On March 12, 2020 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to developing and commercializing next-generation medicines to improve the lives of patients with immune-mediated diseases, reported financial results for the year ended December 31, 2019 and provided an update on anticipated clinical milestones (Press release, Aldeyra Therapeutics, MAR 12, 2020, View Source [SID1234555503]).

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"2019 was a year of continued momentum for our lead programs, as we reported statistically significant and clinical relevant data that underscored the potential of reproxalap as a first-line therapy in allergic conjunctivitis and dry eye disease, conditions that in the aggregate affect more than one billion people worldwide," stated Todd C. Brady, M.D., President and CEO of Aldeyra. "Reproxalap potentially represents a highly differentiated mechanism of action compared with existing therapies. We are excited about the market opportunities for reproxalap as we continue to advance towards the completion of clinical development."

Recent Highlights and Upcoming Milestones

Allergic Conjunctivitis: Aldeyra expects to complete the INVIGORATE Phase 3 clinical trial of topical ocular reproxalap in the second half of 2020. In 2019, Aldeyra announced achievement of the primary endpoint of the Phase 3 ALLEVIATE Trial in allergic conjunctivitis, as well as statistically significant reductions in ocular itching and redness in an allergen chamber clinical trial.

Dry Eye Disease: Based on the achievement of symptom endpoints in two well-controlled clinical trials, Aldeyra plans to meet with the U.S. Food and Drug Administration (FDA) prior to initiating Part 2 of the RENEW Trial, and plans to provide an update on future development in dry eye disease following FDA feedback, expected in the second half of 2020.

Proliferative Vitreoretinopathy (PVR): Aldeyra is currently enrolling patients in the GUARD Phase 3 Trial of ADX-2191, a novel anti-inflammatory and anti-proliferative agent for the prevention of PVR, a rare sight-threatening condition with no approved treatment. The GUARD Trial is a two-part, multi-center, randomized, controlled, adaptive clinical trial evaluating the efficacy of intravitreal injections of ADX-2191 versus standard-of-care for the prevention of PVR. The trial will compare recurrent retinal detachment rates over a 24-week period following surgical repair of retinal detachment due to PVR or open globe injury. Enrollment is currently expected to be completed in 2021.

Systemic Autoimmune Disease: A single and multiple ascending dose Phase 1 clinical trial has been completed for ADX-629, a novel orally administered RASP inhibitor in development for the treatment of systemic autoimmune disease and potentially other serious medical conditions. Top-line results are expected in the second quarter of this year. Initiation of Phase 2 clinical testing is planned for the second half of 2020.

Strategic Prioritization of Late-Stage Programs in Ocular Disease

In a separate news release issued earlier today, Aldeyra announced strategic prioritization of late-stage ocular disease programs in allergic conjunctivitis, dry eye disease, and proliferative vitreoretinopathy. In conjunction with the strategic prioritization, Aldeyra appointed ophthalmology drug development expert James A. Gow, M.D., as Senior Vice President of Clinical Development.

Aldeyra has elected to place on hold its clinical development of topical dermal reproxalap for the treatment of ichthyosis associated with Sjogren-Larsson Syndrome and ADX-1612 for the treatment of post-transplant lymphoproliferative disorder. The initiatives to prioritize the portfolio are expected to extend the company’s cash runway through the end of 2021.

Year Ended December 31, 2019 Financial Review

Aldeyra reported a net loss of $60.8 million for the year ended December 31, 2019, compared with a net loss of $38.9 million in 2018. Basic net loss per share was $2.24 for the year ended December 31, 2019, compared with $1.79 per share in 2018. Losses have resulted from the costs of Aldeyra’s clinical trials and research and development programs, as well as from general and administrative expenses.

Research and development expenses were approximately $44.4 million for the year ended December 31, 2019, compared with approximately $29.8 million for the year ended December 31, 2018. The increase of $14.6 million is primarily related to the increase in clinical and preclinical development and manufacturing costs; an increase in personnel costs; and non-cash compensation costs related to a portion of upfront acquisition consideration that is subject to vesting based on continued service.

Acquired in-process research and development expenses were $6.6 million for the year ended December 31, 2019. Aldeyra did not have acquired in-process research and development expenses for the year ended December 31, 2018. The $6.6 million increase is related to the in-process research and development expenses associated with the January 2019 acquisition of Helio Vision.

General and administrative expenses were approximately $12.2 million for the year ended December 31, 2019, compared with $9.9 million for the year ended December 31, 2018. The increase of $2.3 million is primarily related to an increase in personnel costs and public company costs related to compliance with the Sarbanes-Oxley Act of 2002.

Total operating expenses were approximately $63.1 million for the year ended December 31, 2019, compared with total operating expenses of approximately $39.7 million for the year ended December 31, 2018.

Cash, cash equivalents, and marketable securities were $73.4 million as of December 31, 2019.

Conference Call & Webcast Information

Aldeyra will host a conference call today at 8:00 a.m. ET to discuss its full-year 2019 financial results and provide a corporate update. The dial-in numbers are (866) 211-4098 for domestic callers and (647) 689-6613 for international callers. The Conference ID number is 9984588.

A live webcast of the conference call will also be available on the Investors Relations section of the Aldeyra Therapeutics website at View Source After the live webcast, the event will remain archived on the Aldeyra Therapeutics website for 90 days.

About Reproxalap

Reproxalap is a novel, small-molecule immune-modulating covalent inhibitor of RASP (reactive aldehyde species), which are elevated in ocular and systemic inflammatory disease, and lead to activation of intracellular inflammatory factors, including NF-kB, inflammasomes, and Scavenger Receptor A. Reproxalap’s mechanism of action has been validated with the demonstration of statistically significant and clinically relevant activity in multiple physiologically distinct late-phase clinical indications. Topical ocular reproxalap has now been studied in more than 1,100 patients with no observed safety concerns reported; mild instillation site irritation is the most commonly reported adverse event in clinical trials.

About ADX-2191

ADX-2191, an intravitreal formulation of methotrexate, has received orphan drug and fast track designations from the FDA for the prevention of PVR. The observed clinical activity of ADX-2191 in patients with PVR is believed to be the result of down-regulation of aberrant retinal cell proliferation and activity, thereby leading to reduced retinal scarring that is characteristic of PVR. Aldeyra retains an exclusive license to certain patents related to the use of ADX-2191 for the prevention of PVR.

On March 12, 2020 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on commercializing its novel tetracycline XERAVA to treat serious and life-threatening infections, reported financial results for the fourth quarter and year ended December 31, 2019, provided an overview of recent achievements, and highlighted key milestones for 2020 (Press release, Tetraphase, MAR 12, 2020, View Source [SID1234555502]).

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"We made significant progress throughout 2019, ending the year with $3.6 million in XERAVA net sales for the full year and a quarter-to-quarter net revenue increase in the fourth quarter of 2019 of 49.3%. Our sales consist solely of actual use and not stocking retail or other channels," said Larry Edwards, President and Chief Executive Officer of Tetraphase. "We believe XERAVA is a critically important new addition to the hospital antibiotic armamentarium, and we remain committed to increasing formulary uptake and reaching all of our targeted accounts. The reorganization efforts we undertook in 2019 to create a streamlined organization singularly focused on the commercialization of XERAVA, including the elimination of our research and development function, are central to the success of our mission. With two recently completed equity offerings in November 2019 and January 2020 adding to our balance sheet, we are now in a stronger financial position to execute on our goals."

Key Milestones for 2020

Complete 143 formulary reviews for XERAVA by mid-year
Continue pulling through appropriate utilization at the 1,200 hospitals at which XERAVA is on formulary or available
Continue double-digit quarter over quarter growth for XERAVA
Create additional opportunities in TIER 2 and TIER 3 Accounts
Out-license pipeline assets and enter into arrangements for commercialization of XERAVA in Europe and Latin America
Filing by Everest Medicines for regulatory approval of XERAVA for complicated intra-abdominal infections in China in the fourth quarter of 2020
Fourth Quarter and Recent Highlights

Continued to Progress Launch of XERAVA in U.S. Hospitals With High Antibiotic Usage

The Company continues to see increased formulary uptake, with a 99% success rate for all formulary reviews to date and $3.6 million in XERAVA sales for the full-year 2019. XERAVA sales for 2018 were $178,000. Tetraphase’s salesforce is now focusing on bringing XERAVA to both Tier 1 as well as Tier 2 institutions, which are the highest users of antibiotics defined by days of therapy. The reorder rate for XERAVA continues to be strong, with reorder rates as high as 67% for all accounts and approximately 77% within the Tier 1 account segment. XERAVA is on formulary or available at more than 1,200 accounts and approximately 143 formulary reviews are pending or planned to take place over the next six months.
Completed Two Equity Financings Totaling Net Proceeds of $23.0 Million

In January 2020, the Company completed a private placement with Armistice Capital, LLC, a healthcare-focused institutional investor, priced at-the-market, that generated gross proceeds of approximately $10 million. In addition, the Company concurrently completed a registered direct offering with certain healthcare-focused institutional investors, priced at-the-market, that generated gross proceeds of approximately $7.5 million. The net proceeds from the concurrent January 2020 private placement and registered direct offering were approximately $15.9 million.

In November 2019, the Company completed a registered direct offering with Armistice Capital, LLC, a healthcare-focused institutional investor, priced at-the-market, that generated net proceeds of approximately $7.1 million.

The Company issued warrants in connection with all of the financings listed above.
Fourth Quarter and Full-Year 2019 Financial Results

As of December 31, 2019, Tetraphase had cash and cash equivalents of $21.2 million and 3.5 million shares outstanding. Subsequent to the end of the fourth quarter, the Company raised an additional $15.9 million in net proceeds via an equity offering. The Company expects that its cash and cash equivalents, including the equity proceeds and its expected revenue, will be sufficient to fund operations into the first quarter of 2021.

For the fourth quarter of 2019, Tetraphase reported a net loss of $11.4 million, or $2.75 per share, compared to a net loss of $21.5 million, or $8.00 per share, for the same period in 2018, driven by both increased product revenues and lower operating expenses. For the year ended December 31, 2019, Tetraphase reported a net loss of $70.1 million, or $22.85 per share, compared to a net loss of $72.2 million, or $27.48 per share, for the same period in 2018.

Total revenues were $1.7 million for the fourth quarter of 2019, of which $1.5 million was from sales of XERAVA, compared to $4.3 million for the same period in 2018, of which $178,000 was from sales of XERAVA. Total revenues were $7.4 million for the year ended December 31, 2019, of which $3.6 million was from sales of XERAVA, compared to $18.9 million for the same period in 2018, of which $178,000 was from sales of XERAVA. Total revenues for the fourth quarter and year ended December 31, 2019 consisted of XERAVA product revenue, license and collaboration revenue from the Company’s relationship with Everest Medicines and government contract revenue. The primary driver of the year over year decrease in revenue was a reduction in the up front and regulatory milestones received from the Company’s relationship with Everest Medicines and the wind down of its government awards and grants and the related revenue.

Research and development (R&D) expenses for the fourth quarter of 2019 were $2.5 million, compared to $10.7 million for the same period in 2018. R&D expenses for the year ended December 31, 2019 were $22.8 million, compared to $54.9 million for the same period in 2018. The significant year over year decrease in R&D expenses was driven by the completion of XERAVA development and our corporate reorganization in June 2019, which included the cessation of development of our pipeline candidates.

Selling, general and administrative (SG&A) expenses for the fourth quarter of 2019 were $9.3 million, compared to $14.7 million for the same period in 2018. SG&A expenses for the year ended December 31, 2019 were $49.0 million, compared to $37.1 million for the same period in 2018. The decrease in fourth quarter 2019 SG&A expenses compared to fourth quarter 2018 SG&A expenses was driven by our 2019 corporate reorganization and by up-front Xerava launch expenses incurred in the fourth quarter of 2018. The increase in full year 2019 SG&A expenses compared to full year 2018 SG&A expenses was driven primarily by a full year of commercial and medical affairs expenses in 2019 following the fourth quarter 2018 launch of Xerava.

About XERAVATM

XERAVA (eravacycline for injection) is a tetracycline class antibacterial indicated for the treatment of complicated intra-abdominal infections (cIAI) in patients 18 years of age and older. XERAVA was investigated for the treatment of cIAI as part of the Company’s IGNITE (Investigating Gram-Negative Infections Treated with Eravacycline) Phase 3 program. In the first pivotal Phase 3 trial in patients with cIAI, twice-daily intravenous (IV) XERAVA met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to ertapenem and was well-tolerated. In the second Phase 3 clinical trial in patients with cIAI, twice-daily IV XERAVA met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to meropenem and was well-tolerated. In both trials, XERAVA achieved high cure rates in patients with Gram-negative pathogens, including resistant isolates.

XERAVATM Important Safety Information

XERAVA is a tetracycline class antibacterial indicated for the treatment of complicated intra-abdominal infections in patients 18 years of age and older.

XERAVA is not indicated for the treatment of complicated urinary tract infections.

To reduce the development of drug-resistant bacteria and maintain the effectiveness of XERAVA and other antibacterial drugs, XERAVA should be used only to treat or prevent infections that are proven or strongly suspected to be caused by susceptible bacteria.

XERAVA is contraindicated for use in patients with known hypersensitivity to eravacycline, tetracycline-class antibacterial drugs or to any of the excipients. Life-threatening hypersensitivity (anaphylactic) reactions have been reported with XERAVA.

The use of XERAVA during tooth development (last half of pregnancy, infancy and childhood to the age of eight years) may cause permanent discoloration of the teeth (yellow-gray-brown) and enamel hypoplasia.

The use of XERAVA during the second and third trimester of pregnancy, infancy and childhood up to the age of eight years may cause reversible inhibition of bone growth.

Clostridium difficile associated diarrhea (CDAD) has been reported with use of nearly all antibacterial agents and may range in severity from mild diarrhea to fatal colitis.

The most common adverse reactions observed in clinical trials (incidence ≥ 3%) were infusion site reactions, nausea, and vomiting.

XERAVA is structurally similar to tetracycline-class antibacterial drugs and may have similar adverse reactions. Adverse reactions including photosensitivity, pseudotumor cerebri, and anti-anabolic action which has led to increased blood urea nitrogen, azotemia, acidosis, hyperphosphatemia, pancreatitis, and abnormal liver function tests, have been reported for other tetracycline-class antibacterial drugs, and may occur with XERAVA. Discontinue XERAVA if any of these adverse reactions are suspected.

On March 12, 2020 Rubius Therapeutics, Inc. (Nasdaq:RUBY) a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines, reported fourth quarter and full-year 2019 financial results and announced its plan to focus on the development of its oncology and autoimmunity pipeline (Press release, Rubius Therapeutics, MAR 12, 2020, View Source [SID1234555501]).

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This strategic decision allows Rubius to focus on the areas in which its RED PLATFORMÒ may offer the greatest potential to benefit patients. Development in these therapeutic areas is enabled by the Company’s investment in internal manufacturing at its Smithfield, RI facility, which is now cGMP ready to produce clinical supply for its lead oncology program, RTX-240, a broad immunostimulatory Red Cell Therapeutic for the treatment of solid tumors. The Investigational New Drug (IND) application for RTX-240 has been cleared by the U.S. Food and Drug Administration, and the Company plans to announce when the first patient has been dosed in the Phase 1 clinical trial. As previously announced, Rubius is on track to file an IND for RTX-321, its first artificial antigen-presenting cell for the treatment of HPV-positive cancers, by year-end. The company also expects to manufacture RTX-321 clinical supply at its fully owned manufacturing facility.

"Over the past two years, we have generated exciting oncology preclinical data, demonstrating the ability of our Red Cell Therapeutics to both broadly activate the immune system, and induce tumor-specific responses by activating and expanding antigen-specific T cells with our artificial antigen-presenting cells. By focusing on the development of our oncology and autoimmune pipeline, we believe we will have the greatest opportunity to bring life-saving therapies to patients, enhance shareholder value and extend our cash runway into 2022," said Pablo J. Cagnoni, M.D., president and chief executive officer of Rubius Therapeutics. "With our internal cGMP manufacturing established, we are well positioned to advance this entirely new class of allogeneic cellular medicines."

RTX-240 for the Treatment of Solid Tumors

RTX-240 is an allogeneic cellular therapy that is engineered to broadly stimulate the adaptive and innate immune systems to generate an antitumor response. RTX-240 expresses 4-1BBL and IL-15TP, a fusion of IL-15 and IL-15 receptor alpha, on the cell surface with the goal of improving antitumor activity and overcoming resistance to immunotherapy in patients with solid tumors. RTX-240 may provide a differentiated approach to treating solid tumors or hematologic malignancies in immunotherapy-naïve patients or in patients whose disease has become resistant or refractory to immunotherapies, including checkpoint inhibitors.

RTX-134 Program Update

As a result of the decision to focus on oncology and autoimmunity, the Company is deprioritizing the RTX-134 program for the treatment of phenylketonuria (PKU) and its other rare disease programs. Multiple factors contributed to this decision, including unanticipated delays in the RTX-134 program, primarily due to continued manufacturing challenges at the Company’s contract manufacturing organization (CMO), the anticipated high cost associated with producing chronic, high-dose therapy for enzyme deficiencies and the continued momentum of the Company’s oncology pipeline. Future capital investments and improvements in manufacturing efficiency, together with enhancements to the RED PLATFORM, may enable Rubius to revisit chronic, high dose-dependent conditions in the future.

As previously announced, the first patient was dosed in the Phase 1b PKU clinical trial of RTX-134 in January 2020. While there were no reported adverse events and RTX-134 administration was well tolerated, the results from the first patient were uninterpretable possibly due, in part, to the low dose of cells administered and the sensitivity of the flow cytometry assay used to detect circulating cells. As a result of the deprioritization, the current Phase 1b clinical trial in PKU will be discontinued.

Autoimmune Program Update

Rubius’ autoimmune Red Cell Therapeutics are engineered to express specific autoimmune disease-associated antigens either within the cell or on the cell surface to take advantage of how the body normally maintains self-tolerance, thereby retraining the immune system to no longer see self-antigens as foreign. Red Cell Therapeutics are designed to specifically modulate complex counter-regulatory immune responses, potentially enabling greater efficacy with lower toxicity, and, in some cases, even cures, when compared to currently available non-specific immunosuppressive treatments. Rubius is focusing on T cell-mediated autoimmune diseases and is pursuing Type 1 diabetes, along with a number of other undisclosed programs. The Company expects to provide an update on its preclinical autoimmune pipeline in the future.

Extension of Cash Runway

With the cost savings from the discontinuation of the RTX-134 clinical trial and deprioritization of the other rare disease programs and a reallocation of capital and personnel resources, Rubius’ cash runway will be extended into 2022.

Additional Business Updates

Rubius strengthened its leadership team and board of directors by appointing:

·Internationally recognized autoimmunity and translational leader Laurence Turka, M.D., as chief scientific officer;
·Trained oncologist and immunologist, Christina Coughlin, M.D., Ph.D., as chief medical officer, who has extensive experience leading clinical development and translational medicine teams and has a track record of building successful drug development organizations with a particular focus in cellular therapy and oncology; and
·Anne Prener, M.D., Ph.D., to its board of directors, who has significant experience in drug development and commercialization.

Fourth Quarter 2019 Financial Results

Net loss for the fourth quarter of 2019 was $44.5 million or $0.56 per common share, compared to $27.2 million or $0.35 per common share in the fourth quarter of 2018.

In the fourth quarter of 2019, Rubius invested $30.5 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $16.5 million in the fourth quarter of 2018. This year-over-year increase was driven primarily by $3.9 million in incremental R&D program spending related to the Company’s Phase 1b clinical trial for RTX-134 and towards preclinical and IND-enabling activities for Rubius’ lead oncology programs, including RTX-240. In addition, $6.0 million in incremental R&D spending was driven by increased R&D headcount, a move into larger facilities and purchasing lab supplies to support expanded research activities. Contract research and development costs increased by $3.3 million and R&D stock-based compensation also increased by $0.8 million.

G&A expenses were $14.9 million during the fourth quarter of 2019, as compared to $12.6 million for the fourth quarter of 2018. The higher costs were primarily driven by a $1.0 million increase in personnel and facility costs due to increased headcount in the general and administrative function, as well as increases in professional fees and infrastructure costs to support the Company’s growth.

Full Year 2019 Financial Results

Net loss for the full year 2019 was $163.5 million or $2.08 per common share, compared to $89.2 million or $2.27 per common share for the full year 2018.

For the full year 2019, Rubius invested $112.4 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $51.8 million for the full year 2018. This year-over-year increase was largely due to an additional $31.8 million in R&D personnel, contract research and development, facilities and lab supplies to support the Company’s pipeline expansion and platform investments and $23.7 million in R&D program spending, including costs related to the Company’s RTX-134 Phase 1b clinical trial as well as preclinical and IND-enabling activities for Rubius’ lead oncology programs, including RTX-240. R&D stock-based compensation also increased by $5.2 million.

G&A expenses were $57.2 million during the twelve months of 2019, as compared to $39.9 million for the same period in 2018. The higher costs were primarily driven by an $8.5 million increase in stock-based compensation and $8.8 million increase in personnel costs, professional and facility fees to support the Company’s growth and to operate as a public company.

Cash Position

As of December 31, 2019, cash, cash equivalents and investments were $283.3 million as compared to $404.1 million as of December 31, 2018, providing Rubius with a cash runway into 2022. During the year, the Company used $110.4 million of cash to fund operations and $40.7 million to fund capital expenditures, including work related to the buildout of Rubius’ manufacturing facility. In addition, the Company drew down a second tranche of $25.0 million from its $75.0 million loan agreement with Solar Capital in June 2019, which leaves a third tranche of $25.0 million that can be drawn through June 2020, subject to the satisfaction of certain financial covenants.

Conference Call Details

The company will host a conference call and webcast at 8:00 a.m. EST to discuss this update. The audio webcast will be available on the Events and Presentations page within the Investors and Media section of the Rubius Therapeutics website. The update may also be accessed by dialing 1-800-289-0045 (domestic) or 1-615-622-8086 (international) five minutes prior to the start of the call and providing the passcode 6394385. An archived webcast will be accessible for 90 days after the event.

On March 12, 2020 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported its financial results for the year ended December 31, 2019 and provides a corporate update (Press release, BioLineRx, MAR 12, 2020, View Source [SID1234555500]).

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Highlights and achievements during the fourth quarter 2019 and subsequent period:

Presented updated preliminary Phase 2a data from the triple combination arm of the COMBAT/KEYNOTE-202 study, under collaboration with Merck, evaluating the safety, tolerability and efficacy of motixafortide (BL-8040) in combination with KEYTRUDA (pembrolizumab) and chemotherapy in patients with second-line metastatic pancreatic cancer, demonstrating a 32% overall response rate and a 77% disease control rate out of 22 evaluable patients at that time, with median duration of clinical benefit for all 17 patients with disease control (7 partial response and 10 stable disease patients) of 7.8 months; and reiterated expectation for progression free survival and overall survival data in mid-2020;

Completed recruitment (N=43) of the triple combination arm of the COMBAT/KEYNOTE-202 study;

Announced Notice of Allowance from USPTO for a broad patent covering motixafortide in combination with anti-PD-1 for the treatment of any and all types of cancer;

Received Orphan Drug Designation for motixafortide for the treatment of pancreatic cancer in Europe;

Presented positive triple-combination preclinical data from the evaluation of motixafortide in combination with an anti-PD-1 and chemotherapy in pancreatic cancer, supporting motixafortide’s mechanism of action and providing additional strong rationale for the triple-combination clinical study, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting (SITC) (Free SITC Whitepaper).

"We achieved a significant milestone during the fourth quarter of 2019 with the announcement of preliminary data from the ongoing triple combination arm of our COMBAT/KEYNOTE-202 study in second-line pancreatic cancer," stated Philip Serlin, Chief Executive Officer of BioLineRx. "The promising initial results demonstrated robust and durable responses to the triple combination treatment, with an overall response rate almost double the current chemotherapy standard-of-care treatment in second-line patients, and a trend of patients receiving treatment for an extended period that move from stable disease to partial response. Looking ahead, we have now fully enrolled this study and remain on track to announce progression free and overall survival data in mid-year.

"In parallel, our late-stage trials of motixafortide in AML and stem cell mobilization are progressing, and we also look forward to these key data readouts later this year.

"Regarding our second clinical oncology candidate, the universal anti-cancer vaccine AGI-134, we successfully completed the dose-escalation Part 1 of the ongoing Phase 1/2a clinical trial in a range of solid tumor types, and are currently advancing Part 2 of the study as expeditiously as possible. We look forward to initial results of Part 2 by year-end 2020," Mr. Serlin concluded.

Upcoming 2020 Milestones

Progression-free survival and overall survival data from the triple combination arm of the COMBAT/KEYNOTE-202 Phase 2a study in mid-2020;

Interim results from the Phase 2b AML consolidation study in the second half of 2020;

Top-line results from Phase 3 GENESIS registrational study in stem cell mobilization in the second half of 2020;

Initial results from Part 2 of Phase 1/2a trial of AGI-134 by year-end 2020.

Financial Results for the Year Ended December 31, 2019

Research and development expenses for the year ended December 31, 2019 were $23.4 million, an increase of $3.6 million, or 18.3%, compared to $19.8 million for the year ended December 31, 2018. The increase resulted primarily from higher expenses associated with the motixafortide GENESIS and COMBAT clinical trials, offset by a decrease in expenses related to BL-1230, a project that was terminated in 2018, as well as a decrease in payroll and share-based compensation.

Sales and marketing expenses for the year ended December 31, 2019 were $0.9 million, a decrease of $0.5 million, or 37.0%, compared to $1.4 million for the year ended December 31, 2018. The decrease resulted primarily from a decrease in payroll and related expenses, including a one-time compensation payment in the 2018 period.

General and administrative expenses for the year ended December 31, 2019 were $3.8 million, a decrease of $0.6 million, or 14.0% compared to $4.4 million for the year ended December 31, 2018. The decrease resulted primarily from a decrease in share-based compensation.

The Company’s operating loss for the year ended December 31, 2019 amounted to $28.1 million, compared to an operating loss of $25.6 million for the year ended December 31, 2018.

Non-operating income amounted to $4.2 million for the year ended December 31, 2019, compared to non-operating income of $2.4 million for the year ended December 31, 2018. Non-operating income for the year ended December 31, 2019 primarily relates to fair-value adjustments of warrant liabilities on the Company’s balance sheet, offset by warrant offering expenses. Non-operating income for the year ended December 31, 2018 primarily relates to fair-value adjustments of warrant liabilities on the Company’s balance sheet, as well as a capital gain from realization of the investment in iPharma.

Net financial expenses amounted to $1.5 million for the year ended December 31, 2019 compared to net financial income of $0.2 million for the year ended December 31, 2018. Net financial expenses for the year ended December 31, 2019 primarily relate to interest paid on loans, offset by investment income earned on bank deposits. Net financial income for the year ended December 31, 2018 primarily relates to investment income earned on bank deposits, offset by interest paid on loans.

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The Company’s net loss for the year ended December 31, 2019 amounted to $25.5 million, compared with a net loss of $23.0 million for the year ended December 31, 2018.

The Company held $27.5 million in cash, cash equivalents and short-term bank deposits as of December 31, 2019.

Net cash used in operating activities for the year ended December 31, 2019 was $22.7 million, compared to $24.2 million for the year ended December 31, 2018. The $1.5 million decrease in net cash used in operating activities in 2019 was primarily the result of changes in operating asset and liability items in the two periods., i.e., a decrease in prepaid expenses and other receivables in 2019 versus an increase in 2018, as well as an increase in accounts payable and accruals in 2019 versus a decrease in 2018.

Net cash provided by investing activities for the year ended December 31, 2019 was $5.3 million, compared to $9.6 million for the year ended December 31, 2018. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits during both periods, the acquisition of an additional 20% economic interest in Motixafortide in 2018, as well as a realization of the investment in iPharma during 2018.

Net cash provided by financing activities for the year ended December 31, 2019 was $19.2 million, compared to $13.1 million for the year ended December 31, 2018. The cash flows in 2019 primarily reflect the underwritten public offering of ADSs in February 2019, as well as net proceeds from the ATM program. The cash flows in 2018 reflect the net proceeds of the loan from Kreos Capital, as well as net proceeds from the ATM program.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, March 12, 2020 at 10:00 a.m. EDT. To access the conference call, please dial +1-888-668-9141 from the US or +972-3-918-0609 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until March 14, 2020; please dial +1-877-456-0009 from the US or +972-3-925-5927 internationally.

On March 12, 2020 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, provided an update on recent company developments and reported fourth quarter and full-year financial results for the period ended December 31, 2019 (Press release, SpringWorks Therapeutics, MAR 12, 2020, View Source [SID1234555498]).

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"2019 was a very successful year for SpringWorks. We initiated three clinical trials, including two potentially registrational trials in rare oncology indications, formed MapKure with BeiGene to advance a next-generation B-RAF inhibitor, and signed our first collaboration with GlaxoSmithKline to evaluate nirogacestat in combination with BCMA-directed therapies in patients with relapsed and refractory multiple myeloma," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our strong execution in 2019 positions us to deliver on our planned strategy in 2020 and beyond. We are pursuing a broad effort with industry-leading BCMA developers to advance potentially best-in-class multiple myeloma combination therapies using nirogacestat, are focused on continuing to enroll our ongoing clinical trials and further expanding our portfolio through additional in-licenses and collaborations."

Recent Business Highlights

Initiated a Phase 1 clinical trial of BGB-3245, a selective next-generation B-RAF inhibitor, in adult patients with biomarker-defined advanced or refractory solid

tumors. BGB-3245 is being advanced by MapKure LLC, a clinical-stage company that is jointly owned by SpringWorks and BeiGene, Ltd.

 Entered into a clinical collaboration agreement with Allogene Therapeutics to evaluate nirogacestat in combination with ALLO-715, an investigational anti-B-cell maturation antigen (BCMA) allogeneic CAR T cell therapy, in patients with relapsed or refractory multiple myeloma.

 Presented data at the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting showing activity of nirogacestat alone and in combination with GlaxoSmithKline’s investigational BCMA antibody-drug conjugate (ADC), belantamab mafodotin, in preclinical models of human multiple myeloma.

Dosed the first patients in the Phase 2b ReNeu clinical trial evaluating mirdametinib in children and adults with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN).

Fourth Quarter and Full Year 2019 Financial Results

 Research and Development (R&D) Expenses: R&D expenses were

$12.2 million and $42.5 million for the fourth quarter and year-to-date periods, respectively, compared to $3.7 million and $9.9 million for the comparable periods of 2018, respectively. The increases in R&D expenses in 2019 were primarily attributable to increased clinical study and drug supply costs related to the ongoing Phase 3 DeFi and Phase 2b ReNeu trials, as well as growth in employee costs, including non-cash share-based compensation associated with increases in the number of R&D personnel.

 General and Administrative (G&A) Expenses: G&A expenses were $5.2 million and $16.7 million for the fourth quarter and year-to-date periods, respectively, compared to $2.7 million and $8.6 million for the comparable periods of 2018, respectively. The increases in G&A expenses in 2019 were primarily attributable to growth in employee costs, including non-cash share-based compensation associated with increases in the number of G&A personnel, and increases in consulting and professional services related to the expansion of our business activities.

 Net Loss Attributable to Common Stockholders: SpringWorks reported net losses of $16.2 million, or $0.39 loss per share, and $50.6 million, or $3.81 loss per share, for the fourth quarter and year-to-date periods ended December 31, 2019, respectively. This compares to net losses of $6.2 million, or $10.82 loss per unit, and $17.8 million, or $52.24 loss per unit, for the comparable periods of 2018, respectively.

Cash Position: Cash and cash equivalents were $327.7 million as of December 31, 2019.