On February 25, 2021 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported financial results for the fourth quarter and year ended December 31, 2020, and provided a business update (Press release, Adaptimmune, FEB 25, 2021, View Source [SID1234575726]).

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"We are building the cell therapy company of the future for people with cancer. With our ‘2-2-5-2’ by 2025 strategic plan, we will deliver value with marketed SPEAR T-cell products starting with ADP-A2M4 for people with synovial sarcoma," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "We completed enrollment in our SPEARHEAD-1 trial in approximately 12 months to support our first BLA, which is strong evidence of our ability to execute rapidly. We are focusing the SURPASS trial, using our next-generation ADP-A2M4CD8 product, on lung, gastroesophageal, head and neck, and bladder cancers to identify new indications to take forward to late-stage development."

Planned 2021 data updates1

SPEARHEAD-1 trial with ADP-A2M4 for people with synovial sarcoma
June: preliminary data at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)
November: full update at Connective Tissue Oncology Society (CTOS)
SURPASS Phase 1 trial with ADP-A2M4CD8 (next-generation product targeting MAGE-A4)
September: update at European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper)
Additional clinical updates
September: update at International Liver Cancer Association (ILCA) conference for ADP-A2AFP Phase 1 trial for people with liver cancer
October: update at American Society for Radiation Oncology (ASTRO) for radiation sub-study of the ADP-A2M4 Phase 1 trial2
November: ADP-A2M4 translational data update at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)
"2-2-5-2" by 2025 strategic plan
At an Investor Day held in November 2020, the Company outlined its "2-2-5-2" by 2025 strategic plan encompassing:

"2" – Two marketed products targeting MAGE-A4
Estimated potential addressable population in tumor types with significant MAGE-A4 expression, factored for HLA-A23, is ~39,000 patients per year in the US and EU

Durable responses in synovial sarcoma – on track to file a Biologics License Application (BLA) for ADP-A2M4 in 2022

The first commercial opportunity for SPEAR T-cells targeting MAGE-A4 will be in synovial sarcoma with plans to file a BLA in the US in 2022
In 2020, the Company received positive endorsements from regulatory authorities with Regenerative Medicine Advanced Therapy (RMAT) designation and Orphan Drug Designation (ODD) in the US, and access to PRIority MEdicines (PRIME) regulatory support and ODD in the EU
Data from the Phase 1 trial with ADP-A2M4 (presented at CTOS 2020)
In 16 patients with synovial sarcoma, there was an Overall Response Rate of 44% and a Disease Control Rate of 94%
Responses were durable with a median duration of response of 28 weeks with ongoing responses beyond 72 weeks in two patients (median overall survival had not been reached)
These data are considerably superior4 to response rates observed with available second line therapies in synovial sarcoma
SURPASS-2 in esophageal and esophagogastric junction (EGJ) cancers in 1H 2021

The Company will initiate a Phase 2 trial, SURPASS-2, with ADP-A2M4CD8 (next-generation SPEAR T-cells targeting MAGE-A4 that co-express CD8α intended to increase potency) for patients with esophageal or EGJ cancers in 1H 2021
SURPASS-2 is supported by encouraging data from the Phase 1 SURPASS trial (presented at SITC (Free SITC Whitepaper) 2020) with one confirmed partial response (PR) in a patient with EGJ cancer and tumor reductions in two additional patients (1 with esophageal and 1 with EGJ cancer)
The trial will be conducted at multiple centers in North America and the EU, and is intended to enroll 45 people with esophageal or EGJ cancers to be treated with doses up to 10 billion SPEAR T-cells
The Company also presented preclinical data at SITC (Free SITC Whitepaper) 2020 indicating that AKT inhibition during the manufacture of SPEAR T-cells results in a more consistent expansion and phenotype of the final product. This process is currently being used for the Phase 1 SURPASS trial.
"2" – Two additional BLAs for SPEAR T-cell products

Adaptimmune’s Phase 1 SURPASS trial with ADP-A2M4CD8 continues to enroll patients, focusing on lung, gastroesophageal, head and neck, and bladder cancers – indications for which the Company has reported responses or signs of efficacy with its MAGE-A4 targeted products
In 2020, Adaptimmune initiated SPEARHEAD-2 with its first-generation SPEAR T-cells targeting MAGE-A4 in combination with pembrolizumab for people with head and neck cancers
The Phase 1 trial with ADP-A2AFP for people with liver cancer is ongoing. As presented at ILC 2020, nine patients were treated as of the data cut-off and best responses were:
One patient with a complete response, one with stable disease (SD), and two with progressive disease (PD) among the four patients who received ~5 billion or more SPEAR T-cells
Five patients with SD who received doses of 100 million and 1 billion SPEAR T-cells in the first two dose cohorts
"5" – Five new autologous products in the clinic

Adaptimmune has a deep preclinical pipeline from which it expects to bring five new products into the clinic.
These include multiple possibilities for next-generation autologous SPEAR T-cells such as:
ADP-A2AFP SPEAR T-cells co-expressing CD8α
ADP-A2M4 SPEAR T-cells co-expressing IL-7, IL-15, dnTGFβ, and/ or PDE7
Enhancing SPEAR T-cells with IL-7 for proliferation and survival and CCL19 for migration into tumor in collaboration with Noile-Immune Biotech, Inc.
Enhancing SPEAR T-cells using transmembrane and surface immunoregulatory mechanisms with Alpine Immune Sciences, Inc.
The Company is also developing new products, including:
Expanding into HLAs beyond HLA-A2 to increase the addressable patient population
HLA-independent TCR (HiT) candidates for multiple targets including GPC3
A new program for next-generation TILs co-expressing IL-7 in melanoma in collaboration with leading TIL therapy center (CCIT, Denmark)
"2" – Two allogeneic products in the clinic

In January 2020, Adaptimmune announced it had entered into an agreement to co-develop and co-commercialize stem-cell derived allogeneic cell therapies with Astellas
The first target nominated is a HiT targeting mesothelin
At ASGCT (Free ASGCT Whitepaper) 2020, Adaptimmune presented data with evidence of its allogeneic platform demonstrating differentiation of functional T-cells from human-induced pluripotent stem cells (hiPSCs) that can kill MAGE-A4 expressing target cells in vitro –targeted to become the Company’s first allogeneic product in the clinic
Corporate updates from 2020

Despite the impact of the COVID-19 pandemic on the biotechnology industry, Adaptimmune continued to see improved enrollment in its clinical trials
In Q1, the Company received an upfront payment of $50 million from Astellas. The Company is also entitled to receive research funding of up to $7.5 million per collaboration target per year
Underwritten public offering in Q1 generated net proceeds of approximately $90 million
Underwritten public offering in Q2 generated net proceeds of approximately $244 million
Financial Results for the fourth quarter and year ended December 31, 2020

Cash / liquidity position: As of December 31, 2020, Adaptimmune had cash and cash equivalents of $56.9 million and Total Liquidity5 of $368.2 million.

Revenue: Revenue for the fourth quarter and year ended December 31, 2020 was $1.5 million and $4.0 million, respectively, compared to $0.7 million and $1.1 million for the same periods in 2019. The increase was due to revenue arising under the collaboration agreement with Astellas, which was entered into in January 2020.

Research and development (R&D) expenses: R&D expenses for the fourth quarter and year ended December 31, 2020 were $25.8 million and $91.6 million, respectively, compared to $20.4 million and $97.5 million for the same periods in 2019. R&D expenses in the year ended December 31, 2019 included the accrual of a purchase commitment and higher costs for in-process research and development; excluding the impact of these, research and development expenses have increased as the Company progresses development of its cell therapies.

General and administrative (G&A) expenses: G&A expenses for the fourth quarter and year ended December 31, 2020 were $13.2 million and $45.8 million, respectively, compared to $10.7 million and $43.4 million for the same periods in 2019. The increase in G&A expenses was due to an increase in general corporate costs, including professional fees and insurance.

Net loss: Net loss attributable to holders of the Company’s ordinary shares for the fourth quarter and year ended December 31, 2020 was $36.6 million and $130.1 million ($(0.15 per ordinary share), compared to $29.4 million and $137.2 million ($(0.22) per ordinary share) for the same periods in 2019.
Financial Guidance
The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations into early 2023, as further detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, to be filed with the Securities and Exchange Commission following this earnings release.

Conference Call Information
The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EST (1:00 p.m. GMT) today, February 25, 2021. The live webcast of the conference call will be available via the Events page of Adaptimmune’s corporate website at www.adaptimmune.com. An archive will be available after the call at the same address. To participate in the live conference call, if preferred, please dial (833) 652-5917 (US or Canada) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (2099860).

On February 25, 2021 Celsion Corporation (NASDAQ: CLSN), a clinical-stage development company focused on DNA-based immunotherapy and next-generation vaccines, reported an update on its Phase I/II OVATION 2 Study with GEN-1 in patients with advanced ovarian cancer, including interim observations (Press release, Celsion, FEB 25, 2021, View Source [SID1234575744]). GEN-1 is Celsion’s DNA-mediated interleukin-12 (IL-12) immunotherapy designed using TheraPlas, its proprietary, synthetic, non-viral nanoparticle delivery system platform.

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The OVATION 2 Study combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).

To date, the Company has enrolled approximately one-third, or 34 patients, of the anticipated 110 patients to be enrolled into the OVATION 2 Study, of which 20 are in the treatment arm and 14 are in the control. Currently, 27 patients have had their interval debulking surgery with the following results:

12 of 15, or 80%, of patients treated with GEN-1 had a R0 resection, which indicates a microscopically margin-negative complete resection in which no gross or microscopic tumor remains in the tumor bed.
7 of 12 patients, or 58%, of patients in the control arm had an R0 resection.
This interim data represents a 38% improvement in R0 resection rates for GEN-1- patients compared with control arm patients and is consistent with the reported improvement in resection scores noted in the encouraging Phase I OVATION I Study, the manuscript of which has been submitted for peer review publication.
"As the goal for surgical debulking is to eliminate microscopic disease, more ovarian cancer patients require neoadjuvant chemotherapy. However, little progress has been made in adding additional efficacious immunotherapy agents to standard neoadjuvant chemotherapy," said Premal H. Thaker, M.D., MSc., Professor in Gynecologic Oncology and Director of Gynecologic Oncology Clinical Research at Washington University School of Medicine in St. Louis and lead Principal Investigator for the OVATION 2 Study. "The results seen to date in the OVATION 2 Study are exciting and impactful for ovarian cancer patients."

The Company further reports that 22 clinical sites in the U.S. and Canada have been initiated, with three more sites expected to be added by the end of the first quarter. Clinical investigators met in early February in a virtual meeting and expressed excitement about the potential for GEN-1 to treat advanced ovarian cancer and, despite the challenges and earlier delays posed by the COVID-19 pandemic, they remain committed to completing enrollment in the study during the second half of 2021.

Commenting on the interim patient reports, Dr. Nick Borys, chief medical officer of Celsion, said, "We are gratified that such a high proportion of GEN-1 patients had no residual disease at the time of their debulking surgery. These results are consistent with what we observed in our Phase I (OVATION I) study. This is great news for the patients and the surgeons in our study as a R0 resection suggests a good clinical outcome. We are following our patients carefully to see how well they do long term. The OVATION 2 investigators remain enthusiastic about the potential of GEN-1 to treat late-stage ovarian cancer, which currently has few treatment options."

Celsion announced earlier this week that GEN-1 had received Fast Track designation from the U.S. Food and Drug Administration (FDA). This designation is intended to facilitate the development and expedite the regulatory review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, a Fast Track Drug must show some advantage over available therapy, including:

Showing superior effectiveness, effect on serious outcomes or improved effect on serious outcomes
Avoiding serious side effects of an available therapy
Decreasing a clinically significant toxicity of an available therapy that is common and causes discontinuation of treatment
About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and recently completed a Phase Ib dose-escalation trial (OVATION 1 Study) of GEN-1 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer.

On February 25, 2021 Genome & Company (314130, CEO: Jisoo Pae, Hansoo Park), a global leading immuno-oncology firm, and Debiopharm, a Swiss-based biopharmaceutical company specializing in oncology and infectious diseases, reported having entered into a research collaboration for the discovery of innovative cancer therapies in the expanding antibody-drug conjugates (ADC) class (Press release, Genome & Company, FEB 25, 2021, View Source [SID1234575588]). This research collaboration has been undertaken with the goal of further exploiting this new therapeutic class, potentially offering cancer patients future treatments that efficiently target cancer cells while avoiding impact on healthy tissues.

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The collaboration between the two companies is built on the rapidly expanding Genome & Company’s novel target-based oncology drug pipeline and Debiopharm’s proven track record in oncology. The Swiss company has developed two standards of care in colorectal cancer (oxaliplatin) and in prostate cancer (triptorelin).

Despite ongoing advances in oncology, cancer remains one of the leading causes of death worldwide. Standard-of-care systemic therapies, such as chemotherapy, have benefited cancer patients by killing existing malignant cells, while targeted therapies, such as ADCs, offer new ways of stopping new cancer growth by affecting the genes and proteins necessary for tumor cell survival and replication. In order to accelerate the discovery of innovative tumor-specific treatments, both specialty companies are combining their individual expertise for the development of an optimal ADC candidate based on three important criteria: the target, the cytotoxic payload, and the linker. Specifically, Genome & Company, having generated several antibodies against novel oncology targets discovered based on its own drug development platform, GNOCLE and will now be armed with Debiopharm’s Multilink technology to deliver cytotoxic payloads to tumor cells. Multilink is a unique and innovative technology in that it allows the loading of multiple and different payloads on an antibody. With Debiopharm, Genome & Company will conduct follow-up discussions on the clinical development and the licensing of newly discovered ADC candidates.

"This research collaboration has shown that the Genome & Company has a global level of research and development capability for novel target-based oncology drugs (antibodies) in addition to microbiome therapeutics." said Genome & Company CEO, Jisoo Pae. "We will try to provide novel treatments to cancer patients through active open innovation strategies for the optimal development of novel drugs in the future," he added.

"The collaboration between Genome & Company and Debiopharm is synergizing both our companies’ technology assets and know-how to generate new Antibody Drug Conjugate products. Our aim is to constantly explore different paths to bring novel treatment options to patients with unmet medical needs" said Cédric Sager, CEO of Debiopharm’s development and production facility.

On February 25, 2021 Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, reported financial results for the fourth quarter and full-year of 2020 (Press release, Pacira Pharmaceuticals, FEB 25, 2021, View Source;991.htm [SID1234575626]).

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"Our nation’s opioid crisis has escalated under the shadow of the COVID-19 pandemic, as isolation and lack of access to healthcare has exacerbated mental health challenges, particularly addiction. I am delighted to report that EXPAREL-based protocols are expanding opioid-sparing pain management for a variety of procedures where historically poor postsurgical pain management fueled such addictions. Despite these challenging times, we have quickly adapted to a virtual world and delivered record revenues for 2020," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "Looking ahead to the balance of the year, we remain steadfast in our commitment to providing an opioid alternative to as many patients as possible and redefining the role of opioids as a rescue medication while enabling the migration to hospital outpatient and ambulatory surgery centers for elective surgery."

2020 Full-Year and Fourth Quarter Financial Highlights

Full-year revenues of $429.6 million and fourth quarter revenues of $131.0 million.
Full-year GAAP net income of $145.5 million or $3.41 per share (basic) and $3.33 (diluted).
Fourth quarter GAAP net income of $14.5 million or $0.33 per share (basic) and $0.32 (diluted).
Full-year non-GAAP Adjusted EBITDA of $112.6 million and fourth quarter non-GAAP Adjusted EBITDA of $42.9 million.
Recent Business Highlights

Equity investment in GeneQuine Biotherapeutics.
In January 2021, Pacira announced an equity investment in GeneQuine Biotherapeutics GmbH. Under the terms of the agreement, Pacira made an initial investment of €2.0 million with an additional €4.0 million investment predicated upon GeneQuine achieving certain prespecified near-term milestones related to its lead gene therapy product candidate, GQ-303. Up to €2.5 million of the total Pacira investment will be in the form of a convertible note. In addition, Pacira is entitled to appoint one member to GeneQuine’s board of directors. GeneQuine Biotherapeutics is a privately held biopharmaceutical company advancing a gene therapy platform for the treatment of osteoarthritis (OA) and other musculoskeletal disorders. GeneQuine’s product candidates are next-generation gene transfer vehicles that are highly efficient in entering joint cells to confer multi-year gene expression.

European Commission approves EXPAREL for the treatment of postsurgical pain. In November 2020, the European Commission granted marketing authorization for EXPAREL as a brachial plexus block or femoral nerve block for treatment of post-operative pain in adults, and as a field block for treatment of somatic post-operative pain from small- to medium-sized surgical wounds in adults. The European Commission approval was based on the results of four pivotal Phase 3 studies that demonstrated improvements in pain reduction and opioid use. These studies include: lower extremity nerve block, upper extremity nerve block, and infiltration studies in hard and soft tissue surgeries. The European Commission decision is applicable to all 27 European Union member states plus the United Kingdom, Iceland, Norway and Liechtenstein. Commercial planning is underway, with an anticipated launch in the second half of 2021.
Fourth Quarter 2020 Financial Results

Total revenues were $131.0 million in the fourth quarter of 2020, a 7% increase over the $122.4 million reported for the fourth quarter of 2019.
EXPAREL net product sales were $125.3 million in the fourth quarter of 2020, a 7% increase over the $116.9 million reported for the fourth quarter of 2019.
Fourth quarter 2020 iovera° net product sales were $2.4 million, a 25% decrease versus the $3.2 million reported in the fourth quarter of 2019.
Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $2.0 million in the fourth quarter of 2020, compared to $1.7 million in 2019.
Fourth quarter 2020 royalty revenue was $1.2 million compared to $0.6 million in 2019.
Total operating expenses were $112.2 million in the fourth quarter of 2020, compared to $120.7 million in the fourth quarter of 2019.
Research and development (R&D) expenses were $15.3 million in the fourth quarter of 2020, compared to $19.7 million in the fourth quarter of 2019. The company’s R&D expenses include $5.2 million and $8.7 million of product development and manufacturing capacity expansion costs in the fourth quarters of 2020 and 2019, respectively.
Selling, general and administrative (SG&A) expenses were $52.8 million in the fourth quarter of 2020, compared to $54.2 million in the fourth quarter of 2019.
GAAP net income was $14.5 million, or $0.33 per share (basic) and $0.32 (diluted), in the fourth quarter of 2020, compared to a GAAP net loss of $4.9 million, or $0.12 per share (basic and diluted), in the fourth quarter of 2019.
Non-GAAP net income was $38.8 million, or $0.89 per share (basic) and $0.87 per share (diluted), in the fourth quarter of 2020, compared to non-GAAP net income of $23.8 million, or $0.57 per share (basic) and $0.56 per share (diluted), in the fourth quarter of 2019.
Adjusted EBITDA was $42.9 million in the fourth quarter of 2020, a 48% increase over $29.1 million in the fourth quarter of 2019.
Pacira had 43.5 million basic and 44.7 million diluted weighted average shares of common stock outstanding in the fourth quarter of 2020.
Full-Year 2020 Financial Results

Total revenues were $429.6 million in 2020, a 2% increase over the $421.0 million reported in 2019.
EXPAREL net product sales were $413.3 million in 2020, a 1% increase over the $407.9 million reported in 2019.
Full-year iovera° net product sales were $8.8 million, a 12% increase over the $7.9 million reported in 2019. Pacira began recognizing sales of iovera° in April 2019 after completing its acquisition of MyoScience, Inc., a privately held medical technology company.
Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $4.5 million in 2020, compared to $3.2 million in 2019.
Full-year royalty revenue was $3.0 million compared to $2.1 million in 2019.
Total operating expenses were $383.3 million in 2020, compared to $410.5 million in 2019.
Research and development (R&D) expenses were $59.4 million in 2020, compared to $72.1 million in 2019. The company’s R&D expenses include $23.5 million and $29.7 million of product development and manufacturing capacity expansion costs in 2020 and 2019, respectively.
Selling, general and administrative (SG&A) expenses were $193.5 million in 2020, compared to $200.8 million in 2019.
GAAP net income was $145.5 million, or $3.41 per share (basic) and $3.33 per share (diluted) in 2020, compared to a GAAP net loss of $11.0 million, or $0.27 per share (basic and diluted) in 2019.
Non-GAAP net income was $96.6 million, or $2.26 per share (basic) and $2.21 per share (diluted), in 2020, compared to non-GAAP net income of $70.7 million, or $1.70 per share (basic) and $1.67 per share (diluted), in 2019.
Adjusted EBITDA was $112.6 million in 2020, a 26% increase over $89.2 million in 2019.
Pacira had 42.7 million basic and 43.7 million diluted weighted average shares of common stock outstanding in 2020.
See "Non-GAAP Financial Information" below.

Financial Guidance

The company’s 2021 product sales continue to be negatively impacted by the COVID-19 pandemic, which mandated significant postponement or suspension in the scheduling of elective surgical procedures resulting from public health guidance and government directives. Elective surgery restrictions began to lift on a state-by-state basis in April 2020. In order to provide greater transparency, the company will continue to report monthly intra-quarter unaudited net product sales until it has gained enough visibility around the impacts of COVID-19 to reinstate financial guidance.

Today’s Conference Call and Webcast Reminder

The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Thursday, February 25, 2021, at 8:30 a.m. ET. To participate in the conference call, dial 1-877-845-0779 and provide the passcode 4864607. International callers may dial 1-720-545-0035 and use the same passcode. In addition, a live audio of the conference call will be available as a webcast. Interested parties can access the event through the "Events" page on the Pacira website at investor.pacira.com.

For those unable to participate in the live call, a replay will be available at 1-855-859-2056 (domestic) or 1-404-537-3406 (international) using the passcode 4864607. The replay of the call will be available for one week from the date of the live call. The webcast will be available on the Pacira website for approximately two weeks following the call.

Non-GAAP Financial Information

This press release contains financial measures that do not comply with U.S. generally accepted accounting principles (GAAP), such as non-GAAP net income, non-GAAP net income per share, non-GAAP cost of goods sold, non-GAAP gross margins, non-GAAP research and development (R&D) expense and non-GAAP selling, general and administrative (SG&A) expense and adjusted EBITDA, because such measures exclude acquisition-related charges, product discontinuation costs and other expense; stock-based compensation; amortization of debt discount; loss on early extinguishment of debt, amortization of acquired intangible assets; an income tax benefit and a step-up in basis of inventory in connection with the acquisition of MyoScience, Inc., (gain) loss on investment and other non-operating income and the reversal of a deferred tax valuation allowance.

These measures supplement Pacira’s financial results prepared in accordance with GAAP. Pacira management uses these measures to better analyze its financial results, estimate its future cost of goods sold, gross margins, R&D expense and SG&A expense outlook for 2021 and to help make managerial decisions. In management’s opinion, these non-GAAP measures are useful to investors and other users of our financial statements by providing greater transparency into the operating performance at Pacira and its future outlook. Such measures should not be deemed to be an alternative to GAAP requirements or a measure of liquidity for Pacira. Non-GAAP measures are also unlikely to be comparable with non-GAAP disclosures released by other companies. See the tables below for a reconciliation of GAAP to non-GAAP measures, including adjusted EBITDA.

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])