On November 09, 2015 TG Therapeutics, Inc. (NASDAQ:TGTX) reported its financial results for the third quarter ended September 30, 2015 and recent company developments (Press release, TG Therapeutics, NOV 9, 2015, View Source [SID:1234508143]).

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Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "During the third quarter, we achieved another major milestone for the Company in obtaining a Special Protocol Assessment for our UNITY-CLL trial, a study evaluating the safety and efficacy of our proprietary ‘1303′ combination regimen in patients with front-line as well as previously treated CLL. This is a very important and exciting clinical trial for the Company, as it represents our first pivotal trial for our proprietary combination and, if successful, should provide a broad approval in CLL offering patients in both first-line and relapsed/refractory setting, a novel, non-chemotherapy treatment option. Further, it would provide us a broad label for building additional three and, possibly, four drug proprietary combinations to further improve outcomes for patients with CLL. With Phase 3 programs in oncology now underway for both TG-1101 and TGR-1202, we’re excited to begin exploring the potential of our pipeline products for the treatment of autoimmune disease, an area where B-cell targeted therapies have proven highly effective, and anticipate commencing our first trial in Multiple Sclerosis in the near-term." Mr. Weiss continued, "We also remain focused on aggressively enrolling into our ongoing GENUINE Phase 3 clinical trial, and expect top-line data from this study in the second half of 2016. Finally, from a financial perspective, with more than $115 million in cash and investments we have enough cash to execute on our business plan."

Recent Developments and Highlights

In September 2015, we announced a Special Protocol Assessment (SPA) agreement with the FDA for the first Phase 3 clinical trial of our proprietary combination regimen of TG-1101 (ublituximab) with TGR-1202 ("1303") for patients with chronic lymphocytic leukemia, the UNITY-CLL study.
In September 2015, we announced the initiation of a Phase 1/2 clinical trial investigating the use of TG-1101 and TGR-1202 in combination with pembrolizumab, the anti-PD-1 immune checkpoint inhibitor, in patients with relapsed or refractory CLL, the first clinical trial evaluating the safety, tolerability and effectiveness of the triple combination of a PI3K delta inhibitor with an anti-CD20 mAb and an anti-PD-1 checkpoint inhibitor.

Goals/Objectives for the Remainder of 2015

Continue to aggressively recruit into the GENUINE Phase 3 Clinical Trial of TG-1101 in combination with ibrutinib
Enroll the first patient by year end in our UNITY-CLL Phase 3 clinical trial of TG-1101 plus TGR-1202 in front-line and relapsed/refractory CLL
Announce our next registration trial evaluating 1303 in patients with NHL
Continue to recruit into the triple combination cohort of 1303 plus ibrutinib as well as the triple combination study of 1303 plus pembrolizumab, as well as seek to evaluate additional novel triple combinations of interest
Expand into autoimmune diseases with the first Phase 2 trial in Multiple Sclerosis to commence in the near-term
Continue to advance our pre-clinical compounds, including IRAK4, anti PD-L1 and anti-GITR forward towards clinical development
Continue to seek additional compounds to further complement our current portfolio

Financial Results for the Third Quarter 2015

At September 30, 2015 the Company had cash, cash equivalents, investment securities, and interest receivable of $115.4 million, which includes approximately $67.0 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the year (all of which was previously disclosed in connection with our last quarterly update), as compared to December 31, 2014 when we had $78.9 million.

Our consolidated net loss for the third quarter ended September 30, 2015, excluding non-cash items, was approximately $12.4 million, which included approximately $6.9 million of manufacturing and CMC expenses in preparation for Phase 3 clinical trials and potential commercialization. The consolidated net loss for the third quarter ended September 30, 2015, inclusive of non-cash items, was $13.7 million, or $0.28 per diluted share, compared to a consolidated net loss of $17.5 during the comparable quarter in 2014, representing a decrease in consolidated net loss of $3.8 million. The decrease in consolidated net loss during the third quarter ended September 30, 2015 was primarily the result of $8.1 million of expense ($4.1 million of which was non-cash stock expense) recorded during the quarter ended September 30, 2014 in conjunction with the Company’s licensing agreement for TGR-1202, and a $2.9 million decrease in non-cash compensation expense related to equity incentive grants over the comparable period in 2014. Partially offsetting the aforementioned decreases, other research and development expenses for TG-1101 and TGR-1202 increased $4.8 million and $2.1 million, respectively, over the comparable period in 2014.

Our consolidated net loss for the nine months ended September 30, 2015, excluding non-cash items, was approximately $32.5 million, which included approximately $16.0 million of manufacturing and CMC expenses in preparation for Phase 3 clinical trials and potential commercialization. The consolidated net loss for the nine months ended September 30, 2015, inclusive of non-cash items, was $45.3 million, or $1.01 per diluted share, compared to a consolidated net loss of $37.0 million during the comparable period in 2014, representing an increase in consolidated net loss of $8.3 million. The increase in consolidated net loss during the nine months ended September 30, 2015 was primarily the result of other research and development expenses for TG-1101 and TGR-1202 increasing approximately $14.6 million and $4.5 million, respectively, over the comparable period in 2014. This was offset by $9.3 million of expense ($5.3 million of which was non-cash stock expense) recorded in conjunction with the Company’s licensing agreements for TGR-1202 and the IRAK4 inhibitors program during the nine months ended September 30, 2014, and a decrease of $3.2 million in non-cash compensation expense related to equity incentive grants over the comparable period in 2014.

(Filing, 10-Q, Cleveland BioLabs, NOV 9, 2015, View Source [SID:1234508153])

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On November 05, 2015 Caladrius Biosciences, Inc. (NASDAQ:CLBS) ("Caladrius"), a company combining a leading cell therapy service provider with a development pipeline including a Phase 3 clinical program in immuno-oncology and a portfolio of projects in immune modulation and ischemic repair, reported financial results for the three months ended September 30, 2015 (Filing, 8-K, Caladrius Biosciences, NOV 9, 2015, View Source [SID:1234508122]).

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Business Highlights

• Total revenues for the third quarter of 2015 increased 43% to $5.9 million when compared with $4.1 million in the year-ago third quarter, driven by increases in Clinical Service revenue at the Company’s PCT subsidiary, an external development and manufacturing partner for the growing cell therapy industry

• PCT entered into a long-term dedicated capacity agreement with IRX Therapeutics, Inc. to support their anticipated clinical trial manufacturing needs including the manufacture of EU-compliant cell therapy product, adding to a growing list of immuno-oncology clients with similar arrangements including Kite Pharma and ImmunoCellular Therapeutics

• Continued patient enrollment in a Phase 3 clinical study of lead product candidate CLBS20 for the treatment of recurrent Stage III or Stage IV metastatic melanoma

• Presented data elucidating the mechanism of action of CLBS20, suggesting correlations between a distinct immune response triggered by CLBS20 and overall survival of melanoma patients

• Received an initial award of $300,000 out of a potential award of up to $2.3 million from the National Cancer Institute to fund the first phase of a project for process optimization of CLBS20

• Entered into a collaboration agreement with Sanford Research to develop CLBS03, the Company’s T regulatory cell therapy product candidate, for the treatment of adolescents with recent-onset type 1 diabetes, which the Company expects to advance to a Phase 2 clinical study in early 2016

• Promoted Joseph Talamo to Chief Financial Officer and Todd Girolamo to General Counsel.

Management Commentary

"PCT continues to post strong revenue growth, which we expect to continue as the immuno-oncology sector is experiencing industry-wide advances and there are increasing numbers of such products on the market and in development. We expect PCT to continue to capitalize on its unmatched cell development and manufacturing expertise as industry demand for cell therapy services continues to grow," said David J. Mazzo, Ph.D., Chief Executive Officer of Caladrius. "In addition, we continued to advance our own clinical development programs in immuno-oncology (CLBS20) and immune modulation (CLBS03), while maintaining financial discipline that allowed us to lower our cash burn."

2015 Third Quarter Financial Highlights
Total revenue for the third quarter of 2015 increased 43% to $5.9 million compared with $4.1 million for third quarter of 2014, primarily due to higher reported Clinical Services revenues at PCT.

Research and development expenses in the third quarter of 2015 decreased 24% to $6.3 million compared with $8.5 million for the third quarter of 2014. The decrease was primarily related to lower costs associated with the Company’s ischemic repair and immune modulation programs. The lower costs were partially offset by increased expenses associated with the Intus Phase 3 clinical trial that initiated in 2015.

Selling, general and administrative expenses were approximately $5.1 million for the third quarter of 2015 compared with $7.9 million for same period in 2014. The decrease was primarily due to lower equity-based compensation expenses in the current quarter compared with a year ago and, to a lesser extent, lower expenses associated with other general and administrative activities.

The net loss for the third quarter of 2015 was approximately $11.4 million or $0.21 per share, compared with a net loss of $17.2 million or $0.48 per share for same period in 2014.

As of September 30, 2015, Caladrius had cash, cash equivalents and marketable securities of $29.4 million.

On November 9, 2015 OPKO Health, Inc. (NYSE:OPK), a multinational biopharmaceutical and diagnostics company, reported financial and operating results for the three and nine months ended September 30, 2015 (Press release, Opko Health, NOV 9, 2015, View Source [SID:1234508144]).

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Business Highlights

Completed the Acquisition of Bio-Reference Laboratories on August 20, 2015: Bio-Reference Laboratories is the third largest full-service clinical laboratory in the United States and is known for its innovative technological solutions and pioneering leadership in the areas of genomics and genetic sequencing. Through GeneDx, Bio-Reference Laboratories’ genetic sequencing laboratory, and GenPath Diagnostics, its Oncology and Women’s Health business units, Bio-Reference Laboratories has accumulated a vast array of genetic and genomic data that OPKO will make available to industry and academic scientists to enhance their drug discovery and clinical trial programs. Since closing, OPKO has begun to leverage the national marketing, sales and distribution resources of Bio-Reference Laboratories to enhance sales of OPKO’s 4Kscore test, a blood test that provides a personalized risk score for aggressive prostate cancer, and plans to further leverage the Bio-Reference capabilities with OPKO’s other diagnostic products under development.

4Kscore Recommended in National Comprehensive Cancer Network Guidelines for Prostate Cancer Early Detection: The National Comprehensive Cancer Network (NCCN) included 4Kscore as a recommended test in their 2015 Guidelines for Prostate Cancer Early Detection. The panel making this recommendation concluded that the 4Kscore, as a blood test with greater specificity over the PSA test, is indicated for use prior to a first prostate biopsy, or after a negative biopsy, to assist patients and physicians in further defining the probability of high-grade cancer.

Rayaldee PDUFA Date is March 29, 2016: In late 2014, OPKO announced successful top-line results from both pivotal Phase 3 trials with Rayaldee. These trials were identical randomized, double-blind, placebo-controlled, multisite studies intended to establish the safety and efficacy of Rayaldee as a new treatment for secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency.

Completed Enrollment in Ongoing Phase 3 Trial in Growth Hormone Deficient Adults: The trial is designed to evaluate the safety and efficacy of hGH-CTP with a primary endpoint of superiority compared with placebo in decreasing fat mass in adults with GHD. The trial is a randomized, double-blind, placebo-controlled, multicenter, global study in adults with GHD. The study is divided into two treatment periods: a 26-week, double-blind, placebo-controlled period, followed by a 26-week, open-label extension period. The study is expected to conclude in the second half of 2016; with positive results, a regulatory submission to the FDA will follow study completion.

IND for Long-Acting Factor VIIa-CTP for Hemophilia Filed and Accepted: In March 2015, the FDA accepted OPKO’s IND application to initiate a Phase 2a trial for its long-acting intravenous coagulation Factor VIIa-CTP to treat hemophilia. Clinical trials are expected to commence during Q4 2015.

Clinical Studies for Long-Acting Oxyntomodulin for Obesity and Diabetes Expected to Begin During 2016: OPKO expects to commence studies for its long-acting subcutaneous oxyntomodulin for diabetes and obesity in Q1 2016.

VARUBITM (Rolapitant) was Approved by the FDA on September 2, 2015 and Commercial Launch is Expected to Commence this Month: OPKO’s partner, Tesaro received FDA approval of oral VARUBI, a neurokinin-1 (NK-1) receptor antagonist, in combination with other antiemetic agents in adults for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic chemotherapy. Tesaro expects to commence commercial sales in the U.S. this month. VARUBI has been included in the NCCN Guidelines as a recommended option in combination with other antiemetic agents for patients receiving both high emetic risk intravenous chemotherapy (HEC) and moderate emetic risk intravenous chemotherapy (MEC). Category 1, the highest level category of evidence and consensus, was granted to VARUBI for both HEC and MEC chemotherapy. Following commercialization, OPKO is eligible to receive up to $110 million in additional milestones and tiered, double-digit royalties.

"OPKO has already achieved numerous important milestones during 2015," said Phillip Frost, M.D., Chairman and CEO. "We believe that the Pfizer transaction for hGH-CTP and the acquisitions of EirGen and Bio-Reference Laboratories have had a positive impact on our financial operations and will provide significant revenue opportunities and an expanded commercial platform for us going forward. The addition of our 4Kscore Test to the NCCN guidelines was an important step toward obtaining reimbursement from healthcare payors, a key factor for obtaining broad access to men for the test. Our NDA filing for Rayaldee continues to advance through the FDA drug approval process and we have high expectations for our new treatment option for patients with stage 3 or 4 chronic kidney disease and secondary hyperparathyroidism. Our clinical development programs for Factor VIIa-CTP and oxyntomodulin, each with great commercial potential, are advancing on plan and we expect to initiate human trials for both products in the near future," continued Dr. Frost.

Financial Highlights

Consolidated revenues increased to $143.0 million from $19.8 million for the three months ended September 30, 2015 compared to three months ended September 30, 2014, and increased to $215.5 million from $65.6 million for the nine months ended September 30, 2015 as compared to the 2014 period. The 2015 periods include revenue from Bio-Reference and EirGen beginning with their acquisitions in August and May 2015, respectively. Revenue for the three and nine months ended September 30, 2015 also includes $17.7 million and $47.8 million, respectively, from OPKO’s collaboration with Pfizer.

Net income for the three months ended September 30, 2015 was $128.2 million compared with net loss of $48.7 for the 2014 period and net losses for the nine months ended September 30, 2015 decreased to $31.6 million compared and $118.7 million for the 2014 period. The 2015 three and nine month periods include significant non-recurring and/or non-cash activities, including:
$93.0 million and $87.2 million of income tax benefit reflecting the release of valuation allowances against all of OPKO’s U.S.-based deferred tax assets as a result of the Bio-Reference acquisition in the three and nine month periods of 2015, respectively;

17.3 million gain related to the deconsolidation of OPKO’s previously consolidated variable interest entity, SciVac, in the three month period of 2015 as SciVac completed an initial public offering by merger with Levon Resources Ltd. in July 2015;

$25.9 million of non-recurring operating expense related to the repayment of a grant to the Office of the Chief Scientist in Israel related to the Pfizer transaction in the nine month period of 2015; and
,
Other income and (expense) of $32.2 million and ($34.1) million related to the change in fair value of derivative instruments in the three and nine months of 2015, respectively, compared with $3.3 million and $3.8 million in the 2014 periods. The change in fair value is principally related to an embedded derivative in our January 2013 convertible senior notes due in 2033.

Cash, cash equivalents and marketable securities were $212.1 million as of September 30, 2015.

This reflects receipt of Pfizer upfront payments of $295.0 million, partially offset by a $94.7 million cash payment for the acquisition of EirGen (net of EirGen’s cash on hand) and a one-time $25.9 million payment to the Office of the Chief Scientist in Israel related to the Pfizer transaction.

(Filing, 10-Q, Galena Biopharma, NOV 9, 2015, View Source [SID:1234508155])

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