On February 25, 2021 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide, reported recent business highlights, anticipated upcoming milestones, and financial results for the fourth quarter and full year of 2020 (Press release, BeiGene, FEB 25, 2021, View Source [SID1234575645]).

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"We made significant progress in the fourth quarter of 2020 and more recently with our collaboration agreement with Novartis to develop and commercialize tislelizumab in North America, Europe, and Japan, two positive Phase 3 readouts for tislelizumab demonstrating overall survival benefits over standard of care chemotherapy, and the expansion of our commercial portfolio, including the recent approval of tislelizumab in China in first-line squamous non-small cell lung cancer," said John V. Oyler, Co-Founder, Chief Executive Officer, and Chairman of BeiGene. "Our commercial teams continue to execute, with product revenue of $100 million for the fourth quarter and $309 million for the year, representing increases of 76% and 39%, respectively, over the prior year periods. With the recent inclusion of our products on the National Reimbursement Drug List in China we are working to expand access to our oncology treatments across China and drive further revenue growth."

Recent Business Highlights and Upcoming Milestones

Commercial Operations

Generated $100.10 million and $308.87 million in product revenue in the three and twelve months ended December 31, 2020, respectively. Product revenue consisted of sales in China of tislelizumab and sales of BRUKINSA in China and the United States, as well as sales in China of in-licensed products from our collaborations with Amgen and Bristol Myers Squibb (BMS); and
Announced inclusion of tislelizumab, BRUKINSA (zanubrutinib) and XGEVA (120-mg denosumab) in five indications in the updated National Reimbursement Drug List (NRDL) in China.
Development Programs

BRUKINSA (zanubrutinib), a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to maximize BTK occupancy and minimize off-target effects, approved in the United States and China in selected indications and under development for additional approvals globally.

Announced that the U.S. Food and Drug Administration (FDA) has accepted a supplemental new drug application (sNDA) for the treatment of adult patients with Waldenström’s macroglobulinemia (WM). The Prescription Drug User Fee Act (PDUFA) target action date is October 18, 2021;
Advanced BRUKINSA in new markets, with more than 20 marketing authorization applications submitted outside of the United States and China, covering more than 40 countries and regions, including by BeiGene in the European Union (EU), Canada, Australia, South Korea, Singapore, and Taiwan, and with support from our five distribution partners: Adium Pharma S.A. in Latin America and the Caribbean, NewBridge Pharmaceuticals in the Middle East and North Africa, Erkim in Turkey, Nanolek in Russia, and Medison in Israel. The first approval from these applications was received in the United Arab Emirates for BRUKINSA in patients with relapsed/refractory (R/R) mantle cell lymphoma (MCL);
Achieved full enrollment in the Phase 3 ALPINE trial (NCT03734016) comparing BRUKINSA with ibrutinib in patients with R/R chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL);
Dosed the first patient in a Phase 2, randomized, placebo-controlled clinical trial (NCT04643470) to evaluate the safety and efficacy of zanubrutinib in patients with active proliferative lupus nephritis;
Received inclusion in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines) for chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) for front-line patients with del(17p)/TP53 mutation who are contraindicated to other BTKi therapies and for patients in the second-line who have intolerance or are contraindicated to other BTKi therapies. An additional guideline note was included for patients with marginal zone lymphoma (MZL) who have intolerance or contraindications to ibrutinib. BRUKINSA is not approved in these indications; and
Presented clinical data at the 62nd American Society for Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, including initial results of the Phase 2 MAGNOLIA trial (NCT03846427) in patients with R/R MZL; follow-up results from the non-randomized Arm C in the randomized, open-label, global Phase 3 SEQUOIA trial (NCT03336333) of zanubrutinib as a monotherapy in patients with previously untreated CLL or SLL; results from a Phase 2 trial (NCT04116437) in patients with R/R B-cell malignancies who were intolerant to ibrutinib and/or acalabrutinib; and the first results from a pivotal Phase 2 trial (NCT03332173) in patients with R/R WM that were included in an sNDA of BRUKINSA currently under priority review in China.
Expected Milestones for BRUKINSA

Announce topline results from the Phase 3 SEQUOIA trial (NCT03336333) comparing BRUKINSA with bendamustine plus rituximab in patients with treatment-naïve CLL/SLL as early as 2021;
Announce topline results of the Phase 3 ALPINE trial (NCT03734016) versus ibrutinib in R/R CLL/SLL in the first half of 2022;
Continue to expand BRUKINSA’s registration program globally in new geographies or indications, including potential approvals in 2021 for certain patients with MCL in the EU, Middle East, South America, Canada, Australia, and Russia; and with WM in the U.S., EU, Canada, and Australia; and
Complete enrollment in the pivotal global Phase 2 ROSEWOOD trial (NCT03332017) comparing zanubrutinib and obinutuzumab versus obinutuzumab alone in treating patients with R/R follicular lymphoma (FL) in 2021.
Tislelizumab, a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages; approved in China in selected indications and under development for additional approvals globally.

Announced a collaboration and license agreement with Novartis Pharma AG to develop, manufacture and commercialize tislelizumab in the United States, Canada, Mexico, member countries of the EU, United Kingdom, Norway, Switzerland, Iceland, Liechtenstein, Russia, and Japan. Closing of the transaction is subject to the expiration or early termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act;
Announced approval in China for tislelizumab in combination with chemotherapy as a first-line treatment for patients with advanced squamous non-small cell lung cancer (NSCLC);
Announced positive topline results from the global Phase 3 RATIONALE 303 trial (NCT03358875) of tislelizumab versus docetaxel in the second- or third-line setting in patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy; and from the global Phase 3 RATIONALE 302 trial (NCT03430843) of tislelizumab versus chemotherapy in patients with advanced unresectable or metastatic esophageal squamous cell carcinoma (ESCC) who have received prior systemic treatment;
Achieved full enrollment in the global Phase 3 trial (NCT03783442) of tislelizumab in combination with chemotherapy as a first-line treatment in patients with unresectable, locally advanced or metastatic ESCC; and in the global Phase 3 trial (NCT03777657) of tislelizumab in combination with chemotherapy as a first line treatment in patients with inoperable, locally advanced or metastatic gastric or gastroesophageal junction carcinoma; and
Dosed the first patient in the Phase 3 trial (NCT04486391) of tislelizumab monotherapy versus salvage chemotherapy in patients with R/R classical Hodgkin’s lymphoma (cHL).
Expected Milestones for Tislelizumab

Close transaction with Novartis on the tislelizumab collaboration in the first quarter of 2021, subject to expiration or early termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act;
Submit the first biologics license application (BLA) outside of China in 2021;
Submit supplemental biologics license applications (sBLAs) in China for second/third-line NSCLC and MSI-H/dMMR solid tumors in the first half of 2021; and for second-line ESCC in mid-2021;
Receive approvals in first-line non-squamous NSCLC and second/third-line hepatocellular carcinoma (HCC) in China in 2021;
Announce topline result of the Phase 3 trial (NCT03924986) of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment in patients with nasopharyngeal cancer (NPC) in 2021;
Complete enrollment of Phase 3 trial in first-line small-cell lung cancer (NCT04005716) in the first half of 2021; and
Complete enrollment in the Phase 3 trial (NCT03957590) of tislelizumab versus placebo in combination with chemoradiotherapy in patients with localized ESCC in 2021.
Pamiparib, an investigational selective small molecule inhibitor of PARP1 and PARP2

Expected Milestones for Pamiparib

Receive approval in China for the treatment of patients with deleterious or suspected deleterious germline BRCA-mutated advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy, in the first half of 2021; and
Announce topline results from the Phase 3 trial (NCT03519230) of pamiparib as a maintenance treatment in patients with platinum-sensitive recurrent ovarian cancer (OC) in 2021 or first half of 2022.
Ociperlimab (BGB-A1217), an investigational TIGIT monoclonal antibody

Continued to enroll patients in the global Phase 1 clinical trial (NCT04047862) in combination with tislelizumab and plan for global registration trials.
Expected Milestones for Ociperlimab

Initiate a global Phase 3 AdvanTIG-302 trial (NCT04746924) of ociperlimab in combination with tislelizumab for the first-line treatment of patients with locally advanced, unresectable, or metastatic NSCLC whose tumors have high PD-L1 expression and do not harbor EGFR-sensitizing mutations or ALK translocations. Patient enrollment is expected to begin in the first half of 2021;
Initiate a global Phase 2 trial (NCT04693234) of ociperlimab in combination with tislelizumab in patients with previously treated recurrent or metastatic cervical cancer. Patient enrollment is expected to begin in the first half of 2021; and
Initiate a global Phase 2 trial (NCT04732494) of tislelizumab plus ociperlimab versus tislelizumab plus placebo for the second-line treatment of patients with unresectable, locally advanced, recurrent or metastatic ESCC whose tumors have high PD-L1 expression. Patient enrollment is expected to begin in the first half of 2021.
Early-Stage Programs

Continued to advance our early-stage clinical pipeline of internally-developed product candidates, including BGB-11417 (BCL-2 inhibitor in Phase 1 development for cancer), BGB-A445 (non-ligand competing OX40 monoclonal antibody in Phase 1 development in combination with tislelizumab for solid tumors), and BGB-10188 (PI3Kδ inhibitor in Phase 1 development in combination with BRUKINSA or tislelizumab for cancer); and
Identified a recommended Phase 2 dose for BGB-A425 (TIM-3 inhibitor) in the combination trial (NCT03744468) with tislelizumab in patients with advanced solid tumors.
Expected Milestones for Early-Stage Programs

Initiate a Phase 1 clinical trial (NCT04649385) of BGB-15025 (HPK1 inhibitor) alone and in combination with tislelizumab in patients with advanced solid tumors in the first half of 2021; and
Initiate the Phase 2 portion of the Phase 1/2 trial (NCT03744468) of BGB-A425 in the first half of 2021.
Collaboration with Amgen

Received approval in China of BLINCYTO (blinatumomab) for the treatment of adult patients with R/R B-cell precursor acute lymphoblastic leukemia (ALL);
Received approval in China of XGEVA (denosumab) for the prevention of skeletal-related events (SREs) in patients with bone metastases from solid tumors and in patients with multiple myeloma (MM);
Our collaboration with Amgen continues to progress, with ongoing preparation for the launch of KYPROLIS (carfilzomib) for patients with R/R multiple myeloma following potential approval expected in 2021;
Amgen announced that its investigational KRASG12C inhibitor sotorasib was granted Breakthrough Therapy Designation (BTD) in China for patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC) who have received at least one prior systemic therapy; and
Amgen’s applications to the Human Genetic Resources Administration of China (HGRAC) to conduct clinical trials in mainland China continue to be delayed. Approval from the HGRAC is required to initiate clinical trials involving the collection of human genetic materials in China. BeiGene does not expect this to affect the conduct of the clinical trials in China for its drug candidates other than assets that are part of the Amgen-BeiGene collaboration.
Other Collaboration Programs

Received approval in China for REVLIMID (lenalidomide), licensed from BMS, in combination with rituximab for adult patients with previously treated FL (grade 1-3a);
Announced that the BLA for SYLVANT (siltuximab for injection), was accepted by the China National Medical Products Administration (NMPA) and granted priority review; and announced the acceptance of the BLA and priority review for QARZIBA▼ (dinutuximab beta) for the treatment of high-risk neuroblastoma. These products are licensed in China from EUSA Pharma (EUSA);
Announced an agreement with Strand Therapeutics for an option and license agreement to develop and commercialize Strand’s innovative, multi-functional mRNA treatments for solid tumors in Asia (excluding Japan), Australia, and New Zealand;
Announced an option and license agreement with Boston Immune Technologies and Therapeutics, Inc. (BITT) to develop and commercialize BITT’s innovative tumor necrosis factor (TNF) receptor 2 (TNFR2) antagonist antibodies in Asia (excluding Japan), Australia, and New Zealand;
Treated the first patient in the global, pivotal, single-arm HERIZON-BTC-01 Phase 2b clinical trial (NCT04466891) of zanidatamab (ZW25) in patients with advanced or metastatic HER2-amplified biliary tract cancers (BTC). Zanidatamab , is in late-stage clinical development with Zymeworks Inc. A Phase 3 trial of zanidatamab (ZW25) in combination with chemotherapy with or without tislelizumab in front line HER2 positive gastroesophageal cancer is expected to initiate in 2021. BeiGene has development and commercial rights to zanidatamab in Asia (excluding Japan), Australia, and New Zealand; and
Assembly Biosciences announced that it will not move forward with planned Phase 3 registration studies of vebicorvir (VBR or ABI-H0731) as a chronic suppressive therapy (CST). BeiGene has licensed vebicorvir and two other clinical-stage core inhibitor candidates for the treatment of patients with chronic hepatitis B virus (HBV) infection from Assembly in China.
Manufacturing Operations

Completed GMP qualification for the second phase of our biologics manufacturing facility in Guangzhou, China, with total capacity of 24,000 liters for the completed first and second phases. Approval to manufacture commercial product is expected in the first half of 2021. A third phase of construction is expected to add 40,000 liters, with 30,000 liters already in place. Total capacity of 64,000 liters and the addition of new manufacturing technology platforms are expected to be completed by the first half of 2022.
COVID-19 Impact and Response

The Company expects that the worldwide health crisis of COVID-19 will continue to have a negative impact on its operations, including commercial sales, regulatory interactions, inspections, filings, and clinical trial recruitment, participation, and data read outs. There remains uncertainty regarding the future impact of the pandemic globally. The Company is striving to minimize delays and disruptions, and continues to execute on its commercial, regulatory and clinical development goals globally.
Other Developments

Filed an initial listing application for a proposed public offering and listing of the Company’s ordinary shares on the Science and Technology Innovation Board (STAR Market) of the Shanghai Stock Exchange, which is expected to be completed in 2021, subject to market conditions, shareholder approval, and regulatory approvals;
Appointed Dr. Xiaobin Wu, currently the Company’s President and General Manager, China, to the additional position of the Company’s Chief Operating Officer, effective April 1, 2021;
Appointed Dr. Lai Wang, the Company’s Senior Vice President, Head of Global Research, Clinical Operations and Biometrics, and APAC Clinical Development to the position of Global Head of R&D, effective April 1, 2021; and
Appointed Graham Hardiman to the position of Senior Vice President, Head of Global Human Resources, effective March 1, 2021. Mr. Hardiman joins BeiGene from Pfizer where he most recently held the position of Senior Vice President, Human Resources.
Fourth Quarter and Full Year 2020 Financial Results

Cash, Cash Equivalents, Restricted Cash and Short-Term Investments were $4.66 billion as of December 31, 2020, compared to $4.72 billion as of September 30, 2020, and $985.50 million as of December 31, 2019. Cash and cash equivalents as of December 31, 2020 do not include the $650 million upfront payment from the Novartis collaboration, the closing of which is subject to the expiration or early termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.

For the fourth quarter of 2020, total cash and short-term investments decreased $65.29 million; cash used in operating activities was $332.33 million; capital expenditures were $34.69 million; cash used for upfront license payments was $20.00 million; and cash provided from financing activities, primarily due to the drawing down of bank loans, was $325.66 million.
For the full year 2020, total cash and short-term investments increased $3.67 billion; cash used in operating activities was $1.28 billion; capital expenditures were $117.51 million; cash used for upfront license payments was $109.50 million; and cash provided from financing activities was $5.20 billion.
Revenue for the fourth quarter and full year 2020 was $100.10 million and $308.87 million, respectively, compared to $56.89 million and $428.21 million in the prior year periods. The increase in total revenue in the quarter compared to the prior year is attributable to sales of our internally developed products and initial sales of in-licensed products from Amgen, offset by decreased product sales of in-licensed products from BMS.

Product revenues totaled $100.10 million and $308.87 million for the fourth quarter and full year 2020, respectively, compared to $56.89 million and $222.60 million in the prior year periods, and were comprised of:
Sales of tislelizumab in China of $63.48 million and $163.36 million for the fourth quarter and full year 2020, respectively, compared to none in the prior year periods. Full year 2020 revenue from tislelizumab reflect sales since its launch in China in March 2020;
Sales of BRUKINSA in China and the United States of $18.35 million and $41.70 million for the fourth quarter and full year 2020, respectively, compared to $1.04 million in the prior year periods. Full year 2020 revenue from BRUKINSA reflects sales since its launch in China in June 2020, as well as sales in the United States for the full year;
Sales of BMS in-licensed products in China of $12.62 million and $95.12 million for the fourth quarter and full year 2020, respectively, compared to $55.85 million and $221.56 million in the prior year periods, respectively; and
Sales of Amgen in-licensed products in China of $5.45 million and $8.50 million for the fourth quarter and full year 2020, respectively, compared to none in the prior year periods. We began selling Amgen’s XGEVA in July 2020.
There was no collaboration revenue for the fourth quarter or full year 2020, compared to nil and $205.62 million in the prior year periods, respectively. Collaboration revenue for the full year 2019 included a $150 million payment in connection with the termination of the tislelizumab collaboration agreement with Celgene, which was acquired by BMS.
Expenses for the fourth quarter and full year 2020 were $585.01 million and $1.97 billion, respectively, compared to $444.93 million and $1.39 billion in the prior year periods.

Cost of Sales for the fourth quarter and full year 2020 were $21.08 million and $70.66 million, respectively, compared to $17.98 million and $71.19 million in the prior year periods. Cost of sales increased primarily as a result of the launch of tislelizumab, BRUKINSA, and XGEVA, and were offset by lower sales volumes of the BMS in-licensed products.
R&D Expenses for the fourth quarter and full year 2020 were $355.54 million and $1.29 billion, respectively, compared to $283.26 million and $927.34 million in the prior year periods. The increase in R&D expenses was primarily attributable to increased spending on our ongoing and late-stage pivotal clinical trials, expense related to upfront license payments for in-licensed assets, development expenses associated with the Amgen collaboration, the preparation of additional regulatory submissions, and manufacturing costs related to development programs and pre-commercial activities. Upfront fees related to in-process R&D for in-licensed assets totaled nil and $109.50 million in the fourth quarter and full year 2020, respectively, compared to $20.00 million and $50.00 million in the prior year periods. Employee share-based compensation expense also contributed to the overall increase in R&D expenses, and was $23.48 million and $93.00 million for the fourth quarter and full year 2020, respectively, compared to $21.69 million and $76.29 million in the prior year periods, due to increased headcount and a higher share price.
SG&A Expenses for the fourth quarter and full year 2020 were $208.21 million and $600.18 million, respectively, compared to $143.35 million and $388.25 million in the prior year periods. The increase in SG&A expenses was primarily attributable to increased headcount, largely related to continued expansion of our commercial teams, higher professional service fees and higher external commercial expenses, including selling and marketing, market access studies and promotional activities. The overall increase in SG&A expenses was also attributable to higher SG&A-related share-based compensation expense, which was $25.98 million and $90.48 million for the fourth quarter and full year 2020, respectively, compared to $16.65 million and $57.86 million for the prior year periods, due to increased headcount and a higher share price.
Net Loss for the fourth quarter and full year 2020 was $472.75 million and $1.60 billion, or $0.40 and $1.47 per share, respectively, or $5.20 and $19.13 per ADS, respectively, compared to $388.06 million and $948.63 million, or $0.49 and $1.22 per share, or $6.39 and $15.80 per ADS, respectively, in the prior year periods.

[1] Research and development expense for the fourth quarter and full year 2020 includes upfront fees related to in-process research and development of in-licensed assets totaling nil and $109.50 million, respectively, compared to $20.00 million and $50.00 million in the prior year periods.

On February 25, 2021 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported business updates and financial results for the fourth quarter ended December 31, 2020 (Press release, Alector, FEB 25, 2021, View Source [SID1234575644]).

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"In 2020, we made significant progress across all of our key clinical programs," said Arnon Rosenthal, Ph.D., co-founder and chief executive officer of Alector. "As we look ahead to 2021, we will be sharing additional findings from our ongoing Phase 2 study evaluating AL001 in people with frontotemporal dementia at a medical conference this year and are pleased to be advancing our two Alzheimer’s disease development programs. Our progress across our immuno-neurology programs continues to put us one step closer to potentially offering new therapeutic options for the millions of patients living with various forms of dementia. In addition, we continue to progress research targets that could have broad implications across neurodegenerative diseases and various forms of cancer, underscoring the broad potential of our research platform."

2020 and Recent Clinical Pipeline Highlights and Business Updates

Progranulin Portfolio:

In July 2020, Alector dosed the first participant in INFRONT-3, a randomized, placebo-controlled, pivotal Phase 3 trial evaluating AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). The ongoing trial is evaluating the efficacy and safety of AL001 in at-risk and symptomatic participants with FTD-GRN. Participants in the trial will be given the option to continue receiving treatment in an open-label extension study.

Alector plans to present updated data from the ongoing Phase 2 open-label study evaluating AL001, in 2021. The Company plans to present additional data in pre-symptomatic and symptomatic FTD-GRN participants at medical conferences in 2021. The data will include updated findings on safety, fluid and imaging biomarkers and clinical outcomes assessments, while also providing additional insights to the mechanism of action and safety of AL001 in this population.

In January 2021, the Company announced plans to evaluate AL001 in people with amyotrophic lateral sclerosis (ALS) caused by C9orf72 repeats, which share TDP-43 pathology with FTD-GRN. The Phase 2 study is planned for 2021.
In January 2020, a Phase 1a study evaluating AL101 was initiated in healthy volunteers. The U.S. Food and Drug Administration also granted Fast Track designation to AL101 for the treatment of patients with FTD-GRN in February 2020. The Company anticipates reporting preliminary findings from the Phase 1a study in 2021.
Alzheimer’s Disease Portfolio:

In January 2021, the Company announced that the first participant was dosed in INVOKE-2, Phase 2 trial evaluating AL002 in people with early Alzheimer’s disease. The randomized, double-blind, placebo-controlled, dose-ranging, multi-center Phase 2 study will enroll approximately 265 participants with early Alzheimer’s disease (AD) at up to 90 sites globally. The program is being developed in collaboration with AbbVie.

Data from the Phase 1b study evaluating AL003 in participants with Alzheimer’s disease is expected in 2021. Alector initiated the Phase 1b study in January 2020 and completed enrollment in 2020. The AL003 clinical development program is also being developed in collaboration with AbbVie. Preliminary results from the study are expected to be presented at a scientific medical meeting in 2021.
Early-Stage Pipeline with Potential in Neurodegenerative Diseases and Oncology

In March 2020, the Company entered into a collaboration with Innovent Biologics to develop and commercialize AL008 for oncology indications in China. Planning for the first-in-human study for AL008 is underway. AL008 is a novel, investigational, antibody product candidate with a dual mechanism of action that combines inhibition of the CD47-SIRP-alpha (SIRPα) pathway, with stimulation of activating Fc receptors that has the potential to yield a best-in-class product.

Plans are currently underway to initiate a first-in-human study of a product candidate in our AL009 development program, the Company’s latest prioritized investigational product candidate. Our AL009 program is developing a first-in-class multi-Siglec inhibitor that works to enhance the innate and adaptive immune system response by blocking a critical glycan checkpoint pathway that drives immune inhibition. The product candidate is initially being developed for oncology indications, but may also have therapeutic application in certain neurodegenerative disorders, such as AD.

The Company continues to plan for a first-in-human study for AL044, targeting the MS4A4A receptor within the next 18 months. MS4A4A is a major risk gene for AD that encodes a transmembrane receptor protein that is expressed selectively in microglia in the brain and is associated with control of microglia functionality and potential viability.
Ongoing COVID-19 Response Activities:

Alector continues to actively monitor the evolving COVID-19 pandemic and its ongoing impact on business and clinical operations.
Fourth Quarter and Full Year 2020 Financial Results

Revenue. Collaboration revenue for the quarter ended December 31, 2020, was $4.9 million, compared to $6.0 million for the same period in 2019. Collaboration revenue for the year ended December 31, 2020, was $21.1 million compared to $21.2 million for the same period in 2019. Alector recognizes revenue from the upfront payments under an agreement with AbbVie over time as the services are provided. Revenues are recognized as the program costs are incurred by measuring actual costs incurred to date compared to the overall total expected costs to satisfy the performance obligation. Changes in estimates for revenue recognized over time are recognized on a cumulative basis.

R&D Expenses. Total research and development expenses for the quarter ended December 31, 2020, were $44.4 million, compared to $25.8 million for the same period in 2019. Total research and development expenses for the year ended December 31, 2020, were $156.9 million compared to $100.5 million for the same period in 2019. This increase was mainly driven by an increase in expenses to support the advancement of the clinical and pre-clinical programs across several therapeutic programs and an increase in personnel-related expenses.

G&A Expenses. Total general and administrative expenses for the quarter ended December 31, 2020, were $13.2 million, compared to $12.6 million for the same period in 2019. Total general and administrative expenses for the year ended December 31, 2020 were $59.4 million compared to $35.1 million for the same period in 2019. This increase was primarily due to an increase in personnel-related expenses due to increased headcount and an increase in legal costs associated with our arbitration proceedings.

Net Loss. For the quarter ended December 31, 2020, Alector reported a net loss of $52.2 million, compared to a net loss of $30.5 million for the same period in 2019. For the year ended December 31, 2020, Alector reported a net loss of $190.2 million, compared to a net loss of $105.4 million for the same period in 2019.

Cash Position. Cash, cash equivalents, and marketable securities were $413.3 million as of December 31, 2020. The Company believes that its cash and investments as of December 31, 2020, will be sufficient to fund its anticipated operations through mid-2022.

On February 25, 2021 Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), reported its financial results for the fourth quarter and year ended December 31, 2020 and provided several additional corporate updates (Press release, Ziopharm, FEB 25, 2021, View Source [SID1234575643]). The Company will host a conference call and webcast today at 4:30 pm ET.

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Leadership Transition and Planning

The Company reported that Heidi Hagen, formerly Lead Independent Director, has been appointed Interim Chief Executive Officer, replacing Dr. Laurence Cooper, MD., Ph.D. effective February 25, 2021. Ms. Hagen is remaining a member of the Board of Directors.

Dr. Cooper is also stepping down from his seat on the Board of Directors and is expected to continue with the Company in a scientific advisory capacity to support the Company’s R&D programs. A search for a permanent Chief Executive Officer is underway.

Dr. Cooper said, "With the fantastic news we announced today regarding the IND clearance of our Library TCR-T clinical studies, the Company is well positioned as a leader in immuno-oncology using engineered T-cells. I will work with the Board on transitioning to an advisory role to support the organization on the science side, while allowing the Company to identify a complementary business leader who can drive our path to commercialization."

Ms. Hagen added, "One cannot overstate Laurence’s contribution to Ziopharm. His life’s work has been to bring innovation and hope to patients suffering from the devastating impact of cancer. We will continue down the path Laurence has laid before us, and look forward to his continued involvement to help us address the scientific challenges ahead."

Ms. Hagen has served on the Board since June 2019. She is co-founder of Vineti, a cloud-based software platform company that addresses challenges in data management from order through cell collection, manufacturing, and delivery of personalized treatments such as cell and gene therapies and cancer vaccines. She has extensive experience in operations management and commercializing innovative technologies.

The Company also announced today that James Huang has been appointed Executive Chairman of the Board effective February 25, 2021.

Mr. Huang said, "On behalf of the entire Board we thank Laurence for his leadership and vision and express our full support for Heidi while we conduct a comprehensive search for a permanent CEO. We will strive to identify a leader with the business acumen to drive critical portfolio, development, commercial planning and capital allocation decision making that will help ensure the success of the Company."

Mr. Huang has served on the Board since July 2020 and has served as Chairman since January 2021. He is currently a Managing Partner at Kleiner Perkins Caufield & Byers (KPCB) China. He has founded and financed several innovative life sciences companies, including GenScript, Legend Biotech and Zai Lab. He is also Founding Partner of Panacea Venture, which formed TriArm Therapeutics, the funding partner for Ziopharm’s joint venture, Eden BioCell.

FDA IND Clearance for the Company’s Library TCR-T Clinical Phase I/II Trial

The Company reported it has received IND clearance by the U.S. Food and Drug Administration (FDA) for the Company’s TCR-T trial utilizing six "hotspot" TCRs from its library. The Company anticipates enrolling patients in the Phase I/II clinical trial across a variety of solid tumor cancers in the second half of the year.

The Company is working closely with MD Anderson to begin identifying patients for this trial. The trial will address a range of solid tumors, across gynecologic, colorectal, pancreatic, non-small cell lung and cholangiocarcinoma cancers.

"We are very excited to have received clearance for this IND and look forward to initiating the Library TCR-T trial, representing a tremendous amount of work by the team and the culmination of efforts by so many dedicated employees," said Dr. Eleanor de Groot, Ph.D., Executive Vice President and General Manager of Cell Therapy of Ziopharm. "We believe our cell therapy Sleeping Beauty platform technology has the potential to deliver non-viral engineered T-cell therapies to address significant unmet patient need and are excited by this important transition to clinical development for the Library program."

Additional Details Regarding R&D Day March 11, 2021

The Company provided additional details regarding the previously announced virtual R&D Day focusing on cell therapy on Thursday, March 11, 2021 at 11:00 am ET. Members of Ziopharm’s management team will provide an overview of the Company’s strategy, programs, and pipeline.

The session will also include presentations by leading key opinion leaders: Dr. Steven Rosenberg, Chief of Surgery at the National Cancer Institute; Dr. Carl June, Chair of the Ziopharm Scientific Advisory Board and Director of the Center for Cellular Immunotherapies and Director of Translational Research in the Abramson Cancer Center of the University of Pennsylvania; and Dr. Scott Kopetz, Colorectal Cancer Physician Scientist, NCI Colon Task Force Chair, Professor, and Deputy Chair at The University of Texas, MD Anderson Cancer Center.

"We are very excited to share updates on our suite of distinctive cell therapy programs and delighted to have top key opinion leaders joining us to provide their views and perspectives. We will be highlighting the unique attributes of Ziopharm and the encouraging progress in our programs," said Dr. Raffaele Baffa, M.D., Ph.D., Chief Medical Officer of Ziopharm.

Fourth Quarter 2020 Financial Results

Research and development expenses were $14.0 million for the fourth quarter of 2020, compared to $10.2 million for the fourth quarter of 2019, primarily reflecting increased clinical trial activity.

General and administrative expenses were $8.8 million for the fourth quarter of 2020, compared to $5.8 million for the fourth quarter of 2019. The increase in general and administrative expenses for the fourth quarter of 2020 is primarily due to increased legal costs, investor relations costs and facility charges.

Net loss for the fourth quarter of 2020, was $22.8 million, or $(0.11) per share, compared to a net loss of $15.7 million, or $(0.09) per share, for the fourth quarter of 2019.

Cash and cash equivalents, as of December 31, 2020 were $115.1 million. This cash position is sufficient to fund Company operations into the second quarter of 2022.

A prepayment of approximately $8.1 million remains for work to be conducted by the Company at MD Anderson under the Company’s research and development agreements.
Full Year 2020 Financial Results

Net loss applicable to the common shareholders for the year ended December 31, 2020 was $80.0 million, or $(0.38) per share, basic and diluted, compared to net loss applicable to the common shareholders of $117.8 million, or $(0.70) per share, basic and diluted, for the year ended December 31, 2019.

Research and development expenses were $52.7 million for the year ended December 31, 2020, compared to $38.3 million for the year ended December 31, 2019. The increase in research and development expenses for the year ended December 31, 2020 is primarily due to increased manufacturing, headcount, and clinical trial activity.

General and administrative expenses were $27.7 million for the year ended December 31, 2020, compared to $19.5 million for the year ended December 31, 2019. The increase in general and administrative expenses for the year ended December 31, 2020 is primarily due to increased legal costs, investor relations costs and facility charges.
Fourth Quarter and Full Year 2020 Results Conference Call and Webcast Details
Ziopharm will host a conference call and webcast for the investment community today, February 25, 2021, at 4:30 pm ET. The conference call can be accessed by dialing 877-451-6152 (U.S. and Canada) or +1-201-389-0879 (International). The passcode for the conference call is 13715482. To access the live webcast or the subsequent archived recording, click here or visit the "Investors" section of the Ziopharm website at www.ziopharm.com. The webcast will be recorded and available for replay on the company’s website for 90 days.

R&D Day Conference Call and Webcast Details – March 11, 2021, 11:00am ET
Interested participants can register for and view the webcast using this link or by visiting the "Investors" section of the Ziopharm website at www.ziopharm.com. The live Q&A session can be accessed by dialing 866-548-4713 (U.S. and Canada) or +1-323-794-2093 (International). The conference ID for the call is 5859801. The session will be recorded and available for replay on the Company’s website for 90 days.

On February 25, 2021 Vaxart, Inc. (Nasdaq: VXRT) a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, reported that it plans to initiate the first Phase 2 study of its oral COVID-19 vaccine candidate, VXA-CoV2-1, in 2Q 2021 (Press release, Aviragen Therapeutics, FEB 25, 2021, View Source [SID1234575642]).

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Vaxart made this announcement as it provided financial results for the fourth quarter and full year ended December 31, 2020 and provided a corporate update.

"Recently, we have seen the emergence of new SARS-CoV-2 strains, against which some of the leading injectable vaccines offer reduced protection. At the same time, it has become clear that mass vaccinations by needle take a long time, and new strains may emerge faster than many countries’ medical and governmental infrastructure can inject their populations. A better solution is needed given that COVID-19 may be a challenge for years to come," said Andrei Floroiu, chief executive officer of Vaxart. "As a result of our scientists’ foresight to include both the S and N proteins, VXA-CoV2-1 could be protective against these newly emerging variant strains. We are very excited about the prospect of moving into Phase 2 not only as an oral COVID-19 vaccine candidate, but one with a differentiated mechanism, which could prove to be valuable globally in the fight against coronavirus."

VXA-CoV2-1 triggers mucosal immune responses in humans. Mucosal immunity is believed to be the first line of defense against airborne viruses, such as coronavirus and flu, and may also be important in reducing viral shedding and preventing transmission.
VXA-CoV2-1 targets both the spike protein (S) and nucleoprotein (N). The N protein is more conserved (less prone to mutations) than the S protein, and therefore new viral variants may be less likely to escape protection.
The N protein is also a good target for T-cell responses. Potent T-cell responses alone may offer multi-variant protection against severe COVID-19 illness.
Vaxart is also advancing S-only vaccine candidates targeted specifically against variant strains, including one targeting the South African viral strain. These new candidates are expected to generate strong mucosal and serum antibody responses and may be complementary to the potent T-cell inducer VXA-CoV2-1. Vaxart has previously shown that a bivalent oral vaccine using its platform can induce immune responses without interference.
A conference call and webcast focused on our COVID-19 strategy will be held on Tuesday, March 2, 2021 at 4:30pm Eastern Time, Domestic: 877-407-0784, International: 201-689-8560, Conference ID: 13716984, Webcast: View Source

Recent Business Development Highlights:

Pre-Clinical and Clinical:

VXA-CoV2-1 Phase 1 trial met its primary and secondary endpoints. The vaccine was generally well-tolerated, with no severe adverse events reported. Results from the trial were presented by Dr. Sean Tucker, Vaxart’s Founder and Chief Scientific Officer, at the New York Academy of Sciences Symposium "The Quest for a COVID-19 Vaccine" in early February 2021. The presentation can be viewed on Vaxart’s corporate website on the Investors page under "Events and Presentations".
VXA-CoV2-1 triggered immune responses against SARS-CoV-2 antigens in a majority of subjects, including: CD8+ cytotoxic T-cell response to the S and N proteins (may contribute to long-lasting cross-reactive immunity), activation of B cells that will home to the mucosa, an increase in proinflammatory Th1 cytokines (responsible for creating a productive immune response against viral infection) and IgA responses.
COVID-19 Hamster Challenge Study data showed that 100% of hamsters receiving two oral doses of Vaxart’s recombinant adenoviral vaccine were protected against systemic weight loss, as well as lung weight gain. Conversely, all unvaccinated animals lost at least 8% of their body weight, and all showed evidence of lung disease as measured by relative weight gain in the lungs. Full results from the study will be published when data analysis is complete.
Vaxart’s norovirus vaccine program has been restarted with the addition of a booster dose administered more than 12 months post first vaccination in subjects who participated in the Phase 1b trial. Data are expected to be available in the first half of 2021. Additional studies planned for 2021 include a Phase 1 study in elderly adults age 65+ and a Norovirus Challenge study.
Manufacturing:

Expanded collaboration with Kindred Biosciences for the manufacturing of VXA-CoV2-1 oral vaccine as well as other vaccine candidates. Under the terms of the expanded agreement, the California plant will be used for scaling the COVID-19 clinical trial material into mid-size bioreactors, and its Kansas plant will be used for manufacturing at a 2000L scale in its single use bioreactors.
Vaxart entered into an agreement with Attwill Vascular Technologies, LP for processing and lyophilizing certain compounds and further tableting the lyophilized compounds for the Company’s oral COVID-19 vaccine.
Corporate:

Strengthened the organization, bringing the total number of full-time equivalents to 49 people (including consultants and contractors), by hiring in critical areas, including research, clinical, regulatory, manufacturing, and finance.
Cash Balance:

Vaxart ended the year with cash and cash equivalents of $126.9 million compared to $13.5 million as of December 31, 2019. The increase was primarily due to receipts of $97.0 million from the Company’s $100 million at-the-market facility entered into in July 2020, $26.0 million from the exercise of warrants, $9.2 million from the registered direct offering in March 2020, and $4.9 million from the Company’s $250 million at-the-market facility entered into in October 2020 (October 2020 ATM), partially offset by $23.8 million of cash used in operations.
Subsequent to year end, the Company has raised net proceeds of $65.8 million from the issuance of common stock under the October 2020 ATM.
Financial Results for the Three Months Ended December 31, 2020

Vaxart reported a net loss of $13.9 million for the fourth quarter of 2020 compared to $6.4 million for the fourth quarter of 2019. Net loss per share for the fourth quarter of 2020 was $0.13, unchanged from 2019 due to an increase in the weighted average number of shares outstanding.
Revenue for the fourth quarter was $356,000 compared to $3.9 million in the fourth quarter of 2019. The decrease was principally due to a reduction in royalty revenue related to Inavir sales in Japan as a result of an abnormally low incidence of seasonal influenza, and a decline in contract revenue from Janssen which was substantially completed by September 30, 2020.
Research and development expenses were $8.6 million for the fourth quarter of 2020 compared to $3.3 million for the fourth quarter of 2019. The increase was mainly due to manufacturing and clinical trial expenses related to the COVID-19 vaccine candidate.
General and administrative expenses were $5.1 million for the fourth quarter of 2020 compared to $1.3 million for the fourth quarter of 2019. The increase was mainly due to higher legal and consulting, and an increase in headcount and related costs.
There were no restructuring expenses for the fourth quarter of 2020 compared to $4.9 million for the fourth quarter of 2019.
Financial Results for the Full Year 2020

Vaxart reported a net loss of $32.2 million for full year 2020 compared to $18.6 million for full year 2019. Net loss per share for 2020 was $0.36, down from $0.86 for 2019 due to the increase in net loss being outweighed by the increase in the weighted average number of shares outstanding during 2020.
Revenue in 2020 was $4.0 million compared to $9.9 million in 2019. The decrease was principally due to a reduction in royalty revenue related to Inavir sales in Japan due to abnormally low incidences of seasonal influenza in 2020, compared to higher-than-average incidences in 2019.
Research and development expenses were $19.9 million for 2020 compared to $14.5 million for 2019. The increase was mainly due to higher preclinical, manufacturing and clinical trial expenses related to our COVID-19 vaccine candidate.
General and administrative expenses were $15.2 million for 2020 compared to $6.2 million for 2019. The increase was mainly due to higher legal and consulting fees and an increase in headcount and related costs.
Restructuring charges for 2019 were $4.9 million, compared to a net reversal of $849,000 in 2020, principally due to a settlement with Lonza for less than the total amount invoiced. No further restructuring charges or reversals are expected.
(Press release, Aviragen Therapeutics, FEB 25, 2021, View Source [SID1234575642])

On February 25, 2021 Acceleron Pharma Inc. (Nasdaq: XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Acceleron Pharma, FEB 25, 2021, View Source [SID1234575641]).

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"2020 was a very productive year for Acceleron as we generated important results in our lead pulmonary program to advance sotatercept into a registrational Phase 3 trial and executed on a successful first full year of the commercial launch of REBLOZYL in the United States," said Habib Dable, President and Chief Executive Officer of Acceleron. "I would like to acknowledge our team’s strong commitment to our company and mission as these important milestones along with many others were achieved despite the many challenges resulting from the ongoing global pandemic. Throughout the year, we presented updates from our two Phase 2 trials with our lead pulmonary program, supporting our long-term vision of establishing sotatercept as a backbone therapy for patients with pulmonary arterial hypertension across all stages of the disease."

Added Mr. Dable: "As we continue to grow our capabilities in rare pulmonary disease, we are also advancing ACE-1334 into a Phase1b/Phase 2 trial in patients with systemic sclerosis-associated interstitial lung disease later this year. We look forward to hosting a research and development day to provide additional details on our long-term vision and programs in rare pulmonary disease in the middle of 2021."

Program Highlights

Pulmonary

Sotatercept: Pulmonary Arterial Hypertension (PAH)

Sotatercept is an investigational reverse-remodeling agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. The PULSAR Phase 2 trial evaluating sotatercept in combination with approved PAH-specific medicines in patients with PAH achieved its primary endpoint of improvement in pulmonary vascular resistance and its key secondary endpoint of improvement in 6-minute walk distance.

In November, the Company presented the 24-week echocardiography results from the PULSAR Phase 2 trial at the virtual 2020 American Heart Association (AHA) Scientific Sessions, which earned the AHA’s "Cardiopulmonary Best Abstract" award. In addition, preliminary interim results from the SPECTRA Phase 2 trial were presented at the congress.
In December, the European Commission (EC) granted Orphan Drug designation to sotatercept for the treatment of patients with PAH.
In December, Acceleron initiated its registrational STELLAR Phase 3 trial in patients with PAH.
The Company expects to initiate the HYPERION (early intervention) Phase 3 trial and the ZENITH (WHO Functional Class IV) Phase 3 trial in expanded PAH populations in the middle of 2021.
Results from the open-label extension period of the PULSAR Phase 2 trial and additional results from the SPECTRA Phase 2 trial are expected in the first half of 2021.
ACE-1334: Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD)

ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2. ACE-1334 has shown robust anti-fibrotic activity in multiple preclinical models of fibrosis. ACE-1334 recently completed an ascending-dose Phase 1 clinical trial in healthy volunteers.

In December, the United States Food and Drug Administration (FDA) granted Orphan Drug designation to ACE-1334 for the treatment of patients with systemic sclerosis.
Acceleron expects to start a Phase 1b/Phase 2 study to evaluate the activity of ACE-1334 in patients with SSc-ILD in 2021.
Hematology

REBLOZYL (luspatercept-aamt):

REBLOZYL is the first and only approved erythroid maturation agent designed to promote late-stage red blood cell (RBC) production. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.

The Company recognized approximately $23.0 million in royalty revenue from approximately $115 million in net sales of REBLOZYL in the fourth quarter of 2020. This compares with approximately $19.3 million in royalty revenue from approximately $96 million in net sales of REBLOZYL in the third quarter of 2020.
For the full year 2020, Acceleron recognized approximately $54.8 million in royalty revenue from approximately $274 million in net sales of REBLOZYL.
In February 2021, Acceleron and partner Bristol Myers Squibb announced that Health Canada approved REBLOZYL for the treatment of adult patients with transfusion-dependent anemia requiring at least two RBC red blood cell units over 8 weeks resulting from very low- to intermediate-risk myelodysplastic syndromes (MDS) who have ring sideroblasts and who have failed or are not suitable for erythropoietin-based therapy.
Six clinical abstracts on REBLOZYL were presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition held virtually in December 2020.
The INDEPENDENCE Phase 3 trial is initiating in patients with myelofibrosis.
The Companies expect to present results from the BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta-thalassemia in the first half of 2021.
Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS, with topline results expected in 2022+.
Corporate Highlights

Acceleron is planning to host a research and development day in the middle of 2021 to provide a deep dive into all of the Company’s programs.
Financial Results

Cash Position – Cash, cash equivalents and investments as of December 31, 2020 were $857.5 million. Based on Acceleron’s current operating plan and projections, the Company believes that its current cash, cash equivalents and investments, along with the expected royalty revenue from REBLOZYL sales, will be sufficient to fund the Company’s projected operating requirements for the foreseeable future.
Revenue – Revenue for the fourth quarter of 2020 was $25.9 million, which includes $2.9 million of cost share revenue and $23.0 million of royalty revenue from net sales of REBLOZYL. Revenue for the full year was $92.5 million, which includes $12.7 million of cost share revenue, $54.8 million of royalty revenue from net sales of REBLOZYL, and the recognition of a $25.0 million regulatory-based milestone for the approval of REBLOZYL in Europe. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.
Costs and Expenses – Total costs and expenses for the fourth quarter of 2020 were $83.5 million. This includes R&D expenses of $57.3 million and SG&A expenses of $26.2 million. Total costs and expenses for the full year were $259.8 million. This includes R&D expenses of $173.9 million and SG&A expenses of $85.9 million.
Net Loss – The Company’s net loss for the fourth quarter of 2020 was $57.4 million. The Company’s net loss for the year ended December 31, 2020 was $166.0 million.
Conference Call and Webcast

The Company will host a webcast and conference call to discuss its fourth quarter and full year 2020 financial results on February 25, 2021, at 5:00 p.m. EST.

The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at www.acceleronpharma.com. To participate in the conference call, please dial 833-494-1483 (domestic) or 236-714-2620 (international) and reference code #4282162.

An archived version of the webcast will be available for replay on the Company’s website for approximately one year.