On March 2, 2021 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported financial results for the fourth quarter and twelve months ended December 31, 2020 (Press release, VBI Vaccines, MAR 2, 2021, View Source [SID1234575915]). The Company also provided a corporate update and its outlook for 2021.

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Annual Note from Jeff Baxter, President and CEO:

"2020 was, unfortunately, a historic year, marked by unprecedented disruption, with severe public health, societal, and economic consequence. Every single person, worldwide, felt the devastating effects of the ongoing COVID-19 pandemic. Industries and companies went through extraordinary change and, amidst the temporary adjustments, new normals were established. The impact of this pandemic is likely to be felt for years, if not decades, to come.

The events of 2020 led to impressive collaboration, progress, and transformation across the biotechnology industry, governments, and foundations. We added two new vaccine candidates to our pipeline in 2020 – a multivalent pan-coronavirus vaccine candidate, VBI-2901, and a monovalent COVID-19 vaccine candidate, VBI-2902. To support the advancement of these candidates, we received an award from the Strategic Innovation Fund of the Government of Canada and partnered with both the National Research Council of Canada (NRC), Canada’s largest federal R&D organization, and Resilience Biotechnologies, a Contract Development and Manufacturing Organization. The preclinical results of these two candidates continue to excite us and we are working hard to get these candidates into the clinic in forms that are optimized both for clinical outcome and long-term commercial viability. We recognize the possibility that COVID-19, in some form, may be here to stay, especially with the recent emergence of additional variants, and we are committed to the long-term control of known and emerging coronaviruses.

Our 2020 achievements and progress, however, extend well beyond our coronavirus programs. We successfully completed the pivotal Phase 3 program for our 3-antigen prophylactic hepatitis B (HBV) vaccine candidate and submitted applications for approval in the U.S. and Europe. We believe this vaccine candidate has the potential to be a meaningful intervention for adults in the fight against HBV and we look forward to working with both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) throughout 2021 as they conduct their review.

In addition to the advancement of these prophylactic vaccine candidates, we continue to see meaningful data generated by the clinical studies of our therapeutic vaccine candidates targeting both chronic HBV infection, VBI-2601, and recurrent glioblastoma (GBM), VBI-1901. With both of these candidates, we are seeking to address diseases that are challenging and aggressive, with few, if any, effective treatment options available to patients. Based on the positive data seen to-date, we and our partners expect to initiate subsequent clinical studies in both indications in 2021.

These achievements are a result of the continued hard work, dedication, and flexibility of every member of the VBI team. Our team remains united across the US, Canada, and Israel in our mission to protect and enhance human life, and we thank our shareholders and partners for their support. With $119.1 million in cash, cash-equivalents, and short-term investments on-hand at the end of 2020, we entered 2021 well-positioned to achieve meaningful milestones across all of our lead pipeline programs over the next 12 months, and beyond."

Second Half 2020 Key Program Achievements and Projected Upcoming Milestones

3-Antigen Hepatitis B Vaccine Candidate

November 2020: Biologics License Application (BLA) and Marketing Authorization Application (MAA) submitted to U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), respectively
December 2020 and February 2021: EMA acceptance of MAA filing, and FDA acceptance of BLA filing, initiating the review process
December 2020: Announcement of Syneos Health (Syneos) and VBI partnership for commercialization in the U.S., Europe, and Canada, pending regulatory approvals
November 30, 2021: U.S. Prescription Drug User Fee Act (PDUFA) target action date set by FDA
VBI-2900: Coronavirus Vaccine Program

August 2020: Two clinical candidates selected with the goal of bringing forward candidates that add meaningful clinical and medical benefit to those already approved – be it as a one-dose administration, more durable responses, and/or providing broader protection against known and future mutated strains of COVID-19
VBI-2901 : a trivalent candidate, expressing SARS-CoV-2, SARS-CoV, and MERS-CoV spike proteins
VBI-2902 : a monovalent candidate, expressing the SARS-CoV-2 spike protein
September 2020: Through its Strategic Innovation Fund, the Canadian Government agreed to contribute up to CAD$56 million to support VBI-2900 clinical development through Phase 2 studies, to be contributed as expenses are incurred
December 2020 : Broadened collaboration with NRC to include additional support for pre-clinical evaluation, optimization, and scale-up
March 2021: Phase 1/2 clinical study of VBI-2902 in adults expected to initiate in Canada
2021: Phase 1/2 clinical study of VBI-2901 expected to initiate
VBI-2601 (BRII-179): HBV Immunotherapeutic Candidate

November 2020: Positive interim Phase 1b/2a proof-of-concept data announced suggesting restoration of antibody and T cell responses
Q1 2021: Partner, Brii Biosciences, expected to initiate Phase 2 combination study to assess VBI-2601 (BRII-179) and BRII-835 (VIR-2218), a novel RNAi therapeutic, as potential functional cure in chronically infected patients
VBI-1901: Cancer Vaccine Immunotherapeutic Candidate

November 2020: Positive data announcement from ongoing Phase 1/2a clinical study of VBI-1901 in recurrent GBM patients, including 2 partial responses (PRs), observed with tumor reduction of more than 50%, and 7 stable disease (SD) observations
H2 2021: Expected initiation of a randomized, controlled clinical study with the potential to yield registrational data
Financing

Throughout the fourth quarter of 2020, VBI raised total gross proceeds of $15.9 million, issuing 4.8 million shares at an average price of $3.32 through its Open Market Sales AgreementSM, established July 31, 2020 with Jefferies LLC
Financial Results for the Three and Twelve Months Ended December 2020

Cash Position: VBI ended the fourth quarter of 2020 with $119.1 million cash, cash equivalents, and short-term investments compared with $44.2 million as of December 31, 2019.
Net Cash Used in Operating Activities: Net cash used in operating activities for the full year 2020 was $47.1 million, compared to $48.7 million for the same period in 2019.
Cash Used for Purchase of Property and Equipment: The purchase of property and equipment in 2020 was $1 million, compared to $3.7 million in 2019. The decrease is a result of the completion of the modernization and capacity increase at the manufacturing facility in Rehovot, Israel.
Revenue: Revenue for the three months ended December 31, 2020 and for the full year 2020 was $0.2 million and $1.1 million, respectively, compared to $0.6 million and $2.2 million for the same time periods in 2019, respectively. There was a decrease in product revenue due to limited product availability as we prepared for our U.S. and Europe regulatory submissions for our 3-antigen HBV vaccine candidate, which occurred in Q4 2020. Additionally, there was a decrease as a result of the license revenue earned as part of the License Agreement with Brii Bio in 2020 compared to 2019.
Research and Development (R&D): R&D expenses for the fourth quarter and full year 2020 were $4.8 million and $14.9 million, respectively, compared to $4.3 million and $26.3 million for the same periods in 2019, respectively. The decrease in R&D spend in 2020 was primarily due to a decrease in costs related to the Phase 3 clinical studies of our 3-antigen prophylactic HBV vaccine candidate, which were both completed in 2020, but were ongoing in 2019. The decrease in R&D expenses was offset by increased analytical development, manufacturing, and clinical costs associated with our eVLP vaccine candidates.
General and Administrative (G&A): G&A expenses for the fourth quarter and full year 2020 were $7.1 million and $20.7 million, respectively, compared to $3.8 million and $14.1 million for the same periods in 2019, respectively. The increase in G&A expense in 2020 was primarily due to an increase in pre-commercialization activities related to our 3-antigen prophylactic hepatitis B vaccine, increased insurance costs, and increased people costs.
Impairment Charge: There were no impairment charges in 2020, compared to a charge of $6.3 million in 2019 related to goodwill.
Net Loss: Net Loss and net loss per share for the year ended December 31, 2020 were $46.2 million and $0.21, respectively, compared to a net loss of $54.8 million and a net loss per share of $0.46 for the year ended December 31, 2019. The decrease in net loss resulted primarily from decreased R&D expenses offset by the increased cost of revenues and G&A expenses.

On March 2, 2021 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported financial results for the fourth quarter and full year ended December 31, 2020, including sales of TAVALISSE (fostamatinib disodium hexahydrate) tablets, for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment (Press release, Rigel, MAR 2, 2021, View Source [SID1234575931]).

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"Our team has shown its resilience as we continue to execute on our mission to serve patients who have diseases where few or no approved treatment options exist," said Raul Rodriguez, Rigel’s president and CEO. "Despite the challenges brought on by 2020, we successfully expanded our global ITP reach and positioned ourselves for potential success in wAIHA, announced a major collaboration with Lilly to develop RIP1 inhibitors, and launched a comprehensive COVID-19 clinical program which has gained the support from the NIH, DOD, and several universities. Importantly, we also continued to explore opportunities in immunology, and more recently heme-onc, with our IRAK 1/4 inhibitor program."

Business Update
In February 2021, Rigel and Eli Lilly (Lilly) announced a global strategic collaboration to co-develop and commercialize R552, Rigel’s receptor-interacting serine/threonine-protein kinase 1 (RIP1) inhibitor, for all indications, including autoimmune and inflammatory diseases. In addition, Lilly will lead the development and commercialization of all RIP1 inhibitors in central nervous system (CNS) indications. Under the terms of the agreement, Lilly will pay an upfront cash payment to Rigel of $125 million and is eligible to receive up to $835 million in potential development, regulatory and commercial milestone payments, as well as tiered royalties that will vary depending upon Rigel’s clinical development investment.

Rigel launched a Phase 3 clinical trial to evaluate fostamatinib for the treatment of hospitalized COVID-19 patients. The Phase 3 trial is designed to evaluate the safety and efficacy of fostamatinib in hospitalized COVID-19 patients without respiratory failure that have certain high-risk prognostic factors. This multi-center, double-blind, placebo-controlled, adaptive design study is expected to enroll over 300 evaluable patients that will be randomly assigned to either fostamatinib plus standard of care (SOC) or matched placebo plus SOC (1:1). Treatment will be administered orally twice daily for 14 days. There will be a follow-up period to day 60. The primary endpoint of this study is the proportion of subjects who progress to severe/critical disease within 29 days.

Rigel was awarded $16.5 million from the U.S. Department of Defense’s (DOD) Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) to support Rigel’s Phase 3 clinical trial of fostamatinib in hospitalized COVID-19 patients.

The Phase 2 clinical trial of fostamatinib in hospitalized COVID-19 patients sponsored by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH), in collaboration with Inova Health System has currently enrolled 57 patients. This is a double-blind, placebo-controlled Phase 2 clinical trial that is randomly assigning fostamatinib plus standard of care (SOC) or matched placebo plus SOC (1:1) to approximately 60 evaluable patients who are a 5 to 7 on the 8-point ordinal scale (requiring supplemental oxygen via nasal canula or non-invasive ventilation, requiring mechanical ventilation or extracorporeal membrane oxygenation). The primary endpoint of this study is cumulative incidence of serious adverse events (SAE) through day 29. The NHLBI and Rigel expect to report topline data from this clinical trial in April 2021.

Imperial College London’s ongoing Phase 2 clinical trial of fostamatinib in hospitalized COVID-19 patients is a two-stage open label, controlled trial with patients randomized (1:1:1) to fostamatinib, ruxolitinib, or standard of care. The study has currently enrolled 106 patients. Treatment will be administered twice daily for 14 days and patients will receive a follow-up assessment at day 14 and day 28 after the first dose. The primary objective will be to determine the efficacy of fostamatinib and the efficacy of ruxolitinib compared to standard of care to reduce the proportion of hospitalized patients progressing from mild or moderate to severe COVID-19 pneumonia.

Enrollment is progressing in Rigel’s FORWARD study, a Phase 3 pivotal trial of TAVALISSE in warm autoimmune hemolytic anemia (wAIHA). The study has enrolled 66 of 90 patients targeted for enrollment. Rigel has reached agreement with the U.S. Food and Drug Administration (FDA) on the durable response measure for the primary efficacy endpoint of the study as well as the inclusion of additional secondary endpoints. The company recently announced that the FDA had granted Fast Track designation to TAVALISSE for the treatment of wAIHA. The FDA previously granted TAVALISSE Orphan Drug designation for this indication. If approved, TAVALISSE may be the first-to-market therapy for patients with wAIHA.

Rigel continues to advance the development of its IRAK1/4 program, which includes R835, an orally available, potent and selective inhibitor that inhibits both IRAK1 and IRAK4. The company is currently identifying therapeutic opportunities in the areas of hematology/oncology and rare immunology diseases.

In November 2020, Rigel and its partner Medison Pharma announced that Health Canada approved the new drug submission (NDS) for TAVALISSE for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to other treatments. Medison is also anticipating a decision on a New Drug Application (NDA) in Israel in Q2 2021. In July 2020, Rigel and its partner Grifols S.A. announced the launch of TAVLESSE in Germany and the United Kingdom following approval by the European Commission in January 2020. Currently, TAVALISSE is in a Phase 3b clinical trial in Japan with Rigel’s partner Kissei. This trial is required for approval by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan.

Financial Update
For the fourth quarter of 2020, Rigel reported a net loss of $19.2 million, or $0.11 per share, compared to net loss of $17.2 million, or $0.10 per share, in the same period of 2019.

In the fourth quarter of 2020, total revenues were $18.5 million, consisting of $17.8 million in TAVALISSE net product sales and $697,000 in contract revenues from collaborations. TAVALISSE net product sales of $17.8 million increased by 28% from $13.8 million in the fourth quarter of 2019. Contract revenues from collaborations of $697,000 for the fourth quarter of 2020 consisted of $500,000 from Grifols related to an option for commercialization in additional territories and $197,000 in revenues earned from the performance of certain research and development services from Rigel’s collaboration agreement with Grifols.

Rigel reported total costs and expenses of $37.3 million in the fourth quarter of 2020, compared to $32.7 million for the same period in 2019. The increase in costs and expenses was due to the increase in research and development costs primarily related to the continued work on Rigel’s Phase 1 clinical trial in IRAK 1/4 inhibitor program, as well as its recently launched Phase 3 clinical trial for hospitalized COVID-19 patients, partially offset by the decrease in costs on Rigel’s ongoing Phase 3 clinical trial in wAIHA.

For the full year ended December 31, 2020, Rigel reported a net loss of $29.7 million, or $0.18 per share, compared to a net loss of $66.9 million, or $0.40 per share, for the same period of 2019.

Rigel reported total revenues of $108.6 million for the year ended December 31, 2020, compared to $59.3 million in the same period of 2019. Total revenues for the year ended December 31, 2020 consisted of $61.7 million in TAVALISSE net product sales and $46.9 million in contract revenues from collaborations. TAVALISSE net product sales of $61.7 million increased by 41% from $43.8 million in 2019. The increase in contract revenues from collaborations related to revenue from the upfront fee previously received in 2019, as well as the milestone payment received from Grifols in the first quarter of 2020 upon EC approval of the MAA for fostamatinib in Europe, and the $2.1 million in contract revenues for the achievement of a milestone in accordance with the amended license and collaboration agreement with Daiichi-Sankyo, partially offset by the developmental and commercial milestones from its various collaborative partners in 2019.

Total costs and expenses for the year ended December 31, 2020, were $137.6 million, compared to $128.4 million, for the same period of 2019. The increase in total costs and expenses was primarily related to the increases in research and development costs due to the completion of Rigel’s Phase 1 clinical trial in RIP1 inhibitor program, ongoing work on recently launched Phase 3 clinical trial for hospitalized COVID-19 patients, continued work on Rigel’s Phase 1 clinical trial in IRAK 1/4 inhibitor program and ongoing Phase 3 clinical trials in wAIHA, as well as increases in personnel-related costs, partially offset by decreases in various third-party costs due to the COVID-19 pandemic.

As of December 31, 2020, Rigel had cash, cash equivalents and short-term investments of $57.3 million, compared to $98.1 million as of December 31, 2019.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time
Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call and accompanying slides will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About ITP
In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs) and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 45,000 adult patients in the U.S. and can be a severe, debilitating disease. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients. Warm antibody AIHA (wAIHA), the most common form of AIHA, is characterized by the presence of antibodies that react with the red blood cell surface at body temperature.

About COVID-19 & SYK Inhibition
COVID-19 is the infectious disease caused by Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2). SARS-CoV-2 primarily infects the upper and lower respiratory tract and can lead to acute respiratory distress syndrome (ARDS). Additionally, some patients develop other organ dysfunction including myocardial injury, acute kidney injury, shock resulting in endothelial dysfunction and subsequently micro and macrovascular thrombosis.1 Much of the underlying pathology of SARS-CoV-2 is thought to be secondary to a hyperinflammatory immune response associated with increased risk of thrombosis.2

SYK is involved in the intracellular signaling pathways of many different immune cells. Therefore, SYK inhibition may improve outcomes in patients with COVID-19 via inhibition of key Fc gamma receptor (FcγR) and c-type lectin receptor (CLR) mediated drivers of pathology, such as inflammatory cytokine release by monocytes and macrophages, production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation.3,4,5 Furthermore, SYK inhibition in neutrophils and platelets may lead to decreased thromboinflammation, alleviating organ dysfunction in critically ill patients with COVID-19.

About R5526
The investigational candidate, R552, is an orally available, potent and selective inhibitor of receptor-interacting serine/threonine-protein kinase 1 (RIP1). RIP1 is believed to play a critical role in necroptosis. Necroptosis is a form of regulated cell death where the rupturing of cells leads to the dispersion of their inner contents, which induces immune responses and enhances inflammation. In preclinical studies, R552 prevented joint and skin inflammation in a RIP1-mediated murine model of inflammation and tissue damage. The safety and efficacy of R552 has not been established by the FDA or any healthcare authority.

About R8356
The investigational candidate, R835, is an orally available, potent and selective inhibitor of IRAK1 and IRAK4 that has been shown preclinically to block inflammatory cytokine production in response to toll-like receptor (TLR) and the interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response, and dysregulation of these pathways can lead to a variety of inflammatory pathological conditions. R835 treatment demonstrates amelioration of clinical symptoms in multiple rodent models of inflammatory disease including psoriasis, arthritis, lupus, multiple sclerosis and gout. The safety and efficacy of R835 has not been established by the FDA or any healthcare authority.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.

On March 2, 2021 Purple Biotech Ltd. ("Purple Biotech") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class, effective, and durable therapies by overcoming tumor immune evasion and drug resistance, reported financial results for the six and 12-months ended December 31, 2020 (Press release, Purple Biotech, MAR 2, 2021, View Source [SID1234575947]).

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"We have recently achieved substantial progress in multiple key aspects of our business," said Isaac Israel, Chief Executive Officer. "Most importantly, our promising anti-cancer product development pipeline continues to advance our Phase 1/2 clinical studies for NT219, a dual inhibitor, novel small molecule targeting IRS1/2 and STAT3. We completed patient recruitment of the second dose level in the single agent dose-escalation phase, demonstrating the drug to be safe and well tolerated in the treated patients. We expect to begin imminently our Phase 1b/2 studies for CM24, our monoclonal antibody drug candidate blocking CEACAM1, a novel immune checkpoint that supports tumor immune evasion and survival through multiple pathways. Our robust clinical development activities are supported by a strong balance sheet, as we raised $68.5 million in 2020, ended the year with $60.8 million in cash, and we believe we are well-funded to support our currently planned corporate initiatives until 2024."

"As part of the completion of our transformation to a corporate mission dedicated to developing first-in-class oncology therapies, we were honored to ring the Nasdaq stock market opening bell on January 5, 2021 to commemorate the successful evolution of our business and the Company’s name change to Purple Biotech. We are firmly committed to improving the lives of cancer patients globally, and look forward to executing on the opportunities that lie ahead of us in 2021 and beyond," concluded Mr. Israel.

Recent Corporate Highlights

CM24:

Expanded the planned Phase 1b/2 clinical trial evaluating CM24 in combination with nivolumab in advanced non-small cell lung cancer (NSCLC) with a new cohort that will evaluate CM24 in combination with both nivolumab and nab-paclitaxel (Abraxane) in patients with pancreatic cancer under a clinical collaboration agreement with Bristol Myers Squibb.
Advanced preparations to initiate the Phase 1b/2 study, which is expected to begin imminently.
Received notifications from the U.S. Patent and Trademark Office, European Patent Office and the Chinese Patent Office to grant the patent application entitled "Humanized antibodies against CEACAM1," covering the humanized antibodies capable of specific binding to human CEACAM1 molecules, pharmaceutical compositions and methods of their use in treating and diagnosing cancer and other condition.
Presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program positive results of a Phase 1 study consisting of a monotherapy dose escalating of CM24, as CM24 was found to be safe and well tolerated in all patients. In the study of efficacy evaluable patients (n=24), subjects were highly refractory to prior therapy, having received between two and eight prior therapies (with a median of four). Eight of the evaluable patients (33%) achieved stable disease, with most of these patients treated at the higher dose levels of 3mg/kg and 10mg/kg. Pharmacokinetic analysis revealed non-linearity, and modeling suggested that a higher dose level is required to achieve full saturation of CEACAM1 receptors.
NT-219:

Initiated and dosed the first patient in the Phase 1/2 clinical trial of NT219.
Presented new data supporting the mechanism of action of NT219 at the Epigenetics and Metabolism AACR (Free AACR Whitepaper) Special Virtual Conference by researchers at Tel-Aviv University.
Received notifications from the U.S. Patent and Trademark Office and the Chinese Patent Office to grant a patent application entitled "Combinations of IRS/STAT3 Dual Modulators and Anti-Cancer Agents for Treating Cancer," covering the various combinations of NT219 with multiple EGFR inhibitors, including cetuximab (Erbitux), which will be administrated in combination with NT219 for the treatment of recurrent or metastatic squamous cell carcinoma of the head as well as for neck cancer as part of our ongoing Phase 1/2 study, and osimertinib (TAGRISSO), a 3rd generation EGFR inhibitor approved in the U.S. for first-line treatment of EGFR-mutated non-small-cell lung carcinoma (NSCLC).

CONSENSI:

Growth of sales of Consensi in the U.S. has been slow primarily due to the COVID-19 environment, with minor sales of the drug in the second half of 2020. In addition, our distribution partner has not fulfilled all of its obligations as per the distribution agreement. We are currently evaluating a re-launch program that will be designed to boost sales and maximize the value of Consensi post-COVID-19. At this time, we are not able to provide revenue projections for the rest of 2021 and beyond.
Financial Results for the Six Months Ended December 31, 2020
Research and Development Expenses were $4.4 million, an increase of $3.4 million, or 342%, compared to $1.0 million in the same period of 2019. The increase was due to expenses related to the NT219 clinical trials initiated in 2020 and the preparation for the anticipated initiation of the CM24 clinical trials, including manufacturing costs.

Selling, General and Administrative (SG&A) Expenses were $4.1 million, compared to $2.8 million in the same period of 2019, an increase of $1.3 million. The increase was due mainly to a $0.9 million increase in expenses related to stock options granted to directors and employees in the second and third quarters of 2020 and a $0.3 million increase in director and officer insurance expenses.

Operating Loss was $8.3 million, an increase of $4.7 million, or 131%, compared to $3.6 million in the same period of 2019.

On a non-IFRS basis (as reconciled below), adjusted operating loss was $6.4 million, an increase of $3.6 million, compared to $2.8 million in the same period of 2019.

Net Loss for the second half of 2020 was $0.3 million, or $0.48 per diluted share, compared to a net loss of $3.3 million, or $1.70 per diluted share, in the second half of 2019. The decrease in net loss was due to $7.5 million in income from a change in the fair value of derivatives, partially offset by an increase of $4.5 million in operating expenses.

Financial Results for the Full Year Ended December 31, 2020
Revenues were $1.0 million for the year ended December 31, 2020, unchanged from the $1.0 million reported for the year ended December 31, 2019.

Research and Development Expenses were $7.5 million, an increase of $4.8 million, or 180%, compared to $2.7 million for the year ended December 31, 2019. The increase was due to expenses related to the NT219 clinical trials initiated in 2020 and the preparation for the anticipated initiation of the CM24 clinical trials, including manufacturing costs.

SG&A Expenses were $6.3 million, compared to $6.1 million for the year ended December 31, 2019.

Operating Loss was $12.6 million, an increase of $5.5 million, or 76%, compared to $7.2 million for the year ended December 31, 2019.

On a non-IFRS basis (as reconciled below), adjusted operating loss was $10.0 million, compared to $5.9 million, an increase of $4.1 million, for the year ended December 31, 2019.

Net Loss for the year ended December 31, 2020, was $28.1 million, or $2.44 per diluted share, compared to a net loss of $5.9 million, or $3.00 per diluted share, in the same period of 2019. The increase was due to $17.1 million increase in expenses on account of warrants mainly from a change in the fair value of derivatives and an increase of $4.8 million in R&D expenses.

As of December 31, 2020, Purple Biotech had cash and cash equivalents and short- and long-term deposits of $60.8 million, compared to $4.4 million at December 31, 2019. Purple Biotech believes that its cash position will provide sufficient resources for its currently anticipated ongoing needs until fiscal year 2024.

On March 2, 2021 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) reported that President and Chief Executive Officer Todd C. Brady, M.D., Ph.D. will participate in analyst-led fireside conversations at the H.C. Wainwright Global Life Sciences Conference and the Oppenheimer 31st Annual Healthcare Conference (Press release, Aldeyra Therapeutics, MAR 2, 2021, View Source [SID1234575963]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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H.C. Wainwright Global Life Sciences Conference
Date: Tuesday, March 9, 2021
Time: 7:00 a.m. ET
Oppenheimer 31st Annual Healthcare Conference
Date: Tuesday, March 16, 2021
Time: 9:20 a.m. ET
To view these events, please visit the Investor & Media page of the company’s website. After the presentations, the events will remain archived on the website for 90 days.

On March 2, 2021 Portage Biotech Inc. reported Condensed Consolidated Interim Financial Statements (U.S. Dollars) (Unaudited – See Notice to Reader dated November 30, 2020) (Press release, Portage Biotech, MAR 2, 2021, View Source [SID1234575982])

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

NOTE 1. NATURE OF OPERATIONS Portage Biotech Inc. (the "Company") is incorporated in the British Virgin Islands ("BVI") with its registered office located at FH Chambers, P.O. Box 4649, Road Town, Tortola, BVI. Its Toronto agent, Portage Services Ltd., is located at 6 Adelaide Street East, Suite 300, Toronto, Ontario, M5C 1H6, Canada. The Company is a reporting issuer with the Ontario Securities Commission on the Canadian Stock Exchange under the symbol PBT-U and U.S. Securities and Exchange Commission on the OTC market under the symbol PTGEF. The Company is engaged in the business of researching and developing pharmaceutical and biotechnology products through to clinical "proof of concept" with an initial focus on unmet clinical needs. Following proof of concept, the Company intends to seek to sell or license the products to large pharmaceutical companies for further development and commercialization.

On June 5, 2020, the Company effected a 100:1 reverse stock split. All share and per share information included in the condensed consolidated interim financial statements have been retroactively adjusted to reflect the impact of the reverse stock split. The shares of ordinary shares authorized remained at an unlimited number of ordinary shares without par value. The Company’s existing subsidiaries are in the pre-clinical stage, and as such no revenue has been generated from their operations. Liquidity and Capital Resources The Company has incurred substantial operating losses since inception and expects to continue to incur significant operating losses for the foreseeable future and may never become profitable. The losses result primarily from its conduct of research and development activities.

As of September 30, 2020, the Company had cash balances totaling approximately $4.4 million and working capital of approximately $0.025 million (approximately $4.3 million adjusted for accrued equity issuable and warrant liability settleable on a non-cash basis), as compared to approximately $3.2 million and approximately $1.2 million, respectively, as of March 31, 2020. On June 16, 2020, in a private placement, the Company issued 698,145 restricted ordinary shares for gross proceeds of $6.98 million. The Company incurred costs of approximately $0.25 million in connection with the offering, which were recorded as a reduction of the offering proceeds. The Company historically has funded its operations principally from proceeds from issuances of equity and debt securities. The Company will require significant additional capital to make the investments it needs to execute its longerterm business plan. The Company’s ability to successfully raise sufficient funds through the sale of debt or equity securities when needed is subject to many risks and uncertainties and, future equity issuances would result in dilution to existing stockholders and any future debt securities may contain covenants that limit the Company’s operations or ability to enter into certain transactions. The Company’s believes its current cash will be sufficient to fund operations for at least 12 months from the date of issuance of the financial statements contained herein. However, the Company believes it will need to raise additional funding through strategic relationships, public or private equity or debt financings, grants or other arrangements in order to advance the Company’s existing and new product candidates through development and regulatory processes. If such funding is not available or not available on terms acceptable to the Company, the Company’s current development plan and plans for expansion of its general and administrative infrastructure may be modified or even curtailed. F-5 Portage Biotech Inc. Notes to Condensed Consolidated Interim Financial Statements (U.S. Dollars) (Unaudited – See Notice to Reader dated November 30, 2020)

NOTE 1. NATURE OF OPERATIONS (Cont’d) COVID-19 Effect Beginning in early March 2020, the COVID-19 pandemic and the measures imposed to contain this pandemic have disrupted and are expected to continue to impact the Company’s business operations. The magnitude of the impact of the COVID-19 pandemic on the Company’s productivity, results of operations and financial position, and its disruption to the Company’s business and clinical programs and timelines, will depend, in part, on the length and severity of these restrictions and on the Company’s ability to conduct business in the ordinary course.

NOTE 2. BASIS OF PRESENTATION Statement of Compliance and Basis of Presentation These condensed consolidated interim financial statements have been prepared in accordance with the International Financial Reporting Standards ("IFRS") issued by the International Accounting Standards Board ("IASB"), IAS 34 Interim Financial Reporting and interpretations of the International Financial Reporting Interpretations Committee.

These condensed consolidated interim financial statements do not include all of the information required for full annual financial statements and should be read in conjunction with the audited consolidated financial statements of the Company for the year ended March 31, 2020. These condensed consolidated interim financial statements have been prepared on an historical cost basis except for items disclosed herein at fair value. In addition, these condensed consolidated interim financial statements have been prepared using the accrual basis of accounting, except for cash flow information.

The Company has only one material operating segment. These condensed consolidated interim financial statements were approved and authorized for issue by the Audit Committee and Board of Directors on November 30, 2020.

Consolidation The condensed consolidated interim financial statements include the accounts of the Company and,
(a) Portage Services Ltd., a wholly owned subsidiary incorporated in Ontario on January 31, 2011.
(b) Portage Pharmaceuticals Ltd. ("PPL") a wholly owned subsidiary acquired in a merger on July 23, 2013, incorporated in the British Virgin Islands.
(c) EyGen Limited, ("EyGen"), a wholly owned subsidiary of PPL, incorporated on September 20, 2016, in the British Virgin Islands.
(d) SalvaRx Limited ("SalvaRx"), a wholly owned subsidiary, incorporated on May 6, 2015 in the British Virgin Islands.
(e) Portage Glasgow Ltd ("PGL"), a 65% subsidiary of PPL, incorporated in Glasgow, Scotland.
(f) iOx Therapeutics Ltd ("iOx"), a United Kingdom based immune-oncology company, a 60.49% subsidiary, incorporated in the United Kingdom on February 10, 2015.
(g) Saugatuck, a 70% owned subsidiary incorporated in the British Virgin Islands. F-6 Portage Biotech Inc. Notes to Condensed Consolidated Interim Financial Statements (U.S. Dollars) (Unaudited – See Notice to Reader dated November 30, 2020)

NOTE 2. BASIS OF PRESENTATION (Cont’d) Consolidation (Cont’d)
(h) Portage Developmental Services, a 100% owned subsidiary incorporated in Delaware. All inter-company balances and transactions have been eliminated on consolidation. Non-controlling interest in the equity of a subsidiary is accounted for and reported as a component of stockholders’ equity. Non-controlling interests represent the 39.51% shareholder ownership interest in iOx and the 30% shareholder ownership interest in Saugatuck, and the 35% shareholder ownership interest in PGL, which are consolidated by the Company. Functional and Presentation Currency The Company’s functional and presentation currency is U.S. Dollar. Use of Estimates and Judgments The preparation of the condensed consolidated interim financial statements in conformity with IFRS requires management to make judgments, estimates and assumptions that affect the application of accounting policies and the reported amounts of assets, liabilities, income and expenses. Actual results may differ from these estimates. Estimates and underlying assumptions are reviewed on an ongoing basis. Revisions to accounting estimates are recognized in the period in which the estimates are revised and in any future periods affected. Significant areas where estimates are made include valuation of financial instruments, research and development costs, fair value used for acquisition and measurement of share-based compensation. Significant areas where critical judgments are applied include assessment of impairment of investments and goodwill and the determination of the accounting acquirer and acquiree in the business combination accounting. Reclassifications Certain prior year amounts have been reclassified for consistency with the current year presentation. These reclassifications had no effect on the reported results of operations.

NOTE 3. SIGNIFICANT ACCOUNTING POLICIES The accounting policies are set out in Note 3 to the fiscal 2020 audited consolidated financial statements. These policies have been applied consistently to all periods presented in these condensed consolidated interim financial statements. New accounting standards, interpretations and amendments Standards issued but not yet effective up to the date of issuance of the Company’s condensed consolidated interim financial statements are listed below. This listing is of standards and interpretations issued, which the Company reasonably expects to be applicable at a future date. The Company intends to adopt those standards when they become effective.