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Scholar Rock Reports Third Quarter 2019 Financial Results and Highlights Business Progress

On November 12, 2019 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the third quarter ended September 30, 2019 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, NOV 12, 2019, View Source [SID1234551110]).

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"The third quarter of 2019 has been a time of focused execution as we continue to advance our growing pipeline. Notably, we have been able to accelerate the development of SRK-181 and are now planning to initiate a Phase 1 dose escalation and proof-of-concept trial in patients with solid tumors in the first quarter of 2020, a testament to the strength of our team and scientific progress," said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. "As we wrap up the year and look towards 2020 and beyond, we are well positioned to work towards a number of important milestones across our pipeline, including read-outs from the TOPAZ Phase 2 trial for SRK-015 and the initiation of our Phase 1 trial for SRK-181, that will further elucidate the potential of our product candidates in improving the lives of patients suffering from serious diseases."

Company Highlights and Upcoming Milestones

SRK-015 Program:

Preclinical and Phase 1 SRK-015 Healthy Volunteer Data Presented at World Muscle Society Congress. In October 2019, Scholar Rock presented data for SRK-015, a highly selective inhibitor of myostatin activation, that further enforce the therapeutic rationale for the advancement of SRK-015 into the TOPAZ Phase 2 clinical trial. Preclinical studies demonstrated improved muscle strength following the administration of a mouse analog of SRK-015 (muSRK-015P) in mouse models of early and late stage restoration of SMN, the protein that is deficient in the genetic disease SMA. Multi-fold increases in serum latent myostatin levels following treatment with muSRK-015P were shown across animal species in mice, rats, and cynomolgus monkeys. In the Phase 1 clinical trial in adult healthy volunteers, SRK -015 was well-tolerated, had a well-behaved pharmacokinetic (PK) profile, and demonstrated robust and sustained target engagement.

Scholar Rock is on track to report preliminary PK and pharmacodynamic (PD) data for a subset of patients in the Phase 2 TOPAZ trial by the end of 2019, which will provide initial observations on target engagement of latent myostatin in patients with Type 2 and Type 3 SMA. These preliminary PK/PD results will be followed by interim safety and efficacy results for a subset of patients with six months of treatment exposure expected in the first half of 2020 and top-line data for the full 12-month treatment period expected beginning in the fourth quarter of 2020.

Identification of Second Indication for SRK-015 Planned for 2020. Scholar Rock continues to evaluate multiple potential opportunities for which SRK-015 could offer clinical benefit and is assessing additional potential clinical settings in which the selective inhibition of the activation of myostatin may offer therapeutic benefit.
SRK-181 Program:

Accelerated Initiation of Phase 1 Dose Escalation and Proof-of-Concept Clinical Trial of SRK-181 in Patients with Solid Tumors to the First Quarter of 2020. Scholar Rock intends to develop SRK-181, an isoform-selective inhibitor of TGFβ1 activation as a cancer immunotherapy in combination with anti-PD(L)1 antibodies. The company now plans to initiate a Phase 1 trial in the first quarter of 2020, in patients with locally advanced or metastatic solid tumors that exhibit primary resistance to anti-PD(L)1 antibodies. The two-part trial will consist of a dose escalation portion as both a single-agent and SRK-181 in combination with an approved anti-PD(L)1 antibody, as well as a dose expansion portion consisting of multiple tumor-specific cohorts evaluating SRK-181 in combination with an approved anti-PD(L)1 antibody.
RGMc Program:

Nomination of a Product Candidate from the RGMc Program Planned in 2020. Scholar Rock is evaluating a number of highly specific inhibitors of repulsive guidance molecule C (RGMc) and plans to nominate an antibody as its third product candidate in 2020. RGMc’s known function is localized to hepatocytes and the identification of RGMc selective-antibodies may offer the potential for liver-specific modulation of BMP6 signaling to address iron-restricted anemias.
Third Quarter 2019 Financial Results

For the quarter ended September 30, 2019, net loss was $16.1 million or $0.55 per share compared to a net loss of $10.8 million or $0.44 per share for the quarter ended September 30, 2018.

Revenue was $4.8 million for the quarter ended September 30, 2019 and was related to the Gilead Collaboration Agreement that was executed in December 2018.
Research and development expense was $15.7 million for the quarter ended September 30, 2019 compared to $8.1 million for the quarter ended September 30, 2018. The increase year-over-year primarily reflects preclinical and manufacturing costs for SRK-181, clinical development costs associated with SRK-015, and higher personnel-related costs.

General and administrative expense was $6.2 million for the quarter ended September 30, 2019 compared to $3.2 million for the quarter ended September 30, 2018. The increase year-over-year was primarily attributable to increased headcount, stock compensation, separation-related expense, and professional services.
As of September 30, 2019, Scholar Rock had cash, cash equivalents, and marketable securities of $176.1 million, compared to $175.6 million as of December 31, 2018.

Biomica Reports Positive Preliminary Results in Animal Studies in Its Immuno-oncology Program

On November 12, 2019 Biomica, an emerging biopharmaceutical company developing innovative microbiome-based therapeutics, and a subsidiary of Evogene Ltd. (NASDAQ: EVGN) (TASE: EVGN), reported positive preliminary results from recently completed animal studies in its immuno-oncology program, wherein anti-tumor activity was tested in mice following treatment with Biomica’s rationally designed bacterial consortia BMC121 & BMC127 (Press release, Biomica, NOV 12, 2019, View Source [SID1234551109]).

It has been widely described in the scientific literature[1] that gut microbiome could affect the efficacy of cancer immunotherapy, especially immune-checkpoint inhibitors involving blockade of PD-1 or PD-L1 and CTLA-4. The study, which is part of Biomica’s immuno-oncology program, examined the effect of addition of Biomica’s rationally designed bacterial consortia BMC121 & BMC127 on the response to checkpoint inhibitor (anti-PD1) therapy in a mouse cancer model. BMC121 & BMC127 bacterial consortia were identified & designed based on data obtained from cancer patients who displayed varying degrees of response to immune-checkpoint inhibitors therapy. The specific strains selected for the consortia were designed to trigger beneficial anti-tumor immune-modulatory processes. Identification and selection of the bacteria for BMC121 & BMC127 consortia were done using PRISM, a high-resolution microbiome analysis platform and a detailed functional genomic analysis.

In the study, Biomica’s drug candidates were tested in vivo in combination with checkpoint inhibitor therapy (anti-PD1) using a mouse cancer model. BMC121 and BMC127 demonstrated improved anti-tumor activity compared to checkpoint inhibitor therapy alone also indicating validation of the computational predictions.

Improved antitumor activity following the administration of BMC121 & BMC127, compared to treatment with checkpoint inhibitor therapy alone, was manifested in a number of parameters. These parameters include higher percentage of responders, greater tumor growth inhibition (%TGI) and increased antitumor immune responses, including higher numbers of tumor-infiltrating immunocytes, higher levels of tumor infiltrating CD8 T-lymphocytes, and higher levels of intra-tumor inflammation, indicating an increased anti-tumor immune reaction. The results of these in vivo studies are the first demonstration that treatment with BMC121 & BMC127 may increase the efficacy of immune checkpoint inhibitors therapy and improve anti-tumor activity also indicating validation of the computational predictions.

Prof. Yehuda Ringel, Biomica CSO, stated: "The results of these in vivo studies are the first demonstration of the beneficial potential of BMC121 & BMC127 in cancer immunotherapy. We look forward to conducting broader studies to further validate our computational mechanism-of-action predictions at the beginning of next year."

Dr. Elran Haber, Biomica CEO, stated: "I am very pleased with these encouraging results that show early promise of improving the efficacy of immune checkpoint inhibitors therapy. Although immunotherapies hold great promise in treating cancer, only a portion of patients respond to the available immunotherapy drugs. Our drug candidates were designed to provide specific microbial functional capabilities to trigger beneficial immuno-stimulatory anti-tumor activity, ultimately leading to better patient response to immune-checkpoint inhibitors therapy."

MilliporeSigma Licenses CRISPR Gene-Editing Technology to Evotec

On November 12, 2019 MilliporeSigma reported that it has signed a license agreement providing Evotec SE access to MilliporeSigma’s foundational CRISPR intellectual property (Press release, MilliporeSigma, NOV 12, 2019, View Source [SID1234551108]). Evotec, an international biotechnology company headquartered in Hamburg, Germany, will use MilliporeSigma’s CRISPR genome-editing technology to create edited cell lines for Evotec’s commercial and internal research purposes.

MilliporeSigma’s CRISPR license to Evotec is the impetus for important drug testing and discovery, which will accelerate research and lead to new therapy development
MilliporeSigma’s CRISPR license to Evotec is the impetus for important drug testing and discovery, which will accelerate research and lead to new therapy development
"This CRISPR license is the impetus for important drug testing and discovery that promises to accelerate research and lead to the development of new therapies," said Udit Batra, CEO, MilliporeSigma. "MilliporeSigma has been at the forefront of innovation in gene editing for 15 years and continues to work with industry and academia to solve complex problems using our patented CRISPR technology, both ethically and responsibly."

Evotec plans to use MilliporeSigma’s CRISPR Intellectual Property Portfolio to develop precisely engineered assays to determine the biology and toxicity for potential drug candidates during the drug development cycle.

"Evotec is excited to continue its strong partnership with MilliporeSigma through licensing their groundbreaking CRISPR technology," said Craig Johnstone, Chief Operating Officer of Evotec. "Our vision and passion for innovation includes using CRISPR technology to test and improve the translation of efficacy of the new drugs we develop to bring relief to patients suffering from underserved conditions worldwide."

This new license reinforces past collaborations between MilliporeSigma and Evotec. In November 2016, the companies entered into a set of agreements whereby Evotec would provide screening services using MilliporeSigma’s collection of genetic reagents including CRISPR and shRNA libraries. Combining access to MilliporeSigma’s genome editing libraries with Evotec’s screening expertise offers a faster path to explore and identify new drug targets.

MilliporeSigma holds 20 CRISPR-related patents worldwide in methods and composition, including the fundamental technology of CRISPR Cas9 for genetic integration in mammalian cells.

As a supplier of genome-editing technology, MilliporeSigma supports research with genome editing under careful consideration of ethical and legal standards. MilliporeSigma’s parent company, Merck KGaA, Darmstadt, Germany has established an independent, external Bioethics Advisory Panel to provide guidance for research in which its businesses are involved, including research on or using genome editing. The company has also defined a clear operational position considering scientific and societal issues to inform promising therapeutic approaches for use in research and applications.

Follow MilliporeSigma on Twitter @MilliporeSigma, on Facebook @MilliporeSigma and on LinkedIn.

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About the Life Science Business of Merck KGaA, Darmstadt, Germany
The Life Science business of Merck KGaA, Darmstadt, Germany, which operates as MilliporeSigma in the U.S. and Canada, has some 21,000 employees and 59 manufacturing sites worldwide, with a portfolio of more than 300,000 products focused on scientific discovery, biomanufacturing and testing services. Udit Batra is the global chief executive officer of MilliporeSigma.

Merck KGaA, Darmstadt, Germany completed its $17 billion acquisition of Sigma-Aldrich in November 2015, creating a leader in the $125 billion global life science industry.

Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science and performance materials. Around 56,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From advancing gene-editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2018, Merck KGaA, Darmstadt, Germany generated sales of €14.8 billion in 66 countries.

The company holds the global rights to the name and trademark "Merck" internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany operate as EMD Serono in healthcare, MilliporeSigma in life science, and EMD Performance Materials. Since its founding 1668, scientific exploration and responsible entrepreneurship have been key to the company’s technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. For more information about Merck, KGaA, Darmstadt, Germany, visit www.emdgroup.com.

Fortress Biotech Reports Third Quarter 2019 Financial Results and Recent Corporate Highlights

On November 12, 2019 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on identifying, in-licensing and developing high-potential marketed and development-stage drugs and drug candidates, reported financial results and recent corporate highlights for the third quarter ended September 30, 2019 (Press release, Fortress Biotech, NOV 12, 2019, View Source [SID1234551047]).

Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We are very pleased with the progress made during the third quarter of 2019 as we continue to focus on generating shareholder value in five distinct ways – through potential revenue and balance sheet growth, monetizations, priority review vouchers and future sales royalties. We recently acquired and launched Ximino, the second prescription oral antibiotic for acne in our marketed dermatology portfolio, which we expect will enable us to continue to grow the commercial side of our business. The last quarter of 2019 is expected to be marked by the achievement of additional milestones, notably the anticipated filing of a New Drug Application ("NDA") for IV tramadol for post-surgical pain management."

Dr. Rosenwald continued, "We are also pleased that MB-107, the lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("XSCID"), also known as bubble boy disease, being developed by our partner company Mustang Bio, was granted the Regenerative Medicine Advanced Therapy ("RMAT") designation by the U.S. Food and Drug Administration ("FDA"). We look forward to the presentation of additional clinical data on MB-107 at the upcoming 61st ASH (Free ASH Whitepaper) Annual Meeting and hope to achieve regulatory agreement with the FDA later this year to potentially expedite the development and approval of this critically needed treatment option for XSCID (bubble boy) patients. In addition, positive interim data for cosibelimab, an anti-PD-L1 antibody, were presented at the European Society for Medical Oncology ("ESMO") Congress 2019. The ongoing Phase 1 clinical trial of cosibelimab could support the submission of an initial Biologics License Application ("BLA") to the FDA. We are proud of our achievements during the third quarter and are well positioned to build on the momentum in order to continue to execute on our business plan into 2020 and beyond."

Financial Results:

·As of September 30, 2019, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), and restricted cash totaled $156.0 million, compared to $170.5 million as of June 30, 2019, and $99.2 million as of December 31, 2018, a decrease of $14.5 million for the quarter and an increase of $56.8 million year-to-date.
·Fortress’ net revenue totaled $9.8 million for the third quarter of 2019, which includes $9.5 million in net revenue generated from our marketed dermatology products. This compares to a total of $5.2 million in net revenue for the third quarter of 2018.
·Research and development expenses were $14.6 million for the third quarter of 2019, of which $13.9 million was related to Fortress partner companies. This compares to $16.1 million for the third quarter of 2018, of which $15.1 million was related to Fortress partner companies. Non-cash, stock-based compensation expenses included in research and development were $1.2 million for the third quarter of 2019, compared to $1.8 million for the third quarter of 2018.

·Research and development expenses from license acquisitions totaled $0.7 million for the third quarter of 2019, compared to $3.7 million for the third quarter of 2018.
·General and administrative expenses were $14.3 million for the third quarter of 2019, of which $9.3 million was related to Fortress partner companies. This compares to $12.2 million for the third quarter of 2018, of which $7.4 million was related to Fortress partner companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.5 million for the third quarter of 2019, compared to $2.3 million for the third quarter of 2018.
·Net loss attributable to common stockholders was $12.8 million, or $0.22 per share, for the third quarter of 2019, compared to a net loss attributable to common stockholders of $16.6 million, or $0.37 per share, for the third quarter of 2018. For the first nine months of 2019, net loss attributable to common stockholders was $24.5 million or $0.46 per share, compared to $59.3 million or $1.36 per share for the first nine months of 2018.

Recent Corporate Highlights1:

Marketed Dermatology Products

·In the third quarter of 2019, our marketed products generated net revenue of $9.5 million, compared to $5.2 million in the third quarter of 2018.
·Also, in the third quarter of 2019, we launched Ximino, a prescription oral antibiotic for acne.
·We anticipate 2019 annual net pharmaceutical revenues of approximately $30 million.
·We currently have 37 sales representatives dedicated to the dermatology product portfolio.
·We anticipate launching one to two new prescription products in 2020.
·Our dermatology products are marketed by our partner company, Journey Medical Corporation.

IV Tramadol

·In October 2019, an eAbstract was presented at ANESTHESIOLOGY 2019, the Annual Meeting of the American Society of Anesthesiologists in Orlando, FL, highlighting the Phase 3 data for IV tramadol in the management of post-surgical pain in patients undergoing bunionectomy, an orthopedic model. As previously announced, IV tramadol 50 mg met the primary endpoint, as well as all of the key secondary endpoints. We expect an NDA for IV tramadol to be filed with the FDA by year-end 2019.
·We anticipate that the Avenue Therapeutics merger with InvaGen Pharmaceuticals Inc. will be completed in 2021, if the conditions of the Stock Purchase and Merger Agreement are met, resulting in a potential net distribution to Fortress of approximately $48 million plus potential future product royalties.
·Also, in October 2019, IV tramadol Phase 1 clinical data were published in the peer-reviewed journal Clinical Pharmacology in Drug Development. The paper titled "Comparing the Pharmacokinetics of 2 Novel Intravenous Tramadol Dosing Regimens to Oral Tramadol: A Randomized 3-Arm Crossover Study", can be accessed here.
·IV tramadol is currently in development at our partner company, Avenue Therapeutics, Inc.

MB-107 (Lentiviral Gene Therapy for XSCID)

·In August 2019, MB-107, a lentiviral gene therapy for the treatment of XSCID, also known as bubble boy disease, was granted RMAT designation by the FDA.
·Also in August 2019, we entered into a license agreement with CSL Behring for the Cytegrity stable producer cell line, which will be used to produce the viral vector for the MB-107 lentiviral gene therapy program for the treatment of XSCID.
·Updated Phase 1/2 clinical data for MB-107 have been selected for oral and poster presentations at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held December 7-10, 2019, at the Orange County Convention Center in Orlando, FL.
·MB-107 is currently in development at our partner company, Mustang Bio, Inc.

1 Includes product candidates in development at Fortress, majority-owned and controlled partners and partners in which Fortress holds significant minority ownership positions. As used herein, the words "we," "us" and "our" may refer to Fortress individually or together with our affiliates and/or partners, as dictated by context.

CAEL-101

·In October 2019, the European Commission granted orphan drug designation to CAEL-101 for the treatment of AL amyloidosis. The U.S. Food and Drug Administration (FDA) had previously granted two orphan drug designations to CAEL-101 for the use of CAEL-101 as a therapeutic agent for patients with AL amyloidosis, and the use of CAEL-101 as a radio-imaging agent in amyloidosis.
·We received feedback from the FDA that supports our initiating a pivotal Phase 2/3 program stratified by Mayo stage beginning in 1H2020.
·CAEL-101 is currently in development at Caelum Biosciences, Inc., with its partner Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN).

Cosibelimab (formerly CK-301, anti-PD-L1 antibody)

·In September 2019, positive interim results for cosibelimab were presented at the ESMO (Free ESMO Whitepaper) Congress 2019 in Barcelona, Spain. The poster presentation provided updated interim efficacy and safety results from the ongoing multicenter Phase 1 clinical trial of cosibelimab, including expansion cohorts in cutaneous squamous cell carcinoma ("CSCC") and non-small cell lung cancer ("NSCLC"). A 50% objective response rate was observed in CSCC, and a 40% objective response rate was observed in NSCLC. Cosibelimab appeared to be safe and well-tolerated with a potentially favorable safety profile as compared to the currently available anti-PD-1 therapies.
·CSCC enrollment to support a potential initial BLA submission for cosibelimab is ongoing.
·In November 2019, we announced that pharmacokinetic and target occupancy modeling data for cosibelimab were presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 34th Annual Meeting. The poster, entitled, "Semi-mechanistic PK and target-occupancy modeling to support dose justification for anti-PD-L1 clinical candidate CK-301 (TG-1501) in oncology patients," compares pharmacokinetic and tumor target occupancy data at steady state under various dosing regimens of cosibelimab to those of three marketed anti-PD-L1 monoclonal antibodies, atezolizumab, durvalumab and avelumab. The results demonstrate that cosibelimab dosed at 800 mg and 1200 mg once every two weeks or every three weeks is expected to achieve over 99% PD-L1 target occupancy throughout the dosing interval, which is comparable to atezolizumab and durvalumab, and higher than avelumab, at approved doses.
·Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc.

MB-101 (IL13Rα2-targeted CAR T cell therapy)

In October 2019, we announced that City of Hope received $4.1 million in grant awards for a clinical trial of MB-101 (IL13Rα2-targeted CAR T cell therapy) in combination with nivolumab (commercial name: Opdivo) and ipilimumab (commercial name: Yervoy) in patients with recurrent malignant glioma. The trial, which is now enrolling patients, is the first human study to combine IL13Rα2-targeted CAR T cell therapy with checkpoint inhibitors, as well as the first to locally deliver CAR T cells with systemic nivolumab combination treatment.

·MB-101 is currently in development at our partner company, Mustang Bio, Inc.

MB-103 (HER2-targeted CAR T cell therapy)

In August 2019, we announced that the California Institute for Regenerative Medicine ("CIRM") granted City of Hope $9.28 million to fund an ongoing Phase 1 clinical trial of MB-103 (HER2-targeted CAR T cell therapy) for the treatment of HER2-positive breast cancer with brain metastases.

·MB-103 is currently in development at our partner company, Mustang Bio, Inc.

MB-105 (Prostate Stem Cell Antigen (PSCA)-targeted CAR T cell therapy)

In September 2019, we announced that City of Hope opened and has begun to treat its first patients in a Phase 1 clinical trial of MB-105 (PSCA-targeted CAR T cell therapy) for the treatment of prostate cancer.

·MB-105 is currently in development at our partner company, Mustang Bio, Inc.

MB-106 (CD20-targeted CAR T cell therapy)

Fred Hutchinson Cancer Research Center will present a poster about the design of the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 (CD20-targeted CAR T cell therapy) for high-risk B-cell non-Hodgkin lymphomas at the 61st ASH (Free ASH Whitepaper) Annual Meeting, which will be held December 7-10, 2019, at the Orange County Convention Center in Orlando, FL. The abstract is available here.

·MB-106 is currently in development at our partner company, Mustang Bio, Inc.

MB-108 (Oncolytic Virus C134)

·In October 2019, we announced that the first participant was dosed in a Phase 1 clinical trial to determine the safety and efficacy of MB-108 (oncolytic virus C134), an attenuated herpes simplex virus type 1, in recurrent glioblastoma multiforme.
·MB-108 is currently in development at our partner company, Mustang Bio, Inc.

General Corporate

·In August 2019, we announced the appointment of Kevin L. Lorenz, J.D., to our Board of Directors.

TG Therapeutics Provides Business Update and Reports Third Quarter 2019 Financial Results

On November 12, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the third quarter ended September 30, 2019 and recent company developments (Press release, TG Therapeutics, NOV 12, 2019, View Source [SID1234551046]).

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "In the third quarter our team continued to execute on our core mission of developing combination therapies for patients with B-cell diseases. With the announcement of positive marginal zone lymphoma data earlier this year, and positive follicular lymphoma data only a few weeks ago, we have our first two foundational datasets for which we believe approval is in our reach. Adding on top of this, will be our UNITY-CLL Phase 3 study, which we are targeting a PFS readout around year-end or into the first quarter of next year. With these core pieces in place, our broader combination approach should come into focus, as we execute on a strategy to position U2 as an important stand-alone treatment as well as one that can improve outcomes when combined with other available therapies. We believe our recent ASH (Free ASH Whitepaper) abstracts contain some initial insights into the future of U2." Mr. Weiss continued, "With a pro forma cash position of approximately $100 million at the end of the third quarter, we believe we have sufficient capital resources through our next two major pivotal data releases including UNITY-CLL and the ULTIMATE MS Phase 3 trials."

Recent Developments and Highlights

ASH Presentations: Two triple therapy data abstracts were accepted for presentation at the upcoming 61stAmerican Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, including an oral presentation for the triple combination of U2 (umbralisib and ublituximab) plus venetoclax, and Phase 1 data for TG-1701, the Company’s novel BTK inhibitor, monotherapy and in combination with U2.
Positive Interim Data from FL Cohort of UNITY-NHL Trial: Positive interim data from the follicular lymphoma (FL) cohort of the UNITY-NHL trial was announced, with results meeting the Company’s prespecified ORR target. The Company plans to present the data at a future medical conference as well as discuss the data with the U.S. Food and Drug Administration (FDA).
GENUINE Progression Free Survival (PFS): Final long-term results from the Phase 3 GENUINE study demonstrated that ublituximab in combination with ibrutinib improved progression-free survival (PFS), as determined by Independent Review Committee (IRC).
U2 Published in Blood: Phase I/Ib combination trial of U2 was published in Blood, the Journal of the American Society of Hematology (ASH) (Free ASH Whitepaper).
Ublituximab Data in Multiple Sclerosis: Updated Phase 2 extension trial data for ublituximab in relapsing forms of multiple sclerosis (RMS), as well as the ULTIMATE I & II Phase 3 RMS program study design and demographic data was presented at the 35th Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).
Remaining 2019 and Early 2020 Milestones

Initiate a rolling New Drug Application (NDA) submission for umbralisib to treat adult patients with previously treated marginal zone lymphoma (MZL).
Report potential top-line PFS results from the Phase 3 UNITY-CLL trial evaluating U2 in patients with frontline and previously treated CLL.
Share results from the Phase 2b UNITY-NHL FL cohort with the FDA to determine potential filing opportunities.
Financial Results for the Three and Nine Months Ended September 30, 2019

R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation and non-cash in-licensing expense) was $56.5 million and $118.8 million for the three and nine months ended September 30, 2019, respectively, compared to $32.8 million and $98.7 million for the three and nine months ended September 30, 2018, respectively. The increase in R&D expense is primarily attributable to an increase in manufacturing expenses for Phase 3 clinical trials and potential commercialization (incurred during 2019) of $27 million and $31.6 million during the three and nine months ended September 30, 2019, respectively, as compared to prior periods. This was partially offset by a decrease in clinical trial expense of $3.8 million and $14.2 million for the three and nine months ended September 30, 2019, respectively, as compared to prior periods. We expect our other R&D expenses to decrease during the remainder of 2019 and into 2020 as our clinical trial expenses continue to decrease and the bulk of our CMC expenditures have been incurred during 2019.

G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $2.3 million and $6.6 million for the three and nine months ended September 30, 2019, respectively, as compared to $1.8 million and $6.2 million for the three and nine months ended September 30, 2018, respectively. Other G&A expenses remained consistent with the prior period, and we expect Other G&A expenses to increase modestly through the remainder of 2019.

Net Loss: Net loss was $61.9 million and $133.3 million for the three and nine months ended September 30, 2019, respectively, compared to a net loss of $34.0 million and $119.6 million for the three and nine months ended September 30, 2018, respectively. Excluding non-cash items, the net loss for the three and nine months ended September 30, 2019 was approximately $59.9 million and $127.5 million. Cash used in operations for the three months ended September 30, 2019 was approximately $33 million, as the payments of much of the increase in manufacturing expenses were deferred 12 months, having little impact on the quarter’s cash utilization.

Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $72.5 million as of September 30, 2019. Pro forma cash, cash equivalents and investment securities as of September 30, 2019 (excluding our fourth quarter 2019 operations) are approximately $96.3 million, after giving effect to $23.8 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the fourth quarter of 2019. The Company believes its cash, cash equivalents and investment securities on hand as of September 30, 2019, inclusive of the proceeds raised from the ATM facility subsequent to the third quarter, as well as future availability under the ATM facility, will be sufficient to fund the Company’s planned operations into the fourth quarter of 2020.
Conference Call Information

The Company will host a conference call today, November 12, 2019, at 8:30 am ET, to discuss the Company’s third quarter 2019 financial results and provide a business outlook for the remainder of 2019.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Third Quarter 2019 Business Update Call. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.