On February 24, 2021 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company developing drugs to treat cancers with the greatest medical need for new treatment options, including KRAS-mutated colorectal cancer, pancreatic cancer, castrate-resistant prostate cancer and leukemias, reported that Dr. Mark Erlander, chief executive officer of Cardiff Oncology will participate in the upcoming Cowen 41st Annual Health Care Conference taking place March 1-4, 2021 (Press release, Cardiff Oncology, FEB 24, 2021, View Source [SID1234575532]). Dr. Erlander will participate in a GI Oncology and Pancreatic Cancer panel discussion and one-on-one investor meetings at the conference.

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Details on the panel discussion can be found below.
Panel Discussion: GI Oncology and Pancreatic Cancer Date and Time:
Tuesday, March 2nd; 9:50 – 10:50 am ET
Webcast: Available for live viewing by conference attendees only

On February 24, 2021 Crown Bioscience (CrownBio), JSR Life Sciences and Cambridge Quantum Computing (CQC) reported a partnership agreement to explore the application of quantum technology to drive the identification of multi-gene biomarker discovery for oncology drug discovery (Press release, Crown Bioscience, FEB 24, 2021, View Source [SID1234575531]).

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The partnership will combine CrownBio’s domain expertise and vast data sets generated from 15 years of preclinical and translational research and CQC’s advanced capabilities in quantum algorithms, quantum machine learning, and quantum computing.

Utilizing quantum machine algorithms and CQC’s software development framework for execution on NISQ (Noisy Intermediate-Scale Quantum) computers, the initial approach will focus on deriving insight from the analysis of genetic data to identify cancer treatment biomarkers and drive the next generation of bioinformatics.

The objective is to identify a strategy to implement an early quantum computing application that will ultimately address and explore solutions to broad challenges in life sciences.

Armin Spura, CEO of CrownBio commented, "The agreement continues CrownBio’s commitment to innovation and the application of technology to accelerate and de-risk drug development, leading to stronger drug candidates and a more rapid transition from preclinical phases to the clinic."

llyas Khan, CEO of CQC said, "CQC was founded to develop technologies that could help address some of the most pressing societal challenges, particularly in areas such as human biology, so we are excited to enter into our latest partnership with CrownBio to determine how cutting-edge quantum machine learning methods can be applied to critical use cases."

On February 24, 2021 AIM ImmunoTech Inc. (NYSE American: AIM) reported that its subsidiary, NV Hemispherx Biopharma Europe, reported that it has received formal notification from the European Commission (EC) approving the company’s Orphan Medicinal Product Application for Ampligen as a treatment for pancreatic cancer (Press release, AIM ImmunoTech, FEB 24, 2021, View Source [SID1234575530]).

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Medications that have been designated as Orphan products by the European Medicines Agency (EMA), once commercially approved in the European Union (EU), receive benefits including up to ten years of protection from market competition from similar medicines with similar active component and indication for use that are not shown to be clinically superior.

AIM announced earlier this month that the Committee for Orphan Medicinal Products of the EMA had recommended that Ampligen receive the designation for pancreatic cancer. The company has now received the official approval of that designation.

Prof. Casper van Eijck, M.D. Ph.D., the lead investigator for the expanded access program (EAP) for Ampligen at Erasmus Medical Center, stated, "We are pleased to report that the EMA has approved Ampligen for orphan medicinal product designation. Pancreatic cancer is the seventh leading cause of cancer-related deaths worldwide with over 458,000 worldwide cases of pancreatic cancer in 2018 alone. Despite advancements in the detection and management of pancreatic cancer, the 5-year survival rate is only 5-10%. Due to the positive and statistically significant survival results, versus historical controls, that we observed when using Ampligen in patients with locally advanced/metastatic pancreatic cancer-after systemic chemotherapy-we believe that Ampligen has the potential to be a meaningful extension of the standard of care for advanced pancreatic cancer. We are currently writing the manuscript describing the results of our study and are planning to investigate Ampligen further in the follow-up pancreatic cancer Phase 2/3 clinical trial and in a Phase 1 trial in combination with check point inhibitors at Erasmus Medical Center."

Kazem Kazempour, Ph.D., at Amarex Clinical Research, commented, "We are working closely with AIM in the United States to attain FDA ‘fast-track’ status, as well as possible FDA ‘breakthrough’ designations, and to obtain IND authorizations to conduct a follow-up pancreatic cancer Phase 2/3 clinical trial with sites in the Netherlands at Erasmus MC under Prof. van Eijck, and also at major cancer research centers in the United States."

"Orphan medicinal product designation in the EU and in the US is a critical milestone in AIM’s ongoing efforts to develop Ampligen as a treatment for pancreatic cancer," said AIM CEO Thomas K. Equels. "Pancreatic cancer is one of the deadliest cancers because, far too often, it is not diagnosed until Stage IV, when the disease is so far along that there are limited therapeutic options. We at AIM hope that Ampligen can one day help add precious time to the lives of many people suffering from pancreatic cancer."

Orphan medicinal product designations promote the clinical development of drugs that target rare life-threatening conditions, and which are expected to provide significant therapeutic advantage over existing treatments. An estimated 466,000 people died of pancreatic cancer worldwide in 2020, according to the World Health Organization. The five-year survival rate is only 5-10 percent.

Sponsors who obtain EMA orphan designation "benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market," according to the EMA. Fee reductions are also available depending on circumstances. The EMA has a comprehensive explanation of the benefits of Orphan Drug Designation (ODD) on its website.

The EMA designation follows a similar approval from the U.S. Food and Drug Administration (FDA), which also awarded AIM with orphan drug designation status for Ampligen as a treatment for pancreatic cancer.

The FDA orphan designation followed the company’s September 22, 2020 announcement of statistically significant positive pancreatic cancer survival benefit in the Ampligen arm as compared to a historical control cohort seen in a multi-year Early Access Program conducted at Erasmus University Medical Center in the Netherlands. The use of Ampligen following the current standard of care for pancreatic cancer (FOLFIRINOX) yielded an overall survival of 19 months, 7.9 months greater than FOLFIRINOX treatment alone.

On February 24, 2021 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the fourth quarter of 2020 and recent business highlights (Press release, Ionis Pharmaceuticals, FEB 24, 2021, View Source [SID1234575529]).

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"Last year, we laid out a bold new vision for the Company and took important steps towards our goal of becoming one of the most successful biotechnology companies. Key to our vision is our strategy to maximize the value of our pipeline by commercializing our wholly owned medicines. Our acquisition of Akcea was an important step in building our commercial capabilities while enabling us to further strengthen our organization," said Brett P. Monia, Ph.D., chief executive officer at Ionis. "Last year, we also advanced our late-stage pipeline and expanded the utility of our technology. Looking ahead, we expect data from multiple wholly owned programs in the first half of this year, followed by Phase 3 tofersen data in patients with SOD1-ALS in the second half. These key upcoming catalysts, together with our recent pipeline and technology achievements, position us well to have 12 or more products on the market in 2026. Importantly, we continue to have the financial strength to expand investment in our wholly owned pipeline and commercial capabilities to drive meaningful and increasing value for patients and shareholders."

2020 Summary Financial Results

Achieved 2020 financial guidance
$729 million in total revenues, with half from marketed products
$640 million of operating expenses on a non-GAAP basis(1) and $901 million on a GAAP basis, reflecting investments in Ionis’ wholly owned pipeline
Net income of $111 million on a non-GAAP basis(1) and a net loss of $451 million on a GAAP basis
Strong balance sheet with cash of $1.9 billion at year-end, enabling increasing investment in advancing the pipeline and technology while also preparing to commercialize the Company’s wholly owned medicines
2020 Marketed Products Highlights

SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages
$2 billion in worldwide sales in 2020
More than 11,000 patients worldwide were on therapy at the end of the fourth quarter across post-marketing, expanded access and clinical trial settings
Enrollment began in the RESPOND study evaluating potential SPINRAZA benefit in SMA patients with a suboptimal clinical response to gene therapy
Enrollment began in the pivotal randomized treatment cohort of the DEVOTE study evaluating higher doses of SPINRAZA
TEGSEDI and WAYLIVRA: transformational medicines approved for the treatment of patients with severe rare diseases
Product sales increased more than 65 percent in 2020, compared to 2019
Generated growing revenues as major markets launched in 2020
Restructured European operations through a distribution agreement with Swedish Orphan Biovitrum AB (Sobi)
Q4 2020 and Recent Pipeline Highlights

Phase 3 Pipeline: growing pipeline positioned for 12 or more products on the market in 2026
Advanced IONIS-APOCIII-LRx into the Phase 3 BALANCE study in patients with FCS
Completed enrollment in the tofersen Phase 3 VALOR study in patients with SOD1-ALS
Mid-stage Pipeline: broad and advancing pipeline of potential first-in-class and/or best-in-class medicines
Advanced and expanded the IONIS-AGT-LRx development program
Reported IONIS-AGT-LRx positive topline Phase 2 results in patients with hypertension uncontrolled with two or three antihypertensive medications
Advanced ION904, the follow-on medicine targeting AGT, into Phase 1 development in healthy volunteers
Advanced vupanorsen into Phase 2b development with the initiation of the TRANSLATE-TIMI 70 dose-ranging study in statin-treated patients with dyslipidemia, resulting in a $75 million payment from Pfizer
Advanced ION449 (AZD8233), targeting PCSK9, into Phase 2b development in patients with dyslipidemia and AstraZeneca licensed ION455, a new investigational medicine for the treatment of nonalcoholic steatohepatitis (NASH), resulting in $50 million from AstraZeneca
Unlocked potential new pulmonary disease franchise with positive IONIS-ENAC-2.5Rx data
Reported positive healthy volunteer results supporting aerosol antisense medicine delivery to the lung
Completed dosing in the Phase 2 study in patients with cystic fibrosis
Advanced IONIS-ENAC-2.5Rx into Phase 2 development in patients with chronic obstructive pulmonary disease (COPD)
Highlighted IONIS-MAPTRx (BIIB080) Phase 1/2 study in patients with Alzheimer’s disease in which IONIS-MAPTRx was generally well tolerated and demonstrated durable, time and dose-dependent reductions in CSF tau protein
Upcoming 2021 Pipeline Catalysts

2020 Financial Results and 2021 Financial Guidance

"We achieved our 2020 financial guidance, even in the challenging COVID-19 pandemic environment. Moreover, in 2020 we made significant progress toward our goal of creating a stronger, more efficient company focused primarily on advancing our wholly owned medicines to the market. We acquired Akcea enabling us to retain full value from its rich portfolio. We also restructured our European operations. Together, these transactions unlocked substantial cost savings that we plan to reinvest to drive future revenue growth," said Elizabeth L. Hougen, chief financial officer of Ionis. "Our 2021 guidance reflects our new strategy to maximize the value of our wholly owned pipeline, focused primarily on commercializing our rare neurological and cardiometabolic disease programs. Our guidance also reflects the investments we are making in three key areas of our business – advancing and expanding our wholly owned pipeline, building commercial capabilities in support of our rich pipeline and broadening the reach of our technology. We can increase our investments in these areas while only modestly increasing our expenses because of the significant cost savings we realized from acquiring Akcea and restructuring our European operations. Importantly, with nearly $2 billion of cash at the end of last year, we remain well capitalized with the substantial financial resources to achieve our goals."

2021 Financial Guidance

Ionis’ full year 2021 financial guidance consists of the following components (on a non-GAAP basis)(1):

All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards and expenses related to the Akcea acquisition and restructured European operations and the related tax effects. Please refer to the section below titled "Financial Impacts of Akcea Acquisition and Restructured European Operations" for a breakdown of the costs specific to these transactions. Additionally, please refer to the detailed reconciliation of non-GAAP and GAAP measures, which is provided later in this release.

Revenue

Financial Impacts of Akcea Acquisition and Restructured European Operations

In the fourth quarter of 2020, the Company’s non-GAAP amounts exclude the following expenses related to the Akcea acquisition and restructured European operations because the costs are not part of its normal ongoing operating activities. Refer to the detailed reconciliation of non-GAAP and GAAP measures, which is provided later in this release. (Amounts in millions):

As a result of the Akcea acquisition, Ionis and Akcea began reporting their federal taxes on a consolidated basis in the fourth quarter of 2020. The Company recorded a valuation allowance against all Ionis’ federal net deferred tax assets in the fourth quarter of 2020, due largely to Akcea’s history of losses and the expected impact of this on Ionis’ consolidated federal taxable income. The Company now maintains a valuation allowance against all its consolidated federal and state net deferred tax assets.

Operating Expenses

Ionis’ operating expenses for the year ended December 31, 2020 increased compared to 2019 driven by the Company’s investments in advancing the Phase 3 programs for IONIS-TTR-LRx, IONIS-APOCIII-LRx and other medicines in its wholly owned pipeline. Additionally, the Company incurred approximately $90 million in costs related to the Akcea acquisition and restructured European operations on a GAAP basis. The costs consisted of $31 million of severance, retention and other costs and $59 million of non-cash stock-based compensation expense for the acceleration of Akcea equity awards.

Net Loss Attributable to Noncontrolling Interest in Akcea

Prior to completing its acquisition of Akcea in October 2020, Ionis owned approximately 76 percent of Akcea. The line titled "Net loss attributable to noncontrolling interest in Akcea" on Ionis’ statement of operations reflects the portion of Akcea’s net income or loss attributable to the other owners of Akcea’s common stock. From mid-October 2020 through December 31, 2020, Ionis did not recognize any noncontrolling interest in Akcea on its statement of operations because it owned 100 percent of Akcea. Beginning in 2021, the Company will no longer have an adjustment for noncontrolling interest in Akcea.

Net Income (Loss) Attributable to Ionis Common Stockholders

Ionis recognized a net loss attributable to Ionis’ common stockholders for 2020 compared to net income in 2019 primarily due to higher revenue in 2019, including approximately $400 million in license fees Ionis earned from Pfizer and Novartis. Also contributing to Ionis’ net loss in 2020 was the non-cash adjustment of the valuation allowance Ionis recorded against its federal net deferred tax assets discussed above. Additionally, Ionis’ operating expenses increased in 2020 compared to the same period last year as described above.

Balance Sheet

Ionis ended 2020 with cash, cash equivalents and short-term investments of $1.9 billion, compared to $2.5 billion at December 31, 2019. In October 2020, Ionis used approximately $545 million of its cash for the Akcea acquisition.

Webcast

Today, at 9:00 a.m. Eastern Time, Ionis will conduct a live webcast to discuss this earnings release and related activities. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address.

On February 24, 2021 Vericel Corporation (NASDAQ:VCEL), a leader in advanced therapies for the sports medicine and severe burn care markets, reported financial results and business highlights for the fourth quarter and year ended December 31, 2020, and provided full-year 2021 financial guidance (Press release, Vericel, FEB 24, 2021, View Source [SID1234575528]).

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Fourth Quarter 2020 Financial Highlights

Total net revenue increased 15% to $45.2 million, compared to $39.4 million in the fourth quarter of 2019
MACI net revenue of $34.7 million, Epicel net revenue of $9.6 million and NexoBrid revenue of $1.0 million related to the U.S. Biomedical Advanced Research and Development Authority (BARDA) procurement for national response preparedness
Gross margin of 74%, compared to gross margin of 73% in the fourth quarter of 2019
Net income of $12.2 million, or $0.25 per share, compared to $9.5 million, or $0.20 per share, in the fourth quarter of 2019
Non-GAAP adjusted EBITDA of $16.0 million, or 35% of net revenue, compared to $12.8 million, or 33% of net revenue, in the fourth quarter of 2019
Operating cash flow of $11.3 million
Full-Year 2020 Financial Highlights

Total net revenue increased 5% to $124.2 million, compared to $117.9 million in 2019
MACI net revenue of $94.4 million, Epicel net revenue of $27.5 million and NexoBrid revenue of $2.2 million related to the BARDA procurement for national response preparedness
Gross margin of 68%, compared to gross margin of 68% in 2019
Net income of $2.9 million, or $0.06 per share, compared to a net loss of $9.7 million, or $0.22 per share, in 2019
Non-GAAP adjusted EBITDA of $18.6 million, or 15% of net revenue, compared to $21.2 million, or 18% of net revenue, in 2019
Operating cash flow of $17.6 million
As of December 31, 2020, the company had $100 million in cash and investments, compared to $79 million as of December 31, 2019, and no debt
Business Highlights and Updates

Record fourth-quarter and full-year total net revenue
Record quarterly gross margin, net income and operating cash flow
Record quarterly and full-year MACI implants and net revenue
Record fourth-quarter and full-year Epicel grafts and net revenue, and the second highest quarterly Epicel grafts and revenue in history
Received MACI biopsies from approximately 1,500 surgeons in 2020, an increase from approximately 1,400 surgeons in 2019
Record quarterly high in the number of surgeons taking MACI biopsies in the fourth quarter
Double-digit growth in MACI biopsies in the fourth quarter, achieving a record quarterly high and a record monthly high in December
Announced expansion of MACI coverage by UnitedHealthcare to include patella and multiple cartilage defects in the knee
Appointed Joe Mara as Chief Financial Officer
"We delivered a record fourth quarter across multiple financial and operational metrics and ended the year in a very strong financial position," said Nick Colangelo, President and CEO of Vericel. "With revenue growth for both products in 2020, we demonstrated the resiliency of the Company’s growth profile and are on track for significant growth in the years ahead. Our guidance for 2021 reflects a return to MACI’s pre-COVID growth trajectory, continued momentum for Epicel, and significant adjusted EBITDA growth."

2021 Financial Guidance

Total net revenue for 2021 expected to grow 30%-32% to approximately $161 million to $164 million
Gross margin expected to be 70% to 71%
Adjusted EBITDA margin expected to be 21% to 23%
Fourth Quarter 2020 Results
Total net revenue for the quarter ended December 31, 2020 increased 15% to $45.2 million, compared to $39.4 million in the fourth quarter of 2019. Total net product revenue for the quarter included $34.7 million of MACI (autologous cultured chondrocytes on porcine collagen membrane) net revenue and $9.6 million of Epicel (cultured epidermal autografts) net revenue compared to $33.6 million of MACI net revenue and $5.8 million of Epicel net revenue, respectively, in the fourth quarter of 2019. Total net revenue for the quarter also included $1.0 million of revenue related to the procurement of NexoBrid (concentrate of proteolytic enzymes enriched in bromelain) by BARDA for emergency response preparedness.

Gross profit for the quarter ended December 31, 2020 was $33.6 million, or 74% of net revenue, compared to $28.8 million, or 73% of net revenue, for the fourth quarter of 2019.

Total operating expenses for the quarter ended December 31, 2020 were $21.4 million, compared to $19.6 million for the same period in 2019. The increase in operating expenses was primarily due to incremental employee expenses related to the MACI sales force expansion.

Net income for the quarter ended December 31, 2020 was $12.2 million, or $0.25 per share, compared to $9.5 million, or $0.20 per share, for the fourth quarter of 2019.

Non-GAAP adjusted EBITDA for the quarter ended December 31, 2020 was $16.0 million, or 35% of net revenue, compared to $12.8 million, or 33% of net revenue, in the fourth quarter of 2019. A table reconciling non-GAAP measures is included in this press release for reference.

Full-Year 2020 Results
Total net revenue for the year ended December 31, 2020 increased 5% to $124.2 million, compared to $117.9 million in 2019. Total net product revenue for the year included $94.4 million of MACI net revenue and $27.5 million of Epicel net revenue compared to $91.6 million of MACI net revenue and $26.2 million of Epicel net revenue, respectively, in 2019. Total net revenue in 2020 also included $2.2 million of revenue related to the procurement of NexoBrid by BARDA for emergency response preparedness.

Gross profit for the year ended December 31, 2020 was $84.2 million, or 68% of net revenue, compared to $80.3 million, or 68% of net revenue, in 2019.

Total operating expenses for the year ended December 31, 2020 were $81.9 million, compared to $91.5 million for the same period in 2019. Operating expenses in 2019 included the $17.5 million upfront license payment to MediWound Ltd. for North American rights to NexoBrid.

Net income for the year ended December 31, 2020 was $2.9 million, or $0.06 per share, compared to a net loss of $9.7 million, or $0.22 per share, in 2019.

Non-GAAP adjusted EBITDA for the year ended December 31, 2020 was $18.6 million, or 15% of net revenue, compared to $21.2 million, or 18% of net revenue, in 2019. A table reconciling non-GAAP measures is included in this press release for reference.

As of December 31, 2020, the company had $100 million in cash and investments, compared to $79 million as of December 31, 2019, and no debt.

Conference Call Information
Today’s conference call will be available live at 8:30am Eastern Time and can be accessed through the Investor Relations section of the Vericel website at View Source." target="_blank" title="View Source." rel="nofollow">View Source A slide presentation with highlights from today’s conference call will be available on the webcast and in the Investor Relations section of the Vericel website. Please access the site at least 15 minutes prior to the scheduled start time in order to download the required audio software, if necessary. To participate in the live call by telephone, please call (877) 312-5881 and reference Vericel Corporation’s third-quarter 2020 investor conference call. If calling from outside the U.S., please use the international phone number (253) 237-1173.

If you are unable to participate in the live call, the webcast will be available at View Source until February 24, 2022. A replay of the call will also be available until 11:30am (EDT) on March 3, 2021 by calling (855) 859-2056, or from outside the U.S. by calling (404) 537-3406. The conference ID is 4364298.