On November 5, 2020 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that new data for its approved and investigational medicines will be presented at the all-virtual 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition from 5 – 8 December 2020 (Press release, Hoffmann-La Roche, NOV 5, 2020, View Source [SID1234570026]). Eleven Roche medicines will be featured in more than 80 abstracts, including 22 oral presentations. With studies spanning 16 blood disorders, including non-Hodgkin lymphoma (NHL), leukaemia, multiple myeloma (MM) and haemophilia A, these data highlight the strength and breadth of Roche’s haematology portfolio and pipeline, and commitment to developing innovative treatment solutions for patients in need.

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"We have one of the largest clinical development programmes in malignant and non-malignant haematology and we continuously seek to improve patient outcomes by exploring new therapeutic mechanisms, combinations and clinical trial endpoints," said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. "Our data reflect our ongoing commitment to following the science and improving the lives of patients with some of the most difficult-to-treat blood disorders."

Building on its 20-year legacy in antibody engineering, Roche is exploring novel mechanisms of action for immunotherapies including T-cell engaging bispecific antibodies. Data on three investigational bispecifics will be presented, including:

Progress from Roche’s CD20xCD3 bispecific antibody development programmes, including updated results for mosunetuzumab in relapsed or refractory (R/R) follicular lymphoma and early data in first-line diffuse large B-cell lymphoma (DLBCL). Additionally, data demonstrating high response rates with step-up dosing of glofitamab in people with R/R NHL will be presented.
First clinical safety, efficacy and biomarker data from cevostamab (BFCR4350A), a first-of-its-kind FcRH5xCD3 bispecific antibody targeting FcRH5 on myeloma cells and CD3 on T-cells, will be presented, with initial results from the ongoing phase I GO39775 dose-escalation study in people with heavily pre-treated R/R MM.
Roche will also be sharing longer-term data, including results on novel clinical trial endpoints, that support the known efficacy and safety of its established medicines, including:

Three year follow-up data from the pivotal phase III HAVEN study programme (HAVEN 1-4 studies), reinforcing the efficacy and safety profile of Hemlibra (emicizumab) in people with haemophilia A with and without factor VIII inhibitors.
Results from the first interim analysis of the European Haemophilia Safety Surveillance database, examining real-world data to monitor the ongoing safety of Hemlibra in people with haemophilia A with and without factor VIII inhibitors.
Results on fixed-duration, chemotherapy-free combinations in chronic lymphocytic leukaemia (CLL), including five-year analysis of the phase III MURANO study, investigating Venclexta/Venclyxto (venetoclax) plus MabThera/Rituxan (rituximab) in R/R CLL, with updates on minimal residual disease and long-term outcomes analysis. Venclexta/Venclyxto is being developed by AbbVie and Roche.
Updated results from the phase Ib/II randomised GO29365 study of fixed-duration Polivy (polatuzumab vedotin), plus bendamustine and MabThera/Rituxan, in people with R/R DLBCL, including preliminary results from a single-arm extension cohort of 106 additional patients.
Key abstracts featuring Roche medicines that will be presented at ASH (Free ASH Whitepaper) can be found in the table below.

Follow Roche on Twitter via @Roche, and keep up to date with ASH (Free ASH Whitepaper) Annual Meeting news and updates by using the hashtag #ASH20.

Medicine Abstract title Abstract number/presentation details
Mosunetuzumab
(investigational) Mosunetuzumab Shows Promising Efficacy in Patients with Multiply Relapsed Follicular Lymphoma: Updated Clinical Experience from a Phase I Dose-Escalation Trial #702 oral presentation
Session: 623
Monday 7 December 2020
13:30-15.00 PT (presentation time 14.00 PT)
Single-agent Mosunetuzumab is a Promising Safe and Efficacious Chemotherapy-Free Regimen for Elderly/Unfit Patients with Previously Untreated Diffuse Large B‑Cell Lymphoma #401 oral presentation
Session: 626
Sunday 6 December 2020
12:00-13:30 PT (presentation time 12:15 PT)
Mosunetuzumab, a Novel CD20/CD3 Bispecific Antibody, in Combination With CHOP Confers High Response Rates in Patients with Diffuse Large B-Cell Lymphoma #1184 poster presentation
Session: 626
Saturday 5 December 2020
07:00-15:30 PT
Glofitamab
(investigational) Glofitamab Step-Up Dosing Induces High Response Rates in Patients with Hard-to-Treat Refractory or Relapsed Non-Hodgkin Lymphoma #403 oral presentation
Session: 626
Sunday 6 December 2020
12:00-13:30 PT (presentation time 12.45 PT)
Cevostamab (BFCR4350A; a FcRH5xCD3 bispecific antibody)
(investigational) Initial Clinical Activity and Safety of BFCR4350A, a FcRH5/CD3 T-Cell-Engaging Bispecific Antibody, in Relapsed/Refractory Multiple Myeloma #292 oral presentation
Session: 653
Saturday 5 December 2020
14:00-15.30 PT (presentation time 14.30 PT)
Early Pharmacodynamic Changes in T-Cell Activation, Proliferation, and Cytokine Production Confirm the Mode of Action of BFCR4350A, a FcRH5/CD3 T-Cell-Engaging Bispecific Antibody, in Patients with Relapsed/Refractory Multiple Myeloma #3213 poster presentation
Session: 653
Monday 7 December 2020
07:00-15.30 PT
Hemlibra
(approved use) Safety and Efficacy of Emicizumab in Persons with Hemophilia A With or Without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4) #1800 poster presentation
Session: 322
Sunday 6 December 2020
07:00-15:30 PT
Real-World Safety of Emicizumab: The First Interim Analysis of the European Haemophilia Safety Surveillance (EUHASS) Database #2685 poster presentation
Session: 322
Monday 7 December 2020
07:00-15:30 PT
Venclexta/Venclyxto
(approved use) Five-Year Analysis of MURANO Study Demonstrates Enduring Undetectable Minimal Residual Disease (uMRD) in a Subset of Relapsed/Refractory Chronic Lymphocytic Leukemia (R/R CLL) Patients (Pts) Following Fixed-Duration Venetoclax-Rituximab (VenR) Therapy (Tx) #125 oral presentation
Session: 642
Saturday 5 December 2020
09:30-11:00 PT (presentation time 10:00 PT)
Clonal Dynamics after Venetoclax-Obinutuzumab Therapy: Novel Insights from the Randomized, Phase 3 CLL14 trial #127 oral presentation
Session: 642
Saturday 5 December 2020
09:30-11:00 PT (presentation time 10:30 PT)
Results of Venetoclax and Azacitidine Combination in Chemotherapy Ineligible Untreated Patients with Acute Myeloid Leukemia with FLT3 mutations #1904 poster presentation
Session: 613
Sunday 6 December 2020
07:00-15:30 PT
Results of Venetoclax and Azacitidine Combination in Chemotherapy Ineligible Untreated Patients with Acute Myeloid Leukemia with IDH 1/2 Mutations #461 oral presentation
Session: 613
Sunday 6 December 2020
14:00-15:30 PT (presentation time 14:45 PT)
Characteristics and Outcome of Patients with Chronic Lymphocytic Leukaemia and Partial Response to Venetoclax-Obinutuzumab #1310 poster presentation
Session: 642
Saturday 5 December 2020
07:00-15:30 PT
Polivy
(approved use) Polatuzumab Vedotin Plus Bendamustine and Rituximab in Relapsed/Refractory Diffuse Large B-Cell Lymphoma: Updated Results of a Phase Ib/II Randomized Study and Preliminary Results of a Single-Arm Extension #3020 poster presentation
Session: 626
Monday 7 December 2020
07:00-15:30 PT
Risk Profiling of Relapsed/Refractory Diffuse Large B-Cell Lymphoma Patients By Measuring Circulating Tumor DNA #532 oral presentation
Session: 627
Monday 7 December 2020
07:00-08:30 PT (presentation time 07:30 PT)

About Roche in haematology
Roche has been developing medicines for people with malignant and non-malignant blood diseases for over 20 years; our experience and knowledge in this therapeutic area runs deep. Today, we are investing more than ever in our effort to bring innovative treatment options to patients across a wide range of haematologic diseases. Our approved medicines include MabThera/Rituxan (rituximab), Gazyva/Gazyvaro (obinutuzumab), Polivy (polatuzumab vedotin), Venclexta/Venclyxto (venetoclax) in collaboration with AbbVie, and Hemlibra (emicizumab). Our pipeline of investigational haematology medicines includes T-cell engaging bispecific antibodies, glofitamab and mosunetuzumab, targeting both CD20 and CD3, and cevostamab, targeting FcRH5 on myeloma cells and CD3 on T-cells; Tecentriq (atezolizumab), a monoclonal antibody designed to bind with PD-L1; and crovalimab, an anti-C5 antibody engineered to optimise complement inhibition. Our scientific expertise, combined with the breadth of our portfolio and pipeline, also provides a unique opportunity to develop combination regimens that aim to improve the lives of patients even further.

On November 5, 2020 Genmab A/S (Nasdaq: GMAB) reported that it will initiate a Phase 3 study of epcoritamab in diffuse large B-cell lymphoma (DLBCL) (Press release, Genmab, NOV 5, 2020, View Source [SID1234570025]). The study will evaluate the efficacy and safety of subcutaneous epcoritamab, a fully-human IgG1-bispecific antibody designed to recognize and bind to both CD3 and CD20, versus investigators’ choice of chemotherapy regimen (either bendamustine and rituximab or gemcitabine, oxaliplatin, and rituximab) in patients with relapsed or refractory DLBCL. Epcoritamab is being co-developed by Genmab and AbbVie.

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DLBCL is aggressive and the most common form of non-Hodgkin’s lymphoma worldwide, with 36% of DLBCL patients in the U.S. expected to die from their disease within five years of diagnosis.1 Prevalence rates are expected to increase, driven by growth in aging populations. 2

"In collaboration with AbbVie, we have planned a broad, expansive, accelerated epcoritamab clinical development plan to maximize the potential of this promising bispecific antibody, with the ultimate goal of bringing new differentiated treatment options as soon as possible to patients. We look forward to the data from this first Phase 3 trial, especially for relapsed or refractory DLBCL patients as it remains an area of high unmet medical need," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

About the Study
The Phase 3, open-label, randomized study (GCT3013-05) will include approximately 480 patients with relapsed or refractory DLBCL who failed or are ineligible for autologous stem cell transplant (ASCT). Patients will be randomized to receive either subcutaneous epcoritamab or one of two chemotherapy regimens as per investigator’s choice, either rituximab, gemcitabine and oxaliplatin (R-GemOx) or bendamustine and rituximab (BR). The primary endpoint of the study is overall survival.

About Diffuse Large B-cell Lymphoma
DLBCL is the most common type of non-Hodgkin lymphoma (NHL) in the United States and worldwide, with an average age at diagnosis of mid-60s.2,3 It is an aggressive form of NHL with relative 10-year survival rates of approximately 46% and relative 5-year survival rates of approximately 64%.1,3,4 Prevalence is anticipated to increase, driven by growth in aging populations.1 DLBCL affects B-lymphocytes and can develop in the lymph nodes or in other organs, and may be either localized or generalized.3 The prognosis for relapsed or refractory DLBCL patients is poor, especially for those with high-risk factors, and for most patients with refractory DLBCL there are no curative treatment options.5

About Epcoritamab
Epcoritamab is an investigational IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to tumors to elicit an immune response towards malignant cells. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T cell mediated killing of lymphoma B cells.6 CD20 is a clinically validated therapeutic target, and is expressed on many B-cell malignancies, including diffuse large B-cell lymphoma, follicular lymphoma, mantle cell lymphoma and chronic lymphocytic leukemia.7,8 Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ broad oncology collaboration.

On November 5, 2020 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the following upcoming investor conferences (Press release, Ultragenyx Pharmaceutical, NOV 5, 2020, View Source [SID1234570024]):

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Credit Suisse 29TH Annual Virtual Healthcare Conference, on Tuesday, November 10, 2020 at 2:00 PM ET.

Barclays Gene Editing & Gene Therapy Summit on Monday, November 16, 2020 at 3:00 PM ET.
The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On November 5, 2020 Arbutus Biopharma Corporation (Nasdaq: ABUS), a clinical-stage biopharmaceutical company primarily focused on developing a cure for people with chronic hepatitis B virus (HBV) infection, as well as therapies to treat coronaviruses (including COVID-19), reported its third quarter 2020 financial results and provides a corporate update (Press release, Arbutus Biopharma, NOV 5, 2020, View Source [SID1234570023]).

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William Collier, President and Chief Executive Officer of Arbutus, stated, "Arbutus is focused on discovering and developing a functional cure with a finite treatment duration for chronic HBV by developing a combination of agents, with different mechanisms of action, that target distinct parts of the virus lifecycle. To this end, we continue to make steady progress in our ongoing Phase 1a/1b clinical trial of our lead clinical candidate, AB-729, a subcutaneously delivered RNAi agent. AB-729 is currently being dosed in chronic HBV subjects in four multi-dose cohorts using both the 60 mg dose every 4- and 8-weeks and the 90 mg dose every 8- and 12-weeks."

"Based upon the clinical data generated thus far, AB-729 has demonstrated meaningful reductions in HBsAg with a favorable safety and tolerability profile. We look forward to presenting additional results from the ongoing Phase 1a/1b clinical trial as part of an oral presentation at the upcoming AASLD conference in November."

Pipeline Update

AB-729

Arbutus is currently conducting a single- and multi-dose Phase 1a/1b clinical trial to determine the safety, tolerability, pharmacokinetics, and pharmacodynamics of AB-729 in healthy subjects and in subjects with chronic HBV infection.

Arbutus is currently dosing two 60 mg multi-dose cohorts of subjects with chronic HBV infection with dosing intervals of every four and eight weeks, respectively. Results from the 60 mg multi-dose cohort with a dosing interval of every four weeks and additional follow-up data on the 60 mg and 90 mg single-dose cohorts are expected to be disclosed as part of an oral presentation at the upcoming AASLD conference in November.

Separately, results from the 60 mg multi-dose cohort with a dosing interval of every eight weeks and a 90 mg single-dose cohort in HBV DNA positive subjects are expected in the fourth quarter of 2020.

In September 2020, Arbutus reported additional data from its ongoing Phase 1a/1b clinical trial for AB-729. The clinical data generated thus far demonstrate the robust activity of AB-729 and, at week 12, the 60 mg and 90 mg single-doses achieved meaningful reductions in HBsAg while remaining generally safe and well tolerated.
Arbutus is also currently dosing two 90 mg multi-dose cohorts of subjects with dosing intervals of every eight and twelve weeks, respectively.
AB-836: Oral Capsid Inhibitor

In January 2020, Arbutus selected AB-836 as its next-generation oral capsid inhibitor. AB-836 is from a novel chemical series differentiated from competitor compounds with the potential for increased efficacy and an enhanced resistance profile. Arbutus continues to expect completion of CTA/IND-enabling studies by the end of 2020.
Early HBV R&D Programs

Arbutus’ drug discovery efforts are focused on follow-on compounds for its current HBV pipeline, including the development of oral RNA-destabilizers that have shown compelling antiviral effects in multiple HBV preclinical models. Arbutus is now focused on advancing through lead optimization next-generation oral RNA-destabilizers with chemical scaffolds distinct from Arbutus’ prior generation HBV RNA destabilizer candidate. Arbutus also has several oral anti-PD-L1 inhibitors in lead optimization that are potentially capable of reawakening the immune response to HBV in infected patients.
Clinical Collaboration with Assembly Biosciences, Inc.

In August 2020, the Company entered into a clinical collaboration agreement with Assembly Biosciences, Inc. (Assembly) to evaluate Arbutus’ AB-729 clinical candidate in combination with Assembly’s lead hepatitis B virus (HBV) core/capsid inhibitor candidate vebicorvir (VBR) and standard-of-care nucleos(t)ide reverse transcriptase inhibitor (NrtI) therapy for the treatment of patients with chronic HBV infection. This collaboration will include a randomized, multi-center, open-label Phase 2 clinical trial that will explore the safety, pharmacokinetics, and antiviral activity of the triple combination of AB-729, VBR, and an NrtI compared to the double combinations of VBR with an NrtI and AB-729 with an NrtI. This trial is expected to initiate in the first half of 2021 and enroll approximately 60 virologically-suppressed patients with chronic HBV infection.
Dr. Gaston Picchio, Chief Development Officer of Arbutus, stated, "This clinical collaboration in which both companies share expertise and costs has the potential to provide proof of concept data regarding the safety and efficacy of combining two promising drug candidates and to expedite efforts to advance a much needed HBV treatment regimen."

Research Efforts to Combat COVID-19 and Future Coronavirus Outbreaks

Based on its extensive antiviral drug discovery experience, Arbutus has established an internal research program to identify new small molecule antiviral medicines to treat COVID-19 and future coronavirus outbreaks. This effort, led by Dr. Michael Sofia, Arbutus’ Chief Scientific Officer, is focused on the discovery and development of new molecular entities that address specific viral targets including the nsp12 viral polymerase and the nsp5 viral protease. These targets are essential viral proteins which Arbutus has experience in targeting. Arbutus has also joined forces with the COVID R&D consortium to further support and expedite efforts to address the COVID-19 pandemic.
Genevant Sciences Ltd. Update

On July 31, 2020, Genevant Sciences Ltd. (Genevant) was recapitalized through an equity investment and conversion of previously issued convertible debt securities held by Roivant Sciences Ltd. (Roivant), Arbutus’ largest shareholder. Arbutus participated in the recapitalization of Genevant with an equity investment of $2.5 million. Following the recapitalization, Arbutus owns approximately 16% of the common equity of Genevant. Arbutus’ entitlement to receive future royalties or sublicensing revenue from Genevant remains unchanged.

As previously disclosed, in April 2018 Arbutus entered into an agreement with Roivant to launch Genevant, a company focused on the discovery, development, and commercialization of a broad range of RNA-based therapeutics enabled by Arbutus’ lipid nanoparticle ("LNP") and ligand conjugate delivery technologies. Arbutus licensed exclusive rights to its LNP and ligand conjugate delivery platforms to Genevant for RNA-based applications outside of HBV, except to the extent certain rights had already been licensed to other third parties
COVID-19 Impact

In December 2019 an outbreak of a novel strain of coronavirus (COVID-19) was identified in Wuhan, China. This virus continues to spread globally, has been declared a pandemic by the World Health Organization and has spread to nearly every country in the world. The impact of this pandemic has been, and will likely continue to be, extensive in many aspects of society. The pandemic has resulted in and will also likely continue to result in significant disruptions to businesses. A number of countries and other jurisdictions around the world have implemented extreme measures to try and slow the spread of the virus. These measures include the closing of businesses and requiring people to stay in their homes, the latter of which raises uncertainty regarding the ability to travel to hospitals in order to participate in clinical trials. Additional measures that have had, and will likely continue to have, a major impact on clinical development, at least in the near-term, include shortages and delays in the supply chain, and prohibitions in certain countries on enrolling subjects in new clinical trials. While we have been able to progress with our clinical and pre-clinical activities to date, it is not possible to predict if the COVID-19 pandemic will negatively impact our plans and timelines in the future.

Financial Results

Cash, Cash Equivalents and Investments

Arbutus had cash, cash equivalents and investments totaling $118.3 million as of September 30, 2020, as compared to $90.8 million as of December 31, 2019. During the nine months ended September 30, 2020, Arbutus used $36.4 million in operating activities and made a $2.5 million equity investment in Genevant. These cash outflows were offset by $66.1 million of net proceeds from the issuance of common shares under Arbutus’s ATM program. The Company believes its ending third quarter cash, cash equivalents and investments of $118.3 million are sufficient to fund the Company’s operations into mid-2022.

Net Loss

Net loss attributable to common shares for the three months ended September 30, 2020 was $21.8 million ($0.27 basic and diluted loss per common share) as compared to $85.3 million ($1.50 basic and diluted loss per common share) for the three months ended September 30, 2019. Net loss attributable to common shares for the three months ended September 30, 2019 included: i) non-cash impairment charges of $43.8 million for an in-process research and development ("IPR&D") intangible asset and $22.5 million for goodwill to reduce their carrying values to zero, as well as a corresponding income tax benefit of $12.7 million related to the decrease in the deferred tax liability associated with the IPR&D intangible assets; and ii) a $6.5 million expense related to an arbitration award from the Company’s arbitration with the University of British Columbia.

Net loss attributable to common shares for the three months ended September 30, 2020 and 2019 included non-cash expense for the accrual of coupon on the Company’s convertible preferred shares of $3.0 million and $2.8 million, respectively, and non-cash expense for a proportionate share of Genevant’s net losses of $2.5 million in the third quarter of 2020 and $3.5 million in the third quarter of 2019.

Operating Expenses

Research and development expenses were $12.1 million for the three months ended September 30, 2020 compared to $17.7 million in 2019. The decrease in research and development expenses for the three months ended September 30, 2020 versus the same period in 2019 was due primarily to lower clinical expenses in 2020. General and administrative expenses were $4.1 million for the three months ended September 30, 2020 compared to $3.2 million for the same period in 2019. This increase was due primarily to increased compensation-related expenses and an increase in insurance premiums.

Outstanding Shares

The Company had approximately 84.6 million common shares issued and outstanding as of September 30, 2020. In addition, the Company had approximately 10.9 million stock options outstanding and 1.164 million convertible preferred shares outstanding, which (including the 8.75% annual interest in the form of additional preferred shares) will be mandatorily convertible into approximately 23.0 million common shares on October 18, 2021.

Conference Call and Webcast Today

Arbutus will hold a conference call and webcast today, Thursday, November 5, 2020 at 8:45 AM Eastern Time to provide a corporate update. You can access a live webcast of the call through the Investors section of Arbutus’ website at www.arbutusbio.com or directly at Live Webcast. Alternatively, you can dial (866) 393-1607 or (914) 495-8556 and reference conference ID 7161816.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling (855) 859-2056 or (404) 537-3406, and reference conference ID 7161816.

About AB-729

AB-729 is an RNA interference (RNAi) therapeutic targeted to hepatocytes using Arbutus’ novel covalently conjugated N-acetylgalactosamine (GalNAc) delivery technology that enables subcutaneous delivery. AB-729 inhibits viral replication and reduces all HBV antigens, including hepatitis B surface antigen in preclinical models. Reducing hepatitis B surface antigen is thought to be a key prerequisite to enable reawakening of a patient’s immune system to respond to the virus. Based upon clinical data generated thus far in an ongoing single- and multi-dose Phase 1a/1b clinical trial, AB-729 has demonstrated positive safety and tolerability data and meaningful reductions in hepatitis B surface antigen.

About AB-836

AB-836 is an oral HBV capsid inhibitor. HBV core protein assembles into a capsid structure, which is required for viral replication. The current standard-of-care therapy for HBV, primarily nucleos(t)ide analogues that work by inhibiting the viral polymerase, significantly reduce virus replication, but not completely. Capsid inhibitors inhibit replication by preventing the assembly of functional viral capsids. They also have been shown to inhibit the uncoating step of the viral life cycle thus reducing the formation of new covalently closed circular DNA (cccDNA), the genetic reservoir which the virus uses to replicate itself.

About HBV

Chronic hepatitis B virus (HBV) infection is a debilitating disease of the liver that afflicts over 250 million people worldwide with up to 90 million people in China, as estimated by the World Health Organization. HBV is a global epidemic that affects more people than hepatitis C virus (HCV) and HIV infection combined—with a higher morbidity and mortality rate. HBV is a leading cause of chronic liver disease and need for liver transplantation, and up to one million people worldwide die every year from HBV-related causes. The current standard of care for patients with chronic HBV infection is life-long suppressive treatment with medications that reduce, but do not eliminate, the virus, resulting in very low cure rates. There is a significant unmet need for new therapies to treat HBV.

On November 5, 2020 Orgenesis Inc. (NASDAQ: ORGS) ("Orgenesis" or the "Company"), a global biotech company working to unlock the full potential of cell and gene therapies, reported a business update for the third quarter of 2020. Revenue increased 40% to $1.7 million compared to $1.2 million for the third quarter of 2019 (Press release, Orgenesis, NOV 5, 2020, View Source [SID1234570022]). The Company also reported approximately $88.8 million of cash and cash equivalents as of September 30, 2020.

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Vered Caplan, CEO of Orgenesis, stated, "Orgenesis continues to gain traction with a disruptive, point of care strategy for potentially commercializing life-changing treatments at reduced costs for large numbers of patients. In Q3 2020, we expanded the POCare Platform to include new POCare Therapeutics, Technologies, and a growing global Network."

"Orgenesis recently completed an acquisition of Koligo Therapeutics, Inc., a regenerative medicine company, including substantially all of the assets of Tissue Genesis, LLC. This acquisition helped to expand our therapeutic and technology resources, while adding a highly experienced US team to help further bolster Orgenesis’ POCare Network in the US."

"On the therapeutic front, Orgenesis is focused on several key verticals, including immuno-oncology, anti-viral, and metabolic/auto-immune diseases. A near-term goal is expanding the availability of KYSLECEL from the recent Koligo acquisition. KYSLECEL is commercially available in the United States for chronic and recurrent acute pancreatitis. We are also planning patient recruitment for a phase 2 randomized clinical trial of KT-PC-301, subject to FDA review and clearance of an investigational new drug (IND) application. KT-PC-301 is an autologous clinical development stage cell therapy candidate for COVID-19-related Acute Respiratory Distress Syndrome, which we also acquired as part of the Koligo acquisition. Additionally, Orgenesis is preparing for a Phase 2 study of Ranpirnase for the treatment of conditions caused by human papilloma virus pending a planned IND submission to the FDA.

"Orgenesis intends to leverage our network of regional partners to advance the development and commercialization of our therapeutic pipeline. Towards this end, our partners have committed to funding the clinical programs. In turn, Orgenesis typically grants its partners geographic rights in exchange for future royalties, and a partnership with Orgenesis to support the supply of the targeted therapies. Through this unique model, Orgenesis has already signed contracts, which we expect to generate over $40 million in revenue over the next three years, if fully realized. There are also plans to continue to develop, license and form partnerships around a variety of POCare Technologies to support work in areas such as Tumor Infiltrating Lymphocytes (TILS), CAR-T, CAR-NK, dendritic cell therapies, and mesenchymal stem cell (MSC) based therapies."

"Finally, Orgenesis is ready to announce advancements on proprietary, cell and gene processing units and labs that are being developed using first-in-class automation technologies. Orgenesis Mobile Processing Units & Labs ("OMPULs") are designed to provide an economical industrial alternative for our POCare Network partners to produce cell and gene therapies at the point of care. Orgenesis intends to roll these OMPULs out in centers that we are establishing across the US, Europe, Asia, and the Middle East."

The Company’s complete financial results are available in the Company’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 5, 2020 which is available at www.sec.gov and on the Company’s website.