On March 12, 2020 AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, reported that the European Commission (EC) has approved VENCLYXTO (venetoclax) in combination with obinutuzumab for the treatment of adult patients with chronic lymphocytic leukemia (CLL) who were previously untreated (Press release, AbbVie, MAR 12, 2020, View Source [SID1234555505]). The approval is valid in all 27 member states of the EU, as well as Iceland, Liechtenstein, Norway and the United Kingdom.

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"This approval underscores the growing utility of VENCLYXTO in treating CLL and demonstrates its clinical benefit as a chemotherapy-free combination therapy option for CLL patients in Europe who have not yet been treated," said Neil Gallagher, M.D., Ph.D., chief medical officer and vice president of development. "We look forward to bringing VENCLYXTO to even more patients who can potentially benefit from achieving a deep response and sustained progression free survival, with the added benefit of a finite treatment duration."

This is the third approval for VENCLYXTO, a first-in-class B-cell lymphoma-2 (BCL-2) inhibitor. BCL-2 is a protein that prevents cancer cells from undergoing apoptosis, the process that leads to the natural death or self-destruction of cancer cells. VENCLYXTO is also approved in combination with rituximab for the treatment of adult patients with CLL who have received at least one prior therapy, and as a monotherapy for the treatment of CLL in the presence or absence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor.

This most recent approval is based on results from the Phase 3 CLL14 clinical trial primary analysis (median follow up of 28 months), which demonstrated superior progression-free survival (PFS; the time on treatment without disease progression or death) as assessed by investigators in patients treated with VENCLYXTO plus obinutuzumab compared to patients who received a standard of care chemotherapy regimen of chlorambucil plus obinutuzumab (hazard ratio 0.35; 95% CI (0.23,0.53), p<0.0001, medians not yet reached). At an updated CLL14 efficacy analysis (median follow-up of 40 months), the median PFS had not been reached in the VENCLYXTO + obinutuzumab arm and was 35.6 months [95% CI: 33.7,40.7] in the obinutuzumab + chlorambucil arm (hazard ratio 0.31; 95% CI: 0.22, 0.44). The 36-month PFS estimate in the venetoclax plus obinutuzumab arm was 81.9% [95% CI: 76.5, 87.3] and in the obinutuzumab plus chlorambucil arm was 49.5% [95% CI: 42.4, 56.6]. Additionally, after completing one year of treatment, patients treated with the VENCLYXTO combination experienced deep response as measured by higher rates of undetectable minimal residual disease (MRD) or complete response (CR) as compared to patients receiving a standard of care regimen.1,5

In the trial, adverse events (AEs) were consistent with the known safety profiles of venetoclax and obinutuzumab alone. At least one AE of any grade occurred in 94.3% of patients in the venetoclax combination arm. The most common Grade 3/4 AEs were neutropenia and infections. Tumor lysis syndrome (TLS) was reported in three patients in the venetoclax plus obinutuzumab group (all during treatment with obinutuzumab and before venetoclax).1

"CLL is the most common of the nearly 95,000 new cases of leukemia in Europe each year, and chemotherapy is often the first line of treatment," said Michael Hallek, M.D., lead investigator of the CLL14 study, director of the Department of Internal Medicine and Center of Integrated Oncology at the University Hospital Cologne in Germany, and head of the German CLL Study Group. "Having the option to utilize a first-line, chemotherapy-free treatment combination that can produce a deep response, thus allowing patients to stop treatment, will change the way we treat CLL and have a significant impact on patients."

In January 2020, AbbVie announced that the European Committee for Medicinal Products for Human Use (CHMP) granted a positive opinion for the Marketing Authorization Application for VENCLYXTO plus obinutuzumab for the treatment of patients with previously untreated CLL.

VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.

About Chronic Lymphocytic Leukemia
CLL is a slow-growing form of leukemia, or blood cancer, in which too many immature lymphocytes (a type of white blood cell) are found predominantly in the blood and bone marrow. In 2018, approximately 95,000 new cases of leukemia were diagnosed in Europe.2 CLL is the most common form of leukemia in the Western Hemisphere, accounting for approximately one third of new leukemia diagnoses.3,4

About the CLL14 Trial
The randomized, multicenter, open-label, actively controlled Phase 3 CLL14 trial, which was conducted in close collaboration with the German CLL Study Group (DCLLSG), evaluated the efficacy and safety of a combined regimen of VENCLYXTO and obinutuzumab (n=216) versus obinutuzumab and chlorambucil (n=216) in patients with previously-untreated CLL and coexisting medical conditions (total Cumulative Illness Rating Scale [CIRS] score >6 or creatinine clearance <70 mL/min). The therapies were administered for a fixed duration of 12 cycles for VENCLYXTO in combination with six cycles of obinutuzumab. Cycles were comprised of 28 days. The trial enrolled 432 patients, all of whom were diagnosed according to the International Workshop on Chronic Lymphocytic Leukemia (iwCLL) criteria and were previously untreated. The primary efficacy outcome was PFS as assessed by the investigator.1 Key secondary endpoints were MRD-negativity in peripheral blood and bone marrow, and overall and complete response rates.1

About VENCLYXTO (venetoclax tablets)
VENCLYXTO is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers and other cancerous tumors, BCL-2 builds up and prevents cancer cells from undergoing their natural death or self-destruction process, which is called apoptosis. VENCLYXTO targets the BCL-2 protein and works to restore the process of apoptosis.

VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood and other cancers.

VENCLYXTO is approved in more than 50 countries, including the U.S. AbbVie and Roche are currently working with regulatory agencies around the world to bring this medicine to additional eligible patients in need.

VENCLYXTO (venetoclax) EU Indication and Summary of Important Safety Information5

Indication
Venclyxto in combination with obinutuzumab is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).

Venclyxto in combination with rituximab is indicated for the treatment of adult patients with CLL who have received at least one prior therapy.

Venclyxto monotherapy is indicated for the treatment of CLL:

in the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B?cell receptor pathway inhibitor, or
in the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B?cell receptor pathway inhibitor.
Contraindications
Hypersensitivity to the active substance or to any of the excipients is contraindicated. Concomitant use of strong CYP3A inhibitors at initiation and during the dose-titration phase due to increased risk for tumor lysis syndrome (TLS). Concomitant use of preparations containing St. John’s wort as VENCLYXTO efficacy may be reduced.

Special Warnings & Precautions for Use
Tumour lysis syndrome (TLS), including fatal events, has occurred in patients with previously treated CLL with high tumour burden when treated with VENCLYXTO. VENCLYXTO poses a risk for TLS in the initial 5-week dose-titration phase. Changes in electrolytes consistent with TLS that require prompt management can occur as early as 6 to 8 hours following the first dose of VENCLYXTO and at each dose increase. Patients should be assessed for risk and should receive appropriate prophylaxis for TLS. Blood chemistries should be monitored, and abnormalities managed promptly. More intensive measures (including IV hydration, frequent monitoring and hospitalization) should be employed as overall risk increases.

Neutropenia (grade 3 or 4) has been reported and complete blood counts should be monitored throughout the treatment period. Serious infections including events of sepsis with fatal outcome have been reported. Patients require monitoring of signs and symptoms of infection and prompt treatment. Supportive measures including antimicrobials for any signs of infection should be considered.

Live vaccines should not be administered during treatment or thereafter until B-cell recovery.

Drug Interactions
CYP3A inhibitors may increase VENCLYXTO plasma concentrations. At initiation and dose-titration phase: Strong CYP3A inhibitors are contraindicated due to increased risk for TLS and moderate CYP3A inhibitors should be avoided. If moderate CYP3A inhibitors must be used, physicians should refer to the SmPC for dose adjustment recommendations. At steady daily dose: If moderate or strong CYP3A inhibitors must be used, physicians should refer to the SmPC for dose adjustment recommendations.

CYP3A4 inducers may decrease VENCLYXTO plasma concentrations.

Avoid coadministration with strong or moderate CYP3A inducers. These agents may decrease venetoclax plasma concentrations.

Co-administration of bile acid sequestrants with VENCLYXTO is not recommended as this may reduce the absorption of VENCLYXTO.

Adverse Reactions
The most commonly occurring adverse reactions (≥20%) of any grade in patients receiving venetoclax in the combination studies with obinutuzumab or rituximab were neutropenia, diarrhoea, and upper respiratory tract infection. In the monotherapy studies, the most common adverse reactions were neutropenia/neutrophil count decreased, diarrhoea, nausea, anaemia, fatigue, and upper respiratory tract infection.

The most frequently reported serious adverse reactions (≥2%) in patients receiving venetoclax in combination with obinutuzumab or rituximab were pneumonia, sepsis febrile neutropenia, and TLS. In the monotherapy studies, the most frequently reported serious adverse reactions (≥2%) were pneumonia and febrile neutropenia.

Discontinuations due to adverse reactions occurred in 16% of patients treated with venetoclax in combination with obinutuzumab or rituximab in the CLL14 and MURANO studies, respectively. In the monotherapy studies with venetoclax, 11% of patients discontinued due to adverse reactions.

Dosage reductions due to adverse reactions occurred in 21% of patients treated with the combination of venetoclax and obinutuzumab in the CLL14 study, in 15% of patients treated with the combination of venetoclax and rituximab in the MURANO study and 14% of patients treated with venetoclax in the monotherapy studies.

Specific Populations
Patients with reduced renal function (CrCl <80 mL/min) may require more intensive prophylaxis and monitoring to reduce the risk of TLS. Safety in patients with severe renal impairment (CrCl <30 mL/min) or on dialysis has not been established, and a recommended dose for these patients has not been determined. VENCLYXTO should be administered to patients with severe renal impairment only if the benefit outweighs the risk and patients should be monitored closely for signs of toxicity due to increased risk of TLS.

For patients with severe (Child-Pugh C) hepatic impairment, a dose reduction of at least 50% throughout treatment is recommended.

VENCLYXTO may cause embryo-fetal harm when administered to a pregnant woman. Advise females of reproductive potential to avoid pregnancy during treatment. Advise nursing women to discontinue breastfeeding during treatment.

On March 12, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate company QED Therapeutics reported that patients have been dosed in separate Phase 3 and Phase 2 clinical trials of infigratinib in cancer indications (Press release, BridgeBio, MAR 12, 2020, View Source [SID1234556926]).

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The Phase 3 PROOF 302 trial sponsored by QED is studying infigratinib for the adjuvant (post-surgery) treatment of invasive urothelial carcinoma. A second, investigator-initiated trial, led by Sameek Roychowdhury, M.D., Ph.D., of The Ohio State University (OSU) Comprehensive Cancer Center, is studying infigratinib for the treatment of advanced and metastatic solid tumors with confirmed FGFR gene fusions/translocations or other FGFR alterations.

In the PROOF 302 trial, investigators are enrolling subjects with invasive urothelial cancer harboring susceptible FGFR3 genetic alterations who are at high risk of recurrence following surgical resection. Subjects will be randomized (1:1) to receive once daily oral infigratinib or placebo. The primary outcome is disease-free survival, and secondary outcomes include metastasis-free survival, overall survival, and safety and tolerability measures.

"Many patients with invasive urothelial carcinoma will have their cancer recur within two years after surgery," said PROOF 302 trial lead Sumanta Pal, M.D., professor of medical oncology and therapeutics research at City of Hope Comprehensive Cancer Center. "Correspondingly, I believe there are many patients who could benefit from an oral, post-surgery treatment option that targets FGFR3 alterations, the genetic driver of many urothelial carcinomas."

The Phase 2 study at OSU and selected sites within the Oncology Research Information Exchange Network (ORIEN) will evaluate the efficacy of infigratinib in patients who have advanced or metastatic solid tumors that are positive for FGFR1-3 gene fusions/translocations or other FGFR alterations. The open-label study will assess overall response rate as the primary outcome. Secondary outcomes include progression-free survival, best overall response, disease control rate, overall survival and measures of safety and tolerability.

"Increasingly, oncologists are learning to classify their patients’ cancers based on genetic mutations, going beyond the origin of the tumor," noted Dr. Roychowdhury. "Given the activity we have seen with infigratinib in FGFR2-fusion-driven bile duct cancers and FGFR3-altered urothelial carcinoma, our hope is that infigratinib will demonstrate similar activity in additional cancers that appear to be driven by alterations in FGFR. There appear to be multiple FGFR alterations that can drive cancer growth—and we hope to see these patients benefit too."

For additional information on the PROOF 302 trial, including eligibility, patients should ask their physician, visit clinicaltrials.gov, or email [email protected].

For additional information on the Phase 2 trial in metastatic solid tumors with FGFR gene alterations, including eligibility, patients should ask their physician, visit clinicaltrials.gov, or email [email protected].

On March 12, 2020 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTOR, reported financial results for the fourth quarter and year ended December 31, 2019 (Press release, Selecta Biosciences, MAR 12, 2020, View Source [SID1234555462]).

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"2019 was a pivotal year for Selecta. We made substantial clinical advancements, as we completed enrollment in the head-to-head COMPARE trial for our lead product candidate, SEL-212, in chronic refractory gout, and advanced our gene therapy program through strategic partnerships with AskBio. We also strengthened our organization with the addition of several key members to the executive team and welcomed a new Chairman of our Board of Directors, and completed a $70 million financing," said Carsten Brunn, Ph.D., President and CEO of Selecta. "In the coming year, we look forward to reporting topline data from the COMPARE study, commencing the Phase 3 clinical program of SEL-212, and advancing our gene therapy program into the clinic in partnership with AskBio."

Recent Highlights and Anticipated Upcoming Milestones:

Topline Results from COMPARE Clinical Trial Expected in the Third Quarter of 2020: In December 2019, we announced the completion of enrollment in the head-to-head COMPARE study of Selecta’s lead product candidate, SEL-212 (ImmTOR + pegadricase), vs. pegloticase. Topline data from this trial is expected in the third quarter of 2020. The trial is evaluating a once-monthly dose of SEL-212 compared to biweekly doses of pegloticase, with the primary endpoint of the maintenance of serum uric acid (SUA) levels of <6mg/dL at three and six months.

Meeting with FDA Provides Clarity on Phase 3 Clinical Program of SEL-212: Selecta held a meeting with the U.S. Food and Drug Administration (FDA) in January 2020 to inform the design of the planned Phase 3 clinical program. Selecta plans to commence its Phase 3 clinical program of SEL-212 against placebo in the second half of 2020.

Gene Therapy Program in the Clinic by the End of 2020: In August 2019, Selecta announced a strategic partnership with Asklepios BioPharmaceutical, Inc. (AskBio), to jointly develop, manufacture, and commercialize a broad portfolio of next-generation AAV gene therapies. This

partnership will leverage the unique proprietary technology platforms of both companies with a human proof of concept trial to validate this portfolio of products and their potential for re-dosing in patients, which could represent a significant advancement in the gene therapy field. Selecta and AskBio anticipate entering the clinic by the end of 2020. Additionally, the Company intends to advance its proprietary program in ornithine transcarbamylase (OTC) deficiency.

Broadened Strategic Partnership with AskBio: In December 2019, Selecta and AskBio jointly announced that the companies entered into a license agreement under which AskBio exercised its option to exclusively license rights to develop and commercialize Selecta’s immune tolerance platform, ImmTOR, for use in adeno-associated virus (AAV) gene therapy for the treatment of Pompe disease. Affecting 5,000-10,000 people worldwide, Pompe disease is a rare, genetic, lysosomal storage disease characterized by the abnormal buildup of a sugar molecule called glycogen inside cells. Under the terms of the agreement, Selecta received upfront payments of $7 million and is eligible to receive milestone payments of $237 million plus royalties on product sales.

Raised $70 Million in a Private Placement: In December 2019, Selecta announced the closing of a transaction to sell securities in a private placement with institutional investors and certain members of the Company’s Board of Directors, resulting in gross proceeds of approximately $70 million.

Strengthened Board of Directors: In November 2019, Selecta announced the appointment of Carrie S. Cox to the position of Chairman of the Board of Directors. A renowned industry leader and successful biopharmaceutical executive, Ms. Cox has served on multiple Boards, and has held the position of Chair, for several biopharmaceutical companies.

Fourth Quarter and Full Year 2019 Financial Results:

Cash Position: Selecta had $91.6 million in cash, cash equivalents, and restricted cash as of December 31, 2019, which compares to cash, cash equivalents, restricted cash, and short-term investments of $35.9 million as of September 30, 2019. Selecta believes its available cash, cash equivalents, and restricted cash will be sufficient to meet its operating requirements into the first quarter of 2021.

Net cash used in operating activities was $12.9 million and $51.4 million for the fourth quarter and fiscal year 2019, respectively, as compared to $12.7 million and $59.2 million for the same periods in 2018.

Research and Development Expenses: Research and development expenses for the fourth quarter and fiscal year 2019 were $15.2 million and $42.7 million, respectively, which compares with $10.3 million and $47.7 million for the same periods in 2018. The quarterly increase reflects additional costs incurred specific to our Phase 2 head-to-head (COMPARE) clinical trial of SEL-212, for which we completed enrollment in December 2019. The decrease year over year reflects reduced costs in 2019 due to the completion of prior programs in 2018, combined with reduced salaries and benefits resulting from the headcount reduction in early 2019. The cost reductions were offset by an overall increase in costs incurred on our lead product candidate, SEL-212.

General and Administrative Expenses: General and administrative expenses for the fourth quarter and fiscal year 2019 were $4.1 million and $16.4 million, respectively, which compares with $5.1 million and $18.2 million for the same periods in 2018. The decrease is the result of lower salaries and stock compensation expense resulting from reduced headcount at the end of 2018, combined with reduced patent and professional fees.

Net Loss: For the fourth quarter and fiscal year 2019, Selecta reported a net loss of $14.9 million, or $0.28 per share and $55.4 million, or $1.22 per share, compared to a net loss of $14.7 million, or $0.65 per share, and $65.3 million, or $2.92 per share, for the same periods in 2018.

Conference Call and Webcast Reminder:
Selecta management will host a conference call at 8:30 AM ET today to provide a corporate update and review the company’s fourth quarter 2019 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10138603. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.

On March 12, 2020 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company applying synthetic biology to beneficial microbes to develop novel, living medicines, reported its financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Synlogic, MAR 12, 2020, View Source [SID1234555490]).

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"We have built key Synthetic Biotic platform capabilities in synthetic biology, manufacturing and development to enable the efficient generation of therapeutics that have the potential to address unmet medical need in a range of indications from rare metabolic diseases to cancer," said Aoife Brennan, M.B., Ch.B., Synlogic’s president and chief executive officer. "Building on our experience in our PKU program we have made steady progress on new programs in enteric hyperoxaluria and maple syrup urine disease and we look forward to providing more detail on these initiatives as well as the underlying engine that powers our pipeline as year progresses."

2020 Priorities

Pipeline

Initiation of a Phase 2 clinical trial to evaluate a solid formulation of SYNB1618 in patients with phenylketonuria (PKU) expected in the first half of 2020. The trial is designed to evaluate safety and tolerability of a solid formulation of SYNB1618 as well as its potential to lower blood phenylalanine (Phe) levels in PKU patients. In addition, the study is expected to provide valuable information to validate predictive pharmacodynamic and preclinical modeling.
Evaluation of data expected in 2020 from the monotherapy arm of the Phase 1 clinical study of SYNB1891 in patients with advanced solid tumors or lymphoma. SYNB1891 is an intra-tumorally administered Synthetic Biotic medicine engineered to produce cyclic di-AMP, an agonist of the STING pathway, that is designed to serve as a dual innate activator of the immune system as a potential treatment for solid tumors or lymphoma. SYNB1891 is being evaluated as a monotherapy in an ongoing Phase 1 open-label, multicenter, dose escalation clinical trial (NCT04167137) in patients with advanced solid tumors or lymphoma. Synlogic expects to have data from the monotherapy arm of this study in 2020. After establishing a maximum tolerated dose for SYNB1891 as monotherapy, Synlogic expects to initiate a second arm of the trial in which subjects will receive escalating dose levels of SYNB1891 in combination with a fixed dose of the checkpoint inhibitor, atezolizumab (Tecentriq), to establish a recommended dose for the combination regimen.
Continued development of patient and commercialization-appropriate presentations of SYNB1618. Synlogic has developed and manufactured a solid formulation of its Synthetic Biotic SYNB1618 suitable for future clinical trials and continues to evaluate and develop presentations such as enteric-coated capsules and pressed tablets for eventual commercialization.
Advancement of new Synthetic Biotic programs in metabolic diseases with high unmet medical need. Synlogic is conducting preclinical studies of Synthetic Biotic medicines to treat enteric hyperoxaluria (HOX), an acquired metabolic disorder in which patients develop recurrent kidney stones due to elevated urinary oxalate levels and are at an increased risk of kidney failure. In addition, Synlogic is also developing Synthetic Biotic medicines for the treatment of maple syrup urine disease (MSUD), a rare inherited metabolic disease caused by defective enzymes that metabolize branched chain amino acids (BCAAs) which are components of protein. Elevated blood levels of BCAAs can lead to can lead to seizures, coma, and death. There are currently no approved therapies to treat these disorders.
Presentation and publication of data at major scientific and medical meetings. Synlogic is committed to publishing and presenting data that demonstrate the breadth of Synlogic’s Synthetic Biotic platform.
Corporate

Continued strengthening of Synlogic’s leadership. In January 2020, Synlogic announced the appointment of Michael Burgess, M.B., Ch.B., Ph.D., President, Research & Development, at Turnstone Biologics, to its board of directors. Dr. Burgess is a physician scientist who brings extensive experience in translational drug development from leadership roles at several large Pharma companies including Roche, Bristol-Myers Squibb and Lilly.
Continued exploration of additional strategic collaborations. Synlogic expects to continue to develop strategic collaborations to expand the breadth of its Synthetic Biotic pipeline in therapeutic areas that have high biology risk.
Fourth Quarter 2019 Financial Results

As of December 31, 2019, Synlogic had cash, cash equivalents, and short- and long-term investments of $127.1 million.

For the three months ended December 31, 2019, Synlogic reported a consolidated net loss of $12.8 million, or $0.37 per share, compared to a net loss of $11.9 million, or $0.47 per share, for the corresponding period in 2018.

Research and development expenses were $11.3 million for the three months ended December 31, 2019 compared to $8.9 million for the corresponding period in 2018. The increase in expenses was primarily due to use of synthetic biology services provided under Synlogic’s collaboration with Ginkgo and increased clinical activities, including the SYNB1618 bridging study and initiation of the SYNB1891 Phase 1 clinical study.

General and administrative expenses for the three months ended December 31, 2019 were $3.5 million compared to $4.0 million for the corresponding period in 2018.

Revenue was $1.2 million for the three months ended December 31, 2019 compared to $0.1 million for the three months ended December 31, 2018. Revenue is associated with services performed under the Synlogic’s collaboration with AbbVie to develop a Synthetic Biotic medicine for the treatment of inflammatory bowel disease (IBD). The increase in revenue for the fourth quarter of 2019 compared to the same period in 2018 was a result of revised estimates of time and effort required to reach certain milestones in the collaboration.

Full Year 2019 Financial Results

For the year ended December 31, 2019, consolidated net loss was $51.4 million, or $1.70 per share, compared to a consolidated net loss of $48.4 million, or $2.03 per share, for the year ended December 31, 2018. Revenues were $2.2 million for the year ended December 31, 2019, compared to $2.5 million for the same period in 2018. Total operating expenses were $56.6 million for the year ended December 31, 2019, compared to $53.8 million for the same period in 2018.

Conference Call & Webcast Information

Synlogic will host a conference call and live webcast today at 5:00 p.m. ET today, Thursday, March 12, 2020. To access the live webcast, please visit the "Event Calendar" page within the Investors and Media section of the Synlogic website. Alternatively, investors may listen to the call by dialing +1 (844) 815-2882 from locations in the United States or +1 (213) 660-0926 from outside the United States. The conference ID number is 4089293. For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors and Media section of the Synlogic website.

On March 12, 2020 Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx plant cell-based protein expression system, reported financial results for the fourth quarter and full year ended December 31, 2019, and provided a business update on recent corporate and clinical developments (Press release, Protalix, MAR 12, 2020, View Source [SID1234555506]). The Company will discuss the clinical, corporate and financial highlights on a conference call and live webcast, scheduled for Thursday, March 12th, 2020 at 8:30 am EDT.

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"2019 was a pivotal year for Protalix, as we successfully expanded on our strong foundation and entered into a new phase of development as a world-class recombinant therapeutic company," said Dror Bashan, Protalix’s President and Chief Executive Officer. "With this continued forward momentum toward commercialization of our Fabry program, we believe Protalix is positioned for both near- and long-term success," he continued.

"We are increasingly enthusiastic about our PRX-102 asset now that we have three ongoing, fully-enrolled Phase III clinical trials of PRX-102, and as we anticipate our BLA submission to the U.S. Food and Drug Administration under the Accelerated Approval pathway next quarter," added Mr. Bashan. "Furthermore, we anticipate the final results of our BRIDGE and BRIGHT trials in the first and second halves of 2020, respectively, which will further support our portfolio of data regarding PRX-102. Protalix is firing on all cylinders right now, and the energy among the entire staff reached a new pinnacle in 2019."

"We anticipate 2020 to be a banner year for Protalix as we increase our focus on advancing our clinical pipeline, expanding sales in Brazil of Elelyso, our enzyme replacement therapy for the treatment of Gaucher disease, and leveraging commercial opportunities to expand our global footprint in the treatment of Fabry disease," he concluded.

Conference Call and Webcast Information

The Company will host a conference call on Thursday, March 12, 2020, at 8:30 am, Eastern Daylight Time, to review the clinical, corporate and financial highlights. To participate in the conference call, please dial the following numbers prior to the start of the call:

Domestic: 877-423-9813
International: 201-689-8573
Conference ID: 13699970
Webcast: View Source

The conference call will also be broadcast live and will be available for replay for two weeks in the Events Calendar of the Investors section of the Company’s website, www.protalix.com. Please access the Company’s website at least 15 minutes ahead of the conference call in order to register, download and install any necessary audio software.

2019 Full-Year and Recent Business Highlights

Clinical Advancements

·The Company and its collaboration partner, Chiesi Farmaceutici S.p.A., or Chiesi, plan the submission of a BLA for PRX-102 via the FDA’s Accelerated Approval pathway in the second quarter of 2020, based on data from the completed Phase I/II clinical trial of PRX-102 for the treatment of Fabry disease and the ongoing Phase III BRIDGE clinical trial.
Results from the Company’s Phase I/II clinical trial of PRX-102 were published in an article in the May 2019 edition of the Journal of Inherited Metabolic Disease.

·The Company announced positive 12-month interim on-treatment data from the BRIDGE study. The interim data demonstrate a mean improvement in kidney function in both male and female patients when switched from agalsidase alfa (Replagal) to PRX-102. The data will be included in the anticipated BLA filing to help to support the application.

·The Company and Chiesi announced the completion of enrollment in the Phase III BALANCE clinical trial. The head-to-head BALANCE study is designed to evaluate the safety and efficacy of PRX-102 compared to agalsidase beta (Fabrazyme) on renal function in Fabry patients with progressing kidney disease previously treated with agalsidase beta. To date, more than 66 patients are being treated in the Company’s various extension studies after opting to continue treatment with PRX-102 after completion of an initial study.

·Enrollment was completed in the Phase III BRIGHT clinical trial of PRX- 102, via intravenous infusions of 2 mg/kg administered every 4 weeks. Preliminary pharmacokinetic (PK) data showed PRX-102 to be well-tolerated; and infusion of 2 mg/kg PRX-102 administered every 4 weeks resulted in the presence of continuous active enzyme throughout the entire infusion interval. Infusions every 2 weeks is the current standard of care for the treatment of Fabry disease.

Corporate & Financial Developments

·The Company yesterday successfully secured securities purchase agreements to raise proceeds equal to $43.7 million through a private financing with a number of leading Israeli and U.S.- based investors, including Psagot Investment House, More Investment House, Highbridge Capital, UBS O’Connor, Rosalind Capital, and Alrov Properties and Lodging, among others. Rosario Capital and Houlihan Lokey served as financial advisors in the private placement.

·In December 2019, the Company held a special meeting of stockholders to propose the following two critical financial amendments, which stockholders ultimately approved:
oA reverse stock split (1-for-10); and
oA reduction in the total number of shares of the Company’s common stock that the Company is authorized to issue from 350 million to 120 million shares.

·In December 2019, the Company enhanced its Board of Directors with addition of two accomplished biopharmaceutical executives, Pol F. Boudes, M.D., and Gwen A. Melincoff.

·In August 2019, the Company’s Board of Directors unanimously elected Zeev Bronfeld, an independent director, as Chairman of the Board.

·In July 2019, the Company appointed Eyal Rubin as Senior Vice President and Chief Financial Officer.

·In May 2019, the Company appointed Dror Bashan as President and Chief Executive Officer.

Regulatory Advancements

·In February 2020, Protalix and Chiesi announced the receipt of an agreement letter from the FDA for the Initial Pediatric Study Plan (iPSP) for PRX-102 for the treatment of Fabry disease, outlining an agreed-upon approach to address the needs of pediatric Fabry patients.

Financial Results

For the year ended December 31, 2019, compared to the year ended December 31, 2018

·The Company recorded revenues from selling goods of $15.9 million for the year ended December 31, 2019, an increase of $6.9 million, or 77%, compared to revenues of $9.0 million for the same period of 2018. The increase is primarily due to higher sales of Elelyso in Brazil as well as an increase in sales of drug substance to Pfizer.

·Research and development expenses, net, were $44.6 million for the year ended December 31, 2019, an increase of $11.3 million, or 34%, compared to $33.3 million for the same period of 2018. The increase resulted primarily from an increase of $9.1 million in clinical trial related costs as well as a decrease of $2.1 million in grants received from the Israeli Innovation Authority.

·Selling, general and administrative expenses were $9.9 million for the year ended December 31, 2019, a decrease of $1.0 million, or 9%, compared to $10.9 million for the same period of 2018. The decrease resulted primarily from costs related to the Company’s U.S. Exclusive License and Supply Agreement that the Company entered into in 2018, which were not incurred in 2019.

·Net loss was $18.3 million for the year ended December 31, 2019, or $1.23 per share, basic and diluted, compared to a net loss of $26.5 million, or $1.80 per share, basic and diluted, for the same period of 2018.

·At December 31, 2019, the Company had $17.8 million in cash and cash equivalents.

For the three months ended December 31, 2019, compared to the three months ended December 31, 2018

·The Company recorded revenues from selling goods of $3.8 million during the three-month period ended December 31, 2019, an increase of $2.0 million, or 111%, compared to revenues of $1.8 million for the same period of 2018. The increase is primarily due to higher sales of drug substance to Pfizer Inc. as well as higher sales of Elelyso in Brazil.

·Research and development expenses, net, were $9.6 million for the three-month period ended each of December 31, 2019 and December 31, 2018.

·Selling, general and administrative expenses were $3.0 million for the three-month period ended December 31, 2019, an increase of $0.8 million, or 36%, compared to $2.2 million for the same period in 2018. The increase is primarily due to costs related to the efforts to evaluate and pursue strategic alternatives, business development advisory fees, and legal fees and costs related to replacement of the Chief Executive Officer.

·Net profit for the three months ended December 31, 2019 was $0.3 million, or $0.02 per share, basic and diluted, compared to a net loss of $5.4 million, or $0.40 per share, basic and diluted, for the three months ended December 31, 2018.