AZN AstraZeneca PLC

52.78
-0.61  -1%
Previous Close 53.39
Open 52.87
52 Week Low 36.15
52 Week High 64.94
Market Cap $138,528,022,280
Shares 2,624,630,964
Float 2,624,630,964
Enterprise Value $150,390,917,867
Volume 7,909,753
Av. Daily Volume 4,835,732
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Upcoming Catalysts

Drug Stage Catalyst Date
Roxadustat
Anaemia in Chronic Kidney Disease
PDUFA
PDUFA
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Tagrisso (ADAURA)
Non-small Cell Lung Carcinoma
PDUFA priority review
PDUFA priority review
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Trastuzumab deruxtecan - DESTINY-Gastric01
Gastric cancer
PDUFA priority review
PDUFA priority review
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Imfinzi - PACIFIC-2
Non-small cell lung cancer (NSCLC)
Phase 3
Phase 3
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Imfinzi (PEARL)
Non-small cell lung cancer (NSCLC)
Phase 3
Phase 3
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Calquence (ELEVATE-RR)
Chronic Lymphocytic Leukemia (CLL)
Phase 3
Phase 3
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Lynparza - OlympiA
BRCAm adjuvant breast cancer
Phase 3
Phase 3
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Durvalumab +/- tremelimumab (KESTREL)
Head & neck cancer
Phase 3
Phase 3
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Roxadustat
Myelodysplastic syndromes (MDS)
Phase 3
Phase 3
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Brazikumab
Ulcerative colitis
Phase 2/3
Phase 2/3
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Imfinzi (PACIFIC-5)
Non-small cell lung cancer (NSCLC)
Phase 3
Phase 3
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PT027
Asthma
Phase 3
Phase 3
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Farxiga (Deliver)
Chronic Heart Failure (CHF), preserved ejection fraction (HFpEF)
Phase 3
Phase 3
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Imfinzi + tremelimumab (POSEIDON)
Non-small cell lung cancer (NSCLC)
Phase 3
Phase 3
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Trastuzumab deruxtecan - DESTINY-Breast04
HER low breast cancer
Phase 3
Phase 3
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Trastuzumab deruxtecan - DESTINY-Breast03
Second line HER2+ breast cancer
Phase 3
Phase 3
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Trastuzumab deruxtecan DESTINY-Breast02
Third line HER2+ breast cancer
Phase 3
Phase 3
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Lynparza - PROpel
Castration-resistant prostate cancer -first line
Phase 3
Phase 3
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Imfinzi + tremelimumab - HIMALAYA
Hepatocellular carcinoma (HCC)
Phase 3
Phase 3
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Brazikumab
Crohn's disease
Phase 3
Phase 3
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Imfinzi TOPAZ-1
Biliary-tract cancer
Phase 3
Phase 3
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Durvalumab - EMERALD-1
Locoregional Hepatocellular carcinoma (HCC)
Phase 3
Phase 3
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Imfinzi
Muscle-invasive bladder cancer
Phase 3
Phase 3
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Durvalumab - ADRIATIC
Small cell lung cancer (SCLC) limited disease
Phase 3
Phase 3
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Fasenra MANDARA
Eosinophilic granulomatosis with polyangiitis
Phase 3
Phase 3
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Imfinzi
Non-muscle invasive bladder cancer
Phase 3
Phase 3
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Imfinzi - AEGEAN
Neoadjuvant Non-small cell lung cancer (NSCLC)
Phase 3
Phase 3
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Fasenra
hypereosinophilic syndrome (HES)
Phase 3
Phase 3
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Durvalumab (ADJUVANT)
Adjuvant Non-small cell lung cancer (NSCLC)
Phase 3
Phase 3
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Fasenra
Chronic obstructive pulmonary disease (COPD) and peripheral blood eosinophils
Phase 3
Phase 3
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Imfinzi
Locoregional Hepatocellular carcinoma (HCC)
Phase 3
Phase 3
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Fasenra MESSINA
Eosinophilic oesophagitis
Phase 3
Phase 3
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Imfinzi + tremelimumab (NILE)
Bladder cancer
Phase 3
Phase 3
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Imfinzi CALLA
Cervical cancer
Phase 3
Phase 3
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Imfinzi DuO-O
Ovarian cancer
Phase 3
Phase 3
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Fasenra
Asthma
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
ION449 (AZD8233)
Dyslipidaemia
Phase 2b
Phase 2b
Phase 2b trial initiation announced November 30, 2020.
AZD1222 (ChAdOx1 nCoV-19)
COVID-19 vaccine
Phase 3
Phase 3
Phase 3 initial data met primary endpoint. 70% efficacy (90% and 60% across two dosing regimens).
MEDI0457
HPV-associated squamous cell carcinoma of the head & neck (SCCHN)
Phase 1/2
Phase 1/2
Phase 2 trial ongoing.
Tezepelumab - NAVIGATOR
Asthma
Phase 3
Phase 3
Phase 3 trial met primary endpoint - November 10, 2020.
Brilinta (THALES)
Acute ischaemic stroke
Approved
Approved
FDA approval announced November 6, 2020.
Fasenra OSTRO
Nasal polyps
Phase 3
Phase 3
Phase 3 trial met primary endpoints.
Durvalumab +/- tremelimumab (DANUBE)
Bladder cancer
Phase 3
Phase 3
Phase 3 data released March 6, 2020 did not meet primary endpoint.
Epanova
Hypertriglyceridaemia CVOT
Phase 3
Phase 3
Phase 3 trial to be discontinued due to low likelihood of demonstrating a benefit - January 13, 2020.
Durvalumab +/- tremelimumab (NEPTUNE)
Lung cancer
Phase 3
Phase 3
Phase 3 data released August 21, 2019 did not meet primary endpoint.
Breztri - (ETHOS)
Chronic obstructive pulmonary disease (COPD)
Approved
Approved
FDA approval announced July 24, 2020.
Brilinta (THEMIS)
Type 2 Diabetes / coronary artery disease (CAD)
Approved
Approved
FDA approval announced June 1, 2020.
Lynparza
Castration-Resistant Prostate Cancer
Approved
Approved
FDA Approval announced May 19, 2020.
Lynparza + Avastin- PAOLA-1
First-line ovarian cancer
Approved
Approved
FDA Approval announced May 8, 2020.
Farxiga (Dapa-HF)
Heart failure
Approved
Approved
FDA Approval announced May 5, 2020.
Farxiga (Dapa-CKD)
Chronic Kidney Disease
Phase 3
Phase 3
Phase 3 trial stopped early due to overwhelming efficacy.
Selumetinib
Neurofibromatosis type 1 plexiform neurofibromas
Approved
Approved
FDA Approval announced April 13, 2020.
Imfinzi + tremelimumab (CASPIAN)
Small cell lung cancer (SCLC)
Approved
Approved
FDA approval announced March 30, 2020.
Lynparza + cediranib
Ovarian cancer
Phase 3
Phase 3
Phase 3 trial did not meet primary endpoint - March 12, 2020.
Lynparza (POLO)
Pancreatic cancer
Approved
Approved
FDA Approval announced December 30, 2019.
Enhertu (trastuzumab deruxtecan)
Third line HER2+ breast cancer
Approved
Approved
FDA Approval announced December 20, 2019.
Calquence
Chronic lymphocytic leukaemia
Approved
Approved
FDA Approval announced November 21, 2019.
Anifrolumab
Lupus
Phase 3
Phase 3
Phase 3 TULIP 1 data released August 31, 2018. Primary endpoint not met. TULIP 2 data released August 29, 2019 did meet primary endpoint.
Farxiga - DECLARE
Heart failure in patients with type-2 diabetes
Approved
Approved
FDA Approval announced October 21, 2019.
PT010
Chronic obstructive pulmonary disease (COPD)
CRL
CRL
CRL announced October 1, 2019.
Tagrisso - FLAURA
Non-small cell lung cancer (NSCLC)
Phase 3
Phase 3
Phase 3 final OS data released August 9, 2019. Endpoint met. Presentation at ESMO September 2019 noted OS of 38.6 months versus 31.8 months for comparator arm; PFS HR 0.48.
Calquence
Relapsed/refractory chronic lymphocytic leukaemia
Phase 3
Phase 3
Phase 3 positive interim data released May 7, 2019. Trial to be stopped early.
Selumetinib - ASTRA
Thyroid cancer
Phase 3
Phase 3
Phase 3 trial did not meet primary endpoint - noted July 26, 2018.
Farxiga (Depict)
Type 1 diabetes
CRL
CRL
CRL issued for sNDA announced July 15, 2019.
Durvalumab +/- tremelimumab (EAGLE)
Head & neck cancer
Phase 3
Phase 3
Phase 3 data released December 7, 2018 - primary endpoints not met.
Durvalumab +/- tremelimumab (MYSTIC)
Lung cancer
Phase 3
Phase 3
Phase 3 data released July 27, 2017 - primary endpoint (PFS) not met. Overall survival data also did not meet primary endpoint - November 16, 2018.
Lanabecestat (AZD3293) - AMARANTH
Early Alzheimer's disease
Phase 3
Phase 3
Announced discontinuation of trial due to futility - June 12, 2018.
Lynparza - SOLO 3
Third-line ovarian cancer
Phase 3
Phase 3
Phase 3 data met primary and secondary endpoints. ORR; 72.2% vs 51.4% for chemotherapy. PFS 13.4 months vs 9.2 months for chemo arm (HR 0.62).
Qternmet XR (dapagliflozin, saxagliptin and metformin hydrochloride)
Type 2 diabetes
Approved
Approved
FDA approval announced May 3, 2019.
Lynparza - SOLO 1
First-line ovarian cancer following platinum-based chemotherapy
Approved
Approved
FDA Approval announced December 19, 2018.
Moxetumomab
Cancer - leukaemia
Approved
Approved
FDA approval announced September 13, 2018.
Durvalumab (PACIFIC)
Lung cancer
Approved
Approved
Approval announced February 19, 2018.
Benralizumab - TERRANOVA
COPD
Phase 3
Phase 3
Phase 3 data released May 30, 2018. Primary endpoint not met.
Lokelma (ZS-9)
Hyperkalaemia
Approved
Approved
Second CRL issued March 17, 2017. Approval announced May 18, 2018.
Fasenra (benralizumab) - GALATHEA
Chronic obstructive pulmonary disease (COPD)
Phase 3
Phase 3
Phase 3 trial did not meet primary endpoint - noted May 11, 2018.
Urothelial carcinoma - Bladder cancer
Bladder cancer
Approved
Approved
BLA acceptance announced December 9, 2017. PDUFA under priority review. Approval announced May 1, 2017.
Durvalumab +/- tremelimumab (ARCTIC)
Non-small cell lung cancer (NSCLC)
Phase 3
Phase 3
Phase 3 data released April 24, 2018 - primary endpoints not met.
Benralizumab
Severe, uncontrolled asthma
Approved
Approved
Approval announced November 14, 2017.
Bydureon
Type 2 Diabetes
Phase 3
Phase 3
Phase 3 Cardiovascular Outcome trial data released September 14, 2017 - primary efficacy objective of a superior reduction in MACE missed statistical significance (p=0.061).
Acalabrutinib
Relapsed or Refractory Mantle Cell Lymphoma
Approved
Approved
Priority Review announced August 2, 2017. Approval announced October 31, 2017.
Saxagliptin and dapagliflozin
Type-2 diabetes
Approved
Approved
Approval announced February 28, 2017.
Lynparza
Breast cancer
Approved
Approved
Phase 3 data released February 17, 2016 - primary endpoint met. Late breaker at ASCO June 4, 2017 showed HR of 0.58 (42% reduction of risk of disease progression or death). Approval announced January 12, 2018.
Lynparza - SOLO 2
Second-line ovarian cancer
Approved
Approved
Approval announced August 17, 2017.
Tralokinumab (STRATOS1)
Severe, uncontrolled asthma
Phase 3
Phase 3
Phase 3 trial did not meet primary endpoint - May 10, 2017.
Tagrisso
Epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC)
Approved
Approved
Approval announced March 31, 2017.
Tralokinumab (STRATOS2)
Severe, uncontrolled asthma
Phase 3
Phase 3
Phase 3 data released November 1, 2017 - primary endpoint not met.
Faslodex (fulvestrant)
Monotherapy for expanded use in women with HR+, HER2- advanced breast cancer
Approved
Approved
Expanded approval announced August 28, 2017.

Latest News

  1. AstraZeneca has agreed to sell the rights to Crestor (rosuvastatin) and associated medicines in over 30 countries in Europe, except the UK and Spain, to Grünenthal GmbH (Grünenthal). Crestor is a statin approved for the treatment of dyslipidaemia and hypercholesterolaemia.

    Ruud Dobber, Executive Vice President, BioPharmaceuticals Business Unit, said: “This agreement supports the management of our mature medicines to enable reinvestment into the pipeline and bringing new, innovative treatments to patients. Grünenthal previously acquired the rights to several established AstraZeneca medicines and is well placed to ensure continued access to Crestor for patients across Europe.”

    AstraZeneca will continue to manufacture and supply Crestor to…

    AstraZeneca has agreed to sell the rights to Crestor (rosuvastatin) and associated medicines in over 30 countries in Europe, except the UK and Spain, to Grünenthal GmbH (Grünenthal). Crestor is a statin approved for the treatment of dyslipidaemia and hypercholesterolaemia.

    Ruud Dobber, Executive Vice President, BioPharmaceuticals Business Unit, said: “This agreement supports the management of our mature medicines to enable reinvestment into the pipeline and bringing new, innovative treatments to patients. Grünenthal previously acquired the rights to several established AstraZeneca medicines and is well placed to ensure continued access to Crestor for patients across Europe.”

    AstraZeneca will continue to manufacture and supply Crestor to Grünenthal during a transition period. AstraZeneca will also continue selling the medicine in other countries, including those in North America, in Japan, China and other emerging markets.

    Financial considerations

    The divestment is anticipated to close in the first quarter of 2021, subject to customary closing conditions and regulatory clearances, upon which Grünenthal will make an upfront, non-contingent payment to AstraZeneca of $320m and may also make future milestone payments of up to $30m. Income arising from the upfront and future payments will be reported in AstraZeneca's financial statements within Other Operating Income & Expense. The divestment will not impact the Company's financial guidance for 2020.

    Pursuant to London Stock Exchange listing rule 10.4.1R (notification of class 2 transactions), the gross book value of assets subject to the divestment as of 31 December 2019 was nil. In 2019, Crestor generated Product Sales of $136m and Profit before Tax of $98m in the countries covered by the agreement. The consideration will be paid in cash and the proceeds used for general corporate purposes.

    Crestor

    Crestor (rosuvastatin) is a statin, a lipid-lowering medicine, used to treat blood lipid disorders and to prevent cardiovascular events, such as heart attacks and strokes. Crestor produces its lipid modifying effects in two ways: it blocks an enzyme in the liver causing the liver to make less cholesterol, and it increases the uptake and breakdown by the liver of cholesterol already in the blood. Crestor is approved as a lipid regulating medicine in more than 100 countries.

    AstraZeneca

    AstraZeneca (NASDAQ:AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com 

    — WebWireID267368 —


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  2. New data from the DESTINY-Breast01 trial reinforce the efficacy of ENHERTU® in HER2-positive metastatic breast cancer

    New data from the SERENA-1 Phase I trial show strong efficacy and safety profile for next-generation oral SERD AZD9833 in HR-positive advanced breast cancer

    AstraZeneca will unveil new developments across a range of stages and subtypes of breast cancer at the 2020 San Antonio Breast Cancer Symposium (SABCS), which will be held virtually from 8 to 11 December.

    Key abstracts include:

    • New data from the DESTINY-Breast01 Phase II trial, which reinforce the durable efficacy seen with ENHERTU® (fam-trastuzumab deruxtecan-nxki) in HER2-positive metastatic breast cancer following two or more prior anti-HER2 based regimens
    • New results…

    New data from the DESTINY-Breast01 trial reinforce the efficacy of ENHERTU® in HER2-positive metastatic breast cancer

    New data from the SERENA-1 Phase I trial show strong efficacy and safety profile for next-generation oral SERD AZD9833 in HR-positive advanced breast cancer

    AstraZeneca will unveil new developments across a range of stages and subtypes of breast cancer at the 2020 San Antonio Breast Cancer Symposium (SABCS), which will be held virtually from 8 to 11 December.

    Key abstracts include:

    • New data from the DESTINY-Breast01 Phase II trial, which reinforce the durable efficacy seen with ENHERTU® (fam-trastuzumab deruxtecan-nxki) in HER2-positive metastatic breast cancer following two or more prior anti-HER2 based regimens
    • New results from the SERENA-1 Phase I trial, which demonstrate strong efficacy and safety for a next-generation oral selective oestrogen receptor degrader (SERD), AZD9833 as a monotherapy and in combination with CDK4/6 inhibitor, palbociclib, in HR-positive, HER2-negative advanced breast cancer

    José Baselga, Executive Vice President, Oncology R&D, said: "We are committed to transforming outcomes for women diagnosed or living with breast cancer by advancing a new generation of promising potential new medicines. The updates from the comprehensive DESTINY breast program reflect the potential of ENHERTU to help a wide range of breast cancer patients, while the encouraging data from the SERENA-1 Phase I trial paves the way for a clinical development program to help patients with hormone receptor-positive disease."

    Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: "Significant progress has been made to improve outcomes for those living with breast cancer but there is still much work to be done. At SABCS 2020, our dedication to transforming the lives of those living with breast cancer will be front and center. With new updates from six different approved and potential new medicines, we are directly addressing patients' greatest unmet needs and are potentially redefining treatment. Additionally, we are making an impact through collaborations with the scientific community to accelerate innovation."

    New, longer-term data from DESTINY-Breast01 to be presented at SABCS will highlight the updated efficacy and safety profiles of ENHERTU in patients with previously-treated HER2-positive metastatic breast cancer with an additional 9.4 months of follow up.

    Furthermore, AstraZeneca and Daiichi Sankyo Company, Limited (Daiichi Sankyo) will showcase several TiP abstracts that highlight how the companies are building on the impressive results of ENHERTU in patients with HER2-positive metastatic breast cancer. These include trials to explore the potential of ENHERTU in earlier lines of treatment and stages of disease and in new breast cancer settings, including patients with low levels of HER2 expression. They also include combinations with other anti-cancer medicines such as paclitaxel, FASLODEX (fulvestrant), IMFINZI (durvalumab) and the potential new medicine capivasertib, an AKT-inhibitor.

    AstraZeneca will present new efficacy and safety results from the dose escalation and expansion cohort of SERENA-1, a Phase I clinical trial of next-generation oral SERD AZD9833 as a monotherapy and in combination with the CDK4/6 inhibitor palbociclib in women with HR-positive breast cancer.

    Building on the updated SERENA-1 findings, the Company will present two Phase II trial-in-progress (TiP) abstracts for the potential new medicine AZD9833, evaluating its efficacy and safety in previously treated post-menopausal women with advanced breast cancer and its biological effects in women with treatment-naïve early-stage breast cancer.

    AstraZeneca is also presenting real-world evidence to understand outcomes for patients with germline BRCA mutations, and treatment patterns among patients with HER2-positive metastatic breast cancer. The Company will also showcase data on the potential role of artificial intelligence and digital pathology in measuring levels of HER2 expression in patients with breast cancer.

    Additionally, AstraZeneca recognizes the important role of externally sponsored scientific research (ESR) in expanding the medical and scientific understanding of the Company's medicines, and in identifying associated areas of unmet need in breast cancer. More than half of the AstraZeneca abstracts at this year's SABCS are ESR studies with AstraZeneca medicines across the various subtypes of breast cancer.

    Abstracts to be presented at 2020 SABCS featuring AstraZeneca medicines and pipeline molecules include:*

    Abstract Title

    Lead Author

    Abstract Details

    ENHERTU (fam-trastuzumab deruxtecan-nxki)1

    Updated results from DESTINY-Breast01, a phase 2 trial of trastuzumab deruxtecan (T-DXd) in HER2 positive metastatic breast cancer

    Modi S

    PD3-06

     

    Spotlight Poster-Discussion 3 – Advances in HER2 Positive Disease

     

    Date: Wednesday, December 9, 2020

    Time: 6:30-7:45pm CT

    Trastuzumab deruxtecan (T-DXd; DS-8201) with nivolumab in patients with HER2-expressing,

    advanced breast cancer: a 2-part, phase 1b, multicenter, open-label study

    Hamilton E

    PD3-07

     

    Spotlight Poster-Discussion 3 - Advances in HER2 Positive Disease

     

    Date: Wednesday, December 9, 2020

    Time: 6:30-7:45pm CT

    Novel approach to HER2 quantification: digital pathology coupled with AI-based image and data analysis delivers objective and quantitative HER2 expression analysis for enrichment of responders to trastuzumab deruxtecan (T-DXd; DS-8201), specifically in HER2-low patients

    Gustavson M

    PD6-01

     

    Spotlight Poster-Discussion 6 - Novel Approaches to Pathology and Imaging

     

    Date: Thursday, December 10, 2020

    Time: 3:30-4:45pm CT

    A real-world evidence study of treatment patterns among patients with HER2-positive metastatic breast cancer

    Collins J

    PS7-82

     

    Poster Session 7 - Epidemiology

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Solti-1804 HER2-PREDICT: A biomarker research study of DS8201-A-U301 -U302 and -U303

    Trials [TiP]*

    Prat A

    OT-03-07

     

    Ongoing Trials Posters - Antibody-drug Conjugates

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Trastuzumab deruxtecan (T-DXd; DS-8201) vs trastuzumab emtansine (T-DM1) in high-risk patients with HER2-positive, residual, invasive early breast cancer after neoadjuvant therapy: a randomized, phase 3 trial (DESTINY-Breast05) [TiP]

    Geyer CE Jr

    OT-03-01

     

    Ongoing Trials Posters – Antibody-drug Conjugates

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Trastuzumab deruxtecan (T-DXd; DS-8201) vs investigator's choice of chemotherapy in patients

    with hormone receptor–positive (HR+), HER2 low metastatic breast cancer whose disease has

    progressed on endocrine therapy in the metastatic setting: a randomized, global phase 3 trial (DESTINYBreast06) [TiP]

    Bardia A

    OT-03-09

     

    Ongoing Trials Posters - Antibody-drug Conjugates

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Trastuzumab deruxtecan (T-DXd; DS-8201) combinations in patients with HER2-positive advanced or metastatic breast cancer: a phase 1b/2 open-label, multicenter, dose-finding and dose-expansion study (DESTINY-Breast07) [TiP]

    Andre F

    OT-03-04

     

    Ongoing Trials Posters - Antibody-drug Conjugates

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Trastuzumab deruxtecan (T-DXd; DS-8201) in combination with other anticancer agents in patients with HER2-low metastatic breast cancer: a phase 1b, open-label, multicenter, dose-finding and dose-expansion study (DESTINY-Breast08) [TiP]

    Jhaveri K

    OT-03-05

     

    Ongoing Trials Posters - Antibody-drug Conjugates

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    LYNPARZA (olaparib)2

    Real-world clinical outcomes of patients with BRCA-mutated (BRCAm) HER2-negative metastatic breast cancer: a CancerLinQ® study

    Miller R

    PS7-66

     

    Poster Session 7 - Epidemiology

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    DOLAF- An international multicenter phase II trial of durvalumab (MEDI4736) plus OLAparib plus Fulvestrant in metastatic or locally advanced ER-positive, HER2-negative breast cancer patients selected using criteria that predict sensitivity to olaparib*

    Guiu S

    OT-13-05

     

    Ongoing Trials Posters – Immunotherapy

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Ceralasertib (cer) in combination with olaparib (ola) in patients (pts) with advanced breast cancer: results of Phase I expansion cohorts

    Krebs MG

    PS11-18

     

    Poster Session 11 – Systemic Therapies II – New

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    FASLODEX (fulvestrant)

    Validation of a predictive model for potential response to neoadjuvant endocrine therapy (NET) in postmenopausal women with clinical stage II or III Estrogen Receptor positive (ER+) and HER2 negative (HER2-) breast cancer (BC): an ALTERNATE trial analysis. (Alliance A011106)*

    Ellis MJ

    PD2-10

     

    Spotlight Poster Discussion 2 – Refining Targeted Therapy in HR+ Disease

     

    Date: Wednesday, December 9, 2020

    Time: 5:15-6:30pm CT

    Neoadjuvant chemotherapy (NCT) response in postmenopausal women with clinical stage II or III estrogen receptor positive and HER2 negative breast cancer resistant to endocrine therapy (ET) in the ALTERNATE trial (Alliance A011106)*

    Ma CX

    GS4-05

     

    General Session 4

     

    Date: Friday, December 11, 2020

    Time: 9:45-10:00am ET

    Palbociclib (P) in combination with fulvestrant (F) or letrozole (L) in endocrine-sensitive patients (pts) with hormone receptor (HR)[+]/HER2[-] metastatic breast cancer (MBC): detailed safety analysis from a multicenter, randomized, open-label, phase II trial (PARSIFAL)*

    Perez-Garcia JM

    PS10-17

     

    Poster Session 10 – Systemic Therapies I – Targeted

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Serum thymidine kinase activity in patients with luminal metastatic breast cancer treated with palbociclib and fulvestrant within the PYTHIA trial*

    Malorni L

    PS5-05

     

    Poster Session 5 – Response Prediction Biomarkers II

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    GEICAM/2014-03 (Registem): a prospective registry of unresectable locally advanced or metastatic breast cancer: Characteristics of a subset of patients with triple negative subtype*

    Jara C

    PS7-25

     

    Poster Session 7

    Epidemiology

     

    Wednesday, December 9, 2020: 8:00 AM CT

    Evaluating serum thymidine kinase in hormone receptor positive metastatic breast cancer patients receiving first line endocrine therapy in the SWOG S0226 trial*

    Paoletti I

    PS2-04

     

    Poster Session 2 – Markers, Pathology

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Characteristics of HR+/HER2- patients with recurrent disease by HER2 expression from a prospective registry of unresectable locally advanced or metastatic breast cancer: GEICAM/2014-03 (RegistEM)*

    Alvarez I

    PS7-24

     

    Poster Session 7 - Epidemiology

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    GEICAM/2014-03 (Registem): A prospective registry of advanced breast cancer: a subset of triple negative breast cancer patients with HER2 low expression*

    Jara C

    PS7-35

     

    Poster Session 7 - Epidemiology

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Characteristics of HR+/HER2- patients with recurrent disease from a prospective registry of unresectable locally advanced or metastatic breast cancer: GEICAM/2014-03 (RegistEM)*

    Alvarez I

    PS7-08

     

    Poster Session 7 – Epidemiology

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Assessment of early ctDNA dynamics to predict efficacy of targeted therapies in metastatic breast cancer: Results from plasmaMATCH trial*

    Pascual J

    PS5-02

     

    Poster Session 5 – Response Prediction Biomarkers II

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Plk1 expression and efficacy of palbociclib in advanced hormonal receptor-positive breast cancer patients from PEARL study (GEICAM/2012-03)*

    Guerro-Zotano A

    PS2-01

     

    Poster Session 2 – Markers, Pathology

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am

    Mutational profile from circulating tumor DNA in triple negative breast cancer: results from the prospective registry of unresectable locally advanced or metastatic breast cancer GEICAM/2014-03 (RegistEM)*

    Guerro-Zotano A

    PS5-22

     

    Poster Session 5 – Response Prediction Biomarkers II

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    Targetable ERBB2 mutation status is an independent marker of adverse prognosis in estrogen receptor positive, ERBB2 non-amplified primary lobular breast carcinoma: Validation using a novel gene signature of HER2 activation

    Alsaleem M

    PS6-11

     

    Poster Session 6 – Prognostic Factors

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    AZD9833

    Updated data from SERENA-1: A phase 1 dose escalation and expansion study of the next generation oral SERD AZD9833 as a monotherapy and in combination with palbociclib, in women with ER-positive, HER2-negative advanced breast cancer

    Baird R

    PS11-05

     

    Poster Session 11 – Systemic Therapies II – New

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    SERENA-2: A randomised, open-label, parallel-group, multicentre phase 2 study comparing the efficacy and safety of oral AZD9833 versus fulvestrant in women with advanced ER-positive HER2-negative breast cancer

    Oliveria M

    OT-09-02

     

    Ongoing Trials Posters – Endocrine Therapy

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    A randomised, open-label, parallel-group, multicentre phase 2 study comparing the efficacy and safety of oral AZD9833 versus fulvestrant in women with advanced ER-positive HER2-negative breast cancer (SERENA-2) [TiP]

    Oliveria M

    OT-09-02

     

    Ongoing Trials Posters – Endocrine Therapy

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    A randomised, pre-surgical study to investigate the biological effects of AZD9833 in women with ER-positive HER2-negative primary breast cancer (SERENA-3) [TiP]

    Robertson J F R

    OT-09-05

     

    Ongoing Trials Posters – Endocrine Therapy

     

    Date: Wednesday, December 9, 2020

    Time: 8:00am CT

    *Denotes ESR

    FDA-Approved Indication for ENHERTU

    ENHERTU is a HER2-directed antibody and topoisomerase inhibitor conjugate indicated for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens in the metastatic setting.

    This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

    IMPORTANT SAFETY INFORMATION

    WARNING: INTERSTITIAL LUNG DISEASE and EMBRYO-FETAL TOXICITY

    • Interstitial lung disease (ILD) and pneumonitis, including fatal cases, have been reported with ENHERTU. Monitor for and promptly investigate signs and symptoms including cough, dyspnea, fever, and other new or worsening respiratory symptoms. Permanently discontinue ENHERTU in all patients with Grade 2 or higher ILD/pneumonitis. Advise patients of the risk and to immediately report symptoms.
    • Exposure to ENHERTU during pregnancy can cause embryo-fetal harm. Advise patients of these risks and the need for effective contraception.

    Contraindications

    None.

    WARNINGS AND PRECAUTIONS

    Interstitial Lung Disease / Pneumonitis

    Severe, life-threatening, or fatal interstitial lung disease (ILD), including pneumonitis, can occur in patients treated with ENHERTU. In clinical studies, of the 234 patients with unresectable or metastatic HER2-positive breast cancer treated with ENHERTU, ILD occurred in 9% of patients. Fatal outcomes due to ILD and/or pneumonitis occurred in 2.6% of patients treated with ENHERTU. Median time to first onset was 4.1 months (range: 1.2 to 8.3).

    Advise patients to immediately report cough, dyspnea, fever, and/or any new or worsening respiratory symptoms. Monitor patients for signs and symptoms of ILD. Promptly investigate evidence of ILD. Evaluate patients with suspected ILD by radiographic imaging. Consider consultation with a pulmonologist. For asymptomatic ILD/pneumonitis (Grade 1), interrupt ENHERTU until resolved to Grade 0, then if resolved in ≤28 days from date of onset, maintain dose. If resolved in >28 days from date of onset, reduce dose one level. Consider corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥0.5 mg/kg prednisolone or equivalent). For symptomatic ILD/pneumonitis (Grade 2 or greater), permanently discontinue ENHERTU. Promptly initiate corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥1 mg/kg prednisolone or equivalent). Upon improvement, follow by gradual taper (e.g., 4 weeks).

    Neutropenia

    Severe neutropenia, including febrile neutropenia, can occur in patients treated with ENHERTU. Of the 234 patients with unresectable or metastatic HER2-positive breast cancer who received ENHERTU, a decrease in neutrophil count was reported in 30% of patients and 16% had Grade 3 or 4 events. Median time to first onset was 1.4 months (range: 0.3 to 18.2). Febrile neutropenia was reported in 1.7% of patients.

    Monitor complete blood counts prior to initiation of ENHERTU and prior to each dose, and as clinically indicated. Based on the severity of neutropenia, ENHERTU may require dose interruption or reduction. For Grade 3 neutropenia (Absolute Neutrophil Count [ANC] <1.0 to 0.5 x 109/L) interrupt ENHERTU until resolved to Grade 2 or less, then maintain dose. For Grade 4 neutropenia (ANC <0.5 x 109/L) interrupt ENHERTU until resolved to Grade 2 or less. Reduce dose by one level. For febrile neutropenia (ANC <1.0 x 109/L and temperature >38.3ºC or a sustained temperature of ≥38ºC for more than 1 hour), interrupt ENHERTU until resolved. Reduce dose by one level.

    Left Ventricular Dysfunction

    Patients treated with ENHERTU may be at increased risk of developing left ventricular dysfunction. Left ventricular ejection fraction (LVEF) decrease has been observed with anti-HER2 therapies, including ENHERTU. In the 234 patients with unresectable or metastatic HER2-positive breast cancer who received ENHERTU, two cases (0.9%) of asymptomatic LVEF decrease were reported. Treatment with ENHERTU has not been studied in patients with a history of clinically significant cardiac disease or LVEF <50% prior to initiation of treatment.

    Assess LVEF prior to initiation of ENHERTU and at regular intervals during treatment as clinically indicated. Manage LVEF decrease through treatment interruption. Permanently discontinue ENHERTU if LVEF of <40% or absolute decrease from baseline of >20% is confirmed. When LVEF is >45% and absolute decrease from baseline is 10-20%, continue treatment with ENHERTU. When LVEF is 40-45% and absolute decrease from baseline is <10%, continue treatment with ENHERTU and repeat LVEF assessment within 3 weeks. When LVEF is 40-45% and absolute decrease from baseline is 10-20%, interrupt ENHERTU and repeat LVEF assessment within 3 weeks. If LVEF has not recovered to within 10% from baseline, permanently discontinue ENHERTU. If LVEF recovers to within 10% from baseline, resume treatment with ENHERTU at the same dose. When LVEF is <40% or absolute decrease from baseline is >20%, interrupt ENHERTU and repeat LVEF assessment within 3 weeks. If LVEF of <40% or absolute decrease from baseline of >20% is confirmed, permanently discontinue ENHERTU. Permanently discontinue ENHERTU in patients with symptomatic congestive heart failure.

    Embryo-Fetal Toxicity

    ENHERTU can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risks to a fetus. Verify the pregnancy status of females of reproductive potential prior to the initiation of ENHERTU. Advise females of reproductive potential to use effective contraception during treatment and for at least 7 months following the last dose of ENHERTU. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ENHERTU and for at least 4 months after the last dose of ENHERTU.

    Adverse Reactions

    The safety of ENHERTU was evaluated in a pooled analysis of 234 patients with unresectable or metastatic HER2-positive breast cancer who received at least one dose of ENHERTU 5.4 mg/kg in DESTINY-Breast01 and Study DS8201-A-J101. ENHERTU was administered by intravenous infusion once every three weeks. The median duration of treatment was 7 months (range: 0.7 to 31).

    Serious adverse reactions occurred in 20% of patients receiving ENHERTU. Serious adverse reactions in >1% of patients who received ENHERTU were interstitial lung disease, pneumonia, vomiting, nausea, cellulitis, hypokalemia, and intestinal obstruction. Fatalities due to adverse reactions occurred in 4.3% of patients including interstitial lung disease (2.6%), and the following events occurred in one patient each (0.4%): acute hepatic failure/acute kidney injury, general physical health deterioration, pneumonia, and hemorrhagic shock.

    ENHERTU was permanently discontinued in 9% of patients, of which ILD accounted for 6%. Dose interruptions due to adverse reactions occurred in 33% of patients treated with ENHERTU. The most frequent adverse reactions (>2%) associated with dose interruption were neutropenia, anemia, thrombocytopenia, leukopenia, upper respiratory tract infection, fatigue, nausea, and ILD. Dose reductions occurred in 18% of patients treated with ENHERTU. The most frequent adverse reactions (>2%) associated with dose reduction were fatigue, nausea, and neutropenia.

    The most common adverse reactions (frequency ≥20%) were nausea (79%), fatigue (59%), vomiting (47%), alopecia (46%), constipation (35%), decreased appetite (32%), anemia (31%), neutropenia (29%), diarrhea (29%), leukopenia (22%), cough (20%), and thrombocytopenia (20%).

    Use in Specific Populations

    • Pregnancy: ENHERTU can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risks to a fetus. There are clinical considerations if ENHERTU is used in pregnant women, or if a patient becomes pregnant within 7 months following the last dose of ENHERTU.
    • Lactation: There are no data regarding the presence of ENHERTU in human milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment with ENHERTU and for 7 months after the last dose.
    • Females and Males of Reproductive Potential: Pregnancy testing: Verify pregnancy status of females of reproductive potential prior to initiation of ENHERTU. Contraception: Females: ENHERTU can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to use effective contraception during treatment with ENHERTU and for at least 7 months following the last dose. Males: Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ENHERTU and for at least 4 months following the last dose. Infertility: ENHERTU may impair male reproductive function and fertility.
    • Pediatric Use: Safety and effectiveness of ENHERTU have not been established in pediatric patients.
    • Geriatric Use: Of the 234 patients with HER2-positive breast cancer treated with ENHERTU 5.4 mg/kg, 26% were ≥65 years and 5% were ≥75 years. No overall differences in efficacy were observed between patients ≥65 years of age compared to younger patients. There was a higher incidence of Grade 3-4 adverse reactions observed in patients aged ≥65 years (53%) as compared to younger patients (42%).
    • Hepatic Impairment: In patients with moderate hepatic impairment, due to potentially increased exposure, closely monitor for increased toxicities related to the topoisomerase inhibitor.

    To report SUSPECTED ADVERSE REACTIONS, contact Daiichi Sankyo, Inc. at 1-877-437-7763 or FDA at 1-800-FDA-1088 or fda.gov/medwatch.

    Please see accompanying full Prescribing Information, including Boxed WARNINGS, and Medication Guide.

    IMPORTANT SAFETY INFORMATION for LYNPARZA® (olaparib) tablets

    CONTRAINDICATIONS

    There are no contraindications for LYNPARZA.

    WARNINGS AND PRECAUTIONS

    Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML): Occurred in <1.5% of patients exposed to LYNPARZA monotherapy, and the majority of events had a fatal outcome. The duration of therapy in patients who developed secondary MDS/AML varied from <6 months to >2 years. All of these patients had previous chemotherapy with platinum agents and/or other DNA-damaging agents, including radiotherapy, and some also had a history of more than one primary malignancy or of bone marrow dysplasia.

    Do not start LYNPARZA until patients have recovered from hematological toxicity caused by previous chemotherapy (≤Grade 1). Monitor complete blood count for cytopenia at baseline and monthly thereafter for clinically significant changes during treatment. For prolonged hematological toxicities, interrupt LYNPARZA and monitor blood count weekly until recovery.

    If the levels have not recovered to Grade 1 or less after 4 weeks, refer the patient to a hematologist for further investigations, including bone marrow analysis and blood sample for cytogenetics. Discontinue LYNPARZA if MDS/AML is confirmed.

    Pneumonitis: Occurred in <1% of patients exposed to LYNPARZA, and some cases were fatal. If patients present with new or worsening respiratory symptoms such as dyspnea, cough, and fever, or a radiological abnormality occurs, interrupt LYNPARZA treatment and initiate prompt investigation. Discontinue LYNPARZA if pneumonitis is confirmed and treat patient appropriately.

    Embryo-Fetal Toxicity: Based on its mechanism of action and findings in animals, LYNPARZA can cause fetal harm. A pregnancy test is recommended for females of reproductive potential prior to initiating treatment.

    Females

    Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for 6 months following the last dose.

    Males

    Advise male patients with female partners of reproductive potential or who are pregnant to use effective contraception during treatment and for 3 months following the last dose of LYNPARZA and to not donate sperm during this time.

    Venous Thromboembolic Events: Including pulmonary embolism, occurred in 7% of patients with metastatic castration-resistant prostate cancer who received LYNPARZA plus androgen deprivation therapy (ADT) compared to 3.1% of patients receiving enzalutamide or abiraterone plus ADT in the PROfound study. Patients receiving LYNPARZA and ADT had a 6% incidence of pulmonary embolism compared to 0.8% of patients treated with ADT plus either enzalutamide or abiraterone. Monitor patients for signs and symptoms of venous thrombosis and pulmonary embolism, and treat as medically appropriate, which may include long-term anticoagulation as clinically indicated.

    ADVERSE REACTIONS—First-Line Maintenance BRCAm Advanced Ovarian Cancer

    Most common adverse reactions (Grades 1-4) in ≥10% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for SOLO-1 were: nausea (77%), fatigue (67%), abdominal pain (45%), vomiting (40%), anemia (38%), diarrhea (37%), constipation (28%), upper respiratory tract infection/influenza/nasopharyngitis/bronchitis (28%), dysgeusia (26%), decreased appetite (20%), dizziness (20%), neutropenia (17%), dyspepsia (17%), dyspnea (15%), leukopenia (13%), UTI (13%), thrombocytopenia (11%), and stomatitis (11%).

    Most common laboratory abnormalities (Grades 1-4) in ≥25% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for SOLO-1 were: decrease in hemoglobin (87%), increase in mean corpuscular volume (87%), decrease in leukocytes (70%), decrease in lymphocytes (67%), decrease in absolute neutrophil count (51%), decrease in platelets (35%), and increase in serum creatinine (34%).

    ADVERSE REACTIONS—First-Line Maintenance Advanced Ovarian Cancer in Combination with Bevacizumab

    Most common adverse reactions (Grades 1-4) in ≥10% of patients treated with LYNPARZA/bevacizumab compared to a ≥5% frequency for placebo/bevacizumab in the first-line maintenance setting for PAOLA-1 were: nausea (53%), fatigue (including asthenia) (53%), anemia (41%), lymphopenia (24%), vomiting (22%) and leukopenia (18%). In addition, the most common adverse reactions (≥10%) for patients receiving LYNPARZA/bevacizumab irrespective of the frequency compared with the placebo/bevacizumab arm were: diarrhea (18%), neutropenia (18%), urinary tract infection (15%) and headache (14%).

    In addition, venous thromboembolic events occurred more commonly in patients receiving LYNPARZA/bevacizumab (5%) than in those receiving placebo/bevacizumab (1.9%).

    Most common laboratory abnormalities (Grades 1-4) in ≥25% of patients for LYNPARZA in combination with bevacizumab in the first-line maintenance setting for PAOLA-1 were: decrease in hemoglobin (79%), decrease in lymphocytes (63%), increase in serum creatinine (61%), decrease in leukocytes (59%), decrease in absolute neutrophil count (35%) and decrease in platelets (35%).

    ADVERSE REACTIONS—Maintenance Recurrent Ovarian Cancer

    Most common adverse reactions (Grades 1-4) in ≥20% of patients in clinical trials of LYNPARZA in the maintenance setting for SOLO-2 were: nausea (76%), fatigue (including asthenia) (66%), anemia (44%), vomiting (37%), nasopharyngitis/upper respiratory tract infection (URI)/influenza (36%), diarrhea (33%), arthralgia/myalgia (30%), dysgeusia (27%), headache (26%), decreased appetite (22%), and stomatitis (20%).

    Study 19: nausea (71%), fatigue (including asthenia) (63%), vomiting (35%), diarrhea (28%), anemia (23%), respiratory tract infection (22%), constipation (22%), headache (21%), decreased appetite (21%) and dyspepsia (20%).

    Most common laboratory abnormalities (Grades 1-4) in ≥25% of patients in clinical trials of LYNPARZA in the maintenance setting (SOLO-2/Study 19) were: increase in mean corpuscular volume (89%/82%), decrease in hemoglobin (83%/82%), decrease in leukocytes (69%/58%), decrease in lymphocytes (67%/52%), decrease in absolute neutrophil count (51%/47%), increase in serum creatinine (44%/45%), and decrease in platelets (42%/36%).

    ADVERSE REACTIONS—Advanced gBRCAm Ovarian Cancer

    Most common adverse reactions (Grades 1-4) in ≥20% of patients in clinical trials of LYNPARZA for advanced gBRCAm ovarian cancer after 3 or more lines of chemotherapy (pooled from 6 studies) were: fatigue/asthenia (66%), nausea (64%), vomiting (43%), anemia (34%), diarrhea (31%), nasopharyngitis/upper respiratory tract infection (URI) (26%), dyspepsia (25%), myalgia (22%), decreased appetite (22%), and arthralgia/musculoskeletal pain (21%).

    Most common laboratory abnormalities (Grades 1-4) in ≥25% of patients in clinical trials of LYNPARZA for advanced gBRCAm ovarian cancer (pooled from 6 studies) were: decrease in hemoglobin (90%), mean corpuscular volume elevation (57%), decrease in lymphocytes (56%), increase in serum creatinine (30%), decrease in platelets (30%), and decrease in absolute neutrophil count (25%).

    ADVERSE REACTIONS—gBRCAm, HER2-Negative Metastatic Breast Cancer

    Most common adverse reactions (Grades 1-4) in ≥20% of patients in OlympiAD were: nausea (58%), anemia (40%), fatigue (including asthenia) (37%), vomiting (30%), neutropenia (27%), respiratory tract infection (27%), leukopenia (25%), diarrhea (21%), and headache (20%).

    Most common laboratory abnormalities (Grades 1-4) in ≥25% of patients in OlympiAD were: decrease in hemoglobin (82%), decrease in lymphocytes (73%), decrease in leukocytes (71%), increase in mean corpuscular volume (71%), decrease in absolute neutrophil count (46%), and decrease in platelets (33%).

    ADVERSE REACTIONS—First-Line Maintenance gBRCAm Metastatic Pancreatic Adenocarcinoma

    Most common adverse reactions (Grades 1-4) in ≥10% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for POLO were: fatigue (60%), nausea (45%), abdominal pain (34%), diarrhea (29%), anemia (27%), decreased appetite (25%), constipation (23%), vomiting (20%), back pain (19%), arthralgia (15%), rash (15%), thrombocytopenia (14%), dyspnea (13%), neutropenia (12%), nasopharyngitis (12%), dysgeusia (11%), and stomatitis (10%).

    Most common laboratory abnormalities (Grades 1-4) in ≥25% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for POLO were: increase in serum creatinine (99%), decrease in hemoglobin (86%), increase in mean corpuscular volume (71%), decrease in lymphocytes (61%), decrease in platelets (56%), decrease in leukocytes (50%), and decrease in absolute neutrophil count (25%).

    ADVERSE REACTIONS—HRR Gene-mutated Metastatic Castration-Resistant Prostate Cancer

    Most common adverse reactions (Grades 1-4) in ≥10% of patients in clinical trials of LYNPARZA for PROfound were: anemia (46%), fatigue (including asthenia) (41%), nausea (41%), decreased appetite (30%), diarrhea (21%), vomiting (18%), thrombocytopenia (12%), cough (11%), and dyspnea (10%).

    Most common laboratory abnormalities (Grades 1-4) in ≥25% of patients in clinical trials of LYNPARZA for PROfound were: decrease in hemoglobin (98%), decrease in lymphocytes (62%), decrease in leukocytes (53%), and decrease in absolute neutrophil count (34%).

    DRUG INTERACTIONS

    Anticancer Agents: Clinical studies of LYNPARZA in combination with other myelosuppressive anticancer agents, including DNA-damaging agents, indicate a potentiation and prolongation of myelosuppressive toxicity.

    CYP3A Inhibitors: Avoid concomitant use of strong or moderate CYP3A inhibitors. If a strong or moderate CYP3A inhibitor must be co-administered, reduce the dose of LYNPARZA. Advise patients to avoid grapefruit, grapefruit juice, Seville oranges, and Seville orange juice during LYNPARZA treatment.

    CYP3A Inducers: Avoid concomitant use of strong or moderate CYP3A inducers when using LYNPARZA. If a moderate inducer cannot be avoided, there is a potential for decreased efficacy of LYNPARZA.

    USE IN SPECIFIC POPULATIONS

    Lactation: No data are available regarding the presence of olaparib in human milk, its effects on the breastfed infant or on milk production. Because of the potential for serious adverse reactions in the breastfed infant, advise a lactating woman not to breastfeed during treatment with LYNPARZA and for 1 month after receiving the final dose.

    Pediatric Use: The safety and efficacy of LYNPARZA have not been established in pediatric patients.

    Hepatic Impairment: No adjustment to the starting dose is required in patients with mild or moderate hepatic impairment (Child-Pugh classification A and B). There are no data in patients with severe hepatic impairment (Child-Pugh classification C).

    Renal Impairment: No dosage modification is recommended in patients with mild renal impairment (CLcr 51-80 mL/min estimated by Cockcroft-Gault). In patients with moderate renal impairment (CLcr 31-50 mL/min), reduce the dose of LYNPARZA to 200 mg twice daily. There are no data in patients with severe renal impairment or end-stage renal disease (CLcr ≤30 mL/min).

    INDICATIONS

    LYNPARZA is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated:

    First-Line Maintenance BRCAm Advanced Ovarian Cancer

    For the maintenance treatment of adult patients with deleterious or suspected deleterious germline or somatic BRCA-mutated (gBRCAm or sBRCAm) advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

    First-Line Maintenance HRD Positive Advanced Ovarian Cancer in Combination with Bevacizumab

    In combination with bevacizumab for the maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy and whose cancer is associated with homologous recombination deficiency (HRD) positive status defined by either:

    • a deleterious or suspected deleterious BRCA mutation, and/or
    • genomic instability

    Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

    Maintenance Recurrent Ovarian Cancer

    For the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer, who are in complete or partial response to platinum-based chemotherapy.

    Advanced gBRCAm Ovarian Cancer

    For the treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm) advanced ovarian cancer who have been treated with 3 or more prior lines of chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

    gBRCAm, HER2-Negative Metastatic Breast Cancer

    For the treatment of adult patients with deleterious or suspected deleterious gBRCAm, human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer who have been treated with chemotherapy in the neoadjuvant, adjuvant, or metastatic setting. Patients with hormone receptor (HR)-positive breast cancer should have been treated with a prior endocrine therapy or be considered inappropriate for endocrine therapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

    First-Line Maintenance gBRCAm Metastatic Pancreatic Cancer

    For the maintenance treatment of adult patients with deleterious or suspected deleterious gBRCAm metastatic pancreatic adenocarcinoma whose disease has not progressed on at least 16 weeks of a first-line platinum-based chemotherapy regimen. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

    HRR Gene-mutated Metastatic Castration-Resistant Prostate Cancer

    For the treatment of adult patients with deleterious or suspected deleterious germline or somatic homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with enzalutamide or abiraterone. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

    Please click here for complete Prescribing Information, including Patient Information (Medication Guide).

    Important Safety Information About FASLODEX® (fulvestrant) injection

    Contraindications

    • FASLODEX is contraindicated in patients with known hypersensitivity to the drug or to any of its components. Hypersensitivity reactions, including urticaria and angioedema, have been reported in association with FASLODEX

    Risk of Bleeding

    • Because FASLODEX is administered intramuscularly, it should be used with caution in patients with bleeding diatheses, thrombocytopenia, or anticoagulant use

    Hepatic Impairment

    • FASLODEX is metabolized primarily in the liver. A 250 mg dose is recommended in patients with moderate hepatic impairment (Child-Pugh class B). FASLODEX has not been evaluated in patients with severe hepatic impairment (Child-Pugh class C)

    Injection Site Reaction

    • Use caution while administering FASLODEX at the dorsogluteal injection site due to the proximity of the underlying sciatic nerve. Injection site-related events, including sciatica, neuralgia, neuropathic pain, and peripheral neuropathy, have been reported with FASLODEX injection

    Embryo-Fetal Toxicity and Lactation

    • Pregnancy testing is recommended for females of reproductive potential within seven days prior to initiating FASLODEX
    • Advise pregnant women of the potential risk to a fetus. Advise women of reproductive potential to use effective contraception during FASLODEX treatment and for 1 year after the last dose. Advise lactating women not to breastfeed during treatment with FASLODEX and for 1 year after the final dose because of the potential risk to the infant

    Immunoassay Measurement of Serum Estradiol

    • Due to structural similarity of fulvestrant and estradiol, FASLODEX can interfere with estradiol measurement by immunoassay, resulting in falsely elevated estradiol levels

    Adverse Reactions

    Monotherapy

    • The most common adverse reactions occurring in ≥5% of patients receiving FASLODEX 500 mg were injection site pain, nausea, bone pain, arthralgia, headache, back pain, fatigue, pain in extremity, hot flash, myalgia, vomiting, anorexia, diarrhea, asthenia, musculoskeletal pain, cough, dyspnea, and constipation
    • Increased hepatic enzymes (ALT, AST, ALP) occurred in >15% of FASLODEX patients and were not dose-dependent

    Combination Therapy – FASLODEX plus ribociclib

    • The most frequently reported (≥5%) Grade 3 or 4 adverse reactions in patients receiving FASLODEX plus ribociclib in descending frequency were neutropenia, leukopenia, infections, and abnormal liver function tests
    • The most common adverse reactions (≥20%) of any grade reported in patients receiving FASLODEX 500 mg plus ribociclib 600 mg/day were neutropenia, infections, leukopenia, cough, nausea, diarrhea, vomiting, constipation, pruritus, and rash
    • Additional adverse reactions in patients receiving FASLODEX plus ribociclib included asthenia, dyspepsia, thrombocytopenia, dry skin, dysgeusia, electrocardiogram QT prolonged, dry mouth, vertigo, dry eye, lacrimation increased, erythema, hypocalcemia, blood bilirubin increased, and syncope

    Combination Therapy—FASLODEX plus palbociclib

    • The most frequently reported Grade ≥3 adverse reactions in patients receiving FASLODEX plus palbociclib in descending frequency were neutropenia and leukopenia
    • Adverse reactions (≥10%) of any grade reported in patients receiving FASLODEX 500 mg plus palbociclib 125 mg/day by descending frequency were neutropenia, leukopenia, infections, fatigue, nausea, anemia, stomatitis, diarrhea, thrombocytopenia, vomiting, alopecia, rash, decreased appetite, and pyrexia
    • Additional adverse reactions occurring at an overall incidence of <10% of patients receiving FASLODEX plus palbociclib included asthenia, aspartate aminotransferase increased, dysgeusia, epistaxis, lacrimation increased, dry skin, alanine aminotransferase increased, vision blurred, dry eye, and febrile neutropenia

    Combination Therapy—FASLODEX plus abemaciclib

    • The most frequently reported (≥5%) Grade 3 or 4 adverse reactions in patients receiving FASLODEX plus abemaciclib were neutropenia, diarrhea, leukopenia, anemia, and infections
    • The most common adverse reactions (≥20%) of any grade reported in patients receiving FASLODEX 500 mg plus abemaciclib 150 mg twice daily were diarrhea, fatigue, neutropenia, nausea, infections, abdominal pain, anemia, leukopenia, decreased appetite, vomiting, and headache

    Indications for FASLODEX

    Monotherapy

    FASLODEX is an estrogen receptor antagonist indicated for the treatment of:

    • Hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer in postmenopausal women not previously treated with endocrine therapy
    • HR-positive advanced breast cancer in postmenopausal women with disease progression following endocrine therapy

    Combination Therapy

    FASLODEX is indicated for the treatment of:

    • HR-positive, HER2-negative advanced or metastatic breast cancer in postmenopausal women in combination with ribociclib as initial endocrine-based therapy or following disease progression on endocrine therapy
    • HR-positive, HER2-negative advanced or metastatic breast cancer in combination with palbociclib or abemaciclib in women with disease progression after endocrine therapy

    Please see full Prescribing Information for FASLODEX with Patient Information.

    SELECT SAFETY INFORMATION for IMFINZI® (durvalumab) injection for intravenous use

    • Serious, potentially fatal risks were seen with IMFINZI in the CASPIAN trial. The most frequent serious adverse reactions reported in at least 1% of patients were febrile neutropenia (4.5%), pneumonia (2.3%), anemia (1.9%), pancytopenia (1.5%), pneumonitis (1.1%) and COPD (1.1%).
    • Immune-mediated adverse reactions including immune-mediated pneumonitis, hepatitis, colitis, endocrinopathies (including thyroid disorders, adrenal insufficiency, type 1 diabetes, and hypophysitis), nephritis, dermatologic reactions, other immune-mediated adverse reactions, infection, and infusion-related reactions were reported in patients receiving IMFINZI in the CASPIAN trial.
    • The most common adverse reactions (≥20%) were nausea, fatigue/asthenia and alopecia.
    • Advise women not to become pregnant or breastfeed during treatment with IMFINZI and for at least 3 months after the last dose.
    • The safety and effectiveness of IMFINZI have not been established in pediatric patients.

    U.S. FDA-APPROVED INDICATIONS

    IMFINZI is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who:

    • Have disease progression during or following platinum-containing chemotherapy.
    • Have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

    This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

    IMFINZI is indicated for the treatment of adult patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy.

    IMFINZI, in combination with etoposide and either carboplatin or cisplatin, is indicated for the first-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC).

    Please see complete Prescribing Information, including Patient Information.

    Notes

    AstraZeneca in breast cancer

    Driven by a growing understanding of breast cancer biology, AstraZeneca is starting to challenge, and redefine, the current clinical paradigm for how breast cancer is classified and treated to deliver even more effective treatments to patients in need – with the bold ambition to one day eliminate breast cancer as a cause of death.

    AstraZeneca has a comprehensive portfolio of approved and promising compounds in development that leverage different mechanisms of action to address the biologically diverse breast cancer tumor environment. AstraZeneca aims to continue to transform outcomes for HR-positive breast cancer with foundational medicines fulvestrant and goserelin and the next-generation SERD and potential new medicine AZD9833. PARP inhibitor, LYNPARZA (olaparib), was the first targeted treatment option for metastatic breast cancer patients with an inherited BRCA mutation. AstraZeneca with MSD (Merck & Co., Inc. in the US and Canada) continue to research LYNPARZA in metastatic breast cancer patients with an inherited BRCA mutation, and are exploring new opportunities to treat these patients earlier in their disease state. Building on the first approval of ENHERTU, a HER2-directed antibody drug conjugate, in previously treated HER2-positive metastatic breast cancer, AstraZeneca and Daiichi Sankyo are exploring its potential in earlier lines of treatment and in new breast cancer settings. To bring much needed treatment options to patients with triple-negative breast cancer, an aggressive form of breast cancer, AstraZeneca is testing immunotherapy durvalumab in combination with other oncology medicines, including LYNPARZA and ENHERTU, investigating the potential of AKT kinase inhibitor, capivasertib, in combination with chemotherapy, and collaborating with Daiichi Sankyo to explore the potential of TROP2-directed ADC, datopotamab deruxtecan (DS-1062).

    AstraZeneca in oncology

    AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio ofnew medicines that has the potential to transform patients' lives and the Company's future. With seven new medicines launched between 2014 and 2020, and a broad pipelineof small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers.

    By harnessing the power of four scientific platforms – Immuno-Oncology, Tumor Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalized combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.

    AstraZeneca

    AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal & Metabolism and Respiratory & Immunology. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information, please visit www.astrazeneca-us.com and follow us on Twitter @AstraZenecaUS.

    References

    1. Enhertu is developed and commercialised in collaboration with Daiichi Sankyo worldwide, except in Japan where Daiichi Sankyo maintains exclusive rights.
    2. Lynparza is developed and commercialised in collaboration with MSD (Merck & Co., Inc. in the US and Canada).

    US-48294 | 12/20

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  3. New data show safety and efficacy of roxadustat in treating anemia secondary to lower-risk myelodysplastic syndromes (MDS) regardless of ring sideroblast (RS) or baseline erythropoietin level

    Multiple analyses evaluate cardiovascular safety and efficacy of roxadustat in patients with anemia of chronic kidney disease (CKD) regardless of dialysis status 

    SAN FRANCISCO, Dec. 02, 2020 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ:FGEN) and its partner, AstraZeneca (NASDAQ:AZN), will present additional analyses of roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (MDS) and results from multiple roxadustat phase 3 studies. Roxadustat is a first-in-class oral small molecule hypoxia-inducible factor…

    New data show safety and efficacy of roxadustat in treating anemia secondary to lower-risk myelodysplastic syndromes (MDS) regardless of ring sideroblast (RS) or baseline erythropoietin level

    Multiple analyses evaluate cardiovascular safety and efficacy of roxadustat in patients with anemia of chronic kidney disease (CKD) regardless of dialysis status 

    SAN FRANCISCO, Dec. 02, 2020 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ:FGEN) and its partner, AstraZeneca (NASDAQ:AZN), will present additional analyses of roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (MDS) and results from multiple roxadustat phase 3 studies. Roxadustat is a first-in-class oral small molecule hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) in development for the treatment of patients with anemia of CKD and anemia secondary to lower-risk MDS. FibroGen and its partner will present eight abstracts at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually December 5-8, 2020.

    "We are pleased to share additional 52-week data from the open-label portion of our Phase 3 anemia of MDS trial with the medical community at ASH," said Enrique Conterno, Chief Executive Officer, FibroGen. "In addition, we are presenting Phase 3 cardiovascular safety and efficacy results of roxadustat, which highlight its potential in a broad range of CKD patients."

    MDS is a group of blood disorders characterized by poorly formed or dysfunctional blood cells, resulting in chronic anemia.1 Exploratory analyses to be presented at ASH show the efficacy of roxadustat in transfusion-dependent lower-risk MDS patients regardless of ring sideroblast and baseline erythropoietin status - characteristics used to predict response to treatment.2 Patients with ring sideroblasts (RS+), without ring sideroblasts (RS-), baseline erythropoietin (BL EPO) ≤ 200 mIU/ml, and BL EPO > 200 mIU/ml achieved the primary endpoint of transfusion independence for ≥ 8 weeks during the first 28 treatment weeks (23% of MDS-RS+, 55% of MDS-RS-, 39% of BL EPO ≤ 200 mIU/ml, and 33% of BL EPO > 200 mIU/ml patients). Detailed results will be presented at the meeting.

    Enrollment in the double-blind placebo-controlled portion of the Phase 3 roxadustat study of patients with anemia secondary to lower-risk MDS is ongoing (NCT03263091).

    Roxadustat presentations during the 62nd ASH Annual Meeting and Exposition:

    PresenterPresentation titlePresentation details
    David Henry, MDOral Roxadustat Demonstrates Efficacy in Anemia Secondary to Lower-Risk Myelodysplastic Syndrome Irrespective of Ring Sideroblasts and Baseline Erythropoietin LevelsePoster #1277

    Session 637: Myelodysplastic Syndromes—Clinical Studies (Poster I)

    Sat., Dec. 5: 7:00 AM–3:30 PM PT
    Steven Fishbane, MDRoxadustat Lowers Risk of RBC Transfusion in Patients with Anemia of CKD



    ePoster #748

    Session 101: Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron (Poster I)

    Sat., Dec. 5: 7:00 AM–3:30 PM PT
    Steven Fishbane, MDPooled Efficacy and Cardiovascular Safety Results of Roxadustat Compared with Epoetin Alfa in the Treatment of Anemia in Chronic Kidney Disease Patients on DialysisePoster #749

    Session 101: Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron (Poster I)

    Sat., Dec. 5: 7:00 AM–3:30 PM PT
    Carol Pollock, MDRoxadustat Increases Hemoglobin in Anemic Non-Dialysis-Dependent (NDD) Chronic Kidney Disease (CKD) Patients Independent of InflammationePoster #757

    Session 101: Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron (Poster I)

    Sat., Dec. 5: 7:00 AM–3:30 PM PT
    Roberto Pecoits-Filho, MDRoxadustat Treatment Results in Consistent Improvements in Hemoglobin (Hb) Versus Placebo: An Analysis of 3 Multinational RCTs in Patients with Non-Dialysis-Dependent Chronic Kidney Disease (NDD-CKD)ePoster #758

    Session 101: Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron (Poster I)

    Sat., Dec. 5: 7:00 AM–3:30 PM PT
    Anjay Rastogi, MDRoxadustat Treatment Corrects Anemia to Hemoglobin (Hb) Values ≥10 g/dL in the Majority of Patients with Non-Dialysis-Dependent Chronic Kidney Disease (NDD-CKD)ePoster #761

    Session 101: Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron (Poster I)

    Sat., Dec. 5: 7:00 AM–3:30 PM PT
    Steven Fishbane, MDHemoglobin (Hb) Correction with Roxadustat is Associated with Improved Iron Homeostasis in Patients with Non-Dialysis-Dependent Chronic Kidney Disease (NDD-CKD)ePoster #766

    Session 102: Regulation of Iron Metabolism (Poster I)

    Sat., Dec. 5: 7:00 AM–3:30 PM PT
    Robert Provenzano, MDPooled Efficacy and Cardiovascular Analysis of Roxadustat Compared with Placebo in Anemia Correction in Chronic Kidney Disease Patients Not on DialysisePoster #1671

    Session 101: Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron (Poster II)

    Sun., Dec. 6: 7:00 AM–3:30 PM PT

    About Anemia of CKD

    Chronic kidney disease (CKD) is generally a progressive disease characterized by gradual loss of kidney function that may eventually lead to kidney failure or end stage renal disease, requiring dialysis or kidney transplant. CKD is estimated to occur in approximately 10-12% of adults worldwide and is predicted to become the fifth most common cause of premature death globally by 2040.

    Anemia, a serious medical condition in which patients have insufficient red blood cells and low levels of hemoglobin, is a common early complication of CKD, affecting approximately 20% of CKD patients. Anemia of CKD is associated with an increased risk of hospitalization, cardiovascular complications, and death, and can also cause significant fatigue, cognitive dysfunction and reduced quality of life. Blood transfusions are used for treating severe anemia, however, they may reduce a patient's opportunity for kidney transplant and can increase the risk of infection and/or complications such as heart failure and allergic reactions.

    About MDS

    MDS develops because the bone marrow cells do not develop into mature blood cells. Instead, these blood cells stay within the bone marrow in an immature state. There are many subtypes of MDS. Some cases are mild, while others are more severe, and carry a high risk of becoming acute myelogenous leukemia (AML). It is estimated that more than 10,000 patients are diagnosed with MDS each year in the U.S.,3 and overall prevalence is estimated to be between 60,000 – 170,000 in the country.4

    About Roxadustat

    Roxadustat is a first-in-class orally administered inhibitor of HIF-PH, which increases hemoglobin levels through a mechanism of action that is different from that of traditional erythropoiesis-stimulating agents. As a HIF-PH inhibitor, roxadustat activates a response that occurs naturally when the body responds to reduced oxygen levels in the blood. Roxadustat promotes red blood cell production through increased endogenous production of erythropoietin; improved iron absorption, transport, and mobilization; and downregulation of hepcidin, which helps to overcome the negative impact of inflammation on hemoglobin synthesis and red blood cell production.

    Roxadustat is approved and launched for the treatment of anemia of CKD in Japan and China in adult patients on dialysis (DD) and not on dialysis (NDD). A New Drug Application for the treatment of anemia of CKD in patients both DD and NDD is under review by the U.S. Food and Drug Administration with a decision expected in December 2020. The marketing authorization application for roxadustat for the treatment of anemia of CKD in patients both DD and NDD was accepted by the European Medicines Agency for review on May 21, 2020. Several other licensing applications for roxadustat have been submitted by Astellas and AstraZeneca to regulatory authorities across the globe, which are currently in review.

    Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in the U.S., China and other markets in the Americas and in Australia/New Zealand as well as Southeast Asia.

    About FibroGen

    FibroGen, Inc. is a biopharmaceutical company committed to discovering, developing and commercializing a pipeline of first-in-class therapeutics. The company applies its pioneering expertise in hypoxia-inducible factor (HIF) and connective tissue growth factor (CTGF) biology to advance innovative medicines for the treatment of unmet needs. The Company is currently developing and commercializing roxadustat, an oral small molecule inhibitor of HIF prolyl hydroxylase activity, for anemia associated with chronic kidney disease (CKD). Roxadustat is also in clinical development for anemia associated with myelodysplastic syndromes (MDS) and for chemotherapy-induced anemia (CIA). Pamrevlumab, an anti-CTGF human monoclonal antibody, is in clinical development for the treatment of idiopathic pulmonary fibrosis (IPF), locally advanced unresectable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD), and coronavirus (COVID-19). For more information, please visit www.fibrogen.com.

    Forward-Looking Statements

    This release contains forward-looking statements regarding our strategy, future plans and prospects, including statements regarding the development and commercialization of the company's product candidates, the potential safety and efficacy profile of our product candidates, our clinical programs and regulatory events, and those of our partners. These forward-looking statements include, but are not limited to, statements about our plans, objectives, representations and contentions and are not historical facts and typically are identified by use of terms such as "may," "will", "should," "on track," "could," "expect," "plan," "anticipate," "believe," "estimate," "predict," "potential," "continue" and similar words, although some forward-looking statements are expressed differently. Our actual results may differ materially from those indicated in these forward-looking statements due to risks and uncertainties related to the continued progress and timing of our various programs, including the enrollment and results from ongoing and potential future clinical trials, and other matters that are described in our Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and our Quarterly Report on Form 10-Q for quarter ended September 30, 2020 filed with the Securities and Exchange Commission (SEC), including the risk factors set forth therein. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and we undertake no obligation to update any forward-looking statement in this press release, except as required by law.

    Contacts:

    FibroGen, Inc.

    Investors:

    Michael Tung, M.D.

    Investor Relations

    +1.415.978.1434

    Media:

    Jennifer Harrington

    +1.610.574.9196


    1 American Cancer Society. What Are Myelodysplastic Syndromes? Available at https://www.cancer.org/cancer/myelodysplastic-syndrome/about/what-is-mds.html. Accessed November 2020.

    2 Abu-Zeinah G, DeSancho MT. Understanding Sideroblastic Anemia: An Overview of Genetics, Epidemiology, Pathophysiology and Current Therapeutic Options. J Blood Med. 2020;11:305-318. Published 2020 Sep 25. doi:10.2147/JBM.S232644

    3 Ma X, Does M, Raza A, Mayne ST. Myelodysplastic syndromes: incidence and survival in the United States. Cancer. 2007;109(8):1536.

    4 Cogle CR. Incidence and Burden of the Myelodysplastic Syndromes. Curr Hematol Malig Rep. 2015;10(3):272-281.



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  4. NEW YORK, Nov. 25, 2020 (GLOBE NEWSWIRE) -- Renalytix AI plc (LSE: RENX) (NASDAQ:RNLX), an artificial intelligence-enabled in vitro diagnostics company, focused on optimizing clinical management of kidney disease to drive improved patient outcomes and advance value-based care, today reported financial results for the quarter ended September 30, 2020.

    Recent Highlights

    • Launched KidneyIntelX within the Mount Sinai Health System
    • Submitted final package to FDA seeking clearance of KidneyIntelX
    • Announced collaboration with AstraZeneca to develop and launch precision medicine strategies for cardiovascular, renal and metabolic diseases
    • Completed spin-out of Verici Dx (previously FractalDx)
    • Achieved dual listing on Nasdaq Global Market

    First Quarter

    NEW YORK, Nov. 25, 2020 (GLOBE NEWSWIRE) -- Renalytix AI plc (LSE: RENX) (NASDAQ:RNLX), an artificial intelligence-enabled in vitro diagnostics company, focused on optimizing clinical management of kidney disease to drive improved patient outcomes and advance value-based care, today reported financial results for the quarter ended September 30, 2020.

    Recent Highlights

    • Launched KidneyIntelX within the Mount Sinai Health System
    • Submitted final package to FDA seeking clearance of KidneyIntelX
    • Announced collaboration with AstraZeneca to develop and launch precision medicine strategies for cardiovascular, renal and metabolic diseases
    • Completed spin-out of Verici Dx (previously FractalDx)
    • Achieved dual listing on Nasdaq Global Market

    First Quarter 2021 Financial Results

    Operating expense for the three months ended September 30, 2020 was $5.4 million compared to $2.0 million during the prior year period.

    Research and development expenses were 1.7 million for the three months ended September 30, 2020, increasing $0.5 million from $1.2 million for the three months ended September 30, 2019. The increase R&D expense was due to increased headcount and the associated compensation and related benefits, including share-based payments.

    General and administrative expenses were $4.1 million for the three months ended September 30, 2020, increasing $3.3 million from $0.8 million for the three months ended September 30, 2019. The increase was due to an increase in insurance costs, compensation and related benefits, including share-based payments, due to increased headcount, and in fees associated with the listing on Nasdaq.

    Net loss attributable to ordinary shareholders was $7.2 million for the three months ended September 30, 2020 compared to $1.5 million in the prior year period.

    Cash, cash equivalents and short-term investments of $82.3 million as of September 30, 2020. This includes $76.1 million from the Company's initial public offering on the Nasdaq Global Market after commissions, fees and offering expenses.

    For further information, please contact:

    Renalytix AI plc www.renalytixai.com
    James McCullough, CEOVia Walbrook PR
      
    Stifel (Nominated Adviser, Joint Broker)Tel: 020 7710 7600
    Alex Price / Nicholas Moore 
      
    Investec Bank plc (Joint Broker)Tel: 020 7597 4000
    Gary Clarence / Daniel Adams 
      
    Walbrook PR LimitedTel: 020 7933 8780 or 
    Paul McManus / Lianne CawthorneMob: 07980 541 893 / 07584 391 303
      

    About Kidney Disease

    Kidney disease is now recognized as a public health epidemic affecting over 850 million people globally. The Centers for Disease Control and Prevention (CDC) estimates that 15% of US adults, or 37 million people, currently have chronic kidney disease (CKD).  Further, the CDC reports that 9 out of 10 adults with CKD do not know they have it and 1 out of 2 people with very low kidney function who are not on dialysis do not know they have CKD*. Kidney disease is referred to as a "silent killer" because it often has no symptoms and can go undetected until a very advanced stage.  Each year kidney disease kills more people than breast and prostate cancer. Every day, 13 patients in the United States die while waiting for a kidney transplant.

    * https://www.cdc.gov/kidneydisease/publications-resources/2019-national-facts.html

    About RenalytixAI

    RenalytixAI is a developer of artificial intelligence-enabled clinical in vitro diagnostic solutions for kidney disease, one of the most common and costly chronic medical conditions globally. RenalytixAI's products are being designed to make significant improvements in kidney disease diagnosis, transplant management, clinical care, patient stratification for drug clinical trials, and drug target discovery. For more information, visit www.renalytixai.com.

    Forward Looking Statements

    Statements contained in this release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the ability of KidneyIntelX to lower healthcare costs, improve patient quality of life and set a long-term standard of care, trends in our market and potential benefits of government policy change, the impact of COVID-19 on our business, our expectations for product development, strategic partnerships and collaborations, reimbursement decisions, clinical studies and regulatory submissions, and our business strategies and future growth. Words such as "anticipates," "believes," "estimates," "expects," "intends," "plans," "seeks," and similar expressions are intended to identify forward-looking statements. We may not actually achieve the plans and objectives disclosed in the forward-looking statements, and you should not place undue reliance on our forward-looking statements. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. These risks and uncertainties include, among others: that KidneyIntelX is based on novel artificial intelligence technologies that are rapidly evolving and potential acceptance, utility and clinical practice remains uncertain; we have only recently commercially launched KidneyIntelX; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in our filings with the SEC, including the "Risk Factors" section of our Annual Report. All information in this release is as of the date of the release, and we undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law.

    Investor Contact

    RENALYTIX AI PLC

    Operational Update and Financial Results for the Three Months Ended September 30, 2020

    Unless otherwise indicated, all references in this report, to the terms "Renalytix," "Renalytix AI," "Renalytix AI plc," "the company," "we," "us" and "our" refer to Renalytix AI plc together with its subsidiaries. We recommend that you read the discussion below together with our audited financial statements and the notes thereto, which appear in our Annual Report on Form 20-F for the year ended June 30, 2020, filed with the Securities and Exchange Commission on October 28, 2020 (our "Annual Report").

    The statements in this discussion regarding our expectations regarding our market opportunity and future performance, as well as all other non-historical statements are forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These risks and uncertainties include, but are not limited to, the risks and uncertainties set forth in the "Risk Factors" section of our Annual Report and any subsequent reports that we file with the SEC. See the section titled "Forward-Looking Statements" above.

    OPERATIONAL REVIEW

    Company Overview

    We are an artificial intelligence-enabled in vitro diagnostics company, focused on optimizing clinical management of kidney disease to drive improved patient outcomes and lower healthcare costs. KidneyIntelX, our first-in-class diagnostic platform, employs a proprietary artificial intelligence-enabled algorithm that combines diverse data inputs, including validated blood-based biomarkers, inherited genetics and personalized patient data from electronic health record ("EHR") systems, to generate a unique patient risk score. This patient risk score enables prediction of progressive kidney function decline in chronic kidney disease ("CKD") allowing physicians and healthcare systems to optimize the allocation of treatments and clinical resources to patients at highest risk. CKD affects approximately 37 million individuals in the United States, significantly impacting their quality of life and, according to the United States Renal Data System's 2019 Annual Data Report, resulting in Medicare spending of over $120 billion per year. In response to this substantial kidney disease burden, a U.S. Presidential Executive Order on Advancing American Kidney Health was issued in July 2019 to support change in kidney disease care. We believe we are well-positioned to help meet this urgent medical need with KidneyIntelX, a laboratory developed test ("LDT"), initially indicated for adult patients with type 2 diabetes and existing CKD, which is referred to as diabetic kidney disease ("DKD"). KidneyIntelX has already been granted a common procedural terminology ("CPT code"), national Medicare pricing and a positive coverage determination from a regional, private physician-led health insurance payor. Further, it has been granted breakthrough device designation from the U.S. Food and Drug Administration (the "FDA"). Building on these significant reimbursement and regulatory milestones, we believe our population health-based business model, which includes partnerships with healthcare systems, such as Mount Sinai Health System, will help facilitate commercial adoption of KidneyIntelX in the United States.

    Kidney disease is a worldwide public health crisis, resulting in more deaths per year than breast or prostate cancer. The National Kidney Foundation estimates that one-third of adults in the United States are at risk of developing kidney disease. Advanced kidney disease is generally not reversible and, once the disease progresses to kidney failure, the only available treatments are long-term dialysis and kidney transplant. In 2016, more than 726,000 patients had end-stage kidney disease ("ESKD"), with more than 500,000 requiring dialysis at least three times a week. More than 100,000 patients begin dialysis each year to treat ESKD. Once on dialysis, patients typically experience a five-year mortality rate of up to 65%, about the equivalent rate for brain cancer. As of July 2019, nearly 100,000 Americans were on the waiting list to receive a kidney transplant and 13 patients die in the United States while waiting for a kidney transplant every day. Moreover, the kidney disease crisis is continuing to grow along with the increased prevalence of contributing risk factors, such as obesity and diabetes.

    Managing a CKD population of this scale and associated healthcare costs presents a unique social challenge. The ability to predict which patients will experience progressive kidney function decline, kidney failure, initiation of long-term dialysis or kidney transplant, is critical to changing patient outcomes and health economics. In our clinical validation studies in patients with DKD, we observed that the Kidney Disease: Improving Global Outcomes (KDIGO) classification system, which is the standard clinical assessment to predict risk for progression of CKD, including DKD, only identified approximately 20% of patients that experienced an adverse kidney outcome as very high-risk patients with the recommendation of referral to a nephrologist, while KidneyIntelX identified nearly half of such patients.

    We believe that the utilization of KidneyIntelX across large patient populations will have a significant impact on overall healthcare costs. Health economic benefits are projected to be derived from three key areas: (1) slowing progression to the next stage of CKD, (2) delaying or preventing progression to ESKD and the need for dialysis or kidney transplant and (3) avoiding dialysis crashes. We have partnered with Boston Healthcare Associates, or BHA, to develop a health economic model analyzing the cost and care pathway for patients with DKD at all stages of the disease and the potential cost savings of implementing and utilizing KidneyIntelX. According to the BHA study, based on the Medicare price of $950 per reportable test, KidneyIntelX would generate a positive return for health insurers in under 24 months and deliver a cost savings of up to $1.3 billion over five years per 100,000 patients with DKD.

    Several federal policy and economic events, including the U.S. Presidential Executive Order on Advancing American Kidney Health issued in July 2019 and recent changes in U.S. reimbursement law, are helping disrupt the kidney disease clinical and commercial environment, highlighting the pressing need for solutions such as KidneyIntelX. We believe these favorable policy trends, which began during the Obama administration, will continue to build under a Biden administration and will support broader commercial adoption of KidneyIntelX and other derivative products contemplated in our diagnostics development planning. In addition, in August 2020, the U.S. Centers for Medicare & Medicaid Services ("CMS"), an agency within the U.S. Department of Health and Human Services, submitted for public comment a rule ("Medicare Coverage of Innovative Technology") which, if finalized, would provide an automatic National Medicare Coverage Determination for diagnostic devices that have received breakthrough device designation upon the effective date of the promotional approval by the FDA. The automatic coverage period shall continue for a period of four years, during which manufacturers of breakthrough devices may develop additional evidence regarding the applicability of their products to the Medicare population, so they might continue Medicare coverage beyond the initial four years. We believe that this new proposed CMS rule making, if adopted in its current form, could have a material positive impact on addressable market population with insurance coverage for KidneyIntelX if we obtain FDA clearance for KidneyIntelX.



    Business Highlights

    Launch of KidneyIntelX at Mount Sinai

    In September 2020, we announced the initiation of KidneyIntelX clinical test reporting within the Mount Sinai Health System ("Mount Sinai") in New York City. In addition to patient testing and risk assessment, a central component of this operational milestone was the physician education and support program developed in close collaboration with leadership of the Mount Sinai Departments of Medicine and Population Health Science and Policy, with input from patient advocacy groups and the broader clinical community. This expert experience is reflected in the design of the KidneyIntelX test report and the newly launched product website, www.kidneyintelx.com. We believe this education and support program will be an important resource to help improve care for early stage DKD patients at Mount Sinai and support future deployments of KidneyIntelX.

    Submission to FDA seeking clearance of KidneyIntelX

    In August 2020, we filed a submission seeking clearance of KidneyIntelX with the FDA. This FDA filing builds on our regulatory and commercialization program, which includes our June 2020 announcement that the New York State Department of Health has issued a clinical laboratory permit for commercial clinical testing of KidneyIntelX. In May 2019, we announced that KidneyIntelX was granted breakthrough device designation by FDA, the first such designation for an artificial intelligence-enabled in vitro diagnostic for kidney disease publicly announced by any company. We are now seeking FDA clearance for the intended use of KidneyIntelX, in conjunction with clinical evaluation, as an aid to further assess the risk of progressive decline in kidney function within a period of up to five years in patients over the age of 21 with type 2 diabetes and existing CKD. Patients with CKD and type 2 diabetes account for approximately 25-30% of the estimated 37 million U.S. patients with CKD. Performance data we provided in our FDA 510(k) submission was based on a multi-center validation study of more than 1,100 patients that demonstrated that KidneyIntelX accurately identifies patients with type 2 diabetes in CKD stages 1, 2 and 3 who are at highest risk of progressive decline in kidney function and/or kidney failure.

    COVID-19 studies

    The current COVID-19 pandemic has had a devastating impact around the world. Many reports indicate that acute kidney injury occurs in approximately 20% to 40% of patients hospitalized with COVID-19, is often severe (including need for acute dialysis), and data from Mount Sinai during the initial U.S. surge indicated that 70% of patients that develop acute kidney injury in the setting of COVID-19 either die in the hospital or do not recover kidney function by discharge. We plan to investigate the use of KidneyIntelX for patients with COVID-19 in two clinical studies. The first study, entitled "Pred-MAKER" (Prediction of Major Adverse Kidney Events and Recovery) involves acutely ill patients with COVID-19 admitted to Mount Sinai. The second study, "MASKeD-COVID" (Multi-center Assessment of Survivors for KidneDisease after COVID-19) is designed to understand the long-term kidney epidemiology of CKD in survivors of COVID-19 and validate KidneyIntelX for prediction of long-term kidney outcomes post-COVID hospitalization that will inform further prevention, treatment and clinical care.

    AstraZeneca collaboration

    In August 2020, we announced a collaboration with AstraZeneca (NASDAQ:AZN) to develop and launch precision medicine strategies for cardiovascular, renal, and metabolic diseases. The first stage in the collaboration is examining the uptake of, and patient adherence to, treatments for diabetes as well as common complications of CKD, including hyperkalemia and anemia. The study will provide key insights into the impact of the KidneyIntelX platform to optimize utilization of therapeutics in CKD under current standard of care protocols. Based on the insights gained from the first stage, a multi-center, randomized controlled trial will be initiated to evaluate the impact of KidneyIntelX testing and care navigation software on uptake and adherence to new potassium-binding agents in patients with CKD and hyperkalemia. We believe that this approach will accomplish the following: (1) help improve physician uptake and patient adherence to existing potassium-binding therapeutics and other approved products in CKD through early identification of previously hidden high-risk patient groups; (2) accelerate patient identification and recruitment for clinical trials; and (3) complement commercialization efforts with outcomes from KidneyIntelX results. Importantly, this collaboration extends the potential impact of KidneyIntelX to populations beyond the first indicated use, DKD, that is approved with New York State and under breakthrough review with the FDA. Hyperkalemia affects approximately 10-20% of patients with CKD or chronic heart failure. Anemia affects 15% of patients with CKD, and nearly 50% of individuals with advanced CKD.

    FractalDx (Verici Dx) spin-off

    In April 2020, the Company created a wholly-owned subsidiary, Verici Dx Limited ("Verici Dx"), to hold technology in-licensed from the Icahn School of Medicine at Mount Sinai in late 2018. In May 2020, the Company transferred the in-licensed FractalDx technology and associated assets to Verici Dx in exchange for $2.0 million, which was satisfied by the issuance of convertible loan notes of Verici Dx to the Company.

    We announced on July 8, 2020 that the share capital of Verici Dx had been re-designated into 59,416,134 A Shares of £0.001 each and one golden share of £0.001 (the "Golden Share") and that Renalytix would retain the Golden Share and its associated controlling voting rights. Subsequent to that announcement, the Company entered into a declaration of trust whereby Renalytix AI plc has declared that it holds the Golden Share as nominee and on trust for certain Directors of RenalytixAI and accordingly, the Company itself has no ongoing beneficial interest in Verici Dx shares. This triggered a reconsideration event for ongoing consolidation of Verici Dx and since the Company was still the primary funding source for Verici Dx, the Company continued to hold a controlling financial interest in Verici Dx and continued to consolidate Verici Dx. Consequently, the Company recognized noncontrolling interest of $1.6 million to reflect Verici Dx's distribution of A shares and the Golden Share.

    On November 3, 2020, Verici Dx completed its initial public offering (the "Verici IPO") on AIM thus triggering another reconsideration event for ongoing consolidation of Verici Dx. The Verici IPO resulted in the Company no longer having a controlling financial interest and no longer having a majority equity interest in Verici Dx.

    Nasdaq dual listing

    In July 2020, we completed a dual listing on the Nasdaq Global Market through the issuance of American Depository Shares under ticker symbol "RNLX," expanding our institutional investor base and raising net capital of approximately $76.1 million after commissions, fees and offering expenses. We maintain our listing on the AIM market of London Stock Exchange plc under the symbol "RENX."

    Impact of COVID-19

    The extent of the impact of the COVID-19 pandemic on our business, operations and regulatory and commercialization timelines will depend on certain developments, including the duration and spread of the outbreak and its impact on our partners, laboratory sites, and other third parties with whom we do business, as well as its impact on regulatory authorities and our key scientific and management personnel. For example, to the extent possible, we are conducting business as usual, with necessary or advisable modifications to employee travel and employee work locations. We will continue to actively monitor the rapidly evolving situation related to COVID-19 and may take further actions that alter our business operations, including those that may be required by federal, state or local authorities, or that we determine are in the best interests of our employees, partners and shareholders. At this point, the extent to which the COVID-19 pandemic may impact our business, operations and regulatory and commercialization timelines remains uncertain.

    FINANCIAL REVIEW

    Financial review of the three-month period ended September 30, 2020

    The operating loss for the three months ended September 30, 2020, was $5.4 million (September 30, 2019: $2.0 million) and the net loss attributable to ordinary shareholders for the three months ended September 30, 2020, was $7.2 million (September 30, 2019: loss of $1.5 million).

    Research and Development Costs

    Research and development expenses increased by $0.5 million, from $1.2 million for the three months ended September 30, 2019 to $1.7 million for the three months ended September 30, 2020. The increase R&D expense was due to increased headcount and the associated compensation and related benefits, including share-based payments.

    General and Administrative Costs

    General and administrative expenses increased by $3.3 million, from $0.8 million for the three months ended September 30, 2019 to $4.1 million for the three months ended September 30, 2020. The increase was due to a $1.0 million increase in insurance costs, $0.8 million increase in legal and accounting fees as a result of listing on Nasdaq, $0.6 million in compensation and related benefits, including share-based payments, due to increased headcount, $0.4 million increase in consulting and professional fees, $0.3 million increase in recruiting expense, and an increase of $0.2 million in marketing, facility and other operating expenses.

    Performance of contract liability to affiliate

    In May 2020, the Company and the Icahn School of Medicine at Mount Sinai entered into an operating agreement ("Kantaro Operating Agreement") to form a joint venture, Kantaro Biosciences LLC ("Kantaro"), for the purpose of developing and commercializing laboratory tests for the detection of antibodies against SARS-CoV-2 originally developed by Mount Sinai. During the three months ended September 30, 2020, we recognized $0.5 million related to the performance of our contract liability with Kantaro. This represents the allocation of costs related to performing services on behalf of Kantaro.

    Equity Losses in Affiliate

    As the Company can exert significant influence over, but does not control, the investee's operations through voting rights or representation on Kantaro's board of directors, the Company accounts for the investment using the equity method of accounting. During the three months ended September 30, 2020, we recognized $0.1 million in losses which represents our proportionate share of losses in Kantaro.

    Other Income (Expense), net

    During the three months ended September 30, 2020, we recognized a realized foreign exchange gain of $0.06 million which was offset by an unrealized foreign exchange loss of $2.2 million. During the three months ended September 30, 2019, we received $0.08 million of other income in relation to a collaboration with the University Medical Center Groningen, Netherlands as well as $0.01 million of interest income as a result of interest earned on cash deposits. We recognized a realized foreign exchange gain of $0.02 million during the three months ended September 30, 2019 and had an unrealized foreign exchange gain of $0.43 million.

    Cash Flows

    Net cash used in operating activities

    During the three months ended September 30, 2020, net cash used in operating activities was $10.4 million and was primarily attributable to our $7.6 million net loss and $3.6 million in the net change in our operating assets and liabilities that was offset by $0.8 million in noncash charges. The change in our operating assets and liabilities was primarily attributable to $3.8 million decrease in our prepaid expenses and other current assets. Noncash charges were primarily related to share-based compensation expense of $0.5 million.

    During the three months ended September 30, 2019, net cash used in operating activities was $1.7 million and was primarily attributable to our $1.5 million net loss and $0.2 million in noncash charges.

    Net cash used in investing activities

    During the three months ended September 30, 2020, net cash provided by investing activities was $0.4 million and primarily attributable to $1.0 million in proceeds in short term investments offset by $0.5 million for the purchase of lab and office equipment and $0.1 million of software development costs.

    During the three months ended September 30, 2019, net cash used in investing activities was $13.3 million and primarily attributable to $14.3 million in purchases of short-term investments offset by net proceeds of $1.0 million related to our short-term investments.

    Net cash used in financing activities

    During the three months ended September 30, 2020, net cash provided by financing activities was $76.9 million and was primarily attributable to $79.2 million of proceeds from our initial public offering on the Nasdaq Global Market which was offset by offering costs of $2.3 million associated with the IPO that were paid in the period.

    During the three months ended September 30, 2019, net cash provided by financing activities was $16.4 million and was primarily attributable to $17.3 million of proceeds from our secondary public offering on the AIM which was offset by offering costs of $0.9 million associated with the public offering.

    Cash, cash equivalents and short-term investments

    Net cash, cash equivalents and short-term investments of $82.3 million as of September 30, 2020 increased from $13.3 million as of June 30, 2020 primarily due to the net proceeds of our initial public offering on the Nasdaq Global Market.

    Post-period end

    On November 3, 2020, Verici Dx completed its IPO on AIM and raised gross proceeds of £14.5 million. Verici Dx previously issued the Company $2.5 million in convertible loan notes which reflects the consideration for the FractalDx assets and the funding the Company provided Verici Dx through October 28, 2020. Prior to the Verici IPO, on November 3, 2020, the Company gave notice to convert the existing $2.5 million convertible loan notes into 9,831,681 ordinary shares of Verici Dx.





    RENALYTIX AI PLC

    CONDENSED CONSOLIDATED BALANCE SHEETS (UNAUDITED)

    (in thousands, except share and per share data)September

    30, 2020
    June 30,

    20
    20
       
    Assets  
    Current assets:  
    Cash and cash equivalents$82,253 $13,293 
    Short-term investments   982 
    Prepaid expenses and other current assets 4,378  551 
    Receivable from affiliate 18  18 
    Total current assets 86,649  14,844 
    Property and equipment, net 2,644  1,655 
    Deferred offering costs   2,364 
    Investment in affiliate 1,821  1,937 
    Note receivable from affiliate 83  83 
    Total assets$91,197 $20,833 
           
    Liabilities and Shareholders' Equity  
    Current liabilities:  
    Accounts payable$1,829 $2,218 
    Accrued expenses and other current liabilities 636  683 
    Note payable – current 161  120 
    Payable to affiliate - current 1,183  271 
    Total current liabilities 3,809  3,292 
    Payable to affiliate - noncurrent 173  1,544 
    Note payable - noncurrent 94  135 
    Total liabilities 4,076  4,971 
           
    Commitments and contingencies (Note 9)  
    Shareholders' equity:  
    Ordinary shares, £0.0025 par value per share: 75,438,492 and 62,444,992 shares authorized

       at September 30, 2020 and June 30, 2020, respectively; 72,029,634 and 59,416,134

       shares issued and outstanding at September 30, 2020 and June 30, 2020, respectively
     219  179 
    Additional paid-in capital 147,883  69,650 
    Accumulated other comprehensive income (loss) 1,030  (1,200)
    Accumulated deficit (59,938) (52,717)
    Total shareholders' equity attributable to Renalytix AI 89,194  15,912 
    Noncontrolling interest (2,073)  
    Total shareholders' equity 87,121  15,912 
    Total liabilities and shareholders' equity$91,197 $20,833 

    The accompanying notes are an integral part of these condensed consolidated financial statements.





    RENALYTIX AI PLC

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

    (UNAUDITED)

    (in thousands, except share data)Three Months

    Ended


    September 30,

    2020
    Three Months

    Ended


    September 30,

    2019
       
    Operating expenses:  
    Research and development$1,745 $1,180 
    General and administrative 4,116  837 
    Performance of contract liability to affiliate (458)  
    Total operating expenses and loss from operations (5,403) (2,017)
       
    Equity in losses of affiliate (116)  
    Other (expense) income, net (2,095) 546 
    Net loss (7,614) (1,471)
    Net loss attributable to noncontrolling interest (393)  
    Net loss attributable to ordinary shareholders (7,221) (1,471)
    Other comprehensive income (loss):  
    Foreign exchange translation adjustment 2,255  (622)
    Comprehensive loss (5,359) (2,093)
    Comprehensive loss attributable to noncontrolling interest (67)  
    Comprehensive loss attributable to Renalytix AI$(5,292)$(2,093)
       
    Net loss per ordinary share—basic and diluted$(0.10)$(0.03)
    Weighted average ordinary shares—basic and diluted 69,835,982  58,077,004 
      

    The accompanying notes are an integral part of these condensed consolidated financial statements.  





    RENALYTIX AI PLC

    CONDENSED CONSOLIDATED STATEMENTS OF SHAREHOLDERS' EQUITY (UNAUDITED)

     Ordinary shares

    Additional

    paid-in

    capital

    Accumulated other comprehensive income (loss)

    Accumulated deficit

    Total

    shareholders' (deficit) equity attributable to Renalytix AI

    Noncontrolling interests

    Total

    shareholders' (deficit) equity

    (in thousands, except share

    and per share data)
    SharesAmount
    Balance at June 30, 202059,416,134$179$69,650$(1,200)$(52,717)15,912  $15,912 
    Sale of ordinary shares in

       initial public offering on

       Nasdaq, net of offering

       costs and underwriting

       fees of $9,007
    12,613,500 40 76,094    76,134   76,134 
    Verici distribution in specie   1,638 (25)  1,613 (1,613) —  
    Share-based compensation expense  501    501   501 
    Currency translation adjustments   2,255   2,255 (67) 2,188 
    Net loss     (7,221)(7,221)(393) (7,614

    )
    Balance at September 30, 202072,029,634$219$147,883$1,030 $(59,938)89,194 (2,073)$87,121 



     Ordinary shares

    Additional

    paid-in

    capital

    Accumulated other comprehensive income (loss)

    Accumulated deficit

    Total

    shareholders' (deficit) equity attributable to Renalytix AI

    Noncontrolling interests

    Total

    shareholders' (deficit) equity

    (in thousands, except share

    and per share data)
    SharesAmount
    Balance at June 30, 201953,816,134$162$52,084$(822)$(42,873)8,551 $8,551 
    Sale of ordinary shares in

       secondary offering, net of

       offering costs of $842
    5,600,000 17 16,407    16,424  16,424 
    Share-based compensation expense  247    247  247 
    Currency translation adjustments   (622)  (622) (622)
    Net loss     (1,471)(1,471) (1,471)
    Balance at September 30, 201959,416,134$179$68,738$(1,444)$(44,344)23,129 $23,129 





    RENALYTIX AI PLC

    CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (UNAUDITED)

    (in thousands)Three Months

    Ended

    September 30,

    2020
    Three Months

    Ended

    September 30,

    2019
       
    Cash flows from operating activities:  
    Net loss$(7,614)$(1,471)
    Adjustments to reconcile net loss to net cash used in operating activities  
    Depreciation 27  9 
    Share-based compensation 501  247 
    Realized gain on short-term investments (18) (6)
    Equity losses in affiliate 116   
    Unrealized foreign exchange loss (gain) 178  (415)
    Changes in operating assets and liabilities:  
    Prepaid expenses and other current assets (3,845) 80 
    Accounts payable 810  83 
    Accrued expenses and other current liabilities (111) (207)
    Payable to affiliate (459)  
    Net cash used in operating activities (10,415) (1,680)
       
       
    Cash flows from investing activities:  
    Purchases of property and equipment (441) (31)
    Software development costs (122)  
    Purchase of short-term investments   (14,290)
    Proceeds from short-term investments 1,000  1,000 
    Net cash provided by (used in) investing activities 437  (13,321)
       
       
    Cash flows from financing activities:  
    Gross proceeds from the issuance of ordinary shares, net of underwriting fees 79,182   
    Gross proceeds from the issuance of ordinary shares   17,276 
    Payment of offering costs (2,304) (851)
    Net cash provided by financing activities 76,878  16,425 
    Effect of exchange rate changes on cash 2,060  (233)
    Net increase in cash and cash equivalents 68,960  1,191 
    Cash and cash equivalents, beginning of period 13,293  8,201 
    Cash and cash equivalents, end of period$82,253 $9,392 
       
    Supplemental noncash financing activities:  
    Financing costs in accounts payable and accrued expenses$1 $ 
    Software development costs in accounts payable and accrued expenses$311 $427 
    Purchase of property and equipment in accounts payable and accrued expenses$177 $ 

    The accompanying notes are an integral part of these condensed consolidated financial statements.  





    RENALYTIX AI PLC

    NOTES TO UNAUDITED INTERIM CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

    1. Business and risks

    Renalytix AI plc and its wholly-owned subsidiary, Renalytix AI, Inc., (collectively, Renalytix AI, or the Company) is an artificial intelligence-enabled in vitro diagnostics company, focused on optimizing clinical management of kidney disease to drive improved patient outcomes and significantly lower healthcare costs. KidneyIntelX, the Company's first-in-class diagnostic platform, employs a proprietary artificial intelligence-enabled algorithm that combines diverse data inputs, including validated blood-based biomarkers, inherited genetics and personalized patient data from EHR systems, to generate a unique patient risk score.

    Since inception in March 2018, the Company has focused primarily on organizing and staffing the Company, raising capital, developing the KidneyIntelX platform, conducting clinical validation studies for KidneyIntelX, establishing and protecting its intellectual property portfolio and commercial laboratory operations, pursuing regulatory clearance and developing a reimbursement strategy. To date, the Company has not generated any revenue from the sales of KidneyIntelX tests. The Company has funded its operations primarily through equity financings.

    In April 2020, the Company created a wholly-owned subsidiary, Verici Dx Limited ("Verici Dx"), to hold technology in-licensed from the Icahn School of Medicine at Mount Sinai (ISMMS or Mount Sinai) in late 2018. In May 2020, the Company transferred the in-licensed FractalDx technology and associated assets to Verici Dx in exchange for $2.0 million, which was satisfied by the issuance of convertible loan notes of Verici Dx to the Company. The reduction of capital necessary to implement this transaction was approved by the Company's shareholders at a general meeting held on May 15, 2020 and confirmed by the High Court in England and Wales on June 9, 2020. The Company's board of directors declared the distribution of shares of Verici Dx to the then shareholders of the Company, to effect the FractalDx spin-off, on July 7, 2020, and the distribution occurred on July 10, 2020.

    The Company announced on July 8, 2020 that the share capital of Verici Dx had been re-designated into 59,416,134 A Shares of £0.001 each and one golden share of £0.001 (the "Golden Share") and that Renalytix would retain the Golden Share and its associated controlling voting rights. Subsequent to that announcement, the Company entered into a declaration of trust whereby Renalytix AI plc has declared that it holds the Golden Share as nominee and on trust for certain Directors of Renalytix AI and accordingly, the Company itself has no ongoing beneficial interest in Verici Dx shares. This triggered a reconsideration event for ongoing consolidation of Verici Dx and since the Company was still the primary funding source for Verici Dx, the Company continued to hold a controlling financial interest in Verici Dx and continued to consolidate Verici Dx. Consequently, the Company recognized noncontrolling interest of $1.6 million to reflect Verici Dx's distribution of A shares and the Golden Share.

    As discussed in Note 14, on October 28, 2020, the Company gave notice to convert the outstanding $2.5 million convertible loan notes, which reflects the consideration for the FractalDx assets and the funding the Company provided Verici Dx through October 28, 2020, into 9,831,681 ordinary shares of Verici Dx. On November 3, 2020, Verici Dx completed an initial public offering ("Verici IPO") on AIM thus triggering another reconsideration event for ongoing consolidation of Verici Dx. The Verici IPO resulted in the Company no longer having a controlling financial interest and no longer having a majority equity interest in Verici Dx.

    The Company is subject to risks and uncertainties common to early-stage companies in the diagnostics industry, including, but not limited to, ability to secure additional capital to fund operations, compliance with governmental regulations, development by competitors of new technological innovations, dependence on key personnel and protection of proprietary technology. To achieve widespread usage, KidneyIntelX and additional diagnostic products currently under development will require extensive clinical testing and validation prior to regulatory approval and commercialization. These efforts require significant amounts of additional capital, adequate personnel, and infrastructure and extensive compliance-reporting capabilities.

    2. Going Concern

    On November 6, 2018, the Company sold 18.4 million ordinary shares in its initial public offering, or IPO, at $1.57 per share resulting in net proceeds of approximately $27.4 million and its ordinary shares were admitted to trading on the AIM.

    In July 2019, the Company sold 5.6 million of its ordinary shares to several new and existing investors in exchange for $16.4 million of net cash proceeds.

    In July 2020, the Company closed an IPO on Nasdaq Global Market in which the Company issued and sold 12.6 million ordinary shares, which converted into 6.3 million American depository shares, at a public offering price of $13.50 per share. In addition, the Company completed a concurrent private placement in Europe and other countries outside of the United States of 30,000 ordinary shares at a price of £5.37 per ordinary share (at an exchange rate of GBP:USD 1:1.2563). The Company received net proceeds of approximately $76.1 million as a result of the offering.

    The Company has incurred recurring losses and negative cash flows from operations since inception and had an accumulated deficit of $59.9 million as of September 30, 2020. The Company anticipates incurring additional losses until such time, if ever, that it can generate significant sales of KidneyIntelX or any future products currently in development. Management believes its cash and cash equivalents of $82.3 million as of September 30, 2020, are sufficient to fund the projected operations for at least the next twelve months from the issuance date of these financial statements. Substantial additional capital will be needed by the Company to fund its operations, expand its commercial activities and develop other potential diagnostic related products.

    The Company plans to seek additional funding through public or private equity offerings, debt financings, other collaborations, strategic alliances and licensing arrangements. The Company may not be able to obtain financing on acceptable terms, or at all, and the Company may not be able to enter into strategic alliances or other arrangements on favorable terms, or at all. The terms of any financing may adversely affect the holdings or the rights of the Company's shareholders. If the Company is unable to obtain funding, the Company could be required to delay, curtail or discontinue research and development programs, product portfolio expansion or future commercialization efforts, which could adversely affect its business prospect.

    3. Basis of presentation and summary of significant accounting policies

    The accompanying unaudited interim condensed consolidated financial statements have been prepared in conformity with generally accepted accounting principles in the United States ("U.S. GAAP"). Any reference in these notes to applicable guidance is meant to refer to U.S. GAAP as found in the Accounting Standards Codification ("ASC") and Accounting Standards Updates ("ASU") of the Financial Accounting Standards Board ("FASB").

    In the opinion of management, the accompanying unaudited interim condensed consolidated financial statements include all normal and recurring adjustments (which consist primarily of accruals and estimates that impact the financial statements) considered necessary to present fairly the Company's financial position as of September 30, 2020 and its results of operations and cash flows for the three months ended September 30, 2020 and 2019. Operating results for the three months ended September 30, 2020 are not necessarily indicative of the results that may be expected for the year ending June 30, 2021. The unaudited interim financial statements, presented herein, do not contain the required disclosures under U.S. GAAP for annual financial statements. The accompanying unaudited interim condensed consolidated financial statements should be read in conjunction with the annual audited consolidated financial statements and related notes as of and for the year ended June 30, 2020.

    Principles of consolidation

    The unaudited interim condensed consolidated financial statements include the accounts of Renalytix AI plc, its wholly-owned subsidiary, Renalytix AI, Inc., and Verici Dx Limited in which the Company holds a controlling financial interest as of the financial statement date. As the Company has been the primary funding source for Verici Dx since its distribution to the Company's stockholders, the operations and financial position of Verici Dx are included in the condensed consolidated financial statements of the Company. Participation of the stockholders in the net assets and losses of Verici Dx are reflected in the line items "Noncontrolling interests" in the Company's condensed consolidated balance sheets and "Net loss attributable to the noncontrolling interests" in the Company's condensed consolidated statements of operations and comprehensive loss. Noncontrolling interests adjusts the Company's condensed consolidated results of operations and comprehensive loss to exclude all of the losses of Verici Dx as Renalytix AI has no direct equity ownership in Verici Dx. Changes in the underlying net book value of Verici Dx due to equity issuances are reflected as equity transaction in the Company's condensed consolidated statements of stockholders' equity. All inter-company balances and transactions have been eliminated in consolidation.

    Use of estimates

    The preparation of the condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements and reported amounts of expenses during the reporting period. Actual results could differ from those estimates. Due to the uncertainty of factors surrounding the estimates or judgments used in the preparation of the condensed consolidated financial statements, actual results may materially vary from these estimates.

    Estimates and assumptions are periodically reviewed and the effects of revisions are reflected in the condensed consolidated financial statements in the period they are determined to be necessary. Significant areas that require management's estimate include the assumptions used in determining the fair value of share-based awards, the value of consideration for the acquired in-process research and development and in recording the prepaid/accrual, and associated expense, for research and development activities performed for the Company by third parties.

    Segment information

    The Company manages its operations as a single operating segment for the purposes of assessing performance and making operating decisions. The Company's singular focus is to make significant improvements in kidney disease diagnosis and prognosis, clinical care, patient stratification for drug clinical trials, and drug target discovery. 

    Foreign currency

    The Company's condensed consolidated financial statements are presented in U.S. dollars, the reporting currency of the Company. The functional currency of Renalytix AI plc and Verici Dx Limited is GB Pounds. The functional currency of Renalytix AI, Inc. and Verici Dx Inc. is the U.S. dollar. Assets and liabilities of Renalytix AI plc and Verici Dx Limited are translated at the rate of exchange at year-end, while the statements of operations are translated at the weighted average exchange rates in effect during the reporting period. The net effect of these translation adjustments is shown as a component of accumulated other comprehensive income (loss). Transaction gains and losses resulting from exchange rate changes on transactions denominated in currencies other than the functional currency are included in income in the period in which the change occurs and reported within other (expenses) income in the condensed consolidated statements of operations and comprehensive loss. For the three months ended September 30, 2020 transaction losses were $2.2 million. For the three months ended September 30, 2019 transaction gains were $0.4 million.

    Concentrations of credit risk

    Financial instruments that potentially subject the Company to concentrations of credit risk consist principally of cash. Periodically, the Company maintains deposits in accredited financial institutions in excess of federally insured limits. The Company deposits its cash in financial institutions that it believes have high credit quality and are not exposed to any unusual credit risk beyond the normal credit risk associated with commercial banking relationships, and has not experienced any losses on such accounts. At September 30, 2020 and June 30, 2020, all of the Company's cash was held at two accredited financial institutions.

    Fair value of financial instruments

    At September 30, 2020 and June 30, 2020, the Company's financial instruments included prepaid expenses and other current assets, accounts payable and other current liabilities. The carrying amounts of these assets and liabilities approximates fair value due to their short-term nature.

    Cash and cash equivalents

    The Company considers all highly liquid investments purchased with an original maturity of 90 days or less to be cash equivalents. As of September 30, 2020, the Company had a cash balance of $82.3 million. As of June 30, 2020, the Company had a cash balance of $12.8 million and cash equivalents consisting of $0.5 million held in a money market account.

    Short-term investments

    Short-term investments consist of debt securities with a maturity date greater than three months when acquired. The Company classifies its short-term investments at the time of purchase as available-for-sale securities. Available-for-sale securities are carried at fair value. Unrealized gains or losses on available-for-sale securities are reported in accumulated other comprehensive income (loss), a component of the shareholders' equity, until realized. Short-term investments at June 30, 2020 consisted of U.S. Treasury Bills with a fair value of $1.0 million. Unrealized gains (losses) at June 30, 2020 were de minimis as their maturity date was 91 days from original purchase. The Company had no short-term investments at September 30, 2020.

    Property and equipment

    Property and equipment are recorded at cost. Depreciation is determined using the straight-line method over the estimated useful lives ranging from three to ten years. Expenditures for maintenance and repairs are expensed as incurred while renewals and betterments are capitalized. When property and equipment are sold or otherwise disposed of, the cost and related accumulated depreciation are eliminated from the accounts and any resulting gain or loss is reflected in operations.

    Deferred offering costs

    The Company capitalizes certain legal, professional, accounting and other third-party fees that are directly associated with in-process common equity financings as deferred offering costs until such financings are consummated. After consummation of the equity financing, these costs are recorded as a reduction of additional paid-in capital generated as a result of such offering. Should an in-process equity financing be abandoned, the deferred offering costs will be expensed immediately as a charge to operating expenses in the condensed consolidated statements of operations and comprehensive loss. As of June 30, 2020, the Company had deferred offering costs of $2.4 million related to the IPO on the Nasdaq Global Market which was completed in July 2020. Upon completion of the IPO, the deferred offering costs were reclassified into additional paid-in capital.

    Performance of contract liability to affiliate

    In May 2020, the Company and the Icahn School of Medicine at Mount Sinai entered into an operating agreement ("Kantaro Operating Agreement") to form a joint venture, Kantaro Biosciences LLC ("Kantaro"), for the purpose of developing and commercializing laboratory tests for the detection of antibodies against SARS-CoV-2 originally developed by Mount Sinai. Kantaro has partnered with Bio-Techne Corporation to develop and launch the new test which are designed for use in any authorized clinical testing laboratory without the need for proprietary equipment. During the three months ended September 30, 2020, the Company recognized $0.5 million related to the performance of the contract liability with Kantaro. This represents the allocation of costs for performing services on behalf of Kantaro.

    Equity method investment 

    As the Company can exert significant influence over, but does not control, Kantaro's operations through voting rights or representation on Kantaro's board of directors, the Company accounts for the investment using the equity method of accounting. The Company records its share in Kantaro's earnings and losses in the condensed consolidated statement of operations. The Company assesses its investment for other-than-temporary impairment when events or changes in circumstances indicate that the carrying amount of the investment might not be recoverable and recognize an impairment loss to adjust the investment to its then-current fair value.

    Impairment of long-lived assets

    Long-lived assets are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset to future undiscounted net cash flows expected to be generated. Impairment charges are recognized at the amount by which the carrying amount of an asset exceeds the fair value of the asset. The Company has not recognized any impairment of long-lived assets during the three months ended September 30, 2020 and 2019.

    Software development costs

    The Company follows the provisions of ASC 985, Software, which requires software development costs for software to marketed externally to be expensed as incurred until the establishment of technological feasibility, at which time those costs are capitalized until the software is available for general release and amortized over its estimated useful life. Technological feasibility is established upon the completion of a working model that has been validated.

    Research and development expenses

    Research and development costs consist primarily of costs incurred in connection with the development of KidneyIntelX and other studies for KidneyIntelX to determine clinical value and performance in different chronic kidney disease populations. Research and development costs are expensed as incurred.

    Share-based compensation

    The Company measures equity classified share-based awards granted to employees and nonemployees based on the estimated fair value on the date of grant and recognizes compensation expense of those awards over the requisite service period, which is the vesting period of the respective award. The Company accounts for forfeitures as they occur. For share-based awards with service-based vesting conditions, the Company recognizes compensation expense on a straight-line basis over the service period. The fair value of each stock option grant is estimated on the date of grant using the Black-Scholes option-pricing model, which requires inputs based on certain subjective assumptions, including the expected stock price volatility, the expected term of the option, the risk-free interest rate for a period that approximates the expected term of the option, and the Company's expected dividend yield. The Company was a privately-held organization prior to November 2018 and has been a publicly-traded company for a limited period of time and therefore lacks company-specific historical and implied volatility information for its shares. Therefore, it estimates its expected share price volatility based on the historical volatility of publicly-traded peer companies and expects to continue to do so until such time as it has adequate historical data regarding the volatility of its own traded share price. The expected term of the Company's stock options has been determined utilizing the "simplified" method for awards that qualify as "plain-vanilla" options. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. Expected dividend yield is none based on the fact that the Company has never paid cash dividends on ordinary shares and does not expect to pay any cash dividends in the foreseeable future.

    The Company classifies share-based compensation expense in its condensed consolidated statement of operations and comprehensive loss in the same manner in which the award recipient's payroll costs are classified or in which the award recipient's service payments are classified.

    Comprehensive loss

    Comprehensive loss includes net loss as well as other changes in shareholders' equity that result from transactions and economic events other than those with shareholders. For the periods presented the only other changes in shareholders' equity is from foreign currency translation.

    Net loss per ordinary share

    Basic net loss per ordinary share is computed by dividing net loss by the weighted average number of ordinary shares outstanding during each period. Diluted net loss per ordinary share includes the effect, if any, from the potential exercise or conversion of securities, such as options which would result in the issuance of incremental ordinary shares. Potentially dilutive securities outstanding as of September 30, 2020 and 2019 have been excluded from the computation of diluted weighted average shares outstanding as they would be anti-dilutive. Therefore, the weighted average number of shares used to calculate both basic and diluted net loss per share are the same.

    As of September 30, 2020, and 2019, there were 3,408,858 and 2,833,858 shares issuable upon exercise of outstanding options that were anti-dilutive and excluded from diluted loss per share for the three months ended September 30, 2020 and 2019, respectively.

    Emerging growth company

    The Company is an emerging growth company as defined in the Jumpstart Our Business Startups Act of 2012, as amended (the "JOBS Act"). Under the JOBS Act, companies have extended transition periods available for complying with new or revised accounting standards. The Company has elected to avail itself of this exemption and, therefore, while the Company is an emerging growth company it will not be subject to new or revised accounting standards at the same time that they become applicable to other public emerging growth companies that have not elected to avail themselves of this exemption.

    Recently issued accounting pronouncements

    In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842), in order to increase transparency and comparability among organizations by, among other provisions, recognizing lease assets and lease liabilities on the balance sheet for those leases classified as operating leases under previous U.S. GAAP. For public companies, ASU 2016-02 is effective for fiscal years beginning after December 15, 2018 (including interim periods within those periods) using a modified retrospective approach and early adoption is permitted. In transition, entities may also elect a package of practical expedients that must be applied in its entirety to all leases commencing before the adoption date, unless the lease is modified, and permits entities to not reassess (a) the existence of a lease, (b) the lease classification or (c) the determination of initial direct costs, as of the adoption date, which effectively allows entities to carryforward accounting conclusions under previous U.S. GAAP. In July 2018, the FASB issued ASU 2018-11, Leases (Topic 842): Targeted Improvements, which provides entities an optional transition method to apply the guidance under Topic 842 as of the adoption date, rather than as of the earliest period presented. In June 2020, the FASB issued ASU No 2020-05 that further delayed the effective date of Topic 842 to fiscal years beginning July 1, 2022, and interim periods within those years. The Company is currently evaluating the impact of adopting this guidance to its consolidated financial statements.

    In June 2016, the FASB issued ASU 2016-13, Financial Instruments - Credit Losses: Measurement of Credit Losses on Financial Instruments, which requires measurement and recognition of expected credit losses for financial assets held at the reporting date based on historical experience, current conditions, and reasonable and supportable forecasts. This is different from the current guidance as this will require immediate recognition of estimated credit losses expected to occur over the remaining life of many financial assets. The new guidance will be effective for the Company on July 1, 2023. The Company is currently evaluating the impact of adopting this guidance to its consolidated financial statements.

    In January 2020, FASB issued ASU 2020-01, Investments-Equity Securities (Topic 321), Investments-Equity Method and Joint Ventures (Topic 323), and Derivatives and Hedging (Topic 815), which, generally, provides guidance for investments in entities accounted for under the equity method of accounting. ASU 2020-01 is effective for all entities with fiscal years beginning after December 15, 2021, including interim periods therein. The Company is currently evaluating the impact of adopting this guidance to its consolidated financial statements.

    4. Fair value

    Assets and liabilities recorded at fair value on a recurring basis in the condensed consolidated balance sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or an exit price that would be paid to transfer a liability in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows:

    • Level 1 - Quoted prices (unadjusted in active markets for identical assets or liabilities)



    • Level 2 - Inputs other than quoted prices in active markets that are observable either directly or indirectly



    • Level 3 - Unobservable inputs in which there is little or no market data, which require the Company to develop its own assumptions

    This hierarchy requires the use of observable market data when available and to minimize the use of unobservable inputs when determining fair value. The Company has classified cash equivalents and short-term investments at June 30, 2020, which were comprised of amounts held in a money market account and invested in U.S. Treasury Bills, respectively, and measured at fair value on a recurring basis, as Level 1.

    5. Prepaid expenses and other current assets

    Prepaid expenses and other current assets consisted of (in thousands):

     September 30,

    2020
    June 30,

    2020
    Insurance$3,652 $40
    Other 726  511
     $4,378 $551

    6. Property and equipment

    Property and equipment consists of (in thousands):

     September

    30,
    2020


     June 30,

    2020

     
    Lab equipment$890 $862 
    Software 1,254  744 
    Office equipment 57  31 
    Office furniture 10  10 
    Construction in process 568  113 
    Total 2,779  1,760 
    Less accumulated depreciation (135) (105)
     $2,644 $1,655 

    Depreciation expense was $30,000 and $9,000 for the three months ended September 30, 2020 and 2019, respectively.

    As of September 30, 2020, and June 30, 2020, there was $1.0 and $0.6 million of capitalized software development costs, respectively. Amortization expense related to capitalized software development costs was immaterial for the three months ended September 30, 2020. There was no amortization expense related to capitalized software development costs for the three months ended September 30, 2019.

    7. Accrued expenses and other current liabilities

    Accrued expenses and other current liabilities consisted of (in thousands):

     September 30,

    2020
    June 30,

    2020
    Consulting and professional fees$545 $567
    Research and development   80
    Payroll and related benefits 52  24
    Other 39  12
     $636 $683

    8. Debt

    Paycheck Protection Program

    On April 29, 2020, the Company entered into an original loan agreement with Fortis Private Bank as the lender ("Lender") for a loan in an aggregate principal amount of $255,000 (the "Loan") pursuant to the Paycheck Protection Program (the "PPP") under the Coronavirus Aid, Relief, and Economic Security (CARES) Act and implemented by the U.S. Small Business Administration. The Loan matures in two years and bears interest at a rate of 1% per year, with all payments deferred through the six-month anniversary of the date of the Loan. Principal and interest are payable monthly commencing on October 29, 2020 and may be prepaid by the Company at any time prior to maturity without penalty. The Company may apply for forgiveness of amounts due under the Loan, with the amount of potential loan forgiveness to be calculated in accordance with the requirements of the PPP based on payroll costs, any mortgage interest payments, any covered rent payments and any covered utilities payments during the 8-24 week period after the origination date of the Loan. The Company utilized the proceeds of the Loan for payroll and other qualifying expenses, but there can be no assurances that any portion of the Loan will be forgiven.

    At September 30, 2020, the outstanding principal balance of the Loan is $255,000, of which $120,000 is payable in fiscal year 2021 and $135,000 is payable in fiscal year 2022. The fair value of the Loan as of September 30, 2020 is $245,000, which is determined based on a discounted cash flow model using an estimated market rate of interest of 4.75%, which is classified as a Level 3 fair value measurement.

    9. Commitments and contingencies

    Leases

    In June 2018, the Company entered into an office lease and, in February 2019, the Company entered into a lease for laboratory testing facilities and offices. Each lease is located in New York City and are month-to-month leasing arrangements. Additionally, in February 2019, the Company entered into a lease for an apartment used by executives for traveling requirements. The apartment was located in New York and expired in October 2019. On October 31, 2019, the Company entered into a lease agreement that established a commercial laboratory operation in Salt Lake City, Utah. The lease has a term of five years and is the first long-term lease entered into by the Company. Rent expense for all leases was $0.2 million and $0.1 million for the three months ended September 30, 2020 and 2019, respectively.

    The future minimum payments are as follows (in thousands):

    2021$184
    2022 83
    2023 83
    2024 83
    2025 28
     $461

    Employment agreements

    The Company has entered into employment agreements with certain key executives providing for compensation and severance in certain circumstances, as set forth in the agreements.

    Retirement plans

    The Company maintains a defined contribution 401(k) retirement plan which covers all U.S. employees. Employees are eligible after three months of service. Under the 401(k) plan, participating employees may make contributions in an amount up to the limit set by the Internal Revenue Service on an annual basis. The Company has a safe harbor plan and makes contributions to employee accounts of 5% of compensation (as defined by the plan).

    Legal proceedings

    The Company is not a party to any litigation and does not have contingency reserves established for any litigation liabilities. At each reporting date, the Company evaluates whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies.

    10. License agreements

    Mount Sinai license and sponsored research agreements

    On May 30, 2018, the Company entered into an exclusive license agreement (the ISMMS License Agreement) and, on March 7, 2019, a sponsored research agreement (the ISMMS SRA) with Mount Sinai. Under the terms of the ISMMS License Agreement, ISMMS granted the Company (i) an exclusive, sublicensable license to use certain patent rights covering specific inventions concerning the utilization of biomarkers guided artificial intelligence techniques for detecting kidney functional decline (the ISMMS Technology), (ii) a non-exclusive license under unregistered licensed copyrights and licensed know-how and (iii) an exclusive option to obtain licensed technology conceived after May 30, 2018. The Company is obligated to pay Mount Sinai $1.5 million and $7.5 million in commercial milestone payments upon achieving worldwide net sales of KidneyIntelX of $50.0 million and $300.0 million, respectively. The Company is also obligated to pay Mount Sinai a 4% to 5% royalty on net sales of KidneyIntelX, subject to customary reductions. Royalties are payable on a product-by-product basis from first commercial sale of such product until the later of (1) expiration of the last valid claim of a licensed patent covering such product or (2) on a country-by-country basis, 12 years from first commercial sale of such product in such country. Moreover, the Company is obligated to pay Mount Sinai between 15% and 25% of any consideration received from a sublicensee. Furthermore, we agreed to carry out and fund a clinical utility study for KidneyIntelX at a cost to be determined upon approval of the study protocol by the IRB.

    As part of the ISMMS SRA, the Company has agreed to fund several research projects to further develop the ISMMS Technology. The Company incurred approximately $0.1 million in research and development expenses under the ISMMS SRA for the three months ended September 30, 2019. The Company did not incur any expenses related to the ISMMS SRA for the three months ended September 30, 2020.

    Mount Sinai license agreement for FractalDx

    On December 21, 2018, the Company entered into an exclusive license agreement (the ISMMS FractalDx License Agreement) with ISMMS. Under the terms of the ISMMS FractalDx License Agreement, ISMMS granted the Company (i) an exclusive license, with sub-license rights, to use certain patent rights covering specific inventions concerning the utilization of biomarkers guided artificial intelligence techniques for detecting kidney functional decline (the ISMMS Technology), (ii) a non-exclusive license under unregistered licensed copyrights and licensed know-how and (iii) an exclusive option to obtain licensed technology conceived after May 30, 2018. The Company is obligated to pay Mount Sinai $0.3 million upon receipt of certain regulatory clearance and approval, $0.3 million upon receipt of U.S. CMS reimbursement code or PAMA reimbursement approval. In addition, the Company is obligated to pay Mount Sinai $1.0 million and $4.0 million in commercial milestone payments upon achieving worldwide net sales of FractalDx of $50.0 million and $250.0 million, respectively. The Company is also obligated to pay Mount Sinai a 6% to 8% royalty on net sales of FractalDx, subject to customary reductions. Moreover, the Company is obligated to pay Mount Sinai between 15% and 70% of any consideration received from a sublicensee.

    Royalties are payable on a product-by-product basis from first commercial sale of such product until the later of (1) expiration of the last valid claim of a licensed patent covering such product or (2) on a country-by-country basis, 12 years from first commercial sale of such product in such country. The Company is also subject to an annual license maintenance fee of $25,000 in calendar year 2020 and 2021, $50,000 in calendar year 2022 and 2023, $0.1 million in calendar years 2024 through 2027, and $0.2 million for calendar year 2028 and beyond.

    As discussed in Note 1, in May 2020 the Company transferred the in-licensed FractalDx technology and associated assets to Verici Dx.

    Joslin diabetes center agreement

    In October 2018, the Company purchased a worldwide exclusive license agreement (the "Joslin Agreement") with the Joslin Diabetes Center, Inc. ("Joslin") that was previously entered into with EKF Diagnostics Holding Plc ("EKF"), a related party, in July 2017. The license agreement provides the Company with the right to develop and commercialize licensed products covering a novel methodology of diagnosing and predicting kidney disease using certain biomarkers (the "Joslin Diabetes Technology").

    Under the terms of the Joslin Agreement, the Company is obligated to pay Joslin aggregate commercial milestone payments of $0.3 million and $1.0 million in commercial milestone payments upon achieving worldwide net sales of licensed products and processes of $2.0 million and $10.0 million, respectively. The Company is also obligated to pay Joslin a 5% royalty on net sales of any licensed products or licensed processes, subject to customary reductions. Moreover, the Company is obligated to pay Joslin 25% of any consideration received from a sublicensee.

    The Joslin Agreement initially expires on July 31, 2025 and is subject to an automatic five-year extension unless either party notifies the other party of its intent not to extend the agreement at least 180 days prior to initial expiration. Either party may terminate the Joslin Agreement earlier upon an uncured material breach of the agreement by the other party, the insolvency of the other party, or in the event the other party is unable to perform its obligations under the agreement for a specified period. Additionally, Joslin may terminate the agreement in the event that the Company ceases developing or commercializing licensed products or processes, if the Company fails to maintain certain required insurance policies, and if the Company fails to pay patent expenses related to the licensed patents.

    AstraZeneca statement of work

    In July 2020, we entered into a statement of work (the "AZ SOW") with AstraZeneca Pharmaceuticals LP ("AZ") in advance of entering into a more comprehensive master services agreement. Pursuant to the AZ SOW, the Company will conduct a feasibility study to determine the impact of the use of the KidneyIntelX platform to optimize utilization of various CKD agents and a randomized trial of the KidneyIntelX platform and the Company's care management software versus routine clinical care to improve uptake and adherence of certain CKD agent. Additionally, AZ has agreed to pay the Company up to $1.0 million if certain milestones are achieved. The agreement will terminate upon completion of the activities under the AZ SOW.

    11. Shareholders' equity

    Ordinary shares

    As of September 30, 2020, the Company had 75,438,492 ordinary shares authorized on a fully diluted basis. Each share entitles the holder to one vote on all matters submitted to a vote of the Company's shareholders. Ordinary shareholders are entitled to receive dividends as may be declared by the board of directors. From inception through September 30, 2020, no cash dividends have been declared or paid.

    12. Share-based compensation

    Equity Incentive Plan

    In November 2018, Company established the Renalytix AI plc Share Option Plan (the Plan) and a U.S. Sub-Plan and Non-Employee Sub-Plan. The Plan provides for the Company to grant options, restricted share awards and other share-based awards to employees, directors and consultants of the Company. As of September 30, 2020, there were 3,794,105 shares available for future issuance under the Plan.

    The Plan is administered by the board of directors. The exercise prices, vesting and other restrictions are determined at their discretion, except that all options granted have exercise prices equal to the fair value of the underlying ordinary shares on the date of the grant and the term of stock option may not be greater than ten years from the grant date.

    The options granted as of September 30, 2020 vest equally over twelve quarters following the grant date, with the exception of 80,724 options which vested immediately when granted and 145,000 options which vest 25% on the one year anniversary and equally over twelve quarters following the one year anniversary. If options remain unexercised after the date one day before the tenth anniversary of grant, the options expire. On termination of employment, any options that remain unexercised are either forfeited immediately or after a delayed expiration period, depending on the circumstances of termination. Upon the exercise of awards, new ordinary shares are issued by the Company.

    The Company recorded share-based compensation expense in the following expense categories in the condensed consolidated statements of operations for the three months ended September 30, 2020 and 2019 (in thousands):

     Three Months Ended

    September 30,

      2020 2019
    Research and development$195$134
    General and administrative 296 113
     $491$247

    The fair value of options is estimated using the Black-Scholes option pricing model, which takes into account inputs such as the exercise price, the value of the underlying ordinary shares at the grant date, expected term, expected volatility, risk-free interest rate and dividend yield. The fair value of each grant of options during the three months ended September 30, 2020 and 2019 were determined using the methods and assumptions discussed below.

    • The expected term of employee options is determined using the "simplified" method, as prescribed in SEC's Staff Accounting Bulletin No. 107, whereby the expected life equals the arithmetic average of the vesting term and the original contractual term of the option due to the Company's lack of sufficient historical data.
    • The expected volatility is based on historical volatility of the publicly-traded common stock of a peer group of companies.
    • The risk-free interest rate is based on the interest rate payable on U.S. Treasury securities in effect at the time of grant for a period that is commensurate with the assumed expected term.
    • The expected dividend yield is none because the Company has not historically paid and does not expect for the foreseeable future to pay a dividend on its ordinary shares.

    For the three months ended September 30, 2020 and 2019, the grant date fair value of all option grants was estimated at the time of grant using the Black-Scholes option-pricing model using the following weighted average assumptions:

     Three Months Ended

    September
    30,

     20202019
    Expected term (in years)5.75.7
    Expected volatility67.3%63.6%
    Risk-free rate0.3%1.9%
    Dividend yield—%—%

    The weighted average fair value of the options granted during the three months ended September 30, 2020 and 2019 was $4.31 and $2.05 per share, respectively.

    The following table summarizes the stock option granted to employees and nonemployees for the three months ended September 30, 2020:

     Number of

    shares under

    option plan
    Weighted-

    average

    exercise price

    per option
    Weighted-

    average

    remaining

    contractual

    life (in years)
    Outstanding at June 30, 20203,028,858$1.958.6
    Granted380,000$7.46 
    Outstanding at September 30, 20203,408,858$2.568.5
    Exercisable at September 30, 20201,649,525$1.948.3
    Vested and expected to vest at September 30, 20203,408,858$2.568.5

    As of September 30, 2020, there was $3.3 million in unrecognized compensation cost related to unvested options that will be recognized as expense over a weighted average period of 1.56 years. The aggregate intrinsic value of options outstanding and options exercisable at September 30, 2020 was $7.0 million and $4.0 million, respectively.

    Employee Stock Purchase Plan

    The Company's 2020 Employee Share Purchase Plan (the ESPP) became effective on August 17, 2020. The ESPP authorizes the issuance of up to 850,000 shares of the Company's common stock. The number of shares of the Company's common stock that may be issued pursuant to rights granted under the ESPP shall automatically increase on January 1st of each year, commencing on January 1, 2021 and continuing for ten years, in an amount equal to the lesser of one percent of the total number of shares of the Company's common stock outstanding on December 31st of the preceding calendar year, and 2,000,000 ordinary shares, subject to the discretion of the board of directors or renumeration committee to determine a lesser number of shares shall be added for such year.

    Under the ESPP, eligible employees can purchase the Company's common stock through accumulated payroll deductions at such times as are established by the board of directors or renumeration committee. Eligible employees may purchase the Company's common stock at 85% of the lower of the fair market value of the Company's common stock on the first day of the offering period or on the purchase date. Eligible employees may contribute up to 15% of their eligible compensation. Under the ESPP, a participant may not purchase more than $25,000 worth of the Company's common stock for each calendar year in which such rights is outstanding.

    Effective August 28, 2020, employees who elected to participate in the ESPP commenced payroll withholdings that accumulate through February 27, 2021. In accordance with the guidance in ASC 718-50 – Compensation – Stock Compensation, the ability to purchase shares of the Company's common stock at 85% of the lower of the price on the first day of the offering period or the last day of the offering period (i.e. the purchase date) represents an option and, therefore, the ESPP is a compensatory plan under this guidance. Accordingly, share-based compensation expense is determined based on the option's grant-date fair value as estimated by applying the Black Scholes option-pricing model and is recognized over the withholding period. The Company recognized share-based compensation expense of $10,000 during the three months ended September 30, 2020 related to the ESPP. 

    13. Related-party transactions

    EKF Diagnostic Holdings

    During the three months ended September 30, 2020 and 2019, the Company paid fees to employees of EKF who provided services to Renalytix.

    Icahn School of Medicine at Mount Sinai

    In May 2018, the Company secured its cornerstone license agreement with ISMMS for research and clinical study work and intended commercialization by the Company (see Note 10). As part of the collaboration, ISMMS became a shareholder in the Company and has subsequently made equity investments both in the Company's IPO on AIM in November 2018, the subsequent sale of ordinary shares in July 2019 and the Company's IPO on Nasdaq in July 2020.

    Kantaro Biosciences LLC

    In connection with the formation of Kantaro, the Company entered into a five-year Advisory Services Agreement ("Advisory Agreement") pursuant to which the Company has agreed to provide certain advisory services to Kantaro. Pursuant to the Kantaro Operating Agreement, Kantaro issued 750 Class A Units to Mount Sinai in exchange for Mount Sinai granting licenses to Kantaro under certain intellectual property rights of Mount Sinai and 250 Class A Units to the Company as the sole consideration for the services to be rendered by the Company under the Advisory Agreement. A portion of the Company's units are subject to forfeiture if, prior to December 31, 2020, Kantaro terminates the Advisory Agreement as a result of an uncured material breach of the Advisory Agreement or in the event the Company is acquired by a hospital or health system that serves all or any portion of the service areas served by Mount Sinai. The Company determined the fair value of the services to be provided under the Advisory Agreement was $2.0 million and the fair value of the Class A units received from Kantaro was $2.0 million. Fair value was determined using discounted cash flows which is a Level 3 measurement in the fair value hierarchy. The method requires several judgments and assumptions which include discount rates and future cash flows, among others. As of September 30, 2020, the total liability associated with the services was $1.4 million, of which $1.2 million is classified as a current liability and $0.2 million is classified as a non-current liability. For the three months ended September 30, 2020, the Company recognized $0.5 million in the statement of operations related to services performed under the Advisory Agreement. For the three months ended September 30, 2020, $0.2 million and $0.1 million of costs incurred related to the performance of the Advisory Agreement services were included within research and development and general and administrative expense, respectively.

    In addition to the equity granted at formation, the Company and Mount Sinai each committed to making a loan to Kantaro. Mount Sinai committed to lend an initial amount of $0.3 million and an additional $0.5 million thereafter. The Company committed to lend an initial amount of $83,333 and an additional $166,667 thereafter. Each loan bears interest at a per year rate equal to 0.25%, compounded monthly, until repaid, and is repayable from the first amounts that would otherwise constitute cash available for distribution to the members of Kantaro (provided that each loan repayment will be made, 75% to Mount Sinai and 25% to the Company based on each investor's proportionate ownership). The Company loaned Kantaro $83,333 and had a note receivable for this amount at September 30, 2020. In addition, the Company recognized losses of $0.1 million on their investment in Kantaro during the three months ended September 30, 2020.

    14. Subsequent events

    The Company has evaluated subsequent events from the balance sheet date through the date at which the condensed consolidated financial statements were available to be issued, and determined there are no other items requiring disclosure beyond those disclosed below.

    Verici Dx

    On November 3, 2020, Verici Dx completed an initial public offering on AIM and raised gross proceeds of £14.5 million ("Verici IPO") triggering a reconsideration event for ongoing consolidation of Verici Dx. The IPO of Verici Dx resulted in the Company no longer having a controlling financial interest and no longer having a majority equity interest. Verici Dx previously issued the Company $2.5 million in convertible loan notes which reflects the consideration for the FractalDx assets and the funding the Company provided Verici Dx through October 28, 2020. Prior to the Verici IPO, on October 28, 2020, the Company gave notice to convert the existing $2.5 million convertible loan notes into 9,831,681 ordinary shares of Verici Dx.



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  5. Positive high-level results from an interim analysis of clinical trials of AZD1222 in the UK and Brazil showed the vaccine was highly effective in preventing COVID-19, the primary endpoint, and no hospitalisations or severe cases of the disease were reported in participants receiving the vaccine. There were a total of 131 COVID-19 cases in the interim analysis.

    One dosing regimen (n=2,741) showed vaccine efficacy of 90% when AZD1222 was given as a half dose, followed by a full dose at least one month apart, and another dosing regimen (n=8,895) showed 62% efficacy when given as two full doses at least one month apart. The combined analysis from both dosing regimens (n=11,636) resulted in an average efficacy of 70%. All results were statistically…

    Positive high-level results from an interim analysis of clinical trials of AZD1222 in the UK and Brazil showed the vaccine was highly effective in preventing COVID-19, the primary endpoint, and no hospitalisations or severe cases of the disease were reported in participants receiving the vaccine. There were a total of 131 COVID-19 cases in the interim analysis.

    One dosing regimen (n=2,741) showed vaccine efficacy of 90% when AZD1222 was given as a half dose, followed by a full dose at least one month apart, and another dosing regimen (n=8,895) showed 62% efficacy when given as two full doses at least one month apart. The combined analysis from both dosing regimens (n=11,636) resulted in an average efficacy of 70%. All results were statistically significant. More data will continue to accumulate and additional analysis will be conducted, refining the efficacy reading and establishing the duration of protection.

    An independent Data Safety Monitoring Board determined that the analysis met its primary endpoint showing protection from COVID-19 occurring 14 days or more after receiving two doses of the vaccine. No serious safety events related to the vaccine have been confirmed. AZD1222 was well tolerated across both dosing regimens.

    AstraZeneca will now immediately prepare regulatory submission of the data to authorities around the world that have a framework in place for conditional or early approval. The Company will seek an Emergency Use Listing from the World Health Organization for an accelerated pathway to vaccine availability in low-income countries. In parallel, the full analysis of the interim results is being submitted for publication in a peer-reviewed journal.

    Professor Andrew Pollard, Chief Investigator of the Oxford Vaccine Trial at Oxford, said: “These  findings  show  that  we  have  an  effective  vaccine  that  will  save  many  lives. Excitingly, we've found that one of our dosing regimens may be around 90% effective and if this dosing regime is used, more people could be vaccinated with planned vaccine supply. Today's announcement is only possible thanks  to  the  many  volunteers  in  our  trial,  and  the  hard  working  and  talented  team  of  researchers based around the world.”

    Pascal Soriot, Chief Executive Officer, said: “Today marks an important milestone in our fight against the pandemic. This vaccine's efficacy and safety confirm that it will be highly effective against COVID-19 and will have an immediate impact on this public health emergency. Furthermore, the vaccine's simple supply chain and our no-profit pledge and commitment to broad, equitable and timely access means it will be affordable and globally available, supplying hundreds of millions of doses on approval.”

    The pooled analysis included data from the COV002 Phase II/III trial in the UK and COV003 Phase III trial in Brazil. Over 23,000 participants are being assessed following two doses of either a half-dose/full-dose regimen or a regimen of two full doses of AZD1222 or a comparator, meningococcal conjugate vaccine called MenACWY or saline. The global trials are evaluating participants aged 18 years or over from diverse racial and geographic groups who are healthy or have stable underlying medical conditions.

    Clinical trials are also being conducted in the US, Japan, Russia, South Africa, Kenya and Latin America with planned trials in other European and Asian countries. In total, the Company expects to enrol up to 60,000 participants globally.

    The Company is making rapid progress in manufacturing with a capacity of up to 3 billion doses of the vaccine in 2021 on a rolling basis, pending regulatory approval. The vaccine can be stored, transported and handled at normal refrigerated conditions (2-8 degrees Celsius/ 36-46 degrees Fahrenheit) for at least six months and administered within existing healthcare settings.

    AstraZeneca continues to engage with governments, multilateral organisations and collaborators around the world to ensure broad and equitable access to the vaccine at no profit for the duration of the pandemic.

    COV002

    COV002 is a single-blinded, multi-centre, randomised, controlled Phase II/III trial assessing the safety, efficacy and immunogenicity of AZD1222 in 12,390 participants in the UK. Trial participants to date are aged 18 years or over, who are healthy or have medically stable chronic diseases and are at increased risk for being exposed to the SARS-CoV-2 virus. Participants receive one or two intramuscular doses of a half dose (~2.5 x1010 viral particles) or full dose (~5x1010 viral particles) of AZD1222 or comparator, meningococcal vaccine MenACWY. Participants have blood samples drawn and clinical assessments for safety as well as immunogenicity at multiple timepoints up to one year post-vaccination. Suspected cases presenting with compatible symptoms were tested for virological confirmation by COVID-19 PCR. In addition, weekly swabbing are done for detection of infection and assessment of vaccine efficacy against infection.

    COV003

    COV003 is a single-blinded, multi-centre, randomised, controlled Phase III trial assessing the safety, efficacy, and immunogenicity of AZD1222 in 10,300 participants in Brazil. Trial participants to date are aged 18 years or over, who are healthy or have medically stable chronic diseases and are at increased risk for being exposed to the SARS-CoV-2 virus. Participants are randomised to receive two intramuscular doses of a full dose (~5x1010 viral particles) of AZD1222 or comparator, meningococcal vaccine MenACWY as first dose and a saline placebo as second dose. Participants have blood samples drawn and clinical assessments for safety as well as immunogenicity at multiple timepoints up to one year post-vaccination. Suspected cases presenting with compatible symptoms were tested for virological confirmation by COVID-19 PCR.

    AZD1222
    AZD1222 was co-invented by the University of Oxford and its spin-out company, Vaccitech. It uses a replication-deficient chimpanzee viral vector based on a weakened version of a common cold virus (adenovirus) that causes infections in chimpanzees and contains the genetic material of the SARS-CoV-2 virus spike protein. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body.

    AstraZeneca

    AstraZeneca (NASDAQ:AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com 

    — WebWireID266934 —


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