On March 18, 2021 RedHill Biopharma reported Fourth Quarter and Full Year 2020 Financial Results and Operational Highlights (Press release, RedHill Biopharma, MAR 18, 2021, View Source [SID1234576852])

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Financials:

Full year 2020 net revenues of approximately $64 million, with gross profit of approximately $27.5 million

Solid cash balance of approximately $100 million as of March 4, 2021

Planned commercial operational breakeven by the end of 2021

Commercial Highlights:

Talicia: Consistent month-over-month prescription growth despite pandemic conditions, with 52% new prescription growth in Q4/2020 compared to Q3/2020

Movantik: Market leadership position holding strong and well-positioned for further growth in 2021

R&D Highlights:

Two advanced programs at the forefront of global COVID-19 novel therapeutics development:

Opaganib: Ongoing global Phase 2/3 study in hospitalized patients approximately two thirds enrolled, data expected Q2/2021; Positive U.S. Phase 2 data reported

RHB-107: Ongoing U.S. Phase 2/3 study in symptomatic non-hospitalized patients
RHB-204: Ongoing Phase 3 study for pulmonary NTM disease as oral first-line treatment

Management to host webcast today, at 8:30 a.m. EDT

RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, today reported its financial results and operational highlights for the year ended December 31, 2020.

Dror Ben-Asher, RedHill’s Chief Executive Officer, said: "2020 was a year that our team looks back on with a sense of immense achievement. While navigating the challenging conditions caused by the pandemic, we have delivered broad commercial growth culminating in a very strong end to 2020 for both Movantik and Talicia". Mr. Ben-Asher continued: "At the same time, we have rapidly progressed two novel oral COVID-19 therapies to Phase 3 stage development, covering both hospitalized and non-hospitalized patients and have reported positive clinical and preclinical data, positioning RedHill at the forefront of novel COVID-19 therapeutics development worldwide. With strong momentum across both commercial and R&D operations, we expect 2021 to be a breakout year."

Micha Ben Chorin, Chief Financial Officer at RedHill, added: "RedHill is delivering on a clear strategy designed to enable us to achieve fast growth and increased profit margin. We have been diligent in maintaining a solid balance sheet and we expect to achieve commercial operational breakeven by the end of this year."

Financial highlights for the year ended December 31, 2020

Net Revenues were approximately $64 million for the year ended December 31, 2020, an increase of $58 million compared to the year ended December 31, 2019, attributed to revenues generated from the commercialization of Movantik and Talicia initiated in 2020. Net revenues for the fourth quarter of 2020 were approximately $21.5 million, an increase of $0.5 million compared to the third quarter of 2020, with a 12% increase in product delivery.

Gross Profit was approximately $27.5 million for the year ended December 31, 2020, an increase of $23.5 million compared to the year ended December 31, 2019, primarily due to the increase in net revenues.

Research and Development Expenses were approximately $16.5 million for the year ended December 31, 2020, mainly attributable to the development of our COVID-19 therapeutics and the Phase 3 study of RHB-204 for pulmonary NTM disease, and were, in total, lower than the research and development expenses for the year ended December 31, 2019.

Selling, Marketing and Business Development Expenses were approximately $49 million for the year ended December 31, 2020, compared to approximately $18 million for the year ended December 31, 2019. The increase was attributable to the expansion of our U.S. sales force and marketing activities, in support of the launch of Talicia and post-acquisition commercialization of Movantik.

General and Administrative Expenses were approximately $25 million for the year ended December 31, 2020, compared to approximately $11 million for the year ended December 31, 2019. The increase was mainly attributable to the expansion of commercialization activities related to the Talicia launch and the Movantik acquisition from AstraZeneca.

Operating Loss was approximately $64 million for the year ended December 31, 2020, compared to approximately $43 million for the year ended December 31, 2019. The increase was attributable to the expansion of our commercial operations.

Net Loss was approximately $76 million for the year ended December 31, 2020, compared to approximately $42 million for the year ended December 31, 2019. The increase was attributable to factors mentioned above, as well as interest expenses mainly related to the royalty and debt financing in the first quarter of 2020.

Net Cash Used in Operating Activities was approximately $49 million for the year ended December 31, 2020, compared to approximately $41 million for the year ended December 31, 2019. The increase was attributable to the increase in operating loss, as described above.

Net Cash Used in Investing Activities was approximately $36 million for the year ended December 31, 2020, primarily related to the $52.5 million upfront payment to AstraZeneca for the acquisition of Movantik, partially offset by inflow from current bank deposits and financial assets at fair value through profit or loss.

Net Cash Provided by Financing Activities was approximately $84 million for the year ended December 31, 2020, comprised primarily from financing inflow of approximately $102 million, mainly in debt and equity, partially offset by $16 million classified as restricted cash.

Liquidity and Capital Resources

Cash Balance[1] as of December 31, 2020, was approximately $46 million. Cash balance as of March 4, 2021 was approximately $100 million.

Commercial Highlights

Movantik (naloxegol)[2]
The Company has completed three full quarters of Movantik promotion following its acquisition from AstraZeneca, achieving: A reversal of the trend of declining new prescriptions prior to the acquisition, maintaining Movantik’s position as a segment-leading brand and ending 2020 strongly recording the second highest monthly new prescription volume of 2020 in December.

RedHill acquired the global rights to Movantik from AstraZeneca, excluding Europe and Canada, subsequently adding Israel rights, and replaced a co-commercialization agreement with Daiichi Sankyo (assigned under the agreement with AstraZeneca), with a new royalty-bearing agreement that resulted in RedHill assuming full control over brand strategy and commercialization activities for Movantik in the U.S. and increasing gross margin.

Talicia (omeprazole magnesium, amoxicillin and rifabutin)[3]
Despite the challenging pandemic environment, the Company has persisted in its efforts to support the launch and rapid growth of Talicia, in particular in expanding the prescriber base. This has resulted in the strong accumulation of new Talicia prescribers in the second half of 2020 and consistent month-over-month prescription growth despite pandemic conditions. Talicia ended 2020 strongly, with 52% new prescription growth in the fourth quarter of 2020, as compared to the previous quarter, and achieving its highest weekly new prescription volume in December.

This growth is supported by the addition of Talicia as a preferred brand on leading national formularies, approaching 70% U.S. commercial coverage in the fourth quarter, with further formulary additions expected to add to the previously announced listings of Talicia on the national formularies of Prime Therapeutics, EnvisionRx, and Express Scripts.

Aemcolo (rifamycin)[4]
The Company has implemented plans to support, and build on, the initial momentum that Aemcolo was generating pre-COVID-19 travel restrictions. The Company expects that these plans will drive a resurgence of interest in Aemcolo once travel restrictions are lifted and international travel from the U.S. returns to significant levels.

R&D Highlights

COVID-19 Program: Opaganib (ABC294640, Yeliva)[5]
The late-stage development program for novel, orally-administered, opaganib in patients with severe COVID-19 pneumonia is progressing rapidly. Opaganib has demonstrated dual anti-inflammatory and antiviral activity, targeting a human cell component involved in viral replication and therefore expected to be effective against emerging viral strains with mutations in the spike protein.

The global Phase 2/3 randomized, double-blind, parallel-arm, placebo-controlled study of opaganib in patients with severe COVID-19 pneumonia requiring hospitalization and treatment with supplemental oxygen (NCT04467840), is rapidly advancing in a total of 8 countries and approximately 40 recruiting sites, with additional expansion ongoing. The study has passed three Data Safety Monitoring Board reviews, including a futility review, which is suggestive that the study is progressing as expected. The 464-patient study has already enrolled approximately two thirds of the patients and is expected to deliver top-line data in the second quarter of 2021.

In December 2020, the Company reported positive top-line safety and efficacy data from the U.S. Phase 2 study with opaganib in patients with COVID-19 pneumonia, in which opaganib demonstrated greater improvement in reducing oxygen requirement by end of treatment on Day 14, on top of standard-of-care. The Phase 2 data also showed no material safety differences between the opaganib and placebo treatment arms – further adding to the growing opaganib safety database.

In September 2020, RedHill announced that opaganib demonstrated potent inhibition of SARS-CoV-2, achieving complete blockage of viral replication, as measured after three days incubation, in an in vitro model of human bronchial tissue, comparing favorably with remdesivir, the positive control in the study. Furthermore, treatment of cells infected with SARS-CoV-2 with opaganib did not compromise cell membrane integrity, a measure of cell viability and drug safety, further demonstrating opaganib’s promising potential for treating patients with COVID-19. On top of its anti-inflammatory mechanism, opaganib is also one of very few orally available broad-spectrum antivirals in advanced clinical evaluation for treating COVID-19.

The Company also signed collaborations with several U.S., European and Canadian suppliers, including with Cosmo Pharmaceuticals NV (SIX: COPN) for large-scale ramp-up of opaganib manufacturing, further strengthening manufacturing capabilities and capacity for opaganib.

The Company continues its discussions with U.S. and other government agencies and non-governmental organizations around potential funding to support the rapid advancement of opaganib toward potential emergency use applications and manufacturing scale-up.

COVID-19 Program: RHB-107 (upamostat)[6]
In February 2021, RedHill announced dosing of the first patient in the U.S. Phase 2/3 COVID-19 outpatient study with novel, orally-administered, RHB-107 (upamostat). The study with once-daily RHB-107 is evaluating treatment of patients with symptomatic COVID-19 who do not require hospitalization – the vast majority of COVID-19 patients. The study allows patients to remain in the comfort of their home yet be monitored at a level previously possible only in a hospital setting, enabling increased compliance and retention rates.

RHB-107 is a novel, orally-administered, serine protease inhibitor targeting human cell factors involved in viral entry and is therefore expected to be effective against emerging viral variants with mutations in the spike protein. In previously announced in vitro results, RHB-107 strongly inhibited SARS-CoV-2 viral replication.

RHB-204[7] – Pulmonary Nontuberculous Mycobacteria (NTM) Disease
In November 2020, RedHill commenced its U.S. Phase 3 study to evaluate the efficacy and safety of RHB-204 in adults with pulmonary NTM disease caused by Mycobacterium avium Complex (MAC) infection.

The FDA also granted Fast Track designation for RHB-204 in January 2021, providing early and frequent communications and a rolling review of any New Drug Application (NDA). Having already been granted Qualified Infectious Disease Product (QIDP) designation, RHB-204 is also eligible for NDA Priority Review and Accelerated Approval.

In October 2020, the Company announced that RHB-204 had been granted FDA Orphan Drug Designation. This, along with the previously granted QIDP designation, extends U.S. market exclusivity for RHB-204 to a potential total of 12 years upon FDA approval.

Opaganib – Cholangiocarcinoma and Prostate Cancer
The Phase 2a study evaluating the activity of opaganib in advanced cholangiocarcinoma (bile duct cancer) is ongoing. Enrollment has been completed for the first cohort of 39 patients, evaluating the activity of orally-administered opaganib as a stand-alone treatment. Preliminary data from this cohort indicated a signal of activity in a number of subjects with advanced cholangiocarcinoma. Enrollment is ongoing for a second cohort, evaluating opaganib in combination with hydroxychloroquine, an anti-autophagy agent.

In light of preclinical findings demonstrating tumor regression following combination treatment with opaganib and RHB-107 (upamostat), RedHill plans to add an additional cohort to the ongoing Phase 2a study, evaluating opaganib in combination with RHB-107, subject to discussions with the FDA. Opaganib has been granted FDA Orphan Drug Designation for the treatment of cholangiocarcinoma.

An additional Phase 2 study with opaganib in prostate cancer is ongoing at the Medical University of South Carolina (MUSC). The study is supported by a National Cancer Institute grant awarded to MUSC with additional support from RedHill.

COVID-19 Impact Update

RedHill’s primary concern during the COVID-19 pandemic continues to be the safety and health of its employees, patients, colleagues, and the communities to which we belong.

Operationally, the actions the Company took to mitigate the impact of the COVID-19 pandemic continue to serve us well, with minimal effect on our ongoing operational and supply chain activities. Promotional activity has now been largely re-instated, where safe to do so, and in adherence to social distancing and other public health guidelines. RedHill will continue to assess the potential impact of COVID-19 on its business and operations.

Conference Call and Webcast Information:

The Company will host a conference call and live webcast today, Thursday, March 18, 2021, at 8:30 a.m. EDT to present the fourth quarter and full year 2020 financial results and operational highlights.

The webcast and slides will be broadcast live on the Company’s website, View Source, and will be available for replay for 30 days.

On March 18, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL) a clinical stage biotechnology company dedicated to improving life with its proprietary ARCUS genome editing platform, reported financial results for the fourth quarter and fiscal year ended December 31, 2020 and provided a business update (Press release, Precision Biosciences, MAR 18, 2021, View Source [SID1234576851]).

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"2020 was a pivotal year for Precision with significant advances across our ARCUS-based in vivo gene editing and allogeneic CAR T pipelines. We entered into a research collaboration and exclusive license agreement with Eli Lilly for the research and development of multiple in vivo gene correction therapies aimed at potentially curing genetic disorders, including Duchenne muscular dystrophy. In addition to advancing two new CAR T programs into the clinic, we reported interim study results for our lead CD19 program, which showed continued acceptable safety alongside high objective response rates," commented Matt Kane, CEO and co-founder of Precision BioSciences. "In 2021, we look forward to starting our Phase 1/2a study of PBCAR19B to assess whether our next-generation, stealth, donor CAR T cells can persist longer in the body, a key goal that we believe could lead to deep and durable responses with off-the-shelf CAR T products. We also expect to report in 2021 interim data updates for our three clinical CAR T programs, PBCAR0191, PBCAR20A, and PBCAR269A, and provide an update on our PH1 in vivo gene editing program."

Recent Developments and Upcoming Milestones:

Allogeneic CAR T Portfolio:

PBCAR0191: In December 2020, Precision reported encouraging interim clinical results for its Phase 1/2a study of patients with relapsed/refractory (R/R) non-Hodgkin lymphoma (NHL) and R/R B-cell Acute Lymphoblastic leukemia (B-ALL). For this study, 27 patients across multiple dose levels received PBCAR0191 with either standard lymphodepletion (sLD) (fludarabine 30 mg/m2/day x 3 days + cyclophosphamide 500 mg/m2/day x 3 days) or enhanced lymphodepletion (eLD) (fludarabine 30 mg/m2/day x 4 days + cyclophosphamide 1000 mg/m2/day x 3 days). Response rates were as follows:

83% objective response rate at day 28 or later for patients across NHL (n=4) and B-ALL (n=2) who received PBCAR0191 when coupled with eLD.
At day 28 or later, 75% (3/4) of NHL patients who received PBCAR0191 with eLD achieved a complete response compared to 33% of NHL patients (n=9) using sLD.
The longest demonstrated response was > 11 months in a B-ALL patient.
PBCAR0191 demonstrated a clear dose-dependent increase in peak cell expansion. Compared to sLD, eLD with PBCAR0191 resulted in approximately 95-fold increase in peak cell expansion, and approximately 45-fold increase in area under the curve. PBCAR0191 demonstrated an acceptable safety profile, with no graft versus host disease (GvHD), no grade ≥ 3 cytokine release syndrome, and no grade ≥ 3 neurotoxicity (ICANS).

Precision expects to provide updated interim data for PBCAR0191 by mid-2021.

PBCAR19B: In January 2021, Precision announced that the U.S. Food and Drug Administration (FDA) accepted its investigational new drug application for PBCAR19B, the Company’s next generation, stealth cell, allogeneic CAR T candidate for patients with CD19-positive malignancies such as those with R/R NHL. PBCAR19B is designed to improve the persistence of allogeneic CAR T cells following infusion by reducing rejection by T cells and natural killer (NK) cells. In addition to the CAR gene, the PBCAR19B vector includes a short hairpin RNA that suppresses expression of beta-2 microglobulin (B2M), a component of Class 1 major histocompatibility complex (MHC) molecules found on the cell surface. Reducing or knocking down Class 1 MHC expression on allogeneic CAR T cells has been shown to reduce CAR T cell killing by cytotoxic T cells. The PBCAR19B vector also carries an HLA-E gene intended to reduce rejection of CAR T cells by NK cells that can be stimulated as a result of reduced MHC molecule expression on the cell surface.

The Phase 1 study is expected to begin by mid-year 2021 and will be a non-randomized, open-label, single-dose, dose-escalation and dose-expansion study to evaluate the safety and clinical activity of PBCAR19B in patients with R/R NHL. The primary objective of the study is to identify the maximum tolerated dose and any dose-limiting toxicities.

PBCAR20A: Precision also continues to enroll patients in its Phase 1/2a clinical trial of PBCAR20A, a wholly-owned investigational allogeneic anti-CD20 CAR T therapy for patients with R/R NHL including patients with R/R chronic lymphocytic leukemia or R/R small lymphocytic lymphoma. In February 2021, the study began enrolling patients into dose level 3, a fixed dose of 480 x 106 cells with a max dose of 6.0 × 106 cells/kg. The Company expects to report interim data for the PBCAR20A study in 2021.

PBCAR269A: Precision continues to enroll patients in its Phase 1/2a study of PBCAR269A, its wholly-owned investigational allogeneic CAR T candidate targeting B-cell maturation antigen for the treatment of R/R multiple myeloma, for which Precision has received Fast Track Designation and Orphan Drug Designation from the FDA. In February 2021, the study began enrolling patients into its highest dose cohort, dose level 3 (6.0 × 106 cells/kg) and Precision expects to report interim data in 2021.

Precision also expects to initiate, in the first half of 2021, the combination arm of its ongoing Phase 1/2a clinical study with PBCAR269A and nirogacestat, SpringWorks Therapeutics’ investigational gamma secretase inhibitor. Precision announced its clinical collaboration with SpringWorks in 2020.

In Vivo Gene Correction Portfolio:

Established Genome Editing Research Collaboration with Eli Lilly: In November 2020, Precision and Eli Lilly and Company announced a research collaboration and exclusive license agreement to use Precision’s proprietary ARCUS genome editing platform for the research and development of potential in vivo therapies for genetic disorders. The agreement includes up to six programs, with an initial focus on Duchenne muscular dystrophy and two other undisclosed gene targets.

In connection with the closing of the agreement in January 2021, Precision received an upfront cash payment of $100 million and Eli Lilly made an equity investment of $35 million in Precision’s common stock. Precision is also eligible to receive up to an aggregate of $420 million in potential development and commercialization milestone payments per licensed product, nominating fees for additional targets, and tiered royalties ranging from the mid-single digits to low-teens on product sales should Lilly successfully commercialize a therapy from the collaboration.

PH1: Pre-clinical research continues to progress with Precision’s wholly-owned in vivo gene correction program using its ARCUS genome editing technology to knock out the HAO1 gene as a potential one-time treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disease. The Company expects to provide an update on this program in the first half of 2021.

PCSK9: In February 2021, Molecular Therapy published a paper describing three-year follow-up data showing long-term stable reduction of low-density lipoprotein (LDL) cholesterol levels in nonhuman primates (NHPs) following in vivo gene editing of the PCSK9 gene with ARCUS genome editing. The study was led by James M. Wilson, M.D., Ph.D. and Lili Wang, Ph.D. from the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania. After a one-time vector administration in 2017, NHPs treated with ARCUS have experienced stable reductions of up to 85% in PCSK9 protein levels and a 56% reduction of LDL cholesterol levels.

Corporate:

Intellectual Property Protection: In January 2021, Precision received a Notice of Allowance from the U.S. Patent and Trademark Office for a patent application covering PBCAR19B. The allowed composition claims of this patent application encompass genetically-modified human T cells comprising the Company’s PBCAR19B construct, which is inserted within the T cell receptor alpha constant locus. Once issued, patents arising from this patent family will have standard expiration dates in April 2040.

Leadership: In December 2020, Precision announced that Alex Kelly, the Company’s Chief Corporate Affairs Officer, would serve as the Company’s Interim Chief Financial Officer and Shane Barton, the Company’s Vice President and Corporate Controller, would serve as interim principal accounting officer. These leadership changes followed the departure of Abid Ansari, Precision’s prior Chief Financial Officer, after nearly five years with the organization to pursue a new career opportunity.

Elo Life Systems:

Corporate Structure: In January 2021, Precision disclosed its intention to spin out its wholly-owned subsidiary, Elo Life Systems. Precision is continuing to explore its strategic options, and the timing of any such sale, spinout or other treatment of Elo remains uncertain.

Published Vanilla Genome Paper in Nature Food: In December 2020, researchers at Elo Life Systems in collaboration with Alan Chambers, Ph.D., and the Tropical Research and Education Center at the University of Florida published a paper in Nature Food, reporting on a chromosome-scale, phased Vanilla planifolia genome, which revealed sequence variants for genes that may impact the vanillin pathway, and therefore influence bean quality, including its productivity, flower anatomy, and disease resistance.

Fiscal Year 2020 Financial Results

Cash and Cash Equivalents: As of December 31, 2020, Precision had approximately $89.8 million in cash and cash equivalents, which did not include the $100 million upfront cash payment and $35 million equity investment received from Eli Lilly in connection with the closing of the collaboration and license agreement in January 2021. The Company expects that cash and cash equivalents as of December 31, 2020, cash payments received from Lilly in January 2021, expected operational receipts and available credit will allow the Company to continue its operations into 2023.

Revenues: Total revenues for the year ended December 31, 2020 were $24.3 million, compared to $22.2 million for the year ended December 31, 2019. The increase of $2.1 million in revenue was primarily the result of an increase in collaboration revenue with Servier, offset by a decrease in collaboration revenue from Gilead due to the termination our agreement with them.

Research and Development Expenses: Research and development expenses were $98.1 million for the year ended December 31, 2020, as compared to $82.4 million for the same period in 2019. The increase of $15.7 million was primarily due to increases in direct research and development expenses related to our ongoing CD19 clinical program, increases in employee-related costs associated with increased headcount to support our technology platform development and manufacturing capabilities, and increased costs associated with contract manufacturing organizations and research organizations.

General and Administrative Expenses: General and administrative expenses were $36.1 million for the year ended December 31, 2020, as compared to $27.0 million for the same period in 2019. The increase of $9.1 million was primarily due to increases in employee-related costs associated with increased headcount as well as costs associated with the Company’s growing infrastructure needs.

Net Loss: Net loss was $109.0 million, or $(2.09) per share, for the year ended December 31, 2020, compared to a net loss of $92.9 million, or $(2.21) per share, for the same period in 2019.

On March 18, 2021 PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing novel cancer therapies and infectious disease vaccines based on the Company’s proprietary Versamune T-cell activating technology, reported its financial results for the year ended December 31, 2020 and provided a business update (Press release, PDS Biotechnology, MAR 18, 2021, View Source [SID1234576850]).

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Recent Business Highlights:

Achieved preliminary efficacy benchmark in the Phase 2 combination trial of PDS0101 led by the National Cancer Institute.
Initiated VERSATILE-002, a Phase 2 trial of lead investigational drug candidate PDS0101, in combination with standard of care KEYTRUDA for first-line treatment of patients with metastatic or recurrent HPV-positive head and neck cancer.
Phase 2 trial of lead investigational drug candidate PDS0101, in combination with standard of care chemoradiotherapy for patients with advanced cervical cancer was initiated by the University of Texas MD Anderson Cancer Center.
Expanded consortium for development of PDS0203, a novel, Versamune-based second-generation COVID-19 vaccine to include Blanver Farmoquímica in addition to Farmacore.
Received award commitment of up to $60 million from Brazil’s Ministry of Science, Technology and Innovation (MCTI) to fund clinical development and commercialization of PDS0203.
Strengthened leadership team with the appointment of Seth Van Voorhees as Chief Financial Officer and addition of preeminent oncologist Otis Brawley, M.D. to the board of directors.
"Despite the challenges of 2020, the PDS Biotech team remained focused on the advancement of our Versamune-based drug pipeline," commented Dr. Frank Bedu-Addo, President and Chief Executive Officer of PDS Biotech. "Through that commitment and the strength of our partnerships with leading institutions in the fields of immuno-oncology and infectious disease, we made significant strides in solidifying the safety profile and establishing the efficacy of our Versamune platform and products as we continue to progress our portfolio towards commercialization."

Full Year 2020 Financial Results

For the year ended December 31, 2020, the net loss was approximately $14.8 million, or $0.89 per basic share and diluted share, compared to a net loss of approximately $7.0 million, or $1.44 per basic share and diluted share for the year ended December 31, 2019.

For the year ended December 31, 2020, research and development expenses increased to $7.9 million compared to $6.1 million during the prior year. The increase was primarily the result of increased expenses related to manufacturing and personnel costs for the ongoing clinical studies.

For the year ended December 31, 2020, general and administrative expenses decreased to $7.0 million compared to $11.0 million during 2019. The $4.0 million decrease was due to decreases in personnel costs of $0.4 million, non-cash stock-based compensation of $2.4 million, D&O insurance costs of $0.5 million, legal fees of $0.5 million and professional fees of $0.2 million.

Total operating expenses for 2020 were $14.9 million, a decrease of approximately 29% compared to $21.0 million during the prior year.

The company’s cash balance as of December 31, 2020 was $28.8 million.

Conference Call and Webcast

The conference call is scheduled to begin at 8:00 am ET on Thursday, March 18, 2021. Participants should dial 877-407-3088 (United States) or 201-389-0927 (International) and mention PDS Biotech. Participants can also access the conference call via webcast on the investor relations page of the Company’s corporate website (link).

The event will be archived in the investor relations section of PDS Biotech’s website for 6 months. In addition, a telephonic replay of the call will be available for 6 months. The replay can be accessed by dialing 877-660-6853 (United States) or 201-612-7415 (International) with confirmation code 13716518.

On March 18, 2021 ONK Therapeutics Ltd, an innovative cell therapy company focused on engineered natural killer (NK) cell therapies, reported it has entered into a license with the US National Institutes for Health (NIH) which provides rights to make and use a clinically-validated GMP-grade feeder cell line that will enable robust natural killer (NK) cell expansion, supporting scale-up of the company’s manufacturing process (Press release, ONK Therapeutics, MAR 18, 2021, View Source [SID1234576849]).

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Engineered NK cell therapies are now emerging as an exciting cell therapy platform, that shows promise in overcoming challenges of earlier CAR-T cell therapies, offering alternative therapeutic options for patients with hematological malignancies and solid tumors. Successful ex-vivo expansion of cord-derived NK cells is key to developing large cell batch sizes required to produce truly off-the-shelf engineered NK cell therapy.

This proprietary, GMP grade Epstein Barr Virus-transformed lymphoblastoid cell line (EBV-LCL) has been proven as a method to expand NK cells as part of a clinical trial being led by Dr. Richard Childs at the NIH. The feeder layer enabled a robust expansion of NK cells with greatly enhanced cytotoxic potential and strong expression of Activating Receptors and Death Receptor ligands. Expansion of up to 1,000 fold over 2-3 weeks was demonstrated with the potential for continued exponential expansion over longer periods. This expansion provides a stable and consistent NK cell population that can be subsequently genetically modified and further expanded on a batch scale supportive of clinical trials of ONK’s NK cell therapy candidates.

Prof. Michael O’Dwyer, ONK Therapeutics’ founder and CSO said: "Extensive experience over many years in Dr. Childs’ group at the NIH indicate that co-culture with EBV-LCL cells enables ex-vivo activation and robust proliferation of highly functional NK cells, which can be safely administered to patients. Applying this technology to our cord-derived, dual-targeted NK cell platform will enable the manufacture of large quantities of optimized NK cells for off-the-shelf administration."

ONK Therapeutics is accelerating the development of its NK cell engineering and manufacturing processes for its unique, proprietary dual-targeted NK cell platform that expresses both a chimeric antigen receptor (CAR) targeting a known tumor antigen and a TNF-related apoptosis-inducing ligand variant (TRAILv) targeting the death receptor pathway (i.e. DR4 or DR5).

This agreement with the NIH follows quickly on the heels of a complementary license that ONK Therapeutics recently announced with Anthony Nolan Cell & Gene Therapy Services. This license provides a consistent supply of scalable and ethically-sourced umbilical cord blood and cord-derived NK cells which, in combination with the EBV-LCL feeder layer, will provide the critical starting material for its off-the-shelf cell therapy manufacturing process, to be used for both ONK Therapeutics’ research activities and continued process development work.

Chris Nowers, ONK Therapeutics’ CEO said: "This important license further enhances our manufacturing capability, taking us another step closer to our goal of creating a next generation of uniquely engineered NK cell therapies, with broad applicability across a wide range of targets and tumor types."

On March 18, 2021 Innate Pharma SA (Euronext Paris: IPH – ISIN: FR0010331421; Nasdaq: IPHA) ("Innate" or the "Company") reported its consolidated financial results for the year ending December 31, 2020 (Press release, Innate Pharma, MAR 18, 2021, View Source [SID1234576848]). The consolidated financial statements are attached to this press release.

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"In 2020, we made the strategic decision to re-prioritize our investments in our R&D portfolio, enabling us to concentrate our resources and further strengthen our clinical pipeline," commented Mondher Mahjoubi, Chief Executive Officer of Innate Pharma. "Our priority going forward is to advance the clinical development of our lead proprietary candidate, lacutamab, as well as leverage our multispecific NKCE antibody platform, to create potential innovative therapeutics for patients and provide long-term value to our shareholders."

Pipeline highlights:

Lacutamab (IPH4102, anti-KIR3DL2 antibody):

The TELLOMAK Phase 2 clinical trial, which is evaluating the efficacy and safety of lacutamab in patients with advanced cutaneous T-cell lymphomas, is now fully open to enrollment in countries that had a partial regulatory hold following the successful resolution of Good Manufacturing Practice issues.
In November, the Company announced that the European Medicines Agency (EMA) granted PRIME designation to lacutamab for the treatment of patients with relapsed or refractory Sézary syndrome (SS) who have received at least two prior systemic therapies. This is the first time PRIME designation has been granted for a potential treatment of any sub-type of T-cell lymphoma. This follows the Fast Track designation that was awarded to lacutamab by the U.S. Food and Drug Administration in 2019.
In February 2021, the Company announced lacutamab demonstrated a positive early signal in cohort 2 of KIR3DL2-expressing mycosis fungoides patients in the TELLOMAK clinical trial earlier than anticipated. This cohort reached the pre-determined number of responses needed to advance to stage 2. The Company plans to present this preliminary data at a scientific meeting in 2021.
The Company will initiate two parallel clinical trials to study lacutamab in KIR3DL2-expressing patients with relapsed/refractory peripheral t-cell lymphoma (PTCL):
Phase 1b trial: a Company-sponsored Phase 1b clinical trial to evaluate lacutamab as a monotherapy in KIR3DL2-expressing patients with relapsed PTCL.
Phase 2 KILT (anti-KIR in T Cell Lymphoma) trial: The Lymphoma Study Association (LYSA) will launch an investigator-sponsored, randomized trial to evaluate lacutamab in combination with chemotherapy GEMOX (gemcitabine in combination with oxaliplatin) versus GEMOX alone in KIR3DL2-expressing relapsed/refractory patients.
IPH6101 (NKp46-based NK cell engager), partnered with Sanofi:

Progress was made in the NKCE collaboration with Sanofi, resulting in the decision announced in January 2021 that Sanofi will transition IPH6101/SAR443579 into investigational new drug (IND)-enabling studies. IPH6101 is a NKp46-based NK cell engager (NKCE) using Innate’s proprietary multispecific antibody format (Gauthier et al. Cell 2019). The decision triggered a €7 million milestone payment from Sanofi to Innate.
In January 2021, a GLP-tox study was initiated for the IPH6101/SAR443579 program.
Monalizumab (anti-NKG2A antibody), partnered with AstraZeneca:

In October 2020, AstraZeneca (LES/STO/Nasdaq: AZN) dosed the first patient in its randomized Phase 3 clinical trial, INTERLINK-1, evaluating monalizumab in combination with cetuximab vs. placebo and cetuximab in patients with recurrent or metastatic squamous cell carcinoma of the head and neck (R/M SCCHN) who have been previously treated with platinum-based chemotherapy and PD-(L)1 inhibitors. Dosing of the first patient in this trial triggered a $50 million milestone payment from AstraZeneca to Innate. Innate is eligible to receive an additional $50 million milestone payment after the interim analysis demonstrates the combination meets a pre-defined threshold of clinical activity. To date, the Company has received a total of $400 million from the AstraZeneca partnership for monalizumab.
The Company presented efficacy data on the Phase 2 expansion cohort investigating the combination of monalizumab and cetuximab in patients with recurrent or metastatic head and neck squamous cell cancer (R/M SCCHN) who have been previously treated with platinum-based chemotherapy and PD-(L)1 inhibitors at ASCO (Free ASCO Whitepaper)20 Virtual Scientific Conference held in May 2020. This data showed an overall response rate in line with previously reported data and a manageable safety profile. The Company presented updated results at the ESMO (Free ESMO Whitepaper) Immuno-oncology Virtual Congress in December 2020.
In 2020, the Company expanded a Phase 2 expansion cohort ("cohort 3"), exploring the combination of monalizumab, cetuximab and durvalumab in first-line IO naïve patients with R/M SCCHN, from 20 to 40 patients. Recruitment for cohort 3 is complete, and the Company expects to publish data in 2021.
Avdoralimab in Inflammation (anti-C5aR1 antibody):

• In November 2020, the first patient was dosed in the investigator-sponsored Phase 2 clinical trial in bullous pemphigoid (BP) where the C5aR1 pathway has been shown to be involved in the physiopathology of the disease. The trial is investigating the clinical efficacy of avdoralimab in addition to topical steroids compared to topical steroids alone in BP patients.

Avdoralimab in COVID-19:

The investigator-sponsored Phase 2 clinical trial, FORCE (FOR COVID-19 Elimination), has completed enrollment and is ongoing for patient follow-up and data analysis. More information on this study can be found at clinical trials.gov.
Results from the exploratory translational EXPLORE study supporting this trial were published online in Nature on July 29, 2020.
The investigator-sponsored Phase 2 clinical trial, ImmunONCOVID-20, has resumed, and is currently recruiting. This study is exploring the potential efficacy of monalizumab and avdoralimab amongst other treatment arms, against COVID-19 in cancer patients with mild symptoms and pneumonia respectively.
In August 2020, the Company announced it obtained €6.8 million in public funding from the French government for its COVID-19 R&D activities. This funding is part of the government’s PSPC COVID call for COVID-19 related projects and will enable the Company to cover the development of its current COVID-19 activities, which began in March 2020, including the EXPLORE COVID-19 translational research study and its two Phase 2 clinical trials, FORCE and ImmunONCOVID-20.
Avdoralimab in Oncology:

• In September 2020, the Company announced the decision to stop enrollment in STELLAR‑001, a Phase 1 dose escalation and expansion study in combination with durvalumab in three expansion cohorts: 1) NSCLC patients with secondary resistance to prior immuno-oncology (IO) treatment; 2) IO-naïve HCC patients; and 3) IO-pretreated HCC patients. The decision was made based on the data from the Company’s cohort expansions in NSCLC and IO-naïve HCC.

IPH5201 (anti-CD39 antibody), partnered with AstraZeneca:

• In February 2020, the AstraZeneca sponsored, multicenter, open-label, dose-escalation Phase I trial evaluating IPH5201 as monotherapy or in combination with durvalumab (anti-PD-L1) with or without oleclumab (anti-CD73) in advanced solid tumors started. Following the dosing of the first patient in the trial on March 9, 2020, AstraZeneca made a $5 million milestone payment to Innate under the companies’ October 2018 multi-product oncology development collaboration. Innate made a €2.7 million milestone payment to Orega Biotech SAS pursuant to Innate’s exclusive licensing agreement.

Lumoxiti (CD22-directed immunotoxin):

In December 2020, the Company announced that it will return the US and EU commercialization rights of Lumoxiti (moxetumomab pasudotox-tdfk) to AstraZeneca3. Innate licensed the US and EU rights to AstraZeneca’s FDA-approved Lumoxiti for certain patients with relapsed or refractory hairy cell leukemia in October 2018.
Innate and AstraZeneca are currently in discussions regarding the transition plan for the transfer of the US marketing authorization and distribution of Lumoxiti to AstraZeneca, including timing and costs (see Contingent liabilities).
Corporate Update:

In July 2020, Dr. Joyson Karakunnel was appointed as Executive Vice President and Chief Medical Officer (CMO). Dr. Pierre Dodion, CMO since 2014, retired from this position. Dr. Karakunnel comes to the Company with deep experience in immuno-oncology, and a proven track record in drug development. Most recently, Dr. Karakunnel served as CMO and Senior Vice President at Tizona Therapeutics, where he led the development of the company’s biotherapeutics pipeline.
Laure-Helene Mercier, Executive Vice President, Chief Financial Officer and member of the Executive Board, has decided to step down from her position, after leading the Company through more than 14 years of growth, including an initial public offering in the US. Frederic Lombard will join the company as CFO on April 1, 2021. Mr. Lombard will be joining Innate with more than 20 years of financial experience in the pharmaceutical industry, holding senior finance roles at Ipsen, AstraZeneca and Novartis. Ms. Mercier will remain at the Company until the end of the year to ensure a smooth transition of responsibilities.

Financial highlights for 2020:

The key elements of Innate’s financial position and financial results as of and for the year ended December 31, 2020 are as follows:

Cash, cash equivalents, short-term investments and financial assets amounting to €190.6 million (€m) as of December 31, 2020 (€255.9m as of December 31, 2019), including non-current financial instruments amounting to €38.9m (€37.0m as of December 31, 2019).
Cash and cash equivalents include the milestone payment of $50.0m (€41.2m) following the inclusion by AstraZeneca of the first patient in its Phase 3 randomized clinical trial evaluating monalizumab, INTERLINK-1. It doesn’t include the milestone payment of €7.0m from Sanofi relating to the progress of IPH6101/SAR443579 into new drug (IND)-enabling studies, received in February 2021.
As of December 31, 2020, financial liabilities amount to €19.1m (€18.7m as of December 31, 2019). This change is partly linked to the receipt, in August 2020, of €1.4m in repayable advance in connection with the financing contract signed with BPI Financement (COVID-19).
Revenue and other income amounted to €70.5m in 2020 (2019: €85.8m, -17.9%). It mainly comprises revenue from collaboration and licensing agreements (€56.2m in 2020 vs €69.0m in 2019, -18.6%), and research tax credit (€13.1m in 2020 vs €16.7m in 2019, -21.8%):
Revenue from collaboration and licensing agreement with AstraZeneca amounted to €49.0m in 2020 (€69.0m in 2019, -29.0%) and mainly resulted from (i) the spreading of the upfront and opt-in payments received from AstraZeneca and (ii) the invoicing to AstraZeneca of certain fees for the work performed by Innate for the partnered programs. The variation between the two periods is notably explained by the completion of (i) the recruitment of the Cohort 2 in the monalizumab Phase 2 trials performed by Innate in 2019, and (ii) the preclinical work related to the Phase 1 program of IPH5201, which started in 2020.
Revenue of €7.0m from Sanofi for the progress of IPH6101/SAR443579 into investigational new drug (IND)-enabling studies.
The variation in the research tax credit mainly results from a decrease in the amortization for the intangible assets related to acquired licenses (monalizumab, IPH5201).
Operating expenses of €89.9m in 2020 (2019: €104.6m, -14.1%):
Selling, general and administrative (SG&A) expenses amounted to €31.2m in 2020 (2019: €25.8m, +21.1%). This increase mainly results from the full-year effect of personnel costs related to our US subsidiary, including personnel assigned to Lumoxiti commercial activities.
R&D expenses amounted to €58.6m in 2020 (2019: €78.8m, -25.7%). This variation mainly results from a decrease in direct R&D expenses (mainly related to Lumoxiti, IPH5201 and IPH5301) and in depreciation and amortization of intangible assets acquired by the Company (IPH5201 and monalizumab).
Lumoxiti intangible asset full impairment of €43.5m, following the Company’s decision to return the US and EU commercialization rights of Lumoxiti to AstraZeneca.
The Lumoxiti distribution agreement generated a net income of €0.9m in the first three quarters of 2020 (a net loss of €8.2m in 2019). During the 2020 fourth quarter, the Company recognized net sales from Lumoxiti of €0.7m.
A net loss of €64.0m in 2020 (2019: net loss of €20.8m).