On February 11, 2021 Portage Biotech Inc. (CSE: PBT.U, OTC Markets: PTGEF) ("Portage" or the "Company") reported its 2021 research and development goals, including advancing three of its pipeline assets through clinical trials during the upcoming year (Press release, Portage Biotech, FEB 11, 2021, View Source [SID1234574927]). Portage also announced that it has received approval from the NASDAQ Capital Market ("NASDAQ") to list its common shares on the NASDAQ exchange, and expects to commence trading February 25, 2021 under the symbol "PRTG."

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2021 Research & Development Focus

Portage aims to catalyze research and development to produce a higher volume of quality clinical programs through its development strategy, commercial insights, and deep network of industry relationships. In 2021, the Company will focus on advancing three promising assets through clinical trials:

A Phase 1/2, open-label, dose-escalation and expansion study to evaluate safety and dosing of PORT-3, an invariant natural killer T-cell agonist (iNKT) co-formulated with an NY-ESO-1 vaccine in a nanoparticle, seeks to enroll patients with advanced prostate or ovarian tumors that express NY-ESO-1, a well-known cancer-testis antigen (CTA). The humoral and cellular immune responses along with the restricted expression of NY-ESO-1 make it a good target for cancer immunotherapies. The study is supported by a grant from the EU Horizon 2020 program. The trial has been approved by the regulatory agency and institutional ethics committee and is ready to start recruiting patients.
A Phase 1/2 trial investigating PORT-2, another iNKT agonist, in a liposomal formulation in patients with non-small-cell lung cancer and melanoma is expected to dose its first patients after COVID-19 restrictions in the United Kingdom (U.K.) ease. The trial is sponsored by Oxford University and has been approved by the University’s research ethics board as well as the Medicines and Healthcare products Regulatory Agency in the U.K.
An ongoing Phase 1 trial has transitioned to Phase 2 to investigate PORT-1 (INT230-6), an intratumoral amphiphilic agent, as a monotherapy and in combination with approved immunotherapies pembrolizumab, through a collaboration with Merck, and ipilimumab through a collaboration with Bristol Myers Squibb. The Phase 2 cohorts will focus on patients with difficult-to treat tumor types including breast, squamous cell, bile duct, pancreatic, colon, liver and sarcoma. Additional details are available on www.clinicaltrials.gov (NCT#03058289).
"Moving the first two programs from our iNKT platform into the clinic represents a huge milestone for our team," said Dr. Ian Walters, chief executive officer of Portage Biotech. "Many patients with difficult-to-treat tumors fail to respond to checkpoint inhibitors, but PORT-2 and PORT-3 may prime the immune system and enable a robust anti-cancer response, expanding potential therapeutics for this population."

Early-Stage Research

STING Agonist

The researchers and staff working on a proprietary immune priming and boosting technology using a STING agonist delivered in a virus-like particle have shown proof of concept in animal models and are beginning to progress the lead asset towards the clinic. This platform offers multiple ways to target immune stimulation towards cancer, as well as how to co-deliver multiple signals in a single product. Researchers have developed a way to administer the product systemically and not require direct tumor injections. The team has received grant funding to study this technology in combination with a COVID-19 vaccine to evaluate if it is possible to boost the immune response for immunocompromised or elderly patients.

Nanolipogel Coformulation

Portage is exploring the delivery of multiple signals to boost the immune response towards cancer in a single product. The Company has conducted further research with the technology licensed from Yale University to co-deliver a PD1 blocking signal with a small molecule vascular endothelial growth factor inhibitor. Other co-formulations are planned for this year. Preliminary data look promising, and the Company hopes to name its first clinical candidate this year.

Dr. Walters concluded, "We continue to evaluate and prioritize our early-stage portfolio and have initiated new collaborations with two leading artificial intelligence players to identify new assets that can be added to our portfolio and fast tracked to the clinic. We aim to bring one to two new entities to clinical testing each year."

On February 11, 2021 Eli Lilly and Company (NYSE:LLY) reported that it will participate in the 10th Annual SVB Leerink Global Healthcare Conference on Wednesday, February 24, 2021 (Press release, Eli Lilly, FEB 11, 2021, View Source [SID1234574926]). Ilya Yuffa, Lilly senior vice president and president of Lilly Bio-Medicines, will participate in a fireside chat at 1:40 p.m., Eastern Time.

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s investor website at View Source A replay of the fireside chat will be available for approximately 90 days.

On February 11, 2021 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing novel, targeted and personalized therapies for rare and difficult to treat cancers, reported fourth quarter and full year 2020 financial results on Monday, February 22, 2021, after market close (Press release, Cytori Therapeutics, FEB 11, 2021, View Source [SID1234574925]). Plus Therapeutics’ management team will then host a conference call and webcast at 5:00 p.m. Eastern Time to discuss the financial results and provide a corporate update.

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A live webcast will be available at ir.plustherapeutics.com/events

Please refer to the information below for conference call dial-in information. Callers should dial in approximately 10 minutes prior to the start of the call.

Conference Call Name: Plus Therapeutics Fourth Quarter and Full Year 2020 Financial Results Conference Call
Following the live call, a replay will be available on the Company’s website under the ‘Investor Relations’ section. The webcast will be available on the Company’s website for 90 days following the live call.

On February 11, 2021 Illumina, Inc. (NASDAQ: ILMN) reported its financial results for the fourth quarter and fiscal year 2020 (Press release, Illumina, FEB 11, 2021, View Source [SID1234574924]).

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Record fourth quarter revenue reflects a strong close to 2020:

Revenue of $953 million, an increase of 20% compared to the third quarter of 2020 and $0.5 million higher compared to the prior year period
GAAP net income attributable to Illumina stockholders for the quarter of $257 million, or $1.75 per diluted share, compared to $239 million, or $1.61 per diluted share, for the prior year period
Non-GAAP net income attributable to Illumina stockholders for the quarter of $179 million, or $1.22 per diluted share, compared to $252 million, or $1.70 per diluted share, for the prior year period. Non-GAAP net income excludes net gains from mark-to-market adjustments on our strategic investments, primarily from our marketable equity securities (see the "Reconciliation Between GAAP and Non-GAAP Net Income Attributable to Illumina Stockholders" table for a reconciliation of these GAAP and non-GAAP financial measures)
Cash flow from operations of $406 million compared to $443 million in the prior year period
Free cash flow (cash flow from operations less capital expenditures) of $344 million for the quarter compared to $386 million in the prior year period
"Illumina delivered a strong finish to 2020 with record fourth quarter revenue exceeding our expectations," said Francis deSouza, Chief Executive Officer. "We also had record orders in the quarter, including record sequencing instrument orders and the second highest quarter for NovaSeq instrument orders. Our business delivered strong sequential growth in the second half of 2020 and we expect continued recovery from the pandemic in 2021."

Gross margin in the fourth quarter of 2020 was 66.1% compared to 69.5% in the prior year period. Excluding amortization of acquired intangible assets and expenses related to COVID-19, non-GAAP gross margin was 66.9% for the fourth quarter of 2020 compared to 70.2% in the prior year period.

Research and development (R&D) expenses for the fourth quarter of 2020 were $200 million compared to $161 million in the prior year period. Non-GAAP R&D expenses as a percentage of revenue were 20.9% compared to 16.8% in the prior year period.

Selling, general and administrative (SG&A) expenses for the fourth quarter of 2020 were $298 million compared to $233 million in the prior year period. Excluding acquisition-related expenses, expenses related to COVID-19, and gain on litigation, non-GAAP SG&A expenses as a percentage of revenue were 25.1% compared to 22.2% in the prior year period.

Depreciation and amortization expenses were $50 million and capital expenditures for free cash flow purposes were $62 million during the fourth quarter of 2020. At the close of the quarter, the company held $3.5 billion in cash, cash equivalents and short-term investments, compared to $3.4 billion as of December 29, 2019.

Fiscal year 2020 results:

Revenue of $3,239 million, a 9% decrease compared to $3,543 million in fiscal 2019
GAAP net income attributable to Illumina stockholders of $656 million, or $4.45 per diluted share, compared to $1,002 million, or $6.74 per diluted share, in fiscal 2019
Non-GAAP net income attributable to Illumina stockholders of $664 million, or $4.50 per diluted share, compared to $976 million, or $6.57 per diluted share, in fiscal 2019 (see the "Itemized Reconciliation Between GAAP and Non-GAAP Net Income Attributable to Illumina Stockholders" table for a reconciliation of these GAAP and non-GAAP financial measures)
Cash flow from operations of $1,080 million compared to $1,051 million in fiscal 2019
Free cash flow (cash flow from operations less capital expenditures) of $891 million, compared to $842 million in fiscal 2019
Gross margin for fiscal 2020 was 68.0% compared to 69.6% in the prior year. Excluding amortization of acquired intangible assets, expenses and income related to COVID-19, and restructuring, non-GAAP gross margin was 69.0% for fiscal 2020 compared to 70.6% in the prior year period.

R&D expenses for fiscal 2020 were $682 million compared to $647 million in the prior year. Excluding expenses and income related to COVID-19, non-GAAP R&D expenses as a percentage of revenue were 21.0% compared to 18.2% in the prior year period.

SG&A expenses for fiscal 2020 were $941 million compared to $835 million in the prior year period. Excluding amortization of acquired intangible assets, acquisition-related expenses, expenses and income related to COVID-19, gain on litigation, and restructuring, non-GAAP SG&A expenses as a percentage of revenue were 24.6% compared to 22.0% in the prior year period.

Updates since our last earnings release:

Expanded NextSeq 2000 flow cell offering with launch of the P3 Reagent Kit and started shipping the NextSeq 1000
Announced TSO 500 partnerships with Bristol Myers Squibb, Kura Oncology, Myriad Genetics, and Merck to advance comprehensive genomic profiling
Launched Illumina Connected Analytics enabling customers to manage, analyze, and explore large volumes of multi-omic data in a secure, scalable, and flexible environment
Partnered with Harvard Pilgrim Health Care to expand access to whole-genome sequencing for genetic disease testing
Won patent infringement suit against BGI in the United Kingdom
Repurchased approximately $280 million of common stock in the fourth quarter with $15 million remaining for repurchase under our current plan
Announced a collaboration with Sequoia Capital China to catalyze the genomic startup ecosystem in China
Financial outlook and guidance

The non-GAAP financial guidance discussed below reflects certain pro forma adjustments to assist in analyzing and assessing our core operational performance. Please see our Reconciliation of Non-GAAP Financial Guidance included in this release for a reconciliation of the GAAP and non-GAAP financial measures.

For fiscal 2021, the company expects year-over-year revenue growth in the range of 17% to 20%, and expects GAAP earnings per diluted share of $4.76 to $5.01 and non-GAAP earnings per diluted share of $5.10 to $5.35. This guidance excludes the potential impact from the pending acquisition of GRAIL, which we expect to close in the second half of 2021.

Quarterly conference call information

The conference call will begin at 2:00 pm Pacific Time (5:00 pm Eastern Time) on Thursday, February 11, 2021. Interested parties may access the live teleconference through the Investor Info section of Illumina’s website under the "Company" tab at www.illumina.com. Alternatively, individuals can access the call by dialing 1 (866) 211-4597 or 1 (647) 689-6853 outside North America, both with conference ID 3809129.

A replay of the conference call will be posted on Illumina’s website after the event and will be available for at least 30 days following.

On February 11, 2021 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 its financial results for the quarter and full year ended December 31, 2020 and reaffirmed its financial guidance for 2021 (Press release, Ultragenyx Pharmaceutical, FEB 11, 2021, View Source [SID1234574923]).

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"Last year was a transformative year as we received two approvals, generated clinically meaningful data in our gene therapy and antisense oligonucleotide programs, and completed several strategic business development transactions," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In addition to growing our global commercial footprint in 2021, we will also have a strong, diverse, late-stage clinical development portfolio. We will initiate three pivotal gene therapy studies, a fourth pivotal study with our recently in-licensed biologic, setrusumab, for osteogenesis imperfecta, make additional progress in the Angelman syndrome study, and bring our first mRNA program to the clinic for glycogen storage disease type III."

Fourth Quarter and Full Year 2020 Financial Results

Net Revenues
For the fourth quarter of 2020, Ultragenyx reported $91.5 million in total revenue. Ultragenyx recognized $40.4 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $37.5 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $2.9 million. Total royalty revenue related to European Crysvita sales were $3.6 million. Dojolvi (triheptanoin) product sales in the fourth quarter of 2020 were $6.4 million. Mepsevii (vestronidase alfa) product sales for the fourth quarter of 2020 were $3.7 million. Total revenue for the fourth quarter also includes $37.5 million of revenue related to the collaboration and license agreement with Daiichi Sankyo that was executed in March 2020.

Revenue for the year ended December 31, 2020 was $271.0 million, including $138.9 million in Crysvita revenue in the Ultragenyx territories. Crysvita collaboration revenue in the North American profit share territory was $128.6 million and net Crysvita product sales in other regions were $10.4 million. Total royalty revenue related to European Crysvita royalties was $14.5 million, which includes $1.5 million recognized on sales that occurred prior to January 1, 2020. Dojolvi product revenue for the year ended December 31, 2020 was $13.0 million. Mepsevii product revenue was $15.3 million. For the year ended December 31, 2020, revenue related to our strategic manufacturing partnership with Daiichi Sankyo was $89.2 million.

Operating Expenses
Total operating expenses for the fourth quarter of 2020 were $187.6 million, including non-cash stock-based compensation of $22.8 million. This compares to total operating expenses for the fourth quarter of 2019, which were $130.0 million and total non-cash stock-based compensation of $19.7 million.

Total operating expenses for the year ended December 31, 2020 were $601.1 million, which includes $25.0 million to maintain the option to acquire GeneTx, $13.2 million from a one-time in-process R&D expense related to the strategic collaboration with Solid Biosciences, $7.0 million to license certain vectors from REGENXBIO, and non-cash stock-based compensation of $85.7 million. This is compared with $527.9 million for the same period in 2019, which includes a $15.6 million research and development expense from the Arcturus collaboration amendment, $20.0 million for the upfront payment on the GeneTx agreement, and non-cash stock-based compensation of $82.0 million. The increase in total operating expenses was due to the increase in commercial, on-going clinical and preclinical development, and general and administrative costs as the company commercializes, grows, and advances its portfolio.

For the fourth quarter of 2020, Ultragenyx reported net loss of $24.0 million, or $0.37 per share basic and diluted, compared with a net loss for the fourth quarter of 2019 of $93.8 million, or $1.62 per share, basic and diluted. For the year ended December 31, 2020, net loss was $186.6 million, or $3.07 per share, basic and diluted, compared with a net loss for the same period in 2019 of $402.7 million, or $7.12 per share, basic and diluted. The net loss for the fourth quarter of 2020 and the net loss for the year ended December 31, 2020 includes a $79.1 million and $170.4 million increase in the fair value of investments in equity securities, respectively. Net cash used in operations for the year ended December 31, 2020 was $132.2 million, compared to net cash used of $345.4 million for the same period in 2019.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were $1.2 billion as of December 31, 2020.

2021 Financial Guidance

Crysvita Guidance in Ultragenyx Territories
The company reaffirms the 2021 guidance range for Crysvita that was provided at the beginning of the year. This range is $180 million to $190 million for the North American profit share region and the other regions where product sales are recognized (Latin America and Turkey).

Program Updates and Upcoming Milestones

Dojolvi for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): Launched on July 22, 2020

Five months into the United States launch of Dojolvi for LC-FAOD, the company has received approximately 190 completed start forms from approximately 90 unique prescribers. This has led to approximately 130 patients on reimbursed therapy as of the end of December, a 117% increase over the prior quarter.
In Canada, Dojolvi has been granted priority review by Health Canada and a new drug submission was filed in August 2020, with a decision expected soon. Dojolvi has been submitted to the Brazilian Health Regulatory Agency (ANVISA) seeking marketing authorization. Discussions with the European Medicines Agency (EMA) are ongoing. Responses to named patient treatment requests continue in the EU.
Three global, pivotal stage gene therapy clinical studies are expected in 2021

DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Following Scientific Advice with the EMA and an End of Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA), the company currently expects to initiate a Phase 3 study in the first half of 2021.
DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Based on initial Scientific Advice from the EMA and following a planned EOP2 meeting with the FDA, the company currently expects to initiate a Phase 3 study in the second half of 2021.
UX701 for Wilson disease: The Investigational New Drug (IND) application has cleared and the program recently received Fast Track Designation from the FDA. The company currently expects to initiate a seamless, single-protocol Phase 1/2/3 study in the first half of 2021.
GTX-102 for Angelman Syndrome, partnered with GeneTx Biotherapeutics: Study expected to resume in first half 2021 with additional data anticipated in second half of 2021

GeneTx, with Ultragenyx support, submitted to the FDA a substantial information amendment to the IND including follow-up safety information for the five patients dosed and toxicology data in nonhuman primates that demonstrate no evidence of this safety issue at higher repeat dosing. GeneTx has received feedback and questions from the FDA based on this submission and GeneTx and Ultragenyx have filed a response to FDA and are working on an amendment to the protocol.
A Clinical Trial Application (CTA) has been submitted in Canada and a protocol and information amendment similar to what has been proposed to FDA will be submitted. GeneTx, with Ultragenyx support, is in the process of expanding the study to other countries using the amended dosing and administration plan.
The companies currently expect the Phase 1/2 study to resume enrollment and dosing in the first half of 2021, following resolution of FDA requests and approval to proceed.

UX143 (setrusumab) for Osteogenesis Imperfecta, in collaboration with Mereo BioPharma: Phase 2/3 in pediatric patients anticipated in second half of 2021

The companies expect to conduct a Phase 2/3 study in pediatric patients that first focuses on determining the optimal dose based on increases in bone production via changes in serum P1NP levels over two months and an acceptable safety profile. Following determination of the dose, the study is intended to transition into a pivotal Phase 3 study, evaluating fracture reduction over an estimated 15 to 24 months. Final study design and endpoints are pending regulatory review.
UX053 for Glycogen Storage Disease Type III or Debrancher deficiency: IND on track in the first half of 2021

An IND for the company’s first mRNA program, UX053, for the treatment of GSDIII debrancher deficiency is on track for the first half of 2021, with a Phase 1/2 study expected to initiate in the second half of 2021.
1: Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, February 11, 2021, at 2 p.m. PT/ 5 p.m. ET to discuss the fourth quarter and year 2020 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 6391931. The replay of the call will be available for one year.