On August 9, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the second quarter ended June 30, 2019 and recent company developments (Press release, TG Therapeutics, AUG 9, 2019, View Source [SID1234538544]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "As we enter into the second half of the year, we are pleased with the progress made thus far and excited for the upcoming milestones. With a well-defined path to a regulatory submission for umbralisib in marginal zone lymphoma, we feel confident that we are at a pivotal point in our Company’s lifecycle." Mr. Weiss continued, "As we prepare for our first filing in MZL, we are also building a top-notch commercial organization which will fuel the long-term success of TG. And with pivotal data readouts also expected in CLL and MS over the next 6-12 months, we believe we are at the beginning of an extremely exciting transformational period for our Company."

Recent Developments and Highlights

Marginal Zone Lymphoma – Confirmed Registration Path: Announced confirmation of registration path to submit umbralisib for accelerated approval based on data from the marginal zone lymphoma (MZL) cohort of the UNITY-NHL Phase 2b trial.

Positive Interim Data from MZL Cohort of UNITY-NHL Trial: Presented positive interim data from the MZL cohort of the UNITY-NHL trial during oral presentations at the 55thAmerican Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting and the 2019 International Conference on Malignant Lymphoma (ICML).

Ublituximab Data in Multiple Sclerosis: Presented long-term follow-up data from the Phase 2 trial of ublituximab in patients with relapsing forms of multiple sclerosis (RMS) at the 5th Congress of the European Academy of Neurology (EAN).

Umbralisib Data in CLL and RT: Presented Phase 2 data of umbralisib in patients with chronic lymphocytic leukemia (CLL) who are intolerant to prior BTK or PI3k delta inhibitor therapy, and also presented data from patients with relapsed/refractory CLL or Richter’s Transformation (RT) treated with the triple combination of ublituximab, umbralisib and pembrolizumab.

TG-1801 Preclinical Data: Presented the first preclinical data of TG-1801, the Company’s first-in-class anti-CD47/CD19 bispecific antibody, at the 24th European Hematology Association (EHA) (Free EHA Whitepaper) annual congress.
Remaining 2019 and Early 2020 Milestones

Initiate a rolling New Drug Application (NDA) submission for patients with previously treated MZL

Present final data from the MZL cohort of the UNITY-NHL registration directed trial evaluating umbralisib in MZL

Potential top-line progression free survival (PFS) results from the Phase 3 UNITY-CLL trial evaluating U2 in patients with CLL

Present updated data from our pipeline products and combination studies at upcoming major medical conferences
Financial Results for the Three and Six Months Ended June 30, 2019

R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation and non-cash in-licensing expense) was $31.4 million and $62.3 million for the three and sixth months ended June 30, 2019 compared to $34.8 million and $66.0 million for the three and six months ended June 30, 2018. The decrease in other R&D expense is primarily attributable to a decrease in clinical trial expenses of $9.7 million and $10.4 million, respectively during the three and six months ended June 30, 2019, as compared to prior periods, offset by an increase in manufacturing and CMC expenses for Phase 3 clinical trials and potential commercialization of $7.9 million and $4.7 million, respectively during the three and six months ended June 30, 2019, as compared to prior periods. The current period decrease in other R&D expenses is primarily due to full enrollment in our pivotal Phase III clinical development programs completed in the prior period. We expect other R&D expenses to decrease through the remainder of 2019.

G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $2.3 million and $4.3 million for the three and six months ended June 30, 2019 as compared to $2.3 million and $4.4 million for the three and six months ended June 30, 2018. Other G&A expenses remained relatively flat compared to the prior period, and we expect Other G&A expenses to increase modestly through the remainder of 2019.

Net Loss: Net loss was $36.2 million and $71.4 million for the three and six months ended June 30, 2019, respectively, compared to a net loss of $44.1 million and $85.7 million for the three and six months ended June 30, 2018, respectively. Excluding non-cash items, the net loss for the three and six months ended June 30, 2019 was approximately $34.4 million and $67.6 million. We expect our quarterly net loss to decrease through the remainder of 2019.

Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $85.0 million as of June 30, 2019. Pro forma cash, cash equivalents and investment securities as of June 30, 2019 (excluding our third quarter 2019 operations) are approximately $96.6 million, after giving effect to $11.6 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the third quarter of 2019. The Company believes its cash, cash equivalents and investment securities on hand as of June 30, 2019, inclusive of the proceeds raised from the ATM facility subsequent to the second quarter, as well as future availability under the ATM facility, will be sufficient to fund the Company’s planned operations through the third quarter of 2020.
Conference Call Information

The Company will host a conference call today, August 9, 2019, at 8:30 am ET, to discuss the Company’s second 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 Second 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.

On August 9, 2019 Leap Therapeutics, Inc. (NASDAQ:LPTX), a biotechnology company developing targeted and immuno-oncology therapeutics, reported financial results for the second quarter ended June 30, 2019 (Press release, Leap Therapeutics, AUG 9, 2019, View Source [SID1234538543]).

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"We recently presented positive data from our clinical study of DKN-01 plus KEYTRUDA which demonstrated higher survival and objective response outcomes in patients with advanced gastroesophageal junction and gastric cancer whose tumors expressed high levels of DKK1 (DKK1-high). As we have seen with the single agent partial responses in patients with endometrial cancer, DKN-01’s activity continues to be impressive in biomarker-targeted patient populations," commented Christopher K. Mirabelli, Ph.D, President and Chief Executive Officer of Leap Therapeutics. "In addition, we have enrolled our first patients in the triple chemoimmunotherapy study of TRX518 in combination with BAVENCIO and cyclophosphamide."

Recent Developments

·DKN-01 CLINICAL INVESTIGATOR WEBCAST: On August 6, 2019, Leap hosted a DKN-01 program update webcast with Samuel J. Klempner, MD, Assistant Professor, Massachusetts General Hospital Cancer Center and Harvard Medical School, and Rebecca C. Arend, MD, Assistant Professor and Associate Scientist, Gynecologic Oncology Clinic, UAB Comprehensive Cancer Center Experimental Therapeutics Program. A replay of the webcast and the presentation slides are available under "Events & Presentations" in the Investor section of Leap’s website, View Source

· DKN-01 in ESOPHAGOGASTRIC CANCER: Leap presented data from the KEYNOTE-731 clinical study evaluating DKN-01 in combination with KEYTRUDA in patients with advanced esophagogastric cancer. In gastroesophageal junction and gastric cancer patients who had not received prior PD-1/PD-L1 therapy, the combination of DKN-01 plus KEYTRUDA demonstrated improved outcomes in patients whose tumors are DKK1-high. DKK1-high patients experienced 22.1 weeks median progression free survival (PFS) and 31.6 weeks median overall survival (OS), with a 50% overall response rate (ORR) and 80% disease control rate (DCR) in ten evaluable patients. DKK1-low patients experienced 5.9 weeks PFS and 17.4 weeks OS, with a 20% DCR in fifteen evaluable patients. PD-L1 Combined Positive Scores (CPS) did not predict efficacy to the combination of DKN-01 plus KEYTRUDA. In multi-variate analysis, DKK1-high status correlated with longer PFS independent of PD-L1 CPS.

·DKN-01 in GYNECOLOGICAL CANCERS: The clinical study of DKN-01 as a monotherapy and in combination with paclitaxel in patients with advanced gynecological cancers has been expanded to include focused cohorts of patients with carcinosarcoma. Overall, ninety-six patients have been enrolled in the study, and enrollment is ongoing. Additional response and biomarker data will be available in September at the International Gynecologic Cancer Society Annual Global Meeting.

·TRX518 TRIPLE COMBINATION THERAPY: Leap enrolled the first patients in the clinical trial evaluating TRX518 in combination with cyclophosphamide chemotherapy and BAVENCIO. Dose escalation in the study is ongoing.

· $21 MILLION EQUITY COMMITMENT FACILITY: Leap entered into purchase agreements with Lincoln Park Capital Fund, LLC (LPC) pursuant to which Leap sold $1 million in common stock and has the option, but not the obligation, to sell to LPC up to an additional $20 million in shares of common stock in tranches over a twenty-four month period. The price of shares sold will be based on the market prices prevailing at the time of each sale to LPC. There is no upper limit as to the price per share that LPC may pay for future stock issuances under the agreement, and Leap will control the timing and amount of any future sales.

Selected Second Quarter 2019 Financial Results

Net loss was $8.4 million for the second quarter 2019, compared to $7.4 million for the same period in 2018. This increase was primarily due to an increase in clinical development expense and the recording of a loss in the second quarter 2018 as a result of a decrease in the fair value of the warrant liability.

Research and development expenses were $6.1 million for the second quarter 2019, compared to $4.2 million for the same period in 2018. This increase was primarily due to an increase in clinical trial expenses associated with an increase in patient enrollment and an increase in consulting fees and payroll expenses, partially offset by a decrease in manufacturing costs related to clinical trial material.

General and administrative expenses were $2.3 million for the second quarter 2019, compared to $2.6 million for the same period in 2018. This decrease was primarily due to a decrease in legal, audit and consulting fees.

Cash, cash equivalents and marketable securities totaled $15.7 million at June 30, 2019. Research and development incentive receivables, current and long term, totaled approximately $1.0 million at June 30, 2019. Subsequent to the end of the quarter, Leap announced the commitment facility with LPC and sold $1.0 million in common stock.

On August 9, 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 second quarter ended June 30, 2019 (Press release, Fortress Biotech, AUG 9, 2019, View Source [SID1234538541]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We are pleased to have achieved several important milestones in the second quarter, including positive data for three of our late-stage product candidates in development across our partner companies: IV tramadol for post-surgical pain management; MB-107 gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID); and cosibelimab, an anti-programmed death ligand-1 (PD-L1) antibody for the treatment of multiple advanced cancers. Looking ahead to the remainder of 2019, we anticipate multiple potentially value-creating catalysts, including a New Drug Application filing for IV tramadol and additional important clinical data readouts for many of our product candidates. Our world-class business development team continues to focus on expanding our diverse pipeline with additional high-quality biotech and specialty pharmaceutical assets, further de-risking our product portfolio."

Financial Results:

·As of June 30, 2019, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), and restricted cash totaled $170.5 million, compared to $137.5 million as of March 31, 2019, and $99.2 million as of December 31, 2018, an increase of $33.0 million for the quarter and an increase of $71.3 million year-to-date.
·Fortress’ net revenue totaled $9.3 million for the second quarter of 2019, compared to $6.8 million for the second quarter of 2018.
· Research and development expenses were $18.5 million for the second quarter of 2019, of which $18.0 million was related to Fortress partner companies. This compares to $17.5 million for the second 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 $0.8 million for both the second quarter of 2019 and 2018.
·Research and development expenses from license acquisitions totaled $0.2 million for the second quarter of 2019, compared to a nominal amount for the second quarter of 2018.
·General and administrative expenses were $13.4 million for the second quarter of 2019, of which $9.3 million was related to Fortress partner companies. This compares to $13.1 million for the second quarter of 2018, of which $7.7 million was related to Fortress partner companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.6 million for the second quarter of 2019, compared to $2.4 million for the second quarter of 2018.
·Net loss attributable to common stockholders was $13.1 million, or $0.24 per share, for the second quarter of 2019, compared to a net loss attributable to common stockholders of $21.6 million, or $0.50 per share, for the second quarter of 2018. For the first six months of 2019, net loss attributable to common stockholders was $11.7 million or $0.23 per share, compared to $42.6 million or $0.99 per share for the first six months of 2018.

Recent Corporate Highlights1:

Marketed Dermatology Products

·In the second quarter of 2019, our marketed products generated net revenue of $8.2 million, compared to $6.7 million in the second quarter of 2018.
· We are anticipating the launch of a second prescription oral antibiotic drug for acne during the current quarter, Q3 2019.
·This new asset, coupled with our salesforce expansion to 34 territory managers, will allow us to reach over 5,000 dermatologists across the country. This combination is expected to fuel the growth of our dermatology portfolio in 2019 and beyond.
·Our dermatology products are marketed by our partner company, Journey Medical Corporation.

IV Tramadol

·In June 2019, we announced that our second pivotal Phase 3 trial of IV tramadol achieved the primary endpoint of a statistically significant improvement in Sum of Pain Intensity Difference over 24 hours (SPID24) compared to placebo in patients with postoperative pain following abdominoplasty surgery. In addition, the trial met all of its key secondary endpoints. The study also included a standard-of-care IV opioid as an active comparator, IV morphine 4 mg. In this study, IV tramadol also demonstrated similar efficacy and safety to that of IV morphine.
·IV Tramadol is currently in development at our partner company, Avenue Therapeutics, Inc.

MB-102 (CD123 CAR T)

·In July 2019, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of acute myeloid leukemia (AML).
·In August 2019, we announced that the FDA has approved the Investigational New Drug (IND) application to initiate a multicenter Phase 1/2 clinical trial of MB-102 (CD123 CAR T) in AML, blastic plasmacytoid dendritic cell neoplasm (BPDCN) and high-risk myelodysplastic syndrome (MDS).
·MB-102 is currently in development at our partner company, Mustang Bio, Inc.

MB-108 (Oncolytic Virus C134)

·In May 2019, the FDA granted Orphan Drug Designation to MB-108 (oncolytic virus C134) for the treatment of malignant glioma, a type of brain cancer with a median survival of less than 18 months.
·MB-108 is currently in development at our partner company, Mustang Bio, Inc.

On August 9, 2019 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported second quarter 2019 financial results and business updates, which will be discussed on a conference call scheduled for Monday, August 12 at 10:00 a.m. ET (Press release, Abeona Therapeutics, AUG 9, 2019, View Source [SID1234538540]). Interested parties are invited to participate in the call by dialing 844-369-8770 (toll-free domestic) or 862-298-0840 (international) or via webcast at View Source

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"The second quarter was highlighted by progress made in both of our MPS III programs," said João Siffert, M.D., Chief Executive Officer. "Data from our Transpher A study showed that children with MPS IIIA who were treated early with ABO-102 preserved neurocognitive development within the normative range 12-18 months post treatment. Our MPS IIIB program has also progressed, with enrollment of additional patients in cohort 2 of the Transpher B study. Our team remains highly focused on our lead programs, including the start of our VIITAL Phase 3 clinical trial in recessive dystrophic epidermolysis bullosa, continued enrollment in the MPS III programs, and preparations to start the clinical trial in CLN1 disease."

Second Quarter Financial Results:

Cash, cash equivalents and marketable securities as of June 30, 2019, were $62.5 million compared to $68.3 million as of March 31, 2019. The decrease in cash was driven primarily by the net cash used in operating activities of $15.2 million.

Research and development expenses for the second quarter ended June 30, 2019 were $16.3 million compared to $7.9 million in the same period of 2018. The increase in R&D expense was primarily attributable to increased in-house manufacturing activities and related headcount costs.

General and administrative expenses for the second quarter ended June 30, 2019 were $5.6 million compared to $4.6 million in the same period of 2018. The increase in G&A expenses was primarily due to increased headcount and related facility costs.

Net loss was $0.49 per share for the second quarter of 2019 compared to $0.26 per share in the same period of 2018.

Second Quarter and Recent Highlights:

-July 25, 2019: Announced positive interim data from the Phase 1/2 AAV9 gene therapy clinical trial in MPS IIIA showing preservation of neurocognitive function for the three youngest patients treated with ABO-102, as well as robust and sustained improvements in biomarkers of the disease. No product-related serious adverse events were reported to date.
-June 26, 2019: Appointed Dr. Victor Paulus as Senior Vice President of Regulatory Affairs and Jodie Gillon as Vice President of Patient Advocacy and Clinical Affairs
-June 18, 2019: Received FDA Fast Track Designation for ABO-202 AAV9 gene therapy in CLN1 disease
-May 21, 2019: Announced FDA clearance of Investigational New Drug application for ABO-202 AAV9 gene therapy in CLN1 disease
-May 14, 2019: Announced treatment of first patient in second cohort of Phase 1/2 clinical trial for ABO-101 AAV9 gene therapy in MPS IIIB
-May 1, 2019: Reported preclinical data demonstrating broad therapeutic potential of AIM gene therapy in retinal diseases at Association for Research in Vision and Ophthalmology Annual Meeting
-April 30, 2019: Reported preclinical data demonstrating therapeutic potential of ABO-401 for treatment of cystic fibrosis at American Society of Gene and Cell Therapy annual meeting
-April 4, 2019: Received FDA Fast Track Designation for ABO-101 AAV9 gene therapy for MPS IIIB

Steven H. Rouhandeh, Abeona’s Executive Chairman, said, "Abeona has continued the development of its breakthrough gene and cell therapies for rare genetic diseases through 2019 with important regulatory and clinical achievements secured. We look forward to progressing our MPS programs, and to starting of our Phase 3 VIITAL trial in EB before year end."

On August 9, 2019 Eli Lilly and Company (NYSE: LLY) reported that the U.S. Court of Appeals for the Federal Circuit ruled in favor of Lilly, confirming that the Alimta (pemetrexed for injection) vitamin regimen patent would be infringed by competitors that had stated their intent to market alternative salt forms of pemetrexed prior to the patent’s expiration in May 2022 (Press release, Eli Lilly, AUG 9, 2019, View Source [SID1234538538]).

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The ruling came in the appeals of U.S. District Court decisions in the cases of Eli Lilly and Company v. Dr. Reddy’s Laboratories and Eli Lilly and Company v. Hospira, Inc. Previous rulings in Lilly’s favor had precluded the generic companies from launching the alternative salt forms until the patent expires.

If the patent is ultimately upheld through all remaining challenges, Alimta would maintain U.S. exclusivity until May 2022, preventing marketing of generic products for as long as the patent remains in force.

"We’re pleased with this decision," said Michael J. Harrington, Lilly’s senior vice president and general counsel. "Lilly’s extensive research to discover this patent deserves intellectual property protection, which has been confirmed in every challenge in the U.S. to date."

On June 22, 2018, Lilly announced that the U.S. District Court for the Southern District of Indiana ruled in favor of Lilly that the Alimta vitamin regimen patent would be infringed by the use of Dr. Reddy’s alternative salt form of pemetrexed prior to the patent’s expiration. The district court found the generic product would infringe under the doctrine of equivalents.

In a separate decision on June 15, 2018, the District Court also ruled in favor of Lilly in the case of Eli Lilly and Company v. Hospira, Inc. denying Hospira’s motion for summary judgement and granting Lilly’s cross-motion for summary judgment.

Both Dr. Reddy’s and Hospira had appealed the district court’s decisions, leading to today’s ruling.

In March 2014, the U.S. Court for the Southern District of Indiana upheld the validity of the vitamin regimen patent. In August 2015, the same court ruled in Lilly’s favor regarding infringement of the vitamin regimen patent. The U.S. Court of Appeals for the Federal Circuit confirmed these rulings in a unanimous decision in January 2017, finding the patent is valid and would be infringed by the generic challengers’ proposed products.

Separately, Lilly announced in April 2019 that the U.S. Court of Appeals for the Federal Circuit ruled in the company’s favor regarding patentability of the vitamin regimen for Alimta, upholding an October 2017 decision by the Patent Trial and Appeal Board of the U.S. Patent and Trademark Office.