be able to transform the administration of CAR T therapies and has the potential to be used as an off-the-shelf therapy.
·In October 2021, Christine Brown, Ph.D., Deputy Director, T Cell Therapeutics Research Laboratory and The Heritage Provider Network Professor in Immunotherapy at City of Hope, presented updated Phase 1 clinical data regarding MB-101 (IL13Rα2-targeted CAR T cells) for the treatment of glioblastoma at two scientific conferences, the First Annual Conference on CNS Clinical Trials, co-sponsored by the Society for Neuro-Oncology and American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Special Conference: Brain Cancer.

·In November 2021, Mustang announced the execution of an exclusive license agreement with Leiden University Medical Centre for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.
·Also in November 2021, Mustang announced that it was awarded a grant of approximately $2 million from the National Cancer Institute of the National Institutes of Health. This two-year award will partially fund the Mustang-sponsored multicenter trial to assess the safety, tolerability and efficacy of MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL or CLL.
·In December 2021, Mustang announced that MB-106 data were presented at ASH (Free ASH Whitepaper)2021. Dr. Shadman of Fred Hutch presented updated interim data showing a 95% overall response rate, 65% complete response rate and favorable safety profile from the ongoing Phase 1/2 clinical trial for B-NHL and CLL. A copy of the poster can be viewed online here.
·Also in December 2021, Mustang hosted a key opinion leader webinar featuring a presentation from Dr. Shadman, who discussed interim results from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted, autologous CAR T cell therapy to treat B-NHL and CLL. A replay of the webinar can be found here.
·Additionally in December 2021, Mustang was added to the NASDAQ Biotechnology Index (NASDAQ: NBI).
· In January 2022, Mustang announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL and CLL, were selected for a poster presentation at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the ASTCT and CIBMTR, rescheduled to take place April 23-26, 2022, in Salt Lake City, Utah. A copy of the abstract can be viewed on the meeting website here.
· In February 2022, Mustang was selected as the Bronze winner for the Central region in the Eighteenth Annual Team Massachusetts Economic Impact Awards presented by MassEcon. The award winners will be honored at Gillette Stadium on April 7, 2022.
·Also in February 2022, Phase 1 data on MB-105, a PSCA-targeted CAR T cell therapy administered systemically to patients with PSCA-positive mCRPC, were presented by City of Hope at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium. The data results indicated that PSCA-CAR T-cell therapy is feasible in patients with mCRPC with dose limiting toxicity ("DLT") of cystitis and show preliminary anti-tumor effect at a dose of 100 million cells plus lymphodepletion. It was concluded that escalation up to the next dose level of 300 million cells can proceed in the trial.
·In March 2022, Mustang completed a $75 million long-term debt facility with Runway Growth Capital LLC ("Runway").
·Also in March 2022, Mustang announced that an abstract reporting on Phase 1 trials being conducted at the University of Alabama at Birmingham (UAB) and City of Hope of Mustang Bio’s exclusively licensed oncolytic viral and CAR T-cell therapies for the treatment of patients with glioblastoma (GBM) was selected as a late-breaking poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, taking place April 8 – 13, 2022, in New Orleans, Louisiana. The abstract will also be published in the online Proceedings of the AACR (Free AACR Whitepaper).

On March 23, 2022 iTeos Therapeutics, Inc. (Nasdaq: ITOS), a clinical-stage biopharmaceutical company pioneering the discovery and development of a new generation of highly differentiated immuno-oncology therapeutics for patients, reported financial results for the fourth quarter and full year ended December 31, 2021 and provided recent corporate highlights (Press release, iTeos Therapeutics, MAR 23, 2022, View Source [SID1234610910]).

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"In 2021, we laid a strong foundation for both of our differentiated clinical-stage immunotherapy programs EOS-448, our FcγR-engaging anti-TIGIT antibody, and inupadenant, our adenosine A2A receptor antagonist. The data we shared over the course of the year validates our excitement around both candidates as potentially differentiated therapies capable of harnessing the immune system to improve outcomes for patients with several types of advanced cancers," said Michel Detheux, Ph.D., president and chief executive officer of iTeos. "Anticipating the advancement of both EOS-448 and inupadenant from early to late-stage clinical development with novel combinations, we have built a global team to fuel the execution of our robust clinical development plans throughout 2022. This year is expected to be critical in terms of data generation for the TIGIT and adenosine fields, and we’re excited to play a key role in growing the body of evidence that will inform how these targets can be harnessed for patients as safely and quickly as possible."

Program Highlights

EOS-448: IgG1 anti-TIGIT monoclonal antibody designed to engage the Fc gamma receptor (FcγR) and to enhance the anti-tumor response through multifaceted mechanisms.

In collaboration with GSK, iTeos is initiating various combinations to advance this next generation immuno-oncology agent:
Began dosing in a Phase 1b clinical trial in patients with non-small cell lung cancer (NSCLC) assessing the doublet of GSK’s anti-PD-1 (Jemperli) with EOS-448.
Planning three registration-directed trials combining EOS-448 with Jemperli in 1L NSCLC PDL1 high, head and neck squamous cell carcinoma (HNSCC) and a third indication targeting an additional immune-responsive tumor.
Initiating Phase 1b trials with novel triplets, including Jemperli with EOS-448 and inupadenant in patients with advanced solid tumors and EOS-448 with Jemperli and GSK’s investigational anti-CD96 antibody in patients with NSCLC.
iTeos is evaluating the doublets of pembrolizumab with EOS-448 and inupadenant with EOS-448 in patients with solid tumors in an ongoing Phase 1 trial.
In December 2021, favorable preclinical data generated in collaboration with Fred Hutchinson Cancer Research Center were presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition. Based on this data, iTeos is advancing an open-label dose-escalation/expansion Phase 1/2 trial of EOS-448 as a monotherapy and in combination with Bristol Myers Squibb’s iberdomide – a novel, potent oral cereblon E3 ligase modulator (CELMoD) with or without dexamethasone, in adults with relapsed or refractory multiple myeloma.
The company will present preclinical and clinical analyses supporting the multifaceted mechanism of action of EOS-448, including data on pharmacodynamics within the tumor microenvironment, as part of a late-breaking poster presentation at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting taking place April 8-13, 2022 in New Orleans, Louisiana.
Inupadenant (EOS-850): Designed as an insurmountable and highly selective small molecule antagonist of the adenosine A2A receptor, the only high-affinity adenosine receptor expressed on different immune cells found in the tumor micro-environment.

iTeos is initiating a randomized Phase 2 trial to evaluate the combination of inupadenant with chemotherapy compared to standard of care in an undisclosed solid tumor indication.
The Company is also evaluating inupadenant in combination with pembrolizumab in PD-1 resistant melanoma in an ongoing Phase 2a trial
iTeos is evaluating patient and indication selection biomarkers in an ongoing Phase 1b/2a trial of inupadenant as a monotherapy in patients with solid tumors.
Preclinical programs: Building on the company’s successful track record of advancing differentiated programs from discovery into the clinic, iTeos continues to progress research programs focused on additional targets that address pathways of immunosuppression. In 2021, iTeos nominated an additional candidate targeting a new mechanism in the adenosine pathway for Investigational New Drug-enabling studies.

Upcoming Events

AACR Annual Meeting, April 8-13, 2022
Late-Breaking Abstract Title: Pharmacodynamic assessment of a-TIGIT mAb EOS-448 highlights multiple FcγR-mediated mode-of-actions in blood and tumor of patients with advanced solid tumors; Wednesday, April 13 from 9:00am – 12:30pm CDT
Session Title: Late Breaking Research: Experimental and Molecular Therapeutics 2
Abstract Number: LB189 / Section 16
Fourth Quarter and Full Year 2021 Financial Results

Cash Position: The Company’s cash and cash equivalent position was $848.5 million as of December 31, 2021, as compared to $336.3 million as of December 31, 2020. Cash balance provides runway into 2026.
Research and Development (R&D) Expenses: R&D expenses were $17.4 million for the quarter and $59.4 million for the full year ended December 31, 2021, as compared to $9.2 million for the fourth quarter and $29.9 million for the full year of 2020. The increase was primarily due to an increase in activities related to clinical trials for EOS-448 and Inupadenant, as well as preclinical programs.
General and Administrative (G&A) Expenses: G&A expenses were $9.6 million for the quarter and $40.5 million for the full year ended December 31, 2021, as compared to $5.7 million for the fourth quarter and $15.3 million for the full year of 2020. The increase was primarily due to an increase in professional fees related to the Company’s collaboration with GSK, in addition to an increase in professional fees associated with the Company’s status as a publicly traded company.
Net Income/Loss: Net income attributable to common shareholders was $184.9 million, or a net income of $5.24 per basic share and $4.88 per diluted share, for the quarter ended December 31, 2021, as compared to a net loss of $14.9 million, or a net loss of $0.43 per basic and diluted share, for the fourth quarter of 2020. Net income was $214.5 million, or a net income of $6.10 per basic share and $5.68 per diluted share, for the year ended December 31, 2021, as compared to a net loss of $43.4 million, or a net loss of $2.88 per basic and diluted share, for the full year of 2020.